News & Events

Disc Medicine Presents Positive Results from Phase 1 Clinical Study of DISC-0974 in Healthy Volunteers at the 2022 EHA Annual Congress

First-in-human study of DISC-0974 establishes inhibition of hemojuvelin (HJV) co-receptor as a novel approach to target hepcidin, the master regulator of iron homeostasis

Data demonstrate robust increases in serum iron and improvements in markers of erythropoiesis including statistically significant increases of hemoglobin at the highest dose

Disc plans to initiate separate clinical studies in patients with myelofibrosis and in patients with non-dialysis dependent chronic kidney disease mid-2022

WATERTOWN, Mass. (June 10, 2022) – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates for serious and debilitating hematologic diseases, today presented the results from its phase 1 clinical study of DISC-0974, a first-in-class, monoclonal antibody designed to suppress hepcidin production by inhibiting hemojuvelin (HJV), at the European Hematology Association (EHA) Congress being held in Vienna, Austria. The study data demonstrated robust increases in serum iron and markers of erythropoiesis, decreases in hepcidin levels, and that DISC-0974 was well-tolerated in healthy volunteers, consistent with the mechanism of action.

“We are very excited by the robust activity and encouraging initial safety profile of DISC-0974 observed in this study and which provide strong validation for our approach to targeting the hepcidin axis,” said John Quisel, J.D., Ph.D., Chief Executive Officer. “Having established clinical proof-of-mechanism, we intend to develop DISC-0974 across a wide range of anemias caused by elevated hepcidin, beginning with studies in patients with myelofibrosis and in patients with non-dialysis dependent chronic kidney disease, both of which we plan to initiate this year.”

The phase 1 study was a double-blind, placebo-controlled, single-ascending trial in healthy volunteers to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974. In the study, 34 healthy, adult subjects received either a single dose of DISC-0974 at escalating dose levels (7 mg IV, 14 mg SC, 28 mg SC, or 56 mg SC) or placebo. The study included endpoints to assess pharmacodynamic activity including serum hepcidin, serum iron and transferring saturation, ferritin levels, and measures of erythropoiesis.

Key data presented:

Rapid, dose-dependent and sustained decrease in serum hepcidin and a corresponding, robust increase in measures of circulating iron, including more than a doubling of transferrin saturation from baseline at the highest dose level
Changes in serum iron also corresponded with markers of iron mobilization and erythropoiesis, including decreased ferritin levels, increased reticulocyte hemoglobin, and increased mean corpuscular hemoglobin
At the 56 mg SC dose level, a single administration of DISC-0974 resulted in a statistically significant improvement in hemoglobin compared to placebo (+1.1 g/dL, p=0.009) at Day 42 and a marked increase in red blood cell count
DISC-0974 was well-tolerated at all dose levels with no serious or severe adverse events, no adverse events leading to study withdrawal, and no adverse event greater than Grade 1
Plasma exposure was dose-related in the 14 to 56 mg SC range and effects were observed through 28 days post-dose, indicating a sustained and clinically meaningful duration of action

“Hepcidin has long been known as the master regulatory hormone of systemic iron homeostasis. However, despite its clear biological importance and role in driving anemia across a host of diseases, no agents that primarily target excess hepcidin have been successfully developed,” said Elizabeta Nemeth, Ph.D., Professor of Medicine at the David Geffen School of Medicine at UCLA, and Director of the UCLA Center for Iron Disorders. “The dramatic effects of DISC-0974 on iron and erythropoiesis in this study of healthy volunteers are encouraging and I look forward to following its progress in future clinical trials.”

These data were presented at the European Hematology Association Annual Congress in Vienna and the poster is available on the EHA Congress platform at www.ehaweb.org.

About DISC-0974

DISC-0974 is an investigational, first-in-class monoclonal antibody designed to suppress hepcidin production by inhibiting the hemojuvelin (HJV) co-receptor, a highly selective and critical target of the hepcidin pathway with biological activity that has been validated by human genetic evidence. Hepcidin is the primary regulatory hormone of iron homeostasis and plays a central role by restricting iron absorption and preventing deployment from internal iron stores. DISC-0974 is currently being studied in a phase 1 clinical study of healthy volunteers and is being developed as a potential treatment for anemia of inflammation by suppressing hepcidin and enhancing iron availability for erythropoiesis.

DISC-0974 is an investigational therapy that is not approved for any use in any country. Disc obtained global rights to DISC-0974 and related molecules under a license agreement from AbbVie in October 2019.

About Disc Medicine

Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders. We are 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. For more information, please visit www.discmedicine.com.

More News

Disc Medicine Announces Multiple Presentations Across Hematology Portfolio at the 64th American Society of Hematology Annual Meeting

CAMBRIDGE, Mass. (November 3, 2022) – Disc Medicine, a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments...

Disc Medicine Initiates AURORA, a Phase 2 Clinical Study of Bitopertin in Adults with Erythropoietic Protoporphyria (EPP)

WATERTOWN, Mass. (October 31, 2022) – Disc Medicine, a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel treatments...

Disc Medicine Initiates BEACON, a Phase 2 Clinical Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)

WATERTOWN, Mass. (August 10, 2022) – Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapeutic candidates...

