Category: Health

Eliem Bets Big on Autoimmune Pipeline with $210M Tenet Buyout

Posted on by slightbourne@noblecapitalmarkets.com

The biotech deal scene is heating up, with Eliem Therapeutics becoming the latest to double down on an emerging pipeline through M&A. The clinical-stage company announced it is acquiring private biotech Tenet Medicines for $120 million in stock, while simultaneously raising $120 million in a private placement to fund Tenet’s lead autoimmune disease program.

The transaction allows Eliem to pivot from its previous focus on neurological disorders to prioritize TNT119 – an anti-CD19 antibody that Tenet was developing across multiple autoimmune indications like lupus, immune thrombocytopenia, and membranous nephropathy. With $210 million in projected cash reserves after closing, the combined company plans to rapidly advance TNT119 into Phase 2 studies for systemic lupus erythematosus and immune thrombocytopenia in the second half of 2024.

“TNT119 represents a promising clinical asset across autoimmune diseases where there is a clear need for improved treatments,” said Andrew Levin, Eliem’s Executive Chairman. “This deal allows us to accelerate development of a potential best-in-class therapy while creating value for our shareholders.”

The acquisition continues the torrid pace of deal-making in biotech as companies look to revamp pipelines and build out emerging focus areas through M&A. Just in the first few months of 2024, over $30 billion in biotech acquisitions have been announced according to Bloomberg data.

Major deals included Vertex’s $4.9 billion buyout of Alpine Immune Sciences to gain a promising IgA nephropathy drug, Pfizer’s $43 billion acquisition of Seagen to bolster its oncology portfolio, and Amgen’s $27.8 billion deal for Horizon Therapeutics’ rare disease pipeline. Now Eliem is the latest to join the fray, making a significant bet on the autoimmune space.

“We believe TNT119 has best-in-class potential and could transform treatment for many autoimmune patients,” said Stephen Thomas, who will become interim CEO of the combined company after previously leading Tenet.

In addition to systemic lupus erythematosus and immune thrombocytopenia, TNT119 has also shown preliminary efficacy in treating membranous nephropathy – an autoimmune kidney disorder. By depleting B cells that produce autoantibodies driving the diseases, the therapy could provide a novel approach across this set of serious inflammatory conditions.

To fund TNT119’s ambitious development program, Eliem secured backing from a syndicate of major healthcare investors including RA Capital, Deep Track Capital, Boxer Capital and Janus Henderson in the $120 million private placement.

The wave of biotech deals has been driven by larger players looking to rebuild pipelines, while smaller companies seek resources to push programs forward amid a challenging economic environment and tight funding markets. With its Tenet acquisition, Eliem is aiming to thread that needle – gaining a promising clinical asset while raising enough capital to rapidly usher it through key studies.

For Eliem shareholders, the risky pivot to autoimmune therapies represents a major strategic shift. But if TNT119 can live up to its blockbuster aspirations, it could allow the company to go from an underdog in neurological disorders to a standout in the hot autoimmune space. That big “if” appears to be a gamble Eliem and its investors are willing to take amid biotech’s current deal-making renaissance.

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Release – Ocugen To Participate In Fireside Chat At The Emerging Growth Virtual Healthcare Equity Conference, Presented By Noble Capital Markets

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

April 12, 2024

MALVERN, Pa., April 12, 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 Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder will participate in a fireside chat during the Emerging Growth Virtual Healthcare Equity Conference, presented by Noble Capital Markets, taking place from April 17 – 18, 2024.

Details of the fireside chat are as follows:

Date: Wednesday, April 17, 2024

Time: 10 a.m. ET

Location: Presentation Track 1

Webcast: Available in the Events section of Ocugen website

During the fireside chat and one-on-one sessions, Dr. Musunuri will showcase the Company’s recent business and clinical development progress including FDA clearance to initiate a Phase 3 clinical trial for OCU400—a modifier gene therapy for patients battling retinitis pigmentosa (RP). OCU400 is the first gene therapy to enter a late-stage trial with a broad RP indication.

A live webcast of the fireside chat will be available in the Events section of the Company’s website and as part of a complete catalog of presentations available on Channelchek—the investor portal created by Noble. The webcast will be archived on the Company’s website for 90 days following the event. 

