CAMBRIDGE, Mass. and EXTON, Pa., Oct. 28, 2016 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (NASDAQ:IDRA), a
clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel nucleic acid-based
therapeutics for oncology and rare diseases, today reported its financial and operational results for the third quarter ended
September 30, 2016.
Since June 30, 2016, the Company:
- Announced positive clinical data from the initial cohorts of the phase 1 dose escalation portion of the Company’s ongoing
Phase 1/2 clinical trial of intratumoral IMO-2125 in combination with ipilimumab in patients with PD-1 refractory metastatic
melanoma;
- Presented pre-clinical data updates on both novel mechanism of action and selective targeting of single point mutations with
3rd Generation Antisense (3GA) at the Cold Springs Harbor Laboratory Conference on Regulatory & Non-Coding RNAs
conference and the Annual Meeting of the Oligonucleotide Therapeutic Society, respectively;
- Received acceptance of an abstract entitled “Reactivating the Anti-tumor Immune Response by Targeting Innate and Adaptive
Immunity in a Phase I/II Study of intratumoral IMO-2125 in Combination with Systemic Ipilimumab in Patients with Anti-PD-1
Refractory Metastatic Melanoma” for an oral presentation at the upcoming Society for Immunotherapy of Cancer (SITC) Annual
Meeting in November 2016;
- Received acceptance of an oral presentation entitled “IMO-2125, An Investigational intratumoral Toll-Like Receptor 9 Agonist,
Modulates the Tumor Microenvironment to Enhance Anti-Tumor Immunity” at SITC Annual Meeting;
- Generated estimated net proceeds of $48.9M, after deducting underwriters’ discounts and commissions and estimated offering
expenses, from a public offering of common stock, including from the partial exercise by the underwriters of their option to
purchase additional shares in the offering, which option exercise is expected to close today. The Company believes that,
based on its current operating plan, its existing cash, cash equivalents and investments, including the net proceeds from the
offering, will enable it to fund its operations into the first quarter of 2018 and continue acceleration and development of key
research and clinical development programs; and
- Announced increased prioritization of IMO-2125 with plans to initiate two additional multi-center clinical trials in 2017
both as monotherapy and in combination with check-point inhibitors in multiple tumor types.
“The third quarter of 2016 was a very productive period for our team at Idera and positions us well to close out
the year very strong and carry that momentum into a catalyst rich 2017,” stated Vincent Milano, Idera’s Chief Executive
Officer “As I noted in late September, we are incredibly energized by the IMO-2125 data we announced in such an early phase of
the development and are now mobilized to advance this program rapidly, to potentially alter the lives of other patients who have
exhausted all other good options.”
Continued Milano, “I am also proud of the conduct of our team to complete all the work that was necessary to inform
and enable our recent decision to suspend work on the B-cell program, which, while difficult, allows us to redirect additional
resources to accelerate the development of IMO-2125. We remain excited for the prospects of dermatomyositis with IMO-8400 and we
are looking forward to being in a position to go into greater detail in January on our plans to begin the clinical phases of
development with the 3GA platform.”
Research and Development Program Updates
IMO-2125 and IMO-8400 are the Company’s lead clinical development drug candidates. IMO-2125 is an
oligonucleotide-based agonist of Toll-like receptor (TLR) 9. IMO-8400 is an oligonucleotide-based antagonist of TLRs 7, 8,
and 9. The Company also announced, in late 2015, the first two potential development targets from its proprietary 3GA
technology platform: NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4).
The Company continues to evaluate these and other potential targets. The Company plans to take the first 3GA candidate into
human proof of concept studies in 2017.
Toll-like Receptor (TLR) Agonism
Immuno-Oncology Program
Idera’s development program in immuno-oncology is based on the rationale that intra-tumoral injections of IMO-2125, a TLR9 agonist,
will activate dendritic cells and modulate the tumor microenvironment to potentiate the anti-tumor activity of checkpoint
inhibitors and other immunotherapies. This rationale is supported by pre-clinical data in multiple tumor types. These
pre-clinical studies led Idera into a strategic alliance with the University of Texas MD Anderson Cancer Center to evaluate the
combination of intratumoral IMO-2125 with checkpoint inhibitors.
