In a significant value-creating event, X4 Pharmaceuticals has secured FDA approval for its lead drug Xolremdi, ushering the biotech into the commercial realm and positioning it as an attractive M&A target in the hot rare disease space.
Xolremdi (mavorixafor) nabbed approval to treat WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome, an ultra-rare primary immunodeficiency disorder. As the first approved therapy tackling WHIM’s genetic root cause, the CXCR4 inhibitor fills a massive unmet need where treatment options were previously limited to symptom management.
With its novel mechanism mobilizing infection-fighting white blood cells out of the bone marrow, Xolremdi demonstrated impressive clinical benefits in Phase 3. These included slashing rates of severe infections and boosting critical white blood cell levels like neutrophils and lymphocytes in WHIM patients.
While WHIM’s prevalence is difficult to pinpoint given its rarity, X4 estimates around 1,000 cases in the U.S. However, the commercial opportunity could be larger if the company successfully identifies underdiagnosed patients through its genetic screening initiatives and physician education efforts.
Xolremdi isn’t a curevbut holds potential to bolster long-term immune defenses for WHIM patients who chronically suffer from infections, warts, and abnormally low antibody levels. This positions the drug as a high-value therapy that can command premium pricing despite the tiny patient population.
Indeed, X4 has set an eyebrow-raising $496,400 annual list price for Xolremdi in patients over 50 kg, and $372,300 for smaller individuals. While pricey, these kinds of ultra-orphan drug costs are typical and highlight the blockbuster revenue potential if X4 can rapidly build out its U.S. presence and strike reimbursement wins with payers.
With its first FDA approval in hand, X4 is kicking its commercial launch into high gear. The company has deployed and trained a 24-person field force blanketing the country, established distribution channels, and stockpiled launch supplies ahead of the Xolremdi decision. Smooth execution out of the gate will be crucial.
This pivotal milestone transforms X4 from a clinical-stage biotech into a fully integrated rare disease player with an approved therapy, commercial infrastructure, and revenue stream. That fresh commercial status boosts X4’s profile considerably, increasing the odds it could become an enticing buyout target.
Big Pharma has been extremely hungry to expand rare disease franchises through M&A, driven by high unmet needs, accelerated regulatory pathways, and lucrative pricing opportunities in niche markets. Potential suitors could view X4’s new orphan disease platform as complementary.
Further underlining X4’s M&A appeal, the Xolremdi approval came with a priority review voucher as a bonus. These valuable vouchers can be redeemed to fast-track another regulatory filing or sold to other drugmakers for hundreds of millions.
Additionally, X4 believes mavorixafor has therapeutic potential beyond WHIM. The biotech aims to rapidly initiate a Phase 3 program testing the drug in chronic neutropenia, expanding its pipeline.
While the ultra-rare WHIM population limits how much a solo acquirer could value Xolremdi’s revenue stream, the drug’s approval derisks X4’s pipeline and corporate strategy. With its first FDA win and voucher under its belt, X4 stands as an intriguing M&A target that could fetch a premium from buyers seeking a rare disease foothold.
In a move that further solidifies its position in the inflammatory disease space, Incyte Corporation has agreed to acquire Escient Pharmaceuticals for $750 million. The deal, announced on Tuesday, will add two promising immunology and inflammation candidates to Incyte’s pipeline, complementing its existing dermatology portfolio.
The star assets in the acquisition are EP262 and EP547, both of which have shown promising early results in clinical trials. EP262, the more advanced of the two, is currently being evaluated for the treatment of various inflammatory skin conditions, including atopic dermatitis, psoriasis, and vitiligo. Preclinical data has demonstrated EP262’s ability to improve atopic dermatitis-like lesions and reduce inflammation, making it a potentially valuable addition to Incyte’s dermatology lineup.
EP547, while in an earlier stage of development, is being studied for the treatment of itching associated with kidney and liver diseases – a condition known as pruritus, which can significantly impact a patient’s quality of life. With two phase 1 trials currently underway, EP547 could address a significant unmet need in these patient populations.
