Prader–Willi syndrome

Tonix Pharmaceuticals Receives Rare Pediatric Disease Designation from the FDA for TNX-2900 for the Treatment of Prader-Willi Syndrome

Retrieved on: 
Monday, March 25, 2024
Eating, Prader–Willi syndrome, Obesity, Orphan drug, Appetite, Ageing, Tonix Pharmaceuticals, IND, Polyphagia, Priority review, Oxytocin, Marketing, FDA, Genetic disorder, PWS, Food, Pharmaceutical industry

CHATHAM, N.J., March 25, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to TNX-2900* (intranasal potentiated oxytocin), a proprietary magnesium (Mg2+)-potentiated formulation of intranasal oxytocin, to treat Prader-Willi syndrome (PWS) in children and adolescents. TNX-2900 was previously granted Orphan Drug designation by the FDA in 2022 for the treatment of PWS and the investigational new drug (IND) application was cleared by the FDA in 2023. The Company may be eligible to receive a transferable Priority Review Voucher if TNX-2900 for PWS is approved for marketing. Recently, vouchers have sold for approximately $100 million.

Key Points: 
  • TNX-2900 was previously granted Orphan Drug designation by the FDA in 2022 for the treatment of PWS and the investigational new drug (IND) application was cleared by the FDA in 2023.
  • The Company may be eligible to receive a transferable Priority Review Voucher if TNX-2900 for PWS is approved for marketing.
  • Recently, vouchers have sold for approximately $100 million.
  • “The Rare Pediatric Disease Designation is an important regulatory milestone in the development of TNX-2900.

Tonix Pharmaceuticals Announces Poster Presentation Describing Discovery of Novel Next-Generation Oxytocin Analogues at the American Chemistry Society (ACS) Spring 2024 Meeting

Retrieved on: 
Thursday, March 21, 2024
MGH, Eating, Obesity, Oxytocin, Patient, Weight loss, Binge eating disorder, University, Prader–Willi syndrome, Tonix Pharmaceuticals, IND, American Chemical Society, Magnesium, Adolescence, Insulin resistance, Hospital, Safety, ACS, Social anxiety disorder, Spectrum, Pain, Nasal administration, Poster

CHATHAM, N.J., March 21, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announces a poster presentation at the American Chemistry Society (ACS) Spring 2024 Meeting, held March 17-21, 2024, in New Orleans, Louisiana. A copy of the poster is available under the scientific presentations page of the Tonix website at www.tonixpharma.com.

Key Points: 
  • A copy of the poster is available under the scientific presentations page of the Tonix website at www.tonixpharma.com .
  • The poster presentation titled, Oxytocin Analogs with Enhanced Craniofacial Antinociceptive Effects in Low Magnesium Formulations, describes the discovery and characterization of novel oxytocin analogues that are candidate treatments for craniofacial pain, excessive eating (including Prader Willi Syndrome), and endocrinological conditions including bone health in autism and insulin resistance.
  • “Intranasal oxytocin has several potential therapeutic applications,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals.
  • The Phase 2 ‘STROBE’ study at MGH is investigating the efficacy and safety of TNX-1900 as a novel therapeutic agent to reduce binge eating frequency in adults with binge-eating disorder.

Acadia Appoints Elizabeth H. Z. Thompson, Ph.D. as Executive Vice President, Head of Research and Development

Retrieved on: 
Tuesday, April 2, 2024
Science, Neurology, Biotechnology, Research, Pharmaceutical, Health, Mental Health, Clinical Trials, AbbVie, Prader–Willi syndrome, Scripps Research, IGM, Horizon, Delusion, Executive, Harvey Mudd College, Patient, Acquisition, Rett syndrome, Doctor of Philosophy, Myenteric plexus, Chemistry, Research and development, Hallucination, Rare disease, Amgen, ACAD, FDA, Pharmaceutical industry

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the appointment of Dr. Elizabeth H. Z. Thompson as Executive Vice President, Head of Research and Development.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced the appointment of Dr. Elizabeth H. Z. Thompson as Executive Vice President, Head of Research and Development.
  • Dr. Thompson will lead research and development at Acadia and serve as a member of the company’s Executive Leadership Team, reporting to Steve Davis, Chief Executive Officer of Acadia.
  • “Liz is a preeminent drug research and development leader with extensive experience in the advancement and commercialization of first in class therapies,” said Steve Davis, Chief Executive Officer.
  • She joined Horizon in 2018 and served in roles of increasing responsibility that culminated at Executive Vice President, Research and Development.

