Syndax Pharmaceuticals, Inc. · NASDAQ:SNDX
Syndax Pharmaceuticals — American Society of Hematology Annual Meeting 2024
69 slides from Syndax Pharmaceuticals's conference presentation, Dec 9, 2024
Slide 1: Syndax Investor Meeting, ASH Annual Meeting
Syndax Investor Meeting American Society of Hematology Annual Meeting December 9, 2024
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 and Introduction to Syndax
Welcome and Introduction to Syndax Steve Closter Chief Commercial Officer, Syndax
Slide 4: Syndax: A commercial-stage oncology company with two first-in-class medicines
Syndax: A commercial-stage oncology company with two first-in-class medicines with practice-changing and billion-dollar potential Revuforj® (revumenib) Niktimvo (axatilimab-csfr) • First and only FDA-approved menin inhibitor • FDA approved in 3L chronic GVHD (cGVHD) • Only targeted therapy for KMT2A
Slide 5: High interest in the first and only FDA-approved menin inhibitor at ASH 2024
High interest in the first and only FDA-approved menin inhibitor at ASH 2024 Driving Revuforj awareness at ASH 2024: Branded booth Three oral sessions Product theater Robust HCP engagement 5
Slide 6: Today's guest speakers
Today’s guest speakers Niktimvo (axatilimab-csfr) Chronic GVHD AGAVE-201 Results & Ongoing MAXPIRe IPF Trial Peter Ordentlich, Ph.D. Chief Scientific Officer and Founder, Syndax Revuforj® (revumenib) Acute Leukemia Overview & R/R mNPM1 AUGMENT-101 Results Eytan Stein, M.D Chief, Leukemia Service, Me
Slide 7: Recent AGAVE-201 Trial Results & Ongoing MAXPIRe IPF Trial
Recent AGAVE-201 Trial Results & Ongoing MAXPIRe IPF Trial Peter Ordentlich, Ph.D. Chief Scientific Officer and Founder, Syndax
Slide 8: AGAVE-201 met the primary efficacy endpoint across all cohorts of cGVHD patients
AGAVE-201 met the primary efficacy endpoint across all cohorts of cGVHD patients receiving axatilimab Overall Response Rates With Axatilimab 80 60 Response rate, % 40 74 (95% CI, 63–83) 67 (95% CI, 55–77) 50 20 (95% CI, 39–61) 0.3 mg/kg Q2W is now the FDA-approved dose of 0 Niktimvo (axatilimab-csfr
Slide 9: AGAVE-201 positive results in a heavily pretreated, late stage cGVHD population
AGAVE-201 positive results observed in a heavily pretreated, late stage cGVHD population AGAVE-201 AGAVE-201 study population differentiation Population (ITT) N=241 vs belumosudil study population Age median (min, max), years 53 (7, 81) Median time since cGVHD diagnosis 48 months Significantly longe
Slide 10: Secondary analyses highlight rapid onset of clinical activity with axatilimab
Secondary analyses from AGAVE-201 highlight the potential for rapid onset of clinical activity with axatilimab in cGVHD patients Data presented at ASH 2024 (abstract #98) Median times to organ-specific responses ranged from 1.0-3.2 months across organs 100 93 95 95 88 89 90 85 83 75 76 78 77 80 73 7
Slide 11: Niktimvo showed robust responses across all organs studied
Niktimvo showed robust responses across all organs studied in the heavily pre-treated population enrolled in AGAVE-201 Niktimvo 0.3 mg/kg every two weeks 100 89 Complete response 82 Partial response 80 78 76 13 Subgroup analyses provide further insight Response rate, % 60 into antifibrotic activity
Slide 12: Antifibrotic activity of axatilimab in patients with cGVHD related BOS
Antifibrotic activity of axatilimab highlighted in AGAVE-201 subgroup analysis of patients with cGVHD related bronchiolitis obliterans syndrome (BOS) Data presented at ERS 2024 Rapid and robust BOS response rates despite Clinically meaningful improvements in symptoms of inclusion of patients with se
