Tag: Biotechnology

Unicycive Therapeutics (UNCY) – Data Presentations For OLC and UNI-494 Trials Presented

Posted on by cpereira@noblefcm.com


Wednesday, May 29, 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.

Data From Both Products Shows Clinical Progress. Unicycive presented data at the European Renal Association (ERA) Congress for OLC and UNI-494, its two products currently in clinical development. Earlier this month, the company presented data from its OLC bioequivalence study and a market survey study on phosphate binder compliance at the National Kidney Foundation Spring Clinical Meeting.

OLC Clinical Data Presented At National Kidney Foundation Meeting. Unicycive presented data from its Phase 1 bioequivalence study comparing OLC with lanthanum carbonate, the active compound in the phosphate binder Fosrenol. The second poster presented data from a survey of renal dieticians who help manage patients’ diet and phosphate levels. The survey is consistent with prior data showing that daily pill burden is a factor in patient compliance, and that OLC’s reduced pill burden could lead to better compliance and patient outcomes. 


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. 

Release – Ocugen Set to Join Russell 3000® Index Effective June 28, 2024

Posted on by slightbourne@noblecapitalmarkets.com

Research News and Market Data on OCGN

MALVERN, Pa., May 28, 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 its expected upcoming inclusion in the Russell 3000® Index, according to preliminary Russell reconstruction information posted on the FTSE Russell website. The newly reconstructed index will take effect after the market closes on June 28, 2024.

“Inclusion of Ocugen to the Russell 3000® Index is our latest milestone, adding to what has already been a transformational year for the Company with three of our game-changing modifier gene therapies targeting blindness diseases—both rare and those affecting millions—in clinical trials,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen. “This ranking signifies the value of our pipeline, including the recently initiated Phase 3 liMeliGhT clinical trial of OCU400 for broad retinitis pigmentosa, and robust growth strategy, supporting our efforts to enable long-term shareholder value, garner significant visibility of Ocugen within the investment community, and broaden our shareholder base.”

The annual Russell 3000® Index reconstitution measures the performance of the largest 3,000 U.S. companies representing approximately 96% of the investable U.S. equity market as of Tuesday, April 30.

Membership in the U.S. Russell 3000® remains in place for one year and means automatic inclusion in the appropriate growth and value style indexes. FTSE Russell determines membership for its Russell indexes primarily by objective, market-capitalization rankings, and style attributes.

Russell indexes are widely used by investment managers and institutional investors for index funds and as benchmarks for active investment strategies. Russell’s U.S. indexes serve as the benchmark for about $10.5 trillion in assets as of the close of December 2023. Russell indexes are part of FTSE Russell, a leading global index provider.

About FTSE Russell:
FTSE Russell is a leading global provider of benchmarking, analytics, and data solutions for investors, giving them a precise view of the market relevant to their investment process. A comprehensive range of reliable and accurate indexes provides investors worldwide with the tools they require to measure and benchmark markets across asset classes, styles, or strategies.

FTSE Russell index expertise and products are used extensively by institutional and retail investors globally. For over 30 years, leading asset owners, asset managers, ETF providers and investment banks have chosen FTSE Russell indexes to benchmark their investment performance and create ETFs, structured products, and index-based derivatives.

FTSE Russell is focused on applying the highest industry standards in index design and governance, employing transparent rules-based methodology informed by independent committees of leading market participants. FTSE Russell fully embraces the IOSCO Principles, and its Statement of Compliance has received independent assurance. Index innovation is driven by client needs and customer partnerships, allowing FTSE Russell to continually enhance the breadth, depth and reach of its offering.

FTSE Russell is wholly owned by London Stock Exchange Group. For more information, visit: https://www.lseg.com/en/ftse-russell.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the Company’s expected inclusion in the Russell 3000 ® Index, 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 changes may be made to the preliminary Russell reconstruction lists prior to finalization, 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.

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

Behind the Scenes of Biotech’s Battle for the Billion-Dollar Diet Pill

Posted on by slightbourne@noblecapitalmarkets.com

For biotech investors scouring for the next big opportunity, the massive manufacturing expansions underway at pharma giant Eli Lilly (LLY) are worth a close look. The company just announced another staggering $5.3 billion investment into a key production facility in Indiana to crank up supply of its blockbuster obesity and diabetes drugs.

This commitment brings Lilly’s total investment at the Lebanon, Indiana site to an incredible $9 billion – representing the firm’s largest-ever manufacturing bet in its nearly 150-year history. It highlights the huge demand that Lilly’s game-changing medications like Mounjaro and Zepbound are facing from physicians and patients alike.

For small biotech firms developing the next generation of weight loss, diabetes and metabolism therapies, Lilly’s supply chain moves send an important signal – this market is headed for explosive growth in the coming years. Companies sitting on promising pipeline candidates could emerge as attractive buyout targets.

At the heart of Lilly’s expansion plans are its incretin drugs, which mimic gut hormones to suppress appetite and regulate blood sugar. Mounjaro, approved for diabetes, and Zepbound, greenlit for chronic weight management, both contain the active ingredient tirzepatide.

Since their launches, demand for these effective and convenient once-weekly injectable treatments has far outstripped supply. Shortages have been widespread in the U.S. as Lilly raced to build out its production infrastructure.

The new $9 billion Indiana campus will be instrumental in increasing Lilly’s capacity to manufacture tirzepatide at scale. When fully operational in 2028, it will employ around 900 skilled workers including scientists, engineers and technicians.

