Praxis Precision Medicines Inc. (NASDAQ:PRAX) stock skyrocketed on Thursday.
- PRAX is reaching significant price levels. Check the market position here.
The clinical-stage biopharmaceutical company released topline results for the Phase 3 Essential3 program of ulixacaltamide in essential tremor (ET).
Essential tremor is a neurological disorder that causes involuntary, rhythmic shaking, most commonly in the hands and arms during movement, but it can also affect the head, voice, or legs.
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Study 1: Placebo-controlled Parallel Group Study Topline Efficacy Results
Study 1 showed a statistically significant and clinically meaningful 4.3 point mean improvement in the mADL11 score at Week 8 (p<0.0001).
The effect was sustained from Week 2 throughout the 12-week dosing period. All key secondary endpoints achieved statistical significance.
mADL11 is a modified score that is calculated as the sum of all 11 items and ranges from 0 to 33, where larger values represent increased direct tremor impact on activities of daily living.
Study 2: Randomized Withdrawal Study Topline Efficacy Results
In Study 2, after blinded exposure for 8 weeks with ulixacaltamide, patients meeting the responder criteria (n=80) were randomized to continue receiving ulixacaltamide or switch to placebo for an additional 4 weeks.
55% of patients in the ulixacaltamide arm maintained response vs 33% in the placebo group (p=0.0369).
The first key secondary endpoint – rate of disease improvement – achieved statistical significance, and other secondary endpoints (the Patient Global Impression of change, PGI-C), and Clinical Global Impression of severity, CGI-S) were numerically in favor of ulixacaltamide, but not statistically significant.
Combined Study 1 and Study 2 Hypotheses
Hypothesis 3 and 4 further supported the precision of the effect of ulixacaltamide versus placebo.
- For Hypothesis 3, there was a 4.3 point improvement in mADL11 at Week 8 for the combined Studies 1 and 2 ulixacaltamide groups vs Study 1 placebo (p<0.0001).
- For Hypothesis 4, there was a 4.2 point improvement in mADL11 at Week 8 for the Study 2 ulixacaltamide group vs the Study 1 placebo (p<0.0001), respectively.
Ulixacaltamide was generally well tolerated over 12 weeks of treatment. The most common (≥10% patients) treatment-emergent adverse events (TEAs) were constipation, dizziness, euphoric mood, brain fog, headache, paraesthesia, and insomnia. There were no deaths and no drug-related serious adverse events.
Discontinuations were primarily due to TEAEs, with the most common being dizziness and brain fog.
Praxis has submitted a pre-NDA meeting request to the FDA with plans to submit the NDA by early 2026.
PRAX Price Action: Praxis Precision Medicine shares were up 123.19% at $128 during premarket trading on Thursday. The stock is trading at a new 52-week high, according to Benzinga Pro data.
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