On July 13, 2017 Oxford BioMedica plc ("Oxford BioMedica" or "the Group") (LSE:OXB), a leading gene and cell therapy group, reported that the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee voted unanimously 10 to 0 in favour of approval of Novartis investigational therapy CTL019 (tisagenlecleucel) for the treatment of relapsed and refractory (r/r) paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL) (Press release, Oxford BioMedica, JUL 13, 2017, View Source [SID1234519799]).

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Novartis announced in March that the FDA accepted its BLA filing and granted priority review for CTL019 in paediatric and young adult patients with B-cell ALL. The BLA review is ongoing and is under FDA priority review.

Oxford BioMedica is the sole manufacturer of the lentiviral vector that encodes CTL019. As announced in October 2014, Oxford BioMedica will also receive undisclosed royalties on potential future sales of Novartis CAR-T products. Oxford BioMedica recently signed an agreement with Novartis for the commercial and clinical supply of lentiviral vectors used to generate CTL019 and other undisclosed CAR-T products, for which Oxford BioMedica could potentially receive in excess of $100m from Novartis over the next three years.

John Dawson, Chief Executive Officer of Oxford BioMedica, commented: "The positive vote by 10 to 0 provides further support for CTL019, a novel immunocellular therapy, and we are proud to be a part of this important development. We continue to work closely with Novartis in delivering the lentiviral vector that encodes CTL019, a product the company described earlier this year as having "blockbuster" potential."

On July 12, 2017 Novartis reported that the US Food and Drug Administration (FDA) Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of CTL019 (tisagenlecleucel), an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) (Press release, Novartis, JUL 12, 2017, View Source [SID1234519794]).

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"The panel’s unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need," said Bruno Strigini, CEO, Novartis Oncology. "We’re very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options. We look forward to working with the FDA as they complete their review."

Acute lymphoblastic leukemia comprises approximately 25% of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US[1]. Effective treatment options for patients with r/r ALL are limited. In pediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30%[2],[3],[4].

The ODAC recommendation is based on review of the CTL019 r/r B-cell ALL development program, which includes the Novartis-led ELIANA study (NCT02435849), the first pediatric global CAR-T cell therapy registration trial. Findings from a US multicenter trial and a single site trial examining the safety and efficacy of CTL019 among pediatric and young adult patients with r/r B-cell ALL also supported the recommendation and the Biologics License Application (BLA)[5].

CTL019 was first developed by the University of Pennsylvania (Penn) and uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular responses as well as persistence of CTL019 after it is infused into the patient, which may be associated with long-lasting remissions in patients. In 2012, Novartis and Penn entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Children’s Hospital of Philadelphia (CHOP) was the first institution to investigate CTL019 in the treatment of pediatric patients and led the single site trial.

"It is encouraging to see the FDA panel’s recommendation and continued momentum behind this innovative therapy, which has potential to help young patients with relapsed/refractory B-cell ALL," said the Penn team’s leader, Carl June, MD, the Richard W. Vague Professor of Immunotherapy, director of the Center for Cellular Immunotherapies in Penn’s Perelman School of Medicine and director of the Parker Institute for Cancer Immunotherapy at Penn. "We look forward to continuing to work with Novartis to help make a lasting impact on the way this disease is treated."

"We know firsthand from treating children and young adults with relapsed/refractory B-cell ALL that they desperately need innovative medicines that provide a new approach to managing this aggressive disease," said Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics at the Perelman School of Medicine at Penn, Director of the Cancer Immunotherapy Frontier Program and Chief of the Section of Cellular Therapy and Transplant at CHOP. "Today’s vote in favor of CTL019 is a positive step and we appreciate Novartis’ commitment to pediatric patients."

Earlier this year, Novartis submitted a BLA for CTL019 to the FDA, marking the first submission by Novartis for a CAR-T cell therapy. CTL019 previously received FDA Breakthrough Therapy designation and is under Priority Review by the FDA. The FDA will consider the vote as it reviews the BLA, although it is not obligated to follow the recommendation. Novartis continues to invest in the necessary infrastructure for the potential commercialization of CTL019, including manufacturing and the establishment of a network of certified treatment centers.

Novartis plans additional filings for CTL019 in the US and EU later this year, including applications with the FDA and European Medicines Agency (EMA) for the treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL).

About CAR-T and CTL019
CAR-T is different from typical small molecule or biologic therapies because it is manufactured for each individual patient using their own cells. During the treatment process, T cells are drawn from a patient’s blood and reprogrammed in the manufacturing facility to create T cells that are genetically coded to express a chimeric antigen receptor to recognize and fight cancer cells and other B-cells expressing a specific antigen.

ELIANA (NCT02435849) is the first pediatric global CAR-T cell therapy registration trial, with study enrollment having occurred across 25 centers in the US, Canada, EU, Australia and Japan.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world.

On July 11, 2017 Daiichi Sankyo Co., Limited (hereafter, Daiichi Sankyo), Max Planck Innovation GmbH and the Lead Discovery Center GmbH reported that they have signed an agreement providing Daiichi Sankyo with the option to receive the exclusive rights to a new lead compound for the treatment of cancer to be discovered and developed at the Lead Discovery Center.

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This new partnership builds on biology insights in the field of transcriptional regulation from the work of Prof. Matthias Geyer at the Max Planck Institute of Molecular Physiology in Dortmund and the Research Center caesar (Center of Advanced European Studies and Research) in Bonn, Germany. Combined with the Lead Discovery Center’s strong drug discovery expertise in the design of highly selective kinase inhibitors, Daiichi Sankyo, Max Planck researchers and the Lead Discovery Center will now closely cooperate to further optimize these novel compounds that target cancer cell transcription and proliferation.

