Syndax Announces Publication of SAVE Data on Revuforj® (revumenib) in Combination with Decitabine/Cedazuridine and Venetoclax in Relapsed/Refractory NPM1m, KMT2Ar, and NUP98r AML in the Journal of Clinical Oncology

Rhea-AI Summary

Syndax (Nasdaq:SNDX) reported Phase 1/2 SAVE trial data for all-oral Revuforj (revumenib) + decitabine/cedazuridine + venetoclax in relapsed/refractory NPM1m, KMT2Ar, and NUP98r AML.

Among 42 heavily pretreated patients, ORR was 88%, CRc 71%, CR/CRh 60%, MRD negativity in CRc 80%, 45% proceeded to transplant, and safety was described as generally manageable.

Positive

• Overall response rate 88% (37/42) in heavily pretreated R/R AML
• Composite complete remission (CRc) rate 71% (30/42); CR/CRh 60% (25/42)
• MRD negativity by flow in 80% (24/30) of evaluable CRc responders
• 45% (19/42) of patients proceeded to hematopoietic stem cell transplant
• 1-year overall survival 56% for cohort; median OS after transplant not reached
• CR/CRh rate 50% and ≥12-month median OS in prior venetoclax-exposed patients

Negative

• Common Grade ≥3 TEAEs: febrile neutropenia 36%, lung infection 21%, thrombocytopenia 21%
• Grade ≥3 liver enzyme elevations in 21% of patients
• Grade ≥3 differentiation syndrome and QTc prolongation each reported in 5% of patients
• Median duration of response 5.9 months in NUP98-rearranged subgroup

News Market Reaction – SNDX

+1.88%

1 alert

+1.88% Session close to close

+8.3% Peak Tracked

$1.59B Market Cap

3.49K Volume

In the Jun 11 session, SNDX gained 1.88%, reflecting a mild positive market reaction. Argus tracked a peak move of +8.3% during that session.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement highlights compelling SAVE trial outcomes for Revuforj® combinations in relapsed/r...

Analysis

This announcement highlights compelling SAVE trial outcomes for Revuforj® combinations in relapsed/refractory AML, with an 88% ORR, high CR/CRh rates, and substantial MRD negativity in a heavily pretreated setting. Recent history shows a steady cadence of Revuforj data and new trials, alongside a $250.0M convertible financing and expanded equity plans. Investors may focus on durability metrics, transplant outcomes, safety signals, and how these results integrate with ongoing pivotal studies such as EVOLVE-2 and RAVEN.

Key Figures

Overall response rate: 88% (37/42) CRc rate: 71% (30/42) CR/CRh rate: 60% (25/42) +5 more

8 metrics

Overall response rate 88% (37/42) Phase 1/2 SAVE trial in R/R AML

CRc rate 71% (30/42) Composite complete remission in SAVE trial

CR/CRh rate 60% (25/42) Complete remission or CR with partial hematologic recovery

MRD negativity 80% (24/30) Among evaluable CRc responders by flow cytometry

Transplant rate 45% (19/42) Patients proceeding to HSCT after regimen

Median overall survival 14 months Heavily pretreated R/R AML patients in SAVE

CR/CRh venetoclax-exposed 50% (11/22) Patients with prior venetoclax exposure

Historical median survival 2.4 months Typical outcome in venetoclax-exposed population

Historical Context

5 past events · Latest: Jun 04 (Neutral)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Jun 04	Inducement option grants	Neutral	-3.8%	Stock option inducement awards to new employees under 2023 plan.
Jun 04	Convertible notes financing	Negative	-1.7%	Announcement of $250M 2.25% Convertible Senior Notes due 2031.
Jun 02	Revuforj ASCO data	Positive	-0.3%	New Revuforj post-transplant and pharmacokinetic data at ASCO 2026.
Jun 01	R&D Day announcement	Positive	-3.8%	Planned R&D Day to showcase late-stage and early-stage oncology assets.
May 21	ASCO abstracts accepted	Positive	+0.9%	Four Revuforj abstracts, including an oral, accepted for ASCO 2026.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Recent Revuforj data and corporate updates have often coincided with flat-to-negative next-day moves, even on seemingly positive news, while financing-related events also saw modest declines.

