Ayala Pharmaceuticals Reports Full Year 2020 Financial Results and Provides Business Update

 

– Completed $25 Million Strategic Financing; Extending Cash Runway into
2023

            – Accelerated Development of AL102 Desmoid Tumor Program
into Phase 2/3 Pivotal Trial

– On Track to Report Multiple Milestones in
2021 Across Clinical-Stage Pipeline

REHOVOT, Israel & WILMINGTON, Del., March 25, 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 full year ended December 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs.

“We are pleased with all that we were able to accomplish in 2020 despite the ongoing challenges of the COVID-19 pandemic, keeping clinical execution and patient and employee safety at the forefront of our everyday work. In 2020, we announced encouraging new data from the Phase 2 ACCURACY study of AL101 in R/M ACC, demonstrating initial safety and efficacy for the 4mg monotherapy cohort and we look forward to reporting additional data, including new 6mg cohort results from this program later this year,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “With a strong foundation built in 2020, we have already achieved significant milestones across our broader pipeline in 2021 with the first patient dosing in our Phase 2 TENACITY trial in TNBC, accelerated development pathway and pivotal trial design for AL102 in desmoid tumors, as well as our $25 million strategic financing. We look forward to continuing this momentum with several key clinical milestones expected during the remainder of this year, including two new trial initiations and interim data readouts.”

Business and Clinical Highlights

• Completed $25 million
  Strategic Financing: In February 2021, Ayala announced a $25 million strategic financing with investors including Redmile Group and SIO Capital Management, extending its cash runway into 2023.
• Dosed First Patient in
  Phase 2 TENACITY Clinical Trial of AL101 for the Treatment of
  Notch-Activated Triple Negative Breast Cancer: In January 2021, Ayala announced the dosing of the first patient in the Phase 2 TENACITY clinical trial of its potent, selective small molecule, AL101, for the treatment of patients with Notch-activated recurrent or metastatic (R/M) triple negative breast cancer (TNBC).
• Accelerated Development
  of AL102 for the Treatment of Desmoid Tumors with Pivotal Trial: In January 2021, Ayala announced that based on its end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) on AL102 for the treatment of desmoid tumors, and data from AL101 and AL102 Phase 1 studies
  including durable responses observed in patients with desmoid tumors, the FDA agreed to advance the program into a Phase 2/3 pivotal trial.
• Presented Updated Positive Interim Data from Phase 2 ACCURACY Study of AL101 for the
  Treatment of Recurrent/Metastatic Adenoid Cystic Carcinoma at European
  Society for Medical Oncology (ESMO) Virtual Congress 2020: In September 2020, Ayala presented updated interim data from the 4mg 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, demonstrating meaningful clinical activity of AL101 4mg monotherapy with a 68% disease control rate across 40 evaluable patients. Partial responses were observed in six subjects (15%) and stable disease was observed in 21 subjects (53%).

 

Upcoming Milestones

• On Track to Initiate Phase 2/3 Pivotal RINGSIDE Clinical Trial of AL102 for the
  Treatment of Desmoid Tumors: Ayala expects to initiate the pivotal RINGSIDE clinical trial of AL102 in adult and adolescent patients with desmoid tumors in the first half of 2021. Ayala expects an
  initial interim data read-out from part A and dose selection by mid-2022 with part B of the study
  commencing immediately thereafter.
• Patient
  Enrollment in 6mg Cohort of Phase 2 ACCURACY Study Ongoing: Ayala continues to enroll patients in the 6mg cohort of the Phase 2 ACCURACY study of AL101 for the treatment of R/M ACC, which will contain up to 42 subjects. The Company expects to provide further trial progress updates, including additional data, in the second half of 2021.
• TENACITY Preliminary Data to be Reported in 2021: Ayala expects to report preliminary data from the recently initiated Phase 2 TENACITY clinical trial of AL101 for the treatment of R/M TNBC in the second half of 2021.

 

Full Year 2020 Financial Results

• Cash Position: Cash and cash equivalents were $42.4 million as of December 31, 2020,
  as compared to $16.8 million as of December 31, 2019. The increase in cash and cash equivalents was primarily due to Ayala’s initial public offering in May 2020.
• Collaboration Revenue: Collaboration revenue was $3.7 million for the full year of 2020, as compared to $2.3 million for the same period in 2019. The increase was primarily attributable to the advancement of Ayala’s
  collaboration with Novartis in 2020.
• R&D Expenses: Research and development expenses were $22.4 million for the full year 2020, compared to $14.4 million for the same period in 2019. The increase was primarily driven by an increase in expenses in connection with the advancement of the clinical trials in ACC and TNBC.
• G&A Expenses: General and administrative expenses were $7.4 million for the full year 2020, compared to $4.3 million for the same period in 2019. The increase was primarily related to increased costs in connection with becoming a publicly traded company in 2020.
• Net Loss: Net loss was $30.1 million for the full year 2020, resulting in a basic net loss per share of $3.06 and a diluted net loss per share of $3.06. Net loss was $17.8 million for the same period in 2019, resulting in a basic net loss per share of $3.57 and a diluted net loss per share of $3.57.

 

Financial Guidance

Ayala expects its existing cash balance to fund operating expenses and capital expenditure requirements through multiple potential key clinical and development milestones into 2023.

 

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). Ayala’s lead product candidate, 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 being advanced to a Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com.

 

Contacts:

Investors:
Julie Seidel
Stern Investor Relations, Inc.
+1-212-362-1200
[email protected]

Ayala Pharmaceuticals:
+1-857-444-0553
[email protected]

 

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, patient enrollment and the sufficiency of cash to fund operations. 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.

Source: Ayala Pharmaceuticals

Wednesday, March 24, 2021

Helius Medical Technologies (HSDT)(HSM:CA)
Helius Medical – An Innovative Approach to Neurostimulation

Helius Medical Technologies Inc are a medical technology company. It is focused on the development of products for the treatment of neurological symptoms caused by disease or trauma. The company has developed its first, known as the portable neuromodulation stimulator or PoNS, device, is designed to enhance the brain’s ability to compensate for this damage.

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

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

Initiating Coverage. We are initiating research coverage on Helius Medical Technologies. Through its PoNS device and treatment program, we believe Helius has a first mover advantage in using the tongue for neurostimulation to reduce symptoms of neurological disease or trauma.


PoNS Treatment.  The Portable Neuromodulation Stimulator, or PoNS, is a non-implantable, medical device intended as a short-term treatment of gait deficit due to symptoms from MS and balance deficit due to mmTBI and is to be used in conjunction with physical therapy. PoNS makes use of the tongue as a means neurostimulation. Helius has an extensive patent portfolio on use of the tongue for …

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. 

electroCore, Inc. Announces Exclusive Distribution Agreement with Silvert Medical Nv-Sa. For Western Europe

 

ROCKAWAY, N.J., 
March 23, 2021 (GLOBE NEWSWIRE) — electroCore, Inc. (Nasdaq: ECOR), a commercial-stage bioelectronic medicine company, today announced it has entered into an agreement with Silvert Medical Nv-Sa. (“Silvert Medical”) whereby Silvert Medical will serve as the exclusive distributor of the gammaCore Sapphire™ non-invasive vagus nerve stimulator (nVNS) to patients suffering from primary headache disorders in 
Belgium, Luxembourg, 
the Netherlands, and 
France.

“Silvert Medical is proud and excited to represent electroCore’s non-invasive and drug-free medical treatment for chronic migraine and cluster headache. gammaCore Sapphire matches our mission of ’Innovation and Solutions for Better Patient Care’ perfectly,” said  Eric Silvert, Owner and Managing Director of Silvert Medical. “Our long-term expertise in pain management and neuromodulation combined with our access to key opinion leaders within our territories such as 
Belgium, Luxembourg, 
the Netherlands, and 
France will help establish a solid and lasting business relationship.”

“We are delighted to collaborate with Silvert Medical as we continue to expand the geographical coverage of our nVNS therapy amongst patients suffering with migraine and cluster headache,” said Iain Strickland, electroCore’s Vice President of European Operations. “Silvert Medical is an experienced medical device supplier and we look forward to supporting them in our common goal of introducing our nVNS therapy, gammaCore Sapphire, in new Western European territories.”

The initial term of the agreement is three years, and it contains customary terms and conditions, including minimum purchase commitments.

About Silvert Medical Nv-Sa.

Belgium, Silvert Medical Nv-Sa brings a wealth of experience, knowledge and keen responsiveness as a medical device supplier and provides innovative and disruptive medical device technologies to the 
Belgium, Luxembourg, 
the Netherlands, and 
France clinical and patient community.

