Year: 2019-20

Company: Ascendis Pharma

Liaison(s): Mary Metz, Katherine Wang

Cancer is the second leading cause of death globally, representing a widespread unmet need for effective treatments. Additionally, many rare diseases such as growth hormone deficiency, hypoparathyroidism, and achondroplasia lack sufficient treatment alternatives. As the burden of these diseases continues to grow globally, more pioneering treatment options are increasingly necessary. Ascendis Pharma (Ascendis) understands the urgency behind this global call to action for new therapeutic treatments for cancer and rare diseases in order to address the unmet needs of these vulnerable patient populations worldwide.

Ascendis Pharma is quickly becoming a leader in new cutting-edge therapies using its innovative TransCon technology platform. Ascendis Pharma’s TransCon platform enables the sustained release of a drug in a controlled and predictable manner, thereby prolonging a drug’s therapeutic effect in the body through an increased half-life. TransCon may also provide safer delivery of highly potent drugs that otherwise have significant systemic toxicity. Using this platform, Ascendis strives to make a meaningful impact in patients’ lives.

Ascendis Pharma is currently developing product candidates in two therapeutic areas: endocrinology rare diseases and oncology. All three endocrinology rare disease candidates are in clinical trials and show great promise, and the exciting oncology candidates are still in preclinical development. KGI was selected to assist Ascendis Pharma as it continues to develop new compounds and target new disease areas. The Ascendis Pharma Team Master’s Project has worked to identify and prioritize the top five indications to pursue in oncology for a new TransCon product and assess top indications in a new disease area for one of Ascendis Pharma’s existing TransCon therapeutics.