But this plant is just one piece of Lilly’s supply chain mobilization for incretin drugs. Since 2020, the company has plowed over $18 billion into building, acquiring and expanding manufacturing sites in the U.S. and Europe. New facilities are also coming online in North Carolina, Ireland and Germany through 2026.

These investments are already paying dividends. On its latest earnings call, Lilly hiked its 2023 revenue guidance by $2 billion, citing greater visibility into ramping up production of Mounjaro, Zepbound and its incretin pipeline over the remainder of the year.

For small biotechs, the supply chain frenzy at Lilly underscores the commercial opportunity in obesity, diabetes and metabolism. With over 40% of U.S. adults classified as obese, safe and effective chronic weight management regimens like Lilly’s incretin franchise could disrupt a massive global market worth billions annually.

Take a moment to look at more emerging growth biotechnology companies by taking a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage list.

The manufacturing expansions suggest appetite for these therapies will continue to surge, fueling demand for the next generation of medications offering better efficacy, tolerability and dosing schedules. Smaller drug developers operating in this space could become prime M&A candidates as deep-pocketed pharmas look to build out their obesity and diabetes portfolios.

Case in point: Lilly itself acquired Zepbound through its $8 billion buyout of Protunor Biopharma in 2022. Several major deals have already reshaped the incretin drug landscape in recent years, including Pfizer’s $6.7 billion purchase of Akero Therapeutics for its NASH/diabetes pipeline.

With its bold investments, Lilly is putting its money where its mouth is when it comes to obesity and metabolic disease. For lean biotechs advancing the next wave of therapies in this booming treatment category, that could spell opportunity knocking in the form of lucrative buyout offers or partnerships down the line.

Keep an eye on this space as Lilly’s supply chain moves underscore that the fight against fat is only just beginning for the pharmaceutical industry.

Biogen’s Bold $1.8B Kidney Disease Treatment Acquisition

Posted on by slightbourne@noblecapitalmarkets.com

Biogen Inc. is doubling down on novel therapies for rare diseases, announcing an acquisition of Human Immunology Biosciences that could be valued at up to $1.8 billion. The deal gives Biogen full rights to HI-Bio’s lead drug candidate felzartamab, which is being studied for several chronic kidney conditions with large unmet medical needs.

The transaction reflects Biogen’s strategic shift under CEO Christopher Viehbacher to diversify beyond its core neuroscience franchise. Just months after the $6.5 billion buyout of kidney disease specialist Reata Pharmaceuticals, Biogen is opening its checkbook again to beef up its pipeline of potential rare disease medicines.

HI-Bio’s felzartamab has completed mid-stage trials for two types of kidney disorders – primary membranous nephropathy and transplant glomerulopathy where the immune system attacks a transplanted organ. Importantly, it is also being evaluated for IgA nephropathy, a leading cause of kidney failure with no approved treatments available.

For Biogen, the deal provides another shot on goal as it navigates an uncertain period. While its newly-launched Alzheimer’s drug Leqembi has shown promise, the company was forced to abandon its previous Alzheimer’s treatment Aduhelm after years of controversy. Biogen’s older multiple sclerosis franchises are facing rising competitive threats as well.

The HI-Bio acquisition gives Biogen added pipeline diversification into nephrology and autoimmune diseases. Felzartamab has a unique approach, as it is an anti-FcRn antibody that targets pathogenic IgG antibodies which can damage kidneys and other organs.

If felzartamab can demonstrate positive efficacy and safety in broader Phase 3 testing, it could eventually have multi-billion dollar peak sales potential across its targeted kidney indications according to analyst forecasts. However, there is no guarantee of clinical or regulatory success.

From a financial perspective, Biogen is paying $1.15 billion upfront for private HI-Bio, along with contingent value rights worth up to $650 million if certain development and commercial milestones are achieved. This is relatively modest compared to Biogen’s $6.5 billion acquisition of Reata announced in February.

The HI-Bio deal continues Biogen’s aim to revamp its R&D pipeline through a series of bold acquisitions and partnerships under Viehbacher. The company is betting that assembling a portfolio of high-risk, high-reward clinical candidates for diseases like Alzheimer’s and kidney disorders will ultimately pay off.

For the healthcare sector and public markets, Biogen’s aggressive business development approach is emblematic of the ongoing consolidation wave. With rising costs of drug development and payer pricing pressures, large biopharma companies are increasingly looking to acquisitions of smaller, more focused biotechs to source external innovation.

While Biogen’s M&A strategy carries substantial financial risk, the HI-Bio deal gives it a promising asset that could reshape treatment for serious kidney diseases if it can overcome the high hurdle of clinical success. For healthcare investors, absorbing Biogen’s evolving pipeline story will be crucial in evaluating the company’s future growth prospects.

Want small cap opportunities delivered straight to your inbox?

Release – Tonix Pharmaceuticals Announces Key Findings from Initial Phase of EVERSANA Market Opportunity Analysis for Tonmya™ for the Management of Fibromyalgia

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

CHATHAM, N.J., May 21, 2024 (GLOBE NEWSWIRE) —  Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, announced today that EVERSANA® Life Science Services, LLC, a leading provider of commercialization services to the global life sciences industry, completed the initial phase of an assessment of the U.S market opportunity for TonmyaTM (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets), a potential new first line therapy for the management of fibromyalgia. Tonix previously announced that EVERSANA was selected to support the launch strategy and commercial planning of Tonmya. Specifically, EVERSANA is working with Tonix to assess the fibromyalgia landscape and help plan an efficient go-to-market (GTM) strategy.