For more information, visit http://www.silvertmedical.com/

About electroCore, Inc.

electroCore, Inc. is a commercial stage bioelectronic medicine company dedicated to improving patient outcomes through its platform non-invasive vagus nerve stimulation therapy initially focused on the treatment of multiple conditions in neurology. The company’s current indications are for the preventative treatment of cluster headache and migraine and acute treatment of migraine and episodic cluster headache.

For more information, visit www.electrocore.com.

About gammaCore™

gammaCore™ (nVNS) is the first non-invasive, hand-held medical therapy applied at the neck as an adjunctive therapy to treat migraine and cluster headache through the utilization of a mild electrical stimulation to the vagus nerve that passes through the skin. Designed as a portable, easy-to-use technology, gammaCore can be self-administered by patients, as needed, without the potential side effects associated with commonly prescribed drugs. When placed on a patient’s neck over the vagus nerve, gammaCore stimulates the nerve’s afferent fibers, which may lead to a reduction of pain in patients.

gammaCore is FDA cleared in 
the United States for adjunctive use for the preventive treatment of cluster headache in adult patients, the acute treatment of pain associated with episodic cluster headache in adult patients, and the acute and preventive treatment of migraine in adolescent (ages 12 and older) and adult patients. gammaCore is CE-marked in the 
European Union for the acute and/or prophylactic treatment of primary headache (Migraine, Cluster Headache, Trigeminal Autonomic Cephalalgias and Hemicrania Continua) and Medication Overuse Headache in adults.

gammaCore is contraindicated for patients with:

• An active implantable medical device, such as a pacemaker, hearing aid implant, or any implanted electronic device
• A metallic device, such as a stent, bone plate, or bone screw, implanted at or near the neck
• An open wound, rash, infection, swelling, cut, sore, drug patch, or surgical scar(s) on the neck at the treatment location

Safety and efficacy of gammaCore have not been evaluated in the following patients:

• Patients diagnosed with narrowing of the arteries (carotid atherosclerosis)
• Patients who have had surgery to cut the vagus nerve in the neck (cervical vagotomy)
• Pediatric patients (younger than 12 years)
• Pregnant women
• Patients with clinically significant hypertension, hypotension, bradycardia, or tachycardia

In the US, the FDA has not cleared gammaCore for the treatment of pneumonia and/or respiratory disorders such as acute respiratory stress disorder associated with COVID-19.

Please refer to the gammaCore Instructions for Use for all of the important warnings and precautions before using or prescribing this product.

Forward-Looking Statements

This press release and other written and oral statements made by representatives of electroCore may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, statements about electroCore’s business prospects and clinical and product development plans; its pipeline or potential markets for its technologies; the timing, outcome and impact of regulatory, clinical and commercial developments; the Company’s business prospects in 
Western Europe and other new markets and other statements that are not historical in nature, particularly those that utilize terminology such as “anticipates,” “will,” “expects,” “believes,” “intends,” other words of similar meaning, derivations of such words and the use of future dates. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the ability to raise the additional funding needed to continue to pursue electroCore’s business and product development plans, the inherent uncertainties associated with developing new products or technologies, the ability to commercialize gammaCore™, the potential impact and effects of COVID-19 on the business of electroCore, electroCore’s results of operations and financial performance, and any measures electroCore has and may take in response to COVID-19 and any expectations electroCore may have with respect thereto, competition in the industry in which electroCore operates and overall market conditions. Any forward-looking statements are made as of the date of this press release, and electroCore assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. Investors should consult all of the information set forth herein and should also refer to the risk factor disclosure set forth in the reports and other documents electroCore files with the 
SEC available at www.sec.gov.

Investors:
Rich Cockrell
CG Capital
404-975-4837
[email protected]

Media Contact:
Summer Diaz
electroCore
816-401-6333
[email protected]

Source: electroCore, Inc.

 

Lineage Cell Therapeutics Presents Additional Interim Data On Opregen® For Dry Amd With Geographic Atrophy

 

• Seventy-Five Percent of All Cohort 4 Patients Have Experienced BCVA Increases
• Visual Acuity Continues to Decline in the Majority of Untreated Eyes
• No Acute or Delayed Inflammation or Rejection of OpRegen Observed, Even in Patients Treated with a Reduced Immunosuppressive Regimen

CARLSBAD, Calif.–(BUSINESS WIRE)–Mar. 23, 2021– 

Lineage Cell Therapeutics, Inc.
 (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell transplants for serious medical conditions, today announced new positive interim results from its ongoing, 24-patient Phase 1/2a clinical study of its lead product candidate, OpRegen. OpRegen is an investigational cell therapy consisting of allogeneic retinal pigment epithelium (RPE) cells administered to the subretinal space for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA). Additional interim data were collected on all 24 patients enrolled in the study, including the 12 patients treated in Cohort 4, which feature less advanced disease, better baseline visual acuity, and smaller areas of GA.

Overall, 9/12 (75%) of the Cohort 4 patients’ treated eyes were at or above baseline visual acuity at their last assessment, based on per protocol scheduled visits ranging from 3 months to > 2 years post- transplant. Improvements in best corrected visual acuity (BCVA) reached up to +19 letters on an EDTRS chart. In contrast, 9/12 (75%) of the patients’ untreated eyes were below baseline entry values at the same time points. Among the newly reported data, three (50%) of the more recently treated Cohort 4 patients have exhibited marked improvements in BCVA ranging from +7 to +16 letters at their last scheduled assessments of at least 4.5 months. Two additional Cohort 4 patients remained within 2 letters of their baseline values (one each above and below). One patient measured 7 letters below baseline.

Previously reported structural improvements in the retina and decreases in drusen density have continued with evidence of durable engraftment of OpRegen cells in some treated patients now extending to more than 5 years in the earliest treated patients. A trend towards slower GA progression in treated compared to fellow eyes also continued, although significant changes in GA growth over a 3-month period following treatment are not expected. Overall, OpRegen has been well tolerated with no unexpected adverse events or serious adverse events.

“Data collected from the six additional Cohort 4 patients which we treated last fall has reinforced our prior results and further supports that treatment with OpRegen may provide clinically meaningful outcomes in dry AMD patients with GA. Improvements in BCVA have become apparent within a few months after dosing, consistent with the predicted activity of an RPE cell transplant,” stated  Brian M. Culley, Lineage CEO. “If these early indications of a treatment effect are maintained or improve further, it will be another positive indicator for the potential of OpRegen to improve outcomes in this condition. We continue to monitor all patients on study and in the coming months we will be looking in particular for indications of retinal restoration, reductions in the size and growth of the areas of GA, and functional improvement in visual acuity. Further, the multi-year stability of OpRegen transplants, some in excess of 5 years without signs of rejection, is notable for the durability of our allogeneic cell therapy approach, especially as patients did not require long-term immunosuppression.”

As part of an ongoing effort to administer the minimally effective dose and duration of immunosuppression, reflecting the COVID pandemic and age of typical AMD patients while ensuring the survival of OpRegen cells, no immunosuppression was utilized beyond the perioperative period of up to 3 months in Cohort 4 patients. Notably, the one OpRegen patient who had received a modified immunosuppressive regimen at baseline which included no tacrolimus and only mycophenolate mofetil, does not show any signs of acute or delayed inflammation or rejection of OpRegen cells. One other patient was diagnosed with COVID shortly after treatment with OpRegen and all immunosuppression was halted and then reinstated once the patient was asymptomatic. This second patient similarly showed no signs of acute or delayed inflammation or rejection of OpRegen cells. Other than the reduced regiments described above, immunosuppressants have been discontinued as scheduled, typically within 90 days post-operatively, and no cases of acute or delayed rejection or inflammation due to OpRegen have been reported.

Additional details regarding this data will be presented as part of a corporate update by  Mr. Culley at the Benzinga Global Biotech Small Cap conference on 
March 24, 2021 at 
11:50am Eastern Time / 
8:50am Pacific Time.  Mr. Culley will also be participating in a panel entitled “Coming Together to Address Unmet Medical Needs,” on 
March 24, 2021 at 
12:50pm Eastern Time / 
9:50am Pacific Time. Interested investors are encouraged to register for the event in advance: https://www.benzinga.com/events/small-cap/biotech/. The live and archived webcasts from the event will be available on the Events and Presentations section of Lineage’s website. Additional videos are available on the Media page of the Lineage website.

About OpRegen

OpRegen is currently being evaluated in a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry AMD with GA. The study enrolled 24 patients into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 better vision patients (vision from 20/65 to 20/250 with smaller areas of GA). Cohort 4 also included patients treated with a new “thaw-and-inject” formulation of OpRegen, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events. Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. Additional objectives include the evaluation of the safety of delivery of OpRegen using the Orbit SDS, manufactured by 
Gyroscope Therapeutics, Ltd.

