New Drug Application

Travere Therapeutics Submits Supplemental New Drug Application to the U.S. Food and Drug Administration Seeking Full Approval of FILSPARI® (sparsentan) for the Treatment of IgA Nephropathy (IgAN)

Retrieved on: 
Monday, March 11, 2024
Angiotensin, SAN, European Commission, EMA, Erosion (morphology), Committee, New Drug Application, PROTECT, FDA, Food, Dialysis, Shanda, Hope, Risk, Civil service commission, Therapy, CHMP, CSL Vifor, EGFR, Patient, Immunoglobulin A, Kidney disease, Disease, Kidney, CMA, Proteinuria, Pharmaceutical industry

The sNDA is based on 2-year confirmatory results from the Phase 3 PROTECT Study, the only head-to-head study in IgAN versus an active comparator.

Key Points: 
  • The sNDA is based on 2-year confirmatory results from the Phase 3 PROTECT Study, the only head-to-head study in IgAN versus an active comparator.
  • “Since being introduced under accelerated approval, FILSPARI has positively impacted the lives of many people living with IgAN.
  • “FILSPARI is at the forefront of emerging new treatment options providing hope for a delay in kidney transplant or dialysis.
  • The FDA has 60 days from the receipt of the application to determine whether to accept it for review.

Tonix Pharmaceuticals Reports Improvement in “Brain Fog,” in Fibromyalgia Patients Treated with Tonmya™ in RESILIENT, an NDA-Enabling Phase 3 Clinical Trial, at the 6th International Congress on Controversies in Fibromyalgia

Retrieved on: 
Monday, March 11, 2024
Blood pressure, Tonix Pharmaceuticals, Woman, New Drug Application, FDA, Pain, Food, Sleep, Hope, Hydrogen chloride, FIQ, Human, Fatigue, Weight gain, Patient, Tablet, Insomnia, NDA, Brain, DSM-IV codes, Pharmaceutical industry

CHATHAM, N.J., March 11, 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 presentation of additional efficacy data from RESILIENT, the second positive Phase 3 study evaluating Tonmya (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia, at the 6th International Congress on Controversies in Fibromyalgia in Brussels, Belgium, March 7-8, 2024.

Key Points: 
  • We now report that the effect sizes of the five continuous key secondary outcomes measures ranged from 0.3 to 0.5.
  • The FIQ-R cognitive item showed nominal improvement in Tonmya-treated patients vs placebo-treated patients with a p=0.001 and effect size of 0.31.
  • RELIEF, the first Phase 3 trial of Tonmya 5.6 mg in fibromyalgia, was completed in December 2020.
  • We also believe the favorable tolerability and side effect profiles will be important to patients and doctors managing this debilitating condition on a long-term basis.”

Lexicon Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Monday, March 11, 2024
Research, New Drug Application, American Heart Association, Investment, Obesity, Growth, EASD, Arrowhead Conference, Patient, Chronic kidney disease, Post hoc, Neuropathic pain, Marketing, Heart failure, Hypertension, Hypertrophic cardiomyopathy, Left ventricular hypertrophy, NDA, Express, PROGRESS, AAK1, Risk, Conference, HCM, Clinical trial, Neuroscience, Lexicon, FDA, Research and development, Cardiology, U.S. FDA, Medicare, Administration, Travel, Pharmaceutical industry

ET

Key Points: 
  • ET
    THE WOODLANDS, Texas, March 11, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), today reported financial results for the three months ended December 31, 2023 and provided an update on key corporate milestones.
  • “2023 was a pivotal year for Lexicon,” said Lonnel Coats, Lexicon’s chief executive officer.
  • Unless otherwise stated, all comparisons are for the fourth quarter and full year of 2023 compared to the fourth quarter and full year of 2022.
  • Revenues: Revenues for the fourth quarter and full year of 2023 were $0.7 million and $1.2 million, respectively, primarily from the commercialization of INPEFA.

