CDKL5 deficiency disorder

Marinus Pharmaceuticals Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, March 5, 2024
Health, FDA, Other Health, General Health, Clinical Trials, Pharmaceutical, Biotechnology, Human services, Therapy, Research, API, Investment, Ganaxolone, Food, CDKL5 deficiency disorder, Database, Epilepsy, TSC, Administration, Lennox–Gastaut syndrome, Patient, Security (finance), Medication, Development, NDA, Pharmaceutical industry

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.
  • For the fiscal year 2024, the Company expects ZTALMY U.S. net product revenues of between $32 and $34 million.
  • At December 31, 2023, the Company had cash, cash equivalents and short-term investments of $150.3 million, compared to cash and cash equivalents of $240.6 million at December 31, 2022.
  • Selected Financial Data (in thousands, except share and per share amounts)

Orphan designation: Fenfluramine hydrochloride Treatment of CDKL5 deficiency disorder, 20/03/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
COMP, IRIS, European Commission, CDKL5, CDKL5 deficiency disorder, Fenfluramine, Committee, EMA, Pharmaceutical industry

EU/3/23/2768 - orphan designation for treatment of CDKL5 deficiency disorder

Key Points: 
  • EU/3/23/2768 - orphan designation for treatment of CDKL5 deficiency disorder
    Fenfluramine hydrochloride
    OrphanHuman
    UCB Pharma
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Marinus Pharmaceuticals Announces Seven Presentations at AES 2023, New Publication in Epilepsia and Results From the Second Generation Formulation MAD Study

Retrieved on: 
Monday, November 20, 2023
Research, Finance, Neurology, FDA, Clinical Trials, Professional Services, Biotechnology, Pharmaceutical, Health, Science, Ganaxolone, American Epilepsy Society, CDKL5 deficiency disorder, Annual general meeting, Lennox–Gastaut syndrome, Efficacy, Treatment, Marinus, IND, Epilepsy, Clinical trial, Serum, Patient, Seizure, Status epilepticus, Therapy, CDD, Abstract, Kinetics, Marigold, Physician, Medication, AES, Safety, Pharmaceutical industry, Dietary supplement

Marinus will also host a scientific exhibit showcasing a series of posters highlighting the potential of ganaxolone in the treatment of refractory seizure disorders.

Key Points: 
  • Marinus will also host a scientific exhibit showcasing a series of posters highlighting the potential of ganaxolone in the treatment of refractory seizure disorders.
  • Also announced today, preliminary data from a Phase 1 MAD study of a second generation ganaxolone formulation demonstrated linear kinetics through a wide dose range that could allow individualization of treatment in patients with refractory epilepsy, a key goal for the second generation formulation.
  • The Company now anticipates initiating a clinical trial in Lennox-Gastaut syndrome with a second generation formulation in 2025.
  • While development of the second generation formulation proceeds, Marinus intends to explore new clinical programs for ZTALMY oral suspension in other refractory epilepsies in the second half of 2024.

Lario Therapeutics receives “Company Making a Difference Award” from CDLK5 Forum, recognising its unique approach to precision medicine for genetic epilepsies

Retrieved on: 
Tuesday, November 7, 2023
Biotechnology, Health, Science, Pharmaceutical, Research, DSM-IV codes, CSO, Seizure, CDO, CDD, CDKL5 deficiency disorder, Entrepreneurship, Medicine, Neurology, Brain, CDKL5, Epilepsy, Intellectual disability, Patient, DEE, Excellence, Pharmaceutical industry, IND

The award is in recognition of its development of a validated, precision medicine approach in genetic epilepsies.

Key Points: 
  • The award is in recognition of its development of a validated, precision medicine approach in genetic epilepsies.
  • Lario Tx’s first-in-class, orally active, CNS-penetrant CaV2.3 ion channel inhibitors hold promise as novel anti-seizure therapeutics for multiple epilepsy subtypes, including CDKL5 Deficiency Disorder.
  • The CDKL5 Forum, established by the Loulou Foundation, is the largest annual conference on CDKL5 Deficiency Disorder (CDD).
  • CaV2.3 is now being investigated as a key target for CDD and CaV2.3 inhibitors hold great promise as a precision medicine approach.

Marinus Pharmaceuticals Initiates Global Access Program for ZTALMY® (ganaxolone) Oral Suspension CV

Retrieved on: 
Tuesday, November 7, 2023
FDA, Health, Clinical Trials, Research, Pharmaceutical, Science, Incidence, Food, Seizure, European Commission, Therapy, CDD, Medication, Hypersalivation, Physician, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Allergen, Somnolence, Epilepsy, CV, CDKL5 deficiency disorder, Marigold, Safety, CDKL5, Patient, Lancet, Pharmaceutical industry, Fever

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced the initiation of the Marinus Access Program, a global managed access program for ZTALMY® (ganaxolone) oral suspension CV.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced the initiation of the Marinus Access Program, a global managed access program for ZTALMY® (ganaxolone) oral suspension CV.
  • The new program enables physicians to request ZTALMY for eligible patients with seizures associated with CDKL5 deficiency disorder (CDD) in geographies where the product is not commercially available and as supported by local regulatory requirements.
  • The Marinus Access Program will be managed by Durbin, a leader in the international distribution of specialized pharmaceuticals.
  • “Consistent with our company mission, we are committing appropriate resources to help facilitate broader access to ZTALMY for patients with critical unmet medical needs,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.

