Q32 Bio’s Bempikibart Receives FDA Fast Track Designation for Alopecia Areata

Image Credit: © Nadya Kolobova - stock.adobe.com

Today, Q32 Bio announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for bempikibart (ADX-914), which is used for the treatment of severe alopecia areata.¹

"The Fast Track designation granted by the FDA recognizes the seriousness of AA and the significant current unmet medical need while underscoring bempikibart's potential as a novel, differentiated therapy for patients needing new options," said Jodie Morrison, CEO of Q32 Bio. "This Fast Track designation follows the encouraging clinical activity observed in Part A of our SIGNAL-AA clinical trial and the recent initiation of dosing in the Part A open-label extension and Part B portions of the SIGNAL-AA trial. We look forward to continued collaboration with the FDA as we work to deliver this potentially paradigm-shifting treatment to patients."¹

Bempikibart is a fully human anti-interleukin-7Rα antibody that reregulates adaptive immune function by interfering with signaling mediated by interleukin-7 (IL-7) and thymic stromal lymphopoietin (TSLP). The IL-7 and TSLP receptor pathways have been shown to be drivers in several T cell-mediated pathological processes in many autoimmune diseases.

The phase 1 program demonstrated the pharmacokinetics, pharmacodynamics, and safety of bempikibart in healthy volunteers. ADX-914 is currently being evaluated in phase 2 trials. The double-blind, multi-center, randomized, placebo-controlled, proof of concept SIGNAL-AA study (NCT06018428) dosed its first patient in 2023.² Participants will receive the drug or placebo subcutaneously every 2 weeks for 24 weeks with 12 weeks of follow-up. An open label extension will assess the long-term efficacy, safety, and tolerability of bempikibart in those who achieved hair regrowth.

"Dosing of the first patient in the phase 2 study in alopecia areata, in addition to our ongoing Phase 2 study in atopic dermatitis, demonstrate the breadth of potential applications of bempikibart and our deep commitment to transform the lives of patients with autoimmune and inflammatory diseases," Morrison said.²

The Fast Track designation will fill an unmet medical need and provide a therapy that may be more effective than the limited treatments that are currently available. Q32 Bio is now eligible for more frequent communications with the FDA to discuss development plans, clinical data, and marketing approval. The drug may also be eligible for Accelerated Approval and Priority Review.

"Given the biology implicated in alopecia areata, we believe ADX-914 is a novel approach to addressing the underlying disease process. We believe treatment with ADX-914 could allow for an exceptionally strong and durable clinical response," Jason Campagna, MD, PhD, and chief medical officer of Q32 Bio, told Dermatology Times in a previous interview.

References

1. Q32 Bio Announces FDA Fast Track Designation Granted to Bempikibart (ADX-914) for the Treatment of Alopecia Areata. PR Newswire. April 30, 2025. Accessed April 30, 2025. https://www.prnewswire.com/news-releases/q32-bio-announces-fda-fast-track-designation-granted-to-bempikibart-adx-914-for-the-treatment-of-alopecia-areata-302441850.html

2. Q32 Bio and Horizon Therapeutics plc announce dosing of first patient in phase 2 trial of bempikibart (formerly ADX-914) for severe alopecia areata. News release. September 13, 2023. Accessed April 30, 2025. https://prnmedia.prnewswire.com/news-releases/q32-bio-and-horizon-therapeutics-plc-announce-dosing-of-first-patient-in-phase-2-trial-of-bempikibart-formerly-adx-914-for-severe-alopecia-areata-301925655.html