On February 18, 2020 CANbridge Pharmaceuticals Inc., a biopharmaceutical company developing innovative drug candidates to treat underserved medical conditions in China and other markets, reported the completion of a US$98 million Series D financing (Press release, CANbridge Life Sciences, FEB 18, 2020, View Source [SID1234554425]). The round was led by General Atlantic, a leading global growth equity firm, and WuXi AppTec. Each firm also has the option to invest up to an additional $10 million, upon the satisfaction of certain conditions by CANbridge. Additional participating investors in this round include RA Capital Management, Hudson Bay Capital Management, YuanMing Prudence Fund and Tigermed. The proceeds, the majority of which come from global investors, will be used to accelerate and expand the CANbridge rare disease pipeline through internal development and external partnerships, further build CANbridge’s commercial infrastructure and capabilities in China and supplement working capital.

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CANbridge has a deep rare disease pipeline, including Hunterase, for which it filed a New Drug Application (NDA) in China and has plans to commercialize the drug this year. In addition, CANbridge recently entered into the commercial stage with two approved oncology products, Caphosol, in mainland China, and NERLYNX (neratinib), in Hong Kong.

"That most of this capital raise comes from global life science investors, indicates the level of enthusiasm CANbridge is generating on the world stage as an industry leader in rare disease in China," said James Xue, PhD, Founder, Chairman and CEO, CANbridge Pharmaceuticals. "We look to the future, as CANbridge continues to advance as a commercial-stage company with the powerful backing and endorsement of such investors."

"CANbridge is an early mover and a leader in China’s rare disease market," said Lefei Sun, Managing Director and Head of Healthcare for China at General Atlantic. "The company has built a rich pipeline of candidates, and we believe that CANbridge is well-positioned to capture the substantial market opportunity in the Chinese biotechnology space. We are excited to partner with the CANbridge team to support the company’s vision to bring innovative therapeutics to patients across China."

"CANbridge is developing a compelling and deep pipeline of assets, focused around directly addressing the unmet needs of patients with rare genetic diseases in China and around the world," said Derek DiRocco, PhD, Principal, RA Capital Management. "The leadership team at CANbridge is equipped with an impressive background, anchored in the development of rare disease therapeutics, and is poised to build the regulatory and commercial infrastructure necessary to significantly advance the rare disease segment of China’s biotech industry. RA Capital is excited to partner with CANbridge, and shares in leadership’s vision of building an innovative biotech company that aims to deliver effective therapeutics to patients in need."

On February 18, 2020 Cellectis (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited allogeneic CAR T-cells (UCART), and Servier, an international pharmaceutical company, reported the execution of a binding term sheet to enter into an amendment to the agreement initially signed between the two companies in 2014 (Press release, Cellectis, FEB 18, 2020, View Source [SID1234554424]).

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Under the term sheet, Cellectis shall grant to Servier, through an amendment to the agreement, an expanded exclusive worldwide license to develop and commercialize all next generation gene-edited allogeneic CAR T-cell products targeting CD19, including rights to ALLO-501A, an anti-CD19 candidate in which the rituximab recognition domains have been removed, either directly or through its US sublicensee Allogene Therapeutics.

In this amendment, financial terms will be improved to include an additional USD 27.6 million (EUR 25 million) upfront payment, as well as up to USD 410 million (EUR 370 million) in clinical and commercial milestones. The royalty rate will be increased from tiered high single-digit royalties to flat low double-digit royalties based on net sales of products.

In addition, pursuant to the amendment, Cellectis shall regain exclusive control over the five undisclosed allogeneic CAR T-cell targets previously covered by the initial agreement.

The amendment will be effective upon its execution.

"This amendment to our license agreement with Servier provides to Cellectis an attractive economic upside to product candidates targeting CD19 and enriches our proprietary portfolio of targets," said Dr. André Choulika, Chairman and CEO, Cellectis. "We are committed to positioning Servier and Allogene for streamlined success, so the CD19-directed products have the potential to reach patients faster, while also providing Cellectis the means to expand our proprietary product pipeline."

About UCART19/ALLO-501 and ALLO-501A

UCART19/ALLO-501 and ALLO-501A are two anti-CD19 allogeneic CAR-T product candidates being jointly developed under a clinical development collaboration between Servier and Allogene Therapeutics based on an exclusive license granted by Cellectis to Servier.

Such products utilize Cellectis’ technologies, including TALEN gene editing technology pioneered and controlled by Cellectis. Servier grants to Allogene exclusive rights to UCART19 in the US while Servier retains exclusive rights for all other countries.

