Keros Therapeutics (KROS) Leerink Global Healthcare Conference 2026 summary

Key 2025 execution highlights and forward outlook

• Transitioned elritercept program to Takeda, now advancing in Phase 3 second-line and frontline studies, with a myelofibrosis trial decision expected later this year.

• Cibotercept program discontinued due to safety signals; focus shifted to rinvatercept, which showed proof of mechanism in Phase 1.

• Rinvatercept Phase 1 data demonstrated increases in lean mass, bone mineral density, and reductions in fat mass, supporting advancement into DMD and ALS.

• ALS program selected for the ALS MyMatch consortium, leveraging expert trial infrastructure.

• Early pipeline expansion anticipated, with new assets expected to enter the clinic and runway extending into H1 2028.

Platform and pipeline strategy

• Focus remains on TGF-beta superfamily biology, with ligand traps targeting activin receptors for diverse indications.

• Pipeline includes systemic delivery ligands and multimodal inhibitors, aiming for broad therapeutic applications.

• Differentiation of rinvatercept lies in its ability to inhibit multiple negative regulators of muscle growth, unlike myostatin-only agents.

• Ligand traps show a distinct safety profile compared to receptor-targeting antibodies, avoiding certain adverse effects.

Rinvatercept clinical development in DMD and ALS

• Phase 2 DMD trial will enroll late ambulatory and non-ambulatory cohorts, focusing on safety, lean mass, fat mass, and bone mineral density endpoints.

• Early biomarker changes expected within 3-6 months; initial data could be available in 2027 as patients enroll on a rolling basis.

• ALS program targets slow-progressing patients, with exploratory endpoints including ALS functional score and muscle biomarkers.

• Preclinical ALS data showed improved muscle strength and weight maintenance in treated animals.

• ALS trial benefits from consortium selection, enabling efficient recruitment and expert oversight.