Transfusion-dependent anemia

Geron Corporation Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, February 28, 2024

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing investigational first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business highlights and financial results for the fourth quarter and full year 2023.

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing investigational first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business highlights and financial results for the fourth quarter and full year 2023.
  • The FDA assigned a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024 to the NDA.
  • In September 2023, the European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for imetelstat in the same proposed indication as the NDA.
  • Geron will host a conference call at 8:00 am ET on Wednesday, February 28, 2024, to discuss business updates and fourth quarter and full year 2023 financial results.

Bristol Myers Squibb Receives Positive CHMP Opinion for Reblozyl® (luspatercept) for Treatment of Adults with Transfusion-Dependent Anemia due to Low- to Intermediate-Risk Myelodysplastic Syndromes (MDS)

Retrieved on: 
Friday, February 23, 2024

The recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU).

Key Points: 
  • The recommendation will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU).
  • Upon approval, this would represent the fourth authorized indication for Reblozyl in the EU.
  • The CHMP adopted a positive opinion based on results from the pivotal Phase 3 COMMANDS trial.
  • Safety results were consistent with previous MDS studies and were in line with expected symptoms in this patient population.

Long-Term Data Presented at ASH Support beti-cel as a Potentially Curative Gene Therapy for β-Thalassemia Patients Who Require Regular Transfusions Through Achievement of Durable Transfusion Independence and Normal or Near-Normal Adult Hb Levels

Retrieved on: 
Sunday, December 10, 2023

As of the data cutoff date, TI was maintained through last follow-up (up to 6 years) across ages and genotypes.

Key Points: 
  • As of the data cutoff date, TI was maintained through last follow-up (up to 6 years) across ages and genotypes.
  • Among patients in the Phase 1/2 studies, 68.2% (15/22) achieved TI, with 14 patients sustaining TI through the last follow-up (up to 9 years).
  • The patient continued to benefit from beti-cell therapy and was not receiving chronic red blood cells transfusions as of the last follow-up.
  • The most common beti-cel-related AEs (occurring in ≥3 patients) were abdominal pain (7.9% of patients) and thrombocytopenia (4.8% of patients).

Geron Corporation Reports Business Highlights and Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business highlights and financial results for the third quarter of 2023.

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business highlights and financial results for the third quarter of 2023.
  • Ms. Robertson brings to Geron over 30 years of financial and commercial operations experience.
  • In the third quarter of 2023, the Company received $28.3 million upon the cash exercise of outstanding warrants.
  • Geron will host a conference call at 9:00 am ET on Thursday, November 2, 2023 to discuss business updates and third quarter 2023 financial results.

Geron IMerge Phase 3 Presentations at Upcoming ASH Annual Meeting Reinforce Significant Durability and Breadth of Effect of Imetelstat in Lower Risk MDS

Retrieved on: 
Thursday, November 2, 2023

Four abstracts have been accepted for presentation at the 65th American Society of Hematology (ASH) Annual Meeting taking place from December 9-12 in San Diego, California and virtually.

Key Points: 
  • Four abstracts have been accepted for presentation at the 65th American Society of Hematology (ASH) Annual Meeting taking place from December 9-12 in San Diego, California and virtually.
  • “Additionally, our abstract showcasing a real-world data population level analysis of over 5,000 lower risk MDS patients adds to the significant literature suggesting a correlation between TI and improvement in survival.
  • This analysis suggests imetelstat has clinical activity in lower risk MDS patients independent of risk categories.
  • Achievement of durable TI was associated with improved survival, supporting the clinical benefit of achieving transfusion independence in lower risk MDS.

Geron Announces EMA Validation of Marketing Authorization Application for Imetelstat for the Treatment of Lower Risk MDS

Retrieved on: 
Friday, September 29, 2023

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for imetelstat, a first-in-class investigational telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for imetelstat, a first-in-class investigational telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).
  • “The EMA validation of the marketing authorization application for imetelstat brings us one step closer to potentially offering this first-in-class therapeutic to lower risk MDS patients in the EU suffering from anemia,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer.
  • Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category.
  • Consistent with prior imetelstat clinical experience, the most common serious adverse events were primarily short-lived, manageable cytopenias.

Geron Announces PDUFA Date for Imetelstat NDA in Lower Risk MDS

Retrieved on: 
Tuesday, August 22, 2023

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has assigned a standard review and a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024 for Geron’s New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has assigned a standard review and a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024 for Geron’s New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).
  • In addition, the FDA informed the Company that it is currently planning to hold an advisory committee meeting as part of the NDA review.
  • “We look forward to working with the FDA to complete the NDA review process on a timely basis,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer.
  • “With the PDUFA date now set, we continue to expect an imetelstat launch in the United States by the end of the first half of 2024, subject to FDA approval.”

Geron Announces FDA Acceptance of New Drug Application for Imetelstat for the Treatment of Lower Risk MDS

Retrieved on: 
Monday, August 21, 2023

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has accepted the filing of Geron’s New Drug Application (NDA) for imetelstat, its first-in-class telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has accepted the filing of Geron’s New Drug Application (NDA) for imetelstat, its first-in-class telomerase inhibitor, for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS).
  • “The FDA’s acceptance of our New Drug Application is an important landmark along our steadfast journey to bring telomerase inhibition with imetelstat to the market,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer.
  • Statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups irrespective of ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category.
  • Additionally, Geron expects to submit a Marketing Authorization Application (MAA) in the European Union (EU) in the fourth quarter of 2023.

Roivant Reports Financial Results for the First Quarter Ended June 30, 2023, and Provides Business Update

Retrieved on: 
Monday, August 14, 2023

Dermavant: For the first quarter ended June 30, 2023, Roivant reported VTAMA net product revenue of $16.7M, representing a 26% gross-to-net yield for the quarter.

Key Points: 
  • Dermavant: For the first quarter ended June 30, 2023, Roivant reported VTAMA net product revenue of $16.7M, representing a 26% gross-to-net yield for the quarter.
  • First Quarter Ended June 30, 2023, Financial Summary
    As of June 30, 2023, the company had consolidated cash, cash equivalents and restricted cash of $1.4 billion.
  • Net loss was $327.8 million for the three months ended June 30, 2023, compared to $353.8 million for the three months ended June 30, 2022.
  • Non-GAAP net loss was $211.5 million for the three months ended June 30, 2023, compared to $210.7 million for the three months ended June 30, 2022.

Geron Announces Submission of New Drug Application to FDA for First-in-Class Telomerase Inhibitor Imetelstat

Retrieved on: 
Tuesday, June 20, 2023

“This pioneering achievement to submit the first New Drug Application to the FDA for a telomerase inhibitor reflects the dedication, commitment and teamwork of so many people who believed targeting telomerase could make a significant difference for patients,” said John A. Scarlett, M.D., Chairman and Chief Executive Officer.

Key Points: 
  • “This pioneering achievement to submit the first New Drug Application to the FDA for a telomerase inhibitor reflects the dedication, commitment and teamwork of so many people who believed targeting telomerase could make a significant difference for patients,” said John A. Scarlett, M.D., Chairman and Chief Executive Officer.
  • As allowed under imetelstat’s Fast Track designation in lower risk MDS, Geron has requested that the FDA grant Priority Review of the NDA.
  • Under standard practice, Geron expects FDA communication in 60 days whether the NDA was accepted for review and the timeline of such review (i.e., priority or standard).
  • Additionally, based on IMerge Phase 3, Geron expects to submit a Marketing Authorization Application (MAA) in the EU in the second half of 2023.