In spite of a setback, Praxis Precision Medicines, a Boston-based biotechnology company, plans to move its most advanced experimental drug into late-stage human testing.
On Friday, Praxis said its drug, named ulixacaltamide, failed to meet the main goal of a mid-stage clinical trial testing it as a treatment for essential tremor — a neurological disorder that causes involuntary shaking in the hands and arms and, sometimes, other parts of the body.
The trial found the drug was no better than a placebo at improving patients’ condition, as measured by a scorecard used to evaluate the severity of their tremors as well as how they perform daily activities like speaking, writing and standing.
However, Praxis said its drug showed small, but statistically significant improvements over placebo on three other scorecards that served as secondary goals of the study. It was also well tolerated, according to Praxis, with the most commonly reported adverse events being dizziness, constipation, headache and fatigue.
Considering those results, the company intends to meet with the Food and Drug Administration and then start a Phase 3 study in the back half of this year.
To Marcio Souza, CEO of Praxis, the results “illustrate the clear potential” of the company’s drug to treat essential tremor. “The study showed meaningful improvement in patients’ daily functioning, while also providing insights to guide the program’s advancement to Phase 3, including a deeper understanding of endpoints and trial design,” he said in a statement.
To that end, Souza said the trial’s outcome has made it clear that ADLs, or Activities of Daily Living scores, should be the main goal for a study supporting FDA approval. The failed Phase 2 study used a “modified” version of an scoring system called TETRAS, which has an active daily living section as well as a performance section. One of the main differences between the categories is the former largely focuses on activities affected by upper limb tremors, whereas the latter assesses head, face, voice and lower limb tremors.
Founded in 2015, Praxis aims to treat diseases of the central nervous system. It officially launched in May 2020, with more than $100 million in funding from investors like Blackstone Life Sciences, Novo Holdings and Vida Ventures. Less than three months later, it had raised another $110 million in a Series C1 financing round led by Eventide Asset Management.
By October of that year, Praxis had gone public, pricing shares at $19 apiece and adding another $190 million to its funding haul. The company’s shares have been declining since early 2021, though, and lost most of their value when an experimental depression drug failed a late-stage trial last June. Praxis’ stock fell another 65%, to $1 per share, after Friday’s announcement.
Praxis sports two technology platforms: one for small molecules, and another for antisense oligonucleotides. Ulixacaltamide is from the small molecule side, and was designed to block a calcium channel tied to the abnormal brain cell firing seen in patients with essential tremor.
In the statement from Praxis, William Ondo, a neurology professor at Houston Methodist Hospital and Weill Cornell Medical College, noted the “significant unmet need for an effective and tolerable precision medicine” for essential tremor.
But, he added, the newly released results “give hope to the essential tremor community that a targeted therapy is within reach, and we eagerly anticipate additional data from the ulixacaltamide program.” Ondo has consulted for several neuroscience drug developers, including Neurocrine Biosciences and Cerevel Therapeutics.