Category: Health

Release – Axcella Therapeutics to Participate in the H.C. Wainwright Global Investment Conference

Posted on by aweinsoff@channelchek.com

Research, News, and Market Data on AXLA

September 12, 2022 at 8:00 AM EDT

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sep. 12, 2022– Axcella Therapeutics (Nasdaq: AXLA), a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using multi-targeted endogenous metabolic modulator (EMM) compositions, today announced that management will participate in a fireside chat and one-on-one meetings at the H.C. Wainwright Global Investment Conference taking place September 12 – 14, 2022 both virtually and in-person in New York City.

Details for the fireside chat are as follows:

Date:Tuesday, September 13, 2022
Time:10:30 am Eastern Time
Webcast:https://journey.ct.events/view/6604fa89-69be-476a-b518-b615e1ac7fb6

The conference call webcast will be accessible in the Investors & News section on the company’s website at www.axcellatx.com. An archive of the webcast replay will be available on the Company’s website for up to 90 days.

To request a one-on-one in-person or virtual meeting, please register here.

Internet Posting of Information

Axcella uses the “Investors and News” section of its website, www.axcellatx.com, as a means of disclosing material nonpublic information, to communicate with investors and the public, and for complying with its disclosure obligations under Regulation FD. Such disclosures include, but may not be limited to, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, and public conference calls and webcasts. The information that we post on our website could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

About Axcella Therapeutics (Nasdaq: AXLA)

Axcella is a clinical-stage biotechnology company pioneering a new approach to treat complex diseases using compositions of endogenous metabolic modulators (EMMs). The company’s product candidates are comprised of EMMs and derivatives that are engineered in distinct combinations and ratios to reset multiple biological pathways, improve cellular energetics, and restore homeostasis. Axcella’s pipeline includes lead therapeutic candidates in Phase 2 development for the treatment of Long COVID and NASH. The company’s unique model allows for the evaluation of its EMM compositions through non-IND clinical studies or IND clinical trials. For more information, please visit www.axcellatx.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220912005400/en/

Ashley Robinson
arr@lifesciadvisors.com
(617) 430-7577

Source: Axcella Therapeutics

Release – Ayala Pharmaceuticals Presents Positive Interim Data from RINGSIDE Pivotal Phase 2-3 Trial of AL102 in Desmoid Tumors at ESMO Congress 2022

Posted on by aweinsoff@channelchek.com

Research, News, and Market Data on AYLA

September 12, 2022

– Updated interim results from Part A of RINGSIDE show first confirmed partial response (PR) achieved at week 16 and 3 additional unconfirmed PRs over the follow-up period –

– Consistent early tumor shrinkage with measures deepening over time –

– AL102 was well tolerated at all doses evaluated –

– Part B and Open-Label Extension being initiated with the selected dose of 1.2mg once daily –

– Company to host Key Opinion Leader event on September 28 to discuss results –

REHOVOT, Israel and WILMINGTON, Del., Sept. 12, 2022 (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 tumors and aggressive cancers today announces updated, positive interim results from Part A of the ongoing RINGSIDE Pivotal Phase 2/3 clinical trial evaluating investigational new drug AL102 in desmoid tumors. AL102 is a potent, selective, oral gamma-secretase inhibitor.

The data are being featured in an oral presentation today at the European Society for Medical Oncology (ESMO) Congress 2022. The presentation entitled, “Initial Results of Phase 2/3 Trial of AL102 for Treatment of Desmoid Tumors” is being delivered by Prof. Robin Jones, M.D., Head of the Sarcoma Unit at The Royal Marsden, London, UK.

“The results presented at ESMO from the RINGSIDE study are very encouraging. AL102 demonstrated an early and meaningful effect on tumors within a 16-week period and was well tolerated, which could allow for long term treatment of patients. AL102 has the potential to significantly improve the lives of patients suffering from desmoid tumors who currently have no approved therapy,” said Prof. Robin Jones.

Jeanne Whiting, Executive Director & Co-Founder of the Desmoid Tumor Research Foundation stated, “Desmoid tumors are rare, connective tissue tumors that can have aggressive infiltrative growth and high risk of local recurrence. Patients and physicians struggle with the fact that there are no FDA-approved therapies. We are very encouraged by the results presented by Ayala and the opportunity that AL102 holds in improving outcomes for patients with this rare condition.”

“We are excited to share strong interim results from the AL102 RINGSIDE study at this year’s ESMO Congress which continue to demonstrate early and meaningful anti-tumor activity as monotherapy in patients with desmoid tumors,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “Efficacy was demonstrated across all cohorts of Part A of the study, with early responses that deepened over time. The results also showed that AL102 was well-tolerated across all doses. We are advancing to Part B of RINGSIDE with a selected dose of 1.2mg once daily, as well as enrolling patients in the open label extension study. The results presented today give us increased confidence in the potential for AL102 to improve the lives of patients with desmoid tumors.”

Results as of the Cut-Off Date of July 14, 2022
Part A Interim Efficacy Results:

  • Patient enrollment in Part A of RINGSIDE was completed in February 2022. Patients were dosed in AL102 monotherapy cohorts of 1.2mg (once daily), 2mg (2 days on, 5 days off), or 4mg (2 days on, 5 days off).
  • The activity of AL102 is being evaluated by change in tumor volume (central MRI readings) and response (per RECIST 1.1) determined by blinded independent central review.   At data cut, 28 patients were evaluable for tumor volume and 29 were evaluable for RECIST with a scan at base line and at least one additional scan at week 16.
  • 12 subjects had follow up MRI scans at week 28 and one patient had a scan at week 40.
  • One patient had a partial response (PR) per RECIST at week 16, confirmed at week 28.
  • Three additional unconfirmed PRs were observed, two at week 28 and one at week 40.
  • Continuous tumor volume reduction was observed over time in all patients that underwent 2 or more MRI scans.

