rebonuputemcel (IDCT) / DiscGenics - LARVOL DELTA

DiscGenics Announces US FDA Approval to Proceed with Phase III Clinical Evaluation of Allogeneic, Injectable Disc Progenitor Cell Therapy for Symptomatic Lumbar Degenerative Disc Disease (DiscGenics Press Release) - "DiscGenics, Inc...today announced it has gained acceptance from the U.S. Food and Drug Administration (FDA) for the clinical protocols and Chemistry, Manufacturing, and Controls (CMC) clinical development plan for Phase III clinical program of its allogeneic, injectable disc progenitor cell therapy (IDCT or rebonuputemcel) for painful lumbar degenerative disc disease (DDD), allowing the study to proceed....The Phase III clinical program agreed upon with the FDA will consist of two identical studies running in parallel: the pivotal study (DGX-A02 or PIVOT) and the confirmatory study (DGX-A03 or CONFIRM)....Each study will have a one-year primary endpoint with another one-year follow-up period (two-year total duration). The first subject in the pivotal study is estimated to be enrolled during Q4 2024."

IND • New P3 trial • Back Pain • CNS Disorders • Lumbar Back Pain • Pain

DiscGenics Announces the International Journal of Spine Surgery Has Published Results from an FDA-Approved Study of an Allogeneic Disc Progenitor Cell Therapy for the Treatment of Adults with Lumbar Disc Degeneration (PRNewswire) - P1/2 | N=60 | "DiscGenics, Inc...today announced publication of results in the International Journal of Spine Surgery from the combined Phase I/Phase II, first-in-human clinical study of an allogeneic disc progenitor cell therapy (IDCT or rebonuputemcel) for painful lumbar degenerative disc disease (DDD). The study met the primary safety and efficacy endpoints, showing that a single intradiscal injection of high-dose IDCT (9,000,000 cells/mL) safely increases disc volume and produces statistically significant, clinically meaningful improvements in back pain, disability, and quality of life out to 2 years post-injection in patients with lumbar disc degeneration....At Week 52, the primary study period, the high-dose group had a mean VAS percentage decrease from baseline (−62.8%, P = 0.0005), achieving the endpoint of back pain improvement >30%..."

P1/2 data • Back Pain • CNS Disorders • Lumbar Back Pain • Pain

DiscGenics Announces Research Collaboration with the U.S. Department of Veterans Affairs for Tissue-Engineered Discs with Discogenic Cells (PRNewswire) - "DiscGenics, Inc...today announced a basic science Cooperative Research and Development Agreement (CRADA) with the U.S. Department of Veterans Affairs (VA) to assess the feasibility of using DiscGenics' proprietary Discogenic Cells with the VA's whole, tissue-engineered endplate-modified disc-like angle ply structure (eDAPS)....Through this ongoing collaboration, researchers have determined that eDAPS manufactured using DiscGenics' proprietary nucleus pulposus and annulus fibrosus derived Discogenic Cells are mechanically and biochemically distinct from eDAPS using mesenchymal stem cells (MSCs)."

Licensing / partnership • Back Pain • CNS Disorders • Lumbar Back Pain • Pain

DiscGenics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to IDCT for Degenerative Disc Disease (PRNewswire) - "DiscGenics, Inc...announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to Injectable Disc Cell Therapy (IDCT or rebonuputemcel), an injectable, allogeneic discogenic progenitor cell therapy for the treatment of symptomatic lumbar degenerative disc disease (DDD)."

FDA event • Back Pain • CNS Disorders • Lumbar Back Pain • Musculoskeletal Pain • Pain

Study to Evaluate the Safety and Preliminary Efficacy of IDCT, a Treatment for Symptomatic Lumbar Intervertebral Disc Degeneration (clinicaltrials.gov) - P1 | N=60 | Completed | Sponsor: DiscGenics, Inc. | Phase classification: P1/2 ➔ P1

Phase classification • Musculoskeletal Diseases

DiscGenics Announces Positive Two-Year Clinical Data from Study of Discogenic Progenitor Cell Therapy for Degenerative Disc Disease (PRNewswire) - P1/2 | N=60 | NCT03347708 | Sponsor: DiscGenics, Inc. | "DiscGenics, Inc...announced positive two-year clinical data from its first-in-human clinical study of IDCT (rebonuputemcel), an allogeneic discogenic progenitor cell therapy for lumbar degenerative disc disease (DDD)....Key findings include: The primary safety endpoint of the study was achieved with no subjects in the IDCT treatment groups experiencing treatment-emergent serious adverse events (TESAEs)....In the low dose IDCT group (3,000,000 cell/mL; n=20), there was a trend in improvement of clinical outcomes, though inconsistent. While the vehicle control group (n=10) resulted in some pain relief, it was not associated with clinically meaningful improvements in function or quality of life..."

