Wednesday, January 05, 2022

Filament Health (FLHLF)
New Phase 2 Trial for PEX010 Approved To Begin In 1Q22

Filament Health Corp is a natural psychedelic drug discovery and extraction technology company. Its mission is to see safe, approved, natural psychedelics in the hands of everyone who needs them as soon as possible. Filament engages in natural extraction technology commercialization, utilizing its intellectual property portfolio, in-house GMP facility, and Health Canada psilocybin Dealer’s License.

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

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

Additional Phase 2 Study Announced.  Filament announced that Health Canada approved a new Phase 2 clinical trial to test PEX010 in persistent depressive disorder (PDD). The trial will test a 1 milligram microdose of PEX010 against placebo to determine safety and efficacy of the microdosing.


Study Design Uses A Lower Dose.  The Phase 2 trial will test a 1 mg dose of PEX-010, Filament’s botanically derived psilocybin. It is designed as a placebo-controlled study to test safety and efficacy, with an planned enrollment of about 100 healthy patients with PDD. The trial will be conducted at the Canadian Centre for Psychedelic Science and the University of Toronto, with funding from the …

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. 

Cocrystal Pharma to Present at the H.C. Wainwright BioConnect Virtual Conference

Research, News, and Market Data on Cocrystal Pharma

 

BOTHELL, Wash., Jan. 04, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces that management will present a company overview at the H.C. Wainwright BioConnect Virtual Conference being held January 10-13, 2022. A webcast of the Company’s presentation will be available on the IR Calendar section of the Cocrystal Pharma website beginning Monday, January 10, 2022 at 7:00 a.m. Eastern time.

“This year promises to be highly eventful as we plan to initiate first-in-human studies as quickly as possible with two different SARS-CoV-2 antivirals for potential oral and inhalation COVID-19 treatments. Building on Cocrystal’s platform technology and expertise in developing antivirals, we carefully evaluated broad-spectrum antiviral activity of these SARS-CoV-2 protease inhibitors against all major SARS-CoV-2 variants including Omicron and Delta. We also obtained favorable preclinical safety data on these inhibitors,” said Sam Lee, Ph.D., Cocrystal’s President and interim co-CEO. “Our influenza A Phase 1 trial is also advancing with subject enrollment expected in the current quarter.”

“We are well positioned financially to advance our clinical plans with a strong cash position and clean debt free balance sheet,” said James Martin, CFO and interim co-CEO. “We are pleased to begin the New Year by sharing our exciting plans with the investment community.”

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 our goals of initiating Phase 1 clinical studies as rapidly as possible, expected subject enrollment for our influenza A Phase 1 clinical trial in the first quarter of 2022, and the potential efficacy of antiviral inhibitors against COVID-19. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the risks arising from supply chain disruptions on our ability to obtain products including raw materials and test animals as well as similar problems with our vendors and our current contract research organizations (CROs) and future CROs and contract manufacturing organizations , the ability of our CROs to recruit volunteers for, and to proceed with, clinical trials, the impact of the COVID-19 pandemic including new variants on the national and global economy, the duration of presently discovered COVID-19 variants and our ability to treat new variants, the cooperation of the FDA in accelerating development in our COVID-19 program, our collaboration partners’ technology and software performing as expected, the results of future preclinical and clinical trials, general risks arising from clinical trials, receipt of regulatory approvals, regulatory changes, and development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2020. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
[email protected]

Source: Cocrystal Pharma, Inc.

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

Research, News, and Market Data on Ayala Pharmaceuticals

 

— Data read-outs expected on AL102 in desmoid tumors and AL101 in ACC and TNBC —

REHOVOT, Israel and WILMINGTON, Del., Jan. 04, 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 and aggressive cancers, primarily in genetically defined patient populations, today announced its key objectives for 2022.

“We believe that 2022 will be a pivotal year for Ayala as we continue to advance our unique clinical stage portfolio of gamma secretase inhibitors to treat rare and aggressive cancers with no approved therapies,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “We hope to build on the excellent progress we made in 2021 and look forward to multiple clinical milestones that have the potential to create significant value. Specifically, we expect data read-outs from AL102 in desmoid tumors and from our AL101 programs in adenoid cystic carcinoma and triple negative breast cancer. We plan to advance AL101 into the clinic in a Phase 2 trial in T-cell acute lymphoblastic leukemia. We are pleased, also, with our ongoing collaboration with Novartis to develop AL102 in combination with its anti-BCMA agent for multiple myeloma and hope to be able to share an update on this exciting program as well.”

2021 Key Achievements

• Initiated pivotal Phase 2/3 RINGSIDE study of AL102 in desmoid tumors
• Initiated Phase 2 TENACITY study of AL101 in Notch-activated TNBC
• Initiated Phase 1 trial of AL102 in combination with BCMA targeting agent WVT078 in relapsed/refractory MM (in collaboration with Novartis)
• Presented positive preliminary data from the 6 mg cohort of the ongoing Phase 2 ACCURACY study of AL101 in recurrent/metastatic ACC at ESMO 2021
• Published case studies highlighting clinical activity of AL101 with long-lasting responses in patients with desmoid tumors
• Completed $25 million strategic equity financing

Expected Milestones in 2022

Initial Interim Data from Pivotal Phase 2/3 RINGSIDE Trial in Desmoid Tumors (Mid-2022):

• Ayala expects to report an initial interim data read-out from Part A of the Phase 2/3 RINGSIDE trial of AL102 in desmoid tumors in mid-2022. Part A is open-label and is evaluating safety, tolerability, and tumor volume by MRI after 16 weeks.
• Part B of the study will start immediately after dose selection from Part A and will be a double-blind placebo-controlled study enrolling up to 156 patients with progressive disease, randomized 2:1 between AL102 or placebo.
• If successful RINGSIDE will be used as a registrational study.
  

