Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Merck advances once-monthly HIV PrEP pill to Phase 3 trials
Merck has advanced its once-monthly oral HIV prevention pill, MK-8527, into Phase 3 trials, positioning it as a potential alternative to injectable pre-exposure prophylaxis (PrEP) options like Gilead Sciences’ recently approved twice-yearly drug, Yeztugo. MK-8527 is a novel nucleoside reverse transcriptase translocation inhibitor (NRTTI) that blocks translocation and acts as a chain terminator during the RNA-to-DNA conversion of HIV. Following promising Phase 2 results, the drug will now be evaluated in two global studies: EXPrESSIVE-11, enrolling over 4,000 participants across 16 countries, and EXPrESSIVE-10, a Gates Foundation-backed trial focused on 4,580 women in Kenya, South Africa, and Uganda. “With only 18 percent of global PrEP need currently met, there is a clear and urgent need for options like MK-8527 that may offer the ability to prevent infection,” Trevor Mundel, President of Global Health at the Gates Foundation, said in a Merck news release. “These Phase 3 trials are a key step toward translating progress into longer-acting options that could help turn the tide on HIV.” – Bree Foster
Takeda moves closer to approval for novel type of narcolepsy drug
On Monday, Takeda announced positive results from two Phase 3 studies assessing oveporexton to treat narcolepsy type 1. This kind of narcolepsy is caused by the loss of neurons that produce orexin, which helps regulate the sleep-wake cycle. If approved, oveporexton could become the first oral drug to target orexin receptor 2, with other companies like Centessa Pharmaceuticals working on similar compounds in earlier stages. Takeda did not provide any data in the news release, but instead stated that all primary and secondary endpoints were met, which included measuring improvements in excessive daytime sleepiness, cataplexy, and more. They also noted that the drug was generally well-tolerated from a safety perspective, and that they have plans to present the results at upcoming conferences, and will submit a New Drug Application this year. Andy Plump, President of R&D at Takeda, said in the news release, “The studies were accelerated at an unprecedented pace with the aim to bring this potential treatment to people living with narcolepsy type 1 as quickly as possible.” – Allison Whitten
FDA lifts the veil on rejections with release of 200 CRLs
The FDA recently dropped a trove of more than 200 complete response letters (CRLs), giving the public and drug developers a long-overdue peek behind the curtain. These letters spell out why certain drug applications were rejected between 2020 and 2024, exposing common pitfalls like safety concerns, manufacturing issues, and calls for new clinical trials. Until now, sponsors could cherry-pick what they disclosed, often leaving investors and the industry in the dark. For drug developers, this transparency means fewer blind spots and more clarity on what it actually takes to get a drug approved. It’s a win for smarter trial design, better regulatory strategy, and potentially faster paths to market. The FDA says this is just the beginning. – Andrea Corona
FDA rejects two gene and cell therapies over efficacy and manufacturing concerns
The FDA issued CRLs for two advanced therapies this week. Capricor Therapeutics’ cell therapy deramiocel, developed for Duchenne muscular dystrophy-associated cardiomyopathy, was rejected due to insufficient efficacy data and unresolved Chemistry, Manufacturing and Control (CMC) management issues. Ultragenyx also received a rejection for its AAV gene therapy UX111, targeting Sanfilippo syndrome type A. While the FDA did not raise concerns with the clinical data, manufacturing and facility-related deficiencies were cited. Both companies plan to address the issues and resubmit. The rejections highlight the FDA’s increasing emphasis on manufacturing readiness and product quality when evaluating advanced therapies. For developers, these rejections serve as a reminder that even strong clinical data may not secure approval if manufacturing standards aren’t met. – Bree Foster
Safety concerns for GSK’s blood cancer drug ahead of FDA decision
FDA staff reviewers have flagged GSK’s antibody-drug conjugate (ADC), Blenrep, for concerns about potentially causing eye damage. The scrutiny highlights the need for tighter safety optimization in ADC design — especially when targeting solid or sensitive tissues. In the briefing document, the reviewers noted, “There is potential for ocular toxicity to have a substantial impact on patient functioning and quality of life. These toxicity concerns, coupled with the high rates of dose modifications raise concerns about whether the dosages evaluated in DREAMM-7 and DREAMM-8 have been adequately optimized.” Blenrep was granted accelerated FDA approval in 2020 to treat relapsed or refractory multiple myeloma, but was pulled off the market two years later and the FDA revoked its biologics license after a confirmatory trial did not meet the endpoint of progression-free survival. The FDA is set to make a decision on the approval of Blenrep in combination with other therapies for multiple myeloma by July 23rd. – Allison Whitten
JCR and Acumen partner to tackle brain delivery barrier in Alzheimer’s disease therapy
JCR Pharmaceuticals and Acumen Pharmaceuticals have announced a collaboration to address a key obstacle in Alzheimer's disease drug development: delivering biologics effectively to the brain. The partnership will combine JCR’s J-Brain Cargo platform, which facilitates blood-brain barrier penetration, with Acumen’s AβO-selective antibodies that target toxic soluble amyloid beta oligomers. The goal is to develop a brain-penetrant biologic capable of slowing cognitive decline in patients with early Alzheimer’s disease. JCR will receive an undisclosed upfront payment and could earn up to $555 million in development and commercial milestones, plus tiered royalties, if Acumen exercises its option to advance up to two candidates. The partnership comes against a backdrop of persistent setbacks in Alzheimer’s disease research, where an estimated 98 percent of clinical trials since 2004 have failed to produce effective treatments. – Bree Foster
Otsuka bets on broader inflammation blockade with $613M deal
Otsuka Pharmaceutical is acquiring Cantargia’s CAN10 program for $33 million upfront and up to $580 million in milestones, gaining global rights to develop a promising early-stage treatment for autoimmune diseases. CAN10 targets a protein called interleukin-1 receptor accessory protein (IL1RAP), which helps drive inflammation in a range of serious conditions. Unlike drugs that block a single pathway, CAN10 is designed to shut down multiple signals at once, an approach that could make it more effective where other treatments fall short. The drug is currently in Phase 1 trials, with early testing focused on diseases like hidradenitis suppurativa and systemic sclerosis. Otsuka also gets a backup antibody and future rights to Cantargia’s next-generation IL1RAP programs. For those advancing new autoimmune therapies, the deal signals growing interest in multi-target therapies that aim to tackle complex diseases more effectively. – Andrea Corona
