Established High Dose as the Maximum Tolerable Dose in Current OCU400 Clinical Trial
MALVERN, Pa., Dec. 07, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Data Safety and Monitoring Board (DSMB) for the OCU400 clinical trial recently convened and established high dose as the maximum tolerable dose (MTD) in the dose-escalation phase of the study.
“The DSMB has recommended moving forward to dose subsequent subjects with NR2E3 and RHO gene mutations associated with Retinitis Pigmentosa (RP) and CEP290 gene mutations associated with Leber Congenital Amaurosis (LCA) at the targeted dose in the expansion phase of the study,” said Dr. Peter Y. Chang, MD, FACS, Massachusetts Eye Research & Surgery Institution, DSMB Chair for the OCU400 clinical trial. “No serious adverse events (SAEs) related to OCU400 have been reported to date.”
10 patients with NR2E3 and RHO gene mutations associated with RP have been dosed in the Phase 1/2 clinical trial to date. An additional eight patients with these RP gene mutations, along with three patients with CEP290 gene mutations associated with LCA, will be dosed at MTD and enrollment is expected to be complete by the end of Q1 2023.
Data from the MTD has the potential to navigate strategy for the planned Phase 3 study in the U.S. and other major markets. The Company plans to file a Biologics License Application (BLA) for OCU400 in 2025.
Ocugen is committed to finding solutions for people with inherited retinal disease for whom no effective treatment options exist. Currently, RP is associated with mutations in more than 100 genes, affecting more than 2.5 million people globally. LCA is a rare eye disease associated with mutations in more than 25 genes, affecting more than 150,000 people globally.
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.
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 statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. 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.
Contact: Tiffany Hamilton Head of Communications IR@ocugen.com
Affirms plans to report topline Phase 1 results in 2022 andto initiatePhase 2a influenza human challenge trial in 2023
BOTHELL, Wash., Dec. 01, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) announces that progress with CC-42344 for the treatment of pandemic and seasonal influenza A from its Phase 1 study was presented today at the World Antiviral Congress 2022 underway in San Diego. The presentation, “Novel Broad-Spectrum Influenza A PB2 Inhibitor: Clinical Update,” was delivered by Cocrystal President and co-interim CEO Sam Lee, PhD and the slides are available here.
“We appreciate the opportunity to present our antiviral drug discovery platform technology and favorable interim Phase 1 data,” said Dr. Lee. “Seasonal and pandemic influenza A continue to be major global health threats, especially given the potential for resistance against approved antivirals. Our presentation highlights potential clinical benefits for the treatment of pandemic influenza and the advantages of the oral influenza antiviral CC-42344 drug, which targets the PB2 protein of the influenza polymerase complex. We believe this novel mechanism of action holds promise against all significant influenza A strains including bird flu, while also having a high barrier to resistance. Our assessment to date also indicates that CC-42344 will have a strong synergistic effect when used with approved antivirals.”
In November 2022 Cocrystal reported that orally administered CC-42344 showed a favorable safety profile in both the single- and multiple-ascending dose portions of the Phase 1 study. The Company is currently awaiting pharmacokinetic results from this study and plans to report topline results later this year.
About CC-42344 CC-42344 is a novel PB2 inhibitor discovered using Cocrystal’s proprietary structure-based drug discovery platform technology. CC-42344 targets the influenza polymerase, an essential replication enzyme with several highly conserved regions common to multiple influenza strains. 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®.
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 the potential clinical benefits of CC-42344, including its prospective efficacy against all influenza A strains, high barrier to resistance and synergistic effects with approved antivirals, and our plans to report topline results of the Phase 1 study by the end of 2022. 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 any future impact of COVID-19 (including long-term or pervasive effects of the virus), inflation, interest rate increases and the war in Ukraine on the U.K. and global economy and on our Company, including supply chain disruptions and our continued ability to proceed with our programs, including our influenza A program, the ability of the contract research organization to recruit patients into clinical trials, the results of future preclinical and clinical studies, and general risks arising from clinical trials. 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, 2021. 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.
Baudax Bio is a pharmaceutical company focused on innovative products for acute care settings. ANJESO is the first and only 24-hour, intravenous (IV) COX-2 preferential non-steroidal anti-inflammatory (NSAID) for the management of moderate to severe pain. In addition to ANJESO, Baudax Bio has a pipeline of other innovative pharmaceutical assets including two novel neuromuscular blocking agents (NMBs) and a proprietary chemical reversal agent specific to these NMBs. For more information, please visit www.baudaxbio.com.
Gregory Aurand, Senior Research Analyst, Healthcare Services & Medical Devices, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Stock split approved at November 3, 2022 shareholder meeting. Shareholders of record September 29, 2022, approved the 1-for-40 stock split. The stock split will become effective for December 1, 2022, Nasdaq trading. The Company’s common stock will also commence trading under a new CUSIP number (07160F404).
Curing the deficiency. Baudax Bio received Nasdaq notice that the minimum $1.00 bid price requirement was not being met and had until January 2, 2023, to cure the issue, unless they sought to obtain an additional 180 days to regain compliance. The reverse split should bring the Company into listing compliance.
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*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.
CARLSBAD, Calif.–(BUSINESS WIRE)–Nov. 28, 2022– Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, announced today that its partner Genentech, a member of the Roche Group, has launched a Phase 2a, multicenter, open-label, single arm clinical study of RG6501 (OpRegen), a retinal pigment epithelial cell therapy. The study is intended to optimize subretinal surgical delivery and evaluate the safety and activity of OpRegen in approximately 30, and up to 60 patients with geographic atrophy (GA) secondary to age-related macular degeneration. The primary objectives of the study are to evaluate (i) the proportion of patients with subretinal surgical delivery of OpRegen to target regions under the retina, and (ii) to evaluate the safety of subretinal surgical delivery of OpRegen as measured by the incidence and severity of procedure-related adverse events at 3 months following surgery. A key secondary objective is to evaluate the proportion of patients with qualitative improvement in retinal structure, as determined by Optical Coherence Tomography (SD-OCT) imaging, within 3 months following surgery. RG6501 (OpRegen) is currently being developed under an exclusive worldwide collaboration between Lineage, Roche and Genentech.
