Item 7.01 Regulation FD Disclosure.
On
The information in this Item 7.01 of this Current Report on Form 8K (including Exhibit 99.1) shall not be deemed "filed" for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the "Exchange Act"), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Events. Recent Developments
On
The Company and
In the ongoing Phase 1/2 MGT009 clinical trial, each patient was treated with subretinal delivery of AAV-RPGR in one eye and the patient's other eye served as an untreated control. The primary endpoint of the trial is safety, with secondary endpoints assessing changes in visual function at pre-specified timepoints post-treatment. Baseline values were determined in triplicate.
In the dose escalation phase of the trial, data at the twelve-month time point demonstrated statistically significant improvement in retinal sensitivity in treated eyes in both the low (n=3) and intermediate (n=4) dose cohorts, with six of seven patients demonstrating improved or stable vision in the treated eye one year after treatment.
Data Summary Retinal sensitivity
XLRP is characterized by progressive deterioration of the visual field. Octopus 900 full-field static perimetry and MAIA microperimetry were employed to determine change in retinal sensitivity following intervention.
Perimetry is a sensitive, standard-of-care measure of retinal function that reproducibly determines retinal sensitivity both cross-sectionally and longitudinally, thereby accurately evaluating disease progression over time.
At the 12-month analysis, compared to baseline:
Six out of seven patients in the low (n=3) and intermediate (n=4) dose cohorts
? demonstrated improvement or stability in retinal sensitivity in the treated
eye.
Statistically significant differences in mean retinal sensitivity were observed
? between treated and untreated eyes in the intermediate dose cohort: 1.05 dB;
(90% CI: 0.81, 1.29).
Statistically significant differences were observed in central visual field
? progression rate (V30) between treated and untreated eyes in the low: 1.10
dB-sr/year; (90% CI: 0.10, 2.10) and intermediate: 1.26 dB-sr/year; (90% CI:
0.65, 1.86) dose cohorts.
? Efficacy signals were observed at the first post-treatment assessments at three
months, with improvements sustained or increased at 12 months.
2 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
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 COVID19 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 COVID19 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 10Q for the quarter ended
3
2020, as such factors may be updated from time to time in our other filings with
the
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits. Exhibit No. Description 99.1 Press release ofMeiraGTx Holdings plc , datedNovember 13, 2020 . 104 Cover Page Interactive Data File (formatted in Inline XBRL and contained in Exhibit 101) 4
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