三特異性NK細胞銜接器、創新JAK2變構抑制劑獲FDA批準進入臨床…… | 一周盤點

本期看點：

1. GT Biopharma公司宣布，其新型B7-H3靶向三特異性自然殺傷（NK）細胞銜接器GTB-5550的IND申請已獲得美國FDA批準， 將在7種不同的轉移性癌癥隊列中開展研究。

2. Prelude Therapeutics公司宣布，其新型JAK2V617F突變選擇性抑制劑PRT12396的IND申請已獲美國FDA批準，1期臨床試驗將在 高危真紅細胞增多癥（PV）以及中高危骨髓纖維化（MF）患者中開展。

GTB-5550：IND申請獲得FDA許可

GT Biopharma公司宣布，其新型候選藥物GTB-5550的IND申請已獲得美國FDA批準。GTB-5550是一種靶向B7-H3的三特異性NK細胞銜接器，用于治療表達B7-H3抗原的實體腫瘤。該分子由三種組分組成，通過柔性接頭連接：1）靶向NK細胞表面CD16激活受體的納米體臂；2）野生型IL-15（WT IL-15）連接臂，用于促進NK細胞的增殖、活化和存活；3）靶向腫瘤細胞上B7-H3抗原的納米體臂，從而將NK細胞精準引導至腫瘤部位。

計劃開展的1期臨床試驗共分為兩個部分：1a期為劑量遞增階段，將評估最多6個劑量水平以確定最大耐受劑量（MTD）；1b期為劑量擴展階段，將在7種不同的轉移性癌癥隊列中（包括去勢抵抗性前列腺癌、卵巢癌、乳腺癌、頭頸癌、非小細胞肺癌、胰腺癌和膀胱癌）驗證1a期研究確定的MTD，并進一步評估藥物的耐受性。

PRT12396：IND申請獲得FDA許可

Prelude Therapeutics公司宣布，其新型JAK2V617F突變選擇性抑制劑PRT12396的IND申請已獲美國FDA批準，將啟動一項1期臨床研究。該研究為開放標簽、多中心試驗，旨在評估PRT12396在高危真紅細胞增多癥以及中高危骨髓纖維化患者中的安全性、初步療效和藥代動力學特征。公司預計將于2026年第二季度完成首例患者給藥。

PRT12396是Prelude公司設計并驗證的新型變構抑制劑，能夠結合到V617F突變所在的JAK2 JH2“深口袋”。該突變是骨髓增殖性腫瘤（MPNs）的主要驅動突變，在約95%的PV、60%的原發性血小板增多癥（ET）和55%的MF患者中存在。Prelude公司開發的這類變構抑制劑在MPN臨床前模型中展現了突變特異性抑制作用，有望降低突變等位基因負荷、延緩甚至逆轉疾病進展，從而改善MPN患者的治療結果。

AMXT 1501：啟動1b/2期聯合治療試驗

Aminex Therapeutics公司宣布啟動一項AMXT 1501聯合二氟甲基鳥氨酸（DFMO）的1b/2期臨床試驗。這項多中心、開放標簽研究將評估口服AMXT 1501與口服DFMO聯合標準治療在兩類人群中的安全性、耐受性和初步療效，包括既往治療失敗、絕經前/后、雌激素受體（ER）陽性、人表皮生長因子受體2（HER2）陰性乳腺癌和轉移性黑色素瘤患者。

AMXT 1501是一種新型多胺轉運抑制劑，旨在阻斷腫瘤細胞攝取外源性多胺——這些分子對腫瘤生長和存活至關重要。DFMO是一種已知的多胺生物合成抑制劑。兩者聯合旨在全面抑制多胺代謝，從而抑制腫瘤生長。

參考資料：

[1] Onchilles Pharma Announces IND Clearance for N17350, Advancing the First Next-Generation Cytotoxic Therapeutic Leveraging the ELANE Pathway into the Clinic. Retrieved February 6, 2026, from https://www.onchillespharma.com/post/onchilles-pharma-announces-ind-clearance-for-n17350

[2] GT Biopharma Announces FDA Clearance of Investigational New Drug (IND) Application for GTB-5550 TriKE?, a B7-H3-Targeted Natural Killer (NK) Cell Engager for Solid Tumors Expressing B7-H3. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231077/0/en/GT-Biopharma-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-GTB-5550-TriKE-a-B7-H3-Targeted-Natural-Killer-NK-Cell-Engager-for-Solid-Tumors-Expressing-B7-H.html

[3] Prelude Therapeutics Receives FDA Clearance of Investigational New Drug Application (IND) for PRT12396, a Mutant-selective JAK2V617F Inhibitor. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231175/0/en/Prelude-Therapeutics-Receives-FDA-Clearance-of-Investigational-New-Drug-Application-IND-for-PRT12396-a-Mutant-selective-JAK2V617F-Inhibitor.html

[4] Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM). Retrieved February 6, 2026, from https://affiniatx.com/affinia-therapeutics-announces-fda-acceptance-of-ind-application-to-advance-aftx-201-to-a-phase-1-2-trial-for-the-treatment-of-bag3-associated-dilated-cardiomyopathy-dcm/

[5] PharmaResearch Receives U.S. FDA Clearance to Initiate Phase 1 Clinical Trial of Nano Anticancer Drug, PRD-101. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/pharmaresearch-receives-us-fda-clearance-to-initiate-phase-1-clinical-trial-of-nano-anticancer-drug-prd-101-302681109.html

[6] Spur Therapeutics Presents New Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at 22nd Annual WORLDSymposium?. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/04/3231939/0/en/Spur-Therapeutics-Presents-New-Phase-1-2-Data-on-Its-Gene-Therapy-Candidate-in-Gaucher-Disease-at-22nd-Annual-WORLDSymposium.html

[7] Aminex Therapeutics Announces Multiple Sites Activated for Phase 1b/2 Clinical Trial of Novel Investigational Cancer Treatment AMXT 1501 and DFMO in Patients with Breast Cancer or Melanoma. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/aminex-therapeutics-announces-multiple-sites-activated-for-phase-1b2-clinical-trial-of-novel-investigational-cancer-treatment-amxt-1501-and-dfmo-in-patients-with-breast-cancer-or-melanoma-302678969.html

[8] Neomorph Announces First Patient Dosed in Phase 1/2 Trial Evaluating NEO-811 For the Treatment of Locally Advanced or Metastatic Non-Resectable Clear Cell Renal Cell Carcinoma. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231475/0/en/Neomorph-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-Evaluating-NEO-811-For-the-Treatment-of-Locally-Advanced-or-Metastatic-Non-Resectable-Clear-Cell-Renal-Cell-Carcinoma.html

[9] Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA). Retrieved February 6, 2026, from https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-longer-term-data-demonstrating

[10] uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/06/3233740/0/en/uniQure-Announces-Updated-Preliminary-AMT-191-Phase-I-IIa-Data-Showing-Sustained-Increases-in-%CE%B1-Gal-A-Enzyme-Activity-in-Patients-with-Fabry-Disease.html

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