Item 7.01 Regulation FD Disclosure.
On December 16, 2020, MeiraGTx Holdings plc (the “Company”) issued a press release announcing that the Company would host a webcast and conference call on December 17, 2020 at 8:00 a.m. ET to provide an update on the Company’s AAV-hAQP1 clinical program for the treatment of radiation-induced xerostomia. A copy of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K (“Form 8-K”) and is incorporated herein by reference.
The information in this Item 7.01 of this Current Report on Form 8-K (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 December 17, 2020, the Company provided an update on the first cohort of patients (n=3) treated in the Company’s Phase 1 AQUAx clinical trial of AAV-hAQP1, an investigational gene therapy in development for the treatment of radiation-induced xerostomia. Of the three patients treated in Cohort 1, one patient has reached the 12-month assessment and two have passed the 6-month assessment. In these patients, AAV-hAQP1 has been well tolerated with no dose limiting toxicity and no serious adverse events reported. Encouraging responses have been seen in both patient-reported measures of xerostomia symptoms and in salivary output in the patients treated in Cohort 1, with complete resolution of symptoms in the patient who has reached the 12-month timepoint. Based on the encouraging safety and tolerability profile from Cohort 1, the Company is initiating plans for a Phase 2 efficacy and safety clinical trial for the treatment of patients with radiation-induced xerostomia.
The Phase 1 AQUAx clinical trial is an open-label, non-randomized, dose escalation trial designed to evaluate the safety of the Company’s investigational gene therapy AAV-hAQP1 when administered via Stensen’s duct to a single parotid gland in patients who have been diagnosed with grade 2 or 3 radiation-induced xerostomia and who have remained cancer free for at least five years (or at least two years if HPV+) after receiving radiation treatment for head and neck cancer. Primary endpoint of the trial is safety, with efficacy endpoints including the evaluation of the change in parotid gland salivary output after treatment with AAV-hAQP1 and patient reported measures of xerostomia symptoms.
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-hAQP1, plans to advance AAV-hAQP1 into Phase 2 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
