Tag: Biotechnology

Release – Data and Safety Monitoring Board Reviews Cohort 1 Safety Data and Approves Dosing Cohort 2 in the Clinical Trial of OCU200—a Novel Fusion Protein for Diabetic Macular Edema

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

March 18, 2025

PDF Version

  • OCU200 has a very favorable safety and tolerability profile
  • No serious adverse events related to the study drug have been reported
  • Dosing of the second cohort has been approved

MALVERN, Pa., March 18, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing of the first cohort in the dose-escalation portion of the Phase 1 study and approved continuation of dosing in the second cohort. OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin, with the potential to treat diabetic macular edema (DME).

“OCU200 is given intravitreally,” said Peter Chang, MD, FACS, Co-President and Partner of the Massachusetts Eye Research and Surgery Institution (MERSI). “No serious adverse events related to OCU200 have been reported to date.”

The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive two doses six weeks apart, and patients will be followed for up to 6 months.

“It is encouraging that we have successfully completed dosing in the low dose cohort for OCU200, a novel biologic that has a very favorable safety and tolerability profile,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “There remains a considerable unmet medical need for the 30% to 40% of DME patients who do not respond to current anti-VEGF therapies. OCU200 holds the promise of potentially benefiting all DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) patients.”

Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR, or wet AMD. Patients affected by them share common symptoms, such as blurriness in vision and progressive vision loss, as the diseases progress. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.

OCU200 has the potential to change the treatment landscape for DME, DR, and wet AMD with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors on active endothelial cells that play a crucial role in disease pathogenesis.

OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases. The Company intends to complete the Phase 1 OCU200 clinical trial in the second half of 2025 and to provide preliminary safety and efficacy updates throughout the year.

About OCU200

OCU200 is a recombinant fusion protein that consists of two parts connected by a linker: tumstatin, the active component, acts as an anti-inflammatory, anti-VEGF agent by binding to integrin receptors; and transferrin, which targets the drug to the choroid and retina by binding transferrin receptors on endothelial cells. These features will potentially enable OCU200 to reduce the vascular permeability, inflammation, and neovascularization that drive the pathophysiology of DME, DR, and wet AMD at a significantly lower dose compared to currently approved therapies.

About Ocugen, Inc.

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

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 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; the ability of OCU200 to perform in humans in a manner consistent with nonclinical or preclinical study 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com

Release – Gyre Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GYRE

March 17, 2025

PDF Version

Data from pivotal Phase 3 trial in CHB-associated liver fibrosis expected in Q2 2025

Commercial launch in the PRC of generic nintedanib for the treatment of IPF and avatrombopag maleate tablets for the treatment of CLD-associated thrombocytopenia expected in 2025

Initiation of U.S. Phase 2 trial of F351 in MASH-associated liver fibrosis expected in 2025

Full year 2025 total revenue guidance of $118 to $128 million

SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) — Gyre Therapeutics (“Gyre”) (Nasdaq: GYRE), a self-sustainable, commercial-stage biotechnology company with clinical development programs focusing on organ fibrosis, today announced financial results for the fourth quarter and full year ended December 31, 2024 and provided a business update.

“2025 is shaping up to be a pivotal year for Gyre across both our commercial-stage and clinical-stage portfolios. We plan to expand and enhance our commercial product offerings through the additions of nintedanib for IPF, SSc-ILD and PF-ILD, as well as avatrombopag for CLD-associated thrombocytopenia and chronic idiopathic thrombocytopenia (“ITP”). Given our proven track record and extensive sales and marketing platform, we are confident in our ability to successfully launch and expand these two products in the PRC,” said Han Ying, Ph.D., Chief Executive Officer of Gyre Therapeutics. “In parallel, we expect to share topline data from our pivotal Phase 3 trial in CHB-associated liver fibrosis in the second quarter of 2025, which will help inform our U.S. Phase 2 proof-of-concept trial of F351 in MASH-associated liver fibrosis.”

Full Year 2024 Business Highlights and Upcoming Milestones

Commercial-Stage Updates

  • ETUARY (Pirfenidone) sales update: For the year ended December 31, 2024, Gyre Pharmaceuticals generated $105.0 million primarily in sales of ETUARY.
  • Nintedanib: In May 2024, Gyre Pharmaceuticals executed a comprehensive agreement with Jiangsu Wangao Pharmaceuticals Co., Ltd. to obtain the drug registration certificate for and became the marketing authorization holder of nintedanib, the other product approved for the treatment of treatment of idiopathic pulmonary fibrosis (“IPF”). In addition, it has also been approved for the treatment of SSc-ILD and PF-ILD. Gyre Pharmaceuticals plans to initiate commercialization of the nintedanib product in the PRC in 2025.
  • Avatrombopag: In June 2024, Gyre Pharmaceuticals received approval from China’s National Medical Products Administration (“NMPA”) for avatrombopag maleate tablets for the treatment of thrombocytopenia associated with chronic liver disease (“CLD”) and chronic idiopathic thrombocytopenia (“ITP”) in adult patients undergoing elective diagnostics procedures or therapy. Gyre Pharmaceuticals plans to begin commercialization of avatrombopag in 2025.

Pipeline Development Updates

F351 (Hydronidone):

  • All patients completed 52-week pivotal Phase 3 trial in chronic hepatitis B (“CHB”)-associated liver fibrosis in the PRC. In October 2024, Gyre Pharmaceuticals announced the last patient completed the 52-week pivotal Phase 3 trial. The trial is evaluating 248 patients with CHB-associated liver fibrosis in the PRC with a primary endpoint of the reduction of the liver fibrosis score (Ishak Scoring System) by at least one stage after taking F351 in combination with entecavir. Gyre expects to report topline data in the second quarter of 2025.
  • Plans to initiate a Phase 2 clinical trial in metabolic dysfunction-associated steatohepatitis (“MASH”)-associated liver fibrosis in 2025. Pending the results from the pivotal Phase 3 trial in CHB-associated liver fibrosis, Gyre intends to initiate a Phase 2 proof-of-concept trial in the U.S. to evaluate F351 for the treatment of MASH-associated liver fibrosis in 2025.

F573:

  • F573 is a caspase inhibitor and a potential Category 1 new drug for the treatment of acute/acute on-chronic liver failure (“ALF/ACLF”). Completion of the Phase 2 clinical trial of F573 as a treatment for ALF/ACLF is expected by the end of 2026.

