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
NCNA NUC-1031 (Acelarin)
Front-Line Treatment of Advanced Biliary Tract Cancer
Phase 1b
Data from open label Phase 1b trial released at ASCO GI 2018 - Partial Response 3/8, Stable Disease 1/8.
OPTN XHANCE
Nasal polyps
Approved
Approval announced September 18, 2017 (prior to IPO).
MRK KEYTRUDA - EORTC1325/KEYNOTE-054
Melanoma
Phase 3
Phase 3 data released January 8, 2018 met primary endpoint.
ABBV ABT-494 - SELECT-MONOTHERAPY
Rheumatoid arthritis
Phase 3
Phase 3 data released December 20, 2017 - primary endpoint and key secondary endpoints met.
PFE IXIFI
Remicade (infliximab) biosimilar
Approved
Approval announced December 13, 2017.
TRIL TTI-621
Relapsed/Refractory Diffuse Large B-cell Lymphoma (DLBCL)
Phase 1
Phase 1a/b updated data released at ASH 2017 - 5/18 objective response rate.
SNY Admelog (insulin lispro injection)
Diabetes
Approved
Approval announced December 11, 2017.
PFE Avelumab
Gastric cancer
Phase 3
Phase 3 data released November 28, 2017 - primary endpoint not met.
PHMMF Zepsyre(PM1183)
Ovarian cancer
Phase 3
Phase 3 data released January 19, 2018. Primary endpoint was not met.
BPMC Avapritinib (BLU-285)
Systemic mastocytosis (SM)
Phase 1
Phase 1 updated data due at ASH 2017 showed ORR 72%. Further trial to be initiated 1H 2018.
NKTR NKTR-214 + OPDIVO (nivolumab) - PIVOT-02
Melanoma, Renal Cell Carcinoma and Non-Small Cell Lung Cancers
Phase 1/2
Phase 1/2 promising data presented at SITC November 2017. 63% response rate for melanoma patients. 46% for Renal Cell Carcinoma.
FATE FATE-NK100
Acute myelogenous leukemia (AML)
Phase 1
Phase 1 initial data released at SITC November 10, 2017.
BMY BMS-986205 and Nivolumab
Advanced tumors
Phase 1/2
Late-breaker at SITC November 11, 2017. ORR 14-32%.
AXON Intepirdine
Gait and Balance Impairments in Dementia
Phase 2
Phase 2 data released January 2018 - negative data.
PFE LYRICA (pregabalin)
Postherpetic neuralgia (PHN)
Approved
Approval announced October 12, 2017.
INFI IPI-549 + Nivolumab
Solid tumors
Phase 1
Presentation at SITC November 10, 2017.
ONCS ImmunoPulse IL-12
Melanoma
Phase 2
SITC presentation - November 10, 2017. Updated data showed 57% PFS rate at 15 months.
CELG Ozanimod - SUNBEAM
Relapsing Multiple Sclerosis
Phase 3
Phase 3 data due released Feb 17, 2017 - primary endpoint met, disability endpoint not met. Full data released October 27, 2017 at ECTRIMS meeting.
RYTM Setmelanotide
Bardet-Biedl Syndrome
Phase 2
Preliminary Phase 2 data due released October 31, 2017. 4 of 5 patients showed weight loss of 10-12%.
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.
PFE Staphylococcus aureus vaccine
-
Advisory Committee Meeting held November 7, 2017. No votes cast.
KALA INVELTYS - STRIDE 1
Dry eye disease
Phase 3
Phase 3 data released January 5, 2017 - both primary symptom endpoints met. Primary sign endpoint missed.
KALA INVELTYS - STRIDE 2
Dry eye disease
Phase 3
Phase 3 data released January 5, 2017 - one of two primary endpoints met.
AGEN Shingrix
Shingles
-
Advisory Committee Meeting September 13, 2017 voted 11-0 in favor. Shingrix, containing Agenus' adjuvant, QS-21 Stimulon.
AGEN Shingrix
Shingles
Approved
Approval announced October 20, 2017.
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).
CLLS UCART123
Acute myeloid leukemia (AML) and in blastic plasmacytoid dendritic cell neoplasm (BPDCN).
Phase 1
Release of clinical hold announced November 6, 2017.
CLLS UCART123
Acute myeloid leukemia (AML) and in blastic plasmacytoid dendritic cell neoplasm (BPDCN).
Phase 1
ASH 2017 data showed five out seven patients treated achieved molecular remission.
