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

Business

Sanofi Pasteur and MedImmune collaborate to develop and commercialize MEDI8897 for the prevention of Respiratory Syncytial Virus (RSV) associated illness in newborns and infants in a deal worth $645 million. MEDI889, a highly potent monoclonal antibody that neutralizes RSV by binding the RSV fusion (F) protein expressed on virions and infected cells, is being developed for the passive immunization of the infant population. According to the Centers for Disease Control and Prevention, RSV is the most common cause of lower respiratory tract infections in children younger than 1 year of age. Under the terms of the agreement, Sanofi Pasteur will make an upfront payment of €120 million and pay up to €495 million upon achievement of certain development and sales-related milestones. The two companies will share all costs and profits equally. MedImmune will continue to lead all development activity up to the first approval, will retain MEDI8897 manufacturing activities and Sanofi-Pasteur will lead the future commercialization activities.

Sanofi and Lonza (Basel, Switzerland) partner to share the €270 million cost of a new biological manufacturing plant based in Visp (Switzerland). Under the terms of the deal, Lonza will build the new facility at its Visp site, and the two companies will jointly operate it. Lonza will be able to use its half of the capacity for products for other companies, if it is not needed to do work for Sanofi. Also, if any of Sanofi’s capacity is not being utilized at some point, Lonza will be allowed to use it for its own work. Philippe Luscan, Sanofi’s executive VP of Global Industrial Affairs, said Sanofi needs the extra capacity because 60% of its current pipeline is made of biologics, including monoclonal antibodies. “In addition to the investments we are making in building our own internal production capabilities, the joint venture between Sanofi and Lonza emphasizes our commitment to provide access for patients to high quality therapeutic monoclonal antibodies,” Luscan said.

Royalty Pharma (New York, USA) acquires royalty stream rights for Tysabri, a humanized monoclonal antibody, as a treatment of multiple sclerosis from Perrigo (Dublin, Ireland) for $2.2 billion upfront and up to $650 million in milestones. Biogen markets Tysabri and recorded $1.96 billion sales in 2016.

….The same day Perrigo announced a plan to reduce its workforce by 750 jobs and sees it longtime CFO Judy Brown to leave the company and join Amgen.

Exelexis (South San Francisco, CA, USA) announces two partnerships to study combination with its Cabometyx or cabozantinib. One with Bristol-Myers Squibb where it will combine cabozantinib with Opdivo, or nivolumab, in a phase 3 pivotal study as a frontline for kidney cancer. One with Roche on a combination of cabozantinib and Tecentriq, or atezolizumab, as a treatment of solid tumors.  The data from these stud­ies can be made avail­able to Ex­elixis’ part­ners, Ipsen and Takeda for their own de­vel­op­ment ini­tia­tives.

Synthon Biopharmaceuticals (Nijmegen, The Netherlands) and Sanquin Blood Supply Foundation (Amsterdam, The Netherlands) a non-profit organisation for blood collection, production and supply of blood and plasma products enter a worldwide exclusive license and collaboration agreement to develop anticancer antibodies targeting the CD47-SIRP α pathway.

Approval of drugs and vaccines

US FDA approves ALK’s (Copenhagen, Denmark) Odactra, a sublingual allergy immunotherapy (SLIT) as the first allergen extract to be administered to treat house dust mite (HDM)-induced nasal inflammation (allergic rhinitis). House dust mites are the most common cause of allergy in the world, affecting 90 million people in Europe, North America and Japan, and more than 100 million in China. Nearly 50 percent of all house dust mite-allergic rhinitis patients suffer from concomitant asthma. The HDM SLIT-tablet is already marketed in Europe, in Australia and in Japan.

US FDA approves AstraZeneca’s Qtern, a fixed-dose combination of saxagliptin, a DPP-4 inhibitor and dapagliflozin, a SGLT-2 inhibitor as a dual approach to treat type 2 diabetes who have inadequate control with dapagliflozin or who are already treated with dapagliflozin and saxagliptin.

In its first address to a joint session of US Congress, President Donald Trump focused some of his comments on healthcare issues, namely an attack on Obamacare and on the FDA drug approval process. “We must elim­i­nate the bur­den­some ap­proval process for life-sav­ing drugs so that more lives can be saved. Our slow and bur­den­some ap­proval process at the FDA keeps too many ad­vances from reach­ing those in need,” Trump told the au­di­ence.

Drugs at clinical stage

Hutchinson China MediTech’s (Hong Kong, China) fruquintinib, a VEGFR inhibitor acting as a anticancer drug to treat colon cancer scored positive overall survival and progression free survival in a pivotal phase 3 study Eli Lilly has com­mer­cial­iza­tion rights in China, and an op­tion on global rights.

La Jolla Pharmaceuticals (San Diego, CA, USA) announced that its LJPC-501 met the primary endpoint in a phase 3 clinical trial to treat catecholamine-resistant hypotension. Response was defined as a mean arterial pressure of at least 75 mmHg or a 10 mmHg improvement from baseline pressure at three hours following the start of treatment without increasing vasopressors. Data for secondary endpoints measuring change in cardiovascular and total Sequential Organ Failure Assessment (SOFA) scores were not disclosed.

