MALVERN, Pa., March 13, 2024 (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 dosing is complete in the first cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age related macular degeneration (dAMD). GA affects approximately 1 million people in the United States alone.
“We are very enthusiastic about the potential of OCU410 as a one-time treatment for life with a single sub-retinal injection,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “While there are currently two recently approved products for the treatment of GA, both require approximately 6-12 intravitreal injections annually and target only the complement system. OCU410 addresses multiple pathways causing dAMD, including complement, lipid metabolism, inflammation, and oxidative stress.”
Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial. The enrollment in the first cohort is now complete and 3 subjects received 200µL single subretinal administration of the low dose (2.5×1010 vg/mL) of OCU410.
“As a retinal surgeon, I am encouraged by therapeutic options that can potentially provide long-term benefit to my patients,” said Lejla Vajzovic, MD, FASRS, Director of Duke Surgical Vitreoretinal Fellowship Program, Associate Professor of Ophthalmology with Tenure in Adult and Pediatric Vitreoretinal Surgery and Diseases, Duke University Eye Center. “OCU410 is a novel modifier gene therapy approach that could initiate a paradigm shift in the field of ophthalmology.”
The ArMaDa clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with GA and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.
“The American Macular Degeneration Foundation (AMDF) has supported the research of Dr. Neena Haider, inventor of modifier gene therapy, and OCU410 in particular, and is pleased that Ocugen is now spearheading the clinical trials necessary to bring this therapy closer to patients,” said Matthew Levine, Director of Grants, Advocacy and Partnerships at AMDF. “The continued advancement of OCU410 offers hope to those whose vision is already deteriorating that their remaining vision could be preserved and could potentially prevent others with an early dAMD diagnosis from developing any significant vision loss.”
The Company will continue to provide clinical updates.
About dAMD and GA dAMD affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula. The macula is the part of the retina responsible for clear vision in one’s direct line of sight.
dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function and central vision impairment. dAMD accounts for 85-90% of the total AMD population.
About OCU410 OCU410 utilizes an AAV delivery platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role in-vitro and in-vivo (animal model) studies. These results demonstrate the ability for OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of GA.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ 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 X 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.
– Promising Preclinical Results in Delayed Graft Function of Acute Kidney Injury –
– UNI-494 Phase 1 Single Ascending Dose Portion of Clinical Trial Complete –
LOS ALTOS, Calif., March 13, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today announced that two presentations related to UNI-494 were presented on March 12, 2024 at the 29th International Conference on Advances in Critical Care Nephrology AKI and CRRT 2024.
“We are excited about the tremendous progress we have made with our second clinical development asset, UNI-494,” said, Shalabh Gupta, MD, Chief Executive Officer of Unicycive. “Last week we announced that UNI-494 has been granted orphan drug designation by the FDA for the prevention of delayed graft function (DGF) after kidney transplantation which is a meaningful milestone for the program. The data presented at the CRRT conference demonstrates statistically significant results for UNI-494 in a preclinical model of DGF which provides additional evidence that UNI-494 may be a valuable asset for prevention of DGF and other acute kidney injury clinical conditions.”
“In conjunction with these presentations, we are also excited to announce that our ongoing Phase 1 clinical trial in UNI-494 has successfully completed the single ascending dose (SAD) portion of the study. UNI-494 was well-tolerated up to 160 mg administered as a single dose. This dose was chosen as the go-forward dose based on promising safety, tolerability, and pharmacokinetic data. In the multiple ascending dose (MAD) portion of the study, 80 mg is now being administered twice-a-day to participants enrolled in the study. We expect to complete the Phase 1 trial and report the full results in the second half of this year,” concluded Dr. Gupta.
The oral presentation, entitled, “Intravenous Administration of UNI-494 Ameliorates Acute Kidney Injury in Rat Model of Delayed Graft Function” was delivered by Satya Medicherla, Ph.D., Vice President, Preclinical Pharmacology, Unicycive Therapeutics. Dr. Medicherla presented results from the study evaluating the in vivo efficacy of intravenous (IV) UNI-494 in the unilateral renal ischemia-reperfusion rat model of acute kidney injury (AKI), which is a well-established model of DGF. In the study, a single IV dose of UNI-494 at 5 mg/kg/IV or 10 mg/kg/IV reduced specific kidney functional markers and tubular injury marker with statistically significant results (p<0.01). Importantly, UNI-494 prevented serum and urinary markers of AKI at 5 mg/kg, and proximal tubular injury scores improved in a dose-dependent manner. The study concluded that UNI-494 is a potential candidate for prevention of DGF and other AKI clinical conditions.