P1/2 data • Back Pain • CNS Disorders • Lumbar Back Pain • Musculoskeletal Pain • Pain

Study to Evaluate the Safety and Preliminary Efficacy of IDCT, a Treatment for Symptomatic Lumbar Intervertebral Disc Degeneration (clinicaltrials.gov) - P1/2 | N=60 | Completed | Sponsor: DiscGenics, Inc. | Unknown status ➔ Completed | Trial completion date: Apr 2021 ➔ Nov 2022 | Trial primary completion date: Apr 2020 ➔ Feb 2022

Trial completion • Trial completion date • Trial primary completion date • Musculoskeletal Diseases

Study to Evaluate the Safety and Preliminary Efficacy of IDCT, a Treatment for Symptomatic Lumbar Disc Degeneration (clinicaltrials.gov) - P1/2 | N=38 | Completed | Sponsor: DiscGenics, Inc. | Unknown status ➔ Completed | Trial completion date: Aug 2021 ➔ Nov 2022 | Trial primary completion date: Feb 2021 ➔ Feb 2022

Trial completion • Trial completion date • Trial primary completion date • Musculoskeletal Diseases

Two-Year Clinical Data for DiscGenics's Progenitor Cell Therapy for Lumbar Disc Degeneration to Be Presented at NASS 2022 (PRNewswire) - "DiscGenics, Inc...announced that Matthew F. Gornet, MD, Board Certified Spine Surgeon at The Orthopedic Center of St. Louis, will present two-year follow-up data from the Company's first-in-human clinical study of its allogeneic discogenic progenitor cell therapy (IDCT) for lumbar disc degeneration at the North American Spine Society (NASS) 37th Annual Meeting to be held October 12 – 15, 2022 at McCormick Place West in Chicago....Dr. Gornet will present final two-year clinical data for three key patient-reported outcome measures of pain, function, and quality of life, as well as MRI results across four study groups: low dose IDCT (n=20), high dose IDCT (n=20), vehicle (n=10) and saline placebo (n=10)."

P1/2 data • Back Pain • CNS Disorders • Lumbar Back Pain • Musculoskeletal Pain • Pain

DiscGenics to Present at 2022 Cell & Gene Meeting on the Mesa (PRNewswire) - "DiscGenics, Inc...announced that Flagg Flanagan, Chief Executive Officer and Chairman will present virtually at the annual Cell & Gene Meeting on the Mesa to be held October 11-13 in Carlsbad, California and livestreamed globally. Flanagan will provide an update on the Company's first-in-human clinical study of its allogeneic discogenic progenitor cell therapy (IDCT) for lumbar disc degeneration. In the FDA-approved, prospective, randomized, double-blinded, controlled, multicenter study, high dose IDCT produced clinically meaningful, statistically significant improvements in low back pain, function, and quality of life by 12 weeks following intradiscal injection. As Flanagan will discuss, improvements were sustained at the 52- and 78-week interim readouts with additional data to follow at 104 weeks."

P1/2 data • Back Pain • CNS Disorders • Lumbar Back Pain • Musculoskeletal Pain • Pain

DiscGenics Announces Presentation of Positive Interim Clinical Data from Phase 1/2 Study of Cell Therapy for Degenerative Disc Disease (PRNewswire) - P1/2 | N=60 | NCT03347708 | Sponsor: DiscGenics, Inc. | "DiscGenics, Inc...today announced the presentation of positive interim data from its ongoing Phase 1/2 clinical trial of IDCT (rebonuputemcel), an allogeneic injectable discogenic cell therapy for degenerative disc disease (DDD), at Spine Summit 2022....The primary efficacy endpoint of the study (n=60) was achieved, with statistically significant improvement in back pain observed in the high dose IDCT group. For these patients, low back pain scores improved >30% as measured on a 100mm Visual Analog Scale (VAS) at Weeks 12...and 52....At these same timepoints, high dose IDCT also produced clinically meaningful, statistically significant improvements in function as measured by the Oswestry Disability Index (ODI) and quality of life as measured by the EQ-5D Index Score."