NVS Fingolimod
Phase 3
Phase 3 trial met primary endpoint.
NVS Fingolimod
Phase 3
Phase 3 trial met primary endpoint - data released at ECTRIMS-ACTRIMS meeting October 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.
ENTA EDP-305
Non-alcoholic steatohepatitis (NASH)
Phase 1
Phase 1 data at AASLD meeting - October 23, 2017.
JNJ Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
Approved
Approval announced August 2, 2017.
MDGL MGL-3196
Non-alcoholic steatohepatitis (NASH)
Phase 2
Phase 2 top-line data released December 6, 2017 - primary endpoint met.
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.
BMY Sprycel (dasatinib)
Philadelphia Chromosome-Positive Chronic Phase Chronic Myeloid Leukemia
Approved
Approval announced November 10, 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.
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.
GSK Mepolizumab
Eosinophilic Granulomatosis with Polyangiitis (EGPA)
Approved
Approval announced December 12, 2017.
JNJ XARELTO (rivaroxaban)
Reduce the Risk of Recurrent Venous Thromboembolism (VTE)
Approved
Approval announced October 30, 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.
BLUE HGB-205
Transfusion-Dependent β-Thalassemia (TDT) and Severe Sickle Cell Disease (SCD)
Phase 1/2
Phase 1/2 data presented at ASH 2017 - noted 2/3 patients showed meaningful clinical improvement.
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.
BOLD AT132 - ASPIRO
X-Linked Myotubular Myopathy
Phase 1/2
Phase 1/2 dosing has commenced - noted September 21, 2017 with preliminary data released January 4, 2018.
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.
BMY SGEN ADCETRIS and Opdivo CM-812
Relapsed Hodgkin Lymphoma
Phase 1/2
Data from Phase 1/2 at ASH 2017 showed a 83% ORR and 62% CR.
NVS Kymriah (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%.
ARGX ARGX-110
Relapsed/refractory cutaneous T-cell lymphoma (CTCL)
Phase 2
Phase 2 interim data ASH 2017 - of 22 patients 1 CR, 2 PRs, 10 SD.
ARGX ARGX-110
Acute myeloid leukemia (AML)
Phase 1/2
Phase 1/2 interim data at ASH 2017 - all six patients showed response including 3/6 with complete remission.
ARGX ARGX-113
Myasthenia gravis (MG)
Phase 2
Phase 2 data released December 11, 2017 - 75% of patients showed meaningful improvement.
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
Approved
Approved September 22, 2017 - under priority review.
MRK Keytruda
Cancer - Third-line Gastric or Gastroesophageal Junction Adenocarcinoma
Phase 3
Approved Sept. 22, 2017 under accelerated approval in patients undergoing third-line treatment following data from Phase 2 Keynote-59 trial. Phase 3 data released December 14, 2017 in patients undergoing second-line treatment, did not meet the primary endpoint of overall survival, nor did it show a significant improvement in PFS.
CALA CX-1158-101
Solid tumors - cancer
Phase 1
Phase 1 oral presentation at ASCO, June 5, 2017.
FBIO CAEL‐101
AL amyloidosis
Phase 1/2
Phase 1/2 data at ASH December showed 67% response rate.
INO AZN MEDI0457
HPV-associated squamous cell carcinoma of the head & neck (SCCHN)
Phase 1
Presentation at SITC Meeting - November 10, 2017.
AZN Tralokinumab (STRATOS2)
Severe, uncontrolled asthma
Phase 3
Phase 3 data released November 1, 2017 - primary endpoint not met.
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.
ACHN ACH-4471
C3 glomerulopathy (C3G)
Phase 2
Phase 2 interim data released November 14, 2017.
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.
RARX RA101495
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2 data released December 4, 2017. Primary endpoint met but with competitive concerns.
REGN 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%.
INCY 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.
INCY 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.
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.
SAGE Brexanolone - SAGE-547 (202C)
Postpartum Depression - moderate
Phase 3
Phase 3 data released November 9, 2017. Primary endpoint met.
INCY BMY 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.
DBVT Viaskin Peanut (REALISE)
Peanut allergy
Phase 3
Phase 3 positive safety data released November 20, 2017.
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.
AKTX Coversin
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2 further update December 8, 2017 noted that three additional patients together with earlier five patients met primary endpoint.
ANAB ANB020
Moderate-to-severe adult atopic dermatitis
Phase 2a
Phase 2a positive data released October 10, 2017
ESPR Bempedoic Acid/ Ezetimibe (1002FDC-053)
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.
