Trial did not meet pre-defined stopping criteria at the interim analysis; Marinus has completed RAISE enrollment at approximately 100 patients with topline results expected summer 2024
Future development of IV ganaxolone in refractory status epilepticus to be assessed following review of the final RAISE results
Enrollment in the TrustTSC trial expected to complete mid-May with topline data on track for the first half of Q4 2024
Cost reduction activities to extend cash runway are under review and expected to be implemented Q2 2024
ZTALMY® (ganaxolone) Q1 2024 preliminary net product revenue of between $7.4 and $7.6 million; preliminary unaudited cash, cash equivalents and short-term investments of $113.3 million as of March 31, 2024
RADNOR, PA, USA I April 15, 2024 I Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that an independent Data Monitoring Committee (DMC) has recommended continuing the pivotal Phase 3 RAISE trial evaluating intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE) following an interim analysis.
Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024. Those results will be used to determine whether to continue development of IV ganaxolone. Marinus remains blinded to the RAISE trial data.
“While we are disappointed that RAISE did not meet the early stopping criteria, we will only be able to determine the trial’s outcome once we unblind and analyze the full data set,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “We will also be evaluating potential cost-saving strategies to provide the strongest capital position as we approach enrollment completion in the global Phase 3 TrustTSC trial in tuberous sclerosis complex.”
Ganaxolone development in the RAISE trial is being supported in part by the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50120C00159.
General Business and Financial Update
Marinus expects to complete enrollment in the Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV with approximately 130 patients in mid-May 2024. The Company expects topline results early in the fourth quarter of 2024 and anticipates filing a supplemental New Drug Application to the U.S. Food and Drug Administration in the first half of 2025 with a request for priority review.
Marinus also continues to develop a second-generation ganaxolone formulation intended to provide improved pharmacodynamic and pharmacokinetic profiles that could improve safety, efficacy and tolerability and enable less frequent dosing.
The Company continues the successful U.S. commercial launch of ZTALMY resulting in preliminary unaudited net product revenue of between $7.4 and $7.6 million for the first quarter of 2024. Marinus estimates preliminary unaudited cash, cash equivalents, and short-term investments of $113.3 million as of March 31, 2024. Cost reduction activities to extend the cash runway beyond the fourth quarter of 2024 are under review and are expected to be implemented in the current quarter.
The preliminary first quarter 2024 net product revenue results and cash, cash equivalents, and short-term investments included in this release were calculated prior to the completion of a review by the Company’s independent registered public accounting firm and are therefore subject to adjustment.
About Status Epilepticus
Status epilepticus (SE) is a life-threatening condition resulting from either the failure of the mechanisms responsible for seizure termination or from the initiation of mechanisms which lead to abnormally prolonged seizures.1 SE is the one of the most common neurological emergencies in the U.S., affecting up to 150,000 patients each year, and is associated with substantial morbidity, mortality, and healthcare costs.2,3,4 Patients who do not respond to 1st- and 2nd-line treatments (benzodiazepines and intravenous antiseizure medications) are considered to have refractory SE (RSE).4,5
About Intravenous (IV) Ganaxolone
Ganaxolone is a neuroactive steroid that works by modulating both synaptic and extrasynaptic GABAA receptors via a unique binding site to potentiate two types of inhibitory signaling.6 IV ganaxolone has pharmacokinetic and pharmacodynamic properties well-suited for the treatment of status epilepticus, with rapid and sustained SE cessation observed in pre-clinical and clinical studies.7,8,9 IV ganaxolone has received orphan drug designation from the U.S. Food and Drug Administration for the potential treatment of status epilepticus.
About the RAISE Trial
The RAISE (Randomized Therapy in Status Epilepticus) trial (NCT04391569) is a Phase 3 double-blind, randomized, placebo-controlled clinical trial to evaluate the safety and efficacy of IV ganaxolone in patients with refractory status epilepticus. The RAISE protocol provides for an independent data monitoring committee to conduct an unblinded interim analysis when two-thirds of participants, or approximately 82 patients, have completed the trial.
About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company first introduced FDA-approved prescription medication ZTALMY® (ganaxolone) oral suspension CV in the U.S. in 2022 and continues to invest in the potential of ganaxolone in IV and oral formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings. For more information about Marinus visit www.marinuspharma.com.
1 Trinka E, et al. Epilepsia. 2015;56(10):1515-1523.
2 Betjemann JP, Lowenstein DH. Lancet Neurol. 2015;14(6):615-24.
3 Guterman EL et al. JAMA Neurol. 2021;78(5):588-95.
4 Glauser T et al. Epilepsy Curr. 2016;16:48-61.
5 Brophy GM et al. Neurocrit Care. 2012;17:3-23.
6 Reddy, D. S. Front Cell Neurosci 7, 115 (2013).
7 Zolkowska D et al. Epilepsia. 59(suppl 2):220-227.
8 Gasior M et al. Clin Pharmacol Drug Dev 13: 248-25.
9 Vaitkevicius H et al. Epilepsia 2022; 63(9): 2381-2391.
SOURCE: Marinus Pharmaceuticals
Post Views: 697
Trial did not meet pre-defined stopping criteria at the interim analysis; Marinus has completed RAISE enrollment at approximately 100 patients with topline results expected summer 2024
Future development of IV ganaxolone in refractory status epilepticus to be assessed following review of the final RAISE results
Enrollment in the TrustTSC trial expected to complete mid-May with topline data on track for the first half of Q4 2024
Cost reduction activities to extend cash runway are under review and expected to be implemented Q2 2024
ZTALMY® (ganaxolone) Q1 2024 preliminary net product revenue of between $7.4 and $7.6 million; preliminary unaudited cash, cash equivalents and short-term investments of $113.3 million as of March 31, 2024
RADNOR, PA, USA I April 15, 2024 I Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that an independent Data Monitoring Committee (DMC) has recommended continuing the pivotal Phase 3 RAISE trial evaluating intravenous (IV) ganaxolone for the treatment of refractory status epilepticus (RSE) following an interim analysis.
Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024. Those results will be used to determine whether to continue development of IV ganaxolone. Marinus remains blinded to the RAISE trial data.
“While we are disappointed that RAISE did not meet the early stopping criteria, we will only be able to determine the trial’s outcome once we unblind and analyze the full data set,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus. “We will also be evaluating potential cost-saving strategies to provide the strongest capital position as we approach enrollment completion in the global Phase 3 TrustTSC trial in tuberous sclerosis complex.”
Ganaxolone development in the RAISE trial is being supported in part by the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50120C00159.
General Business and Financial Update
Marinus expects to complete enrollment in the Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV with approximately 130 patients in mid-May 2024. The Company expects topline results early in the fourth quarter of 2024 and anticipates filing a supplemental New Drug Application to the U.S. Food and Drug Administration in the first half of 2025 with a request for priority review.
Marinus also continues to develop a second-generation ganaxolone formulation intended to provide improved pharmacodynamic and pharmacokinetic profiles that could improve safety, efficacy and tolerability and enable less frequent dosing.
The Company continues the successful U.S. commercial launch of ZTALMY resulting in preliminary unaudited net product revenue of between $7.4 and $7.6 million for the first quarter of 2024. Marinus estimates preliminary unaudited cash, cash equivalents, and short-term investments of $113.3 million as of March 31, 2024. Cost reduction activities to extend the cash runway beyond the fourth quarter of 2024 are under review and are expected to be implemented in the current quarter.
The preliminary first quarter 2024 net product revenue results and cash, cash equivalents, and short-term investments included in this release were calculated prior to the completion of a review by the Company’s independent registered public accounting firm and are therefore subject to adjustment.
About Status Epilepticus
Status epilepticus (SE) is a life-threatening condition resulting from either the failure of the mechanisms responsible for seizure termination or from the initiation of mechanisms which lead to abnormally prolonged seizures.1 SE is the one of the most common neurological emergencies in the U.S., affecting up to 150,000 patients each year, and is associated with substantial morbidity, mortality, and healthcare costs.2,3,4 Patients who do not respond to 1st- and 2nd-line treatments (benzodiazepines and intravenous antiseizure medications) are considered to have refractory SE (RSE).4,5
About Intravenous (IV) Ganaxolone
Ganaxolone is a neuroactive steroid that works by modulating both synaptic and extrasynaptic GABAA receptors via a unique binding site to potentiate two types of inhibitory signaling.6 IV ganaxolone has pharmacokinetic and pharmacodynamic properties well-suited for the treatment of status epilepticus, with rapid and sustained SE cessation observed in pre-clinical and clinical studies.7,8,9 IV ganaxolone has received orphan drug designation from the U.S. Food and Drug Administration for the potential treatment of status epilepticus.
About the RAISE Trial
The RAISE (Randomized Therapy in Status Epilepticus) trial (NCT04391569) is a Phase 3 double-blind, randomized, placebo-controlled clinical trial to evaluate the safety and efficacy of IV ganaxolone in patients with refractory status epilepticus. The RAISE protocol provides for an independent data monitoring committee to conduct an unblinded interim analysis when two-thirds of participants, or approximately 82 patients, have completed the trial.
About Marinus Pharmaceuticals
Marinus is a commercial-stage pharmaceutical company dedicated to the development of innovative therapeutics for seizure disorders. The Company first introduced FDA-approved prescription medication ZTALMY® (ganaxolone) oral suspension CV in the U.S. in 2022 and continues to invest in the potential of ganaxolone in IV and oral formulations to maximize therapeutic reach for adult and pediatric patients in acute and chronic care settings. For more information about Marinus visit www.marinuspharma.com.
1 Trinka E, et al. Epilepsia. 2015;56(10):1515-1523.
2 Betjemann JP, Lowenstein DH. Lancet Neurol. 2015;14(6):615-24.
3 Guterman EL et al. JAMA Neurol. 2021;78(5):588-95.
4 Glauser T et al. Epilepsy Curr. 2016;16:48-61.
5 Brophy GM et al. Neurocrit Care. 2012;17:3-23.
6 Reddy, D. S. Front Cell Neurosci 7, 115 (2013).
7 Zolkowska D et al. Epilepsia. 59(suppl 2):220-227.
8 Gasior M et al. Clin Pharmacol Drug Dev 13: 248-25.
9 Vaitkevicius H et al. Epilepsia 2022; 63(9): 2381-2391.
SOURCE: Marinus Pharmaceuticals
Post Views: 697
