Turning Point Therapeutics Presents First Clinical Data From Repotrectinib From Care Study in Pediatric and Young Adult Patients at SIOP 2021 Virtual Congress



SAN DIEGO, Oct 23, 2021 (GLOBE NEWSWIRE) – Turning Point Therapeutics, Inc. (NASDAQ: TPTX), a precision oncology company developing next-generation therapies targeting genetic factors in cancer, announced today hui presentation of early clinical data from the ongoing CARE Phase 1/2 study in pediatric and young adult patients with advanced solid tumors harboring ALK, ROS1 Where NTRK modifications. These data are presented today at the 53rd Virtual Congress of the International Society of Pediatric Oncology (SIOP) which is being held October 21-24.

“These early data on repotrectinib demonstrate encouraging clinical activity in this pediatric patient population,” said Mohammad Hirmand, MD, medical director. “Patient recruitment continues in the Phase 1/2 CARE study, and we look forward to advancing the development of repotrectinib in this patient population. “

Preliminary data from the phase 1/2 CARE study
The primary objective of the phase 1 dose escalation portion of the study is to assess the safety and tolerability and to determine the recommended phase 2 dose (RP2D) of repotrectinib in pediatric patients younger than 12 years. The main objective of the phase 2 part of the study is to determine the anti-tumor activity of repotrectinib in pediatric patients and young adults under 25 years of age. Repotrectinib is given as a capsule or suspension at a dosage based on weight in patients less than 12 years of age. Patients aged 12 to 25 years can enroll directly in the phase 2 part of the study with a dose of repotrectinib of 160 mg once daily for the first 14 days and may increase to 160 mg BID thereafter. .

The first phase 1/2 CARE dataset uses a data cut-off date of August 2, 2021. Ten patients were treated at two dose levels. The safety analysis includes the ten patients treated and the preliminary efficacy analysis includes eight evaluable patients. Patients included in the efficacy analysis had measurable disease at inclusion and at least one evaluable analysis after inclusion. Assessment of response was performed by physician and according to RECIST v1.1 or RANO for CNS tumors. Responses were confirmed by subsequent analysis at least 28 days later.

The results were reported in a pre-recorded presentation by Steven G. Dubois, MD, associate professor of pediatrics, Harvard Medical School available Oct. 23 at 9:12 a.m. ET on the meeting website.

Preliminary safety analysis (n = 10) and pharmacokinetic analysis

  • Repotrectinib was generally well tolerated.

  • The most frequently reported treatment-related adverse events (TREs) were anemia (n = 5) and fatigue (n = 5). Among the anemic patients, three had a history of anemia at baseline.

  • The vertigo (n = 4) was grade 1 or 2 and none led to discontinuation of treatment.

  • No patient discontinued treatment for reasons other than disease progression and no patient experienced an AEFI leading to dose reduction.

  • No dose limiting toxicity was reported.

  • Preliminary pharmacokinetic data indicated that repotrectinib exposure in different age groups was comparable to adult exposure at steady state.

Preliminary efficacy analysis (n = 8)

  • Eight patients were evaluable for efficacy, including four TKI-naïve patients and four TKI-pretreated patients. TKI-naïve patients included those with NTRK-amplified anaplastic ependymoma (n = 1), NTRK fusion glioblastoma multiforme (GBM) / high-grade glioma (n = 1), NTRK fusion sarcoma (n = 1 ) and an inflammatory fusion ROS1 myofibroblastic tumor (IMT) (n = 1). TKI-pretreated patients included those with high-grade NTRK / glioma fusion GBM (n = 2), NTRK fusion mesoblastic nephroma (n = 1), and NTRK fusion sarcoma (n = 1).

  • Three TKI naive patients (2 solid NTRK fusion tumors; 1 ROS1 fusion IMT) had confirmed responses. One of the three responder patients had a high-grade NTRK / glioma fusion GBM, had previously been treated with tumor resection, whole brain radiation therapy and multi-agent chemotherapy, and achieved a complete response (CR) and remained in response for more than 3.8 months from the data deadline. The other two confirmed responders remained in response with a duration of response of 7.3+ and 12.1+ months, respectively.

  • Of the four TKI-pretreated patients, one patient with NTRK fusion sarcoma had a better stable disease response.

Phase 1 dose finding of the study is underway in pediatric patients under 12 years of age to confirm pediatric RP2D. The Phase 2 part of the study is underway for patients aged 12 to 25 years.

Turning Point also announced the publication of preclinical data for repotrectinib in neuroblastoma, the most common pediatric extracranial solid tumor, in the American Association of Cancer Research peer-reviewed journal, Molecular cancer therapy. The preclinical studies described in the publication titled “Translational Strategies for Repotrectinib in Neuroblastoma” show that repotrectinib inhibits tumor growth and prolongs survival in patient-derived neuroblastoma xenograft models. Additionally, studies have indicated that combining repotrectinib with chemotherapy may be a promising treatment paradigm for patients with neuroblastoma.

About Turning Point Therapeutics Inc.
Turning Point Therapeutics is a clinical-stage precision oncology company with a pipeline of investigational drugs discovered in-house and designed to address the key limitations of existing cancer therapies. The Company’s lead drug candidate, repotrectinib, is a next-generation kinase inhibitor targeting the oncogenic factors ROS1 and TRK in non-small cell lung cancer and advanced solid tumors. Repotrectinib, which is being investigated in a Phase 2 registration study in adults and a Phase 1/2 study in pediatric patients, has shown anti-tumor activity and long-lasting responses in treatment-naïve patients. by kinase inhibitor and pretreated. The company’s portfolio of drug candidates also includes elzovantinib, targeting MET, CSF1R and CRS, which is being investigated in a phase 1 trial in patients with advanced or metastatic solid tumors with genetic alterations in MET. ; TPX-0046, targeting RET, which is being studied in a phase 1/2 trial in patients with advanced or metastatic solid tumors with genetic alterations in RET; and TPX-0131, a next-generation ALK inhibitor, which is being investigated in a phase 1/2 trial in previously treated patients with advanced or advanced non-small cell lung cancer. ALK-positive metastatic. The company is committed to developing therapies that mark a turning point for patients in their cancer treatment. For more information visit www.tptherapeutics.com.

Forward-looking statements
Statements in this press release regarding matters which are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Since such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by these forward-looking statements. Words such as “plans”, “will”, “believes”, “anticipates”, “expects”, “intention”, “goal”, “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements in this press release include statements regarding, among other things, the efficacy, safety and therapeutic potential of repotrectinib, as well as the results, conduct, progress and schedule of the phase clinical study. 1/2 CARE of Turning Point Therapeutics. These forward-looking statements are based on Turning Point Therapeutics’ current expectations and involve assumptions which may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in these forward-looking statements due to various risks and uncertainties, which include, without limitation, the risks and uncertainties associated with the business of Turning Point Therapeutics in general, risks and uncertainties. related to the impact of the COVID-19 pandemic on Turning Point Therapeutics’ business and other risks described in documents filed by Turning Point Therapeutics with the Securities and Exchange Commission (SEC), including its Quarterly Report on Form 10 -Q Filed with the SEC August 9, 2021 All forward-looking statements contained in this press release speak only as of the date they were made. Turning Point Therapeutics assumes no obligation to update these statements to reflect events that occur or circumstances that exist after the date they were made.


Scott Lipman



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