3P Biopharmaceuticals and Toleranzia Advance Partnership to Develop TOL2 for MG

3P Biopharmaceuticals and Toleranzia are continuing their partnership to develop Toleranzia’s potential therapy candidate, TOL2, for people living with myasthenia gravis (MG).

“Currently there are enormous opportunities offered by biotechnology as we can help to develop treatments for rare diseases or those without current therapeutic approaches, such as MG. Therefore, this pioneering project offers a bright hope for improving the lives of patients affected by the autoimmune neuromuscular disease Myasthenia Gravis,” Dámaso Molero, CEO at 3P Biopharmaceuticals, said in a press release.

MG is caused by the abnormal production of autoantibodies, which target the body’s own tissues, against protein receptors the at the neuromuscular junction — the site where nerve cells and muscles communicate. With fewer working receptors available, electrical impulses from nerves to muscle cells are impaired, and muscle function is affected. People with MG usually develop autoantibodies against anti-acetylcholine receptors (AChRs).

Current MG therapies include acetylcholinesterase inhibitors, immunosuppressive therapies such as azathioprine or cyclosporine, and thymectomy — surgical removal of the thymus, which is key for the production of antibodies. However, these treatments can cause severe side effects and have limited effectiveness, not targeting the root cause of the disease.

Sweden-based Toleranzia has been developing TOL2, a tolerogen therapy designed to promote immune tolerance and thereby prevent immune system attack. TOL2 uses protein components from the AChR.

In preclinical studies, TOL2 was found safe, without increased risk for side effects that affect the immune system, according to 3P Biopharmaceuticals, adding that available results also indicate the therapy may have a curative effect.

3P Pharmaceuticals and Toleranzia initiated their partnership in 2019 to test and advance the production of TOL2. Specifically, 3P Pharmaceuticals is responsible for the development of lab tests and analytic methods to manufacture TOL2. The goal is to achieve large-scale TOL2 production compliant with current good manufacturing practice (cGMP) by 2021, and then test the treatment in Phase 1 and 2 clinical trials.

Before moving to clinical trials, Toleranzia will complete toxicology and stability studies.

“We are delighted to collaborate with 3P Biopharmaceuticals for the scaling up and manufacturing of our important biological drug candidate TOL2. 3P was selected as the CDMO [Contract Development Manufacturing Organization] of choice based on their state-of-the-art infrastructure, work-force expertise and track-record,” said Bjorn Lowenadler, chief business officer at Toleranzia.

In March 2017, the U.S. Food and Drug Administration granted orphan drug designation to TOL2 for treating MG. This designation intends to encourage the development of therapies for rare and serious diseases, through benefits such as exemption from FDA fees and seven years of market exclusivity upon regulatory approval.