Yesterday’s news unveiled that PTC Therapeutics (PTCT) has entered into an agreement to acquire Agilis Biotherapeutics. The transaction was approved by the Boards of both companies. The acquisition Agilis Biotherapeutics is expected to bring to PTC Therapeutics an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system. It is supposed to add to PTC Therapeutics’ pipeline an advanced gene therapy candidate, …

Novartis’ (NVS) financials results exceeded analysts’ expectations. The firm’s research institutions’ strategy, well-designed structure, plans for innovation and accurate execution led to a wealthy and competitive pipeline with more than 200 projects in clinical development. Novartis is flying into the future finding its way to more effective treatments and possible cures. As a matter of fact, Novartis is at the end of the road towards …

A Miracle-Like Gene-Editing That People will Sooner or Later Recognize. The Wellcome Sanger Institute’s new findings about CRISPR/Cas9 gene editing is intended to lead to further improvement in the safety of the breakthrough CRISPR gene editing procedure before it is used in clinical practice; the information is not intended to condemn it. Unfortunately, innocent investors got panicky when they heard the Wellcome Sanger findings, which …

The BLA Includes Data from More Than 11,000 Patients Amgen (AMGN) and UCB (Euronext Brussels: UCB) resubmitted a Biologics License Application (BLA) to the U.S. FDA for the approval of their osteoporosis investigational monoclonal antibody product Evenity™ (romosozumab). The product is indicated for osteoporosis in postmenopausal women at high risk for fracture.The drug increases bone formation and reduces bone resorption simultaneously to increase bone mineral …

The Week in Review #26 IN THE BIOTECH WORLD - RESEARCH - IMPROVING CRISPR GENE EDITING The problem cited with gene editing is that the editing may stay switched on after it succeeded in reaching its goals. To prevent further unwarranted mutations, scientists from the Bath and Cardiff University have invented a switch that aims at controlling protein expression in cultured cells and mouse embryos with the …

The New England Journal of Medicine (NEJM) published results from the CELESTIAL phase 3 pivotal trial of Exelixis (EXEL) product Cabometyx® (cabozantinib) in previously treated patients with advanced hepatocellular carcinoma (HCC). The data demonstrate that Cabometyx provided a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo. It is important to know that the clinical trial results formed the basis of regulatory …

Finally, we are hearing what we were constantly expecting to hear coming out of Compugen’s (CGEN) technological capability. Yesterday, the U.S. Food and Drug Administration (FDA) has cleared Bayer AG’s investigational new drug (IND) application for BAY 1905254, a first-in-class immuno- oncology therapeutic antibody targeting ILDR2. The target ILDR2 is a novel immune checkpoint protein discovered by Compugen through its predictive computational capabilities, which enable …

The Week in Review #25 When Stocks Plummet for No Relevant Reasons Sometimes investors need to know why some of their stocks plummet when no bad news is broadcast anywhere and when firms announce terrific news promising progressive growth. Yesterday was one of those days where reasons for stocks’ deviant, and probably unwarranted, negative performances must be identified. Let’s have a look at what happened during …

Health Canada Approves PrSYMDEKO™ For People Ages 12 and Older With Certain CFTR Gene Mutations Vertex (VRTX) announced that Health Canada approved SYMDEKO™ (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or who have one copy of the F508del mutation and a second mutation predicted …

The FDA approved Array BioPharma’s (ARRY) products Braftovi™ capsules in combination with Mektovi® tablets for unresectable or metastatic skin cancer melanoma with a BRAF or BRAFV mutation, as detected by an FDA-approved test. BRAFTOVI is not indicated for the treatment of patients with wild-type BRAF melanoma. The products, which represent a new standard of care for BRAF-mutant melanoma are now available to order through select specialty pharmacies in the U.S. market.  The …

