Syndax(SNDX) - 2025 Q2 - Quarterly Results

Executive Summary Q2 2025 Highlights The company reported strong Q2 2025 results driven by significant sales momentum for Revuforj and Niktimvo Q2 2025 Key Financial Highlights | Metric | Value | | :----- | :---- | | Revuforj Net Revenue | $28.6 million | | Revuforj QoQ Growth | 43% | | Niktimvo Net Revenue (Incyte reported) | $36.2 million | | Syndax Collaboration Revenue (Niktimvo) | $9.4 million | | Cash, Cash Equivalents & Investments | $517.9 million | - The company expects to fund operations to profitability with current cash, anticipated product revenue, and interest income[3][4] - Revuforj's sNDA for R/R mNPM1 AML was granted Priority Review by the FDA, with a PDUFA action date of October 25, 2025[3][4] Recent Business Highlights and Anticipated Milestones Revuforj® (revumenib) Updates Revuforj demonstrated strong commercial growth and regulatory progress while advancing multiple clinical trials Commercial Performance & Regulatory Milestones Revuforj achieved substantial net revenue growth and its sNDA for R/R mNPM1 AML received Priority Review Revuforj Net Revenue | Period | Net Revenue | | :----- | :---------- | | Q2 2025 | $28.6 million | | Q1 2025 | (Implied $19.99 million based on 43% growth) | | QoQ Growth | 43% | - The U.S. FDA granted Priority Review to the sNDA for Revuforj for the treatment of R/R mutant NPM1 (mNPM1) acute myeloid leukemia (AML), with a PDUFA target action date of October 25, 2025[4][5] Clinical Trial Data & Ongoing Studies New data showed positive response rates, and multiple trials are ongoing to evaluate revumenib in various settings - Additional results from the pivotal Phase 2 AUGMENT-101 trial in R/R mNPM1 AML showed an Overall Response Rate (ORR) of 48% and a Complete Remission (CR) plus CRh rate of 26%, with 63% of CR/CRh patients being MRD negative[5] - Ongoing trials for revumenib include EVOLVE-2, BEAT AML (showing 67% CR rate), and SAVE (showing 82% ORR), with data from a Phase 1 trial with intensive chemotherapy expected in Q4 2025[6] - Two REVEAL trials evaluating revumenib in combination with standard of care are expected to initiate in Q4 2025, with data from a trial in metastatic MSS colorectal cancer (CRC) expected by year-end 2025[8] Niktimvo™ (axatilimab-csfr) Updates Niktimvo achieved significant launch revenue and is progressing in clinical trials for chronic GVHD and IPF Commercial Performance & Collaboration Niktimvo generated significant net revenue in its first full quarter, resulting in substantial collaboration revenue Niktimvo Net Revenue & Syndax Collaboration | Metric | Q2 2025 Value | | :----- | :------------ | | Niktimvo Net Revenue (Incyte) | $36.2 million | | Niktimvo Q1 2025 Net Revenue (Incyte) | $13.6 million | | Syndax Collaboration Revenue | $9.4 million | - Niktimvo was launched in the U.S. in late January 2025 for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy[8] Clinical Trial Data & Ongoing Studies Pivotal trial data highlighted robust responses, with ongoing combination trials and a Phase 2 IPF study - Data from the pivotal AGAVE-201 trial presented in June 2025 highlighted robust and rapid responses in patients with chronic GVHD[8] - Two trials evaluating axatilimab in combination with standard of care therapies in newly diagnosed chronic GVHD patients are ongoing[12] - Enrollment is ongoing in the MAXPIRe Phase 2 trial in idiopathic pulmonary fibrosis (IPF), with completion expected in Q4 2025 and topline data anticipated in H2 2026[12] Corporate Updates Management Changes The company appointed a new Head of R&D and Chief Medical Officer, and a board member resigned - Dr. Nick Botwood was appointed as Head of Research and Development and Chief Medical Officer in May 2025[12] - William Meury resigned from the Board of Directors in June 2025 to assume the role of President and CEO at Incyte, the company's collaboration partner[12] Second Quarter 2025 Financial Results Balance Sheet Highlights The company maintained a strong cash position of over $500 million as of June 30, 2025 Balance Sheet Highlights (as of June 30, 2025) | Metric | Value | | :----- | :---- | | Cash, cash equivalents, and short and long-term investments | $517.9 million | | Common shares and prefunded warrants outstanding | 86.3 million | Statement of Operations Highlights Q2 2025 total revenue reached $38.0 million, driven by product and collaboration revenue Q2 2025 Total Revenue Breakdown | Revenue Type | Amount | | :----------- | :----- | | Total Revenue | $38.0 million | | Revuforj Net Product Revenue | $28.6 million | | Niktimvo Collaboration Revenue | $9.4 million | Operating Expenses Operating expenses increased year-over-year due to clinical trial activities and commercial launch support Operating Expenses (Three