Diamond–Blackfan anemia

Feinstein Institutes Secures $6.1M NIH Grant for Red Cell Disorder Research

Retrieved on: 
Wednesday, April 3, 2024
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To further our understanding and potential treatments of these disorders, The Feinstein Institutes for Medical Research has been awarded a $6.1 million grant from the National Institutes of Health (NIH).

Key Points: 
  • To further our understanding and potential treatments of these disorders, The Feinstein Institutes for Medical Research has been awarded a $6.1 million grant from the National Institutes of Health (NIH).
  • View the full release here: https://www.businesswire.com/news/home/20240403646471/en/
    Dr. Lionel Blanc will lead the new NIH-funded red cell disorders research (Credit: Feinstein Institutes)
    Led by Lionel Blanc, PhD , professor in the Institute of Molecular Medicine at the Feinstein Institutes, the seven-year initiative aims to build off previous research and will shed light on DBA, SCD and anemia.
  • “Understanding red cell blood disorders, how they develop and progress, is essential given their global impact on millions,” said Dr. Blanc.
  • In 2019, he received a $2.5 million grant from the NIH to study treatment for erythropoietic disorders, including DBA.

Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Second Quarter 2023

Retrieved on: 
Thursday, August 3, 2023
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Fulcrum expects to complete enrollment in the third quarter of 2023 and expects to report topline data in the fourth quarter of 2024.

Key Points: 
  • Fulcrum expects to complete enrollment in the third quarter of 2023 and expects to report topline data in the fourth quarter of 2024.
  • Collaboration Revenue: Collaboration revenue was $0.9 million for the second quarter of 2023 as compared to $1.9 million for the second quarter of 2022.
  • Net Loss: Net loss was $23.8 million for the second quarter of 2023 as compared to $34.1 million for the second quarter of 2022.
  • ET to review the second quarter and 2023 recent business highlights and financial results.

Disc Medicine Announces First Patient Enrolled in Phase 1/2 Clinical Trial of Bitopertin in Diamond-Blackfan Anemia (DBA)

Retrieved on: 
Thursday, July 27, 2023
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Bitopertin is an investigational, orally administered glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis.

Key Points: 
  • Bitopertin is an investigational, orally administered glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis.
  • “The start of this trial represents another step toward demonstrating the potential of bitopertin to address a wide range of hematologic conditions.” said Will Savage, M.D., Ph.D., Chief Medical Officer of Disc.
  • The study includes planned dose escalation within each participant to continually assess for hematologic response.
  • Upon completion of the main treatment period, patients may continue on extended treatment within the trial.

Fulcrum Therapeutics Signs Exclusive Global License Agreement in Rare Hematology

Retrieved on: 
Monday, July 10, 2023
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DBA is a congenital, rare blood disorder that affects an estimated 5,000 individuals worldwide.

Key Points: 
  • DBA is a congenital, rare blood disorder that affects an estimated 5,000 individuals worldwide.
  • DBA is caused by genetic mutations in ribosomal subunits that halt red blood cell maturation and lead to anemia.
  • Fulcrum will assume sole responsibility for research, development, manufacturing and commercialization costs and activities, and will pay tiered royalties on future commercial sales.
  • “By uniting our scientific insights with Fulcrum’s deep expertise in hematology and small molecule development, this agreement will help accelerate the discovery and delivery of ground-breaking therapies for this underserved community.”

Disc Medicine Reports First Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Monday, May 15, 2023
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Interim data will be presented at EHA Congress on June 9, 2023, with an accompanying management call at 7:30 am ET the same day.

Key Points: 
  • Interim data will be presented at EHA Congress on June 9, 2023, with an accompanying management call at 7:30 am ET the same day.
  • Announced a collaboration with NIH to study bitopertin in patients with Diamond-Blackfan Anemia in March 2023; the study is expected to initiate mid-year 2023.
  • First Quarter 2023 Financial Results:
    Cash Position: Cash and cash equivalents were $236.4 million as of March 31, 2023 compared to $194.6 million as of December 31, 2022.
  • Net Loss: The net loss was $22.8 million for the first quarter of 2023, as compared to $9.9 million for the first quarter of 2022.

Disc Medicine Reports Full Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Friday, March 31, 2023
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In August 2022, Disc initiated BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP).

