Aevi Genomic Medicine’s genetic approach to treating ADHD was expected to ascend its lead drug to great heights in the commonly diagnosed neurobehavioral disorder, but this saga has only culminated in another trial failure, forcing the resource-poor company to “reduce the scope” of its operations to preserve capital.
Shares of the Philadelphia-based company $GNMX were obliterated Wednesday afternoon, nosediving nearly 65% after the bell, wading deeper into penny stock territory.
The SAGA trial reported in March 2017 cast doubt on the viability of the drug, AEVI-001, when it failed to hit statistical significance against a placebo on an ADHD rating scale in adolescent patients with a mutation that disrupts the mGluR (metabotropic glutamate receptor) network — an abundant family of receptors in the brain. However, an analysis of the mid-stage data in the study that enrolled 101 patients suggested the drug did have a pronounced impact on the symptoms of inattention. Encouraged, Aevi elected to continue evaluating the non-stimulant molecule.
According to the company, about 1 in 5 ADHD patients carries such a mutation. The American Psychiatric Association estimates around 5% of American children have ADHD, but with increasing awareness, the CDC pegs that number was closer to 9.4% in 2016.
Aevi took AEVI-001 into another mid-stage placebo-controlled study, dubbed ASCEND, in two types of patients aged 6 to 17. In one arm of the trial, 69 patients with gene mutations associated with glutamatergic signaling were enrolled, while for another arm of the trial, 109 patients with no such mutations were enrolled. Data showed the drug did not induce a statistically significant improvement in ADHD symptoms in either category of patient, but the company has not thrown in the towel on the program just yet.
“Aevi Genomic Medicine remains committed to the development of AEVI-001 as a potential treatment for a sub-population of…ADHD patients with genetic mutations that disrupt the mGluR network,” the company reiterated in a statement on Wednesday.
However, the drug developer is strapped for cash. It anticipates reducing the scope of its operations to preserve net working capital, which was approximately $5 million (unaudited) as of December 31, 2018, it said.
The company also has AEVI-004, a co-crystal version of AEVI-001, which is also called fasoracetam. AEVI-004 is designed to improve the stability of the original AEVI-001 molecule, while maintaining similar solubility. Meanwhile, it is also developing two additional molecules: AEVI-002 for severe pediatric-onset Crohn’s disease and AEVI-005 for an undisclosed autoimmune orphan disease, both partnered with Kyowa Hakko Kirin.
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