Historical Catalyst Calendar

Biotech Stock Historical Catalysts listed from 2009.

What's included?

2009 ~ Most small/mid cap FDA Approval and Complete Response Letters.

January 2015 ~ Most small/mid cap Phase 3 data releases.

August 2016 ~ Small/mid cap Phase 2 and Phase 3 data releases.

January 2016 ~ Large cap releases added (data and regulatory).


View the BioPharmCatalyst FDA Calendar for a list of upcoming key catalysts.

We suggest you visit the BioPharmCatalyst PDUFA Calendar if your focus is on upcoming FDA regulatory events (i.e PDUFA dates and Advisory Committee Meetings).


Note: Most bankrupt or acquired companies are not included.

Key catalysts are generally inputted at the end of each trading session.


Ticker Drug Stage Catalyst
PFE LYRICA (pregabalin)
Postherpetic neuralgia (PHN)
Approved
Approval announced October 12, 2017.
MYL Glatiramer Acetate Injection - Generic for Copaxone
Multiple sclerosis (MS)
Approved
Approval announced October 4, 2017.
AGN Botox
Forehead lines
Approved
Approval (third indication) announced October 3, 2017.
AGEN Shingrix
Shingles
-
Advisory Committee Meeting September 13, 2017 voted 11-0 in favor. Shingrix, containing Agenus' adjuvant, QS-21 Stimulon.
PFE XALKORI (crizotinib)
ALK-positive advanced non-small cell lung cancer(NSCLC)
Phase 3
Phase 3 data presented at ESMO 2017 - Overall survial HR=0.760 (p=0.09)
PFE PF-05280014
Herceptin biosimilar
Phase 3
Phase 3 data presented at ESMO - risk ratio of 0.940.
INO INO-5150
Prostate cancer
Phase 1b
Phase 1b interim data presented at ESMO 2017.
UTHR Orenitram
Pulmonary arterial hypertension (PAH)
Phase 3
Phase 3 data due 2H 2018. Independent data monitoring committee (DMC) noted September 8, 2017 that the trial should continue following its interim analysis.
MRK KEYTRUDA + chemo (KEYNOTE-021)
First-Line Nonsquamous Non-small cell lung cancer (NSCLC)
Phase 2
Phase 2 data presented at ESMO 2017 - ORR 57%, PFS 19 months, HR 0.54
SGEN Tisotumab Vedotin
Cervical Cancer
Phase 1/2
Phase 1/2 data presented at ESMO 2017. Response rate 32%.
BGNE BGB-290
Solid tumors - cancer
Phase 1/2
Phase 1/2 preliminary data presented at ESMO 2017 - ORR 33%.
AMGN Tezepelumab
Asthma
Phase 2b
Phase 2b data released September 7, 2017 - primary endpoint met.
AZN Tezepelumab
Asthma
Phase 2b
Phase 2b data released September 7, 2017 - primary endpoint met.
ADMS ADS-4101
Partial onset seizures in patents with epilepsy
Phase 1b
Phase 1b top-line data released September 7, 2017.
BCRX RAPIVAB (peramivir injection)
Acute uncomplicated influenza to pediatric patients
Approved
Approved September 21, 2017.
SRPT Golodirsen - Exon 53
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2 data released September 6, 2017 - endpoints met.
SRPT Golodirsen - Exon 53
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2 late breaking poster presented at World Muscle Society October 7, 2017.
CLLS UCART123
Acute myeloid leukemia (AML) and in blastic plasmacytoid dendritic cell neoplasm (BPDCN).
Phase 1
Clinical hold announced September 4, 2017 following one fatality in the BPDCN clinical trial (ABC study).
NVS Fingolimod
Phase 3
Phase 3 trial met primary endpoint - data due 7th Joint ECTRIMS-ACTRIMS meeting October 25 - 28, 2017.
AZN Faslodex (fulvestrant)
Monotherapy for expanded use in women with HR+, HER2- advanced breast cancer
Approved
Expanded approval announced August 28, 2017.
BPMC BLU-554
Advanced hepatocellular carcinoma (HCC)
Phase 1
Updated Phase 1 presented at ESMO September 10, 2017. 16% ORR in patients with FGFR4-driven HCC. 49% had tumor reduction.
IRWD DUZALLO (LESINURAD AND ALLOPURINOL)
Hyperuricemia associated with gout
Approved
Approval announced August 21, 2017.
FENC Sodium Thiosulphate
Cisplatin-induced ototoxicity
Phase 3
Phase 3 preliminary data released September 13, 2017.
FENC Sodium Thiosulphate
Cisplatin-induced ototoxicity
Phase 3
Phase 3 trial met primary endpoint - full data released October 16, 2017.
TBPH Closed Triple - IMPACT
COPD
Phase 3
Phase 3 data released September 20, 2017 - primary endpoint met.
ABUS Patisiran APOLLO
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
Phase 3
Phase 3 top-line data released September 20, 2017 - all endpoints met. NDA filing due by end of 2017 - entitled to low to mid-single-digit royalty payments on sales of patisiran by Alnylam.
JNJ Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
Approved
Approval announced August 2, 2017.
MRK KEYNOTE-040 KEYTRUDA
Recurrent or metastatic head and neck squamous cell carcinoma (HNSCC)
Phase 3
Phase 3 trial did not meet primary endpoint - July 24, 2017. Awarded accelerated approval in 2016.
MRK LUSDUNA Nexvue
Diabetes
Approved
Tentative approval announced July 20, 2017 - subject to an automatic stay due to a lawsuit from Sanofi claiming patent infringement.
VRTX VX-659 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min)
Phase 1
Phase 1 data released July 18, 2017. ppFEV1 of 9.6 percentage points.
AMGN KYPROLIS (ASPIRE)
Relapsed Multiple Myeloma
Phase 3
Phase 3 trial met primary endpoint. HR 79%.
SVRA Aironite - INDIE
Pulmonary hypertension (PH)
Phase 2
Phase 2 interim data released July 11, 2017.
IMRN IMM-124E
Nonalcoholic steatohepatitis (NASH)
Phase 2
Phase 2 top-line data due 4Q 2017. Interim analysis July 10, 2017 showed no statistical difference with placebo.
SNY Fitusiran
Hemophilia A and B
Phase 2
Phase 2 Open-label Extension Study data released July 10, 2017 at ISTH.
SNY Fitusiran
Hemophilia A and B
Phase 2
Phase 2 trial suspended dosing due to fatal thrombotic event - noted September 7, 2017.
ALNY Fitusiran
Hemophilia A and B
Phase 2
Phase 2 Open-label Extension Study data released July 10, 2017 at ISTH.
ALNY Fitusiran
Hemophilia A and B
Phase 2
Phase 2 trial suspended dosing due to fatal thrombotic event - noted September 7, 2017.
PFE Mylotarg (gemtuzumab ozogamicin)
Acute myeloid leukemia (AML)
-
Advisory Committee Meeting July 11, 2017 voted in favor 6-1 of a favorable risk:benefit profile.
PFE Mylotarg (gemtuzumab ozogamicin)
Acute myeloid leukemia (AML)
Approved
Approval announced September 1, 2017.
BMY ORENCIA
Psoriatic Arthritis (PsA)
Approved
Approval announced July 6, 2017.
MRK KEYTRUDA - KEYNOTE-183
Multiple myeloma
Phase 3
Phase 3 clinical hold announced July 6, 2017 - also applies to KEYNOTE-185.
NBIX INGREZZA
Tardive dyskinesia
Approved
Approval for sNDA 80 mg capsules announced October 5, 2017.
STDY Trevyent
Pulmonary Arterial Hypertension (PAH)
-
Refusal to file Letter (RTF) issued August 31, 2017 in response to NDA filing.
AMGN Vectibix (Panitumumab)
Wild-Type RAS Metastatic Colorectal Cancer
Approved
sBLA approval announced June 29, 2017.
BLUE HGB-205
Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD)
Phase 1/2
Phase 1/2 data presented at EHA June 23, 2017.
PFE Epoetin alfa biosimilar
CRL
CRL issued June 22, 2017.
NVS Tafinlar (dabrafenib) and Mekinist (trametinib)
Non-small cell lung cancer (NSCLC) with BRAF V600E mutation
Approved
FDA Approval announced June 22, 2017.
NVO Victoza (liraglutide)
Type 2 diabetes
Approved
Approval announced August 25, 2017.
AMGN MVASITM (bevacizumab-awwb)
Biosimilar candidate to Avastin (bevacizumab)
Approved
Approved September 14, 2017.
JNJ ZYTIGA (abiraterone acetate) - LATITUDE
metastatic hormone-naïve prostate cancer (mHNPC)
Phase 3
Phase 3 data presented at ASCO June 3, 2017 - PFS 33 months.
BMY CM-358 – Opdivo
Cervical, vaginal and vulvar cancers
Phase 1/2
Phase 1/2 preliminary data presented at ASCO June 2, 2017. ORR=21%.
BMY BMS-986016 - LAG-3 + Opdivo
Melanoma
Phase 1/2
Phase 1/2 ORR rate of 12.5% noted at ASCO June 3, 2017.
NVS CTL019 - JULIET
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)
Phase 2
Phase 2 pivotal primary analysis due International Conference on Malignant Lymphoma (ICML) on June 14, 2017. Abstract released June 7, 2017. ORR 45%.
BMY Opdivo (CM-040)
Previously Treated Hepatocellular Carcinoma
Approved
Approved under priority review September 22, 2017.
MRK Keytruda
Cancer - Third-line Gastric or Gastroesophageal Junction Adenocarcinoma
Phase 3
Approved September 22, 2017 - under priority review.
EPIX EPI-506
Castrate resistant prostate cancer
Phase 1/2
Announced September 11, 2017 to discontinue further clinical development.
CALA CX-1158-101
Solid tumors - cancer
Phase 1
Phase 1 oral presentation at ASCO, June 5, 2017.
ZYME ZW25
HER2-expressing Cancers - ovarian, breast, gastric
Phase 1
Phase 1 initial presented at ASCO June 4, 2017.
BHVN BHV-4157 Trigriluzole
Spinocerebellar Ataxia
Phase 2/3
Phase 2b/3 negative top-line data released October 2, 2017.
VRNA RPL554
COPD
Phase 2a
Phase 2a positive data released September 7, 2017.
MRTX Sitravatinib plus nivolumab
Non-small cell lung cancer (NSCLC)
Phase 2
Phase 2 preliminary data noted 3 out of 11 patients experienced a confirmed partial response.
ABBV ABT-494 - SELECT-BEYOND
Rheumatoid arthritis
Phase 3
Phase 3 top-line released September 11, 2017. Endpoints met but with two patient deaths.
ECYT EC1169
Castrate-resistant prostate cancer (mCRPC)
Phase 1
Phase 1 data due by end of 2017. Noted June 2, 2017 decision to only assess taxane-exposed patients and ending treatment of taxane-naïve patients.
