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Orchard Therapeutics Plc (NASDAQ: ORTX) gains in premarket in reaction to positive interim data from a proof-of-concept study evaluating its gene therapy OTL-203 in Mucopolysaccharidosis type I (MPS-I). All eight patients treated with OTL-203 showed stable cognitive function, motor function, and growth within the normal range at multiple data points post-treatment.
OTL-203 treatment demonstrated rapid and sustained metabolic correction with all patients achieving supra-physiological IDUA expression in dried blood spot samples at 12 months (a primary efficacy endpoint).
FDA and European Medicines Agency (EMA) have accepted Orchard's request for parallel scientific advice to seek feedback in advance of initiating a registrational study for OTL-203 by this year-end.
The company also announced encouraging preliminary biomarker data for OTL-201 in three Mucopolysaccharidosis type IIIA (MPS-IIIA) patients, showing promising tolerability, engraftment, and biomarker data over the initial three-month follow-up period. Recently, a fourth patient was enrolled in the study. The enrollment could complete (n=5) this year, and additional interim data expected later this year.
IR webinar is scheduled today at 4:30 p.m. ET to review OTL-203 and OTL-201 data.
Last year in December, the company received its first product approval for Libmeldy in Europe for eligible patients with early-onset metachromatic leukodystrophy.
Price Action: ORTX jumped 10% at $8.31 in premarket trading on the last check Tuesday.
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