Slide 13: MAXPIRe Phase 2 trial of axatilimab in IPF now enrolling
MAXPIRe Phase 2 trial of axatilimab in IPF is now enrolling patients with topline data anticipated in 2026 A 26-Week, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, and Tolerability of Axatilimab in Subjects with Idiopathic Pulmonary Fibrosis (IPF)
Slide 14: Acute Leukemia Overview
Acute Leukemia Overview Eytan Stein, M.D. Chief, Leukemia Service Memorial Sloan Kettering Cancer Center
Slide 15: Acute myeloid leukemia (AML) treatment paradigm
Acute myeloid leukemia (AML) treatment paradigm HSCT AML treatment Frontline Relapsed / Refractory paradigm “Fit” Intensive Chemo Consolidation (Chemo or HSCT) Salvage chemo OR targeted therapy “Unfit for IC” Azacytidine (+ Venetoclax) Despite recent advances, most patients will be refractory to the
Slide 16: Menin inhibition potential to impact patients with R/R KMT2Ar or mNPM1 AML
Menin inhibition has a near-term potential to impact a significant number of patients with R/R KMT2Ar acute leukemia or mNPM1 AML KMT2Ar Acute Leukemia mNPM1 AML (~10% of AML or ALL1) (~30% of AML2) Unfit (~20%) Fit (~80%) Unfit (~40%) Fit (~60%) Treated with Non- Treated with Intensive Therapy Trea
Slide 17: Treatment response criteria in AML
Treatment response criteria in AML: Tumor clearance equivalent across MLFS and CRc 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 18: Mutation testing, treatment decisions and response cadence with targeted therapies
Mutation testing, treatment decisions and response cadence can vary with targeted therapies Diagnosis & testing Hematologic response curve example – Time to response (TTR) Enasidenib in patients with R/R AML Varies by mutation Time on treatment (TTR + DOR) Duration of response (DOR) Impacted by: Dep
Slide 19: AUGMENT-101 R/R mNPM1 AML Trial Results
AUGMENT-101 R/R mNPM1 AML Trial Results Eytan Stein, M.D. Chief, Leukemia Service Memorial Sloan Kettering Cancer Center
Slide 20: AUGMENT-101: Phase 1/2 trial of revumenib in R/R mNPM1 and KMT2Ar acute leukemia
AUGMENT-101: A Phase 1/2 trial of revumenib monotherapy in R/R mNPM1 and KMT2Ar acute leukemia Phase 1 Phase 2 Dose Escalation Pivotal Trials KMT2Ar FDA approved Ph 1 and Ph 2 data from the ALL/MPAL AUGMENT-101 trial were used to RP2D support the FDA approval for AML R/R acute leukemia with a Adults
Slide 21: AUGMENT-101 baseline characteristics in Phase 2 R/R mNPM1 AML cohort
AUGMENT-101 baseline characteristics in Phase 2 cohort of patients with R/R mNPM1 AML Protocol-Defined Adult Safety Population Baseline Characteristics Efficacy Population N = 84 N = 64 Age, years, median (range) 65 (19, 84) 63 (11, 84) ≥ 18 to <65, n (%) 31 (48) 42 (50) Patients were ≥ 65, n (%) 33
Slide 22: Revumenib efficacy in R/R mNPM1 AML patients in Phase 2
Revumenib demonstrated compelling efficacy in R/R mNPM1 AML patients in Ph 2 portion of AUGMENT-101 Protocol-Defined Adult Best Response, n (%) Efficacy Population N = 64 CR/CRh 15 (23%) Results in protocol-defined (95% CI); one-sided p-value (14%, 36%); 0.0014 Ph 2 efficacy population: CR 12 (19%)
Slide 23: Post-hoc analysis of larger Phase 2 R/R mNPM1 AML population
Post-hoc analysis of larger Ph 2 R/R mNPM1 AML population highlights consistency of revumenib’s profile Post-Hoc Efficacy Evaluable Best Response, n (%) Adult + Pediatric Population1 N=77 CR/CRh 20 (26%) 77 of 84 enrolled patients met (95% CI) (17%, 37%) the efficacy evaluable criteria CR 16 (21%) (
