文章核心观点 - 公司宣布FDA已经为公司提交的治疗儿童和成人苯丙酮尿症(PKU)的新药申请(NDA)设定了2025年7月29日的目标审查日期 [1] - 公司宣布APHENITY III期临床试验结果已在权威医学期刊《柳叶刀》上发表 [1] - 公司表示APHENITY III期临床试验数据和正在进行的开放标签延长研究结果显示,该药物具有持久疗效,可以让患者放宽饮食限制并仍能控制血液中苯丙氨酸水平 [2] 药物概况 - 该药物(sepiapterin)是一种口服合成sepiapterin制剂,通过双重机制作用提高苯丙氨酸羟化酶(PAH)酶活性,从而降低血液中苯丙氨酸水平,有潜力治疗各种类型的PKU患者 [3] 疾病概况 - 苯丙酮尿症(PKU)是一种罕见的遗传代谢性疾病,会影响大脑,是由帮助分解苯丙氨酸的酶缺陷引起的 - 如果不及时治疗或管理不善,体内苯丙氨酸会积累到危险水平,导致严重不可逆的并发症,如永久性智力障碍、癫痫、发育迟缓、记忆力下降、行为和情绪问题 - 全球估计有58,000人患有PKU [4] 公司概况 - 公司是一家专注于罕见疾病领域的全球生物制药公司,致力于发现、开发和商业化具有临床差异化的药物,为患有罕见疾病的儿童和成人带来益处 [5] - 公司的创新能力、全球商业化能力是支撑其丰富多样的转化性药物管线的基础 [5] - 公司的使命是为缺乏治疗选择的患者提供最佳治疗 [5]

- Statistically significant results on primary endpoint of long-term study analyses - - Following FDA pre-NDA feedback, submission on track for December 2024 - WARREN, N.J., Oct. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) provided today several positive updates on the vatiquinone Friedreich ataxia (FA) program. The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progress ...

WARREN, N.J., Oct. 1, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of pediatric and adult patients living with phenylketonuria (PKU). A Prescription Drug User Fee Act (PDUFA) target action date is expected to be provided in the Day 74 Letter. "The FDA filing acceptance for sepiapterin is a critical milestone toward bringing this potential important therapy to children and adults livi ...

Collaboration to Drive PTC's Onshape Initiatives Including Discovery Program, Onshape AI Advisor, and CAD and PDM Conversion Tools BOSTON, Sept. 25, 2024 /PRNewswire/ -- PTC (NASDAQ: PTC) today announced entry into a Strategic Collaboration Agreement (SCA) with Amazon Web Services (AWS) to accelerate the growth of its Onshape® cloud-native computer-aided design (CAD) and product data management (PDM) solution. This collaboration will focus on advancing Onshape product enhancements, customer adoption program ...

文章核心观点 - Roblox和PTC是两家表现不佳但仍有巨大增长潜力的公司 [1][2] - Roblox的用户数、营收和订阅量均呈现强劲增长 [3] - Roblox正在逐步发展其数字广告业务,并且用户群正在逐渐变老,这对广告主来说是有利因素 [4] - PTC的年度经常性收入(ARR)保持低双位数增长,这反映了其软件解决方案的持续需求 [5] - PTC可以利用其产品生命周期管理(PLM)、服务生命周期管理(SLM)等软件解决方案实现交叉销售 [6][7] - 尽管受经济环境影响,但PTC的ARR增长仍然可观,有望在经济好转时进一步提升 [7] Roblox公司分析 - Roblox的用户数、营收和订阅量均呈现强劲增长,用户群正在逐渐变老,这对广告主来说是有利因素 [3][4] - Roblox的在线游戏和社交平台已经证明具有持久性,其增长故事仍处于初期阶段 [4] PTC公司分析 - PTC的年度经常性收入(ARR)保持低双位数增长,反映了其软件解决方案的持续需求 [5] - PTC可以利用其产品生命周期管理(PLM)、服务生命周期管理(SLM)等软件解决方案实现交叉销售 [6][7] - 尽管受经济环境影响,但PTC的ARR增长仍然可观,有望在经济好转时进一步提升 [7]

SolStock PTC Therapeutics, Inc. (NASDAQ:PTCT) continues to make significant progress in its pipeline. This is especially true when you consider that it has already been able to report positive interim results from its phase 2 PIVOT-HD study, which used PTC518 for the treatment of patients with Huntington's Disease [HD]. The last time I wrote about this article in a Seeking Alpha article entitled "PTC Therapeutics: Multiple Regulatory Submissions Along With Data Cuts," data for PTC518 was not released. Not o ...

BOSTON, Sept. 3, 2024 /PRNewswire/ -- PTC (NASDAQ: PTC) today announced that it has appointed Rob Bernshteyn to its Board of Directors, effective September 9, 2024. Bernshteyn has held leadership and executive roles in the enterprise software space for over 30 years, including 14 years as CEO and Chairman of Coupa Software, a provider of business spend management solutions. During his tenure at Coupa, Bernshteyn transformed the company into a high-growth industry leader and led a successful IPO in 2016. He ...

It has been about a month since the last earnings report for PTC Inc. (PTC) . Shares have added about 0.7% in that time frame, underperforming the S&P 500. Will the recent positive trend continue leading up to its next earnings release, or is PTC Inc. due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at its most recent earnings report in order to get a better handle on the important drivers. PTC's Q3 Earnings Meet Estimates PTC reported third ...

WARREN, N.J., Aug. 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the company will present a company overview at the following conferences: Morgan Stanley 22nd Annual Global Healthcare Conference Thursday, Sept. 5 at 9:15 am ET 2024 Wells Fargo Healthcare Conference Friday, Sept. 6 at 12:00 pm ET Cantor 2024 Global Healthcare Conference Tuesday, Sept. 17 at 2:30 pm ET The presentations will be webcast live on the Events and Presentations page under the Investor section o ...

BOSTON, Aug. 27, 2024 /PRNewswire/ -- PTC (Nasdaq: PTC) today announced that it will participate in the following conferences. What: Citi's 2024 Global TMT Conference When: Wednesday, September 4th, 2024 at 8:20am ET What: 2024 RBCCM Global TIMT Conference When: Tuesday, November 19th, 2024 What: Barclays 22nd Annual Global Technology Conference When: Wednesday, December 11th, 2024 What: Nasdaq 51st Investor Conference When: Wednesday, December 11th, 2024 To view the webcast and replay for conferences pleas ...