Gemini Therapeutics and Disc Medicine Announce Merger Agreement

WAYLAND, Mass. and WATERTOWN, Mass. (August 10, 2022) – Gemini Therapeutics, Inc. (Nasdaq: GMTX) (“Gemini”) and Disc Medicine, Inc. (“Disc”), a privately-held,...

Disc Medicine Initiates Phase 1b/2 Clinical Study of DISC-0974 in Myelofibrosis Patients with Severe Anemia

CAMBRIDGE, Mass. (June 23, 2022) – Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapeutic candidates...

Disc Medicine Announces Presentation of Clinical Data from First-in-Human Phase 1 Study of DISC-0974 at European Hematology Association (EHA) 2022 Congress

WATERTOWN, Mass. (May 16, 2022) – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates for...

Disc Medicine Appoints Jay T. Backstrom, M.D., M.P.H., to its Board of Directors and Rahul Khara, Pharm.D., J.D., as General Counsel

CAMBRIDGE, Mass. (January 4, 2022) - Disc Medicine, a clinical-stage biopharmaceutical company focused on the discovery and development of novel treatments for...

Disc Medicine Announces Oral Presentation on Bitopertin at the 63rd American Society of Hematology Annual Meeting

CAMBRIDGE, Mass. (November 11, 2021) – Disc Medicine, a clinical-stage biopharmaceutical company focused on the discovery and development of novel treatments for...

Disc Medicine Announces $90 Million Series B Financing to Advance Portfolio of Clinical-Stage Therapies for Hematologic Diseases

CAMBRIDGE, Mass. (September 2, 2021) – Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapies for...

Disc Medicine Appoints Will Savage, MD, PhD as Chief Medical Officer and Jonathan Yu as Chief Business Officer and Expands Scientific Leadership with Key Hires to Advance Clinical-Stage Pipeline

CAMBRIDGE, Mass. (August 23, 2021) – Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapies for...

Disc Medicine Initiates Phase 1 Clinical Study of DISC-0974, a First-In-Class Inhibitor of Hemojuvelin (HJV) in Development for the Treatment of Anemia of Inflammation

CAMBRIDGE, Mass. (July 28, 2021) – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapies for the...

Disc Medicine Expands Hematology Pipeline with Worldwide Licensing Agreement for Bitopertin, a First-in-Class Modulator of Heme Synthesis

CAMBRIDGE, Mass. (May 27, 2021) – Disc Medicine, Inc. announced today that it has entered into an exclusive worldwide licensing agreement with...

Disc Medicine to Present at the European Hematology Association (EHA) 2021 Virtual Congress

CAMBRIDGE, Mass. (May 24, 2021) – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates for...

Disc Medicine to Present at the Jefferies Virtual Healthcare Conference

CAMBRIDGE, Mass. (May 21, 2021) – Disc Medicine, a company dedicated to the discovery and development of novel therapeutic candidates for serious...

Disc Medicine Appoints William White to its Board of Directors

CAMBRIDGE, Mass. (January 13th, 2021) – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates for...

Disc Medicine to Present at the Virtual 62nd American Society of Hematology Annual Meeting

CAMBRIDGE, Mass. (December 2, 2020) – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates for...

Disc Medicine to Present at the Piper Sandler 32nd Annual Healthcare Conference

CAMBRIDGE, Mass. (November 23, 2020) – Disc Medicine, a hematology company dedicated to the discovery and development of novel therapeutic candidates for...

Disc Medicine Expands Scientific Advisory Board with Leading Experts in Hepcidin Biology

Tomas Ganz, MD, PhD and Elizabeta Nemeth, PhD to Join Disc Medicine Scientific Advisory Board Cambridge, Mass. – October 13, 2020 –...

Disc Medicine to Present at Citi's 15th Annual BioPharma Virtual Conference

Cambridge, Mass. – September 8, 2020 – Disc Medicine, a biotechnology company dedicated to the discovery and development of novel therapeutic candidates...

Disc Medicine Expands Leadership Team with Industry Veterans to Advance Hepcidin Modulating Therapies Toward the Clinic

Jonathan Yu Named Senior Vice President of Corporate Strategy William Savage, MD, PhD Appointed Vice President of Clinical Development

John Quisel, JD, PhD Joins Disc Medicine as President and Chief Executive Officer

Cambridge, Mass. – January 28, 2020 – Disc Medicine, a hematology company applying new insights in hepcidin biology to develop therapies that...

Disc Medicine Completes $50 Million Series A Financing led by Novo Holdings A/S to Advance New Therapies Addressing Ineffective Red Blood Cell Production

Disc Medicine, a hematology company applying new insights in hepcidin biology to develop therapies that restore red blood cell production in hematologic...

Disc Medicine Expands Pipeline Focused on Hepcidin Pathway

Enters into exclusive agreement with AbbVie for series of Hemojuvelin Antagonist Monoclonal Antibodies Cambridge, Mass. – October 29, 2019 – Disc Medicine,...

Introducing Disc Medicine, a New Hematology Company

Introducing Disc Medicine – we are a hematology company harnessing new insights in hepcidin biology to address ineffective red blood cell productions...