Zika virus

Dr. Bruce Aylward, executive director for outbreaks and health emergencies of World Health Organization said that Brazil will host a “fantastic Olympics” and that the Zika mosquito-borne virus will be “way down” by the time the Summer Games begin in Rio de Janeiro, on Aug. 5. He also indicated that the mosquito population is expected to drop off in the area around Rio since it will be the southern hemisphere’s winter by then. He said Olympic venues are also in a relatively confined area, making it easier for authorities to control the local mosquito population.

The epidemic outbreak of Zika virus disease and its highly probable link to fetal development abnormalities requires the urgent need for pregnant women and those of reproductive age to become a priority group for developing and evaluating new vaccines. To do so, public health policy bodies must issue new guidelines and overcome several barriers through concerted efforts and leadership with a clear action plan similar to the one which has allowed advances in pediatric drug development.

Acquisitions /mergers/joint-ventures

OSE Pharma (Paris, France) and Effimune (Nantes, France) announce their merger to create OSE Immunotherapeutics, a new international enterprise that offers innovative immunotherapies based on the activation or regulation of the immune system and that will be able to develop a next generation checkpoint inhibitor. The headquarters of the merged company will be transferred from Paris to Nantes. 

Business

According to the European Commission, Teva Pharmaceutical intends to divest assets worth up to $1 billion to resolve antitrust concerns over the Allergan’s generic business acquisition."Commitments have been submitted and the new legal deadline is set on 10 March," said European Commission spokesman Ricardo Cardoso.

Bristol-Myers Squibb completes the previously announced sale of its HIV R&D portfolio to ViiV (the joint venture of GlaxoSmithKline and Pfizer) but not its currently marketed drugs. Under the terms of the deal, BMS received an upfront payment of $350 million. It can earn up to $518 million in development and regulatory milestones and up to $587 million for the discovery and preclinical programs plus tiered royalties on commercial sales. ViiV will also pay sales-based milestones of up to $750 million for each of the clinical assets and up to $700 million for each of the discovery and preclinical programs.

Baxalta (Bannockburn, Ill, USA) enters a $1.7 billion collaboration with Precision BioSciences (Durham, NC, USA) on the development of allogenic chimeric antigen receptor T cell therapies against six undisclosed cancer targets. Most CAR T cell therapy technologies are focused on isolating cells from cancer patients’ blood and manipulating them to specifically target receptors on tumour cells. Baxalta will employ Precision’s proprietary ARCUS genome editing technology that enables the production of CAR T cells derived from healthy donors in the hope of overcoming the manufacturing-related limitations with existing CAR T therapies. Under the terms of the deal Baxalta will pay an upfront payment of $105 million and as much as $1.6 billion in option and milestone fees. In January of this year Shire has announced that it will acquire Baxalta for $32 billion in a cash-and stock deal and become the largest rare disease company in the world.

Sanofi sells French rights to four of its drugs to Merus Labs (Toronto, Canada) in a deal worth $24.8 million. The drugs included in the agreement are two gynecological disorder treatments Surgestone (progestatif) and Provames (estradiol), Speciafoldine (folic acid), indicated for macrocytic anemia, and Tredemine (niclosamide anhydrous) for the treatment of tapeworm infection. Merus said the deal will continue its strategies of broadening its product line through acquisitions of marketed prescription drugs, as well as expanding its presence in France.

….and terminates its Auvi-Q marketing deal with PDL Biopharma (Incline Village, NV, USA) after major manufacturing issues last year. Auvi-Q is an epinephrine injection device used to treat severe allergic reactions which was licensed to Sanofi in 2009 in a deal worth €210 million. Last October production was halted and a mass recall initiated after a number of devices showed malfunction relating to the accuracy of the dose delivered. At the time the recall had a negative impact of around €100 million. “Last week PDL was advised that Sanofi will terminate its license and development agreement later this year”.

Otsuka Novel Products, the German unit of Otsuka Pharmaceutical said in a release it will participate in the Stop TB Partnership to make the recently European, Japan and South Korea approved delamanid available to more than 100 low and middle income countries to help cut rates of multidrug-resistant tuberculosis.

Seven pharmaceutical companies link into “a ground breaking” consortium aimed at the development on new therapies for Parkinson’s disease. AbbVie, AstraZeneca, Biogen, Eli Lilly, Merck Sharp and Dohme, Pfizer and UCB are the industry fellow peers with Critical Path Institute and Parkinson’s UK as the founders of the project. “There is a strong realisation from the industry that collaboration among industry, academia, and worldwide regulatory agencies, along with the sharing of data, has the potential to create a more efficient development process,” said Diane Stephenson, executive director of the consortium. 

