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1
DAWN
Low-grade Glioma
Phase 2
2023-06-05 00:00:00
Additional Phase 2 presented at ASCO reported an ORR of 67% and clinical benefit rate (CBR) of 93% in 69 heavily pretreated RANO-HGG evaluable patients, noted June 5, 2023.
1
BGNE
Follicular Non-Hodgkin Lymphoma
Phase 2
2023-06-09 00:00:00
Phase 2 data presented at EHA reported that the OS rate at 24 months was 77.3%(zanubrutinib plus obinutuzumab) and 71.4% (obinutuzumab), with median OS not reached (zanubrutinib plusobinutuzumab) and 34.6 months, noted June 9, 2023.
1
KPRX
Rheumatoid Arthritis
Phase 1b
2022-04-25 00:00:00
Phase 1b results demonstrated safety and tolerability of treatment as well as statistically significant improvements in conjunctival hyperemia in the 0.15% arm compared to vehicle control, noted April 25, 2022.
1
AMRX
Parkinson's disease
CRL
2023-07-03 00:00:00
CRL issued by the FDA on July 3, 2023.
0
ACRX
Post-operative pain following open abdominal surgery and hip or knee replacement surgery
Phase 3
2017-08-01 00:00:00
Phase 2 data released August 1, 2017.
0
BMY
Ulcerative Colitis
Approved
2021-05-27 00:00:00
FDA approval announced May 27, 2021.
1
MRK
Non-Small Cell Lung Cancer
Phase 3
2020-11-09 00:00:00
Phase 3 trial to discontinue due to futility - November 9, 2020.
-1
BBIO
Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Phase 2b
2022-06-13 00:00:00
Phase 2b treatment data presented at ENDO resulted in rapid and sustained restoration of normal mineral homeostasis, with mean values of blood calcium, urinary calcium, and blood PTH within the normal range by day 5 of therapy and sustained at 24 weeks, and was well-tolerated without any reported serious adverse events. At week 24 of treatment, 92% (12/13) of participants had achieved normal trough blood calcium levels in the absence of extra-dietary calcium supplements and active vitamin D, and 77% (10/13) of participants had normal urinary calcium excretion
1
AZN
Second line HER2+ breast cancer
Approved
2022-05-05 00:00:00
Approved May 5, 2022.
1
BMY
Mesothelin-positive solid tumors
Phase 1/2
2022-09-28 00:00:00
Phase 1 translational data demonstrated tolerability and clinical benefit. Second RECIST PR in ovarian cancer supports broad potential of treatment, consistent tumor regression in 93% evaluable patients with DCR of 77%. PFS of 5.6 months and overall survival of 11.2 months suggest durability of benefit in mesothelioma. Phase 2 portion of trial underway, noted September 28, 2022.
1
CANF
Rheumatoid arthritis
Phase 2
2020-10-06 00:00:00
Phase 3 interim analysis recommended trial to be discontinued - October 6, 2020.
-1
TCRR
Mesothelin-positive solid tumors
Phase 1/2
2022-09-28 00:00:00
Phase 1 translational data demonstrated tolerability and clinical benefit. Second RECIST PR in ovarian cancer supports broad potential of treatment, consistent tumor regression in 93% evaluable patients with DCR of 77%. PFS of 5.6 months and overall survival of 11.2 months suggest durability of benefit in mesothelioma. Phase 2 portion of trial underway, noted September 28, 2022.
1
VRNA
Maintenance treatment of COPD
Phase 2
2019-08-05 00:00:00
Phase 2 data met primary endpoint - August 5, 2019.
0
CALT
Squamous Cell Carcinoma of the Head and Neck (SCCHN)
Phase 2
2023-07-13 00:00:00
Phase 2 biomarker data reported that 7 out of the 16 evaluable patients were progression-free with either stable disease or partial response, out of which 6 were in the setanaxib arm and 1 was in the placebo arm, noted July 13, 2023.
