FoldRx Pharmaceuticals Closes $43 Million Series B Financing; Funds Will Advance Clinical Development of the Lead Compound, Fx-1006A, and the Company's Pre-Clinical Pipeline.

CAMBRIDGE, Mass. -- FoldRx Pharmaceuticals, Inc., a development-stage biotechnology company focused on protein misfolding diseases, announced today that it has successfully raised $43 million in a Series B financing. The funds will be used for the continued clinical development of FoldRx's lead clinical candidate, Fx-1006A, and to advance its pipeline, which is initially focused on hereditary amyloidosis and neurodegenerative diseases, including Parkinson disease. Texas Pacific Group (TPG) Ventures and Alta Partners led the Series B round, which also included Novartis BioVenture Fund, as well as original investors, HealthCare Ventures and Fidelity Biosciences.

"Therapeutics aimed at protein misfolding and aggregation are increasingly recognized areas of extraordinary potential for treating the underlying cause of a number of intractable diseases," said Geoffrey Duyk M.D., Ph.D., Managing Director of TPG Ventures. "FoldRx is rapidly building a leading position in this field and has made significant progress in its clinical program and with its proprietary platform for translating genetic information into promising clinical targets and leads."

FoldRx is nearing completion of a Phase I trial for its lead drug candidate, Fx-1006A, which the company is developing for Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FAC). The company expects to begin a clinical efficacy trial in FAP patients later this year. Fx-1006A is a first-in-class, disease-modifying, small-molecule that is designed to inhibit the formation of amyloid deposits by binding and stabilizing the transthyretin protein (TTR), precluding misfolding and subsequent deposition of TTR. Both FAP and FAC result from specific mutations in the TTR gene.

"This additional funding represents a strong vote of confidence in our approach to protein misfolding diseases and in the potential of our lead drug candidate," commented Richard Labaudiniere, Ph.D., President and Chief Executive Officer of FoldRx. "We will apply the capital to support our expanding clinical development efforts, especially the initiation of a pivotal efficacy trial in FAP patients with our lead drug candidate, Fx-1006A, later this year."

About Protein Misfolding and Aggregation Diseases

Protein misfolding is increasingly being recognized as an underlying cause in many chronic degenerative diseases. It has been theorized that protein-protein interactions or small-molecule modulators may be used to block the misfolding process or its deleterious consequences. Many of these protein-misfolding diseases are chronic, progressive and fatal, including familial amyloidoses, Parkinson disease, cystic fibrosis, Alzheimer disease and Huntington disease.

TTR, whose deposition leads to FAP and FAC, is a protein that is produced in the liver and circulates in the blood. All disease-associated mutations characterized to date have been shown to destabilize TTR, resulting in misfolding and misassembly including amyloid deposits in various tissues. In FAP, deposition of TTR amyloid occurs in the peripheral nerve tissue and results in sensory neuropathy, starting in the lower extremities and progressing to include autonomic and motor dysfunction. Liver transplantation is currently the only treatment option available for these patients. In FAC, TTR amyloid deposits infiltrate the heart, leading to congestive heart failure. The predominant mutation in FAC is present in more than three percent of African Americans. There are currently no treatments available for FAC. It is believed that stabilization of transthyretin will inhibit further amyloid deposition and stop progression of the disease in FAP and FAC patients.

About FoldRx Pharmaceuticals, Inc.

FoldRx Pharmaceuticals is a development-stage biotechnology company focusing on first-in-class disease-modifying small molecule therapeutics to treat diseases of protein misfolding and aggregation (amyloidosis). Protein misfolding is increasingly being recognized as an underlying cause of many chronic degenerative diseases. By applying FoldRx's proprietary expertise in protein folding and its platform for drug and target discovery, the company is building a pipeline, initially for hereditary amyloidosis and neurodegenerative diseases. FoldRx's initial pipeline includes a program in clinical development to treat two genetic diseases, Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FAC), and a discovery program in Parkinson disease, based on its broad, proprietary yeast-based drug discovery platform. FoldRx was cofounded by Jeffery W. Kelly and Susan Lindquist at Scripps and the Whitehead Institute, respectively. For more information on FoldRx, please visit the company's web site at www.foldrx.com.