Slides
Page 1
FDA Approval of Revuforj® (revumenib) in R/R NPM1 Mutated AML October 24, 2025
Page 2
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 referencing future events, progress, timing or circumstances) are intended to identify forward-looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding future operations, financial results and the financial condition of Syndax Pharmaceuticals, Inc. (“Syndax” or the “Company”), including financial position, strategy and plans, the progress, timing, clinical development and scope of clinical trials and the reporting of clinical data for Syndax’s product candidates, the progress of regulatory submissions and approvals and subsequent commercialization and the potential use of Syndax’s product candidates to treat various cancer indications and fibrotic diseases, and Syndax’s expectations for liquidity and future operations, are forward-looking statements. Many factors may cause differences between current expectations and actual results, including unexpected safety or efficacy data observed during preclinical studies or clinical trials, clinical site activation rates or clinical trial enrollment rates that are lower than expected; changes in expected or existing competition; the impact of macroeconomic conditions (the Russia-Ukraine war, inflation, among others) on Syndax’s business and that of the third parties on which Syndax depends, including delaying or otherwise disrupting Syndax’s clinical trials and preclinical studies, manufacturing and supply chain, or impairing employee productivity; failure of our collaborators to support or advance collaborations or product candidates and unexpected litigation or other disputes. Moreover, Syndax operates in a very competitive and rapidly changing environment. Other factors that may cause our actual results to differ from current expectations are discussed in Syndax’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” sections contained therein. New risks emerge from time to time. It is not possible for Syndax’s management to predict all risks, nor can Syndax assess the impact of all factors on its business or the extent to which any factor ,or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statement. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this presentation may not occur and actual results could differ materially and adversely from those anticipated or implied. Except as required by law, neither Syndax nor any other person assumes responsibility for the accuracy and completeness of the forward- looking statements. Syndax undertakes no obligation to update publicly any forward-looking statements for any reason after the date of this presentation to conform these statements to actual results or to changes in Syndax’s expectations. 2
Page 3
Agenda for today’s call 3 1 Opening Remarks & Key Highlights Michael Metzger , Chief Executive Officer 2 Revuforj (revumenib) Label & Supporting Data Dr. Nick Botwood, Head of R&D and Chief Medical Officer 3 Delivering Revuforj to Patients Steve Closter , Chief Commercial Officer 4 Q&A Session Syndax Management Team
Page 4
Multiple acute leukemia subtypes CONFIDENTIAL 4 REVUFORJ IS NOW FDA-APPROVED FOR A SECOND INDICATION Adults and children ≥1 year of age Now approved for treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options FIRST AND ONLY MENIN INHIBITOR APPROVED FOR:
Page 5
Revuforj is well positioned for near- and long-term success, with a $5B+ U.S. market opportunity across the R/R and 1L setting 5 ➢ Compelling clinical data across: ✓ Multiple genetic subtypes including, NPM1m, KMT2Ar , and NUP98r ✓ Adults and pediatrics ✓ 1L and R/R combinations ➢ First and only menin inhibitor FDA approved for multiple acute leukemia subtypes in adults and pediatrics ➢ Comprehensive 1L clinical development plan underway BEST-IN-CLASS PROFILE ➢ Well established with HCPs — nearly 1 year of commercial Revuforj experience ➢ >1,000 patients treated with Revuforj across commercial and clinical trial experience ➢ Track record of delivering best-in-class HCP/patient experience ➢ Included in NCCN Guidelines for R/R NPM1m AML & KMT2Ar acute leukemia FIRST -MOVER ADVANTAGE Revuforj is FDA approved for the treatment of R/R acute leukemia with a KMT2A translocation as determined by an FDA -authorized test in adult and pediatric patients 1 year and older. Revuforj is also indicated for the treatment of R/R acute myeloid leukemia (AML) with a susceptible NPM1 mutation in adult and pediatric patients 1 year and older who have no satisfac tory alternative treatment options.
Page 6
Revuforj (revumenib) Label & Supporting Data Dr. Nick Botwood, Head of R&D and Chief Medical Officer
Page 7
Alma, diagnosed with R/R NPM1m AML R/R NPM1m AML is an area of high unmet need NPM1 mutations (NPM1m) are the most common genetic alteration in AML1 Outcomes are poor for NPM1m patients who relapse or are refractory to Tx ~1 in 3 patients with AML have an NPM1 mutation1 >50% of NPM1m patients relapse after 1L therapy2-3 Revuforj offers a targeted approach to R/R NPM1m AML 1. Ranieri, et al. Current status and future perspectives in targeted therapy of NPM1 -mutated AML. Leukemia 2022; 2. Hubmann, et al. Molecular response assessment by quantitative real-time polymerase chain reaction after induction therapy in NPM1 -mutated patients identifies those at high ri sk of relapse. Haematologica 2014; 3. Othman, et al. Molecular MRD is strongly prognostic in patients with NPM1 -mutated AML receiving venetoclax-based nonintensive therapy. Blood 2024. R/R, relapsed or refractory; 1L, frontline; Tx, treatment 7
Page 8
Overview of Revuforj (revumenib) U.S. Prescribing Information 8 Indication: • Revuforj is a menin inhibitor indicated for: • the treatment of R/R acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation as determined by an FDA-authorized test in adult and pediatric patients 1 year and older. • the treatment of R/R acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options. Dosage & Administration: • Administered orally, twice daily • Recommended dosage varies by patient weight and concomitant use of strong CYP3A4 inhibitors Safety • Boxed warnings for differentiation syndrome, and QTc prolongation and Torsades de Pointes • Warning and precaution for embryo-fetal toxicity • No contraindications Please see full Prescribing Information, including BOXED WARNINGS, for complete details.
