On February 27, 2019 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 year ended December 31, 2018 and provided a corporate update (Press release, Arca biopharma, FEB 27, 2019, View Source [SID1234533785]).

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"During 2018, we continued to make progress on our lead development program as we: reported Phase 2B results for the GENETIC-AF trial; designed a Phase 3 development plan based on learnings from those results; identified a clear regulatory path; and, began discussions with the FDA to confirm agreement on future development of Gencaro, potentially the first genetically-targeted cardiovascular therapeutic," commented Dr. Michael Bristow, ARCA’s President and Chief Executive Officer. "With the Gencaro FDA SPA agreement now in place, 2019 will be an important year for ARCA as we work towards the initiation of the Phase 3 clinical trial and expand development activities for AB171, a potential genetically-targeted treatment for heart failure and peripheral arterial disease."

Pipeline Update

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

In February 2019, ARCA received a Special Protocol – 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.

– PRECISION-AF is designed as a double-blind, active-controlled, multicenter, international study comparing Gencaro with Toprol-XL (metoprolol succinate) for the prevention of AF recurrence or all-cause mortality (ACM) in HF patients with mid-range ejection fraction (HFmrEF).

– Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the fourth quarter of 2019.
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 fourth quarter.
IND-enabling non-clinical studies are anticipated to begin in the second half of 2019.
IND submission anticipated in the first quarter of 2020.
2018 Summary Financial Results

Cash, cash equivalents and marketable securities were $6.6 million as of December 31, 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 third quarter of 2019.

Research and development (R&D) expenses for the year ended December 31, 2018 were $4.2 million compared to $14.1 million for 2017. The $9.8 million decrease in R&D expenses in 2018 as compared to 2017 was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial in 2017.

General and administrative (G&A) expenses for the year ended December 31, 2018 were $3.9 million compared to $4.6 million in 2017. The Company expects G&A expenses in 2019 to be consistent with those in 2018 as it maintains administrative activities to support our ongoing operations.

Total operating expenses for 2018 were $8.1 million compared to $18.7 million during 2017. The decrease in total operating expenses in 2018 was primarily due to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial.

Net loss was $7.9 million, or $0.57 per share, for 2018 compared to $18.5 million, or $1.77 per share, for 2017.

On February 27, 2019 Helix BioPharma Corp. (TSX: HBP), (FSE: HBP) ("Helix" or the "Company"), an immuno-oncology company developing innovative drug candidates for the prevention and treatment of cancer, reported that together with the Moffitt Cancer Center ("Moffitt"), a poster entitled "Improving survival in pancreatic cancer using Doxorubicin in combination with L-DOS47" will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) ("AACR") Annual Meeting 2019 which is taking place on March 29 – April 3, 2019 at the Georgia World Congress Center, Atlanta, Georgia, USA.

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The presentation will describe the use of a preclinical pancreatic mouse model to study the effects of LDOS47 alone or in combination therapies. This model was generated by modifying a mouse pancreatic cancer cell line (Panc02) to express the human ceacam6 antigen that is recognized by L-DOS47. The model is designed to be used for monotherapy L-DOS47 efficacy studies and for combination studies with various agents, including immunotherapies. In addition, to therapeutic studies, the model may also be helpful in studying the effects of L-DOS47 in modifying tumor pH and the tumor immune environment. The presentation will describe the development of the cell line, generation of tumor grafts, and show responses to L-DOS47 in combination with doxorubicin treatments. The usefulness of the model, including difficulties of this model, will also be described. This work is part of an on-going collaboration with Moffitt and is being performed to support the clinical development of L-DOS47 and the DOS47 platform.

"I would like to thank Dr. Robert (Bob) Gillies and the Moffitt team on leading this work" said Heman Chao, Helix’s Chief Executive Officer. "Following on the excellent safety and tolerability results from the Phase I monotherapy lung cancer study in Poland and continuing good progress made with the U.S. Phase I LDOS47 combination study with pemetrexed and carboplatin, we are very excited to launch the pancreatic program and expand the clinical application of L-DOS47."

Presentation details are as follows:

Session Category: Experimental and Molecular Therapeutics
Session Title: Cellular Responses to Anticancer Agents 1: The Microenvironment and Metastasis
Session Date and Time: Monday April 1, 2019 from 8:00 AM-12:00 PM
Location: Georgia World Congress Center, Exhibit Hall B.

