Xiaochuan Dai

Xiaochuan (David) Dai of UBS requested additional details on the statistical powering for the EVOLVE-2 trial, particularly with the inclusion of dual primary endpoints.

Answer

CEO Michael Metzger confirmed the trial is designed to enroll approximately 415 patients and that its statistical power is based on the more prevalent NPM1 mutant population, although KMT2A rearranged patients will also be enrolled. He stated that specific statistical details beyond the patient number have not been made public.

Xiaochuan Dai of UBS asked about the potential timing for Revuforj's inclusion in NCCN guidelines for NPM1-mutant AML and whether it could happen before FDA approval. He also inquired about discussions with regulators on using MRD negativity as a surrogate endpoint.

Answer

CEO Michael Metzger stated the plan is to submit the NPM1 data for publication and seek NCCN guideline inclusion in Q2, well ahead of a potential year-end FDA approval. President and Head of R&D Dr. Neil Gallagher noted that while the company is active in an NIH consortium on MRD, they do not comment on specifics of interactions with health authorities.

Xiaochuan Dai of UBS asked about the key gating steps required to initiate the Phase 3 KOMET-017 trial in the second half of the year, including progress on site activations and early feedback from investigators.

Answer

CEO Troy Wilson confirmed the trial is in study startup, with activities like contracting and budgeting underway, reiterating the H2 2025 initiation timeline. Executive Mollie Leoni added that feedback from the related KOMET-007 trial has been very positive, with investigators noting the drug's high tolerability, which is building excitement for the Phase 3 study.

Xiaochuan Dai from UBS asked for expectations on the efficacy and duration bar for the upcoming pivotal data in relapsed/refractory NPM1-mutated AML and if data on patients with prior menin inhibitor exposure would be presented at ASH.

Answer

CEO Dr. Troy Wilson reiterated the regulatory bar for approval is a 20-30% CR/CRh rate and a 4-6 month median duration of response. He confirmed that some data on patients with prior menin inhibitor exposure will be presented at ASH but cautioned that the population is heterogeneous and they are still learning how to best treat this group, though activity has been observed.

Xiaochuan 'David' Dai from UBS asked about the NANORAY-312 trial design, specifically the expected proportion of patients receiving cetuximab. He also requested the mechanistic rationale for how NBTXR3 provides additive systemic benefits when combined with PD-1 inhibitors in metastatic head and neck cancer.

Answer

Co-Founder and CEO Laurent Levy explained that cetuximab use is a stratification factor for balance but is not expected to be a key efficacy driver in this frail patient population. On the PD-1 combination mechanism, he emphasized that NBTXR3's primary benefit is providing crucial local tumor control. He added that preclinical studies have shown NBTXR3 can also trigger a deeper systemic immune response, and the company is exploring both local and systemic effects in the upcoming data from Study 1100.

Xiaochuan Dai from UBS asked for details on the preclinical data that provides confidence in the HIV program's potential for viral control.

Answer

David Berman, Head of R&D, pointed to several published papers demonstrating that the molecule can redirect T-cells to kill HIV-infected cells in vitro. CEO Bahija Jallal added a key reason for confidence is the platform's ability to kill cells with very low target expression, a known characteristic of the latent HIV reservoir.