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

Mergers, Acquisitions and Joint Ventures

JSR Life Sciences (Tokyo, Japan) acquires Selexis (Geneva, Switzerland) a cell-line development company and integrates it with KBI Biopharma, a US contract development and manufacturing organization. Selexis’ SUREtechnology Platform™ can be used to generate stable cell lines, producing just about any recombinant protein. Financial details of the deal were not disclosed.

Business

Johnson & Johnson Innovation launches multiple collaborations with universities, institutes and firms to allow use of its units and libraries of chemical compounds for studies on treatments of various diseases, including rheumatoid arthritis, obesity, nonalcoholic steatohepatitis (NASH), chronic kidney disease and Chagas disease. Among the partners were the University of California at San Diego, Monash University, the National Institute of Allergy and Infectious Diseases, Australia's Walter and Eliza Hall Institute of Medical Research and Belgium-based Clarity Genomics.

Numab Therapeutics (Pfäffikon, Switzerland) and Kaken Pharmaceutical (Tokyo, Japan) have established a collaboration and option agreement focused on identifying a multispecific antibody candidate for inflammatory diseases. Numab will use its discovery and engineering platform to identify a candidate, to which Japanese specialty pharma Kaken retains a license and co-development option. The collaboration with Kaken is Numab’s second deal with a Japanese company. In March, Numab reported an agreement with Ono Pharmaceuticals to identify a multispecific antibody candidate for applications in immuno-oncology. Financial terms of the deal were disclosed.

Anagenesis Biotechnologies (Strasbourg, France) enters a strategic collaboration with Boehringer Ingelheim to discover treatments for chronic muskoskeletal diseases such as sarcopenia and cachexia. Anagenesis Biotechnologies develops paraxial mesoderm-derived lineages (striated muscle, brown fat, cartilage, bone, dermis & endothelial cells) to generate new treatments against human diseases. The strategic agreement grants Boehringer Ingelheim an exclusive access to Anagenesis’ proprietary P2MCs technology for chronic muscle diseases

Approval of drugs and vaccines

No significant event this week.

Drugs at clinical stage

The European Medicines Agency grants orphan status to Mithar Pharmaceuticals’ (Liège, Belgium) esterol, or E4, an estrogen candidate for the treatment of newborn patients with hypoxic ischemic encephalopathy.

The US FDA grants breakthrough status to Omeros’ (Seattle, WA, USA) OMS721, a monoclonal drug candidate for the treatment of IgA neuphropathy. OMS721 binds to an enzyme called mannan-binding lectin-associated serine protease-2 (MASP-2) which plays a key role in the lectin pathway of the complement system, which is believed to contribute to significant tissue injury.

The US FDA grants Rare Pediatric Disease Designation to Fibrocell Science’s (Exton, PA, USA) gene therapy candidate FCX-013 for the treatment of moderate-to-severe localized scleroderma. The drug already had an orphan drug status. A Rare Pediatric Disease voucher can be used for accelerated approval of a future product or it can be transferred or sold.

The US FDA grants fast-track status to Pfizer and Eli Lilly’s tanezumab for the treatment of chronic pain in patients with osteoarthritis and chronic back pain and fight the current opoid epidemic. Tanezumab is a humanized monoclonal antibody that inhibits nerve growth factor, the first NGF inhibitor so designated.

Centrexion Therapeutic’s (Boston, MA, USA) CNTX-4975, a synthetic version of trans capsaicin, a medicine traditionally extracted from chili plant, proved to be an effective knee pain killer among osteoarthritis patients, for up to six months

Sanofi and Regeneron Pharmaceuticals’ Praluent, a PCSK9 inhibitor, reported two late phase studies in diabetic patients, where the drug met its primary endpoints in enabling most patients to reduce their lipid levels.

Following initial results of the Fourier trial, the Institute for Clinical and Economic Review, (ICER) a Boston-based cost watchdog, indicated that the current value-based price benchmark of Amgen’s Repatha, a PCSK9 inhibitor, at $5,405 to $7,735 is too high.

Merck & Co stops the enrollment on two combination phase 3 studies for the treatment of multiple myeloma, with Keytruda, its checkpoint inhibitor, following a high rate of deaths. The tri­als in­volve com­bi­na­tions of Keytruda with Cel­gene’s Po­m­a­lyst as well as Celgene’s Revlimid. Keytruda is currently being investigated in a dozen of different combination studies.

Medical Devices and Diagnosis News.

Sanofi teams up with PAREXEL to explore the use of patient sensor and wearable devices to remotely collect patient data during clinical trials.

