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

Editor’s Note

During your business holidays we will not publish our weekly Biopharmaceutical Newsletter. The next issue will be edited on September 3. We wish all your subscribers and readers a restful summer holiday season.

Zika virus

Florida health officials are investigating two possible cases of Zika that were not carried back by a traveler. If confirmed it would be the first evidence the virus has spread to mosquitoes in the continental US. All 1,306 American cases, including the 334 Florida ones, up to now have been in people who traveled to Zika-affected regions or their sexual partners. Brazilian scientists have found a second species, Culex quinquefasciatus, which can transmit the virus. Culex mosquitoes are far more common in temperate zones, such as in the United States.

Acquisitions /mergers/joint-ventures

Galenica (Berne, Switzerland) acquires Relypsa (Redwood, CA, USA) for $1.53 billion in an all cash deal and gains a US commercial organization as well as full rights for Veltassa, a drug that treats high level of potassium in patients with chronic kidney disease. Relypsa’s operations will fold into Galenica’s Vifor Pharma, which is set for a spin-off before 2019. Veltassa has the potential to become a $1 billion blockbuster drug now that the FDA has rejected AstraZeneca’s ZS-9 for the same indication and that Sanofi is co-marketing the drug worldwide.

Juno Therapeutics (Seattle, WA, USA) acquires RedoxTherapies (Boston, MA, USA) for $10 million upfront and gets access to vipadenant, a small molecule adenosine A2a-receptor antagonist to be combined with its own engineered T-cell drugs. Some preclinical models have shown that inhibiting the adenosine pathway by blocking A2a receptor signaling, enhanced the efficacy of adoptive T-cell therapy, checkpoint inhibitors, and vaccines across a number of cancers.

Zimmer Biomet (Warsaw, IN, USA) acquires a majority stake of Medtech (Montpellier, France), a medical robotic technology company, in an all cash transaction which values the company at $164 million.

Business

Celgene (Summit, NJ, USA) enters an agreement with Jounce Therapeutics (Cambridge, MA, USA) to get access to JTX-2011 and options for four more immunotherapies in a deal worth up to $2.5 billion.  Jounce Therapeutics is developing immunotherapies that use different approaches than the known PD-1 inhibitors by  targeting selected targets from a defined pool of B cell, T regulatory cell and tumor-associated macrophage targets. Celgene will make a $225 million upfront payment and investments worth $36 million and milestones fees valued at up to $2.3 billion.

Daiichi Sankyo (Tokyo, Japan) enters a sales and marketing deal with Amgen for nine products and expands its reach into the biosimilar market. Financial details of the deal were not disclosed.

Shire invests into Chronos Therapeutics (Oxford, UK) with equity stake giving it access to three central nervous system programs in returns for milestones and an opportunity to buy back the drugs at a later stage. The programs consists of drugs against fatigue in multiple sclerosis, addictive behaviors and post-traumatic stress disorder. Financial terms of deal were not disclosed.

Galderma (Lausanne, Switzerland) acquires rights to Roche and Chugai’s nemolizumab, an IL-13 receptor humanized antibody, for the treatment of atopic dermatitis and pruritus, in all countries except Japan and Taiwan. The deal represents the first move of Galderma into the biological world and follows LEO Pharma who made a similar move earlier this month. Both companies are clearly fighting with Sanofi/Regeneron’s dupilumab for a share of innovative products in the atopic dermatitis market. Explicit financial terms of the deal were not disclosed.

Nascent Biotech (Vero Beach, FL, USA) grants an exclusive license to Zheijiang Hisun Pharmaceuticals (Taizhou, China) to get China rights to pritumumab, an epithelial monoclonal antibody to treat brain and pancreas cancers. Financial details were not disclosed.

Approval of drugs and vaccines

The European Commission approves:

Gilead’s Epclusa, a once-daily fixed combination therapy of sosfosbuvir and velpatasvir for the treatment of hepatitis.  Epclusa is the first all-oral, pan-genotypic, single tablet regimen cleared for the treatment of adults with genotype 1-6 chronic HCV and was approved by the US FDA last month.

AstraZeneca’s Qtern, a fixed-dose combination of saxaglitipin and dapaglizofin for the treatment of type 2 diabetes, less than two months after the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion.

….and Celgene’s Revlimid, or lenalidomide, to treat patients with relapsed refractory Mantle Cell Lymphoma, a rare sub-type of aggressive non-Hodgkin’s lymphoma. The drug is already approved for the treatment of newly diagnosed and relapsed/refractory multiple myeloma and myelodysplastic syndromes.

US FDA approves CSL Behring’s (King of Prussia, PA, USA) Berinert, a C1 esterase inhibitor (human), for use in patients under 12 years of age with acute facial, abdominal or laryngeal hereditary angioedema attacks. Berinert is the only approved treatment for patients under 12 with the condition.

