elsunersen (PRAX-222) / RogCon, Ionis, Praxis Precision Medicines - LARVOL DELTA

Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy. (PubMed, Nat Med) - "These data provide preliminary insights on the safety and efficacy of elsunersen in a preterm infant. Additional investigation on the benefits of elsunersen in clinical trials is warranted."

Journal • CNS Disorders • Epilepsy

Elsunersen (PRAX-222) for early-seizure-onset SCN2A-DEE (GlobeNewswire) - "EMBRAVE3 will be a global, 24-week, double-blind, sham-procedure controlled study, with approximately 40 early-onset SCN2A-DEE patients, and is anticipated to start enrolling patients by mid-year 2025. The second cohort of the EMBRAVE study evaluating safety and efficacy of elsunersen versus sham procedure continues enrolling patients in Brazil, with topline readout anticipated in the first half of 2026."

New trial • P1/2 data • CNS Disorders • Epilepsy

Clinical Updates from the Elsunersen Emergency Use Program: A Novel ASO for Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy (AES 2024) - "Seizure frequency remains stable with ongoing dosing; maintained after tapering phenytoin at age 14 months, with no worsening of neurodevelopment at age 18 months.In the Australian patient, 2 weeks after the second dose, a 0.5 ml weekly reduction of ethosuximide was achieved, with facial myoclonus subsiding during waking hours. Together with EMBRAVE Part 1 findings, clinical experience in ongoing emergency use programs in the US, Germany, and Australia demonstrate a well-tolerated, consistent and meaningful therapeutic response to elsunersen. Ongoing follow up will determine long-term effect on seizures and associated comorbidities."

Clinical • CNS Disorders • Developmental Disorders • Dystonia • Epilepsy • Movement Disorders • NAV1

A Clinical Trial of PRAX-222 in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy (clinicaltrials.gov) - P1/2 | N=60 | Recruiting | Sponsor: Praxis Precision Medicines | Active, not recruiting ➔ Recruiting | Trial completion date: Feb 2026 ➔ Sep 2027

Enrollment open • Trial completion date • CNS Disorders • Epilepsy • Pediatrics

A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy (clinicaltrials.gov) - P1/2 | N=60 | Active, not recruiting | Sponsor: Praxis Precision Medicines | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Epilepsy • Pediatrics

EMBRAVE: A Clinical Trial of PRAX-222, a Novel Antisense Oligonucleotide, in Pediatric Participants with Early Onset SCN2A Developmental and Epileptic Encephalopathy (AES 2023) - P1/2 | "PRAX-222 has potential to be the first disease-modifying treatment for early onset SCN2A GoF DEE. The EMBRAVE trial is intended to identify and confirm a safe and efficacious PRAX-222 dose for seizure control, with Part 1 designed to assess preliminary safety and inform subsequent dose escalation and trial continuation."

Clinical • CNS Disorders • Epilepsy • Pediatrics

A Novel Antisense Oligonucleotide for the Treatment of Early Onset SCN2A Developmental and Epileptic Encephalopathy: A First-in-Patient Report in a Preterm Infant with Refractory Status Epilepticus (AES 2023) - "First-in-patient findings highlight potential for PRAX-222 to be the first disease-modifying treatment for early onset GoF SCN2A-DEE, with early clinical experience in combination with SCBs indicating safety and a temporal association with seizure reduction including cessation of previous SE. Ongoing follow up will determine long-term effects of repeated PRAX-222 administration on seizure frequency and intensity, and associated comorbidities."

Clinical • Prematurity • CNS Disorders • Epilepsy • Gastrointestinal Disorder • Movement Disorders • Sleep Disorder

Praxis Precision Medicines Receives PRIME Designation from the EMA for elsunersen (PRAX-222) for Treatment of SCN2A Gain of Function Developmental Epilepsies (GlobeNewswire) - "Praxis Precision Medicines, Inc...today announced that the European Medical Agency (EMA) has awarded its Priority Medicines (PRIME) designation for elsunersen (PRAX-222) for the treatment of SCN2A Gain of Function (GoF) developmental and epileptic encephalopathy (DEE). The EMA’s PRIME designation...was granted based on the Part 1 data from the EMBRAVE study that showed a reduction in seizures and improvement in seizure free days, as well as preclinical data."

