Category Archives: Biotech Companies CardioVascular

XyloCor and SmartCella enter into license agreement for use of the Extroducer to administer novel gene therapy XC001 to the heart

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  • The Extroducer® Infusion Catheter System® enables local delivery of XC001 to the heart without the need for surgery.
  • XC001 has achieved positive Phase 1/2 results in the EXACT Trial validating its transformative potential for treatment of refractory angina in patients who have exhausted available treatment options and have a debilitating quality-of-life.

XyloCor Therapeutics, Inc. (“XyloCor”), a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, and SmartWise, a unit of SmartCella Holding AB (“SmartCella”), have entered into a licensing agreement under which XyloCor has rights to the Extroducer® Infusion Catheter System ®, a first-in-class endovascular device designed to deliver advanced therapies directly into the heart [and hard-to-reach tissues] .  XyloCor plans to deploy the Extroducer to support catheter-based endocardial delivery of its lead gene therapy candidate, XC001 (encoberminogene rezmadenovec), in future clinical studies and commercial use.  

XyloCor and SmartCella enter into license agreement for use of the Extroducer to administer novel gene therapy XC001 to the heart

“This agreement with SmartCella will enable XyloCor to build upon its robust foundation of efficacy and safety data for XC001 by offering the potential for improved safety and ease of delivery without surgery via this novel catheter,” said Al Gianchetti, President and CEO of XyloCor. “Teaming up with SmartCella will help in our effort to optimize patient safety and tolerability while maintaining accurate delivery of XC001 to target areas in the heart for patients with refractory angina. It also opens up the potential to develop XC001 earlier in the coronary artery disease progression for even larger patient groups.”

XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple isoforms of vascular endothelial growth factor (VEGF). With the use of the Extroducer catheter, XyloCor can offer patients a better delivery option for local administration of XC001 directly to the heart, that is less invasive and eliminates potential risks associated with surgical administration.

“We welcome the Extroducer delivery of XC001 as it offers a more efficient method for gene therapy administration for patients with refractory angina,” said Timothy D. Henry MD, Interventional Cardiologist and Director of the Lindner Center, The Christ Hospital, Cincinnati, Ohio. “Preclinical models provide strong evidence that this approach will maintain, or even improve the efficacy when compared to surgical delivery and it should lower the risk of complications that may arise from surgical administration. I am looking forward to initiating the Phase 2b trial of XC001 in patients with refractory angina using this innovative administration approach.”

The recently published EXACT Phase 1/2 trial assessed the use of one-time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. In the EXACT trial, 42 patients with class II-IV angina were treated with XC001 directly administered to the heart following minimally invasive surgical access. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements of exercise duration, and angina frequency, due to a decrease in ischemic burden, as measured by Positron Emission Tomography (PET) imaging. Notably, six months after treatment 43% of patients had no chest pain with ordinary activities and 58% reported no angina episodes at 12-month clinical follow up. XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug.  The Phase 2b trial will be a randomized double-blinded study assessing the safety and efficacy of XC001 administered via the Extroducer® Delivery Catheter in coronary artery disease patients with refractory angina.

“The collaboration underscores the transformative potential of the Extroducer in delivering XC001 therapy for patients with refractory angina. A great example of a powerful combination of delivery system and drug therapy representing a substantial advancement in treatment options. The collaboration with such a distinguished partner as XyloCor marks a significant milestone for our global expansion efforts and will also enable us to further explore and harness the future capabilities of the Extroducer, ultimately expanding the benefits to a greater number of patients in need,” said Niklas Prager, CEO of SmartCella.

Terms of the agreement include a global license to XyloCor for use of the Extroducer for the administration of XC001 and provide for SmartCella to supply catheters to XyloCor in clinical trials and commercial use in exchange for an upfront payment, clinical, regulatory and commercial milestones and a royalty on sales. Total deal value amounts to approximately USD 130 million and mid-single digit royalties.

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one-time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

About Extroducer® Infusion Catheter System

The Extroducer® Infusion Catheter System is a first-in-class endovascular delivery device which enables direct-to-tissue drug delivery. The Extroducer® addresses a significant unmet need in the field of novel therapies, enabling targeted delivery of a wide range of modalities for solid tumor treatment, genetic disorders and tissue repair, to name but a few. Using standard equipment and routine interventional radiology approaches, the Extroducer provides access to hard-to-reach tissues by safely penetrating the vessel wall and delivering payload directly to the target location. Smartwise received U.S. Food and Drug Administration (FDA) clearance under 510(k) for the Extroducer® delivery catheter in June 2022.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical-stage biopharmaceutical company developing potential best-in-class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co-founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

About SmartCella

SmartCella, founded in 2014, is an innovative biotechnology company based in Stockholm, Sweden. SmartCella’s vision is to combine first-in-class delivery platforms with cutting-edge cell and mRNA therapies to unleash the full potential of targeted therapies. The company has three main business units, Smartwise, SmartCella Solutions and ProCella. For more information, visit www.smartcella.com.