Recent Company History

Over the last month, Syndax has focused heavily on Revuforj® data and corporate positioning. ASCO 2026 presentations and abstract acceptances highlighted encouraging survival and trial designs, yet 24-hour moves ranged from -0.27% to +0.86%. A planned R&D Day and inducement equity grants preceded declines of -3.78% and -3.77%. A $250.0 million convertible notes placement on Jun 4, 2026 also saw a modest negative reaction. Today’s SAVE trial publication adds another clinically positive data point to this sequence of news-heavy weeks.

Key Terms

minimal residual disease (MRD), hypomethylating agent, hematopoietic stem cell transplant (HSCT), treatment-emergent adverse events (TEAEs), +3 more

7 terms

minimal residual disease (MRD) medical

"Deep responses with 80% (24/30) MRD negativity among evaluable CRc responders"

The presence of minimal residual disease (MRD) means a very small number of cancer cells remain in the body after treatment, too few to cause symptoms or show up on routine scans but detectable with sensitive tests. For investors it matters because MRD status is a strong early indicator of whether a patient is likely to relapse and is increasingly used as a trial endpoint and regulatory signal, affecting a therapy’s market prospects and valuation much like finding glowing embers after a fire signals risk of re-ignition.

hypomethylating agent medical

"revumenib with venetoclax and a hypomethylating agent in multiple settings"

A hypomethylating agent is a type of drug that changes how genes are turned on or off by reversing chemical tags on DNA, which can restore normal cell behavior in certain cancers. For investors, these drugs matter because clinical trial results, regulatory approvals, or setbacks can quickly alter a company’s revenue prospects and valuation, much like fixing a stuck instruction in a machine can restore its output.

hematopoietic stem cell transplant (HSCT) medical

"33% (14/42) a prior hematopoietic stem cell transplant (HSCT)"

A hematopoietic stem cell transplant (HSCT) is a medical procedure that replaces a patient’s damaged or diseased blood-forming cells with healthy stem cells from the patient or a donor, effectively “rebooting” the body’s blood and immune system. It matters to investors because HSCTs drive demand for specific drugs, medical devices, hospital services and long-term care, and their success, costs and regulatory status can strongly influence the financial outlook for companies and healthcare providers involved.

treatment-emergent adverse events (TEAEs) medical

"The most common treatment-emergent adverse events (TEAEs) included elevations"

Adverse events that first appear or worsen after a patient starts a medical treatment; they are the new or intensified negative effects linked in time to taking the drug or therapy. Investors care because the number and severity of these events shape regulators’ decisions, drug labeling, patient uptake and potential legal or cost risks—think of them like customer complaints that can slow sales, trigger recalls, or change a product’s value.

differentiation syndrome medical

"Rates of Grade ≥3 differentiation syndrome (5%) and QTc prolongation (5%)"

Differentiation syndrome is a potentially serious treatment reaction that can occur when certain cancer drugs force immature tumor cells to mature quickly, causing widespread inflammation, fluid buildup, breathing problems or organ stress — like a sudden crowd surge that clogs exits and streets. It matters to investors because the risk and management of this side effect can influence clinical trial results, drug approvals, safety labeling, prescribing use and ultimately a therapy’s market value and adoption.

QTc prolongation medical

"Rates of Grade ≥3 differentiation syndrome (5%) and QTc prolongation (5%)"

QTc prolongation is a lengthening of the heart’s electrical “reset” time between beats, measured on an electrocardiogram and adjusted for heart rate. It matters to investors because prolonged QTc can signal a drug or device safety risk that may lead to regulatory warnings, clinical trial delays, label restrictions, market withdrawals, or higher liability costs—similar to a car whose brakes take longer to reset, increasing the chance of a dangerous failure.

Kaplan-Meier estimate technical

"The median OS observed was similar... based on a Kaplan-Meier estimate."

A Kaplan-Meier estimate is a statistical curve that shows how the chance of avoiding a particular event (like disease progression, treatment failure, or death) changes over time among a group of people in a clinical study. Think of it like a chart tracking how many lightbulbs remain working as hours pass; for investors it matters because it summarizes a drug or treatment’s durability and timing of benefits, which can influence regulatory decisions, market expectations, and valuation.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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– High response rates observed with the all-oral combination in a heavily pretreated population, including 88% (37/42) ORR, 71% (30/42) CRc, and 60% CR/CRh (25/42) –

– Strong activity across subgroups, including 70% (14/20) CR/CRh in venetoclax-naïve and 50% (11/22) CR/CRh in venetoclax-exposed patients –

– Deep responses with 80% (24/30) MRD negativity among evaluable CRc responders –

