Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA investigates Sarepta after third death linked to gene therapy trial
The FDA is intensifying its scrutiny of Sarepta Therapeutics following a third patient death linked to the company’s 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 treatment, the agency ordered an immediate halt to all Elevidys shipments. While the therapies are different, both use the same viral vector, AAVrh74.
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, which markets Elevidys outside the US, has also temporarily paused new orders in countries that reference the FDA for regulatory approval.
Alongside the shipping halt, the FDA paused all LGMD trials, requested a boxed warning for Elevidys, and rescinded AAVrh74’s platform designation. While analysts agreed the pause was likely the best decision given the circumstances, it’s unclear how the FDA’s 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. – Bree Foster
New biotech launches with $216M to develop universal treatment for solid cancers
On Wednesday, new biotech company Dispatch Bio 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 and the Parker Institute for Cancer Immunotherapy (PICI). In a press release, 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’s 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 said,“We can now pursue the ultimate goal – a universal cure for most solid tumor cancers – using cutting-edge modalities.” – Allison Whitten
J&J seeks FDA nod for first oral IL-23 psoriasis drug
Johnson & Johnson (J&J) has filed for FDA approval of icotrokinra, a once-daily oral IL-23 receptor antagonist for moderate-to-severe plaque psoriasis. Backed 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. – Andrea Corona
New NHS trial offers hope to patients with aggressive brain cancer
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 NIHR UCLH Clinical Research Facility is offering a novel approach that delivers immunotherapy before standard treatment. Led by Paul Mulholland and supported by UCL, the Win-Glio trial will recruit 16 newly diagnosed patients over 18 months to receive ipilimumab prior to surgery, radiotherapy, and chemotherapy — targeting the immune system when it is at its strongest. The trial was made possible through a £1 million fundraising campaign led by Dame Siobhain McDonagh MP in memory of her sister, Baroness Margaret McDonagh, who died from glioblastoma in 2023. The new trial builds on insights from a previous study 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. – Bree Foster
FDA rejects Replimune’s melanoma treatment
On Tuesday, the FDA rejected Replimune’s oncolytic virus therapy, RP1, as a treatment for advanced melanoma. In a press release, the company said, “The 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.” Following the announcement, Replimune’s 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, who told Investor’s Business Daily that the new director of the Center for Biologics Evaluation and Research, Vinay Prasad, was likely involved in the rejection since he has authored articles that criticize the FDA’s accelerated approval pathway. The decision suggests the FDA may take a tougher stance on cell and gene therapies moving forward. – Allison Whitten
Modulating vitamin D pathways may open new front in cancer therapy
Researchers have identified SDR42E1, a protein essential for vitamin D absorption and metabolism in intestinal cells, as a promising new target in cancer therapy. The study published in Frontiers in Endocrinology used CRISPR/Cas9 to disrupt SDR42E1 in colorectal cancer cells, slashing cell viability by over 50 percent, and triggering widespread changes in genes linked to sterol metabolism and tumor growth. The discovery positions SDR42E1 as a molecular switch that connects vitamin D pathways with cancer cell survival, potentially enabling new therapeutic strategies that either block SDR42E1 to starve tumors or boost its expression to enhance vitamin D receptor activity. Beyond oncology, this discovery could have far-reaching implications for immune disorders, metabolic disease, and how we think about vitamin D supplementation. – Bree Foster