Preliminary Data from Phase 2 TENACITY Trial of AL101 in Triple Negative Breast Cancer (H2-2022)

• Preliminary data from the Phase 2 TENACITY clinical trial of AL101, for the treatment of patients with Notch-activated recurrent or metastatic (R/M) triple negative breast cancer (TNBC) are expected in H2-2022.
• TENACITY is an open-label, multicenter, single arm study that is expected to initially enroll up to 26 patients with Notch-activated R/M TNBC whose disease has recurred or progressed after three or fewer lines of prior therapy.
• The primary endpoint is the objective response rate. Secondary endpoints include safety, duration of response, progression free survival, and relapse free survival.
• Ayala is currently the only company pursuing clinical development of a Notch inhibitor for TNBC.

Additional Data from Phase 2 ACCURACY Trial of AL101 in Adenoid Cystic Carcinoma (Mid-2022)

• The ongoing ACCURACY trial is an open-label, single-arm Phase 2 clinical trial evaluating AL101 as monotherapy for the treatment of R/M ACC for Notch-activated mutations patients.
• Part 1 of the trial included 45 subjects dosed at 4 mg of AL101 IV once weekly. Final data from the 4 mg and additional data from the 6 mg cohort which includes 42 subjects are expected to be announced during 2022.
• The primary endpoint is the objective response rate as measured by RECIST 1.1 criteria. Secondary endpoints include objective response rate by investigator review, duration of response and progression-free survival by an independent review committee and an investigator review, overall survival, safety and tolerability, and pharmacokinetics.
• AL101, if approved, could potentially be the first systemic therapy for ACC.

Initiate Phase 2 Clinical Trial Evaluating AL101 in T-cell Acute Lymphoblastic Leukemia (H2-2022)

• Ayala plans to begin a Phase 2 clinical trial evaluating AL101 in R/R T-ALL
• Notch is known to be a critical component of T-cell development and is inherently implicated as a tumorigenic driver in T-ALL. Approximately 65% of all T-ALL patients have Notch-activating mutations.

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

Contacts:

Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
[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, the timing and results of any clinical trials or readouts, the sufficiency of cash to fund operations, and the anticipated impact of COVID-19, on our business. 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; our existing collaboration with Novartis is, and any future collaborations will be, important to our business. If we are unable to maintain our existing collaboration or 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, 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.

	Image Credit: Zach Skiles (MIT)

Investigating the Embattled Brain

 

Laura Carter | MIT School of Science

 

A car backfires in a parking lot. An army veteran, recently returned from a combat zone, might duck and cover. He knows that he is no longer in an active war zone, but he was trained to react before thinking, an ability that meant life over death at one point in his life.

That training is so ingrained it has physically altered the way his brain works, weakening the connection between the amygdala, which is responsible for emotions like fear, and the prefrontal cortex, which regulates or controls these emotional responses.

How post-traumatic stress disorder (PTSD) — a mental condition caused by a severe psychological shock that leaves persistent symptoms such as anxiety, depression, sleep disturbance, and even physical pain — affects the brain and its functions is the focus of graduate student Omar Rutledge’s research in the Department of Brain and Cognitive Sciences. He is uniquely situated to study this topic, having been deployed to Iraq himself from March 2003 to July 2004, resulting in firsthand experience with PTSD.

 

Coronavirus Impedes and Inspires

Rutledge, a third-year PhD candidate, is looking at ways to specifically prevent situations in which acting on a triggering event before thinking is no longer a useful survival skill. For example, when our brains sense fear, they send signals that may temporarily alter our skin to conduct electricity more easily — think of the infamous polygraph, or lie-detector, test. In the future, a device like a watch could measure this “skin conductance” and send an alert allowing the wearer to prioritize managing their response to the triggering event, such as breathing more slowly instead of ducking behind an object, effectively retraining the brain to be less responsive to triggering events.

Though Covid-19 has put some aspects of this research on hold, the pandemic has inspired another project based on the need for social distancing. Rutledge wants to test whether the loneliness caused by physical distancing protocols can induce physical or chemical changes in the brain similar to changes affiliated with PTSD.

Imagine walking down the street at night. Someone else approaches from the other direction. If someone is accompanying you, that new person is likely not evaluated as a threat. When you are by yourself? “Most likely,” he asserts. The longer humans are alone, the more other people become perceived as threats.

“There’s this hypervigilance that occurs in loneliness, and there’s also something very similar that occurs in PTSD — a heightened awareness of potential threats. The combination of the two may lead to more adverse reactions in people with PTSD,” says Rutledge, who is the recipient of the Michael Ferrara Graduate Fellowship provided by the McGovern Institute’s Poitras Center for Psychiatric Disorders Research, a fellowship made possible by the many friends and family of Michael Ferrara.

Work has already been done at MIT to investigate short-term loneliness’ effect on the brain on a social level. In his future research plans, Rutledge said he hopes to explore whether and how chronic loneliness causes cognitive impairment. From there, further investigation could determine if loneliness has a deeper impact on veterans who have PTSD.

 

From War Zone to Campus

After making the seemingly impossible transition back from Iraq into civilian life in the States, Rutledge turned to psychology to learn more about what he was experiencing, earning a bachelor’s degree in psychology from the University of Alaska at Fairbanks in 2012. To his dismay, he found little had been done to truly understand the nature of combat-associated PTSD. 