– Robust transplant rate with 45% (19/42) of patients proceeding to transplant and 63% (12/19) resuming revumenib post-transplant –

– Encouraging durability with median overall survival after transplant not reached – 

– Combination was generally well-tolerated –

NEW YORK, June 11, 2026 (GLOBE NEWSWIRE) -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company advancing innovative cancer therapies, today announced that data from the Phase 1/2 SAVE trial of an all-oral regimen of Revuforj^® (revumenib), decitabine/cedazuridine, and venetoclax in relapsed or refractory (R/R) NPM1 mutated (NPM1m), KMT2A-rearranged (KMT2Ar), or NUP98-rearranged (NUP98r) acute myeloid leukemia (AML) were published in the Journal of Clinical Oncology and simultaneously presented at the European Hematology Association (EHA) 2026 Congress in Stockholm, Sweden.

Revuforj is the first and only menin inhibitor that is FDA approved for patients one year and older with R/R AML with a susceptible NPM1 mutation who have no satisfactory alternative treatment options or R/R acute leukemia with a KMT2A translocation as determined by an FDA-authorized test.

“The deep and durable remissions observed among patients with R/R NPM1m, KMT2Ar, or NUP98r AML who received an all-oral combination of revumenib, decitabine/cedazuridine, and venetoclax, highlight the potential for revumenib combinations to advance the standard of care treatment for menin-dependent acute leukemias,” said Nick Botwood, MBBS, Head of Research & Development and Chief Medical Officer at Syndax. “The SAVE data provide strong support for further studying revumenib with venetoclax and a hypomethylating agent in multiple settings, including among newly diagnosed patients who are unfit for intensive chemotherapy in the ongoing pivotal EVOLVE-2 trial and among fit patients in the RAVEN trial.”

“The results observed with the all-oral SAVE regimen in heavily pretreated patients with R/R NPM1m, KMT2Ar, or NUP98r AML are very encouraging,” said Ghayas C. Issa, M.D., Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center and Principal Investigator of the SAVE trial. “Notably, 88% of patients achieved a response with the majority achieving MRD negativity, 45% proceeded to a potentially curative stem cell transplant, and we observed a 14-month median overall survival. We also saw an impressive 50% CR/CRh rate and approximately 12-month median overall survival in patients with prior venetoclax exposure, a population that has historically experienced poor outcomes with a median survival of less than three months.”   

Summary of Key Results from the SAVE Trial in R/R NPM1m, KMT2Ar, and NUP98r AML

The publication entitled, “All-Oral Combination of Revumenib, Decitabine, and Venetoclax for Relapsed or Refractory AML (SAVE)” reports results from the R/R cohort of patients in the Phase 1/2, single-center, open-label SAVE trial. The primary endpoint of Phase 1 was the recommended Phase 2 dose of revumenib in combination therapy, which was identified as dose level 1 (the FDA-approved monotherapy dose of revumenib). The primary endpoint of Phase 2 was the composite complete remission¹ (CRc) rate.

As of January 2026, 42 patients with R/R AML were enrolled in the SAVE trial, including five adolescents. 38% (16/42) had NPM1m, 40% (17/42) had KMT2Ar, and 21% (9/42) had NUP98r. The median age was 40 years (range: 12-82). Patients were heavily pretreated, with a median of two prior lines of therapy (range: 1-5); 52% (22/42) had received prior venetoclax and 33% (14/42) a prior hematopoietic stem cell transplant (HSCT).

The overall response rate (ORR) was 88% (37/42), the CRc rate was 71% (30/42), and the complete remission plus complete remission with partial hematological recovery (CR/CRh) rate was 60% (25/42) for the entire population. Response rates were similar across genotypes, including patients with NPM1m, KMT2Ar, or NUP98r. Overall, 45% (19/42) of patients proceeded to HSCT following treatment with the regimen, including 38% (6/16) of NPM1m patients, 65% (11/17) of KMT2Ar patients, and 22% (2/9) of NUP98r patients. Of the 19 patients that proceeded to HSCT, 63% (12/19) resumed revumenib after HSCT.

The rates of measurable residual disease (MRD) negativity by flow cytometry were 68% (25/37) among evaluable responders, 80% (24/30) among those with CRc, and 80% (20/25) among those with CR/CRh. Among evaluable patients with CRc, 67% (8/12) were MRD negative by NPM1 NGS (<0.01% threshold), with the vast majority of those patients (7/8) below the limit of detection of the assay (5x10^-5), highlighting the depth of MRD clearance.

