AEON Biopharma Completes Enrollment in Phase 2 Clinical Study

AEON Biopharma, Inc. recently announced the completion of patient enrollment in the Phase 2 clinical study of ABP-450 (prabotulinumtoxinA) for the treatment of cervical dystonia. The company expects to announce topline data from the study in the second half of 2022.

“We are excited to report that our Phase 2 clinical study of ABP-450 for the treatment of cervical dystonia continues to advance, and we look forward to announcing topline data in the second half of 2022,” said Marc Forth, Chief Executive Officer of AEON. “If approved, ABP-450 will be the only therapeutic botulinum toxin physiochemically similar to Botox. Paired with our anticipated reimbursement benefits, this would be a significant opportunity, as we estimate the addressable market for treating the cervical dystonia patients in the US alone to be approximately $360 million.”

The Phase 2 randomized, double-blind, placebo-controlled study enrolled a total of 59 patients across a total of 25 study sites in the United States. Patients enrolled into the clinical study are divided evenly across four cohorts, including a low dose (150 units), mid-dose (250 units), high dose (350 units), and placebo. Each patient receives a single treatment cycle of their designated dose of ABP-450 or placebo. Due to the nature of the disease, dosing in initial sessions is tailored to the individual patient based on the patient’s head and neck position, localization of pain, muscle hypertrophy, patient response, and adverse event history. The primary endpoint of the clinical study is to evaluate the safety of a single treatment of ABP-450 over a maximum of 20 weeks. The secondary endpoint is the mean difference of change from baseline to week four of each dosing cohort, as measured on the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS), the standard scale for measuring the severity of cervical dystonia. At the completion of the Phase 2 clinical study, all patients, irrespective of treatment group, will have the option to receive treatment with ABP-450 by rolling over into a 52-week open-label extension study.

Cervical dystonia, also known as spasmodic torticollis, is a neurological condition characterized by involuntary muscle contractions of the neck, which may present as spasms, contractions, or abnormal posture. It is a chronic condition with no cure, causing significant pain and challenges to mobility due to abnormal postures, affecting quality of life and daily activities. Botulinum toxin is the standard of care for the treatment of cervical dystonia, helping to improve pain, posture, and disability. Based on US census data and published clinical studies, the company estimates there are approximately 50,000 addressable cervical dystonia patients in the US, of which 30,000 are currently treated.

ABP-450 contains a 900 kDa botulinum toxin type-A complex produced by the bacterium Clostridium botulinum. The active part of the botulinum toxin is the 150 kDa component, and the remaining 750 kDa of the complex is made up of accessory proteins that the company believes help with the function of the active portion of the botulinum toxin. When injected at therapeutic levels, ABP-450 blocks peripheral acetylcholine release at presynaptic cholinergic nerve terminals by cleaving SNAP-25, a protein integral to the successful docking and release of acetylcholine from vesicles situated within the nerve endings leading to denervation and relaxation of the muscle.

AEON licenses ABP-450 from Daewoong and possesses exclusive development and distribution rights for its therapeutic indications in the United States, Canada, the European Union, and certain other international territories. To produce ABP-450, Daewoong constructed a facility in South Korea, purpose-built to comply with FDA and EMA regulations, and continues to expand its production capacity. ABP-450 is the same botulinum toxin complex that has been approved by regulatory authorities in the United States, the European Union and Canada for an aesthetic indication and has consistently shown to be non-inferior to Botox at doses ranging from 20 units to 360 units.

AEON is a clinical-stage biopharmaceutical company focused on developing ABP-450 (prabotulinumtoxinA) injection for the treatment of debilitating medical conditions with an initial focus on the neurology and gastroenterology markets. The company is dedicated to innovation in the rapidly expanding therapeutic botulinum toxin market and believes its therapeutic-only focus will allow AEON to advance safe and effective treatment options to patients, while delivering differentiated economics to payors and physicians. The company continues to evaluate additional therapeutic indications for development based on a comprehensive product assessment process designed to identify those indications where it believes ABP-450 can attain clinical, regulatory, and commercial success. For more information, visit www.aeonbiopharma.com.