ImCheck Reports Durable Responses and Early Overall Survival Signal with ICT01 in First-line AML at ASH 2025
MWN-AI** Summary
ImCheck Therapeutics presented compelling data on ICT01, a pioneering ?9?2 T-cell activator, at the 67th ASH Annual Meeting in Orlando, demonstrating its potential in treating newly diagnosed acute myeloid leukemia (AML) patients unfit for intensive chemotherapy. The updated results from the Phase I/II EVICTION study revealed that ICT01, when combined with azacitidine and venetoclax (Aza-Ven), showcases robust efficacy characterized by rapid onset of remission and a high rate of minimal residual disease (MRD) negativity.
Of the 57 patients enrolled in the study, 90% achieved meaningful clinical responses by the end of the second treatment cycle, underscoring the efficacy of the 10mg ICT01 dosage. This dosage endorsed by both the FDA and EMA for further development shows promise across various AML subtypes, including those with adverse genetic features, such as TP53 mutations. The overall safety profile remained favorable, with a 30-day mortality of just 4%.
Early survival data was notably encouraging; a 12-month overall survival rate of 62% was observed, surpassing recent outcomes reported for the standard Aza-Ven treatment. The findings support ICT01’s potential to provide deep and durable remissions, enhancing the projected overall survival for high-risk AML patients.
The company emphasized the vital role of ICT01 in filling the current treatment void for older or comorbid patients facing limited options. With the acquisition agreement by Ipsen anticipated to finalize soon, ImCheck moves toward late-stage development, having secured Orphan Drug Designations from regulatory authorities in both the U.S. and Europe. This advancement marks a transformative period for ImCheck, as it aims to revolutionize the treatment landscape for AML and potentially other cancers.
MWN-AI** Analysis
**Market Advice on ImCheck Therapeutics Following ASH 2025 Presentation**
The recent presentation at the American Society of Hematology (ASH) by ImCheck Therapeutics regarding ICT01 in the treatment of first-line acute myeloid leukemia (AML) marks a pivotal moment for the company and investors alike. The reported data displayed robust efficacy, including over 90% of patients achieving complete remission after two cycles of treatment, alongside promising early survival signals. Notably, the 10 mg dose of ICT01 showcased superior efficacy, particularly among adverse-risk patients, which significantly broadens its potential market application in a segment traditionally underserved in AML treatment.
With the FDA’s endorsement of the 10 mg dose for further development and the receipt of Orphan Drug Designations from both the FDA and EMA, regulatory momentum is clearly behind ICT01. This positions ImCheck favorably for upcoming late-stage trials, thus enhancing the long-term investment outlook. With strong clinical activity and a manageable safety profile reflected in a low 30-day mortality rate, investors can anticipate increased interest from market players and potential premium valuations leading to the anticipated completion of the acquisition by Ipsen.
Investors should take note of the broader implications of these results. ICT01 not only holds promise for expansion into various cancer indications but also affirms the potency of the ?9?2 T-cell activator approach in oncology. Given the unmet need in AML and high relapse rates associated with existing treatment regimens, ImCheck's innovative strategy could reshape the competitive landscape.
As the agreement with Ipsen progresses, investors should consider staying abreast of subsequent clinical trial updates and market development strategies for ICT01. Given its favorable positioning, this could emerge as a resilient investment opportunity within the biopharmaceutical landscape, reflecting potential significant growth in the coming years.
**MWN-AI Summary and Analysis is based on asking OpenAI to summarize and analyze this news release.
FOR IMMEDIATE RELEASE
-- ImCheck Reports Durable Responses and Early Overall Survival Signal with ICT01 in First-line AML at ASH 2025 --
- Updated clinical data presented at ASH show robust efficacy, rapid onset of remission, high incidence of MRD 1 -negativity, and an encouraging survival signal with ICT01 in combination with azacitidine and venetoclax (Aza-Ven) in newly diagnosed unfit AML patients
- ICT01 has received Orphan Drug Designation from the FDA and EMA; dose selection has been endorsed by FDA as ICT01 advances toward late-stage development
Marseille, France, December 7, 2025, 5:30 pm ET/ 11:30 pm CET – ImCheck Therapeutics today announced updated results from its Phase I/II EVICTION study evaluating ICT01, a first-in-class ?9?2 T-cell activator, in combination with azacitidine and venetoclax in newly diagnosed AML patients ineligible for intensive chemotherapy. The data were presented by Dr. Sylvain Garciaz (Institut Paoli-Calmettes, Marseille, France) in an oral session at the 67th ASH Annual Meeting, taking place December 6-9, in Orlando, Florida. The data presentation follows ImCheck’s recently announced agreement to be acquired by Ipsen, pending transaction close.
