2016 Medicines in Development for and Related Conditions

Diabetes Drug Name Sponsor Indication Development Phase

AC-201 TWi Biotechnology Phase II completed (caspase 1 inhibitor/ Taipei, Taiwan www.twibiotech.com IL-1 beta inhibitor) (SK-0403) Kowa Research Institute type 2 diabetes Phase II (CD26 antigen antagonist) Morrisville, NC www.kowaus.com bexagliflozin Theracos type 2 diabetes Phase III (SGLT2 inhibitor) Marlborough, MA www.theracos.com

BFKB8488A (RG7992) Genentech type 2 diabetes Phase I (anti-FGFR1/KLB mAb) South San Francisco, CA www.gene.com

BGP-15 N-Gene Research Laboratories type 2 diabetes Phase II (JNK inhibitor/ sensitizer) New York, NY www.n-gene.net

BIOD-123 Biodel type 1 diabetes Phase II (RHI-based ultra-rapid-acting insulin) Danbury, CT www.biodel.com

BIOD-531 Biodel type 2 diabetes Phase II completed (concentrated ultra-rapid-acting Danbury, CT www.biodel.com insulin)

BKR-013 BioKier type 2 diabetes Phase I (-like 1 stimulant) Chapel Hill, NC www.biokier.com

Medicines in Development: Diabetes 1 Diabetes Drug Name Sponsor Indication Development Phase

BMS-986036 Bristol-Myers Squibb type 2 diabetes Phase II (FGF-21 agonist) Princeton, NJ www.bms.com

BTI-320 Boston Therapeutics type 2 diabetes Phase II (alpha glucosidase inhibitor-chewable) Newton, MA www.bostonti.com

Bydureon® AstraZeneca type 2 diabetes Phase III extended release Wilmington, DE (weekly suspension) www.astrazeneca.com

type 2 diabetes (pediatric) Phase III www.astrazeneca.com

Byetta® AstraZeneca type 2 diabetes (pediatric) Phase III exenatide Wilmington, DE www.astrazeneca.com

CLBS03 Caladrius Biosciences type 1 diabetes Phase II (Treg cell-based therapy) Basking Ridge, NJ (Fast Track) www.caladrius.com ORPHAN DRUG

Cycloset® VeroScience type 2 diabetes (pediatric) Phase I Tiverton, RI www.veroscience.com

Dance-501 Dance Biopharma type 1 diabetes, type 2 diabetes Phase II (inhaled recombinant human Brisbane, CA www.dancebiopharma.com insulin)

Medicines in Development: Diabetes 2 Diabetes Drug Name Sponsor Indication Development Phase

Diamyd® Diamyd Medical newly-diagnosed type 1 diabetes Phase I autoimmune diabetes vaccine Stockholm, Sweden www.diamyd.com ORPHAN DRUG University of Alabama at Birmingham Birmingham, AL

DS-8500 Daiichi Sankyo type 2 diabetes Phase II (GPR119 agonist) Parsippany, NJ www.daiichisankyo.com

DV-100 DiaVacs type 1 diabetes Phase I (autologous dendritic cell Edgewater, NJ www.diavacs.us.com immunotherapy) ORPHAN DRUG efpeglenatide Sanofi US type 2 diabetes Phase II (long-acting GLP-1 Bridgewater, NJ www.sanofi.com receptor agonist) /linagliptin/ Boehringer Ingelheim Pharmaceuticals diabetes Phase I extended release Ridgefield, CT www.boehringer-ingelheim.com Eli Lilly www.lilly.com Indianapolis, IN empagliflozin/metformin Boehringer Ingelheim Pharmaceuticals type 2 diabetes application submitted extended release Ridgefield, CT www.boehringer-ingelheim.com Eli Lilly www.lilly.com Indianapolis, IN

Medicines in Development: Diabetes 3 Diabetes Drug Name Sponsor Indication Development Phase Merck type 2 diabetes Phase III (SGLT2 inhibitor) Kenilworth, NJ www.merck.com Pfizer www.pfizer.com New York, NY

