Biopharmaceutical News Week # 2016.46

Author: Jean-Claude Muller, 穆卓Executive Editor at BtoBioInnovation

Zika Virus

Researchers from the Wistar Institute (Philadelphia, PA, USA) have tested a synthetic DNA vaccine that protected 100% of vaccinated animals from Zika infection. There is no approved vaccine or therapy against Zika, but the candidate, which is being developed by the Wistar Institute, Inovio Pharmaceuticals (Plymouth Meeting, PA, USA) and GeneOne Lifesciences Seoul, South Korea), is the furthest along in development.

Mergers, Acquisitions and Joint Ventures

According to the SonntagsZeitung (Switzerland), Novartis is considering selling Alcon, its ophthalmology division, which it gradually acquired from Nestlé in several deals totaling $15 billion, and is weighing acquiring Amneal Pharmaceuticals (Bridgewater, NJ, USA) to expand its Sandoz generic business.

Grünenthal (Aachen, Germany) acquires Thar Pharmaceuticals (Pittsburg, PA, USA) and gets access to T121 a late stage product for the treatment of pain related to complex regional pain syndrome. The value of the deal was not disclosed.

Business

Merck enters a partnership with OpGen (Gaithersburg, MA, USA) on the development of informatics technology and rapid diagnostics for antibiotic resistance. OpGen will gain access to Merck's library of 200,000 bacterial pathogens to support its database of antibiotic-resistant pathogens and rapid DNA test development. Financial terms of the deal were not disclosed.

Bristol-Myers Squibb gets exclusive rights to license and develop Nitto Denko’s (Osaka, Japan) ND-L02-sO201, a nonalcoholic steatohepatitis clinical candidate as well as other small interfering RNA (siRNA) molecules targeting heath shock protein 47 in vitamin A formulation to treat lung fibrosis and other organ fibrosis. Under the terms of the deal BMS shall pay a $100 million upfront, plus undisclosed milestone fees, option payments and royalties.

Eli Lilly grants Eddingpharm (Shanghai, China) exclusive rights to promote and distributes two antibiotics, Ceclor or cefaclor, a cephalosporin, and Vancorin or vancomycin, a tricyclic glycopeptide, in mainland China.

Sanofi Genzyme announces that it will opt-in to to co-develop and co-commercialize Alnylam’s (Cambridge, MA, USA) fitusiran, a RNAi drug, for the treatment of hemophilia in the US, Canada and Western Europe but declined to opt in on ALN-AS1 for the treatment of acute hepatic porphyrias. Sanofi Genzyme will pay up to $75 million in development and regulatory milestones, including a $25 million upon the initiation of a phase 3 trial and royalties up to 20%

Janssen will partner with Amgen to evaluate the combination of Darzalex, or daratumumab, a human IgG1K mAb against CD38 and Kyprolis, or carfilzomib, a selective proteasome inhibitor, as a treatment of multiple myeloma and various cancers.

Bristol-Myers Squibb enters a partnership with Enterome Bioscience (Paris, France) for the discovery and development of microbiome-derived biomarkers, bioactive molecules, novel cancer drugs and biomarkers associated with its own immune-oncology drugs. Enterome will use its two different platforms—a quantitative metagenomics platform allowing the characterization of a personal metagenome or “metagenotype^®” associated with a disease phenotype and a functional metagenomics platform allowing the discovery and the development of new drugs and targets. BMS will pay a $15 million upfront fee and R&D funding and receive exclusive rights to intellectual property and therapies generated during the collaboration.

AstraZeneca and Allergan complete their licensing deal for MEDI2070, an IL-23 monoclonal antibody whereby AstraZeneca could receive a $250 million upfront fee and payments of up to $1.25 billion over a period of up to 15 years.

IBM teams up in a five years and $50 million research project with the Broad Institute of MIT and Harvard University to determine why some cancers are drug-resistant from the start and why others are able to mutate.

Approval of drugs and vaccines

The European Commission has approved Amgen’s Parsabiv, or etelcalcetide, a D-amino peptide calcimimetic, as a treatment for secondary hyperparathyroidism in adults with chronic kidney disease on hemodialysis.

