Tag: Cocrystal Pharma

Research News and Market Data on COCP

January 08, 2025

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• Data show favorable safety and tolerability with dosing up to 800 mg for 10 days
• Plans to initiate human challenge study in 2025 in norovirus-infected subjects

BOTHELL, Wash., Jan. 08, 2025 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces favorable safety and tolerability results at dosing up to 800 mg per day for 10 consecutive days from the multiple-ascending dose (MAD) portion of the ongoing Phase 1 study with its oral protease inhibitor CDI-988, the first pan-viral drug candidate in development as an orally administered treatment of norovirus and coronavirus infections. The Company also announces that an additional cohort with a higher dose of 1,200 mg and a shorter treatment duration of five consecutive days will be conducted to further assess CDI-988’s safety, tolerability and pharmacokinetics.

“Norovirus outbreaks are surging across the U.S. as well as on cruise ships with nearly 900 passengers sickened from gastrointestinal disease in December alone,” said Sam Lee, Ph.D., Cocrystal’s President and co-CEO. “There are currently no approved antivirals or vaccines for norovirus and the ability to curtail outbreaks is limited, making norovirus infection a significant unmet need. We are pleased to report positive Phase 1 results with these data demonstrating that CDI-988 had a favorable safety profile and was well-tolerated, supporting further clinical advancement of this potentially first effective norovirus antiviral.

“CDI-988 is our first pan-viral protease inhibitor with the potential to be used for prophylaxis and treatment for norovirus and coronavirus infections. We look forward to making a significant impact on norovirus outbreaks with this development candidate,” he added. “We expect to begin enrollment in the Phase 1 study higher-dose cohort in the current quarter and to initiate a human challenge study in norovirus-infected subjects later in 2025.”

CDI-988 is a potent, oral, broad-spectrum antiviral inhibitor of a highly conserved region in the active site of noroviruses, coronaviruses and other 3CL viral proteases. It was specifically designed and developed using Cocrystal’s proprietary structure-based drug discovery platform technology. CDI-988 has shown pan-viral activity against multiple norovirus strains, including the genogroup II, genotype 4 (GII.4) viruses that cause severe vomiting, diarrhea and stomach pain.

About Noroviruses

Human noroviruses are highly contagious, constantly evolving, extremely stable in the environment and associated with debilitating illness. Outbreaks occur most commonly in semi-closed communities such as nursing homes, hospitals, cruise ships, schools, disaster relief sites and military settings. Symptoms include vomiting and diarrhea, with or without nausea and abdominal cramps. Norovirus infection can be much more severe and prolonged in specific risk groups including infants, children, the elderly and people with immunodeficiency.

According to the Centers for Disease Control and Prevention (CDC), an estimated 685 million cases and an estimated 200,000 deaths are attributed to norovirus each year worldwide, with an estimated societal cost of approximately $60 billion. In the U.S. alone, noroviruses are responsible for an estimated 21 million cases of acute gastroenteritis annually, including 109,000 hospitalizations, 465,000 emergency department visits and nearly 900 deaths. The estimated annual burden of noroviruses to the U.S. is $10.6 billion, according to the National Institutes of Health (NIH).

Structure-Based Platform Technology

Cocrystal’s proprietary structural biology, along with its expertise in enzymology and medicinal chemistry, enable its development of novel antiviral agents. The Company’s platform provides a three-dimensional structure of inhibitor complexes at near-atomic resolution, providing immediate insight to guide Structure Activity Relationships. This helps to identify novel binding sites and allows for a rapid turnaround of structural information through highly automated X-ray data processing and refinement. The goal of this technology is to facilitate the development of best-in-class antiviral therapies that have fast onset of action and/or shortened treatment time, are safe, well tolerated and easy to administer, are effective against all viral subtypes that cause disease and have a high barrier to viral resistance.

