Phase 1/2 studies evaluated PDA001 in 50 participants with CD.
New analyses of 3 legacy Phase 1, Phase 1b/2a and Phase 1b studies have revealed that patients with Crohn disease (CD) treated with Celularity’s legacy placental-derived mesenchymal-like adherent stromal cell (MLASC) therapy PDA001 experienced a clinically meaningful benefit for up to 2 years.1
“We pioneered the development of our placental-derived allogenic therapy because we believe that our technology has broad application across a number of therapeutic areas with high unmet need,” Robert J. Hariri, MD, PhD, founder, chairperson and chief executive officer,Celularity, said in a statement.1 “We see promising signals like these CD data in several other therapeutic areas, which reinforces our confidence in potential therapeutic benefits of MLASCs as we advance the development of these therapeutic options for patients. These signals are guiding our investment decision to progress our novel genetically modified allogeneic placental-derived MLASC, APPL-001, in CD, where we believe this cell therapy candidate could make a significant difference.”
The studies determined that the optimal treatment schedule of MLASC therapy to be a single course of 2 infusions 7 days apart, with no further maintenance treatment required during the study and follow-up period. In addition to the 2 studies, Celularity is submitting another abstract titled “Randomized, Double-Blind, Placebo-Controlled Dose-Escalation Study to Determine the Safety and Efficacy of Intra Venous Infusion of Human Placenta-Derived Cells for the Treatment of Crohn's Disease,” for presentation for an upcoming scientific meeting.
“These encouraging data showed clinically meaningful and durable response rates. For example, in a phase 1 study, a clinical disease remission rate of 50 percent at two years as measured by the Crohn’s Disease Activity Index (CDAI) was observed following a single treatment course. Additionally, the clinical response rate at 2 years was 83% (5 of 6 patients). The therapy was generally well-tolerated with no dose-limiting toxicities observed,” Sharmila Koppisetti, MD, senior vice president, non-oncology clinical affairs, added to the statement.1
The phase 1/2 studies (NCT01155362) evaluated PDA001 in 50 participants with CD for safety and tolerability.2,3 Primary endpoints included reductions in CDAI scores from baseline and secondary endpoints included induction of a clinical remission based on CDAI scores.
“The immune pathways involved in inflammation and fistula formation associated with Crohn’s disease are now well-characterized and we believe the broad activity of APPL-001 may allow us to target some of the key drivers of these processes. Despite therapeutic advances, Crohn’s disease remains an area of significant morbidity and requires therapies to target both the inflammatory processes and fistula formation. Crohn’s disease is representative of a number of autoimmune inflammatory diseases,” Koppisetti added.1
Celularity has since advanced its MLASC platform into 2 candidates in its pipeline, APPL-001 also for CD and PDA-002 for facioscapulohumeral muscular dystrophy, both of which are in preclinical studies.