Months Ended June 30) | Expense Type | 2025 (in thousands) | 2024 (in thousands) | Change (YoY) | | :----------- | :------------------ | :------------------ | :----------- | | Research and development | $62,227 | $48,655 | +$13,572 | | Selling, general and administrative | $43,805 | $29,061 | +$14,744 | - The increase in R&D expenses was primarily due to multiple revumenib trials, a milestone payment to AbbVie, and medical affairs activities supporting Revuforj[13] - The increase in SG&A expenses was primarily due to higher employee-related expenses and professional fees to support the U.S. commercial launch of Revuforj[14] Net Loss The company reported an increased net loss of $71.8 million for Q2 2025 compared to the prior year Net Loss Attributable to Common Stockholders (Three Months Ended June 30) | Metric | 2025 | 2024 | | :----- | :--- | :--- | | Net Loss | $(71.8) million | $(68.1) million | | Net Loss Per Share | $(0.83) | $(0.80) | Financial Guidance Expense Outlook & Profitability The company maintained its full-year expense guidance and expects to reach profitability with current cash Operating Expense Guidance (Excluding Non-Cash Stock Compensation) | Period | Expected Range | | :----- | :------------- | | Q3 2025 (R&D + SG&A) | $95 to $100 million | | Full Year 2025 (R&D + SG&A) | $370 to $390 million | - The full year 2025 guidance of $370 to $390 million (excluding non-cash stock compensation) represents no change to prior guidance[16] - Syndax expects its operating expense base to remain stable over the next few years, enabling the company to reach profitability with its cash, product revenue, and interest income[17] Conference Call and Webcast Product Information About Revuforj® (revumenib) Revuforj is a first-in-class menin inhibitor with FDA approval and multiple regulatory designations - Revuforj (revumenib) is an oral, first-in-class menin inhibitor, FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation[20] - The FDA granted Priority Review to Syndax's sNDA for revumenib for the treatment of R/R mNPM1 AML, reviewed under the Real-Time Oncology Review (RTOR) program[21] - Revumenib has received Orphan Drug Designation, Fast Track designation, and Breakthrough Therapy Designation for various acute leukemia indications[23][24] About Niktimvo™ (axatilimab-csfr) Niktimvo is a first-in-class CSF-1R antibody for chronic GVHD, co-developed with Incyte - Niktimvo (axatilimab-csfr) is a first-in-class CSF-1R-blocking antibody approved for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy[25] - Syndax holds an exclusive worldwide co-development and co-commercialization license agreement with Incyte for axatilimab[26] About the Real-Time Oncology Review Program (RTOR) The RTOR program is an FDA initiative designed to expedite the review of oncology drugs - RTOR provides a more efficient review process for oncology drugs, allowing for early availability of safe and effective treatments through close engagement with the applicant[28] About Syndax Company Overview Syndax is a commercial-stage biopharmaceutical company focused on advancing innovative cancer therapies - Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company with FDA-approved Revuforj (menin inhibitor) and Niktimvo (CSF-1 receptor blocking antibody)[29][30] - The company is conducting several clinical trials to unlock the full potential of its pipeline[30] Forward-Looking Statements Additional Information Financial Statements (Unaudited) Condensed Consolidated Balance Sheets The balance sheets show key financial positions as of June 30, 2025, compared to December 31, 2024 Condensed Consolidated Balance Sheets (Unaudited, In thousands) | Metric | June 30, 2025 | December 31, 2024 | | :----- | :------------ | :---------------- | | Cash, cash equivalents, short and long-term investments | $517,860 | $692,404 | | Total assets | $596,149 | $724,816 | | Total liabilities | $438,725 | $436,692 | | Total stockholders' equity | $157,424 | $288,124 | | Common stock outstanding | 86,059,077 | 85,694,443 | Condensed Consolidated Statements of Operations The statements of operations show increased revenue and a higher net loss for the three and six-month periods Condensed Consolidated Statements of Operations (Unaudited, In thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :----- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Product revenue, net | $28,600 | — | $48,642 | — | | Collaboration revenue, net | $9,358 | — | $9,111 | — | | Milestone and license revenue | — | $3,500 | — | $3,500 | | Total revenue | $37,958 | $3,500 | $57,753 | $3,500 | | Total operating expenses | $107,311 | $77,716 | $210,863 | $157,230 | | Net loss | $(71,847) | $(68,063) | $(156,693) | $(140,463) | | Basic loss per share | $(0.83) | $(0.80) | $(1.82) | $(1.65) |