Soleno Therapeutics Soleno Therapeutics (SLNO) is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s lead candidate, DCCR extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi Syndrome (PWS), recently completed its Phase 3 development program to support a planned NDA submission.  For more information, please . . . This content is for paid subscribers. …

Pliant Therapeutics in the NEWS Treating Primary Sclerosing Cholangitis  Pliant Therapeutics (PLRX) today announced positive data from a Phase 2a INTEGRIS-PSC clinical trial of the firm’s product bexotegrast in patients with primary sclerosing cholangitis (PSC) and suspected moderate to severe liver fibrosis. PSC is a rare, progressive liver disease of unknown origin, which frequently occurs in the setting of . . . This content is for …

Reat Pharmaceuticals Merger with Biogen Reata Pharmaceuticals announced that at a special meeting held today, preliminary results indicate that the stockholders of the firm have voted to approve the Company’s previously announced acquisition (the “Merger”) by Biogen (BIIB). Around 99.65% of the votes cast by the Company’s Class A common stockholders and Class B common stockholders, voting as . . . This content is for …

CARsgen Therapeutics Holdings Limited Today, September 21, 2023, CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK) - a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, announced the publication of two cases of metastatic pancreatic cancer treated with CARsgen’s CT041- an innovative Claudin (CLDN) 18.2 CAR T-cell Therapy in Journal of Hematology & Oncology . . . This content …

Orchard Therapeutics in the NEWS Today, September 18, 2023, Orchard Therapeutics (ORTX) - a global gene therapy firm, announced that the U.S. FDA has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review. The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024. From Orchard Therapeutics Bobby Gaspar, M.D., …

Prohost Letter #457 Discovering The Root Causes of Diseases   Biotech firms with solid scientific fundamentals are racing towards unveiling the root-causes of various diseases that have yet to be completely unveiled. Knowing the root causes of diseases enabled some of the clinical-stage firms and the revenues-generating firms to bring into the clinic novel advanced treatments. Improving the treatments of severe untreatable diseases depends on …

Neurocrine Biosciences in the NEWS Today, Neurocrine Biosciences (NBIX) announced positive top-line data from Phase 3 CAHtalystTM Adult Study evaluating the efficacy, safety, and tolerability of crinecerfont in adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD). The Phase 3 study met its primary endpoint at Week 24, demonstrating that . . . This content is for paid subscribers. Please click here to …

Crinetics Pharmaceuticals Once Daily, Oral Treatment for Acromegaly Crinetics Pharmaceuticals (CRNX) announced that its oral and once daily investigational compound paltusotine achieved positive results by meeting the primary endpoint and all secondary endpoints of the Phase 3 PATHFNDR-1 study.   PATHFNDR-1 study enrolled participants with acromegaly who were biochemically controlled on octreotide or lanreotide depot monotherapy. PATHFNDR-1 is . . . This content is for …