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-

Mechanism

Cell replacements

Therapeutic Areas

Digestive System DisordersUrogenital DiseasesNeoplasms+ [2]

Active Indication

Fecal IncontinenceUrinary Incontinence, StressDeglutition Disorders+ [1]

Inactive Indication-

Originator Organization

Cook MyoSite, Inc.

Active Organization

Cook MyoSite, Inc.

Inactive 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.

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.

NCT05421689 / RecruitingPhase 1/2IIT

A Multi-Center, Double-Blind, Randomized Placebo-Controlled Investigation of Autologous Muscle Derived Cells (AMDC) for the Treatment of Tongue Dysphagia Resulting From the Treatment of Head and Neck Cancer

The primary objective of this double-blind, randomized, placebo-controlled, multicenter clinical trial is to evaluate the safety of AMDC-GIR during the 24 months following 2 consecutive treatments of tongue dysphagia in male and female patients who have undergone surgery and/or chemo- and/or radiotherapy for squamous cell cancer of the oropharynx.

Start Date26 May 2022

Sponsor / Collaborator

University of California, Davis

[+2]

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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3

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. ; Snyder, Scott ; Kaufman, Melissa R. ; Heit, Michael H. ; Goldman, Howard B. ; Dmochowski, Roger ; Benson, Kevin D. ; Rehme, Christian ; Sokol, Eric R. ; Kennelly, Michael J. ; Quiroz, Lieschen H. ; Canestrari, Emanuele ; Chancellor, Michael B. ; Wolter, Christopher E. ; Jankowski, Ron J. ; Katz, Daniel M. ; Chermansky, Christopher J. ; Karram, Mickey ; 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 Sep 2020·World Journal of UrologyQ2 · MEDICINE

Contemporary application of autologous muscle-derived cells for urinary sphincter regeneration

Q2 · MEDICINE

Review

Author: Kaufman, Melissa R

OBJECTIVEStress urinary incontinence (SUI) remains a prevalent condition with substantial economic and quality-of-life impact. Treatment for incontinence historically culminates with invasive surgical procedures with recognized complication profiles. Innovative directions for SUI therapeutics are on the horizon, including the utilization of adult autologous muscle-derived cells for urinary sphincter regeneration (AMDC-USR). Herein, we visit fundamental concepts of innovative regenerative medicine technologies for urologic applications.METHODSSynopsis of contemporary literature review regarding adult autologous muscle-derived cells for urinary sphincter regeneration is presented.RESULTSCurrent published literature presents safety and efficacy data regarding AMDC-USR injection in 80 patients at 12-month follow-up. In these early studies, no long-term adverse events were reported and patients undergoing cellular injection at higher doses revealed at least 50% reduction in stress leaks and pad weight at 12-month follow-up. All dose groups demonstrated statistically significant improvement in patient-reported incontinence-specific quality-of-life scores at 12-month follow-up. Conclusions from the pooled analyses indicate that injection of AMDC-USR across a range of dosages appears safe. Efficacy data suggest a dose response with more patients responsive to the higher doses of AMDC-USR.CONCLUSIONApplications for utilization of autologous cellular therapies for treatment of SUI, and conceivably multiple additional indications, are approaching realization. Multiple Phase III randomized, placebo-controlled studies for AMDC-USR are concluding or ongoing to launch this regenerative option for the millions of patients who may ultimately benefit.

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

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 adverse events (SAEs). Secondary outcomes were changes in number of FI episodes, Cleveland Clinic Incontinence Score (CCIS), Fecal Incontinence Quality of Life (FIQL), and anorectal manometry at 3, 6, and 12 months compared to baseline.Results:No SAEs 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% 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 CCIS reduction (-2.9; 95% CI: - 3.7, −2.1), and FIQL 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	US	Cook MyoSite, Inc.	21 Mar 2024
Urinary Incontinence, Stress	Phase 3	GB	Cook MyoSite, Inc.	04 Nov 2011
Deglutition Disorders	Phase 2	US	Cook MyoSite, Inc.	26 May 2022
Head and Neck Neoplasms	Phase 2	US	Cook MyoSite, Inc.	26 May 2022
Urinary Incontinence, Stress	Preclinical	DE	Cook MyoSite, Inc.	04 Nov 2011
Urinary Incontinence, Stress	Preclinical	CA	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	(nrjtpnuutp) = xvacycbhwy atbbpmnrfd (ywohnifott ) Not Met View more	Negative	16 Sep 2024
NCT01893138 (Pubmed \| Neurourol Urodyn) Manual	Phase 3	Urinary Incontinence, Stress	297	Placebo	(nrjtpnuutp) = kehiwcafks atbbpmnrfd (ywohnifott ) Not Met View more	Negative	16 Sep 2024
NCT01600755 (CTgov)	Phase 1/2	Fecal Incontinence	53	Iltamiocel	ujwyuihtqj(igsyptvzba) = vtrdphdbvm pxjsofknjz (kftalhywoc, iirmtvqauc - ttjitjiauu) View more	-	16 Feb 2024
NCT01893138 \| EUCTR2014-002919-41-BE (CTgov)	Phase 3	Urinary Incontinence, Stress	311	Iltamiocel (Iltamiocel)	hhghieksrs(xdmmyacgxu) = yxiahdrvvs ztxkehcsnj (gsfvjcpbmm, ryfjmognhk - rarjbuprof) View more	-	05 Jan 2023
NCT01893138 \| EUCTR2014-002919-41-BE (CTgov)	Phase 3	Urinary Incontinence, Stress	311	Placebo (Placebo)	hhghieksrs(xdmmyacgxu) = zjsmapqwtk ztxkehcsnj (gsfvjcpbmm, tauqjgkzdi - itsegrkvhk) View more	-	05 Jan 2023
NCT02291432 (CTgov)	Phase 1/2	Urinary Incontinence, Stress \| Urological Manifestations \| Lower Urinary Tract Symptoms	25	autologous muscle-derived cells (AMDC)	ukqzctzuuu(vgpazuhpfa) = thfgfmctvi nwdtnyxdce (hejpvdplvv, uwozreecvz - lamwwqhfdi) View more	-	26 Oct 2021
NCT01382602 (CTgov)	Phase 3	Urinary Incontinence, Stress	150	AMDC-USR (AMDC-USR)	pciuglwwvp(efgelmocwn) = kvhouvivvl qpanqpzwap (uswpdzynhn, zfoewinqws - zjfthqipad) View more	-	17 May 2018
NCT01382602 (CTgov)	Phase 3	Urinary Incontinence, Stress	150	Placebo (Placebo)	pciuglwwvp(efgelmocwn) = oddasmspfy qpanqpzwap (uswpdzynhn, rlelbhboxq - pbnigzzgrc) View more	-	17 May 2018
NCT01008943 (CTgov)	Phase 2	Urinary Incontinence, Stress	16	Autologous Muscle-Derived Cells (AMDC)	mqoqarfheg(pnuvznoxbw) = lqsqgspird rfzktvcthl (rvvzjgmctj, yoymszassb - dhgapjvtut) View more	-	03 Nov 2015