Biotech with Lysosomal storage disorder treatment raising $3.5 million, doubles staff
A biotechnology company co-founded by a pharmaceutical industry veteran is raising an additional $3 million as part of a Series A round to advance its lead therapeutic for Lysosomal storage disorders to the clinical trial stage. It has raised $1.6 million in equity since January, including funding from two new angel investors.
Since January, Callidus Biopharma has established proof of concept in vitro and has moved into animal trials using a specialized mouse model. It has nearly doubled its staff to 5, and hopes to file an Investigational New Drug application by the first half of 2013. Companies file INDs before they begin Phase 1 clinical trials.
The rare genetic disorders are caused by enzyme deficiencies that result in diseases such as Gaucher disease and Fabry disease. In many cases, these diseases are first diagnosed by underlying ailments such as cardiovascular problems. These genetic disorders would fit under the category of orphan diseases, an area that holds appeal for pharmaceutical companies because they can benefit from extended patent protection and tax breaks and generally higher prices.
Hung Do, the chief science officer of Callidus Biopharma, based at the Pennsylvania Biotechnology Center in Doylestown, Pennsylvania, told MedCity News that he believes the enzyme replacement therapies currently on the market are inefficient and result in only a small amount of the therapeutic drug being delivered to the intended tissues. Annual revenues for current drugs treating Lysosomal storage disorders are estimated at $600 million.
Do notes that as diagnostic techniques grow more sophisticated, other Lysosomal storage diseases are being more easily identified and could overtake Gaucher disease as the most common form of the disorder.
“We’re making decent progress,” said Do. “What we hope to do is to treat some of the symptoms of people with these disorders so their bodies can recover and they can live longer.”
In addition to its therapeutic, the company also has a platform technology to improve therapeutic protein production that Do says can reduce manufacturing costs.