ZVRA

• Commercial Progress for MyPlypha (U.S. for Niemann-Pick Disease Type C [NPC]) • As of June 30, 2026, 184 total prescription enrollment forms have been received, with 14 added in Q2 2026, bringing first half 2026 enrollments to 23. Enrollments include patients from Puerto Rico, covering U.S. territories. • Payer coverage remains at 69% of covered lives, in line with management expectations. The updated 2026 NPC Clinical Practice Treatment Guidelines added MyPlypha and recommend combination therapy (MyPlypha + Miglistat) for all confirmed NPC patients. • MyPlypha is the only FDA-approved NPC treatment with proven efficacy/safety for combination use, which halts disease progression at 12 months with sustained long-term benefit. • Commercial initiatives include the 'Learn NPC, Read Between the Signs' disease awareness campaign, AI-driven patient targeting, genetic testing collaborations, the Expert Connect clinician education program, and a new NPC Knowledge Hub digital resource. Management continues to engage with payers to expand coverage and participate in clinical congresses to build provider awareness. • Enrollments are split approximately 50% pediatric and 50% adult, across newly diagnosed and previously diagnosed patients, and across centers of excellence and community practices, which confirms management's estimated U.S. NPC prevalence of 900 total patients, 300-350 of whom are diagnosed.

• Regulatory and Global Access Updates for Aramathamol • The CHMP issued a negative opinion on Aramathamol's EU Marketing Authorization Application (MAA) after an oral hearing. Zavra is proceeding with the reexamination process, which will be reviewed by a new rapporteur and co-rapporteur. A CHMP opinion is expected in Q4 2026, 120 days after reexamination submission. Zavra has requested a SAG meeting as part of the reexamination process. • The global EAP continues to operate, with 132 total patients enrolled as of Q2 end, 10 added in Q2 2026, primarily in Europe. The French EAP has been consistent at ~$2.5 million in net revenue per quarter, while other EAP reimbursements and ordering patterns remain highly variable across territories.

• Pipeline Progress for Soliptolol (for vascular Ehlers-Danlos syndrome [VEDS]) • Soliptolol is in the ongoing Phase III DISCOVER event-driven trial. A total of 66 patients have been enrolled (4 added in Q2 2026), with 3 of the 28 required confirmed events needed for interim analysis completed to date. • Management is implementing enrollment acceleration strategies, including expanding the network of genetic testing centers. Following a Q1 2026 Type C meeting with the FDA, management plans to reengage with the FDA in H2 2026 to explore clinical development acceleration pathways.

• Intellectual Property Updates • Zavra secured an Orange Book patent listing for MyPlypha in the U.S. that provides protection through 2041. A separate patent term extension request is pending with the U.S. Patent and Trademark Office, which would fall within the existing 2041 protection period if granted.

• Financial Position • Zavra ended the quarter with $260.2 million in total cash, cash equivalents, and investments, providing sufficient financial flexibility to execute strategic priorities independent of capital market conditions.

• No formal full-year financial or enrollment guidance was provided on the call. Management reaffirmed prior estimates of a U.S. NPC addressable market of 300 to 900 total diagnosed patients, which is supported by the diverse age and care setting distribution of current enrollments.
• A CHMP opinion on the Aramathamol MAA reexamination is expected in Q4 2026, consistent with the standard 120-day timeline for the reexamination process.
• Management confirmed it plans to reengage with the FDA regarding the Soliptolol Phase III program in the second half of 2026, following a Q1 2026 Type C meeting that identified potential development acceleration pathways.

Zavra's total net revenue for Q2 2026 was $39.7 million, a 53% increase year-over-year (YoY). Breakdown by product/segment: 1) MyPlypha (U.S. commercial): $30.2 million in net revenue, representing 76.1% of total Q2 2026 net revenue. U.S. MyPlypha revenue grew 23% quarter-over-quarter (QoQ) and 40% YoY. 2) Aramathamol (Global Expanded Access Program [EAP] net reimbursements): $9.0 million, representing 22.7% of total Q2 2026 net revenue. 3) OPRUPA: $0.2 million, representing 0.5% of total Q2 2026 net revenue. 4) Past-due royalty revenue from ComApps: $0.3 million, representing 0.8% of total Q2 2026 net revenue. Total operating expenses for Q2 2026 were $21.0 million, a $3.2 million decrease YoY. R&D expense was $4.5 million (up $1.1 million YoY), while SG&A expense was $16.6 million (down $4.2 million YoY). Reported net income was $8.8 million, or $0.14 per share, with adjusted net income (excluding one-time 2025 items) of $15.2 million, or $0.25 per share, compared to an adjusted net loss of $2.4 million, or $0.04 per share, in Q2 2025. As of June 30, 2026, total cash, cash equivalents, and investments were $260.2 million, an increase of $23.4 million from March 31, 2026.

• Regulatory risk: The EU MAA for Aramathamol received an initial negative CHMP opinion, and there is uncertainty around the outcome of the reexamination process. Management could not provide a probability of success for the reexamination, and final results may differ from the company's expectations of positive benefit-risk.
• Clinical development risk: The Soliptolol Phase III trial is event-driven, with only 3 of 28 required confirmed events completed to date. Uncertainty remains around how quickly enrollment will accelerate and whether the FDA will agree to the company's proposed development acceleration pathways.
• Revenue volatility: Reimbursements for Aramathamol outside the French EAP are highly variable, and ordering patterns across global territories are unpredictable, leading to potential quarterly volatility in EAP revenue.
• General forward-looking risk: All forward-looking statements (including enrollment, regulatory, and clinical projections) are inherently uncertain, and actual results may differ materially from projections due to the risks outlined in the company's SEC filings (Form 10-K, Form 10-Q).

Q: Given Zavra's strong balance sheet, what is the plan for cash allocation, and can you provide details on the EU Aramathamol reexamination process and chances of reversal? / A: Management's capital allocation priorities remain unchanged: it will focus on driving MyPlypha growth, pursuing global MyPlypha expansion, advancing the Soliptolol pipeline, and evaluating disciplined external value-creating opportunities. For the reexamination, a new independent rapporteur and co-rapporteur will review the existing comprehensive evidence package, and management's confidence is based on the strength of the clinical data and clear unmet patient need in the EU. A requested SAG meeting will bring additional expert input to the process.

Q: What new information or data will Zavra add to the Aramathamol reexamination submission that was not included in the original application? / A: The reexamination process focuses specifically on addressing the specific concerns cited in the original negative opinion, rather than adding entirely new clinical data. Zavra already has a robust evidence package including 4 years of open-label extension data, published EAP data, and pediatric sub-study results, and the process allows for new expert interpretation of this existing evidence to address CHMP concerns.

Q: How does 2026 half-year MyPlypha enrollment compare to 2025 full-year enrollment, and what is the outlook for quarterly enrollment trends? / A: Zavra had 52 total enrollments in full-year 2025, and 23 enrollments in the first half of 2026. Management is increasingly confident in the estimated 350-900 patient U.S. addressable market, as the company's AI-driven targeting and disease awareness initiatives are uncovering newly diagnosed adult and pediatric patients earlier in the launch than expected, supporting continued steady enrollment growth going forward.

Q: Does the new Orange Book IP protection for MyPlypha change the company's appetite for investment in the MyPlypha franchise? / A: The extended IP protection to 2041 gives management more flexibility to consider additional long-term investments in the MyPlypha franchise, alongside investments in global expansion and the Soliptolol pipeline. Management will continue to evaluate opportunities to maximize long-term value from the MyPlypha platform.