Over 3,000 referrals received to date meet pre-qualifying eligibility criteria for ulixacaltamide Phase 3 studies in the Essential3 program for essential tremor (ET); enrollment on track to be completed in 1H 2024 with topline results in 2H 2024
Topline results from the
Tolerability and efficacy results from Part 1 of the EMBRAVE study of elsunersen were presented at the
Announced licensing partnership with Tenacia Biotechnology to develop and commercialize ulixacaltamide for ET in
Completed underwritten public offering with over
“2023 was a transformational year for Praxis. Living by our Dare for More™ motto, we made significant advancements across our portfolio of precision therapies for CNS disorders, established strategic collaborations and strengthened our financial position, which we believe will carry us beyond our upcoming milestones in the year ahead,” said
Recent Highlights and Anticipated Milestones:
Cerebrum™ Small Molecule Platform
- Ulixacaltamide for ET: In
November 2023 , Praxis initiated Essential3, the Phase 3 program for ulixacaltamide. Enrollment in Essential3 is expected to complete in the first half of 2024, 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.
- Essential3 incorporates learnings from the Phase 2 Essential1 study including the use of a single 60 mg dose, using a modified Activities of Daily Living scale (mADL11) as the primary endpoint based on feedback from the
U.S. Food and Drug Administration (FDA), and conducting the study in a decentralized manner. In the Phase 2 Essential1 study, mADL11 produced a statistically significant and clinically meaningful response in ulixacaltamide when compared to placebo after 8 weeks of treatment (p=0.042, nominal). - Essential3 has over 3,000 referrals who have met the pre-qualifying eligibility criteria from the ongoing recruitment campaign started in
November 2023 .
PRAX -628 for Focal Epilepsy: The Phase 2a PPR study to evaluate the efficacy and safety ofPRAX -628 across two cohorts, dosed at 15 mg and 45 mg, continues to advance, with plans to report topline results in the first quarter of 2024.- PPR studies measure electroencephalogram (EEG) signatures after intermittent photic stimulation and are widely used as a marker of anti-seizure efficacy and to aid in dose determination.
- Preliminary analysis of the 15 mg cohort exceeded Praxis’ drug activity expectations and confirmed its decision to initiate a Phase 2b study of
PRAX -628 in focal epilepsy in the second half of 2024. - The Phase 2a study builds on positive results from the Phase 1 dose escalation study 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.- 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.
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 mid-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. Up to 20 participants with SCN2A-DEE or SCN8A-DEE are expected to be enrolled.
- 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) Developmental Epilepsies: At theAmerican Epilepsy Society 2023 Annual Meeting, Praxis shared data from Part 1 of the EMBRAVE study, where four patients were dosed once a month for a four-month period. Results showed a 43% median reduction in seizures from baseline, a 48% increase in seizure-free days from baseline and no drug-related treatment-emergent adverse events or serious adverse events.- Elsunersen received PRIME designation from the
European Medical Agency (EMA) for the treatment of SCN2AGoF DEEs . The EMA’s PRIME designation provides enhanced development support for priority medicines that target an unmet need and 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. - Praxis is completing multiple global regulatory interactions in the first quarter of 2024 in anticipation of starting the pivotal phase of the program later this year.
- Elsunersen received PRIME designation from the
Corporate Update:
- In
January 2024 , Praxis completed an underwritten public offering. The net proceeds from the offering were approximately$161.7 million . As ofFebruary 29, 2024 , Praxis had cash and cash equivalents of$247.6 million , which is anticipated to fund operations into 2026. - 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 .
Fourth Quarter and Full Year 2023 Financial Results:
As of
Praxis recognized
Research and development expenses were
General and administrative expenses were
Praxis incurred 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 (
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; 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
CONDENSED CONSOLIDATED BALANCE SHEETS (Amounts in thousands) (Unaudited) | |||||||
Assets | |||||||
Cash and cash equivalents | $ | 81,300 | $ | 61,615 | |||
Marketable securities | — | 38,874 | |||||
Prepaid expenses and other current assets | 3,580 | 10,351 | |||||
Property and equipment, net | 588 | 971 | |||||
Operating lease right-of-use assets | 2,064 | 2,901 | |||||
Other non-current assets | 416 | 416 | |||||
Total assets | $ | 87,948 | $ | 115,128 | |||
Liabilities and stockholders’ equity | |||||||
Accounts payable | $ | 5,815 | $ | 14,672 | |||
Accrued expenses | 7,416 | 15,850 | |||||
Operating lease liabilities | 2,495 | 3,500 | |||||
Deferred revenue | 2,553 | 5,000 | |||||
Common stock | 13 | 5 | |||||
Additional paid-in capital | 723,577 | 606,918 | |||||
Accumulated other comprehensive loss | — | (173 | ) | ||||
Accumulated deficit | (653,921 | ) | (530,644 | ) | |||
Total liabilities and stockholders' equity | $ | 87,948 | $ | 115,128 |
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Amounts in thousands, except share and per share amounts) (Unaudited) | |||||||||||||||
Three Months Ended | Year Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Collaboration revenue | $ | 515 | $ | — | $ | 2,447 | $ | — | |||||||
Operating expenses: | |||||||||||||||
Research and development | 18,388 | 28,329 | 86,766 | 155,040 | |||||||||||
General and administrative | 9,933 | 13,124 | 42,054 | 59,946 | |||||||||||
Total operating expenses | 28,321 | 41,453 | 128,820 | 214,986 | |||||||||||
Loss from operations | (27,806 | ) | (41,453 | ) | (126,373 | ) | (214,986 | ) | |||||||
Other income: | |||||||||||||||
Other income, net | 928 | 280 | 3,096 | 957 | |||||||||||
Total other income | 928 | 280 | 3,096 | 957 | |||||||||||
Net loss | $ | (26,878 | ) | $ | (41,173 | ) | $ | (123,277 | ) | $ | (214,029 | ) | |||
Net loss per share attributable to common stockholders, basic and diluted | $ | (2.97 | ) | $ | (12.98 | ) | $ | (18.69 | ) | $ | (69.65 | ) | |||
Weighted average common shares outstanding, basic and diluted | 9,060,813 | 3,172,981 | 6,594,316 | 3,073,100 |
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