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 Cocrystal’s plans to initiate a human challenge study in 2025 and the expansion of the current ongoing clinical trial with a higher dosage during the current quarter. 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 obtain regulatory authority for and proceed with clinical trials including the recruiting of volunteers for such studies by our clinical research organizations and vendors, the results of such studies, our collaboration partners’ technology and software performing as expected, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes, and potential development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, and potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. 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, 2023. 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:
Alliance Advisors IR
Jody Cain
310-691-7100
jcain@allianceadvisors.com

# # #

Source: Cocrystal Pharma, Inc.

Released January 8, 2025

Research News and Market Data on COCP

December 31, 2024

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• Data support favorable safety and tolerability profile with no serious adverse events (SAEs) or study-related drug discontinuations
• Enrollment to be extended due to low influenza infection among challenged participants; virology results are uninterpretable

BOTHELL, Wash., Dec. 31, 2024 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces plans to extend enrollment in the Phase 2a human challenge study with its investigational, broad-spectrum, oral influenza PB2 inhibitor CDI-42344 due to unexpectedly low influenza infection among study participants who were challenged with a H3N2 viral strain. This randomized, double-blind, placebo-controlled Phase 2a study is evaluating the safety, tolerability, pharmacokinetics (PK), antiviral activity and clinical measurements of CC-42344 at a single site in the United Kingdom.

CC-42344 is a drug candidate in development as an oral treatment for pandemic avian and seasonal influenza A infections. In December 2023, Cocrystal Pharma announced enrollment of the first patient in this study and in May 2024, the Company announced full enrollment of 78 subjects.

“While CC-42344 showed a favorable safety and tolerability profile, we’re disappointed by the low infectivity rate of the challenge influenza strain used in this study. The establishment of robust influenza infection in healthy, uninfected study subjects is critical to determine clinical endpoints for evaluating antiviral molecules. The low infectivity obtained in this study hindered antiviral data analysis,” said Sam Lee, Ph.D., Cocrystal’s President and co-CEO.

“We remain optimistic about CC-42344 due to its unique mechanism of action with a high barrier to developing resistance, which could render it a best-in-class antiviral treatment for pandemic and seasonal influenza infections. We are also encouraged by CC-42344’s favorable safety and tolerability profile from the Phase 2a study to date, with no SAEs and no drug-related discontinuations by study participants.

“We are working with the clinical research organization to prepare a protocol amendment for approval by the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) in order to extend enrollment in this study, and to ensure necessary infection rates among enrolled study subjects,” he added.

About CC-42344

CC-42344 is a new class of antiviral treatment designed to effectively block an essential step in the viral replication and transcription of pandemic and seasonal influenza A and was discovered using the Company’s proprietary structure-based drug discovery platform technology. CC-42344 showed excellent in vitro antiviral activity against pandemic and seasonal influenza A strains, as well as strains that are resistant to Tamiflu^® and Xofluza^®. In late 2022, Cocrystal reported favorable safety and tolerability results from a Phase 1 study in healthy subjects conducted in Australia. The Company initiated the Phase 2a human challenge study in December 2024 following authorization from the MHRA. In June 2024, the Company reported in vitro studies demonstrating that CC-42344 inhibited the activity of the PB2 protein in the new highly pathogenic avian influenza A (H5N1) PB2 protein recently identified in humans.

About Influenza A

Influenza is a major global health threat that may become more challenging to treat due to the emergence of highly pathogenic avian influenza viruses and resistance to approved influenza antivirals. Each year there are approximately 1 billion cases of seasonal influenza worldwide, 3-5 million severe illnesses and up to 650,000 deaths. On average, about 8% of the U.S. population contracts influenza each season. In addition to the health risk, influenza is responsible for an estimated $11.2 billion in direct and indirect costs in the U.S. annually.

Structure-Based Platform Technology

Cocrystal’s proprietary structural biology, along with its expertise in enzymology and medicinal chemistry, enable its development of novel antiviral agents. The Company’s platform provides a three-dimensional structure of inhibitor complexes at near-atomic resolution, providing immediate insight to guide Structure Activity Relationships. This helps to identify novel binding sites and allows for a rapid turnaround of structural information through highly automated X-ray data processing and refinement. The goal of this technology is to facilitate the development of best-in-class antiviral therapies that have fast onset of action and/or shortened treatment time, are safe, well tolerated and easy to administer, are effective against all viral subtypes that cause disease and have a high barrier to viral resistance.

