The FDA has granted investigational product SB623 from SanBio a Regenerative Medicine Advanced Therapy (RMAT) Designation as a result of recent clinical evidence.
SB623 is a proprietary, cell-based investigational product being tested for effectiveness in treating different neurological conditions. The recent RMAT designation result of new clinical evidence from the Phase 2 Study of Modified Stem Cells in Traumatic Brain Injury Trial (STEMTRA).
The drug candidate is reportedly made from modified and cultured adult bone marrow-derived mesenchymal stem cells that undergo temporary genetic modification, according to a statement from SanBio. When implanted directly into injured nerve tissue in the brain, SB623 is expected to trigger natural regenerative abilities that will recover lost motor functions.
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In the study that led to this new designation, 61 patients between the ages of 18-75, 12 months post-TBI, with a Glasgow Outcome Scale Extended Score of 3-6 were included from 13 surgical and 18 assessment sites in the U.S, Japan, and the Ukraine. The primary endpoint of the study was the mean change from baseline in Fugl-Meyer Motor Scale (FMMS), which measures the changes in motor impairment at six months.
The study met its primary endpoint—patients treated with SB623 achieved an average of an 8.3-point improvement from baseline in FMMS at 23 weeks (p=0.040). 39.1% of patients achieved a 10 or more-point improvement compared to one control patient. No new safety signals were identified. The most common adverse events reported were headaches.
“TBIs are one of the most common health conditions worldwide that often cause long-term complications or death. We look forward to working with the FDA on a potentially accelerated clinical development program to address this serious unmet medical need,” Bijan Nejadnik, MD, Chief Medical Officer and Head of Research says in a statement.
The Regenerative Medicine Advanced Therapy Designation is reserved for new regenerative medicine therapies intended to treat, reverse, or cure a serious condition and is granted to those investigational products whose clinical evidence indicates the therapy as the potential to address unmet medical needs. The designation also offers sponsors the eligibility for expedited development and regulatory review, as well as earlier and more frequent consultation with the FDA and potential for Priority Review and Accelerated Approval.
According to a release, SanBio expects a phase 3 trail for SB623 to begin after January 2020.