Terns Pharmaceuticals Inc. (NASDAQ: TERN) has presented encouraging and expanded findings from its ongoing CARDINAL clinical investigation involving TERN-701. This drug targets chronic myeloid leukemia (CML) in patients who have previously undergone treatment. The data, updated as of September 13, 2025, included 63 enrolled patients, demonstrating notable efficacy and a promising safety profile.
A significant portion of patients, 74% (28 out of 38 who could be evaluated for efficacy), achieved a major molecular response (MMR) within 24 weeks. Impressively, 64% (18 out of 28) reached MMR, and all 100% (10 out of 10) maintained this response. The deep molecular response (DMR) rate stood at 29% within the same timeframe, with no patients losing MMR by the data cutoff. The drug also showed a favorable safety profile, with 87% (55 out of 63) of patients continuing treatment, and no dose-limiting toxicities were observed during dose escalation. Terns Pharmaceuticals' Chief Medical Officer, Emil Kuriakose, expressed optimism regarding the unprecedented MMR rates in a highly refractory patient population, including those who had not responded to prior treatments like asciminib and other tyrosine kinase inhibitors.
These positive results support the selection of 320mg and 500mg daily as the recommended Phase 2 doses (RP2Ds) for further study. Analysts, including William Blair, view TERN-701 as a significant challenger to Novartis AG's (NYSE: NVS) Scemblix, a current market leader in CML treatment, citing TERN-701's higher MMR achievement rate of 64% compared to Scemblix and other investigational agents. This suggests a potential disruption in the CML treatment market, estimated to be around $5 billion globally, and offers new avenues for patients battling this condition.
The advancements in medical research, particularly in challenging diseases like CML, highlight the continuous pursuit of better health outcomes for individuals worldwide. Each step forward in developing effective treatments brings us closer to a future where debilitating illnesses are more manageable, fostering a healthier and more vibrant global community.