On January 13, 2020 TIO Bioventures, an emerging life science venture creation fund with a mission to build companies with innovative anti-cancer therapies, reported the launch of Treadwell Therapeutics, a clinical-stage oncology company exploiting cancer cells’ vulnerabilities to develop first-in-class and best-in-class small molecules to address unmet needs in patients with cancer (Press release, Tio Bioventures, JAN 13, 2020, View Source [SID1234553221]). Treadwell, one of TIO Bioventures family of companies launched from its discovery engine, TIO Discovery, was established with an initial $27 million in seed financing from TIO Bioventures to develop a robust pipeline of novel, first-in-class, small molecule drug candidates.

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"At Treadwell, we are developing small molecules designed to leverage world-class, innovative science in cancer biology and immune-oncology with pan-cancer potential, including hematological malignancies from the TIO Discovery development engine," said Dr. Michael Tusche, Treadwell Co-Chief Executive Officer and Partner at TIO Bioventures.

The origins of Treadwell lie in cutting-edge research incubated at the Campbell Family Institute for Breast Cancer Research (CFIBCR) within the University Health Network (UHN), Canada’s largest research hospital. Based on this research, Treadwell scientists have designed and advanced from discovery to Phase 2 clinical development, a first-in-class PLK4 kinase inhibitor, CFI-400945 and a potentially best-in-class TTK inhibitor, CFI-402257. These candidates have demonstrated encouraging safety and efficacy profiles in multiple investigator-sponsored clinical trials. In addition, Treadwell is poised to bring a third molecule into the clinic in early 2020. This agent, CFI-402411, an oral immunomodulatory kinase inhibitor with activity toward HPK1, an untapped immune-oncology target. Preclinical studies have demonstrated the promise of CFI-402411 as a potential monotherapy and in combination with existing checkpoint inhibitors across both solid and hematological cancers. Treadwell’s product candidates are wholly-owned and have been developed in TIO Discovery, a unique R&D engine wholly owned by TIO.

Leadership

The Company announced the appointment of Rachel W. Humphrey, M.D., as Head of R&D at TIO Discovery as well as Chief Medical Officer and Board Member at Treadwell. Dr. Humphrey joins TIO Bioventures and Treadwell with over two decades of experience as a senior clinical leader in oncology drug development for both large pharmaceutical and emerging biotechnology companies. Her experience includes overall leadership of the clinical development of Yervoy (ipilmumab), the first FDA approved checkpoint inhibitor, at Bristol-Myers Squibb, the development of Imfinzi (durvalumab) at Astra Zeneca and the development of Nexavar (sorafenib) at Bayer, as primary driver of the vision, science and direction during critical periods in the development for these transformational medicines.

"TIO Bioventures novel approach to found, launch, and support companies such as Treadwell speaks to this powerful and successful relationship of world-class scientists with deeply experienced management executives, and I am delighted to join Treadwell at such a pivotal time in the Company’s growth," said Dr. Humphrey. "I look forward to working with the team to further drive the development of these candidates with encouraging anti-cancer activity and exciting novel targets."

She will join Treadwell Therapeutics’ existing leadership team composed of world-class experts in immunology and oncology drug development, including Dr. Tak Mak, co-founder and managing partner of TIO Bioventures, who has led the development of multiple successful biotechnology companies.

On January 13, 2020 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) reported that it has initiated a Phase II study in China of surufatinib in combination with Tuoyi (toripalimab) in patients with advanced solid tumors (Press release, Hutchison China MediTech, JAN 13, 2020, https://www.chi-med.com/chi-med-initiates-a-phase-ii-trial-of-surufatinib-in-combination-with-tuoyi-in-patients-with-advanced-solid-tumors/ [SID1234553057]). This follows the recent completion of the Phase I dose finding study and successful establishment of the Phase II combination dosing regimen for surufatinib and Tuoyi.