European regulatory • CNS Disorders • Epilepsy

Praxis Precision Medicines to Present at the American Academy of Neurology 2023 Annual Meeting (GlobeNewswire) - "Praxis Precision Medicines, Inc....announced that it will deliver presentations on its clinical stage programs at the upcoming American Academy of Neurology (AAN) 2023 Annual Meeting, held April 22-27, 2023 in Boston, Massachusetts....'Also, we expect to dose the first patients in our PRAX-222 EMBRAVE study and PRAX-562 EMBOLD study within the coming weeks and are excited to share data supporting these programs. Finally, we look forward to sharing new in-vivo results for PRAX-628 that highlight this program’s potential to be a best-in-class treatment for focal epilepsy patients'."

Clinical data • Preclinical • CNS Disorders • Epilepsy

A Clinical Trial of PRAX-222 in Pediatric Participants With Early Onset SCN2A Developmental and Epileptic Encephalopathy (clinicaltrials.gov) - P1/2 | N=60 | Recruiting | Sponsor: Praxis Precision Medicines

New P1/2 trial • CNS Disorders • Epilepsy • Pediatrics

Praxis Precision Medicines Provides Corporate Update and Reports Fourth Quarter and Full Year 2022 Financial Results (GlobeNewswire) - "Topline results expected for each of three clinical-stage epilepsy programs in 2023 – PRAX-222 first-in-patient EMBRAVE Study safety data mid-2023, PRAX-628 first-in-human Phase 1 data mid-2023, PRAX-562 Phase 2 EMBOLD Study results in 2H23."

P1 data • P2 data • CNS Disorders • Epilepsy

Praxis Precision Medicines to Present at the American Epilepsy Society 2022 Annual Meeting (GlobeNewswire) - "Praxis Precision Medicines, Inc...announced that it will deliver presentations on its epilepsy programs at the American Epilepsy Society (AES) 2022 Annual Meeting, held December 2-6, 2022 in Nashville, Tennessee....'It’s an incredibly exciting time for Praxis and our epilepsy portfolio, with first-in-patient studies for PRAX-222 and PRAX-562 and a first-in-human study for PRAX-628 expected to start shortly'....Together with pharmacokinetic findings demonstrating a 13-fold increase in concentrations compared to preclinical maximal electroshock seizure effects, our results are consistent with earlier work suggesting a wide therapeutic window for PRAX-562."

New trial • P1 data • Preclinical • CNS Disorders • Epilepsy

Invitae's Real-World Ciitizen Data Utilized in Praxis Precision Medicines' PRAX-222 IND Filing (PRNewswire) - "Invitae...and Praxis Precision Medicines, Inc...announced that real-world clinical insights from Invitae's Ciitizen platform were utilized as natural history data to support the submission of Praxis' Investigational New Drug (IND) application for PRAX-222 for the treatment of pediatric patients with early-onset SCN2A developmental and epileptic encephalopathy (DEE)....The data, collected on behalf of SCN2A-DEE patients or their parents/guardians, is de-identified and shared with their consent, and represents the richest aggregation of real-world clinical evidence for SCN2A-DEE patients."

Retrospective data • CNS Disorders • Epilepsy

Praxis Precision Medicines Provides Updates on Clinical Stage Pediatric Epilepsy Programs (GlobeNewswire) - "Praxis Precision Medicines, Inc...announced the plan to start the PRAX-222 EMBRAVE clinical study for the treatment of pediatric patients with early-seizure-onset SCN2A developmental and epileptic encephalopathy (DEE), after the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the initial dose cohort. Following collection of the safety and efficacy data from the first cohort of patients in the EMBRAVE study, the data will be evaluated and submitted to the FDA to seek authorization for further dose escalation....Topline results for the PRAX-562 Phase 2 study for the treatment of pediatric patients with SCN2A and SCN8A DEEs are expected in 2023."

