On March 15, 2018 Selecta Biosciences, Inc. (NASDAQ:SELB), a clinical-stage biopharmaceutical company focused on unlocking the full potential of biologic therapies by avoiding unwanted immune responses, reported financial results for the fourth quarter and full year ended December 31, 2017 and provided a corporate update (Press release, Selecta Biosciences, MAR 15, 2018, View Source [SID1234524817]).

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"Several important achievements have been made over the course of the past several months, and we are excited to be in a strong position to execute on our 2018 milestones," said Werner Cautreels, Ph.D., President and CEO of Selecta. "We remain on track with the development of SEL-212 for chronic severe gout, with higher dose data from our Phase 2 trial to be presented in April and plans underway to initiate our Phase 3 program in 2018. We also recently announced that our next clinical candidate, SEL-403, has entered the clinic for the treatment of patients with mesothelioma at the National Cancer Institute. This also provides us with the opportunity to demonstrate for a second time the translation of our technology in the clinic. When coupled with the Board and management enhancements that were made over the course of the past year, we believe we have positioned Selecta for a momentous year in 2018."

Recent Business Highlights and Activities

Cohorts Added to SEL-212 Phase 2 Trial with Initial Data to be Reported in April 2018: As of March 9, 2018, a total of 111 patients had been dosed in Selecta’s ongoing Phase 2 trial of SEL-212 (SVP-Rapamycin in combination with pegsiticase) for the treatment of chronic severe gout. Selecta has now fully enrolled cohorts that are receiving three monthly doses of either 0.125 or 0.15 milligrams (mg) per kilogram (kg) of SVP-Rapamycin in combination with either 0.2 or 0.4 mg/kg of pegsiticase followed by two monthly doses of pegsiticase alone. The company plans to report further data from this ongoing trial at the upcoming Pan American League of Associations for Rheumatology (PANLAR) Congress on April 9th or 10th, 2018 and will host a conference call at 8:30 a.m. ET on the day of the presentation.

Initiated Dosing of SEL-212 Cohort Expected to Receive Five Combination Doses: In February 2018, Selecta began enrolling patients in the current Phase 2 trial who are expected to receive five monthly doses of SVP-Rapamycin in combination with pegsiticase. The patients will be receiving SVP-Rapamycin doses ranging from 0.1mg/kg-0.15mg/kg in combination with 0.2mg/kg of pegsiticase. The company expects to present data from these patients at a medical meeting in Q3 2018.

SEL-212 End of Phase 2 (EOP2) Meeting Planned for Mid-2018 and Plans for Phase 3 Program Initiation in 2018: The Selecta team is currently compiling the data package for an EOP2 meeting with the FDA, targeted for mid-2018, which will define the company’s design for the Phase 3 program. The team has also begun preparations for the Phase 3 program which the company plans to initiate in 2018.

SEL-403 Phase 1 Trial Initiated: On March 8, 2018, the first patient was dosed in a Phase 1 clinical trial of SEL-403, Selecta’s combination product candidate consisting of SVP-Rapamycin and LMB-100, for the treatment of patients with malignant pleural or peritoneal mesothelioma who have undergone at least one regimen of chemotherapy. LMB-100, which was in-licensed by Selecta in 2017, is a recombinant immunotoxin that targets mesothelin, a protein expressed in nearly all mesotheliomas and pancreatic adenocarcinomas, and a high percentage of other malignancies, including lung, breast and ovarian cancers. This open-label dose-escalation Phase 1 trial is being conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the National Institutes of Health, and is expected to enroll at least 18 patients. The trial will evaluate the safety and tolerability of this treatment and provide data on pharmacokinetics, anti-drug antibody (ADA) levels, as well as an objective response rate assessment.

