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int64
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1
MITO
Healthy Volunteers, Rare neurodegenerative diseases
Phase 1
2022-11-01 00:00:00
Phase 1 data reported that treatment is generally safe and well tolerated, noted November 1, 2022.
1
CNTA
Hemophilia B
Phase 2b
2023-10-31 00:00:00
Phase 2b initiated, noted October 31, 2023.
0
KALV
Hereditary Angioedema (HAE)
Phase 1
2022-10-31 00:00:00
Phase 1 data from the study showed that the ODT tablet formulation has a similar PK profile to the film-coated version currently in development, noted October 31, 2022.
1
ARCT
COVID-19 vaccine
Phase 1/2
2020-12-28 00:00:00
Phase 1/2 data released December 28, 2020.
0
GSK
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
AZN
Paroxysmal Nocturnal Hemoglobinuria (PNH) - children
Approved
2021-06-07 00:00:00
FDA approval announced June 7, 2021.
1
CGTX
Early Alzheimer's disease
Phase 2
2023-10-27 00:00:00
Phase 2 data points to a role for the sigma-2 (σ-2) receptor in regulating key "housekeeping" processes such as autophagy that are impaired in Alzheimer's and other neurodegenerative diseases, noted October 27, 2023.
0
ZLAB
Gastrointestinal Stromal Tumors (GIST) - second-line
Phase 3
2023-06-01 00:00:00
Phase 3 data reported that the mPFS (90% CI) by IRR was 6.44 (2.89, 8.31) months and ORR was 21.1%, noted June 1, 2023.
0
ALLK
Eosinophilic Gastritis and/or eosinophilic duodenitis (EoD)
Phase 3
2022-09-09 00:00:00
Phase 3 24-week data showed that the company met its histologic co-primary endpoint but missed symptomatic co-primary endpoint, noted September 9, 2022.
1
IDYA
Solid tumors, ovarian cancer
Phase 1
2023-09-11 00:00:00
Phase 1 data reported tumor shrinkage observed in multiple HRD solid tumor patients, including an endometrial cancer subject with a first imaging assessment of a partial response and an 87% reduction of the CA-125 tumor marker, noted September 11, 2023.
1
ZLAB
Generalized Myasthenia Gravis (gMG)
Approved
2023-06-20 00:00:00
FDA Approved on June 20, 2023.
1
LEGN
Multiple myeloma
Approved
2022-02-28 00:00:00
Approved February 28, 2022.
1
GSK
Chronic rhinosinusitis with nasal polyps
Approved
2021-07-29 00:00:00
FDA approval announced July 29, 2021.
1
BLTE
Dry Age-Related Macular Degeneration (AMD)
Phase 2/3
2023-04-25 00:00:00
Phase 2 data reported that safe and well tolerated in adolescent Stargardt Disease (STGD1), noted April 25, 2023.
1
PFE
ALK+ NSCLC
Approved
2018-11-02 00:00:00
FDA approval announced November 2, 2018.
1
COLL
Pain
Approved
2015-10-25 00:00:00
Approved October 26, 2015.
1
APRE
Solid tumors
Phase 1/2
2023-10-16 00:00:00
Phase 1/2a data presented at AACR-NCI-EORTC reported that no dose-limiting toxicities have been reported to date with persistent tumor-reducing effect, noted October 16, 2023.
1
JNJ
Newly Diagnosed Multiple Myeloma (NDMM)
Approved
2019-06-28 00:00:00
FDA Approval announced June 28, 2019.
1
SRPT
Limb-girdle muscular dystrophy type 2E (LGMD2E)
Phase 1/2
2021-10-11 00:00:00
Results from most recent study found that treated participants (n=4, ages 4 to 7 years) improved 8.6 points on the NSAA compared to a matched natural history cohort 3 years after treatment- noted October 11, 2021.
