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1
IMGN
Ovarian cancer
Phase 1/2
2022-09-29 00:00:00
Phase 1/2 data reported an ORR of 44%, a median DOR of 11.8 months, and median PFS of 8.2 months, noted September 29, 2022.
0
TNXP
Major depressive disorder (MDD)
Phase 2
2023-11-01 00:00:00
Phase 2 topline data reported that the trial did not meet its primary endpoint, noted November 1, 2023.
1
VTGN
Adjunctive Treatment Major Depressive Disorder
Phase 2
2019-11-14 00:00:00
Phase 2 data did not meet primary endpoint - November 14, 2019.
1
GLPG
B-cell malignancies, Chronic Lymphocytic Leukemia (CLL) and Small Lymphocytic Lymphoma (SLL)
Phase 1/2
2023-06-09 00:00:00
Phase 1/2 data presented at EHA showed that based on current results (n=7). For all 7 pts, GLPG5201 was successfully manufactured at the PoC and administered as a fresh product, with a median vein-to-vein time of 7 days. Rapid complete responses have been observed with a 100% ORR in a heavily pre-treated pt population. Ph 1 recruitment is ongoing to establish a recommended dose for Phase 2, noted June 9, 2023.
1
BBIO
Achondroplasia
Phase 2
2023-06-20 00:00:00
Phase 2 cohort 5 data reported at ENDO showed that at the Cohort 5 dose level resulted in a significant and robust increase in AHV compared to BL, with a change of +3.38cm/year, noted June 20, 2023.
1
HEPA
NASH and Liver Cancer
Phase 2a
2022-01-08 00:00:00
Phase 2a data reported that treatment was well-tolorated with ALT decreased in 50%, 67%, and 87% of the subjects in the placebo, 75 mg, and 225 mg cohorts, respectively, noted January 8, 2022.
1
BMY
Non-transfusion-dependent beta-thalassemia
Phase 2
2021-06-11 00:00:00
Phase 2 data presented at EHA meeting June 11, 2021. 77.7% of patients achieved a hemoglobin increase compared to 0% in the placebo arm.
1
BMY
Non-Small Cell Lung Cancer (NSCLC)
Approved
2022-04-11 00:00:00
Approved April 11, 2022.
1
JNJ
Multiple sclerosis
Approved
2015-04-16 00:00:00
Approved April 16 2015.
1
PFE
Multiple Myeloma
Approved
2023-08-14 00:00:00
FDA Approved on August 14, 2023.
1
INVA
Hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia (HABP/VABP) caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (Acinetobacter)
Approved
2023-05-24 00:00:00
Approved May 24, 2023.
1
MTEM
Relapsed/refractory diffuse large B-cell lymphoma (DLBCL)
Phase 2
2021-04-05 00:00:00
Development to be discontinued - April 5, 2021.
-1
JNJ
Multiple myeloma - candidates for autologous stem cell transplant (ASCT)
Approved
2019-09-26 00:00:00
FDA Approval announced September 26, 2019.
1
AMRN
Covid-19
Phase 3
2021-11-16 00:00:00
Phase 3 trial did not met the primary or secondary endpoints, noted November 16, 2021.
0
RCUS
Metastatic castrate-resistant prostate cancer (mCRPC)
Phase 1/2
2023-08-07 00:00:00
Phase 1/2 trial is not expected to demonstrate sufficient clinical benefit in castrate resistant prostate cancer to warrant further investment, noted August 7, 2023.
1
ITCI
Bipolar depression
Phase 3
2019-07-08 00:00:00
Phase 3 trial did not meet primary endpoint - July 8, 2019.
1
PTGX
Polycythemia vera
Phase 2
2023-11-02 00:00:00
Phase 2 data from abstract reported that treatment resulted in consistent maintenance of hematocrit below 45% and an overall decrease in erythrocyte counts, noted November 2, 2023.
1
INCY
Myelofibrosis
Approved
2011-11-16 00:00:00
Approved November 16, 2011.
1
LPCN
Testosterone replacement therapy (TRT)
Approved
2022-03-29 00:00:00
Approved March 29, 2022.
1
VBIV
Hepatitis B vaccine
Phase 3
2022-09-21 00:00:00
Additional Phase 3 data reported that treatment may be effective only in low level HBsAg carriers with a low tolerizing effect of HBsAg, noted September 21, 2022.
1
CYTH
Niemann-Pick Disease Type C
Phase 1
2022-10-18 00:00:00
Phase 1 data showed that treatment overcomes the NPC1 defect by removing trapped cholesterol from cells both systemically and in the central nervous system (CNS), noted October 18, 2022.
