Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA approves GSK’s functional cure for hepatitis B
Tuesday brought good news for many patients with chronic hepatitis B. GSK’s antisense oligonucleotide Hibsago (bepirovirsen) injection received the green light from the FDA, effectively offering a chance for some people with chronic hepatitis B to stop taking their antivirals entirely. The approval was based on two Phase 3 B-WELL trials, which demonstrated that 24 percent of patients who received the Hibsago treatment qualified to stop taking their medications — compared to zero percent taking the placebo. After 24 weeks without antivirals, Hibsago led to a functional cure in 19 percent of patients who no longer had detectable viral proteins in their blood. “Hibsago provides a new treatment approach that gives some patients the possibility of maintaining control of the virus after all chronic HBV treatment has ended. This approval represents an important advance for people living with this serious disease,” said Wendy Carter, Acting Director of the Office of Infectious Diseases in the Center for Drug Evaluation and Research, in the press release. The approval marks the first to reach the finish line in a competitive landscape, with therapeutic options for hepatitis B from companies like Brii Biosciences now lagging behind. – Allison Whitten
New trial aims to speed up the search for Alzheimer’s treatments
A UK clinical trial is testing whether two existing medicines, metformin and atomoxetine, could help treat Alzheimer’s disease, using a platform design intended to speed up the search for effective therapies. The AD-SMART trial will recruit around 1,200 people with mild to moderate Alzheimer’s across more than 30 UK sites to assess metformin, commonly used to treat type 2 diabetes, and atomoxetine, used to treat attention deficit hyperactivity disorder (ADHD). Researchers believe both medicines could influence biological processes involved in Alzheimer's, potentially helping patients remain independent for longer. The multi-arm, multi-stage design allows researchers to test several treatments alongside standard care and a shared placebo group, drop candidates that show insufficient benefit, and introduce new ones without restarting the trial. Over 18 months, researchers will assess whether each drug safely improves cognitive symptoms, daily functioning, or neuropsychiatric symptoms, while blood tests and MRI scans will help track biological changes associated with disease progression. The first treatments are expected to complete evaluation in 2030, with additional candidates potentially added in the meantime. The drugs were selected by an international panel drawing on earlier laboratory and clinical evidence, alongside input from people with lived experience of Alzheimer’s. – Bree Foster
Inhaled exosome therapy shows promise for ARDS in Phase 2b trial
Nano24 announced positive results from their Phase 2b trial testing EXO-CD24, an inhaled therapy that delivers the immune checkpoint CD24 via exosomes, to treat patients with the life-threatening condition acute respiratory distress syndrome (ARDS). “Exosomes offer a promising foundation for drug delivery because their surfaces and internal cargo can be engineered to perform complementary functions,” said Robert Langer, Head of Nano24’s Scientific Advisory Board, in the press release. Based on data from 90 patients, the trial showed that EXO-CD24 led to 85 percent of patients being free of respiratory failure compared with just 33.3 percent of placebo, and a 72 percent reduced risk of death. The treatment also reduced the mean number of days in the hospital from 14.4 days down to 6.5. With current treatment options only focusing on supportive care, Nano24’s therapy could offer a very welcome change for patients and physicians alike. The company said in the statement that they now plan to move forward with an international Phase 3 trial to confirm efficacy and safety in ARDS. “The findings on the proportion of patients free of respiratory failure at Day 7 and on all-cause mortality are encouraging in this deadly condition. I look forward to taking part in the upcoming Phase 3 study,” said Matthew Baldwin, Associate Professor of Medicine at Columbia University, in the press release. – Allison Whitten
Pfizer and Valneva present full Phase 3 data for Lyme vaccine candidate
Pfizer and Valneva presented Phase 3 VALOR results for their six-valent Lyme disease vaccine candidate, PF-07307405 (LB6V), at the IDWeek congress in Washington, DC, with Amsterdam UMC’s Joppe Hovius presenting efficacy and safety data in healthy participants aged five and older living in Lyme disease-endemic regions of North America and Europe. The presentation builds on March’s topline readout, which showed efficacy of roughly 73 to 75 percent but missed the first prespecified statistical criterion because fewer Lyme disease cases accrued than expected. The European Medicines Agency began reviewing the marketing application in August, and Pfizer has said the findings support the vaccine’s potential to provide clinically meaningful antibody-associated protection. If approved, LB6V would be the first Lyme disease vaccine on the market in decades. – Andrea Corona
Jemperli approval opens a new treatment path for some rectal cancer patients
The FDA has granted accelerated approval to GSK’s Jemperli (dostarlimab) as the first and only immunotherapy for adults with previously untreated stage 2 or 3 locally advanced rectal cancer whose tumors are microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR). Jemperli is a PD-1-blocking antibody that is designed to help the immune system recognize and attack cancer cells. The approval, based on the Phase 2 AZUR-1 trial, could allow eligible patients to avoid chemotherapy, radiation, and surgery, which can cause lasting effects on bowel, urinary, and reproductive function. In the trial, 68 percent of evaluable patients achieved a clinical complete response sustained for at least 12 months, while an exploratory analysis found no evidence of cancer one year or more after treatment in 76 percent of patients. No disease recurrences have been reported among responders after a median follow-up of more than two years. The safety profile was consistent with previous experience, although serious adverse reactions occurred in 20 percent of patients and three percent discontinued treatment because of adverse reactions. Longer follow-up will be needed to establish how durable these responses are, but the results suggest that immunotherapy could offer some patients a way to avoid the lasting effects of conventional treatment. – Bree Foster
FDA launches neuroscience program, starting with Alzheimer’s prevention
The FDA launched the Neuroscience Advancement and Innovation Nexus (NAIN), a program that will bring together experts from its drug, biologics, and device centers to coordinate how it regulates products for conditions of the brain and nervous system. The scope covers neurologic and psychiatric disorders, substance use disorders, and pain. NAIN’s first initiative, the Alzheimer’s Prevention Accelerator, will explore how to run clinical trials aimed at preventing or delaying Alzheimer’s disease, with a focus on trial populations, stage-appropriate endpoints, and the kind of evidence needed to show that cognitive decline was delayed or prevented. The agency said the difficulty of demonstrating clinical benefit in conditions that develop over many years is a core challenge, and pointed to biomarkers and digital health tools as opportunities to address it. Acting FDA Commissioner Kyle Diamantas said the program will help regulatory science keep pace with advances in neuroscience, and the launch is tied to the federal government’s national Alzheimer’s plan. For developers, the accelerator signals that the agency may be open to new endpoint and evidence standards for prevention trials, though no specific policy changes have been announced. – Andrea Corona