OpRegen is a registered trademark of 
Cell Cure Neurosciences Ltd., a majority-owned subsidiary of 
Lineage Cell Therapeutics, Inc.

About Dry AMD

Dry age-related macular degeneration (AMD) is a leading cause of adult blindness in the developed world. There are two forms of AMD: wet AMD and dry AMD. Dry AMD is the more common of the two types, accounting for approximately 85-90% of cases. Wet AMD is the less common of the two types, accounting for approximately 10-15% of cases. Global sales of the two leading wet AMD therapies were in excess of 
$10 billion in 2019. Nearly all cases of wet AMD begin as dry AMD. Dry AMD typically affects both eyes. There are currently no 
U.S. Food and Drug Administration (FDA) or 
European Medicines Agency (EMA) approved treatment options available for patients with dry AMD.

About Lineage Cell Therapeutics, Inc. 

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include three allogeneic (“off-the-shelf”) product candidates: (i) OpRegen^®, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.

Forward-Looking Statements

Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to the expected clinical outcomes of dry AMD patients with GA and the expected timing when indications of retinal and reductions in size and expansion of the areas of GA may become apparent. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including risks and uncertainties inherent in Lineage’s business and other risks in Lineage’s filings with the 
Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the 
SEC, including Lineage’s most recent Annual Report on Form 10-K filed with the 
SEC and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

Lineage Cell Therapeutics, Inc. IR
Ioana C. Hone
([email protected])
(442) 287-8963

Solebury Trout IR
Gitanjali Jain Ogawa
([email protected])
(646) 378-2949

Russo Partners – Media Relations
Nic Johnson or David Schull
[email protected]
[email protected]
(212) 845-4242

Source: Lineage Cell Therapeutics, Inc.

Research coverage of Lineage Cell Therapeutics (LCTX) on Channelchek is provided by Noble Capital Markets, Inc. Please refer to the research disclosures on the most recent LCTX report for more information.

 

Small Caps in the COVID-19 Vaccine and Treatment Space

 

In the United States, there are three covid vaccines currently authorized by the FDA and recommended by the CDC (BioNTech/Pfizer, Johnson & Johnson, and Moderna). While larger companies in the U.S. dominate this space, there are still several opportunities both here and in other countries, with billions of people still awaiting vaccinations and various other covid-related treatments.

There are a number of small-cap companies working in the covid space, on their own candidates both in the U.S. and abroad on improved drug delivery or production methods or various medications for Covid-related illnesses.  Below is a list of small cap companies operating (at least in part) in the covid landscape.

 

Cocrystal Pharma (COCP)

Pre-clinical Antiviral Treatment

In February of 2020, Cocrystal Pharma entered into a license agreement with Kansas State University Research Foundation to develop proprietary antiviral compounds for the treatment of coronavirus infections. Pre-clinical studies of COVID-19 inhibitors were initiated in Q2 2020. A lead pre-clinical module was announced in Q4 2020. 

In a March 2021 press release, Cocrystal provided 2 updates on their COVID-19 programs: (1) Advancement of pre-clinical studies with CDI-45205, which demonstrated in vitro and in vivo activity in animal models against the viral pathogens MERS and SARS and has potential for injection or inhalation delivery; and (2) Application of their proprietary structure-based platform technology to discover novel covid inhibitors with the potential for oral administration.

Research,
News, and Advanced Market Data on Cocrystal Pharma

 

Ceapro, Inc. (CRPOF)

In Q3 2020, Ceapro announced that they had achieved the first milestones in the successful development of a PGX-processed yeast beta-glucan product, which could be a potential inhalable therapeutic for COVID-19.  They also made significant technical upgrades to a demo plant to allow for sufficient production of the yeast beta-glucan for human trials. 

Research,
News, and Advanced Market Data on Ceapro

 

electroCore (ECOR)

electroCore has been exploring the potential use of their existing product, gammaCore Sapphire CV, for use on hospitalized COVID-19 patients experiencing respiratory symptoms. In a March 2021 press release, the company announced the completion of enrollment in the investigator-initiated SAVIOR-1 clinical trial to evaluate gammaCore Sapphire CV for COVID-related respiratory conditions.

gammaCore Sapphire CV has received Emergency Use Authorization from the FDA for acute use at home or in a healthcare setting to treat adult patients (both known and suspected COVID-19 cases) who are experiencing an exacerbation of asthma-related dyspnea and reduced airflow.

Research,
News, and Advanced Market Data on electroCore

 

Ocugen (OCGN)

Ocugen partnered with Bharat Biotech to develop COVAXIN, a whole-virion inactivated COVID-19 vaccine, for the U.S. market.  COVAXIN was approved for clinical trials in India in June 2020, with Phase 1 & 2 clinical trials beginning the next month.  A phase 3 trial with up to 25,800 patients commenced in October of 2020. In December, Bharat announced their partnership with Ocugen to develop the vaccine for the United States market.  The vaccine was authorized for emergency use in India on January 3, 2021, despite a lack of published Phase 3 data.

The execution of the co-development agreement for the U.S. market occurred on February 2, 2021.  Under the agreement, profits from the sale of COVAXIN in the U.S. will be shared, with Ocugen retaining 45%.  Ocugen has established a vaccine scientific advisory board and feels they are making steady progress towards developing an Emergency Use Authorization pathway in the United States, according to a March 2021 press release.

COVAXIN demonstrates efficacy of 81%, according to interim Phase 3 results.

News and Advanced Market Data on Ocugen

 

PDS Biotechnology (PDSB)

In June 2020, PDS Biotechnology and Farmacore Biotechnology announced the co-development of a potential COVID-19 Vaccine based on the Versamune platform.  This partnership allowed them to quickly accelerate towards Phase 1 trials in Brazil.  The vaccine candidate (Versamune-CoV-2FC) was designed to provide rapid induction of neutralizing antibodies, as well as killer T-cells and memory T-cells against COVID-19, and to prevent the spread of infection.

In a March 2021 press release, PDS announced that their vaccine consortium had received a commitment from the Ministry of Science, Technology and Innovation of Brazil to fund clinical development and commercialization.  This award (~$60MM) was based on pre-clinical studies which showed strong potential to induce immune responses against the virus.  Phase 1 and 2 trials to assess safety and efficacy will run together in Brazil, and are expected to enroll 360 pateints.

“PDS Biotech and Farmacore Biotechnology have taken the important step of advancing our Versamune®-based COVID-19 vaccine into the clinic,” said Dr. Frank Bedu-Addo, Chief Executive Officer of PDS Biotech.“ The pre-clinical results demonstrate the vaccine’s potential to induce a broad range of robust anti SARS-CoV-2 immune responses. The rapidly increasing number of SARS-CoV-2 mutations highlights the need for novel, second generation vaccines capable of generating both killer and helper T-cells that can recognize and attack conserved and non-mutating regions of the virus. We applaud Farmacore Biotechnology and Blanver Farmoquímica for reaching this important milestone and look forward to the results of the planned human clinical trials and hopefully a rapid advancement towards commercialization of the product. These clinical trials will also advance our understanding of the potential for novel Versamune®-based vaccines to provide long-term protection against infection with viruses with pandemic potential such as SARS-CoV-2.”

Research,
News, and Advanced Market Data on PDS Biotechnology

 

Sources

https://www.cocrystalpharma.com/news/press-releases/detail/115/cocrystal-pharma-reports-2020-financial-results-provides

https://www.ceapro.com/news/press-releases/detail/203/ceapro-inc-reports-2020-third-quarter-and-nine-month

https://investor.electrocore.com/news-releases/news-release-details/electrocore-announces-fourth-quarter-and-full-year-2020

https://ir.ocugen.com/news-releases/news-release-details/ocugen-provides-business-update-and-full-year-2020-financial

https://pdsbiotech.com/investors/news-center/press-releases/press-releases1/113-2021-news/501-iotechnnouncesthatits19accineonsortium20210311n

 

PDS Biotech Reports Financial Results for the Year Ended December 31, 2020 and Provides Business Update

 

FLORHAM PARK, N.J., March 18, 2021 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing novel cancer therapies and infectious disease vaccines based on the Company’s proprietary Versamune^® T-cell activating technology, today announced its financial results for the year ended December 31, 2020 and provided a business update.

