Flexion Therapeutics to Advance Investigational Gene Therapy FX201 into High Dose Cohort of Phase 1 Clinical Trial in Knee Osteoarthritis and Expand Low and Mid Dose Treatment Groups

Rhea-AI Summary

Flexion Therapeutics (Nasdaq:FLXN) announced the progression of its FX201 gene therapy into the high dose cohort of a Phase 1 trial after a positive review by an independent Data Monitoring Committee. The trial, targeting osteoarthritis treatment, will expand to include up to 20 additional patients in both low and mid dose cohorts. Initial data indicates FX201 is generally safe and well-tolerated, with early signs of clinical activity. Interim results are expected to be presented at a scientific meeting in 2021, with longer-term data anticipated by year-end.

Positive

• Positive review from Data Monitoring Committee supports advancement of FX201 into the high dose cohort.
• Initial data shows FX201 well-tolerated, with no serious adverse events reported.
• Expansion to include 20 additional patients in both low and mid dose treatment groups to gather more data.

Negative

• None.

• First-in-human single ascending dose trial advances to high dose cohort following positive Data Monitoring Committee review of mid dose safety data
  
• Company to expand FX201 low dose and mid dose cohorts to include up to 20 additional patients in each treatment group
• Flexion plans to present interim data from the single ascending dose phase at a scientific meeting in the first half of 2021
  

BURLINGTON, Mass., Feb. 18, 2021 (GLOBE NEWSWIRE) -- Flexion Therapeutics, Inc. (Nasdaq:FLXN) today announced plans to advance FX201 into the high dose cohort of the Phase 1 dose-escalation trial. The trial is evaluating the safety and tolerability of FX201, an investigational, intra-articular, IL-1Ra gene therapy product candidate being developed for the treatment of osteoarthritis (OA). The decision follows an independent Data Monitoring Committee (DMC) review of the mid dose safety data. In addition, the company has expanded the trial to include up to 20 additional patients in both the low and mid dose treatment groups.

"The data from the single ascending dose phase of this study indicate that the low and mid doses of FX201 appear to be well tolerated; furthermore, while early and from a small sample of patients, we are seeing some positive signals of clinical activity in even the lowest dose cohort,” said Michael Clayman, MD, President and Chief Executive Officer of Flexion Therapeutics. “These encouraging results support our decision to expand the trial to further inform our understanding of dose response, evaluate the biological activity of FX201 locally in the knee joint, and shape the design of a potential Phase 2 proof-of-concept trial in patients with knee OA."

Clinical data from the first two cohorts indicate that FX201 appears to be generally safe and well-tolerated at the low and mid doses. There were no serious adverse events and there was no evidence of systemic biodistribution in plasma or shedding in urine observed in any patient. Furthermore, the company believes treatment responses observed through 24 weeks following administration of the lowest dose of FX201 support its potential to provide durable pain relief to patients with knee OA. Longer-term data to assess safety and clinical activity in both the low and mid dose cohorts are still being collected, and the company plans to present these interim data at a scientific meeting in the first half of 2021.   

The open-label, expansion phase of the study is expected to enroll up to an additional 20 patients, in both the low and mid dose treatment groups. Each patient will receive one injection of FX201 into the knee joint and will be followed for 104 weeks. The DMC will continue to review safety data of all enrolled patients in all dose levels.

Data through Week 52 for patients treated in the initial low and mid dose cohorts of the single ascending dose phase are expected by the end of 2021. In addition, preliminary data from the high dose cohort and expanded treatment groups are also anticipated before year-end.

About FX201
FX201 is an investigational gene therapy which utilizes a helper-dependent adenovirus (HDAd) vector devoid of all viral genes that carries a coding sequence for an anti-inflammatory protein called interleukin-1 receptor antagonist (IL-1Ra) under the control of an inflammation-responsive promoter. FX201 is injected directly into the joint space (also termed the intra-articular space) and is intended to deliver as-needed anti-inflammatory activity to joint tissues over the long-term, with the goal to improve outcomes for OA patients.

About Flexion Therapeutics
Flexion Therapeutics (Nasdaq:FLXN) is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis, the most common form arthritis. The Company's core values are focus, ingenuity, tenacity, transparency and fun. Please visit flexiontherapeutics.com.

Forward-Looking Statements
This release contains forward-looking statements that are based on the current expectations and beliefs of Flexion. Statements in this press release regarding matters that are not historical facts, including, but not limited to, statements relating to the future of Flexion; timing and plans with respect to the Phase 1 clinical trial of FX201; and the potential therapeutic and other benefits of FX201, are forward looking statements. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation, the fact that the impacts and expected duration of the COVID-19 pandemic are uncertain and rapidly changing; the risk that we may not be able to maintain and enforce our intellectual property, including intellectual property related to FX201; risks related to clinical trials, including potential delays, safety issues or negative results; and other risks and uncertainties described in our filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 filed with the SEC on November 4, 2020 and subsequent filings with the SEC. The forward-looking statements in this press release speak only as of the date of this press release, and we undertake no obligation to update or revise any of the statements. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

Contact:

Scott Young
Vice President, Corporate Communications & Investor Relations
T: 781-305-7194
syoung@flexiontherapeutics.com

Julie Downs
Associate Director, Corporate Communications & Investor Relations
T: 781-305-7137
jdowns@flexiontherapeutics.com 

FAQ

What is the purpose of the FX201 clinical trial?

The FX201 trial aims to evaluate the safety and tolerability of this gene therapy for treating osteoarthritis.

What were the results of the initial dose cohorts for FX201?

Initial results show FX201 is generally safe and well-tolerated, with no serious adverse events and some early positive clinical activity signals.

When will interim data from the FX201 trial be presented?

Interim data from the single ascending dose phase is expected to be presented at a scientific meeting in the first half of 2021.

How many patients will be included in the expanded FX201 trial cohorts?

The expanded trial will include up to 20 additional patients in both the low and mid dose treatment groups.

What is the expected timeline for data reporting from the FX201 trial?

Data through Week 52 for initial patients is expected by the end of 2021, along with preliminary data from the high dose cohort.