Editor’s Note

Horrible terror attacks did strike in Brussels on March 22 where 31 people were killed and several hundred people were injured. BtoBioinnovation wishes to express its sympathy to all the victims and their families and its strong solidarity with all its Belgian readers and friends.

 Zika virus

According to the Centers for Disease Control and Prevention (CDC, Atlanta, USA), a quarter of Puerto Rico’s 3.5 million people will likely get the Zika virus within a year and eventually 80% may be infected.

Acquisitions /mergers/joint-ventures

Bristol-Myers Squibb acquires Padlock Therapeutics (Cambridge, MA, USA) for $600 million and gains rights to Padlock’s Protein/Peptidyl/Arginine Deiminase inhibitor programme targeting novel rheumatoid arthritis therapies and possibly other autoimmune diseases such a lupus erythematosus. BMS will make an upfront payment of $225 million and up to $375 million in contingent milestone payments.

Magnis Therapeutics (Rochester, MN, USA) and fellow Omnis Pharma (Rochester, MN, USA) have announced their merger to form a new company named Vyriad which will focus on immunovirotherapies. The portfolio of Vyriad consists of eight clinical candidates and seven preclinical products. Vyriad's oncolytic immunovirotherapies are based on the company's engineered oncolytic vesicular stomatitus virus (VSV) and oncolytic measles virus platforms, designed to enable selective destruction of cancer cells without harming normal tissues.  The value of the merger was not disclosed.

Business

Bayer invests $130 million to extend its ongoing strategic collaboration in ophthalmology with Regeneron. Bayer had already partnered Regeneron’s Eylea or aflibercept, as a single agent and in a combination therapy for wet, age–related macular degeneration. The new pact will include a combination of the angiopoietin2 antibody nesvacumab with aflibercept. Under the terms of the deal, Bayer will make an upfront payment of $50 million and commit up to $80 million in milestones and get non US marketing rights and split profits.

Merck KGaA enters a collaboration with R-Biopharm (Darmstadt, Germany) to develop and market companion diagnostics across a broad range of therapeutic indications. Financial terms were not disclosed.

Merck & Co collaborate with Harvard University on the development of small-molecule treatments for acute myeloid leukemia (AML) and other cancers in a $20 million deal.  Harvard University is also eligible for undisclosed milestone fees plus sales royalties on products arising from the collaboration. The approach relates to compounds that were developed in Professor Matthew Shair’s laboratory and consists in inhibiting enzymes that regulate the transcription of genetic programs altered in AML.

Evotec (Hamburg, Germany) spins out some of its assets into a new company called Topas Therapeutics and develop a new investigational drug for the treatment of multiple sclerosis. Topas will receive €14 million from Evotec, Epidarex Capital, EBML Ventures and Gimy. Evotec will be the largest shareholder. Timm Jenssen, the former Evotec CSO will become Topas’ CEO.

Cellectis (Paris, France) enters into a license and supply agreement with Takara Bio (New York, NY, USA) to access recombinant human fibronectin fragment RetroNectin® and secure Cellectis’ manufacturing processes and expand the its UCART production capabilities.

Combinations Therapies

Currently many novel immunotherapies are monoclonal antibodies that block protein-protein interactions between T cell checkpoint inhibitors and their ligands. Recently it was shown that small molecules can also induce potent and selective immuno-modulation and that combination of immunotherapy agents can have substantial synergistic effects. As a result the number of partnerships and agreements dealing with immunotherapy combinations is steadily increasing. These deals are usually aimed at conducting validation studies of specific combinations in order to identify best product combination for a given cancer or a given subset of cancer as well as identification of specific biomarkers. Three such agreements were announced during this week.

Roche/Genentech collaborates with Kite Pharma (Santa Monica, CA, USA) to evaluate the combination therapy of Roche’s atezolizumab and Kite’s KTE-C19 for the treatment of refractory, aggressive non-Hodgkin lymphoma. KTE-C19 works by genetically modifying a patient's T cells to express a Chimeric Antigen Receptor (CAR) designed to target the antigen CD19, a protein which is expressed on the cancer cell surface of B cell. Atezolizumab is a monoclonal antibody that targets PD-L1, which is expressed on tumor cells and tumor-infiltrating immune cells.

