Pipeline

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

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

February 5, 2018

Company Drug/Device Medical Condition Status
Immuron IMM-529 Clostridium Difficile Infection Phase I/II trials initiated enrolling 60 subjects
SillaJen pexastimogene devacirepvec (Pexa-Vec) metastatic colon cancer Phase I/II trials initiated
Mapi Pharma GA Depot primary progressive multiple sclerosis Phase II trials initiated enrolling 12-24 subjects
Merus MCLA-128 metastatic breast cancer Phase II trials initiated enrolling 120 subjects in the U.S. and Europe
Novan SB206 molluscum contagiosum Phase II trials initiated enrolling 192 children and adolescents
Aldeyra Therapeutics topical ocular reproxalap dry eye disease Phase IIb trials initiated enrolling 300 subjects
Dermira lebrikizumab moderate to severe atopic dermatitis Phase IIb trials initiated enrolling 350 subjects in the U.S.
BeiGene tislelizumab advanced unresectable or metastatic esophageal squamous cell carcinoma Phase III trials initiated enrolling 350 subjects globally
Cara Therapeutics Korsuva (CR845/difelikefalin) hemodialysis patients suffering from moderate to severe chronic kidney disease-associated pruritus Phase III trials initiated enrolling 350 subjects in the U.S.
Diffusion Pharmaceuticals trans sodium crocetinate newly-diagnosed inoperable glioblastoma multiforme Phase III trials initiated enrolling 264 subjects
Aquestive Therapeutics Riluzole Oral Soluble Film amyotrophic lateral sclerosis Orphan Drug designation granted by the FDA
Dauntless Pharmaceuticals DP1038 acromegaly Orphan Drug designation granted by the FDA
Ensysce Biosciences PF614 moderate to severe chronic pain Fast Track designation granted by the FDA
Faron Pharmaceuticals Traumakine acute respiratory distress syndrome Fast Track designation granted by the FDA
Protalix Biotherapeutics PRX-102 Fabry disease Fast Track designation granted by the FDA
Synthon [vic-]trastuzumab duocarmazine (SYD985) HER2-positive metastatic breast cancer Fast Track designation granted by the FDA
Takeda Pharmaceutical TAK-426 Zika virus vaccine Fast Track designation granted by the FDA
LimFlow LimFlow Percutaneous Deep Vein Arterialization System end-stage critical limb ischemia Breakthrough Device designation granted by the FDA
Roche balovaptan autism spectrum disorder Breakthrough Therapy Designation granted by the FDA
AngioDynamics NanoKnife System stage III pancreatic cancer Expedited Access Pathway designation granted by the FDA
Abeona Therapeutics EB-101 recessive dystrophic epidermolysis bullosa Regenerative Medicine Advanced Therapy designation granted by the FDA
CutisPharma CutisPharma Firvanq (vancomycin hydrochloride) Clostridium difficile associated diarrhea and enterocolitis caused by Staphylococcus aureus FDA approved
Synergy Pharmaceuticals Trulance (plecanatide) irritable bowel syndrome with constipation FDA approved

