Oryzon Genomics (ORY) investor relations material

Financial and operational highlights

• Maintains strong liquidity, trading over $1 billion last year and securing $60 million in oversubscribed financing, providing runway through Q2 next year.

• Market cap fluctuates between EUR 250–280 million (~$300 million).

• Collaborations with the US National Cancer Institute and academic centers have enabled broad clinical development with modest financial outlay.

Oncology program and clinical progress

• Lead asset iadademstat targets AML and other hematological malignancies, showing high potency and favorable safety profile.

• Orphan drug designation granted by FDA and EMA; over 225 patients treated with strong tolerability.

• Recent trial in AML (ALICE-2) showed 100% response rate in first 15 patients, with 93% high-quality responses and 80% complete responses.

• Strategy focuses on high-risk subgroups (TP53, NRAS/KRAS/FLT3 mutations) for accelerated approval, aiming for a seamless Phase II/III trial with ~300 patients.

• Competitive advantages include broader patient applicability and better safety compared to menin and FLT3 inhibitors.

Market potential and commercial outlook

• Market analysis suggests pricing of EUR 250,000–350,000 per patient/year for TP53 subgroup, with broad payer acceptance.

• KOLs indicate willingness to prescribe across subgroups if efficacy and safety are maintained; initial US peak sales projected over $1 billion.

• Commercial launch targeted for 2029, with TP53 as initial driver and expansion to other subpopulations over time.