On March 7, 2018 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the first patient has been dosed in a global phase 2 study evaluating the safety and efficacy of DS-8201, an investigational HER2-targeting antibody drug conjugate (ADC), in patients with HER2-expressing advanced colorectal cancer who have received at least two prior lines of standard treatment (Press release, Daiichi Sankyo, MAR 7, 2018, View Source [SID1234524505]).

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An increase in the number of approved targeted therapies for advanced colorectal cancer over the past decade has helped improve outcomes for some patients, however efficacy and tolerability of second and third-line treatments remain limited.[1], [2], [3], [4], [5]Approximately 3 percent of colorectal cancers overexpress the HER2 protein, which is a well-established therapeutic target in breast and gastric cancer.1 In addition, research indicates that HER2 amplification may be associated with resistance to anti-epidermal growth factor receptor (EGFR)-targeted therapy and shorter survival.[6],[7] Currently, no approved HER2-targeting therapies exist for patients with colorectal cancer.

"Given the existing unmet medical need for advanced colorectal cancer, we are exploring the smart delivery of chemotherapy with DS-8201 as a potential new type of targeted treatment for patients with HER2-expressing disease who have progressed on or become resistant to standard therapies," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "Similar to our breast and gastric cancer programs, we are pursuing a development path focused first on patients with HER2-overexpressing tumors followed by potential expansion to include patients with advanced colorectal cancer with lower levels of HER2 expression."

About the DS-8201 Colorectal Cancer Phase 2 Study

The global, multi-center, phase 2, open-label, three-cohort study will investigate the safety and efficacy of DS-8201 in patients with HER2-expressing advanced colorectal cancer. The first part of the study will enroll patients with HER2-positive (defined as IHC3+ or IHC2+/ISH+) advanced colorectal cancer. The primary endpoint of the study is overall response rate. Secondary endpoints include progression-free survival, overall survival, duration of response, disease control rate, pharmacokinetics and safety. Exploratory endpoints include time to response and biomarker analysis. This part of the study is expected to enroll approximately 50 patients in North America, Europe and Japan.

Following the outcome of the first part of the study, two additional exploratory cohorts may proceed to enroll patients whose tumors have lower levels of HER2-expression. For more information about the study, visit ClinicalTrials.gov.

About Colorectal Cancer

Colorectal cancer is the third most common cancer worldwide. In 2012, there were approximately 1.36 million new cases diagnosed and 690,000 deaths worldwide.[8] Approximately 25 percent of patients have metastatic disease at diagnosis, meaning the disease has spread to distant organs, and about 50 percent of patients with colorectal cancer will eventually develop metastases.[9] Prognosis for these patients remains poor.[10]

About DS-8201

DS-8201 is the lead product in the investigational ADC Franchise of the Daiichi Sankyo Cancer Enterprise. ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Designed using Daiichi Sankyo’s proprietary ADC technology, DS-8201 is a smart chemotherapy comprised of a humanized HER2 antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker. It is designed to target and deliver chemotherapy inside cancer cells and reduce systemic exposure to the cytotoxic payload (or chemotherapy) compared to the way chemotherapy is commonly delivered.

In addition to the phase 2 study in HER2-expressing advanced colorectal cancer, DS-8201 is currently in pivotal phase 2 clinical development for HER2-positive unresectable and/or metastatic breast cancer resistant or refractory to T-DM1 (DESTINY-Breast01) in North America, Europe and Asia, and pivotal phase 2 development for HER2-positive advanced gastric cancer resistant or refractory to trastuzumab (DESTINY-Gastric01) in Japan and South Korea. DS-8201 is also in phase 1 development for other HER2-expressing advanced/unresectable or metastatic solid tumors.

DS-8201 has been granted Breakthrough Therapy designation for the treatment of patients with HER2-positive, locally advanced or metastatic breast cancer who have been treated with trastuzumab and pertuzumab and have disease progression after ado-trastuzumab emtansine (T-DM1), and Fast Track designation for the treatment of HER2-positive unresectable and/or metastatic breast cancer in patients who have progressed after prior treatment with HER2-targeted therapies including T-DM1 by the U.S. Food and Drug Administration (FDA). DS-8201 is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

On March 7, 2018 The University of Texas MD Anderson Cancer Center and Berkeley Lights, Inc. reported the launch of Optera Therapeutics Corp, a biopharmaceutical company developing cell therapies with scalable manufacturing solutions for cancer (Press release, Optera Therapeutics, MAR 7, 2018, View Source [SID1234525317]).

