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

Editor’s Note

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“Printemps Solidaire”, a French charity organization, in Paris Friday evening, with Bill and Melinda Gates as well as Michaëlle Jean, former Governor General of Canada, and current Secretary-General of the Organisation Internationale de la Francophonie

Business

Novartis and Allergan will collaborate on a phase 2 clinical trial assessing the potential value of the combination of Allergan’s cenicriviroc, a dual inhibitor of CCR2 and CCR5 two inflammatory cytokines, with Novartis’s Farnesoid X receptor (FXR) agonist in patients with non-alcoholic steatohepatitis (NAS). “Our clin­i­cal col­lab­o­ra­tion with Al­ler­gan ex­pands our de­vel­op­ment pro­grams for NASH, bring­ing to­gether sci­ence and ex­per­tise to in­ves­ti­gate a po­ten­tial new com­bi­na­tion ther­apy in an ef­fort to make a pos­i­tive change for peo­ple liv­ing with this con­di­tion,” said Vas Narasimhan, No­var­tis’ CMO and head of drug de­vel­op­ment.  “We be­lieve that col­lab­o­ra­tion is key to de­vel­op­ing the best pos­si­ble treat­ments that are ur­gently needed for NASH pa­tients.” Financial details of the deal were not disclosed.

H3 Biomedicine, a subsidiary of Eisai expands its existing partnership with Foundation Medicine focusing on the discovery and development of precision medicine oncology drugs in using FondationCORE, which contains more than 100,000 real-world genomic profiles, to identify new cancer targets. Foundation Medicine has partnered with more than 30 pharmaceutical companies.

Approval of drugs and vaccines

The US FDA approves Roche/Genentech’s Lucentis 0.3 mg, or ranibizumab, as a monthly treatment of diabetic retinopathy. Lucentis becomes the first and only US FDA approved medicine to treat diabetic retinopathy in people who have been diagnosed either with or without diabetic macular oedema, a complication of diabetic retinopathy that causes swelling in the back of the eye.

The US FDA expands the approval of Roche’s Tecentriq to treat patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin chemotherapy.  The drug was previously cleared for people with locally advanced or mUC who have disease progression during or following any platinum-containing chemotherapy, or within 12 months of receiving chemotherapy before surgery or after surgery.

After a serious slowdown in 2016, the US FDA approval has started off to an excellent result with already 12 new drugs approved in Q1 2017. The agency has another 20 applications pending for the year 2017.

Drugs at clinical stage

The US FDA grants breakthrough status to Novartis’ CTL019, a chimeric antigen receptor T cell therapy candidate, as a treatment for relapsed/refractory diffuse large B-cell lymphoma. (r/r DLBCL), who have failed two or more prior therapies. DLBCL is the most common form of lymphoma and accounts for  around 30% of all non-Hodgkin lymphoma cases The FDA previously granted accelerated review to the drug as a treatment for acute lymphoblastic leukemia in young patients.

The US FDA grants breakthrough status as well as Regenerative Medicine Advanced Therapy (RMTA) status to Enzyvant Therapeutics’ (Basel, Switzerland) RVT-801 as a treatment for patients with a severe lack of thymus tissue and a weakened immune system because of complete DiGeorge syndrome.

The US FDA grants orphan status to Aldeyra Therapeutic’s (Lexington, MA, USA) ADX-102, as a treatment for patients with congenital ichthyosis associated with Sjögren-Larsson syndrome (SLS). SLS is a rare inborn error of aldehyde metabolism caused by mutations in fatty acid aldehyde dehydrogenase, leading to elevated toxic fatty aldehyde levels that are thought to contribute to severe ichthyosis (scaly, thickened, dry skin), neurological disorders, and retinal disease. There is currently no US FDA approved therapy for SLS.

Eli Lilly and partner Incyte disagree with the FDA’s conclusions rejecting Olumiant, or baricitinib, their rheumatoid arthritis treatment, indicating they might be able to speed up the resubmission of the drug with more discussion.  The rejection will benefit Pfizer’s Xeljanz:  “Xeljanz will remain the only oral JAK inhibitor on the US market for several more quarters,” Credit Suisse analyst Vamil Divan said. A new FDA filing should not occur sooner than 12 months from now and a launch in “2019 or later.”

AbbVie’s veliparib, a PARP inhibitor, has failed to meet key targets in two late-stage studies testing its potential in lung and breast cancer. The studies were assessing veliparib in combination with carboplatin and paclitaxel in patients with squamous non-small cell lung cancer (NSCLC) and triple negative breast cancer (TNBC). Currently approved PARP inhibitors include AstraZeneca’s Lynparza or olaparib, Tesaro's Zejula, or niraparib and Clovis’ Rubraca, or rucaparib, all as treatments for ovarian cancer.

Medical Devices and Diagnosis News.

Bristol-Myers Squibb and Nordic Bioscience (Herlev, Denmrk) enter a collaboration to develop translational biomarker technology to diagnose and monitor fibrotic diseases such as nonalcoholic steatohepatitis (NASH). .

Johnson & Johnson has acquired 3D-printing technology from Tissue Regeneration Systems (TRS), a company which emerged in 2008 from the University of Michigan and the University of Wisconsin. The deal gives J&J’s DePuy Synthes Products unit technology for creating personalized bioabsorbable implants designed to aid the healing of bones. J&J plans to use the technology to print implants that treat orthopedic and craniomaxillofacial deformities and injuries. The resulting technology consists of two elements. One part is the 3D-printing technology itself. TRS bases the geometry of each personalized device on CT scans, resulting in implants that should be a close match for the missing bits of bone. TRS’ implants use scaffold technology designed to create devices that are porous enough to be integrated into the bone. As healing happens, the device is fully replaced by bone. The second elements consists in a mineral coating, with a “plate-like nanostructure that resembles living bone,” TRS thinks it can support bone regeneration and proliferation. The idea is to provide a surface that autologous cells and growth factors stick to and grow on.