Marea Therapeutics Launches with $190 Million to Accelerate a New Generation of Medicines for Cardiometabolic Diseases

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Leveraging large-scale human genetics to advance clinical-stage pipeline of first-in-class treatments that target critical, unaddressed and genetically validated causes of cardiometabolic diseases

Lead program, MAR001, is a first-in-class ANGPTL4 inhibitor in Phase 2 clinical development aiming to address the untreated lipid and metabolic drivers of cardiovascular events in high-risk patients

Company incubated by Third Rock Ventures with distinguished scientific founders and leading investor syndicate including Sofinnova Investments, Forbion, Perceptive Xontogeny Venture Fund, venBio, Omega Funds, Alpha Wave Global and Surveyor Capital (a Citadel company)

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Marea Therapeutics, a clinical-stage biotechnology company incubated by Third Rock Ventures to develop a new generation of medicines for cardiometabolic diseases, launched today with $190 million in combined Series A and B financings. The Series A round was led by Third Rock Ventures and the Series B round was led by Sofinnova Investments and co-led by Forbion, Perceptive Xontogeny Venture Fund and venBio, with the participation of Alpha Wave Global, Omega Funds, Surveyor Capital (a Citadel company) and founding investor Third Rock Ventures. This financing will fund the company’s MAR001 Phase 2 development plan and further progression of additional pipeline programs.

“Marea aims to transform the way cardiometabolic diseases are treated by leveraging large-scale human genetics and expertise in adipose function and biology to pursue genetically validated targets focusing on central – but unaddressed – drivers of cardiometabolic disease risk,” said Josh Lehrer, M.D., M.Phil., FACC, chief executive officer of Marea. “This approach could be the next frontier for patients with cardiometabolic disease who remain at very high risk, despite currently available therapies.”

“With initial clinical validation, world-class scientific founders and investors, and an experienced board and leadership team, Marea is poised to become a premier cardiometabolic disease company,” said Jeffrey Tong, Ph.D., board member and partner, Third Rock Ventures. “We aim to accelerate a new generation of medicines, including MAR001, that treat key unaddressed drivers of cardiometabolic disease, potentially providing important new therapeutic options for millions of patients.”

Targeting Cardiometabolic Diseases at their Source

Marea’s lead program, MAR001, is a monoclonal antibody that targets ANGPTL4, a protein that is highly expressed in adipose tissue. By inhibiting ANGPTL4 and thereby preferentially augmenting adipose tissue lipoprotein lipase (LPL) activity, MAR001 aims to lower remnant cholesterol, improve adipose tissue and metabolic function, and reduce cardiovascular events. Remnant cholesterol is carried by triglyceride-rich lipoproteins, is highly atherogenic, and drives cardiovascular events independent of classical risk factors like LDL cholesterol, diabetes or obesity1. There are currently no available targeted therapies to lower remnant cholesterol and improve metabolic function.

Human genetics has demonstrated ANGPTL4 as a highly promising therapeutic target to lower remnant cholesterol with loss of function alleles leading to remnant cholesterol clearance, improved triglyceride distribution, improved insulin sensitivity, and protection from both cardiovascular disease and type 2 diabetes all via an adipose specific mechanism.

Preclinical models with MAR001 demonstrated reduction in triglycerides, remnant cholesterol and ectopic fat (storage of triglycerides in tissues other than adipose tissue), and improved insulin sensitivity. MAR001 has demonstrated strong Phase 1 results and is in Phase 2 clinical development for adults with metabolic dysfunction.

“ANGPTL4 human genetics shows the potential to essentially reverse the adipose dysfunction responsible for the metabolic syndrome- which is not adequately treated by current therapies including weight loss and LDL cholesterol treatment. More than five million cardiovascular patients in the U.S. alone have elevated remnant cholesterol putting them at risk for a heart attack,” said Ethan J. Weiss, M.D., co-founder and chief scientific officer of Marea. “MAR001 has the potential to provide unique benefit to these patients by correcting the underlying adipose dysfunction leading to both elevated remnant cholesterol and metabolic dysfunction.”

