Rallybio (RLYB) 7th Annual Evercore HealthCONx Conference summary

Portfolio overview and recent progress

• Four rare disease programs in development: lead program for a fetal hematological condition, complement C5 inhibitor, Matriptase-2 antibody for iron overload, and an oral therapy for hypophosphatasia.

• Significant turnaround in the portfolio over the past year, with notable advances in all programs.

Complement C5 inhibitor program update

• Manufacturing improvements eliminated host cell protein impurities, resolving prior tolerability issues.

• Enhanced assay methods confirmed 99% knockdown of free C5, aligning with efficacy expectations.

• Next steps include adaptive dosing in healthy volunteers and plans to study PNH patients, with data expected in the second half of next year.

• Additional indications under consideration include antiphospholipid syndrome and generalized myasthenia gravis.

Matriptase-2 antibody for iron overload

• Inhibits Matriptase-2 to increase hepcidin, reducing iron absorption and release, targeting transfusion-dependent patients.

• Regeneron’s clinical progress validates the mechanism; internal engineering aims for best-in-class duration of action.

• Preclinical data supporting the improved molecule to be presented at ASH.