Lennox–Gastaut syndrome

Aquestive Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
Mardi, mai 7, 2024
Angioedema, FDA, Mouth, NDA, Food, Swelling, Skin, Research, Attention deficit hyperactivity disorder, Insurance, Trial of the century, ADHD, Lennox–Gastaut syndrome, Dermatology, Seizure, Prescription, Public expenditure, Riluzole, Vomiting, Patent, Patient, Adrenaline, Common stock, Anaphylaxis, Epilepsy, Diazepam, PD, Therapy, Serdexmethylphenidate/dexmethylphenidate, Type, Pharmaceutical industry

“We continue to successfully execute on our growth strategy as demonstrated by our first quarter results,” said Daniel Barber, Chief Executive Officer of Aquestive.

Key Points: 
  • “We continue to successfully execute on our growth strategy as demonstrated by our first quarter results,” said Daniel Barber, Chief Executive Officer of Aquestive.
  • Total revenues increased to $12.1 million in the first quarter 2024 from $11.1 million in the first quarter 2023.
  • Research and development expenses increased to $5.9 million in the first quarter 2024 from $3.5 million in the first quarter 2023.
  • Non-GAAP adjusted EBITDA loss was $7.2 million in the first quarter 2024, compared to non-GAAP adjusted EBITDA loss of $3.9 million in the first quarter 2023.

Marinus Pharmaceuticals Provides Business Update and Reports First Quarter 2024 Financial Results

Retrieved on: 
Mercredi, mai 8, 2024
Biotechnology, Pharmaceutical, Health, Epilepsy, FDA, Lennox–Gastaut syndrome, Administration, Investment, NDA, Food, Research, Human services, New Drug Application, CDD, Security (finance), API, Development, Seizure, Data monitoring committee, GABAA, Therapy, Ganaxolone, TSC, Data, Patient, Tuberous sclerosis, Medication, File, GAAP, 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 first quarter ended March 31, 2024.

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 first quarter ended March 31, 2024.
  • “Our unwavering commitment is to develop innovative treatment options for individuals with seizure disorders,” said Scott Braunstein, M.D., Chairman and Chief Executive Officer of Marinus.
  • Increased full year 2024 projected U.S. ZTALMY net product revenues to between $33 and $35 million from a range of $32 and $34 million.
  • Marinus will focus its resources on the continued commercialization and development of ZTALMY, including the TrustTSC trial.

Komodo Health & Partners Present 33 Real-World Evidence Research Studies at ISPOR 2024

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Lundi, mai 6, 2024
Technology, Health, Venture Capital, Professional Services, Artificial Intelligence, Oncology, Other Science, Mental Health, Research, Software, Health Technology, Science, COVID-19, Clinical Trials, Komodo, University of Washington, Lennox–Gastaut syndrome, FDA, Public, University, Target, International Society, RWE, Pregnancy, Policy, AI, Prevalence, Colorectal cancer, Myriad Genetics, LGS, Chronic inflammatory demyelinating polyneuropathy, Neutropenia, CRC, Conference, Workflow, REMS, Genetics, Schizophrenia, Takeda Pharmaceutical Company, Mental health, Neurology, RWD, MPH, Patient, Hypertension, Epilepsy, Gestational age, Standard of care, Stillbirth, Pharmacoeconomics, ZS, Merck, ZS Associates, Breast cancer, Mitigation, Pharmaceutical industry

Komodo Health today announced the publication of 33 new health economics and outcomes research (HEOR) studies using its Healthcare Map and AI-powered technology platform.

Key Points: 
  • Komodo Health today announced the publication of 33 new health economics and outcomes research (HEOR) studies using its Healthcare Map and AI-powered technology platform.
  • The studies, to be featured at the 2024 International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Global Conference, shed light on important disease trends spanning women’s health, oncology, mental health, hypertension, neurology, and more.
  • Komodo Health researchers studied the differences in patient outcomes for pregnant women who continued or discontinued the use of antidepressants during pregnancy.
  • Komodo Health researchers analyzed whether the COVID-19 pandemic exacerbated inequities in breast cancer care through its impact on breast cancer staging and HCRU.

