TERN-701 Achieves 64% MMR at 24 Weeks in Tough-to-Treat CML Patients—ASH Presentation Highlights Potential Breakthrough
Phase 1 Data Show Major Molecular Response (MMR) Rates at Least Double Competing Therapies
Terns Pharmaceuticals made headlines this week with the announcement that data from its Phase 1 CARDINAL trial for TERN-701—targeting chronic myeloid leukemia (CML) patients who have failed previous therapies—will be presented orally at the American Society of Hematology (ASH) 2025 Annual Meeting. The key takeaway: 64% of these heavily pretreated, refractory patients achieved a major molecular response (MMR) by 24 weeks. That rate is at least twice as high as what's been observed in similar Phase 1 studies, positioning TERN-701 as a potential game-changer for the CML treatment landscape.
Consistent Efficacy in Challenging Subgroups and Encouraging Safety
For clinicians and investors watching the CML space, consistency across patient subgroups is just as important as headline efficacy. In this cohort:
- Overall (cumulative) MMR rate by 24 weeks was 75% (24 of 32 patients)
- In patients who failed their last tyrosine kinase inhibitor (TKI), the MMR rate was 69%
- For those with prior asciminib exposure, MMR hit 60%
- No patient who achieved MMR at cutoff had lost it
All these results came with a safety profile that appears especially promising: 87% of all patients enrolled remained on therapy at the June 30 data cutoff, and the majority of side effects were mild (grade 1 or 2).
| Group | MMR Rate by 24 Weeks | Patients (n) |
|---|---|---|
| All Evaluable | 75% | 32 |
| Lack of efficacy to last TKI | 69% | 16 |
| Prior asciminib | 60% | 10 |
| Prior asciminib/ponatinib/investigational TKI | 67% | 12 |
Safety Profile: No Dose-Limiting Toxicities Observed, Mostly Mild Side Effects
Out of 55 enrolled patients, 87% remained on treatment, with only four discontinuations due to disease progression, and just one for adverse events. Importantly, no dose-limiting toxicities (DLTs) were observed at any dose level up to 500 mg. The majority (74%) of treatment-emergent adverse events (TEAEs) were mild, and there were no dose-dependent safety concerns. Grade 3 or higher events were uncommon and mostly involved neutropenia or thrombocytopenia.
- Most common mild TEAEs: Diarrhea (22%), headache (18%), and nausea (16%)
- Grade 3 or higher: Neutropenia (7%), thrombocytopenia (4%)
Market Context: Why the TERN-701 Results Could Shift the CML Landscape
Historically, new treatments for CML—especially those targeting relapsed/refractory patients—struggle to demonstrate significant MMR rates and tolerability in tough-to-treat populations. By comparison, TERN-701's efficacy signals (with no patients losing MMR at cutoff and durable responses across difficult patient groups) stand out. The company emphasizes the potential for "best-in-disease" therapy, particularly with additional data to be shared at ASH in December. Investors and medical professionals alike will be keen to see if the larger data set confirms these unprecedented rates.
What to Watch: Upcoming ASH Oral Presentation and Investor Call
TERN's management team will host a webcast and conference call after the ASH presentation on December 8th, where they plan to discuss expanded results and next development steps for TERN-701. With its safety and efficacy edge now on the radar, TERN-701’s upcoming data release is set to be a key focus for anyone tracking advances in hematology—and potential shifts in the competitive landscape.
The CML treatment arena is no stranger to setbacks or high hurdles. Yet with a 64% MMR in a highly refractory patient group—and a side effect profile that keeps patients on therapy—TERN-701 is positioned to spark meaningful conversations as the full data emerge in December.
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