Agennix Receives Special Protocol Assessment Approval from FDA for the Pivotal
Trial of Talactoferrin Alfa in First-Line Non-Small Cell Lung Cancer
Trial will also Support a Marketing Authorization Application in the European
Union
HOUSTON, Jan. 17 /PRNewswire/ -- Agennix Incorporated announced today that
the U.S. Food and Drug Administration (FDA) has approved the design of a
single, pivotal, Phase 3 trial evaluating its lead molecule, talactoferrin
alfa, in combination with chemotherapy as first-line treatment in patients
with non-small cell lung cancer (NSCLC) under the Special Protocol Assessment
(SPA) process. Separately, Agennix received Scientific Advice from the
European Medicines Agency (EMEA) indicating that this single trial will also
support a Marketing Authorization Application (MAA) in the European Union.
Agennix had previously received Fast Track designation from the FDA for this
indication.
"The Special Protocol Assessment agreement with the FDA is an important
step for advancing the investigation of talactoferrin in NSCLC," said Dr.
Roman Perez-Soler, Director, Division of Medical Oncology, Albert Einstein
College of Medicine, New York City. "The Phase 2 data from the two separate
randomized, double-blind, placebo-controlled NSCLC trials are very promising.
I look forward to participating in the Phase 3 talactoferrin trials for both
NSCLC indications -- first-line in combination with chemotherapy, and
talactoferrin monotherapy in patients who have failed two or more previous
therapies."
In the randomized, double-blind, placebo-controlled Phase 2 NSCLC trials,
talactoferrin (or placebo) was administered either in combination with
chemotherapy in chemo-naive patients, or as monotherapy in patients who had
failed previous chemotherapy. Both trials met their prospectively defined
primary endpoints -- improvement in response rate (first-line combination
trial) and improvement in survival (monotherapy trial), with supporting trends
on secondary efficacy endpoints. Both trials also showed statistically
significant reductions in total adverse events and in significant (grade 3 or
higher) adverse events in the talactoferrin arms.
Agennix is preparing to initiate Phase 3 trials in both NSCLC indications
(talactoferrin in combination with chemotherapy in previously untreated
patients and talactoferrin monotherapy in patients who have failed two or more
previous therapies).
"Talactoferrin has the potential to be an important advance in the
treatment of NSCLC," said Dr. Waun Ki Hong, head of the Division of Cancer
Medicine at M.D. Anderson Cancer Center and member of Agennix's Scientific
Advisory Board. "The activity and safety profile seen in the talactoferrin
trials to date are very exciting and provide hope for improved treatment of
this disease. I look forward to confirmation of the Phase 2 results in the
Phase 3 trials."
About the Phase 3, First-Line NSCLC Study
The Phase 3, multinational, randomized, double-blind, placebo-controlled
study will enroll 1,100 previously untreated patients with Stage IIIB or IV
NSCLC. The patients will be randomly assigned to receive up to six cycles of
standard chemotherapy (carboplatin + paclitaxel) plus either oral
talactoferrin or placebo. Following six cycles of chemotherapy, or
discontinuation prior to six cycles for reasons other than progression,
patients will receive talactoferrin or placebo as maintenance therapy until
disease progression. Progression-free survival and overall survival will be
the primary endpoints for accelerated approval and regular approval,
respectively. Secondary endpoints include adverse event reductions, confirmed
response rate, duration of response and safety.
About the SPA
The SPA approval represents a binding agreement (in the absence of new
public health concerns unrecognized at the time of protocol assessment)
between the Company and the FDA that the design and planned analyses of the
study will adequately support a biologics license application (BLA). As part
of the SPA process, the FDA reviewed and approved the trial protocol,
statistical analysis plan, independent radiological review charter, data
safety monitoring board charter, case report forms and site reference manual.
Before approving the SPA, the FDA also evaluated product characterization data
for batch-to-batch consistency for Phase 3 and commercial talactoferrin
production.
About Talactoferrin Alfa
Talactoferrin, a novel dendritic cell activator (DCA), is a unique
recombinant form of human lactoferrin, an important immunomodulatory protein.
