Arrowhead Completes Enrollment in Phase 3 YOSEMITE Study of Zodasiran for HoFH

Sarepta Therapeutics reported two-year results on September 30 for its Elevidys gene therapy, covering 25 ambulatory patients aged 8 to 12 compared with 99 patients in an external control group, with the company citing functional benefits relative to those controls. The comparison used an external control rather than a randomized concurrent control group, a design point the company said matters when interpreting the findings. Commercially, Sarepta generated $328.7 million in second-quarter product revenue, including $230.6 million from its PMO therapies and $98.1 million from Elevidys, alongside GAAP operating income of $13.3 million and approximately $945 million in cash, restricted cash and investments. Total second-quarter revenue declined to $401.3 million from $611.1 million a year earlier, reflecting lower Elevidys revenue and changes in collaboration revenue. Elevidys still carries an FDA boxed warning for serious liver injury and acute liver failure, including fatal outcomes, and in November 2025 the FDA restricted its indication to ambulatory patients aged four and older with a confirmed mutation in the DMD gene following reports of fatal liver failure in nonambulatory patients; the September follow-up findings do not remove those restrictions or the warning. The shares traded at approximately 0.9x trailing sales, compared with 3.2x for rare-disease drugmaker BioMarin Pharmaceutical, while short interest stood at 28.86% of the float and hedge fund holders fell to 37 in the second quarter from 41 in the first, with AQR Capital Management the largest holder at 4.3 million shares and Marshall Wace LLP raising its position by 205% to 2.2 million shares.