ECYT EC1456
Solid tumors (NSCLC)
Phase 1
Phase 1 enrollment to be stopped - announced June 2, 2017.
LLY Galcanezumab - EVOLVE-1, EVOLVE-2 and REGAIN
Migraine prevention
Phase 3
Phase 3 data released May 12, 2017 - primary endpoints hit in all trials. BLA filing due 2H 2017.
RARX RA101495
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2 initial data from two patients released June 27, 2017.
REGN Praluent (alirocumab)
High low-density lipoprotein (LDL) cholesterol
Approved
sBLA Approval announced April 25, 2017.
SNY Praluent (alirocumab)
High low-density lipoprotein (LDL) cholesterol
Approved
sBLA Approval announced April 25, 2017.
MTNB MAT2203
Vulvovaginal candidiasis (VVC)
Phase 2
Phase 2 top-line data released June 26, 2017. Not successful in demonstrating meaningful clinical response.
MTNB MAT2203
Refractory mucocutaneous candidiasis
Phase 2
Phase 2a open label data due released June 3, 2017 at ASM Microbe.
BGNE BGB-A317
Solid tumors
Phase 1/2
Phase 1/2 preliminary data presented at ASCO June 5, 2017.
NLNK Indoximod + Sipuleucel-T
Metastatic castration resistant prostate cancer (mCRPC)
Phase 2
Phase 2 poster presentation at ASCO June 5, 2017.
BMY Opdivo + Yervoy
Malignant pleural mesothelioma (MPM)
Phase 2
Phase 2 late breaker at ASCO June 5, 2017. Control rate 44%. ORR 19%.
BMY Epacadostat with Opdivo (nivolumab) - (ECHO-204)
Solid tumors
Phase 1/2
Phase 1/2 trial enrolling. Oral presentation at ASCO June 5, 2017.
MRK Epacadostat with Keytruda - ECHO-202/KEYNOTE-037
NSCLC, RCC, Triple-Negative Breast, Ovarian, Urothelial Carcinoma, Solid Tumors
Phase 1/2
Phase 1/2 data presented at ASCO 2017. 35% ORR noted in NSCLC arm. UC, RCC, SCCHN data also presented June 5.
MRK Epacadostat with Keytruda - ECHO-202/KEYNOTE-037
NSCLC, RCC, Triple-Negative Breast, Ovarian, Urothelial Carcinoma, Solid Tumors
Phase 1/2
Updated data at ESMO Saturday, 9 September 2017 showed ORR 56% in melanoma patients.
INCY Epacadostat with Keytruda - ECHO-202/KEYNOTE-037
NSCLC, RCC, Triple-Negative Breast, Ovarian, Urothelial Carcinoma, Solid Tumors
Phase 1/2
Phase 1/2 data presented at ASCO 2017. 35% ORR noted in NSCLC arm. UC, RCC, SCCHN data also presented June 5.
INCY Epacadostat with Keytruda - ECHO-202/KEYNOTE-037
NSCLC, RCC, Triple-Negative Breast, Ovarian, Urothelial Carcinoma, Solid Tumors
Phase 1/2
Updated data at ESMO Saturday, 9 September 2017 showed ORR 56% in melanoma patients.
TSRO Niraparib and Keytruda (TOPACIO)
Triple-negative Breast Cancer or Ovarian Cancer
Phase 1/2
Phase 1/2 enrolling. Initial data presented at ASCO investor event. ORR 4/13 patients. Further data due 2H 2017.
ABBV Veliparib
Squamous non-small cell lung cancer (NSCLC) and triple negative breast cancer (TNBC)
Phase 3
Phase 3 trial did not meet primary endpoints - April 19, 2017.
INCY Epacadostat with Opdivo (nivolumab) - (ECHO-204)
Solid tumors
Phase 1/2
Phase 1/2 trial enrolling. Oral presentation at ASCO June 5, 2017.
OTIC OTO-104 AVERTS-1
Ménière's disease
Phase 3
Phase 3 data released August 30 failed to meet primary and key secondary endpoints.
ARDX Tenapanor (T3MPO-2)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data released October 11, 2017. Primary endpoint met but with relatively high rates of diarrhea.
ZFGN ZGN-1061
Diabetes
Phase 1
Phase 1 positive safety data released May 4, 2017. Phase 2 initiation due 2H 2017.
ACHN JNJ-4178
Hepatitis C (HCV)
Phase 2a
Phase 2a ongoing. Updated data presented at EASL April 22, 2017.
ACHN JNJ-4178
Hepatitis C (HCV)
Phase 2a
Janssen noted September 11, 2017 that no further development planned.
MRK Januvia (Sitagliptin)
Type 2 Diabetes
CRL
CRL issued April 7 2017. sNDA for approved drug requested to include data on cardiovascular effects
BMY CM-142 – Opdivo
(dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC)
Approved
Approval announced August 1, 2017.
BMY CM - 067 Opdivo in combination with Yervoy
Melanoma
Phase 3
Phase 3 first overall survival (OS) positive data released April 3, 2017
AZN Tagrisso
Epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC)
Approved
Approval announced March 31, 2017.
SPPI Poziotinib
Non-small cell lung cancer (NSCLC)
Phase 2
Phase 2 initiation announced March 30, 2017 with preliminary data due released October 18, 2017 - ORR 73%.
NVO Fiasp (fast-acting insulin aspart)
Type 1 and type 2 diabetes
Phase 3
Approved September 29, 2017.
MTFB Iclaprim - REVIVE-2
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 data released October 4, 2017 - primary endpoint met. NDA filing due 1Q 2018.
MTFB Iclaprim -REVIVE-1
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 data released April 18, 2017 - primary endpoint of non-inferiority met.
AUPH Voclosporin - AURION trial
Lupus
Phase 2
48-week data from open-label AURION study presented March 27, 2017 - primary endpoint met.
AGN JUVÉDERM VOLLURE
Correction of moderate to severe facial wrinkles and folds
Approved
Approval announced March 20, 2017.
AKTX Coversin
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2 interim data released April 24, 2017. Phase 3 program to commence 4Q 2017 with initial data due 1Q 2019.
ANAB ANB020
Moderate-to-severe adult atopic dermatitis
Phase 2a
Phase 2a positive data released October 10, 2017
ESPR Bempedoic Acid/ Ezetimibe/ Atorvastatin (1002-038)
Hypercholesterolemia
Phase 2
Phase 2 data released August 8, 2017. Primary endpoint met.
GALT GR-MD-02
Plaque psoriasis
Phase 2
Phase 2 data released March 6, 2017.
MRK Odactra (MK-8237)
House dust mite allergies
Approved
Approved March 1, 2017.
PFE Avelumab
Urothelial Carcinoma
Approved
Approval announced May 9, 2017.
VRX Latanoprostene bunod
Open angle glaucoma (OAG) or ocular hypertension (OHT)
CRL
CRL issued August 7, 2017.
RNN RX-3117
Advanced bladder cancer
Phase 2a
Phase 2a initial presented at ASCO June 4, 2017.
DEPO GRALISE
Postherpetic neuralgia - shingles
Approved
Approved January 28, 2011.
JAZZ JZP-6
Fibromyalgia
CRL
CRL received October 11, 2010.
ARLZ VIMOVO
Gastric ulcers
Approved
Approved April 30, 2010.
SGEN ADCETRIS
Hodgkin lymphoma and Anaplastic large cell lymphoma
Approved
Approval announced August 19, 2011.
PCRX EXPAREL
Postsurgical pain
Approved
Approved October 31, 2011.
INCY Jakafi
Myelofibrosis
Approved
Approved November 16, 2011.
ATRS Anturol
Overactive bladder
Approved
Approved December 8, 2011.
INSY SUBSYS
Cancer pain
Approved
Approved January 5, 2012.
ALKS BYDUREON
Type 2 diabetes
Approved
Approved January 27, 2010.
ALKS BYDUREON
Type 2 diabetes
CRL
CRL received March 15, 2010.
FCSC Laviv
Wrinkles
CRL
CRL issued December 18, 2009.
FCSC Laviv
Wrinkles
Approved
Approved June 22, 2011.
PRTK Intermezzo
Insomnia
CRL
CRL issued November 2, 2009.
PRTK Intermezzo
Insomnia
Approved
Approved November 23, 2011.
GTXI Toremifene
Reduce fractures in men with prostate cancer
CRL
CRL issued November 2, 2009.
SPPI FUSILEV
Colorectal cancer
CRL
CRL received October 9, 2009.
SPPI FUSILEV
Colorectal cancer
Approved
Approved April 29, 2011.
ZGNX Sumavel
Migraine
Approved
Approved July 16, 2009.
ARDM Sumavel
Migraine
Approved
Approval granted July 16, 2009.
SPPI ZEVALIN
Non-Hodgkin’s lymphoma
CRL
CRL received July 5, 2009.
SPPI ZEVALIN
Non-Hodgkin’s lymphoma
Approved
Approved September 4, 2009.
CRIS Vismodegib
Basal Cell Carcinoma
Approved
Approved January 30, 2012.
CORT Corlux - Korlym
Cushing's Syndrome
PDUFA
Approved February 17, 2012.
PLX Taliglucerase alfa
Gaucher disease
CRL
CRL issued February 25, 2011.
PLX Taliglucerase alfa
Gaucher disease
Approved
Approved May 1, 2012.
EBS BioThrax
Anthrax Vaccine
Approved
Approved May 17, 2012.
RGEN RG1068
Detection of pancreatic duct abnormalities
CRL
CRL received June 22, 2012.
VVUS Qnexa
Obesity
CRL
October 28, 2010.
VVUS Qnexa
Obesity
Approved
Approved July 17, 2012.
ARNA Lorcaserin
Obesity
Approved
Approved June 27, 2012.
SUPN Oxtellar
Epilepsy
Approved
Approved October 22, 2012.
EXEL Cabozantinib
Medullary thyroid cancer
Approved
Approved November 29, 2012.
NAVB Lymphoseek
Lymphatic-tissue tracing agent
CRL
CRL issued September 10, 2012.
NAVB Lymphoseek
Lymphatic-tissue tracing agent
Approved
Approved March 13, 2013.
AVDL Bloxiverz (neostigmine methylsulfate)
Reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery
Approved
Approved June 3, 2013.
TBPH VIBATIV (telavancin hydrochloride) for injection
Hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP)
Approved
Approved June 21, 2013.
ABBV Ibrutinib
Relapsed or refractory MCL mantle cell lymphoma
Approved
Approved November 13, 2013.
DRRX Posidur
Post-operative pain relief
CRL
CRL issued February 12, 2014.
ABBV Imbruvica
Cancer - Chronic Lymphocytic Leukemia Who Have Received at Least One Prior Therapy
Approved
Approved February 12, 2014.
ABBV Imbruvica
Deletion 17p
Approved
Approved July 19, 2014.
ABBV VIEKIRA PAK
HCV - genotype 1
Approved
Approved December 19, 2014.
ABBV IMBRUVICA
Waldenström’s Macroglobulinemia
Approved
Approved January 29, 2015 - PCYC
AMGN Corlanor
Chronic Heart Failure
Approved
Approved April 15, 2015.
