Category: Press Release

ARCA biopharma Announces Randomization of First European Patients in GENETIC-AF Phase 2B/3 Clinical Trial

Approximately Six Million Patients in Europe Estimated to Have Atrial Fibrillation

—————————————————————————————————–

Geographic Expansion of Trial Intended to Support European Regulatory Submissions, European Partnering Discussions and Enrollment

Westminster, CO, October 6, 2016 – ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to the development of genetically-targeted therapies for cardiovascular diseases, today announced the randomization of the first European patients in the GENETIC-AF Phase 2B/3 clinical trial.  GENETIC-AF, which has been enrolling patients in the United States and Canada, is evaluating GencaroTM as a potential treatment for atrial fibrillation (AF).  Last week, ARCA hosted a GENETIC-AF European Investigator Meeting which brought together European physicians and research coordinators for study-specific training and information sharing.  It is estimated that approximately six million patients in Europe have atrial fibrillation.

ARCA is evaluating Gencaro, a pharmacologically unique beta-blocker and mild vasodilator, as a potential treatment for AF in the GENETIC-AF Phase 2B/3 clinical trial.  ARCA has identified common genetic variations that it believes predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted therapy for the prevention of atrial fibrillation.

“We are excited to expand the GENETIC-AF trial to European investigative sites that have expressed interest in participating in the on-going trial,” said Michael R. Bristow, President and Chief Executive Officer.  “We expect that the additional investigative sites in certain European countries will support the latter part of Phase 2B, and potentially the Phase 3 stage, of the trial.  We believe inclusion of European investigative sites may also support potential European regulatory submissions and on-going business development activity.”

Based on the current enrollment rate, the Company expects to randomize at least 150 patients into the trial by the end of 2016. The GENETIC-AF Data Safety Monitoring Board (DSMB) will conduct an interim efficacy, safety and futility analysis of data from at least 150 patients with evaluable data. The Company expects the outcome of this interim analysis in the second quarter of 2017.

About ARCA biopharma

 ARCA biopharma is dedicated to developing genetically-targeted therapies for cardiovascular diseases through a precision medicine approach to drug development.  The Company’s lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for atrial fibrillation.  ARCA has identified common genetic variations that it believes predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted atrial fibrillation prevention treatment.  ARCA has a collaboration with Medtronic, Inc. for support of the GENETIC-AF trial.  For more information, please visit www.arcabio.com.

Safe Harbor Statement

 This press release contains “forward-looking statements” for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995.  These statements include, but are not limited to, statements regarding, potential timing for patient enrollment in the GENETIC-AF trial, potential timeline for GENETIC-AF trial activities, the potential for any European regulatory submissions for Gencaro, the potential outcome of the GENETIC-AF Phase 2B interim analysis, the sufficiency of the Company’s capital to support its operations, the potential for genetic variations to predict individual patient response to Gencaro, Gencaro’s potential to treat atrial fibrillation, future treatment options for patients with atrial fibrillation, and the potential for Gencaro to be the first genetically-targeted atrial fibrillation prevention treatment.  Such statements are based on management’s current expectations and involve risks and uncertainties.  Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: the Company’s financial resources and whether they will be sufficient to meet the Company’s business objectives and operational requirements; results of earlier clinical trials may not be confirmed in future trials, the protection and market exclusivity provided by the Company’s intellectual property; risks related to the drug discovery and the regulatory approval process; and, the impact of competitive products and technological changes.  These and other factors are identified and described in more detail in ARCA’s filings with the SEC, including without limitation the Company’s annual report on Form 10-K for the year ended December 31, 2015, and subsequent filings.  The Company disclaims any intent or obligation to update these forward-looking statements.

 Investor & Media Contact:

Derek Cole

720.940.2163

derek.cole@arcabio.com

###

ARCA biopharma to Present at the Dawson James Securities Second Annual Growth Stock Conference

 

Westminster, CO, October 4, 2016 – ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, today announced that it will present a corporate overview at the Dawson James Securities Second Annual Growth Stock Conference.

 

Date:                                         Thursday, October 20, 2016

Time:                                         1:15 p.m. (Eastern Time)

Presenter:                               Thomas Keuer, Chief Operating Officer

Webcast/Presentation:    The live webcast and accompanying slides will be available at http://wsw.com/webcast/dawson2/abio and https://arcabio.com/investors/investor-presentations/ , respectively.

Replay Information:          A replay is expected to be available approximately two hours after the presentation on October 20, 2016, and will remain available for 90 days.  The replay can be accessed at http://wsw.com/webcast/dawson2/abio.

 

About ARCA biopharma

 ARCA biopharma is dedicated to developing genetically-targeted therapies for cardiovascular diseases through a precision medicine approach to drug development.  The Company’s lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for atrial fibrillation.  ARCA has identified common genetic variations that it believes predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted atrial fibrillation prevention treatment.  ARCA has a collaboration with Medtronic, Inc. for support of the GENETIC-AF trial.  For more information, please visit www.arcabio.com.

