Syndax(US:SNDX)2021-08-10 02:49Financial Data and Key Metrics Changes - The net loss for Q2 2021 was $22.9 million, or $0.44 per share, compared to a net loss of $17.1 million, or $0.42 per share, for the same period last year [35] - The net loss was below the guidance of $35 million to $40 million due to the shifting of timing for some CMC activities [35] - The company ended Q2 2021 with $253.1 million in cash and cash equivalents, providing a cash runway into 2023 [36] Business Line Data and Key Metrics Changes - The Phase I trial of SNDX-5613 has shown a 48% overall response rate and a 23% CR/CRh rate in patients with relapsed or refractory acute leukemia [15][16] - Axatilimab is in a pivotal trial (AGAVE-201) for chronic graft versus host disease, with enrollment ongoing and top-line data expected in 2023 [28][29] 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 [29] - The company is exploring the use of SNDX-5613 in combination with venetoclax and azacitidine for newly diagnosed AML patients [22] Company Strategy and Development Direction - The company aims to be first to market in relapsed/refractory disease and to expand the 5613 franchise into additional indications [21] - There is a focus on obtaining regulatory approval for drugs that extend and improve the lives of patients with cancer and other diseases [40][41] - The company plans to continue expanding its pipeline through product acquisitions or in-licensing of differentiated assets [32] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the ongoing registrational programs for both SNDX-5613 and axatilimab, viewing them as major achievements [40] - The management team is confident in their financial resources to advance programs and achieve key milestones [42] Other Important Information - The company has received Fast Track Designation from the FDA for SNDX-5613, with an end of Phase I meeting anticipated [17] - The Phase II portion of the AUGMENT-101 trial will enroll distinct cohorts, including pediatric and adult patients, potentially leading to a broad regulatory approval [20] Q&A Session Summary Question: Would you be willing to disclose the AUC or time above the IC90 for the recommended Phase II doses? - The company will look into this and get back with the information [46][47] Question: Will the updated data at year-end include those phased -- the initial experience from the recommended Phase II doses? - Yes, the updated data will include information from the intermediate doses [47] Question: What clarity do you hope to get from the FDA meeting? - The company seeks agreement on the recommended Phase II dose, endpoints, sample size, and potential registrational status for Phase II expansion cohorts [48] Question: Is it correct to think that there may be an exposure or PK extension benefit for 5613, given that venetoclax is a CYP3A4 inhibitor? - Venetoclax is metabolized by CYP3A4, and the company anticipates dose adjustments for both drugs when used in combination [51] Question: How are you thinking about the evolving landscape for axatilimab, especially with recent approvals? - The company believes axatilimab will provide a complementary mechanism for patients who have received other therapies [54] Question: Could you tell us more about the efforts to minimize QT prolongation? - The company has been working with clinical sites to ensure better electrolyte management, which has shown to reduce QT prolongation [56][57] Question: Could you elaborate on the trial design for the Beat AML trial? - The trial will start with a Phase I to determine the right dose, followed by a randomized trial [59] Question: How many patients do you think you could have as the Phase II pivotal starts? - The company has not specified the number but indicated that backfilling cohorts could count towards the Phase II enrollment [61] Question: What chemotherapies are you looking to combine with in AUGMENT-102? - The company plans to combine with FLAG-based regimens for AML and a 4-drug regimen typically used in ALL [63] Question: Are there other frontline trials you want to run? - The company is exploring various development opportunities, including combinations with standard care treatments [64]