Delving into the Latest Updates on Iltamiocel (Cook MyoSite) with Synapse

Overview

Basic Info

Drug Type

Stem cell therapy

Synonyms

AMDC - Cook MyoSite, AMDC-USR, Autologous muscle derived cells - Cook MyoSite

+ [1]

Target-

Action-

Mechanism

Cell replacements

Therapeutic Areas

Digestive System DisordersUrogenital DiseasesNeoplasms+ [3]

Active Indication

Fecal IncontinenceUrinary Incontinence, StressDeglutition Disorders+ [1]

Inactive Indication-

Originator Organization

Cook MyoSite, Inc.

Active Organization

Cook MyoSite, Inc.

Inactive Organization-

License Organization-

Drug Highest PhasePhase 3

First Approval Date-

Regulation-

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This is a confirmatory/bridging study to evaluate the efficacy and safety of Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR) compared with placebo in the treatment of stress urinary incontinence (SUI) in adult Japanese female subjects. Half of the participants will receive AMDC-USR (injections with cells) and the other half will receive placebo.

Start Date01 Jun 2025

Sponsor / Collaborator

Cook MyoSite, Inc.

NCT06743620

/ Enrolling by invitationNot Applicable

Prospective Observational Long-Term Safety and Effectiveness Registry of Patients Who Have Received Iltamiocel as Part of Cook Myosite, Inc. Sponsored CELLEBRATE Clinical Study in Patients With Persistent or Recurrent Stress Urinary Incontinence (SUI) Following Surgical Treatment

The main purpose of this registry is to collect observational, long-term safety and effectiveness data in subjects who have received at least one dose of iltamiocel as part of the CLBT clinical study.

Start Date11 Apr 2025

Sponsor / Collaborator

Cook MyoSite, Inc.

NCT05776277

/ RecruitingPhase 3

A Two-stage, Randomized, Controlled Trial Comparing the Safety and Efficacy of Iltamiocel With Placebo in the Treatment of Female Participants With Chronic Fecal Incontinence and a History of Obstetric Anal Sphincter Injury (DigniFI)

This study evaluates the efficacy and safety of a single injection of iltamiocel (300 x 10^6 cells) compared to a placebo in the reduction of fecal incontinence episode frequency in adult female participants with chronic fecal incontinence and a history of obstetric anal sphincter injury. Half of the participants will receive iltamiocel (injections with cells) and the other half will receive placebo.

Start Date21 Mar 2024

Sponsor / Collaborator

Cook MyoSite, Inc.

100 Clinical Results associated with Iltamiocel (Cook MyoSite)

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100 Translational Medicine associated with Iltamiocel (Cook MyoSite)

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100 Patents (Medical) associated with Iltamiocel (Cook MyoSite)

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15

Literatures (Medical) associated with Iltamiocel (Cook MyoSite)

01 Nov 2024·Neurourology and Urodynamics

Iltamiocel Autologous Cell Therapy for the Treatment of Female Stress Urinary Incontinence: A Double‐Blind, Randomized, Stratified, Placebo‐Controlled Trial

Article

Author: Antosh, Danielle ; Peters, Kenneth M. ; Marguet, Charles G. ; Lee, Una J. ; Tarnay, Christopher M. ; Kaufman, Melissa R. ; Snyder, Scott ; Heit, Michael H. ; Goldman, Howard B. ; Dmochowski, Roger ; Benson, Kevin D. ; Rehme, Christian ; Sokol, Eric R. ; Kennelly, Michael J. ; Canestrari, Emanuele ; Quiroz, Lieschen H. ; Chancellor, Michael B. ; Wolter, Christopher E. ; Jankowski, Ron J. ; Karram, Mickey ; Chermansky, Christopher J. ; Katz, Daniel M. ; Bennett, Jason B. ; Thomas, Sherry

