Cocrystal Pharma Reports Third Quarter Financial Results and Provides an Update on Development Programs and Milestones

 

• Submitted pre-IND briefing package to the FDA for intranasal/pulmonary CDI-45205 for the treatment of COVID-19 and sets goal of initiating IND-enabling study and Phase 1 in 2022
• Advance oral COVID-19 program with goal of initiating IND-enabling study and Phase 1 in 2022
• Received regulatory clearance to initiate a Phase 1 trial in Australia with orally administered CC-42344 for the treatment of pandemic and seasonal influenza A
  

BOTHELL, Wash., Nov. 15, 2021 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”), a clinical-stage biotechnology company, reports financial results for the three and nine months ended September 30, 2021, and provides updates on its antiviral pipeline, upcoming milestones and business activities.

“Today we are reporting progress with our promising COVID-19 antiviral development programs along with setting the goal of beginning first-in-human clinical studies with our novel SARS-CoV-2 protease inhibitors in 2022,” said Sam Lee, Ph.D., co-interim CEO and President of Cocrystal. “We expect comments from the U.S. Food and Drug Administration (FDA) by mid-December on our pre-IND briefing package for our intranasal/pulmonary-delivered SARS-CoV-2 protease inhibitor CDI-45205, our most advanced SARS-CoV-2 program. The FDA’s comments will be useful in designing the Phase 1 and Phase 2 clinical trial protocols for this program.

“Under our second SARS-CoV-2 program, this one targeting oral administration, by year-end 2021 we expect to select a lead candidate from novel SARS-CoV-2 protease inhibitors discovered using our proprietary structure-based technology,” he added. “We will then prepare a pre-IND briefing package to submit to the FDA with the goal of initiating a clinical trial in 2022. Work also continues with a third COVID-19 program using our platform to discover oral replication inhibitors for the treatment of COVID-19.

“Regarding our influenza programs, our supplier is manufacturing our novel PB2 inhibitor CC-42344 for pandemic and seasonal influenza A. We expect patient enrollment in our Phase 1 trial to begin in early 2022 with data readouts later that same year,” said Dr. Lee. “With the influenza A/B program, we anticipate providing an update on the development of the compounds jointly discovered with Merck using our technology platform in the first quarter 2022.”

“With the proceeds from financings completed earlier this year and a clean balance sheet, we believe our capital is sufficient to fund planned operations. This outlook includes our goal of having multiple high-value antiviral compounds in clinical development next year as we advance our programs toward commercialization,” said James Martin, co-interim CEO and CFO. “We are aggressively pursuing the advancement of these potent compounds as we navigate a challenging global supply chain. Thankfully our strong partnership with CROs has benefited us to that extent.”

Antiviral Pipeline Overview

COVID-19 and Other Coronavirus Programs

• Intranasal/Pulmonary Protease Inhibitor
  • Our lead therapeutic molecule CDI-45205 was among the broad-spectrum viral protease inhibitors obtained from Kansas State University Research Foundation (KSURF) under an exclusive license agreement announced in 2020. We believe the protease inhibitors obtained from KSURF have the ability to inhibit the inactive SARS-CoV-2 polymerase replication enzymes into an active form.
  • CDI-45205 and several analogs showed potent in vitro activity against the SARS-CoV-2 Delta (India/B.1.617.2), Gamma (Brazil/P.1), Alpha (United Kingdom/B.1.1.7) and Beta (South African/B.1.351) variants, surpassing the activity observed with the original or wild-type Wuhan strain.
  • CDI-45205 demonstrated good bioavailability in mouse and rat pharmacokinetic studies via intraperitoneal injection, and no cytotoxicity against a variety of human cell lines. CDI-45205 also demonstrated a strong synergistic effect with the FDA-approved COVID-19 medicine remdesivir.
  • A proof-of-concept animal study demonstrated that daily injection of CDI-45205 exhibited favorable in vivo efficacy in mice infected with MERS-CoV-2.

• Oral Protease Inhibitors
  • Using our drug discovery platform, we discovered and are developing novel SARS-CoV-2 3CL protease inhibitors and anticipate identifying a preclinical 3CL lead for oral administration by the end of 2021.
  • We plan to submit a pre-IND briefing package to the FDA in the first half of 2022 to support an IND application, with the goal of progressing to IND-enabling study and Phase 1 in late 2022.
  • The main SARS-CoV-2 protease inhibitors showed potent in vitro pan-viral activity against human common coronaviruses, rhinoviruses, and respiratory enteroviruses that frequently cause the common cold, as well as against noroviruses that can cause symptoms of acute gastroenteritis.

• Replication Inhibitors
  • We are using our platform to seek to discover replication inhibitors for developing orally administered therapeutic and prophylactic treatments for SARS-CoV-2. Replication inhibitors have the potential to work with protease inhibitors in a combination therapy regimen.

Influenza Programs

• Pandemic and Seasonal Influenza A
  • Our novel PB2 inhibitor CC-42344 has shown excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as strains resistant to Tamiflu and Xofluza. CC-42344 also has favorable pharmacokinetic and drug-resistance profiles.
  • We have completed preclinical IND-enabling studies with CC-42344 and have received clearance from the Australian Human Research Ethics Committees (HREC) to initiate a Phase 1 clinical trial. Our supplier is manufacturing drug and we expect to begin patient enrollment in the first quarter of 2022.

• Pandemic and Seasonal Influenza A/B program
  
  • In January 2019 we entered into an Exclusive License and Research Collaboration Agreement with Merck Sharp & Dohme Corp. to discover and developed certain proprietary influenza A/B antiviral agents that are effective against both influenza A and B strains. This agreement includes milestone payments of up to $156 million plus royalties on sales of products discovered under the agreement.
  • In January 2021 we announced completion of all research obligations under the agreement. Merck is now solely responsible for further preclinical and clinical development of the influenza A/B antiviral compounds discovered under this agreement. We anticipate providing a program update during the first quarter of 2022.

• The World Health Organization (WHO) estimates there are approximately 1 billion cases of influenza annually worldwide, resulting in 3 million to 5 million cases of severe illness and 290,000 to 650,000 deaths. The Centers for Disease Control and Prevention (CDC) estimates that since 2010 influenza has caused 9 million to 45 million illnesses in the U.S. annually, resulting in 140,000 to 810,000 hospitalizations and 12,000 to 61,000 deaths each year.
  

Norovirus Program

• We are developing certain proprietary broad-spectrum antiviral compounds to treat norovirus infections.
• Norovirus is a global public health problem responsible for nearly 90% of epidemic, non-bacterial outbreaks of gastroenteritis around the world.

Hepatitis C Program

• We are seeking a partner to advance the development of CC-31244 following completion of a Phase 2a trial. This compound has shown favorable safety and preliminary efficacy in a triple regimen Phase 2a study in combination with Epclusa (sofosbuvir/velpatasvir) for the ultra-short duration treatment of individuals infected with the hepatitis C virus (HCV).
• HCV is a viral infection of the liver that causes both acute and chronic infection. According to the WHO, in 2017 an estimated 71 million people worldwide had chronic HCV infection, including 3.5 million in the U.S. Approximately 399,000 people die each year from HCV infection, mostly from cirrhosis and hepatocellular carcinoma.

Third Quarter Financial Results

Throughout 2020 Cocrystal reported quarterly revenues under an influenza A/B collaboration with Merck consisting of research and development (R&D) services performed by Cocrystal and reimbursed by Merck.

As discussed above, in January 2021 Merck assumed all activities and expenses associated with the continued development of the influenza A/B compounds discovered under this collaboration. As anticipated, Cocrystal reported no revenues for the third quarter of 2021 compared with $489,000 in revenues for the third quarter of 2020. Under the terms of the Merck collaboration, Cocrystal is eligible to receive up to $156 million in payments related to designated developments, regulatory and sales milestones, as well as royalties on product sales.

R&D expenses for the third quarter of 2021 were $2.2 million compared with $2.1 million for the third quarter of 2020, with the increase primarily related to COVID-19 and influenza programs advancement. The Company expects R&D expenses to increase in the fourth quarter of 2021 due to the advancement of our influenza A program into clinical trials and progress with our pre-clinical COVID-19 program toward clinical development. General and administrative (G&A) expenses for the third quarter of 2021 were $1.8 million compared with $1.1 million for the prior-year quarter, with the increase primarily due to litigation settlements.

The net loss for the third quarter of 2021 was $3.9 million, or $0.04 per share, compared with a net loss for the third quarter of 2020 of $2.7 million, or $0.05 per share.

Year to Date Financial Results

The Company reported no revenues for the first nine months of 2021 versus $1.5 million for the first nine months of 2020. The 2020 revenues were from reimbursed R&D services performed under the influenza A/B program with Merck.

R&D expenses for the first nine months of 2021 increased 22% to $6.5 million and G&A expenses decreased 6% to $4.0 million, both compared with the first nine months of 2020. The higher R&D expenses in 2021 were primarily due to COVID-19 and influenza programs advancement.

The net loss for the nine months ended September 30, 2021 was $10.5 million, or $0.12 per share, compared with a net loss for the nine months ended September 30, 2020 of $8.2 million, or $0.16 per share.

The Company reported unrestricted cash of $61.6 million as of September 30, 2021 compared with $33.0 million as of December 31, 2020. The Company reported working capital of $61.2 million as of September 30, 2021.

About Cocrystal Pharma, Inc.
Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Cautionary Note Regarding Forward-Looking Statements
press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our goals of initiating Phase 1 studies in 2022, selection of a lead candidate for our second SARS-CoV-2 program by year end, our attempts to discover replication inhibitors, our initiation of the Australian clinical trial, our development of antiviral treatments for norovirus, our expectations concerning R&D expenses, our plans to provide a program update on the Merck influenza A/B research, and our liquidity. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the risks arising from supply chain disruptions on our ability to obtain products including raw materials and test animals as well as similar problems with our vendors and our current CRO and future CROs and CMOs, the impact of the COVID-19 pandemic on the national and global economy, the ability of our CROs to recruit volunteers for, and to proceed with, clinical trials, possible delays resulting from the lockdown in Australia, the cooperation of the FDA in accelerating development in our COVID-19 program, our reliance on Merck for further development in the influenza A/B program under the license and collaboration agreement, our collaboration partners’ technology and software performing as expected, the results of future preclinical and clinical trials, general risks arising from clinical trials, receipt of regulatory approvals, regulatory changes, and development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2020. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
jcain@lhai.com

Financial Tables to follow

 COCRYSTAL PHARMA, INC.

CONSOLIDATED BALANCE SHEETS
(in thousands)

		September 30, 2021				December 31, 2020
		(unaudited)
Assets
Current assets:
Cash		$	61,644			$	33,010
Restricted cash			50				50
Accounts receivable			–				556
Prepaid expenses and other current assets			747				399
Total current assets			62,441				34,015
Property and equipment, net			493				591
Deposits			46				46
Operating lease right-of-use assets, net (including $167 and $37, respectively, to related party)			526				498
Goodwill			19,092				19,092
Total assets		$	82,598			$	54,242
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable and accrued expenses		$	961			$	1,080
Current maturities of finance lease liabilities			29				39
Current maturities of operating lease liabilities (including $167 and $39, respectively, to related party)			204				178
Derivative liabilities			34				61
Total current liabilities			1,228				1,358
Long-term liabilities:
Finance lease liabilities			14				34
Operating lease liabilities			344				345
Total long-term liabilities			358				379
Total liabilities			1,586				1,737
Commitments and contingencies (note 9)
Stockholders’ equity:
Common stock, $0.001 par value; 150,000 shares authorized; 97,469 and 70,439 shares issued and outstanding as of September 30, 2021 and December 31, 2020, respectively			98				71
Additional paid-in capital			336,322				297,342
Accumulated deficit			(255,408	)			(244,908	)
Total stockholders’ equity			81,012				52,505
Total liabilities and stockholders’ equity		$	82,598			$	54,242

COCRYSTAL PHARMA, INC.

CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share data)

		Three months ended September 30,								Nine months ended September 30,
		2021				2020				2021				2020
Revenues:
Collaboration revenue		$	–			$	489			$	–			$	1,504
			–				489				–				1,504
Operating expenses:
Research and development			2,165				2,077				6,489				5,336
General and administrative			1,788				1,121				4,030				4,284
Total operating expenses			3,953				3,198				10,519				9,624
Loss from operations			(3,953	)			(2,709	)			(10,519	)			(8,120	)
Other income (expense):
Interest expense, net			(1	)			(2	)			(4	)			(6	)
Foreign exchange loss			(4	)			–				(4	)			–
Change in fair value of derivative liabilities			17				41				27				(29	)
Total other income (expense), net			12				39				19				(35	)
Net loss		$	(3,941	)		$	(2,670	)		$	(10,500	)		$	(8,155	)
Net loss per common share, basic and diluted		$	(0.04	)		$	(0.05	)			(0.12	)			(0.16	)
Weighted average number of common shares outstanding, basic and diluted			97,469				57,555				85,301				50,491

Source: Cocrystal Pharma, Inc.

Ayala Pharmaceuticals Reports Third Quarter 2021 Financial Results and Provides Business Update

 

– Presented Preliminary Clinical Data from 6mg Cohort of Phase 2 ACCURACY Trial of AL101 in R/M ACC Demonstrating 70% Disease Control Rate at ESMO 2021 –

– Presented Pre-Clinical Proof of Concept Data for Enhanced Activity of AL101 in Combination with Approved Cancer Therapies in ACC-

– Published Case Studies Highlighting Clinical Activity of AL101 with Long-Lasting Responses in Patients with Desmoid Tumors –

– Enrollment in All Ongoing Studies is on Track and Have Progressed as Planned –

– Multiple Milestones Across Clinical-Stage Pipeline Expected in 2022 –

REHOVOT, Israel & WILMINGTON, Del., Nov. 15, 2021 (GLOBE NEWSWIRE) — Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, today reported financial results for the period ended September 30, 2021 and highlighted recent progress and upcoming milestones for its pipeline programs.

“As we gear up for multiple important milestones in 2022 across all of our clinical-stage programs in various indications, including desmoid tumors, triple negative breast cancer, adenoid cystic carcinoma and potentially multiple myeloma, we remain steadfast in our approach to developing gamma secretase inhibitors to treat these genetically defined cancers,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “We are incredibly pleased with the safety and efficacy profile of AL101 for the treatment of recurrent/metastatic adenoid cystic carcinoma harboring Notch-activating mutations, as presented at ESMO in September, as well as the strong preclinical rationale for potential combination treatment in this indication and other tumor types. We also continued to progress our pivotal RINGSIDE trial evaluating AL102 for the treatment of desmoid tumors as enrollment continues across multiple sites globally and look forward to reporting preliminary results from this trial in mid-2022. In addition, we are very pleased with our ongoing collaboration with Novartis and the status of the study of our AL102 in combination with their anti BCMA agent for multiple myeloma.”

Recent Business Highlights and Upcoming Milestones:

• Published Two Case Studies Highlighting Clinical Activity of AL101 in Desmoid Tumors in Current Oncology: In September 2021, Ayala published two case studies of adult patients with desmoid tumors treated with AL101. Both patients experienced significant tumor burden and symptomatic and life-threatening disease due to disease bulk and location. With AL101 treatment, both subjects achieved long-lasting partial responses with a maximal decrease in tumor size from baseline of 41% after approximately 1 year (55 weeks) of treatment in Case One, and a maximal decrease in tumor size from baseline of 60% after about 1.6 years (82 weeks) of treatment in Case Two.
  
  
• On Track to Report Initial Interim Data from Part A of the Pivotal Phase 2/3 RINGSIDE Trial for the Treatment of Desmoid Tumors in Mid-2022: Enrollment continues to progress globally in the Phase 2/3 RINGSIDE Trial of AL102. Ayala expects to report an initial interim data read-out from part A of the trial in mid-2022, with part B of the study commencing thereafter.
  
  
• Phase 1 Trial of AL102 in Combination with Novartis’ BCMA Targeting Agent, WVT087 for the Treatment of Relapsed/Refractory Multiple Myeloma Continues to Progress: Enrollment progresses as planned in the Phase 1 combination trial of AL102 with Novartis’ investigational anti-B-cell maturation antigen (BCMA) agent, WVT078, for the treatment of relapsed and/or refractory (R/R) multiple myeloma (MM).
  
  
• Presented Preliminary Clinical Data from the Ongoing Phase 2 ACCURACY Trial and ACC at European Society for Medical Oncology (ESMO) Virtual Congress 2021: In September 2021, Ayala presented updated interim data from the 6mg cohort of its ongoing Phase 2 ACCURACY study of AL101 for the treatment of recurrent/metastatic adenoid cystic carcinoma (R/M ACC) harboring Notch activating mutations. The data demonstrated meaningful clinical activity of AL101 6mg monotherapy with a 70% disease control rate across 33 evaluable patients. Partial responses were observed in three subjects (9%) and stable disease was observed in 20 subjects (61%). The 6mg dose of AL101 was well tolerated with manageable side effects consistent with those observed in the 4mg cohort.
  
  
• Presented Preclinical Proof of Concept Data of AL101 in Combination with Approved Cancer Therapies in ACC at ESMO: In September 2021, Ayala also presented a preclinical study evaluating the potential of combination therapy of AL101 in PDX models of ACC, comparing the differential gene expression of ACC tumors versus normal matched tissue regardless of Notch activation status. AL101 in combination demonstrated significant tumor growth inhibition, including regressions, compared to each drug alone, and the study indicated that crosstalk between signaling pathways may increase the efficacy of AL101 in R/M ACC regardless of Notch mutational status.
  
  
• Phase 2 TENACITY Trial of AL101 for the Treatment of Triple Negative Breast Cancer Continues to Progress: Ayala continues to enroll patients in the Phase 2 TENACITY clinical trial of AL101, for the treatment of patients with Notch-activated recurrent or metastatic (R/M) triple negative breast cancer (TNBC). The Company expects to report preliminary data from this ongoing trial in 2022.
  
  

Third Quarter 2021 Financial Results

• Cash Position: Cash and cash equivalents were $40.8 million as of September 30, 2021, as compared to $42.0 million as of December 31, 2020.
• Collaboration Revenue: Collaboration revenue was $0.6 million for the third quarter of 2021, as compared to $0.7 million for the same period in 2020.
• R&D Expenses: Research and development expenses were $7.4 million for the third quarter of 2021, compared to $5.4 million for the same period in 2020. The increase was primarily driven by the advancement in our clinical trials.
• G&A Expenses: General and administrative expenses were $2.2 million for the third quarter of 2021, compared to $1.9 million for the same period in 2020.
• Net Loss: Net loss was $9.8 million for the third quarter of 2021, resulting in a basic and diluted net loss per share of $0.68. Net loss was $7.4 million for the same period in 2020, resulting in a basic and diluted net loss per share of $0.59.
  
  

About Ayala Pharmaceuticals

Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM) (in collaboration with Novartis). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations and in a Phase 2 clinical trial for patients with TNBC (TENACITY) bearing Notch activating mutations and other gene rearrangements. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE) and is being evaluated in a Phase 1 clinical trial in combination with Novartis’ BMCA targeting agent, WVT078, in Patients with relapsed/refractory Multiple Myeloma. For more information, visit www.ayalapharma.com.

Investors:
Julie Seidel
Stern Investor Relations, Inc.
+1-212-362-1200
Julie.seidel@sternir.com

Ayala Pharmaceuticals:
+1-857-444-0553
info@ayalapharma.com

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to our development of AL101 and AL102, the promise and potential impact of our preclinical or clinical trial data, the timing of and plans to initiate additional clinical trials of AL101 and AL102, upcoming milestones, including without limitation the timing and results of any clinical trials or readouts and patient enrollment. These forward-looking statements are based on management’s current expectations. The words “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our operations, including our preclinical studies and clinical trials, and the continuity of our business; we have incurred significant losses, are not currently profitable and may never become profitable; our need for additional funding; our cash runway; our limited operating history and the prospects for our future viability; the lengthy, expensive, and uncertain process of clinical drug development, including potential delays in regulatory approval; our requirement to pay significant payments under product candidate licenses; the approach we are taking to discover and develop product candidates and whether it will lead to marketable products; the expense, time-consuming nature and uncertainty of clinical trials; enrollment and retention of patients; potential side effects of our product candidates; our ability to develop or to collaborate with others to develop appropriate diagnostic tests; protection of our proprietary technology and the confidentiality of our trade secrets; potential lawsuits for, or claims of, infringement of third-party intellectual property or challenges to the ownership of our intellectual property; risks associated with international operations; our ability to retain key personnel and to manage our growth; the potential volatility of our common stock; costs and resources of operating as a public company; unfavorable or no analyst research or reports; and securities class action litigation against us. These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission (SEC) on March 24, 2021 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

AYALA PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share amounts)

AYALA PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
(In thousands, except share & per share amounts)

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Cocrystal Pharma Reports Third Quarter Financial Results and Provides an Update on Development Programs and Milestones Cocrystal Pharma announced financial results for the three and nine months ended September 30, 2021, and provides updates on its antiviral pipeline, upcoming milestones and business activities Research, News & Market Data on Cocrystal Pharma Watch recent presentation from Cocrystal Pharma

 

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	Image Credit: Raleigh McElvery and Sebastian Swanson (MIT)

For Stem Cells, Bigger Doesn’t Mean Better

 

MIT Biologists Show that Enlargement of Blood Stem Cells Restricts their Ability to Generate New Blood Cells During Aging

Anne Trafton | MIT News Office

MIT biologists have answered an important biological question: Why do cells control their size?

Cells of the same type are strikingly uniform in size, while cell size differs between different cell types. This raises the question of whether cell size is important for cellular physiology.

The new study suggests that cellular enlargement drives a decline in the function of stem cells. The researchers found that blood stem cells, which are among the smallest cells in the body, lose their ability to perform their normal function — replenishing the body’s blood cells — as they grow larger. However, when the cells were restored to their usual size, they behaved normally again.

The researchers also found that blood stem cells tend to enlarge as they age. Their study shows that this enlargement contributes to stem cell decline during aging.

“We have discovered cellular enlargement as a new aging factor in vivo, and now we can explore if we can treat cellular enlargement to delay aging and aging-related diseases,” says Jette Lengefeld, a former MIT postdoc, who is now a principal investigator at the University of Helsinki.

Lengefeld is the lead author of the study, which appears today in Science Advances. The late Angelika Amon, an MIT professor of biology and member of the Koch Institute for Integrative Cancer Research, is the senior author of the study.

Outsized Effects

It has been known since the 1960s that human cells grown in a lab dish enlarge as they become senescent — a nondividing cellular state that is associated with aging. Every time a cell divides, it can encounter DNA damage. When this happens, division is halted to repair the damage. During each of these delays, the cell grows slightly larger. Many scientists believed that this enlargement was simply a side effect of aging, but the Amon lab began to investigate the possibility that large cell size drives age-related losses of function.

Lengefeld studied the effects of size on stem cells — specifically, blood stem cells, which give rise to the blood cells of our body throughout life. To study how size affects these stem cells, the researchers damaged their DNA, leading to an increase in their size. They then compared these enlarged cells to other cells that also experienced DNA damage but were prevented from increasing in size using a drug called rapamycin.

After the treatment, the researchers measured the functionality of these two groups of stem cells by injecting them into mice that had their own blood stem cells eliminated. This allowed the researchers to determine whether the transplanted stem cells were able to repopulate the mouse’s blood cells.

They found that the DNA-damaged and enlarged stem cells were unable to produce new blood cells. However, the DNA-damaged stem cells that were kept small were still able to produce new blood cells.

In another experiment, the researchers used a genetic mutation to reduce the size of naturally occurring large stem cells that they found in older mice. They showed that if they induced those large stem cells to become small again, the cells regained their regenerative potential and behaved like younger stem cells.

“This is striking evidence supporting the model that size is important for the functionality of stem cells,” Lengefeld says. “When we damage the stem cells’ DNA but keep them small during the damage, they retain their functionality. And if we reduce the size of large stem cells, we can restore their function.”

Until now, studying the function of cell size has been difficult to do, says Jan Skotheim, a professor of biology at Stanford University.

“We know that cells, through various mechanisms, operate to keep their size within a specific range, but we haven’t really understood why,” says Skotheim, who was not involved in the research. “This is really the first work showing the function of cell size in any animal.”

Keeping Cells Small

When the researchers treated mice with rapamycin, beginning at a young age, they were able to prevent blood stem cells from enlarging as the mice got older. Blood stem cells from those mice remained small and were able to build blood cells like young stem cells even in mice 3 years of age — an old age for a mouse.