HARMONY BIOSCIENCES INITIATES GLOBAL PHASE 3 REGISTRATIONAL TRIAL (TEMPO STUDY) OF PITOLISANT IN PATIENTS WITH PRADER-WILLI SYNDROME

Retrieved on: 
Wednesday, April 3, 2024
ODD, Narcolepsy, Prader–Willi syndrome, TEMPO, Cataplexy, Patient, Ageing, EDS, Safety, Harmony, Plasma protein binding, FDA, PWS, Pitolisant, Pharmaceutical industry

Approximately 15,000 – 20,000 people in the U.S. are living with PWS, the majority experiencing behavioral symptoms and more than half with EDS.

Key Points: 
  • Approximately 15,000 – 20,000 people in the U.S. are living with PWS, the majority experiencing behavioral symptoms and more than half with EDS.
  • "Our progress in expanding the clinical applications of pitolisant in PWS and in other rare diseases, remains promising.
  • Pitolisant is marketed as WAKIX® in the U.S. and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy.
  • Pitolisant is not approved for use in patients with PWS and is currently being evaluated as an investigational agent in this patient population.

Soleno Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full-Year 2023 Financial Results

Retrieved on: 
Wednesday, March 6, 2024
Therapy, Quarter, Research, FDA, Food, HQ, Sale, Prader–Willi syndrome, Exercise, PWS, Human resources, Patient, NDA, Pharmaceutical industry

REDWOOD CITY, Calif., March 06, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today provided a corporate update, and reported financial results for the fourth quarter and full-year ended December 31, 2023.

Key Points: 
  • REDWOOD CITY, Calif., March 06, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today provided a corporate update, and reported financial results for the fourth quarter and full-year ended December 31, 2023.
  • DCCR continued to be generally well-tolerated in the randomized withdrawal period with no new or unexpected safety signals.
  • “Following recent feedback from the FDA, Soleno remains focused on preparing an NDA submission for DCCR in PWS mid-2024,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics.
  • Total other income (expense), net, was $2.6 million for the quarter ended December 31, 2023, and $0.1 million in the same period of 2022.

PRISM MarketView Highlights Work of Emerging Companies on Rare Disease Day

Retrieved on: 
Thursday, February 29, 2024
Rare disease, Therapy, Regenerative Medicine Advanced Therapy, FAP, Concha bullosa, Sulindac, Chemistry, Fast track (FDA), Muscular dystrophy, FDA, Sensation, News, Eflornithine, DMD, Prader–Willi syndrome, BLA, Chromosome, RDEB, PDUFA, Skin, Voyager, PRISM, Duchenne muscular dystrophy, Safety, Neurocrine Biosciences, Rare Disease Day, Genetics, Fatigue, Walking, PWS, Patient, DSM-IV codes, Medicine, Disease, Exosome, FA, CMC, Priority review, Pharmaceutical industry

All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.

Key Points: 
  • All areas of medicine, and all organs and body systems, are impacted by rare diseases, including rare neurological and neuromuscular diseases, metabolic, skin and bone diseases, and chromosomal disorders.
  • Today on Rare Disease Day, PRISM MarketView highlights emerging companies working to deliver life changing treatments for those living with rare diseases.
  • RDEB is a severe rare disease characterized by extremely fragile, and extensive blistering and wounds.
  • Capricor is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases.

Harmony Biosciences Reports Fourth Quarter and Full Year 2023 Financial Results and Business Updates

Retrieved on: 
Thursday, February 22, 2024
Acquisition, PDUFA date, Marketing, Total, Research, Narcolepsy, Share repurchase, FDA, Food, Shanda, Treatment, Research and development, Growth, PDUFA, Generator, Track, Fragile X syndrome, Conference, Prader–Willi syndrome, GAAP, Food and Drug Administration, Administration, Patient, Initiate, Development, Benchmarking, Bank statement

Net product revenue for the quarter ended December 31, 2023 was $168.4 million, compared to $128.3 million for the same period in 2022.

