Acesion Pharma ApSis a Danish clinical stage biotech company founded in 2011 and based in Copenhagen. Acesion Pharma develops more efficacious and safe drugs for the treatment of atrial fibrillation (AF), the most common type of cardiac arrhythmia. Existing drug therapies generally have a limited effect or are associated with risk of serious adverse events, and there is therefore a considerable patient need for developing better and safer drugs. Inhibition of SK channels, an ion channel with relevance for regulating the heart rhythm, constitute a new and promising principle for the treatment of AF. Acesion Pharma aims to develop first-in-class SK channel inhibitors as a more efficacious and safe treatment of AF.
Adient’s mission is the development of absorbable medical devices with the premise that implants should be safe and efficacious during their useful lifetime, and then simply vanish without intervention, alleviating costly removal procedures and downstream complications.
Aeromics is a clinical stage drug development company developing Aquaporin inhibitors for the treatment of cerebral edema in stroke, based on pioneering discoveries by Dr. Peter Agre (2003 Nobel Prize in Chemistry). Cerebral edema is predictive of patient outcome in ischemic strokes, and yet there are no approved therapeutics that effectively target and treat cerebral edema as a causal driver of stroke severity. Despite the vital role of aquaporins in maintaining water homeostasis and its implications in various disease states, therapeutic agents targeting aquaporins have remained elusive until now. Aeromics’ drug candidate, AER-271, targets aquaporin-4 (AQP4), the primary water channel in the brain, and has best-in-class credentials for acute intravenous therapy for stroke-related cerebral edema. AER-271 has proven pharmacology demonstrating control of cerebral edema in models for ischemic stroke, cardiac arrest, and water toxicity.
Aggamin is an early-stage biotechnology company founded in early 2010 to develop and commercialize therapies for a variety of vascular diseases. The lead program is focused on preeclampsia — a currently untreatable disease affecting 5% of pregnancies with an annual cost to the U.S health care system of $10 billion. Lead product is an extracorporeal device for preeclampsia that reduces circulating pathogenic proteins to improve maternal and fetal health. The company objectives are to manufacture and commercialize the preeclampsia device as a safe first-in-class treatment for preeclampsia that will control maternal symptoms and extend pregnancy, thus improving both maternal and neonatal outcomes.
Allosteros Therapeutics, Inc. was a drug discovery company developing novel, potent, and selective small molecule of a critical signaling pathway that promotes heart disease. Inhibitors of a Ca2+-dependent protein kinase called CaMKII show positive preclinical proof of concept in atrial fibrillation and ventricular arrhythmia by normalizing Ca2+ homeostasis that also underlies heart failure. The company was founded in 2010 by pioneering scientists, Drs. Howard Schulman (CEO and CSO) and Mark Anderson (Head of Medicine, Johns Hopkins; Advisor). They have progressed with capital efficiency toward candidate selection and plan stepwise development of CaMKII inhibitors for several cardiovascular indications with venture capital and pharma partners.
Portfolio Name: Antag Therapeutics
Antag Therapeutics is a Copenhagen-based preclinical stage biotechnology company focused on developing therapies for dietary-related metabolic diseases. The company’s current lead therapy is a glucose-dependent insulinotropic peptide receptor (GIPR) antagonist for inducing weight loss in obese patients with Type-2 Diabetes. The Company’s technology is based upon decades of incretin physiology and diabetes research from the laboratories of Professors Mette Rosenkilde and Jens Juul Holst at the University of Copenhagen, which played a pivotal role in the establishment of dipeptidyl peptidase 4 (DPP-4) inhibitor and glucagon-like peptide-1 (GLP-1) agonist drug classes.
Apama Medical, founded in 2009 by serial entrepreneur Amr Salahieh, is a medical device company committed to improving patient outcomes by developing a novel catheter ablation system for the underpenetrated and rapidly growing atrial fibrillation (AF) market. Recognizing the need for a more effective means of treating AF.
Apama developed a unique multipoint RF balloon catheter system designed to improve procedural efficiency and versatility, addressing fundamental obstacles to the more widespread adoption of atrial ablation therapy. AF remains the most common clinically significant cardiac arrhythmia affecting an estimated 12.9 million patients globally.