On February 18, 2020 Cancer Research UK reported that a genetic study has found evidence to suggest that women who take statins in the long term could be less likely to develop ovarian cancer (Press release, Cancer Research UK, FEB 18, 2020, View Source [SID1234554423]).

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"Our findings open up the possibility of repurposing a cheap drug to help prevent ovarian cancer – especially in women who are at a higher risk." – Professor Richard Martin
The same result was also found in women who carry the BRCA1/2 gene fault. Having the BRCA1/2 fault puts women at a higher risk of ovarian cancer than the general population*.

The research published in JAMA studied genes and the extent to which they inhibit the enzyme HMG-CoA reductase – which is responsible for regulating cholesterol in the body – and is the exact enzyme targeted by statin drugs to reduce cholesterol.

While the study suggests that statins could lower ovarian cancer risk, more research needs to be done specifically looking at their use and impact on women’s risk of developing the disease.

The researchers based at the University of Bristol looked at 63,347 women between the ages of 20 and 100 years old, of whom 22,406 had ovarian cancer. They also looked at an additional 31,448 women who carried the BRCA1/2 fault, of whom 3,887 had ovarian cancer. The study used an approach called Mendelian randomization, which involves analysing the genetic data from thousands of people.

Statins may protect against the development of ovarian cancer because they’ve been shown to induce apoptosis – one of the body’s ways of getting rid of old, faulty or infected cells – and to stop tumours from growing in laboratory studies. Another line of thought is that statins lower circulating cholesterol, which helps regulate cell growth, though this research suggests that lower circulating cholesterol was not the method by which statins may reduce ovarian cancer risk.

The findings suggest that long-term statin use could be associated with an estimated 40% reduction in ovarian cancer risk in the general population, although the estimate comes from looking at gene variation rather than statins themselves, and the exact mechanism by which these genes are associated with lower ovarian cancer risk is unclear.

Ovarian cancer is the 6th most common cancer in women in the UK. There are around 7,400 cases each year**, and out of those with a known stage at diagnosis, almost 6 in 10 are diagnosed at a late stage***. Around 4,100 women die from the disease every year in the UK.

There is no test that reliably picks up ovarian cancer at an early stage, so chemoprevention could be an important approach to saving lives.

Professor Richard Martin, from the University of Bristol, said: "Our findings open up the possibility of repurposing a cheap drug to help prevent ovarian cancer – especially in women who are at a higher risk. It’s incredibly interesting that women whose bodies naturally inhibit the enzyme targeted by statins have a lower risk of ovarian cancer, but we don’t recommend anyone rushes to take statins specifically to reduce ovarian cancer risk because of this study.

"It’s a promising result and I hope it sparks more research and trials into statins to demonstrate conclusively whether or not there’s a benefit."

Dr Rachel Orritt, Cancer Research UK’s health information manager, said: "This study is a great first step to finding out if statins could play a role in lowering ovarian cancer risk, and justifies future research into this area.

"But there’s not yet enough evidence to know if statins themselves could reduce the risk of developing ovarian cancer safely. And it’s important to remember that the risk of developing ovarian cancer depends on many things including age, genetics and environmental factors. Speak to your doctor first if you have any concerns about your risk."

On February 18, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported financial results and business highlights for the fourth quarter and full year ended December 31, 2019 (Press release, bluebird bio, FEB 18, 2020, View Source [SID1234554422]).

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"2019 was truly a transformative year for bluebird, with our first commercial product now launched in Europe and exciting progress across our first four clinical programs and pipeline," said Nick Leschly, chief bluebird. "Notably, our data in SCD continues to build, and at the ASH (Free ASH Whitepaper) annual meeting in December we presented data that showed a 99% reduction in the annualized rate of vaso-occlusive crises (VOC) and acute chest syndrome (ACS) in HGB-206 Group C patients with history of VOCs and ACS who had at least six months follow-up. In β-thalassemia, the consistency with which patients who do not have a β0/β0 genotype in our Northstar-2 (HGB-207) study are achieving transfusion independence is very encouraging – and we’re starting to see indications that we may be able to see similar outcomes with many patients with β0/β0 genotypes as well in our Northstar-3 (HGB-212 study). These data put us in a strong position as we progress our European launch, currently underway in Germany. At the end of 2019, we also announced positive top-line data from the pivotal KarMMa study of ide-cel. We and our partners at BMS look forward to submitting these data to the FDA in the first half of this year. Amidst all of our progress in 2019, our birds demonstrated time and again their dedication to patients and ability to meet and learn from the many challenges we have faced along the way. I look forward to facing the challenges of 2020 with this amazing flock."

