Single center, prospective, randomized, double-blind, pragmatic Phase 2 clinical study in severely injured trauma patients within hours of hospitalization who have survived initial resuscitation.

Start Date09 Nov 2020

Sponsor / Collaborator

Healios KK

[+1]

NCT04367077

/ RecruitingPhase 2/3

A Phase 2/3 Study to Assess the Safety and Efficacy of MultiStem® Therapy in Subjects With Acute Respiratory Distress Syndrome (ARDS) Due to Coronavirus Disease (COVID-19)

Multicenter investigation featuring an open-label lead-in followed by a double blinded, randomized, placebo-controlled Phase 2/3 part to evaluate the safety and efficacy of MultiStem therapy in subjects with moderate to severe Acute Respiratory Distress Syndrome (ARDS) due to pathogens including COVID-19.

Start Date28 Apr 2020

Sponsor / Collaborator

Healios KK

NCT03807804

/ CompletedPhase 2

An Open-label, Standard Therapy as a Controlled, Multicenter Phase 2 Study to Evaluate the Efficacy and Safety of HLCM051(MultiStem) in Patients With Acute Respiratory Distress Syndrome (ARDS) Caused by Pneumonitis

The primary object of this clinical study is to investigate the efficacy of HLCM051 in patients with ARDS caused by pneumonitis.

Start Date01 Jan 2019

Sponsor / Collaborator

Healios KK

100 Clinical Results associated with Invimestrocel (Athersys)

100 Translational Medicine associated with Invimestrocel (Athersys)

100 Patents (Medical) associated with Invimestrocel (Athersys)

Literatures (Medical) associated with Invimestrocel (Athersys)

01 Jun 2025·Regenerative Therapy

Clinical efficacy of invimestrocel for acute respiratory distress syndrome caused by pneumonia: Comparison with historical data using propensity score analysis

Article

Author: Fujimoto, Kiminori ; Tasaka, Sadatomo ; Kondoh, Yasuhiro ; Imanaka, Hideaki ; Ichiyasu, Hidenori ; Hashimoto, Satoru ; Ichikado, Kazuya ; Kawayama, Tomotaka ; Sawada, Masanori ; Kotani, Toru ; Nunomiya, Shin ; Sakagami, Takuro ; Johkoh, Takeshi ; Kawamura, Kodai

Introduction:

Acute respiratory distress syndrome (ARDS) is a life-threatening inflammatory lung injury often resulting from pneumonia. The efficacy and safety of invimestrocel in patients with pneumonia-induced ARDS have been demonstrated previously in a phase II randomized, open-label trial (the ONE-BRIDGE study). In this study, we aimed to compare data from the intervention (invimestrocel) arm of the ONE-BRIDGE study with matched historical data from a previously established cohort to provide further support for the beneficial effects of invimestrocel in patients with pneumonia-induced ARDS.

Methods:

Twenty patients from the invimestrocel arm of the ONE-BRIDGE study (Invimestrocel group) and 104 from the historical cohort were included in this study. A matched historical data group (n = 20) was extracted from the historical cohort based on the propensity score analysis using age, sex, PaO₂/F_IO₂ ratio, and high-resolution computed tomography scores. The primary outcomes measured were ventilator-free days (VFDs) during the first 28 days following treatment and mortality on days 28, 60, 90, and 180.

Results:

Patients in the Invimestrocel group showed higher VFDs (14.8 ± 11.0 vs. 6.7 ± 9.4 days; 95 % confidence interval [CI], 1.4-14.7; p = 0.0110) and survival rates (log-rank testing; hazard ratio, 0.330; 95 % CI, 0.116-0.938) than those in the matched historical data group.

Conclusions:

The addition of invimestrocel to the standard treatment for pneumonia-induced ARDS may result in early withdrawal from the ventilator and lower mortality. However, further randomized, blinded, and placebo-controlled studies without or addressing multiplicity are required to confirm these findings.

