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Groupstrong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.Q9-landscape

Syndax Pharmaceuticals — revumenib (SNDX-5613), the only menin inhibitor licensed down to infancy

also called Syndax · SNDX · Revuforj · SNDX-5613

Syndax owns revumenib, the first menin inhibitor approved by the FDA, and it is the only one licensed for children as young as one year old. Its main trial has enrolled patients from 30 days of age, which makes Syndax the single most plausible industry partner for a lab working on infant leukaemia.

The same thing, for a scientist

Syndax Pharmaceuticals (Waltham, MA) developed revumenib (SNDX-5613), a reversible small-molecule inhibitor of the menin-KMT2A interaction. Revumenib is FDA-approved as REVUFORJ for relapsed/refractory acute leukaemia with a KMT2A translocation and for R/R NPM1-mutant AML, in adult and paediatric patients 1 year and older [E1]. The registrational study AUGMENT-101 (NCT04065399) enrols from 30 days of age with weight-banded dosing, and the combination study AUGMENT-102 (NCT05326516) used the same 30-day floor [E2][E3]. The pipeline is now moving upstream into frontline intensive-chemotherapy combinations and a phase 3 in newly diagnosed NPM1-mutant AML (NCT07211958), where the age floor rises to 12 years [E4].

Go deeper
What Syndax owns that an academic lab cannot generate1 deeper

Three things, only one of which is the molecule.

The compound, with a label. Revumenib is not an investigational tool compound; it is REVUFORJ, an approved drug with an FDA-authorised companion test, indicated for R/R acute leukaemia with a KMT2A translocation and for R/R NPM1-mutant AML in adult and paediatric patients 1 year and older 1. A lab can buy revumenib as a research chemical and can request it under a materials-transfer agreement, but it cannot generate the regulatory asset or the clinical-grade supply.

A paediatric-enabled trial network. AUGMENT-101 runs across 57 registered sites in the United States, Australia, Canada, France, Germany, Israel, Italy, Lithuania, the Netherlands, Puerto Rico and Spain, with paediatric centres explicitly among them (Children's Healthcare of Atlanta, The Hospital for Sick Children Toronto, the Princess Máxima Center) 2. That is the infrastructure a single academic laboratory most conspicuously lacks.

Banked on-treatment patient material. The registrational cohort was 94 treated patients with central confirmation of KMT2A rearrangement, and the resistance work that defined MEN1 interface mutations came out of exactly this trial's specimens 5. Serial marrows taken before and during menin inhibition in KMT2A-rearranged disease are, at present, obtainable essentially only through the sponsors.

What a small lab brings in exchange is mechanism: why response rates in KMT2A-rearranged disease sit near 23% CR+CRh rather than near 100%, and what the non-responding fraction is doing. Syndax has a commercial reason to want that answer and no structural reason to generate it in-house.

The age question, answered precisely

This is the decisive fact for an infant-ALL-focused programme and it is worth stating exactly rather than approximately.

StudyNCTPhaseStatusMinimum age
AUGMENT-101NCT040653991/2Recruiting30 days 2
AUGMENT-102 (revumenib + chemotherapy, R/R)NCT053265161Completed30 days 3
Frontline revumenib + intensive chemotherapyNCT062265711Active, not recruiting18 years 4
Phase 3, newly diagnosed NPM1-mutant AMLNCT072119583Recruiting12 years 4
Revumenib + azacitidine + venetoclax (St. Jude-led, Syndax collaborator)NCT061770671Recruiting1 year (to 30 years) 4

The pattern matters. The relapsed/refractory programme is genuinely infant-inclusive at 30 days. The frontline programme is not: as revumenib moves into newly diagnosed disease, the floor rises to 12 or 18 years, because frontline AML trials are built around adult standard-of-care backbones. An infant-ALL lab that wants to engage on frontline biology is engaging with a programme that currently excludes its patients; one that wants to engage on relapsed disease is engaging with the only industry programme in this class that does not.

