TARA-002

Reported interim data from ADVANCED-2 trial in non-muscle invasive bladder cancer (NMIBC); TARA-002 demonstrated 68% complete response rate at six months in BCG-Unresponsive patients Company expects to complete enrollment of the BCG-Unresponsive registrational cohort of the ADVANCED-2 trial in 2H 2026 On track to initiate the ADVANCED-3 registrational trial in BCG-Naïve patients in 2H 2026 Received Breakthrough Therapy and Fast Track d esignations for TARA-002 in lymphatic malformations (LMs); regulatory update expected in 1H 2026 Expect to report interim results from THRIVE-3 registrational trial of IV Choline Chloride in patients dependent on long-term parenteral support in 2H 2026 Completed oversubscribed $86 million public offering with participation from new and existing investors Cash, cash equivalents and investments of approximately $198 million as of December 31, 2025, expected to support planned operations into 2028 NEW YORK, March 10, 2026 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage biotechnology company developing transformative therapies for the treatment of cancer and rare diseases, today provided a business update and announced financial results for the fourth quarter and full year ended December 31, 2025. “Over the last few months, we reported meaningful clinical and regulatory progress across our portfolio of late-stage programs and are building on this momentum as we advance toward several important milestones in 2026,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “For TARA-002 in non-muscle invasive bladder cancer (NMIBC), the clinical dataset continues to support its potential as a differentiated intravesical therapy with compelling efficacy, a favorable safety pro a streamlined approach to administration in the clinical setting for both Bacillus Calmette-Guérin (BCG)-Unresponsive and BCG-Naïve patients. We remain on track to complete enrollment in our BCG-Unresponsive registrational cohort in the ADVANCED-2 trial and to initiate the ADVANCED-3 registrational trial in BCG-Naïve patients in the second half of 2026.” Mr. Shefferman added, “For our rare disease programs, we reported positive interim results for TARA-002 in lymphatic malformations (LMs), followed by the receipt of Breakthrough Therapy and Fast Track designations for TARA-002 in LMs from the U.S. Food and Drug Administration (FDA). We are working with the FDA to bring this promising treatment to patients expeditiously and expect to provide a regulatory update in the first half of 2026. In addition, our THRIVE-3 registrational trial of intravenous (IV) Choline Chloride for patients on parenteral support remains on track, with interim results expected in the second half of 2026. With cash runway into 2028, we are well positioned to continue executing across our pipeline and advance our mission of delivering transformational therapies for patients with cancer and rare diseases.” Recent Progress and Highlights TARA-002 in NMIBC At the ASCO Genitourinary Cancers Symposium in February 2026, the Company announced updated interim results from the ADVANCED-2 trial in evaluable NMIBC patients with carcinoma in situ or CIS (± Ta/T1) who are BCG-Unresponsive and BCG-Naïve. As of the