Stock Market Plummet and Alnylam Pharmaceuticals Good News Today, the stock market is plummeting. In fact, most stocks have been falling since early morning. The reason has nothing to do with the company’s activities. The best companies with the best science and scientists are sinking into the bottom of the deepest ocean today. Even though the Stock Market is surrounded by negativity, we will still …

Wave Life Sciences On March 26, 2025, Wave Life Sciences (WVE) announced positive data from the Phase 2 FORWARD-53 trial of WVE-N531, an exon skipping oligonucleotide being investigated in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. FORWARD-53 Trial demonstrates sustained and industry-leading exon . . . This content is for paid subscribers. Please click here to subscribe or here to log …

PTC Therapeutics Inc On March 20, 2025, PTC Therapeutics, Inc. (PTCT) shared new data from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the full spectrum of phenylketonuria (PKU . . . This content is for paid …

AstraZeneca and Daiichi Sankyo AstraZeneca (AZN) and Daiichi Sankyo’s (DSKYF) (trastuzumab deruxtecan) has been recommended for approval in . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Sanofi New Data Paris, February 28, 2025, Sanofi (SNY) will present 26 abstracts, including one late-breaking presentation and five additional oral presentations, on approved and investigational medicines at the American Academy of Dermatology (AAD) Annual Meeting in Orlando, FL, US, from March 7 to 11 . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Verona Pharma Conference Announcements Verona Pharma (VRNA) announces that senior management will present a company overview at the following conferences in March 2025: TD Cowen 45th Annual Health Care Conference Date: Monday, March 3, 2025: Time: 11:10 a.m. ET / 4:10 p.m. GMT Location: Boston, MA Leerink Partners 2025 Global Healthcare . . . This content is for paid subscribers. Please click here to subscribe or …

Gilead Sciences On February 20, 2025, Gilead Sciences (GILD) announced that the European Commission (EC) granted conditional marketing authorization to seladelpar in combination with ursodeoxycholic acid (UDCA) for the treatment of Primary Biliary Cholangitis (PBC) in adults who have inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA. Now approved, Seladelpar (an orphan-designated . . . This content is for …

Boehringer Ingelheim Today, Boehringer Ingelheim, a private Company announced that the FIBRONEER™-ILD trial met its primary endpoint, which was the absolute change from baseline in FVC (mL) at week 52 versus placebo. FVC is a measure of lung function.  Initial data readouts of the FIBRONEER™-trials support a generally consistent safety and tolerability profile when compared to the Phase II IPF study, with overall adverse events comparable to …

Vertex Pharmaceuticals Yesterday, January 30, 2025, Vertex Pharmaceuticals (VRTX) announced FDA approval of journavx™(suzetrigine), a First-in-Class treatment for adults with moderate-to-severe acute pain. Today, Vertex Announced the CASGEVY® Reimbursement Agreement for treating Sickle Cell Disease in England. We start with the Approval of the JOURNAVX™ News Yesterday, after the stock market closed, Vertex Pharmaceuticals announced that the  U . . . This content is for paid …

AstraZeneca FDA Approval On January 27, 2025, Daiichi Sankyo (TSE: 4568) and AstraZeneca (AZN) product ENHERTU® (fam-trastuzumab deruxtecan-nxki) was approved in the U.S. for the treatment of adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 low or HER2 ultralow breast cancer, as determined by an FDA-approved test, that has progressed on one . . . This content is for paid subscribers. Please click here …

Verastem Oncology in the NEWS On December 30, 2024, Verastem Oncology (VSTM) announced that the U.S. FDA has accepted for review the New Drug Application (NDA) under the accelerated approval pathway for avutometinib. The drug is in combination with defactinib, an oral FAK inhibitor, for the treatment of adult patients with recurrent low-grade serous ovarian cancer (LGSOC . . . This content is for paid …

Vertex Pharmaceuticals Vertex Pharmaceuticals (VRTX) announced a United States  FDA has approved ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), a once-daily triple combination Cystic Fibrosis transmembrane conductance regulator (CFTR) modulator for the treatment of cystic fibrosis (CF) in people 6 years and . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Broadcom Inc Q4 Financial Results for Broadcom Inc (AVGO) reported financial results for its fourth quarter and fiscal year ended November 3, 2024, provided guidance for its first quarter of fiscal year 2025, and announced its quarterly dividend.  From Broadcom Hock Tan, President and CEO of Broadcom Inc. Said, "Broadcom's fiscal year 2024 revenue grew 44% year-over-year . . . This content is for paid subscribers. …

Johnson & Johnson On December 9, 2024, Johnson & Johnson (JNJ) announced new results from the Phase 3 CARTITUDE-4 study demonstrating that a single infusion of CARVYKTI®  has significantly increased minimal residual disease (MRD) negativity rates in patients with relapsed or refractory multiple myeloma (RRMM) patients who were lenalidomide-refractory and had received one to three prior lines . . . This content is for paid subscribers. …

Intra-Cellular Therapies in the NEWS On December 3, 2024, Intra-Cellular Therapies (ITCI) announced the submission of the supplemental New Drug Application (sNDA) to the United States FDA to approve CAPLYTA as adjunctive therapy to antidepressants for the treatment of Major Depressive Disorder (MDD) in adults. Intra-Cellular Therapies is . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Gilead Sciences and Tubulis Agreement Gilead Sciences (GILD) and Tubulis announced that they entered into an exclusive option and license agreement to discover and develop an antibody-drug conjugate (ADC) against a solid tumor target. Gilead will gain access to Tubulis’ proprietary Tubutecan and Alco5 platforms. The companies will collaborate to . . . This content is for paid subscribers. Please click here to subscribe or here to …

AstraZeneca in the NEWS AstraZeneca (AZN) Phase III trial CAPItello-281, which combines Truqap (capivasertib), abiraterone, and androgen deprivation therapy (ADT), demonstrates statistically significant and clinically meaningful improvement in the primary endpoint of radiographic progression-free survival (rPFS) vs. abiraterone and ADT with placebo in patients with PTEN-deficient de novo metastatic hormone-sensitive prostate cancer . . . This content is for paid subscribers. Please click here to subscribe or here to …

BridgeBio Pharma in the NEWS BridgeBio Pharma (BBIO) focused on genetic diseases, announced that the United States FDA approved Attruby™ (acoramidis), an orally administered near-complete stabilizer of Transthyretin (TTR) for the treatment of adults with ATTR-CM. The aim is to reduce cardiovascular death and cardiovascular-related hospitalization. The approval is based on positive results seen in the ATTRibute-CM Phase 3 . . . This content is …

Viking Therapeutics Announcement On Nov. 19, 2024, Viking Therapeutics, Inc. (VKTX) announced that the final results from the company's Phase 2b clinical trial of VK2809, the firm’s novel liver-selective thyroid hormone receptor beta agonist, in patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH) referred to also as metabolic dysfunction associated steatohepatitis (MASH) were highlighted in an oral late . . . This content is for paid subscribers. …

Intellia Therapeutics Announcement On Nov. 16, 2024,  Intellia Therapeutics (NTLA), a clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. Nex-z is an investigational in vivo CRISPR-based . . . This content is for paid subscribers. Please click here to …