Part A Interim Efficacy Results of Selected Dose of 1.2mg daily:

  • At week 16 there were 9 evaluable patients for RECIST in the selected dose of 1.2mg once daily with one PR observed, confirmed at week 28. The remaining 8 patients had stable disease, of which 7 patients had a tumor reduction.
  • At week 28 there were three patients evaluable for RECIST in the selected dose of 1.2mg daily with one confirmed and one unconfirmed PR and one stable disease with all patients showing tumor reduction and deepening of tumor shrinkage since previous scan.
  • At the selected dose of 1.2mg once daily, at week 16 there were 9 evaluable patients for volume change with 7 patients experiencing tumor volume reduction. At week 28 there were three evaluable patients for volume change in the selected dose of 1.2mg once daily with all three patients experiencing continuous tumor shrinkage.

Part A Safety:

  • AL102 was generally well tolerated at all doses
  • Most adverse events were grade 1 or 2 and included mainly diarrhea
  • No grade 4 or 5 events were observed and low rates of grade 3 events.
  • At the selected dose (1.2 mg once daily) 3 out of the 14 patients (21.4%) had grade 3 events.
  • Ovarian dysfunction was observed in about 22% of women with childbearing potential (N=23)

About the RINGSIDE study
The RINGSIDE pivotal Phase 2/3 study is a randomized global multi-center trial. Part A of the study is evaluating the efficacy, safety, tolerability, and tumor volume by MRI after 16 weeks of AL102 in patients with desmoid tumors. It enrolled 42 patients and is evaluating 3 doses of AL102. Patients who participated in Part A are eligible to enroll into an open-label extension study at the Part B selected dose of 1.2 mg daily, and long-term efficacy and safety will be monitored.

Part B of the study is a double-blind, placebo-controlled segment enrolling up to 156 patients with progressive disease, comparing AL102 at 1.2 mg once daily to placebo. The primary endpoint for Part B will be progression-free survival (PFS) with secondary endpoints including objective response rate (ORR), duration of response (DOR), tumor volume reduction, and patient-reported Quality of Life (QOL) measures. For more information on the RINGSIDE Phase 2/3 study with AL102 for the treatment of desmoid tumors, please visit ClinicalTrials.gov and reference Identifier NCT04871282 (RINGSIDE).

About Desmoid Tumors
Desmoid tumors also called aggressive fibromatosis or desmoid-type fibromatosis, are rare connective tissue tumors that typically arise in the upper and lower extremities, abdominal wall, head and neck area, mesenteric root, and chest wall with the potential to arise in additional parts of the body. Desmoid tumors do not metastasize, but often aggressively infiltrate neurovascular structures and vital organs. People living with desmoid tumors are often limited in their daily life due to chronic pain, functional deficits, general decrease in their quality of life and organ dysfunction. Desmoid tumors have an annual incidence of approximately 1,700 patients in the United States and typically occur in patients between the ages of 15 and 60 years. They are most commonly diagnosed in young adults between 30-40 years of age and are more prevalent in females. Today, surgery is no longer regarded as the cornerstone treatment of desmoid tumors due to a high rate of recurrence post-surgery and there are currently no FDA-approved systemic therapies for the treatment of unresectable, recurrent or progressive desmoid tumors.

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 tumors and aggressive cancers. 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 (ACC), T-cell Acute Lymphoblastic Leukemia (T-ALL), Desmoid Tumors and Multiple Myeloma (MM). 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. AL102 is currently in a Pivotal Phase 2/3 clinical trials for patients with desmoid tumors (RINGSIDE). For more information, visit www.ayalapharma.com. 

Contacts:

Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
jallaire@lifesciadvisors.com

