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 …

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 …

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 …

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 …

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 …

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 …

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, …

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 …

Incyte: A Good Company Facing Irrelevant Investors’ Reaction  Incyte decided to stop an early ongoing trial of a combination treatment ruxolitinib (Incyte) plus regorafenib (Bayer) for colon cancer after observing the combination wasn’t working. Both drugs are approved for different indications and both are doing well in the market. At this moment in the market chaos, this news looked as if a terrible thing that must cause an unwarranted …

Halozyme Therapeutics (HALO) seems to have closed the previously announced $150 million royalty-backed debt transaction with investment funds managed by Pharmakon Advisors and Athyrium Capital Management.  The debt is said to be secured by future royalties of ENHANZE™ products, received from Halozyme's collaborations Roche and Baxalta only. Halo declared delight with the conclusion of this deal as it is a non-dilutive financing and because it finds …

Theravance Biopharma (TBPH) announced the commercial launch of VIBATIV® (telavancin) in Canada. VIBATIV is a once-daily antibiotic with a dual mechanism of action against Gram-positive bacteria, including difficult-to-treat methicillin-resistant Staphylococcus aureus (MRSA). The drug is approved in Canada for adult patients with hospital-acquired bacterial pneumonia (HAP) and ventilator-associated bacterial pneumonia (VAP) known or suspected to be caused by susceptible isolates of Staphylococcus aureus including methicillin-susceptible S. aureus (MSSA) and MRSA. VIBATIV is …

The Market is Surely Ignoring the Historical Summit Biotech achievements are standing on Right Now     The latest biotech sector’s breakthroughs we presented was the novel powerful gene editing techniques CRISPR/cas9 and TALEN. Our article in the Prohost Letter #390 meant to enable the reader to understand that the evolution in biological sciences, especially in sequencing and analyzing the genome of the sick and healthy cells, has reached a stage …