VRX Latanoprostene bunod
Open angle glaucoma (OAG) or ocular hypertension (OHT)
Approved
Approval announced November 2, 2017.
RNN RX-3117
Advanced bladder cancer - 3rd line
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.
OHRP OHR-102 (MAKO)
Neovascular (Wet) Age-related Macular Degeneration (AMD)
Phase 3
Phase 3 data released January 5, 2017 - primary endpoint not met.
AGN Botox
Depression
Phase 2
Phase 2 top-line data released April 5, 2017 - primary endpoint missed. Phase 3 planned.
AGN Cariprazine
Bipolar I depression
Phase 3
Phase 3 data released December 18, 2017 - primary endpoint met.
AGN Cariprazine
Maintenance Treatment of Schizophrenia
Approved
Approval announced November 13, 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.
GLPG GLPG2222 - ALBATROSS
Cystic fibrosis (CF)
Phase 2
Phase 2a postive top-line data released November 20, 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.
MRK LLY (MK-3475-189/KEYNOTE-189)
First Line Metastatic Non-squamous Non-small Cell Lung Cancer (NSCLC)
Phase 3
Phase 3 trial met primary endpoints of overall survival (OS) and progression-free survival (PFS) - January 16, 2018. Data to be presented at an upcoming medical meeting.
VRX LUMIFY (brimonidine tartrate ophthalmic solution 0.025%)
Eye Brightener
Approved
Approval announced December 22, 2017.
VRX Brodalumab
Psoriasis
Approved
Approved February 15, 2017.
REGN 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 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 EYLEA + nesvacumab
Diabetic macular edema (DME)
Phase 2
Phase 2 data released November 27, 2017 - insufficient efficacy shown to warrant further development.
REGN EYLEA + nesvacumab
AMD - age-related macular degeneration
Phase 2
Phase 2 data released November 27, 2017 - insufficient efficacy shown to warrant further development.
REGN SNY Dupixent (dupilumab)
Eosinophilic esophagitis
Phase 2
Phase 2 data released May 4, 2017.
REGN SNY 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 Xeljanz
Psoriatic arthritis
Approved
Approval announced December 14, 2017.
PFE Talazoparib
BRCA-mutated breast cancer
Phase 3
Phase 3 data presented December 8, 2017. HR 0.54.
PFE Sutent (Sunitinib)
Renal cell carcinoma (RCC)
-
Advisory Committee Meeting September 19, 2017 voted 6-6 the benefits outweigh risks.
PFE Sutent (Sunitinib)
Renal cell carcinoma (RCC)
Approved
Approval announced 16 November, 2017.
PFE MRK Ertugliflozin
Type 2 diabetes
Approved
Approval announced December 20, 2017.
PFE BOSULIF (BOSUTINIB)
First-Line Treatment of Philadelphia Chromosome Positive Chronic Myeloid Leukemia
Approved
Approval announced December 19, 2017.
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 LEE011: MONALEESA-7
Breast cancer - (pre-menopausal)
Phase 3
Phase 3 trial met primary endpoint - November 8, 2017.
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 Semaglutide
Type 2 diabetes
Approved
Approval announced December 5, 2017.
NVO Nonacog beta pegol
haemophilia B
Approved
Approval announced May 31, 2017.
MYL Ogivri - trastuzumab biosimilar
Trastuzumab biosimilar
Approved
Approval announced December 1, 2017.
MRK Letermovir
Cytomegalovirus (CMV) Infection
Approved
Approval announced November 9, 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 Ramucirumab - RAINFALL
First-line gastric cancer
Phase 3
Phase 3 PFS data met primary endpoint but overall survival not improved. Will not seek regulatory approval.
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 - 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
Approved
Approved October 4, 2017.
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.
LLY Taltz
Psoriatic Arthritis
Approved
Approval announced December 1, 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 DARZALEX (Daratumumab) - ALCYONE
Frontline multiple myeloma (non-transplant)
Phase 3
LBA at ASH 2017 - HR 0.50.
JNJ STELARA (USTEKINUMAB)
Plaque psoriasis
Approved
Approval announced October 13, 2017.
JNJ SIMPONI ARIA
Psoriatic arthritis (PsA)
Approved
Approval announced October 20, 2017.
JNJ SIMPONI ARIA
Ankylosing Spondylitis
Approved
Approval announced October 20, 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 GSK Trelegy Ellipta (FF/UMEC/VI)
Chronic obstructive pulmonary disease (COPD)
Approved
Approval announced September 18, 2017.
