Sirolimus Albumin-Bound

Fully enrolled registration-intended PRECISION1 trial; two-thirds interim analysis planned for Q3 2024 FYARRO® sales of $5.4 million for Q1 2024, reflects distributor ordering patterns and fewer commercial patient initiations at the start of the year Conference call to be held today at 8:30 am EDT LOS ANGELES, May 8, 2024 /PRNewswire/ -- Aadi Bioscience, Inc. (NASDAQ: AADI), a commercial-stage, precision oncology company focused on developing and commercializing therapies for cancers with alterations in the mTOR pathway, today announced financial results for the first quarter ended March 31, 2024, and highlighted recent corporate progress. "I'm pleased to announce that PRECISION1 is now fully enrolled across a broad array of tumor types and our promising development plan continues to gain momentum. We look forward to providing the two-thirds interim analysis in the third quarter, and full results in early 2025. Additionally, the recently initiated Phase 2 trials in EEC and NETs are enrolling well, and we anticipate initial data from these later this year," said Dave Lennon, President and CEO of Aadi Bioscience. "On the commercial side, FYARRO continues to perform well and has cemented its position as the preferred treatment for malignant PEComa after just two years on the market. The impacts to sales in the first quarter are well-understood events and we expect to return to sales growth in Q2." Recent Operational Highlights FYARRO net product sales were $5.4 million in the first quarter of 2024. This decrease of 8.8% from the prior year period reflects impacts from distributor ordering patterns and fewer new patient initiations than the historical average, which we anticipate will correct in subsequent quarters. Registration-intended PRECISION1 trial is now fully enrolled. PRECISION1 is exploring nab-sirolimus in solid tumors with TSC1 or TSC2 inactivating alterations. The two-thirds interim analysis is expected in Q3 2024, and study completion is expected by year-end. Enrollment into two Phase 2 trials ongoing. These tumor specific trials are investigating the potential of nab-sirolimus for difficult-to-treat mTOR-driven cancers: advanced or recurrent endometrioid-type endometrial cancer (EEC) in combination with letrozole, and neuroendocrine tumors (NETs). At Aadi's request, Mirati/Bristol Myers Squibb and Aadi mutually agreed to terminate their collaboration and clinical supply agreement. Aadi is prioritizing investment in its Phase 2 trials in EEC and NETs. The Phase 1/2 trial with Mirati/Bristol Myers Squibb evaluated the combination of nab-sirolimus + adagrasib in non-small cell lung cancer with a KRASG12C mutation. First Quarter 2024 Financial Results Cash, cash equivalents and short-term investments as of March 31, 2024, were $88.3 million as compared to $108.8 million as of December 31, 2023, which is expected to fund operations into Q4 2025 based on current plans. Total revenue for the quarter ended March 31, 2024, was $5.4 million, resulting from sales of FYARRO. Net loss for the three months ended March 31, 2024, was $18.3 million as compared to $15.2 million for the three months ended March 31, 2023. Conference Call Information The Aadi management team is hosting a conference call and webcast today at 8:30 am EDT (5:30 am PDT) to provide a corporate update and discuss results for the first quarter 2024. Participants may access a live webcast of the call on the "Investors & News" page of the Aadi Bioscience website at aadibio.com. To participate via telephone, please register in advance at this link. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. A replay of the conference call and webcast will be archived on the Company's website for at least 30 days. About Aadi Bioscience Aadi is a commercial-stage precision oncology company focused on the development and commercialization of therapies for cancers with alterations in the mTOR pathway, a key regulator of cell growth and cancer progression. To unlock the full potential of mTOR inhibition, Aadi uniquely combines nanoparticle albumin-bound (nab) technology with the potent mTOR inhibitor, sirolimus. Aadi received FDA approval and commercializes FYARRO® for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). Aadi is exploring nab-sirolimus in PRECISION1, a Phase 2 tumor-agnostic registration-intended trial in mTOR inhibitor-naïve malignant solid tumors harboring TSC1 or TSC2 inactivating alterations. Aadi is also exploring nab-sirolimus in two tumor specific Phase 2 trials for difficult-to-treat mTOR-driven cancers: neuroendocrine tumors (NETs), and advanced or recurrent endometrioid-type endometrial cancer (EEC) in combination with letrozole. More information on the Company's development pipeline is available on the Aadi website at and connect with us on Twitter and LinkedIn. Forward-Looking Statements This press release contains certain forward-looking statements regarding the business of Aadi Bioscience that are not a description of historical facts within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are based on the Company's current beliefs and expectations and may include, but are not limited to, statements relating to: the Company's cash runway extending into the fourth quarter of 2025; the anticipated timing of commencement, enrollment, data releases and completion of the Company's clinical trials, including the expected PRECISION 1 two-thirds interim analysis in Q3 2024 and full results in early 2025 and Phase 2 trials in EEC and NETs later in 2024; the Company's anticipated growth and continued advancements, including in potential additional indications; expectations regarding the beneficial characteristics, safety, efficacy, therapeutic effects and the size of the potential targeted markets with respect to FYARRO, including in NETs and EEC; plans and potential for success relating to commercializing FYARRO; and the sufficiency of the Company's existing capital resources and the expected