Syndax(US:SNDX)2021-05-12 02:31Financial Data and Key Metrics Changes - The net loss for Q1 2021 was $27.7 million, or $0.54 per share, compared to a net loss of $19.1 million, or $0.56 per share for the same period last year [37] - The company ended Q1 2021 with $271.3 million in cash and cash equivalents, providing a cash runway into 2023 [38] Business Line Data and Key Metrics Changes - The Phase 1 trial of SNDX-5613 reported a 48% overall response rate with 67% of responses being MRD negative in patients with relapsed or refractory acute leukemia [11][10] - Axatilimab's Phase 2 expansion cohort has been fully enrolled, with updated data expected later this year [27] Market Data and Key Metrics Changes - Approximately 14,000 patients in the U.S. suffer from chronic graft versus host disease, representing a significant commercial opportunity [31] Company Strategy and Development Direction - The company aims to initiate Phase 2 trials for SNDX-5613 by the end of Q2 2021 and is focused on obtaining regulatory approval for drugs that extend and improve the lives of cancer patients [22][43] - There is ongoing research into expanding the 5613 franchise beyond initial approvals in relapsed refractory acute leukemias [26] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the broad franchise opportunities for both SNDX-5613 and axatilimab, indicating confidence in advancing their programs and achieving key milestones [44] - The management team highlighted the importance of identifying and bringing in novel molecules to deepen the company's portfolio [45] Other Important Information - The company expects R&D expenses for Q2 2021 to be between $30 million and $35 million, with total operating expenses between $35 million and $40 million [39] - Full-year 2021 guidance remains unchanged, with R&D expenses expected to be between $90 million and $100 million [40] Q&A Session Summary Question: Clarification on NPM1 patients and complete hematologic recovery - One of the two NPM1 patients had a complete response and full hematologic recovery, while no further information was provided on the second patient [48] Question: Goals for the new cohort in the dose escalation study - The cobicistat arm is exploratory and does not affect the doses moving into Phase 2 [49] Question: Concerns about the therapeutic window of SNDX-5613 - The recommended Phase 2 dose meets pre-specified criteria for efficacy, and the response rates are consistent with the overall population [51] Question: Specific goals of the interim dose cohort - The interim dose cohort is not a gating item for moving into Phase 2, but results will be evaluated before proceeding [54] Question: Protocol for stem cell transplants in the study - There is no formal protocol for stem cell transplants; it is based on the investigator's decision after achieving a complete response [59] Question: Effect of SNDX-5613 on ALL patients - Qualitatively, the effect appears similar in ALL as in AML, but the numbers are small [77] Question: Response rate for NPM1 patients compared to current standards - The response rate for relapsed refractory acute leukemia is generally low with chemotherapy, making the observed rates with SNDX-5613 significant [78]