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

Media:
Tricia Persad-Bevil
JPA
+44-7792-524442

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 AL102, the promise and potential impact of AL102, the timing and results of our clinical trials or readouts, the prevalence of desmoid tumors and the treatment required to manage the disease, and the design of our clinical trials. 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: we have incurred significant losses since inception and anticipate that we will continue to incur losses for the foreseeable future; we are not currently profitable, and we may never achieve or sustain profitability; we will require additional capital to fund our operations, and if we fail to obtain necessary financing, we may not be able to complete the development and commercialization of AL101 and AL102; we have a limited operating history and no history of commercializing pharmaceutical products, which may make it difficult to evaluate the prospects for our future viability; we are heavily dependent on the success of AL101 and AL102, our most advanced product candidates, which are still under clinical development, and if either AL101 or AL102 does not receive regulatory approval or is not successfully commercialized, our business may be harmed; due to our limited resources and access to capital, we must prioritize development of certain programs and product candidates; these decisions may prove to be wrong and may adversely affect our business; the outbreak of COVID-19, may adversely affect our business, including our clinical trials; our ability to use our net operating loss carry forwards to offset future taxable income may be subject to certain limitations; our product candidates are designed for patients with genetically defined cancers, which is a rapidly evolving area of science, and the approach we are taking to discover and develop product candidates is novel and may never lead to marketable products; we were not involved in the early development of our lead product candidates, therefore, we are dependent on third parties having accurately generated, collected and interpreted data from certain preclinical studies and clinical trials for our product candidates; enrollment and retention of patients in clinical trials is an expensive and time-consuming process and could be made more difficult or rendered impossible by multiple factors outside our control; if we do not achieve our projected development and commercialization goals in the timeframes we announce and expect, the commercialization of our product candidates may be delayed and our business will be harmed; our product candidates may cause serious adverse events or undesirable side effects, which may delay or prevent marketing approval, or, if approved, require them to be taken off the market, require them to include safety warnings or otherwise limit their sales; the market opportunities for AL101 and AL102, if approved, may be smaller than we anticipate; we may not be successful in developing, or collaborating with others to develop, diagnostic tests to identify patients with Notch-activating mutations; we have never obtained marketing approval for a product candidate and we may be unable to obtain, or may be delayed in obtaining, marketing approval for any of our product candidates; even if we obtain FDA approval for our product candidates in the United States, we may never obtain approval for or commercialize them in any other jurisdiction, which would limit our ability to realize their full market potential; we have been granted Orphan Drug Designation for AL101 for the treatment of ACC and may seek Orphan Drug Designation for other indications or product candidates, and we may be unable to maintain the benefits associated with Orphan Drug Designation, including the potential for market exclusivity, and may not receive Orphan Drug Designation for other indications or for our other product candidates; although we have received Fast Track designation for AL101, and may seek Fast Track designation for our other product candidates, such designations may not actually lead to a faster development timeline, regulatory review or approval process; we face significant competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively; we are dependent on a small number of suppliers for some of the materials used to manufacture our product candidates, and on one company for the manufacture of the active pharmaceutical ingredient for each of our product candidates; if we are unable to enter into new collaborations, or if these collaborations are not successful, our business could be adversely affected; enacted and future healthcare legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates, if approved, and may affect the prices we may set; if we are unable to obtain, maintain, protect and enforce patent and other intellectual property protection for our technology and products or if the scope of the patent or other intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize products and technology similar or identical to ours, and we may not be able to compete effectively in our markets; we may engage in acquisitions or in-licensing transactions that could disrupt our business, cause dilution to our stockholders or reduce our financial resources; and risks related to our operations in Israel could materially adversely impact our business, financial condition and results of operations.

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, 2021 filed with the U.S. Securities and Exchange Commission (SEC) on March 28, 2022 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.

Release – Onconova Therapeutics Announces Updated Data from Investigator-sponsored Phase 1-2a Trial Evaluating Rigosertib in Combination with Nivolumab in Advanced KRAS-mutated Non-Small Cell Lung Cancer at the ESMO Congress 2022

Posted on by aweinsoff@channelchek.com

Research, News, and Market Data on ONTX

  • Data show an early signal of efficacy in an extensively pre-treated population with 1 complete response and 2 partial responses achieved in 14 evaluable patients

  • Responses achieved in patients with 3 distinct and different KRAS mutations, confirming the MOA of rigosertib being KRAS+ agnostic

  • 4 of 14 (29%) evaluable patients demonstrated disease control

  • The combination of rigosertib and nivolumab has been well tolerated with no synergistic toxicities observed to-date

NEWTOWN, Pa., Sept. 12, 2022 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced updated data from an investigator-sponsored Phase 1/2a trial of oral rigosertib plus the anti-PD-1 immune checkpoint inhibitor (ICI) nivolumab in advanced KRAS-mutated (KRAS+) non-small cell lung cancer (NSCLC). The data, which are featured in a poster at the European Society for Medical Oncology (ESMO) Congress 2022, show an early and encouraging signal of efficacy in the trial’s extensively pre-treated population. The studied doublet has been well tolerated to-date.

“The emerging data being presented at ESMO are encouraging, as treatment with rigosertib plus nivolumab led to both complete and partial responses in patients with KRAS-mutated lung cancers who failed prior ICI therapy,” said Dr Rajwanth Veluswamy, the principal investigator of the study. “Objective responses showcased rigosertib’s KRAS mutation-agnostic mechanism of action, as each responding patient had a tumor with a different underlying variant. This differentiates rigosertib from agents targeting a single KRAS mutation variant, and positions it to potentially address the unmet needs of a much broader patient population. In addition, the ESMO data demonstrated activity in multiple patients with both low PD-L1 expression at diagnosis and STK11/LKB1 co-mutations, both poor predictive features for current lung cancer treatments.”

Key data from the presentation include:

Demographics:

  • All enrolled patients failed at least one line of prior therapy with a PD-1 checkpoint inhibitor (includes evaluable and non-evaluable patients)
  • 80% of enrolled patients failed at least two lines of prior therapy

Response results (as of August 15th, 2022-data cutoff date):

  • 3 of 14 evaluable patients achieved an objective response
    • 1 patient achieved a complete response (CR) as per RECIST Criteria, with complete resolution of the primary lung tumor as well as sites of metastatic disease.
    • 2 patients achieved a partial response (PR)
    • Responses were achieved in patients with 3 distinct KRAS mutations (CR: KRAS G12V; PRs: KRAS G12C/STK11 and Q61H/STK11)
  • The mean duration of response is 6.75 months
  • 4 of 14 evaluable patients achieved disease control (CR, PR, or stable disease)

Safety results:

  • The studied doublet has been generally well tolerated. Treatment-related adverse events (TRAE) have been mostly mild and manageable.
  • One dose limiting toxicity of grade 3 hyponatremia has been observed (previously documented with rigosertib)
  • Urinary toxicities well documented with rigosertib are the most common TRAE
  • No unexpected safety events or synergistic toxicities have been observed

Steven M. Fruchtman, M.D., President and Chief Executive Officer of Onconova, commented, “The evidence of efficacy observed in the trial’s highly challenging population suggests rigosertib may synergize with ICI and potentially provide clinical benefit to patients with limited therapeutic options. This hypothesis is supported both by these latest clinical data and the results of preclinical studies in multiple indications. Looking forward, we expect the maturation of the trial’s current results, as well as the new data we expect to collect by enrolling additional patients, to provide key insights that will inform the next steps for rigosertib’s current investigator-sponsored study program.”   

