2015 Medicines in Development for Neurological DisordersA Report on Disorders of the Brain, Spinal Cord and Nerves
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
AC-1204 Accera mild to moderate Alzheimer's disease Phase II/III(caprylic triglyceride) Broomfield, CO www.accerapharma.com
ACI-35 AC Immune Alzheimer's disease Phase I(active anti-Tau vaccine) Lausanne, Switzerland www.acimmune.com
Janssen Research & Development www.janssenrnd.com
Raritan, NJ
AD-4833/TOMM40 Takeda Pharmaceutical delay onset of mild cognitive Phase III(insulin sensitizer) Deerfield, IL impairment due to Alzheimer's www.takeda.com
Zinfandel Pharmaceuticals disease
Chapel Hill, NC
aducanumab (BIIB037) Biogen Alzheimer's disease Phase I
(amyloid beta-protein inhibitor) Cambridge, MA www.biogen.com
ALZ-801 Alzheon Alzheimer's disease Phase I(amyloid beta-protein inhibitor) Framingham, MA www.alzheon.com
ANAVEX Plus Anavex Life Sciences Alzheimer's disease Phase II
(ANAVEX 2-73/donepezil) New York, NY www.anavex.com
ASP3662 Astellas Pharma US Alzheimer's disease Phase I
Northbrook, IL www.astellas.us
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
AVN-101 AllaChem Alzheimer's disease Phase II
(serotonin 6 receptor antagonist) Hallandale, FL www.allachem.comAvineuro Pharmaceuticals
San Diego, CA
AVN-322 AllaChem Alzheimer's disease Phase II
(serotonin 6 receptor antagonist) Hallandale, FL www.allachem.comAvineuro Pharmaceuticals
San Diego, CA
AVP-786 Avanir Pharmaceuticals agitation in Alzheimer's disease Phase I(dextromethorphan analogue/quinidine) Aliso Viejo, CA www.avanir.com
Concert Pharmaceuticals www.concertpharma.com
Lexington, MA
AVP-923 Avanir Pharmaceuticals agitation in Alzheimer's disease Phase II
(dextromethorphan/quinidine San Diego, CA (see also headache, Parkinson's) www.avanir.com
fixed-dose combination)
AZD3293 AstraZeneca Alzheimer's disease Phase II
(beta-secretase inhibitor) Wilmington, DE www.astrazeneca.com
Eli Lilly www.lilly.com
Indianapolis, IN
azeliragon (TTP488) vTv Therapeutics Alzheimer's disease (Fast Track) Phase III
(RAGE antagonist) High Point, NC www.vtvtherapeutics.com
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
BACE inhibitor CoMentis Alzheimer's disease Phase I completed
South San Francisco, CA www.comentis.com
BACE inhibitor Eli Lilly Alzheimer's disease Phase I
Indianapolis, IN www.lilly.com
BACE inhibitor Janssen Pharmaceuticals early-stage Alzheimer's disease Phase II
Titusville, NJ www.janssenpharmaceuticalsinc.com
Shionogi www.shionogi.co.jp
Osaka, Japan
BAN2401 Biogen Alzheimer's disease Phase II
(amyloid beta-protein inhibitor) Cambridge, MA www.biogen.com
Eisai www.eisai.com
Woodcliff Lake, NJ
BI 409306 Boehringer Ingelheim Alzheimer's disease Phase II
(phosphodiesterase 9A inhibitor) Pharmaceuticals www.boehringer-ingelheim.com
Ridgefield, CT
bisnorcymserine QR Pharma advanced Alzheimer's disease Phase I
(BNC) Berwyn, PA www.qrpharma.com
bryostatin-1 Neurotrope BioScience Alzheimer's disease Phase II
Newark, NJ www.neurotropebioscience.com
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
CAD106 GlaxoSmithKline Alzheimer's disease Phase II completed
(beta-amyloid protein inhibitor) Research Triangle Park, NC www.gsk.com
CPC-201 Chase Pharmaceuticals Alzheimer's disease Phase II
Washington, DC www.chasepharmaceuticals.com
crenezumab Genentech mild to moderate Alzheimer's disease Phase II
(beta-amyloid protein inhibitor) South San Francisco, CA www.gene.com
E2609 Biogen Alzheimer's disease Phase II(BACE1 protein inhibitor) Cambridge, MA www.biogen.com
Eisai www.eisai.com
Woodcliff Lake, NJ
ELND005 Transition Therapeutics agitation/aggression in Alzheimer's Phase II
(amyloid-beta protein inhibitor) Toronto, Canada disease (Fast Track) www.transitiontherapeutics.com
encenicline Forum Pharmaceuticals Alzheimer's disease Phase III(alpha-7 potentiator) Watertown, MA www.forumpharma.com
gantenerumab Genentech early-stage Alzheimer's disease Phase III
(amyloid-beta protein inhibitor) South San Francisco, CA www.gene.com
GC021109 GliaCure mild to moderate Alzheimer's disease Phase I(purinoceptor P2Y6 agonist) Boston, MA (Fast Track) www.gliacure.com
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
idalopirdine Lundbeck Alzheimer's disease Phase III
(serotonin 6 receptor antagonist) Deerfield, IL www.lundbeck.com
Otsuka Pharmaceutical www.otsuka.co.jp
Tokyo, Japan
immune globulin Grifols USA Alzheimer's disease Phase III
Los Angeles, CA www.grifols.com
JNJ-54861911 Janssen Research & Development Alzheimer's disease Phase II
Raritan, NJ www.janssenrnd.com
LMTX TauRx Pharmaceuticals Alzheimer's disease Phase III(tau protein aggregation/ Singapore www.taurx.comTDP-43 aggregation inhibitor)
Lu-AF20513 Lundbeck Alzheimer's disease Phase I
(amyloid beta-protein inhibitor) Deerfield, IL www.lundbeck.com
Otsuka Pharmaceutical www.otsuka.co.jp
Tokyo, Japan
LY3002813 Eli Lilly Alzheimer's disease Phase I(N3pG-Aß mAb) Indianapolis, IN www.lilly.com
MEDI1814 MedImmune Alzheimer's disease Phase I
(anti-amyloid beta mAb) Gaithersburg, MD www.medimmune.com
mGlu2 agonist Eli Lilly Alzheimer's disease psychosis Phase I
Indianapolis, IN www.lilly.com
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
MK-7622 Merck Alzheimer's disease Phase II
(quinazolinone) Whitehouse Station, NJ (adjunct therapy) www.merck.com
MK-8931 Merck Alzheimer's disease Phase III
(BACE1 protein inhibitor) Whitehouse Station, NJ www.merck.com
MSDC-0160 Metabolic Solutions Development Alzheimer's disease Phase II completed(mitochondrial protein stimulant) Kalamazoo, MI www.msdrx.com
NIC-515 Humanetics Alzheimer's disease Phase II(amyloid precursor protein secretase Minneapolis, MN www.humaneticscorp.com
inhibitor)
PF-05212377 (SAM-760) Pfizer Alzheimer's disease Phase II
(5HT6 antagonist) New York, NY www.pfizer.com
PF-06648671 Pfizer Alzheimer's disease Phase I
New York, NY www.pfizer.com
Posiphen® QR Pharma Alzheimer's disease Phase II
R-phenserine Berwyn, PA (see also Parkinson's) www.qrpharma.com
PTI-80 ProteoTech Alzheimer's disease Phase I
(amyloid beta/tau protein inhibitor) Kirkland, WA www.proteotech.com
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
RG1577 Roche Alzheimer's disease Phase II
(MAO-B inhibitor) New York, NY www.roche.com
RG7345 Roche Alzheimer's disease Phase I
(anti-Tau mAb) New York, NY www.roche.com
rilapladib GlaxoSmithKline Alzheimer's disease Phase II
(Lp-PLA2 inhibitor) Research Triangle Park, NC www.gsk.com
RVT-101 Axovant Sciences mild to moderate Alzheimer's disease Phase II completed(5-HT6 receptor antagonist) New York, NY www.axovant.com
RVX208 Resverlogix Alzheimer's disease Phase I completed(BET protein inhibitor) Calgary, Canada www.resverlogix.com
SAR228810 Sanofi US Alzheimer's disease Phase I
(anti-protofibrillar AB mAb) Bridgewater, NJ www.sanofi.com
sGC 1061 sGC Pharma Alzheimer's disease Phase I(nomethiazole) Vancouver, Canada sgcpharma.com
solanezumab Eli Lilly Alzheimer's disease Phase III
(amyloid beta protein inhibitor) Indianapolis, IN www.lilly.com
SUVN-502 Suven Life Sciences Alzheimer's disease Phase I(serotonin 6 receptor antagonist) Hyderabad, India www.suven.com
Alzheimer's Disease
Product Name Sponsor Indication Development Phase
SUVN-G3031 Suven Life Sciences cognitive dysfunction in Phase I(histamine H3 receptor antagonist) Hyderabad, India Alzheimer's disease www.suven.com
T-817MA Toyama Chemical Alzheimer's disease Phase II(amyloid beta-protein inhibitor) Tokyo, Japan www.toyama-chemical.co.jp
T3D-959 T3D Therapeutics Alzheimer's disease Phase I(PPAR-delta/gamma agonist) Research Triangle Park, NC www.t3dtherapeutics.com
TPI 287 Cortice Biosciences mild to moderate Alzheimer's disease Phase I
(abeotaxane) New York, NY (see also brain tumors, other) www.corticebio.com
UB-311 United Biomedical Alzheimer's disease Phase I(amyloid beta-protein inhibitor) Hauppauge, NY www.unitedbiomedical.com
VX-745 EIP Pharma Alzheimer's disease Phase II
(p38 mitogen-activated protein Cambridge, MA www.eippharma.com
kinase inhibitor)
Amyotrophic Lateral Sclerosis
Product Name Sponsor Indication Development Phase
arimoclomal ALS Foundation hereditary or familial Phase II/III
Orphan Drug Washington, DC amyotrophic lateral sclerosis (ALS) www.orphazyme.com
Orphazyme (Fast Track)
