- 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 -
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.
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:
https://www.veracast.com/webcasts/syndax/events/SNDX1Q24.cfm
www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.
About Revumenib
Revumenib is a potent, selective, small molecule inhibitor of the menin-KMT2A binding interaction that is being developed for the treatment of KMT2A-rearranged (KMT2Ar), also known as mixed lineage leukemia rearranged or MLLr, acute leukemias including ALL and AML, and mutant nucleophosmin (mNPM1) AML. Positive topline results from the Phase 2 AUGMENT-101 trial in R/R KMT2Ar acute leukemia showing the trial met its primary endpoint were presented at the 65th American Society of Hematology Annual Meeting, and data from the Phase 1 portion of AUGMENT-101 in acute leukemia was published in Nature. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation. Revumenib was granted Breakthrough Therapy Designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement.
About Axatilimab
Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and IPF. Positive topline results from the Phase 2 AGAVE-201 trial showing the trial met its primary endpoint were recently presented at the 65th American Society of Hematology Annual Meeting, and Phase 1/2 data of axatilimab in chronic GVHD were published in the Journal of Clinical Oncology. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Syndax has exercised its option under the collaboration agreement to co-commercialize axatilimab in the U.S. and will provide 30% of the commercial effort. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.
About the Real-Time Oncology Review Program (RTOR)
https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program
About Syndax
www.syndax.com or follow the Company on X (formerly Twitter) and LinkedIn.
Forward-Looking Statements
Syndax Contact
[email protected]
Tel 781.684.9827
SNDX-G
SYNDAX PHARMACEUTICALS, INC. | ||||||||
(unaudited) | ||||||||
CONDENSED CONSOLIDATED BALANCE SHEETS | ||||||||
March 31, | December 31, | |||||||
(In thousands) | 2024 | 2023 | ||||||
Cash, cash equivalents, short and long-term investments | $ | 521,994 | $ | 600,527 | ||||
Total assets | $ | 543,028 | $ | 612,880 | ||||
Total liabilities | $ | 51,139 | $ | 58,684 | ||||
Total stockholders' equity | $ | 491,889 | $ | 554,196 | ||||
Common stock outstanding | 84,979,686 | 84,826,632 | ||||||
Common stock and common stock equivalents* | 98,658,929 | 96,316,640 | ||||||
*Common stock and common stock equivalents: | ||||||||
Common stock | 84,979,686 | 84,826,632 | ||||||
Common stock warrants (pre-funded) | 285,714 | 285,714 | ||||||
Common stock and pre-funded stock warrants | 85,265,400 | 85,112,346 | ||||||
Options to purchase common stock | 11,872,530 | 10,684,858 | ||||||
Restricted Stock Units | 1,520,999 | 519,436 | ||||||
Total common stock and common stock equivalents | 98,658,929 | 96,316,640 | ||||||
SYNDAX PHARMACEUTICALS, INC. | ||||||||
(unaudited) | ||||||||
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||
Three Months Ended March 31, | ||||||||
(In thousands, except share and per share data) | 2024 | 2023 | ||||||
Operating expenses: | ||||||||
Research and development | $ | 56,492 | $ | 34,054 | ||||
Selling, general and administrative | 23,022 | 11,961 | ||||||
Total operating expenses | 79,514 | 46,015 | ||||||
Loss from operations | (79,514) | (46,015) | ||||||
Other income (expense), net | 7,114 | 4,889 | ||||||
Net loss | $ | (72,400) | $ | (41,126) | ||||
Net loss attributable to common stockholders | $ | (72,400) | $ | (41,126) | ||||
Net loss per share attributable to common | ||||||||
stockholders--basic and diluted | $ | (0.85) | $ | (0.59) | ||||
Weighted-average number of common stock | ||||||||
used to compute net loss per share attributable | ||||||||
to common stockholders--basic and diluted | 85,213,200 | 69,438,890 | ||||||
SOURCE Syndax Pharmaceuticals, Inc.