Published SEL-403 Preclinical Data in January 2018:Proceedings of the National Academies of Sciences (PNAS) published a paper in January 2018 co-authored by the company and researchers from the NCI. The paper, entitled "Tolerogenic nanoparticles restore the anti-tumor activity of recombinant immunotoxins by mitigating immunogenicity," focuses on SEL-403 preclinical work conducted by Selecta and Dr. Ira Pastan’s lab at the NCI. Dr. Pastan is Senior Investigator, Head, Molecular Biology Section, at NCI’s Center for Cancer Research and a Fellow of the National Academy of Sciences.

Enhanced Leadership of the Company: Selecta announced the addition of two management team members in the fourth quarter of 2017: Chief Financial Officer and Head of Corporate Strategy John Leaman, M.D., and Chief Commercial Officer Stephen Smolinski. Dr. Leaman most recently served as Head of Corporate Development at InfaCare Pharmaceutical Corp., a specialty pharmaceutical company that was acquired by Mallinckrodt plc. Mr. Smolinski most recently served as Vice President and Head of Sanofi/Genzyme’s North American Rheumatology Business Unit. In early January, the company announced that Dr. Omid Farokhzad, a member of Selecta’s Board and a cofounder of the company, was appointed Chairman of the Board effective December 31, 2017.

Received Payment from Spark Therapeutics: In the fourth quarter of 2017, Selecta received a cash payment of $2.5 million under a license agreement, and proceeds from share purchases under a stock purchase agreement in the amount of $5.0 million ($7.5 million in the aggregate), bringing the total amount of proceeds received by Selecta from Spark Therapeutics to $30.0 million. These payments are associated with the December 2016 license and stock purchase agreements that provided Spark Therapeutics with exclusive worldwide rights to SVP-Rapamycin for co-administration with Spark’s gene therapy vectors for Hemophilia A and up to four additional pre-specified and undisclosed indications.
Fourth Quarter Financial Results:

Revenue: For the fourth quarter of 2017, the company’s total revenue was less than $0.1 million, which compares with $2.9 million for the fourth quarter of 2016. The decline is primarily the result of the termination of the Company’s collaboration with Sanofi, and reduced revenue recognized from the company’s nicotine vaccine candidate grant award from the National Institute on Drug Abuse.

Research and Development Expenses: Research and development expenses for the fourth quarter of 2017 were $13.6 million, which compares with $11.0 million for the fourth quarter of 2016. The increase is primarily the result of greater clinical costs related to the company’s Phase 2 trial of SEL-212, planning for the SEL-212 Phase 3 program and incremental headcount-related expenses.

General and Administrative Expenses: General and administrative expenses for the fourth quarter of 2017 were $5.7 million, which compares with $5.8 million for the fourth quarter of 2016. The decrease is primarily the result of greater headcount and related salaries needed to support a clinical-stage public company offset by a reduction in sublicensing payments made to the Massachusetts Institute of Technology resulting from the agreement with Spark Therapeutics.

Net Loss: For the fourth quarter of 2017, Selecta reported a net loss attributable to common stockholders of $(19.5) million, or $(0.88) per share, compared to a net loss of $(14.1) million, or $(0.77) per share, for the same period in 2016.

Cash Position: Selecta had $97.0 million in cash, cash equivalents, short-term deposits, investments and restricted cash as of December 31, 2017, which compares with a balance of $104.8 million at September 30, 2017. Selecta continues to expect that its cash, cash equivalents, short-term deposits, investments and restricted cash will be sufficient to fund the company’s operating expenses and capital expenditure requirements into mid-2019.
Conference Call Reminder
Selecta management will host a conference call at 8:30 a.m. ET today to provide a corporate update and review the company’s fourth quarter and year end 2017 financial results. Investors and the public can access a live and archived webcast of this call via the Investors & Media section of the company’s website, View Source Individuals may also participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10116990.

On March 15, 2018 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics targeting the underlying mechanisms of cancer, reported that preclinical data for CG’806, its pan-FLT3/pan-BTK inhibitor, and APTO-253, its c-Myc inhibitor, will be presented in three separate posters at the 2018 AACR (Free AACR Whitepaper) Annual Meeting in Chicago, Il (Press release, Aptose Biosciences, MAR 15, 2018, View Source;p=RssLanding&cat=news&id=2338208 [SID1234524790]).

