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

US President Donald Trump has chosen Scott Gottlieb to lead the US FDA. Gottlieb, a medical doctor, is currently a fellow at the American Entreprise Institute and has previously served as deputy commissioner for medical and scientific affairs at the FDA during President George W. Bush’s administration. He has also been acting as an advisor to Bristol-Myers Squibb, Cell Biotherapy and GlaxoSmithKline.

Business

Allergan (Dublin, Ireland) has signed a deal with Editas Medicine (Cambridge, MA, USA) to secure exclusive rights to license up to five CRISP genome editing based ophthalmology treatments, including LCA10, a preclinical drug candidate for Leber congenital amaurosis. Allergan will make a $90 million upfront payment, as well as development milestones and royalties on sales. In 2016, Editas had entered a similar collaboration with Advertum Biotechnologies.

Sanofi enters a non-exclusive agreement with Voluntis (Suresnes, France), a software company, to develop a mobile phone application to support type 2 diabetic patients. The agreement builds on an existing alliance established in 2011.

Daiichi Sankyo partners with Heptares (Welwyn Garden City, UK) to discover and develop targeted G protein-coupled receptor (GPCR) small molecule drug candidates for the treatment of pain. Under the terms of the deal, Daiichi Sankyo will make a $4 million upfront payment, will fund R&D fees of about $8 million, make development milestones payments plus royalties on future sales. Heptares has ongoing R&D agreements in place with AstraZeneca, Kymab and Teva.

CRISPR Therapeutics (Basel, Switzerland) and Casebia Therapeutics (Cambridge, MA, USA), its joint venture with Bayer, licenses cell engineering platform from MaxCyte (Gaithersburgh, ML, USA). Under the terms of the agreement, both companies will get non-exclusive rights to commercial use of MaxCyte's Flow Electroporation technology for use in developing CRISPR/Cas9-based therapies for hemoglobin deficiencies and severe combined immunodeficiency. Financial terms of the deal were not disclosed bu are linked with upfront, milestone and sales payments.

Roche, Mylan (Canonsburg, PA, USA) and partner Biocon (Bengalore, India), are close to settle their patent dispute over the Herceptin biosimilar licensing deal.  Under the new terms Roche will grant Mylan and Biocon a global license to Herceptin in return for Mylan's commitment to back off of two challenges pending at the US Patent and Trademark Office.  The Herceptin biosimilar could be approved by the US FDA in September 2017.

AstraZeneca enters a collaboration with SenzaGen (Lund, Sweden) to develop a genomic method for differentiating between respiratory allergens and irritants to aid in discovering treatments for respiratory diseases. The companies will use SenzaGen's genomic allergen rapid detection platform called GARD, which measures genetic biomarkers of immune reactions to predict risk of hypersensitivity, and AstraZeneca's inhaled substance libraries to develop a genomic signature that can be used to identify molecules that cause respiratory tract irritancy. Financial terms were not disclosed.

Roche and Bavarian Nordic (Copenhagen, Denmark) enter a collaboration to assess the value of the combination of Tecentriq, Roche’s anti-PD-L1 antibody with CV301, Bavarian Nordic’s cancer vaccine candidate in bladder cancer. The phase 2 to be conducted by Bavarina Nordic is set to start at the end of the year and is designed to evaluate the drug combination versus Tecentriq alone in patients with locally advanced or metastatic urothelial carcinoma. CV301 is designed to generate a T cell response to both CEA and MUC1, two tumour antigens that are highly over expressed in bladder cancers.

PTC Therapeutics (South Plainfield, NJ, USA) acquires Marathon Pharmaceuticals’ (Northbrook, IL, USA) Emflaza, a treatment of Duchenne’s muscular dystrophy for $140 million in cash and stock. The drug had been highly advertised after Marathon had announced a price of $89,000 for a yearly treatment.

Approval of drugs and vaccines

US FDA approves:

Novartis’ Kisquali or ribociclib, which had breakthrough therapy status, as a treatment of HR positive/HER2 negative metastatic breast cancer in a first-line setting. Ribociclib is an inhibitor of two cyclin dependent kinases, CDK4 and CDK6. Under priority review the drug was approved four and half months after NDA filling.