F230:

  • F230, a selective endothelin receptor agonist for the treatment of pulmonary arterial hypertension (“PAH”), is expected to begin a Phase 1 trial in 2025.

F528:

  • F528, a novel anti-inflammation agent with the potential to modify the progression of chronic obstructive pulmonary disease (“COPD”), is undergoing preclinical studies as a potential first-line therapy for the treatment of COPD. Gyre plans to submit an IND application in 2026.

Corporate Updates

  • In January 2025, appointed Ping Zhang to the Company’s Board of Directors as the lead independent director and member of the Nominating Committee. In addition, Ying Luo, Ph.D., resigned as Chairman and member of the Board of Directors of Gyre and Gyre Pharmaceuticals, Gyre’s majority indirectly owned subsidiary in the People’s Republic of China (“PRC”), to focus on other responsibilities at GNI Group Ltd. Songjiang Ma has been appointed Chairman of the Board of Directors of Gyre Pharmaceuticals.

Financial Results

Cash Position

As of December 31, 2024, Gyre had cash, cash equivalents, short-term and long-term bank deposits of $51.2 million.

Financial Results for the Three Months Ended December 31, 2024

  • Revenues: Revenues for the three months ended December 31, 2024 were $27.9 million, compared to $27.1 million for the same period in 2023. The $0.8 million increase was primarily driven by a $1.0 million increase in ETUARY’s revenue and a $0.2 million decrease in generic drug revenue. The growth in ETUARY sales was attributed to the active expansion of the IPF treatment market, increased market penetration, and a stronger focus on ETUARY sales. To support future revenue growth, Gyre Pharmaceuticals plans to commercially launch two new products, nintedanib and avatrombopag, in 2025, which will be supported by its extensive sales and marketing platform in the PRC.
  • Cost of Revenues: For the three months ended December 31, 2024, cost of revenues was $1.2 million, compared to $1.3 million for the same period in 2023. The $0.1 million decrease was primarily driven by a $0.2 million decrease in generic drug cost due to the decrease in sales and a $0.1 million decrease in factory stoppage loss due to factory renovation in 2023, offset by a $0.2 million increase due to the increase of ETUARY’s cost due to the increase in sales.
  • Selling and Marketing Expense: For the three months ended December 31, 2024, selling and marketing expense was $16.9 million, compared to $16.5 million for the same period in 2023. The increase was primarily driven by a $2.1 million increase in promotion expense and conference expenses, offset by a $1.1 million decrease in selling and marketing payroll costs, a $0.3 million decrease in stock-based compensation expense and a $0.3 million decrease in travel and miscellaneous expenses.
  • Research and Development Expense: For the three months ended December 31, 2024, research and development expense was $3.7 million, compared to $4.6 million for the same period in 2023. The decrease was primarily driven by a $0.5 million decrease in pre-clinical and clinical research expenses and a $0.5 million decrease in stock-based compensation expense, offset by a $0.1 million increase in miscellaneous expense.
  • General and Administrative Expense: For the three months ended December 31, 2024, general and administrative expense was $5.5 million, compared to $10.1 million for the same period in 2023. The decrease was primarily driven by a $5.8 million decrease in stock-based compensation cost, offset by a $0.8 million increase in the functional and administrative department’s personnel cost and a $0.4 million increase in professional expense, including legal and consulting fees.
  • Income (Loss) from Operations: For the three months ended December 31, 2024, income from operations was $0.7 million, compared to $91.1 million loss from operation for the same period in 2023. The increase in income from operations was driven primarily by acquired in-process research and development expense recognized in the fourth quarter of 2023 and there was no such expense in the same period in 2024.
  • Net Income (Loss): For the three months ended December 31, 2024, net income was $0.6 million, compared to $101.0 million net loss for the same period in 2023.
  • Non-GAAP Adjusted Net Income: For the three months ended December 31, 2024, non-GAAP adjusted net income was $1.1 million, compared to $2.1 million for the same period in 2023. The decrease was primarily driven by the costs of being a public company for three months in 2024, as compared to two months in 2023.

Financial Results for the Full Year Ended December 31, 2024

  • Revenues: Revenues for the full year ended December 31, 2024 were $ 105.8 million, compared to $113.5 million for the same period in 2023. The $7.7 million decrease was primarily driven by a $7.1 million decrease in ETUARY’s revenue and a $0.6 million decrease in generic drug revenue as a result of decreased sales volumes. The decrease in ETUARY and generic drug sales volumes was due to fluctuations in the Chinese economy that significantly affected demand for anti-fibrosis drugs and decreasing healthcare spending generally. To support future revenue growth, Gyre plans to commercially launch two new products, nintedanib and avatrombopag, in 2025, which will be supported by Gyre Pharmaceuticals’ extensive sales and marketing platform across the PRC.
  • Cost of Revenues: For the full year ended December 31, 2024, cost of revenues was $3.9 million, compared to $4.6 million for the same period in 2023. The $0.7 million decrease was primarily driven by a $0.5 million factory stoppage loss due to factory renovation in 2023, which did not occur in 2024, and a $0.2 million decrease due to decreased sales volumes.
  • Selling and Marketing Expense: For the full year ended December 31, 2024, selling and marketing expense was $57.5 million, compared to $61.2 million for the same period in 2023. The decrease was primarily driven by a $2.4 million decrease in conference costs and promotion expense due to decreased sales activities, a $0.9 million decrease in selling and marketing payroll costs due to the decrease of sales of ETUARY in 2024, a $0.3 million decrease in share base compensation expense, and a $0.1 million decrease in miscellaneous expenses.
  • Research and Development Expense: For the full year ended December 31, 2024, research and development expense was $12.0 million, compared to $13.8 million for the same period in 2023. The decrease was primarily from Gyre Pharmaceuticals, and was driven by a $0.3 million decrease in materials and utilities, a $1.3 million decrease in pre-clinical research expense due to several research and development projects advancing to the clinical trials stage or reaching the application phase in 2024, and a $0.4 million decrease in staff cost due to reduced headcount, and a $0.5 million decrease in stock-based compensation, related to options being fully vested in 2023, which did not occur in 2024, This overall decrease was partially offset by a 0.7 million increase in general research and development expense from Gyre Therapeutics due to increased consulting fees.
  • General and Administrative Expense: For the full year ended December 31, 2024, general and administrative expense was $16.1 million, compared to $14.7 million for the same period in 2023. The increase was primarily driven by costs associated with being a public company, including a $1.9 million increase in professional expense, a $2.1 million increase in miscellaneous expenses and a $3.0 million increase in the functional and administrative department’s personnel cost, offset by a $5.6 million decrease in stock-based compensation cost.
  • Income (loss) from Operations: For the full year ended December 31, 2024, income from operations was $16.2 million, compared to $67.2 million loss for the same period in 2023. The increase in income from operations was driven primarily by acquired in-process research and development expense recognized in 2023 and there was no such expense in the same period in 2024.
  • Net Income (loss): For the full year ended December 31, 2024, net income was $17.9 million, compared to $85.5 million net loss for the same period in 2023.
  • Non-GAAP Adjusted Net Income: For the full year ended December 31, 2024, non-GAAP adjusted net income was $16.9 million, compared to $25.4 million for the same period in 2023. The decrease was primarily driven by a $7.7 million decline in revenue and a $1.1 million increase in operating expenses. Despite these changes, the gross profit margin remained consistent.

