AEON Biopharma Reprioritizes Strategy to Pursue ABP-450 Biosimilar Pathway

AEON Biopharma, Inc. has announced plans to advance a pivotal Phase 3 clinical study for its leading candidate, ABP-450 (prabotulinumtoxinA) injection, aimed at treating cervical dystonia (CD). The company intends to utilize the 351(k) regulatory pathway for biosimilars, with BOTOX® serving as the reference product. The decision comes after AEON provided comprehensive data from analytical, pharmacological, and animal studies to the FDA, which will contribute to a future Biologics License Application (BLA).

ABP-450, sharing the same 900kDa molecular weight as BOTOX, will be the focus of a head-to-head comparison with the market leader. AEON believes that a successful Phase 3 comparative study could yield the necessary clinical data to support the determination that ABP-450 is highly similar to BOTOX for all currently approved and future therapeutic uses. AEON's President and CEO, Marc Forth, expressed optimism about the biosimilar strategy, stating that it offers a significant opportunity to bring ABP-450 to the U.S. market under a single approval for all therapeutic indications of BOTOX by conducting just one comparative study.

The company plans to move forward with this Phase 3 study following a Biosimilar Initial Advisory Meeting with the FDA, scheduled for the third quarter of 2024. The study will involve approximately 400 patients and will aim to demonstrate the non-inferiority of ABP-450 to BOTOX, serving as the foundation for the BLA filing.

In September 2022, AEON released data from its Phase 2 clinical trial of ABP-450 for treating CD, with the results also presented at the International Parkinson and Movement Disorders Society Congress in August 2023. The Phase 2 trial was a randomized, double-blind, placebo-controlled study involving 57 patients across 20 study sites in the United States. Patients were divided into four cohorts: a low dose (150 units), mid-dose (250 units), high dose (350 units) of ABP-450, and a placebo group. Each patient received a single treatment cycle and was followed for up to 20 weeks, with the primary efficacy endpoint assessed at four weeks post-dosing.

The study demonstrated that the two lower doses of ABP-450 (150 units and 250 units) led to statistically significant improvements in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score from baseline to Week 4. The 150 units group showed an improvement of 14.01 points (p=0.007), while the 250 units group improved by 11.28 points (p=0.0406). All doses showed sustained benefits, with the median duration of effect across all dosing arms lasting at least 20 weeks.

AEON Biopharma is a clinical-stage biopharmaceutical company focused on developing its proprietary botulinum toxin complex, ABP-450, for various medical conditions, primarily within the neurosciences market. ABP-450 is already approved and marketed for cosmetic indications under the name Jeuveau by Evolus and is manufactured by Daewoong in compliance with current Good Manufacturing Practice standards. The product is approved as a biosimilar in Mexico and India, and AEON holds exclusive development and distribution rights for therapeutic indications in the United States, Canada, the European Union, the United Kingdom, and selected other international territories.

The management team at AEON brings significant experience in biopharmaceutical and botulinum toxin development and commercialization, positioning the company well for future advancements in the field.