Aria CV, Inc.is developing a medical device that treats pulmonary hypertension, a life-threatening disease with few therapeutic options. Pulmonary hypertension, or high blood pressure in the vessels that carry blood from the heart to the lungs, increases the workload on the heart. The increased workload leads to right heart failure, the ultimate cause of mortality in this disease. Patients are currently treated with drugs that can cost over $100,000 per year but are generally considered to have limited efficacy. Even with optimal drug therapy, the average survival time is on the order of six years, making pulmonary hypertension a tremendous unmet medical need. Aria CV, Inc. is developing a medical device that reduces the workload on the heart. Preclinical testing suggests the device has the potential to enhance both survival and quality of life of those suffering from pulmonary hypertension.
AtaCor Medical is developing novel cardiac pacing and defibrillation systems that do not require any direct connections to the patient’s heart. Today, when patients require pacemaker or defibrillator therapy, hardware is inserted into the veins and attached to the inside of the heart, a process requiring extensive x-ray imaging. Once implanted, this hardware can lead to serious complications with challenging removal procedures, even for newer “leadless” pacemakers. AtaCor’s substernal pacing and defibrillation leads are inserted without any need for medical imaging, allowing for faster, less-invasive therapy delivery. The unique placement avoids the need to access the patient’s vascular system and leaves the heart completely untouched. AtaCor’s technology will offer a new treatment option for both transient and chronic bradycardia, including the escalating need for temporary pacing in conjunction with TAVR procedures, as well as ventricular fibrillation and tachycardia.
Basking Biosciences, Inc. is developing the first reversible thrombolytic therapy for Acute Ischemic Stroke (AIS). The company was founded in 2019 by Bruce Sullenger, PhD of Duke University and Shahid Nimjee, MD, PhD of The Ohio State University. Basking is focused on exploiting the properties of RNA biology to develop aptamers with high affinity and specificity against proteins involved in coagulation and hemostasis. DTRI-031 is an RNA aptamer that binds to von Willebrand Factor (vWF) and has demonstrated potent vessel recanalization ability across multiple murine and canine preclinical models. DTRI-025 is a sequence specific reversal oligonucleotide that rapidly and durably neutralizes the pharmacologic effect of DTRI-031.
BioKier is developing a proprietary oral treatment for diabetes based on understanding the mechanism of the anti-diabetic effects of metabolic surgery and malabsorption as previously described. Independent of its weight loss effect, metabolic surgery substantially improves or even resolves diabetes within days in 80 to 90 percent of obese Type 2 diabetes patients. This surgery is equally effective in non-obese diabetes patients. BioKier has devised a proprietary method to mimic the effects of intestinal shortening surgery and potentially improve or resolve diabetes without the physical trauma and cost of bariatric surgery. Diabetes has long been recognized as a major contributor to the development of cardiovascular disease. Heart disease and stroke are the major causes of death and disability among people with Type 2 diabetes.
Capricor Therapeutics, Inc. (NASDAQ:CAPR)is a clinical stage biotechnology company focused on the development and commercialization of regenerative medicine and large molecule products for the treatment of diseases. As a developer of innovative therapies, Capricor Therapeutics is strategically positioned at the forefront of one of the largest segments of the U.S. healthcare industry – heart disease. Capricor Therapeutics’ lead product candidates will target the prevention and treatment of heart failure and heart attacks. Capricor’s allogeneic cardiosphere-derived cell (CDC) product, CAP-1002, aims to attenuate and potentially improve damage to the heart caused by a heart attack.
Capricor’s translational approach to product development is based on the comprehensive research foundation provided through our academic partnerships with leading scientists at top-tier research institutions.
Capricor and Nile Therapeutics completed a merger in 2013 to form Capricor Therapeutics, Inc. (NASDAQ: CAPR).
Cardialen, Inc. was formed in 2008 to develop and commercialize pain-free cardioversion therapy, with a primary focus on atrial fibrillation (AF). The company is developing implantable low-energy (low voltage) pain-free atrial cardioverters to help address the major unmet needs of this condition. AF is the most common cardiac arrhythmia worldwide and has been recently estimated to affect over 3.0 million people in the United States. AF, unlike ventricular fibrillation (VF), is not immediately life threatening. However, AF greatly increases the risk of stroke and premature death, impairs quality of life and generates high healthcare costs. Current therapies (drugs, external cardioversion, catheter ablation and surgery of various types) do not fully meet AF patient needs.
CardiAQ Valve Technologies (CVT) is developing the world’s first self-conforming and self-anchoring technology for transcatheter mitral valve implantation. The initial indication for this percutaneous valve will be treating functional mitral regurgitation to prevent heart failure. CVT is located in Irvine, California and is led by CEO Rob Michiels, formerly of CoreValve. Three years after our initial investment CVT had attracted over $40M in follow on funding from angels and venture capital firms.
