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Adaptimmune confirms GSK Nomination of Second Adaptimmune Target under Strategic Multi-Target Collaboration

On January 9, 2017 Adaptimmune Therapeutics plc (Nasdaq:ADAP), a leader in T-cell therapy to treat cancer, reported that GlaxoSmithKline plc (NYSE:GSK) (LSE:GSK) has nominated a second target, PRAME (preferentially expressed antigen in melanoma), under the strategic collaboration and licensing agreement between the companies (Press release, Adaptimmune, JAN 9, 2017, View Source [SID1234517335]). Adaptimmune will be responsible for PRAME preclinical TCR development and delivery of the IND package to GSK. The nomination of a second target meets a milestone set forth in the agreement.

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Adaptimmune and GSK initially announced their strategic collaboration and licensing agreement in June 2014 for up to five programs, the first being the NY-ESO SPEAR T-cell therapy program, and the agreement was subsequently expanded in February 2016 to accelerate development of Adaptimmune’s NY-ESO SPEAR T-cell therapy toward registration trials in synovial sarcoma. Following the nomination of PRAME as a second target, Adaptimmune will take the program through preclinical testing to IND. GSK retains the right to nominate up to three additional targets, if GSK exercises its option on NY-ESO; however, this excludes targets on which work is already under way, including Adaptimmune’s proprietary MAGE-A10, MAGE-A4 and AFP programs.

"The nomination of this next target marks an important step forward for the collaboration," commented Helen Tayton-Martin, Adaptimmune’s Chief Operating Officer and responsible for the alliance. "The early clinical results we have seen in synovial sarcoma with our SPEAR T-cell therapy targeting NY-ESO-1, the first target nominated by GSK, have been promising thus far, and we are accelerating that program toward registration studies. The nomination of PRAME as GSK’s second target is further validation of our technology, and our goal is to deliver this IND package as expeditiously as possible."

Under the terms of the agreement, the potential development milestones Adaptimmune is eligible to receive solely in relation to the PRAME program could amount to approximately $300 million, if GSK exercises its option and successfully develops this target in more than one indication and more than one Human Leukocyte Antigen (HLA) type. Adaptimmune would also receive tiered sales milestones and mid-single to low double-digit royalties on worldwide net sales.

TG Therapeutics Announces Orphan Drug Designation for the Combination of TG-1101 and TGR-1202 for Treatment of Chronic Lymphocytic Leukemia

On January 9, 2017 TG Therapeutics, Inc. (NASDAQ:TGTX) reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation covering the combination of TG-1101 (ublituximab), the Company’s novel, glycoengineered anti-CD20 monoclonal antibody, and TGR-1202 the Company’s oral, next generation PI3K delta inhibitor, for the treatment of patients with chronic lymphocytic leukemia (CLL) (Press release, TG Therapeutics, JAN 9, 2017, View Source [SID1234517334]). The combination of TG-1101 and TGR-1202 is currently being evaluated in the UNITY-CLL Phase 3 Trial for patients with both frontline and previously treated CLL as well as the UNITY-DLBCL Phase 2b Trial for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

"Receiving orphan drug designation for our proprietary combination of TG-1101 and TGR-1202 provides another layer of exclusivity protection on top of the composition of matter patents which were issued for TG-1101 and TGR-1202 last year and patents on the combination that have been filed and are pending approval," stated Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics. "Additionally, with enrollment into our UNITY-CLL Phase 3 trial currently exceeding our expectations, we expect to be able to commence a regulatory filing for the combination in 2018 and having orphan drug designation will provide certain cost saving advantages for us during the regulatory approval process."

ABOUT ORPHAN DRUG DESIGNATION

Orphan drug designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity.

ABOUT CHRONIC LYMPHOCYTIC LEUKEMIA

Chronic lymphocytic leukemia (CLL) is a type of cancer in which the bone marrow makes too many lymphocytes (a type of white blood cell). CLL usually gets worse slowly and is one of the most common types of leukemia in adults. It often occurs during or after middle age. It is estimated that there are approximately 20,000 new cases of CLL diagnosed each year in the United States.

Update on Dispute between Exelixis and Genentech, a Member of the Roche Group

On January 9, 2017 Exelixis, Inc. (Nasdaq:EXEL) reported that Genentech, Inc. has withdrawn its counterclaim against Exelixis in the ongoing JAMS arbitration concerning alleged breaches of the parties’ collaboration agreement (Press release, Exelixis, JAN 9, 2017, View Source [SID1234517332]). Genentech had asserted a counterclaim for breach of contract, which sought monetary damages and interest related to cost allocations under the collaboration agreement. When notifying the arbitral panel, and Exelixis, of this unilateral action, Genentech further stated that it is changing the manner in which it allocates promotional expenses of the COTELLIC (cobimetinib) plus Zelboraf (vemurafenib) combination therapy.