“The primary research and analysis conducted by EVERSANA provides valuable insights and informs the vision for the commercialization of Tonmya,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “We are excited to refine our business strategy for the anticipated launch of Tonmya in 2025. It has been approximately fifteen years since patients suffering with fibromyalgia have been provided a new therapeutic option. The market research conducted by EVERSANA with physicians indicates a continued high unmet need, with the need ranked > 4.0 on a 5-point scale.”

Dr. Lederman continued, “We were surprised to learn that opioids dominate the fibromyalgia prescription market, despite the well-known risks of dependency. Opioids are prescribed to more patients following fibromyalgia diagnosis than all the FDA-approved fibromyalgia drugs combined. If approved, we believe that Tonmya can provide a non-opioid, centrally-acting solution for fibromyalgia patients. This is corroborated by EVERSANA’s primary market research in which physicians indicated median interest of 4.0 on a 5-point scale and intended use of Tomya in 40% of their fibromyalgia patients, upon approval.1 We look forward to submitting the NDA for Tonmya in the second half of this year and expect an FDA decision on approval in 2025.”

EVERSANA’s analysis of the market size was based on medical claims databases and epidemiological studies on prevalence and diagnosis2. Primary research with physicians was conducted to test the potential adoption of Tonmya based on its activity and tolerability.  

Key findings from claims data analyses2:

  • ~2.7 million adults in the U.S. currently diagnosed and treated for fibromyalgia.
    • ~90 percent of patients diagnosed with fibromyalgia are female.
  • Percentage of fibromyalgia patients who were prescribed certain drugs3:
    • FDA-approved fibromyalgia drug prescriptions:
      1. Duloxetine (generic Cymbalta®): 23%, pregabalin (generic Lyrica®): 13%, and Savella® (milnacipran): 1%
    • Off-label drug prescriptions:
      1. Gabapentin: 26%, hydroxycodone: 20%, cyclobenzaprine: 18%, oxycodone: 19%, tramadol: 13%, meloxicam: 12%, and amitriptyline: 6%
    • Off-label opioids prescriptions:
      1. Hydroxycodone: 20%, oxycodone: 19%, and tramadol: 13%
  • Targeted prescriber strategy for launch.
    • High prescribers are rheumatologists, pain medicine specialists, primary care physicians, neurologists, and psychiatrists.
  • ~50 percent of diagnosed fibromyalgia patients are covered by Medicare.
    • Medicare population stands to benefit from a decrease in out-of-pocket prescription drug liability to $2,000 per year when the changes from the Inflation Reduction Act are enacted in 2025.

Key findings from primary physician research4:

  • High level of dissatisfaction with currently prescribed drugs.
    • Physicians interviewed estimate that 85% of their patients fail first-line therapy due to efficacy and tolerability issues and 79% of their patients are on multiple therapies.
    • Prescribers indicate high unmet need in fibromyalgia, ranked >4.0 on a 5-point scale.
  • High level of interest in Tonmya from physicians interviewed as a potential new treatment option based on favorable activity and tolerability profile observed in Tonmya fibromyalgia clinical studies.
    • Median interest in Tonmya of 4 on a 5-point scale.
    • Physicians indicated intended use of Tonmya in 40% of their fibromyalgia patients.1

Tonmya is a centrally acting, non-opioid medication. As previously announced, Tonix’s second statistically significant Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results (p=0.001 or better) were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function.

Tonix plans to submit an NDA to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for Tonmya for the management of fibromyalgia and has scheduled a Type B pre-NDA meeting with FDA for the second quarter of 2024.

1 40% was the median preference share among physicians interviewed, assuming no market access barriers.
2 EVERSANA analysis of claims database, May 2024; commissioned by Tonix
3Note: the total number of patients is greater than 100% because of switching between medications and polypharmacy.
4 EVERSANA primary physician research, May 2024; commissioned by Tonix

About Tonmya* (also known as TNX-102 SL)

Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for the management of fibromyalgia. In December 2023, the company announced highly statistically significant and clinically meaningful topline results in RESILIENT, a second positive Phase 3 clinical trial of Tonmya for the management of fibromyalgia. In the study, Tonmya met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue and improving overall fibromyalgia symptoms and function. RELIEF, the first positive Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints.

*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.

About EVERSANA®

EVERSANA® is a leading independent provider of global services to the life sciences industry. The company’s integrated solutions are rooted in the patient experience and span all stages of the product life cycle to deliver long-term, sustainable value for patients, prescribers, channel partners and payers. The company serves more than 650 organizations, including innovative start-ups and established pharmaceutical companies, to advance life sciences solutions for a healthier world. To learn more about EVERSANA, visit eversana.com or connect through LinkedIn and X.

Tonix Pharmaceuticals Holding Corp.*

Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya, a product candidate for which two positive Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.

*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.

Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.

This press release and further information about Tonix can be found at www.tonixpharma.com.

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 by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of 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, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully commercialize any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.

Investor Contact

Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Peter Vozzo
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505

Media Contact

Katie Dodge
LaVoieHealthScience
kdodge@lavoiehealthscience.com
(978) 360-3151

Matt Braun
EVERSANA
Matt.braun@eversana.com

Source: Tonix Pharmaceuticals Holding Corp.