GSK Dolutegravir + rilpivirine
HIV
Approved
Approval announced November 21, 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 Shingrix
Shingles
Approved
Approval announced October 20, 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 GS-0976
Nonalcoholic steatohepatitis (NASH)
Phase 2
Phase 2 data released October 24, 2017. Positive high dose data, low dose negative.
GILD Vosevi (Sofosbuvir/Velpatasvir/Voxilaprevir)
Hepatitis C virus (HCV)
Approved
Approval announced July 18, 2017.
CELG REVLIMID - RELEVANCE
Newly Diagnosed Follicular Lymphoma
Phase 3
Phase 3 data released December 21, 2017 - co-primary endpoints not met.
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 Ozanimod - RADIANCE
Relapsing Multiple Sclerosis
Phase 3
Phase 3 data due released May 22, 2017 - primary endpoint met, disability endpoint not met. Full data released October 28, 2017 at ECTRIMS meeting.
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 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-238 – Opdivo (Adjuvant)
Melanoma
Approved
Approval announced December 20, 2017.
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.
BMY CM-078 – Opdivo
Second-line Non-small cell lung cancer (NSCLC)
Approved
Phase 3 trial met primary endpoint - November 30, 2017.
BIIB BAN2401 (Aβ mAb)
Alzheimer’s disease
Phase 2
Noted December 21, 2017 that Independent Data Monitoring Committee determined trial did not meet criteria for success.
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 MRK Lynparza
Breast cancer
Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met.
AZN MRK 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 MRK Lynparza
Breast cancer
Approved
Approval announced January 12, 2018.
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 Acalabrutinib
Relapsed or Refractory Mantle Cell Lymphoma
Approved
Priority Review announced August 2, 2017. Approval announced October 31, 2017.
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.
AZN Benralizumab
Severe, uncontrolled asthma
Approved
Approval announced November 14, 2017.
AMGN Repatha
Cardiovascular disease
Phase 3
Phase 3 cardiovascular outcomes study data released March 17, 2017 - endpoints met but down on expectations.
AMGN Repatha
Cardiovascular disease
Approved
Approval announced December 1, 2017.
AMGN XGEVA
Multiple Myeloma
Approved
sBLA approval announced January 5, 2017.
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.
ALXN Eculizumab
Refractory generalized myasthenia gravis (gMG)
Approved
Approval announced October 23, 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 Venclexta (MURANO)
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
Phase 3
Phase 3 data released September 18, 2017 - PFS primary endpoint met. LBA at ASH data showed PFS of 85% after 24 months.
ABBV Risankizumab
Psoriasis
Phase 3
Phase 3 data released October 26, 2017 - primary endpoints 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 - T-Force GOLD
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).
ARDX RDX7675
Hyperkalemia
Phase 2
Onset of action trial data released November 21, 2017 - noted significantly reduced serum potassium in patients. Development to be discontinued.
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.
EGRX PEMFEXY,
Nonsquamous Non-Small Cell Lung Cancer (NSCLC) and Mesothelioma
Approved
Tentative approval announced October 27, 2017. Full approval pending patent litigation with Eli Lilly.
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.
RARE DTX301
Ornithine Transcarbamylase (OTC) Deficiency
Phase 1/2
Phase 1/2 initial from first cohort released January 7, 2018.
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.
GBT GBT440
Hypoxemia in patients with idiopathic pulmonary fibrosis (IPF) who are on supplemental oxygen at rest
Phase 2a
Phase 2a discontinuation announced October 23, 2017 due to lack of efficacy.
SCYX Intravenous SCY-078
Invasive candidiasis
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 CELG 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%.
BLUE CELG bb2121
Relapsed/refractory multiple myeloma
Phase 1
Phase 1 data released at ASH 2017 - ORR 94%, CR 56%.
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.
MNOV MN-166
Progressive multiple sclerosis (progressive MS)
Phase 2b
Phase 2b top-line data released October 28, 2017 at Joint ECTRIMS – ACTRIMS meeting. Primary endpoint met.
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.
RVNC RT002
Plantar fasciitis
Phase 2a
Phase 2a data released January 8, 2017. Primary endpoint was not met.
IRWD IW-1973
Type 2 diabetic patients with hypertension
Phase 2
Phase 2a data released December 4, 2017 - Noted data encouraging but with one serious adverse event.