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

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Cadrenal Therapeutics (CVKD) – Tecarfarin Receives Orphan Drug Designation For Second Indication

Posted on by cpereira@noblefcm.com


Friday, April 12, 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.

Orphan Designation Received For Circulatory Assist Devices. Cadrenal Therapeutics has received Orphan Drug designation for tecarfarin as an anticoagulant in patients with circulatory-assist devices, including ventricular assist devices (VADs) and total artificial hearts. This is the second Orphan designation for tecarfarin. The first indication, atrial fibrillation associated with end stage renal disease (AFib with ESRD), is expected to begin a Phase 3 trial in 2Q24.

Circulatory Assist Device Patients Do Not Have Effective Anticoagulants. Mechanical devices to assist blood circulation can activate the clotting cascade, putting patients who receive them at risk of clots or thromboembolisms. Commonly used anticoagulants in the direct oral anticoagulant (DOAC) category have not shown efficacy in these patients and have been contraindicated. This leaves the patients at high risk for thromboembolic events and hospitalizations.


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*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Vertex Banks on Autoimmune Therapy in $4.9 Billion Alpine Acquisition

Posted on by slightbourne@noblecapitalmarkets.com

Boston-based biotech giant Vertex Pharmaceuticals announced today that it has agreed to acquire Alpine Immune Sciences for $4.9 billion in cash, placing a major bet on the smaller company’s promising drug candidate for treating serious autoimmune diseases.

The crown jewel of the acquisition is Alpine’s lead molecule povetacicept, a dual antagonist of the BAFF and APRIL proteins that have been implicated in driving several autoimmune and inflammatory conditions. Through Phase 2 trials, povetacicept has demonstrated potentially best-in-class efficacy for treating IgA nephropathy (IgAN), a serious progressive kidney disease caused by autoimmune complexes.

IgAN is the most common cause of primary glomerulonephritis (inflammation of the kidney’s filtering units) worldwide, affecting approximately 130,000 people in the U.S. alone. The disease frequently leads to end-stage renal failure, yet there are currently no approved treatments that target the underlying causes of IgAN. Povetacicept is slated to enter pivotal Phase 3 clinical trials in the second half of 2024.

“Alpine is a compelling strategic fit that furthers our ambition of creating transformative medicines for serious diseases with high unmet need,” said Reshma Kewalramani, Vertex’s CEO and President. “We look forward to bringing povetacicept, a potential best-in-class treatment for IgAN, to patients faster.”

But Vertex is betting big that povetacicept’s impact could extend far beyond just IgAN. Due to its dual mechanism targeting BAFF and APRIL, the drug candidate holds promise as a potential “pipeline-in-a-product” for treating other autoimmune diseases affecting the kidneys like membranous nephropathy and lupus nephritis. Clinical trials are also evaluating povetacicept’s utility for autoimmune cytopenias that destroy blood cells.

The $4.9 billion acquisition allows Vertex, a leader in cystic fibrosis treatments, to expand into autoimmune and inflammatory diseases – one of the hottest areas of drug development. It also provides Vertex with Alpine’s protein engineering expertise that could unlock new therapeutic modalities.

“Povetacicept has demonstrated potential best-in-class attributes and has broad development potential across autoimmune conditions with significant unmet need,” said Mitchell Gold, Alpine’s CEO. “We’re excited for the opportunity to make a meaningful difference as part of Vertex.”

The deal is structured as an all-cash tender offer, with Vertex paying $65 per share for Alpine’s outstanding stock – a substantial 92% premium over Alpine’s closing price on April 9th. Vertex expects to finance the $4.6 billion net transaction cost through a combination of existing cash on hand and new debt financing.

The acquisition, which was unanimously approved by both companies’ boards, is expected to close in the second quarter of 2024 pending regulatory approval and other customary closing conditions. It marks Vertex’s second acquisition in the autoimmune disease space in recent years, having purchased protein therapeutics firm Semma Therapeutics in 2019 for $950 million.

With povetacicept’s promising data and Vertex’s resources behind it, the combined company will be well-positioned to rapidly advance a potentially transformative new class of autoimmune therapies. But at a lofty price tag nearing $5 billion, the deal places a major bet that the Alpine drug can live up to its blockbuster aspirations.

