Final week, Ascendis Pharma A/S ASND launched topline information on Monday from a trial that included 84 kids with achondroplasia, a genetic dysfunction that causes dwarfism and disproportionate quick stature.
Goldman Sachs hosted an investor assembly with BioMarin Pharmaceutical Inc’s BMRN Alexander Hardy (President and CEO) and Brian Mueller (EVP, CFO).
They reiterated monetary steering after reviewing their assumptions round Voxzogo competitors post-Ascendis Pharma’s better-than-expected Part 3 achondroplasia information.
Additionally Learn: Uncommon Illness-Targeted BioMarin Narrows Gross sales Focus, Cuts Price For Hemophilia A Gene Remedy; Lifts Annual Steerage.
The analyst notes that administration believes a U.S./EU launch by Ascendis Pharma might violate its C-type natriuretic peptide (CNP) patents.
Nevertheless, excluding this concern, the corporate nonetheless expects to succeed in roughly $4 billion in complete income for fiscal 12 months 2027, pushed by rising Voxzogo adoption within the U.S., aided by its broad label with out decrease age restrictions, which might present a aggressive edge over Ascendis Pharma.
The corporate is concentrating on an adjusted working margin of low-to-mid 40% (beginning at 40% in 2026).
The brand new construction, introduced at an investor occasion, covers three key enterprise items: skeletal situations, enzyme therapies, and Roctavian—its hemophilia A gene remedy.
BioMarin has been implementing a $500 million “price transformation program” to contribute to non-GAAP working margin targets set for 2026 and past.
Goldman Analyst writes, “We additionally see exterior enterprise improvement as a possible supply of upside and diversification past BioMarin’s steering. Nonetheless, we word continued investor debate on the outlook given rising aggressive strain on BioMarin’s key progress driver.”
The analyst maintains a Purchase ranking with a value goal of $139.
Worth Motion: BMRN inventory is down 0.57% at $69.09 on the final verify on Wednesday.
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