Knopp Biosciences develops small‑molecule therapeutics for immunological and neurological disorders, with a clinical‑stage oral compound, dexpramipexole, in Phase 2 for moderate‑to‑severe eosinophilic asthma. The company also advances a preclinical Kv7 platform targeting developmental and epileptic encephalopathies, other rare epilepsies, neuropathic pain, and tinnitus. Knopp is directing capital toward completing its Phase 2 dexpramipexole asthma trial and initiating IND‑enabling studies and first‑in‑human trials of KB‑3061, a potential precision medicine for KCNQ2 epileptic encephalopathy. Knopp is privately held and has received NIH award support for its epilepsy and neuroscience research. Management expects topline Phase 2 dexpramipexole data in 1Q 2021 and planned human studies of KB‑3061 in 2021. The company is positioning its pipeline to address high unmet medical need across immunological and neurological indications. Knopp Biosciences is a Pittsburgh startup continuing clinical work on the small‑molecule dexpramipexole. The company is studying the compound, thought to maintain mitochondrial function, across a range of indications after it failed to show efficacy in Biogen Idec’s Phase 3 ALS trials. Knopp recently raised about $5 million in a fundraise from 18 investors. The NIH’s National Institute of Allergy and Infectious Diseases has given Knopp the go-ahead to research dexpramipexole and is working with the company on a clinical trial in hypereosinophilic syndrome (HES). Knopp previously raised $140 million in 2010 when dexpramipexole was still promising for ALS. The company is pursuing repurposing and additional clinical study rather than abandoning the compound. Knopp Biosciences is a biotechnology company developing dexpramipexole for amyotrophic lateral sclerosis (ALS) in collaboration with Biogen. Dexpramipexole is designed to protect stressed neurons and other cell types from dying, and Biogen holds commercialization rights. The drug has completed two Phase 2 studies and has an ongoing Phase 2 trial, with data expected in the fourth quarter this year or early 2013 per Biogen’s third-quarter earnings report. Knopp is also developing next-generation modulators of mitochondrial bioenergetics for Alzheimer’s disease, diabetes and heart failure. The company recently raised $14.9 million to accelerate discovery of its mitochondrial mediators and to support near-term clinical development of a combination treatment for Parkinson’s disease. If dexpramipexole meets agreed milestones, Knopp stands to receive an additional $255 million from Biogen.

Total raised

$34M

Funding rounds

3

Latest round

Series C

Latest activity

Nov 2020