Licenses Alios HCV Protease Inhibitors
Vertex Pharmaceuticals and Alios BioPharma have agreed to
collaborate on development of 2 novel nucleotide analog HCV
NS5B polymerase inhibitor candidates, now at the preclinical
is an edited excerpt from a recent Vertex press release describing
the licensing deal. The full announcement, including important
safety information, is available online at http://investors.vrtx.com/releasedetail.cfm?ReleaseID=584023.
and Alios BioPharma Announce Exclusive
Worldwide Licensing Agreement for Two Nucleotide
Drug Candidates, Broadening Vertex's Efforts to
Develop New Combinations of Medicines for Hepatitis C
gains worldwide rights to two distinct nucleotide analogues,
ALS-2200 and ALS-2158, that act on hepatitis C polymerase
provides multiple opportunities to develop new "all-oral"
Mass., and South San Francisco, Calif. -- Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) and Alios BioPharma, Inc. today announced
an exclusive worldwide licensing agreement that will add two distinct
nucleotide analogues to Vertex's hepatitis C portfolio. The compounds,
which were discovered by Alios and are known as ALS-2200 and ALS-2158,
have shown in in vitro studies to be potent inhibitors
of the hepatitis C virus (HCV) polymerase, an enzyme essential
for replication of the virus. The addition of these compounds
provides Vertex with multiple opportunities to develop potential,
new, all-oral combination regimens for chronic hepatitis C. Vertex
expects ALS-2200 and ALS-2158 to enter clinical development later
"We are excited to begin working with Vertex, as we believe
that the Alios nucleotide analogues provide an important opportunity
to improve patient care in hepatitis C," said Lawrence M.
Blatt, PhD, Founder and Chief Executive Officer of Alios BioPharma.
"For more than a decade, Vertex has been a leader in the
development of new approaches for treating hepatitis C, and together
we have the potential to create an all-oral, interferon-free,
combination therapy that could improve the safety, efficacy and
ease of administration for patients. We look forward to initiating
clinical development later this year."
"The recent approval of Incivek [telaprevir] was a milestone
in hepatitis C care, and today's announcement underscores our
long-term commitment to further improving the treatment of this
disease with new combinations of medicines," said Peter Mueller,
PhD, Chief Scientific Officer and Executive Vice President of
Global Research and Development at Vertex. "Alios has discovered
anti-HCV nucleotides that have the potential to be leading agents
in hepatitis C. Based on impressive in vitro data, we look forward
to evaluating ALS-2200 and ALS-2158 together and in combination
with our approved and investigational hepatitis C medicines with
the goal of creating a highly potent all-oral regimen in the years
About ALS-2200 and ALS-2158
ALS-2200 and ALS-2158, currently in preclinical development, are
highly potent nucleotide analogues that appear in in vitro and
non-clinical studies to have a high barrier to drug resistance
and the potential to be dosed once-daily. Both compounds are designed
to inhibit the replication of the hepatitis C virus by acting
on the NS5B polymerase. Each compound has its own unique mechanism
of action, which supports the potential for developing these compounds
together as a dual nucleotide regimen and as part of combination
therapy regimens with Vertex's other approved and investigational
medicines for chronic hepatitis C, including Incivek (telaprevir),
an FDA-approved hepatitis C protease inhibitor, and VX-222, an
investigational hepatitis C non-nucleoside polymerase inhibitor.
Data from in vitro studies showed that both ALS-2200 and ALS-2158
had a synergistic effect when combined together and with Incivek
and VX-222. Additionally, in those in vitro studies, both compounds
showed antiviral activity across all genotypes, or forms, of the
hepatitis C virus, including genotypes more prevalent outside
of the U.S. Pan-genotypic compounds for hepatitis C have the potential
to be used across a broad range of people with hepatitis C worldwide.
As part of this agreement, Vertex gains worldwide rights to both
compounds, further enabling the company to potentially expand
development and commercialization efforts in hepatitis C to areas
outside North America over the coming years. The agreement also
includes a research program that will focus on the discovery of
additional nucleotide analogues that act on the hepatitis C polymerase.
Vertex will have the option to select compounds for development
emerging from the research program.
Future Development Plans: Alios and Vertex plan to initiate
clinical development of each compound in the fourth quarter of
2011, which is expected to include studies of the compounds in
healthy volunteers followed by short-duration safety and viral
kinetic studies in people with hepatitis C. The goal of the first
clinical studies of these compounds is to generate data to enable
the initiation of Phase 2 studies as early as the end of 2012.
These Phase 2 studies are expected to evaluate multiple combination
regimens of ALS-2200, ALS-2158, Incivek and VX-222. The combination
studies would be designed to generate sustained viral response
(SVR or viral cure) data. Additional details on the clinical development
program for ALS-2200 and ALS-2158 will be provided later in 2011
upon initiation of the first clinical study.
Terms of the Transaction
As part of the agreement, Alios will receive a $60 million up-front
payment from Vertex for the worldwide rights to ALS-2200 and ALS-2158.
Vertex is responsible for development costs related to ALS-2200
and ALS-2158 and will also provide research funding to Alios.
In addition, Alios would be eligible to receive research and development
milestone payments up to $715 million if both compounds are approved.
Vertex expects to pay approximately $35 million in development
milestones in 2011. Alios is also eligible to receive up to $750
million in sales milestones on sales of all approved medicines
under the collaboration. The agreement also includes tiered royalties
on product sales.
creates new possibilities in medicine. Our team discovers, develops
and commercializes innovative therapies so people with serious
diseases can lead better lives. Vertex scientists and our collaborators
are working on new medicines to cure or significantly advance
the treatment of hepatitis C, cystic fibrosis, epilepsy and other
Founded more than 20 years ago in Cambridge, MA, we now have ongoing
worldwide research programs and sites in the U.S., U.K. and Canada.
For more information and to view Vertex's press releases, please
About Alios BioPharma
BioPharma is a biotechnology company located in South San Francisco,
California, that is developing novel medicines aimed at the treatment
of viral diseases. Alios has an innovative team of highly experienced
scientists and clinical researchers who are developing direct
acting antiviral agents against several human viral pathogens
of public health importance including, Hepatotropic viruses, Respiratory
viruses and other chronic, acute and emerging viral diseases.
Additionally, Alios is developing molecular activators of an interferon
induced, broad spectrum antiviral innate immune pathway called
RNase-L. The overall goal for the Alios therapeutic platform is
to maximize patient benefits in areas of high unmet medical need
through optimization of potency, safety and tolerability.
For more information, visit www.aliosbiopharma.com.
Vertex Pharmaceuticals. Vertex and Alios BioPharma Announce Exclusive
Worldwide Licensing Agreement for Two Nucleotide Drug Candidates,
Broadening Vertex's Efforts to Develop New Combinations of Medicines
for Hepatitis C. Press release. June 13, 2011.