Slide 24: Responses were rapid and durable in R/R mNPM1 AML patients
Responses were rapid and meaningfully durable in R/R mNPM1 AML patients in Ph 2 portion of AUGMENT-101 26-203 02-207 17-208 1 04-208 26-203 25-203 2 09-216 02-207 3 17-208 Protocol-Defined Adult 4 46-204 04-208 46-202 5 09-216 6 25-203 Efficacy Population 03-204 03-203 01-222 7 46-204 8 46-202 9 N =
Slide 25: Responses observed across all major subgroups of R/R mNPM1 AML
Responses were observed across all major subgroups of R/R mNPM1 AML patients in Ph 2 portion of AUGMENT-101 Importantly, responses were seen in: • Heavily pretreated patients • Patients with prior venetoclax exposure 25
Slide 26: Safety results from Phase 2 R/R mNPM1 AML cohort
Safety results from Phase 2 R/R mNPM1 AML cohort in the AUGMENT-101 trial support favorable revumenib safety and tolerability profile Grade ≥3 Treatment-Related Adverse Safety Population Events [TRAEs] (≥5% of patients) N = 84 Patients with Grade ≥3 TRAE 50 (60%) • Safety results in this older, heav
Slide 27: Latest AUGMENT-101 R/R KMT2Ar and SAVE Trial Results
Latest AUGMENT-101 R/R KMT2Ar and SAVE Trial Results Ghayas Issa, M.D. Associate Professor of Leukemia The University of Texas MD Anderson Cancer Center
Slide 28: Updated Results and Longer Follow-Up From AUGMENT-101 Phase 2 in R/R KMT2Ar Acute Leukemia
Updated Results and Longer Follow-Up From the AUGMENT-101 Phase 2 Study of Revumenib in All Patients With Relapsed or Refractory (R/R) KMT2Ar Acute Leukemia Ibrahim Aldoss, Ghayas C. Issa, James S. Blachly, Michael J. Thirman, Gabriel N. Mannis, Martha L. Arellano, John F. DiPersio, Elie Traer, C. M
Slide 29: Treatments Are Needed for KMT2Ar Acute Leukemia
Treatments Are Needed for KMT2Ar Acute Leukemia • Many patients experience relapse after OS in Adult Patients With R/R KMT2Ar AML After ≥3L Therapy chemotherapy and/or HSCT1 • In adults, remission rates after relapse (CR, 5%) and median OS (2.4 months) after ≥2 salvage therapies remain low1 • Outcom
Slide 30: AUGMENT-101 KMT2Ar Analyses
AUGMENT-101 KMT2Ar Analyses Phase 2 Phase 2 Phase 11 Interim Analysis2 Updated Analysis DCO: March 31, 2022 DCO: July 24, 2023 DCO: February 29, 2024 • 6 months after last • 4 months after last patient efficacy-evaluable enrolled patient enrolled for IA 2022 2023 2024 N=68 N=94 N=116 Safety • All pa
Slide 31: Phase 2 KMT2Ar: Patient Demographics
Phase 2 KMT2Ar: Patient Demographics Efficacy population Safety population Parameter (n=97)a (N=116)b Age, y, median (range) 37.0 (0.6–75.0) 35.5 (0.6–75.0) Age <18 y, n (%) 20 (20.6) 28 (24.1) Age ≥18 to <65 y, n (%) 65 (67.0) 74 (63.8) Age ≥65 y, n (%) 12 (12.4) 14 (12.1) Female, n (%) 55 (56.7) 6
Slide 32: Phase 2 KMT2Ar: Baseline Characteristics
Phase 2 KMT2Ar: Baseline Characteristics Efficacy population Safety population Parameter (n=97)a (N=116)b Leukemia type, n (%) AML 78 (80.4) 95 (81.9) ALL 13 (13.4) 15 (12.9) MPAL/other 6 (6.2) 6 (5.2) Co-mutations, n (%)c FLT3-ITD 5 (5.2) 7 (6.0) FLT3-TKD 2 (2.1) 3 (2.6) RAS 12 (12.4) 12 (10.3) TP5
Slide 33: Phase 2 KMT2Ar: Revumenib Efficacy
Phase 2 KMT2Ar: Revumenib Efficacy Efficacy Efficacy population population Parameter (n=97)a Parameter (n=97)a ORR, n (%) 62 (63.9) Best response, n (%) CR 15 (15.5) CR+CRh rate, n (%) 22 (22.7) CRh 7 (7.2) 95% CI 14.8–32.3 CRi 2 (2.1) CRc, n (%) 41 (42.3) CRp 17 (17.5) 95% CI 32.3–52.7 MLFS 20 (20.
