Ocugen Inc - OCGN STOCK NEWS

Ocugen (NASDAQ: OCGN) announced positive safety data from the first cohort of its Phase 1 clinical trial for OCU200, a novel fusion protein designed to treat diabetic macular edema (DME). The Data and Safety Monitoring Board (DSMB) has approved proceeding with the second cohort after reviewing safety data from the initial group.

The trial is structured as a multicenter, open-label, dose-escalation study across three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). Patients receive two intravitreal injections six weeks apart with a 6-month follow-up period. No serious adverse events related to OCU200 have been reported.

OCU200, combining tumstatin and transferrin proteins, targets approximately 12 million people in the US and 130 million worldwide affected by DME, diabetic retinopathy (DR), and wet age-related macular degeneration. The company aims to complete the Phase 1 trial in H2 2025, particularly addressing the 30-40% of DME patients who don't respond to current anti-VEGF therapies.

Ocugen (NASDAQ: OCGN) reported its Q4 and full year 2024 financial results, highlighting significant progress in its gene therapy programs. The company reached FDA alignment for OCU410ST's Phase 2/3 pivotal trial for Stargardt disease, potentially accelerating development by 2-3 years. Positive clinical data was reported across multiple programs:

- OCU400 showed statistically significant (p=0.005) improvement in visual function at 2-year follow-up
- OCU410 completed Phase 2 dosing with favorable safety profile
- OCU410ST demonstrated 52% slower lesion growth and significant visual improvement in Phase 1

Financial highlights include:
- Q4 2024 R&D expenses: $8.3M (vs $7.8M in Q4 2023)
- Q4 2024 net loss: $0.05 per share (vs $0.04 in Q4 2023)
- Cash position: $58.8M as of December 31, 2024
- Secured $65M in equity/debt financing in H2 2024
- Runway extended into Q1 2026

Ocugen (NASDAQ: OCGN) has received positive ATMP classification from EMA's Committee for Advanced Therapies for two gene therapy candidates: OCU410 for Geographic Atrophy (GA) and OCU410ST for Stargardt disease.

Key highlights:

• OCU410 Phase 2 dosing is complete, with Phase 3 trials planned for next year, targeting MAA/BLA filings in 2028
• FDA endorsed OCU410ST Phase 2/3 pivotal trial, with potential submissions in 2027
• Preliminary 9-month OCU410 data showed 44% slower lesion growth and 2-line improvement in visual function
• OCU410ST 6-month data demonstrated 52% slower lesion growth and statistically significant visual improvement

Both therapies utilize AAV platform delivering RORA gene through a single subretinal injection. GA affects 2-3 million people in US/Europe, while Stargardt disease impacts 100,000 people, with no approved treatments in Europe.

Ocugen (NASDAQ: OCGN) has received FDA alignment to proceed with a Phase 2/3 pivotal confirmatory clinical trial for OCU410ST, their modifier gene therapy candidate for Stargardt disease. The GARDian trial demonstrated promising results, including:

- 52% slower lesion growth in treated eyes vs untreated eyes at 6-month follow-up
- Statistically significant 2-line improvement in visual function
- Favorable safety profile with no serious adverse events

The upcoming Phase 2/3 trial will involve 51 subjects, with 34 receiving a single subretinal injection of OCU410ST and 17 serving as untreated controls. The company aims for potential BLA filing by 2027. OCU410ST received orphan drug designations from both FDA and EMA in 2023 and 2024. Stargardt disease affects 44,000 patients in the U.S. with no current treatment options.

Ocugen (NASDAQ: OCGN), a biotechnology company specializing in gene and cell therapies, biologics, and vaccines, has scheduled a conference call and live webcast for Wednesday, March 5, 2025, at 8:30 a.m. ET. The event will cover the company's fourth quarter and full year 2024 financial results along with business updates.

The company will release its earnings announcement before the market opens on the same day. Investors can join via phone using the toll-free number (800) 715-9871 for U.S. callers or (646) 307-1963 for international participants, with Conference ID 5045393. A webcast will be available on Ocugen's investor site, with a replay accessible for approximately 45 days after the event.

Ocugen (NASDAQ: OCGN) has completed dosing in the Phase 2 portion of its Phase 1/2 ArMaDa clinical trial for OCU410, a novel gene therapy for geographic atrophy (GA), an advanced form of dry age-related macular degeneration (dAMD). The trial enrolled 51 subjects randomized into treatment and control arms.

The Phase 1/2 study (N=60) showed promising results with a 44% slower lesion growth in treated eyes versus untreated eyes at 9 months, and a clinically meaningful 2-line improvement in visual function. The therapy demonstrated a favorable safety profile with no serious adverse events.

OCU410 is designed as a one-time treatment, targeting multiple disease pathways, unlike current U.S. treatments that require monthly or bi-monthly injections. The trial is being conducted at 14 leading retinal surgery centers across the U.S., with Phase 3 studies planned for 2026 and potential BLA/MAA filings by 2028.

Ocugen (NASDAQ: OCGN) has received a positive opinion from the European Medicines Agency's Committee for Advanced Therapies (CAT) for OCU400's Advanced Therapy Medicinal Product (ATMP) classification. OCU400, the first gene therapy to enter Phase 3 with a broad retinitis pigmentosa (RP) indication, is currently in its Phase 3 liMeliGhT clinical trial.

The trial involves 150 participants divided into two arms: 75 with RHO gene mutations and 75 gene agnostic, with 2:1 randomization to treatment versus control groups. The ATMP classification accelerates regulatory review and enables more frequent EMA interactions as Ocugen plans for Marketing Authorization Application filing in 2026.

Both FDA and EMA have confirmed that a single pivotal Phase 3 trial will suffice for regulatory submissions. Ocugen aims to file simultaneously in the U.S. and Europe, targeting market entry by 2027. RP affects approximately 310,000 patients in the U.S., EU, and Canada, with no current treatments available to slow or stop progression for multiple forms of the disease.

Ocugen (NASDAQ: OCGN) announced that the FDA has cleared their Investigational New Drug (IND) application for OCU500, a first-in-class inhaled COVID-19 vaccine. The Phase 1 clinical trial, scheduled to begin in Q2 2025, will be sponsored by NIAID under Project NextGen to evaluate safety, tolerability, and immunogenicity.

The trial will enroll 80 adults aged 18-64, split into low and high-dose groups, testing both inhalation and intranasal spray delivery methods. The vaccine uses novel chimpanzee adenovirus-vectored (ChAd36) technology licensed from Washington University. CDC data shows COVID-19 remains significant, with 4.4-7.9 million infections and 14,000-25,000 deaths from October 2024 to January 2025.

NIAID will fully fund the Phase 1 trial through Project NextGen's $5 billion initiative. Ocugen plans to expand this mucosal platform to address other respiratory threats including seasonal influenza, bird flu, and RSV.

Ocugen (NASDAQ: OCGN) has initiated the Phase 1 clinical trial of OCU200 for diabetic macular edema (DME) with the first patient dosed. The trial is a multicenter, open-label, dose-escalation study evaluating three dosage levels: 0.025 mg, 0.05 mg, and 0.1 mg, with patients receiving two intravitreal injections six weeks apart.

OCU200 is a novel recombinant fusion protein targeting integrin receptors, designed to treat DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD). The treatment aims to address an unmet need, particularly for the 30-40% of patients who don't respond to current anti-VEGF therapies. These conditions affect approximately 12 million people in the US and 130 million worldwide.

The company plans to pursue OCU200's approval as a first-line therapy for all three conditions, with patient follow-up extending to three months post-treatment.