Encoded Therapeutics Provides Pipeline Updates From Its Vector Engineering Platform Ahead of Four Preclinical Presentations at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Mardi, avril 23, 2024
Biotechnology, Genetics, Health, Factorization of polynomials, Lennox–Gastaut syndrome, Brain, DRG, DSM-IV codes, SCN9A, Dorsal root ganglion, Dravet syndrome, ETF, Central nervous system disease, Gene silencing, LGS, Transgene, Neuroscience, Abstract, Code, CNS, GABAergic, Engineering, Res, Interneuron, MAPT, Angelman syndrome, Neuropathic pain, Central nervous system, Therapy, MicroRNA, Vaccine

Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.

Key Points: 
  • Presentations will highlight progress of preclinical programs for Angelman syndrome, Lennox-Gastaut syndrome (LGS), STXBP1-related disorders (STXBP1-RD), Alzheimer’s disease (MAPT) and neuropathic pain (SCN9A), together with innovations in the company’s vector engineering platform.
  • “Our ASGCT presentations showcase the depth and versatility of our vector engineering platform to develop highly specific precision therapies for CNS disorders with high unmet need,” said Stephanie Tagliatela, Chief Scientific Officer at Encoded.
  • “Combining our novel regulatory elements and transgenes provides control of expression in target cells, potentially enabling treatment of devastating, intractable diseases.
  • Engineered transgenes include transcription factors (eTFs) that upregulate the expression of endogenous genes as well miRNA sequences derived from Encoded’s miRNA discovery platform.

Lennox-Gastaut Syndrome (LGS) Foundation Announces Exciting New Research Funded in Time for the 9th Annual Walk 'n' Wheel for LGS Research

Retrieved on: 
Mardi, avril 16, 2024
Research program, Weill Cornell Medical Center, Hope, Risk, Research, LGS, Lennox–Gastaut syndrome, Friends, DRS, Walking, SAN

SAN DIEGO, April 16, 2024 /PRNewswire/ -- On April 20, 2024, the Lennox-Gastaut Syndrome (LGS) Foundation will mobilize families, friends, and communities around the world to 'Step Towards the Cures, Together' at their 9th Annual Walk 'n' Wheel for LGS Research.

Key Points: 
  • SAN DIEGO, April 16, 2024 /PRNewswire/ -- On April 20, 2024, the Lennox-Gastaut Syndrome (LGS) Foundation will mobilize families, friends, and communities around the world to 'Step Towards the Cures, Together' at their 9th Annual Walk 'n' Wheel for LGS Research.
  • Lennox-Gastaut Syndrome (LGS) is a rare, severe, life-threatening epilepsy syndrome that develops in children and leads to lifelong disability.
  • A critical part of this Research Program is funding patient-led research by awarding Cure LGS 365 Research Grants.
  • The LGS Foundation recently funded an exciting new research study conducted by Drs.

Avicanna Reports Full Year 2023 Audited Financial Results 

Retrieved on: 
Mardi, avril 2, 2024
Cream, Cannabidiol, Cannabinoid, Dravet syndrome, Brazilian Health Regulatory Agency, Interest, Medical cannabis, Seizure, Business, Lennox–Gastaut syndrome, Patient, Pharmacy, Active ingredient, Research, Acquisition, RDC, Ultra, Stock, CBD, Insurance, Health Canada, Bank statement, GMP, Dermatology, TSX, Therapy, Pharmaceutical industry

TORONTO, April 02, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce year end 2023 results and audited financial statements.

Key Points: 
  • TORONTO, April 02, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce year end 2023 results and audited financial statements.
  • Cash used in operations of $1.38 million; an 81% reduction compared to $7.4 million in 2022.
  • “2023 was a tremendous year for Avicanna where we solidified of our leadership position in the Canadian medical cannabis sector through our proprietary products and the launch of MyMedi.ca.
  • We have successfully demonstrated the scalability of our revenues and made significant improvements in our fundamentals while advancing all our business pillars.

Avicanna Announces the Canadian Launch of 10% CBD (THC Free) Proprietary Formulation

Retrieved on: 
Mercredi, mars 27, 2024
Cannabinoid, Absorption, Dravet syndrome, Seizure, Lennox–Gastaut syndrome, Patient, Research, CBD, Health Canada, TSX, THC, Plasma protein binding, RHO

TORONTO, March 27, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce the launch of RHO Phyto Micro Drop 100, a 10% CBD (THC free), proprietary oral formulation in Canada. RHO Phyto Micro Drop 100, is designed to deliver enhanced absorption of cannabinoids through an inverted emulsion technology. RHO Phyto Micro Drop 100 will be available to Canadian patients with medical authorization exclusively at the MyMedi.ca medical cannabis care platform.