TBPH BREO ELLIPTA
Asthma
Approved
Approved April 30, 2015.
CHRS CHS-0214
Rheumatoid arthritis
Phase 3
Phase 3 data released January 2016. Endpoints met. However, commercialization not possible in US before 2029 due to patent issues
ICPT Obeticholic acid (OCA) - CONTROL
NASH patients taking statins
Phase 2
Phase 2 primary endpoint met - July 31, 2017. One patient death noted but DSMB noted unlikely to be related to OCA (company unsure).
NVS Zykadia
First-line use in patients with ALK+ metastatic NSCLC
Approved
Priority review granted February 22, 2017. Approval announced May 26, 2017.
PFE Inotuzumab Ozogamicin
Relapsed or Refractory Acute Lymphoblastic Leukemia in Adults
Approved
Approval announced August 17, 2017.
XBIT MABp1
Hidradenitis Suppurativa (HS)
Phase 2
Phase 2 data released February 16, 2017.
MYL MYL-1401H
Neulasta biosimilar
CRL
CRL issued October 10, 2017.
AGN Botox
Depression
Phase 2
Phase 2 top-line data released April 5, 2017 - primary endpoint missed. Phase 3 planned.
AGN Cariprazine
Schizophrenia/ Negative symptoms
-
Refusal to file letter issued for sNDA filing - September 22, 2017.
AGN Sarecycline
Severe acne vulgaris
Phase 3
Phase 3 data released March 27 2017 - primary endpoints met. NDA filing due 2H 2017.
SNY Patisiran APOLLO
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
Phase 3
Phase 3 top-line data released September 20, 2017 - all endpoints met. NDA filing due by end of 2017.
JNJ XARELTO (rivaroxaban)
Coronary Artery Disease or Peripheral Artery Disease
Phase 3
Phase 3 trial stopped early due to positive efficacy - February 8, 2017.
PBYI Roche (RHHBY) - Perjeta + Herceptin (APHINITY)
Adjuvant HER2+ breast cancer
Phase 3
Note this trial is conducted by competitor Roche (RHHBY), NOT by Puma. The event was likely to have a significant impact on the share price of Puma (e.g. positive data would likely have a negative impact on Puma and vice versa). Data were released March 2, 2017 - primary endpoint was met.
PBYI Roche (RHHBY) - Perjeta + Herceptin (APHINITY)
Adjuvant HER2+ breast cancer
Phase 3
Note this trial is conducted by competitor Roche (RHHBY), NOT by Puma. Primary endpoint was met March 2, 2017. Data released June 5, 2017 - HR 0.81. Disease-free-survival +0.9% over Herceptin.
MRK KEYTRUDA
First and Second line locally advanced or metastatic urothelial cancer - bladder cancer
Approved
Approved May 18, 2017.
GEMP Gemcabene - ROYAL-1
Heterozygous Familial Hypercholesterolemia (HeFH)
Phase 2b
Phase 2b data released August 7, 2017 - primary endpoint met but lower than expectations.
GEMP Gemcabene - COBALT-1
Homozygous Familial Hypercholesterolemia (HoFH)
Phase 2b
Phase 2b interim data released January 30, 2017.
GEMP Gemcabene - COBALT-1
Homozygous Familial Hypercholesterolemia (HoFH)
Phase 2b
Phase 2b data released June 28, 2017 - primary endpoint met.
PFE PF-06425090
C. difficile Infection
Phase 2
Phase 2 positive data released January 26, 2017.
NVS ACZ885 (canakinumab) - CANTOS
CV risk reduction
Phase 3
Phase 3 trial met primary endpoint - data to be presented later in 2017 at a medical congress.
NVS ACZ885 (canakinumab) - CANTOS
CV risk reduction
Phase 3
Data presented August 27, 2017 showed a 77% reduction in lung cancer mortality.
NVAX RSV F Vaccine immunogenicity trial
Respiratory Syncytial Virus (RSV) in older adults
Phase 2
Phase 2 safety and immunogenicity data released July 24, 2017 - efficacy trial to be initiated 2018.
LLY Lasmiditan - SPARTAN
Migraine
Phase 3
Phase 3 data released August 4, 2017 - endpoints met. NDA filing due 2H 2018.
VRX Brodalumab
Psoriasis
Approved
Approved February 15, 2017.
SNY Dupixent (dupilumab) - LIBERTY ASTHMA QUEST
Asthma
Phase 3
Phase 3 data released September 11, 2017 - primary endpoints met. 46% improvement after 52 weeks in 300 mg group. sBLA filing due by end of 2017.
SNY Dupixent (dupilumab)
Eosinophilic esophagitis
Phase 2
Phase 2 data released May 4, 2017.
SNY Dupixent (dupilumab)
Atopic dermatitis
Approved
Approved under priority review - March 28, 2017.
SNY Sarilumab
Rheumatoid arthritis
Approved
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
REGN REGN2222
Respiratory syncytial virus (RSV)
Phase 3
Phase 3 data released August 14, 2017 - primary endpoint not met.
REGN Dupixent (dupilumab) - LIBERTY ASTHMA QUEST
Asthma
Phase 3
Phase 3 data released September 11, 2017 - primary endpoints met. 46% improvement after 52 weeks in 300 mg group. sBLA filing due by end of 2017.
REGN Dupixent (dupilumab)
Eosinophilic esophagitis
Phase 2
Phase 2 data released May 4, 2017.
REGN Dupixent (dupilumab)
Atopic dermatitis
Approved
Approved under priority review - March 28, 2017.
REGN Sarilumab
Rheumatoid arthritis
Approved
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
PFE XTANDI (PROSPER)
Non-metastatic high risk hormone-sensitive prostate cancer
Phase 3
Phase 3 data released September 14, 2017 - primary endpoint met.
PFE Xeljanz
Psoriatic arthritis
-
PDUFA date December 2017. No exact date given. December 31, 2017 noted as last possible date using guidance of "December 31". Advisory Committee Meeting August 3, 2017 voted 10-1 in favor of recommending approval.
PFE Sutent (Sunitinib)
Renal cell carcinoma (RCC)
-
Advisory Committee Meeting September 19, 2017 voted 6-6 the benefits outweigh risks.
PFE hGH-CTP (MOD-4023)
Adults - growth hormone deficiency
Phase 3
Phase 3 data released December 30, 2016 - primary endpoint not met.
PFE Avelumab
Metastatic Merkel cell carcinoma
Approved
Priority review announced November 29, 2016. Approval announced March 23, 2017.
PFE IBRANCE (palbociclib)
HR+, HER2- Metastatic Breast Cancer
Approved
sNDA acceptance announced December 21, 2016. Approval announced March 31, 2017.
NVS RELAX-AHF-2 (RLX030)
Acute heart failure
Phase 3
Phase 3 data released March 22, 2017 - primary endpoints not met.
NVS RTH258 (brolucizumab)
Neovascular AMD
Phase 3
Phase 3 data released June 20, 2017 - primary and key secondary endpoints met.
NVS Kymriah (CTL019)
Relapsed/Refractory B-Cell Acute lymphoblastic leukemia
-
Advisory Committee Meeting July 12, 2017 voted 10-0 recommending approval.
NVS Kymriah (CTL019)
Relapsed/Refractory B-Cell Acute lymphoblastic leukemia
Approved
PDUFA date under priority review announced March 29, 2017. Approval announced early - August 30, 2017.
NVS Rydapt (Midostaurin - PKC412)
Acute myeloid leukemia (AML)
Approved
Priority review granted November 14, 2016. Approval announced April 28, 2017.
NVS LEE011
HR+/HER2- advanced breast cancer
Approved
Priority review granted November 1, 2016. Approval announced March 13, 2017.
NVO Semaglutide (NN9536)
Obesity
Phase 2
Phase 2 data released June 23, 2017 with Phase 3 trial to commence 2018.
NVO Semaglutide
Type 2 diabetes
-
Advisory Committee Meeting October 18, 2017 voted 16-0 in favor.
NVO Nonacog beta pegol
haemophilia B
Approved
Approval announced May 31, 2017.
MRK MK-8931 (017) - Verubecestat
Mild-to-moderate Alzheimer's
Phase 3
Phase 3 trial stopped due to lack of efficacy - February 14, 2017.
MRK Keytruda - KEYNOTE-045
Bladder Cancer
Phase 3
Phase 3 trial met its primary endpoint - October 2016. Updated data presented at ASCO 2017. HR 0.70. OS 10.3 months.
MRK Anacetrapib (MK-0859)
Raising HDL-C and reducing LDL-C
Phase 3
Phase 3 data due released June 27, 2017. Primary endpoint met.
MRK Anacetrapib (MK-0859)
Raising HDL-C and reducing LDL-C
Phase 3
Phase 3 data due released June 27, 2017. Primary endpoint met but noted August 29, 2017 that key secondary endpoint was not met.
MRK Anacetrapib (MK-0859)
Raising HDL-C and reducing LDL-C
-
Phase 3 data due released June 27, 2017. Primary endpoint met but noted August 29, 2017 that key secondary endpoint was not met. Announced October 11, 2017 that it will not seek regulatory approval.
MRK Keytruda
First-Line Treatment of Metastatic Non-Squamous Non-Small Cell Lung Cancer
Approved
Approved May 10, 2017.
MRK KEYTRUDA
Relapsed or Refractory Classical Hodgkin Lymphoma
Approved
Approval announced March 14, 2017.
MRK KEYTRUDA
Microsatellite Instability-High Cancer
Approved
PDUFA date March 8, 2017 extended to June 9, 2017 due to submission of extra data. Approved May 23, 2017.
MRK RENFLEXIS - SB2 (infliximab biosimilar)
Biosimilar candidate of Remicade
Approved
BLA acceptance announced May 23, 2016 by partner Samsung Bioepis. Approval annuonced April 21, 2017.
MRK ISENTRESS
HIV-1
Approved
Approval announced May 30, 2017.
LLY Ramucirumab
Second-line bladder cancer
Phase 3
Phase 3 PFS data released May 31, 2017 - primary endpoint met. Final OS results are currently expected in mid-2018.
LLY Abemaciclib - MONARCH 3
HR+, HER2-locoregionally recurrent or metastatic breast cancer (MBC)
Phase 3
Phase 3 interim analysis April 24, 2017 met primary endpoint. Detailed data due 2H 2017 with regulatory filings due 3Q 2017.
LLY Abemaciclib - MONARCH 1 and 2
Second-line hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer and HR+, HER2- breast cancer
Phase 3
Phase 3 data released March 20, 2017 - PFS primary endpoint met.
LLY Abemaciclib - JUNIPER
Thrid line KRAS mutation positive Non-small cell lung cancer (NSCLC)
Phase 3
Phase 3 data released October 10, 2017 - primary endpoint not met
LLY Baricitinib
Rheumatoid arthritis
CRL
Announced January 19 2016 that NDA Filing had been submitted. Three month delay announced January 13, 2017. CRL received April 14, 2017.