“EP262 and EP547 are complementary additions to our portfolio, providing an opportunity to leverage our expertise, address the needs of patients with inflammatory diseases and additional potential launch opportunities starting in 2029,” said Hervé Hoppenot, Incyte’s CEO.
For Incyte, the acquisition of Escient Pharmaceuticals represents a strategic move to strengthen its position in the lucrative dermatology market. The company already has an approved JAK1/JAK2 inhibitor cream, Opzelura, which is indicated for atopic dermatitis and vitiligo. With EP262 and additional pipeline candidates, Incyte aims to expand its offerings and capture a larger share of the growing inflammatory skin disease market.
The deal also follows Incyte’s recent acquisition of the cancer drug Monjuvi from MorphoSys, further demonstrating the company’s commitment to bolstering its portfolio through strategic partnerships and acquisitions.
Escient Pharmaceuticals, a relatively young company founded in 2018, had already attracted significant investor interest, raising over $200 million in private financing rounds from the likes of Sanofi. The company’s promising early-stage pipeline and expertise in inflammatory diseases likely made it an attractive target for Incyte.
The transaction, which is expected to close in the third quarter of 2024, will provide Incyte with a diverse range of assets spanning multiple inflammatory and immunological indications. As the company continues to expand its offerings, it remains well-positioned to capitalize on the growing demand for innovative treatments in these therapeutic areas.
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.
MALVERN, Pa., April 19, 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 second 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, gene-agnostic option for the treatment of GA,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “OCU410 regulates multiple pathways involved with the disease, including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement) with a single sub-retinal injection.”
Dosing in the second cohort is complete and 3 subjects received 200 mL single subretinal administration of the medium dose (5×1010 vg/mL) of OCU410. Up to 13 leading retinal surgery centers across the United States are participating in the ArMaDa clinical trial.
“Currently we have two FDA approved, anti-complement therapies for GA targeting a single pathway of the disease, which has multifactorial and complex etiology,” said Syed M. Shah, MD, Vice Chair of Research and Digital Medicine and Director of Retina Service at Gundersen Health System, La Crosse, WI, and the lead investigator for the OCU410 Phase 1/2 trial. “The limited benefit comes with the burden of continued multiple intravitreal injections spanning over several years. This novel modifier gene therapy has the potential to transform the therapeutic landscape in GA treatment.”
A Data and Safety Monitoring Board meeting will convene next month to review the 4-week safety data of the medium dose cohort before proceeding with high dose, which is the final dose in the Phase 1 dose-escalation study.
The ArMaDa Phase 1/2 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 Company will continue to provide clinical updates on an ongoing basis.
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 as well as inhibiting the complement system in 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, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. 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.
In a bold strategic move, small-cap biotech Cullinan Oncology is transforming into an autoimmune disease company and rebranding as Cullinan Therapeutics. The Massachusetts company announced the major pivot alongside a $280 million private placement financing that extends its cash runway into 2028.
Cullinan is staking its future on the emerging potential of CAR-T cell therapies to treat autoimmune conditions like systemic lupus erythematosus (SLE). The company plans to advance its lead candidate CLN-978, a bispecific T cell engager originally developed for lymphoma, into SLE. An Investigational New Drug (IND) filing is targeted for the third quarter of 2024, with additional autoimmune indications likely to follow.
The strategic refocusing comes as preliminary data from small academic studies hint that CAR-T cells could induce durable remissions in autoimmune patients by depleting pathogenic B cells and modulating the immune system. In February, researchers reported that 8 out of 15 SLE patients achieved remission for over 1 year after CAR-T treatment, allowing them to discontinue all other medications.
“The unmet need in autoimmune diseases is vast, with most patients cycling through treatment after treatment without achieving remission,” said Cullinan CEO Alejandra Carvajal. “CAR-T cell therapy represents a potential paradigm shift, with an entirely novel mechanism to re-educate the immune system.”
Cullinan is one of the first movers in the autoimmune CAR-T space, but it won’t be alone for long. Peers like Kyverna, Cabaletta, Allogene and Arbor have all initiated programs or partnerships in the last year to develop similar cell therapies.