Key Points: 
  • TORONTO, March 27, 2024 (GLOBE NEWSWIRE) -- Avicanna Inc. (“Avicanna” or the “Company) (TSX: AVCN) (OTCQX: AVCNF) (FSE: 0NN) a biopharmaceutical company focused on the development, manufacturing, and commercialization of plant-derived cannabinoid-based products is pleased to announce the launch of RHO Phyto Micro Drop 100, a 10% CBD (THC free), proprietary oral formulation in Canada.
  • RHO Phyto Micro Drop 100, is designed to deliver enhanced absorption of cannabinoids through an inverted emulsion technology.
  • RHO Phyto Micro Drop 100 will be available to Canadian patients with medical authorization exclusively at the MyMedi.ca medical cannabis care platform.
  • RHO Phyto Micro Drop 100 contains the same proprietary oral formulation as the product Trunerox™.

Takeda Announces Positive Topline Results from Phase 2 Study Evaluating Mezagitamab (TAK-079), a Potential Best-in-Class Anti-CD38 Monoclonal Antibody, for Primary Immune Thrombocytopenia

Retrieved on: 
Mercredi, mars 13, 2024
Neurology, Biotechnology, Pharmaceutical, Oncology, General Health, Health, FDA, Clinical Trials, Fast track (FDA), Psoriasis, Cell, Disease, Risk, Patient, Megakaryocyte, Immunoglobulin G, ITP, B cell, Quality of life, Platelet, Autoantibody, Gastrointestinal tract, CD38, Bleeding, Safety, Dravet syndrome, Blood, Lennox–Gastaut syndrome, Fatigue, Fiscal, Food, Autoimmune disease, Pharmaceutical industry

Takeda ( TSE:4502/NYSE:TAK ) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP).

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP).
  • An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results.
  • Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024.
  • Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

Ovid Therapeutics Reports Business Updates, Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Vendredi, mars 8, 2024
Graviton, Epilepsy, Pharmacy technician, CCM, Research, Transcranial magnetic stimulation, DS, Neuron, Vigabatrin, LGS, Psychosis, Biomarker, Element, Ligand Pharmaceuticals, Lennox–Gastaut syndrome, Patient, Acceleration, Medicine, Status epilepticus, Dravet syndrome, ROCK2, KCC2, Hemangioma, IND, Seizure, Pharmaceutical industry

In addition, Takeda is expected to read out results from two pivotal Phase 3 studies evaluating soticlestat.

Key Points: 
  • In addition, Takeda is expected to read out results from two pivotal Phase 3 studies evaluating soticlestat.
  • Of these potential future payments, Ovid sold a 13% interest to Ligand Pharmaceuticals for $30 million in October 2023.
  • Financial runway: Ovid anticipates its cash runway will support operations and clinical development programs into the first half of 2026.
  • Ovid also anticipates several events for its current pipeline programs and clinical results for soticlestat from Takeda in 2024.

Aquestive Therapeutics Reports Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Mardi, mars 5, 2024
PD, Research, FDA, ATM, Food, Growth, Anaphylaxis, Loss, PDUFA, Attention deficit hyperactivity disorder, Emergency, Safety, Therapy, Allergy, Patent, Diazepam, Patient, Serdexmethylphenidate/dexmethylphenidate, Lennox–Gastaut syndrome, Net, ADHD, Adrenaline, Clutch (eggs), Trial of the century, NDA, Riluzole, Seizure, Pharmaceutical industry

Aquestive received positive feedback in October 2023 from the U.S. Food and Drug Administration (FDA) on the Company's pivotal Phase 3 Pharmacokinetic (PK) clinical protocol for Anaphylm.

Key Points: 
  • Aquestive received positive feedback in October 2023 from the U.S. Food and Drug Administration (FDA) on the Company's pivotal Phase 3 Pharmacokinetic (PK) clinical protocol for Anaphylm.
  • Aquestive commenced dosing in December 2023 in the Phase 3 pivotal PK clinical study of Anaphylm.
  • Non-GAAP adjusted EBITDA loss was $2.8 million in the fourth quarter 2023, compared to a $9.6 million loss in the fourth quarter 2022.
  • During the fourth quarter 2023, the Company accessed capital net proceeds of $3.7 million under its "At-the-Market" (ATM) facility.