“In our Phase 1/2a clinical trial, RG6501 (OpRegen) demonstrated the potential to slow, stop, or reverse the progression of GA in patients with GA. These results can be maintained beyond one year following a one-time, approximately 30-minute outpatient procedure,” stated Brian M. Culley, Lineage CEO. “We are excited that Roche and Genentech are advancing the OpRegen program in a larger clinical study and will seek not only to optimize and potentially improve its delivery, but also confirm safety and activity of OpRegen in this patient population. We expect the findings from this Phase 2a study will be highly informative and may increase the probability of success in any future larger, comparative trials.”
“We are excited for the launch of this Phase 2a study, where one of the key objectives is to optimize the delivery of OpRegen to achieve the best outcomes,” stated Seppi Lin, Vice President of OMNI Early Clinical Development at Genentech. “This study will provide key insights for the future development of the OpRegen program. Geographic Atrophy (GA) secondary to AMD is a disease area of high unmet need with no approved therapies, and we are committed to developing effective and innovative medicines for patients with serious eye diseases.”
About the Phase 2a Study
The planned Phase 2a clinical study is a multicenter, open-label, single arm study to optimize subretinal surgical delivery and to evaluate the safety and activity of OpRegen in patients with GA secondary to AMD. Approximately 30 and up to 60 patients may be enrolled across multiple sites and will receive OpRegen administered as a single subretinal injection to one eye with impaired vision. Study treatment will consist of a single subretinal injection of OpRegen at a dose of up to approximately 200,000 cells delivered to target areas of GA in the study eye.
The primary objectives of the study are to evaluate (i) the proportion of patients with subretinal surgical delivery of OpRegen to target regions under the retina, and (ii) to evaluate the safety of subretinal surgical delivery of OpRegen as measured by the incidence and severity of procedure-related adverse events at 3 months following surgery. A key secondary objective is to evaluate the proportion of patients with qualitative improvement in retinal structure, as determined by SD-OCT imaging within 3 months following surgery.
About Geographic Atrophy
Geographic atrophy (GA) is an advanced form of age-related macular degeneration (AMD) characterized by severe loss of visual function. GA is a leading cause of adult blindness in the developed world, affecting at least 5 million people globally. There are two forms of advanced AMD: neovascular AMD and GA. GA and neovascular AMD can occur simultaneously in the same eye, and patients treated for neovascular AMD may still go on to develop GA. GA typically affects both eyes. There are currently no U.S. Food and Drug Administration (FDA) or European Medicines Agency (EMA) approved treatment options available for patients with GA.
About Lineage Cell Therapeutics, Inc.
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its robust proprietary cell-based therapy platform and associated in-house development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed to either replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical and preclinical programs are in markets with billion dollar opportunities and include five allogeneic (“off-the-shelf”) product candidates: (i) OpRegen, a retinal pigment epithelial cell therapy in Phase 2a development for the treatment of geographic atrophy secondary to age-related macular degeneration, is being developed under a worldwide collaboration with Roche and Genentech, a member of the Roche Group; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase 1/2a development for the treatment of acute spinal cord injuries; (iii) VAC2, a dendritic cell therapy produced from Lineage’s VAC technology platform for immuno-oncology and infectious disease, currently in Phase 1 clinical development for the treatment of non-small cell lung cancer; (iv) ANP1, an auditory neuronal progenitor cell therapy for the potential treatment of auditory neuropathy; and (v) PNC1, a photoreceptor neural cell therapy for the potential treatment of vision loss due to photoreceptor dysfunction or damage. For more information, please visit www.lineagecell.com or follow the company on Twitter @LineageCell.
Forward-Looking Statements
Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to: our collaboration and license agreement with Roche and Genentech and the timing of anticipated FDA interactions; the potential of RG6501 (OpRegen) to be a safe, effective and durable, one-time treatment for geographic atrophy secondary to age related macular degeneration; the significance of this Phase 2a study, including the potential that it will be informative or increase the potential of success in future larger, comparative trials; and preclinical activities, clinical trials, and clinical data updates related to our programs. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the following risks: that positive findings in early clinical and/or nonclinical studies of a product candidate may not be predictive of success in subsequent clinical and/or nonclinical studies of that candidate; that competing alternative therapies may adversely impact the commercial potential of OpRegen; that Roche and Genentech may not successfully advance OpRegen or be successful in completing further clinical trials for OpRegen and/or obtaining regulatory approval for OpRegen in any particular jurisdiction; that Lineage may not be able to manufacture sufficient clinical quantities of its product candidates in accordance with current good manufacturing practice; and those risks and uncertainties inherent in Lineage’s business and other risks discussed in Lineage’s filings with the Securities and Exchange Commission (SEC). Lineage’s forward-looking statements are based upon its current expectations and involve assumptions that may never materialize or may prove to be incorrect. All forward-looking statements are expressly qualified in their entirety by these cautionary statements. Further information regarding these and other risks is included under the heading “Risk Factors” in Lineage’s periodic reports with the SEC, including Lineage’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the SEC and its other reports, which are available from the SEC’s website. You are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date on which they were made. Lineage undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.
MALVERN, Pa., Nov. 28, 2022 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that the Company’s Chief Scientific Officer, Arun Upadhyay, Ph.D., will participate in the 3rd Annual Dry AMD Therapeutics Summit at the Revere Hotel in Boston, Mass. from November 30-December 2, 2022.
