– NDA filing for revumenib in R/R KMT2Ar acute leukemia granted Priority Review under RTOR; PDUFA action date set for September 26, 2024 – – BLA filing for axatilimab in chronic GVHD granted Priority Review; PDUFA action date set for August 28, 2024 – – Enrollment completed in AUGMENT-101 mNPM1 cohort; topline data expected in 4Q24 to potentially support sNDA filing in 1H25 – – Company to host conference call today at 8:00 a.m. ET – WALTHAM, Mass., May 8, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (NASDAQ:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today reported its financial results for the quarter ended March 31, 2024, and provided a business update. "With the potential near-term approvals of revumenib and axatilimab in the third quarter as well as pivotal data from the mNPM1 cohort of the AUGMENT-101 trial in the fourth quarter, the Company is on track to have a historic year punctuated by major value-creating milestones," said Michael A. Metzger, Chief Executive Officer. "Syndax is unparalleled as a SMID cap oncology company with the potential launch of two first- and best-in-class agents into multi-billion-dollar markets with the opportunity for expansion beyond their initial indications. We remain keenly focused on laying the foundation and building an experienced team of experts to ensure our successful transition into a commercial organization." Recent Pipeline Progress and Anticipated Milestones Revumenib In March 2024, the Company announced that the FDA had granted Priority Review for the New Drug Application (NDA) filing for revumenib, a potent, selective small molecule menin inhibitor, for the treatment of adult and pediatric relapsed or refractory (R/R) KMT2A-rearranged (KMT2Ar) acute leukemia. The NDA filing is being reviewed under the FDA's Real-Time Oncology Review Program (RTOR) and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2024. RTOR allows for a more efficient review and close engagement between the sponsor and the FDA throughout the submission process, which historically has led to earlier approvals. In March 2024, the Company also announced the completion of enrollment in the AUGMENT-101 pivotal trial cohort of patients with R/R mutant nucleophosmin (mNPM1) acute myeloid leukemia (AML). Topline data is expected in the fourth quarter of 2024 and could support a supplemental NDA (sNDA) filing for revumenib in R/R mNPM1 AML in the first half of 2025. Positive results from a subset of the pivotal AUGMENT-101 trial in pediatric patients with R/R KMT2Ar AML and acute lymphoid leukemia (ALL) treated with revumenib were featured in a plenary session at the American Society of Pediatric Hematology/Oncology (ASPHO) Annual Meeting in April 2024. Multiple Phase 1 combination trials of revumenib in mNPM1 and KMT2Ar acute leukemias are ongoing across the treatment landscape. The trials are expanding to validate recommended Phase 2 doses, with additional data expected in the second half of 2024. These trials include: BEAT AML: Evaluating the combination of revumenib with venetoclax and Azacytidine in front-line AML patients. This trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. SAVE: Evaluating the all-oral combination of revumenib with venetoclax and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias. The trial is being conducted by investigators from the MD Anderson Cancer Center. AUGMENT-102: Evaluating the combination of revumenib with fludarabine and cytarabine in patients with R/R acute leukemias. A Phase 1 trial of revumenib in combination with 7+3 chemotherapy followed by maintenance treatment in newly diagnosed patients with mNPM1 or KMT2Ar acute leukemias was initiated during the quarter. The Company plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2Ar acute leukemia patients unfit to receive intensive chemotherapy by year-end 2024. Enrollment is ongoing in a Phase 1 proof-of-concept clinical trial of revumenib in patients with unresectable metastatic microsatellite stable colorectal cancer. The Company expects to provide an update on the trial in the second quarter of 2024.  Axatilimab In February, the Company announced that the FDA had accepted the Biologics License Application (BLA) filing for axatilimab, an anti-CSF-1R antibody, in patients with chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy. The application was granted Priority Review and assigned a PDUFA action date of August 28, 2024. Enrollment is ongoing in a 26-week randomized, double-blinded, placebo-controlled Phase 2 trial of axatilimab on top of standard of care in patients with idiopathic pulmonary fibrosis (IPF). Our partner Incyte plans to initiate two combination trials with axatilimab in chronic GVHD in 2024, including a Phase 2 combination trial with ruxolitinib and a Phase 3 combination trial with steroids. Corporate Updates In March 2024, the Company announced the appointment of Steven Closter as Chief Commercial Officer. Mr. Closter brings to Syndax more than 30 years of commercial experience in the biopharmaceutical industry. First Quarter 2024 Financial Results As of March 31, 2024, Syndax had cash, cash equivalents, and short and long-term investments of $522.0 million and 85.3 million common shares and prefunded warrants outstanding. First quarter 2024 research and development expenses increased to $56.5 million from $34.1 million for the comparable prior year period. The increase in research and development expenses was primarily due to increased clinical development and manufacturing costs, increased employee-related expenses and professional fees, and development milestone expense recognized in the current period.  First quarter 2024 selling, general and administrative expenses increased to $23.0 million from $12.0 million for the comparable prior year period. The increase in selling, general and administrative expenses was primarily due to increased employee-related expenses and professional fees as well as increased commercialization activities for revumenib and axatilimab. For the three months ended March 31, 2024, Syndax reported a net loss attributable to common stockholders of $72.4 million, or $0.85 per share, compared to a net loss attributable to common stockholders of $41.1 million, or $0.59 per share, for the comparable prior year period. Financial Guidance For the second quarter of 2024, the Company expects research and development expenses to be $50 to $55 million and total operating expenses to be $80 to $85 million. For the full year of 2024, the Company continues to expect research and development expenses to be $240 to $260 million and total operating expenses to be $355 to $375 million, which includes an estimated $43 million in non-cash stock compensation expense. The Company believes that it has sufficient cash runway to fund its research, clinical development and commercial operations through 2026. Conference Call and Webcast In connection with the earnings release, Syndax's management team will host a conference call and live audio webcast at 8:00 a.m. ET today, Wednesday, May 8, 2024. The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company's website. Alternatively, the conference call may be accessed through the following: Conference ID: Syndax1Q24Domestic Dial-in Number: 800-590-8290International Dial-in Number: 240-690-8800Live webcast: https://www.veracast.com/webcasts/syndax/events/SNDX1Q24.cfm For ...