In late 2015, Idera announced the initiation of a Phase 1/2 clinical trial of intratumoral IMO-2125 in combination
with ipilimumab, a CTLA4 antibody, which is being conducted at the University of Texas MD Anderson Cancer Center. This trial
is being conducted in patients with relapsed or refractory metastatic melanoma who have failed prior PD-1 therapy. In September
2016, the Company announced positive preliminary clinical data from the initial dosing cohorts in the ipilimumab arm of the dose
escalation portion of the trial. The trial has recently been amended to also include the evaluation of the combination of
intratumoral IMO-2125 with pembrolizumab, an anti-PD1 antibody, with enrollment in this arm now underway. The Company plans
to expand the trial to additional clinical trial sites to conduct the phase 2 portion of the trial.
The results announced are summarized as follows:
Safety
- 10 patients in 3 dosing cohorts (4mg, 8mg and 16mg) were dosed and assessable for safety, as of the September 19, 2016 cutoff
date;
- IMO-2125 in combination with ipilimumab was being generally well tolerated at all 3 dose levels;
- Immune related adverse events have been observed in 3 subjects: 2 responding patients have experienced hypophysitis and 1
patient has discontinued the study due to a recurrence of immune related hepatitis previously observed on pre-study therapy with
ipilimumab;
- No dose limiting toxicities (DLTs) were identified and the study is currently enrolling at the highest (32mg) dosing cohort
in combination with ipilimumab.
Clinical activity
- 6 patients treated in the first two dosing cohorts (4mg and 8mg) were assessable for initial clinical activity, as of the
September 19, 2016 cutoff date;
- 3 of the 4 patients with cutaneous melanoma were investigator-assessed responders with one Complete Response (CR) and 2
Partial Responses (PR).
Translational observations
- Translational data seen through the first two dosing cohorts (4mg and 8mg) were promising relative to the induction of immune
responses and consistent with the underlying hypothesis of the mechanism of action;
- Detailed information on the translational findings from biopsies taken in the first two dosing cohorts and the relationship
of these to clinical response is the subject of an accepted oral presentation on November 11, 2016 at the 2016 Society for
Immunotherapy of Cancer (SITC) Annual Meeting by Cara Haymaker, Ph.D., University of Texas, MD Anderson Cancer Center.
The Company also announced plans to take another data cut at the end of 2016 and request an End of Phase 1 (EOP)
meeting with the U.S. Food and Drug Administration to discuss next steps and the path to a regulatory filing. The Company
also anticipates requesting a meeting with the European Medicines Agency (EMA) for scientific advice.
Idera also plans to initiate trials to explore IMO-2125 as a monotherapy in multiple tumor types as well as a Phase
2 basket study of IMO-2125 in combination with check point inhibitors in additional tumor types beyond melanoma. Both of
these studies are planned to initiate in 2017.
Toll-like Receptor (TLR) Antagonism
Dermatomyositis Clinical Development Program
In late 2015, Idera announced the initiation of a Phase 2 clinical trial of IMO-8400 in patients with dermatomyositis, a rare
auto-immune condition, which negatively affects skin and may result in debilitating muscle weakness. TLRs have been reported to
play a role in the pathogenesis of the disease. This randomized, double-blind, placebo controlled Phase 2 trial is expected
to enroll 36 patients will be conducted at approximately 22 clinical sites worldwide. The Company plans to complete
enrollment of this trial by the end of 2017 and have clinical data available in early 2018.
B-cell Lymphoma Clinical Development Program
In September 2016, Idera announced that the company had suspended the clinical development of IMO-8400 for B-cell lymphomas,
including studies in Waldenstroms Macroglobulinemia (WM) and Diffuse Large B-Cell Lymphoma (DLBCL), and planned to explore
strategic options in these indications. This decision was based upon the prioritization of the clinical development plans for
IMO-2125 and the Company’s assessment that the level of clinical activity seen in the WM trial does not support monotherapy, the
very slow enrollment rate in DLBCL and the Company’s commercial assessment of IMO-8400. IMO-8400 was generally well tolerated
at all dose levels evaluated in the studies.
