Bayer-owned BlueRock Therapeutics’ cell therapy hopeful has shown clinically meaningful improvements 36 months in an early-phase Parkinson’s disease study.
Bemdaneprocel improved MDS-unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part II and III scores, as well as Parkinson’s disease diary measures. Blue Rock did not specify whether the data was statistically significant.
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In the Phase I trial (NCT04802733), MDS-UPDRS Part III scores revealed that patients treated with high-dose bemdaneprocel experienced a clinically meaningful 17.9-point reduction compared with baseline.
This trend was also observed in the low-dose group, with patients experiencing a 13.5-point drop from baseline.
Meanwhile, bemdaneprocel also demonstrated improvements to MDS-UPDRS Part II, prompting a mean 4.3 drop compared with baseline.
MDS-UPDRS Part II is commonly used to measure a patient’s self-reported capacity to do daily activities, while MDS-UPDRS Part III characterises motor symptom severity.
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By GlobalDataThese measurements were taken in patients in the “off” state who were receiving bemdaneprocel.
“On” or “off” states are used to categorise the motor symptom severity experienced by patients with Parkinson’s, which can fluctuate over time. “On” refers to when symptoms are controlled, while “off” is characterised by a worsening of Parkinson’s presentation.
Through the assessment of patient Parkinson’s disease diaries, it was also determined that high dose bemdaneprocel triggered a mean increase of one hour in time spent in the “on” state.
Patients in the high-dose cohort also spent less time in the “off” state, with figures dropping by 0.93 hours compared with baseline at the three-year mark.
The drug was also proven to be safe and tolerable, with no treatment-related adverse events (TEAEs) reported. The transplanted cells also survived in the brain post-immunosuppression therapy discontinuation.
Bemdaneprocel shows potential
According to Claire Henchcliffe, one of the study’s principal investigators and chair of the UC Irvine School of Medicine’s Department of Neurology, bemadaneprocel represents a “new approach to restoring dopamine inputs,” which are lost in Parkinson’s patients.
“The new 36-month data is a critical next step in evaluating the long-term safety of bemdaneprocel,” Henchcliffe stated.
While she stresses that caution must be taken when evaluating the positive outcome trends, Henchcliffe noted that “initial signals are there, particularly in the higher dose cohort”.
The therapy was previously granted Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA), and is claimed to be the most clinically advanced Parkinson’s disease cell therapy treatment in the US.
To further explore the efficacy and safety of bemdaneprocel, BlueRock will take the cell therapy to a Phase III trial named ExPDite-2.
The future of Parkinson’s treatment
Though a range of medications have got the FDA green light in Parkinson’s disease, there are currently no approved medications that are able to slow or halt disease progression.
This leaves an area of huge unmet need for patients, which pharma companies around the globe are now looking to address.
One of these companies is Swiss pharma giant Roche, which is advancing its asset for early stage disease, prasinezumab into Phase III, despite the drug failing to reach its primary endpoint in the Phase IIb PADOVA study (NCT04777331). The monoclonal antibody (mAb) works by selectively binding to alpha-synuclein (α-syn) aggregations.
Annovis Bio has also taken its Parkinson’s hopeful, buntanetap tartrate, to Phase III, after the drug showed signs towards a disease modifying impact in mid-stage trials.
Though there is still a way to go before a disease-modifying therapy (DMT) gets to market for Parkinson’s, this could offer patients renewed hope.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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