Pharmaceutical Industry Roundup: Sarepta's Rebound, Alzheimer's Breakthroughs, and Q2 Earnings

Sarepta's Elevidys Back on Track as FDA Lifts Hold

Sarepta Therapeutics received a significant reprieve as the FDA recommended lifting the voluntary hold on Elevidys, the company's gene therapy for Duchenne muscular dystrophy (DMD), for ambulatory patients. This decision came after the agency determined that the death of an 8-year-old Brazilian DMD patient who had received Elevidys was not caused by the drug. The news marks a positive turn for Sarepta, whose stock has experienced volatility in recent weeks due to concerns about patient deaths in its gene therapy trials.

Alzheimer's Research Advances at AAIC25

At the Alzheimer's Association Annual Conference in Toronto, Roche's trontinemab emerged as a standout therapy. In a Phase Ib/IIa trial, this next-generation anti-amyloid antibody demonstrated rapid clearance of amyloid from patients' brains after just seven months of treatment. This timeframe significantly outpaces the 18-month clearance period observed with Biogen and Eisai's Leqembi and Eli Lilly's Kisunla.

While Leqembi and Kisunla have shown some progress in slowing Alzheimer's progression, their modest effect size and limited patient applicability leave room for both disease-modifying and symptomatic treatments. Companies like Bristol Myers Squibb and Acadia Pharmaceuticals are developing symptomatic treatments to address this gap, with several readouts expected in the near future.

Q2 Earnings and Leadership Changes in Big Pharma

The pharmaceutical industry's second-quarter earnings reports are in full swing, with several major players announcing their results:

• AstraZeneca reported estimate-beating numbers, with CEO Pascal Soriot emphasizing the need for global sharing in pharmaceutical R&D.
• Merck announced a $3 billion cut to support an aggressive launch schedule for new products.
• Novo Nordisk, ahead of its earnings report, named a new CEO and lowered its 2025 sales guidance for the second time this year.

These financial updates reflect the dynamic nature of the pharmaceutical market and the strategic moves companies are making to position themselves for future growth and innovation.

Regulatory and Policy Developments

In a controversial move, Health Secretary Robert F. Kennedy Jr. has announced plans to dissolve the U.S. Preventive Services Task Force, citing it as "too woke." Additionally, Kennedy addressed what he termed the "broken" vaccine injury compensation program, vowing to "fix" the U.S. Vaccine Injury Compensation Program (VICP) and return it to its "original Congressional intent." These proposed changes could have significant implications for public health policy and vaccine regulation in the United States.