Pipeline

Pipeline new from Stealth Biotherapeutics, Translate Bio, Adynxx, and BiondVax Pharmaceuticals

April 16, 2018

Company Drug/Device Medical Condition Status
Abbott optical coherence tomography (OTC) high-risk, complex coronary artery disease Phase I trials initiated enrolling 3,650 subjects in North America, Europe and Asia
Translate Bio MRT5005 first-in-human clinical trial in patients with cystic fibrosis Phase I/II clearance given by the FDA
Adynxx Inc. brivoligide (AYX1) post-surgical pain Phase II results announced
GTx, Inc. enobosarm post-menopausal stress urinary incontinence (SUI) Phase II trials initiated enrolling 493 subjects at over 60 clinical trial centers across the U.S.
BiondVax Pharmaceuticals Ltd. M-001 universal flu vaccine Phase II trials initiated enrolling 9,630 subjects age 50 years and older across four-to-six countries over a period of two flu seasons
Therapix Ltd. THX-110 adult subjects with Tourette syndrome Phase IIa topline results announced
AbbVie upadacitinib moderate to severe Rheumatoid Arthritis Phase III topline results announced evaluating 37 subjects
Stealth Biotherapeutics elamipretide Barth syndrome Orphan Drug Designation granted
Clovis Oncology Inc. Rubraca (rucaparib) tablets adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy FDA approval granted

Pipeline new from Eli Lilly, AstraZeneca, Wize Pharma, Amgen and Allergan

April 9, 2018

Company Drug/Device Medical Condition Status
Wize Pharma, Inc. LO2A moderate to severe Conjunctivochalasis (CCH) Phase II trials initiated enrolling 62 subjects
Synlogic SYNB1020 hyperammonemia in patients with cirrhosis Phase Ib/IIa trials initiated
Amylyx Pharmaceuticals, Inc. AMX0035 Amyotrophic lateral sclerosis (ALS) Phase II open-label extension initiated enrolling 132 patients in the U.S.
Rigel Pharmaceuticals fostamatinib IgA nephropathy (IgAN) Phase II topline results announced evaluating subjects in the U.S., Asia and Europe
Kazia Therapeutics Limited GDC-0084 glioblastoma multiforme (GBM) Phase II trials initiated
Eli Lilly and Company Cyramza (ramucirumab) hepatocellular carcinoma (HCC) Phase III positive topline results announced evaluating 292 patients across 20 countries in North America, Asia, Europe and Latin America
Allergan, Inc. and Gedeon Richter Ltd. cariprazine bipolar I depression Phase III positive topline results announced evaluating 493 subjects
GenSight Biologics GS010 (rAAV2/2-ND4) Leber Hereditary Optic Neuropathy (LHON) Phase III topline results announced evaluating 37 subjects
Wize Pharma, Inc. LO2A dry-eye syndrome (DES) in patients with Sjögren’s syndrome Phase IV trials initiated enrolling 60 subjects
TARIS Biomedical TAR-200 (GemRIS) organ-confined or locally-advanced Muscle Invasive Bladder Cancer Fast Track Designation granted
Tetra Discovery Partners BPN14770 Fragile X Syndrome Orphan Drug Designation granted
PharmAbcine Inc. TTAC-0001 Glioblastoma Multiforme (GBM) Orphan Drug Designation granted
Scholar Rock SRK-015 spinal muscular atrophy (SMA) Orphan Drug Designation granted
AstraZeneca moxetumomab pasudotox hairy cell leukemia (HCL) Priority Review granted
Dr. Reddy’s Laboratories Ltd. DFN-02 migraine NDA filed with the FDA
AstraZeneca Pharmaceuticals LP BYDUREON type 2 diabetes with inadequate glycemic control FDA approval granted
Amgen Blincyto (blinatumomab) B-cell precursor acute lymphoblastic leukemia (ALL) FDA approval granted

