2016 Medicines in Development for Diabetes and Related Conditions Diabetes Drug Name Sponsor Indication Development Phase AC-201 TWi Biotechnology type 2 diabetes Phase II completed (caspase 1 inhibitor/ Taipei, Taiwan www.twibiotech.com IL-1 beta inhibitor) anagliptin (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/insulin 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 (glucagon-like peptide 1 stimulant) Chapel Hill, NC www.biokier.com Medicines in Development: Diabetes 1
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2016 Medicines in Development for Diabetes and Related ConditionsDiabetesDrug Name Sponsor Indication Development Phase
AC-201 TWi Biotechnology type 2 diabetes Phase II completed(caspase 1 inhibitor/ Taipei, Taiwan www.twibiotech.comIL-1 beta inhibitor)
anagliptin (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/insulin sensitizer) New York, NY www.n-gene.net
BTI-320 Boston Therapeutics type 2 diabetes Phase II(alpha glucosidase inhibitor-chewable) Newton, MA www.bostonti.com
Bydureon® AstraZeneca type 2 diabetes Phase IIIexenatide extended release Wilmington, DE (weekly suspension) www.astrazeneca.com
type 2 diabetes (pediatric) Phase IIIwww.astrazeneca.com
Byetta® AstraZeneca type 2 diabetes (pediatric) Phase IIIexenatide Wilmington, DE www.astrazeneca.com
CLBS03 Caladrius Biosciences type 1 diabetes Phase II(Treg cell-based therapy) Basking Ridge, NJ (Fast Track) www.caladrius.comORPHAN DRUG
Cycloset® VeroScience type 2 diabetes (pediatric) Phase Ibromocriptine Tiverton, RI www.veroscience.com
Dance-501 Dance Biopharma type 1 diabetes, type 2 diabetes Phase II(inhaled recombinant human Brisbane, CA www.dancebiopharma.cominsulin)
Medicines in Development: Diabetes 2
DiabetesDrug Name Sponsor Indication Development Phase
Diamyd® Diamyd Medical newly-diagnosed type 1 diabetes Phase Iautoimmune diabetes vaccine Stockholm, Sweden www.diamyd.comORPHAN DRUG University of Alabama at Birmingham
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.comnon receptor type 1 antagonist)
Medicines in Development: Diabetes 7
DiabetesDrug 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
TT401 Transition Therapeutics type 2 diabetes in patients with Phase II completed(glucagon-like peptide-1/glucagon Toronto, Canada accompanying obesity www.transitiontherapeutics.comdual 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.comIndianapolis, 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 IIinsulin 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
DiabetesDrug Name Sponsor Indication Development Phase
Victoza® Novo Nordisk type 2 diabetes (pediatric) Phase IIIliraglutide 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 submittedinsulin degludec/liraglutide Plainsboro, NJ www.novonordisk.com
Locilex® Dipexium Pharmaceuticals mild diabetic foot infection Phase IIIpexiganan 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.comreceptor antagonist) Jersey City, NJ
nasal glucagon Eli Lilly severe hypoglycemia in patients Phase IIIIndianapolis, IN with diabetes treated with insulin www.lilly.com
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.comSan Diego, CA
RG7880 Genentech diabetic foot ulcers Phase I(IL-22 modulator) South San Francisco, CA www.gene.com
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 Statesand abroad, PhRMA-member companies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in the United States. Someproducts may not be in active clinical trials. The information may not be comprehensive. For more, specific information about a particular product, contact the individualcompany directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA's website, www.phrma.org.
DefinitionsApplication 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, aloneor 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 maydemonstrate substantial improvement over existing therapies on one or more clinically-significant endpoints, such as substantial treatment effects observed early inclinical 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 aserious 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 studiesand usually take place in multiple sites around the world.