“In a Phase 1 trial, a single dose of MAR001 significantly lowered remnant cholesterol levels and improved metabolic biomarkers. We are very excited about this compound’s potential,” said Maha Katabi, Ph.D., general partner, Sofinnova Investments. “Led by renowned experts in genetics and cardiometabolic diseases, Marea is well positioned to advance MAR001 and other pipeline programs, potentially unlocking a new era in cardiovascular care.”

Marea is also advancing a pipeline of additional candidates aimed to address additional untapped nodes driving cardiometabolic diseases.

Management and Organization

Marea is led by a dynamic team of scientists and company builders with deep know-how and experience in human genetics, adipocyte biology and cardiometabolic disease drug development.

Marea founders include:

  • Charles Homcy, M.D., partner emeritus, Third Rock Ventures
  • Sir Stephen O’Rahilly, M.D., FRS, professor of clinical biochemistry and medicine, University of Cambridge
  • Joshua Rabinowitz, M.D., Ph.D., professor of chemistry & integrative genomics, Princeton University
  • Ethan J. Weiss, M.D., chief scientific officer

Marea management team members include:

  • Christine Garrett, Ph.D., chief strategy officer
  • Mark Joing, MBA, chief development operations officer
  • Josh Lehrer, M.D., M.Phil., FACC, chief executive officer
  • Ethan J. Weiss, M.D., scientific founder and chief scientific officer

Marea board members include:

  • Ted Love, M.D., chairman, former president and chief executive officer of Global Blood Therapeutics (acquired by Pfizer) and chairman, Biotechnology Innovation Organization (BIO)
  • Antoine Boulanger, Ph.D., principal, Forbion
  • Jung Choi, MBA, entrepreneur in residence, Third Rock Ventures
  • Chris Garabedian, venture portfolio manager, Perceptive Advisors
  • Maha Katabi, Ph.D., general partner, Sofinnova Investments
  • Josh Lehrer, M.D., M.Phil., FACC, chief executive officer
  • Aaron Royston, M.D., managing partner, venBio
  • Jeffrey Tong, Ph.D., partner, Third Rock Ventures

“With its focused scientific approach and differentiated first-in-class development programs, Marea has the potential to become a leading company in the cardiometabolic disease space,” said Dr. Love. “I am thrilled to partner with this talented board and management team to advance MAR001 and other programs for patients who are in need of breakthrough cardiometabolic disease therapies.”

About Marea

Marea Therapeutics is a clinical-stage biotechnology company harnessing the latest advances in human genetics to develop first-in-class, next-generation medicines for cardiometabolic diseases. The company’s lead program, MAR001, is in Phase 2 clinical development to lower remnant cholesterol in adults with metabolic dysfunction and high risk for cardiovascular disease. Marea is led by a dynamic team of scientists and company builders with deep know-how and experience in cardiometabolic diseases, human genetics and adipocyte biology. To learn more, please visit www.mareatx.com and follow us on LinkedIn and X.

1 https://doi.org/10.1161/CIRCIMAGING.121.012615Circulation: Cardiovascular Imaging. 2021;14:e012615

Contacts

Media:

1AB

Katie Engleman

katie@1abmedia.com

Investors:

1AB

Steve Klass

steve@1abmedia.com

Cardurion Pharmaceuticals Presents Positive Clinical Results from CARDINAL‑HF Phase 2a Clinical Trial of PDE9 Inhibitor in Patients With Heart Failure

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– PDE9 inhibitor, CRD-740, demonstrated favorable safety profile and achieved statistical significance for the trial’s primary endpoint, median increase in plasma cyclic guanosine monophosphate (cGMP) –

– This clinical trial confirms that high levels of PDE9 inhibition lead to increases in cGMP, reflecting increased activation of the myocardial natriuretic peptide (NP) signaling pathway –

– Targeting PDE9 represents a novel approach to activating the NP signaling pathway, a highly validated pathway with established clinical benefits in heart failure –

– Clinical results presented today at the Annual Congress of the Heart Failure Association of the European Society of Cardiology –

BURLINGTON, Mass.–(BUSINESS WIRE)–Cardurion Pharmaceuticals, Inc. (“Cardurion”), a clinical-stage biotechnology company developing next-generation therapeutics for the treatment of cardiovascular diseases, today announced the presentation of positive clinical data from CARDINAL‑HF, the first Phase 2 proof-of-concept clinical trial of a phosphodiesterase-9 (PDE9) inhibitor in patients with heart failure. These data were presented today at the Annual Congress of the Heart Failure Association of the European Society of Cardiology taking place on May 11-14 in Lisbon, Portugal.

