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

Mergers, Acquisitions and Joint Venture

Thermo Fisher (Waltham, MA, USA) acquires Patheon (Durham, NC, USA), a contract development and manufacturing company for $7.2 billion and becomes a highly competitive biologic contract development and manufacturing organization (CDMO) through direct access to its bioprocessing technologies and capabilities.

According to the Sunday Times, GlaxoSmithKline is strongly considering exercising its option to buy out Novartis 36.5% stake in their joint-venture consumer health business for $10.3 billion. Buying out Novartis would give GSK control of the biggest consumer health operations on the globe. GSK has "been consistent all along that this is a business they want to own, and will move forward when the time is right,” one investor told the Sunday Times.

Business

Boehringer Ingelheim extends its business in China through:

the opening of a contract manufacturing plant in the Zhang Jiang Hi-tech Park in Pudong (Shanghai) housing 2,000L of disposable bioreactor capacity to produce therapeutic proteins from mammelion cell culture

….and an agreement with Peking University to identify targets and develop new drugs for cancer, cancer immunology and immune modulation, respiratory conditions and cardiometabolic and central nervous system diseases, as well as gene therapies and regenerative drugs. The collaboration is Boehringer's second recent research partnership with a Chinese university. In November 2016, Boehringer collaborated with the China Southeast University Institute of Life Sciences (Nanjing, China) to develop regenerative therapies to treat hearing loss The partnership is part of Boehringer's Research Beyond Borders (RBB) program, designed to incubate promising technologies that could complement the company's core therapeutic areas, but do not yet fit into any of its ongoing programs.   Peking University has recently partnered with Bayer in establishing a joint research center for translational research and drug discovery and with Johnson & Johnson to identify agonists and antagonists for G protein-coupled receptors (GPCRs) to treat CNS diseases.

Biogen licenses Cirara, a late-stage stroke drug from Remedy Pharmaceuticals (New York City, NY, USA), for $120 million. Cirara is an intravenous formulation of glyburide, an established oral treatment for type 2 diabetes, which had mixed success in a Phase 2 trial, managing a 25% drop in neurological death against a 7% reduction with placebo, but missing its primary endpoint of reducing the need for surgical intervention. The FDA has approved a Phase 3 trial of Cirara and awarded the drug orphan Fast track status. If approved Cirara would be the first new stroke treatment since ateplase, almost twenty years ago, which is still the only approved drug for the condition.

Amazon is looking to hire a general manager to enter into the pharmacy market through a e-commerce company.

Approval of drugs and vaccines

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the marketing authorization of Sanofi’s insulin lispro 100 Units/m, a biosimilar insulin, to treat adults and children who have diabetes and need insulin to keep their blood sugar level controlled

The US FDA approves Merck’s Keytruda, or pembrozulimabn as a first-line treatment for patients with locally advanced/metastatic urothelial carcinoma who are not eligible to cis-platinum containing chemotherapy and as a second-line treatment in patients with advanced/metastatic urothelial carcinoma whose disease progressed with chemotherapy.

The China FDA (CFDA) has posted a series of new proposals to increase new drug approval.  One of the key feature consists in the offering to grant “conditional marketing authorization” for orphan drugs, which are already approved in Europe or the US without in-China clinical trial data. The CFDA is also offering to grant conditional approval for drugs that treat life-threatening conditions where significant unmet medical needs exist, if early- or mid-stage data can predict the drugs’ clinical benefits. This policy that resembles the EMA's “conditional marketing authorization” and the US FDA’s “breakthrough therapy” program.

Drugs at clinical stage

The US FDA grants breakthrough status:

to Ignyta’s (San Diego, CA, USA) entrectinib, as a treatment of NTRK fusion-positive tumors.

….and to Synthetic Biologic’s (Rockville, MD, USA) SYN-004, or ribaxamase, for the prevention of Clostridium difficile infection. Ribaxamase is an orally-delivered beta-lactamase intended to degrade penicillins and cephalosporins within the gastrointestinal tract to protect the microbiome

The US FDA grants rare pediatric disease status to Imara’s (Cambridge, MA, USA) IMR-687, as a treatment for a sickle cell disease, a severe, genetic form of anemia, in which a mutated from of hemoglobin distorts the red blood cells at low oxygen levels into sickle, or crescent-shaped cells. IMR-687 is an orally highly potent and selective phosphodiesterase 9 (PDE9) inhibitor potentially acting as a disease-modifying therapy.

The US FDA grants fast-track status:

to Sangamo Therapeutics’ (Richmond, CA, USA) SB-525 a gene therapy as a treatment of patients with hemophilia A. Sangamo entered a partnership last week with Pfizer to develop the drug.

….and to Sage Therapeutics’ (Cambridge, MA, USA) SAGE6217 as a treatment for major depression. SAGE-217 is an orally-active, second-generation neuroactive steroid that acts as a positive allosteric modulator of synaptic and extrasynaptic GABA_A receptor subtypes.

The US FDA grants orphan status

to Cytokinetic’s (South San Francisco, CA, USA) CK-2127107, a skeletal troponin activator, as a treatment for patients with spinal muscular atrophy (SMA). The drug is being co-developed with Astellas Pharma for the treatment of SMA, chronic obstructive pulmonary disease and certain other diseases associated with skeletal muscle weakness and/or fatigue.

…and to Loxo Oncology’s (Stamford, CN, USA) LOXO-101, or larotrectinib, as a treatment for patients with solid tumors who have NTRK-fusion proteins or rare genetic abnormalities. LOXO-101 inhibits a group of enzymes called tropomyosin receptor kinases (TRK) that play a key role in cellular communications.

