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

Zika virus

Engineers from the University of Pennsylvania have created a $2 genetic color test to identify the Zika virus which can be used in the field. The current test requires laboratory equipment." We engineered a low-cost, point-of-care system that consists of a diagnostic cassette and a processor,” said Changchun Liu, a research assistant professor in the Department of Mechanical Engineering and Applied Mechanics in the statement. “The cassette isolates, concentrates and purifies nucleic acids and carries out enzymatic amplification. The test results are indicated by the change in the color of a dye, which can be inspected visually." Once a saliva sample is put in the cartridge, the test takes 40 minutes.

Sanofi Pasteur enters a partnership with US Army to expand R&D of an experimental Zika vaccine which could be tested in humans in the coming months.

Acquisitions /mergers/joint-ventures

Bristol-Myers Squibb acquires Cormorant Pharmaceuticals (Stockholm, Sweden) for up to $520 million and expands its oncology pipeline with the rights to HuMax-IL8 antibody program and lead candidate HuMax-IL8, a monoclonal antibody targeted against interleukin-8 (IL-8).Targeting IL-8, offers the potential to enhance immune response and increase the efficacy of existing cancer medicines through combination therapy.

Sanofi hostile bid over Medivation is over. After several months of struggle Medivation has invited a “number of parties” of potential buyers through confidential agreements including Sanofi, which has agreed to drop its campaign to replace the current Medivation board members.

Business

Johnson & Johnson has exercised its option to license FR104, a CD28 antagonist, from OSE Immunotherapeutics (Nantes, France) and commits to pay up to €155 million for the global rights to the drug for use in autoimmune diseases and transplantations. CD28 antagonism can inhibit the proliferation of T cells, the destructive actions of effector memory T cells and the synthesis of cytokines.

Takeda Pharmaceutical announces a definitive agreement with Altos Therapeutics (Los Altos, CA, USA) to develop ATC-1906 as a treatment of gastroparesis and its symptoms. Gastroparesis is a chronic gastric motility disorder characterized by delayed gastric emptying. Symptoms include early satiety, post-prandial fullness, nausea, vomiting, and abdominal discomfort. Diabetic gastroparesis and idiopathic gastroparesis have high unmet need. Under the terms of the agreement Takeda will make an undisclosed upfront payment and get the exclusive option to acquire Altos when early stage clinical trials are completed.

….and acquires non-US rights to TiGenix’s (Leuven Belgium) Cx601, a stem cell treatment for complex fistulas in Crohn’s disease patients. Cx601 is a suspension of allogeneic adipose-derived stem cells injected intralesionally. TiGenix already submitted a marketing authorization application to the European Medicines Agency for Cx601. Under the terms of the agreement, Takeda will make an upfront payment of $28 million and pay up to $396 million in milestone fees, plus double-digit royalties.

AstraZeneca pays Fibrogen (San Francisco, CA, USA) $62 million under the 2013 collaboration agreement to develop roxadustat or FG-4592, an orally active small molecule inhibitor of hypoxia-inducible factor (HIF) prolyl hydroxylase, as a treatment of anemia in chronic kidney disease. HIF is a protein transcription factor that "turns on" the production of red blood cells (erythropoiesis).

Approval of drugs and vaccines

The European Commission approves:

AbbVie and Biogen’s Zinbryta or daclizumab, as a treatment for relapsing forms of multiple sclerosis. Zinbryta has an immunomodulatory mechanism of action that regulates inflammation without broadly depleting the immune system, and immune cell effects are reversible within six months. Zinbryta blocks the activation of autoreactive T-cells, a major contributor to inflammation in the nervous system of people with MS.

Bial’s (Porto, Portugal) Ongentys or opicapone, as an adjunctive therapy to preparations of levodopa/DOPA decarboxylase inhibitors in Parkinson’s disease patients who cannot be stabilised on this combinations. Opicapone is a long-acting, peripherally selective inhibitor of catechol-O-methyltransferase.

Amgen’s Kyprolis, or carfilzomib, as an extended indication along with dexamethasone for the treatment of multiple myeloma. The drug was previously approved in combination with dexamethasone and Revlimid, or lenalidomide, as a treatment for the disease.

Shire’s Restive, or teduglutide, a glucagon-like peptide-2 (GLP-2), for the treatment of short bowel syndrome in children, a rare but potentially life-threatening gastrointestinal condition.

….and Takeda and Seattle Genetic’s Adcetris, or brentuximab vedolin, as a treatment of CD-30 positive Hodgkin lymphoma with high risk or relapse or progression after autologous stem cell transplant.

US FDA approves AbbVie’s Humira or adalimumab for the treatment of non-infectious intermediate, posterior and panuveitis. Non-infectious uveitis is an immune-mediated disease that can flare and impair vision. Humira works by targeting and blocking tumor necrosis factor alpha, a source of inflammation that can play a role in uveitis.

The Japanese Ministry of Health and Welfare (MHLW) approves:

Sanofi and Regeneron’s Praluent or alirocumab for the treatment of hypercholesterolemia patients with a high risk of cardiovascular events. This is the second PCSK9 inhibitor approval in the country following Repatha or evolocumab from Astellas and Amgen.

…and Kyowa Hakko Kirin (Tokyo, Japan) and Amgen’s Lumice or brodalumab for the treatment of psoriasis.

Drugs at clinical stage

US FDA grants orphan status to Five Prime Therapeutics (South San Francisco, CA, USA) FPA144 for the treatment of gastric cancer and gastroesophageal junction in patients whose tumors overexpress the FGFR2b protein.

US FDA grants fast-track status to Merrimack Pharmaceuticals’ (Cambridge, MA, USA) MM-121, or serinatumab, for patients with heregulin-positive locally advanced or metastatic non-small-cell lung cancer in combination with docetaxel or pemetrexed.

