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

Mergers, Acquisitions and Joint Ventures

Otsuka  Pharmaceutical (Tokyo, Japan) acquires Neurovance (Cambridge, MA, USA) for up to $250 million and gets access to centanafadine or EB-1020, a nonstimulant, triple reuptake inhibitor that targets norepinephrine, dopamine, and, to a lesser extent, serotonin reuptake, a Phase 3 attention-deficit/hyperactivity (AHDH) drug candidate . Under the term of the deal Otsuka will make a $100 million upfront payment and $150 million in various development, approval and sales milestones.

Business

Vertex Pharmaceuticals (Boston, MA, USA) secures rights to Concert Pharmaceutical’s (Lexington, MA, USA) cystic fibrosis (CF) programmes including CTP-656, a deuterated version of  Vertex's Kalydeco ivacaftor, which received recent US FDA Orphan Drug status. Under the terms of the deal Vertex will make a $160 million upfront payment and up to $90 million in milestones.

Pharnext (Paris, France) partners with Galapagos (Mechelen, Belgium) to use Pharnext’s Pleotherapy platform and combine Galapagos’ preclinical drug candidates with existing drugs to develop new treatments. The Pleotherapy platform uses a genomic-based network pharmacology approach to understand the molecular pathways involved in disease and identify synergistic combinations of drugs that have previously been approved for other indications, against those pathways. Pharnext’s lead candidate, PXT3003, currently in Phase 3 clinical development for the treatment of Charcot-Marie-Tooth disease type 1A, is a combination of low doses of baclofen, naltrexone, and sorbitol and has orphan drug designation in Europe and the US.  Pharnext’s second clinical candidate PXT864, as a treatment for Alzheimer disease, Parkinson disease and for amyotrophic lateral sclerosis, is a low doses combination of baclofen and acamprosate.  Financial terms of the deal were not disclosed.

Santhera Pharmaceuticals (Liestal, Switzerland) establishes a US operation in Boston to support the launch of Raxone, or idebenone,  an anti-oxidant benzoquinone, which enhances cellular respiration by interacting with the mitochondrial electron transport chain, to treat patients with Leber’s hereditary optic neuropathy (LHON) an inherited disorder characterized by the degeneration of the retina.  The drug was approved in Europe in September 2015 for this indication. Santhera is also developing Raxone to treat Duchenne muscular dystrophy, through an US FDA Orphan Drug designation as well as for the treatment of primary progressive multiple sclerosis.

Approval of drugs and vaccines

US FDA approves Allergan and Serenity Pharmaceutical’s (Milford, PA, USA) Noctiva, or desmopressin acetate nasal spray, a synthetic analog of vasopressin, as a treatment of patients with nocturnal polyuria, as the first treatment for this indication. The drug, which is to be taken around 30 minutes before going to bed, works by increasing the absorption of water through the kidneys, which leads to less urine production.

During China’s annual national congress meetings, China CFDA chief Bi Jingquan said that the agency will tackle some of the major drug approval barriers before foreign biopharmaceutical companies. That includes “eliminating unreasonable regulations”, hiring more staff and revamping procedures at the Center for Drug Evaluation to speed up the drug approval process, and better implementation of intellectual property laws. Still, as pointed out by a congress member, it could take a foreign drug about 6 to 10 months before an CTA approval, a marketing authorization could take as long as 62 months.

Drugs at clinical stage

The European Medicines Agency grants aTyr Pharmoceutical’s (San Diego, CA, USA) Resolaris orphan drug status as a treatment for limb girdle muscular dystrophy. The drug received US FDA fast track status in January.

US FDA grants qualified infectious disease product designation (QDIP), to PolyPid’s (Patah Tikva, Israel) D-PLEX, or doxycycline/polymer-lipid encapsulation matrix, a secured antibiotic drug reservoir for the local prevention and treatment of surgical infections

US FDA grants orphan status to Boehringer Ingelheim’s BI836858, an anti CD33 monoclonal antibody as a treatment for myelodysplastic syndromes (MDS). MDS are a group of bone marrow disorders, in which the bone marrow carries cell abnormalities and is unable to produce blood cells efficient. There are many subtypes of the disease – including the blood cancer acute myeloid leukemia (AML) – all with variable prognoses and treatment options.