Rapamycin, a drug that can inhibit cell growth, is now used to treat some cancers and to prevent organ transplant rejection, and has raised interest for its ability to extend lifespan in mice and other organisms. It may be useful in slowing down the enlargement of stem cells and therefore could have beneficial effects in humans, Lengefeld says.

“If we find drugs that are specific in making large blood stem cells smaller again, we can test whether this improves the health of people who suffer from problems with their blood system — like anemia and a reduced immune system — or maybe even help people with leukemia,” she says.

The researchers also demonstrated the importance of size in another type of stem cells — intestinal stem cells. They found that larger stem cells were less able to generate intestinal organoids, which mimic the structure of the intestinal lining.

“That suggested that this relationship between cell size and function is conserved in stem cells, and that cellular size is a marker of stem cell function,” Lengefeld says.

The research was funded, in part, by the Howard Hughes Medical Institute, the Jane Coffin Childs Memorial Fund, the Swiss National Science Foundation, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the Koch Institute Support (core) grant from the National Cancer Institute, and the MIT Stem Cell Initiative.

 

Suggested Reading:

Cells that Can be Produced from Stem Cells

Preventing the Immune System from Rejecting Gene Therapy

Pros and Cons of FDA Being Funded in Part by Drug Companies

Therapeutic Research Advanced by Stem Cell Science

 

The Investor Forum at the World Stem Cell Summit Corporate presentations and Q&As with executives representing breakthroughs in medical science. Increasing longevity. Avoiding and curing diseases. Investment Ideas. View Presentations

 

 

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Ayala Pharmaceuticals Reports Third Quarter 2021 Financial Results and Provides Business Update

 

– Presented Preliminary Clinical Data from 6mg Cohort of Phase 2 ACCURACY Trial of AL101 in R/M ACC Demonstrating 70% Disease Control Rate at ESMO 2021 –

– Presented Pre-Clinical Proof of Concept Data for Enhanced Activity of AL101 in Combination with Approved Cancer Therapies in ACC-

– Published Case Studies Highlighting Clinical Activity of AL101 with Long-Lasting Responses in Patients with Desmoid Tumors –

– Enrollment in All Ongoing Studies is on Track and Have Progressed as Planned –

– Multiple Milestones Across Clinical-Stage Pipeline Expected in 2022 –

REHOVOT, Israel & WILMINGTON, Del., Nov. 15, 2021 (GLOBE NEWSWIRE) — Ayala Pharmaceuticals, Inc. (Nasdaq: AYLA), a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations, today reported financial results for the period ended September 30, 2021 and highlighted recent progress and upcoming milestones for its pipeline programs.

“As we gear up for multiple important milestones in 2022 across all of our clinical-stage programs in various indications, including desmoid tumors, triple negative breast cancer, adenoid cystic carcinoma and potentially multiple myeloma, we remain steadfast in our approach to developing gamma secretase inhibitors to treat these genetically defined cancers,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “We are incredibly pleased with the safety and efficacy profile of AL101 for the treatment of recurrent/metastatic adenoid cystic carcinoma harboring Notch-activating mutations, as presented at ESMO in September, as well as the strong preclinical rationale for potential combination treatment in this indication and other tumor types. We also continued to progress our pivotal RINGSIDE trial evaluating AL102 for the treatment of desmoid tumors as enrollment continues across multiple sites globally and look forward to reporting preliminary results from this trial in mid-2022. In addition, we are very pleased with our ongoing collaboration with Novartis and the status of the study of our AL102 in combination with their anti BCMA agent for multiple myeloma.”

Recent Business Highlights and Upcoming Milestones:

• Published Two Case Studies Highlighting Clinical Activity of AL101 in Desmoid Tumors in Current Oncology: In September 2021, Ayala published two case studies of adult patients with desmoid tumors treated with AL101. Both patients experienced significant tumor burden and symptomatic and life-threatening disease due to disease bulk and location. With AL101 treatment, both subjects achieved long-lasting partial responses with a maximal decrease in tumor size from baseline of 41% after approximately 1 year (55 weeks) of treatment in Case One, and a maximal decrease in tumor size from baseline of 60% after about 1.6 years (82 weeks) of treatment in Case Two.
  
  
• On Track to Report Initial Interim Data from Part A of the Pivotal Phase 2/3 RINGSIDE Trial for the Treatment of Desmoid Tumors in Mid-2022: Enrollment continues to progress globally in the Phase 2/3 RINGSIDE Trial of AL102. Ayala expects to report an initial interim data read-out from part A of the trial in mid-2022, with part B of the study commencing thereafter.
  
  
• Phase 1 Trial of AL102 in Combination with Novartis’ BCMA Targeting Agent, WVT087 for the Treatment of Relapsed/Refractory Multiple Myeloma Continues to Progress: Enrollment progresses as planned in the Phase 1 combination trial of AL102 with Novartis’ investigational anti-B-cell maturation antigen (BCMA) agent, WVT078, for the treatment of relapsed and/or refractory (R/R) multiple myeloma (MM).
  
  
• Presented Preliminary Clinical Data from the Ongoing Phase 2 ACCURACY Trial and ACC at European Society for Medical Oncology (ESMO) Virtual Congress 2021: In September 2021, Ayala presented updated interim data from the 6mg cohort of its ongoing Phase 2 ACCURACY study of AL101 for the treatment of recurrent/metastatic adenoid cystic carcinoma (R/M ACC) harboring Notch activating mutations. The data demonstrated meaningful clinical activity of AL101 6mg monotherapy with a 70% disease control rate across 33 evaluable patients. Partial responses were observed in three subjects (9%) and stable disease was observed in 20 subjects (61%). The 6mg dose of AL101 was well tolerated with manageable side effects consistent with those observed in the 4mg cohort.
  
  
• Presented Preclinical Proof of Concept Data of AL101 in Combination with Approved Cancer Therapies in ACC at ESMO: In September 2021, Ayala also presented a preclinical study evaluating the potential of combination therapy of AL101 in PDX models of ACC, comparing the differential gene expression of ACC tumors versus normal matched tissue regardless of Notch activation status. AL101 in combination demonstrated significant tumor growth inhibition, including regressions, compared to each drug alone, and the study indicated that crosstalk between signaling pathways may increase the efficacy of AL101 in R/M ACC regardless of Notch mutational status.
  
  
• Phase 2 TENACITY Trial of AL101 for the Treatment of Triple Negative Breast Cancer Continues to Progress: Ayala continues to enroll patients in the Phase 2 TENACITY clinical trial of AL101, for the treatment of patients with Notch-activated recurrent or metastatic (R/M) triple negative breast cancer (TNBC). The Company expects to report preliminary data from this ongoing trial in 2022.
  
  

Third Quarter 2021 Financial Results

• Cash Position: Cash and cash equivalents were $40.8 million as of September 30, 2021, as compared to $42.0 million as of December 31, 2020.
• Collaboration Revenue: Collaboration revenue was $0.6 million for the third quarter of 2021, as compared to $0.7 million for the same period in 2020.
• R&D Expenses: Research and development expenses were $7.4 million for the third quarter of 2021, compared to $5.4 million for the same period in 2020. The increase was primarily driven by the advancement in our clinical trials.
• G&A Expenses: General and administrative expenses were $2.2 million for the third quarter of 2021, compared to $1.9 million for the same period in 2020.
• Net Loss: Net loss was $9.8 million for the third quarter of 2021, resulting in a basic and diluted net loss per share of $0.68. Net loss was $7.4 million for the same period in 2020, resulting in a basic and diluted net loss per share of $0.59.
  
  

About Ayala Pharmaceuticals

Ayala Pharmaceuticals, Inc. is a clinical-stage oncology company focused on developing and commercializing small molecule therapeutics for patients suffering from rare and aggressive cancers, primarily in genetically defined patient populations. Ayala’s approach is focused on predicating, identifying and addressing tumorigenic drivers of cancer through a combination of its bioinformatics platform and next-generation sequencing to deliver targeted therapies to underserved patient populations. The company has two product candidates under development, AL101 and AL102, targeting the aberrant activation of the Notch pathway with gamma secretase inhibitors to treat a variety of tumors including Adenoid Cystic Carcinoma, Triple Negative Breast Cancer (TNBC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM) (in collaboration with Novartis). AL101, has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and is currently in a Phase 2 clinical trial for patients with ACC (ACCURACY) bearing Notch activating mutations and in a Phase 2 clinical trial for patients with TNBC (TENACITY) bearing Notch activating mutations and other gene rearrangements. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE) and is being evaluated in a Phase 1 clinical trial in combination with Novartis’ BMCA targeting agent, WVT078, in Patients with relapsed/refractory Multiple Myeloma. For more information, visit www.ayalapharma.com.

Investors:
Julie Seidel
Stern Investor Relations, Inc.
+1-212-362-1200
Julie.seidel@sternir.com

Ayala Pharmaceuticals:
+1-857-444-0553
info@ayalapharma.com

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements relating to our development of AL101 and AL102, the promise and potential impact of our preclinical or clinical trial data, the timing of and plans to initiate additional clinical trials of AL101 and AL102, upcoming milestones, including without limitation the timing and results of any clinical trials or readouts and patient enrollment. These forward-looking statements are based on management’s current expectations. The words “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the impact of the COVID-19 pandemic on our operations, including our preclinical studies and clinical trials, and the continuity of our business; we have incurred significant losses, are not currently profitable and may never become profitable; our need for additional funding; our cash runway; our limited operating history and the prospects for our future viability; the lengthy, expensive, and uncertain process of clinical drug development, including potential delays in regulatory approval; our requirement to pay significant payments under product candidate licenses; the approach we are taking to discover and develop product candidates and whether it will lead to marketable products; the expense, time-consuming nature and uncertainty of clinical trials; enrollment and retention of patients; potential side effects of our product candidates; our ability to develop or to collaborate with others to develop appropriate diagnostic tests; protection of our proprietary technology and the confidentiality of our trade secrets; potential lawsuits for, or claims of, infringement of third-party intellectual property or challenges to the ownership of our intellectual property; risks associated with international operations; our ability to retain key personnel and to manage our growth; the potential volatility of our common stock; costs and resources of operating as a public company; unfavorable or no analyst research or reports; and securities class action litigation against us. These and other important factors discussed under the caption “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2020 filed with the U.S. Securities and Exchange Commission (SEC) on March 24, 2021 and our other filings with the SEC could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. New risk factors and uncertainties may emerge from time to time, and it is not possible to predict all risk factors and uncertainties. While we may elect to update such forward-looking statements at some point in the future, except as required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Although we believe the expectations reflected in such forward-looking statements are reasonable, we can give no assurance that such expectations will prove to be correct. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

AYALA PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In thousands, except share and per share amounts)

AYALA PHARMACEUTICALS, INC.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS
(Unaudited)
(In thousands, except share & per share amounts)

Cocrystal Pharma Reports Third Quarter Financial Results and Provides an Update on Development Programs and Milestones

 

• Submitted pre-IND briefing package to the FDA for intranasal/pulmonary CDI-45205 for the treatment of COVID-19 and sets goal of initiating IND-enabling study and Phase 1 in 2022
• Advance oral COVID-19 program with goal of initiating IND-enabling study and Phase 1 in 2022
• Received regulatory clearance to initiate a Phase 1 trial in Australia with orally administered CC-42344 for the treatment of pandemic and seasonal influenza A
  

BOTHELL, Wash., Nov. 15, 2021 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”), a clinical-stage biotechnology company, reports financial results for the three and nine months ended September 30, 2021, and provides updates on its antiviral pipeline, upcoming milestones and business activities.

“Today we are reporting progress with our promising COVID-19 antiviral development programs along with setting the goal of beginning first-in-human clinical studies with our novel SARS-CoV-2 protease inhibitors in 2022,” said Sam Lee, Ph.D., co-interim CEO and President of Cocrystal. “We expect comments from the U.S. Food and Drug Administration (FDA) by mid-December on our pre-IND briefing package for our intranasal/pulmonary-delivered SARS-CoV-2 protease inhibitor CDI-45205, our most advanced SARS-CoV-2 program. The FDA’s comments will be useful in designing the Phase 1 and Phase 2 clinical trial protocols for this program.