01 Feb 2024·JAMA neurology

Allogeneic Stem Cell Therapy for Acute Ischemic Stroke

Article

Author: Minematsu, Kazuo ; Ikawa, Fusao ; Yamano, Yoshihisa ; Osanai, Toshiya ; Aoyama, Atsuo ; Inenaga, Chikanori ; Asaoka, Katsuyuki ; Shimoda, Yusuke ; Kato, Koichi ; Sugiyama, Taku ; Tempaku, Akira ; Taguchi, Akihiko ; Yasui, Keizo ; Iguchi, Yasuyuki ; Hirano, Teruyuki ; Takagi, Yasushi ; Urabe, Takao ; Ohtaki, Masafumi ; Kuga, Yoshihiro ; Sakai, Nobuyuki ; Yoshimura, Shinichi ; Abe, Tatsuya ; Hess, David C. ; Kitagawa, Kazuo ; Isobe, Masanori ; Takahashi, Shinichi ; Yonehara, Toshiro ; Moroi, Junta ; Shimamura, Norihito ; Haraguchi, Kouichi ; Oki, Koichi ; Kasuya, Hidetoshi ; Matsuo, Takayuki ; Yasaka, Masahiro ; Haraguchi, Koichi ; Tanikawa, Rokuya ; Houkin, Kiyohiro ; Kudo, Kohsuke ; Uchiyama, Shinichiro ; Savitz, Sean I. ; Maruichi, Katsuhiko ; Toyoda, Kazunori ; Kawabori, Masahito ; Kamiyama, Kenji ; Marushima, Aiki ; Takizawa, Katsumi ; Tominaga, Atsushi ; Niiya, Yoshimasa ; Matsuoka, Hideki ; Izawa, Yoshikane ; Fujimura, Miki ; Terasaki, Tadashi ; Ito, Masaki ; Sato, Atsushi ; Kuroda, Satoshi ; Kimura, Kazumi ; Tokunaga, Koji

Importance:

Cell therapy is a promising treatment approach for stroke and other diseases. However, it is unknown whether MultiStem (HLCM051), a bone marrow–derived, allogeneic, multipotent adult progenitor cell product, has the potential to treat ischemic stroke.

Objective:

To assess the efficacy and safety of MultiStem when administered within 18 to 36 hours of ischemic stroke onset.

Design, Setting, and Participants:

The Treatment Evaluation of Acute Stroke Using Regenerative Cells (TREASURE) multicenter, double-blind, parallel-group, placebo-controlled phase 2/3 randomized clinical trial was conducted at 44 academic and clinical centers in Japan between November 15, 2017, and March 29, 2022. Inclusion criteria were age 20 years or older, presence of acute ischemic stroke (National Institutes of Health Stroke Scale [NIHSS] score of 8-20 at baseline), confirmed acute infarction involving the cerebral cortex and measuring more than 2 cm on the major axis (determined with diffusion-weighted magnetic resonance imaging), and a modified Rankin Scale (mRS) score of 0 or 1 before stroke onset. Data analysis was performed between May 9 and August 15, 2022.

Exposure:

Patients were randomly assigned to either intravenous MultiStem in 1 single unit of 1.2 billion cells or intravenous placebo within 18 to 36 hours of ischemic stroke onset.

Main Outcomes and Measures:

The primary end points were safety and excellent outcome at day 90, measured as a composite of a modified Rankin Scale (mRS) score of 1 or less, a NIHSS score of 1 or less, and a Barthel index score of 95 or greater. The secondary end points were excellent outcome at day 365, mRS score distribution at days 90 and 365, and mRS score of 0 to 1 and 0 to 2 at day 90. Statistical analysis of efficacy was performed using the Cochran-Mantel-Haenszel test.

Results:

This study included 206 patients (104 received MultiStem and 102 received placebo). Their mean age was 76.5 (range, 35-95) years, and more than half of patients were men (112 [54.4%]). There were no between-group differences in primary and secondary end points. The proportion of excellent outcomes at day 90 did not differ significantly between the MultiStem and placebo groups (12 [11.5%] vs 10 [9.8%], P = .90; adjusted risk difference, 0.5% [95% CI, −7.3% to 8.3%]). The frequency of adverse events was similar between treatment groups.

Conclusions and Relevance:

In this randomized clinical trial, intravenous administration of allogeneic cell therapy within 18 to 36 hours of ischemic stroke onset was safe but did not improve short-term outcomes. Further research is needed to determine whether MultiStem therapy for ischemic stroke has a beneficial effect in patients who meet specific criteria, as indicated by the exploratory analyses in this study.