In the CARDINAL-HF Phase 2a trial, CRD-740 met the primary endpoint in patients with heart failure with reduced ejection fraction (HFrEF), demonstrating a statistically significant median increase in plasma cyclic guanosine monophosphate (cGMP) after four weeks of treatment. Plasma cGMP is a biomarker for intracellular cGMP whose levels reflect the activity of the protective myocardial natriuretic peptide (NP) signaling pathway, which has proven clinical benefits in heart failure. CRD-740 was generally well tolerated in the trial.

“These very promising data from the first Phase 2 proof-of-concept clinical trial of a PDE9 inhibitor in patients with heart failure represent an important next step in the development of this novel mechanism,” said James Udelson, MD, Chief of Cardiology at Tufts Medical Center and Principal Investigator for the CARDINAL-HF trial. “The results of this trial are impressive, showing robust PDE9 inhibition with significant increases in plasma and urinary cGMP, along with a favorable safety profile, both of which support moving into further clinical testing in heart failure.”

“Therapeutic targeting of the NP signaling pathway is precedented by today’s standard of care treatment for patients with heart failure, and PDE9 inhibition represents a new mechanism for activating this pathway to seek improved patient outcomes. Significant unmet needs remain, as heart failure is a prevalent and growing chronic condition that causes significant morbidity and mortality for millions of people,” said Scott D. Solomon, MD, Professor of Medicine at Harvard Medical School.

The oral presentation of these results, entitled, “A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Tolerability and Pharmacodynamic Effects of CRD-740, a PDE9 Inhibitor, in Participants with Chronic Heart Failure,” were presented today by James Udelson, M.D., Principal Investigator of CARDINAL-HF and Chief of Cardiology at Tufts Medical Center, in the “Late-Breaking Clinical Trials: Medical Therapy” session at the Annual Congress of the Heart Failure Association of the European Society of Cardiology. Key findings presented include:

  • CRD-740 was generally well-tolerated in patients with HFrEF, a disease representing approximately one-half of patients with chronic heart failure.
  • PDE9 inhibition with CRD-740 achieved statistically significant median increases in plasma cGMP at four weeks, compared to placebo, which was the primary endpoint in the trial. Statistically significant median increases in urinary cGMP were also observed in the patients who received CRD-740, compared to placebo.
  • Increases in cGMP of the magnitude seen in the trial demonstrate that CRD-740 achieves high levels of PDE9 inhibition, which prevents cGMP metabolism by the PDE9 enzyme and leads to increased activation of the NP signaling pathway. Activation of the clinically validated NP signaling pathway has been shown to benefit patients with chronic heart failure in outcome studies with sacubitril/valsartan.
  • These increases in plasma and urinary cGMP were observed in patients who received CRD‑740 with and without background treatment with sacubitril/valsartan, supporting the potential for CRD-740 as a monotherapy, and as a new approach to augment efficacy in the setting of sacubitril/valsartan therapy and to further activate the NP signaling pathway.

Based on the results of the CARDINAL-HF Phase 2a trial, Cardurion has launched two Phase 2 clinical trials in 640 patients, including a dose-ranging trial in patients with HFrEF and a proof-of-concept trial in patients with heart failure with preserved ejection fraction (HFpEF).

“These findings from Cardurion’s pioneering program in PDE9 support our conviction that PDE9 inhibition presents an important new mechanism for independently targeting and further activating the well-validated NP signaling pathway. The data from this trial suggest that PDE9 inhibition has the potential to provide benefit to patients when administered alone or in combination with guideline directed medical therapy, and ultimately become standard of care for patients with both types of heart failure,” said Peter Lawrence, Chief Executive Officer of Cardurion Pharmaceuticals.

“We are delighted to share these clinical results for CRD-740 as our team advances PDE9 inhibition as a novel approach to addressing the unmet needs of patients with chronic heart failure,” said Howard Surks, MD, Chief Medical and Scientific Officer of Cardurion Pharmaceuticals. “We thank our patients and investigators for their partnership and look forward to continuing the development of our PDE9 inhibitors to improve outcomes for patients.”

