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6
RVMD
Solid Tumors
Phase 1
10/13/23
Phase 1 preliminary data shared at AACR-NCI-EORTC reported a median time to response of 1.3 months, and median time on treatment of 3.5 months in NSCLC, with a median time to response of 1.4 months, and median time on treatment of 2.4 months in CRC, noted October 13, 2023.
6
IMUX
Relapsing-remitting multiple sclerosis (RMS)
Phase 2
10/13/23
Phase 2 data presented at ECTRIMS reported that VidoCa was associated with a Nfl reduction from baseline up to W6 of -3% for the pooled 30 and 45mg VidoCa group compared to +2.5% for placebo, and up to W24 of -19% and +7%, respectively, noted October 13, 2023.
6
AVIR
Dengue
Phase 2
4/17/23
Additional Phase 2 data presented at ECCMID reported that AT-752 was well tolerated and had no clinically relevant effects on cardiac repolarization, heart rate, PR or QRS intervals, noted April 17, 2023.
6
INZY
ENPP1 deficiency
Phase 2
9/26/23
Phase 2 interim data from cohort 1-3 reported an improvement in the Global Impression of Change (GIC) observed in all three dose cohorts in ABCC6 Deficiency (PXE) trial, noted September 26, 2023.
6
MRK
Cervical Cancer
Approved
10/13/21
Approved October 13, 2021.
6
NVS
HR+/HER2− metastatic breast cancer
Phase 2
12/6/22
Phase 2 data reported that treatment doubled the median PFS vs. combination CT at 24.0 months compared to 12.3 months, noted December 6, 2022.
6
RHHBY
Hepatocellular Carcinoma (HCC), Solid tumors
Phase 1
4/17/23
Additional biomarker data presented at AACR reported on-target changes in gene signatures in the peripheral blood that were dependent upon drug exposure levels, noted April 17, 2023.
6
REGN
Wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR)
Approved
8/18/23
Approved August 18, 2023.
6
IONS
Cardiovascular (CV) risk reduction and hypertriglyceridemia
Phase 2b
11/24/21
Phase 2b updated data demonstrated that in the dose-ranging study in subjects with elevated non-HDL-C and triglycerides (TG), the study met its primary endpoint, achieving a statistically significant reduction in non-HDL-C at all doses tested at 24 weeks, compared to placebo, noted November 24, 2021.
6
SVRA
Nontuberculous mycobacteria (NTM)
Phase 2
3/12/20
Phase 2 top-line data released March 12, 2020.
5
ALNY
IgA nephropathy
Phase 2
8/29/22
Phase 2 new results demonstrated an equivalent 36% reduction in 24-hour urine total protein and a higher proportion, 32 vs 13%, of patients treated in comparison to placebo, respectively, achieving greater than or equal to 50% reduction in 24-hour UPCR, noted August 29, 2022.
6
SUPN
Bipolar depression
Phase 3
5/5/20
Phase 3 development has been terminated - May 5, 2020.
4
DVAX
Asthma
Phase 2a
11/29/18
Phase 2a trial did not meet primary endpoint - November 29, 2018.
4
ABBV
Non-radiographic axial spondyloarthritis
Approved
10/21/22
FDA. Approved on October 21, 2022.
6
ACOR
Parkinson’s disease (PD)
Phase 3
11/15/17
Phase 3 data due 1Q 2018. Noted November 15, 2017 seven cases of sepsis, five of which were fatal.
5
RHHBY
Third-line advanced or metastatic colorectal cancer
Phase 3
5/10/18
Phase 3 data released May 10, 2018. Primary endpoint not met.
4
GSK
Meningitis, meningococcal disease caused by serogroups A, B, C, W and Y
Phase 3
5/12/23
Phase 3 data reported that the trial met all primary endpoints, noted May 12, 2023.
5
HALO
Generalized Myasthenia Gravis (gMG)
Approved
6/20/23
FDA Approved on June 20, 2023.
6
AZN
Factor Xa inhibitor reversal agent
Approved
12/31/18
Prior Approval Supplement (PAS) FDA Approval announced December 31, 2018.
6
VRTX
Beta-thalassemia
Phase 1/2
6/11/21
Phase 1/2 data presented at EHA meeting June 11, 2021. All 15 patients were transfusion independent with follow-up ranging from 4 to 26 months after CTX001 infusion and had clinically meaningful improvements in total hemoglobin from 8.9 to 16.9 g/dL and fetal hemoglobin from 67.3% to 99.6% at last visit.
