The U.S. Food and Drug Administration (FDA) Psychopharmacologic Drugs Advisory Committee (PDAC) voted 12 to 2 that the benefits outweigh the risks of Acadia’s (ACAD) drug Nuplazid™ (pimavanserin) for the treatment of psychosis associated with Parkinson’s disease. The Prescription Drug User Fee Act (PDUFA) action date for the FDA decision is May 1, 2016.  Nuplazid’s NDA was granted a breakthrough designation and Priority Review status for the treatment …

Alder Reports Phase 2b Trial of ALD403 Meets Primary and Secondary Endpoints Demonstrating Migraine Prevention in Patients with Chronic Migraine Alder Also Reports Positive Phase 1 Data Supporting Quarterly Single Injection Dosing Strategy for ALD403 Alder BioPharmaceuticals (ALDR) announced positive rrsults from two clinical trials evaluating its preventive chronic migraine monoclonal antibody drug ALD403. The drugtargets calcitonin gene-related peptide (CGRP). ALD403 acted rapidly and prevented migraine over …

It is an ingenuous decision made by investors who rushed to consider betrixaban Phase 3 APEX study a failure. Betrixaban is a once-daily orally administered Factor Xa inhibitor anticoagulant.  The Phase 3 AREX study was evaluating the superiority of oral betrixaban’s extended-duration anticoagulation vs. the injectable standard-of-care anticoagulation for the prevention of venous thromboembolism (VTE) in acute medically ill patients. The 7,513-subject study designed to assess the relative risk …

Amgen (AMGN) and UCB (Euronext Brussels: UCB) announced positive results from the BRIDGE pivotal Phase 3 placebo-controlled study evaluating the efficacy and safety of romosozumab in treating men with osteoporosis.  The study met the primary endpoint, demonstrating a statistically significant increase in bone mineral density (BMD) at the lumbar spine in men with osteoporosis treated with romosozumab compared with placebo at 12 months. All secondary endpoints comparing romosozumab with placebo …

Published articles in peer review journals cited that pharmacologic inhibition of RORγt regulates Th17 signature gene expression and suppresses cutaneous inflammation in vivo. Here we are, a clinical stage small company called Vitae Pharmaceuticals (VTAE) has just announced positive results from its Phase 2a proof-of-concept clinical trial of its RORyt inhibitor drug VTP-43742 in psoriatic patients. VTP-43742 is wholly owned by Vitae. It is an orally active RORyt inhibitor said to …

Immunotherapy for Cancer Blueprint Medicines Blueprint Medicines (BPMC) entered into a worldwide collaboration and exclusive license agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (collectively, Roche) for the discovery, development and commercialization of up to five small molecule therapeutics targeting kinases believed to be important in cancer immunotherapy. Under the terms of the agreement, Blueprint Medicines will receive an upfront cash payment of $45 million …

First therapy to demonstrate statistically significant improvement in overall survival and induction response rate in a pivotal Phase 3 trial in high-risk Acute Myeloid Leukemia (AML)   NDA submission for VYXEOS planned for later this year   Celator Pharmaceuticals (CPXX) announced positive results from Phase 3 trial of VYXEOS™ (cytarabine: daunorubicin) Liposome for Injection (also known as CPX-351) in patients with high-risk (secondary) acute myeloid leukemia (AML) compared to the standard …

London, UK, 14 March 2016:  GW Pharmaceuticals (GWPH), is a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform. The firm announced the positive results of the first pivotal Phase 3 study of its investigational medicine Epidiolex®(cannabidiol or CBD) for the treatment of Dravet syndrome. In this study, the firm’s product, Epidiolex, achieved the primary endpoint of a significant reduction in convulsive …

The Diagnosed Dead Is Far from Dying Xoma (XOMA): Stock rallied following report announcing an upfront payment from Novartis, which multiplied the firm’s revenues 10 times over 2014. Novartis paid Xoma $37 million, Novo Nordisk paid $5 million and Pfizer paid the firm a little less than $4 million. CEO John Varian said, “Novel antibodies and technologies created by XOMA scientists to target diseases such as …

