| Presentation preference | Oral presentation |
| Title | Safety and Efficacy of Brepocitinib, a TYK2/JAK1 Inhibitor, in Active Non-Infectious Uveitis: 52-Week Results from a Phase 2 Study (NEPTUNE) |
| Accept poster if oral is not possible ? | No |
| Purpose | To assess the safety and efficacy of oral brepocitinib for the treatment of active non-infectious intermediate, posterior, and panuveitis (NIU) |
| Methods | Participants (n=26) with active, inflammatory chorioretinal/retinal vascular lesions or ≥ 2+ vitreous haze (VH) were randomized 2:1 to receive brepocitinib 45 mg or 15 mg once daily for 52 weeks, and prednisone 60 mg/day for 2 weeks that was tapered over the subsequent 6 weeks. Efficacy was assessed via the treatment failure (TF) rate, macular edema (ME), and fluorescein angiography (FA) scoring. |
| Results | TF rate in the 45 mg and 15 mg dose arms were 29% and 44% at Week 24, and 35% and 56% at Week 52, respectively. In each treatment arm, only one additional patient failed from Week 24 to 52. The resolution of ME in 43% of participants and the prevention of ME in 100% of participants without ME at baseline in the 45 mg arm at Week 24 were sustained through Week 52. No participant in the 15 mg arm with ME at baseline had resolution of ME at Week 52. Dose-dependent improvements in FA scores at Week 24 were sustained through Week 52. The adverse event rate was similar between arms. There were no deaths or cardiovascular, thromboembolic, or malignancy events. |
| Conclusion | In an active NIU population with a high expected rate of treatment failure, oral brepocitinib demonstrated dose-dependent, clinically-meaningful efficacy across multiple endpoints that was sustained through 52 weeks, with a safety profile consistent with prior brepocitinib studies. The safety and efficacy of brepocitinib in NIU is being confirmed in the ongoing phase 3 trial (CLARITY). |
| Conflict of interest | Yes |
| Details of conflicting interests | Professor Nguyen serves as a scientific advisor for Priovant. |
1
| Last name | NGUYEN |
| Initials of first name(s) | QD |
| Department | Byers Eye Institute at Stanford University |
| City | Palo Alto |
| Country | United States |
2
| Last name | Wang |
| Initials of first name(s) | R |
| Department | Texas Retina Associates |
| City | Dallas |
| Country | United States |
3
| Last name | Dacey |
| Initials of first name(s) | M |
| Department | Rocky Mountain Uveitis |
| City | Denver |
| Country | United States |
4
| Last name | Thomas |
| Initials of first name(s) | A |
| Department | Tennessee Retina |
| City | Nashville |
| Country | United States |
5
| Last name | Grewal |
| Initials of first name(s) | D |
| Department | Duke Eye Center, Duke University |
| City | Durham |
| Country | United States |
6
| Last name | Mudd, Jr. |
| Initials of first name(s) | P |
| Department | Priovant Therapeutics Inc. |
| City | Durham |
| Country | United States |
