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.
Aeromics is a clinical stage drug development company developing the world’s first drug targeting a molecular water channel for the treatment of cerebral edema (brain swelling) in stroke. Each year almost 900,000 Americans and an additional 900,000 Europeans suffer a new or recurrent stroke, 85% of which are ischemic. Stroke is the third leading cause of death in the USA and the leading cause of disability. The annual medical costs are estimated at ~$70B in the U.S. and are expected to double by 2025. 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.
A paradigm shift in our understanding of water physiology was the discovery aquaporins, proteins that form water channels to allow water into and out of cells, by Peter Agre (2003 Nobel Prize in Chemistry). 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.
Allosteros Therapeutics, Inc. is a drug discovery company has developed 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.
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 previously described and malabsorptive. 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).
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.
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.
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 CEO.
Herantis Pharma Plc is an innovative drug development company focused on regenerative medicine and unmet clinical needs. Our 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.
Kantum Pharma is a privately-held biopharmaceutical company focused on developing therapies to prevent or reduce inflammaton 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 AKI. Kantum is pursuing additional applications of this technology for therapeutics and diagnostics to ameliorate the impact of numerous inflammatory disorders.
Acute kidney injury (AKI) is characterized by an abrupt loss of kidney function—this common medical complication affects 4,000,000 and kills 300,000 people every year in the US alone. AKI affects one in five hospitalized adults and one in three hospitalized children, and is associated with annual healthcare costs in the USA exceeding $10B. AKI occurs in roughly 30% of all cardiac surgeries, and Kantum Pharma is uniquely positioned to develop a novel specific AKI therapeutic to reduce its incidence, severity and sequelae. In parallel, Kantum will develop a test to enable doctors to diagnose AKI before it develops. This combination of early diagnosis and specific therapy to reduce or prevent AKI incidence and progression promises to transform clinical outcomes for patients undergoing cardiac surgery.
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.
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.
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.
Provasculon was acquired by Mesoblast 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.
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.
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.
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.