On June 27, 2017 TP Therapeutics, Inc., a privately held, clinical-stage biopharmaceutical company focusing on addressing oncology drug resistance, announced today that U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its leading clinical compound TPX-0005 for "treatment of non-small cell lung adenocarcinomas harboring ALK, ROS1, or NTRK oncogenic rearrangements (Press release, TP Therapeutics, JUN 27, 2017, View Source [SID1234519701])."

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The FDA grants orphan drug designation to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including seven years of market exclusivity upon regulatory product approval, exemptions from certain FDA application fees, and tax credits for qualified clinical trials costs.

About TPX-0005

TPX-0005 is a potent and orally bioavailable small molecule kinase inhibitor for ALK, ROS1, and TRK family. The clinical benefits of targeting ALK, ROS1, or TRK fusion kinase have been demonstrated with crizotinib, ceritinib, alectinib, and brigatinib, already approved for the treatment of ALK+ non-small cell lung cancer (NSCLC), crizotinib for ROS1+ NSCLC, and larotrectinib and entrectinib in clinical studies for TRK+ cancers. However, the successes of these therapies are overshadowed by the development of acquired resistance. The acquired solvent front mutations including ALK G1202R, ROS1 G2032R, TRKA G595R and TRKC G623R render a common clinical resistance to the current ALK, ROS1, and TRK inhibitors. TPX-0005 is a potent kinase inhibitor against wildtype and mutated ALK, ROS1 and TRK family kinases, especially the clinically significant solvent front mutations, gatekeeper mutations, and emerging compound mutations after multiple line treatments. TPX-0005 will provide new opportunities in the clinic to inhibit the abnormal signaling of ALK, ROS1, or TRK family in solid malignancies, and overcome multiple resistance mechanisms from the refractory patients. TPX-0005 is currently being evaluated in a Phase 1/2, open-label, multi-center, first-in-human study of the safety, tolerability, pharmacokinetics and anti-tumor activity in patients with advanced solid tumors harboring ALK, ROS1, or NTRK1-3 rearrangements (TRIDENT-1, NCT03093116). For additional information about TPX-0005 trial, please refer to www.clinicaltrials.gov. Interested patients and physicians can also contact the TP Therapeutics Oncology Clinical Trial Hotline at 1-858-276-0005 or email [email protected].

On June 27, 2017 Cellectis (Alternext: ALCLS; Nasdaq: CLLS), a clinical-stage biopharmaceutical company focused on developing immunotherapies based on gene-edited CAR T-cells (UCART), reported the first administration in the Phase I clinical study in Acute Myeloid Leukemia (AML) for its investigational product UCART123, one of the Company’s wholly-controlled TALEN gene-edited product candidates (Press release, Cellectis, JUN 27, 2017, View Source [SID1234519699]). This marks the first allogeneic, "off-the-shelf" gene-edited CAR T-cell product candidate targeting CD123 to be investigated in clinical trials.

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This clinical research in AML is led by Principal Investigator Dr. Gail J. Roboz, Professor of Medicine at Weill Cornell Medicine and Director of the Clinical and Translational Leukemia Programs at Weill Cornell Medicine and NewYork-Presbyterian Hospital.

The clinical trial will investigate the safety and efficacy of UCART123 in patients with AML. AML is a devastating clonal hematopoietic stem cell neoplasm which is characterized by uncontrolled proliferation and accumulation of leukemic blasts in bone marrow, peripheral blood and, occasionally, in other tissues. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure. In the U.S., there are an estimated 19,950 new AML cases per year, with 10,430 estimated deaths per year. While complete response rates can be as high as 80 percent in younger patients who undergo initial induction cytotoxic chemotherapy, the majority of AML patients relapse and die from the disease. AML patients with high-risk genetic features have an especially urgent unmet medical need, as their outcomes are dismal with all existing treatment modalities, including allogeneic stem cell transplantation.

