- GTA182 demonstrates favorable safety, dose-proportional pharmacokinetics, robust PRMT5 pathway inhibition, and encouraging early antitumor activity
- In MTAP-deleted NSCLC, preliminary results show a 57.1% objective response rate and evidence of intracranial activity
SAN FRANCISCO, Dec. 05, 2025 (GLOBE NEWSWIRE) -- Apeiron Therapeutics, a precision oncology company leveraging structural biology and AI-guided molecular modeling for drug discovery, today presented new clinical data from its ongoing Phase 1 study evaluating GTA182, an oral, brain-penetrant, MTA-cooperative PRMT5 inhibitor, in patients with MTAP-deleted advanced solid tumors, including preliminary antitumor activity in non-small cell lung cancer (NSCLC). These data were presented by Shun Lu, M.D., Ph.D., Professor at Shanghai Chest Hospital and the leading principal investigator on the trial, in a mini-oral session at the ESMO Asia Congress 2025 in Singapore.
“We are pleased to share the first clinical data for GTA182, which demonstrate strong target engagement, early tumor responses, and encouraging signs of intracranial activity in patients with MTAP-deleted advanced solid tumors,” said Mingxi Li, Ph.D., Chief Executive Officer of Apeiron Therapeutics. “These results highlight GTA182’s differentiated profile as a brain-penetrant, MTA-cooperative PRMT5 inhibitor designed specifically for MTAP-deleted tumors. The emerging safety, PK/PD, and antitumor activity data support continued dose exploration and advancement toward expansion cohorts.”
As of October 9, 2025, the data cutoff, 41 subjects with MTAP-deleted advanced solid tumors were treated at doses ranging from 20 to 450 mg once daily. This was a heavily pre-treated patient population representative of the genomic setting, and the maximum tolerated dose had not been reached. GTA182 was generally well tolerated, with the most frequent treatment-related adverse events being anemia (51.2%), decreased appetite (43.9%), and asthenia (39.0%). Grade ≥3 treatment-related adverse events occurred in 26.8% of subjects, consistent with expectations for selective PRMT5 inhibition.
Pharmacokinetic analyses demonstrated dose-proportional increases in exposure up to 450 mg, with low interpatient variability. Consistent with GTA182’s mechanism of action, robust PRMT5 pathway inhibition was observed, with plasma SDMA levels decreasing by a mean of 71.8% (95% CI: 69.7%–73.8%) at active doses (100–450 mg). These pharmacodynamic effects correlated with systemic exposure and support ongoing dose optimization.
Among the 30 efficacy-evaluable subjects treated at active doses, GTA182 demonstrated encouraging and durable antitumor activity, with an objective response rate (ORR) of 30.0% (95% CI: 14.7%–49.4%) and a disease control rate (DCR) of 83.3% (95% CI: 65.3%–94.4%). In the subgroup of MTAP-deleted NSCLC patients (n=14), the ORR was 57.1% (95% CI: 28.9%–82.3%) and the DCR was 85.7% (95% CI: 57.2%–98.2%), with responses observed across dose levels. Notably, two of three patients with baseline brain metastases experienced marked intracranial tumor shrinkage, consistent with GTA182’s brain-penetrant design.
All presentations and posters will be available to registered attendees for on-demand viewing on the ESMO website.
About GTA182
GTA182 is a proprietary small molecule discovered through Apeiron’s AI-guided drug discovery platform. Currently in Phase 1 clinical development, GTA182 demonstrated potent and selective inhibition of PRMT5, exhibiting over 100-fold selectivity for MTAP-deleted tumor cell lines. Importantly, GTA182 is brain-penetrant and has shown tumor growth inhibition and regression in in vivo preclinical models, including glioblastoma (GBM) and various non-CNS cancer models with MTAP deletions.
About Apeiron Therapeutics
Apeiron is redefining medical discovery through the integration of artificial intelligence, streamlining the drug development process from target selection to clinical trials. With a strategic presence in the San Francisco Bay Area and Shanghai, Apeiron combines world-class talent and cutting-edge technologies to drive biomedical innovation. We are committed to advancing breakthrough therapies that address the most pressing unmet medical needs.
For more information, please visit https://www.apeiron-bio.com.
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