Copenhagen, Denmark
University of Miami
Miami, FL
CC100 Chemigen ALS Phase I completed
Indianapolis, IN
dexpramipexole Knopp Biosciences ALS (Fast Track) Phase II
Orphan Drug Pittsburgh, PA www.knoppbio.com
GM6 Genervon Biopharmaceuticals ALS (Fast Track) Phase II
(peptide therapeutic) Pasadena, CA (see also Parkinson's, stroke) www.genervon.com
Orphan Drug
H.P. Acthar® Gel Mallinckrodt Pharmaceuticals ALS Phase II
repository corticotropin injection St. Louis, MO www.mallinckrodt.com
MN-166 MediciNova ALS Phase II
(ibudilast) La Jolla, CA (see also multiple sclerosis) www.medicinova.com
neurotrophic factor-producing adult BrainStorm Cell Therapeutics ALS (Fast Track) Phase II
stem cell therapy (MSC-NTF cells) Hackensack, NJ www.brainstorm-cell.com
Orphan Drug
Amyotrophic Lateral Sclerosis
Product Name Sponsor Indication Development Phase
NP001 Neuraltus Pharmaceuticals ALS Phase II(macrophage modulator) Palo Alto, CA www.neuraltus.com
NSI-566 Neuralstem ALS Phase II(spinal cord-derived neural Germantown, MD (see also spinal cord injury, stroke) www.neuralstem.comstem cell therapy)
Orphan Drug
ozanezumab GlaxoSmithKline ALS Phase II
(NOGO-A inhibitor) Research Triangle Park, NC www.gsk.com
tirasemtiv Cytokinetics ALS (Fast Track) Phase II
(troponin complex stimulant) South San Francisco, CA www.cytokinetics.com
Orphan Drug
VM202 ViroMed ALS Phase I/II
(modified hepatocyte growth factor Seoul, South Korea (see also pain) www.viromed.co.kr
gene therapy)
Brain Injury
Product Name Sponsor Indication Development Phase
Cirara™ Remedy Pharmaceuticals traumatic brain injury Phase II completed
RP-1128 New York, NY (see also stroke) www.remedypharmaceuticals.com
Brain Injury
Product Name Sponsor Indication Development Phase
NH001 NeuroHealing Pharmaceuticals vegetative state (coma) or minimally Phase II
(apomorphine subcutaneous) Waban, MA conscious state following traumatic www.neurohealing.com
Orphan Drug brain injury (Fast Track)
NNZ-2256 Neuren Pharmaceuticals traumatic brain injury (Fast Track) Phase II
(cytokine inhibitor/neuropeptide Bethesda, MD (see also genetic) www.neurenpharma.com
receptor modulator)
VOLT02 Levolta Pharmaceuticals traumatic brain injury Phase II
(progesterone receptor agonist) Bethlehem, PA www.levoltapharma.com
Brain Tumors
Product Name Sponsor Indication Development Phase
2B3-101 BBB Therapeutics glioma Phase I(doxorubicin liposomal) Leiden, Netherlands www.bbbtherapeutics.com
Orphan Drug
ABT-414 AbbVie glioblastoma Phase I/II(monoclonal antibody-drug conjugate) North Chicago, IL www.abbvie.com
Orphan Drug
ADU-623 Aduro Biotech glioblastoma Phase I
(therapeutic bivalent vaccine) Berkeley, CA www.aduro.com
Brain Tumors
Product Name Sponsor Indication Development Phase
AG-120 Agios Pharmaceuticals glioma Phase I
(IDH1 inhibitor) Cambridge, MA www.agios.com
aglatimagene besadenovec Advantagene glioma Phase II
(gene therapy) Auburndale, MA www.advantagene.com
Orphan Drug
glioma (pediatric) Phase I/II
www.advantagene.com
aldoxorubicin CytRx glioblastoma Phase II(delivery-enhanced doxorubicin) Los Angeles, CA www.cytrx.com
AMG 595 Amgen anaplastic astrocytoma, Phase I(anti-EGFRvIII antibody-drug conjugate) Thousand Oaks, CA recurrent glioblastoma www.amgen.com
ANG 1005 Angiochem recurrent glioblastoma (Fast Track) Phase II
(peptide-paclitaxel conjugate) Montreal, Canada www.angiochem.com
Orphan Drug
antineoplaston A10/AS2-1 Burzynski Research Institute diffuse intrinsic brainstem glioma Phase II
Orphan Drug Houston, TX www.burzynskiresearch.com
Avastin® Genentech glioblastoma (1st-line) Phase III
bevacizumab South San Francisco, CA www.gene.com
BGJ398 Novartis Pharmaceuticals recurrent glioblastoma Phase II(FGF-R kinase inhibitor) East Hanover, NJ www.novartis.com
Brain Tumors
Product Name Sponsor Indication Development Phase
buparlisib Novartis Pharmaceuticals glioblastoma Phase I/II
(PI3K inhibitor) East Hanover, NJ www.novartis.com
Cometriq™ Exelixis astrocytoma, glioblastoma Phase II completed
cabozantinib South San Francisco, CA www.exelixis.com
Cotara® Peregrine Pharmaceuticals recurrent glioblastoma (Fast Track) Phase II completed
iodine I 131 derlotuximab biotin Tustin, CA www.peregrineinc.com
Orphan Drug
crenolanib Arog Pharmaceuticals glioma (pediatric) Phase II(PDGFR inhibitor) Dallas, TX www.arogpharma.com
CTO Tactical Therapeutics glioma (combination therapy) Phase I/II
(carboxyamidotriazole orotate) New York, NY (Fast Track) www.tacticaltherapeutics.com
DCVax®-L Northwest Biotherapeutics glioblastoma Phase IIIdendritic cell-based vaccine Bethesda, MD www.nwbio.com
Orphan Drug
DM-CHOC-PEN Dekk-Tek brain tumors Phase II(non-neurotoxic derivative of New Orleans, LA www.dekk-tec.com
penclomidine)
DNX-2401 DNAtrix glioblastoma (Fast Track) Phase I(oncolytic virus) Houston, TX www.dnatrix.com
Orphan Drug
Brain Tumors
Product Name Sponsor Indication Development Phase
E7050 Eisai recurrent glioblastoma Phase I/II(golvatinib) Woodcliff Lake, NJ www.eisai.com
ERC1671 Epitopoietic Research grade IV glioblastoma Phase II
(therapeutic vaccine) Pasadena, CA www.ercbelgium.com
Orphan Drug University of California
Irvine, CA
ETS2101 e-Therapeutics glioma Phase I
(dexanabinol) Oxfordhsire, United Kingdom www.etherapeutics.co.ukMoores UCSD Cancer Center
La Jolla, CA
galunisertib Eli Lilly glioblastoma Phase II(TGF-beta 1 kinase inhibitor) Indianapolis, IN www.lilly.com
glioma Phase I/II
www.lilly.com
Gilotrif® Boehringer Ingelheim Pharmaceuticals recurrent glioma Phase II
afatinib Ridgefield, CT www.boehringer-ingelheim.com
heat shock protein vaccine Agenus recurrent or newly diagnosed Phase II
(Prophage Series G-200) Lexington, MA glioblastoma www.agenus.com
Orphan Drug
Brain Tumors
Product Name Sponsor Indication Development Phase
ICT-107 ImmunoCellular Therapeutics newly diagnosed glioblastoma Phase II(dendritic cell-based vaccine) Calabasas, CA www.imuc.com
Orphan Drug
ICT-121 ImmunoCellular Therapeutics recurrent glioblastoma Phase I(dendritic cell-based vaccine) Calabasas, CA www.imuc.com
indoximod NewLink Genetics primary malignant brain tumors Phase I/II
(IDO inhibitor) Ames, IA www.newlinkgenetics.com
INXN-2001 ZIOPHARM Oncology glioblastoma, anaplastic Phase I/II
(Ad-RTS-IL-12 gene therapy) Boston, MA oligoastrocytoma www.ziopharm.com
Jevtana® Sanofi US malignant central nervous system Phase I/II
cabazitaxel Bridgewater, NJ tumors www.sanofi.com
KX02 Kinex Pharmaceuticals glioma Phase I(Src kinase/pre-tubulin inhibitor) Buffalo, NY www.kinexpharma.com
Orphan Drug
LDE225 Novartis Pharmaceuticals medulloblastoma Phase II(SMO protein inhibitor) East Hanover, NJ www.novartis.com
macitentan Actelion Pharmaceuticals glioblastoma Phase I
(endothelin A/B receptor antagonist) South San Francisco, CA www.actelion.com
Brain Tumors
Product Name Sponsor Indication Development Phase
marizomib Triphase Accelerator glioblastoma Phase I
(proteasome inhibitor) San Diego, CA www.triphaseco.com
mibefradil Cavion high-grade glioma Phase I(T-type calcium channel antagonist) Charlottesville, VA www.cavionpharma.com
Orphan Drug
mipsagargin GenSpera glioblastoma Phase II(thapsigargin analogue) San Antonio, TX www.genspera.com
MM-398 Merrimack Pharmaceuticals glioma Phase I(encapsulated irinotecan Cambridge, MA www.merrimackpharma.com
nanotherapeutic)
NEO-100 NEONC Technologies recurrent glioblastoma Phase I
(highly purified perillyl alcohol) Los Angeles, CA www.neonctech.com
Orphan Drug
NVX-108 NuvOx Pharma glioblastoma Phase I/II(radiation-sensitizing agent) Tucson, AZ www.nuvoxpharma.com
National Cancer Institute
Bethesda, MD
Opdivo® Bristol-Myers Squibb glioblastoma Phase III
nivolumab Princeton, NJ www.bms.com
PEPIDH1M Annias Immunotherapeutics glioblastoma Phase I/II(dendritic cell vaccine) Chapel Hill, NC www.anniasimmuno.com
Brain Tumors
Product Name Sponsor Indication Development Phase
PLX-3397 Plexxikon glioblastoma Phase II(CSF-1R inhibitor) Berkeley, CA www.plexxikon.com
Poly-ICLC Oncovir glioblastoma Phase II
Orphan Drug Washington, DC www.oncovir.com
Reolysin® Oncolytics Biotech high grade glioma (pediatrics) Phase I
pelareorep Calgary, Canada www.oncolyticsbiotech.com
Orphan Drug
rindopepimut Celldex Therapeutics glioblastoma (1st-line) (Fast Track) Phase III(EGFR antagonist) Hampton, NJ (Fast Track) www.celldex.com
