AEON Biopharma, Inc. announced the topline results from its Phase 2 clinical trial of ABP-450 (prabotulinumtoxinA) for the preventive treatment of episodic migraine. The Phase 2 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of ABP-450 for the prevention of episodic migraine in adults who suffer from fewer than 15 headache days per month and between 6 to 14 migraine days per month. The study enrolled a total of 305 patients across approximately 60 sites in the United States, Canada and Australia.

Patients had at least a one-year history of episodic migraine (with or without aura) according to the ICHD-3 (2018) definition and diagnostic criteria. Patients were randomized approximately evenly across a low dose group receiving 150 units (150U) of ABP-450, a high dose group receiving 195 units (195U) of ABP-450, and a placebo group. All patients received two treatment cycles 12 weeks apart utilizing the Company?s novel treatment paradigm involving fewer injections than the current botulinum toxin treatment option for chronic migraine.

The primary endpoints for the clinical study were the change in mean monthly migraine days, or MMD, from the four-week baseline period to weeks 21 to 24 of the treatment period and the incidence of treatment emergent adverse events (TEAEs), in each case as compared to placebo. The key secondary and exploratory endpoints included the percentage of patients achieving a reduction from baseline (the responder rate) of at least 50% in MMD and 75% in MMD during the weeks 21 to 24 of the treatment period and improvements on certain patient and rating scales. On the primary endpoint, the topline data showed ABP-450 had a treatment effect with mean reductions in MMD of 4.8 days at the 150U dose (n = 99) and 5.0 days at the 195U dose (n = 108), compared to only 4.2 days in placebo (n = 98) at weeks 21-24, but did not meet statistical significance.

On the key secondary endpoint of MMD responder rates, ABP-450 did show statistical significance and clinically meaningful separation from placebo. 69% of patients at the 195U dose achieved a 50% or greater reduction in MMD, which was statistically significant to placebo at 52% (p=0.0132). 37% of patients receiving the 195U dose and 34% of patients receiving the 150U dose achieved 75% or greater reduction in MMD, which were both statistically significant to placebo at 23% (p=0.0245 and p=0.0439, respectively).

On the exploratory endpoint of Patient Global Impression of Severity (PGI-S), the results showed statistically significant improvements of -0.9 at the 150U dose and -1.0 at the 195U dose, compared to placebo at -0.6 (p=0.0436 and p=0.0028, respectively). ABP-450 demonstrated a favorable safety profile for patients with episodic migraine. The Company believes the totality of the data provides evidence of a dose response favoring the higher 195U dose and lends support to its decision to progress ABP-450 into Phase 3. The Company plans to request an end-of-phase 2 meeting with the U.S. Food and Drug Administration (FDA) to discuss the protocol and study design for Phase 3, and the meeting is expected to take place in the first half of 2024.

Migraine is a complex neurological disease characterized by recurrent episodes of headaches that affects approximately 40 million people in the United States and approximately a billion people worldwide, making migraine the third most prevalent illness in the world. Patients that live with migraine experience symptoms that include recurring throbbing headache pain, nausea, vomiting, and sensitivity to light, sound, touch and smell. Migraine can be categorized as episodic migraine or chronic migraine.

The Company projects that approximately 9.4 million Americans live with episodic migraine, which is characterized by fewer than 15 headache days per month and between 6 to 14 migraine days per month, but each individual attack can be just as debilitating.