Roivant Sciences and Priovant Therapeutics executives outlined an expansion of the brepocitinib development program into lichen planopilaris (LPP) and discussed newly released Phase 3 results for batoclimab in thyroid eye disease (TED), which failed to meet its primary endpoint.
Brepocitinib expands into lichen planopilaris
Matt Gline, CEO of Roivant Sciences, said the companies are moving with “urgency” to broaden the potential of brepocitinib across multiple indications, focusing on orphan immunology diseases with high unmet need, mechanistic alignment with JAK1/TYK2 inhibition, and some level of clinical proof-of-concept.
Executives described LPP as a severe inflammatory scalp disorder that can cause scarring and generally irreversible hair loss. Gline said symptoms can include “itch, burning, redness, scaling,” and in many cases “intensely painful” disease. He emphasized there are no FDA-approved therapies and said patients often require chronic, aggressive multi-drug regimens with limited efficacy and poor tolerability. Gline estimated the condition may have “up to 100,000 U.S. patients,” calling it a “large orphan size.”
Ben Zimmer, CEO of Priovant Therapeutics, added that LPP is associated with “an increased risk of many severe comorbidities, including both skin cancer and other autoimmune diseases.” He said many off-label treatments are tried but are often discontinued due to tolerability and limited benefit.
Rationale: TH1-driven biology and existing proof-of-concept
Zimmer said Priovant believes LPP biology aligns with brepocitinib’s mechanism, describing LPP as “driven primarily by TH1 polarized T cell aberrant behavior.” He pointed to interferon-gamma and IL-12 as critical TH1 pathway cytokines and said a JAK1/TYK2 inhibitor is positioned to suppress signaling for both.
Zimmer also cited “case reports and investigator-initiated trials” involving JAK1 and TYK2 inhibitors as supportive clinical validation for the mechanism. He discussed a small, investigator-initiated, placebo-controlled trial of brepocitinib at Mount Sinai that used the LPPAI endpoint, which he characterized as noisy and not preferred by clinicians. While cautioning against overinterpreting the small dataset, he said the treated arm “clearly” improved over time.
Zimmer said biomarker data from that study was especially compelling, pointing to effects on “multiple markers of TH1-driven disease activity,” including interferon-gamma, IL-12, and chemokines such as CCL5.
Trial design: combined Phase 2b/3 with endpoint refinement
Gline said the LPP program is designed to function as a “straight to registrational” approach. The study includes a 72-patient Phase 2b portion, followed by a seamless transition into a Phase 3 portion expected to enroll approximately 270 patients, with a sample size re-estimation after Phase 2b. He said the structure is intended to support endpoint validation and regulatory alignment while maintaining development speed, but the company is “not ready to guide today” on enrollment timelines.
In Q&A, management described replacing LPPAI with more defined measures. Zimmer said LPPAI lacks standardized definitions for symptom scoring, contributing to variability. Priovant plans to use an Investigator’s Global Assessment (IGA) that measures erythema and scale with defined criteria, along with secondary endpoints evaluating symptoms such as pain and itch via numeric rating scales.
Zimmer said Priovant’s “base case” assumption is that the Phase 3 primary endpoint will be “IGA zero/one with two-point reduction,” while acknowledging the Phase 2b portion is meant to help understand endpoint behavior in a new indication. He added that the company plans to “wash patients out of background meds ahead of the enrollment quite aggressively,” consistent with other Priovant trials.
Batoclimab Phase 3 TED miss; read-through to Graves’ discussed
Gline also addressed Phase 3 TED results for batoclimab from Immunovant (NASDAQ:IMVT), describing the failure to meet the primary endpoint (a ≥2 mm proptosis responder rate) as “obviously disappointing.” He noted batoclimab had shown success in an earlier Phase 2 TED study but said this Phase 3 outcome does not support continued development in TED.
Gline emphasized that Immunovant’s future development focus is on IMVT-1402 rather than batoclimab, describing the TED readout as “effectively the last study to read out from the first generation program.” He said the TED trial design included 12 weeks of high-dose batoclimab followed by 12 weeks of lower dose, and he repeatedly pointed to better performance during the higher-dose period. Across endpoints, he said outcomes “got worse as you went from week 12 to week 24” after the dose step-down, which he framed as supportive of the importance of deeper IgG suppression.
In a pooled subset of about 20 hyperthyroid patients across the two studies, Gline reported a 75% mean IgG reduction and an 80% responder rate using the same thyroid hormone normalization definition the company used in a Phase 2 Graves’ disease study. He said the responder rate declined after 24 weeks as IgG suppression lessened, and he suggested the pattern reinforces the concept that “the deeper you can get IgG lower, the better you’re gonna do” in Graves’ disease.
Gline said hyperthyroid patients in the TED studies appeared to do “somewhat better” than the overall population on proptosis outcomes. He also noted that anti-thyroid drug doses were required to remain stable throughout the TED studies, limiting conclusions about dose reduction in clinical practice.
Looking ahead, Gline said the company expects both ongoing Graves’ studies with IMVT-1402 to read out next year and characterized enrollment as “generally going well.”
About Immunovant (NASDAQ:IMVT)
Immunovant Inc is a clinical-stage biopharmaceutical company focused on the development of novel monoclonal antibody therapies that target the neonatal Fc receptor (FcRn) to treat severe autoimmune diseases. By inhibiting FcRn, Immunovant’s approach is designed to reduce levels of pathogenic immunoglobulin G (IgG) antibodies, which play a central role in the pathology of disorders such as myasthenia gravis and immune thrombocytopenia.
The company’s lead asset, efgartigimod, is an engineered Fc fragment that selectively binds to FcRn, accelerating the degradation of circulating IgG.