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Tonix Pharmaceuticals (TNXP) – Increasing The Focus on Fibromyalgia

Posted on by cpereira@noblefcm.com


Thursday, April 11, 2024

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics and diagnostics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of immunology, rare disease, infectious disease, and central nervous system (CNS) product candidates. Tonix’s immunology portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-15001 which is a humanized monoclonal antibody targeting CD40-ligand being developed for the prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second half of 2022. Tonix’s rare disease portfolio includes TNX-29002 for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan-Drug Designation by the FDA. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and monkeypox called TNX-8013, next-generation vaccines to prevent COVID-19, and an antiviral to treat COVID-19. Tonix’s lead vaccine candidates for COVID-19 are TNX-1840 and TNX-18504, which are live virus vaccines based on Tonix’s recombinant pox vaccine (RPV) platform. TNX-35005 (sangivamycin, i.v. solution) is a small molecule antiviral drug to treat acute COVID-19 and is in the pre-IND stage of development. TNX-102 SL6, (cyclobenzaprine HCl sublingual tablets), is a small molecule drug being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Tonix expects to initiate a Phase 2 study in Long COVID in the second quarter of 2022. The Company’s CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead CNS candidate, TNX-102 SL, is in mid-Phase 3 development for the management of fibromyalgia with a new Phase 3 study launched in the second quarter of 2022. Finally, TNX-13007 is a biologic designed to treat cocaine intoxication that is expected to start a Phase 2 trial in the second quarter of 2022. TNX-1300 has been granted Breakthrough Therapy Designation by the FDA.

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.

We Expect Tonmya To Drive TNXP Forward. Since announcing successful Phase 3 RESILIENT trial data in late December, Tonix has been shifting its development and financial strategy to focus on filing the NDA for Tonmya approval during 2H24. The company has raised capital and plans lower R&D expenses to preserve cash. We have revised our earnings estimates and price target while maintaining our Outperform rating.

Tonix Will Focus On Products For CNS Disorders and Reduce Expenses. Tonix has developed broad pipeline of products in infectious disease, biodefense, and immunology that grew from its original work in neurology and immunology. In the coming year, it will focus on the NDA filing and premarketing activities for Tonmya. We have reduced our clinical expenses estimates, since the large trials have been completed and no late-stage trials are scheduled.


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This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Century Therapeutics Makes Bold Move in Autoimmune Disease and Cell Therapy

Posted on by slightbourne@noblecapitalmarkets.com

Century Therapeutics, a pioneering biotech company developing induced pluripotent stem cell (iPSC)-derived cell therapies, announced a transformative set of initiatives that could reshape the landscape of cell therapy in cancer and autoimmune diseases.

The centerpiece is an ambitious expansion of Century’s lead program CNTY-101, a novel CD19-targeting immune cell therapy, into multiple autoimmune indications beyond the previously planned systemic lupus erythematosus (SLE) trial. This strategic pivot is backed by a $60 million private financing round and bolstered by the acquisition of Clade Therapeutics.

CNTY-101 is an allogeneic, iPSC-derived natural killer (iNK) cell therapy that has shown promising potential in eradicating cancerous B-cells in early clinical trials. Century believes its unique design, including gene edits to enable repeat dosing without lymphodepletion, could make it an ideal therapy for autoimmune diseases driven by B-cell dysregulation.

“We’ve seen compelling translational data pointing to CNTY-101’s potential in diseases like lupus,” said Century CEO Brent Pfeiffenberger. “This financing allows us to aggressively pursue that opportunity across multiple autoimmune indications with high unmet need.”

While the SLE trial remains on track for 2024, Century plans additional regulatory filings for CNTY-101 in autoimmune diseases in the second half of this year, supported by the $60 million raise from investors like Bain Capital Life Sciences and Adage Capital.

But the company didn’t stop there. Century also acquired Clade Therapeutics and its innovative iPSC-derived alpha beta T-cell platform for $35 million upfront, with potential future milestones. The deal adds three promising preclinical cancer and autoimmune programs to Century’s pipeline.

More importantly, it provides Century with next-generation capabilities to manufacture highly-functional, engineered T-cell therapies from iPSCs, something the field has long sought after.

“Clade’s groundbreaking platform replicates the natural T-cell development process, overcoming key limitations of current therapies,” said Century R&D President Hy Levitsky, M.D. “Combined with our iPSC-derived NK and gamma delta T-cells, this gives us unparalleled ability to create potential cures across a wide range of diseases.”

