Full Press Release Details
Announces Initiation of Palifosfamide Pivotal Study
Front-Line Therapy Trial for Patients with Metastatic Soft Tissue Sarcoma
to Host Conference Call and Webcast with Outside Experts
Tuesday, July 20th at 8:00
NEW YORK, NY (July 19, 2010) -
ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP) today announced the initiation of the
pivotal Phase III clinical trial for palifosfamide (ZymafosTM) in
patients with front-line metastatic soft tissue sarcoma. The study, called
PICASSO 3, is an international, randomized, double-blinded, placebo-controlled
trial designed to enroll approximately 424 patients with metastatic soft tissue
sarcoma who have never been treated with chemotherapy for metastatic disease.
The study is designed to evaluate the safety and efficacy of palifosfamide
administered with doxorubicin compared with doxorubicin administered with
placebo, with no crossover between arms. Progression-free survival (PFS) is the
primary endpoint for accelerated approval, with overall survival (OS) as the
primary endpoint for full approval. Palifosfamide has Orphan Drug status in both
Europe and the United States.
pivotal trial protocol was developed in a process with the U.S. Food and Drug
Administration (FDA) that included discussion at an End of Phase II meeting and
a subsequent dialogue for Special Protocol Assessment (SPA). FDA advised that
PFS could be used as a primary endpoint outside of formal SPA with the study
outcome subject to review. Regulatory acceptability will depend on the magnitude
of the difference between the trial study arms as well as a risk and benefit
analysis. Having reached consensus with FDA, including on the methodology of
radiologic evaluation of PFS, and based upon external expert opinion, the
Company elected to initiate the pivotal Phase III trial without formal SPA and
retaining PFS as a primary endpoint.
PICASSO 3 trial has 85% power to detect a 0.60 hazard ratio (HR) advantage for
the palifosfamide combination arm for PFS. Following a pre-determined number of
PFS events and Independent Data Monitoring Committee (IDMC) review and
recommendation, coupled with review of the then available survival data by the
IDMC (to which the Company and Investigators will remain blinded), the Company
could file for accelerated approval based on PFS. The Company and its external
advisors estimate that a median increase in PFS of 3 months or greater over the
control arm (control arm median PFS estimated to be 4.3 months) could achieve a
targeted hazard ratio (HR=0.60; p=0.0005, one-tailed) and also predict a
demonstrable improvement in overall survival. Sarcoma and oncology experts
consulted by the Company believe that this would represent a clinically
meaningful improvement in PFS in this disease setting and that this study design
is a statistically reliable evaluation regarding whether the experimental
intervention is safe and provides clinically meaningful benefit. The PICASSO 3
study is designed as a close follow-on study to the publicly-reported PICASSO
trial, which demonstrated a statistically significant improvement in PFS
(HR=0.39; median PFS improvement of 3.4 months, p=0.023) for the combination
over doxorubicin alone.
will be conducted at approximately 150 centers in North America, Europe, South
America, Australia, Israel and Korea.
Steering Committee for the pivotal trial includes:
stated: "Metastatic soft-tissue sarcoma is a disease for which we have seen few
advances in treatment and no U.S. regulatory approvals in over two decades.
Palifosfamide has demonstrated promising activity and tolerability in Phase II,
including a clinically meaningful improvement in PFS."
3 is powered to show a significant improvement in disease control, as assessed
by PFS," added Dr. Demetri. "This design allows for the possibility of
accelerated approval using a PFS endpoint, an outcome which leaders in the
sarcoma community believe could provide patients in vital need of more treatment
options with earlier access to a new therapy."
tissue sarcomas are cancers of the body soft tissues, including cartilage,
muscle, fat, nerves, blood vessels and other connective tissue. They may develop
in any part of the body, but are most common in the trunk, arms, and legs.
According to the American Cancer Society, 10,520 new cases of adult soft tissue
sarcomas will be diagnosed in the United States in 2010. No new therapies have
been approved for use in sarcoma in the U.S. in over 20 years.
Conference Call and Webcast
Tuesday, July 20th at 8:00 a.m. EST
Company will host a conference call and live audio webcast Tuesday, July 20th at 8:00
a.m. EST. Dr. Maki will be joining the ZIOPHARM management team in a discussion
regarding the initiation of the pivotal Phase III clinical trial for
palifosfamide (ZymafosTM) in
patients with front-line metastatic soft tissue sarcoma. Also participating in
the call will be Josephine Torrente, attorney with Hyman, Phelps & McNamara,
Washington, DC, as outside regulatory strategic advisor and counsel to ZIOPHARM.
The call can be accessed by calling (877) 375-9144 (U.S. and Canada) or +1 253
237-1150 (international). To access the live audio webcast, or the
subsequent archived recording, visit the "Investors - Events &
Presentations" section of the ZIOPHARM website at www.ziopharm.com. The
webcast will be recorded and available for replay on the company's website for
ZIOPHARM Oncology, Inc.:
biopharmaceutical company engaged in the development and commercialization of a
diverse portfolio of cancer drugs. The Company is currently focused on three
ZIO-201) references a novel composition (tris formulation) that comprises the
functional active metabolite of ifosfamide, a standard of care for treating
sarcoma, lymphoma, testicular, and other cancers. Palifosfamide delivers
only the cancer fighting component of ifosfamide. It is expected to overcome the
resistance seen with ifosfamide and cyclophosphamide, two of the most commonly
used DNA-alkylating drugs used to treat cancers.
mitochondrial-targeted agent (organic arsenic) being developed for the treatment
of various hematologic and solid cancers.
novel, oral tubulin binding agent that targets both mitosis and cancer cell
migration. In addition, indibulin is expected to have several potential
benefits, including oral dosing, application in multi-drug resistant tumors, no
neuropathy and minimal overall toxicity
operations are located in Boston, MA with an executive office in New York
City. Further information about ZIOPHARM may be found at www.ziopharm.com.
Safe Harbor Statement:
press release contains forward-looking statements for ZIOPHARM Oncology, Inc.
that involve risks and uncertainties that could cause the Company's actual
results to differ materially from the anticipated results and expectations
expressed in these forward-looking statements. These statements are based on
current expectations, forecasts and assumptions that are subject to risks and