SRK-181

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today reported financial results and corporate updates for the second quarter ended June 30, 2024. “ Scholar Rock continues to execute across our portfolio of highly selective myostatin inhibition programs, further cementing our position as the global leader in harnessing the life-changing potential of TGF-beta superfamily biology,” said Jay Backstrom, M.D., MPH, President & Chief Executive Officer of Scholar Rock. “ With the only muscle-targeted program to demonstrate clinical proof-of-concept in SMA, our confidence in our lead program apitegromab continues to be supported by the clinical data generated over the past four years. At 48 months, over 90% of TOPAZ patients with nonambulatory SMA remained on apitegromab treatment on top of SMN therapy and we continued to observe sustained clinical benefit. We look forward to reporting topline data from the Phase 3 SAPPHIRE trial of apitegromab in SMA in the fourth quarter of this year.” Dr. Backstrom continued, “ In addition, we are pleased with the progress of our cardiometabolic program. The enrollment of the Phase 2 proof-of-concept EMBRAZE study evaluating apitegromab in obesity has been advancing ahead of schedule and as a result, we are updating our guidance for topline data to the second quarter of 2025. We also presented new preclinical data supporting SRK-439’s potential to help patients retain lean muscle mass at our investor event in May and at the ADA 84 th Scientific Sessions in June. The hallmark of our approach in designing both apitegromab and SRK-439 is the exquisite selectivity for pro- and latent forms of myostatin. Our data in SMA suggest that this selectivity matters for patients, and we are excited to show how SRK-439 can become an integral component of the treatment and management of obesity helping to preserve lean muscle mass for sustainable and healthy weight loss management.” Company Highlights and Upcoming Milestones SMA Program Apitegromab is an investigational, fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle and is being developed as a potential first muscle-targeted therapy for the treatment of SMA. Apitegromab is the only muscle-targeted therapy to show clinical proof-of-concept in SMA. On track to report topline data from Phase 3 SAPPHIRE clinical trial in 4Q 2024 . If the trial is successful and apitegromab is approved, the Company expects to initiate a commercial product launch in 2025. Reported that long-term apitegromab data continued to show substantial and sustained motor function improvements over 48 months 1 . The mean change in Hammersmith Functional Motor Scale (HFMSE) from baseline in nonambulatory patients (ages 2-21) on combination therapy (nusinersen and 20 mg/kg of apitegromab) was 5.3 points (95% CI: 1.5, 9.2; n=23), and for patients 2-12 was 6.4 points (95% CI: 1.8, 11.0, n=19). The mean change in RULM for the 2-21 age group was 3.6 points (95% CI: 2.0, 5.3; N=22) and for the 2-12 age group was 4.5 (95% CI: 2.7, 6.3; n=18). Of the 35 participants in the pooled nonambulatory population, 33 remained in the study over 4 years. The data analysis excluded the scores of 11 patients after undergoing scoliosis surgery, a known confounding factor for motor function assessment. Additional details will be discussed on the conference call this morning. 12 - Month Data 24-Month Data 36-Month Data 48-Month Data Age 2-21 Years Mean Change from Baseline in HFMSE (95% Confidence Interval) 3.6 points (1.2, 6.0) n=32 4.2 points (1.9, 6.6) n=29 4.0 points (1.0, 6.9) n=28 5.3 points (1.5, 9.2) n=23 Age 2-12 Years Mean Change from Baseline in HFMSE (95% Confidence Interval) 4.6 points (1.8, 7.4) n=26 5.2 points (2.3, 8.0) n=23 4.8 points (1.3, 8.3) n=23 6.4 points (1.8, 11.0) n=19 Age 2-21 Years Mean Change from Baseline in RULM (95% Confidence Interval) 1.3 points (0.2, 2.3) n=31 2.3 points (1.2, 3.3) n=31 2.4 points (1.1, 3.7) n=27 3.6 points (2.0, 5.3) n=22 Age 2-12 Years Mean Change from Baseline in RULM (95% Confidence Interval) 1.2 points (0.1, 2.4) n=25 2.2 points (1.0, 3.5) n=25 2.8 points (1.4, 4.2) n=22 4.5 points (2.7, 6.3) n=18 For the 48-month evaluation, an observed