- FDA grants Orphan Drug Designation for IMO-8400 for the
treatment of Waldenström's macroglobulinemia
- Four-week safety review by Data Review Committee for second
dose cohort completed in ongoing WM trial; Third dose cohort opened
for enrollment
Idera Pharmaceuticals, Inc. (Nasdaq:IDRA), a clinical-stage
biopharmaceutical company developing nucleic acid therapeutics for
patients with cancer and rare diseases, today announced that the
U.S. Food and Drug Administration (FDA) has granted orphan drug
designation for IMO-8400, an antagonist of the endosomal Toll-like
receptors (TLRs) 7, 8 and 9, for the treatment of Waldenström's
macroglobulinemia (WM). Additionally, Idera is providing a progress
update on the ongoing Phase 1/2 clinical trial being conducted in
WM.
Idera is currently conducting a Phase 1/2 clinical trial of
IMO-8400 in patients with WM (ClinicalTrials.gov identifier
NCT02092909) who have a history of relapse or failure to respond to
one or more prior therapies. In B‐cell lymphomas characterized by
the MYD88 L265P oncogenic mutation, including WM, preclinical
studies have shown that TLR signaling is overactivated, thereby
enabling tumor cell survival and proliferation. About 90 percent of
WM patients are reported to harbor the MYD88 L265P oncogenic
mutation.
The objectives of the trial are to evaluate the compound's
safety, tolerability and potential clinical activity. The protocol
includes three dose-escalation cohorts of IMO-8400 administered
subcutaneously. The trial's independent data review committee has
completed its review of four-week safety data from the second dose
cohort (1.2 mg/kg/week) and has determined that Idera may open
enrollment in the third dose cohort (2.4 mg/kg/week). Final 24-week
safety and clinical activity data are anticipated in the second
half of 2015.
Orphan drug designation is granted by the FDA Office of Orphan
Products Development to drugs intended for the treatment of a rare
disease or condition that affects fewer than 200,000 people in the
United States. This designation provides certain incentives,
including eligibility for federal grants, research and development
tax credits, waiver of PDUFA filing fees and a seven-year marketing
exclusivity period, once the product is approved and as long as
orphan drug designation is maintained.
The approval of an orphan drug designation request does not
alter the standard regulatory requirements and processes for
obtaining marketing approval of an investigational drug. Sponsors
must establish safety and efficacy of a compound in the treatment
of a disease through adequate and well-controlled studies.
About Waldenström's macroglobulinemia (WM)
Waldenström's macroglobulinemia is a non-Hodgkin lymphoma of
malignant lymphoplasmacytic B-cells that commonly involves the
blood and bone marrow and may spread to almost any organ in the
body. Approximately 1,000 to 1,500 new cases of WM are diagnosed in
the United States each year.1 Symptoms include fatigue, night
sweats, headaches, visual problems, pain and abnormal bleeding due
to complications such as anemia, retinopathy and peripheral
neuropathy.2 The disease is incurable and there are currently no
drugs specifically approved for the treatment of WM in patients
harboring the MYD88 L265P oncogenic mutation. About 90 percent of
WM patients are reported to harbor the MYD88 L265P oncogenic
mutation.3
About IMO-8400
Idera's Toll-like receptor (TLR) antagonist drug candidates have
been created using a proprietary chemistry-based drug discovery
platform. IMO-8400 is a first-in-class synthetic
oligonucleotide-based antagonist of endosomal TLRs 7, 8, and 9. In
April 2014, Idera presented preclinical data at the American
Association for Cancer Research Annual Meeting from preclinical
studies in which IMO-8400 inhibited the survival and proliferation
of human B-cell lymphoma cells, including WM cells, harboring the
oncogenic MYD88 L265P genetic mutation. IMO-8400 has been
well-tolerated in a Phase 1 trial in 42 healthy subjects at single
and multiple escalating doses up to 0.6 mg/kg for four weeks, and
has shown inhibition of immune responses mediated by TLRs 7, 8, and
9. In March 2014 and December 2014, Idera announced top-line data
from a Phase 2 trial that showed evidence of tolerability and
clinical activity in patients with psoriasis who were treated with
IMO-8400 at doses of up to 0.6 mg/kg/week for 12 weeks. Idera is
pursuing clinical development of IMO-8400 in genetically defined
forms of B-cell lymphoma, including WM and diffuse large B-cell
lymphoma in patients harboring the MYD88 L265P mutation, and in
rare autoimmune diseases, including dermatomyositis.
