Axovant Announces Positive 12-month Data on AXO-Lenti-PD and Provides Updates Across Gene Therapy Pipeline Programs


NEW YORK and BASEL, Switzerland, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Axovant Gene Therapies Ltd. (NASDAQ: AXGT), a clinical-stage company developing innovative gene therapies for neurological diseases, today announced recent progress in its gene therapy programs, including a 12-month update on its Parkinson’s program.

Exhibit 1
Average UPDRS Part III (motor) “OFF” score change from baseline to 12 months (cross-study comparison)

Major 2019 Accomplishments and Upcoming 2020 Milestones

AXO-Lenti-PD gene therapy in Parkinson’s disease:

  • At 12 months post-dosing, patients in the first dose cohort (4.2 x 106 TU) demonstrated an average 22-point change from baseline in motor function as assessed by the UPDRS Part III “OFF” score, which represents a 37% improvement. Individual patient improvements were 24-points and 20-points, respectively. Previously, at 6 months post-dosing, these patients demonstrated an average 17-point change from baseline, or 29% improvement, on the same scale. The 12-month timepoint is considered an important timeframe for assessment of therapeutic response, differentiation from sham/placebo effect, and durability of gene therapy in Parkinson’s disease.• AXO-Lenti-PD was observed to be well-tolerated with no serious adverse events attributable to the gene therapy.• Improvement in the UPDRS Part III “OFF” score in the first cohort exhibited evidence of dose response when compared to the low (n=3), medium (n=6), and high (n=6) dose cohorts of ProSavin that were previously evaluated in a separate Phase 1/2 study at 12 months (see Exhibit 1).• The UPDRS Part II “OFF” score, which assesses activities of daily living, showed a 13-point average change from baseline, or a 44% improvement from baseline to 12 months.• Only one of two patients in the first cohort was able to record a Hauser diary. Improvements were observed across various diary measures from baseline to 12 months for the single patient.• The PDQ-39 score index, a well-validated quality of life measure in Parkinson’s disease, demonstrated an average 15-point change from baseline, or 30% improvement from baseline to 12 months.
  • Enrollment continues in the second dose cohort of the SUNRISE-PD Phase 2 study of AXO-Lenti-PD. In Q1 2020, Axovant expects to present initial 6-month data from the first two patients in the second cohort (1.4 x 107 TU) who were administered a 3-fold higher dose of vector than the first dose cohort.
  • Based on the outcome of the dose-escalation part of the study and successful development of a suspension-based manufacturing process, Axovant expects to initiate the randomized, sham-controlled part of the SUNRISE-PD Phase 2 study by the end of calendar year 2020.
  • A recent placebo-controlled study in a non-human primate model of Parkinson’s disease published in Molecular Therapy: Methods and Clinical Development (Badin, 2019) compared two doses of AXO-Lenti-PD against control-group animals receiving a null vector. The study demonstrated statistically significant differences in PD clinical response scores at 6-months in this diseased-animal model (p

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