Author: Prohost

Amylyx Pharmaceuticals Announces FDA Approval of RELYVRIO™ for of ALS Leading to a Weird Stock Market Reception

Amylyx Pharmaceuticals in the NEWS Amylyx Pharmaceuticals (AMLX) announced that the U.S. FDA has approved RELYVRIO™ (sodium phenylbutyrate and taurursodiol) for the treatment of adults with amyotrophic lateral sclerosis (ALS). The product, RELYVRIO, (previously known as AMX0035 in the U.S.) has significantly slowed the loss of physical function in people living with ALS in a randomized, placebo-controlled . . . This content is for paid …

From Biogen and Eisai: Lecanemab Confirmatory Phase 3 Clarity Met Primary Endpoint for Early Alzheimer’s Disease  

Biogen & Eisai Co and Early Alzheimer's Disease  From Tokyo and Cambridge, Eisai Co., Ltd. and Biogen Inc. (BIIB) announced positive topline results from Eisai’s large global Phase 3 confirmatory Clarity AD clinical trial of lecanemab. The product is an investigational anti-amyloid beta (Aβ) protofibril antibody for the treatment of mild cognitive impairment (

From bluebird bio: FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy

FDA Approves bluebird bio Product Skysona for Early, Active CALD SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys with this devastating and fatal neurodegenerative disease. The U.S. FDA has granted Accelerated Approval of bluebird bio’s (BLUE) SKYSONA® (elivaldogene autotemcel), also known as eli-cel. SKYSONA® is indicated to slow the progression of neurologic dysfunction in boys 4-17 …

Akero Therapeutics: Efruxifermin Met Primary Endpoint for Both Dose Groups in NASH Patients

Akero Therapeutics Akero Therapeutics (AKRO) - a clinical-stage company, today released topline data from HARMONY, a 24-week Phase 2b study evaluating the efficacy and safety of its lead product candidate efruxifermin (EFX) in patients with pre-cirrhotic nonalcoholic steatohepatitis (NASH), fibrosis stage 2 or 3 (F2-F3). The study met its primary endpoint for both the 50mg . . . This content is for paid subscribers. Please …

Amylyx Pharmaceuticals Announces FDA Advisory Committee Supports Approval of AMX0035 for ALS

Amylyx Pharmaceuticals in the News Amylyx Pharmaceuticals (AMLX): The U.S. FDA Peripheral and Central Nervous System Drugs Advisory Committee has voted (7 yes votes and 2 no votes) that the available evidence of effectiveness is sufficient to support approval of AMX0035 (sodium phenylbutyrate and taurursodiol ) for the treatment of amyotrophic lateral sclerosis (ALS). The advisory committee’s decision was based on a review . . . This content is …

IVERIC Bio: Undeniable Promising News Today

IVERIC Bio ~ September 6, 2022 IVERIC bio (ISEE) announced positive results from GATHER2 second Phase 3 clinical trial of Zimura® (avacincaptad pegol) - a novel investigational complement C5 inhibitor for the treatment of geographic atrophy (GA). GATHER2 met its prespecified primary endpoint of mean rate of growth (slope) in GA area at 12 months with statistical significance and a . . . This content …

Intellia Therapeutics to Present Data from Clinical Trial of NTLA-2002 Aimed at Treating Hereditary Angioedema

Intellia Therapeutics to Present Interim Clinical Data from NTLA-2002 Trial for Hereditary Angioedema An abstract featuring interim clinical data from the Phase 1/2 study of Intellia Therapeutics’ (NTLA) product NTLA-2002 has been selected for an oral presentation at the 2022 Bradykinin Symposium, taking place September 15-16 in Berlin, Germany. NTLA-2002 NTLA-2002 is an investigational in vivo CRISPR/Cas9 therapy in development as a single-dose treatment to prevent …

Fate Therapeutics: Improving on Cellular Therapies

Fate Therapeutics: Developing Next Generation Cellular Therapies Fate Therapeutics (FATE) is a clinical-stage biopharmaceutical company developing first-in-class improved cellular immunotherapies for cancer. Fate Therapeutics uses its proprietary induced pluripotent stem cell (iPSC) product platform in clinical development and manufacture of universal off-the-shelf cell products. The . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Important Data Will Be Presented by Regenxbio on August 30th

Regenxbio to Present Important Data  Regenxbio (RGNX) will present important material at the Society for the Study of Inborn Errors of Metabolism Annual Symposium, taking place in Germany from August 30 through September 2, 2022. The presentations are meant to highlight new data from the Phase I/II/III CAMPSIITE™ trial of RGX-121, an investigational one-time AAV Therapeutic . . . This content is for paid subscribers. …

The FDA Advisory Committee Will Soon Review the NDA of Amylyx Pharmaceuticals’ Product AMX0035 for ALS  

On September 7th the FDA Will Review Amylyx Pharmaceuticals NDA for ALS Treatment Amylyx Pharmaceuticals (AMLX) announced that the U.S. FDA Peripheral and Central Nervous System Advisory Committee (PCNSDAC) will reconvene to review the New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate and taurursodiol known also as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS

Hutchmed: Treating Advanced Metastatic Colorectal Cancer and Other Treatment Resistant Cancers

Hutchmed: Treating Advanced Refractory Metastatic Colorectal Cancer On August 8, 2022, Hutchmed (HCM) announced that pivotal global Phase 3 FRESCO-2 trial evaluating the investigational use of fruquintinib met its primary endpoint of overall survival (OS) in patients with advanced, refractory metastatic colorectal cancer (CRC). The FRESCO-2 Study A multi-regional clinical trial conducted in the U.S., Europe, Japan and Australia demonstrates that investigated fruquintinib plus best supportive …

Reata Pharmaceuticals Announces Extension of Review Period for Omaveloxolone NDA for Friedreich’s Ataxia

Reata Pharmaceuticals in the NEWS Reata Pharmaceuticals (RETA) on August 8, 2022, after the U.S. financial markets closed, received a communication from the U.S. Food and Drug Administration (FDA) informing us that they have extended the review timeline for the New Drug Application (NDA) for omaveloxolone for the treatment of Friedreich’s ataxia by three . . . This content is for paid subscribers. Please click here …

Gilead Sciences: A Promising Acquisition. See Also: Genmab and BioNTech: A Promising Expansion

Gilead Sciences to Acquire MiroBio At $405 Million Gilead Sciences (GILD) and MiroBio - a privately held U.K based biotechnology company focused on restoring immune balance with agonists targeting immune inhibitory receptors, announced that they entered into a definitive agreement pursuant to which Gilead will acquire MiroBio for approximately $405 million in . . . This content is for paid subscribers. Please click here to subscribe …

Regenxbio to File a BLA Using the Accelerated Approval Pathway for RGX-121 for the Hunter Syndrome

Regenxbio in the NEWS  Regenxbio (RGNX) announced, on August 3, 2022, its intention to file a Biologics License Application (BLA) in 2024 using the FDA's accelerated approval pathway for RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome.   The Company has also announced that a pivotal program for RGX-121 is . . . This content is for paid …

Bristol-Myers Squibb Combination Improving the Treatment of Metastatic Melanoma; AbbVie Improving the Treatment of Crohn’s Disease

Offsetting the Biotech Firms' Good News There is no doubt that good news is filling the media about outperformances of both development-stage small biotech firms and revenues-generating firms. This good news has been offset by inflation, hiked interest rates, that aimed at bringing down the inflation, the continued presence of COVID-19 and an unwarranted European  war, which has only demonstrated the terrible impact on the …