On November 14, 2018 ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, reported financial results for the quarter ended September 30, 2018 and provided a corporate update (Press release, Arca biopharma, NOV 14, 2018, View Source [SID1234531458]).

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"We remain focused on advancing the development of our pipeline of genetically-targeted therapeutics to address the unmet medical needs of patients with cardiovascular disease," commented Dr. Michael Bristow, ARCA’s President and Chief Executive Officer. "FDA is currently giving further consideration to our SPA application for the Phase 3 clinical trial of Gencaro following our provision of supporting information in response to an initial No Agreement letter. We have requested a meeting with the FDA and look forward to meeting with them in December to discuss any remaining issues in the application."

Pipeline Update

GencaroTM (bucindolol hydrochloride) – a pharmacologically unique beta-blocker and mild vasodilator being developed as a potential genetically-targeted treatment for heart failure (HF) patients at risk for atrial fibrillation (AF).

At the end of October, the Company received a No Agreement letter from the U.S. Food and Drug Administration (FDA) on its Special Protocol Assessment (SPA) application for the Phase 3 PRECISION-AF clinical trial. After further correspondence with the FDA and the provision of information supporting the SPA application, the FDA has agreed to reconsider the SPA request. ARCA has requested a meeting with the FDA to review the SPA application, which the Company anticipates will occur in December.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for heart failure (HF) and peripheral arterial disease (PAD).

Chemistry, manufacturing and controls (CMC) activities were continued in the third quarter.
IND-enabling non-clinical studies are anticipated to begin in the first half of 2019.
Third Quarter 2018 Summary Financial Results

Cash, cash equivalents and marketable securities totaled $8.1 million as of September 30, 2018, compared to $11.8 million as of December 31, 2017. ARCA believes that its current cash, cash equivalents and marketable securities will be sufficient to fund its operations, at its projected cost structure, through the end of the first quarter of 2019.

Research and development (R&D) expenses for the quarter ended September 30, 2018 totaled $0.7 million compared to $3.5 million for the corresponding period of 2017. The $2.7 million decrease in R&D expenses in the third quarter of 2018 as compared to the third quarter 2017 was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial. The Company expects R&D expenses in 2018 to be lower than 2017 as the GENETIC-AF clinical trial has been completed.

General and administrative (G&A) expenses for the quarter ended September 30, 2018 were $0.9 million, relatively unchanged compared to the $1.0 million in the third quarter of 2017. ARCA expects G&A expenses in 2018 to be consistent with those in 2017 as it maintains administrative activities to support ongoing operations.

Total operating expenses for the quarter ended September 30, 2018 were $1.7 million compared to $4.5 million for the third quarter of 2017. The decrease in total operating expenses for the second quarter of 2018 was primarily due to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial.

Net loss was $1.6 million, or $0.11 per share, for the third quarter of 2018 compared to $4.4 million, or $0.39 per share, for the third quarter of 2017.

On November 14, 2018 Trillium Therapeutics Inc. (NASDAQ/TSX: TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported financial and operating results for the nine months ended September 30, 2018 (Press release, Trillium Therapeutics, NOV 14, 2018, View Source [SID1234531305]).

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2018 Third Quarter Highlights:

Presented an update at the European Organisation for Research and Treatment of Cancer, Cutaneous Lymphoma Task Force (EORTC CLTF) meeting on the safety and efficacy of the ongoing multicenter, open-label phase 1 intratumoral trial of TTI-621 in 23 patients with relapsed/refractory mycosis fungoides/Sézary syndrome, 20 of whom only received induction therapy consisting of 1-6 injections over 2 weeks. Local delivery of TTI-621 was well tolerated, with no treatment-related > Grade 3 adverse events or dose-limiting toxicity observed. Reductions in CAILS scores, which measure local lesion responses, were observed in 89% of patients, with 44% exhibiting reductions of 50% or greater. These responses occurred rapidly within the 2-week induction period. Similar CAILS scores changes were seen in adjacent non-injected lesions, suggesting locoregional effects that were not confined to the site of injection. Evidence of a systemic effect was observed in 1 of 2 patients receiving continuation monotherapy beyond the 2-week induction therapy. In addition, data suggest a combination effect with pegylated IFN-alpha-2a.