ALDR ALD403 - PROMISE 2
Frequent episodic migraine
Phase 3
Phase 3 PROMISE 2 data released January 8, 2017 - endpoints met.
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.
TGTX Umbralisib (TGR-1202) + Pembrolizumab + Ublituximab (TG-1101)
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
Phase 1/2
Phase 1/2 poster at ASH 2017 showed 78% (7 of 9) ORR.
EIGR Pegylated Interferon Lambda (LIMT HDV)
Hepatitis delta virus (HDV)
Phase 2
Noted July 24, 2017 that enrollment has been completed. Interim released October 23, 2017.
COLL Xtampza ER (oxycodone)
Severe pain
Approved
Approval announced November 7, 2017.
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 ALNY 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%.
KDMN KD025
Chronic graft-versus-host disease (cGVHD)
Phase 2
Updated data at ASH December 2017 showed ORR 63%.
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.
EARS AM-111 HEALOS
Acute inner ear hearing loss
Phase 3
Phase 3 HEALOS trial did not meet primary endpoint - November 28, 2017.
IMDZ G100
Follicular non-Hodgkin Lymphoma - cancer
Phase 2
Phase 2 data ASH 2017 - ORR 39%.
LIPO-202
Reduction of localized fat deposits under the chin (submental fat)
Phase 2
Phase 2 data released June 26, 2017 - no efficacy shown.
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.
AFMD AFM13 with Keytruda
Hodgkin Lymphoma - Cancer
Phase 1b
Phase 1b poster at ASH 2017.ORR 83% (5/6).
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.
RNN RX-3117
Relapsed and refractory pancreatic cancer
Phase 2a
Updated Phase 2a data presented at ASCO GI January 19, 2018. 1/43 partial responses; 11/43 (28%) stable disease.
ADAP NY-ESO T-cell therapy
Synovial sarcoma - cancer
Phase 1/2
Oral presentation at CTOS November 9, 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
Major Depressive Disorder (MDD)
Phase 2
Phase 2 initiation announced December 19, 2016. Top-line data released December 7, 2017 - primary endpoint met.
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.
SAGE SAGE-217
Parkinson’s disease (PD)
Phase 2
Part B data released November 2, 2017.
RXII RXI-109-1402
Connective tissue growth factor (CTGF)
Phase 2
Phase 2 data released December 18 2017 - primary effectiveness objective was met.
CPRX Firdapse
Lambert-Easton Myasthenic Syndrome (LEMS)
Phase 3
Refusal to file letter reported on February 17, 2016. Data from second Phase 3 trial released November 27, 2017 - endpoints met.
ACOR Tozadenant
Parkinson’s disease (PD)
Phase 3
Phase 3 data due 1Q 2018. Noted November 15, 2017 seven cases of sepsis, five of which were fatal.
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 (Ocaliva)
Primary biliary cirrhosis (PBC) - POISE
Approved
Approved May 27 2016.
AXSM AXS-02 CREATE-1
Complex regional pain syndrome (CRPS)
Phase 3
Phase 3 interim efficacy analysis January 9, 2017 recommended trial to discontinue due to futility.
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.
AGRX Twirla
Contraceptive patch
CRL
CRL issued 2013. Further CRL issued December 22, 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.
RDHL RIZAPORT (RHB-103)
Migraine
-
Noted December 5, 2017 that an Incomplete Response Letter has been received from the FDA and termination of license agreement with IntelGenx Corp.
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 or Opdivo - Indigo301
Melanoma - cancer
Phase 2
Phase 2 interim analysis released at AACR meeting April 4, 2017.
NLNK Indoximod in combination with KEYTRUDA or Opdivo - Indigo301
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-NHL 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.
ONCE SPK-8011
Hemophilia A
Phase 1/2
Further data due at ASH 2017 showed promising reductions in bleeding shown but inconsistent levels of Factor VII.
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.
PTCT Ataluren
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
CRL
CRL issued October 25, 2017.
STML SL-401
High Risk Myeloproliferative Neoplasms
Phase 2
ASH 2017 data showed 65% (11/17 evaluable) of CMML and MF patients had spleen reductions >25%.
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.
ADAP NY-ESO T-cell therapy
Myxoid round cell liposarcoma (MRCLS)
Phase 1/2
Poster at CTOS November 9, 2017.
KURA Tipifarnib
Chronic myelomonocytic leukemia (CMML) - cancer
Phase 2
Preliminary data at ASH 2017 - ORR 33% among 9 patients.
