PDUFA

Citius Pharmaceuticals, Inc. Reports Fiscal First Quarter 2024 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, February 14, 2024

CRANFORD, N.J., Feb. 14, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products today reported business and financial results for the fiscal first quarter 2024 ended December 31, 2023.

Key Points: 
  • "We had a strong first quarter of fiscal 2024 during which we achieved multiple mission critical goals.
  • FIRST QUARTER 2024 Financial Results:
    As of December 31, 2023, the Company had $20.3 million in cash and cash equivalents.
  • R&D expenses were $2.6 million for the first quarter ended December 31, 2023, compared to $3.4 million for the first quarter ended December 31, 2022.
  • G&A expenses were $3.7 million for the first quarter ended December 31, 2023, compared to $2.6 million for the first quarter ended December 31, 2022.

Citius Pharmaceuticals Resubmits the Biologics License Application of LYMPHIR™ (Denileukin Diftitox) for the Treatment of Adults with Relapsed or Refractory Cutaneous T-Cell Lymphoma

Retrieved on: 
Wednesday, February 14, 2024

CRANFORD, N.J., Feb. 13, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products today announced the resubmission of the Company's Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for LYMPHIR™ (denileukin diftitox), an IL-2-based immunotherapy for the treatment of patients with relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

Key Points: 
  • The resubmission follows ongoing engagement with the FDA resulting from a Complete Response Letter (CRL) received on July 28, 2023.
  • Citius believes it has addressed enhanced product testing and additional manufacturing controls noted in the letter.
  • There were no safety or efficacy issues cited and no additional trials required.
  • Based on Center for Drug Evaluation and Research timelines, FDA acceptance of the completed resubmission package and issuance of a Prescription Drug User Fee Act (PDUFA) date is expected within 30 days of resubmission.

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

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.

Basilea reports strong 2023 full-year results with profitability significantly above guidance, whilst substantially expanding the R&D portfolio

Retrieved on: 
Tuesday, February 13, 2024

53 LR

Key Points: 
  • 53 LR
    Basilea Pharmaceutica Ltd, Allschwil (SIX: BSLN), a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced today its results for the financial year ended December 31, 2023.
  • Our strong financial prospects are reflected in our guidance for 2024, which assumes continued significant top line and bottom line growth.
  • Basilea recorded an operating profit of CHF 19.2 million (FY 2022: CHF 18.5 million), which is 28% above guidance.
  • In 2023, a positive net cash flow of CHF 14.2 million was provided by operating activities, twice as much as in the previous year (FY 2022: CHF 7.1 million).

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

Retrieved on: 
Monday, February 12, 2024

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.

FDA Accepts Xspray Pharma’s NDA-resubmission for Dasynoc® – PDUFA Date set to 31 July 2024

Retrieved on: 
Monday, February 12, 2024

The FDA has now assigned a Prescription Drug User Fee Act (PDUFA) date to 31st of July, 2024.

Key Points: 
  • The FDA has now assigned a Prescription Drug User Fee Act (PDUFA) date to 31st of July, 2024.
  • This is the FDA’s deadline for completing the approval process, marking a significant milestone for Dasynoc®, Xspray’s innovative protein kinase inhibitor (PKI) product candidate for CML treatment.
  • With an established PDUFA date set to 31st of July, Xspray Pharma continues to strategically plan for the commercial launch of Dasynoc® on September 1, 2024.
  • As the PDUFA date approaches, the company remains focused on its goal to improve the lives of those affected by CML with this novel therapy.

Optimi Health Congratulates Lykos Therapeutics on FDA Priority Review of New Drug Application And Releases MDMA Production Video

Retrieved on: 
Monday, February 12, 2024

Additionally, Optimi is pleased to unveil a six-minute video showcasing its end-to-end GMP MDMA encapsulation and production process.

Key Points: 
  • Additionally, Optimi is pleased to unveil a six-minute video showcasing its end-to-end GMP MDMA encapsulation and production process.
  • The FDA's priority review designation underscores the significance of this potential breakthrough in providing effective treatment options for those impacted by this debilitating condition.
  • Bill Ciprick, CEO of Optimi Health, stated, "We commend Lykos Therapeutics and MAPS founder, Rick Doblin, for their unwavering dedication to advancing innovative therapies for PTSD.
  • Stevens stated, "The ability to showcase our GMP MDMA production process is a testament to our commitment to excellence and transparency.

MAPS Congratulates Lykos Therapeutics on Acceptance of New Drug Application for MDMA-Assisted Therapy for PTSD

Retrieved on: 
Saturday, February 10, 2024

"Thirty-eight years ago, one of my primary motivations for founding MAPS was to carve a path for psychedelic-assisted therapies to become FDA-approved treatments.

Key Points: 
  • "Thirty-eight years ago, one of my primary motivations for founding MAPS was to carve a path for psychedelic-assisted therapies to become FDA-approved treatments.
  • We hope that potential FDA approval of MDMA-assisted therapy for PTSD is only the first of many psychedelic-assisted therapies that become available by prescription."
  • Neither MDMA-assisted therapy for PTSD, nor any other psychedelic-assisted therapy, has been established for the treatment of any mental health condition.
  • The safety and efficacy of MDMA-assisted therapy have not been established for the treatment of PTSD.

Human Acellular Vessel™ (HAV™) Biologics License Application Granted Priority Review by U.S. FDA for the Treatment of Vascular Trauma

Retrieved on: 
Friday, February 9, 2024

DURHAM, N.C., Feb. 09, 2024 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review to Humacyte’s Biologics License Application (BLA) seeking approval of the Human Acellular Vessel (HAV) in urgent arterial repair following extremity vascular trauma when synthetic graft is not indicated, and when autologous vein use is not feasible.

Key Points: 
  • This targeted PDUFA date is based on the Priority Review grant, which is a mechanism reserved by FDA for products that, if approved, would significantly improve the treatment, diagnosis, or prevention of serious conditions.
  • Priority Review applications have a six-month review time instead of ten months for a standard review.
  • The Priority Review aligns with the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2023 for urgent arterial repair following extremity vascular trauma.
  • The HAV is an investigational product and has not been approved for sale by the FDA or any other regulatory agency.

Madrigal Pharmaceuticals Announces Publication of the Phase 3 MAESTRO-NASH Trial of Resmetirom in the New England Journal of Medicine

Retrieved on: 
Thursday, February 8, 2024

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis.
  • Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement.
  • More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.