Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is among the most promising biotech stocks to buy according to hedge funds. According to a recent disclosure with the SEC, Assenagon Asset Management S.A. lifted its holdings in Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) by a whopping 439.0% during the second quarter. Following the acquisition of 947,865 shares, the firm now owns 1,163,755 shares of the company’s stock, reflecting an investment worth $42,314,000 and ownership of about 1.23%. During the latest ...

NOVATO, Calif., Sept. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the appointment of Eric Olson as chief business officer (CBO) and executive vice president effective September 22, 2025, following the planned retirement of Thomas Kassberg. Mr. Olson will be responsible for leading the company’s business development, corporate development and alliance management functions. “We thank Tom for his 14 years of impactful contributions to Ultragenyx, including transf ...

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best strong buy stocks to invest in according to Wall Street. In a report released on September 22, Kristen Kluska from Cantor Fitzgerald reiterated a Buy rating on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and set a price target of $105.00. Ultragenyx (RARE) Falls 26% as New Treatment Fails to Get FDA Green Light Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) reported $166 million in total revenue for fiscal Q2 2025, with Crysvita revenue comin ...

Regeneron Pharmaceuticals (NASDAQ:REGN) announced on Friday that the U.S. FDA approved a label expansion for its anti-cholesterol agent Evkeeza, marketed globally with Ultragenyx Pharmaceuticals (NASDAQ:RARE). Accordingly, the injectable, first approved in 2021 for adults and adolescents with homozygous familial hypercholesterolemia ((HoFH)), will also be ...

PresentationTazeen AhmadBofA Securities, Research Division Good morning to everybody listening in. Welcome to the Bank of America Healthcare Conference. I am Tazeen Ahmad, one of the senior SMID biotech analyst. It is my pleasure to have with me our next presenting company, Ultragenyx. Presenting for Ultragenyx and sitting with me is Eric Crombez, who is Chief Medical Officer. Eric, thanks for making the trip over from the -- [indiscernible] not as far. ...

Sands Capital, an investment management company, released its “Sands Capital Select Growth Strategy” Q2 2025 investor letter. A copy of the letter can be downloaded here.  U.S. large-cap growth equities recovered from a sharp sell-off in early April by delivering strong returns in the second quarter. The quarterly performance was driven by muted inflation, strong corporate earnings, and improving sentiment around artificial intelligence (AI) and global trade. The portfolio returned 27.7% in the quarter, out ...

PresentationGreat. Thank you, everyone, for joining. I'm Max Skor, biotech analysts with Morgan Stanley. And I'm happy to be hosting this session with Ultragenyx, Eric Crombez, Chief Medical Officer. Thank you very much for joining us today. And I just wanted to briefly touch on important disclosures. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales repre ...

Key Takeaways RARE's DTX401 showed a lasting 61% mean reduction in daily cornstarch needs at week 96 in GSDIa patients.Patients reported fewer hypoglycemic events, better fasting tolerance, and higher quality of life.RARE has begun rolling BLA submission for DTX401, with completion expected later this year.Ultragenyx Pharmaceutical (RARE) reported positive longer-term data from a late-stage study of its AAV gene therapy, DTX401, for the treatment of glycogen storage disease type Ia (GSDIa). GSDIa is a rare, ...

核心观点 - Ultragenyx Pharmaceutical Inc 公布 DTX401 治疗糖原累积症 Ia 型 (GSDIa) 的 96 周 III 期临床试验结果 显示每日玉米淀粉摄入量持续显著减少 同时血糖控制得到改善 安全性和耐受性良好 [2][4][9] 临床试验结果 - 第 96 周时 DTX401 治疗组 (n=20) 和交叉组 (n=19) 的每日玉米淀粉摄入量较基线平均减少 61% [4] - 夜间玉米淀粉摄入量减少更为显著 DTX401 组平均减少 70% 交叉组平均减少 75% 三分之二参与者至少取消一次夜间剂量 [5] - 尽管玉米淀粉摄入大幅减少 参与者低血糖水平维持低位 正常血糖范围 (70-120 mg/dL) 改善 空腹耐受性提高 严重低血糖 (< 54 mg/dL) 得到防护 [1][5] - 日本开放标签队列 (n=3) 的早期数据显示 第 24 周时每日玉米淀粉摄入量平均减少 95% 所有参与者最终在第 24 周 (n=2) 和第 36 周 (n=1) 完全停止每日玉米淀粉摄入 [10] 患者生活质量改善 - 根据患者整体印象变化 (PGIC) 量表 83% 的 DTX401 组和 95% 的交叉组报告疾病负担改善 (+1 至 +3 变化) [7] - 参与者访谈显示 治疗最常报告的功能改善包括身体、社交和饮食/日常 regimen 影响方面 同时低血糖和疲劳感减少 [8] - 减少夜间玉米淀粉剂量可带来 uninterrupted sleep 从而改善患者及其家庭的生活质量和减轻疲劳 [6] 安全性与耐受性 - DTX401 表现出可接受且预期的安全性特征 与 I/II 期研究结果一致 [9] - 预期的肝脏反应可通过预防性皮质类固醇方案管理 未观察到 AAV8 类别的背根神经节毒性、恶性肿瘤或血栓性微血管病 [9] - 所有研究组均观察到高甘油三酯血症 但 DTX401 组更常见 [9] 研究设计与药物背景 - III 期 GlucoGene 研究是一项 48 周随机、双盲、安慰剂对照研究 共治疗 46 名 8 岁及以上参与者 DTX401 剂量为 1.0 x 10^13 GC/kg (通过 ddPCR 测量) [11] - 修改的意向治疗 (mITT) 人群包括 44 名参与者 提供 DTX401 (n=20) 或安慰剂 (n=24) 治疗后的 48 周内疗效数据 [11] - DTX401 是一种研究性 AAV8 基因疗法 旨在提供 G6Pase 的稳定表达和活性 使治疗的肝细胞能够响应管理葡萄糖的正常激素信号 [12] - GSDIa 是一种罕见、严重且危及生命的疾病 由 G6PC 基因致病性变异引起 估计在商业可及地区影响约 6,000 人 [13]

公司业务定位 - 专注于罕见病领域 致力于开发变革性治疗方法 此前该领域缺乏有效治疗方案 [2] - 公司成立15年 正处于关键发展阶段 目标在2027年实现全年GAAP盈利 [2] 产品管线布局 - 拥有4个商业化项目 收入呈现显著增长趋势 [2] - 近期计划推出3个重要产品 包括2个基因疗法项目(401和111)以及骨骼发育不全治疗项目(143) [3] - 安格曼综合征项目受到市场广泛关注 但未包含在近期上市计划中 [3] 核心治疗领域 - 基因治疗为公司重点发展方向 拥有多个基因疗法管线 [3] - 治疗领域覆盖遗传性代谢疾病和神经发育障碍等罕见病 [2][3]