- 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 inhibiting ABCG2 in both erythrocytes and hepatocytes

- In EPP/XLP, the plasma is the relevant compartment that mediates disease by facilitating deposition of PPIX in the skin and liver

- PORT-77 is currently being investigated in the Phase 2A proof-of-concept trial, GATEWAY, with the primary endpoint of plasma PPIX reduction

- Orphan Drug and Fast Track Designations validate the need for novel therapies in EPP/XLP and our strategy to develop differentiated therapies with PORT-77

GondolaBio, a clinical-stage biopharmaceutical company developing novel medicines for patients living with genetic diseases, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Orphan Drug Designation and Fast Track Designation to its affiliate, Portal Therapeutics, for PORT-77, an investigational oral, small molecule ABCG2 inhibitor, for the treatment of EPP and XLP.

“For individuals living with protoporphyria, avoiding sunlight is a daily struggle that significantly affects their quality of life. The constant risk of skin and liver damage underscores the urgency of bringing disease-modifying treatments to this population as there remains a great unmet need,” said Neil Kumar, Co-founder and CEO of GondolaBio. “GondolaBio’s unique decentralized structure allows us to pursue treatments for rare diseases like EPP and XLP, and through these designations, we look forward to further collaboration with FDA as we advance this program, with plans to report full Phase 2 data in the near-term.”

In preclinical and Phase 1 healthy volunteer studies, PORT-77 has demonstrated the ability to significantly lower plasma PPIX, which has the potential to address both the phototoxic and hepatobiliary impacts of EPP and XLP. PPIX reduction occurred within hours after dosing via rapid ABCG2 inhibition with no serious adverse events and no safety or tolerability signals identified to date.

The FDA grants Orphan Drug Designation to support the development and evaluation of new treatments for rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. Benefits include potential eligibility for expedited review pathways, tax credits for qualified clinical trials, fee waivers, and seven years of market exclusivity after approval.

Fast Track is a process designed by the FDA to facilitate the development and expedite the review of investigational drugs intended to treat serious conditions and for which nonclinical or clinical data demonstrate the potential to address unmet medical need. Therapeutic candidates with Fast Track Designation may also be eligible for priority review and accelerated approval if supported by clinical data.

About Erythropoietic Protoporphyria and X-linked Protoporphyria

EPP and XLP, which affects greater than 25,000 people in the U.S. and EU, are genetic photodermatoses that result in the accumulation of protoporphyrin IX (PPIX). Affected patients experience cutaneous damage and excruciating pain when exposed to sunlight. Approximately 20-30% of patients with EPP/XLP will experience some type of liver damage with up to 5% of patients progressing to acute liver failure and requiring a liver transplant. There are no approved disease-modifying treatments available.

About PORT-77

PORT-77 is an oral, small molecule ABCG2 inhibitor currently in Phase 2 development for EPP and XLP. PORT-77 is designed to limit efflux of PPIX from the red blood cell to the plasma and from hepatocytes to the biliary tract, representing a potentially disease-modifying, best-in-class treatment that addresses the root cause of EPP and XLP. The ongoing clinical program is designed to determine if PORT-77 can effectively reduce plasma PPIX to improve sunlight sensitivity and reduce liver damage.

About GondolaBio

GondolaBio is a clinical-stage biopharmaceutical company focused on developing novel medicines for patients living with genetic diseases. A sister company of BridgeBio Pharma, GondolaBio aims to leverage cutting-edge biological research to create breakthrough medicines addressing high unmet needs across multiple therapeutic areas, modalities, and stages ranging from early discovery to clinical development. Its team of experienced drug discoverers, developers and innovators are committed to translating advances in genetic science into medicines that can help patients as quickly as possible.

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