Biopharmaceutical News Week # 2016.41

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

Editor’s Note

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Zika virus

Florida officials have announced a new area of Zika transmission in Miami, which has already infected five individuals. It is the third established Zika outbreak in Miami, because it has both the Aedes mosquitoes that transmit the disease, and an influx of travelers from other areas where the virus is spreading.

According to recent research people infected with Zika virus will likely not be susceptible again. The same report indicates that Zika RNA was detected in blood plasma as early as one day after virus infection. It also was detected in saliva, urine, cerebrospinal fluid and semen, and was briefly detected in vaginal secretions. Zika RNA was cleared from blood plasma and urine within 10 days, but viral RNA was detectable in saliva and seminal fluids until at least three weeks after Zika virus was no longer present in the blood.

Business

Kymab (Cambridge, UK) signs a cross-licensing deal with EpimAb Therapeutics (Shanghai, China) to develop bispecific therapeutic antibodies for multiple immune-oncology targets. Both companies plan to combine antibodies issued from Kymab's proprietary Kymouse platform with EpimAb's proprietary Fabs-In-Tandem Immunoglobulin platform to generate multiple bispecific antibodies. Under the terms of the deal, Kymab will have development and commercialisation rights to these bispecific antibodies in all geographical regions outside of China, and EpimAb will have the rights to the Chinese market. Financial terms were not disclosed.

AstraZeneca grants exclusive rights to 3SBIO (Hong Kong, China) to market diabetes treatments Byetta and Bydureon, or exenatide in China. Under the terms of the deal AstraZeneca will receive an upfront payment of $50 million and another $50 million in milestone fees

Transgene (Strasbourg, France) enters into a collaboration with Pfizer and Merck KGaA to evaluate the combination of TG4001 with Avelumab, a PD-L1 IgG1 monoclonal antibody, in human papilloma virus (HPV)-positive head and neck squamous cell carcinoma after failure of standard therapy.

Takeda enters into a collaboration with Crescendo Biologics (Cambridge, UK) to discover immuno-oncology therapies as well as drug conjugates based on Crescendo’s Humabodies. Humabody products are a novel class of small, robust and potent proteins which can rapidly penetrate and accumulate in tumors whilst clearing quickly from circulation to minimize systemic toxicity. These novel therapeutics have different pharmacological properties to antibodies. Under the terms of the deal Takeda will make a $36 million upfront payment and research fees and additional milestones payment of up to $754 million as well as sales royalties.

Approval of drugs and vaccines

US FDA approves Lundbeck’s (Copenhagen, Denmark) Carnexiv, the first intravenous formulation of carbamazepine for the treatment of epilepsy, as a short replacement therapy for oral forms.

…and Protein Science’s (Meriden, CT, USA) quadrivalent formulation of Flubok influenza vaccine, which contains three times more active ingredient compared with other quadrivalent influenza vaccines.

Drugs at clinical stage

US FDA grants orphan status to Theranexus’ (Orsay, France) THN102 in the treatment of narcolepsy

US FDA grants priority review:

to Janssen and Genmab’s Darzalex or daratumumab, in combination with Revlimid or lenalidomide and dexamethasone for the treatment of multiple myeloma.

to Genentech’s Lucentis, or ranibizumab for the treatment of myopic choroidal neovascularisation (mCNV), a complication of severe near-sightedness that can lead to blindness. The approval would make Lucentis the first anti-vascular endothelial growth factor (VEGF) therapy approved to treat mCNV.

…and to Neurocrine’s (San Diego, CA, USA) Ingrezza or valbenazine, a highly selective vesicular monoamine transporter 2 inhibitor, as a treatment of tardive dyskinesia. Tardive dyskinesia is characterized by involuntary, repetitive movements of the face, trunk, or extremities, including lip smacking, grimacing, tongue protrusion, facial movements, blinking, puckering, and pursing of the lips. All these symptoms are rarely reversible.

US FDA grants fast track as well as qualified infectious disease product designation to Viamet Pharmaceuticals’ (Research Triangle, NC, USA) VT-1161, a CYP51 inhibitor, for recurrent vulvovaginal candidiasis.

Merck’s Keytruda (pembrolizumab) significantly reduced the risk of disease progression by 50% and the risk of death by 40% among non-small cell lung cancer patients expressing PD-L1 levels of 50% or greater. Median progression-free survival for patients on Keytruda came in at 10.3 months, versus 6 months for those on chemotherapy. At the same time Bristol-Myers Squibb indicated that a subgroup analysis of Opdivo, in first line non-small cell lung cancer patients, was not superior to chemotherapy. Since the first announcement, in August, of negative results in another subgroup of patients the stock value of BMS has dropped by 33%.

Novartis’ LEE011, or ribociclib, has demonstrated superiority over standard of care letrozole as a first line treatment for HR-positive, HER-2 negative advanced or metastatic breast cancer.

Exelixis’ (South San Francisco, CA, USA) Cabometyx, or cabozantinib, compares favorably with Pfizer’s Sutent, or sunitinib, in the treatment of metastatic kidney cancer.

