Cocrystal Pharma Receives Pre-IND Responses from the FDA on Oral CC-42344 for Treating Influenza A

Rhea-AI Summary

Cocrystal Pharma, Inc. receives positive Pre-IND feedback from the FDA for CC-42344 oral treatment for influenza A, setting the stage for Phase 2b trial. The company is progressing well with ongoing Phase 2a study in the UK and preparing for Phase 1 study in Australia. CC-42344 shows promising antiviral activity against various influenza strains and exhibits favorable safety profiles.

Medical Research Analyst

The feedback from the FDA on Cocrystal Pharma's Pre-IND submission for CC-42344 is a significant step forward for the company's clinical development pipeline. The Pre-IND process is a critical juncture in drug development, providing a forum for dialogue between the FDA and the sponsor regarding the drug's proposed testing pathway. The FDA's guidance on clinical trial design, drug manufacturing and necessary nonclinical studies is instrumental in optimizing the chances of a successful IND submission and subsequent trial phases.

From a medical research perspective, the progression of CC-42344 into Phase 2b trials, following what appears to be a successful Phase 1 study in Australia, indicates a promising safety and tolerability profile. The drug's mechanism of action, targeting a conserved site of the influenza A polymerase complex, suggests potential as a broad-spectrum antiviral. This is particularly relevant given the resistance observed in current treatments like Tamiflu and Xofluza. The in vitro efficacy against a range of influenza A strains could position CC-42344 as a significant player in flu treatment, assuming clinical trials confirm these findings.

Pharmacologist

CC-42344's inhibition of the PB2 site in the polymerase complex of influenza A represents an innovative approach within antiviral pharmacology. The PB2 site is essential for the virus's replication and its high conservation across strains could make CC-42344 less susceptible to resistance development compared to drugs that target less conserved viral components. Preclinical data suggesting superior lung exposure and efficacy in influenza-infected human lung epithelia are promising for the inhaled formulation's potential as both a treatment and prophylaxis.

However, the transition from preclinical to clinical efficacy is not always straightforward due to the complexity of human biology. The favorable pharmacokinetics observed in preclinical studies must be replicated in diverse human populations to ensure the drug's effectiveness and safety. The implications for stakeholders include the potential for a new, effective treatment option for influenza A, which could have significant market implications given the global burden of flu. Nevertheless, the long-term impact will depend on the outcomes of the ongoing and planned clinical trials.

Financial Analyst

Investor interest in Cocrystal Pharma is likely to be heightened following the FDA's Pre-IND feedback, as regulatory clarity can reduce development risk and potentially shorten the time to market for CC-42344. The company's stock market performance may reflect this reduced risk profile, but investors should also consider the capital requirements and timeline associated with advancing CC-42344 through clinical trials. The potential market for an effective influenza A treatment is substantial, but the drug's commercial success will depend on its clinical trial results, pricing, insurance coverage and competitive landscape.

Moreover, Cocrystal's proprietary drug discovery platform technology could be a key value driver, suggesting a pipeline of future antiviral candidates. The mention of anticipated topline results from the Phase 2a challenge study could serve as a near-term catalyst for the company's valuation, contingent upon positive outcomes. In the long-term, success in the clinic could position Cocrystal as an acquisition target for larger pharmaceutical companies seeking to expand their antiviral portfolios.

Feedback provides greater clarity on regulatory requirements for planned Phase 2b trial

BOTHELL, Wash., March 19, 2024 (GLOBE NEWSWIRE) -- Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces it has received Pre-Investigational New Drug (Pre-IND) feedback from the U.S. Food and Drug Administration (FDA) regarding CC-42344 as a potential oral treatment for pandemic and seasonal influenza A. A Pre-IND review provides the opportunity to obtain FDA guidance and clarification on critical steps such as the proposed clinical trial design, as well as clinical drug manufacturing and nonclinical studies deemed necessary before filing the trial design. The feedback was provided in a written response to a Pre-IND package and questions submitted by the Company in January 2024.

“We value the FDA guidance as we prepare to file the IND for our Phase 2b trial and open enrollment of patients in the U.S.,” said Sam Lee, Ph.D., Cocrystal’s President and co-CEO. “This is an important milestone that provides greater clarity on the regulatory requirements and our planned oral CC-42344 clinical program.”

A Phase 2a challenge study of oral CC-42344 is underway in the United Kingdom to evaluate safety, and viral and clinical measures in healthy volunteers who are infected with the influenza A virus. The Company expects to report topline results from this study in the second half of this year.

In addition, preparations are underway to begin a Phase 1 study in Australia with the Company’s inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis for those exposed to the virus. Recent preclinical data showed that inhaled CC-42344 exhibited highly effective delivery into the lung, superior lung exposure, efficacy in influenza-infected human lung epithelia, and a favorable safety profile.

CC-42344 Influenza A PB2 Inhibitor
CC-42344 is a novel, broad-spectrum, investigational antiviral candidate for the treatment of pandemic and seasonal influenza A. CC-42344 inhibits the first step in the viral replication process of influenza A by binding to a highly conserved PB2 site of the polymerase complex that is essential to replication. In vitro testing with CC-42344 showed excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to Tamiflu^® and Xofluza^®, while also demonstrating favorable pharmacokinetic and safety profiles. In addition, oral CC-42344 demonstrated favorable safety and tolerability results in a Phase 1 study in Australia. This antiviral candidate was discovered using the Company’s proprietary structure-based drug discovery platform technology.

About Seasonal Influenza
Each year there are approximately 1 billion cases of seasonal influenza worldwide, with 3-5 million severe illnesses and up to 650,000 deaths, according to the World Health Organization. On average about 8% of the U.S. population contracts influenza each season. In addition to the health risk, influenza is responsible for approximately $10.4 billion in direct costs for hospitalizations and outpatient visits for adults in the U.S. annually.

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), noroviruses and hepatitis C viruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the expected results of the Phase 2a trial for CC-42344 for the oral treatment of influenza A in the second half of 2024, efforts in preparation for an anticipated Phase 2b trial for oral treatment of influenza A following the Phase 2a trial, efforts to begin a Phase 1 study in Australia for an inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis, and the potential efficacy and clinical benefits of, and market for, such product candidates. 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, risks relating to our ability to proceed with the studies including recruiting volunteers and procuring materials for such studies by our clinical research organizations and vendors, the results of such studies and our ability to obtain FDA approval to initiate the Phase 2b study. 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, 2022. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

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

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

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What is the purpose of the Pre-IND feedback received by Cocrystal Pharma, Inc. from the FDA?

The Pre-IND feedback provides guidance and clarification on critical steps such as clinical trial design, drug manufacturing, and nonclinical studies for CC-42344 as a potential oral treatment for influenza A.

What is the current status of Cocrystal Pharma's Phase 2a study for CC-42344 in the UK?

The Phase 2a challenge study of oral CC-42344 is underway in the UK to evaluate safety, viral, and clinical measures in healthy volunteers infected with influenza A.

What are the key features of CC-42344 as an antiviral candidate for influenza A treatment?

CC-42344 is a novel, broad-spectrum antiviral candidate that inhibits the first step in the viral replication process of influenza A by binding to a highly conserved PB2 site. It shows excellent antiviral activity against various influenza strains and favorable safety profiles.

How was CC-42344 discovered?

CC-42344 was discovered using Cocrystal Pharma's proprietary structure-based drug discovery platform technology.

What are the upcoming plans for CC-42344 clinical studies in Australia?

Preparations are underway to begin a Phase 1 study in Australia with the Company’s inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis for those exposed to the virus.