Trial Registration:

ClinicalTrials.gov Identifier: NCT02961504

01 Jun 2018·International journal of stroke : official journal of the International Stroke SocietyQ2 · MEDICINE

Treatment evaluation of acute stroke for using in regenerative cell elements (TREASURE) trial: Rationale and design

Q2 · MEDICINE

Article

Author: Minematsu, Kazuo ; Uchiyama, Shinichiro ; Houkin, Kiyohiro ; Taguchi, Akihiko ; Terasaka, Shunsuke ; Osanai, Toshiya

Rationale:

MultiStem® (HLM051) is one of the promising allogenic cell products for acute ischemic stroke with strong evidence. A previous phase 2 randomized, double-blind, placebo-controlled, multicenter dose-escalation trial showed the safety of MultiStem® for acute ischemic stroke, with a time window beyond that of rt-PA and endovascular thrombectomy. We aim to obtain stronger evidence and to show the efficacy of the MultiStem® for treatment of ischemic stroke.

Sample size:

Estimated sample size is 220 (110 patients per group), which has 90% power at 5% significance level.

Methods and design:

TREASURE is a randomized, double-blind, placebo-controlled, multicenter phase 2/3 trial. The trial will be done at 31 medical centers in Japan. Patients with acute ischemic stroke including motor or speech deficit defined by a National Institution of Health Stroke Scale (NIHSS) score of 8–20 at baseline will be randomized 1:1 to receive a single intravenous infusion of MultiStem® or placebo within 18–36 h of stroke onset.

Study outcomes:

Primary outcome in this study is the proportion of patients with an excellent outcome at day 90 defined by the functional assessment.

Trial registration:

ClinicalTrials.gov (NCT02961504).

Conclusion:

The TREASURE trial will provide a novel treatment option and expand the therapeutic window for patients with stroke if the results are positive.

News (Medical) associated with Invimestrocel (Athersys)

14 May 2026

·www.biospace.com

HEALIOS K.K. to Present at Jefferies Global Healthcare Conference 2026

NEW YORK, May 14, 2026 (GLOBE NEWSWIRE) -- HEALIOS K.K. (“Healios”) is pleased to announce that Richard Kincaid, Chief Financial Officer, will present at the Jefferies Global Healthcare Conference in New York City as follows: Date: Thursday, June 4, 2026 Time: 7:35AM EDT Webcast: Click Here To schedule a 1x1 meeting with Healios, please contact your Jefferies representative at healthcareconference@jefferies.com . The live and archived webcast will be accessible from the Healios website. The replay of the webcast will be accessible for 60 days. About Healios: HEALIOS K.K. is Japan’s leading clinical stage biotechnology company harnessing the potential of stem cells for regenerative medicine. It aims to offer new therapies for patients suffering from diseases without effective treatment options. Healios is a pioneer in the development of regenerative medicines in Japan and owns proprietary, global platforms utilizing both somatic stem cells and iPS cells. In the somatic stem cell field, Healios is developing HLCM051 (invimestrocel), a proprietary cell product comprised of multipotent adult progenitor cells (“MAPCs”) derived from the bone marrow of healthy adult donors. HLCM051 has been shown to exhibit powerful anti-inflammatory and immunomodulatory properties with applicability in a range of disease states, is manufactured consistently at scale in 3D bioreactors, and has demonstrated both safety and suggested efficacy in hundreds of patients across multiple indications. Healios is seeking to advance HLCM051 on a global basis for ARDS, trauma, and ischemic stroke. In the iPSC regenerative medicine field, Healios has developed HLCN061, a next generation NK cell treatment for solid tumors that has been functionally enhanced through gene-modification. These cells have demonstrated robust anti-tumor efficacy in animal models, benefit from a scalable 3D bioreactor manufacturing process, and are currently being prepared for initial human testing in collaboration with Akatsuki Therapeutics, Inc. The company has also established a proprietary, gene-edited “universal donor” induced pluripotent stem cell line to develop next generation regenerative treatments in immuno-oncology, ophthalmology, liver diseases, and other areas of severe unmet medical need. Healios was established in 2011 and has been listed on the Tokyo Stock Exchange since 2015 (TSE Growth: 4593). Contact: U.S. Investor Relations: Lisa M. Wilson T: 212-452-2793 E: lwilson@insitecony.com Richard Kincaid Executive Officer and Chief Financial Officer E-mail: ir@healios.jp Company Name: HEALIOS K.K. Representative: Hardy TS Kagimoto, Chairman & CEO (TSE Growth Code: 4593)

Cell Therapy

04 Apr 2024

·insights.citeline.com

Healios Acquires Bankrupt Athersys, Expands Cell Therapy R&D Programs

Tokyo-based Healios will lead the global clinical studies for Athersys’ cell therapy MultiStem for ARDS and start US Phase II trials for trauma following their long-term collaboration.

