Ocugen(OCGN) - 2024 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Research and development expenses for Q4 2024 were $8.3 million, up from$7.8 million in Q4 2023. For the full year, R&D expenses decreased to $32.1 million from$39.6 million in 2023 [34] - General and administrative expenses for Q4 2024 were $6.3 million, compared to$5.2 million in Q4 2023. For the full year, G&A expenses decreased to $26.7 million from$32.0 million in 2023 [34] - Net loss for Q4 2024 was approximately $13.9 million or$0.05 per share, compared to a net loss of approximately $11 million or$0.04 per share in Q4 2023. Full year net loss was $54.1 million or$0.20 per share, compared to a net loss of $63.1 million in 2023 or$0.26 per share [35] - Cash and restricted cash totaled $58.8 million as of December 31, 2024, compared to$39.5 million at the end of 2023, providing a cash runway into Q1 2026 [36] Business Line Data and Key Metrics Changes - OCU400, targeting retinitis pigmentosa, has shown a statistically significant improvement in low-luminance visual acuity (LLVA) with a p-value of 0.005% at two years, demonstrating long-term durability [14][15] - OCU410ST received orphan medicinal product designation from the EMA and demonstrated a 100% stabilization or improvement in visual function in treated eyes, with a p-value of 0.02% [17][18] - OCU410, targeting geographic atrophy, showed a 44% slower lesion growth at nine months compared to untreated eyes, with no serious adverse events reported [24][26] Market Data and Key Metrics Changes - Approximately 300,000 people in the US and EU are affected by retinitis pigmentosa, with OCU400 addressing a significant unmet medical need [13] - There are no approved treatments for Stargardt disease, affecting about 100,000 patients in the US and Europe, highlighting the potential market for OCU410ST [17] - Geographic atrophy affects approximately 2 to 3 million patients in the US and EU, with 8 million patients globally, indicating a large market opportunity for OCU410 [23] Company Strategy and Development Direction - The company aims to file three Biologics License Applications (BLAs) in the next three years: OCU400 in 2026, OCU410ST in 2027, and OCU410 in 2028, reflecting a bold ambition supported by FDA alignment [11] - The company is focused on advancing its gene therapy platform and has secured $65 million in financing to support its clinical programs [12] - The strategy includes leveraging the ATMP classification for OCU400 and OCU410ST to expedite regulatory review and enhance interactions with the EMA [14][17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving BLA targets due to a strong track record and ongoing clinical progress across all programs [56] - The company is optimistic about the potential of its therapies to address significant unmet medical needs and improve patient outcomes [11][27] - Management highlighted the importance of safety and tolerability profiles in differentiating their products from existing therapies [64] Other Important Information - The company is actively enrolling patients in clinical trials for its therapies, with plans to complete enrollment for OCU400 in the first half of 2025 [16] - The investigational new drug application for OCU500, an inhaled mucosal vaccine for COVID-19, has been cleared by the FDA, with a Phase 1 trial expected to start in Q2 2025 [30][31] Q&A Session Summary Question: Does the runway factor in newly launching studies like OCU410ST Phase 2/3? - Yes, it is already budgeted [42] Question: When can we expect data from the DME program? - Safety and efficacy reports for OCU200 are expected towards the end of the year, focusing on BCVA and dose response [44][47] Question: What gives confidence in achieving the aggressive BLA filing targets? - The track record of starting gene therapy programs and the ongoing progress across all three programs provide confidence [54][56] Question: How will OCU410 compete with existing therapies? - The safety and tolerability profile of OCU410 is a significant advantage, as current therapies have concerning safety profiles [61][64] Question: Will there be grant revenue associated with the OCU500 Phase 1 trial? - NIAID is sponsoring the program, and the company has completed its obligations; funding will not be recorded as revenue [78][81] Question: What is the manufacturing strategy for OCU410ST? - Commercial-scale manufacturing will be used for both Phase 1 and Phase 2/3 studies [85] Question: Will the study include both US and ex-US sites? - The study will primarily be conducted in the US, with Canada as the only ex-US site [87]