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PORT-77 · 2 trials · 3 indications
Characterize the dose-response of PORT-77 on plasma PPIX levels compared to placebo
Evaluate safety and tolerability of each dose of PORT-77 over the course of the study
Assess the effect of different doses of PORT-77 on average daily time in sunlight without pain compared to placebo
Change over the course of the study in plasma PPIX concentrations
| Arm | Type | Description |
|---|---|---|
| Phase 2b (Treatment Group 1) | EXPERIMENTAL | PORT-77 Dose 1 |
| Phase 2b (Treatment Group 2) | EXPERIMENTAL | PORT-77 Dose 2 |
| Phase 2b (Treatment Group 3) | PLACEBO_COMPARATOR | Placebo |
| Phase 3 (Treatment Group 1) | EXPERIMENTAL | PORT-77 |
| Phase 3 (Treatment Group 2) | PLACEBO_COMPARATOR | Placebo |
| Phase 3 (Open-Label Extension) | EXPERIMENTAL | PORT-77 |
| PORT-77 | EXPERIMENTAL | Active |
| Placebo | PLACEBO_COMPARATOR | Placebo |
| Name | Type | Description |
|---|---|---|
| PORT-77 | DRUG | Oral tablets |
| Placebo | DRUG | Oral tablets |
Inclusion Criteria: * Aged 12 years or older. * Clinical history of EPP, XLP, or EPP2 supported by genetic confirmation or historical laboratory test results * History of consistent, non-painful prodrome within approximately 45 minutes of sunlight exposure and prior to phototoxic attacks * Demonstr...
PORT-77 is an investigational oral small molecule being developed for erythropoietic protoporphyria, a rare inherited disorder. It is also being studied in X-linked protoporphyria. BridgeBio Pharma is running a Phase 2 program that includes a completed Phase 2a study and an ongoing placebo-controlled Phase 2 trial.
PORT-77 is being developed by BridgeBio Pharma, Inc., which trades on the Nasdaq under the ticker BBIO. BridgeBio is a commercial-stage biopharmaceutical company focused on genetic diseases and rare conditions, and PORT-77 is part of its rare disease pipeline.
PORT-77 is in Phase 2 clinical development. One Phase 2a study in adults with erythropoietic protoporphyria has been completed, and a larger Phase 2 trial is currently recruiting. PORT-77 is investigational and has not been approved by the FDA for any indication.
PORT-77 has been evaluated in two Phase 2 trials. The completed GATEWAY Phase 2a study, NCT06971900, enrolled 28 adults with erythropoietic protoporphyria. The ongoing Phase 2 trial, NCT07820085, is recruiting 225 participants aged 12 and older with EPP or X-linked protoporphyria in the United States.
NCT07820085 is a randomized, double-blind, placebo-controlled Phase 2 trial of oral PORT-77 in participants with erythropoietic protoporphyria or X-linked protoporphyria. It plans to enroll 225 participants aged 12 years and older, is recruiting in the United States, and does not enroll healthy volunteers.
PORT-77 is an oral small molecule drug candidate in Phase 2 development by BridgeBio Pharma for erythropoietic protoporphyria and X-linked protoporphyria, two rare inherited disorders of heme biosynthesis. It is investigational and has not received regulatory approval.