“I am very pleased that Dr. Upadhyay will share more about our Modifier Gene Therapy platform—a novel approach to treating retinal diseases—during the Dry AMD Summit,” said Dr. Shankar Musunuri, Chairman, CEO & Co-Founder of Ocugen. “Our candidate, OCU410, is being developed to utilize the nuclear hormone receptor gene RORA for the treatment of dry AMD and we plan to file an IND application to initiate Phase 1/2 clinical trials in Q2 2023. We are enthusiastic about the potential for OCU410 to address unmet medical need in this category.”
Panel Discussion: Reviewing the Current State of Dry AMD Preclinical Models & What the Future Looks Like Date: Wednesday, November 30, 2022 Time: 12:45 p.m. ET
Presentation: A Modifier Gene Therapy Approach for Treatment of Dry AMD Date: Thursday, December 1, 2022 Time: 11:45 a.m. ET
About Ocugen, Inc. Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on Twitter and LinkedIn.
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 statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. 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.
Contact: Tiffany Hamilton Head of Communications IR@ocugen.com
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technology platforms. Our Versamune®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Raising Our Price Target to $19 Per Share. Data presentations from clinical trials for PDS0101 have driven PDSB within 10% of our previous price target. We continue to see PDS0101 as a highly promising treatment for HPV-positive cancers and see several important milestones to drive the stock higher in the coming year.
Price Target Is Based On PDS010 Sales Potential. Our new price target is based on discounted sales estimates for PDS010 sales in HPV-positive cancer. Following an End-of-Phase 2 meeting with the FDA following the June 2022 VERSATILE-002 data presentation, the company announced that it was accelerating its timetable for the pivotal trial for PDS0101. We expect this trial to begin in 2023 and project product launch in 2026.
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*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.
Chief Medical Officer, Dr. Lauren V. Wood, to present on the development of targeted immunotherapies for solid tumors based on the Versamune® platform
Dr. Siva Gandhapudi, Director of Immunology Product Development, to present on development of a universal flu vaccine based on the Infectimune™ platform
FLORHAM PARK, N.J., Nov. 21, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced two executives will be giving presentations at the World Vaccine & Immunotherapy Congress, which will take place from November 28 through December 1, 2022, in San Diego.
PDS Biotech speakers at the conference include:
Speaker
Time
Subject
Dr. Lauren V. Wood, Chief Medical Officer
Tuesday, Nov. 29, 4:50 p.m. Pacific Time
Development of targeted immunotherapies for solid tumors based on the Versamune® platform
Dr. Siva Gandhapudi, Director of Immunology Product Development
Wednesday, Nov. 30, 2:30 p.m. Pacific Time
Development of a universal flu vaccine based on the InfectimuneTM platform
More than 800 attendees are expected to attend the World Vaccine & Immunotherapy Congress to examine challenges around scientific, commercial, public health and policy issues in manufacturing, clinical trials, regulation, immune profiling, biomarkers, platform technologies, and additional topics.
“We welcome the opportunity to highlight the Versamune® and Infectimune™ platforms and their potential to address significant unmet medical needs to the biotechnology and scientific community,” said Dr. Frank Bedu-Addo, PDS Biotech President and CEO. “We are looking forward to advancing our product development in both oncology and infectious disease, ultimately to improve patient care.”
About Versamune®
Current immunotherapies have demonstrated an ability to treat certain cancers, but limitations with their effectiveness persist. Versamune® is a novel investigational T cell activating platform designed to stimulate a precise immune system response to cancer-specific proteins. PDS Biotech is advancing multiple Versamune® based clinical and pre-clinical programs with its lead clinical candidate – PDS0101 – targeting HPV-positive cancers.
About InfectimuneTM
Infectimune™ is a novel proprietary investigational T cell immune activating platform technology designed to train the immune system to better protect against disease. The Infectimune™ platform stimulates the immune system to recognize the pathogen and induce potent killer T cell and memory T cell response for long-term protection safely with no clinically relevant toxicities.
About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune® and Infectimune™ T cell-activating technology platforms. We believe our targeted Versamune® based candidates have the potential to overcome the limitations of current immunotherapy by inducing large quantities of high-quality, potent polyfunctional tumor specific CD4+ helper and CD8+ killer T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the potential to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV-positive cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.
Forward Looking Statements
This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0202 and other Versamune® and Infectimune™ based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0202 and other Versamune® and Infectimune™ based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Versamune® is a registered trademark and Infectimune™ is a trademark of PDS Biotechnology.
BOTHELL, Wash., Nov. 17, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) today announced that CC-42344 demonstrated a favorable safety profile in both the single-ascending dose and the multiple-ascending dose portions of the ongoing Phase 1 study. CC-42344 is a broad-spectrum oral antiviral for the treatment of pandemic and seasonal influenza A with a novel mechanism of action.
“We are encouraged by the clean safety profile observed with all dose levels in both the single-ascending and multiple-ascending dose portions of the Phase 1 study, and we will be assessing the pharmacokinetic data from this trial in the coming weeks,” said Sam Lee, Ph.D., Cocrystal’s President and co-interim CEO. “We remain on track to reach an important milestone of reporting topline Phase 1 study results later this year.
“Influenza is among the most serious global public health threats, particularly with the emergence of pandemic strains and resistance to available drugs,” he added. “Based on a novel mechanism of action and a high barrier to resistance, we believe CC-42344 holds potential to be a best-in-class oral treatment for pandemic and seasonal influenza.”
The randomized, double-controlled, dose-escalating Phase 1 study in Australia was designed to assess the safety, tolerability and pharmacokinetics (PK) of orally administered CC-42344 in healthy adults. In July 2022 Cocrystal reported that PK data from the single-ascending dose portion of the study support once-daily dosing. In October 2022 enrollment in the multiple-ascending dose portion of the trial was completed. The Company plans to present topline study results at the upcoming World Antiviral Congress on December 1, 2022 and to submit an application with the United Kingdom Medicines and Healthcare Products Regulatory Agency to conduct a Phase 2a human challenge study in early 2023. Subject to regulatory agency clearance, the Phase 2a study is expected to be initiated in the second half of 2023.
