Clinical Research Focus. 43rd Edition

Sarepta Reverses Course on Elevidys Suspension After FDA Review

Earlier this month, Sarepta paused all U.S. shipments of its gene therapy Elevidys following three patient deaths, triggering a rare standoff with the FDA. However, in a swift regulatory turnaround, the agency has now cleared the therapy for continued use in ambulatory DMD patients, citing no link between the drug and a reported fatality.

Learn more

EMA Recommends First Long-Acting Injection for HIV Prevention

The EMA has recommended marketing authorization for Apretude (cabotegravir), the first long-acting injectable for HIV pre-exposure prophylaxis (PrEP) in the EU. Administered every two months, Apretude offers a more convenient alternative to daily oral PrEP—marking a major step forward in global HIV prevention efforts. This innovation could improve adherence, expand access, and significantly reduce new infections, particularly in high-risk populations.

Learn more

NIH Unveils Ultra-High-Resolution MRI to Map the Brain Like Never Before

NIH researchers have developed a groundbreaking MRI scanner capable of imaging the human brain at a microscopic level—achieving resolutions as fine as 0.2 millimeters. This leap in imaging power will allow scientists to visualize brain structures previously only seen in post-mortem tissue, opening new frontiers in neuroscience, neurodegenerative disease research, and precision diagnostics. The scanner’s first human images mark a major milestone in noninvasive brain mapping.

Learn more

Takeda’s Narcolepsy Drug Scores Double Phase 3 Wins

Takeda’s selective OX2R agonist has achieved dual success in pivotal phase 3 trials for narcolepsy, putting the company on track to submit for regulatory approval later this fiscal year. The strong efficacy results position the drug as a potential blockbuster in a field with limited treatment options—signaling renewed momentum in CNS drug development and presenting valuable partnering opportunities for sponsors focused on rare and sleep-related disorders.

Learn more

Novel Oxazolidinones Show Promise as Safer TB Treatment Alternatives

Recent Phase IIb clinical trials evaluated two next-generation oxazolidinones—sutezolid and delpazolid—in combination with bedaquiline, delamanid, and moxifloxacin for drug-sensitive pulmonary tuberculosis. Both antibiotics demonstrated strong antimicrobial efficacy with significantly improved tolerability compared to linezolid. There were no reports of hematological toxicity or peripheral neuropathy over 16 weeks, positioning these compounds as potential safer alternatives for TB patients requiring extended therapy durations. Future trials will determine their place in next-generation TB regimens.

Learn more

AstraZeneca’s Gefurulimab Hits Phase 3 Endpoint in Myasthenia Gravis

AstraZeneca’s anti-CD40 antibody Gefurulimab has met its primary endpoint in a phase 3 trial for generalized myasthenia gravis (gMG), marking a significant step forward in autoimmune neurology. The positive data support the potential of CD40 pathway modulation as a novel therapeutic approach for gMG and position AstraZeneca as a future competitor in a market currently led by Alexion and Argenx. Regulatory submission is expected soon.

Learn more

Celcuity’s Gedatolisib Shows Landmark Results in Phase 3 Breast Cancer Trial

Celcuity has reported groundbreaking results from its Phase 3 VIKTORIA-1 trial, with gedatolisib demonstrating a 76% reduction in risk of progression or death in patients with HR+/HER2–, PIK3CA wild-type advanced breast cancer. Both triplet and doublet regimens significantly outperformed standard therapy, offering new hope for a population with limited targeted options. Regulatory submission is planned for Q4 2025, positioning gedatolisib as a potential first-in-class treatment.

Learn more

AI Model Shows Promise for Streamlining Alzheimer’s Clinical Trials

Researchers at the University of Cambridge have developed an AI model that can improve patient selection in Alzheimer’s clinical trials by identifying slow and fast disease progressors. Applied retrospectively, it revealed significant treatment effects in early-stage patients, suggesting AI could make trials faster, cheaper, and more targeted.

Learn more