AbbVie (NYSE: ABBV), in cooperation with Neurocrine Biosciences (NBIX), announced that the United States FDA approved Orilissa™ (elagolix) under priority review for women with moderate to severe endometriosis pain. Orilissa represents the first FDA-approved oral treatment for moderate to severe endometriosis pain in over a decade. The drug is expected to be available in U.S. retail pharmacies in early August 2018. Endometriosis is a condition where …

The good news from Biogen’s (BIIB) Q2 exciting financial results comprised impressive news for Ionis (IONS) from the commercialization of Ionis’ approved product, Spinraza for Spinal muscular atrophy (SMA).  Biogen is also responsible for the commercialization of Ionis’ products Tegsedi™ (inotersen) and Waylivra™ (volanesorsen) – two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. In the second quarter of Biogen’s 2018 …

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, …

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 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 …

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 …

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 …

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 …

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 …

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 …

Array BioPharma (ARRY) announced updated results from the Phase 3 COLUMBUS trial in BRAF-mutant advanced melanoma. The results showed that the median overall survival (mOS) was 33.6 months for patients treated with the combination of encorafenib and binimetinib compared to 16.9 months for patients treated with vemurafenib as a monotherapy. The combination reduced the risk of death compared to treatment with vemurafenib alone. The results, which was presented at the American Society …

The real news coming from the presentation at ASCO related to the combination of Nektar Therapeutics (NKTR) and Bristol-Myers Squibb (BMY) was viewed as positive by many analysts and investor until a sell-side negative article led to an unwarranted giant selloff of the stock. Last Week Nektar Stock gained $10 dollars over news that the firm has already submitted a New Drug Application (NDA) to the FDA for NKTR-181, which is …

CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX) announced that The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND. The IND was submitted to the FDA in April to support the planned initiation of a Phase …

The U.S. Food and Drug Administration (FDA) has accepted for filing Exelixis’ (EXEL) supplemental New Drug Application (sNDA) for Cabometyx® (cabozantinib) tablets for patients with previously treated advanced hepatocellular carcinoma (HCC). The FDA has determined that the application is sufficiently complete to permit a substantive review. The filing has been assigned a Prescription Drug User Fee Act (PDUFA) action date of January 14, 2019. Commenting on the news, Gisela Schwab, M.D., President, Product Development and …

Cidara Therapeutics (CDTX), a biotechnology company developing novel anti-infective products, including immunotherapies, announced that the U.S. National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH), awarded it and Rutgers University a five-year, $5.5 million partnership grant.     The grant aimed at funding the continued research and development of Cidara’s innovative Cloudbreak™antibody-drug conjugate (ADC) platform to identify novel immunotherapy agents for the treatment and prevention of serious and life-threatening multi-drug resistant (MDR) Gram …

Based in Boston, MA, Rhythm Pharmaceuticals (RYTM) – is a company focused on discovering and developing treatments for rare genetic disorders of obesity. It is currently evaluating the efficacy and safety of setmelanotide, its first-in-class melanocortin-4 receptor (MC4R) agonist, in Phase 3 studies in patients with pro-opiomelanocortin (POMC) deficiency obesity (which includes deficiencies in both the POMC and PCSK1 genes) and Leptin receptor (LEPR) deficiency obesity. Observing Rhythm’s research and trials …

SPARK THERAPEUTICS, SANGAMO THERAPEUTICS AND SALK INSTITUTE CURING HEMOPHILIA?   Hemophilia is an inherited bleeding disorder that causes abnormal or exaggerated bleeding and poor blood clotting. Hemophilia A and Hemophilia B are inherited in an X-linked recessive genetic pattern, so males are commonly affected while females are usually carriers of the disease. Hemophilia A is caused by clotting Factor VIII deficiency and hemophilia B (Christmas disease) is caused by Factor IX deficiency. A …