Medical Devices and Diagnosis News

US FDA approves St Jude Medical’s (Saint Paul, MN, USA) Pressure WireX Guide Wire that measures fractional flow reserve (FFR) helping them gauge how severely narrowed a patient’s arteries are. It is designed to improve percutaneous coronary intervention procedures in patients with narrowed coronary arteries.

St. Jude Medical is warning of a possible problem with a lithium-based battery used in some of its high-voltage cardiac rhythm management (CRM) devices that could result in a short circuit. The problem is connected to two deaths out of almost 400,000 patients who have had the devices implanted. There is a risk the batteries used in the devices could produce “lithium clusters” during high-voltage charging. The company said pacemakers produced by the company aren’t affected by this warning because they operate on low-voltage batteries.

Abbott will work with Celgene and Agios Pharmaceuticals (Cambridge, MA, USA) to develop tests to identify isocitrate dehydrogenase (IDH) mutations in individuals with acute myeloid leukemia in order to establish if they respond to Abbott’s new treatment for the disease.IDH1 and IDH2 mutations appear in 20% of AML patients and having a tool that could identify them sooner could improve treatment.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence :

recommends the use of Celgene Otezla for the treatment of psoriatic arthritis when their disease has not responded to previous treatments. Otezla's NHS list price is £265.18 for a 14-day treatment initiation pack and £550.00 for a 28-day pack. The drug was approved in Europe in January 2015

recommends the use of AstraZeneca’s Forxiga or dapagliflozin for the treatment of type 2 diabetes as part of a triple therapy. Two other medicines, Jardiance (empagliflozin) and Invokana (canagliflozin), are already routinely available on the NHS options for triple therapy.

….and turns down Janssen’s Imbruvica for treating people with Waldenstrom’s macroglobulinaemia, a rare type of non-Hodgkin’s lymphoma, noting that there was no cost-effectiveness evidence submitted for some part of the population.

The UK National Institute for Health and Care Excellence and NHS England are proposing to introduce a 'fast-track' appraisal system designed to accelerate patient access to new technologies Under the plans health technologies likely to have a cost per QALY (quality adjusted life year) of up to £10,000 would be assessed more quickly under a 'lighter touch' process. Patients could benefit from 'fast-track' treatments much more quickly than is currently the norm as final guidance would be made available within 30 days as opposed to the current 90 days.

Drug price increases are hitting US hospitals even harder than consumer. According to a study by the American Hospital Association, the nation's biggest hospital lobby, and the Federation of American Hospitals, annual inpatient drug spending was up an average of 23.4% between 2013 and 2015, and 38.7% on a per admission basis. Although blockbuster drugs tend to get most of the attention, extreme price hikes were observed in both high-volume drugs used frequently in hospitals as well as low-volume drugs, both generic and brand-name. Most of the drugs reviewed in the sample were non-innovator drugs, yet, about half had no generic competition.

Following a similar recommendation from Democratic nominee Hillary Clinton, Connecticut Representative, Rosa DeLauro plans to introduce a bill next month to set up a “national review board” that would punish “excessive” price hikes by issuing fines, shortening monopolies and clawing back previous tax benefits. The board would include representatives from multiple government agencies who would watch over both pharma and medical device prices. As DeLauro puts it, companies benefit from “taxpayer-sponsored research, our skilled workforce, our infrastructure, and tax incentives, only to then gouge those same taxpayers–putting life-saving medicine out of reach for many.”

International Charity organization Médecins Sans Frontières (MSF) has turned down Pfizer offer of a million free doses of Prevnar although the charity has urged Pfizer for years to cut the prices of its pneumococcal vaccine. U.S. Director Jason Cone said “free is not always better” and indicated that the conditions that come with such donations can delay vaccination campaigns and “undermine long-term efforts to increase access, ….including other humanitarian organizations.” Pfizer’s Prevnar 13 was the world’s bestselling vaccine last year. MSF’s decision follows a move by GSK last month to lower the price of its own pneumococcal vaccine, Synflorix, for refugees

Miscellaneous

US FDA has issued a warning letter to Japanese Nippon Fine Chemical for obstructing an inspection at its plant. The FDA said that during an attempted inspection in the firm "limited and/or refused an FDA inspection." Investigators were limited in their access to the facility located in Takasago City, and at one point an NFC manager directed employees to form a human barrier by standing shoulder to shoulder to block access to areas of the laboratory and equipment used to analyze drugs destined for U.S. distribution. Additionally, the company refused to provide copies of documents that included customer complaints of drugs it had manufactured. These complaints said the drugs contained glass, hair, cardboard, metal, product discoloration and in one case, a black spider. The company also prevented the inspector from taking photographs of equipment at the facility.

On its annual list of the world’s 100 top CEO’s Harvard Business Review nominated Novo Nordisk CEO Lars Rebien Sørensen as N°1 for the second year in a row.HBR analyzes long-term CEO performance, measuring returns, sustainability and governance over their entire tenure at the helm

Bioevents

15^th Annual BIO Investor Forum on October 18-19 in San Francisco (USA)

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)

9^th Biotech Showcase Investor Conference on January 9-11, 2017 in San Francisco (USA)

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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