Applied Genetic Technologies Corporation ( AGTC Quick Quote AGTC - Free Report) announced positive interim six-month data from the phase I/II study evaluating its adeno-associated virus ("AAV")-based gene therapy candidate in patients with X-linked retinitis pigmentosa ("XLRP"). Based on this promising data, the company is planning to initiate a pivotal study by the end of 2020. Data from the pivotal study will support regulatory submissions seeking approval for the gene therapy candidate.
The XLRP disorder is a rare, inherited eye disease causing progressive vision loss in boys and young men inclusive of night blindness in early childhood and progressive constriction of the visual field.
Shares of Applied Genetic skyrocketed more than 120% on Jan 9 following data read-out. The stock has surged 194.3% in the past year against the
industry’s decrease of 3.4%.
The phase I/II study is evaluating peripheral or central dosing of the AAV-based gene therapy candidate in XLRP patients. Interim data from the study showed that patients treated centrally demonstrated durable improvement in visual function after six months of dosing. Among the centrally-treated patients, 78% saw a five letter or more improvement.
Management stated that such progress is never reported in any other XLRP study. Data also showed that patients who achieved improvement in either visual sensitivity or Best Corrected Visual Acuity ("BCVA") also experienced greater clarity and reduced night blindness.
Earlier interim data from the study reported in September showed that the candidate boasts a favorable safety profile. Moreover, results demonstrated that peripherally-treated patients had stable visual function through six months.
Applied Genetic stated that sustained improvement in visual sensitivity noticed in centrally-treated patients is compelling. The company will enroll additional patients for the XLRP study in the first quarter of 2020. Data from this extra patient population is expected to generate most robust set of updates that might help the company design the pivotal study.
Apart from XLRP, the company is evaluating the candidate in patients with achromatopsia, an eye disorder leading to partial or total color blindness. The company will report interim six-month data from the escalation cohort of this study later this month. Positive data read-out can drive stocks further.
We note that gene therapies are increasingly becoming the latest treatment options with several big and small pharma/biotech companies developing gene therapy-based treatments for several diseases.
A popular name in the gene therapy space is Sarepta Therapeutics
SRPT, which is developing several gene therapy candidates for muscular dystrophies. Other promising gene therapy players include REGENXBIO Inc. RGNX, Solid Biosciences SLDB, Editas Medicine and uniQure. Zacks Rank
Applied Genetic currently carries a Zacks Rank #3 (Hold). You can see
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