- Axovant Sciences (AXON): Axovant is hoping its Alzheimer's drug will be the first one in 15 years to get approved, with key data from its late-stage trial coming in September 2017.
- Eli Lilly (LLY)
- Anavex Life Sciences (AVXL)
- Baxter (BAX)
- Avanir (AVNR)
Colorectal cancer, also known as colon cancer, rectal cancer or bowel cancer, is the second leading cause of cancer death in men and women combined in the United States, according to the American Cancer Society.
Amyotrophic Lateral Sclerosis (ALS), a debilitating neurological disease.
Chronic Obstructive Pulmonary Disease (COPD)
COPD is a term used to encompass emphysema and chronic bronchitis, which are both respiratory diseases caused largely by smoking. There is no cure for COPD, only treatments that can slow the progression of the disease.
Cystic fibrosis: About 75,000 people worldwide have cystic fibrosis, an inherited disease caused by defects in a gene that controls water and salt levels in cells. The illness leads to a build-up of mucus in the respiratory system, lung damage, and life-threatening infections. Many sufferers die before they are 40.
- Orkambi from Vertex Pharmaceuticals (VRTX)
- Galapagos (NASDAQ:GLPG) the Belgium-based biotech doesn't plan to start testing of a three-drug combo until the fourth quarter of 2017.
- Novartis (NYSE:NVS) has an experimental CF drug in phase 2 -- QBW251. That study isn't expected to wrap up until 2019. The big pharmaceutical company terminated another phase 2 study of QBW251.
- MannKind (NASDAQ:MNKD) : inhaled insulin product, Afrezza
Fabry disease
Glaucoma: a condition caused by damage to the optic nerve, is the second-leading cause of blindness in the world. It is expected to affect more than 4 million Americans by 2030, up from 2.7 million today.
- Aerie Pharmaceuticals (AERI): Rhopressa
Hepatitis C
Hepatitis C, a contagious viral infection, can lead to cirrhosis (liver deterioration and scarring), liver failure, and liver cancer if left untreated. The Centers for Disease Control and Prevention (CDC) estimates that up to 3.9 million Americans have the chronic form of the disease, which is curable with medication.
Hemophilia
A rare disorder When blood can't clot properly, excessive bleeding (external and internal) occurs after any injury or damage. Fewer than 200,000 US cases per year.
- Roche (RHHBY)
- Shire (SHPG)
- Uniqure (QURE) - hemophilia B gene therapy.
High cholesterol
- Repatha from Amgen (AMGN)
- Praluent from Regeneron (REGN) and Sanofi (SNY)
Irritable bowel syndrome (IBS)
An intestinal disorder causing pain in the belly, gas, diarrhea, and constipation.
Kidney cancer
Renal cell carcinoma is the most common type of adult kidney cancer, making up about 85% of diagnoses.
Transitional cell carcinoma. This is also called urothelial carcinoma. It accounts for 10% to 15% of the kidney cancers diagnosed in adults.
Sarcoma. Sarcoma of the kidney is rare. This type of cancer develops in the soft tissue of the kidney.
Wilms tumor. Most common in children.
Lung Cancer
Lung cancer is the deadliest tumor type, killing 154,000 Americans each year.
Mesothelioma is a rare, aggressive form of cancer that develops in the lining of the lungs, abdomen, or heart.
- Merck (MRK) is largely thought to be in the lead on immuno-oncology combinations with chemo. In January, the FDA accepted an application from Merck to combine its immuno-oncology drug Keytruda with chemotherapy in advanced lung cancer.
- AstraZeneca (AZN) is testing immuno-oncology drugs durvalumab and tremelimumab alone and combined in advanced lung cancer. The company expects to have progression-free survival data in mid-2017 and final overall survival data in 2018.
- Immuno-oncology drugs teach the body to identify cancer by blocking the interaction between an immune system cell and the specific enzymes or proteins a cancer cell uses to hide. Durvalumab is what's called an anti-PD-L1 antibody. Tremelimumab is an anti-CTLA-4 antibody.
- OncoMed (OMED): Tarextumab, developed in partnership with GlaxoSmithKline (GSK)
- Cyclacel Pharmaceuticals (CYCC)
- Five Prime Therapeutics (FPRX)
- Novocure (NVCR)
- Biogen Idec (BIIB)
Nonalcoholic fatty liver disease (NAFLD)
NAFLD, a condition in which excess fat builds up in the liver, is far more common, affecting up to 40 percent of Americans, according to the National Institute of Diabetes and Digestive and Kidney Diseases. A subset of that group has a type of NAFLD called nonalcoholic steatohepatitis (NASH), where liver cells become damaged and inflamed, which can lead to scarring and cancer. Most people with NAFLD have no symptoms, though some experience fatigue and pain in the upper-right abdomen.
- No drugs are approved for NAFLD or NASH
Osteoporosis
- Radius Health (RDUS)
Pancreatic cancer: Merrimack Pharmaceuticals Inc (NASDAQ:MACK)
Parkinson's: Biotie Therapies (BITI)
Pulmonary arterial hypertension (PAH)
Schizophrenia
Schizophrenia remains among the top ten disabling conditions worldwide for young adults and affects more than 21 million people worldwide. According to Datamonitor, an independent market research firm, in 2016 approximately 3.3 million people suffered from schizophrenia in the United States, Japan and the five major European Union markets of France, Germany, Italy, Spain and the United Kingdom.
Schizophrenia remains among the top ten disabling conditions worldwide for young adults and affects more than 21 million people worldwide. According to Datamonitor, an independent market research firm, in 2016 approximately 3.3 million people suffered from schizophrenia in the United States, Japan and the five major European Union markets of France, Germany, Italy, Spain and the United Kingdom.
- Minerva Neurosciences, Inc. (NERV)
Ulcerative colitis
- Strasimod from Arena Pharmaceuticals (ARNA)
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