Page 9
Key data from AUGMENT-101 pivotal trial of Revuforj in R/R NPM1m AML 9 23% CR/CRh (primary endpoint) 2.8 months median time to CR/CRh 4.5 months median duration of CR/CRh 11% of pts. underwent HSCT following Revuforj 47% overall response rate1 23-months mOS observed among responders in subgroup analysis2 63% MRD negativity among pts. with CR/CRh2 Key efficacy data from AUGMENT-101 included in Revuforj USPI Additional data from AUGMENT-101 published in Blood or presented at medical meetings 1. Arellano, et al. Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT -101 study. Blood 2025. 2. Arellano, et al. Revumenib for Patients With Relapsed or Refractory (R/R) Nucleophosmin 1–Mutated (NPM1m) Acute Myeloid Leukemia (AML): Updated Results From the Phase 2 AUGMENT -101 Study. Poster presented at Society of Hematologic Oncology (SOHO) 2025. The primary Phase 2 endpoints of the AUGMENT -101 trial were the rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) and safety. Other secondary endpoints, such as overall response rate (ORR), and post -hoc analyses, such as median overall survival (mOS) among responders should be interpreted with caution due to the lack of statis tical power.
Page 10
Comprehensive Revuforj data generation strategy underway to further solidify revumenib’s leading position 10 Present additional rev data, including first RWE & Ph 1 1L combination data with I.C. by YE25 2H25 1L, frontline; R/R, relapsed or refractory; ven/aza, venetoclax/azacitidine; I.C., intensive chemotherapy; rev, revumenib; Ph, phase; RWE, real world evidence Completed milestones Anticipated milestones Added to NCCN Guidelines for R/R NPM1m AML Published promising Ph 1 rev + ven/aza 1L data Initiated EVOLVE pivotal 1L trial of rev + ven/aza Initiate 1L REVEAL trials of rev + I.C. in 4Q25 FDA approval of second indication Reported encouraging Ph 1 R/R NUP98r data Published pivotal R/R NPM1m AML data 2024 Original FDA approval Added to NCCN Guidelines for R/R KMT2Ar acute leukemia 1H25
Page 11
Delivering Revuforj to Patients Steve Closter , Chief Commercial Officer
Page 12
12 Approval unlocks $2B U.S. market opportunity for Revuforj in R/R acute leukemia alone With the largest addressable population and anticipated duration of therapy, Revuforj is poised to become the largest targeted AML therapy R/R Acute Leukemia with KMT2A translocation R/R AML with NPM1m 1L “Unfit” AML with KMT2Ar or NPM1m 1L “Fit” AML with KMT2Ar or NPM1m Est. annual incidence ~2,000 ~4,500 ~3,500 ~5,500 U.S. Market Opportunity ($ B) $5B+ TAM $2B+ TAM 20-25% 25-30% 40-45% IDH1/2 inhibitors FLT3 inhibitors Revuforj Addressable AML population Revuforj FLT3 inhibitors IDH inhibitors NPM1 mutations and KMT2A translocations are routinely tested for, enabling efficient patient identification
Page 13
13 Strong prescriber base & HCP familiarity ✓ Same HCPs treat both KMT2A and NPM1 patients ✓ Nearly 1 year of commercial experience ✓ >1,000 patients treated across commercial and clinical trial setting ✓ 65% Tier 1/2 account penetration through 2Q25, plus robust community adoption Excellent payer support & reimbursement ✓ On formulary for 97% of covered lives ✓ Reimbursement supported by NCCN Guideline listings ✓ Centers have experience successfully navigating reimbursement ✓ Completed extensive education on R/R NPM1m Demonstrated ease of access to Revuforj ✓ Best-in-class HCP/patient support, including dedicated patient hub ✓ Efficient limited distribution model ✓ <4 days average time from script to 1st fill World-class commercial team, proven track record ✓ >20 yrs of experience average with deep relationships in hem-onc ✓ Delivered $56M Revuforj net revenue through 2Q25, exceeding AML analogs ✓ Equipped with advanced tools for HCP targeting Note: All launch metrics are from November 2024 through the end of June 2025. Revuforj is positioned to lead in R/R NPM1m with an industry-leading profile and solid foundation for success
Page 14
Syndax will continue to focus on three strategic imperatives to deliver for patients and drive continued Revuforj growth 14 1 Leave no appropriate patient behind 2 Engage all key stakeholders Deliver premium HCP/patient experience 3 High-risk patients require rapid identification of Tx opportunities Complex patient journey makes it crucial to engage all decision makers Critically ill patients need immediate access without barriers
Page 15
15 Two first- & best-in-class drugs $5B+ TAM $5B+ TAM Two exceptional product launches Syndax is on the road to profitability with two medicines with multi-billion- dollar potential
Page 16
16 Lilah, diagnosed with R/R AML FUELED BY A PASSION FOR PATIENTS