About CC-42344 CC-42344 is an oral PB2 inhibitor discovered using Cocrystal’s proprietary structure-based drug discovery platform technology. It is specifically designed to be effective against all significant pandemic and seasonal influenza A strains and to have a high barrier to resistance due to the way the virus’ replication machinery is targeted. CC-42344 targets the influenza polymerase, an essential replication enzyme with several highly conserved regions common to multiple influenza strains. 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 PK 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), 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.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our being on track to report topline results of the Phase 1 study later in 2022, the potential of CC-42344 to be a best-in-class candidate for the treatment of seasonal and pandemic influenza, and our expectations and plans to submit an application to the United Kingdom Medicines and Healthcare Products Regulatory Agency to conduct a Phase 2a human challenge study in early 2023 and to initiate the Phase 2a study in the second half of 2023. 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 any future impact of COVID-19 (including long-term or pervasive effects of the virus), inflation, interest rate increases and the war in Ukraine on the U.K. and global economy and on our Company, including supply chain disruptions and our continued ability to proceed with our programs, including our influenza A program, the ability of the contract research organization to recruit patients into clinical trials, the results of future preclinical and clinical studies, and general risks arising from clinical trials. 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, 2021. 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.
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.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
3Q22 Reported With Pipeline Update. Cocrystal reported a 3Q22 loss of $5.7 million or $0.70 per share. The product pipeline continues to make progress, with the first data from its influenza vaccine, CC-42344, scheduled for presentation in December. Two COVID-19 programs continue in preclinical studies, with an IND filing expected in 1Q23. The company ended the quarter with $42.1 million in cash.
Influenza Trial Data To Be Presented. Enrollment has been completed in the Phase 1 study for CC-42344 for seasonal and pandemic influenza. Safety and pharmacokinetic data is scheduled for presentation at the World Antiviral Congress in December 2022. The trial results will be submitted to regulatory authorities in the United Kingdom for a Phase 2a human challenge study to determine efficacy, expected to begin in 2H23.
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.
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China. Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.
Robert LeBoyer, Vice President, Research Analyst, Life Sciences , Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
3Q22 Showed Continued Progress In Ongoing Trials. Onconova announced a loss of $5.4 million or $(0.26) per quarter for 3Q22, ending the quarter with $42.6 million in cash. In addition to updates on its current clinical trials, the company announced its intentions to start a new Phase 1/2a trial testing narazaciclib in low-grade endometrioid endometrial cancer (LGEEC). This new trial is expected to begin in 1Q23 with first data announced in 4Q23.
New Trial Planned In Endometrial Cancer. The Phase 1/2a trial will test the combination of narazaciclib with letrozole (Femara, an aromatase inhibitor from Novartis) in recurrent low-grade endometrioid endometrial cancer (LGEEC). The trial will start with safety cohorts testing the combination, with a 200 mg dose of narazaciclib with the standard 2.5 mg dose of letrozole. This is the narazaciclib dose that is in its fifth cohort in the Phase 1 trial for solid tumors.
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.
Completed enrollment in the Phase 1 study with novel, broad-spectrum antiviral PB2 candidate CC-42344 for the treatment of pandemic and seasonal influenza A; remains on track to report topline results in 2022
Announced oral presentation of CC-42344 Phase 1 influenza A data at the World Antiviral Congress 2022 being held in December
Selected novel antiviral candidate CDI-988 for clinical development as an oral treatment for SARS-CoV-2 with Phase 1 study expected to begin in the first quarter of 2023
BOTHELL, Wash., Nov. 14, 2022 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) reports financial results for the three and nine months ended September 30, 2022, and provides updates on its antiviral pipeline, upcoming milestones and business activities.
“We are reporting continued progress in advancing our highly promising antiviral portfolio,” said Sam Lee, Ph.D., President and co-interim CEO of Cocrystal. “With enrollment completed in our CC-42344 Phase 1 study for the treatment of pandemic and seasonal influenza A, we are on track to report topline safety and pharmacokinetic (PK) results later this year. The full trial results will be a key component of UK regulatory agency submission for our influenza A Phase 2a human challenge study. Subject to the agency’s review and clearance of our submission, we expect Phase 2a study initiation in the second half of 2023.
“We advanced our oral COVID-19 program with the selected the novel, broad-spectrum protease inhibitor CDI-988 as our lead development candidate,” he added. “We are conducting IND-enabling toxicology studies with CDI-988 and plan to file for regulatory clearance to begin a first-in-human trial in Australia in the first quarter of 2023. Preclinical development activities are ongoing in our norovirus program with plans to select a lead candidate in the first half of 2023.”
“The recent increase in patients hospitalized with viral disease particularly among the pediatric population underscores the need for effective, broad-spectrum antivirals and provides rationale for our approach in developing candidates with barriers to drug resistance,” said James Martin, CFO and co-interim CEO. “We continue to be well positioned to execute on our clinical and regulatory goals given our clean capital structure, cost-efficient business model and a cash balance we believe is sufficient to fund planned operations for the next three years. That said, we continue to pursue non-dilutive funding to further support development of our promising antiviral programs.”
Antiviral Product Pipeline Overview
Many commercial antiviral drugs are only effective against certain strains of a virus and are less effective or not effective at all against other strains or variants. Cocrystal is developing novel drug candidates that specifically target proteins involved in viral replication. Despite the numerous strains that may exist or emerge, these targeted enzymes are required for viral replication and are essentially similar (highly conserved) across all strains. By targeting these highly conserved regions of the replication enzymes, our antiviral compounds are designed to be effective against major virus strains.
