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

The US Senate voted 57 to 42 to confirm Dr. Scott Gottlieb as the next FDA commissioner. The biopharmaceutical industry has backed him from the beginning and is happy to endorse someone well known and his views on the agency.

Business

Sanofi enters a $237 million collaboration with Exscientia (Dundee, UK) on the discovery of bispecific small-molecule drugs targeting metabolic diseases. Exscientia will use its artificial intelligence (AI) platform to identify combinations of synergistic drug targets and discover bispecific small molecules against those targets. Exscientia will be responsible for the design of the drugs and Sanofi will provide the chemical synthesis efforts.

Pfizer enters a $545 million agreement with Sangamo Therapeutics (Richmond, CA, US) and extends its gene therapy portfolio to treat hemophilia A, with SB-525, which is due to enter clinical trial in the coming weeks. Under the terms of the deal, Pfizer will make a $70 million upfront payment, up to $475 million in milestones and tiered double-digit royalties on net sales. Sangamo will be responsible to conducting a Phase 1/2 study of SB-525 and certain manufacturing activities. Pfizer will be responsible for subsequent development, research, manufacturing and commercialization of SB-525 and additional products. The drug will be competing with BioMarin’s BMN 270, which is currently in phase 2B for the same indication.

Merck & Co enters a clinical trial collaboration with Array BioPharma (Boulder, CO, USA) to evaluate the combination of Keytruda, or pembrolizumab, with Array’s binimetinib, a MEK inhibitor, as a treatment for metastatic colorectal cancer.

Merck KGaA, Pfizer and EpiThany Biosciences (San Francisco, CA, USA) enter a collaboration to assess the combination of Merck and Pfizer’s Bavencia, or avelumab, with EpiThany’s EP-101 STEMVAC, a T-helper type 1 (Th1)-selective, multi-antigen DNA vaccine designed to trigger a targeted antitumor immune response, in breast cancer

Commense (Boston, MA, USA) co-founded by PureTech Health, licenses a microbiome-based therapeutic from the University of British Columbia (Vancouver, Canada) as a therapeutic for the prevention of asthma and other allergic diseases. The approach involves the use of four types of bacteria, Faecalibacterium, Lachnospira, Veillonella, and Rothia (known as FLVR), and is founded on the discovery that children with atopy, wheeze, and asthma have experienced a transient imbalance in FLVR during early life.

Sandoz, the generic division of Novartis, has gained US rights to Durect’s (Cupertino, CA, USA) Posimer, or bupivacaine, a non-opioid, postsurgical analgesic using Durect’s sucrose acetate isobutyrate extended release formulation. Under the terms of the deal Sandoz will make a $20 million upfront payment, followed by $43 million development and regulatory milestones as well a $230 million in milestones based on sales and tiered double-digit royalties on sales.

Approval of drugs and vaccines

The European Commission approves:

Merck & Co (MSD) Keytruda, an anti PD-1 therapy, for the treatment of classical Hodgkin’s lymphoma (cHL), a type of lymphoma that develops in white blood cells.

…. and EUSA Pharma’s (Hemel Hemsted, UK) dinutuxima beta, a monoclonal chimeric antibody targeting GD2, a specific antigen on neuroblastoma cells to treat high-risk neuroblastoma in children affected by this rare form of cancer. Every year, in Europe, around 1,200 children are diagnosed with neuroblastoma, a cancer arising from neural crest cells, which are involved in the foetal development of the nervous system and other tissues. Almost half of children are diagnosed at an advanced stage of their disease and are considered ‘high-risk’ with a poor prognosis.

The US FDA approves:

Mitsubishi Tanabe Pharma’s (Tokyo, Japan) Radicava, or edaravone, as the first new treatment option in 22 years in the US for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Patients with the disease can lose the ability to control their muscles, making it hard to walk, eat, breathe and talk. Most die within two to five years after diagnosis. The agency was so impressed by the data that it asked the Japanese company to file in the US based solely on data generated in Japan. The drug does not reverse the disease but it slows down its progression. Patients on the treatment experienced a 33% reduction in the rate of decline in physical function compared with those on placebo. Edavarone has been available in Japan for several years as a treatment for stroke. Competing drugs are AB Science’s masitinib (see under drugs at clinical stage) and Cytokinetic’s tirasemtiv.The last approved drug for ALS was Sanofi’s Rilutek in 1995.

