Syndax Pharmaceuticals, Inc. · NASDAQ:SNDX
Syndax Pharmaceuticals — Investor Day (December 2025)
53 slides from Syndax Pharmaceuticals's investor day presentation, Dec 8, 2025
Slide 1: Syndax Investor Meeting, American Society of Hematology Annual Meeting
Syndax Investor Meeting American Society of Hematology Annual Meeting December 8, 2025
Slide 2: Forward-looking statements disclosure
Forward-looking statements disclosure This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "plan," "anticipate" and similar expressions (as well as other words or expressions referenci
Slide 3: Welcome & Opening Remarks
Welcome & Opening Remarks Michael Metzger Chief Executive Officer, Syndax
Slide 4: Agenda
Agenda Welcome & Opening Remarks Michael Metzger, CEO Niktimvo (axatilimab-csfr) Latest R/R Chronic GVHD Data Zachariah DeFilipp, M.D., Director of BMT Clinical Research, Massachusetts General Hospital Panelist Discussion and Audience Q&A Revuforj® (revumenib) Acute Leukemia Overview & Real-World Ev
Slide 5: Syndax is a commercial-stage oncology company on the road to profitability
Two first- & Syndax is a commercial- best-in-class drugs $5B+ TAM stage oncology company on the road to profitability with two drugs with multi- billion-dollar potential Two exceptional $5B+ TAM product launches 5
Slide 6: Strong Syndax presence at ASH 2025 highlights leadership in menin and CSF-1R inhibition
Strong Syndax presence at ASH 2025 highlights leadership in menin and CSF-1R inhibition 23 presentations by Syndax & collaborators 3 oral presentations 9 poster presentations 3 oral presentations 8 poster presentations 100s of engagements with HCPs Syndax-supported 2 CME events 1 product theater 6
Slide 7: Revuforj is positioned for long-term growth & success in menin inhibition
Revuforj is positioned for long-term growth & success in menin inhibition BEST-IN-CLASS PROFILE ➢ Unmatched efficacy across multiple pt subtypes ➢ Well-tolerated with clear, flexible dosing ➢ Can be used concomitantly with other commonly used drugs, including gastric acid reducing agents First and o
Slide 8: Growing Revuforj demand underscores exceptional product profile
Growing Revuforj demand underscores exceptional product profile On track to treat 1,000 Cumulative net revenue since launch KMT2A pts by YE 2025 $88M 3Q25 metrics: $56M • 25% growth in total Building usage observed prescriptions & new post-HSCT in KMT2A pts patient starts over 2Q25 $28M • ~750 new p
Slide 9: Niktimvo is poised to deliver on the promise of CSF-1R inhibition in chronic GVHD
Niktimvo is poised to deliver on the promise of CSF-1R inhibition in chronic GVHD and beyond POSITIONED FOR SUCCESS ➢ Novel mechanism in chronic GVHD to address inflammation and fibrosis ➢ Responses were rapid, durable & observed across all organs studied in AGAVE-201 First and only CSF-1R-blocking
Slide 10: Strong Niktimvo results highlight the importance of this novel medicine
Strong Niktimvo results highlight the importance of this novel medicine to patients and Syndax Profitable to Syndax in first full quarter (proportion of net Cumulative net revenue since launch revenue retained by Syndax (reported by Incyte) expected to materially grow $96M 3Q25 metrics: over time) •
Slide 11: Niktimvo in Recurrent/Refractory Chronic GVHD
Niktimvo in Recurrent/Refractory Chronic GVHD Zachariah DeFilipp, M.D. Director of BMT Clinical Research, Massachusetts General Hospital; Associate Professor of Medicine, Harvard Medical School
Slide 12: Chronic GVHD is an area of high unmet need
