Pipeline

New Pipeline Updates from CStone Pharmaceuticals, Mallinckrodt plc and TB Alliance

August 19, 2019

Company Drug/Device Medical Condition Status
Coordination Pharmaceuticals Inc. CPI-200 advanced tumors Phase 1 trial initiated enrolling 27 subjects
Rimo Therapeutics Inc. RiMO-301 advanced tumors Phase 1 trial initiated enrolling 18 subjects
CStone Pharmaceuticals BLU-667 RET-altered non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC) and other advanced solid tumors Phase 1 trial initiated
Bio-Thera Solutions, Ltd. BAT2506 rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis (AS) and ulcerative colitis Phase 1 trial initiated enrolling 182 healthy volunteers
Mallinckrodt plc MNK-6106 (L-ornithine phenylacetate) hepatic (liver) cirrhosis and hepatic encephalopathy (HE) Phase 2a trial initiated enrolling 48 adult male and non-pregnant female subjects with hepatic cirrhosis who have a history of at least two or more documented episodes of HE in the last 12 months – with one in the last six months – and who present with hyperammonemia at the time of screening
Kedrion Biopharma 10 percent intravenous immunoglobulin primary immunodeficiency diseases (PI) Phase 3 trial initiated enrolling 50 subjects at 16 sites in the U.S.
binx health binx io (first-of-kind, highly sensitive and specific, molecular point-of-care (POC) diagnostic testing platform) chlamydia (CT) and gonorrhea (NG) 510(k) clearance granted by the FDA
Regeneron Pharmaceuticals, Inc. EYLEA (aflibercept) Injection 2 mg, single-dose, sterilized prefilled syringe multiple retinal diseases, including wet age-related macular degeneration and diabetic eye diseases Approval granted by the FDA
TB Alliance Pretomanid Tablets in combination with bedaquiline and linezolid Extensively Drug-Resistant Tuberculosis (XDR-TB) and treatment intolerant/non-responsive Multidrug-Resistant (MDR) TB Approval granted by the FDA

 

New Pipeline Updates from Daiichi Sankyo, Precigen and Altavant Sciences

August 12, 2019

Company Drug/Device Medical Condition Status
Precigen, Inc. PRGN-3005 UltraCAR-T advanced, recurrent platinum resistant ovarian, fallopian tube or primary peritoneal cancer Phase 1 trial initiated
Soricimed Biopharma, Inc. SOR-C13 late stage pancreatic cancer Phase 1b trial initiated enrolling 36 subjects with advanced solid tumor cancers at The University of Texas MD Anderson Cancer Center (MDACC)
89Bio Ltd. BIO89-100 nonalcoholic steatohepatitis (NASH) or nonalcoholic fatty liver disease (NAFLD) and high risk of NASH Phase 1b/2a trial initiated enrolling 83 subjects
Provention Bio, Inc. PRV-3279 lupus Phase 1b/2a trial initiated enrolling 16 healthy volunteers
Eureka Therapeutics, Inc. ET140202 ARTEMIS T-Cell therapy advanced hepatocellular carcinoma (HCC) liver cancer Phase 1/2 trial initiated enrolling subjects with metastatic or locally advanced, inoperable liver cancer and have progressed or have not been able to tolerate at least one line of treatment at City of Hope in Duarte, CA
RegeneRx Biopharmaceuticals, Inc. RGN-137 epidermolysis bullosa (EB) Phase 2 trial initiated
POXEL SA PXL770 NASH Phase 2a trial initiated enrolling 100 subjects with nonalcoholic fatty liver disease (NAFLD) who likely have NASH at sites in the U.S.
Altavant Sciences rodatristat ethyl pulmonary arterial hypertension (PAH) Phase 2a trial initiated enrolling 36 subjects
Astellas Pharma, Inc. fezolinetant moderate-to-severe vasomotor symptoms (VMS) Phase 3 trial initiated enrolling 450 subjects at 20 sites in the U.S., Canada and Europe
Apyx Medical Corporation next-generation J-Plasma Precise Handpiece open and laparoscopic surgical procedures 510(k) clearance granted by the FDA
Alnylam Pharmaceuticals, Inc. givosiran acute hepatic porphyria (AHP) Priority Review granted by the FDA
Provention Bio, Inc. teplizumab (PRV-031) prevention or delay of clinical type 1 diabetes (T1D) in at-risk individuals Breakthrough Therapy designation granted by the FDA
Inotrem S.A. nangibotide (LR12) septic shock IND approval granted by the FDA
Daiichi Sankyo Turalio (pexidartinib) capsules subjects with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not responsive to improvement with surgery Approval granted by the FDA
Bracco Diagnostics, Inc. VARIBAR THIN LIQUID (barium sulfate) for oral suspension dysphagia Approval granted by the FDA

