Irinotecan Hydrochloride Lioposome

DSMB Recommends Continuation without Modification for Third Time Safety Review Included 395 Patients Interim Survival Analysis Still Expected Early 2025 given Lower-Than-Expected Event Rate Low Event Rate Suggests Potential for Prolonged Survival Rapid Enrollment Positions Company for Enrollment Completion by Q1 2025; Earlier Than Expected MINNEAPOLIS, June 24, 2024 (GLOBE NEWSWIRE) -- Panbela Therapeutics Inc. (OTCQB: PBLA), a clinical-stage biopharmaceutical company developing disruptive therapeutics for the treatment of patients with urgent unmet medical needs, today announced that the independent Data Safety Monitoring Board (DSMB) has completed its third pre-specified safety review of the ongoing Phase 3 ASPIRE clinical trial evaluating ivospemin in combination with gemcitabine and nab-Paclitaxel for the first-line treatment of metastatic pancreatic ductal adenocarcinoma (mPDAC). The DSMB recommended study continuation without modification, marking the third consecutive positive safety review. The safety database now includes 395 patients, compared to 214 patients on November 29, 2023. "We are pleased with the DSMB's recommendation to continue the ASPIRE trial without modification, now for the third time, which is encouraging," said Jennifer K. Simpson, PhD, MSN, CRNP, President & Chief Executive Officer of Panbela Therapeutics. "We also remain encouraged by the lower-than-expected event rate, which suggests that patients in the ASPIRE trial have experienced prolonged survival. We are confirming our expectation that the interim survival analysis will be available as early as the first quarter of 2025. This is a positive development for patients and underscores the potential of ivospemin in addressing a significant unmet need in the treatment of metastatic pancreatic ductal adenocarcinoma." Key Takeaways: The DSMB's recommendation to proceed without modification affirms support for ivospemin’s safety profile. The safety database has expanded to 395 patients, providing a robust foundation for evaluating ivospemin's safety. The lower-than-expected event rate suggests the potential for prolonged survival among ASPIRE trial participants. Rapid enrollment positions Panbela to remain on path to complete enrollment in Q1 2025, earlier than initially anticipated. Panbela also highlighted the significance of the ASPIRE trial in the context of recent advancements in mPDAC treatment, such as the Napoli 3 trial, which led to the approval of liposomal irinotecan (Onivyde) in combination with fluorouracil, oxaliplatin, and leucovorin (NALIRIFOX). Despite this approval, which was based on a median overall survival benefit of 1.9 months compared to gemcitabine and nab-paclitaxel, the prognosis for patients with mPDAC remains poor, with median overall survival still less than 12 months. The incremental benefits in median survival have been modest in the past 11 years, with the recent approval of Onivyde in the NALIRIFOX regimen demonstrating a 1.9-month survival benefit compared to the approval of gemcitabine and nab-paclitaxel, which was