Astex Pharmaceuticals Announces Initiation of SGI-110 Phase 2 Trial in Advanced Hepatocellular Carcinoma (HCC) Patients

Astex Pharmaceuticals, Inc.
Press release

Astex Pharmaceuticals Announces Initiation of SGI-110 Phase 2 Trial in Advanced
Hepatocellular Carcinoma (HCC) Patients

DUBLIN, Calif., 2013-01-02 12:00 CET (GLOBE NEWSWIRE) -- Astex Pharmaceuticals,
Inc. (Nasdaq:ASTX), a pharmaceutical company dedicated to the discovery and
development of novel small molecule therapeutics, announced that it has
initiated a Phase 2, open-label, single-arm, non-randomized clinical trial,
evaluating SGI-110 in the treatment of advanced hepatocellular carcinoma (HCC)
patients who failed prior treatment with Nexavar (sorafenib). 

Advanced Hepatocellular Carcinoma (HCC) is a disease with a very high unmet
medical need and patients who do not respond to the frontline standard of care,
sorafenib, have few therapeutic options. The Phase 2 study is based on
preclinical work showing that SGI-110 is effective in inducing global DNA and
gene specific hypomethylation, thus enabling the re-expression of tumor
suppressor genes in HCC cell lines (Jueliger, S. 2012, November. Poster
presentation, EORTC-NCI-AACR, Dublin, Ireland). 

"There is a high unmet need for therapies for advanced HCC patients who have
failed sorafenib treatment," said Mohammad Azab, MD, chief medical officer. "We
are pleased to initiate this study which expands the investigational breadth of
our development program for SGI-110 to include HCC in addition to the ongoing
studies in myelodysplastic syndromes (MDS), acute myeloid leukemia (AML) and
ovarian cancer." 

About the Study

The study uses a Simon's 2-stage design. Part A would enroll 15 patients in
Stage 1 who will be evaluated for safety, disease control, and for methylation
status of certain tumor suppressor genes known to be involved in HCC. If Part A
is successful, Part B will enroll 31 additional patients in Stage 2. The
primary endpoint of the study is disease control rate (DCR) at 16 weeks,
defined as percentage of patients who achieve a best clinical response of
complete or partial response, or stable disease at that time. Secondary
endpoints of the study include the incidence and severity of adverse events,
global DNA methylation levels, methylation status of selected tumor suppressor
genes in tumor tissue, progression-free survival and overall survival. 

Additional information about the study can be found online at
clinicaltrials.gov: http://clinicaltrials.gov/ct2/show/NCT01752933 

About SGI-110

SGI-110 is a small molecule, DNA-hypomethyating agent with demonstrated
activity in restoring silenced tumor suppressor gene expression in cancer cells
by reversal of DNA methylation. SGI-110 is being evaluated in a first-in-human
Phase 1-2 clinical trial in patients with intermediate or high-risk
myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), in
collaboration with multiple cancer centers, including clinical investigators of
the Stand Up to Cancer Epigenetics Dream Team. SGI-110 is also being
investigated in a Phase 2 clinical trial in combination with carboplatin in
platinum-resistant recurrent ovarian cancer patients. 

SGI-110 is wholly owned by Astex Pharmaceuticals.

About Astex Pharmaceuticals

Astex Pharmaceuticals is dedicated to the discovery and development of novel
small molecule therapeutics with a focus on oncology. The Company is developing
a proprietary pipeline of novel therapies and is creating de-risked products
for partnership with leading pharmaceutical companies. Astex Pharmaceuticals
developed DACOGEN® (decitabine) for Injection and receives significant
royalties on global sales. 

For more information about Astex Pharmaceuticals, Inc., please visit
http://www.astx.com. 

The Astex Pharmaceuticals, Inc. logo is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=12273 

Forward-Looking Statements

This press release contains "forward-looking" statements within the meaning of
Section 21A of the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended, and is subject to the safe harbor
created thereby. These statements are typically preceded by words such as
"believes," "expects," "anticipates," "intends," "will," "may," "should," or
similar expressions. Actual results could differ materially from those
projected in the forward-looking statements as a result of a number of risks
and uncertainties. These forward-looking statements include, but are not
limited to, expectations regarding the advancement of drug candidates in the
clinic; the Company's ability to develop the current and future pipeline into
commercially viable drugs; the expectations regarding our clinical trials
including the timing of clinical proof of concept data from these trials.
Important factors that could cause actual results to differ materially from the
expectations reflected in the forward-looking statements include, but are not
limited to: the outcomes of the on-going clinical trials; risks and
uncertainties related to the research and development of SGI-110. References
made to the discussion of risk factors are detailed in the Company's filings
with the Securities and Exchange Commission including reports on its most
recently filed Form 10-K and Form 10-Q. These forward-looking statements are
made only as of the date hereof, and we disclaim any obligation to update or
revise the information contained in any such forward-looking statements,
whether as a result of new information, future events or otherwise. 


         CONTACT: Timothy L. Enns
         Astex Pharmaceuticals, Inc.
         Senior Vice President
         Corporate Communications & Marketing
         Tel: +1 (925) 560-2810
         E-mail:  
         
         Susanna Chau
         Astex Pharmaceuticals, Inc.
         Manager
         Investor Relations
         Tel: +1 (925) 560-2845
         E-mail:  
         
         Alan Roemer
         The Trout Group
         Managing Director
         Tel: +1 (646) 378-2945
         E-mail:  
         
         Kari Watson
         MacDougall Biomedical Communications
         Senior Vice President
         Tel: +1 (781) 235-3060
         E-mail:  
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Astex Pharmaceuticals Announces Initiation of SGI-110 Phase 2 Trial in Advanced Hepatocellular Carcinoma (HCC) Patients