United Therapeutics (UTHR) investor relations material

Key clinical and commercial milestones

• Reported best-ever clinical trial results for pulmonary hypertension and pulmonary fibrosis, with Tyvaso and ralinepag showing significant improvements in patient outcomes and forced vital capacity, respectively.

• Physicians forecast Tyvaso as the primary therapy for pulmonary fibrosis and ralinepag for pulmonary hypertension, targeting up to 100,000 and 50,000 patients in the US, respectively.

• Expecting FDA filing for ralinepag mid-2024 and approval by mid-2025, with rapid commercialization and projected $3 billion in revenue from 15,000 patients within two years of launch.

• Peak revenues for ralinepag could reach five times launch revenues by the 2030s, driven by expansion to patients on dual oral background therapy.

• Tyvaso's launch in pulmonary fibrosis could rapidly reach 30,000 patients, with peak revenues potentially 10x launch revenues due to broader disease prevalence.

Product pipeline and innovation

• TETON-1 confirmatory trial for Tyvaso in idiopathic pulmonary fibrosis expected to mirror strong TETON-2 results, supporting broad adoption.

• Tyvaso positioned for use in three patient groups: those intolerant to background therapy, those on background therapy, and those on newer IPF drugs, with proven synergy and improved tolerability.

• Announced Trezist, a soft mist inhaler with 90% reduction in cough, aiming for FDA filing in 2026 and launch in 2027, expected to transform inhalation therapy.

• Pipeline includes once-daily inhaler, PRN rescue inhaler, and a triple-combination oral pill, with launches planned through 2028.

Organ manufacturing and AI-enabled platforms

• Advancing xenokidney clinical trials, with two patients showing normal kidney function and plans to enroll up to 50 patients by 2027, aiming for BLA filing in 2028 and potential approval in 2030.

• UThymoKidney combines kidney and thymus transplantation to promote immune tolerance, with first patient transplant expected within three months.

• AI-enabled digital lung model accelerates drug development, accurately predicts trial outcomes, and is being presented to the FDA as supportive evidence for future filings.