CVT was acquired by Edwards Lifesciences in 2015.
Portfolio Name: CellAegis
CellAegis Devices (Toronto, Canada) has patented and developed the non-invasive autoRIC Device, which delivers Remote Ischemic Conditioning (RIC) therapy to patients with acute and chronic cardiovascular conditions. RIC therapy is based on the principle of adaptive physiology, where short, deliberate occlusion of blood flow to the arm activates the cellular survival network against ischemia. This medical procedure protects the heart as well as other organs from ischemia and reperfusion injury.
The autoRIC Device has CE Mark and Health Canada approval for adjunct treatment during heart attacks, cardiothoracic or surgical procedures, and is being developed for chronic conditions such as heart failure and stroke. The autoRIC Device was developed from the clinical work of Dr. Andrew Redington and colleagues, who are part of the Fondation Leducq TransAtlantic network.
CroíValve (croí is Irish for heart) is a medical device company based in Ireland developing a novel device to treat tricuspid regurgitation (TR), a severe heart condition that occurs when the tricuspid valve fails to seal properly. TR results in blood flow retrograde from the right ventricle into the right atrium, with progressive symptoms including fatigue, fluid retention leading to abdominal and leg swelling, liver and kidney failure, and death. CroíValve’s solution is a coaptation valve which restores tricuspid valve function by supporting the native leaflets while preserving cardiac anatomy. CroíValve’s device promises to be safer, more effective, less traumatic, and easier to deliver than competing technologies.
DecImmune is developing a monoclonal antibody therapeutic to mitigate injury post-myocardial infarction. DecImmune’s technology works by preventing the localized cascade of deleterious effects initiated by the innate host immune system during revascularization procedures, and is designed to maximize the long term retention of left ventricular function.
More recent studies show that our antibody also mitigates the preoteinuria associated with diabetic nephropathy. DecImmune, located in Kendall Sq, was founded by Professors Michael Carroll of Childrens’ Hospital and Francis Moore of the Brigham and Womens’ Hospital. DecImmune is supported by leading group of venture investors, experienced management and multiple Federal SBIR grants.
EP Sciences, LLC is a New York-based company founded in 2011 by the Coridea medical device incubator. The company is developing a novel waveform to reduce the severe pain associated with implantable cardioverter defibrillator (ICD) shocks in patients at risk of sudden cardiac arrest. While roughly 800,000 Americans have an implanted defibrillator, the fear of an initial shock, post-traumatic stress from having experienced a shock, and the risk of receiving inappropriate shocks greatly limit the patient and physician acceptance of ICD therapy. EP Sciences aims to improve the tolerance of ICD shocks to better assist patients suffering from ventricular fibrillation and other life-threatening cardiac arrhythmias.
Epirium is a clinical stage biopharmaceutical company that has developed unique insights related to the biology of mitochondrial biogenesis and tissue regeneration, potentially resulting in novel and clinically significant therapeutic approaches to currently intractable neuromuscular and neurodegenerative diseases, including primary and secondary cardiomyopathies. The Company has identified and established an IP-protected platform of small molecules that constitute a new class of therapeutics with the potential to stimulate mitochondrial biogenesis and tissue regeneration. Epirium intends to advance its first clinical candidate (EB 002) this year, initially in Becker muscular dystrophy, followed by drug development targeting other progressive neuromuscular and neurodegenerative disorders associated with mitochondrial depletion.
FineHeartis a French medical device company focused on creating innovative technologies in the cardiovascular space.
FineHeart has developed the ICOMS (Implantable Cardiac Output Management System), a novel, wirelessly powered, fully implantable mechanical circulatory support device that is able to optimize cardiac output while preserving the heart’s innate contractility. It is a game changing therapy for long term circulatory assistance in severe heart failure patients. FineHeart was founded by a team of four internationally renowned cardiologists, led by Dr. Stéphane Garrigue (ICOMS Inventor) and Dr. Philippe Ritter, co-inventor of cardiac resynchronization therapy, with a vision of making a miniaturized, fully implantable, pulsatile circulatory support system that would overcome many of the challenges associated with left ventricular assist devices (LVADs) currently on market. The overarching goal is to restore normal cardiac output and allow heart failure patients to return to a normal quality of life. The founding scientists and clinicians have been joined by four managers with specific expertise in the medical device industry, led by Arnaud Mascarell (CEO).
