Hope Biosciences Reports Positive Phase II Results for Parkinson’s Stem Cell Therapy
The Phase II trial showed significant motor function improvements and safety for an allogeneic stem cell therapy in Parkinson’s disease.
Key findings
- Hope Biosciences Research Foundation reported positive top-line Phase II results for an allogeneic adipose-derived mesenchymal stem cell therapy in early to moderate Parkinson’s disease.
- In a 60-person randomized, double-blind study, the clinician-rated motor function endpoint (MDS-UPDRS Part III) showed a significant treatment effect over placebo by the sixth infusion.
- The nonprofit says the therapy was safe and tolerable, and it plans to discuss next steps with the FDA.
Hope Biosciences Research Foundation (HBRF) reports positive top-line results from a Phase II trial evaluating its allogeneic adipose-derived mesenchymal stem cell therapy, HB-adMSCs, in patients with early- to moderate-stage Parkinson’s disease (PD).
Trial design and endpoints
The trial (NCT04995081) was a balanced randomized, double-blind, single-center study that enrolled 60 participants (30 treatment, 30 placebo). Participants received six intravenous infusions of 200 million stem cells over 32 weeks, with end of study at 52 weeks.
Primary endpoints were described as clinically significant changes in motor function using:
- MDS-UPDRS Part II (Motor Experiences of Daily Living; patient-reported)
- MDS-UPDRS Part III (Motor Function; clinician-rated)
Top-line efficacy results (clinician-rated Part III)
HBRF reports the trial met its primary endpoint, with statistically significant improvements in motor function for the treatment group versus placebo.
The clinician-rated MDS-UPDRS Part III demonstrated a progressive and clinically meaningful treatment effect. Improvements in the treatment group increased over successive infusions, with effect sizes growing over time (Cohen’s d: Infusion 4 = 0.34, Infusion 5 = 0.40, Infusion 6 = 0.87).
By the sixth and final infusion, the treatment group achieved a mean change from baseline of -9.82 points versus -0.50 in placebo (RMA adjusted mean difference -9.32; 95% CI [-15.11, -3.54]; p=0.0023), substantially exceeding the MCID of -3.25. Bayesian analyses reinforced these findings, confirming statistical and clinical relevance. Treatment was safe and tolerable in both groups.
What HBRF says it learned about dosing frequency and outcome measures
“The timing of patient improvements is emerging as a key takeaway,” says Donna Chang, President, HBRF. “Early infusions showed small improvements in motor function. By the sixth infusion, however, the treatment group showed the largest cumulative improvement. At end of study, we saw a decline in scores from that high point. Taken together, this means that improvements in motor function are possible through treatment with this cellular therapeutic, and that consistent, repeated treatment may be the most promising path forward for sustained enhancement in motor function for individuals living with Parkinson’s Disease.”
Chang also pointed to differences between clinician-rated and patient-reported outcomes as a trial design consideration.
“The subjective nature of patient reporting is something that must be balanced in trial design through inclusion of more objective data-gathering mechanisms that are vital for evaluating therapeutic benefit,” expounds Chang. “Taking all the data together, in this trial there is a clear treatment effect. We look forward to close out meetings with FDA, and a hopefully fruitful conversation about how we as a community of researchers can continue to balance patient- and clinician-reported outcomes.”
Company snapshot (from the release)
HBRF is a 501(c)(3) nonprofit organization. The group says it has completed six FDA-authorized protocols in PD, including what it describes as a global-first Intermediate Sized Expanded Access protocol for patients aged 76 years and older. It also says this Phase II study is its second clinical trial using allogeneic cellular therapeutics, and that allogeneic approaches can broaden access by lowering manufacturing costs and enabling treatment for individuals who cannot bank their own stem cells.
“We have had the privilege of serving a significant number of men and women living with Parkinson’s, with extremely diverse disease stories,” continues Chang. “For this trial, we are eager not only for results of the detailed analysis currently underway, but also to look across the research suite for trends that may inform treatment pathways for Parkinson’s in the future. With these encouraging Phase II results in hand, we are hopeful to advance to a Phase III confirmatory trial that could bring us closer to a meaningful new therapeutic option for patients.”
More information: hopebio.org.
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