Pipeline new from Flex Pharma, Eigere, Urovant Sciences and MiMedx Group

April 2, 2018

Company Drug/Device Medical Condition Status
GT Biopharma Inc. GTP-004 myasthenia gravis Phase I trials initiated
LivaNova PLC Perceval sutureless aortic heart valve diseased native or malfunctioning prosthetic aortic valve Phase I trials initiated enrolling 160 subjects in China
Five Prime Therapeutics, Inc. FPA150 B7-H4 expression observed in multiple solid tumors, including breast, bladder and gynecologic cancers Phase I trials initiated
vTv Therapeutics Inc. TTP399 type 1 diabetes Phase Ib trials initiated
Allecra Therapeutics AAI101 complicated urinary tract infections (cUTIs) Phase II trials initiated
Flex Pharma Inc. FLX-787 multiple sclerosis Phase II trials initiated enrolling 57 subjects
Eiger Biopharmaceuticals, Inc. exendin 9-39 post-bariatric hypoglycemia (PBH) Phase II trials initiated enrolling 36 subjects
ProMetic Life Sciences Inc. PBI-4050 Alström syndrome Phase II trials initiated enrolling 12 subjects
MiMedx Group, Inc. AminoFix Injectable Plantar Fasciitis Phase IIb trials initiated enrolling 145 subjects
MiMedx Group, Inc. AminoFix Injectable Osteoarthritis (OA) of the knee Phase IIb trials initiated enrolling 318 subjects
BrainStorm Cell Therapeutics Inc. NurOwn amyotrophic lateral sclerosis (ALS) Phase III trials initiated enrolling subjects from Canada
Urovant Sciences vibegron overactive bladder Phase III trials initiated enrolling 1,400 subjects

Pipeline new from AstraZeneca, Cavion, Heron and AMO Pharma

March 26, 2018

Company Drug/Device Medical Condition Status
Avita Medical RECELL deep partial-thickness (second-degree) burns Phase I trials initiated
Surface Oncology SRF231 advanced solid tumors and hematologic malignancies Phase I trials initiated
Cidara Therapeutics, Inc. rezafungin acetate candidemia and/or invasive candidiasis Phase II trials initiated
Arena Pharmaceuticals, Inc. etrasimod ulcerative colitis Phase II trials initiated
AMO Pharma AMO-02 (tideglusib) neuromuscular diseases Phase II trials initiated
Cavion, Inc. CX-8998 generalized epileptic syndromes Phase IIa trials initiated
Antibe Therapeutics ATB-346 gastric or duodenal ulcers of at least 3 mm diameter with unequivocal depth Phase IIb trials initiated enrolling 244 subjects
Regeneron Pharmaceuticals, Inc. EYLEA (aflibercept) moderately severe to severe non-proliferative diabetic retinopathy Phase III trials initiated
AstraZeneca dapagliflozin type 2 diabetes Phase III trials initiated enrolling 321 subjects
Heron Therapeutics HTX-011 Bunionectomy and hernia repair Phase III trials initiated enrolling 412 subjects

