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A Phase 3 Win and a 130% Takeover Premium Lit Up Rare Blood-Disease Stocks on Monday

  • Disc Medicine reported its Phase 3 APOLLO trial for bitopertin met all primary & secondary endpoints for erythropoietic protoporphyria, reducing protoporphyrin IX by 41.4% & increasing pain-free light exposure by 12.2 hours.
  • Disc Medicine plans to resubmit its New Drug Application by end-2026 & expects an FDA decision by mid-2027, with shares rising 38.86% to $78.93.
  • Silence Therapeutics agreed to be acquired by Merck KGaA for $20.00 per ADS, a 130% premium, with an equity value of ~$1.3 billion & a closing expected in Q1 2027.
  • Silence's acquisition deal focuses on divesiran, an siRNA therapy for polycythemia vera that showed an 88% response rate in Phase 2 trials, causing shares to rise 125.58% to $19.58.
  • Protagonist Therapeutics received $275 million from Takeda following FDA approval of rusfertide (MIMRYLO) for polycythemia vera & holds eligibility for up to $875 million in additional milestones.
  • Agios Pharmaceuticals received Priority Review for mitapivat in sickle cell disease with a PDUFA date of November 1, 2026, after a Phase 3 trial showed 40.6% hemoglobin response rates.
  • Keros Therapeutics expects a $20 million development milestone from Takeda following the first patient dose in the ELRiSE MF Phase 3 trial for elritercept in myelofibrosis.
  • Market movement was driven by biology targeting red blood cells & iron, specifically via hepcidin pathways & heme production, affecting conditions like polycythemia vera & erythropoietic protoporphyria.