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Cell-based immunotherapies where patients are treated with their own immune cells, such as chimeric antigen receptor (CAR-T) and T cell receptor (TCR) engineered T cells, tumor-infiltrating lymphocytes (TIL) and endogenous T cells (ETC), have demonstrated promise for treating cancer. Optera Therapeutics will develop cancer cell therapies discovered at MD Anderson and apply Berkeley Lights’ advanced cell therapy manufacturing systems with the goal of making these novel therapies accessible to all.

"MD Anderson is dedicated to improving the standard of care for our patients as we strive to realize our mission to end cancer," said Patrick Hwu, M.D., division head of Cancer Medicine. "Our hope is that – by combining our cell therapy research expertise with advanced automation capabilities – we will enhance our ability to deliver these treatments to every patient who needs them."

Optera Therapeutics is developing cell therapies under investigation at MD Anderson by leaders in the field of cellular immunology including Cassian Yee, M.D., professor of Melanoma Medical Oncology; Katy Rezvani, M.D., Ph.D., Chief, Section of Cellular Therapy at Department of Stem Cell Transplantation and Cellular Therapy; Elizabeth Shpall, M.D., professor of Stem Cell Transplantation and Cellular Therapy; Chantale Bernatchez, Ph.D., assistant professor of Melanoma Medical Oncology; Sattva Neelapu, M.D., professor of Lymphoma and Myeloma; and Greg Lizee, Ph.D., associate professor, Department of Melanoma Medical Oncology.

"Cell therapy is transforming the standard of care for cancer patients," said Yee. "Optera will capitalize on truly disruptive technology and allow us to extend our ability to treat more patients, for more cancers, in a shorter period of time."

Eric Hobbs, chief executive officer of Berkeley Lights, Inc., added, "The Berkeley Lights team is privileged and excited to join forces with our colleagues at MD Anderson who have devoted their lives to defeating cancer. We are absolutely driven to make their life-saving cell therapies accessible to all."

On March 7, 2018 Crinetics Pharmaceuticals, Inc., a rare disease therapeutics company focused on endocrine disorders and endocrine-related cancers, reported that Scott Struthers, Ph.D., founder and chief executive officer, will present at the Cowen and Company 38th Annual Health Care Conference on Wednesday, March 14, 2018 at 10:30 a.m. ET. The conference is being held from March 12-14, 2018 in Boston (Press release, Crinetics Pharmaceuticals, MAR 7, 2018, http://www.crinetics.com/crinetics-pharmaceuticals-to-present-at-cowen-and-company-38th-annual-health-care-conference/ [SID1234525094]).

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On March 7, 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 fourth quarter and year ended December 31, 2017 (Press release, Idera Pharmaceuticals, MAR 7, 2018, View Source [SID1234524648]).

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"Our company made tremendous progress throughout 2017 advancing our development programs and we expect the first half of 2018 to be marked by a number of key inflection points for each of these programs," stated Vincent Milano, Idera’s chief executive officer. "In 2018, we are continuing to enroll patients in the ILLUMINATE 204 Phase 2 trial of IMO-2125, with the next planned data update at ASCO (Free ASCO Whitepaper) and completion of enrollment expected by year end. We also recently initiated the Phase 3 ILLUMINATE 301 trial. For IMO-8400, we plan to report top-line data from our Phase 2 trial in dermatomyositis by the end of the 2nd quarter. We also intend to finalize our development plans for our lead nucleic acid chemistry research candidate, IDRA-008 shortly," continued Milano.

"In January, 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," Milano expressed.

