European Medicines Agency

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

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星期三, 三月 6, 2024

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.

Kamada Issues 2024 CEO Letter to Shareholders

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星期三, 三月 6, 2024

The recently completed 2023 was another successful period in our commercial journey as a global leader in the specialty plasma-derived field.

Key Points: 
  • The recently completed 2023 was another successful period in our commercial journey as a global leader in the specialty plasma-derived field.
  • Looking ahead, we expect the momentum from 2023 to extend throughout 2024, with profitability to be further increased as compared to last year.
  • These significant catalysts are propelling our continued annual double-digit profitable growth with substantial upside potential and limited downside risk.
  • On behalf of the entire Kamada team, we look forward to continuing to support patients and clinicians with the important lifesaving products that we develop, manufacture, and commercialize.

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

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星期二, 三月 5, 2024

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.

CymaBay Announces European Medicines Agency Accepts for Review the Marketing Authorization Application for Seladelpar for the Treatment of Primary Biliary Cholangitis

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星期一, 三月 4, 2024

Seladelpar is an investigational, potent, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist or delpar, in development for PBC treatment.

Key Points: 
  • Seladelpar is an investigational, potent, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist or delpar, in development for PBC treatment.
  • The study’s primary endpoint measure reflects registrational studies of the current second line PBC treatment authorized by the EMA.
  • In this study seladelpar demonstrated statistically significant improvements in biochemical markers of disease progression and PBC-related pruritus (itch) pre-specified endpoints.
  • “Validation of the European Marketing Authorization Application for seladelpar and the start of the agency’s formal review is an important step forward in seladelpar’s journey to potential approval in Europe.

BridgeBio Pharma and Bayer Announce European Licensing Agreement for Acoramidis in ATTR-CM

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星期一, 三月 4, 2024

In exchange, BridgeBio will receive up to $310 million USD comprising of upfront and near-term milestone payments, as well as additional undisclosed sales milestones.

Key Points: 
  • In exchange, BridgeBio will receive up to $310 million USD comprising of upfront and near-term milestone payments, as well as additional undisclosed sales milestones.
  • BridgeBio will also receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in Europe.
  • “We are excited to have found a like-minded partner in Bayer that shares our belief in the potential of acoramidis to ameliorate the lives of ATTR-CM patients.
  • BridgeBio has also presented analyses from ATTRibute-CM at the European Society of Cardiology Congress 2023 and at the American Heart Association Scientific Sessions 2023.

89bio Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Updates

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星期四, 二月 29, 2024

SAN FRANCISCO, Feb. 29, 2024 (GLOBE NEWSWIRE) -- 89bio, Inc. (Nasdaq: ETNB), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for the treatment of liver and cardiometabolic diseases, today reported its financial results for the fourth quarter and full year ended December 31, 2023, and provided corporate updates.

Key Points: 
  • ENLIGHTEN-Fibrosis is expected to enroll non-cirrhotic patients with fibrosis stage F2-F3 and ENLIGHTEN-Cirrhosis is expected to enroll NASH patients with compensated cirrhosis (F4).
  • ENLIGHTEN-Fibrosis and ENLIGHTEN-Cirrhosis are expected to initiate in the first and second quarter of 2024, respectively.
  • Completed follow-on offering in the fourth quarter of 2023 for $172.5 million in gross proceeds.
  • The foregoing financial information is unaudited and subject to change, and actual results may vary from the foregoing.

SpringWorks Therapeutics Announces European Medicines Agency Validation for Marketing Authorization Application of Nirogacestat for the Treatment of Adults with Desmoid Tumors

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星期四, 二月 29, 2024

STAMFORD, Conn., Feb. 29, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors.

Key Points: 
  • STAMFORD, Conn., Feb. 29, 2024 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for nirogacestat, an oral gamma secretase inhibitor, for the treatment of adults with desmoid tumors.
  • If approved, nirogacestat will be the first therapy to receive marketing authorization in the European Union (EU) for the treatment of desmoid tumors.
  • Nirogacestat previously received Orphan Drug designation from the European Commission for the treatment of soft tissue sarcoma.
  • “The validation of our marketing authorization application is an important step towards bringing nirogacestat to patients with desmoid tumors in the European Union who currently do not have an approved therapy,” said Saqib Islam, Chief Executive Officer of SpringWorks.

GENFIT Announces Revenues and Cash Position as of December 31, 2023

Retrieved on: 
星期四, 二月 29, 2024

As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.

Key Points: 
  • As of December 31, 2023, the Company’s cash and cash equivalents amounted to €77.8 million compared with €140.2 million as of December 31, 2022.
  • As of September 30, 2023, cash and cash equivalents totaled €93.9 million.
  • Revenues for 2023 amounted to €28.6 million compared to €20.2 million for the same period in 2022.
  • The 2023 Universal Registration Document, the 2023 Annual Financial Report (included in the 2023 Universal Registration Document), and the Annual Report on Form 20-F will be published by the end of April 2024.

CymaBay Reports Fourth Quarter and Year Ended December 31, 2023 Financial Results and Provides Corporate Update

Retrieved on: 
星期三, 二月 28, 2024

NEWARK, Calif., Feb. 28, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, today announced corporate updates and financial results for the year and fourth quarter ended December 31, 2023.

Key Points: 
  • NEWARK, Calif., Feb. 28, 2024 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a clinical-stage biopharmaceutical company focused on developing therapies for liver and other chronic diseases with high unmet need, today announced corporate updates and financial results for the year and fourth quarter ended December 31, 2023.
  • “2023 was a seminal year for CymaBay with critical achievements in the development of our investigational therapeutic, seladelpar.
  • Net loss for the year ended December 31, 2023 and 2022 was $105.4 million and $106.0 million, or ($0.99) and ($1.21) per share, respectively.
  • Net loss for the three months ended December 31, 2023 was higher than the three months ended December 31, 2022 primarily due to higher operating expenses.

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

Retrieved on: 
星期三, 二月 28, 2024

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.