6
EGRX
Exertional heat stroke (EHS)
CRL
8/10/20
CRL announced August 10, 2020.
5
BCRX
Hereditary Angioedema
Phase 2
5/7/23
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.
6
RARE
Osteogenesis Imperfecta
Phase 2
10/14/23
Additional Phase 2 data reported that the annualized fracture rate across all 24 patients in the Phase 2 portion of the study was reduced by 67%, noted October 14, 2023.
6
KOD
Treatment-Naive Wet Age-Related Macular Degeneration (AMD)
Phase 3
10/13/23
Phase 3 data presented at the Retina Society meeting reported a favorable safety profile with low rates of intraocular inflammation and no cases of intraocular inflammation with vasculitis or vascular occlusion, noted October 13, 2023.
6
VRTX
Children with cystic fibrosis (CF) ages 1 month to less than four months old
Approved
5/3/23
FDA approved on May 3, 2023.
6
FDMT
Wet age-related macular degeneration (AMD)
Phase 1/2
7/29/23
Phase 1/2 additional dose response data demonstrated in favor of highest tested dose of 3E10 vg/eye, including 100% reduction in supplemental anti-VEGF injections (4 of 4 evaluable patients injection-free) and a clinically meaningful reduction in central subfield thickness (CST) at 36 weeks in patient population with high anti-VEGF need, noted July 29, 2023.
6
HRMY
Excessive daytime sleepiness associated with Prader-Willi Syndrome (PWS)
Phase 2
7/20/23
EoP2 meeting completed, noted July 20, 2023
6
MBIO
B-cell Non-Hodgkin Lymphoma & Chronic Lymphocytic Leukemia (CLL)
Phase 1/2
8/16/23
Initial Phase 1/2 data reported clinical responses from four of four indolent lymphoma patients, including complete response in follicular lymphoma patient previously treated with CD19 CAR-T cell therapy, noted August 16, 2023.
6
CADL
Pancreatic Cancer
Phase 2
11/3/23
Phase 2 initial overall survival and immunological biomarker data presented at SITC revealed notable improvements in patients with borderline resectable pancreatic ductal adenorcarcinoma (PDAC) after experimental treatment with CAN-2409, noted November 3, 2023.
6
JNJ
metastatic hormone-naïve prostate cancer (mHNPC)
Approved
2/8/18
sNDA approval announced February 8, 2018.
6
AZN
COVID-19
Phase 3
1/26/23
FDA no longer allows treatment to be used as Emergency Use Authorization (EUA) due to the lack of effectiveness against the currently circulating variants, noted January 26, 2023.
6
ABBV
Waldenström’s Macroglobulinemia
Approved
1/29/15
Approved January 29, 2015 - PCYC
6
EQRX
CNS metastases and EGFR-mutated non-small cell lung cancer (NSCLC)
Phase 3
6/6/22
Phase 3 data reported that CNS PFS events were observed in 11 patients (38%) treated, and the confirmed CNS ORR were 82.8% and 75.0% in patients treated with Au and G, respectively, noted June 6, 2022.
6
AGEN
Solid cancers
Phase 1
6/4/18
Phase 1 presentation at ASCO 2018. 31% ORR.
5
AGEN
Advanced Solid tumors, Colorectal cancer
Phase 1b
10/21/23
Phase 1b data presented at ESMO reported an overall response rate (ORR) of 24% was observed, compared to 2.8% reported with standard of care (SOC), and a 12-month overall survival (OS) of 74% and median OS (mOS) not yet reached, noted October 21, 2023.
6
NGM
Non-alcoholic steatohepatitis (NASH)
Phase 2b
5/24/21
Phase 2b top-line data released May 24, 2021. Primary endpoint not met.
4
ALXO
Head and neck squamous cell carcinoma (HNSCC)
Phase 2
11/9/21
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.
6
SNDX
Triple negative breast cancer (TNBC)
Phase 2
3/7/19
Phase 2 top-line data March 7, 2019 failed to meet endpoints.
4
AZN
Non-small cell lung cancer (NSCLC)
Approved
11/11/22
Approved November 11, 2022.