The poster, entitled, “UNI-494 Phase 1 Safety, Tolerability and Pharmacokinetics: Trial in Progress” was presented by Guru Reddy, PH.D., Vice President of Preclinical R&D, Unicycive Therapeutics. The poster describes the ongoing Phase 1 dose-escalating single-center, double-blind, placebo-controlled, randomized clinical trial in healthy volunteers. The trial consists of two parts: Part 1 is a single ascending dose (SAD) study to determine the maximum tolerated dose (n=40); Part 2 is a multiple ascending dose (MAD) study to understand the effect of multiple doses administered of UNI-494 (n=20). The trial is designed to evaluate the safety, tolerability, and pharmacokinetics of UNI-494 in healthy subjects. The SAD study was successfully completed, and a dose of 80 mg twice-a-day (BID) was carried over to the MAD study which is currently ongoing.
The publications will be available on the Unicycive website here.
About UNI-494
UNI-494 is a novel nicotinamide ester derivative and a selective ATP-sensitive mitochondrial potassium channel activator. Mitochondrial dysfunction plays a critical role in the progression of acute kidney injury and chronic kidney disease. UNI-494 has a novel mechanism of action that restores mitochondrial function and may be beneficial for the treatment of several diseases including kidney disease. Unicycive is currently conducting a Phase 1 dose-ranging safety study in healthy volunteers in the United Kingdom that is expected to complete in 2H of 2024. UNI-494 is protected by issued patent(s) in the U.S. and Europe and a wide range of patent applications worldwide. UNI-494 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the prevention of Delayed Graft Function (DGF) in kidney transplant patients.
About Delayed Graft Function
Delayed Graft Function (DGF) refers to the acute kidney injury (AKI) that occurs in the first week after kidney transplantation, which necessitates dialysis intervention. As the name indicates, DGF can result in sub-optimal or impaired graft function and is one of the most common and serious complications of kidney transplantation. Poor kidney function in the first week of graft life is detrimental to the longevity of the allograft. DGF is also associated with higher rates of tissue rejection and decreased patient survival. Currently, there are no FDA approved drugs for the treatment of DGF.
Ischemia/reperfusion injury (IRI) is known to be a major causative factor for the AKI that results in DGF during kidney transplantation. Ischemic preconditioning, that works by activating KATP channels in mitochondria, is a natural endogenous mechanism which protects cells from IRI in the heart, kidney, liver, and other organs. UNI-494 is a pharmacological approach that emulates and enhances this natural phenomenon of ischemic preconditioning.
About Unicycive Therapeutics
Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. UNI-494 is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. For more information, please visit Unicycive.com and follow us on LinkedIn and YouTube.
Forward-looking statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified using words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Unicycive’s expectations, strategy, plans or intentions. These forward-looking statements are based on Unicycive’s current expectations and actual results could differ materially. There are several factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Unicycive’s Annual Report on Form 10-K for the year ended December 31, 2022, and other periodic reports filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Unicycive specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Cadrenal Therapeutics Reported FYQ2023. Cadrenal announced a loss of $8.4 million or $(0.62) per share for FY2023 and $1.1 million or $(0.07) per share for 4Q23. The full-year loss consisted of Research and Development expense of $4.1 and General and Administrative Expense of $3.6 million, close to our estimates. Cash on December 31 was $8.5 million.
Tecarfarin IND Is Expected During 1H24. Cadrenal continues to work toward finalizing the trial protocols for the tecarfarin Phase 3 study, its oral anticoagulant for prevention of systemic thromboembolism in end-stage kidney disease (ESKD) patients with atrial fibrillation (irregular heartbeat, AFib). This is an Orphan population that can not use the commonly prescribed direct oral anticoagulants (DOAC) drugs.
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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.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Pivotal Trial Enrollment Complete. Unicycive announced that it has completed enrollment in the pivotal trial testing OLC (oxylanthanum carbonate), its phosphate binder, in ESRD patients on renal dialysis. This is a milestone for the trial that also confirms the timeframes we had expected for the trial results, NDA application, and product launch.
Data and NDA Expected Later In 2024. The pivotal trial is an open-label single arm study. Its primary endpoint is tolerability, with secondary endpoints of safety and pharmacokinetics. Statistical analysis is not required. The trial has a target enrollment of 60 patients and is expected to announce data in late 2Q24/mid2024.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
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.
Emerging Growth Public Healthcare Company Executive Presentations
Q&A Sessions Moderated by Noble’s Analysts and Bankers
Scheduled 1×1 Meetings with Qualified Investors
Noble Capital Markets, a full-service SEC / FINRA registered broker-dealer, dedicated exclusively to serving emerging growth companies, is pleased to present the Emerging Growth Virtual Healthcare Conference, taking place April 17th and 18th, 2024. This virtual gathering is set to be an immersive experience, bringing together a unique blend of investors, industry leaders, and experts in the life sciences, healthcare, and medical device sectors.
Part of Noble’s Robust 2024 Events Calendar
The Emerging Growth Virtual Healthcare Conference is part of Noble’s 2024 event programming, featuring a range of c-suite interviews, in-person non-deal roadshows throughout the United States, two more sector-specific virtual equity conferences, and culminating in Noble’s preeminent in-person investor conference, NobleCon20, to be held at Florida Atlantic University in Boca Raton, Florida December 3-4. Keep an eye out for the official press release on NobleCon20 coming soon.