In AUGMENT-101's registrational cohort the median age was 37 years with a range of 1.3 to 75 years 5. So the 30-day floor is real but the enrolled population is adult-weighted. A paediatric-specific dataset is thin even here.

Is a collaboration realistically possible?

Yes, and more plausibly than with any other company on this list, for three reasons.

First, Syndax has already demonstrated willingness to supply drug to investigator-initiated academic studies. It is a listed collaborator on the St. Jude-sponsored phase 1 of revumenib with azacitidine and venetoclax in patients aged 1 to 30 (NCT06177067), and revumenib appears as a sub-study agent inside the Beat AML master protocol (NCT03013998) and in MD Anderson's investigator-sponsored SAVE trial (NCT05360160) 4. The route for a small lab is therefore well-trodden: not a co-development deal, but a compound-supply and correlative-science agreement attached to someone else's protocol.

Second, the company's unanswered scientific problem is the one an academic mechanism lab is built to answer. AUGMENT-101 delivered a 22.8% CR+CRh rate with a 63.2% overall response rate in KMT2A-rearranged R/R leukaemia 5. The gap between those two numbers, and the fact that responses are often not durable, is a biology problem rather than a chemistry problem.

Third, the asymmetry of what each side lacks is clean. Syndax has patients and drug and no incentive to run deep single-cell or chromatin work on non-responders; a small lab has that capability and no patients.

The realistic caution. Syndax is a commercial-stage company with an approved product and a phase 3 running; its bandwidth for unsolicited academic approaches is finite, and anything touching resistance mechanisms in an approved drug is commercially sensitive. The practical entry point is through a trial investigator at a paediatric site already on AUGMENT-101, not through the company's business-development function.

What to read sceptically

Everything on the Syndax corporate site about pipeline breadth and differentiation is a claim by an interested party and should be treated as such. The verifiable core is narrow and is what this node cites: the FDA label text retrieved from openFDA 1, the ClinicalTrials.gov registry records including the minimum-age fields 234, and the peer-reviewed AUGMENT-101 report 5.

One substantive caveat on the registry data. Registry minimum-age fields describe what the protocol permits, not who was actually enrolled. A 30-day floor with weight-banded dosing (95 mg/m² for weight under 40 kg) does not establish that infants were enrolled in meaningful numbers — the published median age of 37 argues they were not 5. Anyone planning around paediatric revumenib exposure should treat the number of treated infants as an open question rather than an established fact.