 

New Pipeline Updates from AC Immune SA, Abivax and Merck

August 5, 2019

Company Drug/Device Medical Condition Status
Miracor Medical SA PiCSO therapy heart attack Phase 1 trial initiated enrolling 114 subjects with anterior STEMI, TIMI 0 & 1 Flow, at nine clinical sites in Western Europe
Reflow Medical, Inc. Temporary Spur Stent System peripheral artery disease (PAD), occlusive disease affecting below-the-knee (BTK) arteries Phase 1 trial initiated
I-Mab Biopharma Tracon Pharmaceuticals, Inc. TJD5 (TJ004309) advanced solid tumors Phase 1 trial initiated
AC Immune SA ACI-35.030 Alzheimer’s disease (AD) Phase 1b/2a trial initiated
Proteostasis Therapeutics, Inc. cystic fibrosis transmembrane conductance regulator (CFTR) modulator combinations doublet (PTI-808 and PTI-801) and triplet (PTI-808, PTI-801 and PTI-428) cystic fibrosis (CF) Phase 2 trial initiated enrolling 30 F508del homozygous and 30 F508del heterozygous subjects
Arena Pharmaceuticals olorinab gastrointestinal (GI) disorders Phase 2 trial initiated enrolling 240 subjects experiencing abdominal pain associated with IBS, including IBS with constipation (IBS-C) or IBS with diarrhea (IBS-D) in sites across the U.S.
Relmada Therapeutics, Inc. REL-1017 (dextromethadone) treatment-resistant depression Phase 2 trial initiated enrolling 62 subjects
BELLUS Health Inc. BLU-5937 chronic cough Phase 2 trial initiated enrolling 65 subjects with refractory chronic cough at 12 sites in the U.S. and U.K.
Auris Medical Holding Ltd. AM-125 acute vertigo Phase 2 trial initiated enrolling 138 subjects who suffer from acute vertigo following surgical removal of a vestibular schwannoma in six European countries and Canada
Resolve Therapeutics RSLV-132 Systemic Lupus Erythematosus Phase 2a trial initiated enrolling 64 subjects at 20 sites across the U.S.
Abivax ABX464-301 moderate to severe active rheumatoid arthritis (RA) Phase 2a trial initiated enrolling 60 subjects in France, Poland, Czech Republic and Hungary
Modra Pharmaceuticals B.V. ModraDoc006/r metastatic Castration-Resistant Prostate Cancer (mCRPC) Phase 2b trial initiated enrolling 100 subjects with mCRPC eligible for first line systemic chemotherapy at 40 sites in the U.S. and Europe
Biohaven Pharmaceutical Holding Company Ltd. verdiperstat Multiple System Atrophy (MSA) Phase 3 trial initiated enrolling 250 subjects at 50 sites in the U.S. and Europe
AZTherapies, Inc. ALZT-OP1 early Alzheimer’s disease Phase 3 trial initiated enrolling 620 subjects with early stage Alzheimer’s disease, ages 55-79
Nektar Therapeutics Bristol-Myers Squibb bempegaldesleukin (NKTR-214) in combination with Opdivo (nivolumab) subjects with previously untreated unresectable or metastatic melanoma Breakthrough Therapy designation granted by the FDA
Endotronix, Inc. Cordella Pulmonary Artery (PA) Pressure Sensor System chronic heart failure IDE approval granted by the FDA
Intersect ENT PROPEL mini steroid Straight Delivery System (SDS) frontal and ethmoid sinus surgery Approval granted by the FDA
Retrophin, Inc. THIOLA EC (tiopronin) 100 mg and 300 mg tablets cystinuria Approval granted by the FDA
Merck KEYTRUDA (pembrolizumab) subjects with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10) with disease progression after one or more prior lines of systemic therapy Approval granted by the FDA
Bayer Nubeqa (darolutamide) non-metastatic castration-resistant prostate cancer (nmCRPC) Approval granted by the FDA