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This China Phase II clinical study is part of a global collaboration with Shanghai Junshi Biosciences Co. Ltd. ("Junshi"), to evaluate surufatinib, Chi-Med’s oral angio-immuno kinase inhibitor, in combination with Tuoyi, Junshi’s anti-programmed cell death protein 1 ("PD-1") monoclonal antibody which was approved by China’s National Medical Products Administration ("NMPA") in December 2018. The study is designed to test the potential synergistic anti-tumor effects of the combination’s ability to simultaneously target multiple cell types and signaling pathways in the tumor microenvironment.

The Phase II study plans to explore multiple solid tumor patient populations. The primary outcome measures are objective response rate (ORR) and safety. The secondary outcomes include duration of response (DoR), progression-free survival ("PFS"), disease control rate (DCR) and overall survival ("OS"). The lead principal investigator of the study is Professor Lin Shen, Vice President of Peking University Hospital and Cancer Institute. Additional details may be found at clinicaltrials.gov, using identifier NCT04169672.

Christian Hogg, Chief Executive Officer of Chi-Med, said, "We are excited to move into Phase II development on the surufatinib toripalimab combination and look forward to identifying patient groups that could benefit from this innovative treatment regimen."

In December 2019, surufatinib was granted Priority Review status by the Center for Drug Evaluation (CDE) of China’s NMPA for its New Drug Application ("NDA") for the treatment of patients with advanced non-pancreatic neuroendocrine tumors ("NET").

In November 2019, the NDA for surufatinib for the treatment of non-pancreatic NET was accepted for review by the NMPA, and the U.S. Food and Drug Administration granted Orphan Drug designation to surufatinib for the treatment of pancreatic NET.

About Surufatinib
Surufatinib (previously known as HMPL-012 or sulfatinib) is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor ("VEGFR") and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies. Surufatinib is in several late-stage and proof-of-concept clinical trials in China and proof-of-concept clinical trials in the U.S.

According to Frost & Sullivan, the market for anti-angiogenesis VEGF/VEGFR inhibitors in China has grown from US$500 million in 2015 to over US$1.5 billion in 2019 and is expected to reach US$5 billion by 2026.

Chi-Med currently retains all rights to surufatinib worldwide.

Non-Pancreatic NET in China: In 2015, we initiated the SANET-ep study, a Phase III study of surufatinib in advanced neuroendocrine tumors – extra-pancreatic patients in China for whom there is no effective therapy. In June 2019, a 198-patient interim analysis was conducted, leading the independent data monitoring committee to determine that the study met the pre-defined primary endpoint of progression-free survival ("PFS") and should be stopped early. The positive results were highlighted in an oral presentation at the 2019 European Society for Medical Oncology Congress in September 2019. In November 2019, the NDA was accepted for review by the NMPA (clinicaltrials.gov identifier: NCT02588170) and subsequently granted Priority Review status.

Pancreatic NET in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pancreatic NET in China. The primary endpoint is PFS. We expect an interim analysis in the first half of 2020 and enrollment to complete in 2020 (clinicaltrials.gov identifier: NCT02589821).

NET in the U.S. and Europe: We are planning a U.S. registration study in NET patients based on the encouraging data from the Phase II and Phase III studies of surufatinib in NET in China (clinicaltrials.gov identifier: NCT02267967), and the ongoing Phase II study in the U.S. (clinicaltrials.gov identifier: NCT02549937).

Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier NCT03873532).

Immunotherapy combinations: In November 2018 and September 2019, we entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies. This included global collaborations to evaluate the combination of surufatinib with Tuoyi, approved in China by Junshi, and with Tyvyt, approved in China by Innovent Biologics, Inc.

On January 13, 2020 AbbVie (NYSE: ABBV), a research-based global biopharmaceutical company, reported its fourth-quarter and full-year 2019 financial results on Friday, February 7, 2020 before the market opens (Press release, AbbVie, JAN 13, 2020, View Source [SID1234553075]). AbbVie will host a live webcast of the earnings conference call at 8 a.m. Central time (9 a.m. Eastern). It will be accessible through AbbVie’s Investor Relations website investors.abbvie.com. An archived edition of the session will be available later that day.

