Roche AG’s Genentech unit disclosed outcomes of two studies testing IL-33-targeting astegolimab vs. placebo on top of standard-of-care maintenance therapy in subjects with moderate to very severe chronic obstructive pulmonary disease (COPD). The pivotal phase IIb effort called Aliento met the primary endpoint of statistically significant reduction in the annualized exacerbation rate (AER) at 52 weeks when astegolimab was given every two weeks. The phase III experiment known as Arnasa did not meet the primary endpoint of a statistically significant reduction in COPD patients’ AER at the same time point. Roche plans to discuss the data, to be detailed at a medical conference, with regulators.

Sarepta confronts FDA, won’t stop Elevidys shipments

Sarepta Therapeutics Inc. is declining a U.S. FDA request to voluntarily halt shipping its gene therapy Elevidys (delandistrogene moxeparvovec) in the U.S. On July 18, Sarepta said had it received “an informal request” from the FDA to stop the shipments following a third patient’s death, tied to the gene therapy SRP-9004, which uses the same vector. The fatality was in a phase I study of limb-girdle muscular dystrophy, with the previous two occurring in clinical trials to treat Duchenne muscular dystrophy. “Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population,” the company said.

For big pharma, China is a shopper’s dream

Big pharma is increasingly shopping in China to fill its pipelines as it faces looming patent cliffs on major blockbusters coupled with growing pricing pressures on drugs. China’s out-licensing deals grew to represent 32% of global deals in the first half of 2025, up from 21% in 2024, and only 5% in 2020, Jefferies Hong Kong-based analyst Cui Cui wrote in a July 2025 report on China dealmaking.

New non-opioid approach for pain: Tafalgie reports phase I data

After two decades of research elucidating the basic science, Tafalgie Therapeutics has delivered the first clinical data for its non-opioid analgesic, saying it heralds a new pharmaceutical approach to treating pain. The molecule, TT5, is a peptide derivative of the endogenous TAFA4 protein, which company co-founder Aziz Moqrich discovered acts in the peripheral nervous system to reduce lesion-induced hyperexcitability and to restore the excitation/inhibition balance. The effect is to modulate the intensity of pain signals sent to the brain via the spinal cord.

In ongoing mRNA evolution, SML Biopharm sights new cancer vaccines

SML Biopharm Co. Ltd. is harnessing mRNA technology to develop novel immunotherapy-based cancer vaccines, including two candidates for cervical and head and neck cancers caused by human papillomavirus (HPV) infection. “Preventive and therapeutic mRNA-based vaccines are obviously important … but the ultimate goal of mRNA technology is to create personalized cancer vaccines capable of triggering the immune system,” SML Biopharm founder and CEO Nam Jae-hwan told BioWorld.

Makary chooses new CDER director from industry ranks

Despite Health and Human Services Secretary Robert Kennedy’s oft-repeated vow to rid the FDA of industry influence, FDA Commissioner Marty Makary today announced George Tidmarsh, an industry veteran, will be the next director of the agency’s Center for Drug Evaluation and Research (CDER). A pediatric oncologist and neonatologist with a decades-long pedigree of founding biopharma startups and shepherding new drugs through R&D and the regulatory process, Tidmarsh will be only the third person to hold that title since Janet Woodcock took the reins in 1994. But unlike Woodcock, who made the FDA her career, and Patrizia Cavazzoni, who resigned as CDER director in January, Tidmarsh comes to the position with no prior FDA experience. In announcing the appointment, Makary played up Tidmarsh’s contributions in the private sector, saying he “is an accomplished physician-scientist and leader whose experience spans the full arc of drug development – from bench to bedside.”

IAS 2025: Cheat, parasitize, break the virus – fresh ideas fuel HIV research

HIV research is slowly returning results. Antiretroviral therapy (ART) has represented a paradigm shift for People Living with HIV (PLHIV), allowing for undetectable levels of the virus in the blood. Pre-exposure prophylaxis (PrEP) has significantly reduced the risk of infection. However, in 2024 there were 1.3 million new infections and 630,000 HIV-related deaths. The virus persists dormant in cellular reservoirs, there is still no effective vaccine or cure. Scientists are considering options ranging from longer-term ART that space out injections by several years to long-lasting PrEP that acts as a vaccine while immunization is achieved. What else can be done? Target the virus and kill it. How? In every possible way.

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