 

New Pipeline Updates from Abbott, Merck and CStone Pharmaceuticals

July 29, 2019

Company Drug/Device Medical Condition Status
Ascentage Pharma APG-2575 hematologic malignancies Phase 1 trial initiated enrolling subjects with acute myelogenous leukemia (AML) and non-Hodgkin’s lymphoma (NHL) in China
Halozyme Therapeutics efgartigimod (ARGX-113) severe autoimmune diseases Phase 1 trial initiated
AC Immune SA ACI-3024 neurodegenerative diseases that are characterized by the presence of pathological Tau aggregates Phase 1 trial initiated enrolling healthy volunteers
NuCana plc NUC-7738 cancer Phase 1 trial initiated
CStone Pharmaceuticals ivosidenib (TIBSOVO) relapsed or refractory acute myeloid leukemia (R/R AML) with an IDH1 mutation Phase 1 trial initiated
Prescient Therapeutics Limited PTX-100 cancer Phase 1b trial initiated enrolling subjects in Australia
Axial Biotherapeutics AB-2004 gastrointestinal dysfunction and associated behavioral symptoms of Autism Spectrum Disorder (ASD) Phase 1b/2a trial initiated enrolling 25 male subjects with ASD
ImaginAb Inc. CD8 tracer 89Zr-Df-IAB22M2C cancer Phase 2 trial initiated enrolling subjects at Dana-Farber Cancer Institute in Boston
DelMar Pharmaceuticals, Inc. VAL-083 MGMT-unmethylated glioblastoma multiforme (GBM) Phase 2 trial initiated enrolling 24 newly-diagnosed subjects who have undergone surgery and chemoradiation with temozolomide (TMZ) at the University of Texas MD Anderson Cancer Center (MDACC)
EIP Pharma, Inc. neflamapimod cognitive dysfunction associated with dementia with Lewy bodies (DLB) Phase 2 trial initiated enrolling 80 subjects at several sites in the U.S. and Netherlands
Oyster Point Pharma, Inc. OC-01 Nasal Spray dry eye disease Phase 3 trial initiated enrolling 750 subjects at 20 centers in the U.S.
LifeMax Laboratories, Inc. LM-030 Netherton Syndrome Rare Pediatric Disease designation granted by the FDA
Merck and Eisai KEYTRUDA in combination with LENVIMA subjects with advanced unresectable hepatocellular carcinoma (HCC) not amenable to locoregional treatment Breakthrough Therapy designation granted by the FDA
LivaNova PLC LifeSPARC Advanced Circulatory Support (ACS) system cardiac and respiratory failure 510(k) clearance granted by the FDA
Aridis Pharmaceuticals, Inc. AR-501 cystic fibrosis Orphan Drug designation granted by the FDA
Arrowhead Pharmaceuticals, Inc. ARO-ANG3 homozygous familial hypercholesterolemia (HoFH) Orphan Drug designation granted by the FDA
Apic Bio, Inc. APB-102 genetic SOD1 amyotrophic lateral sclerosis (ALS) Orphan Drug designation granted by the FDA
Teva Pharmaceuticals Industries Ltd. AirDuo Digihaler (fluticasone propionate 113 mcg and salmeterol 14 mcg) Inhalation Powder asthma Approval granted by the FDA
Novadoz Pharmaceuticals Abiraterone Acetate 250mg tablet prostate cancer when used with a steroid medication (prednisone or methylprednisolone) Approval granted by the FDA
Bayer Gadavist (gadobutrol) injection coronary artery disease (CAD) Approval granted by the FDA
Abbott MitraClip heart valve repair device mitral regurgitation Approval granted by the FDA
Merck RECARBRIO (imipenem, cilastatin and relebactam) for injection (1.25 grams) adults with complicated urinary tract and complicated intra-abdominal bacterial infections where limited or no alternative treatment options are available Approval granted by the FDA
Celgene OTEZLA (apremilast) adult with oral ulcers associated with Behçet’s Disease Approval granted by the FDA
Baxter Myxredlin ready-to-use insulin for IV infusion Approval granted by the FDA
Pfizer, Inc. RUXIENCE (rituximab-pvvr) adult subjects with non-Hodgkin’s lymphoma (NHL), chronic lymphocytic leukemia (CLL) and granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) Approval granted by the FDA
Samsung Bioepis Co., Ltd. HADLIMA (adalimumab-bwwd) heumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, adult Crohn’s disease, ulcerative colitis and plaque psoriasis Approval granted by the FDA
Eli Lilly and Company Baqsimi (glucagon) nasal powder 3 mg severe hypoglycemia Approval granted by the FDA

