Tag: Biotechnology

AbbVie’s Acquisition of Landos Biopharma Highlights Potential in Small-Cap Biotech

Posted on by slightbourne@noblecapitalmarkets.com

In a strategic move that could have significant implications for the small-cap biotech sector, pharmaceutical giant AbbVie Inc. (NYSE: ABBV) announced its acquisition of Landos Biopharma, Inc. (NASDAQ: LABP), a clinical-stage biopharmaceutical company focused on developing novel oral therapeutics for autoimmune diseases. The deal, valued at approximately $212.5 million including contingent value rights, underscores the growing interest and potential in the small-cap biotech space, particularly in the field of inflammatory and autoimmune diseases.

Under the terms of the agreement, AbbVie will acquire Landos at $20.42 per share in cash upon closing, plus a contingent value right of up to $11.14 per share, subject to the achievement of a clinical development milestone. The acquisition is expected to close in the second quarter of 2024, subject to customary closing conditions, including approval by Landos’ stockholders.

The primary asset driving this deal is NX-13, Landos’ lead investigational asset and a first-in-class, oral NLRX1 agonist with a bimodal mechanism of action. NX-13 is currently in Phase 2 clinical trials for the treatment of ulcerative colitis (UC), a chronic inflammatory bowel disease affecting millions worldwide.

“With this acquisition, we aim to advance the clinical development of NX-13, a differentiated, first-in-class, oral asset with the potential to make a difference in the lives of people living with ulcerative colitis and Crohn’s disease,” said Roopal Thakkar, M.D., AbbVie’s senior vice president and chief medical officer, global therapeutics.

NX-13’s unique bimodal mechanism of action, which is both anti-inflammatory and facilitates epithelial repair, could provide a novel approach to treating UC and other inflammatory bowel diseases. If successful, it could address a significant unmet need in this therapeutic area.

The acquisition underscores AbbVie’s commitment to strengthening its portfolio in inflammatory and autoimmune diseases, which represent a substantial market opportunity. According to estimates, the global inflammatory bowel disease treatment market is projected to reach $8.6 billion by 2027, driven by factors such as increasing prevalence, rising healthcare expenditure, and a growing focus on developing targeted therapies.

For small-cap investors, this deal highlights the potential value and attractiveness of emerging biotech companies with promising pipeline candidates. As larger pharmaceutical companies seek to bolster their portfolios and drive innovation, strategic acquisitions of small-cap biotechs with compelling assets can provide attractive exit opportunities and significant returns for investors.

This acquisition also comes at an opportune time, as Noble Capital Markets’ upcoming virtual healthcare event on April 17-18 will showcase emerging growth companies in the healthcare, biotech, and medical device industries. Investors interested in exploring opportunities in the small-cap biotech space should mark their calendars for this event, which promises to provide valuable insights and potential investment prospects in this dynamic sector.

With the rising interest in novel therapies for inflammatory and autoimmune diseases, the AbbVie-Landos deal serves as a reminder of the potential value that can be unlocked in the small-cap biotech realm. As larger players seek to fortify their pipelines, the spotlight on promising small-cap innovators is likely to intensify, presenting exciting opportunities for investors in this space.

Eledon Pharmaceuticals (ELDN) – First Transplant Of A Pig Kidney Uses Tegoprubart To Prevent Immune Rejection

Posted on by cpereira@noblefcm.com


Friday, March 22, 2024

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.

First Transplant Of A Genetically Modified Kidney To A Human. Eledon announced that tegoprubart, its drug in development to prevent organ transplant rejection, was used in the first transplant of an engineered pig kidney into a human patient. The kidney was genetically engineered by eGenesis, Inc, a company that has been collaborating with Eledon. Tegoprubart is currently in a Phase 2 clinical trial testing it against tacrolimus in prevention of kidney transplant rejection.

Eledon Has A Research Collaborated With eGenesis. The pig kidney was engineered by eGenesis, Inc, a company that formed a research collaboration with Eledon in January 2023. eGenesis has proprietary technology for engineering genetic changes to animal organs to make them compatible for transplantation into humans. As described in our Research Note on October 16, these genetic changes reduce the cross-species immunological barriers. The agreement allows eGenesis to use tegoprubart in its xenotransplantation research.