The move establishes Century as a preeminent player in allogeneic, off-the-shelf iPSC cell therapy, with an arsenal of NK cells, alpha beta T-cells, and gamma delta T-cells for oncology and autoimmune diseases. It diversifies the pipeline with complementary assets while providing a renewable cell source to manufacture consistent, high-quality therapies.

While still in early stages, some analysts view this as an aggressive and smart play by Century to stay ahead of the competition in this rapidly evolving space. By expanding into autoimmune diseases, acquiring transformative technology, and putting significant capital behind it all, Century is cementing its position as an iPSC cell therapy leader looking to deliver on the modality’s long-awaited promise.

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Biotech Buzz: Adial and Skye Bioscience Deliver Promising Updates Amid Sector Momentum

Posted on by slightbourne@noblecapitalmarkets.com

The biotech and healthcare sectors have seen a flurry of activity in recent weeks, with companies making strides through drug developments, clinical trials, and corporate milestones. Two firms generating buzz today are Adial Pharmaceuticals (ADIL) and Skye Bioscience (SKYE), both reporting encouraging news that has fueled investor interest.

Adial Pharmaceuticals, a clinical-stage biopharmaceutical company focused on developing addiction therapies, announced the publication of a peer-reviewed article highlighting the promising safety data and high patient compliance observed with its lead investigational drug AD04 in a Phase 3 clinical trial for alcohol use disorder (AUD).

The study, published in the European Journal of Internal Medicine, comprehensively analyzed the liver safety profile of low-dose AD04 compared to a placebo in patients with AUD and a specific genetic profile. Notably, AD04 did not significantly impact biochemical markers of liver injury like ALT, AST, and bilirubin levels, underscoring its potential to address AUD while mitigating liver damage risks.

Moreover, AD04 demonstrated an impressive safety and tolerability profile, with low adverse event occurrence, high medication adherence, and minimal dropout rates – a rarity for AUD treatments. Adial’s CEO, Cary Claiborne, expressed enthusiasm about providing a precision treatment tailored to individuals with AUD, potentially offering a novel approach to managing alcohol consumption and liver harm.

Separately, Skye Bioscience (SKYE), a clinical-stage biotech focused on the endocannabinoid system, achieved a significant milestone by uplisting its common stock to the Nasdaq Global Market. Trading under the ticker “SKYE” is expected to commence on April 11th.

The Nasdaq uplisting is a testament to Skye’s recent accomplishments, including advancing its Phase 2 clinical programs, strengthening its financial position, and broadening its shareholder base. As CFO Kaitlyn Arsenault noted, the move aims to enhance visibility, liquidity, and ultimately drive long-term shareholder value.

Skye’s pipeline includes SBI-100 Ophthalmic Emulsion, a CB1 agonist being studied in a Phase 2 trial for glaucoma and ocular hypertension, with top-line data expected this quarter. Additionally, the company plans to launch a Phase 2 clinical trial in Q3 2024 for nimacimab, a peripheral CB1 inhibitor, targeting obesity through monotherapy and combination arms with a GLP-1R agonist.

These developments from Adial and Skye underscore the vibrant activity within the biotech and healthcare sectors, where companies are continuously striving to advance innovative therapies and achieve corporate milestones.

Contributing to the sector’s momentum is the upcoming Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference, taking place on April 17-18. This premier event will bring together industry leaders, investors, and emerging companies, providing a platform to showcase groundbreaking research, discuss market trends, and explore potential partnerships and collaborations.

With the biotech and healthcare industries consistently evolving, such conferences play a crucial role in fostering collaboration, facilitating knowledge-sharing, and driving progress towards improving patient outcomes and advancing healthcare solutions.

As companies like Adial and Skye continue to make strides, the broader biotech and healthcare sectors remain vibrant and poised for growth, fueled by scientific advancements, regulatory approvals, and investor confidence. The upcoming Noble Capital Markets Virtual Healthcare Conference promises to further catalyze innovation and propel the industry forward.

Click here to register for the Emerging Growth Virtual Healthcare Equity Conference.