Science & Technology

Scientists from A*STAR’s Singapore Immunology Network (SIgN) and KK Women’s and Children Hospital (KKH) in Singapore have discovered that a fetus’s immune system is established as early as the second trimester of pregnancy, and may be able to initiate immune responses independently of the mother’s immune system. These findings debunk commonly-held assumptions about fetal immunity, including the idea that the fetus’s immune system develops much later in the pregnancy cycle, and is unable to mount an independent immune response, being dependent on the mother’s immune system to defend itself against external pathogens. The scientists also found that an unborn baby’s immune system contains a unique mechanism to prevent rejection of the mother’s cells, even as it develops independently. This mechanism is mediated by dendritic cells expressing the protein Arginase-2.

UK Scientists have shown that the use of daily aspirin, as a blood thinner in people aged over 75 years is linked with a much higher risk of major and potentially fatal stomach bleeds than previously thought. While short-term use of aspirin following a stroke or heart attack has “clear benefits”, the authors of the study, led by Oxford University, argue that patients over 75 who take the drug on a daily basis should be prescribed a proton-pump inhibitor to reduce the risk of bleeding.

Cost, Pricing and Market Access

The UK National Institute for Health & Care Excellence (NICE) recommends the use of Eli Lilly’s Lartruvo for the treatment of soft tissue sarcoma. The Final Appraisal Determination was published one month earlier than expected, and is one of the first medicines to be made available within the new Cancer Drugs Fund, as well as the first monoclonal antibody to be recommended in combination with doxorubicin to treat the condition.

Following four years of battle, the UK National Institute for Health & Care Excellence (NICE) has now published a Final Appraisal Determination (FAD) recommending, the use of routine use of Roche’s Kadcyla for women who have advanced and inoperable HER2-positive breast cancer even after treatment with Herceptin and a taxane, separately or in combination.

… but rejects Roche’s Esbriet for the treatment of  patients with moderate forms of the lung scarring disease idiopathic pulmonary fibrosis (IPF). IPF is a fatal disease caused by irreversible, progressive scarring of the lungs, which makes breathing difficult and prevents the heart, muscles and vital organs from receiving enough oxygen to work properly.

The US Senate Health, Education, Labor and Pensions (HELP) Committee and the White House are working on a bipartisan plan to address drug pricing. Lamar Alexander (R-Ky.), the committee's chair, said the committee will hold a second hearing in July "to hear about the process — beginning with a manufacturer’s development of a drug, the different steps through which the drug travels before arriving in the patient’s hands, how this is paid for, and what the costs are at each of the different steps."

Roche has been accused of charging too much for its breast cancer drug trastuzumab by South African authorities. Competition commissioner Tembikosi Bonakele accused Roche and Genentech of engaging in “excessive price discrimination and exclusionary conduct in the provision of breast cancer medicine in South Africa”.

Miscellaneous

Sanofi will invest up to $2 billion on its biologics manufacturing over the next several years. Philippe Luscan, executive VP of global industrial affairs, said that the company will invest €600 million ($673 million) a year on its biologics production over the next two to three years. “The investments will reinforce our three major hubs in the U.S., Germany and France,” where Sanofi is doing production of biologics like monoclonal antibodies (mAbs), spokesman Nicolas Kressmann said in a telephone interview. The investments will be for capacity expansion and hiring at different sites in those hubs, he said. These investments come on top of the €270 million Sanofi and Lonza made earlier this year at Lonza’s site in Visp (Switzerland), which they will share for the production of biologics.

Medicxi (London UK), a European venture group, launches a new $300 million fund, that will focus on late-stage biotech investments, with the backing of Novartis and Google Verily. The focus for this Medicxi Growth 1 — MG1 — fund will start on pro­grams at the Phase IIb level, man­aged by fully de­vel­oped com­pa­nies .The fund will be of­fer­ing an al­ter­na­tive route to com­pa­nies that might oth­er­wise feel the need to launch an IPO, part­ner out on un­fa­vor­able terms or look too early to the US mar­kets for the fi­nan­cial support needed to ex­e­cute a Phase III ef­fort.

People on the move

Harvard University President Drew Gilpin Faust, will step down next year after 11 years leading the 380-year-old institution.

Jean-Paul Kress (former Sanofi Pasteur MSD and former Sanofi Genzyme) has been appointed as as Executive Vice President and President, International, and Head of Global Therapeutic Operations at Biogen (Cambridge, MA, USA)

Bioevents

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/

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