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 …

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 …

ASTRAZENECA AstraZeneca’s (AZN) Checkpoint Inhibitor Imfinzi significantly improved Overall Survival (OS) for Stage III Non-Small Cell Cancer Imfinzi (durvalumab) achieved positive overall survival (OS) results for the Phase III PACIFIC trial in patients with unresectable Stage III non-small cell lung cancer (NSCLC) whose cancer did not progress following platinum-based chemotherapy and radiation. An Independent Data Monitoring Committee concluded that the trial met the second of two primary …

Prohost Letter #421 PRIVATE  LESSONS - Stock prices of publicly-­traded biotech companies oscillate for the same reasons other industries’ stock prices swing all day long. The fluctuations are the outcome of investors’ and daily traders’ unrelenting buying and selling . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Cara Therapeutics (CARA) develops novel and proprietary class of product candidates, led by Korsuva (CR845/difelikefalin) injection. This product is a first-in-class peripheral kappa opioid receptor (KOR) agonist designed to alleviate pruritus (itching) and pain.  In Phase 2 trials, Korsuva injection demonstrated statistically significant reductions in itch intensity and concomitant improvement in the quality of life in hemodialysis patients with moderate-to-severe chronic kidney disease-associated pruritus (CKD-aP). Additionally, Korsuva has demonstrated statistically …

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 …

The U.S. Food and Drug Administration (FDA) approved Amgen’s (AMGN) new once a month drug Aimovig™ (erenumab-aooe) for the prevention and treatment of migraine in adults. Aimovig is the first and only FDA-approved treatment specifically developed to prevent migraine. The drug blocks calcitonin gene-related peptide receptor (CGRP-R) believed to play a critical role in the occurrence of migraine. The drug will be marketed by Amgen and Novartis (NVS).  Aimovig 70 mg is …

Exelixis (EXEL) announced that its partner Ipsen received approval from the European Commission (EC) for CABOMETYX®(cabozantinib) 20 mg, 40 mg and 60 mg for the first-line treatment of adults with intermediate or poor-risk advanced renal cell carcinoma (RCC) in the European Union. Under the terms of the Collaboration and License Agreement with Ipsen, Exelixis will receive a milestone payment of $50 million for the EC approval, of which approximately $46 million was recognized as collaboration revenue in the first quarter …

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 …

Ionis Pharmaceuticals (IONS) and Akcea Therapeutics (AKCA), an affiliate of Ionis Pharmaceuticals, announced that the Advisory Committee of the U.S. Food and Drug Administration’s (FDA) Division of Metabolism and Endocrinology Products voted 12-8 in support of approving the firms’ product WAYLIVRATM (volanesorsen) for familial chylomicronemia syndrome (FCS). The PDUFA date for the FDA review and ruling is August 30, 2018. WAYLIVRA, potentially the first and only treatment for people living with this serious and …

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 …

The Week in Review #22 What Went Right or Wrong in the Biotech Sector During the Departing Week? - A lot happened in the biotech sector during the week, which departed at midnight. Topping the list of the week’s events was the approval of Portola’s (PTLA) product  Andexxa . . . This content is for paid subscribers. Please click here to subscribe or here to log in.