Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
New therapy offers hope for type 1 diabetes
Vertex Pharmaceuticals has reported promising results from its cell therapy for type 1 diabetes, with 10 of 12 patients (83 percent) achieving insulin independence one year after receiving a single infusion of stem cell-derived islet cells. Presented at the American Diabetes Association’s annual meeting, the Phase 1/2 FORWARD trial showed that patients not only stopped taking insulin but also maintained healthy blood sugar levels without severe hypoglycemic events. The therapy, zimislecel, restored insulin production as confirmed by detectable connecting peptide (C-peptide) levels in all participants. While patients require immunosuppressive drugs to prevent rejection of the transplanted cells, researchers believe the benefits could justify the risks. The study is now advancing into Phase 3, with a potential regulatory filing expected by 2026. If successful, zimislecel could offer a transformative treatment option for people living with type 1 diabetes. – Bree Foster
Compass’ psilocybin drug hits key goal, but the full impact is still unclear
Compass Pathways reported positive results from the first Phase 3 trial of its psilocybin-based therapy, COMP360, for treatment-resistant depression (TRD), marking the first late-stage success for a classical psychedelic in mental health. Patients receiving a single 25mg dose of COMP360 alongside psychological support showed a statistically significant 3.6-point improvement on the Montgomery-Åsberg Depression Rating Scale (MADRS) compared to placebo at six weeks. While the result exceeds the FDA’s benchmark for approval, Leonid Timashev, an analyst at RBC Capital Markets, told BioPharma Dive that it falls short of what many clinicians view as a more robust effect. Despite the positive trial outcome, Compass shares dropped by nearly 50 percent, highlighting lingering investor concerns over effect size, safety monitoring, and the therapy’s intensive delivery model, which includes an all-day monitored session and multiple psychotherapy visits. Parts 2 and 3 of the trial will assess outcomes through 26 and 52 weeks, respectively, with additional data expected next year. This data will be critical in determining whether COMP360 can secure FDA approval and carve a viable path to commercialization in a still-nascent medicine market. – Bree Foster
First look at RFK Jr.’s new vaccine panel
The newly appointed members of the Advisory Committee on Immunization Practices (ACIP) met this week after Robert F. Kennedy Jr. fired the original 17 members. In their first vote, the committee voted 5-2 to recommend a monoclonal antibody from Merck approved earlier this month to protect infants against respiratory syncytial virus (RSV). The committee is also expected to discuss the use of the preservative thimerosal, an ingredient that RFK Jr. wrote about removing from vaccines in a book he authored, despite the fact that it has already been removed from the majority of vaccines and scientific evidence shows no evidence of harm. – Allison Whitten
Nuvalent drug shrinks tumors in hard-to-treat lung cancer
Nuvalent reported strong results from its Phase 1/2 ARROS-1 trial of zidesamtinib, a selective tyrosine kinase inhibitor (TKI) that targets the ROS1 protein, showing great potential in heavily pretreated ROS proto-oncogene 1, receptor tyrosine kinase (ROS1)-positive non-small cell lung cancer (NSCLC). The therapy achieved a 44 percent overall response rate among 117 patients who had previously received one or more ROS1-targeted therapies. Notably, 93 percent of patients who had received just one prior TKI remained in remission for at least 18 months, while the broader trial population showed a 78 percent response durability at 12 months and 62 percent at 18 months. These results point to zidesamtinib’s potential as a durable and effective treatment option, even in patients who have progressed on next-generation therapies. The drug also demonstrated meaningful intracranial activity and a favorable safety profile, avoiding the neurotoxicities linked to existing dual-targeted TKIs. Nuvalent is now preparing to begin a rolling FDA submission in July under the Real-Time Oncology Review program, paving the way for a new standard of care in ROS1-positive NSCLC. – Bree Foster
FDA approves lung cancer drug and device to treat peripheral nerve injuries
- AstraZeneca and Daiichi Sankyo received FDA approval for their lung cancer drug, datopotamab deruxtecan. The approval is specifically for the drug to treat patients with epidermal growth factor receptor (EGFR)-mutated NSCLC who have received prior treatment, and the decision was based on results from the TROPION-Lung05 Phase 2 trial and TROPION-Lung01 Phase 3 trial.
- TISSIUM received FDA De Novo approval to commercialize their biopolymer device, Coaptium Connect, which provides a sutureless procedure to repair peripheral nerve injuries. TISSIUM reported that their device achieved 100 percent procedural success with patients regaining full flexion and extension at the injury without pain for 12 months afterwards. The company now plans to begin a commercial rollout of the device in the US. – Allison Whitten
Novo Nordisk compares their hemophilia A drug to Genentech’s Hemlibra
Novo Nordisk announced positive safety results from their Phase 3 FRONTIER trial evaluating Mim8 as a prophylactic treatment for people with hemophilia A. The data showed that patients who switched from Genentech’s Hemlibra medication, which has enjoyed significant uptake in the market, to Mim8 with no washout period was well-tolerated without adverse events. The data also showed an increase in the generation of the thrombin protein needed in blood coagulation. Novo Nordisk also reported that patients noted a preference for the Mim8 pen-injector over the Hemlibra injection device. The company plans to submit Mim8 for regulatory review later this year. – Allison Whitten