Slide 34: Phase 2 KMT2Ar: Duration of CR+CRh
Phase 2 KMT2Ar: Duration of CR+CRh 100 Median duration of CR+CRh for 22 patients achieving response: 6.4 months (95% CI, 1.9–NR) 80 8 patients continue to remain in follow-up without relapse or death Patients, % 60 40 20 Censored 0 Time, months 0 3 6 9 12 15 No. at risk 22 12 8 4 3 0 The updated med
Slide 35: Phase 2 KMT2Ar: Duration of Treatment
Phase 2 KMT2Ar: Duration of Treatment 1 2 3 4 5 6 Time to first response, median (range): 7 8 9 • ORR (n=62): 1.0 month (0.9–3.1) 10 11 12 • CR+CRh (n=22): 2.0 months (0.9–4.6) 13 14 15 16 17 18 Individual patient response 19 20 Adult HSCT 21 22 Pediatrics Ongoing treatment at data cutoff 23 24 CR/C
Slide 36: Phase 2 KMT2Ar: Patients Who Proceeded to HSCT
Phase 2 KMT2Ar: Patients Who Proceeded to HSCT Parameter n (%) Proceeded to HSCT in ORR, n (%) 21/62 (33.9) Of patients who proceeded to HSCT: • 11/14 patients with CRc had Proceeded to HSCT in CR or CRh 8/21 (38.1) MRD status availablea: • 9 (81.8%) MRD negative Proceeded to HSCT in CRp or CRi 6/21
Slide 37: Phase 2 KMT2Ar: Revumenib Safety Profile
Phase 2 KMT2Ar: Revumenib Safety Profile Safety population (N=116)a All terms, n (%) TEAEs TRAEs Any grade 116 (100.0) 96 (82.8) Grade ≥3 106 (91.4) 63 (54.3) Serious AE 90 (77.6) 42 (36.2) AEs leading to: Dose reduction 11 (9.5) 10 (8.6) Discontinuation 16 (13.8) 6 (5.2) Death 19 (16.4) 4 (3.4) Dat
Slide 38: Phase 2 KMT2Ar: Revumenib Safety Profile (cont)
Phase 2 KMT2Ar: Revumenib Safety Profile (cont) Any-grade TEAEs that occurred in ≥25% patients Grade ≥3 TEAEs that occurred in ≥10% patients Safety population Safety population All terms, n (%) (N=116)a All terms, n (%) (N=116)a Nausea 52 (44.8) Febrile neutropenia 45 (38.8) Febrile neutropenia 46 (
Slide 39: Phase 2 KMT2Ar: DS and QTc Prolongation
Phase 2 KMT2Ar: DS and QTc Prolongation Safety population (N=116)a All terms DS QTc prolongation • Steroids were used to treat DS in Any grade TEAE, n (%) 31 (26.7) 34 (29.3) 100% of cases, and hydroxyurea was Grade 3b 16 (51.6) 15 (44.1) used in 32.3% of those cases Grade 4b 1 (3.2) 0 Grade 5 0 0 •
Slide 40: NPM1mt with Extramedullary AML on SAVE
NPM1mt with Extramedullary AML on SAVE 74 yo M, R/R AML, NPM1, KRAS, PTPN11, FLT3-TKD mutations, previously treated with 7+3+Mido → HIDAC+GO+Mido → Aza+Ven: NR → SAVE: NR (reduction in disease burden but residual PET+) Pre-treatment C1 D28 C2 D28 BM blasts 1%, MRD MFC neg BM blasts 6%, MRD MFC neg
Slide 41: Conclusions
Conclusions • In this updated phase 2 analysis, revumenib continues to consistently provide clinically meaningful responses in heavily pretreated patients with R/R KMT2Ar acute leukemias, including high rates of MRD negativity and ability to proceed to HSCT • The safety profile of revumenib is manag
Slide 42: Phase I/II Study of All-Oral Revumenib with Decitabine/Cedazuridine and Venetoclax in R/R AML (SAVE)