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CG’806 Poster Presentation Details

CG’806, a first-in-class pan-FLT3/pan-BTK inhibitor, demonstrates superiority to other FLT3 and BTK inhibitors against primary patient samples
Date & Time: Sunday, April 15, 2018, 1:00 p.m. – 5:00 p.m.
Session Category: Experimental and Molecular Therapeutics
Session Title: Experimental Agents and Combinations for Hematologic Malignancies 1
Abstract Number: 4316
Location: McCormick Place South, Exhibit Hall A, Poster Section 37

CG’806, a first-in-class pan-FLT3/pan-BTK inhibitor, targets multiple pathways to kill diverse subtypes of acute myeloid leukemia and Bcell malignancy in vitro
Date & Time: Sunday, April 15, 2018, 1:00 p.m. – 5:00 p.m.
Session Title: Experimental Agents and Combinations for Hematologic Malignancies 1
Abstract Number: 4239
Location: McCormick Place South, Exhibit Hall A, Poster Section 37

APTO-253 Poster Presentation Details

APTO-253 is a new addition to the repertoire of drugs that can exploit DNA BRCA1/2 deficiency
Date & Time: Tuesday, April 17, 2018, 1:00 p.m. – 5:00 p.m.
Session Title: DNA Damage and Cell Cycle Regulation Experimental Therapeutics
Abstract Number: 2138
Location: McCormick Place South, Exhibit Hall A, Poster Section 38

All abstracts will be available on the AACR (Free AACR Whitepaper) website, www.aacr.org, and published in the 2018 Proceedings of the AACR (Free AACR Whitepaper).

About CG’806
CG‘806 is an oral, first-in-class pan-FLT3/pan-BTK multi-kinase inhibitor. This small molecule demonstrates potent inhibition of wild type and mutant forms of FLT3 (including internal tandem duplication, or ITD, and mutations of the receptor tyrosine kinase domain and gatekeeper region), eliminates acute myeloid leukemia (AML) tumors in the absence of toxicity in murine xenograft models, and represents a potential best-in-class therapeutic for patients with AML. Likewise, CG’806 demonstrates potent, non-covalent inhibition of the wild type and Cys481Ser mutant forms of the BTK enzyme, as well as other oncogenic kinase pathways operative in B cell malignancies, suggesting CG’806 may be developed for various B cell malignancy patients (including CLL, MCL, DLBCL and others) that are resistant/refractory/intolerant to covalent BTK inhibitors. CG’806 is currently in pre-clinical development in partnership with CrystalGenomics.

About APTO-253
APTO-253 is a clinical-stage small molecule targeted therapeutic agent that inhibits expression of the c-Myc oncogene, leading to cell cycle arrest and programmed cell death (apoptosis) in human-derived solid tumor and hematologic cancer cells, without causing general myelosuppression of the healthy bone marrow. The c-Myc oncogene is overexpressed in hematologic cancers, including acute myeloid leukemia (AML). Aptose researchers have reported the ability of APTO-253 to induce cell death, or apoptosis, in multiple blood cancer cell lines including AML, as well as in vitro synergy with various classes of conventional approved and investigational therapies for AML or myelodysplastic syndromes (MDS).

On March 15, 2018 Checkpoint Therapeutics, Inc. ("Checkpoint") (NASDAQ: CKPT), a
clinical-stage, immuno-oncology biopharmaceutical company focused on the acquisition, development
and commercialization of novel treatments for patients with solid tumor cancers, reported that
preclinical data on its BET inhibitor CK-103 (also known as TG-1601) will be presented in a poster session
at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting to be held April 14 – 18, 2018, in
Chicago (Press release, Checkpoint Therapeutics, MAR 15, 2018, View Source [SID1234525089]).