1
ONTX
2nd-line HR-MDS (high risk myelodysplastic syndromes)
Phase 3
2020-08-24 00:00:00
Phase 3 trial did not meet primary endpoint - August 24, 2020.
1
CGEM
Non-Small Cell Lung Cancer
Phase 1/2
2021-12-16 00:00:00
Updated Phase 1/2a reported that 14 patients achieved a confirmed PR for a 39% confirmed response rate and one additional patient had a PR that was pending confirmation at the time of the data cut-off. The median duration of response was >15 months and the median progression free survival was 12 months in the initial cohort of Phase 1 patients, noted December 16, 2021.
1
IMAB
COVID-19
Phase 2
2021-08-11 00:00:00
Phase 2 interim analysis released August 11, 2021. Mechanical ventilation free (MVF) rate (83.6% vs 76.7%) by day 30, lower mortality rate (4.9% vs 13.3%) compared to placebo.
0
OBSV
Endometriosis
Phase 3
2022-05-25 00:00:00
Phase 3 data presented at SEUD reported that at 24 weeks the mean percent reduction from baseline in MBL in the non-responder groups were 72%, 76%, 75% and 74%, across all doses, noted May 25, 2022.
1
BLU
Chronic cough
Phase 1
2023-04-05 00:00:00
Phase 1 bioavailability equivalence data reported that treatment was well tolerated and demonstrated equivalent bioavailability to twice-daily Immediate Release formulation, noted April 5, 2023.
1
SLS
Acute Myeloid Leukemia (AML)
Phase 3
2022-11-14 00:00:00
The median OS is considerably longer than originally anticipated, and thus the overall duration of the REGAL study is now expected to be longer than initially predicted
0
AZN
Heart failure in patients with type-2 diabetes
Approved
2019-10-21 00:00:00
FDA Approval announced October 21, 2019.
1
JSPR
Acute myeloid leukemia/Myelodysplastic syndrome (AML/MDS)
Phase 1
2023-11-02 00:00:00
Phase 1 results from abstracts reported that 10 of 13 AML in CR and 14 of 16 MDS subjects had MRD at pre-HCT screening assessed by cytogenetics, flow cytometry, and/or next generation sequencing, noted November 2, 2023.
0
MRTX
Non-small cell lung cancer (NSCLC)
Phase 3
2023-05-24 00:00:00
Phase 3 final analysis did not met its primary endpoint of overall survival, noted May 24, 2023.
0
ABBV
Episodic Migraine
Phase 3
2023-04-21 00:00:00
Phase 3 trial met its primary and secondary endpoints, noted April 21, 2023.
0
TGTX
Relapsing forms of Multiple Sclerosis (RMS)
Phase 2a
2018-06-18 00:00:00
Phase 2a updated data released at EAN June 18, 2018. Annualized Relapse Rate (ARR) of 0.07.
0
GLPG
Systemic sclerosis
Phase 2
2020-09-10 00:00:00
Phase 2 trial met primary endpoint - September 10, 2020.
0
OVID
CDKL5 Deficiency
Phase 2
2020-09-30 00:00:00
Phase 2 data released September 30, 2020. Median motor seizure frequency reduction was 24%.
1
PFE
Bladder cancer
Approved
2020-06-30 00:00:00
FDA approval announced June 30, 2020.
1
ASMB
Hepatitis B
Phase 1b
2022-12-19 00:00:00
Phase 1b data reported that in the cohort of 50 mg, six of eight HBV patients on treatment reached lower limit of quantification for HBV DNA by day 21 and patients showed a mean reduction of 3.1 logs in plasma HBV DNA, noted December 19, 2022.
1
GILD
Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia
Phase 1/2
2023-06-05 00:00:00
Additional Phase 1/2 data presented at ASCO reported that median (95% CI) OS was 14.2 mo (3.2-26.0) for pts with prior blina and NR (18.6-NE) for pts without prior blina; Grade ≥3 TRAEs occurred in 80% and 97% of pts, respectively, noted June 5, 2023.
