Stemline In-Licenses Worldwide Rights to Novel Selective RET Inhibitor (SL-1001); Expands Oncology Pipeline

On March 11, 2019 Stemline Therapeutics, Inc. (Nasdaq: STML), a commercial-stage biopharmaceutical company focused on the development and commercialization of novel oncology therapeutics, reported that it has exclusively licensed worldwide rights to develop and commercialize a novel, oral, selective small molecule RET (rearranged during transfection) kinase inhibitor from the CRT Pioneer Fund LP, a £70 million specialist oncology investment fund managed by Sixth Element Capital, a UK based fund manager (Press release, Stemline Therapeutics, MAR 11, 2019, View Source [SID1234534227]). The RET inhibitor was rationally designed by scientists at Cancer Research UK Manchester Institute at the University of Manchester (United Kingdom). The preclinical compound has been designated SL-1001 and is expected to enter the clinic in 2020.

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RET kinase genetic alterations have been found in a diverse range of cancers and, we believe, is a clinically validated target in multiple indications. SL-1001 is an oral RET kinase inhibitor that has demonstrated potent, selective, preclinical anti-cancer activity, both in vitro and in vivo, in RET-driven tumor models.

"SL-1001 is an ideal strategic fit for us, given its targeted mechanism of action and potential for streamlined development," commented Stemline’s CEO, Ivan Bergstein. He continued, "Building upon our team’s proven track record of success, culminating with the recent ELZONRIS approval, we intend to advance SL-1001 swiftly through the clinical and regulatory process with an eye towards achieving our goal of helping more patients succeed in their fight against cancer."

Robert James, Managing Partner at Sixth Element Capital said, "We are delighted to have partnered SL-1001 with Stemline. We are confident that Stemline’s skills and resources will take the project forward rapidly and offer an alternative treatment option to patients with cancer driven by RET kinase".

ArQule to Participate in the 31st Annual ROTH Conference on March 17-19, 2019

On March 11, 2019 ArQule, Inc. (Nasdaq: ARQL) reported that Paolo Pucci, Chief Executive Officer, and Marc Schegerin, Senior Vice President and Head of Strategy, Finance and Communication, will participate in the 31st Annual ROTH Conference on March 17-19, 2019 in Dana Point, California (Press release, ArQule, MAR 11, 2019, View Source [SID1234534212]).

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BioXcel Therapeutics Announces Late Breaking Data Presentation at AACR 2019 Annual Meeting

On March 11, 2019 BioXcel Therapeutics, Inc. ("BTI") (BTAI) reported that it will present a late breaking poster featuring data from a preclinical study of the Company’s BXCL701 and an OX40-agonist antibody as a potential combination therapy for treatment of certain solid tumors at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 being held from March 29 to April 3, 2019 in Atlanta, Georgia (Press release, BioXcel Therapeutics, MAR 11, 2019, View Source [SID1234534213]).

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Details of the accepted poster are below:

Abstract #077 / Poster #22: Dipeptidyl Peptidase Inhibitor BXCL701 synergizes with an OX40-agonist antibody resulting in synergistic anti-tumor response and survival in an animal model of colorectal cancer by bridging the innate and adaptive arms of the immune system

Date:

Monday, April 01, 2019

Time:

8:00 AM-12:00 PM ET

Session:

Late Breaking Research – Immunotherapy 1

Location:

Georgia World Congress Center, Exhibit Hall B, Section 41

Dr. Vince O’Neill, Chief Medical Officer of BTI commented, "The encouraging results from this preclinical study of BXCL701 and an OX40 agonist, an investigational monoclonal antibody immunotherapy, represent a potentially valuable therapeutic option for cancer patients. We believe that this immunotherapy combination can effectively activate both the innate and adaptive immune system to fight cancer. We are excited to showcase this compelling data set and its potential at the AACR (Free AACR Whitepaper) annual meeting."

About BXCL701

BXCL701 is an orally-available systemic innate-immune activator with dual mechanisms of action. It has demonstrated single agent activity in melanoma, with an established safety profile from 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting Fibroblast Activation Protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other immuno-oncology agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. BXCL701’s primary mechanism of action has recently been highlighted in multiple peer reviewed journals, providing an important validation of the scientific rationale behind BXCL701.