1
PSTV
Leptomeningeal metastases (LM)
Phase 1/2
2023-11-03 00:00:00
FDA granted Orphan Drug Designation (ODD), noted November 3, 2023.
0
RHHBY
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 3
2022-12-12 00:00:00
Phase 3 results reported that trial met primary endpoints, noted December 12, 2022.
0
EDIT
Sickle Cell Disease
Phase 1/2
2023-06-09 00:00:00
Phase 1/2 data presented at EHA reported that all five patients treated with EDIT-301 successfully engrafted and all four RUBY patients treated are free of vaso-occlusive events since infusion, noted June 9, 2023.
1
MRNA
Respiratory syncytial virus (RSV) vaccine
BLA Filing
2023-07-05 00:00:00
BLA rolling submission, noted July 5, 2023.
0
MRK
Pulmonary arterial hypertension (PAH)
Phase 2
2021-05-19 00:00:00
Phase 2 update at the American Thoracic Society 2021 International Conference May 19, 2021. Generally well tolerated, improvements in resting and exercise hemodynamics at week 24 shown.
1
MRK
Personalized cancer vaccine, high-risk melanoma
Phase 2
2023-10-23 00:00:00
Phase 2 additional data presented at ESMO reported that local recurrence was more common in MRs; MNRs had more distant events. This trend was supported by stronger separation in DMFS vs RFS between MRs and MNRs, noted October 23, 2023.
1
MRK
Complicated urinary tract infections (cUTI) and Complicated intra-abdominal infections (cIAI)
Approved
2019-07-17 00:00:00
FDA Approval announced July 17, 2019.
1
AUTL
B-Cell Acute Lymphoblastic Leukemia
Phase 2
2023-06-02 00:00:00
Phase 2 safety and efficacy data presented at ASCO showed that 76% of patients treated achieved a response (CR/CRi), primary endpoint has been met based on previously communicated interim analysis, noted June 2, 2023
1
GRTX
Pancreatic cancer
Phase 1/2
2021-09-08 00:00:00
Phase 1/2 trial final results announced - GC4419 was well tolerated, with similar rates of early and late adverse events in the active and placebo arms September 8, 2021.
1
NBIX
Parkinson's Disease
Phase 2
2020-12-22 00:00:00
Phase 2 trial placed on clinical hold - December 22, 2020.
0
MOR
Psoriatic Arthritis
Approved
2020-07-14 00:00:00
FDA Approval announced July 14, 2020.
1
AVRO
Gaucher disease
Phase 1/2
2021-10-19 00:00:00
Phase 1/2 safety data reported no SAEs and no AEs for more than 14 months post-treatment, noted October 19, 2021.
1
ABBV
Atopic dermatitis
Approved
2022-01-14 00:00:00
Approved January 14, 2022.
1
ACOR
Parkinson’s disease (PD)
Phase 3
2017-11-15 00:00:00
Phase 3 data due 1Q 2018. Noted November 15, 2017 seven cases of sepsis, five of which were fatal.
0
EBS
Opioid overdose
Approved
2023-03-29 00:00:00
Approved March 29, 2023.
1
RIGL
COVID-19
Phase 2
2021-04-13 00:00:00
Phase 2 trial met primary endpoint of safety - April 13, 2021.
0
MRTX
MTAP-deleted cancers
Phase 1
2023-08-08 00:00:00
Phase 1 data reported a safety profile and early signs of clinical activity, noted August 8, 2023.
0
PFE
Biosimilar bevacizumab
Approved
2019-06-28 00:00:00
FDA Approval announced June 28, 2019.
1
BMY
RET-altered NSCLC
Phase 1/2
2021-04-05 00:00:00
Phase 1/2 interim data noted 3/14 partial responses - April 5, 2021.
0
AZN
Lung cancer
Phase 3
2018-11-16 00:00:00
Overall survival data also did not meet primary endpoint - November 16, 2018.
1
LLY
COVID-19 antibody
Approved
2022-02-11 00:00:00
EUA granted by FDA February 11, 2022.
0
MRK
Non-small cell lung cancer (NSCLC)
Approved
2019-04-11 00:00:00
FDA Approval announced April 11, 2019.
1
MESO
Inflammatory bowel disease (IBD), Ulcerative colitis or Crohn’s colitis
Phase 1/2
2022-02-20 00:00:00
Phase 1/2 interim analysis reported that all ulcerative colitis patients had improved clinical and endoscopy scores within two weeks, and all Crohn’s colitis patients showed treatment remissions or responses by three months, noted February 20, 2022.