Cell TherapyPhase 2AcquisitionImmunotherapy

09 Jan 2024

·www.biospace.com

Athersys Adds to Surge of Biotechs Filing for Bankruptcy, Sells to Healios

Pictured: Hand holding Chapter 11 Bankruptcy Docs/iStock, designer491 Continuing the surge of biotech bankruptcies, Athersys filed for Chapter 11 on January 5, according to an SEC filing. All assets of the regenerative medicine and cell therapy company are being divested to its research partner, Healio, to the tune of $2 million in the form of a credit bid. The bankruptcy filing was not a surprise. After reporting disappointing results from its MultiStem pivotal trial in October 2023, the company said it was exploring options but, if unable to obtain adequate financing, would have to protection under bankruptcy laws to “conduct an orderly wind down of operations.” Athersys ended the third quarter of 2023 with only $1 million in cash, despite cost reduction efforts which included layoffs earlier in the year. Even a $10.4 million raise from investors and licensing partners in November was not enough to stave off Chapter 11. Healios will now take the reins on Athersys’ MultiStem program, which has been in development since 1994. The off-the-shelf therapy developed from adult stem cells was being studied in ischemic stroke—a program which was already partnered with Healios—traumatic injury and acute respiratory distress syndrome. The treatment was attractive as a stem cell option because it could be given to patients without prior immune suppression or tissue matching. Last year was a particularly tough one for biotech, presenting a record high number of bankruptcies, BioSpace found, with 41 biotech and pharma companies filing for bankruptcy. By comparison, 20 companies filed in 2022 and only nine in 2021. Experts identified the primary drivers for the surge as the post-COVID-19 economy, a shift toward data-driven financing activity, rising inflation rates and the rapid rate of innovation leading to increased competition in the space. “It’s a terrible market to get financing,” Ira Leiderman, managing director of the healthcare practice at Cassel Salpeter & Co., told BioSpace previously. “Companies are not getting financed, and they have no choice but to break the glass and push the bankruptcy button.” Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.

Cell Therapy

100 Deals associated with Invimestrocel (Athersys)

R&D Status

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
COVID-19	Phase 3	United States	Healios KK	28 Apr 2020
Respiratory Distress Syndrome, Acute	Phase 3	United States	Healios KK	28 Apr 2020
Ischemic stroke	Phase 3	Japan	Healios KK	15 Nov 2017
Multiple Organ Failure	Phase 2	United States	Healios KK	09 Nov 2020
Systemic Inflammatory Response Syndrome	Phase 2	United States	Healios KK	09 Nov 2020
Wounds and Injuries	Phase 2	United States	Healios KK	09 Nov 2020
Pneumonia	Phase 2	Japan	Healios KK	01 Jan 2019
Respiratory Distress Syndrome, Adult	Phase 2	Japan	Healios KK	01 Jan 2019
Non-St Elevated Myocardial Infarction	Phase 2	United States	Healios KK	01 Dec 2015
Colitis, Ulcerative	Phase 2	United States	Pfizer Inc.	01 Feb 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03807804 (ONE-BRIDGE, Pubmed)	Phase 2	Respiratory Distress Syndrome, Acute	30	invimestrocel plus standard treatment	minmwyourx(sfrokolrbq) = kxtbpysisv xuajexkqrq (vfhmhupnem ) View more	Positive	22 Aug 2023
NCT03545607 (MASTERS-2, PipelineReview) Manual	Phase 3	Ischemic stroke	206	invimestrocel	bjtelqmrjt(echdwfjqxc) = The primary endpoint, Excellent Outcome at 90 days, did not reach statistical significance in this population. bvtetkxjtq (hqgpxbphzx ) View more	Positive	20 May 2022
				Placebo
NCT01436487 (MASTERS, Pubmed \| Lancet Neurol) Manual	Phase 2	Acute Ischemic Stroke	129	multipotent adult progenitor cells	islrjqzzqh(koykdgismm) = There were no dose-limiting toxicity events in either group. qnulfbnufr (xmhskahgre ) View more	Positive	01 May 2017
				Placebo

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Hiro LS Researcher

The AI Search Engine Built to Accelerate Biopharma Decisions

Search across billion-scale life sciences data to uncover signals, validate evidence, and act with confidence.

Ask any biopharma research question→

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, Patsnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Discover Synapse Data Servers

Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis