The third generation EGFR inhibitor ck-101 was identified as orphan drug

Source: Sina Pharmaceutical News September 11, 2017, checkpoint therapeutics, a holding company of Fortress biotech, said that the FDA of the United States has granted the company the qualification of orphan drugs for the treatment of EGFR mutation positive non-small cell lung cancer (NSCLC) with the third generation EGFR inhibitor ck-101 (also known as rx518) under research At present, ck-101 is in the phase of dose increasing research in clinical 1 / 2 phase trial The clinical phase 1 part of this study was used to evaluate the safety and tolerability of increasing dose CK ‐ 101 in the treatment of advanced solid tumor patients, so as to obtain the maximum tolerable dose and the recommended dose of clinical phase 2 part The clinical phase 2 study was used to evaluate the safety and efficacy of ck-101 in the treatment of EGFR-T790M mutation positive non-small cell lung cancer James F Oliviero, President and CEO of checkpoint therapeutics, said: "the identification of harvest orphan drug is a milestone in the development of ck-101, which will support the clinical development process of the drug for EGFR mutation positive NSCLC treatment The status of ck-101 orphan drug granted by FDA is based on the published US compound patent of ck-101 If we obtain orphan drug qualification, we will get additional market monopoly period besides the patent " The US FDA orphan drug qualification is designed to give a preferential policy to those drugs or biological products that have clinical efficacy in the treatment, diagnosis or prevention of rare diseases with fewer than 200000 patients in the US The incentives of orphan drug policy to pharmaceutical enterprises are as follows: orphan drug qualification can provide drug applicants with various development incentives, including qualified clinical test tax credit; for prescription drugs with orphan drug qualification, their listing application is not subject to prescription drug applicant payment Act (PDUFA) Cost limitation; orphan drug qualification will be granted a market monopoly period of 7 years beyond the patent protection period Ck-101 is an oral third generation EGFR selective covalent inhibitor In March 2015, checkpoint therapeutics reached an agreement with neupharma to obtain the exclusive license for the global development and commercialization of the drug in addition to some Asian countries and regions In August 2016, FDA accepted the ind application of the drug and started the administration of the first enrolled patient in the clinical phase 1 / 2 study in September 2016 The indications that ck-101 is planned to develop include second-line therapy as a patient with EGFR T790M mutant NSCLC, and first-line therapy as a patient with EGFR L858R and del 19 mutations In addition, preclinical studies also showed that ck-101 has therapeutic potential in combination with immunocheckpoint inhibitors (PD-1 or PD-L1), CMET inhibitors and MEK inhibitors.