Roivant Sciences (ROIV) Study result summary

Program expansion and new study initiation

• Initiated a seamless phase II-B/III registrational trial for brepocitinib in lichen planopilaris (LPP), enrolling first subjects in March 2026, targeting a severe inflammatory scalp disorder with no FDA-approved therapies and high unmet need.

• LPP affects up to 100,000 U.S. patients, causes irreversible scarring hair loss, pain, and significant comorbidities, and is poorly responsive to current treatments.

• The trial design features a 72-patient phase II-B portion, followed by a phase III pivotal part with approximately 270 patients, using IGA 0/1 as the primary endpoint, and includes a double-blind period with open-label extension.

• Background medications will be aggressively washed out prior to enrollment to ensure clean efficacy assessment.

• The study leverages strong relationships with tertiary derm centers and overlapping prescriber bases, expected to move rapidly due to high investigator and patient enthusiasm.

Scientific rationale and supporting data

• LPP is driven by Th1-polarized T cell activity, aligning with brepocitinib’s dual JAK1/TYK2 inhibition mechanism and supported by efficacy in other Th1-driven diseases.

• Proof-of-concept data from a placebo-controlled IIT showed brepocitinib reduced disease activity, itch, and improved quality of life in LPP patients, with significant reduction in Lichen Planopilaris Activity Index and inhibition of Th1 markers.

• Biomarker data from a Mount Sinai study demonstrated clear suppression of interferon gamma, IL-12, and other Th1 markers with brepocitinib.

• The new trial will use more precise and clinically meaningful endpoints than previous studies, aiming to reduce measurement noise.

Batoclimab phase III study results in thyroid eye disease (TED)

• Two phase III studies of batoclimab in TED did not meet their primary endpoint of >2mm proptosis responder rate at week 24, with proptosis responder rates in the low-20% range for batoclimab versus high teens for placebo.

• Greater improvement was observed in the first 12 weeks (high-dose) than in the subsequent 12 weeks (lower dose), with efficacy declining as IgG suppression was reduced.

• Hyperthyroid patients in the TED study showed better proptosis response and thyroid normalization rates, consistent with prior Graves’ disease studies.

• Batoclimab demonstrated consistent efficacy in both proptosis and thyroid function endpoints in hyperthyroid subpopulations.

• Safety profile remained consistent with previous studies, with no new safety signals.