 

New Pipeline Updates from Cyxone, Trovagene and Sensorion

July 22, 2019

Company Drug/Device Medical Condition Status
Compass Therapeutics CTX-471 metastatic or locally advanced solid cancers Phase 1 trial initiated
Cyxone T20K multiple sclerosis (MS) Phase 1 trial initiated
SOTIO SO-C101 advanced/metastatic solid tumors Phase 1/1b trial initiated
TARIS Bio TAR-200 in combination with Opdivo (nivolumab) muscle-invasive bladder cancer (MIBC) Phase 1b trial initiated enrolling 25 subjects with MIBC who are scheduled for radical cystectomy
Metacrine, Inc. MET409 non-alcoholic steatohepatitis (NASH) Phase 1b trial initiated
IDEAYA Biosciences, Inc.o IDE196 solid tumors that harbor GNAQ/11 mutations or PRKC fusions Phase 1/2 trial initiated
Trovagene, Inc. onvansertib in combination with FOLFIRI and Avastin (bevacizumab) metastatic colorectal cancer (mCRC) with a KRAS mutation (NCT03829410) Phase 1b/2 trial initiated
EIP Pharma, Inc. neflamapimod Huntington’s disease Phase 2 trial initiated enrolling 16 subjects at a site in Cambridge UK
Cara Therapeutics, Inc. Oral KORSUVA (CR845/difelikefalin) pruritus in subjects with atopic dermatitis (AD) Phase 2 trial initiated enrolling 240 adult subjects with AD
Dilafor AB tafoxiparin pregnant women planned for labor induction Phase 2b trial initiated
NeuroRx, Inc. NRX-101 Severe Bipolar Depression and Acute Suicidal Ideation and Behavior (ASIB) Phase 2b/3 trial initiated enrolling 140 subjects with Severe Bipolar Depression and ASIB who are stabilized following a single IV infusion of ketamine JPS Health Network in Fort Worth, Texas
Mirum Pharmaceuticals maralixibat progressive familial intrahepatic cholestasis (PFIC) in pediatric subjects Phase 3 trial initiated enrolling 30 subjects with residual BSEP function (PFIC2 non-truncating) aged one to 17 years
CStone Pharmaceuticals avapritinib gastrointestinal stromal tumors (GIST) Phase 3 trial initiated
Sensorion SENS-401 Sudden Sensorineural Hearing Loss (or SSNHL) IND approval granted by the FDA
Karyopharm Therapeutics selinexor in combination with dexamethasone relapsed refractory multiple myeloma in subjects who have received at least three prior therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent and one anti-CD38 monoclonal antibody Approval granted by the FDA
ConTIPI Medical, Ltd. ProVate pelvic prolapse in women Approval granted by the FDA
Azurity Pharmaceuticals Katerzia (amlodipine) Oral Suspension, 1 mg/mL hypertension (high blood pressure) in adults and pediatric patients six years of age and older and coronary artery disease in adults Approval granted by the FDA

 

New Pipeline Updates from Emmaus Life Sciences, Inc., GlaxoSmithKline and Janssen

July 15, 2019

Company Drug/Device Medical Condition Status

Emmaus Life Sciences, Inc.

pharmaceutical-grade L-glutamine (PGLG)

diverticulosis

Phase 1 trial initiated enrolling 10 patients at multiple sites

Enanta Pharmaceuticals, Inc.