JNJ INVOKANA CANVAS
Type 2 diabetes
Phase 3
Cardiovascular Outcomes trial data released June 12, 2017 - 14% reduction in the risk of the composite primary endpoint of CV mortality.
JNJ Imetelstat - IMbarkStudy
Myelofibrosis
Phase 2/3
Phase 2 clinical hold lifted Nov 2014. Phase 2 initiated September 2015. Internal review conducted September 2016. Low dose discontinued, high dose enrollment suspended. Further review conducted April 2017 - further development pending FDA feedback and longer term data.
JNJ STELARA (USTEKINUMAB)
Plaque psoriasis
Approved
Approval announced October 13, 2017.
JNJ Guselkumab
Plaque psoriasis
Approved
Approval announced July 13, 2017.
JNJ Sirukumab
Rheumatoid arthritis
-
BLA filing announced September 23, 2016. PDUFA estimate September 23, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.
JNJ Sirukumab
Rheumatoid arthritis
CRL
CRL issued September 22, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.
JNJ DARZALEX (Daratumumab) + pomalidomide and dexamethasone
Relapsed or refractory multiple myeloma
Approved
Approval announced June 16, 2017 - note: submitted by Janssen Biotech, Inc.
JAZZ Xyrem
Pediatric narcolepsy patients with cataplexy
Phase 2/3
Phase 2/3 positive data released April 24, 2017. sNDA filing due 4Q 2017.
JAZZ JZP-110
Excessive sleepiness associated with obstructive sleep apnea
Phase 3
Phase 3 data released March 20, 2017 - primary endpoints met.
JAZZ JZP-110
Excessive sleepiness associated with narcolepsy
Phase 3
Phase 3 data released April 26, 2017. NDA to be filed late 2017.
JAZZ Vyxeos
Acute myeloid leukemia
Approved
Approval announced August 3, 2017.
INVA FF/UMEC/VI
Chronic obstructive pulmonary disease (COPD)
Approved
Approval announced September 18, 2017.
GSK Mepolizumab
Chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype
Phase 3
Phase 3 METREX trial met primary endpoint. Phase 3 METREO failed to meet endpoint.
GSK Shingrix
Shingles
-
Advisory Committee Meeting September 13, 2017 voted 11-0 in favor. PDUFA estimate October 24, 2017.
GSK FF/UMEC/VI
Chronic obstructive pulmonary disease (COPD)
Approved
Approval announced September 18, 2017.
GSK Benlysta (belimumab) - subcutaneous
Systemic lupus erythematosus (SLE)
Approved
Approval announced July 21, 2017.
GILD Bictegravir/F/TAF
HIV
Phase 3
Phase 3 data released May 30 - primary endpoint met. NDA filing due 2Q 2017.
GILD Vosevi (Sofosbuvir/Velpatasvir/Voxilaprevir)
Hepatitis C virus (HCV)
Approved
Approval announced July 18, 2017.
CELG GED-0301 (CD-002)
Crohn’s disease
Phase 3
Phase 3 trial to discontinue due to lack of efficacy - noted October 19, 2017.
CELG Ozanimod - RADIANCE
Relapsing Multiple Sclerosis
Phase 3
Phase 3 data due released May 22, 2017 - primary endpoint met, disability endpoint not met.
CELG Demcizumab plus carboplatin and pemetrexed - DENALI
First-line non-squamous Non-small cell lung cancer (NSCLC)
Phase 2
Announced November 1, 2016 that it has discontinued further enrollment. Phase 2 data released May 8, 2017 - endpoints not met.
CELG Demcizumab plus Abraxane - YOSEMITE
First-line pancreatic cancer
Phase 2
Phase 2 data released April 10, 2017 - primary endpoint not met.
CELG JCAR017
Relapsed/refractory B cell non-Hodgkin lymphoma - cancer
Phase 1/2
Pivotal trial to be initiated in 2017. ASCO Poster Session June 5, 2017 - ORR 86%.
CELG Durvalumab - FUSION
Relapsed and/or refractory multiple myeloma (RRMM), myelodysplastic syndromes and AML
Phase 1/2
Phase 1/2 MM trials placed on clinical hold - September 7, 2017. AML/MDS trials to continue.
CELG IDHIFA (enasidenib) - AG-221
Relapsed/Refractory IDH2 mutant-positive Acute Myeloid Leukemia -
Approved
Approval announced August 1, 2017.
CELG REVLIMID
Multiple myeloma (MM) - autologous stem cell transplant (ASCT)
Approved
Approved February 22, 2017.
BMY CM-602 – Opdivo + Elo + SOC
Myeloma
Phase 3
Noted September 6, 2017 that trial has been placed on partial hold.
BMY CM-238 – Opdivo (Adjuvant)
Melanoma
Phase 3
Phase 3 interim data released June 6, 2017 - primary endpoint met. Data presented at ESMO September 10, 2017 - HR 0.65 compared to Yervoy.
BMY CM-214 – Opdivo + Yervoy
First-line Renal cell carcinoma (RCC)
Phase 3
Phase 3 data released August 15, 2017 - one of two co-primary endpoints met.
BMY CM-214 – Opdivo + Yervoy
First-line Renal cell carcinoma (RCC)
Phase 3
Phase 3 trial stopped early due to clear efficacy.
BMY CM-143 – Opdivo
Second-line Glioblastoma (GBM) cancer
Phase 3
Phase 3 data released April 3, 2017 - primary endpoint not met.
AZN Tralokinumab (STRATOS1)
Severe, uncontrolled asthma
Phase 3
Phase 3 trial did not meet primary endpoint - May 10, 2017.
AZN Tagrisso - FLAURA
First-line lung cancer
Phase 3
Phase 3 data released July 27, 2017 - PFS improvement noted.
AZN Tagrisso - FLAURA
First-line lung cancer
Phase 3
Late breaker at ESMO September 9, 2017 showed PFS 18.9 months compared with 10.2 SOC.
AZN Lynparza - SOLO 2
Second-line ovarian cancer
Phase 3
Data presented March 14, 2017 showed significant PFS benefit.
AZN Lynparza - SOLO 2
Second-line ovarian cancer
Approved
Approval announced August 17, 2017.
AZN Lynparza
Breast cancer
Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met.
AZN Lynparza
Breast cancer
Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met. Late breaker at ASCO June 4, 2017 showed HR of 0.58 (42% reduction of risk of disease progression or death).
AZN Durvalumab +/- tremelimumab (MYSTIC)
Lung cancer
Phase 3
Phase 3 data released July 27, 2017 - primary endpoint not met.
AZN Durvalumab (PACIFIC)
Lung cancer
Phase 3
Phase 3 interim analysis released May 12, 2017 - PFS primary endpoint already met. OS endpoint to be assessed.
AZN Durvalumab (PACIFIC)
Lung cancer
Phase 3
Late breaker at ESMO September 9, 2017 showed PFS 16.8 months v 5.6 months for placebo.
AZN Bydureon
Type 2 Diabetes
Phase 3
Phase 3 Cardiovascular Outcome trial data released September 14, 2017 - primary efficacy objective of a superior reduction in MACE missed statistical significance (p=0.061).
AZN Urothelial carcinoma - Bladder cancer
Bladder cancer
Approved
BLA acceptance announced December 9, 2017. PDUFA under priority review. Approval announced May 1, 2017.
AZN ZS-9
Hyperkalaemia
CRL
CRL issued May 26, 2016.
AZN ZS-9
Hyperkalaemia
CRL
Second CRL issued March 17, 2017.
AZN Saxagliptin and dapagliflozin
Type-2 diabetes
Approved
Approval announced February 28, 2017.
AMGN Repatha
Cardiovascular disease
Phase 3
Phase 3 cardiovascular outcomes study data released March 17, 2017 - endpoints met but down on expectations.
AMGN BLINCYTO
Ph+ R/R ALL
Approved
PDUFA date under priority review August 14, 2017. Approval announced July 11, 2017.
AMGN Parsabiv
Secondary hyperparathyroidism (SHPT)
Approved
Approved February 7, 2017.
AMGN EVENITY (Romosozumab)
Osteoporosis
Phase 3
PDUFA date July 19, 2017. However, likely to be delayed due to safety data from ARCH trial released May 22, 2017.
AMGN EVENITY (Romosozumab)
Osteoporosis
CRL
CRL issued July 16, 2017.
AGN ESMYA (ulipristal acetate)
Uterine Fibroids
Phase 3
Phase 3 data released January 18, 2017 - endpoints met.
AGN AVYCAZ (ceftazidime and avibactam)
Complicated Urinary Tract Infections (cUTI)
Approved
sNDA approval announced January 30, 2017.
AGN Oxymetazoline HCl cream 1.0%
Facial Erythema (Redness) Associated with Rosacea
Approved
Approved January 19, 2017.
AGN Linaclotide 72 mcg dose for use in the treatment of adults
Chronic idiopathic constipation (CIC)
Approved
sNDA approval announced January 26, 2017.
AGN MVASITM (bevacizumab-awwb)
Biosimilar candidate to Avastin (bevacizumab)
Approved
Approved September 14, 2017.
ABBV Venclexta (MURANO)
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
Phase 3
Phase 3 data released September 18, 2017 - PFS primary endpoint met.
ABBV Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
Approved
Approval announced August 2, 2017.
ABBV ABT-494 (upadacitinib) - SELECT-NEXT
Rheumatoid arthritis
Phase 3
Phase 3 top-line data released June 7, 2017 - primary endpoints met.
ABBV ABT-494
Crohn’s Disease
Phase 2
Phase 2 positive data at DDW Meeting May 9, 2017. Phase 3 trial to be initiated later in 2017.
ABBV ABT-494
Atopic Dermatitis
Phase 2b
Phase 2b data released September 7, 2017 - primary endpoint met.
ABBV Imbruvica
Marginal zone lymphoma
Approved
sNDA filing under priority review approved January 19, 2017.
ABBV Glecaprevir/Pibrentasvir (G/P)
Hepatitis C virus (HCV)
Approved
Approval announced August 3, 2017.
DVAX SD-101 + Pembrolizumab
Melanoma - cancer
Phase 1/2
Phase 1b/2 poster at ASCO June 3, 2017. Noted June 2 ORR of 7/7 in anti-PD-1/L1-naïve patients
NTRP Bryostatin
Alzheimer's disease
Phase 3
Phase 2 data released May 1, 2017 - primary endpoint not met.
NBIX INGREZZA
Tourette syndrome - juvenile
Phase 2
Phase 2 data released May 23 - primary endpoint not met.
HALO Subcutaneous rituximab
Various blood cancers
Approved
Approval announced June 22, 2017.
LIFE Resolaris
Early onset facioscapulohumeral muscular dystrophy (FSHD)
Phase 1/2
Phase 1/2 final data presented October 5, 2017 at the International Annual Congress of the World Muscle Society (WMS).
CTIC PIXUVRI - PIX306 Trial
B-cell non-Hodgkin lymphoma
CRL
CRL received April 9, 2010.
