IDM Pharma Announces Updated Data From Mifamurtide (L-MTP-PE) Compassionate Access Program
- Data Presented at the 21st Annual Meeting of The American Society of
Pediatric Hematology/Oncology (ASPHO) Meeting -
IRVINE, Calif., May 15 /PRNewswire-FirstCall/ — IDM Pharma, Inc. (the
Company) (Nasdaq: IDMI) today announced the presentation of updated data
from a compassionate access program evaluating mifamurtide (L-MTP-PE) in
patients with lung metastases as a result of the progression of
osteosarcoma, a rare and often fatal bone tumor that typically affects
children and young adults. The data show that L-MTP-PE in combination with
other therapies is safe, well- tolerated and exhibited signs of disease
control.
The compassionate access program provides L-MTP-PE treatment to
patients who have either failed or cannot tolerate treatment with existing
therapies compared to the Phase 3 pivotal study, which evaluated newly
diagnosed patients.
“The results that we have seen to date from the Phase 3 clinical trial
in patients with non-metastatic osteosarcoma are being supported in
patients with lung metastases as the L-MTP-PE compassionate access
experience continues to be encouraging,” said Peter Anderson, M.D., Ph.D.,
principal investigator and professor of pediatrics, Children’s Cancer
Hospital at The University of Texas M. D. Anderson Cancer Center in
Houston. “With a formalized protocol now in place, we can address the
increasing number of requests for compassionate access to L-MTP-PE and meet
the unmet needs of certain patients by providing this much needed treatment
option.”
Study design and findings
Twenty-nine high-risk osteosarcoma patients (ages 10 – 21) were
enrolled and 27 have been treated with L-MTP-PE in the compassionate access
program to date (May 8, 2008). Patients in the program had documented
diagnosis of high grade osteosarcoma with relapsed or recurrent disease
(locally or metastatic) with resectable or not completely resectable
disease, or who are unable to complete recommended chemotherapy due to
toxicity.
L-MTP-PE (2 mg/m2 IV over 1 hour) was administered twice a week for 12
weeks followed by once a week for 24 weeks. In addition, some patients in
the program were also treated with other agents including aerosol
recombinant granulocyte monocyte colony stimulating factor (GM-CSF) an
immune stimulating agent (n=20), ifosfamide (n=4), and/or gemcitabine
(n=2).
Results to date are as follows:
– Nine patients are alive with disease.
– Nine patients have no evidence of disease.
– Nine patients have died.
– There are two patients for whom the status is unknown.
Treatment with L-MTP-PE combined with other agents including aerosol
GM- CSF was generally well tolerated. Patients treated with chemotherapy
had no unexpected toxicities and toxicity from L-MTP-PE infusions was
minimal. There were no reports of grade 3 or 4 drug-related toxicities with
the exception of fever grade 3 and flu-like symptoms with the first dose.
This was prevented with ibuprofen and acetaminophen after subsequent doses.
One patient developed pleural and pericardial effusion that was possibly
L-MTP-PE and/or GM-CSF related and the patient was removed from the study.
In March, the Company announced that it had formalized a clinical
protocol with the FDA, which provides L-MTP-PE to eligible, high-risk
osteosarcoma patients through a compassionate access study. The
compassionate access study is being conducted initially at M. D. Anderson
and Memorial-Sloan Kettering Cancer Center in New York.
“Patients are our first priority and we are committed to providing
L-MTP- PE to those who desperately need treatment,” said Timothy P.
Walbert, president and chief executive officer, IDM Pharma. “The potential
survival and quality of life benefits for patients treated with L-MTP-PE
continue to be supported through physician experience and we remain
committed to advancing L- MTP-PE through the European and U.S. regulatory
processes to bring this important treatment to market.”
Update on L-MTP-PE Regulatory Status
In January 2008 the Company announced that following presentation of
data at an oral explanation hearing before the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency, the CHMP
determined in a non-binding opinion that L-MTP-PE suggested a possible
clinical benefit in terms of survival and granted the Company a clock stop,
or time extension. The clock stop allows the Company additional time to
respond to all the remaining questions regarding the marketing
authorization application for L-MTP-PE (MAA). The CHMP has requested
clarification of the existing data in order to gain assurance about the
quality of the data before drawing any final conclusions from the data
presented. In addition, the Company is required to address a number of
remaining questions relating to chemistry, manufacturing and controls (CMC)
and the Company expects to provide responses and data regarding these
issues to the CHMP in advance of its meeting scheduled for June 23-26,
although the CHMP may have additional questions or require additional
information regarding these issues. In April, the European regulatory
authorities conducted an inspection of the Children’s Oncology Group (COG)
to assess the quality of the overall survival data from the 2006
confirmatory database included in our applications for regulatory approval,
and to review Good Clinical Practices compliance of COG in terms of patient
randomization and stratification, overall survival data collection, and
study monitoring. The Company supported the COG in this effort.
