New Drug Application

Travere Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, February 15, 2024
SPARTAN, Research, New Drug Application, Growth, Table, CSL Vifor, DSM-IV codes, Webcast, Conditional, HCU, FY, Medication, Diagnosis, Plasma, Bank statement, Committee, FSGS, UPCR, GAAP, Therapy, CHMP, CMA, International, SAN, Total, Shanda, Kidney disease, Safety, Clinical trial, Quarter, FDA, Food, Risk, Income tax, PMDA, Patient, Disease, Kidney, Pivotal, Proteinuria, Pharmaceutical industry

Net product sales for the fourth quarter of 2023 were $39.9 million, compared to $25.8 million for the same period in 2022.

Key Points: 
  • Net product sales for the fourth quarter of 2023 were $39.9 million, compared to $25.8 million for the same period in 2022.
  • Research and development (R&D) expenses for the fourth quarter of 2023 were $59.7 million, compared to $58.1 million for the same period in 2022.
  • Total other income, net, for the fourth quarter of 2023 was $5.7 million, compared to $1.1 million for the same period in 2022.
  • ET to discuss company updates as well as fourth quarter and full year 2023 financial results.

Agios Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, February 15, 2024
Agios Pharmaceuticals, Security, AGIO, Total, Research, New Drug Application, ASH, FDA, Research and development, PKU, Growth, Sale, Exposition, Thalassemia, Society, MDS, Phenylketonuria, Administration, Sickle cell disease, Anti-transglutaminase antibodies, Patient, Public expenditure, Net, Small RNA, IND, Pharmaceutical industry

CAMBRIDGE, Mass., Feb. 15, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today reported business highlights and financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • Launch: Generated $7.1 million in U.S. net revenue for the fourth quarter of 2023, a 4 percent decrease from the third quarter of 2023, primarily driven by lower customer inventory levels at the end of the fourth quarter of 2023, partially offset by favorable gross-to-net adjustments.
  • A total of 109 patients are on PYRUKYND® therapy, a 9 percent increase from the third quarter of 2023.
  • Cost of Sales: Cost of sales was $0.6 million for the fourth quarter of 2023 and $2.9 million for the full year ended Dec. 31, 2023.
  • ET to discuss fourth quarter and full year 2023 financial results and recent business highlights.

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: 
Wednesday, February 14, 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.

PolyPid Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, February 14, 2024
SHIELD, Marketing, Research, New Drug Application, Interim analysis, Completeness, PLEX, Conference, Monoclonal antibody, Dicarboxylic acid, Patient, Survival, PIPE, G&A, Pharmaceutical industry

Financial results for the full year ended December 31, 2023

Key Points: 
  • Financial results for the full year ended December 31, 2023
    R&D expenses, net for the year ended December 31, 2023, were $16.1 million, compared to $28 million in 2022.
  • G&A expenses for the year ended December 31, 2023, were $5.5 million, compared to $8.0 million for 2022.
  • Marketing and business development expenses for the year ended December 31, 2023, were $1.2 million, compared to $2.9 million for 2022.
  • PolyPid expects that its pro forma cash balance will be sufficient to fund operations into late third quarter 2024.

U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Augtyro™ (repotrectinib) for the Treatment of Patients with NTRK-Positive Locally Advanced or Metastatic Solid Tumors

Retrieved on: 
Wednesday, February 14, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, NSCLC, BMY, Bristol Myers Squibb, New Drug Application, Neoplasm, NTRK, ROS1, Food, File, Shanda, PDUFA, Therapy, Disease, Patient, Fusion, ALK, CARE, Priority review, Pharmaceutical industry

The filing acceptance is based on results from the registrational Phase 1/2 TRIDENT-1 trial (adult patients with NTRK-positive solid tumors) and CARE study (pediatric patients with NTRK-positive solid tumors).

Key Points: 
  • The filing acceptance is based on results from the registrational Phase 1/2 TRIDENT-1 trial (adult patients with NTRK-positive solid tumors) and CARE study (pediatric patients with NTRK-positive solid tumors).
  • The FDA granted the application Priority Review status and assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 15, 2024.
  • “While great advancements have been made over the last decade, patients with NTRK-positive locally advanced or metastatic solid tumors still experience significant unmet needs.
  • In the TRIDENT-1 study, Augtyro demonstrated clinically meaningful response rates in patients with NTRK-positive locally advanced or metastatic solid tumors.

Appili Therapeutics Reports Financial and Operational Results for Third Quarter of Fiscal Year 2024

Retrieved on: 
Tuesday, February 13, 2024
Metronidazole, Toxicology, New Drug Application, Research, Paromomycin, FDA, TSX, Drug development, Infection, Dysphagia, Sale, Skin, Government, USAFA, Indian stock exchange, Patent, Patient, Pharming, IND, Bank statement, CMC, Pharmaceutical industry

HALIFAX, Nova Scotia, Feb. 13, 2024 (GLOBE NEWSWIRE) -- Appili Therapeutics Inc. (TSX:APLI; OTCPink: APLIF) (the “Company” or “Appili”), a biopharmaceutical company focused on drug development for infectious diseases and biodefense products, today announced its financial and operational results for the third quarter of its fiscal year 2024, which ended on December 31, 2023. All figures are stated in Canadian dollars unless otherwise stated.

Key Points: 
  • In September 2023, Appili announced the FDA approval of LIKMEZ™ (ATI-1501) , our proprietary taste-masked liquid suspension reformulation of metronidazole, through our U.S. partner, Saptalis Pharmaceuticals LLC.
  • Collaborating with Saptalis, Appili earned US$600,000 in milestone payments in fiscal 2024.
  • Appili was granted a pre-IND meeting with the FDA to discuss ATI-1701’s regulatory, CMC, toxicology, and Phase 1 strategies and expects to receive feedback in early 2024.
  • As of February 13, 2024, the Company had 121,266,120 issued and outstanding Common Shares, 7,957,000 stock options, and 44,856,874 warrants outstanding.

