本期看點

1. 抗體偶聯藥物（ADC）CX-2051在晚期結直腸癌（CRC）中取得了積極的1期臨床試驗結果，總緩解率（ORR）為28%，疾病控制率（DCR）達94%。

2. IL-4Rα靶向單抗APG808在治療哮喘的1b期臨床試驗中表現亮眼，該療法在12周內對一種與哮喘惡化相關的2型炎癥生物標志物具有強效且持續的抑制作用，有望支持其實現每兩個月或更長時間一次的給藥方案。

3. FDA批準了Lantern Pharma公司的新型“合成致死”小分子藥物LP-184的IND申請。

CX-2051：公布1期臨床試驗的中期數據

CytomX Therapeutics公布了其ADC療法CX-2051在晚期結直腸癌（CRC）中的積極1期中期數據。CX-2051是一種條件激活型ADC，其使用的細胞毒性有效載荷是喜樹堿的衍生物。喜樹堿是一種拓撲異構酶-1抑制劑，這類藥物已在ADC領域針對多個靶點顯示出強大的臨床抗癌活性。CX-2051已在包括結直腸癌在內的多個臨床前模型中顯示出強大的臨床前活性和耐受性。

截至2025年4月7日的數據，ORR為28%（5/18），其中10 mg/kg劑量組的ORR達到43%（3/7），顯著高于目前三線及三線以上結直腸癌治療藥物的緩解率（通常僅有個位數的百分比）。DCR為94%（17/18），中位無進展生存期為5.8個月。截至數據截止時，18名患者中有10名仍在接受治療。安全性方面，CX-2051的耐受性良好，未出現劑量限制性毒性（DLT），大多數治療相關不良事件為1級或2級。

APG808：公布1b期臨床試驗的中期數據

Apogee Therapeutics公司公布了其候選單抗APG808用于治療輕度至中度哮喘患者的1b期臨床試驗的積極中期數據。APG808是一種新型、皮下注射、半衰期延長的IL-4Rα靶向單抗，具有用于治療哮喘、慢性阻塞性肺病（COPD）及其他炎癥與免疫學適應癥的潛力。IL-4Rα是一個已在八種2型過敏性疾病中得到臨床驗證的靶點。在1期臨床試驗中，APG808的藥代動力學（PK）特征有望支持每2-3個月一次的維持治療給藥方案。

此次公布的結果顯示，多劑量給藥APG808可使FeNO（一種與哮喘惡化相關的2型炎癥生物標志物）較基線大幅下降53%，并在12周時仍維持約50%的抑制水平，顯示出對哮喘的持續控制能力。APG808的優化配方和潛在“best-in-class”的PK特征，結合在12周內對FeNO的強效且持續的抑制作用，有望支持其實現每兩個月或更長時間一次的給藥方案，相較于目前每兩周一次的標準治療具有優勢。此外，APG808整體耐受性良好，安全性特征與已有的抗IL-4Rα療法一致。

Soquelitinib：公布1期臨床試驗中隊列1-3的數據

Corvus Pharmaceuticals公司公布了其小分子白細胞介素-2誘導的T細胞激酶（ITK）抑制劑soquelitinib用于治療中度至重度特應性皮炎的1期臨床試驗數據。ITK是一種主要表達于T細胞中的酶，在T細胞和自然殺傷（NK）細胞的免疫功能中起重要作用。通過抑制ITK，soquelitinib有望抑制自身免疫和炎癥反應。

此次公布的結果顯示，所有3個隊列中，soquelitinib治療組在具有臨床意義的終點濕疹面積和嚴重度指數較基線減少75%（EASI 75）和研究者總體評估（IGA）評分為0/1（皮膚癥狀清除或幾乎清除）上，均顯示出相較于安慰劑的顯著療效。與隊列1和隊列2（100 mg每天兩次和200 mg每天一次，每日總劑量200 mg）相比，隊列3（200 mg每天兩次，每日總劑量400 mg）的緩解更早且更深。安全性方面，soquelitinib的耐受性良好，在所有隊列中均未觀察到DLT，也未出現具有臨床意義的實驗室指標異常。此外，所有隊列中均未發生中斷給藥的情況。

▲治療第28天達到終點IGA 0/1、EASI 75的患者占比（圖片來源：參考資料[1]）

ELVN-001：公布1期臨床試驗的新數據

Enliven Therapeutics公司公布其小分子激酶抑制劑ELVN-001治療慢性粒細胞白血病（CML）患者的1期臨床試驗的新數據。ELVN-001是一種強效、高選擇性、潛在“best-in-class”的小分子激酶抑制劑，專門針對CML患者的致癌驅動因子BCR-ABL融合蛋白。ELVN-001還具有針對耐藥性最強的BCR-ABL1耐藥突變體T315I和其他已知耐藥突變體的活性。

