Category: Today’s Highlights

Vertex Stock Soars Pre-Market Announcing Positive Results from Phase 2 Study of VX-548 for the Treatment of Painful Diabetic Peripheral Neuropathy

Vertex Pharmaceuticals in the NEWS Today, December 13, 2023, Vertex  Pharmaceuticals (VRTX) announced positive results from its Phase 2 dose-ranging study of the selective NaV1.8 inhibitor VX-548 in people with painful diabetic peripheral neuropathy (DPN). Treatment with all . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Roche Announces Positive Phase III Results for Inavolisib Combination in Advanced Hormone Receptor-Positive, HER2-Negative Breast Cancer with a PIK3CA Mutation

Roche in the NEWS From Basel, today, December 5, 2023 - Roche (RHHBY) announced positive results from the Phase III INAVO120 study of the investigational therapy, inavolisib, in combination with palbociclib (Ibrance®) and fulvestrant as a potential first-line treatment option for people with PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, endocrine-resistant, locally advanced or metastatic breast . . . This content is for paid subscribers. Please click here to …

AbbVie Inc to Acquire ImmunoGen

AbbVie Inc Acquisition of Immunogen in the News AbbVie Inc. (ABBV) and ImmunoGen (IMGN) today announced a definitive agreement under which AbbVie will acquire . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Immunovant Inc: Positive IMVT-1402 Results that Would Confirm Best-in-Class Potential in Treating Autoimmune Diseases

Immunovant Inc in the NEWS Immunovant, Inc. (IMVT) announced initial data from 600 mg MAD cohort of a Phase 1 clinical trial of its product IMVT-1402 in healthy adults. The Phase 1 clinical trial is a randomized, double-blind, placebo-controlled ascending dose study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of IMVT . . . This content is for paid subscribers. Please click here to subscribe …

Anavex Announced US Patent Office Granted a Patent No. 11,813.242 Entitled A273 a Therapeutic for Insomnia, Anxiety and Agitation

Anavex Life Sciences Granted US Patent Anavex Life Sciences (AVXL) – a development-stage biopharmaceutical company announced that the United States Patent and Trademark Office (USPTO) granted U.S. Patent No. 11,813,242 entitled “A273 as a therapeutic for insomnia, anxiety, and agitation.  This patent expands Anavex’s existing patent coverage of ANAVEX®273 (blarcamesine), including U.S. Patent No. 11 . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vertex Pharmaceuticals and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY

Vertex Pharmaceuticals and CRISPR Therapeutics Announce Authorization of CASGEVY Conditional Approval for Both Sickle Cell Disease and Transfusion Dependent Beta Thalassemia Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) today announced that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) granted them conditional marketing authorization for CASGEVYTM (exagamglogene autotemcel ), a CRISPR/Cas9 gene . . . This content is for paid subscribers. Please …

CRISPR Therapeutics Announces Preclinical Data at the American Heart Association Scientific Sessions 2023

CRISPR Therapeutics Data Presentation Announcement CRISPR Therapeutics (CRSP) today announced preclinical data from the Company’s investigational programs for the treatment of cardiovascular disease at the American Heart Association (AHA) Scientific Sessions 2023. The data will be presented on Saturday, November 11, 2023, in two oral sessions, entitled: "CTX310: An Investigational in vivo CRISPR-Based Therapy Efficiently and Durably Reduces ANGPTL3 Protein . . . This content …

ImmunoGen Reports Recent Progress and Third Quarter 2023 Financial Results

ImmunoGen in the NEWS Immunogen (IMGN) - a developer of  antibody-drug conjugates (ADCs) for cancer treatment , today reviewed recent progress in the business and reported financial results for the quarter ended September 30, 2023. From ImmunoGen Mark Enyedy, ImmunoGen’s President and Chief Executive Officer said, “Building on the momentum generated in the first half of 2023, we delivered a . . . This content …

Good News for CRISPR Therapeutics and Vertex Pharmaceuticals

CRISPR Therapeutics and Vertex Pharmaceuticals NEWS CRISPR Therapeutics (CRSP) announced the completion of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee meeting for exagamglogene autotemcel (exa-cel) for the treatment of Sickle Cell Disease (SCD) in people ages 12 and older with recurrent vaso-occlusive crises (VOCs). Exa . . . This content is for paid subscribers. Please click here to subscribe …

Intellia Therapeutics: FDA Clearance of its Systemically Administered NDA of NTLA-2001 in Global Phase 3 Trial

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.

Akero Therapeutics’ October 10, 2023 Press Release

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)

Neurocrine Biosciences Announced Positive Results from Phase 3 CAHtalyst™ Study

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 . . . …

Pliant Therapeutics Announces Positive Safety and Exploratory Efficacy Data from Phase 2a INTEGRIS-PSC Clinical Trial of Bexotegrast

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 …

Reata Stock Holders Approved the Firm’s Merger with Biogen

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 …

Orchard Therapeutics: FDA Acceptance of BLA Application for Product OTL-200 for the Treatment of Metachromatic Leukodystrophy

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: Positive Top-Line Data from Phase 3 Study of Crinecerfont in Adults for the Treatment of Congenital Adrenal Hyperplasia

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 Investigational Product Met Primary and Secondary Endpoints in Phase 3 Treatment of Acromegaly 

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 …