1
MNKKQ
Jaundice
CRL
2018-08-22 00:00:00
CRL issued August 22, 2018.
0
FATE
B-Cell Chronic Lymphoma, chronic lymphocytic leukemia
Phase 1
2021-12-31 00:00:00
Phase 1 interim data released August 19, 2021. 10/14 patients (71%) achieved an objective response; 7/14 (50%) complete responses. Phase 1 data reported 5 of 6 patients achieve objective response with 4 with a complete response, noted December 13, 2021.
1
VERA
IgA Nephropathy
Phase 2b
2023-06-17 00:00:00
Phase 2b 36-week presented at ERA 2023 showed that atacicept 150 mg resulted in a delta of 43% versus placebo in mean proteinuria reduction in per-protocol analysis and demonstrated statistically significant stabilization of eGFR versus placebo in this high-risk population. Phase 3 initiated on June, noted June 17, 2023.
1
JNJ
metastatic hormone-naïve prostate cancer (mHNPC)
Approved
2018-02-08 00:00:00
sNDA approval announced February 8, 2018.
1
MESO
Steroid-refractory acute graft versus host disease (aGVHD) in children
CRL
2023-08-03 00:00:00
Additional CRL announced August 3, 2023.
0
PFE
Type 2 diabetes
Approved
2017-12-20 00:00:00
Approval announced December 20, 2017.
1
NKTX
Acute myeloid leukemia / myelodysplastic syndromes
Phase 1
2023-06-27 00:00:00
Additional Phase 1 data demonstrated that 4 of 6 patients achieved complete response (67% CR/CRi, 50% CR rate), and 2 CRs with MRD negativity 1 patient deepened response to MRD negative CRi with additional cycles, notedJune 27, 2023.
1
HALO
Breast cancer
Approved
2019-02-28 00:00:00
FDA approval announced February 28, 2019.
1
UBX
Osteoarthritis of the knee
Phase 2
2020-08-17 00:00:00
Phase 2 top-line data failed to meet primary endpoint - August 17, 2020.
1
XNCR
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, DLBCL
Phase 1
2022-12-12 00:00:00
Phase 1 data reported an overall response rate (ORR) was 52.0% (13/25), and the complete response rate was 24.0% (6/25), noted December 12, 2022.
0
TYME
Pancreatic cancer
Phase 2/3
2021-06-10 00:00:00
Phase 2/3 enrollment to be terminated and trial will be closed down. Company citing slower than expected enrollment for the decision - June 10, 2021.
-1
RXRX
Clostridium Difficile Infection
Phase 1
2023-09-05 00:00:00
Phase 1 trial achieved its primary objectives of assessing the safety, tolerability and pharmacokinetic profile of REC-3964. REC-3964 has been well tolerated with no serious adverse events (SAEs) reported, noted September 5, 2023.
1
ATHX
Ischemic stroke
Phase 3
2023-10-10 00:00:00
Phase 3 trial was insufficiently powered to achieve the primary endpoint, noted October 10, 2023.
1
LABP
Ulcerative Colitis (UC)
Phase 1b
2022-08-03 00:00:00
Phase 1b top-line data reported that no serious adverse events (SAEs) were reported, noted August 3, 2022.
0
RHHBY
1L Extensive-Stage Small Cell Lung Cancer
Phase 3
2022-06-07 00:00:00
Phase 3 results noted that the combination did not provide benefit in patients with untreated ES-SCLC, noted June 7, 2022.
1
GILD
COVID-19 Coronavirus
Approved
2022-01-21 00:00:00
Approval extended to non-hospitalized and pediatric patients January 21, 2022.
1
XENE
Acne
Phase 2
2017-03-24 00:00:00
Phase 2 data released March 24 2017 - endpoints not met.
0
EGRX
Increase blood pressure in adults
Approved
2021-12-15 00:00:00
Approved December 15, 2021.