January 28, 2026 data cutoff: In the BCG-Unresponsive cohort of patients, TARA-002 demonstrated a complete response (CR) rate at any time of 65.7% (23/35) with CR rates of 68.2% (15/22) at six months and 33.3% (5/12) at 12 months. Among responders, the Kaplan-Meier (KM) estimated probability of maintaining a CR for six months was 71.1% (95% CI: 46.7, 95.5). Additionally, among responders, 100% (5 of 5) maintained their CR from nine to 12 months. Re-induction therapy was successful with 61.5% (8 of 13) of re-induced patients converting to a CR at six months. In the BCG-Naïve cohort of patients, TARA-002 demonstrated a CR rate at any time of 72.4% (21/29) with CR rates of 66.7% (18 of 27) at six months and 57.9% (11 of 19) at 12 months. Among responders, the KM estimated probability of maintaining a CR for six months was 73.1% (95% CI: 52.9, 93.4). In addition, among responders, 100% (11 of 11) maintained their CR from nine to 12 months. Re-induction therapy was successful with 66.7% (4 of 6) of re-induced patients converting to a CR at six months. The majority of treatment-related adverse events (TRAEs) were Grade 1 and transient with no Grade 3 or greater TRAEs and no related serious adverse events as assessed by study investigators. No patients discontinued treatment due to TRAEs. The most common TRAEs were dysuria, bladder spasm, fatigue and micturition urgency. Most bladder irritations resolved shortly after administration or within a few hours to a few days. The Company has completed enrollment of the BCG-Naïve cohort and expects to complete enrollment of the BCG-Unresponsive cohort in the second half of 2026. The Company remains on track to commence ADVANCED-3, a registrational trial of TARA-002 compared to intravesical chemotherapy in BCG-Naïve patients (who have never been exposed and those who have not received BCG within the last 24 months and are ineligible to receive BCG, have a contraindication to BCG, cannot tolerate BCG, do not have access to BCG or refuse BCG) in the second half of 2026. TARA-002 in LMs In January 2026, the Company announced that the FDA granted TARA-002 Fast Track and Breakthrough Therapy designations for the treatment of pediatric patients with macrocystic and mixed cystic LMs. TARA-002 was previously granted Rare Pediatric Disease designation for the treatment of LMs. The Company remains on track to share a regulatory update on the registrational path for TARA-002 in LMs in the first half of 2026. IV Choline Chloride for Patients on Parenteral Support (PS) THRIVE-3, the Company’s registrational Phase 3 clinical trial, is ongoing, and the Company expects to report interim results in the second half of 2026. Corporate Update Fourth Quarter and Full Year 2025 Financial Results As of December 31, 2025, unrestricted cash and cash equivalents and marketable debt securities totaled $197.9 million, including proceeds from the Company’s $86.3 million public offering in December 2025. The Company expects its cash and cash equivalents and marketable debt securities will be sufficient to fund its planned operations and milestones into 2028. Research and development expenses for the fourth quarter of 2025 increased to $13.1 million from $9.5 million for the