Released May 21, 2024

This $850M Biotech Deal Could Disrupt the Atopic Dermatitis Market

Posted on by slightbourne@noblecapitalmarkets.com

In a strategic move to strengthen its dermatology portfolio, pharmaceutical giant Johnson & Johnson has agreed to acquire Proteologix, a privately-held biotech developing bispecific antibody therapies for inflammatory skin diseases like atopic dermatitis. The $850 million all-cash deal gives J&J access to promising clinical and preclinical stage assets.

The crown jewel of the acquisition is Proteologix’s lead candidate PX128, a Phase 1-ready bispecific antibody targeting two key drivers of atopic dermatitis and asthma – interleukin-13 (IL-13) and thymic stromal lymphopoietin (TSLP). By simultaneously blocking these complementary inflammatory pathways, PX128 could provide a substantial efficacy boost over current monospecific antibody treatments.

Proteologix’s second asset, the preclinical bispecific PX130, goes after IL-13 and IL-22 for the treatment of moderate to severe atopic dermatitis. J&J cited the differentiated design of these dual-acting antibodies, highlighting their potential for infrequent, convenient dosing that could improve adherence.

The acquisition aligns with J&J’s strategic focus on building an immunology pipeline centered around bispecific antibodies for improved disease control across a range of inflammatory conditions.

Atopic dermatitis, a chronic inflammatory skin disease, impacts over 100 million adults worldwide, representing a massive market opportunity. However, up to 70% of patients fail to achieve remission on standard systemic treatments, underscoring a significant unmet need.

“We see an opportunity for best-in-disease efficacy for both PX128 and PX130,” said David Lee, global immunology therapeutic area head at J&J. The company believes the bispecifics could be game-changers for underserved patient subgroups by more comprehensively targeting the heterogenous drivers of atopic dermatitis.

The deal comes on the heels of positive Phase 3 data from Eli Lilly’s IL-13 antibody lebrikizumab in atopic dermatitis. After manufacturing setbacks, Lilly resubmitted its lebrikizumab filing in April and anticipates a decision later this year, setting up a potential commercial clash with J&J’s dual-acting antibodies down the road.

Proteologix, based in California, will be eligible for additional milestone payments on top of the $850 million upfront cash paid by J&J. The transaction, expected to close in mid-2024 pending regulatory approval, will fold in Proteologix’s other preclinical bispecific antibody programs focused on autoimmune diseases and cancer.

For J&J, the deal provides a promising path toward next-generation, differentiated therapies for the significant population of atopic dermatitis patients struggling with existing treatment options. Proteologix’s dual-acting bispecific antibodies represent potentially transformative medicines for a disease area that has proven stubbornly difficult to treat.

The acquisition reinforces J&J’s commitment to immunology and dermatology while bolstering its pipeline with innovative, clinically advanced assets that could drive future growth. As the atopic dermatitis market heats up, J&J has made a preemptive strike to secure a competitive edge through its newest biotech addition.

Want small cap opportunities delivered straight to your inbox?

Channelchek’s free newsletter will give you exclusive access to our expert research, news, and insights to help you make informed investment decisions.

Get Instant Access

Release – MAIA Biotechnology Abstract Accepted For Poster Presentation At American Society Of Clinical Oncology (ASCO) 2024 Annual Meeting

Posted on by aweinsoff@channelchek.com

Research News and Market Data on MAIA

May 16, 2024 8:37am EDT

  • Poster to present new efficacy data from Phase 2 THIO-101 trial in non-small cell lung cancer (NSCLC)

CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that an abstract about its Phase 2 THIO-101 clinical trial named “A phase 2, multicenter, open-label, dose-optimization study evaluating telomere-targeting agent THIO sequenced with cemiplimab in patients with advanced NSCLC: Updated results” was accepted for poster presentation at the American Society of Clinical Oncology (ASCO) 2024 Annual Meeting, to take place May 31-June 4, 2024, in Chicago, Illinois. The poster is scheduled for presentation on June 3, 2024, from 1:30pm to 4:30pm CST.

“We are proud to accept ASCO’s invitation to present at its 2024 Annual Meeting, the most significant gathering of oncology professionals worldwide,” said Vlad Vitoc, M.D., MAIA’s Chairman and Chief Executive Officer. “We look forward to revealing the newest efficacy results from THIO-101 and discussing our pioneering telomere targeting science underlying THIO, the first and only cancer treatment of its kind in clinical development.”

MAIA’s abstract will be available online at the ASCO Annual Meeting 2024 website during the week prior to the conference start date, and the poster will be published on maiabiotech.com on the day of the presentation, June 3, 2024.

The 2024 ASCO Annual Meeting will feature more than 200 sessions and 5,000 posters complementing the theme, “The Art and Science of Cancer Care: From Comfort to Cure.”

About ASCO

Founded in 1964, the American Society of Clinical Oncology is the world’s leading professional organization for physicians and oncology professionals caring for people with cancer. Its mission is to conquer cancer through research, education, and promotion of the highest quality, equitable patient care. ASCO’s vision is a world where cancer is prevented or cured, and every survivor is healthy. asco.org

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/20240516196926/en/

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

Source: MAIA Biotechnology, Inc.