Recent Business Highlights:

• Achieved preliminary efficacy benchmark in the Phase 2 combination trial of PDS0101 led by the National Cancer Institute.
• Initiated VERSATILE-002, a Phase 2 trial of lead investigational drug candidate PDS0101, in combination with standard of care KEYTRUDA^® for first-line treatment of patients with metastatic or recurrent HPV-positive head and neck cancer.
• Phase 2 trial of lead investigational drug candidate PDS0101, in combination with standard of care chemoradiotherapy for patients with advanced cervical cancer was initiated by the University of Texas MD Anderson Cancer Center.
• Expanded consortium for development of PDS0203, a novel, Versamune^®-based second-generation COVID-19 vaccine to include Blanver Farmoquímica in addition to Farmacore.
• Received award commitment of up to $60 million from Brazil’s Ministry of Science, Technology and Innovation (MCTI) to fund clinical development and commercialization of PDS0203.
• Strengthened leadership team with the appointment of Seth Van Voorhees as Chief Financial Officer and addition of preeminent oncologist Otis Brawley, M.D. to the board of directors.

“Despite the challenges of 2020, the PDS Biotech team remained focused on the advancement of our Versamune^®-based drug pipeline,” commented Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. “Through that commitment and the strength of our partnerships with leading institutions in the fields of immuno-oncology and infectious disease, we made significant strides in solidifying the safety profile and establishing the efficacy of our Versamune^® platform and products as we continue to progress our portfolio towards commercialization.”

Full Year 2020 Financial Results

For the year ended December 31, 2020, the net loss was approximately $14.8 million, or $0.89 per basic share and diluted share, compared to a net loss of approximately $7.0 million, or $1.44 per basic share and diluted share for the year ended December 31, 2019.

For the year ended December 31, 2020, research and development expenses increased to $7.9 million compared to $6.1 million during the prior year. The increase was primarily the result of increased expenses related to manufacturing and personnel costs for the ongoing clinical studies.

For the year ended December 31, 2020, general and administrative expenses decreased to $7.0 million compared to $11.0 million during 2019. The $4.0 million decrease was due to decreases in personnel costs of $0.4 million, non-cash stock-based compensation of $2.4 million, D&O insurance costs of $0.5 million, legal fees of $0.5 million and professional fees of $0.2 million.

Total operating expenses for 2020 were $14.9 million, a decrease of approximately 29% compared to $21.0 million during the prior year.

The company’s cash balance as of December 31, 2020 was $28.8 million.

Conference Call and Webcast

The conference call is scheduled to begin at 8:00 am ET on Thursday, March 18, 2021. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and mention PDS Biotech. Participants can also access the conference call via webcast on the investor relations page of the Company’s corporate website (link).

The event will be archived in the investor relations section of PDS Biotech’s website for 6 months. In addition, a telephonic replay of the call will be available for 6 months. The replay can be accessed by dialing 877-660-6853 (United States) or 201-612-7415 (International) with confirmation code 13716518.

About PDS Biotechnology

PDS Biotech is a clinical-stage immunotherapy company with a growing pipeline of cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary Versamune^® T-cell activating technology platform. Versamune^® effectively delivers disease-specific antigens for in
vivo uptake and processing, while also activating the critical type 1 interferon immunological pathway, resulting in production of potent disease-specific killer T-cells as well as neutralizing antibodies. PDS Biotech has engineered multiple therapies, based on combinations of Versamune^® and disease-specific antigens, designed to train the immune system to better recognize disease cells and effectively attack and destroy them. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.

Forward Looking Statements

This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,”  “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune^® based products; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune^®
based products and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the acceptance by the market of the Company’s product candidates, if approved; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration or other regulatory authority approval of, or other action with respect to, the Company’s product candidates; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.

Media & Investor Relations Contact:

Deanne Randolph
PDS Biotechnology
Phone: +1 (908) 517-3613
Email: [email protected]

Jacob Goldberger
CG Capital
Phone: +1 (404) 736-3841
Email: [email protected]

 

PDS BIOTECHNOLOGY CORPORATION AND SUBSIDIARIES
Condensed Consolidated
Balance Sheets

 

PDS BIOTECHNOLOGY CORPORATION AND SUBSIDIARIES
Condensed Consolidated
Statements of Operations and Comprehensive Loss

SOURCE: PDS Biotechnology

Ocugen Provides Business Update and Full Year 2020 Financial Results

 

Conference Call and Webcast Today at 8:30 a.m. ET

• COVID-19 vaccine candidate, COVAXIN™, demonstrates efficacy of 81% in Phase 3 interim results
• Emergency Use Authorization pathway with U.S. regulatory authorities in development for COVAXIN™
• European Commission grants orphan medicinal product designation for OCU400 for retinitis pigmentosa and leber congenital amaurosis and Ocugen is on track to submit an Investigational New Drug application for OCU400 in 2021
• On track to initiate four Phase 1/2 clinical trials encompassing Ocugen’s ophthalmology pipeline in 2021 and 2022

MALVERN, Pa., March 18, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen”) (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19, today reported full year 2020 financial results along with a general business update.

“We made strong progress toward our goal of offering a differentiated vaccine to save lives from COVID-19 and in our work toward curing blindness diseases. We are actively working with U.S. regulatory authorities to develop a plan around Emergency Use Authorization in the United States for COVAXIN™ and are preparing to file an Investigational New Drug application to initiate our first two clinical trials for OCU400 in the second half of this year. Proceeds from our recent registered direct offering provide the financial resources to drive our COVAXIN™ development efforts and ophthalmology pipeline forward,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen.

Business Highlights:

• Execution of Co-Development Agreement for COVAXIN™ in the U.S. Market – On February 2, 2021, Ocugen entered into a Co-Development, Supply and Commercialization Agreement with Bharat Biotech International Limited (“Bharat Biotech”) for the development and commercialization of COVAXIN™ in the U.S. market. Upon receipt of Emergency Use Authorization (“EUA”), Bharat Biotech will supply a specified minimum number of doses of COVAXIN™ and then support the technology transfer for manufacturing for the U.S. market. Ocugen will share the profits from the sale of COVAXIN™ in the U.S. market with Bharat Biotech, with Ocugen retaining 45% of the profits.
• Steady Progress to Develop EUA Pathway in the United States for COVAXIN™ Supported by U.S. Leading Experts in Vaccines – Key members of Ocugen’s management team and key advisors possess proven expertise and a track record of success in vaccine development and commercialization. Ocugen has established a vaccine scientific advisory board composed of leading academic and industry experts with extensive experience in the vaccine field. Collectively, the team is working with U.S. regulatory authorities to develop the regulatory pathway to EUA in the U.S. market.
• COVAXIN™ Demonstrates Efficacy of 81% in Phase 3 Interim Results – Interim results from Bharat Biotech’s Phase 3 trial in India showed that COVAXIN™ was well tolerated and demonstrated 81% efficacy in preventing COVID-19 in those without prior infection after the second dose. In addition, COVAXIN™ has been shown to induce immune responses against multiple protein antigens of the virus potentially reducing the possibility of mutant virus escape. This breadth of immune responses has been demonstrated by the ability of antibodies induced by COVAXIN™ to neutralize the U.K. variant of SARS-CoV-2. This broad-antigen containing vaccine has the potential to be effective against new emerging variants.
• First Gene Therapy Candidate OCU400 On Track to Enter the Clinic in 2H21 – Based on Ocugen’s modifier gene therapy platform, Ocugen’s product candidate OCU400 represents a novel approach in that it has the potential to address multiple retinal diseases with one product. Ocugen is planning to file an Investigational New Drug application to initiate two Phase 1/2 clinical trials of OCU400 later this year for the treatment of two disease genotypes.
• European Commission (“EC”) Grants Orphan Medicinal Product Designation for OCU400 for Retinitis Pigmentosa (“RP”) and Leber Congenital Amaurosis (“LCA”) – Designation by the EC further supports the potential broad spectrum application of OCU400 to treat many IRDs. IRDs associated with RP and LCA diseases are caused by mutations in over 175 genes, and it is impractical to develop therapies that are specific to each gene.
• Capital Raised – Ocugen’s cash, cash equivalents, and restricted cash totaled approximately $46.6 million as of February 28, 2021. Subsequent to December 31, 2020, Ocugen generated net proceeds of $4.8 million under an at-the-market offering and net proceeds of $21.2 million under a registered direct offering.

Full Year 2020 Financial Results:

• Ocugen’s cash, cash equivalents, and restricted cash totaled $24.2 million as of December 31, 2020, compared to $7.6 million as of December 31, 2019. The Company had 184.0 million shares of common stock outstanding as of December 31, 2020.
• Research and development expenses for the year ended December 31, 2020 were $6.4 million compared to $8.1 million for the year ended December 31, 2019. General and administrative expenses for the year ended December 31, 2020 were $8.0 million compared to $6.1 million for the year ended December 31, 2019. Ocugen reported a $0.31 net loss per share for the year ended December 31, 2020 compared to a $1.46 net loss per share for the year ended December 31, 2019.