EDP-514

hepatitis B virus (HBV)

Phase 1a/1b trial initiated enrolling 98 subjects

Neurovive Pharmaceutical AB

KL1333

mitochondrial disease

Phase 1a/1b trial initiated enrolling healthy subjects in the UK after successful completion of the first stage of enrollment

Auris Medical
Holding Ltd.

AM-201

antipsychotic-induced weight gain and somnolence

Phase 1b trial initiated enrolling healthy volunteer subjects in Europe

4D pharma plc

MRx-4DP0004

asthma

Phase 1/2 trial initiated enrolling 90 subjects not adequately controlled on their current inhaler maintenance therapy

Vedanta Biosciences

VE416

peanut allergy

Phase 1b/2 trial initiated enrolling 40 subjects 12 years of age and older at MassGeneral Hospital for Children in Boston

ActoBio Therapeutics, Inc.

AG019

early-onset type 1 diabetes (T1D)

Phase 1b/2a trial initiated enrolling subjects 12 to 17 years of age

ActoBio Therapeutics, Inc.

AG019 combined with teplizumab (PRV-031)

early-onset type 1 diabetes (T1D)

Phase 1b/2a trial initiated enrolling adult subjects

Biohaven Pharmaceutical Holding Company Ltd.

rimegepant

treatment refractory trigeminal neuralgia

Phase 2 trial initiated enrolling subjects at Johns Hopkins Medical Center

GlaxoSmithKline

otilimab

moderate to severe rheumatoid arthritis (RA)

Phase 3 trial initiated

BiondVax Pharmaceuticals Ltd.

M-001

influenza

Phase 3 trial initiated

Alexion Pharmaceuticals, Inc.

SOLIRIS (eculizumab)

neuromyelitis optica spectrum disorder (NMOSD) in adult subjects who are anti-aquaporin-4 (AQP4) antibody positive

Approval granted by the FDA

Janssen

DARZALEX (daratumumab) in combination with lenalidomide and dexamethasone (Rd)

subjects with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT)

Approval granted by the FDA

Pfizer

ZIRABEV (bevacizumab-bvzr)

metastatic colorectal cancer; unresectable, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer (NSCLC); recurrent glioblastoma; metastatic renal cell carcinoma (RCC); and persistent, recurrent or metastatic cervical cancer

Approval granted by the FDA

Janssen

Genmab

Darzalex (daratumumab) in combination with lenalidomide and dexamethasone as a first-line treatment

multiple myeloma subjects who are ineligible for autologous stem cell transplant (ASCT)

Approval granted by the FDA

Alexion Pharmaceuticals

Soliris (eculizumab)

neuromyelitis optica spectrum disorder (NMOSD) in adult subjects that express a specific biomarker

Approval granted by the FDA

Teva Pharmaceuticals

1% Sodium Hyaluronate

osteoarthritis (OA) of the knee in subjects who have failed to respond adequately to conservative non-pharmacologic therapy and simple analgesics (e.g., acetaminophen)

Approval granted by the FDA

Retrophin

Thiola EC (tiopronin) 100 mg and 300 mg tablets

cystinuria

Approval granted by the FDA

Grifols

Xembify 20% subcutaneous immunoglobulin

primary immunodeficiencies

Approval granted by the FDA

 

