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DISC-1459

Phase 3

Erythropoietic Protoporphyria (EPP) | Small molecule | Other |Disc Medicine, Inc.|Last Updated: Jun 4, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment183
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06910358Study of Bitopertin in Participants With EPP or XLP (APOLLO)PHASE3 ACTIVE NOT_RECRUITING 183 — — Apr 4, 2025Oct 1, 2026Jun 4, 202627 United States, Australia +10
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Study Endpoints
Primary Endpoints
Average monthly total time in sunlight on days without pain from a phototoxic reaction between 10:00 to 18:00 (10:00 AM to 6:00 PM) after 6 months (24 weeks) of treatment
24 weeks
Percent change from baseline in whole-blood metal-free PPIX levels at 6 months
24 weeks
Safety and tolerability, as assessed by adverse events (AEs) and laboratory results, over the 6-month treatment period
24 weeks
Secondary Endpoints
Occurrence of phototoxic reactions over the 6-month treatment period
24 weeks
Cumulative total time in sunlight on days without pain from a phototoxic reaction between 10:00 to 18:00 (10:00 AM to 6:00 PM) over the 6-month (24-week) treatment period
24 weeks
Change from baseline in 2-week average daily sunlight exposure time (minutes) to first prodromal symptom (eg, burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post-sunrise and 1 hour pre-sunset at 6 months
24 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATOR -
DISC-1459 oral doseEXPERIMENTAL -
Interventions
NameTypeDescription
PlaceboDRUGOral dose, once a day for 24 weeks
DISC-1459DRUGOral dose, once a day for 24 weeks
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites27

Inclusion Criteria: 1. Aged 12 years or older at the time of study consent. 2. Diagnosis of EPP or XLP, based on medical history by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis. 3. Minimum daily Sun Exposure Diary compliance ≥85% on...

Countries:United StatesAustraliaBelgiumCanadaFranceGermanyIrelandItalyNetherlandsSpainSwedenUnited Kingdom
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Recent Changes (Last 90 Days)
LOWJun 4, 2026NCT06910358lastUpdatePostDate: changed
LOWJun 4, 2026NCT06910358lastUpdatePostDate: changed
LOWJun 4, 2026NCT06910358lastUpdatePostDate: changed
LOWJun 4, 2026NCT06910358lastUpdatePostDate: changed
LOWJun 2, 2026NCT06910358lastUpdatePostDate: changed
LOWJun 2, 2026NCT06910358lastUpdatePostDate: changed
LOWJun 2, 2026NCT06910358lastUpdatePostDate: changed
MEDIUMMay 26, 2026NCT06910358Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT06910358studyFirstPostDate: changed