About the CARDINAL-HF clinical trial

CARDINAL-HF is the first Phase 2 proof-of-concept trial of a PDE9 inhibitor for the treatment of patients with heart failure. The Phase 2a clinical trial is a randomized, placebo-controlled trial of 60 chronic, stable patients with heart failure with reduced ejection fraction (HFrEF) who were receiving guideline-directed medical therapy (GDMT). The primary endpoints of the trial are safety and tolerability of CRD-740 and changes in plasma cGMP at four weeks, a precedented biomarker for activation of the NP signaling pathway. Other endpoints include changes in urinary cGMP and N-terminal pro b-type natriuretic peptide (NTpro-BNP) and effect of CRD-740 administration on the Kansas City Cardiomyopathy questionnaire (KCCQ), which measures symptoms, physical and social limitations, and quality of life in patients with heart failure.

The Executive Committee for the CARDINAL-HF trial includes Dr. James Udelson, Principal Investigator and Chief of Cardiology at Tufts Medical Center; Dr. Scott Solomon, Professor of Medicine at Harvard Medical School; and Dr. John McMurray, Professor of Medical Cardiology and Deputy Director of the Institute of Cardiovascular and Medical Sciences at the University of Glasgow. Drs. Udelson, Solomon and McMurray, along with Dr. Eugene Braunwald, the Distinguished Hersey Professor of Medicine at Harvard Medical School, also lead Cardurion’s Clinical Advisory Board. The CARDINAL-HF Steering Committee is further comprised of leading international heart failure clinical investigators.

About PDE9 inhibition

PDE9 inhibitors target phosphodiesterase (PDE) 9, an enzyme whose elevated activity in patients with heart failure reduces the beneficial effects of the natriuretic peptide (NP) pathway, fundamental to cardiovascular homeostasis. The beneficial effects of the NP signaling pathway in the heart muscle cell are mediated by cyclic guanosine monophosphate (cGMP) and activation of its downstream signaling molecule protein kinase G1. PDE9 is an enzyme that selectively degrades cGMP; therefore, by inhibiting PDE9, our goal is to preserve cGMP generation and enhance the beneficial natriuretic peptide signaling within heart muscle cells.

About Chronic Heart Failure

Heart failure is a prevalent and growing condition that is responsible for substantial morbidity and mortality. Approximately 6.5 million people in the United States suffer from heart failure, and 50 percent of these patients die from the condition within five years of diagnosis. One in five patients with heart failure are hospitalized annually, and 25% of these patients will be admitted again within a month of discharge. Approximately half of all patients with heart failure have reduced ejection fraction (HFrEF) and half have preserved ejection fraction (HFpEF). Despite the availability of drugs indicated to reduce morbidity and mortality in patients with both types of heart failure, substantial unmet medical need remains.

About Cardurion Pharmaceuticals

Cardurion Pharmaceuticals is a clinical-stage biotechnology company focused on the discovery and development of novel, next-generation therapeutics for the treatment of cardiovascular diseases. Cardurion was founded by physician-scientists with world-class expertise in cardiovascular signaling pathways, and a shared passion to find and develop a pipeline of novel treatment options to improve the lives of patients. Cardurion has two groundbreaking clinical programs in development, a PDE9 inhibitor targeting heart failure and the first ever CaMKII inhibitor in clinical development targeting multiple cardiovascular indications.

Cardurion Pharmaceuticals has facilities in Burlington, Massachusetts and Shonan, Japan. For more information, please visit the company’s website at https://cardurion.com.

Contacts

Kathryn Morris

The Yates Network

kathryn@theyatesnetwork.com

Cagent Vascular appoints Chairman Brian Walsh to expanded role as Chief Executive Officer

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WAYNE, Pa.–(BUSINESS WIRE)–#CLI–Cagent Vascular announced today that Brian Walsh has been named Chief Executive Officer by the Cagent Vascular Board of Directors. He has served as Chairman of the Board since March 2018 and will continue in this capacity. Mr. Walsh will succeed Carol Burns as CEO.

Cagent Vascular

Mr. Walsh previously served as Head of Carl Zeiss Meditech Cataract Technology from 2018 until 2024. Prior to that, he was President and CEO of IanTECH Medical, Inc. (acquired by Carl Zeiss Meditech AG) and Transcend Medical, Inc which was acquired by Alcon, a division of Novartis Co (NYSE: NVS) at the time. Brian Walsh brings over 25 years of medical device and health care industry experience in Marketing, Sales, and Executive Leadership which positions him as the ideal candidate to drive the market adoption of Cagent Vascular’ s flagship product, Serranator.