Conference Call and Webcast Details

Ocugen has scheduled a conference call and webcast for 8:30 a.m. eastern time today to discuss the financial results and recent business highlights. Ocugen’s senior management team will host the call, which will be open to all listeners. There will also be a question and answer session following the prepared remarks.

The call can be accessed by dialing (844) 873-7330 (U.S.) or (602) 563-8473 (international) and providing the conference ID 2375087. To access a live audio webcast of the call on the “Investors” section of the Ocugen website, please click here. A replay of the webcast will be archived on Ocugen’s website for approximately 45 days following the call.

About Ocugen, Inc.
Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing gene therapies to cure blindness diseases and developing a vaccine to save lives from COVID-19. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug – “one to many” and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema, and diabetic retinopathy. We are co-developing Bharat Biotech’s COVAXIN™ vaccine candidate for COVID-19 in the U.S. market. For more information, please visit www.ocugen.com.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include information about qualitative assessments of available data, potential benefits, expectations for clinical trials, and anticipated timing of clinical trial readouts and regulatory submissions. This information involves risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preliminary and interim data (including the Phase 3 interim data related to COVAXIN™), including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when data from Bharat Biotech’s clinical trials will be published in scientific journal publications and, if so, when and with what modifications; whether the U.S. Food and Drug Administration (“FDA”) will be satisfied with the design of and results from preclinical and clinical studies of COVAXIN™, which have been conducted by Bharat Biotech in India; whether and when any biologics license and/or EUA applications may be filed in the United States for COVAXIN™; whether and when any such applications may be approved by the FDA; decisions by the FDA impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN™ in the United States, including development of products or therapies by other companies. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (“SEC”), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

This press release contains a preliminary estimate of Ocugen’s cash, cash equivalents, and restricted cash as of February 28, 2021. The preliminary estimate should not be viewed as a substitute for interim financial statements prepared in accordance with U.S. generally accepted accounting principles. The preliminary estimate is based on preliminary unaudited information and management estimates as of February 28, 2021, is not a comprehensive statement of Ocugen’s financial results, and is subject to the completion of Ocugen’s financial closing procedures. As a result, this preliminary estimate may differ from the actual results that will be reflected in Ocugen’s financial statements when they are completed and publicly disclosed. Additional information and disclosures would be required for a more complete understanding of Ocugen’s financial position as of February 28, 2021. Ocugen’s independent registered public accounting firm has not conducted an audit or review of, and does not express an opinion or any other form of assurance with respect to, the preliminary estimate.

Corporate Contact:
Ocugen, Inc.
Sanjay Subramanian
Chief Financial Officer and Head of Corporate Development
[email protected]

Media Contact:
LaVoieHealthScience
Lisa DeScenza
[email protected]
+1 978-395-5970

(tables to follow)

OCUGEN, INC.
CONSOLIDATED BALANCE SHEETS
(UNAUDITED)

OCUGEN, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(UNAUDITED)

SOURCE: Ocugen

Cocrystal Pharma Reports 2020 Financial Results, Provides Business Update Including Antiviral Program Milestones

 

Outlines 2021 product pipeline milestones for COVID-19, influenza A and norovirus antiviral programs

Ends 2020 with cash exceeding $33 million and a clean capital structure

BOTHELL, Wash., March 17, 2021 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP), (“Cocrystal” or the “Company”), a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication machinery of influenza viruses, the SARS-CoV-2 virus, hepatitis C viruses and noroviruses, reports financial results for the year ended December 31, 2020 and provides updates on its antiviral pipeline and business activities.

“We made across-the-board progress last year with our pipeline of broad-spectrum antiviral drugs that feature a high barrier to drug resistance, and we anticipate further advancements in 2021,” said Sam Lee, Ph.D., President of Cocrystal. “In just over a year since initiating our COVID-19 program, we selected a broad-spectrum protease inhibitor, CDI-45205, as a lead drug candidate and are conducting remaining preclinical studies. This antiviral agent presents a novel approach to COVID-19 and other coronaviruses with the potential as both a therapeutic for infected patients and as a prophylactic to protect those who may become exposed. We have also made strides in our norovirus program and expect to complete a proof-of-concept study in norovirus in the first half of this year.”

“Our influenza programs are also advancing well,” stated Gary Wilcox, Ph.D., Chairman and Chief Executive Officer of Cocrystal. “We successfully completed our obligations under the exclusive worldwide licensing and collaboration agreement with Merck for the discovery and development of influenza A/B drug candidates, with Merck now assuming sole responsibility for further program development. With our in-house influenza A program, we are conducting the remaining preclinical studies with CC-42344 and plan to initiate a Phase 1 study in the third quarter. Influenza continues to be a major global health concern and our antiviral agents are being developed to be effective against both seasonal and pandemic influenza.

“We are employing our proprietary technology platform to discover and develop antiviral drug candidates that address major global medical concerns, such as influenza and coronaviruses, that present significant market opportunities,” he added. “We are well positioned to execute on our goals with the guidance and support of our outstanding leadership team and our scientific advisory board, which includes two Nobel laureates. Following successful financings completed last year, we ended 2020 with more than $33 million of cash and have sufficient capital to fund our current operations and development programs beyond 2022.”

Antiviral Development Pipeline Milestones and Updates

 COVID-19 Programs

	?	Advancing preclinical studies with CDI-45205. This compound, obtained under an agreement with Kansas State University Research Foundation (KSURF), demonstrated in vitro and in vivo activity in animal models against the viral pathogens MERS and SARS and has significant potential for delivery either by injection or inhalation.
	?	Applying the Company’s proprietary structure-based platform technology to discover novel COVID-19 inhibitors with the potential for oral administration.

Influenza A Program

	?	Completing IND-enabling activities with CC-42344 and planning to initiate a Phase 1 study during the third quarter of 2021. CC-42344 has demonstrated excellent preclinical antiviral activity against influenza A strains, including avian pandemic strains and oseltamivir-resistant and baloxavir-resistant strains, while also showing favorable pharmacokinetic and safety profiles.
		Influenza remains a major global concern with cases approximating 1 billion annually. The World Health Organization estimates that worldwide, annual influenza epidemics result in approximately 3-5 million cases of severe illness and about 250,000 to 500,000 deaths. Approved influenza therapies have major limitations due to drug-resistant issues and emerging virus mutations. Cocrystal is designing influenza drug candidates to be active against drug-resistant strains, effective against future mutations, and available for delivery through multiple routes of administration, including oral, inhalation and injection.

Hepatitis C Program

?

Seeking a partner to advance the development of CC-31244. This compound showed positive safety and preliminary efficacy data from a triple regimen Phase 2a study in combination with Epclusa (sofosbuvir/velpatasvir) for the ultra-short treatment of individuals infected with the hepatitis C virus.

Norovirus Therapy Program

?

Expect to complete a proof-of-concept animal study in the first half of 2021 with a potential first-in-class non-nucleoside inhibitor with potent and broad-spectrum Noro polymerase inhibitors.

2020 and Recent Highlights

Licensing and Collaboration Agreements

	?	Completed all research obligations under the Merck exclusive worldwide license and collaboration agreement for influenza A/B antiviral compounds. As of mid-January 2021, Merck has assumed all responsibility for further program development.
	?	Entered into two exclusive, royalty-bearing license agreements with KSURF to develop and commercialize therapeutic, diagnostic and prophylactic products against coronaviruses, caliciviruses and picornaviruses based on antivirals discovered by KSURF.
	?	Extended a drug discovery collaboration with HitGen and InterX, combining three independent platforms to discover and optimize molecules that could lead to novel antiviral drug candidates.

Scientific Publications and Presentations

	?	Announced the publication by collaborators of positive preclinical animal data demonstrating potent inhibition of coronavirus antiviral compounds in the prestigious medical journal Science Translational Medicine.
	?	Presented an overview of the Company’s drug discovery platform technology, including its unique ability to develop broad-spectrum antiviral therapeutics and its advantages compared with the traditional drug discovery and development process, presented at the “reimagine Health Research Symposium” in January 2021.

Corporate Developments

	?	Appointed Nobel laureate Roger D. Kornberg, Ph.D. to the Cocrystal Board of Directors, adding to his positions as Chief Scientist and Chairman of the Scientific Advisory Board.
	?	Received $2.0 million in Merck payments and $35.8 million in net proceeds from common-stock financings.
	?	Negotiated and executed approved settlement of the class-action lawsuit, the derivative lawsuit and two related derivative actions with agreement to pay $450,000 for its share of the total class action settlement and make certain corporate governance changes.