The Company expects to receive a final opinion from the CHMP in the
third quarter and a final decision from the European Commission in the
fourth quarter of 2008.
As previously announced, in the United States the Company continues to
work with the COG as well as external experts and advisors to gather
patient follow up data from the Phase 3 clinical trial of L-MTP-PE and to
respond to other questions in the non-approvable letter the Company
received from the U.S. Food and Drug Administration (FDA). The Company
expects to submit the amended New Drug Application (NDA) in the fourth
quarter of 2008.
L-MTP-PE was granted orphan drug status in the United States in 2001
and in Europe in 2004. In Europe, the MAA was filed in November 2006 and in
the U.S, the NDA was submitted to FDA in October 2006 and was accepted for
review in December 2006.
About Osteosarcoma
Between two and three percent of all childhood cancers are
osteosarcoma. Because osteosarcoma usually develops from osteoblasts, it
most commonly affects children and young adults experiencing their
adolescent growth spurt. Boys and girls have a similar incidence rate until
later in their adolescence, when boys are more commonly affected. While
most tumors occur in larger bones, such as the femur, tibia, and humerus,
and in the area of the bone that has the fastest growth rate, they can
occur in any bone. The most common symptom is pain, but swelling and
limited movement can occur as the tumor grows.
Osteosarcoma is an orphan disease with fewer than 1,000 new cases
diagnosed in the United States each year. A similar incidence of the
disease exists in Europe. According to the Children’s Oncology Group, the
survival of children with osteosarcoma has remained at 60-65 percent since
the mid-1980s. The standard treatment for osteosarcoma is tumor resection
with combination chemotherapy before and after surgery.
About IDM Pharma
IDM Pharma is focused on the development of innovative cancer products
that either destroy cancer cells by activating the immune system or prevent
tumor recurrence by triggering a specific adaptive immune response. IDM
Pharma is dedicated to maximizing the full therapeutic and commercial
potential of each of its innovative products to address the needs of
patients and the physicians who treat these patients.
For more information about the company and its products, visit
http://www.idm-pharma.com.
Forward-Looking Statements
This press release includes forward-looking statements that reflect
management’s current views of future events including the objectives of the
compassionate access study, the Company’s plans to address the remaining
questions with respect to the MAA for L-MTP-PE during the clock-stop
granted by the CHMP, and the expected timing of a final opinion from the
CHMP and of a final regulatory decision regarding the MAA in the European
Union, as well as the Company’s plans to collect, analyze and submit
additional Phase 3 data in an amended NDA for L-MTP-PE, including the
expected timing for such amended NDA, and to respond to other matters
raised by the FDA. Actual results may differ materially from the
forward-looking statements due to a number of important factors, including,
but not limited to, whether the Company will be able to respond to the
remaining issues with regard to the MAA, including verification of data
quality and CMC items, to the satisfaction of the CHMP, whether the CHMP
will ask the Company for further information at or following the June 2008
meeting to address remaining issues with regard to the MAA, which would
delay the timing of a final opinion from the CHMP, whether the final
opinion of the CHMP will be consistent with the non-binding opinion of the
CHMP, whether the European Commission will follow the final opinion of the
CHMP once issued, whether the timing for the final opinion of the CHMP and
the regulatory decision in Europe will occur as expected by the Company,
the possibility that additional data from the Phase 3 clinical trial of
L-MTP-PE and other information in any amendment to the NDA for L-MTP-PE
submitted by the Company may not provide adequate support for regulatory
approval of L-MTP- PE in the United States within the timeframe expected by
the Company, if at all, and whether the Company will be able to manufacture
and commercialize L- MTP-PE even if it is approved by regulatory
authorities, and whether the cash resources of the Company will be
sufficient to fund operations as planned. These and other risks affecting
the Company and its drug development programs, intellectual property
rights, personnel and business are more fully discussed in the Company’s
Quarterly Report on Form 10-Q filed with the SEC for the quarter ended
March 31, 2008 and other periodic reports filed with the SEC. The Company
expressly disclaims any intent or obligation to update these
forward-looking statements, except as required by law.
SOURCE IDM Pharma, Inc.