Chronic GVHD (cGVHD) is an area Niktimvo provides a new mechanism of of high unmet need action for addressing cGVHD • Occurs in ~50% of pts who receive an allo-HSCT • Affects multiple organs with inflammatory & fibrotic pathology; many organs respond poorly to traditional SOC therapies • Major cause
Slide 13: AGAVE-201 data provide evidence for less frequent dosing schedule
Data from AGAVE-201 trial of axatilimab in R/R cGVHD provide evidence of the feasibility of a less frequent dosing schedule Overall response rates with axatilimab 80 Key observations: • 19 pts transitioned from axatilimab 0.3 mg/kg Q2W 60 (FDA approved dose) to 0.6 mg/kg Q4W dosing Response rate, %
Slide 14: Transition to 0.6mg/kg Q4W axatilimab dosing was well tolerated in AGAVE-201
Transition to 0.6mg/kg Q4W axatilimab dosing in R/R cGVHD was well tolerated in the AGAVE-201 trial 0.3 mg/kg Q2W 0.3 mg/kg Q2W to Most common to 0.6 mg/kg Q4W (n=19) Grade≥3 and 0.6 mg/kg Q4W (n=19) Similar safety profile TEAEs1, n (%) Before After serious TEAEs, n (%) Before After before and after
Slide 15: Follow-up data from AGAVE-201 highlight potential for long-term benefit
Follow-up data from AGAVE-201 trial of axatilimab in R/R cGVHD highlight potential for long-term benefit Poster with additional data will be presented on Dec 8 at 6pm 205 discontinued Tx Of the treated pts, 14% have 241 pts enrolled (86%) continued axatilimab with a 239 received As of March (across
Slide 16: Interim data from Ph 2 trial of axatilimab + ruxolitinib in 1L cGVHD
Interim data from Ph 2 trial of axatilimab + ruxolitinib in 1L cGVHD highlights feasibility of potential steroid-sparing regimen Poster with additional data will be presented on Dec 8 at 6pm Phase 2 trial design Pre-specified interim safety analysis Population: 1:1:1 Axatilimab + • 44 pts enrolled;
Slide 17: Enrollment ongoing in Ph 3 trial of axatilimab + corticosteroids in 1L cGVHD
Enrollment ongoing in Ph 3 trial of axatilimab + corticosteroids in 1L cGVHD AXemplify-357 trial design Global recruitment underway Treatment for ≤24 months 1:1 Key eligibility criteria Axatilimab + • ≥12 years corticosteroids • New-onset moderate to severe cGVHD 56 27 SITES 13 SITES (N ≈ 240) Corti
Slide 18: Conclusions
Conclusions • Transition to axatilimab 0.6 mg/kg Q4W was feasible and well tolerated in R/R cGVHD patients treated with 0.3 mg/kg Q2W in the AGAVE-201 trial • Patients had a prolonged duration of therapy, with a median of 1.7 years, and 84% of patients continued treatment, suggesting ongoing clinica
Slide 19: Niktimvo Panelist Discussion & Audience Q&A
Niktimvo Panelist Discussion & Audience Q&A
Slide 20: Acute Leukemia Treatment Paradigm & First Revuforj Real-World Evidence from Moffitt
Acute Leukemia Treatment Paradigm & First Revuforj Real-World Evidence from Moffitt Cancer Center David Sallman, M.D. Associate Member in the Department of Malignant Hematology at Moffitt Cancer Center
Slide 21: Acute myeloid leukemia (AML) treatment paradigm
Acute myeloid leukemia (AML) treatment paradigm TREATMENT GOALS VARY HSCT HSCT if indicated if eligible Frontline: RELAPSED/ FRONTLINE (1L) MAINTENANCE MAINTENANCE • In KMT2Ar, HSCT is REFRACTORY (R/R) generally the goal “Fit for IC” • In NPM1m, multiple factors impact the Intensive Chemo (IC) Targe
Slide 22: Treatment response criteria in AML
Treatment response criteria in AML: Tumor clearance equivalent across CRc and MLFS Response Tumor Platelets recovered Neutrophils recovered ORR CRc CR/CRh CR < 5% Yes Yes CRh < 5% Half normal levels Half normal levels CRp < 5% No Yes CRi < 5% Either has recovered MLFS < 5% Neither has recovered 5-25