For a broken bone, a doctor diagnoses the problem via X-ray, develops a plan to correct the issue, employs the necessary steps for repair, and then evaluates if the treatment succeeded. There is no analogous process for mental disorders.

“We can’t scan your individual brain and come up with a list of things that we can do to improve your situation. There’s nothing like that,” Rutledge says. “But that doesn’t mean we can’t try. That’s something that’s been on my mind since the very beginning.” He went on to earn a master’s degree in biomedical imaging at the University of California at San Francisco, which he completed in 2015.

For his next step, he planned to pursue a doctorate in a neuroscience program in order to go beyond understanding what is physically happening in the brain and begin to tie the brain to the mind using various tools.

But he never imagined being able to do this work at MIT.

 

A New Kind of Mission

Rutledge’s firsthand combat experience has enabled prior studies into PTSD with veterans to go deeper despite dredging up painful memories. “Even though I may be reopening my own wounds by listening to others share their stories, if I can help other veterans heal, I feel it’s worth it. In the process, it makes me a little bit stronger as well,” he says.

Last year, Rutledge received a James S. (1972) and Muguette Alder Fellowship, which is awarded annually to a graduate student in brain and cognitive sciences working on bipolar disorder and related diseases or, more broadly, mental illness, and is sponsored by a gift from Jim and Muguette Alder.

With Rutledge now a part of the “Gab Lab,” John Gabrieli, the Grover Hermann Professor of Health Sciences and Technology, cognitive neuroscience professor in the Department of Brain and Cognitive Sciences, and member of McGovern Institute, has someone who can advocate for PTSD research at MIT.

“I feel like it has been a mission of mine to do this kind of work,” explains Rutledge. “In the world of PTSD research, I look to my left and to my right, and I don’t see other veterans, certainly not a former infantry guy. If there are so few of us in this space, I feel like I have an obligation to make a difference for all who suffer from the traumatic experiences of war.”

 

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From Trippy Drugs to Therapeutic Aids – How Psychedelics Got Their Groove Back

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Does Your Personality Make You More Likely to be Scammed?

 

 

 

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Genprex Expands Gene Therapy Oncology Pipeline to Include Small Cell Lung Cancer

Research, News, and Market Data on Genprex

 

Tuesday, January 04, 2022

Baudax Bio, Inc. (BXRX)
Taking the Pain Away; Initiating Coverage with an Outperform Rating

Baudax Bio is a biopharmaceutical company focused on developing therapies for post-operative pain, peri-operative pain, and anesthesia. The company currently has one approved therapy in ANJESO for post-operative pain. Proprietary ANJESO (meloxicam) injection is the first and only once-daily IV analgesic. The company also has a pipeline of early-stage candidates with two novel neuromuscular blocking agents (NMBAs), a proprietary related reversal agent to their NMBAs, and Dex-IN, an intranasal formulation of dexmedetomidine (Dex) that has sedative, analgesic, and anti-anxiety properties.

Gregory Aurand, Senior Research Analyst, Noble Capital Markets, Inc.

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

Baudax Bio Is Developing Non-Addictive Therapies For Pain Management.  The opioid crisis continues and Baudax has large market opportunity proprietary treatments for post-operative pain, peri-operative pain, and anesthesia, with one non-addicting product FDA approved and commercialized for post-operative pain.


ANJESO Is the First and Only Once-Daily IV Analgesic.  ANJESO is approved for management of moderate to severe pain, alone or in combination with other non-NSAID analgesics for up to 24 hours of pain relief. The product is a once-daily NSAID IV injection with preferential Cox-2 activity …

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. 

Genprex Expands Gene Therapy Oncology Pipeline to Include Small Cell Lung Cancer

Research, News, and Market Data on Genprex

 

	Image Credit: Zach Skiles (MIT)

Investigating the Embattled Brain

 

Laura Carter | MIT School of Science

 

A car backfires in a parking lot. An army veteran, recently returned from a combat zone, might duck and cover. He knows that he is no longer in an active war zone, but he was trained to react before thinking, an ability that meant life over death at one point in his life.

That training is so ingrained it has physically altered the way his brain works, weakening the connection between the amygdala, which is responsible for emotions like fear, and the prefrontal cortex, which regulates or controls these emotional responses.

How post-traumatic stress disorder (PTSD) — a mental condition caused by a severe psychological shock that leaves persistent symptoms such as anxiety, depression, sleep disturbance, and even physical pain — affects the brain and its functions is the focus of graduate student Omar Rutledge’s research in the Department of Brain and Cognitive Sciences. He is uniquely situated to study this topic, having been deployed to Iraq himself from March 2003 to July 2004, resulting in firsthand experience with PTSD.

 

Coronavirus Impedes and Inspires

Rutledge, a third-year PhD candidate, is looking at ways to specifically prevent situations in which acting on a triggering event before thinking is no longer a useful survival skill. For example, when our brains sense fear, they send signals that may temporarily alter our skin to conduct electricity more easily — think of the infamous polygraph, or lie-detector, test. In the future, a device like a watch could measure this “skin conductance” and send an alert allowing the wearer to prioritize managing their response to the triggering event, such as breathing more slowly instead of ducking behind an object, effectively retraining the brain to be less responsive to triggering events.

Though Covid-19 has put some aspects of this research on hold, the pandemic has inspired another project based on the need for social distancing. Rutledge wants to test whether the loneliness caused by physical distancing protocols can induce physical or chemical changes in the brain similar to changes affiliated with PTSD.

Imagine walking down the street at night. Someone else approaches from the other direction. If someone is accompanying you, that new person is likely not evaluated as a threat. When you are by yourself? “Most likely,” he asserts. The longer humans are alone, the more other people become perceived as threats.