1
MNOV
Alcohol Use Disorder (AUD)
Phase 2b
2023-06-29 00:00:00
Phase 2b data reported that treatment was not superior to placebo, noted June 29, 2023.
1
ERAS
Colorectal cancer and gastrointestinal (GI) cancer
Phase 1/2
2023-06-05 00:00:00
Initial Phase 1b/2 data showed a 50% response rate, including two confirmed partial responses (cPR) and one unconfirmed partial response (uPR), and 67% disease control rate in EC-naïve response evaluable patients at the highest dose of ERAS-007 tested (100 mg BID-QW), development deprioritized as clinical efficacy data do not support continued evaluation, noted June 5, 2023
1
IONS
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Approved
2018-10-05 00:00:00
FDA Approval announced October 5, 2018,
1
NVS
Spinal muscular atrophy (SMA) Type 1
Approved
2019-05-24 00:00:00
FDA Approval announced May 24, 2019.
1
FBIO
Papulopustular Rosacea
Phase 3
2023-07-11 00:00:00
Phase 3 topline data achieved the co-primary and all secondary endpoints and subjects completed the 16-week treatment with no significant safety issues, noted July 11, 2023.
1
PLX
Fabry disease
Phase 3
2022-05-31 00:00:00
Phase 3 data reported that most treatment emergent adverse events were mild to moderate, noted May 31, 2022.
1
ADAG
Solid tumors
Phase 1/2
2022-11-10 00:00:00
Phase 1b/2 data reported two partial responses and one confirmed response, noted November 10, 2022.
0
ADAP
Hepatocellular carcinoma
Phase 1
2021-09-05 00:00:00
Phase 1 data displayed one complete response and a disease control rate of 64% with stable disease for ≥16 weeks in two patients. Phase 1 top-line data has an acceptable safety profile. Dose escalation is complete September 5, 2021.
0
VNDA
Pneumonia associated with COVID-19
Phase 3
2020-08-18 00:00:00
Phase 3 data August 18, 2020 noted statistically significant improvement by Day 7. However, improvement was not statistically significant at Day 28.
1
DVAX
COVID-19 vaccine
Phase 2/3
2021-09-22 00:00:00
Phase 2/3 interim analysis showed primary and secondary endpoints met. 100% efficacy against severe COVID-19 & hospitalization and 84% efficacy against moderate-to-severe COVID-19 caused by any strain of SARS-CoV-2 in SPECTRA, and 79% efficacy against COVID-19 of any severity caused by the globally dominant Delta variant, noted September 22, 2021.
1
PTCT
Friedreich ataxia
Phase 3
2023-05-23 00:00:00
Phase 3 trial did not meet its primary endpoint, noted May 23, 2023.
1
ABBV
Alzheimer’s disease
Phase 2
2023-09-07 00:00:00
Phase 2 enrollment to be completed, noted September 7, 2023.
0
REGN
Atopic dermatitis 6-11 year-olds
Approved
2020-05-26 00:00:00
FDA Approval announced May 26, 2020.
1
IMRX
Solid tumors
Phase 1
2023-04-18 00:00:00
Phase 1 data presented at AARC showed that dosing was well tolerated with no dose limiting toxicities (DLTs) or serious adverse events (SAEs) observed, noted April 18, 2023.
1
WVE
Huntington’s disease
Phase 1/2
2021-03-29 00:00:00
https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-provides-update-phase-1b2a-precision-hd
0
AZN
Metastatic breast cancer
Phase 3
2023-06-12 00:00:00
Phase 3 trial results showed the combination reduced the risk of disease progression or death by 40% vs. Faslodex alone, noted June 12, 2023
1
REGN
Chronic spontaneous urticaria (CSU)
CRL
2023-10-20 00:00:00
CRL issued by the FDA, noted October 20, 2023.
0
CMMB
Liver Fibrosis
Phase 2a
2023-01-03 00:00:00
Phase 2 top-line data reported that trial met its primary endpoints, noted January 3, 2023.
