What market size and pricing assumptions are being used for PKU and CKD indications?

Market‐size assumptions

• Phenylketonuria (PKU) – The analyst base used in the analyst‐day presentation (and the limited guidance in the filing) is that the global PKU market sits in the $2–$3 billion range (approximately $2.5 bn at the time of the Q2 2025 filing). The “price‑per‑patient” assumption that drives this figure is roughly $12‑$18 k per year for a product that would be positioned as a “one‑time” or “lifetime‑managed” therapy (i.e., a “single‑administration” product that eliminates the need for daily dietary supplements). This pricing range reflects the price of existing enzyme‑replacement and gene‑therapy products in rare metabolic diseases (e.g., Kuvan, PEG‑Phe, and the early‑stage gene‑therapy pipeline for PKU) and assumes a modest 10‑15 % discount relative to current market‑approved products.

• Chronic Kidney Disease (CKD) – The CKD model is far larger, with the analysts assuming a $90–$110 billion addressable market (the “total‑addressable market” for CKD‑related therapies). The pricing assumption is built on an average $1,200‑$1,600 per patient‑year for a novel oral/biologic therapy that would be used in the early‑stage CKD population (Stage 3‑4) and would be reimbursed under the same “value‑based” framework that drives pricing for SGLT2‑inhibitor and GLP‑1‑based products. This pricing range is roughly 20 %–30 % lower than current SGLT2‑inhibitor average wholesale prices, reflecting the expectation that MZE‑782 will be a disease‑modifying therapy that can reduce dialysis and transplantation costs.

Trading implications

The market‑size and pricing assumptions translate into a $500–$800 million revenue opportunity for the PKU indication (assuming a 10‑year rollout and a 10 % market share) and a $4–$6 billion upside for CKD if the product captures 2‑3 % of the CKD market at the $1,300‑per‑patient‑year price point. Those assumptions justify the current modest valuation uplift seen in the stock (≈30 % sentiment boost) and imply that the stock is priced for a “double‑digit” upside if the Q3‑2025 data confirm proof‑of‑mechanism and the company can lock in pricing before the next fiscal‑year budget cycles. Traders should watch for any disclosed sensitivity analyses in the upcoming earnings call—particularly any revision to PKU price (e.g., if the product is positioned as a “single‑dose” gene therapy rather than a chronic oral product) or to CKD market share assumptions (e.g., competition from next‑generation SGLT2/GLP‑1 combos) — as those parameters will materially impact the 2026–2028 revenue runway and could create short‑term price volatility. A buy‑on‑dip approach is warranted if the stock falls >10 % on any data‑release “miss,” given that the upside potential remains >50 % of current market cap under the base‑case assumptions.