Slide 23: First Revuforj real-world evidence highlights usage across multiple genetic subtypes
First Revuforj real-world evidence highlights usage across multiple genetic subtypes and settings Patient characteristics (N=17) Age, median (range), years 54 (23-79) Female, n (%) 7 (41) First RWE from Moffitt Cancer Center shows use in: 8 (47%) KMT2Ar*, 5 (29%) Molecular subtypes, n (%) NPM1m, 3 (
Slide 24: Excellent activity observed with Revuforj in the real-world setting
Excellent activity observed with Revuforj in the real-world setting across lines of therapy and molecular subsets Efficacy data1, n/N (%) ORR 10/13 (77) High rates of remission, MRD CRc (CR + CRh + CRi) 8/13 (62) negativity, and HSCT observed CR + CRh 4/13 (31) among real-world cohort including KMT2
Slide 25: Responses were rapid and durable with Revuforj in the real-world setting
Responses were rapid and durable with Revuforj in the real-world setting, even in heavily pretreated KMT2Ar and NPM1m patients Time to response & relapse rate Time to first response, 0.8 (0.5-6.5) months median (range) Time to best response, 2.2 (0.7-6.5) months median (range) 3/15 (20) Relapse1, n/
Slide 26: Revuforj was well tolerated in this real-world cohort
Revuforj was well tolerated in this real-world cohort which predominantly received combination treatment Excellent tolerability Safety data, n/N (%) in real-world setting Any Grade 3 or 4 AE (non-hematological) 4/17 (24) 3/15 (20) Grade ≥3 QTc prolongationa (Grade 3 only) Low rate of 2/17 (12) revum
Slide 27: Revumenib in Post-HSCT Maintenance Setting
Revumenib in Post-HSCT Maintenance Setting Elias Jabbour, M.D. Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center
Slide 28: Retrospective review provides insight into growing use of revumenib maintenance post-HSCT
Retrospective review provides insight into growing use of revumenib maintenance post-HSCT Analysis included 10 pediatric pts treated at MD Anderson Cancer Center who underwent HSCT High unmet need in following response to revumenib-based therapies post-HSCT maintenance Patient Characteristics (N=10)
Slide 29: Encouraging early efficacy in children who received revumenib maintenance post-HSCT
Encouraging early efficacy in retrospective review of children who received revumenib maintenance post-HSCT Age, Molecular Days from Rev maintenance # • Pts restarted revumenib a years subtype HSCT to rev cycles completed median of 111 days post-HSCT 1 3 KMT2Ar 175 3 (range: 58-175) 2 12 KTM2Ar 137
Slide 30: Revumenib maintenance post-HSCT was well tolerated in children with HOX-driven AML
Revumenib maintenance post-HSCT was well tolerated in children with HOX-driven AML Adverse events during revumenib maintenance post-HSCT Most adverse events were low grade and manageable Thromboctyopenia (G3) 3 Neutropenia (G2) 2 Thromboctyopenia (G2) 1 Failure to thrive (G2) 1 No patient discontinu
Slide 31: SAVE Phase 2 Trial of Revumenib with Venetoclax and Decitabine/Cedazuridine
SAVE Phase 2 Trial of Revumenib with Venetoclax and Decitabine/Cedazuridine in Newly Diagnosed AML Elias Jabbour, M.D. Professor, Department of Leukemia, Division of Cancer Medicine, MD Anderson Cancer Center
Slide 32: SAVE, a Phase 1/2 trial of the all-oral combination in R/R and ND AML
SAVE, a Phase 1/2 trial of the all-oral combination of revumenib + venetoclax and decitabine/cedazuridine in R/R and newly diagnosed (ND) AML Revumenib* Objectives DL 0: 110 mg/160 mg +/- CYP3A4i Primary: DL 1: 160 mg/270 mg +/- CYP3A4i (USPI dose) • Age ≥12 years • Phase 1 in R/R (3+3 design): PO Q