The Emerging Growth Virtual Healthcare Conference will feature 2 days of corporate presentations from up to 50 innovative public healthcare, biotech, and medical device companies, showcasing their latest advancements and investment opportunities. Each presentation will be followed by a fireside-style Q&A session proctored by one of Noble’s analyst or bankers, with questions taken from the audience during the presentation. Panel presentations are planned, featuring key opinion leaders in the healthcare sector, providing valuable insights on emerging trends. Scheduled one-on-one meetings with public company executives, coordinated by Noble’s dedicated Investor Outreach team, are also available to qualified investors.
Why Your Company Should Present
Looking to increase awareness in your company and increase liquidity? Paid participation in Noble’s investor conferences, both virtual and in-person, provides that opportunity, with a tailored experience aimed at delivering substantial value. After 40 years of serving emerging growth companies, and the investors who follow them, Noble has built an investor base eager to discover where the next success story lies.
Noble’s investor base is relevant and, in many cases, new to your company. Noble’s dedicated Investor Outreach team provides unmatched exposure to investors that can invest in your company, including small money managers, family offices, RIAs, wealth managers, self-directed investors, and institutions. Most of Noble’s investors specifically seek undervalued, overlooked, emerging investment opportunities.
The cost to present includes your corporate presentation with a Q&A session proctored by one of Noble’s analysts or bankers, a webcast recording, scheduled 1×1 meetings with qualified investors, and marketing on Channelchek.
Benefits for Investors
The emerging growth healthcare space may be poised for a breakout year. The recent dislocation in the healthcare and biotech spaces has created compelling valuation profiles for many companies. Hear directly from the c-suite of the next innovators in this space and learn about new investment opportunities. The Q&A portion of each presentation gives you the opportunity to have your questions answered during or after the proctored session. The planned panel presentations are sure to provide expert insight on growing trends in the healthcare space. And, for qualified investors, one-on-one meetings are available with company executives; scheduled by Noble’s dedicated Investor Outreach team. All from the comfort of your own desk, and at no cost.
LOS ALTOS, Calif., March 07, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today announced enrollment has been completed in the open-label, single-arm, multicenter, multidose pivotal clinical trial with Oxylanthanum Carbonate (OLC). OLC is a next-generation lanthanum-based phosphate binding agent utilizing proprietary nanoparticle technology being developed to treat hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis.
“The completion of enrollment in our pivotal OLC clinical trial is a critical achievement for Unicycive as we strive to bring an improved therapy to chronic kidney disease patients struggling with hyperphosphatemia,” said Shalabh Gupta, MD, Chief Executive Officer of Unicycive. “Positive results from the trial will provide the basis to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA). We remain on track with topline data expected from the trial towards the latter part of the second quarter of this year and plan to file the NDA shortly thereafter. We want to thank the clinical trial participants, investigators, and sites whose significant interest in OLC drove strong recruitment as we pursue the goal of improving treatment options in hyperphosphatemia.”
“In this pivotal trial, we are looking to evaluate the tolerability, safety and pharmacokinetics of clinically effective doses of OLC in patients with CKD on dialysis. We believe the novel characteristics of OLC show its potential to be a best-in-class product to treat hyperphosphatemia by reducing the pill burden volume by more than 4-fold compared to the most prescribed phosphate binder. If approved, OLC will target the multibillion-dollar hyperphosphatemia market and will be a new potential therapy for physicians to administer to their patients,” added Dr. Gupta.
About the Oxylanthanum Carbonate (OLC) Pivotal Clinical Trial
The trial is expected to have 60 evaluable patients. Once participants are enrolled into the trial, they will go through a washout period for two weeks to clear their current phosphate binder from their system. Participants will initially be dosed at 500 mg of OLC three times a day (TID) and be titrated to a clinically effective dose that is defined as the dose required to achieve a serum phosphate range of ≤5.5 mg/dL. The maximum dose of OLC tested will be 3000 mg/day (1000 mg TID). As a reminder, all approved phosphate binders, including Fosrenol®, are administered on a dose titration schedule based on the control of serum phosphate. Once titrated to a clinically effective dose, participants will then be treated for four weeks to evaluate serum phosphate levels.
The primary endpoint for the trial will evaluate the tolerability of clinically effective doses of OLC in patients with CKD on dialysis. The secondary endpoints will evaluate safety and pharmacokinetics. There is no statistical analysis required to demonstrate efficacy as bioequivalence to Fosrenol was previously established; and there is no other clinical trial required to submit an NDA under the 505(b)(2) regulatory pathway.