Presented an update at the Discovery on Target conference on the safety and efficacy of the ongoing multicenter, open-label phase 1a/b intravenous trial of TTI-621 in patients with relapsed/refractory hematologic malignancies. Based on an expanded data set of 163 patients, weekly infusions of TTI-621 were shown to be well tolerated. Thrombocytopenia was the most frequent grade 3 or higher treatment-emergent adverse event, occurring in 20% of patients. Platelet reductions, however, were shown to be transient and pre-dose platelet levels remained steady during the course of the study. Notably, the reversible thrombocytopenia did not lead to an increased risk of bleeding and had no impact on drug delivery, nor was there a significant impact of TTI-621 on hemoglobin levels. Monotherapy efficacy was observed in patients with mycosis fungoides (19% ORR, n=21), peripheral T-cell lymphoma, or PTCL (25% ORR, n=12), and diffuse large B-cell lymphoma, or DLBCL (25% ORR, n=8), and in DLBCL patients when combined with rituximab (25% ORR, n=24). This clinical activity was observed in patients receiving relatively low doses of drug (0.2 mg/kg for monotherapy or 0.1 mg/kg in combination with rituximab). Dose intensification beyond 0.2 mg/kg is currently ongoing, and doses of 0.5 mg/kg have been well tolerated for up to 27 weeks.
"The growing body of data from the TTI-621 intratumoral trial in patients with CTCL continues to look encouraging, and we believe that there are several potential paths forward in this indication, and possibly in other accessible tumors," said Dr. Niclas Stiernholm, president and CEO of Trillium Therapeutics. "Additionally, data from over 160 patients in our intravenous trial indicate that systemically delivered TTI-621 has single-agent and combination activity at relatively low doses, and ongoing efforts to dose intensify appear encouraging. We believe that flexibility is the best way to address this new IO pathway, and having two development candidates in the clinic, and options of intravenous and intratumoral delivery, as monotherapy or in combination, gives Trillium the most comprehensive approach to targeting CD47."

Third Quarter 2018 Financial Results:

As of September 30, 2018, Trillium had cash and cash equivalents and marketable securities, and working capital, of $52.1 million and $41.8 million, respectively, compared to $81.8 million and $68.9 million, respectively, at December 31, 2017. The decrease in cash and cash equivalents and marketable securities was due mainly to cash used in operations of $30.8 million, net of an unrealized foreign exchange gain of $1.3 million. The decrease in working capital was due mainly to cash used in operations, increases to amounts receivable and prepaid expenses, and a decrease to accounts payable and accrued liabilities due to clinical trial payments.

Net loss for the nine months ended September 30, 2018 of $33.9 million was lower than the loss of $34.4 million for the nine months ended September 30, 2017. The net loss was lower mainly due to a net foreign currency gain of $1.5 million for the nine months ended September 30, 2018, compared to a net foreign currency loss of $4.9 million in the prior year period, and lower manufacturing costs, partially offset by higher clinical trial expenses and the expense relating to the amendment of the SIRPaFc license agreement.

On November 14, 2018 Seattle Genetics, Inc. (Nasdaq:SGEN) reported dosing of the first patient in a phase 1 clinical trial evaluating the safety and tolerability of SEA-BCMA for patients with relapsed or refractory multiple myeloma (MM) (Press release, Seattle Genetics, NOV 14, 2018, View Source [SID1234531306]). SEA-BCMA is an investigational antibody empowered using Seattle Genetics’ proprietary Sugar Engineered Antibody (SEA) technology designed to enhance antibody dependent cellular cytotoxicity. The target of SEA-BCMA, the cell surface protein B-cell maturation antigen (BCMA), is broadly expressed on malignant plasma cells in multiple myeloma. SEA-BCMA has demonstrated encouraging antitumor activity in preclinical studies.

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"Despite recent advances in the treatment of multiple myeloma, it remains an incurable disease with a need for active and well-tolerated agents," said Roger D. Dansey, M.D., Chief Medical Officer of Seattle Genetics. "BCMA is a validated therapeutic target for multiple myeloma. SEA-BCMA represents a novel empowered antibody treatment approach that has demonstrated antitumor activity and an acceptable safety profile in preclinical evaluation to date. We look forward to evaluating SEA-BCMA through our clinical development program and hope to meaningfully improve outcomes for multiple myeloma patients."

The phase 1 trial is an open-label, multi-center, dose-escalation and expansion clinical study designed to enroll approximately 65 patients with relapsed or refractory MM in the United States. The study is expected to be conducted in two parts, with a dose escalation cohort to evaluate safety and tolerability and to determine the maximum tolerated dose of SEA-BCMA, followed by an expansion cohort to obtain further data regarding safety and antitumor activity.

Data presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018 demonstrate that SEA-BCMA exhibits encouraging preclinical antitumor activity and has the potential to be an active, tolerable and combinable agent.

For more information about the phase 1 study of SEA-BCMA for patients with multiple myeloma and enrolling centers, please visit www.clinicaltrials.gov (Identifier: NCT03582033).