Full Year 2025 Financial Guidance

For the full year 2025, the Company expects to generate revenues of $118 to $128 million, representing growth of 11.3% to 20.8% over 2024 revenue, primarily driven by the anticipated commercial launches of nintedanib and avatrombopag and sales of ETUARY.

 Guidance Range
  
Total Revenue$118 to $128 million
  

Please note the following regarding the total revenue guidance:

  • Guidance assumes a constant foreign currency exchange rate.
  • Guidance assumes no significant economic disruption or downturn.

Use of Non-GAAP Financial Measures by Gyre Therapeutics, Inc.

Gyre reports financial results in accordance with accounting principles generally accepted in the United States (“GAAP”). This release presents the financial measure “adjusted net income,” which is not calculated in accordance with GAAP. The most directly comparable GAAP measure for this non-GAAP financial measure is “net income.” Adjusted net income presents Gyre’s results of operations after excluding gain from change in fair value of warrants, stock-based compensation, and provision for income taxes. This is meant to supplement, and not substitute, Gyre’s financial information presented in accordance with GAAP. Adjusted net income as defined by Gyre may not be comparable to similar non-GAAP measures presented by other companies. Management believes that presenting adjusted net income provides investors with additional useful information in evaluating the Gyre’s performance and valuation. See the reconciliation of adjusted net income to net income in the section titled “Reconciliation of GAAP to Non-GAAP Financial Measures” below.

About Hydronidone (F351)

F351 is a structural analogue of the approved anti-fibrotic (IPF) drug Pirfenidone and has been shown to inhibit in vitro both p38γ kinase activity and TGF-β1-induced excessive collagen synthesis in hepatic stellate cells (“HSCs”), which are recognized as critical event in the development and progression of fibrosis in the liver. This is further supported by its anti-proliferative effects on the HSCs in the liver. In vitro anti-fibrotic effects of F351 were also confirmed in several established in vivo models of liver fibrosis such as CCI4-induced liver fibrosis mouse model, DMN-induced liver fibrosis rat model, and HSA-induced liver rat model, as well as mouse model of MASH fibrosis (CCI4+Western High Fat Diet).

About Gyre Pharmaceuticals

Gyre Pharmaceuticals is a commercial-stage biopharmaceutical company committed to the research, development, manufacturing and commercialization of innovative drugs for organ fibrosis. Its flagship product, ETUARY® (Pirfenidone capsule), was the first approved treatment for IPF in the PRC in 2011 and has maintained a prominent market share (2024 net sales of $105.0 million). In addition, Gyre Pharmaceuticals is evaluating F351 in a Phase 3 clinical trial in CHB-associated liver fibrosis in the PRC, which is expected to readout topline data by Q2 2025. F351 received Breakthrough Therapy designation by the NMPA Center for Drug Evaluation in March 2021. Gyre Pharmaceuticals is also developing treatments for PD, DKD, COPD, PAH and ALF/ACLF. In October 2023, Gyre Therapeutics acquired an indirect majority interest in Gyre Pharmaceuticals (also known as Beijing Continent Pharmaceuticals Co., Ltd.).

About Gyre Therapeutics

Gyre Therapeutics is a biopharmaceutical company headquartered in San Diego, CA, with a primary focus on the development and commercialization of F351 (Hydronidone) for the treatment of MASH-associated fibrosis in the U.S. Gyre’s development strategy for F351 in MASH is based on the company’s experience in MASH rodent model mechanistic studies and CHB-induced liver fibrosis clinical studies. Gyre is also advancing a diverse pipeline in the PRC through its indirect controlling interest in Gyre Pharmaceuticals, including ETUARY therapeutic expansions, F573, F528, and F230.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical facts included in this press release, are forward-looking statements, including statements concerning: the expectations regarding Gyre’s research and development efforts, timing of expected clinical readouts, including timing of topline data from Gyre Pharmaceuticals’ Phase 3 clinical trial evaluating F351 for the treatment of CHB-associated liver fibrosis in the PRC, initiation of Gyre’s Phase 2 trial in the U.S. for F351 for the treatment of MASH-associated liver fibrosis, timing of completion of Gyre’s Phase 2 clinical trial in the PRC of F573 for ALF/ACLF, initiation of Phase 1 trial of F230 for the treatment of PAH and IND submission of F528 in COPD, the expectations regarding commercial launch of nintedanib and avatrombopag maleate tablets, interactions with regulators, expectations regarding future product sales, and Gyre’s financial position and cash resources. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our plans, estimates, and expectations, as of the date of this press release. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this press release. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: Gyre’s ability to execute on its clinical development strategies; positive results from a clinical trial may not necessarily be predictive of the results of future or ongoing clinical trials; the timing or likelihood of regulatory filings and approvals; competition from competing products; the impact of general economic, health, industrial or political conditions in the United States or internationally; the sufficiency of Gyre’s capital resources and its ability to raise additional capital. Additional risks and factors are identified under “Risk Factors” in Gyre’s Annual Report on Form 10-K for the year ended December 31, 2023 filed on March 27, 2024 and in other filings with the Securities and Exchange Commission.

