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Disc Medicine’s bitopertin succeeds in Phase 3 after FDA rejection

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Disc Medicine reported positive top-line results from its Phase 3 APOLLO trial of bitopertin, an investigational treatment for erythropoietic protoporphyria (EPP), a rare inherited disorder that can cause severe pain and skin reactions after light exposure. The 183-person trial met both primary endpoints: compared with placebo, bitopertin reduced blood levels of metal-free protoporphyrin IX (PPIX) by 41.4% and gave patients an average of 12.2 additional hours per month of pain-free time in light during month six. The company said all secondary endpoints were also met, including fewer painful phototoxic reactions. Bitopertin was generally well tolerated, with no new safety concerns reported. The FDA had issued a Complete Response Letter in February, agreeing that bitopertin lowered PPIX but saying earlier studies had not established that this translated into improved light tolerance. The APOLLO results directly address that concern. Disc plans to resubmit its application by th...

Roche and Hansoh strike potential $15B-plus cancer-drug deal

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AbbVie’s Crohn’s combo outperformed Skyrizi alone in Phase 2a trial

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Gilead’s oral UC drug posts encouraging Phase 2 results

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Novartis stops second phase of Fabhalta lupus-nephritis trial

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Merck KGaA to acquire Silence Therapeutics for about $1.3 billion

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Sanofi partners with ScionxBio on long-acting RNA

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CHMP backs Lilly’s oral GLP-1 pill and five other new medicines

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China proposes export controls on select biopharma technologies

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SimBioSys wins FDA clearance for MRI-based breast cancer recurrence tool

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Estuary launches with $103M to advance kidney disease gene therapies

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City Therapeutics raises $200M in IPO as Frazier biotech SPAC lists

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OrbiMed raises $3B across two healthcare funds

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