“Cagent Vascular is an exceptional company with a disruptive technology platform and a highly talented team,” Mr. Walsh said. “It is a privilege to lead this company through the next phase of growth. Our focus in the weeks, months, and years ahead will be on driving superior execution as we scale up and realize further market adoption.

Marc-Andre Marcotte, a Cagent Vascular board member and Partner at Sectoral Asset Management, said, “Brian Walsh’s tenure as Chairman of the Board and previous executive leadership positions give him the perfect vantage point from which to take the company forward. The board is dedicated to working with Brian and team to accelerate market adoption of Serranator and help elevate the patient care journey. On behalf of the board, I thank Carol for her significant contributions both as co-founder and as CEO over the past 10 years.”

About Cagent Vascular

Cagent Vascular, founded and led by serial medical technology entrepreneurs, is the leader in Serration Technology to treat peripheral artery disease. The company has developed a transformative improvement to conventional angioplasty, which is the most commonly performed procedure to restore blood flow. Although angioplasty has been used clinically for over 50 years, there remain significant opportunities to optimize the therapy and to improve the treatment outcomes of cardiovascular disease. To learn more about Cagent Vascular’s mission to fight PAD, visit www.cagentvascular.com.

The information contained herein obtained from Cagent Vascular management is believed to be reliable. This does not constitute the solicitation of the purchase or sale of securities. Except for historical information contained herein, matters discussed in this document are forward-looking statements, the accuracy of which is subject to risks and uncertainties.

This project is supported by the Ben Franklin Technology Partners of Southeastern PA, an initiative of the Pennsylvania Department of Community and Economic Development funded by the Ben Franklin Technology Development Authority.

Contacts

Cagent Communications and Media Contact:

Lauren Pfeiffer

+1 610 668-2006

LPfeiffer@cagentvascular.com

Protembis Announces Completion of € 30 Million Series B Financing Round and the Addition of Keith D Dawkins MD to the Board of Directors

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AACHEN, Germany–(BUSINESS WIRE)–#CerebralProtection–Protembis GmbH (Protembis), a privately-held emerging cardiovascular medical device company, announced today the completion of a € 30 million Series B financing round to support the enrollment of the PROTEMBO Investigational Device Exemption (IDE) Pivotal Trial (NCT05873816). The funding round was structured in two separate capital increases which have both been completed. It was co-led by a European consortium of VC investors including Sweden-based Segulah Medical Acceleration, Italy-based XGEN Venture, and Germany-based TechVision Fund. Other investors include Coparion, several large family offices, angel investors and a multinational medical device strategic.

“We are delighted to announce the completion of the round and would like to thank our existing investors as well as the new investors for their trust and confidence,” said Karl von Mangoldt and Conrad Rasmus Co-CEOs of Protembis. “It reflects the fact that the field of cerebral embolic protection is buoyant, and that future growth will be driven by younger and lower risk patients who have zero tolerance for brain injury risk when selecting to undergo transcatheter aortic valve replacement.”

Additionally, Protembis is proud to announce the addition of Keith D. Dawkins, MD, to the Board of Directors. Dr Dawkins has more than 35 years’ experience in the cardiovascular environment. With over 20 years as a practicing interventional cardiologist in the UK he has held research roles as a Fulbright Scholar at Stanford University, served as President of the British Cardiovascular Intervention Society, and authored more than 750 academic publications and presentations. Dr Dawkins has been the Chief Medical Officer (CMO) of Shockwave (NASDAQ:SWAV) since 2019, and this was preceded by his role as Global CMO at Boston Scientific where he held senior positions since 2008. He also serves as a member of the boards of Ventric Health LLC and JenaValve Technology Inc., as well as Chairman of InnovHeart s.r.l. Dr Dawkins will contribute his significant expertise to the clinical strategies of Protembis and the pre-commercial programs as the IDE study reaches its conclusion.

“To have such a visionary leader as Dr Dawkins join our Board of Directors is an exciting indication of the opportunities that cerebral embolic protection holds for future transcatheter therapies. We look forward to close collaboration as the field develops and our superiority trial gains momentum,” said Azin Parhizgar, PhD, Chairwoman of the Board of Directors.

“As a long-term believer in the need to protect the brain from all new lesions during transcatheter aortic valve replacement, I am very pleased to join Protembis,” said Dr Dawkins. “The ProtEmbo System and the clinical trial design are both novel and I am confident that they will be highly disruptive to the field of cerebral embolic protection, removing or mitigating many of the current issues that concern the physician community.”

About ProtEmbo® and Protembis

The ProtEmbo® Cerebral Protection System is an intra-aortic filter device that protects the entire brain from embolic material liberated during transcatheter aortic valve replacement (TAVR). It is a low-profile non thrombogenic system that shields all cerebral vessels, delivered through the left radial artery for optimal placement and stability. This is an ideal access site enabling physicians to avoid interference with TAVR equipment typically delivered through the femoral artery.

Protembis is a privately held emerging medical device company that has developed the ProtEmbo® Cerebral Protection System. The company strives to provide a simple and reliable solution to protect patients from brain injury during left-sided heart procedures, improving patient quality of life and reducing overall healthcare costs associated with brain injury during such procedures. The ProtEmbo System is currently undergoing clinical investigations.

Contacts

Protembis GmbH

Conrad Rasmus & Karl von Mangoldt

+49(0)241 9903 3622

management@protembis.com
www.protembis.com

Ocedurenone: A Novel Therapy for Uncontrolled Hypertension in Advanced Chronic Kidney Disease

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EMJ Nephrology interviewed two World-renowned experts in nephrology and cardiology, George Bakris and Faiez Zannad, regarding the challenges of treating uncontrolled hypertension (uHTN) in patients with advanced chronic kidney disease (CKD), a large unmet need for safe, effective therapies, and the potential of one therapy, Ocedurenone, to address this need.

Original article: EMJ-8.1-2023-1.pdf (emg-health.com)

Large Unmet Need

Patients with advanced CKD have a high burden of disease, compounded by serious comorbidities, most commonly hypertension. Controlling hypertension is vital in these patients to reduce the associated risks of morbidity and mortality, as well as reducing cardiorenal risk, but treatment options are limited due to safety concerns with the use of existing agents.

Current Options

Bakris:”The PATHWAY-2 trial showed that in people with resistant hypertension without advanced CKD, spironolactone produced a greater reduction in blood pressure than a β-blocker and an α-blocker. However, it presents safety risks to patients when administered.”

“Then there is eplerenone, given twice a day, while safe, has a much weaker effect on blood pressure reduction than spironolactone. That’s all we’ve got, so that’s what people use.”

Dr Bakris also mentioned two existing nonsteroidal MRAs, esaxerenone which is “approved exclusively in Japan, for which there are no placebo-controlled comparisons”, and Finerenone which “reduces blood pressure (placebo subtracted) variously from 3 mmHg (ARTS-DN, FIGARO-DKD, and FIDELIO-DKD studies) to approximately 8 mmHg (subset analysis from ARTS-DN study, eGFR of approximately 68, though office blood pressure from the same study showed around 3 mmHg reduction)”.

Future Option – Ocedurenone

Bakris:”Now, Ocedurenone, a non-steroidal MRA, shows clear promise in patients whose standard of care includes two, three, or more drugs. Ocedurenone is the only drug that has consistently steadied resistant hypertension in people with advanced CKD.”

In the first dedicated study of patients with uncontrolled hypertension and advanced CKD (BLOCK-CKD), Ocedurenone demonstrated efficacy in lowering blood pressure, together with a favorable safety profile. The experts were optimistic that it could provide a much-needed treatment option for these patients, with the added potential for reduction in the risk of cardiorenal outcomes.

“This study showed that Ocedurenone reduces SBP by 11 mmHg (placebo-subtracted), a reduction that could lead to potential approval. No other agent to date has steadied blood pressure as well in this group of patients. And the point is that these are the patients who need the most help.”

Zannad reinforced the importance of the hyperkalemia data for the clinical safety of Ocedurenone:”With Ocedurenone, we may for the first time have an MRA that is both safe and efficacious.”

Bakris added:”A Phase 3 study (CLARION-CKD) of Ocedurenone in a similar patient population is ongoing. I remain optimistic that the signal seen in the BLOCK-CKD study will be seen in this larger study.”

“I remain excited about Ocedurenone. Its tolerability and efficacy in lowering blood pressure in a very high-risk group will have a significant impact on morbidity.”

“Mineralocorticoid receptors are so ubiquitous which may allow for indications beyond CKD and cardiovascular conditions. This is likely just the beginning for Ocedurenone.”

About Ocedurenone (KBP-5074)

KBP Biosciences is a global, clinical-stage biotechnology company, headquartered in Princeton, NJ, focused on discovering, developing, and commercializing innovative small-molecule therapeutics for the treatment of serious cardiorenal and infectious diseases with large unmet medical needs.

Ocedurenone (KBP-5074) is a non-steroidal MRA discovered and developed by KBP Biosciences. At present, the Phase 3 clinical trial of the first indication for Ocedurenone, advanced CKD and uncontrolled hypertension, is underway.

About KBP Biosciences, please visit https://www.kbpbiosciences.com/.

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

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ARMGO Pharma is a privately held biopharmaceutical company dedicated to applying original, targeted science to the discovery and development of novel small-molecule therapeutics to treat debilitating cardiac, musculoskeletal, and neurological disorders. The company’s proprietary drugs, known as Rycals®, are a new class of oral agents that repair calcium leaks through the Ryanodine Receptor (RyR).

ARMGO Pharma - developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

ARM210 is a potential disease modifying therapy for genetic diseases, which targets the Ryanodine Receptor calcium channel (RyR), an intracellular calcium-release channel that becomes leaky in these and other diseases. Intracellular calcium leaks via mutant RyR1 channels impair muscle contraction leading to muscle weakness and loss of function, and activate toxic pathways that damage muscle, causing the symptoms in RYR1-RM.

ARM210 is a small molecule that binds to leaky RyR channels and repairs the leak, as demonstrated in vitro in muscle biopsies from RYR1-RM patients. The unique mechanism of action of ARM210 makes it an ideal potential disease modifying therapy for RYR1-RM. Muscle biopsies of the patients in this trial have been previously shown to respond biochemically to ARM210 in vitro. This trial will evaluate the safety and explore the biochemical effect of oral administration of ARM210 in these same patients.

ARMGO Pharma has been awarded an exclusive, worldwide license from Columbia University for its RyR technology based on the research of founding scientist Andrew R. Marks, M.D. In December 2021, ARMGO Pharma raises a $35 million Series B  funding round to progress clinical studies of lead molecule ARM210 in cardiac and skeletal muscle diseases.

ARMGO Pharma – developing small molecule Ryanodine Receptor modulators for treating cardiac, musculoskeletal and neurological disorders

More info : www.armgo.com

Keywords : ARMGO Pharma , small molecule , therapeutics , RyR technology , ARM210 , cardiovascular , musculoskeletal disorders , neurological disorders , CNS , neuroscience , Rycals , Ryanodine Receptor , RyR , Ryanodine Receptor modulators

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

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Nanostics is focused on development and commercialization of novel, non-invasive diagnostic tests for cancer. Its core technology is an advanced liquid biopsy platform that can accurately diagnose cancer from a single drop of blood. Its lead product, ClarityDx Prostate, is the most accurate diagnostic test to diagnose aggressive prostate cancer, and is positioned to emerge as the world’s leading diagnostic tool for prostate cancer.

Nanostics - micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

EVs carrying disease-specific biomarkers like nucleic acid and proteins are continuously released from cells and can be found in biological fluids including blood, urine, semen, and cerebrospinal fluid.  The levels of disease-specific EVs are closely related to disease progression making EVs promising targets for minimally invasive diagnostic assays.

Technology designed to accurately detect and measure EVs has the potential to greatly improve liquid biopsy diagnostics. At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

At Nanostics, we use micro-flow cytometry (µFCM) to quantify EV size, concentration, and marker abundance for millions of EVs in minutes. We then use advanced machine learning to analyze the vast amount of data generated using µFCM to provide rapid and precise results that continuously improve with every test.

The EV and machine learning platform ClarityDX® is set to transform the diagnostic landscape and make easy-to-use, minimally invasive predictive tests a reality in the near future.

The ability to detect and measure EVs is transforming the diagnostic landscape in immunology, neurology, cardiology, and oncology. The ClarityDX® platform technology is well-positioned for future pipeline products including tests for screening, early diagnosis, complementary diagnosis, monitoring, and management for many diseases and medically-related events. Nanostics’ R&D team continues to expand the product pipeline through in-house and partnered projects to include additional liquid biopsy diagnostic tests to improve patient care.

Nanostics – micro-flow cytometry to quantify extracellular vesicle size, concentration and marker abundance, with advanced machine learning algorithms to determine disease states

More info : http://www.nanosticsdx.com

Keywords : machine learning, exosomes, extracellular vesicles, diagnostics, oncology, cancer, prostate cancer, cardiovascular, neuroscience, biofluids , liquid biopsy , micro-flow cytometry