COVID-19 and Other Coronavirus Programs By targeting viral replication enzymes and protease, we believe it is possible to develop an effective treatment for all coronavirus diseases 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.
During 2022 Cocrystal entered into two agreements with the National Institute of Allergy and Infectious Diseases (NIAID) for exploratory preclinical studies to evaluate our 3CL protease inhibitors for the treatment of COVID-19. The NIAID collaboration announced in April 2022 for in vitro and in vivo studies evaluating the antiviral activity of our compounds has been successfully completed. In June 2022 we expanded our collaboration with the NIAID with a second agreement in which we provided our proprietary process chemistry information for oral 3CL protease inhibitors to the NIAID to support scale-up synthesis of a key intermediate of these compounds. This collaboration is ongoing.
Oral Protease Inhibitor CDI-988
We selected CDI-988 as our lead candidate for development as a potential oral treatment for SARS-CoV-2. CDI-988, which was designed and developed using our proprietary structure-based drug discovery platform technology, targets a highly conserved region in the active site of SARS-CoV-2 3CL (main) protease required for viral RNA replication.
CDI-988 exhibits superior in vitro potency against SARS-CoV-2 with activity maintained against current variants of concern, and demonstrated a safety profile and PK properties that are supportive of daily dosing.
We are currently conducting good laboratory practice (GLP) toxicology studies in preparation for a Phase 1 study.
We plan to initiate a Phase 1 study in the first quarter of 2023. We believe the FDA’s guidance for further development of our antiviral candidate CDI-45205 (described below) provides us with a clearer pathway for our planned Phase 1 study with CDI-988, as well as directives for designing a subsequent Phase 2 study.
Intranasal/Pulmonary Protease Inhibitor CDI-45205
An IND-enabling study is ongoing with CDI-45205, our novel SARS-CoV-2 3CL (main) protease inhibitor being developed as a potential treatment for COVID-19 and its variants.
We received guidance from the FDA regarding further preclinical and clinical development of CDI-45205 that provides a clearer pathway for future clinical development.
CDI-45205 and several analogs showed potent in vitro activity against the main SARS-CoV-2 variants to date including the Omicron variant, surpassing the activity observed with the original Wuhan strain.
CDI-45205 demonstrated good bioavailability in mouse and rat PK studies via intraperitoneal injection, and no cytotoxicity against a variety of human cell lines. CDI-45205 also demonstrated a strong synergistic effect with the FDA-approved COVID-19 medicine remdesivir.
CDI-45205 was among the broad-spectrum viral protease inhibitors we obtained from Kansas State University Research Foundation (KSURF) under an exclusive license agreement announced in April 2020. We believe the protease inhibitors obtained from KSURF have the ability to inhibit the inactive SARS-CoV-2 polymerase replication enzymes into an active form.
Replication Inhibitors
We are using our proprietary structure-based drug discovery platform technology to discover replication inhibitors for orally administered therapeutic and prophylactic treatments for SARS-CoV-2. Replication inhibitors hold potential to work with protease inhibitors in a combination therapy regimen.
InfluenzaPrograms Influenza is a severe respiratory illness caused by either the influenza A or B virus that results in outbreaks of disease mainly during the winter months.
Pandemic and Seasonal Influenza A
A novel PB2 inhibitor, CC-42344 has shown excellent antiviral activity against influenza A strains including pandemic and seasonal strains, as well as strains resistant to Tamiflu® and Xofluza®. CC-42344 also has favorable PK and drug-resistance profiles.
In March 2022 we initiated enrollment in our randomized, double-controlled, dose-escalating Phase 1 study to evaluate the safety, tolerability and pharmacokinetics of orally administered CC-42344 in healthy adults.
In April 2022 we announced preliminary Phase 1 study data, demonstrating a favorable safety and PK profile in the first two cohorts administered single ascending doses of CC-42344 at 100 mg and 200 mg.
In July 2022 we reported PK results from the single ascending dose of the study supporting once-daily dosing.
In November 2022 we announced the Phase 1 study had reached full enrollment and reiterated our expectation to report topline results in 2022.
We entered into an agreement with a UK-based clinical research organization to conduct a human challenge Phase 2a study evaluating safety, viral and clinical measures of orally administered CC-42344 in influenza A-infected subjects. Under the human challenge model, healthy adults will be infected with the influenza A virus under carefully controlled conditions, which we believe will hasten trial enrollment and ensure subjects are infected with influenza A.
We expect to submit an application with the United Kingdom Medicines and Healthcare Products Regulatory Agency in early 2023 to conduct a human challenge Phase 2a study. Pending clearance by the agency, we expect to initiate the study in the second half of 2023.
Pandemic and Seasonal Influenza A/B Program
In January 2019 we entered into an Exclusive License and Research Collaboration Agreement with Merck Sharp & Dohme Corp. (Merck) to discover and develop certain proprietary influenza antiviral agents that are effective against both influenza A and B strains. This agreement includes milestone payments of up to $156 million plus royalties on sales of products discovered under the agreement.
In January 2021 we announced completion of all research obligations under the agreement. Merck is now solely responsible for further preclinical and clinical development of compounds discovered under this agreement, and continues development activities with the compounds discovered under this agreement.
In April 2022 Merck indicated it was continuing development of the compounds discovered under this agreement.
Norovirus Program
We are developing certain proprietary broad-spectrum, non-nucleoside polymerases for the treatment of human norovirus infections using our proprietary structure-based drug design technology platform. We also hold exclusive rights to norovirus protease inhibitors for use in humans under the KSURF license.
We are targeting the selection of a preclinical lead in the first half of 2023.
Norovirus is a global public health problem responsible for nearly 90% of epidemic, non-bacterial outbreaks of gastroenteritis around the world.