Daiichi Sankyo together with the Max Planck Society, supported by Max Planck Foundation, will jointly fund the respective drug discovery efforts at the Lead Discovery Center. Once the project has achieved proof-of-concept in relevant in vivo models, Daiichi Sankyo has the exclusive rights to license the program at pre-defined terms for subsequent preclinical and clinical development. The agreement includes an upfront payment as well as development and sales milestones plus royalties. The licensing revenues will be shared between Max Planck Society, the Lead Discovery Center and all contributing researchers and institutions.

"The Lead Discovery Center is our prime partner for innovative drug discovery projects and developing novel compounds with a high therapeutic potential from the Max Planck Institutes. This agreement with Daiichi Sankyo, a recognized leader in the development and supply of innovative pharmaceutical products, again shows the high quality of research projects driven at the Max Planck laboratories. Moreover, the agreement is a great opportunity to advance the research findings into pharmaceutical development, providing potential new treatment options for patients with cancer," according to Dr. Matthias Stein-Gerlach, patent and licensing manager at Max Planck Innovation, Max Planck Society´s technology transfer organization.

"This project collaboration and option agreement is building on the excellent experiences that Daiichi Sankyo and the Lead Discovery Center previously made from a discovery alliance that started in 2014, as well as close ties and many interactions between Daiichi Sankyo and the Max Planck Society, such as the collaboration with the Axel Ullrich lab. Max Planck Innovation has been instrumental to close this partnership," adds Dr. Bert Klebl, Managing Director and CSO at the Lead Discovery Center.

"It is a great pleasure for us to start this research collaboration with Max Planck Innovation and the Lead Discovery Center to further generate innovation for our cancer drug discovery efforts," said Antoine Yver, MD, MSc, Executive Vice President and Global Head of Oncology Research and Development, Daiichi Sankyo. "We are excited about the integration of Max Planck Society’s high quality science and the Lead Discovery Center’s expertise in lead discovery into Daiichi Sankyo’s drug research and development platform."

Daiichi Sankyo, the Lead Discovery Center and the Max Planck Society aim to further expand their collaboration into additional programs in the future.

About Max Planck Innovation
Max Planck Innovation is responsible for the technology transfer of the Max Planck Society and, as such, serves as a link between industry and basic research. With its interdisciplinary team it advises and supports scientists in evaluating their inventions, filing patents, and founding companies. Max Planck Innovation offers the industry unique access to the innovations of the Max Planck Institutes, and therefore performs an important task: the transfer of basic research results into products, which contributes to economic and social progress. Further information at: www.max-planck-innovation.de

About the Lead Discovery Center
The Lead Discovery Center was established in 2008 by the technology transfer organization Max Planck Innovation, as a novel approach to capitalize on the potential of excellent basic research for the discovery of new therapies for diseases with high medical need. The Lead Discovery Center takes on promising early-stage projects from academia and transforms them into innovative pharmaceutical leads that reach initial proof-of-concept in animals. In close collaboration with high-profile partners from academia and industry, the Lead Discovery Center is building a strong and growing portfolio of small molecule leads with exceptional medical and commercial potential.

The Lead Discovery Center sustains a preferred partnership with the Max Planck Society and has formed alliances with AstraZeneca, Bayer, Boehringer Ingelheim, Merck KGaA, Daiichi Sankyo, Qurient, Johnson & Johnson Innovation, Roche and Sotio as well as leading translational drug discovery centers around the globe. Further information at: www.lead-discovery.de

About Daiichi Sankyo Cancer Enterprise
The vision of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking in order to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by our Antibody Drug Conjugate (ADC) and Acute Myeloid Leukemia (AML) Franchises, our cancer pipeline includes more than 20 small molecules, monoclonal antibodies and ADCs stemming from our powerful research engines: our two laboratories for biologic/immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in development include: quizartinib, an oral FLT3 inhibitor, for newly-diagnosed and relapsed/refractory AML with FLT3-ITD mutations; DS-8201, an ADC for HER2-expressing breast and gastric cancer, and other HER2-expressing solid tumors; and pexidartinib, an oral CSF-1R inhibitor, for tenosynovial giant cell tumor (TGCT), which is also being explored in a range of solid tumors in combination with the anti-PD1 immunotherapy pembrolizumab. For more information, please visit: www.DSCancerEnterprise.com

Exicure has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Exicure, 2017, JUL 11, 2017, View Source [SID1234521827]).

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On July 11, 2017 Sanofi reported it will acquire Protein Sciences, a privately held vaccines biotechnology company based in Meriden, Connecticut in the United States. Under the terms of the agreement, Sanofi will make an upfront payment of $650 million and pay up to $100 million upon achievement of certain milestones.

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Protein Sciences received approval from the US Food and Drug Administration (FDA) in October 2016 for their Flublok Quadrivalent Influenza Vaccine (QIV). Flublok is the only recombinant protein-based influenza vaccine approved by the FDA.

"The acquisition of Protein Sciences will allow us to broaden our flu portfolio with the addition of a non-egg based vaccine," said David Loew, Sanofi Executive Vice President and Head of Sanofi Pasteur, Sanofi’s vaccines division.

"Protein Sciences was actively looking for an opportunity to grow its business, particularly in the US," said Manon M.J. Cox, President and Chief Executive Officer, Protein Sciences. "As part of Sanofi Pasteur, we expect our Flublok influenza vaccine to benefit from Sanofi Pasteur’s expertise in the field of influenza vaccines."

The acquisition, which has been unanimously approved by the board of directors of Protein Sciences and a majority of Protein Sciences shareholders, is expected to close in the third quarter of 2017, subject to customary regulatory approvals.

(Press release, Sanofi, JUL 11, 2017, View Source [SID1234670341])