About ALS Biopharma

We are an emerging biotechnology company dedicated to the discovery of small-molecule therapeutics and diagnostics for the treatment of the debilitating neurological orphan indication of amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease). We incorporate modern aspects of drug discovery including compound library synthesis and screening. In partnership with top academic and industrial laboratories, ALS Biopharma, LLC is researching both small molecule and biologic protein-based approaches to treat the disease.


ALS Biopharma
3805 Old Easton Road
Doylestown, PA 18902
(215) 589-6416

Location and Facilities

We are affiliated with the Pennsylvania Drug Discovery Institute (www.padrugdiscovery.org) at the Pennsylvania Biotechnology Center of Bucks County in Doylestown, PA (www.pabiotechbc.org).

Our laboratories at the PBC include a complete array of chemistry and biochemistry tools and equipment such as a real time PCR, laminar flow hoods, table top centrifuges, Thermofinnagan LCQ and ABI Qtrap Mass Specs, Waters HPLC systems, a Biomek robot liquid handler, and other equipment that is routine for modern molecular biology, high throughput screening, and medicinal chemistry.

New Press Release

May 25, 2017: ALS Biopharma, LLC is pleased to announce that the U.S. Food and Drug Administration (FDA) has provided fast-track designation to trigriluzole for the treatment of ataxia. Trigriluzole, also known as FC-4157 and BHV-4157, is an investigational new drug being developed by Biohaven Pharmaceutical Holding Company Ltd. See the entire press release here.

July 5, 2016: ALS Biopharma, LLC is pleased to announce that the U.S. Food and Drug Administration (FDA) has completed its review of the FC-4157 (also known as BHV-4157) investigational new drug application (IND) filed on May 31, 2016 by Biohaven Pharmaceutical Holding Company Ltd. See the entire press release here.

August 18, 2015: ALS Biopharma is pleased to announce a new research collaboration with Biohaven for the development of certain prodrugs, as described in this press release. The full text of the press release can also be seen here.

Recent Publications

Coyne, A. N.; Yamada, S. B.; Siddegowda, B. B.; Estes, P. S.; Zaepfel, B. L.; Johannesmeyer, J. S.; Lockwood, D. B.; Pham, L. T.; Hart, M. P.; Cassel, J. A.; Freibaum, B.; Boehringer, A. V.; Taylor, J. P.; Reitz, A. B.; Gitler, A. D.; Zarnescu, D. C.; Fragile X protein mitigates TDP-43 toxicity by remodeling RNA granules and restoring translation. Hum. Mol. Genet. 2015, Sep 18. pii: ddv389.

Cassel, J. A.; Ilyin, S.; McDonnell, M. E.; Reitz, A. B. Novel Inhibitors of Heat Shock Protein Hsp70-Mediated Luciferase Refolding that Bind to DnaJ, Bioorg. Med. Chem. Lett. 2012, 20, 3609-3614.

Cassel, J. A.; McDonnell, M. E.; Velvadapu, V.; Andrianov, V.; Reitz, A. B. The effects of small molecule inhibitors of nucleic acid binding to TDP-43 on TDP-43 metabolism and function. Biochimie, 2012, 94, 1974-1981.

Cassel, J. A.; McDonnell, M. E.; Velvadapu, V.; Andrianov, V.; Reitz, A. B. Characterization of a series of 4-aminoquinolines that stimulate caspase-7 mediated cleavage of TDP-43 and inhibit its function. Biochemie, 2012, 94, 1974-1981.

Cassel, J. A.; Ilyin, S.; McDonnell, M. E.; Reitz, A. B. Novel Inhibitors of Heat Shock Protein Hsp70-Mediated Luciferase Refolding that Bind to DnaJ., Bioorg. Med. Chem. Lett. 2012, 20, 3609-3614.

Pawlyk, A. C.; Cassel, J. A.; Reitz, A. B. Current Nervous System Related Drug Targets for the Treatment of Amyotrophic Lateral Sclerosis. Curr. Pharm. Des. 2010, 16, 2053.

Cassel, J. A.; Blass, B. E.; Reitz, A. B.; Pawlyk, A. C. Development of a Nonradiometric Assay for Nucleic Acid Binding to TDP-43 Suitable for High-Throughput Screening Using AlphaScreen® Technology. J. Biomolecular Screening, 2010, 15, 1099-1106.