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 extension of enrollments, regulatory approval and achieving the necessary infection rate. 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 obtain regulatory authority for and proceed with clinical trials including the recruiting of volunteers for such studies by our clinical research organizations and vendors, the results of such studies, our collaboration partners’ technology and software performing as expected, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes, and potential development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, and potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. 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, 2023. 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:
Alliance Advisors IR
Jody Cain
310-691-7100
jcain@allianceadvisors.com

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

Research News and Market Data on COCP

November 13, 2024

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BOTHELL, Wash., Nov. 13, 2024 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) reports financial results for the three and nine months ended September 30, 2024, and provides updates on its antiviral product pipeline, upcoming milestones and business activities.

“The coming months are critically important to Cocrystal as we expect to report topline results from two ongoing clinical studies with our best-in-class antiviral candidates in major medical indications,” said Sam Lee, Ph.D., President and co-CEO of Cocrystal. “In the Phase 2a influenza A challenge study with our oral PB2 inhibitor CC-42344, we expect to report topline results before year end. Earlier this year, we received feedback from the U.S. Food and Drug Administration (FDA) on a Pre-IND package that improves our clarity on the regulatory path and requirements for the late-stage influenza A clinical study we plan to conduct in the U.S.

“The multiple-ascending dose portion in our Phase 1 pan-norovirus/pan-coronavirus study with oral protease inhibitor CDI-988 is underway and we are on track to report topline results in late 2024 or early 2025,” he added. “We view the development of an effective antiviral for norovirus as a significant opportunity for Cocrystal. There is no approved vaccine or antiviral for norovirus, which is highly contagious and the most common cause of acute gastroenteritis. In in vitro studies, CDI-988 exhibited pan-viral activity against multiple norovirus strains, including the strain that is responsible for major outbreaks.”

“Our significant clinical progress so far this year puts us on track for an active 2025,” said James Martin, CFO and co-CEO of Cocrystal. “I’m pleased to report that based on our currently projected expenditures and our cost-efficient business model, we expect our cash will be sufficient to fund the advancement of our planned development programs through the coming 12 months.”

Antiviral Product Pipeline Overview

We apply our proprietary structure-based drug discovery platform technology for developing broad-spectrum antivirals that inhibit viral replication. By designing and selecting antiviral drug candidates that target the highly conserved regions of the viral enzymes, we seek to develop drugs that are effective against the virus and mutations of the virus, and also reduce off-target interactions that may cause undesirable side effects. Our drug discovery process differs from traditional, empirical medicinal chemistry approaches that often require iterative high-throughput compound screening and lengthy hit-to-lead processes.

Influenza Programs

Influenza is a major global health threat that may become more challenging to treat due to the emergence of highly pathogenic avian influenza viruses and resistance to approved influenza antivirals. Each year there are approximately 1 billion cases of seasonal influenza worldwide, 3-5 million severe illnesses and up to 650,000 deaths. On average, about 8% of the U.S. population contracts influenza each season. In addition to the health risk, influenza is responsible for an estimated $11.2 billion in direct and indirect costs in the U.S. annually.

• Oral CC-42344 for the treatment of pandemic and seasonal Influenza A infections
  • Our novel PB2 inhibitor CC-42344 showed excellent in vitro antiviral activity against pandemic and seasonal influenza A strains, as well as strains that are resistant to Tamiflu^® and Xofluza^®.
  • In December 2022 we reported favorable safety and tolerability results from the oral CC-42344 Phase 1 study.
  • In December 2023 we began a randomized, double-blind, placebo-controlled Phase 2a human challenge study to evaluate the safety, tolerability, viral and clinical measurements of CC-42344 in influenza A-infected subjects in the United Kingdom, following authorization from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
  • In May 2024 we completed enrollment in the Phase 2a human challenge study.
  • In June 2024 we reported that in vitro studies demonstrated CC-42344 inhibits the activity of the new highly pathogenic avian influenza A (H5N1) PB2 protein recently identified in humans exposed to infected dairy cows.
  • We expect to report topline results from the Phase 2a human challenge study by yearend and plan to file an IND application in 2025 to conduct a late-stage study in the U.S.
    