"After being granted rapid approval from Regulatory Authorities and Institutional Review Boards to initiate UCART123 studies, the enrollment and treatment of the first patient represents a major milestone for Cellectis, and we are eager to hit the ground running with the recruitment of our first patient for our second UCART123 Phase I study in BPDCN soon," said Dr. Loan Hoang-Sayag, Cellectis Chief Medical Officer. "This first program targeting CD123 will be a paradigm shift for our Company, as it will provide a wealth of valuable additional knowledge and data to drive our gene-edited allogeneic CAR T-cell platform."

"We are excited to be enrolling our first patient with UCART123 and are hopeful that this novel immunotherapy modality will prove to be a significant and effective weapon against AML," said Dr. Roboz.

The clinical trial is part of a strategic translational research alliance that was formed between Cellectis and Weill Cornell Medicine in 2015. Dr. Monica Guzman, an associate professor of pharmacology in medicine at Weill Cornell Medicine, is co-principal investigator whose work focuses on preclinical and early-stage testing to optimize the development of stem cell-targeted cancer drugs.

On June 27, 2017 MabVax Therapeutics Holdings, Inc. (Nasdaq: MBVX), a clinical-stage oncology drug development company with a fully human antibody discovery platform focused on the rapid translation into clinical development of products to address unmet medical needs in the treatment of cancer, reported that the first patient has been dosed in the Phase 1 clinical trial evaluating MVT-1075, the Company’s fully human antibody radioimmunotherapy (RIT), for the treatment of CA19-9 positive malignancies including pancreatic, colon and lung cancers.

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This Phase 1 first-in-human clinical trial is an open-label, multi-center study evaluating the safety and efficacy of MVT-1075 in up to 22 patients with CA19-9 positive malignancies in the U.S. The primary objective is to determine the maximum tolerated dose and safety profile in patients with recurring disease who have failed prior therapies. Secondary endpoints are to evaluate tumor response rate and duration of response by RECIST 1.1, and to determine dosimetry and pharmacokinetics. This dose-escalation study utilizes a traditional 3+3 design. The investigative sites will include Honor Health in Scottsdale, Arizona and Memorial Sloan Kettering Cancer Center in New York City.

"MabVax is pleased to announce that we have advanced into the clinic with the dosing of the first patient in our MVT-1075 RIT lead development program. This significant milestone is a tribute to the integrated collaboration of our research and development staff with our clinical collaborators to bring this potentially life-changing therapy to patients with pancreatic, colon and small cell lung cancers," said David Hansen, MabVax’s President and Chief Executive Officer. "This is the third HuMab-5B1 based clinical trial that the Company has initiated in the last sixteen months. We believe that the development of this product candidate expands our pipeline to include a more potent therapy combining the clinically demonstrated tumor targeting characteristics of our fully human HuMab-5B1 antibody and commercially validated 177Lutetium, to deliver a dose of radiation to the targeted cancer cells. We look forward to continuing enrollment, and expect to report interim trial results by the end of 2017."

Supporting the MVT-1075 RIT clinical investigation are the Company’s successful Phase 1a safety and target specificity data reported at the annual meetings of the American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the Society for Nuclear Medicine and Molecular Imaging (SNMMI) earlier this month, including the clinical results for the Company’s HuMab-5B1 products, MVT-5873, a single agent therapeutic antibody and MVT-2163, an immuno-PET imaging agent. The combined results from 50 patients in the Phase 1 MVT-5873 and MVT-2163 studies, established safety and provided significant insight into drug biodistribution and an optimal dosing strategy, which the Company has incorporated into the MVT-1075 program. In April, the Company reported preclinical results for MVT-1075 at the American Association of Clinical Research (AACR) (Free AACR Whitepaper) Annual Meeting, demonstrating marked suppression, and in some instances, regression of tumor growth in xenograft animal models of pancreatic cancer, potentially making this product an important new therapeutic agent in the treatment of pancreatic, colon and lung cancers.

For additional information about the Phase 1 MVT-1075 clinical trial, please visit clinicaltrials.gov, and reference Identifier NCT03118349.







About MVT-1075

MVT-1075 is a radioimmunotherapy product that combines established efficacy of radiation therapy with tumor specific targeting. It has the potential to deliver a more potent HuMab-5B1 based product. MVT-1075 uses small doses of the Company’s MVT-5873 antibody, coupled to a radioisotope to target pancreatic cancer cells and kill them.