IDM Pharma Joins NORD to Raise Awareness of Rare Diseases
(Nasdaq: IDMI) today announced that it is joining the National Organization
for Rare Disorders (NORD) and more than 100 patient organizations, caregivers,
researchers and companies developing treatments for rare diseases to observe
Rare Disease Day on
rare diseases, the challenges encountered by those affected and the importance
of research to develop diagnostics and treatments. IDM Pharma is currently
developing mifamurtide (MEPACT in
rare bone cancer in children and young adults.
“Osteosarcoma impacts approximately 1,200 newly diagnosed children and
young adults every year in each of
participation in Rare Disease Day, we hope to bring increased attention to the
impact rare diseases — like osteosarcoma — have on the lives of young
patients and their families, and help break down the barriers some patients
experience when trying to access proper diagnosis and treatment.”
IDM Pharma has developed mifamurtide to treat non-metastatic, resectable
osteosarcoma. The Company is currently awaiting a final decision on European
Marketing Authorization, anticipated within the first quarter, following the
positive opinion adopted by the Committee for Medicinal Products for Human Use
clinical trial ever completed in osteosarcoma, which enrolled approximately
800 patients and was a National Cancer Institute (NCI) funded cooperative
group study conducted by the Children’s Oncology Group (COG). The study
evaluated patient outcomes with the addition of mifamurtide to three- or
four-drug adjuvant chemotherapy (cisplatin, doxorubicin, and methotrexate,
with or without ifosfamide). Results demonstrated that the addition of
mifamurtide to chemotherapy resulted in approximately a 30 percent decrease in
the risk of death with 78 percent of patients surviving after six years of
follow-up after treatment with mifamurtide.
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
Osteosarcoma is an orphan disease with approximately 1,200 new cases
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 Rare Disease Day
Started in 2008 in
being observed in
objective of Rare Disease Day 2009 is to raise awareness with policy makers
and the public of rare diseases and of their impact on patients’ lives. For
additional information on the day, please visit http://www.rarediseases.org.
Mifamurtide U.S. Regulatory Status
As previously announced, in
work with the COG as well as external experts and advisors to gather patient
follow up data from the Phase 3 clinical trial of mifamurtide 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 plans to submit an
amended New Drug Application (NDA) for mifamurtide in mid-2009 and expects to
be in a position to provide an update on the progress of the filing, including
timing, following a meeting scheduled with the FDA in March.
Mifamurtide was granted orphan drug status in
The NDA was submitted to FDA in
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 its innovative products to address the needs of patients and the
physicians who treat these patients.
The Company believes it has adequate cash resources to support its
operations through the first half of 2009 based on its current development and
operating plans. The Company does not currently have operational sales and
marketing infrastructure for mifamurtide and does not currently have plans or
sufficient funds to secure this capability.
The Company continues to evaluate strategic alternatives, which may
include seeking strategic partners, a merger and/or the sale of all or part of
its operations and assets, or raising additional capital to secure operational
sales and marketing infrastructure for mifamurtide.
For more information about the company and its products, visit
This press release includes forward-looking statements that reflect
management’s current views of future events including statements regarding the
timeframe in which the Company’s cash will be sufficient to meet planned
operations, the Company’s plans to collect, analyze and submit additional
Phase 3 data and to respond to other matters raised by the FDA in an amended
NDA for mifamurtide, including the expected timing for submission of the
amended NDA, and the Company’s plans to evaluate strategic alternatives.
Actual results may differ materially from the forward-looking statements due
to a number of important factors, including, but not limited to, the timing of
filing an amended NDA with the FDA, the possibility that additional data from
the Phase 3 clinical trial of mifamurtide and other information in any
amendment to the NDA for mifamurtide submitted by the Company may not provide
adequate support for regulatory approval of mifamurtide in the U.S. within the
timeframe expected by the Company, if at all, whether the Company will be able
to complete any potential strategic transaction on terms acceptable to the
Company’s stockholders, how the volatile economic environment will affect the
Company’s efforts to complete a strategic transaction, 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
SEC. The Company expressly disclaims any intent or obligation to update these
forward-looking statements, except as required by law.
SOURCE IDM Pharma, Inc.