Editor’s Note

New drug approvals hit a record high in 2014 and one can expect a similar figure in 2015. Among these new approvals some will constitute real innovative breakthrough therapies but they often come at a very high cost. Several Institutes and Non-Profit organizations have published studies evaluating the evidence on the value of several new treatments and have indicated that some of the recently launched drugs were vastly overpriced. Some payers have now gone as far as removing highly costly drugs from their funding lists, other have decided to not reimburse or postpone reimbursement of novel treatments. We will report on this major issue whenever consolidated information becomes available.

Acquisitions /mergers/joint-ventures

Actelion (Allschwil, Swizterland) has entered acquisition talks with ZS Pharma (San Mateo, CA, US) and has reportedly made a $2.5 billion offer. ZS-9, ZS Pharma’s lead investigational drug is aimed at treating hyperkaliemia and prevent heart failure.

Amgen acquires Dezima Pharma (Naarden, The Netherlands) from Forbion Capital Partners in a deal worth up to $1.55 billion and boosts its cardiovascular portfolio. Dezima’s lead product, TA-8995, is an orally active Cholesteryl Ester Transfer Protein (CETP) inhibitor for the treatment of dyslipidemia. Under the terms of the deal Dezima shall receive a $300 million upfront payment and up to 1.25 billion in further milestones and sales royalties.

Business

Daiichi Sankyo gets an exclusive worldwide license to develop and market Translational Sciences (Memphis, TN, USA) TS23, a clot dissolving antibody and boost its thrombosis pipeline. “TS23 inactivates a2-antiplasmin (a2AP) – a major inhibitor of clot-dissolving enzyme plasmin – and dissolves clots through a unique mechanism that avoids the risk of haemorrhage and neurotoxicity associated with TPA therapy” according to the company. Under the terms of the agreement, Translational Sciences will receive undisclosed milestone payments as well as sales royalties.

NantPharma has entered into an exclusive license agreement to develop and market Amgen‘s cancer drug AMG 337.  The compound, the second one NantPharma licenses from Amgen, is a small molecule inhibitor of the cell surface enzyme c-Met, a known target to tackle gastric and esophageal cancers. Financial terms were not disclosed.

Sanofi enters into a discovery agreement with PeptiDream (Tokyo, Japan). Under the terms of the deal PeptiDream will use its proprietary peptide discovery platform to generate peptides of interest against targets selected by Sanofi. Financial terms were undisclosed.

Polyphor (Allschwil, Switzerland), a privately held company specialized in the discovery and development of macrocycle drugs, enters into research collaboration with Taisho Pharmaceuticals (Tokyo, Japan) to identify novel drug candidates. Financial terms of the agreement were not disclosed.

Celgene enters a partnership with Nurix (San Francisco, CA, USA) focused on the discovery, development and commercialization of small-molecule drugs for cancer, inflammatory and immunology indications that function through the ubiquitin proteasome system. Under the terms of the agreement, Nurix will receive an upfront payment of $150 million, an undisclosed equity investment plus sales royalties and potential milestone and option fees of up to $405 million and Celgene will get global rights of products arising from the collaboration.

The US Departement of Health and Human Services (HHS) enters into a five-year collaboration with AstraZeneca to develop novel antibiotics caused by bioterrorism agents and antibiotic multi-resistant infections. The Biomedical Advanced Research and Development Authority (BARDA) of the HHS will make an initial payment of $50 million and up to an additional $150 million for the development of additional products. The first product to be explored is a combination of two known antibiotics, Aztreonam and Avibactam.

Bavarian Nordic (Kvistgard, Denmark) could get as much as $33 million from Johnson & Johnson to improve stability of their joint Ebola vaccine.

Approval of drugs, vaccines, diagnostics and devices

US FDA approves Octapharma’s Nuwiq, a B-domain deleted, human cell line-derived recombinant factor VIII not chemically modified or fused with another protein, for the control of bleeding in patients with haemophilia A. The treatment was already approved in Europe in August 2014. Nuwiq is not indicated for the treatment of von Willebrand disease.  

Swissmedic approves Novartis’ Entresto (sacubitril/valsartan), previously known as LCZ696, to reduce the risk of cardiovascular mortality and morbidity in patients with heart failure with reduced ejection fraction

Drugs at clinical stage

Erytech Pharma (Lyon France) submits its first ever European Medicines Agency (EMA) marketing authorization for ERY-SP (GRASPA) to treat patients with acute lymphoblastic leukemia (ALL).  ALL is a blood cancer affecting mainly the white blood cells and is most prevalent for children between the ages of two and five. ERY-ASP consists of an enzyme, L-asparaginase, encapsulated inside donor-derived red blood cells. L-asparaginase depletes asparagine, a naturally occurring amino acid essential for the survival and proliferation of cancer cells, from circulating blood plasma.

Sanofi anti-diabetes combination drug LixiLan meets its primary end-point of superior HbAc1 reduction in a late-stage study to Lantus.  LixiLan is a fixed-dose combination of insulin glargine (Lantus) and lixisenatide (Lyxumia) a GLP-1 receptor antagonist. The efficacy was evaluated over a 30-week period in patients not adequately controlled on basal insulin alone or in combination with oral anti-diabetic drugs. 