As a result of Genentech’s decision to change its cost allocation approach, Exelixis is relieved of $18.7 million of disputed costs previously charged by Genentech. Exelixis has invoiced Genentech an additional $7.1 million with interest for expenses that Exelixis paid previously.

Genentech’s revised allocation applies retrospectively and prospectively and will substantially reduce Exelixis’ exposure to costs associated with promotion of the COTELLIC + Zelboraf combination in the United States. Exelixis and Genentech have shared promotional costs since commercial activities were initiated in early 2013. As detailed in previous regulatory filings, Exelixis charged its Profit and Loss Statement approximately $38 million for promotional costs through the third quarter of 2016. With the new approach that Genentech has adopted unilaterally, Exelixis’ liability for promotional costs will be reduced to approximately $15 million for the same period.

Other significant issues remain in dispute between the parties. Genentech’s action does not address the claims in Exelixis’ Demand for Arbitration related to Genentech’s clinical development, pricing and promotional costs for COTELLIC in the United States, nor does it fully resolve claims over revenue allocation. And, Genentech has not confirmed how it intends to allocate promotional costs incurred with respect to the collaboration’s promotion of other combination therapies that include cobimetinib for other indications that are in development and may be approved. Exelixis will continue to press its position before the arbitral panel to obtain a just resolution of these claims and the clarity it requires.

About the Dispute

On June 3, 2016, Exelixis filed a Demand for Arbitration before JAMS in San Francisco, California asserting claims against Genentech related to its clinical development, pricing and promotion of COTELLIC, and cost and revenue allocations in connection with COTELLIC’s promotion in the United States. The arbitration demand asserts that Genentech has breached the parties’ contract by, amongst other breaches, failing to meet its diligence and good faith obligations. The demand seeks various forms of declaratory, monetary, and equitable relief, including without limitation that the cost and revenue allocations for COTELLIC be shared equitably consistent with the collaboration agreement’s terms, along with attorneys’ fees and costs of the arbitration. Genentech had asserted a counterclaim for breach of contract, which sought monetary damages and interest related to the cost allocations under the collaboration agreement.

AbbVie Announces Four New Global Research Collaborations Focused on Advancing Next-Generation Science

On January 9, 2017 AbbVie (NYSE: ABBV), a global biopharmaceutical company, reported four new collaborations and investments with leading healthcare innovators to advance early-stage research in key therapeutic areas such as oncology and immunology (Press release, AbbVie, JAN 9, 2017, View Source [SID1234517331]). AbbVie is committed to investing in and developing transformational science and technologies to advance the next-generation of therapies across its robust pipeline in key therapeutic areas. With more than 50 compounds in clinical development, AbbVie’s pipeline spans significant areas of medical need including oncology, immunology, neuroscience and virology.

"We can develop tomorrow’s most important therapies by investing in today’s leading technologies and scientific achievements," said Michael Severino, M.D., executive vice president, research and development and chief scientific officer, AbbVie. "Combining the strengths of AbbVie’s discovery and development expertise and novel research from external partners will accelerate the pace of innovation into new medicines for the next-generation of medical treatment."

Oncology

AbbVie and Pure MHC, a privately-held target discovery company, will embark on a research and license agreement to discover and validate peptide targets for use with T-cell receptor therapeutics in several types of cancers.
T-cells play a key role in regulating immune responses to tumors. Receptors on the surface of T-cells recognize tumor antigens in the form of small peptides and provide a selective pathway for targeted cancer therapies. Pure MHC has developed technology to identify novel, tumor-associated peptides based on innovation licensed from the University of Oklahoma.

The collaboration between AbbVie and Pure MHC will seek to identify a library of peptide targets for further research across multiple tumor types and advance AbbVie’s ongoing development of next-generation immuno-oncology therapies.

AbbVie has entered into an exclusive license with Dong-A-ST, a leading specialty healthcare company in South Korea, for MerTK inhibitors in pre-clinical development for use in conjunction with immuno-oncology therapies.
MerTK is a protein that is believed to contribute to the promotion of immunosuppressive tumor microenvironment. Inhibition of this activity may help promote an inflammatory state, alerting the immune system to attack tumors and augmenting the efficacy of targeted cancer therapies such as checkpoint inhibitors (anti-PD1/PD-L1) and pro-apoptotic agents. The collaboration will explore the combination of MerTK inhibitors in conjunction with AbbVie’s portfolio of anti-cancer agents across multiple types of solid tumors.

The addition of this mechanism will advance AbbVie’s existing research into immuno-oncology therapies and complement its oncology pipeline under development for nearly 20 cancers and tumor types.

