Acquisitions /mergers/joint-ventures

AstraZeneca is in advanced talks to acquire Acerta Pharma (Oss, The Netherlands), a privately held company focusing on immune-oncology drugs, for $5 billion. Acerta’s leading drug is acalabrutinib (ACP-196) a selective, irreversible Bruton’s tyrosine kinase inhibitor for the treatment of lymphomas and chronic lymphocytic leukemia.

Business

Novo Nordisk teams up with IBM to use its Watson computing system in diabetes research. Watson is a “smart big data system” able to process large amounts of data to uncover patterns and insights, propose evidence-based answers, and learn from each interaction. The Watson system is already in use at the Memorial Sloan Kettering Cancer Center to develop new tools to diagnose and treat cancer, and Johnson & Johnson to discover new tools for people who have had knee-replacement surgery.

Celgene and Inception IBD (San Diego, CA, USA) have entered into a collaboration to discover and develop small molecule therapies, based on genomic targets identified from tissues of patients, against Crohn’s disease and ulcerative colitis. Under the terms of the agreement Celgene will pay milestones payments of up to $40 million in cash and receive rights to acquire Inception.

Merck & Co expands its current ongoing immuno-oncology collaboration:

• with Eli Lilly to conduct combination therapies studies with LY2835219 or abemaciclin and Merck’s Keytruda or pembrozilumab as a potential treatment for multiple tumors. Abemaciclib is a cell cycle inhibitor, designed to block the growth of cancer cells by specifically inhibiting CDK4 and CDK6. Pembrolizumab is a humanised monoclonal antibody that works as a checkpoint inhibitor by blocking the interaction between PD-1, a checkpoint which down regulates the immune system, expressed on T cells and its ligands PD-L1 and PD-L2.
• and with Amgen to evaluate the combination of Blincyto or blinatumab and Keytruda or pembrolizumab for the treatment of diffuse large B-cell lymphoma. Amgen’s Blincyto is a CD19 bispecific T cell engager (BiTE) that helps the body's immune system to detect and attack malignant cancer cells. Merck's Keytruda is a checkpoint inhibitor. 

Pfizer has entered an agreement with BioAtla (San Diego, CA, USA) to develop and market novel antibody therapies based on BioAtla Conditionally Active Biologics (CAB) and Pfizer’s antibody drug conjugate (ADC) platforms. The deal gives both parties a license to each other's respective technology to develop several CAB-ADC antibodies. Under the terms of the agreement Pfizer gains an exclusive option to develop and commercialise BioAtla CAB antibodies that target CTLA4. BioAtla and Pfizer will both receive milestone payments and royalties based on CAB-ADC antibody candidates developed and commercialised by the other party. BioAtla could receive up to $1 billion in upfront, regulatory and sales milestone payments as well as tiered royalties on net sales.

GlaxoSmithKline entered an agreement with Zymeworks (Vancouver, Canada) to develop and market novel Fc-engineered monoclonal and bi-specific antibody drugs produced by Zymeworks Effector Functionand Control Technology (EFECT) Platform.  Under the terms of the deal Zymeworks could receive as much as $110 million in milestones payment per product plus sales milestone and GSK will have the right to develop a minimum of 4 products across multiple disease areas.

Roche announces new agreements in oncology and immune-oncology with two Boston based companies:

• with SQZ Biotech in a deal worth up to $500 million, to develop SQZ’ CellSqueeze drug delivery system to boost the immune system of cancer patients. SQZ’ technology is based on engineering B cells where proteins are introduced into a patient’s cells to activate killer T cells and attack cancer cells.
• and with Pieris Pharmaceuticals to develop novel cancer immunotherapies based on the anticalin technology in a $409 million (CHF 415 million) deal. Under its first partnered immune-oncology agreement Pieris will discover and characterize novel anticalin-based drug candidates and Roche will advance them through preclinical and clinical development. Roche will pay up to CHF 415 million including CHF 6.5 million in upfront money for research funding, and payments tied to various milestones. Pieris is also eligible for escalating royalties on future product sales.

