DJ Orchard Therapeutics Sees Positive Results from Stem-Cell Gene Therapies
By Chris Wack
Orchard Therapeutics PLC said it saw positive results from new data from several of its hematopoietic stem cell gene therapies in development for neurodegenerative disorders, including multiple clinical outcomes for OTL-203 in MPS-I.
The company on Tuesday said patients with the Hurler subtype of MPS-I suffer from a range of symptoms, including neurocognitive impairment and skeletal deformities that lead to impaired growth in the first decade of life.
Eight patients with MPS-IH have been treated with OTL-203 in a proof-of-concept study being conducted at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. As of November, follow-up in all patients has reached at least 12 months. Orchard said a request for parallel scientific advice from the U.S. Food and Drug Administration and European Medicines Agency has been accepted with the intention of seeking feedback in advance of initiating a registrational study for OTL-203 by year-end 2021.
Treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen, the company said. Anti-alpha-L-iduronidase antibodies present prior to gene therapy as a result of ERT weren't seen in any patient within two months following treatment.
Orchard said 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. Longitudinal growth that was within age-appropriate reference ranges was seen in all patients post-treatment, with follow-up ranging from 9 months to 2 years. Improved range of motion was shown in all patients compared to pre-treatment, with follow-up ranging from 9 months to 1.5 years, it said.
Orchard Therapeutics shares were up 12% to $8.11 in premarket trading.
Write to Chris Wack at chris.wack@wsj.com
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February 09, 2021 07:27 ET (12:27 GMT)
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