Roivant Sciences’ Brepocitinib Delivers Positive Phase III Uveitis Results

Key Points

• Positive Phase III results: Roivant’s brepocitinib met the primary endpoint and all secondary endpoints in the CLARITY-1 and CLARITY-2 trials for noninfectious uveitis. In pooled results, treatment failure occurred in 33.5% of brepocitinib patients versus 78% on placebo over 48 weeks.
• Steroid-free disease control and symptom improvement: More than 60% of patients in one study achieved steroid-free quiescence, while the drug also improved macular edema, imaging outcomes and patient-reported visual function compared with placebo.
• Regulatory plans: Roivant reported a well-tolerated safety profile and plans to submit a supplemental new drug application for noninfectious uveitis in the first half of 2027. The company is also developing brepocitinib for cutaneous sarcoidosis and lichen planopilaris.

Roivant Sciences NASDAQ: ROIV reported positive Phase III results from its CLARITY-1 and CLARITY-2 studies evaluating brepocitinib, which the company referred to as Brepo, in noninfectious uveitis (NIU), an inflammatory eye disease that can result in vision loss.

Matt Gline, Roivant’s chief executive officer, said the trials met their primary endpoint of time to treatment failure as well as all secondary endpoints in each individual study and in pooled analyses. The studies compared brepocitinib with placebo over a 48-week treatment period.

“We got the largest efficacy benefit ever seen for a non-steroidal therapy,” Gline said during the company’s call, while noting that comparisons with prior studies of other treatments are cross-trial comparisons and subject to limitations.

Primary Endpoint and Steroid-Free Disease Control

In both CLARITY trials, the median time to treatment failure was not reached among brepocitinib-treated patients by the end of the 48-week study period, because more than half of those patients had not experienced treatment failure. In comparison, median time to treatment failure was approximately 12 weeks in one placebo study and 13 weeks in the other, according to Roivant.

The company said the studies used an aggressive corticosteroid taper. Patients began treatment with 60 milligrams of steroids for two weeks and were fully tapered off steroids by week eight.

In pooled data, 33.5% of brepocitinib-treated patients experienced treatment failure by the end of the study, compared with 78% of placebo-treated patients. Roivant said the benefit was consistent across subgroups, including patients previously treated with tumor necrosis factor, or TNF, therapies.

Gline said roughly one-third of patients enrolled across the trials had prior TNF exposure. In the subset of patients with prior TNF experience, the company said brepocitinib’s treatment-failure results were comparable with results in the broader trial population.

Roivant also highlighted steroid-free quiescence, a measure of disease control in which patients have a “quiet eye” without corticosteroid treatment. The company said more than 60% of patients achieved steroid-free quiescence in one study, compared with roughly 20% of placebo patients.

Macular Edema, Imaging and Patient-Reported Outcomes

Roivant said the treatment demonstrated separation from placebo on fluorescein angiography measures, an imaging approach used by physicians to monitor uveitis. The company also reported clinically meaningful resolution of macular edema among patients who entered the studies with the condition.

About one-third of the study population had macular edema at baseline. Of those patients, Roivant said approximately 40% in CLARITY-1 and slightly more than 40% in CLARITY-2 achieved clinically meaningful resolution by the end of the study, compared with low rates in the placebo groups.

Ben Zimmer, chief executive officer of Priovant, said macular edema results could represent a differentiating feature versus adalimumab and other TNF therapies used off-label in the disease. However, he said it remains to be determined what information could be included in a potential product label.

The company also reported improvement versus placebo in a visual function questionnaire beginning at week six in both trials. The patient-reported measure included domains such as general vision, pain, near and distance activities, mental health, dependency and driving.

Safety, Regulatory Plans and Commercial Discussion

Gline said brepocitinib’s safety profile was well tolerated and consistent with prior studies of the drug, the JAK inhibitor class and the existing label for LISRAYA. He said patients with NIU often otherwise rely on high doses of background steroids, which can be difficult to use over extended periods.

Roivant plans to submit a supplemental new drug application for brepocitinib in NIU during the first half of 2027. The company did not provide details regarding potential review timing, saying it would update investors after submission acceptance and receipt of a PDUFA date.

Gline said NIU is the third leading cause of blindness in the United States and described a substantial need for new treatment options. He cited estimates of between 70,000 and 190,000 patients with posterior or pan-uveitis from various causes, while later noting that approximately 50,000 patients are systemically treated today.

The company expects to discuss the NIU commercial opportunity, patient population and physician-call strategy in greater detail at an Investor Day scheduled for Dec. 9 in New York City. Gline said a future NIU launch would require a commercial effort distinct from the organization supporting LISRAYA in dermatomyositis, though some patient-support infrastructure could be reused.

Roivant said brepocitinib is also being developed in other indications, including cutaneous sarcoidosis and lichen planopilaris, with both programs enrolling patients.

About Roivant Sciences (NASDAQ:ROIV)

Roivant Sciences Ltd. NASDAQ: ROIV is a biopharmaceutical company focused on discovering, developing and commercializing medicines. The company organizes its activities through subsidiaries and partner companies, often referred to as “Vants,” which specialize in particular therapeutic areas and stages of drug development.

Roivant's research and development activities have included immunology, dermatology, women's health, rare diseases and other specialty-care fields. Its portfolio has included Vtama (tapinarof) cream, a nonsteroidal topical treatment approved in the United States for plaque psoriasis and atopic dermatitis.

This instant news alert was generated by narrative science technology and financial data from MarketBeat in order to provide readers with the fastest reporting and unbiased coverage. Please send any questions or comments about this story to contact@marketbeat.com.