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

January 29, 2018

Company Drug/Device Medical Condition Status
Apexian Pharmaceuticals APX3330 advanced solid tumors Phase I trials initiated
Bavarian Nordic BN-Brachyury cancer metastasis Phase I trials initiated enrolling 10 subjects
Protalex PRTX-100 (PRTX-100-203 Study) persistent/chronic Immune Thrombocytopenia Phase Ib trials initiated enrolling 30 subjects in Europe
IntelGenx Montelukast VersaFilm Alzheimer’s disease Phase IIa trials initiated
Biomarck Pharmaceutical BIO-11006 non-small cell lung cancer Phase II trials initiated in India
Melinta Therapeutics radezolid bacterial vaginosis Phase II trials initiated
TrovaGene PCM-075 + Zytiga metastatic castration-resistant prostate cancer Phase II trials initiated enrolling 25 subjects
Evofem Biosciences Amphora prevention of urogenital chlamydia and gonorrhea in women Phase IIb/III trials initiated enrolling 850 subjects in the U.S.
GC Pharma GC1102 hepatitis B virus infection following liver transplantation Phase II/III trials planned
Swedish Orphan Biovitrum SOBI003 mucopolysaccharidosis type IIIA Fast Track designation granted by the FDA
Astellas Pharma gilteritinib acute myeloid leukemia Orphan Drug designation granted by the EC
BERG BPM31510 pancreatic cancer Orphan Drug designation granted by the FDA
Enzychem Lifesciences EC-18 acute radiation syndrome Orphan Drug designation granted by the FDA
Nohla Therapeutics NLA101 hematopoietic stem cell transplantation Orphan Drug designation granted by the EC
Omeros OMS721 primary Immunoglobulin A nephropathy Orphan Drug designation granted by the EMA
Orphazyme arimoclomol Niemann-Pick disease Type C Rare Pediatric Disease designation granted by the FDA
Genmab daratumumab (Darzalex) + bortezomib, melphalan and prednisone newly diagnosed multiple myeloma Priority Review granted by the FDA
Baxter International Bivalirudin anticoagulant in patients undergoing percutaneous coronary intervention FDA approved
Bristol-Myers Squibb Yervoy (ipilimumab) advanced (unresectable or metastatic) melanoma in pediatric patients 12 years of age and older EC expanded indication approved
Takeda Pharmaceutical Adcetris (brentuximab vedotin) CD30-positive cutaneous T-cell lymphoma EC extended current conditional marketing authorization

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

January 22, 2018

Company Drug/Device Medical Condition Status
Tetra Bio-Pharma PPP005 safety study Phase I trials planned in Canada
Constellation Pharmaceuticals CPI-1205 oncology Phase Ib/II trials initiated
CytRx Aldoxorubicin metastatic pancreatic cancer Phase Ib/II trials initiated enrolling 173 subjects
Transgene TG4010 MUC1 expressing tumors Phase II trials initiated enrolling 39 subjects
Ophthotech Zimura (avacincaptad pegol) autosomal recessive Stargardt disease (STGD1) Phase IIb trials initiated enrolling 120 subjects
MiMedx AmnioFix Injectable recalcitrant plantar fasciitis pain Phase III trials initiated enrolling 164 subjects
LivaNova Aortic Valve Implant reporting of reduced leaflet motion Post-Market trials initiated enrolling 230 subjects in the U.S. and Canada
Aspyrian Therapeutics RM-1929 locoregional recurrent head and neck squamous cell carcinoma Fast Track designation granted by the FDA
Centrexion CNTX-4975 pain associated with knee osteoarthritis Fast Track designation granted by the FDA
Concert Pharmaceuticals CTP-543 moderate to severe alopecia areata Fast Track designation granted by the FDA
Cour Pharmaceuticals TIMP-GLIA Celiac disease Fast Track designation granted by the FDA
Benitec BioPharma BB-301 oculopharyngeal muscular dystrophy Orphan Drug designation granted by the FDA
Helsinn, MEI Pharma pracinostat acute myeloid leukemia Orphan Drug designation granted by the EMA
Novartis Kymriah (tisagenlecleucel) relapsed or refractory diffuse large B-cell lymphoma Priority Review granted by the FDA, Accelerated Assessment granted by the EMA
ACON Laboratories, Innovus Pharma UriVarx Urinary Tract Infection test strips overactive bladder, urinary incontinence FDA administration clearance
AstraZeneca, Merck Lynparza (olaparib) deleterious or suspected deleterious gBRCAm HER2-negative metastatic breast cancer FDA approved
Avion Pharmaceuticals Balcoltra (levonorgestrel and ethinyl estradiol tablets and ferrous bisglycinate tablets) oral contraceptive FDA approved
Boehringer Ingelheim Gilotrif (afatinib) metastatic non-small cell lung cancer with non-resistant EGFR mutations FDA approved
GlaxoSmithKline Fluarix Quadrivalent (Influenza Vaccine) flu in patients 3 years of age or older FDA expanded indication approved
Innovus Pharma Xyralid (DIN 02471434) pain and symptom relief for hemorrhoids Health Canada approved
Myriad Genetics BRACAnalysis CDx HER2-negative metastatic breast cancer with germline BRCA mutation FDA approved