Pipeline

March 19, 2018
Company Drug/Device Medical Condition Status
XW Laboratories Inc. XW10172 Narcolepsy Phase I trials initiated
Cadent Therapeutics CAD-1883 Neurological (Essential Tremor) and psychiatric disease (Spinocerebellar ataxia) Phase I trials initiated
Arrowhead Pharmaceuticals, Inc. ARO-AAT Alpha-1 liver disease Phase I trials initiated
Voyager Therapeutics VY-AADC Advanced Parkinson’s disease Phase Ib trials initiated
Protalix Biotherapeutics, Inc. OPRX-106 Ulcerative colitis Phase II trials initiated enrolling 24 subjects
Merrimack Pharmaceuticals, Inc. MM-121 Lung cancer Phase II trials initiated expanding from 80 to 100 subjects
Biogen SPINRAZA (nusinersen) Spinal muscular atrophy (SMA) Phase II trials initiated enrolling 25 pre-symptomatic infants six weeks old or younger
MyoKardia Mavacamten Hypertrophic cardiomyopathy (HCM) Phase II trials initiated
AM-Pharma recombinant human Alkaline Phosphatase (recAP) Acute Kidney Injury (AKI) Phase II trials initiated enrolling 301 subjects with sepsis
Eleven Biotherapeutics, Inc. Vicinium Non-muscle invasive bladder cancer (NMIBC) previously treated with bacillus Calmette-Guérin (BCG) Phase III trials initiated
AbbVie Elagolix Uterine fibroids Phase III trials initiated
Merck KEYTRUDA (pembrolizumab) Advanced cervical cancer with disease progression on or after chemotherapy Priority Review granted by the FDA
Cerapedics, Inc. P-15L Transforaminal lumbar interbody fusion (TLIF) surgery for degenerative disk disease IDE Approval granted by the FDA
Proteostasis Therapeutics PTI-428 Cystic fibrosis Breakthrough Designation granted by the FDA

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