TAIPEI, February 22, 2024 – REGiMMUNE Limited, a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies for immune disorders and cancer, today announced the positive results of their phase 2b clinical trial for the prevention of acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic cell transplantation (alloHCT) at the 2024 Tandem Meeting of ASTCT and CIBMTR.

aGVHD, primarily mediated by effector T-lymphocytes, is a major cause of morbidity and mortality after alloHCT. RG1-2001 contains the active moiety α-GalCer, which binds the CD1d receptor of antigen-presenting cells resulting in activation of iNK T cells, resulting in a cytokine-dependent Treg proliferation. This phase 2b trial is an open-label, multi-center, single-arm study. RGI-2001, added to standard immunosuppression, is being evaluated for the potential to reduce the incidence or severity of aGVHD in patients following alloHCT. The results are compared with a contemporaneous CIBMTR cohort (NCT04014790).

Through day 100, the incidence of grades II-IV aGVHD was 22.9% of subjects on RGI-2001 compared to 38.8% of those in the CIBMTR cohort. RGI-2001 also demonstrates higher rates of acute GVHD-free survival (72.9% in grade II-IV and 91.7% in grades III-IV compared with 50.7% and 77.3% in those on CIBMTR cohort, respectively) and overall survival (91.7% compared with 79.2% on CIBMTR cohort). Relapse rates between the 2 cohorts are similar, suggesting no compromise of the graft-versus-leukemia effect.

“We are thrilled to be selected for oral presentation at the Tandem Meeting. The results showed promising safety and efficacy outcomes using RGI-2001 in addition to the standard GVHD prevention medications, providing a strong foundation to advance our plans for phase 3 study.” said Kenzo Kosuda, CEO of REGiMMUNE.

REGiMMUNE concludes that RGI-2001 added to standard-of-care shows positive results for acute GVHD prevention with survival benefit and no increase in relapse compared to CIBMTR control. Based on these results, a phase 3 randomized controlled study is being planned to confirm the efficacy and safety of RGI-2001 in alloHCT.

Details of presentation, please see RGI-2001 Tandem 2024 Oral Presentation



About RGI-2001

RGI-2001 is a liposomal injection formulation of an alpha-galactosylceramide (alpha-GalCer) analog. Alpha-GalCer binds to the CD1d receptor of antigen-presenting cells resulting in activation of invariant natural killer (iNKT) cells, resulting in the activation and expansion of regulatory T cells (Tregs), thus establishing immune tolerance. RGI-2001 is REGiMMUNE’s lead drug candidate, currently in Phase 3 planning for the prophylaxis of acute graft-versus-host disease (aGVHD).

About REGiMMUNE Limited

REGiMMUNE is a clinical-stage biopharmaceutical company focused on creating innovative immunotherapies by harnessing the power of regulatory T cells (Tregs). REGiMMUNE is creating a pipeline of novel product candidates that either enhance Treg activities for immune diseases or suppress Treg activities for cancer.

Corporate Contact

Miranda Yu
Investor Relations Manager
REGiMMUNE Corporation Ltd.
+886 2 2555 3377 #511




2024222日,台北 瑞格國際生技,一家致力於開發免疫性疾病和癌症新型療法的臨床階段生技公司,今天在美國移植與細胞療法學會 (ASTCT)和國際血液與骨髓移植研究中心 (CIBMTR)聯合舉辦的Tandem學會上宣布RGI-2001臨床2b期試驗結果,該試驗旨在預防異體造血幹細胞移植 (alloHCT)後急性移植物抗宿主病 (aGVHD)。

aGVHD主要由作用型T淋巴細胞引發,是alloHCT後造成手術病患併發症和死亡的主要原因,RG1-2001含有活性成分a-GalCer,該成分結合抗原呈現細胞的CD1d受體,進而活化iNKT細胞,促使Treg細胞增殖。本次發表的臨床2b期試驗是一項開放、多中心、單臂研究,RGI-2001與標準療法的免疫抑制劑合併使用,評估其在alloHCT術後患者中降低aGVHD發病率或嚴重程度的潛力 (NCT04014790),試驗結果將與使用標準療法的病患進行比較,該數據由CIBMTR提供。

接受RGI-2001治療後的第100天,受試者II-IV級aGVHD的發生率為22.9%,而在CIBMTR群體中為38.8%。試驗結果顯示RGI-2001有較高的無aGVHD存活率 (II-IV級為72.9%,III-IV級為91.7%,而在CIBMTR群體中分別為50.7%和77.3%),以及整體存活率 (為91.7%,而在CIBMTR群體中為79.2%)。兩個群體之間的原疾病復發率相似,表明使用RGI-2001沒有影響移植物對抗血癌 (graft versus leukemia, GVL)作用。



更多相關資訊,請參考簡報檔 RGI-2001 Tandem 2024 Oral Presentation




RGI-2001係為α-半乳糖神經醯胺(α-Galactosylceramide, α-GalCer)之微脂體注射製劑,α-GalCer與抗原呈遞細胞(Antigen Presenting Cells)上之CD1d配體結合,透過活化恆定自然殺手T細胞(iNKT),近一步活化並擴增調節性T細胞,建立免疫耐受性。RGI-2001為瑞格國際生技主要之候選藥物,聚焦急性移植物抗宿主疾病(aGvHD)之預防,將於2024年開展美國之臨床3期試驗。




余明穎 (Miranda Yu)
+886 2 2555 3377 #511