Immunology

AbbVie and Zebra Biologics, Inc., a discovery stage biotechnology company, have entered into a partnership to discover agonist antibody therapeutics for inflammatory diseases. Zebra will utilize its novel and patented function-based antibody discovery platform to generate antibodies that activate biological pathways associated with targets designated by AbbVie. Zebra and AbbVie will collaborate closely on the identification and pre-clinical validation of emerging candidates. The targets were not disclosed.
Zebra will lead the discovery of candidate agonist antibodies for designated targets and will collaborate with AbbVie in pre-clinical validation of select clinical candidates. Upon advancement of clinical candidates, AbbVie would be responsible for clinical development, manufacturing, regulatory approval and world-wide commercialization.

Genomics

AbbVie and Genomics Medicine Ireland (GMI), a life sciences startup company, have partnered to conduct population genomics research in Ireland. The alliance will sequence the genomes of 45,000 volunteers across Ireland. The data to be included will originate from people with several types of immune-mediated diseases, neurological disorders and cancer, as well as people unaffected by these diseases. By incorporating the genotypic and phenotypic data across a wide sample population, the partnership aims to better understand human biology and disease etiology to discover new therapeutic targets and identify biomarkers.
AbbVie will utilize the research to select targets for drug development, as well as potential development of companion diagnostics for selected conditions.

GMI is backed by investors ARCH Venture Partners, Polaris Partners, GV (formerly Google Ventures) and the Ireland Strategic Investment Fund.

Kite Pharma Establishes a Strategic Partnership With Daiichi Sankyo to Develop and Commercialize Axicabtagene Ciloleucel (KTE-C19) in Japan

On January 9, 2016 Kite Pharma (Nasdaq:KITE) reported that it has entered into a strategic partnership with Daiichi Sankyo Co., Ltd. (Daiichi Sankyo) for axicabtagene ciloleucel (approved USAN for KTE-C19) in Japan (Press release, Kite Pharma, JAN 9, 2017, View Source [SID1234517325]). Axicabtagene ciloleucel, Kite’s lead product candidate, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells.

Under the terms of the partnership agreement, Daiichi Sankyo will be responsible for development and commercialization of axicabtagene ciloleucel in Japan. Daiichi Sankyo will make an upfront payment to Kite of $50 million and Kite will be eligible to receive future payments totaling up to $200 million for development and commercial milestones. Kite is also entitled to receive sales royalties in the low to mid double digit range.

"We are thrilled to partner with Daiichi Sankyo, a market leader in Japan who shares our vision for engineered T-cell therapy and has strong development capabilities in oncology," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "We have a strategic roadmap to commercialize axicabtagene ciloleucel globally while focusing Kite’s development and commercialization efforts in the United States and Europe. Daiichi Sankyo’s commitment to bring autologous T-cell therapy to patients in Japan will complement our strategy and demonstrates the significant value in our pipeline, as well as the commercial potential for autologous T-cell therapy globally."

As part of the transaction, Kite will provide certain technical transfer services to Daiichi Sankyo. In addition, Daiichi Sankyo has a certain period of time to license additional Kite product candidates for Japan including KITE-718, Kite’s T cell receptor product candidate targeting MAGE-A3/A6 and certain other product candidates that proceed to a U.S. investigational new drug application filing over the next three years. Upfront and milestone payments for each additional product candidate could equal up to $200 million plus low to mid double digit sales royalties. Kite retains all development and commercialization rights outside of Japan.

"We are very enthusiastic about this partnership with Kite which has the most advanced technology platform in this area and the potential for cell-based therapy to change the way in which we treat cancer in Japan," said Koichi Akahane, Japan Head of Oncology R&D, Daiichi Sankyo. "We believe we can leverage the pioneering research conducted by Kite to potentially accelerate development and commercial availability of axicabtagene ciloleucel in Japan for those patients suffering from B-cell malignancies."

Japan, the third-largest pharmaceutical market in the world, has implemented an accelerated approval pathway for regenerative medicine and cell therapy products under the Pharmaceuticals and Medical Devices Act (PMD Act) in November 2014. There are approximately 9,300 new cases of aggressive NHL and 1,400 new cases of acute lymphoblastic leukemia (ALL) diagnosed per year in Japan, with similar treatment rates and prescribing patterns as the United States and Europe.1

Kite announced in December 2016 that it has initiated the rolling submission to the U.S. Food and Drug Administration (FDA) of the Biologics License Application (BLA) for KTE-C19 as a treatment for patients with refractory aggressive non-Hodgkin lymphoma (NHL).

About axicabtagene ciloleucel

Kite Pharma’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.