Merck’s Keytruda, or pembrolizumab, as a treatment for both adults and pediatric patients with refractory classical Hodgkin lymphoma. The approval constitutes the first approval of a checkpoint inhibitor in a blood cancer.

….and Exela Pharmaceutical’s RTU, or sodium nitroprusside in a 0.9% sodium chloride injection for immediate use in immediate lowering of high blood pressure. Nipride RTU know since more than thirty years, is also approved for producing controlled hypotension to reduce bleeding during surgery and for the treatment of acute heart failure to reduce left ventricular end-diastolic pressure, pulmonary capillary wedge pressure, peripheral vascular resistance and mean arterial blood pressure. Nipride RTU carries a Boxed Warning describing the possibility of excessive hypotension and cyanide toxicity.

The UK Medicines and Healthcare Products Regulatory Agency has issued a positive scientific opinion for Sanofi’s dupilumab through the Early Access to Medicine Scheme (EAMS), giving patients with atopic dermatitis access to the drug before its marketing authorization. The EAMS was established to secure early availability of innovative new unlicensed medicines to UK patients that have a high degree of unmet clinical need, and has to date largely focused on life-limiting conditions such as cancer.

Drugs at clinical stage

US FDA has issued a second complete response letter rejecting AstraZeneca’s ZS-9 for the treatment of hyperkalemia.  AstraZeneca acquired the drug from ZS pharma for $2.7 billion in 2015.

Amgen reports positive results with Repatha, or evolucumab, a PCSK9 inhibitor, in showing efficacy in reducing low-density lipoprotein cholesterol (LDL-C) in patients receiving apheresis and in the Fourier trial where the drug cut heart attack risks by 27% and the risk of stroke by 21%.

AstraZeneca’s Lynparza, or niraparib, a PARP inhibitor, delayed the recurrence of ovarian cancer by more than two years compared to a placebo, according to results from a phase 3 study. The strong benefit observed with Lynparza as ovarian cancer maintenance therapy exceeded previous expectations. PARP inhibitors are drugs which work by blocking an enzyme, poly (ADP-ribose) polymerase, that cancer cells use to repair their DNA after being damaged by chemotherapy.

TiGenix’s (Louvain, Belgium) Cx601 showed positive results in maintaining treatment benefit for two years in patients with complex perianal fistulas in Crohn's disease. Cx601 is an adipose-derived allogenic stem cell suspension.

Medical Devices and Diagnosis News.

US FDA approves the new version of Medtronic’s Reveal Linq, which now includes algorithms that ramp up the implant’s accuracy in reporting abnormal heartbeats. The Reveal Linq is a tiny cardiac monitor which is implanted in the upper left side of the chest. It transmits data wirelessly to a patient bedside monitor and its battery life allows it to monitor abnormal heart rhythms for up to three years. When compared to the original Reveal Linq, the device with TruRhythm Detection cut down on false reports of bradycardia by 95% and reduced false pause detections by 47%. The new device also includes an algorithm for atrial fibrillation that learns a patient’s heart rhythm over time, tamping down on false detections of fibrillation episodes,

Health Canada has approved Roche’s Cobas EGRF Mutation Test v2 for marketing authorization. The assay includes expanded mutation coverage of the EGFR gene in DNA derived from tumor tissue and liquid biopsy and can be used to select non-small cell lung cancer (NSCLC) patients who have a T790M mutation as well as NSCLC patients with exon 19 deletions or L858 mutations. The US FDA has approved Roche’s test back in 2015 as a companion diagnostic for AstraZeneca's Tagrisso or osimertinib for NSCLC patients and in June 2016 as a test to detect EGFR mutations associated with NSCLC in blood.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence (NICE) issued a draft guidance turning down Sanofi’s Cerdelga for the treatment of Gaucher’s disease indicating that the drug, as an oral therapy, could provide “valuable quality-of-life benefits,” but it had to consider the “substantial additional costs”. According to NICE, Cerdelga would cost £84,559 in the first year of treatment based on list prices and as much as £571,487 by year five.

Miscellaneous

The US FDA fees for product review is expected to more than double under the Trump administration’s proposed 2018 budget. In return, the administration said that the increased budget would help speed up the approval process.

Bioevents

DCAT Week’ 17 on March 20-23, in New York City (USA)

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)

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

9^th Annual 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)

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.