- Axovant Gene Therapies doesn't expect to begin enrolling patients with Parkinson's disease in a planned Phase 2 study of the company's experimental gene therapy until 2022, a delay tied to a longer-than-expected timeline for developing a new manufacturing process.
- Axovant, which pivoted to gene therapy research in 2018, relies on U.K. drugmaker Oxford Biomedica to make its Parkinson's disease treatment, known as AXO-Lenti-PD. Oxford informed Axovant in mid-October of delays in generating key manufacturing data and issues with the "fill/finish" step of production.
- Shares in Axovant plunged by 40% Friday morning as investors and analysts reset their expectations for development progress for AXO-Lenti-PD. The company has so far treated six Parkinson's patients with two doses of its gene therapy.
Manufacturing has proven a challenging hurdle to clear for several cell and gene therapy developers this year, with Axovant becoming the latest to have its plans come undone by production hang-ups.
In recent months, Sarepta Therapeutics, Voyager Therapeutics and Iovance Biotherapeutics have run into delays, with the Food and Drug Administration asking each for more information related to their manufacturing processes.
Making cell and gene therapies is particularly complex, and many of the methods being used are just now being scaled up by companies for larger clinical trials or potential commercialization. How a cell or gene treatment is produced can greatly impact its clinical profile, often more so than for the pills or protein drugs with which the industry is most familiar.
Unlike the other three companies, Axovant's issues don't involve the FDA. Rather, Axovant's manufacturing partner Oxford appears to have run into problems developing a "suspension-based manufacturing process" that the company planned to use for supplying a key study of AXO-Lenti-PD.
"As a result," Axovant said, "the company believes that it is unlikely that its planned randomized, sham-controlled trial of AXO-Lenti-PD will enroll patients by the end of calendar year 2021."
The drugmaker said it will provide an update on program timelines in early 2021, or as soon as they are further clarified.
AXO-Lenti-PD is a successor to a lentiviral gene therapy Oxford developed called Prosavin, which was studied in 15 patients with Parkinson's disease. Axovant licensed a modified version of Prosvain from Oxford in June 2018, four months after its former CEO David Hung left in the wake of clinical failures for the company's one-time Alzheimer's and dementia drug candidate, intepirdine.
AXO-Lenti-PD is designed to deliver three genes that encode for enzymes that synthesize dopamine, the critical neurotransmitter affected in Parkinson's disease.
On Friday, Axovant presented data for the four patients treated with AXO-Lenti-PD in the second phase of its currently ongoing trial. None experienced serious adverse events judged to be related to treatment, according to the company, although one of the four experienced unrelated confusion and a wound infection.
Axovant will have to complete development of the suspension-based manufacturing process before it can move forward with further testing.