“There’s this hypervigilance that occurs in loneliness, and there’s also something very similar that occurs in PTSD — a heightened awareness of potential threats. The combination of the two may lead to more adverse reactions in people with PTSD,” says Rutledge, who is the recipient of the Michael Ferrara Graduate Fellowship provided by the McGovern Institute’s Poitras Center for Psychiatric Disorders Research, a fellowship made possible by the many friends and family of Michael Ferrara.

Work has already been done at MIT to investigate short-term loneliness’ effect on the brain on a social level. In his future research plans, Rutledge said he hopes to explore whether and how chronic loneliness causes cognitive impairment. From there, further investigation could determine if loneliness has a deeper impact on veterans who have PTSD.

 

From War Zone to Campus

After making the seemingly impossible transition back from Iraq into civilian life in the States, Rutledge turned to psychology to learn more about what he was experiencing, earning a bachelor’s degree in psychology from the University of Alaska at Fairbanks in 2012. To his dismay, he found little had been done to truly understand the nature of combat-associated PTSD. 

For a broken bone, a doctor diagnoses the problem via X-ray, develops a plan to correct the issue, employs the necessary steps for repair, and then evaluates if the treatment succeeded. There is no analogous process for mental disorders.

“We can’t scan your individual brain and come up with a list of things that we can do to improve your situation. There’s nothing like that,” Rutledge says. “But that doesn’t mean we can’t try. That’s something that’s been on my mind since the very beginning.” He went on to earn a master’s degree in biomedical imaging at the University of California at San Francisco, which he completed in 2015.

For his next step, he planned to pursue a doctorate in a neuroscience program in order to go beyond understanding what is physically happening in the brain and begin to tie the brain to the mind using various tools.

But he never imagined being able to do this work at MIT.

 

A New Kind of Mission

Rutledge’s firsthand combat experience has enabled prior studies into PTSD with veterans to go deeper despite dredging up painful memories. “Even though I may be reopening my own wounds by listening to others share their stories, if I can help other veterans heal, I feel it’s worth it. In the process, it makes me a little bit stronger as well,” he says.

Last year, Rutledge received a James S. (1972) and Muguette Alder Fellowship, which is awarded annually to a graduate student in brain and cognitive sciences working on bipolar disorder and related diseases or, more broadly, mental illness, and is sponsored by a gift from Jim and Muguette Alder.

With Rutledge now a part of the “Gab Lab,” John Gabrieli, the Grover Hermann Professor of Health Sciences and Technology, cognitive neuroscience professor in the Department of Brain and Cognitive Sciences, and member of McGovern Institute, has someone who can advocate for PTSD research at MIT.

“I feel like it has been a mission of mine to do this kind of work,” explains Rutledge. “In the world of PTSD research, I look to my left and to my right, and I don’t see other veterans, certainly not a former infantry guy. If there are so few of us in this space, I feel like I have an obligation to make a difference for all who suffer from the traumatic experiences of war.”

 

Suggested Reading:

Will Companies that Make Covid Test Kits Rally?

From Trippy Drugs to Therapeutic Aids – How Psychedelics Got Their Groove Back

Have Reddit Posts Been Paving the Way for State Marijuana Reform?

Does Your Personality Make You More Likely to be Scammed?

 

 

 

Stay up to date. Follow us:

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

Research, News, and Market Data on Ayala Pharmaceuticals

 

— Data read-outs expected on AL102 in desmoid tumors and AL101 in ACC and TNBC —

REHOVOT, Israel and WILMINGTON, Del., Jan. 04, 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 and aggressive cancers, primarily in genetically defined patient populations, today announced its key objectives for 2022.

“We believe that 2022 will be a pivotal year for Ayala as we continue to advance our unique clinical stage portfolio of gamma secretase inhibitors to treat rare and aggressive cancers with no approved therapies,” said Roni Mamluk, Ph.D., Chief Executive Officer of Ayala. “We hope to build on the excellent progress we made in 2021 and look forward to multiple clinical milestones that have the potential to create significant value. Specifically, we expect data read-outs from AL102 in desmoid tumors and from our AL101 programs in adenoid cystic carcinoma and triple negative breast cancer. We plan to advance AL101 into the clinic in a Phase 2 trial in T-cell acute lymphoblastic leukemia. We are pleased, also, with our ongoing collaboration with Novartis to develop AL102 in combination with its anti-BCMA agent for multiple myeloma and hope to be able to share an update on this exciting program as well.”

2021 Key Achievements

• Initiated pivotal Phase 2/3 RINGSIDE study of AL102 in desmoid tumors
• Initiated Phase 2 TENACITY study of AL101 in Notch-activated TNBC
• Initiated Phase 1 trial of AL102 in combination with BCMA targeting agent WVT078 in relapsed/refractory MM (in collaboration with Novartis)
• Presented positive preliminary data from the 6 mg cohort of the ongoing Phase 2 ACCURACY study of AL101 in recurrent/metastatic ACC at ESMO 2021
• Published case studies highlighting clinical activity of AL101 with long-lasting responses in patients with desmoid tumors
• Completed $25 million strategic equity financing

Expected Milestones in 2022

Initial Interim Data from Pivotal Phase 2/3 RINGSIDE Trial in Desmoid Tumors (Mid-2022):

• Ayala expects to report an initial interim data read-out from Part A of the Phase 2/3 RINGSIDE trial of AL102 in desmoid tumors in mid-2022. Part A is open-label and is evaluating safety, tolerability, and tumor volume by MRI after 16 weeks.
• Part B of the study will start immediately after dose selection from Part A and will be a double-blind placebo-controlled study enrolling up to 156 patients with progressive disease, randomized 2:1 between AL102 or placebo.
• If successful RINGSIDE will be used as a registrational study.
  

