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 …

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 …

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 . . . …

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, …

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 …

Exelixis: Delightful Overseas Cabozantinib News Ipsen (EPA:IPN) announced that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA) provided a positive opinion for Exelixis’ (EXEL) product Cabometyx® (cabozantinib) 20, 40, 60 mg regarding the first-line treatment of adults with intermediate- or poor- risk advanced renal cell carcinoma (aRCC). The CHMP positive opinion will be reviewed by the European Commission (EC), which has the …

In spite of their successes, Immuno-oncology products, which we still like to call immunotherapy for several reasons, are a work in progress. The most two highlighted approaches that enable the immune system to clear cancers are the checkpoint inhibition and the chimeric antigen receptor (CAR T) genetically engineered immune system T cells therapies. Therapeutic vaccines had some successes, but the outcome was inferior to the two approaches that have …

What Do We Make of the Following Good News? HERON THERAPEUTICS Will HTX-011 Help in the War Against Opioids?  Heron Therapeutics (HRTX), a commercial-stage biotechnology company announced positive results from its completed Phase 3 studies of the investigational agent HTX-011 in people undergoing bunionectomy and hernia repair. HTX-011 is described as the first and only long-acting local anesthetic to demonstrate in Phase 3 studies significantly reduced pain …

Alnylam (ALNY) announced that Sanofi Genzyme declined its opt-in for the development and commercialization of the RNAi product lumasiran, an investigational RNAi therapeutic for the treatment of Primary Hyperoxaluria Type 1 (PH1). Based on this decision, Alnylam intends to rapidly advance lumasiran into a Phase 3 pivotal study in late 2018, and to commercialize lumasiran globally, assuming product approval. Alnylam has also announced that the FDA has granted lumasiran …

Regeneron’s (REGN) pipeline products have still a lot of good news to announce. The latest is about its product Dupixant (dupilumab) which has already been FDA approved for atopic dermatitis. Now, there are reasons to believe that this drug could be also approved as an add-on maintenance for people with moderate to severe Asthma. The news has come from both Regeneron and its partner Sanofi (SNY), stating that …

FERTILITY Fertility is what guarantees the presence of humans on the planet Earth. The reproductive system in men and women are the engines of fertility that creates living beings. These important engines get disturbed in some people, which represents a problem that needs repair. Life sciences arts have done a good job towards overcoming the causes that would prevent a couple from bearing children. Still …

Portola Pharmaceuticals and the CHMP’s Oral Explanations for Andexanet Alfa and Betrixaban Portola (PTLA) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has communicated a positive trend vote on the Marketing Authorization Application (MAA) for Portola’s factor Xa inhibitor antidote andexanet alfa. The agency has also communicated a negative trend vote for betrixaban, the firm’s oral, Factor Xa inhibitor. Andexanet Alfa Bill Lis, the chief executive officer …