- 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
“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
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
- 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 theUnited 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.
- Influenza A/B Program
- Preclinical lead optimization of replication inhibitor antiviral candidates is underway.
COVID-19 and Other Coronavirus Programs
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
- 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.
- In
Norovirus Program
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
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 inAustralia . 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.
2023 Financial Results
Research and development (R&D) expenses for 2023 were
During 2023 the Company received
Interest income for 2023 was
The net loss for 2023 was
Cocrystal reported unrestricted cash as of
About
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, 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, the expected sufficiency of our cash balance to advance our programs and fund our planned operations, and our liquidity. 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 the high interest rates in response to inflation, uncertainty in the financial markets, the possibility of a recession and geopolitical conflict in
Investor Contact:
LHA Investor Relations
310-691-7100
jcain@lhai.com
Media Contact:
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Jabraham@jqapartners.com
CONSOLIDATED BALANCE SHEETS
(in thousands)
Assets | |||||||||
Current assets: | |||||||||
Cash | $ | 26,353 | $ | 37,144 | |||||
Restricted cash | 75 | 75 | |||||||
Tax credit receivable | 890 | 716 | |||||||
Prepaid expenses and other current assets | 1,773 | 2,243 | |||||||
Total current assets | 29,091 | 40,178 | |||||||
Property and equipment, net | 271 | 342 | |||||||
Deposits | 46 | 46 | |||||||
Operating lease right-of-use assets, net (including | 1,851 | 274 | |||||||
Total assets | $ | 31,259 | $ | 40,840 | |||||
Liabilities and stockholders’ equity | |||||||||
Current liabilities: | |||||||||
Accounts payable and accrued expenses | $ | 3,022 | $ | 976 | |||||
Current maturities of finance lease liabilities | - | 7 | |||||||
Current maturities of operating lease liabilities (including | 240 | 233 | |||||||
Total current liabilities | 3,262 | 1,216 | |||||||
Long-term liabilities: | |||||||||
Operating lease liabilities (including | 1,613 | 57 | |||||||
Total long-term liabilities | 1,613 | 57 | |||||||
Total liabilities | 4,875 | 1,273 | |||||||
Commitments and contingencies | |||||||||
Stockholders’ equity: | |||||||||
Common stock | 10 | 8 | |||||||
Additional paid-in capital | 342,288 | 337,489 | |||||||
Accumulated deficit | (315,914 | ) | (297,930 | ) | |||||
Total stockholders’ equity | 26,384 | 39,567 | |||||||
Total liabilities and stockholders’ equity | $ | 31,259 | $ | 40,840 |
CONSOLIDATED STATEMENTS OF OPERATIONS
(unaudited)
(in thousands, except per share data)
2023 | 2022 | ||||||||
Operating expenses: | |||||||||
Research and development | $ | 15,169 | $ | 12,392 | |||||
General and administrative | 5,990 | 5,745 | |||||||
Legal settlement | (2,600 | ) | 1,600 | ||||||
Impairments | - | 19,092 | |||||||
Total operating expenses | 18,559 | 38,829 | |||||||
Loss from operations | (18,559 | ) | (38,829 | ) | |||||
Other (expense) income: | |||||||||
Interest income (expense), net | 640 | (2 | ) | ||||||
Change in fair value of derivative liabilities | - | 12 | |||||||
Foreign exchange loss | (65 | ) | (18 | ) | |||||
Total other income (expense), net | 575 | (8 | ) | ||||||
Net loss | $ | (17,984 | ) | $ | (38,837 | ) | |||
Net loss per common share: | |||||||||
Loss per share, basic and diluted | $ | (1.87 | ) | $ | (4.77 | ) | |||
Weighted average number of common shares outstanding, basic and diluted | 9,651 | 8,143 |
# # #
Source:
2024 GlobeNewswire, Inc., source