Avenue Therapeutics, Inc. (Nasdaq: ATXI) has announced its financial results for the third quarter of 2024, alongside updates on its clinical pipeline. The company anticipates topline data from its Phase 1b/2a trial of AJ201 for spinal and bulbar muscular atrophy (SBMA) by the end of 2024. The trial, which enrolled 25 patients, is designed to assess the safety and tolerability of AJ201. Avenue also plans to advance BAER-101 into a Phase 2a trial for epilepsy and initiate a Phase 3 safety study for IV tramadol, both contingent upon securing additional funding.
AJ201 for Spinal and Bulbar Muscular Atrophy
The Phase 1b/2a clinical trial of AJ201 is a 12-week, multicenter, randomized, double-blind study. It enrolled 25 patients with clinically and genetically defined SBMA, who were randomly assigned to receive either AJ201 (600 mg/day) or a placebo. The primary endpoint focuses on evaluating the safety and tolerability of AJ201. Secondary endpoints include pharmacokinetic and pharmacodynamic assessments, specifically measuring changes from baseline in mutant androgen receptor (AR) protein levels in skeletal muscle and changes in the expression of Nrf2-activated genes in skeletal muscle. Exploratory objectives involve monitoring changes in fat and muscle composition via MRI scans, which are considered potential biomarkers for longer-term clinical improvement. Details of the study are available on ClinicalTrials.gov (Identifier: NCT05517603).
BAER-101 for Epilepsy
Avenue Therapeutics is planning a Phase 2a clinical trial of BAER-101 in patients with focal epilepsy and other seizure disorders, pending additional financing. Preclinical mouse models have shown that BAER-101 can fully suppress seizure activity, with a fast onset and stable effect throughout testing.
IV Tramadol Phase 3 Safety Study
Avenue has reached an agreement with the FDA on the safety study protocol and statistical analysis approach for its Phase 3 study of IV tramadol. The study will randomize approximately 300 post-bunionectomy patients to receive either IV tramadol or IV morphine for pain relief over a 48-hour post-operative period. Patients will have access to IV hydromorphone for breakthrough pain. The company aims to initiate this Phase 3 safety study pending additional financing or a partnership, with the goal of completing the study and submitting it to the FDA within 12 months of initiation.
Financial Overview
As of September 30, 2024, Avenue Therapeutics reported cash and cash equivalents totaling $2.6 million, compared to $4.9 million at June 30, 2024, and $1.8 million at December 31, 2023. Research and development expenses for Q3 2024 were $2.3 million, up from $0.9 million in Q3 2023. General and administrative expenses decreased to $0.8 million from $1.2 million in the same period. The net loss attributable to common stockholders for Q3 2024 was $(3.1) million, or $(1.92) per share, compared to a net income of $0.5 million, or $4.86 per share, for Q3 2023.
