SAN DIEGO–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.”
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.
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 firstname.lastname@example.org.
About TP Therapeutics, Inc.
TP Therapeutics, Inc. (TP) is a clinical-stage structure-based drug design company founded in October 2013 by Dr. J. Jean Cui, the lead inventor of Pfizer’s oncology drug crizotinib. TP Team is focusing on the design and development of novel chemical entities for established oncogene drivers with high incidence of secondary resistance mutations, newly identified disease-driven targets, and potential targets regulating tumor microenvironment and tumor immunity. For more information, please visit us at www.tptherapeutics.com.