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ArQule to Present Data at the 2018 American Association for Cancer Research (AACR) Annual Meeting

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, , MAR 15, 2018, View Source [SID1234524785]). 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.

Novelion Therapeutics Reports Fourth Quarter and Full Year
2017 Financial Results

On March 15, 2018 Novelion Therapeutics Inc. (NASDAQ: NVLN), a biopharmaceutical company dedicated to developing and commercializing therapies for individuals living with rare diseases ("Novelion" or the "Company"), reported financial results for the fourth quarter and full year ended December 31, 2017 and provided an overview of recent business activities (Press release, QLT, MAR 15, 2018, View Source [SID1234524839]).

Chief Operating Officer Jeff Hackman said, "We are focused on executing near-term plans that we believe will position our company for sustainable future growth. These priorities include cost control and expense management, reviewing our holding and capital structure with a view toward optimizing our assets for shareholders, advancing the metreleptin development program, and continuing to provide important therapies to our patients."

Business Update

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JUXTAPID: Novelion reported net revenues of JUXTAPID of $20.1 million in the fourth quarter of 2017, $14.2 million, or 71%, of which were from prescriptions written in the U.S.

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MYALEPT: Novelion reported net revenues of MYALEPT of $18.8 million in the fourth quarter of 2017, $13.3 million, or 71%, of which were from prescriptions written in the U.S.

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Novelion reported total consolidated net revenues of $138.4 million for the year ended December 31, 2017.

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Novelion ended 2017 with $55.4 million in unrestricted cash, compared with $70.5 million at the end of the third quarter of 2017.

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As announced separately today, subsidiary Aegerion Pharmaceuticals entered into a new secured financing facility with affiliates of Sarissa Capital Management and Broadfin Capital LLC providing for a $20 million term loan to Aegerion, strengthening Aegerion’s balance sheet and liquidity, and positioning the Company for ongoing capital structure review.

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In January 2018, Novelion undertook significant cost reduction plans as it continues to manage its limited cash resources.

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With respect to its European application to register metreleptin for marketing authorization, after taking into account the results of an oral hearing of the European Medicines Agency’s Committee for Medicinal Products for Human Use ("CHMP"), which occurred in February

2018, the Company expects the opinion of the CHMP in the second quarter of 2018 and the European Commission’s approval decision in mid-2018.

Fourth Quarter 2017 Financial Results

On November 29, 2016, the Company completed its acquisition of Aegerion Pharmaceuticals, Inc. ("Aegerion"). The acquisition has been accounted for as a business combination in which Novelion was considered the acquirer of Aegerion. As such, under U.S. Generally Accepted Accounting Principles ("GAAP"), the financial statements of Novelion are treated as the historical financial statements of the consolidated companies, with the results of Aegerion being included from November 29, 2016. This release also includes pro forma adjusted non-GAAP financial information showing pro forma results of operations of Novelion as if the acquisition had occurred on January 1, 2016. Reconciliation of the financial results on a GAAP versus non-GAAP basis are provided below the financial information that follows.

GAAP total net revenues for the fourth quarter of 2017 were $38.9 million compared to the prior year’s fourth quarter net revenues of $13.6 million. GAAP net revenues for JUXTAPID in the fourth quarter of 2017 were $20.1 million compared to $8.6 million in the prior year. GAAP net revenues for MYALEPT in the fourth quarter of 2017 were $18.8 million compared to $5.0 million for the same period in 2016.

GAAP total operating expenses for the fourth quarter of 2017 were $35.9 million compared to total operating expenses of $22.0 million for the same period in 2016. GAAP SG&A expenses were $24.1 million in the fourth quarter of 2017 compared to $16.0 million for the same period in 2016. GAAP R&D expenses were $11.8 million in the fourth quarter of 2017 compared to $6.0 million for the same period in 2016.

On a pro forma basis, during the fourth quarter of 2017, SG&A expenses were $22.5 million compared to $55.9 million for the same period in 2016. The decrease in pro forma SG&A expenses in the fourth quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and legal and consulting fees.

On a pro forma basis, during the fourth quarter of 2017, R&D expenses were $11.6 million compared to $14.2 million for the same period in 2016. The decrease in pro forma R&D expenses in the fourth quarter of 2017 compared with the same period in 2016 was primarily related to a reduction in headcount and the timing of vendor related activities.

GAAP net loss in the fourth quarter of 2017 was $24.6 million compared to GAAP net loss of $19.9 million during the same period in 2016.

On a pro forma basis, net loss in the fourth quarter of 2017 was $3.3 million, compared to $20.1 million for the same period in 2016.

Full Year 2017 Financial Results

GAAP total net revenues for the year ended December 31, 2017 were $138.4 million compared to $13.6 million in 2016. GAAP net revenues for JUXTAPID for the year ended December 31, 2017 were $72.1 million compared to $8.6 million in 2016. GAAP net revenues for MYALEPT for the year ended December 31, 2017 were $66.3 million compared to $5.0 million in 2016.

GAAP total operating expenses for the year ended December 31, 2017 were $148.0 million compared to total operating expenses of $44.3 million in 2016. GAAP SG&A expenses were $96.5 million for the year ended December 31, 2017 compared to $29.5 million in 2016. GAAP R&D expenses were $49.0 million for the year ended December 31, 2017 compared to $14.8 million in 2016.

Cost of product sales were $77.2 million in the year ended December 31, 2017. Cost of product sales in the current year includes $18.8 million reserves recorded for excess and obsolete inventory, which are derived from projected sales activities, respective product shelf-life and their respective fair value. Additionally, cost of product sales was also comprised of the cost of inventory sold, amortization of acquired product rights, which resulted from the acquisition of Aegerion, and estimated royalties payable related to the sales of lomitapide and metreleptin. We expect cost of product sales for metreleptin to increase in 2018 and for the next several years, due primarily to an increasing time-based royalty rate on net sales of metreleptin in the U.S.

