業種
Biopharmaceuticals & Rare Diseases
Biopharmaceuticals & Rare Diseases のすべてのレポート — 全 3 件。
38/100
Rocket Pharmaceuticals: Cash Buys Time, but Danon Still Has to Earn Back the Market's Trust
Rocket Pharmaceuticals (RCKT.US) is a rare-disease gene-therapy developer whose sole approved product, Kresladi, still generates no revenue, leaving cash and a $180 million voucher sale as the real near-term story. The stock trades near the value of its cash alone because the market still distrusts the pivotal Danon cardiac program after a 2025 clinical hold, even though Phase 1 data and a modified lower-dose cohort keep the thesis alive. Rating Hold: cash buys time, but Danon still has to earn back trust and Kresladi is too small to carry the valuation on its own.
45/100
BioMarin Pharmaceutical: A Rare-Disease Cash Engine in Transition
BioMarin is a profitable rare-disease biopharma whose enzyme franchise generated about 2.106 billion USD of 2025 revenue and funds the company, while VOXZOGO (926.9 million USD) drives both growth and sentiment. The Amicus acquisition diversified the portfolio into Fabry and Pompe disease and lifted 2026 revenue guidance to 3.825-3.925 billion USD, yet weekly and oral achondroplasia rivals from Ascendis and BridgeBio keep the margin of safety thin. Rating Hold: cash generation and Amicus diversification make BioMarin sturdier than sentiment implies, but VOXZOGO competition caps the upside at today's price.
55/100
38Buffett
argenx（ARGX.US / ARGX.BR）：Zen Horizon レポート
argenx は希少自己免疫疾患の抗体医薬で世界をリードし、FcRn 阻害薬という領域を最初に切り開いたファーストインクラス企業である。中核製品 Vyvgart（efgartigimod）はすでに3適応症（gMG と CIDP が全世界、ITP は日本）で承認され、投与患者は約19000人、FY2025 の製品売上高は41.5億ドル（+90%）、営業利益は初の黒字となる10.5億ドルを計上した。Vision 2030 は患者50000人・ラベル10件・第III相分子5件を掲げる。レーティングはウォッチ：資産としての質は本物だが、882.41ドルという株価はすでに順調な実行を織り込んでおり、720ドル以下への調整が出て初めてエントリーの窓が開く。