based on a median overall survival benefit of 1.8 months over gemcitabine alone. "We believe that the addition of ivospemin (SBP-101) to the standard-of-care regimen of gemcitabine and nab-paclitaxel has the potential to significantly improve outcomes for patients with mPDAC, beyond the incremental benefits observed with the recently approved therapy," added Dr. Simpson." We remain committed to advancing this important study and look forward to sharing the interim results in Q1 2025." Panbela remains committed to advancing the ASPIRE trial and evaluating ivospemin's potential to improve outcomes for patients with mPDAC. Despite recent advancements in treatment, the median overall survival for patients with mPDAC remains less than 12 months. The company looks forward to the interim survival analysis in early 2025, which will provide important insights into ivospemin's potential to address this significant unmet medical need. About Panbela’s Pipeline The pipeline consists of assets currently in clinical trials with an initial focus on familial adenomatous polyposis (FAP), first-line metastatic pancreatic cancer, neoadjuvant pancreatic cancer, colorectal cancer prevention and ovarian cancer. The combined development programs have a steady cadence of anticipated catalysts with programs ranging from pre-clinical to registration studies. Ivospemin (SBP-101) Ivospemin is a proprietary polyamine analogue designed to induce polyamine metabolic inhibition (PMI) by exploiting an observed high affinity of the compound for pancreatic ductal adenocarcinoma and other tumors. It has shown signals of tumor growth inhibition in clinical studies of metastatic pancreatic cancer patients, demonstrating a median overall survival (OS) of 14.6 months and an objective response rate (ORR) of 48%, both exceeding what is typical for the standard of care of gemcitabine + nab-paclitaxel suggesting potential complementary activity with the existing FDA-approved standard chemotherapy regimen. In data evaluated from clinical studies to date, ivospemin has not shown exacerbation of bone marrow suppression and peripheral neuropathy, which can be chemotherapy-related adverse events. Serious visual adverse events have been evaluated and patients with a history of retinopathy or at risk of retinal detachment will be excluded from future SBP-101 studies. The safety data and PMI pro in the previous Panbela-sponsored clinical trials provide support for continued evaluation of ivospemin in the ASPIRE trial. Flynpovi ™ Flynpovi is a combination of CPP-1X (eflornithine) and sulindac with a dual mechanism inhibiting polyamine synthesis and increasing polyamine export and catabolism. In a Phase III clinical trial in patients with sporadic large bowel polyps, the combination prevented > 90% subsequent pre-cancerous sporadic adenomas versus placebo. Focusing on FAP patients with lower gastrointestinal tract anatomy in the recent Phase III trial comparing Flynpovi to single agent eflornithine and single agent sulindac, FAP patients with lower GI anatomy (patients with an intact colon, retained rectum or