PIPELINE WATCH: Phase III Trials Start In Atypical HUS, Fabry Disease And Colorectal Cancer
Executive Summary
Pipeline Watch has been updated to bring greater depth and detail to a potential therapeutic product’s progress through the R&D pipeline. The table brings you a snapshot of selected late-stage clinical trial events in the pharma and biotech industries.
The table records clinical developments using data from Informa Pharma Intelligence’s Biomedtracker. It lists drugs for which an important event was recorded between October 21 and October 27, 2016, divided by event type.
Events can include Phase II and Phase III trial initiations, development discontinuations, and the release of top-line and full clinical trial results. A brief summary of product approvals is also included.
A companion resource, The Pink Sheet’s regulatory-focused FDA Performance Tracker, follows regulatory milestones such as new submissions and user fee calculations, complete response letters and breakthrough therapy designations.
Lead company/partner |
Compound |
Indication |
Comments |
Phase III Suspended |
|||
Locilex (pexiganan) cream |
diabetic foot ulcer infections |
OneStep-1 and -2; did not meet primary endpoint. |
|
VGX-3100 |
cervical dysplasia |
FDA clinical hold on proposed trial due to concerns about delivery device. |
|
durvalumab |
head and neck cancer |
FDA partial clinical hold due to bleeding events. New patient enrolment halted. |
|
Phase III Results |
|||
Xarelto (rivaroxaban) |
treating and preventing venous thromboembolism |
Real world data confirms safety and efficacy. |
|
Latuda (lurasidone) |
schizophrenia in adolescents |
Safe and effective, supplemental NDA submitted. |
|
Okambi (lumacaftor,ivacaftor) and Kalydeco (invacaftor) |
cystic fibrosis |
They modify progression in long-term data. |
|
brincidofovir |
adenovirus infection |
AdVise; viremia declined in blood cell transplant patients. |
|
Descovy (emtricitabine and tenofovir alafenamide) |
HIVAIDS |
Switching from Truvada (emtricitabine and tenofovir disoproxil fumarate) |
|
Phase III Interim/Top-line Results |
|||
Keytruda (pembrolizumab) |
advanced bladder cancer |
KeyNote-045; improved overall survival. |
|
AstraZeneca PLC |
Lynparza (olaparib) |
ovarian cancer |
|
biosimilar adalimumab (BI 695501) |
rheumatoid arthritis |
||
plasma-derived plasminogen |
plasminogen deficiency |
US BLA filing to start within weeks. |
|
Taltz (ixekizumab) |
psoriatic arthritis |
SPIRIT-P2; positive results, 2017 supplemental filings. |
|
Lucentis (ranibizumab) |
polypoidal choroidal vasculopathy |
EVEREST II; an additional indication. |
|
Daiichi Sankyo Co. Ltd./Eli Lilly & Co. |
Effient (prasugrel) |
ischemic stroke |
PRASTO-1 and 2; an additional indication in Japanese patients. |
Rhopressa (netarsudil) ophthalmic solution |
glaucoma, ocular hypertension |
Rocket 4; achieves endpoint, NDA to be resubmitted. |
|
Shingrix vaccine |
shingles |
ZOSTER-004; supports flexible dosing, and using with flu vaccine. |
|
Gilead Sciences Inc. |
sofosbuvir, velpatasvir and voxilaprevir |
hepatitis C |
POLARIS-1,2,3,4; once daily single tablet. |
revefenacin |
chronic obstructive pulmonary disease |
Met primary endpoint and well tolerated. |
|
Phase III Initiated |
|||
ALXN1210 |
atypical hemolytic uremic syndrome and paroxysmal nocturnal hemoglobinuria |
A longer-acting anti-C5 antibody, given every eight weeks. |
|
Traumakine (FP-1201-lyo) |
acute respiratory distress syndrome |
A European study is already underway. |
|
KRN23 |
pediatric X-linked hypophosphatemia |
A fully human IgG1 Mab. |
|
PRX-102 |
Fabry disease |
A modified human alpha-GAL-A protein. |
|
napabucasin |
colorectal cancer |
The CanStem303C study. |
|
Phase II Suspended |
|||
CRS-207, ADU-623 |
ovarian, brain, and pancreatic cancer |
||
GlaxoSmithKline PLC |
losmapimod |
chronic obstructive pulmonary disease |
Development stopped by company. |
Phase II Results |
|||
MIN-101 |
schizophrenia, negative symptoms |
Six-month extension study; improved symptoms. |
|
avacopan (CCX168), a complement inhibitor |
C3 glomerulopathy |
A patient with this rare disease responded. |
|
maribavir |
CMV infection |
Reduced viremia in transplant patients. |
|
oliceridine |
pain |
Safe and effective. |
|
Opdivo (nivolumab) |
heavily pretreated classical Hodgkin’s lymphoma |
CheckMate-205; safe and effective in an expanded cohort. |
|
Phase II Completed |
|||
Samumed |
SM04554 |
androgenetic alopecia |
Safe and effective. |
Phase II Interim/top-Line Results |
|||
apatorsen; an antisense heat shock protein 27 production inhibitor |
metastatic bladder cancer |
Borealis-2; positive survival results. |
|
sotagliflozin |
type 1 diabetes |
inTandem4; confirms Phase III dose. |
|
Aerosurf (lucinactant) |
respiratory distress syndrome |
In premature infants. |
|
nolasiban (OBE001); oral oxytocin antagonist |
in vitro fertilization |
IMPLANT; proceeding to Phase III in Europe. |
|
GLPG1837 |
cystic fibrosis |
SAPHIRA 2; well tolerated |
|
relamorelin, a ghrelin agonist |
diabetic gastroparesis |
Safe and effective, in diabetes patients. |
|
axalimogene filolisbac |
cervical cancer |
Increased overall survival rate |
|
Viaskin Peanut |
peanut allergy |
OLFUS-VIPES; two year follow-up showed durable responses. |
|
Gilead Sciences Inc. |
selonsertib (GS-4997) |
non-alcoholic steatohepatitis (NASH), pulmonary arterial hypertension, diabetic kidney disease |
Showed antifibrotic activity. |
Phase II Initiation |
|||
ONCOS-102/GM-CSF gene therapy |
malignant mesothelioma |
Increases tumor infiltrating lymphocytes. |
|
SEL-212 |
gout |
In refractory patients. |
|
Stealth BioTherapeutics |
elamipretide |
congestive heart failure |
The IDDEA-HF study. |
ACT-541468 |
insomnia |
In adult patients. |
|
Theranexus |
THN102 |
narcolepsy |
Being compared with modafinil. |
Sanifit |
SNF472 |
calciphylaxis |
An orphan disease. |
Source: Informa Pharma Intelligence’s Biomedtracker.
Lead Company |
Partner Company |
Drug |
Indication |
Market |
Comments |
REGULATORY APPROVAL |
|||||
Merck & Co |
- |
Zinplava (bezolotoxumab) |
recurrence of Clostridium difficile-associated diarrhea |
US |
|
Apricus Biosciences Inc. |
Vitaros (alprostadil) cream |
erectile dysfunction |
Greece |
Launch expected in first half of 2017. |
|
SUPPLEMENTAL REGULATORY APPROVAL |
|||||
Merck & Co |
- |
Keytruda (pembrolizumab) |
first line non-small cell lung cancer |
US |
For tumors with a high PD-L1 expression and no EGFR or ALK tumor aberrations. |
- |
Cosentyx (secukinumab) |
psoriatic arthritis, ankylosing spondylitis |
Switzerland |
Targets IL-17. |
Source: Biomedtracker.