About Multiple Myeloma

Multiple myeloma (MM) is an aggressive cancer that forms in white blood cells called plasma cells. Cancerous plasma cells can crowd out healthy blood cells, impair bone strength and weaken the immune system. MM is the second most common blood cancer in the United States. According to the American Cancer Society, more than 30,000 new cases of MM were expected in the U.S. in 2018, with over 12,500 deaths. Despite recent medical advances, MM still remains an incurable disease. It is managed with sequential lines of treatment that typically yield shorter durations of disease control with each subsequent relapse, and some patients receive more than four lines of treatment over the course of their disease.

About SEA-BCMA

SEA-BCMA is a novel investigational antibody empowered using Seattle Genetics’ proprietary Sugar Engineered Antibody (SEA) technology that leads to enhanced antibody dependent cellular cytotoxicity. SEA-BCMA is a non-fucosylated BCMA-directed antibody that is designed to block proliferative tumor cell signaling, mediate antibody dependent cellular phagocytosis and induce enhanced cell lysis through antibody dependent cellular cytotoxicity. The cell surface protein BCMA is expressed on cells of several cancer types, including multiple myeloma and other B-cell malignancies. BCMA is a validated therapeutic target for multiple myeloma.

On November 14, 2018 Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic or the Corporation) reported today its unaudited financial results for the third quarter ended September 30, 2018 (Press release, ProMetic Life Sciences, NOV 14, 2018, View Source [SID1234531350]).

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"Several initiatives that we have been pursuing during the past quarter should bear fruit in the coming weeks and months," said Pierre Laurin, Prometic’s President and Chief Executive Officer. "We have implemented cost-control measures to reduce our cash use while at the same time have made significant progress to advance our two lead drug candidates, Ryplazim (plasminogen) and PBI-4050. As previously stated, our current business plan calls for a significant reduction in R&D expenditures of up to $30 million in 2019 as compared to this year’s budget. Our primary objective remains to close the gap between the fundamental enterprise value we have built and our current market valuation by strengthening our financial position through the closing of commercial partnerships and equity-related initiatives."

Commenting on the third quarter 2018 financial results, Bruce Pritchard, Prometic’s Chief Operating Officer and Chief Financial Officer, said, "We are ahead of target with the financial guidance provided during the AGM in May 2018 and our last few conference calls. We have effectively implemented cost control measures as evidenced by the trending quarterly decrease in R&D, Administration and Sales & Marketing expenses as well as decreases in net loss and cash flows used in operating activities. Cash used in operations year-to-date was $57 M compared to $95 M for the same period in 2017".

Small Molecule Therapeutics Highlights

PBI-4050 – Received a Rare Pediatric Disease designation from the US Food and Drug Administration for the treatment of Alström syndrome
PBI-4050 – Published a paper further elucidating the mechanism of action of its lead drug candidate, PBI-4050, on liver fibrosis in the Journal of Pharmacology and Experimental Therapeutics. The paper entitled "PBI-4050 reduces stellate cell activation and liver fibrosis through modulation of intracellular ATP levels and LKB1-AMPK-mTOR pathway" details the antifibrotic signaling pathway modulated by PBI-4050.
PBI-4050 – Hosted a Key Opinion Leader meeting in New York on PBI-4050 as a novel treatment for Alström syndrome and non-alcoholic steatohepatitis (NASH).
Plasma-Derived Therapeutics Highlights

RyplazimTM (Plasminogen) – Completed a Type C meeting in which the FDA agreed with the Company’s proposed action plan for the implementation of additional analytical assays and in-process controls related to the RyplazimTM (plasminogen) manufacturing process. As a result of the feedback received during the Type C meeting, Prometic is finalizing the process performance qualification (PPQ) protocols in anticipation of commencing the manufacturing of additional RyplazimTM (plasminogen) conformance lots.

Subsequent Events to Third Quarter 2018

Closed a deal with Structured Alpha LP (SALP), an affiliate of Thomvest Asset Management Inc., to extend the maturity dates of the USD $80 million (CAD $100 million) line of credit and the Original Issue Discount Notes to September 2024.
2018 Third Quarter Financial Results

Revenues
Total revenues for the third quarter ended September 30, 2018 were $12.3 million and $36.8 million for the nine months ended September 30, 2018. Revenues from the sale of goods, representing most of the 2018 revenues to date, were $35.3 million during the first nine months ended September 30, 2018 compared to $11 million during the corresponding 2017 period. The $24.3 million increase for 2018 is mainly due to $19.7 million in sales of normal source plasma which occurred in the second and third quarters of 2018 following a change in the production forecast due to the delay of the BLA approval for RyplazimTM (plasminogen). The remainder of the increase of $4.6 million for the nine month period is due to an increase in third party sales in the bioseparation segment. 2018 bioseparation sales are expected to exceed $21 million, which would represent a 30% increase compared to 2017 bioseparation revenues. A comparable level of revenue growth for 2019 is anticipated and is mainly due to the expansion of manufacturing activities by existing clients who utilize Prometic’s products in their production processes, the adoption of products by new clients, the introduction of new products and the continuing expansion of the market for bioseparation products.

