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 …

Seattle Genetics and Pieris pharmaceutical Collaboration Agreement Pieris Pharmaceuticals (PIRS) and Seattle Genetics (SGEN) signed a multi-program immuno-oncology collaboration agreement intended to evaluate novel bi-specific immuno-oncology agents. These agents would combine Pieris’ Anticalin proteins drugs with Seattle Genetics’ targeted antibodies to develop multiple targeted bi-specific immuno-oncology treatments for both solid and blood cancers. The Story Pieris Pharmaceuticals created recombinant engineered Anticalin protein-based therapeutics. These proteins are versions of the human protein lipocalin, which naturally bind, …

Expected or not, we believe that the news announcing that the U.S. Food and Drug Administration (FDA) approval Vertex’s (VRTX) cystic fibrosis combo product Symdeko™ (tezacaftor/ivacaftor and ivacaftor) is good news. It is indeed excellent news for the many cystic fibrosis people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis trans-membrane conductance regulator (CFTR) gene or who have at least one mutation that …

A Stanford research team investigating a new immunotherapy approach, which consists of injecting tiny amounts of two immuno-stimulating molecules directly into the cancers themselves, announced good news. The first of the two injections contained a CpG oligonucleotide a short stretch of DNA that promotes the expression of OX40, a protein that resides on the surface of the immune system’s T cells. The second injection comprised an antibody that binds …

Vertex (VRTX) announced the selection of two next-generation correctors, VX-659 and VX-445, to advance into Phase 3 development as part of two different triple combination regimens for people with cystic fibrosis (CF). The decision was based on data from initial Phase 2 trials, including new data from ongoing Phase 2 studies demonstrating mean absolute improvements in percent predicted forced expiratory volume in one second from baseline through four weeks of treatment …

ChemoCentryx (CCXI) develops new drugs for inflammatory, autoimmune diseases and cancers. The firm’s approach comprises targeting the chemokine and chemoattractant systems with the aim of discovering, developing and commercializing orally-administered therapies for inflammation and malignancy and maybe other disease conditions. In the News A few days ago, ChemoCentryx announced positive overall survival (OS) results from Phase Ib clinical trial of its second CCR2 inhibitor drug CCX872 designed for the treatment of locally advanced/metastatic pancreatic cancer and …

Amgen (AMGN): Phase 3b results from LIBERTY study assessing the safety and efficacy of Aimovig™(erenumab) 140 mg in patients with an episodic migraine who had experienced two to four previous preventive treatment failures demonstrate that the study met its primary and secondary endpoints.    With regard to meeting the primary endpoint, the results demonstrate that patients on Aimovig experienced at least a 50 percent reduction from baseline in …