Released May 16, 2024

Release – PDS Biotech Provides Business Update and Reports First Quarter 2024 Financial Results

Posted on by aweinsoff@channelchek.com

Research News and Market Data on PDSB

Provided Positive, Updated Data from Phase 2 VERSATILE-002 Clinical Trial with Versamune® HPV in Combination with KEYTRUDA® in Recurrent or Metastatic Head and Neck Cancer

Expanded Global Intellectual Property Surrounding Versamune® Platform

PRINCETON, N.J., May 15, 2024 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, today provided a business update and reported financial results for the first quarter of 2024. The press release will be available in the Investor Relations section of the Company’s website at www.pdsbiotech.com.

Recent Developments

  • Hosted a Key Opinion Leader event on May 8, 2024, during which prominent experts in head and neck squamous cell cancer (“HNSCC”) discussed positive, updated VERSATILE-002 data and the unmet need in HPV16-positive HNSCC. A replay of the event can be found here.
  • Announced updated results from the VERSATILE-002 Phase 2 trial evaluating first line treatment of patients with HPV16-positive recurrent or metastatic HNSCC using Versamune® HPV + KEYTRUDA® (pembrolizumab) (n=53).
    • Median overall survival is 30 months; Published results for immune checkpoint inhibitors are 7-18 months.
    • The cohort met its primary endpoint of best overall response (BOR).
    • BOR by investigator assessment is 34% (Combined Positive Score (CPS) ≥1; n=18/53); 48% (CPS≥20; n=10/21); Published results for ICIs are <20% (CPS>1) and <25% (CPS≥20).
      • CPS is used to assess PD-L1 expression
    • Progression free survival is 6.3 months (CPS≥1); 14.1 months (CPS≥20); Published results for immune checkpoint inhibitors 2-3 months.
    • VERSATILE-002 data to date indicate a durable response in first line recurrent or metastatic HNSCC patients with CPS≥1.
    • The combination of Versamune® HPV + pembrolizumab was well tolerated.
  • Announced an updated clinical strategy with a two-part registrational trial focused on the triple combination of Versamune® HPV + PDS01ADC + pembrolizumab as a first line treatment in HPV16-positive recurrent or metastatic HNSCC.
    • PDS01ADC is the Company’s novel, investigational tumor-targeting IL-12-fused antibody-drug conjugate (ADC), which has shown promise in a clinical trial of Versamune® HPV + PDS01ADC + an investigational ICI conducted by the National Cancer Institute.
    • Part one of the clinical trial will focus on dose optimization with a data readout based on safety and objective response rate.
    • The randomized second part of the trial will include an interim data readout with overall survival as its primary endpoint.
  • Further strengthened management with the addition of Stephan Toutain, M.S., MBA, as Chief Operating Officer. Mr. Toutain brings extensive operational and commercial experience to PDS Biotech.

Versamune® Platform Intellectual Property

  • Company received patents granted by the Israel Patent Office and IP Australia that will extend protections for the Company’s novel investigational T cell activating Versamune® platform through Dec. 2038 and Nov. 2036, respectively.
    • The Israel Patent Office granted patent No. 275154 titled, “Methods and compositions comprising cationic lipids for stimulating type I interferon genes,” extending protections for compositions using the Versamune® platform and comprising of cationic lipid for activating type I interferons. This patent covers all formulations and compositions that include Versamune to activate a T cell response.
    • IP Australia granted patent No. 2016354590 titled, “Lipids as synthetic vectors to enhance antigen processing and presentation ex-vivo in dendritic cell therapy.” This patent covers the use of Versamune® compositions that reduce the populations of immune suppressive cells in the tumor and its application for the development of dendritic cell-based approaches to immunotherapy.

First Quarter 2024 Financial Results
Reported net loss was approximately $10.6 million, or $0.30 per basic share and diluted share, for the three months ended March 31, 2024, compared to a net loss of $9.7 million, or $0.32 per basic share and diluted share, for the three months ended March 31, 2023. The increase was due to higher operating and net interest expenses.

Research and development expenses increased to approximately $6.7 million for the three months ended March 31, 2024, from $5.8 million for the three months ended March 31, 2023. The increase of $0.9 million was primarily attributable to an increase of $1.2 million in clinical studies and medical affairs offset by a decrease of $0.1 million in personnel costs, $0.1 million in professional fees and $0.1 million in manufacturing expenses.

General and administrative expenses decreased to approximately $3.4 million for the three months ended March 31, 2024, from approximately $3.6 million for the three months ended March 31, 2023. The decrease of $0.2 million was primarily attributable to an increase of $0.3 million in professional fees offset by a decrease of $0.5 million in personnel costs.

Total operating expenses increased to approximately $10.1 million for the three months ended March 31, 2024 from $9.4 million for the three months ended March 31, 2023.

Net interest expenses increased to approximately $0.5 million for the three months ended March 31, 2024 from $0.2 million for the three months ended March 31, 2023.

Cash and cash equivalents as of March 31, 2024, totaled approximately $66.6 million.

About PDS Biotechnology
PDS Biotechnology is a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines. The Company plans to initiate a pivotal clinical trial in 2024 to advance its lead program in advanced head and neck squamous cell cancers (HNSCC). PDS Biotech’s lead program is a proprietary dual-acting combination of IL-12 fused antibody drug conjugate (ADC) PDS01ADC and T-cell activator Versamune® HPV in regimen with a standard-of-care immune checkpoint inhibitor. We believe that proof-of-concept long-term data have shown positive survival results and tumor shrinkage with this combination and indicate favorable tolerability.