Roche announced that adding Per­jeta to Her­ceptin and chemother­apy out­per­formed the two older stan­dards alone in re­duc­ing the risk of death or re­lapse for early-stage death or re­lapse for early-stage breast can­cer pa­tients after surgery.

Cempra (Chapell Hill, NC, USA) announces positive results with CEM-102, the sodium salt of fusidic acid, for the treatment of acute bacterial skin and skin structure infections (ABSSI) in showing no inferiority to oral linezolid as measured by the proportion of patients achieving clinical response. CEM-102, branded as TAKSTA, has Qualified Infectious Disease Product (QIDP) status in the US, which provides for accelerated review of the marketing application and an additional five-year period of market exclusivity. Fusidic acid is a bacteriostatic antibiotic that has been in use since 1962.

Roche, Genentech and partner AC Immune (Lausanne, Switzerland) are launching a second phase 3 clinical trial with crenezumab, an anti-amyloid beta drug, as a treatment for prodromal or mild Alzheimer’s patients. The companies believe crezumab to be superior to Eli Lilly’s solanezumab, which recently failed in three consecutive pivotal studies, because the drug targets soluble amyloid beta oligomers, believed to be the most neurotoxic component, as well as beta amyloid monomers.

Kite Pharma (Santa Monica, CA, USA) lead CAR-T drug, axi­cab­ta­gene ciloleu­cel (KTE-C19), showed positive and significant outcomes at six months as a treatment of aggressive non-Hodgkin lymphoma (NHL). “These re­sults with axi­cab­ta­gene ciloleu­cel are ex­cep­tional and sug­gest that more than a third of pa­tients with re­frac­tory ag­gres­sive NHL could po­ten­tially be cured after a sin­gle in­fu­sion of axi­cab­ta­gene ciloleu­cel,” said Jeff Wiezorek, MD, Se­nior Vice Pres­i­dent of Clin­i­cal De­vel­op­ment at Kite.

….At the same time, Juno Therapeutics (Seattle, WA, USA) announces the termination of its clinical program with JCA015, a CAR-T therapy, after the death of several patients, due to unexpected toxicity with cerebral edema or brain swelling. The FDA had issued a clinical hold in October and had lifted it when it thought that the elim­i­nat­ion of fludaribine from the pre­con­di­tion­ing reg­i­men for pa­tients would re­solve the safety issue. Flu­dara­bine is still com­monly used in most CAR-T stud­ies, needed to help these cell ther­a­pies take ef­fect in pa­tients.

Medical Devices and Diagnosis News.

US FDA approves Bracco Diagnostics’ (Milano, Italy) Varibar Pudding or barium sulfate oral paste, for modified barium swallow (MBS) examinations to oral and pharyngeal function and morphology. Oropharyngeal impairment can arise from congenital abnormalities or neurologic causes for impaired swallowing such as cerebrovascular accidents, tumors, motor neuron disease, demyelinating diseases, connective tissue diseases, metabolic disorders, and infections.   Varibar pudding acts as a positive contrast agent for radiographic studies as it is formulated with the minimal coatings required to clearly visualize the swallowing process.

US FDA approves Velano Vascular’s (San Francisco, CA, USA) second iteration of its needle-free blood draw device, which attaches to the peripheral IV line that most hospital patients already have, cutting down on repeated needle sticks that are the norm for blood collection. A peripheral IV, used to deliver fluids, medication or nutrients, may be used to draw blood, but only upon insertion. The single-use, disposable PIVO device attaches to the IV line and advances a small, flexible catheter through it. From here, the device aspirates blood samples without needing to stick the patient multiple times, said Velano Vascular CEO Eric Stone.

US FDA grants 510 (k) clearance to Philips’ ultrasound ElastQ Imaging, a non-invasive technique, as an alternative to biopsies for physicians assessing damage from liver diseases including hepatitis C and NASH. ElastQ uses shear wave elastography to simultaneously image and assess the stiffness of the liver. The approach works by creating and measuring shear waves in soft tissue. Variations in the speed of the wave correspond to the stiffness of the tissue. Liver stiffness is a surrogate marker of liver conditions such as fibrosis and cirrhosis

At its recent annual meeting, Novartis Chairman Joerg Reinhardt described the Google smart lens program as a high-risk project. Expectations for the smart contact lens collaboration, which includes devices that autofocus and track blood glucose level, ramped up when Novartis CEO Joe Jimenez in 2015 said a candidate would be in the clinic in 2016. “I personally think it’s a highly risky project. Nevertheless, it gives us at Novartis and Alcon the opportunity to work together with a technological leader like Google and to learn from that in ways that we can then apply in other areas. It’s an important pilot project,” Reinhardt said.

Science & Technology

Sanofi, with the support of Science 37’s (Los Angeles, CA, USA) launches digital clinical trials to improve recruitment and reduce trial times. Science 37’s approach allows patients to be monitored and report to researchers via an Apple iPhone equipped with the company’s NORA® technology. Qualified study participants are provided with the phone, a data plan and any other sensors or connected devices needed for the trial, along with the medicines being researched. Participants can reach study staff at any time via the mobile device, while also remaining under the care of their local health care professionals. “With digital clinical trials we can get and analyze the data on how a new medicine works in the real world a lot sooner, which means patients get the medicines they need sooner.” said Lionel Bascles, Global Head of Clinical Sciences and Operations of Sanofi.

