Japanese Clinical Study of DiscGenics' Cell Therapy for Disc Degeneration Passes Initial Safety Review

SALT LAKE CITY and TOKYO,Jan. 7,2020 -- DiscGenics,Inc.,a clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine,today announced that IDCT has passed the initial planned safety review of its double-blinded clinical study evaluating the allogeneic,injectable disc cell therapy in Japanese patients with mild to moderate degenerative disc disease (DDD),a major cause of chronic low back pain.

IDCT is a homologous,allogeneic,injectable cell therapy that utilizes biomedically engineered progenitor cells,known as Discogenic Cells,that have been derived from intervertebral disc tissue to offer a non-surgical,potentially regenerative solution for the treatment of mild to moderate DDD.

This prospective,randomized,double-blinded,sham-controlled study commenced earlier this year. It is being conducted at six prestigious universities across Japan to evaluate the safety and preliminary efficacy of IDCT at two dosage levels in Japanese patients with single-level,symptomatic lumbar DDD.

In this first planned safety review,an independent data safety monitoring committee (DSMC) reviewed unblinded data from the first five subjects,each of whom was randomly assigned to receive an intradiscal injection of the low dose IDCT study treatment,high dose IDCT study treatment or sham control.

The DSMC reported there were no safety issues and recommended that the study continue with no changes to the protocol.

"We are delighted to have cleared this initial planned safety review and to be advancing to our next phase of patient enrollment,as we believe IDCT has the potential to be a revolutionary treatment option for those suffering from chronic low back pain," said Flagg Flanagan,Chief Executive Officer and Chairman of the Board of Directors for DiscGenics. "This is a blinded,study where neither the treating clinicians nor the patients know what treatment is being administered. As a result,performance of periodic safety checks by an unblinded and independent body is essential to ensuring the ongoing safety of IDCT in a clinical setting."

About Degenerative Disc Disease

DDD is a painful,chronic and progressive disease that is characterized by inflammation and breakdown of extracellular matrix within the intervertebral disc. The condition is estimated to affect more than one million people,including 200,000 patients needing surgical intervention every year in Japan. Current treatment options for DDD are limited to physical rehabilitation programs and pain management,in the earlier stages. In the more advanced stages,oftentimes a patient's only option is surgical intervention to remove the painful disc(s),fuse two or more vertebral bones together and/or replace bone or tissue altogether. However,back surgeries often have limited success and may result in subsequent adjacent level degeneration.

About the Japanese IDCT Safety Study

The Japanese IDCT safety study is a prospective,sham-controlled,multicenter clinical study to evaluate the safety and preliminary efficacy of IDCT in subjects with single-level,symptomatic lumbar intervertebral disc degeneration. The trial is underway in six sites across the country and will enroll 38 subjects. Those subjects who meet all eligibility criteria are being randomized to one of four treatment cohorts: low dose IDCT (n=15),high dose IDCT (n=15),and sham (n=8). Each subject receives a single intradiscal injection of his or her assigned treatment into the target symptomatic lumbar intervertebral disc. Following treatment,subjects will be observed and evaluated for a period of six months,with a six-month extension period.

Through this study,IDCT is being evaluated under a Clinical Trial Notification (CTN) allowance by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) and will be regulated through a conditional approval process.

For more information,please visit https://clinicaltrials.gov/ct2/show/NCT03955315.

DiscGenics is also conducting an ongoing phase I/II prospective,vehicle- and placebo-controlled,multicenter clinical trial of IDCT in the U.S. Through this study,IDCT is being evaluated under an investigational new drug (IND) allowance by the U.S. Food and Drug Administration (FDA) and will be regulated as a drug-biologic through a therapeutics biologics application (BLA). Importantly,the U.S. study has passed all three planned mid-trial safety reviews and has commenced the final enrollment stage.

About DiscGenics

DiscGenics is a privately held,clinical stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. As the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc,DiscGenics believes it has a unique opportunity to harness the restorative potential of the human body to heal millions of patients suffering from the debilitating effects of back pain. DiscGenics' first product candidate,IDCT,is a homologous,injectable cell therapy that utilizes biomedically engineered progenitor cells derived from intervertebral disc tissue,to offer a non-surgical,potentially regenerative solution for the treatment of patients with mild to moderate degenerative disc disease. For more information,visit discgenics.com.

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