Farixga® AstraZeneca type 1 diabetes Phase III Wilmington, DE www.astrazeneca.com

type 2 diabetes (pediatric) Phase III www.astrazeneca.com

GIP/GLP-1 co-agonist peptide Eli Lilly diabetes Phase I Indianapolis, IN www.lilly.com

GLWL-01 GLWL Research type 2 diabetes Phase I Montreal, Canada

HDV insulin Diasome Pharmaceuticals type 1 diabetes, type 2 diabetes Phase II (insulin tregopil) Cleveland, OH www.diasomepharmaceuticals.com

HIP2B CureDM type 2 diabetes Phase I completed (human proislet peptide) Wilmington, DE www.curedm.com

HMS5552 Hua Medicine type 2 diabetes Phase I completed (GKA activator) Shanghai, China www.huamedicine.com

Medicines in Development: Diabetes 4 Diabetes Drug Name Sponsor Indication Development Phase imeglimin Poxel type 2 diabetes Phase II completed (glimins) Lyon, France www.poxel.com

IN-105 Biocon type 1 diabetes, type 2 diabetes Phase I completed (insulin tregopil) Bangalore, India www.biocon.com biosimilar Biocon type 1 diabetes, type 2 diabetes Phase III Bangalore, India www.biocon.com Mylan www.mylan.com Canonsburg, PA insulin glargine biosimilar Gan&Lee Pharmaceuticals type 1 diabetes Phase I Beijing, China www.ganlee.com insulin/pramlintide AstraZeneca type 1 diabetes Phase I completed combination Wilmington, DE www.astrazeneca.com Juvenile Diabetes Research Foundation New York, NY

Invokana® Janssen Research & Development type 1 diabetes Phase II Raritan, NJ www.janssenrnd.com

type 2 diabetes (pediatric) Phase I www.janssenrnd.com

Medicines in Development: Diabetes 5 Diabetes Drug Name Sponsor Indication Development Phase

IONIS-GCCRRX Ionis Pharmaceuticals type 2 diabetes Phase II (glucocorticoid ) Carlsbad, CA www.ionispharma.com

IONIS-GCGRRX Ionis Pharmaceuticals type 2 diabetes Phase II ( antagonist) Carlsbad, CA www.ionispharma.com

IONIS-PTP1BRX Ionis Pharmaceuticals type 2 diabetes Phase II (PTP-1B inhibitor) Carlsbad, CA www.ionispharma.com

ITCA 650 Intarcia Therapeutics type 2 diabetes Phase III (continuous subcutaneous Boston, MA www.intarcia.com delivery of exenatide)

Janumet® Merck type 2 diabetes (pediatric) Phase III /metformin Kenilworth, NJ www.merck.com

Janumet® XR Merck type 2 diabetes (pediatric) Phase III sitagliptin/metformin Kenilworth, NJ www.merck.com extended-release

Januvia® Merck type 2 diabetes (pediatric) Phase III sitagliptin Kenilworth, NJ www.merck.com

Medicines in Development: Diabetes 6 Diabetes Drug Name Sponsor Indication Development Phase

Jardiance® Boehringer Ingelheim Pharmaceuticals type 1 diabetes Phase III empagliflozin Ridgefield, CT www.bowhringer-ingelheim.com Eli Lilly www.lilly.com Indianapolis, IN

type 2 diabetes (pediatric) Phase I www.bowhringer-ingelheim.com www.lilly.com

JNJ-54728518 Janssen Research & Development type 2 diabetes Phase I (GLP-1 receptor agonist) Raritan, NJ www.janssenrnd.com

JNJ-64565111 Janssen Research & Development type 2 diabetes Phase I (glucagon/GLP-1 dual agonist) Raritan, NJ www.janssenrnd.com

JTT-251 Akros Pharma type 2 diabetes Phase I (PDH kinase inhibitor) Princeton, NJ www.jt.com

JTT-851 Akros Pharma type 2 diabetes Phase II (G protein-coupled receptor 40) Princeton, NJ www.jt.com

KDT501 KinDex Pharmaceuticals insulin resistance and impaired Phase II (GPR120 protein stimulant) Seattle, WA glucose tolerance in type 2 diabetes www.kindexpharmaceuticals.com

