Prescription Drug User Fee Act

Phathom Pharmaceuticals Reports Second Quarter 2023 Results

Retrieved on: 
Thursday, August 10, 2023

Second Quarter 2023 Financial Results:

Key Points: 
  • Second Quarter 2023 Financial Results:
    Net loss for the second quarter ended June 30, 2023, was $41.0 million, compared to $50.9 million for second quarter 2022.
  • Second quarter 2023 net loss included a non-cash charge related to stock-based compensation of $7.3 million compared to $5.9 million for second quarter 2022.
  • Research and development expenses for the second quarter 2023 were $12.8 million, a decrease of $6.0 million compared to $18.8 million for second quarter 2022.
  • General and administrative expenses for the second quarter 2023 were $18.9 million, a decrease of $7.6 million compared to $26.5 million for second quarter 2022.

Optinose Reports Second Quarter 2023 Financial Results and Operational Updates

Retrieved on: 
Thursday, August 10, 2023

YARDLEY, Pa., Aug. 10, 2023 (GLOBE NEWSWIRE) -- Optinose (NASDAQ:OPTN), a pharmaceutical company focused on patients treated by ear, nose and throat (ENT) and allergy specialists, today reported financial results for the quarter ended June 30, 2023, and provided operational updates.

Key Points: 
  • Conference call and webcast to be held today at 8:00 a.m. Eastern Time
    YARDLEY, Pa., Aug. 10, 2023 (GLOBE NEWSWIRE) -- Optinose (NASDAQ:OPTN), a pharmaceutical company focused on patients treated by ear, nose and throat (ENT) and allergy specialists, today reported financial results for the quarter ended June 30, 2023, and provided operational updates.
  • “We are pleased with the progress we made in the second quarter of 2023 towards our strategic objectives,” stated CEO Ramy Mahmoud, MD, MPH.
  • “That means we are outperforming our initial expectations for full year 2023 XHANCE net revenues while operating far more efficiently.
  • Members of the Company’s leadership team will host a conference call and presentation to discuss financial results and corporate updates beginning at 8:00 a.m. Eastern Time today.

CorMedix Inc. Reports Second Quarter and Six Month 2023 Financial Results and Provides Business Update

Retrieved on: 
Tuesday, August 8, 2023

CorMedix completed an equity offering in July 2023 which generated net proceeds of approximately $43.2 million.

Key Points: 
  • CorMedix completed an equity offering in July 2023 which generated net proceeds of approximately $43.2 million.
  • Operating expenses in the second quarter of 2023 increased approximately 43% to $11.8 million, compared with $8.3 million in the second quarter of 2022.
  • In addition, CorMedix received approximately $43.2 million in net proceeds from its equity financing that closed in July of 2023.
  • The management team of CorMedix will host a conference call and webcast today, August 8, 2023, at 8:30 AM Eastern Time, to discuss recent corporate developments and financial results.

bluebird bio Reports Second Quarter 2023 Financial Results and Highlights Operational Progress

Retrieved on: 
Tuesday, August 8, 2023

bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today reported financial results and business highlights for the second quarter ended June 30, 2023, including recent commercial and operational progress, and regulatory updates.

Key Points: 
  • bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “Company”) today reported financial results and business highlights for the second quarter ended June 30, 2023, including recent commercial and operational progress, and regulatory updates.
  • bluebird is pursuing FDA approval for lovo-cel for patients ages 12 and older who have a history of vaso-occlusive events (VOEs).
  • Excluding $45 million of restricted cash, which is currently unavailable for use, bluebird estimates cash runway into the second quarter of 2024.
  • Please see our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023 for further information regarding our cash runway guidance and other financial results.

CRISPR Therapeutics Provides Business Update and Reports Second Quarter 2023 Financial Results

Retrieved on: 
Monday, August 7, 2023

R&D Expenses: R&D expenses were $101.6 million for the second quarter of 2023, compared to $123.2 million for the second quarter of 2022.

Key Points: 
  • R&D Expenses: R&D expenses were $101.6 million for the second quarter of 2023, compared to $123.2 million for the second quarter of 2022.
  • G&A Expenses: General and administrative expenses were $19.0 million for the second quarter of 2023, compared to $26.3 million for the second quarter of 2022.
  • Collaboration Expense: Collaboration expense, net, was $44.6 million for the second quarter of 2023, compared to $33.9 million for the second quarter of 2022.
  • Net Loss: Net loss was $77.7 million for the second quarter of 2023, compared to a net loss of $185.8 million for the second quarter of 2022.

Mirum Pharmaceuticals Reports Second Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, August 3, 2023

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for second quarter 2023 and provided a business update.