Cullinan has stopped enrolling patients in CLN-978’s lymphoma study to fully transition to autoimmune disorders. But the company is retaining its existing oncology pipeline, including lead asset zipalertinib in non-small cell lung cancer.
To fund the new autoimmune endeavors, Cullinan raised $280 million through the sale of shares and convertible securities to institutional investors. The private placement was led by venBio Partners and included Cullinan’s existing investors.
“This successful financing provides Cullinan with the resources to rapidly advance our CLN-978 program in SLE and beyond,” stated Carvajal. “We’re excited to lead the way into this new frontier at the intersection of cell therapy and autoimmune disease.”
Cullinan is making a high-risk, high-reward bet on still-unproven science. CAR-T’s efficacy in autoimmune conditions has only been explored in small patient numbers so far. But if the approach proves transformative, Cullinan could be at the vanguard of disrupting the large autoimmune drug market.
The hefty $280 million raise buys Cullinan plenty of runway to generate data from larger trials evaluating CLN-978 and shaping its future autoimmune portfolio. For autoimmune disease patients in need of new options, all eyes will be on Cullinan’s pioneering role in the promising CAR-T space.
MALVERN, Pa., April 10, 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 the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).
EMA provided this opinion based on safety and tolerability of OCU400 demonstrated in the Phase 1/2 study. The Phase 3 liMeliGhT study will have a sample size of 150 participants primarily in the U.S.—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic (representing multiple gene mutations associated with RP). In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.
The positive scientific advice from EMA is in alignment with U.S. FDA clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial of OCU400. OCU400 is the first gene therapy to enter Phase 3 with a broad RP indication. Previously, OCU400 received broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU.
“We are very grateful to EMA for their collaborative discussions and support in providing a gene-agnostic therapeutic option to RP patients with severe unmet medical need,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “This positive opinion is a critical step in providing our game-changing modifier gene therapies to patients globally.”
The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. With this milestone, OCU400 remains on track for 2026 BLA and MAA approval targets.
About OCU400 OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.
About RP RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.
There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients.
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 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 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, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, 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, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. 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.
Boundless Bio, a biotech company pioneering a new approach to treating cancer, made its public debut on the Nasdaq stock exchange today in a $100 million initial public offering. The Cambridge, Massachusetts company is the latest biotech firm to go public in 2024 after last year’s IPO drought, pricing its shares at $16 each under the ticker symbol “BOLD.”
The $100 million capital raise will provide a major boost to Boundless Bio’s pipeline of experimental cancer therapies that target extrachromosomal DNA (ecDNA), double-stranded DNA molecules that exist outside of chromosomes and can contain amplified oncogenes driving tumor growth.
“EcDNA represents an exciting new frontier in cancer biology and a promising opportunity for therapeutic intervention,” said Zachary Hartman, CEO of Boundless Bio. “With this successful IPO, we are now well-capitalized to advance our novel ecDNA-targeted candidates through clinical trials and hopefully translate this cutting-edge science into meaningful treatments for patients.”
Leading the way for Boundless is BBI-355, the company’s most advanced program that inhibits checkpoint kinase 1, an enzyme involved in ecDNA replication and transcription. BBI-355 is currently being evaluated in the Phase 1/2 POTENTIATE study, with initial data from up to 90 patients expected in the second half of this year.
Not far behind is BBI-825, an oral ribonucleotide reductase inhibitor that targets a different mechanism related to ecDNA biology. This second clinical candidate entered Phase 1/2 testing just last month in the STARMAP trial, with early results anticipated in late 2025.
In addition to developing therapeutics, a portion of the $100 million IPO proceeds will fund Boundless Bio’s efforts to create a diagnostic test called ECHO to detect ecDNA levels in cancer patients’ tumors. The company believes this could enable more precise treatment by identifying patients most likely to respond to ecDNA-targeted therapies.
The successful Nasdaq listing bucks the trend of a biotech IPO market that was essentially frozen in 2023 amid volatile market conditions. But investor sentiment appears to have rebounded in 2024, with Boundless Bio becoming the seventh biotech to go public so far this year.
