The Hope Biosciences Research Foundation (HBRF) is eyeing a Phase III confirmatory trial for its stem cell therapy after the treatment displayed disease-modifying potential in a mid-stage study.
During the Phase II trial (NCT04995081) in patients with early to moderate Parkinson’s disease, the allogenic adipose-derived mesenchymal stem cell therapy (HB-adMSC) offered statistically significant improvements in motor function compared with patients receiving placebo – meeting the trial’s endpoint.
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This was measured through patient-reported Motor Experiences of Daily Living (MDS-UPDRS Part II) and clinician-rated Motor Function (MDS-UPDRS Part III) scores, with patients receiving the stems cells experiencing a placebo-adjusted -9.32-point reduction in MSD-UPDRS Part III value by the sixth and final infusion.
This notably exceeded the baseline -3.25-point minimal clinically important difference (MCID) that patients recognised as meaningful.
While the HBRF has not yet shared specific data pertaining to the safety of these mesenchymal stem cells, the non-profit noted that they were well tolerated within the treatment arm.
According to HBRF’s president, Donna Chang, the data from this trial suggests that “consistent, repeated treatment may be the most promising path forward for sustained enhancement in motor function for individuals living with Parkinson’s disease”.
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By GlobalDataFollowing the positive results of this trial, the HBRF hopes to take these stem cells to a confirmatory Phase III trial. To achieve this, the non-profit will close out meetings with the US Food and Drug Administration (FDA), which will help determine how the organisation can best balance patient and clinician-reported outcomes.
Taking steps to drug Parkinson’s
While a wide range of symptom-managing drugs have been approved for use in Parkinson’s disease, a disease modifying therapy (DMT) in this indication is yet to make it to market.
This has led companies in the seven major markets (7MM: the US, France, Germany, Italy, Spain, the UK and Japan) to home in on DMT research, with alpha-synuclein aggregation accounting for 26% of the current pipeline.
One alpha-synuclein therapy currently in late-stage development is Annovis Bio’s Posiphen (buntanetap tartrate), which displayed promise in early Parkinson’s patients with mild dementia during a Phase III trial.
Outside of this modality, Bayer-owned BlueRock Therapeutics is exploring the potential of its cell therapy, bemdaneprocel – which is now set to enter pivotal Phase III trials after it exhibited trends towards a disease-modifying impact in a Phase I trial.
Biopharma Cerevance is evaluating its non-dopaminergic GPR6 inhibitor, solengepras in the Phase III ARISE study (NCT06553027), despite the therapy failing to demonstrate a significant benefit over placebo in a Phase II study.
Meanwhile, Kenai recently dosed its first patient in a Phase I trial evaluating its cellular neuron replacement therapy, RNDP-001, in moderate-to-severe idiopathic Parkinson’s disease. Initial data related to the tolerability, safety and brain imaging impacts are expected in 2026.
GlobalData, parent company of Clinical Trials Arena, currently forecasts that the Parkinson’s disease market will be worth $7.9bn across the seven major markets in 2033.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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