Another day, another badly needed treatment for a dangerous disease is on its way to be FDA approved. The day was yesterday, Thursday, April 7, 2016. The company is Intercept (ICPT), the disease is primary biliary cirrhosis renamed primary biliary cholangitis (PBC), the drug is called Ocaliva™ (obeticholic acid). The good news came from the FDA's Gastrointestinal Drugs Advisory Committee, which voted 17 to 0to recommend accelerated approval of Ocaliva™.    Now …

Years ago, a friend who is a highly regarded Immunologist lectured us during one of our visits to his home city Milano in Italy about the important role well written press releases play in highlighting the prominence of unknown, yet, capable biotech firms. “If not for its announced news, millions of interested people around the world, including myself, wouldn’t have heard on time about Genentech …

Pfizer (PF) terminated its acquisition agreement with Allergan Plc (AGN) The new set of rules released by the U.S. Treasury Department to make it difficult for U.S. companies to conduct tax inversions contributed Pfizer’s decision. Analysts noted that the new regulations seem almost directly aimed at Pfizer-Allergan. In one of two major parts of the new rules, the U.S. government would not consider the assets acquired …

PFIZER Pfizer (PF) announced that its oral drug tofacitinib in doses of 5 mg and 10 mg twice daily  met Phase 3 endpoints on psoriatic arthritis. The trial was conducted on adult patients with active psoriatic arthritis (PsA). The recruited patients had an inadequate response to at least one conventional anti-rheumatic drug (csDMARD) and were tumor necrosis factor inhibitor (TNFi)-naïve. Patients enrolled in the study were …

Prohost Letter #394 Part 2 PART 2 - Part 1 of the Prohost letter issue #394 posted on March 31,2016 ended with the review of Ariad Pharmaceuticals (ARIA), one of the biotech firms that have at least one marketed product that we believe have the chance to boost their revenues in the near-future. In this part 2 of the same Prohost Letter, we add other firms …

Many in Wall Street have been anxious, sitting on fire while waiting to learn, which biotech company Gilead Sciences (GILD) would decide upon acquiring that would deepen and enhance its pipeline. In Today’s news, a firm called Nimbus Therapeutics, LLC today announced that it has signed a definitive agreement under which terms Gilead will acquire Nimbus Apollo, Inc., a Nimbus Therapeutics wholly-owned subsidiary. Important is that the acquisition …

Prohost Letter #394 Part 1 Gilead - Some people sold Gilead’s (GILD) stock on Wednesday, March 23 after news announced on Tuesday that a jury decided that Gilead has infringed on two HCV molecules patents that Ionis (IONS), formerly Isis, and Merck had secured from their research. Ionis CEO said in a press release that his firm and Merck used their expertise during their collaboration in …

Incyte (INCY) and Eli Lilly (LLY): The New England Journal of Medicine published detailed results of RA-BEACON — a pivotal phase 3 global study of baricitinib, a once-daily oral treatment for moderate-to-severe rheumatoid arthritis (RA). The study met its primary endpoint of ACR 20 response for baricitinib compared with placebo at week 12. ACR 20 response rates were (P≤0.001 for each baricitinib dose versus placebo): 55 percent for baricitinib 4 mg; 49 percent …

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 …