The Stamford, CT-based biopharmaceutical firm says it employs a precision medicine approach to acquiring, developing and commercializing drugs for rare diseases and cancer. Lead candidate is small molecule nirogacestat for the potential treatment of desmoid tumors, noncancerous growths that occur in connective tissue, typically in the abdomen, arms and legs. Nirogacestat, in-licensed from Pfizer two years ago, inhibits an intramembrane enzyme called gamma secretase which has also been a target for Alzheimer’s treatments since it plays a key role in beta amyloid formation.
Candidate #2 is small molecule MEK inhibitor mirdametinib for the potential treatment of a rare peripheral nerve sheath tumor called neurofibromatosis type 1-associated plexiform neurofibroma.
2019 Financials (6 mo.): Operating Expenses: $26.5M (+289%); Net Loss: ($25.3M) (-283%); Cash Burn: ($20.3M) (-352%).
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