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.