On January 3, 2019 Takeda Pharmaceutical Company Limited ("Takeda") (TSE: 4502) reported new research collaborations in immuno-oncology (I-O), an area of key strategic focus for the company (Press release, Takeda, JAN 3, 2019, View Source [SID1234532414]). Through these collaborations, Takeda seeks to accelerate the discovery of next-generation cancer immunotherapies, including novel cell therapy approaches that may provide important opportunities for addressing the needs of patients with hard-to-treat cancers.

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"We are excited by the recent momentum in oncology R&D, especially around the curative potential of cell-based therapies through our growing partnership network," said Phil Rowlands, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. "We look forward to continuing to collaborate with some of the leading pioneers in the field to fuel research and discovery with the aim of targeting novel mechanisms of action in the cancer-immunity cycle to help us fulfill our aspiration to cure cancer."

Takeda will collaborate with Memorial Sloan Kettering Cancer Center (MSK) to discover and develop novel chimeric antigen receptor T-cell (CAR-T) products for the treatment of multiple myeloma, acute myeloid leukemia and additional solid tumor indications. The broad, multi-faceted collaboration will be co-led by CAR-T therapy pioneer Michel Sadelain, M.D., Ph.D., Director of the Center for Cell Engineering at MSK and scientific founder of Juno Therapeutics.
Takeda exercised an option under its existing research collaboration with Noile-Immune Biotech Inc. (Noile), which originated in September 2017. Due to the success of the collaboration, Takeda exclusively licensed NIB-102 and NIB-103 for the treatment of various solid tumor indications, and will co-develop these CAR-T cell therapies with Noile utilizing the company’s proprietary "Prime" (proliferation inducing and migration enhancing) CAR-T platform. The company plans to gain regulatory approval for human testing of NIB-102 by the end of this year.
Takeda’s exercised option for an exclusive oncology-targeted Humabody license from Crescendo Biologics will allow Takeda to additionally evaluate these Humabody VHs for the development of novel CAR-T therapeutics. The development will leverage the unique properties of single-domain tumor-targeted binders as an alternative to conventional single-chain variable fragment (scFv)-based approaches.
Takeda’s diversification into next-generation cell therapy builds directly on its three strategic pillars in oncology: hematologic malignancies, lung cancer and immuno-oncology. Through collaboration with external partners and its newly established translational cell therapy engine, Takeda plans to deliver a rich pipeline of early-stage assets in the coming years.

Takeda has established a new internal translational cell therapy engine with bioengineering, chemistry, manufacturing and control (CMC), clinical and translational expertise. The group aims to rapidly translate innovative and differentiated cell therapy concepts in to the clinic under the leadership of Stefan Wildt, Ph.D., Head of Pharmaceutical Sciences and Translational Engine, Cell Therapies.

"There’s an incredible opportunity to combine promising external innovation with the power of a fit-for-purpose translational cell therapy engine to accelerate the development of truly novel cell therapies," said Stefan Wildt. "We have assembled a very talented team with deep and relevant cell therapy development experience who will help us achieve this goal."

On January 3, 2019 Adaptive Biotechnologies reported that Chad Robins, chief executive officer and co-founder, will present at the 37th Annual J.P. Morgan Healthcare Conference on January 8, 2019 at 11:30 am PT (2:30 pm ET) in San Francisco (Press release, Adaptive Biotechnologies, JAN 3, 2019, View Source [SID1234532413]). Robins will also participate in a panel presentation, Reinventing the Disease Continuum Through Precision Health, on January 7, 2019 at 5:00 pm PT (8:00 pm ET) to discuss the impact of immune profiling on the future of immune-driven medicine.

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A live webcast of the presentation and Q&A session can be accessed from the Newsroom under Events on the Adaptive website at adaptivebiotech.com. Following the live presentation, a replay of the webcast will be available on the Company’s website for 90 days.

On January 3, 2019 Exicure, Inc. (OTCQB: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional spherical nucleic acid (SNA) constructs, reported that its CEO, Dr. David Giljohann, will present a corporate update on Tuesday, January 8, 2019 from 2:30 pm to 3:00 pm PT at the Biotech Showcase 2019 (Press release, Exicure, JAN 3, 2019, View Source [SID1234532412]). The presentation will be in the Franciscan A room on the Ballroom level of the Hilton San Francisco Union Square.

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A live audio webcast will be available on the Investors section of Exicure’s website: www.exicuretx.com. The webcast will be archived for approximately 30 days following the event.

On January 3, 2019 UroGen Pharma Ltd. (Nasdaq:URGN), a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, with a focus on uro-oncology, reported the appointment of Elizabeth (Liz) Barrett as President and Chief Executive Officer effective immediately (Press release, UroGen Pharma, JAN 3, 2019, View Source [SID1234532411]). She will also serve on the Company’s Board of Directors. Ms. Barrett, who will be based in New York, replaces Ron Bentsur. Mr. Bentsur will step down from his position at the Company but will continue to serve in an advisory capacity as needed to ensure a smooth transition.

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Ms. Barrett will present at the 37th Annual J.P. Morgan Healthcare Conference on Thursday, January 10, 2019 at 10:30 a.m. Pacific Time. The event will be held in San Francisco at the Westin St. Francis.