We believe that with a novel investigational “inside-outside” mechanism, the PDS01ADC and Versamune® HPV immunotherapy has shown compelling results with potential to successfully disrupt a tumor’s inside defenses, while also generating potent, targeted killer T-cells to attack the tumor from the outside. We believe that data from more than 350 patients, as well as ongoing clinical trials across multiple tumor types and standard treatment regimens, have validated the potential for both platforms and point to potential broad utility.

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. For more information, please visit www.pdsbiotech.com.

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 PDS01ADC, 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 PDS01ADC, 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; the Company’s ability to continue as a going concern; 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.

Keytruda® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.

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

View full release HERE.

Release – Unicycive Therapeutics Presents Bioequivalence Data On Oxylanthanum Carbonate (OLC) At The National Kidney Foundation Spring Clinical Meeting

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

Unicycive Therapeutics Presents Bioequivalence Data On Oxylanthanum Carbonate (OLC) At The National Kidney Foundation Spring Clinical Meeting

May 15, 2024 7:03am EDT Download as PDF

– OLC Demonstrates Bioequivalence to Lanthanum Carbonate –

– Additional Poster Highlights Key Features of OLC as Perceived by Renal Dieticians –

LOS ALTOS, Calif., May 15, 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 posters related to the Company’s lead product candidate, oxylanthanum carbonate (OLC), were presented at the National Kidney Foundation (NKF) Spring Clinical Meeting. OLC 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).

“The NKF Spring Clinical Meeting was an important meeting for Unicycive as we were able to present data from the OLC bioequivalence study and our second poster was featured as a top-rated submission,” said Shalabh Gupta, MD, Chief Executive Officer of Unicycive. “Phosphate binders are integral to the management of hyperphosphatemia in patients with CKD and their effectiveness is adversely affected by non-adherence and limitations of phosphate binding capacity relative to dietary intake. Our data confirms that OLC is bioequivalent to lanthanum carbonate (LC) by showing similar outcomes in both groups with respect to mean change in urinary phosphate excretion. Importantly, OLC demonstrated a well-tolerated safety profile with no serious adverse events. This data is important as it serves as one of the key components for our New Drug Application filing with the FDA under the 505(b)(2) regulatory pathway.”

Dr. Gupta, concluded, “We are grateful to Dr. Hill Gallant who delivered a poster presentation on a survey of renal dieticians who play a critical role in helping patients manage serum phosphate and are close witnesses to patients’ experiences and challenges with phosphate management. The findings of the survey concluded that strategies that reduce pill burden and increase ease of use for patients are needed, reinforcing our belief that, if approved, the characteristics of OLC including the reduction of pill volume may increase compliance and improve the quality of life for patients living with this condition.”

Presentation Details:

Title: Two-Way Crossover Study to Establish Pharmacodynamic Bioequivalence Between Oxylanthanum Carbonate and Lanthanum Carbonate
Lead Author: Vandana Mathur, MD
Results: The poster presentation described the results of the randomized, crossover bioequivalence study comparing OLC with lanthanum carbonate (LC). The study was a Phase 1, single-center, randomized 1:1, open-label, controlled, 2-way crossover study designed to demonstrate the pharmacodynamic (PD) equivalence between two phosphate binders: orally administered OLC as compared to LC. Both treatments were administered at doses of one 1000 mcg tablet three-times-a-day (TID) in healthy volunteers who received the same standardized meals to control for daily phosphate intake. OLC tablets are swallowed whole as opposed to the LC tablets that must be chewed completely. The study consisted of a screening period, two dosing periods separated by a 14-day washout period, and a follow-up period 7 days after last study drug dose. The primary PD variable was Least Squares (LS) mean change in urinary phosphate excretion from baseline (48hrs prior to dosing) to Evaluation Period (Days 1-3). The baseline characteristics were balanced between OLC/LC and LC/OLC sequences. LS mean change from baseline for OLC (-320.4mg/day) was similar to the LS mean change from BL for LC (-324.0mg/day). The 90% confidence interval for the LS mean change in urinary phosphate excretion from baseline (test-reference) was (-37.83, 45.12), which was entirely contained within the predefined ±20% acceptance range of (-64.80, 64,80). There were no serious adverse events (SAEs) and no treatment discontinuations. The incidence of treatment-emergent adverse events (TEAEs) and related AEs were also the same in both groups at 35% and 25%, respectively.

Title: Renal Dietitians Perceive Phosphate Binder and Low-Phosphate Diet Non-Compliance as Top Reasons for Above Target Serum Phosphorous Concentrations
Lead Author: Kathleen Hill Gallant, PhD, RD
Results: The poster presentation delivers results of a recent dietitian survey evaluating perceived reasons for noncompliance to phosphate binder (PB) therapy and identifies the most appealing potential aspects of OLC. The poster concluded that strategies that reduce pill burden and increase ease of use for patients may promote PB treatment compliance, which may improve patient outcomes. OLC, which is a smaller tablet that can be swallowed whole without chewing, may address compliance issues seen with current PBs. In fact, 47% of dieticians noted the high perceived potency of OLC and 34% noted its perceived lower number of pills required as the most appealing aspects of OLC.

Recent studies reported PB non-adherence rates of up to 78% in patients with end-stage kidney disease on dialysis. For the analysis, 100 renal dietitians were surveyed and there were several key findings. The most common reasons for phosphate levels above the target range were non-compliance to PB prescription (36%) or low phosphate diet (34%). The two leading reasons for PB discontinuation were too many pills and formulation issues. One-third of dietitians attributed non-compliance with patients forgetting to take their PBs with meals or snacks and 16% attributed it to high pill burden.