Slide 33: SAVE enrolled a cohort of newly diagnosed AML patients with advanced age
SAVE enrolled a cohort of newly diagnosed AML patients with advanced age Characteristic All (N=21) NPM1m (n=14) KMT2Ar (n=7) Median age, years (range) 70 (60-83) 73 (66-83) 64 (60-77) ≥70 years, n (%) 11 (52%) 10 (71%) 1 (14%) Female, n (%) 15 (71%) 9 (64%) 6 (86%) Secondary AML, n (%) 5 (24%) 2 (14
Slide 34: The SAVE combination was generally well-tolerated in newly diagnosed AML
The SAVE combination was generally well-tolerated in newly diagnosed AML TEAEs (any grade, >30% ) (N=21) TEAEs (Grade ≥3, any G4-5) G3 G4 G5 G ≥3 Vomiting 14 (67%) Febrile neutropenia 0 10 (48%) 0 10 (48%) Elevated AST/ALT 13 (62%) Thrombocytopenia 2 (10%) 5 (24%) 0 7 (33%) ↑K+ 13 (62%) Neutropenia
Slide 35: High response & MRD negativity rates observed with SAVE combination
High response & MRD negativity rates observed with SAVE combination in newly diagnosed AML All patients NPM1m KMT2Ar Best Response (N=21) (n=14) (n=7) ORR 18 (86%) 12 (86%) 6 (86%) CR/CRh 17 (81%) 11 (79%) 6 (86%) CR 16 (76%) 10 (71%) 6 (86%) CRh 1 (5%) 1 (7%) 0 CRp 1 (5%) 1 (7%) 0 MLFS 0 0 0 Early
Slide 36: High rates of molecular NPM1m MRD negativity observed in 1L patients
Within a few cycles of treatment with the SAVE combination, high rates of molecular NPM1m MRD negativity were observed in 1L patients 80% of NPM1m patients were MRD (-) Two NPM1m patients who relapsed were MRD (+) in bone marrow by NGS at EOC3+ in bone marrow by NGS NPM1m VAF % MRD response (%) Rela
Slide 37: Encouraging survival observed with SAVE combo in an older 1L population
Encouraging survival observed with SAVE combo in an older 1L population OS and EFS in the entire cohort OS by genotype Median follow-up of 9 months Median follow-up of 9 months mOS: not reached; 12m OS: 57% (95% CI, 36-90) NPM1m mOS: not reached; 12m OS: 53% (95% CI, 29-97) mEFS: not reached; 12m EF
Slide 38: Promising duration of response in 1L patients who received SAVE combo
Promising duration of response in 1L patients who received SAVE combo, with majority of patients remaining on therapy Age (years) FLT3-TKD, BCOR, DNMT3A, TET2, NK, monocytic at dx. Synchronous EMD and BM relapse mid-C8 with CK. EMD + NPM1 cytoplasmic expression by IHC. KMT2A::AFDN t(6;11). Prolonged
Slide 39: Conclusions
Conclusions • In the SAVE trial, the all-oral combination of revumenib + venetoclax and decitabine/cedazuridine was generally well-tolerated in newly diagnosed NPM1m and KMT2Ar AML patients • High response and MRD negativity rates observed with SAVE combination in older adults with newly diagnosed N
Slide 40: Phase 1 Trial of Revumenib with Intensive Chemotherapy in Newly Diagnosed AML
Phase 1 Trial of Revumenib with Intensive Chemotherapy in Newly Diagnosed AML - SNDX-5613-0708 David Swoboda, M.D. Director of Leukemia, Tampa General Hospital
Slide 41: Phase 1 trial of revumenib with intensive chemotherapy (SNDX-5613-0708)
Phase 1 trial of revumenib with intensive chemotherapy (IC) in newly diagnosed patients with NPM1m, KMT2Ar, or NUP98r AML (SNDX-5613-0708) Revumenib + IC induction, IC and/or HSCT consolidation, and post-consolidation revumenib maintenance Dose escalation Dose expansion Study endpoints Revumenib wit
Slide 42: Newly diagnosed NPM1m and KMT2Ar patients enrolled across two dose levels
Newly diagnosed NPM1m and KMT2Ar patients were enrolled across two dose levels of revumenib + IC DL1 DL2 Total (n = 13) (n = 17) (N = 30) Age, median (range), years 43 (20-71) 57 (19-68) 49 (19-71) Key differences Female, n (%) 8 (61.5) 14 (82.4) 22 (73.3) between DL1 & DL2: Race, n (%) White 9 (69.
