PRAX
Praxis Precision Medicines Provides Vormatrigine Program Update
Article Summary
HOLD- Praxis Precision Medicines Inc. (NASDAQ: PRAX) announced results from the Phase 2/3 POWER1 study evaluating vormatrigine in patients with focal onset seizures did not meet its primary success measure, the 50% response rate, while seizure reduction during the second half of the study on higher dose (30 mg) was more pronounced.
- Praxis Precision Medicines is pausing enrollment in the POWER2 study to reassess the program and determine potential modifications, with the higher dose arm generally well-tolerated with adverse event-related discontinuations less than 10% and approximately 90% of patients transitioning to and remaining in the open label extension study.
- The next-generation, functionally selective small molecule targeting the hyperexcitable state of sodium-channels in the brain is currently being developed as a once daily oral treatment for adult focal onset seizure and generalized epilepsy, with preclinical data demonstrating it is differentiated from standard care with potential to be best-in-class for focal epilepsy.
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Growth Stock Scoring Breakdown
| METRIC | VALUE | WEIGHT | ANALYSIS |
|---|---|---|---|
| Sales Growth TTM ? | -100.0% | 25% | 0.0 ptsDeclining (-100.0%) - NOT a growth stock |
| EPS Growth Next 5Y ? | 0.0% | 25% | 10.0 ptsVery Weak (0.0%) - Minimal expectations |
| Target Price Upside ? | 94.6% | 20% | 100.0 ptsMassive Upside (94.6%) - Target: $652.18 vs Current: $335.16 |
| Gross Margin % ? | — | 15% | 40.0 ptsNo Gross Margin Data |
| Drawdown from 52-Wk High ? | -8.6% | 15% | 20.0 ptsBarely a Dip (-8.6%) - Near recent highs |
Disclaimer: This rating is for informational purposes only and is not financial advice.
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About PRAX
- Praxis Precision Medicines, Inc., a clinical-stage biopharmaceutical company, engages in the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance in the United States.
- The company's platforms include Cerebrum, a small molecule platform for CNS small molecule therapies to develop orally available precision therapies; and Solidus to discover and develop antisense oligonucleotide.
- It also develops ulixacaltamide, a small molecule inhibitor of T-type calcium channels that in NDA for the treatment of essential tremor; relutrigine, a small molecule, which is in NDA for the treatment of SCN2A- and SCN8A-developmental and epileptic encephalopathies (DEE), as well as in Phase 3 trial for broad DEEs; vormatrigine, a small molecule targeting the hyperexcitable state of sodium-channels in the brain that is in Phase 3 trial for the treatment of adjunctive focal epilepsy, as well as in Phase 2 trial for monotherapy focal epilepsy; and Elsunersen, a clinical-stage ASO designed to selectively decrease SCN2A gene expression, which is in Phase 3 trial for the treatment of SCN2A-DEE.
- In addition, the company is developing product candidates in pre-clinical trial comprising PRAX-020 to treat KCNT1; PRAX-080 that targets PCDH19 mosaic expression disorders; PRAX-090 for targeting SYNGAP1 loss-of-function; and PRAX-100 that targets SCN2A LoF mutations.
- It has a cooperation and license agreement with RogCon Inc.; and a research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.
- The company was formerly known as EpiPM Therapeutics, Inc. and changed its name to Praxis Precision Medicines, Inc. in October 2016.
- Praxis Precision Medicines, Inc. was incorporated in 2015 and is headquartered in Boston, Massachusetts.