Nature of Business

9 Months Ended

Sep. 30, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Nature of Business

Nature of Business

Ocugen, Inc., together with its wholly owned subsidiaries ("Ocugen" or the "Company"), is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. The Company is headquartered in Malvern, Pennsylvania, and manages its business as one operating segment.

Vaccines

Intramuscular COVID-19 Vaccine Candidate

In February 2021, the Company entered into a Co-Development, Supply and Commercialization Agreement with Bharat Biotech International Limited ("Bharat Biotech"), pursuant to which the Company obtained an exclusive right and license under certain of Bharat Biotech's intellectual property rights, with the right to grant sublicenses to develop, manufacture, and commercialize BBV152, known as COVAXIN, for the prevention of COVID-19 caused by SARS-CoV-2 in the United States, its territories, and possessions. In June 2021 and April 2022, the Company entered into amendments to the Co-Development, Supply and Commercialization Agreement (as so amended, the "Covaxin Agreement"), pursuant to which the parties agreed to expand the Company's rights to develop, manufacture, and commercialize COVAXIN to include Canada and Mexico, respectively, in addition to the United States, its territories, and possessions (the "Ocugen Covaxin Territory"). COVAXIN is a whole-virion inactivated COVID-19 vaccine candidate and is formulated with the inactivated SARS-CoV-2 virus, an antigen, and an adjuvant.

The Company has completed enrollment in a Phase 2/3 immuno-bridging and broadening clinical trial evaluating COVAXIN for adults ages 18 years and older, pursuant to an Investigational New Drug ("IND") application cleared by the U.S. Food and Drug Administration ("FDA"). The clinical trial was designed to evaluate whether the immune response observed in participants in a completed Phase 3 clinical trial in India is similar to a demographically representative, adult population in the United States. The Company intends to work with government agencies in the United States to obtain funding in order to comply with the requirements of a Biologics License Application ("BLA") submission.

Mucosal COVID-19 Vaccine Candidate

In September 2022, the Company entered into an exclusive license agreement ("WU License Agreement") with The Washington University ("Washington University"), pursuant to which the Company obtained the rights to develop, manufacture, and commercialize a mucosal COVID-19 vaccine, OCU500, in the United States, Europe, and Japan (collectively, the "Mucosal COVID-19 Vaccine Territory"). OCU500 is a recombinant, replication-deficient, adenovirus-vectored vaccine with a prefusion stabilized spike protein. As this vaccine can potentially be delivered through the mucosal route (intranasal or inhalation), the Company believes that OCU500 has the potential to generate rapid local immunity in the nose, mouth, upper airways, and lungs where SARS-CoV-2 enters and infects the body, which the Company believes may help reduce or prevent infection and transmission as well as provide protection against new COVID-19 variants. The Company intends to work closely with government agencies in the Mucosal COVID-19 Vaccine Territory to obtain funding to initiate clinical trials and manufacture OCU500.

Modifier Gene Therapy Platform

The Company is developing a modifier gene therapy platform designed to fulfill unmet medical needs in retinal diseases, including inherited retinal diseases ("IRDs"), such as retinitis pigmentosa ("RP"), Leber congenital amaurosis ("LCA"), dry age-related macular degeneration ("AMD"), and Stargardt disease. The Company's modifier gene therapy platform is based on nuclear hormone receptors ("NHRs"), which have the potential to restore homeostasis, the basic biological processes in the retina. The Company believes that its modifier gene therapy platform, through its use of NHRs, represents a novel approach that has the potential to address multiple retinal diseases caused by mutations in multiple genes with one product, and potentially address complex diseases that are potentially caused by imbalances in multiple gene networks.

