Proteostasis Therapeutics Raises $45 Million to Develop Therapies for Protein Homeostasis Disorders

(Nanowerk News) Newly formed Cambridge, Mass.-based Proteostasis Therapeutics Inc. has raised $45 million in a Series A financing to develop therapies, which the firm has dubbed proteostasis regulators, to treat multiple genetic and degenerative disorders associated with protein homeostasis deficiencies, such as Alzheimer's disease, emphysema, Type II diabetes and Huntington's disease.
The complexities of protein homeostasis, referred to as proteostasis, only began to really be appreciated in the past 18 to 24 months, said CEO David Pendergast.
"This area has exploded," he declared.
Proteostasis enables the development of healthy cells and protects against disease. Deficiencies in proteostasis may lead to metabolic, oncological, neurodegenerative and cardiovascular disorders.
Proteostasis Therapeutics is focused on developing small molecules that ameliorate disease by manipulating proteostasis to repair rather than replace damaged proteins. There are about 500 to 600 key proteins arranged in about a dozen pathways, which Pendergast called the proteostasis network, that maintains the health of the other 22,000 proteins of the proteome, he explained.
"The whole idea is to be able to induce with small molecules this proteostasis network as a response to disease rather than the classical approaches of blocking pathways to try to stop a pathology," Pendergast told BioWorld Today.
He said the firm is targeting two broad areas of disease: aggregation diseases, those where proteins aggregate and lose the ability to respond to challenges over time, such as in Alzheimer's and Parkinson's diseases, and genetic diseases, those where a protein has misfolded, as in the case of cystic fibrosis.
Proteostasis Therapeutics has demonstrated in animal models that the proteostasis network can be manipulated or controlled with small molecules to bring it back to an effective level of dealing with a challenge to ameliorate disease and stop symptoms from further developing, Pendergast said.
In genetic diseases, he added, small molecules can be used to stimulate the network to fix the defect.
Pendergast said his firm expects to be in the clinic in about three to five years. However, currently the firm has one employee: Pendergast. The company plans to soon hire about 20 people, including a chief scientific officer and senior technical staff. The firm also is actively seeking a facility.
Proteostasis Therapeutics, which was founded in late 2006 with $1 million seed money from HealthCare Ventures, has operated thus far as a virtual firm, Pendergast said.
Once Proteostasis hires its key personnel and finds a building, "we will spend the first 18 to 24 months expanding our understanding of the network and its relationship to our selected disease areas as well as evolving our chemical series as potential treatments in those areas," he said.
Considering the median Series A financings for biotechs in the first half of this year was $17.5 million, with the maximum at $32 million, Proteostasis' $45 million funding is significant. (See BioWorld Today, Aug. 25, 2008.)
"Obviously, we believe that this is a big idea, and an important biology with significant therapeutic applications. Obviously, so did the other investors," Pendergast said. The firm is expecting the funds to take it through to clinical trials, he added.
Pendergast noted that the company also plans to partner with large pharma firms and disease associations to augment its funds.
"We didn't want to have the stops along the way where you have to stop and take six months off to raise an additional round, and others agreed that that was a good idea," he said.
Investors in the round included HealthCare Ventures, Fidelity Biosciences, New Enterprise Associates, Novartis Option Fund and Genzyme Ventures.
Source: Proteostasis Therapeutics (press release)
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