DEE

Longboard Pharmaceuticals Announces Positive Interim Results from the Open-Label Extension (OLE) of the Phase 1b/2a PACIFIC Study Evaluating Bexicaserin in Participants with Developmental and Epileptic Encephalopathies (DEEs)

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月曜日, 6月 10, 2024
Biotechnology, General Health, Health, Pharmaceutical, Clinical Trials, Lethargy, Pneumonia, Patient, Appetite, OLE, Seizure, DEE, Lennox–Gastaut syndrome, COVID-19, DSM-IV codes, Medication, Sinusitis, Microsoft Access, Tolerability, Dravet syndrome, Lower respiratory tract infection, Pharmaceutical industry

Additionally, we saw compelling seizure reduction in the PACIFIC placebo patients who transitioned to bexicaserin in the OLE.

Key Points: 
  • Additionally, we saw compelling seizure reduction in the PACIFIC placebo patients who transitioned to bexicaserin in the OLE.
  • “Given the tremendous unmet need in patients living with DEEs, we are committed to rapidly advancing the development of bexicaserin.
  • The interim analysis was conducted when participants reached the approximate 6-month point in the OLE Study.
  • 100% of PACIFIC Study completers elected to enroll in the OLE with 95.1% (39 out of 41) remaining in the ongoing open-label study.

Longboard Pharmaceuticals Reports Full Year 2023 Financial Results and Provides Corporate Updates

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火曜日, 3月 12, 2024
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Medication, Seizure, Standard of care, LGS, DEE, DS, Safety, SAD, FDA, DSM-IV codes, Pharmaceutical industry

Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported full year 2023 financial results.

Key Points: 
  • Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported full year 2023 financial results.
  • “I am extremely proud of what our team has accomplished starting with the immense effort that went into the PACIFIC Study in participants with DEEs.
  • We appreciate the tremendous support from our existing and new shareholders who play an integral part in the continued success of Longboard.
  • Of note, results were on top of current standard of care; participants were typically on 3-4 other anti-seizure medications.

Praxis Precision Medicines Provides Corporate Update and Reports Fourth Quarter and Full-Year 2023 Financial Results

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火曜日, 3月 5, 2024
Central nervous system, Quarter, EMA, Focal seizure, Research, New Drug Application, UCB, FDA, Partnership, Food, Part, Quantitative electroencephalography, SCN8A, Tremor, SCN2A, Safety, PPR, European Medicines Agency, CNS, EEG, Pharmacokinetics, Part 1, Patient, Essential tremor, Medicine, Epilepsy, DEE, Trial of the century, NDA, Ageing, Central nervous system disease, Seizure, Pharmaceutical industry

Fourth Quarter and Full Year 2023 Financial Results:

Key Points: 
  • Fourth Quarter and Full Year 2023 Financial Results:
    As of December 31, 2023, Praxis had $81.3 million in cash and cash equivalents, compared to $100.5 million in cash, cash equivalents and marketable securities as of December 31, 2022.
  • Research and development expenses were $18.4 million for the fourth quarter of 2023, compared to $28.3 million for the fourth quarter of 2022.
  • General and administrative expenses were $9.9 million for the fourth quarter of 2023, compared to $13.1 million for the fourth quarter of 2022.
  • Praxis incurred a net loss of $26.9 million for the fourth quarter of 2023, including $5.7 million of stock-based compensation expense, compared to $41.2 million for the fourth quarter of 2022, including $6.4 million of stock-based compensation expense.

Longboard Pharmaceuticals Announces Positive Topline Data from the PACIFIC Study, a Phase 1b/2a Clinical Trial, for Bexicaserin (LP352) in Participants with Developmental and Epileptic Encephalopathies (DEEs)

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火曜日, 1月 2, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, Diarrhea, DSM-IV codes, Cannabidiol, Lethargy, Partnership, Constipation, Clobazam, Aes, Lamotrigine, Caregiver, Principal, Research, LGS, TID, DEE, ASMS, Medication, Data, Physician, Somnolence, MD, Doctor of Philosophy, DS, SAE, Seizure, Combination, Pharmacokinetics, Appetite, Participation, Patient, Diagnosis, Safety, Levetiracetam, MSCE, Pharmaceutical industry

Of the 52 participants enrolled in the study, 43 participants were randomized to bexicaserin (DS=4, LGS=24, DEE Other=15) and 9 to placebo (DS=0, LGS=5, DEE Other=4).