Orphan Drug
recurrent glioblastoma Phase II
(Fast Track) www.celldex.com
Seprehvir® Virttu Biologics glioblastoma Phase I completed
HSV1716 Glasgow, United Kingdom www.virttu.comNational Cancer Institute
Bethesda, MD
selinexor (oral) Karyopharm Therapeutics glioblastoma Phase II
(exportin-1 protein inhibitor/ Newton, MA www.karyopharm.com
nuclear protein inhibitor)
Brain Tumors
Product Name Sponsor Indication Development Phase
SL-701 Stemline Therapeutics glioblastoma, glioma Phase I/II
(immunotherapy) New York, NY www.stemline.com
Orphan Drug
SurVaxM MimiVax malignant glioma Phase I(BIRC5 protein inhibitor) Buffalo, NY www.mimivax.com
TH-302 Threshold Pharmaceuticals high grade glioma Phase I/II
(evofosfamide) South San Francisco, CA www.thresholdpharm.com
University of Texas
San Antonio, TX
Toca 511 + Toca FC Tocagen anaplastic astrocytoma, Phase II/III
(gene therapy) San Diego, CA glioblastoma www.tocagen.com
TPI 287 Cortice Biosciences glioblastoma Phase II
(abeotaxane) New York, NY (see also Alzheimer's, other) www.corticebio.com
TRC105 TRACON Pharmaceuticals glioblastoma Phase II
(ENG protein inhibitor) San Diego, CA www.traconpharma.com
National Cancer Institute
Bethesda, MD
TSC Diffusion Pharmaceuticals glioblastoma Phase I/II
(trans-sodium crocetinate) Charlottesville, VA www.diffusionpharma.com
Orphan Drug
Brain Tumors
Product Name Sponsor Indication Development Phase
TVI-Brain-1 TVAX Biomedical glioma Phase II completed
(immunotherapy) Lenexa, KS www.tvaxbiomedical.com
Orphan Drug
VAL-083 DelMar Pharmaceuticals glioblastoma Phase I/II(N7-alkylating agent) Vancouver, Canada www.delmarpharma.com
Orphan Drug
VB 111 VBL Therapeutics recurrent glioblastoma (Fast Track) Phase II(targeted anti-angiogenic agent) Tel Aviv, Israel www.vblrx.com
Orphan Drug
veliparib AbbVie brain metastasis Phase II
North Chicago, IL www.abbvie.com
Epilepsy
Product Name Sponsor Indication Development Phase
Afinitor® Novartis Pharmaceuticals tuberous sclerosis complex seizures Phase III
everolimus East Hanover, NJ www.novartis.com
Aptiom® Sunovion Pharmaceuticals epilepsy in adults (monotherapy) application submitted
eslicarbazepine acetate Marlborough, MA www.sunovion.com
AZ-002 Alexza Pharmaceuticals acute repetitive seizures Phase II
(inhalation alprazolam) Mountain View, CA www.alexza.com
Epilepsy
Product Name Sponsor Indication Development Phase
brivaracetam UCB epilepsy (adjunctive treatment) application submittedSmyrna, GA www.ucb.com
Carbella™ Lundbeck epilepsy application submitted
carbamazepine intravenous Deerfield, IL www.lundbeck.com
Orphan Drug
cannabidiol oral solution INSYS Therapeutics treatment resistant seizures Phase I/II
Chandler, AZ www.insysrx.com
CPP-115 Catalyst Pharmaceuticals complex partial seizures, Phase I(4-aminobutyrate transaminase inhibitor) Coral Gables, FL infantile spasms www.catalystpharma.com
Orphan Drug (see also Tourette's)
Epidiolex® GW Pharmaceuticals Dravet syndrome (Fast Track) Phase III
cannabidiol Cambridge, United Kingdom (pediatric) www.gwpharm.com
Orphan Drug
Lennox-Gastaut syndrome Phase II
www.gwpharm.com
Fycompa® Eisai primary generalized tonic-clonic application submittedperampanel Woodcliff Lake, NJ seizures www.eisai.com
partial-onset seizures (pediatric) Phase II
www.eisai.com
Epilepsy
Product Name Sponsor Indication Development Phase
ganaxolone Marinus Pharmaceuticals focal onset seizures (adults) Phase III(GABA-A receptor agonist) Radnor, PA www.marinuspharma.com
Orphan Drug
PCDH19 female epilepsy (pediatric) Phase II
www.marinuspharma.com
INS-001 Insero Health epilepsy Phase I completed
(synthetic huperzine) Miami Beach, FL www.insero.co
IPX218 Impax Laboratories epilepsy Phase II completed
Hayward, CA www.impaxlabs.com
Lyrica® Pfizer generalized tonic-clonic seizures, Phase III
pregabalin New York, NY partial onset seizures (pediatric) www.pfizer.com
(see also pain)
MP-102 Marathon Pharmaceuticals infantile spasms Phase III
Northbrook, IL www.marathonpharma.com
Onfi® Lundbeck Dravet syndrome (pediatric) Phase III
clobazam Deerfield, IL www.lundbeck.com
Plumiaz™ Acorda Therapeutics cluster seizures Phase III
diazepam nasal spray Ardsley, NY www.acorda.com
Orphan Drug
Epilepsy
Product Name Sponsor Indication Development Phase
SAGE-547 SAGE Therapeutics super refractory status epilepticus Phase III
(GABAA synaptic and extra-synaptic Cambridge, MA (Fast Track) www.sagerx.com
receptor modulator)
Orphan Drug
essential tremors Phase II
www.sagerx.com
topiramate IV CURx Pharmaceuticals epilepsy in patients unable to take Phase I completed
Orphan Drug San Diego, CA oral therapy www.curxpharma.com
USL261 Upsher-Smith Laboratories seizure clusters (Fast Track) Phase III
(intransal midazolam) Maple Grove, MN www.upsher-smith.com
Orphan Drug
Vimpat® UCB partial onset seizures (pediatric) Phase IIIlacosamide Smyrna, GA www.ucb.com
primary generalized tonic clonic Phase II
seizures www.ucb.com
YKP3089 SK Life Science partial onset seizures Phase II
Fair Lawn, NJ www.skbp.com
ZX008 Zogenix Dravet syndrome (pediatric) Phase II completed(serotonin receptor agonist) San Diego, CA www.skbp.com
Orphan Drug
Genetic Disorders
Product Name Sponsor Indication Development Phase
ABT-RTA 408 AbbVie Friedrich's ataxia Phase II(NF/E2 related factor 2 stimulant) North Chicago, IL (see also other) www.abbvie.com
Reata Pharmaceuticals www.reatapharma.com
Irving, TX
Actimmune® Horizon Pharma Friedreich's ataxia (Fast Track) Phase II
interferon gamma-1b Deerfield, IL www.horizonpharma.com
Orphan Drug
BMN 190 BioMarin Pharmaceutical late infantile neuronal ceroid Phase I/II(rhTPP1 enzyme replacement therapy) San Rafael, CA lipofuscinosis type 2 (CLN2) disease www.bmrn.com
Orphan Drug
BVS857 Novartis Pharmaceuticals spinal and bulbar muscular atrophy Phase II
East Hanover, NJ www.novartis.com
EPI-743 Edison Pharmaceuticals Friedrich's ataxia (Fast Track), Phase II
(vatiquinone) Mountain View, CA Leigh syndrome, Rett syndrome www.edisonpharma.com
Orphan Drug
EryDex EryDel ataxia telangiectasia Phase I
dexamethasone erythrocyte Urbino, Italy (see also muscular dystrophy) www.erydel.com
(encapsulated)
Ferriprox® ApoPharma pantothenate kinase-associated Phase III
deferiprone Toronto, Canada neurodegeneration www.apopharma.com
Genetic Disorders
Product Name Sponsor Indication Development Phase
ISIS-SMNRX Biogen spinal muscular atrophy (Fast Track) Phase III
(antisense) Cambridge, MA (pediatric) www.biogen.com
Orphan Drug Isis Pharmaceuticals www.isispharm.com
Carlsbad, CA
ISIS-TTRRX GlaxoSmithKline familial amyloid polyneuropathy Phase III
(antisense) Research Triangle Park, NC (Fast Track) www.gsk.com
Orphan Drug Isis Pharmaceuticals www.isispharm.com
Carlsbad, CA
Lenti-D™ Bluebird Bio cerebral adrenoleucodystrophy Phase II/III
adrenoleucodystrophy gene therapy Cambridge, MA (pediatric) www.bluebirdbio.com
Orphan Drug
NLX-101 Neurolixis Rett syndrome Phase I
(serotonin 1A receptor agonist) Dana Point, CA www.neurolixis.com
Orphan Drug
NNZ-2256 Neuren Pharmaceuticals Rett syndrome (Fast Track) Phase II
(cytokine inhibitors/neuropeptide Bethesda, MD (see also brain injury) www.neurenpharma.com
receptor modulator)
Orphan Drug
patisiran (ALN-TTRO2) Alnylam Pharmaceuticals familial amyloid polyneuropathy Phase III
(siRNA inhibitor targeting TTR) Cambridge, MA (Fast Track) www.alnylam.com
Genzyme www.genzyme.com
Cambridge, MA
Genetic Disorders
Product Name Sponsor Indication Development Phase
RE-024 Retrophin pantothenate kinase-associated Phase III
(phosphopantothenate replacement) San Diego, CA neurodegeneration (Fast Track) www.retrophin.com
Orphan Drug
RG3039 Repligen spinal muscular atrophy (Fast Track) Phase I(DcpS inhibitor) Waltham, MA www.repligen.com
Orphan Drug
RG7800 PTC Therapeutics spinal muscular atrophy Phase I(splicing of the SMN2 gene modifier) South Plainfield, NJ www.ptcbio.com
Roche www.roche.com
New York, NY
RP103 Raptor Pharmaceuticals Leigh syndrome and other Phase II
(cysteamine bitartrate Novato, CA mitochondrial diseases www.raptorpharma.com
delayed release) (see also Huntington's)
RT001 Retrotope Friedreich's ataxia Phase I/II
(fatty acid replacement) Los Altos, CA www.retrotope.com
SHP622 Shire ViroPharma Friedreich's ataxia Phase I
(indolepropionic acid) Exton, PA www.shire.com
