Today Recursion announced an exciting new milestone: the in-licensing of a clinical-stage drug candidate for Neurofibromatosis type 2 (NF2) that was identified using our artificial intelligence platform [1]. This brings the company to a total of two clinical-stage programs after Recursion announced a cleared IND and initiated Phase 1 studies in Cerebral Cavernous Malformation in the past 6 months [2]. The story of REC-2282 is one that speaks to our approach of using petabytes of high-dimensional data we generate in-house and advanced machine learning to access unique insights into the biology of potential new medicines and prioritize results in ways that others cannot.

NF2: A Devastating Rare Disease

NF2 is a rare tumor syndrome and debilitating medical condition affecting thousands of patients worldwide, and many of these patients are in their teens, twenties and thirties. Patients with the disease suffer from the growth of benign tumors in the brain, spinal cord, and sometimes throughout the body, producing severe clinical symptoms including deafness, paralysis and severe pain. Untreated, these tumors can lead to early mortality, and without an FDA-approved medicine, surgery (and in some cases radiation) is the only option for most patients. However, surgical treatment of NF2 is not always effective because tumors are difficult to access within the cranial cavity, and are sometimes too numerous to remove. The right new medicine could significantly improve the lives of patients with the condition.

While no medical treatments are currently approved for NF2, over a dozen experimental medicines are in the drug development pipeline and are being tested in the disease. Many of these potential new medicines are drugs that have been developed for other indications but are currently being tested in NF2 [3], an approach called repurposing. Repurposing has been an attractive method for rare diseases because previously studied medicines can benefit from a shorter path to marketing and reduced development costs. In addition, the FDA has further facilitated drug development for rare diseases such as NF2 through provisions of the Orphan Drug Act [4].