New Pipeline Updates from Pepscan, Precigen, Inc. and Dignitana AB

July 1, 2019

Company Drug/Device Medical Condition Status
Cyxone T20K multiple sclerosis (MS) Phase I trial initiated enrolling healthy male volunteers in the Netherlands
NantKwest Inc. PD-L1 t-haNK solid tumors Phase I trial initiated
Pepscan clinical neoantigen peptides cancer Phase I trial initiated
I-Mab Biopharma TJC4 advanced malignant tumors Phase I trial initiated
Asieris Pharmaceuticals APL-1202 in combination with BCG (Bacillus Calmette Guerin) non-muscle invasive bladder cancer (NMIBC) Phase Ib trial initiated enrolling subjects in China
Precigen, Inc. PRGN-3006 UltraCAR-T relapsed or refractory acute myeloid leukemia (AML) or higher risk myelodysplastic syndrome (MDS) Phase I/Ib trial initiated
Syntrix Pharmaceuticals SX-682 in combination with KEYTRUDA (pembrolizumab) metastatic melanoma Phase I/II trial initiated enrolling 77 subjects at Massachusetts General Hospital and Dana Farber Cancer Institute
Minovia Therapeutics Mitochondrial Augmentation Therapy (MAT) Pearson syndrome Phase I/II trial initiated enrolling seven subjects
Xeris Pharmaceuticals, Inc. developmental ready-to-use glucagon Post-bariatric hypoglycemia (PBH) following bariatric surgery Phase II trial initiated enrolling 12 subjects with PBH
iLiAD Biotechnologies BPZE1 pertussis Phase IIb trial initiated
X4 Pharmaceuticals, Inc. mavorixafor (X4P-001) WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) Phase III trial initiated enrolling 28 subjects in 20 countries
XW Laboratories Inc. XWL-008 narcolepsy Orphan Drug designation granted by the FDA
Aridis Pharmaceuticals, Inc. AR-501 lung infection associated with cystic fibrosis Orphan Drug designation granted by the FDA
LifeMax Laboratories, Inc. LM-030 Netherton Syndrome Orphan Drug designation granted by the FDA
Boehringer-Ingelheim
Eli Lilly and Company
empagliflozin chronic heart failure Fast Track designation granted by the FDA
Arrowhead Pharmaceuticals Inc. ARO-AAT alpha-1 liver disease Fast Track designation granted by the FDA
ANI Pharmaceuticals, Inc. Vancomycin Hydrochloride for Oral Solution USP, 250 mg/5 ml enterocolitis caused by Staphylococcus aureus (including methicillin-resistant strains) and antibiotic-associated pseudomembranous colitis caused by C. difficile Approval granted by the FDA
Regeneron Pharmaceuticals, Inc.
Sanofi
Dupixent (dupilumab) chronic rhinosinusitis with nasal polyposis (CRSwNP) Approval granted by the FDA
Dignitana AB DigniCap Delta chemotherapy-induced hair loss Approval granted by the FDA

 

New Pipeline Updates from Boehringer Ingelheim, OxThera AB and Amgen

June 24, 2019

Company Drug/Device Medical Condition Status
Cellular Biomedicine Group Inc. anti-CD20 CAR-T diffuse large B-cell lymphoma (DLBCL) Phase I trial initiated enrolling 12 subjects in China
Boehringer Ingelheim
OSE Immunotherapeutics SA
SIRPα antagonist monoclonal antibody BI 765063 (formerly OSE-172) advanced solid tumors Phase I trial initiated
Rocket Pharmaceuticals, Inc. RP-A501 Danon disease Phase I trial initiated enrolling 12-24 pediatric and young adult male subjects
BioNTech SE
Genmab A/S
DuoBody-PD-L1x4-1BB metastatic or unresectable malignant solid tumors Phase I/IIa trial initiated
Reistone Biopharma Company SHR0302 Ulcerative Colitis Phase II trial initiated
Reistone Biopharma Company SHR0302 Crohn’s disease Phase II trial initiated
Zydus Cadila Saroglitazar Magnesium Non-Alcoholic Fatty Liver Disease (NAFLD) and Non-Alcoholic SteatoHepatitis (NASH) Phase II trial initiated enrolling 104 subjects at 20 sites across the U.S.
InflaRx N.V. IFX-1 Pyoderma Gangraenosum (PG) Phase IIa trial initiated enrolling 12 subjects with moderate to severe PG at three sites in Canada
Corvidia Therapeutics Inc. Ziltivekimab (previously known as COR-001) advanced chronic kidney disease (CKD) Phase IIb trial initiated enrolling 240 subjects at 50 sites across the U.S.
Arena Pharmaceuticals, Inc. etrasimod 2 mg ulcerative colitis (UC) Phase III trial initiated enrolling subjects with moderately to severely active UC
OxThera AB Oxabact Primary Hyperoxaluria Phase III trial initiated enrolling 22 subjects at 10 sites across Europe and the U.S.
Torque TRQ-1501 relapsed or refractory solid tumors and lymphomas that express any of five tumor-associated antigens (PRAME, WT-1, SSX2, Survivin and NY-ESO-1) Fast Track designation granted by the FDA
Bio-Techne ExoDx Prostate IntelliScore (EPI) test prostate cancer Breakthrough Device designation granted by the FDA
Camurus CAM2029 acromegaly IND approval granted by the FDA
Triumvira Immunologics, Inc. TAC01-CD19 CD19-postive B-cell malignancies IND approval granted by the FDA
Amgen
Allergan plc
KAJINTI (trastuzumab-anns) HER2-overexpressing adjuvant and metastatic breast cancer Approval granted by the FDA
Amgen
Allergan plc
KAJINTI (trastuzumab-anns) HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma Approval granted by the FDA
Novo Nordisk Victoza (liraglutide) Type 2 diabetes in children and adolescents aged 10-17 years Approval granted by the FDA
Kowa Pharmaceuticals America, Inc. LIVALO (pitavastatin) heterozygous familial hypercholesterolemia (HeFH) in subjects eight years and older Approval granted by the FDA