 Safe Harbor Statement

This press release and the anticipated presentation contain “forward-looking statements” for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995.  These statements include, but are not limited to, statements regarding, potential timing for patient enrollment in the GENETIC-AF trial, potential timeline for GENETIC-AF trial activities, the potential outcome of the GENETIC-AF Phase 2B interim analysis, the sufficiency of the Company’s capital to support its operations, the potential for genetic variations to predict individual patient response to Gencaro, Gencaro’s potential to treat atrial fibrillation, future treatment options for patients with atrial fibrillation, and the potential for Gencaro to be the first genetically-targeted atrial fibrillation prevention treatment.  Such statements are based on management’s current expectations and involve risks and uncertainties.  Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: the Company’s financial resources and whether they will be sufficient to meet the Company’s business objectives and operational requirements; results of earlier clinical trials may not be confirmed in future trials, the protection and market exclusivity provided by the Company’s intellectual property; risks related to the drug discovery and the regulatory approval process; and, the impact of competitive products and technological changes.  These and other factors are identified and described in more detail in ARCA’s filings with the SEC, including without limitation the Company’s annual report on Form 10-K for the year ended December 31, 2015, and subsequent filings.  The Company disclaims any intent or obligation to update these forward-looking statements.

 

Investor & Media Contact:

Derek Cole

720.940.2163

derek.cole@arcabiopharma.com

###

ARCA biopharma Announces 100th Patient Randomized into the Genetic-AF Phase 2B/3 Clinical Trial

ARCA Biopharma announces 100th patient randomized into the GENETIC-AF Phase 2B/3 clinical trial

 

GENETIC-AF Evaluating Gencaro as Potentially First Genetically-Targeted 

Treatment for Atrial Fibrillation

 

Outcome of Interim Efficacy Analysis Anticipated in the Second Quarter of 2017

Westminster, CO, August 18, 2016 – ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company applying a precision medicine approach to developing genetically-targeted therapies for cardiovascular diseases, today announced that the 100thpatient has been randomized into GENETIC-AF, a seamless design Phase 2B/3 clinical trial evaluating GencaroTM (bucindolol hydrochloride) as a potential treatment for atrial fibrillation. This represents two-thirds of the minimum number of patients needed for a pre-specified Phase 2B interim efficacy analysis.

Based on the current enrollment rate, the Company expects to randomize at least 150 patients into the trial by the end of 2016. The GENETIC-AF Data Safety Monitoring Board (DSMB) will conduct an interim efficacy, safety and futility analysis of data from at least 150 patients with evaluable data. The Company expects the outcome of this interim analysis in the second quarter of 2017.

GENETIC-AF Clinical Trial

GENETIC-AF is a Phase 2B/Phase 3, multi-center, randomized, double-blind, adaptive design clinical trial comparing the safety and efficacy of Gencaro to Toprol-XL (metoprolol succinate) for the treatment of atrial fibrillation (AF) in approximately 620 patients. Eligible patients will have heart failure with reduced left ventricular ejection fraction (HFREF), have a history of paroxysmal AF (episodes lasting 7 days or less) or persistent AF (episodes lasting more than 7 days and less than 1 year) in the past 6 months, and have the beta-1 389 arginine homozygous genotype that the Company believes responds most favorably to Gencaro.  The primary endpoint of the study is time to first event of symptomatic AF/atrial flutter (AFL) or all-cause mortality.  The trial is currently enrolling patients in the United States and Canada.

About ARCA biopharma

 ARCA biopharma is dedicated to developing genetically-targeted therapies for cardiovascular diseases through a precision medicine approach to drug development. The Company’s lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for atrial fibrillation.  ARCA has identified common genetic variations that it believes predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted atrial fibrillation prevention treatment.  ARCA has a collaboration with Medtronic, Inc. for support of the GENETIC-AF trial.  For more information, please visit www.arcabiopharma.com.

Safe Harbor Statement

This press release contains “forward-looking statements” for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding, potential timing for patient enrollment in the GENETIC-AF trial, potential timeline for GENETIC-AF trial activities and related recommendations of the DSMB, the sufficiency of the Company’s capital to support its operations, the potential for genetic variations to predict individual patient response to Gencaro, Gencaro’s potential to treat atrial fibrillation, future treatment options for patients with atrial fibrillation, and the potential for Gencaro to be the first genetically-targeted atrial fibrillation prevention treatment. Such statements are based on management’s current expectations and involve risks and uncertainties.  Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: the Company’s financial resources and whether they will be sufficient to meet the Company’s business objectives and operational requirements; results of earlier clinical trials may not be confirmed in future trials, the protection and market exclusivity provided by the Company’s intellectual property; risks related to the drug discovery and the regulatory approval process; and, the impact of competitive products and technological changes.  These and other factors are identified and described in more detail in ARCA’s filings with the Securities and Exchange Commission, including without limitation the Company’s annual report on Form 10-K for the year ended December 31, 2015, and subsequent filings.  The Company disclaims any intent or obligation to update these forward-looking statements.

