With all the great competition in the biotech sector when it comes to cancer therapeutics, companies who are focusing on other diseases are sometimes not being discussed, like SAGE Therapeutics, Inc. (NASDAQ: SAGE) a clinical-stage biopharmaceutical company. SAGE is engaged in developing and commercializing novel medicines to treat life-threatening, central nervous system (CNS) disorders. The company anticipates releasing phase 2a results by end of June.
CNS disorders represent 35% of the worldwide disease burden. CNS disorders affect the ability of the brain and spinal cord to function properly. Millions of Americans suffer from these disorders, which include epilepticus (persistent seizure), orphan genetic epilepsies such as Rett syndrome, Dravet syndrome, and essential tremor.
There are drugs available to treat some CNS disorders, but often they don’t meet urgent medical needs patients have, and often cause severe side effects as well. SAGE claims that their novel platform, SAGE drug discovery platform, makes it possible for them to enable pharmacologic customization. SAGE explains that they have developed a technology through which they can both design and optimize molecules and control their important properties; for example, half-life (how long the drug is effective), or brain penetration (the ability to get into the central nervous system). Using the SAGE drug discovery platform could lead to new drug candidates that bind with targets in the brain with more precision, increased safety and tolerability, as well as with fewer side effects than current CNS therapies.
The company’s main product candidate, SAGE-547, is currently undergoing two exploratory phase 2 clinical trials for treatments of 2 CNS disorders - Postpartum Depression and Essential Tremor.
One of these trials has already shown positive results. On June 8th, SAGE published data from an exploratory phase 2a trial of SAGE-547 to establish proof of principle in treatment of severe postpartum depression. Since this is an exploratory trial, the drug was tested on a very small group of people, only four in fact. But the results are impressive. All four patients suffering from postpartum depression responded to the therapy, and the evaluation was recorded based on the Hamilton Rating Scale for Depression. The patient's average rating on the scale was 26.5 before treatment was initiated and it has dropped to 1.8 after a 60-hour treatment period. This is a symptom free level. According to SAGE, 20% of all women suffer postpartum depression and the four women who participated in trial have not responded any antidepressant therapy beforehand.
The stock of SAGE surged more than 10% after the data was published.
The data exploratory phase 2 clinical trials for treatment of Essential Tremor remains to be published by the end of this month. Essential Tremor is one of the most common neurological diseases and is the most common movement disorder. Its cause is unknown. Typically the disorder causes a tremor of the arms, hands or fingers and occasionally could involve the head, vocal cords or other body parts during movements and essential activities like eating or writing. Approximately 4% of people at the age of 40 suffer from Essential Tremor, and even higher percentages among people in their 60s, 70s, 80s, and 90s. Whether or not SAGE-547 will as effective on Essential Tremor as it is on postpartum depression remains to be seen.
SAGE Therapeutics is in an excellent financial position, with no debt and about $113 million of cash. The company burned approximately $14.5 million on operating activities last quarter, indicating enough resources to continue its operations until mid 2016 without raising extra debt.
We will continue our coverage SAGE Therapeutics after more data is published.