Data on Parkinson Disease Gene Therapy, AXO-Lenti-PD, Anticipated in March


Axovant Sciences has announced that, in addition to receiving positive feedback from the FDA on its clinical development, it is expecting data from the first 2 patients dosed to read out in March 2019.

Dr Pavan Cheruvu

Axovant CEO Pavan Cheruvu, MD

Pavan Cheruvu, MD

In December, the FDA provided positive feedback to Axovant Sciences in a pre-Investigational New Drug (IND) meeting regarding its phase 2 clinical trial of AXO-Lenti-PD, an investigational gene therapy treatment for Parkinson disease.1

Ultimately, the regulatory agency informed Axovant that the totality of phase 1/2 clinical and pre-clinical data collected on ProSavin, the initial vector construct of the therapy, will possibly be supportive of the development program for its AXO-Lenti-PD treatment.

Now, in recent announcement of its upcoming clinical development milestones, Axovant CEO Pavan Cheruvu, MD, said that the company “is harnessing innovations in gene therapy to address the unmet needs of patients with debilitating neurological diseases,” and is anticipating that this year will emphasize its. “steadfast commitment to becoming a leader in the development of novel gene therapies.”

“We are eager to accelerate our pipeline of product candidates through important clinical milestones and data readouts in 2019,” he added.2

Michael Hayden, MBChB, PhD, who joined Axovant’s advisory board in December, said in a statement that “it was a pleasure to bring together these pre-eminent leaders in gene therapies and neurodegenerative disorders to discuss the latest developments in their fields. Insights gained from these discussions will help advance Axovant’s strategy as the company embarks on an active year with a deep pipeline of gene therapies.”2

In the face-to-face December meeting, the agency provided feedback on the target patient population, which has been decided as adult patients with Parkinson disease who are refractory to additional medical management due to motor complications, as well as a number of other notable points. It has deemed if the trial, now dubbed SUNRISE-PD (NCT03720418), is successful in showing meaningful safety and efficacy data, those data will constitute adequate early-phase, exploratory figures to possibly support a marketing application.

Additionally, the agency agreed that the current proposed manufacturing process and quality control testing is adequate for the clinical program. The FDA agreed, in principle, with the proposed approach to demonstrate compatibility between the current manufacturing process and the planned serum-free, suspension manufacturing process that Axovant plans to use to support scale-up and commercialization.

AXO-Lenti-PD, previously known as OXB-102, is designed to deliver 3 genes in vivo which encode the enzymes necessary for dopamine synthesis in the brain. It was licensed from Oxford Biomedica in June 2018.

In the phase 1/2 clinical study of the ProSavin precursor in 15 patients with Parkinson disease, the primary end point was met, showing a significant improvement from baseline in Unified Parkinson’s Disease Rating Scale (UPDRS) Part 3 score at both 6 and 12 months (P = .0001).2 The patients were grouped to receive either the low dose (n = 3), mid-dose (n = 6), and a high dose (n = 6). At baseline, patients recorded a mean UPDRS score of 38 (standard deviation [SD], 9), which improved to a mean of 26 at month 6 (P = .0001) and 27 at month 12 (P = .0001). Thus far, this improvement has been shown to be sustained for up to 6 years of follow-up.

During the first 12 months of follow-up, 54 drug-related adverse events (AEs) were reported, with 51 considered mild and 3 deemed moderate. The most common were increased on-medication dyskinesias (20 events, 11 patients) and on-off phenomena (12 events, 9 patients). No serious AEs related to the study drug or surgical procedure were reported.

SUNRISE-PD was initiated in the UK in late 2018, dosing its second patients in November. Thus far, both patients have shown tolerance to the surgical procedure and were discharged without any serious AEs. Data from these first 2 patients are anticipated in March 2019.

The primary efficacy measure of the randomized, sham-controlled portion of the study will be assessed at 12 months using data from Hauser patient diaries, while secondary efficacy data will be collected on the UPDRS Part 3 “off” scores of patients.




Announces Feedback from FDA Meeting Regarding AXO-Lenti-PD for Parkinson’s Disease and Progress in Ongoing SUNRISE-PD Phase 2 Clinical Trial [press release]. Basel, Switzerland:


Sciences; Published December 6, 2018. Accessed January 23, 2019.



Announces Presentation at JP Morgan Healthcare Conference and Anticipated 2019 Clinical Development Milestones [press release]. Basel, Switzerland:


Sciences; Published January 3, 2019. Accessed January 23, 2019.

3. Palfi S, Gurruchaga JM,


GS, et al. Long-term safety and tolerability of ProSavin,

a lentiviral

vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet. 2014;383(9923):1138-1146.


: 10.1016/S0140-6736(13)61939-X.

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