PRV

Akari Therapeutics Reports Full-Year 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, April 1, 2024
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BOSTON and LONDON, April 01, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the full year 2023 as well as recent company highlights.

Key Points: 
  • BOSTON and LONDON, April 01, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the full year 2023 as well as recent company highlights.
  • The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX.
  • Akari is continuing to recruit patients into the Part A portion of the Phase 3 clinical trial that has treated 10 patients to date.
  • During 2023, Akari also advanced the long-acting version of nomacopan (PASylated nomacopan) into the final stages of pre-clinical development as a treatment for geographic atrophy (GA).

Applied Therapeutics Provides FDA Update on PDUFA Target Action Date for Govorestat for the Treatment of Classic Galactosemia

Retrieved on: 
Thursday, March 28, 2024
Galactitol, MAA, New Drug Application, Patient, Ageing, NDA, PRV, EMA, Doctor of Philosophy, Marketing, Priority review, Therapy, ARI, Food, FDA, CHMP, PDUFA, Pharmaceutical industry

The FDA has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024.

Key Points: 
  • The FDA has set a new Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024.
  • In February 2024, the Company announced that the FDA accepted and granted Priority Review to the NDA.
  • “While the PDUFA action date extension represents a delay, we remain confident in the potential for govorestat approval for Galactosemia and we will continue to work closely with the FDA throughout the review process,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics.
  • If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product.

Orchard Therapeutics Receives FDA Approval of Lenmeldy™ (atidarsagene autotemcel), the Only Therapy for Eligible Children with Early-onset Metachromatic Leukodystrophy in the U.S.

Retrieved on: 
Monday, March 18, 2024
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“MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.

Key Points: 
  • “MLD is a rapidly progressing, life-limiting and ultimately fatal rare disease that has a devastating impact on afflicted children and their families.
  • This achievement is the culmination of decades of research and development in partnership with our academic and clinical collaborators at the San Raffaele-Telethon Institute for Gene Therapy.
  • It was previously given both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from FDA.
  • Orchard Therapeutics will provide more details about the launch of Lenmeldy in the U.S. through a separate announcement this week.

Aditxt Signs a Definitive Agreement to Acquire Appili Therapeutics Inc., Developer of a Biodefense Vaccine Funded by the U.S. Department of Defense (DoD)

Retrieved on: 
Tuesday, April 2, 2024
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The U.S. FDA's approval of LIKMEZ highlighted Appili’s capacity to identify and develop significant opportunities within the infectious disease domain.

Key Points: 
  • The U.S. FDA's approval of LIKMEZ highlighted Appili’s capacity to identify and develop significant opportunities within the infectious disease domain.
  • Appili licensed the manufacturing and commercialization rights in the U.S. and other selected territories to Saptalis Pharmaceuticals, LLC (“Saptalis”).
  • Appili, in collaboration with Saptalis, continued the product's development, ultimately achieving FDA approval in the United States.
  • With FDA approval, future revenue is expected to be derived from milestone payments and royalties from Saptalis under the license agreement.

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

Retrieved on: 
Wednesday, March 6, 2024
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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.

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

Retrieved on: 
Thursday, February 29, 2024
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Further, Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement.

Key Points: 
  • Further, Capricor will receive a meaningful mid-range double-digit revenue share of product revenue under the terms of this Agreement.
  • Next steps for Cohort A: plan to report top-line data in the fourth quarter of 2024.
  • Revenues for the fourth quarter of 2023 were approximately $12.1 million compared with approximately $1.0 million for the fourth quarter of 2022.
  • Operating expenses: Total operating expenses for the fourth quarter of 2023 were approximately $13.2 million compared with approximately $9.0 million for the fourth quarter of 2022.

Mesoblast Reports Financial Results and Operational Update for Half-Year Ended December 31, 2023

Retrieved on: 
Wednesday, February 28, 2024
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NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational update and reported financial results for the period ended December 31, 2023.

Key Points: 
  • NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational update and reported financial results for the period ended December 31, 2023.
  • Reduction in net cash usage for operating activities:
    For the three months ended December 31, 2023, net cash usage was US$12.3 million, a 25% reduction versus the comparative quarter in FY2023.
  • For the six months ended December 31, 2023, net cash usage was US$26.6 million, a 14% reduction versus the comparative period in FY2023.
  • Manufacturing reduced by 47% for the six months ended December 31, 2023, from US$12.8 million to US$6.7 million.

Applied Therapeutics Announces FDA Acceptance and Priority Review of New Drug Application for Govorestat for the Treatment of Classic Galactosemia

Retrieved on: 
Wednesday, February 28, 2024
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NEW YORK, Feb. 28, 2024 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of the New Drug Application (NDA) for govorestat (AT-007) for the treatment of Classic Galactosemia. The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024. 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.

Key Points: 
  • The NDA was granted Priority Review status, and the FDA assigned a Prescription Drug User Free Act (PDUFA) target action date of August 28, 2024.
  • Govorestat was previously granted Pediatric Rare Disease designation, and will qualify for a Priority Review Voucher (PRV) upon approval.
  • “The FDA’s acceptance of the NDA for govorestat for the treatment of Galactosemia represents a critical milestone for Applied Therapeutics and more importantly, for patients with Galactosemia and their families.
  • If approved, govorestat would be the first medication indicated for the treatment of Galactosemia and would be Applied Therapeutics’ first commercial product.

Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Recent Corporate Developments

Retrieved on: 
Wednesday, February 28, 2024
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Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023.
  • Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results.
  • Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability.
  • All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

Valneva Reports Full Year 2023 Revenue and Cash, Provides First 2024 Guidance

Retrieved on: 
Thursday, February 15, 2024
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The Company will publish its 2023 audited consolidated financial statements on March 20, 2024.

Key Points: 
  • The Company will publish its 2023 audited consolidated financial statements on March 20, 2024.
  • Peter Bühler, Valneva’s Chief Financial Officer, commented, “In 2023, Valneva successfully executed on key strategic objectives despite a difficult economic environment.
  • Total revenues in 2022 included €280.0 million of revenue recognition mainly related to the COVID-19 supply agreements in the prior year.
  • Other income is anticipated between €95 million and €105 million, including the PRV sold in early 2024 for €95 million.