BioCryst
Pharmaceuticals, Inc. (Nasdaq: BCRX) today provided an update regarding the
company's development programs for Fodosine(TM).
Phase IIb Trial in T-ALL Voluntarily Placed on Hold by BioCryst Due to
Stability Issue with the Intravenous Formulation of Fodosine(TM)
BioCryst is voluntarily placing its Phase IIb clinical trial of
intravenous (I.V.) Fodosine(TM) in the treatment of patients with T-cell
acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) on hold. The oral
capsules being used in other clinical trials of Fodosine(TM), including the
CTCL trial, are not affected and those trials continue.
Recent stability results detected particulate matter in clinical
batches of the intravenous formulation of Fodosine(TM). Preliminary
findings have suggested that the particulates are associated with the vial
stoppers used in clinical packaging and may result from an interaction
between a component of the stopper and a component of the drug solution.
Assay data do not suggest any decrease in the potency of the
Fodosine(TM) active drug product. The proportion of study drug vials
affected has not been exactly ascertained; therefore BioCryst determined
that it was appropriate to suspend all patient treatment with any
intravenous formulation. BioCryst has informed the Food and Drug
Administration (FDA) that an internal investigation is ongoing and that
patient treatment has been halted. BioCryst and Mundipharma, its partner in
the development of Fodosine(TM), are working together to identify the best
path forward for this formulation.
"We are working closely with our partner, Mundipharma to collect and
analyze all of the relevant data. In the meantime, we are putting
enrollment of new patients on hold until we have a clearer understanding of
the situation," said Jon P. Stonehouse, Chief Executive Officer of
BioCryst. "This issue is limited to the intravenous formulation of
Fodosine(TM) and does not affect the oral formulations currently being used
in our other studies of Fodosine(TM). We continue to be enthusiastic about
Fodosine(TM) and anticipate initiating a Phase IIb study in CTCL later this
year."
Completed Phase IIa Trial of Fodosine(TM) in Patients with T-ALL Shows
Lower Response Rate
In the now-completed Phase IIa trial of Fodosine(TM) in the treatment
of T-ALL, patients continued to enroll after data were last presented at
the Annual Meeting of the American Society of Hematology (ASH) in December
2006. A preliminary analysis of the data now available suggests the
response rates may be lower than previously reported at the ASH meeting.
"As the study progressed toward the target number of 80 subjects
treated, the response rate appears to have decreased from the 18% reported
at ASH in December, 2006. While the company considers these results
preliminary, we don't think the final response rate will be as high as
18%," said W. James Alexander, M.D., M.P.H., Senior Vice President,
Clinical and Regulatory Operations and Chief Medical Officer of BioCryst.
"We are still in the process of collecting all the data for analysis."
Company Will Focus Efforts on Fodosine(TM) in Treatment of CTCL
Mr. Stonehouse commented, "As an emerging biotechnology company,
BioCryst is focused on bringing compelling products to market as quickly as
possible for the benefit of patients and shareholders. Among the primary
reasons BioCryst chose to pursue treatment of T-ALL as the lead indication
in the Fodosine(TM) program were the fact that T-ALL was the indication in
which we first saw clinical activity and the belief that T-ALL could
potentially be the fastest route to approval for the drug. Over the past
year we have generated significant clinical data indicating the activity
and safety of Fodosine(TM) in CTCL. Given the necessary delays associated
with addressing the formulation issues now affecting the continued progress
of the T-ALL trial, as well as the limited number of patients with T-ALL
who are eligible to be treated under our protocol, BioCryst's strategy
going forward will be to focus its resources on CTCL as the disease
indication which the company believes could provide the fastest route to
marketing approval of Fodosine(TM). The results in treatment of CTCL with
the oral formulation of Fodosine(TM) remain encouraging and we believe that
this decision will allow us the greatest chance of getting Fodosine(TM) to
a larger number of patients quickly. To this end we have requested a
Special Protocol Assessment (SPA) with the FDA as an integral part of the
clinical design of the next trial in CTCL. We filed our initial application
for the SPA with the FDA last week and look forward to working closely with
the Agency to move this program forward expeditiously."
About BioCryst
BioCryst Pharmaceuticals, Inc. is a leader in the use of
crystallography and structure-based drug design for the development of
novel therapeutics to treat cancer, cardiovascular diseases, autoimmune
diseases, and viral infections. The company is advancing multiple internal
programs toward potential commercialization including Fodosine(TM) in
oncology, BCX-4208 in transplantation and autoimmune diseases and peramivir
in seasonal and life- threatening influenza. BioCryst has a worldwide
partnership with Roche for the development and commercialization of
BCX-4208, and is collaborating with Mundipharma for the development and
commercialization of Fodosine(TM) in markets across Europe, Asia, Australia
and certain neighboring countries. In January 2007 the U.S. Department of
Health and Human Services (DHHS) awarded a $102.6 million, four-year
contract to BioCryst for advanced development of peramivir to treat
seasonal and life-threatening influenza. In February 2007, BioCryst
established a partnership with Shionogi & Co., to develop and commercialize
peramivir in Japan. For more information about BioCryst, please visit the
company's web site at biocryst.
Forward-looking statements
This press release contains forward-looking statements, including
statements regarding future results, performance or achievements. These
statements involve known and unknown risks, uncertainties and other factors
which may cause our actual results, performance or achievements to be
materially different from any future results, performances or achievements
expressed or implied by the forward-looking statements. These statements
reflect our current views with respect to future events and are based on
assumptions and subject to risks and uncertainties. Given these
uncertainties, you should not place undue reliance on these forward-looking
statements. Some of the factors that could affect the forward-looking
statements contained herein include that development and commercialization
of Fodosine(TM) in both T-ALL and CTCL may not be successful, that we may
not resolve satisfactorily the particulate matter issue with the
intravenous formulation of Fodosine(TM), that we may not obtain a
satisfactory SPA for Fodosine(TM) for treatment of CTCL promptly or at all,
that DHHS could reduce or eliminate funding for peramivir, that we or our
licensees may not be able to enroll the required number of subjects in
planned clinical trials of our product candidates and that such clinical
trials may not be successfully completed, that BioCryst or its licensees
may not commence as expected additional human clinical trials with our
product candidates, that our product candidates may not receive required
regulatory clearances from the FDA, that ongoing and future clinical trials
may not have positive results, that we may not be able to complete
successfully the Phase IIb trial for Fodosine(TM) that is currently planned
to be pivotal, that we or our licensees may not be able to continue future
development of our current and future development programs, that our
development programs may never result in future product, license or royalty
payments being received by BioCryst, that BioCryst may not reach favorable
agreements with potential pharmaceutical and biotech partners for further
development of its product candidates, that BioCryst may not have
sufficient cash to continue funding the development, manufacturing,
marketing or distribution of its products and that additional funding, if
necessary, may not be available at all or on terms acceptable to BioCryst.
Please refer to the documents BioCryst files periodically with the
Securities and Exchange Commission, specifically BioCryst's most recent
Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, current reports
on Form 8-K which identify important factors that could cause the actual
results to differ materially from those contained in the projections or
forward-looking statements.
BioCryst Pharmaceuticals, Inc.
biocryst