Facts and figures
groupSyndax Pharmaceuticals, Inc. (clinical and commercial oncology organisation; menin inhibitor franchise)
institutionSyndax Pharmaceuticals, Inc., Waltham, Massachusetts, USA
focusMenin-KMT2A inhibition in KMT2A-rearranged, NPM1-mutant and NUP98-rearranged acute leukaemia; also axatilimab (anti-CSF1R) in chronic GVHD and IPF
stageCommercial-stage; revumenib FDA-approved, phase 3 frontline programme running
lead assetrevumenib (SNDX-5613), brand name REVUFORJ — reversible small-molecule menin-KMT2A interaction inhibitor
pediatric inclusivetrue
min age eligible30 days on AUGMENT-101 (NCT04065399) and AUGMENT-102 (NCT05326516); FDA label covers patients 1 year and older; frontline programme rises to 12 years (NCT07211958) and 18 years (NCT06226571)
approval statusFDA approved as REVUFORJ for (i) R/R acute leukemia with a KMT2A translocation and (ii) R/R NPM1-mutant AML with no satisfactory alternative, both in adult and pediatric patients 1 year and older
partnering angleNot a co-development deal. The realistic route is compound supply plus a correlative-science addendum attached to an existing investigator-initiated protocol at a paediatric AUGMENT-101 site, offering mechanism work on non-responders and on the gap between 63% ORR and 23% CR+CRh that the company has no in-house incentive to run.
key papers
Menin Inhibition With Rev…2,024Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101): 2024Revumenib for Relapsed or…2,025Revumenib for Relapsed or Refractory Acute Leukemia With a KMT2A Translocation: 2025Revumenib: a new era in a…2,025Revumenib: a new era in acute leukemia treatment: 2025
trials
[
  "NCT04065399",
  "NCT05326516",
  "NCT06226571",
  "NCT07211958",
  "NCT05406817",
  "NCT06177067",
  "NCT03013998",
  "NCT05360160"
]
key papers — the numbers behind the chart
[
  {
    "title": "Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101)",
    "year": 2024,
    "venue": "Journal of Clinical Oncology",
    "pmid": "39121437",
    "doi": "10.1200/JCO.24.00826"
  },
  {
    "title": "Revumenib for Relapsed or Refractory Acute Leukemia With a KMT2A Translocation",
    "year": 2025,
    "venue": "Annals of Pharmacotherapy",
    "pmid": "40437770",
    "doi": "10.1177/10600280251341279"
  },
  {
    "title": "Revumenib: a new era in acute leukemia treatment",
    "year": 2025,
    "venue": "Trends in Cancer",
    "pmid": "39863449",
    "doi": "10.1016/j.trecan.2025.01.006"
  }
]
How this could bite us back
  • An approved product makes a company less, not more, willing to share resistance data: anything that suggests the drug fails in a definable subgroup is commercially costly, so the most scientifically valuable material is the hardest to obtain.
  • The 30-day minimum age is a protocol permission, not evidence of infant experience; planning a collaboration around paediatric revumenib exposure that does not exist would waste a cycle.
  • Syndax's strategic centre of gravity is moving to frontline adult NPM1-mutant AML, where the market is large. KMT2A-rearranged infant disease is a small, awkward indication that already has its label; company attention to it can only decline.
  • Revumenib requires management of strong CYP3A4 interactions and carries QTc liability, which constrains the combination space an academic partner might want to explore.
Still open
  1. How many patients under 2 years of age have actually received revumenib on AUGMENT-101 or AUGMENT-102? The protocols permit enrolment from 30 days but the published median age of 37 years suggests the true infant denominator may be in single digits, and nobody has reported it.
  2. Why does the frontline programme exclude children when the relapsed programme does not? If this is a regulatory or backbone-design constraint rather than a safety one, a paediatric frontline study is a gap someone should fill.
  3. Does revumenib response in KMT2A-rearranged infant ALL differ from KMT2A-rearranged AML in the same age band? The registrational cohort pooled lineages and the abstract does not break this out.
  4. What fraction of the roughly 40% of patients who respond but do not reach CR+CRh carry pre-existing MEN1 interface variants versus a menin-independent transcriptional escape?
Every source behind this entry (6)
  1. E1established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.database

    REVUFORJ (revumenib) is FDA-approved for relapsed or refractory acute leukemia with a KMT2A translocation and for relapsed or refractory NPM1-mutant AML, in adult and pediatric patients 1 year and older.

    REVUFORJ (revumenib) US prescribing information, INDICATIONS AND USAGE, retrieved via the openFDA drug label API · US Food and Drug Administration; Syndax Pharmaceuticals, Inc. · openFDA drug label API 2026
    What this evidence cannot show

    The label is a regulatory document, not a study; it states what the drug is licensed for, not how well it works in any particular age band. The NPM1 indication carries the qualifier 'who have no satisfactory alternative treatment options'. Retrieved 2026-09-18; labels are revised and the indication text can change.

    Evidence that argues against this (1)
    • An approval down to age one year is a licensing decision and not evidence of experience in infants. The paediatric data supporting it is thin at the youngest ages, and the registrational efficacy estimate comes from a population whose median age is far above the beat's. A label boundary is what a regulator permits, not what has been studied. U.S. Food and Drug Administration 2026
  2. E2established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.trial_registry

    AUGMENT-101 (NCT04065399) is a Syndax-sponsored phase 1/2 study of revumenib in relapsed/refractory acute leukaemia, currently recruiting, with a registered minimum age of 30 days, target enrolment 447, and phase 2 cohorts covering KMT2A-rearranged ALL/MPAL, KMT2A-rearranged AML and NPM1-mutant AML.