tafamidis meglumine Pfizer transthyretin familial amyloid application submitted
(transthyretin dissociation inhibitor) New York, NY polyneuropathy (Fast Track) www.pfizer.com
Orphan Drug
Genetic Disorders
Product Name Sponsor Indication Development Phase
WTX101 Wilson Therapeutics Wilson's disease Phase II(bis-choline tetrathiomolybdate) Burlington, MA (hepatolenticular degeneration) www.wilsontherapeutics.com
Orphan Drug
Headache
Product Name Sponsor Indication Development Phase
ALD403 Alder Biopharmaceuticals migraine prevention Phase II
(anti-CGRP mAb) Bothell, WA www.alderbio.com
AMG 301 Amgen migraine Phase I(mAb) Thousand Oaks, CA www.amgen.com
AMG 334 Amgen migraine prevention Phase II(CGRP receptor antagonist mAb) Thousand Oaks, CA www.amgen.com
AVP-825 Avanir Pharmaceuticals migraine application submitted
(sumatriptan intranasal) Aliso Viejo, CA www.avanir.com
AVP-923 Avanir Pharmaceuticals migraine Phase II
(dextromethorphan/quinidine Aliso Viejo, CA (see also Alzheimer's, Parkinson's) www.avanir.com
fixed-dose combination)
Botox® Allergan chronic migraine prevention Phase IIIonabotulinumtoxinA Fort Worth, TX (adolescents) www.allergan.com
(see also pain, spasticity)
Headache
Product Name Sponsor Indication Development Phase
DFN-02 Dr. Reddy's Laboratories acute migraine Phase IIITelangana, India www.drreddys.com
DFN-11 Promius Pharma acute migraine application submitted
(drug-device combination) Princeton, NJ www.promiuspharma.com
ELS-M11 Achelios Therapeutics migraine Phase I/II
(topical treatment) Chapel Hill, NC www.achelios.com
IPX232 Impax Laboratories migraine Phase I completed
Hayward, CA www.impaxlabs.com
lasmiditan CoLucid Pharmaceuticals migraine Phase III
(serotonin-1F receptor agonist) Cambridge, MA www.colucid.com
LY2951742 Eli Lilly chronic cluster headache Phase III
(CGRP mAb) Indianapolis, IN (see also pain) www.lilly.com
migraine Phase II
www.lilly.com
NXN-188 NeurAxon acute migraine Phase II
(nNOS inhibitor/5HT) Mississauga, Canada www.neuraxon.com
Headache
Product Name Sponsor Indication Development Phase
Rizaport™ IntelGenx migraine application submitted
rizatriptan oral film Saint Laurent, Canada www.intelgenx.com
RedHill Biopharma www.redhillbio.com
Tel Aviv, Israel
Semprana™ Allergan acute migraine (adults) application submitteddihydroergotamine Ft. Worth, TX www.allergan.com
SUD-001 SUDA migraine Phase II completed
(sumatriptan oral spray) Osborne Park, Australia www.sudaltd.com.au
TEV-48125 Teva Pharmaceuticals chronic and episodic migraine Phase II
(CGRP receptor antagonist) North Wales, PA www.tevapharm.com
TI-001 Trigemina high frequency migraine Phase II
(oxytocin intranasal) Moraga, CA www.trigemina.com
TNX-201 Tonix Pharmaceuticals episodic tension-type headache Phase I
(cyclobenzaprine very low dose) New York, NY www.tonixpharma.com
VVD-101 Vivid Pharma delayed alcohol-induced Phase II
(aspirin/sumatriptan Springfield, MO headache
fixed-dose combination)
Huntington's Disease
Product Name Sponsor Indication Development Phase
laquinimod Teva Pharmaceuticals Huntington's disease Phase II
North Wales, PA (see also multiple sclerosis) www.tevapharm.com
MP-112 Marathon Pharmaceuticals Huntington's disease Phase III
Northbrook, IL www.marathonpharma.com
OMS824 Omeros Huntington's disease (Fast Track) Phase II
(PDE10 inhibitor) Seattle, WA www.omeros.com
Orphan Drug
PBT2 Prana Biotechnology Huntington's disease Phase II completed(metal protein-attenuating compound) Parkville, Australia www.pranabio.com
Orphan Drug
PF-02545920 Pfizer Huntington's disease Phase II
(PDE10 inhibitor) New York, NY www.pfizer.com
Orphan Drug
pridopidine Teva Pharmaceuticals Huntington's disease Phase II/III
Orphan Drug North Wales, PA www.tevapharm.com
RP103 Raptor Pharmaceuticals Huntington's disease Phase III
(cysteamine bitartrate Novato, CA (see also genetic) www.raptorpharma.com
delayed release
Orphan Drug
Huntington's Disease
Product Name Sponsor Indication Development Phase
SD-809 Auspex Pharmaceuticals chorea associated with Phase III
(VMAT2 inhibitor) La Jolla, CA Huntington's disease www.auspexpharma.com
Orphan Drug (see also Tourette's, other)
Multiple Sclerosis
Product Name Sponsor Indication Development Phase
adipose-derived mesenchymal American CryoStem multiple sclerosis Phase I
stem cell therapy Eatontown, NJ www.americancryostem.com
ALKS 8700 Alkermes multiple sclerosis Phase I(monomethyl fumarate prodrug) Waltham, MA www.alkermes.com
anti-LINGO-1 (BIIB033) Biogen multiple sclerosis Phase II
Cambridge, MA www.biogen.com
ATX-MS-1467 EMD Serono multiple sclerosis Phase I
(immune tolerizing agent) Rockland, MA www.emdserono.com
AX-024 Artax Biopharma multiple sclerosis Phase I
(Nck protein modulator) Cambridge, MA www.artaxbiopharma.com
BAF312 Novartis Pharmaceuticals secondary-progressive multiple Phase III(S1PR-1/5 receptor agonist) East Hanover, NJ sclerosis www.novartis.com
Multiple Sclerosis
Product Name Sponsor Indication Development Phase
BIIB061 Biogen multiple sclerosis Phase I
(oral re-myelination) Cambridge, MA www.biogen.com
daclizumab high-yield process AbbVie relapsing-remitting multiple sclerosis application submitted(human IgG1 mAb) North Chicago, IL www.abbvie.com
Biogen www.biogen.com
Cambridge, MA
Gilenya® Novartis Pharmaceuticals multiple sclerosis (pediatric) Phase III
fingolimod East Hanover, NJ (see also other) www.novartis.com
GZ402668 Genzyme relapsing multiple sclerosis Phase I
(anti-CD52 mAb) Cambridge, MA www.genzyme.com
idebenone Santhera Pharmaceuticals primary-progressive multiple sclerosis Phase II
Liestal, Switzerland (see also muscular dystrophy) www.santhera.com
National Institute of Neurological
Disorders and Stroke
Bethesda, MD
INT131 InteKrin Therapeutics multiple sclerosis Phase II
(PPAR-gamma agonist/modulator) Redwood City, CA www.intekrin.com
Multiple Sclerosis
Product Name Sponsor Indication Development Phase
laquinimod Teva Pharmaceuticals relapsing-remitting multiple sclerosis Phase III
North Wales, PA (Fast Track) www.tevapharm.com
(see also Huntington's)
progressive multiple sclerosis Phase II
(Fast Track) www.tevapharm.com
masitinib AB Science USA progressive multiple sclerosis Phase II/III(tyrosine kinase inhibitor) Chatham, NJ www.ab-science.com
MEDI-551 MedImmune multiple sclerosis Phase I(anti-CD19 mAb) Gaithersburg, MD (see also other) www.medimmune.com
MN-166 MediciNova progressive multiple sclerosis Phase II
(ibudilast) La Jolla, CA (see also ALS) www.medicinova.com
MRF-008 Myelin Repair Foundation relapsing-remitting multiple sclerosis Phase I(guanabenz) Saratoga, CA www.myelinrepair.org
National Institute of Neurological
Disorders and Stroke
Bethesda, MD
NeuroVax™ Immune Response BioPharma secondary-progressive multiple Phase II
immunostimulant vaccine Atlantic City, NJ sclerosis (Fast Track) www.immuneresponsebiopharma.com
Orphan Drug
Multiple Sclerosis
Product Name Sponsor Indication Development Phase
ocrelizumab Genentech primary-progressive multiple sclerosis, Phase III(anti-CD20 mAb) South San Francisco, CA relapsing multiple sclerosis www.gene.com
ofatumumab GlaxoSmithKline multiple sclerosis Phase II
(CD20 mAb) Research Triangle Park, NC (see also other) www.gsk.com
PF530 Pfenex multiple sclerosis Phase I
(interferon beta-1b biosimilar) San Diego, CA www.pfenex.com
PF-06342674 Pfizer multiple sclerosis Phase I(IL-7 receptor inhibitor) New York, NY www.pfizer.com
ponesimod Actelion Pharmaceuticals relapsing multiple sclerosis Phase II
(S1P1 receptor agonist) South San Francisco, CA www.actelion.com
rHIgM22 Acorda Therapeutics multiple sclerosis Phase I(myelin protein stimulant) Ardsley, NY www.acorda.com
RPC1063 Receptos relapsing multiple sclerosis Phase III(S1P1 agonist) San Diego, CA www.receptos.com
RNS60 Revalesio relapsing-remitting multiple sclerosis Phase II(charge-stabilized nanostructure Tacoma, WA www.revalesio.com
saline product)
Multiple Sclerosis
Product Name Sponsor Indication Development Phase
Tcelna® Opexa Therapeutics secondary-progressive multiple Phase II
imilecleucel-T The Woodlands, TX sclerosis (Fast Track) www.opexatherapeutics.com
Trimesta™ Synthetic Biologics relapsing-remitting multiple sclerosis Phase II
estriol succinate Rockville, MD www.syntheticbiologics.com
Tysarbi® Biogen secondary-progressive multiple Phase III
