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 …

Acorda (ACOR) stock was up around 10% reaching over $28 in premarket trading. The stock is trading now around $27 with its gains oscillating between $2 – $2.20. The enthusiasm for ACOR seems to be about rumors that Biogen (BIIB) or possibly other Asian drug makers might have demonstrated the interest in Acorda. What’s going on with Acorda? In June 2017, the stock rallied on news about positive clinical trial …

Checkpoint inhibitors approved for a few firms as immunotherapy for cancer are increasingly demonstrating their superiority in extending the lives of patients when given as first-line treatments. In the news, Merck’s (MRK) anti-PD-1 therapy, pembrolizumab (Keytruda®) in combination with pemetrexed (Alimta®) and cisplatin or carboplatin, for the first-line treatment of patients with metastatic non-squamous non-small cell lung cancer (NSCLC), met its dual primary endpoints of overall survival …

VERTEX From Europe, Vertex (VRTX) was informed that the European Commission has granted an extension of the Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor). Orkambi is for cystic fibrosis (CF) in people with two copies of the F508del mutation, including children ages 6 through 11 Reimbursement: In countries such as Ireland, reimbursement agreements will lead to rapid access to ORKAMBI. In other countries across the European Union, Vertex will begin a country-by-country reimbursement process. Clinical trial Results …

On Dec. 12, 2017, Denali Therapeutics (DNLI) announced the closing of its initial public offering of 15,972,221 shares of its common stock at a price to the public of $18.00 per share. On January 5, 2018, less than one month since it closed the public offering, Denali and Takeda (TSE:4502) announced entering into a strategic option and collaboration agreement to develop and commercialize up to three specified therapeutic product candidates for neurodegenerative …

The Food and Drug Administration (FDA) accepted the supplemental Biologics License Application (BLA) for Seattle Genetics’ (SGEN) product Adcetris (brentuximab vedotin) in combination with chemotherapy for the frontline treatment of patients with advanced classical Hodgkin lymphoma. The Agency granted Priority Review for the application, and the Prescription Drug User Fee Act (PDUFA) target action date is May 1, 2018. The BLA is based on positive results from a phase 3 ECHELON-1 trial designed to determine if Adcetris …

Based on statistically significant results from the randomized phase 2 CABOSUN trial in patients with previously untreated renal cell carcinoma (RCC), the U.S. Food and Drug Administration (FDA) approved Exelixis’ (EXEL)drug Cabometyx® (cabozantinib) for the expanded indication of patients with RCC. The results demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) versus sunitinib, a current standard of care. Today’s label expansion follows the initial FDA approval …

Juno Therapeutics (JUNO) is one of the casualties that occurred during the 59th American Society of Hematology (ASH) Annual Meeting. The stock experienced undeserved selloff except for the fact that it had a deserved rally that we believe will remain as long as the firm is spending effort towards improving its cancer treatment. This cancer treatment is the  living genetically engineered chimeric antigen receptor (CAR) T cell expected …

Gilead Sciences (GILD) has unsurprisingly agreed to acquire Cell Design Labs in a structured buy out valued for up to $567 million, including the shares of Cell Design Labs held by Kite, the CAR T firm that Gilead has recently acquired. Brian Atwood, President and CEO of Cell Design Labs, Inc., said, “Bringing our robust technology platforms under the Gilead umbrella, with its outstanding research …

On Nov. 21, 2017, Portola Pharmaceuticals (PTLA) announced that the U.S. Food and Drug Administration (FDA) has informed it that it will respond to the Prior Approval Supplement (PAS) request to change the current manufacturing release specification within the standard 60-day extension period. No additional information was requested at this time. The new action date is January 30, 2018. Bill Lis, chief executive officer of Portola said, “As …

DelMar Pharmaceuticals (DMPI) -- A biopharmaceutical company focused on the development of cancer therapies provided an overview of three scientific posters presented at the 22nd Annual Meeting and Education Day of the Society for Neuro-Oncology (SNO) held on November 16-19, 2017 in San Francisco, CA. DelMar might have surprised the financial community reporting that 93% of patients with Glioblastoma multiforme (GBM) were alive and 40% of …