Staff Writer

PTC Therapeutics Inc. has completed a $50 million financing, the proceeds for which will be used to prepare for the anticipated launch of the drug in Duchenne's muscular dystrophy in the first half of 2011.

South Plainfield, N.J.-based PTC also plans to use some of the funds to study other possible indications for ataluren.

The pivotal trial of ataluren in Duchenne's muscular dystrophy has been completed and top-line pivotal data are expected in the first half 2010. If the data are positive, PTC could be on track to file a new drug application for ataluren in mid-2010 and potential accelerated approval in the first half of 2011.

In addition, PTC has an ongoing Phase III cystic fibrosis study of ataluren, with results likely to come in 2011.

The company also has a Phase II proof-of-concept study of ataluren in hemophilia that is expected to have results in 2010, and a Phase II proof-of-concept study is planned for a fourth indication that has not been disclosed.

Ataluren (PTC124) is aimed at a subset of patients with genetic disorders who have what is known as a nonsense mutation, in which the genetic code prematurely stops protein production, resulting in the loss of dystrophin protein in the case of Duchenne's muscular dystrophy and the CFTR protein in cystic fibrosis.

It is designed to ignore the stop signal, allowing production of a full-length protein to continue in patients with the nonsense mutation. Ataluren does not change the patient's genetic code nor does it introduce any genetic materials into the body, according to PTC Therapeutics.

Genetic testing, or genotyping, can determine whether a patient's disease is caused by a nonsense mutation. PTC Therapeutics has cataloged more than 2,400 genetic disorders where nonsense mutations are the cause of the disease in 15 percent of patients.

PTC plans to commercialize ataluren on its own in the U.S. and Canada and has partnered with Genzyme Corp. to commercialize the drug in Europe and the rest of the world.

The bulk of the funds from the financing will go toward the North American marketing effort, as well as for preparations for subsequent sales in the cystic fibrosis market, Stuart W. Peltz, president and CEO of PTC Therapeutics, told BioWorld Today.

The company now has a cash runway extending well into 2011. He said ataluren will be the first registered drug directed at the underlying cause of Duchenne's muscular dystrophy, and targeted at an "ultra-orphan market" that he said is well suited for the company.

PTC also will have enough funding to keep its other programs moving forward. Its oncology drug candidate (PTC299), which is designed to inhibit VEGF protein production, is in early Phase II testing in four different types of cancer, with results expected in 2010. The company also has a hepatitis C program partnered with Merck & Co. Inc. that is headed for Phase I testing next year.

The financing round was led by the Column Group, a new investor, and existing investor Delphi Ventures. The co-lead investors were joined by current investors Credit Suisse First Boston, HBM BioVentures, Novo A/S, Celgene and other existing and new investors. In connection with the Delphi investment, general partner Deepa Pakianathan, will join PTC's board of directors.

In other financing news,

• Labopharm Inc., of Laval, Quebec, has notified YA Global Master SPV Ltd. that it intends to draw down C$1 million (US$940,000) under a previously announced standby equity distribution agreement. Under the terms, the common shares to be issued to YA under the drawdown will be priced at a discount of up to 5 percent to the daily volume weighted average price over the 10 consecutive trading days following Labopharm's notice. The drawdown and share issuance is subject to a minimum price of $2 per share. Under the agreement, YA Global has committed to provide up to $25 million of capital during the next three years, at Labopharm's discretion, through the purchase of newly issued Labopharm shares. The maximum number of shares that the company can issue is limited to the lower of 11.4 million shares and 19.9 percent of the common shares issued and outstanding at any given time.

• Poniard Pharmaceuticals Inc., of South San Francisco, is raising about $6.5 million through the sale of 3.5 million common shares to Azimuth Opportunity Ltd. priced at about $1.87 each under its existing committed equity financing facility. The company intends to use proceeds to focus on regulatory and partnering activities for picoplatin development, other general corporate purposes and working capital. Based on cash reserves, including proceeds from this offering, Poniard believes it has sufficient cash to carry the firm to mid-2010. The company entered the $60 million CEFF with Azimuth in August, three months before picoplatin stumbled in a Phase III study of small-cell lung cancer. Shares of Poniard (NASDAQ:PARD) fell 9 cents Tuesday to close at $1.81. (See BioWorld Today, Aug. 21, 2009; and Nov. 17, 2009.)

• Titan Pharmaceuticals Inc., of South San Francisco, entered a $3 million loan and security agreement with Oxford Finance Corp. The loan carries an interest rate of about 13 percent per annum and is payable over three years. Titan will pay Oxford an initial facility fee of $60,000 and a final payment fee of $180,000. In addition, Titan recently received about $500,000 from the sale of 300,000 shares of common stock at $1.70 per share in a private placement to a single investor who participated in the company's December 2007 financing. The shares are currently restricted from trading, and Titan has agreed to register the shares for resale, possibly by April 15, 2010. The financing provides Titan with the immediate resources necessary to complete the process of becoming a reporting company again, and to keep the development of Probuphine on track, Titan said.