A new remote digital-based application, named Aparito, with the potential to significantly cut the amount of time children need to stay in hospital is being trialled at Great Ormond Street, Royal Manchester Children's Hospital and Newcastle General hospital. Aparito's system combines wearable technology and a smart phone app that facilitates the capture of vital patient information at home and in real time, allowing doctors to monitor the development of diseases remotely without the need for invasive, hospital-based tests. Aparito is able to record "much more valuable data than is possible to gather from patients being monitored while in hospital," and it can also provide "a realistic insight into the severity and impact of the disease on day-to-day life, unseen before". "Tracking the development of rare diseases can be very time consuming and invasive for our patients who are all children," noted Dr Paul Gissen from Great Ormond Street.

Regeneron Pharmaceuticals and Intouch Solutions (Overland Park, KS, USA) have created a virtual and augmented reality application called “In My Eyes” that allows viewers to experience the blurriness, wavy lines or black patches caused by different types of retinal disease. The three diseases featured are neovascular age-related macular degeneration, or wet AMD; diabetic macular edema, or DME; and macular edema following retinal vein occlusion, or MEfRVO. The application, available in the iTunes and Google app stores, can be used with or without Virtual Reality goggles and works in two modes.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence approves the routine use of Intercept’s (New York, NY, USA) Ocaliva, or obeticholic acid, as a treatment for people with chronic liver disease primary biliary cholangitis (PBC), a rare autoimmune disease and a leading cause of liver transplantation in adult women. The drug had been approved by European regulators in December 2016. "This very rapid decision by NICE, one of the fastest approvals to date for an orphan medication, is an important affirmation of the scientific innovation, clinical value and cost-effectiveness of Ocaliva by one of the most respected health technology assessment bodies," said Lisa Bright, Intercept's president, International.

….and published draft guidelines, rejecting recently Europe approved Merck’s Keytruda, as a first-line treatment for non-small cell lung cancer, in indicating that the magnitude of the overall survival gain was uncertain because the data is immature and the high cost of the drug. One 100 mg vial of Keytruda costs £2,630, and even with an undisclosed, agreed-to discount by Merck, that is too pricey for the Agency.

Miscellaneous

Regulators in the EU and the US have signed a transatlantic agreement to recognise inspections of manufacturing sites for human medicines carried out in their respective countries. Under the new deal, regulators on both sides of the Atlantic will now rely on each other's inspections in their own territories to ensure that sites operate in compliance with Good Manufacturing Practice (GMP), to optimise use of capacity and reduce duplication. The agreement follows robust evidence that EU and US systems have comparable regulatory and procedural frameworks for inspections of manufacturers of human medicines. The move will enable both EU authorities and the US FDA to hone their focus on other parts of the world where active pharmaceutical ingredients (API) and medicines for the EU or US markets are manufactured, to ensure their quality, safety and efficacy. At the end of last year the Japanese Pharmaceutical and Medical Devices Agency (PDMA) had signed a similar agreement with European regulators in order “to allow more sites to be monitored and reduce unnecessary duplication”.

Several Novartis products have been suspended from marketing authorization by the South Korea Ministry of Food and Drug Safety following alleged bribery. South Korea’s Health Ministry is likely to hand out more fines and a sales ban of up to half a year, according to The Investor’s.  The Ministry of Food and Drug Safety last week levied a $177,000 fine and a three-month ban on twelve drugs, the report states.

Within the top 20 pharmaceutical companies in the world investing the largest sums in R&D in the fiscal year 2015-16, 10 are US based, 9 are European based, one is located in Japan. Novartis and Roche are in the front seats

People on the move:

Jakob Riis, Novo Nordisk’s Head of North America, has resigned from the company and will be replaced by Doug Langa the current SVP for Market Access. Riis had expected to succeed Lars Rebien Sørensen as Novo Nordisk’s CEO.

Onxeo (Paris, France) appoints two seasoned executives to accelerate its drug portfolio. Françoise Bono, previously with Evotec and Sanofi is named Chief Scientific Officer to lead Preclinical activities. Olivier de Beaumont, previously with Stallergenes Greer is appointed Chief Medical Officer to Lead Clinical Development and Operations as well as Medical and Regulatory Affairs.

Bioevents

European Life Sciences CEO Forum & Exhibition on March 6-7, in Zurich (Switzerland)

BIO Asia on March 14-15, in Tokyo (Japan)

BIO-Europe Spring on March 20-22, in Barcelona (Spain)

BIOVISION on April 4-6, in Lyon (France)

Strategic Alliance Management Congress on May 1-3, in Philadelphia (USA)

BIOTrinity on May 8-10, in London (UK)

9^th Annual China BIO Partnering Forum on May 10-11 in Shunde (China)

The Health Industry Sumitt (tHIS) 2017 on May 15-18 in Shanghai (China)

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

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

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/

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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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