Profile & Drug Development Brain Cancer Glioblastoma Multiforme (GBM)

ISR Teports PROFILE & DRUG DEVELOPMENT BRAIN CANCER GLIOBLASTOMA MULTIFORME (GBM) www.ISRreports.com WHAT IS GBM? GBM is the most common and most aggressive form of primary malignant brain tumor. 240,000 PEOPLE are diagnosed with GBM globally each year. That's equivalent to the population of Durham, NC. that's 2-3 CASES SYMPTOMS per 100,000 • Seizures • Nausea and vomiting • Headaches Hemiparesis Memory, personality, or neurological deficits adults GBM is slightly more common in MALES and slightly more common in CAUCASIANS (in US). TREATMENT No available treatment for GBM is curative and options are extremely limited. The most optimal treatment for GBM is a combination of surgery, radiotherapy, and chemotherapy. SURGERY GOAL: removes as much tumor tissue as possible. It is believed that surgery gives patients a better prognosis when they begin other treatment. RADIATION 1. Radiation aims to kill as many of the cells left behind from surgery as possible . 2. Radiation is usually given 5 times a week for 6 weeks. 3. Radiation is combined with the oral chemotherapy, Temozolomide. Temozolomide increases survival time from 12.1 MONTHS to 14.6 MONTHS. CHEMOTHERAPY GOAL: Chemotherapy and radiation aim to put the remaining cancer cells into a nondividing, sleeping state. 25% of patients benefit from adjuvant chemotherapy. Rapid tumor growth, highly resistant tumors, and a lack of effective therapies contribute to making this one of THE DEADLIEST CANCER TYPES. SURVIVAL YEAR Breast Cancer: 89% Colorectal: 64% Glioblastoma: 4.8% Lung: 15% Prostate: 99% Without treatment, the mean life expectancy is approximately 17 4.5 MONTHS. With standard of care treatment the average life expectancy rises to approximately 15 MONTHS. THE FUTURE GBM is classified as an ORPHAN DISEASE This is defined as a disease 24 that affects fewer than 200,000 people (in the U.S.). Potential therapies can be eligible for ORPHAN DRUG DESIGNATION, which allows the developing company to receive tax breaks and market exclusivity upon approval of the therapy. 50 POTENTIAL NEW THERAPIES in active development, positive results in early stage trials. 50 These new therapies include: • Immunotherapeutic Vaccines • Anti-Angiogenesis • Gene Therapy • Cell Signaling Pathway Inhibitors • Cytotoxics 76 GBM 76 CLINICAL TRIALS were started in 2013 58 58 PHARMA & BIOTECH COMPANIES have sponsored GBM trials since 2010. Sources: ISR Reports, brain.mgh.harvard.edu, cancer.org, http://visual.ly/brain-cancer-info- graphic, www.mdanderson.org, seer.can- cer.gov, International Agency for Research on Cancer Scientific Publications Number 155, 2002, www.cbtrus.org, http://globocan.iarc.fr, neurosurgery.ucsd.edu/glioblastoma/, emedi- cine.medspace.com, ISR reports [email protected] @ISRREPORTS ISRREPORTS.COM RATE