“This is an incredibly promising time for Boundless Bio and for companies working on novel modalities that could reshape cancer treatment,” said Tricia Lorida, a biotech analyst at SVB Securities. “While ecDNA therapies are still at an early stage, there is certainly excitement around targeting these unique DNA drivers of tumor growth and genomic instability.”
Boundless Bio’s IPO was led by Goldman Sachs, Guggenheim Securities, Piper Sandler, and Leerink Partners as joint book-running managers. The company granted underwriters a 30-day option to purchase up to an additional 937,500 shares at the IPO price, which could raise the total deal proceeds to $115 million if exercised in full.
With the $100 million-plus capital infusion, Boundless Bio is well-positioned to advance its pioneering work in the emerging field of ecDNA biology as the company aims to unlock new therapeutic options for cancer patients. The successful IPO marks an ambitious first step, but much will ride on the clinical data readouts expected over the next couple of years.
The successful $100 million IPO by Boundless Bio could pave the way for more biotech companies to tap the public markets in 2024 as investor appetite appears to be returning. After a dismal 2023 that saw very few biotechs go public, the new year has brought a flurry of IPO activity, with Boundless Bio becoming the seventh biotech to debut on the Nasdaq. Other drug developers waiting in the wings may seize the opportunity to join the IPO queue if market conditions remain favorable. An opening of the IPO window would provide a crucial capital infusion for biotech firms to continue advancing their R&D programs amid a challenging funding environment. While clinical data will ultimately determine the fates of these newly public companies, a reinvigorated IPO market bodes well for biotech innovation lingering in the pipeline.
LOS ALTOS, Calif., March 25, 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 multiple presentations will be delivered on the Company’s product candidates, oxylanthanum carbonate (OLC) and UNI-494, at the 61st European Renal Association (ERA) Congress taking place May 23-26, 2024, in Stockholm, Sweden.
Shalabh Gupta, MD, Chief Executive Officer of Unicycive, commented, “The ERA Congress is one of the most prominent nephrology meetings of the year, and we are excited to deliver presentations on both OLC and UNI-494. In addition to presenting preclinical data supporting both of our programs, we will also be reporting on our two clinical trials in progress. We look forward to participating in this important event.”
Oxylanthanum Carbonate (OLC)
Title:
Enhanced Urinary Phosphorous Reduction: Comparative Study of Oxylanthanum Carbonate and Tenapanor in Rats
UNI-494 Phase I Tolerability and Pharmacokinetics: Trial in Progress
Lead Author:
Guru Reddy, Ph.D., Vice President of Preclinical R&D, Unicycive
Type:
Focused Oral Presentation
Dates/Times:
May 25, 2024 from 12:45 p.m. – 12:50 p.m. CEST
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 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 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 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 late preclinical development for the treatment of 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.
The financial markets are abuzz over WaveDancer, Inc.’s approved merger deal to acquire Firefly Neuroscience, an artificial intelligence company pioneering brain health diagnostics and analytics software. With stockholders from both companies green-lighting the transaction, the stage is set for a new player to emerge in the rapidly evolving neurological healthcare technology space.
WaveDancer (NASDAQ: WAVD), currently an IT services provider to government and commercial clients, is essentially acquiring the capabilities and pipeline of private AI neuroscience firm Firefly through a merger of one of its subsidiaries into the latter. Once the deal closes in Q2 2024, the combined entity will rebrand as Firefly Neuroscience, Inc. and trade under the new ticker AIFF on the Nasdaq Capital Market.
The new Firefly Neuroscience will be laser-focused on commercializing and advancing the development of Firefly’s innovative Brain Network Analytics (BNA) software platform, cleared by the U.S. Food and Drug Administration. Leveraging AI, machine learning, and a massive database of over 17,000 EEG brain scans, the BNA Platform aims to revolutionize the diagnosis and treatment of mental health conditions like depression and anxiety, as well as neurological disorders such as dementia, concussions, and ADHD.