The ESMO poster (#1018P) is titled “Phase 1/2 Trial of Rigosertib and Nivolumab for KRAS Mutated Non-Small Cell Lung Cancer (NSCLC) Patients.” It is currently available for viewing on the congress’s virtual platform and is being presented by the trial’s principal investigator, Rajwanth Veluswamy, M.D., Assistant Professor, Medicine, Hematology and Medical Oncology, Icahn School of Medicine at Mount Sinai, today during Poster Session 14. The poster is available on the “Scientific Presentations” section of the Onconova website.

About the Investigator-sponsored Phase 1/2a Trial

This Phase 1/2a trial is designed to evaluate the combination of rigosertib and nivolumab in advanced KRAS+ metastatic NSCLC patients who have progressed on standard-of-care with anti-PD-1 monotherapy or anti-PD-1 in combination with chemotherapy. It includes a dose-escalating Phase 1 portion followed by a Phase 2a dose-expansion portion. Patients in the trial receive oral rigosertib twice daily on days 1-21, and intravenous nivolumab on days 1 and 15 of 28-day cycles. The primary endpoints of the trial are safety assessments to determine maximum tolerated dose, and overall response rate. Secondary endpoints include progression-free survival and overall survival. For more information on the trial, see ClinicalTrials.gov Identifier: NCT04263090.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China.

Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.

For more information, please visit www.onconova.com.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding the timing of Onconova’s and investigator-initiated clinical development and trial data, and the mechanisms, therapeutic effects, and indications for Onconova’s product candidates. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Mark Guerin
Onconova Therapeutics, Inc.
267-759-3680
ir@onconova.us 
https://www.onconova.com/contact/

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
bmackle@lifesciadvisors.com 

Cocrystal Pharma (COCP) – Influenza Program Update

Posted on by cpatches


Monday, September 12, 2022

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.

Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.

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

Cocrystal Has Reported Progress In Its Influenza Program.  Cocrystal has reported Phase 1 data and plans for a Phase 2a  trial for CC-42344, its oral drug for seasonal and pandemic influenza.  The drug is an inhibitor of the enzyme PB2 (polymerase basic 2), a subunit of the RNA polymerase required for the replication cycle of the virus.  This early point of action could make it effective against all strains of influenza.

Phase 1 Data Confirmed Once-Daily Dosing.  In July 2022, Cocrystal announced pharmacokinetic data from its Phase 1 trial. The single ascending-dose stage met its goals, confirming administration on a once-daily basis.  Patient enrollment continues in the multiple ascending dose portion.  The company plans to present the study data at a medical meeting later in the year.


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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. 

Release – Baudax Bio to Present at the 24th Annual H.C. Wainwright Healthcare Conference

Posted on by aweinsoff@channelchek.com

Research, News, and Market Data on BXRX

September 08, 2022 4:05pm EDT

MALVERN, Pa., Sept. 08, 2022 (GLOBE NEWSWIRE) — Baudax Bio, Inc. (NASDAQ:BXRX), a pharmaceutical company focused on innovative products for acute care settings, today announced that Gerri Henwood, the Company’s President and Chief Executive Officer, will present at the 24th Annual H.C. Wainwright Healthcare Conference, taking place in New York, New York on Wednesday, September 14, 2022 at 3:00 p.m. Eastern Time.

A webcast of the presentation will be available on the “Events” page within the investors section of the Baudax Bio website at https://www.baudaxbio.com/news-and-investors. The webcast will be archived on the company’s website for 90 days following the event.

About Baudax Bio

Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. Baudax Bio markets ANJESO®, the first and only 24-hour, non-opioid, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two clinical-stage, novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.

CONTACT:

Investor Relations Contact:

Argot Partners
Sam Martin / Kaela Ilami
(212) 600-1902
baudaxbio@argotpartners.com

Media Contact:

Argot Partners
David Rosen
(212) 600-1902
david.rosen@argotpartners.com

Source: Baudax Bio, Inc.

Released September 8, 2022

Release – Ocugen CSO to Participate in 3rd Annual Gene Therapy for Ophthalmic Disorders Conference

Posted on by cpatches

Research, News, and Market Data on OCGN

MALVERN, Pa., Sept. 08, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Company’s Chief Scientific Officer, Arun Upadhyay, PhD, will be among the featured speakers at the 3rd Annual Gene Therapy for Ophthalmic Disorders conference, which is being held Sept. 13-16 in Danvers, Massachusetts.

Details regarding Dr. Upadhyay’s presentation are as follows:

Event:3rd Annual Gene Therapy for Ophthalmic Disorders Conference
Topic:Highlighting the Modifier Gene Therapy Approach for the Treatment of Retinitis Pigmentosa
Date:September 14, 2022
Time:9:15 a.m. ET
Location:DoubleTree by Hilton Boston North Shore, Danvers, Mass.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.

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 statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. 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.

Contact:
Tiffany Hamilton
Head of Communications
IR@ocugen.com

Ortho Regenerative Technologies Inc. (ORTIF) – Ortho Regenerative Technologies Inc. Is Now ChitogenX Inc.

Posted on by cpatches


Thursday, September 08, 2022

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

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

What’s in a name?  The rebranding identifies the Company’s CHITOSAN based regenerative medicine technology while expanding the scope of the technology beyone orthopedics. While the near term pipeline is focused on the repair of orthopedic soft tissue (rotator cuff and meniscus) as initial markets with great need, the Company’s proprietary platform has much broader potential regenerative applications, including in cardiovascular, dermatology, wound healing, oncology and neurology.