“Under our second SARS-CoV-2 program, this one targeting oral administration, by year-end 2021 we expect to select a lead candidate from novel SARS-CoV-2 protease inhibitors discovered using our proprietary structure-based technology,” he added. “We will then prepare a pre-IND briefing package to submit to the FDA with the goal of initiating a clinical trial in 2022. Work also continues with a third COVID-19 program using our platform to discover oral replication inhibitors for the treatment of COVID-19.

“Regarding our influenza programs, our supplier is manufacturing our novel PB2 inhibitor CC-42344 for pandemic and seasonal influenza A. We expect patient enrollment in our Phase 1 trial to begin in early 2022 with data readouts later that same year,” said Dr. Lee. “With the influenza A/B program, we anticipate providing an update on the development of the compounds jointly discovered with Merck using our technology platform in the first quarter 2022.”

“With the proceeds from financings completed earlier this year and a clean balance sheet, we believe our capital is sufficient to fund planned operations. This outlook includes our goal of having multiple high-value antiviral compounds in clinical development next year as we advance our programs toward commercialization,” said James Martin, co-interim CEO and CFO. “We are aggressively pursuing the advancement of these potent compounds as we navigate a challenging global supply chain. Thankfully our strong partnership with CROs has benefited us to that extent.”

Antiviral Pipeline Overview

COVID-19 and Other Coronavirus Programs

• Intranasal/Pulmonary Protease Inhibitor
  • Our lead therapeutic molecule CDI-45205 was among the broad-spectrum viral protease inhibitors obtained from Kansas State University Research Foundation (KSURF) under an exclusive license agreement announced in 2020. We believe the protease inhibitors obtained from KSURF have the ability to inhibit the inactive SARS-CoV-2 polymerase replication enzymes into an active form.
  • CDI-45205 and several analogs showed potent in vitro activity against the SARS-CoV-2 Delta (India/B.1.617.2), Gamma (Brazil/P.1), Alpha (United Kingdom/B.1.1.7) and Beta (South African/B.1.351) variants, surpassing the activity observed with the original or wild-type Wuhan strain.
  • CDI-45205 demonstrated good bioavailability in mouse and rat pharmacokinetic studies via intraperitoneal injection, and no cytotoxicity against a variety of human cell lines. CDI-45205 also demonstrated a strong synergistic effect with the FDA-approved COVID-19 medicine remdesivir.
  • A proof-of-concept animal study demonstrated that daily injection of CDI-45205 exhibited favorable in vivo efficacy in mice infected with MERS-CoV-2.

• Oral Protease Inhibitors
  • Using our drug discovery platform, we discovered and are developing novel SARS-CoV-2 3CL protease inhibitors and anticipate identifying a preclinical 3CL lead for oral administration by the end of 2021.
  • We plan to submit a pre-IND briefing package to the FDA in the first half of 2022 to support an IND application, with the goal of progressing to IND-enabling study and Phase 1 in late 2022.
  • The main SARS-CoV-2 protease inhibitors showed potent in vitro pan-viral activity against human common coronaviruses, rhinoviruses, and respiratory enteroviruses that frequently cause the common cold, as well as against noroviruses that can cause symptoms of acute gastroenteritis.

• Replication Inhibitors
  • We are using our platform to seek to discover replication inhibitors for developing orally administered therapeutic and prophylactic treatments for SARS-CoV-2. Replication inhibitors have the potential to work with protease inhibitors in a combination therapy regimen.

Influenza Programs

• Pandemic and Seasonal Influenza A
  • Our novel PB2 inhibitor CC-42344 has shown excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as strains resistant to Tamiflu and Xofluza. CC-42344 also has favorable pharmacokinetic and drug-resistance profiles.
  • We have completed preclinical IND-enabling studies with CC-42344 and have received clearance from the Australian Human Research Ethics Committees (HREC) to initiate a Phase 1 clinical trial. Our supplier is manufacturing drug and we expect to begin patient enrollment in the first quarter of 2022.

• Pandemic and Seasonal Influenza A/B program
  
  • In January 2019 we entered into an Exclusive License and Research Collaboration Agreement with Merck Sharp & Dohme Corp. to discover and developed certain proprietary influenza A/B antiviral agents that are effective against both influenza A and B strains. This agreement includes milestone payments of up to $156 million plus royalties on sales of products discovered under the agreement.
  • In January 2021 we announced completion of all research obligations under the agreement. Merck is now solely responsible for further preclinical and clinical development of the influenza A/B antiviral compounds discovered under this agreement. We anticipate providing a program update during the first quarter of 2022.

• The World Health Organization (WHO) estimates there are approximately 1 billion cases of influenza annually worldwide, resulting in 3 million to 5 million cases of severe illness and 290,000 to 650,000 deaths. The Centers for Disease Control and Prevention (CDC) estimates that since 2010 influenza has caused 9 million to 45 million illnesses in the U.S. annually, resulting in 140,000 to 810,000 hospitalizations and 12,000 to 61,000 deaths each year.
  

Norovirus Program

• We are developing certain proprietary broad-spectrum antiviral compounds to treat norovirus infections.
• Norovirus is a global public health problem responsible for nearly 90% of epidemic, non-bacterial outbreaks of gastroenteritis around the world.

Hepatitis C Program

• We are seeking a partner to advance the development of CC-31244 following completion of a Phase 2a trial. This compound has shown favorable safety and preliminary efficacy in a triple regimen Phase 2a study in combination with Epclusa (sofosbuvir/velpatasvir) for the ultra-short duration treatment of individuals infected with the hepatitis C virus (HCV).
• HCV is a viral infection of the liver that causes both acute and chronic infection. According to the WHO, in 2017 an estimated 71 million people worldwide had chronic HCV infection, including 3.5 million in the U.S. Approximately 399,000 people die each year from HCV infection, mostly from cirrhosis and hepatocellular carcinoma.

Third Quarter Financial Results

Throughout 2020 Cocrystal reported quarterly revenues under an influenza A/B collaboration with Merck consisting of research and development (R&D) services performed by Cocrystal and reimbursed by Merck.

As discussed above, in January 2021 Merck assumed all activities and expenses associated with the continued development of the influenza A/B compounds discovered under this collaboration. As anticipated, Cocrystal reported no revenues for the third quarter of 2021 compared with $489,000 in revenues for the third quarter of 2020. Under the terms of the Merck collaboration, Cocrystal is eligible to receive up to $156 million in payments related to designated developments, regulatory and sales milestones, as well as royalties on product sales.

R&D expenses for the third quarter of 2021 were $2.2 million compared with $2.1 million for the third quarter of 2020, with the increase primarily related to COVID-19 and influenza programs advancement. The Company expects R&D expenses to increase in the fourth quarter of 2021 due to the advancement of our influenza A program into clinical trials and progress with our pre-clinical COVID-19 program toward clinical development. General and administrative (G&A) expenses for the third quarter of 2021 were $1.8 million compared with $1.1 million for the prior-year quarter, with the increase primarily due to litigation settlements.

The net loss for the third quarter of 2021 was $3.9 million, or $0.04 per share, compared with a net loss for the third quarter of 2020 of $2.7 million, or $0.05 per share.

Year to Date Financial Results

The Company reported no revenues for the first nine months of 2021 versus $1.5 million for the first nine months of 2020. The 2020 revenues were from reimbursed R&D services performed under the influenza A/B program with Merck.

R&D expenses for the first nine months of 2021 increased 22% to $6.5 million and G&A expenses decreased 6% to $4.0 million, both compared with the first nine months of 2020. The higher R&D expenses in 2021 were primarily due to COVID-19 and influenza programs advancement.

The net loss for the nine months ended September 30, 2021 was $10.5 million, or $0.12 per share, compared with a net loss for the nine months ended September 30, 2020 of $8.2 million, or $0.16 per share.

The Company reported unrestricted cash of $61.6 million as of September 30, 2021 compared with $33.0 million as of December 31, 2020. The Company reported working capital of $61.2 million as of September 30, 2021.

About Cocrystal Pharma, Inc.
Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), hepatitis C viruses and noroviruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Cautionary Note Regarding Forward-Looking Statements
press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our goals of initiating Phase 1 studies in 2022, selection of a lead candidate for our second SARS-CoV-2 program by year end, our attempts to discover replication inhibitors, our initiation of the Australian clinical trial, our development of antiviral treatments for norovirus, our expectations concerning R&D expenses, our plans to provide a program update on the Merck influenza A/B research, and our liquidity. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the risks arising from supply chain disruptions on our ability to obtain products including raw materials and test animals as well as similar problems with our vendors and our current CRO and future CROs and CMOs, the impact of the COVID-19 pandemic on the national and global economy, the ability of our CROs to recruit volunteers for, and to proceed with, clinical trials, possible delays resulting from the lockdown in Australia, the cooperation of the FDA in accelerating development in our COVID-19 program, our reliance on Merck for further development in the influenza A/B program under the license and collaboration agreement, our collaboration partners’ technology and software performing as expected, the results of future preclinical and clinical trials, general risks arising from clinical trials, receipt of regulatory approvals, regulatory changes, and development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2020. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
jcain@lhai.com

Financial Tables to follow

 COCRYSTAL PHARMA, INC.

CONSOLIDATED BALANCE SHEETS
(in thousands)

		September 30, 2021				December 31, 2020
		(unaudited)
Assets
Current assets:
Cash		$	61,644			$	33,010
Restricted cash			50				50
Accounts receivable			–				556
Prepaid expenses and other current assets			747				399
Total current assets			62,441				34,015
Property and equipment, net			493				591
Deposits			46				46
Operating lease right-of-use assets, net (including $167 and $37, respectively, to related party)			526				498
Goodwill			19,092				19,092
Total assets		$	82,598			$	54,242
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable and accrued expenses		$	961			$	1,080
Current maturities of finance lease liabilities			29				39
Current maturities of operating lease liabilities (including $167 and $39, respectively, to related party)			204				178
Derivative liabilities			34				61
Total current liabilities			1,228				1,358
Long-term liabilities:
Finance lease liabilities			14				34
Operating lease liabilities			344				345
Total long-term liabilities			358				379
Total liabilities			1,586				1,737
Commitments and contingencies (note 9)
Stockholders’ equity:
Common stock, $0.001 par value; 150,000 shares authorized; 97,469 and 70,439 shares issued and outstanding as of September 30, 2021 and December 31, 2020, respectively			98				71
Additional paid-in capital			336,322				297,342
Accumulated deficit			(255,408	)			(244,908	)
Total stockholders’ equity			81,012				52,505
Total liabilities and stockholders’ equity		$	82,598			$	54,242

COCRYSTAL PHARMA, INC.

CONSOLIDATED STATEMENTS OF OPERATIONS
(in thousands, except per share data)

		Three months ended September 30,								Nine months ended September 30,
		2021				2020				2021				2020
Revenues:
Collaboration revenue		$	–			$	489			$	–			$	1,504
			–				489				–				1,504
Operating expenses:
Research and development			2,165				2,077				6,489				5,336
General and administrative			1,788				1,121				4,030				4,284
Total operating expenses			3,953				3,198				10,519				9,624
Loss from operations			(3,953	)			(2,709	)			(10,519	)			(8,120	)
Other income (expense):
Interest expense, net			(1	)			(2	)			(4	)			(6	)
Foreign exchange loss			(4	)			–				(4	)			–
Change in fair value of derivative liabilities			17				41				27				(29	)
Total other income (expense), net			12				39				19				(35	)
Net loss		$	(3,941	)		$	(2,670	)		$	(10,500	)		$	(8,155	)
Net loss per common share, basic and diluted		$	(0.04	)		$	(0.05	)			(0.12	)			(0.16	)
Weighted average number of common shares outstanding, basic and diluted			97,469				57,555				85,301				50,491

Source: Cocrystal Pharma, Inc.

Monday, November 15, 2021

Helius Medical Technologies (HSDT)(HSM:CA)
3Q21 Results; Raises $9.8 million, net

Helius Medical Technologies is a neurotech company focused on neurological wellness. The Company’s purpose is to develop, license and acquire unique and non-invasive platform technologies that amplify the brain’s ability to heal itself. The Company’s first commercial product is the Portable Neuromodulation Stimulator (PoNSTM). For more information, visit www.heliusmedical.com.

Joe Gomes, Senior Research Analyst, Noble Capital Markets, Inc.

Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

3Q21 Operating Results. Revenue of $109,000 and a net loss of $4.7 million, or $2.01 per share. This compares to revenue of $131,000 and a net loss of $3.5 million, or $2.70 per share, in the third quarter last year. (Share count increased to 2.4 million from 1.3 million y-o-y.) We had forecast revenue of $79,000 and a net loss $4.2 million, or $1.75 per share.


Early Days.  Helius remains in the top of the first inning in terms of PoNS commercialization. The better than expected revenue is due to a slow re-opening of Canada from COVID restrictions. The Company has established 36 clinics throughout the country. The main difference in the loss is due to $0.9 million of severance cost related to the former CFO …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

ProMIS Neurosciences Announces Third Quarter 2021 Results

 

TORONTO and CAMBRIDGE, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) — ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF) (“ProMIS or the Company”), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, today announced its operational and financial results for the three and nine months ended September 30, 2021.

“We are pleased to be ramping up our efforts to advance our lead asset, PMN 310, closer toward the clinic,” said Gene Williams, ProMIS’ Chairman and CEO. “The financing we secured earlier this year is enabling us to unlock the potential of our platform, which we believe could have significant impact on the treatment of several neurological diseases, including Alzheimer’s Disease (AD), Parkinson’s Disease and ALS. The strengthening of our management team and Board this quarter has also enabled us to leverage worldwide development and patent expertise and strengthen our overall competitive position.”

Corporate Highlights

• On July 2, 2021, the Company announced the voting results of its annual meeting of shareholders held on June 30, 2021, in Vancouver, British Columbia, Canada. All resolutions described in the Management Proxy Circular and placed before the meeting were approved by the shareholders.
• On July 8, 2021, the Company announced that it had filed and obtained a receipt for the Prospectus with the securities regulators in each of the provinces and territories of Canada, except Quebec.
• On August 25, 2021, we announced the closing of a public offering for gross proceeds of US$20,125,000 (CDN$25,522,525).
• On October 7, 2021, we announced that we would hold a special general meeting of shareholders (the “Special Meeting”) on December 1, 2021. We set October 18, 2021, as the record date for the Special Meeting. The purpose of the Special Meeting is to ask shareholders to grant the Board of Directors the authority, exercisable in the Board’s discretion, to consolidate (or reverse split) the Company’s issued and outstanding common shares in furtherance of a potential listing of the Company’s shares on a stock exchange in the United States.

People

• On September 1, 2021, the Company appointed Josh Mandel-Brehm to the board of directors. Mr. Mandel-Brehm has held various key business development and operations leadership roles at leading biotechnology companies.
• On September 23, 2021, the Company appointed Maggie Shafmaster, JD, PhD, to the board of directors. Dr Shafmaster has approximately 30 years of experience providing intellectual property advice to biotechnology and pharmaceutical industries.

On October 22, 2021, the Company announced the expansion of its senior management team. The following changes were announced:

• Eugene Williams, formerly Executive Chairman, takes on the role of Chairman and Chief Executive Officer (“CEO”), with immediate effect.
• Dr. Elliot Goldstein resigned from his current role as CEO with immediate effect and continues to support us as President and special consultant to the CEO.
• Gavin Malenfant joins our senior management team as Chief Operating Officer. Mr. Malenfant brings more than 30 years of biopharmaceutical experience to our team, with special focus on providing expert management and oversight of drug development programs. The top priority in the near term will be to support the timely development of the PMN310 program to completion of IND enabling activities, anticipated in the second half of 2022. He will be working with Mr. Williams and the leadership of the PMN310 project team, whose key members include:
  • Michael Grundman, MD, Senior Medical Adviser. Prior to joining the pharmaceutical industry, Dr. Grundman was Associate Director of the Alzheimer’s Disease Cooperative Study at the University of California, San Diego (“UCSD”) and is currently an Adjunct Professor of Neurosciences at UCSD. Dr. Grundman previously served on the FDA Peripheral and Central Nervous System Advisory Committee.
  • Ernest Bush, PhD, Head of Pharmacology/Toxicology. Dr. Bush has 35 years of experience working in the field of biomedical research and development, driving development of innovative therapies for treatment of human diseases. He has served as a consultant in non-clinical development providing advice and insight into Investigational New Drug (“IND”) enabling programs, pre-clinical data-set analysis for due diligence and evaluation and audits of Good Laboratory Practices (“GLP”) bioanalytical and toxicology facilities and studies.
  • Dennis Chen, PhD, Head of Manufacturing. Dennis has more than 25 years of prior pharmaceutical experience in working with companies from virtual to global and all phases of development. Dennis provides Regulatory Affairs, Chemistry, Chemistry, Manufacturing and Controls (“CMC”) and Biopharmaceutical Development support to ProMIS with expertise in peptides, proteins and oligonucleotides.

Financial Results

Results of Operations – Three months ended September 30, 2021 and 2020

The following table summarizes our results of operations for the three months ended September 30, 2021 and 2020:

		Three Months Ended
		September 30,
		2021				2020				Change
Revenues		$	5,101			$	–			$	5,101
Operating expenses
Research and development		$	1,192,865			$	1,053,769			$	139,096
General and administrative			852,695				510,264				342,431
Total operating expenses			2,045,560				1,564,033				481,527
Loss from operations			2,040,459				1,564,033				476,426
Other income			(1,078,483	)			–				(1,078,483	)
Net loss		$	961,976			$	1,564,033			$	(602,057	)

Research and Development

Research and development expenses consist of the following:

		Three Months Ended
		September 30,
		2021				2020				Change
Direct research and development expenses by program:		$	590,940			$	390,917			$	200,023
Indirect research and development expenses:
Personnel related (including stock-based compensation)			218,210				488,888				(270,678	)
Consulting expense			180,604				73,885				106,719
Patent expense			187,734				98,411				89,323
Amortization expense			15,377				1,668				13,709
Total research and development expenses		$	1,192,865			$	1,053,769			$	139,096

The increase in research and development expense for the three months ended September 30, 2021, compared to the three months ended September 30, 2020, is primarily attributed to increased costs associated with external contract research organizations for internal programs of $200,023 as the company ramps up key internal programs, increased patent expense of $89,323 due to increased maintenance fees, increased outside consultants of $106,719 and increase in amortization of property and equipment and intangible asset of $13,709 offset by decreased contracted research salaries and associated costs of $247,792 due to reduction in compensation to management and attrition of contract staff and decreased share-based compensation of $22,886 due to the forfeiture of share options.

General and Administrative

General and administrative expenses consist of the following:

		Three Months Ended September 30,
		2021				2020				Change
Personnel related (including stock-based compensation)		$	374,055			$	242,571			$	131,484
Professional and consulting fees			470,493				337,446				133,047
Facility-related and other			8,147				(69,753	)			77,900
Total general and administrative expenses		$	852,695			$	510,264			$	342,431

The increase for the three months ended September 30, 2021, compared to the three same period in 2020, is primarily attributable to by an increase in share-based compensation of $212,237 due to the grant of share options, increased legal expense of $52,959, increased to other professional fees of $150,162 and foreign exchange losses of $88,451 due to the foreign exchange on U.S. denominated assets and liabilities offset by a reduction in contracted corporate salaries and associated facility costs of $91,304 due to reduction in compensation to management and attrition of contracted staff and decreased investor relations of $70,074 due to scale down of investor relations activities and consultants

Other Income

The increase in other income is primarily the change in the fair value of the derivative liability associated with the convertible debenture financing warrant liability arising from the August 2021 financing.

Results of Operations – Nine months ended September 30, 2021 and 2020

The following table summarizes our results of operations for the nine months ended September 30, 2021 and 2020:

		Nine Months Ended
		September 30,
		2021				2020				Change
Revenues		$	5,101			$	1,578			$	3,523
Operating expenses
Research and development			2,451,985				2,926,242				(474,257	)
General and administrative			1,554,509				2,051,506				(496,997	)
Total operating expenses			4,006,494				4,977,748				(971,254	)
Loss from operations			4,001,393				4,976,170				(974,777	)
Other expense			4,857,346				–				4,857,346
Net loss		$	8,858,739			$	4,976,170			$	3,882,569

Research and Development

Research and development expenses consist of the following:

		Nine Months Ended
		September 30,
		2021				2020				Change
Direct research and development expenses by program:		$	1,173,873			$	885,179			$	288,694
Indirect research and development expenses:
Personnel related (including stock-based compensation)			476,161				1,566,083				(1,089,922	)
Consulting expense			370,052				176,898				193,154
Patent expense			386,018				293,078				92,940
Other operating costs			45,881				5,004				40,877
Total research and development expenses		$	2,451,985			$	2,926,242			$	(474,257	)

The decrease in research and development expense for the nine months ended September 30, 2021, compared to the nine months ended September 30, 2020, reflects the conservation of cash resources and decreased contract salaries and associated costs of $921,576 due to reduction in compensation to management and attrition of contracted staff and decreased share-based compensation of $168,346 due to forfeiture of unvested/vested share options due to termination of consulting arrangement offset by increased costs associated with external contract research organizations for internal programs of $288,694 as the company ramps up key internal programs, increased patent expense of $92,940 due to increased maintenance fees, increased consulting expense of $193,154 and increase in amortization of property and equipment and intangible asset of $40,877.

General and Administrative

General and administrative expenses consist of the following:

		Nine Months Ended September 30,
		2021				2020				Change
Personnel related (including stock-based compensation)		$	802,356			$	910,769			$	(108,413	)
Professional and consulting fees			950,285				1,083,745				(133,460	)
Facility-related and other			(198,132	)			56,992				(255,124	)
Total general and administrative expenses		$	1,554,509			$	2,051,506			$	(496,997	)

The decrease for the nine months ended September 30, 2021, compared to the same period in 2020, is primarily attributable to a reduction in contracted corporate salaries and associated facility costs of $342,084 due to reduction in compensation to management and attrition of contracted staff, decreased investor relations of $451,033 due to a reduction of investor relation activities and consultants and foreign exchange gains of $204,561 on U.S. denominated assets and liabilities offset by increased legal expense of $152,531, increased other professional fees of 165,042 and increased share-based compensation of $183,108, related to the grant of share options.

Other Expense

The increase in other expense is primarily the valuation of the derivative liability associated with the convertible debenture financing.

Outlook

Going forward we will focus on therapeutic programs in our core business area of differentiated antibodies for neurodegenerative and other mis-folded protein diseases.

PMN310, ProMIS antibody therapy selective for toxic oligomers in Alzheimer’s disease, is our highest priority. In Q3, we made significant progress, in line with plans, on all the program elements discussed in the prospectus supplement in August 2021, including cell line development, GLP toxicology work, and CMC manufacturing. We are on track to complete all IND enabling work in H2 2022.

The top priority for our scientific validation efforts, largely centered in Dr. Neil Cashman’s lab at UBC, is currently our ALS portfolio. This portfolio includes antibodies targeting mis-folded forms of TDP-43, RACK1, SOD1, and ataxin2. The most advanced of these is the program targeting TDP-43. We have initiated both in vitro assays (assessing the impact of drug on motor neuron cell lines) and in vivo (mouse model) assays and expect readouts over the next several months.

In addition, we are continuing to expand the application of our unique discovery platform, with which we can “rationally design” antibodies to be selective for only mis-folded, pathogenic proteins involved in disease. Our Chief Physics Officer, David Wishart, and his team are pursuing multiple novel targets. We have acquired access to the AlphaFold database of over 300,000 normal protein conformations, which is the starting point for our predictions of conformational epitopes on mis-folded molecular species using our proprietary computational algorithm Collective Coordinates.

About ProMIS Neurosciences, Inc.

ProMIS Neurosciences, Inc. is a development stage biotechnology company focused on discovering and developing antibody therapeutics selectively targeting toxic oligomers implicated in the development and progression of neurodegenerative diseases, in particular Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Parkinson’s disease (PD). The Company’s proprietary target discovery engine is based on the use of two complementary techniques. The Company applies its thermodynamic, computational discovery platform – ProMIS™ and Collective Coordinates – to predict novel targets known as Disease Specific Epitopes on the molecular surface of misfolded proteins. Using this unique approach, the Company is developing novel antibody therapeutics for AD, ALS and PD. ProMIS is headquartered in Toronto, Ontario, with offices in Cambridge, Massachusetts. ProMIS is listed on the Toronto Stock Exchange under the symbol PMN, and on the OTCQB Venture Market under the symbol ARFXF.

