Terns Pharmaceuticals, Inc. (NASDAQ:TERN) on Monday presented updated and expanded data from the ongoing CARDINAL trial of TERN-701 in previously treated chronic myeloid leukemia (CML) patients at the American Society of Hematology Annual Meeting and Exposition.
As of the cutoff date of September 13, 2025, 63 patients were enrolled.
Data
Of 38 efficacy-evaluable patients, the overall (cumulative) major molecular response (MMR) rate was 74% (28/38) by 24 weeks, with 64% (18/28) achieving MMR and 100% (10/10) maintaining MMR.
Deep molecular response (DMR) achievement rate by 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 the data cutoff.
No dose-limiting toxicities were observed in dose escalation, and a maximum tolerated dose was not reached.
The majority of treatment-emergent adverse events were low grade with no apparent dose relationship.
TERN-701 exposures were approximately dose-proportional across the dose range.
The safety profile and higher MMR achievement rate of 75% over 24 weeks at doses of 320mg and above supports selection of 320mg and 500mg QD as the recommended phase 2 doses (RP2Ds) for expansion.
“We are particularly encouraged to see unprecedented rates of MMR in a highly refractory population, including compelling response achievement in patients with a lack of efficacy on prior asciminib, ponatinib, and/or other marketed and investigational TKIs,” stated Emil Kuriakose, chief medical officer of Terns.
The company highlighted that in the RP2D dose range, a 36% DMR achievement rate by 24 weeks was observed, highlighting the fast response kinetics of TERN-701.
Novartis AG (NYSE:NVS) markets asciminib as Scemblix as a targeted prescription medication used to treat adults with Philadelphia chromosome-positive chronic myeloid leukemia.
The safety profile and higher MMR achievement rate of 75% over 24 weeks at doses of 320mg and above support the selection of 320mg and 500mg QD as the recommended phase 2 doses (RP2Ds) for expansion,” said Amy Burroughs, CEO 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 roughly $5 billion,” William Blair wrote on Tuesday.
From an efficacy perspective, analyst Andy Hsieh is impressed by the reported MMR achievement rate of 64%, which doubled that of approved Scemblix and investigational Enliven Therapeutics, Inc.’s (NASDAQ:ELVN) ELVN-001 agents that reported MMR achievement rates in the range of 24%-32% in comparable disease settings.
“There were six additional efficacy evaluable patients included in the presentation as compared to the abstract, four of whom achieved MMR, and we believe the consistency of efficacy data adds to our bullish conviction,” William Blair added.
TERN Price Action: Terns Pharma shares were down 4.25% at $38.52 during premarket trading on Tuesday, according to Benzinga Pro data.
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