BioMarin Restructures, Outlines Roadmap To $4B Revenues By 2027
BioMarin Restructures, Outlines Roadmap To $4B Revenues By 2027
BioMarin Pharmaceutical Inc (NASDAQ:BMRN) plans to land $4 billion in revenue by 2027.
BioMarin Pharmaceutical Inc（納斯達克：BMRN）計劃到2027年實現400億美元的營業收入。
The company is targeting an adjusted operating margin of low-to-mid 40%'s (starting at 40% in 2026).
該公司以40%（從2026年起）低至中等調整後的營業利潤率爲目標。
The new structure, announced at an investor event Wednesday, covers three key business units. They include skeletal conditions, enzyme therapies, and Roctavian — its hemophilia A gene therapy.
新架構在週三的投資者活動中公佈，涵蓋了三個關鍵業務部門。它們包括骨骼狀況、酶療法和Roctavian - 血友病A基因治療。
BioMarin has been implementing a $500 million "cost transformation program" to contribute to non-GAAP operating margin targets set for 2026 and beyond.
BioMarin一直在實施一項5000萬美元的「成本轉型計劃」，以支持2026年及以後的非GAAP營業利潤率目標。
CEO Alexander Hardy highlighted five experimental programs through 2031 for Voxzogo beyond achondroplasia, a type of genetic condition that causes dwarfism.
首席執行官Alexander Hardy強調了Beyond achondroplasia的五個實驗計劃，該病屬於引起侏儒症的一種遺傳狀況。
Voxzogo sales reached $470 million in 2023. According to BioMarin, the drug could prove a $5 billion opportunity with a total addressable patient population of around 420,000 across its global markets.
Voxzogo銷售額在2023年達到4.7億美元。根據BioMarin的說法，該藥物在全球市場的總可尋址患者人口約爲42萬，可能是一個50億美元的機會。
The next most advanced Voxzogo program is in hypochondroplasia, another form of dwarfism. Phase 3 data readouts are expected in 2026, and a targeted launch is expected in 2027.
下一個最先進的Voxzogo項目是hypochondroplasia，這是另一種侏儒症形式。預計2026年將獲得3期數據，並計劃在2027年推出。
BioMarin plans to launch two products outside its approved medicines by 2027 or 11 by 2034. The company's pipeline includes programs for diseases such as Duchenne muscular dystrophy, other forms of dwarfism, and von Willebrand disease.
BioMarin計劃到2027年或2034年推出11個產品。該公司的產品線包括用於諸如Duchenne肌營養不良症、不同形式的侏儒症和von Willebrand病等疾病的項目。
Recently, BioMarin updated its Roctavian business to focus commercial operations on three markets: the U.S., Germany, and Italy.
最近，BioMarin更新了其Roctavian業務，將商業運營重點放在美國、德國和意大利三個市場上。
These markets are where the medicine is approved and reimbursed as a severe hemophilia A treatment.
這些市場是該藥物被批准和報銷爲重型血友病A治療的地區。
The company will adjust its Roctavian efforts and reduce additional investments in development and manufacturing.
該公司將調整Roctavian的工作，並減少在開發和製造方面的額外投資。
BioMarin anticipates reducing annual direct Roctavian expenses to approximately $60 million beginning in 2025.
BioMarin預計從2025年開始，每年將Roctavian的直接費用降低到約6000萬美元。
In July, the FDA approved BioMarin's Brineura (cerliponase alfa) to slow the loss of ambulation in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
7月份，FDA批准BioMarin的Brineura（塞利泊瑞酶α）用於減緩一切年齡患有神經細胞溶脂蛋白沉積症2型（CLN2疾病）的孩子失去行動能力的情況，也被稱爲三肽酶1（TPP1）缺乏症。
Previously, Brineura was indicated in symptomatic children three years of age and older with late infantile CLN2 disease. This expanded indication now includes children of all ages with CLN2 disease, regardless of whether they are symptomatic or presymptomatic.
先前，Brineura被指示用於有症狀的三歲及以上晚期嬰幼兒CLN2疾病的孩子。這個擴大的適應症現在包括所有年齡段的CLN2疾病兒童，無論他們是否有症狀或潛在症狀。
William Blair says, "However, BioMarin has a rich pipeline, only three programs could potentially contribute to its top line in the next three years, which includes label expansion of Palynziqinto adolescents and Voxzogo into HCH. Therefore, we think commercial execution and continued differentiation from competitors will continue to drive the stock in the near term."
William Blair表示：「然而，BioMarin擁有豐富的項目線路圖，只有三個項目在未來三年內有可能對其收入產生貢獻，其中包括將Palynziq擴大到青少年和將Voxzogo擴大到HCH的標籤擴展。因此，我們認爲商業執行和與競爭對手的持續差異化將繼續推動該股票在短期內的發展。」
Price Action: BMRN stock closed at $89.86 on Wednesday.
股價變動：BMRN股票週三收盤價爲89.86美元。
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譯文內容由第三人軟體翻譯。