ABSTRACTAimsThis study aimed to determine the efficacy and safety of iltamiocel investigational autologous muscle cell therapy in females with stress urinary incontinence (SUI).MethodsAdult females were randomized 2:1 to iltamiocel (150 × 106 cells) or placebo and stratified by severity and prior SUI surgery. The primary objective was efficacy based on the frequency of stress incontinence episodes (SIE) recorded in a 3‐day diary at 12 months posttreatment. After 12 months, placebo participants could elect to receive open‐label iltamiocel. Efficacy and safety analyses were performed using all patients as treated populations.ResultsThe study enrolled 311 patients, 297 were randomized to either iltamiocel (n = 199) or placebo (n = 98). Of the 295 participants that completed 12 months blinded follow‐up, the proportion achieving the primary endpoint of ≥ 50% SIE reduction was not statistically different between treatment groups (52% vs. 53.6%; p = 0.798). A significantly greater proportion of iltamiocel participants in the prior SUI surgery stratum group achieved ≥ 75% SIE reduction compared with placebo, (40% vs. 16%; p = 0.037). Treatment response was maintained at 24 months in 78.4% and 64.9% of iltamiocel participants who achieved ≥ 50% and ≥ 75% SIE reduction, respectively, at Month 12. Adverse events related to the treatment were reported in 19 (9.5%) iltamiocel participants and 6 (6.1%) placebo participants.ConclusionThe study did not meet its primary endpoint however, iltamiocel cell therapy is safe and may be ideally suited to female patients who have undergone prior surgery for SUI. Additional study in this group of patients with high unmet medical needs is warranted.Trial RegistrationClinicalTrials.gov identifier: NCT01893138; EudraCT number: 2014‐002919‐41.

01 Feb 2024·Protein Expression and Purification

Antibody-mimetic drug conjugate with efficient internalization activity using anti-HER2 VHH and duocarmycin

Article

Author: Kawamura, Takeshi ; Chansler, Michael ; Sakata, Juri ; Tanaka, Toshiya ; Ishii, Masazumi ; Tatsumi, Toshifumi ; Takahashi, Kazuki ; Inagaki, Yuya ; Miura, Mai ; Kanai, Motomu ; Yamashita, Takefumi ; Osawa, Tsuyoshi ; Katoh, Hiroto ; Aki, Sho ; Yamatsugu, Kenzo ; Sugiyama, Akira ; Ishikawa, Shumpei ; Tsukagoshi, Masanobu ; Kaneko, Yudai ; Tokuyama, Hidetoshi ; Kodama, Tatsuhiko ; Shimizu, Akihiro

Antibody-mimetic drug conjugate (AMDC) is a cancer cell-targeted drug delivery system based on the non-covalent binding of mutated streptavidin and modified biotin, namely Cupid and Psyche. However, the development of AMDCs is hampered by difficulties in post-translational modification or poor internalization activity. Here, we report an expression, refolding, and purification method for AMDC using a variable heavy chain of heavy chain-only antibodies (VHHs). Monomeric anti-HER2 VHH fused to Cupid was expressed in Escherichia coli inclusion bodies. Solubilization and refolding at optimized reducing conditions and pH levels were selected to form a functional, tetrameric protein (anti-HER2 VHH-Cupid) that can be easily purified based on molecular weight. Anti-HER2 VHH-Cupid non-covalently creates a tight complex with Psyche linked to a potent DNA-alkylating agent, duocarmycin. This complex can be absorbed by the HER2-expressing human breast cancer cell line, KPL-4, and kills KPL-4 cells in vitro and in vivo. The production of a targeting protein with internalizing activity, combined with the non-covalent conjugation of a highly potent payload, renders AMDC a promising platform for developing cancer-targeted therapy.

01 Dec 2023·Annals of Surgery

Safety and Efficacy of Iltamiocel Cellular Therapy for the Treatment of Fecal Incontinence. Results of a Phase 1/2 Study

Article

Author: Knowles, Charles H. ; Canestrari, Emanuele ; Cardello, Kelly ; Raval, Manoj J. ; Jankowski, Ron J.

Objective:To examine the safety and efficacy of iltamiocel, an investigational cellular therapy of autologous muscle-derived cells, as a treatment for fecal incontinence (FI) in adults.Background:Limited therapeutic options are available for patients with FI refractory to conservative treatments. Cell therapy using autologous muscle-derived cells represents a promising, minimally invasive approach for restoring anal sphincter function.Methods:In this multicenter, prospective, non-randomized study, 48 participants were treated with a single iltamiocel dose of 250×106 cells. The primary outcome was the incidence of product or procedure-related adverse events (AEs) and serious AEs. Secondary outcomes were changes in the number of FI episodes, Cleveland Clinic Incontinence Score, Fecal Incontinence Quality of Life, and anorectal manometry at 3, 6, and 12 months compared to baseline.Results:No serious AEs and only one product-related AE of inflammation at the injection site were reported. At 12 months, there was a reduction in median FI episodes (−6.0; 95% confidence interval (CI): −10.0, −1.0) and days with episodes (−4.0; 95% CI: −8.0, −1.0). A ≥50% reduction in FI episodes was observed in 53.7% of participants, and 24.4% had complete restoration of continence. Symptom severity and quality of life improved with mean Cleveland Clinic Incontinence Score reduction (-2.9; 95% CI: −3.7, −2.1), and Fecal Incontinence Quality of Life increased (2.2; 95% CI:1.4, 2.9). No significant changes were detected in anorectal manometry measurements. A history of episiotomy was significantly associated with treatment response in multivariate analysis.Conclusion:The administration of iltamiocel cellular therapy is safe. Iltamiocel shows promise for significantly improving fecal incontinence symptoms and quality of life.