natalizumab Cambridge, MA sclerosis www.biogen.com
(see also stroke)
vatelizumab Genzyme multiple sclerosis Phase II
(anti-VLA 2 mAb) Cambridge, MA www.genzyme.com
VAY736 Novartis Pharmaceuticals relapsing-remitting multiple sclerosis Phase II(BAFF-R modulator) East Hanover, NJ www.novartis.com
VX15 Vaccinex multiple sclerosis Phase I(CD100 antigen inhibitor) Rochester, NY www.vaccinex.com
XP23829 XenoPort relapsing multiple sclerosis Phase I(NF-kappa B inhibitor) Santa Clara, CA www.xenoport.com
Muscular Dystrophy
Product Name Sponsor Indication Development Phase
AAV1-FS344 Milo Biotechnology Becker muscular dystrophy Phase II/III(gene therapy-delivered Cleveland, OH (see also other) www.milobiotechnology.com
myostatin inhibitor)
Orphan Drug
Duchenne muscular dystrophy Phase I/II
www.milobiotechnology.com
ACE-083 Acceleron Pharma muscular dystrophy Phase I
(recombinant fusion protein) Cambridge, MA (see also other) www.acceleronpharma.com
ATYR1940 aTyr Pharma facioscapulohumeral muscular Phase I/II(histidine-tRNA ligase modulator) San Diego, CA dystrophy www.atyrpharma.com
Orphan Drug
BMN 044 BioMarin Pharmaceutical Duchenne muscular dystrophy Phase II
(antisense RNA modulator) San Rafael, CA (EXON 44) www.bmrn.com
Orphan Drug
BMN 045 BioMarin Pharmaceutical Duchenne muscular dystrophy Phase II
(antisense RNA modulator) San Rafael, CA (EXON 45) www.bmrn.com
Orphan Drug
Carmeseal MD™ Phrixus Pharmaceuticals Duchenne muscular dystrophy Phase II
P-188 NF Ann Arbor, MI www.phrixuspharmaceuticals.com
Orphan Drug
Muscular Dystrophy
Product Name Sponsor Indication Development Phase
drisapersen BioMarin Pharmaceutical Duchenne muscular dystrophy application submitted
(RNA interference) San Rafael, CA (EXON 51) (Fast Track) www.bmrn.com
Orphan Drug
EryDex EryDel Duchenne muscular dystrophy Phase I
dexamethasone erythrocyte Urbino, Italy (see also genetic) www.erydel.com
(encapsulated)
eteplirsen Sarepta Therapeutics Duchenne muscular dystrophy Phase III
Orphan Drug Cambridge, MA (EXON 51) (Fast Track) www.sarepta.com
HT-100 Akashi Therapeutics Duchenne muscular dystrophy Phase II
(halofuginone) Cambridge, MA (Fast Track) www.akashirx.com
Orphan Drug
idebenone Santhera Pharmaceuticals Duchenne muscular dystrophy Phase III
Orphan Drug Liestal, Switzerland (Fast Track) www.santhera.com
(see also multiple sclerosis)
ISIS-DMPKRX Biogen myotonic dystrophy, type 1 Phase I
(antisense) Cambridge, MA www.biogen.com
Orphan Drug Isis Pharmaceuticals www.isispharm.com
Carlsbad, CA
MP-104 Marathon Pharmaceuticals Duchenne muscular dystrophy Phase III(steroid receptor agonist) Northbrook, IL (pediatric) (Fast Track) www.marathonpharmaceuticals.com
Orphan Drug
Muscular Dystrophy
Product Name Sponsor Indication Development Phase
omigapil Santhera Pharmaceuticals congenital muscular dystrophy Phase I(GAPDH inhibitor) Liestal, Switzerland (pediatric) www.santhera.com
Orphan Drug
PF-06252616 Pfizer Duchenne muscular dystrophy Phase II
(myostatin inhibitor) New York, NY (Fast Track) www.pfizer.com
Orphan Drug
SRP-4053 Sarepta Therapeutics Duchenne muscular dystrophy Phase I/II
(genetic transcription inhibitor) Cambridge, MA (EXON 53) (pediatric) www.sarepta.com
tadalafil Eli Lilly Duchenne muscular dystrophy Phase III
Indianapolis, IN (pediatric) www.lilly.com
Translarna™ PTC Therapeutics Duchenne muscular dystrophy application submitted
ataluren South Plainfield, NJ (ages 5 years and older) www.ptcbio.com
Orphan Drug (Fast Track)
VBP15 ReveraGen BioPharma Duchenne muscular dystrophy Phase I(glucocorticoid receptor agonist) Silver Spring, MD www.reveragen.com
Pain
Product Name Sponsor Indication Development Phase
AB-001 Frontier Biotechnologies chronic low back pain Phase II
(topical patch) Nanjing, China www.frontierbiotech.com
Pain
Product Name Sponsor Indication Development Phase
AF 219 Afferent Pharmaceuticals osteoarthritis pain Phase II completed
(P2X3 purinoreceptor antagonist) San Mateo, CA www.afferentpharma.com
AGN-214868 Allergan postherpetic neuralgia Phase II
(SNARE protein antagonist) Fort Worth, TX www.allergan.com
ALO-02 Pfizer severe pain application submitted
(oxycodone-naltrexone core) New York, NY www.pfizer.com
(abuse resistant)
Amiket™ Immune Pharmaceuticals neuropathic pain (Fast Track) Phase II
amitriptyline/ketamine New York, NY www.immunepharmaceuticals.com
Ampligen® Hemispherx Biopharma chronic fatigue syndrome application submitted
rintatolimod Philadelphia, PA www.hemisphrx.net
Orphan Drug
APD371 Arena Pharmaceuticals chronic pain Phase I(cannabinoid receptor CB2 agonist) San Diego, CA www.arenapharm.com
ARA 290 Araim Pharmaceuticals neuropathic pain in sarcoidosis Phase II
(non-eryhropoietic peptide) Tarrytown, NY (Fast Track) www.araimpharma.com
Orphan Drug
ATx08-001 Aestus Therapeutics postherpetic neuralgia Phase II completed
(PPAR-gamma agonist) East Windsor, NJ www.aestustherapeutics.com
Pain
Product Name Sponsor Indication Development Phase
ATx09-002 Aestus Therapeutics lower back pain Phase I completed
(glycogen phosphorylase inhibitor) East Windsor, NJ www.aestustherapeutics.com
AV-101 VistaGen Therapeutics neuropathic pain Phase I completed(NMDA receptor antagonist) South San Francisco, CA www.vistagen.com
AXS-02 Axsome Therapeutics complex regional pain syndrome in clinical trials
(zoledronic acid) New York, NY (Fast Track) www.axsome.com
Orphan Drug
AYX1 Adynxx prevention of the transition to Phase II(early growth response protein 1 San Francisco, CA persistant or chronic pain www.adynxx.com
inhibitor) (Fast Track)
AZD5213 AstraZeneca neuropathic pain Phase II
(histamine-3 receptor antagonist) Wilmington, DE (see also Tourette's) www.astrazeneca.com
Belbuca™ BioDelivery Sciences moderate to severe chronic pain application submitted
buprenorphine buccal film Raleigh, NC www.bdsi.com
Endo Pharmaceuticals www.endo.com
Malvern, PA
BI 1026706 Boehringer Ingelheim osteoarthritis pain Phase I
(oral analgesic) Pharmaceuticals www.boehringer-ingelheim.com
Ridgefield, CT
Botox® Allergan osteoarthritis pain Phase IIonabotulinumtoxinA Fort Worth, TX (see also headache, spasticity) www.allergan.com
Pain
Product Name Sponsor Indication Development Phase
BuTab ER Relmada Therapeutics chronic pain Phase Ienteric-coated buprenorphine New York, NY www.relamada.com
Butrans® Transdermal System CIII Purdue Pharma severe chronic pain Phase III
buprenorphine transdermal system Stamford, CT (ages 7 - 16 years) www.purduepharma.com
C746 Cosmederm Bioscience postherpetic neuralgia Phase I
(cytokine inhibitor) San Diego, CA www.cosmederm.com
cebranopadol Actavis chronic nociceptive pain, Phase IIParsippany, NJ neuropathic pain www.actavis.comGrünenthal www.grunenthal.com
Aachen, Germany
Celebrex® Pfizer chronic pain application submitted
celecoxib New York, NY www.pfzier.com
CEP-33237 Teva Pharmaceuticals chronic pain application submitted
(hydrocodone extended release) North Wales, PA www.tevapharm.com
(abuse resistant)
clonidine topical gel BioDelivery Sciences International painful diabetic neuropathy Phase III
Raleigh, NC (Fast Track) www.bdsi.com
CMX-020 oral Cmxtwenty neuropathic pain Phase I(arachidonic acid analog) Milwaukee, WI www.cmxtwenty.com
Pain
Product Name Sponsor Indication Development Phase
CNTX-4975 Centrexion painful intermetatarsal neuroma Phase II(synthetic capsaicin) Baltimore, MD (Morton's neuroma) www.centrexion.com
CNV1014802 Biogen trigeminal neuralgia Phase II
(Nav1.7 sodium channel inhibitor) Cambridge, MA www.biogen.com
Orphan Drug
COL-172 Collegium Pharmaceutical pain (Fast Track) Phase I
(oxymorphone extended release) Canton, MA www.collegiumpharma.com
(abuse resistant)
CR845 Cara Therapeutics chronic pain Phase II
(opioid kappa receptor agonist) Shelton, CT www.caratherapeutics.com
Cymbalta® Eli Lilly fibromyalgia (adolescents) Phase III
duloxetine Indianapolis, IN www.lilly.com
DA-9801 Dong-A ST painful diabetic neuropathy Phase II completed(nerve growth factor stimulant) Seoul, South Korea www.en.donga-st.com
d-methadone Relmada Therapeutics painful diabetic neuropathy Phase I/II
(REL-1017) New York, NY www.relmada.com
Dex-SL (sublingual) RecroPharma chronic pain Phase II(dexmedetomidine) Malvern, PA www.recropharma.com
Pain
Product Name Sponsor Indication Development Phase