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Details on the poster presentation are as follows:

Title: TG-1601 is a novel BET inhibitor with strong binding affinity and long-lasting effect in preclinical
models
Poster Session: Experimental and Molecular Therapeutics / Canonical Targets 2
Abstract Number: 5790
Poster Number: 16
Date and Time: Wednesday, April 18, 2018, 8 a.m. – 12 p.m. CDT
Location: McCormick Place South, Exhibit Hall A, Poster Section 36

Following the presentation, the poster will be available on the Publications page of the Pipeline section of
Checkpoint’s website, www.checkpointtx.com.

Additional information on the meeting can be found on the AACR (Free AACR Whitepaper) website www.aacr.org.

On March 15, 2018 Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of genes, reported financial results for the fourth quarter and year ended December 31, 2017 and provided an update on recent accomplishments and planned upcoming events (Press release, Syros Pharmaceuticals, MAR 15, 2018, View Source [SID1234524818]).

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"2017 was an important year for Syros, marked by clinical and preclinical data for SY-1425 and SY-1365 that lay a clear path forward for the further development of both programs," said Nancy Simonian, M.D., Chief Executive Officer of Syros. "Additionally, our pioneering gene control platform continued to deliver, enabling us to expand our early-stage pipeline in cancer and monogenic diseases and enter into a collaboration with Incyte designed to allow us to benefit patients with diseases beyond our current areas of focus. We built on our strong foundation, adding to the leadership team and fortifying our cash position to fund our planned operations into 2020 and drive SY-1425 and SY-1365 to key value inflection points. As we enter 2018, we believe we are well-positioned to execute on our near-term and long-term goals to achieve our vision of becoming a fully integrated biopharmaceutical company with medicines that provide a profound and durable benefit for patients."

Upcoming Milestones

Syros plans to report clinical data in the fourth quarter of 2018 from a cohort in its ongoing Phase 2 trial evaluating SY-1425 in combination with azacitidine in RARA and IRF8 biomarker-positive newly diagnosed acute myeloid leukemia (AML) patients who are not suitable candidates for standard chemotherapy
.
Syros plans to report clinical data in the fourth quarter of 2018 from a pilot cohort in its ongoing Phase 2 trial evaluating SY-1425 in combination with daratumumab in RARA and IRF8 biomarker-positive relapsed or refractory AML and higher-risk myelodysplastic syndrome (MDS) patients.

·Syros plans to open expansion cohorts in mid-2018 in its ongoing Phase 1 trial of SY-1365 evaluating it as a single agent and in combination with carboplatin in multiple ovarian cancer patient populations. Based on emerging preclinical data showing anti-tumor activity of SY-1365 in hormone receptor-positive (HR-positive) breast cancer models, the Company announced today that it also plans to add an expansion cohort evaluating SY-1365 in combination with fulvestrant in HR-positive metastatic breast cancer patients who progress after treatment with a CDK4/6 inhibitor plus an aromatase inhibitor.

·Syros plans to report clinical data in the fourth quarter of 2018 from the dose escalation portion of its ongoing Phase 1 trial of SY-1365 in advanced solid tumor patients.

·Syros plans to select a new development candidate from its preclinical pipeline by the end of 2018.

Recent Platform and Pipeline Highlights

In January 2018, Syros announced that the U.S. Patent and Trademark Office issued two patents covering methods for stratifying patients with AML and MDS for treatment with SY-1425.

· In January 2018, Syros announced a clinical supply agreement with Janssen Research and Development. Under the terms of the agreement, Janssen is supplying daratumumab for the combination dosing cohort in biomarker-positive relapsed or refractory AML and higher-risk MDS patients in Syros’ ongoing Phase 2 trial of SY-1425.

In December 2017, Syros presented initial clinical data from its ongoing Phase 2 trial of SY-1425 in biomarker-positive patients with AML and MDS at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, showing biological and clinical activity as a single agent and supporting ongoing development of SY-1425 in combination with other therapies:

·Clinical activity was observed in 43% of evaluable relapsed or refractory AML and higher-risk MDS patients, including improvement in blood counts and reductions in bone marrow blasts.