case analysis was conducted using available data by analysis timepoint, censoring any HFMSE and RULM assessments after the patient received scoliosis surgery. The analysis population pooled the nonambulatory patients (Cohorts 2 and 3) and included patients receiving either low dose (2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year 2). A total of 11 patients in the population had scoliosis surgery during the study and their data was excluded from any HFMSE or RULM assessments at 48 months. Visit windows were applied to utilize data from unscheduled or early termination visits if the patient was missing the HFMSE or RULM total score at the scheduled visit. The ONYX open-label, multicenter extension study is ongoing. The extension study is evaluating the long-term safety and efficacy of apitegromab in patients with Type 2 and Type 3 SMA who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of patients on combination therapy in the TOPAZ study have completed 4 years of apitegromab treatment and enrolled into ONYX. Cardiometabolic Program SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin A binding), and is initially being developed for the treatment of obesity. Presented preclinical data from the SRK-439 program. In May, the Company announced new preclinical data comparing SRK-439 and an anti-activin receptor II (anti-ActRII) antibody that supported SRK-439’s potential as best in class in preserving lean muscle mass in patients on GLP-1 receptor agonists (GLP-1 RAs). In June, the company presented new preclinical data at the American Diabetes Association 84 th Scientific Sessions supporting the potential of SRK-439 to increase lean mass and contribute to a favorable body composition following withdrawal from GLP-1 RA treatment. Initiated Phase 2 EMBRAZE proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity in May. The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective investigational myostatin inhibitor, to preserve muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Data are expected in the second quarter of 2025 and will be used to guide clinical development of SRK-439. The Company plans to file an IND for SRK-439 for the treatment of obesity in 2025. Other Pipeline Updates New SRK-181 data from the Phase 1 DRAGON proof-of-concept trial presented at the ASCO Annual Meeting in June. SRK-181 is an investigational selective inhibitor of latent TGFβ-1 activation and developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer. Clinical data showed encouraging responses in heavily pretreated and anti-PD-(L)1 resistant patients across multiple tumor types. Enrollment of the DRAGON trial was completed in December 2023, and patients who remain on the study continue to be treated. Published data on SRK-373 was featured on the cover of Science Signaling in July . The article describes the selectivity of SRK-373 for LTBP-presented TGFβ-1, as well as efficacy data from two preclinical models that establish the feasibility of selectively targeting this particular form of TGFβ-1 for the treatment of fibrosis. SRK-373 is an investigational selective inhibitor of matrix associated TGFβ-1 in development for the treatment of fibrosis. Second Quarter 2024 Financial Results For the quarter ended June 30, 2024, net loss was $58.5 million or $0.60 per share compared to a net loss of $37.9 million or $0.47 per share for the quarter ended June 30, 2023. The Company did not record any revenue for the quarter ended June 30, 2024 or for the quarter ended June 30, 2023. Research and development expense was $42.4 million for the quarter ended June 30, 2024, compared to $26.9 million for the quarter ended June 30, 2023. The increase was primarily attributable to clinical trial and employee compensation costs. General and administrative expense was $17.1 million for the quarter ended June 30, 2024, compared to $12.2 million for the quarter ended June 30, 2023. The increase was due to employee-related costs. As of June 30, 2024, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $190.5 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into the second half of 2025. “ Our year-to-date progress across our pipeline of