Post by icemandios on Mar 28, 2024 12:48:11 GMT
Cocrystal Pharma Reports 2023 Financial Results And Provides Updates On Its Antiviral Drug-Development Programs
FDA feedback following the Company’s submission of a Pre-IND briefing package improves clarity on regulatory requirements for Phase 2b influenza A clinical trial with oral CC-42344, a broad-spectrum PB2 inhibitor
Topline data expected in 2024 from Phase 2a influenza A human challenge study and Phase 1 study with oral CDI-988, the first potential dual coronavirus-norovirus oral antiviral
Initiation of Phase 1 study expected in 2024 with inhaled CC-42344, a potential influenza treatment and post-exposure prophylactic
“We are highly encouraged by the FDA’s feedback to our Pre-Investigational New Drug (Pre-IND) package, which provides greater clarity on the regulatory requirements for a planned Phase 2b clinical trial with our novel broad-spectrum oral PB2 inhibitor CC-42344 for pandemic and seasonal influenza A,” said Sam Lee, Ph.D., President and co-CEO of Cocrystal. “This is a major step in the clinical and regulatory process for this program. We plan to file an IND for late-stage clinical development of oral CC-42344 that includes data from our ongoing Phase 2a human challenge study, which are expected later this year.
“During 2024 we also expect to initiate a Phase 1 study in healthy volunteers with inhaled CC-42344 as a potential prophylactic and therapeutic for influenza A. Our inhaled formulation of CC-42344 has shown the ability to directly target influenza-infected respiratory epithelial cells in lung, allowing for higher accumulation of drug in the pulmonary system and potentially producing a rapid clinical response while reducing potential systemic side effects,” he added. “Also during the coming year we expect topline data from the ongoing first-in-human study with our pan-coronavirus and pan-norovirus oral protease inhibitor CDI-988 , which is expected to serve as a Phase 1 study for both indications.”
“All our programs target high-value unmet indications with novel, broad-spectrum, best-in-class antiviral candidates whose design and development uses our proprietary structure-based drug discovery platform technology,” said James Martin, CFO and co-CEO. “I’m pleased to report that under our cost-efficient business model, we believe our cash position is sufficient to fund current operations including planned clinical studies beyond the next 12 months.”
Antiviral Product Pipeline Overview
We are developing therapeutics that inhibit the viral replication function of RNA viruses that cause acute and chronic diseases. Our drug-discovery process focuses on the highly conserved regions of the viral enzymes and inhibitor-enzyme interactions at the atomic level. By designing and selecting antiviral drug candidates that interrupt the viral replication process and have specific binding characteristics, we seek to develop drugs that are effective against the virus and mutations of the virus, and also have reduced 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 severe respiratory illness that is caused by the influenza A or B virus and results in disease outbreaks mainly during the winter months. Influenza is a major global health threat that may become more challenging to treat in the future 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, 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 .
Pandemic and Seasonal Influenza A
Our novel PB2 inhibitor CC-42344 has shown excellent in vitro antiviral activity against influenza A strains including pandemic and seasonal strains, as well as strains that are resistant to Tamiflu ® and Xofluza ® .
In March 2022 we initiated enrollment in a randomized, double-blind, dose-escalating Phase 1 study to evaluate the safety, tolerability and pharmacokinetics (PK) of oral CC-42344 in healthy adults.
In July 2022 we reported PK results from the single-ascending dose portion of the study that support once-daily dosing.
In December 2022 we reported favorable safety and tolerability results from the oral CC-42344 Phase 1 study.
In October 2023 we announced authorization from the United Kingdom Medicines and Healthcare Products Regulatory Agency to conduct a Phase 2a human challenge study.
In December 2023 we began treating influenza-infected subjects in the randomized, double-blind, placebo-controlled Phase 2a human challenge study to evaluate the safety, tolerability, viral and clinical measurements of influenza A infection in subjects treated with oral CC-42344.
In March 2024 we received feedback from the FDA on a Pre-IND package improving clarity on clinical trial design, drug manufacturing and nonclinical studies necessary to file a Phase 2b trial design.