Pipeline new from Merck, Biogen, Voyager and Cadent

March 19, 2018

Company Drug/Device Medical Condition Status
XW Laboratories Inc. XW10172 Narcolepsy Phase I trials initiated
Cadent Therapeutics CAD-1883 Neurological (Essential Tremor) and psychiatric disease (Spinocerebellar ataxia) Phase I trials initiated
Arrowhead Pharmaceuticals, Inc. ARO-AAT Alpha-1 liver disease Phase I trials initiated
Voyager Therapeutics VY-AADC Advanced Parkinson’s disease Phase Ib trials initiated
Protalix Biotherapeutics, Inc. OPRX-106 Ulcerative colitis Phase II trials initiated enrolling 24 subjects
Merrimack Pharmaceuticals, Inc. MM-121 Lung cancer Phase II trials initiated expanding from 80 to 100 subjects
Biogen SPINRAZA (nusinersen) Spinal muscular atrophy (SMA) Phase II trials initiated enrolling 25 pre-symptomatic infants six weeks old or younger
MyoKardia Mavacamten Hypertrophic cardiomyopathy (HCM) Phase II trials initiated
AM-Pharma recombinant human Alkaline Phosphatase (recAP) Acute Kidney Injury (AKI) Phase II trials initiated enrolling 301 subjects with sepsis
Eleven Biotherapeutics, Inc. Vicinium Non-muscle invasive bladder cancer (NMIBC) previously treated with bacillus Calmette-Guérin (BCG) Phase III trials initiated
AbbVie Elagolix Uterine fibroids Phase III trials initiated
Merck KEYTRUDA (pembrolizumab) Advanced cervical cancer with disease progression on or after chemotherapy Priority Review granted by the FDA
Cerapedics, Inc. P-15L Transforaminal lumbar interbody fusion (TLIF) surgery for degenerative disk disease IDE Approval granted by the FDA
Proteostasis Therapeutics PTI-428 Cystic fibrosis Breakthrough Designation granted by the FDA

Pipeline new from Seattle Genetics, Daiichi Sankyo, Aimmune Therapeutics, Polyganics and more

March 12, 2018

Company Drug/Device Medical Condition Status
Seattle Genetics, Inc. SGN-CD48A Relapsed or refractory multiple myeloma Phase I trial initiated enrolling 75 subjects
Daiichi Sankyo Company, Ltd. DS-8201 HER2-expressing advanced colorectal cancer Phase II trial initiated enrolling 50 subjects in North America, Europe and Japan
GTx, Inc. Enobosarm Stress urinary incontinence (SUI) Phase II trial initiated enrolling 18 female subjects
InflaRx IFX-1 Moderate or severe Hidradenitis Suppertiva (HS) Phase IIb trials initiated enrolling 175 subjects in the U.S., Germany, Greece, Denmark and the Netherlands
Aimmune Therapeutics, Inc. AR101 Peanut allergy Phase III trials initiated enrolling 850 subjects ages 4-49
Vanda Pharmaceuticals, Inc. Hetlioz Jet lag disorder Phase III trials initiated enrolling 318 subjects
Biohaven Pharmaceutical Holding Company Ltd. Zydis ODT formulation of rimegepant Migraine Phase III trials initiated enrolling 850 subjects
Bioverativ, Inc BIVV009 Cold agglutinin disease Phase III trials initiated
MeiraGTx AAOO2 Achromatopsia PRIME designation grated by the EMA
Tocagen Inc. Toca 511 (vocimagene amiretrorepvec) & Toca FC (flucytosine) Glioma Orphan drug designated granted by the EMA
InVivo Therapeutics Holdings Corp. Neuro-Spinal Scaffold Acute spinal cord injury (SCI) FDA approval granted
Polyganics Liver and Pancreas Sealant Patch Fluid leakage after hepato-pancreato-biliary (HPB) procedures FDA breakthrough device designation granted

Pipeline new from Galera, Jazz, Merrimack, and DBV Technologies

March 5, 2018

Company Drug/Device Medical Condition Status
MabVax Therapeutics Holdings, Inc. MVT-1075 Pancreatic, colon and lung cancer Phase I
BioAtla, LLC BA3011 Advanced solid tumors including non-small cell lung cancer (NSCLC) castration resistant prostate cancer and pancreatic cancer Phase I/II trials initiated
Galera Therapeutics, LLC GC4419 Locally advanced pancreatic cancer (LAPC) Phase I/II trials initiated
Jazz Pharmaceuticals Defitelio (defibrotide sodium) Acute Graft-versus-Host-Disease (aGvHD) in adult and pediatric patients after allogenic hematopoietic stem cell transplant (HSCT) Phase II trials initiated enrolling 150 subjects in the U.S., Canada and European Union
DBV Technologies Viaskin Milk IgE-medicated cow’s milk protein allergy Phase II trials initiated enrolling 198 subjects
Merrimack Pharmaceuticals, Inc. MM-121 (seribantumab) Heregulin-positive, hormone rececptor-positive and HER2-negative post-menopausal metastic breast cancer Phase II trials initiated
ARCA biopharma Gencaro (bucindolol hydrochloride) Atrial fibrillation Phase IIb trials initiated enrolling 267 subjects from the U.S., Canada and Europe
Nuvelution Pharma, Inc. AUSTEDO Tourette syndrome in pediatric patients Phase II/III trials initiated