KQ-791 Kaneq Bioscience type 2 diabetes Phase I (protein tyrosine phosphatase Montreal, Canada www.kaneq.com non receptor type 1 antagonist)

Medicines in Development: Diabetes 7 Diabetes Drug Name Sponsor Indication Development Phase

KU-5039 Kareus Therapeutics insulin resistance, type 2 diabetes Phase I (AMP kinase stimulant) La Chaux-de-Fonds, Switzerland www.kareustherapeutics.com

LC15-0444 LG Life Sciences type 2 diabetes Phase III completed (DPP-4 inhibitor) Seoul, South Korea www.lgls.com /metformin NuSirt Biopharma type 2 diabetes Phase II fixed-dose combination Nashville, TN www.nusirt.com

LGD-6972 Ligand Pharmaceuticals type 2 diabetes Phase II (glucagon receptor antagonist) San Diego, CA www.ligand.com

LIK066 Novartis Pharmaceuticals type 2 diabetes Phase II (SGLT 1/2 inhibitor) East Hanover, NJ www.novartis.com lingliptin/pioglitazone Boehringer-Ingelheim Pharmaceuticals type 2 diabetes Phase III fixed-dose combination Ridgefield, CT www.boehringer-ingelheim.com Eli Lilly www.lilly.com Indianapolis, IN

LixiLan Sanofi US type 2 diabetes application submitted /insulin glargine Bridgewater, NJ www.sanofi.com fixed-ratio (new delivery system)

LY900014 Eli Lilly type 1 diabetes, type 2 diabetes Phase I (glucose lowering drug) Indianapolis, IN www.lilly.com

Medicines in Development: Diabetes 8 Diabetes Drug Name Sponsor Indication Development Phase

Lyxumia® Sanofi US type 2 diabetes (pediatric) Phase I lixisenatide Bridgewater, NJ www.sanofi.com

MABp1 Xbiotech type 2 diabetes Phase II completed (IL-1 Alpha mAb) Austin, TX www.xbiotech.com

MEDI0382 MedImmune type 2 diabetes Phase I/II (GLP-1/glucagon dual agonist) Gaithersburg, MD www.medimmune.com

MEDI4166 MedImmune type 2 diabetes Phase II (PCSK9/GLP-1 mAb + peptide fusion) Gaithersburg, MD www.medimmune.com

MER3001 Mercia Pharma type 1 diabetes Phase II (regulatory T-lymphocyte stimulant) New York, NY www.merciapharma.com metformin DR Elcelyx Therapeutics type 2 diabetes Phase II (metformin delayed release) San Diego, CA www.elcelyx.com

MK-1293 Merck type 1 diabetes, type 2 diabetes application submitted (insulin glargine biosimilar) Kenilworth, NJ www.merck.com Samsung Bioepis www.samsungbioepis.com Seoul, South Korea

MK-2640 Merck type 1 diabetes Phase I Kenilworth, NJ www.merck.com

Medicines in Development: Diabetes 9 Diabetes Drug Name Sponsor Indication Development Phase

MK-8521 Merck type 2 diabetes Phase II (GLP-1/glucagon receptor co-agonist) Kenilworth, NJ www.merck.com

MK-8835A Merck type 2 diabetes Phase III (ertugliflozin + sitagliptin) Kenilworth, NJ www.merck.com Pfizer www.pfizer.com New York, NY

MK-8835B Merck type 2 diabetes Phase III (ertugliflozin + metformin) Kenilworth, NJ www.merck.com Pfizer www.pfizer.com New York, NY

MLR-1023 Melior Pharmaceuticals type 2 diabetes Phase II (lyn protein- stimulant) Exton, PA www.meliorpharmaceuticals.com

Nesina® Takeda Pharmaceuticals type 2 diabetes (pediatric) Phase II completed Deerfield, IL www.takeda.com

NGM313 NGM Biopharmaceuticals type 2 diabetes Phase I (KLB-FGFR1c modulator) South San Francisco, CA www.ngmbio.com

NM504 MicroBiome Therapeutics metformin intolerant type 2 diabetes Phase 0 (metagenome modulator) New Orleans, LA www.mbiome.com