Key Points: 
  • Of the 52 participants enrolled in the study, 43 participants were randomized to bexicaserin (DS=4, LGS=24, DEE Other=15) and 9 to placebo (DS=0, LGS=5, DEE Other=4).
  • The median number of countable motor seizures per 28-day period at baseline was 38.8 in the bexicaserin group compared to 20.8 in the placebo group.
  • Most participants (85.7%) in the bexicaserin treated group (n=35) that started the maintenance period tolerated the highest dose (12 mg).
  • 100% of the participants who completed the PACIFIC Study elected to enroll in the ongoing 52-week open-label extension study.

Praxis Precision Medicines to Showcase Largest Pipeline of Precision Epilepsy Programs and Breadth of Commitment to Epilepsy Treatments at Upcoming Meetings

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火曜日, 11月 28, 2023
Molecule, Clinical trial, Audiogenic, Conference, PAME, Drug discovery, EEG, SCN2A, Drug development, Cerebrum, DEE, AES, Mortality, American Epilepsy Society, SCN8A, Standard of care, Pharmacokinetics, Annual general meeting, CNS, Part, Biomarker, Family Gathering, Generalized tonic–clonic seizure, Central nervous system, Patient, Safety, BTS, ASM, MES, Pharmaceutical industry

Presentations at the American Epilepsy Society (AES) Annual Meeting, as well as related scientific and patient advocacy meetings, will cover new patient data, updates on clinical progress and new paradigms for developing epilepsy therapies.

Key Points: 
  • Presentations at the American Epilepsy Society (AES) Annual Meeting, as well as related scientific and patient advocacy meetings, will cover new patient data, updates on clinical progress and new paradigms for developing epilepsy therapies.
  • ET [Platform D | Epilepsy Therapies]
    Summary: Combined preclinical and clinical data demonstrate PRAX-628 is differentiated from standard of care, with the potential to be best-in-class for focal epilepsy.
  • Summary: This study provides novel insights into the clinical validity of commonly used preclinical seizure models across the clinical epilepsy spectrum.
  • Oral presentation highlighting clinical updates from our PRAX-562 small molecule platform at the annual clinician, researcher, and family gathering for SCN8A

Praxis Precision Medicines Receives PRIME Designation from the EMA for elsunersen (PRAX-222) for Treatment of SCN2A Gain of Function Developmental Epilepsies

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木曜日, 11月 16, 2023
CNS, PRIME, EMA, Patient, Part, Seizure, DEE, Central nervous system, GOF, SLC6A1 epileptic encephalopathy, Pharmaceutical industry, Praxis

BOSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines , Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the European Medical Agency (EMA) has awarded its Priority Medicines (PRIME) designation for elsunersen (PRAX-222) for the treatment of SCN2A Gain of Function (GoF) developmental and epileptic encephalopathy (DEE).

Key Points: 
  • BOSTON, Nov. 16, 2023 (GLOBE NEWSWIRE) -- Praxis Precision Medicines , Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for central nervous system (CNS) disorders characterized by neuronal excitation-inhibition imbalance, today announced that the European Medical Agency (EMA) has awarded its Priority Medicines (PRIME) designation for elsunersen (PRAX-222) for the treatment of SCN2A Gain of Function (GoF) developmental and epileptic encephalopathy (DEE).
  • The EMA’s PRIME designation provides enhanced development support for priority medicines that target an unmet need and was granted based on the Part 1 data from the EMBRAVE study that showed a reduction in seizures and improvement in seizure free days, as well as preclinical data.
  • “Elsunersen has the potential to significantly impact the lives of patients with SCN2A-DEE and their families,” said Marcio Souza, president and chief executive officer of Praxis.
  • These medicines are considered priority medicines by the EMA, whose aim is to optimize development plans and accelerate evaluations so medicines that address significant unmet medical needs can reach patients faster.