Hepatitis C Program
We are seeking a partner to advance the development of CC-31244 following the successful completion of a Phase 2a study. This compound has shown favorable safety and preliminary efficacy in a triple-regimen Phase 2a study in combination with Epclusa (sofosbuvir/velpatasvir) for the ultra-short duration treatment of individuals infected with the hepatitis C virus (HCV).
HCV is a viral infection of the liver that causes both acute and chronic infection. In June 2022, the World Health Organization estimates that 58 million people worldwide have chronic HCV infections.
Third Quarter Financial Results
Research and development (R&D) expenses for the third quarter of 2022 were $3.9 million compared with $2.1 million for the third quarter of 2021, with the increase primarily due to the ongoing influenza A Phase 1 trial and advancement of the preclinical COVID-19 program. The Company anticipates higher R&D spending during the remainder of 2022 in preparation for additional clinical trials. General and administrative (G&A) expenses were $1.8 million for the third quarters of both 2022 and 2021.
The net loss for the third quarter of 2022 was $5.7 million, or $0.70 per share, compared with the net loss for the third quarter of 2021 of $3.9 million, or $0.48 per share.
Nine Month Financial Results
R&D expenses for the first nine months of 2022 were $9.1 million compared with $6.1 million for the first nine months of 2021. G&A expenses were $4.5 million for the first nine months of both 2022 and 2021.
The net loss for the first nine months of 2022 was $34.3 million, or $4.23 per share, and included the items described above. The net loss for the first nine months of 2021 was $10.5 million, or $1.44 per share.
Cocrystal reported unrestricted cash of $42.1 million as of September 30, 2022 compared with $58.7 million as of December 31, 2021. Net cash used in operating activities for the first nine months of 2022 was $16.5 million. The Company reported working capital of $43.3 million with 8.1 million common shares outstanding as of September 30, 2022.
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.
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 a Phase 1 study for our CDI-988 candidate in the first quarter of 2023, our expectations of reporting data from the Phase 1 clinical study of our CC-42344 candidate later in 2022 and timeline for filing with the UK regulatory agency and commencing a Phase 2a study in 2023, our plans to select a lead candidate for our norovirus program in the first half of 2023, the viability and efficacy of potential treatments for coronavirus and other diseases, expectations for the global market for therapeutics, our attempts to discover replication inhibitors, our ability to execute our clinical and regulatory goals, our expectations concerning R&D expenses, the expected sufficiency of our cash balance to fund our planned operations, our liquidity and our continued pursuit of non-dilutive funding. 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 the impact of COVID-19 (including long-term and pervasive effects of the virus), inflation, interest rate increases and the Ukraine war on our Company, our collaboration partners, and on the national and global economy, including manufacturing and research delays arising from raw materials and labor shortages, supply chain disruptions and other business interruptions including and adverse impacts on our ability to obtain raw materials and test animals as well as similar problems with our vendors and our current Contract Research Organization (CRO) and any future CROs and Contract Manufacturing Organizations, the results of the studies for CC-42344, the ability of our CROs to recruit volunteers for, and to proceed with, clinical studies, our reliance on Merck for further development in the influenza A/B program under the license and collaboration agreement, our and our collaboration partners’ technology and software performing as expected, financial difficulties experienced by certain partners, the results of future preclinical and clinical trials, general risks arising from clinical trials, receipt of regulatory approvals, regulatory changes, 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 which may result in variants that are resistant to a product candidate we develop, and the outcome of our appeal of the summary judgment. 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, 2021. 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.
Announced preliminary PDS0101 efficacy and safety data for Phase 2 clinical studies led by The University of Texas MD Anderson Cancer Center (IMMUNOCERV) and The National Cancer Institute at SITC 2022
Announced successful end-of-Phase 2 meeting with FDA for VERSATILE-002, allowing preparation for a registrational trial
Company to host conference call and webcast today at 8:00 AM EST
FLORHAM PARK, N.J., Nov. 14, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, will discuss its financial results for the quarter ended September 30, 2022 and provide a business update on its conference call today.
Recent Business Highlights:
PDS0101 Lead Drug Candidate
VERSATILE-002 Phase 2 Clinical Trial
Hosted a key opinion leader roundtable discussion on current treatment of head and neck cancer, and how PDS0101 might fit into the treatment paradigm.
Announced a successful end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA), allowing progression to a registrational trial for VERSATILE-002.
IMMUNOCERV Phase 2 Clinical Trial
Presented preliminary data on the clinical efficacy and safety of the combination of PDS0101 and chemoradiotherapy, as well as immunological correlates in the IMMUNOCERV Phase 2 trial being conducted at The University of Texas MD Anderson Cancer Center in locally, advanced cervical cancer at the Society for Immunotherapy of Cancer Conference (SITC) 2022.
NCI-led Triple Combination Phase 2 Clinical Trial
Presented data on immunological correlates associated with clinical benefit in patients with HPV-positive checkpoint inhibitor (CPI) refractory cancer treated with the PDS0101-based triple combination in the National Cancer Institute (NCI)-led Phase 2 clinical trial at the Society for Immunotherapy of Cancer Conference (SITC) 2022.
Reported expanded interim data for the Phase 2 PDS0101 based triple combination trial led by the NCI targeting advanced HPV-positive cancers.
Announced completion of recruitment into the NCI-led PDS0101-based triple combination Phase 2 trial, and also reported selection of the CPI refractory arm as the preferred patient group to target in a registrational study with the triple combination.
PDS0102 and PDS0103 Drug Candidates
Presented preclinical data on both PDS0102 and PDS0103, demonstrating the versatility of the Versamune® platform and generation of TARP and MUC1 specific polyfunctional CD8+ T cells presented at the American Association of Cancer Research (AACR) Special Conference: Tumor Immunology and Immunotherapy 2022.
PDS0202 Universal Flu Candidate
Presented data from the preclinical universal flu vaccine program at the American Society of Virology meeting, demonstrating the potential ability of PDS0202 to neutralize multiple strains of influenza in animals.
