Syndax Pharmaceuticals, Inc. · NASDAQ:SNDX
Syndax Pharmaceuticals — JP Morgan Healthcare Conference 2024
24 slides from Syndax Pharmaceuticals's conference presentation, Jan 8, 2024
Slide 1: Corporate Presentation at the JP Morgan Healthcare Conference
Corporate Presentation at the JP Morgan Healthcare Conference / January 8, 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: Syndax: An oncology company with 2 near-commercial heme assets
Syndax: An oncology company with 2 near-commercial heme assets Significant Presence at ASH 2023 Two clinically potential first- and best-in-class hematology assets • Revumenib, novel menin inhibitor, targeting KMT2Ar and mNPM1 acute leukemia • Axatilimab, first CSF-1R mAb targeting cGVHD Safety and
Slide 4: Revumenib – Menin Inhibitor
Revumenib – Menin Inhibitor Potential approval in adult and pediatric R/R KMT2Ar acute leukemia in 2024 with opportunities for additional indications
Slide 5: Despite recent advances in AML and ALL, treatment options are needed
Despite recent advances in AML and ALL, treatment options are needed for patients with KMT2Ar and mNPM1 acute leukemias KMT2Ar Acute Leukemia NPM1 Mutant AML 10% of AML or ALL1 30% of AML2 Revumenib has demonstrated positive clinical results in both Most patients relapse after Most frequent genetic
Slide 6: Revumenib is a potential first- and best-in-class therapy for KMT2Ar and mNPM1 acute leukemia
xxx Revumenib is a potential first- and best-in-class therapy for KMT2Ar and Response mNPM1 acute leukemia Revumenib monotherapy results to date: Durability MRD-negative • Clinically meaningful efficacy in R/R KMT2Ar and mNPM1 acute leukemia Patient • Durable responses observed in post-transplant ma
Slide 7: Pivotal AUGMENT-101 trial: Topline data supported NDA filing
Pivotal AUGMENT-101 trial: Topline data supported NDA filing for KMT2Ar AML/ALL under RTOR in 4Q23; potential filing for mNPM1 in 1H25 Phase 1 Phase 2 Dose Escalation Ongoing Pivotal Trials Expected Milestones KMT2Ar KMT2Ar Enrollment completed AUGMENT-101-2A: KMT2Ar ALL Topline data AUGMENT-101-2B:
Slide 8: KMT2Ar pivotal data establishes compelling clinical activity
KMT2Ar pivotal data establishes compelling clinical activity across acute leukemia patients; drives durable, MRDneg responses Overall response rate 63% in pooled analysis Enables a high rate of deep, durable MRDneg CR/CRh rate responses in late line R/R patients 23% in pooled analysis MRD negative r
Slide 9: Revumenib creates potential new opportunities for patients
Revumenib creates potential new opportunities for patients – enabling a meaningful shift in the treatment of R/R KMT2Ar acute leukemia Anticipated KMT2Ar treatment paradigm pending approval of revumenib Revumenib Revumenib HSCT maintenance Relapsed or Refractory 63% respond 39% receive 71% received
Slide 10: mNPM1 AML: Phase 1 data suggest robust efficacy
xxx mNPM1 AML: Phase 1 data suggest robust efficacy Pivotal trial enrollment ongoing Phase 1 Dose Escalation n (%) • 3/7 responders proceeded to HSCT Total mNPM1 @ RP2D 14 physician utilization • 1 patient restarted revumenib post PositionedCR/CRh to drive meaningful clinical5 (36%) benefit and HSCT
Slide 11: Beat AML: Ven/Aza + revumenib in newly diagnosed mNPM1 or KMT2Ar AML
Beat AML: Ven/Aza + revumenib in newly diagnosed mNPM1 or KMT2Ar AML Summary of Enrolled Patients & Response Data Safety Summary N=13 KMT2Ar: 5 • No increased safety issues outside of known Total enrolled reported ven/aza toxicities mNPM1: 8 Response and Transplant • 1 Hematologic DLT observed in DL
Slide 12: Save AML: Ven/HMA combo in relapsed mNPM1, NUP98 or KMT2Ar AML/MPAL
Save AML: Ven/HMA combo in relapsed mNPM1, NUP98 or KMT2Ar AML/MPAL Summary of Enrolled Patients & Response Data Safety Summary N (%) Subtype Total enrolled 9 KMT2Ar: 5; mNPM1: 1 • No discontinuations for TRAEs, No ≥ Gr 3 QTc NUP98r: 2 56% received prior VEN • Myelosuppression comparable to venetocl
