Axovant Reports Positive Interim Results From First Cohort Of SUNRISE-PD Phase 2 Trial Of AXO-Lenti-PD Gene Therapy In Parkinson’s Disease

NEW YORK and BASEL, Switzerland, March 11, 2019 (GLOBE NEWSWIRE) -- Axovant (Nasdaq: AXGT), a clinical-stage company developing innovative gene therapies, today reported 3-month data from the first dose cohort in the open-label, dose-escalation portion of the ongoing SUNRISE-PD Phase 2 trial of AXO-Lenti-PD for the treatment of Parkinson’s disease. The cohort consisted of two patients with advanced Parkinson’s disease who received a one-time administration of the lowest dose of AXO-Lenti-PD (4.2x106 TU). AXO-Lenti-PD was generally well-tolerated, and no serious adverse events were reported.

“Our focus in this first cohort of the SUNRISE-PD study was on the safety and tolerability of AXO-Lenti-PD, as well as the evaluation of efficacy using well-validated, objective measures. These early data support the safety of the lowest dose of AXO-Lenti-PD, similar to what was observed with the earlier generation construct, ProSavin, and also suggest substantially greater biological activity than the highest dose of ProSavin previously tested,” said Dr. Gavin Corcoran, Axovant’s Executive Vice President of Research and Development. “These findings are highly encouraging, and we look forward to advancing to higher dose cohorts where we will explore the full clinical potential of AXO-Lenti-PD in patients with Parkinson’s disease.”

The Unified Parkinson’s Disease Rating Scale (UPDRS) Part III score is a physician-rated scale assessing motor function, ranging from 0 to 108 with lower scores indicating improvement. The OFF score is assessed after patients are washed out of their oral levodopa therapy, thereby capturing the benefit of therapy without the potentially confounding effect of background medical treatment. Patients in the first cohort experienced an average UPDRS Part III (motor) OFF score improvement of 25 points at 3 months after administration of AXO-Lenti-PD, representing an average improvement of 42% from baseline. Individual patient improvements at 3 months were 14 points and 36 points, respectively.

Improvements were seen across all subparts of the UPDRS scale, with an average UPDRS Total OFF score improvement of 54.5 points at 3 months after receiving AXO-Lenti-PD, representing an average improvement of 55% from baseline. Patients experienced an average improvement of 22 points from baseline on the UPDRS Part II (activities of daily living) OFF score, and an average improvement of 7 points from baseline on the UPDRS Part IV (complications of therapy) OFF score.

Taken together, these results suggest that the lowest dose of AXO-Lenti-PD at 3 months may have greater efficacy compared to the highest dose of ProSavin previously tested. A detailed summary of results on the UPDRS OFF scale for AXO-Lenti-PD from the SUNRISE-PD study and for ProSavin from a prior clinical trial is shown in Table 1 below.

Table 1

MeasureAXO-Lenti-PD Cohort 1

(Low Dose: 4.2x106 TU)

at 3 months (N=2)*

ProSavin Cohort 3

(High Dose: 1.0x108 TU)

at 3 months (N=6)**

UPDRS Total OFF Score. Range from 0 to 199
Month 344.561.3
Improvement from Baseline 54.518.0
UPDRS Part I OFF Score (Mentation, Behavior and Mood). Range from 0 to 16
Month 30.50.8
Improvement from Baseline 0.51.7
UPDRS Part II OFF Score (Activities of Daily Living). Range from 0 to 52
Month 37.519.3
Improvement from Baseline 22.02.3
UPDRS Part III OFF Score (Motor). Range from 0 to 108
Month 334.034.0
Improvement from Baseline 25.010.8
UPDRS Part IV OFF Score (Complications of Therapy). Range from 0 to 23
Month 32.57.2
Improvement from Baseline 7.03.2

  These data are based on a cross-trial comparison and not a head-to-head clinical trial, and as a result, these data may not be directly comparable

*  Ongoing SUNRISE-PD Phase 2 trial of AXO-Lenti-PD

** Prior Phase 1/2 open-label trial of ProSavin

Patients in Cohort 1 also experienced a mean 18% improvement in dyskinesia as measured by the Rush Dyskinesia Rating Scale ON score, which assesses functional disability during activities of daily living while patients are on oral levodopa.

Additionally, a patient-recorded diary was collected from patients in Cohort 1. Although variability was present in the patient-recorded diary entries, both patients exhibited improvement in diary ON time with dyskinesia, with an average reduction of 3.5 hours (57%) from baseline. An improvement was also observed in diary ON time with troublesome dyskinesia, with an average reduction of 1.3 hours (85%) from baseline. Results from the patient-recorded diary are summarized in Table 2 below.

Table 2

Diary MeasureBaselineMonth 3Improvement from Baseline
ON time without dyskinesia8.0 hours10.1 hours2.1 hours
ON time with dyskinesia6.1 hours2.6 hours3.5 hours
ON time without troublesome dyskinesia12.5 hours12.4 hours-0.1 hours
ON time with troublesome dyskinesia1.5 hours0.2 hours1.3 hours
OFF time1.8 hours3.4 hours-1.6 hours

At month 3, the average levodopa equivalent daily dose (LEDD) was decreased by 208 mg for patients in Cohort 1. This represents an average reduction of 19% from baseline.

Based on initial feedback received from members of the Data Monitoring Committee (DMC), Axovant plans to proceed to the planned second dose cohort of AXO-Lenti-PD. The dose to be tested in the second cohort of patients is 1.4x107 TU. The first subject in this second cohort is expected to be dosed in the second quarter of 2019.

“This early data suggests that AXO-Lenti-PD has the potential to significantly improve motor function in patients with advancing Parkinson’s disease. The mechanism of action of AXO-Lenti-PD, which is designed to deliver all three genes necessary for endogenous dopamine biosynthesis, as well as our prior clinical experience with ProSavin, led us to expect that the major benefit would be in improving the OFF state – and the results so far are very encouraging in this regard,” said Dr. Roger Barker, one of the principal investigators on the SUNRISE-PD Phase 2 study and Professor of Clinical Neuroscience and Honorary Consultant in Neurology at the University of Cambridge and Addenbrooke's Hospital. “I am hopeful that this development program will translate into a significant new therapeutic option for patients with Parkinson’s disease.”

Pavan Cheruvu, M.D., chief executive officer of Axovant, will present at the Cowen and Company 39th Annual Health Care Conference on March 11, 2019 at 12:00 PM (Eastern Time). A copy of the Company’s presentation slides and link to a live webcast of the presentation can be found below.

About AXO-Lenti-PD

AXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinson’s disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration.

About Axovant

Axovant, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. The company’s current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations. For more information, visit

About Roivant

Roivant Sciences aims to improve health by rapidly delivering innovative medicines and technologies to patients. It does this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visit

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