NN1218 Novo Nordisk type 1 diabetes, type 2 diabetes application submitted (faster-acting ) Plainsboro, NJ www.novonordisk.com

Medicines in Development: Diabetes 10 Diabetes Drug Name Sponsor Indication Development Phase

NN1406 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (liver-preferential prandal Plainsboro, NJ www.novonordisk.com insulin analogue)

NN1436 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (long-acting basal insulin Plainsboro, NJ www.novonordisk.com analogue)

NN1953 Novo Nordisk type 1 diabetes, type 2 diabetes Phase II (long-acting basal insulin Plainsboro, NJ www.novonordisk.com analogue)

NN9748 Novo Nordisk type 2 diabetes Phase I (appetite-regulating hormone Plainsboro, NJ www.novonordisk.com peptide tyrosine)

NN9828 Novo Nordisk newly-diagnosed type 1 diabetes Phase II (anti-IL-21 mAb) Plainsboro, NJ (basal cell preservation) www.novonordisk.com

Onglyza® AstraZeneca type 2 diabetes (pediatric) Phase III Wilmington, DE www.astrazeneca.com

ORMD-0801 Oramed Pharmaceuticals type 1 diabetes, type 2 diabetes Phase II (insulin oral) Jerusalem, Israel www.oramed.com otelixizumab GlaxoSmithKline new onset type 1 diabetes Phase II (cluster of differentiation 3) Research Triangle Park, NC www.gsk.com ORPHAN DRUG

Medicines in Development: Diabetes 11 Diabetes Drug Name Sponsor Indication Development Phase

PB1023 PhaseBio Pharmaceuticals type 2 diabetes Phase II (weekly GLP-1 agonist) Malvern, PA www.phasebio.com

PE0139 PhaseBio Pharmaceuticals type 2 diabetes Phase II (weekly basal insulin) Malvern, PA www.phasebio.com

PF-06291874 Pfizer type 2 diabetes Phase II (glucagon receptor antagonist) New York, NY www.pfizer.com

PF-06293620 Pfizer type 2 diabetes Phase I New York, NY www.pfizer.com

PF-06342674 Pfizer type 1 diabetes Phase I (IL-7 receptor inhibitor) New York, NY www.pfizer.com

Prolastin®-C Grifols new-onset type 1 diabetes Phase II alpha-1-antitrypsin Los Angeles, CA www.grifolsusa.com modified process

Qtern AstraZeneca type 2 diabetes application submitted saxagliptin/dapagliflozin Wilmington, DE www.astrazeneca.com fixed-dose combination

REMD-477 REMD Biotherapeutics type 1 diabetes, type 2 diabetes Phase II (GPCR antagonist) Camarillo, CA www.remdbio.com

Medicines in Development: Diabetes 12 Diabetes Drug Name Sponsor Indication Development Phase Avolynt type 2 diabetes Phase II (SGLT2 inhibitor) Research Triangle Park, NC www.avolynt.com

Ryzodeg® Novo Nordisk type 1 diabetes (pediatric) Phase III completed /insulin aspart Plainsboro, NJ www.novonordisk.com

SAR342434 Sanofi US type 1 diabetes, type 2 diabetes Phase III () Bridgewater, NJ www.sanofi.com

SAR425899 Sanofi US type 2 diabetes Phase I (GLP-1/GCGR dual agonist) Bridgewater, NJ www.sanofi.com

SAR438335 Sanofi US type 2 diabetes Phase I (GLP-1R/GIPR dual agonist) Bridgewater, NJ www.sanofi.com

SAR440067 Sanofi US type 1 diabetes, type 2 diabetes Phase I (LAPS insulin 115; long acting Bridgewater, NJ www.sanofi.com ) (NN9535) Novo Nordisk type 2 diabetes Phase III (GLP-1 analogue) Plainsboro, NJ www.novonordisk.com semaglutide oral (NN9924) Novo Nordisk type 2 diabetes Phase III (GLP-1 analogue) Plainsboro, NJ www.novonordisk.com

Medicines in Development: Diabetes 13 Diabetes Drug Name Sponsor Indication Development Phase