1
BCRX
Paroxysmal nocturnal hemoglobinuria (PNH), Healthy Volunteers
Phase 1
2023-10-26 00:00:00
Phase 1 PoC trial with PNHl initiated, noted October 26, 2023.
0
MRKR
Lymphoma who have failed or are ineligible to receive anti-CD19 CAR T cell treatment
Phase 1
2023-09-11 00:00:00
Phase 1 clinical update showed that patients with Non-Hodgkin's Lymphoma who relapsed after anti-CD19 CAR T cell therapy tolerated initial dose level well and achieved complete response after MT-601 treatment, noted September 11, 2023.
1
ONCY
Pancreatic cancer
Phase 2
2021-05-20 00:00:00
Phase 2 data in ASCO abstract noted response rate 1/12 (8.3%); stable disease 42%.
0
MRK
BRCAm HER2-negative breast cancer
Approved
2022-03-11 00:00:00
Approved March 11, 2022.
1
BMY
Tumors harboring ALK, ROS1, or NTRK1-3 alterations
Phase 1/2
2021-10-23 00:00:00
Phase 1/2 early clinical data demonstrated confirmed responses in (3/4) TKI-naïve patients, noted October 23, 2021.
1
RHHBY
Autosomal Dominant Alzheimer's Disease (ADAD
Phase 3
2022-08-02 00:00:00
Additional Phase 3 data reported that cognitive test scores of API ADAD composite 22.9%, FCSRT 19.9% and RBANS total score 43.8%, noted August 2, 2022.
0
TSVT
Newly Diagnosed Multiple Myeloma
Phase 2a
2022-12-10 00:00:00
Cohort 2a efficacy and safety data presented at ASH demonstrated complete and durable responses in a significant proportion of patients, noted on December 10, 2022.
1
BIIB
Rheumatoid arthritis (RA)
Approved
2023-09-29 00:00:00
FDA Biosimilar of ACTEMRA approved on September 29, 2023.
1
BGNE
HER2-amplified biliary tract cancers
Phase 2
2023-06-05 00:00:00
Phase 2 pivotal trial recruitment completed, noted April 28, 2022. Phase 2 full data presented at ASCO reported a confirmed objective response rate (cORR) of 41.3%, median duration of response (DOR) of 12.9 months, and median progression-free survival (PFS) of 5.5 months, noted June 5, 2023.
1
AVIR
COVID-19
Phase 2
2021-10-19 00:00:00
Phase 2 trial did not meet primary endpoint, noted October 19, 2021.
1
VCYT
Prostate cancer
Phase 3
2023-10-04 00:00:00
Phase 3 trial validation of a genomic classifier presented at the ASTRO meeting noting that only the Decipher Prostate risk score was independently associated with metastasis-free survival (MFS; HR 1.12, 95% CI) and distant metastasis (DM; sHR 1.22, 95% CI), compared to standard risk factors including Gleason score, T-stage and prostate-specific antigen (PSA) level, noted October 4, 2023.
1
JSPR
Sickle Cell Disease and Beta Thalassemia
Phase 1/2
2023-02-16 00:00:00
Phase 1/2 follow up data reported that two sickle cell disease participants have achieved 100% donor myeloid chimerism through 100 days follow-up, noted February 16, 2023.
1
MRK
Heart Failure
Approved
2021-01-20 00:00:00
FDA approval announced January 20, 2021.
1
CUE
Wilms' Tumor (WT1)-expressing cancers
Phase 1
2023-11-03 00:00:00
Phase 1 data presented at SITC demonstrated a 75%-80% DCR at the 4mg/kg and 2mg/kg doses respectively with two patients demonstrating tumor reductions of -30% and -29%, noted November 3, 2023.
1
RLYB
Paroxysmal nocturnal hemoglobinuria (PNH); Generalized Myasthenia Gravis (gMG)
Phase 1
2023-09-03 00:00:00
Phase 1 multiple ascending trial data reported maximum exposures of greater than 1 µM and 3 µM, respectively, and greater than 99% reductions in free C5 concentrations, noted September 3, 2023.
1
BHVN
Amyotrophic lateral sclerosis (ALS)
CRL
2019-07-19 00:00:00
CRL issued July 19, 2019.
0
SGEN
HER2 amplified metastatic colorectal cancer
Approved
2023-01-19 00:00:00
Approved January 19, 2023.