2018-02-26: New Progress in Treatments for Cystic Fibrosis, Skin Conditions and Cancer

February 26, 2018

Company Drug/Device Medical Condition Status
Arch Biopartners AB569 antibiotic-resistant bacterial infections in the lungs of cystic fibrosis and chronic obstructive pulmonary disease Phase I trials initiated in the U.S.
Xencor XmAb18087 neuroendocrine tumors (NET) and gastrointestinal stromal tumors (GIST) Phase I trials initiated
PQ Bypass DETOUR Long (>15 cm) superficial femoral artery (SFA) blockages Phase I trials initiated enrolling 292 subjects in the U.S. and Europe
Therachon TA-46 achondroplasia Phase I trials initiated enrolling 70 subjects in the Netherlands
OncoPep PVX-4106 metastatic triple negative breast cancer (TNBC) who are human leukocyte antigen A2 positive (HLA-A2+) Phase Ib trials initiated
Bonti EB-001 scar reduction Phase II trials initiated
AbbVie JAK1-selective inhibitor adult patients with moderate to severe atopic dermatitis Phase II trials initiated
IRX Therapeutics IRX-2 squamous cervical intraepithelial neoplasia 3 or vulvar intraepithelial neoplasia 3 Phase II trials initiated
Glenmark Pharmaceuticals GBR 830 moderate to severe atopic dermatitis Phase IIa trials initiated enrolling 64 subjects
Centrexion Therapeutics CNTX-4975 Chronic moderate to severe knee osteoarthritis Phase III trials initiated enrolling 325 subjects
AstraZeneca IMFINZI® unresectable Stage III non-small cell lung cancer following CRT Phase III trials initiated
Celgene OTEZLA \(apremilast) active Behçet’s Disease Phase III RELIEF trials initiated
Vertex Pharmaceuticals Incorporated VX-659 cystic fibrosis Phase III trials initiated enrolling 360 patients
Eli Lilly Taltz (ixekizumab) moderate to severe genital psoriasis Phase IIIb trials initiated enrolling 149 subjects
Evofem Biosciences Amphora urogenital chlamydia infection in women Fast Track designation granted by the FDA
Novartis Cosentyx (secukinumab) moderate to severe plaque psoriasis FDA approved label update

