Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
A once-weekly HIV pill just cleared two Phase 3 trials
Gilead Sciences and Merck announced this week that their investigational once-weekly single-tablet HIV regimen, islatravir/lenacapavir, met its primary efficacy endpoint at week 48 in both the ISLEND-1 and ISLEND-2 Phase 3 trials. The combination pairs Merck's islatravir, a next-generation nucleoside analog that blocks HIV replication through multiple mechanisms including reverse transcriptase translocation inhibition, with Gilead's lenacapavir, a first-in-class capsid inhibitor that disrupts HIV at multiple stages of its lifecycle. In ISLEND-1, the once-weekly tablet was non-inferior to Biktarvy, Gilead's current daily standard-of-care regimen, in virologically suppressed patients. In ISLEND-2, it was non-inferior to a broad range of daily antiretroviral regimens. Safety was comparable across arms with no new concerns identified. Both companies plan to file the Phase 3 data with regulatory authorities globally. If approved, islatravir/lenacapavir would become the first long-acting oral HIV treatment taken once weekly, a meaningful shift in a field where daily dosing has long been the norm and where adherence remains one of the most persistent barriers to sustained viral suppression. – Andrea Corona
SonoThera raises $125M in series B for microbubble-based genetic medicines
The vast majority of gene therapies are delivered via viral vectors, which have safety risks that include liver toxicity and immune reactions. SonoThera is trying a different approach based on the invention from its Chief Scientific Officer, Steven Feinstein, who developed ultrasound contrast agents called microbubbles to image the heart. The company is now using these microbubbles to deliver genetic medicines to any organ of choice by using ultrasound to create a path and then subsequently pop the bubbles to open cells for the genetic material to enter. In a Series B funding round, SonoThera just banked $125 million from big players in the space like Otsuka Pharmaceutical, Johnson & Johnson, and Leaps by Bayer. The funding will help SonoThera move forward with its proprietary platform that supports gene editing and gene silencing with DNA and RNA therapies. Their lead programs target Duchenne muscular dystrophy — where their therapy will become the first to deliver the full-length dystrophin gene — and autosomal dominant polycystic kidney disease (ADPKD). “Despite remarkable scientific progress, many diseases remain beyond the reach of today’s genetic medicines. We founded SonoThera to take a fundamentally different approach, with a platform designed to broaden the therapeutic possibilities of the field,” said Kenneth Greenberg, cofounder and CEO at SonoThera in the press release. – Allison Whitten
GSK adds trio of lung cancer assets in $10.6B deal
GSK said on Tuesday that it has agreed to acquire Nuvalent for $10.6 billion, adding three lung cancer programs in a deal the drugmaker says fits its strategy of buying late-stage assets with validated targets and the potential to outperform existing therapies. Zidesamtinib (NVL-520), a highly selective ROS1 inhibitor, and neladalkib (NVL-655), a next-generation ALK inhibitor, are two late-stage, potential best-in-class candidates for treatment of non-small cell lung cancer (NSCLC). Both have received FDA breakthrough therapy and orphan drug designations and are under regulatory review with target decision dates in late 2026. The third asset, NVL-330, is a potential best-in-class HER2 inhibitor currently in Phase 1 trials for HER2-altered NSCLC. GSK said the lead drugs could launch as soon as 2026 if approved and have multi-blockbuster potential, citing pivotal data presented at the IASLC World Conference on Lung Cancer and the American Society of Clinical Oncology annual meeting showing durable responses, improved tolerability, and enhanced activity against brain metastases. The deal, one of GSK’s largest in years, strengthens its lung cancer franchise as large drugmakers seek to replenish pipelines ahead of looming patent expiries. – Bree Foster
Sanofi to discontinue late-stage autoimmune trial
On Wednesday, Sanofi announced the end of their Phase 3 MOBILIZE trial testing riliprubart, a complement C1 inhibitor, in chronic inflammatory demyelinating polyneuropathy — a rare neurological disease in which the immune system attacks the myelin sheath that surrounds neurons — after an independent data monitoring committee determined that the study is unlikely to prove efficacious. The news comes after encouraging Phase 2 results were announced in 2024. Sanofi stated in the news release that they will evaluate whether to continue other ongoing studies of the drug in the VITALIZE Phase 3 trial, which compares riliprubart to intravenous immunoglobulin treatment. The disappointing results add to a string of setbacks for Sanofi’s drugs last year in psoriasis, chronic obstructive pulmonary disease (COPD), and multiple sclerosis. – Allison Whitten
Dual Lassa–rabies vaccine shows early promise in human trial
Researchers at the University of Maryland School of Medicine have reported promising early clinical results for a single vaccine designed to protect against both Lassa fever and rabies, two deadly viral diseases that disproportionately affect parts of Africa. In a first-in-human trial published in Nature Medicine, 54 adults received two doses of the investigational vaccine, known as LASSARAB, or a licensed rabies vaccine, with interim data showing no serious adverse events and robust antibody responses against both Lassa virus and rabies. The study comes as Lassa fever — which has no licensed vaccine and is classified as a priority pathogen by the World Health Organization — continues to cause thousands of deaths each year in West Africa, while rabies remains almost universally fatal once symptoms appear. The candidate can also be freeze-dried, simplifying immunization efforts in regions with limited healthcare infrastructure. Further follow-up and larger trials are planned to assess the durability of protection and advance the vaccine toward broader clinical testing. – Bree Foster
Novo Nordisk discloses cyberattack involving clinical trial patient data
Novo Nordisk disclosed a cybersecurity incident this week in which unauthorized parties gained access to a limited number of internal IT systems and copied certain non-public data externally, including pseudonymized information tied to patients participating in some of its clinical trials. The exposed data categories may include patient ID numbers, sex, year of birth, biomarkers, health, immunogenicity data, and lifestyle factors such as body mass index (BMI) and smoking status. The company said the data is not directly linked to patients by name or other direct identifiers, and that identifying any individual from the exposed information would require access to additional data that was not part of the breach. Novo Nordisk has taken certain internal IT systems temporarily offline, launched an investigation with external cybersecurity experts, and is in contact with relevant authorities. Core business operations remain unaffected. The incident raises a pointed question for the industry: As clinical trial datasets grow richer and more interconnected, how well are the systems housing them protected? – Andrea Corona