Huapont Life Science (Chongqing, China) has signed a non-binding agreement to acquire 70% stake in Swiss Biological Medicine Group which owns the hospital Paracelsus Suisse located in Saint Gallen (Switzerland).

Approval of drugs, vaccines, diagnostics and devices

World’s First Dengue Vaccine approved in Mexico

The Mexican Federal Commission for the Protection against Sanitary Risks (COFEPRIS) have granted marketing authorization to Sanofi Pasteur’s Dengvaxia, a tetravalent dengue vaccine, making it the first vaccine ever to be approved in the world, for the prevention of all four dengue virus serotypes in people 9 to 45 years of age but not yet in young children, one of the most at–risk population. ”This is a historic milestone for our company, for the global public health community and, most importantly, for half the world’s population who lives at risk of dengue” said Olivier Brandicourt, CEO of Sanofi. According to the WHO, dengue is the fastest growing mosquito-borne disease in the world today, causing nearly 400 million infections every year. Sanofi Pasteur expects 20 countries to approve the vaccine in coming weeks as it targets countries with the most cases.

US FDA approves:

• Baxalta’s (Bannockburn, Ill, USA) Vonvendi, the first and only engineered protein-based treatment, for the treatment of von Willebrand disease, the world’s most common inherited bleeding disorder.

• Alexion Pharmaceutical’s (Cheshire, Conn, USA) Kanuma, or sebelipase alfa, an enzyme-replacement, for the treatment of the ultra-rare genetic fatal lysosomal acid lipase (LAL) deficiency.The drug had received European approval in September (see newsweek #36 & 37). Kanuma is made using genetically engineered chicken, containing an rDNA construct responsible for producing recombinant human LAL in their egg whites and is only the second biological product produced by genetically engineered animals to be approved by the FDA.

• Roche’s Alencensa or alectinib, a drug with breakthrough status, for second line treatment of patients with anaplastic lymphoma kinase (ALK) positive non-small cell lung cancer. The approval comes three month after the agency had accepted the NDA for review and three months ahead of the PDUFA dates. Alectinib is an ALK inhibitor that Roche licensed from Chugai Pharmaceutical in Japan.

• Teva Pharmaceutical Industries’ injectable Bendeka, or bendamusine as a treatment for indolent B-cell non-Hodgin lymphoma and chronic lymphocytic leukemia.

Korea’s Ministry of Food and Drug Safety (MFDS) have approved Samsung Bioepsis’ Renflexis, a biosimilar version of Merck’s Remicade or inflimixab for the treatment of a range of autoimmune disorders. Renflexis is the second biosimilar from Samsung Biopsis, a JV between Samsung Biologics and Biogen, approved in South Korea. Both Renflexis and Brenzys are manufactured by Biogen in Hillerǿd (Denmark).

Drugs at clinical stage

US FDA grants:

• breakthrough status to Kite Pharma’s (Santa Monica, CA) KTE-C9 for the treatment of patients with aggressive and refractory diffuse large B-cell on-Hodgkin lymphoma.

• orphan status to Eiger BioPharmaceutical’s (Palo, Alto, CA, USA) ubenimex, a small molecule that acts as a dual inhibitor of aminopeptidase and leukotriene A4 hydrolase, for use in patients with pulmonary arterial hypertension. The drug, also known as Bestatin, is already approved in Japan as an adjunct to chemotherapy for acute non-lymphocytic leukemia.

• orphan status to PhaseBio Pharmaceutical’s (Malvern, PA, USA) injectable Vasomera for the treatment of cardiomyopathy associated to dystrophinopathies such as Duchenne muscular dystrophy, Becker muscular dystrophy and X-linked dilated cardiomyopathy. The drug which acts as a vasoactive intestinal peptide receptor 2 agonist, has also received orphan drug designation for the WHO group 1 pulmonary arterial hypertension.

