Keros Therapeutics (KROS) investor relations material

Core scientific and development focus

• Deep expertise in TGF-beta biology, focusing on druggable targets and relevant indications.

• Unique perspective on biologics in the TGF-beta space guides molecule and therapeutic area selection.

• KER-065 (rinvatercept) targets myostatin and activin to promote muscle regeneration while minimizing off-target effects.

• Development strategy pivoted from obesity back to neuromuscular diseases, especially DMD, due to strategic pipeline management.

• Rinvatercept aims to address muscle weakness, bone loss, and fat accumulation in DMD.

Clinical rationale and competitive landscape

• DMD selected for its balance of scientific rationale and feasible clinical endpoints within 12 months.

• Existing treatments like glucocorticoids and exon skipping offer limited functional benefit; gene therapy is not curative.

• Rinvatercept may provide additive or synergistic benefit with current standards of care.

• Phase I data showed good tolerability, increases in lean mass, reductions in fat mass, and improved bone mineral density.

• Dose titration strategy manages hemoglobin increases; early proof-of-concept will focus on safety and biomarker changes.

Clinical trial design and future plans

• Phase II will enroll late ambulatory and early non-ambulatory DMD patients, focusing on those on glucocorticoids.

• Early proof-of-concept expected within 3–6 months, with functional benefits assessed over 12 months.

• Data reporting cadence begins in 2027, starting with safety and biomarker data, followed by functional outcomes.

• Pivotal trial likely to be placebo-controlled, with estimated 100 patients, pending regulatory input.

• Market opportunity seen as additive to existing therapies, with potential for glucocorticoid sparing.