P1/2 data • Back Pain • CNS Disorders • Lumbar Back Pain • Pain

DiscGenics Completes Patient Enrollment in Japanese Clinical Study of Cell Therapy for Disc Degeneration as US Study Achieves One-Year Follow-up Milestone (DiscGenics Press Release) - "DiscGenics...today announced that it has completed enrollment in its Japanese safety study of IDCT, an allogeneic, injectable Discogenic Cell therapy for lumbar disc degeneration, a major cause of chronic low back pain....This Japanese 38-subject trial is being conducted at seven sites across the Country and passed the first of two planned mid-trial safety reviews by an Independent Data Monitoring Committee (IDMC)... All currently enrolled subjects have completed their one-year study visits in our U.S. clinical study of IDCT for lumbar disc degeneration. The study has a one-year extension period, so subjects will continue to be evaluated for safety, primary efficacy, and secondary outcome measures at two additional study visits over the coming year."

Enrollment closed • Trial status • Back Pain • CNS Disorders • Lumbar Back Pain • Pain

DiscGenics Raises $50 Million in Series C Funding (PRNewswire) - "DiscGenics, Inc....today announced it has raised $50 million in a Series C funding round led by Ci:z Investment LLP with participation from new investors, Eagle Fund SP1 LLP, Medical Incubator Japan (MIJ), and CareNet of Japan....DiscGenics will use the new capital to support ongoing clinical trials of its injectable Discogenic Cell Therapy (IDCT) for lumbar degenerative disc disease (DDD), to fund future commercialization activities in the U.S. and Japan, and for the scale up and scale out of its allogeneic cell manufacturing facility in Salt Lake City, UT."

Financing • Back Pain • CNS Disorders • Pain

DiscGenics announces completion of enrollment in U.S. phase 1/2 clinical trial of discogenic cell therapy for degenerative disc disease (OrthoSpineNews) - "DiscGenics, Inc....today announced that it has completed enrollment in its Phase 1/2 first-in-human U.S. clinical study of IDCT, an allogeneic, injectable Discogenic Cell therapy for degenerative disc disease (DDD)."

Enrollment closed

Study to Evaluate the Safety and Preliminary Efficacy of IDCT, a Treatment for Symptomatic Lumbar Intervertebral Disc Degeneration (clinicaltrials.gov) - P1/2; N=60; Active, not recruiting; Sponsor: DiscGenics, Inc.; Recruiting ➔ Active, not recruiting

Clinical • Enrollment closed

DiscGenics announces publication of preclinical data for disc degeneration treatment in The Spine Journal (PRNewswire) - "DiscGenics, Inc....today announced the online publication of a study in The Spine Journal...that summarizes the results of preclinical testing for Discogenic Cells, the active ingredient in IDCT, DiscGenics' investigational cell therapy for disc degeneration."

Preclinical

DiscGenics receives FDA fast track designation for cell therapy for disc degeneration (PRNewswire) - "DiscGenics, Inc....today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its investigational cell therapy, IDCT, currently being evaluated in regulator-allowed clinical trials in the U.S. and Japan for the reduction in pain and disability associated with degenerative disc disease (DDD), a major cause of chronic low back pain."

Fast track designation

Study to Evaluate the Safety and Preliminary Efficacy of IDCT, a Treatment for Symptomatic Lumbar Disc Degeneration (clinicaltrials.gov) - P1/2; N=38; Recruiting; Sponsor: DiscGenics, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open

First Utahns treated in nationwide FDA-allowed clinical study of locally developed cell therapy for chronic low back pain (PRNewswire) - "DiscGenics, Inc....today announced the first Utahns have been treated in its nationwide clinical study of IDCT, a locally developed injectable disc cell therapy for degenerative disc disease (DDD)....The clinical study, which passed its initial planned safety review earlier this year, is designed to evaluate the safety and preliminary efficacy of IDCT, a homologous and allogeneic cell therapy, in the treatment of mild to moderate DDD."

Trial status