PORT-77 for Erythropoietic Protoporphyria

(EPP Trial)

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Portal Therapeutics, Inc.

Must not be taking: Supplements

Disqualifiers: Cancer, Hepatic impairment, Pregnancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 9 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called PORT-77 for individuals with erythropoietic protoporphyria (EPP), a condition that causes painful reactions to sunlight. The study aims to determine if PORT-77 can reduce symptoms compared to a placebo (a pill with no active medicine). Individuals diagnosed with EPP who continue to experience symptoms despite treatments like afamelanotide might be suitable candidates. Participants should be able to avoid sunlight and adhere to the study's guidelines. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires participants to avoid using medications or supplements that could interfere with PORT-77 starting 28 days before the first dose and throughout the study. It's best to discuss your current medications with the study team to see if they might be affected.

Is there any evidence suggesting that PORT-77 is likely to be safe for humans?

Research has shown that PORT-77 is safe and well-tolerated. In studies, this oral treatment demonstrated promising safety results. Tests in labs and on animals revealed positive outcomes without major safety concerns.

Although PORT-77 is still under study for erythropoietic protoporphyria (EPP), it has received special recognition from the FDA. Recognitions such as fast track and orphan drug status indicate the treatment's potential and importance for development. This often occurs when early safety data are encouraging.

So far, evidence supports that PORT-77 is well-tolerated, with no significant side effects reported in early testing. However, since it remains under study, further research will help confirm its safety in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for erythropoietic protoporphyria (EPP), like beta-carotene and afamelanotide, focus on reducing photosensitivity and managing symptoms. But PORT-77 is unique because it works differently, targeting the underlying cause of EPP by potentially modifying protoporphyrin IX accumulation in the body, which is responsible for the condition's symptoms. Researchers are excited about PORT-77 because it offers a new mechanism of action that could provide a more effective and targeted approach to managing EPP.

What evidence suggests that PORT-77 might be an effective treatment for erythropoietic protoporphyria?

Research has shown that PORT-77, which participants in this trial may receive, might help treat erythropoietic protoporphyria (EPP) by protecting the skin and liver from damage. In studies with mice exhibiting EPP-like symptoms, PORT-77 reduced skin and liver problems related to the condition. PORT-77 blocks a protein called ABCG2, which helps lower harmful substances in the body. These early findings suggest that PORT-77 could enable people with EPP to spend time outside without pain. Although detailed information from human studies is not yet available, the potential remains promising. ^{1 2 3 4 6}

Who Is on the Research Team?

Chief Medical Officer

Principal Investigator

Portal Therapeutics, Inc.

Are You a Good Fit for This Trial?

Adults with a condition called erythropoietic protoporphyria (EPP) who experience symptoms even under treatment can join. They must understand English, follow sun exposure and contraception guidelines, have a BMI between 18.0-35.0 kg/m2, and weigh at least 50 kg.

Inclusion Criteria

Able to read and understand English

Willing and able to avoid exposure to sunlight when outside of the clinical research site

Willing and able to follow protocol-specified contraception guidance

Exclusion Criteria

History or presence of any illness or clinically significant medical or psychiatric condition or disease that, in the opinion of the PI or designee, might confound the results of the study or pose an additional risk to the participant by their participation in the study

Participation in another clinical study within 28 days or within 5 half-lives (if known), prior to screening

Estimated glomerular filtration rate <80 mL/min/1.73 m2 using the CKD-EPI equation at screening

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive PORT-77 or placebo for 9 days

9 days

Daily visits for dosing and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

2-4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PORT-77

Trial Overview The trial is testing PORT-77 against a placebo to see if it helps with phototoxicity in EPP patients. Participants will be randomly assigned to receive either the real drug or a placebo for comparison.

How Is the Trial Designed?

Group I: PORT-77 Experimental Treatment 1 Intervention

Active

Group II: Placebo Placebo Group 1 Intervention

Placebo

Who Is Running the Clinical Trial?

Portal Therapeutics, Inc.

Lead Sponsor

Trials

1

Recruited

110+

Celerion

Industry Sponsor

Trials

85

Recruited

7,600+

Dr. Susan Thornton

Celerion

Chief Executive Officer since 2010

PhD in Molecular Biology from the University of Pennsylvania

Dr. Lorraine M. Rusch

Celerion

Chief Medical Officer since 2020

MD from an unspecified institution

Citations

1. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/11/530259/PORT-77-a-Novel-and-Potent-Inhibitor-of-ABCG2

PORT-77, a Novel and Potent Inhibitor of ABCG2, Protects ... In vivo efficacy of PORT-77 was investigated in the FECH mutant mice by evaluating EPP associated skin phototoxicity and liver damage. PPIX ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06971900

NCT06971900 | GATEWAY: A Phase 2a Study of PORT-77 ... No Results Posted Record History. Study Overview. Brief Summary. A Phase 2a study of PORT-77 in adults with erythropoietic protoporphyria (EPP). Official Title.

3. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124027587

PORT-77, a Novel and Potent Inhibitor of ABCG2, Protects ... In vivo efficacy of PORT-77 was investigated in the FECH mutant mice by evaluating EPP associated skin phototoxicity and liver damage. PPIX ...

4. gondolabio.com gondolabio.com/portal-therapeutics-receives-u-s-fda-orphan-drug-and-fast-track-designations-for-port-77-for-the-treatment-of-erythropoietic-protoporphyria-epp-and-x-linked-protoporphyria-xlp/

GondolaBio Receives U.S. FDA Orphan Drug and Fast ... PORT-77 has the potential to be the first therapy in EPP/XLP that may prevent both skin and liver damage with a rapid onset of action by ...

5. porphyrianews.com porphyrianews.com/columns/tfw-treatment-erythropoietic-protoporphyria-becomes-national-priority/

TFW treatment for EPP becomes a national priority Patient-reported outcomes suggest more time outdoors and fewer painful days, which is the heartbeat behind the numbers. Educational aside: A ...

6. porphyrianews.com porphyrianews.com/news/port-77-gets-2-fda-designations-potential-treatment-epp-xlp/

PORT-77 gets 2 FDA designations as treatment for EPP ... PORT-77, an oral small molecule being developed by Gondolabio, received both FDA fast track and orphan drug designations for EPP and XLP.