Pipeline

September 4, 2018

Company	Drug/Device	Medical Condition	Status
Cycle Pharmaceuticals Ltd	Ketorolac Tromethamine Tablets 10mg (as continuation treatment post IV/IM dosing of ketorolac tromethamine)	Moderately severe acute pain	Granted FDA approval of Abbreviated New Drug Application (“ANDA”)
Compass Pathways	Psilocybin, the active ingredient in “magic mushrooms”	Treatment-resistant depression	Received FDA approval for Phase IIb Clinical Trial
Akcea Therapeutics	Waylivra (volanesorsen)	Familial chylomicronemia syndrome (FCS)	Received Complete Response Letter from FDA
Poxel SA	Imeglimin	Type 2 diabetes	Patient enrollment completed in TIMES 3 trial of the Phase III registration program
Ceterix Orthopaedics, Inc.	NovoStitch® Pro Meniscal Repair System – size 0 suture cartridge	Meniscal tears	501 (k) clearance granted by the FDA
Reflection Biotechnologies Limited	RBIO-101 program (AAV.CYP4V2), an AAV-based gene therapy product	Bietti's Crystalline Dystrophy (BCD)	Granted Orphan Drug Designation by the FDA
Berlin Cures	BC 007, a DNA-based aptamer compound that binds to and eliminates functional pathogenic autoantibodies	Cardiomyopathy	Announced successful completion Phase I trial
Novaremed AG	Lead compound NRD.E1, a novel, first-in-class, small molecule	Diabetic neuropathy pain	Announced funding goal reached to begin global Phase IIb trial
Promore Pharma AB	LL-37	Venous leg ulcers	Approval to start Phase IIb clinical trial in Sweden and Poland
Pfizer	Tafamidis	Transthyretin Cardiomyopathy	Announced Phase III findings that therapy cut death risk by 30 percent in patients with distinct disease type
Ortho Dermatologics	New Drug Application for ALTRENO™ (tretinoin 0.05%) lotion	Acne vulgaris in patients 9 years of age and older	Granted approval by the FDA
Kala Pharmaceuticals, Inc.	INVELTYS™ (loteprednol etabonate ophthalmic suspension) 1%	Post-operative inflammation and pain following ocular surgery	Granted approval by the FDA
Biocodex	DIACOMIT (stiripentol)	Seizures associated with Dravet syndrome (DS) in patients 2 years of age and older taking clobazam	Granted approval by the FDA
Dompé	Oervate™ (cenegermin-bkbj ophthalmic solution)	neurotrophic keratitis (NK)	Granted approval by the FDA
Seqirus	Next-generation cell-based vaccine manufacturing process at Holly Springs, N.C.	Influenza	Granted approval by the FDA
Shire	Takhzyro (lanadelumab-flyo) injection	Hereditary angioedema (HAE) in people 12 years of age and older	Granted approval by the FDA