 

New Pipeline Updates from Genentech, Merck and Ivenix, Inc.

June 17, 2019

Company Drug/Device Medical Condition Status

Terns Pharmaceuticals, Inc.

TERN-101

non-alcoholic steatohepatitis (NASH)

Phase I trial initiated

OnKure, Inc.

OKI-179

advanced solid tumors

Phase I trial initiated

Purdue Pharma, L.P.

nalmefene hydrochloride (HCl)

opioid overdose

Phase I trial initiated

Goldfinch Bio, Inc.

GFB-887

kidney disease

Phase I trial initiated

Aerpio Pharmaceuticals, Inc.

AKB-9778

primary open angle glaucoma (POAG)

Phase Ib trial initiated enrolling 48 subjects

Adocia

ADO09

type 1 diabetes

Phase Ib trial initiated enrolling 24 subjects in Germany

Tolero Pharmaceuticals, Inc.

TP-0903

previously treated chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL)

Phase I/II trial initiated

Pharming Group N.V.

RUCONEST recombinant human C1 esterase inhibitor (rhC1INH)

pre-eclampsia

Phase I/II trial initiated

Noveome Biotherapeutics, Inc.

ST266

persistent corneal epithelial defects (PEDs)

Phase II trial initiated

Ivenix, Inc.

Ivenix Infusion System

infusion

510(k) clearance granted by the FDA

CorMatrix Cardiovascular, Inc.

Cor PATCH

cardiovascular disease

510(k) clearance granted by the FDA

Bolder Biotechnology, Inc.

BBT-059

Acute Radiation Syndrome (ARS)

Orphan Drug designation granted by the FDA

Palatin Technologies, Inc.

PL-8177

non-infectious intermediate, posterior, pan and chronic anterior uveitis

Orphan Drug designation granted by the FDA

Genentech

Rituxan (rituximab) in combination with glucocorticoids (GCC)

granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in children two years of age and older

Priority Review granted by the FDA

SpeeDx Pty. Ltd.

ResistancePlus GC

N. gonorrhea

Breakthrough Device designation granted by the FDA

Xenikos B.V.

T-Guard

steroid-refractory acute graft-versus-host disease (SR-aGVHD)

IND approval granted by the FDA

Innovative Health Solutions, Inc.

IB-Stim

irritable bowel syndrome (IBS) in patients ages 11-18

Clearance granted by the FDA

Genentech

Polivy (polatuzumab vedotin-piiq) in combination with bendamustine plus Rituxan (rituximab)

diffuse large B-cell lymphoma (DLBCL)

Accelerated approval granted by the FDA

Merck

KEYTRUDA

metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC)

Approval granted by the FDA

Senseonics Holdings, Inc.

