On July 10, 2014 Bristol-Myers Squibb reported that it, following discussions with the U.S. Food and Drug Administration (FDA), the company is planning a third quarter submission of a Biologics Licensing Application (BLA) for Opdivo (nivolumab) for previously treated advanced melanoma (Press release Bristol-Myers Squibb, JUL 10, 2014, View Source [SID:1234500639]). This will mark the second tumor type for which Bristol-Myers Squibb has a regulatory submission underway for Opdivo in the U.S.

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"We continue to collaborate closely with the FDA on Opdivo and the planned submission in advanced melanoma represents an important step forward in our company’s commitment to deliver innovative treatment options for patients with cancer," said Michael Giordano, MD, Head of Oncology Development, Bristol-Myers Squibb.

The advanced melanoma BLA is based on data from Checkmate -037, a multinational, multicenter, randomized open-label Phase 3 trial evaluating Opdivo compared to dacarbazine (DTIC) or carboplatin/paclitaxel in patients with unresectable or metastatic melanoma who have been previously treated with Yervoy (ipilimumab) and, if BRAF-mutation positive, a BRAF inhibitor regimen.

Bristol-Myers Squibb has proposed the name Opdivo (pronounced op-dee-voh), which, if approved by health authorities, will serve as the trade name for nivolumab.

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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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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On July 4, 2014 Ono Pharmaceutical reported that it had received manufacturing and marketing approval for the human anti-human PD-1 monoclonal antibody "OPDIVO Intravenous Infusion 20 mg/100 mg"("OPDIVO") for the treatment of unresectable melanoma (Press release Ono, JUL 4, 2014, View Source [SID:1234500642]).

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Melanoma is considered to be a form of tumor characterized by the malignant transformation of pigment-producing cells located in the skin. In Japan, there has been an unmet need for an effective treatment for patients with surgically unresectable melanoma, who have an extremely poor prognosis that no treatment exists to significantly improve.

OPDIVO is a human anti-human PD-1 monoclonal antibody. PD-1 (programmed death-1), a receptor expressed on the surface of lymphocytes, plays a role in a regulatory pathway that suppresses activated lymphocytes in the body (negative signal). Available evidence suggests that cancer cells exploit this pathway to escape from immune responses. OPDIVO is thought to provide benefit by blocking PD-1-mediated negative regulation of lymphocytes (i.e., the interaction of PD-1 with its ligands PD-L1 and PD-L2), thereby enhancing the ability of the immune system to recognize cancer cells as foreign and eliminate them. OPDIVO is the world’s first approved drug targeting PD-1.

"We are delighted to obtain a manufacturing and marketing approval as a drug targeting PD-1, which receives a lot of attention in tumor immunity, for the first time in the world." said Gyo Sagara, the President and Representative Director of ONO. "ONO would like to obtain approvals for additional indications on ongoing development for other cancers to bring many patients OPDIVO as soon as possible."

Accumulating further clinical data is important in ensuring that OPDIVO will be used more safely and effectively. ONO is committed to taking actions necessary for the proper use of OPDIVO by implementing a post-marketing use-results survey (all-case surveillance) and collecting clinical data on the safety and efficacy of OPDIVO pursuant to the conditions for its approval.

Because of the very limited number of patients treated with OPDIVO in Japanese clinical trials, ONO is required to perform a post-marketing use-results survey covering all cases until data on a certain minimum number of patients have been accumulated. Through these activities, ONO should identify the characteristics of patients to be treated with OPDIVO and collect safety and efficacy data as soon as possible, thereby taking actions necessary to ensure the proper use of OPDIVO

On July 3, 2014 GlaxoSmithKline and Genmab reported that the European Commission (EC) has granted marketing authorization for a new indication for the use of Arzerra (ofatumumab), a human monoclonal antibody against CD20, in combination with chlorambucil or bendamustine for the treatment of patients with chronic lymphocytic leukemia (CLL) who have not received prior therapy and who are not eligible for fludarabine-based therapy (Press release Genmab, JUL 3, 2014, View Source [SID:1234500611]).

"Today’s decision by the European Commission for the first-line use of Arzerra offers a new treatment option for appropriate CLL patients and enables physicians flexibility in their choice of adjunct chemotherapy – chlorambucil or bendamustine," said Dr. Paolo Paoletti, President of Oncology, GSK.

"We are very pleased to receive this decision that Arzerra is approved in the EU in the front-line setting in combination with two different alkylating chemotherapies. This is another important milestone and we look forward to a successful launch under this new indication of the drug in Europe in the coming months. We hope to receive additional approvals in frontline across the globe in the future," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

The EC authorization of the first-line indication for Arzerra (ofatumumab) is based on results from two trials:

A randomized, Phase III open-label, parallel-arm, multicenter, pivotal study (OMB110911, COMPLEMENT 1) evaluating the combination of ofatumumab and chlorambucil (N=221) versus chlorambucil alone (N=226) in CLL patients for whom fludarabine-based treatment is considered inappropriate. In this study, treatment with ofatumumab and chlorambucil demonstrated a statistically significant, 71 per cent improvement in median progression-free survival (PFS) compared to chlorambucil alone (22.4 months versus 13.1 months, respectively) (HR=0.57 [95 per cent CI, 0.45, 0.72] p<0.001).
A single-arm, multicenter, Phase II study (OMB115991) evaluating ofatumumab in combination with bendamustine in 44 patients with previously untreated CLL for whom fludarabine-based treatment was considered inappropriate. Results of this study demonstrated that ofatumumab in combination with bendamustine provided an overall response rate (ORR) of 95 per cent [95 per cent CI, 85, 99] and a complete response rate (CR) of 43 per cent.

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