On May 9, 2018 Idera Pharmaceuticals, Inc. ("Idera") (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, reported its financial and operational results for the first quarter ended March 31, 2018 (Press release, Idera Pharmaceuticals, MAY 9, 2018, View Source [SID1234526315]).
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"Our company continues to make significant progress advancing our two lead TLR modulating clinical development candidates, tilsotolimod and IMO-8400," stated Vincent Milano, Idera’s chief executive officer. Milano continued, "As we now advance through the second quarter, we are continuing to enroll patients in the ILLUMINATE oncology trials, with the next planned data from ILLUMINATE-204 in PD-1 refractory metastatic melanoma to be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) ("ASCO") Annual Meeting and completion of enrollment expected by year end. For IMO-8400, we plan to report top-line data from our Phase 2 trial in dermatomyositis in June. As it pertains to our nucleic acid chemistry research group, we completed our data analysis for IDRA-008 which has led us to a decision to not advance that program into the clinic."
"In January of this year, we announced our proposed merger with BioCryst Pharmaceuticals, Inc. that we believe will build greater and more sustainable value for the benefit of stockholders as well as patients with rare diseases beyond what we could achieve alone. The Idera Board determined this combination was compelling from both a strategic and financial perspective following a careful evaluation of a range of strategies to enhance long-term stockholder value. The transaction will create a leading rare disease company with a robust pipeline including two promising Phase 3 rare disease programs and combines synergistic discovery engines that will not only expand the number of rare diseases we can target but create meaningful opportunities for differentiation in the market through joint small molecule and oligo treatments. Importantly, joining with BioCryst will also enable us to achieve cost synergies and increase our financial strength and flexibility. Subject to shareholder approval, we expect to close the transaction in the third quarter," Milano expressed.
Clinical Development Program Updates:
TLR Modulation Technology Development Candidates
ILLUMINATE (tilsotolimod) Clinical Development
ILLUMINATE 301 – Randomized phase 3 trial of tilsotolimod in combination with ipilimumab versus ipilimumab alone in patients with PD-1 refractory metastatic melanoma:
Trial initiated in Q1 2018;
Approximately 80 sites planned for trial participation across 12 countries;
Planned enrollment of approximately 300 patients with Overall Response Rate ("ORR") and Overall Survival as primary endpoints; and
U.S. Food and Drug Administration granted Fast Track Designation for tilsotolimod in combination with ipilimumab for treatment of PD-1 refractory metastatic melanoma in fourth quarter of 2017.
ILLUMINATE 204 – Phase 1/2 trial of intratumoral tilsotolimod in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:
Ipilimumab Combination Arm – Phase 2 Expansion Ongoing at RP2D of 8mg
Enrollment (60 patients) completion expected by year end 2018;
5 of the first 10 evaluable patients at the 8 mg dose of tilsotolimod were responders (50% ORR);
Additional data from the Phase 2 expansion of ILLUMINATE-204 selected for presentation at upcoming ASCO (Free ASCO Whitepaper) meeting in Chicago, IL.
Melanoma/Skin Cancers poster discussion session on June 4, 2018 at 4:45 PM CT; and
Investor/Analyst Event featuring lead ILLUMINATE-204 investigator, Adi Diab, MD from the University of Texas, MD Anderson Cancer Center to be held at 6:30 PM CT, also on June 4, 2018. As a convenience to those unable to attend, this event will be webcast.
Pembrolizumab Combination Arm – Phase 1 Dose Escalation Ongoing
Enrollment into the last dosing cohort (32 mg) ongoing;
The previously reported partial response (PR) in 1 of the first 6 patients in the 16 mg cohort of intratumoral tilsotolimod in combination with pembrolizumab has evolved into a confirmed complete response (CR).
ILLUMINATE 101 – Phase 1b trial of intratumoral tilsotolimod monotherapy in patients with refractory solid tumors:
Completed enrollment in first two cohorts (11 patients treated with 8 mg dose of tilsotolimod, 8 patients treated with 16 mg dose of tilsotolimod);
Two patients in cohort 1 (8 mg) continue in follow-up; 2 patients in cohort 2 (16 mg) continue tilsotolimod monotherapy and two patients continue in follow-up; and
6 of 8 planned patients for cohort 3 (23 mg) enrolled.
(IMO-8400) Development Activities
PIONEER-211 – Randomized placebo controlled Phase 2 trial of IMO-8400 in adult patients with dermatomyositis:
Enrollment concluded during Q3 2017 (30 patients); and
Topline phase 2 trial data expected in June 2018.
Nucleic Acid Chemistry Research Group
We are developing our nucleic acid chemistry technology to "turn off" the mRNA associated with disease causing genes. Our focus is on creating candidates targeted to specific genes to treat cancer and rare diseases.
We had selected IDRA-008 as our first nucleic acid chemistry research program candidate. IDRA-008 targets the Apolipoprotein C-III (APOC-III) gene and was being developed for the treatment of Familial Chylomicronemia Syndrome (FCS) and Familial Partial Lipodystrophy (FPL) which had available pre-clinical animal models and well-known clinical endpoints.
During the first quarter of 2018, we completed our pre-clinical analysis for IDRA-008 and based upon the outcome of pre-clinical pharmacology studies, including a comparative pharmacology study with the competitive development asset volanesorsen, and IND-enabling safety evaluation, we made a data-driven decision to not advance IDRA-008 into clinical development.
We are currently conducting analysis throughout our research portfolio to identify other candidates for future clinical development based on our nucleic acid technology expertise and potential strategic commercial opportunity.
Financial Results
First Quarter Results
Net loss applicable to common stockholders for the three months ended March 31, 2018 was $20.1 million, or $0.10 per basic and diluted share, compared to net loss applicable to common stockholders of $15.1 million, or $0.10 per basic and diluted share, for the same period in 2017. Revenue in each of the three months ended March 31, 2018 and 2017 was nominal. Research and development expenses for the three months ended March 31, 2018 totaled $13.6 million compared to $11.5 million for the same period in 2017. General and administrative expense for the three months ended March 31, 2018 totaled $7.0 million compared to $4.1 million for the same period in 2017.
During the three months ended March 31, 2018, holders of warrants, including Baker Brothers, exercised warrants to purchase shares of the Idera’s common stock which generated $9.6 million in cash proceeds. As of March 31, 2018, our cash and cash equivalents totaled $107.5 million compared to $112.6 million as of December 31, 2017. We currently anticipate that, based on our current operating plan and without taking into account the transaction with BioCryst Pharmaceuticals, Inc. ("BioCryst"), our existing cash, cash equivalents and investments will fund our operations into the third quarter of 2019.
Corporate Updates:
On January 22, 2018, BioCryst and Idera jointly announced the signing of a definitive merger agreement to create a company focused on the development and commercialization of medicines to serve patients suffering from rare diseases. The combined company will be renamed upon closing, and will be led by Vincent Milano, the current chief executive officer of Idera. Jon Stonehouse, the current chief executive officer of BioCryst, will serve as a member of the Board of Directors. The transaction is subject to approval by the stockholders of both companies, as well as the satisfaction of customary closing conditions. The transaction is expected to be completed by the end of the third quarter of 2018.