Clinical Trials

Using new discoveries and insights in hematology, Disc Medicine is
advancing the frontiers of biology to create life-changing medicines.
Disc is currently conducting multiple clinical trials to evaluate its drugs
for use in people with hematologic diseases. It takes a village to create
even the smallest steps forward in the development of therapeutics. Our
work would not be possible without the patients, volunteers,
investigators, families, and hospitals that participate in our clinical trials
– we are honored to be making a difference with you.

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AURORA is a Phase 2 randomized, double-blind, placebo-controlled, parallel dosing clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in adults with EPP in the United States.

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BEACON is a Phase 2 study and a randomized, open-label, multiple dose clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in patients with EPP or XLP being conducted in Australia.

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Anemia of Myelofibrosis (MF)

In June 2022, Disc Medicine initiated an open label, multi-center, Phase 1b/2 trial and will evaluate the safety, tolerability, and efficacy of DISC-0974 in myelofibrosis patients with anemia.

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Anemia of Chronic Kidney Disease (CKD)

In February 2023, Disc Medicine initiated a multi-center, Phase 1b/2 trial and will evaluate the safety, tolerability, and efficacy of DISC-0974 in NDD-CKD patients with anemia.

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Diamond-Blackfan Anemia

Disc Medicine is collaborating with the National Heart Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) to evaluate bitopertin, a therapeutic candidate designed to modulate heme biosynthesis, in a phase 2 clinical study of patients with Diamond-Blackfan anemia (DBA).

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Healthy Volunteers

In October 2023, Disc Medicine initiated a randomized, double-blind, placebo-controlled, single- and multiple-ascending dose Phase 1 trial in healthy volunteers. Following completion of this study, Disc plans to initiate a trial in healthy volunteers, for which DISC-3405 has received Fast Track Designation.

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Disc Medicine’s Expanded Access Policy

Disc is dedicated to the discovery and development of novel medicines for patients with a wide range of serious diseases. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.

Expanded access, sometimes referred to as compassionate use, refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition or immediately life-threatening disease or condition in patients who lack satisfactory therapeutic alternatives. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.

At this time, we believe that participation in one of our clinical trials is the appropriate way to access our investigational therapies. If you have additional questions, please speak with your physician or contact clinicaltrials@discmedicine.com. We anticipate acknowledging receipt of requests sent to this email within five business days.

Disc Medicine may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on clinicaltrials.gov after such record becomes active.