About Idera Pharmaceuticals
Idera Pharmaceuticals is a clinical-stage biopharmaceutical
company developing a novel therapeutic approach for the treatment
of genetically defined forms of B-cell lymphoma and rare autoimmune
diseases. Idera's proprietary technology involves creating novel
nucleic acid therapeutics designed to inhibit over-activation of
Toll-like receptors (TLRs). In addition to its TLR programs, Idera
is developing gene silencing oligonucleotides that it has created
using its proprietary technology to inhibit the production of
disease-associated proteins by targeting RNA. To learn more about
Idera, visit www.iderapharma.com.
References:
1 American Cancer Society. What are the key statistics about
Waldenstrom macroglobulinemia? Available at:
http://www.cancer.org/cancer/waldenstrommacroglobulinemia/detailedguide/waldenstrom-macroglobulinemia-key-statistics-w-m.
Accessed December 2014.
2 American Cancer Society. Signs and Symptoms of Waldenstrom
macroglobulinemia. Available at:
http://www.cancer.org/cancer/waldenstrommacroglobulinemia/detailedguide/waldenstrom-macroglobulinemia-signs-symptoms.
Accessed December 2014.
3 Treon SP, et al. MYD88 L265P somatic mutation in Waldenström's
macroglobulinemia. N Engl J Med. 2012 Aug 30;367(9):826-33.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. All statements, other than statements of historical fact,
included or incorporated in this press release, including
statements regarding the Company's strategy, future operations,
collaborations, intellectual property, cash resources, financial
position, future revenues, projected costs, prospects, plans, and
objectives of management, are forward-looking statements. The words
"believes," "anticipates," "estimates," "plans," "expects,"
"intends," "may," "could," "should," "potential," "likely,"
"projects," "continue," "will," and "would" and similar expressions
are intended to identify forward-looking statements, although not
all forward-looking statements contain these identifying words.
Idera cannot guarantee that it will actually achieve the plans,
intentions or expectations disclosed in its forward-looking
statements and you should not place undue reliance on the Company's
forward-looking statements. There are a number of important factors
that could cause Idera's actual results to differ materially from
those indicated or implied by its forward-looking statements.
Factors that may cause such a difference include: whether results
obtained in preclinical studies and clinical trials such as the
preclinical and clinical data described in this release will be
indicative of the results that will be generated in future clinical
trials; whether interim results obtained in preclinical studies and
clinical trials will be indicative of the final results of such
preclinical studies and clinical trials; whether products based on
Idera's technology will advance into or through the clinical trial
process on a timely basis or at all, warrant submission for
regulatory approval and receive approval from the United States
Food and Drug Administration or equivalent foreign regulatory
agencies; whether, if the Company's products receive approval, they
will be successfully distributed and marketed; whether the
Company's cash resources are sufficient to fund the Company's
proposed programs and the Company's operations for the period
anticipated; whether IMO-8400 will continue to qualify for orphan
drug designation and receive market exclusivity pursuant to the
applicable orphan drug regulations; and such other important
factors as are set forth under the caption "Risk Factors" in the
Company's Quarterly Report on Form 10-Q for the quarter ended
September 30, 2014. Although Idera may elect to do so at some point
in the future, the Company does not assume any obligation to update
any forward-looking statements and it disclaims any intention or
obligation to update or revise any forward-looking statement,
whether as a result of new information, future events or
otherwise.
CONTACT: Investor and Media Contact
Robert Doody
Vice President, Investor Relations and
Corporate Communications
484-639-7235
rdoody@iderapharma.com
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