With a median follow-up of 22 months, the median duration of response among patients with CR/CRh was 10.5 months for the entire cohort, 10.7 months among NPM1m patients, not reached among KMT2Ar patients, and 5.9 months among NUP98r patients. The observed 1-year overall survival (OS) rate was 56% for the entire cohort, 63% among NPM1m patients, 47% among KMT2Ar patients, and 67% among NUP98r patients. Median OS after HSCT was not reached. Patients who were MRD negative by flow cytometry had a longer median duration of response (20 months vs. 2.9 months) and OS (not reached vs. 8.4 months) compared to those who were MRD positive.

Notably, clinical activity was observed among patients with prior exposure to venetoclax, a population in whom outcomes are typically poor, with a historical estimated median survival of 2.4 months. In this trial, the CR/CRh rate was 50% (11/22) in patients with venetoclax exposure versus 70% (14/20) in those without. The median OS observed was similar between the two groups (at least 12 months in both groups), based on a Kaplan-Meier estimate. This observation supports a potential biologic synergy between BCL2 inhibition and menin inhibition and the possibility that menin inhibition may restore sensitivity to BCL2 inhibition after resistance has developed.

Revumenib was generally well-tolerated in combination with decitabine/cedazuridine and venetoclax. The most common treatment-emergent adverse events (TEAEs) included elevations in aspartate aminotransferase or alanine aminotransferase (71%), nausea (52%), and vomiting (48%). The most common Grade ≥3 TEAEs were febrile neutropenia (36%), lung infection (21%), thrombocytopenia (21%), and elevations in aspartate aminotransferase or alanine aminotransferase (21%). Rates of Grade ≥3 differentiation syndrome (5%) and QTc prolongation (5%) were both low.

About Revuforj^® (revumenib)

Revuforj (revumenib) is the first and only menin inhibitor that is FDA approved for the treatment of adult and pediatric patients one year and older with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options or R/R acute leukemia with a KMT2A translocation as determined by an FDA-authorized test.

Multiple trials of revumenib are ongoing or planned across the treatment landscape, including in combination with standard of care therapies in newly diagnosed patients with NPM1m or KMT2Ar AML.

Revuforj (revumenib)

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME, QTc PROLONGATION, and TORSADES DE POINTES

Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

QTc prolongation and Torsades de Pointes have occurred in patients receiving Revuforj. Correct hypokalemia and hypomagnesemia prior to and during treatment. Do not initiate Revuforj in patients with QTcF > 450 msec. If QTc interval prolongation occurs, interrupt, reduce, or permanently discontinue Revuforj.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, rash, and/or hypotension.

In clinical trials, DS occurred in 60 (25%) of 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia. Among those with a KMT2A translocation, DS occurred in 33% of patients with acute myeloid leukemia (AML), 33% of patients with mixed-phenotype acute leukemia (MPAL), and 9% of patients with acute lymphoblastic leukemia (ALL); DS occurred in 18% of patients with NPM1m AML. DS was Grade 3 or 4 in 12% of patients and fatal in 2 patients. The median time to initial onset was 9 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%.

Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10 mg IV every 12 hours in adults or dexamethasone 0.25 mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids.

QTc Interval Prolongation and Torsades de Pointes: Revuforj can cause QT (QTc) interval prolongation and Torsades de Pointes.

Of the 241 patients treated with Revuforj at the recommended dosage for relapsed or refractory acute leukemia in clinical trials, QTc interval prolongation was reported as an adverse reaction in 86 (36%) patients. QTc interval prolongation was Grade 3 in 15% and Grade 4 in 2%. The heart-rate corrected QT interval (using Fridericia’s method) (QTcF) was greater than 500 msec in 10%, and the increase from baseline QTcF was greater than 60 msec in 24%. Revuforj dose reduction was required for 7% due to QTc interval prolongation. QTc prolongation occurred in 21% of the 34 patients less than 17 years old, 35% of the 146 patients 17 years to less than 65 years old, and 46% of the 61 patients 65 years or older. One patient had a fatal outcome of cardiac arrest, and one patient had non-sustained Torsades de Pointes.

Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to and throughout treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation.

• Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec
• Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec
• Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia
  

Embryo-Fetal Toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj.

ADVERSE REACTIONS

Fatal adverse reactions occurred in 9 (4%) patients who received Revuforj, including 4 with sudden death, 2 with differentiation syndrome, 2 with hemorrhage, and 1 with cardiac arrest.