surgical pouch), showed statistically significant benefit compared to both single agents (p≤0.02) in delaying surgical events in the lower GI for up to four years. The safety pro Flynpovi did not significantly differ from the single agents and supports the continued evaluation of Flynpovi for FAP. CPP-1X CPP-1X (eflornithine) is being developed as a single agent tablet or high dose powder sachet for several indications including prevention of gastric cancer, treatment of neuroblastoma and recent onset Type 1 diabetes. Preclinical studies as well as Phase I or Phase II investigator-initiated trials suggest that CPP-1X treatment may be well-tolerated and has potential activity. About Panbela Panbela Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing disruptive therapeutics for patients with urgent unmet medical needs. Panbela’s lead assets are Ivospemin (SBP-101) and Flynpovi. Further information can be found at . Panbela’s common stock is eligible for quotation on the OTCQB under the symbol “PBLA”. Cautionary Statement Regarding Forward-Looking Statements This press release contains “forward-looking statements,” including within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “anticipate,” “believe,” “can,” “design,” “expect,” “focus,” “intend,” “looking forward,” “may,” “plan,” “positioned,” “potential,” and “will.” All statements other than statements of historical fact are statements that should be deemed forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on our current beliefs, expectations, and assumptions regarding the future of our business, future plans and strategies, projections, anticipated events and trends, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Our actual results and financial condition may differ materially and adversely from the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause our actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the following: (i) our ability to obtain additional capital, on acceptable terms or at all, required to implement our business plan; (ii) our lack of diversification and the corresponding risk of an investment in our Company; (iii) our ability to obtain or maintain a listing on a national securities exchange; (iv) progress and success of our randomized Phase II/III clinical trial; (v) our ability to demonstrate the safety and effectiveness of our product candidates: ivospemin ( SBP-101 ), Flynpovi, and eflornithine (CPP-1X) (v) our ability to obtain regulatory approvals for our product candidates, SBP-101, Flynpovi and CPP-1X in the United States, the European Union or other international markets; (vii) the market acceptance and level of future sales of our product candidates, SBP-101, Flynpovi and CPP-1X ; (viii) the cost and delays in product development that may result from changes in regulatory oversight applicable to our product candidates, SBP-101, Flynpovi and CPP-1X ; (ix) the rate of progress in establishing reimbursement arrangements with third-party payors; (x) the effect of competing technological and market developments; (xi) the costs involved in filing and prosecuting