Drug Discovery Weekly — 2026-08-21

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statnews.com

statnews.com

statnews.com

Regeneron drug for disease that causes dangerous bone growth earns FDA approval

FDA & Regulatory Decisions

PASATRU (garetosmab) (Regeneron) — Approval

• Indication: Fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic disease causing abnormal bone formation.
• Significance: This is the first therapy shown to reduce new bone lesions and flare-ups in FOP. The approval was based on a 63-patient trial using CT-scan efficacy endpoints, marking a significant milestone for a disease with no prior approved treatments.
• Timeline: Immediate launch expected.

GENGLYCOS (Ultragenyx) — Accelerated Approval

• Indication: Gaucher disease type 1a (GSDIa).
• Significance: Provides a new treatment option for patients with this rare lysosomal storage disorder.
• Timeline: Immediate launch expected.

Capricor Cell Therapy — Regulatory Update

• Indication: Duchenne muscular dystrophy.
• Significance: The FDA is reportedly "willing to review" an updated submission for Capricor’s cell therapy, a positive signal for the company's pipeline.
• Timeline: Review pending.

Clinical Trial Milestones

KEYTRUDA QLEX (Merck) — Commercial Launch

• Sponsor: Merck
• Results: Merck reported second-quarter 2026 financial results highlighting continued strength in oncology, including initial uptake of the subcutaneous formulation KEYTRUDA QLEX.
• What's Next: Continued commercial rollout and integration with the existing KEYTRUDA franchise.

Moderna mRNA Cancer Vaccine — Phase 3 Data

• Sponsor: Moderna
• Results: Recent reports indicate that Moderna's mRNA cancer vaccine has shown promising results, contributing to the biotech sector's upswing.
• What's Next: Further regulatory submissions expected based on ongoing trial data.

Pharma Deals & M&A

• Acquisition: BioMarin + Alesta Therapeutics — BioMarin has agreed to acquire Alesta Therapeutics to gain ALE1, a treatment for a rare disease. This deal adds to the half-dozen bigger-ticket acquisitions of rare disease drugmakers this year.

• Asset Acquisition: LEO Pharma + Tanabe Pharma — LEO Pharma has agreed to acquire worldwide rights to dersimelagon from Tanabe Pharma in a transaction worth up to $435 million in upfront and near-term milestone payments. The deal adds a late-stage oral medicine for rare dermatology conditions.

• Collaboration: OmniAb + Eli Lilly — OmniAb has signed an ion channel discovery collaboration with Eli Lilly, leveraging its AI-driven platform for target identification.

AI & Computational Drug Discovery

• OmniAb + Eli Lilly Partnership: The collaboration between OmniAb and Eli Lilly focuses on ion channel discovery, highlighting the growing integration of AI platforms into big pharma's early-stage R&D.

• Nature Reviews Perspective: A recent Perspective in Nature Reviews Drug Discovery discusses the current state of AI in drug discovery, noting that while interest is high, evidence for clinically relevant impact remains limited due to insufficient focus on clinical translation during model development.

Pipeline Watch: Key Upcoming Catalysts

• December 2026: FDA decision expected for a CD20 antibody for systemic lupus erythematosus (SLE). The drug was recently approved for lupus nephritis.

• Mid-2026: Arrowhead expects to report results for Redemplo for severe hypertriglyceridemia, with an approval filing expected shortly after if data are positive.

Week in Context

This week's developments underscore the continued momentum in rare disease therapeutics, both in terms of regulatory approvals and corporate strategy. Regeneron's approval for FOP and Ultragenyx's for Gaucher disease type 1a demonstrate that the FDA is moving forward with novel mechanisms of action for ultra-rare conditions, often based on smaller, well-designed trials. This regulatory success is driving M&A activity, as seen with BioMarin's acquisition of Alesta and LEO Pharma's deal for dersimelagon. Big pharma is aggressively acquiring late-stage rare disease assets to bolster their pipelines ahead of patent cliffs.

Simultaneously, the integration of AI into drug discovery is moving from hype to strategic partnership. The collaboration between OmniAb and Eli Lilly for ion channel discovery signals that pharma giants are increasingly relying on specialized AI platforms for target identification. However, expert commentary in Nature Reviews suggests that the field still faces hurdles in translating AI models into clinically viable drugs, indicating that while the technology is promising, rigorous validation remains critical. The week also saw continued strength in oncology, with Merck's KEYTRUDA QLEX gaining traction, highlighting the ongoing importance of this therapeutic area.