ACOR Ampyra
Improve walking in patients with multiple sclerosis
Approved
Approved January 22, 2010.
ACOR Ampyra
Improve walking in patients with multiple sclerosis
-
Decision on Inter Partes Review (IPR) released March 10, 2017 - patents upheld.
PIRS PRS-080
Anemia of chronic disease
Phase 1b
Phase 1b data released at ERA-EDTA June 4, 2017.
AMPH Naloxone Intranasal
Opioid overdose
CRL
CRL announced February 21, 2017.
AMPH Primatene Mist (epinephrine inhalation aerosol)
Asthma
CRL
CRL issued December 27, 2016.
NDRM ND0612H (Trial 006)
Severe Parkinson's Disease
Phase 2
Phase 2 trial met key primary and secondary endpoints - March 1, 2017.
LXRX Sotagliflozin
Type 1 Diabetes
Phase 2
Phase 2 trial did not meet primary endpoint.
EGLT OXAYDO
Acute and chronic moderate to severe pain
CRL
CRL announced June 20, 2017.
SCYX Intravenous SCY-078
Phase 1
Clinical hold placed on the initiation of intravenous (IV) SCY-078 until the FDA completes a review of all data. Clinical hold follows three mild-to-moderate thrombotic events in healthy volunteers - announced March 2, 2017.
XBIT 514G3
Staphylococcus Aureus Bacteremia
Phase 1/2
Phase 1/2 top-line data released April 3, 2017. 4 patient deaths noted.
BTX Renevia
Restoring normal skin contours in for patients with HIV
-
Pivotal trial data released June 14, 2017 - primary endpoint met.
VRTX Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - Two Copies of the F508del Mutation
Phase 3
Phase 3 data released March 28, 2017 - primary endpoint met. NDA filing due 3Q 2017.
ALDX ADX-102
Dry eye syndrome
Phase 2a
Phase 2a data released September 11, 2017 - noted statistically and clinically significant activity.
AZRX MS1819-SD
Exocrine pancreatic insufficiency (EPI) caused by chronic pancreatitis (CP)
Phase 2
Phase 2a data from first six patients released September 27, 2017. CFA improvement of 21% noted in highest dose.
BLUE bb2121
Relapsed/refractory multiple myeloma
Phase 1
Phase 1 interim data released November 30, 2016 - 78% ORR. Pivotal trial to be initiated 2017. Updated data at ASCO June 5, 2017 - ORR 89%.
RETA Bardoxolone methyl - CARDINAL
Chronic Kidney Disease Caused by Alport Syndrome
Phase 2/3
Phase 2/3 trial initiation announced February 23, 2017. Data from Phase 2 part of trial released July 24, 2017.
EGRX Ryanodex
Exertional heat stroke (EHS)
CRL
CRL issued July 26, 2017. Additional trial required.
NEOS NT-0201
Attention deficit hyperactivity disorder (ADHD)
Approved
Approval announced September 15, 2017.
HALO FOLFIRINOX and PEGPH20
Pancreatic and gastric cancers
Phase 1/2
Phase 1/2 enrollment closed due to lack of efficacy.
PBYI Neratinib
HER2-positive metastatic breast cancer patients with brain metastases
Phase 2
Phase 2 data released at ASCO June 3, 2017.
EXEL Esaxerenone (CS-3150)
Essential hypertension
Phase 3
Phase 3 trial met primary endpoint - September 24, 2017. Japanese regulatory filing due 1Q 2018.
VRTX VX-440 in combination with tezacaftor and ivacaftor
Cystic fibrosis who have one F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation
Phase 2
Phase 2 data released July 18, 2017. ppFEV1 of 12.0 percentage points.
VRTX VX-152 in combination with tezacaftor and ivacaftor
Cystic fibrosis who have one F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation
Phase 2
Phase 2 data released July 18, 2017. ppFEV1 of 9.7 percentage points.
TENX Levosimendan
Septic shock
Phase 2b
Phase 2b trial did not meet primary endpoint.
PRTA PRX003
Psoriatic Arthritis
Phase 1b
Phase 1b data released September 28, 2017 - insufficient meaningful clinical effect for further development.
NOVN SB206
Human papillomavirus (HPV)
Phase 2
Phase 2 data released November 29, 2016. Primary endpoint met.
NOVN SB208
Fungal infections
Phase 2
Phase 2 data released April 12, 2017. Primary endpoint met.
NOVN SB204
Acne vulgaris
Phase 3
Phase 3 top-line data released January 27, 2017. Met one of three co-primary endpoints in NI-AC301 trial. Met all endpoints in NI-AC302 trial.
VRTX KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
Approved
Approved December 29, 2014.
SCMP Amitiza/Lubiprostone
Opioid-induced bowel dysfunction
Approved
Approved April 23, 2013.
IMGN Kadcyla
2nd-line HER2+ Metastatic Breast Cancer
Approved
Approved February 22, 2013 - under priority review.
HEB Ampligen
CFS
CRL
CRL issued December 1, 2009.
HEB Ampligen
CFS
CRL
CRL February 4, 2013.
CPRX Firdapse
MuSK-antibody positive myasthenia gravis (MuSK-MG)
Phase 2
Phase 2 top-line data met endpoints - March 15, 2017. Pivotal trial planned.
MDCO Inclisiran
Hypercholesterolemia
Phase 2
Phase 2 Day 180 data presented at AHA Scientific Sessions on November 15, 2016.
KDMN KD025
Chronic graft-versus-host disease (cGVHD)
Phase 2
Phase 2 interim data presented July 11, 2017. ORR 71%.
GLPG GLPG1690 - FLORA
Idiopathic pulmonary fibrosis
Phase 2a
Phase 2a top-line data released August 10, 2017. Disease progression halted. Further trials planned.
ZYNE ZYN002
Fragile X syndrome
Phase 2
Phase 2 data released September 28, 2017. Company noted data were positive with primary endpoint met. Note though that trial was uncontrolled/open-label.
ZYNE ZYN002 - STOP
Osteoarthritis
Phase 2
Phase 2 data released August 14, 2017 - primary endpoint not met. However, company still intends to initiate Phase 3 trial pending discussions with FDA.
NEOT LIPO-202
Reduction of localized fat deposits under the chin (submental fat)
Phase 2
Phase 2 data released June 26, 2017 - no efficacy shown.
VRTX Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - One Copy of the F508del Mutation and a Second Mutation that Results in Residual CFTR Function
Phase 3
Phase 3 data released March 28, 2017 - primary endpoint met. NDA filing due 3Q 2017.
PRTK Omadacycline - oral
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 data released July 17, 2017 - all endpoints met.
BLUE LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)
Phase 3
Phase 3 early data released at EHA June 23, 2017.
EGRX EP-3101 (bendamustine RTD)
Chronic lymphocytic leukemia; Indolent non-Hodgkin's lymphoma - cancer
Approved
Tentative approval July 2 2014. Teva has also received orphan drug and related pediatric exclusivity expiring in September 2015 and May 2016 for the CLL and NHL indications, respectively.
LPTX CureXcell
Diabetic Foot Ulcers
Phase 3
Phase 3 trial did not meet primary endpoint.
INCY Baricitinib
Rheumatoid arthritis
CRL
Announced January 19 2016 that NDA Filing had been submitted. Three month delay announced January 13, 2017. CRL received April 14, 2017.
CHMA Mycapssa
Acromegaly
CRL
CRL April 15, 2016.
NVIV Neuro-Spinal Scaffold
Complete Thoracic AIS A Spinal Cord Injury
Phase 3
Noted July 31, 2017 that enrollment has been temporarily halted as it discusses potential changes to patient enrollment criteria due to patient deaths - enrollment now due to be completed 1H 2018 with Humanitarian Device Exemption (HDE) application due 2H 2018.
TGTX TG-1101 - ULTIMATE I
Multiple Sclerosis
Phase 2
Phase 2 preliminary results presented at American Academy of Neurology (AAN) annual meeting April 28, 2017.
SAGE SAGE-217
Major Depressive Disorder (MDD)
Phase 2
Phase 2 top-line data from open-label trial released February 13, 2017
SAGE SAGE-217
Parkinson’s disease (PD)
Phase 2
Phase 2 initiation announced December 13, 2016. Data from Part A data released May 9, 2017. Part B data due 2H 2017.
CLVS Rucaparib ARIEL3
Ovarian cancer patients with a BRCA-like mutation
Phase 3
Phase 3 data released June 20 2017 - primary endpoint met. sNDA filing due 3Q or early 4Q 2017.
OPK hGH-CTP
Adults - growth hormone deficiency
Phase 3
Phase 3 data released December 30, 2016 - primary endpoint not met.
VICL ASP0113 Vaccine
Kidney transplant recipients
Phase 2
Phase 2 data released September 19, 2016 - did not meet primary endpoint.
GTXI Enobosarm
Breast cancer - advanced AR+ TNBC
Phase 2
Noted August 14, 2017 that trial will not continue due to insufficient clinical benefit response (CBR).
LIFE Resolaris
Limb girdle muscular dystrophy (LGMD) 2B / Adult patients with facioscapulohumeral muscular dystrophy (FSHD)
Phase 1/2
Phase 1b/2 data released December 13, 2016.
ZSAN M207
Migraine
Phase 3
Phase 3 trial data due released February 13, 2017 - primary endpoints met.
TTPH Eravacycline (TP-434) - IGNITE3
cUTI - complicated urinary tract infections
Phase 3
Phase 3 trial did not meet endpoints September 8, 2015.
ICPT Obeticholic acid (OCA)
Primary biliary cirrhosis (PBC) - POISE
Approved
Approved May 27 2016.
AGRX Twirla
Contraceptive patch
Phase 3
CRL issued 2013. Phase 3 top-line data released January 3, 2017 - poor data but intends to reflile NDA 1H 2017.
CORI Corplex Donepezil
Alzheimer's disease
-
Pilot bioequivalence data due released May 11, 2017. Pivotal trial to commence fall of 2017 with preliminary data due February 2018.
NKTR Cipro DPI
Bronchiectasis
Phase 3
Phase 3 second trial did not meet endpoint - April 5, 2017.
NVCR Tumor Treating Fields (TTFields) - INNOVATE
Ovarian cancer
Phase 2
Phase 2 data presented December 12, 2016. Further promising PFS data released March 31, 2017.
NLNK Indoximod in combination with KEYTRUDA
Melanoma - cancer
Phase 2
Phase 2 interim analysis released at AACR meeting April 4, 2017.
NLNK Indoximod in combination with KEYTRUDA
Melanoma - cancer
Phase 2
Updated data released September 7, 2017 showed improvement in Complete Response (CR) to 20% (10/51 patients) compared to CR of 12% (6/51 patients).
OCUL DEXTENZA
Allergic conjunctivitis
Phase 3
Phase 3 trial did not meet primary endpoint - June 6, 2016.