• fast-track status to Acceleron Pharma (Cambridge, MA, USA) and Celgene’s luspatercept for the treatment of anemia in patients with myeolodysplastic syndromes. Luspatercept (ACE-536) is an investigational protein therapeutic that increases red blood cell levels by targeting molecules in the TGF-β superfamily. 

Novartis presented new results at the American Society of Haematology (ASH) meeting, with PKC412 or midostaurin, an oral multi-target kinase inhibitor, which increased survival by 23% in patients with newly-diagnosed FLT3 mutation-positive acute myeloid leukemia and with CART investigational candidate CTL019 which showed encouraging results in patients with relapsed/refractory non-Hodgkin lymphoma. The results of phase 2 study, found an overall response rate at three months of 47 % in diffuse large B-cell lymphoma and 73 % in patients with follicular lymphoma.

Gene Therapy News:

GlaxoSmithKline files for European approval for gene therapy targeting Wiskott-Aldrich syndrome (WAS), a rare disease in children. WAS is an X-linked recessive disease characterized by eczema, thrombocytopenia and resulting bloody diarrhoea, and immune deficiency. The WAS therapy comes as the second gene therapy project Glaxohas filed for approval in Europe with its partners Telethon (France) and San Raffaele (Italy) . 

Uniqure's Glybera did become the first gene therapy ever to get approved in October 2012 for treating adults with the ultra-rare disease familial lipoprotein lipase deficiency. But owing to several factors the first commercial patient was not treated until two months ago, and Uniqure has given up on Glybera in the United States. One of the major problems with Glybera is its cost, and it made headlines for being a therapy with a price tag of over $1million per treatment.  

University College of London Business has teamed up with Syncona, a private investment group, to launch a new biopharmaceutical company named Freelin Therapeutics which will focus on development of gene therapies for bleeding and other major disorders.

Eli Lilly stops the development of its long-acting insulin candidate peglispro which was dealing with liver toxicity issues and will take a $55 million charge in the fourth quarter of 2015. Lilly indicated that it will focus research and development efforts on other assets in its portfolio and pipeline such as its biosimilar of Sanofi’s Lantus which has a huge upside potential.

Merck KGaA does no longer intend to file for approval of evofosfamide, an investigational hypoxia-activated prodrug of a bis-alkylating agent, in advanced soft tissue sarcoma and advanced pancreatic adenocarcinoma, after two Phase 3 studies failed to meet their primary endpoints. Merck said that resources be redeployed into other high-profile future products, such as avelumab and all other priority programs in oncology, immuno-oncology and immunology.

Science and Technology

According to Danish researcher Frank Aastrup, the superbug identified in Chinese hospital has already gone global. He found the mcr-1 gene in one person in Denmark and in 5 samples from poultry meat imported from Germany.

Scientist at Blood Systems Research Institute (BSRI) in San Francisco and Washington University School of Medicine in Saint Louis, with Dr. Graham Simmons as lead researcher of the project, have identified two human neutralizing antibodies that engage epitopes including residue E2-W64 which are highly potent at inhibiting the chikungunya virus (CHIKV) at multiple stages of infection. These antibodies prevent CHIKV from both entering and exiting cells and the approach could eventually lead to a single vaccine that protects against the CHIKV as well as other viruses.

Medical Devices and Diagnosis News

Medtronic and Samsung partner to develop neuromodulation applications for ubiquitous consumer smart devices as an extension to their previous deal in diabetes. The project aims at enabling physicians and patients to use Android neuromodulation devices, which often come with dedicated controllers, to more easily manage therapy, track symptoms and wirelessly monitor data. The neuromodulation brought sales of $485 million for Medtronic last quarter.