• Inhaled CC-42344 for the therapeutic and prophylactic treatment of pandemic and seasonal Influenza A infections
  
  • Our preclinical testing showed superior pulmonary pharmacology with CC-42344 including high exposure to drug and a long half-life.
  • We completed CC-42344 inhalation formulation development.
  • We initiated GLP toxicology studies.
• Influenza A/B Program
  • Our work to develop a preclinical lead of novel influenza replication inhibitors is underway.

Norovirus Program

Norovirus is a highly contagious infection and is the most common cause of acute gastroenteritis. Worldwide, norovirus causes about one out of five cases of acute gastroenteritis that leads to diarrhea and vomiting. An estimated 685 million cases and an estimated 50,000 child deaths are attributed to norovirus each year worldwide, with an estimated societal cost of $60 billion. By targeting viral replication, we believe it is possible to develop an effective treatment and/or short-term prophylactic for closed environments for all genogroups of norovirus.

• Oral pan-viral protease inhibitor CDI-988 for the treatment of norovirus and coronavirus infections
  • Our novel broad-spectrum protease inhibitor CDI-988 is being evaluated as a potential oral treatment for noroviruses and coronaviruses.
  • CDI-988 has shown in vitro pan-viral activity against multiple norovirus strains, including the genogroup II, genotype 4 (GII.4) norovirus strain that is responsible for major norovirus outbreaks.
  • In May 2023 we announced approval of our application to the Australian regulatory agency for a randomized, double-blind, placebo-controlled Phase 1 study to evaluate the safety, tolerability and pharmacokinetics (PK) of oral CDI-988 in healthy volunteers.
  • In August 2023 we announced our selection of CDI-988 as our lead for the oral treatment for norovirus, in addition to coronavirus.
  • In September 2023 we began dosing subjects in a first-in-human study in healthy volunteers with oral CDI-988.
  • In July 2024 we reported favorable safety and tolerability results from the single-ascending dose cohort in the Phase 1 study.
  • In September 2024 we advanced CDI-988 into the multiple-ascending dose cohort of the Phase 1 study.
  • We expect to report topline results from the CDI-988 Phase 1 study in late 2024 or early 2025.

COVID-19 and Other Coronavirus Programs

By targeting viral replication enzymes and proteases, we believe it is possible to develop effective treatments for all diseases caused by coronaviruses including COVID-19 and its variants, Severe Acute Respiratory Syndrome (SARS) and Middle East Respiratory Syndrome (MERS). CDI-988 showed potent in vitro pan-viral activity against common human coronaviruses, rhinoviruses and respiratory enteroviruses, as well as against noroviruses. The global COVID-19 therapeutics market is estimated to exceed $16 billion by the end of 2031.

• Oral pan-viral protease inhibitor CDI-988 for the treatment of coronaviruses and noroviruses
  • CDI-988 exhibited superior in vitro potency against SARS-CoV-2 and demonstrated a favorable safety profile and PK properties.
  • In September 2023 we dosed the first subject in our dual pan-norovirus/pan-coronavirus oral CDI-988 study, which is expected to serve as a Phase 1 study for both indications.
  • In July 2024 we reported favorable safety and tolerability results from the single-ascending dose cohort in the Phase 1 study.
  • In September 2024 we advanced CDI-988 into the multiple-ascending dose cohort of the Phase 1 study.
  • We expect to report topline results from the CDI-988 Phase 1 study in late 2024 or early 2025.