……and with Janssen to evaluate the combination of their respective monoclonal antibodies, Darzalex or daratumumab and atezolizumab as a treatment for multiple myeloma and other solid tumor cancer.

Cellectis (Paris, France) announces a partnership with MabQuest (Pully, Switzerland) to develop a new class of monoclonal antibodies targeting PD-1. These anti-PD-1 antibodies have potential uses for multiple indications in immunotherapy and Cellectis plans to use them either in combination therapy with its gene-edited Universal Chimeric Antigen Receptor T-cells (UCART) product candidates, in combination with other already approved immunotherapy drugs or as single-agent.

Approval of drugs, vaccines, diagnostics and devices

US FDA, via the Animal Rule, approves Elusys Therapeutics’ Anthim (obiltoxaximab) as a treatment of inhalation of anthrax, a rare disease that can occur after exposure to infected animals or contaminated animal products, or as a result of an intentional release of anthrax spores. When inhaled, anthrax bacteria replicate in the body and produce toxins that can cause massive and irreversible tissue injury and death. The spores are resistant to destruction and can be spread by release in the air. Obiltoxaximab is a monoclonal antibody that has been designed to neutralize the toxins produced by anthrax. The FDA Animal Rule allows efficacy findings from adequate and well-controlled animal studies to support marketing clearance when it is not feasible or ethical to conduct efficacy trials in humans.

US FDA approves Eli Lilly’s Talz or ixekizumab, an IL-17 monoclonal antibody, as a treatment of moderate to severe plaque psoriasis in adults. The pivotal study showed clear superiority over Pfizer’s etanercept.

The Therapeutic Goods Administration of Australia and the New Zealand Medicines and Medical Devices Safety Authority has approved Actelion’s (Allschwil, Switzerland) Uptravi or selixipag for the treatment of pulmonary arterial hypertension. The drug was approved by the US FDA in December 2015

According to a report by Express Script, US FDA has approved 56 new drugs in 2015, overshooting the record high of 53 in 1996. The approvals include Zarxio the first biosimilar approved in the US.

Drugs at clinical stage

The European Commission grants orphan drug status to Biomarin Pharmaceutical’s (San Rafael, CA, USA) BM 270 as a treatment of haemophilia A, an inherited bleeding disorder caused by a deficiency in factor VIII.  BMN 270 is an AAV 5 gene therapy product designed to restore factor VIII plasma concentrations.

US FDA grants breakthrough status to Novimmune's (Geneva, Switzerland) NI-0501, an anti-interferon-gamma monoclonal antibody, as a treatment for primary hemophagocytic lymphohistiocytosis (HHL) that is refractory or has recurred or progressed during chemotherapy. HLH is a hyperinflammatory syndrome characterized by uncontrolled and aberrant activation of the immune system and a cytokine storm presenting with non-remitting fever, pancytopenia, coagulopathy, and hemophagocytosis potentially leading to death.

US FDA grants fast-track status to MediciNova’s (La Jolla, CA, USA) MN-166, or ibudilast, for the treatment of relapsed-remitting multiple sclerosis. MN-166 is an orally available, small molecule phosphodiesterase-4 and -10 inhibitor and a macrophage migration inhibitory factor inhibitor that suppresses pro-inflammatory cytokines and promotes neurotrophic factors. Ibudilast has been marketed in Japan and Korea since 1989 to treat post-stroke complications and bronchial asthma. MediciNova licensed ibudilast from Kyorin Pharmaceutical.

US FDA grants orphan status to DelMar Pharmaceuticals’ (Vancouver, BC, Canada and Menlo Park, CA, USA) VAL-083 for medulloblastoma therapy. The investigational drug has already received orphan status designation in the US and in Europe as a treatment of glioblastoma.

