Zolgensma Findings The FDA submission of the gene therapy product Zolgensma was based on positive findings from a trial of 15 babies treated with it. Recently; however, at the Orlando meeting of the Muscular Dystrophy Association Novartis (NVS) presented interim results for 22 babies with Type 1 SMA which is considered the . . . This content is for paid subscribers. Please click here to subscribe …

Novartis Has More Promising Data from Their Phase 3 Gene Therapy Trial with AveXis AveXis Inc., a Novartis (NVS) company, announced positive interim data from its Phase 3 STR1VE trial of Zolgensma® (onasemnogene abeparvovec-xio (AVXS-101) gene therapy in spinal muscular atrophy (SMA) Type 1. About Spinal Muscular Atrophy (SMA . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Amgen and UDB Granted FDA Approval of Evenity™ Amgen (AMGN) and UCB Pharmaceuticals (

The Amgen/Novartis Story In May 2018 Amgen (AMGN) & Novartis (

The Market is Outperforming Today. There is important news today about Novartis; part of which we are posting now. There is still more news related to Novartis as well as other biotech and biopharmaceutical companies. All will be in the upcoming Prohost Letter #430. We believe that the news we are posting will have a positive impact on the firms that announced them. Today’s news …

Following Seattle Genetics' (SGEN) and Astellas Pharma's announcement of positive results, from pivotal trial of the conjugated monoclonal antibody drug Enfortumab Vedotin in locally advanced or metastatic urothelial cancer, the stock experienced a selloff. Prohost Observations   We do not believe that there is anything wrong with the positive results coming out of  EV-201 trial investigating Enfortumab Vedotinfor locally advanced or metastatic urothelial cancer who have received previous …

The uncontrolled blowing toxic wind contaminates this country’s environment, it brings down the oversold scientifically solid and promising biotech stocks together with the undersold disappointing firms’ stocks. All we want is peace of mind, fairness and well being for America and the rest of the world. We are confident that this wish will materialize when the country’s leaders work together in harmony to make it …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

AGENUS Agenus (AGEN) announced the upcoming launch of its Biotech Electronic Security Token (BEST); the first digital security offering in healthcare. Tokenization enables qualified investors to directly invest in a single biotech product while preserving shareholder equity. Garo Armen, Chairman and CEO of Agenus said, "Today, by announcing the first of its kind asset backed digital security offering in healthcare, we open the doors for a transformative …