US FDA approves Pfizer’s the Investigational New Drug (IND) application for UCART19, a CAR T cell candidate for acute lymphoblastic leukemia (ALL) and B-cell malignancies co-developed with Cellectis (Paris, France) and Servier (Suresnes, France).

Roche reactivates the development of gantenerumab, a HuCAL-de­rived an­ti­body, with a higher dosis, as a treatment for Alzheimer’s disease. Morphosys, Roche’s partner for this project, indicated that there are plans in place to launch two Phase 3 stud­ies in mild to pro­dro­mal pa­tients a group of pa­tients who are just be­gin­ning to demon­strate symp­toms of the mem­ory loss. Last week we had described that Roche/Genentech has also resumed a pivotal phase 3 with crenezumab in Alzheimer’s disease.

US FDA lifts the hold on Seattle Genetic’s vadastuximab talirine, which had induced four death in study with acute myeloid leukemia (AML), after the company says im­ple­ment­ed added risk mit­i­ga­tion mea­sures in all vadas­tux­imab talirine stud­ies, in­clud­ing “re­vised el­i­gi­bil­ity cri­te­ria and stop­ping rules for veno-oc­clu­sive dis­ease.”

TG Therapeutics (New York City, USA) indicated that TG-1101 or ubli­tux­imab, an anti-CD20 mon­o­clonal an­ti­body, plus Im­bru­vica was superior to Im­bru­vica alone in treat­ing high-risk chronic lym­pho­cytic leukemia, or CLL. The com­bi­na­tion arm scored an over­all re­sponse rate of 80% com­pared to only 47% in the single drug arm. 

Medical Devices and Diagnosis News.

US FDA approves Tryton Medical’s (Durham, NC, USA) stent for coronary bifurcation lesions. Currently, only the main artery is stented in coronary artery disease involving bifurcation lesions. Tryton Medical's stent is deployed in the side branch alongside a drug-eluting stent in the main branch.

US FDA approves Zoll Medical’s, a Asahi Kasei Group Company, wearable defibrillator, which automatically detects and treats ventricular arrhythmias in high-risk hospital patients. The Hospital Wearable Defibrillator (HWD) is designed to continuously protect patients at risk of ventricular fibrillation or ventricular tachycardia during their hospital stay. The HWD automatically detects ventricular fibrillation or tachycardia and immediately delivers a treatment shock.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence endorses the extended use of Merck’s Erbitux use with FOLFOX or FOLFORU as a first-line treatment for patients with RAS wild-type metastatic colorectal cancer.

A study published in Health Affairs found that Big Pharma makes more from high US drug prices than it spends on R&D worldwide and that there was a 59% spread between the average prices for the world’s 20 top-selling drugs in other developed countries and those in the US. In other words, a drug that costs $41 in other countries costs $100 in the US. The US premium created $116 billion in revenue for 15 of the world’s top biopharmaceutical companies, compared with $76 billion in global R&D spending from that group and is not compensated by drug rebates.

Médecins Sans Frontières, a French non-profit organisation, hosted a “hackaton” over the week-end in New York City to protest against Pfizer and GlaxoSmithKline’s high pneumococcal vaccine pricing.

Miscellaneous

A French report by the Curie Institute (Paris, France) showed that trained “sniffing dogs” in the early Kdog protocol were a “100% efficacious” in detecting breast cancers in patients. The two trained dogs could “identify smelling components” found on sweat impregnated tissues. The study will be extended to 1,000 samples of breast cancers and will be performed by four different dogs.

People on the move:

Bristol-Myers Squib appoints Thomas Lynch, a Massachusetts General Hospital veteran, as is new Chief Scientific Officer.

Polyphor (Allschwil, Switzerland) appoints Debra Barker, from Novartis, as its new Chief Medical and Development Officer.

Bioevents

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)

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.