Gyre expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.

For Investors:
Stephen Jasper
stephen@gilmartinir.com

View Full Release Here.

Release – GeoVax to Report Fourth Quarter and Full Year 2024 Financial Results and Provide Corporate Update on March 27, 2025

Posted on by aweinsoff@channelchek.com

Research News and Market Data on GOVX

GeoVax to Host Conference Call at 4:30 PM ET

Atlanta, GA, March 17, 2025 – GeoVax Labs, Inc. (Nasdaq: GOVX), a clinical-stage biotechnology company developing immunotherapies and vaccines against cancer and infectious diseases, today announced that it will report 2024 financial results on Thursday, March 27, 2025, after the close of U.S. markets. Following the release, management will host a live conference call and webcast, including Q&A, at 4:30 p.m. ET to provide a corporate update and discuss financial results.

Conference Call Details

To access the live conference call, participants may register here. The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the GeoVax website at geovax.com/investors. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. While not required, it is recommended that participants join the call ten minutes prior to the scheduled start. An archive of the audio webcast will be available on GeoVax’s website approximately two hours after the conference call and will remain available for at least 90 days following the event.

About GeoVax

GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel vaccines for many of the world’s most threatening infectious diseases and therapies for solid tumor cancers. The company’s lead clinical program is GEO-CM04S1, a next-generation COVID-19 vaccine for which GeoVax was recently awarded a BARDA-funded contract to sponsor a 10,000-participant Phase 2b clinical trial to evaluate the efficacy of GEO-CM04S1 versus an approved COVID-19 vaccine. In addition, GEO-CM04S1 is currently in three Phase 2 clinical trials, being evaluated as (1) 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, (2) a booster vaccine in patients with chronic lymphocytic leukemia (CLL) and (3) a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. In oncology the lead clinical program is evaluating a novel oncolytic solid tumor gene-directed therapy, Gedeptin(R), having recently completed a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. A Phase 2 clinical trial in first recurrent head and neck cancer, evaluating Gedeptin(R) combined with an immune checkpoint inhibitor is planned. GeoVax has a strong IP portfolio in support of its technologies and product candidates, holding worldwide rights for its technologies and products. The Company has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information about the current status of our clinical trials and other updates, visit our website: www.geovax.com.

Company Contact:

info@geovax.com

678-384-7220

Investor Relations Contact:

geovax@precisionaq.com

212-698-8696

Media Contact:

sr@roberts-communications.com

202-779-0929

Cadrenal Therapeutics (CVKD) – FY2024 Report Reviews A Pivotal Year For Tecarfarin

Posted on by cpereira@noblefcm.com


Friday, March 14, 2025

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.

FY2024 Was A Productive Year. Cadrenal reported a 4Q24 loss of $4.2 million or $(2.55) per share and FY2024 loss of $10.7 million or $(8.73) per share. An important development discussed in our Research Note on March 5 was Cadrenal’s announcement of a collaborative agreement with Abbott (ABT, Not Rated) for support of its pivotal trial testing tecarfarin in patients with left ventricular assist (LVAD) devices. Cash and equivalents on December 31 were $10.0 million.

Tecarfarin Is In Development For Several Patient Populations With Coagulation Needs. Many patients that are at risk for cardiovascular events (stroke, embolism, deep vein thrombosis) take anticoagulants in the direct oral anticoagulant class (DOACs, such as Eliquis or Xarelto). However, there are several patient populations that must take warfarin, an older drug, due to lack of efficacy or high bleeding risk. Tecarfarin is being developed to replace warfarin in these populations. Cadrenal has Orphan Drug designation from the FDA for implanted mechanical devices (LVADs) and prevention of systemic thromboembolism in end-stage kidney disease (ESKD) and atrial fibrillation (AFib).


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 – Unicycive to Highlight Patient Reported Outcomes Data at Upcoming Medical Meetings Showing Oxylanthanum Carbonate Reduced Pill Burden and Improved Adherence in Treatment of Hyperphosphatemia

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

March 13, 2025 7:00am EDT Download as PDF

LOS ALTOS, Calif., March 13, 2025 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease, today announced that it will present patient reported outcomes data from its pivotal UNI-OLC-201 clinical study characterizing the potential impact of oxylanthanum carbonate (OLC) on the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis. These data will be presented at three medical meetings, including the 2025 Annual Dialysis Conference (ADC), March 13-16, 2025, National Kidney Foundation (NKF) Spring Clinical Meetings, April 10-13, 2025, and the 2025 American Nephrology Nurses Association (ANNA) National Symposium, being held on May 1-4, 2025.

Unicycive’s investigational drug OLC leverages proprietary nanoparticle technology to reduce the number and size of pills that patients must take. If approved, OLC may provide patients and their physicians with a welcome new option to control hyperphosphatemia. The New Drug Application (NDA) for OLC was accepted by the U.S. Food and Drug Administration (FDA) for the treatment of hyperphosphatemia in patients with chronic kidney disease on dialysis. The FDA set a Prescription Drug User Fee Act (PDUFA) Target Action Date of June 28, 2025.

“Despite the availability of several approved phosphate binders, hyperphosphatemia remains uncontrolled in 75% of people in the U.S. on dialysis due to challenges of insufficient potency, pill burden and unpalatable formulations. There is a critical need for more effective solutions,” said Shalabh Gupta, M.D., Chief Executive Officer of Unicycive. “Innovative solutions such as OLC that improve phosphate control and minimize pill size and count have the potential to significantly improve adherence, empowering those on dialysis to manage their treatment more effectively.”

