Ocugen Updates Business and Q2 2024 Financials

Ocugen, Inc., a biotechnology firm specializing in gene and cell therapies, disclosed its financial results for the second quarter of 2024. The company highlighted significant strides made during the first half of the year, particularly with its modifier gene therapy platform. This includes the ongoing Phase 3 clinical trial of OCU400 for retinitis pigmentosa (RP) and advancements in the Phase 2 trial of OCU410 for geographic atrophy (GA).

Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen, emphasized the importance of these achievements in bringing potential one-time therapies to patients afflicted with RP and GA, who urgently need new treatment options. The efforts of the Ocugen team were acknowledged for keeping the clinical trials on track.

The OCU400 Phase 3 trial involves 150 participants, divided into two groups based on specific gene mutations. The trial's primary endpoint is the Luminance Dependent Navigation Assessment (LDNA), which measures visual function more precisely than previous mobility measurements. The trial aims to determine the proportion of participants who exhibit at least a 2 Lux improvement in the study eyes.

The FDA recently approved an expanded access program (EAP) for OCU400, allowing its use in treating adult RP patients regardless of mutation. This gene therapy candidate is the first to receive such approval for RP. The EAP supports the gene-agnostic mechanism of action of this novel therapy.

The potential of novel modifier gene therapy to address multiple inherited retinal diseases, as well as multifactorial causes of blindness like dry age-related macular degeneration (dAMD), is promising. OCU410 and OCU410ST are designed to treat geographic atrophy secondary to dAMD and Stargardt disease, respectively. These therapies utilize the RORA gene, potentially offering a one-time treatment with a single sub-retinal injection.

OCU410 is designed to target multiple disease pathways, unlike current treatments that focus solely on the complement system and require frequent injections. OCU410 aims to regulate lipid metabolism, inflammation, oxidative stress, and the complement system with a single injection. OCU410ST, on the other hand, has received Orphan Drug Designation from the FDA for treating Stargardt disease, which currently has no approved treatments. The third cohort of the clinical trial is currently being dosed with the high dose, and OCU410ST has the potential to become the first one-time gene therapy for Stargardt disease.

Ocugen is actively seeking strategic partnerships to support the global accessibility of these therapies. At the 2024 BIO International Convention, the company engaged with potential partners to explore opportunities for its dynamic pipeline.

In June, Ocugen's inclusion in the Russell Index was announced, enhancing the value of its pipeline and recognizing the company's growth strategy. This inclusion is expected to increase visibility within the investment community and broaden the shareholder base. Dr. Musunuri expressed optimism for the remainder of 2024 as Ocugen continues to establish itself as a biotechnology leader.

Financially, Ocugen secured $32.6 million from a public offering of common stock, which extends its cash runway into the third quarter of 2025. As of June 30, 2024, the company had cash and equivalents totaling $16 million, down from $39.5 million at the end of 2023. Total operating expenses for the second quarter of 2024 were $16.6 million, compared to $24 million for the same period in 2023. The net loss per share narrowed to $0.04, from $0.10 the previous year.

Ocugen is progressing with several first-in-class therapies, including OCU400, OCU410, and OCU410ST. Additionally, they are planning a Phase 3 trial for NeoCart®, contingent on funding, and discussions continue with government agencies and partners regarding the development of their inhaled mucosal vaccine platform for COVID-19.

The company is also working with the FDA to lift the clinical hold on OCU200, an ophthalmic biologic product.

Ocugen is hosting a conference call and webcast to discuss these results and recent business highlights, where management will address questions from participants.

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