New Drug Application

Supernus Announces Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 27, 2024
Central nervous system, Topiramate, Investigational New Drug, PD, Total, New Drug Application, FDA, Food, Growth, PDUFA, Safety, GAAP, CNS, Depreciation, IQVIA, Patient, AISRS, Attention deficit hyperactivity disorder, DSM-IV codes, ADHD, Phase, MDD, Prescription, MADRS, Pharmaceutical industry

ROCKVILLE, Md., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, today announced financial results for the fourth quarter and full year of 2023 and associated Company developments.

Key Points: 
  • Fourth quarter 2023 net sales of Qelbree® increased 97% to $46.4 million compared to fourth quarter 2022; Full year 2023 net sales of Qelbree increased 129% to $140.2 million compared to full year 2022.
  • Fourth quarter 2023 net sales of GOCOVRI® increased 10% to $32.0 million compared to fourth quarter 2022; Full year 2023 net sales of GOCOVRI increased 15% to $119.6 million compared to full year 2022.
  • Full year 2023 operating loss (GAAP) was $(5.3) million; Full year 2023 operating earnings (non-GAAP)(2), were $125.1 million.
  • ROCKVILLE, Md., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Supernus Pharmaceuticals, Inc. (Nasdaq: SUPN), a biopharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, today announced financial results for the fourth quarter and full year of 2023 and associated Company developments.

SpringWorks Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Highlights Recent Business Updates

Retrieved on: 
Tuesday, February 27, 2024
Research, New Drug Application, Vomiting, Common, Rash, Multiple myeloma, Patient, Orange Book, Investigational New Drug, Neoplasm, Nausea, Panitumumab, BCMA, Therapy, European Medicines Agency, Food and Drug Administration, Plantar fibromatosis, MAPK, NDA, EGFR, Diarrhea, Patent, B-cell maturation antigen, IND, MEK, NRAS, Security, EMA, Aes, MAA, FDA, Research and development, Food, TEA, RAF, Medicare, NCCN, Administration, Cancer, Pharmaceutical industry

Launched OGSIVEO in the U.S. and achieved net product revenue of $5.4 million in the first partial quarter of the launch.

Key Points: 
  • Launched OGSIVEO in the U.S. and achieved net product revenue of $5.4 million in the first partial quarter of the launch.
  • Presented additional patient-reported outcome data from the Phase 3 DeFi trial at the 2023 Connective Tissue Oncology Society Annual Meeting.
  • Revenues: OGSIVEO net product revenues were $5.4 million in the fourth quarter of 2023, the first partial quarter of the U.S. launch.
  • Cash Position: Cash, cash equivalents and marketable securities were $662.6 million as of December 31, 2023.

Tonix Pharmaceuticals Announces Positive Results from Clinical Pharmacokinetic Bridging Study of Tonmya™ to Support Development and Partnering in Japan and China

Retrieved on: 
Tuesday, February 27, 2024
Fibromyalgia, Tonix Pharmaceuticals, Investigational New Drug, New Drug Application, Marketing, Female, Hydrogen chloride, Data, PMDA, Patient, Male, Tablet, IND, NDA, NMPA, CTN, NCE, Pharmaceutical industry

CHATHAM, N.J., Feb. 27, 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 positive results from its clinical pharmacokinetic (PK) bridging study of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in healthy adult male and female ethnic Japanese and Chinese volunteers. Results indicate that key pharmacokinetic parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study. Tonmya was generally well tolerated in the ethnic Japanese and Chinese healthy volunteers. The company expects these data to fulfill the requirement for a bridging study, and to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE). Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.

Key Points: 
  • Results indicate that key pharmacokinetic parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study.
  • The company expects these data to fulfill the requirement for a bridging study, and to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE).
  • Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.
  • “This bridging study is an important first step as we begin evaluating the potential for approval and marketing Tonmya in Japan and China.

Day One Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Progress

Retrieved on: 
Monday, February 26, 2024
BRAF, Pfizer, Research, New Drug Application, Doctor of Philosophy, MD, Investment, FDA, Food, Priority review, Day One, Event, Patient, Medicine, Clinical trial, Nature Medicine, NDA, Head, Ageing, MEK, Pharmaceutical industry

BRISBANE, Calif., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its fourth quarter and full year 2023 financial results and highlighted recent corporate achievements.