The ASH 2025 dataset builds on the promising efficacy signals previously shared at ASCO [2] 2025 , highlighting rapid, deep, and durable responses with a favorable safety profile and encouraging early overall survival. Notably, responses were strongest at the 10mg ICT01 dose, which has now been endorsed by the FDA for further development. In 2025, ICT01 also received Orphan Drug Designation from both the FDA 3 and EMA 4 .
“ICT01 continues to demonstrate rapid and durable responses across AML subtypes. The updated ASH data further strengthens our confidence in ICT01’s ability, when added to Aza-Ven, to deliver deep, lasting remissions that have the potential to translate into meaningful overall survival improvement for patients” said Stephan Braun , MD, PhD, Chief Medical Officer of ImCheck Therapeutics. “We see an early onset of complete remissions, consistently strong efficacy at the selected dose across molecular subtypes, and signs of durable responses even in adverse-risk patients. These findings together with the encouraging early survival signal create a compelling case as we advance toward late-stage development.”
Key Highlights :
- Patient population : At the data cut-off on October 6, 2025, 57 patients aged 51 to 87 had been enrolled. Of these, 41 received 10 mg ICT01 and 16 received 75 mg ICT01, each in combination with Aza-Ven.
- Rapid responses: More than 90% of patients treated with ICT01 (10 mg) achieved CRc 4 as their best response already by end of Cycle 2.
- Broad molecular activity: The 10 mg dose selected for future studies produced higher CR/CRc rates across molecular subtypes, including favorable-, intermediate-, and adverse-risk AML (e.g., TP53-mutated) versus the 75 mg dose.
- Durability emerging: At a median follow-up of 10.8 months median DoR 5 was not yet reached for the 10 mg ICT01 dose.
- Early survival signal: A 12-month OS 6 rate of 62% was observed, which is numerically higher than the ~54% reported for the Aza-Ven regimen in recent Phase 3 trials.
- Favorable benefit–risk profile: Safety of the novel triplet regimen ICT01-Aza-Ven remains well manageable, with a 30-day mortality rate of 4%, and no deaths attributed to ICT01.
- Regulatory momentum: ICT01 received Orphan Drug Designation from both FDA and EMA. The 10 mg ICT01 dose has been endorsed by FDA for further clinical development of the triplet regimen.
“ImCheck has in hand the alignment of strong clinical activity, a favorable safety profile, Orphan Drug Designations on both sides of the Atlantic, and now a clear regulatory-endorsed dose for ICT01 for accelerated late-stage development, ” added Pierre d’Epenoux , Chief Executive Officer of ImCheck Therapeutics. “ These results arrive at a transformative moment for ImCheck, following the announced acquisition agreement with Ipsen. We remain deeply grateful to the patients, investigators, and our team for bringing ICT01 to this important inflection point .”
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About the medical need in AML
Acute myeloid leukemia (AML) remains a significant clinical challenge, particularly for older or unfit patients who cannot tolerate intensive chemotherapy. While the combination of venetoclax and azacitidine has become the standard non-intensive regimen, it is not curative, and relapse rates remain high. Most patients are not eligible for stem cell transplantation, often due to age, comorbidities, or insufficient response, and face limited treatment options and poor overall survival. Despite AML’s known sensitivity to immune-mediated control, current immunotherapies targeting PD-1, TIM-3, or CD47 have not delivered meaningful clinical benefit. This underscores the urgent need for novel immuno-oncology approaches. Recently, ?9?2 T cells, with their cytotoxic activity and unique dual role in both innate and adaptive immunity, have emerged as promising immune modulators. Their association with reduced relapse and prolonged survival, particularly in the post-transplant setting, suggests that enhancing their anti-leukemic potential could offer a meaningful new treatment option for high-risk AML patients.
About the EVICTION Study
EVICTION is a first-in-human, dose-escalation (Part 1) and cohort-expansion (Part 2) clinical study of ICT01 in patients with various advanced relapsed or refractory solid or hematologic cancers that have exhausted standard-of-care treatment options. Part 1 (Phase I) is designed to characterize the preliminary safety, tolerability, and pharmacodynamic activity of increasing doses of ICT01 as monotherapy (Group A: solid tumors; Group B: hematologic tumors) and in combination with pembrolizumab (Group C: solid tumors). Part 2 comprises randomized dose-optimizing and efficacy estimating expansion cohorts of monotherapy (Group D: ovarian cancer; Group E: prostate cancer) and combination treatment of patients with AML (Group F), melanoma (Group G), urothelial cell carcinoma (Group H), or head-and-neck squamous cell carcinoma (Group I). More information on the EVICTION study can be found at clinicaltrials.gov (NCT04243499).