 

Investor & Media Contact:

Derek Cole

720.940.2163

derek.cole@arcabiopharma.com

ARCA Biopharma Announces Second Quarter 2016 Financial Results and Provides Business Update

Outcome of GENETIC-AF Clinical Trial Interim Efficacy Analysis Anticipated

in the Second Quarter of 2017

 

GENETIC-AF Evaluating Gencaro as Potentially First Genetically-Targeted

Treatment for Atrial Fibrillation

 

Westminster, CO, August 9, 2016 – ARCA biopharma, Inc. (Nasdaq: ABIO), a biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases, today reported financial results for the quarter ended June 30, 2016, and provided a business update.

“We believe that our precision medicine approach to drug development, tailoring medical treatment to the genetic characteristics of patient subpopulations, can enable more effective therapies, improve patient outcomes and reduce healthcare costs,” commented Dr. Michael Bristow, ARCA’s President and CEO.  “Our on-going GENETIC-AF Phase 2B/3 clinical trial is evaluating GencaroTM as potentially the first genetically-targeted treatment for atrial fibrillation.  We believe enrollment is on track for having a sufficient number of patients by the end of this year for the DSMB Phase 2B interim efficacy analysis, the outcome of which we expect in the second quarter of 2017.  With our experienced team, strong balance sheet, supportive collaborations with Medtronic and LabCorp, and high quality investors, we feel ARCA is well positioned as a late stage cardiovascular company dedicated to serving patients with these chronic diseases.”

Second Quarter 2016 Summary Financial Results

Cash, cash equivalents and marketable securities totaled $31.4 million as of June 30, 2016, compared to $38.8 million as of December 31, 2015.  The Company believes that its current cash, cash equivalents and marketable securities will be sufficient to fund its operations, at its projected cost structure, through at least the end of 2017.  ARCA had approximately 9.1 million outstanding shares of common stock as of June 30, 2016.

Research and development (R&D) expense for the three months ended June 30, 2016 was $2.9 million compared to $1.7 million for the corresponding period of 2015, an increase of approximately $1.2 million.  R&D expense for the six months ended June 30, 2016 was $5.5 million compared to $3.5 million for the corresponding period of 2015, an increase of approximately $2.1 million.  The increase in R&D expense in the three and six month periods ended June 30, 2016 is due, primarily, to the increased clinical expense of the GENETIC-AF clinical trial.  The Company expects R&D expense in 2016 to be higher than 2015 as it activates new clinical sites and enrolls additional patients in the GENETIC-AF clinical trial.

General and administrative (G&A) expense for the three months ended June 30, 2016 was $1.0 million compared to $1.0 million for the corresponding period in 2015.  G&A expense was $2.1 million for the six months ended June 30, 2016 as compared to $2.0 million for the corresponding period in 2015, an increase of $93,000.  The increase in G&A expense is due, primarily, to increased franchise taxes.  The Company expects G&A expense in 2016 will be higher than in 2015 as the Company increases administrative activities to support the GENETIC-AF clinical trial.

Total operating expense for the three months ended June 30, 2016 was $3.9 million compared to $2.7 million for the corresponding period in 2015.  Total operating expense for the six months ended June 30, 2016 was $7.6 million compared to $5.5 million for the corresponding period in 2015.

Net loss was $3.9 million, or $0.43 per share, for the second quarter of 2016, compared to $2.7 million, or $0.69 per share, for the second quarter of 2015.  For the first half of 2016, net loss was $7.5 million, or $0.83 per share, compared to $5.5 million, or $1.57 per share, for the first half of 2015.

GENETIC-AF Clinical Trial

GENETIC-AF is a Phase 2B/Phase 3, multi-center, randomized, double-blind, adaptive design clinical trial comparing the safety and efficacy of Gencaro to Toprol-XL (metoprolol succinate) for the treatment of atrial fibrillation (AF) in approximately 620 patients.  Eligible patients will have heart failure with reduced left ventricular ejection fraction (HFREF), a history of paroxysmal AF (episodes lasting 7 days or less) or persistent AF (episodes lasting more than 7 days and less than 1 year) in the past 6 months, and the beta-1 389 arginine homozygous genotype that the Company believes responds most favorably to Gencaro.  The primary endpoint of the study is time to first event of symptomatic AF/atrial flutter (AFL) or all-cause mortality.  The combined Phase 2B/Phase 3 trial is designed for 90 percent power at a p-value of less than 0.01 significance level to detect a 25 percent reduction in the primary endpoint for patients in the Gencaro arm compared to patients in the Toprol-XL arm.  The trial is currently enrolling patients in the United States and Canada.