Cost, Pricing and Market Access

NICE : 

Recommends AstraZeneca’s Lynpraza, or olaparib, as the first cancer drug for the maintenance treatment of relapsed platinum sensitive ovarian fallopian tube and peritoneal cancer in people who are positive for BRAC1 or BRAC2 mutation. The recommendations also stipulate that Lynparza should be available for people only if they have had three or more courses of platinum-based chemotherapy, and that the drug cost for people who remain on treatment after 15 months is met by the company.

and Astellas’ Xtandi or enzalutamide for the treatment of prostate cancer in people whose first treatment has failed.

….But rejects Janssen’s Zytiga or abiraterone for the same indication. NICE indicated that the drug is a well-tolerated treatment that delays chemotherapy, but it was not convinced that the “uncertain evidence” for long-term benefit justified accepting estimates of cost-effectiveness that were significantly above what it would normally consider acceptable. Zytiga’s incremental cost-effectiveness ratio compared with best supportive care was calculated to be between £35,500 and £59,600, while that for Xtandi came in at £34,500.

Praluent versus Repatha: Round 4 & 5 for Sanofi/Regeneron.  Praluent makes it as the preferred product with UnitedHealth Group’s OptumRx and United Healthcare.Praluent is now on formularies covering more than 100M patients in the US.

Miscellaneous

Japan’s Ministry of Health Labor and Welfare suspends Chemo-Ser-Therapeutic Research Institute known as Kaketsuken for acknowledged violations and a massive cover-up as far back as 1974. The company is alleged to have used additives that had not been approved, changed a heating process without authorization and used expired raw materials .The government discovered also unauthorized production methods during an inspection last May. Seiji Miyamoto, the managing director resigned just before the panel's report was revealed. Board members also either resigned or were demoted.

Professor Guido Rassi, who took office as European Medicines Agency (EMA) executive director on November 16, has outlined his plan for the next five years. He said: "I am confident that EMA, working closely with the national competent authorities in Member States, is ready to successfully address these new challenges; He highlighted five building blocks on which EMA's response to these shifts would be built

–      Focusing on research and development for medicines that address public health needs 

–      Commitment to transparency, as the “first regulator in the world to allow researchers and academics, and the public as a whole, access to the clinical data on which marketing authorisations are based"

–      Patient involvement: "All that we do must ultimately benefit patients,” he said

–      Best use of all available evidence, including a focus on the “impact of medicines in real life”

–      Global reference authority for the regulation of medicines; with development and manufacturing of medicines now global, regulatory authorities cannot work in isolation, he said. “We are reinforcing our role as a global reference authority, to provide the regulatory oversight that our citizens expect."

He also stressed the importance of strengthening cooperation between Member States, the European Commission and other European and international partners, as well as involving communities and stakeholders for a more holistic approach to medicines evaluation and surveillance across the whole lifespan of a medicine.

Democratic Presidential candidate Hillary Clinton plans to force US companies moving their headquarters overseas to pay an “exit tax” which would tax foreign earnings at the time of the inversion deal.

MedTech Europe, the main industry association for the medical device industry, joins the movement to stop the industry sponsorship of conducting medical educations for physicians. The association outlined other ways the company could support medical education through grants and other fundings.

Alphabet Google Life Science s has been renamed “Verily” a word with 13^th century English origin that means “truly” or “certainly”.

Bioevents

• BIOTech ShowCase 2016 on January 11-13 in San Francisco (USA)
• JP Morgan 33^rd Annual Healthcare Conference on January 12-16 in San Francisco (USA)
• BIO CEO Investor Conference on February 8-9 in New York City (USA)
• 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)
• 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)
• Strategic Alliance Management Congress on May 16-18 in Philadelphia (USA)
• China BIO Partnering on May 18-19 in Suzhou (China)
• BIO International Convention on June 6-9 in San Francisco (USA)

And recently announced:

• BIO Pharm America on September 13-15 in Boston (USA)
• 7^th Innovation Days on October 3-4 in Paris (France)
• Biofit 2015 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, Special Advisor at I&IR, jcm@btobioinnovation.com

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