2020 Financial Results

Revenues for the year ended December 31, 2020 were $2.0 million, which consisted entirely of research and development (R&D) services performed by Cocrystal and reimbursed program expenses paid by Merck for the influenza A/B program. This compares with revenues of $6.6 million for the year ended December 31, 2019, which included $4.4 million in exchange for conveyance of intellectual property rights at the signing of the Merck collaboration agreement, $1.8 million for R&D services and $358,000 for program expense reimbursements.

In mid-January 2021 Merck assumed all activities and expenses associated with the continued development of the influenza A/B compounds. Cocrystal does not expect to report revenues or offsetting R&D expenses related to this agreement in 2021. Cocrystal is eligible to receive milestone payments related to designated development, regulatory and sales milestones with the potential to earn up to $156 million, as well as royalties on product sales.

R&D expenses for 2020 were $6.3 million compared with $4.0 million for 2019, with the increase primarily due to costs related to advancing the coronavirus, influenza and norovirus programs. General and administrative expenses for 2020 were $5.3 million compared with $4.9 million for the prior year, with the increase primarily due to professional fees incurred as a result of the now settled class action matter mentioned above in Corporate Developments. The operating loss for 2020 was $9.6 million compared with an operating loss of $48.4 million in 2019, which included a $46.1 million non-cash goodwill impairment charge on an intangible asset in 2019.

Other expense for 2020 was $62,000, which was primarily due to a $54,000 loss on the fair value of derivative liabilities. This compares with other income for 2019 of $237,000, that was primarily due to a $256,000 gain on derivative liabilities.

The net loss for 2020 was $9.6 million, or $0.17 per share, compared with a net loss for 2019 of $48.2 million, or $1.51 per share, which included the $46.1 million impairment.

The Company reported $33.1 million in cash and cash equivalents as of December 31, 2020, compared with $7.4 million as of December 31, 2019. The Company reported working capital of $32.6 million as of December 31, 2020.

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

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the expected further advancements in our programs, including the planned initiation of the influenza A Phase 1 study during the third quarter of 2021 and our plans regarding the expected completion of a norovirus proof-of-concept animal study in the first half of 2021; our expectations and estimates regarding the future applications and effectiveness of, and the market opportunities for, our product candidates; our expectations regarding future operating results; the expected results of Cocrystal’s collaboration with Merck, including potential receipt of future milestone payments of up to $156,000,000 and royalties; and future 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 the impact of the COVID-19 pandemic on the national and global economy, on our collaboration partners and on our Company, including supply chain disruptions and our continued ability to proceed with our programs, our reliance on Merck for further development in the influenza A/B program under the license and collaboration agreement, , the results of future preclinical and clinical studies, 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
[email protected]

Financial Tables to follow

COCRYSTAL PHARMA, INC.

CONSOLIDATED BALANCE SHEETS
(in thousands)

		December 31, 2020				December 31, 2019
Assets
Current assets:
Cash		$	33,010			$	7,418
Restricted cash			50				50
Accounts receivable			556				644
Prepaid expenses and other current assets			399				169
Total current assets			34,015				8,281
Property and equipment, net			591				431
Deposits			46				50
Operating lease right-of-use assets, net (including $39 to related party)			498				677
Goodwill			19,092				19,092
Total assets		$	54,242			$	28,531
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable and accrued expenses		$	1,080			$	1,999
Current maturities of finance lease liabilities			39				103
Current maturities of operating lease liabilities (including $39 to related party)			178				177
Derivative liabilities			61				7
Total current liabilities			1,358				2,286
Long-term liabilities:
Finance lease liabilities			34				14
Operating lease liabilities			345				523
Total long-term liabilities			379				537
Total liabilities			1,737				2,823
Commitments and contingencies
Stockholders’ equity:
Common stock, $0.001 par value; 100,000 shares authorized as of December 31, 2020 and December 31, 2019; 70,439 and 35,150 shares issued and outstanding as of December 31, 2020 and December 31, 2019, respectively			71				36
Additional paid-in capital			297,342				260,932
Accumulated deficit			(244,908	)			(235,260	)
Total stockholders’ equity			52,505				25,708
Total liabilities and stockholders’ equity		$	54,242			$	28,531

COCRYSTAL PHARMA, INC.

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

		December 31,
		2020				2019
Revenues:
Collaboration revenue		$	2,014			$	6,564
Operating expenses:
Research and development			6,307				4,004
General and administrative			5,293				4,863
Impairments			–				46,103
Total operating expenses			11,600				54,970
Loss from operations			(9,586	)			(48,406	)
Other (expense) income:
Interest expense, net			(8	)			(19	)
Change in fair value of derivative liabilities			(54	)			256
Total other income (expense), net			(62	)			237
Loss before income taxes			(9,648	)			(48,169	)
Income tax			–				–
Net loss		$	(9,648	)		$	(48,169	)
Net loss per common share:
Loss per share, basic and diluted		$	(0.17	)		$	(1.51	)
Weighted average number of common shares outstanding, basic and diluted			55,217				31,859

Source: Cocrystal Pharma, Inc.

Helius Medical Technologies, Inc. Appoints Sherrie Perkins to its Board of Directors

 

NEWTOWN, Pa., March 17, 2021 (GLOBE NEWSWIRE) — Helius Medical Technologies, Inc. (Nasdaq:HSDT) (TSX:HSM) (“Helius” or the “Company”), a neurotech company focused on neurological wellness, today announced the appointment of Sherrie Perkins, effective March 15, 2021.

“Ms. Perkins’ career history includes over 20 years of experience in the healthcare industry, including significant experience advising neuromodulation companies on their commercial and marketing activities,” said Blane Walter, Chairman of Helius’ Board of Directors. “We are pleased to expand our Board of Directors with the appointment of Ms. Perkins and look forward to leveraging her expertise and strategic insight as Helius prepares its next phase of market development activities.”

“I am delighted to join the Board of Directors of Helius Medical Technologies and appreciate the opportunity to help the company advance its unique therapy in ways that provide meaningful benefit to patients, physicians, and payers,” said Ms. Perkins.

Ms. Perkins currently serves as a member of the Venture Mentoring Service at the University of Texas MD Anderson Cancer Center, providing guidance and perspective on commercialization-related topics that are important and relevant to the progression of various ventures.

From 2017 to 2019, Ms. Perkins served as a consultant to LivaNova, PLC (NASDAQ: LIVN), a global medical technology company that designs, develops, manufactures and sells innovative therapeutic solutions in the fields of neuromodulation and cardiovascular disease. She previously spent 17 years at LivaNova and its affiliates in several roles, including serving as Vice President in the sleep apnea, new ventures space within the company from 2015 to 2017, and as Vice President of Marketing and New Business Development of Cyberonics, Inc. from 2011 to 2015.

Ms. Perkins received a B.S. in Medical Technology from Mississippi State University and an M.A. in Management from Central Michigan University.

About
Helius Medical Technologies, Inc.

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 (PoNS
^TM). For more information, visit www.heliusmedical.com.

About
the PoNS™ Device and PoNS Treatment™

The Portable Neuromodulation Stimulator (PoNS™) is authorized for sale in Canada as a class II, non-implantable, medical device intended as a short term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from multiple sclerosis (MS), and chronic balance deficit due to mild-to-moderate traumatic brain injury (mmTBI) and is to be used in conjunction with physical therapy. The PoNS™ is an investigational medical device in the United States, the European Union (“EU”), and Australia (“AUS”). The device is currently under review for de novo classification and clearance by the FDA. It is also under premarket review by the AUS Therapeutic Goods Administration. PoNS™ is currently not commercially available in the United States, the European Union or Australia.

Investor
Relations Contact:

Westwicke Partners on behalf of Helius Medical Technologies, Inc.
Jack Powell, Vice President
[email protected]

Cautionary
Disclaimer Statement: 

Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as “believe,” “continue,” “look forward,” “will,” “committed to,” “goal,” “expect,” “remain,” “hope” and similar expressions. Such forward-looking statements include, among others, statements regarding the Company’s future growth and operational progress, the next phase of the Company’s market development activities, clinical and regulatory development plans for the PoNS device, and potential regulatory clearance of the PoNS device, including expected timing for the FDA to resume its review of our request for de novo classification and clearance and expected timing for receipt of the FDA’s decision on such request.

