By Fineline Cube Ipsen S.A. (EPA: IPN, OTCMKTS: IPSEY) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for IPN60340 in combination with venetoclax and azacitidine (Ven‑Aza) for first‑line unfit acute myeloid leukemia (AML), an aggressive blood cancer affecting older adults.
Regulatory Milestone
| Item | Detail |
|---|---|
| Company | Ipsen S.A. (EPA: IPN, OTCMKTS: IPSEY) |
| Drug | IPN60340 (first‑in‑class monoclonal antibody) |
| Designation | Breakthrough Therapy Designation (BTD) |
| Agency | U.S. FDA |
| Indication | First‑line unfit AML in combination with Ven‑Aza |
| Mechanism of Action | Targets BTN3A, a key immune‑regulatory molecule broadly expressed across cancer |
| Previous Designations | Orphan Drug Designation (FDA & EMA, July 2025) |
| Clinical Data | Phase 1/2 EVICTION trial showed improved outcomes and favorable tolerability |
Clinical Evidence – Phase 1/2 EVICTION Trial
- Population: Patients with first‑line unfit AML
- Regimen: IPN60340 + venetoclax + azacitidine (Ven‑Aza)
- Key Findings:
- Well‑tolerated safety profile
- Improved clinical outcomes vs. historical Ven‑Aza controls
- Durable responses observed in heavily pretreated elderly population
- Next Steps: Ipsen plans to discuss Phase 2/3 development plans with FDA in H1 2026
Market Opportunity & Competitive Landscape
| Parameter | US | EU | Global |
|---|---|---|---|
| New AML Cases (2026E) | 21,000 | 18,000 | 75,000 |
| Unfit for Intensive Chemo | 12,600 (60 %) | 10,800 (60 %) | 45,000 (60 %) |
| Ven‑Aza Standard of Care | 70 % penetration | 65 % penetration | 68 % penetration |
| IPN60340 Addressable Market | 8,800 | 7,000 | 30,600 |
| Annual Cost (Ven‑Aza) | $180,000 | €150,000 | – |
| IPN60340 Peak Market Share | 0 % | 0 % | 18 % |
| Peak Revenue (2032E) | $285 million | $189 million | $824 million |
Key Competitors:
- Ven‑Aza (AbbVie/Roche) – Current standard of care for unfit AML
- Glasdegib + LDAC (Pfizer) – Alternative for unfit patients
- IPN60340 – First BTN3A‑targeted therapy with potential to enhance Ven‑Aza efficacy
Strategic Positioning
- First‑in‑Class Advantage: IPN60340 is the only BTN3A‑targeted antibody in clinical development for AML, offering a novel immune‑modulating mechanism
- Combination Strategy: BTD validates the IPN60340 + Ven‑Aza regimen, positioning it as a potential new standard for unfit AML
- Regulatory Pathway: BTD enables accelerated FDA review, priority meetings, and potential rolling submission
- Global Expansion: Ipsen plans EMA submission in H2 2026 following FDA BTD
Forward‑Looking Statements
This brief contains forward‑looking statements regarding clinical development timelines, regulatory pathways, and commercial projections for IPN60340. Actual results may differ due to clinical trial outcomes, competitive dynamics, and regulatory review processes.-Fineline Info & Tech