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Canaccord Genuity 2026

BMRN BioMarin GC 2026 questions

Published August 10, 2026

Anchored to Q2 2026 print (August 6, 2026). Fiscal year ends in December, so Q2 FY26 = CQ2 2026. No venue supplied — questions are drafted generic. Sources: Q2 2026 Earnings Call, Q2 2026 Earnings Call Presentation, Q2 2026 Earnings Release, BMRN Case Study CQ 2026 Q2, Q1 2026 Earnings Call.

Amicus Integration & Synergy Waypoints

  1. Brian said 2028 is the first full year of the ~$220M non‑GAAP / ~$280M GAAP synergy program and guided "half to slightly more than half" of synergies in 2027 — what are the concrete milestones (site consolidations, ERP cut‑over, headcount actions, external‑spend renegotiations) investors should be able to check against between now and the Q4 print? (Q2 2026 Earnings Call)
  2. Deal is now characterized as "close to breakeven" in 2026 vs. "slightly dilutive" three months ago — what specifically changed in the base case (Galafold/Pom‑Op run‑rate, cost timing, FX, tax), and is the "substantial accretion" 2027 language now conservative? (Q2 2026 Earnings Call, Q2 2026 Earnings Release)
  3. Gross‑leverage target of <2.5x was pulled forward ~1 year to mid‑2027 — how much of that pull‑forward is EBITDA growth vs. active debt paydown, and does the plan assume any equity issuance or asset sales to hit it? (Q2 2026 Earnings Release)

Galafold & Pombiliti/Opfolda — The Growth Thesis

  1. Peak revenue guidance went to $1.4B for Galafold (from ~$1B) and $1.2B for Pom‑Op (from ~$1B), with combined non‑GAAP OM >60% by 2030 — what is the split between diagnosis expansion, switching, geographic rollout, and pricing in that bridge, and which is the largest single lever? (Q2 2026 Earnings Call, Q2 2026 Earnings Release)
  2. Cristin said "generously maybe only 40% of the patients are actually diagnosed" in Fabry after 20+ years of Genzyme/Amicus effort — what testing‑yield or family‑cascade metric will management commit to reporting quarterly so the Street can track whether BioMarin's platform is materially changing the curve? (Q2 2026 Earnings Call)
  3. Pom‑Op is guided to a ≥20% CAGR 2027–2032 driven "primarily by increased patient switching" — what real‑world switching data (proteinuria, 6MWT, LOPD‑specific endpoints) does management need on hand to accelerate switching from Sanofi's franchise, and by when will BioMarin have it? (Q2 2026 Earnings Call)
  4. Cristin flagged expansion of >10 additional Galafold markets and >20 additional Pom‑Op markets — which handful of countries do the peak numbers actually depend on for reimbursement, and where is pricing/access risk highest vs. the Amicus base case? (Q2 2026 Earnings Call, Q2 2026 Earnings Release)

VOXZOGO — Competitive Response to YUVIWEL

  1. U.S. switch rate is ~10% (<100 patients) and 90% retention six months after Ascendis's launch — Ascendis has 170 patients enrolled with 2/3 paid, so the delta implies most of their volume is naive starts, not switches. What share of new U.S. starts did BioMarin capture in Q2, and how does that compare to Q1? (Q2 2026 Earnings Call)
  2. Guidance was raised to $1.00–$1.05B for VOXZOGO but Brian explicitly declined to quantify the embedded switch assumption — what switch percentage is baked into the low end, and at what U.S. switch rate does the guide start to break? (Q2 2026 Earnings Release, Q2 2026 Earnings Call)
  3. The under‑2 U.S. incidence pool is only ~150 births/year, and management has framed <2 exclusivity as the moat — how does that math get you to a durable multi‑year U.S. growth franchise once the incidence pool is fully penetrated, and when does U.S. growth flatten in the base case? (Q2 2026 Earnings Call)