Phase I/II Study of the All-Oral Combination of Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax in R/R AML (SAVE) Ghayas C. Issa1, Branko Cuglievan2, Naval Daver1, Courtney D. DiNardo1, Aziz Farhat1, Nicholas J. Short1, David McCall2, Allison Pike1, Sheila Tan1, Brianna K
Slide 43: Rationale for SAVE Combination
Rationale for SAVE Combination Abstract #216 SAVE • HMA + venetoclax is standard for older/unfit AML • Oral decitabine/cedazuridine (ASTX727) approved (MDS,CMML), equivalent efficacy as IV decitabine1 • KMT2Ar or NPM1mt leukemias susceptible to apoptosis through BCL2 inhibition2-5 • Bcl-2 + menin in
Slide 44: SAVE Phase I/II Study Design
SAVE Phase I/II Study Design Abstract #216 SAVE Revumenib (SNDX-5613) DL 0: 113 mg Primary objectives: DL 1: 163 mg (RP2D of monotherapy) • Phase I (3+3 design) PO Q12h D1-D28 + a strong CYP3A4i Safety, MTD and RP2D (posaconazole or voriconazole) • Phase II • Age ≥12 years Efficacy • R/R AML or Myel
Slide 45: Baseline Characteristics - SAVE in R/R AML
Baseline Characteristics - SAVE in R/R AML Abstract #216 SAVE Characteristic N = 33 Characteristic N=9 Median age, years [range] 35 [12-81] Co-mutations, n (%) 12-17 years, n (%) 5 (15%) FLT3 10 (30%) Female, n (%) 19 (58%) ITD/TKD 5 (15%) / 5 (15%) BM blasts, % [range] 36 [1-94] WT1 10 (30%) AML, n
Slide 46: Dose Escalation – Ph1 SAVE (3+3 Design)
Dose Escalation – Ph1 SAVE (3+3 Design) Abstract #216 SAVE Revumenib (SNDX-5613) DL 0: 113 mg PO Q12h D1-28 + a strong DL 1 DL 1 DL 1 as RP2D DL 1: 163 mg PO Q12h D1-28 CYP3A4i 1/3 DLT Total of 1/6 DLT, ASTX727 D1-D5 Grade 4 0/3 DLT with review of Venetoclax D1-D14 prolonged ↓Plts overall PK, safety
Slide 47: Patient Disposition – SAVE in R/R AML
Patient Disposition – SAVE in R/R AML Abstract #216 SAVE Patient Disposition, n (%) N = 33 Remaining on study 13 (39%) Ongoing response without HSCT 10 (30%) Maintenance post-HSCT 3 (9%) Completed study including maintenance post-HSCT 2 (6%) Proceeded to HSCT 13 (39%) Proceeded to maintenance 7 (54%
Slide 48: Adverse Events – SAVE in R/R AML
Adverse Events – SAVE in R/R AML Abstract #216 SAVE TEAEs (any grade, >30% ) N = 33 TEAEs (Grade ≥3, and any G4-5) G3 G4 G5 G ≥3 QT prolongation 21 (64%) Febrile neutropenia 11 (33%) 0 0 11 (33%) Elevated AST/ALT 19 (58%) Lung infection 11 (33%) 0 0 11 (33%) Nausea 18 (55%) Elevated AST/ALT 5 (15%)
Slide 49: High response rate with SAVE in R/R AML
High response rate with SAVE in R/R AML Abstract #216 SAVE Best Response All patients KMT2Ar NPM1mt NUP98r n (%) (N=33) (N=16) (N=12) (N=5) ORR 27 (82%) 14 (88%) 8 (67%) 5 (100%) CR/CRh 16 (48%) 7 (44%) 6 (50%) 3 (60%) CR 13 (39%) 6 (38%) 6 (50%) 1 (20%) CRh 3 (9%) 1 (6%) 0 2 (40%) CRp 4 (12%) 3 (19
Slide 50: SAVE leads to rapid responses in refractory cases
