Ampligen update

Source: Philadelphia Business Journal

Date:   March 30, 2007

Author: John George <jgeorge@bizjournals.com> 

Read article on Philadelphia Business Journal website



Hemispherx finalizing fatigue drug filing

Hemispherx BioPharma Inc. is finalizing its new drug application for Ampligen, an experimental treatment for chronic fatigue syndrome that has spent more than 30 years in clinical studies. Dr. William Carter, the Center City biotechnology company’s chairman and CEO, said he expects the application will be completed in early April.

Carter said he is "cautiously optimistic" the company will be able to secure an accelerated review from the Food and Drug Administration, given the absence of any approved treatments for chronic fatigue syndrome. The Centers for Disease Control and Prevention estimates the disorder afflicts 1 million people in the United States. Accelerated reviews are typically conducted in six months, as opposed to the one-year review for most new drug applications.

If the company gets such status for its Ampligen filing, and the FDA approves the application without raising any issues, Hemispherx would be in a position to launch the product by the end of this year or early 2008, Carter said.

Carter expressed a sense of vindication that his company has reached this point. "It’s been a long and arduous journey… There were a lot of skeptics," he said.

The skepticism remains evident in the company’s stock price. Hemispherx’s stock was trading recently at $1.69 a share, near its 52-week low of $1.65.

According to documents filed earlier this month with the Securities and Exchange Commission, Hemispherx has recorded an accumulated deficit of about $167 million since its inception. Hemispherx posted a net loss of $19.4 million last year, up from a deficit of $12.4 million in 2005. The increase was due primarily to higher research and development costs

associated with Ampligen and Alferon N, which the company acquired in 2003, is a treatment for genital warts and Hemispherx’s only FDA-approved product in the market. The company is testing both Ampligen and Alferon N as potential treatments for viral infections such as avian flu.

Carter co-invented Ampligen in the early 1970s when he was a researcher at Johns Hopkins University. Ampligen is a synthetic, specifically configured double-stranded RNA compound designed to work by stimulating and enhancing the ability of a patient’s immune system to fight disease. Initially, the drug was tested as a potential treatment for cancer and later for AIDS then for chronic fatigue syndrome. Carter even tinkered with the idea of using the compound to treat tobacco and create a "healthy" cigarette.

Hemispherx has spent the past 15 years testing the drug as a treatment for patients with chronic fatigue syndrome. The company estimates the global market for an effective treatment for the disorder to be in excess of $1 billion.

"We’re not the first company to go into this disease category," Carter said.

In a presentation with stock analysts last month, the company noted drugs developed by a half-dozen major pharmaceutical and biotechnology companies – including GlaxoSmithKline, Shire and Cephalon – have been considered

as potential treatments for chronic fatigue syndrome, but said none proved to be effective.

Jenifer Antonacci, a Cephalon spokeswoman, said the company’s flagship drug Provigil, approved as a treatment for narcolepsy and other sleep-related disorders, was tested in patients with chronic fatigue syndrome about two years ago. She said the study was conducted by researchers from outside of the company, with Cephalon support. The clinical trial, which involved 14 patients, did not produce sufficient results for Frazer-based Cephalon to consider seeking approval to expand Provigil’s label to include the disorder.

Matt Cabrey, a Shire spokesman, said that while company executives have discussed the potential of testing its products for chronic fatigue syndrome, Shire never launched any formal product development program and has no current plans to do so. Based in England, Shire’s U.S. headquarters are in Wayne.

Up Close

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COMPANY:                       Hemispherx BioPharma Inc.

LOCATION:                      Philadelphia

CHAIRMAN AND CEO:        Dr. William Carter

TYPE OF COMPANY:           Biopharmaceutical

2006 NET LOSS:               $19.4 million

2006 REVENUE:                $933,000

52-WEEK HIGH/LOW:         $3.85, $1.65

RECENT PRICE:                  $1.69

MARKET CAPITALIZATION:     $122.6 million

EMPLOYEES:                     52 full time, 19 part time

BIG DEVELOPMENT:       Finalizing new drug application for Ampligen, an

                                 experimental treatment for chronic fatigue syndrome.

Source:                        SEC, company, Yahoo! Finance

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(c) 2007 Philadelphia Business Journal