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Incyte Achieves $25 Million Milestone for Approval of Jakavi® (ruxolitinib) in Japan

On July 8, 2014 Incyte reported that it has earned a $25 million milestone payment from Novartis in connection with the approval of Jakavi (ruxolitinib) in Japan for the treatment of patients with myelofibrosis (Press release Incyte, JUL 8, 2014, View Source [SID:1234500622]). Incyte will record this amount as contract revenue in the third quarter. Under the Incyte-Novartis Collaboration and License Agreement, Novartis received exclusive development and commercialization rights to ruxolitinib outside of the United States for all hematologic and oncologic indications and sells ruxolitinib under the name Jakavi. Ruxolitinib is marketed by Incyte in the United States as Jakafi (ruxolitinib).

“We are very pleased with the progress that Novartis is making in the global roll-out of Jakavi,” stated Hervé Hoppenot, President and Chief Executive Officer of Incyte. “The recent approval of Jakavi in Japan is further evidence of that progress.”

Novartis also continues to make progress in obtaining formal pricing and reimbursement approval for a third major European country and expects this to occur in the second half of 2014. Once achieved, Incyte will earn an additional $60 million milestone payment.

Austrian Biotech Company “APEIRON” Announces Start of its First Clinical Trial for Neuroblastoma Patients in Japan

On July 7, 2014 APEIRON Biologics reported that a phase I clinical trial with its neuroblastoma immunotherapy APN311 has received all necessary approvals to start recruiting patients. The trial will be locally conducted by Nagoya University Hospital and is part of a long-term collaboration to make this therapy available to patients suffering from this severe type of children’s cancer (Press release, Apeiron Biologics, JUL 7, 2014, View Source [SID:1234502247])r. In Japan, it won a grant by the Japanese government as an Investigator-initiated clinical trial that utilizes collaboration between the Department of Pediatrics and the Center for Advanced Medicine and Clinical Research (CAMCR) at Nagoya University Hospital. "This is a great achievement for us as we have supported this joint effort from the very beginning. Two years ago the ground was prepared when the Austrian Embassy – Commercial Section in Tokyo hosted a scientific meeting for Japanese pediatric oncologists to learn about this innovative therapy of pediatric neuroblastoma developed by Apeiron ", says Dr. Martin Glatz, Commercial Counsellor of the Austrian Embassy. "The meeting two years ago was part of a focus program aiming to bring more Austrian medical research and biotech companies to the Japanese market. The market has seen tremendous changes recently with companies exploring new opportunities and the government addressing regulatory issues," he added. Hans Loibner, PhD, CEO of Apeiron, commented, "We are very happy and proud to be rewarded with this milestone achievement after all the effort that was invested. I would like to particularly thank the physicians from Nagoya University, Dr. Seiji Kojima and Dr. Yoshiyuki Takahashi, as well as Dr. Masaaki Mizuno, Dr. Katsuyoshi Kato and Dr. Shinobu Shimizu at CAMCR, as we owe it to their dedication and tireless work that this trial can now start. We are confident that Japanese patients will benefit from the introduction of APN311 to Japan and look forward to making the next steps towards approval of this promising antibody therapy of neuroblastoma by the Japanese regulatory authorities."

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6-K – Report of foreign issuer [Rules 13a-16 and 15d-16]

On July 7, 2014 Compugen reported that it has achieved the initial milestone in the cancer immunotherapy collaboration it entered last year with Bayer HealthCare (Bayer) (Filing 6-K , Compugen, JUL 7, 2014, View Source [SID:1234500619]). The collaboration provides for the development and commercialization of therapeutic antibodies against two checkpoint protein candidates discovered by Compugen. The milestone being announced today relates to the first preclinical milestone for one of two checkpoint protein candidates for which Compugen will receive a $1.2 million payment out of the $30 million potential milestone payments associated with joint preclinical research for the two programs.

