Molecular Insight Pharmaceuticals Presents Positive Data From Azedra(TM) Phase 2a Clinical Trial in Neuroblastoma Tumors
23 Giugno 2010 - 10:01PM
Marketwired
Molecular Insight Pharmaceuticals, Inc. (NASDAQ: MIPI) today
presented positive data from a completed Phase 2a clinical study of
Azedra™, (Ultratrace™ iobenguane I 131 or Ultratrace I-131-MIBG),
the Company's lead oncology therapeutic that targets neuroblastoma,
a common childhood cancer. Phase 2a data presented at the Advances
in Neuroblastoma Research (ANR) Conference in Stockholm, Sweden, on
June 23, 2010, reported that the study successfully provided
safety, toxicity and response data.
Data from the Phase 2a study were presented in an abstract
titled A Phase IIa Trial of Ultratrace (no-carrier added)
Iobenguane I-131 (MIBG): A New Approaches to Neuroblastoma Therapy
(NANT) Study (Abstract 1906390) and in an oral presentation by lead
investigator Katherine K. Matthay, M.D., the Mildred V. Strouss
Professor of Translational Cancer Research and Chief, Pediatric
Hematology-Oncology in Department of Pediatrics at the University
of California, San Francisco. Key conclusions from Dr. Matthay's
presentation, which included data on an additional seven patients
enrolled since the abstract was submitted, include: (a) doses of 12
to 18mCi/kg of Ultratrace iobenguane were tolerated without dose
limiting toxicity (DLT); and (b) Ultratrace iobenguane is active in
tumors, without significant toxicity, with promising responses as
shown by a preliminary observed response rate, using International
Neuroblastoma Response Criteria (INRC), in 5 of 13, or 38 percent
of currently evaluable patients. The data support proceeding to a
pivotal Phase 2 study utilizing the 18 mCi/kg administered
therapeutic dose.
Currently, I-131-MIBG is used to treat neuroblastoma, with a
response rate greater than 30 percent in relapsed disease. The
presence of non-radioactive "carrier" MIBG molecules may inhibit
uptake of I-131-MIBG, resulting in less tumor radiation and
increased risk of cardiovascular side effects. Azedra consists of
the iobenguane molecule radiolabeled with iodine-131 using the
Company's proprietary Ultratrace technology platform, a technique
that avoids large quantities of cold (non-radioactive) iobenguane
in the formulation of the product. In previous animal studies, the
lack of cold iobenguane suggested two significant clinical
benefits: greater tumor uptake and reduced pharmacological toxicity
or side effects.
Dr. Matthay noted: "The primary aim of this Phase 2a study was
to establish the maximum tolerated dose (MTD) of Ultratrace
iobenguane I-131 with autologous hematopoietic stem cell (AHSC)
support. We showed that Ultratrace iobenguane I-131 with AHSC
support is feasible at 18 mCi/kg without significant toxicity and
with promising partial and complete responses that support
proceeding to a pivotal Phase 2 study at18 mCi/kg."
The primary objectives of the study were to determine MTD and
toxicity graded by Common Terminology Criteria for Adverse Events
(CTCAE) of Azedra in patients with refractory high-risk
neuroblastoma; estimate radiation absorbed doses to measurable
lesions and to a standard set of normal organs following a 0.1
mCi/kg intravenous administration of Azedra; and assess objective
tumor response following treatment. Secondary objectives include
evaluation of objective tumor response by INRC.
Eligible patients were age 1 to 30 years old with resistant
neuroblastoma, MIBG uptake, and cryopreserved AHSC. A diagnostic
dose of Ultratrace iobenguane (1-5 mCi) was followed by 3 dosimetry
scans. The administered therapeutic dose was then adjusted based on
absorbed dose estimates and normal organ tolerance limits. AHSC
were infused 14 days post therapy. Response and toxicity were
evaluated at day 60. The Ultratrace iobenguane was escalated in
3mCi/kg increments from 12-21 mCi/kg using 3+3 design. Dose
limiting toxicity was defined as failure to engraft or grade 3 or 4
non-hematologic toxicity except grade 3 pre-defined exclusions. No
DLT was observed and there were no related serious adverse events
reported. Most commonly reported adverse events (AE) included
myelosuppression, gastrointestinal (nausea/vomiting, salivary gland
pain/swelling, increased liver function tests, headache and fever.
