While waiting for Mueller’s report we stopped speculating on the stocks in what we saw as an unreadable market. Following the delivery of the Mueller report we hesitated to prophesy as we waited to see how the market would perform and the reasons for its performance. We usually try to speculate about the performance of biotech firms’ stocks based on their scientific capabilities, including their …

Governor Cooper to cut ribbon on bluebird bio facility Bluebird bio (BLUE) announced today, in its press release, the official opening of its first wholly owned manufacturing facility in Durham, N.C. The new facility will produce the lentiviral vector for the company’s investigational gene and cell therapies, including bb2121 and bb21217 for the treatment of multiple myeloma, and potentially LentiGlobin™ for transfusion-dependent β-thalassemia (TDT) and …

Biogen’s (BIIB) decision to discontinue the global Phase 3 trials, ENGAGE and EMERGE which is designed to evaluate the efficacy and safety of its product aducanumab in patients with Alzheimer’s disease, has caused an exaggerated selloff of the stock. The failure to treat Alzheimer’s disease is well explained by Michel Vounatsos, Biogen’s Chief Executive Officer, who stated that the failure of aducanumab drug confirms the …

News We Loved to Hear  The First and Only Approval of a Specific Product for Postpartum Depression ZULRESSOTM The FDA granted approval of Sage Therapeutics’ (SAGE) ZULRESSOTM (brexanolone) injection. A product for the treatment of the condition known as postpartum depression (PPD). This makes Zulresso the first and only medicine specifically approved by the FDA to treat PPD; the most common medical complication of childbirth. The product …

Atossa Genetics (ATOS) announced that the FDA has issued a “Safe to Proceed” letter under their “expanded access” program permitting the use of Atossa’s oral Endoxifen as a post-mastectomy treatment in a pre-menopausal, estrogen-receptor positive (ER+) breast cancer patient. The patient completed a 3-week course of Atossa’s oral Endoxifen prior to her surgery under an FDA-approved expanded access program. Atossa’s stock rallied from under $2 to …

We love to read news about gene therapy as it is the only possibility for curing diseases that no known treatment can halt or delay their progression. The attempts to use gene therapy failed ambitious researchers for several decades before the revolution that led to understanding the genome and even decades  more after the publishing of the genomic map that actually started the real revolution. …

Thanks to abundant cabozantinib sales’ revenues Exelixis (EXEL) is advancing a new generation of its medicines with its new product XL092 entering clinical trials. The drug is a next-generation oral tyrosine kinase inhibitor that targets VEGF receptors & MET and was the subject of an active Investigational New Drug (IND) application the firm had submitted to the U.S. Food and Drug Administration (FDA) in December …

CRISPR Therapeutics' (CRSP) stock added around $8 today while reporting financial results for the fourth quarter and full year ended December 31, 2018. Here is what Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics, has stated,“This past year was truly transformational for CRISPR Therapeutics as we achieved milestones across our key programs in β-thalassemia, sickle cell disease and immuno-oncology. We’re pleased with the progress we’ve made in …

We congratulate our subscribers who kept Spark Therapeutics (ONCE) in spite of the stock’s kind of paralysis for no serious reasons. Spark, a fully integrated, commercial gene therapy company, announced today that it has entered into a definitive merger agreement with Roche (RHHBY). The firm is to fully acquire Spark Therapeutics at a price of $114.50 per share, in an all-cash transaction. This move represents  a total equity value …

Cytokinetics’ (CYTK) investigational product omecamtiv mecarbil, the firm’s potential treatment for heart failure with reduced ejection fraction, is advancing in a late phase clinical trial. Cytokinetics is conducting METEORIC-HF clinical trial, in collaboration with Amgen (AMGN), with funding and strategic support from Servier. The trial aims at evaluating the effect of omecamtiv mecarbil compared to placebo on exercise capacity as determined by cardiopulmonary exercise testing (CPET) in patients …

Keytruda and Cabometyx Are Both Winners Keytruda failed to meet co-primary endpoints in Pivotal Phase 3 trial The pivotal Phase 3 KEYNOTE-240 trial evaluating Keytruda, Merck’s (MRK) checkpoint inhibitor anti-PD-1 therapy plus best supportive care, for the treatment of patients with advanced hepatocellular carcinoma (HCC) who were previously treated with systemic therapy, did not meet its co-primary endpoints of overall survival (OS) and progression-free survival …

