Travere Therapeutics (TVTX) investor relations material

Strategic focus and growth pillars

• Emphasis on rare diseases with a commercial-stage portfolio centered on four pillars: FILSPARI for IgA nephropathy and FSGS, pegtibatinase for classical homocystinuria, and the newly in-licensed civorebrutinib targeting rare kidney indications.

• FILSPARI maintains foundational positioning in IgA nephropathy and has seen strong uptake in FSGS following its recent approval for this indication.

• Pegtibatinase is advancing in a pivotal phase III HARMONY study for classical homocystinuria, with data expected in the second half of next year.

• Civorebrutinib, a next-generation BTK inhibitor, is expected to complement existing therapies and expand the pipeline's reach in rare renal diseases.

• The company is open to further pipeline expansion, focusing on rare renal and metabolic opportunities.

FILSPARI commercial performance and market opportunity

• FILSPARI exceeded internal expectations in its FSGS launch, with broad physician adoption and no evidence of warehousing or acute demand spikes.

• Over 30,000 FSGS patients are currently eligible, with potential for further growth as diagnosis rates improve and more patients become eligible.

• FILSPARI is the most prescribed medicine for IgA nephropathy, with over 2,000 Patient Start Forms in the latest quarter across both indications.

• Peak sales opportunity for FILSPARI is projected at over $3 billion, targeting more than 100,000 patients across IgA nephropathy and FSGS.

• Recent method-of-use patent allowance for IgAN extends exclusivity to October 2037, with a similar patent in process for FSGS.

Pipeline development and market expansion

• Pegtibatinase addresses an underserved population in classical homocystinuria, with about 3,500 addressable patients in the U.S. and similar numbers abroad.

• The HARMONY study aims for a readout in the second half of next year, with prior data showing robust reductions in homocysteine levels.

• Civorebrutinib is positioned to address three orphan indications—PMN, immune-mediated FSGS, and minimal change disease—covering a combined population of around 130,000 patients.

• The company leverages its rare renal infrastructure for new pipeline assets and considers both partnership and independent commercialization strategies in Europe.

• Combination therapy is expected to become standard in IgA nephropathy, with FILSPARI maintaining a foundational role alongside emerging therapies.