Severe Oral Mucositis

Severe oral mucositis (SOM) is induced by radiation treatment and is a frequent major side effect observed in patients with head and neck cancer (HNC). SOM induces intense oral pain and limits a patient’s ability to eat and drink, which leads to severe weight loss and a requirement for enteral or parenteral nutritional support. Thirty percent of patients that develop SOM are hospitalized, and symptoms can force patients to stop cancer treatment for an undefined period of time or terminate early, thus reducing cancer treatment efficacy. Thus, SOM impacts both quality of life and clinical outcomes in HNC patients. Currently, there are no FDA approved preventive or therapeutic options for patients that develop radiation induced SOM.

The global incidence of HNC was approximately 690,000 new cases in 2012 (Globocan 2012) and recent studies showed that 50% of these patients suffered from SOM. By 2030, a significant increase in the incidence of HNC is expected with 1.03M new cases per year.

The incidence of HNC in the U.S. is estimated to be 62,000 cases in 2016 and will continue to increase to more than 93,000 new cases in 2030. A similar increase is also predicted in the EU5.

These projections include all HNC patients regardless of the anatomic location of their disease. However, the most rapidly growing sub-population of HNC are patients with oropharyngeal cancer (OPC). The oropharynx is the part of the throat at the back of the mouth, which includes the soft palate, the base (rear one third) of the tongue, and the tonsils. In the U.S., >70% of OPC is already driven by the human papilloma virus (HPV) and the incidence of HPV+ OPC is also increasing exponentially in the rest of the world. OPC is projected to be the major form of HNC by 2030 with >50% of OPC being HPV+.

Recent data has demonstrated that HPV+ OPC patients have a 6.86-fold increase in the risk of developing SOM during radiation treatment and that onset of SOM occurs sooner than HPV- OPC patients. These observations suggest that HPV+ OPC patients may be more likely to benefit from Validive (clonidine MBT) treatment than other HNC patients. The incidence of HPV+ OPC has outpaced the incidence of HPV- HNC by 4-5-fold over the past decade and this trend is projected to continue for the next 15-20 years and mirrors the increase in HPV infections in the general population.

Thus, the HPV+ OPC population will continue to drive market growth for Validive (clonidine MBT) and represents a molecularly defined population for the clinical development of this drug.

Phase II Data

Supports Further Development in OPC

Phase II Data