1
JNJ
Multiple Myeloma
Phase 3
2023-06-05 00:00:00
Phase 3 results shared at ASCO demonstrated a statistically significant improvement in progression-free survival, with a hazard ratio of 0.26, noted June 5, 2023
1
VKTX
Non-alcoholic steatohepatitis (NASH) and fibrosis
Phase 2b
2023-05-16 00:00:00
Phase 2b data reported that trial met primary endpoint, noted May 16, 2023.
0
RCKT
Glaucoma
Phase 2
2017-07-07 00:00:00
Phase 2 trial failed - noted July 7, 2017.
-1
FBRX
Atopic Dermatitis
Phase 2
2021-09-02 00:00:00
Phase 2 failed to meet primary endpoint September 02, 2021.
1
RHHBY
Neovascular age related macular degeneration (nAMD)
Phase 3
2021-01-25 00:00:00
Phase 3 trial met primary endpoint - January 25, 2021.
0
ARCT
COVID-19 variant vaccine
Phase 1/2
2022-01-24 00:00:00
Additional Phase 1/2 data reported neutralization against several variants of concern after day 15 and 29, noted January 24, 2022.
0
WVE
Huntington’s disease
Phase 1/2
2021-03-29 00:00:00
Phase 1/2 data showed no statistically significant change in mutant huntingtin protein (mHTT). Development to be stopped
1
DBVT
Peanut allergy - ages 4 to 11 years
CRL
2020-08-04 00:00:00
CRL issued August 4, 2020
0
VRNA
COVID-19
Phase 2
2021-04-23 00:00:00
Phase 2 top-line data released April 23, 2021 - safe and well tolerated. One patient death was reported in the ensifentrine treatment group.
1
BHVN
Multiple system atrophy (MSA)
Phase 3
2021-09-27 00:00:00
Phase 3 trial did not met primary or secondary endpoints, noted September 27, 2021.
0
NBIX
Negative symptoms of schizophrenia
Phase 2
2021-03-02 00:00:00
Phase 2 trial did not meet primary endpoint - March 2, 2021.
1
ETON
Nutritional requirements for patients with liver disease
Approved
2022-04-11 00:00:00
Approved April 11, 2022.
1
TERN
Nonalcoholic steatohepatitis (NASH)
Phase 2a
2021-11-12 00:00:00
Phase 2a safety and efficacy clinical data reported no differences from placebo in percentage change in low density lipoprotein (LDL) cholesterol or high density lipoprotein (HDL) cholesterol. Reductions in alanine transaminase (ALT) and MRI proton density fat fraction (MRI-PDFF) were reported in the 10 and 15 mg groups, and significant reductions in gamma-glutamyl transferase (GGT) in all dose groups, noted November 12, 2021.
1
IMVIQ
Ovarian Cancer
Phase 1/2
2022-06-06 00:00:00
Results from Phase 1 and Phase 2 expansion cohort presented at ASCO reported that G3/G2 immune-related AE and injection site reactions were observed in 1/1 and 1/3 pts treated at DL1, and in 1/0 and 2/2 pts at DL2, respectively. At Phase 1, one patient with MSI-High clear cell subtype had an ongoing CR after 26 months of follow-up, 2 patients had PR and 6 SD. There were 1 PR and 3 SD on P2EC, of which 1 and 2, respectively, achieved response greater than 12 weeks, noted June 6, 2022.
1
GILD
Urothelial Cancer
Approved
2021-04-13 00:00:00
FDA approval announced April 13, 2021.
1
BMY
Chronic lymphocytic leukemia (CLL)
Phase 1/2
2023-06-06 00:00:00
Phase 1/2 data presented at ASCO reported that the primary endpoint of CR/CRi rate was met at 18.4%, and the ORR was 42.9% and was not statistically significant, noted June 6, 2023.
1
GHRS
Treatment-Resistant Depression (TRD)
Phase 2
2021-12-06 00:00:00
Phase 2 trial met primary and secondary endpoints, noted December 6, 2021.
0
BTAI
Neuroendocrine Prostate Cancer (NEPC)
Phase 2a
2023-01-11 00:00:00
Phase 2a data reported that combination with pembrolizumab has demonstrated an encouraging response rate, noted January 11, 2023.
1
LLY
Crohn's disease
Phase 3
2023-10-12 00:00:00
Phase 3 trial met the co-primary and all major secondary endpoints, noted October 12, 2023.
