AEON Biopharma Announces Strategic Reprioritization to Pursue a Biosimilar Pathway for ABP-450
AEON Biopharma has announced a strategic shift to pursue a biosimilar pathway for ABP-450, its lead candidate for treating cervical dystonia (CD). The company plans to conduct a Phase 3 study comparing ABP-450 directly with BOTOX® via the FDA's 351(k) regulatory pathway. This move follows promising Phase 2 trial results and aims to secure approval for all of BOTOX’s current and future therapeutic indications. An in-person FDA meeting is scheduled for Q3 2024 to discuss this plan. If successful, the Phase 3 study could pave the way for a Biologics License Application (BLA) filing.
- AEON is advancing a biosimilar pathway for ABP-450, which could streamline approval for multiple therapeutic indications under a single study.
- The company has scheduled an FDA meeting for Q3 2024, providing a clear timeline for regulatory discussions and potential approvals.
- Phase 2 results demonstrated significant improvements in TWSTRS scores for lower doses of ABP-450, indicating strong efficacy.
- The planned Phase 3 study will involve approximately 400 patients, providing robust data for regulatory evaluation.
- The Phase 3 study will require substantial investment and time, with results not expected until after Q3 2024.
AEON Biopharma's strategic reprioritization towards a biosimilar pathway for ABP-450 can have substantial implications for the company’s financial health. The biosimilar market is growing and entering it with ABP-450 as a competitor to BOTOX® could be financially lucrative. The company's announcement of a single pivotal clinical development study in cervical dystonia (CD) indicates a cost-effective approach. By leveraging the 351(k) biosimilar regulatory pathway, AEON aims for a single approval that covers multiple indications, potentially reducing research and development (R&D) expenses significantly compared to developing separate drugs for each indication.
However, investors should note the inherent risks associated with such ventures. The success of the Phase 3 study is critical. Any setbacks could result in delays and additional costs. Furthermore, the competitive landscape for biosimilars, especially against a well-established product like BOTOX, poses significant challenges. Potential investors must weigh these risks against the potential for market share capture upon successful approval.
The utilization of the 351(k) regulatory pathway signifies AEON’s strategic move to tap into the biosimilar market, which requires demonstrating that ABP-450 is 'highly similar' to BOTOX®. This pathway reduces the regulatory burden because it allows the company to rely on extensive analytical and preclinical testing already completed. The planned head-to-head Phase 3 study in cervical dystonia aims to establish non-inferiority to BOTOX, which, if successful, could pave the way for a Biologics License Application (BLA).
From a medical standpoint, the fact that ABP-450 shares the exact same 900kDa molecular weight as BOTOX is promising. However, achieving non-inferiority in clinical efficacy and safety is a high bar. The Phase 2 results show promise, with statistically significant improvements in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) scores, but these need to be replicated in a larger patient cohort. The outcome of the Phase 3 study will be important in determining ABP-450’s future.
– Plan to move ahead with a head-to-head comparison to BOTOX® in a cervical dystonia Phase 3 study via the 351(k) pathway following an in-person FDA meeting scheduled for Q3 2024 –
IRVINE, Calif., July 09, 2024 (GLOBE NEWSWIRE) -- AEON Biopharma, Inc. (“AEON” or the “Company”) (NYSE: AEON), a clinical-stage biopharmaceutical company focused on developing a botulinum toxin complex for the treatment of multiple therapeutic indications, today 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® (onabotulinumtoxinA), 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.
“We believe the biosimilar strategy offers a tremendous potential opportunity for us to bring ABP-450 to the U.S. market under a single approval for all of BOTOX’s currently approved and future therapeutic indications by conducting a single comparative study,” commented Marc Forth, AEON’s President and Chief Executive Officer. “We look forward to discussing our plans with the FDA during a Biosimilar Initial Advisory Meeting currently scheduled for the third quarter.”
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. Patients were divided evenly across four cohorts, including a low dose (150 units), mid-dose (250 units) and high dose (350 units) treatment of ABP-450, and placebo. Each patient received a single treatment cycle of their designated dose of ABP-450 or placebo. Patients were followed for up to a total of 20 weeks, and the primary efficacy endpoint was assessed at four weeks after dosing. 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.
About AEON Biopharma
AEON is a clinical stage biopharmaceutical company focused on developing its proprietary botulinum toxin complex, ABP-450 (prabotulinumtoxinA) injection, or ABP-450, for debilitating medical conditions, with an initial focus on the neurosciences market. ABP-450 is the same botulinum toxin complex that is currently approved and marketed for cosmetic indications by Evolus under the name Jeuveau. ABP-450 is manufactured by Daewoong in compliance with current Good Manufacturing Practice, or cGMP, in a facility that has been approved by the U.S. Food and Drug Administration, Health Canada and European Medicines Agency. The product is approved as a biosimilar in Mexico and India. AEON has exclusive development and distribution rights for therapeutic indications of ABP-450 in the United States, Canada, the European Union, the United Kingdom, and certain other international territories. The Company has built a highly experienced management team with specific experience in biopharmaceutical and botulinum toxin development and commercialization. To learn more about AEON, visit www.aeonbiopharma.com.
Forward-Looking Statements
Certain statements in this press release may be considered forward-looking statements. Forward-looking statements generally relate to future events or AEON’s future financial or operating performance. For example, statements regarding meetings with the FDA, the timing of a potential Phase 3 comparative study in CD, the translation of clinical trial results into support for a BLA filing, and potential determination that ABP-450 is highly similar to the reference product for currently approved and future therapeutic indications are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as "plan", "possible", "forecast", "expect", "intend", "will", "estimate", "anticipate", "believe", "predict", "potential" or "continue", or the negatives of these terms or variations of them or similar terminology. Such forward-looking statements are subject to risks, uncertainties, and other factors which could cause actual results to differ materially from those expressed or implied by such forward-looking statements.
These forward-looking statements are based upon estimates and assumptions that, while considered reasonable by AEON and its management, are inherently uncertain. Factors that may cause actual results to differ materially from current expectations include, but are not limited to: (i) the outcome of any legal proceedings that may be instituted against AEON or others; (ii) AEON’s future capital requirements, including with respect to potential obligations pursuant to the forward purchase agreements; (iii) AEON’s ability to raise financing in the future; (iv) AEON’s ability to continue to meet continued stock exchange listing standards; (v) the possibility that AEON may be adversely affected by other economic, business, regulatory, and/or competitive factors; and (vi) other risks and uncertainties set forth in the section entitled “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” in the Company’s filings with the Securities and Exchange Commission (the "SEC"), which are available on the SEC’s website at www.sec.gov.
Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. You should not place undue reliance on forward-looking statements, which speak only as of the date they are made. AEON does not undertake any duty to update these forward-looking statements.
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