What are the expected timelines and milestones for the Phase 2 trial in NPM1‑mutated AML, and what are the criteria for success? | TUH (Aug 14, 2025) | Candlesense
TUH

What are the expected timelines and milestones for the Phase 2 trial in NPM1‑mutated AML, and what are the criteria for success?

Timeline & Milestones

TuHURA plans to open enrollment for the Phase 2 study of TBS‑2025 + its menin inhibitor in relapsed/refractory NPM1‑mutated AML in the second half of 2025. The protocol calls for a 12‑month enrollment window, with the first patient‑in (FPI) expected by Q3 2025. An interim safety and pharmacodynamic read‑out is slated for ~6 months after the first dose (approximately Q1 2026), followed by a primary efficacy analysis (complete remission + CRi rate, MRD negativity, and duration of response) at the 12‑month endpoint (Q4 2025 – Q1 2026). A data‑cut meeting with the FDA is anticipated after the primary read‑out, positioning the program for a possible Phase 3 launch in 2027 if pre‑specified criteria are met.

Success Criteria

Regulatory and commercial success will hinge on three benchmarks:

1. Safety/Tolerability – ≤30 % dose‑limiting toxicities (DLTs) and no unexpected grade ≥ 3 events attributable to the combination.

2. Efficacy Signal – ≥40 % overall CR/CRi rate in the relapsed/refractory NPM1‑mutated cohort, with ≥30 % achieving MRD‑negative remission, a threshold that would exceed historic outcomes for single‑agent menin inhibition.

3. Pharmacodynamic Confirmation – ≥80 % reduction in menin‑target gene expression (e.g., HOXA9/MEIS1) in bone‑marrow biopsies, demonstrating on‑target activity.

Trading Implications

The upcoming Q3‑2025 FPI and the 6‑month safety read‑out represent catalyst dates. A clean safety profile coupled with an early efficacy signal (≥30 % CR/CRi) could push TUH shares 15‑25 % higher, especially as the market re‑prices the upside of a potential Phase 3‑ready asset in a high‑unmet‑need AML niche. Conversely, any DLTs or efficacy below the 40 % threshold would likely trigger a 10‑15 % sell‑off and may force a redesign of the development plan. Technically, TUH is trading near the 20‑day EMA with modest volume; positioning a buy‑on‑dip at the 20‑day EMA (≈$3.75) with a stop just below the 50‑day EMA (~$3.45) aligns risk with the anticipated data windows. Keep an eye on competitor menin‑inhibitor pipelines (e.g., Revumenib) and AML macro‑trends—positive AML trial news has historically lifted broader AML biotech indices, providing a tailwind for TUH if the trial progresses as outlined.

Other Questions About This News

How does the upcoming Phase 3 trial timeline and design impact the valuation and risk profile of the stock? What are the expected regulatory pathways and timelines for FDA or EMA approval of TBS-2025, especially in combination therapy? Will the Phase 3 initiation be funded entirely by cash on hand, or will the company need to raise additional equity or debt? How does TUH’s current valuation compare to peers developing similar immuno‑oncology assets, such as anti‑PD‑1/PD‑L1 or other checkpoint inhibitors? What potential partnership or licensing opportunities could arise from the successful Phase 2/3 results, and how might they affect dilution or share price? What are the key risks (clinical, regulatory, competitive) that could materially affect the stock price after the upcoming data readouts? How will the acquisition of Kineta and the addition of TBS-2025 affect TUH's cash runway and need for additional financing? How does the potential combination of TBS-2025 with a menin inhibitor compare to existing therapies for relapsed/refractory AML in terms of efficacy and safety? What is the market size and commercial potential for a VISTA‑inhibiting monoclonal antibody in AML and other indications?