Simponi® Janssen Research & Development type 1 diabetes Phase II golimumab Raritan, NJ www.janssenrnd.com Sanofi US type 1 diabetes Phase III (oral SGLT-1/2 inhibitor) Bridgewater, NJ www.sanofi.com

type 2 diabetes Phase II www.sanofi.com

Tanzeum® GlaxoSmithKline type 1 diabetes Phase II Research Triangle Park, NC www.gsk.com (MP-513) Mitsubishi Tanabe Pharma type 2 diabetes Phase I (DPP-4 inhibitor) Development America www.mt-pharma-development-america.com Jersey City, NJ teplizumab MacroGenics type 1 diabetes (prevention) Phase II (CD3 antigen inhibitor) Rockville, MD www.macrogenics.com ORPHAN DRUG

TF0023 Techfields Pharma type 2 diabetes Phase II (thrombin inhibitor) Dover, DE www.tfpharma.com

Toujeo® Sanofi US type 1 diabetes (pediatric) Phase III insulin glargine (U300) Bridgewater, NJ www.sanofi.com

Medicines in Development: Diabetes 14 Diabetes Drug Name Sponsor Indication Development Phase

Tradjeta® Boehringer Ingelheim Pharmaceuticals type 2 diabetes (pediatric) Phase II insulin glargine Bridgewater, NJ www.sanofi.com Eli Lilly www.lilly.com Indianapolis, IN

TT401 Transition Therapeutics type 2 diabetes in patients with Phase II completed (glucagon-like peptide-1/glucagon Toronto, Canada accompanying obesity www.transitiontherapeutics.com dual receptor agonist)

TTP273 vTv Therapeutics type 2 diabetes Phase II (oral GLP-1R agonist) High Point, NC www.vtvtherapeutics.com

TTP399 vTv Therapeutics type 2 diabetes Phase II (glucokinase activator) High Point, NC www.vtvtherapeutics.com ultra-rapid insulin Adocia type 1 diabetes, type 2 diabetes Phase II (BioChaperone® insulin lispro) Lyon, France www.adocia.fr Eli Lilly www.lilly.com Indianapolis, IN ultra-rapid insulin Eli Lilly type 1 diabetes, type 2 diabetes Phase II (insulin lispro UR formulation) Indianapolis, IN www.lilly.com

U-Strip Diabetes Transdermal Specialties type 1 diabetes, type 2 diabetes Phase II insulin transdermal Broomall, PA www.transdermalspecialities.com

VC-01 ViaCyte type 1 diabetes Phase I/II (pancreatic beta cell replacement) San Diego, CA www.viacyte.com

Medicines in Development: Diabetes 15 Diabetes Drug Name Sponsor Indication Development Phase

Victoza® Novo Nordisk type 2 diabetes (pediatric) Phase III Plainsboro, NJ www.novonordisk.com

VK0612 Viking Therapeutics type 2 diabetes Phase II (FBPase inhibitor) San Diego, CA www.vikingtherapeutics.com

Xultophy® Novo Nordisk type 2 diabetes application submitted insulin degludec/liraglutide Plainsboro, NJ www.novonordisk.com

ZP2929 Zealand Pharma type 1 diabetes Phase I (glucagon/GLP-1 receptor agonist) Copenhagen, Denmark www.zealandpharma.com

ZYDPLA 1 Cadila Healthcare type 2 diabetes Phase I completed (CD26 antigen inhibitor) Gujarat, India www.zyduscadila.com

Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase abicipar pegol Allergan diabetic macular edema Phase II completed (VEGF-A inhibitor) Parsippany, NJ www.allergan.com Molecular Partners Zurich, Switzerland

Acthar® Gel Mallinckrodt diabetic nephropathy Phase II corticotropin gel St. Louis, MO www.mallinckrodt.com

Medicines in Development: Diabetes 16 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

AKB-9778 Aerpio Therapeutics diabetic macular edema Phase II (Tie-2 activator) Cincinnati, OH www.aerpio.com

ALG-1001 Allegro Ophthalmics diabetic macular edema, Phase II ( therapy) San Juan Capistrano, CA non-proliferative diabetic retinopathy www.allegroeye.com alpha-2c adrenergic receptor Bayer HealthCare Pharmaceuticals diabetic foot ulcers Phase I antagonist Whippany, NJ www.bayer.com