Twenty-one Drugs and Devices have Entered a New Trial Phase This Week

February 19, 2018

Company Drug/Device Medical Condition Status
Applied Therapeutics AT-001 diabetic complications Phase I trials initiated
Aravive Biologics AVB-S6-500 n/a Phase I trials initiated enrolling 40 subjects in the U.S.
Mallinckrodt ExpressGraftC9T1 skin tissue diabetic foot ulcers Phase I trials initiated enrolling six subjects
Pulmatrix Pulmazole (PUR1900) allergic bronchopulmonary aspergillosis related to asthma Phase I trials initiated enrolling 42 subjects
Vyera Pharmaceuticals TUR-006 toxoplasmosis Phase I trials initiated
MyoKardia MYK-491 dilated cardiomyopathy Phase Ib trials initiated enrolling 64 subjects
Arrowhead Pharmaceuticals ARO-HBV chronic hepatitis B virus infection Phase I/II trials planned enrolling 30 subjects in New Zealand
Audentes Therapeutics AT342 Crigler-Najjar syndrome Phase I/II trials initiated
Sienna Biopharmaceuticals SNA-125 psoriasis and the associated pruritus Phase I trials initiated enrolling 15 subjects
Imara IMR-687 sickle cell disease Phase IIa trials initiated enrolling 50 subjects in the U.S.
Nohla Therapeutics NLA101 acute myeloid leukemia Phase II trials initiated enrolling 220 subjects in the U.S.
Citius Pharmaceuticals, Inc. Mino-Lok Therapy catheter related bacteremias Phase III trials initiated enrolling 700 subjects in the U.S.
Intercept Pharmaceuticals obeticholic acid cirrhosis due to NASH Phase III trials planned enrolling 540 subjects
Pfizer PF-04965842 moderate to severe atopic dermatitis Breakthrough Therapy designation granted by the FDA
Abeona Therapeutics ABO-202 infantile Batten disease Orphan Drug designation granted by the FDA
AstraZeneca, Merck selumetinib neurofibromatosis type 1 Orphan Drug designation granted by the FDA
Xeris Pharmaceuticals ready-to-use glucagon Hyperinsulinemic Hypoglycemia Orphan Drug designation granted by the FDA
60 Degrees Pharmaceuticals Tafenoquine prevention of malaria Priority Review designation granted by the FDA
Genentech Rituxan (rituximab) pemphigus vulgaris Priority Review designation granted by the FDA
Janssen Erleada (apalutamide) non-metastatic castration-resistant prostate cancer FDA approved
NorthStar Medical Radioisotopes RadioGenix System Non-uranium Sourced Molybdenum-99 Production of Imaging Isotope Technetium-99m FDA approved

Eighteen Drugs and Devices have Entered a New Trial Phase This Week

February 12, 2018

Company Drug/Device Medical Condition Status
Fibrocell Science FCX-013 moderate to severe localized scleroderma IND submitted to the FDA
Daiichi Sankyo U3-1402 metastatic or unresectable epidermal growth factor receptor-mutated non-small cell lung cancer Phase I trials initiated enrolling 60 subjects globally
Palatin Technologies PL-8177 ulcerative colitis and other inflammatory bowel diseases Phase I trials initiated enrolling 52 subjects
Phosplatin Therapeutics PT-112 relapsed or refractory multiple myeloma Phase I/II trials initiated
Gemphire gemcabene pediatric nonalcoholic fatty liver disease Phase IIa trials initiated enrolling 40 adolescent children between the ages of 12 and 17
OncBioMune Pharmaceuticals ProscaVax advanced prostate cancer Phase II trials initiated
Xcovery ensartinib (X-396) advanced malignant melanoma harboring alterations in ALK Phase II trials initiated
Endo International collagenase clostridium histolyticum cellulite Phase III trials initiated enrolling 840 women in the U.S.
Pharma Two B P2B001 early Parkinson’s disease Phase III trials initiated enrolling 525 subjects
Tessa Therapeutics T cell immunotherapy nasopharyngeal cancer Phase III trials initiated enrolling 35 subjects
GlaxoSmithKline Bexsero meningitis B vaccine Breakthrough Therapy designation granted by the FDA
Sage Therapeutics SAGE-217 major depressive disorder Breakthrough Therapy designation granted by the FDA
Zogenix ZX008 (low-dose fenfluramine) seizures associated with Dravet syndrome Breakthrough Therapy designation granted by the FDA
MeiraGTx AAV2/8-hCARp.hCNGB3 (A002) achromatopsia due to mutations in the CNGB3 gene Rare Pediatric Disease designation granted by the FDA
Adhesys Medical VIVO surgical sealant Expedited Access Pathway designation granted by the FDA
Alnylam Pharmaceuticals patisiran hereditary ATTR amyloidosis NDA accepted and Priority Review granted by the FDA
Allergan Avycaz (ceftazidime and avibactam) hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia Expanded use approved by the FDA
Ferring Pharmaceuticals Zomacton (somatropin) human growth hormone deficiency FDA approved