Clinical Development Program Updates:
ILLUMINATE (IMO-2125) Clinical Development

ILLUMINATE 204 – Phase 1/2 trial of IMO-2125 in combination with ipilimumab or pembrolizumab in patients with PD-1 refractory metastatic melanoma:

As of March 7, 2018, enrolled 26 patients at 8 mg (RP2D) dose with ipilimumab, enrollment completion expected by year end 2018;
5 of the first 10 evaluable patients at the 8 mg dose of IMO-2125 were responders (50% Overall Response Rate [ORR]);
7 trial sites currently enrolling patients with goal of expansion to 10 sites during first half of 2018;
In pembrolizumab combination arm of the trial, phase 1 dose escalation continues into the last dosing cohort (32 mg); and
Next clinical data update expected at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June 2018.
ILLUMINATE 101 – Phase 1b trial of intratumoral IMO-2125 monotherapy in patients with refractory solid tumors:

Completed enrollment in first two cohorts (11 patients treated with 8 mg dose of IMO-2125, 8 patients treated with 16 mg dose of IMO-2125);
Two subjects in cohort 1 (8 mg) continue IMO-2125 monotherapy on the 101 study as of March 7, 2018; and
Enrollment of third cohort has commenced (8 patients to be treated with 23 mg dose of IMO-2125).
ILLUMINATE 301 – Randomized phase 3 trial of IMO-2125 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 (OS) as primary endpoints; and
U.S. Food and Drug Administration granted Fast Track Designation for IMO-2125 in combination with ipilimumab for treatment of PD-1 refractory metastatic melanoma in fourth quarter of 2017.
Pioneer (IMO-8400) Development Activities

PIONEER – 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 Q2 2018.
Nucleic Acid Chemistry Research Group

IDRA-008 Development Activities:

Selected apolipoprotein C-III (APOC-III) as first gene target for development for treatment of familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL);
Completion of pre-clinical toxicology and IND-enabling studies in Q1 2018;
Pre-clinical pharmacology study in cyno-model comparing IDRA-008 to competitive clinical development asset, volanesorsen expected to readout towards the latter part of Q1 2018;
Development decision for IDRA-008 expected during Q2 2018; and
The Company is continuing to evaluate rare-disease opportunities for application of its core nucleic acid chemistry research capability and expertise to yield innovative oligonucleotide therapeutic concepts that address significant unmet medical needs.
Financial Results
Fourth Quarter Results
Net loss applicable to common stockholders for the three months ended December 31, 2017 was $14.9 million, or $0.08 per basic and diluted share, compared to net income applicable to common stockholders of $0.8 million, or $0.01 per basic and diluted share, for the same period in 2016. Revenue in the fourth quarter of 2017 was nominal and primarily related to our collaboration with GSK. Revenue in the fourth quarter of 2016 was $15.3 million, primarily related to the agreement we entered into with Vivelix in November 2016 in which we received an upfront, non-refundable fee of $15 million. Research and development expenses for the three months ended December 31, 2017 totaled $10.4 million compared to $11.0 million for the same period in 2016. General and administrative expense for the three months ended December 31, 2017 totaled $4.8 million compared to $3.5 million for the same period in 2016.

Full Year Results
Net loss applicable to common stockholders for the year ended December 31, 2017 was $66.0 million or $0.42 per basic and diluted share, compared to net loss applicable to common stockholders of $38.4 million, or $0.30 per basic and diluted share, for the same period in 2016. Revenue for the year ended December 31, 2017 was $0.9 million compared to revenue of $16.2 million for the same period in 2016. Revenue in the 2017 period primarily related to our collaboration with GSK. Revenue in the 2016 period primarily related to collaborations with both GSK and Vivelix, including an upfront, non-refundable fee of $15 million received in connection with the Vivelix Agreement. Research and development expenses for the year ended December 31, 2017 totaled $50.7 million compared to $39.8 million for the same period in 2016. General and administrative expenses for the year ended December 31, 2017 totaled $16.7 million compared to $15.1 million for the same period in 2016.

As of December 31, 2017, our cash, cash equivalents and investments totaled $112.6 million compared to $109.0 million as of December 31, 2016. We currently anticipate that, based on our current operating plan and without taking into account the transaction with BioCryst, our existing cash, cash equivalents and investments will fund our operations into the second quarter of 2019.

Corporate Updates:

On January 22, 2018, BioCryst Pharmaceuticals, Inc. ("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 second quarter of 2018.

On March 7, 2018 -Bristol-Myers Squibb Company (NYSE: BMY)reported that it will present at Barclays Global Healthcare Conference on Wednesday, March 14, 2018, in Miami. Murdo Gordon, executive vice president and chief commercial officer, will answer questions about the company at 1:35 p.m. ET (Press release, Bristol-Myers Squibb, MAR 7, 2018, View Source [SID1234524638]).

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