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Release – MAIA Biotechnology Welcomes Prominent Medical Oncology Scientist Dr. Saadettin Kilickap to Its Scientific Advisory Board

Posted on by aweinsoff@channelchek.com

Research News and Market Data on MAIA

March 21, 2024 3:00pm EDT

  • Acclaimed academic researcher has led 40+ multicenter phase 2 and phase 3 clinical studies

CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced the appointment of Professor Saadettin Kilickap, M.D. to its Scientific Advisory Board (SAB).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240321173734/en/

Professor Saadettin Kilickap, M.D., Scientific Advisor to MAIA Biotechnology (Photo: Business Wire)

Dr. Kilickap is a professor at the Istinye University Faculty of Medicine, Department of Medical Oncology, Liv Hospital in Turkey. His research focuses on medical oncology and cancer epidemiology, including solid tumors such as lung cancer, breast cancer, melanoma, and gastrointestinal system cancers, as well as targeted therapies and immunotherapy.

“Saadettin has served as principal or sub-investigator in more than 40 national and international multi-center phase 2 and phase 3 studies, many of which were related to lung cancer,” said Chief Executive Officer Vlad Vitoc, M.D. “He is a prominent voice on medical oncology and cancer epidemiology, with a special interest in quality of life for cancer patients. We are delighted to welcome him as Scientific Advisor as we begin to clear major clinical inflection points this year and progress with our groundbreaking cancer research.”

Prior to his current appointment, Dr. Kilickap was a professor at the Preventive Oncology Department of Hacettepe University Cancer Institute of Turkey. Earlier he worked in the Department of Hematology-Oncology at Regensburg University in Germany and was a faculty member at the Sivas Cumhuriyet University Faculty of Medicine, Department of Internal Medicine, in Turkey.

Dr. Kilickap graduated with honors from Gazi University Faculty of Medicine and went on to complete his internal medicine residency training at Hacettepe University Faculty of Medicine, both located in Ankara, Turkey. He completed his fellowship training at Hacettepe and became a medical oncology specialist in 2009. In the same year, he graduated from the Cancer Epidemiology Master’s Program at the Hacettepe University Oncology Institute, Department of Preventive Oncology.

Dr. Kilickap has authored more than 240 scientific articles published in international peer-reviewed journals, and delivered more than 50 poster presentations at international congresses.

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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240321173734/en/

Investor Relations Contact
+1 (872) 270-3518
ir@maiabiotech.com

Source: MAIA Biotechnology, Inc.

Released March 21, 2024

AstraZeneca Makes $2.4 Billion Bet on Next-Gen Cancer Radioconjugates

Posted on by slightbourne@noblecapitalmarkets.com

In a bold move to fortify its oncology pipeline, pharmaceutical giant AstraZeneca (NASDAQ: AZN) has agreed to acquire clinical-stage biotech Fusion Pharmaceuticals (NASDAQ: FUSN) for up to $2.4 billion. The deal gives AstraZeneca full access to Fusion’s pioneering radioconjugate (RC) therapies and expertise as it aims to transform cancer treatment and unseat traditional chemotherapy and radiation regimens.

Fusion specializes in developing a promising new class of precision oncology drugs called RCs, which dispense powerful, targeted radiation directly to cancer cells via targeting molecules like antibodies. By delivering potent radioisotope payloads to tumors in this manner, RCs may improve upon external beam radiation’s limitations and indiscriminately toxic effects.

Under the agreed terms, AstraZeneca will pay $21 per share in cash upfront to acquire all outstanding Fusion shares, valuing the biotech at approximately $2 billion. This headline price represents a staggering 97% premium over Fusion’s closing price prior to deal announcement. AstraZeneca has also committed up to $3 per share in additional contingent value rights tied to a future regulatory milestone, which could push the total deal value to $2.4 billion if achieved.

For AstraZeneca, the acquisition paves the way for a major expansion into promising RC therapeutics, which could revolutionize how cancers are treated in the future. The crown jewel of the deal is FPI-2265, Fusion’s lead RC candidate that uses the alpha particle-emitting isotope actinium-225 to target PSMA proteins in metastatic castration-resistant prostate cancer. FPI-2265 is already in Phase 2 clinical trials with early data expected in 2024.

Beyond this advanced asset, AstraZeneca gains control of Fusion’s broader pipeline of RC programs and candidates across multiple solid tumor types. Just as importantly, the transaction provides AstraZeneca with Fusion’s specialized R&D capabilities, manufacturing infrastructure, and actinium-225 supply chain specifically tailored for developing these next-wave radiotherapeutics.