The Company believes that OCU400, its first product candidate being developed with its modifier gene therapy platform, has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse IRDs,

including RP and LCA. OCU400 has received Orphan Drug Designations ("ODDs") from the FDA for the treatment of certain disease genotypes: nuclear receptor subfamily 2 group E member 3 ("NR2E3"), centrosomal protein 290 ("CEP290"), rhodopsin ("RHO"), and phosphodiesterase 6B ("PDE6ß") mutation-associated inherited retinal degenerations. Additionally, OCU400 has received Orphan Medicinal Product Designation ("OMPD") from the European Commission ("EC") based on the recommendation of the European Medicines Agency ("EMA") for RP and LCA.

The Company has initiated a Phase 1/2 clinical trial, a multicenter, open-label, dose ranging study to assess the safety of unilateral subretinal administration of OCU400 in subjects with NR2E3 and RHO-related RP in the United States, pursuant to an IND application cleared by the FDA. The first patient was dosed in March 2022 and the Company has completed dosing patients in two out of three cohorts. The Company initiated dosing in the third cohort in October 2022. The Company expects to complete enrollment in the third cohort by the end of 2022. Patients with LCA associated with CEP290 mutations will be included in the current Phase 1/2 clinical trial.

The Company is also developing OCU410 and OCU410ST to utilize the nuclear receptor genes RAR-related orphan receptor A ("RORA") for the treatment of dry AMD and Stargardt disease, respectively. The Company is currently executing IND-enabling studies for OCU410 consistent with FDA discussions. The Company intends to submit IND applications in the second quarter of 2023 to initiate Phase 1/2 clinical trials. The Company has engaged CanSino Biologics, Inc. ("CanSinoBIO") as its manufacturing partner for its modifier gene therapy platform. CanSinoBIO has produced the clinical trial supplies for use in the Phase 1/2 clinical trials.

Novel Biologic Therapy for Retinal Diseases

The Company's biologic product candidate, OCU200, is a novel fusion protein designed to treat severely sight-threatening diseases, such as diabetic macular edema ("DME"), diabetic retinopathy ("DR"), and wet AMD. The Company has completed the technology transfer of manufacturing processes to its contract development and manufacturing organization ("CDMO") and has produced clinical trial supplies to initiate a Phase 1 clinical trial. The Company is currently executing IND-enabling studies consistent with FDA discussions. The Company intends to submit an IND application in the first quarter of 2023 to initiate a Phase 1 clinical trial targeting DME.

Regenerative Medicine – Cell Therapy Platform

NeoCart is a three-dimensional tissue-engineered disc of new cartilage that is manufactured by growing chondrocytes, the cells responsible for maintaining cartilage health. The chondrocytes are derived from the patient on a unique scaffold. The Company believes NeoCart has the potential to accelerate healing and reduce pain by reconstructing a patient's previously damaged knee cartilage. It treats pain at the source, creating a similar, functional joint surface as it was before the injury. Ultimately, the therapy's goal is to prevent a patient's progression toward osteoarthritis, reduce pain, and improve function of the joint. In May 2022, the FDA granted a Regenerative Medicine Advanced Therapy ("RMAT") designation to NeoCart for the repair of knee cartilage injuries in adults. The Company is currently working with the FDA to finalize the Phase 3 clinical trial protocol necessary to advance the clinical development of NeoCart for eventual market authorization.

Going Concern Consideration

The condensed consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States ("GAAP") assuming the Company will continue as a going concern. As of September 30, 2022, the Company had cash and cash equivalents of approximately $101.6 million, which the Company believes will enable it to fund its operations into the fourth quarter of 2023. The cash runway is based on management's updated projections, including the removal of external expenditures for COVAXIN beyond the ongoing Phase 2/3 immuno-bridging and broadening clinical trial. The Company anticipates that the continued development of its COVID-19 vaccine candidates will require government funding to support the regulatory pathway of such candidates. There can be no assurance, however, that the Company will be successful in obtaining such funding in sufficient amounts, on terms acceptable to the Company, or at all. As the result of the updated projections, the Company believes that its cash and cash equivalents will enable the funding of its operating expenses and capital expenditure requirements through at least one year from the date the condensed consolidated financial statements are issued.