Tuberous sclerosis

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

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星期三, 五月 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.

Marinus Pharmaceuticals Provides Update on the Phase 3 RAISE Trial and Reports Preliminary First Quarter 2024 Financial Results

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星期一, 四月 15, 2024
Oncology, Health, FDA, Clinical Trials, Research, Science, Pharmaceutical, Epilepsy, Administration, Marinus, Drug discovery, Investment, Safety, Food, Human services, New Drug Application, Development, Data monitoring committee, Therapy, Ganaxolone, Patient, Tuberous sclerosis, DMC, Medication, Pharmaceutical industry

Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024.

Key Points: 
  • 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 expects to complete enrollment in the Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV with approximately 130 patients in mid-May 2024.
  • Marinus estimates preliminary unaudited cash, cash equivalents, and short-term investments of $113.3 million as of March 31, 2024.

Briefing on Capitol Hill Urges More Government Action and Investment in the Epilepsies

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星期三, 五月 24, 2023
Epilepsy, Seizure, FY, Capitol Hill, Traumatic brain injury, Research program, SCN8A, Quality of life, Human, Tuberous sclerosis, Research, Child, Doctor of Philosophy, Government, Organization, TSC, Congress, National Hospital for Neurology and Neurosurgery, Children's National Hospital, Patient, Epilepsy Foundation, Tuberous sclerosis complex tumor suppressors, Fiscal year, Letter, Neurology, Pharmaceutical industry, SUDEP

WASHINGTON, May 24, 2023 /PRNewswire/ -- In cooperation with the Congressional Epilepsy Caucus, organizations including the Epilepsy Foundation, Epilepsies Action Network, CURE Epilepsy, Rare Epilepsy Network and DEE-P Connections hosted a briefing on Capitol Hill on May 17 to educate about the epilepsies and call for more government action and investment in the epilepsies.

Key Points: 
  • WASHINGTON, May 24, 2023 /PRNewswire/ -- In cooperation with the Congressional Epilepsy Caucus, organizations including the Epilepsy Foundation, Epilepsies Action Network, CURE Epilepsy, Rare Epilepsy Network and DEE-P Connections hosted a briefing on Capitol Hill on May 17 to educate about the epilepsies and call for more government action and investment in the epilepsies.
  • (R-NC-3) and Jim Costa (D-CA-21) spoke about their personal connections to epilepsy and the importance of the caucus.
  • "My son developed epilepsy when he was 14 and it took us months to get the right treatment for him," said Congressman Murphy.
  • The annual total healthcare burden for people living with the epilepsies and/or seizures is at least $54 billion.

Jazz Pharmaceuticals receives NICE recommendation for the reimbursement of Epidyolex® (cannabidiol) for the treatment of seizures associated with tuberous sclerosis complex in England

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星期二, 一月 31, 2023
Newton, Dravet syndrome, Tuberous sclerosis, TSA, Heart, Jazz Pharmaceuticals, EMA, GW, Strategic planning, Medicines and Healthcare products Regulatory Agency, Seizure, NHS, Medicine, Skin, TSC, Epilepsy, Lennox–Gastaut syndrome, Cannabidiol, NICE, Wales, GW Pharmaceuticals, Brain, MHRA, Patient, National Institute for Health and Care Excellence, Ann Mercy Hunt, Clobazam, Healthcare Improvement Scotland, European Medicines Agency, Pharmaceutical industry

TSC can be diagnosed in infancy but many children are not diagnosed until later in childhood when their seizures begin and other symptoms appear.

Key Points: 
  • TSC can be diagnosed in infancy but many children are not diagnosed until later in childhood when their seizures begin and other symptoms appear.
  • [4] It is estimated that between 3,700 and 11,000 people in the UK live with TSC.
  • TSC is a very difficult to manage condition, with common issues including epilepsy in eight out of ten people.
  • Please note that, in relation to this Jazz media announcement, no honorarium was provided to the Tuberous Sclerosis Association.

Marinus Pharmaceuticals Announces $32.5 Million Revenue Interest Financing Agreement with Sagard Healthcare Partners

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星期一, 十月 31, 2022
Health, Pharmaceutical, Morgan Stanley, U.S. Securities and Exchange Commission, Brain, Private Securities Litigation Reform Act, Culture, Forward-looking statement, Patient, IV, Tuberous sclerosis, Adult, CDKL5, Clinical trial, Marinus, Company, EMA, Security (finance), Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo, Medication, FDA, Epilepsy, Ganaxolone, CV, Seizure, GABAA, Ecosystem, Achievement, COVID-19, Therapy, Pharmaceutical industry, Sagard

The total amount payable by Marinus to Sagard is capped at 1.9x of the upfront payment, with the ability to redeem the revenue interest financing agreement at lower multiples within the first four years from funding.