0
AMTI
Pouchitis
Phase 2
2023-03-03 00:00:00
Additional Phase 3 data demonstrated oral's gut-restricted profile with tissue-level pharmacodynamics (PD) effects and no systemic exposure by design. Translational analysis reveals IL-10 biological responses in 3mg and 10mg dose arms, noted March 3, 2023.
1
AMGN
Solid tumors with MTAP deletion, NSCLC
Phase 1
2022-03-15 00:00:00
Phase 1 updated data reported no drug-related SAE's or observed dose limiting toxicity (DLT) through Cohort 5, noted March 15, 2022.
1
ALNY
Gout
Phase 1
2022-12-15 00:00:00
Phase 1 pharmacodynamic data suggest extrahepatic contributes significantly to serum urate in humans, noted December 15, 2022.
0
BYSI
Non small cell lung cancer
Phase 3
2022-12-13 00:00:00
Additional data reported that plinabulin demonstrated a superior benefit for Gr4N, Gr3/4N, all GrN and DSN compared to the control, noted December 13, 2022.
1
LLY
Hemophilia A
Phase 1/2
2021-07-09 00:00:00
Phase 1/2 clinical hold announced July 9, 2021.
0
KRYS
Alpha-1 antitrypsin deficiency (AATD)
Phase 1a
2023-09-05 00:00:00
FDA granted Orphan Drug Designation, noted September 5, 2023.
0
KZIA
Glioblastoma multiforme
Phase 2
2022-06-03 00:00:00
Phase 2 final data presented at ASCO reported that the median overall survival (OS) in the intent-to-treat (ITT) population was 15.7 months, compared to 12.7 months which was historically reported with temozolomide in this patient group. Median progression-free survival (PFS) in the ITT population was 8.6 months, noted June 3, 2022.
1
OCGN
Graft Versus Host Disease
Phase 3
2020-06-01 00:00:00
Phase 3 trial stopped early due to insufficient efficacy.
-1
RVMD
Pancreatic cancer, non-small cell lung cancer (NSCLC), and colorectal cancer.
Phase 1
2023-09-19 00:00:00
Phase 1 dosing initiated, noted Septemeber 19, 2023.
0
BCRX
Hereditary Angioedema
Phase 2
2023-05-07 00:00:00
Additional long-term 96 weeks at 150mg data reported that the attack-free status was consistently high with ORLADEYO 150 mg regardless of patients' age, gender and prior prophylactic treatment, noted May 7, 2023.
1
AMGN
Uncontrolled Gout
Approved
2022-07-08 00:00:00
Approved July 8, 2022.
1
CINC
Uncontrolled Hypertension (rHTN)
Phase 1
2022-11-28 00:00:00
Phase 1 topline data reported that trial did not meet primary endpoint, noted November 28, 2022.
1
KURA
Head and neck squamous cell carcinomas (HNSCC)
Phase 2
2023-10-21 00:00:00
Phase 2 data presented at ESMO reported that the Tipifarnib ORR was higher in 2L setting than 3L+ setting (29% vs. 15%), noted October 21, 2023.
1
SMMT
C. difficile infection (CDI)
Phase 3
2022-10-20 00:00:00
Phase 3 trial results reported that treatment was numerically higher sustained clinical response rate (73.0% vs 70.7%) than vancomycin, but did not achieve the pre-specified superiority endpoint, noted October 20, 2022.
1
PXMD
Human African Trypanosomiasis (HAT)
Phase 2b
2023-07-24 00:00:00
Phase 2b trial met its primary endpoint, noted July 24, 2023.
0
LIAN
Hypertrophic cardiomyopathy (HCM)
Approved
2022-04-28 00:00:00
Approved April 28, 2022.
1
MIRM
Progressive Familial Intrahepatic Cholestasis (PFIC)
Phase 3
2022-10-24 00:00:00
Phase 3 top-line data reported that trial met primary endpoint, noted October 24, 2022.
0
CGEN
Platinum resistant ovarian cancer
Phase 1/2
2023-06-05 00:00:00
Phase 1/2 data presented at ASCO reported an objective response rate (ORR) of 2/9 [22%] pts; 2 pts with SD. Disease control rate [CR + PR + SD] 4/9 [44%], noted June 5, 2023.
1
AMGN
Asthma
Approved
2021-12-17 00:00:00
Approved December 17, 2021.
1
ELDN
Amyotrophic Lateral Sclerosis
Phase 2a
2022-05-31 00:00:00
Phase 2a topline data showed that treatment met the primary endpoints of safety and tolerability. Adverse events were equally distributed across dose levels, noted May 31, 2022.
1