Third Generation Antisense Platform (3GA)
Idera’s proprietary third-generation antisense (3GA) platform technology is focused on silencing the mRNA associated with disease
causing genes. Idera has designed 3GA oligonucleotides to overcome specific challenges associated with earlier generation
antisense technologies and RNAi technologies.
In late 2015, Idera announced the identification of NLRP3 (NOD-like receptor family, pyrin domain containing
protein 3) and DUX4 (Double Homeobox 4) as initial gene targets to advance into IND-enabling activities, which will occur
throughout 2016. Potential disease indications related to these targets include, but are not limited to, interstitial
cystitis, lupus nephritis, uveitis and facioscapulohumeral muscular dystrophy (FSHD). Over the first three quarters of 2016, Idera
has generated additional 3GA compounds for a series of additional gene targets, which join NLRP3 and DUX4 as potential gene targets
for the first clinical development program. The Company is currently conducting clinical, regulatory and commercial
analysis activities and conducting IND-enabling studies. The Company plans to enter the clinic in 2017 for the first clinical
development program. These additional compounds will enable the Company to continue to expand its potential future pipeline
opportunities for both internal development as well as partnerships in areas outside of Idera’s focus.
In August 2016, Idera presented new pre-clinical data demonstrating the novel mechanism of action of the 3GA
platform at the Cold Springs Harbor Laboratory Conference on Regulatory & Non-Coding RNAs. Subsequently, Idera presented in
September 2016, new pre-clinical data demonstrating how the 3GA platforms unique mechanism of action supports selective targeting
of single point mutations as well as a pre-clinical data presentation of 3GA targeting of NLRP3 for the treatment of inflammatory
disorders. These presentations were made at the 12th Annual Meeting of the Oligonucleotide Therapeutics Society
(OTS).
In late 2015, Idera entered into a collaboration and license agreement with GSK to research, develop and
commercialize compounds from its 3GA technology for the treatment of undisclosed, selected renal targets. As per the terms of
the agreement, Idera received an upfront payment of $2.5 million and is eligible to receive up to approximately $100 million in
milestone payments, including the $2.5 million payment, in addition to royalties.
Financial Results
Third Quarter 2016 Results
Net loss for the three months ended September 30, 2016 was $12.9 million, or $(0.10) per basic and diluted share,
compared to a net loss of $11.4 million, or $(0.10) per basic and diluted share, for the same period in 2015. Revenue totaled $0.3
million and $0.9 million during the three and nine months ended September 30, 2016, respectively. There was nominal revenue
recognized during the corresponding 2015 periods. For the nine month period ended September 30, 2016, the Company’s net loss was
$39.2 million, or $(0.32) per basic and diluted share, compared to a net loss of $36.6 million, or $(0.32) per diluted share, for
the same period in 2015.
Research and development expenses for the three months ended September 30, 2016 totaled $9.4 million compared to
$7.5 million for the same period in 2015. For the nine month period ended September 30, 2016, research and development expenses
totaled $28.8 million compared to $25.1 million for the same period in 2015.
General and administrative expense for the three months ended September 30, 2016 totaled $3.9 million compared to
$4.0 million for the same period in 2015. For the nine month period ended September 30, 2016, general and administrative expenses
totaled $11.6 million compared to $11.7 million for the same period in 2015.
As of September 30, 2016, Idera’s cash, cash equivalents and investments totaled $53.4 million compared to $87.2
million as of December 31, 2015.
In October 2016, the Company completed a public offering of its common stock, generating estimated net proceeds of
$48.9M, after deducting underwriters’ discounts and commissions and estimated offering expenses, including from the partial
exercise by the underwriters of their option to purchase additional shares in the offering, which option exercise is expected to
close today. The Company believes that, based on its current operating plan, its existing cash, cash equivalents and
investments, including the net proceeds from the offering, will enable it to fund its operations into the first quarter of 2018 and
continue acceleration and development of key research and clinical development programs.