patent applications and enforcing or defending patent claims; and (xii) such other factors as discussed in Part I, Item 1A under the caption “Risk Factors” in our most recent Annual Report on Form 10-K, any additional risks presented in our Quarterly Reports on Form 10-Q and our Current Reports on Form 8-K. Any forward-looking statement made by us in this press release is based on information currently available to us and speaks only as of the date on which it is made. We undertake no obligation to publicly update any forward-looking statement or reasons why actual results would differ from those anticipated in any such forward-looking statement, whether written or oral, whether as a result of new information, future developments or otherwise. Contact Information: Investors: James Carbonara Hayden IR (646) 755-7412 james@haydenir.com Media: Tammy Groene Panbela Therapeutics, Inc. (952) 479-1196 IR@panbela.com

▎药明康德内容团队编辑 本期看点 1. CD19靶向候选CAR-T细胞疗法GLPG5101在1项早期临床试验中治疗5例套细胞淋巴瘤（MCL）患者，客观缓解率（ORR）和完全缓解率（CRR）均为100%。 2. 靶向信号调节蛋白（SIRP）的抗体疗法ELA026治疗初治恶性肿瘤相关噬血细胞性淋巴组织细胞增多症（mHLH）的总缓解率达100%，并提高了此类患者的两个月生存率。 3. 抗体疗法Natrunix联用化疗治疗晚期胰腺癌，在早期临床试验中延长了患者的生存期并改善了化疗的耐受性。 药明康德内容团队整理 GLPG5101：公布1/2期临床试验数据 Galapagos公司公布了其CD19靶向候选CAR-T细胞疗法GLPG5101治疗复发/难治性非霍奇金淋巴瘤（NHL）的1/2期临床试验ATALANTA-1的新数据。该细胞疗法从制备到回输到患者体内的中位时间仅需7天。 截至2023年12月20日的数据，在研究的第一部分，中位随访时间为13.1个月，接受GLPG5101治疗的16例可评估患者的ORR为87.5%（14/16），CRR为75%（12/16）。71%的患者在数据截止时保持缓解。在研究的第二部分，中位随访时间为4.2个月，15例可评估患者的ORR和CRR均为93.3%（14/15）。100%的患者在数据截止时保持持续缓解。合并两部分的研究结果后根据肿瘤类型进行划分，弥漫性大B细胞淋巴瘤（DLBCL）患者的ORR为78%（7/9），CRR为56%（5/9）；滤泡性淋巴瘤（FL）或边缘区淋巴瘤（MZL）患者的ORR和CRR均为94%（16/17）；套细胞淋巴瘤患者的ORR和CRR均为100%（5/5）。 安全性方面，大多数治疗伴发不良事件为1/2级，在研究的第一部分观察到2例3级细胞因子释放综合征（CRS），在研究的第二部分观察到1例3级免疫效应细胞相关神经毒性综合征（ICANS）。 ELA026：公布1b期临床试验的新数据 Electra Therapeutics公司公布了其靶向信号调节蛋白（SIRP）的单克隆抗体ELA026治疗继发性噬血细胞性淋巴组织细胞增生症（sHLH）的1b期研究的最新临床数据。ELA026靶向细胞表面的SIRP，能够靶向清除血液循环中驱动炎症的髓系细胞和T细胞，并且不破坏SIRPα/CD47免疫检查点功能。该疗法旨在治疗伴有异常髓系和T细胞活性的重度炎性疾病。 此次公布的结果显示，ELA026在sHLH患者中具有良好的安全性，不良事件可控。在已完成入组的队列1和2中（n=12），ELA026在第4周时的总缓解率为75%，在8例未接受过治疗的mHLH（sHLH的一种亚型）患者中，总缓解率为100%，两个月时的生存率为88%。此类患者在自然史研究中的两个月时的死亡率约为50%。 Natrunix：公布1/2期临床试验数据 XBiotech公司公布了其靶向白细胞介素-1α（IL-1α）的抗体疗法Natrunix联用化疗治疗晚期胰腺癌的1/2期多中心研究的数据。除了能够作为一种抗癌疗法，该公司认为Natrunix还有望改善化疗的耐受性。该研究中采用的化疗方案为ONIVYDE（ON）+5-氟尿嘧啶（5FU）+亚叶酸（LV）。 此次公布的结果显示，与安慰剂+化疗组相比，Natrunix+化疗组在24周的治疗周期内发生任何类型的不良事件的数量（297例对比336例）、发生重大不良事件的患者比例（9/33对比12/32）和住院天数（80天对比120天）均明显减少。此外，接受Natrunix+化疗组患者的生存期得到了延长，接受治疗第330天时有8例患者存活，而安慰剂+化疗组没有患者存活超过330天。 ▲患者的生存曲线（图片来源：参考资料[9]） Petrelintide：公布1b期临床试验数据 Zealand Pharma公司公布了其在研长效、潜在“best-in-class”减重疗法petrelintide的1b期试验第二部分的顶线结果。Petrelintide（ZP8396）是一种适合每周一次皮下注射的长效胰淀素类似物，具有中性pH下的化学和物理稳定性。胰淀素在胰腺β细胞中产生，并与胰岛素一起分泌以响应摄入的营养物质。目前的临床或临床前数据表明，petrelintide有潜力实现与GLP-1受体激动剂相当的体重减轻，但具有更好的耐受性，为患者提供更好的用药体验和体重减轻，同时保留患者的肌肉质量。 此次公布的结果显示，患者在接受16周高剂量petrelintide治疗后，体重较基线平均减少了8.6%，而安慰剂组仅为1.7%。安全性方面，petrelintide显示良好的耐受性，没有出现严重的不良事件。所有胃肠道（GI）不良事件均为轻度，除了一名受试者在第三次治疗后因中度恶心和呕吐而停止治疗。值得注意的是，没有其他受试者因不良事件停止治疗或报告呕吐，只有两例轻度腹泻报告。恶心在活性组中报告比例为16.7-33.3%，在安慰剂组为16.7%。少数参与者报告了注射部位反应，均为轻度。未观察到患者产生抗药抗体。新闻稿指出，该积极结果支持petrelintide作为GLP-1受体激动剂类药物之外，在体重管理上的另一选择。 