Vor Biopharma (VOR) investor relations material

Company evolution and asset overview

• Transitioned from a cell therapy focus to a new entity centered on telitacicept, licensed globally from RemeGen in China.

• Telitacicept is approved in China for multiple autoimmune diseases and is in late-stage development for myasthenia gravis (MG) and Sjögren's disease.

• Global rights and development responsibilities have been assumed, with phase III trials underway in both indications.

Clinical development and trial progress

• Global phase III trial in MG is enrolling well, with top-line data expected in the first half of 2027.

• China contributes 8-10% of patients; new geographies and additional US sites have been added.

• Investigators report strong patient demand and positive tolerability feedback, with extended open-label access provided.

• Sjögren's phase III trial began in March, targeting 250 patients and a two-year enrollment timeline.

Mechanistic and efficacy differentiation

• Dual BAFF/APRIL inhibition targets IgG, IgA, and IgM, offering broader and more durable efficacy than FcRn inhibitors.

• Data from China show robust and sustained improvement in MG, attributed to immune repertoire remodulation.

• Breadth and durability of response are emphasized as key differentiators, with 100% responder rate in MG-ADL in China.