First Quarter Financial Results Presentation / May 8, 2024
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1Q catalyst updates set the stage for a transformative 2024
1Q milestones
Granted Priority Review for revumenib NDA submission under RTOR for R/R KMT2Ar acute leukemia
Granted Priority Review for axatilimab BLA submission in chronic GVHD after failure of at least two prior lines of systemic therapy
Completed enrollment of mNPM1 AML cohort in revumenib pivotal AUGMENT-101 trial
Looking ahead in 2024
PDUFA action date of September 26, 2024, for revumenib, followed by launch
PDUFA action data of August 28, 2024, for axatilimab, followed by launch
Pivotal revumenib AUGMENT-101 data in mNPM1 AML in 4Q
Additional revumenib combination data in frontline and R/R patients
Phase 3 frontline trial initiations for axatilimab and revumenib
Update from Phase 1 metastatic CRC trial in 2Q
KMT2Ar, KMT2A rearrangement; mNPM1, mutated nucleophosmin; RTOR, Real-time oncology
review; R/R, relapsed or refractory; AML, Acute myeloid leukemia; GVHD, Graft-versus-host3 disease; CRC, Colorectal cancer
Pivotal AUGMENT-101 trial: KMT2Ar AML/ALL filing under Priority Review; potential filing for mNPM1 in 1H25
Phase 1 | Phase 2 | |
Dose Escalation | Ongoing Pivotal Trials | |
KMT2Ar | ||||
AUGMENT-101-2A: KMT2Ar ALL | ||||
Adult and ped1 | AUGMENT-101-2B:KMT2Ar AML | |||
with R/R KMT2Ar | RP2D2 | |||
or mNPM1acute | ||||
leukemia | mNPM1 | |||
AUGMENT-101-2C:mNPM1 AML | ||||
Primary endpoint
CR Rate (CR + CRh)
Expected Milestones
KMT2Ar | |
Enrollment completed | |
Topline data | |
Filing (RTOR) | |
mNPM1 | |
Enrollment complete | |
Topline data | 4Q24 |
Filing | 1H25 |
Note: Patients taken to HSCT can restart treatment with revumenib post-transplant; Abbreviations: KMT2Ar, KMT2A rearrangement; mNPM1, mutated nucleophosmin | ||
1 | Allows patients ≥30 days of age | 4 |
2 | 276mg q12h or 163mg q12h w/ strong CYP3A4 inhibitor | |
3 | Completed enrollment of a sufficient number of KMT2Ar patients to support a registration filing |
mNPM1 AML Phase 1 results suggest robust efficacy with durable, MRDneg responses
Phase 1
Dose Escalation
n (%) | |
Total mNPM1 @ RP2D | 14 |
CR/CRh | 5 (36%) |
MRDneg CR/CRh | 5 (100%) |
ORR | 7 (50%) |
No treatment related discontinuations
No grade 4 or 5 QTc events
Only differentiation syndrome ≤ grade 2 observed
3/7 | 1 |
(43%) of responders | patient restarted |
proceeded to HSCT | revumenib post HSCT* |
3/5 | TRAEs |
patients achieving | in-line with overall |
CR/CRh maintained | AUGMENT-101 Phase |
response beyond 6 | 1/2 experience |
months, 2 for >22 months |
Pivotal revumenib AUGMENT-101 data in
mNPM1 AML expected in 4Q24
- Data cutoff of July 24, 2023; 2023 amendment allowed patients to restart treatment with revumenib post-transplant following HSCT; mNPM1, Mutated nucleophosmin; HSCT, Haematopoietic stem cell transplant; RP2D, Doses that met
exposure equivalent of 226 mg q12h or 276mg q12h without strong CYP3A4 inhibitor or 113 mg q12h or 163 mg q12h with | 5 |
strong CYP3A4 inhibitor | |
Revumenib could provide significant benefit in mNPM1 and KMT2Ar acute leukemias across the treatment paradigm
mNPM1 & KMT2Ar
acute leukemiaFrontlineMaintenance treatment paradigm
Revumenib clinical development program (KMT2Ar and mNPM1 acute leukemia) - ongoing trials
Pivotal
BEAT AML | INTERCEPT | ||
Phase 1/2 | Rev + Ven/Aza | Rev Monotherapy Tx | |
Relapsed /
Refractory
AUGMENT-101
Rev Monotherapy
AUGMENT-102
Rev + Chemo
Rev + Intensive | Maintenance | SAVE | |
Rev + Ven + INQOVI® | |||
Chemo "7+3" | |||
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Axatilimab may be a practice-changing intervention for cGVHD
Unique MOA for cGVHD
- First agent to target disease- causing macrophages to impact fibrosis & inflammation
- Potential synergy with SOC
High and durable ORR
- 74% ORR at 0.3 mg/kg
- 60% of patients treated at 0.3 mg/kg remained in response at 12mo
Well tolerated supporting broad use
- Low rate of SAEs and discontinuations at 0.3 mg/kg
- Antibody reduces potential for DDIs versus small molecule competitors
Enrolled population reflects real world
- Efficacy observed in patients following treatment with current SOC
- Option to switch to Q4W dose at 6mo
cGVHD, Chronic graft-versus-host disease; MOA, Mechanism of action; SOC, Standard of care; DDI, Drug-drug interactions; ORR, Overall response rate; Q4W, Every 4 weeks; SAEs, Serious adverse events | 7 |
Pre-launch priorities to maximize launch potential
1 | Developing an efficient, effective and purpose- |
built infrastructure and customer-facing model | |
2 | Ensuring market access and patient support services are |
available at time of launch |
3 | Developing relationships with key stakeholders |
including payors and healthcare providers | |
4 | Delivering disease state awareness and education |
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Revumenib's profile supports use as backbone therapy across treatment continuum - providing access to >$4B US market opportunity
Significant growth potential with indications in earlier lines of treatment
Market Opportunity ($ B)
Est. patient population
~ $1.5 - $2B market
R/R
KMT2Ar + mNPM1
Acute Leukemia
5,000 - 6,500
~ $1B market
1L "Unfit"
KMT2Ar + mNPM1 Acute Leukemia
~3,500
~ $2B market
1L "Fit"
KMT2Ar + mNPM1 Acute Leukemia
~5,500
> $4B market
Total
Source: Data on file; Redbook 2023 | 9 |
Axatilimab has the potential to expand into additional high value indications and new geographies
2024 & Beyond
~215,000 US ~335,000 WW2
>35,000 WW1
~14,000 US1
Expansion into fibrotic indications: IPF POC* ongoing
Expansion into EU, Japan, ROW
R/R chronic GVHD: PDUFA action date set for August 28, 2024
Expansion into 1st L chronic GVHD: Jakafi® combo trial init 2024 Steroid combo trial init 2024
1 SmartImmunology Insights cGVHD report March 2020; 2 SmartImmunology Insights IPF report March 2020. * IPF trial will be conducted and funded by Syndax | 10 |
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Syndax Pharmaceuticals Inc. published this content on 08 May 2024 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 08 May 2024 11:56:02 UTC.