Release – Ocugen, Inc. Announces Positive Scientific Advice From The European Medicines Agency Related To The Approval Pathway For OCU400—Modifier Gene Therapy For Broad Retinitis Pigmentosa Indication

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

April 10, 2024

MALVERN, Pa., April 10, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) reviewed the study design, endpoints and planned statistical analysis of the pivotal OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP) and provided acceptability of the U.S.-based trial for submission of a Marketing Authorization Application (MAA).

EMA provided this opinion based on safety and tolerability of OCU400 demonstrated in the Phase 1/2 study. The Phase 3 liMeliGhT study will have a sample size of 150 participants primarily in the U.S.—one arm of 75 participants with RHO gene mutations and the other arm with 75 participants that are gene agnostic (representing multiple gene mutations associated with RP). In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

The positive scientific advice from EMA is in alignment with U.S. FDA clearance of the IND amendment to initiate the Phase 3 liMeliGhT clinical trial of OCU400. OCU400 is the first gene therapy to enter Phase 3 with a broad RP indication. Previously, OCU400 received broad Orphan Drug Designation for RP and Leber congenital amaurosis in the EU.

“We are very grateful to EMA for their collaborative discussions and support in providing a gene-agnostic therapeutic option to RP patients with severe unmet medical need,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “This positive opinion is a critical step in providing our game-changing modifier gene therapies to patients globally.”

The EMA opinion is an extremely favorable outcome, as it will potentially reduce the time and cost to gain marketing authorization in the EU. With this milestone, OCU400 remains on track for 2026 BLA and MAA approval targets.

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

About RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients.

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

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Release – Unicycive Therapeutics to Present At The Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

April 10, 2024 7:03am EDT

LOS ALTOS, Calif., April 10, 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 Shalabh Gupta, M.D., Chief Executive Officer will present a company update at the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference on Thursday April 18, 2024 at 11:00 a.m. ET.

A link to the live and archived webcast may be accessed on the Unicycive website under the Investors section: Events and Presentations. An archive of the webcast will be available for three months.

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.

Investor Contact:

ir@unicycive.com
(650) 543-5470

SOURCE: Unicycive Therapeutics, Inc.

Released April 10, 2024

Release – Cadrenal Therapeutics Receives FDAOrphan Drug Designation For Tecarfarin For Prevention Of Thromboembolism And Thrombosis In Patients With LVADs, RVADs, Biventricular Assist Devices, And Total Artificial Hearts

Posted on by aweinsoff@channelchek.com

Research News and Market Data on CVKD

FDA designation provides potentially seven years of market exclusivity after approval and expanded partnering opportunities for tecarfarin

PONTE VEDRA, Fla., April 9, 2024 — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage novel oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions, announced today that the United States Food and Drug Administration (FDA) has granted tecarfarin Orphan Drug Designation (ODD) for the prevention of thromboembolism and thrombosis in patients with an implanted mechanical circulatory support device (left ventricular assist device (LVAD), right ventricular assist device (RVAD), collectively known as ventricular assist devices (VADs), biventricular assist device, and total artificial heart).



“This second orphan drug designation highlights the expanded need for tecarfarin where existing anticoagulation therapies are inadequate,” said Quang Pham, Founder, Chairman and Chief Executive Officer of Cadrenal Therapeutics. “We are dedicated to advancing tecarfarin through clinical development options as swiftly as possible.”

The FDA’s ODD program provides incentives to sponsor organizations for the development of innovative treatments for rare diseases that affect fewer than 200,000 people in the U.S. Since its adoption in 1983, the Orphan Drug Act has helped countless individuals living with these conditions gain access to life-enhancing and life-saving therapies. ODD also provides certain benefits to drug developers, including assistance in the drug development process, tax credits for certain clinical research, and a waiver of the New Drug Application user fee. The designation is made to promote safe and efficacious products for the treatment of rare conditions.

All patients with VADs require chronic anticoagulation to prevent the formation of thrombus (clot) which can cause the device to fail or can result in a clot breaking off (embolizing), resulting in a stroke or other vascular catastrophe.

The current market-leading direct oral anticoagulants (DOACs), such as Eliquis, are not indicated for patients with VADs due to a lack of evidence of benefit. Moreover, a recent study revealed that the level of anticoagulation achieved with warfarin, the only currently available Vitamin K Antagonist (VKA), is maintained in the target range only 56% of the time which has been shown to increase the risk of clotting and bleeding complications.

ABOUT CADRENAL THERAPEUTICS, INC.