The European Commission has granted expanded approval of Janssen’s Stelara, or ustekinumab, for adults with moderately to severely active Crohn’s disease who have had inadequate response with conventional therapy.

US FDA approves:

Gilead Science’s Vemlidy, or tenofovir alafenamide, a prodrug of tenofovir, a nucleotide analog prodrug reverse transcriptase inhibitor, as a once-daily treatment for patients with chronic hepatitis B virus infection and compensated liver disease. The drug will have a boxed warning about risks of severe hepatomegaly with steatosis, lactic acidosis and post-treatment aggravation of infection.

Bristol-Myers Squibb’s Opdivo, or nivolumab, as a treatment of metastatic recurrent squamous cell carcinoma of the head and neck following progression on platinum-based therapy.

Duchesnay’s (Blainville, Canada) Bonjesta, or doxylamine succinate (an antihistamine) and pyridoxine hydrochloride (a vitamin B6 analog) extended release tablets, a fixed dose combination of 20 mg of each drug, for treating nausea and vomiting in pregnant women who are unresponsive to conservative management. Duchesnay also markets Diclegis, which contains doxylamine succinate 10mg and pyridoxine HCl 10mg, for a similar indication.

…and Endoceutic’s (Quebec, Canada) Introsa or prasterone, as a treatment for postmenopausal wome with moderate to severe dyspareunia.

Drugs at clinical stage

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommends the use of Novartis’s Arzerra or ofatumumab, in combination with fludarabine as a treatment for relapsed chronic lymphocytic leukemia.

The European Medicines Agency’s Committee for Orphan Medicinal Products has granted orphan status to MediciNova’s MN-166, or ibudilast, as a treatment for amytrophic lateral sclerosis.

US FDA grants breakthrough status to Seattle Genetics’ Adcetris, or brentuximab vedotin, as a treatment for two cutaneous T-cell lymphoma subtypes:  those with CD30-expressing mycosis fungoides (MF) and those primary cutaneous anaplastic large cell lymphoma (pcALCL) who have received one prior line of therapy. MF and pcALCL are the most common subtypes of cutaneous T-cell lymphoma, accounting for more than 75% of cases.

US FDA grants fast track status to Centexion Therapeutics’s (Baltimore, MD, USA) CNTX-4975, a highly potent, ultrapure, synthetic form of capsaicin, designed to be administered via injection into the site of pain, as a treatment for Morton’s neuroma, a rare pain disorder affecting the foot.

US FDA grants priority review to Novartis’ PKC412, or midostaurin, an oral multi-targeted kinase inhibitor, as a treatment for newly diagnosed FLT3-mutatd acute myeloid leukemia or advanced systemic mastocytosis.

US FDA grants orphan status:

to Genexine’s (Seoul, South Korea) GX-H9, a long acting recombinant human growth hormone fused to a hybrid FC, as a treatment for growth hormone deficiency.

to Armo Biosciences’ (Redwwod, CA, USA) AM0010, a pegylated interleukin-10, as a second-line therapy for patients with metastatic pancreatic cancer in combination with FOLFOX.

…and to Cytori Therapeutics’ (San Diego, GA, USA) ECCS-50 for the autologous retreatment patients with scleroderma.

Sanofi and Regeneron’s sarilumab, a human monoclonal antibody that binds to the interleukin-6 (IL-6) receptor showed to be superior to AbbVie’s Humira in improving rheumatoid arthritis symptoms in a late stage study. A few days ago the FDA had issued a complete response letter (CRL) refusing the approval of the drug citing insufficiencies at a Sanofi “fill and finish” site.

AMG 334 or erenumab, a fully human monoclonal antibody specifically designed to target and block the Calcitonin Gene-Related Peptide (CGRP) receptor, which is thought to play a key role in mediating the pain of migraine, being developed by Amgen and Novartis to prevent chronic migraine, has met its primary endpoint in a late stage study.

Medical Devices and Diagnosis News.