 

The GENETIC-AF Data and Safety Monitoring Board (DSMB) will conduct a pre-specified interim analysis of study endpoints for efficacy, safety and futility to recommend whether or not the trial should proceed to Phase 3.  The DSMB will make its recommendation based on a predictive probability analysis of certain trial data after at least 150 patients have evaluable endpoint data.  An enrolled patient has evaluable endpoint data either when they experience their first endpoint event, or after they complete the 24-week follow up period.  The DSMB interim analysis will focus on analyses of the AF/AFL endpoints in the trial using both clinical-based intermittent monitoring and device-based continuous monitoring techniques.  Should the DSMB interim analysis indicate that the data are consistent with pre-trial statistical assumptions and the potential for achieving statistical significance for the Phase 3 endpoint, the DSMB may recommend that the study continue to Phase 3.  The DSMB may also recommend that the study be halted.  Based on the current enrollment rate, the Company expects to enroll at least 150 patients in the trial by the end of 2016.  The Company expects the outcome of the interim analysis in the second quarter of 2017.

 

About ARCA biopharma

 

ARCA biopharma is dedicated to developing genetically-targeted therapies for cardiovascular diseases through a precision medicine approach to drug development.  The Company’s lead product candidate, GencaroTM (bucindolol hydrochloride), is an investigational, pharmacologically unique beta-blocker and mild vasodilator being developed for atrial fibrillation.  ARCA has identified common genetic variations that it believes predict individual patient response to Gencaro, giving it the potential to be the first genetically-targeted atrial fibrillation prevention treatment.  ARCA has a collaboration with Medtronic, Inc. for support of the GENETIC-AF trial.  For more information, please visit www.arcabiopharma.com.

 

Safe Harbor Statement

 

This press release contains “forward-looking statements” for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995.  These statements include, but are not limited to, statements regarding, potential timing for patient enrollment in the GENETIC-AF trial, potential timeline for GENETIC-AF trial activities and related recommendations of the DSMB, the sufficiency of the Company’s capital to support its operations, the potential for genetic variations to predict individual patient response to Gencaro, Gencaro’s potential to treat atrial fibrillation, future treatment options for patients with atrial fibrillation, and the potential for Gencaro to be the first genetically-targeted atrial fibrillation prevention treatment.  Such statements are based on management’s current expectations and involve risks and uncertainties.  Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, the risks and uncertainties associated with: the Company’s financial resources and whether they will be sufficient to meet the Company’s business objectives and operational requirements; results of earlier clinical trials may not be confirmed in future trials, the protection and market exclusivity provided by the Company’s intellectual property; risks related to the drug discovery and the regulatory approval process; and, the impact of competitive products and technological changes.  These and other factors are identified and described in more detail in ARCA’s filings with the SEC, including without limitation the Company’s annual report on Form 10-K for the year ended December 31, 2015, and subsequent filings.  The Company disclaims any intent or obligation to update these forward-looking statements.

 

Investor & Media Contact:

Derek Cole

720.940.2163

derek.cole@arcabiopharma.com

###

(Tables Follow)

 

 

 

ARCA BIOPHARMA, INC.

BALANCE SHEET DATA

(in thousands)

                                                           

June 30, 2016           December 31, 2015

Cash, cash equivalents and marketable securities $31,366 $38,802
Working capital $25,038 $37,412
Total assets $32,635 $39,574
Total stockholders’ equity $30,891 $38,070

 

 

 

ARCA BIOPHARMA, INC.

STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(unaudited)

  Three Months Ended     Six Months Ended  
  June 30,     June 30,  
2016       2015     2016     2015  
  (in thousands, except share and per share amounts)  
Costs and expenses:
Research and development $ 2,913 $ 1,747 $ 5,507 $ 3,454
General and administrative 1,028 972 2,102 2,009
Total costs and expenses 3,941 2,719 7,609 5,463
Loss from operations (3,941 ) (2,719 ) (7,609 ) (5,463 )
Interest and other income 47 2 68 3
Interest expense (2 ) (3 )
Net loss $ (3,894 ) $ (2,719 ) $ (7,541 ) $ (5,463 )
Change in unrealized gain on marketable securities 7 13
Comprehensive loss $ (3,887 ) $ (2,719 ) $ (7,528 ) $ (5,463 )
Net loss per share:
Basic and diluted $ (0.43 ) $ (0.69 ) $ (0.83 ) $ (1.57 )
Weighted average shares outstanding:
Basic and diluted 9,065,922 3,951,895 9,059,554 3,490,477