These statements involve substantial known and unknown risks and uncertainties. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include uncertainties associated with the clinical development process and FDA regulatory submission and approval process, including that the Company’s request for de novo classification and clearance may be declined by the FDA, that the FDA is not required to and may not respond to the Company’s request in the timeframe indicated by its de novo review goals or in the time the Company expects, whether the Company’s response will be satisfactory to the FDA, whether the FDA will require additional information, whether the Company will be able to provide it in a timely manner and whether such additional information will be satisfactory to the FDA, uncertainties regarding the Company’s capital requirements to achieve its business objectives, the impact of the COVID-19 pandemic, uncertainties associated with future clinical trials and other development activities, and other risks detailed from time to time in the filings made by the Company with securities regulators, including the risks and uncertainties described in the “Risk Factors” sections of the Company’s Annual Report on Form 10-K for the year ended December 31, 2020 and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at www.sec.gov or www.sedar.com.The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law.

The Toronto Stock Exchange has not reviewed and does not accept responsibility for the adequacy or accuracy of the content of this news release.

Aeterna Zentaris Announces Exclusive License Agreement and Research Contract with Julius-Maximilians-University Wuerzburg for Development of a Potential Oral Prophylactic Bacterial Vaccine Against COVID-19

 

– Company secures next step to continue to build-out pipeline of assets

– Company exercised its option to enter into an exclusive license of intellectual property for the development of a proprietary and orally active bacterial vaccine platform technology currently undergoing pre-clinical studies for the prevention of coronavirus diseases, including COVID-19

CHARLESTON, S.C., March 15, 2021 (GLOBE NEWSWIRE) — Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS), through its wholly-owned subsidiary Aeterna Zentaris GmbH, (“Aeterna” or the “Company”), a specialty biopharmaceutical company commercializing and developing therapeutics and diagnostic tests, today announced that the Company exercised its option announced on February 2, 2021 and has entered into an exclusive worldwide sub-licensable patent and know-how license agreement for a potential COVID-19 vaccine currently in preclinical development that was invented at the Julius-Maximilians-University Wuerzburg (the “University”), one of Germany’s leading research and teaching universities. Additionally, the Company has entered into a research agreement with the University to conduct supplementary research activities and preclinical development studies on the potential vaccine.

The vaccine technology developed at the University uses the approved typhoid fever vaccine Salmonella Typhi Ty21a as a carrier strain and has the potential to be an orally active, live-attenuated bacterial vaccine to prevent SARS-CoV-2 infection leading to COVID-19.

“Over the last months, we learned that the original SARS-CoV-2 strain mutates rapidly, and these mutant strains continue to spread throughout the population. It has been reported that the currently available vaccines for COVID-19 are still effective against the known mutant strains. However, we believe there is the potential to develop an improved vaccine which relies on several SARS-CoV-2 antigens in parallel with the goal of improving the immune response against mutated viruses. Additionally, our vaccine has the potential to become a cost-effective oral alternative with less demanding storage and logistics requirements”, commented Dr. Klaus Paulini , Chief Executive Officer of Aeterna Zentaris. “We look forward to advancing our scientific collaboration with Prof. Rudel and his group at the University. Aeterna plans to select from a set of vaccine candidates to perform further in vitro and in vivo characterization before selecting the most active and stable bacterial strain for further preclinical and potentially clinical development. The goal is to develop an oral dosage form of COVID-19 vaccine which is also active against mutated viruses that can be stored in a common fridge and manufactured with relatively low costs of goods.

Prof. Thomas Rudel of the University added, “We are looking forward to our collaboration with Aeterna and a new phase of accelerated preclinical and potential clinical development. Our oral vaccine candidates based on the Salmonella Typhi Ty21a vaccine platform technology open the possibility to integrate more than one SARS-CoV-2 related antigen into the expression system of the modified vaccine bacteria and may improve the immune response of an individual against mutated viruses.”

About the Potential COVID-19 Vaccine

The approved Salmonella Typhi Ty21a bacterial strain is the basis of the new vaccine approach against corona virus infections. The typhoid fever vaccine Ty21a is effective, safe, easy to handle, and the capsule formulation can be stored at fridge temperature of 2°C to 8°C. The most common vaccine capsule Vivotif ^® has been used worldwide in more than 150 million administered doses.

The carrier strain has been modified by plasmid insertion with two expression cassettes together with a special E. coli -based secretion system to secrete two or more coronavirus antigens fused to an immunological adjuvant peptide. Additionally, a balanced lethal system based on an essential tRNA synthetase has been integrated to stabilize the plasmid in the absence of antibiotic resistance genes. The specific bacterial vector strain is expected to enable oral application and release of the proteins into the gut system which may consequently stimulate mucosal and systemic immunity.

Transaction Terms and Conditions

On March 14, 2021, the Company exercised the Option and entered into the License Agreement. Pursuant to the terms of the License Agreement, the Company has been granted an exclusive, world-wide, license to certain patents and know-how owned by the University to research and develop, manufacture, and sell a potential COVID-19 vaccine using the University’s bacterial vaccine platform technology (the “Licensed Rights”). The Company will pay an up-front payment under the License Agreement of €140,000 as well as make certain milestones payments to be paid upon the achievement of certain development, and regulatory and sales milestones as well as a percentage of any sub-licensing revenue received by the Company and royalty payments on net sales of the licensed vaccine products (including for by the Company or its sub-licensees). The License Agreement will expire upon the latter of (i) the existence of a valid patent claim of a Licensed Right or (ii) 10 years after the first commercial sale of a product that was developed, manufactured, marketed, and sold using a least one Licensed Right. The License Agreement may be terminated by the Company by providing six (6) months’ notice to the University.

Pursuant to the License Agreement, the University has also granted the Company an exclusive option for the exclusive use of the Licensed Rights in an undisclosed field. The Company has six (6) months from the date of the License Agreement to exercise that is option.

Additionally, the Company has entered into the Research Agreement under which the Company has engaged the University on a fee-for-service basis to conduct supplementary research activities and preclinical development studies on the potential vaccine.

About COVID-19

COVID-19 is the disease caused by a new coronavirus called SARS-CoV-2, and was first reported in December 2019 in Wuhan, Hubei province, China. Most people infected with the COVID-19 virus will experience mild to moderate respiratory illness and recover without requiring special treatment. Older people, and those with underlying medical problems like cardiovascular disease, diabetes, chronic respiratory disease, and cancer are more likely to develop serious illness.

Globally over 116 million confirmed cases and over 2.5 million deaths are reported since the start of the pandemic. Currently, there are no definite approved therapies endorsed by the World Health Organization for COVID-19, focusing on supportive care and preventive immunization.

About Aeterna Zentaris Inc.

Aeterna Zentaris Inc. is a specialty biopharmaceutical company commercializing and developing therapeutics and diagnostic tests. The Company’s lead product, macimorelin, is the first and only U.S. FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Macimorelin is currently marketed in the United States under the tradename Macrilen™ through a license agreement with Novo Nordisk where Aeterna receives royalties on net sales. According to a commercialization and supply agreement, Megapharm Ltd. will seek regulatory approval and then commercialize macimorelin in Israel and the Palestinian Authority. Additionally, upon receipt of pricing and reimbursement approvals, Aeterna expects that macimorelin will be marketed in Europe and the United Kingdom through a recently established license agreement with Consilient Health Ltd. and Aeterna will receive royalties on net sales and other potential payments.

Aeterna is also leveraging the clinical success and compelling safety profile of macimorelin to develop it for the diagnosis of childhood-onset growth hormone deficiency (CGHD), an area of significant unmet need.

Aeterna is actively pursuing business development opportunities for the commercialization of macimorelin in Asia and the rest of the world, in addition to other non-strategic assets to monetize their value. For more information, please visit www.zentaris.com and connect with the Company on Twitter , LinkedIn and Facebook .