AmiKet™ Immune Pharmaceuticals painful diabetic neuropathy Phase II completed /ketamine New York, NY www.immunepharma.com topical cream

ARA 290 Araim Pharmaceuticals diabetic macular edema Phase I ( receptor agonist) Tarrytown, NY www.araimpharma.com

ASP8232 Astellas Pharma US diabetes macular edema Phase II (VAP-1 inhibitor) Northbrook, IL www.astellas.com AbbVie diabetic nephropathy in Phase III ( A antagonist) North Chicago, IL patients with type 2 diabetes www.abbvie.com

BI 1026706 Boehringer Ingelheim Pharmaceuticals diabetic macular edema Phase II Ridgefield, CT www.boehringer-ingelheim.com

Medicines in Development: Diabetes 17 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

Brilinta® AstraZeneca cardiovascular outcomes in patients Phase III ticagrelor Wilmington, DE with type 2 diabetes and coronary www.astrazeneca.com artery disease, but without a previous history of myocardial infarction or stroke

Bydureon® AstraZeneca cardiovascular outcomes in Phase III exenatide extended release Wilmington, DE type 2 diabetes www.astrazeneca.com GlaxoSmithKline diabetic gastroparesis Phase II ( receptor agonist) Research Triangle Park, NC www.gsk.com clonidine topical gel BioDelivery Sciences International painful diabetic neuropathy Phase III (alpha 2 adrenergic receptor agonist) Raleigh, NC (Fast Track) www.bdsi.com

CureXcell® MacroCure diabetic foot ulcer Phase III leukocyte cell therapy Radnor, PA www.macrocure.com

CVBT-141B CardioVascular BioTherapeutics diabetic foot ulcers Phase II (fibroblast -1) Dallas, TX www.cvbt.com

DM199 DiaMedica diabetic nephropathy Phase I (recombinant tissue Minneapolis, MN www.diamedica.com kallikrein-1 [rKLK1]) emixustat Acucela proliferative diabetic retinopathy Phase II ( isomerohydrolase inhibitor) Seattle, WA www.acucela.com

Medicines in Development: Diabetes 18 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

ETX-101 ETX Pharma diabetic gastroparesis Phase III Oakland, MD www.etxpharma.com

EVK-001 Evoke Pharma diabetic gastroparesis Phase III (dopamine receptor antagonist) Solana Beach, CA www.evokepharma.com

ExpressGraft™ Stratatech diabetic foot ulcers Phase I anti-infective skin Madison, WI www.stratatechcorp.com replacement therapy

Eylea® Bayer HealthCare Pharmaceuticals diabetic retinopathy without Phase III Whippany, NJ macular edema www.bayer.com Regeneron Pharmaceuticals www.regeneron.com Tarrytown, NY

Eylea® aflibercept + nesvacumab Bayer HealthCare Pharmaceuticals diabetic macular edema Phase II (Ang2 antibody) Whippany, NJ www.bayer.com Regeneron Pharmaceuticals www.regeneron.com Tarrytown, NY

Farixga® AstraZeneca cardiovascular outcomes in Phase III dapagliflozin Wilmington, DE type 2 diabetes www.astrazeneca.com finerenone Bayer HealthCare Pharmaceuticals diabetic nephropathy Phase III (mineralocorticoid receptor antagonist) Plainsboro, NJ www.bayer.com

Galnobax® NovaLead Pharma diabetic foot ulcer Phase I/II beta-1 adrenergic receptor agonist Shrewsbury, MA www.novaleadpharma.com

Medicines in Development: Diabetes 19 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

Granexin™ Gel FirstString Research diabetic foot ulcer Phase III ACT 1 peptide wound-healing Mount Pleasant, SC www.firststringresearch.com gel formulation

GS-4997 Gilead Sciences diabetic nephropathy Phase II (ASK-1 inhibitor) Foster City, CA www.gilead.com

GSK1278863 GlaxoSmithKline diabetic foot ulcers Phase I (topical prolyl hydroxylase Research Triangle Park, NC www.gsk.com inhibitor mAb)

