8:48 AM
 | 
Apr 12, 2018
 |  BC Extra  |  Company News

Orchard gains GSK's rare disease gene therapy portfolio

Editor's Note: This article was updated on Apr 12, 2018 at 4:38 PM PDT

GlaxoSmithKline plc (LSE:GSK; NYSE:GSK) said it will divest its rare disease gene therapy portfolio, including Strimvelis, to Orchard Therapeutics Ltd. (London, U.K.).

GSK will be eligible to receive undisclosed commercial milestones and royalties. The pharma will hold a 19.9% stake in Orchard and will receive a seat on its board.

In addition to Strimvelis, an ex vivo gene therapy to treat a form of severe combined immunodeficiency (SCID), Orchard will gain two programs in registrational trials, OTL-200 (GSK2696274) to treat metachromatic leukodystrophy (MLD) and OTL-103 (GSK2696275) to treat Wiskott-Aldrich syndrome, as well as an undisclosed clinical program to treat beta-thalassemia. It also will acquire options to exclusively license from Fondazione Telethon and Ospedale San Raffaele three preclinical programs in mucopolysaccharidosis I (MPS I, Hurler syndrome), chronic granulomatous disease and globoid cell leukodystrophy, following clinical proof-of-concept trials.

Orchard said the assets fit its pipeline of autologous ex vivo lentiviral gene therapies to treat primary immune deficiencies and inherited metabolic disorders (see BioCentury, April 21, 2017).

GSK has been pruning its pipeline since last July, when it announced plans to discontinue more than 30 programs and reevaluate its rare diseases unit. In its 2Q17 earnings, the pharma said it would direct 80% of its R&D investments toward "priority assets" to treat respiratory disorders, infectious diseases including HIV, oncology and immuno-inflammation (see BioCentury Extra, July 26, 2017).

According to GSK’s website, the deal leaves the pharma with a pipeline of programs across those four disease areas plus vaccines. Its pipeline also includes its NY-ESO SPEAR T cell therapy to treat several tumor types. GSK gained exclusive rights to the therapy after exercising a 2014 option from Adaptimmune Therapeutics plc (NASDAQ:ADAP). The therapy comprises autologous T cells genetically modified to express T cell receptors (TCR) targeting cancer/testis antigen 1B (NY-ESO-1; CTAG1B) (see BioCentury Extra, Sept. 7, 2017).

A GSK spokesperson told BioCentury on Thursday that the pharma is "investing heavily" in its cell and gene therapy platform with an emphasis in cancer.

Strimvelis, a retroviral vector delivering adenosine deaminase (ADA) for ex vivo hematopoietic stem cell therapy, is approved in Europe to treat children with severe combined immunodeficiency due to ADA deficiency (ADA-SCID). It is the only asset in the acquired portfolio that is not an autologous ex vivo lentiviral gene therapy.

Orchard plans to submit a BLA to FDA this year for its ADA-SCID candidate OTL-101, Orchard President and CEO Mark Rothera said. OTL-101, which is in a registrational trial, comprises autologous CD34+ hematopoietic stem cells transduced ex vivo with an EFS lentiviral vector delivering the ADA gene (see BioCentury Extra, Dec. 20, 2017).

Orchard anticipates submitting regulatory applications for the MLD program next year and the WAS program in 2020 or 2021, according to Rothera. The biotech said initial submissions will be to EMA.

"We really have a suite of launches to look forward to," Rothera said.

Rothera said Orchard will have access to certain GSK commercial resources this year as the biotech builds up its own commercial infrastructure.

In December, Orchard raised a $110 million series B round led by Baillie Gifford and ORI Capital, which the company said at the time would provide runway into 2019, including the launch of OTL-101. Temasek, Cowen Healthcare Investments, Juda Capital, Pavilion Capital, RTW Investments, Agent Capital, 4BIO Capital, F-Prime Capital and UCL Technology Fund participated in the series B.

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