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Mirum Pharmaceuticals has entered into an agreement to acquire Bluejay Therapeutics, gaining global rights to brelovitug with Breakthrough Therapy and PRIME designations, and expanding Mirum’s rare liver disease portfolio
As per the deal, Mirum will acquire all Bluejay shares for $250M in cash and $370M in Mirum stock, plus up to $200M in sales-based…
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The US FDA has approved Ctexli (chenodiol) to treat cerebrotendinous xanthomatosis (CTX) in adults
Efficacy was established in crossover withdrawal P-III (RESTORE) trial assessing Ctexli (250mg, PO, TID) vs PBO in pts for 24wks.
Study showed reduced levels of plasma cholestanol & urine 23S-pentol in pts compared to PBO
Ref: Prnewswire |Â Image:Â Mirum
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An equity hedge fund manager company, BVF Partners invests in small cap and early-stage biotechnology companies
In 2023, BVF participated in five investment rounds and added 20 companies to its portfolio
For a curated report on a specific investor or venture capital, reach out to us at connect@pharmashots.com
Established in 1993 in San Francisco, California,…
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The CHMP has granted positive opinion to Livmarli (oral solution) for PFIC patients (age: ≥3mos.) along with COMP for maintaining ODD. EC’s decision is anticipated in Q3’24 Â
The opinions were supported by the P-III (MARCH) trial involving 93 patients with PFIC types (PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 & unidentified mutational status), showing decreased…