Marinus Pharmaceuticals Highlights Advancing Pipeline and Commercial Strategy at Investor and Analyst Event 

Retrieved on: 
Tuesday, September 19, 2023
Pharmaceutical, Professional Services, Health, Finance, Status epilepticus, Biomedical Advanced Research and Development Authority, Therapy, Office of Inspector General, U.S. Department of Health and Human Services, Hospital, Human services, Ganaxolone, CDKL5 deficiency disorder, Medication, Administration, Development, ATM, RAISE, Patient, Investment, Administration for Strategic Preparedness and Response, Disease, Epilepsy, Washington University School of Medicine, Webcast, Growth, BARDA, Barnes-Jewish Hospital, Pharmaceutical industry, RSE, Neurology

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, is today hosting an Investor and Analyst Event in New York to review the Company’s ongoing clinical-stage programs and commercial planning activities.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, is today hosting an Investor and Analyst Event in New York to review the Company’s ongoing clinical-stage programs and commercial planning activities.
  • The event begins at 9 a.m.
  • “I look forward to showcasing how we are planning to deliver on our commitment to improve the lives of more patients with refractory seizure disorders during today’s Investor and Analyst Event,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
  • Importantly, we have extended our cash runway into the fourth quarter of 2024 through strategic use of our existing ATM facility.

Marinus Pharmaceuticals Provides Business Update and Reports Second Quarter 2023 Financial Results

Retrieved on: 
Thursday, August 10, 2023
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, RSE, Biomedical Advanced Research and Development Authority, Therapy, SG, U.S. Department of Defense Strategy for Operating in Cyberspace, European Commission Investment Plan for Europe, Human services, Office of Inspector General, U.S. Department of Health and Human Services, Lennox–Gastaut syndrome, API, CDKL5 deficiency disorder, Clinical trial, G&A, GAAP, Medication, TSC, Development, Administration, RAISE, European Commission, Patent, Patient, Investment, Administration for Strategic Preparedness and Response, Epilepsy, Ganaxolone, MAD, Growth, Security (finance), R, Research, BARDA, IND, Marigold, Pharmaceutical industry, Cryptocurrency, Fine chemical

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the second quarter ended June 30, 2023.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today reported business highlights and financial results for the second quarter ended June 30, 2023.
  • Recognized $4.2 million and $7.6 million in net product revenues for the three and six months ended June 30, 2023, respectively.
  • Net product revenue consists of ZTALMY product sales, which was launched in the U.S. in the third quarter of 2022.
  • At June 30, 2023, the Company had cash, cash equivalents, and short-term investments of $175.3 million, compared to $240.6 million at December 31, 2022.

BridgeBio Pharma Announces Opportunity for Accelerated Approval Pathway in Limb-girdle Muscular Dystrophy Type 2I (LGMD2I/R9) Based on Glycosylated Alpha-dystroglycan (⍺DG) Levels and Announces First Patient Dosed in FORTIFY Phase 3 Study

Retrieved on: 
Monday, July 31, 2023
WMS, Creatine kinase, Myopathy, University, PALO, CDKL5 deficiency disorder, Fear, Pathology, Quality of life, Clinical trial, B cell, University of California, ClinicalTrials.gov, Accelerated approval (FDA), FKRP, Patient, Love, Cancer, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Safety, Neurology, Food, NSAD, LGMD, Disease, Pharmaceutical industry, Medical imaging, Agency

BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418.

Key Points: 
  • BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418.
  • The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418.
  • FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval.
  • “Currently, our Phase 2 data suggest that glycosylated αDG levels are improved and sustained over time following treatment with BBP-418.

European Commission approval of Ztalmy® (ganaxolone) for the adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder

Retrieved on: 
Monday, July 31, 2023
Orion, Ganaxolone, Seizure, CDD, CDKL5 deficiency disorder, Genetic disorder, Civil service commission, Patient, CDKL5, European Commission, European, Pharmaceutical industry, EU

The European Commission has granted marketing authorisation in the European Union (EU) for Ztalmy® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age.

Key Points: 
  • The European Commission has granted marketing authorisation in the European Union (EU) for Ztalmy® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age.
  • ZTALMY may be continued in patients 18 years of age and older.
  • Ganaxolone is developed by Marinus Pharmaceuticals, Inc. and under a collaboration agreement Orion has the right to sell and market ZTALMY in Europe.
  • Following the European Commission approval, Orion is focusing on making ZTALMY available for patients in Europe and has pricing and reimbursement processes planned or underway in Europe.

Marinus Pharmaceuticals Announces European Commission Approval of ZTALMY® (ganaxolone) for the Adjunctive Treatment of Epileptic Seizures Associated with CDKL5 Deficiency Disorder

Retrieved on: 
Monday, July 31, 2023
Pharmaceutical, Professional Services, Health, Finance, Seizure, Therapy, Civil service commission, Somnolence, Orion Corporation, Orion, European Directive on Traditional Herbal Medicinal Products, CDKL5 deficiency disorder, CDKL5, Hypersalivation, Allergen, Medication, Committee, Incidence, Patient, Physician, Marinus, Epilepsy, European Medicines Agency, CDD, Lancet, EC, Marigold, European Commission, Safety, Pharmaceutical industry, Fever

Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that the European Commission (EC) has granted approval of ZTALMY® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age.

Key Points: 
  • Marinus Pharmaceuticals , Inc. (Nasdaq: MRNS), a pharmaceutical company dedicated to the development of innovative therapeutics to treat seizure disorders, today announced that the European Commission (EC) has granted approval of ZTALMY® (ganaxolone) oral suspension for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients two to 17 years of age.
  • ZTALMY may be continued in patients 18 years of age and older.
  • In the Marigold open label extension study, patients treated with ZTALMY for at least 12 months (n=48) experienced a median 49.6% reduction in major motor seizure frequency.
  • In July 2021, Marinus and Orion Corporation entered into a collaboration agreement which grants Orion the right to commercialize ZTALMY in Europe.