Unicycive abstracts to be presented at the upcoming medical meetings include:

ADC

  • Title: Patient-Reported Outcomes in a Pivotal Clinical Study of Hyperphosphatemia: Oxylanthanum Carbonate Reduces Pill Burden by Half and Improves Adherence – Poster #: A-6870
  • Presentation Details: Friday, March 14, 5:30-7:30 p.m. PT
  • Presenting Author: Doug Jermasek

NKF Spring Clinical Meetings

  • Title: Patient-Reported Outcomes in a Pivotal Clinical Study of Hyperphosphatemia: Oxylanthanum Carbonate Reduces Pill Burden by Half and Improves Adherence – Poster #: G-018
  • Presentation Details: Thursday, April 10, from 5:15-7:30 p.m. ET
  • Presenting Author: Guru Reddy, PhD
  • Title: Pill Burden and Large Tablet Size Are Key Barriers to Phosphate Binder Adherence in Dialysis Patients – Poster #: G-297
  • Presentation Details: Thursday, April 10, from 5:15-7:30 p.m. ET
  • Presenting Author: Dr. Hill Gallant, PhD, RD, Associate Professor of Nutrition in the Department of Food Science and Nutrition at the University of Minnesota-Twin Cities

ANNA

  • Title: Pill Burden and Large Tablet Size Are Key Barriers to Phosphate Binder Adherence in Dialysis Patients
  • Presentation Details: Friday, May 2, starting at 8:45 a.m. PT

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 $2.28 billion, with the North America accounting for more than $1 billion of that total.2 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.3

Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. The NDA submission package is based on data from three clinical studies (a Phase 1 study in healthy volunteers, a bioequivalence study in healthy volunteers, and a tolerability study of OLC in CKD patients on dialysis), multiple preclinical studies, and the chemistry, manufacturing and controls (CMC) data. OLC is protected by a strong global patent portfolio including issued patents on composition of matter with exclusivity until 2031, and with the potential for patent term extension until 2035.

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.4

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. Positive pivotal trial results were reported in June 2024 for OLC, and a New Drug Application (NDA) is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) Target Action Date of June 28, 2025. OLC is protected by a strong global patent portfolio including an issued patent on composition of matter with exclusivity until 2031, and with the potential patent term extension until 2035 after OLC approval. Unicycive’s second asset, UNI-494, is a patent-protected new chemical entity in clinical development for the treatment of conditions related to acute kidney injury. UNI-494 has successfully completed a Phase 1 trial. For more information, please visit Unicycive.com and follow us on LinkedIn, X, 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.

1Reason Research, LLC 2022 survey. Results here.
2 Fortune Business Insights™, Hyperphosphatemia Treatment Market, 2023-2030
3 US-DOPPS Practice Monitor, May 2021; http://www.dopps.org/DPM
4 Block GA, Klassen PS, Lazarus JM, Ofsthun N, Lowrie EG, Chertow GM. Mineral metabolism, mortality, and morbidity in maintenance hemodialysis. J Am Soc Nephrol. 2004 Aug;15(8):2208-18. doi: 10.1097/01.ASN.0000133041.27682.A2. PMID: 15284307.

Investor Contacts:

Kevin Gardner
LifeSci Advisors
kgardner@lifesciadvisors.com

Media Contact:

Rachel Visi
Real Chemistry
redery@realchemistry.com

Primary Logo

Source: Unicycive Therapeutics, Inc.

Released March 13, 2025

Mallinckrodt and Endo Announce $6.7B Merger to Create Specialty Pharma Giant

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Mallinckrodt and Endo will combine to form a diversified pharmaceutical powerhouse.
– The merger will create a company with $3.6 billion in projected 2025 revenue and $1.2 billion in adjusted EBITDA.
– The new entity will focus on branded specialty pharmaceuticals while planning to separate its generics and sterile injectables business.

Pharmaceutical companies Mallinckrodt and Endo have agreed to merge in a $6.7 billion deal that will create a new powerhouse in the specialty medication market, the companies announced Thursday.

The stock-and-cash transaction, expected to close in the second half of 2025, combines Mallinckrodt’s rare disease portfolio with Endo’s sterile injectables business, positioning the merged entity to compete more effectively in high-margin specialty pharmaceutical segments.

Shares of both companies jumped on the news, with Mallinckrodt stock up 7.2% and Endo shares surging 12.3% in morning trading.

Under the terms of the agreement, Endo shareholders will receive $80 million in cash while maintaining a 49.9% stake in the combined company. Mallinckrodt shareholders will hold the remaining 50.1% interest, with Mallinckrodt serving as the parent company.

The merged firm projects $3.6 billion in revenue for 2025 with $1.2 billion in adjusted EBITDA. Management expects to achieve $150 million in annual cost synergies by the third year post-merger, with $75 million realized in the first year.

Goldman Sachs is providing $900 million in committed financing to support the transaction. The combined company will operate with a net leverage ratio of approximately 2.3x, giving it significant financial flexibility for future growth initiatives.

Siggi Olafsson, CEO of Mallinckrodt, will lead the combined entity. The companies emphasized that the complementary nature of their businesses would maximize operational efficiencies while maintaining focus on innovation.

A key component of the merger strategy involves the eventual separation of the combined sterile injectables and generics businesses. While these operations will initially be integrated, management plans to spin off this unit as a standalone company, pending board approval and market conditions.

The core branded specialty pharmaceuticals business will focus on rare diseases and hospital-based therapies, areas where both companies have established market positions. With 17 manufacturing facilities and 30 distribution centers predominantly in the United States, the company will employ approximately 5,700 people worldwide.

According to Endo’s interim CEO Scott Hirsch, the merger will leverage complementary strengths and create immediate scale advantages in key therapeutic areas. The planned separation of the generics business aims to further sharpen focus on high-growth specialty markets.

The Mallinckrodt-Endo merger comes amid increasing consolidation in the pharmaceutical sector as companies look to gain scale and portfolio diversification.

Analysts at Morgan Stanley noted that the deal makes strategic sense for both companies, particularly given the challenges they’ve faced individually in recent years. The combined entity will have greater resources to invest in R&D and a stronger position in negotiations with payers and hospital systems.

However, some analysts expressed caution about integration risks and the ambitious timeline for the planned business separation. Healthcare analysts at JP Morgan pointed out that executing a merger of this scale while simultaneously preparing for a business spinoff creates significant operational complexity. The management team will need to carefully balance these priorities to deliver the promised synergies.

The combined company will be listed on the New York Stock Exchange following the transaction’s completion.

Take a moment to take a look at Noble Capital Markets’ Biotechnology Research Analyst Robert Leboyer’s coverage list.

Bristol Myers Squibb to Acquire 2seventy Bio in $286 Million All-Cash Deal

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– BMS is acquiring 2seventy bio for $5.00 per share, an 88% premium to its last closing price.
– The deal strengthens BMS’s cell and gene therapy portfolio, particularly in multiple myeloma treatment.
– The acquisition comes amid increased M&A activity in biotech, signaling confidence in the sector’s long-term potential.

