NEW YORK & SEOUL, South Korea–(BUSINESS WIRE)–AnHeart Therapeutics and NewG Lab Therapeutics, a subsidiary of NewG Lab Pharma, announced that the Ministry of Food and Drug Safety (MFDS), Republic of Korea has granted the Designation of Orphan Drug (ODD) to taletrectinib, a next generation tyrosine kinase inhibitor (TKI) for the treatment of adult patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).
The MFDS’s ODD is for the pharmaceuticals used to treat diseases with a prevalence of 20,000 patients or less in the Korean population, and for which the appropriate treatment methods and medicines have not been developed. The efficacy and safety of the pharmaceuticals with ODD status should be significantly improved over existing alternative drugs if the drug is designated as an orphan drug, it can be approved earlier by expedited review process.
“There is a high unmet need for this specific patient population in lung cancer, where very few treatment options are available,” said Junyuan (Jerry) Wang, PhD, Chief Executive Officer, AnHeart Therapeutics. “We look forward to working with the regulatory agency in Republic of Korea, to accelerate the development of taletrectinib for patients in Korea.”
“Receiving the ODD status from the MFDS in Korea is a significant milestone in the development of taletrectinib for Asian cancer patients in need of better therapies,” an official from NewG Lab Pharma said. “The recent ODD will allow for accelerated approval and commercialization of taletrectinib in Korea.”
The global phase 2 clinical trial (TRUST-II study) for adult patients with advanced or metastatic ROS1-positive NSCLC who are ROS1 TKI naïve or previously treated with crizotinib is ongoing at multiple sites in Korea including Asan Medical Center in Seoul and Chonnam National University Hospital in Hwasun.
Taletrectinib was granted Breakthrough Therapy Designation by US FDA in August 2022. The FDA decision was supported by the preliminary clinical data of taletrectinib from an ongoing Phase 2 trial (TRUST study) in Chinese ROS1-positive NSCLC patients and two completed Phase 1 clinical trials conducted in the United States and Japan.
According to interim results from Phase 2 TRUST clinical trial announced at ASCO 2022, in ROS1 TKI-naïve patients, the confirmed overall response rate (cORR) was 92.5% (62/67), including 2 confirmed complete response (cCR); and disease control rate (DCR) was 95.5% (64/67). In crizotinib-pretreated patients, the cORR was 50% (19/38), and DCR was 78.9% (30/38). Of the 12 patients with brain metastasis and measurable brain lesions at baseline, the intra-cranial overall response rate (IC-ORR) and intra-cranial disease control rate (IC-DCR) were 91.7% and 100%, respectively.
In July 2020, AnHeart Therapeutics out-licensed the clinical development and commercialization rights of taletrectinib in Republic of Korea, to NewG Lab Co. NewG Lab will be responsible for development, market authorization and commercialization activities for taletrectinib in Korea.
About Taletrectinib
Taletrectinib is a novel, potential best-in-class, next-generation ROS1 inhibitor designed to effectively target ROS1 fusions with potential to treat both TKI-naïve and pre-treated patients. ROS1 fusion is estimated to be an oncogenic driver in approximately 2 percent of patients with NSCLC. ROS1 fusions are also observed in several other cancers such as cholangiocarcinoma, glioblastoma, ovarian, gastric, and colorectal cancers.
Taletrectinib has demonstrated excellent potency against crizotinib resistant secondary mutations, good brain penetration and intracranial antitumor activity, and favorable safety profiles in ROS1 fusion-positive NSCLC patients. In these patients, few neurological adverse events were observed, which likely benefits from the selective inhibition of ROS1 over TRKB by taletrectinib.
Taletrectinib has received FDA Breakthrough Therapy Designation (BTD) for the treatment of adult patients with advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) who are ROS1 tyrosine kinase inhibitor (TKI) treatment naïve or previously treated with crizotinib.
More information about the ongoing China TRUST (Taletrectinib ROS1 LUng STudy) phase 2 trial and the global TRUST-II phase 2 trial can be found by searching clinical trial identifiers NCT04395677 and NCT04919811, respectively at https://clinicaltrials.gov. For questions about the ongoing trials, please contact trials@anhearttherapeutics.com.
About AnHeart Therapeutics
AnHeart Therapeutics (“AnHeart”) is a clinical-stage global biopharmaceutical company developing a broad pipeline of novel precision oncology therapeutics with high unmet medical needs. Its lead asset, taletrectinib, is a potential best-in-class next-generation ROS1 inhibitor currently in Phase 2 trials for ROS1 TKI-naïve and TKI-pretreated patients with ROS1 fusion-positive non-small cell lung cancer (NSCLC). The Company’s pipeline also includes AB-218, a mIDH1 inhibitor in Phase 2 trials with good brain penetration for multiple solid tumors with mIDH1 mutations and AB-329, an AXL inhibitor in Phase 1 studies to be used in combination with checkpoint inhibitor or chemotherapies in NSCLC or other solid tumors. The Company operates from offices in the US and China. For more information, visit https://www.anhearttherapeutics.com/.
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