Regenxbio Results from RGX-121 for MPSII Regenxbio (RGNX) announced interim data from Phase I/II trial of RGX-121 for Mucopolysaccharidosis Type II (MPS II). RGX-121 is designed to deliver the gene that encodes iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) using the AAV9 vector. About Mucopolysaccharidosis Type . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Amylyx Pharmaceuticals With the advancement of biological sciences, and increased information about the pathways of diseases, we are getting more interested in biotech firms that are focused on treating life-threatening diseases as the risk of failure is decreasing.  The successful outcome of trials would tremendously boost the values of these firms as we have witnessed in many small biotech firms that became large firms and …

From Amarin Amarin (AMRN) announced the U.S. FDA has approved a new indication and label expansion for Vascepa® (icosapent ethyl) capsules. According to Amarin’s press release, with this label expansion approval, Vascepa has become . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Exelixis Positive Results from IMspire 150 Exelixis (EXEL) announced positive results from IMspire 150; the phase 3 trial of atezolizumab (TECENTRIQ®), cobimetinib (COTELLIC®) and vemurafenib (ZELBORAF®) in people with previously untreated BRAF V600 mutation-positive advanced melanoma.  The IMspire150 study met its primary endpoint of progression-free survival (PFS). Adding atezolizumab to cobimetinib and vemurafenib helped reduce the risk of disease worsening or death, compared to placebo plus …

Ionis Pharmaceuticals Ionis Pharmaceuticals (IONS) announces a private placement of $507.5 million principal amount of 0.125% convertible senior notes due 2024. To read this news go to the original  content to download multimedia by clicking here.  During the announcement of 2019 third-quarter financial results the conference call started citing the firm's improved revenues and income. Revenues have increased by more than 50% to nearly $630 million and …

Nektar Therapeutics Presents NKTR-255 at ASH Nektar Therapeutics’ (NKTR) IL-15 agonist investigational candidate NKTR-255 presentations at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition have been promising. Positive data presented from Fred Hutchinson Cancer Research Center preclinical studies conducted in collaboration with researchers from the Dana-Farber Cancer Institute and more. NKTR-255 is currently . . . This content is for paid subscribers. …

Gilead’s CAR-T Product Yescarta Results Kite, a Gilead (GILD) subsidiary, announced new data from the ZUMA-1 trial of the CAR T Yescarta (axicabtagene ciloleucel) in adult patients with refractory large B-cell lymphoma. The data from the pivotal phase 2 study demonstrate that patients with refractory large B cell lymphoma were alive three years following a single infusion of Gilead’s CAR T immuno-oncology product Yescarta.  Analysis from …

Firstly, Forty-Seven and IVERIC bio  We remind that the two condemned stocks Forty-Seven (FTSV) and  IVERIC bio (

The Market May be Plummeting But Prohost Picks are Not Biotech stocks had a strong start, but the gains disappeared in many stocks as the market demonstrated bearishness following a long-lasting rally. Currently, during the strong economic conditions, commentators attribute the reasons for the market’s upside and downside moves to China’s positive, or negative ongoing negotiations rather than to fair correction of overbought or oversold …

Novartis to Acquire The Medicines Company Novartis (NVS) decided to buy The Medicines Company (MDCO) at $6.8 million. The Medicines Company’s shareholders will be getting $85 per share in cash  – a 45% premium to its unaffected share price and a 57% premium to its unaffected one-week average price. The Transaction is expected to be completed in the first quarter of 2020 The Medicines Company …

Alnylam Granted FDA Early Approval of GIVLAARI™ for AHP Alnylam’s (ALNY) stock outperformed as its subcutaneous injection GIVLAARI™ (givosiran) has been granted FDA early approval. GIVLAARI™ is created and developed for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, life-threatening attacks and, for some patients, chronic manifestations that demeans patients’ daily functioning and …

Vertex Pharmaceuticals and CRISPR Therapeutics Rallied Today Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. A patient with transfusion-dependent beta-thalassemia (TDT) received CTX001 in the first quarter of 2019. Data for this patient reflect nine months of safety and efficacy follow-up. …

PTC Therapeutics Risdiplam Results for SMA PTC Therapeutics (PTCT) has positive results from part 2 of the SUNFISH trial evaluating the safety and efficacy of the product risdiplam. The results demonstrate that the trial has met its primary endpoint of change from baseline after 1 year of treatment with risdiplam compared to placebo. The change was measured by the Motor . . . This content …

Reata Pharmaceuticals CARDINAL Study Met Primary and Key Secondary Endpoints Reata Pharmaceuticals (RETA) announced that the Phase 3 portion of the CARDINAL study of bardoxolone methyl (bardoxolone) in patients with chronic kidney disease (CKD) caused by Alport syndrome met its primary and key secondary endpoints.   This news is surely music in the ears of families with members . . . This content is for paid …

Exelixis and Ipsen Received Approval from Health Canada for CABOMETYX® From Exelixis (EXEL) we learned that its partner, Ipsen Biopharmaceuticals Canada, has received approval from Health Canada of CABOMETYX® (cabozantinib) tablets for the treatment of hepatocellular . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

FDA Approval for Celgene and Acceleron Drug REBLOZYL® The U.S. Food and Drug Administration (FDA) has approved Celgene’s (CELG) and Acceleron Pharma’s (XLRN) drug REBLOZYL® (luspatercept-aamt) for anemia only in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.  REBLOZYL® is the first and only FDA-approved erythroid maturation agent, representing a new class of therapy that works by regulating late-stage red blood cell maturation …

Exelixis and Daiichi Sankyo Partner to Prevent and Treat Diabetic Nephropathy Exelixis (EXEL) and Daiichi Sankyo (

Halozyme Therapeutics is Focusing on ENHANZE® Technology Halozyme Therapeutics (HALO) decides to focus on its ENHANZE® drug delivery technology, halt the development activities for PEGPH20 and close its oncology operations. These decisions were made after the failure of HALO-301 Phase 3 clinical study evaluating PEGPH20 as a first-line therapy for patients with metastatic pancreas cancer to reach the primary endpoint of overall survival. The PEGPH20 failure …

Karyopharm Therapeutics Third Quarter Financial Results Generated Revenues For the first time since its establishment Karyopharm Therapeutics (KPTI) financial results report revenues from a strong launch of its first approved product. Indeed, the firm’s third-quarter 2019 financial results are the first quarter in which Karyopharm generated revenues. In early July the U.S. FDA granted the company’s product Xpovio® (selinexor . . . This content is for …

Compugen FDA Approval for COM902 Compugen Announces FDA Clearance of IND Application for COM902 Phase 1 clinical study in patients with advanced malignancies expected to begin in early 2020 Clinical development of COM902 will enable testing the combination treatment with COM701, Compugen’s anti-PVRIG inhibitor  Compugen (CGEN) is a clinical-stage therapeutic discovery and development company utilizing its broadly applicable, predictive computational discovery platforms to identify novel …