Slide 13: Revumenib could provide significant benefit across the treatment paradigm
Revumenib could provide significant benefit in mNPM1 and KMT2Ar acute leukemias across the treatment paradigm mNPM1 & KMT2Ar acute leukemia Frontline Maintenance Relapsed / Refractory treatment paradigm Revumenib clinical development program (KMT2Ar and mNPM1 acute leukemia) AUGMENT-101 Pivotal Rev
Slide 14: Revumenib supports >$4B US market opportunity
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 ~ $2B market > $4B market Market Opportunity ($ B) ~ $1B market ~ $1.5 - $2B market R/R 1L “Unf
Slide 15: Axatilimab – anti-CSF-1R
Axatilimab – anti-CSF-1R Potential approval in patients with refractory chronic graft-versus-host disease in 2024 with opportunities for additional indications
Slide 16: The growing cGVHD market presents an attractive opportunity
The growing cGVHD market presents an attractive opportunity The estimated global chronic GVHD market is expected to expand due to1: 14,000 50% • Rising prevalence of blood patients living of patients require cancers boosted by an with cGVHD treatment beyond increase in the aging in the US1 systemic
Slide 17: Axatilimab is positioned to address what physicians and patients are seeking
Axatilimab is positioned to address what physicians and patients are seeking in a new cGVHD therapy Durable Axatilimab Key responses Attributes • Demonstrates compelling clinical profile Symptomatic Safe and • Unique MOA in cGVHD improvement tolerable Patient • Benefits in fibrotic and inflammatory
Slide 18: Pivotal AGAVE-201 trial design
Pivotal AGAVE-201 trial: A global pivotal trial designed to identify an optimal dose of axatilimab in chronic GHVD patients 0.3 mg/kg Q2W* Inclusion criteria: Stratification factors: Randomization R/R • 2 years and older1 • Prior treatment with 1:1:1 could provide a differentiated, ibrutinib, practi
Slide 19: At 0.3 mg/kg every 2 weeks, responses were durable
At 0.3 mg/kg every 2 weeks, responses were durable and accompanied by a reduction in symptom burden Met the primary endpoint in patients ORR by cycle 7 day 1 74% (95% CI [63, 83]) with R/R cGVHD Durable responses with a reduction in Of responders maintained symptom burden 60% a response at 1 year We
Slide 20: Organ responses in 0.3 mg/kg Q2W
Organ responses in 0.3 mg/kg Q2W Responses notable in fibrosis-dominated organs, including esophagus (78%), joints and fascia (76%), lung (47%), and skin (27%) 100 Complete response Partial response 80 13.0 Response rate, % 44% had 60 reduction in BSA involved by 56.4 10.0 sclerosis 88.9 40 81.8 65.
Slide 21: 2024 & Beyond
Axatilimab has the potential to expand into additional high value indications and new geographies Expansion into fibrotic indications: ~215,000 US IPF POC* initiated in 4Q23 ~335,000 WW2 2024 & Beyond Expansion into EU, Japan, ROW >35,000 WW1 R/R chronic GVHD: ~14,000 US1 BLA submitted YE23 2024 Pot
Slide 22: Financial highlights
Financial highlights Ticker SNDX (NASDAQ) Cash and equivalents (30 Sept 2023) $379.3 million # Approximate net proceeds from follow-on offering and ATM in 4Q23 $258.0 million # Shares outstanding* (19 Dec 2023) 84.8 million 2023 Operating Expense Guidance FY23 Research and development $160 - $165 mi
Slide 23: Expected upcoming clinical milestones
REVUMENIB Menin-KMT2A disruption • Potential approval in adult and pediatric R/R KMT2Ar acute leukemia in 2024 • Initiate combination trial with intensive chemo (7+3) in 1Q24 Expected • Phase 1 metastatic CRC data from dose escalation phase in 1H24 upcoming • Complete pivotal mNPM1 enrollment in lat
Slide 24: TON
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