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 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, 2023, 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.

Released May 15, 2024

Release – Ocugen Announces Dosing Completion Of Subjects With Stargardt Disease In Cohort 2 Of Phase 1/2 GARDian Clinical Trial Of OCU410ST—A Modifier Gene Therapy

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

May 15, 2024

MALVERN, Pa., May 15, 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 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease as a one-time treatment for life.

“The completion of dosing for Cohort 2 participants signifies an important clinical milestone for our pioneering modifier gene therapy,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the ongoing positive safety and tolerability profile demonstrated by OCU410ST, enabling us to consider higher doses in patients as we progress with the dose-escalation study. We look forward to sharing preliminary safety and efficacy data from Phase 1 of the clinical trial.”

Six patients with Stargardt disease have been dosed in the Phase 1/2 clinical trial to date. An additional three patients will be dosed with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.

“There remains a great unmet medical need for patients with Stargardt disease, which is the most common inherited retinal disease affecting the center of the vision and does not have any FDA-approved treatment options. OCU410ST is a novel modifier gene therapy that provides hope to these patients,” said Benjamin Bakall, MD, PhD, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at University of Arizona, College of Medicine – Phoenix. “I am excited that we completed dosing of the last patient in Cohort 2, who received medium dose of this novel therapeutic leveraging a gene-agnostic approach, at Associated Retina Consultants (ARC) in Phoenix, AZ with the surgical team led by Dr. Mark Kwong, Medical Director of ARC.”

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 Cohort 3 (high dose), which is the final dose in the Phase 1 dose-escalation study.

The GARDian clinical trial will assess the safety and efficacy 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 (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 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 group.

Ocugen is committed to finding solutions for people with inherited retinal disease for whom no effective treatment options exist. While an orphan disease, Stargardt affects approximately 100,000 people in the United States and Europe combined.

The Company expects to provide a clinical trial update for OCU410ST in the third quarter of 2024.

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 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.

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

Ocugen (OCGN) – 1Q24 Reported With Upcoming Clinical Milestones As Expected

Posted on by cpereira@noblefcm.com


Wednesday, May 15, 2024

Ocugen, Inc. is a biotechnology company focused on developing and commercializing novel gene therapies, biologicals, and vaccines. The lead product in its gene therapy program, OCU400, is in Phase 1/2 clinical trials for retinitis pigmentosa.

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.

1Q24 Reported With Clinical Trial Updates. Ocugen reported 1Q24 loss of $11.9 million or $(0.05) per share, and gave updates on progress of its clinical trial during the quarter. Its three main ophthalmic disease programs continued their progress and completed stages of development. Data announcements and milestones are expected for the rest of FY2024. Cash on March 31, 2024 was $26.4 million.

OCU400 Phase 3 Trial Expected Shortly. The Phase 3 trial for OCU400, known as the liMeliGhT incorporating “Master Gene Therapy” into its name, has received FDA clearance and is expected to begin treating retinitis pigmentosa (RP) patients in 2Q24. The trial has a planned enrollment of 150 patients, divided into an arm with 75 RHO mutation patients and 75 patients with any RP associated mutation (gene agnostic). The primary endpoint is luminance dependent navigation assessment (LDNA. As results from the Phase 1/2 study in Leber congenital amaurosis (LCA) become available, the trial could be expanded enroll LCA patients later in 2024.


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. 

GeoVax Labs (GOVX) – 1Q24 Reported With Clinical Milestones Later In FY2024

Posted on by cpereira@noblefcm.com


Wednesday, May 15, 2024

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel therapies and vaccines for solid tumor cancers and many of the world’s most threatening infectious diseases. The company’s lead program in oncology is a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, presently in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax’s lead infectious disease candidate is GEO-CM04S1, a next-generation COVID-19 vaccine targeting high-risk immunocompromised patient populations. Currently in three Phase 2 clinical trials, GEO-CM04S1 is being evaluated as a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, and as a booster vaccine in patients with chronic lymphocytic leukemia (CLL). In addition, GEO-CM04S1 is in a Phase 2 clinical trial evaluating the vaccine as a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. GeoVax has a leadership team who have driven significant value creation across multiple life science companies over the past several decades.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Clinical Trial Treatment Continues With Milestones Ahead. GeoVax reported a 1Q24 loss of $5.6 million or $(2.47) per share. The company gave updates for its three trials testing GM04S1 and its Phase 1/2 trial testing Gedeptin, including the expected announcement about future clinical development for Gedeptin. Cash on March 31, 2024 was $0.8 million.

Gedeptin Is Finishing Its Phase 1/2 Study. The Phase 1/2 trial testing Gedeptin in advanced head and neck cancer has completed enrollment, with the next steps of development in planning stages. The current plan is to continue development in head and neck cancer, with an announcement coming regarding future trials. This could include testing in combination with an immune checkpoint inhibitor (ICI). Trial data and development announcements are expected in 3Q24.


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. 

Release – Ocugen Provides Business Update With First Quarter 2024 Financial Results

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

May 14, 2024

PDF Version

CONFERENCE CALL AND WEBCAST TODAY AT 8:30 A.M. ET

  • All three first-in-class modifier gene therapy product candidates currently in the clinic with OCU400 Phase 3 in progress
  • OCU400 on track to meet 2026 Biologics License Application (BLA) and Market Authorization Application (MAA) approval targets

MALVERN, Pa., May 14, 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 reported first quarter 2024 financial results along with a general business update.