Key Points: 
  • The total amount payable by Marinus to Sagard is capped at 1.9x of the upfront payment, with the ability to redeem the revenue interest financing agreement at lower multiples within the first four years from funding.
  • Upon achievement of the cap amount, the revenue interest financing agreement will terminate.
  • Sagard is excited to complete this transaction with Marinus and help support the ongoing launch of ZTALMY and the development of ganaxolone, said Raja Manchanda, Partner at Sagard.
  • As a firm, Sagard operates platforms that invest across five asset classes: venture capital, private equity, private credit, real estate and healthcare royalties.

Praxis Precision Medicines Showcases Largest Targeted Epilepsy Portfolio in Industry at 2022 Epilepsy Day

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星期三, 四月 27, 2022
Drug development, Calcium channel, Judgement, IND, Media, U.S. Securities and Exchange Commission, COVID-19, Twitter, Genetics, Regulation of tobacco by the U.S. Food and Drug Administration, NASDAQ, SYNGAP1, SCN2A, Security (finance), Annual report, Epilepsy, Food, Private Securities Litigation Reform Act, CNS, Central nervous system, Brain, GLOBE, Lists of diseases, Patient, Goal, LinkedIn, ASO, Review, Event, Trial of the century, Update, Drug discovery, Tuberous sclerosis, Webcast, Investor, Movement disorders, SCN8A, Central nervous system disease, FDA, TSC, Pharmaceutical industry, Praxis

We have made considerable progress enabling a high-value portfolio across each of our three franchises, and we look forward to sharing our continued progress across the entire Praxis pipeline.

Key Points: 
  • We have made considerable progress enabling a high-value portfolio across each of our three franchises, and we look forward to sharing our continued progress across the entire Praxis pipeline.
  • A replay of the webcast will be available on Praxis website for 90 days following the event.
  • Praxis Precision Medicines is a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for CNS disorders characterized by neuronal excitation-inhibition imbalance.
  • Praxis has established a broad portfolio with multiple programs, including product candidates across psychiatric disorders, movement disorders, epilepsy and other exploratory CNS indications, with three clinical-stage product candidates.

Nobelpharma America Announces Presence at the National Association of Pediatric Nurse Practitioners 43rd National Conference on Pediatric Health Care in Dallas and Online

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星期二, 三月 15, 2022
Epilepsy, Patient, Practitioner, Tuberous sclerosis, NPA, Conference, Itch, Multimedia, TSC, Nose, Skin, National Association, LLC, Child, Pediatric nurse practitioner, Advertising, Red, CEO, Hamartoma, Bleeding, Degenerative disease, Lists of diseases, Chin, National Conference, Autism, Disease, Medicine, Pharmaceutical industry, Online shopping

BETHESDA, Md., March 15, 2022 /PRNewswire/ -- Nobelpharma America, LLC, a pharmaceutical and medical device company headquartered in Bethesda, Maryland,today announcedits presence at the 43rd National Conference on Pediatric Health Care , organized by the National Association of Pediatric Nurse Practitioners, with booth #359 onsite in Dallas March 22-24, 2022 and an online booth at the virtual conference April 26-29, 2022.

Key Points: 
  • BETHESDA, Md., March 15, 2022 /PRNewswire/ -- Nobelpharma America, LLC, a pharmaceutical and medical device company headquartered in Bethesda, Maryland,today announcedits presence at the 43rd National Conference on Pediatric Health Care , organized by the National Association of Pediatric Nurse Practitioners, with booth #359 onsite in Dallas March 22-24, 2022 and an online booth at the virtual conference April 26-29, 2022.
  • "Our presence at the National Conference on Pediatric Health Care enables us to engage with child health care experts who are often on the front lines of supporting children's health concerns."
  • Nobelpharma America will have an interactive live booth and a virtual exhibit as part of their involvement at the National Conference on Pediatric Health Care.
  • Both booths will display corporate information on Nobelpharma America as well as its efforts in the rare disease community.

Ovid Therapeutics Reports Business Update, Fourth Quarter and Full Year 2021 Financial Results

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星期二, 三月 15, 2022
KCC2, NDA, Epilepsy, SEC, Program, Research, Technology, Acquisition, GLOBE, Patient, Dravet syndrome, Achievement, Lennox–Gastaut syndrome, GAAP, Budget, Risk, COVID-19, Angelman syndrome, AstraZeneca, Spasm, Phase 3, Columbia University, Forward-looking statement, MB, Security (finance), NASDAQ, Lists of diseases, IND, Marinus, GABA, Seizure, Clinical trial, Tuberous sclerosis, Company, Fragile X syndrome, Takeda Pharmaceutical Company, Royalty, U.S. Securities and Exchange Commission, New Drug Application, RNA, Pharmaceutical industry, Bank, Takeda, CDKL5

NEW YORK, March 15, 2022 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with epilepsies and seizure-related disorders, today provided a business update and reported financial results for the fourth quarter and full year ended December 31, 2021.