Preliminary Data from Phase 2 TENACITY Trial of AL101 in Triple Negative Breast Cancer (H2-2022)

• Preliminary data from the Phase 2 TENACITY clinical trial of AL101, for the treatment of patients with Notch-activated recurrent or metastatic (R/M) triple negative breast cancer (TNBC) are expected in H2-2022.
• TENACITY is an open-label, multicenter, single arm study that is expected to initially enroll up to 26 patients with Notch-activated R/M TNBC whose disease has recurred or progressed after three or fewer lines of prior therapy.
• The primary endpoint is the objective response rate. Secondary endpoints include safety, duration of response, progression free survival, and relapse free survival.
• Ayala is currently the only company pursuing clinical development of a Notch inhibitor for TNBC.

Additional Data from Phase 2 ACCURACY Trial of AL101 in Adenoid Cystic Carcinoma (Mid-2022)

• The ongoing ACCURACY trial is an open-label, single-arm Phase 2 clinical trial evaluating AL101 as monotherapy for the treatment of R/M ACC for Notch-activated mutations patients.
• Part 1 of the trial included 45 subjects dosed at 4 mg of AL101 IV once weekly. Final data from the 4 mg and additional data from the 6 mg cohort which includes 42 subjects are expected to be announced during 2022.
• The primary endpoint is the objective response rate as measured by RECIST 1.1 criteria. Secondary endpoints include objective response rate by investigator review, duration of response and progression-free survival by an independent review committee and an investigator review, overall survival, safety and tolerability, and pharmacokinetics.
• AL101, if approved, could potentially be the first systemic therapy for ACC.

Initiate Phase 2 Clinical Trial Evaluating AL101 in T-cell Acute Lymphoblastic Leukemia (H2-2022)

• Ayala plans to begin a Phase 2 clinical trial evaluating AL101 in R/R T-ALL
• Notch is known to be a critical component of T-cell development and is inherently implicated as a tumorigenic driver in T-ALL. Approximately 65% of all T-ALL patients have Notch-activating mutations.

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

Contacts:

Investors:
Joyce Allaire
LifeSci Advisors LLC
+1-617-435-6602
[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, the timing and results of any clinical trials or readouts, the sufficiency of cash to fund operations, and the anticipated impact of COVID-19, on our business. 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; our existing collaboration with Novartis is, and any future collaborations will be, important to our business. If we are unable to maintain our existing collaboration or 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, 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.

Cocrystal Pharma to Present at the H.C. Wainwright BioConnect Virtual Conference

Research, News, and Market Data on Cocrystal Pharma

 

BOTHELL, Wash., Jan. 04, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces that management will present a company overview at the H.C. Wainwright BioConnect Virtual Conference being held January 10-13, 2022. A webcast of the Company’s presentation will be available on the IR Calendar section of the Cocrystal Pharma website beginning Monday, January 10, 2022 at 7:00 a.m. Eastern time.

“This year promises to be highly eventful as we plan to initiate first-in-human studies as quickly as possible with two different SARS-CoV-2 antivirals for potential oral and inhalation COVID-19 treatments. Building on Cocrystal’s platform technology and expertise in developing antivirals, we carefully evaluated broad-spectrum antiviral activity of these SARS-CoV-2 protease inhibitors against all major SARS-CoV-2 variants including Omicron and Delta. We also obtained favorable preclinical safety data on these inhibitors,” said Sam Lee, Ph.D., Cocrystal’s President and interim co-CEO. “Our influenza A Phase 1 trial is also advancing with subject enrollment expected in the current quarter.”

“We are well positioned financially to advance our clinical plans with a strong cash position and clean debt free balance sheet,” said James Martin, CFO and interim co-CEO. “We are pleased to begin the New Year by sharing our exciting plans with the investment community.”

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 our goals of initiating Phase 1 clinical studies as rapidly as possible, expected subject enrollment for our influenza A Phase 1 clinical trial in the first quarter of 2022, and the potential efficacy of antiviral inhibitors against COVID-19. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, the risks arising from supply chain disruptions on our ability to obtain products including raw materials and test animals as well as similar problems with our vendors and our current contract research organizations (CROs) and future CROs and contract manufacturing organizations , the ability of our CROs to recruit volunteers for, and to proceed with, clinical trials, the impact of the COVID-19 pandemic including new variants on the national and global economy, the duration of presently discovered COVID-19 variants and our ability to treat new variants, the cooperation of the FDA in accelerating development in our COVID-19 program, our collaboration partners’ technology and software performing as expected, the results of future preclinical and clinical trials, general risks arising from clinical trials, receipt of regulatory approvals, regulatory changes, and development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2020. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
[email protected]

Source: Cocrystal Pharma, Inc.

Tuesday, January 04, 2022

Baudax Bio, Inc. (BXRX)
Taking the Pain Away; Initiating Coverage with an Outperform Rating

Baudax Bio is a biopharmaceutical company focused on developing therapies for post-operative pain, peri-operative pain, and anesthesia. The company currently has one approved therapy in ANJESO for post-operative pain. Proprietary ANJESO (meloxicam) injection is the first and only once-daily IV analgesic. The company also has a pipeline of early-stage candidates with two novel neuromuscular blocking agents (NMBAs), a proprietary related reversal agent to their NMBAs, and Dex-IN, an intranasal formulation of dexmedetomidine (Dex) that has sedative, analgesic, and anti-anxiety properties.

Gregory Aurand, Senior Research Analyst, Noble Capital Markets, Inc.