industry-leading myostatin inhibition programs, combined with our highly experienced and disciplined team, provides us with a robust foundation for growth as we advance towards multiple milestones and our potential evolution into a commercial-stage company,” said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock. Conference Call Information Management will provide an update on the Company and discuss second quarter 2024 results via conference call on Thursday, August 8 at 8:15 am ET. To access the live conference call, participants may register here . The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the Scholar Rock website at http://investors.scholarrock.com . To participate via telephone, please register in advance here . 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. An archived replay of the webcast will be available on the Company’s website for approximately 90 days. About Apitegromab Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof-of-concept in spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency. About the Phase 3 SAPPHIRE Trial SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population. These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. For more information about SAPPHIRE, visit www.clinicaltrials.gov . Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established. About EMBRAZE EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating the efficacy, safety and pharmacokinetics of apitegromab in adults with a body mass index (BMI) of >27 (overweight) or a BMI of >30 (obese) and taking a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of EMBRAZE is 100 subjects aged 18-65 who are overweight or obese without diabetes. As part of the study design, the treatment period is 24 weeks, and all subjects will receive a GLP-1 RA. In addition, all subjects will be randomized 1:1 to receive either apitegromab or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. The primary endpoint is change from baseline at Week 24 in lean mass assessed by dual-energy X-ray absorptiometry. Secondary endpoints include additional weight loss measures, safety and tolerability, and pharmacokinetic outcomes. Exploratory endpoints at Weeks 24 and 32 include cardiometabolic parameters (e.g. HbA1c), body composition, and physical function. About SRK-439 SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of cardiometabolic disorders, including obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass during weight loss. The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency. About Scholar Rock Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily of cell proteins and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role. Scholar Rock is the only company to show clinical proof-of-concept for a muscle-targeted treatment in spinal muscular atrophy (SMA). This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn. Availability of Other Information About Scholar Rock Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com , including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn. The information that we post on our website or on Twitter or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended. Scholar Rock ® is a registered trademark of Scholar Rock, Inc. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab and SRK-181 and its preclinical programs, including SRK-439, and indication selection and development timing, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, its cash runway, expectations regarding the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab, or Part A or Part B of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA or Part B of the Phase 1 clinical trial of SRK-181; Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; and Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and our ability to continue as a going concern; as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. Scholar Rock Holding Corporation Condensed Consolidated Statements of Operations (unaudited) (in thousands, except share and per share data) Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Operating expenses Research and development $ 42,373 $ 26,867 $ 85,466 $ 56,602 General