Recent Highlights:

TRANSFUSION-DEPENDENT β-THALASSEMIA

LAUNCH IN GERMANY – In January 2020, bluebird bio announced the launch of ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene), a gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available in Germany. The company signed its first agreements with statutory health insurances utilizing bluebird’s innovative value-based payment model and providing coverage for ZYNTEGLO for up to 50% of patients in Germany, and the first qualified treatment center was established at University Hospital of Heidelberg to provide ZYNTEGLO to patients. The company anticipates treating the first commercial patient in the first half of 2020.
UPDATED LENTIGLOBIN FOR β-THALASSEMIA DATA – At the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting in December 2019, bluebird bio presented new data from its studies of LentiGlobin gene therapy for β-thalassemia (betibeglogene autotemcel) in patients with TDT: long-term data from the completed Phase 1/2 Northstar study (HGB-204), updated data from the Phase 3 Northstar-2 study (HGB-207) in patients with non-β0/β0 genotypes, and updated data from the Phase 3 Northstar-3 study (HGB-212) in patients with β0/β0 genotypes or an IVS-I-110 mutation.
BIOLOGICS LICENSE APPLICATION (BLA) SUBMISSION – bluebird bio has initiated its rolling BLA submission of LentiGlobin for β-thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for β-thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.
NORTHSTAR-2 FINAL INFUSION – In January 2020, the final patient enrolled in the pediatric cohort of Northstar-2 (HGB-207) was infused with LentiGlobin for β-thalassemia.
SICKLE CELL DISEASE (SCD)

HGB-211 – bluebird bio is announcing today plans to launch HGB-211, the company’s second Phase 3 study of LentiGlobin for sickle cell disease (SCD). This study is expected to enroll approximately 18 patients ages 2-14 years with SCD and elevated stroke risk, stroke being one of the most severe complications during childhood and adolescence. The primary endpoint of the study will be transcranial doppler response without transfusion. HGB-211 is in addition to the company’s previously announced Phase 3 study (HGB-210) and is intended to support potential approval of LentiGlobin for SCD in pediatric patients at elevated stroke risk. HGB-211 is expected to begin enrolling patients in 2020.
UPDATED LENTIGLOBIN FOR SCD DATA – At the ASH (Free ASH Whitepaper) meeting in December 2019, bluebird bio presented new data from patients in Groups A, B and C in the Phase 1/2 HGB-206 study in patients with SCD. Group C patients are being treated under a study protocol utilizing hematopoietic stem cell (HSC) mobilization and apheresis with plerixafor, and a refined manufacturing process to increase vector copy number and engraftment potential of gene-modified HSCs. The company also disclosed that the target enrollment in HGB-206 has been achieved.
MULTIPLE MYELOMA

KARMMA TOPLINE – In December 2019, Bristol-Myers Squibb and bluebird bio announced positive top-line results from the pivotal Phase 2 KarMMa study of ide-cel in relapsed and refractory multiple myeloma. The study met its primary endpoint and key secondary endpoint, demonstrating deep and durable responses in a heavily pre-treated multiple myeloma patient population. Safety results are consistent with the data presented in CRB-401 study.
BB21217 DATA – At the ASH (Free ASH Whitepaper) meeting in December 2019, bluebird bio and Bristol-Myers Squibb presented updated data from ongoing CRB-402 Phase 1 study of BCMA-targeted CAR T cell therapy bb21217 in relapsed and refractory multiple myeloma. The dose escalation part of CRB-402 is complete, and the dose expansion part of the study is ongoing.
COMPANY

FORTY SEVEN COLLABORATION –In November 2019, bluebird bio and Forty Seven announced that they have entered into a research collaboration to pursue clinical proof-of-concept for Forty Seven’s novel antibody-based conditioning regimen, FSI-174 (anti-cKIT antibody) plus magrolimab (anti-CD47 antibody), with bluebird’s ex vivo lentiviral vector hematopoietic stem cell (LVV HSC) gene therapy platform. Under the terms of the agreement, bluebird bio will provide its ex vivo LVV HSC gene therapy platform and Forty Seven will contribute its innovative antibody-based conditioning regimen for the collaboration.
Upcoming Anticipated Milestones:

Regulatory
Submission of a BLA to the U.S. FDA for ide-cel in patients with relapsed and refractory multiple myeloma in the first half of 2020, in partnership with Bristol-Myers Squibb.
Submission of a BLA to the U.S. FDA and a Marketing Authorization Application to the European Medicines Agency for Lenti-D in patients with cerebral adrenoleukodystrophy by the end of 2020.
Clinical
Submission for presentation of ide-cel clinical data from the KarMMa study in the first half of 2020, in partnership with Bristol-Myers Squibb.
Submission for presentation of ide-cel clinical data from the CRB-401 study in 2020, in partnership with Bristol-Myers Squibb.
Initiation of the Phase 3 HGB-210 study of LentiGlobin for SCD in patients with a history of vaso-occlusive crises in the first half of 2020.
Initiation of the Phase 3 HGB-211 study of LentiGlobin for SCD in patients at risk of stroke in 2020.
Updated data presentation from ALD-102 in patients with CALD by the end of 2020.
Updated data presentation from the Northstar-2 (HGB-207) clinical study in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes by the end of 2020.
Updated data presentation from the Northstar-3 (HGB-212) clinical study in patients with TDT and a β0/β0 genotype or an IVS-I-110 mutation by the end of 2020.
Updated data presentation from HGB-206 clinical study in patients with SCD by the end of 2020.
Commercial and Foundation Building
ZYNTEGLO first commercial patients treated in the first half of 2020.
ZYNTEGLO access and reimbursement in additional EU countries established by the end of 2020.
Fourth Quarter and Full Year 2019 Financial Results

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2019 and December 31, 2018 were $1.24 billion and $1.89 billion, respectively. The decrease in cash, cash equivalents and marketable securities is primarily related to cash used in support of ordinary course operating and commercial-readiness activities.
Revenues: Collaboration and license and royalty revenues were $10.0 million for the three months ended December 31, 2019 compared to $19.2 million for the three months ended December 31, 2018. Collaboration and license and royalty revenues were $44.7 million for the year ended December 31, 2019 compared to $54.6 million for the year ended December 31, 2018. The decrease in both periods was primarily attributable to a decrease in collaboration revenue under our arrangement with Bristol-Myers Squibb, partially offset by an increase in license and royalty revenue.
R&D Expenses: Research and development expenses were $161.8 million for the three months ended December 31, 2019 compared to $119.7 million for the three months ended December 31, 2018. Research and development expenses were $582.4 million for the year ended December 31, 2019 compared to $448.6 million for the year ended December 31, 2018. The increase in both periods was primarily driven by costs incurred to advance and expand the company’s pipeline.
SG&A Expenses: Selling, general and administrative expenses were $76.2 million for the three months ended December 31, 2019 compared to $53.5 million for the three months ended December 31, 2018. Selling, general and administrative expenses were $271.4 million for the year ended December 31, 2019 compared to $174.1 million for the year ended December 31, 2018. The increase in both periods was largely attributable to costs incurred to support the company’s ongoing operations and growth of its pipeline as well as commercial-readiness activities.
Net Loss: Net loss was $223.3 million for the three months ended December 31, 2019 compared to $149.0 million for the three months ended December 31, 2018. Net loss was $789.6 million for the year ended December 31, 2019 compared to $555.6 million for the year ended December 31, 2018.
LentiGlobin for β-thalassemia Safety

Non-serious adverse events (AEs) observed during the HGB-204, HGB-207 and HGB-212 clinical studies that were attributed to LentiGlobin for β-thalassemia were hot flush, dyspnoea, abdominal pain, pain in extremities, thrombocytopenia, leukopenia, neutropenia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to LentiGlobin for β-thalassemia for TDT.

Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease.

With more than five years of follow-up to date, there have been no new unexpected safety events, no deaths, no graft failure and no cases of vector-mediated replication competent lentivirus or clonal dominance. In addition, there have been no new reports of veno-occlusive liver disease (VOD) as of the data cutoff presented at ASH (Free ASH Whitepaper).

About LentiGlobin for β-Thalassemia (betibeglogene autotemcel)

The European Commission granted conditional marketing authorization for LentiGlobin for β-thalassemia, to be marketed as ZYNTEGLO (autologous CD34+ cells encoding βA-T87Q-globin gene) gene therapy, for patients 12 years and older with TDT who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or significantly reduced hemoglobin (Hb). In order to survive, people with TDT maintain Hb levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload.

LentiGlobin for β-thalassemia adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived hemoglobin, at levels that may eliminate or significantly reduce the need for transfusions.

The conditional marketing authorization for ZYNTEGLO is only valid in the 28 member states of the EU as well as Iceland, Liechtenstein and Norway. For details, please see the Summary of Product Characteristics (SmPC).

The U.S. Food and Drug Administration granted LentiGlobin for β-thalassemia Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.

bluebird bio has initiated its rolling BLA submission of LentiGlobin for β-thalassemia for approval in the U.S. and is engaged with the FDA in discussions regarding the requirements and timing of certain information to be provided in the BLA, including information regarding various release assays for LentiGlobin for β-thalassemia. Subject to these ongoing discussions, the company is currently planning to complete the BLA submission in the second half of 2020.

