Developing new drugs that are not yet approved for patient use and taking them through clinical studies is a lengthy process. The clinical testing of experimental drugs is done in different phases, each successive phase involving more patients. The phases are Phase 1 for safety, Phase 2 to assess efficacy and Phase 3 are to assess definitive efficacy and safety compared with standard of care. Once the clinical trials are complete, the results are presented to the regulatory authority, which in the US is the Food and Drug Administration (FDA). If approved by the FDA, the drug can be made commercially available.
The Phase 1/2a Cethrin trial for acute spinal cord injury was designed to test safety, and it was an open-label trial, meaning both patients and physicians knew who received the therapy. The Phase 1/2a trial was conducted by clinical investigators with many years of neurosurgery training and experience. The next phase will be a Phase 2B trial, designed as a placebo-controlled, double-blind trial to test efficacy. That means that neither patient nor physician will know who receives the therapy, but safety is ensured by an independent review board. Results from the Phase 2b trial will only be known once the trial is completed and unmasked.
Placebo-controlled trials are essential to drug approval because disorders of the central nervous system often show substantial “placebo effects” . To ensure drug efficacy, generally, two pivotal trials are needed for regulatory approval. Cethrin has been granted Fast Track status by the FDA, allowing for accelerated review in the U.S. In addition, the drug’s Orphan Drug status and the significant need for effective therapies for SCI positions Cethrin to be approved with a successful pivotal study.
BioAxone is preparing for the Phase 2b Cethrin trial while seeking a funding partner to support the studies.
BA-1049 is in preclinical development with a lead indication in glaucoma. BA-1049 is formulated as eye drops, given once daily.