Vision-guided mobility
Markedly impaired mobility in low illumination is a hallmark symptom of XLRP. As part of the trial, patients completed a vision-guided mobility maze at baseline and nine months to assess their ability to navigate across a broad range of controlled light levels (1 lux = deep twilight, 4 lux = residential street lighting, 16 lux = twilight conditions, 64 lux = car park and 256 lux = office work). These results were first presented at the EURETINA 2020 Virtual Congress in October 2020.
At the nine-month analysis, compared to baseline:
| ● | Five of six patients demonstrated improvement in walk time for the treated eye at lux levels 1, 4 or 16. |
| ● | Significant improvement was observed between treated and untreated eyes in the low and intermediate dose cohorts (n=6) at 1 lux, -16.1 seconds (90% CI: 9.91, 22.1) and 4 lux, -3.71 seconds (90% CI: 2.83, 4.96); with the greatest improvement at the lowest light level (1 lux). |
| ● | Vision-guided mobility assessment was not carried out in the high dose cohort at the nine-month timepoint due to a protocol revision implemented to align with the dose-expansion cohort assessment schedule. |
Safety and tolerability
Safety data obtained to date continue to suggest AAV-RPGR is well-tolerated. No dose-limiting events occurred. As previously presented, signs of inflammation were observed in two of three patients in the high dose cohort, which may have been associated with decreased activity of the AAV-RPGR treatment in these patients. Inflammation was effectively managed with an extended steroid protocol.
Based on the safety and efficacy profile, the low and intermediate doses are being evaluated in the ongoing randomized, controlled expansion portion of the Phase 1/2 study, which completed enrollment in the first half of 2020.
The Company and development partner Janssen are preparing to initiate the pivotal Phase 3 Lumeos clinical trial of AAV-RPGR for the treatment of patients with XLRP.
Forward Looking Statement
This Form 8-K contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this Form 8-K that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of AAV-RPGR, plans to advance AAV-RPGR into Phase 3 clinical trial and anticipated milestones regarding our clinical data and reporting of such data and the timing of results of data, including in light of the COVID‑19 pandemic, as well as statements that include the words “expect,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “should,” “anticipate” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, raise additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID‑19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10‑Q for the quarter ended September 30,