Eversense Continuous Glucose Monitoring System

diabetes

Approval granted by the FDA

 

New Pipeline Updates from Immunovant, Minoryx Therapeutics and Bayer

June 10, 2019

Company Drug/Device Medical Condition Status
Cellenkos, Inc. CK0801 bone marrow failure syndromes, including aplastic anemia, hypoplastic myelodysplasia and primary myelofibrosis Phase I trial initiated
Xencor, Inc. XmAb22841 advanced solid tumors Phase I trial initiated
Alnylam Pharmaceuticals, Inc. ALN-AGT hypertension Phase I trial initiated enrolling 168 subjects
UCB
Neuropore Therapies, Inc.
UCB0599 Parkinson’s disease Phase Ib trial initiated
Amphera B.V. MesoPher pancreatic cancer Phase II trial initiated enrolling 10 adult subjects with surgically resected pancreatic cancer who have received adjuvant standard of care treatment
Immunovant IMVT-1401 moderate-to-severe active Graves' ophthalmopathy (GO) Phase II trial initiated enrolling eight subjects
Rejenevie Therapeutics AR-100 immune restoration therapy immunology Phase II trial initiated enrolling 20 healthy subjects 55 years of age or older in Freeport, Bahamas
Ironwood Pharmaceuticals, Inc.
Allergan PLC
MD-7246 abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) Phase II trial initiated enrolling 400 subjects
Verona Pharma PLC metered-dose inhaler formulation of ensifentrine (RPL554) moderate-to-severe chronic obstructive pulmonary disease (COPD) Phase II trial initiated enrolling 36 subjects at two sites in the UK
Minoryx Therapeutics leriglitazone (MIN-102) Friedreich’s Ataxia Phase II trial initiated enrolling 36 subjects 12 years of age or older at numerous sites across Europe
GENFIT elafibranor nonalcoholic fatty liver disease (NAFLD) Phase II trial initiated enrolling 16 subjects with NAFL as identified with magnetic resonance spectroscopy (H-MRS)
Arcutis, Inc. ARQ-151 atopic dermatitis Phase IIa trial initiated
Akero Therapeutics, Inc. AKR-001 non-alcoholic steatohepatitis (NASH) Phase IIa trial initiated enrolling 80 subjects
Ansun Biopharma, Inc. DAS181 lower respiratory tract parainfluenza virus Phase III trial initiated enrolling immunocompromised subjects at sites in the U.S., Europe and Asia
Eyenovia Inc. MicroPine pediatric progressive myopia Phase III trial initiated enrolling 400 subjects ages three to 12 years of age
Incyte pemigatinib (INCB54828) cholangiocarcinoma Phase III trial initiated enrolling 432 subjects
Allergan plc
Gedeon Richter PLC
VRAYLAR (cariprazine) manic or mixed episodes associated with bipolar I disorder in adults sNDA approval granted by the FDA
Cadent Therapeutics CAD-1883 spinocerebellar ataxia (SCA) Orphan Drug designation granted by the FDA
Bayer Aliqopa (copanlisib) marginal zone lymphoma (MZL) Breakthrough Therapy designation granted by the FDA
Imara, Inc. IMR-687 sickle cell disease Fast Track designation granted by the FDA
NovoCure NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy malignant pleural mesothelioma (MPM) Approval granted by the FDA
AveXis Inc. Zolgensma (onasemnogene abeparvovec-xioi) spinal muscular atrophy (SMA) in children less than two years of age Approval granted by the FDA
Novartis Piqray (alpelisib) tablets in combination with the endocrine therapy fulvestran hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated advanced or metastatic breast cancer in postmenopausal women and men Approval granted by the FDA
Novartis Jakafi (ruxolitinib) steroid-refractory acute GVHD in adult and pediatric subjects 12 years and older Approval granted by the FDA
Celgene Corporation REVLIMID (lenalidomide) in combination with a rituximab product (R²) previously treated follicular lymphoma (FL) or marginal zone lymphoma (MZL) in adults Approval granted by the FDA