Ms. Barrett brings over 30 years of unique experience across oncology, specialty care, surgical franchises, and consumer marketing in multiple geographic regions. Her demonstrated expertise in pharmaceutical development and commercialization of oncology products will be critical as UroGen transforms into a leading commercial-stage entity and delivers potentially paradigm-shifting products to oncology patients in the urological setting.

"Liz has proven to be an exceptional leader with a well-established track record in oncology and the vision and experience to lead UroGen as it prepares for the expected launch of UGN-101, the Company’s first potential product, in 2019," said Arie Belldegrun, M.D., FACS, Chairman of the Board of UroGen. "Through the persistence and dedication of Ron Bentsur and the team, UroGen has become a leader in uro-oncology, and has energized much needed innovation in devastating urologic diseases that are often overlooked. We thank Ron for his commitment to UroGen and are pleased to support him in his future endeavors to focus on innovative early stage life sciences companies."

Ms. Barrett was CEO of Novartis Oncology and a member of the Executive Committee of Novartis. She previously served as Global President of Oncology at Pfizer Inc. At Pfizer, she held numerous leadership positions, including President of Global Innovative Pharma for Europe, President of the Specialty Care Business Unit for North America, and President of United States Oncology. Prior to Pfizer, she was Vice President and General Manager of the Oncology Business Unit at Cephalon Inc. Ms. Barrett also worked at Johnson & Johnson. She started her career at Kraft Foods Group Inc. Ms. Barrett holds a Bachelor of Science from the University of Louisiana and a Master of Business Administration from Saint Joseph’s University.

"I’ve built a career with some of the best companies in the industry and have had the opportunity to be entrepreneurial within each of those positions. This is an opportune time to take that experience and apply it to a smaller biotech company on the cusp of transformation," said Ms. Barrett. "UroGen is well positioned to firmly establish the viability of its RTGel platform as the Company completes its first NDA submission to the FDA and prepares for potential commercialization later this year. I cannot think of a more exciting time to join UroGen and work with its outstanding team as we begin to revolutionize uro-oncology and beyond."

"I’ve had the great honor of leading UroGen through its early stages of development, its initial public offering, completed enrollment of our first pivotal trial, and now, initiation of our first rolling NDA submission," said Ron Bentsur. "I have no doubt that Liz will be a great leader during UroGen’s next stage and will have the experience needed to build on our clinical success and prepare the company for commercialization. I look forward to supporting the Company’s ongoing success."

UroGen recently announced its initiation of the UGN-101 Rolling NDA Submission to the U.S. Food and Drug Administration (FDA). The completed submission is expected in mid-2019, with potential approval anticipated in 2019. The Company plans to present the topline data in January 2019.

A live audio webcast of the J.P. Morgan presentation will be available on the Investors section of UroGen’s website www.urogen.com. A replay of the webcast will be available on the website for approximately 30 days.

On January 3, 2019 Aadi Bioscience, Inc. (Aadi), a clinical stage biopharmaceutical company focused on treating diseases driven by mTOR activation, reported that its drug TARZIFYX (sirolimus albumin-bound nanoparticles for injectable suspension, ABI-009) has received Breakthrough Therapy Designation status from the FDA for the indication of Advanced (metastatic or locally advanced) Malignant PEComa (perivascular epithelioid cell tumor) (Press release, Aadi, JAN 3, 2019, View Source [SID1234532410]). The designation was granted based on data provided to the FDA from Aadi’s ongoing phase 2 registration trial (NCT02494570) in Advanced Malignant PEComa. In October 2018, Aadi also received Fast Track Designation from the FDA for TARZIFYX for the same indication. Aadi received orphan drug designation for TARZIFYX for the treatment of PEComa in December 2017.

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Aadi’s Phase 2 registration trial for Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT), a rare form of sarcoma, has completed enrollment. Aadi previously received agreement from the FDA that this open label study in 30 efficacy evaluable patients with a primary endpoint of overall response rate could support the submission of an NDA for approval to treat this rare disease.

"We are extremely pleased with the progress of the AMPECT study and recognition of the high unmet need in this patient population via the Breakthrough Designation. Our small, but dedicated team is gearing up for NDA filing in 2019," said Neil Desai, Ph.D., Founder and Chief Executive Officer of Aadi. "There has been no prior clinical trial in this rare disease and standard chemotherapies used in sarcoma treatment have very limited activity in this patient population."

mTOR inhibition is relevant in a number of disease states and has not been fully exploited in oncology, cardiovascular disease, CNS diseases, mitochondrial disease, diseases of ageing, etc. Due to the unique properties of Aadi’s drug including effective CNS penetration, it is being tested in nine ongoing clinical studies across these indications with encouraging results even outside of oncology in early studies in pulmonary hypertension and surgically refractory epilepsy. Aadi’s ongoing and planned clinical trials include Oncology (pediatric tumors, advanced sarcoma, first-line treatment of advanced colorectal cancer, newly diagnosed and recurrent glioblastoma, advanced neuroendocrine tumors), Cardiovascular indications (pulmonary arterial hypertension), CNS indications (surgically refractory epilepsy) and Mitochondrial disease (Leigh Syndrome).