SOPHIRIS BIO AND NANOBIOTIX SOPHIRIS BIO  Sophiris Bio (SPHS) has a product called Topsalysin (PRX302) in Phase 2 clinical trial for the treatment of localized prostate. The product is in Phase 3 clinical trial for lower urinary tract symptoms of benign prostatic hyperplasia (BPH). Topsalysin is a first-in-class, pore-forming protein activated only with the enzymatic action of PSA, which is found in high concentrations around …

The Week in Review #24 Biotech Stocks’ Performances, Products on the Move and Biotech Research Biotechnology stocks’ performance should be based on the firm’s intrinsic news that announces the power of their technologies and the successes or failures of their lead products and, to a lesser extent of their early-phase trials’ products. To be taken in consideration too are their financial statuses, especially of firms …

Incyte (INCY) announced positive topline results from its pivotal Phase 2 REACH1 trial evaluating ruxolitinib (Jakafi®) in combination with corticosteroids for patients with steroid-refractory acute graft-versus-host disease (GVHD). The study met its primary endpoint, with a 55% overall response rate (ORR) at Day 28. In addition, the best overall response rate (BORR), the number of patients achieving a response at any time point during the …

On May 25, in Prohost Letter Issue #421, we wrote about RegenxBio (RGNX), which from behind the scenes it created several gene therapy products that are being used by AveXis, a firm that was recently acquired by Novartis based on its successful pipeline products. In the article (please read it if you missed it and read it again if you forgot it, or were not …

CRISPR Therapeutics, Intellia Therapeutics, and Caribou Biosciences, Inc., announced the news about the granted U.S. Patent No. 10,000,772 (“the ‘772 patent”) by the U.S. Patent and Trademark Office (USPTO). This patent covers methods of using optimized guide RNA formats (including single guide and dual guide formats) in certain environments, including eukaryotic cells (such as human, animal and plant cells). The optimized formats modify the part of …

Sarepta Therapeutics (SRPT) has news from preliminary Duchenne muscular dystrophy trials. Although the number of recruited patients was only three, the strong positive data excited the media and Wall Street investors who rushed to buy the stock, making it add over $50 in today’s morning trading. The Story At the Company’s R&D Day, Jerry Mendell, M.D. of Nationwide Children’s Hospital presented positive preliminary results from Phase 1/2a …

The Week in Review #23  ASCO  2018 - Small  publicly-­traded  oncology  biotech  firms,  large  oncology  biotech  firms,  and  giant  biopharmaceutical  firms,  in  addition  to  some  private  biotech  companies,  all  presented  results  and  observations  about  their  cancer  drugs  at  the  2018  American  Society  of  Clinical  Oncology  (ASCO)   meeting.  Most  presentations  were  useful,...

The U.S. Food and Drug Administration (FDA) has approved Agilent Technologies’ (NYSE: A) Dako PD-L1 IHC 22C3 pharmDx assay for an expanded use. The expended use is expected to help physicians to identify cervical cancer patients who are most likely to benefit from treatment with Merck’s (MRK) checkpoint inhibitor lmmuno-oncology product Keytruda PD-L1 IHC 22C3 pharmDx assay was FDA approved for non-small cell lung cancer and subsequently expanded approval for gastric …

– Keytruda becomes the first anti-PD-1 therapy to be approved for PMBCL   – This is the 2nd indication for Keytruda for a hematologic malignancy. – The approval is based on tumor response rate and durability of response. The FDA granted Merck (MRK) approval for its immuno-oncology checkpoint inhibitor Keytruda® for adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or for those who relapsed after two …

Slashing of the CRISPR gene editing companies’ stocks, including CRISPR Therapeutics (CRSP), Editas (EDIT) and Intellia (NTLA) is caused by two studies published in Nature Medicine, which revealed that cells whose genes are edited by CRISPR/ Cas9 could develop tumors in treated patients. Nobody could blame those who sold the CRSPR gene editing stocks’ because, although many scientists believe that the cited cause can be overcome and others stated that …