DISC-1459

Phase 3Small molecule

DISC-1459 is one of 3 small molecule programs targeting Erythropoietic Protoporphyria (EPP). It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Erythropoietic Protoporphyria (EPP)Active, not recruiting

Erythropoietic ProtoporphyriaEnrolling by invitation

Erythropoietic Protoporphyria (EPP)Phase 3

Active, not recruiting

Erythropoietic ProtoporphyriaPreclinical

Enrolling by invitation

Competition

Competitive Landscape

2 competitors in Erythropoietic Protoporphyria

#DrugCompanyPhaseTrials

DISC-1459

DDisc Medicine, Inc

Phase 3

1 2

Nydrazid

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Preclinical

1 3

Iron

WWatson Pharmaceuticals

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 3Active, not recruiting

Erythropoietic Protoporphyria (EPP)

NCT06910358

Clinical

Trials by Indication[1]

Erythropoietic Protoporphyria (EPP)2 trials

APOLLO: A Randomized, Double-Blind, Placebo-Controlled Study of Bitopertin to Evaluate the Efficacy, Safety, and Tolerability in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Active, not recruiting·DDisc Medicine, Inc·Phase 3·NCT06910358·183 enrolled

Expanded Access Program Protocol: Bitopertin (DISC-1459) For The Treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)

Available·DDisc Medicine, Inc··NCT07603401

Concentration1 trial

Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

Completed·DDisc Medicine, Inc·Phase 2·NCT05308472·75 enrolled

Erythropoietic Protoporphyria1 trial

HELIOS: An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

Enrolling by invitation·DDisc Medicine, Inc·Preclinical·NCT05883748·230 enrolled

Sources

References

[1]
ClinicalTrials.gov — 4 trial records for DISC-1459. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — DISC-1459 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for DISC-1459. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Disc Medicine, Inc company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: DDisc Medicine, Inc

DISC-1459

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Trials by Indication[1]

Erythropoietic Protoporphyria (EPP)2 trials

Concentration1 trial

Erythropoietic Protoporphyria1 trial

References

Drug Info

Quick Facts

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