Biopharmaceutical News Week # 2016.37

Biopharmaceutical News Week # 2016.37

Author: Jean-Claude Muller, 穆卓Executive Editor at BtoBioInnovation, jcm@btobioinnovation.com

Editor’s Note

We have received an interesting number of positive, encouraging and motivating replies to our enquiry concerning the future of this biopharmaceutical newsletter, although the total number of replies received came at the low end of our expectations. The editorial board has therefore decided that, for the time being, this newsletter will now only be forwarded, free of charge, to the subscribers and the readers who have provided feedback to our recent enquiries.

Acquisitions /mergers/joint-ventures

Allergan anchors its gastroenterology R&D portfolio with the acquisition of Tobira Therapeutics in a deal worth up to $1.7 billion representing a six fold cash premium. Allergan will get access to cenicroviric and evogliptin, two investigational drugs for the treatment of nonalcoholic steatohepatitis (NASH). Under the terms of the deal Allergan will pay Tobira and upfront fee of $28.35 per share and up to $49.84 per share in a contingent value right payment tied to achieving development and regulatory milestones. The cash price made by Allergan is more than six time Tobira’s market value of $89 million the day prior to the announcement.

….and acquires privately held Akarna Therapeutics for $50 million upfront plus undisclosed contingent milestones fees related to the development of AKN-083, a preclinical non–bile acid farnesoid X receptor (FXR) agonist for treatment of nonalcoholic steatohepatitis (NASH). Akarna’s portfolio includes additional development-stage FXR compounds aimed at treating inflammatory and fibrotic diseases.

Johnson & Johnson acquires Abbott Medical Optics, a global leader in ophthalmologic surgery with its intraocular lenses and in consumer eye health, in a cash deal worth $4.36 billion. The market for cataracts is growing with an aging population and currently around 20 million people are blind from age-related cataracts and the condition affects sights in at least 100 million eyes.

Business

Teva enters a licensing deal with Regeneron to develop fasinumab, a NGF pain treatment, in a deal worth up to $2.6 billion. NGF drugs had been high expectation treatments several years ago, but safety issues had sidelined the drugs although recent approaches seem to overcome the problem. Under the terms of the deal Teva and Regeneron will co-develop the drug and Teva will be paying $250 million in cash as an upfront payment and cover the almost $500 million development cost and splitting future profits.

Tardeva Therapeutics enters a worldwide license agreement with Madrigal to discover, develop and commercialize new oncology treatments based on Madrigal HSP90 drug conjugate platform, including PEN-866, in a deal worth up to $249 million. Madrigal acquired the HSP90 drug conjugate platform through its merger with Synta Pharmaceuticals. PEN-866 is a small-molecule drug conjugate consisting of an HSP90 ligand conjugated to SN-38, the highly potent, active metabolite of irinotecan. Tardeva will make an undisclosed upfront payment and milestones of up to $163 million as well as tiered single-digit royalties.

AbbVie partners with BioArtic (Karolinska Institute, Sweden) to develop and market novel antibodies targeting α-synuclein to treat several diseases including Parkinson’s disease. BioArtic’s portfolio is led by BAN0805, an antibody targeting toxic forms of the α-synuclein protein and also develops a biomarker assay aimed at achieving early diagnosis by measuring monomeric and toxic forms of α-synuclein. Through a collaboration with Uppsala University, Bioarctic has recently launched a positron emission tomography (PET) program to detect protofibrils in neurodegenerative disorders using antibodies.

Takeda Pharmaceuticals enters a partnership with Zydus Calida to address emerging infectious diseases such as chikungunya. The chikungunya virus is most often spread to people by Aedes aegypti and Aedes albopictus mosquitoes, the same vectors that spread dengue and zika. Since 2005, India, Indonesia, Maldives, Myanmar and Thailand have reported over 1.9 million cases and almost 1.3 million suspected cases of Chikungunya have being recorded in the Caribbean islands, Latin American countries, and the USA. Financial terms were not disclosed

Genentech is partnering with BioNTech (Mainz, Germany) to develop personalized cancer vaccines using synthetic mRNA’s designed to produce therapeutic proteins in patient’s cells, a technology closed to the ones of Gritstone, Moderna and Neon Therapeutics. These new personalized cancer vaccines, used in combination, are expected to help expand on the effectiveness of checkpoint inhibitors. Financial terms of the deal were not disclosed. BioNTech has previously signed deals with Sanofi and Eli Lilly.

