CAMBRIDGE, Mass. and EXTON, Pa., April 11, 2017 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (Nasdaq:IDRA), a
clinical-stage biopharmaceutical company developing toll-like receptor and RNA therapeutics for patients with cancer and rare
diseases, today announces successful completion of the phase 1 portion of the ongoing Phase 1/2 clinical trial of intratumoral
IMO-2125. Intratumoral IMO-2125 is an agonist of TLR9, in combination with ipilimumab for the treatment of anti-PD-1 refractory
metastatic melanoma. Enrollment has begun for the Phase 2 portion of the trial with the 8mg dose of intratumoral IMO-2125.
The Phase 1 dose escalation of IMO-2125 in combination with pembrolizumab is ongoing.
“We are very pleased with the progress to date in the Phase 1 dose escalation trial of IMO-2125 in combination with ipilimumab,
and with the outcomes observed,” stated Joanna Horobin, M.B., Ch.B., Idera’s Chief Medical Officer. “IMO-2125 in combination
with ipilimumab demonstrated preliminary evidence of meaningful clinical activity in this anti-PD-1 refractory metastatic melanoma
patient population which represents a high unmet medical need. All dose levels have been well tolerated and did not
exacerbate the safety issues commonly observed with ipilimumab. Furthermore, data from multiple parameters of immune markers
from tumor biopsies have been very informative in establishing proof-of-mechanism and supporting the dose selection for the Phase 2
portion of trial.”
The Phase 2 portion of the trial utilizes a Simon two-stage design to evaluate the objective response rate of IMO-2125 in
combination with ipilimumab, compared to historical data for ipilimumab alone in the anti-PD-1 refractory metastatic melanoma
population. The ipilimumab arm of IMO-2125-204 has already met the pre-specified futility assessment to advance immediately into
the second stage of the Phase 2 portion of the trial given that 2 patients treated at the Phase 2 dose experienced confirmed
responses, including one complete response (CR).
All dose levels of IMO-2125 in the Phase 1 portion of the trial have been well tolerated; however the 8 mg dose level has been
selected for the Phase 2 portion of the trial based on its safety, clinical efficacy, and data from multiple translational immune
parameters supporting the mechanism. Phase 2 will evaluate twenty-one patients dosed at 8mg, of which 9 are already enrolled.
The MD Anderson Cancer Center will continue to lead the trial and will be joined by additional centers. In addition to potential
interim updates, the company expects to have overall response rate (ORR) data available in the first quarter of 2018.
Additionally, the company has begun and will continue to engage in discussions with regulatory authorities regarding the path to
registration for IMO-2125 in combination with ipilimumab in PD-1 refractory metastatic melanoma patients.
The Phase 1 clinical trial of intratumoral IMO-2125 in combination with pembrolizumab in PD-1 refractory melanoma patients is
enrolling as expected, and patient enrollment in a phase 1 trial of intratumoral IMO-2125 monotherapy in multiple tumor types has
been activated and the first patient is expected to enroll early this quarter.
“I am very encouraged by the tremendous progress that has been made to date to advance us to this important stage in IMO-2125’s
development cycle,” stated Vincent Milano, Idera’s Chief Executive Officer. “There is a very clear unmet medical need for
those patients for whom current checkpoint inhibitor therapies are not providing adequate solutions. We are incredibly
focused on advancing this program as rapidly as possible for these patients, and we are also looking forward to exploring areas
outside of melanoma in which intratumoral IMO-2125 may also serve an important role through its unique mechanism of action within
the tumor microenvironment.”