ARS Pharmaceuticals Reviews Recent Clinical Updates and Commercial Opportunity at neffy® Investor Day

Retrieved on: 
Thursday, March 7, 2024
Type 1, Allergy, SAN, Anxiety, EMA, Caregiver, CRL, New Drug Application, ARS, FDA, Food, Larynx, University of South Florida, Anaphylaxis, Vomiting, Entrepreneurship, Symantec Endpoint Protection, Angioedema, University, Event, Tongue, Webcast, Patient, Pediatrics, Adrenaline, Population growth, Prescription, Irritation, Face, Pharmaceutical industry

The event included presentations by members of the ARS Pharma management team and by two distinguished allergists, Dr. Jonathan Spergel, M.D., Ph.D. and Dr. Thomas B. Casale, M.D.

Key Points: 
  • The event included presentations by members of the ARS Pharma management team and by two distinguished allergists, Dr. Jonathan Spergel, M.D., Ph.D. and Dr. Thomas B. Casale, M.D.
  • Of the patients that do fill their prescription, 80-90% either do not carry or use their treatment as indicated.
  • In late-February, the Company announced positive clinical data from a Phase 2 trial evaluating neffy in adults with chronic spontaneous urticaria.
  • The webcast replay and accompanying slides from today’s investor event may be accessed through the Events & Presentations page in the Investors & Media section of the Company's website.

Applied Therapeutics Reports Fourth Quarter and Year-end 2023 Financial Results

Retrieved on: 
Wednesday, March 6, 2024
Janus, Sorbitol, Marketing, EMA, Doctor of Philosophy, New Drug Application, MAA, Interim analysis, P-value, FDA, Food, Growth, Treatment, INSPIRE, Priority review, PDUFA, Chairperson, Therapy, CHMP, European Medicines Agency, Patient, Private placement, PRV, NDA, Ageing, Pharmaceutical industry

The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • The FDA also noted that it is planning to hold an advisory committee meeting to discuss the application.
  • Govorestat was previously granted Pediatric Rare Disease designation and will qualify for a Priority Review Voucher (PRV) upon approval.
  • The Company expects a decision by the EMA in the fourth quarter of 2024.
  • Full study results will be presented at an upcoming medical conference, along with results of the Diabetic Peripheral Neuropathy sub-study, which are still being analyzed.

Tonix Pharmaceuticals Selects EVERSANA® to Support Launch Strategy and Commercialization Planning of Tonmya™ for the Management of Fibromyalgia

Retrieved on: 
Wednesday, March 6, 2024
Therapy, Fibromyalgia, Tonix Pharmaceuticals, New Drug Application, FDA, Food, Hydrogen chloride, Fatigue, Patient, Tablet, Pharmaceutical industry

CHATHAM, N.J., March 06, 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, announced today that EVERSANA®, a leading provider of commercialization services to the global life sciences industry, has been selected to support the launch strategy and commercial planning of Tonmya (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in the U.S. Specifically, EVERSANA will work with Tonix to assess the fibromyalgia landscape and help plan an efficient go-to-market strategy.

Key Points: 
  • Specifically, EVERSANA will work with Tonix to assess the fibromyalgia landscape and help plan an efficient go-to-market strategy.
  • “EVERSANA shares our commitment to delivering novel therapeutics to patients in need,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals.
  • “We are excited to further refine our business strategy for the anticipated launch of Tonmya in 2025.
  • As previously announced, Tonix’s second positive Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia.

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

Retrieved on: 
Tuesday, March 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.

Allyx Therapeutics Announces Expansion into Parkinson’s Disease Clinical Research with Lead Compound ALX-001 Following IND Acceptance

Retrieved on: 
Tuesday, March 5, 2024
Bangladesh Technical Education Board, SBIR, Research, New Drug Application, Synapse, FDA, Food, Conference, Small Business Innovation Research, Safety, Therapy, Pharmacokinetics, Patient, IND, Glutamate (neurotransmitter), Pharmaceutical industry

A 28-day safety study of ALX-001 in Parkinson’s disease patients will be initiated in Q1 2024.