· Myeloid differentiation was observed, including the induction of CD38 in 85% of evaluable patients.

·SY-1425 generally well-tolerated with chronic, daily dosing with the majority of adverse events being low grade.

· In December 2017, Syros presented new preclinical data on SY-1365 at ASH (Free ASH Whitepaper). The data showed anti-tumor activity in leukemia and lymphoma cell lines and in vivo models of AML. Additionally, the data pointed to a potential biomarker of response to SY-1365 and demonstrated synergistic activity with venetoclax, a BCL2 inhibitor, in preclinical AML models.

In December 2017, Syros presented new preclinical data on SY-1365 at the San Antonio Breast Cancer Symposium (SABCS). The data demonstrated anti-tumor activity across a broad panel of breast cancer cell lines and pointed to potential biomarkers of response. Syros also presented on its analysis of regulatory regions of the genome in cancer stem cell-enriched triple negative breast cancer (TNBC) cell lines, which revealed key genes that may be involved in driving disease relapse and metastasis in TNBC and suggest potential new targets for future drug discovery and development.

Recent Corporate Highlights

Syros reported the appointment of Joseph J. Ferra as Chief Financial Officer.

In January 2018, Syros announced the closing of an underwritten public offering of 4,816,753 shares of common stock at a public offering price of $9.55 per share, including the exercise in full by the underwriters of their option to purchase additional shares of common stock. Syros received aggregate gross proceeds ofapproximately $46 million, before deducting underwriting discounts and commissions and estimated offering expenses. In connection with the offering, Incyte Corporation, exercised its right to purchase shares of Syros common stock directly from the company at the public offering price, in a concurrent private placement, resulting in proceeds of approximately $1.4 million.

·In January 2018, Syros announced a global target discovery and validation collaboration with Incyte focused on myeloproliferative neoplasms (MPNs). Under the terms of the agreement, Syros will use its proprietary platform to identify novel therapeutic targets with a focus in MPNs. Incyte has options to obtain exclusive worldwide rights to intellectual property resulting from the collaboration for up to seven validated targets and, upon exercise of its options, will have exclusive worldwide rights to develop and commercialize any therapies under the collaboration that modulate those validated targets. Incyte paid Syros $10 million in upfront cash and purchased a total of $10 million in Syros common stock at a price of $12.61 per share. In addition, Syros could receive up to $54 million from Incyte in target validation and option exercise fees and up to $115 million in potential development, regulatory and commercial milestone payments per target for up to seven validated targets, plus low single-digit royalties on sales of products that result from the collaboration.

· In November 2017, Syros announced the appointment of Jeremy P. Springhorn, Ph.D., as Chief Business Officer.

Fourth Quarter 2017 Financial Results

Cash, cash equivalents and marketable securities as of December 31, 2017 were $72.0 million, compared with $83.6 million on December 31, 2016. Cash, cash equivalents and short-term investments as of December 31, 2017 do not include the aggregate gross proceeds of approximately $46 million from Syros’ underwritten public offering of common stock, which closed in February 2018, the $1.4 million in proceeds from the private placement of stock with Incyte concurrent with the public offering, or the $10 million upfront payment and purchase of $10 million in Syros common stock received in January 2018 in connection with entry into the collaboration with Incyte.

For the fourth quarter of 2017, Syros reported a net loss of $15.3 million, or $0.58 per share, compared to a net loss of $11.0 million, or $0.47 per share, for the same period in 2016. Stock-based compensation included in the net loss was $1.3 million for the fourth quarter of 2017, compared to $0.7 million for the same period in 2016.

Research and development (R&D) expenses were $11.8 million for the fourth quarter of 2017, as compared to $8.4 million for the same period in 2016. Stock-based compensation included in R&D expenses was $0.5 million for the fourth quarter of 2017, compared to $0.2 million for the same period in 2016.

