Amylyx Pharmaceuticals  Amylyx Pharmaceuticals (AMLX) announced an update on the ongoing review of its Marketing Authorization Application (MAA) for AMX0035 (taurursodiol]) regarding the treatment of adults with amyotrophic lateral sclerosis (ALS) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Following an oral explanation held at the . . . This content is for paid subscribers. Please click here …

Inside the Revolution In the Treatments of Untreatable Diseases We are now living a great revolution in the treatment of diseases. We expect to discover novel treatments capable of defeating cancers’ resistance to therapeutics old and new. We are following on all the firms that are discovering the unknown about life-threatening diseases, including the genetically derived diseases or else. Yesterday, May 25, 2023, we studied …

Eye on Reata Pharmaceuticals Recently, Reata Pharmaceuticals (RETA) announced a setback of its chronic kidney disease product known as bardoxolone. The failure of this drug occurred after Reata's product Skyclaris was FDA approved for the treatment of Friedrich's ataxia (FA) - a rare genetic disease that causes difficulty walking, loss of sensation in the legs and arms . . . This content is for paid …

Intercept Pharmaceuticals  Intercept Pharmaceuticals Inc (ICPT) is a biopharmaceutical company focused on the development and commercialization of novel therapeutics aimed at treating progressive non-viral liver diseases, including primary biliary cholangitis (PBC), nonalcoholic steatohepatitis (NASH) and severe alcohol-associated hepatitis (sAH). On May 19, 2023, Intercept Pharmaceuticals, announced that NASDAQ has halted trading of the company’s common stock. Today’s Meeting Today, the U.S. FDA Gastrointestinal Drugs Advisory …

Prostate Cancer Prostate cancer is the most common type of cancer found in men, accounting for more than a quarter of all newly diagnosed cancer cases. Early detection is linked to improved survival rates, but typical screening recommendations — such as digital rectal exams or testing for the prostate specific antigen (PSA) biomarker — do not give the full picture. Halfway through 5-year study, ExoDx …

Celldex Therapeutics Announcement Celldex Therapeutics (CLDX) announced today preclinical data from its CDX-622 bispecific antibody with dual targeting of the alarmin TSLP and stem cell factor (SCF), also known as KIT receptor ligand, developed from its bispecific antibody platform. TSLP and SCF demonstrated their contribution to the pathophysiology of various inflammatory, fibrotic and allergic disorders. The data . . . This content is for paid …

Longeveron in the NEWS On May 9, 2023, Longeveron (LGVN) announced new long-term follow-up data from the Company’s ELPIS I trial of Lomecel-BTM for patients with hypoplastic left heart syndrome (HLHS). What is Hypoplastic Left Heart Syndrome HLHS is a rare and life-threatening congenital heart defect affecting approximately 1,000 babies per year. Infants born with HLHS have an underdeveloped or absent left ventricle. Current standard-of-care is …

CureVac B.V. CureVac (CVAC) is a global biopharmaceutical company in the field of messenger RNA (mRNA) technology with more than 20 years of expertise in developing, optimizing and manufacturing this versatile biological molecule for medical purposes. CureVac’s proprietary technology uses optimized mRNA as a data carrier to instruct the human body to produce its own proteins capable . . . This content is for paid …

Gilead Sciences CHMP Adopted Positive Opinion for Hepcludex® Today, May 4, 2023, Gilead Sciences (GILD) acknowledged that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Hepcludex® (bulevirtide) for adults having chronic HDV and compensated liver disease,  recommended granting full Marketing Authorization (MA) that . . . This content is for paid subscribers. …

May 01, 2023 (GLOBE NEWSWIRE) –Today, Intellia Therapeutics (NTLA) released its 2023 Corporate Responsibility report. The report provides a comprehensive update of the Company’s performance and progress across key Environmental, Social and Governance (ESG) areas of focus. John Leonard, M.D., Intellia President and Chief Executive Officer said, “As the leaders in a new era of medicine, we believe ESG principles support long-term value creation for …

 ImmunoGen Reports Recent Progress and First Quarter 2023 Financial Results  On April 28, 2023, ImmunoGen (IMGN) reviewed recent progress in the business and reported financial results for the quarter ended March 31, 2023.  Immunogen's drug ELAHERE® (mirvetuximab soravtansine-gyn) is a first-in-class antibody drug conjugate (ADC) comprising a folate receptor alpha-binding antibody, cleavable linker, and the maytansinoid . . . This content is for paid subscribers. Please …

Vertex Pharmaceuticals ORKAMBI Receives Positive CHMP Opinion Today, April 27, Vertex Pharmaceuticals  (VRTX) announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) embraced a positive opinion for the label extension of ORKAMBI® (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 . . . This content is for paid subscribers. Please click here to subscribe or here to log …

Atea Pharmaceuticals Receives Fast Track Designation for Bemnifosbuvir Atea Pharmaceuticals (AVIR) announced that the U.S. FDA has granted Fast Track designation to its product bemnifosbuvir for the treatment of resistant COVID-19.   Bemnifosbuvir is an oral, direct acting antiviral drug candidate being evaluated in the global Phase 3 SUNRISE-3 registrational trial for the treatment of COVID . . . This content is for paid subscribers. …

Apellis Pharmaceuticals Validation for Pegcetacoplan Apellis Pharmaceuticals (APLS) announced the receival of validation of its marketing applications by regulatory authorities in Canada, Australia, the United Kingdom, and Switzerland for intravitreal pegcetacoplan regarding the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The decisions by the local regulatory authorities in these four countries are . . . This content is for paid subscribers. Please …

Important News from CymaBay Therapeutics Primary Biliary Cholangitis Today, April 21, 2023, CymaBay Therapeutics (CBAY) announced that results from the ENHANCE phase 3 global study evaluating seladelpar for Primary Biliary Cholangitis (PBC) have been published in Hepatology. The authors . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

From Novartis on April 20, 2023 The ALITHIOS open-label extension study demonstrated that continuous treatment with Kesimpta® (ofatumumab) for up to five years in relapsing multiple sclerosis (RMS) was associated with reduced risk of disability progression versus those who switched later from teriflunomide to Kesimpta. Outcomes related to both disability progression and brain volume change up to five years favored earlier initiation of Kesimpta in people …

News from Innoviva That We Consider Positively Impacting and Extremely Important Innoviva, Inc. (INVA) (Innoviva), announced that the FDA Antmicrobial Drugs Advisory Committee (AMDAC) has unanimously voted 12-0 in favor of approval of sulbactam-durlobactam based on a favorable benefit-risk assessment for the treatment of adults with hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused . . . This content is for paid subscribers. Please click here …

Merck in the NEWS Merck (MRK) announced that the U.S. FDA has accepted for review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA (pembrolizumab) in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of HER2 negative patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma. The American Agency's acceptance of . . . This content is …

Sanofi Commercial Control of Beyfortus Against RSV In the morning, we promised to tell our readers how AstraZeneca's (AZN), Sobi, and Sanofi (

Regenxbio in the NEWS Today, the U.S. FDA granted Fast Track designation for Regenxbio (RGNX) Gene therapy RGX-202 a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne). Therapies granted Fast Track designation are given the opportunity for more frequent interactions with the FDA and may qualify for priority review. The FDA has granted RGX-202 . . . This content is for paid …