Opexa Therapeutics Reaches Clinical Development Milestone Event in Development of a Personalized Therapy for Secondary Progressive Multiple Sclerosis
Opexa Therapeutics, Inc., a Woodlands-based biotechnology company that is developing Tcelna (imilecleucel-T), a personalized T-cell based immunotherapy for the treatment of multiple sclerosis, reported in May that it had reached the targeted enrollment in its phase IIb clinical trial in Secondary Progressive Multiple Sclerosis.
One hundred ninety patients have been enrolled in the randomized clinical trial, termed the “Abili-T” trial. Opexa was founded in 2001 by BCM Technologies, the college’s venture development subsidiary, and an exclusive license agreement conferring rights to a group of technologies developed at BCM targeting the diagnosis and treatment of autoimmune disorders was executed in September 2001. The company has been devoted to shepherding the clinical development of Tcelna and has expended considerable resources to commercially scale and refine the process for manufacturing this personalized immunotherapy.
Tcelna offers promise to patients with Secondary Progressive Multiple Sclerosis, a stage of MS for which there are high unmet medical needs and few treatment options. The Abili-T trial is a multicenter phase IIb study, involving 35 clinical sites in the U.S. and Canada. Patients enrolled in the trial will receive two annual courses of the Tcelna treatment, which consists of five subcutaneous injections per year. The trial’s primary efficacy outcome is the percentage of brain volume change (atrophy) in 24 months. Opexa expects to have top line data from the trial in mid-2016. The company has received Fast Track designation from the FDA for Tcelna in Secondary Progressive MS, which stands to expedite the review process. Opexa has a pharmaceutical partner, Merck Serono, with which it signed an option/ license agreement in 2013. Merck Serono can exercise its option prior to or upon completion of the Abili-T trial, and if it does elect to exercise then Opexa will stand to receive milestone payments and royalties.
The Opexa story provides a great example of the level of sustained focus and commitment that a company must undertake when it initiates development of a therapeutic technology licensed from an academic institution, particularly a personalized immunotherapy that is custom-made for each patient. At the time the company was founded, the process for manufacturing such a therapy in the academic setting was not sufficiently scalable or reproducible to be successfully deployed in the commercial sector—much additional development was needed. The Opexa team has been hard at work for well over a decade to improve, scale, refine, and standardize the process for making this personalized immunotherapy available to patients in distant locations.
BLG Director Michael Dilling stated, “I have nothing but admiration for the dedication of the Opexa team in their quest to gain regulatory approval for Tcelna to make it widely available to a patient population with high unmet medical needs. We’re very pleased to see them achieve this key clinical milestone. Developing any new therapeutic is challenging, but developing a personalized immunotherapy has its own unique set of challenges that the company has successfully addressed. The Opexa story provides a perfect illustration of the purpose behind technology licensing—to get promising discoveries into the commercial sector where they can be developed and help patients.”