General and administrative (G&A) expenses were $3.7 million for the fourth quarter of 2017, as compared to $2.9 million for the same period in 2016. Stock-based compensation included in G&A expenses was $0.8 million for the fourth quarter of 2017, compared to $0.5 million for the same period in 2016.

Full Year 2017 Financial Results

For the full year ended December 31, 2017, net loss was $54.0 million, or $2.13 per share, as compared to a net loss of $47.7 million, or $4.05 per share, for the same period in 2016.

Stock based compensation included in the net loss was $4.4 million for the year ended December 31, 2017, compared to $4.2 million for the same period in 2016.

R&D expenses were $41.9 million for the year ended December 31, 2017, as compared to $37.8 million for the same period in 2016. The increase was due to an increase in expenses from third parties that conduct research and development and preclinical activities on our behalf, including an increase in clinical development costs for SY-1425 and SY-1365, offset by a decrease in preclinical development work for SY-1365 as toxicology studies were completed and the Phase 1 clinical trial was initiated. Stock-based compensation included in R&D expenses was $1.7 million for the year ended December 31, 2017, compared to $3.0 million for the same period in 2016.

· G&A expenses were $13.9 million for the year ended December 31, 2017, as compared to $10.5 million for the same period in 2016. The increase was largely due to an increase in employee-related costs, including salary, benefits and stock-based compensation, as well as increased consulting, licensing, and professional fees to support the overall growth of the Company. Stock-based compensation included in G&A expenses was $2.7 million for the year ended December 31, 2017, compared to $1.2 million for the same period in 2016.

Financial Guidance

Based on its current plans, Syros believes that its cash, cash equivalents and short-term investments as of December 31, 2017, together with cash received in connection with entry into the collaboration with Incyte and the underwritten public offering and concurrent private placement of common stock that closed in February 2018, will be sufficient to enable it to fund its planned operating expense and capital expenditure requirements into 2020.

On March 15, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that pre-clinical and clinical data on the company’s pipeline of drug candidates will be presented at the 2018 AACR (Free AACR Whitepaper) Annual Meeting taking place in Chicago from April 14-April 18 (Press release, ArQule, MAR 15, 2018, View Source [SID1234524791]). Data will be presented in one oral and nine poster sessions on trials conducted by ArQule and its collaborators for BTK inhibitor, ARQ 531, AKT inhibitors, miransertib and ARQ 751, as well as FGFR inhibitor, derazantinib.

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The oral presentation will highlight data from a phase 1b trial for miransertib in combination with anastrozole in PIK3CA or AKT1-mutant endometrial and ovarian cancers conducted by Dr. David M. Hyman and colleagues at Memorial Sloan Kettering.

Oral Presentation Details

April 15, 2018

Title:A phase 1b study of Miransertib (ARQ 092) in combination with anastrozole in patients with PIK3CA or AKT1-mutant ER+ endometrial and ovarian cancer
Time: 3:00 – 5:00 p.m. CT
Location:Room N427 – McCormick Place North, Level 4
Sponsor: Memorial Sloan Kettering, New York, NY

Poster Presentation Details

April 15, 2018

Title:ARQ 531, a Novel and Reversible Inhibitor of Bruton’s Tyrosine Kinase, Displays Favorable Oral Bioavailability and Exposure in patients with B-cell malignancies
Time: 1:00 – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 43, Poster Board 18
Sponsor:ArQule, Inc.

