Gilead Sciences’ (GILD) and Galapagos’ (GLPG) product filgotinib – a highly selective JAK1 inhibitor has met the primary efficacy endpoint in Phase 2 TORTUGA trial in adults with moderately to severely active ankylosing spondylitis (AS). Patients treated with filgotinib achieved significantly greater improvements compared to those who took a placebo. The results came from the Phase 2 of the TORTUGA – a multi-center, randomized, double-blind, …

We reiterate that undeniably outstanding news about gene therapy is taking time to penetrate investors’ ears. They are still falling victim to bad rules of thumb and critics’ magnifications of the negative impact of some stocks’ dilution when companies with superior programs try to finance through a public offering. The stocks’ punishments following financing is not a rule to apply to all firms, especially those …

Before we sell PTC Therapeutics (PTCT), after the stock had reached and surpassed our latest and highest 2018 target, the firm did the right thing that we expected was good enough to encourage investment in this firm.  The good thing is acquiring Agilis Biotherapeutics – a small firm that succeeded in creating innovative gene therapy programs for rare genetic disorders that affect the central nervous …

When the Goal is Reaching into the Future, Pessimists Have No Place on the Ship The revolution in the biotech sector has proven to be moving fast towards reaching goals that were impossible to imagine a couple of years ago that they could be attainable. This is not true only regarding lay investors, or desperate patients, but also regarding the most professional clinicians, researchers and …

Celyad (CYAD) is an immuno-oncology firm, which created CAR T CELL treatments for cancers, including  solid tumors. Celyad announced its consolidated financial results for the six-month period ending 30 June 2018. Celyad’s report is endorsed by Europe. Dr. Christian Homsy, Celyad’s CEO started by expressing the firm’s great enthusiasm towards the progress it has made in the first half of 2018, which include a significant advancement of …

Aerie Pharmaceuticals (AERI) announced results from a double-masked, randomized, placebo-controlled study designed to evaluate the effect of its glaucoma product Rhopressa® (netarsudil ophthalmic solution) 0.02% on aqueous humor dynamics in patients with open-angle glaucoma or ocular hypertension. This study is the first performed on glaucoma patients to confirm that Rhopressa® lowers intraocular pressure (IOP) primarily through trabecular meshwork outflow facility.  In the trials, the enrolled patients …

Regeneron Pharmaceuticals (REGN) and bluebird bio, Inc. (BLUE) announced a collaboration to apply their respective technology platforms to the discovery, development, and commercialization of novel immune cell therapies for cancer. The collaborators will specifically leverage Regeneron’s VelociSuite® platform technologies for the discovery of human antibodies as well as T cell receptors (TCRs) directed against tumor-specific proteins and peptides, and bluebird bio will contribute its field-leading expertise in gene transfer and …

The Future is Starting Now Recognizing that the future has already started now is important as it makes analysts use different criteria for the fair evaluation of the future growth of biotechnology startups and revenue-generating companies. This is our main topic the Prohost Letter that we are working diligently working on posting at any time now. This is extremely important to know before the hype …

The Committee for Medicinal Products for Human Use (CHMP) announced a Positive Opinion recommending marketing authorization of Alnylam’s (ALNY) RNAi product Onpattro™ (patisiran). The product is designed to treat hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy. The CHMP opinion is based on conclusive positive results from APOLLO Phase 3 study on patients with hATTR amyloidosis. The results were published in The New England Journal …

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 …

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 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 …

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 …

– 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 …

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 …

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 …

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 …

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 …

The U.S. Food and Drug Administration (FDA) has approved Portola’s (PTLA) drug Andexxa®, the first and only antidote indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. Commenting on the approval, Stuart J. Connolly, M.D., ANNEXA-4 Executive Committee chairman and professor in the Department of Medicine of the Faculty of Health Sciences at McMaster University in Hamilton, Ontario said, “Today’s approval represents …