….but sees Novo Nordisk’s claiming superiority of its GLP-1 drug Victozia (liraglutide), over Sanofi’s Luximia (lixisenatide) in reducting HbAc1 levels.

US FDA designates Bristol-Myers Squibb checkpoint inhibitor Opdivo (nivolumab) a Breakthrough Therapy for the treatment of advances metastatic renal carcinoma. Opdivo is already approved for the treatment of metastatic melanoma and advanced squamous non-small cell lung cancer.

Science and Technology

In a short recent study, co-authored by Max Donelan from the Simon Fraser University in Canada and published in Current Biology, it was shown that our nervous system, responsible for movements like walking, continuously optimizes movement, in real time, to use the least amount of energy possible.  “This is good news if you’re an athlete,” Donelan says. “You’d like your body to be helping you move as cheaply as possible if you’re trying to win a race so you have as many energy reserves as you possibly can. But if you’re exercising to sweat off calories, it’s not great news”, he says.

InfoPeHR Technologies, a Florida based company, founded by Bernard Brigonnet (former CEO of the Medicen Paris Region Biocluster) has developedInfoPeHR®, the first portable personal Electronic Health Record (EHR) containing patient’s medical history, complete with imaging in diagnostic quality.The product uses WAAVES©, a revolutionary imaging compression technology, hundreds of time more powerful than Jpeg, and restoring images in near original quality. The WAAVES© technology was initially developed by Sylvain Hochberg within the CIRA company (Paris, France).

Medical Devices and Diagnosis News

Royal Philips intends to roll out a series of projects for various chronic diseases that combine consumer-oriented devices data with electronic medical records and will start first with diabetes. The Philips system includes a patient application and online community. It will connect to the patient's electronic medical record, as well as multiple personal health devices such as blood glucose monitors and activity trackers.

Miscellaneous

US President Barack Obama has nominated Dr. Robert Califf, a prominent cardiologist, to lead the Food and Drug Administration. When confirmed by the Senate, Dr. Califf, who is the FDA deputy commissioner since March, will succeed to Margaret Hamburg, who announced her resignation earlier this year.

Eli Lilly and Boehringer Ingelheim announced at the 51^st European Association for the Study of Diabetes in Stockholm that Jardiance (empaglifozin) was the first SGLT-2 drug to show it could lower the combined risk of heart attack, stroke and death from cardiovascular causes by 14% in high risk type 2 diabetes patients. The EMPA-REG study was a long-term, multicenter, double-blind, placebo controlled study involving 7,000 people with diabetes at high risk of cardiovascular event. The mean observation period was 3.1 years.

In our previous newsletter we had indicated that the Institute for Clinical and Economic Review (ICER), a US cost watchdog, had indicated that Amgen’s Repatha and Sanofi/Regeneron’s Praluent were vastly overpriced. This week ICER published that it was not so with Novartis new heart failure drug Entresto, priced at $4,600 per-year. According to ICER “the price is well-aligned with the degree of benefit it brings to patients and that Entresto is "cost-effective" in the long term, judged according to "commonly accepted" benchmarks. Entresto, also known as LCZ696, is acombination drug consisting of two antihypertensive drugs valsartan and sacubitril in a 1:1 mixture which has been approved by the US FDA in July an d this week in Switzerland.

NICE has issued a preliminary rejection for the use of Novartis’s Farydak (panobinostat) by the National Health Service (UK). Farydak is the first cancer medicine to target enzymes known as histone deacetylases, offering a novel mechanism of action different from other multiple myeloma treatments on the market which was approved earlier this month in Europe.

…and for Clegene’s Abraxane (nab paclitaxel), in combination with gemcitabine, for the treatment of pancreatic cancer on grounds that the cost does not justify its “limited benefits”. 

Business intelligence provider GBI Research projects the global multiple myeloma treatment market value to jump from $7.3 billion last year to $8.9 billion by 2021. This increase represents a moderate Compound Annual Growth Rate (CAGR) of 4.6%. According to the study current multiple myeloma treatments may progressively be supplemented by improved treatments, specifically in the relapsed/refractory setting. Some of the new treatments are ixazomib, filanesib, elotuzumab, and daratumumab. The economic impact of these novel potential treatments should be rather low and the market value growth should mainly be due to an increased prevalence in the aging population.

Merck Serono, the biopharmaceutical division of Merck KGaA, invests €65 million to expand its R&D facility at Darmstadt Headquarters. The 16,000 square meters laboratory building will host 200 researchers when complete in 2017 and further demonstrate that “the Darmstadt site will continue to serve as a core R&D hub for Merck”.

Bioevents

• 15^th Annual Biotech in Europe Forum for Global Partnering & Investment on September 29-30 in Basel (Switzerland)
• Innovation Days on October 5-6 in Paris (France)
• FierceBiotech Executive Summitt on October 12 in London (UK)
• BIO Japan on October 13-16 in Yokohama (Japan)
• BIO Latin America on October 14-16 in Rio de Janeiro (Brazil)
• BIO Investor Forum on October 20-21 in San Francisco (USA)
• BIO Europe 2015 on November 2-4 in Munich (Germany)
• Biofit 2015 on December 1-2 in Strasbourg (France)

Author: Jean-Claude Muller, Special Advisor at I&IR, jcm@btobioinnovation.com

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