Title:The novel Bruton’s tyrosine kinase inhibitor ARQ 531 disrupts survival signaling and triggers apoptosis in AML cells
Time: 1:00 – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 37, Poster Board 3
Sponsor:University of Genoa, Genova, Italy

Title:Results of A Phase 1 Dose Escalation Study of ARQ 751 in Adult Subjects with Advanced Solid Tumors with AKT1, 2, 3 Genetic Alterations, Activating PI3K Mutations, PTEN-null, or other known actionable PTEN mutations
Time: 1:00 – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 42, Poster Board 17
Sponsor:MD Anderson Cancer Center, Houston, TX

April 16, 2018

Title:ARQ 531, a potent reversible BTK inhibitor exhibits potent antitumor activity in ibrutinib resistant diffuse large B-cell lymphoma
Time:8:00 a.m. – 12:00 p.m. CT
Location: Exhibit Hall A, Poster Section 41, Poster Board 8
Sponsor:ArQule, Inc.

Title:Preclinical Evaluation of the Tyrosine Kinase Inhibitor ARQ531 in AML
Time:8:00 a.m. – 12:00 p.m. CT
Location: Exhibit Hall A, Poster Section 38, Poster Board 13
Sponsor: The Ohio State University, Columbus OH

Title: In vivo combination of Miransertib (ARQ 092) with anti-PD-1 antibody, Trametinib, Lapatinib, Trastuzumab and Paclitaxel
Time:1:00 p.m. – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 41, Poster Board 23
Sponsor:ArQule, Inc.

April 17, 2018

Title:Derazantinib (ARQ 087) Pharmacodynamics: Alterations in FGF19/21/23 and phosphate in Patients with Cholangiocarcinoma
Time:8:00 a.m. – 12:00 p.m. CT
Location: Exhibit Hall A, Poster Section 43, Poster Board 22
Sponsor:ArQule, Inc.

Title:In vitro and in vivo effect of ARQ 531 on Trk family kinases
Time:1:00 p.m. – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 36, Poster Board 26
Sponsor:ArQule, Inc.

Title:Combinations of imatinib mesylate with AKT inhibitor (Miransertib, ARQ 751) or FGFR inhibitor (Derazantinib) show synergy in GIST cell lines and pre-clinical models
Time:1:00 p.m. – 5:00 p.m. CT
Location: Exhibit Hall A, Poster Section 37, Poster Board 7
Sponsor:Fox Chase Cancer Center, Philadelphia, PA

About Miransertib and ARQ 751

Miransertib (ARQ 092) and ARQ 751 are orally bioavailable, selective small molecule inhibitors of the AKT serine/threonine kinase. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications.

Miransertib, the lead compound in ArQule’s AKT program, has completed phase 1a clinical testing and has advanced into phase 1b expansion testing in cohorts of patients with endometrial cancer, lymphomas and tumors harboring either AKT or PI3K mutations. A company sponsored phase 1/2 trial is being conducted in the U.S. and E.U. for Overgrowth Diseases, including PROS and Proteus syndrome. Miransertib is also in a phase 1 trial being conducted by the NIH for Proteus syndrome.

About ARQ 531

ARQ 531 is an investigational, orally bioavailable, potent and reversible Bruton’s tyrosine kinase (BTK) inhibitor. Biochemical and cellular studies have shown that ARQ 531 inhibits both the wild type and C481S-mutant forms of BTK. The C481S mutation is a known emerging resistance mechanism for first generation irreversible BTK inhibitors. In preclinical studies ARQ 531 has demonstrated high oral bioavailability as well as good ADME, pharmacokinetic and metabolic properties. The Company initiated a phase 1 trial in the third quarter of 2017. BTK is a therapeutic target that has been clinically proven to inhibit B-cell receptor signaling in blood cancers.

About Derazantinib

Derazantinib is a potent, orally administered inhibitor of the fibroblast growth factor receptor (FGFR) family, a key driver of cell proliferation, differentiation, and migration. In a Phase 1/2 study in patients with iCCA harboring FGFR2 gene fusions, treatment with derazantinib resulted in an objective response rate of 21%, nearly 3 times higher than standard-of-care chemotherapy. ArQule is currently conducting a registrational study with derazantinib in patients with FGFR2 fusion-positive second-line iCCA. The open-label single-arm trial is recruiting in both the United States and Europe with objective response rate as the primary endpoint.