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 …

Prohost Letter #422 SUMMARY OF THE PAST ISSUE ARTICLE - REVISITING PROMISING FIRMS (PART1) - The firms we tackled in the past issue comprised: 1. Firms we liked and added to our portfolio. 2. Firms we posted their good news, but did not select... 

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 …