Yes, the title is real and correct and the condition is ASD. The US Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation for Roche’s (RHHBY) drug balovaptan developed for ASD. Balovaptan has shown the potential to improve social interaction and communication in people with ASD. Balovaptan is an investigational small molecule drug that acts as a vasopressin (V1a) receptor antagonist. Evidence from both human and animal studies implicate …

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 …

Celgene (CELG) and Juno (JUNO) announced the signing of a definitive merger agreement upon which Celgene has agreed to acquire Juno. Celgene will pay $87 per share in cash, or a total of approximately $9 billion, net of cash and marketable securities acquired and Juno shares already owned by Celgene (approximately 9.7% of outstanding shares). The transaction was approved by the boards of directors of both companies. Juno …

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 …

Juno Therapeutics’ (JUNO) stock price orbited the moon after-hours trading yesterday, rising 45%, following a wall street journal report stating that Celgene (CELG) was in discussions to take over Juno and that the negotiations could result in a deal in the coming weeks. Juno was established to create cellular immuno-oncology products- chimeric antigen receptor T cell (CAR T) to treat cancer. Neither Juno nor Celgene would …

The Week in Review #16 More Exciting Rallies in Prohost Picks - The past week and for several week fascinating outperformances have been seen for many of the Prohost Portfolio stocks. We continue to articulate that the good news is not only in the stocks’ rallies, but also in the outstanding stories behind them . . . This content is for paid subscribers. Please click here …

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 …

Prohost Letter #416 Part 2 2017 - A YEAR OF BREAKTHROUGH TECHNOLOGIES, BREAKTHROUGH PRODUCTS, IMPROVEMENTS ON PROMISING BREAKTHROUGH TREATMENTS, DECIPHERING IMPORTANT LIFE SCIENCE MYSTERIES, PINPOINTING BIOLOGICAL PATHWAYS FOR TREATMENT RESISTANCE, MIRACLE CURES, AND… A LOT OF DISTORTED NEGATIVE INTERPRETATIONS - Prioritizing our choices of breakthroughs has been a dilemma . . . This content is for paid subscribers. Please click here to subscribe or here to log …

As part of its ongoing strategy to build an innovative pipeline beyond its two internally-discovered, commercially available compounds, cabozantinib and cobimetinib, Exelixis (EXEL) entered into an exclusive collaboration and license agreement with StemSynergy Therapeutics. The aim of the collaboration is discovering and developing novel cancer products. targeting Casein Kinase 1 alpha (CK1α), a component of the Wnt signaling pathway implicated in key cancer processes. Exelixis and StemSynergy intend to conduct preclinical …

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 …

Sangamo’s (SGMO) has abundant positive news since it offered proof of concept and feasibility of its zinc finger protein transcription factor (ZFP-TF)-mediated gene regulation approach and other approaches. The most recent announcement was made early today by Sangamo and Pfizer (PFE) about a new collaboration. The cooperation aimed at developing a potential therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene. The firms will …

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 …