截至2025年1月21日的數據，在36例可評估的患者中，44%（16/36）在24周時達到主要分子學緩解（MMR），其中最后一線治療時對酪氨酸激酶抑制劑（TKI）耐藥的患者中有40%（10/25）達到了MMR，曾接受asciminib或ponatinib治療的患者中也有36%（9/25）達到MMR，包括1例攜帶已知asciminib耐藥突變的患者。所有達到或維持MMR的患者在數據截止時仍保持緩解。在該臨床試驗中所納入的患者群體此前接受過大量治療的情況下，ELVN-001與已獲批的針對BCR-ABL融合蛋白的TKI在1期試驗中所觀察到的MMR相比，結果依然更好。安全性方面，ELVN-001在所有劑量組中仍表現出良好的耐受性，與其選擇性激酶譜一致。

LP-184：IND申請獲得FDA許可

Lantern Pharma公司宣布，FDA已批準其抗癌新藥LP-184的IND申請。LP-184將與免疫檢查點抑制劑聯用，針對攜帶

KEAP1

STK11

KEAP1

參考資料（可上下滑動查看）

[1] Corvus Pharmaceuticals Announces Data from Cohorts 1-3 of Placebo-Controlled Phase 1 Clinical Trial of Soquelitinib for Atopic Dermatitis. Retrieved May 16, 2025, from https://investor.corvuspharma.com/news-releases/news-release-details/corvus-pharmaceuticals-announces-data-cohorts-1-3-placebo

[2] MaaT Pharma Announces Promising Final Data Readout for Phase 1b Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS). Retrieved May 16, 2025, from https://www.businesswire.com/news/home/20250511855391/en/MaaT-Pharma-Announces-Promising-Final-Data-Readout-for-Phase-1b-Evaluating-MaaT033-in-Amyotrophic-Lateral-Sclerosis-ALS

[3] Cantargia announces successful Phase 1 results: PK/PD data of subcutaneously administered CAN10 confirms every 4-week dosing choice in Phase 2. Retrieved May 16, 2025, from https://cantargia.com/en/press-releases/cantargia-announces-successful-phase-1-results-pk-pd-data-of-subcutaneously-administered-can10-confirms-every-4-week-dosing-choice-in-phase-2

[4] Apogee Therapeutics Announces Positive Interim Results from the Phase 1b Trial of APG808, its Novel Half-life Extended IL-4Rα Antibody, in Patients with Mild-to-Moderate Asthma. Retrieved May 16, 2025, from https://www.globenewswire.com/news-release/2025/05/12/3078912/0/en/Apogee-Therapeutics-Announces-Positive-Interim-Results-from-the-Phase-1b-Trial-of-APG808-its-Novel-Half-life-Extended-IL-4R%CE%B1-Antibody-in-Patients-with-Mild-to-Moderate-Asthma.html

[5] CytomX Announces Positive Interim Data From Phase 1 Dose Escalation Study of EpCAM Antibody Drug Conjugate (CX-2051) Candidate in Patients with Advanced Colorectal Cancer (CRC). Retrieved May 16, 2025, from https://www.globenewswire.com/news-release/2025/05/12/3078935/37704/en/CytomX-Announces-Positive-Interim-Data-From-Phase-1-Dose-Escalation-Study-of-EpCAM-Antibody-Drug-Conjugate-CX-2051-Candidate-in-Patients-with-Advanced-Colorectal-Cancer-CRC.html

[6] MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome. Retrieved May 16, 2025, from https://www.prnewswire.com/news-releases/mavrix-bio-announces-fda-clearance-of-ind-application-to-initiate-first-in-human-study-of-gene-therapy-for-angelman-syndrome-302451763.html

[7] Ensoma Announces FDA Clearance of IND Application for First In Vivo HSC-Directed Gene Insertion Therapy. Retrieved May 16, 2025, from https://ensoma.com/press-release/ensoma-announces-fda-clearance-of-ind-application-for-first-in-vivo-hsc-directed-gene-insertion-therapy/

[8] Faron Pharmaceuticals presents promising Phase 1/2 data from BEXMAB Study at MDS 2025 plenary session. Retrieved May 16, 2025, from https://faron.com/releases-and-publications/faron-pharmaceuticals-presents-promising-phase-1-2-data-from-bexmab-study-at-mds-2025-plenary-session/

[9] Lantern Pharma Secures FDA Clearance for Planned Phase 1b/2 Trial of LP-184 in Biomarker-Defined, Treatment-Resistant NSCLC Patients with High Unmet Clinical Need. Retrieved May 16, 2025, from https://ir.lanternpharma.com/news-events/press-releases/detail/179/lantern-pharma-secures-fda-clearance-for-planned-phase-1b2

[10] Innovo Therapeutics Announces Positive Phase 1 Results for INV-101, a Novel Immuno-Metabolic Modulator for Autoimmune Diseases. Retrieved May 16, 2025, from https://www.businesswire.com/news/home/20250512131551/en/Innovo-Therapeutics-Announces-Positive-Phase-1-Results-for-INV-101-a-Novel-Immuno-Metabolic-Modulator-for-Autoimmune-Diseases

