Release Date: August 08, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Ocugen Inc (OCGN, Financial) successfully raised $32.6 million, extending their financial runway into the third quarter of 2025.
- The company received FDA approval for an expanded access program for OCU400, a gene therapy candidate for retinitis pigmentosa (RP), marking a significant regulatory milestone.
- OCU400 has received key regulatory designations, including orphan drug designations from both the FDA and the European Medicines Agency, and RMAT designation from the FDA.
- The company is actively progressing with multiple clinical trials, including the OCU400 Phase 3 liMeliGhT trial and the OCU410 Phase 2 ArMaDa trial.
- Ocugen Inc (OCGN) was included in the Russell Index, reflecting recognition of its pipeline value and strategic growth.
Negative Points
- Ocugen Inc (OCGN) reported a decrease in cash, cash equivalents, and restricted cash from $39.5 million at the end of 2023 to $16 million as of June 30, 2024.
- Total operating expenses for the second quarter of 2024 were $16.6 million, indicating high expenditure relative to cash reserves.
- The company is still working to lift the clinical hold on OCU200, which may delay its progress.
- There is a significant unmet need in the market for RP treatments, with OCU400 still in clinical trials and not yet approved.
- The company faces competition from existing treatments that target specific gene mutations, while OCU400 aims to address multiple mutations.
Q & A Highlights
Q: What is the primary target of the OCU400 expanded access program (EAP)?
A: The EAP targets patients who do not meet the inclusion/exclusion criteria of the Phase 3 study. It offers more flexibility for patients aged 18 and older with a certified genetic diagnosis of retinitis pigmentosa (RP) and remaining photoreceptors. The decision to participate is made by the treating physician and the patient. - Huma Qamar, Chief Marketing Officer
Q: What is the expected difference between the treatment and untreated arms in the OCU400 Phase 3 study, and how is the study powered?
A: The study is powered at a 2:1 ratio with 150 patients, where 50 are in the untreated group. It is powered at greater than 95%, assuming a 50% response rate. Responders are defined as those who achieve two levels or higher in the proprietary LDNA mobility test. - Shankar Musunuri, CEO
Q: What can be expected from the OCU410 study update later this year?
A: The update will provide preliminary safety and efficacy results for the OCU410 study, which targets geographic atrophy secondary to dry age-related macular degeneration. - Huma Qamar, Chief Marketing Officer
Q: Can you provide details on upcoming milestones or data presentations for the OCU400 trial, given the 2026 BLA target?
A: Updates will focus on recruitment rates and meeting the BLA timeline. With RMAT and orphan designations, a rolling submission of the BLA and MAA is possible, starting late next year. Clinical recruitment is expected to conclude early next year, with data available a year later, aiming for approvals in late 2026. - Shankar Musunuri, CEO
Q: What is the status of lifting the clinical hold on OCU200, and will the trial launch immediately after?
A: Ocugen is working with the FDA to submit the requested information to lift the clinical hold. A simple Phase 1 study is designed, and the path forward will be defined post-lift. The focus remains on gene therapy, but OCU200 is a promising program. - Shankar Musunuri, CEO
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
