German pharmaceutical giant Bayer (ETR: BAYN) announced that its gene therapy subsidiary AskBio Inc. has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its investigational gene therapy AB-1005 as a treatment for Parkinson’s disease (PD).
Drug Profile
AB-1005 is an investigational gene therapy based on an adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. The therapy allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery.
Clinical Trial Results
Data from the open-label, non-controlled Phase Ib study showed that AB-1005 was well tolerated, with no treatment-related serious adverse events observed. In the moderate PD cohort, patients showed trends for improvement or stability on several PD-relevant clinical scales at 36 months compared to baseline, including the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and self-reported PD motor diaries. There were also trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]). Most participants in the mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.
Ongoing Phase II Study
The Phase II REGENERATE-PD study for AB-1005 is currently enrolling patients in the US.-Fineline Info & Tech
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