Science & Technology

Scientists at the University of California in Berkeley are explaining why accidental untying of a shoelace while walking occurs without warning. In a paper published in the Proceedings of the Royal Society A, three engineers deciphered why a shoelace becomes untied. First, the repeated impact of the shoe on the floor during walking serves to loosen the knot. Then, the whipping motions of the free ends of the laces caused by the leg swing produce slipping of the laces. This leads to eventual runaway untangling of the knot. The demonstrated the situation using slow-motion video footage and a series of experiments, showing the failure of the knot happening in a matter of seconds. The controlled experiments showed that increasing inertial effects of the swinging laces leads to increased rate of knot untying, that the directions of the impact and swing influence the rate of failure, and that the knot structure has a profound influence on a knot's tendency to untie under cyclic impact loading.

Cost, Pricing and Market Access

The UK National Institute for Health & Care Excellence (NICE) has issued a positive Final Appraisal Determination (FAD) supporting Novartis’ Cosentyx, or secukinumab, for the treatment of progressive psoriatic arthritis. Cosentyx, the first in a class of medicines called interleukin-17 (IL-17) inhibitors to be recommended by the NICE, is a targeted biologic therapy that selectively binds to IL-17A and stops it from binding to its receptors and causing inflammation. “Secukinumab is the first IL-17A inhibitor which has been shown to significantly improve joint and skin symptoms of psoriatic arthritis, and provides patients with improved physical functioning and quality of life. There is a growing body of evidence that supports the potential of secukinumab to become the standard of care for patients living with this debilitating condition” said Professor Paul Emery, director of Leeds Institute of Rheumatic and Musculoskeletal Medicine and Leeds Musculoskeletal Biomedical Research Unit.

Ipsen (Boulogne-Billancourt, France) is in talks with the UK NICE following a second rejection of Cabometyx, or cabozantinib, as a treatment for advanced renal cell carcinoma (RCC). The drug was approved in Europe in September 2016 and is available in the UK through Ipsen’s Managed Access Programme. Earlier this year NICE had concluded that the additional benefits of Cabometyx, when compared with current care, "were highly uncertain as the meta-analysis evidence presented by the company was unreliable,"  Now, a second Appraisal Consultation Document (ACD) published following a further analysis of cost-effectiveness information has also rejected routine prescribing of the drug after vascular endothelial growth factor (VEGF) targeted therapy on the NHS in the RCC setting. The committee noted that, in all the analyses, the incremental analysis showed that Cabometyx was associated with an incremental cost effectiveness ratio of more than £60,000 per QALY gained compared with Novartis’ Afinitor (everolimus), and thus well outside the bounds of what is normally considered value for money for the NHS.

According to Novartis’ CEO Joe Jimenez, the Chinese pharmaceutical market may exceed $300 billion in sales by 2020. “They recognize they have a population that really wants to be part of getting access to health care and new medicines,” he said. “They’re starting to bring down those barriers.” While foreign companies have historically had to wait several years to bring treatments already approved in other countries into China, the country is speeding up its review of new medicines to combat increasing rates of diseases like cancer and hepatitis.

Miscellaneous

Pfizer and ICU Medical, which recently acquired the Hospira Infusion Systems business from Pfizer, acknowledged that they have been caught up in the US Department of Justice (DOJ) investigation into possible price collusion in sales of saline solution and infusion equipment and have been called to testify before a federal grand jury. The disclosures come a few days after Baxter International's acknowledgement that it had been called to testify before the grand jury in the Eastern District of Pennsylvania in connection with an investigation by the DOJ’s Antitrust Division into saline sales, shortages and pricing. Baxter and Hospira, reportedly control about 90% of the US saline market.

Médecins Sans Frontières (MSF) an international charity organisation, deploys a new online tool to fight lower prices for Pfizer and GlaxoSmithKline’s pneumococcal vaccines. Now MSF will roll out an online platform dubbed “Your stock, your voice” aimed at letting people track whether they hold shares in either company. Then, people can use their voice to “demand some changes from Pfizer and GSK to ask them to lower the price of the pneumonia vaccine,” Vivian Peng, MSF vaccines campaign communications officer said. “A lot of people in the US put money away in their retirement funds and their savings and don’t really know where their money goes,” Peng said. “If you can see where your money is going, you have this hidden power to make your voice heard.”  The launch comes a month after MSF hosted a “hackathon” in New York City to bring together designers and developers to build the platform. It is the latest in a years-long effort by the group to get Pfizer and GSK to lower their pneumococcal vaccine prices to $5 per child for all three doses.

Boehringer Ingelheim and Lilly announced the launch, in the US, of Synjardy XR as adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes when treatment with both empagliflozin and metformin HCl is appropriate. Synjardy XR combines empagliflozin, a sodium-glucose co-transporter 2 (SGLT2) inhibitor, and metformin, a biguanide.  Synjardy XR received US FDA approval in December 2016 and carries a boxed warning about metformin-associated lactic acidosis.

People on the move:

Juno Therapeutics (Seattle, WA, USA) has recruited Sunil Argarwal from Genentech to head up the new R&D organization to be established in the San Francisco area.

Bioevents

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

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

China BIO Partnering Forum on May 10-11 in Suzhou (China)

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

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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