Slide 43: Both revumenib dose levels were generally well-tolerated with IC
Both revumenib dose levels were generally well-tolerated in combination with IC in newly diagnosed NPM1m and KMT2Ar AML DL1 (n = 13) DL2 (n = 17) Total (N = 30) Any Grade Any Grade Any Grade Parameter, n (%) Key observations: grade ≥3 grade ≥3 grade ≥3 Any AE 12 (92.3) 11 (84.6) 15 (88.2) 14 (82.4)
Slide 44: Rapid count recovery observed in responders who received revumenib + IC
Rapid count recovery observed in responders who received revumenib + IC in the 1L setting NPM1m + KMT2Ar AML with CRc Median (range), Cycle 1 DL1 (n = 12) DL2 (n = 12) Days to neutrophil recovery (≥1,000 cells/μL) 29 (19-35) 29 (22-43) Days to platelet recovery (≥100,000 cells/μL) 28 (22-31) 29 (22-
Slide 45: Promising early results among NPM1m and KMT2Ar patients who received revumenib + IC
Promising early results observed among both NPM1m and KMT2Ar patients who received revumenib + IC in 1L setting Key observations: • Follow-up time was shorter in DL2 than DL1 o 5/14 pts still on treatment in DL2 had <21 days of revumenib Tx • Treatment was ongoing in 77% (10/13) and 82% (14/17) of p
Slide 46: Deep responses observed among NPM1m and KMT2Ar patients in 1L setting
Deep responses observed among NPM1m and KMT2Ar pts who received revumenib + IC in 1L setting Efficacy among response- DL1 DL2 Total evaluable population (n = 12) (n = 14) (N = 26) Responses in DL2 could ORR, n (%) 12 (100) 13 (92.9) 25 (96.2) further deepen as data 95% CI 73.5–100 66.1-99.8 80.4-99.
Slide 47: Conclusions
Conclusions • Overall safety profile of revumenib + IC is comparable to IC alone; no new safety signals were identified at either dose level in combination with IC o QTc prolongation was infrequent and managed successfully using established mitigation approaches; no cases of DS were reported • Preli
Slide 48: Developing Revuforj and Niktimvo Into Industry Leading Franchises
Developing Revuforj and Niktimvo Into Industry Leading Franchises Nick Botwood, M.B.B.S Head of Research & Development and Chief Medical Officer, Syndax
Slide 49: Comprehensive Revuforj 1L clinical development plan
Comprehensive Revuforj 1L clinical development plan designed to rapidly deliver practice-changing evidence and support global registrations RELAPSED / FRONTLINE REFRACTORY INELIGIBLE FOR IC (‘UNFIT’) ELIGIBLE FOR IC (‘FIT’) AUGMENT-102 BEAT AML SNDX-708 Rev + FLA in KMT2Ar, NPM1m, Rev + ven/aza in N
Slide 50: Syndax and Incyte are working to advance Niktimvo into earlier lines of cGVHD
Syndax and Incyte are working to advance Niktimvo into earlier lines of cGVHD treatment and other fibrotic diseases, starting with IPF Chronic graft-versus-host disease (cGVHD) RECURRENT/ FRONTLINE REFRACTORY Phase 2 Trial AXemplify-357 Ph 3 Trial AGAVE-201 Ph 2 Trial Axa + ruxolitinib Axa + cortico
Slide 51: Revuforj Panelist Discussion & Audience Q&A
Revuforj Panelist Discussion & Audience Q&A
Slide 52: Fueled by a passion for patients
FUELED BY A PASSION FOR PATIENTS Lilah, diagnosed with R/R AML 52
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