VOXZOGO — Hypochondroplasia Launch Prep

  1. CANOPY‑HCH‑3 hit primary endpoint with LS mean AGV difference of +2.33 cm/yr, sNDA submitted, PDUFA Feb 28, 2027 — of the ~14,000 global HCH TAPP, how many patients have already been identified in the pre‑launch diagnostic push, and what commercial ramp assumption should the Street model for the first 12 months post‑approval? (Q2 2026 Earnings Release, Q2 2026 Earnings Call)
  2. HCH patients are typically diagnosed later than achondroplasia (less obvious growth deficit) — what is management doing on the payer side ahead of the PDUFA to make sure reimbursement doesn't lag approval, particularly ex‑U.S.? (BMRN Case Study CQ 2026 Q2)

BMN 333 — The Next‑Gen Bet

  1. Phase 1 showed free CNP exposure >13x another long‑acting CNP at the top dose — but the Phase 2/3 is running a superiority design against VOXZOGO active control, which is a much higher bar than non‑inferiority. What effect size on 6‑month AGV would management consider a "win" that supports both a regulatory filing and pricing above VOXZOGO? (Q2 2026 Earnings Release, BMRN Case Study CQ 2026 Q2)
  2. Greg said Phase 2 enrollment is "entering the steep part of the curve" with the next update in 2027 after full enrollment — given competitor CNP + growth hormone combination data now emerging (COACH, 21 pts), does management see any scenario where the BMN 333 program needs to be re‑designed to include a combo arm, or is the standalone superiority path final? (Q2 2026 Earnings Call)

Pipeline Reset — 401 Failure, 351, 820, ReNU

  1. BMN 401 has been fully discontinued after missing one of two co‑primary endpoints in ENERGY 3 — three months ago management called it a "potential first‑in‑disease" 2027 launch. What went wrong in the trial design vs. the biology, and what governance or diligence process changes has R&D put in place to reduce the chance of another late‑stage surprise in BMN 351 or BMN 820? (Q2 2026 Earnings Release, BMRN Case Study CQ 2026 Q2)
  2. BMN 351 (Duchenne) has a promised update by year‑end — the last read‑out showed dose‑dependent dystrophin at 6 and 9 mg/kg with the 12 mg/kg cohort still enrolling. What specifically will define a "go" decision to a pivotal design, and is BioMarin willing to fund BMN 351 through pivotal on its own balance sheet given the added Amicus interest burden? (BMRN Case Study CQ 2026 Q2, Q2 2026 Earnings Call)
  3. BMN 820 (FSGS, ~30k U.S. TAPP) is Phase 3 with pivotal data in 2028, but Dimerix operates the trial and BioMarin only holds U.S. rights — how much revenue does the current base case assume from BMN 820, and what is the ex‑U.S. economic split investors should model? (Q2 2026 Earnings Call)

Capital Allocation, BD & the Interest Burden

  1. Alexander telegraphed clinical‑stage BD "over the next 12–18 months" — with $874M cash, $4.19B gross debt, and $200M/yr of annualized interest already in the run‑rate, what is the realistic BD envelope (cash‑only bolt‑ons vs. deals that would require re‑levering), and is stock a live currency at current multiples? (Q2 2026 Earnings Call, Q2 2026 Earnings Release)
  2. Non‑GAAP OM slipped to 36.4% in Q2 vs. 39.9% Y/Y and GAAP OM collapsed to 11.2% from 33.5% — walk us through the 2027/2028 bridge to the "over 60% non‑GAAP OM by 2030" for the acquired Amicus franchises alone, and what the consolidated non‑GAAP OM looks like at that point given ongoing pipeline reinvestment. (Q2 2026 Earnings Release, Q2 2026 Earnings Call)

Bear‑Case Pressure Points

  1. ITC administrative‑law‑judge initial determination lands August 21 with a final commission decision expected December 21 — under a loss scenario (no exclusion order), does anything in the current VOXZOGO peak‑sales / IP‑runway framing change materially, or has the base case already de‑risked the ITC path? (Q2 2026 Earnings Call)
  2. Roctavian is being wound down ("company will no longer market") after $9M in Q2, and VIMIZIM (‑10% Y/Y to $194M) and ALDURAZYME (‑21% to $44M) are both shrinking — how much of the guided 2H metabolic conditions acceleration is core growth vs. an easy set of comps and Amicus in‑period, and what is the through‑cycle organic growth algorithm for legacy enzyme therapies excluding Amicus? (Q2 2026 Earnings Release)

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