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 …

Yes, it is Sangamo’s (SGMO) zinc finger nuclease (ZFN) gene-editing technology, which for the first time in history enabled a patient to receive a treatment to edit the DNA of his cells directly inside the body. That was the news coming today from Sangamo. The Story Sangamo’s gene editing product known as SB-913 -- an investigational in vivo genome editing therapy was introduced for the first time …

Halozyme Therapeutics (HALO) announced that it will present nonclinical data at the 32nd Annual Meeting of the Society for Immunotherapy of Cancer (SITC) which demonstrate the potential for PEGPH20, Halozyme's pegylated recombinant human hyaluronidase, to increase the infiltration of immune cells into the tumor microenvironment and enhance the efficacy of immuno-oncology drugs in an HA-accumulating murine colon tumor model. The study shows that degradation of hyaluronan (HA) …

A couple of days ago, Cellectis (CLLS), which uses its own proprietary gene editing technology known as “transcription activator-like effector nuclease (TALEN®)” in developing immunotherapies, received the following good news: The FDA has lifted the clinical hold, which the Agency has put on Phase 1 trials of Cellectis’ UCART123 product candidate.       The Story In February 2017, Cellectis received an Investigational New Drug (IND) approval …

EXELIXIS For Exelixis’ excellent news, please read our latest article, "Exelixis’ (EXEL) Exceptional Financial Results and Corporate Achievements", under Today’s Highlights NEUROCRINE BIOSCIENCE Prohost Picked this firm on April 24, 2017, at $51 (see the Prohost Portfolio in the Prohost Letter). Today, Thursday, November 2, NBIX closed on  at $72.78 Up $11.85     In its Q3 report, Neurocrine Biosciences (NBIX) announced financial results that beat …

Cabozantinib Net Revenue for Q3 is $96.4 million. Total Revenue of $152.5 million  Net Income of $81.4 million, Diluted EPS $0.26 per Share Have these headlines said it all? Of course not, but what they said was sufficient to appreciate this firm when compared to other firms that were granted approvals more than three to four years ago, yet they are still spending much than what they earn. Exelixis’ (EXEL) drug …

Agenus (AGEN) announced that The Food and Drug Administration (FDA) granted marketing authorization to GlaxoSmithKline's (GSK) product Shingrix, a herpes zoster vaccine containing Agenus' proprietary immune adjuvant QS-21 Stimulon®. Shingrix vaccine is designed to prevent herpes zoster (shingles) in adults aged 50 years and older. The adjuvant in the vaccine is Agenus’ product QS-21 Stimulon. This adjuvant is to help improve the vaccine's effectiveness by …

Spectrum Pharmaceuticals (SPPI) announced an oral presentation of interim data from a Phase 2 clinical study evaluating the pan-HER inhibitor poziotinib in EGFR Exon 20 Mutant Non-Small-Cell Lung Cancer (NSCLC) by scientists from the MD Anderson Cancer Center. The data were presented at the 18th IASLC World Conference on Lung Cancer in Yokohama, Japan on October 15-18, 2017. The trial results from Poziotinib demonstrated evidence of significant anticancer activity in NSCLC …

Yes, indeed, the FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease LUXTURNA has the potential to be the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United State First adeno-associated viral (AAV . . . This content is for paid subscribers. Please click here to subscribe …

Good news emanating from late phase trials by development-stage firms is always refreshing for Prohost, especially when the news brings hope for the suffering patients whose diseases are not adequately responding to existing treatments. Today GOOD news is coming from late Phase trials announcing that a product called Tenapanor has met all its Aprimary and secondary endpoints for irritable colon syndrome with constipation (IBS-C), which …

MOTIF BIO Antibiotic Motif Bio’s (MTFB) antibiotic drug Iclaprim met the Phase 3 Revive-2 trial’s primary endpoints of non-inferiority against the current standard of care antibiotic, vancomycin in acute bacterial skin and skin structure infections (ABSSSI). The drug demonstrated a 78% clinical skin infection cure. According to Motif Bio, Iclaprim was nearly identical compared to vancomycin. The firm observed, though, that within 48 to 72 hours, a …

AstraZeneca’s (AZN) checkpoint PD-L1 inhibitor Imfinzi and NewLink Genetics’ (NLNK) IDO inhibitor indoximod will be combined and tested in Phase 2 randomized, placebo-controlled clinical trials together with the standard of care chemotherapy treatment for metastatic pancreatic cancer. AstraZeneca and NewLink Genetics will jointly fund the phase 2 trial of the combination that  NewLink will be sponsoring  It is a Phase 2  trial that will be …

EXELIXIS Being called overvalued by one analyst does not mean that Exelixis (EXEL) is, indeed, overvalued. We believe it is not and that’s why we decided to accumulate the stock on any weaknesses in EXEL. We do not intend to sell this stock at this time for what we believe are the wrong reasons. Some bloggers tried to interpret the reasons that led to the …