“The WaveDancer favorable shareholder vote is an important step in consummating the merger and reinventing WaveDancer as an AI-enabled neurological health platform,” stated Jamie Benoit, Chairman and CEO of WaveDancer. The company plans to divest its legacy IT services operations to fully concentrate on scaling up Firefly’s neuroscience technology business post-merger.
Firefly’s unique AI platform has already garnered significant attention and investment from the medical community, with the company raising approximately $60 million to date to build out its EEG database, develop the software, secure patents, and attain FDA clearance. Now poised to transition into a publicly-traded commercial entity, Firefly Neuroscience could rapidly accelerate adoption of its solutions among pharmaceutical companies, clinical trials, and medical practitioners globally.
The combined firm will hit the ground running, with Firefly management slated to present at the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference on April 17-18, 2024. This high-profile investor forum will provide an ideal platform to lay out Firefly Neuroscience’s growth strategy and market opportunity to Wall Street.
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…Read More
While the technology is highly specialized, analysts forecast significant potential upside for Firefly’s brain mapping and analytics capabilities across a range of healthcare markets struggling with neurological and mental health challenges. The company’s AI edge in these areas could position it to drive improved patient outcomes, lower costs through earlier interventions, and open new avenues for drug development and therapies.
For WaveDancer shareholders, the transaction marks a bold pivot into a cutting-edge industry riding powerful tailwinds of AI adoption in healthcare settings. Assuming a seamless merger and transition, the company could quickly morph into a promising pure-play on validated, scalable neurotechnology solutions backed by substantial technical assets and IP.
As the deal approaches the finish line, all eyes will be on Firefly Neuroscience’s market debut and ability to execute on the immense growth prospects its AI brain analytics platform presents. Healthcare investors would be wise to keep a close watch as this under-the-radar neuroscience firm prepares to seize a starring role on Wall Street.
In the ever-evolving world of technology, few terms have captured the imagination of investors quite like artificial intelligence (AI). From autonomous vehicles to virtual assistants, AI has permeated nearly every facet of modern life, disrupting traditional business models and creating new opportunities for growth and innovation.
One sector that is increasingly feeling the transformative impact of AI is healthcare. As the industry grapples with challenges such as rising costs, workforce shortages, and the need for more personalized and efficient care, AI is emerging as a powerful tool to address these issues and unlock new frontiers in medicine.
The applications of AI in healthcare are vast and varied, ranging from drug discovery and disease diagnosis to patient monitoring and virtual nursing assistants. At the forefront of this revolution are companies that are harnessing the power of AI to develop cutting-edge solutions and drive technological advancements in the field.
One area where AI is making significant strides is medical imaging and diagnostics. Companies like Enlitic, a pioneer in deep learning for radiology, are developing AI systems that can analyze medical images with unprecedented accuracy, aiding in the early detection of diseases and reducing the risk of misdiagnosis. By automating and enhancing the analysis of X-rays, CT scans, and MRI images, these AI solutions have the potential to improve patient outcomes while reducing the workload on healthcare professionals.
Another promising application of AI in healthcare is drug discovery and development. Traditionally, the process of bringing a new drug to market has been time-consuming and costly, often taking years and billions of dollars in research and clinical trials. However, AI is revolutionizing this process by analyzing vast amounts of data, identifying promising drug candidates, and accelerating the drug discovery pipeline.
Companies are leveraging machine learning algorithms to search through millions of potential drug compounds, predicting their efficacy and safety profiles with remarkable accuracy. This not only speeds up the drug development process but also increases the likelihood of successful clinical trials and faster time-to-market for new therapies.
Beyond drug discovery and medical imaging, AI is also playing a crucial role in personalized medicine and patient care. Companies are developing AI-powered virtual healthcare assistants that can provide personalized medical advice, triage patients, and even monitor chronic conditions remotely. By leveraging natural language processing and machine learning, these AI solutions can offer accessible and affordable healthcare services, particularly in underserved or remote areas.
For investors, the proliferation of AI in healthcare presents both opportunities and challenges. On the one hand, the potential for groundbreaking innovations and disruptive technologies in this sector could translate into significant returns for those who identify and invest in the right companies early on. However, the healthcare industry is also heavily regulated, and navigating the complex web of regulatory approvals and clinical trials can be a significant hurdle for AI-driven healthcare solutions.
