Intellia Therapeutics' NTLA-2001 Intellia Therapeutics' NTLA-2001 is the first-ever investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development.   Today, October 18, 2023, Intellia Therapeutics (NTLA) announced that the U.S. Food and . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Intellia Therapeutics Receives PRIME Designation from EMA Intellia Therapeutics (NTLA) announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to its product NTLA-2002.  As we already know, NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to prevent potentially life-threatening swelling attacks in people with Hereditary Angioedema  (HAE). From Intellia Therapeutics . . . This content is for paid subscribers. Please …

Inovio Receives FDA Feedback for INO-3107 INOVIO (INO) - a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, announced that it received feedback from the U.S. FDA that data from its completed Phase 1/2 trial of INO-3107 for the treatment of Recurrent . . . This content is for paid subscribers. …

Akero Therapeutics Press Release Akero Therapeutics Reports Encouraging 36-Week Analysis of 96-Week Phase 2b SYMMETRY Study, with a Trend on Fibrosis Improvement and Statistically Significant Results for NASH Resolution, Markers of Liver Injury and Fibrosis, Insulin Sensitization and Lip Akero Therapeutics (AKRO)

Akero Therapeutics to Present Results from Phase 2b SYMMETRY Study Investigating Efruxifermin in Patients with Compensated Cirrhosis Due to NASH Investor webcast on Tuesday, October 10th at 8:00 a.m. ET to present clinical data.   Akero Therapeutics (AKRO) will hold an investor conference . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Apellis Pharmaceuticals Launch Update of SYFOVRE® On October 5, 2023, Apellis Pharmaceuticals (APLS) provided an update on the launch of its product SYFOVRE® (pegcetacoplan injection) for geographic atrophy (GA) secondary to age-related macular degeneration (AMD). About Geographic Atrophy GA is an advanced form of age-related macular degeneration and a leading cause of blindness, impacting more than one million Americans . . . This content is for …

Neurocrine Biosciences Announcement Today, Neurocrine Biosciences (NBIX) announced positive top-line data from the Phase 3 CAHtalyst™ Pediatric Study evaluating the safety, efficacy and tolerability of the firm’s product crinecerfont treating children and adolescents with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. The Disease Congenital adrenal hyperplasia refers to a group of genetic conditions that result in an enzyme deficiency that alters . . . …

Akero Therapeutics Akero Therapeutics (AKRO) announced publication in The Lancet Gastroenterology & Hepatology of results from the HARMONY Phase 2b trial in nonalcoholic steatohepatitis (NASH). The paper, which is available online, provides data on the safety, tolerability, and efficacy of once-weekly subcutaneous injections . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

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., …

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 …

Alpine Immune Sciences Treating IgA nephropathy Alpine Immune Sciences (ALPN) announced a successful initiation of the second IgA nephropathy (IgAN) dose cohort in RUBY-3, a phase 1b/2a study in autoimmune glomerulonephritis. The safety Monitoring Committee (SMC) determined that repeat . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Halozyme Therapeutics in the NEWS Halozyme Therapeutics (HALO) announced today that the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain has approved Roche's (

Exelixis in the NEWS Exelixis CABOMETYX is approved for the treatment of many cancers. The product has been game changer in the treatment of various difficult to treat cancers before the products that target PD-L1 and approved for several various  cancers were developed by MRK and BMY.     Exelixis (EXEL) announced that the Alliance for Clinical Trials in Oncology independent Data . . . …

Arcus Biosciences Arcus Biosciences (RCUS) is a clinical-stage, global biopharmaceutical company developing differentiated molecules and combination medicines for people with cancer and chronic inflammatory diseases, and other diseases. In partnership with firms and physicians around the world, Arcus is expediting the development of first- or best-in-class medicines against well-characterized biological targets and pathways while studying novel, biology-driven . . . This content is for paid …

Regeneron Pharmaceuticals EYLEA HD Approved by the FDA Regeneron Pharmaceuticals (REGN) announced that the U.S. FDA has approved EYLEA HD (aflibercept) Injection 8 mg for the treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR). The recommended dose for EYLEA HD is 8 mg . . . This content is for paid subscribers. Please click here to subscribe or …