Innovative Therapeutic Interventions in Healthcare
Medosome Biotec Pipeline is driven by a commitment to improving patient outcomes and enhancing healthcare accessibility. Through pioneering research and strategic collaborations, we are dedicated to bringing novel solutions to the forefront of clinical practice, with a focus on efficacy, safety, and global impact.
Drug Pipeline
Pyruvate Dehydrogenase Complex Deficiency (PDCD) is a rare pediatric disease of mitochondrial energy failure. Life expectancy of affected children is severely truncated. Its prevalence in the U.S. is estimated to be in the low-mid hundreds. PDCD is the most common cause of congenital lactic acidosis (CLA) and leads typically to early death, either from unrelenting lactic acidosis and/or from progressive neurological and neuromuscular degeneration due to cellular energy failure. Treatment of PDCD remains a serious, unmet challenge. In collaboration with our strategic and academic partners we recently completed a FDA Phase III clinical trial (NCT 0216484; www.clincaltrials.gov) to evaluate dichloroacetate (DCA) for the treatment of PDCD. There has never been a controlled trial of any intervention for PDCD. We plan to submit a New Drug Application (NDA) for marketing approval to the FDA by August 2024.
Dichloroacetate (sodium salt; DCA) has therapeutic potential in treating other common life-threatening diseases of children and adults. These include pulmonary arterial hypertension (PAH), diabetes, heart disease, cancer and sepsis and represent strategic areas for future company growth for genetically-based personalized dosing of DCA.
For more information on DCA, PDCD and other indications potentially responsive to DCA please click on the button below.
Your body processes information through a network of neurons – specialized cells that transmit electrical signals. These neurons are part of a highly complex nervous system that coordinates actions and sensory information by transmitting electrical signals to and from different parts of the body.
The nervous system can be divided into two parts: the central nervous system (CNS) composed of the brain and spinal chord that processes information and the peripheral nervous system (PNS) that transmits sensory information to the CNS. Following processing, the information is transmitted back through the PNS to body parts (such as muscles) resulting in an appropriate response to the stimulus (such as muscle contractions causing movement).
The passage of electrical signals in neurons is analgous to the movement of electricity in a wire. Critical to the efficient flow of electricity in a wire is the insulating material surrounding it. Breaks in the insulating material result in a “short" - and subsequently - a loss of electrical current and signal.
Likewise, neurons are coated with an insulating material – myelin - that facilitates the efficient flow of electrical signals throughout the nervous system. Myelin is produced by specialized cells called oligodendrocytes. The cells wrap around the neuron ensheathing it in myelin. The abundance of myelin is most obvious in the neural network of the brain, where it is referred to a white matter.
There are a number of diseases that attack the myelin sheath and interfere with the flow of electricitcal signals in the nervous system. These indications include: multiple sclerosis (MS), periventricular white matter injury (periventricular leukomalacia) and neuromyelitis optica. Alzheimer’s is also associated with a loss of white matter in the brain
In general, controlling or managing demyelinating diseases such as MS has focused on preventing the destruction of the myelin. Our approach to treating demyelinating disease is different. We have identified a drug – MyeGen-0298 - that stimulates myelinating cells (oligodendrocytes) and  the formation of myelin. Furthermore, the combination of myelin protecting drugs (conventional treatment) and myelin stimulating drugs (such as our MyeGen-0298) may offer a superior treatment for the management of demyelinating diseases.
We are focusing on treating two important demyelinating diseases: periventricular leukomalacia (PL) and multiple sclerosis (MS). For more information on PL , MS and other demyelinating diseases potentially responsive to Myegen-0298 please click on the button below.
High levels of glucose in the blood causes many complications; most prevalent among these are diabetic retinopathy (DR). DR is characterized by progressive damage to the blood vessels of the retina. Despite recent advances in therapeutics and management, DR is the leading cause of blindness among adults aged 20–74 years. An estimated 4.1 million are affected by DR. DR appears to be higher in patients with type 1 than in those with Type 2 diabetes: approximately 25% of patients with Type 1 diabetes start to develop symptoms of DR within 5 years after diabetes onset, and the number increases to 80% by 15 years.
A multitude of animal studies have substantiated the beneficial effects of Ang (1-7) for the treatment of diabetes and associated complications, including DR. The biggest impediment to translate this fundamental knowledge into clinical applications, such as the management and treatment of DR, is the large-scale production of high quality Ang-(1-7) with sufficient target tissue bioavailability. Medosome Biotec and its research partners have identified a solution that offers tremendous commercial potential for treating DR as well as other vascular-related diseases: the oral delivery of Ang-(1-7) produced in the probiotic bacteria, Lactobacillus. This oral delivery and expression system represent an innovative, more efficient and cost-effective strategy to enhance the protective axis of the renin angiotensin system at both circulating and target tissues for clinical application
For more information on diabetic retinopathy, Ang-(1-7) and how the probiotic-based peptide delivery system works click on the button below.
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For questions, collaborations, or further information about our ongoing trials and therapeutic developments, please contact us. Together, let us continue to push the boundaries of medical innovation and transform the landscape of healthcare for the betterment of individuals and communities worldwide.