Flexion Therapeutics’ FX201, a Gene Therapy Candidate for the Treatment of Osteoarthritis, Receives IND Clearance by FDA Triggering Milestone Payment from Flexion to GeneQuine Biotherapeutics

Flexion’s IND application for FX201, which has the potential to provide long-term symptomatic relief and modify disease progression in knee osteoarthritis, was cleared by the FDA triggering a milestone payment from Flexion to GeneQuine. A Phase 1 single ascending dose study with FX201 has been initiated by Flexion and the first patient is expected to be treated by the end of 2019. GeneQuine believes that the FX201 IND approval will facilitate the transition of GeneQuine’s independent lead program GQ-303 from preclinical to clinical stage expected in 2021, as GQ-303 is also a gene therapy candidate for the treatment of osteoarthritis based on the same gene therapy vector technology.

In 2017, Flexion Therapeutics (Nasdaq: FLXN) acquired the osteoarthritis gene therapy product candidate FX201 from GeneQuine Biotherapeutics for up to $64 million. Following successful completion of a GLP toxicology program and confirmation of efficacy in a rat osteoarthritis model, Flexion has now received clearance of the IND application for FX201 by the FDA, which triggers a milestone payment from Flexion to GeneQuine. Flexion has also initiated a Phase 1 multicenter, open-label, single ascending dose study and expects to treat the first patient by the end of 2019. The clinical trial is intended to evaluate safety and tolerability in knee osteoarthritis patients and may give initial insight into its clinical activity, including effects on pain, function and structural progression.

GeneQuine’s preclinical-stage lead candidate GQ-303 is a helper-dependent adenoviral vector (HDAd), which GeneQuine is developing independently for treatment of osteoarthritis. GQ-303 contains the gene for proteoglycan 4 (also known as lubricin) and has demonstrated symptomatic and disease-modifying efficacy in several rodent osteoarthritis models. As FX201 and GQ-303 are of the same type of gene therapy vector (HDAd) and are both intended to be administered by intraarticular injection for the treatment of osteoarthritis, GeneQuine believes that Flexion’s successful preclinical development of FX201 and approval of the IND significantly de-risks development of GeneQuine’s candidate GQ-303 towards the clinic.

Clearance of the FX201 IND by Flexion is also an important step in the general pursuit to apply gene therapy to prevalent, non-monogenic disorders such as osteoarthritis. (Read more …)

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