TGTX TG-1101 and TGR-1202 - UNITY-DLBCL study
Diffuse large B-cell lymphoma (DLBCL) cancer
Phase 2b
Phase 2b enrollment commenced June 2016. First interim analysis August 10, 2017 determined that enrollment in TGR-1202 arm will stop but continue in TG-1101 + TGR-1202 arm. TGR-1202 arm replaced by combo of TG-1101, TGR-1202, and bendamustine.
IRWD Linaclotide 72 mcg dose for use in the treatment of adults
Chronic idiopathic constipation (CIC)
Approved
sNDA approval announced January 26, 2017.
ONCE SPK-8011
Hemophilia A
Phase 1/2
Phase 1/2 promising initial data released from first two patients - August 2, 2017. 11% and 14% Factor VIII.
PRTK Omadacycline
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 top-line data met endpoints - June 16, 2016
PTCT Ataluren
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
-
Received a Refusal to File letter for their NDA Filing - February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. New PDUFA date October 24, 2017. Advisory Committee Meeting September 28, 2017 voted that although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective.
LXRX Sotagliflozin
Type 1 Diabetes
Phase 3
Phase 3 data from second trial released December 21, 2016 - primary endpoint met.
LXRX Sotagliflozin
Type 1 Diabetes
Phase 3
Phase 3 data released September 9, 2016 met primary endpoint. Secondary endpoint data due May 2017. Data from second trial released December 21, 2016 - primary endpoint met. Data from third trial also met primary endpoint - June 9, 2017.
AMRN Vascepa REDUCE-IT outcomes trial
High Triglycerides With Mixed Dyslipidemia
Approved
Approved July 26, 2012.
AMRN Vascepa REDUCE-IT outcomes trial
High Triglycerides With Mixed Dyslipidemia
-
Interim analysis announced September 12, 2016 - trial to proceed. Second interim analysis August 14, 2017 also recommended trial to continue. Final data due 2Q or 3Q 2018.
FLXN Zilretta - FX006
Osteoarthritis of the knee
Approved
Approval announced October 6, 2017.
KURA Tipifarnib
Relapsed or refractory peripheral T-cell lymphoma
Phase 2
Phase 2 data presented at ICML conference on June 14, 2017 - 3/18 partial responses.
KURA Tipifarnib
HRAS mutant solid tumors - cancer
Phase 2
Phase 2 ongoing data released March 6, 2017 - further data due 2H 2017.
KURA Tipifarnib
HRAS mutant solid tumors - cancer
Phase 2
Phase 2 trial met primary endpoint prior to completion of enrollment.
SELB SEL-212
Tophaceous gout
Phase 2
Phase 2 dosing commenced late October 2016. Initial data released at EULAR June 15, 2017.
RGLS RG-101
Hepatitis C virus (HCV)
Phase 2
Clinical hold placed June 2016. Noted June 12, 2017 that it plans to discontinue program.
XOMA XOMA 213
Hyperprolactinemia
Phase 2
Phase 2 proof-of-concept results released April 24, 2017.
ANTH Blisibimod
IgA nephropathy
Phase 2
Previously announced that Phase 2 primary endpoint not met following interim analysis. Trial to continue as a Phase 2 trial (initially planned as a Phase 2/3 trial). Top-line data released August 28, 2017.
PTIE Remoxy
Chronic pain
CRL
CRL received June 24, 2011.
PTIE Remoxy
Chronic pain
CRL
CRL received September 26, 2016.
IPCI Rexista
Pain relief
CRL
CRL issued September 25, 2017. Advisory Committee Meeting July 26, 2017 had previously voted 22-1 against recommending approval.
TXMD Yuvvexy
Moderate-to-severe vaginal pain
CRL
CRL announced May 8, 2017. Company noted that FDA cited a lack of long-term endometrial safety data.
GILD Axicabtagene ciloleucel - KTE-C19 (ZUMA-3)
Adult patients with r/r ALL - cancer
Phase 1
Phase 2 data due 2018. Phase 1 data released at ASCO June 5, 2017. Complete remission 73%.
GILD Yescarta (axicabtagene ciloleucel)
Refractory Non-Hodgkin's Lymphoma (NHL) - cancer
Phase 2
Phase 2 pivotal top-line data released September 26, 2016. Primary analysis due 1Q 2017. BLA filing due late 2016.
GILD Yescarta (axicabtagene ciloleucel)
Refractory Non-Hodgkin's Lymphoma (NHL) - cancer
Phase 2
Phase 2 primary analysis released February 28, 2017 showed trial met primary endpoint. 31% CR for DLBCL.
GILD Yescarta (axicabtagene ciloleucel)
Refractory Non-Hodgkin's Lymphoma (NHL) - cancer
Approved
Approved October 18, 2017 (acquired from Kite Pharma).
MYOK MYK-461 - PIONEER-HCM
Hypertrophic cardiomyopathy (HCM)
Phase 2
Phase 2 data released August 7, 2017 - primary endpoint met.
EIGR Lonafarnib - LOWR HDV
Chronic hepatitis delta virus (HDV)
Phase 2
Phase 2 end of trial data released April 21, 2017. Meeting with FDA scheduled for 4Q 2017.
QURE AMT-060
hemophilia B
Phase 1/2
Phase 1/2 long term updated data presented July 10, 2017.
BMRN BMN 270
Hemophilia A
Phase 1/2
Phase 1/2 updated data released July 11, 2017. 97% reduction in Annualized Bleed Rate (ABR).
ALKS Aristada
Schizophrenia
Approved
Approved October 5 2015. sNDA filed for 2-month option with approval announced June 6, 2017.
N91115
Cystic Fibrosis - two copies of the F508del mutation
Phase 2
Phase 2 data released November 28, 2016. Primary endpoint not met.
LXRX Telotristat etiprate (XERMELO)
Carcinoid Syndrome
Approved
Approved February 28, 2017.
AAAP Lutathera
Inoperable progressive midgut NETs
CRL
PDUFA date under priority review was December 28, 2016. CRL issued December 21, 2016.
TSRO Rolapitant - intravenous (IV)
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
CRL
CRL issued January 11 2017.
SGYP Trulance (plecanatide)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data from first trial met primary endpoint - December 9, 2016.
SGYP Trulance (plecanatide)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data from second trial met primary endpoints - December 9 and 22, 2016.
RDUS TYMLOS (Abaloparatide)
Osteoporosis
Approved
PDUFA date originally scheduled for March 30 2017 delayed by three months to June 30, 2017. Approval announced April 28, 2017.
ENDP OPANA ER
Management of pain
-
Advisory Committee Meeting March 14, 2017 voted 18 to eight that the benefits of reformulated OPANA ER no longer outweigh its risks.
CEMP Solithera - intravenous
Community-acquired bacterial pneumonia (CABP)
CRL
CRL announced December 29, 2016. Further trials required.
ARQL ARQ 087
intrahepatic cholangiocarcinoma (iCCA)
Phase 2
Phase 2 data at ASCO June 3, 2017 - ORR 21%. Phase 3 trial planned for 3Q 2017.
ATRS OTREXUP
Rheumatoid arthritis (RA)
Approved
Approved Oct 14, 2013.
AUPH Voclosporin - AURORA
Lupus
Phase 2b
Phase 2b data released August 2016. Primary endpoint met but with high number of deaths.
AUPH Voclosporin - AURORA
Lupus
Phase 2b
Phase 2b data released August 2016. Primary endpoint met but with high number of deaths. Further data released March 1, 2017 - no additional deaths and endpoints met.
AVEO TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer
CRL
CRL issued June 10, 2013.
AVEO TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer
Phase 3
Phase 3 futility analysis released October 6, 2017 - trial to continue as planned.
AVIR BTA585
Respiratory syncytial virus (RSV)
Phase 2a
Phase 2a data released February 1, 2017 - primary endpoint not met.
AVIR Vapendavir - SPIRITUS trial
Human rhinovirus (HRV)
Phase 2b
Phase 2b top-line data released February 13, 2017 - primary endpoint not met.
AXON Nelotanserin
Visual hallucinations in subjects with Lewy body dementia
Phase 2
Phase 2 preliminary data released February 13, 2017. Primary endpoint hit, secondary endpoints missed. Full study to be completed mid-2017. Phase 3 trial to be initiated 2H 2017.
AXON Intepirdine - MINDSET
Alzheimer's disease
Phase 3
Phase 3 data released September 26, 2017 - primary endpoints not met.
BCRX BCX7353 - APeX-1
Reduce or eliminate attacks in HAE patients
Phase 2
Phase 2 interim data released February 27, 2017.
BCRX BCX7353 - APeX-1
Reduce or eliminate attacks in HAE patients
Phase 2
Phase 2 interim data released February 27, 2017. Top-line data released May 25, 2017.
BCRX RAPIVAB (peramivir)
INFLUENZA ACUTE
Approved
Approved December 22, 2014.
BDSI Clonidine gel
Painful diabetic neuropathy (PDN)
Phase 2b
Phase 2b data released December 13, 2016 - primary endpoints not met.
BDSI BEMA Buprenorphine/Naloxone (BNX) - BUNAVAIL
Opioid dependence
Approved
Approved June 6, 2014.
BLCM BPX-501 - BP-004
Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer
Phase 1/2
Phase 1/2 update June 23, 2017.
BLPH INOpulse delivery device
Pulmonary Hypertension associated with Idiopathic Pulmonary Fibrosis (PH-IPF)
Phase 2
Phase 2 initiated May 2016. Data presented at American Thoracic Society (ATS) Conference, May 21 2017. Phase 2b planned.
BLUE Lenti-D - Starbeam
Childhood cerebral adrenoleukodystrophy (CCALD)
Phase 2/3
Phase 2/3 trial initiated October 2013. Interim top-line data released June 26, 2017. Further data released October 4, 2017 - 88% free of free of major functional disabilities.
BMRN Vimizim (GALNS)
(MPS IVA) Morquio A Syndrome
Approved
Approved February 14, 2014.
BPMX BPX-01
Acne
Phase 2b
Phase 2b data released May 3, 2017 - primary endpoint met, secondary missed.
BTX OpRegen
Dry age-related macular degeneration (AMD)
Phase 2
Phase 2 second cohort underway. Interim data presented at May 8, 2017 at ARVO conference.
BVXV M-001
Universal flu vaccine
Phase 2b
Phase 2b data released July 20, 2017 - endpoints met.
CAPR CAP-1002 HOPE-Duchenne
Duchenne Muscular Dystrophy (DMD)
Phase 1/2
Phase 1/2 data released April 25, 2017. Noted improvement in certain measures of cardiac and upper limb function. 12-month data due 4Q 2017.
CAPR CAP-1002 HOPE-Duchenne
Duchenne Muscular Dystrophy (DMD)
Phase 1/2
Phase 1/2 data to be presented October 6, 2017.
CAPR CAP-1002 ALLSTAR
Myocardial infarction (heart attack)
Phase 2
Phase 2 data released May 12, 2017 - primary endpoint unlikely to be met.
CARA IV CR845
Acute pain
Phase 3
Interim conditional power analysis conducted June 21, 2017 - trial completion due 4Q 2017.