0
PFE
Urothelial Carcinoma
Approved
2017-05-09 00:00:00
Approval announced May 9, 2017.
1
KPTI
Myelofibrosis
Phase 2
2021-12-11 00:00:00
Additional Phase 2 results reported that of the ten patients who were on treatment for at least 24 weeks, four (40%) patients achieved SVR of ≥35% and six (60%) patients achieved SVR of ≥25%, noted December 11, 2021.
1
INZY
ABCC6 deficiency
Phase 1/2
2023-10-13 00:00:00
Phase 1/2 interim clinical data presented at ASBMR reported that treatment was generally well-tolerated on October 13, 2023.
1
OCS
Inflammation and pain following ocular surgery
Phase 3
2023-08-08 00:00:00
Phase 3 trial met both primary endpoints, noted August 8, 2023.
0
BGNE
Chronic lymphocytic leukemia/small lymphocytic leukemia
Phase 1
2023-06-09 00:00:00
Phase 1 data presented at EHA reported that of 39 pts available for tumor assessment, 5 with NHL and 9 with CLL/SLL achieved responses (NHL: 3 partial responses [PR] at 160 mg/d, 1 PR at320 mg/d, 1 complete response [CR] at 640 mg/d; CLL/SLL: 2 CR and 2 PR at 80 mg/d, 3 PR at 160 mg/d, 2 PR at320 mg/d), noted June 9, 2023.
1
RHHBY
Spinal Muscular Atrophy (SMA) in babies
Approved
2022-05-31 00:00:00
Approved May 31, 2022.
1
AZN
Chronic obstructive pulmonary disease (COPD)
Approved
2020-07-24 00:00:00
FDA approval announced July 24, 2020.
1
APLS
Amyotrophic lateral sclerosis (ALS)
Phase 2
2023-05-25 00:00:00
Phase 2 top-line results did not meet its primary endpoint of the Combined Assessment of Function and Survival (CAFS) rank score at Week 52. The study also did not meet key secondary efficacy endpoints. As a result the study will be discontinued, noted May 25, 2023.
1
RYZB
Gastroenteropancreatic neuroendocrine tumors
Phase 1b
2023-10-22 00:00:00
Phase 1b data presented at ESMO reported that the confirmed ORR was 29.4%, with all five patients having a confirmed PR, noted October 22, 2023.
1
EVLO
Atopic dermatitis
Phase 2
2023-04-26 00:00:00
Phase 2 trial did not meet its primary endpoint, noted April 26, 2023.
1
VIR
Influenza
Phase 2
2023-07-20 00:00:00
Phase 2 trial did not meet its primary endpoint, noted July 20, 2023.
1
BDRX
Children with Newly Diagnosed Diffuse Midline Gliomas (DMGs)
Phase 1
2023-07-10 00:00:00
Phase 1 enrollment completed, noted July 10, 2023.
0
GSK
HIV
Approved
2020-08-06 00:00:00
FDA approval announced August 6, 2020.
1
AVTX
Major depressive disorder (MDD)
Phase 2
2016-11-29 00:00:00
Phase 2 top-line data released November 29, 2016 failed to meet primary endpoint.
1
IMAB
Acute Myeloid Leukemia / Myelodysplastic Syndrome
Phase 2
2022-12-12 00:00:00
Phase 2 data reported an increased CALR expression in CD33+ blasts after lemzoparlimab and AZA combination treatment and higher immune infiltrates including total, CD91+ macrophages and CD8/Treg ratio in bone marrow at baseline is associated with better clinical response, suggesting the important role of activation of tumor derived pro-phagocytic signal and effector immune cells in the anti-tumor activity mediated by combination treatment, noted December 12, 2022.
1
ADPT
Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL)
Approved
2023-05-19 00:00:00
Approved May 19, 2023.
1
HRMY
Adult cataplexy
Approved
2020-10-14 00:00:00
FDA approval announced October 14, 2020.