Furthermore, as with any emerging technology, there are ethical considerations and potential risks associated with the use of AI in healthcare. Concerns around data privacy, algorithmic bias, and the potential for AI to perpetuate or exacerbate existing healthcare disparities must be carefully addressed to ensure the responsible and equitable deployment of these technologies.
Despite these challenges, the investment community is eagerly watching the AI healthcare space, recognizing the immense potential for transformative innovations and lucrative returns. As the adoption of AI in healthcare continues to accelerate, companies that can successfully navigate the regulatory landscape, mitigate risks, and deliver tangible solutions that improve patient outcomes and healthcare efficiency are likely to emerge as leaders in this burgeoning field.
For savvy investors, the key to capitalizing on the AI healthcare revolution lies in conducting thorough due diligence, understanding the competitive landscape, and identifying companies with robust AI capabilities, strong intellectual property portfolios, and a clear path to commercialization and scalability.
While AI may be a buzzword that often moves markets, in the healthcare sector, it represents a genuine paradigm shift with the potential to save lives, reduce costs, and transform the way we approach healthcare delivery. As such, investors who can separate the hype from the reality and identify the true pioneers in this space may be well-positioned to reap the rewards of this technological revolution.
MALVERN, Pa., Feb. 22, 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 GARDian clinical trial for OCU410ST (AAV5-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease is an orphan blindness disease that affects approximately 35,000 people in the United States alone.
“This is an important clinical milestone for our first-in-class, potential one-time therapy for the treatment of Stargardt disease,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen. “Although Stargardt is one of the most common inherited retinal diseases, there remains no treatment option to address this condition. OCU410ST provides hope to these patients who may eventually lose their vision.”
Up to 10 leading retinal surgery centers across the United States are participating in the GARDian clinical trial. In the first cohort, 3 subjects received 200L single subretinal administration of the low dose (3.75×1010 vg/mL) of OCU410ST.
“I am very pleased to participate in this study and to offer a novel modifier gene therapy option to my patients,” said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at the University of Arizona, College of Medicine—Phoenix. “Until now, we have not had any effective treatment that can prevent the vision loss in patients with Stargardt disease. Now, I believe that this approach can offer a new therapeutic option to address the disease itself.”
The GARDian clinical trial will assess the safety of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose ranging study consisting of three dose levels [low dose (3.75×10E10 vg/mL), medium dose (7.5×10E10 vg/mL), and high dose (2.25×10E11 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated. The Company will continue to provide clinical updates.
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.
Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.
About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.
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.
Shares of pharmaceutical giant AstraZeneca fell over 6% despite the company projecting double-digit growth for 2024. Investors were disappointed by AstraZeneca missing Q4 earnings expectations due to rising costs. However, smaller healthcare firms may offer more upside potential.
AstraZeneca reported fourth quarter core earnings per share of $1.45, below analyst estimates of $1.50. Higher research and development costs weighed on profits. Meanwhile, total revenue edged above expectations at $12.02 billion.
The company expects low double-digit percentage increases in both total revenue and core earnings per share in 2024. This robust guidance is driven by AstraZeneca’s oncology and rare disease drugs.
However, shares dropped as investors focused on the earnings miss and product mix in the latest quarterly results. While AstraZeneca maintains a strong long-term outlook, its scale and mature product portfolio limit rapid growth.
This has led some investors to turn their attention to younger healthcare companies in search of higher growth potential. Smaller biotechs and emerging medtech firms can offer more upside, albeit with higher risk.
For example, cancer immunotherapy developer Silverback Therapeutics went public in late 2020 and has seen its stock price triple over the last year. The company is advancing treatments that harness the body’s immune system to fight cancer.
Other high-growth areas include digital health, where newly public firms like GoodRx are disrupting pharmacy and drug pricing. And healthcare tech provider Oak Street Health has surged over 200% since its 2020 IPO.
These younger healthcare firms tend to have higher volatility compared to big pharmas like AstraZeneca. But their focus on new innovations and faster growth in underpenetrated markets make them appealing for growth-oriented investors.