Of the Upcoming Game-Changing Actions THE EXPECTED FDA APPROVAL OF JAZZ DRUG DEFIBROTIDE On March 31, the FDA is expected to decide on the approval of Jazz Pharmaceuticals’ (JAZZ) drug defibrotide. The drug is for patients with hepatic veno-occlusive (VOD), also known as sinusoidal obstruction syndrome (SOS), with multi-organ failure (MOF) post-hematopoietic stem-cell transplantation (HSCT). Optimism about the FDA decision is prevailing among analysts and observers as the results of the …

As we kept describing it, Illumina’s (ILMN) is the engine behind the biological revolution reflected in the current existence of treatments, which aim at targets that were unknown before this firm enabled discovering them. Illumina’s establishing and launching the independent company, Grail, will speed the successful outcome of the war against cancer. It will contribute to advancing the diagnosis of cancer through a tremendous increase in the sensitivity …

Prohost Letter #393 IMMUNOTHERAPY - PROHOST PORTFOLIO - NEW PICKS? - A Brief Reminder of the CAR T –TCR Immunotherapy Approaches In this issue we continue our journey inside the territory of cancer immunotherapy. We remind that in the Prohost Letter issue #391, we dealt with the immunotherapy based on genetically engineered T Cells, enabling them to seek out, attack and neutralize cancer cells. The immune system’s T …

Preparing a Prohost Letter dedicated to immunotherapy and the firms that are behind the promising novel approaches towards enabling the immune system to treat, or cure diseases if possible, we fell on other genius approaches. In recent Prohost Letters, we discussed the CAR T immunotherapy approach for the management of advanced, or metastatic cancers and in the upcoming Issue, which was due two days ago, …

Agenus (AGEN) is a project in the making since the firm has decided to play an integral part in immuno-oncology, doing everything possible towards building checkpoint modulator antibodies, in addition to its protective and therapeutic vaccines. Missing analysts’ expectation by one cent, which leads to stocks’ punishment has become an accepted routine, satisfying daily traders and other investors. The substance, however, which  interests us, is in …

Exelixis’ (EXEL) quarterly results beat analysts’ expectations, which was the least of our concerns. The details of the firm’s actions during the past year, however, confirmed our conviction that the recently approved products will be taken care of responsibly by the firm that created them and believed in them against all pessimistic opinions. Indeed, more important than the firm’s current revenues and spending was the firm’s ability …

Prohost Letter #392 February 28, 2016 - How could it be possible for any genius to make intelligent guesses about the market's next moves when highly regarded experts, including Nobel Prize winners in economics, remained quizzical about the recent horrifying performances of publicly traded stocks? Since the beginning of 2016, the stock market has been made to look as if a runaway train with many passengers …

Baxalta (BXLT) and Precision BioSciences a genome editing company announced a global collaboration to develop a broad series of allogeneic chimeric antigen receptor (CAR) T cell therapies directed towards areas of major unmet need in multiple cancers. CAR T elegant genetically engineered approach that enables the T cells to pinpoint and neutralize cancer cells was created to succeed and Baxalta’s decision to enter this immunotherapy program demonstrates how …

Recent Breakthrough Designated Products CANCER Acute Myeloid Leukemia PKC412 (midostaurin) developed by Novartis AG (NVS) is an oral, multi-targeted kinase inhibitor for acute myeloid leukemia (AML) with FLT3 mutations. It is also being studied for aggressive systemic mastocytosis/mast cell leukemia. FMS-like tyrosine kinase-3 (FLT3) is a cell-surface receptor for tyrosine kinase, which has a role in the upsurge of certain blood cell types. The drug has positive data …

AMGEN AND OSTEOPOROSIS Amgen And UCB Announce Positive Top-Line Results From The Phase 3 Study of Romosozumab In Postmenopausal Women With Osteoporosis The FRAME Study Met All Primary Endpoints by Reducing the Incidence of New Vertebral Fracture Through 12 and 24 Months Amgen (AMGN)and UCB (Euronext Brussels: UCB): Top-line results from the Phase 3 placebo-controlled FRActure study in postmenopausal woMen with ostEoporosis (FRAME). The results  showed …

On February 4, 2015, we wrote an article titled, “Aerie Pharmaceuticals is speeding towards improving the treatment of glaucoma”, posted under News & Comments at the Prohost website, we wrote the following: YOUNG BUT STRONG COMPETITOR IN THE GLAUCOMA HUGE MARKET Aerie Pharmaceuticals has eye drops that could become a game changer in the management of glaucoma. The firm’s lead drug, Rhopressa™, is a one drop once-daily …