Shares of Spruce Biosciences skyrocketed almost 1,400% late Monday after the Food and Drug Administration granted its rare-disease drug a breakthrough designation.
The biotech company is working on a treatment for Sanfilippo syndrome type B, a rare and genetic disorder that affects the brain and the nervous system.
Through breakthrough designation, the FDA provides a drugmaker extensive guidance to help streamline development. The agency also confirmed Spruce Bio can use a key biomarker in the cerebral spinal fluid to help determine the drug's clinical benefit and potentially win accelerated approval.
Following news of the FDA's determination, Spruce Biosciences stock catapulted 1,378.5% to close at 130.40.
Toxic Buildup In Spinal Fluid
Sanfilippo syndrome is characterized by the toxic buildup of heparan sulfate in the brain. This leads to progressive neurodegeneration causing cognitive and developmental impairment.
Spruce Biosciences bought its drug in April from bankrupt drugmaker Allievex. The drug replaces an enzyme that's missing in patients with Sanfilippo syndrome type B. The company estimates it affects fewer than one out of 200,000 people in the U.S. But it's hard to know the exact number given newborn screenings don't look for it.
In earlier testing, Spruce Bio's approach led to normal levels of heparan sulfate in the cerebral spinal fluid.
Spruce Bio shares hit a record high Monday after returning to trading on the Nasdaq less than a month ago.
Follow Allison Gatlin on X/Twitter at @AGatlin_IBD.