About Idera Pharmaceuticals, Inc.
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 using a TLR-targeting technology,
to design synthetic oligonucleotide-based drug candidates to act by modulating the activity of specific TLRs. In addition to its
TLR programs, Idera has created a third generation antisense technology platform using its proprietary technology to inhibit 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,
clinical trials, 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 the Company’s cash resources
will be sufficient to fund the Company’s continuing operations and the further development of the Company’s programs for the period
anticipated; whether interim results from a clinical trial, such as the 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 such as the
results described in this release 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 technology will advance into or through the
clinical trial process when anticipated or at all or warrant submission for regulatory approval; whether such products will receive
approval from the U.S. 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 collaborations will be successful; and
such other important factors as are set forth under the caption "Risk factors" in the Company’s prospectus supplement filed on
October 7, 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.
|
Idera Pharmaceuticals, Inc.
Condensed Statements of Operations - Unaudited
(In thousands, except per share data)
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended |
|
|
Nine Months
Ended |
|
|
|
September
30, |
|
|
September
30, |
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
|
|
|
Alliance Revenue |
$ |
323 |
|
|
$ |
20 |
|
|
|
$ |
918 |
|
|
$ |
59 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating Expenses |
|
|
|
|
|
|
|
|
|
|
Research & Development |
|
9,393 |
|
|
|
7,454 |
|
|
|
|
28,817 |
|
|
|
25,134 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
General & Administrative |
|
3,907 |
|
|
|
4,030 |
|
|
|
|
11,601 |
|
|
|
11,688 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Total Operating Expenses |
|
13,300 |
|
|
|
11,484 |
|
|
|
|
40,418 |
|
|
|
36,822 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from Operations |
|
(12,977 |
) |
|
|
(11,464 |
) |
|
|
|
(39,500 |
) |
|
|
(36,763 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Other Income (Expense), Net |
|
74 |
|
|
|
99 |
|
|
|
|
289 |
|
|
|
198 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net Loss |
$ |
(12,903 |
) |
|
$ |
(11,365 |
) |
|
|
$ |
(39,211 |
) |
|
$ |
(36,565 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net loss per common share applicable to common stockholders |
$ |
(0.10 |
) |
|
$ |
(0.10 |
) |
|
|
$ |
(0.32 |
) |
|
$ |
(0.32 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Shares used in computing basic and diluted net loss per common share applicable to
common stockholders |
|
121,389 |
|
|
|
118,248 |
|
|
|
|
121,332 |
|
|
|
113,821 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Idera Pharmaceuticals, Inc.
Condensed Balance Sheet Data
(In thousands)
|
|
|
|
|
|
|
|
|
|
|
|
At September
30, |
|
At December
31, |
|
|
|
|
|
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
|
|
|
|
|
(Unaudited) |
|
|
|
|
|
|
|
|
|
Cash, Cash Equivalents & Investments |
$ |
53,418 |
|
|
$ |
87,157 |
|
|
|
|
|
|
|
|
Other Assets |
|
5,060 |
|
|
|
5,119 |
|
|
|
|
|
|
|
|
Total Assets |
$ |
58,478 |
|
|
$ |
92,276 |
|
|
|
|
|
|
|
|
Total Liabilities |
$ |
8,606 |
|
|
$ |
8,694 |
|
|
|
|
|
|
|
|
Total Stockholders' Equity |
|
49,872 |
|
|
|
83,582 |
|
|
|
|
|
|
|
|
Total Liabilities & Stockholders' Equity |
$ |
58,478 |
|
|
$ |
92,276 |
|
|
|
|
|
|
|
IDERA PHARMACEUTICALS Contact: Robert A. Doody, Jr. VP, Investor Relations & Communications Phone (617) 679-5515 RDOODY@IDERAPHARMA.COM