Keros Therapeutics (KROS) investor relations material

Strategic and operational updates

• Partnered most advanced asset and transitioned it to Takeda, with completion expected by year-end.

• Deprioritized Suborosev in PAH after safety concerns and stopped further development.

• Initiated a strategic review, resulting in a $375 million capital return and company restructuring, reducing headcount by half.

• Focused efforts on KER-065 as the main clinical asset and a preclinical pipeline for future value creation.

• Maintains sufficient capital post-return to fund KER-065 in DMD and a second neuromuscular indication, with runway into 2028.

KER-065 program and clinical rationale

• KER-065 aims to increase muscle regeneration and strength in Duchenne muscular dystrophy (DMD), addressing muscle breakdown and inflammation.

• Demonstrated preclinical and clinical benefits: increased muscle mass, reduced fat, improved bone mineral density, and enhanced utrophin/dystrophin expression.

• Differentiated from prior myostatin-only inhibitors by targeting activin as well, with improved safety profile and no observed bleeding events.

• Early clinical data show functional benefit in late ambulatory DMD patients, with no ambulation loss over five months.

• Dose titration planned to manage hemoglobin increases, with patient population and disease stage guiding trial design.

Clinical development plans and regulatory engagement

• Phase 2 trial design likely open-label, focusing on late ambulatory and non-ambulatory DMD patients.

• Sample size expected to be larger than 12 but less than 60, referencing prior ACE-031 studies.

• Regulatory submissions underway following study closure and new bone data presentation at ASBMR.

• Sequencing of additional indications planned, with DMD as the initial focus due to resource constraints.

• Broader neuromuscular and bone disorder opportunities considered for future expansion as resources allow.