The most talked about and market moving research calls around Wall Street are now in one place. Here are today's research calls that investors need to know, as compiled by The Fly.Top 5 Upgrades: Rothschild & Co Redburn upgraded Kenvue (KVUE) to Buy from Neutral with a price target of $22, down from $22.50. The shares are down over 20% this month with the Trump administering linking usage of Tylenol by expectant mothers with an increased risk of autism in infants, but the firm views proposals laid out th ...

Key Takeaways Ionis' phase III study showed zilganersen stabilized gait speed in Alexander disease patients.The drug demonstrated favorable safety and consistent benefit across secondary measures.Ionis plans an FDA filing in Q1 2026 as it expands its wholly owned medicine portfolio.Ionis Pharmaceuticals (IONS) announced positive topline results from a pivotal phase III study evaluating its investigational RNA-targeted therapy zilganersen for treating a rare neurological condition called Alexander disease (A ...

Ionis Pharmaceuticals (NASDAQ:IONS) has reported promising results from a pivotal study of its experimental medicine, zilganersen, for the treatment of Alexander disease, a rare and progressive neurological disorder. The study found that a 50 mg dose of zilganersen significantly improved ...

Ionis Pharmaceuticals, Inc. IONS released topline results on Monday from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive, and often fatal neurological condition.The neurological disorder destroys the myelin sheath protecting nerve fibers, caused by a mutation in the GFAP gene.Zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization on the primary endpoint of gait speed as assessed by the 10-Meter Walk Te ...

Ionis Pharmaceuticals said on Monday its experimental brain disorder drug met the main goal in an early-to-late-stage study. ...

KalVista Pharmaceuticals (NASDAQ:KALV) and Ionis Pharmaceuticals (NASDAQ:IONS) announced on Friday that the European Commission has granted final approval for two of their treatments targeting rare genetic disorders. Cambridge, Massachusetts-based KalVista (NASDAQ:KALV) said that the EC and Swiss medical regulator, the ...

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Sobi® today announced that TRYNGOLZA® (olezarsen) has been approved in the European Union (EU) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). The approval follows the positive opinion of the Committee for Medicinal Products for Human Use. The approval is based on positive data from the Phase 3 Balance study, in which TRYNGOLZA 80 mg demon. ...