Verily, Alphabet’s life sciences spinoff has been selected by the National Institutes of Health (NIH) to co-launch President Obama's announced Precision Medicine Initiative (see newsweek # 2015.3 on www.btobioinnovation.com). Verily will work with Nashville, TN-based Vanderbilt University to roll out the pilot program, which is expecting to enroll 79,000 volunteers by the end of this year. The participants will provide personal data that could trigger discoveries surrounding health and help treat disease. Earlier this year Verily said that it would join forces with the American Heart Association (AHA) and AstraZeneca to support scientists to come up with innovative approaches to treating heart disease.

Approval of drugs, vaccines, diagnostics and devices

The European Commission approves

Bayer’s Kovaltry, an unmodified full-length recombinant factor VIII product for hemophilia A patients. Hemophilia A affects 1 in 10,000 males, including more than 30,000 in Europe.

Eli Lilly’s Potrazza, or necitumumab, as a treatment for patients with advanced non-small cell lung cancer, (NSCLC) in combination with gemcitabine and cisplatin chemotherapy to treat patients with epidermal growth factor receptor (EGFR) expressing squamous NSCLC.

Shield Therapeutics’ (London, UK) Feraccru, or a complex of ferric (Fe) iron with maltol, for the treatment of iron defiency anaemia in patients with inflammatory bowel disease.

US FDA approves

UCB’s Briviact, or brivaracetam, as an add-on treatment for partial seizures in epilepsy patients aged 16 years and over. Brivaracetam is believed to act by binding to SV2A, a ubiquitous synaptic vesicle glycoprotein that may prepare vesicles for fusion. Ease of administration is one of the main advantages of Briviact which can be initially delivered without titration, enabling patients to receive a full therapeutic dose right away. Briviact was approved in Europe in January this year, and is already available in the UK and Germany.

Pfizer’s Xeljanz an extended-release version for the treatment of rheumatoid arthritis giving patients access to the first once-daily, oral JAK inhibitor for the condition.

US FDA issues a Refusal to File letter to PTC Therapeutic’s (South Plainfield, NJ, USA) NDA application to market its investigational drug Translaran or ataluren -an oral, first-in-class, protein restoration therapy-, for nonsense mutation Duchenne muscular dystrophy, stating that “the application was not sufficiently complete to permit a substantive review”. The European CHMP had granted a conditional marketing authorization to PTC Therapeutics Translara in May 2014.

Drugs at clinical stage

The European Commission grants

three orphan status to Acerta Pharma’s (Oss, The Netherlands) acabrutinb (ACP-196) for the treatment of chronic lymphocytic leukemia/ small lymphocytic lymphoma, mantle cell lymphoma and lymphoplasmacytic lymphoma (Waldenstrom's macroglobulinemia). Acalabrutinib, is an orally available second generation inhibitor of an enzyme called Bruton's tyrosine kinase (BTK). AstraZeneca finalized its $4B investment in Acerta (55% stake) in December 2015.

….and to Retrophin’s (San Diego, CA, USA) RE-024 as a treatment for pantothenate kinase-associated degeneration. RE-024 holds orphan and fast track status by the US FDA.

US FDA grants:

breakthrough status to Immunomedics’ (Morris Plain, NJ, USA) sacituzumab govitecan, an antibody-drug conjugate as a treatment candidate for patients with triple-negative breast cancer who have not responded to other therapies.

And fast-track status to Nivalis Therapeutics’ (Boulder, COL, USA) N91115 a cystic fibrosis drug  which acts as a stabilizer of the cystic fibrosis transmembrane conductance regulator protein  The drug already holds orphan designation for the same indication.

Japan’s Ministry of Health, Labor and Welfare (MHLW) will review 3 drugs and 2 medical devices under its fast track approval process known as Sakigake. A MHLW spokesman said three of the products are regenerative medicine candidates. A gene therapy by Daiichi Sankyo and developed at the University of Tokyo's Institute of Medical Science to treat brain tumors. STR01 by Nipro (Osaka) for a potential stem cell treatment for spinal cord injuries. And an unspecified candidate using autologous cardiac stem cells to treat heart disease in children. On the medical device side, there is resorbable barrier to curb the risk of organ adhesion developed by Otsuka Pharmaceutical and a titanium bridge from Nobelpharma to protect vocal chords. Products to be considered under the Sakigake approval process must display a novel mechanism of action, be scalable commercially, show high efficacy and be developed and planned for approval in Japan ahead of the rest of the world. (see newsletter # 2015.15 onwww.btobioinnovation.com).

