Business Overview and History
Syndax Pharmaceuticals (NASDAQ:SNDX) is a commercial-stage biopharmaceutical company that has emerged as a leader in the oncology space, developing a robust pipeline of innovative cancer therapies. With the recent FDA approvals of its two first-in-class medicines, Syndax has positioned itself as a formidable player in the industry, poised to deliver meaningful value to patients and shareholders alike.
Syndax Pharmaceuticals, Inc. was incorporated in Delaware in 2005 with a mission to develop and commercialize innovative cancer therapies. The company's journey began with the in-licensing of entinostat, an oral, small-molecule inhibitor that targets both cancer cells and immune regulatory cells. Over the years, Syndax has strategically expanded its pipeline, adding key assets such as axatilimab, a first-in-class CSF-1R blocking antibody, and revumenib, a novel menin inhibitor.
In 2011, Syndax established a wholly owned subsidiary in the United Kingdom, which was later dissolved in June 2024. The company then established a wholly owned subsidiary in the United States in 2014 and another in the Netherlands in 2021, though these entities had no material activities to date.
Throughout its history, Syndax has faced several challenges in developing its pipeline of cancer therapies. The company has had to navigate the complex and highly regulated pharmaceutical industry, including the lengthy and costly process of conducting clinical trials and obtaining regulatory approvals. To support its research and development efforts, Syndax has relied on various funding sources, including public stock offerings, revenue from license agreements, and a royalty financing agreement.
In 2016, Syndax entered into a licensing agreement with UCB Biopharma Sprl to acquire the worldwide rights to axatilimab, an asset the company has since advanced through clinical development. In 2017, Syndax in-licensed a portfolio of preclinical, orally available menin inhibitors from Vitae Pharmaceuticals, a subsidiary of AbbVie, further strengthening its oncology pipeline.
The company's strategic focus on developing differentiated cancer therapies has paid off, as evidenced by the recent FDA approvals of Revuforj (revumenib) and Niktimvo (axatilimab-csfr) in 2024. Revuforj was approved for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients, while Niktimvo was approved for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy.
In 2021, Syndax entered into a collaboration and license agreement with Incyte Corporation for the worldwide development and commercialization of axatilimab. Under the terms of this agreement, Incyte received exclusive commercialization rights outside of the United States, while Syndax and Incyte are co-commercializing axatilimab in the U.S.
Financial Overview
Syndax reported net revenue of $23.68 million for the full year 2024, driven by the launch of Revuforj and milestone payments received from its collaboration with Incyte for the approval of Niktimvo. The company's net loss for the year was $318.76 million, reflecting ongoing investment in research and development activities.
For the fourth quarter of 2024, Syndax reported revenue of $7.68 million, primarily from Revuforj sales, and a net loss of $94.17 million. This represents a significant increase in revenue compared to the same quarter in 2023 when the company had no product sales.
Research and development expenses for the year ended December 31, 2024, were $241.65 million, with $107.91 million attributed to revumenib-related costs and $49.64 million to axatilimab-related costs. Selling, general and administrative expenses were $120.88 million, including $33.54 million in commercial-related expenses to support the launch and commercialization of Revuforj and Niktimvo.
As of December 31, 2024, Syndax had $692.4 million in cash, cash equivalents, and short-term and long-term investments, positioning the company to fund its planned operations through profitability. The company's balance sheet was further strengthened in 2024 by a $350 million upfront payment received from Royalty Pharma in exchange for a capped royalty on U.S. net sales of Niktimvo.
Financials
Syndax's financial ratios paint a picture of a company with a strong liquidity position and a focus on long-term growth. The company's current ratio stood at 5.82 as of December 31, 2024, indicating a robust ability to meet short-term obligations. Additionally, Syndax's debt-to-equity ratio of 1.20 suggests a balanced capital structure, with a mix of debt and equity financing to support its operations.
Liquidity
As of December 31, 2024, Syndax had $692.4 million in cash, cash equivalents, and short-term and long-term investments, positioning the company to fund its planned operations through profitability. This strong liquidity position provides Syndax with the financial flexibility to continue advancing its clinical development programs and support the commercial launches of its approved products.
Product Pipeline and Development Milestones
Revuforj (revumenib)
Revuforj, Syndax's first-in-class menin inhibitor, has been a key driver of the company's recent success. The FDA's approval of Revuforj in November 2024 for the treatment of R/R acute leukemia with a KMT2A translocation marked a significant milestone for the company and the broader oncology community.