Visit us at www.promisneurosciences.com, follow us on Twitter and LinkedIn

For Investor Relations please contact:
Alpine Equity Advisors
Nicholas Rigopulos, President
nick@alpineequityadv.com
Tel. 617 901-0785

The TSX has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This information release contains certain forward-looking information. Such information involves known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements to be materially different from those implied by statements herein, and therefore these statements should not be read as guarantees of future performance or results. All forward-looking statements are based on the Company’s current beliefs as well as assumptions made by and information currently available to it as well as other factors. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Due to risks and uncertainties, including the risks and uncertainties identified by the Company in its public securities filings, actual events may differ materially from current expectations. The Company disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Source: ProMIS Neurosciences Inc.

ProMIS Neurosciences Announces Third Quarter 2021 Results

 

TORONTO and CAMBRIDGE, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) — ProMIS Neurosciences, Inc. (TSX: PMN) (OTCQB: ARFXF) (“ProMIS or the Company”), a biotechnology company focused on the discovery and development of antibody therapeutics targeting toxic oligomers implicated in the development of neurodegenerative diseases, today announced its operational and financial results for the three and nine months ended September 30, 2021.

“We are pleased to be ramping up our efforts to advance our lead asset, PMN 310, closer toward the clinic,” said Gene Williams, ProMIS’ Chairman and CEO. “The financing we secured earlier this year is enabling us to unlock the potential of our platform, which we believe could have significant impact on the treatment of several neurological diseases, including Alzheimer’s Disease (AD), Parkinson’s Disease and ALS. The strengthening of our management team and Board this quarter has also enabled us to leverage worldwide development and patent expertise and strengthen our overall competitive position.”

Corporate Highlights

• On July 2, 2021, the Company announced the voting results of its annual meeting of shareholders held on June 30, 2021, in Vancouver, British Columbia, Canada. All resolutions described in the Management Proxy Circular and placed before the meeting were approved by the shareholders.
• On July 8, 2021, the Company announced that it had filed and obtained a receipt for the Prospectus with the securities regulators in each of the provinces and territories of Canada, except Quebec.
• On August 25, 2021, we announced the closing of a public offering for gross proceeds of US$20,125,000 (CDN$25,522,525).
• On October 7, 2021, we announced that we would hold a special general meeting of shareholders (the “Special Meeting”) on December 1, 2021. We set October 18, 2021, as the record date for the Special Meeting. The purpose of the Special Meeting is to ask shareholders to grant the Board of Directors the authority, exercisable in the Board’s discretion, to consolidate (or reverse split) the Company’s issued and outstanding common shares in furtherance of a potential listing of the Company’s shares on a stock exchange in the United States.

People

• On September 1, 2021, the Company appointed Josh Mandel-Brehm to the board of directors. Mr. Mandel-Brehm has held various key business development and operations leadership roles at leading biotechnology companies.
• On September 23, 2021, the Company appointed Maggie Shafmaster, JD, PhD, to the board of directors. Dr Shafmaster has approximately 30 years of experience providing intellectual property advice to biotechnology and pharmaceutical industries.

On October 22, 2021, the Company announced the expansion of its senior management team. The following changes were announced:

• Eugene Williams, formerly Executive Chairman, takes on the role of Chairman and Chief Executive Officer (“CEO”), with immediate effect.
• Dr. Elliot Goldstein resigned from his current role as CEO with immediate effect and continues to support us as President and special consultant to the CEO.
• Gavin Malenfant joins our senior management team as Chief Operating Officer. Mr. Malenfant brings more than 30 years of biopharmaceutical experience to our team, with special focus on providing expert management and oversight of drug development programs. The top priority in the near term will be to support the timely development of the PMN310 program to completion of IND enabling activities, anticipated in the second half of 2022. He will be working with Mr. Williams and the leadership of the PMN310 project team, whose key members include:
  • Michael Grundman, MD, Senior Medical Adviser. Prior to joining the pharmaceutical industry, Dr. Grundman was Associate Director of the Alzheimer’s Disease Cooperative Study at the University of California, San Diego (“UCSD”) and is currently an Adjunct Professor of Neurosciences at UCSD. Dr. Grundman previously served on the FDA Peripheral and Central Nervous System Advisory Committee.
  • Ernest Bush, PhD, Head of Pharmacology/Toxicology. Dr. Bush has 35 years of experience working in the field of biomedical research and development, driving development of innovative therapies for treatment of human diseases. He has served as a consultant in non-clinical development providing advice and insight into Investigational New Drug (“IND”) enabling programs, pre-clinical data-set analysis for due diligence and evaluation and audits of Good Laboratory Practices (“GLP”) bioanalytical and toxicology facilities and studies.
  • Dennis Chen, PhD, Head of Manufacturing. Dennis has more than 25 years of prior pharmaceutical experience in working with companies from virtual to global and all phases of development. Dennis provides Regulatory Affairs, Chemistry, Chemistry, Manufacturing and Controls (“CMC”) and Biopharmaceutical Development support to ProMIS with expertise in peptides, proteins and oligonucleotides.

Financial Results

Results of Operations – Three months ended September 30, 2021 and 2020

The following table summarizes our results of operations for the three months ended September 30, 2021 and 2020:

		Three Months Ended
		September 30,
		2021				2020				Change
Revenues		$	5,101			$	–			$	5,101
Operating expenses
Research and development		$	1,192,865			$	1,053,769			$	139,096
General and administrative			852,695				510,264				342,431
Total operating expenses			2,045,560				1,564,033				481,527
Loss from operations			2,040,459				1,564,033				476,426
Other income			(1,078,483	)			–				(1,078,483	)
Net loss		$	961,976			$	1,564,033			$	(602,057	)

Research and Development

Research and development expenses consist of the following:

		Three Months Ended
		September 30,
		2021				2020				Change
Direct research and development expenses by program:		$	590,940			$	390,917			$	200,023
Indirect research and development expenses:
Personnel related (including stock-based compensation)			218,210				488,888				(270,678	)
Consulting expense			180,604				73,885				106,719
Patent expense			187,734				98,411				89,323
Amortization expense			15,377				1,668				13,709
Total research and development expenses		$	1,192,865			$	1,053,769			$	139,096

The increase in research and development expense for the three months ended September 30, 2021, compared to the three months ended September 30, 2020, is primarily attributed to increased costs associated with external contract research organizations for internal programs of $200,023 as the company ramps up key internal programs, increased patent expense of $89,323 due to increased maintenance fees, increased outside consultants of $106,719 and increase in amortization of property and equipment and intangible asset of $13,709 offset by decreased contracted research salaries and associated costs of $247,792 due to reduction in compensation to management and attrition of contract staff and decreased share-based compensation of $22,886 due to the forfeiture of share options.

General and Administrative

General and administrative expenses consist of the following:

		Three Months Ended September 30,
		2021				2020				Change
Personnel related (including stock-based compensation)		$	374,055			$	242,571			$	131,484
Professional and consulting fees			470,493				337,446				133,047
Facility-related and other			8,147				(69,753	)			77,900
Total general and administrative expenses		$	852,695			$	510,264			$	342,431

The increase for the three months ended September 30, 2021, compared to the three same period in 2020, is primarily attributable to by an increase in share-based compensation of $212,237 due to the grant of share options, increased legal expense of $52,959, increased to other professional fees of $150,162 and foreign exchange losses of $88,451 due to the foreign exchange on U.S. denominated assets and liabilities offset by a reduction in contracted corporate salaries and associated facility costs of $91,304 due to reduction in compensation to management and attrition of contracted staff and decreased investor relations of $70,074 due to scale down of investor relations activities and consultants

Other Income

The increase in other income is primarily the change in the fair value of the derivative liability associated with the convertible debenture financing warrant liability arising from the August 2021 financing.

Results of Operations – Nine months ended September 30, 2021 and 2020

The following table summarizes our results of operations for the nine months ended September 30, 2021 and 2020:

		Nine Months Ended
		September 30,
		2021				2020				Change
Revenues		$	5,101			$	1,578			$	3,523
Operating expenses
Research and development			2,451,985				2,926,242				(474,257	)
General and administrative			1,554,509				2,051,506				(496,997	)
Total operating expenses			4,006,494				4,977,748				(971,254	)
Loss from operations			4,001,393				4,976,170				(974,777	)
Other expense			4,857,346				–				4,857,346
Net loss		$	8,858,739			$	4,976,170			$	3,882,569

Research and Development

Research and development expenses consist of the following:

		Nine Months Ended
		September 30,
		2021				2020				Change
Direct research and development expenses by program:		$	1,173,873			$	885,179			$	288,694
Indirect research and development expenses:
Personnel related (including stock-based compensation)			476,161				1,566,083				(1,089,922	)
Consulting expense			370,052				176,898				193,154
Patent expense			386,018				293,078				92,940
Other operating costs			45,881				5,004				40,877
Total research and development expenses		$	2,451,985			$	2,926,242			$	(474,257	)

The decrease in research and development expense for the nine months ended September 30, 2021, compared to the nine months ended September 30, 2020, reflects the conservation of cash resources and decreased contract salaries and associated costs of $921,576 due to reduction in compensation to management and attrition of contracted staff and decreased share-based compensation of $168,346 due to forfeiture of unvested/vested share options due to termination of consulting arrangement offset by increased costs associated with external contract research organizations for internal programs of $288,694 as the company ramps up key internal programs, increased patent expense of $92,940 due to increased maintenance fees, increased consulting expense of $193,154 and increase in amortization of property and equipment and intangible asset of $40,877.

General and Administrative

General and administrative expenses consist of the following:

		Nine Months Ended September 30,
		2021				2020				Change
Personnel related (including stock-based compensation)		$	802,356			$	910,769			$	(108,413	)
Professional and consulting fees			950,285				1,083,745				(133,460	)
Facility-related and other			(198,132	)			56,992				(255,124	)
Total general and administrative expenses		$	1,554,509			$	2,051,506			$	(496,997	)

The decrease for the nine months ended September 30, 2021, compared to the same period in 2020, is primarily attributable to a reduction in contracted corporate salaries and associated facility costs of $342,084 due to reduction in compensation to management and attrition of contracted staff, decreased investor relations of $451,033 due to a reduction of investor relation activities and consultants and foreign exchange gains of $204,561 on U.S. denominated assets and liabilities offset by increased legal expense of $152,531, increased other professional fees of 165,042 and increased share-based compensation of $183,108, related to the grant of share options.

Other Expense

The increase in other expense is primarily the valuation of the derivative liability associated with the convertible debenture financing.

Outlook

Going forward we will focus on therapeutic programs in our core business area of differentiated antibodies for neurodegenerative and other mis-folded protein diseases.

PMN310, ProMIS antibody therapy selective for toxic oligomers in Alzheimer’s disease, is our highest priority. In Q3, we made significant progress, in line with plans, on all the program elements discussed in the prospectus supplement in August 2021, including cell line development, GLP toxicology work, and CMC manufacturing. We are on track to complete all IND enabling work in H2 2022.

The top priority for our scientific validation efforts, largely centered in Dr. Neil Cashman’s lab at UBC, is currently our ALS portfolio. This portfolio includes antibodies targeting mis-folded forms of TDP-43, RACK1, SOD1, and ataxin2. The most advanced of these is the program targeting TDP-43. We have initiated both in vitro assays (assessing the impact of drug on motor neuron cell lines) and in vivo (mouse model) assays and expect readouts over the next several months.

In addition, we are continuing to expand the application of our unique discovery platform, with which we can “rationally design” antibodies to be selective for only mis-folded, pathogenic proteins involved in disease. Our Chief Physics Officer, David Wishart, and his team are pursuing multiple novel targets. We have acquired access to the AlphaFold database of over 300,000 normal protein conformations, which is the starting point for our predictions of conformational epitopes on mis-folded molecular species using our proprietary computational algorithm Collective Coordinates.

About ProMIS Neurosciences, Inc.

ProMIS Neurosciences, Inc. is a development stage biotechnology company focused on discovering and developing antibody therapeutics selectively targeting toxic oligomers implicated in the development and progression of neurodegenerative diseases, in particular Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Parkinson’s disease (PD). The Company’s proprietary target discovery engine is based on the use of two complementary techniques. The Company applies its thermodynamic, computational discovery platform – ProMIS™ and Collective Coordinates – to predict novel targets known as Disease Specific Epitopes on the molecular surface of misfolded proteins. Using this unique approach, the Company is developing novel antibody therapeutics for AD, ALS and PD. ProMIS is headquartered in Toronto, Ontario, with offices in Cambridge, Massachusetts. ProMIS is listed on the Toronto Stock Exchange under the symbol PMN, and on the OTCQB Venture Market under the symbol ARFXF.