Cadrenal Therapeutics is developing tecarfarin for unmet needs in anticoagulation therapy. Tecarfarin is a late-stage novel oral and reversible anticoagulant (blood thinner) to prevent heart attacks, strokes, and deaths due to blood clots in patients with rare cardiovascular conditions. Tecarfarin has orphan drug and fast-track designations from the FDA for the prevention of systemic thromboembolism (blood clots) of cardiac origin in patients with end-stage kidney disease (ESKD) and atrial fibrillation (AFib) and just received orphan drug designation for the prevention of thrombosis and thromboembolism in patients with ventricular assist devices (VADs). Cadrenal is also pursuing additional regulatory strategies for unmet needs in anticoagulation therapy for patients with thrombotic antiphospholipid syndrome (APS). Tecarfarin is specifically designed to leverage a different metabolism pathway than the oldest and most commonly prescribed Vitamin K Antagonist (warfarin). Tecarfarin has been evaluated in eleven (11) human clinical trials and more than 1,000 individuals. In Phase 1, Phase 2, and Phase 2/3 clinical trials, tecarfarin has generally been well-tolerated in both healthy adult subjects and patients with chronic kidney disease. For more information, please visit www.cadrenal.com.

Safe Harbor Statement

Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding tecarfarin addressing unmet needs in anticoagulation therapy and the ability to derive the anticipated and potential benefits from the recent Orphan Drug Designation for tecarfarin, including seven years of market exclusivity, and the expanded development and commercial partnering for tecarfarin as a result of the Orphan Drug Designation. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the Company’s ability to derive the anticipated benefits from the recent Orphan Drug Designation for tecarfarin and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s filings with the Securities and Exchange Commission, including periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.

For more information, please contact:

Cadrenal Therapeutics:
Matthew Szot, CFO
858-337-0766
press@cadrenal.com

Investors:
Lytham Partners, LLC
Robert Blum, Managing Partner
602-889-9700
CVKD@lythampartners.com

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SOURCE Cadrenal Therapeutics, Inc.

Ocugen (OCGN) – Phase 3 IND Clearance Is Good News We’ve Been Waiting For

Posted on by cpereira@noblefcm.com


Tuesday, April 09, 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.

Phase 3 Trial Is Expected to Start Shortly. Ocugen announced that its amended IND for the OCU400 Phase 3 clinical trial has been cleared by the FDA. This allows the clinical trial to start enrolling patients, consistent with previous guidance for an April 2024 start. The company now has 3 clinical trials in progress in its ophthalmic disease programs.

Phase 3 Could Start Immediately. The trial is designed to compare patients treated with OCU400 to untreated controls. There are two arms, one enrolling patients with the RHO mutation and a “gene agnostic” arm enrolling patients who may have any of the mutations associated with RP. Each arm will randomize patients at 2:1, with 50 in each treatment group and 25 in each control group for a total enrollment of 150 patients. Patients will be treated with a single injection of OCU400.


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Release – Ocugen, Inc. Announces U.S. FDA Clearance of IND Amendment to Initiate OCU400 Phase 3 Clinical Trial — First Gene Therapy to Enter Phase 3 with a Broad Retinitis Pigmentosa Indication

Posted on by cpatches

Research News and Market Data on OCGN

MALVERN, Pa., April 08, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) amendment to initiate a Phase 3 clinical trial of OCU400, a modifier gene therapy product candidate being developed for retinitis pigmentosa (RP).

“The initiation of the OCU400 Phase 3 clinical trial is a significant milestone for patients with RP and a pivotal event for Ocugen as a company,” said Dr. Shankar Musunuri, Chairman, CEO and Co-Founder of Ocugen. “OCU400 is the first gene therapy program to enter Phase 3 with a broad RP indication. Until now, there has been only one marketed product to treat one of the 100 gene mutations associated with RP. Now there is real hope for all RP patients who haven’t had a treatment option.”

The Phase 3 study 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. In each arm, participants will be randomized 2:1 to the treatment group (2.5 x 1010 vg/eye of OCU400) and untreated control group, respectively.

In the Phase 1/2 OCU400 clinical trial, a Multi-Luminance Mobility Testing (MLMT) scale was the primary functional endpoint. For the Phase 3 OCU400 clinical trial, an updated mobility course will be used—Luminance Dependent Navigation Assessment (LDNA)— that includes a wider range of light intensity (0.04-500 Lux) and Lux Levels (0-9) with a uniform correlation between Lux level and Lux intensity.

