Tuberous sclerosis

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

Retrieved on: 
Wednesday, May 24, 2023
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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

Retrieved on: 
Tuesday, January 31, 2023
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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

Retrieved on: 
Monday, October 31, 2022
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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

Retrieved on: 
Wednesday, April 27, 2022
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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

Retrieved on: 
Tuesday, March 15, 2022
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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

Retrieved on: 
Tuesday, March 15, 2022
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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

Retrieved on: 
Friday, March 4, 2022
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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

Retrieved on: 
Tuesday, February 8, 2022
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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.

BridgeBio Pharma Announces Dosing of First Patient in Phase 1/2 Trial of Investigational Gene Therapy for Congenital Adrenal Hyperplasia (CAH)

Retrieved on: 
Thursday, January 27, 2022
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PALO ALTO, Calif., Jan. 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for the treatment of classic congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union.

Key Points: 
  • Our investigational gene therapy offers patients a potential single-dose intervention designed to restore their bodys hormone and steroid balance by making their own cortisol and aldosterone, said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy.
  • This is the second gene therapy trial we have initiated in less than four months, and we are excited to advance this trial and our other gene therapy programs in the hope of improving patients lives.
  • As an endocrinologist, its incredibly exciting to reimagine a new approach to treating this disease, added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy.
  • Adrenas Therapeutics, the affiliate company of BridgeBio focused on developing BBP-631 for CAH, is part of BridgeBio Gene Therapys portfolio.

Ovid Therapeutics Expands Epilepsy Franchise with Novel KCC2 Activators

Retrieved on: 
Monday, January 3, 2022
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The company seeks to optimize and accelerate development of these KCC2 transporter activators in epilepsies and potentially other neuropathic conditions.

Key Points: 
  • The company seeks to optimize and accelerate development of these KCC2 transporter activators in epilepsies and potentially other neuropathic conditions.
  • The KCC2 transporter is an exciting and novel target that we believe holds great promise in treating epilepsies, said Jeremy Levin, D.Phil, MB BChir, Chairman and CEO of Ovid.
  • Ovids focus in neuroscience and experience in developing novel anti-epileptics make it an optimal choice to advance KCC2 activators, including OV350, noted Iain Chessell, Global Head of Neuroscience, BioPharmaceuticals R&D, AstraZeneca.
  • OV350 is an early-stage compound that has shown encouraging in-vitro and in-vivo proof of concept in resistant forms of epilepsy.