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

Baudax Bio Is Developing Non-Addictive Therapies For Pain Management.  The opioid crisis continues and Baudax has large market opportunity proprietary treatments for post-operative pain, peri-operative pain, and anesthesia, with one non-addicting product FDA approved and commercialized for post-operative pain.


ANJESO Is the First and Only Once-Daily IV Analgesic.  ANJESO is approved for management of moderate to severe pain, alone or in combination with other non-NSAID analgesics for up to 24 hours of pain relief. The product is a once-daily NSAID IV injection with preferential Cox-2 activity …

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. 

Ocugen Partner, Bharat Biotech, Announces Positive Immunogenicity and Safety Data From COVAXIN™ (BBV152), COVID-19 Candidate Vaccine, in Children 2 – 18 YEARS

 

Research, News, and Market Data on Ocugen

 

• COVAXIN™ whole virus inactivated vaccine generated broad antibody response comparable to those seen in a large phase 3 trial in adults 18+
  
• Adverse events were primarily mild, and no serious adverse events (SAEs) were noted
• Immunobridging trial demonstrated safety, reactogenicity and immunogenicity of COVAXIN™ in children from 2 years old and up
• No cases of myocarditis or blood clots were reported, as expected with inactivated vaccines

MALVERN, Pa., Dec. 30, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc. (Nasdaq: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing novel therapeutics and vaccines, announced today that its partner, Bharat Biotech, posted results from a Phase 2/3 trial conducted in India of candidate vaccine, COVAXIN™ (BBV152), in children, aged 2 – 18 years, demonstrating a robust neutralizing antibody response and favorable safety profile on the pre-print server, medRXiv. Using a two-dose regimen administered 28-days apart, antibody responses in subjects were comparable to adult data from a previous phase 3 study. Those results demonstrated a greater than 93% reduction in severe disease. These pediatric data were the basis of Ocugen’s pediatric Emergency Use Authorization (EUA) submission in the United States for children 2-18 on November 5, 2021.

“These data represent the first positive vaccine data in children as young as two years of age and come at an important time as COVID-19 infections are seeing a spike across the globe,” said Shankar Musunuri, PhD, MBA, Chairman of the Board, Chief Executive Officer and Co-Founder of Ocugen. “The results suggest that COVAXIN™, which is made on the same platform used in traditional polio pediatric vaccines for decades, when administered in a two-dose series to children between 2-18, may offer an option that is safe while delivering a robust immune response.”

In the study, immunogenicity against key COVID-19 proteins was measured using geometric mean titer (GMT), a test that measures the amount of antibodies in the blood in response to the presence of the virus. GMT was measured across three age groups and demonstrated strong immune response after the second dose. There were no serious adverse events such as myocarditis, pericarditis, or blood clots, reported in any of the three age groups.

This immunobridging trial was intended to determine if COVAXIN™ generates the same protective immunity in children as it does in adults. To be considered equivalent, pediatric participants needed to achieve comparable GMT’s to those generated by adults in a large phase 3 clinical trial. At Day 56, across all ages, the mean SARS-CoV-2 antibody GMT was more than 30% higher in children aged 2-18 than the mean GMT from Phase 3 clinical trial involving 25,800 adult subjects aged 18+. These data demonstrate children 2-18 generate antibody response comparable to adults fully vaccinated with COVAXIN^™ (BBV152).

The study also showed increased antibody titers against 3 viral antigens (Spike – S1, Receptor Binding Domain – RBD and Nucleocapsid – NP) providing a broad immune response to COVID-19. In each case a more than 4-fold increase in neutralizing antibody titers versus baseline (titers measured in the same participants before being vaccinated) was seen in all age groups after the second dose.

Participants were not reported to have experienced any severe adverse events. The majority of adverse events noted (49%, n=374) were mild (e.g., injection site pain) and a small proportion (19%, n=6) were moderate (e.g., fever, headache). Mild to moderate adverse events resolved within 24 hours.

The data were also submitted to the Drugs Controller General of India (DCGI) to support a pediatric indication as recommended by an independent committee, known as the Subject Expert Committee (SEC), that advises the Indian regulatory agency. Bharat Biotech intends to submit data from the full Phase 3 trial to a peer-reviewed scientific publication soon.

About the Phase 2/3 Trial in Children (BBIL/BBV152/2021)
The seven-month Phase 2/3 open label, multicenter, study enrolled 525 children ages 2 years to 18 from 6 clinical trial sites across India. Conducted between May and July 2021, it was designed to evaluate the safety, reactogenicity and immunogenicity of COVAXIN™ (BBV152) on a two-dose schedule (28 days apart) in three age groups: ages 12-18, ages 6-12, and ages 2-6. Children with prior infection of SARS-CoV-2 were not excluded from the trial.

About COVAXIN™
COVAXIN™ (BBV152) is an investigational vaccine candidate product in the U.S. It was developed by Bharat Biotech in collaboration with the Indian Council of Medical Research (ICMR) – National Institute of Virology (NIV). COVAXIN™ (BBV152) is a highly purified and inactivated vaccine that is manufactured using a vero cell manufacturing platform.

With more than 125 million doses having been administered to adults outside the U.S., COVAXIN™ (BBV152) is currently authorized under emergency use in 17 countries, and applications for emergency use authorization are pending in more than 60 other countries. The World Health Organization (WHO) recently added COVAXIN™ (BBV152) to its list of vaccines authorized for emergency use. And, as many as 110 countries have agreed to mutual recognition of Covid-19 vaccination certificates with India that includes vaccination using COVAXIN™ (BBV152). The trade name, COVAXIN™, has not been evaluated by the FDA.

