An oral therapy designed to clear the lipid burden that overwhelms the lysosome in pediatric lysosomal storage diseases — leading with Niemann-Pick disease type C1 and Krabbe disease.
We are a team of drug developers and physician-scientists building an oral therapy to clear the toxic lipid buildup behind fatal pediatric lysosomal storage diseases.
Our leadership brings more than four decades of combined experience in clinical drug development. Our Chief Executive Officer spent fourteen years at Amgen and Biogen and has worked on more than 20 clinical and commercial programs. Our Chief Medical Officer is a physician-scientist who has led multiple IND-to-Phase 2 programs in neonatal and rare disease. Our Chief Financial Officer has led multiple public financings as a Nasdaq-listed company CFO.
Our lead program, UNDC-005, is an oral hydroxypropyl-alpha-cyclodextrin complexed with sodium caprate, designed to capture and clear the excess lipid that builds up when lysosomal transport fails. In an exploratory human assessment, approximately 50% of an oral dose was recovered in urine — a measure of the fraction reaching the circulation — against approximately 1–2% for the uncomplexed molecule. That human data underpins our intended 505(b)(2) regulatory pathway, which, if accepted by the FDA, would allow UNDC-005 to enter Phase 2b/3 without a standalone Phase 1 program.
We are advancing UNDC-005 first in Niemann-Pick disease type C1 — a rare, progressive, and fatal lysosomal storage disorder — with an IND targeted for the first half of 2027. Krabbe disease follows as our second lead indication, with five additional lysosomal storage diseases advancing as basket cohorts.
For children with a genetic lysosomal disorder, the course of the disease has been largely predetermined from birth.
Inherited at birth
NPC1 is caused by autosomal recessively inherited loss-of-function mutations in the NPC1 gene. The gene encodes a transmembrane transport protein in the lysosomal membrane that moves cholesterol and other lipids out of the lysosome. The defect is present from the beginning.
Diagnosed in childhood
Most patients are diagnosed as children. Because the disease is neurovisceral and takes a cognitive toll early, NPC1 is often described as pediatric dementia.
Progressive decline
Without a working transporter, lipid builds up inside the lysosome. The toxic buildup backs up the cell and stalls the internal transport and recycling systems that neurons depend on, and neurological function declines. Patients typically survive only into their late teens or early twenties.
The options today
Approved therapies are not directed at clearing the accumulated lysosomal lipid itself. Investigational cyclodextrin therapies in late-stage development require intravenous or intrathecal administration and carry a documented risk of permanent hearing loss.
UNDC-005 is being developed as an oral therapy that targets the lipid accumulation itself, using a molecule whose cavity is geometrically unable to bind cholesterol — the mechanism believed to cause hearing loss in the beta-cyclodextrin class.