This strategic acquisition doubles down on AstraZeneca’s burgeoning radio-isotope therapy initiatives. The pharma giant already has a collaboration with Fusion exploring lung cancer applications using one of its RC molecules. By bringing Fusion’s RC therapy capabilities fully in-house, AstraZeneca can now swiftly integrate and scale up this cutting-edge treatment modality across its industry-leading oncology portfolio.

For Fusion investors, the buyout represents a lucrative exit at a substantial premium, especially compared to the company’s $300 million market cap prior to deal reports. While always bittersweet to see a promising biotech get absorbed, Fusion’s technology and team are now set to be fueled by abundant AstraZeneca resources in pursuing RC breakthroughs.

The transaction, expected to close in Q2 2024 pending customary approvals, foreshadows a future where RCs could potentially supplant chemotherapy and radiotherapy as more precise, less toxic foundational cancer regimens. While still an emerging field, AstraZeneca’s bold multi-billion dollar investment signals its confidence in harnessing RCs’ unique advantages over traditional oncology treatments.

As AstraZeneca executes an ambitious pivot toward next-generation RC therapies, biotech and pharma investors would be wise to monitor this rapidly evolving space. The acquisition of Fusion continues to position the pharma titan at the vanguard of replacing archaic cancer protocols with targeted radioconjugate precision medicines.

Release – Ocugen, Inc. Appoints Huma Qamar, MD, MPH As Chief Medical Officer

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

March 18, 2024

MALVERN, Pa., March 18, 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 the appointment of Dr. Huma Qamar as Chief Medical Officer (CMO).

“I’m very pleased to name Dr. Qamar as the CMO at Ocugen,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This appointment recognizes her invaluable contributions to building a strong clinical team, overseeing a well-conducted Phase 1/2 gene therapy trial, and establishing relationships with leading medical and academic institutions and key opinion leaders.”

Dr. Qamar is a distinguished healthcare professional with a rich background in clinical research, having contributed her expertise to esteemed Ivy League institutions including Yale University, Harvard University, and University of Pennsylvania. Her expertise encompasses the development of Phase I-IV clinical protocols and execution of clinical studies, FDA inspections, and effective leadership of medical affairs teams.

“Having been a part of Ocugen’s mission and vision for more than three years now, it’s a pleasure to lead Ocugen’s clinical development at this pivotal time,” said Dr. Qamar. “I am committed to addressing patients’ unmet medical needs and bringing novel modifier gene therapy products to the market globally.”

Dr. Qamar has expertise in multiple therapeutic areas, including gene and cell therapy, vaccines, oncology (Heme-Onc, CAR-T, rare tumors, sarcoma, melanoma, women’s health, GU & GI, fetal oncology), rheumatology, dermatology, neurology, cardiology, hepatology, and infectious diseases.

“Dr. Qamar has a deep understanding of Ocugen’s ongoing clinical trials and a close working relationship with the clinical and regulatory teams, both of which are fundamentally important to the success of Ocugen’s programs,” said Dr. Prabhavathi Fernandes, Independent Lead Director, Ocugen board. “I’m confident that her determination and dedication will bring the Company’s first-in-class candidates through the upcoming clinical milestones to deliver on Ocugen’s long-term strategy.”

Prior to joining Ocugen, Dr. Qamar was Senior Vice President, Head of R&D at FSD Pharma—demonstrating her commitment to advancing scientific innovation and pharmaceutical development.

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. 

Contact: 
Tiffany Hamilton 
Head of Communications 
Tiffany.Hamilton@ocugen.com  

New Eli Lilly-Amazon Deal Signals Emerging Opportunities in Direct-to-Consumer Pharmaceuticals

Posted on by slightbourne@noblecapitalmarkets.com

The newly announced partnership between pharmaceutical giant Eli Lilly and e-commerce behemoth Amazon to enable direct-to-consumer medication delivery is sending shockwaves through the biotech and healthcare sectors. The deal, which allows customers to receive select Eli Lilly prescription drugs like diabetes, migraine, and weight-loss treatments via Amazon’s online pharmacy, represents a major shift in how pharmaceutical companies get products into the hands of consumers

For emerging biotech and healthcare companies watching this space, the Eli Lilly-Amazon partnership illuminates massive growth opportunities in the burgeoning direct-to-consumer pharmaceutical market. Cutting out the middlemen of insurance providers and brick-and-mortar pharmacies enables pharma companies to get closer to patients and potentially earn higher margins.