DS-1971 Daiichi Sankyo chronic pain Phase I
Parsippany, NJ www.dsi.com
DSP-2230 Sunovion Pharmaceuticals neuropathic pain Phase I(voltage-gated sodium channel Marlborough, MA www.sunovion.com
inhibitor)
Egalet-001 Egalet chronic lower back pain Phase III(morphine extended release) Malvern, PA (Fast Track) www.egalet.com(abuse resistant)
Egalet-002 Egalet chronic pain Phase III(oxycodone extended release) Malvern, PA www.egalet.com(abuse resistant)
Eladur® Durect postherpetic neuralgia Phase II
bupivacaine transdermal Cupertino, CA www.durect.com
Orphan Drug Impax Laboratories www.impaxlabs.com
Hayward, CA
ELI-200 Elite Pharmaceuticals chronic pain Phase I
(opioid/naltrexone) Northvale, NJ www.elitepharma.com
(abuse resistant)
ELI-201 Elite Pharmaceuticals chronic pain Phase I
(oxycodone/naltrexone) Northvale, NJ www.elitepharma.com
(abuse resistant)
Pain
Product Name Sponsor Indication Development Phase
ELI-202 Elite Pharmaceuticals chronic pain Phase I
(opioid/naltrexone) Northvale, NJ www.elitepharma.com
(abuse resistant)
EMA401 Spinifex Pharmaceuticals postherpetic neuralgia Phase I(angiotensin type 2 receptor Preston, Australia www.spinifexpharma.com
antagonist)
fulranumab Janssen Research & Development osteoarthritis pain Phase III
(nerve growth factor inhibitor) Raritan, NJ www.janssenrnd.com
FX005 Flexion Therapeutics end-stage osteoarthritis pain Phase II
(sustained release p38 inhibitor) Burlington, MA www.flexiontherapeutis.com
FX006 Flexion Therapeutics moderate to severe osteoarthritis Phase III
(sustained release steroid) Burlington, MA pain www.flexiontherapeutis.com
GDC-0276 Genentech chronic pain Phase I(Nav1.7 inhibitor) South San Francisco, CA www.gene.com
Xenon Pharmaceuticals www.xenon-pharma.com
Burnaby, Canada
GLYX-13 Naurex neuropathic pain Phase II(rapastinel) Evanston, IL www.naurex.com
HLT Patch Nuvo Research musculoskeletal pain Phase II(lidocaine/tetracaine topical patch) Ontario, Canada www.nuvoresearch.com
Pain
Product Name Sponsor Indication Development Phase
hydromorphone Mallinckrodt Pharmaceuticals chronic pain Phase III
(intrathecal administration) St. Louis, MO www.mallinckrodt.com
IMC-1 Innovative Med Concepts fibromyalgia Phase II completed
(celecoxib/famciclovir combination) Tuscaloosa, AL www.innovativemedconcepts.com
KIT-301 Kitov Pharmaceuticals osteoarthritis pain Phase II completed
(naproxen/antihypertensive Washington, DC www.kitovpharma.com
combination)
KRN5500 DARA BioSciences painful chronic chemotherapy- Phase II
(protein synthesis inhibitor) Raleigh, NC induced peripheral neuropathy www.darabio.com
Orphan Drug (Fast Track)
LevoCap ER Relmada Therapeutics chronic pain Phase II
levorphanol New York, NY www.relamada.com
LY2951742 Eli Lilly osteoarthritis pain Phase II
(CGRP mAb) Indianapolis, IN (see also headache) www.lilly.com
Lyrica® Pfizer peripheral neuropathic pain Phase III
pregabalin New York, NY (see also epilepsy) www.pfizer.com
Lyrica® CR Pfizer fibromyalgia, postherpetic Phase III
pregabalin controlled release New York, NY neuralgia www.pfizer.com
Pain
Product Name Sponsor Indication Development Phase
MDT-15 pellets Medtronic Spinal and Biologics pain associated with lumbosacral Phase II
Minneapolis, MN radiculopathy and sciatica www.medtronic.com
mGlu2/3 receptor agonist Eli Lilly chronic pain Phase I
Indianapolis, IN www.lilly.com
mirogabalin Daiichi Sankyo fibromyalgia Phase III
(CACNA2D1 protein modulator) Parsippany, NJ www.daiichisankyo.com
neridronic acid Grünenthal complex regional pain syndrome Phase II/III
(bone resorption factor inhibitor) Aachen, Germany www.grunenthal.com
neublastin (BG00010) Biogen neuropathic pain Phase II
Cambridge, MA www.biogen.com
NKTR-171 Nektar Therapeutics neuropathic pain Phase I(sodium channel antagonist) San Francisco, CA www.nektar.com
NKTR-181 Nektar Therapeutics chronic pain Phase III(mu-opioid analgesic) San Francisco, CA (Fast Track) www.nektar.com
NXN-462 NeurAxon neuropathic pain Phase I completed
(nNOS inhibitor) Mississauga, Canada www.neuraxon.com
O-desmethyltramadol Syntrix Biosystems chronic pain Phase II
Auburn, WA www.syntribio.com
Pain
Product Name Sponsor Indication Development Phase
OLT1177 Olatec Industries osteoarthritis pain Phase II
(topical gel) Rye Brook, NY
oxycodone controlled-release tablets Purdue Pharma chronic pain (adolescents) Phase III completed
(tamper resistant) Stamford, CT www.purduepharma.com
palmitoylethanolamide Prismic Pharmaceuticals severe chronic pain in clinical trials
Scottsdale, AZ www.prismicpharma.com
PF329 Signature Therapeutics chronic pain Phase I
(hydromorphone ER) Palo Alto, CA www.signaturerx.com
(abuse resistant)
PF-05089771 Pfizer chronic neuropathic pain Phase II
(Nav 1.7 modulator) New York, NY www.pfizer.com
PF-06372865 Pfizer chronic pain Phase II
(GABA A agonist) New York, NY www.pfizer.com
PTI-202 Durect moderate to severe chronic pain Phase I
(hydromorphone controlled release) Cupertino, CA www.durect.com
(abuse resistant) Pain Therapeutics www.paintrials.com
Austin, TX
Remoxy® Durect moderate to severe chronic pain Phase III
oxycodone extended release Cupertino, CA www.durect.com
Pain Therapeutics www.paintrials.com
Austin, TX
Pain
Product Name Sponsor Indication Development Phase
resiniferatoxin Sorrento Therapeutics intractable cancer pain Phase I/II(TRPV-1 receptor agonist) San Diego, CA www.sorrentotherapeutics.com
Orphan Drug
rexlemestrocel-L Mesoblast pain associated with moderate Phase II
(cell replacement) New York, NY disc degeneration www.mesoblast.com
S-718632 Egalet chronic pain Phase I(hydrocodone extended release) San Diego, CA www.egalet.com
(abuse resistant) Shionogi www.shionogi.co.jp
Otsuka, Japan
Sativex® GW Pharmaceuticals cancer pain (Fast Track) Phase III
nabiximols Cambridge, United Kingdom (see also spasticity) www.gwpharm.com
Otsuka America Pharmaceutical www.otsuka.com
Princeton, NJ
SoluMatrix® Meloxicam Iroko Pharmaceuticals osteoarthritis pain application submittedcyclo-oxygenase 2 inhibitor Philadelphia, PA www.iroko.com
SoluMatrix® Naproxen Iroko Pharmaceuticals osteoarthritis pain Phase IIcyclo-oxygenase inhibitor Philadelphia, PA www.iroko.com
SPI-205 Spectrum Pharmaceuticals chemotherapy-induced neuropathy Phase I(nerve growth factor modulator) Henderson, NV www.sppirx.com
Pain
Product Name Sponsor Indication Development Phase
tanezumab Eli Lilly osteoarthritis pain Phase III
(nerve growth factor inhibitor) Indianapolis, IN www.lilly.com
Pfizer www.pfizer.com
New York, NY
cancer pain Phase II
www.lilly.com
www.pfizer.com
TD-9855 Theravance chronic pain Phase I
(norepinephrine/serotonin South San Francisco, CA www.theravance.com
reuptake inhibitor)
tetrodotoxin (TTX) WEX Pharmaceuticals chemotherapy-induced severe Phase II
(sodium channel antagonist) Vancouver, Canada neuropathic pain www.wexpharma.com
TNX-102 SL Tonix Pharmaceuticals fibromyalgia Phase II
(cyclobenzaprine very low dose) New York, NY www.tonixpharma.com
TPH-023 Teikoku Pharma USA chronic low back pain Phase II
(flurbiprofen tape) San Jose, CA www.teikokuusa.com
TRV 734 Trevena moderate to severe chronic pain Phase I(opioid mu receptor agonist) King of Prussia, PA www.trevenainc.com
TV-45070 topical Teva Pharmaceuticals neuropathic pain, osteoarthritis pain Phase II
(sodium channel inhibitor) North Wales, PA www.tevapharm.com
Pain
Product Name Sponsor Indication Development Phase
V116517 Purdue Pharma chronic pain Phase II completed
Stamford, CT www.purduepharma.com
V158866 Vernalis neuropathic pain due to spinal Phase II(fatty acid amide hydrolase inhibitor) Berkshire, United Kingdom cord injury www.vernalis.com
VBY-129 ViroBay neuropathic pain Phase I
Menlo Park, CA www.virobayinc.com
VM202 ViroMed painful diabetic neuropathy Phase II
(modified hepatocyte growth factor Seoul, South Korea (see also ALS) www.viromed.co.kr
gene therapy)
XTAMPZA ER™ Collegium Pharmaceuticals chronic low back pain (Fast Track) application submitted
oxycodone extended release Canton, MA www.collegiumpharma.com
(abuse resistant)
Zavryl™ PLx Pharma osteoarthritis pain Phase II
ibuprofen/phosphatidylcholine Houston, TX www.plxpharma.com
ZTlido™ Scilex Pharmaceuticals postherpetic neuralgia Phase I
lidocaine transdermal patch Malvern, PA www.scilexpharma.com
Parkinson's Disease