prior year period, and for the full year increased to $42.6 million compared to $31.7 million for 2024. The fourth quarter and full year increases were primarily due to the start-up and advancement of clinical trials across our portfolio. General and administrative expenses for the fourth quarter of 2025 increased to $6.0 million from $4.8 million for the prior year period, and for the full year increased to $21.9 million compared to $17.5 million for 2024. The fourth quarter and full year increases were primarily due to increases in personnel-related expenses and other general and administrative expenses primarily related to professional and consulting services. For the fourth quarter of 2025, Protara incurred a net loss of $17.3 million, or $0.37 per share, compared with a net loss of $12.8 million, or $0.48 per share, for the same period in 2024. Net loss for the year ended December 31, 2025 was $57.4 million, or $1.34 per share, compared with a net loss of $44.6 million, or $2.17 per share, for the year ended December 31, 2024. Net loss for the fourth quarter of 2025 included approximately $1.0 million in stock-based compensation expenses. Net loss for the year ended December 31, 2025 included approximately $3.8 million in stock-based compensation expenses. About ADVANCED-2 ADVANCED-2 ( NCT05951179 ) is a Phase 2 open-label trial assessing intravesical TARA-002 in non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ or CIS (± Ta/T1) who are Bacillus Calmette-Guérin (BCG)-Unresponsive (Cohort B N=75-100) or BCG-Naïve (Cohort A N=31). Trial subjects received an induction course, with or without a reinduction, of six weekly intravesical instillations of TARA-002, followed by a maintenance course of three weekly instillations every three months. The BCG-Unresponsive cohort is designed to be registrational based on the FDA’s August 2024 Draft Guidance for Industry on BCG-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment. About TARA-002 TARA-002 is an investigational cell therapy in development for the treatment of NMIBC and of LMs, for which it has been granted Rare Pediatric Disease, Breakthrough and Fast Track designations by the FDA. TARA-002 is a first-in-class TLR2/NOD2 agonist and novel immunopotentiator derived from inactivated Streptococcus pyogenes with a mechanism of action that includes the activation of innate and adaptive immune pathways within the bladder wall. When TARA-002 is administered, it is hypothesized that innate and adaptive immune cells within the cyst or tumor are activated and produce a pro-inflammatory response with the release of cytokines such as tumor necrosis factor (TNF)-alpha, interferon (IFN)-gamma, IL-6, IL-10 and IL-12. TARA-002 also directly kills tumor cells and triggers a host immune response by inducing immunogenic cell death, which further enhances the antitumor immune response. TARA-002 was developed from the same master cell bank of genetically distinct group A Streptococcus pyogenes as OK-432, a broad immunopotentiator marketed as Picibanil ® in Japan by Chugai Pharmaceutical Co., Ltd. Protara has successfully shown manufacturing comparability between TARA-002 and OK-432. About Non-Muscle Invasive Bladder Cancer Bladder cancer