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

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火曜日, 11月 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.

Longboard Pharmaceuticals Provides Corporate Update and Reports Third Quarter 2023 Financial Results

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木曜日, 11月 2, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, Other Health, Insurance, MSCE, ATM, FINANCIAL, MPH, Medication, Development of the human body, American Epilepsy Society, MD, Children's Hospital of Philadelphia, Dravet syndrome, SAD, Lennox–Gastaut syndrome, Caregiver, Clinical trial, Longboard, DSM-IV codes, NUALS Arts and Literary Annual Meet, Philadelphia, AES, Pacific studies, Investment, Hospital, DEE, Child, Pharmaceutical industry

Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported financial results for the third quarter ended September 30, 2023.
  • “We look forward to PACIFIC Study topline data in January 2024 evaluating LP352 in people living with Developmental and Epileptic Encephalopathies, or DEEs.
  • DEE caregivers and healthcare providers continue to be frustrated with the lack of new treatment options and remain focused on finding treatment options that balance safety, efficacy and burden.
  • We look forward to Phase 1 SAD data in the first half of 2024,” stated Kevin R. Lind, Longboard’s President and Chief Executive Officer.

Longboard Pharmaceuticals Announces Speakers for Investor & Analyst Event in New York on October 11, 2023

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木曜日, 10月 5, 2023
Science, Neurology, Other Science, Biotechnology, Research, Pharmaceutical, Health, Clinical Trials, SLC6A1 epileptic encephalopathy, Child, MD, DEE, MPH, Pacific studies, Hospital, MSCE, Philadelphia, Principal, KCNQ2 developmental and epileptic encephalopathy, Seizure, Medication, Development of the human body, DSM-IV codes, Children's Hospital of Philadelphia, International, Pharmaceutical industry

Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced the speakers for its upcoming Investor & Analyst Event focused on the Developmental and Epileptic Encephalopathy (DEE) landscape and LP352, a first-in-class 5-HT2C receptor superagonist in development for the potential treatment of seizures associated with DEEs.

Key Points: 
  • Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today announced the speakers for its upcoming Investor & Analyst Event focused on the Developmental and Epileptic Encephalopathy (DEE) landscape and LP352, a first-in-class 5-HT2C receptor superagonist in development for the potential treatment of seizures associated with DEEs.
  • *In-person attendance is limited to invited research analysts and institutional investors only.
  • All other guests are invited to view the live or archived webcast virtually on the Investor Relations section of Longboard’s website at https://www.longboardpharma.com/ .
  • The archived webcast will be available for at least 30 days following the event.

The SYNGAP1 Foundation Partners with Unite Us to Address Unmet Needs of SYNGAP1 Families and Improve Quality of Life

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火曜日, 9月 26, 2023
Learning, Partnership, Prevalence, Volume units used in petroleum engineering, Spectrum, CNF, SYNGAP1-related intellectual disability, National Partnership for Women & Families, Intellectual disability, Organization, AVP, Patient, Intellectual, DEE, Health, Chloride, Nursing, Mobile phone

Through the partnership, The SYNGAP1 Foundation and Unite Us aim to address the unmet social needs of people and families nationwide.

Key Points: 
  • Through the partnership, The SYNGAP1 Foundation and Unite Us aim to address the unmet social needs of people and families nationwide.
  • CNF partnered with Unite Us in March of 2022 to connect families in the CNF network to vital resources and has now sponsored the SYNGAP1 Foundation to connect more families to community-based resources.
  • Partners in the network are connected through Unite Us' shared technology platform, which enables them to send and receive secure electronic referrals, address people's social needs, and improve health across communities.
  • CNF, the SYNGAP1 Foundation, and Unite Us will securely connect people in need with community-based resources that can be difficult to navigate otherwise.