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
Head and neck squamous cell carcinomas (HNSCC)
Phase 2
Phase 2 ongoing data released March 6, 2017 - further data due 2H 2017.
KURA Tipifarnib
Head and neck squamous cell carcinomas (HNSCC)
Phase 2
Phase 2 trial met primary endpoint prior to completion of enrollment.
TBPH Closed Triple
COPD
Approved
Approval announced September 19, 2017.
SELB SEL-212
Tophaceous gout
Phase 2
Phase 2 dosing commenced late October 2016. Initial data released at EULAR June 15, 2017.
GALT GR-MD-02
Non-alcoholic steatohepatitis (NASH) with cirrhosis
Phase 2b
Phase 2 top-line data released December 5, 2017 did not meet primary endpoint across all patients - noted subgroup improvement.
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 DRRX Remoxy
Chronic pain
CRL
CRL received June 24, 2011.
PTIE DRRX 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 - TX-004HR
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 Axicabtagene ciloleucel - KTE-C19 (ZUMA-3)
Adult patients with r/r ALL - cancer
Phase 1/2
Complete remission 73%. Data released at ASH 2017 showed complete remission of 71%.
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.
SAGE Brexanolone - SAGE-547 (202B)
Postpartum Depression (Severe)
Phase 3
Phase 3 data released November 9, 2017. Primary endpoint met.
SYRS SY-1425
Relapsed or refractory Acute myeloid leukemia (AML) and relapsed high-risk Myelodysplastic Syndrome (MDS) - cancer
Phase 2
Phase 2 data at ASH showed 1 complete response from 48 patients.
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.
EIGR Ubenimex - LIBERTY
Pulmonary arterial hypertension (PAH)
Phase 2
Phase 2 trial did not meet endpoints - January 16, 2018.
QURE AMT-060
hemophilia B
Phase 1/2
Phase 1/2 long term updated data presented July 10, 2017.
BMRN Valoctocogene roxaparvovec (BMN 270)
Hemophilia A
Phase 1/2
Phase 1/2 updated data released July 11, 2017. 97% reduction in Annualized Bleed Rate (ABR).
BMRN Valoctocogene roxaparvovec (BMN 270)
Hemophilia A
Phase 1/2
Phase 1/2 updated data released at ASH 2017. Noted median annualized bleed rates dropped to zero after four weeks among six patients.
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.
TSRO Rolapitant - intravenous (IV)
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
PDUFA
CRL January 11 2017. FDA Approval announced October 25, 2017 following resubmission.
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.
MLNT Solithera - intravenous
Community-acquired bacterial pneumonia (CABP)
CRL
CRL announced December 29, 2016. Further trials required.
ARQL Derazantinib (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 Intepirdine - HEADWAY-DLB
Dementia with Lewy bodies (DLB)
Phase 2b
Phase 2 data released January 2018 - negative data.
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 Nelotanserin
Visual hallucinations in subjects with Lewy body dementia
Phase 2
Phase 2 data released January 8, 2018. Dementia endpoint missed.
AXON Intepirdine - MINDSET
Alzheimer's disease
Phase 3
Phase 3 data released September 26, 2017 - primary endpoints not met.
AXSM AXS-02 COAST-1
Knee Osteoarthritis Associated with Bone Marrow Lesions
Phase 3
Phase 3 interim efficacy analysis January 9, 2017 recommended trial continue to full enrollment.
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
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.
BLUE LentiGlobin - HGB-206
Sickle disease
Phase 1
Data at ASH 2017 showed 51% and 28% anti-sickling HbAT87Q at six and nine months for two patients.
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.
MLNT Solithromycin
NASH
Phase 2
Phase 2 interim data released September 30, 2016.
MLNT Solithromycin
NASH
Phase 2
Noted February 28, 2017 that development has been suspended due to lack of efficacy.
MLNT 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 2
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.
CYTK Tirasemtiv - VITALITY
Amyotrophic lateral sclerosis (ALS)
Phase 3
Phase 3 data released November 21, 2017 - primary endpoint not met.
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.
DBVT Viaskin Peanut (PEPITES)
Peanut allergy
Phase 3
Phase 3 top-line data released October 20, 2017 - primary endpoint not met.
DEPO SEFELSA
Hot flashes
CRL
CRL issued May 31, 2013.
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.
DVAX HEPLISAV-B
Hepatitis B
Approved
Final approval announced November 9, 2017
EARS AM-111 ASSENT
Acute inner ear hearing loss
Phase 3
Phase 3 AS