GTP-004 will be named DAS-MG from now on, DAS Therapeutics said on its website.
The investigational treatment combines a fixed dose of pyridostigmine — an approved therapy for myasthenia gravis-associated muscle weakness — with an antagonist that prevents the gastrointestinal side effects linked to pyridostigmine use.
Pyridostigmine, sold under the brand name Mestinon, was first approved by the U.S. Food and Drug Administration in 1955. Since then, generic formulations of pyridostigmine have been released in the market.
Myasthenia gravis is characterized by the blockage of acetylcholine receptors that mediate the communication between nerve and muscle cells. This blockage is triggered by the overproduction of antibodies by the host’s immune cells against its own tissues.
An acetylcholine esterase inhibitor, pyridostigmine works by preventing the destruction of the signaling molecule acetylcholine. This inhibitory mechanism ensures that more acetylcholine is available to counteract the blockage of acetylcholine receptors and increase effective nerve-muscle cell communications.
Pyridostigmine has demonstrated efficacy in managing myasthenia gravis symptoms. However, its use has been associated with severe gastrointestinal adverse effects that may lead patients to not tolerate the treatment.
The new combo therapy DAS-MG is designed to resolve this problem, by providing patients an effective treatment for their symptoms while at the same time resolving the cause of treatment discontinuation.
DAS-MG was successfully evaluated in a Phase 1 study in healthy volunteers, where it was shown to be safe and well-tolerated. Supported by the positive trial results, DAS Therapeutics is planning to soon initiate a Phase 2 clinical trial in patients with myasthenia gravis.
“We are pleased that DAS Therapeutics will continue to advance [GTP-004] in a Phase 2 clinical trial and toward product registration,” Anthony Cataldo, chairman and CEO of GT Biopharma, said in a press release.
Under the terms of the purchase agreement, DAS Therapeutics now has full rights to develop and commercialize DAS-MG. The company now owns all clinical data, composition-of-matter and use patents related to the investigational therapy.
GT Biopharma will receive $200,000 as an up-front payment, annual royalties from sales, and a $6 million payment based on achievement of commercial milestone.