Astellas Pharma (Tokyo, Japan) and Courtagen Life Sciences (Woburn, MA, USA) enter a collaboration to develop treatments for mitochondrial diseases using Courtagen’s ZiPhyr bioinformatics platform.

Biotheranostics (San Diego, CA, USA) a spinoff of BioMerieux (Lyon, France) enters a partnership with Syapse (Palo Alto, CA, USA) to combine data from its Cancer TYPE ID and Breast Cancer Index genomic tests with Syapse’s precision medicine platform in order to support clinicians to better assess diagnostic data obtained from cancer patients.

Approval of drugs and vaccines

The European Commission approves Ipsen’s (Boulogne-Billancourt, France) Cabometyx, or cabozantinib, as a treatment for adults with advanced renal cell carcinoma who received prior VEGF-targeted therapy. The US FDA approved cabometyx for the same indication in April 2016. . Exelixis and Ipsen jointly commercialize and further develop cabozantinib indications outside of the United States, Canada and Japan.

US FDA approves:

Sarepta Therapeutic’s Exondys 51, or eteplirsen, for the treatment of Duchenne muscular dystrophy despite a prior negative advisory committee decision stating that the data was insufficient to support its recommendation. The agency’s decision came months after an advisory panel voted against an approval and FDA insiders, notably Ron Farkas, who recently left the agency. Janet Woodcock, the agency's Center for Drug Evaluation and Research Director, said the decision "reflects FDA's ability to apply flexibility to address challenges we often see with rare, life-threatening diseases."

…..and three simultaneous approvals for the expanded use of Novartis’s Ilaris or canakinumab for the treatment of three rare types of Periodic Fever Syndromes: Tumor Necrosis Factor-Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF).

Drugs at clinical stage

The Committee for Medicinal Products for Human Use (CHMP) has given its backing for approval to eleven new medicines. Three cancer treatments: Eli Lilly’s Lartreuvo or olaratumab for advanced soft tissue sarcoma, Pfizer’s Ibrance for locally-adavanced or metastatic breast cancer and Takeda’s Ninlaro for the treatment of multiple myeloma. Other recommendations were handed to Boehringer Ingelheim’s Glyxambi or (empagliflozin/linagliptin) for type 2 diabetes; Amgen's Parsabiv (etelcalcetide) for secondary hyperparathyroidism; and Advanced Accelerator Applications'SomaKit-TOC (edotreotide) for the diagnosis of gastro-entero-pancreatic neuroendrocrine tumours. Sigma-tau Arzneimittel's hybrid application for Chenodeoxycholic acid sigma-tau (chenodeoxycholic acid) got a positive opinion for cerebrotendinous xanthomatosis. Four generic medicines also won CHMP backing: Ivabradine JensenR (ivabradine) and Ivabradine Zentiva (ivabradine) to treat angina pectoris and heart failure, Zentiva (emtricitabine/tenofovir disoproxil) to treat HIV infection and Granpidam (sildenafil) for the treatment of patients with pulmonary arterial hypertension.

European regulators have issued a green light for:

Allergan's Truberzi, giving patients potential access to the first approved treatment for irritable bowel syndrome with diarrhoea (IBS-D).

…and for Janssen’s Stelara or ustekinumab, a dual interleukin-12, interleukin 23 inhibitor for the treatment of moderate to severe active Crohn’s disease in adults intolerant or not responding to conventional therapy.

US FDA grants fast–track designation to Windtree Therapeutics Aerosurf or lucinactant as a treatment for respiratory distress syndrome in premature infants with a gestation age of 26 to 32 weeks. Aerosurf is an investigational drug/device product that combines Windtree's proprietary aerosolized KL₄ surfactant with its novel aerosol delivery system (ADS) based primarily on its capillary aerosol generator technology.