ChemoCentryx Reports Fourth Quarter and Full Year 2018 Financial Results and Recent Highlights

On March 11, 2019 ChemoCentryx, Inc., (Nasdaq:CCXI), reported financial results for the fourth quarter and full year ended December 31, 2018 and provided an overview of the Company’s recent corporate highlights (Press release, ChemoCentryx, MAR 11, 2019, View Source [SID1234534214]).

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"We at CCXI marched through 2018, achieving essential objectives both tactical and strategic," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "Those 2018 achievements now set the stage for a suite of successive late-stage data readouts starting in the fourth quarter of this year with top-line results from the pivotal ADVOCATE Phase III trial of avacopan in ANCA-associated vasculitis."

"But ADVOCATE was just the beginning of the pipeline in a drug strategy for avacopan. Another step was the launching in December of the AURORA trial of avacopan in hidradenitis suppurativa, a disfiguring and debilitating skin disorder. AURORA is a large, potentially registration-supporting clinical trial of avacopan, and we aim for top-line data as soon as mid-2020. The third step in the strategy is avacopan in C3 glomerulopathy. The C3G trial has shown strong enrollment trends; putting us again in a position to obtain top-line data for this devastating condition in 2020. Keen observers will be mindful that our renal orphan disease franchise has a second unique asset: the CCR2 inhibitor CCX140 for the treatment of focal segmental glomerulosclerosis in our LUMINA trials program."

"CCXI’s financial position remains strong and provides us the financial foundation to run these trials simultaneously, and to achieve the data objectives outlined here. Our basic science and discovery platform continues to create value – identifying novel modes of action of our late stage pipeline assets avacopan and CCX140, while also moving toward additional, novel pipeline candidates in renal and dermal disease. For all of these reasons, I think this is one of the most exciting and most promising times in the history of the enterprise."

Recent Highlights


Launched the Company’s Phase IIb clinical trial of avacopan for the treatment of Hidradenitis Suppurativa (HS), called the AURORA trial. HS is a chronic disabling skin autoimmune disease characterized by recurrent, painful, nodules, boils and abscesses. A proof-of-concept study has demonstrated that HS is driven by neutrophils produced by C5a. The AURORA trial aims to enroll 390


patients with moderate to severe HS. The primary endpoint will assess avacopan against placebo at 12 weeks of treatment, using the HiSCR (hidradenitis suppurativa clinical response) scale, which has been validated by the FDA. All groups will be followed for an additional 24 weeks. Secondary endpoints include percent improvement from baseline to week 12 between groups, validated secondary measurements and quality of life measured using the HS Quality of Life Instrument (HiSQOL) and SF36 health assessment.


Advanced enrollment in the Company’s clinical trial of avacopan in patients with the kidney disease C3 Glomerulopathy (C3G). Enrollment in this randomized control trial of 88 C3G patients now approaches 50%. C3G is a rare disorder that often affects the young, requiring dialysis and often kidney transplant with recurring disease common. There is no approved effective treatment for C3G.


Reached a milestone in the Company’s clinical trials of CCX140 in two sub-populations of Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease: the LUMINA 1 trial, evaluating patients with sub-nephrotic primary FSGS, is over 50% enrolled. Enrollment in LUMINA 2, evaluating nephrotic syndrome primary FSGS, continues.


Maintained a robust balance sheet, with reported cash and investments exceeding $175 million at December 31, 2018, excluding an additional cash raise of $20 million in January 2019.

Fourth Quarter and Full Year 2018 Financial Results

Cash and investments, totaled $177.0 million at December 31, 2018, excluding $20.0 million in gross proceeds from the January 2019 issuance of common stock under the Company’s equity distribution agreement.

Revenue was $9.3 million for the fourth quarter of 2018, compared to $56.3 million for the same period in 2017. For the full year ended December 31, 2018, revenue was $42.9 million, compared to $82.5 million for 2017. The decrease in revenue from 2017 to 2018 was due to the adoption of ASC 606 under the modified retrospective transition method and reflected the cumulative effect of initially applying the new revenue standard of $47.3 million as an adjustment to the opening balance of accumulated deficit and an increase in deferred revenue. Revenue recognized prior to January 1, 2018 has not been restated and continues to be reported under the accounting standards in effect for those periods.