1
IGMS
2L Colorectal cancer
Phase 1
2023-06-05 00:00:00
Phase 1 data reported that the median progression-free survival (PFS) in the 24 patients was 5.6 months as of the data cut-off date, noted June 5, 2023.
0
LLY
Alopecia Areata
Approved
2022-06-13 00:00:00
Approval announced June 13, 2022.
1
MCRB
Recurrent C. Difficile infection
Approved
2023-04-26 00:00:00
Approved April 26, 2023.
1
CYTO
Vertigo
Phase 2
2022-08-30 00:00:00
Phase 2 data reported that trial met primary endpoint, noted August 30, 2022.
0
PTCT
SMA type 1/2/3 switching
Phase 2
2021-06-11 00:00:00
Phase 2 data reported a sustained >2-fold increase in median SMN protein levels versus baseline in all patients who received prior treatment, noted June 11, 2021.
1
MYMD
Sarcopenia and frailty
Phase 2
2023-10-04 00:00:00
Phase 2 new data showed statistically significant results across Cohort 4 (1050mg), noted October 4, 2023.
1
LNTH
Chronic pain
Approved
2016-07-19 00:00:00
Approved July 19 2016.
1
ZLAB
Gastric cancer
Phase 1/2
2023-06-01 00:00:00
Phase 1/2 data reported that the most common treatment-related adverse events (TRAEs) were gastrointestinal disorders, including nausea (11/19, 57.9%), vomiting (6/19, 31.6%), and abdominal pain (3/19, 15.8%), with no Grade ≥ 4 TRAEs were reported, noted June 1, 2023.
1
RAPT
Advanced Cancer
Phase 1/2
2023-11-03 00:00:00
Phase 1/2 update SITC showed a confirmed objective response rate (ORR) of 40% in PD-L1 positive (TPS ≥1%) patients with no prior checkpoint inhibitor therapy (CPI). Median progression-free survival (PFS) of 6.3 months in PD-L1 positive patients at time of data cut off, noted November 3, 2023
1
FWBI
Exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP)
Phase 2
2023-07-13 00:00:00
Phase 2 topline Initial data from the study indicated the enhanced adrulipase formulation was safe and well tolerated and demonstrated an improvement over prior formulations of adrulipase. However, the preliminary data also indicate that it is likely the primary efficacy endpoint was not achieved, noted July13,. 2023.
1
BMY
Mesothelioma
Approved
2020-10-02 00:00:00
FDA approval announced October 2, 2020.
1
AZN
HR Positive, HER2 Negative Breast Cancer
Phase 3
2023-10-23 00:00:00
Phase 3 data presented at ESMO showed reduction of the risk of disease progression or death by 37%, providing a 2-month median PFS benefit, and was well tolerated in post-endocrine therapy setting, noted October 23, 2023.
1
SYRS
Myelodysplastic Syndrome
Phase 2
2020-12-05 00:00:00
Phase 2 presentation December 5, 2020 at ASH. Overall response rate (ORR) was 67% (12/18).
1
RVPH
Schizophrenia
Phase 3
2023-10-30 00:00:00
Phase 3 trial met primary endpoint, noted October 30, 2023.
0
HRMY
22q11.2 Deletion Syndrome
Phase 2
2023-04-13 00:00:00
Additional Phase 2 data reported a statistically significant improvements in PARS-R, total score and all 5 subscales of ADAMS and all 5 subscales of ABC-C, noted April 13, 2023.
1
ARCT
COVID-19 variant vaccine
Phase 1/2
2022-08-18 00:00:00
Additional Phase 1/2 data reported broad neutralizing antibody response against Omicron variants of concern, including BA.5, lasting for up to at least six months after administration of low-dose (5 mcg) ARCT-154 booster, noted August 18, 2022.
0
NVS
ABSSSI
Approved
2014-08-06 00:00:00
Approved August 6, 2014 under priority review
1