Faron Pharmaceuticals (FARON) investor relations material

Key learnings and clinical data

• Achieved high complete response (CR) rates in high-risk MDS, increasing from 28% to 45% over time, with durable responses exceeding 12 months and particularly strong results in TP53-mutated patients (CR 70%).

• Survival in last-line patients improved to 14.5 months, compared to historical 5–6 months, with notable transfusion independence and favorable safety profile.

• Negative results from the competitor VERONA trial (venetoclax plus AZA) highlighted the need for new approaches and validated the differentiated efficacy of BEX, especially in low blast count and complex mutation subgroups.

Regulatory and trial design updates

• FDA guidance now allows CR with duration as an approval endpoint in MDS, reducing reliance on overall survival (OS) due to confounding post-study treatments.

• Planned Phase II/III trial will use a staged approach: unblinded Phase II to confirm CR as the endpoint, followed by a more cost-efficient Phase III, reducing patient numbers and trial duration.

• This approach is supported by regulatory experts, key opinion leaders, and pharma partners, and is expected to mitigate known and unknown risks.

Business and partnering status

• Pharma partnering discussions have resumed with strong interest in the new development plan; previous offers were declined due to undervaluation.

• At least EUR 40 million is being raised for the next trial phase, with cost-saving options including open-label design and running trials in lower-cost countries.

• Market size for high-risk MDS remains unchanged, but competition is now limited after the failure of venetoclax.