ASCO 2024: Olverembatinib Shows 92.3% CBR in SDH-Deficient GIST

Ascentage Pharma, a biopharmaceutical company based in SUZHOU, China and Rockville, Maryland, has revealed updated clinical data on its third-generation tyrosine kinase inhibitor (TKI), olverembatinib (HQP1351). This information was presented during an oral report at the 60th American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago, Illinois. Notably, this marks the third consecutive year that findings from olverembatinib studies have been showcased at this prestigious event.

The ASCO Annual Meeting is a key platform for unveiling innovative oncology research and advanced cancer therapies. This year, Ascentage Pharma presented four clinical studies involving three of its proprietary drug candidates, including olverembatinib, during the meeting.

The latest clinical data focused on olverembatinib's effectiveness and safety in treating patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors (GIST) that are resistant to previous TKI treatments. This Phase I study enrolled 26 patients, revealing that six experienced partial responses, while 18 maintained stable disease for more than four treatment cycles. The clinical benefit rate (CBR) stood at a remarkable 92.3%, with a median progression-free survival (PFS) of 25.7 months.

Developed by Ascentage Pharma, olverembatinib is an orally administered TKI and the first third-generation BCR-ABL inhibitor approved in China. It is currently approved for treating adult patients with TKI-resistant chronic-phase chronic myeloid leukemia (CML-CP) or accelerated-phase CML (CML-AP) with the T315I mutation, as well as for adult patients with CML-CP who are resistant or intolerant to first- and second-generation TKIs. In China, olverembatinib is co-commercialized by Ascentage Pharma and Innovent Biologics.

While initially developed for hematologic malignancies, olverembatinib is also undergoing clinical evaluations for GIST. The drug has received Breakthrough Therapy Designation from China's National Medical Products Administration (NMPA) for treating patients with SDH-deficient GIST who have undergone first-line treatment.

Professor Haibo Qiu from Sun Yat-Sen University Cancer Center, who presented the report, highlighted the significant treatment gap for SDH-deficient GIST, stressing the need for new therapeutic options. According to Qiu, olverembatinib has demonstrated substantial clinical benefits in this patient group, with favorable safety and tolerability profiles, and a median PFS of 25.7 months.

Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, echoed these sentiments, emphasizing the drug's potential to address urgent unmet clinical needs. Encouraged by the results, Ascentage Pharma aims to expedite the clinical development of olverembatinib to provide a safe and effective treatment option for patients globally.

Key data presented at ASCO 2024 included updated efficacy results of olverembatinib in patients with TKI-resistant SDH-deficient GIST. The study, led by first author Haibo Qiu, MD, PhD, from Sun Yat-sen University Cancer Center, involved 26 patients aged between 13 and 56 years who had previously received multiple TKIs. The findings showed that olverembatinib was well tolerated and provided a clinical benefit rate exceeding 90%, with a median PFS of 25.7 months.

Ascentage Pharma remains committed to addressing unmet clinical needs worldwide. Leveraging robust R&D capabilities, the company continues to develop innovative therapies and expand its global partnerships with leading biotech and pharmaceutical companies.

In conclusion, olverembatinib's promising clinical efficacy and manageable safety profile represent a significant advancement in the treatment of SDH-deficient GIST, offering hope for improved patient outcomes in this challenging field.