Key Points: 
  • In August 2022, Disc initiated BEACON, an open-label Phase 2 clinical study of bitopertin in patients with EPP and X-linked protoporphyria (XLP).
  • In October 2022, Disc initiated AURORA, a Phase 2 randomized, placebo-controlled clinical study of bitopertin in adults with EPP.
  • Disc completed a reverse merger with Gemini Therapeutics in December 2022, which resulted in Disc becoming publicly listed on NASDAQ, and raised approximately $90 million in operating capital.
  • Full Year 2022 Financial Results:
    Cash Position: Cash and cash equivalents were $194.6 million as of December 31, 2022 compared to $88.0 million as of December 31, 2021.

Disc Medicine Announces Collaboration with National Institutes of Health for Phase 2 Clinical Study of Bitopertin in Patients with Diamond-Blackfan Anemia (DBA)

Retrieved on: 
Tuesday, March 21, 2023
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The study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA) and is expected to initiate mid-year 2023.

Key Points: 
  • The study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA) and is expected to initiate mid-year 2023.
  • “Disc now has ongoing development programs of bitopertin in both erythropoietic porphyria and DBA.
  • The phase 2 study will be a pilot, single-arm, dose-escalation trial of bitopertin in DBA patients who either have steroid-refractory and/or relapsed disease or are unable to tolerate systemic corticosteroids.
  • Upon completion of the main treatment period, patients may continue on extended treatment within the trial.

Disc Medicine Announces $62.5 Million Financing led by Bain Capital Life Sciences to Advance Portfolio of Novel Hematology Programs

Retrieved on: 
Tuesday, February 14, 2023
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The financing was led by Bain Capital Life Sciences, who contributed $50 million, with participation from existing investors, Access Biotechnology and OrbiMed.

Key Points: 
  • The financing was led by Bain Capital Life Sciences, who contributed $50 million, with participation from existing investors, Access Biotechnology and OrbiMed.
  • The offering is expected to close on or about February 15, 2023, subject to customary closing conditions.
  • The shares of common stock and pre-funded warrants were offered pursuant to a shelf registration statement on Form S-3 (File No.
  • “We are delighted to welcome Bain Capital Life Sciences as an investor during a transformational time for Disc.

Jubilant Therapeutics Inc. receives Orphan Drug Designation for JBI-802 for Acute Myeloid Leukemia (AML) and Small Cell Lung Cancer (SCLC)

Retrieved on: 
Thursday, January 5, 2023
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BEDMINSTER, N.J., Jan. 5, 2023 /PRNewswire/ -- Jubilant Therapeutics Inc ., a clinical stage biopharmaceutical company advancing small molecule precision therapeutics to address unmet medical needs in oncology and autoimmune diseases, today announced that the United States Food and Drug Administration (US FDA) has granted Orphan Drug Designation for JBI-802 for the treatment of small cell lung cancer (SCLC) and acute myeloid leukemia (AML).

Key Points: 
  • BEDMINSTER, N.J., Jan. 5, 2023 /PRNewswire/ -- Jubilant Therapeutics Inc ., a clinical stage biopharmaceutical company advancing small molecule precision therapeutics to address unmet medical needs in oncology and autoimmune diseases, today announced that the United States Food and Drug Administration (US FDA) has granted Orphan Drug Designation for JBI-802 for the treatment of small cell lung cancer (SCLC) and acute myeloid leukemia (AML).
  • This unique profile has shown synergistic anti-tumor activity and it is expected to overcome tolerability limitations of first-generation, single target epigenetic modulators.
  • "JBI-802 is the lead product candidate from our TIBEO (Therapeutic Index and Brain Exposure Optimization) Discovery Engine.
  • It is our unique approach of structure-based drug design to generate novel pharmacophores with improved target product profile compared to existing agents.

Portfolio Company Disc Medicine Enters Definitive Merger Agreement with Gemini Therapeutics

Retrieved on: 
Wednesday, August 10, 2022
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Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.

Key Points: 
  • Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.
  • Disc is building a portfolio of innovative, first-in-class therapeutic candidates that affect fundamental pathways of red blood cell biology.
  • Disc Medicine is committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic disease.
  • Disc has a clinical-stage development pipeline composed of investigational product candidates that affect heme biosynthesis and iron metabolism.