IBI-20089 Icon Bioscience diabetic macular edema Phase I (glucocorticoid receptor agonist) Sunnyvale, CA www.iconbioscience.com

INL-002 Innocoll diabetic foot ulcers Phase III (gentamicin topical Newtown Square, PA www.innocoll.com matrix implant)

Invokana® Janssen Research & Development diabetic nephropathy in patients Phase III canagliflozin Raritan, NJ with type 2 diabetes www.janssenrnd.com

Jardiance® Boehringer Ingelheim Pharmaceuticals cardiovascular outcomes in application submitted empagliflozin Ridgefield, CT type 2 diabetes www.boehringer-ingelheim.com Eli Lilly www.lilly.com Indianapolis, IN

Jetrea® ThromboGenics diabetic retinopathy Phase II ocriplasmin Iselin, NJ www.thrombogenics.com

Medicines in Development: Diabetes 20 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

KVD001 KalVista Pharmaceuticals diabetic macular edema Phase I (plasma kallikrein inhibitor) Cambridge, MA www.kalvista.com

Locilex® Dipexium Pharmaceuticals mild diabetic foot infection Phase III pexiganan New York, NY www.dipexiumpharmaceuticals.com

MPC-300-IV Mesoblast diabetic nephropathy Phase II (mesenchymal stem cell therapy) New York, NY www.mesoblast.com

MT-3995 Mitsubishi Tanabe Pharma diabetic nephropathy Phase I (selective mineralocorticoid Development America www.mt-pharma-development-america.com receptor antagonist) Jersey City, NJ nasal glucagon Eli Lilly severe hypoglycemia in patients Phase III Indianapolis, IN with diabetes treated with insulin www.lilly.com nemonoxacin TaiGen Biotechnology diabetic foot ulcers Phase II completed (DNA ) Taipei, Taiwan www.taigenbiotech.com.tw nepafenac Alcon diabetic retinopathy Phase III (cyclooxygenase inhibitor) Fort Worth, TX www.alcon.com

NM504 MicroBiome Therapeutics diabetic dysbiosis (microbial imbalance), Phase I (metagenome modulator) New Orleans, LA prediabetes www.mbiome.com

Medicines in Development: Diabetes 21 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

NOV-9 MorphoSys diabetic retinopathy Phase I Martinsried/Planegg, Germany www.novartis.com Novartis Pharmaceuticals East Hanover, NJ

Nu-3 topical Lakewood Amedex diabetic foot ulcers Phase I/II (cell membrane structure modulator) Sarasota, FL www.lakewoodamedex.com

OC-10X OcuCure Therapeutics proliferative diabetic retinopathy Phase I completed (vascular disrupting agent) Roanoke, VA www.ocucure.com

Optina Ampio Pharmaceuticals diabetic macular edema Phase II danazol low-dose Englewood, CO www.ampiopharma.com

PAN-90806 PanOptica diabetic retinopathy Phase I (VEGF inhibitor) Bernardsville, NJ www.panopticapharma.com

PDA-002 Celgene diabetic foot ulcers, Phase II (human placental-derived Summit, NJ diabetic neuropathy www.celgene.com adherent cell therapy)

PF-655 Quark Pharmaceuticals diabetic macular edema Phase II (RNA interference) Fremont, CA www.quarkpharma.com

PMZ-2123 Pharmazz brain edema in diabetic Phase I ( antagonist) Willowbrook, IL ketoacidosis www.pharmazz.com

Medicines in Development: Diabetes 22 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase relamorelin Allergan diabetic gastroparesis (Fast Track) Phase II (ghrelin peptide agonist) Parsippany, NJ www.allergan.com Motus www.motusrx.com Boston, MA

RG-125/AZD4076 AstraZeneca non-alcoholic fatty liver disease Phase I (microRNA therapeutic) Wilmington, DE (NAFLD) in patients with type 2 www.astrazeneca.com Regulus Therapeutics diabetes or prediabetes www.regulusrx.com San Diego, CA

RG7880 Genentech diabetic foot ulcers Phase I (IL-22 modulator) South San Francisco, CA www.gene.com