In 1988, Dr. Bert O'Malley, chair of molecular and cellular biology at
Baylor College of Medicine, Houston, Texas, discovered a way to produce this
protein in the laboratory, thus paving the way for testing its potential to
help fight serious diseases that cause enormous suffering worldwide. "Baylor
views this as exciting news, not only for the important research being done at
this medical school but also for the historic battle being conducted to find
effective treatments for the most lethal forms of cancer," said Dr. Peter
Traber, President of Baylor College of Medicine. "It is our sincere hope that
the Phase 3 trials will be successful and this drug can be eventually used
globally."
Lactoferrin, found in the highest concentration in milk, is expressed
throughout the body in immune cells and on all body surfaces exposed to the
external environment. Lactoferrin plays an important role in helping to
establish the immune system, including the Gut Associated Lymphoid Tissue
(GALT), in infants. Talactoferrin is produced in Aspergillus niger, a
filamentous fungus, and is structurally identical to native human lactoferrin
in all material respects, differing only in its glycosylation.
Talactoferrin is an orally administered protein that mediates its activity
through the gut and the GALT -- the largest lymphoid organ in the body. It
acts through a novel mechanism of dendritic cell recruitment and activation.
Following oral administration, talactoferrin is transported by the M-cells
into the small intestinal Peyer's Patches, where it recruits circulating
immature dendritic cells bearing tumor antigens to the GALT and induces their
maturation. DC maturation in the presence of tumor antigens and lymphoid
effector cells induces a strong systemic innate and adaptive immune response
mediated by anti-cancer Natural Killer (NK) cells, CD8+ lymphocytes and NK-T
cells. This results in the activation of tumor-draining lymph nodes, cellular
infiltration of distant tumors and tumor-cell death. Mounting the initial
immune response in the GALT -- away from the primary tumor and using a
physiologically important pathway -- minimizes the effect of the cancer's
local immunosuppressive defenses.
About NSCLC
In the United States, lung cancer is the second most frequent cancer in
both men (next to prostate cancer) and women (next to breast cancer). It
remains the major cause of cancer death, killing more people than breast
cancer, prostate cancer and colorectal cancer combined, and accounting for
almost 30% of all cancer-related deaths.
NSCLC accounts for approximately 80% of all new lung cancer cases, with
approximately 150,000 patients in the United States and 300,000 patients in
Europe diagnosed each year. Most patients diagnosed with NSCLC have late-
stage disease (Stage IIIB or IV), which is not surgically resectable. The
current U.S. standard of care for these patients is systemic chemotherapy.
Even with the available therapy, the five-year survival rate for these
patients is less than 3%.
About Agennix
Agennix is a private biotechnology company developing a first-in-class
molecule with activity in several types of cancer and in other indications
with unmet medical needs. This molecule, talactoferrin, is a targeted
dendritic cell activator with a novel mechanism of action. Agennix is
preparing to initiate Phase 3 trials in two NSCLC indications (talactoferrin
in combination with chemotherapy in previously untreated patients and
talactoferrin monotherapy in patients who have failed two or more previous
therapies), a Phase 2b trial in renal cell cancer, and Phase 2 trials in other
indications. Talactoferrin's potential advantages in NSCLC and in other tumor
types include its promising anti-tumor activity, its well tolerated safety
profile including a reduction of some chemotherapy toxicities, its oral route
of administration, and its apparent usefulness in multiple tumor types both as
a single agent and in combination with other drugs. Agennix retains all of
the commercial and economic rights to talactoferrin for all indications
worldwide, and has strong global intellectual property protection for
talactoferrin.
More information about Agennix is available on the Company's web site at
http://www.agennix.com/.
SOURCE Agennix Incorporated
Rick Barsky, Chief Executive Officer, or Atul Varadhachary, President & COO,
both of Agennix, Inc., +1-713-552-1091; or Brad Miles, Media, of BMC
Communications Group for of Agennix, Inc., +1-212-477-9007 ext. 17; or
Jonathan Fassberg, Investors, of Trout Group for of Agennix, Inc., +1-646-378-
2916
