AskBio has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for its investigational gene therapy, AB-1005.
The therapy, if approved will be used to slow disease progression and ameliorate motor outcomes in patients with Parkinson's disease.
This decision was based on a review of data provided by AskBio — including clinical resultrs from AB-1005's Phase Ib trial.
36 months of data collected within this trial revealed that AB-1005 was well tolerated and able to either improve or stabilise Parkinson's-relevant clinical scales compared with baseline measures.
This includes MDS-UPDRS scores and self-reported PD motor diaries, as well as trends related to the reduction in Parkinson's medication intake.
“The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with Parkinson’s disease and their loved ones,” said Gustavo Pesquin, CEO, AskBio.
“This milestone could potentially expedite the development of our important investigational gene therapy programme, and it highlights our promising data and the potential of AB-1005 for patients and the medical community."
The first participants in the Phase II REGENERATE-PD clinical trial have been randomised in the US, with the trial currently recruiting participants.
AskBio expects to conduct studies in the UK, Germany, Poland and other places in the US on top of this — which will likely open for enrollment by the middle of 2025.
“The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with Parkinson’s disease,” said Christian Rommel, Executive VP, Global Head of R&D and Member of the Pharmaceuticals Leadership Team at Bayer.
“This is the latest example of what can be achieved through the joint commitment of AskBio and Bayer to deliver breakthrough innovation for patients.”