GI Windows is a clinical-stage medical device company developing a non-surgical approach to create anastomoses in the GI tract. Their patented self-assembling magnets are designed to enable an incision-less and durable intestinal bypass to treat chronic diseases, such as Type-2 Diabetes and Obesity, without the cost and invasiveness of surgical interventions.
Gila Therapeuticsis a clinical-stage biotherapeutic company focused on pharmacological treatment of obesity employing intra-oral application of naturally occurring satiety hormones to induce early fullness, leading to weight loss. Targeting a unique and recently discovered direct neural pathway between the tongue and brain, Gila is developing formulations that when applied to the tongue prior to meals directly stimulates the brain’s satiety centers intended to reduce food intake without any systemic exposure.
Gila, based in Minnesota, was founded in 2014 by Andres Acosta, Sergei Zolotukhin and Thomas Vasicek who serves as the CSO. Scott Schorer serves as the CEO.
HAYA Therapeutics is dedicated to treating heart failure through the discovery and development of innovative first-in-class RNA-based therapeutics, with a specific focus on long noncoding RNAs.
Herantis Pharma Plc is an innovative drug development company focused on regenerative medicine and unmet clinical needs. Their first-in-class assets are based on globally leading scientific research in their fields: CDNF for disease modification in neurodegenerative diseases, primarily Parkinson’s and ALS; and Lymfactin® for breast cancer associated lymphedema, with potential also in primary lymphedema. The shares of Herantis are listed on the First North Finland marketplace run by Nasdaq Helsinki stock exchange.
Infobionic™ is an emerging mHealth company with a proprietary wireless remote patient monitoring platform, MoMe™, for superior arrhythmia monitoring and diagnosis. An arrhythmia is an irregular heart beat (rate or rhythm) and 4M Americans suffer from recurrent arrhythmia. Atrial Fibrillation (AF) is the most common form with 200,000 new cases each year. The initial market application will be with physicians who specialize in Electrophysiology.
The Massachusetts General Hospital (MGH), the Technical University of Munich (TUM), and Broadview Ventures, Inc. are collaborating on NIRF-IVUS Molecular Imaging of Atherosclerosis. The improved detection and stratification of high-risk plaques, i.e. atherosclerotic plaques at risk of causing heart attacks and strokes, is an unmet need. Early detection enables earlier intervention, prevention and tailored pharmacotherapy. Farouc Jaffer, MD, PhD, of MGH and Vasilis Ntziachristos, PhD of TUM, in conjunction with Intravascular Imaging Incorporated (“I-Cubed”) , are developing and testing prototype devices, software and systems for a comprehensive catheter-based approach to detect high-risk plaques in human arteries.
Ischemia Care is a commercial-stage diagnostic company developing a platform of RNA expression blood tests to determine the cause of ischemic stroke and TIA. ISC’s first test, ISCDX, launched upon the completion of the BASE clinical trial (NCT02014896) which enrolled over 1,700 subjects at 20 major hospitals in the US. ISCDX seeks to characterize the underlying cause of the roughly 35% of incident strokes known as cryptogenic, enabling physicians to better care for their patients and more effectively prevent recurrent strokes. ISCDX was launched commercially in 2019.
Kantum Pharma is a privately-held biopharmaceutical company focused on developing therapies to prevent or reduce inflammation initiated through the uridine diphosphate (UDP)-glucose/P2Y14 purinergic receptor signaling pathway in organs such as the kidneys, lungs and female reproductive tract. The Company’s initial small molecule therapeutic program, KB-1801, has been shown preclinically to reduce renal inflammation and has the potential to transform the treatment paradigm for acute kidney injury, which occurs in 30% of cardiac surgeries and kills over 300,000 people each year in the US. Kantum is pursuing additional applications of this technology for therapeutics and diagnostics to ameliorate the impact of numerous inflammatory disorders.
Lyra Therapeutics (NASDAQ:LYRA) has developed the XTreo™ platform based on expertise in materials science, drug development and formulation. This proprietary technology platform is designed to enable sustained delivery of medications for many months of therapy, targeting tissues deep in the ENT passages and potentially other diseased tissues that are not accessible with conventional therapeutic approaches.
In addition to ENT indications, this platform technology has been tested as a pediatric resorbable, self-expanding scaffold to address a significant unmet clinical need for children with cardiovascular defects. The scaffold technology is capable of providing a combination of strength and flexibility to support lumen patency and apposition during vessel growth. While resorption facilitates re-intervention, if necessary, with no permanent implant left behind.
