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Syndax Pharmaceuticals, Inc. (NASDAQ:SNDX): Poised for Transformative Growth with Promising Pipeline and Imminent Regulatory Approvals
Published on September 24, 2024 | Ticker: SNDX

Syndax Pharmaceuticals, Inc. (NASDAQ:SNDX) is a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. The company's two lead product candidates, revumenib and axatilimab, have both received priority review designations from the FDA, setting the stage for potential approvals in the third quarter of 2024. With a strong financial position, a differentiated portfolio, and experienced leadership, Syndax is well-positioned to capitalize on the significant market opportunities ahead.

Revumenib: A Breakthrough in Acute Leukemia Treatment

Syndax's lead product candidate, revumenib, is a potent, selective small molecule inhibitor of the menin-KMT2A (also known as MLL) interaction. This interaction is a key driver of certain acute leukemias, including KMT2A-rearranged (KMT2Ar) acute lymphoblastic leukemia (ALL), KMT2Ar acute myeloid leukemia (AML), and nucleophosmin 1 (NPM1) mutant AML. The FDA has granted priority review for revumenib's New Drug Application (NDA) in adult and pediatric patients with relapsed or refractory (R/R) KMT2Ar acute leukemia, with a target action date of September 26, 2024.

The pivotal AUGMENT-101 trial has demonstrated impressive results for revumenib in this patient population. In the KMT2Ar cohort, single-agent revumenib induced a high percentage of blast responses, enabling many patients to undergo potentially curative hematopoietic stem cell transplant. Importantly, responses have been durable, with patients continuing revumenib monotherapy following transplant.

Syndax is also exploring the use of revumenib in the NPM1 mutant AML setting. The company has completed enrollment in the NPM1 cohort of the AUGMENT-101 trial, and topline data is expected in the fourth quarter of 2024. If successful, this could support a supplemental NDA filing for revumenib in R/R NPM1 AML in the first half of 2025.

Beyond the relapsed/refractory indications, Syndax is advancing revumenib in combination trials across the treatment landscape. These include the BEAT-AML trial evaluating revumenib with venetoclax and azacitidine in frontline AML, the SAVE trial evaluating the all-oral combination of revumenib, venetoclax, and decitabine/cedazuridine in R/R AML or mixed phenotype acute leukemias, and the AUGMENT-102 trial evaluating revumenib in combination with fludarabine and cytarabine in patients with R/R acute leukemias. The company also plans to initiate a pivotal trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed KMT2Ar or NPM1 mutant acute leukemia patients unfit for intensive chemotherapy by the end of 2024.

Axatilimab: Addressing Unmet Needs in Chronic Graft-Versus-Host Disease

Syndax's second lead product candidate, axatilimab, is a monoclonal antibody that blocks the colony stimulating factor 1 receptor (CSF-1R). The FDA has granted priority review for axatilimab's Biologics License Application (BLA) in patients with chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy, with a target action date of August 28, 2024.

The pivotal AGAVE-201 trial demonstrated that treatment with single-agent axatilimab led to an overall response rate of 74%, with 60% of responders still in response at one year. Importantly, axatilimab has a differentiated mechanism of action compared to currently approved therapies, targeting inflammation and fibrosis through the inhibition of disease-associated macrophages.

Beyond cGVHD, Syndax is exploring the potential of axatilimab in other fibrotic diseases, such as idiopathic pulmonary fibrosis (IPF). The company is currently enrolling a Phase 2 clinical trial evaluating axatilimab in patients with IPF.

Syndax's partnership with Incyte Corporation for the development and commercialization of axatilimab in the United States and globally further strengthens the company's position in the cGVHD market. Syndax has exercised its option to co-commercialize axatilimab in the U.S., providing 30% of the commercial efforts and sharing in the profits.

Financials

As of March 31, 2023, Syndax had $522 million in cash, cash equivalents, and short- and long-term investments, which the company expects will fund its operations through 2026. This strong financial position allows Syndax to invest in the successful commercialization of revumenib and axatilimab, as well as advance its pipeline and pursue strategic business development opportunities.

For the first quarter of 2023, Syndax reported a net loss of $72.4 million and no revenue. The company's research and development expenses for the quarter were $56.5 million, while selling, general, and administrative expenses were $23.0 million. Syndax's management provided guidance for the full year 2023, expecting research and development expenses to be $240 million to $260 million and total operating expenses to be $355 million to $375 million.

Business Overview

Syndax was founded in 2005 and is headquartered in Waltham, Massachusetts. The company's focus is on developing innovative therapies for the treatment of cancer. Syndax's strategy is to in-license and develop promising drug candidates with differentiated profiles, leveraging the technical and business expertise of its management team and scientific collaborators.

The company's pipeline includes two late-stage product candidates, revumenib and axatilimab, both of which have received priority review designations from the FDA. Revumenib is being developed for the treatment of acute leukemias, while axatilimab is targeting chronic graft-versus-host disease and other fibrotic diseases.

Syndax has established strategic collaborations to advance the development of its product candidates. In 2021, the company entered into a collaboration agreement with Incyte Corporation to further develop and commercialize axatilimab. Syndax also has licensing agreements with UCB Biopharma Sprl for axatilimab and with Vitae Pharmaceuticals, Inc. (a subsidiary of AbbVie Inc.) for revumenib.

Risks and Challenges

While Syndax's pipeline holds significant promise, the company faces several risks and challenges common to the biopharmaceutical industry. These include the inherent uncertainty of clinical development, the ability to obtain regulatory approvals, the successful commercialization of its product candidates, and the need to continue expanding its pipeline through in-licensing or acquisition of additional product candidates.

Additionally, Syndax's reliance on third-party manufacturers and the potential for supply chain disruptions, as well as the competitive landscape in the target indications, could impact the company's ability to achieve its goals. Intellectual property protection and the potential for generic competition also pose risks to Syndax's long-term success.

Conclusion

Syndax Pharmaceuticals is poised for a transformative year in 2024 with the potential approvals of revumenib and axatilimab. The company's differentiated pipeline, strong financial position, and experienced management team position it well to capitalize on the significant market opportunities in acute leukemias and chronic graft-versus-host disease. As Syndax transitions into a commercial-stage organization, investors will be closely watching the successful launches of these two first-in-class therapies, which could drive substantial long-term value for the company and its shareholders.

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