About Hyperphosphatemia
Hyperphosphatemia is a serious medical condition that occurs in nearly all patients with End Stage Renal Disease (ESRD). If left untreated, hyperphosphatemia leads to secondary hyperparathyroidism (SHPT), which then results in renal osteodystrophy (a condition similar to osteoporosis and associated with significant bone disease, fractures and bone pain); cardiovascular disease with associated hardening of arteries and atherosclerosis (due to deposition of excess calcium-phosphorus complexes in soft tissue). Importantly, hyperphosphatemia is independently associated with increased mortality for patients with chronic kidney disease on dialysis. Based on available clinical data to date, over 80% of patients show signs of cardiovascular calcification by the time they become dependent on dialysis.
Dialysis patients are already at an increased risk for cardiovascular disease (because of underlying diseases such as diabetes and hypertension), and hyperphosphatemia further exacerbates this. Treatment of hyperphosphatemia is aimed at lowering serum phosphate levels via two means: (1) restricting dietary phosphorus intake; and (2) using, on a daily basis, and with each meal, oral phosphate binding drugs that facilitate fecal elimination of dietary phosphate rather than its absorption from the gastrointestinal tract into the bloodstream.
About Oxylanthanum Carbonate (OLC)
Oxylanthanum carbonate is a next-generation lanthanum-based phosphate binding agent utilizing proprietary nanoparticle technology being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD). OLC has over forty issued and granted patents globally. Its potential best-in-class profile may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden for patients in terms of the number and size of pills per dose that are swallowed instead of chewed. Based on a survey conducted in 2022, Nephrologists stated that the greatest unmet need in the treatment of hyperphosphatemia with phosphate binders is a lower pill burden and better patient compliance.1 The global market opportunity for treating hyperphosphatemia is projected to be in excess of $2.5 billion in 2023, with the United States accounting for more than $1 billion of that total. Despite the availability of several FDA-cleared medications, 75 percent of U.S. dialysis patients fail to achieve the target phosphorus levels recommended by published medical guidelines.
Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. As part of the clinical development program, two clinical studies were conducted in over 100 healthy volunteers. The first study was a dose-ranging Phase I study to determine safety and tolerability. The second study was a randomized, open-label, two-way crossover bioequivalence study to establish pharmacodynamic bioequivalence between OLC and Fosrenol. Based on the topline results of the bioequivalence study, pharmacodynamic (PD) bioequivalence of OLC to Fosrenol was established.
Fosrenol® is a registered trademark of Shire International Licensing BV. 1Reason Research, LLC 2022 survey. Results here.
About Unicycive Therapeutics
Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. UNI-494 is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. For more information, please visit Unicycive.com and follow us on LinkedIn and YouTube.
Forward-looking statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified using words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Unicycive’s expectations, strategy, plans or intentions. These forward-looking statements are based on Unicycive’s current expectations and actual results could differ materially. There are several factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Unicycive’s Annual Report on Form 10-K for the year ended December 31, 2022, and other periodic reports filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Unicycive specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Commercial Scale Production Facility Achieves Validation. GeoVax has announced that its manufacturing technology for producing commercial-scale MVA-based vaccines has been validated for production. This signifies the successful transfer of the technology from the research facility to the commercial cGMP manufacturing plant, with production runs that meet specifications for use in clinical trials and commercial sales. This was a milestone we had anticipated that allows higher quantities, faster production, and greater yields of its proprietary MVA-based vaccines.
The New Manufacturing Facility Cam Produce The Pipeline Vaccines. Products that can be made in the system include CM04S1, its vaccine currently in two different Phase 2 trials to stimulate protective responses against COVID for immunocompromised patients with hematological cancers and in Phase 2 as a universal booster for COVID. The facility can also produce the vaccines in development for infectious diseases including smallpox, Ebola, zika, and malaria.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
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.
Manufacturing Process for Phase 3 and Commercial Production
Being Developed for GeoVax MVA-Based Vaccines
Atlanta, GA, March 6, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced a significant milestone toward implementation of a validated chicken embryonic fibroblast (CEF) based production system for the company’s MVA-based vaccines, with the release of its first lot of GEO-CM04S1 (next-generation Covid-19 vaccine) produced with a commercial manufacturing platform. This milestone marks the successful completion of the transfer and scale-up of manufacturing from the research-focused Center for Biomedicine & Genetics at City of Hope (Duarte, CA) to the experienced CDMO ABL Europe (a subsidiary of Oxford Biomedica), the Company’s cGMP (current Good Manufacturing Procedures) manufacturing partner.
David Dodd, GeoVax President and CEO, commented, “The successful establishment of cGMP production at ABL Europe represents great progress for GeoVax and the CM04S1 program. This latest manufacturing milestone also validates our choice of ABL Europe as our partner for cGMP production of our MVA-based vaccine candidates. This gives us a high degree of confidence in our manufacturing process as we move into late-stage clinical development for CM04S1, addressing a critically important unmet medical need for immunocompromised populations.”
Dodd continued, “While we continue the use of CEF-based production for our CM04S1 clinical programs, it is important to also recognize the significant advancements made in our commercial-scale production capabilities. Our multi-product license of ProBioGen’s AGE1.CR.PIX® suspension cell line enhances our capacity to produce MVA-based vaccines (including CM04S1 and GEO-MVA) and immunotherapies at an unprecedented scale. These developments signify GeoVax’s commitment to improving vaccine accessibility through cost-effective and scalable manufacturing processes.”