and administrative 17,125 12,215 32,451 22,989 Total operating expenses 59,498 39,082 117,917 79,591 Loss from operations (59,498 ) (39,082 ) (117,917 ) (79,591 ) Other income (expense), net 990 1,157 2,556 2,287 Net loss $ (58,508 ) $ (37,925 ) $ (115,361 ) $ (77,304 ) Net loss per share, basic and diluted $ (0.60 ) $ (0.47 ) $ (1.20 ) $ (0.97 ) Weighted average common shares outstanding, basic and diluted 96,813,116 80,117,983 96,352,858 79,865,424 Scholar Rock Holding Corporation Condensed Consolidated Balance Sheets (unaudited) (in thousands) June 30, 2024 December 31, 2023 Assets Cash, cash equivalents and marketable securities $ 190,494 $ 279,938 Other current assets 8,643 8,256 Total current assets 199,137 288,194 Other assets 27,728 22,841 Total assets $ 226,865 $ 311,035 Liabilities and Stockholders' Equity Current liabilities $ 32,987 $ 32,741 Long-term liabilities 60,258 53,076 Total liabilities 93,245 85,817 Total stockholders' equity 133,620 225,218 Total liabilities and stockholders' equity $ 226,865 $ 311,035

▎药明康德内容团队编辑 本期看点 1. 新型口服双重抑制剂TU2218联用PD-1抑制剂pembrolizumab治疗晚期实体瘤患者，在一项1b期临床试验中的疾病控制率（DCR）达100%。 2. 用于治疗遗传性血管水肿（HAE）的在研体内CRISPR基因编辑疗法NTLA-2002早期临床数据亮眼，单次给药后使患者的月度发作率平均下降了98%。 3. 现货型基因疗法PRGN-2012治疗复发性呼吸乳头状瘤病（RRP）患者的1/2期临床试验达到主要终点，有望成为首个获得FDA批准的用于治疗RRP的疗法。 4. 男性无激素避孕药YCT-529完成了1a期临床试验，在临床前研究中，该候选疗法预防小鼠妊娠的有效率为99%，且100%可逆。 药明康德内容团队整理 TU2218：公布1b期临床试验的中期数据 TiumBio公司公布了新型口服双重抑制剂TU2218联用PD-1抑制剂pembrolizumab治疗晚期实体瘤患者1b期临床试验的中期结果。TU2218靶向转化生长因子β受体1（TGFR1）和血管内皮生长因子受体2（VEGFR2）。肿瘤微环境中的TGF-ß和VEGF通路会干扰免疫检查点抑制剂的抗肿瘤活性，因此TU2218有望通过抑制这两种通路最大限度地提高免疫疗法的疗效。 此次公布的结果显示，5名接受2期推荐剂量（RP2D）TU2218联用pembrolizumab治疗的患者的DCR为100%，其中两名患者达到了部分缓解（PR），3名患者达到了疾病稳定（SD）。所有剂量组中，12名接受联合治疗的患者中有2人达到了PR，6人达到了SD。在这项研究中，TU2218联用pembrolizumab的耐受性良好，没有观察到剂量限制性毒性（DLT），这与之前的单药治疗试验一致。 NTLA-2002：公布1期临床试验的长期数据 Intellia Therapeutics公司宣布其用于治疗遗传性血管水肿的在研疗法NTLA-2002的1期临床试验的长期数据。NTLA-2002是一种体内CRISPR基因编辑候选疗法，通过脂质纳米颗粒（LNP）以mRNA形式递送CRISPR-Cas9基因编辑系统，靶向敲除血浆前激肽释放酶（KLKB1）基因，以永久性降低血浆中激肽释放酶活性，从而防止遗传性血管水肿的发作。 此次公布的结果显示，中位随访时间为20.1个月时，单次NTLA-2002治疗让患者的月均疾病发作率平均减少了98%，中度至重度发作平均减少了99%。在测试的每个剂量水平上，HAE发作率均显著减少。单次输注后，患者最长的无发作期超过26个月，并且仍在持续。此外，100%接受NTLA-2002治疗的患者在停止其他预防性治疗后无需再进行预防性治疗。 PRGN-2012：公布1/2期临床试验的新数据 Precigen公司公布了其现货型基因疗法PRGN-2012治疗复发性呼吸乳头状瘤病患者的1/2期临床试验数据。PRGN-2012旨在激发对抗受HPV6或HPV11感染细胞的免疫反应，以治疗RRP。此前，PRGN-2012已获得FDA和欧盟委员会授予的孤儿药资格，还获得了FDA授予的突破性疗法认定。 此次公布的结果显示，该1/2期临床试验达到了主要的安全性和疗效终点。中位随访时间为20个月时，51%（18/35）接受PRGN-2012治疗的患者达到了完全缓解（CR），且不再需要进行手术治疗，CR持续时间超过12个月。86%的患者在接受PRGN-2012治疗后的一年内需要进行的手术次数较治疗前一年减少，从治疗前一年的中位次数4次减少到接受治疗后的0次。此外，PRGN-2012显著改善了达到CR患者的Derkay评分（一种用于测量RRP生长程度的量表）和生活质量评分。安全性方面，PRGN-2012的耐受性良好，无DLT，也没有＞2级的治疗相关不良事件。 ▲PRGN-2012的1/2期临床试验结果（图片来源：参考资料[9]） 该公司计划在2024年下半年通过加速批准途径提交PRGN-2012的滚动生物制品许可申请（BLA）。新闻稿指出，如果获得批准，PRGN-2012有可能成为首个获得FDA批准用于治疗RRP的疗法。 YCT-529：公布1a期临床试验数据 YourChoice Therapeutics公司公布其男性无激素避孕药YCT-529的1a期临床试验结果。YCT-529是一种视黄酸受体-α（RAR-a）抑制剂，通过阻断RAR-α来阻止睾丸中精子的产生和释放。临床前研究表明，YCT-529在预防小鼠妊娠方面有99%的有效性，且100%可逆。该研究的结果表明，YCT-529是安全的。 C-CAR031：公布1期临床试验的初步数据 阿斯利康（AstraZeneca）公布其GPC3靶向CAR-T疗法C-CAR031的最新临床试验结果。GPC3是一种在肝癌中过表达的表面抗原，该蛋白在健康组织中几乎不存在。C-CAR031是一种GPC3靶向的自体CAR-T疗法，这款CAR-T疗法还同时“装备”了TGFβ以抵抗免疫抑制微环境，以增强其效力。 此次公布的结果显示，中位随访时间为5.82个月时，22例可评估疗效的GPC3阳性晚期肝细胞癌（HCC）患者的DCR为90.9%，客观缓解率为50.0%，且该疗法的安全性可控。阿斯利康的早期肿瘤学开发负责人Matt Hellmann博士在接受行业媒体STAT采访时表示：“这是首个在肝癌治疗上显示具疗效的CAR-T疗法。这也是GPC3靶点的首次临床验证。” ▲C-CAR031的疗效结果摘要（图片来源：参考资料[1]） CB-010：公布1期临床试验数据 Caribou Biosciences公司公布了其同种异体CAR-T细胞疗法CB-010治疗复发/难治性B细胞非霍奇金淋巴瘤患者的新数据。CB-010利用基因编辑技术生成，除了表达靶向CD19的嵌合抗原受体，还利用基因编辑技术敲除了免疫检查点蛋白PD-1的表达，旨在提高抗肿瘤活性的持久性。 截至2024年4月1日的数据，CB-010的疗效和安全性可与已获批的自体CAR-T细胞疗法相媲美。13名部分人类白细胞抗原（HLA）匹配（≥4个等位基因）患者的总缓解率达92%，CR率为46%，中位无进展生存期（PFS）获得改善，为14.4个月。 ▲接受CB-010治疗患者的无进展生存曲线（图片来源：参考资料[4]） ▲接受CB-010治疗患者的疗效结果（图片来源：参考资料[4]） INB-200：公布1期临床试验的新数据 IN8bio公司公布其耐化疗的转基因自体γδ T细胞疗法INB-200用于治疗新确诊的多形性胶质母细胞瘤的积极早期数据。INB-200是一种强大的协同治疗方法，能在患者接受化疗时持续存在，并保持其识别、参与和杀死癌细胞的自然能力。 截至2024年5月30日的数据，中位随访时间为11.7个月，92%接受INB-200治疗的可评估患者的中位PFS超过了7个月（即接受标准治疗方案的患者的中位PFS）。一名患有IDH突变神经胶质瘤的患者在接受治疗后的34.9+个月时仍然存活，且疾病没有进展。近期发表的一项IDH抑制剂治疗IDH突变脑胶质瘤患者的临床试验中，对照组患者的中位PFS为11.1个月，试验组患者的中位PFS为28.5个月。安全性方面，在任何队列中均未报告与治疗相关的严重不良事件、DLT、细胞因子释放综合征、输注反应或免疫效应细胞相关神经毒性综合征。 IFx-2.0：公布1b期临床试验数据 TuHURA Biosciences公司公布了其个体化的候选癌症疫苗IFx-2.0治疗对免疫检查点抑制剂（ICI）耐药的晚期Merkel细胞癌（MCC）和皮肤鳞状细胞癌（cSCC）患者的1b期试验的初步结果。