is the sixth most common cancer in the United States, with non-muscle invasive bladder cancer (NMIBC) representing approximately 80% of bladder cancer diagnoses, or approximately 65,000 patients in the U.S. each year. NMIBC is cancer found in the tissue that lines the inner surface of the bladder that has not spread into the bladder muscle. About Lymphatic Malformations Lymphatic Malformations (LMs) are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Protara’s focus is on macrocystic and mixed cystic LMs, for which there are no currently approved therapies. They are most frequently present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels and lymphatics; recurrent infection; and cosmetic and other functional disabilities. TARA-002 has been granted Rare Pediatric Disease, Breakthrough Therapy and Fast Track designations by the FDA for the treatment of LMs. About IV Choline Chloride for Patients on Parenteral Support IV Choline Chloride is an investigational, intravenous phospholipid substrate replacement therapy in development for patients receiving parenteral support (PS). Choline is a known important substrate for phospholipids that are critical for healthy liver function and play an important role in modulating gene expression, cell membrane signaling, brain development and neurotransmission, muscle function and bone health. PS patients are unable to synthesize choline from enteral nutrition sources, and there are currently no available PS formulations containing choline. Approximately 78% of patients dependent on PS are choline-deficient and of those approximately 63% have some degree of liver dysfunction, which can lead to hepatic failure. Every year in the U.S. there are approximately 90,000 people who require PS at home and of those approximately 30,000 are on long-term PS. IV Choline Chloride has the potential to become the first FDA approved IV choline formulation for PS patients. It has been granted Orphan Drug designation by the FDA for the prevention and/or treatment of choline deficiency in patients on long-term parenteral nutrition and has been granted Fast Track designation as a source of choline when oral or enteral nutrition is not possible, insufficient or contraindicated. The U.S. Patent and Trademark Office has issued Protara a U.S. patent claiming a choline composition and a U.S. patent claiming a method for treating choline deficiency with a choline composition, each with a term expiring in 2041. About Protara Therapeutics, Inc. Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin, as well as a Phase 2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral support who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit . Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials (including the timing of any particular phases of such trials and the timing of the announcement of any data produced during such trials or phases thereof); statements related to expectations regarding interactions with the U.S. Food and Drug Administration (FDA); Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year and future periods. