Orchard Therapeutics Announces First Patient Dosed with Cryopreserved Formulation of OTL-103 Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome
Program on Track for MAA and BLA Regulatory Submissions in 2021
Recent Lancet Hematology Publication Describes Encouraging Safety and Efficacy Data from Interim Analysis of OTL-103 Fresh Formulation Registrational Trial
BOSTON and LONDON,
“We are excited to have reached this milestone with the infusion of cryopreserved gene-modified stem cells in a patient with Wiskott-Aldrich Syndrome, a life-threatening immune disorder for which there is no available therapy outside of allogeneic stem cell transplantation, which carries a significant risk of morbidity and mortality,” said
While the registrational trial for OTL-103 for WAS used a fresh cell formulation, Orchard plans to commercialize this and other HSC gene therapies, if approved, using a cryopreserved formulation. In total, nearly 40 patients have been treated with a cryopreserved product across the company’s portfolio. The WAS cryopreserved formulation trial will enroll up to six patients, with the number of patients with successful engraftment measured at six months as the primary endpoint. This patient data will be used to supplement the company’s in vitro CMC comparability work between the fresh and cryopreserved formulation. Please refer to www.clinicaltrials.gov (NCT03837483) for additional clinical trial information.
Fresh Formulation Interim Results Recently Published in Lancet Hematology
An interim analysis (data cutoff
“Patients with WAS suffer from severe bleeding episodes, such as intracranial bleeds or severe gut bleeds, which can be fatal. Without treatment, the median survival for WAS patients is 14 years of age,” said Alessandro Aiuti, professor of Pediatrics at the
“We are encouraged by recently published data demonstrating that the fresh formulation of OTL-103 can reduce the frequency and severity of both infections and severe bleeding episodes in WAS patients,” said
About WAS and OTL-103
WAS is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age and treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in
Orchard’s portfolio of autologous, ex vivo, hematopoietic stem cell gene therapies includes Strimvelis, a gammaretroviral vector based gene therapy and the first such treatment approved by the
Orchard currently has offices in the U.K. and the U.S., including
This press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “anticipates,” and “future” or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, planned marketing and licensing application submissions and next steps for Orchard’s programs, including the therapeutic potential of its product candidates, including OTL-103. These statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchard’s product candidates, including OTL-103, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchard’s ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchard’s product candidates, and the risk of delays in Orchard’s ability to commercialize its product candidates, if approved. Orchard undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. For additional disclosure regarding these and other risks faced by Orchard, see the disclosure contained in Orchard’s public filings with the Securities and Exchange Commission.
Source: Orchard Therapeutics Limited