[11] Capsida Receives FDA IND Clearance for Its First-in-Class, IV-administered Gene Therapy for STXBP1 Developmental and Epileptic Encephalopathy. Retrieved May 16, 2025, from https://capsida.com/capsida-receives-fda-ind-clearance-for-its-first-in-class-iv-administered-gene-therapy-for-stxbp1-developmental-and-epileptic-encephalopathy/

[12] AAVantgarde presents positive clinical data from its AAVB-081 program for Usher 1B and preclinical data from its AAVB-039 program for Stargardt at the ARVO 2025 annual meeting. Retrieved May 16, 2025, from https://www.globenewswire.com/news-release/2025/05/12/3078987/0/en/AAVantgarde-presents-positive-clinical-data-from-its-AAVB-081-program-for-Usher-1B-and-preclinical-data-from-its-AAVB-039-program-for-Stargardt-at-the-ARVO-2025-annual-meeting.html

[13] Artiva Biotherapeutics Announces Longer-term Phase 1/2 Data Demonstrating Prolonged Durability for AlloNK? in Combination with Rituximab in Patients with B-cell-Non-Hodgkin Lymphoma at the ASGCT 28th Annual Meeting. Retrieved May 16, 2025, from https://www.globenewswire.com/news-release/2025/05/13/3080625/0/en/Artiva-Biotherapeutics-Announces-Longer-term-Phase-1-2-Data-Demonstrating-Prolonged-Durability-for-AlloNK-in-Combination-with-Rituximab-in-Patients-with-B-cell-Non-Hodgkin-Lymphoma.html

[14] Sernova Biotherapeutics Provides Positive Interim Data from Ongoing Phase 1/2 Clinical Trial of Cell Pouch Bio-hybrid Organ in Patients Living with Type 1 Diabetes. Retrieved May 16, 2025, from https://www.globenewswire.com/news-release/2025/05/14/3080975/0/en/Sernova-Biotherapeutics-Provides-Positive-Interim-Data-from-Ongoing-Phase-1-2-Clinical-Trial-of-Cell-Pouch-Bio-hybrid-Organ-in-Patients-Living-with-Type-1-Diabetes.html

[15] Enliven Therapeutics Announces Updated Positive Data from Phase 1 Clinical Trial of ELVN-001 in CML and Oral Presentation at the EHA 2025 Congress. Retrieved May 16, 2025, from https://www.prnewswire.com/news-releases/enliven-therapeutics-announces-updated-positive-data-from-phase-1-clinical-trial-of-elvn-001-in-cml-and-oral-presentation-at-the-eha-2025-congress-302455412.html

[16] AbCellera Receives Authorization from Health Canada to Initiate the Phase 1 Clinical Trial of ABCL635 for Vasomotor Symptoms Due to Menopause. Retrieved May 16, 2025, from https://investors.abcellera.com/news/news-releases/2025/AbCellera-Receives-Authorization-from-Health-Canada-to-Initiate-the-Phase-1-Clinical-Trial-of-ABCL635-for-Vasomotor-Symptoms-Due-to-Menopause/default.aspx

[17] Biomea Fusion’s BMF-500 Selected for Poster Presentation at EHA 2025 Highlighting Phase I Data in Relapsed/Refractory Acute Leukemia. Retrieved May 16, 2025, from https://www.globenewswire.com/news-release/2025/05/14/3081603/0/en/Biomea-Fusion-s-BMF-500-Selected-for-Poster-Presentation-at-EHA-2025-Highlighting-Phase-I-Data-in-Relapsed-Refractory-Acute-Leukemia.html

[18] Athira Pharma to Present Data from First-in-Human Phase 1 Clinical Trial of ATH-1105 at the 4th Annual ALS Drug Development Summit. Retrieved May 16, 2025, from https://investors.athira.com/news-releases/news-release-details/athira-pharma-present-data-first-human-phase-1-clinical-trial

[19] Rocket Pharmaceuticals Presents Preliminary Data from Phase 1 Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy at 28th Annual Meeting of the American Society of Gene and Cell Therapy. Retrieved May 16, 2025, from https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-preliminary-data-phase-1

[20] START Doses First U.S. Patient in Moderna's Phase 1 Clinical Trial of mRNA-4106, a Pan-Tumor Antigen Therapy Candidate, in Solid Tumors. Retrieved May 16, 2025, from https://www.prnewswire.com/news-releases/start-doses-first-us-patient-in-modernas-phase-1-clinical-trial-of-mrna-4106-a-pan-tumor-antigen-therapy-candidate-in-solid-tumors-302455728.html

[21] Pasithea Therapeutics Announces Initiation of Phase 1/1B Study of PAS-004 in Adult NF1 Patients and Activation of First Clinical Trial Site. Retrieved May 16, 2025, from https://ir.pasithea.com/news-events/press-releases/detail/120/pasithea-therapeutics-announces-initiation-of-phase-11b

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