Apellis Pharmaceuticals Negative CHMP Opinion Apellis Pharmaceuticals (APLS) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion on the marketing authorization application (MAA) of intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). This opinion was expected based . . . This content is for paid subscribers. …

Akous Inc to Present at the Association for Research in Otolaryngology Akouos, Inc. - a wholly owned subsidiary of Eli Lilly and Company (LLY) announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which restored the hearing within 30 days of AK-OTOF administration in the first participant, with more than a decade history of deep . . . This content is for paid …

Compugen's Soaring Stock Many recent events  led to Compugen's (CGEN) soaring stock after a huge decline in its price. In spite of the tremendous stock decline, Prohost never doubted the fact that Compugen has solid scientific fundamentals and great scientists. The firm has done a great  job utilizing its broadly applicable predictive computational discovery capabilities to identify new drug targets and biological . . . …

Cytokinetics We recently became enthusiastic about Cytokinetics’ (CYTK) current clinical trial results. A few days ago, Cytokinetics announced positive results from SEQUOIA-HCM (Safety, Efficacy, and Quantitative Understanding of Obstruction Impact of Aficamten in HCM), the pivotal Phase 3 clinical trial of aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Why the Great Enthusiasm? The following has been behind our enthusiasm . . . This …

Sarepta Therapeutics in the NEWS On 12, 22, 2023, Sarepta Therapeutics (SRPT) - a precision genetic medicine for rare diseases, announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows: “ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed . . . This content is for paid …

The information is the first of a few biotech Companies that we believe are promising in treating severe devastating  untreatable sicknesses.  Arcturus Therapeutics Holdings Based in San Diego, California, Arcturus Therapeutics Holdings (ARCT) was founded in 2013. The Company is a late-stage clinical developing novel mRNA medicines and vaccines which already  demonstrated promising results in early trials. The Company is creating . . . This content …

Anavex Life Sciences Promising News From Anavex Life Sciences  (AVXL) we learned that The Committee for Medicinal Products for Human Use (CHMP) within the European Medicines agency (EMA) agreed that oral blarcamesine for Alzheimer’s disease is eligible for submission of application for a Union Marketing Authorization in the EU under the European Medicines Agency’s centralized procedure . . . This content is for paid subscribers. Please click here …

Crispr Therapeutics and Vertex Pharmaceuticals Gene Editing Therapey Will Be Marketed On Nov. 16, 2023, Crispr Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX)

Novartis Approval Novartis (NVS) product Fabhalta (iptacopan) is a Factor B inhibitor that acts in the proximate alternative complement pathway of the immune system, It provides comprehensive control of red blood cell (RBC) destruction within and outside the blood vessels (intra- and extravascular hemolysis.   Fabhalta treatment of paroxysmal nocturnal hemoglobinuria (PNH) increased hemoglobin levels (≥ 2 g . . . This content is for paid …

Altimmune Positive Topline Results Altimmune (ALT) announced positive results from the MOMENTUM 48-Week Phase 2 Obesity Trial of Pemvidutide.  Altimmune is a clinical-stage biopharmaceutical company focused on developing treatments for obesity and liver fibrosis. The Company’s lead product candidate, pemvidutide, is a GLP-1/glucagon dual receptor agonist developed for treating obesity and metabolic dysfunction-associated steatohepatitis (MASH

FDA Approval of Eli Lilly Product Zepbound for Obesity On Wednesday, Nov. 8, 2023,  the U.S. FDA approved Eli Lilly (LLY) product ZepboundTM (tirzepatide) injection for the treatment of obesity.  Zepbound is the first and only obesity treatment of its kind that activates both GIP (glucose-dependent insulinotropic polypeptide) and GLP-1 (glucagon-like peptide-1) hormone receptors. Zepbound is indicated for adults with obesity (with a BMI of …

Three Biotech Firm in the NEWS Verona Pharma Verona Pharma plc (VRNA) announced that senior management will present a company overview at the following conferences in November 2023: Jefferies London Healthcare Conference Date: Wednesday, November 15, 2023 Time: 10:00 AM EST / 3:00 PM GMTLocation: London, UK Piper Sandler 35th Annual Healthcare . . . This content is for paid subscribers. Please click here to subscribe or …

Neurocrine Biosciences’ Product INGREZZA Improved Chorea Associated with Huntington's Disease Through Week 50  Yesterday, November 2, 2023, Neurocrine Biosciences (NBIX) announced interim results from the KINECT®-HD2 study about INGREZZA® (valbenazine) capsules used for long-term treatment of adults with chorea associated with Huntington's disease (HD). The Interim data suggest that one-capsule, once-daily of INGREZZA improved . . . This content is for paid subscribers. Please click here …

Anavex Life Sciences NEWS Anavex Life Sciences (AVXL) reported a new peer reviewed publication in journal Neurobiology of Aging, titled “Early treatment with an M1 and sigma 1 receptor agonist . . . 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 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 …

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. …