Syndax Pharmaceuticals, Inc. · NASDAQ:SNDX
Syndax Pharmaceuticals — J.P. Morgan Healthcare Conference 2023
22 slides from Syndax Pharmaceuticals's conference presentation, January 2023
Slide 1: Determined to realize a future in which people with cancer live longer and better
Determined to realize a future in which people with cancer live longer and better than ever before CORPORATE PRESENTATION | JANUARY 2023
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: Revumenib and axatilimab on-track for potential marketing applications in 2023
Revumenib and axatilimab on-track for potential marketing applications in 2023 with several opportunities for expansion Revumenib Received BTD Corporate Axatilimab for KMT2Ar and Menin-MLL acute leukemia Anti-CSF-1R Pipeline disruption Expand within acute leukemia and Expand into earlier lines of cG
Slide 4: Significant unmet need remains in acute leukemia
Significant unmet need remains in acute leukemia No FDA-approved therapies targeting KMT2Ar or mNPM1 acute leukemias BTD KMT2A Acute Leukemias mNPM1 Mutant AML Annual global incidence 5,000 - 7,000 Annual global incidence ~20,000 ~ 10% AML or ALL ~ 30% AML • Most frequent genetic alterations in AML
Slide 5: Revumenib turns off leukemic transcriptional programs by binding to Menin
Revumenib turns off leukemic transcriptional programs by binding to Menin and displacing KMT2A (MLL) complexes KMT2Ar or mNPM1 acute leukemias Menin inhibition with revumenib revumenib Menin Menin KMT2A* KMT2A* HOX Menin OFF HOX KMT2A* Leukemia MEIS OFF MEIS Differentiation / Apoptosis Gene transcri
Slide 6: AUGMENT-101: Phase 1/2 trial of revumenib in patients with acute leukemias
AUGMENT-101: Phase 1/2 trial of revumenib in patients with acute leukemias; pivotal trials enrolling Phase 1: Dose escalation Phase 2: Ongoing Pivotal Trials Arm A 339 mg (no strong CYP3A4i) q12h 276 mg* AUGMENT-101-2A: KMT2Ar ALL 226 mg* q12h q12h Adult and ped1 113 mg with R/R q12h AUGMENT-101-2B:
Slide 7: AUGMENT-101 patients heavily pretreated with a poor prognosis
AUGMENT-101 patients heavily pretreated with a poor prognosis Safety Population Safety Population Baseline Characteristics Baseline Characteristics N=68 N=68 Median age, years (range) 42.5 (0.8, 79) KMT2Ar, n (%) 46 (68) t(9;11) 10 (15) Adult (n=60) 50.5 t(11;19) 9 (13) Pediatric (n=8) 2.5 t(4;11) 6
Slide 8: No patients have discontinued due to treatment related adverse events
No patients have discontinued due to treatment related adverse events Safety Safety Any-grade treatment related AE Population ≥Grade 3 treatment related AE Population (≥5%) N=68 N=68 Patients with ≥1 treatment- Patients with ≥Gr 3 treatment- 53 (78) 11 (16) related AE, n (%) related AE, n (%) 10% of
Slide 9: Updated AUGMENT-101 data continues to support best-in-class profile for revumenib
Updated AUGMENT-101 data continues to support best-in-class profile for revumenib Efficacy Population Best Response1 n = 60 (%) • Median duration of CR/CRh Overall Response Rate2 32/60 (53%) response of 9.1 mos Response CR 12 (20%) • Median time to CR/CRh CRh 6 (10%) response of 1.9 mos CRp 5 (8%) •
Slide 10: Durable remissions in transplant patients treated in the Phase 1 portion
Durable remissions in transplant patients treated in the Phase 1 portion of AUGMENT-101 trial 12 patients proceeded to HSCT1 Patients who achieved MRDneg status 11/12 (92%) Remain in remission (1 receiving maintenance in CU2) 9/12 (75%) Remained in remission > 1 year 4/12 (33%) Median follow-up 12.3
Slide 11: Trials underway to establish revumenib as a backbone of treatment
Trials underway to establish revumenib as a backbone of treatment for mNPM1 or KMT2Ar acute leukemia Relapsed/Refractory Front-Line Maintenance Revumenib Development INTERCEPT trial Validates use of menin Validates the use of menin AUGMENT-101: allows pts to inhibition in NPM1 and inhibition with Tr