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law. Protara Therapeutics, Inc. Consolidated Balance Sheets (in thousands, except share and per share data) December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 49,657 $ 162,798 Marketable debt securities 105,897 7,494 Prepaid expenses and other current assets 3,950 1,863 Total current assets 159,504 172,155 Restricted cash, non-current 745 745 Marketable debt securities, non-current 42,336 - Property and equipment, net 759 1,027 Operating lease right-of-use asset 3,174 4,255 Other assets 2,950 3,272 Total assets $ 209,468 $ 181,454 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 3,468 $ 4,429 Accrued expenses and other current liabilities 6,229 5,408 Operating lease liability 1,242 1,124 Total current liabilities 10,939 10,961 Operating lease liability, non-current 2,117 3,359 Total liabilities 13,056 14,320 Commitments and contingencies Stockholders’ Equity: Preferred stock, $0.001 par value, authorized 10,000,000 shares: Series 1 convertible preferred stock, 8,028 shares authorized at December 31, 2025 and 2024, 5,615 and 7,991 shares issued and outstanding as of December 31, 2025 and 2024, respectively. - - Common stock, $0.001 par value, authorized 100,000,000 shares: Common stock, 53,587,260 and 35,044,772 shares issued and outstanding as of December 31, 2025 and 2024, respectively. 54 35 Additional paid-in capital 498,687 412,077 Accumulated deficit (302,419 ) (244,980 ) Accumulated other comprehensive income (loss) 90 2 Total stockholders’ equity 196,412 167,134 Total liabilities and stockholders’ equity $ 209,468 $ 181,454 PROTARA THERAPEUTICS, INC. AND SUBSIDIARIES Consolidated Statements of Operations and Comprehensive Loss (in thousands, except share and per share data) (Unaudited) (Audited) For the Three Months Ended December 31, For the Years Ended December 31, 2025 2024 2025 2024 Operating expenses: Research and development $ 13,124 $ 9,499 $ 42,633 $ 31,704 General and administrative 5,955 4,813 21,916 17,450 Total operating expenses 19,079 14,312 64,549 49,154 Income (Loss) from operations (19,079 ) (14,312 ) (64,549 ) (49,154 ) Other income (expense), net: Interest and investment income (expense) 1,523 1,156 6,380 4,171 Other income (expense) 249 387 730 387 Other income (expense), net 1,772 1,543 7,110 4,558 Net income (loss) (17,307 ) (12,769 ) (57,439 ) (44,596 ) Other comprehensive income (loss): Net unrealized gain (loss) on marketable debt securities (53 ) (27 ) 88 33 Other comprehensive income (loss): (53 ) (27 ) 88 33 Comprehensive income (loss) $ (17,360 ) $ (12,796 ) $ (57,351 ) $ (44,563 ) Net income (loss) per share attributable to common stockholders, basic and diluted $ (0.37 ) $ (0.48 ) $ (1.34 ) $ (2.17 ) Weighted-average shares outstanding, basic and diluted 46,041,210 26,432,563 42,836,129 20,592,847 Company Contact: Justine O'Malley Protara Therapeutics Justine.OMalley@protaratx.com 646-817-2836