Merck & Co’s combination therapy of Keytruda with chemotherapy as first-line therapy of non-small cell lung cancer (NSCLC) on the basis that the chemo/check­point combination triggered a much higher con­cen­tra­tion of over­all re­sponses for NSCLC than chemotherapy alone (55% vs. 29%). High PD-L1 ex­pressers did even bet­ter, though the ap­proval comes through for all pa­tients re­gard­less of bio­marker sta­tus.

… and Novartis combination of Kisquali or ribociclib, a kinase inhibitor with Femara, or letrozole, an aromatase inhibitor as a first-line endocrine-based treatment for HR-positive, HER2-negative advanced or metastatic breast cancer in postmenopausal women.

Drugs at clinical stage

The US FDA grants breakthrough status to:

Rhythm’ (Bsoton, MA, USA) setmelanotide, as a treatment for obesity caused by a MC4 receptor defect in the leptin-melanocortin pathway. Setmelanotide is a melanocortin-4 receptor (MC4R) agonist, already approved by the FDA as a breakthrough therapy for pro-opiomelanocortin (POMC)-deficiency obesity. The drug is currently being evaluated for the treatment of other genetic disorders of obesity—Prader-Willi syndrome, Bardet-Biedl syndrome, Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders.

…and to Proteon Therapeutics’ (Waltham, MA, USA) vonapanitase for increasing arteriovenous fistula secondary patency and improving use for hemodialysis.

The US FDA grants orphan status to:

Xencor’s (Moravia, CA, USA) XmAB5871 as a treatment for patients with IgG4-related disease, a newly defined fibro-inflammatory autoimmune disorder.

…and to Corline Biomedical’s (Uppsala, Sweden) heparin substance.

The US FDA grants accelerated approval to Merck KGaA and Pfizer's PD-L1-targeted Bavencio, or avelumab, for use in patients with locally advanced or metastatic urothelial carcinoma in cases involving disease progression on or after treatment with chemotherapy that contains platinum.

The US FDA grants fast-track status to XyloCor Therapeutics' (Newton Square, PA, USA) XC001, as a treatment to improve exercise tolerance in patients with chronic angina that are refractory to standard medical therapy and not responsive to conventional revascularization procedures. XC001 is an investigational angiogenic gene therapy that promotes angiogenesis to provide arterial blood flow to myocardial regions with inadequate blood supply.

Roche’s immunotherapy Tecentriq, or atezolizumab, failed to improve overall survival to chemotherapy in pre-treated patients with advanced bladder cancer. The result comes as a stunning setback after the drug was initially approved as a second-line treatment for bladder cancer.

AstraZeneca’s tralokinumab, an interleukin 3 (IL-3) antibody failed to sig­nif­i­cantly re­duce an­nual ex­ac­er­ba­tion rates in asthma patients. AstraZeneca sold off the der­ma­tol­ogy rights for tralok­inumab to Leo Pharma which says it gath­ered pos­i­tive data on the drug re­cently.

One of Kite Pharma’s (Santa Monica, CA, USA) CAR-T patients treated with KTE-C19 died from cerebral edema. the same brain swelling con­di­tion that went on with Juno’s CAR-T drug.

The “Agence National de la Sécurité des Médicaments”, the French regulatory authority,  has ordered AB Science (Paris, France) to halt a clinical trial, in France, with AB06006, a drug aimed at treating ALS patients (see under drug approval) following deviation from Good Clinical Practice standards.

Medical Devices and Diagnosis News.

The US FDA approves Boston Scientific’s Resonate line of implantable cardioverter defibrillators and cardiac resynchronization therapy defibrillator (CRT-D) systems. The products all use the EnduraLife battery, a technology Boston Scientific claims has twice the capacity as that used by Medtronic in its Evera XT VR DVBB2D4 device.

Science & Technology

A study conducted by scientists at the University of Melbourne, the University of Chicago and others from the Netherlands and France published in the Proceedings of the National Academy of Sciences and involving 641 children aged 1 to 12 years living in a small village in the African country of Gabon found that each child carried a different strain of the Plasmodium falciparum parasite, each with a different combination of about 60 “var genes” responsible for encoding surface antigens. “This structure allows each parasite to look different to the immune system, and provides the possibility for the malaria parasite to keep re-infecting the same people,” similar to but much more complicated than flu, said Karen Day, professor of population science at the University of Melbourne. This unexpected result may threaten the discovery of broad and efficient vaccine.

Cost, Pricing and Market Access

Sanofi joins other companies and commits to keep any US price increases at or below an official health inflation measure that is projected to hit 5.4% in 2017. The company will also report numbers on its aggregate price increases annually, disclosing hikes to gross prices and net prices, which reflect rebates and discounts granted to payers.

Bioevents

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/

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.