Orchard Therapeutics: PRIME Designation in Europe and an IPO Registration

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Orchard Therapeutics, with locations in Boston and London, UK, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for OTL-300, a lentiviral gene therapy for transfusion-dependent beta-thalassemia (TDBT).

PRIME designation provides “enhanced interaction with the EMA to both optimize development and accelerate evaluations of applications,” the company states. The designation for OTL-300 is based on preclinical and early clinical data.

TDBT is a rare inherited blood disorder caused by one of more than 200 mutations in the HBB gene. The disease results in failure to grow and gain weight, risk of infection, and life-threatening anemia, all of which occur in the first two years of life. The only cure is allogeneic hematopoietic stem cell transplantation, which is dangerous with a high risk of death. Most patients with the disease receive chronic blood transfusions and often develop serious side effects, such as iron overload.

Orchard acquired OTL-300 from GlaxoSmithKline in April 2018. The therapy came out of a collaboration deal between GSK and the Hospital San Raffaele and the Telethon Foundation, initiated in 2010.

“We are encouraged by the early clinical trial data in TDBT patients, which was started in 2015 in hopes of extending the hematopoietic stem cell gene therapy approach that has been so successful in such conditions as adenosine deaminase severe combined immunodeficiency (ADA-SCIT), Wiskott-Aldrich syndrome (WAS) and Metachromatic Leukodystrophy (MLD) to other diseases with unmet medical needs such as TDBT,” stated Giuliana Ferrari, leader of the thalassemia gene therapy project at the San Raffaele-Telethon Institute for Gene Therapy. “Hematopoietic stem cell gene therapy could offer a promising alternative to the current standards of care for adults and children living with TDBT.”

Part of the data collected include that from an ongoing proof-of-concept clinical trial of nine patients. Five of seven patients showed significant cuts in blood transfusion frequency and volume requirements with a least one year’s follow-up. Three of the four pediatric patients were transfusion-free after about one month of treatment, and in two out of three adult patients, reductions in transfusion volume requirements were observed. One of the adults was transfusion-free over a nine-month period.

The company also filed yesterday with the U.S. Securities and Exchange Commission (SEC) for an initial public offering (IPO). The company hopes to raise up to $173 million with the IPO. It plans to list on the Nasdaq under the ORTX symbol.

On August 13, Orchard closed on a Series C financing worth $150 million. The round was led by Deerfield Management with new investments from RA Capital Management, Venrock, Foresite Capital, Perceptive Advisors, Cormorant Asset Management, ArrowMark Partners, Sphera Global Healthcare, Medison Ventures, Driehaus Capital Management and Ghost Tree Capital Group, in addition to U.S.-based healthcare funds. Existing investors also participated, including Temasek, Baillie Gifford, RTW Investments, Cowen Healthcare Investments and Agent Capital.

The funds will be used to advance the company’s three most advanced clinical programs. They are OTL-101 for adenosine deaminase severe combined immunodeficiency (ADA-SCID), OTL-200 for metachromatic leukodystrophy (MLD) and OTL-103 for Wiskott-Aldrich syndrome. It will also be used for its rare disease gene therapy pipeline.

“This financing provides Orchard with additional capital to rapidly progress our most advanced clinical programs to commercialization,” said Frank Thomas, Orchard’s chief financial officer and chief business officer, in a statement. “We are advancing our pipeline of potentially transformative gene therapies in primary immune deficiencies and neurometabolic disorders to reach patients as quickly as possible.”

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