Positive preliminary analysis of 15 mg cohort in the
Enrollment for both ulixacaltamide Phase 3 Studies in the Essential3 program on track to be completed in 1H 2024 with topline results in 2H 2024
Recently announced licensing partnership with Tenacia Biotechnology to develop and commercialize ulixacaltamide in
- Topline results of
PRAX -628 in Phase 2a PPR study in the first quarter of 2024, followed by the expected initiation of the Phase 2b study in focal epilepsy in 2024 - Topline results for both Phase 3 studies evaluating ulixacaltamide in essential tremor in the second half of 2024
- Complete regulatory interactions to advance elsunersen (
PRAX -222) towards a pivotal study for the treatment of SCN2A gain-of-function (GOF) developmental epilepsies - Topline results of the Phase 2 EMBOLD study of
PRAX -562 in SCN2A and SCN8A developmental epilepsies in the first half of 2024
“We are thrilled to continue building on the momentum of an exciting 2023 and start the year with four assets in the clinic, including our lead program, ulixacaltamide, and
- Praxis made strong progress in developing
PRAX -628 for focal epilepsy, which affects over 1 million Americans. Initial data fromPRAX -628 show it has a good tolerability profile, a potentially wide therapeutic index and reaching efficacious concentrations within 24 hours. (poster) - Praxis is conducting a Phase 2a PPR study to evaluate the efficacy of
PRAX -628 across two cohorts, dosed at 15 mg and 45 mg. PPR studies measure EEG signatures after intermittent photic stimulation and are widely used as a marker of anti-seizure efficacy and to aid in dose determination. This approach has been validated with several approved and new therapies. - A preliminary analysis of the 15 mg cohort showed that this cohort exceeds the expectations in terms of drug activity. The full data set will be disclosed after completion of the ongoing 45 mg cohort and full analysis of the Study.
- In 2023, Praxis disclosed results from a Phase 1 dose escalation study of
PRAX -628 in healthy volunteers:PRAX -628 was generally well-tolerated at all tested doses. Pharmacokinetic data demonstrated dose-dependent exposure supporting once-daily dosing without titration to achieve potentially therapeutically effective drug concentration levels. (press release)- Further analysis of patients in the Phase 1 study using qEEG data showed a pharmacodynamic effect at all dose levels and was significantly different from placebo. (poster)
Ulixacaltamide for Essential Tremor
Essential tremor affects up to seven million people in
- Enrollment in Essential3 remains on track, with plans to complete in the first half of 2024 and topline results expected in the second half of 2024, supporting a planned New Drug Application (NDA) submission in 2025.
- Key elements of the Essential3 registrational program are:
- Two simultaneous Phase 3 trials, including a 12-week, parallel design study and a 12-week randomized withdrawal study for stable responders
- Using the modified Activities of Daily Living 11 (mADL11) as a primary endpoint
- In the Phase 2 Essential1 study, mADL11 produced a consistent and meaningful response in ulixacaltamide when compared to placebo after 8 weeks of treatment (p=0.042, nominal)
- Using a single 60 mg dose in the Phase 3 trials
- Safety database for a NDA by ICH guidelines requiring 300 patients with six-months of exposure and 100 patients with one-year of exposure
- On
January 5, 2024 , Praxis announced a partnership with Tenacia Biotechnology to develop and commercialize ulixacaltamide inGreater China (press release)
Elsunersen (
SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) is a debilitating and fatal monogenic epilepsy disorder caused by variants in the SCN2A gene. More than 70% of early-onset SCN2A-DEE patients live with uncontrolled seizures, and approximately 75% live with severe intellectual disability with patients rarely surviving beyond their teenage years.
- In
December 2023 at theAmerican Epilepsy Society annual conference, Praxis shared data from Part 1 of the EMBRAVE study, where four patients were dosed once a month for a four-month period (poster)- Patients showed a 43% median reduction in seizures from baseline
- Patients showed a 48% increase in seizure-free days from baseline
- There were no drug-related treatment-emergent adverse events or serious adverse events
- Praxis has an upcoming meeting with the FDA to discuss next steps in developing elsunersen
Praxis continues to progress
- Praxis expects topline results from the EMBOLD study in pediatric patients with SCN2A-DEE and SCN8A-DEE in the first half of 2024
About Ulixacaltamide
Ulixacaltamide is a differentiated and highly selective small molecule inhibitor of T-type calcium channels designed to block abnormal neuronal burst firing in the Cerebello-Thalamo-Cortical (CTC) circuit correlated with tremor activity. Ulixacaltamide, the most advanced program within Praxis’ Cerebrum™ small molecule platform, is currently in development for the treatment of essential tremor. www.praxisessentialtremor.com.
About
About Elsunersen (
Elsunersen (
About SCN2A-DEE
SCN2A developmental and epileptic encephalopathy (SCN2A-DEE) is a debilitating and fatal monogenic epilepsy disorder caused by a variant in the SCN2A gene. The SCN2A gene is critical in the formation of sodium channel proteins in the brain, which control the flow of sodium ions into neurons. This movement of sodium ions is a major component of generating electrical signals called action potentials, the way in which the cells communicate. SCN2A-DEE presents with a wide range of phenotypes. Early-onset SCN2A-DEE presents before three months and can lead to profound impact on patients, including drug-resistant seizures, significant cognitive impairment, movement disorders such as dystonia or ataxia and problems in other body systems such as gastrointestinal or ocular. Currently there are no approved treatments for SCN2A-DEE, and the standard-of-care typically involves a regimen of many concurrent anti-seizure medications as well as medications to manage co-morbidities. Despite these interventions, more than 70% of early-onset SCN2A-DEE patients live with uncontrolled seizures, and approximately 75% live with severe intellectual disability with patients rarely surviving beyond their teenage years.
About
About Praxis
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws, including express or implied statements regarding Praxis’ future expectations, plans and prospects, including, without limitation, statements regarding the anticipated timing of our clinical trials and the development of our product candidates, as well as other statements containing the words “anticipate,” “believe,” “continue,” “could,” “endeavor,” “estimate,” “expect,” “anticipate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” “target,” “will” or “would” and similar expressions that constitute forward-looking statements under the Private Securities Litigation Reform Act of 1995.
The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation: uncertainties inherent in clinical trials; preliminary analyses from ongoing studies differing materially from final data from preclinical studies and completed clinical trials; the expected timing of clinical trials, data readouts and the results thereof, and submissions for regulatory approval or review by governmental authorities; regulatory approvals to conduct trials; and other risks concerning Praxis’ programs and operations as described in its Annual Report on Form 10-K for the year ended
Investor Contact: Praxis Precision Medicines investors@praxismedicines.com 857-702-9452 Media Contact:Dan Ferry Life Science Advisors Daniel@lifesciadvisors.com 617-430-7576
Source:
2024 GlobeNewswire, Inc., source