On February 27, 2019 Vaccibody AS reported that clinical and preclinical data will be presented at the upcoming 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, which will be held from March 29- April 3, 2019 in Atlanta, Georgia (Press release, Vaccibody, FEB 27, 2019, View Source [SID1234533782]). The abstracts will be published in advance of the AACR (Free AACR Whitepaper) Annual meeting at the AACR (Free AACR Whitepaper) Online Itinerary Planner: View Source!/6812.

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"We are excited that four separate abstracts presenting Vaccibody’s pipeline have been accepted for poster presentation at this year’s AACR (Free AACR Whitepaper) Annual Meeting," said Agnete Fredriksen, PhD, President and CSO of Vaccibody. Two of the abstracts cover Vaccibody’s two lead candidates VB10.16 and VB10.NEO and are presenting the 6M interim data and the clinical design, respectively. In addition, two abstracts present preclinical data for VB10.NEO that demonstrates the progress we have made characterizing the unique Vaccibody platform and its ability to induce strong tumor protective immune responses alone and in combination with bempegaldesleukin (NKTR-214).

Details of the four poster presentations are as follows:

Abstract #CT209

Title: Safety, efficacy and immunogenicity of VB10.16, a therapeutic DNA vaccine targeting human papillomavirus (HPV) 16 E6 and E7 proteins for high grade cervical intraepithelial neoplasia (CIN 2/3): 6-month data from an exploratory open-label phase I/2a trial

Session Date and Time: Tuesday Apr 2, 2019 1:00 PM – 5:00 PM local time

Abstract #CT217

Title: An open-label, Phase I/IIa study of VB10.NEO (DIRECT-01) in combination with checkpoint blockade in patients with locally advanced or metastatic solid tumors including melanoma, NSCLC, renal cell carcinoma, urothelial cancer or SSCHN

Session Date and Time: Tuesday Apr 2, 2019 1:00 PM – 5:00 PM local time

Abstract #5033

Title: "Vaccibody DNA vaccine platform VB10.NEO induces strong neo-antigen specific CD8+ T cell responses critical to cure established tumors in pre-clinical models"

Session Data and Time: Wednesday Apr 3, 2019 8:00 AM – 12:00 PM local time

Abstract #2256

Title: "Combination of neoantigen DNA plasmid vaccine VB10.NEO and NKTR-214, a CD122-biased immunostimulatory cytokine, induces strong neoantigen-specific T cell responses and sustained tumor regression in pre-clinical models"

Session Data and Time: Monday Apr 1, 2019 1:00 PM – 5:00 PM local time

About VB10.16

VB10.16 is an investigational therapeutic DNA vaccine developed to treat human papillomavirus type 16 (HPV16) induced pre-malignancies and malignancies. The drug candidate has demonstrated favorable 6M interim clinical data in a Phase I/IIa study in pre-cancerous HPV16 induced high grade cervical intraepithelial neoplasia (HSIL; CIN 2/3).

About VB10.NEO

VB10.NEO, is a proprietary therapeutic DNA vaccine which uses the patient’s own neoantigens for the personalized treatment of cancer patients. A phase I/IIa neoantigen clinical trial is currently enrolling patients with locally advanced or metastatic melanoma, non-small cell lung carcinoma, clear renal cell carcinoma as well as urothelial cancer or squamous cell carcinoma of the head and neck.

On February 27, 2019 Sirona Biochem Corp. (TSX-V: SBM) (Frankfurt: ZSB) (Xetra: ZSB) (the "Company") reported that it has closed an oversubscribed, non-brokered private placement for gross proceeds of $1,783,500 (Press release, Sirona Biochem, FEB 27, 2019, View Source [SID1234533781]). The private placement consists of 17,835,000 units, (the "Units") at a price of $0.10 per Unit. Each Unit consists of one common share and one transferable share purchase warrant, each whole warrant is exercisable into one additional common share of the Company for a period of 3 years from the date of issue at a price of $0.16 per share.

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All securities issued under the placement are subject to a statutory hold period expiring on June 27, 2019.

The Company compensated finders by way of cash fees of $62,136 and 621,360 warrants.

The financing consisted of participation by PRO-group member Shaun Chin of PI Financial, for 500,000 units, as well as two insiders. Dr. Howard Verrico, CEO, has participated with the purchase of 960,000 Units and Christopher Hopton, CFO, has participated with a purchase of 350,000 Units.