Amgen and UCB’s investigational osteoporosis drug, romosozumab, achieved primary endpoints a late stage clinical trial in reducing the number of new fractures in post-menopausal women versus placebo. Romosozumab is an investigational bone-forming monoclonal antibody designed to inhibit the protein sclerostin. It has a dual effect on bone, increasing bone formation and decreasing bone resorption. 

Science and Technology

A three-dimensional 'map' of a critical protein that malaria parasites use to invade human red blood cells could lead to a vaccine countering the most widespread species of the parasite. "We have produced the first three-dimensional, atomic resolution structure of the protein using the Australian Synchrotron in Melbourne. We now basically have a map of where the proteins are binding their receptors, which gives us the instructions we need to begin designing inhibitors that could be used in a malaria vaccine” said Dr Wai-Hong Tham from the Walter and Eliza Hall Institute (Parkville, Melbourne, Australia).

Japanese researchers have developed a peptide vaccine, targeting the hormone angiotensin II, to study hypertension in mice, which has the potential to protect the brain when administered prior to a stroke. Vaccinated rats displayed higher levels of angiotensin II antibodies in functional tissues on the side of the brain where the stroke occurred.

Cost, Pricing and Market Access

According to EvaluatePharma “Outlook to 2020” report the worldwide prescription drug sales are expected to reach one trillion dollars by 2020 (CAGR: 5% between 2015 and 2020). Industry’s R&D pipeline is valued at $493 billion. Continued confidence in the sector is being driven by the high number of novel drug approvals and the emergence of breakthrough drugs. The only major concern are global pricing and market access for the new products and the Biopharmaceutical industry will have to come forward with new ways for pricing its innovative products (see our article in the November/December 2015 issue of MedNous)

Merck & Co’s CEO, Kenneth Frazier, knows that scrutiny over price hikes is rapidly increasing. The price of Januvia, Merck’s major diabetes treatment, went up by 93% from 2010 to 2015, a study found last year. Januvia is among a group of product that have come into the pricing debate recently. Frazier told The Wall Street Journal, "there is a challenge" surrounding pricing that the pharma industry is "going to have to think through." And that includes finding "new and better ways to link what we charge for the drug to the value that it actually creates in the marketplace," he said. He pointed out, there are "barriers in the system" to sharing the risk with consumers. Merck, he says, is "actually very eager" to try outcomes-based pricing, but "the laws around what we can and cannot do in our pricing model were made for a different environment. They weren't made for performance-based contracting or risk-sharing or any of those kinds of experimental approaches to pricing."

In final guidelines, the National Institute for Health and Care Excellence (NICE) indicates that Merck & Co’s Ezetrol, or ezetimibe, can be used as a monotherapy to treat adult patients with primary (heterozygous‑familial or non‑familial) hypercholesterolaemia in whom initial statin therapy is contraindicated. Ezetrol is also recommended in combination with initial statin therapy as an option when cholesterol concentration is not appropriately controlled, or where a person is unable to have higher doses. Ezetrol is a cholesterol-absorption inhibitor that blocks intestinal absorption of dietary and biliary cholesterol and related plant sterols, but without affecting uptake of triglycerides or fat-soluble vitamins.

Miscellaneous

LabCorp owned Covance (Princeton, NJ, USA) sells its preclinical laboratories in Alnwick (UK) to ARC Trinova, a new entity formed by the investment group Shott Trinova. Covance has acquired the Alnwick site from Sanofi back in 2010 and employed 130 people. As part of the agreement ARC Trinova agreed to retain a minimum of 50 employees. New owner said it “will add capabilities in active pharmaceutical ingredient (API) process development and scale-up to the multi-kilogram level”.

Robert Califf, US President Barack Obama’s pick to lead the US FDA, cleared a Senate procedural vote by 80-6 to make Califf’s final approval likely to take place rapidly.

Bayer has named Werner Baumann as its new chairman of the management board succeeding current chief executive Maijn Dekkers. Baumann will retain his current role as head of strategy and portfolio management when he takes on the chairman position on May 1, much earlier than anticipated as Dekkers is now stepping down at the end of April instead of the end of the year.

Bioevents

• BIO Asia 2016 on March 15-16 in Tokyo (Japan)
• 9^th European Life Science CEO Forum and Exhibition on March 15-16 in Zürich (Switzerland)
• Global Healthcare Forum on March 16-17 in Singapore (Singapore)
• BioPharma Asia Convention on March 22-24 in Singapore (Singapore)
• 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)
• 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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