Geron (GERN) 25th Annual Needham Virtual Healthcare Conference summary

Company overview and strategic vision

• Commercial-stage hematology-oncology company launched first telomerase inhibitor, imetelstat, in mid-2024, targeting low-risk MDS patients, with initial focus on the U.S. market.

• Reported $184 million net revenue in 2025 and set guidance for $220–$240 million in 2026, supported by a $400 million balance sheet.

• Leadership emphasizes building a hematology powerhouse, leveraging a streamlined team and focused investments.

• Vision includes expanding beyond low-risk MDS into myelofibrosis and exploring further opportunities for telomerase inhibitors.

Product positioning and clinical differentiation

• Imetelstat is positioned as a second-line agent for low-risk MDS, with NCCN guidelines preferring it over HMAs and applicability in both RS positive and RS negative patients.

• Demonstrates disease-modifying activity, targeting mutated MDS clones, with cytopenia as an on-target effect linked to durable responses.

• Physician education is ongoing, especially in the U.S., to address initial unfamiliarity and optimize adoption in earlier lines of therapy.

• Real-world and clinical data show robust responses, particularly in RS negative and high transfusion burden patients.

Market dynamics and adoption strategies

• 70% of current use is in third-line or later, but strategy is shifting to focus on the 8,000-patient second-line U.S. population.

• Investments are being redirected from legacy functions to digital promotion, regional meetings, and targeted education to accelerate adoption.

• NCCN guideline updates and luspatercept’s move to frontline therapy create tailwinds for imetelstat’s second-line positioning.

• Ex-U.S. commercialization is under evaluation, with EMA approval secured and ongoing HTA and pricing work in Europe.