Under the partnership revealed this week, patients can receive Eli Lilly medications prescribed through the LillyDirect online platform or by their regular doctor, with Amazon handling the fulfillment and two-day delivery logistics. Axios’ Jacob Gardner points out this allows Eli Lilly “to reach more patients directly and sidestep more traditional pharmaceutical sales constraints.”

The collaboration helps both industry titans accomplish key objectives. For Eli Lilly, it expands their direct-to-consumer reach at a pivotal time following the approval of blockbuster weight-loss drug Zepbound last November. Amazon, meanwhile, continues growing its healthcare presence following the acquisition of PillPack and launch of Amazon Pharmacy in 2020.

Executives at emerging biotech and pharmaceutical companies would be wise to study this latest deal’s blueprint. By partnering with logistics giants like Amazon, FedEx, or UPS on the shipping side or digital health platforms on the consumer-facing end, they could unlock highly lucrative direct-to-consumer sales channels.

Beyond cutting out middlemen that take a cut of sales, direct-to-consumer pharma models can foster stronger patient relationships, bolster brand loyalty, and provide a wealth of data and analytics on consumer behaviors. Those insights allow companies to precisely tailor marketing and pricing strategies to drive further growth.

From the investor perspective, directly delivering cutting-edge treatments straight to patient doorsteps holds massive upside potential. Drug developers can keep more of the profits by circumventing insurance providers. But investing in the right direct-to-consumer pharmaceutical plays requires careful due diligence.

Investors need to scrutinize logistics capabilities, consumer marketing and branding strengths, and data analytics competencies in evaluating these emerging opportunities. The biggest winners will have a clear advantage in one or more of those mission-critical areas.

The overarching theme is clear – by cutting out the tangle of middlemen in the traditional pharmaceutical ecosystem, innovative companies embracing the direct-to-consumer model could potentially earn higher revenues, margins, and valuations. The ripple effects of the Eli Lilly-Amazon deal are likely just beginning for the healthcare investing space.

For investors willing to conduct thorough research and identify the pioneers, the emerging direct-to-consumer pharmaceutical market could birth the next generation of blockbuster biotech and healthcare companies.

Learn more about Noble Capital Markets’ Emerging Growth Virtual Healthcare Equity Conference on April 17-18 here.

Release – PDS Biotech Announces Publication of Preclinical Research and Grant of U.S. Composition of Matter Patent for Infectimune®

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

Preclinical research published in Vaccines demonstrates Infectimune® significantly improved quantity and quality of potent multifunctional CD4 T cells compared to vaccine adjuvants

Patent for composition of matter and use of Infectimune® based influenza vaccines granted in the United States

PRINCETON, N.J., March 13, 2024 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or “the Company”), a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer immunotherapies and infectious disease vaccines based on the Company’s proprietary T cell-activating platforms, today announced the publication of preclinical research and a patent granted by the United States Patent and Trademark Office (USPTO) that strengthen the foundation of the Company’s infectious disease vaccine platform Infectimune®.

Preclinical research published in Vaccines demonstrated that the recombinant protein influenza vaccine Flublok® combined with Infectimune® (R-DOTAP) significantly promoted improved quantity and quality of potent multifunctional CD4 T cells when compared to infectious disease adjuvants. The research concluded that Infectimune® is a leading candidate for use in the next generation of preventive vaccines that may provide more effective and broader protection than current vaccines allow. Infectimune® is being used in PDS0202, the Company’s universal influenza vaccine intended to provide broad protection against multiple flu strains.

“Recently published preclinical research shows that Infectimune® overcomes the limitations of current vaccines by promoting CD4+ T cells, known to target many viral proteins and highly conserved regions of viruses,” said Frank Bedu-Addo, Ph.D., President and Chief Executive Officer of PDS Biotech. “Prior preclinical studies of Infectimune® based influenza vaccines have also shown its potential to provide several advantages over existing influenza vaccines through the induction of broad cross-protective immunity to different subtypes of influenza. We believe this represents a significant advancement in the potential to protect against influenza, its mutations or antigenic drifts.”

Additionally, the USPTO granted U.S. Patent Number 11,904,015 titled, “Vaccine Compositions and Methods of Use,” directed to vaccine compositions employing the Infectimune® platform and influenza antigens to elicit T cells and antibodies in February 2024. The patent protects compositions containing the Infectimune® platform and influenza antigens and methods of using the Infectimune® platform with pathogenic antigens generally.