Lyra Therapeutics went public via IPO in May, 2020.
Mellitus is dedicated to advancing diabetes detection and monitoring through products based on GCD59, a biomarker of glycemic control that is unique because of its direct relationship to the complications of diabetes. Our premier application is a patient-friendly test for gestational diabetes, enabling more timely intervention to improve the health of both mother and child. We plan to commercialize our products through strategic partners as we expand indications to benefit all individuals with diabetes.
miRagen Therapeutics, Inc., is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative microRNA (miRNA)-targeting therapies in disease areas of high unmet medical need. miRagen’s lead product candidate, MRG-106, a synthetic microRNA antagonist (LNA antimiR) of microRNA-155, is currently being studied in a Phase 1 clinical trial in patients suffering from cutaneous T-cell lymphoma (CTCL) of the mycosis fungoides (MF) sub-type. miRagen is also conducting a Phase 1 clinical trial of MRG-201, its lead anti-fibrosis product candidate and a synthetic microRNA mimic (promiR) to microRNA-29b, in human volunteers. miRagen seeks to leverage in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance promising technologies and high-potential product candidates for its own pipeline and in conjunction with strategic collaborators.
NIDO is a medical device company developing SEPIA, an innovative device to improve epicardial ablation for ventricular tachycardia (VT), based on technology developed at Boston Children’s Hospital. SEPIA will enable real-time visualization of the epicardial surface and provide angle and force control to ablation catheters, resulting in improved ablation efficiency, decreased VT recurrence and fewer complications.
The company was co-founded by Dr. Pedro del Nido, Chairman of Cardiovascular Surgery and Professor at Harvard Medical School, and Dr. Nikolay Vasilyev, former Assistant Professor of Surgery at Harvard Medical School, currently Medical Director for the Surgicals Portfolio at Pfizer.
NuPulse, Inc is a clinical-stage medical device company that is developing novel technology to treat patients with heart failure, one of the leading causes of death worldwide. The device, which requires minimally invasive surgery, provides patients an alternative to medical therapy. NuPulse’s founders have extensive clinical, engineering and life science experience, which will allow them to alter the paradigm for the treatment of heart failure.
Provasculon is developing novel therapeutic proteins that help organs damaged by ischemia to heal. Ischemia is the primary cause of injury and loss of function following a heart attack, in chronic heart failure, and in peripheral vascular disease. Provasculon’s technology works by recruiting and activating stem cells that regenerate blood vessels and promote repair of injured tissue. In animal models, Provasculon’s lead protein therapeutic can restore hearts that have been damaged by severe ischemia to near normal function.
Provasculon was founded by pre-eminent leaders in cardiovascular regenerative medicine from Harvard University. The company has assembled an experienced team of successful scientists and entrepreneurs to develop regenerative protein therapeutics through proof of principal Phase 2 clinical studies, and is supported by a syndicate of well-respected venture capital firms
Provasculon has been acquired by Mesoblast, an Australian based company developing allogeneic or ‘off-the-shelf’ regenerative medicine products focus on repair of damaged tissues and modulation of inflammatory responses in conditions with significant unmet medical needs, in 2013.
Portfolio Name: Pulmokine
Pulmokine is a biopharmaceutical company developing a novel class of kinase inhibitors for pulmonary related diseases. Its first program, currently in clinical development, is an inhaled small molecule inhibitor of PDGFR for the treatment of pulmonary arterial hypertension (PAH).
Gossamer Bio (NASDAQ:GOSS) has licensed the PAH program (GB002) and will manage and fund the development through regulatory approval.
Puzzle Medical Devices Inc. is developing ModulHeart, a percutaneous heart pump for patients with advanced HF. The device’s modular design allows for hemodynamic support through multiple parallel endovascular pumps anchored in the descending aorta. This novel design results in low pump speeds, secure aortic anchoring, and positioning distal to the cerebral arteries. As a result, the device is expected to preserve blood integrity, reduce the risk of embolic events, enable ambulatory and outpatient use. These attributes will allow for safe long-term implantation, with minimal risk of bleeding, stroke, and pump thrombosis.
RapidPulse is developing a novel cyclic aspiration system enabling rapid and consistent mechanical thrombectomy of blood clots for the treatment of acute ischemic stroke.