About GEO-CM04S1
GEO-CM04S1 is a next-generation Covid-19 vaccine based on GeoVax’s MVA viral vector platform, which supports the presentation of multiple vaccine antigens to the immune system in a single dose. CM04S1 presents both the spike and nucleocapsid antigens of SARS-CoV-2 and is specifically designed to induce both antibody and T cell responses to non-variable parts of the virus. The more broadly specific and functional engagement of the immune system is designed to protect against the continually emerging variants of Covid-19. Results released during 2023 demonstrated the safety and efficacy of CM04S1 and emphasize the role it will play in protecting immunocompromised patients from greater risk of severe disease, hospitalization and death from SARS-CoV-2 infection.
About GEO-MVA
In response to the global need to address the continued emerging threat from Mpox (monkeypox), GeoVax previously announced having secured rights from the National Institutes of Health (NIH) covering preclinical, clinical and commercial uses of the NIH-MVA as a vaccine against Mpox or smallpox. The Company is currently pursuing different regulatory pathways toward achievement of an expedited approval and intends to become the first U.S.-based supplier of the MVA vaccine to prevent Mpox and smallpox.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation Covid-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized Covid-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable Covid-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information, visit our website: www.geovax.com.
Forward-Looking Statements
This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.
Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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.
Combination THIO 180mg + cemiplimab achieved 38% overall response rate (ORR) in difficult-to-treat, third-line non-small cell lung cancer (NSCLC)
ORR of 38% significantly exceeds standard of care ORR in NSCLC third-line in patients without a targetable mutation who progressed on checkpoint inhibitors and chemotherapy
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced positive efficacy data for third-line treatment in its Phase 2 THIO-101 clinical trial evaluating THIO sequenced with the immune checkpoint inhibitor (CPI) cemiplimab (Libtayo®) in advanced non-small cell lung cancer (NSCLC).
As of January 8, 2024, overall response rate (ORR), characterized as partial or complete response to therapy, was 38% (3 out of 8 patients) in the efficacy evaluable population for combination THIO 180mg + cemiplimab in third-line treatment for NSCLC patients who failed treatment with immune checkpoint inhibitors in prior lines of therapy, with or without chemotherapy.
“As an impressive measure of efficacy, the strong response rate of 38% in third-line treatment supports our premise that THIO administration prior to cemiplimab can improve tumor responses to immunotherapy in advanced NSCLC patients resistant to CPIs and other standard treatments,” said Vlad Vitoc, M.D., MAIA’s Chairman and Chief Executive Officer. “Around 60-70% of NSCLC patients do not have a targetable mutation and cannot benefit from a biomarker-targeted therapy, making it the greatest unmet medical need population in lung cancer. In currently available treatments for these patients in third-line, response rates range around 6%.1 We are encouraged by the excellent efficacy findings in THIO-101 to date, adding impressive ORR to unprecented disease control rates (DCR), and further demonstrating the potential of our first-in-class treatment to redefine the standard of care for NSCLC patients.”
The efficacy evaluable population defined in the THIO-101 protocol considers all subjects who received at least one dose of THIO treatment and have at least one postbaseline tumor assessment (scans). Two third-line patients in the 180mg dose cohort did not have recorded scans at the data cutoff. Safety remained consistent with previous reports.
The Company recently announced early completion of enrollment in the THIO-101 trial. THIO-101 is expected to be the first completed clinical study of a telomere-targeting agent in the field of cancer drug discovery and treatment.
About THIO
THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About THIO-101, a Phase 2 Clinical Trial
THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. It is the first trial designed to evaluate THIO’s anti-tumor activity when followed by PD-(L)1 inhibition. The trial is testing the hypothesis that low doses of THIO administered prior to cemiplimab (Libtayo®) will enhance and prolong immune response in patients with advanced NSCLC who previously did not respond or developed resistance and progressed after first-line treatment regimen containing another checkpoint inhibitor. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer compound and a priming immune activator (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint. Treatment with cemiplimab (Libtayo®) followed by THIO has been generally well-tolerated to date in a heavily pre-treated population. For more information on this Phase II trial, please visit ClinicalTrials.gov using the identifier NCT05208944.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
1Journal of Thoracic Oncology, Volume 4, Number 12, December 2009. *Note: no updated 3rd line NSCLC data in recent years.
THIO-101 Phase 2 trial nears completion with survival and response data forthcoming; exploration of multiple cancer indications and next-generation molecules continues.
Shareholder Letter available in Investor Relations section of MAIA’s corporate website.
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA” or the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today published a 2024 Letter to Shareholders by Chairman and Chief Executive Officer Vlad Vitoc, M.D., detailing the Company’s immuno-oncology cancer treatment candidates and development pipeline.