“We’ve experienced several important clinical and regulatory milestones since the beginning of 2024 that we believe are leading the way to a new treatment paradigm for patients with blindness diseases,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “It’s very encouraging to have FDA clearance and EMA support for the Phase 3 clinical trial of our lead modifier gene therapy candidate that offers a potential one-time treatment for life.”

With FDA clearance to begin the Phase 3 liMeliGhT clinical trial, OCU400 becomes the first gene therapy to progress to late-stage trials with a broad retinitis pigmentosa (RP) indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. The gene-agnostic mechanism of action for OCU400 provides hope for a much larger RP patient population. In the U.S. and Europe combined, RP affects nearly 300,000 people.

Ocugen expects to begin dosing patients in the Phase 3 liMeliGhT clinical trial in the second quarter of 2024. The Phase 3 trial will have a sample size of 150 participants—one arm of 75 participants with the RHO gene mutation and the other arm with 75 participants that are gene-agnostic. Luminance Dependent Navigation Assessment (LDNA) is the primary endpoint for the study and focuses on the proportion of responders, in treated and untreated groups, achieving an improvement of at least 2 Lux levels from baseline in the study eyes.

Leveraging a dual-track strategy, the Company plans to expand the Phase 3 OCU400 clinical trial in the second half of 2024 to include patients with Leber congenital amaurosis (LCA), contingent on favorable results from the Phase 1/2 study.

Modifier gene therapy has the potential to treat inherited retinal diseases as well as multifactorial blindness diseases affecting millions of patients. Leveraging the nuclear receptor gene RAR-related orphan receptor A (RORA), OCU410 is designed to regulate all four pathways involved with dry age-related macular degeneration (dAMD)—including lipid metabolism, inflammation, oxidative stress, and membrane attack complex (complement). Ocugen is developing OCU410 as a one-time gene therapy for the treatment of geographic atrophy (GA), an advanced stage of dAMD, affecting 2-3 million people in the U.S. and Europe combined. OCU410ST is being developed as a one-time gene therapy for the treatment of Stargardt disease, affecting approximately 100,000 people in the U.S. and Europe combined.

In April, dosing was completed in the second cohort (medium dose) of the Phase 1/2 ArMaDa clinical trial for OCU410. Dosing in the first cohort (low dose) of the Phase 1/2 GARDian trial for OCU410ST was completed earlier in the first quarter and in April 2024, the Data Safety and Monitoring Board approved the continuation to cohort 2 (medium dose).

“Our efforts in the first quarter of the year evidence the importance of our gene therapy programs and the need to operate the business to ensure their success,” said Dr. Musunuri. “We are opportunistic about Ocugen’s cell therapy and vaccine platforms, knowing that these technologies have great therapeutic and financial potential and are pursuing partnerships to support our entire pipeline.”

Ophthalmic Gene Therapies —First-in-class

  • OCU400—Received FDA clearance of IND amendment to initiate OCU400 Phase 3 liMeliGhT clinical trial in RP. EMA provided acceptability of the U.S.-based trial for submission of Marketing Authorization Application (MAA). Currently, the multi-center Phase 3 clinical trial is in progress.
  • OCU410 – Currently in Phase 1/2 stage of clinical development with active patient enrollment. Dosing is complete in the second cohort (medium dose) in the dose-escalation phase of the study. Once the third cohort (high dose) is complete, the Company will move into the Phase 2 clinical trial—the expansion phase—in the third quarter of 2024.
  • OCU410ST – Currently in Phase 1/2 stage of clinical development with active patient enrollment. Dosing is complete for cohort 1 (low dose). Initiated enrollment in cohort 2 (medium dose) in the dose-escalation phase of the trial.

Regenerative Cell Therapies—First-in-class

  • NeoCart® – Completed renovating an existing facility into a current Good Manufacturing Practice (“GMP”) facility in accordance with the FDA’s regulations. Intend to initiate the Phase 3 trial, contingent on adequate availability of funding.

Vaccines Portfolio—First-in-class

  • Mucosal Vaccine Platform – NIAID is collaborating with Ocugen on clinical development of OCU500. Planning to submit IND by mid-2024 to initiate Phase 1 clinical trial.

Biologics

  • OCU200—Continue to work with FDA to address comments to lift the clinical hold.

First Quarter 2024 Financial Results

  • The Company’s cash and cash equivalents totaled $26.4 million as of March 31, 2024, compared to $39.5 million as of December 31, 2023. The Company had 257.3 million shares of common stock outstanding as of March 31, 2024.
  • Total operating expenses for the three months ended March 31, 2024 were $13.2 million and included research and development expenses of $6.8 million and general and administrative expenses of $6.4 million. This compares to total operating expenses for the three months ended March 31, 2023 of $18.5 million that included research and development expenses of $10.2 million and general and administrative expenses of $8.3 million.
  • Ocugen reported a $0.05 net loss per common share for the three months ended March 31, 2024 compared to a $0.08 net loss per common share for the three months ended March 31, 2023.

Conference Call and Webcast Details

Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s senior management team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.

Attendees are invited to participate on the call or webcast using the following details:

Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers
Conference ID: 8699924
Webcast: Available on the events section of the Ocugen investor site

A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.

About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; Ocugen’s financial condition, 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 annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

View full release here.