Neurogene, Inc. (New York)

A female Rett syndrome patient died Wednesday after receiving Neurogene’s gene therapy in a clinical trial, the company disclosed in a new regulatory filing , after saying earlier this week that she was in critical condition. After being dosed with the treatment on Nov. 5, the patient experienced systemic hyperinflammatory syndrome, a rare and life-threatening immune response that can occur with high doses of adeno-associated virus gene therapy. “The entire Neurogene team is sending our deepest condolences to her family and loved ones,” Neurogene said in a statement forwarded by a spokesperson. “We appreciate the support for our program from the Rett syndrome patient community, the investigators in our trial and the FDA.” Rett syndrome mostly affects girls and causes a loss in functions and the ability to communicate. No other serious adverse events have been reported in the trial, including in the first two participants who received the same dose and five participants who received a lower dose, the company said earlier this week . The first four patients who got the lower dose of the therapy gained motor skills and improved on other measures, Neurogene said last week. The company said it has discontinued the high-dose arm of the clinical trial, but is proceeding with the low-dose cohort. Neurogene is planning a registrational study that could support FDA approval. This article was updated with a statement from Neurogene.

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 '

Neurogene has released details of an adverse event in a clinical trial testing its gene therapy for Rett syndrome, after news of the safety issue first emerged last week. On Monday, the company said in a news release that the patient experienced systemic hyperinflammatory syndrome, a rare and life-threatening immune response that can occur with high doses of adeno-associated virus gene therapy. The female patient is in critical condition, according to the company. “While no words could possibly provide comfort to her family, we ask the Rett syndrome community to join us in sending heartfelt thoughts to her family, friends and the dedicated clinicians who are caring for her,” Neurogene CEO Rachel McMinn said. “The safety of the participants in our clinical trial is and remains our foremost priority.” Rett syndrome mostly affects girls and causes a loss in functions and the ability to communicate. Neurogene disclosed the case last week but didn’t provide details because the news emerged only hours before a clinical trial update. No other serious adverse events have been reported, including in the first two participants who received the same dose and five participants who received a lower dose, the company said. The first four patients who got the lower dose of the therapy gained motor skills and improved on other measures, Neurogene reported last week. The company said it has discontinued the high-dose arm of the clinical trial, but is proceeding with the low-dose cohort. Neurogene is planning a registrational study that could support FDA approval. The safety issue has overshadowed other promising data from the early-stage trial. Neurogene’s stock $NGNE was down about 40% on Monday.