Astellas Revives XLMTM Gene Therapy Trial After Patient Deaths: Key Biotech Updates

Stay updated on the latest breakthroughs and strategic moves in biotech with our curated newsletter. This week’s highlights include groundbreaking gene therapy trials, major acquisitions, and transformative clinical trial results.

Major Biotech Acquisitions and Strategic Moves

Eli Lilly announced a definitive agreement to acquire Ajax Therapeutics, a privately held biotech developing treatments for blood cancers, for up to $2.3 billion. Ajax’s lead candidate is a next-generation JAK2 inhibitor currently undergoing a Phase 1 trial in patients with myelofibrosis.

Ligand Pharmaceuticals revealed plans to acquire Xoma, a company specializing in aggregating drug royalties, for $739 million.

Clinical Trial Results and Setbacks

Compass Therapeutics reported that its experimental drug for advanced biliary cancer delayed tumor progression but failed to improve overall survival compared to standard chemotherapy in a clinical trial.

Shares of BridgeBio surged following reports that Pfizer had settled two of three patent disputes related to Vyndamax, a treatment for the heart condition ATTR-CM. While settlement terms remain undisclosed, any extension of Vyndamax’s patent protection is expected to benefit BridgeBio, which markets a competing drug called Attruby for the same condition.

CRISPR Therapy Shows Promise in Rare Disease

Intellia Therapeutics announced that its one-time CRISPR treatment, lonvo-z, reduced attack rates by 87% in a Phase 3 trial for hereditary angioedema. The trial demonstrated that over 60% of patients became attack-free, with no serious safety concerns reported. This positions lonvo-z as a potential functional cure or significant improvement over existing therapies.

Astellas Re-enters Clinic with XLMTM Gene Therapy

Astellas Pharma is re-entering clinical trials for its next-generation gene therapy for X-linked myotubular myopathy (XLMTM) after pausing development following patient deaths. The company is implementing protocol changes to enhance patient safety and resume testing.

Key Takeaways

• Eli Lilly’s $2.3B acquisition of Ajax Therapeutics strengthens its pipeline in blood cancer treatments.
• Ligand Pharmaceuticals’ $739M purchase of Xoma expands its royalty aggregation strategy.
• Intellia’s CRISPR therapy achieves an 87% reduction in attack rates for hereditary angioedema, offering a potential cure.
• Astellas’ XLMTM gene therapy resumes trials with enhanced safety measures after initial setbacks.