Bristol Myers Squibb (BMY) has announced a definitive agreement to acquire 2seventy bio (TSVT) in an all-cash deal valued at approximately $286 million. This acquisition further strengthens BMS’s foothold in the oncology space, particularly through its access to Abecma, an FDA-approved CAR T-cell therapy for multiple myeloma. The deal is expected to close in the second quarter of 2025, pending regulatory approvals and shareholder consent.

BMS’s acquisition of 2seventy bio aligns with its broader strategy to expand its presence in the high-growth cell and gene therapy market. 2seventy bio has focused exclusively on Abecma, a treatment developed in collaboration with BMS, to extend and improve the lives of patients with relapsed or refractory multiple myeloma. With this acquisition, BMS will take full control of Abecma’s commercialization and development, streamlining operations and potentially accelerating future advancements.

Chip Baird, CEO of 2seventy bio, emphasized the significance of the transaction, stating: “This acquisition ensures Abecma continues to reach patients in need while maximizing value for our stakeholders.” BMS, with its expansive resources and global reach, is well-positioned to drive further innovation in the cell therapy space.

The biotech sector has seen a resurgence in M&A activity, with pharmaceutical giants seeking to bolster their pipelines amid ongoing scientific advancements and a challenging regulatory landscape. The acquisition of 2seventy bio comes at a time when investors are looking for signs of stability in biotech, and deals like this reinforce confidence in the sector’s long-term growth potential.

The broader biotechnology sector, as measured by the iShares Biotechnology ETF (IBB), has posted gains year-to-date, reflecting renewed investor interest in the space. As larger pharmaceutical companies look to capitalize on cutting-edge therapies, small and mid-cap biotech firms with promising assets are becoming increasingly attractive acquisition targets. The deal values 2seventy bio at a significant premium, rewarding shareholders with an 88% increase from its prior trading price. However, it also raises questions about the long-term independence of innovative biotech firms. While consolidation can lead to greater efficiency and resource allocation, it may also reduce competition and limit the number of standalone biotech companies driving early-stage innovation.

For BMS, the acquisition is a strategic move to reinforce its oncology pipeline amid growing competition in the CAR T-cell therapy space. With this deal, BMS is betting on continued demand for personalized cell-based therapies and positioning itself to lead in this evolving field. Biotech acquisitions are often driven by the need for pharmaceutical companies to secure new revenue streams as patents on existing drugs expire. By acquiring 2seventy bio, BMS gains a competitive advantage in the high-value oncology segment, ensuring its ability to remain a dominant force in the industry.

Bristol Myers Squibb’s acquisition of 2seventy bio represents a significant development in the biotech sector. As M&A activity accelerates, the deal underscores the importance of targeted therapies in oncology and highlights the ongoing push by pharmaceutical giants to secure cutting-edge treatments. For investors, this acquisition may serve as a signal that biotech remains a strong sector, with potential for both innovation and consolidation in the years ahead.

Gyre Therapeutics, Inc (GYRE) – Initiation of Coverage: Focused On Fibrosis

Posted on by cpereira@noblefcm.com


Tuesday, March 11, 2025

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 Are Initiating Coverage Of Gyre Therapeutics With An Outperform Rating. Gyre Therapeutics is a pharmaceutical company developing drugs for inflammatory diseases that lead to fibrosis. It currently markets Etuary (pirfenidone) in China for idiopathic pulmonary fibrosis. The lead drug in the pipeline is Hydronidone, a new molecule derived from pirfenidone, that is in a Phase 3 clinical trial in China. The data announcement is expected to report Phase 3 clinical trial results in March 2025.

Hydronidone Was Developed To Improve Efficacy and Side Effects. Hydronidone is a structural analogue of pirfenidone that was developed to improve efficacy with a more tolerable side effect profile. It is in Phase 3 trial in China for fibrosis of the liver after hepatitis B (HBV) infections. Hydronidone targets steps in the Transforming Growth Factor (TGF)-ß1 pathway as well as the downstream genes and liver cells it activates to produce fibrotic tissue. Data from the Phase 3 in China will be used to design a Phase 2a trial in the US, expected to begin in late FY2025.


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. 

Sun Pharma to Acquire Checkpoint Therapeutics in $355 Million Deal

Posted on by slightbourne@noblecapitalmarkets.com
Key Points:
– Sun Pharma announced a $355 million acquisition of Checkpoint Therapeutics to expand its oncology portfolio.
-The biotech sector is showing strength, with the IBB ETF up 3.5% year-to-date.
– The acquisition brings FDA-approved cancer treatment UNLOXCYT™ to Sun Pharma’s global portfolio.

Sun Pharmaceutical Industries has announced its acquisition of Checkpoint Therapeutics in a $355 million deal aimed at strengthening its presence in the oncology sector. Checkpoint, a commercial-stage biotech company, has developed UNLOXCYT™ (cosibelimab-ipdl), the first and only FDA-approved anti-PD-L1 treatment for metastatic or locally advanced cutaneous squamous cell carcinoma (cSCC). This acquisition is expected to accelerate global access to this innovative cancer treatment and expand Sun Pharma’s onco-dermatology portfolio.

The broader biotech sector is emerging as a bright spot in an otherwise volatile market. The iShares Biotechnology ETF (IBB) is up 3.5% year-to-date, reflecting increased investor confidence in the sector’s growth potential. Unlike other areas of the stock market that have struggled amid rising interest rates and economic uncertainty, biotech has benefited from continued innovation, regulatory approvals, and M&A activity.

The deal provides Sun Pharma with immediate access to a groundbreaking cancer treatment, allowing the company to leverage its global footprint to scale distribution. With approximately 1.8 million new cSCC cases diagnosed annually in the U.S. alone, there is a substantial market opportunity for UNLOXCYT™. Sun Pharma expects to enhance Checkpoint’s commercialization efforts and drive greater adoption of the therapy in key markets worldwide.

In addition to the $4.10 per share cash payment, Checkpoint shareholders will receive a contingent value right (CVR) of up to $0.70 per share if UNLOXCYT™ secures approval in major European markets by specific deadlines. This structure incentivizes timely regulatory approvals and ensures continued development efforts.

The Sun Pharma-Checkpoint deal is the latest in a wave of biotech acquisitions, reflecting growing interest from larger pharmaceutical firms seeking to expand their specialty drug pipelines. Given the sector’s recent performance and ongoing medical advancements, further consolidation in biotech could be on the horizon.