“At MAIA Biotechnology, our tenacious pursuit of innovative medicines to improve and extend people’s lives has led us to the forefront of cancer research. As we wrap up the Phase 2 clinical trial of our lead molecule THIO in non-small cell lung cancer (NSCLC) and pursue additional indications and a pipeline of next-generation THIO-like molecules, we are creating a robust and transformational cancer treatment franchise,” states Dr. Vitoc at the opening of his shareholder letter.
Letter Highlights
THIO-101 Phase 2 clinical trial nears completion; survival and response data updates forthcoming.
Along with NSCLC, MAIA’s pipeline of immuno-oncology therapies includes multiple hard-to-treat cancers.
More than 80 THIO-like compounds have been developed for the Company’s second-generation telomere targeting program.
Company’s pipeline includes THIO-102 Phase 2 and THIO-103 Phase 2/3 clinical trials (planning stage), and Investigational New Drug (IND)-enabling studies for second-generation telomere targeting agents.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
LOS ALTOS, Calif., March 04, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today announced that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) to UNI-494 for the prevention of Delayed Graft Function (DGF) in kidney transplant patients. UNI-494 is a cytoprotective agent that elicits an ischemic preconditioning effect by activating KATP channels in mitochondria to restore mitochondrial function.
“We are pleased to announce that the FDA has granted orphan drug designation to UNI-494 for the prevention of delayed graft function after kidney transplantation, an unmet medical need for which there are no FDA-approved drugs,” said Shalabh Gupta, MD, Chief Executive Officer of Unicycive. “Obtaining ODD is an important milestone in the development of UNI-494 that may provide certain tax credits for qualified clinical trials, exemption of user fees, and the potential for seven years of market exclusivity after approval. DGF is one of the most serious complications resulting from kidney transplantation, and we believe that the mechanism of action of UNI-494 is ideally suited for the prevention of this orphan condition.”
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan drug designation to agents that have the potential to offer a safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 individuals in the United States.
As previously announced, on March 12, 2024, Unicycive will present data on the efficacy of UNI-494 in animal models of DGF and a poster describing the ongoing Phase 1 clinical trial design for UNI-494 in healthy volunteers at the 29th International Conference on Advances in Critical Care Nephrology AKI and CRRT 2024.
About Delayed Graft Function
Delayed Graft Function (DGF) refers to the acute kidney injury (AKI) that occurs in the first week after kidney transplantation, which necessitates dialysis intervention. As the name indicates, DGF can result in sub-optimal or impaired graft function and is one of the most common and serious complications of kidney transplantation. Poor kidney function in the first week of graft life is detrimental to the longevity of the allograft. DGF is also associated with higher rates of tissue rejection and decreased patient survival. Currently, there are no FDA approved drugs for the treatment of DGF.
Ischemia/reperfusion injury (IRI) is known to be a major causative factor for the AKI that results in DGF during kidney transplantation. Ischemic preconditioning, that works by activating KATP channels in mitochondria, is a natural endogenous mechanism which protects cells from IRI in the heart, kidney, liver, and other organs. UNI-494 is a pharmacological approach that emulates and enhances this natural phenomenon of ischemic preconditioning.
About UNI-494
UNI-494 is a novel nicotinamide ester derivative and a selective ATP-sensitive mitochondrial potassium channel activator. Mitochondrial dysfunction plays a critical role in the progression of acute kidney injury and chronic kidney disease. UNI-494 has a novel mechanism of action that restores mitochondrial function and may be beneficial for the treatment of several diseases including kidney disease. Unicycive is currently conducting a Phase 1 dose-ranging safety study in healthy volunteers in the United Kingdom that is expected to complete in 2H of 2024. UNI-494 is protected by issued patent(s) in the U.S. and Europe and a wide range of patent applications worldwide.
About Unicycive Therapeutics
Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. UNI-494 is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. For more information, please visit Unicycive.com and follow us on LinkedIn and YouTube.
Forward-looking statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified using words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Unicycive’s expectations, strategy, plans or intentions. These forward-looking statements are based on Unicycive’s current expectations and actual results could differ materially. There are several factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Unicycive’s Annual Report on Form 10-K for the year ended December 31, 2022, and other periodic reports filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Unicycive specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.
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, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
PDSB Has Clinical Trial Milestones For 2024. We expect PDS Biotech is expecting to start the Phase 3 trial for PDS0101in head and neck cancer in 1Q24. This follows strong data from the Phase 2 trial presented during 2023. Several Investigator-initiated trials (IITs) testing PDS0101 in other indications have presented data showing improvements over current standards of care, its mechanism of action, and its potential use in related cancers. The second product, PDS01ADC, has also shown strong data in a Triple Combination trial. In our opinion, the stock has not reflected these results in the stock price.
A Phase 3 Trial For PDS0101 Is Expected Shortly. The Phase 3 VERSAMUNE-003 trial for PDS0101will enroll patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) who have failed treatment with standard chemotherapy but have not been treated with an immune checkpoint inhibitor (ICI naïve). The trial design follows the Phase 2 VERSAMUNE-002, which showed strong improvements in overall response rate (ORR) and overall survival (OS) benefits compared with current treatments.
