Clinical Trials
APOLLO is a Phase 3, randomized, double‑blind, placebo-controlled clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in adults and adolescents 12 or older with erythropoietic protoporphyria (EPP) or X‑linked protoporphyria (XLP).
HELIOS is an open-label extension clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in adults and adolescents 12 or older with EPP or XLP who have participated in previous bitopertin EPP or XLP clinical trials.
This study has been completed and is no longer enrolling.
AURORA was 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.
This study has been completed and is no longer enrolling.
BEACON was a Phase 2, open-label, multiple‑dose clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in patients with EPP or XLP in Australia.
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.
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.
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).
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 Polycythemia Vera patients, for which DISC‑3405 has received Fast Track Designation.
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.
If you are interested in learning more about our clinical programs and how you can get involved, please email us at