Key Points: 
  • NEW YORK, March 15, 2022 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with epilepsies and seizure-related disorders, today provided a business update and reported financial results for the fourth quarter and full year ended December 31, 2021.
  • R&D strategy focused on novel epilepsy medicines: Ovid is harnessing its deep expertise to advance two novel mechanisms of action for treating epilepsies.
  • Fourth Quarter and Year-EndDecember 31, 2021Financial Results
    Cash and cash equivalents as of December 31, 2021 was$187.8 million.
  • General and administrative expenses were$8.3 millionand$37.2 millionfor the fourth quarter and year endedDecember 31, 2021, respectively, as compared to$10.4 millionand$30.6 millionfor the same periods in 2020.

Ovid Therapeutics to Participate at the Cowen 42nd Annual Health Care Conference

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星期五, 三月 4, 2022
Risk, Lennox–Gastaut syndrome, KCC2, Patient, Research, Forward-looking statement, RNA, Dravet syndrome, Neuro, Webcast, SEC, COVID-19, Conference, Acquisition, Spasm, Epilepsy, GABA, Angelman syndrome, Technology, Lists of diseases, Seizure, Takeda, Event, Acceleration, Tuberous sclerosis, GLOBE, NASDAQ, Pharmaceutical industry

NEW YORK, March 04, 2022 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people affected by epilepsies and rare neurological disorders, today announced that management will participate virtually in the Orphan Neuro and Neuromuscular panel discussion at the Cowen 42nd Annual Health Care Conference on Monday, March 7, 2022, at 9:10 a.m.

Key Points: 
  • NEW YORK, March 04, 2022 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people affected by epilepsies and rare neurological disorders, today announced that management will participate virtually in the Orphan Neuro and Neuromuscular panel discussion at the Cowen 42nd Annual Health Care Conference on Monday, March 7, 2022, at 9:10 a.m.
  • Additionally,Ovidmaintains a significant financial interest in the future regulatory development and potential commercialization of soticlestat, which Takeda is responsible for advancing globally.
  • Two Phase 3 trials for soticlestat in Dravet syndrome and Lennox-Gastaut syndrome are active.
  • You can identify forward-looking statements because they contain words such as will, appears, believes and expects.

Healx and Ovid Therapeutics to Enter Strategic Partnership

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星期二, 二月 8, 2022
NASDAQ, Tuberous sclerosis, Industry, Acceleration, GLOBE, RNA, GABA, Forward-looking statement, Dravet syndrome, Fragile X syndrome, MB, Spasm, Takeda, Acquisition, Behavioral Problems, Lennox–Gastaut syndrome, Seizure, Research, Combination, Twitter, NEW, CNS, Therapy, Clinical trial, Risk, Technology, Patient, Drug combination, Epilepsy, Brain, Lists of diseases, SEC, CEO, Drug discovery, Genetics, Angelman syndrome, KCC2, Interest, Ono Pharmaceutical, Government budget balance, Disease, Partnership, Globe, LinkedIn, COVID-19, Medical imaging, Pharmaceutical industry, Sildenafil

NEW YORK, N.Y. and CAMBRIDGE, United Kingdom, Feb. 08, 2022 (GLOBE NEWSWIRE) -- Ovid Therapeutics (NASDAQ:OVID) (“Ovid”), a biopharmaceutical company committed to developing medicines that transform the lives of people with neurological diseases, and Healx, the AI-powered, patient-inspired technology company pioneering the next generation of drug discovery, announced that Healx has secured from Ovid an exclusive option to license rights to develop and commercialize gaboxadol. Under the agreement, Healx plans to investigate the compound as part of a potential combination therapy for Fragile X syndrome, as well as a treatment for other indications.

Key Points: 
  • Gaboxadol has previously been tested as a monotherapy for Fragile X syndrome in a Phase 2a clinical study by Ovid.
  • Alongside the initial focus on Fragile X syndrome, Healx plans to use its cutting-edge indication expansion technology to identify other conditions that could also be treated by gaboxadol.
  • Should Healx exercise its option, Ovid will receive milestone payments for specific clinical, regulatory, and commercial achievements associated with gaboxadols development.
  • Additionally, Healx will pay Ovid tiered royalties on net sales associated with marketed therapies containing gaboxadol.