Remedy Pharmaceuticals, Inc. is a privately-held, late clinical stage pharmaceutical company focused on developing and bringing lifesaving treatment to people affected by acute central nervous system edema. Remedy is developing CIRARA for the improvement of functional outcomes by reducing the formation of cerebral edema in patients following Large Hemispheric Infarction (massive ischemic stroke affecting the total or sub-total territory of the middle cerebral artery). There are currently no therapeutic approaches targeted at reducing the development of edema early after Large Hemispheric Infarction, and CIRARA is now in Phase 3 trials with the aim of meaningfully reducing the high mortality and morbidity of this disease.
Remedy Pharmaceutical’s CIRARA program was acquired by Biogen in 2017.
Renovacor is a preclinical stage biotechnology company whose mission is to develop improved therapies for genetically derived cardiovascular diseases. The company is currently developing a gene therapy for a rare, familial form of dilated cardiomyopathy. Renovacor’s lead gene therapy product aims to restore cardiac function in patients with symptomatic heart failure due to BAG3 gene mutation.
Vectorious Medical Technologies, a Tel-Aviv based company, has developed the V-LAP™, a miniature wireless pressure sensor implanted into the heart’s left atrium. The V-LAP™ enables patients to take a direct daily measurement of the heart’s left atrial pressure (LAP), the earliest and most accurate indicator of heart function, and therefore to optimize treatment at an early enough stage to prevent decompensation. As a result, patients will enjoy an enhanced quality of life, physicians will be able to provide superior care and hospitals will see fewer readmissions, saving the healthcare system billions per annum.
VentriNova, Inc. is developing gene therapy for the regeneration of heart tissue in patients with heart failure and after myocardial infarction. Chief Scientific Officer and Founder Hina Chaudhry, MD leads VentriNova, which was incorporated in 2006. VentriNova has funded a $1 million Sponsored research Agreement with Mt. Sinai School of Medicine.
Vascular Graft Solutions (VGS) is a leading developer of innovative technologies to improve surgical revascularization, vascular reconstruction procedures, and arteriovenous fistula. VGS is improving the standard-of-care in cardiovascular surgery while focusing on patients’ quality-of-life, surgeons, and health economics.
XII Medical is developing an innovative, minimally invasive technology to address obstructive sleep apnea (OSA), a condition affecting approximately 45 million people in the US. OSA is characterized by recurrent, functional collapse of the upper airway during sleep, causing substantially reduced or complete cessation of airflow and leading to nocturnal hypoxemia. Untreated OSA is associated with long term negative health outcomes, including an increased risk of cardiovascular disease, poor neurocognitive performance, reduced quality of life, and increased all-cause mortality. Treating OSA has specifically been shown to improve disease markers across a range of cardiovascular conditions, yet many patients are unable to tolerate current therapeutic options, with adherence rates of roughly 50%.
Zumbro Discovery is a biotechnology company founded by cardiologists, John Burnett MD and Horng Chen MD, that head up the Cardiorenal Research Laboratory at the Mayo Clinic. ZD100 is Zumbro’s lead drug candidate for the treatment of resistant hypertension (RH) which afflicts approximately 9 MM Americans and for which there is no FDA approved drug therapy. ZD100, which is a modified and patented version of the peptide released by the human heart to regulate blood pressure, has been shown to be safe and has demonstrated efficacy in treating RH patients in very early stage clinical trials.
Portfolio Name: ZZ Biotech
ZZ Biotech LLC is a clinical stage company developing innovative biologic treatments for ischemic stroke. ZZ Biotech was formed in 2006 to focus on the development of 3K3A-APC, a novel second-generation variant of a naturally occurring human protein, activated Protein C (APC). The major unwanted side effect of APC is bleeding, which limits its pharmacologic dosing in man. 3K3A-APC has markedly reduced anticoagulant activity, but preserved cell-protective and anti-inflammatory activities compared to wild-type APC. In animal models of stroke 3K3A-APC has shown an advantage over wild-type APC in enhanced efficacy and reduced risk for bleeding. Additionally, the combination of 3K3A-APC and tissue Plasminogen Activator (tPA) provides benefits well beyond those found with either agent alone.
ZZ Biotech has completed a multicenter Phase 2 clinical trial (RHAPSODY) of 3K3A-APC in moderate to severe acute ischemic stroke patients treated with intravenous tPA, intra-arterial thrombectomy, or both. The study was supported by the National Institutes of Health through a pair of NeuroNEXT grants. RHAPSODY established the maximally-tolerated dose (MTD) of 3K3A-APC in ischemic stroke patients and demonstrated substantial reduction in both total hemorrhage volume and hemorrhage incidence among patients treated with 3K3A-3PC vs. those treated with placebo.