Third Quarter Financial Results

Research and development (R&D) expenses for the third quarter of 2024 were $3.2 million, compared with $4.2 million for the third quarter of 2023, with the decrease primarily due to lower clinical study expenses. General and administrative (G&A) expenses for the third quarters of 2024 and 2023 remained relatively stable at $1.8 million.

The net loss for the third quarter of 2024 was $4.9 million, or $0.49 per share, compared with a net loss for the third quarter of 2023 of $4.2 million, or $0.41 per share, that included a $1.6 million payment to the Company in 2023 for a legal settlement.

Nine Month Financial Results

R&D expenses for the first nine months of 2024 were $10.5 million, compared with $10.9 million for the first nine months of 2023. G&A expenses for the first nine months of 2024 were $4.1 million, compared with $4.6 million for the first nine months of 2023.

The net loss for the first nine months of 2024 was $14.2 million, or $1.40 per share, compared with a net loss for the first nine months of 2023 of $13.5 million, or $1.43 per share.

Cocrystal reported unrestricted cash as of September 30, 2024 of $13.0 million, compared with $26.4 million as of December 31, 2023. Net cash used in operating activities for the first nine months of 2024 was $13.3 million, compared with $11.3 million for the first nine months of 2023. The Company had working capital of $12.3 million and 10.2 million common shares outstanding as of September 30, 2024.

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 our plans for the future development of preclinical and clinical drug candidates, our expectations regarding future characteristics of the product candidates we develop, the expected time of achieving certain value-driving milestones in our programs, including preparation, commencement and advancement of clinical studies for certain product candidates in 2024 and 2025, the viability and efficacy of potential treatments for diseases our product candidates are designed to treat, expectations for the markets for certain therapeutics, our ability to execute our clinical and regulatory goals and deploy regulatory guidance towards future studies, and the expected sufficiency of our cash balance to advance our programs and fund our planned operations. 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 and uncertainties arising from future inflation, potential future increases in interest rates uncertainty in the financial markets, the possibility of a recession, and geopolitical conflict including in Ukraine and Israel on our Company, our collaboration partners, and on the U.S., UK, Australia and global economies, including manufacturing and research delays arising from raw materials and labor shortages, supply chain disruptions and other business interruptions on our ability to proceed with studies as well as similar problems with our vendors and our current and any future clinical research organization (CROs) and contract manufacturing organizations (CMOs), the ability of our CROs to recruit volunteers for, and to proceed with, clinical studies, our and our collaboration partners’ technology and software performing as expected, financial difficulties experienced by certain partners, the results of any current and future preclinical and clinical studies, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes including potential downward pressure on government spending on healthcare in the wake of the recent presidential election in the U.S., the impact of the recent U.S. presidential election on regulation affecting the FDA and other healthcare agencies and potential staffing issues, potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. 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, 2023. 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:
Alliance Advisors IR
Jody Cain
310-691-7100
jcain@allianceadvisors.com

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

Financial Tables

Source: Cocrystal Pharma, Inc.

Released November 13, 2024

Research News and Market Data on COCP

JUNE 20, 2024

BOTHELL, Wash., June 20, 2024 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc.’s (Nasdaq: COCP) novel, broad-spectrum antiviral CC-42344 inhibits activity in the highly pathogenic avian influenza A (H5N1) PB2 protein recently identified in infected dairy cattle, according to recently completed in vitro studies. CC-42344 is a new class of antiviral drugs designed to block essential steps in the replication and transcription of the influenza A virus.

Cocrystal is conducting an influenza A Phase 2a clinical study with orally administered CC-42344 and expects to report topline results in the second half of 2024. This study is evaluating the safety, tolerability, antiviral and clinical benefits in influenza A infected subjects. In late 2022 Cocrystal reported favorable safety and tolerability results from a Phase 1 study in healthy volunteers.