    NCT04065399
    What this evidence cannot show

    Registry entries are sponsor-submitted and describe protocol eligibility, not realised enrolment. The 30-day floor applies to phase 1 and phase 2 cohorts 2A-2C only; cohort 2D requires age 18 and above and weight 40 kg and above. Record retrieved and read 2026-09-18.

    Evidence that argues against this (1)
    • A registry field is a snapshot with a date on it. Status, site count, target enrolment and eligibility are edited by sponsors between checks, and this graph has already recorded one trial whose status string changed under it. Anything planned on these numbers has to re-query them rather than read them here. A registered minimum age of 30 days is an eligibility field and does not report how many neonates or infants were actually enrolled, which is the number that decides whether infant-relevant on-treatment material exists. U.S. National Library of Medicine 2026
  3. E3established confidence 5 of 5: Multiple independent lines of evidence; treated as settled in the field.trial_registry

    AUGMENT-102 (NCT05326516), the phase 1 study of revumenib in combination with chemotherapy in relapsed/refractory KMT2A-, NPM1- and NUP98-altered leukaemias, is completed, enrolled 30 participants, and had a registered minimum age of 30 days.

    NCT05326516
    What this evidence cannot show

    n=30 is small and the study is a dose-escalation, so efficacy inference is not supported. Registry field values retrieved via the ClinicalTrials.gov API on 2026-09-18.

    Evidence that argues against this (1)
    • A registry field is a snapshot with a date on it. Status, site count, target enrolment and eligibility are edited by sponsors between checks, and this graph has already recorded one trial whose status string changed under it. Anything planned on these numbers has to re-query them rather than read them here. Thirty participants across KMT2A-, NPM1- and NUP98-altered leukaemias in a completed phase 1 leaves very few per lesion, so any lesion-specific reading of that study rests on single figures. U.S. National Library of Medicine 2026
  4. E4strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.trial_registry

    Syndax's frontline revumenib programme raises the minimum age: NCT06226571 (phase 1, revumenib plus intensive chemotherapy, newly diagnosed AML) requires age 18 and above, and the phase 3 NCT07211958 in newly diagnosed NPM1-mutant AML requires age 12 and above; separately, revumenib is supplied to investigator-led paediatric studies including the St. Jude-sponsored NCT06177067 (ages 1-30, Syndax listed as collaborator) and appears as a sub-study agent in the Beat AML master protocol NCT03013998.

    NCT07211958
    What this evidence cannot show

    Being listed as a collaborator on ClinicalTrials.gov does not disclose the terms of the arrangement; it may mean drug supply only, or funding, or neither exclusively. The registry does not state what Syndax contributes to NCT06177067. Retrieved 2026-09-18.

    Evidence that argues against this (1)
    • Qualifies the claim that Syndax is 'the single most plausible industry partner for a lab working on infant leukaemia'. The only dedicated menin-inhibitor trial in infant KMT2A-rearranged leukaemia is COG AALL2121 (NCT05761171), a phase 2 of revumenib plus chemotherapy in patients 1 month to under 6 years, and the registry record lists the Children's Oncology Group as sole sponsor with NO collaborators recorded at all, funded under NCI grant U10CA180886. Syndax is not a named party to it. Contrast the studies where Syndax is a listed collaborator (NCT06177067, St. Jude, ages 1 to 30) - the registry does name it there, so its absence from AALL2121 is informative rather than a recording gap. Two consequences for the partnering plan. First, the route by which revumenib actually reaches infants runs through NCI/CTEP and COG, not through Syndax business development, so a lab planning an infant revumenib correlative project should be negotiating with COG and the AALL2121 study chair. Second, and more cautionary: Syndax is not visibly invested in the infant cohort even though it exists, which is weak evidence against the entry's assumption that the company would welcome mechanism work on that population. Caveat: ClinicalTrials.gov collaborator fields are sponsor-submitted and a drug-supply agreement under an NCI CRADA need not be recorded as a collaboration, so absence from the field is suggestive, not conclusive. Read: registry record, retrieved 2026-09-18. Children's Oncology Group 2026
  5. E5strong confidence 4 of 5: Replicated in peer-reviewed work by more than one group.paper

    In the registrational phase 2 portion of AUGMENT-101, 94 patients with KMT2A-rearranged R/R acute leukaemia were treated (median age 37 years, range 1.3-75); among 57 efficacy-evaluable patients the CR+CRh rate was 22.8% and the overall response rate 63.2%.

    Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101) · Issa GC, Aldoss I, Thirman MJ, DiPersio J, Arellano M, Blachly JS, et al. · Journal of Clinical Oncology 2024
    PMID 39121437DOINCT04065399
    What this evidence cannot show

    Single-arm, open-label, no control group; the efficacy population (n=57) is a subset of the treated population (n=94) restricted to central confirmation, which inflates apparent precision. Median age 37 means the paediatric and infant experience inside this number is small and not separately reported in the abstract. Grade 3 or worse differentiation syndrome occurred in 16.0%. Sponsor employees are co-authors. According to PubMed; DOI https://doi.org/10.1200/JCO.24.00826

    Evidence that argues against this (1)
    • The most decision-relevant counter in this entry, and it bears on the project's central hypothesis rather than on Syndax. This item frames the 22.8% CR+CRh rate as revumenib's problem - 'the gap between those two numbers ... is a biology problem rather than a chemistry problem' - and the partnering angle is built on offering to explain it. A second, chemically distinct menin inhibitor produced essentially the same number in a different genotype. In the registrational phase 2 of KOMET-001, 92 adults with relapsed/refractory NPM1-mutant AML given ziftomenib 600 mg daily had a CR/CRh rate of 22%, an overall response rate of 33%, median overall survival 6.6 months and median duration of response 4.6 months. Revumenib in KMT2A-rearranged R/R disease: 22.8% CR+CRh. Two compounds with different scaffolds and different binding modes, two different driver genotypes, two different trial populations, and a complete-remission rate that lands within one percentage point. That pattern is more consistent with a ceiling imposed by disrupting this interface in relapsed disease than with a deficiency of either molecule, and if it holds it weakens the premise that a better menin-interface inhibitor, or a better understanding of why revumenib specifically underperforms, is where the gain lies. The lab's offer to Syndax should be framed around that class-level question, which the company cannot answer from its own single-compound data, and the lab's own thesis should treat the ~22% figure as a hypothesis about the interface rather than about a drug. Caveats: this is a cross-trial comparison of single-arm studies with different eligibility, different genotypes and different median ages (37 for AUGMENT-101, 69 for KOMET-001); overall response rates differ substantially (63.2% versus 33%), which argues the two are not measuring the same thing; and no formal comparison has been made. Read: abstract only. Wang ES et al. 2025
  6. E6moderate confidence 3 of 5: One strong study, or several smaller consistent ones.review

    Independent review literature confirms revumenib's FDA approval for R/R KMT2A-rearranged acute leukaemia on the basis of the AUGMENT-101 phase 2 cohort and reports grade 3/4 QT prolongation in 12% and differentiation syndrome in 29% of patients.

    Revumenib for Relapsed or Refractory Acute Leukemia With a KMT2A Translocation · Lynch EJ, Faro SJE, Lindstrom AM, Sethi NA, Wang WY, Seligson ND · Annals of Pharmacotherapy 2025
    PMID 40437770DOI
    What this evidence cannot show

    A narrative drug review, not primary data; adverse-event percentages differ slightly from the primary JCO report because they draw on a larger safety population (135 patients) and on label data rather than the registrational efficacy cohort. Reviews inherit the biases of the trials they summarise. According to PubMed; DOI https://doi.org/10.1177/10600280251341279

    Evidence that argues against this (1)
    • Grade 3/4 QT prolongation in 12% and differentiation syndrome are the numbers that matter for a paediatric plan and they were measured in a largely adult population with adult comorbidity and concomitant medication. The review restates the trial's own figures, so it is not independent confirmation of them. Journal of Clinical Oncology 2025
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