“This patent adds to the intellectual property governing our novel investigational universal influenza vaccine,” continued Dr. Bedu-Addo. “Possessing multiple layers of intellectual property for our assets is an important value driver for PDS Biotech and is a key component of our business strategy.”

About Infectimune®
Infectimune® is a novel investigational immune activating platform that generates broad and robust antibody and T-cell responses that provide durable protection against infectious disease. Infectimune® based vaccines are given by intramuscular injection and generate robust and durable protection against infectious agents in preclinical studies. Infectimune® based vaccines have demonstrated safety in preclinical studies and appear to provide more robust and longer-lasting protection against infectious disease.

About PDS Biotechnology
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune®, Versamune® plus PDS01ADC and Infectimune® T cell-activating platforms. We believe our targeted immunotherapies have the potential to overcome the limitations of current immunotherapy approaches. Our Versamune® platform activates the right type, quantity and potency of tumor attacking T cells. Our IL-12 fused antibody drug conjugate (PDS01ADC) is designed to target the tumor to promote suppression of the tumor’s defenses while promoting T-cell activity in the tumor. To date, Versamune® HPV16 (PDS0101), PDS01ADC and PDS0101 co-administered with PDS01ADC, our lead clinical candidates, have demonstrated the ability to shrink tumors and/or stabilize disease when used as single agents or in combination with approved therapies in patients with a broad range of solid tumors in multiple Phase 2 clinical trials. We plan to advance our lead program into a pivotal trial for the treatment of recurrent/metastatic HPV16-positive head and neck cancer. Our Infectimune® based vaccines have demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T-cell responses, including long-lasting memory T-cell responses in pre-clinical studies to date. To learn more, please visit www.pdsbiotech.com or follow us on X at @PDSBiotech.

Forward Looking Statements
This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS0101, PDS0203 and other Versamune® and Infectimune® based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS0101, PDS0203 and other Versamune® and Infectimune® based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to the Company’s currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.  

Versamune® and Infectimune® are registered trademarks of PDS Biotechnology Corporation. Flublok® is a registered trademark of Protein Sciences Corporation.

Investor Contact:
Mike Moyer
LifeSci Advisors
Phone +1 (617) 308-4306
Email: mmoyer@lifesciadvisors.com

Media Contact:
Gina Mangiaracina
6 Degrees
Phone +1 (917) 797-7904
Email: gmangiaracina@6degreespr.com

Release – Ocugen, Inc. Announces Dosing Completion of Subjects with Geographic Atrophy In Cohort 1 Of Phase 1/2 Clinical Trial Evaluating The Safety And Efficacy Of OCU410

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

March 13, 2024

PDF Version

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. 

Contact: 
Tiffany Hamilton 
Head of Communications 
Tiffany.Hamilton@ocugen.com  

Release – Unicycive Therapeutics Delivers Both An Oral And Poster Presentation On UNI-494 At The AKI and CRRT Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

March 13, 2024 7:03am EDT Download as PDF

– 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.

Investor Contact:

ir@unicycive.com
(650) 543-5470

SOURCE: Unicycive Therapeutics, Inc.

Source: Unicycive Therapeutics, Inc.

Released March 13, 2024

Cadrenal Therapeutics (CVKD) – FY2023 Reported With Continued Progress Toward The Phase 3 Tecarfarin Trial

Posted on by cpereira@noblefcm.com


Wednesday, March 13, 2024

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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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. 

Unicycive Therapeutics (UNCY) – Trial Meets Milestone With Complete Enrollment

Posted on by cpereira@noblefcm.com


Monday, March 11, 2024

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.


Get the Full Report

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. 

Healthcare Investment Ideas on Display at the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference

Posted on by cpatches

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Watch the Presentation and Q&A Replays

  • 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.

Check out the calendar of upcoming in-person non-deal roadshows here.

Sign up to receive more information on Noble’s other virtual conferences here.

What to Expect

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.

How to Register

Limited presenting slots are available

Publicly traded companies in the healthcare space can submit their registration details here.

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Release – Unicycive Therapeutics Completes Enrollment In Pivotal Clinical Trial For Oxylanthanum Carbonate (OLC)

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

March 07, 2024 4:12pm EST Download as PDF

TOPLINE DATA EXPECTED IN Q2, 2024

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.

Investor Contact:

ir@unicycive.com
(650) 543-5470

SOURCE: Unicycive Therapeutics, Inc.

Source: Unicycive Therapeutics, Inc.

Released March 7, 2024