Cost of sales and other production expenses
Cost of sales and other production expenses were $9.2 million for the third quarter ended September 30, 2018 compared to $3.8 million for the corresponding period in 2017, representing an increase of $5.5 million. Cost of sales and other production expenses were $30.4 million during the nine months ended September 30, 2018 compared to $7.7 million for the corresponding period in 2017, representing an increase of $22.7 million. The increase was due primarily to the cost of the plasma inventory sold.

Research and Development (R&D)
Total R&D expenses were $24.1 million for the third quarter ended September 30, 2018 compared to $23.3 million for the third quarter ended September 30, 2017. Total R&D expenses were $70.5 million for the nine months ended September 30, 2018 compared to $72.2 million for the corresponding period in 2017, representing a decrease of $1.7 million. The completion of the pivotal phase 3 clinical programs for IVIG and for Ryplazim (plasminogen) and termination of non core preclinical and clinical programs will translate into a significant R&D cost reduction in 2019 compared to 2018.

Administration, Sales & Marketing
Administration, selling and marketing expenses were $6.2 million for the third quarter ended September 30, 2018 compared to $7.7 million for the third quarter ended September 30, 2017. The $1.4 million decrease was due to a reduction in consulting fees and employee compensation expenses. Administration, selling and marketing expenses declined slightly at $20.9 million during the nine months ended September 30, 2018 compared to $22.7 million for the corresponding period in 2017.

Finance Costs
Finance costs were $5.9 million for the third quarter ended September 30, 2018 compared to $2.1 million during the corresponding period of 2017, representing an increase of $3.8 million. Finance costs were $15.5 million for the nine months ended September 30, 2018 compared to $5.3 million during the corresponding period of 2017, representing an increase of $10.2 million. This increase reflects higher debt levels during the nine months ended September 30, 2018 compared to the same period of 2017.

Net Loss
Prometic incurred a net loss of $28.9 million for the third quarter ended September 30, 2018 compared to a net loss of $17.8 million for the third quarter ended September 30, 2017. Prometic incurred a net loss of $96.6 million for the nine months ended September 30, 2018 compared to a net loss of $78.4 million for the corresponding period of 2017. The main reason for the increase in the net loss is that the results for the quarter and the nine months ending September 30, 2017 included $19.7 million in milestone and licensing revenues related to the licensing agreement signed with Jiangsu Renshou Pharmaceutical Co, Ltd.

With the delay of the anticipated launch of its most advanced product, RyplazimTM (plasminogen), the Corporation had to finance its R&D activities via various sources. To date, the Corporation has financed its activities through the sale of products in the bioseparations segment, collaboration arrangements and licensing arrangements, the issuance of debt and equity, operational restructuring as well as investment tax credits. Prometic is currently actively involved in negotiating both equity and equity-linked financing initiatives and continues to be in dialogue with potential licensing partners. Although the Corporation believes that it will be able to obtain the necessary funding as in the past, there can be no assurance of the success of these plans.

Conference Call Information

Prometic will host a conference call at 11:00 am (ET) on Thursday November 15, 2018. The telephone numbers to access the conference call are (647) 427-7450 and 1-888-231-8191 (toll-free). A replay of the call will be available as of Thursday November 15, 2018 at 2:00 pm. The numbers to access the replay are 1-416-849-0833 and 1-855-859-2056 (passcode: 1190238). A live audio webcast of the conference call, with slides, will be available through the following: View Source

Additional Information in Respect to the Third Quarter Ended September 30, 2018

Prometic’s MD&A and condensed interim consolidated financial statements for the quarter ended September 30, 2018 will be filed on SEDAR (View Source) and will be available on the Company’s website at www.prometic.com.

On November 14, 2018 ArQule, Inc. (Nasdaq: ARQL) reported that Paolo Pucci, Chief Executive Officer, and Marc Schegerin, Senior Vice President, Head of Strategy, Finance, and Communication will present at the 30th Annual Piper Jaffray Healthcare Conference on November 28, 2018, at 1:50pm ET at the Lotte New York Palace in New York City (Press release, ArQule, NOV 14, 2018, View Source [SID1234531291]).

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The live webcast of the presentation will be available via the "Investors & Media" section of ArQule’s website, www.arqule.com, under "Events & Presentations." A replay of the webcast will be available shortly after the conclusion of the presentation.