Btobioinnovation.com

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

Editor’s Note

After a little more than four years in existence, the Editorial Board of BtoBioinnovation has decided to terminate the edition of the Biopharmaceutical Newsweek letter. We would like to thank the hundreds of subscribers and readers who have been supportive of this exciting venture. 

At the inception of this project, we had decided to edit a short, updated, focused, and free of charge, weekly newsletter with no advertising, no flamboyant or misleading titles, “no traffic trap”, but with the right level of validated content useful for educated people in the Biopharmaceutical business development sector.  Our goal was not to reach a very large number of readers, but rather to be considered as a value-based differentiated information string, without pretending or claiming “smart brevity”. In short, a “smart friend” in your pocket to grasp what is most relevant to your own business, in the midst of an overwhelming daily information flow.  Over time we had expected to receive the support of a sponsor and/or to attract a group of “addicted readers” that needed this newsletter and would be willing to “pay for value” in order to combine a free a charge edition with a more comprehensive proposition priced at its fair market value. None of these scenario occurred and now our available resources and the current economic model, do not permit us to continue to deliver an accurate and updated content covering the business activities of the fast changing Biopharmaceutical sector.

We apologize for the inconvenience this may trigger for some of you and wish all of you, around the globe, plenty of success in your own business activities.

Mergers, Acquisitions and Joint Ventures

Parexel International (Waltham, MA, USA), a leading biopharmaceutical services company, is being acquired by Pamplona Capital Management, a private equity group, located in London, New York and Boston, in a deal valued at around $5 billion. The purchase price represents a 27.9% premium to Parexel’s unaffected closing stock price on May 5, 2017, and a 23.3% premium to the company’s undisturbed 52-week high.

Business

We did not attend the 2017 BIO International Convention earlier this week in San Diego, but we were told that it finished with a record-breaking total of 41,408 BIO Partnering meetings. We are convinced that some of those will turn into forthcoming successful deals in a short foreseeable future.

Approval of drugs and vaccines

The European Commission approves Novartis generic unit Sandoz’s Rixaton, a biosimilar version of Roche’s MabThera or rituximab, for the treatment of certain blood cancers, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis.

The European Medicines Agency (EMA) validates Shire’s Marketing Authorization Application for VEYVONDI to prevent and treat bleeding episodes and peri-operative bleeding in adults diagnosed with von Willebrand Disease (VWD), the most common inherited bleeding disorder. Already approved by the US FDA VEYVONDI is the first and only recombinant von Willebrand factor (rVWF) treatment for adults living with VWD.

The US FDA approves :

Novartis combination of Tafinlar or dabrafenib and Mekinist or trametinib for the treatment of patients with metastatic non-small cell lung cancer whose tumors express the BRAF V600E mutation. The combination, which had Breakthrough Therapy status, was approved in Europe in March for the same indication.

Melinta Therapeutic’s (New Haven, CON, USA) Baxdela or delafloxacin, a fluoroquinolone, for the treatment of acute bacterial skin and skin structure infections. Baxdela holds a Qualified Infectious Disease Product (QIDP) status which provides for an additional five-year period of market exclusivity for the indication.

Shire’s Mydayis, or SHP465, (mixed salts of a single-entity amphetamine product), as a treatment of attention deficit hyperactivity disorder (ADHD). Mydayis contains the same active ingredient than Adderall XR but is formulated to last up to 16 hours.

The European Medicines Agency, the US FDA and Japan's Pharmaceuticals and Medical Devices Agency agreed to align their requirements for antibiotic trials to help stimulate the development of new treatments.

Drugs at clinical stage

A US FDA Advisory Committee voted 17-2 to support a cardiovascular indication for Novo Nordisk’ Victoza, indicating that the antidiabetic drug can lower cardiovascular risks for high-risk Type 2 diabetes patients. In the assessed dossier, Victoza reduced major adverse cardiovascular events by 13% compared to placebo, cardiovascular deaths by 22%, any deaths by 15%, and advanced diabetic kidney disease by 22%.

The US FDA rejects Pfizer’s Epogen biosimilar for the second time, one month after an advisory committee gave an endorsement to this biosimilar version of Amgen's blockbuster. The FDA complete response letter (CRL) cited concerns about Pfizer’s fill-finish plant in McPherson, (Kansas, USA), whose problems led the FDA to already deny approval of a generic version of Teva’s Copaxone, and relates directly to matters noted in a warning letter issued in February 2017.