Amezalpat（TPST-1120）：公布1b/2期临床试验的新数据 Tempest Therapeutics公司公布了其在研口服、选择性小分子PPAR⍺拮抗剂amezalpat（TPST-1120）在一项全球随机的1b/2期临床试验中获得的积极结果。临床前数据显示，amezalpat可直接杀死肿瘤细胞，并靶向肿瘤微环境中的抑制性免疫通路。这两种类型中的靶向细胞皆依赖脂肪酸代谢，该代谢过程由PPARα转录因子调节。在一项针对接受过多次治疗的晚期实体瘤患者的1期临床试验中，amezalpat单药治疗和与PD-1抑制剂nivolumab联合应用均导致肿瘤缩小及生物标志物的变化。 此次公布的结果显示，与标准治疗方案相比，amezalpat与标准治疗方案（抗PD-L1抗体atezolizumab和抗VEGF抗体bevacizumab）联用，在无法切除或转移性肝细胞癌（HCC）患者的一线治疗中，显著提高患者的中位总生存期（OS）达6个月（21个月对比15个月）。此外，按照RECIST v1.1标准评估，amezalpat组的确认客观缓解率（cORR）为30%，对照组为13.3%。安全性方面，amezalpat的耐受性良好，两组之间的安全数据相当。 ▲Amezalpat试验的OS结果（图片来源：参考资料[29]） MB-106：公布1/2期临床试验数据 Mustang Bio公司公布了其在研、可通过门诊给药、靶向CD20的自体CAR-T细胞疗法MB-106治疗华氏巨球蛋白血症（WM）的1/2期临床试验最新数据。该试验中的所有十例患者均曾接受过布鲁顿酪氨酸激酶抑制剂（BTKi）的治疗，并且在接受BTKi治疗期间病情持续恶化。患者之前接受过中位9线疗法，只有一例患者在接受MB-106治疗后开始接受额外的抗WM治疗。 总体而言，接受MB-106治疗的患者中有90%（9/10）对治疗产生应答，包括3名患者达完全缓解（CR）、2名达非常好的部分缓解和4名部分缓解。此外，1例患者的病情稳定。其中一名获得CR的患者已持续缓解31个月，该患者的免疫球蛋白M（IgM）水平在接受MB-106治疗后迅速降至正常范围，此后一直保持正常。 安全性方面，9例患者发生了CRS（5例患者为1级，4例患者为2级），一例患者经历了1级ICANS。尽管剂量增加，但并未观察到3级或4级CRS或2、3或4级ICANS。 NX-594：公布1a/b期临床试验数据 Nurix Therapeutics公司公布了其在研口服BTK降解剂NX-5948治疗复发难治性慢性淋巴细胞白血病（CLL）的1a/b期临床试验的积极数据。NX-5948是一种生物利用度高、可穿越血脑屏障的口服BTK小分子降解剂。 ▲NX-5948的疗效结果（图片来源：参考资料[5]） 此次公布的结果显示，NX-594在复发难治性CLL患者身上引起了快速、深度的缓解，ORR达69.2%，这些患者之前曾接受大量治疗并带有BTK抑制剂耐药突变。最早在第一次肿瘤扫描（8周）时就观察到患者出现缓解，许多患者在治疗时间延长后缓解加深。截至4月17日数据截止，所有缓解仍在持续。 ▲一例患者接受NX-5948治疗24周内，脑脊液中的恶性肿瘤细胞消除（图片来源：参考资料[5]） ▲一例曾接受过11线疗法的患者在接受NX-5948治疗后肿瘤持久消退（图片来源：参考资料[2]） 此外，一例在进入研究时伴有中枢神经系统受累的CLL患者在接受NX-5948治疗的24周内消除了脑脊液（CSF）中的恶性肿瘤细胞，并在36周时达到接近CR的标准。还有一例曾接受过11线疗法的患者在接受NX-5948治疗后肿瘤持久消退已超过6个月。安全性方面，NX-5948在所有评估剂量下均耐受性良好，最常见的治疗不良事件为紫癜/挫伤、血小板减少和中性粒细胞减少。Nurix预计在2025年启动NX-5948的关键临床试验。 SL-172154：公布1b期临床试验数据 Shattuck Labs公布了其在研疗法SL-172154在1b期剂量扩展临床试验的最新中期数据。SL-172154（SIRPα-Fc-CD40L）是一款在研重定向检查点激动剂（ARC）融合蛋白，旨在同时抑制CD47/SIRPα检查点的相互作用并激活CD40共刺激受体，以增强晚期癌症患者的抗肿瘤免疫反应。 此次公布的结果显示，SL-172154与阿扎胞苷（AZA）联用作为高风险骨髓增生异常综合征（HR-MDS）和TP53突变（TP53m）急性髓性白血病（AML）患者的一线疗法时，患者的ORR分别达67%与43%，CRR分别为42%与29%。截至2024年4月23日，SL-172154表现出可控的安全性。输液相关反应（IRRs）是SL-172154治疗过程中最常见的不良事件。 KT-333：公布1期临床试验的新数据 Kymera Therapeutics公布了其在研药物KT-333的1期临床试验的更新数据。KT-333是一种潜在“first-in-class”、强效、高选择性的STAT3靶向小分子降解剂。根据新闻稿，KT-333是靶向未成药转录因子的首个进入临床阶段的降解剂。 ▲KT-333试验临床结果摘要（图片来源：参考资料[3]） 此次公布的结果显示，KT-333完成初步临床概念验证，在多种血液恶性肿瘤中展示了抗肿瘤活性。其中，两名经典霍奇金淋巴瘤患者达到CR，1名STAT3突变的NK细胞淋巴瘤患者达到CR。安全性方面，KT-333耐受性良好，主要不良事件为1级和2级，包括口腔炎和疲劳。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] Shattuck Labs Announces Updated Positive Interim Data from the Phase 1B Dose Expansion Clinical Trial of SL-172154 in Combination with Azacitidine (AZA) in Frontline Higher-Risk Myelodysplastic Syndromes (HR-MDS) and TP53 mutant (TP53m) Acute Myeloid Leukemia (AML) Patients. Retrieved June 21, 2024 from https://www.globenewswire.com/news-release/2024/06/14/2898850/0/en/Shattuck-Labs-Announces-Updated-Positive-Interim-Data-from-the-Phase-1B-Dose-Expansion-Clinical-Trial-of-SL-172154-in-Combination-with-Azacitidine-AZA-in-Frontline-Higher-Risk-Myel.html [2] Kymera Therapeutics Presents New Clinical Data from the Ongoing Phase 1 Trial of STAT3 Degrader KT-333 at EHA Annual Meeting. Retrieved June 21, 2024 from