Tickers will change.  The corporate rebranding was approved at the last Annual General and Special Meeting of Shareholders held July 21, 2022, following the passing of a special resolution authorizing a name change. Effective on or around September 12th, shares will begin trading on the CSE as CHGX.  Subject to change, it is expected that the OTCQB ticker will also be updated to CHGX.


Get the Full Report

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. 

Release – Avivagen Announces Publication of Peer-Reviewed Article in Food and Chemical Toxicology Journal

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Research, New, and Market Data on VIVXF

Publication of this Scientific Paper on the Safety of Oxidized Beta Carotene  is a milestone on the path to broader regulatory clearance to market and sell OxC-beta™ Products in the U.S. and supports wide-spread adoption of the technology around the world

Ottawa, ON /Business Wire/ September 7, 2022 /– Avivagen Inc.  (TSXV:VIV, OTCQB:VIVXF) (“Avivagen”), a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that safely enhances feed intake and supports immune function, thereby supporting general health and performance, is pleased to announce that its scientific paper entitled “Safety and uptake of fully Oxidized Beta Carotene” (the active ingredient in the company’s OxC-beta™ product line) has been published in the Journal of Food and Chemical Toxicology. A link to the article is provided below.

“With any innovative product, adoption and uptake require a robust understanding of the benefits and safety of that product and necessitate significant scrutiny. Avivagen has been working tirelessly to showcase the multiple, compelling use cases of OxC-beta and concurrent proven safety. This new peer reviewed publication, one of now ten such publications on OxC-beta, represents the cumulative work on the attractive safety profile of our product. This pioneering work by Dr. Graham Burton, co-founder of Avivagen, and his team lays important groundwork to seek further regulatory approvals for OxC-beta in the U.S. and around the world, for multiple use cases and with strong, proven scientific underpinnings expected by our end market customers.”, said Kym Anthony, Avivagen CEO.  “We look forward to sharing these results with existing and new customers and providing this to regulatory bodies around the world as we look to further expand our access to large markets.”

Please refer to the full content of the peer-reviewed publication on the “Safety and uptake of fully oxidized Beta Carotene” by following the link below: https://www.sciencedirect.com/science/article/pii/S0278691522005853

About OxC-beta™ Technology
β-carotene has been primarily recognized for the health benefits it provides when it is transformed into vitamin A by biochemical reaction with oxygen in the body. Avivagen discovered that spontaneous oxidation of β-carotene produces a mixture of oxidation compounds other than vitamin A that also provide important health benefits. The mixture of β-carotene oxidation compounds, OxC-beta, a product developed by Avivagen, contains neither β-carotene or vitamin A, indicating its health benefits stem uniquely from the oxidation compounds themselves. Incorporation of OxC-beta into products for livestock, companion animal and humans show a range of practical benefits. A peer reviewed paper just published in the prestigious Food and Chemical Toxicology journal (https://doi.org/10.1016/j.fct.2022.113387) has reported that OxC-beta has a very high margin of safety and the discovery that the natural counterpart of OxC-beta is present at significant levels in mouse tissues and blood. A standard toxicology study conducted in rats established a No Observed Adverse Effect Level that is several thousand-fold higher than the level of supplementation with OxC-beta used in livestock, companion animals and humans. These findings are highly significant in establishing the safety of OxC-beta with respect to regulatory approvals of the product in various major jurisdictions.

About Avivagen
Avivagen is a life sciences corporation focused on developing and commercializing products for livestock, companion animal and human applications that, by safely supporting immune function, promote general health and performance.  It is a public corporation traded on the TSX Venture Exchange under the symbol VIV and is headquartered in Ottawa, Canada, based in partnership facilities of the National Research Council of Canada. For more information, visit www.avivagen.com. The contents of the website are expressly not incorporated by reference in this press release.

About OxC-beta™ Livestock
Avivagen’s OxC-beta™ technology is derived from Avivagen discoveries about β-carotene and other carotenoids, compounds that give certain fruits and vegetables their bright colours. Through support of immune function the technology provides a non-antibiotic means of promoting health and growth. OxC-beta™ Livestock is a proprietary product shown to be an effective and economic alternative to the antibiotics commonly added to livestock feeds. The product is currently available for sale in the United States, Philippines, Mexico, Taiwan, New Zealand, Thailand, Brazil, Australia, Vietnam and Malaysia.

Avivagen’s OxC-beta™ Livestock product is safe, effective and could fulfill the global mandate to remove all in-feed antibiotics as growth promoters. Numerous international livestock trials with poultry and swine using OxC-beta™ Livestock have proven that the product performs as well as, and, sometimes, in some aspects, better than in-feed antibiotics.

Forward Looking Statements
This news release includes certain forward-looking statements that are based upon the current expectations of management. Forward-looking statements involve risks and uncertainties associated with the business of Avivagen Inc. and the environment in which the business operates. Any statements contained herein that are not statements of historical facts may be deemed to be forward-looking, including those identified by the expressions “aim”, “anticipate”, “appear”, “believe”, “consider”, “could”, “estimate”, “expect”, “if”, “intend”, “goal”, “hope”, “likely”, “may”, “plan”, “possibly”, “potentially”, “pursue”, “seem”, “should”, “whether”, “will”, “would” and similar expressions.

