Syndax Pharmaceuticals (SNDX) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic milestones and product pipeline

• Positioned to become a commercial-stage company with two first-in-class assets: revumenib for KMT2A and NPM1 acute leukemia, and axetilimab for chronic graft-versus-host disease (GVHD).

• Revumenib has a PDUFA date at the end of September, with anticipated approval in Q3 2024; axetilimab's PDUFA is at the end of August.

• NPM1 indication data expected soon after initial approval, with a potential second approval in early 2025.

• Axetilimab is partnered with Incyte, targeting a Q4 launch and expansion into frontline settings.

• Early foray into solid tumors with ongoing phase I in MSS colorectal cancer, showing some disease stabilization.

Clinical data and product differentiation

• Revumenib pivotal trial in KMT2A patients showed a mid-60% response rate, with CR/CRh in the mid-20% range, outperforming standard of care.

• 40-50% of responders proceeded to transplant, with maintenance therapy enabling remissions up to three years.

• NPM1 phase I data: 36% CR/CRh, 50% overall response, 100% MRD negativity among responders, and 43% went to transplant.

• Combination studies with venetoclax and chemotherapy show high response rates and MRD negativity, supporting expansion into frontline and unfit populations.

• Axetilimab demonstrated a 74% overall response rate in third-line+ GVHD, with durable responses and a novel anti-fibrotic, anti-inflammatory mechanism.

Commercial strategy and market opportunity

• Combined KMT2A and NPM1 relapsed/refractory population estimated at 6,000 patients, with frontline potentially 1.5x larger.

• Estimated initial market opportunity for revumenib is ~$2 billion, assuming NPM1 launch.

• Launch plans include a focused sales force of 30-50, already fully staffed with experienced hematology/oncology reps.

• Commercial efforts target both hematology/oncology and transplant specialists, leveraging portfolio synergy and Incyte's expertise for axetilimab.

• Axetilimab revenue will be recognized as a 50/50 profit split with Incyte, reported as partnership revenue.