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On January 13, 2020 Genocea Biosciences presented the corporate presentation (Presentation, Genocea Biosciences, JAN 13, 2020, View Source [SID1234553092]).

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On January 13, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported its expected milestones for 2020-2021, which highlight the company’s progress advancing its clinical development candidates: omidubicel, an advanced cell therapy in Phase 3 clinical development as a potential life-saving treatment option for patients in need of bone marrow transplant, and GDA-201, an investigational, natural killer (NK) cell-based cancer immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma (NHL) and multiple myeloma (Press release, Gamida Cell, JAN 13, 2020, View Source [SID1234553108]).

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"This is an incredibly important year for Gamida Cell. With patient enrollment completed, we are expecting topline data from the Phase 3 study of omidubicel in the first half of this year. Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and has the potential to be the first FDA-approved bone marrow transplant graft. Positive data from our study would represent an important step toward bringing potentially curative medicines to patients," stated Julian Adams, Ph.D., chief executive officer at Gamida Cell. "We are also very excited by the progress of GDA-201, an investigational therapy in a class that we believe has the potential to be the next generation of cell therapies to dramatically improve the treatment of blood cancers. GDA-201 has shown promise for the treatment of non-Hodgkin lymphoma, including an aggressive form of lymphoma called diffuse large B cell lymphoma, and we anticipate announcing additional data in the first half of 2020."

Today Gamida Cell also announced the appointment of Jas Uppal, Ph.D. to the newly created role of chief regulatory and quality officer. Dr. Uppal brings more than 25 years of global experience in the pharmaceutical industry, including expertise in hematology, immunology and neurology. During her career, she has played key roles in building regulatory organizations and leading multiple successful product launches.

"We are delighted to welcome Jas to Gamida Cell. Her strategic, global experience in building teams and securing multiple product approvals for novel biologics will be invaluable as we move omidubicel and GDA-201 through critical regulatory milestones," stated Dr. Adams.

Dr. Uppal most recently served as vice president, global head of regulatory affairs of oncology, endocrinology and rare diseases at Ipsen Biopharmaceuticals, where she held worldwide responsibility for Ipsen’s oncology, endocrinology and rare diseases portfolio. In this role, she led all areas of product development and managed a team of regulatory professionals. Prior to Ipsen, she served as vice president, global head of regulatory affairs at Karyopharm Therapeutics, where she was responsible for developing a global regulatory strategy and approach for multiple Phase 2 and Phase 3 programs that were being developed to treat hematological malignancies and solid tumors. Earlier in her career, Dr. Uppal held several regulatory-related positions over the course of 12 years at Biogen Idec (now Biogen) that culminated in her role as director of global emerging markets and head of development sciences. Dr. Uppal has participated in over 30 new drug approvals worldwide and has more than 30 publications in peer reviewed journals. She holds a Ph.D. in biochemistry from Kings College, University of London.

Expected 2020-2021 Milestones

Gamida Cell targets achieving the following milestones during 2020-2021:

Omidubicel

Report topline data from the Phase 3 study in the first half of 2020
Present data from the Phase 3 study at a medical meeting in the second half of 2020
Submit the biologics license application to the FDA in the second half of 2020, assuming positive data
Report additional data from the Phase 1/2 study in patients with severe aplastic anemia in the second half of 2020
Launch omidubicel in 2021, contingent upon FDA approval
GDA-201

Present additional data from the Phase 1 study in the first half of 2020
Submit company-sponsored investigational new drug application to FDA in the second half of 2020
Initiate a Phase 1/2 clinical study in patients with non-Hodgkin lymphoma in 2021
2020 Financial Outlook

Gamida Cell ended 2019 with cash, cash equivalents and available-for-sale securities of approximately $56 million (unaudited). The company expects that its current cash, cash equivalents and available-for-sale securities will support the company’s ongoing operating activities into the fourth quarter of 2020. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding that may be received or business development activities that may be undertaken.

About Omidubicel

Omidubicel, the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based cell expansion technology to develop GDA-201 (formerly known as NAM-NK), an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.4

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.