Praxis Precision Medicines, Inc. is a clinical-stage biopharmaceutical company, which engages in the genetic epilepsies into the development of therapies for central nervous system, or CNS, disorders characterized by neuronal excitation-inhibition imbalance. It focuses on two proprietary platforms: Cerebrum and Solidus. The Cerebrum platform utilizes a deep understanding of neuronal excitability and neuronal networks and applies a series of computational and experimental tools to develop orally available precision therapies. The Solidus platform consists of antisense oligonucleotide, or ASO. It is a targeted precision medicine discovery and development engine anchored on a proprietary, computational methodology. The company was founded by Kiran Reddy, David Goldstein, and Steven Petrou on September 22, 2015 and is headquartered in Boston, MA.
相关临床试验
31
8 进行中
药物批准
29
批准总数
监管机构
1
监管机构数
成立时间
2015
进行中（未招募）
7
22.6%
已完成
10
32.3%
Enrolling By Invitation
1
3.2%
招募中
11
35.5%
终止
1
3.2%
撤回
1
3.2%
- An investigational antisense oligonucleotide (ASO) reduced seizures by 90% in a 17-year-old with SCN2A-related developmental and epileptic encephalopathy, a rare genetic disorder. - Connor Dalby, who had never walked unassisted in his life, began taking independent steps approximately four months after his first intrathecal dose at age 14. - The allele-selective approach, published in *Nature Medicine*, knocks down the mutated SCN2A gene while sparing the healthy copy, a strategy that may generalize to many patients. - Researchers suggest this n-of-1 success demonstrates the potential for precision genetic medicines to address ultra-rare diseases at their molecular root cause.
- The developmental and epileptic encephalopathies (DEE) market reached USD 2.2 billion across the 7MM in 2025, with the United States accounting for approximately 59% of the total market share. - The diagnosed prevalent population of DEE in the 7MM is projected to rise from approximately 288,000 in 2025 to 326,000 by 2036, reflecting improved genetic testing and diagnostic awareness. - A robust pipeline featuring antisense oligonucleotides, gene therapies, and precision medicines—including zorevunersen, bexicaserin, and relutrigine—is expected to reshape the treatment landscape for genetically defined DEE subtypes. - Praxis Precision Medicines' relutrigine received FDA Priority Review acceptance in March 2026 with a PDUFA target action date of September 27, 2026, for SCN2A and SCN8A DEEs.
- Praxis Precision Medicines' experimental epilepsy drug vormatrigine failed to meet its primary endpoint of reducing monthly seizure frequency in the POWER1 Phase 2/3 registrational study. - The company has paused enrollment in the POWER2 study to reassess the vormatrigine program, though the drug showed encouraging signals on higher doses and maintained a solid safety profile. - Despite the setback, vormatrigine met a key secondary endpoint with a 50% responder rate and demonstrated more pronounced seizure reduction during the second half of the study. - Praxis shares dropped 12% in after-hours trading following the announcement, though the company continues advancing its other late-stage candidates relutrigine and ulixacaltamide.
- Vima Therapeutics secured $100 million in funding to develop VIM0423, an oral combination therapy targeting muscarinic cholinergic receptors for movement disorders. - The company plans to initiate mid-stage clinical trials for Parkinson's disease and dystonia, with results expected in 2027. - VIM0423 addresses a significant unmet need, as approximately 160,000 Americans with isolated dystonia currently lack FDA-approved oral treatment options. - The drug is designed to offer improved safety and tolerability compared to existing therapies that target similar receptors.
- Bristol Myers Squibb's Breyanzi received FDA approval for treating relapsed or refractory marginal zone lymphoma, becoming the first cell therapy available for this rare lymphatic tumor. - The approval represents Breyanzi's fifth indication and addresses a significant unmet need in marginal zone lymphoma, which accounts for approximately 7% of all B-cell non-Hodgkin lymphoma cases. - Breyanzi has demonstrated strong commercial performance with $747 million in sales in 2024 and $966 million over the first nine months of 2025.
- Praxis Precision Medicines completed a positive pre-NDA meeting with the FDA for ulixacaltamide, an essential tremor treatment, with plans to file the company's first NDA in early 2026. - The company reported positive Phase 2 results for relutrigine in developmental and epileptic encephalopathies, showing robust improvement in motor seizures with a well-tolerated safety profile. - Both programs represent significant milestones for Praxis's CNS portfolio, targeting neurological disorders characterized by neuronal excitation-inhibition imbalance. - The FDA has confirmed a meeting to review relutrigine data, with NDA filing timeline to be determined following regulatory discussions.
- Praxis Precision Medicines announced positive results from the EMBOLD study of relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies, with the Data Monitoring Committee recommending early study termination for efficacy. - The first-in-class sodium channel modulator represents a potential breakthrough for devastating pediatric conditions that currently have no approved treatment options and carry extremely high mortality rates. - The FDA has confirmed an upcoming meeting to review the data and discuss the New Drug Application pathway, with topline results to be presented at the American Epilepsy Society Annual Meeting on December 6, 2025.
- Praxis Precision Medicines will present comprehensive preclinical and clinical data across its precision epilepsy pipeline at the American Epilepsy Society Annual Meeting from December 5-9, 2025. - The company will unveil full results from the RADIANT study showing vormatrigine rapidly reduces seizures in adults with treatment-resistant epilepsy. - Multiple presentations will highlight sustained seizure reduction data from relutrigine's EMBOLD open-label extension study and preclinical findings demonstrating greater potency than fenfluramine in Dravet syndrome. - The presentations underscore Praxis's commitment to translating genetic insights into transformative therapies for central nervous system disorders characterized by neuronal excitation-inhibition imbalance.
- Praxis Precision Medicines reports successful essential tremor readouts from both studies and plans to file for regulatory approval early this year. - The company is conducting interim analysis for its relutrigine DEE program targeting rare SCN2A and SCN8A epilepsy indications in Q4. - The larger EMERALD study continues recruiting and is expected to read out next year, with potential NDA filing following positive interim results.
- Ulixacaltamide demonstrated a statistically significant 4.3-point improvement in daily living activities at Week 8 compared to placebo (p<0.0001) in the Essential3 Phase 3 program for essential tremor. - The drug met all primary and key secondary endpoints across two pivotal studies, with 55% of patients maintaining response versus 33% on placebo in the withdrawal study. - Praxis has submitted a pre-NDA meeting request to the FDA and plans to file for approval by early 2026, potentially addressing a significant unmet need for seven million Americans with essential tremor.