CARA CR845
Osteoarthritis (OA)
Phase 2b
Phase 2b data released June 29, 2017 - trial failed.
CATB Edasalonexent (CAT-1004)
Duchenne muscular dystrophy (DMD)
Phase 1/2
Phase 1/2 top-line data released January 31 - primary endpoint not met.
CATB Edasalonexent (CAT-1004)
Duchenne muscular dystrophy (DMD)
Phase 1/2
Phase 1/2 data from open-label extension trial presented October 4, 2017 - deemed sufficient to proceed to Phase 3 development.
CBAY Seladelpar
Primary biliary cholangitis (PBC)
Phase 2
Phase 2 top-line data released late May 2016. Positive interim data from second trial data released July 17, 2017.
CDTX CD101 Topical - RADIANT
Vulvovaginal candidiasis (VVC)
Phase 2
Phase 2 data released February 21, 2017 - efficacy not evident, Development to be discontinued.
CEMP Solithromycin
NASH
Phase 2
Phase 2 interim data released September 30, 2016.
CEMP Solithromycin
NASH
Phase 2
Noted February 28, 2017 that development has been suspended due to lack of efficacy.
CEMP Taksta - Fusidic acid
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 data released February 24, 2017 - endpoints met.
CERC CERC-301
Major depressive disorder (MDD)
Phase 2
Phase 2 top-line data released November 29, 2016 failed to meet primary endpoint.
CHRS CHS-1420
Psoriasis
Phase 3
Phase 3 top-line data met endpoints August 2016. Full data due released January 10, 2016.
CLSN GEN-1 OVATION
Ovarian cancer
Phase 1b
Phase 1b interim data released at ASCO 2017 - ORR 86%.
CLVS Rociletinib
Mutant EGFR T790M-positive lung cancer
CRL
CRL June 28, 2016. Phase 3 TIGER-3 also terminated.
CMRX Brincidofovir - Advise trial
Adenovirus Infection
Phase 3
Phase 3 interim analysis released May 2016. Final data released February 22, 2017.
CRBP Anabasum (Resunab)
Cystic Fibrosis
Phase 2
Phase 2 data released March 30, 2017 - primary endpoint met but some concerns regarding efficacy.
CRIS CUDC-907
Diffuse-large B-cell lymphoma (DLBCL)
Phase 2
Phase 2 initial data released August 3, 2017 - ORR 19%.
CRVS CPI-444
Solid tumors - cancer
Phase 1b
Phase 1/1b preliminary data released November 2016. Further update at ASCO June 5, 2017.
CVM Multikine
Head and Neck cancer
Phase 3
Initiated Dec 2010. Announced partial clinical hold September 27, 2016. Full clinical hold noted May 2017. Clinical hold released - August 14, 2017.
CYTR Aldoxorubicin with doxorubicin
Soft tissue sarcomas refractory cancer
Phase 3
Phase 3 PFS data released July 2016 showed no significant improvement in PFS. Second analysis released November 29, 2016 showed improvement in population with leiomyosarcoma and liposarcoma.
CYTX ECCS-50
Scleroderma
Phase 3
Pivotal trial data released June 24, 2017 - endpoints not met.
DCTH Melphalan Hepatic Delivery System (Melphalan HDS)
Hepatocellular carcinoma (HCC) - cancer
CRL
CRL issued September 13, 2017.
DEPO SEFELSA
Hot flashes
CRL
CRL issued May 31, 2013.
DERM Cimzia
Moderate-to-severe plaque psoriasis
Phase 3
Phase 3 data from first trial released October 2016 - endpoints met. Second trial data released December 8, 2016 - endpoints met. Data from third and final trial met primary endpoint - January 19, 2017.
DMTX DTX101
hemophilia B
Phase 1/2
Phase 1/2 initial data released January 31, 2017.
PLXP Locilex
Mild infections of diabetic foot ulcers
Phase 3
Phase 3 top-line data did not meet endpoints - October 25, 2016 - TICKER CHANGED FROM DPRX FOLLOWING MERGER
DRRX POSIMIR - PERSIST 3
Post-operative pain relief
Phase 3
CRL Feb 12 2014. Phase 3 commenced November 2015 in response to CRL. Noted October 19, 2017 that primary endpoint was not met.
DVAX HEPLISAV-B
Hepatitis B
CRL
CRL issued February 25, 2013.
DVAX HEPLISAV-B
Hepatitis B
CRL
CRL November 14, 2016
DVAX HEPLISAV-B
Hepatitis B
-
Advisory Committee Meeting July 28, 2017 voted 11 -1 in favor of recommending approval.
EBIO Isunakinra (EBI-005)
Moderate to severe dry eye disease
Phase 3
Phase 3 top-line data showed endpoints not met May 18, 2015.
EBS BioThrax
Anthrax Vaccine Adsorbed
Approved
Approved November 24, 2015.
EBS Anthrax Immune Globulin Intravenous (Human) [AIGIV]
Anthrax
PDUFA
Approved March 25, 2015.
Polymyxin B Hemoperfusion (PMX)
Endotoxemic septic shock
Phase 3
Phase 3 trial failed to meet endpoints.
EGRX KANGIO (bivalirudin injection)
Percutaneous Coronary Intervention (PCI) and Percutaneous Transluminal Coronary Angioplasty (PTCA)
CRL
CRL March 18 2016. FDA requested further characterization of bivalirudin-related substances in the drug product.
EGRX Docetaxel Injection
Breast, non-small cell lung, prostate gastric adenocarcinoma, and head and neck cancer
Approved
Approved December 24, 2015.
EGRX Ryanodex
Malignant hyperthermia - cancer
Approved
Approved July 23, 2014 under priority review.
ENDP BEMA Buprenorphine
Management of pain severe enough to require daily, around-the-clock, long-term opioid treatment for which alternative treatment options are inadequate
Approved
Approved October 26, 2015.
ENDP AVEED (testosterone undecanoate) intramuscular injection
Men diagnosed with hypogonadism
CRL
CRL issued September 5, 2013.
ENDP AVEED (testosterone undecanoate) intramuscular injection
Men diagnosed with hypogonadism
Approved
Approved March 6, 2014.
ENTA VIEKIRA PAK
HCV - genotype 1
Approved
Approved December 19, 2014.
EXEL Cabozantinib - CABOSUN
First-Line RCC - cancer
Phase 2
Late breaker at ESMO September 2017 showed PFS 8.6 months versus 5.3 months for sunitinib. HR 0.48
EXEL XL184 cabozantinib (CELESTIAL)
Advanced hepatocellular cancer (HCC)
Phase 3
Phase 3 data released October 16, 2017 - primary endpoint met. sNDA filing due 1Q 2018.
EXEL CABOMETYX (cabozantinib)
Metastatic renal cell cancer (RCC)
Approved
Approved April 25, 2016.
EXEL Vemurafenib
BRAF V600 Mutation-Positive Advanced Melanoma - Cancer
Approved
Approved November 10, 2015.
FCSC FCX-007
Recessive dystrophic epidermolysis bullosa
Phase 1/2
Phase 1/2 initial data from three patients released September 26, 2017.
FGEN Roxadustat
Anemia in chronic kidney disease
Phase 3
Phase 3 data from China trial released January 30, 2017. Primary endpoints met.
AVDL Akovaz
Hypotension
Approved
Approved May 2, 2016.
AVDL VAZCULEP (phenylephrine hydrochloride)
Clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia
Approved
Approved June 30, 2014.
FOLD SD-101
Epidermolysis Bullosa (EB)
Phase 3
Phase 3 top-line data released September 13, 2017 - endpoints not met.
FOLD ATB200
Pompe Disease
Phase 1/2
Phase 1/2 additional data released February 15, 2017. Further data released May 15, 2017.
FOLD ATB200
Pompe Disease
Phase 1/2
Phase 1/2 additional data released February 15, 2017. Further data released May 15, 2017 and October 4, 2017 - update expected 1H 2018 regarding regulatory path forward.
FOMX FMX101 - FX2017-22
Acne
Phase 3
Phase 3 data released March 27, 2017 - three of four primary endpoints met across two trials.
FPRX Cabiralizumab (FPA008)
Pigmented Villonodular Synovitis (PVNS)
Phase 1/2
Phase 1/2 initial data released at ASCO June 4, 2017.
FPRX FP-1039
Mesothelioma cancer
Phase 1b
Data at ESMO showed ORR 48%.
GALE NeuVax (E75) - PRESENT
Low-to-intermediate HER2+ breast cancer
Phase 3
Phase 3 trial terminated due to poor efficacy.
GBT GBT440 HOPE-KIDS 1
Sickle cell disease - children age 6-17.
Phase 1/2
Phase 2a initiated June 2016. Preliminary data released at European Hematology Association (EHA) - June 23, 2017.
GERN Imetelstat - IMbarkStudy
Myelofibrosis
Phase 2/3
Phase 2 clinical hold lifted Nov 2014. Phase 2 initiated September 2015. Internal review conducted September 2016. Low dose discontinued, high dose enrollment suspended. Further review conducted April 2017 - further development pending FDA feedback and longer term data.
GLYC GMI-1271
Acute myeloid leukemia (AML) newly diagnosed and refractory - cancer
Phase 2
Phase 2 enrollment completed - announced May 31, 2017. Data updates at ASCO June 5, 2017.
GNCA GEN-003
Genital herpes
Phase 2b
Phase 2b virologic efficacy data released September 2016.
GNCA GEN-003
Genital herpes
Phase 2b
Phase 2b data released January 5, 2017.
GNCA GEN-003
Genital herpes
Phase 2b
Phase 2b data released January 6, 2017. 12-month data released July 24, 2017. Phase 3 to commence 4Q 2017.
GNCA GEN-003
Genital herpes
-
Announced September 25, 2017 that development will be ceased.
GWPH Sativex
Cancer pain
Phase 3
Phase 3 trials did not meet endpoints.
HALO PEGPH20
Pancreatic cancer
Phase 2
Primary endpoint met.
HALO PEGPH20
Pancreatic cancer
Phase 2
Phase 2 data released January 5, 2017. Oral presentation of Phase 2 data at ASCO June 4, 2017 showed ORR of 45%.
HALO HYQVIA
Subcutaneous treatment for patients with primary immunodeficiency (PI)
CRL
CRL issued August 1, 2012.
HALO HYQVIA
Subcutaneous treatment for patients with primary immunodeficiency (PI)
Approved
Approved September 12, 2014.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
CRL
CRL issued March 19, 2010.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
CRL
CRL March, 28 2013.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
Approved
CRL Mar 28 2013. Approved August 10, 2016.
HTBX HS-410
Bladder cancer
Phase 2
Phase 2 trial did not meet primary endpoint - November 30, 2016.
HZNP ACTIMMUNE
Friedreich's Ataxia (FA)
Phase 3
Phase 3 data released December 8, 2016 - primary endpoint not met
IDRA IMO-2125
Cancer - melanoma
Phase 1/2
Phase 1/2 data released September 10, 2017. 6/9 disease control with one CR. Phase 3 trial to be initiated 1Q 2018.
