PHAR

• Company Transformation & Strategic Pillars

  • The firm is transforming from a single-asset business to a diversified rare disease company with three core pillars: Ruconest as a durable cash flow source, Joenja as a high-growth early-stage asset, and a high-value pipeline with two potential blockbuster opportunities.
  • The long-term goal is to become a leading global rare disease company.

• Ruconest Commercial & Operational Updates

  • The 10% year-over-year revenue decline reflects U.S. competitive dynamics, planned withdrawal from international markets, and temporary inventory normalization.
  • Q2 2026 new patient enrollments rose to 84 from ~50 in Q1 2026, returning to near 2025 Q2 levels, with 17 new prescribers added during the quarter.
  • Ruconest holds a differentiated position for high-burden HAE patients with frequent/severe attacks, with clinical data showing 97% of acute attacks require only one dose, 93% of attacks are controlled for at least three days, and a median 4.5-hour time to complete attack resolution. Its specialized manufacturing process limits biosimilar competition, supporting long-term commercial durability.

• Joenja Commercial & Operational Updates

  • Commercial momentum is accelerating in Europe, with launch underway in Germany and planned Japan launch in Q3 2026.
  • The SNDA filing for lower 20 and 30 milligram pediatric doses was submitted to the FDA on the day of the call, with a PDUFA date of October 24 for higher 40 and 50 milligram doses.
  • Joenja has a well-established safety and efficacy profile in APDS, with durable benefits observed over up to seven years of treatment.

• Pipeline Updates

  • Two Phase II studies of leniolisib (Joenja) for broader primary immunodeficiency (including CVID) are fully enrolled, with top-line results expected in Q4 2026. These indications have an addressable patient population up to 40 times larger than APDS. Positive results would support a potential registrational Phase III trial and could push Joenja's peak annual sales potential above $1 billion.
  • A pivotal study readout for Napazimone in primary mitochondrial disease is expected in 2027.

• Financial Performance

  • Reported operating profit was positive in Q2 2026 despite lower revenue and $9 million in incremental investments year-over-year. The firm maintained disciplined cost controls, with adjusted operating expenses up just 1% year-over-year in the first half of 2026 despite $13 million in incremental R&D investment.

• Full-year 2026 revenue guidance was revised downward by $30 million to a range of $375 to $395 million, representing 0% to 5% year-over-year growth versus 2025. The revision reflects updated visibility into HAE market dynamics following the launch of the competing oral on-demand treatment.
• Ruconest U.S. revenue is expected to decline approximately 3% full-year 2026, with revenue expected to stabilize in the second half of 2026 and return to growth. Management reaffirms Ruconest will remain a durable long-term cash flow engine.
• Joenja full-year 2026 revenue growth is expected to be in the high 30% year-over-year, up from 29% growth in 2025, supported by geographic expansion (Germany, Japan) and the expected U.S. pediatric label expansion.
• Full-year 2026 operating expense guidance was reduced by $15 million to a range of $315 to $320 million, representing 1% to 3% year-over-year growth. This includes over $40 million in incremental R&D investment and an approximately $9 million benefit from the 20% structural G&A headcount reduction announced in October 2025.
• Full-year 2026 cost of goods sold, including the $4.9 million Q2 2026 manufacturing inventory impairment, is expected to be approximately 11% of revenue, corresponding to a gross margin of ~89%.
• Management confirms existing cash and future operating cash flow are sufficient to fund the current development pipeline and all prelaunch activities.

Total Q2 2026 revenue was $90.2 million, a 3% year-over-year decline.

• Ruconest: Q2 revenue was $72.3 million, a 10% year-over-year decline, contributing 80.2% of total Q2 revenue. Sequentially, revenue was up 24% from Q1 2026. First half 2026 Ruconest revenue was 12% lower than the prior year period. The active patient base remained at 93% of year-ago levels one year after the launch of a competing oral on-demand treatment for HAE.
• Joenja: Q2 revenue was $17.9 million, a 40% year-over-year increase, contributing 19.8% of total Q2 revenue. U.S. Joenja revenue grew 31% year-over-year to $15.4 million, while international revenue grew 150% year-over-year to $2.5 million. First half 2026 Joenja revenue grew 37% year-over-year. By the end of Q2 2026, 132 patients were on paid Joenja therapy, up 16% year-over-year.

• Competitive pressure from the newly launched oral on-demand HAE treatment has caused year-over-year revenue decline for Ruconest, even as the active patient base remains resilient.
• Ruconest manufacturing inventory was impaired by $4.9 million in Q2 2026, as some inventory may not be usable for commercial sale; a final resolution of the issue is expected by the end of Q3 2026.
• The planned closure of the Ebrie, France production support site in Q4 2026 has resulted in a $1.7 million non-recurring provision in Q2 2026.
• Success of Joenja's expansion into broader primary immunodeficiency indications is contingent on positive Phase II results and successful completion of a subsequent registrational Phase III trial, with no guarantee of regulatory approval.
• Revenue growth from Joenja's geographic and pediatric expansion is dependent on successful pricing and reimbursement approval in new markets, as well as conversion of identified patients to paid therapy.

Q: What progress has been made on Joenja's EU commercial launches, and what are the next steps after positive PID Phase II results? / A: Management has identified 387 potentially eligible APDS patients across 8 core ex-U.S. markets. Launch is active in the UK and Germany, with Japan launching in August 2026 after securing pricing and reimbursement. Following Phase II readout in Q4 2026, management will review results, evaluate the dose regimen, and discuss a registrational path with global regulators. A single combined randomized Phase III trial for CVID is currently planned, pending positive results. (347 characters)

Q: Has the Q2 2026 recovery in Ruconest new patient enrollments continued into Q3 2026, and what is the expected conversion pace of eligible U.S. pediatric APDS patients after approval? / A: The momentum in new patient enrollments seen in Q2 has remained consistent through early July, with some expected seasonal variation typical for the HAE market. 60 eligible pediatric patients are already identified in the U.S., split equally between higher and lower dose groups. Management expects a similar uptake/conversion rate as seen in the adult population, with EAP patients converting first in the months after approval. (398 characters)

Q: What share of new Ruconest enrollees are high-burden patients, and are CVID patients already diagnosed for potential commercial access if the new indication is approved? / A: All 84 Q2 new Ruconest enrollments are first-time patients (no restarts), and the vast majority are high-burden patients with frequent, severe attacks who prefer Ruconest's reliable, rapid onset of action. Unlike APDS (which required extensive new patient identification and genetic testing outreach), CVID is already a well-established clinically diagnosed condition, with most eligible patients already under physician care and on replacement therapy. (392 characters)

Q: Could the upcoming Phase II PID data drive off-label Joenja revenue before full regulatory approval? / A: Management will not promote off-label use of Joenja for PID or CVID before formal regulatory approval. All commercial growth in the near term will be driven by the existing approved APDS indication, supported by U.S. pediatric expansion and geographic expansion into new markets, while the new indications go through the full clinical and regulatory process. (289 characters)