Protara Therapeutics（纳斯达克代码：TARA）高管在 Oppenheimer 第 36 届年度医疗生命科学会议上介绍了他们的临床阶段项目更新。他们强调了 TARA-002 研究在非肌肉侵袭性膀胱癌（NMIBC）中的中期结果，报告六个月时的完全应答率为 68%。该公司还在推进其淋巴畸形项目，并计划与 FDA 进行注册路径的沟通。首席执行官 Jesse Shefferman 强调了 TARA-002 作为 NMIBC 替代治疗的潜力，特别是在 BCG 短缺的情况下Protara Therapeutics NASDAQ: TARA 的高管在 Oppenheimer 第 36 届年度医疗生命科学会议的炉边谈话中概述了公司两个临床阶段项目的进展，强调了其正在进行的非肌肉侵袭性膀胱癌（NMIBC）TARA-002 研究的更新中期结果，并提供了淋巴畸形（LM）监管规划的最新情况。公司还讨论了其针对接受长期肠外营养患者的静脉注射（IV）氯化胆碱项目。 获取 Protara Therapeutics 的提醒： TARA-002：灭活细菌免疫增强剂 首席执行官 Jesse Shefferman 将 TARA-002 描述为一种来自基因上不同的 链球菌 的全细胞细菌治疗，经过制造过程完全灭活，同时保留旨在刺激免疫系统的抗原。他表示，该产品在日本被称为 OK-432，在日本市场已有数十年，是大囊性和混合型淋巴畸形的标准治疗。他还指出，OK-432 在日本有用于某些癌症（头颈癌、甲状腺癌、胃癌和肺癌）化疗辅助治疗的标签，并将其描述为潜在的早期免疫肿瘤学药物。 Shefferman 强调，Protara 正在两个晚期环境中开发 TARA-002： NMIBC，包括针对 BCG 不响应的原位癌（CIS）的注册研究 淋巴畸形，重点关注 STARBORN-1 研究中的大囊性和混合病变 ADVANCED-2 中期结果和耐久性讨论 讨论集中在 ADVANCED-2 的中期数据上，这是 Protara 正在进行的 TARA-002 在 BCG 不响应 NMIBC 中的研究。Gershell 引用了公司报告的中期结果：在 22 名患者中，六个月的 完全反应（CR）率为 68%，在 15 名可评估患者中，12 个月的 CR 率为 33%。他指出，正式的 ASCO GU 演讲定于第二天进行。 在回答关于 12 个月数据的问题时，Shefferman 将较低的 12 个月标志性 CR 率归因于样本量小以及肿瘤学数据集早期可能出现的波动。他表示，在最新的读数之前，公司在 12 个月反应率方面 “一直在中间 40% 的范围内”，但两名待定的患者最终不再反应，导致该比例下降。 Shefferman 表示，该数据集代表了计划中的 100 名患者 研究的前 15 名患者，并表示公司预计随着样本量的增加，12 个月的反应率将回升到 40% 的范围。他还表示，Protara 观察到，之前的 BCG 暴露似乎并不是反应的主要驱动因素，因为在 BCG 初治和 BCG 不响应组中的六个月反应数据变得更具可解释性。 ADVANCED-3：超越 BCG 不响应 CIS 的定位 两位发言人将 BCG 不响应 CIS 描述为注册的途径，而不是公司最大的商业机会。Shefferman 表示，公司在 NMIBC 中的 “大动作” 是 ADVANCED-3，这是一个计划中的随机对照试验，旨在支持在 BCG 不可用或不适合时使用 TARA-002，可能还包括 BCG 暴露或治疗不足的患者，他表示由于 BCG 短缺，这一人群正在增长。 Shefferman 表示，Protara 预计 ADVANCED-2 将在 “2026 年底之前顺利完成招募”，并将该研究的完成视为建立药房准入、报销基础设施和在泌尿科实践中存在的方式，同时推进 ADVANCED-3。 关于未满足的需求，Shefferman 表示，公司向 FDA 提交了七年的 NMIBC 索赔数据，并观察到大约 35% 的本应接受 BCG 的患者未接受 BCG。他估计这一人群每年大约有 11,000 到 15,000 名患者，基于向该机构提交的索赔数据，引用了患者拒绝和 BCG 不耐受等因素。他表示，FDA 对 ADVANCED-3 设计的接受是基于与膀胱内化疗选项（吉西他滨 或 丝裂霉素，由研究者选择）的比较。他还描述了一个可能每年有数万人的更大 “暴露” 人群，他表示，对于这一人群，实际的选择是化疗。 Shefferman 还讨论了影响采用的考虑因素，包括治疗管理时间和社区实践中的操作负担。他对比了学术中心和社区泌尿科医生的需求，并表示 TARA-002 的持续供应对那些担心能否在一段时间内确保完整 BCG 课程的临床医生来说可能是有意义的。 STARBORN-1 淋巴畸形：招募和 FDA 互动 关于 LM 项目，Shefferman 表示公司继续在 STARBORN-1 中招募患者，并且自去年 11 月中期数据发布以来，已有额外患者接受了给药并进行了读数。他表示，Protara 已与 FDA 计划召开会议，讨论 注册路径，并补充说来自多个学科的 FDA 代表将参与。 在被问及潜在治疗人群时，Shefferman 表示他认为可接触的机会可能在 1,000 到 1,200 名患者每年 的范围内，主要由新发病例以及一些由于先前选择有限而变得 “对治疗无动于衷” 的既往治疗患者组成。 关于 LM 和 NMIBC 表现之间的潜在商业分离，Shefferman 表示给药方式差异显著——他描述 LM 给药为每剂 1 KE，最多四剂，而 NMIBC 给药为 40 KE，第一年可能多达 ~21 瓶——并表示公司预计会有不同的品牌、渠道和编码。 IV 氯化胆碱：长期肠外营养和 IP Protara 还讨论了其 静脉用胆碱氯化物 项目，目标是长期接受完全肠外营养（TPN）的患者。Shefferman 估计美国大约有 35,000 名此类患者，将慢性 TPN 定义为每周超过四天且每年超过六个月。 他引用了公司观察性 THRIVE-1 研究的结果，指出大约 80% 的肠外支持患者缺乏胆碱，且 68% 存在某种形式的肝损伤。他描述了胆碱作为磷脂酰胆碱前体的作用，并将缺乏与肝功能和脂肪运输联系起来，指出多种物种的缺胆碱饮食在几天或几周内与肝功能测试升高相关。 Shefferman 表示 Protara 正在追求一个基于药代动力学的终点，证明给药后血清胆碱水平增加，次要终点评估肝功能。他还表示，公司拥有一项 橙皮书上市产品索赔专利，有效期至 2041 年，相关知识产权正在其他地区申请。 关于 Protara Therapeutics NASDAQ: TARA Protara Therapeutics, Inc 是一家临床阶段的生物制药公司，专注于开发针对病毒驱动癌症的新型免疫疗法。公司的主要研究领域包括人乳头瘤病毒（HPV）相关恶性肿瘤，旨在利用和增强身体的免疫反应以靶向肿瘤细胞。Protara 的治疗策略结合了特异性抗原疫苗接种方法与 T 细胞激活调节剂，以改善 HPV 介导疾病患者的临床结果。 Protara 的主要免疫疗法候选药物是 PTX-35，这是一种旨在激活 OX40 的实验性单克隆抗体，OX40 是 T 细胞上的一种共刺激受体。 另请参阅 五只我们认为比 Protara Therapeutics 更好的股票 华尔街害怕的黄金图表…… 美国的 1776 再次发生 在 2026 年 5 月之前购买这只黄金股票 用这四个简单步骤 “防止美联储影响” 你的银行账户 一位前 CIA 特工对即将到来的崩溃的看法 此即时新闻提醒由叙事科学技术和 MarketBeat 的金融数据生成，以便为读者提供最快的报道和公正的覆盖。请将有关此故事的任何问题或评论发送至 contact@marketbeat.com。 你现在应该投资$1,000 在 Protara Therapeutics 吗？ 在考虑 Protara Therapeutics 之前，你需要听听这个。 MarketBeat 每天跟踪华尔街顶级评级和表现最佳的研究分析师及其推荐给客户的股票。MarketBeat 已识别出五只顶级分析师正在悄悄向客户推荐的股票，建议在更广泛的市场注意到之前购买……而 Protara Therapeutics 并不在名单上。 虽然 Protara Therapeutics 目前在分析师中获得了中等买入评级，但顶级分析师认为这五只股票更值得购买。 在这里查看这五只股票 Protara Therapeutics NASDAQ: TARA 的高管在 Oppenheimer 第 36 届年度医疗生命科学会议的炉边谈话中概述了公司两个临床阶段项目的进展，强调了其正在进行的非肌肉侵袭性膀胱癌（NMIBC）TARA-002 研究的更新中期结果，并提供了淋巴畸形（LM）监管规划的最新情况。