US FDA approves Medtronic’s defibrillator that tailors resynchronization therapy to each patient using an algorithm to adjust pacing; The EffectivCRT algorithm automatically adjusts heart pacing for individual patients. The algorithm will be particularly useful in heart failure patients who have atrial fibrillation, an irregular heart rate that can impede a patient’s response to cardiac resynchronization therapy.

GE Healthcare and University of California San Francisco enter a collaboration to build up a library of deep learning algorithms. Deep learning algorithms learn to spot patterns in images and other data. By pairing such algorithms with powerful computers and growing repositories of medical images and data, GE and rivals such as IBM think they can give clinicians the information they need to quickly diagnose patients and choose the most appropriate intervention.

One week after receiving a CE mark for its MRI-compatible CRT pacemaker, St. Jude Medical announced an FDA full-body MRI label for its recharge-free spinal cord stimulation system. This is the second major update to St. Jude’s Proclaim Elite Spinal Cord Stimulation System, which was approved in November 2015. It is indicated for the treatment of chronic back pain and frees the patient from needing to regularly charge the device.

Cost, Pricing and Market Access

Roche and Bristol-Myers Squibb have cut their cancer drug price (Roche’s Perjeta for the treatment of breast cancer, BMS’ Sprycel for the treatment of leukemia) in order to receive UK National Institute for Health and Care Excellence (NICE) recommendation.

Miscellaneous

In a recent report published in the October issue of MedNous, Robin Davidson, an independent biotech equity analyst, writes “EU-based companies collectively have above average representation in cell and gene therapies” and even more striking “in gene therapies EU-based companies rank only slightly behind their US peers, but are significantly ahead in terms of approved products. Three gene therapy products have achieved regulatory approval in Europe: Chiesi/Uniqure’s Glybera, GSK’s Stimevelis and MolMed’s Zalmoxia –which compares with none as yet in the US”. The report describes 41 selected advanced therapies from EU-based companies in gene therapies, oncolytic viruses, cancer vaccines, cell therapies as well as engineered T-cells but stresses the point that although EU-based companies preform extremely well and are highly competitive versus their US counterparts, access to the capital market pushed them to the US given the dominance of the US for the funding of biotech companies.

The World Health Organisation has announced that immunisation campaigns for GSK's malaria vaccine will begin to 2018 for phase one pilot deployments in sub-Saharan Africa. GSK's RTSS or Mosquirix is the world's first malaria vaccine, but is only partially effective and needs to be given in a four dose schedule. The WHO has therefore said that it should be deployed on a pilot basis before a wide-scale use.

Just one month after the global charity Médecins Sans Frontières (MSF) publicly refused a Pfizer’s offer for a million free Prevenar 13 doses, its pneumococcal vaccine, Pfizer announced  a“ major expansion” to its humanitarian assistance with the vaccine. To help address emergencies, Pfizer will reduce the price Prevenar 13 to the “lowest prevailing global” cost of $3.10 per dose in such cases. The move follows years of public shaming by MSF, which repeatedly asked Pfizer and GlaxoSmithKline to lower their prices.

Roche announces a $100 million investment in its new international cancer immunotherapy Centers of Research Excellence network, which includes 21 academic centers worldwide.

Bioevents

BIO Latam on November 29-30 in San Juan (Puerto Rico)

Biofit 2016 on November 30-December 1 in Lille (France)

9^th Biotech Showcase Investor Conference on January 9-11, 2017 in San Francisco (USA)

JP Morgan Health Conference on January 9-12, 2017, in San Francisco (USA)

BIO CEO & Investor Conference on February 13-17, 2017, New York City (USA)

BIO Asia on March 14-15, 2017, in Tokyo (Japan)

BIO-Europe Spring on March 20-22, 2017 in Barcelona (Spain)

This document has been prepared by btobioinnovation and is provided to you for information purposes only.  The information contained in this document has been obtained from sources that btobioinnovation believes are reliable but btobioinnovation does not warrant that it is accurate or complete. The views presented in this document are those of btobioinnovation’s editor at the time of writing and are subject to change.  btobioinnovation has no obligation to update its opinions or the information in this document.

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