https://www.globenewswire.com/news-release/2024/06/14/2898896/0/en/Kymera-Therapeutics-Presents-New-Clinical-Data-from-the-Ongoing-Phase-1-Trial-of-STAT3-Degrader-KT-333-at-EHA-Annual-Meeting.html [3] Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of KT-333, a Targeted Protein Degrader of STAT3, in Patients with Relapsed or Refractory Hematologic and Solid Tumor Cancers. Retrieved June 21, 2024 from https://www.kymeratx.com/wp-content/uploads/2024/06/Kymera-Therapeutics-EHA-KT-333-Poster_June-2024.pdf [4] Nurix Therapeutics Presents Positive Results from Ongoing Clinical Trial of NX-5948 in Patients with Relapsed Refractory Chronic Lymphocytic Leukemia (CLL) at the European Hematology Association Congress (EHA2024). Retrieved June 21, 2024 from https://ir.nurixtx.com/news-releases/news-release-details/nurix-therapeutics-presents-positive-results-ongoing-clinical [5] EHA2024 Presentation. Retrieved June 21, 2024 from https://ir.nurixtx.com/static-files/e77e9b9c-2e5f-4409-b819-6a5bc02dce47 [6] Galapagos presents encouraging new data for CD19 CAR-T candidate GLPG5101 in non-Hodgkin lymphoma at EHA 2024. Retrieved June 21, 2024 from https://www.globenewswire.com/news-release/2024/06/14/2899182/0/en/Galapagos-presents-encouraging-new-data-for-CD19-CAR-T-candidate-GLPG5101-in-non-Hodgkin-lymphoma-at-EHA-2024.html [7] Editas Medicine Announces New Safety And Efficacy Data From The EdiTHAL Trial Of Reni-Cel In 7 Patients With Transfusion-Dependent Beta Thalassemia, Presented At The European Hematology Association (EHA) Annual Congress. Retrieved June 21, 2024 from https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-new-safety-and-efficacy-data-edithal [8] Editas Medicine Reports New Safety And Efficacy Data From The RUBY Trial Of Reni-Cel In 18 Patients With Sickle Cell Disease, Presented At The European Hematology Association (EHA) Annual Congress. Retrieved June 21, 2024 from https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-reports-new-safety-and-efficacy-data-ruby-trial [9] XBiotech Results from Randomized Double-Blinded Phase 1/2 Study Suggest Potential Breakthrough Treatment for Advanced Pancreatic Cancer. Retrieved June 21, 2024 from https://www.globenewswire.com/news-release/2024/06/18/2900654/35550/en/XBiotech-Results-from-Randomized-Double-Blinded-Phase-1-2-Study-Suggest-Potential-Breakthrough-Treatment-for-Advanced-Pancreatic-Cancer.html [10] Zealand Pharma announces positive topline results from the Phase 1b 16-week multiple ascending dose clinical trial with long-acting amylin analog petrelintide. Retrieved June 21, 2024 from https://www.globenewswire.com/news-release/2024/06/20/2901879/0/en/Zealand-Pharma-announces-positive-topline-results-from-the-Phase-1b-16-week-multiple-ascending-dose-clinical-trial-with-long-acting-amylin-analog-petrelintide.html [11] Spyre Therapeutics Announces First Participants Dosed in Phase 1 Trial of SPY001, its Novel Half-life Extended anti-α4β7 Antibody, for the Treatment of Inflammatory Bowel Disease. Retrieved June 21, 2024, from https://www.prnewswire.com/news-releases/spyre-therapeutics-announces-first-participants-dosed-in-phase-1-trial-of-spy001-its-novel-half-life-extended-anti-47-antibody-for-the-treatment-of-inflammatory-bowel-disease-302174975.html [12] Taysha Gene Therapies Announces Positive Clinical Data Across Adult and Pediatric Patients from Low Dose Cohort in Ongoing REVEAL Phase 1/2 Trials Evaluating TSHA-102 in Rett Syndrome. Retrieved June 21, 2024, from https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-positive-clinical-data-across/ [13] Assembly Biosciences Doses First Participant in Phase 1b Clinical Trial Evaluating Next-Generation Capsid Assembly Modulator Candidate ABI-4334 for the Treatment of Chronic Hepatitis B Virus Infection. Retrieved June 21, 2024, from https://www.globenewswire.com/news-release/2024/06/18/2900341/16259/en/Assembly-Biosciences-Doses-First-Participant-in-Phase-1b-Clinical-Trial-Evaluating-Next-Generation-Capsid-Assembly-Modulator-Candidate-ABI-4334-for-the-Treatment-of-Chronic-Hepatit.html [14] Cytokinetics Announces Initiation of Phase 1 Study of Aficamten in Healthy Japanese Participants. Retrieved June 21, 2024, from https://ir.cytokinetics.com/news-releases/news-release-details/cytokinetics-announces-initiation-phase-1-study-aficamten [15] Mozart Therapeutics Doses First Cohort of Participants in Phase 1a/b Clinical Trial of MTX-101, in Development for Treatment of Autoimmune Diseases. Retrieved June 21, 2024, from https://www.prnewswire.com/news-releases/mozart-therapeutics-doses-first-cohort-of-participants-in-phase-1ab-clinical-trial-of-mtx101-in-development-for-treatment-of-autoimmune-diseases-302173782.html [16] Zymeworks Announces FDA Clearance Of Investigational New Drug Application For ZW171, A Novel 2+1 T-Cell Targeting Bispecific Antibody For Mesothelin-Expressing Cancers. Retrieved June 21, 2024, from https://ir.zymeworks.com/news-releases/news-release-details/zymeworks-announces-fda-clearance-investigational-new-drug [17] Ryvu Therapeutics presents clinical and preclinical data on RVU120 at the 2024 European Hematology Association Congress. Retrieved June 21, 2024, from https://www.prnewswire.com/news-releases/ryvu-therapeutics-presents-clinical-and-preclinical-data-on-rvu120-at-the-2024-european-hematology-association-congress-302173184.html [18] Sumitomo Pharma Presents New Clinical Data on DSP-5336 at the European Hematology Association 2024 Congress. Retrieved June 21, 2024, from https://www.prnewswire.com/news-releases/sumitomo-pharma-presents-new-clinical-data-on-dsp-5336-at-the-european-hematology-association-2024-congress-302173004.html [19] Zura Bio Presents Data for the Tibulizumab (ZB-106) Program at EULAR 2024. Retrieved June 21, 2024, from https://www.businesswire.com/news/home/20240614812438/en [20] Immune-Onc Therapeutics to Present Additional Positive Interim Data from IO-202 Phase 1b Expansion Cohort in Patients with Chronic Myelomonocytic Leukemia (CMML) at 2024 European Hematology Association (EHA) Annual Congress. Retrieved June 21, 2024, from https://www.businesswire.com/news/home/20240614920813/en [21] Cabaletta Bio Reports Positive Initial Clinical Data from Phase 1/2 RESET-Myositis™ and RESET-SLE™ Trials of CABA-201. Retrieved June 21, 2024, from