IMDZ CMB305
Soft tissue sarcomas
Phase 2
Phase 2 data at ASCO June 2, 2017.
IMDZ CMB305
Soft tissue sarcomas
Phase 2
Phase 2 data at ESMO September 2017. PFS 2.6 months, Disease Control Rate 61%.
IMGN Kadcyla GATSBY trial
Cancer - metastatic HER2+ gastric cancer
Phase 3
Phase 3 endpoint not met - October 22, 2015.
IMUC ICT-107 dendritic cell vaccine
Glioblastoma antigens and cancer stem cells
Phase 3
Phase 2b failed to meet primary endpoint. Phase 3 initiated dosing June 2016 - noted June 21, 2017 that further enrollment to be suspended.
INCY Jakafi (ruxolitinib) (RESPONSE)
Polycythemia Vera
Approved
Approved December 4, 2014.
VSTM Duvelisib - DUO
Cancer - relapsed/refractory chronic lymphocytic leukemia (CLL)
Phase 3
Phase 3 data released September 6, 2017 - primary endpoint met.
INNL Cogenzia
Diabetic foot ulcer infections
Phase 3
Phase 3 data did not meet endpoints - November 3, 2016.
INSY Dronabinol Oral Solution
AIDS-related anorexia
Approved
Announced approval July 5, 2016.
IONS Inotersen (IONIS-TTRRx)
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Phase 3
Phase 3 data released May 15, 2017 - primary endpoint met but with safety concerns.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Phase 3
Phase 3 ENDEAR trial met primary endpoint August 1, 2016.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Phase 3
Phase 3 CHERISH data met primary endpoint - November 7, 2016.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Approved
Approved December 23, 2016.
AKCA Volanesorsen - APPROACH
Familial chylomicronemia syndrome (FCS)
Phase 3
Phase 3 data released March 6, 2017 - primary endpoint met. Relative safety concerns noted.
IPXL IPX066
Parkinson's
CRL
CRL issued January 21, 2013.
IPXL IPX066
Parkinson's
Approved
Approved January 8, 2015.
IRWD Linaclotide Colonic Release
Irritable Bowel Syndrome with Constipation (IBS-C)
Phase 2b
Phase 2b data released December 22, 2016.
ITCI Lumateperone
Schizophrenia
Phase 3
Phase 3 trial failed - September 28, 2016.
ITEK Trabodenoson and latanoprost
Glaucoma
Phase 2
Phase 2 trial failed - noted July 7, 2017.
JUNO JCAR017
r/r B cell non-Hodgkin lymphoma - cancer
Phase 1/2
Pivotal trial to be initiated in 2017. ASCO Poster Session June 5, 2017 - ORR 86%.
KERX Auryxia
Hyperphosphatemia
Approved
Approved Sept 5, 2014.
KMDA KamRAB
Prophylaxis of rabies disease
Approved
Approval announced August 25, 2017.
KMDA Inhaled formulation of AAT
AATD - Alpha-1 Antitrypsin deficiency
Phase 2
Top-line data released August 30, 2016. Primary endpoint met
KPTI Selinexor - SEAL
Dedifferentiated liposarcoma
Phase 2/3
Phase 2/3 trial initiated January 2016.Topline data for the Phase 2 portion released September 20, 2017 - PFS HR 0.60. Phase 3 data due 2019.
KPTI Selinexor - STORM
Quadruple Refractory Multiple Myeloma
Phase 2
Phase 2 positive top-line data released September 6, 2016
KPTI Selinexor - SADAL
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)
Phase 2b
Phase 2b additional interim data released April 4, 2017 at AACR. Final top-line data due mid-2018.
KPTI Selinexor SOPRA
Cancer - Relapsed/Refractory Acute Myeloid Leukemia (AML)
Phase 2
Data Safety Monitoring Board (DSMB) noted March 2, 2017 that trial will not meet primary endpoint.
IOVA LN-144
Refractory metastatic melanoma
Phase 2
Phase 2 second cohort commenced enrollment - May 19, 2017. Initial data presented at ASCO June 5, 2017.
LJPC LJPC-501
Hypotension
Phase 3
Phase 3 data released February 27, 2016 - primary endpoint hit. Secondary endpoints not mentioned. NDA filing due 2H 2017.
LJPC LJPC-501
Hypotension
Phase 3
Phase 3 data released February 27, 2016 - primary endpoint hit. Secondary endpoints not mentioned. Data released May 22, 2017 confirmed primary endpoint was met but with lack of mortality improvement.
LOXO Larotrectinib (LOXO-101)
Solid tumors that harbor a TRK fusion.
Phase 2
Announced 76% ORR at ASCO - June 3, 2017. Primary analysis by the end of 2017 with NDA filing late 2017 or early 2018.
LPCN LPCN 1021
Men with low testosterone (Low T)
CRL
CRL issued June 29, 2016.
LPCN LPCN 1021
Men with low testosterone (Low T)
-
CRL issued June 2016. Flexibility trial data announced June 19, 2017 - primary endpoint met with NDA fiing due 3Q 2017.
MACK MM-302 HERMIONE
Cancer - HER2-positive locally advanced or metastatic breast cancer
Phase 2
Phase 2 trial discontinued December 21, 2016 due to poor efficacy.
MACK ONIVYDE
Cancer - second line pancreatic
Approved
Approved October 22, 2015.
MCRB SER-109 - ECOSPOR III
Recurrent C. Difficile infection
Phase 2
Phase 2 interim data did not meet primary endpoint.
MCRB SER-287
Mild-to-Moderate Ulcerative Colitis
Phase 1b
Phase 1b data released October 2, 2017 - did not perform better than placebo.
MDCO Meropenem-vaborbactam
Complicated urinary tract infections (cUTI)
Phase 3
Phase 3 data met endpoints June 27, 2016.
MDCO Meropenem-vaborbactam
Complicated urinary tract infections (cUTI)
Approved
Approved August 29, 2017.
MDCO Cangrelor
Reduction of thrombotic cardiovascular events including stent thrombosis in patients with coronary artery disease undergoing percutaneous coronary intervention (PCI)
CRL
CRL April 30 2014.
MDCO RAPLIXA
Hemostasis
Approved
Approved April 30, 2015.
MDCO IONSYS
Acute postoperative pain
Approved
Approved April 30, 2015.
MDCO Oritavancin
ABSSSI
Approved
Approved August 6, 2014 under priority review
MESO MPC-150-IM
Chronic heart failure (CHF)
Phase 3
Noted April 10, 2017 that interim analysis has been completed - trial to continue as planned.
MNKD AFREZZA
Type 1/2 diabetes
CRL
CRL issued March 15, 2010.
MNKD AFREZZA
Type 1/2 diabetes
CRL
CRL received January 19, 2011.
MNKD AFREZZA
Type 1/2 diabetes
Approved
Approved June 27, 2014.
MNTA Generic Copaxone
Multiple sclerosis
Approved
Approved April 16 2015.
MRNS Ganaxolone
Focal onset seizures
Phase 3
Phase 3 data did not meet endpoints - June 13, 2016
NAVB Lymphoseek
Head and Neck Cancer
Approved
Approved June 13, 2014.
NBIX INGREZZA
Tourette syndrome - adults
Phase 2
Phase 2 data released January 18, 2017 did not meet primary endpoint.
NBRV Lefamulin - LEAP 1
Moderate to severe Community Acquired Bacterial Pneumonia (CABP)
Phase 3
Phase 3 data released September 18, 2017 - primary endpoints met.
NEOS Adzenys XR-ODT
Attention deficit hyperactivity disorder (ADHD)
Approved
Approved January 27, 2016.
NEOS Cotempla XR-ODT
Attention deficit hyperactivity disorder (ADHD)
Approved
Approval announced June 19, 2017.
NEOT LIPO-202
Reduction of central abdominal bulging
Phase 3
Phase 3 endpoints not met December 14, 2015.
NKTR NKTR-181
Lower back pain
Phase 3
Phase 3 data released March 20, 2017 - endpoints met.
NKTR NKTR-214
Solid tumors - cancer
Phase 1/2
Phase 1/2 initial data released at ASCO June 5, 2017.
NKTR Etirinotecan pegol NKTR-102 (BEACON)
Cancer - Metastatic Breast Cancer
Phase 3
Phase 3 topline data mid March 17, 2015 did not reach primary endpoint.
NKTR ADYNOVATE
Hemophilia A
Approved
Approved November 16, 2015.
NKTR MOVANTIK (Naloxegol)
Opioid-induced constipation (OIC)
Approved
Approved September 16, 2014.
NLNK HyperAcute Pancreas - IMPRESS
Surgically resected pancreatic cancer
Phase 3
Phase 3 failed to meet primary endpoint - May 9, 2016.
NVAX RSV vaccine - Resolve trial
Respiratory Syncytial Virus (RSV) in older adults
Phase 3
Phase 3 released September 15, 2016 did not demonstrate efficacy.
NYMX NX-1207 Fexapotide
BPH
Phase 3
Phase 3 endpoints met in extension trial July 2015.
OCRX OCR-002 (IV)
Hepatic encephalopathy
Phase 2b
Phase 2b data released January 30, 2017 - primary endpoint not met.
OCUL DEXTENZA
Ocular inflammation and pain following cataract surgery
CRL
CRL July 25 2016 - due to manufacturing deficiencies.
OCUL DEXTENZA
Ocular inflammation and pain following cataract surgery
CRL
CRL July 25 2016 - due to manufacturing deficiencies. NDA resubmitted, PDUFA date July 19, 2017. Further CRL issued July 11, 2017 with FDA noting deficiencies in manufacturing processes and analytical testing.
OMED Demcizumab plus Abraxane - YOSEMITE
First-line pancreatic cancer
Phase 2
Phase 2 data released April 10, 2017 - primary endpoint not met.
OMED Demcizumab plus carboplatin and pemetrexed - DENALI
First-line non-squamous Non-small cell lung cancer (NSCLC)
Phase 2
Announced November 1, 2016 that it has discontinued further enrollment. Phase 2 data released May 8, 2017 - endpoints not met.
OMED Tarextumab - PINNACLE trial
Small cell lung cancer
Phase 2
Phase 2 data released April 17, 2017 - primary endpoint not met.
OMER Omidria
Cataract surgery
Approved
Approved June 2, 2014.
ONCE SPK-9001
Hemophilia B
Phase 1/2
Phase 1/2 updated data at ASH 2016. Further update released April 6, 2017.
ONCE SPK-9001
Hemophilia B
Phase 1/2
Phase 1/2 updated data released July 10, 2017. 99% fall in infusion rate.
ONCE Luxturna (voretigene neparvovec)
Inherited retinal dystrophies
-
Advisory Committee Meeting October 12, 2017 voted 16-0 in favor of recommending approval.
OPHT Fovista
Wet age-related macular degeneration (Wet-AMD)
Phase 3
Phase 3 data released December 12, 2016 - primary endpoint not met.