Mirum Pharmaceuticals

The FDA is set to decide on ten drug applications, including Ionis' olezarsen for familial chylomicronemia syndrome, Lexicon's sotagliflozin for type 1 diabetes, AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC, Zealand Pharma's glepaglutide for short bowel syndrome, Rhythm Pharmaceuticals' Imcivree for genetic obesity in children, Checkpoint Therapeutics' cosibelimab for cutaneous squamous cell carcinoma, Mirum's chenodiol for cerebrotendinous xanthomatosis, Bristol Myers Squibb's subcutaneous Opdivo, and Neurocrine's crinecerfont for congenital adrenal hyperplasia.

Mirum Pharmaceuticals plans to launch a Phase 2 clinical trial for MRM-3379, an oral treatment for fragile X syndrome, in 2025. MRM-3379, designed to block PDE4D enzyme, aims to increase cAMP levels and potentially alleviate symptoms. The trial follows Mirum's acquisition of global rights to the drug from Enthorin Therapeutics and Dart Neuroscience.

GSK's phase III GLISTEN study shows linerixibat significantly improves cholestatic pruritus in PBC patients, with potential to be the first global therapy for PBC-related itch. Preliminary safety data aligns with previous studies. Full data to be presented at a future medical conference.

Mirum Pharmaceuticals' volixibat granted breakthrough therapy status by FDA for pruritus in PBC, based on Phase 2b VANTAGE trial interim results showing significant itch reduction. Trial now in confirmatory phase, aiming to support regulatory approval. Volixibat works by inhibiting ileal bile acid transporter to reduce bile acid accumulation and alleviate PBC symptoms.

Paola Laverde filed a complaint against Berkeley Mayoral candidate Sophie Hahn with the state's Fair Political Practices Commission (FPPC) for failing to report her 50% share of her spouse's yearly earnings from Mirum Pharmaceuticals, estimated at $500,000 to $1 million annually, and substantial stock holdings in biotech companies, estimated at $4 million. The complaint, supported by thorough documentation, highlights Hahn's omissions in her FPPC Form 700 filings, which are required to disclose personal financial interests and potential conflicts of interest.

Ed Arce of H.C. Wainwright maintains Buy rating on Mirum Pharmaceuticals (MIRM) with $66.00 price target, citing FDA's Breakthrough Therapy Designation for volixibat in treating cholestatic pruritus in PBC patients, and its potential to offer substantial improvement over existing therapies.

FDA grants breakthrough therapy designation to Mirum's volixibat for treating cholestatic pruritus in PBC patients. Volixibat, under Phase IIb VANTAGE study, showed significant pruritus reduction and 75% patient improvement in serum bile acids. The drug selectively inhibits ileal bile acid transporter, reducing systemic bile acids. The confirmatory VANTAGE study is ongoing, with full enrolment expected by 2026.

FDA grants Breakthrough Therapy Designation to Mirum's volixibat for cholestatic pruritus in PBC, based on Phase 2b VANTAGE study's positive interim analysis. Confirmatory study ongoing, with enrollment completion expected in 2026.

The FDA granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients, based on positive Phase 2b VANTAGE study results. The study's confirmatory phase is ongoing, with enrollment expected to complete in 2026.

The FDA granted breakthrough therapy designation to volixibat, an oral ileal bile acid transporter inhibitor, for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients. The designation is based on the phase 2b VANTAGE study's interim analysis, showing statistically significant improvements in pruritus and serum bile acids, with positive safety signals. Confirmatory trial completion is expected in 2026.