Cocrystal demonstrated the potential efficacy of CC-42344 against the new avian flu strain with the recently published genome sequence for H5N1. Using its proprietary structure-based platform, Cocrystal created a high-resolution crystal structure of this avian PB2 protein and confirmed that CC-42344 binds to its highly conserved PB2 region. The in vitro data were generated testing CC-42344 against the avian H5N1 PB2 protein and further support CC-42344’s activity similar to that of Cocrystal’s data with pandemic and seasonal influenza A.

“The findings validate our broad-spectrum approach to the treatment and prevention of pandemic flu. This is important as there are no specific FDA-approved vaccines to prevent infections by this virus in humans,” said Sam Lee, PhD, President and co-CEO of Cocrystal. “These findings support our previously reported preclinical data showing that CC-42344 is highly active against seasonal and pandemic influenza A strains, including emerging mutations. CC-42344 is an inhibitor compound providing a unique mechanism of action with a high barrier to resistance.”

“Recent CDC reported avian flu outbreaks in the U.S., which include the first cases of humans exposed to infected dairy cows, are concerning,” said James Martin, CFO and co-CEO of Cocrystal. “The CDC reported three additional cases of avian influenza infection from exposure to dairy cows in early June and avian flu is now confirmed in more than 100 dairy herds in 12 U.S. states.”

About Avian Influenza A H5N1
Avian influenza A H5N1 was reported in 889 cases and caused 463 deaths in 23 countries between 2003 and April 2024, according to the World Health Organization (WHO). On April 1, 2024, the CDC reported a case of highly pathogenic avian influenza A H5N1 in a farmworker in Texas during a multistate outbreak of avian influenza in dairy cows. Two more cases were subsequently reported in farmworkers in Michigan.

The CDC analyzed sera (blood) collected from people of all ages in all 10 Health & Human Services regions during the 2022-2023 and 2021-2022 flu seasons. These samples were challenged with H5N1 virus to see whether there was an antibody reaction. Data from this study suggest that there is extremely low to no population immunity to clade 2.3.4.4b A (H5N1) viruses in the U.S. Antibody levels remained low regardless of whether or not the participants received a seasonal flu vaccination, meaning that seasonal flu vaccination did not produce antibodies to H5N1 viruses. 

Cocrystal Pharma determined the high resolution X-ray crystal structure of the recent avian influenza A (H5N1) PB2 protein and confirmed activity of CC-42344 in vitro (NIH GeneBank ID:influenza A/Texas/37/2024(H5N1). The crystal structure of the avian influenza A (H5N1) PB2 protein showed new mutations located outside the PB2 active site. Subsequent studies showed that CC-42344 binds to the active site of the avian influenza A (H5N1) PB2 protein as previously demonstrated with the pandemic and seasonal influenza A PB2. Preliminary in vitro assays confirmed that CC-42344 exhibits high potency against the avian influenza A (H5N1) PB2 protein.

About CC-42344
CC-42344 is Cocrystal’s novel, broad-spectrum, antiviral investigational candidate for the treatment of pandemic and seasonal influenza A. CC-42344 inhibits the first step in influenza A’s viral replication by binding to a highly conserved PB2 site of the influenza polymerase complex that is essential to replication and was discovered using Cocrystal’s proprietary structure-based drug discovery platform technology.

Cocrystal is conducting a Phase 2a human challenge study in the United Kingdom to evaluate safety, viral and clinical measures of oral CC-42344 in healthy volunteers who are challenged with influenza A. CC-42344 was advanced into Phase 2a testing following favorable safety and tolerability results reported in a Phase 1 study in healthy volunteers conducted in Australia. In vitro testing showed CC-42344’s 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.

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 potential efficacy of CC-42344 against the avian influenza A H5N1 virus, the expected timing and results of the Phase 2a trial for CC-42344 for the oral treatment of influenza A in 2024, and the potential market for such product candidate. 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 obtain regulatory authority for and proceed with clinical trials including recruiting volunteers and procuring materials for such studies by our clinical research organizations and vendors, the results of such studies, our and our collaboration partners’ technology and software performing as expected, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes, and potential development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. 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, 2023. 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

# # #

Source: Cocrystal Pharma, Inc.

Released June 20, 2024