However, due diligence is required as many of these stocks go on to underperform or even fail. Factors like clinical trial results, regulatory approvals, and market adoption can make or break emerging health stocks.
Diversification across multiple companies can help mitigate the risk. Investing in a healthcare-focused ETF is one method to gain diversified exposure to both mature drugmakers and higher-growth emerging stocks.
Additionally, many biotech and medtech IPOs have been impacted by the 2022/2023 bear market. This offers an opportunity for investors to buy promising stocks at lower valuations.
Overall, AstraZeneca maintains a healthy long-term outlook supported by its deep pipeline of new drugs. But near-term headwinds like rising costs and the latest earnings miss dragged shares lower.
This illustrates how even strong incumbent firms face challenges sustaining rapid growth. For investors seeking higher growth potential, carefully selected emerging healthcare stocks can provide more upside.
However, realizing this potential requires thorough due diligence. Not all emerging companies succeed, making diversification and patience key when investing in new healthcare names. But buying into the right stocks early can result in tremendous gains over the long-term.
The health sector’s constant innovation ensures exciting new companies will continue disrupting incumbents. While mature pharmas like AstraZeneca play a key role in the market, fast-growing upstarts are where outsized returns often lie.
Atlanta, GA, February 6, 2024 – GeoVax Labs, Inc. (Nasdaq: GOVX), a biotechnology company developing immunotherapies and vaccines against cancers and infectious diseases, today announced positive initial safety and immune response findings from its Phase 2 clinical trial at one month following administration of its Covid-19 vaccine, GEO-CM04S1. The trial, evaluating GEO-CM04S1 as a heterologous booster in 63 healthy adults who had previously received the Pfizer or Moderna mRNA vaccine (ClinicalTrials.gov Identifier: NCT04639466), was fully enrolled at the end of Sept 2023.
The study is designed to evaluate the safety and immunogenicity of two GEO-CM04S1 dose levels. The trial remains blinded to dose of vaccine received, with study subjects being followed for a total of one year. To date, there have been no serious adverse events, and adverse events were in line with other routine vaccinations. The immunological responses measured throughout the study period include both neutralizing antibodies against SARS-CoV-2 variants and specific T-cell responses. Consolidated data from all subjects tested one-month post-vaccination, documented statistically significant increases in neutralizing antibody responses against multiple SARS-CoV-2 variants, ranging from the original Wuhan strain through Delta and Omicron XBB 1.5; additional testing against the JN.1 variant is underway.
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. GEO-CM04S1 encodes for both the spike (S) and nucleocapsid (N) antigens of SARS-CoV-2 and is specifically designed to induce both antibody and T cell responses to those parts of the virus less likely to mutate over time. The more broadly functional engagement of the immune system is designed to protect against severe disease caused by continually emerging variants of Covid-19. Vaccines of this format should not require frequent and repeated modification or updating. These latest findings lend support to previously published findings in cell transplant patients of the ability of GEO-CM04S1 to stimulate functional antibody responses against a broad array of evolving SARS-CoV-2 virus variants (Chiuppesi et al, Vaccines, Sept 2023).
Kelly McKee, Jr., MD, MPH, GeoVax Chief Medical Officer, stated, “There is a critical need to address the recognized shortcomings of currently approved SARS-CoV-2 vaccines. Annual (or even more frequent) vaccination in a seemingly never-ending race to keep pace with this rapidly evolving virus is an unsustainable strategy. GEO-CM04S1 continues to demonstrate the ability to elicit broadly reactive functional antibodies in conjunction with robust and durable T-cell responses, offering the prospect of a next-generation solution to address these shortcomings.”
“We are thrilled by these data and our investigational vaccine designed to protect against severe disease caused by emerging variants of Covid-19,” said David Dodd, GeoVax Chairman and CEO. “These interim data reinforce our resolve to bring our expertise in the development of innovative vaccines to address critical public health needs using new approaches and technologies. We look forward to providing further updates regarding the successful progress of the clinical development of GEO-CM04S1.”
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.