LentiGlobin for β-thalassemia continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more information about the ongoing clinical studies, visit www.northstarclinicalstudies.com or clinicaltrials.gov and use identifier NCT02906202 for Northstar-2 (HGB-207), NCT03207009 for Northstar-3 (HGB-212).

bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for β-thalassemia. For more information visit: View Source or clinicaltrials.gov and use identifier NCT02633943 for LTF-303.

On February 18, 2020 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, 2019 and provided a corporate update (Press release, Arca biopharma, FEB 18, 2020, View Source [SID1234554421]).

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Dr. Michael Bristow, ARCA’s President and Chief Executive Officer, commented, "During 2019, we continued to advance development of Gencaro as a potential genetically-targeted treatment for heart failure patients with atrial fibrillation. In published or submitted for presentation/publication material, we have identified important new effectiveness evidence from the GENETIC-AF trial that have enhanced our understanding of the clinical profile of Gencaro. These data have allowed us to broaden the design of the single, pivotal Phase 3 clinical trial that was developed after consultation with the U.S. Food and Drug Administration via a Special Protocol Assessment agreement. There are currently no FDA approved drug therapies indicated for the treatment of atrial fibrillation in heart failure patients with left ventricular ejection fraction values greater than 40%. Based on our clinical data to date, we believe Gencaro has the potential to help address this substantial unmet medical need. We look forward to further evaluating the pharmacogenetic benefits of Gencaro for patients with our targeted genetic profile in PRECISION-AF, the Phase 3 clinical trial, which, subject to obtaining additional financing, we plan to initiate in the fourth quarter of 2020."

Pipeline Update – Second Half Review

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).

The U.S. Food and Drug Administration (FDA) issued a Special Protocol Assessment (SPA) agreement for a single Phase 3 clinical trial (PRECISION-AF) to examine Gencaro as a genetically-targeted therapy for the prevention of AF recurrence in certain heart failure patients.
PRECISION-AF is designed as a double-blind, active-controlled, multicenter, international, adaptive study comparing Gencaro with TOPROL-XL (metoprolol succinate) for the prevention of AF recurrence or all-cause mortality in approximately 400 heart failure patients who have left ventricular injection fraction (LVEF) values ≥ 40% and ≤ 55% and the genotype which ARCA believes responds best to Gencaro (ADRB1 Arg389Arg).
Clinical data from the GENETIC-AF Phase 2B clinical trial was published in the Journal of the American College of Cardiology: Heart Failure in May 2019 and was presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in September 2019. These data indicate that the response to Gencaro may be greater in HF patients with less severe left ventricular dysfunction, a patient population with no FDA approved drug therapeutic options for AF prevention or heart failure.
Subject to securing additional financing, ARCA anticipates initiating PRECISION-AF in the fourth quarter of 2020.
AB171 – a thiol-substituted isosorbide mononitrate being developed as a potential genetically-targeted treatment for HF and peripheral arterial disease (PAD).

Subject to securing additional financing, the Company anticipates conducting non-clinical studies to support a potential IND submission and initiation of clinical development in 2021.
2019 Summary Financial Results

Cash and cash equivalents were $8.4 million as of December 31, 2019, compared to $6.6 million as of December 31, 2018. ARCA believes that its current cash and cash equivalents will be sufficient to fund its operations, at its current cost structure, after giving effect to potential cost reductions, through the end of the third quarter of 2020.

Research and development (R&D) expenses for the year ended December 31, 2019 were $1.8 million compared to $4.2 million for 2018. The $2.4 million decrease was primarily due to decreased clinical expenses following the completion of the GENETIC-AF clinical trial. If the PRECISION-AF clinical trial is initiated in the second half of 2020, R&D expense in 2020 is expected to be higher than 2019.

General and administrative (G&A) expenses were $4.0 million for 2019 compared to $3.9 million for 2018. The Company expects G&A expenses in 2020 to be consistent with those in 2019 as it maintains administrative activities to support its ongoing operations.

Total operating expenses for 2019 were $5.8 million compared to $8.1 million for 2018. The decrease in total operating expenses was primarily attributable to the decrease in R&D expense due to the completion of the GENETIC-AF clinical trial.

Net loss was $5.5 million, or $4.15 per basic and diluted share, for 2019 compared to $7.9 million, or $10.31 per basic and diluted share, for 2018.

The Company will need to raise additional capital, and/or complete a partnership or other possible strategic transaction, to fund future operations and develop Gencaro or any other product candidates.