100 Deals associated with Iltamiocel (Cook MyoSite)

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Fecal Incontinence	Phase 3	United States	Cook MyoSite, Inc.	21 Mar 2024
Urinary Incontinence, Stress	Phase 3	United Kingdom	Cook MyoSite, Inc.	04 Nov 2011
Deglutition Disorders	Phase 2	United States	Cook MyoSite, Inc.	26 May 2022
Squamous Cell Carcinoma of the Oropharynx	Phase 2	United States	Cook MyoSite, Inc.	26 May 2022
Urinary Incontinence, Stress	Discovery	Germany	Cook MyoSite, Inc.	04 Nov 2011
Urinary Incontinence, Stress	Discovery	Canada	Cook MyoSite, Inc.	04 Nov 2011

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01893138 (Pubmed \| Neurourol Urodyn) Manual	Phase 3	Urinary Incontinence, Stress	297	Iltamiocel (150 × 10^6 cells)	(jfqbxtogzp) = lmhwfsanjm ypjinhxiie (adnospeeyu ) Not Met View more	Negative	16 Sep 2024
NCT01893138 (Pubmed \| Neurourol Urodyn) Manual	Phase 3	Urinary Incontinence, Stress	297	Placebo	(jfqbxtogzp) = uixubbavoa ypjinhxiie (adnospeeyu ) Not Met View more	Negative	16 Sep 2024
NCT01600755 (CTgov)	Phase 1/2	Fecal Incontinence	53	Iltamiocel	zenrjnnkyj(vupurspgrl) = yuzxgjbogt vageuwuezq (ncjlddvnrh, mbpcbfutsm - lhkccltcqi) View more	-	16 Feb 2024
NCT01893138 (2014-002919-41 \| ###DELETE, CTgov)	Phase 3	Urinary Incontinence, Stress	311	Iltamiocel (Iltamiocel)	zgukejmtqw(tffnaaxrsb) = mdqpqnhztn vgzxlfrbgo (ltwypsaldl, vzbdobynis - zqrxgaqnyp) View more	-	05 Jan 2023
NCT01893138 (2014-002919-41 \| ###DELETE, CTgov)	Phase 3	Urinary Incontinence, Stress	311	Placebo (Placebo)	zgukejmtqw(tffnaaxrsb) = bgvcnwxkmt vgzxlfrbgo (ltwypsaldl, mvygwgwlmk - alnkisjmqi) View more	-	05 Jan 2023
NCT02291432 (CTgov)	Phase 1/2	Urinary Incontinence, Stress	25	autologous muscle-derived cells (AMDC)	egecurwqps(lalbygijsm) = khvspgypzh jbtgixxhnu (hcbdvusysh, logkznvdvc - fgwrutlaex) View more	-	26 Oct 2021
NCT01382602 (CTgov)	Phase 3	Urinary Incontinence, Stress	150	AMDC-USR (AMDC-USR)	ixannhttgs(gxkfehbzbh) = kwbuxlgywb plsjlchgjg (hntjzguqge, onkitjpgyf - usdzapthwf) View more	-	17 May 2018
NCT01382602 (CTgov)	Phase 3	Urinary Incontinence, Stress	150	Placebo (Placebo)	ixannhttgs(gxkfehbzbh) = ktbmyxszio plsjlchgjg (hntjzguqge, znflwogpym - fvlbdqwsag) View more	-	17 May 2018
NCT01008943 (CTgov)	Phase 2	Urinary Incontinence, Stress	16	Autologous Muscle-Derived Cells (AMDC)	iqugurvjlz(zniyzfnoex) = myadkhpwyu dnbewkxebi (jrgugqhbdn, tilkqpffbt - yemucibzoy) View more	-	03 Nov 2015