6
RHHBY
Relapsed/Refractory Multiple Myeloma (RRMM)
Phase 1
12/12/22
Phase 1 1 year data reported that the best overall response (BOR) achieved was: stringent complete response (sCR) in 7 patients, CR in 3 patients, very good partial response (VGPR) in 5 patients, and PR in 1 patient, noted December 12, 2022.
6
SNY
Hidradenitis suppurativa / atopic dermatitis
Phase 1
12/14/22
Phase 1 data reported that a 37% mean reduction in EASI in AD and 42% of HS patients has AN Count 0/1/2, noted December 14, 2022.
6
VBIV
Hepatitis B vaccine
Phase 3
9/21/22
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.
6
RCUS
Non-small cell lung cancer (NSCLC)
Phase 2
6/5/23
Phase 2 presentation at ASCO reported one complete response in the monotherapy and in the DZ combo, noted June 5, 2023.
6
TAK
Negative symptoms of schizophrenia
Phase 2
3/2/21
Phase 2 trial did not meet primary endpoint - March 2, 2021.
4
GILD
Large B-cell lymphoma (LBCL) after one prior line of therapy who were deemed ineligible for high-dose chemotherapy (HDCT) and autologous stem cell transplantation (ASCT)
Phase 2
9/18/23
Phase 2 trial met its primary endpoint, noted September 18, 2023.
5
CLLS
Large B-cell lymphoma
Phase 1
6/15/23
Additional data reported at ICML across all 33 patients the CR rate was 42% with 30% maintaining a CR at Month 6. Results indicated complete responses are more common with lymphodepletion regimens containing 90 mg of ALLO-647 (FCA90). Median duration of response for both the overall population and the patients treated with the Phase 2 regimen (n=12) was 23.1 months, noted June 15, 2023.
6
XERS
Type 1 diabetes
Phase 2
6/18/20
Phase 2 data released June 18, 2020. 62% reduction of hyperglycemia after a glucose challenge.
6
ARGX
Myasthenia gravis (MG)
Approved
12/17/21
Approved December 17, 2021.
6
ALDX
Dry eye disease
Phase 3
7/12/22
Phase 3 crossover results reported that met both primary endpoints, noted July 12, 2022.
5
TEVA
Osteoarthritis pain of the hip or knee
Phase 3
8/5/20
Phase 3 data achieved the co-primary endpoints for fasinumab 1 mg monthly. Fasinumab every two months did not reach statistical significance - August 5, 2020.
5
SRPT
Duchenne Muscular Dystrophy
Approved
6/22/23
FDA acelerated Approval on June 22, 2023.
6
JNCE
Non-small cell lung cancer and bladder cancer
Phase 2
11/2/20
Phase 2 interim analysis noted the trial will not meet pre-specified interim criteria for continuation of enrollment - November 2, 2020.
4
PFE
Spinal Muscular Atrophy (SMA)
Phase 3
9/14/23
Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.
5
ABBV
Preventive treatment of migraine
Approved
4/18/23
Approved April 17, 2023.
6
MRK
HER2-positive gastric or Gastroesophageal junction (GEJ) adenocarcinoma
Phase 3
10/20/23
Phase 3 trial data presented at ESMO demonstrated a statistically significant PFS improvement in advanced HER2-positive gastric or GEJ adenocarcinoma in tumors expressing PD-L1 (Combined Positive Score [CPS] ≥1) compared to trastuzumab and chemotherapy alone October 20, 2023.
6
RHHBY
Coronavirus COVID-19
Phase 3
7/29/20
Phase 3 trial did meet primary endpoint - July 29, 2020.
6
PRQR
Autosomal dominant retinitis pigmentosa (adRP)
Phase 1/2
11/18/21
Phase 1/2 data reported 3 cases of cataract worsening, with maximum benefit in BCVA observed after 5 weeks and declined thereafter. A mean BCVA benefit of +1.4 letters was reported across all subjects. In doses 75 - 300µg, the mean BCVA benefit was +5 letters, maximum benefit observed was +7 letters, noted November 18, 2021.
6
NVS
Acute myeloid leukemia (AML)
Approved
4/28/17
Priority review granted November 14, 2016. Approval announced April 28, 2017.