New P2 trial • New trial • P2 data • CNS Disorders • Epilepsy

Praxis Precision Medicines Provides Corporate Update and Reports Second Quarter 2022 Financial Results (GlobeNewswire) - "Praxis plans to initiate a PRAX-562 Phase 2, placebo-controlled trial for treatment of developmental epileptic encephalopathies (DEEs) in pediatric patients in the second half of 2022....Topline results are expected in mid-2023....The Company submitted the required data in July 2022 and expects to start its first-in-patient study with PRAX-222 in the second half of 2022."

New trial • P2 data • CNS Disorders • Epilepsy

Human testing for Praxis' lead epilepsy drug held up after FDA clinical hold (FierceBiotech) - "The FDA has slapped a clinical hold on Praxis Precision Medicines’ PRAX-222, a drug aimed at treating early-onset epilepsy and one of the biotech’s three product candidates set to enter clinical-stage development by the end of this year....The FDA hold comes after Praxis filed an investigational new drug application with the agency for the treatment-seeking approval to enter a human study. Though the reasons for the pause haven’t been released yet, Praxis expects to receive an explanation letter from the FDA within 30 days."

FDA event • CNS Disorders • Epilepsy

Praxis Precision Medicines Showcases Largest Targeted Epilepsy Portfolio in Industry at 2022 Epilepsy Day - "Updates from Praxis’ three most advanced epilepsy programs, each of which is expected to be clinical stage by year-end 2022: PRAX-222: Investigational New Drug (IND) submitted to the U.S. Food and Drug Administration (FDA) proposing a seamless study design for SCN2A patients with early-onset seizures; PRAX-562:...A Phase 2 clinical study in cohorts of children with SCN2A epilepsy, SCN8A epilepsy, and Tuberous Sclerosis Complex (TSC) is expected to initiate in 2H2022; PRAX-628: Expected initiation of a Phase 1 study in the fourth quarter of 2022 and a Phase 2 study in focal epilepsy in 2023; A review of the significant progress in Praxis’ early-stage epilepsy pipeline, including plans to declare antisense oligonucleotide (ASO) candidates for PRAX-080 for PCDH19 and PRAX-090 for SYNGAP1 in 2023."

New P1 trial • New P2 trial • CNS Disorders • Epilepsy

Praxis Precision Medicines Provides Corporate Update and Reports Fourth Quarter and Full Year 2021 Financial Results (GlobeNewswire) - "Praxis plans to initiate a PRAX-562 Phase 2, placebo-controlled trial for treatment of developmental epileptic encephalopathies (DEEs) in the second quarter of 2022. Praxis intends to initiate a seamless study of PRAX-222, its lead antisense oligonucleotide (ASO) candidate, for the treatment of SCN2A-DEE in the second quarter of 2022."

New P2 trial • New trial • CNS Disorders • Epilepsy

Praxis Precision Medicines to Present Data from Rare Disease Programs at the American Epilepsy Society 2021 Annual Meeting (GlobeNewswire) - "Praxis Precision Medicines, Inc...announced that data from its rare disease programs will be delivered in three presentations at the American Epilepsy Society 2021 Annual Meeting, held December 3-7, 2021 in Chicago."

Preclinical • CNS Disorders • Epilepsy

Praxis Precision Medicines Provides Corporate Update and Reports Second Quarter 2021 Financial Results (GlobeNewswire) - "Praxis plans to complete the ongoing Investigational New Drug (IND) enabling toxicology study by the end of 2021 for its lead antisense oligonucleotide (ASO) candidate, PRAX-222, and to initiate a Phase 1/2 trial of PRAX-222 for treatment of SCN2A-DEE in the first half of 2022."

New P1/2 trial • Preclinical • CNS Disorders • Epilepsy

Trait-based mapping to identify the genetic factors underlying anaerobic germination of rice: Phenotyping, GXE, and QTL mapping. (PubMed, BMC Genet) - "The study helped us understand the effect of phenotyping method on anaerobic germination, which will lead to better phenotyping for this trait in future studies. The QTLs identified through this study will allow the improvement of breeding lines for the trait through marker-assisted selection or through forward breeding approaches such as genomic selection. The high frequency of the BJ1 allele of these QTLs will enhance the robustness of germination under anaerobic conditions in inbred and hybrid rice varieties."

Journal