Key Points: 
  • Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for second quarter 2023 and provided a business update.
  • Mirum to pay $210 million upfront and up to $235 million in potential tiered annual net sales-based milestones.
  • Second quarter 2023 global net sales grew 86% as compared to second quarter 2022, on track for 50% US net product sales growth in 2023 as compared to 2022.
  • Total operating expenses were $61.8 million for the quarter ended June 30, 2023 in comparison to $48.9 million for the quarter ended June 30, 2022.

SpringWorks Therapeutics Reports Second Quarter 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Wednesday, August 2, 2023

STAMFORD, Conn., Aug. 02, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported second quarter financial results for the period ended June 30, 2023 and provided an update on recent company developments.

Key Points: 
  • STAMFORD, Conn., Aug. 02, 2023 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical company focused on developing life-changing medicines for patients with severe rare diseases and cancer, today reported second quarter financial results for the period ended June 30, 2023 and provided an update on recent company developments.
  • In May 2023, SpringWorks announced full enrollment of the Phase 2 trial evaluating nirogacestat as a monotherapy in patients with recurrent ovarian granulosa cell tumors.
  • SpringWorks expects to file an Investigational New Drug Application for SW-682 in the fourth quarter of 2023.
  • Net Loss Attributable to Common Stockholders: SpringWorks reported net loss of $77.9 million, or $1.25 per share, for the second quarter of 2023.

Jemperli (dostarlimab-gxly) plus chemotherapy approved in the US as the first new frontline treatment option in decades for dMMR/MSI-H primary advanced or recurrent endometrial cancer

Retrieved on: 
Monday, July 31, 2023

Hesham Abdullah, Senior Vice President, Global Head of Oncology Development, GSK, said: “Today’s expanded approval of Jemperli redefines the treatment landscape for patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer.

Key Points: 
  • Hesham Abdullah, Senior Vice President, Global Head of Oncology Development, GSK, said: “Today’s expanded approval of Jemperli redefines the treatment landscape for patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer.
  • Until now, chemotherapy alone has been the standard of care with many patients experiencing disease progression.
  • These results and today’s approval underscore our belief in the potential for Jemperli to transform cancer treatment as a backbone immuno-oncology therapy.”
    With this approval, Jemperli is now indicated earlier in treatment in combination with chemotherapy for patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer.
  • The most common treatment-emergent adverse events (≥ 20%) in patients receiving Jemperli plus chemotherapy were rash, diarrhea, hypothyroidism and hypertension.

Apellis Pharmaceuticals Reports Second Quarter 2023 Financial Results

Retrieved on: 
Monday, July 31, 2023

EMPAVELI for the treatment of PNH:

Key Points: 
  • EMPAVELI for the treatment of PNH:
    Apellis reported $22.3 million in EMPAVELI U.S. net product revenue for the second quarter 2023.
  • Cost of sales were $8.4 million for the second quarter 2023, compared to $0.1 million for same period in 2022.
  • Apellis reported a net loss of $122.0 million for the second quarter 2023, compared to a net loss of $156.0 million for the same period in 2022.
  • Apellis will host a conference call and webcast to discuss its second quarter 2023 financial results and business highlights today, July 31, 2023, at 8:30 a.m.

U.S. FDA Accepts Biologics License Application for GC Biopharma's GC5107B (Immune Globulin Intravenous (Human), 10% Liquid)

Retrieved on: 
Monday, July 31, 2023

YONGIN, South Korea, July 31, 2023 /PRNewswire/ -- GC Biopharma (006280.KS), a global biopharmaceutical company dedicated to specialty plasma-derived therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's resubmission of the Biologics License Application (BLA) for its GC5107B (Immune Globulin Intravenous (Human) for patients with primary humoral immunodeficiency (PI).

Key Points: 
  • YONGIN, South Korea, July 31, 2023 /PRNewswire/ -- GC Biopharma (006280.KS), a global biopharmaceutical company dedicated to specialty plasma-derived therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's resubmission of the Biologics License Application (BLA) for its GC5107B (Immune Globulin Intravenous (Human) for patients with primary humoral immunodeficiency (PI).
  • The Prescription Drug User Fee Act (PDUFA) target action date is January 13, 2024.
  • If approved, GC Biopharma would be able to provide more treatment options for patients with PI in the U.S.
    GC Biopharma aims to enter the U.S. market in the second half of 2024 if GC5107B is approved by the FDA as anticipated.
  • While the U.S. immunoglobulin market size is estimated at about US$ 10.4 billion in 2022 (as per MRB[1] 2022), there have been sporadic shortages as there are only a small number of immunoglobulin manufacturers considering the need for a massive investment in facility and sophisticated production know-hows.