For investors, the strong performance of biotech stocks and M&A activity suggest that the sector could be positioned for continued growth. As traditional sectors face headwinds, biotech’s mix of innovation, regulatory catalysts, and strategic acquisitions make it an attractive space to watch.

Ocugen (OCGN) – Ocugen Reports FY2024 With Progress Toward “3 BLA Filings In 3 Years”

Posted on by cpereira@noblefcm.com


Thursday, March 06, 2025

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.

Clinical Progress Expected To Lead To Filings For Three Product Approvals. Ocugen reported a 4Q24 loss of $13.9 million or $(0.05) per share and FY2024 loss of $54.1 million or $(0.20) per share. The company made significant progress in its clinical trials during the quarter and since the start of FY2025. It has also received regulatory designations that accelerate product approval. The company had $58.5 million in cash on December 31, sufficient to fund operations through 1Q26.

Clinical Trial Advances Point To Three BLAs In 3 Years. Ocugen has made significant progress with three products for three diseases that lead to vision loss. The three ongoing trials are Phase 3 for OCU400, the Phase 2/3 for OCU410ST in Stargardt disease, and the Phase 1/2 trial for GA. These trials are on schedule for filing applications for approval in 2026, 2027, and 2028 respectively. OCU400 and OCU410ST have Orphan Drug designations that can accelerate approval, while GA is a large market of over 10 million patients in the US alone.


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 Fourth Quarter and Full Year 2024 Financial Results

Posted on by aweinsoff@channelchek.com

Research News and Market Data on OCGN

March 5, 2025

PDF Version

Conference Call and Webcast Today at 8:30 a.m. ET

  • Reached an alignment with FDA on Phase 2/3 pivotal confirmatory clinical trial for OCU410ST for Stargardt disease potentially expediting clinical development by two to three years
  • Completed dosing in OCU410 Phase 2 ArMaDa clinical trial (N=51) for geographic atrophy (GA). Data and Safety Monitoring Board (DSMB) recently reviewed interim safety data from Phase 2. All subjects from multiple dose levels in Phase 2 to date demonstrated a very favorable safety and tolerability profile.
  • Positive 2-year long-term data across multiple mutations of retinitis pigmentosa (RP) from the Phase 1/2 clinical trial of OCU400 demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects at two years when compared to untreated eyes
  • OCU400 remains on track to meet 1H 2025 recruitment completion and potential BLA/MAA filings by mid-2026

MALVERN, Pa., March 05, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported fourth quarter and full year 2024 financial results along with a general business update.

“Ongoing data from all three clinical programs, along with patient testimonials, continue to support the benefit of one-time treatment from our revolutionary modifier gene therapy platform—as we are seeing consistent stabilization or improvement in treated eyes while maintaining a favorable safety and tolerability profile,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Ocugen. “Throughout 2024, the Company continually advanced its clinical programs in line with enrollment and dosing timelines, and we will continue to drive the product pipeline forward in 2025.”

Recently, the Company announced it will proceed with a Phase 2/3 pivotal confirmatory clinical trial of OCU410ST for Stargardt disease after alignment with the FDA. This accelerated regulatory pathway for OCU410ST is driven by the incredible unmet medical need that exists for 100,000 Stargardt patients in the U.S. and Europe who have no treatment option available. Accelerating the clinical timeline of OCU410ST is expected to save significant costs in addressing disease burden even sooner than anticipated.

The OCU410ST Phase 2/3 clinical trial will randomize 51 subjects, 34 of whom will receive a single, subretinal, 200-μL injection of OCU410ST at a concentration of 1.5 x 1011 vector genomes (vg)/mL in the eye with worse visual acuity, and 17 of whom will serve as untreated controls. One-year data will be utilized for the BLA filing planned for 2027. 6-month data from Phase 1 of the OCU410ST GARDian clinical trial demonstrated considerably slower lesion growth (52%) from baseline in treated eyes versus untreated fellow eyes and clinically meaningful 2-line (10-letter) improvement in visual function (BCVA), which is statistically significant (p=0.02) in treated eyes.

This week, the DSMB for the OCU410 ArMaDa clinical trial for GA convened and reviewed the safety and tolerability profile of an additional 15 subjects from the Phase 2 portion of the study. No serious adverse events related to OCU410 have been reported to date in all 60 subjects, including Phase 1. Unlike currently available treatments for GA, there were no cases of ischemic optic neuropathy, vasculitis, intraocular inflammation, endophthalmitis or choroidal neovascularization, and no adverse events of special interest.

Preliminary 9-month efficacy data of OCU410 in GA patients showed considerably slower lesion growth (44%) from baseline and clinically meaningful 2-line (10-letter) improvement in visual function (LLVA) in treated eyes compared to untreated eyes in the Phase 1 portion of the trial. Furthermore, a single subretinal OCU410 treatment preserves more retinal tissue around GA lesions of treated eyes at 9 months compared to published data on currently available GA therapies. GA, an advanced form of dry age-related macular degeneration, affects 2-3 million people in the U.S. and Europe combined.

Two-year data from the Phase 1/2 OCU400 clinical trial for RP demonstrated a durable and statistically significant (p=0.005) improvement in visual function (LLVA) in all evaluable treated subjects when compared to untreated eyes. 100% (10/10) of treated evaluable subjects showed improvement or preservation in visual function compared to untreated eyes, and OCU400 was observed to have a favorable safety and tolerability profile in all subjects across multiple mutations.

In parallel with clinical milestones, considerable regulatory accomplishments were achieved, including orphan medicinal product designation (OMPD) from the European Medicines Agency (EMA) for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD), and positive opinions from the EMA’s Committee for Advanced Therapies (CAT) for OCU400, OCU410, and OCU410ST Advanced Therapy Medicinal Product (ATMP) classification.

Ocugen’s biologic candidate moved into the clinic and patients are currently being dosed in the OCU200 Phase 1 clinical trial for diabetic macular edema (DME). OCU200 has the potential to change the treatment landscape for DME, diabetic retinopathy, and wet age-related macular degeneration with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors that play a crucial role in disease pathogenesis and holds the promise to benefit all DME patients, including the 30-40% of patients who do not respond to current anti-VEGF therapies.