Dr. Anat Cohen Dayag, Compugen’s President and CEO, stated, “We are very pleased by the achievement of this initial drug development milestone for one of the two programs in our collaboration with Bayer. After investing more than a decade of extensive multidisciplinary research in establishing our broadly applicable predictive discovery infrastructure, we selected the area of checkpoint-based cancer immunotherapy as our first focused discovery effort. Therefore, it is extremely satisfying to see our growing competitive position, in terms of both advancement of our therapeutic programs in immuno-oncology and continuing discoveries of novel targets in this exciting area, which is increasingly being viewed as a potential major breakthrough in cancer treatment.”

About the Bayer HealthCare/Compugen Collaboration and License Agreement
The collaboration and license agreement provides the framework for the research, development, and commercialization of antibody-based therapeutics for cancer immunotherapy against two novel Compugen-discovered immune checkpoint regulators. Under the terms of the agreement, Bayer and Compugen are jointly pursuing a preclinical research program for each of the two candidates. Subsequently, Bayer will have full control over further development and have worldwide commercialization rights for potential cancer therapeutics.

Under the terms of the agreement, Compugen has received an upfront payment of $10 million and is eligible to receive over $500 million in potential milestone payments for both checkpoint programs, plus an additional $30 million of potential milestone payments associated with joint preclinical research for the two programs. Compugen is also eligible to receive royalties on global net sales from any resulting products under the collaboration.

Novartis personalized cell therapy CTL019 receives FDA Breakthrough Therapy designation

On July 7, 2014 Novartis reported that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy status to CTL019, an investigational chimeric antigen receptor (CAR) therapy for the treatment of pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL) (Press release Novartis, JUL 7, 2014, View Source [SID:1234500616]). The Breakthrough Therapy filing was submitted by the University of Pennsylvania’s Perelman School of Medicine (Penn) which has an exclusive global agreement with Novartis to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers.

This is the fifth Breakthrough Therapy designation for Novartis, continuing the company’s trajectory as a leader in developing innovative therapies to help treat diseases in which there remains significant unmet medical need. Novartis’ Zykadia(TM) (ceritinib, previously known as LDK378), for the treatment of anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC), is one of the first medicines to receive an FDA approval following earlier receipt of Breakthrough Therapy designation by the FDA.

"This Breakthrough Therapy designation underscores the potential of CTL019 as a life-saving therapy for patients with relapsed/refractory ALL, who are in desperate need of new treatment options," said David Epstein, Division Head, Novartis Pharmaceuticals. "Novartis welcomes increased dialogue with the FDA and a potentially expedited review to streamline the development of CTL019 and hopefully bring this promising therapy to patients as quickly as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the fast track program features, as well as more intensive FDA guidance. It is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

"This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population," said Carl H. June, M.D., Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine and director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania. "We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in Phase II trials."

Novartis recently established the Cell and Gene Therapies Unit under the leadership of Usman Azam, Global Head, to bring an intense focus on advancing innovative cell-based therapies, including the development of CARs. Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019.

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Nexavar® (sorafenib tablets) now Approved in Canada for the Treatment of Differentiated Thyroid Cancer

On July, 7, 2014 Bayer reported that the Health Canada approval of Nexavar for the treatment of patients with locally advanced or metastatic, progressive, differentiated thyroid carcinoma (DTC), refractory to radioactive iodine (RAI) (Press release Bayer, JUL 7, 2014, View Source [SID:1234500615]).

The approval is based on data from the Phase III DECISION (stuDy of sorafEnib in loCally advanced or metastatIc patientS with radioactive Iodine refractory thyrOid caNcer) trial. In the trial, sorafenib significantly extended progression-free survival (PFS), the primary endpoint of the study, compared to placebo (HR=0.59 [95% CI, 0.45-0.76]; p<0.0001), which represents a 41 percent reduction in the risk of disease progression or death for patients who received sorafenib compared to placebo-treated patients. The median PFS was 10.8 months in patients treated with sorafenib, compared to 5.8 months in patients receiving placebo. The safety and tolerability profile of sorafenib in patients in the trial was generally consistent with the known profile of sorafenib. The most common treatment-emergent adverse events in the sorafenib arm were hand-foot skin reaction, diarrhea, alopecia, weight loss, fatigue, hypertension and rash. Results from the trial were presented at the Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) in June 2013 and published in The Lancet in April 2014.