Ankar Pharma, a Spanish-Filipino startup, is working to develop AP-1 (formerly VP3.15) its premier drug candidate for multiple sclerosis (MS). The pipeline drug is a first-in-class small molecule with a dual mechanism of action and reported high activity in preclinical models. It currently remains in preclinical testing.
History of AP-1
Preclinical studies have shown the compound’s potential efficacy in treating inflammatory diseases, induced artificially in murine models. AP-1 was reported to decrease MS clinical symptoms (according to neurological scores) in an experimental allergic encephalitis (EAE) model, an accepted model worldwide for multiple sclerosis studies, working both as a chronic preventive treatment and at the peak of the disease. According to the company, AF-1’s efficacy in EAE models was similar or better to that produced by fingolimod, the first oral drug approved for relapsing-remitting MS treatment. Preclinical results for AP-1 have been published in scientific journals, most recently in Journal of Chemical Neuroanatomy.
How AP-1 Works
The exact mode of action of AP-1 is not currently understood. However, in in-vitro and ex-vivo studies, it was concluded that the drug has a dual mechanism of action, blocking two crucial enzymes (GSK-3 and PDE7) responsible for the activation of chronic inflammatory pathways. Inflammation caused by the immune system is one of the primary causes of damage to the myelin sheath in MS. By inhibiting these enzymes, AP-1 aims to protect the nervous system by reducing inflammation. It also is thought to promote the growth and maturation of oligodendrocytes, and to induce the remyelination of damaged nerve fibers.
Next Steps for AP-1
According to its website, Ankar Pharma is actively looking for partners to bring the compound into clinical trials in healthy volunteers and MS patients.
Given the drug’s positive effects in early studies on the nervous system and its demonstrated ability to penetrate to the brain, the company is also considering AP-1 as a potential treatment of ophthalmic diseases. The optic nerves are among the first to be affected by demyelinization in MS.
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