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 …

Following the announcement of two new licensing agreements for its ENHANZE® drug-delivery technology, Halozyme Therapeutics (HALO) raised financial guidance for 2017 as follows: - Net revenue of $245 million to $260 million, an increase of $130 million from the prior range of $115 million to $130 million, reflecting the portion of upfront payments from the new agreements expected to be recorded as revenue in 2017; - Operating expenses of $240 million …

Positive Phase 2 Results for Tipifarnib in HRAS Mutant Head and Neck Cancer Four of the first six HRAS Mutant HNSCC Patients enrolled on study achieve confirmed RECIST Partial Responses and Durable Responses Greater than One Year   On September 7, 2017, i.e., five days ago, Kura Oncology (KURA) announced positive results from a Phase 2 trial from its lead product candidate, tipifarnib, in patients with …

Marinus Pharmaceuticals’ (MRNS) stock rallied following its announcement of top-line data from Phase 2 open-label study in patients with the severe epilepsy disorder known as CDKL5. The disease is a rare  severe genetic derived epilepsy that results in seizures, pervasive neuro-developmental delay and disabling behavioral issues. The disease has no approved treatments. The good news that boosted the stock price early today has come out …

In the news, the FDA has placed a clinical hold on two phase 1 trials of Cellectis’ (CLLS) off- the-shelf product UCART123 after the death of a 78-year old patient. Cellectis is working with the FDA towards finding ways that help mitigate the risks expected to have caused the fatality. We experienced deaths with other CAR T products with the cytokine release syndrome on the …

Eli Lilly and Company (LLY) and Incyte (INCY) announced that, after discussions with the U.S. Food and Drug Administration (FDA) in late August, Lilly will resubmit the New Drug Application (NDA) for baricitinib before the end of January 2018. That was fast, we mean the re-submission. To remind you, the firms had submitted Baricitinib for review seeking marketing approval for rheumatoid arthritis in the U.S., the …

Earlier this month, on Aug. 24, 2017, Propeller Health announced the expansion of its 2015 collaboration with GlaxoSmithKline (GSK), enabling both companies to prepare for and undertake commercial activities using the Propeller clip-on sensor and software platform for use with  ELLIPTA Inhaler. The inhaler, which is marketed by GSK, is produced by Innoviva (INVA) and approved by the FDA in other countries around the world. …