“A sensitive mobility course, LDNA, was developed by Ocugen in collaboration with FDA for the Phase 3 clinical trial to allow enrollment of patients with early to advanced stages of disease,” said Dr. Arun Upadhyay, Chief Scientific Officer at Ocugen. “We are extremely encouraged that with this Phase 3 study design more than 50% of intent to treat RHO patients would meet the responder criteria, which is demonstrating 2 or greater Lux level of improvement after one year of treatment based on Phase 1/2 study results.”

Currently there are approximately 110,000 patients in the United States with RP and 1.6 million patients globally. Of these patients, more than 10% have the RHO genetic mutation.

“We believe that the gene-agnostic clinical trial design provides an appropriate therapeutic option to include patients who have greater potential of benefiting from treatment,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “We are looking forward to working with our selected trial sites and leading retinal surgeons to deliver this novel modifier gene therapy to potentially address unmet medical needs.”

Ocugen previously announced that OCU400 has received orphan drug and RMAT designations from the FDA. With the initiation of the Phase 3 clinical trial, OCU400 remains on track for the 2026 BLA approval target.

About OCU400
OCU400 is the Company’s gene-agnostic modifier gene therapy product based on NHR gene, NR2E3. NR2E3 regulates diverse physiological functions within the retina—such as photoreceptor development and maintenance, metabolism, phototransduction, inflammation and cell survival networks. Through its drive functionality, OCU400 resets altered/affected cellular gene-networks and establishes homeostasis—a state of balance, which has the potential to improve retinal health and function in patients with inherited retinal diseases.

About RP
RP is a group of rare, genetic disorders that involve a breakdown and loss of cells in the retina, leading to vision loss and blindness. Currently, RP is associated with mutations in more than 100 genes.

There are no approved treatment options that slow or stop the progression of multiple forms of RP. Proposed treatments for RP include gene-replacement therapy, retinal implant devices, retinal transplantation, stem cells, vitamin therapy, and other pharmacological treatments. Current gene-replacement therapies are promising but are limited to treating just a single mutation. In addition, while gene therapies may provide a new functional gene, they do not necessarily eliminate the underlying genetic defect, which may still cause stress and toxic effects leading to retina degeneration. Therefore, the development of gene-specific replacement therapy is highly challenging, especially when multiple and unknown genes are involved. Thus, novel therapeutic approaches targeting broader RP disease in a gene agnostic manner offer greater hope for patients.

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

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

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

Release – Maia Biotechnology To Present At Two Investor Conferences In April 2024

Posted on by aweinsoff@channelchek.com

Research News and Market Data on MAIA

April 05, 2024 8:01am EDTDownload as PDF

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 Chairman and Chief Executive Officer Vlad Vitoc, M.D. will present at two investor conferences during the month of April: the 14th Annual LD Micro Invitational taking place April 8-9, 2024, in New York City; and the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference taking place April 17-18, 2024.

LD Micro:

Presentation:04:30PM ET on April 09, 2024*
Format:In-person and virtual
Location:Sofitel New York, New York City
Conference registration:Visit the conference website
Livestream registration:Visit Sequire Events
1×1 meetings:Upon request by registered investors

Noble Capital Markets:

Presentation:11:00AM ET on April 17, 2024*
Format:Virtual
Conference registration:Visit the conference website
1×1 meetings:Upon request by registered investors

*Please note that the presentation date and time are subject to change. Participants should refer to the final program agendas for up-to-date information.

MAIA’s lead candidate is THIO, a small molecule telomere-targeting anticancer agent that acts by producing direct telomeric DNA damage and inducing cancer-specific immune responses. THIO’s efficacy in non-small cell lung cancer (NSCLC) is being evaluated in THIO-101, a Phase 2 go-to-market clinical trial nearing completion, which is expected to be the first completed clinical study of a telomere-targeting agent in the field of cancer drug discovery and treatment. MAIA plans to pursue the FDA’s accelerated approval program for THIO.

Recent news from MAIA’s THIO-101 trial includes:

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/20240405374193/en/

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

Source: MAIA Biotechnology, Inc.

Released April 5, 2024