Research and development expenses were $15.1 million for the fourth quarter of 2018, compared to $12.9 million for the same period in 2017. Full year 2018 research and development expenses were $62.7 million compared to $49.5 million in 2017. The increase in research and development expenses from 2017 to 2018 was primarily due to the advancement of the avacopan ADVOCATE Phase III pivotal trial which completed enrollment in July 2018, initiation and patient enrollment of the avacopan Phase II clinical trials in patients with C3G and HS and the CCX140 Phase II clinical trials in patients with FSGS.

General and administrative expenses were $5.6 million for the fourth quarter of 2018, compared to $4.1 million for the same period in 2017. Full year 2018 general and administrative expenses were $20.4 million, compared to $16.5 million in 2017. The increase from 2017 to 2018 was primarily due to higher employee-related expenses, including those associated with our commercialization planning efforts, and higher professional fees.

Net loss for the fourth quarter of 2018 was $10.8 million, compared to net income of $39.7 million for the same period in 2017. Full year 2018 net loss was $38.0 million, compared to net income of $17.9 million in 2017.

Total shares outstanding at December 31, 2018 were approximately 50.7 million shares.

The Company expects to utilize cash and investments in the range of $75.0 million and $85.0 million in 2019.

Conference Call and Webcast

The Company will host a conference call and webcast today, March 11, 2019 at 5:00 p.m. Eastern Time / 2:00 p.m. Pacific Time. To participate by telephone, please dial 877-303-8028 (Domestic) or 760-536-5167 (International). The conference ID number is 2269237. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.

IMV Inc. Announces Partial Exercise of Option to Purchase Additional Shares in
Connection with Underwritten Public Offering of Common Shares

On March 11, 2019 IMV Inc. ("IMV" or the "Corporation") (NASDAQ: IMV; TSX: IMV), a clinical-stage immuno-oncology corporation, reported that the underwriters of its previously announced underwritten public offering (the "Offering") of common shares have partially exercised their option to purchase additional common shares, resulting in the issuance of an additional 504,855 common shares of the Corporation at a price of C$5.45 per share for additional gross proceeds of approximately C$2.75 million (Press release, IMV, MAR 11, 2019, View Source [SID1234534215]). As a result of the exercise of this option, the Corporation has raised total gross proceeds of approximately C$29.46 million from the Offering, before deducting the underwriting commissions and Offering expenses.

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The Corporation intends to use the net proceeds of the Offering to accelerate the development of DPX-Survivac in combination with Keytruda as part of the basket trial select advanced or recurrent solid tumours in bladder, liver (hepatocellular carcinoma), ovarian or non-small-cell lung cancers, as well as tumours shown to be positive for the microsatellite instability high biomarker and for general corporate purposes.

Wells Fargo Securities and Raymond James acted as joint book-running managers for the Offering. B. Riley FBR acted as co-manager.

The Offering was made pursuant to a U.S. registration statement on Form F-10, declared effective by the U.S. Securities and Exchange Commission (the "SEC") on June 6, 2018 (the "Registration Statement"), and the Company’s existing Canadian short form base shelf prospectus (the "Base Prospectus") dated June 5, 2018. A preliminary prospectus supplement relating to the Offering was filed on February 28, 2019 with the securities commissions in the provinces of British Columbia, Alberta, Saskatchewan, Manitoba, Ontario, Québec, Nova Scotia and Newfoundland and Labrador in Canada, and with the SEC in the United States, and a final prospectus supplement relating to the Offering (the "Supplement") was filed on March 1, 2019 with the securities commissions in the provinces of British Columbia, Alberta, Saskatchewan, Manitoba, Ontario, Québec, Nova Scotia and Newfoundland and Labrador in Canada, and with the SEC in the United States. The Supplement and the accompanying Base Prospectus contain important detailed information about the Offering. The Supplement and the accompanying Base Prospectus can be found on SEDAR at www.sedar.com and on EDGAR at www.sec.gov. Copies of the Supplement and accompanying Base Prospectus may also be obtained from Wells Fargo Securities, Attn: Equity Syndicate, 375 Park Avenue, New York, NY 10152, by telephone at (800) 326-5897, or by email at [email protected] or from Raymond James, Attn: Equity Syndicate, 880 Carillon Parkway, St. Petersburg, Florida 33716, or by telephone at (800) 248-8863, or e-mail at [email protected].

This news release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any province, state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such province, state or jurisdiction.