Novartis announces positive results with Siponimod (BAF312), a drug acting on sphingosine-1-phospahte (SP1) receptors, for the treatment of multiple sclerosis, showing that the drug cut the risk of disability progression in adults with secondary progressive forms of the disease.

Johnson & Johnson confirms that the US FDA has put JNJ-63709178, a bispecific antibody that targets CD3 and CD123, partnered with Genmab for the treatment of acute myeloid leukemia on clinical hold after patients in the trial suffered serious adverse events.

Vical (San Diego, CA, USA) and Astellas’ lead vaccine ASP0113 as a treatment for herpes and kidney transplant patients failed to show efficacy in a phase 2 study. Both companies are remaining committed to further assess ASP0113 in hematopoietic cell transplant patients.

Lundbeck announces that a Phase 3 study of idalopirdine, a 5HT-6 agonist for the treatment of Alzheimer’s disease, failed to meet its primary endpoint (ADAS-cog) when added to donepezil. Secondary endpoints also failed to show a significant difference from placebo. Two other phase 3 studies will continue as planned by Lundbeck and Otsuka which has co-promotion rights in key areas.

AstraZeneca pulls back its European application to market cediranimib, an ovarian cancer drug, in combination with platinum-based chemotherapy. The EMA had concerns over the trial and whether drug's potential side effects outweighed its benefit.

Gilead Sciences stops the study of a combined phase2/3 study with GS-5745, an investigational anti-MMP9 antibody in patients with moderate to severe ulcerative colitis. The decision follows a planned interim analysis of 150 patients by the Data Monitoring Committee which recommended that the study be terminated early. Gilead said it has also reviewed the data and determined "there is insufficient evidence of a treatment benefit" in the patients randomised to receive GS-5745.

Medical Devices and Diagnosis News

Bayer is introducing Kyleena, a new intra-uterine system (IUS) for the prevention of pregnancy for up to 5 years. Kyleena contains 19.5 mg of the progestin hormone levonorgestrel. It is inserted by a healthcare provider and will be available by prescription only. The device demonstrated its contraceptive efficacy in a randomized, open-label study conducted in 11 countries in Europe and the Americas, Kyleena is Bayer’s third IUS. It markets two other levonorgestrel-releasing devices: Mirena, which is approved for up to 5 years of use, and Skyla, which is intended to be used for three years. Bayer also markets oral birth control medication.

Cost, Pricing and Market Access

The UK National Institute for Health Care and Excellence have recommended use of Alexion's Strensiq to treat a rare and often fatal condition called hypophosphatasia (HPP), but have restricted its reach to children with certain forms of the condition. Juvenile and adult-onset forms of HPP have much lower mortality rates than those appearing in infancy, where 100 % of those diagnosed in the first six months of their lives die before they reach one year.

Miscellaneous

GlaxoSmithKline names Emma Walmsley, its current head of consumer healthcare to be its new chief executive and will replace Andrew Witty who announced its decision to retire on March 31, 2017.

Sanofi is taking Merck to court claiming it has infringed 10 patents covering Lantus and Lantus Solostar. Sanofi had previously fought with Eli Lilly on similar infringements but subsequently settled the case with a deal that allows Lilly to launch a Lantus biosimilar in December 2016.

193 nations at the United Nations General Assembly signed a historic declaration to combat antimicrobial resistance. Each signatory has agreed that drug resistant infections must be tackled as a priority, in light of projections that, by 2050, 10 million people a year will be dying as a result at a cost to the global economy of some $100 trillion, if the problem isn't tackled now. As such, the nations have committed to: developing surveillance and regulatory systems on the use and sales of antimicrobial medicines for humans and animals; encouraging new ways to develop novel antibiotics and improving rapid diagnostics; and raising awareness among health professionals and the public on how to prevent drug resistant infections. "This Declaration is the culmination of six years of hard work and I am extremely proud that every UN member state is now engaged in the enormous task of tackling the greatest future threat to our civilisation," said chief medical officer Professor Dame Sally Davies. Earlier this month, G20 leaders had already committed to exploring how best to stimulate research and the development of new antimicrobial products, as it is widely recognised that the current commercial model does not encourage activity in this area.

Bioevents

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.

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