Ocugen(OCGN) - 2023 Q4 - Annual Report

Gene Therapy Development - The company is developing a modifier gene therapy platform targeting retinal diseases, with OCU400 receiving FDA clearance for a Phase 3 trial for retinitis pigmentosa (RP) expected to begin in Q2 2024[434][439]. - In the Phase 1/2 trial for OCU400, 89% of participants showed preservation or improvement in treated eyes, with 78% demonstrating stabilization or improvement in MLMT scores from baseline[438]. - The Phase 3 trial for OCU400 will enroll 150 subjects, with a 1:1 distribution into two arms (RHO: N=75, Gene Agnostic: N=75), and subjects will be followed for a year post-dosing[439]. - OCU410 and OCU410ST are in development for dry age-related macular degeneration (dAMD) and Stargardt disease, respectively, with OCU410 targeting multiple pathways for better safety and efficacy outcomes[442]. - OCU410 and OCU410ST programs are currently in Phase 1/2 clinical development, with the first patient dosed in November 2023 for Stargardt disease[454]. - The first patient for the OCU410 program targeting geographic atrophy was dosed in December 2023, with ongoing dosing in the Phase 1/2 trial[459]. Vaccine Development - The company is also advancing an inhaled mucosal vaccine platform, including OCU500 for COVID-19, with clinical trials expected to begin mid-2024[434]. - The inhaled mucosal vaccine platform includes OCU500, OCU510, and OCU520, with OCU500 selected for clinical trials by NIAID Project NextGen[482]. - The company is collaborating with NIAID for early clinical studies of OCU500 and is planning to submit an IND in 2024[434]. - The company faces challenges in sourcing raw materials for vaccine production, which could impact clinical trials and manufacturing timelines[460]. - The company is actively seeking government funding for the development of its vaccine candidates, which is crucial for commercialization[482]. Regulatory and Financial Challenges - The FDA has provided Orphan Drug Designation (ODD) and Regenerative Medicine Advanced Therapy (RMAT) designation for OCU400, supporting its broad therapeutic potential[436]. - The EMA has reviewed the Phase 3 trial design for OCU400 and provided acceptability for submission of a Marketing Authorization Application (MAA)[440]. - Regulatory challenges may impact the approval process and commercialization of product candidates, potentially affecting the company's financial position[445]. - The FDA has placed the IND application for OCU200 on clinical hold, pending additional information related to CMC[480]. - OCU400 and OCU410ST have received orphan drug designations from the FDA, but there is no guarantee of maintaining these designations[462]. Financial Performance - The company incurred net losses of approximately $63.1 million and $86.8 million for the years ended December 31, 2023 and 2022, respectively, with an accumulated deficit of $286.2 million as of December 31, 2023[483][499]. - Total revenue for the year ended December 31, 2023 was $6.036 million, an increase of $3.548 million compared to $2.488 million for the year ended December 31, 2022[492][493]. - Research and development expenses decreased by $16.586 million to $39.573 million for the year ended December 31, 2023, primarily due to the termination of the COVAXIN program and reduced activities for OCU200 and OCU400[492][494]. - General and administrative expenses decreased by $3.406 million to $31.994 million for the year ended December 31, 2023, mainly due to reductions in consulting and pre-commercial expenses[492][496]. - Cash used in operating activities was $62.1 million for the year ended December 31, 2023, compared to $60.1 million for the year ended December 31, 2022[505][506]. - The company raised an aggregate of $301.0 million since inception, with $287.2 million from the sale of common stock and warrants as of December 31, 2023[503]. - Cash provided by financing activities was $20.9 million for the year ended December 31, 2023, a decrease from $59.5 million for the year ended December 31, 2022[509]. - The company had cash and cash equivalents of $39.5 million as of December 31, 2023[503]. - As of December 31, 2023, the company had cash and cash equivalents of approximately $39.5 million, which is insufficient to meet capital requirements over the next 12 months[518]. - The company anticipates needing to raise significant additional capital to fund operations until it recognizes significant revenue from product sales[518]. - Stock-based compensation expense was $9.2 million for the year ended December 31, 2023, down from $10.5 million in 2022[534]. - The company expects to recognize $8.6 million of unrecognized stock-based compensation expense over a remaining weighted-average period of 1.5 years[534]. - The company has borrowed $2.5 million from EB-5 Life Sciences under the U.S. government's Immigrant Investor Program[512]. - Future minimum operating lease base rent payment obligations amounted to $5.8 million as of December 31, 2023[511]. - There is substantial doubt about the company's ability to continue as a going concern within one year after the issuance of the consolidated financial statements[519]. Operational Developments - The company has completed renovations to a facility to support clinical studies and initial commercial launch of its regenerative medicine cell therapy platform[434]. - The company is developing NeoCart, a regenerative medicine cell therapy, with plans to initiate a Phase 3 trial in the second half of 2024, contingent on funding availability[481]. - The company terminated the Canada Consulting Agreement in June 2023 due to the FDA canceling all EUAs for monovalent COVID-19 vaccine formulations[513]. - The company is exploring various strategies for funding, including public and private placements of equity and/or debt, and potential collaborations with pharmaceutical companies[518]. - The company has no off-balance sheet arrangements during the periods presented[520].