Product Name Sponsor Indication Development Phase
AAV2-GDNF National Institute to Neurological Parkinson's disease Phase I
Disorders and Stroke www.uniqure.com
Bethesda, MD
University of California
San Francisco, CA
UniQure
Lexington, MA
ADS-5102 Adamas Pharmaceuticals levodopa-induced dyskinesia in Phase III
(amantadine ER) Emeryville, CA Parkinson's disease www.adamaspharma.com
Orphan Drug
APL-130277 Cynapsus Therapeutics Parkinson's disease Phase III
(apomorphine sublingual film) Toronto, Canada www.cynapsus.ca
Apokyn® US WorldMeds idiopathic Parkinson's disease Phase IIIapomorphine for continuous Louisville, KY www.usworldmeds.comsubcutaneous infusion
Orphan Drug
AQW051 Vanda Pharmaceuticals levodopa-induced dyskinesia in Phase II completed
(alpha7 nicotine acetylcholine Washington, DC Parkinson's disease www.vandapharmaceuticals.com
receptor partial agonist)
autologous stem cell therapy NeuroGeneration Parkinson's disease Phase I completed
Los Angeles, CA www.neurogeneration.com
Parkinson's Disease
Product Name Sponsor Indication Development Phase
AVP-923 Avanir Pharmaceuticals levodopa-induced dyskinesia in Phase II
(dextromethorphan/quinidine San Diego, CA Parkinson's disease www.avanir.com
fixed-dose combination) (see also Alzheimer's, headache)
BIIB054 Biogen Parkinson's disease Phase I
(alpha-synuclein modulator) Cambridge, MA www.biogen.com
CVT-301 Acorda Therapeutics Parkinson's disease Phase III
(levodopa inhalation) Ardsley, NY www.acorda.com
D1 potentiator Eli Lilly Parkinson's disease Phase I
Indianapolis, IN www.lilly.com
dipraglurant-IR Addex Therapeutics levodopa-induced dyskinesia in Phase II
Geneva, Switzerland Parkinson's disease www.addextherapeutics.com
eltoprazine Amarantus BioScience levodopa-induced dyskinesia in Phase II(serotonin 1A/1B partial agonist) San Francisco, CA Parkinson's disease www.amarantus.com
fipamezole Santhera Pharmaceuticals Parkinson's disease (Fast Track) Phase II
Liestal, Switzerland www.santhera.com
GM6 Genervon Biopharmaceuticals Parkinson's disease Phase II
(peptide therapeutic) Pasadena, CA (see also ALS, stroke) www.genervon.com
Parkinson's Disease
Product Name Sponsor Indication Development Phase
IPX203 Impax Pharmaceuticals Parkinson's disease Phase II(carbidopa/levodopa fixed-dose Hayward, CA www.impaxlabs.com
combination)
IPX231 Impax Pharmaceuticals Parkinson's disease Phase I completed
Hayward, CA www.impaxlabs.com
IRX4204 Io Therapeutics early Parkinson's disease Phase I completed
(retinoid X receptor agonist) Santa Ana, CA www.io-therapeutics.com
KW-6002 Kyowa Hakko Kirin America Parkinson's disease application submitted
(istradefylline) Princeton, NJ (adjunctive treatment) www.kyowa-kirin.com
LY03003 Luye Pharma early Parkinson's disease Phase I
(rotigotine controlled release) Princeton, NJ www.luye.cn/en
OS-320 Osmotica Pharmaceutical levodopa-induced dyskinesia in Phase III
(amantadine ER) Marietta, GA Parkinson's disease www.osmotica.com
P2B-001 Phase Two B Parkinson's disease Phase II/III
(pramipexole/rasagiline) Rehovot, Israel www.phase2b.com
PF-06649751 Pfizer Parkinson's disease Phase I
New York, NY www.pfizer.com
Parkinson's Disease
Product Name Sponsor Indication Development Phase
Posiphen® QR Pharma Parkinson's disease Phase II
R-phenserine Berwyn, PA (see also Alzheimer's) www.qrpharma.com
PRX002/RG7935 Prothena Biosciences Parkinson's disease Phase I(alpha-synuclein inhibitor) South San Francisco, CA www.prothena.com
Roche www.roche.com
New York, NY
rasagiline transdermal patch Teikoku Pharma USA Parkinson's disease Phase I completedSan Jose, CA www.teikokuusa.com
SYN120 Biotie Therapies Parkinson's disease dementia Phase II(serotonin 2A/6 receptor antagonist) South San Francisco, CA www.biotie.com
tozadenant Biotie Therapies Parkinson's disease Phase II/III(adenosine A2A receptor antagonist) South San Francisco, CA www.biotie.com
V 81444 Vernalis Parkinson's disease Phase I/II completed(adenosine A2A receptor antagonist) Berkshire, United Kingdom www.vernalis.com
VY-AADC01 Voyager Therapeutics Parkinson's disease Phase I
(gene therapy) Cambridge, MA www.voyagertherapeutics.com
University of California
San Francisco, CA
Parkinson's Disease
Product Name Sponsor Indication Development Phase
Xadago® Newron Pharmaceuticals early-stage Parkinson's disease application submitted
safinamide Bresso, Italy (add-on therapy to dopamine, www.newron.com
Zambon Pharma mid-to late-stage Parkinson's www.zambonpharma.com
Bresso, Italy disease (add-on therapy to levodopa)
XP21279 XenoPort Parkinson's disease Phase II(dopamine receptor agonist) Santa Clara, CA www.xenoport.com
Spasticity
Product Name Sponsor Indication Development Phase
baclofen extended release Sun Pharma Advanced Research muscle spasticity in multiple sclerosis Phase III
Mumbai, India www.sunpharma.in
Botox® Allergan lower limb spasticity (pediatric), Phase IIIonabotulinumtoxinA Fort Worth, TX upper limb spasticity (pediatric www.allergan.com
Orphan Drug and adults)
(see also headache, pain)
Dysport® Ipsen Biopharmaceuticals upper limb spasticity (adults) application submitted
abobotulinumtoxinA Basking Ridge, NJ (see also Tourette's) www.ipsenus.com
focal spasticity of the lower and Phase III
upper limbs (pediatric) www.ipsenus.com
OS-440 Osmotica Pharmaceutical muscle spasticity in multiple sclerosis Phase III(arbaclofen extended release) Marietta, GA www.osmotica.com
Spasticity
Product Name Sponsor Indication Development Phase
Sativex® GW Pharmaceuticals muscle spasticity in multiple sclerosis Phase II
nabiximols Cambridge, United Kingdom (see also pain) www.gwpharm.com
Otsuka America Pharmaceutical www.otsuka.com
Princeton, NJ
Xeomin® Merz Pharmaceuticals lower limb spasticity in patients Phase III
incobotulinumtoxinA Greensboro, NC with cerebral palsy www.merzusa.com
post-stroke spasticity Phase III
www.merzusa.com
Spinal Cord Injury
Product Name Sponsor Indication Development Phase
AC105 Acorda Therapeutics spinal cord injury (Fast Track) Phase II
(nervous system modulator) Ardsley, NY www.acorda.com
AST-OPC1 Asterias Biotherapeutics spinal cord injury Phase I/II(human embryonic stem cell (hESC) Menlo Park, CA www.asteriasbiotherapeutics.com
derived cell therapy)
BA-210 BioAxone BioSciences acute spinal cord injury (Fast Track) Phase II(Rho GTP-binding protein-inhibitor) Cambridge, MA www.bioaxonebio.com
Orphan Drug
HuCNS-SC® StemCells cervical and thoracic spinal Phase II
adult neural stem cell therapy Newark, CA cord injury www.stemcellsinc.com
Spinal Cord Injury
Product Name Sponsor Indication Development Phase
NSI-566 Neuralstem chronic spinal cord injury Phase I(spinal cord-derived neural Germantown, MD (see also ALS, stroke) www.neuralstem.comstem cell therapy)
SUN13837 Daiichi Sankyo spinal cord injury Phase II
(fibroblast growth factor receptor) Parsippany, NJ www.dsi.com
Stroke
Product Name Sponsor Indication Development Phase
3K3A-APC ZZ Biotech acute ischemic stroke Phase II(recombinant mutant form of human Houston, TX www.zzbiotech.com
wild-type activated protein C)
Brilinta® AstraZeneca reduction of vascular events in acute Phase III
ticagrelor Wilmington, DE ischemic stroke or transient www.astrazeneca.com
ischemic attack
Cirara™ Remedy Pharmaceuticals severe ischemic stroke Phase II
RP-1127 New York, NY (see also brain injury) www.remedypharmaceuticals.com
CNTO 0007 Janssen Research & Development ischemic stroke Phase I completed
(cell replacement therapy) Raritan, NJ www.janssenrnd.com
dalfampridine Acorda Therapeutics chronic post-stroke walking deficits Phase III
Ardsley, NY www.acorda.com
Stroke
Product Name Sponsor Indication Development Phase
Durlaza™ New Haven Pharmaceuticals stroke prevention application submitted
aspirin controlled release Branford, CT www.newhavenpharma.com
EG-1962 Edge Therapeutics prevention of cerebral ischemia Phase I/II
(nimodipine microparticles) Berkeley Heights, NJ following aneurysmal subarachnoid www.edgetherapeutics.com
Orphan Drug hemorrhage
GM6 Genervon Biopharmaceuticals acute ischemic stroke (Fast Track) Phase II
(peptide therapeutic) Pasadena, CA (see also ALS, Parkinson's) www.genervon.com
MP-124 Mitsubishi Tanabe Pharma acute ischemic stroke Phase I
(PARP inhibitor) Jersey City, NJ www.mt-pharma-america.com
MultiStem® Athersys ischemic stroke Phase II