公司还讨论了其针对接受长期肠外营养患者的静脉注射（IV）氯化胆碱项目。 获取 Protara Therapeutics 的提醒： TARA-002：灭活细菌免疫增强剂 首席执行官 Jesse Shefferman 将 TARA-002 描述为一种来自基因上不同的 链球菌 的全细胞细菌治疗，经过制造过程完全灭活，同时保留旨在刺激免疫系统的抗原。他表示，该产品在日本被称为 OK-432，在日本市场已有数十年，是大囊性和混合型淋巴畸形的标准治疗。他还指出，OK-432 在日本有用于某些癌症（头颈癌、甲状腺癌、胃癌和肺癌）化疗辅助治疗的标签，并将其描述为潜在的早期免疫肿瘤学药物。 Shefferman 强调，Protara 正在两个晚期环境中开发 TARA-002： NMIBC，包括针对 BCG 不响应的原位癌（CIS）的注册研究 淋巴畸形，重点关注 STARBORN-1 研究中的大囊性和混合病变 ADVANCED-2 中期结果和耐久性讨论 讨论集中在 ADVANCED-2 的中期数据上，这是 Protara 正在进行的 TARA-002 在 BCG 不响应 NMIBC 中的研究。Gershell 引用了公司报告的中期结果：在 22 名患者中，六个月的 完全反应（CR）率为 68%，在 15 名可评估患者中，12 个月的 CR 率为 33%。他指出，正式的 ASCO GU 演讲定于第二天进行。 在回答关于 12 个月数据的问题时，Shefferman 将较低的 12 个月标志性 CR 率归因于样本量小以及肿瘤学数据集早期可能出现的波动。他表示，在最新的读数之前，公司在 12 个月反应率方面 “一直在中间 40% 的范围内”，但两名待定的患者最终不再反应，导致该比例下降。 Shefferman 表示，该数据集代表了计划中的 100 名患者 研究的前 15 名患者，并表示公司预计随着样本量的增加，12 个月的反应率将回升到 40% 的范围。他还表示，Protara 观察到，之前的 BCG 暴露似乎并不是反应的主要驱动因素，因为在 BCG 初治和 BCG 不响应组中的六个月反应数据变得更具可解释性。 ADVANCED-3：超越 BCG 不响应 CIS 的定位 两位发言人将 BCG 不响应 CIS 描述为注册的途径，而不是公司最大的商业机会。Shefferman 表示，公司在 NMIBC 中的 “大动作” 是 ADVANCED-3，这是一个计划中的随机对照试验，旨在支持在 BCG 不可用或不适合时使用 TARA-002，可能还包括 BCG 暴露或治疗不足的患者，他表示由于 BCG 短缺，这一人群正在增长。 Shefferman 表示，Protara 预计 ADVANCED-2 将在 “2026 年底之前顺利完成招募”，并将该研究的完成视为建立药房准入、报销基础设施和在泌尿科实践中存在的方式，同时推进 ADVANCED-3。 关于未满足的需求，Shefferman 表示，公司向 FDA 提交了七年的 NMIBC 索赔数据，并观察到大约 35% 的本应接受 BCG 的患者未接受 BCG。他估计这一人群每年大约有 11,000 到 15,000 名患者，基于向该机构提交的索赔数据，引用了患者拒绝和 BCG 不耐受等因素。他表示，FDA 对 ADVANCED-3 设计的接受是基于与膀胱内化疗选项（吉西他滨 或 丝裂霉素，由研究者选择）的比较。他还描述了一个可能每年有数万人的更大 “暴露” 人群，他表示，对于这一人群，实际的选择是化疗。 Shefferman 还讨论了影响采用的考虑因素，包括治疗管理时间和社区实践中的操作负担。他对比了学术中心和社区泌尿科医生的需求，并表示 TARA-002 的持续供应对那些担心能否在一段时间内确保完整 BCG 课程的临床医生来说可能是有意义的。 STARBORN-1 淋巴畸形：招募和 FDA 互动 关于 LM 项目，Shefferman 表示公司继续在 STARBORN-1 中招募患者，并且自去年 11 月中期数据发布以来，已有额外患者接受了给药并进行了读数。他表示，Protara 已与 FDA 计划召开会议，讨论 注册路径，并补充说来自多个学科的 FDA 代表将参与。 在被问及潜在治疗人群时，Shefferman 表示他认为可接触的机会可能在 1,000 到 1,200 名患者每年 的范围内，主要由新发病例以及一些由于先前选择有限而变得 “对治疗无动于衷” 的既往治疗患者组成。 关于 LM 和 NMIBC 表现之间的潜在商业分离，Shefferman 表示给药方式差异显著——他描述 LM 给药为每剂 1 KE，最多四剂，而 NMIBC 给药为 40 KE，第一年可能多达 ~21 瓶——并表示公司预计会有不同的品牌、渠道和编码。 IV 氯化胆碱：长期肠外营养和 IP Protara 还讨论了其 静脉用胆碱氯化物 项目，目标是长期接受完全肠外营养（TPN）的患者。Shefferman 估计美国大约有 35,000 名此类患者，将慢性 TPN 定义为每周超过四天且每年超过六个月。 他引用了公司观察性 THRIVE-1 研究的结果，指出大约 80% 的肠外支持患者缺乏胆碱，且 68% 存在某种形式的肝损伤。他描述了胆碱作为磷脂酰胆碱前体的作用，并将缺乏与肝功能和脂肪运输联系起来，指出多种物种的缺胆碱饮食在几天或几周内与肝功能测试升高相关。 Shefferman 表示 Protara 正在追求一个基于药代动力学的终点，证明给药后血清胆碱水平增加，次要终点评估肝功能。他还表示，公司拥有一项 橙皮书上市产品索赔专利，有效期至 2041 年，相关知识产权正在其他地区申请。 关于 Protara Therapeutics NASDAQ: TARA Protara Therapeutics, Inc 是一家临床阶段的生物制药公司，专注于开发针对病毒驱动癌症的新型免疫疗法。公司的主要研究领域包括人乳头瘤病毒（HPV）相关恶性肿瘤，旨在利用和增强身体的免疫反应以靶向肿瘤细胞。Protara 的治疗策略结合了特异性抗原疫苗接种方法与 T 细胞激活调节剂，以改善 HPV 介导疾病患者的临床结果。 Protara 的主要免疫疗法候选药物是 PTX-35，这是一种旨在激活 OX40 的实验性单克隆抗体，OX40 是 T 细胞上的一种共刺激受体。 另请参阅 五只我们认为比 Protara Therapeutics 更好的股票 华尔街害怕的黄金图表…… 美国的 1776 再次发生 在 2026 年 5 月之前购买这只黄金股票 用这四个简单步骤 “防止美联储影响” 你的银行账户 一位前 CIA 特工对即将到来的崩溃的看法 此即时新闻提醒由叙事科学技术和 MarketBeat 的金融数据生成，以便为读者提供最快的报道和公正的覆盖。请将有关此故事的任何问题或评论发送至 contact@marketbeat.com。 你现在应该投资$1,000 在 Protara Therapeutics 吗？ 在考虑 Protara Therapeutics 之前，你需要听听这个。 MarketBeat 每天跟踪华尔街顶级评级和表现最佳的研究分析师及其推荐给客户的股票。MarketBeat 已识别出五只顶级分析师正在悄悄向客户推荐的股票，建议在更广泛的市场注意到之前购买……而 Protara Therapeutics 并不在名单上。 虽然 Protara Therapeutics 目前在分析师中获得了中等买入评级，但顶级分析师认为这五只股票更值得购买。 在这里查看这五只股票