Statements set out in this news release relating to the potential positive impacts expected to arise from the publication of the above referenced article, the possibility for further regulatory approvals and expanded use cases for Avivagen’s products, the future growth and prospects for Avivagen and the possibility for OxC-beta™ Livestock to replace antibiotics in livestock feeds as growth promoters are forward-looking statements. These forward-looking statements are subject to a number of risks and uncertainties that could cause actual results or events to differ materially from current expectations. For instance, Avivagen’s products may not gain market acceptance or regulatory approval in new jurisdictions or for new applications and may not be widely accepted as a replacement for antibiotics as growth promoters in livestock feeds due to many factors, many of which are outside of Avivagen’s control.  Readers are referred to the risk factors associated with the business of Avivagen set out in Avivagen’s most recent management’s discussion and analysis of financial condition available at www.SEDAR.com. Except as required by law, Avivagen assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those reflected in the forward-looking statements.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

For more information:
Avivagen Inc.
Drew Basek
Director of Investor Relations
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Phone: 416-540-0733
E-mail: d.basek@avivagen.com

Kym Anthony
Chief Executive Officer
100 Sussex Drive, Ottawa, Ontario, Canada K1A 0R6 Head Office Phone: 613-949-8164
Website: www.avivagen.com
Copyright © 2022 Avivagen Inc. OxC-beta™ is a trademark of Avivagen Inc.

Release – Cocrystal Pharma to Participate in the H.C. Wainwright Global Investment Conference

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Research, News, and Market Data on COCP

BOTHELL, Wash., Sept. 06, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) announces that management will participate virtually in the hybrid in-person and virtual H.C. Wainwright 24th Annual Global Investment Conference being held September 12-14, 2022.

An on-demand presentation by James Martin, CFO and interim co-CEO, will be available to conference attendees on the H.C. Wainwright platform beginning Monday, September 12 at 7:00 a.m. Eastern time. Management is available throughout the conference for virtual one-on-one meetings. Institutional investors and industry professionals can register to attend the conference virtually or in-person at the Lotte New York Palace. The Cocrystal presentation will be posted to the Company’s website beginning September 12.

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.

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

Media Contact:
JQA Partners
Jules Abraham
917-885-7378
Jabraham@jqapartners.com

Source: Cocrystal Pharma, Inc.

Released September 6, 2022

Release – Ocugen, Inc. to Present at H.C. Wainwright 24th Annual Global Investment Conference

Posted on by cpatches

Research, News, and Market Data on OCGN

MALVERN, Pa., Sept. 06, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen, will participate in an in-person fireside chat at the H.C. Wainwright 24th Annual Global Investment Conference being held September 12-14, 2022 in New York, NY.

Details regarding Dr. Musunuri’s fireside chat are as follows:

Event:H.C. Wainwright 24th Annual Global Investment Conference
Date:September 12, 2022
Time:3:30 – 4:00 p.m. ET
Location:Lotte New York Palace Hotel
Webcast:Live Fireside Chat

A live video webcast beginning at 3:30 p.m. ET on the day of the presentation will be available on the events page of the Ocugen investor site. The webcast replay will be archived for 90 days following the event.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.

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 statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. 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.

Contact:
Tiffany Hamilton
Head of Communications
IR@ocugen.com

Release – Tonix Pharmaceuticals Announces Oral Presentations Involving TNX-1500 (Fc-Modified Anti-CD40L mAb) at the International Congress of The Transplantation Society (TTS 2022)

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Research, News, and Market Data on TNXP

CHATHAM, N.J., Sept. 06, 2022 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced three oral presentations by academic collaborators at the 29th International Congress of The Transplantation Society (TTS 2022) being held September 10-14, 2022 in Buenos Aires, Argentina, and virtually. Copies of the presentations will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference.

Oral Presentation Details

Title:Long-term rejection free renal allograft survival with Fc-modified anti-CD154 antibody monotherapy in nonhuman primates
Date:Monday, September 12, 2022
Time:4:35 p.m. EDT (17:35 ART)
Session:Campfire Session: Models, mechanisms & therapies
PresenterGrace Lassiter, M.D., Research Fellow of the Kawai Lab
  
Title:Monotherapy with TNX-1500, a Fc-modified anti-CD154mAb, prolongs cardiac allograft survival in cynomolgus monkeys
Date:Tuesday, September 13, 2022
Time:3:25 p.m. EDT (16:25 ART)
Session:Mini-Oral Abstracts Session: Snap-shots of thoracic transplantation
PresenterKohei Kinoshita, M.D., Research Fellow of the Pierson Lab
  
Title:Long-term (>1 year) rejection/TMA free survival of kidney xenografts with triple xenoantigen knockout and multiple human transgenes in nonhuman primates
Date:Wednesday, September 14, 2022
Time:10:00 a.m EDT (11:00 ART)
Session:Mini-Oral Abstracts Session: Xenotransplantation
PresenterGrace Lassiter, M.D., Research Fellow of the Kawai Lab

Tonix Pharmaceuticals Holding Corp.*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022 and expects interim data in the first half of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is expected to enter the clinic with a Phase 2 study in the fourth quarter of 2022. Tonix’s rare disease portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the first half of 2023. Tonix’s infectious disease pipeline consists of a vaccine in development to prevent smallpox and monkeypox, next-generation vaccines to prevent COVID-19, and a platform to make fully human monoclonal antibodies to treat COVID-19. TNX-801, Tonix’s vaccine in development to prevent smallpox and monkeypox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in Kenya in the first half of 2023. Tonix’s lead vaccine candidate for COVID-19 is TNX-1850, a live virus vaccines based on Tonix’s recombinant pox live virus vector vaccine platform. A Phase 1 study of the COVID-19 vaccine is expected to be initiated in the second half of 2023.

*All of Tonix’s product candidates are investigational new drugs or biologics and have not been approved for any indication.

This press release and further information about Tonix can be found at www.tonixpharma.com.

