MALVERN, Pa., April 02, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today provided a general business update with certain financials for the year ending 2023.
“In 2023, our diligent efforts laid the foundation for continued advancement towards our clinical and operational goals with a focus on our game-changing modifier gene therapy platform,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “2024 is already proving to be a pivotal year with considerable developments in our modifier gene therapy programs for blindness diseases, and an increased understanding of the value of these assets among our stakeholders. Promising initial OCU400 Phase 1/2 study results for RP patients have been recognized by the FDA through the RMAT designation, potentially accelerating our path to deliver this critical therapy. With the FDA’s support, we feel confident that this trial will continue to demonstrate the benefits of our gene-agnostic mechanism utilizing a potential one-time treatment for life via a single sub-retinal injection.”
Additionally, the Company’s OCU410 and OCU410ST modifier gene therapy programs are currently enrolling patients with GA secondary to dry AMD (dAMD) and Stargardt disease, respectively. In February 2024, Ocugen completed dosing patients in the first cohort of its Phase 1/2 OCU410ST trial for Stargardt disease, and in March, dosing was completed for the first cohort of the OCU410 trial for GA.
The current treatment landscape for both GA and Stargardt disease is extremely limited. The estimated 1 million patient GA market in the U.S. saw some momentum with recent drug approvals. However, these treatment options have significant limitations, as they require multiple injections per year (impacting compliance) and only target one pathway contributing to GA. OCU410 -regulates multiple pathways involved with the disease including: lipid metabolism, regulation of inflammation, oxidative stress, and membrane attack complex (complement); and has the potential to provide a one-time treatment for life. Presently, there is no approved treatment for people living with Stargardt disease – an orphan blindness disease that affects approximately 40,000 people in the U.S. alone.
“As we pursue our pioneering efforts to change the paradigm for gene therapy, our dedication is unwavering to patients living with a constant fear of losing their sight,” said Dr. Musunuri. “With millions of people affected by these conditions, our mission is clear: to deliver treatments that cannot only stop disease progression but potentially help to preserve or improve sight and allow patients to maintain independence.”
Ocugen’s team has strategically allocated resources to drive the ongoing progress of its gene therapy trials and continues to pursue government funding to support its vaccines programs. During the fourth quarter of 2023, the Company announced its mucosal vaccine candidate, OCU500, was chosen for the multi-billion-dollar NIAID Project NextGen initiative. As a result, OCU500 is slated to enter clinical trials mid-2024. In the planned Phase 1 clinical trial, OCU500 will be tested via two different mucosal routes: inhalation into the lungs and as a nasal spray. All administration of the clinical trial is being led by NIAID.
NeoCart®, the Company’s 3-D regenerative cell therapy platform for cartilage repair, remains on track to begin a Phase 3 trial by the latter half of 2024 subject to availability of funding. Ocugen completed renovations on a world-class cGMP facility last year to produce NeoCart®, which has since received its full final clearance and occupancy certificate. Simultaneously, the Company is evaluating opportunities for NeoCart to maximize value for shareholders and patients.
Modifier Gene Therapies
- OCU400 – Received alignment with FDA for broad RP indication in the Phase 3 clinical trial of OCU400—the first gene therapy program to receive a broad indication for RP. The modified Phase 3 trial design will include 150 adult and pediatric RP patients with RHO and other gene mutations associated with RP. In December, the FDA granted RMAT designation to OCU400 for the treatment of RP. RP affects more than 100,000 people in the U.S. and 1.6 million globally.
- OCU410 – Currently in Phase 1/2 stage of clinical development with active patient enrollment. The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410 for GA secondary to dAMD in December 2023. Dosing is complete for Cohort 1 (low dose).
- OCU410ST – Currently in Phase 1/2 stage of clinical development with active patient enrollment. The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410ST for Stargardt disease in November 2023. Dosing is complete for Cohort 1 (low dose). The Data and Safety Monitoring Board for the OCU410ST clinical trial determined that the safety and tolerability profile for OCU410ST is favorable and approved to proceed dosing with the medium dose of OCU410ST in the dose-escalation phase of the study.
Financial Results
- The Company intends to restate its consolidated financial statements as of and for the year ended December 31, 2022, in connection with the filing of its 2023 Form 10-K. Similarly, the Company will include restated unaudited financial information for each of the first three quarters of 2023 and 2022 in its 2023 Form 10-K (each such annual and quarterly period to be restated, a “Restated Period”).
- The identified errors in each of the Restated Periods relate to the Company’s non-cash accounting for the estimated costs in one of its collaboration arrangements. However, the Company does not expect the errors to result in any impact on its cash position, cash runway, or financial projections.
- Ocugen’s cash, cash equivalents, and investments totaled $39.5 million as of December 31, 2023, compared to $90.9 million as of December 31, 2022. The Company estimates that its current cash, cash equivalents, and investments will enable it to fund its operations into the fourth quarter of 2024. The Company had 256.6 million shares of common stock outstanding as of December 31, 2023.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