Visit us at www.promisneurosciences.com, follow us on Twitter and LinkedIn

For Investor Relations please contact:
Alpine Equity Advisors
Nicholas Rigopulos, President
nick@alpineequityadv.com
Tel. 617 901-0785

The TSX has not reviewed and does not accept responsibility for the adequacy or accuracy of this release. This information release contains certain forward-looking information. Such information involves known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements to be materially different from those implied by statements herein, and therefore these statements should not be read as guarantees of future performance or results. All forward-looking statements are based on the Company’s current beliefs as well as assumptions made by and information currently available to it as well as other factors. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. Due to risks and uncertainties, including the risks and uncertainties identified by the Company in its public securities filings, actual events may differ materially from current expectations. The Company disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Source: ProMIS Neurosciences Inc.

Friday, November 12, 2021

Onconova Therapeutics (ONTX)
3Q Report Highlights Clinical Progress

Onconova Therapeutics Inc is a clinical-stage biopharmaceutical company operating in the US. It focuses on discovering and developing novel small molecule product candidates primarily to treat cancer. The company has created a library of targeted agents designed to work against cellular pathways important to cancer cells. Its product candidates are Single-agent IV rigosertib, Oral rigosertib + azacitidine, IV Briciclib, Recilisib, and ON 123300. The key product candidate Rigosertib is a small molecule which blocks cellular signaling by targeting RAS effector pathways.

Robert LeBoyer, Senior Research Analyst, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

3Q21 reported. Onconova Therapeutics reported 3Q21 loss of $3.5 million or $(0.22) per share, compared with our estimated loss of $5.2 million or $(0.33) per share. On its quarterly conference call, the company reviewed clinical progress during the quarter and discussed plans for trials in 2022. Cash balance at the end of the quarter was $59.4 million.


Narazaciclib updates.  The company gave an update on the two clinical dose escalation trials for narazaciclib, the new name for ON 123300. The US trial is enrolling its second dose cohort, while the study in China is in its fourth cohort. Data to establish safety and select the dose for Phase 3 is expected in 1H22. This Phase 2 study is expected to include multiple cancer types including patients with …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

Thursday, November 11, 2021

PDS Biotechnology Corp (PDSB)
3Q21 Contained Clinical Progress and New Product Licenses

PDS Biotechnology Corp operates as a clinical stage biotechnology company, principally involved in drug discovery in the United States. It is primarily engaged in the treatment of various early-stage and late-stage cancers, including head and neck cancer, prostate cancer, breast cancer, cervical cancer, anal cancer, and other cancers. Its products are based on the proprietary Versamune platform technology, which activates and directs the human immune system to unleash a powerful and targeted attack against cancer cells.

Robert LeBoyer, Senior Research Analyst, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

PDS Biotechnology reported 3Q21 loss of $6.9 million or $(0.24) per share, compared with our estimated loss of $6.1 million or $(0.21) per share. The company held a conference call in which it reviewed two licensing agreements announced earlier in November 2021. These products advance the pipeline and broaden the development of products that use the Versamune technology. Cash balance at the end of the quarter was $69.7 million.


PDS0102 Reached A Development Milestone PDS0102 has been in preclinical testing for prostate cancer, breast cancer, and acute myeloid leukemia (AML) using the TARP antigen with the Versamune platform.  Based on its strong results, PDS made a license agreement with the NCI (National Cancer Institute) that gives the company rights to NCI’s intellectual property related to the antigen. The license …

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary.  Proper due diligence is required before making any investment decision. 

TherapeuticsMD Announces Leadership Changes; Appointment of Industry Veteran, Hugh O’Dowd, as Chief Executive Officer

 

– Mr. O’Dowd to succeed  Robert G. Finizio, effective on or before 
December 31, 2021 –

–  Mr. Finizio appointed Vice Chair of the Board –

BOCA RATON, Fla.–(BUSINESS WIRE)–Nov. 11, 2021– 
TherapeuticsMD, Inc. (NASDAQ: TXMD) (TXMD or the Company), an innovative, leading women’s healthcare company, today announced key leadership changes, including the appointment of Hugh O’Dowd, the Company’s current President, as the Company’s Chief Executive Officer and member of the board of directors. Mr. O’Dowd will succeed  Robert G. Finizio, the Company’s Co-founder and current Chief Executive Officer, effective on or before 
December 31, 2021.  Mr. Finizio will continue with the Company and has been appointed Vice Chair of the Board of Directors.

“I want to thank Rob for his strong leadership and vision over the past 13 years,” said Honorable  Tommy Thompson, Chairman of the Board of 
TherapeuticsMD. “Rob is an innovator who has made an indelible mark not only on this company, but on the women’s healthcare industry as a whole.”

“Founding TherapeuticsMD has been one of the highlights of my career. Hugh is an experienced leader with a strong track record of delivering results, and in the few short months since joining the company, he has already made invaluable contributions. I am confident that he is the right person to bring 
TherapeuticsMD to the next level of growth,” said  Mr. Finizio.

“TherapeuticsMD is an innovator in women’s healthcare, and I welcome the opportunity to drive operating performance and craft our long-term strategy,” stated Mr. O’Dowd. “Rob has created a dynamic company and established TXMD’s foundation for growth and I will build upon our mission of empowering women of all ages through better healthcare.”

Mr. O’Dowd previously served as President, Chief Executive Officer, and member of the Board of Directors of 
Neon Therapeutics, Inc., a clinical-state immuno-oncology company until its acquisition by BioNTech SE in 
May 2020. Prior to Neon Therapeutics, Mr. O’Dowd spent more than 20 years in a variety of senior leadership roles at 
Novartis Pharmaceuticals Corporation, where he served as Country President and General Manager of the 
United Kingdom and 
Ireland, Senior Vice President and Chief Commercial Officer of Novartis Oncology, and Vice President, Latin America Region Head for the Oncology business unit. During his time as Chief Commercial Officer Oncology, Mr. O’Dowd was responsible for the oncology portfolio strategy for the world’s then second-largest oncology/hematology organization, including global brand leadership, business development/licensing, and commercialization. Mr. O’Dowd currently serves as Director and Non-executive Chairman of 
ONK Therapeutics Ltd, an innovative natural killer cell therapy company, and as a Director of Polyphor AG, a clinical-stage biopharmaceutical company focused on the discovery and development of antibiotics and immuno-oncology compounds. Mr. O’Dowd received an MBA from the 
Kellstadt Graduate School of Business at 
DePaul University in 
Chicago and a B.A. from 
Loyola University Chicago.

About TherapeuticsMD, Inc.

TherapeuticsMD, Inc. is an innovative, leading healthcare company, focused on developing and commercializing novel products exclusively for women. Our products are designed to address the unique changes and challenges women experience through the various stages of their lives with a therapeutic focus in family planning, reproductive health, and menopause management. The company is committed to advancing the health of women and championing awareness of their healthcare issues. To learn more about 
TherapeuticsMD, please visit therapeuticsmd.com or follow us on Twitter: @TherapeuticsMD and on Facebook: 
TherapeuticsMD.

Forward-Looking Statements

This press release by 
TherapeuticsMD, Inc. may contain forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to TherapeuticsMD’s objectives, plans and strategies as well as statements, other than historical facts, that address activities, events or developments that the company intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as “believes,” “hopes,” “may,” “anticipates,” “should,” “intends,” “plans,” “will,” “expects,” “estimates,” “projects,” “positioned,” “strategy” and similar expressions and are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and the company undertakes no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties, many of which are outside of the company’s control. Important factors that could cause actual results, developments and business decisions to differ materially from forward-looking statements are described in the sections titled “Risk Factors” in the company’s filings with the 
Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, as well as reports on Form 8-K, and include the following: the effects of the COVID-19 pandemic; the company’s ability to maintain or increase sales of its products; the company’s ability to develop and commercialize IMVEXXY®, ANNOVERA®, and BIJUVA® and obtain additional financing necessary therefor; whether the company will be able to comply with the covenants and conditions under its term loan facility; whether the company will be able to successfully divest, or obtain an investment in, its vitaCare business and how the proceeds that may be generated by any such divestiture or investment will be utilized; the effects of supply chain issues on the supply of the company’s products; the potential of adverse side effects or other safety risks that could adversely affect the commercialization of the company’s current or future approved products or preclude the approval of the company’s future drug candidates; whether the FDA will approve the lower dose of BIJUVA and the manufacturing supplement for ANNOVERA; the company’s ability to protect its intellectual property, including with respect to the Paragraph IV notice letters the company received regarding IMVEXXY and BIJUVA; the length, cost and uncertain results of future clinical trials; the company’s reliance on third parties to conduct its manufacturing, research and development and clinical trials; the ability of the company’s licensees to commercialize and distribute the company’s products; the ability of the company’s marketing contractors to market ANNOVERA; the availability of reimbursement from government authorities and health insurance companies for the company’s products; the impact of product liability lawsuits; the influence of extensive and costly government regulation; the impact of leadership transitions; the volatility of the trading price of the company’s common stock and the concentration of power in its stock ownership.

Lisa M. Wilson

In-Site Communications, Inc.
212-452-2793
lwilson@insitecony.com

Source: 
TherapeuticsMD, Inc.

TherapeuticsMD Announces Third Quarter 2021 Financial Results

 

– Quarterly total net product revenue of 
$24.5 million, an increase of 41.1% over Q3 2020 –

– ANNOVERA^® TRx of 8,351, an increase of 62.7% over Q3 2020 –

– Cost savings initiative to reduce SG&A by 
$40 million in 2022; anticipated additional savings of approximately 
$20 million annualized tied to the divestiture of vitaCare –

– Hugh O’Dowd, President, to become Chief Executive Officer;  Robert Finizio appointed Vice Chair of the Board –

– Conference call scheduled for 
8:30 a.m. ET today –

BOCA RATON, Fla.–(BUSINESS WIRE)–Nov. 11, 2021– 
TherapeuticsMD, Inc. (“TXMD” or the “Company”) (NASDAQ: TXMD), an innovative, leading women’s healthcare company, today reported financial results for the third quarter ended 
September 30, 2021. In addition, today the Company announced a significant cost savings initiative designed to reduce its annual costs in 2022 by at least 
$40 million. This figure does not include savings from, or the costs associated with, the divestiture of vitaCare, which are estimated at approximately 
$20 million annually.

“We have made significant changes to our business strategy, which we believe will help us achieve our goal of EBITDA breakeven in the second half of 2022. Specifically, we put a cost savings plan in place and we have implemented a more concerted focus on healthcare professionals. These refinements are already yielding results, as evidenced by the steady progress made during the third quarter, notably the strong year-over-year growth in ANNOVERA prescriptions,” said Hugh O’Dowd, President of 
TherapeuticsMD.

“I would like to thank Rob for his leadership and vision in creating an innovative healthcare company with products that benefit women across their lifecycles. I would also like to formally welcome  Mark Glickman as our Chief Commercial Officer. His commercial acumen is already having a positive impact on our day-to-day operations. Looking ahead, I am confident that we can reduce our annual expenses significantly, deliver value to shareholders, and most importantly, bring our high-quality products to the women who need them,” concluded O’Dowd.

Third Quarter 2021 Financial Results and Business Highlights

Net Product Revenue (in thousands)

		Three Months Ended
		September 30,
		2021				2020
Product revenue:
ANNOVERA		$	11,807			$	6,419
IMVEXXY			8,016				6,841
BIJUVA			3,298				1,646
Prescription vitamin			1,348				2,436
Product revenue, net			24,469				17,342
License revenue			937				2,000
Total revenue, net		$	25,406			$	19,342

ANNOVERA (segesterone acetate and ethinyl estradiol vaginal system)

• ANNOVERA net product revenue of 
  $11.8 million for the third quarter of 2021 increased by 
  $5.4 million compared to 
  $6.4 million for the third quarter of 2020.
• Approximately 8,350 ANNOVERA prescriptions were dispensed to patients during the third quarter of 2021. Prescriptions increased 62.7% compared to the third quarter of 2020.
• Over 4,500 health care providers (HCPs) prescribed ANNOVERA during the third quarter, of which nearly 29% were new writers.
  • Growth in prescribers of approximately 1,600 over third quarter of 2020.
  • Cumulatively over 9,450 HCPs have prescribed ANNOVERA.