核心观点 - 多只生物技术股票上周创52周新高 均在过去数月内实现显著涨幅 主要驱动因素包括旗舰产品商业化进展、监管催化剂和临床开发里程碑 [1][3][5][8][11][15][17][21] Aurinia Pharmaceuticals Inc (AUPH) - 旗舰产品LUPKYNIS是FDA首个批准用于活动性狼疮肾炎成人患者的口服疗法 2021年上市后持续稳健增长 [1][2] - 2025年预计产品净销售额2.5-2.6亿美元 较去年2.162亿美元增长16-20% [2] - 开发中的Aritinercept是BAFF和APRIL双抑制剂 用于自身免疫性疾病 预计2024年下半年启动至少两项临床研究 [3] - 股价从2024年4月19日4.79美元涨至上周52周高点12.87美元 1.5年内涨幅达168% [3] Arrowhead Pharmaceuticals Inc (ARWR) - RNAi治疗平台公司 首个商业化产品Plozasiran（用于家族性乳糜微粒血症综合征）正处于FDA审查中 预计2025年11月18日做出决定 [4] - 家族性乳糜微粒血症综合征是超罕见遗传病 特征为血液甘油三酯水平通常超过880 mg/dL 胰腺炎风险极高 [5] - 与Ionis Pharmaceuticals存在专利诉讼纠纷 涉及Plozasiran涉嫌侵权 [6] - 股价从2024年12月30日18.86美元涨至上周52周高点31.13美元 9个月内涨幅达65% [5] Beta Bionics Inc (BBNX) - 医疗设备公司 开发糖尿病管理解决方案 第二季度净销售额2320万美元 同比增长54% [7][8] - 2025年预计净销售额8800-9300万美元 较去年6510万美元增长35-43% [8] - 旗舰产品iLet Bionic Pancreas是胰岛素输送系统 可自动调节6岁及以上1型糖尿病患者的血糖水平 [7] - 股价从2025年6月2日17.24美元涨至上周52周高点26.66美元 4个月内涨幅近55% [8] Kiniksa Pharmaceuticals International plc (KNSA) - 旗舰产品Arcalyst是FDA首个且唯一批准用于复发性心包炎的疗法 新开处方和重复处方量均增长 [10][11] - 2025年预计产品净收入6.25-6.4亿美元 较去年4.17亿美元增长50-53% [11] - 股价从2025年4月29日25.53美元涨至上周52周高点37.34美元 4个月内涨幅达46% [11] Ionis Pharmaceuticals Inc (IONS) - 旗舰药物Tryngolza于2024年12月获FDA批准用于家族性乳糜微粒血症综合征 正在探索严重高甘油三酯血症适应症 预计2025年底前向FDA提交补充新药申请 [12] - Tryngolza在2025年第二季度实现净销售额1900万美元 上半年总计2600万美元 [12] - 第二款药物DAWNZERA于上月获FDA批准 用于预防12岁及以上遗传性血管性水肿发作 [13] - 与Arrowhead存在相互专利诉讼 涉及Tryngolza专利有效性及Plozasiran侵权指控 [14] - 股价从2025年7月15日42.55美元涨至上周52周高点64.72美元 2个月内涨幅达52% [15] Stoke Therapeutics Inc (STOK) - 开发治疗Dravet综合征的潜在疾病修饰药物Zorevunersen 上周创52周高点24.60美元 [16][17] - Dravet综合征是严重发育性和癫痫性脑病 特征为严重反复发作的癫痫以及显著认知和行为障碍 [16] - 全球III期EMPEROR试验正在进行中 预计2027年下半年获得数据 与Biogen合作开发 [17] - 股价从2025年7月22日13.50美元涨至上周52周高点24.60美元 2个月内涨幅达82% [17] Fortress Biotech Inc (FBIO) - 等待子公司Cyprium Therapeutics研发药物CUTX-101的监管决定 FDA预计于2025年9月30日做出决定 [18] - CUTX-101是皮下注射铜组氨酸盐制剂 用于治疗罕见X连锁隐性儿科疾病Menkes病 [18] - 通过产品收入、股权持有、股息和特许权使用费流增强股东长期价值 拥有9家子公司和合作伙伴公司从事生物制药开发和商业化 [19][20] - 股价从2025年8月29日2.46美元涨至上周52周高点3.97美元 1个月内涨幅达61% [21]

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today celebrated 10 years of Surf Away+, an inclusive adaptive event for rare neurological disease communities. Originally created with the goal of providing people with spinal muscular atrophy (SMA) access to adaptive surfing, the event has expanded to include a myriad of adaptive experiences and will welcome additional communities impacted by neurological diseases, with more than 200 people from the Alexander diseas. ...

文章核心观点 - 作者长期关注Ionis Pharmaceuticals公司 过去多年已撰写12篇相关文章记录该公司发展历程 [1] - 投资被视为学习过程 投资失败是学费支出 成功经验则成为知识积累 [1] - 作者分享数十年投资经验及约5年针对多只股票的深入研究 近年重点关注医疗健康类股票 [1] 作者持仓披露 - 作者通过股票持有、期权或其他衍生品对IONS和AQST持有多头仓位 [2] - 文章表达作者个人观点 未获得除Seeking Alpha外任何补偿 [2] - 作者与所提及公司无商业关系 但可能在72小时内买入提及公司股票 [2] 平台免责声明 - Seeking Alpha声明过往业绩不保证未来结果 不提供投资建议 [3] - 发表观点不代表Seeking Alpha整体意见 平台非持牌证券交易商或投资顾问 [3] - 分析师团队包含专业投资者和个体投资者 未必持有专业资质认证 [3]