Preclinical development is underway with inhaled CC-42344 as a potential therapeutic and post-exposure prophylaxis for influenza A. CC-42344 has exhibited superior pulmonary exposure in preclinical testing. We expect to begin a Phase 1 clinical study with inhaled CC-42344 in Australia in 2024.
Preclinical lead optimization of replication inhibitor antiviral candidates is underway.
By targeting viral replication enzymes and protease, we believe it is possible to develop effective treatments for all diseases caused by coronaviruses including COVID-19, Severe Acute Respiratory Syndrome (SARS) and Middle East Respiratory Syndrome (MERS). Our main SARS-CoV-2 protease inhibitors showed potent in vitro pan-viral activity against common human coronaviruses, rhinoviruses and respiratory enteroviruses that cause the common cold, as well as against noroviruses that can cause symptoms of acute gastroenteritis. Driven by the anticipated emergence of new COVID-19 variants, the global COVID-19 therapeutics market is estimated to exceed $16 billion by the end of 2031.
Oral Protease Inhibitor CDI-988
In October 2022 we announced the selection of CDI-988 as our lead candidate for development as a potential oral treatment for SARS-CoV-2. CDI-988 exhibited superior in vitro potency against SARS-CoV-2 with activity maintained against variants of concern, and demonstrated a safety profile and PK properties that support once-daily dosing.
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 PK of oral CDI-988 in healthy volunteers.
In August 2023 we announced the selection of CDI-988 as our lead oral candidate for norovirus, in addition to coronavirus.
In September 2023 we dosed the first subject in our dual norovirus-coronavirus oral CDI-988 study, which is expected to serve as a Phase 1 study for both indications.
We expect topline data from the Phase 1 study with CDI-988 in 2024.
Norovirus is a highly contagious infection and is the most common cause of acute gastroenteritis, accounting for nearly one in five cases . 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 $60 billion.
3CL inhibitor CDI-988 has shown pan-viral activity against multiple norovirus strains, including the genogroup II, genotype 4 (GII.4) norovirus strain that is responsible for major norovirus outbreaks. By targeting viral replication, we believe it is possible to develop an effective treatment or short-term prophylactic for closed environments for all genogroups of norovirus.
In August 2023 we announced our selection of the novel broad-spectrum oral 3CL protease inhibitor CDI-988 as our lead potential oral treatment for norovirus, in addition to coronavirus.
In September 2023 we began subject dosing in a first-in-human study in healthy volunteers in Australia. The randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability and PK of oral CDI-988 in healthy volunteers is expected to serve as a Phase 1 study for both indications.
We expect topline data from the Phase 1 study with CDI-988 in 2024.
Research and development (R&D) expenses for 2023 were $15.2 million, compared with $12.4 million for 2022. The increase was primarily due to advancing influenza candidate CC-42344 into a Phase 2a study and advancing the dual norovirus-coronavirus candidate CDI-988 into a Phase 1 study. General and administrative (G&A) expenses for 2023 were $6.0 million, compared with $5.7 million for 2022.
During 2023 the Company received $2.6 million related to litigation with an insurer, which included a $1.6 million refund from the registry of the United States Court of Appeals for the Third Circuit, reflecting the recovery of funds following a successful appeal, and $1.0 million in a settlement agreement with the insurer.
Interest income for 2023 was $640,000, compared with interest expense of $2,000 for 2022. The interest income in 2023 was related to interest earned from cash held in banks and deposits with the court registry, and the interest expense in 2022 was related to finance lease agreements.
The net loss for 2023 was $18.0 million, or $1.87 per share, compared with the net loss for 2022 of $38.8 million, or $4.77 per share, which included a $19.1 million non-cash impairment-loss of goodwill.
Cocrystal reported unrestricted cash as of December 31, 2023 of $26.4 million, compared with $37.1 million as of December 31, 2022. Net cash used in operating activities for 2023 was $14.7 million, compared with $21.4 million for 2022. The Company had working capital of $25.0 million and 10.2 million common shares outstanding as of December 31, 2023.
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 .