Pegcetacoplan

MALVERN, Pa., Nov. 19, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced positive preliminary efficacy and safety data from the Phase 1 dose-escalation portion of the Phase 1/2 OCU410 ArMaDa clinical trial for geographic atrophy (GA), secondary to dry age-related macular degeneration (dAMD). Key findings include: no drug-related serious adverse events, reduced lesion growth, preservation of retinal tissue, and—most importantly—there was a positive effect on the functional visual measure of low luminance visual acuity (LLVA). Currently, there are approximately three million people living with GA in the United States (U.S.) and Europe combined. Patients in the U.S. have only one option available, anti-complement therapy, which requires multiple injections and only addresses one aspect of the disease. There remains no treatment option for GA in Europe. The OCU410 Phase 1 trial is evaluating nine patients in three dose cohorts (low, medium, and high). The following data was observed for the three patients in the low dose cohort at six months: Considerably slower lesion growth (21.4%) from baseline in treated vs. untreated fellow eyes that followed the natural history of the disease. This result is favorable when compared to published data on pegcetacoplan injected every month or every other month over six months.OCU410 treatment showed increasing preservation of retinal tissue around the GA lesions of treated eyes over six months, which also compared favorably to published data on pegcetacoplan given monthly and every other month.100% of the OCU410 treated eyes showed stabilization of visual function demonstrating treatment benefit as measured by LLVA. “Currently approved treatments for GA have not shown significant benefit in visual function. More importantly, we often do not realize the logistical challenge and emotional burden both patients and their caregivers must endure for every month or every other month visits,” said Syed M. Shah, MD, FACS, Director of Retina Service, Vice Chair for Research & Digital Health at Emplify Health – La Crosse, Wisconsin. “Based on the science and preliminary data, OCU410 has the potential to improve structural as well as functional outcomes. This ‘one-and-done’ treatment paradigm can be a gamechanger for how we treat patients with GA.” “OCU410 addresses multiple aspects of the disease beyond the complement pathway,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “The latest OCU410 data emphasizes the potential of novel modifier gene therapy as a one-time treatment for dAMD. We remain very encouraged by the latest safety and efficacy data and positive patient outcomes.” Ocugen also announced promising data from the Phase 1/2 OCU410ST GARDian clinical trial for Stargardt disease and data on Leber congenital amaurosis (LCA) from the Phase 1/2 OCU400 clinical trial. All these findings, as well as commentary from study investigators and patient perspectives, were shared at the Company’s recent Clinical Showcase. The data affirms the potential for modifier gene therapy to address both rare inherited retinal diseases and blindness diseases affecting millions. A full replay of the showcase is available on the Events section of the Ocugen website. For more information about Ocugen’s ongoing clinical trials, please contact clinical.request@ocugen.com 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 patient’s 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. Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release. Contact:Tiffany HamiltonHead of CommunicationsTiffany.Hamilton@ocugen.com

Today, a brief rundown of news involving Astellas and Cytokinetics, as well as updates from Cybin, Neurogene and Novartis that you may have missed.The Food and Drug Administration rejected a request by Astellas Pharma to update the prescribing information for its geographic atrophy drug Izervay, the company said Tuesday. According to Astellas, the FDA turned back an application that would have enabled patients to receive injections of Izervay every other month, instead of monthly, because of a statistical matter related to labeling language and not the drug's “safety and benefit/risk.“ The decision, for now, maintains a dosing advantage for Apellis Pharmaceuticals rival medicine Syfovre, which can be administered once every 25 to 60 days. Apellis shares climbed about 10%. Ben FidlerFormer National Cancer Institute director Norman Sharpless and serial biotech entrepreneur Nathaniel David have teamed to launch a new startup creator, Jupiter Bioventures, with $70 million in funding. The firm will use small seed funds to acquire intellectual property, vet it through in-house experiments and then build companies around the concepts that show promise. Those companies, which will work on cancer or other unspecified diseases, can then go off and raise cash from Jupiter or other investors, the firm said in a Tuesday statement. The funding from the Mayo Clinic, Mission BioCapital and others will help Jupiter form eight to 10 startups. Ben FidlerBayer will pay Cytokinetics 50 million euros, or about $53 million, to license the biotechnology company's experimental heart drug aficamten in Japan. Under a deal the companies announced Tuesday, Bayer will conduct a Phase 3 trial of aficamten in Japanese people with obstructive hypertrophic cardiomyopathy, while Cytokinetics will expand into Japan an ongoing trial in people with non-obstructive disease. The studies are meant to support potential authorization in Japan, where Bayer will now hold exclusive commercialization rights. Cytokinetics could earn up to 90 million euros more if certain milestones are met through the market launch of aficamten. Ned PagliaruloNeurogene will continue a Phase 1/2 trial of its gene therapy for Rett syndrome, but only at the lowest planned dose, following a side effect that led to the hospitalization of one trial volunteer who received a high dose. The company said its executives had met with FDA officials and was cleared to continue testing the low dose. The changes in the trials design mean Neurogene will be unable to complete enrollment of the low-dose group by the end of 2024, the company said. The trial volunteer experienced systemic hyperinflammatory syndrome, which can be caused by exposure to high levels of the type of adeno-associated virus used to deliver Neurogenes therapy. Company shares fell by about 40% following the Monday announcement and have lost three-quarters of their value since the company first disclosed the adverse event. Jonathan GardnerShares of Cybin also dipped on Monday, after the company, which is developing psychedelic-based medicines, released results from a small, mid-stage clinical trial. The trial has been evaluating two doses of “CBY003,“ a version of a psychedelic compound found in certain mushroom species, to see if they can be helpful add-ons to psychotherapy for people with major depressive disorder. Cybin said that, one year into the study, patients' depression symptoms had substantially diminished. And all of those given the high dose were deemed “responders.“ Yet Cybin's stock price, which was up 15% at market's open but ended Monday down almost 9%, suggests investors still have questions about the program. Jacob BellNovartis has licensed a new radiopharmaceutical drug from startup Ratio Therapeutics, promising up to $745 million in total payments for rights to an experimental medicine targeting a protein called somatostatin receptor 2. Per the agreement, Ratio and Novartis will collaborate to research and select an SSTR2 candidate that Novartis will then take into clinical development. SSTR2 has been identified as a potential target in small cell lung cancer and some neuroendocrine tumors. Ratio was founded in 2022 with $20 million in funding and support from Bayer and Lantheus Holdings. It has since raised an additional $70 million in two funding rounds, receiving support from Bristol Myers Squibb. Jonathan Gardner '