Forward-Looking Statements

This press release contains forward-looking statements (as defined by applicable securities legislation) made pursuant to the safe-harbor provision of the U.S. Securities Litigation Reform Act of 1995, which reflect our current expectations regarding future events. Forward-looking statements in this press release include those relating to the potential of the University’s coronavirus vaccine platform technology (and any vaccine candidates using that technology) to be effective as a vaccine against COVID-19 (SARS-CoV-2) or any other coronavirus disease (or to offer an alternative to other approved vaccines against COVID-19, the ability to obtain approval to commence any clinical trial or the timeline to develop any potential vaccine and the characteristics of any potential vaccine (including cost, storage temperatures and oral availability and Aeterna’s expectation that, upon receipt of pricing and reimbursement approvals, macimorelin will be marketed in Europe and the United Kingdom and the initiation of Study P02, which is expected to be initiated in Q2 of 2021. Forward-looking statements involve known and unknown risks and uncertainties, including those discussed in this press release and in our Annual Report on Form 20-F, under the caption “Key Information – Risk Factors” filed with the relevant Canadian securities regulatory authorities in lieu of an annual information form and with the U.S. Securities and Exchange Commission. Known and unknown risks and uncertainties could cause our actual results to differ materially from those in forward-looking statements. Such risks and uncertainties include, among others, that the University’s coronavirus vaccine platform technology (and any vaccine candidates using that technology) has never been tested in humans and so further pre-clinical or clinical studies of that technology and any vaccine developed using that technology may not be effective as a vaccine against COVID-19 (SARS-CoV-2) or any other coronavirus disease, that such technology or vaccines may not receive the necessary approvals to be studied in human clinical trials, that the timeline to develop a vaccine may be longer than expected, that such technology or vaccines may not be capable of being used orally, may not have the same characteristics (including storage temperatures) as vaccines previously approved using the Salmonella Typhi Ty21a carrier strain, any such vaccine developed using the University’s technology may not lower the evolution of resistant viral mutants or may not be competitive with vaccines developed by third parties against COVID-19, our ability to raise capital and obtain financing to continue our currently planned operations, our ability to continue to list our Common Shares on the NASDAQ, our now heavy dependence on the success of Macrilen™ (macimorelin) and related out-licensing arrangements and the continued availability of funds and resources to successfully commercialize the product, including our heavy reliance on the success of the License Agreement with Novo Nordisk, the global instability due to the global pandemic of COVID-19, and its unknown potential effect on our planned operations, including studies, our ability to enter into out-licensing, development, manufacturing, marketing and distribution agreements with other pharmaceutical companies and keep such agreements in effect, our reliance on third parties for the manufacturing and commercialization of Macrilen™ (macimorelin), potential disputes with third parties, leading to delays in or termination of the manufacturing, development, out-licensing or commercialization of our product candidates, or resulting in significant litigation or arbitration, uncertainties related to the regulatory process, unforeseen global instability, including the instability due to the global pandemic of the novel coronavirus, our ability to efficiently commercialize or out-license Macrilen™ (macimorelin), our reliance on the success of the pediatric clinical trial in the European Union (“E.U.”) and U.S. for Macrilen™ (macimorelin), the degree of market acceptance of Macrilen™ (macimorelin), our ability to obtain necessary approvals from the relevant regulatory authorities to enable us to use the desired brand names for our product, our ability to successfully negotiate pricing and reimbursement in key markets in the E.U. for Macrilen™ (macimorelin), the outcome of our pre-clinical and clinical development efforts of in-licensed products, any evaluation of potential strategic alternatives to maximize potential future growth and shareholder value may not result in any such alternative being pursued, and even if pursued, may not result in the anticipated benefits, our ability to take advantage of business opportunities in the pharmaceutical industry, our ability to protect our intellectual property, and the potential of liability arising from shareholder lawsuits and general changes in economic conditions. Investors should consult our quarterly and annual filings with the Canadian and U.S. securities commissions for additional information on risks and uncertainties. Given these uncertainties and risk factors, readers are cautioned not to place undue reliance on these forward-looking statements. We disclaim any obligation to update any such factors or to publicly announce any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, unless required to do so by a governmental authority or applicable law.

Investor Contact:

Jenene Thomas
JTC Team
T (US): +1 (833) 475-8247
E: [email protected]

Onconova Therapeutics Reports Full Year 2020 Financial Results, Provides Business Update

 

Conference call begins at 4:30 p.m. Eastern time today

NEWTOWN, Pa., March 11, 2021 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX) (“Onconova”), a clinical-stage biopharmaceutical company focused on discovering and developing novel therapies for patients with cancer, announces financial results for the twelve months ended December 31, 2020 and provides a business update.

Highlights of the fourth quarter of 2020 and recent weeks include:

• ON 123300, Onconova’s proprietary multi-kinase inhibitor, received clearance from the U.S. Food and Drug Administration (FDA) to begin Phase 1 studies
• ON 123300 also received Institutional Review Board (IRB) approval at one U.S. clinical trial site
• The Phase 1 solid tumor study with ON 123300 in China is ongoing and continues to enroll patients
• Raised net proceeds of $35.2 million from two equity offerings; cash and cash equivalents as of February 28, 2021 were approximately $49.5 million
• An independent investigator-initiated study with oral rigosertib in combination with a PD-1 inhibitor in advanced KRAS mutated non-small cell lung cancer is ongoing
• A Special Meeting of Stockholders to consider changes to the capital structure of the Company will reconvene on April 1, 2021

Management Commentary

“The fourth quarter and recent weeks have been active and productive at Onconova as we continue to advance our lead product ON 123300 into the clinic,” said Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova. “We submitted an Investigational New Drug application to the FDA for a Phase 1 study in advanced cancers including HR+/HER 2- metastatic breast cancer patients resistant to approved second-generation CDK 4/6 inhibitors. In December 2020, we received clearance from the FDA to begin the study, and have since received IRB approval at our first site. We expect the first patient to be enrolled in the second quarter of this year. Two further sites are in the study set-up process.

“This Phase 1 study will assess the safety, tolerability and pharmacokinetics of ON 123300 administered orally at increasing doses starting at 40 mg daily continuously.

“Our partner in China, HanX Pharmaceuticals, continues enrolling a similar patient population in a Phase 1 dose-escalation study with ON 123300 at two sites. The initial dose cohort has been completed and the second dose cohort is enrolling. We are pleased that ON 123300 appears to be well tolerated so far as no dose-limiting toxicities have been seen to date. The HanX study is dosing patients on a 21-day cycle. Collectively, the U.S. and China Phase 1 studies are expected to provide data regarding the safety profile of ON 123300 and potentially provide preliminary efficacy signals in patients with advanced cancer.”

Commenting on ongoing investigator-sponsored studies with oral rigosertib, the company’s RAS pathway inhibitor, Dr. Fruchtman added, “We are currently supporting investigator-initiated studies that are exploring the use of oral rigosertib for cancers driven by mutation of the RAS gene including a Phase 1 study in combination with a PD-1 inhibitor for patients with progressive K-RAS mutated non-small cell lung cancer. This study is open and continues to enroll patients, with the objectives to identify the recommended Phase 2 dose and to characterize the safety profile of the combination treatment. Results are expected in 2021.

“In addition, an investigator-initiated Phase 1b/2 study with oral rigosertib monotherapy in advanced squamous cell carcinoma associated with recessive dystrophic epidermolysis bullosa is open. A preclinical study is also evaluating oral rigosertib in clear cell renal carcinoma. We anticipate additional investigator-initiated studies in RAS-driven cancers in combination with PD-1 inhibitors, including in metastatic melanoma. Other than the cost of supplying oral rigosertib to the investigators, Onconova does not expect to incur significant expense for these studies,” Dr. Fruchtman stated.

View the full press release at Onconova’s website: investor.onconova.com/

Company Contact:
Avi Oler
Onconova Therapeutics, Inc.
267-759-3680

[email protected]
https://www.onconova.com/contact

Investor Contact:
LHA Investor Relations
Kim Sutton Golodetz
212-838-3777

[email protected]

SOURCE: Onconova Therapeutics, Inc.

Friday, March 12, 2021

Lineage Cell Therapeutics (LCTX)
Q4 2020 EPS: Catalysts Rich 2021 Ahead

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical programs are in markets with billion dollar opportunities and include three allogeneic (“off-the-shelf”) product candidates: (i) OpRegen®, a retinal pigment epithelium transplant therapy in Phase 1/2a development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; and (iii) VAC, an allogeneic dendritic cell therapy platform for immuno-oncology and infectious disease, currently in clinical development for the treatment of non-small cell lung cancer. For more information, please visit www.lineagecell.com or follow the Company on Twitter @LineageCell.

Ahu Demir, Ph. D., Biotechnology Research Analyst, Noble Capital Markets, Inc.

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

Full Year 2020 financials. Lineage reported net loss of $20.6 million or ($0.14) EPS. The company has $41.6 million cash, cash equivalents, and marketable securities as of December 31, 2020. Total revenues were $1.8 million and total operating loss was $27.9 million – $12.3 million in R&D expenses and $15.6 million in SG&A expenses. We estimated $1.9 million in revenue, $12.3 million in R&D expenses, and $16.9 million in SG&A expenses and implemented the changes in our model. The company also had a good start to the year with the sale of additional marketable securities resulting in net proceedings above $21 million. With this, the company now has $57 million in cash only (161 million shares outstanding) bringing cash runway to 2023.


Value-generating inflection points.  We expect multiple inflection points to generate value for the shares in 2021 selected catalysts include a) Interim data analysis from Cohort 4 OpRegen program 3-months data in Q1 and 6-months data at the annual Association for Research in Vision and Ophthalmology (ARVO) meeting on May 1-7, b) conducting regenerative medicine advanced therapy (RMAT) meeting 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.