About the Phase 1/2 trial of IMO-2125 in combination with ipilimumab
The Phase 1/2 trial of intratumoral IMO-2125 in combination with ipilimumab is being conducted in patients who are refractory to
anti-PD-1 therapy. The phase 1 portion of the trial was conducted at MD Anderson Cancer Center and the phase 2 portion of the
trial will expand to include additional centers. In the Phase 1 portion of the trial, four dose levels of IMO-2125 (4, 8, 16
and 32 mg) have been administered intratumorally in one targeted lesion at weeks 1, 2, 3, 5, 8 and 11, in combination with the
standard dosing regimen of ipilimumab, beginning on week 2. The Phase 2 expansion portion of the trial utilizes a Simon
two-stage design. If at least 2 of the first 10 patients treated at the Phase 2 dose experience confirmed response the futility
hurdle has been met and the trial may continue to enroll. Phase 2 will evaluate 21 patients at the phase 2 dose. Tumor biopsies
have been collected pre- and post-24 hours of the first dose of IMO-2125, as well as at 8 and 13 weeks to evaluate multiple immune
markers. Clinical activity has been evaluated by the RECIST v1.1 criteria. Clinical data from this study has been
presented at SITC 2017, ASCO-SITC 2017 and AACR 2017, and can be found also on Idera’s corporate website at http://www.iderapharma.com/our-approach/key-publications/.
About IMO-2125
Toll-like receptors (TLRs) play a central role in the innate immune system, the body's first line of defense
against invading pathogens, as well as damaged or dysfunctional cells including cancer cells. The innate immune system is also
involved in activating the adaptive immune system, which marshals highly specific immune responses to target pathogens or tissue.
Cancer cells may exploit regulatory checkpoint pathways to avoid being recognized by the immune system, thereby shielding the tumor
from immune attack. Checkpoint inhibitors such as agents targeting CTLA4 or programmed cell death protein 1 (PD1) are designed to
enable the immune system to recognize tumor cells. In this setting, intratumoral TLR9 agonist administration may increase the
tumor-infiltrating lymphocytes (TILs), and thereby potentiate anti-cancer activity of checkpoint inhibitors in the injected tumor
as well as systemically.
IMO-2125, Idera’s TLR9 agonist, has been created using the company's proprietary chemistry-based discovery platform.
IMO-2125 has been shown in various scientific presentations and publications to activate dendritic cells and induce interferon.
Idera selected IMO-2125 to advance into clinical development in combination with checkpoint inhibitors based on this immunological
profile. In previously completed clinical trials, subcutaneous administration of IMO-2125 was very well tolerated in about
114 patients with hepatitis C. Idera has conducted further preclinical and clinical research evaluating the potential of
IMO-2125 to enhance the anti-tumor activity of other checkpoint inhibitors in cancer immunotherapy with data has been presented at
several scientific and medical conferences during the past few years. The posters from these presentations can be found at
http://www.iderapharma.com/our-approach/key-publications.
About Metastatic Melanoma
Melanoma is a type of skin cancer that begins in a type of skin cell called melanocytes. As is the case in many forms of
cancer, melanoma becomes more difficult to treat once the disease has spread beyond the skin to other parts of the body such as by
through the lymphatic system (metastatic disease). Melanoma accounts for only one percent of skin cancer cases, but causes a
large majority of skin cancer deaths. The American Cancer Society estimates that in 2017, there will be 87,110 new cases of
melanoma in the U.S., and about 9,730 will die of this disease. Based on proprietary Idera research, the company anticipates
by the year 2025, there will be roughly 13,000 anti-PD-1 refractory metastatic melanoma patients.
About Idera Pharmaceuticals
Idera Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel nucleic acid-based therapies for the treatment
of certain cancers and rare diseases. Idera’s proprietary technology involves designing synthetic oligonucleotide-based drug
candidates to modulate the activity of specific TLRs. In addition to its TLR programs, Idera has used its proprietary knowledge to
create a third generation antisense technology platform which inhibits the production of disease-associated proteins by targeting
RNA. To learn more about Idera, visit www.iderapharma.com.
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 interim results from a clinical trial, such as preliminary results reported in
this release, will be predictive of the final results of the trial, whether results obtained in preclinical studies and clinical
trials will be indicative of the results that will be generated in future clinical trials, including in clinical trials in
different disease indications; whether products based on Idera's IMO-2125 will successfully advance through the clinical trial
process on a timely basis or at all 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; and
such other important factors as are set forth under the caption "Risk Factors" in the Company's Annual Report on form 10K for the
period ended December 31, 2016. 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.
Investor and Media Contact Robert Doody Vice President, Investor Relations and Corporate Communications Office: 617-679-5515 Mobile: 484‐639‐7235 rdoody@iderapharma.com
