Plans to initiate two efficacy studies investigating
Continued strong recruitment for the pivotal Essential3 trials in Essential Tremor (ET), with over 50,000 pre-screening forms and over 7,000 referrals received to date that meet pre-qualifying eligibility criteria; topline results expected in the second half of 2024
Randomization in
Completed two underwritten public offerings in 2024; Cash and investments of
“In the first quarter we made significant progress on all fronts. We strengthened our financial resources and reported positive photo paroxysmal response (PPR) results in
Recent Highlights and Anticipated Milestones:
Cerebrum™ Small Molecule Platform
- Ulixacaltamide for ET: Essential3, the Phase 3 program for ulixacaltamide, continues to progress through patient qualification, enrollment and trial execution, with topline results expected in the second half of 2024 to support a planned New Drug Application (NDA) submission in 2025.
- Essential3 is comprised of two simultaneous Phase 3 studies including a 12-week, parallel design study and a 12-week randomized withdrawal study for stable responders.
- Since beginning recruitment in
November 2023 , there have been over 50,000 pre-screening forms and over 7,000 referrals received to date meeting pre-qualifying eligibility criteria. Randomization pace is now dictated by appropriate patient selection and pre-randomization stability and severity criteria. - At the
American Academy of Neurology 2024 Annual Meeting, Praxis shared additional posters on the Essential3 study design [link] and the benefits of ulixacaltamide over propranolol as analyzed from the Essential1 study [link].
PRAX -628 for Focal Onset Seizures: Praxis reported positive results, with 100% of patients responding in a Phase 2a proof of concept study evaluatingPRAX -628 in epilepsy patients with PPR and confirmed plans to initiate two efficacy studies in focal onset seizures.- The PPR study had two cohorts, where all patients responded.
- In the 15 mg cohort (n=5), 80% of patients achieved a complete response and 20% achieved a partial response.
- In the 45 mg cohort (n=3), 100% of patients achieved a complete response.
- Safety was consistent with the prior dose escalation study and pharmacokinetic analysis confirmed therapeutic exposures.
- Praxis plans to initiate two efficacy studies in focal onset seizures.
- The first study is expected to begin in the second half of 2024, with topline results expected in 2025.
- The second study is expected to initiate in the first half of 2025, with topline results expected in the first half of 2026.
- The PPR study had two cohorts, where all patients responded.
PRAX -562 for SCN2A and SCN8A DEEs: Praxis expects topline results from thePRAX -562 Phase 2 EMBOLD study for the treatment of pediatric patients with DEEs in the third quarter of 2024.- The EMBOLD study is a randomized, double-blind, placebo-controlled Phase 2 clinical study to evaluate the safety, tolerability, efficacy (motor seizure frequency) and pharmacokinetics of
PRAX -562 in pediatric patients aged 2 to 18 years with DEEs, followed by an open-label extension.
- The EMBOLD study is a randomized, double-blind, placebo-controlled Phase 2 clinical study to evaluate the safety, tolerability, efficacy (motor seizure frequency) and pharmacokinetics of
Solidus™ Antisense Oligonucleotide (ASO) Platform
- Elsunersen (
PRAX -222) for SCN2A Gain-of-Function (GoF)DEEs : Praxis is completing multiple global regulatory interactions in the first half of 2024 in anticipation of starting the pivotal phase of the program later in 2024.
Corporate Update:
- In
January 2024 , Praxis completed an underwritten public offering with net proceeds of approximately$161.6 million . - In
January 2024 , Praxis announced a licensing partnership with Tenacia Biotechnology to develop and commercialize ulixacaltamide for the treatment of ET inGreater China , including mainlandChina ,Hong Kong ,Macau andTaiwan , with total potential consideration of over$275 million . - In
April 2024 , Praxis completed an additional underwritten public offering with net proceeds of approximately$215.8 million . - As of
April 30, 2024 , Praxis had cash, cash equivalents, and marketable securities of$451.2 million .
First Quarter 2024 Financial Results:
As of
Research and development expenses were
General and administrative expenses were
Praxis reported a net loss of
As of
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 late-stage development for the treatment of essential tremor, www.praxisessentialtremor.com.
About
About Elsunersen
Elsunersen is an ASO designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause of early onset SCN2A-DEE to treat seizures and other symptoms in patients with GoF SCN2A mutations. In vitro studies of elsunersen have demonstrated reduction in both SCN2A gene expression and protein levels. In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and increased survival in SCN2A mouse models, with potential to be the first disease-modifying treatment for SCN2A-DEE. Elsunersen has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPD) from the FDA, and ODD and PRIME designations from the
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, the development of our product candidates, the anticipated timing of regulatory submissions and interactions and our projected cash runway, 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; 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
CONDENSED CONSOLIDATED BALANCE SHEETS (Amounts in thousands) (Unaudited) | |||||||
Assets | |||||||
Cash and cash equivalents | $ | 151,980 | $ | 81,300 | |||
Marketable securities | 91,309 | — | |||||
Prepaid expenses and other current assets | 4,388 | 3,580 | |||||
Property and equipment, net | 476 | 588 | |||||
Operating lease right-of-use assets | 1,840 | 2,064 | |||||
Other non-current assets | 416 | 416 | |||||
Total assets | $ | 250,409 | $ | 87,948 | |||
Liabilities and stockholders’ equity | |||||||
Accounts payable | $ | 9,404 | $ | 5,815 | |||
Accrued expenses | 6,976 | 7,416 | |||||
Operating lease liabilities | 2,224 | 2,495 | |||||
Deferred revenue | 2,122 | 2,553 | |||||
Common stock | 13 | 13 | |||||
Additional paid-in capital | 923,141 | 723,577 | |||||
Accumulated other comprehensive loss | 3 | — | |||||
Accumulated deficit | (693,474 | ) | (653,921 | ) | |||
Total liabilities and stockholders' equity | $ | 250,409 | $ | 87,948 |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands, except share and per share amounts) (Unaudited) | |||||||
Three Months Ended | |||||||
2024 | 2023 | ||||||
Collaboration revenue | $ | 431 | $ | 683 | |||
Operating expenses: | |||||||
Research and development | 26,984 | 25,504 | |||||
General and administrative | 15,333 | 13,270 | |||||
Total operating expenses | 42,317 | 38,774 | |||||
Loss from operations | (41,886 | ) | (38,091 | ) | |||
Other income: | |||||||
Other income, net | 2,333 | 636 | |||||
Total other income | 2,333 | 636 | |||||
Net loss | $ | (39,553 | ) | $ | (37,455 | ) | |
Net loss per share attributable to common stockholders, basic and diluted | $ | (2.84 | ) | $ | (10.58 | ) | |
Weighted average common shares outstanding, basic and diluted | 13,904,374 | 3,540,185 |
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