1
JNJ
Plaque psoriasis
Approved
2017-10-13 00:00:00
Approval announced October 13, 2017.
1
GSK
Chronic obstructive pulmonary disease (COPD)
Approved
2017-09-18 00:00:00
Approval announced September 18, 2017.
1
IMMP
Melanoma
Phase 1
2018-05-30 00:00:00
Phase 1 data released May 30, 2018 showed ORR of 61% (11/18 patients).
1
TCON
Wet age-related macular degeneration (Wet-AMD)
Phase 2
2020-03-09 00:00:00
Development to be discontinued - March 9, 2020.
-1
ENTA
HCV - genotype 1
Approved
2014-12-19 00:00:00
Approved December 19, 2014.
1
SGEN
Squamous cell carcinoma of the head and neck (SCCHN)
Phase 2
2023-04-17 00:00:00
Phase 2 interim results presented at AACR reported a confirmed ORR of 40%, with one complete response and 5 partial responses, noted April 17, 2023.
1
NVCR
Mesothelioma
Phase 2
2018-04-17 00:00:00
Phase 2 top-line data released April 17, 2018 noted improvements in overall survival and progression free survival. Data to be presented at upcoming conference.
1
IONS
Immunoglobulin A nephropathy (IgAN)
Phase 2
2022-11-07 00:00:00
IONIS-FB-LRx achieved a 44% mean reduction in proteinuria in patients treated for 6 months
1
NCNA
Solid tumors
Phase 2
2023-10-16 00:00:00
Phase 1/2 part 2 for both as a monotherapy in solid tumors and in combination with the anti-PD-1 therapy pembrolizumab in patients with metastatic cutaneous melanoma showed that treatment has been well tolerated both as a monotherapy and in combination with pembrolizumab. Encouraging signs of efficacy, including tumor volume reductions and prolonged time on treatment have been observed in both the monotherapy and combination cohorts, noted October 16, 2023.
1
ZURA
Primary Sjogren's Syndrome
Phase 2
2022-01-27 00:00:00
Phase 2 study withdrawn noted January 27, 2022.
0
EFTR
relapsed/refractory Acute Myeloid Leukemia (AML)
Phase 1
2023-10-24 00:00:00
Phase 1 initiated, noted October 24, 2023.
0
VIGL
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
Phase 1
2023-09-11 00:00:00
Phase 2 data analysis demonstrated that VGL101 continued to have a favorable safety and tolerability profile and proof-of-target engagement in SAD/MAD cohorts up to 60 mg/kg with six-month interim data expected 4Q 2023, noted Septemeber 11, 2023.
1
KA
Solid tumors
Phase 1/2
2023-11-03 00:00:00
Phase 1/2 biomarker data to be presented at SITC, noted Noember 3 2023.
1
MOR
Plaque psoriasis
Approved
2019-02-27 00:00:00
Approval announced February 27, 2019.
1
ESPR
LDL-C and type 2 diabetes
Phase 2
2019-08-28 00:00:00
Phase 2 data met primary endpoint.
0
JNJ
Relapsed or refractory (R/R) multiple myeloma.
Approved
2022-10-26 00:00:00
Approved October 26, 2022.
1
VRDN
Thyroid Eye Disease (TED)
Phase 1/2
2023-04-27 00:00:00
Phase 1/2 clinical data reported that 80% (4/5) of VRDN-001 responders showed durability of effects at 12 weeks, noted April 27, 2023.
1
CYAD
Colorectal cancer
Phase 1b
2022-03-02 00:00:00
Phase 1b trial initiated December 16, 2021. Phase 1b trial voluntarily paused due to reports of two fatalities that presented with similar pulmonary findings, noted February 28, 2022. Clinical hold announced.
1
ATAI
Healthy volunteers
Phase 1
2023-08-08 00:00:00
Phase 1 was generally well-tolerated with no SAE reported. At the 60mg IV dose, rates of sedation and dissociation were consistent with prior studies of PCN-101 at this dose-level, noted August 8, 2023.
