PTC Therapeutics Inc. raised $26.6 million in a private placement to support clinical development of PTC124, its drug to treat genetic disorders due to nonsense mutations.

The drug is expected to enter Phase II trials this quarter in Duchenne's muscular dystrophy and cystic fibrosis, said Mark Boulding, senior vice president of business development and legal for South Plainfield, N.J.-based PTC.

"Hopefully, we'll have results from those in the next quarter," he said.

Funds also will go toward advancing the company's preclinical oncology and antiviral programs, as well as supporting further drug discovery work across multiple therapeutic areas.

To date, PTC has raised more than $130 million. Boulding told BioWorld Today the recent financing, PTC's fourth institutional round, should sustain the company into the first part of 2007.

In addition to its private placements, the company also has brought in money through several grants. Just this year, the company received $1.5 million from the Muscular Dystrophy Association and $1.7 million from Cystic Fibrosis Foundation Therapeutics Inc. to help fund development of PTC124.

An orally administered, small-molecule drug, PTC124 is designed to suppress nonsense mutations - single-point alterations in the genetic code that prematurely halt the translation process and cause shortened, non-functional proteins.

"The mutation makes you lose the functionality of a protein," Boulding said, "and it's that lack of a protein that causes the disease."

In Duchenne's muscular dystrophy (DMD), the nonsense mutation can be found in the dystrophin gene, and those mutations make up about 15 percent of the DMD cases.

About 10 percent of the cystic fibrosis cases are caused by a nonsense mutation of the CF transmembrane regulator gene. PTC124 is designed to allow the cellular machinery to bypass the mutation to restore full-length proteins.

The drug has been granted orphan status for both indications.

In addition to PTC124, the company also has PTC299, a preclinical anti-angiogenic compound expected to enter the clinic next year. That drug likely will be tested against solid tumor cancers. PTC also expects to select compounds next year to begin testing in its infectious disease program.

The financing was led by Credit Suisse First Boston Private Equity and HBM BioVentures, of Baar, Switzerland. It included new and existing investors Vulcan Ventures, of Seattle; Novo A/S, of Bagsvaerd, Denmark; Delphi Ventures, of Menlo Park, Calif.; Bay City Capital, of San Francisco; Novartis BioVentures, of Basel, Switzerland; Amgen Ventures, of Thousand Oaks, Calif.; and HealthCap, of Stockholm, Sweden.