Key Points: 
  • A 28-day safety study of ALX-001 in Parkinson’s disease patients will be initiated in Q1 2024.
  • “We are working with urgency to understand how the unique mechanism of action of ALX-001 at mGluR5, which preserves and protects synapses, can introduce meaningful clinical benefits for people living with Alzheimer’s disease and Parkinson’s disease,” commented Allyx Therapeutics co-founder and CEO Stephen Bloch, M.D.
  • As studies in Parkinson’s disease begin, the company will continue to advance clinical development of ALX-001 in Alzheimer’s disease, which is funded through grants from the National Institutes of Health and other Alzheimer’s disease research nonprofit organizations.
  • Fox Foundation for Parkinson’s Research contributes to research efforts around the potential of ALX-001, enabling our work to move into clinical research without delay,” commented Tim Siegert, Ph.D., chief operating officer and co-founder of Allyx Therapeutics.

Zevra Therapeutics Provides FDA Update on the PDUFA Action Date for Arimoclomol as a Treatment for Niemann-Pick Disease Type C

Retrieved on: 
Monday, March 4, 2024
New Drug Application, Niemann–Pick disease, FDA, Food, PDUFA, Therapy, NPC, Patient, Disease, NDA, Pharmaceutical industry

CELEBRATION, Fla., March 04, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra or the Company), a rare disease therapeutics company, today announced the U.S. Food and Drug Administration (FDA) has extended the review period for the New Drug Application (NDA) for arimoclomol, an investigational orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC). In addition, the FDA has set a new Prescription Drug User Fee Act (“PDUFA”) action date of September 21, 2024, and has re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled.

Key Points: 
  • In addition, the FDA has set a new Prescription Drug User Fee Act (“PDUFA”) action date of September 21, 2024, and has re-affirmed its intent to present the resubmission for discussion at an advisory committee meeting to be scheduled.
  • As previously reported, Zevra received acceptance of the NDA resubmission for arimoclomol in early January 2024, with an original PDUFA action date of June 21, 2024.
  • The FDA has determined that the additional information constitutes a Major Amendment to the NDA, thereby resulting in an extension of the PDUFA action date.
  • As the review continues, Zevra will maintain the early access program for arimoclomol and work tirelessly to bring this potential therapy to patients as soon as possible.”

EyePoint Pharmaceuticals Announces Appointment of Ramiro Ribeiro, M.D., Ph.D. as Chief Medical Officer

Retrieved on: 
Monday, March 4, 2024
Therapy, Ophthalmology, Digital health, New Drug Application, Alcon, Pontifical university, Retina, FDA, Geographic atrophy, University of Southern California, Safety, Compensated emancipation, University, Patient, Employment, Drug, NDA, Research

WATERTOWN, Mass., March 04, 2024 (GLOBE NEWSWIRE) -- EyePoint Pharmaceuticals, Inc. (NASDAQ: EYPT), a company committed to developing and commercializing therapeutics to help improve the lives of patients with serious retinal diseases, today announced the appointment of Ramiro Ribeiro, M.D., Ph.D. as Chief Medical Officer to succeed Dario Paggiarino, M.D. who has served as EyePoint’s Chief Medical Officer since 2016. Dr. Ribeiro is a trained retinal specialist and joins EyePoint from Apellis Pharmaceuticals, where he served as Vice President, Head of Clinical Development.

Key Points: 
  • who has served as EyePoint’s Chief Medical Officer since 2016.
  • Dr. Ribeiro is a trained retinal specialist and joins EyePoint from Apellis Pharmaceuticals, where he served as Vice President, Head of Clinical Development.
  • “Ramiro is a proven leader with a strong scientific and clinical background, and a track record of successfully bringing novel therapies for patients.
  • I also want to express my sincere gratitude to Dr. Paggiarino for his numerous contributions to EyePoint during his tenure.