Key Points: 
  • “We have a monumental year ahead of us at Day One with the upcoming PDUFA date for tovorafenib,” said Jeremy Bender, Ph.D., chief executive officer of Day One.
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
  • In November 2023, Nature Medicine published the registrational Phase 2 FIREFLY-1 trial results investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pediatric low-grade glioma (pLGG).
  • In the fourth quarter of 2023, Day One continued its commercial preparedness for the approval and launch of tovorafenib with the hiring of 18 sales representatives in the U.S.

Milestone Pharmaceuticals Announces Plan to Resubmit NDA for Etripamil for the Treatment of PSVT

Retrieved on: 
Monday, February 26, 2024
New Drug Application, Aes, FDA, Food, Paroxysmal supraventricular tachycardia, PDUFA, Refusal, PSVT, Patient, Type, NDA, Pharmaceutical industry

MONTREAL and CHARLOTTE, N.C., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Milestone® Pharmaceuticals Inc. (Nasdaq: MIST) today announced plans to resubmit the New Drug Application (NDA) for etripamil to the U.S. Food and Drug Administration (FDA) for paroxysmal supraventricular tachycardia (PSVT).

Key Points: 
  • Following the previously announced receipt of a Refusal to File letter , Milestone held a Type A Meeting with FDA.
  • FDA indicated that the timing of adverse events (AEs) in question had minimal impact on the overall characterization of the etripamil safety profile.
  • The Company expects a standard NDA review period following resubmission of the NDA for etripamil for PSVT, which is planned for 2Q2024.
  • "We thank FDA for its thoughtful consideration of our materials and direction on the NDA resubmission," said Joseph Oliveto, President, and Chief Executive Officer of Milestone Pharmaceuticals.

Ardelyx Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, February 22, 2024
Fosun Pharma, Kyowa Kirin, Research, New Drug Application, Investment, Dialysis, Growth, Sale, Death, Diarrhea, Webcast, Abdominal distension, Orphan, XBR, Chronic kidney disease, Incidence, Peritoneal dialysis, Rat, Record, Calendar, ESG, CKD, Hyperphosphatemia, Safety, Human, Nephrology, IBS-C, Master of Science, Gastroenterology, FDA, Food, Social, Emeritus, Dehydration, Risk, Constipation, Society, Patient, Pharmaceutical industry

WALTHAM, Mass., Feb. 22, 2024 (GLOBE NEWSWIRE) -- Ardelyx, Inc. (Nasdaq: ARDX), a biopharmaceutical company founded with a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs, today reported financial results for the fourth quarter and full year ended December 31, 2023 and provided a business update.

Key Points: 
  • During the fourth quarter of 2023, IBSRELA U.S. net product sales revenue was $28.1 million, reflecting 26% growth compared to the third quarter of 2023.
  • Ardelyx currently expects full-year 2024 U.S. net product sales revenue for IBSRELA to be between $140.0 and $150.0 million.
  • U.S. net product sales revenue in the fourth quarter of 2023 were $2.5 million.
  • U.S. net product sales revenue in 2023 for XPHOZAH was $2.5 million following its commercial launch in November 2023.

Rhythm Pharmaceuticals Reports Fourth Quarter 2023 Financial Results and Business Update

Retrieved on: 
Thursday, February 22, 2024
EMANATE, New Drug Application, Investment, Obesity, BMI, Webcast, Initiate, BBS, Stage 2, Hyperpigmentation, Documentation, Polyphagia, European, GAAP, European Medicines Agency, PCSK1, Type, Type 2, G&A, LG Chem, BV, Completeness, Shanda, Bardet–Biedl syndrome, Clinical trial, LEPR, Medicaid, IND, Prescription, Research, Hunger, Acquisition, Quarter, EMA, MC4R, FDA, Prevalence, PMDA, Patient, POMC, Body mass index, Pharmaceutical industry