RO6867461 Roche center-involving diabetic Phase II Basel, Switzerland macular edema www.roche.com

SF0166 SciFluor Life Sciences diabetic macular edema Phase I/II (integrin alphaVbeta3 antagonist) Cambridge, MA www.scifluor.com squalamine Ohr Pharmaceutical diabetic macular edema, Phase II (VEGF/PDGF/bFGF inhibitor) New York, NY proliferative diabetic retinopathy www.ohrpharmaceutical.com River Vision Development diabetic macular edema Phase I completed (IGF-IR antagonist) New York, NY

Medicines in Development: Diabetes 23 Diabetes-Related Conditions Drug Name Sponsor Indication Development Phase

Tradjenta® Boehringer Ingelheim Pharmaceuticals cardiovascular outcomes in Phase III Ridgefield, CT type 2 diabetes, diabetic www.boehringer-ingelheim.com Eli Lilly nephropathy www.lilly.com Indianapolis, IN

TV1001sr TheraVasc diabetic neuropathy Phase II (sodium nitrite oral) Cleveland, OH www.theravasc.com

VM202 ViroMed painful diabetic peripheral Phase III (modified hepatocyte growth Seoul, South Korea neuropathy, chronic non-healing www.viromed.co.kr factor gene therapy) ischemic diabetic foot ulcers

VPI-2690B Vascular Pharmaceuticals diabetic nephropathy Phase II (IGF-1 receptor antagonist) Research Triangle Park, NC www.vascularpharma.com

The content of this report has been obtained through public, government and industry sources, and the Springer "Adis Insight” database based on the latest information. Report current as of October 12, 2016. The medicines in this listing include medicines being developed by U.S.-based companies conducting trials in the United States and abroad, PhRMA-member companies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in the United States. Some products may not be in active clinical trials. The information may not be comprehensive. For more, specific information about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA's website, www.phrma.org.

Definitions

Application Submitted—An application for marketing has been submitted by the company to the U.S. Food and Drug Administration (FDA).

Breakthrough Therapy—Upon request by a sponsor, the FDA can grant this designation to expedite the development and review of a drug or biologic intended, alone or in combination with one or more other drugs, to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over existing therapies on one or more clinically-significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug or biologic is designated as a breakthrough therapy, the FDA will expedite the development and review. With this designation, all Fast Track features convey to the medicine.

Medicines in Development: Diabetes 24 Fast Track—Upon request by a sponsor, the FDA can grant this designation to facilitate the development and expedite the review of a drug or biologic to treat a serious condition and fill an unmet medical need. When considering a biopharmaceutical company’s request for Fast Track designation for an investigational drug or biologic, the FDA evaluates whether it will affect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious one, and whether a condition can be adequately addressed by available therapy. With Fast Track designation, early and frequent communication between the FDA and the biopharmaceutical company is encouraged throughout the entire drug development and review process to help to quickly resolve any questions or issues that arise, potentially leading to an earlier approval and access by patients.

Orphan Designation—Upon request by a sponsor, the FDA can grant special status (“orphan designation”) to a drug or biologic to treat a rare disease or condition. In order to receive an orphan designation, a qualifying drug or biologic must be intended for the treatment, diagnosis, or prevention of a rare disease or condition that affects usually fewer than 200,000 people in the United States.

Phase 0—First-in-human trials conducted in accordance with FDA's 2006 guidance on exploratory Investigational New Drug (IND) studies designed to speed up development of promising drugs by establishing very early on whether the agent behaves in human subjects as was anticipated from preclinical studies.

Phase I—Researchers test the investigational drug or biologic in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate its initial safety and tolerability profile, determine a safe dosage range, and identify potential side effects.

Phase II—The investigational drug or biologic is given to volunteer patients, usually between 100 and 500, to determine whether it is effective, identify an optimal dose, and to further evaluate its short-term safety.

Phase III—The investigational drug or biologic is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients (but sometimes many more), to generate statistically significant evidence to confirm its safety and effectiveness. Phase III studies are the longest studies and usually take place in multiple sites around the world.

Medicines in Development: Diabetes 25