该候选疗法旨在克服患者对ICI的原发性耐药。 此次公布的结果显示，连续三周每周给药一次IFx-2.0的耐受性良好。5名此前接受ICI治疗后在3.8个月内疾病就发生进展的晚期MCC患者中，有4人在接受IFx-2.0治疗后重新对ICI治疗产生了持久的应答，包括1例CR、1例病理完全缓解（pCR）和两例PR。患者的平均缓解时间为25个月，其中两名患者已分别持续缓解19个月和23个月，并且仍在持续缓解中。 MDNA11：公布1/2期临床试验的新数据 Medicenna Therapeutics公司公布了其下一代长效IL-2超级激动剂MDNA11治疗晚期实体瘤的新临床结果。MDNA11具有优秀的CD122（IL-2受体β）结合作用，而没有CD25（IL-2受体α）亲和力，从而能优先刺激癌症杀伤效应T细胞和NK细胞。 此次公布的结果显示，MDNA11的耐受性良好，没有报告DLT和血管渗漏综合征。MDNA11在胰腺癌患者中表现出持久的缓解，1例转移性胰腺导管腺癌患者在基线时存在的所有病灶均完全消退，已维持超过104周，在停止治疗4个月后仍然保持着缓解。一例双检查点抑制剂治疗后进展的黑色素瘤患者的靶病灶在第28周时完全消退，在第44周的扫描时靶病灶仍保持完全消退。目前，该患者仍在接受MDNA11的治疗，非靶病灶也在继续消退。 WTX-124：公布1/1b期临床试验数据 Werewolf Therapeutics公司公布了其条件激活的白介素-2（IL-2）候选疗法WTX-124治疗接受过ICI治疗的局部晚期或转移性实体瘤患者的1/1b期临床试验数据。结果显示，WTX-124在此类患者中具有临床活性，且耐受性良好。截至2024年5月1日的数据，接受WTX-124单药治疗的患者中有1人获得了持久并经过确认的CR，2人达到了PR。获得缓解的患者在治疗的前两个治疗周期内就出现了缓解，并且靶病灶实现了100%的消退。此外，没有发现WTX-124联用pembrolizumab出现新的安全信号。 ▲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] Phase I study of C-CAR031, a GPC3-specific TGFβRIIDN armored autologous CAR-T, in patients with advanced hepatocellular carcinoma (HCC). Retrieved June 4, 2024 from https://meetings.asco.org/abstracts-presentations/234377 [2] Virion Therapeutics Reports Positive First-in-human, First-in-class, Phase 1b Immunogenicity Data of VRON-0200, a Novel Checkpoint Modifier for HBV Functional Cure at EASL Congress. Retrieved June 5, 2024, from https://www.prnewswire.com/news-releases/virion-therapeutics-reports-positive-first-in-human-first-in-class-phase-1b-immunogenicity-data-of-vron-0200-a-novel-checkpoint-modifier-for-hbv-functional-cure-at-easl-congress-302161948.html [3] TiumBio Presents Phase 1b Interim Results for TU2218 in Combination with Pembrolizumab at ASCO 2024 Annual Meeting. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/tiumbio-presents-phase-1b-interim-results-for-tu2218-in-combination-with-pembrolizumab-at-asco-2024-annual-meeting-302160236.html [4] Caribou Biosciences Presents Encouraging Clinical Data from CB-010 ANTLER Phase 1 Trial in Second-line LBCL Patients at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/02/2891868/0/en/Caribou-Biosciences-Presents-Encouraging-Clinical-Data-from-CB-010-ANTLER-Phase-1-Trial-in-Second-line-LBCL-Patients-at-the-2024-American-Society-of-Clinical-Oncology-ASCO-Annual-M.html [5] Werewolf Therapeutics to Present Data from Ongoing Phase 1/1b Clinical Trial of WTX-124 as Monotherapy and in Combination with Pembrolizumab in Solid Tumors. Retrieved June 7, 2024, from https://investors.werewolftx.com/news-releases/news-release-details/werewolf-therapeutics-present-data-ongoing-phase-11b-clinical [6] Medicenna To Present Evidence of Durable Single Agent Activity and Potent Immune Effector Response with MDNA11 in the Dose Escalation Portion of Phase 1/2 ABILITY-1 Study at the 10th Annual Oncology Innovation Forum. Retrieved June 7, 2024, from https://ir.medicenna.com/news-releases/news-release-details/medicenna-present-evidence-durable-single-agent-activity-and [7] Intellia Therapeutics Announces Positive Long-Term Data from Ongoing Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE). Retrieved June 3, 2024, from https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-positive-long-term-data-ongoing [8] TuHURA Biosciences and Kintara Therapeutics Announce Positive Results from Phase 1b Trial of IFx-2.0, a Novel Personalized Cancer Vaccine, in Checkpoint Inhibitor Resistant Advanced Merkel Cell Carcinoma (MCC) and Cutaneous Squamous Cell Carcinoma (cSCC). Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/tuhura-biosciences-and-kintara-therapeutics-announce-positive-results-from-phase-1b-trial-of-ifx-2-0--a-novel-personalized-cancer-vaccine-in-checkpoint-inhibitor-resistant-advanced-merkel-cell-carcinoma-mcc-and-cutaneous-squamo-302161301.html [9] Precigen Announces Groundbreaking Pivotal Study Data for PRGN-2012 in Patients with Recurrent Respiratory Papillomatosis in Which More than Half of Patients Achieved Complete Response. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/precigen-announces-groundbreaking-pivotal-study-data-for-prgn-2012-in-patients-with-recurrent-respiratory-papillomatosis-in-which-more-than-half-of-patients-achieved-complete-response-302161493.html [10] YourChoice Therapeutics Completes Phase 1a Clinical Study for First Hormone-Free Male Birth Control Pill. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603886060/en/ [11] IN8bio Presents Progression-Free Survival Update from Phase 1 Study of INB-200 at 2024 American Society of Clinical Oncology Annual Meeting. Retrieved June 7, 2024, from https://investors.in8bio.com/news-releases/news-release-details/in8bio-presents-progression-free-survival-update-phase-1-study [12] 4DMT Presents Positive Interim Data from Phase 1/2 AEROW Clinical Trial of Aerosolized 4D-710 for Modulator-Ineligible/-Intolerant Cystic Fibrosis at 47th European Cystic Fibrosis Conference. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/06/2894575/0/en/4DMT-Presents-Positive-Interim-Data-from-Phase-1-2-AEROW-Clinical-Trial-of-Aerosolized-4D-710-for-Modulator-Ineligible-Intolerant-Cystic-Fibrosis-at-47th-European-Cystic-Fibrosis-C.html [13] Mythic Therapeutics Presents Initial Dose Escalation Data from Ongoing Phase 1 KisMET-01 Study on MYTX-011 at the American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603700748/en [14] Syndax Announces Plans to Advance into Phase 1b Portion of Trial Evaluating Revumenib in Relapsed or Refractory Metastatic MSS CRC. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/syndax-announces-plans-to-advance-into-phase-1b-portion-of-trial-evaluating-revumenib-in-relapsed-or-refractory-metastatic-mss-crc-302165405.html [15] BridgeBio Oncology Therapeutics announces first patient dosed with BBO-8520 in the Ph. 1 ONKORAS-101 trial for KRASG12C NSCLC. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240606977682/en/ [16] Vivet Therapeutics Presents Interim Data from its Phase 1/2 GATEWAY Trial for the Treatment of Wilson Disease at EASL Congress 2024. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/vivet-therapeutics-presents-interim-data-from-its-phase-12-gateway-trial-for-the-treatment-of-wilson-disease-at-easl-congress-2024-302164666.html [17] Altimmune Presents Data at EASL International Liver Congress™ 2024 Supporting the Disease Modifying Potential and Differentiated Therapeutic Profile of Pemvidutide in Metabolic Dysfunction-Associated Steatohepatitis (MASH). Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/6/5/2893731/0/en/Altimmune-Presents-Data-at-EASL-International-Liver-Congress-2024-Supporting-the-Disease-Modifying-Potential-and-Differentiated-Therapeutic-Profile-of-Pemvidutide-in-Metabolic-Dysf.html [18] Quell Therapeutics Advances QEL-001, its Multi-modular Engineered CAR-Treg Cell Therapy, into Efficacy Cohort of LIBERATE Phase 1/2 Trial in Liver Transplant Patients. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/04/2893353/0/en/Quell-Therapeutics-Advances-QEL-001-its-Multi-modular-Engineered-CAR-Treg-Cell-Therapy-into-Efficacy-Cohort-of-LIBERATE-Phase-1-2-Trial-in-Liver-Transplant-Patients.html [19] Surrozen Initiates Dosing of First Patient in Phase 1b Clinical Trial of SZN-043 for Severe Alcohol-Associated Hepatitis. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/04/2893050/0/en/Surrozen-Initiates-Dosing-of-First-Patient-in-Phase-1b-Clinical-Trial-of-SZN-043-for-Severe-Alcohol-Associated-Hepatitis.html [20] Cue Biopharma Presents Updated Data from Phase 1 Trial of CUE-101 in Recurrent/Metastatic HPV+ Head and Neck Cancer at the 2024 ASCO Annual Meeting. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/04/2892974/0/en/Cue-Biopharma-Presents-Updated-Data-from-Phase-1-Trial-of-CUE-101-in-Recurrent-Metastatic-HPV-Head-and-Neck-Cancer-at-the-2024-ASCO-Annual-Meeting.html [21] Merus Presents Interim Data on MCLA-145 Monotherapy and in Combination with Pembrolizumab at the 2024 ASCO® Annual Meeting. Retrieved June 7, 2024, from https://ir.merus.nl/news-releases/news-release-details/merus-presents-interim-data-mcla-145-monotherapy-and-combination [22] BlueSphere Bio Announces IND Clearance of its First in Human Candidate and New Cell Therapy Portfolio for High-Risk Leukemia Patients. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892552/0/en/BlueSphere-Bio-Announces-IND-Clearance-of-its-First-in-Human-Candidate-and-New-Cell-Therapy-Portfolio-for-High-Risk-Leukemia-Patients.html [23] Grey Wolf Therapeutics Presents First Clinical Data for GRWD5769, a First-in-Class ERAP1 Inhibitor, at the 2024 American Society for Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/grey-wolf-therapeutics-presents-first-clinical-data-for-grwd5769-a-first-in-class-erap1-inhibitor-at-the-2024-american-society-for-clinical-oncology-asco-annual-meeting-302161297.html [24] Puma Biotechnology Announces Presentation of Findings from a Phase I/Ib Study of Alisertib in Advanced EGFR-Mutated Lung Cancer. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603691086/en [25] Cyclacel Pharmaceuticals Reports New Clinical Data at 2024 ASCO Annual Meeting Highlighting Oral Fadraciclib’s Potential as a Precision Medicine for Cancer. Retrieved June 7, 2024, from https://investor.cyclacel.com/news-releases/news-release-details/cyclacel-pharmaceuticals-reports-new-clinical-data-2024-asco [26] Obsidian Therapeutics Announces Additional OBX-115 Safety and Efficacy Data in Oral Presentation at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603008329/en [27] Replimune Presents RP1 Data from the IGNYTE anti-PD1 Failed Melanoma Cohort and RP2 Data in Uveal Melanoma at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://ir.replimune.com/news-releases/news-release-details/replimune-presents-rp1-data-ignyte-anti-pd1-failed-melanoma [28] RS Oncology Announces Positive Data from a Phase 1 Clinical Trial of RSO-021, a First-in-Class Therapeutic for Malignant Pleural Mesothelioma. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603559175/en [29] Ascendis Pharma Presents New Data and Updated Results from Phase 1/2 IL-Believe Trial at ASCO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892565/0/en/Ascendis-Pharma-Presents-New-Data-and-Updated-Results-from-Phase-1-2-IL-Believe-Trial-at-ASCO-2024.html [30] Scholar Rock Presents New Data from SRK-181 Phase 1 DRAGON Trial at ASCO 2024 Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603004778/en [31] invoX Pharma Presents Positive Clinical Data from Phase 1 Study of FS222 in Patients with Advanced Solid Tumours at the 2024 American Society of Clinical Oncology Annual Meeting. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603068133/en [32] Eledon Presents Updated Data from Ongoing Phase 1b Trial Evaluating Tegoprubart for Prevention of Rejection in Kidney Transplantation. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892558/0/en/Eledon-Presents-Updated-Data-from-Ongoing-Phase-1b-Trial-Evaluating-Tegoprubart-for-Prevention-of-Rejection-in-Kidney-Transplantation.html [33] ImmuneOncia Announces Biomarker Results from Phase 1 Clinical Trial of CD47 Antibody at ASCO. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240603017832/en [34] Entera Bio Reports Phase 1 Clinical Data of First-in-Class, Oral PTH(1-34) Peptide Candidate (EB612) for Patients with Hypoparathyroidism at ENDO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892199/0/en/Entera-Bio-Reports-Phase-1-Clinical-Data-of-First-in-Class-Oral-PTH-1-34-Peptide-Candidate-EB612-for-Patients-with-Hypoparathyroidism-at-ENDO-2024.html [35] Enlivex Receives Regulatory Authorization for the Initiation of a Placebo-Controlled Phase I/II Trial Evaluating Allocetra in Up To 46 Patients with Thumb Osteoarthritis. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892206/0/en/Enlivex-Receives-Regulatory-Authorization-for-the-Initiation-of-a-Placebo-Controlled-Phase-I-II-Trial-Evaluating-Allocetra-in-Up-To-46-Patients-with-Thumb-Osteoarthritis.html [36] Indaptus Therapeutics Announces New Positive Data from Ongoing Phase 1 Trial of Decoy20. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892212/0/en/Indaptus-Therapeutics-Announces-New-Positive-Data-from-Ongoing-Phase-1-Trial-of-Decoy20.html [37] Alkermes Presents Data From Phase 1b Study of ALKS 2680 Demonstrating Improved Wakefulness in Patients With Narcolepsy Type 1 at SLEEP 2024. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/alkermes-presents-data-from-phase-1b-study-of-alks-2680-demonstrating-improved-wakefulness-in-patients-with-narcolepsy-type-1-at-sleep-2024-302161336.html [38] IDRx Reports Updated Preliminary Phase 1 Data from Ongoing Phase 1/1b StrateGIST 1 Trial Supporting Best-in-Class Potential for IDRX-42 in Patients with GIST. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240531639390/en [39] Century Therapeutics Presents Interim Results from Phase 1 ELiPSE-1 Study at ASCO 2024 Annual Meeting. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892097/0/en/Century-Therapeutics-Presents-Interim-Results-from-Phase-1-ELiPSE-1-Study-at-ASCO-2024-Annual-Meeting.html [40] MediciNova Announces Data from Phase 1b/2a Clinical Trial of MN-166 (ibudilast) in Glioblastoma Patients at the American Society of Clinical Oncology (ASCO) Annual Meeting 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892064/7767/en/MediciNova-Announces-Data-from-Phase-1b-2a-Clinical-Trial-of-MN-166-ibudilast-in-Glioblastoma-Patients-at-the-American-Society-of-Clinical-Oncology-ASCO-Annual-Meeting-2024.html [41] FibroGen Announces FDA Clearance of Investigational New Drug Application for FG-3165, a Galectin-9 Targeting Monoclonal Antibody, for the Treatment of Patients with Solid Tumors. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/03/2892568/0/en/FibroGen-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-FG-3165-a-Galectin-9-Targeting-Monoclonal-Antibody-for-the-Treatment-of-Patients-with-Solid-Tumors.html [42] STORM Therapeutics Presented Interim Phase 1 Clinical Data on its METTL3 RNA Methyltransferase Inhibitor STC-15 at ASCO 2024. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/storm-therapeutics-presented-interim-phase-1-clinical-data-on-its-mettl3-rna-methyltransferase-inhibitor-stc-15-at-asco-2024-302160864.html [43] Black Diamond Therapeutics Presents Promising BDTX-1535 Clinical Data in Patients with Recurrent Glioblastoma at 2024 American Society of Clinical Oncology (ASCO) Annual Meeting. Retrieved June 7, 2024, from https://investors.blackdiamondtherapeutics.com/news-releases/news-release-details/black-diamond-therapeutics-presents-promising-bdtx-1535-clinical [44] Immunocore reports updated Phase 1 data of brenetafusp (IMC-F106C), an ImmTAC bispecific targeting PRAME, in immune checkpoint pre-treated cutaneous melanoma patients at ASCO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/05/31/2891735/0/en/Immunocore-reports-updated-Phase-1-data-of-brenetafusp-IMC-F106C-an-ImmTAC-bispecific-targeting-PRAME-in-immune-checkpoint-pre-treated-cutaneous-melanoma-patients-at-ASCO-2024.html [45] Kymera Therapeutics Presents New Clinical Data from Ongoing Phase 1 Trial of MDM2 Degrader KT-253 at ASCO Annual Meeting. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/01/2891812/0/en/Kymera-Therapeutics-Presents-New-Clinical-Data-from-Ongoing-Phase-1-Trial-of-MDM2-Degrader-KT-253-at-ASCO-Annual-Meeting.html [46] Accutar Biotechnology Presents Phase 1 Data of AC699 Monotherapy in Patients with ER+ / HER2- Breast Cancer at ASCO 2024. Retrieved June 7, 2024, from https://www.businesswire.com/news/home/20240601126229/en [47] Molecular Partners Presents Positive Data From Completed Phase 1 Trial Of MP0317 (FAP X CD40 DARPin) Monotherapy In Patients With Advanced Solid Tumors At ASCO 2024. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/01/2891809/0/en/Molecular-Partners-Presents-Positive-Data-From-Completed-Phase-1-Trial-Of-MP0317-FAP-X-CD40-DARPin-Monotherapy-In-Patients-With-Advanced-Solid-Tumors-At-ASCO-2024.html [48] Updated Data from the Phase 1/2 Study of Olomorasib in KRAS G12C-Mutant Advanced Solid Tumors Presented at the 2024 ASCO® Annual Meeting. Retrieved June 7, 2024, from https://www.prnewswire.com/news-releases/updated-data-from-the-phase-12-study-of-olomorasib-in-kras-g12c-mutant-advanced-solid-tumors-presented-at-the-2024-asco-annual-meeting-302160829.html [49] ALX Oncology Presents First Evorpacept Combination Data with an Antibody-Drug Conjugate from Phase 1 ASPEN-07 Clinical Trial in Patients with Advanced Bladder Cancer. Retrieved June 7, 2024, from https://www.globenewswire.com/news-release/2024/06/02/2891851/0/en/ALX-Oncology-Presents-First-Evorpacept-Combination-Data-with-an-Antibody-Drug-Conjugate-from-Phase-1-ASPEN-07-Clinical-Trial-in-Patients-with-Advanced-Bladder-Cancer.html 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