Net proceeds of the placement will be used for general working capital while the company continues its efforts toward licensing and commercialization of various pipeline products.

The Company also announced that it has granted incentive stock options under its Stock Option Plan to directors and officers of the Company for the purchase of up to 700,000 common shares at a price of $0.12 per share. Directors will receive 400,000 options at $0.12 with a 5 year expiry and Officers will receive 300,000 options at $0.12 with a 10 year expiry.

On February 27, 2019 Aduro Biotech, Inc. (NASDAQ: ADRO) reported financial results for the fourth quarter and full year ended December 31, 2018 (Press release, Aduro Biotech, FEB 27, 2019, View Source;p=RssLanding&cat=news&id=2389321 [SID1234533779]).

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"2018 was an important year for Aduro as we prioritized our core technologies and determined a go-forward strategy that is committed to maintaining a leadership role in the STING and APRIL pathways and advancing our clinical programs in areas of high unmet medical need," said Stephen T. Isaacs, chairman, president and chief executive officer of Aduro. "With our strategic focus established and $277.9 million in cash taking us into 2022, we are eager to execute on our clinical development plans for 2019 and beyond."

Key Accomplishments in Fiscal Year 2018

STING

Presented first-in-human monotherapy clinical data for ADU-S100, a novel stimulator of interferon genes (STING) pathway activator, and preliminary observations of ADU-S100 in combination with spartalizumab during the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) 33rd Annual Meeting
Entered into a research collaboration and exclusive license agreement with Eli Lilly and Company (Lilly) for the cGAS-STING pathway inhibitor program for autoimmune and inflammatory diseases
Presented preclinical data on the role of intratumoral STING activation by ADU-S100 in combination with checkpoint inhibitors in anti-tumor immunity at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2018 and the SITC (Free SITC Whitepaper) 33rd Annual Meeting
Published a paper titled, "Magnitude of Therapeutic STING Activation Determines CD8+ T-Cell Mediated Anti-Tumor Immunity," in the peer-reviewed journal, Cell Reports
APRIL

Presented preclinical data on BION-1301 for the treatment of IgA nephropathy at the American Society of Nephrology (ASN) Kidney Week 2018
Presented preclinical data on BION-1301 for the treatment of multiple myeloma at the AACR (Free AACR Whitepaper) Annual Meeting 2018 and 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition
Published a paper titled, "APRIL signaling via TACI mediates immunosuppression by T regulatory cells in multiple myeloma: therapeutic implications," in the peer-reviewed journal, Leukemia
Financial Results

Cash Position – Cash, cash equivalents and marketable securities totaled $277.9 million at December 31, 2018, compared to $349.7 million at December 31, 2017. In the fourth quarter of 2018, Aduro collected an $18.1 million cash tax refund from the Internal Revenue Service related to its carryback claim for 2017 losses.

Revenue – Revenues were $2.8 million for the fourth quarter of 2018 and $15.1 million for the full year 2018, compared to $3.8 million and $17.2 million, respectively, for the same periods in 2017. The decrease in revenue in both periods was primarily due to the adoption of the ASC 606 accounting standard on January 1, 2018, which resulted in a change in revenue recognition methodology under our Novartis collaboration agreement.

Expenses – Research and development expenses were $17.6 million for the fourth quarter of 2018 and $75.8 million for the full year 2018, compared to $22.9 million and $89.4 million, respectively, for the same periods in 2017. The decrease in research and development expenses for both periods was primarily due to lower expenses for our antibody and LADD programs.

General and administrative expenses were $9.0 million for the fourth quarter of 2018 and $36.0 million for the full year 2018, compared to $8.8 million and $33.8 million, respectively, for the same periods in 2017. The increase in general and administrative expenses for both periods was primarily due to higher professional services and consulting costs as well as stock-based compensation expense.

Loss on impairment of intangible assets was $4.0 million for the fourth quarter and full year 2018. This expense was recorded due to the discontinuation of one of our acquired early research programs.

Net Loss – Net loss for the fourth quarter and year ended December 31, 2018 was $26.3 million, or $0.33 per share, and $95.4 million, or $1.21 per share, respectively. This compared to net loss of $26.1 million, or $0.34 per share, and $91.9 million, or $1.26 per share, respectively, for the same periods in 2017. The increase in net loss for the year was primarily due to the income tax benefit recorded in 2017.