Today, at the 2019 American Society of Clinical Oncology (ASCO) Genitourinary Cancers Symposium (GU) held at Moscone West in San Francisco, CA, Nektar (NKTR) will present preliminary efficacy, safety and immune monitoring data from the ongoing metastatic urothelial carcinoma (mUC) patient cohort in the PIVOT-02 study evaluating NKTR-214 in combination with Bristol-Myers immunotherapy product OPDIVO (nivolumab). NKTR-214 is a CD122-biased agonist designed to provide sustained …

RegenxBio (RGNX), a clinical-stage gene therapy firm with a proprietary NAV® Technology Platform, will host an analyst and investor event focused on its ophthalmology program; including its lead product candidate RGX-314, on Thursday, February 21, 2019, from 9:00 a.m. to 12:00 p.m. EST in New York City. A live webcast will be available on the company’s website. The agenda will include discussion on RegenxBio’s NAV® Technology Platform; a review of the market landscape and unmet …

Exelixis’ (EXEL) financial results for the fourth quarter and full year 2018 are as inspiring as they are outstanding compared to many other small biotech firms that succeeded in putting products on the market. Revenues  Exelixis total revenues for the quarter ended December 31, 2018 were $228.6 million, compared to $120.1 million for the comparable period in 2017. Total revenues for the year ended December 31, 2018 were $853.8 million, compared to $452.5 million for the comparable period in 2017. The …

Portola Pharmaceuticals (PTLA) announced full results from ANNEXA-4, Phase 3b/4 trial of its Factor Xa inhibitor antidote Andexxa®  in patients experiencing acute major bleeding while taking a Factor Xa inhibitor. The data were presented at the International Stroke Conference 2019 and published simultaneously online by The New England Journal of Medicine (NEJM).  Full data from 352 patients showed that Andexxa rapidly, and significantly, reversed anti-Factor Xa activity when administered as a bolus and sustained …

The United States FDA granted Breakthrough Therapy Designation to AMT-061. The European Medicines Agency gave it access to the Priority Medicine (PRIME) regulatory initiative. Gene therapy firm, uniQure (QURE), announced updated clinical data in patients treated in the ongoing Phase 2b study of AMT-061; an investigational AAV5-based gene therapy containing a patent-protected  FIX9-Padua (Factor 9) for the treatment of patients with severe and moderately severe hemophilia B. The announced data, which …

ImmunoGen & Biogen News About ImmunoGen ImmunoGen (IMGN) is a creator and developer of antibody-drug conjugates (ADCs) aimed at improving cancer treatments. ImmunoGen aims to disrupt the progression of cancer through its conjugated targeted monoclonal antibodies. ImmunoGen’s lead ADC product candidate mirvetuximab soravtansine is in a Phase 3 study for folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer, and in Phase 1b/2 trial in combination regimens.

Halozyme Therapeutics (HALO) and argenx (ARGX) announced a global collaboration and license agreement offering argenx the right to use Halozyme's ENHANZE® drug delivery technology to develop multiple subcutaneous product formulations for current or future argenx product candidates. The agreement provides argenx exclusive access to ENHANZE® for any product targeting the human neonatal Fc receptor, FcRn, including argenx's lead asset efgartigimod (ARGX-113) and up to two additional targets, potentially shortening …

FDA Granted Rare Pediatric Disease Designation for RegenxBio’s Product RGX-181 for CLN2 RegenxBio RegenxBio (RGNX), a development-stage firm that focuses on designing and developing gene therapy based on its proprietary NAV® Technology Platform, announced that the U.S. FDA granted Rare Pediatric Disease Designation to its RGX-181 therapy. RegenxBio is a leading clinical-stage biotechnology company that promises to cure genetic-derived diseases with its gene therapy. RegenxBio’s NAV Technology Platform is …

Merck (MRK) announced its Q4 2018 financial results. The firm outperformed itself and delighted us with regard to picking its stock on time. Merck compensated those who bet on it the day it was added to the Prohost Portfolio that comprises only the biotech and bio-pharmaceutical companies with solid scientific fundamentals. The press release issued regarding Merck's financial results started with Kenneth C. Frazier, Chairman and …