Cellectar’s CLR 131 Receives FDA Orphan Drug Designation for the Treatment of Pediatric Osteosarcoma
September 25 2018 - 8:30AM
Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of drugs for the treatment of cancer, announces
that the U.S. Food and Drug Administration (FDA) Office of Orphan
Products Development has granted Orphan Drug Designation (ODD) to
CLR 131, the company’s lead Phospholipid Drug Conjugate™ (PDC)
product candidate, for the treatment of pediatric osteosarcoma, a
rare pediatric cancer. CLR 131 also received Rare Pediatric Disease
Designation for osteosarcoma, as announced by the company on
September 17, 2018.
“Osteosarcoma is the most common type of primary bone cancer
occurring most frequently in children. Currently, there are no
commercially available drugs for pediatric sarcoma, including
osteosarcoma,” said John Friend, M.D., chief medical officer of
Cellectar. “This orphan designation for osteosarcoma is the fourth
such designation granted by the FDA to CLR 131 for the treatment of
rare pediatric cancers in the last six months, and we look forward
to evaluating CLR 131 in these deadly and underserved
diseases.”
The FDA grants ODD to therapies targeting conditions that affect
fewer than 200,000 people in the U.S. The designation provides
seven-year market exclusivity, increased engagement and assistance
from the FDA, tax credits for certain research, research grants and
a waiver of the New Drug Application user fee. In 2018 the FDA also
granted CLR 131 orphan drug and rare pediatric disease designations
for the treatments of neuroblastoma, rhabdomyosarcoma and Ewing’s
sarcoma.
Cellectar plans to initiate a Phase 1 clinical study evaluating
CLR 131 for the treatment of pediatric patients with osteosarcoma,
Ewing’s sarcoma, rhabdomyosarcoma, neuroblastoma, high-grade glioma
and lymphomas. The trial is designed to evaluate the safety,
tolerability, pharmacokinetics and pharmacodynamics of CLR 131 in
pediatric patients with these cancer types. Further details about
the trial can be found at clinicaltrials.gov using the
identifier number NCT03478462.
About OsteosarcomaOsteosarcoma derives from
bone forming mesenchymal, or connective tissue, cells and is the
most commonly diagnosed primary bone malignancy among children and
adolescents. The incidence is about 4.4 cases per 1 million per
year in children younger than 24 years. While there is a 70% cure
rate among patients with localized disease, 5-year overall survival
rates are approximately 20% for among patients who develop
metastatic disease. Additionally, among patients who experience
disease progression or recurrence survival for is less than
30%.
About CLR 131CLR 131 is Cellectar’s
investigational radioiodinated PDC therapy that exploits the
tumor-targeting properties of the company's proprietary
phospholipid ether (PLE) and PLE analogs to selectively deliver
radiation to malignant tumor cells, thus minimizing radiation
exposure to normal tissues. CLR 131 is in a Phase 2 clinical study
in R/R MM and a range of B-cell malignancies and a Phase 1b
clinical study in patients with R/R MM exploring fractionated
dosing. The objective of the multicenter, open-label, Phase 1b
dose-escalation study is the characterization of safety and
tolerability of CLR 131 in patients with R/R MM. Patients in
Cohorts 1-4 received single doses of CLR 131 ranging from 12.5
mCi/m2 to 31.25 mCi/m2 as well as a fractionated dose of 15.625
mCi/m2 given twice over seven days in Cohort 5. All study doses and
regimens have been deemed safe and well tolerated by an independent
Data Monitoring Committee. The company is currently initiating a
Phase 1 study with CLR 131 in pediatric solid tumors and lymphoma,
and is planning a second Phase 1 study in combination with external
beam radiation for head and neck cancer.
About Cellectar Biosciences, Inc.Cellectar
Biosciences is focused on the discovery, development and
commercialization of drugs for the treatment of cancer. The company
plans to develop proprietary drugs independently and through
research and development (R&D) collaborations. The core drug
development strategy is to leverage our PDC platform to develop
therapeutics that specifically target treatment to cancer cells.
Through R&D collaborations, the company’s strategy is to
generate near-term capital, supplement internal resources, gain
access to novel molecules or payloads, accelerate product candidate
development and broaden our proprietary and partnered product
pipelines.
The company's lead PDC therapeutic, CLR 131, is in a Phase 1
clinical study in patients with R/R MM and a Phase 2 clinical study
in R/R MM and a range of B-cell malignancies. The company is
currently initiating a Phase 1 study with CLR 131 in pediatric
solid tumors and lymphoma, and is planning a second Phase 1 study
in combination with external beam radiation for head and neck
cancer. The company’s product pipeline also includes two
preclinical PDC chemotherapeutic programs (CLR 1700 and 1900) and
partnered assets include PDCs from multiple R&D
collaborations.
For more information please visit www.cellectar.com.
Forward-Looking Statement DisclaimerThis news
release contains forward-looking statements. You can identify these
statements by our use of words such as "may," "expect," "believe,"
"anticipate," "intend," "could," "estimate," "continue," "plans,"
or their negatives or cognates. These statements are only estimates
and predictions and are subject to known and unknown risks and
uncertainties that may cause actual future experience and results
to differ materially from the statements made. These statements are
based on our current beliefs and expectations as to such future
outcomes. Drug discovery and development involve a high degree of
risk. Factors that might cause such a material difference include,
among others, uncertainties related to the ability to raise
additional capital, uncertainties related to the disruptions at our
sole source supplier of CLR 131, the ability to attract and retain
partners for our technologies, the identification of lead
compounds, the successful preclinical development thereof, the
completion of clinical trials, the FDA review process and other
government regulation, the volatile market for priority review
vouchers, our pharmaceutical collaborators' ability to successfully
develop and commercialize drug candidates, competition from other
pharmaceutical companies, product pricing and third-party
reimbursement. A complete description of risks and uncertainties
related to our business is contained in our periodic reports filed
with the Securities and Exchange Commission including our Form 10-K
for the year ended December 31, 2017 and our Form 10-Q for the
quarterly period ended June 30, 2018. These forward-looking
statements are made only as of the date hereof, and we disclaim any
obligation to update any such forward-looking statements.
CONTACT: LHA Investor RelationsMiriam
Weber Miller212-838-3777mmiller@lhai.com
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