Orchard Therapeutics PLC (ORTX) stock prices were down by a marginal 2.44% as of the market closing on June 30th, 2021, bringing the price per share down to USD$4.39 at the end of the trading day. Subsequent pre-market fluctuations saw the stock surge by 8.66%, bringing it up to USD4.77.
Collaboration with Pharming
July 1st 2021 saw the company announce its strategic collaboration with Pharming Group N.V. to research, develop, manufacture, and commercialize OTL-105. The newly disclosed investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy is designed to treat hereditary angioedema (HAE), which is a rare disorder that causes recurring swelling attacks in the face, throat, extremities, and abdomen and can be fatal.
OTL-105
The investigational HSC gene therapy OTL-105 is designed to prevent hereditary angioedema attacks by increasing C1 esterase inhibitor (C1-INH). It inserts one or more functional replicas of the SERPING1 gene into the HSCs of patients ex vivo, which are then transplanted back into the patient for suitable durable C1-INH production. Preclinical studies to date have seen the treatment demonstrate high levels of SERPING1 gene expression via lentiviral-mediated transduction in multiple cell lines and primary human CD34+ HSCs.
Details of the Agreement
As per the terms of the agreement, Pharming has been granted worldwide rights to OTL-105 after having been also given the responsibility for its clinical development, regulatory filings, and commercialization of the investigational gene therapy, which will include covering associated costs. ORTX will oversee the completion of IND-enabling activities as well as the manufacturing of the treatment during pre-clinical and clinical development, with the funding being provided by Pharming. Furthermore, both companies will collaboratively explore the application of non-toxic conditioning regimen for use in tandem with OTL-105 administration.
Financial Details
The company will be given an upfront payment of USD$17.5 million, which will consist of USD$10 million in cash and USD$7.5 million equity investment from Pharming. The equity investment will include a premium to the company’s recent share price. ORTX is also eligible to receive up to USD$189.5 million in regulatory, development, and sales milestones, as well as royalty payments on future worldwide sales in the range of mid-single to low double-digits.
Future Outlook for ORTX
Armed with the recent collaboration that is likely to expand the scope of ORTX’s operations over the future, the company is poised to capitalize on the increased opportunities afforded to it. Investors are hopeful that company will be able to continue its trajectory of success and usher in unprecedented growth.
