Roivant Sciences (NASDAQ:ROIV) outlined plans to expand development of its JAK1/TYK2 inhibitor brepocitinib into lichen planopilaris (LPP) and provided an update on a Phase III thyroid eye disease (TED) trial for batoclimab during a conference call hosted by the company.
Brepocitinib expands into lichen planopilaris
Roivant CEO Matt Gline said the company is moving “with urgency” to broaden brepocitinib across multiple indications, focusing on orphan immunology diseases with high unmet need, aligned biology for dual JAK1/TYK2 inhibition, and limited or no approved options. Alongside existing programs in dermatomyositis, non-infectious uveitis, and cutaneous sarcoidosis, Gline announced LPP as a new addition, describing it as “a fourth leg to the stool.”
Management noted the company views LPP as an “orphan-sized” opportunity, with Gline estimating “probably up to 100,000 U.S. patients,” and said the literature suggests prevalence and diagnosis are increasing over time.
Priovant details disease burden and biological rationale
Ben Zimmer, CEO of Priovant, said LPP carries substantial burden beyond scalp symptoms, citing an association with increased risk of severe comorbidities, “including both skin cancer and other autoimmune diseases.” He added that clinicians often attempt multiple off-label therapies but that outcomes are limited and discontinuations are common due to tolerability and efficacy.
Zimmer said Priovant believes brepocitinib is well matched to LPP biology, describing the condition as driven primarily by “Th1 polarized T cell aberrant behavior.” He noted that interferon gamma and IL-12 are critical Th1 cytokines and that a JAK1/TYK2 inhibitor can suppress signaling of both. Zimmer pointed to experience in cutaneous sarcoidosis—another Th1-driven condition studied with brepocitinib—as supportive of the mechanistic fit.
Zimmer also referenced case reports and investigator-initiated studies of JAK1 and TYK2 inhibitors as clinical validation for the mechanism. He discussed a small, placebo-controlled investigator-initiated trial at Mount Sinai that used the LPPAI endpoint, which he described as “a generally noisy instrument” that is not preferred by clinicians. While urging caution about overinterpreting a small dataset, Zimmer said the broader takeaway supported proof-of-concept and helped underpin the company’s decision to move quickly.
Zimmer emphasized biomarker findings from that study, saying the “most powerful” aspect was evidence of brepocitinib activity on multiple markers of Th1-driven inflammation, including interferon gamma, IL-12, and chemokines such as CCL5.
Combined Phase IIb/III trial underway; IGA endpoint planned
Gline said the LPP program has effectively begun as a “direct to registrational combined Phase IIb/III program,” with the study getting underway “last month.” He described a 72-patient Phase IIb portion that will transition immediately into a pivotal Phase III portion under a largely continuous design. The Phase III sample size is expected to be approximately 270 patients, with a sample size re-estimation after Phase IIb.
Management said the design is intended to support endpoint validation and regulatory alignment, while maintaining a pace closer to a straight-to-registrational program. Gline said the company is not yet providing enrollment timeline guidance but noted enthusiasm from investigators and patient communities.
On endpoints, Zimmer said Priovant is using a more structured approach than LPPAI. He described LPPAI as a composite measure with both physician-assessed and patient-reported components, but with limited definitions for raters. The company’s approach will include an investigator global assessment (IGA) focused on erythema and scale using defined criteria, with secondary endpoints to measure symptoms such as pain and itch using numerical rating scales.
In Q&A, Gline and Zimmer indicated the Phase IIb primary endpoint is expected to be an IGA 0/1 response with a two-point reduction, and said they expect—though cannot fully confirm until FDA discussions after Phase IIb—that the Phase III primary endpoint will match. Zimmer noted that placebo rates for rigorous IGA endpoints in inflammatory skin disorders tend to be low, particularly when requiring improvement to 0/1.
On background therapies, Gline said medications were washed out pre-baseline in the Mount Sinai investigator-initiated trial and highlighted that LPP patients often have polypharmacy. Zimmer said the registrational program plans to wash patients out of background medications “quite aggressively,” consistent with Priovant’s approach in other trials.
Batoclimab Phase III TED trial misses primary endpoint; Graves’ signals discussed
Gline also addressed topline Phase III results in TED for batoclimab, Immunovant’s first-generation anti-FcRn antibody, stating the studies “failed to meet their primary endpoint.” He said the TED program was effectively the last readout for the first-generation molecule, with future development focused on IMVT-1402.
The TED primary endpoint was a ?2 mm proptosis responder rate, which the trial did not achieve. Gline said the company is not pursuing further progress in TED with batoclimab, while noting the dataset provided scientific insights. He described the trial design as 12 weeks of high-dose batoclimab aimed at deep IgG suppression followed by 12 weeks of lower dosing. He said performance was generally better during the initial 12-week high-dose period than during the subsequent lower-dose period, a pattern he said was consistent across endpoints.
Gline said the trial showed “meaningful numerical separation” from placebo on change in proptosis at week 12, and noted that when pooling two TED studies, that measure was “nominally significant” in a post-hoc analysis, while emphasizing the limitations of such analyses. He added that proptosis improvements diminished between weeks 12 and 24 after dose reduction.
Management also highlighted results in a small subset of hyperthyroid patients included within TED enrollment criteria. Gline said there were about 20 hyperthyroid patients across active treatment arms in the pooled dataset. He reported a 75% mean IgG reduction and an 80% responder rate by a thyroid hormone definition (T3 and T4 below the upper limit of normal without increased antithyroid drug dosing), which he said matched the responder rate observed in a prior Phase II Graves’ study at week 12. He also said responder rates declined in the second 12-week period as IgG suppression lessened.
In response to analyst questions, Gline said hyperthyroid patients in the TED studies performed “somewhat better” on proptosis than the overall TED population. He also noted that antithyroid drug dose titration was not allowed in TED, limiting insight into real-world dose adjustments in that setting.
Gline said enrollment in the ongoing IMVT-1402 Graves’ program is “going well,” and reiterated expectations that both Graves’ studies will read out next year. He noted the Graves’ program excludes moderate to severe TED patients and is focused on endocrinology-driven sites, though the company expects to assess ocular symptom outcomes in Graves’ as well.
About Roivant Sciences (NASDAQ:ROIV)
Roivant Sciences is a biopharmaceutical company focused on the development and commercialization of innovative therapies through a network of subsidiary businesses known as “Vants.” Founded in 2014, Roivant acquires or in-licenses clinical-stage assets that have progressed beyond proof of concept and seeks to advance them efficiently toward regulatory approval. By organizing each program into a dedicated subsidiary, the company aims to streamline decision-making, allocate resources more effectively, and accelerate development timelines.
The core activities of Roivant involve identifying promising drug candidates across a range of therapeutic areas, including neurology, rare diseases, immunology, oncology, and women’s health.