US FDA grants extended approval:

to Eli Lilly and Boerhinger Ingelheim’s Synjardy a fixed combination of empagliflozin and metformin, as an adjunctive treatment along with diet and exercise for the treatment of type diabetic patients who have not received prior treatment.

…and to Janssen’s Therapeutic’s Prezista, or duranavir, an HIV-1 treatment for use in pregnant and postpartum patients.

China CFDA grants GlaxoSmithKline market authorization for Cervarix, a human papillomavirus vaccine, for women ages 9 to 25 using a three-injection schedule

Drugs at clinical stage

The European Commission grants Cell Medica (London, UK) orphan designation for CMD-003 or baltleucel-T, for the treatment for extranodal NK/T-cell lymphoma, a rare type of non-Hodgkin lymphoma, and post-transplant lymphoproliferative disorder, a condition caused by B-cell proliferation due to therapeutic immunosuppression after organ transplantation.

US FDA grants breakthrough status:

To AveXis’ (Princeton, NJ, USA) AVXS-101 for the treatment of children with spinal muscular atrophy type 1.

…and to Pfizer and Spark Therapeutics’ (Philadelphia, PA, USA) SPK 9001 for the treatment of haemophila B patients. SPK-9001 is a novel bio-engineered adeno-associated virus capsid expressing a codon-optimised, high-activity human factor IX variant, as a potential one-time therapy.

US FDA grants fast-track status:

To Advaxis’ (Princeton, NJ, USA) AXAl, or axalimogene filolisbac, as an adjuvant treatment for high-risk locally advanced cervical cancer.  Axalimogene filolisbac is an immunotherapy based on the Lm Technology, which uses live attenuated bioengineered Listeria bacteria to deliver a fusion protein inside cancer cells which then stimulates the immune system to attack the cancer.

To ProQR Therapeutic’s (Leiden, The Netherlands) QR-010 as a treatment for cystic fibrosis in patients with delta F508 mutation. QR-010 is a first-in-class RNA (ribonucleic acid)-based oligonucleotide designed to tackle the underlying cause of cystic fibrosis by repairing the mRNA in patients with the F508 mutation. The drug also holds orphan-drug status in the US and in Europe.

To Auris Medical Holding’s (Zug, Switzerland) Keyzilen or AM-101, a small molecule N-methyl-D-aspartate (NMDA) receptor antagonist formulated in a biocompatible gel for intratympanic injection, for the treatment of acute peripheral tinnitus following otitis media or cochlear injury.

…and to Cerulean’s (Waltham, MA, USA) CRLX101, a nanoparticle-drug conjugate, for the treatment of platinum-resistant ovarian, fallopian tube or primary peritoneal cancer.

US FDA grants orphan status to Oncternal Therapeutic’s (San Diego, CA, USA) TK216 for the treatment of Ewing sarcoma, a rare pediatric cancer

Roche’s Gazyva, a blood cancer drug, has failed to show significant improvements versus MabThera/Rituxan in patients with previously untreated diffuse large B-cell lymphoma,

Medical Devices and Diagnosis News

EchoPixel (Mountain View, CA, USa) teams up with Hewlett-Packard to roll out virtual reality medical imaging system in clinics. EchoPixel’s True 3D system creates holographic images that can be viewed in real-time during surgery.

The US FDA has released a draft guidance advising companies that are developing in vitro companion diagnostics and therapies at the same time to meet with appropriate FDA review centers before starting clinical trials and to ensure that the development timelines of both products are synchronized so they are approved around the same time. Early integration of in vitro diagnostics into clinical trial design is recommended in the guidance.

Cost, Pricing and Market Access

The UK National Institute for Health and Care Excellence (NICE) has endorsed the use of Eli Lilly’s Almita or pemetrexed for the maintenance treatment of locally advanced or metastatic non-squamous non-small-cell lung cancer in adults

Miscellaneous

Merck KGaA (Darmstadt, Germany) announces the launch of a $115 million 280,000-square-foot life science campus in Burlington (MA, USA). The 850 full-time employees currently located in Billerica (MA, USA) will relocate to the new facility when construction is complete in 2017. “As a global leader in life science, we are committed to strengthening our presence in one of the most important science and technology hubs in the world,” Udit Batra, Merck’s life sciences CEO, said in a statement.

According to the Straits Time (Singapore) three medical researchers, who had published high-profile papers on the understanding of myostatin, a "fat-burning" protein that could trim weight and aid in the treatment of obesity and diabetes, have been accused of fraudulent conduct. The lead researcher has been terminated from appointments at NTU's School of Biological Sciences and the Singapore Institute for Clinical Sciences,  which both come under the Agency for Science, Technology and Research (A*Star), the newspaper said.

Bioevents

BIO Pharm America on September 13-15 in Boston (USA)

7^th Innovation Days on October 3-4 in Paris (France)

BIO Europe on November 7-9 in Cologne (Germany)

BIO Latam on November 29-30 in San Juan (Puerto Rico)

Biofit 2016 on November 30-December 1 in Lille (France)

IMPORTANT NOTICE

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.