About ICT01
ICT01 is a humanized, anti-BTN3A (also known as CD277) monoclonal antibody that selectively activates ?9?2 T cells, which are responsible for immunosurveillance of malignancy and infections. The three isoforms of BTN3A targeted by ICT01 are overexpressed on many solid tumors (e.g., melanoma, urothelial cell, colorectal, ovarian, pancreatic, and lung cancer) and hematologic malignancies (e.g., leukemia and lymphomas) and also expressed on the surface of innate (e.g., ?? T cells and NK cells) and adaptive immune cells (T cells and B cells). BTN3A is essential for the activation of the anti-tumor immune response of ?9?2 T cells.
As demonstrated by data presented at past AACR, ASCO, ASH, ESMO and SITC conferences, ICT01 selectively activates circulating ?9?2 T cells leading to migration of ?9?2 T cells out of the circulation and into the tumor tissue and triggers a downstream immunological cascade through secretion of pro-inflammatory cytokines, including but not limited to IFN? and TNF?, further augmenting the anti-tumor immune response. Anti-tumor activity and efficacy of ICT01 have been shown in patients across several cancer indications.
About IMCHECK THERAPEUTICS
ImCheck Therapeutics is developing a new generation of immunotherapeutic antibodies targeting butyrophilins, a novel superfamily of immunomodulators. By unlocking the power of ?9?2 T cells, ImCheck’s innovative approach has the potential to transform treatments across oncology, autoimmune, and infectious diseases.
The lead clinical-stage program, ICT01, has been advancing to late-stage trials, demonstrating a unique mechanism of action that modulates both innate and adaptive immunity. These “first-in-class” activating antibodies may deliver superior clinical outcomes compared to first-generation immunotherapy approaches, in particular in rationale combinations with immune checkpoint inhibitors and immunomodulatory anti-cancer drugs. Additionally, ImCheck’s pipeline compounds are progressing toward clinical development for autoimmune and infectious diseases.
The company benefits from the pioneering research of Prof. Daniel Olive (INSERM, CNRS, Institut Paoli-Calmettes, Aix-Marseille University), a global leader in ?9?2 T cells and butyrophilins, as well as the expertise of a seasoned management team and the commitment of leading U.S. and European investors. As announced on October 22, Ipsen (Euronext: IPN; ADR: IPSEY) and ImCheck have entered into a definitive share purchase agreement in which Ipsen will acquire all issued and outstanding shares of ImCheck, with the transaction close anticipated by the end of Q1 2026.
For further information: https://www.imchecktherapeutics.com/
1 MRD: Minimal residual disease. A patient who tests “MRD negative” after treatment for leukemia has less than one leukemia cell per 1 thousand bone marrow cells
2 ASCO: American Society of Clinical Oncology
3 FDA: U.S. Food and Drug Administration
4 EMA: European Medicines Agency
5 DoR: Duration of response
6 OS: Overall survival
Press contacts
US and EU
Trophic Communications
Gretchen Schweitzer
+49 (0) 172 861 8540
schweitzer@trophic.eu
France
ATCG-PARTNERS
Céline Voisin
+33 (0)9 81 87 46 72 / +33 (0)6 62 12 53 39
imcheck@atcg-partners.com
Attachment
FAQ**
How does the recent acquisition agreement with Ipsen, pending transaction close, impact ImCheck's strategic development and investment potential, specifically regarding ICT01 and its clinical outcomes?
With the promising early survival signals seen with ICT01, how could the partnership with Ipsen ADR IPSEY enhance the commercialization prospects for ImCheck's treatment in AML?
Given the robust efficacy data from the ASH 2025 meeting, what are the anticipated next steps for ICT01’s late-stage development under Ipsen ADR IPSEY’s guidance?
How does the Orphan Drug Designation for ICT01 from the FDA and EMA influence ImCheck’s market positioning and competitive advantage in the AML treatment landscape, particularly in light of the acquisition by Ipsen ADR IPSEY?
**MWN-AI FAQ is based on asking OpenAI questions about Ipsen ADR (OTC: IPSEY).
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