SI
SI
discoversearch

We've detected that you're using an ad content blocking browser plug-in or feature. Ads provide a critical source of revenue to the continued operation of Silicon Investor.  We ask that you disable ad blocking while on Silicon Investor in the best interests of our community.  If you are not using an ad blocker but are still receiving this message, make sure your browser's tracking protection is set to the 'standard' level.
Biotech / Medical : biotech fireworks -- Ignore unavailable to you. Want to Upgrade?


To: JMarcus who wrote (472)12/4/2002 8:56:25 PM
From: JMarcus  Read Replies (1) | Respond to of 7424
 
I just confirmed from the ASH website that Avigen's Mark A. Kay will be giving an oral presentation on the Coagulin B Phase I/II trial results at 10:45 AM ET on December 9th. Here's the link (but you'll need to get a user ID and password first).

hematology.org

Marc



To: JMarcus who wrote (472)12/7/2002 4:24:57 PM
From: Doc Bones  Respond to of 7424
 
Gene Therapy Could Soon Get a Needed Boost [Barrons]

This pump in Barrons will probably give AVGN a boost at the open on Monday. - Doc

By BILL ALPERT
December 9th, 2002

Gene therapy has suffered many setbacks, as doctors have vainly tried to rewrite the faulty genetic instructions that cause inborn diseases like cystic fibrosis. Investors are betting that Monday will bring good news when doctors, at a meeting of the American Society of Hematology, report on a genetic fix for the bleeding disorder hemophilia. Doctors from Philadelphia's Children's Hospital and Stanford University have snuck corrective genes into patients using a tamed virus made by Avigen, a biotech firm whose shares have jumped to $9.50 from about $5 in the past month.

Research abstracts released in advance of the meeting describe long-lasting improvement in hemophilic dogs that got the Avigen treatment. Two patients getting a low-dose treatment showed no improvement in clotting, but the abstracts were written before the outcome was known for patients that got the higher dose that worked in lab animals.

Avigen, and doctors at the Children's Hospital of Philadelphia, told me they couldn't talk before Monday's announcements. But a full calendar of scheduled press conferences next week suggests that outcomes were good for the higher-dose gene therapy. Avigen's Coagulin-B treatment addresses a portion of hemophilia sufferers who lack a clotting protein called Factor IX. The more common form of hemophilia results from lack of Factor VIII, and afflicts up to 50,000 men in the developed world. Avigen's Factor VIII treatment is still in the lab.

Gene therapy needs some good news. In 1999, a teenager died from his immune system's reaction to an attempted gene treatment. Last year, successful treatments of the congenital immune disorder popularly known as "bubble-boy" disease got derailed when the treatment apparently triggered a cancer-like disorder in one boy.

Avigen needs good news, too. After trading near 90 two years ago, shares of the Alameda, Calif., firm dropped nearly to 5 this year -- below the firm's $7 a share in book value, and even below its $6.22 a share in balance-sheet cash. The firm lost $21 million, or $1.06 a share, in the nine months ended Sept. 30, but it still had $125 million in cash. In October, Avigen laid off a quarter of its staff, to trim its cash-flow burn rate to $25 million a year.

Along with hemophilia, Avigen is working on genetic fixes for Parkinson's Disease and heart failure. CIBC's Matt Geller likes the shares. "We've been looking at gene therapy for a long time and waiting for that moment when there's a breakthrough," says Geller. "This could be that breakthrough."

Geller has no earnings in his Avigen model, and in truth, it's hard to predict earnings when nobody has figured out how genetic therapy will be priced. Hemophilia patients use over $1 billion a year in drugs, for instance. If one dose of Avigen's Coagulin avoided a lifetime of drug expenditures, what would that be worth?

<snip>

online.wsj.com