Pfizer announced that tofacitinib, its JAK inhibitor, met key primary and secondary clinical endpoints in a phase 3 study assessing both safety and efficacy in ulcerative colitis.

UCB and Amgen report that romosozumab met its primary endpoints in demonstrating increase in bone mineral density at the lumbar spine in male osteoporosis patients. All secondary endpoints comparing romosozumab with placebo were also met.Romosozumab is a monoclonal antibody designed to inhibit the protein sclerostin with a dual effect on bone, increasing bone formation and decreasing bone resorption.

Medical Devices and Diagnosis News

Janssen through its Disease Interception Accelerator (DIA) is starting two new projects dialing up diagnostic and therapeutic approaches in Congestive Obstructive Pulmonary Disease (COPD) and gestational diabetes. DIA will work with Boston University School of Medicine to uncover disease pathways linked to COPD, and Singapore's Agency for Science, Technology and Research (A*STAR) on biomarkers for gestational diabetes. DIA, which was launched in 2015, is now handling more than two dozen research collaborations.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence (NICE) has recommended Novartis’ Entresto (sacubitril/valsartan) as an “innovative and cost-effective treatment”, and as an option for treating symptomatic chronic heart failure with reduced ejection fraction in adult patients, but restricting its use to those with a left ventricular ejection fraction of 35% or less.

….and now recommends Janssen’s Zytiga or abiraterone for the treatment of patients with prostate cancer for whom chemotherapy is not yet indicated, following additional data provides by the drug sponsor. NICE had turned down Zigita in December 2015.

According to a report by Alcimed (Paris, France) the vaccine world market has increased by 24% from $26 billion to $32.3 billion between 2011 and 2014 and should reach about $80 billion in 2025. Currently four major players (GSK, Merck & Co, Sanofi and Pfizer) generate 65% of the worldwide sales but only 20% of the total volumes.  Therapeutic vaccines represent an interesting growth platform estimated at $5 billion in 2020.

Miscellaneous

Merck & Co versus Gilead Sciences in hepatitis C patent litigation: Round 1. In a jury verdict Merck and its partner Ionis win a first round in a royalty fight with Gilead’s hepatitis C drugs Sovaldi and Harvoni. The jury will next weigh in on damages Merck and Ionis should collect from Gilead. Gilead says it will appeal and ask for a bench trial, thinking that a panel of judges would view Merck's patent claims differently.

Novartis has settled civil charges of committing bribery in China from 2009 to 2013 and agreed to pay $25 million to the Chinese government.  According to the SEC, Novartis bribed healthcare professionals in China to boost its sales and tried to hide the bribes, which included entertainment, gifts, travel, and meals, by recording them as legitimate expenses.

Bioevents

• Annual China Healthcare Investment Conference on March 29-31 in Shanghai (China)
• BIO Europe Spring on April 4-6 in Stockholm (Sweden)
• BIO-IT World Conference & Expo’16 on April 5-7 in Boston (USA)
• BIO Vision on April 13-14 in Lyon (France)
• BIO Trinity on April 25-27 in London (UK)
• Bio€quity Europe 2016 on May 10-11 in Copenhagen (Denmark)
• Meet2Win: The French Oncology Convention on May 12-13 in Bordeaux (France)
• Strategic Alliance Management Congress on May 16-18 in Philadelphia (USA)
• China BIO Partnering Forum on May 18-19 in Suzhou (China)
• BIO International Convention on June 6-9 in San Francisco (USA)

• BIO Pharm America on September 13-15 in Boston (USA)
• 7^th Innovation Days on October 3-4 in Paris (France)
• BIO Europe on November 7-9 in Cologne (Germany)
• BIO Latam on November 29-30 in San Juan (Puerto Rico)
• Biofit 2016 on November 30-December 1 in Lille (France)

IMPORTANT NOTICE: 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.

Author: Jean-Claude Muller, 穆卓Special Advisor at I&IR and Executive Editor at BtoBioInnovation, jcm@btobioinnovation.com

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