AEON Biopharma, Inc. announced plans to advance a single pivotal clinical development study in cervical dystonia ("CD") for its lead candidate, ABP-450 (prabotulinumtoxinA) injection, utilizing the 351(k) regulatory pathway for biosimilars with the market leader, BOTOX®?(onabotulinumtoxin A), as the reference product. In its briefing package to the FDA, AEON has provided extensive data from analytical, pharmacological, and animal studies that would contribute to a Biologics License Application ("BLA") filing. A successful Phase 3 comparative study in CD would potentially provide the necessary clinical data to support a determination that ABP-450 is highly similar to the reference product for up to all eight currently approved, in addition to future, therapeutic indications.

ABP-450 shares the exact same 900kDa molecular weight as BOTOX. Under the 351(k) biosimilar regulatory pathway, the Company plans rely upon extensive analytical and preclinical testing, much of which has already been completed. Following the FDA meeting scheduled to take place in the third quarter of 2024, the Company expects to move ahead with a planned Phase 3 CD study in approximately 400 patients to evaluate a direct comparison of ABP-450 to BOTOX.

The purpose of the study is to demonstrate non-inferiority to BOTOX and would serve as the basis for a BLA filing. AEON announced the data from its Phase 2 clinical trial of ABP-450 for the treatment of CD in September 2022, and also presented the results at the International Parkinson and Movement Disorders Society Congress®? (IP-MDS) in August 2023.

The Phase 2 trial (ClinicalTrials.gov Identifier: NCT04849988) was a randomized, double-blind, placebo-controlled study that analyzed a total of 57 patients across a total of 20 study sites in the United States. Due to the nature of the disease, dosing was tailored to the individual patient by the investigator based on the severity of the patient's head and neck position, localization of pain, muscle hypertrophy, patient response, and adverse event history. The results from the study demonstrated that the two lower doses of ABP-450 (150 units and 250 units) led to statistically significant improvements in Toronto Western Spasmodic Torticollis rating scale (TWSTRS) total score from baseline to Week 4, including 14.01 points for 150 units (p=0.007) and 11.28 points for 250 units (p=0.0406).

All doses of ABP-450 in the study demonstrated sustained benefits, with the median duration of effect across all dosing arms of at least 20 weeks, patients' last visit.