Dear Subscribers, This is what we learned this morning: FTX: we learned about the arrest of Sam Bankman Fried and about the firm’s hearing today. We heard from several sources that FTX has been a house of cards built on deception. It forced us to ask how many of these house of cards are stock market investors tempted to buy; watching stocks of small bad …

IVERIC bio's Rally IVERIC Bio (ISEE) announced that the U.S. (FDA) has granted Breakthrough Therapy designation for avacincaptad pegol (ACP), also known as Zimura®. Zimura is a novel investigational complement C5 inhibitor aimed at treating geographic atrophy (GA) secondary to Age-Related . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Reata Pharmaceuticals Announcements The FDA decision not to hold an Advisory Committee Meeting to discuss Reata Pharmaceuticals product Omaveloxolone for the treatment of Friedreich’s ataxia hints to good news about the approval of this product approval on  February 2023.   The NDA for Omaveloxolone for Friedreich’s ataxia is under review. A PDUFA Date is decided upon on February 28, 2023. Recently, Reata Pharmaceuticals (

Searching for Biotech Firms The search is for firms biotech firms that have reached clinical stage with novel products that had some earlier, encouraging test results, whose technologies and lead products must have demonstrated  improvement over current treatments of devastating diseases, including cancers and genetic-derived diseases.   Recently, we became interested in a firm called Acumen’s Pharmaceuticals (

Acumen Pharmaceuticals Product ACU193 On October 23, 2022, Acumen Pharmaceuticals (ABOS) announced that the first clinical-stage monoclonal antibody ACU193 - an anti-amyloid beta oligomer antibody, which selectively targets toxic soluble amyloid beta oligomers (AβOs), has been granted  FDA Fast Track designation for the treatment of early Alzheimer . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Gilead Sciences and MacroGenics Collaboration Announcement Gilead Sciences (GILD) and MacroGenics (MGNX) announced an exclusive option and collaboration agreement to develop MGD024 using MacroGenics DART . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Reata Pharmaceuticals Announcement Regarding the NDA for Omaveloxolone for Friedreich’s Ataxia Reata Pharmaceuticals (RETA) announced that the U.S. Food and Drug Administration (FDA) does not plan to hold an advisory committee meeting in connection with its review of the Company’s New Drug Application (NDA) for omaveloxolone for the treatment of patients with Friedreich’s ataxia. From Reata Pharmaceuticals CEO Warren Huff, Reata’s Chief Executive Officer, said, …

IgA Nephropathy  Searching for companies focused on treating severe diseases that have yet to find approved drugs is one of Prohost Biotech's tasks.  Recently we wrote about firms that have, in their pipelines, products that aim at treating NASH and ALS.  Now, we are currently interested in finding firms that have products that can successfully treat IgA nephropathy, among other diseases that have yet to …

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 …

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 (

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 …

Intellia Therapeutics  Intellia Therapeutics.(NTLA) and Regeneron Pharmaceuticals (REGN) today announced positive interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Rhythm Pharmaceuticals Rhythm Pharmaceuticals (RYTM): After soaring, based on great scientific and drug performance in treating genetic obesity, the selloff of the stock has occurred today after the firm’s announcement and pricing of its public offering of 4.8M shares of its common stock at $26 a share. Akero Therapeutics Akero Therapeutics (

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 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 ~ 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 …

TG Therapeutics Results Published in NEJM TG Therapeutics (TGTX) results from the ULTIMATE I & II Phase 3 trials evaluating the firm’s investigational monoclonal antibody product ublituximab in patients with relapsing forms of multiple sclerosis (RMS) were

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: 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.

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. …