Syndax Pharmaceuticals, Inc. · NASDAQ:SNDX
Syndax Pharmaceuticals — Q4 2023 Earnings Presentation
18 slides from Syndax Pharmaceuticals's earnings presentation, Feb 27, 2024
Slide 1: Fourth Quarter and Full Year 2023 Results
SNDX Fourth Quarter and Full Year 2023 Results / February 27, 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: Recent Accomplishments: Building momentum in 2024 off an impactful 4Q23
Recent Accomplishments Presented positive revumenib and axatilimab data @ ASH 2023: AGAVE-201, AUGMENT-101, SAVE, BEAT AML and AUGMENT-102 Completed revumenib NDA submission under RTOR for R/R KMT2Ar acute leukemia; expect to receive a PDUFA action data in 1Q24 Building Granted priority review and a
Slide 4: Revumenib KMT2Ar pivotal data establishes compelling clinical activity across acute leukemia patients
Revumenib 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 MRDn
Slide 5: Revumenib creates potential new opportunities for patients in R/R KMT2Ar acute leukemia
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% 39% 71% (n = 57) respond receive
Slide 6: mNPM1 AML Phase 1 results suggest robust efficacy with durable, MRDneg responses
mNPM1 AML Phase 1 results suggest robust efficacy with durable, MRDneg responses Phase 1 Dose Escalation 3/7 (43%) of responders proceeded to HSCT n (%) 1 patient restarted revumenib post HSCT* Total mNPM1 @ RP2D 14 CR/CRh 5 (36%) 3/5 patients achieving CR/CRh maintained response beyond 6 months, 2
Slide 7: Revumenib could provide significant benefit in mNPM1 and KMT2Ar acute leukemias across the treatment paradigm
Revumenib could provide significant benefit in mNPM1 and KMT2Ar acute leukemias across the treatment paradigm mNPM1 & KMT2Ar Relapsed / acute leukemia Frontline Maintenance Refractory treatment paradigm Revumenib clinical development program (KMT2Ar and mNPM1 acute leukemia) - ongoing trials Pivotal
Slide 8: BEAT AML: Ven/Aza + revumenib in frontline mNPM1 or KMT2Ar AML
BEAT AML: Ven/Aza + revumenib in frontline mNPM1 or KMT2Ar AML Summary of Enrolled Patients & Response Data n = 13 Safety Summary KMT2Ar: 5 Total enrolled mNPM1: 8 • No increased safety issues outside of Response and Transplant known reported ven/aza toxicities CRc 13 (100%) • Only 1 DLT (113 mg q12
Slide 9: SAVE AML: Ven/HMA combo in relapsed mNPM1, NUP98r or KMT2Ar AML/MPAL
SAVE AML: Ven/HMA combo in relapsed mNPM1, NUP98r or KMT2Ar AML/MPAL Summary of Enrolled Patients & Response Data Safety Summary N (%) Subtype • No discontinuations KMT2Ar: 5; mNPM1: 1 for TRAEs Total enrolled 9 NUP98r: 2 56% received prior ven • No ≥ Gr 3 QTc Median prior Tx 3 67% received prior HS
Slide 10: Revumenib's profile supports use as backbone therapy providing access to >$4B US market
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 11: AGAVE-201 results support axatilimab's promising safety and efficacy profile
AGAVE-201 results support axatilimab's promising safety and efficacy profile BLA Filing granted Priority Review by the FDA with a PDUFA action date of August 28, 2024 Met the primary endpoint in patients ORR by cycle 7 day 1 with R/R chronic GVHD 74% (95% CI [63, 83]) Durable responses with a reduct
Slide 12: Organ responses in 0.3 mg/kg Q2W
Organ responses in 0.3 mg/kg Q2W >85% of patients reported a reduction in chronic GVHD-related symptom burden 100 Complete response Partial response 80 13.0 Response rate, % 60 44% had 56.4 10.0 88.9 reduction in 40 81.8 BSA involved by 65.2 31.3 sclerosis 20.0 20 42.5 20.3 66% had 17.2 improvement
Slide 13: Anti-fibrotic effects of CSF-1R blockade extends to lung fibrosis
Anti-fibrotic effects of CSF-1R blockade extends to lung fibrosis Preclinical data indicates CSF-1R inhibition prevents pulmonary fibrosis by depletion of interstitial macrophages Saline control Bleomycin Bleomycin + Ab535 Axatilimab Phase 1/2 trial data in patients with bronchiolitis obliterans syn
Slide 14: Axatilimab Phase 2 trial in IPF trial now enrolling patients
Axatilimab Phase 2 trial in IPF trial now enrolling patients A 26-Week, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, and Tolerability of Axatilimab in Subjects with Idiopathic Pulmonary Fibrosis (IPF) PRIMARY ENDPOINT Axatilimab 0.3 mg/kg Q2W (N=
Slide 15: 2024 & Beyond: Axatilimab expansion into additional indications and geographies
Axatilimab has the potential to expand into additional high value indications and new geographies Expansion into fibrotic indications: ~215,000 US IPF POC* ongoing 2024 & Beyond ~335,000 WW2 Expansion into EU, Japan, ROW >35,000 WW1 R/R chronic GVHD: ~14,000 US1 PDUFA action date set for August 28,
Slide 16: Financial highlights and financial guidance
Financial highlights and financial guidance Ticker SNDX (NASDAQ) Cash and equivalents (31 December 2023) $600.5 M Shares outstanding* (31 December 2023) 85.1 M 2024 Operating Expense Guidance 1Q24 FY24 Research and development $56 – $62 M $240 – $260 M Total operating expenses^ $82 – $88 M $355 – $3
Slide 17: Expected upcoming clinical milestones
REVUMENIB Menin-KMT2A disruption • Approval and launch in R/R KMT2Ar acute leukemia in 2024 • Complete pivotal mNPM1 enrollment in late 1Q24/early 2Q24; data in 4Q24 • Update from Phase 1 metastatic CRC trial in 2Q24 • Additional data from revumenib Phase 1 combination studies (BEAT-AML, Expected SA
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