MeiraGTx Announces Data from Ongoing Clinical Trial of AAV-RPGR for the Treatment of X-Linked Retinitis Pigmentosa to be Presented at AAO 2020 Virtual Meeting

Rhea-AI Summary

MeiraGTx Holdings plc (Nasdaq: MGTX) announced the presentation of twelve-month results from its ongoing Phase 1/2 clinical trial of AAV-RPGR, an investigational gene therapy for X-linked retinitis pigmentosa (XLRP), at the American Academy of Ophthalmology 2020 Virtual Meeting from November 13-15, 2020. The data, which will be shared by presenter Michel Michaelides, has shown significant improvements in vision and retinal sensitivity. AAV-RPGR has received FDA Fast Track and Orphan Drug designations, enhancing its development prospects.

Positive

• Successful twelve-month data from the Phase 1/2 trial indicating significant improvement in vision.
• AAV-RPGR received Fast Track and Orphan Drug designations from FDA.

Negative

• None.

12-Month Dose Escalation Data from Phase 1/2 Clinical Trial to be Presented

LONDON and NEW YORK, Oct. 28, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that twelve-month results from the ongoing Phase 1/2 clinical trial (NCT03252847) of AAV-RPGR, an investigational gene therapy for the treatment of X-linked retinitis pigmentosa (XLRP), will be presented in an oral session at the American Academy of Ophthalmology 2020 Virtual Meeting taking place November 13-15, 2020.

Details of the presentation are listed below. Data is embargoed until the date and time of presentation.

Title: Phase 1/2 Trial of AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa (XLRP): 12-month Results
Presenter: Michel Michaelides, BSc MB BS MD(Res) FRCOphth FACS
Date and Time: Friday, November 13, 2:55pm PST (5:55pm EST)
Session: Late Breaking Developments, Part I

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July and October 2020, MeiraGTx announced six-month and nine-month data, respectively, from the ongoing Phase 1/2 MGT009 clinical trial which demonstrated AAV-RPGR was generally well tolerated and produced significant improvement in vision, including improvement in retinal sensitivity and vision-guided mobility, in the dose escalation phase of the trial.

About AAV-RPGR
AAV-RPGR is an investigational gene therapy for the treatment of patients with X-linked retinitis pigmentosa (XLRP) caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. AAV-RPGR has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIME, ATMP and Orphan designations by the European Medicines Agency (EMA). 

About the Phase 1/2 MGT009 Clinical Trial
MGT009 is a multi-center, open-label Phase 1/2 trial (NCT03252847) of AAV-RPGR gene therapy for the treatment of patients with XLRP associated with disease-causing variants in the RPGR gene. MGT009 consists of three phases: dose-escalation, dose-confirmation, and dose-expansion. Each patient was treated with subretinal delivery of AAV-RPGR in the eye that was more affected at baseline. The patient’s other eye served as an untreated control. In dose-escalation (n=10), adults were administered low, intermediate, or high dose AAV-RPGR. The primary endpoint was safety. Visual function was assessed at baseline, three, six, nine and 12 months with Octopus 900 full-field static perimetry and mesopic fundus-guided microperimetry (MP); mean retinal sensitivity, visual field modeling and analysis (VFMA; Hill-of-vision volumetric measure), and pointwise comparisons were examined.

About X-Linked Retinitis Pigmentosa (XLRP)
XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.

About MeiraGTx
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

For more information, please visit www.meiragtx.com.

Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release 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 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, acquire 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 June 30, 2020, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management’s estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:
MeiraGTx
Elizabeth (Broder) Anderson
(646) 860-7983
elizabeth@meiragtx.com

Or

Media:
W2O pure
Christiana Pascale
(212) 257-6722
cpascale@purecommunications.com

FAQ

What are the twelve-month results of the Phase 1/2 clinical trial for MGTX?

The twelve-month results indicate significant improvements in vision and retinal sensitivity, suggesting the potential efficacy of AAV-RPGR in treating XLRP.

When will the results of MGTX's clinical trial be presented?

The results will be presented at the American Academy of Ophthalmology virtual meeting on November 13, 2020.

What is the significance of AAV-RPGR in the treatment of XLRP?

AAV-RPGR is designed to deliver functional copies of the RPGR gene to the retina, aiming to improve visual function in patients with XLRP.

What designations has AAV-RPGR received from the FDA?

AAV-RPGR has received Fast Track and Orphan Drug designations from the FDA, which can expedite its development and review process.

What is the focus of MeiraGTx's clinical development?

MeiraGTx is focused on developing gene therapies for inherited retinal diseases, neurodegenerative diseases, and xerostomia.