allogeneic stem cell therapy Cleveland, OH www.athersys.com
NA-1 NoNO stroke Phase II
(PDZ domain inhibitor) Toronto, Canada www.nonoinc.ca
NSI-566 Neuralstem ischemic stroke Phase I/II(spinal cord-derived neural Germantown, MD (see also ALS, spinal cord injury) www.neuralstem.comstem cell therapy)
PF-05230907 Pfizer intracerebral hemorrhage Phase I
New York, NY www.pfizer.com
Stroke
Product Name Sponsor Indication Development Phase
ponezumab (PF-04360365) Pfizer cerebral amyloid angiopathy Phase II
(beta amyloid inhibitor) New York, NY www.pfizer.com
Pradaxa® Boehringer Ingelheim Pharmaceuticals stroke (secondary prevention) Phase III
dabigatran Ridgefield, CT www.boehringer-ingelheim.com
Sanguinate™ Prolong Pharmaceuticals reduction or prevention of delayed Phase IIPEGylated hemoglobin and South Plainfield, NJ cerebral ischemia following www.prlongpharma.com
carbon monoxide subarachnoid hemorrhage
SB-623 Sunovion Pharmaceuticals chronic stroke Phase II
(cell replacement therapy) Marlborough, MA www.sunovion.com
sodium nitrite Hope Pharmaceuticals reversal of cerebral vasospasm after Phase II
Scottsdale, AZ subarachnoid hemorrhage www.hopepharm.com
Stemedyne™-MSC Stemedica Cell Technologies ischemic stroke Phase II
mesenchymal stem cell therapy San Diego, CA www.stemedica.com
Tysarbi® Biogen acute ischemic stroke Phase II
natalizumab Cambridge, MA (see also multiple sclerosis) www.biogen.com
Tourette's Syndrome
Product Name Sponsor Indication Development Phase
AZD5213 AstraZeneca Tourette's syndrome Phase II
(histamine-3 receptor antagonist) Wilmington, DE (see also pain) www.astrazeneca.com
CPP-115 Catalyst Pharmaceuticals Tourette's syndrome Phase I(4-aminobutyrate transaminase inhibitor) Coral Gables, FL (see also epilepsy) www.catalystpharma.com
Dysport® Ipsen Biopharmaceuticals Tourette's syndrome Phase II
abobotulinumtoxinA Basking Ridge, NJ (see also spasticity) www.ipsenus.com
ecopipam Psyadon Pharmaceuticals Tourette's syndrome Phase II
Germantown, MD www.psyadonrx.com
NBI-98854 Neurocrine Biosciences Tourette's syndrome Phase I
(VMAT2 inhibitor) San Diego, CA (see also other) www.neurocrine.com
SD-809 Auspex Pharmaceuticals Tourette's syndrome (adolescents) Phase I
(VMAT2 inhibitor) La Jolla, CA (see also Huntington's, other) www.auspexpharma.com
Orphan Drug
SNC-102 Synchroneuron Tourette's syndrome Phase II
(acamprosate controlled release) Duxbury, MA (see also other) www.synchroneuron.com
vigabatrin Catalyst Pharmaceutical Partners Tourette's syndrome Phase I/II
(CCP-109) Coral Gables, FL www.catalystpharma.com
Other
Product Name Sponsor Indication Development Phase
AAV1-FS344 Milo Biotechnology inclusion body myositis Phase I/II(gene therapy-delivered Cleveland, OH (see also muscular dystrophy) www.milobiotechnology.com
myostatin inhibitor)
ABT-RTA 408 AbbVie mitochondrial myopathies Phase II(NF/E2 related factor 2 stimulant) North Chicago, IL (see also genetic) www.abbvie.com
Reata Pharmaceuticals www.reatapharma.com
Irving, TX
ACE-083 Acceleron Pharma inclusion body myositis Phase I
(recombinant fusion protein) Cambridge, MA (see also muscular dystrophy) www.acceleronpharma.com
anti-eTau Bristol-Myers Squibb neurodegenerative disease Phase I(BMS 986168) Princeton, NJ www.bms.com
AZD3241 AstraZeneca multiple system atrophy Phase II
(myeloperoxidase inhibitor) Wilmington, DE www.astrazeneca.com
Bendavia™ Stealth BioTherapeutics mitochondrial myopathies Phase II
(mitochondrial permeability transition Newton, MA www.stealthbt.com
pore inhibitor)
Benlysta® GlaxoSmithKline myasthenia gravis Phase II
belimumab Research Triangle Park, NC www.gsk.com
BYM338 Novartis Pharmaceuticals sporadic inclusion body myositis Phase II/III(type II-B activin receptor East Hanover, NJ www.novartis.com
modulator mAb)
Other
Product Name Sponsor Indication Development Phase
Cinryze® Shire ViroPharma neuromyelitis optica Phase I
C1 esterase inhibitor (human) Exton, PA www.shire.com
CK-2127107 Astellas Pharma US skeletal muscle function improvement Phase I completed
Northbrook, IL associated with neuromuscular www.cytokinetics.com
Cytokinetics dysfunction, muscle weakness and/or
South San Francisco, CA muscle fatigue
ferric carboxymaltose Luitpold Pharmaceuticals restless legs syndrome Phase III
Shirley, NY www.luitpold.com
Firdapse™ Catalyst Pharmaceuticals Lambert-Eaton myasthenia syndrome Phase III
amifampridine Coral Gables, FL www.catalystpharma.com
Orphan Drug
Gilenya® Novartis Pharmaceuticals chronic inflammatory demyelinating Phase III
fingolimod East Hanover, NJ polyradiculoneuropathy www.novartis.com
(see also multiple sclerosis)
HT-3951 Dart NeuroScience neurodegenerative disorders Phase I
(monoamine oxidase B inhibitor) San Diego, CA www.dartneuroscience.com
JZP-110 Jazz Pharmaceuticals excessive daytime sleepiness Phase III
(neurotransmitter modulator) Palo Alto, CA associated with narcolepsy www.jazzpharma.com
Orphan Drug
Other
Product Name Sponsor Indication Development Phase
JZP-386 Concert Pharmaceuticals narcolepsy Phase I completed
(dopamine receptor agonist) Lexington, MA www.concertpharma.com
Jazz Pharmaceuticals www.jazzpharma.com
Palo Alto, CA
MEDI-551 MedImmune neuromyelitis optica Phase II
(anti-CD19 mAb) Gaithersburg, MD (see also multiple sclerosis) www.medimmune.com
NBI-98854 Neurocrine Biosciences tardive dyskinesia (Fast Track) Phase III
(VMAT2 inhibitor) San Diego, CA (see also Tourette's) www.neurocrine.com
ofatumumab GlaxoSmithKline neuromyelitis optica Phase II
(CD20 mAb) Research Triangle Park, NC (see also multiple sclerosis) www.gsk.com
Privigen® CSL Behring chronic inflammatory demyelinating Phase III
Immune globulin King of Prussia, PA polyradiculoneuropathy www.cslbehring.com
QPI 1007 Quark Pharmaceuticals non-arteritic anterior ischemic Phase I(gene expression inhibitor) Fremont, CA optic neuropathy www.quarkpharma.com
RGN-352 RegeneRx Biopharmaceuticals central nervous system disorders Phase I completed
(injectable thymosin beta-4) Rockville, MD www.regenerx.com
SA237 Chugai Pharma USA neuromyelitis optica Phase III
(anti-IL-6 receptor mAb) Berkeley Heights, NJ www.chugai-pharm.com
Other
Product Name Sponsor Indication Development Phase
SD-809 Auspex Pharmaceuticals tardive dyskinesia Phase III
(VMAT2 inhibitor) La Jolla, CA (see also Huntington's, Tourette's) www.auspexpharma.com
Orphan Drug
SNC-102 Synchroneuron tardive dyskinesia Phase II
(acamprosate controlled-release) Duxbury, MA (see also Tourette's) www.synchroneuron.com
Soliris® Alexion Pharmaceuticals myasthenia gravis, Phase III
eculizumab Cheshire, CT neuromyelitis optica www.alxn.com
Orphan Drug
TAK-137 Takeda Pharmaceutical neurological disorders Phase I(AMPA receptor modulator) Deerfield, IL www.takeda.com
TAK-935 Takeda Pharmaceutical neurological disorders Phase I(steroid hydroxylase inhibitor) Deerfield, IL www.takeda.com
Tardoxal™ Medicure Pharma tardive dyskinea (Fast Track) Phase IIpyridoxal phosphate Somerset, NJ www.medicure.com
TPI 287 Cortice Biosciences corticobasal dementia, progressive Phase I
(abeotaxane) New York, NY supranuclear palsy www.corticebio.com
Orphan Drug (see also Alzheimer's, brain tumors)
UX001 Ultragenyx Pharmaceutical hereditary inclusion body myositis Phase I/II(aceneuramic acid extended release) Novato, CA www.ultragenyx.com
Other
Product Name Sponsor Indication Development Phase
Zyrem® Jazz Pharmaceuticals narcolepsy (pediatric) Phase III
sodium oxybate Palo Alto, CA www.jazzpharma.com
Definitions
Application Submitted—An application for marketing has been submitted by the company to the U.S. Food and Drug Administration (FDA).
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 status”) 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 fewer than 200,000 people in the United States, or that affects more than 200,000 persons but is not expected to recover
the costs of its development and marketing.
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.
The content of this report has been
obtained through public, government and
industry sources, and the Adis “R&D Insight”
database based on the latest information.
Report current as of June 15, 2015. 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. 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, at
www.phrma.org.