“I am extremely proud of our team at Ocugen, who are working tirelessly to enroll patients across all four clinical programs in close collaboration with the study centers and investigators,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “It is very gratifying to think about the potential unmet medical need that can be addressed through modifier therapy for patients who currently have no options and are facing the prospect of losing their vision altogether.”

Finally, the Investigational New Drug (IND) application for OCU500—the Company’s inhaled mucosal vaccine for COVID-19 was cleared by the FDA. The National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, is expected to sponsor and conduct the Phase 1 trial to assess the safety, tolerability, and immunogenicity of OCU500 administered via two different routes, inhalation into the lungs and intranasally as a spray.

To extend the cash runway into the first quarter of 2026, and support Ocugen’s clinical trials, the Company secured $65 million in equity/debt financings in the second half of 2024. Further supporting Ocugen’s efforts to enable long-term shareholder value, garner significant visibility within the investment community, and broaden the shareholder base, the Company was included in the Russell Index in May 2024.

“Meaningful progress in 2024 and recent success with FDA brought us closer to our goal of three potential BLAs in the years—2026, 2027, 2028, encompassing RP, Stargardt and GA treatments, respectively,” said Dr. Musunuri. “While this is an ambitious goal, I am confident that we have the strategic and scientific expertise, and an unrelenting commitment to patients, to deliver on our commitment.”

Business Updates

Novel Modifier Gene Therapy Platform—Potentially Targeting Three BLA Filings in the Next Three Years

  • OCU400 –The European Commission (EC) provided a positive opinion from the EMA’s CAT for OCU400 ATMP classification. Actively enrolling patients in the U.S. and Canada in the Phase 3 liMeliGhT clinical trial for the treatment of RP and are on track to complete enrollment in the first half of 2025 and file BLA and MAA submissions in mid-2026.
  • OCU410ST –The EMA granted OMPD for OCU410ST for the treatment of ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19 (RP19), and cone-rod dystrophy 3 (CORD3). The FDA approved a Phase 2/3 pivotal confirmatory clinical trial for BLA filing. Plan to initiate the Phase 2/3 study mid-2025 and targeting BLA submission by 2027. The EC provided a positive opinion from the EMA’s CAT for OCU410ST ATMP classification.
  • OCU410 – Completed dosing in Phase 2 of the OCU410 Phase 1/2 ArMaDa clinical trial ahead of schedule. Intend to complete 1-year follow up in the first quarter of 2026 and targeting a BLA filing in 2028. The EC provided a positive opinion from the EMA’s CAT for OCU410 ATMP classification.

Financial Results

  • Fourth quarter — Research and development expenses for the three months ended December 31, 2024, were $8.3 million compared to $7.8 million for the three months ended December 31, 2023. General and administrative expenses for the three months ended December 31, 2024, were $6.3 million compared to $5.2 million for the three months ended December 31, 2023. Ocugen reported a $0.05 net loss per common share for the three months ended December 31, 2024, compared to a $0.04 net loss per common share for the three months ended December 31, 2023.
  • Full year — Research and development expenses for the year ended December 31, 2024, were $32.1 million compared to $39.6 million for the year ended December 31, 2023. General and administrative expenses for the year ended December 31, 2024, were $26.7 million compared to $32.0 million for the year ended December 31, 2023. Ocugen reported a $0.20 net loss per common share for the year ended December 31, 2024, compared to a $0.26 net loss per common share for the year ended December 31, 2023.
  • Ocugen’s cash and restricted cash, totaled $58.8 million as of December 31, 2024, compared to $39.5 million as of December 31, 2023. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the first quarter of 2026. The Company had 291.4 million shares of common stock outstanding as of December 31, 2024.

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 leadership 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: 5045393
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, 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.

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 and efficacy thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, expected cash runway into the first quarter of 2026, the ability to initiate new clinical programs, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing and future 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
AVP, Head of Communications
Tiffany.Hamilton@ocugen.com 

View Full Release Here.

Cadrenal Therapeutics (CVKD) – Development Agreement With Abbott Brings Another Tecarfarin Indication To Clinical Trials

Posted on by cpereira@noblefcm.com


Wednesday, March 05, 2025

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

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

Cadrenal and Abbott Announce The LVAD Collaboration We’ve Been Waiting For. Cadrenal announced a development agreement with Abbott (ABT, Not Rated) to develop Tecarfarin in patients with Abbott’s HeartMate3 LVAD (left ventricle assist device). Under the agreement, Abbott will support Cadrenal’s pivotal TECH-VLAD (TECarfarin Anticoagulation and Hemocompatibility with Left Ventricular Assist Devices) trial in its design, site selection, recruitment, and its HeartMate3 experience.

LVAD Patients Have An Unmet Need For A New Anticoagulant. While Direct Oral Anticoagulation Drug (DOAC) category has been highly successful, there are several populations where they are not effective or have safety risks. Patients with LVAD devices can only use warfarin, a drug that has variable efficacy with several drawbacks, including a requirement for frequent patient monitoring. LVAD patients have less effective anticoagulation and remain at high risk for coagulation events (bleeding, stroke, myocardial infarct). 


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. 

Ocugen (OCGN) – OCU410 and OCU410ST Receive ATMP Classification In Europe

Posted on by cpereira@noblefcm.com


Tuesday, March 04, 2025

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.

Ocugen Now Has Three Products With ATMP Designation. Two Ocugen products, OCU410 and OCU410ST, received Advanced Therapy Medicinal Product (ATMP) designation from the European Medicines Agency Committee for Advanced Therapies (EMA-CAT). These join OCU400, which received this designation for retinitis pigmentosa (RP) in February 2025. The designation is similar to the Breakthrough Therapy designation from the FDA, allowing increased interactions with the regulators and accelerating regulatory review.

OCU410 Has Completed Phase 2 Dosing In GA. The Phase 2 portion of the ArMaDa (pronounced ‘Armada”) trial has completed enrollment for OCU410 in geographic atrophy (GA), a lesion that results from advancing dry Age-related Macular Degeneration (dry AMD). The study has enrolled 51 patients randomized into a high dose arm, medium dose arm, or control. The completion of the dosing phase was ahead of our expected time frame, keeping the company on schedule to conduct Phase 3 in 2026 and potentially file for regulatory approval in 2028.


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.