Novartis reports Phase 3 results showing that brolucizumab or RTH 258, a humanized single-chain antibody fragment with high affinity for all VEGF-A isoforms, as a treatment of neovascular age-related macular degeneration (wet AMD), showed to be non-inferior to Regeneron Pharmaceuticals and Bayer’s EYLEA or aflibercept. Brolucizumab showed efficacy over 48 weeks based on one injection every 12 weeks. EYLEA is administered every eight weeks (after a loading phase for both therapies).

Novartis reports highly positive Phase 3 results for canakinumab or ACZ885. Canakinumab, which works by in­hibit­ing IL-1ß, a key cy­tokine, for a pro­longed pe­riod, in reducing in­flam­ma­tion to lower risk to pa­tients. The drug sig­nif­i­cantly re­duced the risk of a com­pos­ite of car­dio­vas­cu­lar death, non-fatal my­ocar­dial in­farc­tion and non-fatal stroke in pa­tients with a prior heart at­tack and in­flam­ma­tory ath­er­o­scle­ro­sis. Canakinumab is already ap­proved for the treatment of Juvenile Idiopathic Arthritis, a rare disease and commercialized under the brand name Ilaris. Novartis announcement prompted Regeneron Pharmaceuticals to issue a statement clarifying its royalty rights to the drug which starts at 4% and moves up to 15% if annual sales of canakinumab exceed $1.5 billion.

Novartis’ CAR-T candidate CTL019, or tisagenlecleucel-T shows positive results in young patients with B-cell acute lymphoblastic leukemia (ALL). A US FDA Advisory Committee review, to assess the value of the drug as a treatment of ALL, is set for July 12.

Seattle Genetics announced it has discontinued a late phase clinical trial with Vadastuximab Talirine, as a treatment of frontline acute myeloid leukemia (AML), due to patient deaths, just three month after the US FDA has lifted its clinical hold. The deaths in the study in­cluded fatal in­fec­tions but were not as­so­ci­ated with liver tox­i­c­ity, which had al­ready killed 4 pa­tients, forc­ing the FDA to put a hold on the study in De­cem­ber 2016.

Medical Devices and Diagnosis News.

UK-based scientists have developed a new three-in-one blood test able to identify the patients, with advanced prostate cancer, most likely to benefit from PARP inhibitors, with the potential to “transform” treatment of the disease. By testing cancer DNA in the bloodstream, researchers from The Institute of Cancer Research (ICR), London, and The Royal Marsden NHS Foundation Trust, were also able to pick out patients not responding to treatment so that they could be switched to alternative therapy in just four to eight weeks.

Science & Technology

Researchers at the University of Southampton (UK) and La Jolla Institute for Allergy & Immunology (CA, USA), have identified a new type of immune cell that could be able to predict which lung cancer patients will benefit most from treatment with an immunotherapy. The study, published in Nature Immunotherapy, showed that lung cancer patients with a high level of tissue-resident memory T-cells in their tumour were 34% less likely to die.

Cost, Pricing and Market Access

The 142 pages US Senate Republic draft bill, called the Better Care Reconciliation Act of 2017, to repeal and replace the Affordable Care Act, or Obamacare has now been released and could cap federal payments to the states for most beneficiaries at the medical component of the Consumer Price Index starting in 2020. Senator Rand Paul of Kentucky says he and three other Republican senators are preparing to announce their opposition to the Senate health care bill as it is written, telling The Associated Press in an interview that the bill released Thursday resembles ‘‘Obamacare’’ too closely and does not go far enough to repeal former President Barack Obama’s law. Their opposition could put the bill in jeopardy because Senate majority leader Mitch McConnell can lose only two Republican senators and still pass the legislation.

Miscellaneous

Brazil-based Axis Biotec Brasil, in a joint venture with PlantForm Corporation (Canada) is set to construct a pilot production facility in Rio de Janeiro to support the development of a biosimilar version of AbbVie’s Humira using “tobacco-based bioreactors”. The platform could produce monoclonal antibodies up to 90% cheaper than mammalian systems. The plant will cost around $750,000 and will house PlantForm’s vivoXpress technology based on Nicotinia benthamia plants.

The Association of the British Pharmaceutical Industry (ABPI) suspends Astellas UK membership for a further 12 months, because of a number of “serious breaches” of the Code of Practice. According to the organisation, recent cases have shown “wholly inadequate oversight and control at both Astellas UK and Astellas Pharma Europe and a ‘lamentable lack of concern for patient safety’,” which nearly led to the company’s expulsion from the Association. Astellas UK was originally suspended in June last year because of breaches related to an advisory board meeting and deception, including providing false information to the ABPI’s policing arm the Prescription Medicines Code of Practice Authority by Astellas Pharma Europe.

Bioevents

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.