Serious adverse reactions were reported in 184 (76%) patients. The most frequent serious adverse reactions (≥10%) were infection (29%), febrile neutropenia (20%), bacterial infection (15%), differentiation syndrome (13%), and hemorrhage (11%).

The most common adverse reactions (≥20%) including laboratory abnormalities, were phosphate increased (51%), hemorrhage (48%), nausea (48%), infection without identified pathogen (46%), aspartate aminotransferase increased (44%), alanine aminotransferase increased (40%), creatinine increased (38%), musculoskeletal pain (37%), febrile neutropenia (37%), electrocardiogram QT prolonged (36%), potassium decreased (34%), parathyroid hormone intact increased (34%), alkaline phosphatase increased (33%), diarrhea (29%), bacterial infection (27%), triglycerides increased (27%), phosphate decreased (25%), differentiation syndrome (25%), fatigue (24%), edema (24%), viral infection (23%), decreased appetite (20%), and constipation (20%).

DRUG INTERACTIONS

Drug interactions can occur when Revuforj is concomitantly used with:

• Strong CYP3A4 inhibitors: reduce Revuforj dose
• Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj
• QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec
  

SPECIFIC POPULATIONS

Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose.  

Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj.

Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible.

Pediatric: monitor bone growth and development in pediatric patients.

Geriatric: no overall differences were observed in the effectiveness of Revuforj between patients who were 65 years and older, and younger patients. Compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older.

To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Full Prescribing Information, including BOXED WARNINGS.

About Syndax

Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company advancing innovative cancer therapies. Highlights of the Company's pipeline include Revuforj^® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit www.syndax.com or follow the Company on X and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Revuforj's or Niktimvo’s commercial availability; changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

References

1. Composite complete remission (CRc) includes CR, CRh, CRp, and CRi. Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS, and PR. 
   CR = Complete remission
   CRh = Complete remission with partial hematologic recovery
   CRp = Complete remission with incomplete platelet recovery
   CRi = Complete remission with incomplete count recovery
   MLFS = Morphologic leukemia-free state
   PR = Partial response

Syndax Contact

Sharon Klahre
Syndax Pharmaceuticals, Inc.
sklahre@syndax.com
Tel 781.684.9827

SNDX-G

FAQ

What did Syndax (SNDX) report from the SAVE trial of Revuforj in R/R AML?

Syndax reported high response rates from the Phase 1/2 SAVE trial of all-oral Revuforj plus decitabine/cedazuridine and venetoclax in relapsed/refractory AML. According to Syndax, 88% of 42 heavily pretreated patients responded and 71% achieved composite complete remission (CRc).

What were the remission and MRD negativity rates in the SAVE trial for SNDX Revuforj?

The SAVE regimen showed 60% CR/CRh and 71% CRc in relapsed/refractory AML. According to Syndax, 80% of evaluable CRc responders achieved MRD negativity by flow cytometry, indicating deep remissions across NPM1m, KMT2Ar, and NUP98r genotypes.

How did prior venetoclax exposure affect outcomes in the SAVE trial for SNDX?

Patients with prior venetoclax exposure still demonstrated notable activity with the SAVE regimen. According to Syndax, CR/CRh was 50% (11/22) in venetoclax-exposed versus 70% (14/20) in venetoclax-naïve patients, with Kaplan–Meier–estimated median overall survival of at least 12 months in both groups.

What overall survival and transplant outcomes were reported in the SAVE study of Revuforj?

The SAVE trial showed encouraging survival and transplant rates in high-risk AML. According to Syndax, 45% of patients proceeded to stem cell transplant, 1-year overall survival was 56% for the cohort, and median overall survival after transplant was not reached at 22 months’ follow-up.

What safety profile was observed with the Revuforj, decitabine/cedazuridine, and venetoclax combination?

The all-oral combination was described as generally well-tolerated but with notable cytopenias and infections. According to Syndax, common Grade ≥3 events included febrile neutropenia (36%), lung infection (21%), thrombocytopenia (21%), and Grade ≥3 liver enzyme elevations in 21% of patients.

How might SAVE trial results influence future studies of SNDX Revuforj combinations?

SAVE results are being used to guide broader development of revumenib-based regimens. According to Syndax, the data support further study with venetoclax and hypomethylating agents in pivotal trials, including EVOLVE-2 for newly diagnosed unfit patients and RAVEN for fit patients.