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. and Canadian markets. For more information, please visit www.ocugen.com.

About Bharat Biotech 
Bharat Biotech has established an excellent track record of innovation with more than 145 global patents, a wide product portfolio of more than 16 vaccines, 4 bio-therapeutics, registrations in more than 123 countries, and the World Health Organization (WHO) Pre-qualifications. Located in Genome Valley in Hyderabad, India, a hub for the global biotech industry, Bharat Biotech has built a world-class vaccine & bio-therapeutics, research & product development, Bio-Safety Level 3 manufacturing, and vaccine supply and distribution. 

Having delivered more than 4 billion doses of vaccines worldwide, Bharat Biotech continues to lead innovation and has developed vaccines for influenza H1N1, Rotavirus, Japanese Encephalitis, Rabies, Chikungunya, Zika, and the world’s first tetanus-toxoid conjugated vaccine for Typhoid. Bharat’s commitment to global social innovation programs and public-private partnerships resulted in introducing path-breaking WHO pre-qualified vaccines BIOPOLIO®, ROTAVAC®, and Typbar TCV® combatting polio, rotavirus, typhoid infections, respectively. The acquisition of the rabies vaccine facility, Chiron Behring, from GlaxoSmithKline (GSK) has positioned Bharat Biotech as the world’s largest rabies vaccine manufacturer. To learn more about Bharat Biotech, visit www.bharatbiotech.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, including statements about data published on the preprint server, medRxiv, by Bharat Biotech, which found that COVAXIN™ generated a potentially robust neutralizing antibody response and favorable safety profile in its Phase 2/3 study in pediatric patients, and the potential for this data to support our application to the U.S. Food and Drug Administration (FDA) for emergency use authorization (EUA) of COVAXIN™ in pediatric patients. 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; the risk that we may not resolve the current clinical hold on COVAXIN™ in the near term or at all, or that the FDA could make other decisions that adversely impact our ability to advance the development of COVAXIN™ in the United States, and the implications that this clinical hold may have for our request for emergency use authorization for COVAXIN for pediatric use, including the timing and scope of any such authorization; risks associated with preliminary and interim data, including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; 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 data and results from the preclinical and clinical studies of COVAXIN™, which have been conducted by Bharat Biotech in India, will be accepted by the FDA or otherwise sufficient to support our EUA submission; the size, scope, timing and outcome of any additional trials or studies that we may be required to conduct to support an EUA or biologics license application (BLA); any additional chemistry, manufacturing, and controls information that we may be required to submit to the FDA; whether and when a BLA for COVAXIN™ will be submitted to or approved by the FDA; whether developments with respect to the COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada or other jurisdictions; market demand for COVAXIN™ in the United States or Canada; decisions by the FDA or Health Canada impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN™ in the United States or Canada, 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.

Ocugen Contact:  Ken Inchausti Head, Investor Relations & Communications [email protected] Please submit investor-related inquiries to: [email protected]

Ocugen Partner, Bharat Biotech, Announces Positive Immunogenicity and Safety Data From COVAXIN™ (BBV152), COVID-19 Candidate Vaccine, in Children 2 – 18 YEARS

 

Research, News, and Market Data on Ocugen

 

• COVAXIN™ whole virus inactivated vaccine generated broad antibody response comparable to those seen in a large phase 3 trial in adults 18+
  
• Adverse events were primarily mild, and no serious adverse events (SAEs) were noted
• Immunobridging trial demonstrated safety, reactogenicity and immunogenicity of COVAXIN™ in children from 2 years old and up
• No cases of myocarditis or blood clots were reported, as expected with inactivated vaccines

MALVERN, Pa., Dec. 30, 2021 (GLOBE NEWSWIRE) — Ocugen, Inc. (Nasdaq: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing novel therapeutics and vaccines, announced today that its partner, Bharat Biotech, posted results from a Phase 2/3 trial conducted in India of candidate vaccine, COVAXIN™ (BBV152), in children, aged 2 – 18 years, demonstrating a robust neutralizing antibody response and favorable safety profile on the pre-print server, medRXiv. Using a two-dose regimen administered 28-days apart, antibody responses in subjects were comparable to adult data from a previous phase 3 study. Those results demonstrated a greater than 93% reduction in severe disease. These pediatric data were the basis of Ocugen’s pediatric Emergency Use Authorization (EUA) submission in the United States for children 2-18 on November 5, 2021.

“These data represent the first positive vaccine data in children as young as two years of age and come at an important time as COVID-19 infections are seeing a spike across the globe,” said Shankar Musunuri, PhD, MBA, Chairman of the Board, Chief Executive Officer and Co-Founder of Ocugen. “The results suggest that COVAXIN™, which is made on the same platform used in traditional polio pediatric vaccines for decades, when administered in a two-dose series to children between 2-18, may offer an option that is safe while delivering a robust immune response.”

In the study, immunogenicity against key COVID-19 proteins was measured using geometric mean titer (GMT), a test that measures the amount of antibodies in the blood in response to the presence of the virus. GMT was measured across three age groups and demonstrated strong immune response after the second dose. There were no serious adverse events such as myocarditis, pericarditis, or blood clots, reported in any of the three age groups.

This immunobridging trial was intended to determine if COVAXIN™ generates the same protective immunity in children as it does in adults. To be considered equivalent, pediatric participants needed to achieve comparable GMT’s to those generated by adults in a large phase 3 clinical trial. At Day 56, across all ages, the mean SARS-CoV-2 antibody GMT was more than 30% higher in children aged 2-18 than the mean GMT from Phase 3 clinical trial involving 25,800 adult subjects aged 18+. These data demonstrate children 2-18 generate antibody response comparable to adults fully vaccinated with COVAXIN^™ (BBV152).

