Rare Daily Staff

Inotek Pharmaceuticals, following the mid-stage clinical failure of its lead therapy, said it has entered into a definitive merger agreement with gene-therapy developer Rocket Pharmaceuticals to focus on gene therapies to treat rare diseases.

Inotek had been focused on the development and commercialization of therapies for ocular diseases. In July, the company announced top-line results of its mid-stage, fixed-dose combination trial of trabodenoson and latanoprost for the treatment of glaucoma. The trial failed to meet its primary endpoint and the company discontinued development of the therapy to evaluate strategic alternatives.

Under the terms of the merger, which is subject to shareholder approval, shareholders of Rocket will receive newly issued Inotek common shares in a private placement. Rocket shareholders are expected to own approximately 81 percent of the combined company and current Inotek shareholders will own approximately 19 percent of the combined company. The percentage of the combined company that Rocket’s shareholders will own as of the close of the transaction is subject to adjustment based on the amount of Inotek’s net cash at the closing date.

The newly-merged company will retain the Rocket Pharmaceuticals name and will be headquartered in New York City. Gaurav Shah, who was formerly a CART-19 Global Program Head in the Cell and Gene Therapies Unit at Novartis, will serve as CEO of the merged company.

Rocket’s focus for its lentiviral gene therapies is bone marrow disorders caused by gene mutations. Lead programs include Fanconi Anemia, Leukocyte Adhesion Deficiency-1, and Pyruvate Kinase Deficiency. Current treatment options available for patients with these diseases are limited and include allogeneic bone marrow transplant procedures, which are often complicated by graft versus host disease and a lack of available donors. Rocket’s gene therapy approach could be potentially curative and replace or pre-empt the need for transplant.

Longer term, Rocket is also developing a lentiviral-based gene therapy for infantile malignant osteopetrosis, an inherited bone disorder.

In addition, Rocket is advancing an AAV-based program for an undisclosed rare pediatric disease with a significant estimated patient population size of more than 15,000 in the United States and European Union. Rocket expects to enter the clinic in 2018, with clinical proof of concept data from one or more of the lentiviral programs in 2018.

“Our vision is to create a fully-integrated platform gene therapy company with a portfolio of distinct treatments for devastating genetic diseases,” said Shah. “FA, LAD-1 and PKD are near term opportunities in rare bone marrow-derived disorders. The AAV-based approach, while earlier, will target treatment of a broader range of challenging diseases.”

September 13, 2017

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