timeframe to fund the Company's future operating expenses and capital expenditure requirements. Actual results could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, uncertainties associated with the clinical development and regulatory approval of FYARRO in additional indications, including potential delays in the commencement, enrollment and completion of clinical trials for additional indications; risks associated with the ability to successfully commercialize FYARRO; failure to demonstrate the efficacy of FYARRO in clinical trials for additional indications; the risk that unforeseen adverse reactions or side effects may occur in the course of commercializing, developing and testing FYARRO; and risks related to the Company's estimates regarding future expenses, capital requirements and need for additional financing. Additional risks and uncertainties that could cause actual outcomes and results to differ materially from those contemplated by the forward-looking statements are included in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, including under the caption "Item 1A. Risk Factors," and in Aadi's subsequent Quarterly Reports on Form 10-Q, and elsewhere in Aadi's reports and other documents that Aadi has filed, or will file, with the SEC from time to time and available at . All forward-looking statements in this press release are current only as of the date hereof and, except as required by applicable law, Aadi undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise. All forward-looking statements are qualified in their entirety by this cautionary statement. This cautionary statement is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Contact: [email protected] SOURCE Aadi Bioscience

▎药明康德内容团队报道根据中国国家药监局药品审评中心（CDE）官网，在刚刚结束的4月份，有10多款新药上市申请（NDA）或新适应症上市申请获得CDE受理。通过梳理，这些新药涵盖了抗PCSK9单抗、抗IL-1β单抗、抗IL-4Rα单抗、PD-1/CTLA-4双抗、GLP-1受体激动剂等等，它们拟用于治疗结直肠癌、宫颈癌、乳腺癌等癌症，痛风性关节炎、季节性过敏性鼻炎等炎症性疾病，以及糖尿病、肢端肥大症等。本文将结合公开资料介绍部分产品的信息，看看它们有哪些特点，以及有望惠及哪些患者。君实生物：昂戈瑞西单抗注射液作用机制：抗PCSK9单抗适应症：高胆固醇血症、血脂异常等昂戈瑞西单抗是君实生物自主研发的注射用重组人源化抗PCSK9单克隆抗体。根据君实生物新闻稿，本次该产品2项新适应症上市申请获CDE受理，用于治疗：1）杂合子型家族性高胆固醇血症；2）他汀类药物不耐受或禁忌使用的原发性高胆固醇血症和混合型血脂异常。本次新适应症上市申请主要基于两项3期注册临床试验（JS002-005、JS002-007）。优锐医药等：注射用牛肺表面活性剂作用机制：吸入性药械联合产品适应症：新生儿呼吸窘迫综合征（NRDS）由优锐医药（Nuance Pharma）、Lyomark Pharma等联合申报的5.1类新药注射用牛肺表面活性剂上市申请获CDE受理。根据优锐医药公开资料，这是该公司引进的一款无创、吸入性药械联合产品AeroFact，该产品用于治疗新生儿呼吸窘迫综合征（NRDS）。NRDS是一种新生儿科的呼吸障碍，在早产儿中高发，并严重危及生命，临床已获批的疗法为肺表面活性剂器官插管治疗，具有较高的创伤风险。由于肺表面活性剂具有技术难度的物理特性，以及治疗早产儿患者的固有困难，以气雾形式将该药物通过早产儿鼻腔向肺输送非常具有挑战。AeroFact是一种经鼻吸入的牛肺表面活性剂给药系统，结合了成熟的牛肺表面活性剂Alveofact（Lyomark Pharma公司），以及采用Aerogen公司的新一代输送技术和经鼻输送系统，可以实现持续、高效给药。而及时无创、高效的治疗对于患儿未来一生的生活质量影响深远。AeroFact此前已经获得美国FDA授予的快速通道资格和孤儿药资格。金赛药业：注射用金纳单抗作用机制：抗IL-1β单抗适应症：急性痛风性关节炎根据长春高新公告，其子公司金赛药业申报的注射用金纳单抗上市申请获CDE受理，针对适应症为急性痛风性关节炎。金纳单抗是一种抗白介素1-β（IL-1β）全人源单克隆抗体，可以快速和特异性地阻断IL-1β与其受体的结合，从而阻断炎症发生。在骨关节炎患者中，IL-1β刺激多种金属蛋白酶的产生，导致结缔组织破裂，抑制胶原蛋白的产生，从而对关节软骨产生负面影响。此外，IL-1β对破骨细胞的成熟有直接及间接的刺激作用，从而介导关节炎中骨侵蚀的发展。IL-1拮抗剂目前已是临床常用的痛风性关节炎的治疗药物之一。锐康迪医药：注射用双羟萘酸帕瑞肽微球作用机制：第二代生长抑素类似物适应症：肢端肥大症Recordati集团中国全资子公司锐康迪医药申报的注射用双羟萘酸帕瑞肽微球上市申请获CDE受理。