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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.
Progress across the pipeline in multiple clinical trials, including
Phase 2 program of GEO-CM04S1, next-generation Covid-19 vaccine, and
multicenter Phase 1/2 study of Gedeptin®, targeting advanced head and neck cancer
Catalyst-rich 2024 with data readouts planned throughout the year
Company to host conference call and webcast today at 4:30 p.m. ET
ATLANTA, GA, February 29, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced its financial results and key operational accomplishments for the year ended December 31, 2023.
“2023 marked another year of advancement of our ongoing clinical programs for GEO-CM04S1, our next-generation Covid-19 vaccine, and for Gedeptin® in cancer therapy,” stated David Dodd, GeoVax’s Chairman and CEO. “This past year, we completed enrollment for the Phase 2 clinical trial assessing GEO-CM04S1 as a universal booster for the mRNA Covid vaccines, while expanding to multiple sites for our Phase 2 trial among immunocompromised/stem cell transplant patients, evaluating GEO-CM04S1 as a primary vaccine, in direct comparison to mRNA vaccines. In addition, the third Phase 2 trial of GEO-CM04S1 was initiated, evaluating our vaccine among immunocompromised/Chronic Lymphocytic Leukemia patients as a booster for such patients having initially received a mRNA vaccine, also in a direct comparison to a mRNA vaccine.
“Results released during 2023 demonstrated the safety and efficacy of GEO-CM04S1 and emphasize the role it will play in protecting immunocompromised patients from greater risk of severe disease, hospitalization and death from SARS-CoV-2 infection,” Dodd continued. “Perhaps of most importance, the results to date have demonstrated potential protective immunity of GEO-CM04S1 against multiple strains of SARS-CoV-2, from the ancestral Wuhan strain through Delta and the highly virulent Omicron XBB.1.5, without the need for vaccine reconfiguration. This critically important potential feature appears unique to our Covid-19 vaccine.”
Dodd concluded, “Relative to our oncology progress, GeoVax completed enrollment for the Phase 1/2 clinical trial of Gedeptin among advanced head and neck cancer patients. Results to date have demonstrated safety of this therapy and consistent reduction in treated tumors. During first half 2024, we anticipate reporting additional results from the Gedeptin Phase 1/2 clinical trial, as well as our plans for an expanded Phase 2 clinical trial. Overall, these achievements can be attributed to the successful execution of our long-term strategy, with an end goal to bring unique, patented products to market, addressing unmet medical needs. We look forward to reporting further progress in these studies and are encouraged to be entering into a data-rich 2024.”
2023 Clinical Trial Progress and Operational Developments
GEO-CM04S1
Enrollment completed for the Phase 2 clinicaltrial assessing GEO-CM04S1 as a potential universal booster for patients previously vaccinated with Pfizer or Moderna vaccines. The data presented during this period showcased promising results, indicating the potential of GEO-CM04S1 as a versatile Covid-19 vaccine capable of providing immunity against various strains, including the Wuhan, Delta, and Omicron variants. The trial involves 63 healthy adults who previously received mRNA vaccines as their primary vaccine. The data showed no serious adverse events and significant increases in neutralizing antibody, as well as cellular immune responses against multiple SARS-CoV-2 variants. Final results from this trial are anticipated during the fourth quarter of 2024.
Initiation of a Phase 2 booster trial targeting immunocompromised patients with chronic lymphocytic leukemia (CLL), who typically have reduced immune responses to mRNA vaccines due to their medical condition. This investigator-initiated trial expects to enroll 80 patients and directly compare GEO-CM04S1 with the Pfizer/BioNTech Bivalent vaccine. Results from an interim analysis are anticipated during the first half of 2024.
Data presentations from the immunocompromised/stem cell transplant patient Phase 2 trial of GEO-CM04S1 at the World Vaccine Congress, as well as initial results published in the peer-reviewed journal, Vaccines. The findings demonstrated robust immunogenicity, illustrating the vaccine’s ability to induce both antibody and T cell responses, essential for conferring protection, particularly in immunocompromised individuals. The article also highlighted the unique feature of GEO-CM04S1 providing protective immune levels from the ancestral Wuhan strain through Delta and the highly virulent Omicron XBB.1.5 variant. This study has been expanded to a multi-site trial, with further results anticipated throughout 2024.
Gedeptin®
Completion of patient enrollment for the Phase 1/2 clinical trial of Gedeptin among advanced head and neck cancer patients. The data presented at the AACR-AHNS Head and Neck Cancer Conference emphasized the safety and feasibility of Gedeptin therapy, providing insights into its potential as a treatment option for patients with limited therapeutic alternatives. The initial Phase 1/2 trial aims to guide future studies, potentially expanding the application of Gedeptin in other solid tumor areas and in combination with immune checkpoint inhibitors. During 2024, we expect to announce plans relative to an expanded Phase 2 study among advanced head and cancer patients, following discussions with regulatory authorities. In addition, we plan to outline plans for further Gedeptin clinical development, both in additional monotherapy and in combination-therapy (e.g., Gedeptin + immune-checkpoint inhibitor) indications.