SAVE leads to rapid responses in refractory cases Abstract #216 SAVE #FLT3-ITD expansion and phenotype switch to B-ALL + AML Largely stable mutational landscape at progression on targeted sequencing except for above#. MEN1 mutational analysis ongoing. Median follow-up of 9.3 months (IQR: 7.6 – 12.5)
Slide 51: Duration of Response on SAVE for R/R AML
Duration of Response on SAVE for R/R AML Abstract #216 SAVE Median follow-up of 9.3 months (IQR: 7.6 – 12.5) (N=33) Data Cutoff 11/18/2024
Slide 52: Efficacy by Subgroups on SAVE in R/R AML
Efficacy by Subgroups on SAVE in R/R AML Abstract #216 SAVE Baseline Ch. Prior Tx Genotype Co-mutation Data Cutoff 11/18/2024
Slide 53: Conclusions
Conclusions Abstract #216 SAVE • The all-oral SAVE [revumenib (SNDX-5613), oral decitabine/cedazuridine (ASTX727) and VEnetoclax] → acceptable safety and high efficacy in children and adults with R/R AML susceptible to menin inhibition • High rates of response in heavily pretreated population – ORR
Slide 54: BEAT AML Frontline Combination Trial Results
BEAT AML Frontline Combination Trial Results Joshua Zeidner, M.D. Associate Professor of Medicine, Chief, Leukemia Research, University of North Carolina, Lineberger Comprehensive Cancer Center
Slide 55: BEAT-AML and Syndax partnered on frontline SOC combos
BEAT-AML and Syndax partnered on frontline SOC combos based on strong efficacy and tolerability observed in relapsed / refractory patients HSCT AML treatment Frontline Relapsed / Refractory paradigm “Fit” Intensive Chemo Consolidation (Chemo or HSCT) Salvage chemo Or targeted therapy “Unfit for IC”
Slide 56: Azacitidine/venetoclax outcomes in frontline therapy
Azacitidine/venetoclax (aza/ven) outcomes in frontline therapy • Aza/Ven is an important Tx advance for newly diagnosed older/unfit AML • CR rates = 37% • Composite CR rates = 66% • CR+CRh = 65% • 40% achieved CR+CRh after cycle 1 • However, long-term outcomes are poor (2-year OS = 38%) and majority
Slide 57: BEAT AML: Aza/Ven + Rev in frontline mNPM1 or KMT2Ar AML
BEAT AML: Aza/Ven + Rev in frontline mNPM1 or KMT2Ar AML Phase 1 Azacitidine + Venetoclax + Revumenib Induction Continue Revumenib treatment Marrow indefinitely; until (DL1a or DL2a) Patients ≥ 60 yr remission progression, old and unfit for + Azacitidine transplant, intensive chemo (75 mg/m2 QD Marr
Slide 58: Current status of enrollment
Current status of enrollment Dose Escalation Dose Expansion Dose Expansion - Dose Expansion – Randomized Randomized #2 DL1a: N = 7 DL2a: N = 13 DL2a: N = 6 DL1a: N = 14 DL1a: N = 1 DL2a: N = 3 DL2a: N = 2 Combined *Amended to require no strong DL1a: N = 21 anti-fungals in expansion 2 DL2a: N = 22
Slide 59: BEAT AML patient demographics
BEAT AML patient demographics Patient Demographics DL1 DL2 All patients enrolled Genetics All All NPM1 KMT2A All Number of Patients (%) N=22 N=24 N=37 (80%) N=9 (20%) N=46 Age (Years), Median (Range) 73 (61-92) 69.5 (60-84) 72 (61-92) 67 (60-81) 71 (60-92) Age ≥ 75 Years, no. (%) 10 (45) 7 (29) 16 (
