Voyager’s Parkinson’s gene therapy journey just got longer

Voyager Therapeutics is going full-steam ahead with a phase II trial of its Parkinson’s disease gene therapy candidate, VY-AADC. But yesterday the company poured cold water on hopes that this study might be enough for approval – and investors sent its stock down 12% in premarket trading. The company said in its third-quarter earnings announcement that the US FDA now considered the phase II study exploratory, appearing to row back from previous comments that, if successful, the trial might be enough for a filing. Although the topic dominated yesterday’s earnings call, Voyager’s chief executive, Andre Turenne, would only say that the company intended to meet the agency this year and that it had not changed its plan, which currently involves staggering the start of the phase II and III trials. Dosing is yet to begin in the 42-patient, placebo-controlled phase II study, which will evaluate an infusion of 2.5 x 10^12 vector genomes, between the mid and high doses used in VY-AADC’s phase I trial. That study found a promising response, measured using “on” time, with the middle dose, but disappointing results with the high dose; Voyager speculated that this might have been due to overactivity with the latter.

Selected novel Parkinson's disease projects in phase II development
Project Company Description Trial(s) Primary completion
ITI-214 Intra-Cellular Therapies PDE1 inhibitor NCT03257046 Reported
EPI-589 Sumitomo Dainippon Pharma Redox cofactor  NCT02462603 Dec 2018
Ferriprox Apotex Iron chelator Fairparkii, NCT02655315*; Sky, NCT02728843 Dec 2018; Aug 2019
Foliglurax Lundbeck mGluR4 regulator Ambled, NCT03162874 Apr 2019
Liraglutide (Victoza/Saxenda) Novo Nordisk GLP-1 receptor agonist NCT02953665* Jul 2019
CDNF Parkinson’s Project Herantis Pharma Cerebral dopamine neurotrophic factor NCT03295786 Aug-19
CX-8998 Cavion Calcium channel Cav3.2 blocker NCT03436953 Sep 2019
PRX002 Prothena Alpha-synuclein accumulation antibody Pasadena, NCT03100149 Mar 2020
VY-AADC Voyager Therapeutics AAV gene therapy NCT03562494 Dec 2020
GZ402671 Sanofi Glucosylceramide synthase inhibitor Moves-PD, NCT02906020 Mar 2021
BIIB054 Biogen Alpha-synuclein accumulation antibody Spark, NCT03318523 Mar 2021
*Investigator-sponsored. Source: EvaluatePharma.

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