1
INCY
Cancer - first-line metastatic melanoma
Phase 3
2018-04-06 00:00:00
Phase 3 trial did not meet primary endpoint - noted April 6, 2018.
1
ADCT
Diffuse Large B-Cell Lymphoma
Phase 2
2023-06-09 00:00:00
Phase 2 data presented at EHA showed that 31% of patients who achieved a complete response remained event-free for two or more years and did not need new anticancer therapy, noted June 9, 2023.
1
MRK
Head and neck squamous cell carcinoma (HNSCC)
Phase 2
2021-11-09 00:00:00
Phase 2 clinical data showed that patients with 1L HNSCC who have not received prior treatment for their advanced disease (n=13), the dose demonstrates an initial ORR of 38.5% with a 12-month OS rate of 87.5% and mOS not reached in combination with pembrolizumab + 5FU + platinum. These results compare favorably with benchmark survival data from standard pembrolizumab + chemotherapy, noted November 9, 2021.
1
SLDB
Duchenne Muscular Dystrophy
Phase 1/2
2022-03-14 00:00:00
Phase 1/2 2-year data from first three patients in the high dose (2E14 vg/kg) cohort suggest sustained or improved motor function, pulmonary function and clinically validated patient-reported outcomes compared with expected declines reported by natural history data, noted March 14, 2022.
1
BMY
Renal Cell Carcinoma (RCC)
Phase 3
2022-09-08 00:00:00
Phase 3 data presented at ESMO reported that the median PFS was not reached for the triple combination and was 11.3 months for the combination of nivolumab and ipilimumab. Objective response rates in the PFS intent-to-treat population was 43% and 36%, respectively, noted September 8, 2022.
1
TEVA
Rituxan (rituximab) biosimilar
Approved
2018-11-28 00:00:00
FDA Approval announced November 28, 2018.
1
EDIT
Leber Congenital Amaurosis type 10 (LCA10)
Phase 1/2
2021-09-29 00:00:00
Phase 1/2 initial data displayed 2 (66%) subjects in the mid-dose cohort showed improvements in BCVA, FST, and/or mobility navigation. However, retinal tears were reported by 2 (50%) subjects, 1 report of hypotony in the low dose cohort, as well as 1 retinal hemorrhage. Treatment in the adult high-dose cohort continues and pediatric mid-dose cohort commencing September 29, 2021.
1
LLY
Obesity
Phase 2
2023-06-24 00:00:00
Phase 2 met primary and secondary endpoints, noted June 24, 2023.
0
ZNTL
Ovarian cancer
Phase 1b
2023-06-05 00:00:00
Phase 1b dose escalation data presented at ASCO reported that confirmed ORR of 50.0% and mPFS of 7.4 months in combination with paclitaxel, and a confirmed ORR of 35.7% and mPFS of 10.4 months in combination with carboplatin, noted June 5, 2023.
1
GRTS
1L Microsatellite-Stable Colorectal Cancer (MSS-CRC)
Phase 1/2
2022-01-13 00:00:00
Phase 1/2 data demonstrated that patients who experienced a molecular response, continue to have an OS advantage compared to those patients who did not have a molecular response. All patients alive at the time of the ESMO 2021 data presentation remain alive after an additional 22 weeks of follow-up, noted January 13, 2021.
1
IPSEY
Alagille syndrome (ALGS)
Approved
2023-06-13 00:00:00
FDA Approved on June 13, 2023.
1
ETNB
Nonalcoholic steatohepatitis (NASH)
Phase 1/2
2022-06-27 00:00:00
Additional Phase 1b/2a data presented at EASL reported that in the cohort of NASH subjects PGZ led to a meaningful reductions in extra-hepatic metabolic parameters, noted June 27, 2022.