该产品还于今年3月被CDE纳入优先审评，拟用于治疗无法手术或手术后未治愈和通过另一种生长抑素类似物治疗控制不佳的成人肢端肥大症患者。公开资料显示，这是一款长效帕瑞肽产品（pasireotide pamoate），已经于2014年相继获得欧洲药品管理局（EMA）和FDA批准治疗成人肢端肥大症患者。此外，博鳌乐城维健罕见病临床医学中心已经引进该产品治疗肢端肥大症患者。长效帕瑞肽（即双羟萘酸帕瑞肽）为第二代生长抑素类似物（SRL）。与第一代SRL相比，长效帕瑞肽增强了生长抑素受体（SSTR） 的结合谱，即对SSTR1、2、3和SSTR5亚型具有高亲和力，尤其对SSTR5的亲和力最高，具有更好的临床和生化控制率，且安全性良好。百时美施贵宝：伊匹木单抗+纳武利尤单抗作用机制：双免疫联合疗法适应症：结直肠癌百时美施贵宝申报的纳武利尤单抗联合伊匹木单抗上市申请获受理，同时被CDE纳入优先审评，针对适应症为一线治疗不可切除或转移性微卫星高度不稳定性（MSI-H）或错配修复缺陷型（dMMR）结直肠癌患者。伊匹木单抗是一款CTLA-4抗体，纳武利尤单抗是一款PD-1抑制剂。根据百时美施贵宝于今年1月公布的临床研究（CheckMate-8HW）数据，与化疗相比，两药联合疗法一线治疗MSI-H/dMMR表型的mCRC患者，将疾病进展或死亡风险降低了79%。此外，双免疫联合疗法组的PFS改善从大约三个月时就开始出现，并贯穿始终。恒瑞医药：氟唑帕利胶囊+阿帕替尼作用机制：PARP抑制剂+抗血管生成药物适应症：HER2阴性乳腺癌恒瑞医药两款创新药上市申请获CDE受理，并纳入优先审评，针对适应症为：氟唑帕利胶囊单药或联合甲磺酸阿帕替尼用于伴有胚系BRCA突变（gBRCAm）的HER2阴性乳腺癌患者的治疗。氟唑帕利是恒瑞医药研发的1类创新药，为一种新型口服PARP抑制剂；阿帕替尼是恒瑞医药研发一种高选择性的靶向血管内皮细胞生长因子受体2（VEGFR2）的抗血管生成药物。根据恒瑞医药新闻稿介绍，2023年12月，氟唑帕利单药及联合甲磺酸阿帕替尼治疗乳腺癌的3期FZPL-Ⅲ-303研究主要研究终点结果达到方案预设的优效标准。研究期中分析结果表明，与研究者选择化疗组相比，氟唑帕利联合甲磺酸阿帕替尼组及氟唑帕利单药组的无进展生存期均取得了显著的且有临床意义的改善。康方生物：卡度尼利作用机制：PD-1/CTLA-4双抗适应症：宫颈癌根据康方生物新闻稿，卡度尼利第3个适应症上市申请获CDE受理，拟加含铂化疗联合/不联合贝伐珠单抗对比安慰剂加含铂化疗联合/不联合贝伐珠单抗用于一线治疗持续、复发或转移性宫颈癌。卡度尼利为一款PD-1/CTLA-4双特异性抗体，此前已获得中国NMPA批准上市，单药治疗既往接受含铂化疗治疗失败的复发或转移性宫颈癌。此次新适应症上市申请获得受理是基于AK104-303研究，研究显示，卡度尼利在一线治疗晚期宫颈癌全人群中展现出突出疗效。常山药业：艾本那肽注射液作用机制：GLP-1受体激动剂适应症：2型糖尿病根据常山药业新闻稿，艾本那肽为其控股子公司常山凯捷健生物开发的GLP-1受体激动剂，本次申报的适应症为治疗2型糖尿病。艾本那肽注射液是一款长效胰高血糖素样肽-1（GLP-1）类药物，采用独特的药物亲和力偶合（DAC）技术，对GLP-1受体激动剂类药物Exendin-4进行化学修饰，通过抵抗快速降解和快速肾清除克服了天然Exendin-4较短血浆半衰期的缺点，达到一周给药1次的效果。临床研究显示，在2型糖尿病受试者中连续给药24周，艾本那肽注射液可显著降低受试者血糖水平，且起效较快，第5周即可观察到显著的有效性，治疗后第5~17周血糖持续降低，并在第17周趋于稳定，疗效可维持至52周。康诺亚：司普奇拜单抗注射液作用机制：抗IL-4Rα单抗适应症：季节性过敏性鼻炎根据康诺亚新闻稿，其1类创新药司普奇拜单抗治疗季节性过敏性鼻炎的新药上市许可申请已获CDE受理。司普奇拜单抗（CM310）是一种针对白介素4受体α亚基（IL-4Rα）的高效、人源化抗体，此前该产品已在中国递交治疗成人中重度特应性皮炎的上市申请，并被纳入优先审评。在针对对鼻用糖皮质激素或其他治疗方法控制不佳的成人季节性过敏性鼻炎患者的3期临床研究中，司普奇拜单抗组显著优于安慰剂组，具备高度显著的统计学差异，且安全性良好。除了上述新药，还有其他新药的上市申请于今年4月获CDE受理。限于篇幅，本文不再一一介绍。希望这些新药能早日在中国获批上市，造福患者！ 参考资料（可上下滑动查看）：[1]中国国家药监局药品审评中心（CDE）官网. Retrieved Apr 30 , 2024, from https://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d[2] 君实生物宣布昂戈瑞西单抗2项新适应症上市申请获得国家药品监督管理局受理. Retrieved Apr 2 , 2024, from https://mp.weixin.qq.com/s/K4faNuVdjIuEy19ng5GJnA[3]优锐医药携手蔻德罕见病中心，助力中国罕见病领域研究发展 . Retrieved July 13 , 2022, from https://mp.weixin.qq.com/s/cz3M5QF8G6C5S28mmDPUrQ{4}长春高新关于子公司注射用金纳单抗上市申请获得受理的公告. Retrieved Apr 12 , 2024, from http://www.cninfo.com.cn/new/disclosure/detail?stockCode=000661&announcementId=1219583997&orgId=gssz0000661&announcementTime=2024-04-12[5]恒瑞氟唑帕利单药或联合阿帕替尼用于HER2阴性乳腺癌申报上市且拟被纳入优先审评. Retrieved Apr 12 , 2024, from https://mp.weixin.qq.com/s/J3GO-9B3T1sPXBF975iwUA[6]康方生物双抗卡度尼利一线治疗晚期宫颈癌全人群的sNDA获CDE受理. Retrieved Apr 24, 2024, from https://mp.weixin.qq.com/s/mxxYyUI8PGdbGZvDvhdUhw[7]艾本那肽注射液（GLP-1受体激动剂）新药上市申请获国家药品监督管理局正式受理. Retrieved Apr 24 2024, from https://mp.weixin.qq.com/s/eVAKxE3x3BoxBvgIRXbw1A[8]司普奇拜单抗治疗季节性过敏性鼻炎的药品上市许可申请获受理. Retrieved Apr 30, 2024, from https://mp.weixin.qq.com/s/2S57RdLpPNTpFWbMsLznGw本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。