Slide 12: Revumenib expansion opportunities in acute leukemia and solid tumors
Revumenib expansion opportunities in acute leukemia and solid tumors have potential to add meaningful value Est. US market opportunity for Potential first/best-in-class agent mNPM1 and KMT2Ar AML Eligible • Clear activity in refractory, advanced patients 3 $4,000 12,000 mNPM1 and KMT2Ar acute leukem
Slide 13: Axatilimab: CSF-1R mAb with potential best-in-class profile
Axatilimab: CSF-1R mAb with potential best-in-class profile Axatilimab inhibition reduces pathogenic monocytes and macrophages CSF-1 CSF-1R Circulating Chronic graft versus host disease: Monocyte Blood vessel Circulating Monocyte • Develops in 40% of HSCT1; estimated US prevalence ~14,0002 CSF-1R CS
Slide 14: Phase 1/2 trial highlights the therapeutic benefit of axatilimab for R/R cGVHD
Phase 1/2 trial highlights the therapeutic benefit of axatilimab for patients with R/R cGVHD Phase 1: Dose escalation Phase 2: Expansion n = 17 patients Cohort 4 Enrolled n=6 cGVHD pts Dose: 1mg/kg q2wk progressed Cohort 3 3.0 mg/kg on 2 or Cohort 2 n=3 q2wks n= 23 patients more prior Cohort 5 thera
Slide 15: Baseline characteristics suggest heavily pre-treated population
Baseline characteristics suggest heavily pre-treated population Ph 1, n=17 Ph 2, n = 23 TOTAL ALL DOSES 1mg/kg Q2W N=40 Age, median (range),yrs 60 (29, 73) 57 (16, 69) 59 (16, 73) Female, n (%) 6 (35) 9 (39) 15 (38) Myeloablative transplant, n (%) 9 (53) 17 (74) 26 (65) Related Donor, n (%) 9 (53) 9
Slide 16: Axatilimab clinical profile demonstrates tolerability
Axatilimab clinical profile demonstrates tolerability AE Overview, No. (%) Any ≥ grade 3 AE in ≥ 2 patients, No. (%) Ph 1, n=17 Ph 2, n = 23 TOTAL Ph 1, n=17 Ph 2, n = 23 TOTAL ALL DOSES 1mg/kg Q2W N=40 ALL DOSES 1mg/kg Q2W N=40 Any AE 17 (100) 22 (96) 39 (98) Hypertension 3 (18) 1 (4) 4 (10) ≥ grad
Slide 17: Axatilimab Phase 1/2 trial showed rapid and durable responses
Axatilimab Phase 1/2 trial showed rapid and durable responses Phase 2 cohort Individual responses and durability of Cumulative response rate in highlights Phase 2 cohort cGVHD-involved organs ORR by cycle 82% 7 day 1 FFS at 12 77% months Median time 4 wks to response Responses observed across all or
Slide 18: Axatilimab responses accompanied by reduction in cGVHD symptom burden
Axatilimab clinical responses were accompanied by a reduction in cGVHD symptom burden and improvements in all affected organs Change in cGVHD symptoms from baseline in all evaluable patients, as measured by the Lee Symptom Scale Of evaluable patients had 58% a >7 point improvement in the Lee Symptom
Slide 19: Axatilimab pivotal trial enrollment complete; data expected in mid-23
: Axatilimab pivotal trial enrollment complete; data expected in mid-23 Inclusion criteria: • 2 years and older1 0.3 mg/kg Q2W Randomization • Recurrent or refractory active R/R cGVHD after ≥ 2 lines of cGVHD 1:1:1 1 mg/kg Q2W systemic therapy N = ~ 240 Stratification factors: 3 mg/kg Q4W • Prior th
Slide 20: Axatilimab has the potential to expand into additional high value indications
Axatilimab has the potential to expand into additional high value indications and new geographies Prevalence estimates ~215,000 US, Expansion into ~335,000 WW2 fibrotic indications: IPF POC* init 1H23 >35,000 WW1 Expansion into EU, Japan, ROW ~14,000 US1 Expansion to 1st L cGVHD: JAK combo trial 1Q2
Slide 21: Financial highlights and FY 2022 financial guidance
Financial highlights and FY 2022 financial guidance Ticker SNDX (NASDAQ) Cash and equivalents (as of September 30, 2022) $337.8 million Approximate net proceeds from 4Q22 follow-on offering $161.5 million Shares outstanding* (as of December 14, 2022) 69.2 million 2022 Operating Expense Guidance FY 2
Slide 22: Thank you. Questions?
Thank you. Questions? 22
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