1
ASMB
Hepatitis B
Phase 1a
2023-04-18 00:00:00
Phase 1a clinical data for the remaining 200 mg multiple dose cohort for ABI-4334 continue to demonstrate encouraging clinical profile with strong projected activity against both viral replication and cccDNA formation, noted April 18, 2023.
1
RHHBY
Duchenne Muscular Dystrophy
Approved
2023-06-22 00:00:00
FDA acelerated Approval on June 22, 2023.
1
TBPH
Gastroparesis
Phase 2b
2017-08-02 00:00:00
Phase 2b data released August 2, 2017. Primary endpoint not met.
0
PFE
Recurrent renal cell carcinoma (RCC)
Phase 3
2018-04-10 00:00:00
Phase 3 trial discontinued due to futility.
-1
ALBO
Nonalcoholic steatohepatitis (NASH)
Phase 2
2020-08-18 00:00:00
Phase 2 trial met primary endpoint. However, development in NASH will not continue - August 18, 2020.
0
ASMB
Hepatitis B virus (HBV)
Phase 2
2022-11-01 00:00:00
Phase 2 preliminary data from 65 patients randomized to receive AB-729+VBR+NA (n=32), AB-729+NA (n=17) or VBR+NA (n=16) for 48 weeks showed that adding VBR to AB-729+NA does not result in greater on-treatment improvements in markers of HBV infection as compared to AB-729+NA alone, noted November 1, 2022.
1
RLYB
Fetal and neonatal alloimmune thrombocytopenia (FNAIT)
Phase 1b
2023-06-24 00:00:00
Phase 1b data presented at ISTH showed that subcutaneous administration produced a dose-dependent, rapid and complete elimination of transfused HPA-1a positive platelets in HPA-1a negative subjects, with both doses meeting the prespecified proof-of-concept criteria of ≥90% reduction in mean platelet elimination half-life. Mean platelet elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo, noted June 24, 2023.
1
TGTX
Chronic Lymphocytic Leukemia
Phase 1/2
2021-09-20 00:00:00
Phase 1/2 data demonstrated that 47 patients have now been treated as of the data cutoff with 57% of patients previously exposed to a BTK inhibitor, best Overall Response Rate (ORR) was 100% amongst evaluable patients (n=46), including 37% complete response (CR) rate presented at iwCLL September 20, 2021.
1
NCNA
2L Colorectal cancer
Phase 2
2023-10-13 00:00:00
Phase 2 patients recruiting well with no new safety signals, noted October 13, 2023.
0
REGN
Coronavirus COVID-19
Phase 3
2020-09-01 00:00:00
Phase 3 global trial did not meet primary endpoint - September 1, 2020.
1
MYOV
Advanced prostate cancer
Approved
2020-12-18 00:00:00
FDA approval announced Decmber 18, 2020.
1
SLS
Malignant pleural mesothelioma (MPM)
Phase 1
2023-06-28 00:00:00
Phase 1 topline data reported that the median overall survival for patients who entered the study as Stage IV patients was 62.3 weeks, noted June 28, 2023.
0
AZN
Small cell lung cancer (SCLC)
Phase 3
2021-09-18 00:00:00
Phase 3 data showed sustained efficacy after a median follow up of more than three years, with a 29% reduction in the risk of death versus chemotherapy alone. Updated median OS was 12.9 months versus 10.5 for chemotherapy. 17.6% of patients treated with IMFINZI plus chemotherapy were alive at three years, versus 5.8% of patients treated with chemotherapy alone, September 18, 2021.
1
PFE
Transthyretin amyloid cardiomyopathy (ATTR-CM)
Approved
2019-05-06 00:00:00
FDA approval announced May 6, 2019.
1
AMGN
Alzheimer’s Disease
Phase 2/3
2019-07-11 00:00:00
Phase 2/3 trial discontinued due to lack of efficacy.
-1
ETON
Nutritional deficiencies
CRL
2020-11-12 00:00:00
CRL received October 2020 (announced November 12, 2020).