https://www.globenewswire.com/news-release/2024/06/14/2898772/0/en/Cabaletta-Bio-Reports-Positive-Initial-Clinical-Data-from-Phase-1-2-RESET-Myositis-and-RESET-SLE-Trials-of-CABA-201.html [22] ORYZON Presents Preliminary Data From Ongoing Phase Ib FRIDA Trial with Iadademstat Plus Gilteritinib in Relapsed/Refractory FLT3-mut AML Patients at EHA-2024. Retrieved June 21, 2024, from https://www.globenewswire.com/news-release/2024/06/14/2898942/0/en/ORYZON-Presents-Preliminary-Data-From-Ongoing-Phase-Ib-FRIDA-Trial-with-Iadademstat-Plus-Gilteritinib-in-Relapsed-Refractory-FLT3-mut-AML-Patients-at-EHA-2024.html [23] Enlivex Announces Positive Interim Data Readout from a Phase I/II Trial Evaluating Allocetra in End-Stage Knee Osteoarthritis. Retrieved June 21, 2024, from https://www.globenewswire.com/news-release/2024/06/17/2899579/0/en/Enlivex-Announces-Positive-Interim-Data-Readout-from-a-Phase-I-II-Trial-Evaluating-Allocetra-in-End-Stage-Knee-Osteoarthritis.html [24] Cessation Therapeutics Announces Oral Presentation of Preliminary Findings from First-in-Human Study of Anti-Fentanyl Monoclonal Antibody, CSX-1004. Retrieved June 21, 2024, from https://www.businesswire.com/news/home/20240619579616/en [25] Innate Pharma Shares Updated Results From the Sanofi Developed Blood Cancer Phase 1/2 SAR443579/IPH6101 Trial. Retrieved June 21, 2024, from https://www.businesswire.com/news/home/20240616126553/en [26] Tempest Unveils New Survival Data for Amezalpat (TPST-1120) in Randomized First-Line HCC Study Demonstrating a Six-Month Improvement over Control Arm. Retrieved June 21, 2024, from https://ir.tempesttx.com/news-releases/news-release-details/tempest-unveils-new-survival-data-amezalpat-tpst-1120-randomized [27] Escend Announces Poster Presentation at the Annual Meeting of the European Hematology Association (EHS) from an Investigator-Initiated Phase I/II study evaluating ES-3000 in Myelodysplastic Syndrome (MDS). Retrieved June 21, 2024, from https://www.prnewswire.com/news-releases/escend-announces-poster-presentation-at-the-annual-meeting-of-the-european-hematology-association-ehs-from-an-investigator-initiated-phase-iii-study-evaluating-es-3000-in-myelodysplastic-syndrome-mds-302177666.html [28] Tubulis Doses First Patient in Phase I/IIa Trial Investigating ADC Candidate TUB-040 in Ovarian Cancer and Lung Adenocarcinoma. Retrieved June 21, 2024, from https://www.businesswire.com/news/home/20240620456143/en/ [29] Amezalpat (TPST-1120) Randomized 1L HCC Data Updat. Retrieved June 20, 2024 https://ir.tempesttx.com/static-files/8ba2d665-884e-47f7-89c2-adbba73378b2 [30] Mustang Bio Announces Favorable Efficacy and Safety Data from Complete Waldenstrom Macroglobulinemia Cohort of Phase 1/2 Clinical Trial of MB-106, CD20-Targeted Autologous CAR-T Therapy. Retrieved June 18, 2024 from https://ir.mustangbio.com/news-events/press-releases/detail/180/mustang-bio-announces-favorable-efficacy-and-safety-data 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。 版权说明：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「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新