Mesenchymal Stem Cell Therapy IND Cleared for Crohn Disease Fistulas

The FDA has cleared Ossium Health’s investigational new drug application for their mesenchymal stem cell (MSC) therapy OSSM-001 for the potential treatment of refractory perianal fistulas in patients with Crohn disease.¹

“The FDA’s clearance of our OSSM-001 product to enter clinical trials is a major milestone in our development of advanced therapies that leverage Ossium’s proprietary manufacturing platform to treat patients with diseases of inflammation,” Kevin Caldwell, chief executive officer and co-founder, Ossium, said in a statement.¹ 

OSSM-001 will be evaluated in a phase 1, double-blind, randomized, placebo-controlled trial which Ossium plans to initiate by the end of 2022. Participants in the trial will receive a single dose of 100 or 300 million allogeneic bone marrow-derived MSCs. The study will evaluate safety, tolerability, and feasibilty of OSSM-001 treatment, with secondary endpoints for fistula healing.

“OSSM-001 is the second Ossium cell therapy product to enter the clinic and the first of our MSC products to be used in patients. The initiation of this trial is a crucial step toward the development of a new treatment paradigm for patients with refractory perianal fistulas, which represent one of the most challenging manifestations of Crohn’s disease,” Caldwell added.¹

READ MORE: Mesenchymal Stem Cell Therapy Improves Joint Function in Rheumatoid Arthritis

Ossium is developing a similar, vertebral bone marrow-derived MSC therapy that is being evaluated in a phase 1/2 study (NCT05075811) for the potential treatment of refractory pouch fistula Crohn disease, also in collaboration with Cleveland Clinic. The study is evaluating safety and tolerability of the therapy as well as clinical healing.

Another MSC therapy, remestemcel-L, is being developed by Mesoblast, for the potential treatment of Crohn colitis. This therapy demonstrated rapid mucosal healing and disease remission in patients with refractory ulcerative (NCT04543994) or Crohn colitis (NCT04548583), according to results from a phase 1b/2a study recently published in the Journal of Crohn’s and Colitis.²

Remestemcel-L is being assessed at the Cleveland Clinic in up to 48 patients with refractory Crohn or ulcerative colitis at high risk of progressing to surgery. Participants receive a single dose of 150 to 300 million cells ofremestemcel-L or placebo delivered via direct injection using a 23 G sclerotherapy needle during colonoscopy. 

“Mesenchymal stromal cells (remestemcel-L) offer a safe therapeutic for the treatment of medically refractory Ulcerative Colitis and Crohn colitis,” principal investigator Amy L. Lightner, MD, associate professor of surgery, department of colon and rectal surgery, and associate chief, surgical research, Cleveland Clinic, said in a statement.³ “Early data suggests improved clinical and endoscopic scores as early as two weeks following remestemcel-L delivery.”

REFERENCES

1. Ossium Health Announces FDA Clearance of its Investigational New Drug Application for Treatment of Perianal Fistulizing Crohn’s Disease. News release. Ossium Health. March 16, 2022. https://www.businesswire.com/news/home/20220316005295/en/Ossium-Health-Announces-FDA-Clearance-of-its-Investigational-New-Drug-Application-for-Treatment-of-Perianal-Fistulizing-Crohn%E2%80%99s-Disease
2. Lightner A, Ream J, Nachand D, Fulmer C, Regueiro M, Steele S. P428 A Phase IB/IIA study of remestemcel-L, an allogeneic bone marrow derived mesenchymal stem cell product, for the treatment of medically refractory Crohn’s colitis: A preliminary analysis. JCrohns Colitis.2022. 16(S1); i412–i413. doi:10.1093/ecco-jcc/jjab232.555
3. Rapid improvement and remission in patients with refractory ulcerative colitis and Crohn’s Colitis after direct injection of remestemcel-L by colonoscopy. News release. Mesoblast Limited. February 20, 2022. https://www.biospace.com/article/releases/rapid-improvement-and-remission-in-patients-with-refractory-ulcerative-colitis-and-crohn-s-colitis-after-direct-injection-of-remestemcel-l-by-colonoscopy/?s=110