Financing
Entered into a venture loan and security agreement with Horizon Technology Finance Corporation, which provides PDS Biotech with up to $35 million in term loans.
“The third quarter has been monumental for PDS Biotech, and we continue to make strides towards commercialization of our lead candidate, PDS0101,” stated Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. We’ve remained focused on progressing our four Phase 2 clinical programs, most recently, announcing data from our IMMUNOCERV trial in locally, advanced cervical cancer. 100% (9/9) of patients had a clinical response with tumor shrinkage of over 60% at the midpoint evaluation, and 89% (8/9) of patients had a complete response with no evidence of disease at day 170, when treated with a combination of PDS0101 and chemotherapy. Furthermore, we released expanded interim data from our NCI-led PDS0101-based triple combination trial demonstrating 66% (19/29) survival at median follow up of 16 months in checkpoint inhibitor refractory HPV-positive cancer patients that appears to show signs of clinical efficacy, durability, and safety, consistent with the data presented at the American Society for Clinical Oncology 2022. And, with VERSATILE-002 in which PDS0101 is combined with KEYTRUDA®, we are preparing for a registrational trial after our successful end-of-Phase 2 meeting with the FDA. To date, we have presented PDS0101 Phase 2 efficacy data in over 60 patients and safety data in over 100 patients.”
Matthew Hill, Chief Financial Officer of PDS Biotech, stated, “We are excited about the progress we have made in our development programs, and we have strengthened the balance sheet to support our ongoing efforts. This August, we increased our cash position by entering into a loan agreement with Horizon Technology Finance Corporation, where we received an initial tranche of $25 million in term loans. This financing provides PDS Biotech with the financial resources and runway needed to prepare for a registrational trial for our lead candidate, PDS0101, and to advance our preclinical pipeline.”
Third Quarter 2022 Financial Results Net loss for the three months ended September 30, 2022 was approximately $7.4 million, or $0.26 per basic share and diluted share, compared to a net loss of approximately $7.0 million, or $0.24 per basic and diluted share, for the three months ended September 30, 2021. The higher net loss reported for the three months ended September 2022 is primarily due to additional costs for expansion of the Company’s research and development, including costs associated with our ongoing clinical trials and additional general and administrative costs.
Research and development expenses increased to $4.4 million for the three months ended September 30, 2022 from $3.7 million for the three months ended September 30, 2021. The increase of $0.7 million in 2022 was primarily attributable to an increase of $0.2 million in clinical study and research costs, $0.3 million in personnel costs and $0.4 million in manufacturing services partially offset by a decrease of $0.2 million in professional fees and facilities.
General and administrative expenses decreased to $2.9 million for the three months ended September 30, 2022 from $3.3 million for the three months ended September 30, 2021. The decrease of $0.4 million is primarily attributable to a decrease of $0.5 million in personnel an increase of $0.2 million in professional fees partially offset by a decrease of $0.1 million in facilities costs.
Total operating expenses were approximately $7.3 million for the three months ended September 30, 2022, from approximately $7.0 million for the three months ended September 30, 2021.
PDS Biotech’s cash balance as of September 30, 2022 was approximately $71.6 million.
Conference Call and Webcast The conference call is scheduled to begin at 8:00 AM EST today, November 14, 2022. Participants should dial 877-407-3088 (United States) or 201-389-0927 (International) and reference conference ID 13733006. To access the webcast, please use the following link. The event will be archived in the investor relations section of PDS Biotech’s website for six months.
About PDS0101 PDS Biotech’s lead candidate, PDS0101, combines the utility of the Versamune® platform with targeted antigens in HPV-positive cancers. In partnership with Merck & Co., PDS Biotech is evaluating a combination of PDS0101 and KEYTRUDA® in a Phase 2 study in first-line treatment of recurrent or metastatic head and neck cancer, and also in second line treatment of recurrent or metastatic head and neck cancer in patients who have failed prior checkpoint inhibitor therapy. A Phase 2 clinical study is also being conducted in both second- and third-line treatment of multiple advanced HPV-positive cancers in partnership with the National Cancer Institute (NCI). A third Phase 2 clinical trial in first line treatment of locally advanced cervical cancer is being performed with The University of Texas, MD Anderson Cancer Center. A final Phase 2 clinical trial of PDS0101 monotherapy in first line treatment of newly diagnosed patients HPV16-positive head and neck cancer patients is being conducted at the Mayo Clinic.
KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
About PDS Biotechnology PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune® and Infectimune™ T cell-activating technology platforms. We believe our targeted Versamune® based candidates have the potential to overcome the limitations of current immunotherapy by inducing large quantities of high-quality, potent polyfunctional tumor specific CD4+ helper and CD8+ killer T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the potential to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV-positive cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.
Forward Looking Statements This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune™ based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control, including record inflation, unforeseen circumstances or other disruptions to normal business operations arising from or related to COVID-19 and the ongoing military conflict between Russia and Ukraine. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in the Company’s annual and periodic reports filed with the SEC. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. Versamune® is a registered trademark and Infectimune™ is a trademark of PDS Biotechnology.
89% (8/9) had no evidence of disease (complete response) on day 170
Data Presented at Society for Immunotherapy of Cancer (SITC 2022)
FLORHAM PARK, N.J., Nov. 14, 2022 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, today announced that updated clinical data from the ongoing IMMUNOCERV Phase 2 clinical trial was presented during a poster session on November 11 at the 37th Annual Meeting for the Society for Immunotherapy of Cancer (SITC 2022). These data expand upon results detailed in the abstract submitted to SITC 2022 that was released to the public on November 7.
The enhanced data was presented in the poster titled, “IMMUNOCERV, an Ongoing Phase II Trial Combining PDS0101, an HPV-Specific T Cell Immunotherapy, with Chemotherapy and Radiation for Treatment of Locally Advanced Cervical Cancers,” which highlights key findings from The University of Texas MD Anderson Cancer Center-led IMMUNOCERV Phase 2 clinical trial (NCT04580771). The study is investigating PDS0101 in combination with standard-of-care chemoradiotherapy (CRT) for the potential treatment of cervical cancer in patients with large tumors over 5 cm in size and/or cancer that has spread to the lymph nodes (lymph node metastasis). New data from the study presented at SITC 2022 include:
9 of the 17 patients have now completed a day 170 post-treatment Positron Emission Tomography, Computed Tomography (PET CT) scan to assess the status of their cancer. This includes 78% (7/9) of treated patients with advanced cervical cancer (FIGO stage III or IV).
100% (9/9) of patients treated with the combination of PDS0101 and CRT had a clinical response with tumor shrinkage >60% at mid-point evaluation by MRI.
89% (8/9) of patients treated with the combination of PDS0101 and CRT demonstrated a complete response (CR) on day 170 by PET CT. One patient who received 3 of the 5 scheduled doses of PDS0101 showed signs of residual disease. One patient who had a CR died from an event unrelated to either their underlying disease or treatment.
1-year disease-free survival and 1-year overall survival of 89% (8/9) in patients treated with the combination of PDS0101 and CRT.
As previously reported, data confirm PDS0101 treatment activates HPV16-specific CD8+ T cells. This increase was not seen in patients who did not receive PDS0101. The increase in HPV16-specific T cells generated by the treatment is positively correlated with tumor cell death, suggesting cytotoxic CD8+ T cells are important mediators of antigen-specific immunity.
The data affirm that PDS0101 activates Type 1 interferon pathway in humans, mimicking the mechanism previously demonstrated in preclinical studies in animal models.
Toxicity of PDS0101 remains limited to low-grade local injection site reactions.
“The updated data from the ongoing IMMUNOCERV Phase 2 clinical trial presented during SITC 2022 add to the encouraging results observed thus far and suggest that the combination of PDS0101 and CRT may hold promise as a potential first-line treatment for advanced, localized cervical cancer,” said Dr. Frank Bedu-Addo, CEO of PDS Biotech. “Importantly, 100% of patients responded to treatment with the combination of PDS0101 and CRT. We believe this study also provides further confirmation that PDS0101 induces the right type, quality, and potency of killer T cells in humans that may translate to effective treatment of cervical cancer. We look forward to the continued advancement of the IMMUNOCERV Phase 2 clinical trial and the opportunity to report updated data during 2023.”
In addition, a second poster titled “Immune Correlates Associated with Clinical Benefit in Patients with Checkpoint Refractory HPV-Associated Malignancies Treated with Triple Combination Immunotherapy,” was presented at SITC 2022 and reported data from the Phase 2 triple combination trial being led by the Center for Cancer Research at the National Cancer Institute (NCI), part of the National Institutes of Health. The study is investigating PDS0101 in combination with two investigational immune-modulating agents: M9241, a tumor-targeting IL-12 (immunocytokine), and bintrafusp alfa, a bifunctional checkpoint inhibitor (PD-L1/ TGF-β). The triple combination is being studied in checkpoint inhibitor (CPI)-naïve and -refractory patients with advanced HPV-positive anal, cervical, head and neck, vaginal, and vulvar cancers who have failed prior therapy. For most patients who are CPI refractory, there is no effective therapy. The immune correlates before and after treatment in the CPI refractory patient population were studied. Highlights from the study presented at SITC 2022 included:
A more than two-fold increase in HPV16-specific T cells in the blood of 79% (11/14 tested) of the evaluated patients.
Increases on day 15 in several monitored immune correlates, such as granzyme B and interferon-gamma (IFN-γ), were associated with a clinical response.
Immune responses were associated with increases in natural killer cells, soluble granzyme B (associated with active killer T cells), IFN-γ, tumor necrosis factor-alpha (TNF-α), etc., two weeks after the first treatment cycle thus signaling a pro-inflammatory response.
These immunogenicity findings highlight the potential role of the combination in altering immune suppressive forces, and support previously announced results documenting promising clinical outcomes in the CPI-refractory population receiving the triple combination.
About PDS Biotechnology PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune® and Infectimune™ T cell-activating technology platforms. We believe our targeted Versamune® based candidates have the potential to overcome the limitations of current immunotherapy by inducing large quantities of high-quality, potent polyfunctional tumor-specific CD4+ helper and CD8+ killer T cells. To date, our lead Versamune® clinical candidate, PDS0101, has demonstrated the potential to reduce tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV-expressing cancers in multiple Phase 2 clinical trials. Our Infectimune™ based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on Twitter at @PDSBiotech.
About PDS0101 PDS Biotech’s lead candidate, PDS0101, combines the utility of the Versamune® platform with targeted antigens in HPV-expressing cancers. In partnership with Merck & Co., PDS Biotech is evaluating a combination of PDS0101 and KEYTRUDA® in a Phase 2 study in first-line treatment of recurrent or metastatic head and neck cancer, and also in second-line treatment of recurrent or metastatic head and neck cancer in patients who have failed prior checkpoint inhibitor therapy. A National Cancer Institute-supported Phase 2 clinical study of PDS0101 in a triple combination therapy is also being conducted in checkpoint inhibitor refractory patients with multiple advanced HPV-associated cancers. A third Phase 2 clinical trial in first-line treatment of locally advanced cervical cancer is being led by The University of Texas MD Anderson Cancer Center. A final Phase 2 clinical trial of PDS0101 monotherapy in first-line treatment of newly diagnosed patients HPV16+ head and neck cancer patients is being conducted at the Mayo Clinic.
KEYTRUDA® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
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Versamune® is a registered trademark and Infectimune™ is a trademark of PDS Biotechnology.