Slide 60: Clinical outcomes of Aza/Ven/Rev
Clinical outcomes of Aza/Ven/Rev Treatment Outcomes DL1 (n=18) DL2 (n=19) All (n=37) Genetics All All NPM1 KMT2a All Response Rates for Efficacy Evaluable Patients1 Number of Patients (%) 18 19 28 (76%) 9 (24%) 37 Composite CR (CRc)2, no. (%) 17/18 (94) 18/19 (95) 27/28 (96) 8/9 (89) 35/37 (95) Over
Slide 61: Safety of Aza/Ven/Rev
Safety of Aza/Ven/Rev • No non-hematologic DLT’s seen at any dose level • 1 Hematologic DLT observed in DL1a escalation: platelets exceeded 42 days to recover, no other DLTs across both dose levels • Grade >3 non-hematologic AE’s rarely observed and no difference between dose levels • Most common tr
Slide 62: Treatment cycle duration analysis supports cytopenias related to venetoclax
Treatment cycle duration analysis supports cytopenias related to ventoclax Induction Cycle Duration Time to Initiation of Continuation Cycles Treatment Cycle Lengths DL1a DL2a All Delay # of Continuation Proportion of All (weeks) Cycles Continuation Cycles No. of Patients 7 19 26 No Delay 6 17% Medi
Slide 63: Revumenib randomized Phase 3 study design
Revumenib randomized Phase 3 study design Azacitidine Venetoclax Revumenib* Newly diagnosed AML w/ NPM1mut Primary endpoint: OS • >75 years or unfit 1:1 for intensive chemo Azacitidine • KMT2Ar to be included but not as part of primary analysis Venetoclax • HSCT allowed Placebo * Final results from
Slide 64: Conclusions and next steps
Conclusions and next steps • Revumenib can be safely added to Aza/Ven • No new safety signals or toxicity seen • Enrollment ongoing in an expansion cohort randomized between DL1 and DL2 • Aza/Ven/Rev is highly active in newly diagnosed unfit AML patients with NPM1 mut or KMT2Ar • Overall Response ra
Slide 65: Close and Q&A
Close and Q&A Steve Closter Chief Commercial Officer, Syndax
Slide 66: Expected upcoming milestones
Revuforj (revumenib) Menin-KMT2A inhibition • Initiation of pivotal combination trial with ven/aza in newly diagnosed mNPM1 or KMT2Ar acute leukemias by YE24 • Publish and present pivotal R/R mNPM1 AML data at a medical conference in 1H25 • sNDA filing in R/R mNPM1 AML in 1H25 Expected upcoming Nikt
Slide 67: Building blocks to Syndax's transformational era
Building blocks to Syndax’s transformational era 2025 and beyond 2023-2024 Transformational era of Before 2023 sustainable and profitable growth Advanced clinical, regulatory, and operational excellence Established scientific • Well positioned to change the leadership treatment landscape and drive •
Slide 68: Determined to realize a future in which people with cancer live longer and better
Determined to realize a future in which people with cancer live longer and better than ever before. 68
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Syndax Pharmaceuticals — American Society of Hematology Annual Meeting 2024 on 6ix