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.

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 …

It is obvious that Exelixis’ (EXEL) first quarter results do not justify the selling of EXEL during the whole quarter, but rather condemn it. It is an undeniable fact that Exelixis is doing a great job squeezing the best out of its approved drugs while conducting persistent research and development regarding its investigational pipeline products. The unveiled facts comprise the following: – Strong commercial performance for CABOMETYX in advanced renal cell …

Following the news announcing that Amgen’s (AMGN) drug KYPROLIS® (carfilzomib) extended the lives of patients with relapsed or refractory multiple myeloma in combination with lenalidomide + dexamethasone, we got news from a small company called Karyopharm Therapeutics (KPTI) announcing positive news from a trial with its product selinexor in multiple myeloma. The news announced that Karyopharm’s oral selective inhibitor of nuclear export (SINE) compound selinexor demonstrated positive results from the Phase 2b STORM study …

A couple of days following the news that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on Amgen’s (AMGN) product Prolia® (denosumab) for the treatment of bone loss associated with long-term systemic glucocorticoid therapy, the same Committee recommended a label variation for KYPROLIS® (carfilzomib) with an addition that includes the final overall survival (OS) data from the Phase 3 ASPIRE trial. In the ASPIRE trial, KYPROLIS …

The Week in Review #21 Part 2 THE  MARKET (2) - Our remarks in the past Week in Review included our confidence that the attempts by negative investors to drive the market crazy will not deprive the market from its innate capability to sense and react to the authentic factors it usually reacts to . . . This content is for paid subscribers. Please click here to …

The Independent Data Monitoring Committee (IDMC) decided that ImmunoGen (IMGN) Phase 3 registration trial evaluating mirvetuximab soravtansine as a single-agent therapy for platinum-resistant ovarian cancer will continue as planned without modification. ImmunoGen has completed full enrollment of the trial two months ahead of schedule and expects top-line results from FORWARD I during the first half of 2019. Enrollment was initially planned to be completed by the end of June. FORWARD I is …

NEKTAR Nektar Therapeutics (NKTR) and Takeda Pharmaceutical (TKPYY) are collaborating to evaluate Nektar’s investigational medicine, NKTR-214, with Takeda’s investigational medicine, TAK-659, as a potential combination for multiple cancer settings.  NKTR-214 is an experimental immuno-stimulatory recognized to expand specific cancer-fighting T cells and natural killer (NK) cells directly into the cancer micro-environment. The drug also increases the expression of PD-1 on these immune cells. TAK-659 inhibits both spleen tyrosine kinase (SYK), which is involved in . . . …

The Market The Stock Market is Still under pressure, which is frightening common investors, pressuring them to sell. We see no real reasons for the stock market to be acting as a Bear when the criteria of bullishness are obvious and several. We see no need for us to be scared, overreact and unwillingly contribute to the selloff. In the News ALNYLAM Alnylam (ALNY) announced positive …

The Week in Review #21 Part 1 THE MARKET - Like symphonic orchestras that bring to the listeners’ ears, minds and souls melodies composed by legendary authors, the stock market reflects what’s going on in the world, which would positively or negatively impact the lives and well being of the earth’s residents . . . This content is for paid subscribers. Please click here to subscribe or here to …

Prohost Letter #420 REAL BREAKTHROUGHS - One Has Already Been Used and the Other Has Extraordinary Promises In  the  NEWS - THE POWER OF CHECKPOINT INHIBITOR IMMUNOTHERAPY WOW It is about the checkpoint inhibitors immunotherapy, which is bringing to the world of cancer a stunning revelation of a combination treatment offering an almost cure for a lung cancer...

Bellicum Pharmaceuticals (BLCM): The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on studies of BPX-501 in the U.S. The decision follows consultation with the FDA and agreement on amendments to the study protocols including guidance on monitoring and management of neurologic adverse events. Bellicum will be working with U.S. clinical sites to resume patient recruitment based on the amended protocols. The FDA clinical hold did not affect the BP-004 …

When we reiterated that Illumina (ILMN) is the engine of the biotechnology evolution we meant it. We have to add, though, that the evolution made possible by Illumina’s sequencers and next-generation sequencing (NGS) engine is tremendously improving research and medical practice.  Regarding medical practice, the most recent news announced a global strategic partnership between Illumina and Loxo Oncology (LOXO) to co-develop and commercialize a multi-gene panel for broad tumor …

NOVARTIS TO ACQUIRE AVEXIS A few months ago, Novartis (NVS) CEO agreed to cash out of the company’s consumer health joint venture with GlaxoSmithKline (GSK), which brought in cash intended to further improve and expand Novartis’ pipeline. Here we are. Yesterday, Novartis announced its intention to acquire the gene therapy firm AveXis (AVXS) at $8.7 billion, paying $218 for each share of this firm. In a conference call on Monday, …

In today’s news, Incyte’s (INCY) immuno-oncology IDO1 inhibitor product Epacadostat in combination with Merck’s immuno-oncology product Keytruda® did not meet the endpoints of the trial in patients with unresectable or metastatic melanoma. The stock was subjected to a selloff – a market’s reaction pattern that occurs whenever negative news comes out of experimentation without consideration of the extent of their impact. A serious question to ask now would …

THE BIOTECH EVOLUTION: AN UNPARALLELED SPEED The Tremendous Regret Investors Will Feel If They Sell the Best of the Best Biotechnology Firms Only Because the Market Is Weird   Even if one is scared and feels he should sell shares, he must be wise enough to select those stocks whose prices are closer to reaching the targets he estimated as representing their fair values. Biotech investors who lend their ears to …

Alnylam (ALN) is probably experiencing a stock selloff following the announcement of successful results from Pfizer’s (PFE) drug tafamidis.    Indeed, results from Phase 3 ATT-ACT clinical trial demonstrate that tafamidis succeeded in treating Transthyretin Cardiomyopathy, a subtype of TTR amyloidosis caused by the destabilization of the protein transthyretin. Tafamidis demonstrated a statistically significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months. The drug was …

IN THE NEWS PTC THERAPEUTICS  PTC Therapeutics (PTCT) stock, which dipped yesterday, probably on news announcing a public offering of 4,000,000 shares of its common stock at a public offering price of $27.04 per share. Yesterday, in a crazy market that looked as if the bull and bear are boxers knocking each other down every 10 minutes, PTCT lost around $5. The stock seems to rebound in today’s premarket …