TOL2 tolerated well in preclinical study

Preclinical toxicology report supports testing in humans, Toleranzia says

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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TOL2, a medication being developed by Toleranzia for the treatment of myasthenia gravis (MG), was found to be well tolerated in a preclinical toxicology study, supporting the company’s decision to test it in humans.

No side effects were observed when lab animals were given a dose of TOL2 into-the-vein (intravenously) for 10 days, a schedule that reflects the intended clinical dosing regimen.

“I am pleased to conclude that with these positive results we have met one of the most critical milestones in the TOL2 development program,” Charlotte Fribert, PhD, CEO of Toleranzia, said in a press release.

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With these preclinical results in hand, the company is planning to file an application seeking a green light to move TOL2 into first-in-human clinical testing in people with a diagnosis of MG.

“The data is fully in line with our expectations and allows us to move forward with the work to submit a clinical trial application and, following its approval by applicable authorities, to initiate our first clinical study in patients with myasthenia gravis,” Fribert said.

MG occurs when the immune system produces self-reactive antibodies that interfere with the way nerve and muscle cells communicate, leading to the condition’s hallmark symptoms of muscle weakness and fatigue. Most people with MG test positive for self-reactive antibodies against the acetylcholine receptor (AChR), a protein on the surface of muscle cells.

Jointly developed by Toleranzia and 3P Biopharmaceuticals, TOL2 is a tolerogen that contains pieces of the AChR protein. This is expected to restore tolerance to the body’s own AChR, preventing the autoimmune reaction that drives MG.

Study done by Charles River Laboratories

While it is still too early to understand whether TOL2 may ease MG symptoms, the preclinical toxicology study done by Toleranzia’s partner, Charles River Laboratories, suggests the medication may be safe and well tolerated during and after a dosing period.

Preclinical toxicology testing was conducted under the principles of good laboratory practice, a set of rules to ensure high-quality and reliable preclinical data.

In 2017, the U.S. Food and Drug Administration has granted TOL2 orphan drug designation for the treatment of MG. The designation provides a series of incentives, including reduced development costs and a potential for seven years of market exclusivity should TOL2 be approved.