Nanobiotix (Paris, France) announces first positive human data showing the potential ability of NBTXR3 to transform “cold” tumors into “hot” tumors and thus could become a backbone in immuno-oncology. Many tumors exhibit little or no response to therapies targeting the immune system and are considered “cold”.  The explanation for the lack of response in its simplest form is a lack of immunogenicity.  The ability of NBTXR3 to generate intratumoral immunogenic cell death (ICD) could be a key to significantly increase the number of patients who can engage their immune system to fight their cancer.  NBTXR3, is a first-in-class radio-enhancer nanoparticle designed for direct injection into cancerous tumors. It has been engineered to increase the dose and efficacy of radiotherapy without increasing toxicity or causing damage to surrounding healthy tissues. NBTXR3 is currently in late-stage clinical development as a single agent. 

Eli Lilly’s Galcanezumab, a CGRP inhibitor significantly reduced the number of “migraine days” in three late-stage clinical trials.  Lilly is competing with Amgen and Novartis with a similar CGRP drug.

Polyphor (Allschwil, Switzerland) announces successful end of phase 2 meeting with the US FDA for Murepavadin as a treatment for nosocomial pneumonia (Ventilator-associated and hospital-acquired pneumonia). Murepavadin or POL708, is the first representative of the Outer Membrane Protein Targeting Antibiotics (OMPTA) class, a class of antibiotics against Gram-negative bacteria with a novel mode of action.

CAR-T Cell Therapies work by genetically engineering T-cells to produce chimeric antigen receptors (CARs) on their surface. These cells are enhanced to have cancer-killing ability recognizing a target expressed (such as CD19) on tumor cells. An impressive number of treatments using this technology are already being investigated in several clinical trials.

Selected CAR-T therapies in clinical development (Source: Novartis)

Science & Technology

Médecins Sans Frontières, an international charity organization, said it is in talks with WHO to consider Merck & Co’s Ebola vaccine as a valuable vaccine candidate against a new outbreak of the Zaire Ebola strain in Africa. Meanwhile, international vaccine alliance Gavi has purchased 300,000 vaccine doses for stockpiling in case of an Ebola emergency.

Cost, Pricing and Market Access

Aspen Pharmacare (Durban, South Africa) is under investigation by the European Commission over its pricing practices for five cancer medicines. EU antitrust regulators will investigate whether the South African generics firm abused a dominant market position for the niche medicines by price gouging. This is the first time the Commission has investigated concerns about excessive pricing practices in the pharmaceutical industry and will cover reported increases of ‘several hundred percent’. Commissioner Margrethe Vestager, in charge of competition policy, said: "When we get sick, we may depend on specific drugs to save or prolong our lives. Companies should be rewarded for producing these pharmaceuticals to ensure that they keep making them into the future. “But when the price of a drug suddenly goes up by several hundred percent, this is something the Commission may look at. More specifically, in this case we will be assessing whether Aspen is breaking EU competition rules by charging excessive prices for a number of medicines." The five generic medicines involved – chlorambucil, melphalan, mercaptopurine, tioguanine and busulfan – are used for treating a broad range of cancers, including hematologic tumours. The investigation will cover all countries in the European Economic Area with the exception of Italy, which fined the company €5 million last September after Aspen increased its prices for treatments for onco-haematological patients by up to 1,500%

Miscellaneous

Henri Termeer, the founder of Genzyme, died last Friday, at the age of 71, at his Marblehead home in Massachusetts.

Johnson & Johnson has launched eleven new drugs over the last six years and expects to double this figure with at least eleven late stage drugs in its pipeline with a peak sale potential of $1 billion. Bill Hait, global head of R&D, said he ex­pects 14 new meds to ar­rive in next five years, with 50 line ex­ten­sions on al­ready ap­proved ther­a­pies.

Teva is laying off 500 people at its sterile injectable plant in Godollo, near Budapest (Hungary), that halted production last year after the US FDA identified major manufacturing failings. The announcement comes just weeks after Israeli media reported that the financially struggling and management-challenged drug company was looking to cut up to 6,000 jobs.

Novartis reduces its work force at its Basel campus by 500 jobs mainly in drug de­vel­op­ment, “tra­di­tional” man­u­fac­tur­ing and co­or­di­na­tion group, with the staffers ei­ther being laid off, re­tir­ing early or mov­ing to other lo­ca­tions. At the same time, the company announces the hiring of 350 new em­ploy­ees for “high-tech” po­si­tions in Switzer­land, also for drug de­vel­op­ment as well as “in­no­v­a­tive” bi­o­log­ics man­u­fac­tur­ing. No­var­tis has started the di­a­logue and con­sul­ta­tion with its em­ployee rep­re­sen­ta­tives in Switzer­land.

People on the move

According to CNBC Verily is courting former US FDA commissioner Robert Califf to joint the company in a leadership position.

Bioevents

2017 BIO-IT World Conference & Expo on May 23-25 in Boston (USA)

9^thAnnual China BIO International Partnering Forum on May 31-June 1 in Zhuhai (China)

BIO International Convention on June 19-22 in San Diego (USA)

5^thAntibody Industrial Symposium, on June 27-28, in Tours (France)

MedFIT on June 28-29 in Grenoble (France)

Nordic Life Science Days (NLS) 2017 on September 12-14 in Malmö-Copenhagen (Sweden and Denmark)

BIOPharm America on September 26-27 in Boston (USA)

BIO Latin America on October 26-28 in Sao Paulo (Brazil)

BIO Europe on November 6-8 in Berlin (Germany)

World Congress on Biomedical Engineering 2017 on November 9-11 in Xian (China)

BIOFIT on November 28-29 in Strasbourg (France)

http://btobioinnovation.com/events/pharma-biotech-events/

http://btobioinnovation.com/events/in-vitro-diagnostic-events/

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