SAGE has built a robust product pipeline initially focused on acute and orphan CNS indications with strong preclinical to clinical translation and accelerated development timelines that are enabling us to rapidly bring important medicines to patients. In addition, SAGE is leveraging its Positive and Negative Allosteric Modulator (PANAM) platform to advance early-stage programs geared toward prevalent, chronic neuropsychiatric indications.
Status Epilepticus (SE) is an acute, life-threatening form of epilepsy or seizures that occurs in approximately 200,000 U.S. patients each year, with a mortality rate of nearly 20%. Refractory SE occurs in the approximately one-third of SE patients for which first and second line treatment options are not effective. These patients are moved to an ICU setting with little or no treatment options.
SAGE is developing SAGE-547, a proprietary positive GABAA receptor allosteric modulator, to move rapidly into Phase 1/2 clinical development for the orphan indication of refractory status epilepticus (SE) in 2013. Early data demonstrates that SAGE-547 may be more effective in treating SE than benzodiazepines due to its unique mechanism of modulating key GABAA receptor subtypes. SAGE-547 already has demonstrated robust findings in preclinical models of SE, including significantly increased efficacy over current standard-of-care benzodiazepine treatment, and compelling early clinical findings in a patient with refractory SE. In addition, SAGE is currently developing advanced next generation compounds of novel GABAA PAMs for the treatment of SE and other forms of seizure and epilepsy.
The need for safe and effective anesthesia products is growing significantly with more than 80 million general anesthesia procedures annually in the U.S. and EU and more than 50 million procedural sedations per year. There is a significant demand for innovative anesthesia products that can improve quality and safety while reducing the cost of anesthesia care. With the number of outpatient procedures increasing, a procedural sedation product with an enhanced safety profile that does not require administration by an anesthesiologist could make a dramatic impact on this market.
SAGE has advanced lead chemistry that has demonstrated similar on and off rates and dramatically improved therapeutic index in preclinical models compared to the existing standard therapy, propofol. Propofol is widely used but can pose the significant risk of respiratory and cardiac depression, is painful on injection and has been associated with bacterial contamination and shortages due to its formulation. SAGE's SGE-202 program is an IV anesthetic for procedural sedation that is designed to offer increased efficacy and safety over propofol. The program is moving toward a Phase 1 clinical trial in 2014.
Fragile X Syndrome (FXS) is a monogenic form of autism and an orphan condition causing significant behavioral impairments, such as anxiety and social phobia, and cognitive deficits. Fragile X affects 60,000 – 80,000 patients in the U.S. There are no currently approved therapies for FXS, but patients are often prescribed treatments for anxiety, ADHD and epilepsy.
SAGE is developing a proprietary Positive Allosteric Modulator (PAM) targeting GABAA that would provide symptomatic and potentially disease-modifying therapeutic benefits to patients with FXS, with a focus on ameliorating anxiety and social deficits. Data show that our compounds can uniquely modulate GABAA receptors and may correct the inhibitory deficits seen in brains of patients with FXS. SAGE is advancing its FXS program toward a Phase 1 clinical trial in 2014.
Traumatic brain injury (TBI) results from a mechanical impact to the head – like those occurring in car accidents, sports-related collisions or combat-related blast injuries – and affects more than 1.7 million people in the U.S. each year. These TBIs result in 230,000 hospitalizations, 50,000 deaths and 80,000-90,000 permanent disabilities. Currently, 5.3 million Americans live with TBI-related disabilities, compared with the more than 4 million disabled by Alzheimer's disease. TBIs are becoming more prevalent as a result of sports-related impact and are one of the most frequent causes of morbidity and mortality on the modern battlefield. There are no currently approved neuroprotective treatments for TBI.
SAGE is preparing to enter Phase 2 clinical development in 2013 with a proprietary, positive allosteric modulator.
SAGE is continuing to build its robust pipeline of proprietary positive allosteric modulators targeting GABAA receptors at a unique binding site (non-benzodiazepine site). By targeting GABAA receptors at a novel binding site, SAGE is able to mitigate the issues with prior agents targeting GABAA receptors including tolerance, dependence and sedation. SAGE is developing highly potent, selective molecules amenable to formulation into multiple routes of administration. SAGE is focused on possible future programs in epilepsy, anxiety, autism/autism spectrum disorders, pain, depression, Alzheimer's disease cognition, post-traumatic stress disorder (PTSD) and multiple sclerosis.
SAGE is continuing to build its robust pipeline of proprietary positive and negative allosteric modulators targeting NMDA receptors at a unique, previously unknown binding site (non-glycine site). By targeting NMDA receptors at a novel binding site, SAGE is able to mitigate the issues typically seen with similar approaches including limited efficacy, tolerance, and safety. SAGE is focused on possible future programs in schizophrenia (negative and cognitive symptoms), autism, depression and pain.
SAGE is committed to building a leading product focused neuroscience company and views partnering as a key component of that success. With its insights into the biology of CNS disease, extensive PANAM Platform, and experienced team of executives, SAGE is poised to rapidly develop and deliver multiple product opportunities across a wide range of CNS indications in the near term to have a significant impact on the lives of patients.
If you are interested in partnering with SAGE, please contact Jamil M. Beg at email@example.com.