|BioCryst results fail to impress|
|By Patrick Crutcher|
|Wednesday, 15 September 2010 23:18|
For BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX), this could have been a better week.Today, they reported top-line results from their Phase II study of oral forodesine in the treatment of relapsed or refractory cutaneous T-cell lymphoma(CTCL), which only showed an 11% response rate. On top of that, SVNT got approval of KRYSTEXXA for the treatment of gout, an indication which BCRX is currently developing BCX-4208 for. They also showed early Phase 2 data from their exploratory Phase 2 study to investigating oral forodesine as monotherapy for chronic lymphocytic leukemia (CLL). Fortunately, they could be presenting material information on Thursday, which could help restore some confidence.
Unfortunately for investors and BCRX, the results in CTCL failed to impress and might not be enough to support approval. Their trial was conducted under a SPA with the FDA (2007). The lack of any patients achieving a complete response certainly doesn’t help their case either; however the high-rate of stable disease might help their odds. The data are as follows:
Eleven of 101(11%) later stage patients enrolled achieved a partial cutaneous response, while no patients achieved a complete response. Of the remaining later stage patients, 56 (55%) had stable disease as their best response, 30 (30%) had progressive disease, with a median time to progression of 353 days, and four (4%) were not evaluable.
Their patients had been treated with a median of 4 other therapies before the trial, but their cancer did not respond to treatment or recurred later. Final results from the study are expected later in 2010.
Additionally, they posted interim results in CLL, which may have prevented a big dip because of the promising nature in this early Phase 2 trial. Here the primary endpoint is overall response rate. The interim analysis showed that 3 patients demonstrated a confirmed partial response to forodesine and 6 patients remain enrolled in the study and on treatment, of which 5 had been treated with forodesine twice-daily for longer than one year. They also stated they would present final results later this year at an upcoming medical meeting.
Luckily, BCRX’s sole focus is not oncology, so they were spared a serious beating. BCRX main focus has been on the development on peramivir. In 2006, it received Fast-Track designation for peramivir. It has been approved for use in Japan, South Korea, and Mexico. In early 2010, they partnered with Merck Serono for sales in Europe, Russia, Canada and Singapore and Hikma Pharmaceuticals PLC for the Middle East and North Africa (MENA) region, excluding Israel. They have 2 ongoing Phase 3 trials to support FDA approval for influenza. The promise of peramivir has certainly brought BCRX a lot of attention. They were awarded an additional $77M in Sept. 2009, on top of their initial $109M/4-year contract by the U.S. Department of Health & Human Services (HHS) to complete Phase 3 development of peramivir to treat seasonal and life-threatening influenza in 2007. Back in late 2009, the FDA has issued an emergency use authorization (EUA) for the investigational intravenous (i.v.) peramivir in certain adult and pediatric patients with confirmed or suspected 2009 H1N1 influenza infection who are admitted to a hospital.
On Wednesday, BCRX presented data related to intravenous (i.v.) peramivir for the treatment of influenza at the 50th Annual Interscience Conference on Antimicrobial Agents & Chemotherapy (ICAAC) in Boston, Massachusetts. On Thursday, BRCX is hosting and webcasting their Investor Day in New York City at 9AM EST. It is expected they will give an update on the influenza season and the use of their products overseas.
Investors should know that BCRX just recently updated their clinical trial page for BCX-4208 as being “completed” for their first Phase 2 study in gout. In late April, they posted promising top-line results from their Phase 2 study in gout and have recently begun a second trial using BCX-4208 in gout.
BCRX is cash rich and has no debt hanging over them. Institutional holdings make up a large portion of their shareholders. You can find their August 2010 Q2 presentation here. At these levels, I won’t be buying unless something materially changes. Be sure to check out their webcast on Thursday, which can be accessed here.