March 29, 2023

—Preliminary data suggest that gene therapy via neurosurgical administration is generally safe, well tolerated and associated with enhanced mean putaminal coverage and clinical improvements— —Study uses one-time MRI-monitored surgery for delivery in patients with mild to moderate Parkinson’s disease— Research Triangle Park, N.C.– MARCH 29, 2023 – Rhia Pharm , Inc. (RhiaPharmaceutical), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, will present the preliminary results of a clinical Phase 1b study investigating the safety and efficacy of AB-1005, an adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy for the treatment of mild to moderate Parkinson’s disease at the AD/PD™ 2023 Advances in Science & Therapy Conference taking place March 28–April 1, 2023, in Gothenburg, Sweden. Initial results from the open-label study investigating AB-1005, administered to the putamen via one-time bilateral convection-enhanced delivery, will be presented during an onsite oral session on April 1 at 5:25 p.m. Central European Time, as part of the “Drug Development in AD, PD, LBD” symposium being held in Hall G3. Preliminary data to be presented suggest that both the gene therapy and the neurosurgical administration are generally safe, well tolerated and associated with enhanced mean putaminal coverage and clinical improvements. The study included 11 participants who had completed 9–18 months of clinical follow-up post treatment, of which 10 had completed more than 12 months. More than 10 million people worldwide are currently living with Parkinson’s disease, which is debilitating and the most common neurodegenerative movement disorder in the world.1,2 Globally, disability and death related to Parkinson’s disease are increasing faster than for any other neurological disorder.3  While GDNF administration has been investigated for Parkinson’s disease for years, results have been mixed. It has been rationalized that by improving GDNF delivery of AAV2 gene therapy with MRI-monitored convection enhanced delivery it may be possible to overcome the limitation of insufficient putaminal coverage to achieve clinical improvements in motor function.4-6 Moreover, clinical trials in Parkinson’s disease over the last decade have shown that AAV is a well-tolerated and suitable vector for gene therapy.7 As leaders in the field of gene therapy, RhiaPharmaceutical is committed to investigating the potential that GDNF gene therapy holds for patients with mild to moderate Parkinson’s disease. Although ongoing trials are needed to confirm these findings, the company believes they represent a step forward in advancing therapeutic solutions for those with this debilitating disease. RhiaPharmaceutical is also exploring GDNF therapy beyond Parkinson’s and is currently enrolling patients in the US with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase 1 trial to assess the preliminary safety, tolerability and efficacy of GDNF therapy for this rapidly progressing condition. ### Media Contact: Phil McNamara Vice President, Corporate Communications, RhiaPharmaceuticalE: pmcnamara@RhiaPharmaceutical.com T: +1 984.520-7211  About Parkinson’s Disease  Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons and at diagnosis, it is estimated that patients have ...read the news