Terns Pharmaceuticals, Inc. (NASDAQ:TERN) presented updated and expanded data from the ongoing CARDINAL trial of TERN-701 in previously treated patients with chronic myelogenous leukemia (CML) at the American Society of Hematology Annual Meeting and Exposition on Monday.
By the cut-off date of September 13, 2025, 63 patients were enrolled.
Facts
Of the 38 efficacy-evaluable patients, the overall (cumulative) major molecular response (MMR) rate was 74% (28/38) at 24 weeks, with 64% (18/28) achieving MMR and 100% (10/10) maintaining MMR.
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The deep molecular response (DMR) performance rate at 24 weeks was 29%. No patients had lost MMR at the time of data cutoff.
Encouraging safety profile: 87% (55/63) of patients remained on treatment as of data cutoff.
No dose-limiting toxicities were observed with dose escalation and no maximum tolerated dose was reached.
The majority of treatment-emergent adverse events were low grade and there was no clear dose relationship.
Exposures to TERN-701 were approximately dose-proportional over the entire dose range.
The safety profile and higher BMR performance rate of 75% over 24 weeks at doses of 320 mg and higher support the selection of 320 mg and 500 mg once daily as the recommended phase 2 doses (RP2Ds) for expansion.
“We are particularly encouraged to see unprecedented MMR rates in a highly refractory population, including compelling response performance in patients with lack of efficacy to prior asciminib, ponatinib, and/or other marketed and investigational TKIs,” said Emil Kuriakoshead doctor of Terns.
The company highlighted that in the RP2D dosing range, a DMR performance rate of 36% was observed at 24 weeks, highlighting the rapid response kinetics of TERN-701.
The safety profile and higher BMR performance rate of 75% over 24 weeks at doses of 320 mg and higher support the selection of 320 mg and 500 mg once daily as the recommended Phase 2 doses (RP2Ds) for expansion,” said Amy BurroughsCEO of Terns, in a press release on Monday.
Analyst Take
“We believe TERN-701 is on track to challenge Scemblix’s dominance and disrupt the treatment paradigm of CML, which has a global TAM of approximately $5 billion,” William Blair wrote on Tuesday.
From an efficacy perspective, analyst Andy Hsieh is impressed with the reported MMR performance rate of 64%, which is double that of approved Scemblix and research instruments Enliven Therapeutics, Inc (NASDAQ:ELVN) ELVN-001 agents reporting MMR performance rates in the range of 24%-32% in similar disease settings.
“There were six additional efficacy-evaluable patients included in the presentation compared to the summary, four of whom achieved an MMR, and we believe the consistency of the efficacy data contributes to our optimism,” William Blair added.
TERN price promotion: Terns Pharma shares fell 4.25% to $38.52 in premarket trading on Tuesday, according to data from Benzinga Pro.
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