산업
Biopharmaceuticals & Rare Diseases
Biopharmaceuticals & Rare Diseases 산업의 모든 리포트 — 총 3건.
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 리포트
argenx는 희귀 자가면역질환 항체 치료제 분야의 글로벌 선두주자이자 FcRn 억제제 영역을 개척한 first-in-class 기업이다. 핵심 제품 Vyvgart(efgartigimod)는 현재 3개 적응증(gMG, 글로벌 CIDP, 일본 ITP)에서 승인을 받았고 치료 환자 수는 약 19000명, FY2025 순매출은 41.5억 달러(+90%), 영업이익은 사상 첫 흑자인 10.5억 달러를 기록했다. Vision 2030은 환자 50000명, 적응증 라벨 10개, 임상 3상 분자 5개를 목표로 한다. 투자의견 관찰: 자산의 질은 확실히 우수하지만 882.41달러의 주가에 이미 순조로운 실행이 반영되어 있어, 진입 구간은 720달러 이하로 조정될 때 비로소 열린다.