The FDA faced a packed week of safety reviews and regulatory shakeups.<\/p>\n
Credit: iStock.com\/Andrzej Rostek<\/p>\n
Another death linked to Sarepta\u2019s gene therapy, a promising glioblastoma trial, a rejection for Replimune, and more led the news this week.<\/p>\n
Welcome to the Weekly Rundown where the DDN\u00a0 editors cover this week\u2019s top biotech and pharma news.<\/p>\n
FDA investigates Sarepta after third death linked to gene therapy trial<\/p>\n
The FDA is intensifying its scrutiny of\u00a0Sarepta Therapeutics<\/a> following a third patient death linked to the company\u2019s gene therapy programs. Just hours after Sarepta reported the death of a 51-year-old man with limb-girdle muscular dystrophy (LGMD) from acute liver failure (ALF) following SRP-9004<\/a> treatment, the agency ordered an immediate halt to all Elevidys<\/a> shipments. While the therapies are different, both use the same viral vector, AAVrh74.<\/p>\n Earlier this year, two non-ambulatory teenagers also died from ALF after receiving Elevidys for Duchenne muscular dystrophy (DMD). In response, Sarepta voluntarily paused shipments of the therapy for non-ambulatory patients in June while it worked to develop a more robust immunosuppressive protocol. The company initially declined to extend this pause to ambulatory patients but has since reversed course and now says it will suspend all Elevidys shipments. Roche<\/a>, which markets Elevidys outside the US, has also temporarily paused new orders in countries that reference the FDA for regulatory approval.<\/p>\n Alongside the shipping halt, the FDA paused all LGMD trials, requested a boxed warning for Elevidys, and rescinded AAVrh74\u2019s platform designation. While analysts agreed<\/a> the pause was likely the best decision given the circumstances, it\u2019s unclear how the FDA\u2019s investigation will play out or what the future of Sarepta will be. That uncertainty is felt most acutely by the Duchenne patient community, which is now left waiting to learn if, and when, Elevidys will become available again.\u00a0\u2013 Bree Foster\u00a0<\/p>\n New biotech launches with $216M to develop universal treatment for solid cancers<\/p>\n On Wednesday, new biotech company Dispatch Bio<\/a> announced its official launch with a lofty goal: to develop a universal treatment for solid tumors. Backed by $216 million in funding, the company was founded in 2022 as a collaboration between Arch Venture Partners<\/a> and the Parker Institute for Cancer Immunotherapy<\/a> (PICI). In a press release<\/a>, Dispatch Bio explained that their novel immunotherapy will deliver a viral vector with a new universal antigen that serves to both break down the tumor\u2019s immunosuppressive microenvironment and tag solid tumor cells for immune cells to destroy. In the press release, Founder and Chairman of PICI and Dispatch Bio Board of Director Sean Parker<\/a> said,\u201cWe can now pursue the ultimate goal \u2013 a universal cure for most solid tumor cancers \u2013 using cutting-edge modalities.\u201d \u2013 Allison Whitten<\/p>\n J&J seeks FDA nod for first oral IL-23 psoriasis drug<\/p>\n Johnson & Johnson (J&J) has filed<\/a> for FDA approval of icotrokinra, a once-daily oral IL-23 receptor antagonist for moderate-to-severe plaque psoriasis. Backed<\/a> by four pivotal Phase 3 trials, the peptide therapy met all primary and key secondary endpoints, outperforming the oral TYK2 inhibitor deucravacitinib and demonstrating efficacy in hard-to-treat areas. As the first oral drug in its class, icotrokinra combines the precision of biologics with the convenience of a pill, and could shift systemic treatment away from injectables. J&J has also launched the first head-to-head trial comparing an oral psoriasis therapy to an injectable biologic, positioning icotrokinra as a potential first-line option across multiple inflammatory diseases. \u2013 Andrea Corona<\/p>\n New NHS trial offers hope to patients with aggressive brain cancer<\/p>\n Glioblastoma is one of the most common, complex, treatment-resistant and deadliest forms of brain cancer. The current standard of care typically extends life by only a few months and often comes at the cost of reduced quality of life. Now, a new clinical trial launched at the\u00a0NIHR UCLH Clinical Research Facility<\/a> is offering a novel approach that delivers immunotherapy before standard treatment. Led by\u00a0Paul Mulholland<\/a> and supported by\u00a0UCL<\/a>, the Win-Glio trial will recruit 16 newly diagnosed patients over 18 months to receive ipilimumab prior to surgery, radiotherapy, and chemotherapy \u2014 targeting the immune system when it is at its strongest. The trial was made possible through a \u00a31 million fundraising campaign led by\u00a0Dame Siobhain McDonagh MP<\/a> in memory of her sister,\u00a0Baroness Margaret McDonagh<\/a>, who died from glioblastoma in 2023. The new trial builds on insights from a\u00a0previous study<\/a> in which a single patient, Ben Trotman, received the same treatment and has remained tumour-free more than two and a half years later. This is a remarkable milestone, given the average glioblastoma prognosis of just 12-18 months. \u00a0\u2013 Bree Foster\u00a0<\/p>\n FDA rejects Replimune\u2019s melanoma treatment<\/p>\n On Tuesday, the FDA rejected Replimune<\/a>\u2019s oncolytic virus therapy, RP1, as a treatment for advanced melanoma. In a press release<\/a>, the company said, \u201cThe FDA has indicated that the IGNYTE trial is not considered to be an adequate and well-controlled clinical investigation that provides substantial evidence of effectiveness. Furthermore, the FDA said the trial cannot be adequately interpreted due to the heterogeneity of the patient population.\u201d Following the announcement, Replimune\u2019s shares hit a record low, sinking by 77.2 percent. In the release, the company also stated their surprise at the rejection, given that these concerns were not brought up during previous reviews. Others were surprised too, including Wedbush advisor Robert Driscoll<\/a>, who told Investor\u2019s Business Daily<\/a> that the new director of the Center for Biologics Evaluation and Research, Vinay Prasad<\/a>, was likely involved in the rejection since he has authored articles that criticize the FDA\u2019s accelerated approval pathway. The decision suggests the FDA may take a tougher stance on cell and gene therapies moving forward. \u2013 Allison Whitten<\/p>\n Modulating vitamin D pathways may open new front in cancer therapy<\/p>\n