IMVEXXY^® (estradiol vaginal inserts)

• IMVEXXY net product revenue of 
  $8.0 million for the third quarter of 2021 increased by 
  $1.2 million compared to 
  $6.8 million for the third quarter of 2020.
• Approximately 113,000 IMVEXXY prescriptions were dispensed to patients during the third quarter of 2021.
• Full re-targeting initiative taking place in the fourth quarter of 2021, with implementation in the first quarter of 2022.
  • Plan to rejuvenate growth and optimize HCP focus.

BIJUVA^® (estradiol and progesterone)

• BIJUVA net product revenue of 
  $3.3 million for the third quarter of 2021 increased by 
  $1.7 million compared to 
  $1.6 million for the third quarter of 2020.
• BIJUVA net product revenue for the third quarter of 2021 includes 
  $0.7 million of export sales through our international licensing and supply agreement with 
  Theramex HQ UK Limited.
• Re-targeting initiative taking place, similar to the process with IMVEXXY.

Cost of Goods Sold and Gross Margin

• Cost of goods was 
  $5.3 million with product gross margin of 78% for the third quarter of 2021 compared to 
  $3.3 million with product gross margin of 81% for the third quarter of 2020. The lower product gross margin for the third quarter of 2021 reflects the impact of 
  $0.7 million of BIJUVA export sales, which were sold at cost.

Operating Expense, Net Loss and Related Information

• Total operating expense of 
  $60.0 million for the third quarter of 2021 increased by 
  $19.0 million compared to 
  $41.0 million for the third quarter of 2020. Included in total operating expense for the third quarter of 2021 was 
  $7.3 million of severance related expenses recorded for certain former senior executives.
• Net loss for the third quarter of 2021 was 
  $47.4 million, or 
  $0.11 per basic and diluted share, compared to net loss for the third quarter of 2020 of 
  $32.6 million, or 
  $0.12 per basic and diluted share.

Balance Sheet

• As of 
  September 30, 2021, the Company’s cash on hand totaled 
  $104.8 million, compared with 
  $80.5 million as of 
  December 31, 2020.
• For the first nine months of 2021, the Company received 
  $182.9 million in net proceeds from its at-the-market and underwritten equity offerings.
• As of 
  September 30, 2021, the remaining outstanding principal amount under the Company’s Financing Agreement was 
  $200.0 million, which reflects a repayment of 
  $50.0 million of principal during the first nine months of 2021.

The contract manufacturing organization that manufactures ANNOVERA has recently experienced an increase in difficulties with the manufacturing process for ANNOVERA resulting in batch failures. The Company filed a supplemental NDA with the FDA to modify the manufacturing (testing) specification for ANNOVERA to allow for normal manufacturing variation that would increase the consistency of manufacturing and supply of ANNOVERA. The Company expects that the FDA will act on the supplemental NDA by the Prescription Drug User Fee Act (“PDUFA”) date of 
December 12, 2021. If the FDA does not approve the supplemental NDA by the PDUFA date, the Company may not be able to meet the revenue covenants under its Financing Agreement in the near term. The manufacturing difficulties relate only to our ability to meet the release specification, and any ANNOVERA rings currently being sold meet the exacting quality specifications. For more information regarding the covenants under the Financing Agreement, please see the Company’s filings with the 
SEC.

Conference Call and Webcast Details

TherapeuticsMD will host a conference call and live audio webcast today at 
8:30 a.m. ET to discuss these financial results and provide a business update.

Date:	Thursday, November 11, 2021
Time:	8:30 a.m. ET
Telephone Access (US):	866-665-9531
Telephone Access (International):	724-987-6977
Access Code for All Callers:	6341637

A live webcast and audio archive for the event may be accessed on the home page or from the “Investors & Media” section of the 
TherapeuticsMD website at www.therapeuticsmd.com. Please connect to the website prior to the start of the presentation to ensure adequate time for any software downloads that may be necessary to listen to the webcast. A replay of the webcast will be archived on the website for at least 30 days. In addition, a digital recording of the conference call will be available for replay beginning two hours after the call’s completion and for at least 30 days with the dial-in 855-859-2056 or international 404-537-3406 and Conference ID: 6341637.

Please see the Full Prescribing Information, including indication and Boxed WARNING, for each 
TherapeuticsMD product as follows:

• IMVEXXY (estradiol vaginal inserts) at https://imvexxy.com/pi.pdf
• BIJUVA (estradiol and progesterone) capsules at https://www.bijuva.com/pi.pdf
• ANNOVERA (segesterone acetate and ethinyl estradiol vaginal system) at www.annovera.com/pi.pdf

Forward-Looking Statements

This press release by 
TherapeuticsMD, Inc. may contain forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to TherapeuticsMD’s objectives, plans and strategies as well as statements, other than historical facts, that address activities, events or developments that the company intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as “believes,” “hopes,” “may,” “anticipates,” “should,” “intends,” “plans,” “will,” “expects,” “estimates,” “projects,” “positioned,” “strategy” and similar expressions and are based on assumptions and assessments made in light of management’s experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements in this press release are made as of the date of this press release, and the company undertakes no duty to update or revise any such statements, whether as a result of new information, future events or otherwise. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties, many of which are outside of the company’s control. Important factors that could cause actual results, developments and business decisions to differ materially from forward-looking statements are described in the sections titled “Risk Factors” in the company’s filings with the 
Securities and Exchange Commission, including its most recent Annual Report on Form 10-K and Quarterly Reports on Form 10-Q, as well as reports on Form 8-K, and include the following: the effects of the COVID-19 pandemic; the company’s ability to maintain or increase sales of its products; the company’s ability to develop and commercialize IMVEXXY®, ANNOVERA®, and BIJUVA® and obtain additional financing necessary therefor; whether the company will be able to comply with the covenants and conditions under its term loan facility; whether the company will be able to successfully divest, or obtain an investment in, its vitaCare business and how the proceeds that may be generated by any such divestiture or investment will be utilized; the effects of supply chain issues on the supply of the company’s products; the potential of adverse side effects or other safety risks that could adversely affect the commercialization of the company’s current or future approved products or preclude the approval of the company’s future drug candidates; whether the FDA will approve the lower dose of BIJUVA and the manufacturing supplement for ANNOVERA; the company’s ability to protect its intellectual property, including with respect to the Paragraph IV notice letters the company received regarding IMVEXXY and BIJUVA; the length, cost and uncertain results of future clinical trials; the company’s reliance on third parties to conduct its manufacturing, research and development and clinical trials; the ability of the company’s licensees to commercialize and distribute the company’s products; the ability of the company’s marketing contractors to market ANNOVERA; the availability of reimbursement from government authorities and health insurance companies for the company’s products; the impact of product liability lawsuits; the influence of extensive and costly government regulation; the impact of leadership transitions; the volatility of the trading price of the company’s common stock and the concentration of power in its stock ownership.

– Financial Statements to Follow –

TherapeuticsMD, Inc. and Subsidiaries Consolidated Balance Sheets (In thousands, except per share data)
		September 30, 2021				December 31, 2020
		(Unaudited)
Assets:
Current assets:
Cash		$	104,841			$	80,486
Accounts receivable, net of allowance for credit losses of  $1,351 and  $1,118 as of  September 30, 2021 and  December 31, 2020, respectively			37,402				32,382
Inventory			7,362				7,993
Prepaid and other current assets			10,374				7,543
Total current assets			159,979				128,404
Fixed assets, net			1,388				1,942
License rights and other intangible assets, net			39,617				41,445
Right of use assets			8,391				9,566
Other non-current assets			253				253
Total assets		$	209,628			$	181,610
Liabilities and stockholders’ equity (deficit):
Current liabilities:
Current maturities of long-term debt		$	15,000			$	—
Accounts payable			19,592				21,068
Accrued expenses and other current liabilities			51,674				38,170
Total current liabilities			86,266				59,238
Long-term debt, net			171,738				237,698
Operating lease liabilities			8,226				8,675
Other non-current liabilities			758				—
Total liabilities			266,988				305,611
Commitments and contingencies
Stockholders’ equity (deficit):
Preferred stock, par value  $0.001; 10,000 shares authorized, none issued			—				—
Common stock, par value  $0.001; 600,000 shares authorized, 424,879 and 299,765 issued and outstanding as of  September 30, 2021 and  December 31, 2020, respectively			425				300
Additional paid-in capital			950,615				754,644
Accumulated deficit			(1,008,400	)			(878,945	)
Total stockholders’ deficit			(57,360	)			(124,001	)
Total liabilities and stockholders’ equity (deficit)		$	209,628			$	181,610

TherapeuticsMD, Inc. and Subsidiaries Consolidated Statements of Operations (Unaudited – in thousands, except per share data)
		Three Months Ended								Nine Months Ended
		September 30,								September 30,
		2021				2020				2021				2020
Product revenue, net		$	24,469			$	17,342			$	67,102			$	40,294
License revenue			937				2,000				1,171				2,000
Total revenue, net			25,406				19,342				68,273				42,294
Cost of goods sold			5,282				3,279				14,101				10,394
Gross profit			20,124				16,063				54,172				31,900
Operating expenses:
Selling and marketing			30,005				22,373				86,193				91,056
General and administrative			28,435				16,637				66,691				53,740
Research and development			1,605				2,027				5,666				8,038
Total operating expenses			60,045				41,037				158,550				152,834
Loss from operations			(39,921	)			(24,974	)			(104,378	)			(120,934	)
Other (expense) income:
Interest expense and other financing costs			(7,518	)			(7,680	)			(25,341	)			(20,969	)
Other income, net			19				42				264				466
Total other (expense), net			(7,499	)			(7,638	)			(25,077	)			(20,503	)
Loss before income taxes			(47,420	)			(32,612	)			(129,455	)			(141,437	)
Provision for income taxes			—				—				—				—
Net loss		$	(47,420	)		$	(32,612	)		$	(129,455	)		$	(141,437	)
Loss per common share, basic and diluted		$	(0.11	)		$	(0.12	)		$	(0.33	)		$	(0.52	)
Weighted average common shares, basic and diluted			422,216				272,565				388,111				271,969

TherapeuticsMD, Inc. and Subsidiaries
Consolidated Statements of Cash Flows
(Unaudited – in thousands)
		Nine Months Ended  September 30,
		2021				2020
Cash flows from operating activities:
Net loss		$	(129,455	)		$	(141,437	)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization			3,091				3,039
Charges (credits) to provision for doubtful accounts			540				(47	)
Inventory charge			1,082				5,744
Debt financing fees			4,158				1,645
Share-based compensation			12,779				8,502
Other			726				1,719
Changes in operating assets and liabilities:
Accounts receivable			(5,560	)			384
Inventory			(451	)			(3,816	)
Prepaid and other current assets			(2,831	)			2,038
Accounts payable			(1,476	)			(3,072	)
Accrued expenses and other current liabilities			13,504				(3,813	)
Other non-current liabilities			758				—
Total adjustments			26,320				12,323
Net cash used in operating activities			(103,135	)			(129,114	)
Cash flows from investing activities:
Payment of patent related costs			(675	)			(1,065	)
Purchase of fixed assets			(34	)			(39	)
Net cash used in investing activities			(709	)			(1,104	)
Cash flows from financing activities:
Proceeds from sale of common stock, net of costs			182,881				—
Proceeds from exercise of options and warrants			302				272
Proceeds from sale of common stock related to employee stock purchase plan			134				—
Repayments of debt			(50,000	)			—
Borrowings of debt			—				50,000
Payment of debt financing fees			(5,118	)			(1,250	)
Net cash provided by financing activities			128,199				49,022
Net increase in cash			24,355				(81,196	)
Cash, beginning of period			80,486				160,830
Cash, end of period		$	104,841			$	79,634
Supplemental disclosure of noncash financing activities:
Warrants issued in relation to debt financing agreement			—				7,428
Supplemental disclosure of cash flow information:
Interest paid		$	19,675			$	12,032

 

James D’Arecca
Chief Financial Officer
561-961-1900

Lisa M. Wilson

In-Site Communications, Inc.
212-452-2793
lwilson@insitecony.com

Source: 
TherapeuticsMD, Inc.