0
ZYME
HER2-positive Gastroesophageal Adenocarcinoma (GEA)
Phase 2
2021-09-16 00:00:00
Phase 2 data showed confirmed objective response rate (cORR) of 75% and disease control rate (DCR) of 89% overall, with a cORR of 93% and DCR of 100% in the proposed Phase 3 regimen noted September 16, 2021.
1
PFE
Postherpetic neuralgia (PHN)
Approved
2017-10-12 00:00:00
Approval announced October 12, 2017.
1
DRRX
Post-operative pain relief
Approved
2021-02-02 00:00:00
FDA approval announced February 2, 2021.
1
MCRB
Mild-to-Moderate Ulcerative Colitis
Phase 2b
2021-12-16 00:00:00
Phase 2b microbiome analyses reported engraftment and anticipated changes in disease-relevant metabolites post-administration were not observed, noted December 16, 2021.
1
MNPR
Soft tissue sarcoma
Phase 1b
2023-11-01 00:00:00
t, as well, which may result in further tumor size reduction, noted August 8, 2023. Phase 1b data showed that 9 out of the 14 patients have had stable disease (SD, as defined by RECIST 1.1 criteria) after camsirubicin treatment., noted November 1, 2023.
1
INCY
Atopic dermatitis
Approved
2021-09-21 00:00:00
Approved September 21, 2021.
1
NYMX
Benign prostatic hyperplasia (BPH)
Phase 3
2015-07-27 00:00:00
Phase 3 endpoints met in extension trial July 2015.
0
BCYC
Solid tumors
Phase 1/2
2022-09-06 00:00:00
Phase 1/2 dose escalation cohort data reported an overall response rates of 22% and 67% in ovarian and urothelial cancers, with one complete response, noted September 7, 2022.
0
ABBV
Preventive treatment of migraine
Approved
2023-04-18 00:00:00
Approved April 17, 2023.
1
BGNE
Cervical Cancer (CC)
Phase 2
2023-10-22 00:00:00
Phase 2 data presented at ESMO reported that the ORR was 22.5%, with 13 complete responses; 76.8% had a durable response of ≥6 mo, noted October 22, 2023.
1
URGN
Overactive Bladder (OAB)
Phase 2
2020-08-27 00:00:00
Phase 2 trial did not meet primary endpoint - August 27, 2020.
1
CLLS
Multiple myeloma
Phase 1/2
2021-12-13 00:00:00
Additional Phase 1/2 data noted a 71% overall response rate (ORR) and that 46% of patients achieved a very good partial response or better (VGPR+) including 25% complete response or stringent complete response (CR/sCR), noted December 13, 2021.
1
VNDA
Insomnia
Approved
2014-01-31 00:00:00
Approved January 31, 2014.
1
NVCR
Recurrent glioblastoma
Phase 2
2022-12-12 00:00:00
Phase 2 trial data reported that median progression-free survival was 4.5 months compared to 2.2 months in the historical control, noted December 12, 2022.
0
REGN
Multiple myeloma
Phase 2
2022-12-12 00:00:00
Phase 2 data reported a 64% response rate, with 45% of responders achieving a very good partial response or better, noted December 12, 2022.
1
ATRA
Epstein-Barr virus (EBV-PTLD) after solid organ transplant (SOT)
Phase 3
2021-11-04 00:00:00
Phase 3 data reported a 50% objective response rate (ORR), noted November 4, 2021.
0
MDNA
Recurrent Glioblastoma (GBM)
Phase 2b
2023-07-20 00:00:00
Phase 2b trial published in the journal Neuro-Oncology, and when compared to a well-matched external control, bizaxofusp more than doubled the median survival in end-stage rGBM patients, noted July 20, 2023. Phase 3 trial planned.
0
EGRX
Chronic lymphocytic leukemia; Indolent non-Hodgkin's lymphoma - cancer
Approved
2014-07-02 00:00:00
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.