Tonix Pharmaceuticals Announces Engagement of Rho as CRO to Support Submission of New Drug Application to the FDA for Approval of Tonmya™ for the Management of Fibromyalgia

Retrieved on: 
Tuesday, February 13, 2024
Fibromyalgia, Tonix Pharmaceuticals, New Drug Application, FDA, Food, Overalls, Hydrogen chloride, Fatigue, Tablet, NDA, CMC, Rho, Pharmaceutical industry

CHATHAM, N.J., Feb. 13, 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 it has engaged Rho, Inc. (Rho), a global contract research organization (CRO), to support Tonix’s preparation and planned submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of Tonmya™ (cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia.

Key Points: 
  • “We intend to meet with the FDA in the first half of this year and to submit our NDA in the second half of the year.
  • In the study, Tonmya met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia.
  • “We are delighted in our continued support of Tonix and their Tonmya program,” said Tara Gladwell, President and COO at Rho.
  • “We are hopeful that this work may result in a new treatment for the millions of Americans that suffer from this debilitating, chronic disorder.”

Sosei Heptares Operational Highlights and Consolidated Results for 12 Months ended 31 December 2023

Retrieved on: 
Tuesday, February 13, 2024
New Drug Application, GSK, Cancer Research UK, JPY, DSM-IV codes, IPK, Trial of the century, EP4, GPR35, GPR52, Genentech, Neurocrine Biosciences, Deficit spending, Therapy, IBD, G&A, Glutamate (neurotransmitter), Partnership, Pfizer, Cerebral vasospasm, Senior, GPCR, NAM, Acquisition, FDA, Inflammatory Bowel Diseases, Schizophrenia, Patient, Tokyo Stock Exchange, Medicine, IPJ, Insomnia, Movement, Pharmaceutical industry

Tokyo, Japan and Cambridge, UK, 13 February 2024 – Sosei Group Corporation (“Sosei Heptares” or “the Company”; TSE: 4565) provides an update on operational activities and reports its consolidated results for the 12 months ended 31 December 2023.

Key Points: 
  • Tokyo, Japan and Cambridge, UK, 13 February 2024 – Sosei Group Corporation (“Sosei Heptares” or “the Company”; TSE: 4565) provides an update on operational activities and reports its consolidated results for the 12 months ended 31 December 2023.
  • Chris Cargill, President & CEO of Sosei Heptares, commented: “2023 has been a transformational year for Sosei Group and the progress made by our teams across all areas of the business has been exceptional.
  • Marketing approval for PIVLAZ® in South Korea – for the prevention of cerebral vasospasm and related conditions after aneurysmal subarachnoid hemorrhage (“aSAH”) securing.
  • A Phase 1 study of NBI-1117569 has begun and a Phase 1 study of NBI-1117567 is expected to begin in 2024.

CymaBay Announces FDA Acceptance of NDA and Priority Review for Seladelpar for the Treatment of Primary Biliary Cholangitis

Retrieved on: 
Monday, February 12, 2024
Alkaline phosphatase, Liver, Child, Ursodeoxycholic acid, New Drug Application, BTD, Cirrhosis, Inflammation, FDA, Quality of life, Food, Breakthrough, Chief, Conditional sentence, PDUFA, ALP, PBC, Seladelpar, Therapy, Fatigue, Patient, Itch, Mortality, NDA, Pharmaceutical industry

NEWARK, Calif., Feb. 12, 2024 /PRNewswire/ -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for seladelpar, an investigational treatment for the management of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act (PDUFA) target action date of August 14, 2024 and notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • PBC is a rare, chronic condition that can lead to inflammation and eventually liver cirrhosis.
  • People living with PBC can also experience symptoms that significantly impact their quality of life such as pruritus (itch) and fatigue.
  • Seladelpar is an investigational, potent, selective, orally active PPARδ agonist, or delpar, in development for PBC treatment.
  • "We are encouraged by the agency's decision to grant priority review for seladelpar, and its recognition of the significant need for new treatment options for people living with PBC.

PolyPid Announces Enrollment of the 100th Patient in the Ongoing SHIELD II Phase 3 Trial

Retrieved on: 
Monday, February 12, 2024
SHIELD, Late, Infection, New Drug Application, Interim analysis, Completeness, Patient, PIPE, Pharmaceutical industry

PETACH TIKVA, Israel, Feb. 12, 2024 (GLOBE NEWSWIRE) -- PolyPid Ltd. (Nasdaq: PYPD) (“PolyPid” or the “Company”), a late-stage biopharma company aiming to improve surgical outcomes, today announced that it has enrolled and randomized the 100th patient in its ongoing SHIELD II Phase 3 trial for D-PLEX100 for the prevention of surgical site infections in patients undergoing abdominal colorectal surgery with large incisions. There are currently approximately 40 centers open.

Key Points: 
  • There are currently approximately 40 centers open.
  • The Company intends to conduct an unblinded interim analysis once a total of approximately 400 patients complete their 30-day follow-up, which is expected to occur in mid-2024.
  • “As anticipated, since last November, we have doubled the number of open centers, and enrollment into SHIELD II has recently begun to ramp-up,” said Dikla Czaczkes Akselbrad, PolyPid’s Chief Executive Officer.
  • The PIPE syndicate was comprised of new and existing investors, including participation from new U.S. life sciences-focused investors, DAFNA Capital Management and Rosalind Advisors.