Nearly 80 percent of the reported AEs were Grade 1 and 2 by CTCAE
criteria.
About Azedra Azedra, a novel, targeted
radiotherapeutic, recognizes the norepinephrine transporter
molecular target that is over-expressed in neuroendocrine tumor
cells allowing targeted accumulation at these tumor sites. Azedra,
developed using Molecular Insight's proprietary Ultratrace™
technology, permits more efficient and high specific activity
labeling with the therapeutically active 131-Iodine isotope which
could result in maximum delivery of the therapeutic potential of
iobenguane I 131, a radiotherapeutic currently marketed in Europe
to treat neuroendocrine tumors. At a given therapeutic dose, Azedra
minimizes the amount of non-radioactive iobenguane molecules
administered to the patient, potentially reducing pharmacologic
toxicities, especially cardiovascular events or toxicity, and
possibly enabling better tolerated and effective treatment.
Azedra is currently in advanced clinical trials for the
treatment of malignant pheochromocytoma, a rare, hard-to-access
neuroendocrine tumor that develops in the core of an adrenal
gland.
Neuroendocrine Tumors Neuroendocrine
Tumors (NETs) are rare tumors of the nervous and endocrine systems.
Until becoming metastatic, they are typically slowly growing and
difficult to diagnose. Functional NETs secrete excess hormone,
leading to a variety of clinical syndromes, some life threatening
and all adversely affecting quality of life.
A type of NET, neuroblastoma is the most common extracranial
solid cancer in childhood and the most common cancer in infancy,
with an annual incidence of about 650 new cases per year in the
U.S. Neuroblastoma comprises six to ten percent of all childhood
cancers, and fifteen percent of cancer deaths in children. Close to
50 percent of neuroblastoma cases occur in children younger than
two years old. This type of malignant tumor arises from any neural
crest element of the sympathetic nervous system or SNS.
Neuroblastoma most frequently originates in one of the patient's
adrenal glands, but can also develop in nerve tissues in the neck,
chest, abdomen or pelvis.
About Molecular Insight Pharmaceuticals,
Inc. Molecular Insight Pharmaceuticals is a clinical-stage
biopharmaceutical company and pioneer in molecular medicine. The
Company is focused on the discovery and development of targeted
therapeutic and imaging radiopharmaceuticals for use in oncology.
Molecular Insight has five clinical-stage candidates in
development. For further information on Molecular Insight
Pharmaceuticals, please visit www.molecularinsight.com.
Forward-Looking Statements Statements in
this release that are not strictly historical in nature constitute
"forward-looking statements." Such statements include, but are not
limited to, statements about the potential therapeutic benefit of
Azedra in enabling safe, better tolerated and effective treatment,
and Azedra's potential proceeding to a pivotal Phase 2 study. Such
forward-looking statements involve known and unknown risks,
uncertainties, and other factors that may cause the actual results
of Molecular Insight to be materially different from historical
results or from any results expressed or implied by such
forward-looking statements. These factors include, but are not
limited to, risks and uncertainties related to the progress,
timing, cost, and results of clinical trials and product
development programs; difficulties or delays in obtaining
regulatory approval for product candidates; competition from other
pharmaceutical or biotechnology companies; and the additional risks
discussed in filings with the Securities and Exchange Commission
(SEC). The Company's SEC filings are available through the SEC's
Electronic Data Gathering Analysis and Retrieval system (EDGAR) at
http://www.sec.gov/. Press releases for Molecular Insight
Pharmaceuticals, Inc. are available on our website:
http://www.molecularinsight.com/. If you would like to receive
press releases via e-mail, please contact:
investor@molecularinsight.com. All forward-looking statements are
qualified in their entirety by this cautionary statement, and
Molecular Insight undertakes no obligation to revise or update this
release to reflect events or circumstances after the date
hereof.
Contact: Investors Charles H. Abdalian, Jr. Senior
Vice President of Finance Chief Financial Officer (617) 871-6618
cabdalian@molecularinsight.com Media Martin A. Reynolds
Manager Corporate Communications (617) 871-6734
mreynolds@molecularinsight.com Susan Pietropaolo BCC Partners (845)
638-6290 (201) 923-2049 spietropaolo@bccpartners.com
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