The Hepatitis C Virus (HCV) infects about 4 million Americans and 170 million people worldwide.  Untreated, it leads to chronic liver disease or cirrhosis and it is the leading cause of liver transplant in the U.S.   The HCV market is predicted to reach $20 billion by 2020; a leading reason that Gilead Sciences (NASDAQ: GILD) dished-out $11.1 billion to acquire Pharmasset and its HCV drug in development last November.  Also in the chase to grab a part of the market, Bristol-Myers Squibb (NYSE: BMY) spent $2.5 billion in January to acquire Inhibitex and its HCV asset, INX-189.   Some others in the space that offer solid upside potential, either stand-alone with their pipelines or as acquisition targets, include Idenix Pharma (NASDAQ: IDIX), Achillion Pharmaceuticals (NASDAQ: ACHN) and Aethlon Medical (OTCBB: AEMD).

Aethlon is particularly interesting, as it is not developing a drug like many of the traditional biotechs.  The revenue-stage company has developed the Hemopurifier®, a purification device able to selectively cleanse the blood of  a broad-spectrum of viruses, including Human Immunodeficiency Virus (HIV) and HCV.  On Tuesday, Aethlon released human treatment data resulting from the administration of Hemopurifier® therapy.  The results suggest that Aethlon may have an enduring opportunity in the HCV space.

In an ongoing study at Medanta Medicity Institute in India, the two most recent HCV-infected patients that have undergone treatment using Aethlon Medical Inc.’s Hemopurifier® therapy in combination with peginterferon and ribavirin (PR) have achieved an undetectable viral load at day seven. Aethlon’s Hemopurifier® is a first-in-class medical device that selectively targets the rapid clearance of HCV from the entire circulatory system to improve benefit, dose, duration and tolerability of drug therapies.

Hepatitis C Patients Cured

The goal of HCV therapy is a sustained virologic response, or SVR, (meaning that the undetectable viral load is maintained, i.e. no relapse) for 24 weeks after completion of a treatment regimen, whether it be device or drug based.  The best predictor of SVR is a rapid virologic response (RVR) which is defined as undetectable viral load 30 days after initiating therapy.

To date, Aethlon seems to be impacting RVR rates through the use of its Hemopurifier® as an adjunct to standard of care..  The landmark IDEAL Study, the largest prospective HCV study ever conducted with 3,070 HCV genotype-1 patients across 118 U.S. centers, provides a good point of reference as to the significance of Aethlon’s revolutionary therapy.

In short, the data collected in the IDEAL Study showed that only 10.35% (318 of the 3,070 patients) of PR-treated patients achieved a rapid virologic response (RVR).  RVR is defined as undetectable HCV RNA at day 30.  Of those 318 patients, 274 (86.2%) achieved a sustained virologic response.

The IDEAL study did not detail viral load at day seven, but it did at day 14.   The study revealed that just 131 of the 3,070 (4.3%) of patients achieved undetectable HCV RNA at day-14.  An amazing 91 percent, or 118 of those 131 patients, achieved a SVR. Based on this data, it is highly beneficial to achieve a RVR to greatly increase the likelihood of a patient being effectively cured of Hepatitis C.  Aethlon has reported that almost 67% of HCV genotype-1 patients treated to date have achieved an RVR.

“The intermittent administration of our Hemopurifier® during just the first days of interferon-based therapy is contributing to accelerate the pace and likelihood of viral load declining to undetectable levels,” said Aethlon Chairman and CEO, Jim Joyce.

More on the Study

In the study, HCV-infected individuals are enrolled to receive up to three, six-hour Hemopurifier® treatments during the first three days of PR drug therapy.  Aethlon reported that Hemopurifier® therapy has been well tolerated and without device-related adverse events in ten treated patients.  Of these ten patients, seven patients were infected with HCV genotype-1; two patients were infected with HCV genotype-3; and one patient was infected with HCV genotype-5.

To date, undetectable HCV RNA is reported in eight of the 10 treated patients.  Of the two patients with detectable HCV RNA, one discontinued PR therapy as a result of a diabetes related condition.  HCV RNA is undetectable in the four patients that have been monitored for 48 weeks since receiving Hemopurifier® therapy.

Among the 10 treated patients, Aethlon reported that six genotype-1 patients received the three treatment Hemopurifier® protocol, which resulted in four (67%) patients achieving a RVR.  Comparatively, the IDEAL study predicts it would normally require approximately 40 PR treated patients to achieve 4 RVR outcomes.  Both patients who achieved undetectable HCV RNA at day-7 also achieved a RVR. Beyond the high likelihood of a SVR, genotype-1 patients that achieve a RVR also have the opportunity to reduce the duration of PR drug therapy from 48 weeks to 24 weeks.

What’s Next?

As a result of Hemopurifier® + PR therapy outcomes, Aethlon is seeking to establish clinical sites that will provide non-Indian citizens with access to Hemopurifier® therapy.  A response from the Medicity internal review board (IRB) related to Aethlon’s request to expand clinical programs at the Medicity is currently pending. Aethlon also disclosed that it is preparing to resubmit an investigational device exemption (IDE) to the FDA, which will incorporate the Medicity data, in an effort to gain approval to initiate HCV clinical programs in the United States.

To date, approximately 100 Hemopurifier® treatments have been administered in human studies. Previously, studies of the Hemopurifier® have been conducted at the Apollo, Fortis, and Sigma New Life hospitals in India. These studies demonstrated that Hemopurifier® therapy could safely reduce viral load in both HIV and HCV-infected dialysis patients without the administration of antiviral drug therapies.  The Medicity study represents the first Hemopurifier® study in non-dialysis patients.  In vitro studies have further validated the ability of the Hemopurifier® to capture a broad-spectrum of viral pathogens classified as bioterror or pandemic threats.

The Big Picture

The unique mechanism of the Hemopurifier® really differentiates Aethlon from others in the biotechnology space, creating a lack of direct competition and a broad spectrum of uses for their technology.  Whereas others are vying for market capture with their infusion of drugs, Aethlon complements therapies as an adjunct by eliminating or subtracting the target disease from circulation without adding drug toxicity.   In this regard, the therapy provides a strategy to improve both Interferon-based drugs (such as that of Roche) and anti-viral drugs such as those being advanced by Gilead.  The ability to enhance therapeutic benefit to anti-viral drugs could widen Aethlon’s field of potential partners.

With yesterday’s news, investors may have gotten a better sense of the opportunity at hand with shares rising 32.35 percent yesterday with 585,300 shares changing hands.  Even with the rise and close at 9 cents, Aethlon is still only carrying a $12.6 million market cap, which leaves plenty of headroom for the company to grow long legs and establish a serious market presence.