On November 2, 2020 Syndax Pharmaceuticals, Inc. ("Syndax," the "Company" or "we") (Nasdaq:SNDX), a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies, reported its financial results for the third quarter ended September 30, 2020. In addition, the Company provided a clinical and business update (Press release, Syndax, NOV 2, 2020, View Source [SID1234569690]).

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"We continue to make exciting progress in the Phase 1 portion of the AUGMENT-101 trial of SNDX-5613, our highly selective, potent, oral menin inhibitor, in adult and pediatric patients with acute leukemias that harbor MLL-r and NPM1 genetic alterations," said Briggs W. Morrison, M.D., Chief Executive Officer of Syndax. "We are highly encouraged by initial data from this study, which demonstrated clear clinical activity in this difficult to treat population of patients with genetically defined acute leukemias. We remain on track to present Phase 1 data from AUGMENT-101 and commence the Phase 2 portion in early 2021."

Dr. Morrison added, "In addition, following recent interactions with the FDA, we are excited to take the next step of initiating a pivotal trial for axatilimab, our anti-CSF-1R monoclonal antibody, in patients with cGVHD which we expect by the end of this year. We are encouraged by the clinical activity and overall safety we’ve seen in the ongoing trial, and firmly believe axatilimab has the potential to serve as an effective intervention for patients with cGVHD. We look forward to sharing updated results from the Phase 1 trial during an oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting in December."

Pipeline Updates

SNDX-5613

In August 2020, the Company enacted the following enhancements to the Phase 1 portion of the AUGMENT-101 trial in patients with MLL-r and NPM1 mutant acute leukemias: focusing enrollment exclusively on patients with mixed lineage leukemia rearranged (MLL-r) and nucleophosmin (NPM1) mutant acute leukemias; backfilling any dose escalation cohort up to a total of 12 patients if efficacy has been observed at that dose level; and expansion of enrollment to include pediatric patients over 30 days old. These enhancements were supported by initial clinical data, as well as insights from emerging data in the pediatric compassionate use setting. Enrollment in the amended Phase 1 portion remains ongoing, with a data presentation expected in early 2021. In early 2021, the Company also anticipates commencing the Phase 2 portion of AUGMENT-101, which it believes could serve as the basis for registration. SNDX-5613 was previously granted Orphan Drug Designation for the treatment of adult and pediatric acute myeloid leukemia by the U.S. Food and Drug Administration (FDA).
Axatilimab

The Company reported that following its End-of-Phase 1 meeting with the FDA, it has aligned on a regulatory path for axatilimab, its anti-CSF-1R monoclonal antibody, for the treatment of chronic graft versus host disease (cGVHD). The Company plans to commence a pivotal Phase 2 trial, AGAVE-201, to assess the safety and efficacy of different doses and schedules of axatilimab for the treatment of patients with cGVHD. The primary endpoint will assess objective response rate based on the 2014 NIH consensus criteria for GVHD with key secondary endpoints including duration of response and improvement in modified Lee Symptom Scale score. The Company expects to begin enrollment by year-end, with topline data anticipated in 2023.
Enrollment remains ongoing in the Phase 2 portion of the Phase 1/2 trial evaluating axatilimab for the treatment of patients with cGVHD. In previously announced preliminary data from the Phase 1 portion of the trial, axatilimab demonstrated compelling clinical activity and a well-tolerated safety profile. The Company will present updated data from the Phase 1 portion in an oral presentation during the American Society of Hematology (ASH) (Free ASH Whitepaper) Virtual Annual Meeting in December. Abstracts for the meeting, which will be held December 5-8, 2020, will be available on Thursday, November 5, 2020 at 9:00 a.m. ET.
Financial Update and Guidance

As of September 30, 2020, Syndax had cash, cash equivalents and short-term investments of $170.2 million and 44.4 million shares and share equivalents issued and outstanding which included 38.8 million shares of common stock and pre-funded warrants to purchase 5.6 million shares of common stock.

Third quarter 2020 research and development expenses increased to $14.4 million from $9.9 million for the prior year period. The increase was primarily due to increased clinical activity for SNDX-5613 and axatilimab and a $2.0 million milestone payable to UCB upon the achievement of a certain milestone.

General and administrative expenses for the third quarter 2020 increased to $5.8 million from $3.6 million for the prior year period. This increase was primarily due to employee related expenses including a one-time non-cash stock compensation expense.

For the three months ended September 30, 2020, Syndax reported a net loss attributable to common stockholders of $20.4 million or $0.46 per share compared to $12.8 million or $0.41 per share for the prior year period.

Financial Guidance

For the fourth quarter of 2020, research and development expenses are expected to be $15 to $20 million, and total operating expenses are expected to be $20 to $25 million.

Conference Call and Webcast

In connection with the earnings release, Syndax’s management team will host a conference call and live audio webcast at 4:30 p.m. ET today, Monday, November 2, 2020.

The live audio webcast and accompanying slides may be accessed through the Events & Presentations page in the Investors section of the Company’s website at www.syndax.com. Alternatively, the conference call may be accessed through the following:

For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company’s website, www.syndax.com.