Washington Editor

WASHINGTON - The federal government awarded $17.2 million to PTC Therapeutics Inc. for antibacterials against biowarfare or bioterrorism agents.

The company, of South Plainfield, N.J., plans to use the three-year contract to fund the discovery and development of broad-spectrum, small-molecule compounds through the initiation of Phase I trials. Its research focuses on altering post-transcriptional processes with small molecules to combat bacterial infections.

PTC's management could not discuss the grant because of an SEC-imposed quiet period. The company is aiming for an initial public offering, with a registration statement filed six months ago.

But its press release described in vitro tests that demonstrated its lead antibacterial compound is significantly active against several drug-resistant strains of bacteria, including methicillin-resistant and multidrug-resistant Staphylococcus aureus and vancomycin-resistant Enterococci. Those antibacterial effects have been achieved without toxicity in human cell lines, with the lead compounds exhibiting favorable pharmacokinetic properties.

The Defense Threat Reduction Agency made the award, the second government grant announced by PTC in recent days. A part of the Department of Defense, its Chemical and Biological Technologies Directorate provides science and technology support for the nation's defense through programs and initiatives to improve U.S. defensive capabilities against chemical and biological weapons and to protect the safety of U.S. forces operating in a contaminated environment.

Last week, PTC received a three-year, $2.2 million grant from the DoD to fund preclinical and clinical development of a compound called PTC299 as a potential treatment for breast cancer. Its lead product is PTC124, a drug in Phase II for cystic fibrosis and Duchenne's muscular dystrophy.

In addition to its government grants, the company has raised more than $130 million in private funding to date.

Biogeneric Bill Debuts In Congress

On Friday, Rep. Henry Waxman (D-Calif.) and Sens. Charles Schumer (D-N.Y.) and Hillary Rodham Clinton (D-N.Y.) introduced a biogeneric bill in their respective chambers.

Called the "Access to Life-Saving Medicine Act," the legislation would establish an abbreviated pathway for approval of generic biopharmaceuticals. There is no such statutory pathway at present, and Waxman recently told generic companies that he was crafting a legal fix. (See BioWorld Today, Sept. 20, 2006.)

Specifically, the measure would amend Section 351 of the Public Health Service Act for the approval abbreviated applications for biological products that are comparable to previously approved biologicals. It does not require that each comparable biologic be interchangeable, though.

Labeled H.R. 6257, it is written to set up a case-by-case review process that would require clinical testing only when necessary.

In its opposition to the bill, the Biotechnology Industry Organization (BIO) covered familiar ground in stressing that the FDA should continue to require a full complement of data, including clinical evidence, to demonstrate safety and effectiveness. The trade association also cautioned that a biogeneric approval pathway should not reduce or infringe the intellectual property rights of pioneer companies.

FDA Issues Production Guidances

Amid continued calls to modernize flu vaccine production, the FDA last week issued a draft guidance to aid manufacturers in developing cell-based viral vaccines, including those to address emerging and pandemic threats. In addition, the agency put out a final guidance to help manufacturers more generally maintain quality and improve efficiency.

The preliminary cell-based production guidelines relate to efforts around developing surge capacity for emergencies, something that chicken egg manufacturing couldn't address to combat a pandemic flu. It provides general advice on the selection, characterization and qualification of cell substrates and viral seeds used to manufacture those products. The public has 90 days to comment on the draft.

A newly issued and finalized guidance, called "Quality Systems Approaches to Pharmaceutical Current Good Manufacturing Practice (CGMP) Regulations," is designed to help manufacturers maintain quality and improve efficiency when implementing modern quality systems and risk management approaches to meet current CGMP requirements. It describes a comprehensive quality systems model, highlighting its consistency with the CGMP regulatory requirements for manufacturing human and veterinary drugs, including biological drug products. The guidance also explains how manufacturers implementing such quality systems can be in full compliance with parts 210 and 211 of those regulations.

Federal Funding For Avant Vaccine

Avant Immunotherapeutics Inc. is getting $2.6 million in government support for the continued development of an oral combination vaccine to protect against anthrax and plague.

Funded through the defense appropriations bill for the new government fiscal year - one of just two appropriations bills passed by Congress last week - the award will help fund the research and clinical development phase of the product, which is designed to quickly confer protection with a single oral dose. The company, of Needham, Mass., began the work in January 2003. It is being carried out at its research facility in Overland, Mo., and its vaccine production site in Fall River, Mass.

The program is administered by the DoD.

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