On a pro forma basis, for the year ended December 31, 2017, SG&A expenses were $90.7 million compared to $200.1 million in 2016. For the year ended December 31, 2017, R&D expenses on a pro forma basis were $48.2 million compared to $52.1 million in 2016.

GAAP net loss for the year ended December 31, 2017 was $126.7 million compared to GAAP net loss of $52.9 million in 2016.

On a pro forma basis, net loss for the year ended December 31, 2017 was $30.0 million, compared to $136.0 million in 2016.

As of December 31, 2017, the Company’s consolidated unrestricted cash balance was $55.4 million, compared to $70.5 million at September 30, 2017 and $108.9 million at December 31, 2016. As of December 31, 2017, there were 18.7 million shares outstanding. At December 31, 2017, total debt principal was $325 million, reflecting the principal amount of convertible debt, before discount, issued by Aegerion and consolidated as a result of the acquisition.

AngioDynamics to Present at the Needham 17th Annual Healthcare Conference

On March 15, 2018 –AngioDynamics, Inc. (NASDAQ:ANGO), a leading provider of innovative, minimally invasive medical devices for vascular access, peripheral vascular disease, surgery and oncology, reported that Michael C. Greiner, Executive Vice President and Chief Financial Officer, will present at the Needham 17th Annual Healthcare Conference at 4:00 p.m. ET on Tuesday, March 27, 2018 in New York, NY (Press release, , 15 15, 2018, View Source [SID1234524828]).

A live webcast of the presentation will be accessible through the "Investors" section of the Company’s website at www.angiodynamics.com and will be available for replay following the event.

X4 Pharmaceuticals to Report Clinical Biomarker Data with X4P-001 at the Upcoming 2018 American Association for Cancer Research (AACR) Annual Meeting

On March 15, 2018 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare disease, reported that an abstract highlighting X4P-001, the Company’s CXCR4 antagonist, has been selected for presentation at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 14-18, 2018 in Chicago, IL (Press release, X4 Pharmaceuticals, MAR 15, 2018, View Source [SID1234524826])

"These results demonstrate enhanced immune cell activation and lymphocyte infiltration with X4P-001, alone and in combination with the anti-PD-1 immunotherapy pembrolizumab (Keytruda), in the melanoma tumor microenvironment."

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Details of the poster presentation on X4P-001 are as follows:

Poster Title: X4P-001, an oral bioavailable CXCR4 antagonist, enhances immune cell infiltration and activation in the tumor microenvironment of melanoma
Author: Robert H.I. Andtbacka, Huntsman Cancer Institute, University of Utah
Session: Immune Response to Therapies 1
Abstract #: 613
Date & Time: Sunday April 15, 2018 1:00 PM – 5:00 PM CT

"We are pleased to present these clinical biomarker data from our ongoing Phase 1b clinical study with X4P-001 in patients with melanoma," said Sudha Parasuraman, MD, Chief Medical Officer of X4 Pharmaceuticals. "These results demonstrate enhanced immune cell activation and lymphocyte infiltration with X4P-001, alone and in combination with the anti-PD-1 immunotherapy pembrolizumab (Keytruda), in the melanoma tumor microenvironment."

About X4P-001-IO in Cancer

X4P-001-IO is an investigational selective, oral, small molecule inhibitor of CXCR4 (C-X-C receptor type 4) that regulates the tumor microenvironment thereby enhancing endogenous anti-tumor responses. CXCR4 is a chemokine receptor that modulates immune function and angiogenesis through the trafficking of key immune cells such as T- cells, dendritic cells, and myeloid derived suppressor cells. CXCR4 signaling is disrupted in a broad range of cancers, facilitating tumor growth by allowing cancer cells to evade immune detection and creating a pro-tumor microenvironment. X4P-001-IO is being investigated in three separate clinical studies in solid tumors.

Obsidian to Present Data on its Destabilizing Domain Technology and Product Programs at the Upcoming 2018 American Association for Cancer Research (AACR) Annual Meeting

On March 15, 2018 Obsidian Therapeutics, Inc., a biotechnology company dedicated to the development of next-generation cell and gene therapies with pharmacologic operating systems, reported that an abstract highlighting preclinical data on its technology and therapeutic applications has been selected for presentation at the 2018 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 14-18, 2018 in Chicago, IL (Press release, , 15 15, 2018, View Source [SID1234524825]).

Details of the poster presentation on Tuesday, April 17, 2018, are as follows:

Poster Title: Enhancing Adoptive Cell Therapies Through Exogenous Regulation: Destabilizing Domains For Next-Generation CAR-T
Lead Author: Celeste Richardson, PhD
Session: Adoptive Cell Therapy – Poster Session 3
Abstract #: 3580 / 18
Date & Time: April 17, 2018, from 8:00am-12:00pm CT
Link to Abstract: View Source!/4562/presentation/7518

About Destabilizing Domains

Obsidian uses Destabilizing Domains (DDs) to enable pharmacologic regulation of protein activity for next-generation cell and gene therapies. Obsidian’s DDs are small, fully-human protein domains that confer conditional stability to a fused payload protein. In the absence of a specific small molecule ligand the fusion protein is rapidly degraded, whereas in the presence of the ligand, the fusion protein becomes stable and functional. Obsidian uses this approach to equip engineered cells with controllable functions that can be precisely tuned by the administration of non-immunosuppressive, small molecule medicines that are readily available and dispensed by the treating physician.