Forward Looking Statements

Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2021, as filed with the Securities and Exchange Commission (the “SEC”) on March 14, 2022, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com 
(862) 904-8182

Olipriya Das, Ph.D. (media)
Russo Partners
Olipriya.Das@russopartnersllc.com 
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com 
(443) 213-0505

Source: Tonix Pharmaceuticals Holding Corp.Released September 6, 2022

Biotech and Pharmaceutical Companies How they are Different

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Image Credit: Pixabay (Pexels)

The Differences Investors Should Know Between Biotech Companies and Pharmaceutical Manufacturers

In mid-June of this year, the stock market seemed to have changed. The oil sector that had been up for most of the year began trending down, and most other industries that had been hard hit began moving up in a mid-year “V” shape. The new leader replacing oil was biotech, which had been one of the hardest hit at the beginning of the year. It makes sense that biotech trades with its own cycles – it’s a very different sector. There is reason to believe that fundamentals favor the biotech sector now, below we discuss the nuances of these very interesting companies, and explain how they are different than other drug companies..

With biotech stocks, regulators, financiers, the highly educated, geniuses, technology, and investors all come together to help people in the most important way – life and health. For investors, it provides its own set of challenges, and at least two possible rewards. If an investor uncovers the stock that has the right mix of science, money and regulatory approval, they may not only cash in like few other sectors, but also be part of improving the health of generations to come.

Above, I did use the word “possible”. This is true of all stocks in all sectors, but the volatility in the biotech sector is a demonstration of the adage that for bigger potential rewards, one would have to increase their risk. Companies in this industry, often startups with great patents, burn through millions of dollars, often with zero earnings, and with any potential for earnings years off and contingent on being right about their science and the products it can provide. This means stock selection in biotech requires a deeper understanding of the company and potential for its work than in other sectors.


What’s included in the Biotechnology Sector?

Biotechnology focuses on novel drug development and clinical research aimed at treating diseases and medical conditions.

They are almost always unprofitable, and many have no revenue at all. In fact, some stock analysts would suggest that the main distinction between a biotech and a pharmaceutical company lies in profitability. Biotechnology is often characterized as being more research and development than manufacturing. They serve as incubators for the ideas that lead to the next “miracle” cures. But, drug development has a long development timeline. A successful candidate can take a decade or more to go from idea to available medicine. That’s if it is among the roughly 10% of prospective candidates that reaches approval for use.

Why would anyone invest in a company that is developing a pipeline of potential products that each have about a 10% chance of approval? Because breakthrough drugs, and the potential for breakthroughs, each step forward on the path toward approval often provides a huge upward move in the stock price. Even if not eventually approved. Conversely failure to move forward often comes with a rapid decline from disappointment.


Differences Between Biotech and Pharmaceuticals

There is a lot of overlap and a gray area between what is biotech and what is pharmaceutical. Here are some guidelines of where the differences often lay.

Biotechnology ventures tend to be more entrepreneurial pursuits with a lot on the line for the success of an idea, while pharmaceutical companies have an ongoing business and can better gauge and manage business risk. One reason is pharmaceutical companies often have a large diversified portfolio of approved drugs they market. They may also have drugs in various stages of research and development, but these costs are usually investments in future products using current revenue.

A biotechnology firm is often based on “new” science and driven to develop a useful and profitable application for the science. An example of a growing segment of biotech is biopharma which develops drugs originating living organisms.

Large pharmaceutical companies often pay stable dividends to shareholders, this is rare in companies that fall under the biotech umbrella, if there are earnings it may be reinvested in moving forward the study of drugs in development. Many biotechs’ strength is to explore and develop, not to market the successful result. Large pharmaceutical companies, like Johnson and Johnson (JNJ)or Eli Lilly (LLY) are marketing machines, they look to smaller biotech to be an incubator to keep their portfolio of products fresh.

The two industries also stand apart when it comes to valuation and business evaluation. Models and valuation derived from cash flow are quite relevant in assessing pharmaceutical stocks. While many analysts gamely attempt to construct discounted cash flow models for early-stage biotechs, the reality is that success is often thumbs-up or thumbs-down (“drug works” or “drug doesn’t work”).


The FDA Has Final Say

As the regulatory body that approves new drugs for the U.S. market, as well as issues permits for human clinical trials, the Food and Drug Administration (FDA) is the gatekeeper for every biotech firm. Investors should have some understanding of the FDA process and requirements or follow an analyst covering the sector that does. In order to get FDA approval, biotechs must establish a sufficient body of information that the drug is safe and effective. This is generally done through a series of at least three clinical trials.

If the trials meet the goals for safety and efficacy, the company files a request for approval called a New Drug Application (NDA). If the NDA is approved, the FDA sets the date a decision will be made by on the application.


Product Pipeline

A biotech’s pipeline is the future of the company. Investors focus a great deal on the number of entrants in the pipeline, the stages of study they are at, and the overall science, and competing products (if any). It’s critical to establish valuation forecasts.

Different biotech companies tend to operate in different stages of a product’s development. The stages are, most commonly, preclinical testing, enabling studies, clinical trials, and biologics licensing. A company that is focused on the early stage of research and development will conduct preliminary research to determine whether an idea may successfully be used against a disease or ailment. It attempts to answer, “can this technology become a product?”. This step requires translating the technology into a product. Preclinical testing is the phase of product development when a company must show that its proposed product is safe. They must also determine what the pharmacokinetics (PK) pharmacodynamics (PD) are. PK shows what happens to a drug once it enters the body, ie: how quickly its absorbed, distributed, metabolized, and excreted. Studies done for the express purpose of convincing the FDA to approve the start of clinical trials in humans are called Investigational New Drug (IND)-enabling.

Once a startup has received approval from the FDA to begin clinical trials for their product, they progress through a series of phases depending on how their product fares. These phases are:

Phase 1 This first trial’s main purpose is to determine that the product is safe for use in humans. The number of participants is kept as small as possible, and may be limited to patients who are farther along in a disease state or who have exhausted other options for treatment.

