Why it is profiled?The company will be presenting key efficacy data from a phase IIb trial on April 14.
Opexa Therapeutics Inc. (OPXA) is a biotechnology company involved in the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis and diabetes.
The company's lead product candidate is Tovaxin, a novel personalized T-cell therapeutic vaccine for the treatment of multiple sclerosis. In patients with multiple sclerosis, the T-cells, which help to protect the body against infection, get activated and attack the myelin in the central nervous system. These activated T- cells are known as MRTCs (myelin-reactive T-cells).
Opexa produces Tovaxin by isolating MRTCs from a multiple sclerosis patient's blood. The MRTCs are multiplied to large quantities in a lab and then attenuated (weakened). The attenuated T-cells (Tovaxin vaccine) are then reintroduced into the patient via subcutaneous injection. Once the attenuated MRTCs are injected into the body, the immune system of the patient attacks not only those weakened MRTCs, but it attacks all of the pathogenic MRTCs that attack the myelin sheath of nerve fibers, thereby getting rid of the MRTCs.
In September 2008, Opexa announced top-line data from a phase IIb trial dubbed TERMS (Tovaxin for Early Relapsing Multiple Sclerosis).
The TERMS study enrolled 150 patients with relapsing-remitting multiple sclerosis or high risk Clinically Isolated Syndrome. In the study, 100 patients received Tovaxin and 50 received placebo. According to the study protocol, patients received a total of five subcutaneous injections at weeks 0, 4, 8, 12 and 24. The primary efficacy endpoint of the TERMS trial was the cumulative number of gadolinium-enhanced brain lesions using MRI scans summed over weeks 28, 36, 44 and 52.
Though the top-line results from the study demonstrated a positive trend in the reduction in ARR (annualized relapse rate) for patients treated with Tovaxin as compared to placebo, the results did not achieve statistical significance. In addition, the study did not achieve statistical significance with its primary endpoint, the cumulative number of gadolinium-enhanced brain lesions.
The study also showed Tovaxin to possess an impressive safety profile with no serious adverse events related to treatment. According to the company, the positive annualized relapse rate results combined with an excellent safety profile and convenient dosing place Tovaxin in a very favorable position for continued development as an innovative multiple sclerosis therapy.
Last September, the company revealed additional favorable data from the TERMS study. The additional analysis showed that 83% of patients treated with Tovaxin had remained relapse-free for a year and the annualized relapse rate after treatment decreased to 0.20, a 42% reduction compared to placebo.
The company will be presenting key efficacy data from the TERMS study on April 14 at the upcoming American Academy of Neurology 62nd Annual Meeting to be held in Toronto, Canada.
In the U.S.,about 400,000 people suffer from multiple sclerosis, and globally, there are approximately 2.5 million multiple sclerosis patients.
Some of the FDA-approved drugs to treat multiple sclerosis include Biogen Idec Inc.'s (BIIB) Avonex and Tysabri, Bayer/Schering AG's Betaseron, and Merck Serono's Rebif. The multiple sclerosis drug market, which was worth $9 billion in 2008, is estimated to reach as much as $16 billion by 2016.
Multiple sclerosis remains a challenging autoimmune disease to treat. There has been no cure for multiple sclerosis to date and hence therapies that are easy to use and can safely prevent or stop the progression of disease represent the greatest unmet need.
Opexa's first set of priorities is advancing the clinical development program of Tovaxin, which will continue in parallel with on-going partnering discussions and progressing discussions with the regulatory agencies.
Being a development stage company, Opexa has had no commercial revenues to date. From inception in January 2003 through December 2009, the company had incurred a net loss of $89.24 million.
In August 2009, Opexa sold its stem cell technology to Novartis AG (NVS) and received an upfront cash payment of $3 million and subsequently received $0.5 million as a technology transfer fee milestone. Opexa will receive an additional $0.5 million technology transfer fee this year. The company is also eligible to receive certain clinical and commercial milestone payments as well as royalty payments from the sale of any products resulting from the use of the technology and retains an option on certain manufacturing rights.
The total payments to Opexa, including the upfront payment, the technology transfer fee and development and commercial milestone payments could exceed $50 million, not including royalties.
To fund activities related to further clinical development of Tovaxin and for other working capital and operational purposes, Opexa raised $5.1 million in a registered direct offering of common shares and warrants last December. As of December 31, 2009, the company had cash and cash equivalents of about $8.2 million.
Opexa shares have thus far hit a 52-week low of $0.31 and 52-week high of $6.93. The stock gained 3.17% on Wednesday to close the day's trade at $2.60 on a volume of 917 thousand shares.
Other stocks "in the spotlight"
For comments and feedback contact: editorial@rttnews.com
May 01, 2026 15:54 ET Central banks dominated the economics news flow this week with almost all major ones announcing their latest policy decisions and many boosted expectations for a rate hike in June. In other news, several countries released the preliminary data for first quarter economic growth. In the U.S., comments by Fed Chair Jerome Powell were also in focus as his term ends this month.