
Our Approach
Riverside Pharmaceuticals utilizes publicly accessible databases to discover novel structure-activity relationships, which are then validated through in vitro and in vivo assays. Insights from this lead to new therapeutic approaches via drug repurposing or novel compound design.
Riverside is agile. While our projects typically begin with a disease focus, Riverside is not limited by this approach. Instead, we let newly discovered activities shape our programs, exploiting and maximizing the clinical potential of existing compounds.
Operating virtually, Riverside cost-effectively leverages best-in-class scientific expertise through tactical use of consultants, collaborators, and contract research organizations.

Our Pipeline
Riverside’s drug discovery and drug repurposing pipeline includes programs in:

Neuromuscular diseases (SMA, muscular dystrophies)
Riverside’s lead neuromuscular disease compound, RPL201, is an off-patent, previously clinical stage compound with an excellent tolerability profile in more than 200 human subjects. Preclinical and clinical evidence supports potential utility for several disorders, including spinal muscular atrophy (SMA) and various muscular dystrophies, including Duchenne’s. RPL201’s “off-target” activities enhance skeletal muscle functioning. Additional actions of RPL201 may also beneficially impact spinal motor neuron functioning in these disorders.

CNS disorders
Riverside is exploring early stage programs in neuropathic pain, epilepsies, schizophrenia, and other CNS disorders. Identified compounds consist of metabolites or proprietary analogs of drugs that are either marketed (and off-patent) or in development, and where Riverside has identified a chemical moiety which enhances the therapeutic activity and/or side effect profile of the precursor compound. These are early preclinical or discovery stage programs.

Asthma and COPD
Riverside’s RPL701 is a formerly clinical stage compound which shows promise for respiratory disorders, including asthma and COPD. RPL701 has an excellent safety profile in clinical trials, but was dropped from development by its original pharmaceutical sponsor due to lack of efficacy. Riverside is now repurposing RPL701 for respiratory diseases based on its action at a novel molecular target.