Phase 2 The second phase continues to confirm that the product is safe, but increases the number of participants and tries to determine the optimum dose and regimen.

Phase 3 Typically the last clinical trial phase and is designed to determine whether the treatment is truly effective, and is expanded to include enough people so the potential side effects are more likely to be captured in the data.

The biologics license application (BLA) is the process by which a biologic that has successfully completed clinical trials applies to the FDA to be sold to the public. A new drug application (NDA) is the same process, but for pharmaceuticals.


Take Away

Biotech investing can be very rewarding. Investors need to understand the company and the risks, and also the stage in the pipeline and the success of recent trials. Failed attempts by companies in this sector at devising the next wonder drug outnumber successes. One success may easily make up for some failures for investors.   With research into the company and even reliance on trusted analysts that specialize in biotechs, an investor can increase their chances of uncovering oversized profits. We encourage you, if you are not signed up for Channelchek and the research by Noble Capital Markets equity analysts delivered to your inbox each morning, to take the time and register with us now.


Paul Hoffman

Managing Editor, Channelchek

Sources

https://www.universitylabpartners.org/blog/product-development-in-life-science

https://biotechhealth.com/biotech-vs-pharma/

https://www.qualio.com/blog/biotech-vs-pharma

Release – Baudax Bio Announces Closing of $6.2 Million Public Offering

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Baudax Bio Announces Closing of $6.2 Million Public Offering

Research, News, and Market Data on Baudax Bio

September 01, 2022 2:56pm EDT

MALVERN, Pa., Sept. 01, 2022 (GLOBE NEWSWIRE) — Baudax Bio, Inc. (the “Company” or “Baudax Bio”) (NASDAQ: BXRX), a pharmaceutical company focused on therapeutics for acute care settings, today announced the closing of its previously announced public offering of an aggregate of 11,819,172 shares of its common stock (or pre-funded warrants in lieu thereof), together with accompanying common stock purchase warrants, at a public offering price of $0.525 per share (or pre-funded warrant) and accompanying warrants. Each share of common stock (or pre-funded warrant) was sold in the offering together with a Series A-1 warrant to purchase one share of common stock at an exercise price of $0.525 per share and a Series A-2 warrant to purchase one share of common stock at an exercise price of $0.525 per share. The Series A-1 warrants are exercisable immediately and will expire five years from the date of issuance, and the Series A-2 warrants are exercisable immediately and will expire thirteen months from the date of issuance.

H.C. Wainwright & Co. acted as the exclusive placement agent for the offering.

The gross proceeds from the offering, before deducting the placement agent’s fees and other offering expenses, were approximately $6.2 million. The Company intends to use the net proceeds from this offering for pipeline development activities and general corporate purposes.

The securities described above were offered pursuant to a registration statement on Form S-1 (File No. 333-266499), which was declared effective by the Securities and Exchange Commission (the “SEC”) on August 29, 2022. The offering was made only by means of a prospectus which is a part of the effective registration statement. A final prospectus relating to the offering has been filed with the SEC. Electronic copies of the final prospectus may be obtained on the SEC’s website at http://www.sec.gov and may also be obtained by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 856-5711 or e-mail at 
placements@hcwco.com.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Baudax Bio

Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. Baudax Bio markets ANJESO®, the first and only 24-hour, non-opioid, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO®, the Company has a pipeline of other innovative pharmaceutical assets including two clinical-stage, novel neuromuscular blocking (NMBs) agents and a proprietary chemical reversal agent specific to these NMBs.

Forward Looking
Statements

This press release contains forward-looking statements that involve risks and uncertainties. Such forward-looking statements reflect Baudax Bio’s expectations about its future performance and opportunities that involve substantial risks and uncertainties. When used herein, the words “anticipate,” “believe,” “estimate,” “may,” “upcoming,” “plan,” “target,” “goal,” “intend” and “expect” and similar expressions, as they relate to Baudax Bio or its management, are intended to identify such forward-looking statements. Forward-looking statements may include, without limitation, statements regarding market conditions and the use of net proceeds from the offering. These forward-looking statements are based on information available to Baudax Bio as of the date of publication on this internet site and are subject to a number of risks, uncertainties, and other factors that could cause Baudax Bio’s performance to differ materially from those expressed in, or implied by, these forward-looking statements. These risks and uncertainties include, among other things, risks related to market and other conditions, the ongoing economic and social consequences of the COVID-19 pandemic, Baudax Bio’s ability to advance its current product candidate pipeline through pre-clinical studies and clinical trials, Baudax Bio’s ability to raise future financing for continued development of its product candidates such as BX1000, BX2000 and BX3000, Baudax Bio’s ability to pay its debt and satisfy conditions necessary to access future tranches of debt, Baudax Bio’s ability to comply with the financial and other covenants under its credit facility, Baudax Bio’s ability to manage costs and execute on its operational and budget plans, Baudax Bio’s ability to achieve its financial goals; and Baudax Bio’s ability to obtain, maintain and successfully enforce adequate patent and other intellectual property protection. These forward-looking statements should be considered together with the risks and uncertainties that may affect Baudax Bio’s business and future results included in Baudax Bio’s filings with the Securities and Exchange Commission at www.sec.gov. These forward-looking statements are based on information currently available to Baudax Bio, and Baudax Bio assumes no obligation to update any forward-looking statements except as required by applicable law.

Investor Relations
Contact:
Argot Partners
Sam Martin / Kaela Ilami
(212) 600-1902
baudaxbio@argotpartners.com

Media Contact:
Argot Partners
David Rosen
(212) 600-1902
david.rosen@argotpartners.com 


Primary Logo

Source: Baudax Bio, Inc.

Released September 1, 2022