The study also showed increased antibody titers against 3 viral antigens (Spike – S1, Receptor Binding Domain – RBD and Nucleocapsid – NP) providing a broad immune response to COVID-19. In each case a more than 4-fold increase in neutralizing antibody titers versus baseline (titers measured in the same participants before being vaccinated) was seen in all age groups after the second dose.

Participants were not reported to have experienced any severe adverse events. The majority of adverse events noted (49%, n=374) were mild (e.g., injection site pain) and a small proportion (19%, n=6) were moderate (e.g., fever, headache). Mild to moderate adverse events resolved within 24 hours.

The data were also submitted to the Drugs Controller General of India (DCGI) to support a pediatric indication as recommended by an independent committee, known as the Subject Expert Committee (SEC), that advises the Indian regulatory agency. Bharat Biotech intends to submit data from the full Phase 3 trial to a peer-reviewed scientific publication soon.

About the Phase 2/3 Trial in Children (BBIL/BBV152/2021)
The seven-month Phase 2/3 open label, multicenter, study enrolled 525 children ages 2 years to 18 from 6 clinical trial sites across India. Conducted between May and July 2021, it was designed to evaluate the safety, reactogenicity and immunogenicity of COVAXIN™ (BBV152) on a two-dose schedule (28 days apart) in three age groups: ages 12-18, ages 6-12, and ages 2-6. Children with prior infection of SARS-CoV-2 were not excluded from the trial.

About COVAXIN™
COVAXIN™ (BBV152) is an investigational vaccine candidate product in the U.S. It was developed by Bharat Biotech in collaboration with the Indian Council of Medical Research (ICMR) – National Institute of Virology (NIV). COVAXIN™ (BBV152) is a highly purified and inactivated vaccine that is manufactured using a vero cell manufacturing platform.

With more than 125 million doses having been administered to adults outside the U.S., COVAXIN™ (BBV152) is currently authorized under emergency use in 17 countries, and applications for emergency use authorization are pending in more than 60 other countries. The World Health Organization (WHO) recently added COVAXIN™ (BBV152) to its list of vaccines authorized for emergency use. And, as many as 110 countries have agreed to mutual recognition of Covid-19 vaccination certificates with India that includes vaccination using COVAXIN™ (BBV152). The trade name, COVAXIN™, has not been evaluated by the FDA.

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. and Canadian markets. For more information, please visit www.ocugen.com.

About Bharat Biotech 
Bharat Biotech has established an excellent track record of innovation with more than 145 global patents, a wide product portfolio of more than 16 vaccines, 4 bio-therapeutics, registrations in more than 123 countries, and the World Health Organization (WHO) Pre-qualifications. Located in Genome Valley in Hyderabad, India, a hub for the global biotech industry, Bharat Biotech has built a world-class vaccine & bio-therapeutics, research & product development, Bio-Safety Level 3 manufacturing, and vaccine supply and distribution. 

Having delivered more than 4 billion doses of vaccines worldwide, Bharat Biotech continues to lead innovation and has developed vaccines for influenza H1N1, Rotavirus, Japanese Encephalitis, Rabies, Chikungunya, Zika, and the world’s first tetanus-toxoid conjugated vaccine for Typhoid. Bharat’s commitment to global social innovation programs and public-private partnerships resulted in introducing path-breaking WHO pre-qualified vaccines BIOPOLIO®, ROTAVAC®, and Typbar TCV® combatting polio, rotavirus, typhoid infections, respectively. The acquisition of the rabies vaccine facility, Chiron Behring, from GlaxoSmithKline (GSK) has positioned Bharat Biotech as the world’s largest rabies vaccine manufacturer. To learn more about Bharat Biotech, visit www.bharatbiotech.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, including statements about data published on the preprint server, medRxiv, by Bharat Biotech, which found that COVAXIN™ generated a potentially robust neutralizing antibody response and favorable safety profile in its Phase 2/3 study in pediatric patients, and the potential for this data to support our application to the U.S. Food and Drug Administration (FDA) for emergency use authorization (EUA) of COVAXIN™ in pediatric patients. 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; the risk that we may not resolve the current clinical hold on COVAXIN™ in the near term or at all, or that the FDA could make other decisions that adversely impact our ability to advance the development of COVAXIN™ in the United States, and the implications that this clinical hold may have for our request for emergency use authorization for COVAXIN for pediatric use, including the timing and scope of any such authorization; risks associated with preliminary and interim data, including the possibility of unfavorable new clinical trial data and further analyses of existing clinical trial data; the risk that the results of in-vitro studies will not be duplicated in human clinical trials; 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 data and results from the preclinical and clinical studies of COVAXIN™, which have been conducted by Bharat Biotech in India, will be accepted by the FDA or otherwise sufficient to support our EUA submission; the size, scope, timing and outcome of any additional trials or studies that we may be required to conduct to support an EUA or biologics license application (BLA); any additional chemistry, manufacturing, and controls information that we may be required to submit to the FDA; whether and when a BLA for COVAXIN™ will be submitted to or approved by the FDA; whether developments with respect to the COVID-19 pandemic will affect the regulatory pathway available for vaccines in the United States, Canada or other jurisdictions; market demand for COVAXIN™ in the United States or Canada; decisions by the FDA or Health Canada impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of COVAXIN™ in the United States or Canada, 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.

Ocugen Contact:  Ken Inchausti Head, Investor Relations & Communications [email protected] Please submit investor-related inquiries to: [email protected]