The approval was based on positive data from the AUGMENT-101 trial, which demonstrated robust and durable rates of remission in the target patient population. In the initial five weeks of the Revuforj launch, the company reported $7.7 million in net product revenue, highlighting the strong demand for the therapy.
Syndax is now focused on expanding the label for Revuforj, with plans to submit a supplemental new drug application (sNDA) in the second quarter of 2025 for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The company is also advancing a robust clinical development strategy to establish Revuforj as the preferred menin inhibitor for the treatment of acute leukemias across the treatment continuum, including in the frontline setting.
Additionally, Syndax is exploring the potential of revumenib in solid tumors, specifically investigating its activity in metastatic colorectal cancer. This expansion into solid tumors represents a significant opportunity to broaden the therapeutic applications of revumenib.
Niktimvo (axatilimab-csfr)
Niktimvo, Syndax's first-in-class CSF-1R blocking antibody, was approved by the FDA in August 2024 for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. The approval was based on data from the AGAVE-201 study, which demonstrated durable responses across all organs studied and patient subgroups.
Syndax is collaborating with Incyte to further develop and commercialize Niktimvo. The companies are advancing multiple trials evaluating Niktimvo in combination with standard-of-care therapies in newly diagnosed cGVHD patients, as well as in the treatment of idiopathic pulmonary fibrosis (IPF).
The commercial launch of Niktimvo in the U.S. market in late January 2025 represents another important milestone for Syndax. The company is confident in Niktimvo's potential to capture a significant portion of the estimated $1.5 billion to $2 billion U.S. market opportunity for third-line or later cGVHD treatment.
Competitive Landscape and Risks
Syndax operates in a highly competitive oncology space, with several established players and emerging competitors vying for market share. The company's approved products, Revuforj and Niktimvo, face competition from other targeted therapies, such as Imbruvica (ibrutinib) and Rezurock (belumosudil), which are approved for the treatment of cGVHD.
Additionally, Syndax's pipeline assets, including revumenib and axatilimab, may face challenges from other investigational therapies in development by companies like AbbVie, Gilead Sciences, and Novartis. The ability of Syndax to maintain its competitive edge will depend on the company's ability to continue advancing its pipeline, securing regulatory approvals, and executing successful commercial strategies.
The biotechnology industry is also inherently risky, with factors such as clinical trial failures, regulatory hurdles, and reimbursement challenges potentially impacting Syndax's operations and financial performance. The company's reliance on strategic collaborations, such as its partnership with Incyte, also introduces the risk of potential delays or disruptions in the development and commercialization of its assets.
Future Outlook and Guidance
Looking ahead to 2025, Syndax has provided financial guidance that reflects its continued investment in research and development and commercial infrastructure. For the first quarter of 2025, the company expects R&D expenses to be between $65 million and $70 million, with total R&D plus SG&A expenses projected to be in the range of $105 million to $110 million.
For the full year 2025, Syndax anticipates R&D expenses to be between $260 million and $280 million, with total R&D plus SG&A expenses expected to be in the range of $415 million to $435 million. This guidance includes an estimated $45 million in non-cash stock compensation expense.
While the company has not provided specific revenue guidance, the projected expenses reflect Syndax's commitment to advancing its clinical pipeline and supporting the commercial launch of its approved products. The substantial cash position of $692.4 million at the end of 2024 provides a strong foundation for these planned investments.
Conclusion
Syndax has demonstrated its ability to navigate the complex oncology landscape, delivering two first-in-class medicines to patients in 2024. The successful launches of Revuforj and Niktimvo, coupled with the company's robust pipeline and strong financial position, position Syndax for continued growth and value creation.
As Syndax looks ahead, the company's focus on expanding the approved indications for its products, advancing its clinical development programs, and exploring strategic partnerships will be key to its long-term success. With a talented management team, a diversified pipeline, and a steadfast commitment to serving the needs of cancer patients, Syndax is poised to cement its position as a leading player in the oncology space.
The company's strong cash position and strategic financial management provide a solid foundation for future growth, while the ongoing clinical development programs offer potential for expanding the therapeutic reach of its innovative products. As Syndax continues to execute its commercial strategy and advance its pipeline, investors and patients alike will be watching closely to see how this emerging leader in oncology therapeutics shapes the future of cancer treatment.