In 2014, BELLUS Health entered into a development and license agreement with AMO Pharma Limited (AMO Pharma) for the worldwide rights to AMO-01 (formerly TLN-4601) for the treatment of neurologic and psychiatric disorders in return for royalties on sales and revenue sharing.

AMO Pharma announced in May 2018 the initiation of patient recruitment in an interventional (Phase 2) study of AMO-01 in the treatment of Phelan-McDermid syndrome, a rare genetic condition caused by a chromosomal deletion.

In pre-clinical studies, AMO-01, an inhibitor of the Ras-ERK pathway, has been shown to rescue the neuronal phenotype of the multiple knockout mouse models of intellectual disability. This provides strong scientific evidence to support the conclusion that inhibition of the Ras-ERK cascade may have therapeutic benefit in the treatment of intellectual disabilities in humans.

AMO Pharma is a private company developing new treatments for serious and debilitating diseases including rare genetic disorders.

Share this

Sign up for email alerts

Email Address *
Enter the code shown above.