1
BBIO
Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Phase 2
2023-10-09 00:00:00
Phase 2 long-term data at Month 21 demonstrate a well-tolerated safety profile, and stabilization in 10 meter walk test (10MWT), 100-m timed test (100mTT), and the North Star Assessment (NSAD) scores, noted October 9, 2023.
1
AIMD
Sjögren's syndrome
Phase 3
2022-11-03 00:00:00
Phase 3 results showed increased unstimulated whole saliva and improvements in mouth dryness and seven other SS symptoms, noted November 3, 2022.
1
JNJ
HIV in Women
Phase 2b
2021-08-31 00:00:00
Phase 2b data did not meet endpoints, study will not continue - August 31, 2021.
1
ATHX
Acute Respiratory Distress Syndrome
Phase 2
2021-08-06 00:00:00
Phase 2 open-label data released August 6, 2021. 20/28 days were ventilator free compared with 11/28 for standard therapy.
0
NKTR
Bronchiectasis
Phase 3
2017-04-05 00:00:00
Phase 3 second trial did not meet endpoint - April 5, 2017.
1
IONS
ATTR cardiomyopathy
Phase 3
2023-07-31 00:00:00
Phase 3 trial initiation announced January 10, 2020. Phase 3 enrollment completed, noted July 31, 2023.
0
IMGO
Essential Thrombocythemia
Phase 2
2022-12-12 00:00:00
Phase 2 data reported that 100% of patients treated with bomedemstat for ≥24 weeks achieved a platelet count in the normal range and 89% (25/28) achieved a durable (≥12 weeks) platelet count, noted December 12, 2022.
1
CINC
Treatment Resistant Hypertension (rHTN)
Phase 2
2022-11-07 00:00:00
Phase 2 presentation at AHA reported that iomarker activity clinically demonstrate how baxdrostat mechanistically achieves selective blood pressure lowering effects with no impact on cortisol, noted November 7, 2022.
1
GILD
HIV
Phase 2/3
2022-02-16 00:00:00
Phase 2/3 one-year results reported that 83%of participants receiving lenacapavir in combination with an optimized background regimen achieved an undetectable viral load at Week 52, noted February 16, 2022.
1
PFE
Alzheimer’s disease
Phase 2/3
2021-01-18 00:00:00
Phase 2/3 top-line data did not meet co-primary endpoints - January 18, 2021.
1
HARP
Small cell lung cancer
Phase 1/2
2023-10-23 00:00:00
Phase 1/2 data from ESMO reported a confirmed response rate 35% (11/31), across all tumor types and patient cohorts treated with 1 mg priming dose, and a 32% (6/19) confirmed response rate in patients with small cell lung cancer (SCLC), noted October 23, 2023.
1
ITRM
Complicated urinary tract infections (cUTI)
Phase 3
2020-06-01 00:00:00
Phase 3 data did not meet primary endpoint - June 1, 2020.
1
FSTX
Head and neck cancer
Phase 2
2022-08-11 00:00:00
Phase 2 data reported that treatment was well tolerated and cleared the pre-determined futility, noted August 11, 2022.
1
OTIC
Meniere's disease
Phase 3
2017-08-30 00:00:00
Phase 3 data released August 30 failed to meet primary and key secondary endpoints.
1
LLY
Obesity or overweight with weight-related comorbidities
Phase 3
2023-07-27 00:00:00
Phase 3 trial met both co-primary endpoints, noted July 27, 2023.
0
TOVX
Head and Neck Squamous Cell Carcinoma
Phase 1
2023-10-23 00:00:00
Phase 1 data shared at ESMO 23 showed enhanced patient survival, correlating with VCN-01 mediated increases in the CPS score, a key determinant of outcomes with anti-PD-(L)1 checkpoint inhibitor therapies, noted October 23, 2023.
1