US FDA puts a hold on Juno Therapeutics’ (Seattle, WA, USA) JCAR015, a CAR-T immunotherapy drug targeting CD19, after the death of two patients in a phase 2 clinical trial in patients with relapsed or refractory B cell acute lymphoblastic leukemia

Pfizer’s Sutent, or sunitinib malate, was successful in a late stage phase 3 clinical trial for the adjuvant treatment of renal cell carcinoma patients at high risk of recurrence after surgery. Sutent, a pan-tyrosine kinase inhibitor, was the first cancer drug simultaneously approved for two different indications, by the US FDA back in 2006,

Medical Devices and Diagnosis News

US FDA approves Abbott’s Vascular Absorb GT1 a bioresorbable vascular scaffold system, as the first full absorbable stent to treat coronary artery disease. The GT1 releases Novartis’ everolimus which limits the growth of scar tissue and is gradually absorbed by the body in about three years.

UK National Institute for Health and Care Excellence (NICE) has published a new quality standard that aims to speed up cancer diagnosis. The guidelines recommends that GPs have direct access to tests such as endoscopy, ultrasound, MRI, X-ray and CT for people with suspected cancer, to help speed up the diagnostic process. There also needs to be better access to upper gastrointestinal endoscopy for suspected stomach and oesophageal cancer, and patients who don't meet the referral criteria for suspected colorectal cancer should have a faecal blood test.

Science and Technology

Scientists at the Johns Hopkins University have shed light on the relationship of the build-up of tau protein and the accumulation of beta-amyloid in brain nerve cells. The new study shows that beta-amyloid buildup “is insufficient” to cause tau to “convert” from a normal state into an abnormal state in which it accumulates. Instead, it indirectly affects tau buildup by bringing about a “chain of chemical signaling events” that leads to its “conversion” into its clumping state. “For the first time, we think we understand that the accumulation of amyloid plaque alone can damage the brain, but that’s actually not sufficient to drive the loss of nerve cells or behavioral and cognitive changes,” Wong said in a statement. “What appears to be needed is a second insult–the conversion of tau–as well.”

Scientists from Brigham and Women Hospital at Harvard Medical School have found that two types of bacteria, Methanobrevibacter and Akkermansia, present in the gut microbiome are involved in inflammatory diseases and autoimmune disorders such as multiple sclerosis and thus offer new ways to diagnose and potentially treat the disease.

An Australian team of researchers at the Walter and Eliza Hall Institute of Medical Research (Victoria, Australia) led by Alan Cowman has made further progress towards the discovery of a malaria vaccine in blocking three proteins (Rh5, Ripr and CYRPA) necessary for infection in the Plasmodium falciparium parasite. The three proteins form a complex that is “essential in the sequential molecular events leading to parasite invasion.” These findings published in Cell Host & Microbe “hold great promise for understanding the function of these proteins and their development as vaccines,” Cowman said in a statement.

Cost, Pricing and Market Access

UK National Institute for Health and Care Excellence (NICE) recommends Ferring’s Firmagon, or degarelix, for the treatment of hormone dependent prostate cancer and spinal metastases. Clinical studies showed the drug improved progression-free survival, compared with luteinizing hormone-releasing hormone agonists. The drug was actually first approved by the European Medicines Agency in 2009 and has been available in the UK since 2010.

Top executives at Eisai are considering legal action against NICE for not including Lenvima, a thyroid cancer treatment, for coverage and delaying a reconsideration. Lenvima was approved by the European Commission in May 2015, but it will not receive a recommendation from the NICE until at least April 2017. "Eisai may be forced to consider its level of investment in the UK because it is unacceptable that drugs which are manufactured in England cannot be provided to people in England, without delays of many years," Gary Hendler, Eisai EMEA head, said

Miscellaneous

US healthcare industry added 38,500 jobs in June and a total of 234,600 since January 2016, according to the initial seasonally adjusted figures from the US Bureau of Labor Statistics. Hospitals have created 89,800 jobs for the first six months and 15,000 in June.

Novo Nordisk expands its Active Product Ingredient (API) facility in Kalundborg (Denmark) to make it the world largest insulin plant. The expansion is set to be fully operational in 2018. Novo said the 1.1-million-square-meter plant currently has 3,400 employees and produces 50% of the world's insulin. “The new facilities will give us greater flexibility and enable the installation of equipment that will enhance efficiency and increase the long-term production capacity of the plant," Michael Hallgren, senior VP and head of production in Kalundborg, said in a statement. In March of this year Novo kicked off construction of its first US insulin API plant, an $1.8 billion project in Clayton, NC.

One week after Astellas was suspended for one year from the Association of the British Pharmaceutical Industry (ABPI) following breaches of the Code of Practice, AstraZeneca and Genzyme have been named and shamed in advertisement for breaches of the Code of Practice. AstraZeneca  breached three clauses of the Code by producing a leavepiece which provided misleading instructions on how to use the EMIS Web clinical system – which allows healthcare professionals to record, share and use vital information – such that controlled type 2 diabetic patients might be inappropriately treated with its Forxiga (dapagliflozin). Genzyme was found be in breach of a stream of Code clauses after making a presentation about Fabrazyme (agalsidase beta) to an expert advisory group, that the PMCPA says was, "misleading, inconsistent with the Fabrazyme SPC and disparaging of a competitor product". The complaint was logged by Shire, which took issue over material used by Genzyme compared Fabrazyme with its Replagal (agalsidase alfa), both of which were indicated for long-term enzyme replacement therapy in patients with confirmed diagnosis of Fabry Disease.

Bioevents

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)

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