-- Acquired global rights to oral MC4R agonist LB54640 from LG Chem --

Key Points: 
  • ET --
    BOSTON, Feb. 22, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today reported financial results and provided a business update for the fourth quarter and full year ended December 31, 2023.
  • Fourth Quarter and Full Year 2023 Financial Results:
    Cash Position: As of December 31, 2023, cash, cash equivalents and short-term investments were approximately $275.8 million, as compared to $333.3 million as of December 31, 2022.
  • S,G&A Expenses: S,G&A expenses were $32.4 million in the fourth quarter of 2023 and $117.5 million for the year ended December 31, 2023, compared to $26.3 million in the fourth quarter of 2022 and $92.0 million for the year ended December 31, 2022.
  • ET today to review its fourth quarter and year end 2023 financial results and recent business activities.

Axsome Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Tuesday, February 20, 2024
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Auvelity® net product sales were $49.0 million and $130.1 million for the fourth quarter and full year of 2023, the first full year of launch.

Key Points: 
  • Auvelity® net product sales were $49.0 million and $130.1 million for the fourth quarter and full year of 2023, the first full year of launch.
  • Total costs of revenue were $7.4 million and $26.1 million for the fourth quarter and full year of 2023, respectively.
  • Approximately 84,000 prescriptions were written for Auvelity in the fourth quarter of 2023, representing a 23% sequential increase versus the third quarter of 2023.
  • Axsome will host a conference call and webcast today at 8:00 AM Eastern to discuss fourth quarter and full year 2023 financial results as well as to provide a corporate update.

Ayala Pharmaceuticals Announces Completion of Enrollment in Phase 3 RINGSIDE Study Evaluating AL102 in Desmoid Tumors

Retrieved on: 
Tuesday, February 20, 2024
Progression-free survival, PFS, QOL, New Drug Application, Physician, Quality of life, Plantar fibromatosis, Patient, OTCQX, NDA, Nursing

REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that patient enrollment has been completed in the Phase 3 RINGSIDE study evaluating AL102 in desmoid tumors.

Key Points: 
  • REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that patient enrollment has been completed in the Phase 3 RINGSIDE study evaluating AL102 in desmoid tumors.
  • ”Completion of enrollment in RINGSIDE represents a significant milestone in the development of AL102,” said Kenneth Berlin, President and Chief Executive Officer of Ayala.
  • “There has been a high-level of enthusiasm from clinical trial investigators, support staff, and patients during the enrollment of RINGSIDE.
  • RINGSIDE has been designed as a registration study to support a New Drug Application (NDA) in desmoid tumors.

VERRICA PHARMACEUTICALS INVESTIGATION INITIATED by Former Louisiana Attorney General: Kahn Swick & Foti, LLC Investigates the Officers and Directors of Verrica Pharmaceuticals Inc. - VRCA

Retrieved on: 
Friday, March 8, 2024
Class Action Lawsuit, Professional Services, Legal, CMO, New Drug Application, KSF, Food, Skin, Symantec Endpoint Protection, NDA, Molluscum contagiosum

Former Attorney General of Louisiana, Charles C. Foti, Jr., Esq., a partner at the law firm of Kahn Swick & Foti, LLC (“KSF”), announces that KSF has commenced an investigation into Verrica Pharmaceuticals Inc. (NasdaqGM: VRCA).

Key Points: 
  • Former Attorney General of Louisiana, Charles C. Foti, Jr., Esq., a partner at the law firm of Kahn Swick & Foti, LLC (“KSF”), announces that KSF has commenced an investigation into Verrica Pharmaceuticals Inc. (NasdaqGM: VRCA).
  • On September 20, 2021, the Company disclosed the receipt of a Complete Response Letter (“CRL”) resulting from deficiencies at its contract manufacturer’s facility.
  • KSF’s investigation is focusing on whether Verrica’s officers and/or directors breached their fiduciary duties to its shareholders or otherwise violated state or federal laws.
  • About Kahn Swick & Foti, LLC
    KSF, whose partners include former Louisiana Attorney General Charles C. Foti, Jr., is one of the nation’s premier boutique securities litigation law firms.