Key Points: 
  • Net product revenue for the quarter ended December 31, 2023 was $168.4 million, compared to $128.3 million for the same period in 2022.
  • The average number of patients on WAKIX increased by approximately 350 sequentially to approximately 6,150 for the quarter ended December 31, 2023.
  • Total Operating Expenses in the fourth quarter of 2023 included one-time Zynerba transaction related costs of $9.8 million.
  • ET
    We are hosting our fourth quarter and full year 2023 financial results conference call and webcast today at 8:30 a.m. Eastern Time.

Tonix Pharmaceuticals to Participate in the 2024 BIO CEO & Investor Conference

Retrieved on: 
Tuesday, February 20, 2024
Infection, Therapy, Fibromyalgia, TNX-102, Tonix Pharmaceuticals, Orphan drug, Smallpox, NIAID, Research, Annual report, NIH, CD40L, FDA, BIO, Prader–Willi syndrome, Sumatriptan, Government, Upsher-Smith Laboratories, Conference, Disease, Autoimmunity, University, COVID-19, PWS, Patient, Acute stress disorder, Security (finance), Long, Breakthrough therapy, Risk, IND, NDA, Failure, Long COVID, Cancer, Pharmaceutical industry

CHATHAM, N.J., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals, will present at the BIO CEO & Investor Conference on February 26, 2024 at 1:30 p.m.

Key Points: 
  • CHATHAM, N.J., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals, will present at the BIO CEO & Investor Conference on February 26, 2024 at 1:30 p.m.
  • Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering.
  • Tonix intends to meet with the FDA in the first half of 2024 and submit an NDA for the approval of Tonmya for the management of fibromyalgia in the second half of 2024.
  • A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2024.

Acadia Pharmaceuticals Reports Fourth Quarter and Full Year 2023 Financial Results and Operating Overview

Retrieved on: 
Tuesday, February 27, 2024
Biotechnology, FDA, Other Health, Health, General Health, Pharmaceutical, Mental Health, Other Science, Research, Science, Clinical Trials, Senior, PIN, Growth, Schizophrenia, Prader–Willi syndrome, COMPASS, Polyphagia, Rett syndrome, GAAP, ACAD, Pharmaceutical industry

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD), today announced its financial results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Acadia Pharmaceuticals Inc. (Nasdaq: ACAD), today announced its financial results for the fourth quarter and full year ended December 31, 2023.
  • Initiated a Phase 2 clinical trial of ACP-204 for the treatment of Alzheimer’s disease psychosis in the fourth quarter of 2023.
  • Net product sales of NUPLAZID were $143.9 million for the fourth quarter of 2023, an increase of 5% as compared to $136.5 million for the fourth quarter of 2022.
  • Net product sales of DAYBUE were $87.1 million for the fourth quarter of 2023 and $177.2 million for the full year 2023.

The Angelman Syndrome Foundation (ASF) and Cedars-Sinai Guerin Children's team up to launch new clinic for families living with Angelman syndrome

Retrieved on: 
Tuesday, February 20, 2024
Social work, Angelman syndrome, Diet, Physician, Rush University Medical Center, Prader–Willi syndrome, Hope, Common, Occupational therapy, Cedars-Sinai Medical Center, Genetics, DSM-IV codes, Walking, Patient, MPH, Speech, Spectrum, Cerebral palsy, UBE3A, Neurology, Gastroenterology, Seizure, Pharmaceutical industry

AURORA, Ill., Feb. 20, 2024 /PRNewswire-PRWeb/ -- The Angelman Syndrome Foundation is proud to announce a new collaboration with Cedars-Sinai Guerin Children's to launch a new clinic for patients with Angelman syndrome, a rare neuro-genetic disorder.

Key Points: 
  • The Angelman Syndrome Foundation is proud to announce a new collaboration with Cedars-Sinai Guerin Children's to launch a new clinic for patients with Angelman syndrome, a rare neuro-genetic disorder.
  • AURORA, Ill., Feb. 20, 2024 /PRNewswire-PRWeb/ -- The Angelman Syndrome Foundation is proud to announce a new collaboration with Cedars-Sinai Guerin Children's to launch a new clinic for patients with Angelman syndrome, a rare neuro-genetic disorder.
  • The clinic is led by Cesar Ochoa-Lubinoff, MD, MPH , director of Developmental-Behavioral Pediatrics at Cedars-Sinai Guerin Children's, who previously co-directed the Angelman syndrome clinic at Rush University Medical Center in Chicago.
  • "In my experience, families facing Angelman syndrome are resilient, but they need a supportive medical team in place," Ochoa-Lubinoff said.