Advanced Vaccine Manufacturing Process
Significant advancements made in MVA manufacturing capabilities focused on implementing a transformative manufacturing process in support of MVA-based vaccines and immunotherapies. The multi-product license with ProBioGen involving the AGE1.CR.PIX® suspension cell line enhances GeoVax’s capacity to produce MVA-based vaccines and immunotherapies at an unprecedented scale. Additionally, the agreement with Advanced Bioscience Laboratories, Inc. (ABL) secures cGMP production capabilities in support of GeoVax transitioning to worldwide commercialization capability. These developments signify GeoVax’s commitment to improving vaccine accessibility through cost-effective and scalable manufacturing processes. Our intent is to successfully develop our products for worldwide commercialization and distribution, in conjunction with partnering and collaborative relationships.
Corporate and Intellectual Property Developments
Achieved notable milestones in intellectual property development, securing multiple patents covering a range of vaccine candidates. The expanded rights under the NIH Covid-19 license to include Mpox and smallpox further diversify GeoVax’s vaccine portfolio, potentially offering broader protection against infectious diseases. Additionally, the issuance of patents for Ebola, Marburg, Malaria, and HIV vaccines underscores GeoVax’s innovative approach to vaccine development and its dedication to advancing global health initiatives. As of February 2024, the following actions were taken by global patent offices, further strengthening the Company’s intellectual property assets:
The Japanese Patent Office issued a Decision of Grant notifying GeoVax of the allowance of the Company’s Patent Application No. 2022-153352 titled “Compositions and Methods for Generating an Immune Response to a Tumor Associated Antigen.” The allowed claims are directed to recombinant MVA viral vectors comprising specific MUC-1 nucleic sequences used in GeoVax’s MUC-1 tumor-associated antigen immunotherapy program. Pharmaceutical compositions for inducing immune responses, preventing or reducing neoplasm growth, or treating cancer are also covered by the granted claims.
The U.S. Patent and Trademark Office issued Patent No. 11,896,657 to GeoVax, pursuant to the Company’s patent application No. 17/584,231 titled “Replication-Deficient Modified Vaccinia Ankara (MVA) Expressing Marburg Virus Glycoprotein (GP) and Matrix Protein (VP40).” The allowed claims generally cover GeoVax’s vector platform for expressing Marburg virus antigens in virus-like particles (VLPs) utilizing an MVA viral vector.
The U.S. Patent and Trademark Office issued Patent No. 11,897,919 pursuant to the Company’s patent application No. 17/409,574 titled “Multivalent HIV Vaccine Boost Compositions and Methods of Use.” The allowed claims generally cover a priming vaccination with a DNA vector encoding multiple HIV antigens in virus-like particles (VLPs), followed by a boost vaccination with GeoVax’s vector platform for expressing HIV-1 antigens in VLPs utilizing an MVA viral vector.
Appointed J. Marc Pipas, M.D., as Executive Medical Director, Oncology. Dr. Pipas has extensive clinical, research, and leadership expertise in oncology, built on a long and successful academic career at Dartmouth-Hitchcock Medical Center/Norris Cotton Cancer Center, an NCI Comprehensive Cancer Center. He brings a deep understanding of oncologic therapeutics and clinical trial management, as well as a network of research contacts and leadership skills honed by many years of experience.
2023 Full Year Financial Results
Net Loss: Net loss for the year ended December 31, 2023, was $26.0 million, as compared to $14.0 million for the year ended December 31, 2022.
R&D Expenses: Research and development expenses were $20.7 million for 2023, compared to $9.1 million in 2022, with the increase primarily due to the costs of conducting clinical trials for GEO-CM04S1 and Gedeptin, costs of manufacturing materials for use in our clinical trials, technology license fees, personnel costs, costs of preclinical research activities and higher travel costs.
G&A Expenses: General and administrative expenses were $6.0 million for 2023, compared to $5.0 million in 2022, with the increase primarily attributable to higher personnel costs, investor relations consulting costs, legal fees, patent costs and travel expenses.
Cash Position: GeoVax reported cash balances of $6.5 million on December 31, 2023, as compared to $27.6 on December 31, 2022.
Summarized financial information is attached. Further information is included in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission.
Conference Call Details
Management will host a conference call scheduled to begin at 4:30 p.m. ET today, February 29, 2024, to review financial results and provide an update on corporate developments. A question-and-answer session will follow management’s formal remarks.
A webcast replay of the call will be available for three months via the same link as the live webcast approximately two hours after the end of the call.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation Covid-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized Covid-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable Covid-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information, visit our website: www.geovax.com.
Forward-Looking Statements
This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.
Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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.