6
AVTX
Non-Eosinophilic Asthma (NEA)
Phase 2
6/26/23
Phase 2 did not meet the primary endpoint measured by reduction in asthma related events compared to placebo, noted June 26, 2023.
6
TPST
Hepatocellular carcinoma (HCC)
Phase 1/2
10/11/23
Phase 1b/2 trial had a confirmed objective response rate of 30% for the TPST-1120 triplet arm versus 13.3% for the atezolizumab + bevacizumab control arm; duration of response ("DoR") has not yet been reached, noted October 11, 2023.
6
HCM
Intrahepatic cholangiocarcinoma (IHCC)
Phase 2
5/25/23
Phase 2 first in human data reported that the best overall response by investigator assessment was confirmed partial response in seven (31.8%) pts and stable disease in an additional 12 pts, noted May 25, 2023.
5
CYCN
Alzheimer's disease with vascular features (ADv)
Phase 2a
5/11/23
Phase 2a data was inconclusive, noted May 11, 2023.
5
MRK
Gastric cancer
Phase 3
4/25/19
Phase 3 data released April 25, 2019 noted that monotherapy arm met noninferiority primary endpoint. Combo arm did not meet OS/PFS endpoints.
5
BIIB
Small Fiber Neuropathy
Phase 2
9/16/21
Phase 2 200mg dosing data met primary endpoint, did not meet statistical significance for secondary endpoints. Phase 2 330 mg dosing data did not meet the primary endpoint or secondary endpoints, September 16, 2021.
4
HRMY
Developmental and Epileptic Encephalopathies (DEE)
Phase 2
9/18/19
Phase 2 top-line data released September 18, 2019. 10/46 reported a serious adverse event (SAE).
4
GTHX
Second/third-line small-cell lung cancer
Approved
2/12/21
FDA approval announced February 12, 2021.
6
VRCA
Basal cell carcinoma
Phase 2
8/10/23
Phase 2 data presented at AAD Innovation Academy show consistency inclinical and histological clearance of treated BCC lesions was observed by Day 49 post-treatment with the 8 mg dose of VP-315, with 4 of 6 subjects (67%) showing complete tumor clearance, noted August 10, 2023.
6
AZN
Heart failure
Approved
5/5/20
FDA Approval announced May 5, 2020.
6
ICPT
NASH patients with compensated cirrhosis
Phase 3
8/2/23
NASH Programs discontinued, noted August 2, 2023.
4
SGEN
Frontline CD30-positive mature T-cell lymphomas - cancer
Approved
11/16/18
FDA Approval announced November 16, 2018.
6
LGND
Postpartum Depression - moderate
Approved
3/19/19
FDA approval announced March 19, 2018.
6
AADI
Open-angle glaucoma (OAG)
Phase 2
12/11/20
Phase 2 data met primary endpoint with twice-daily dose group but not with once-daily group.
6
OVID
Adolescents with Angelman syndrome
Phase 3
12/1/20
Phase 3 trial did not meet primary endpoint - December 1, 2020.
4
LPCN
Postpartum Depression
Phase 1
5/16/23
Pilot clinical PK bridge study data reported that treatment is well-tolerated with no hypoxia or sedation-related adverse events, noted May 16, 2023.
6
AZN
Paroxysmal nocturnal hemoglobinuria (PNH)
Approved
12/21/18
FDA approval announced December 21, 2018.
6
ZVRA
Vascular Ehlers-Danlos Syndrome
CRL
6/25/19
CRL issued June 25, 2019.
5
HCM
Primary immune thrombocytopenia (ITP)
Phase 3
8/21/23
Phase 3 trial met is primary endpoint, noted August 21, 2023.
5
PHGE
Acne
Phase 2
11/15/21
Program to be discontinued to focus resources on CF and AD programs, noted November 15, 2021.
4
PTCT
Spinal Muscular Atrophy (SMA) Type 1
Approved
8/7/20
FDA Approval announced August 7, 2020.
6
MRK
COVID-19
Phase 3
4/1/22
Phase 3 final analysis reported that at day 3 infectious SARS-CoV-2 was detected in 0.0% of treated patients, compared with 21.8% with placebo, noted April 1, 2022.
6
OTIC
Hearing loss
Phase 1/2
12/17/20
Phase 1/2 data released December 17, 2020. 6 out of 9 (67%) patients demonstrated a clinically-meaningful improvement on at least one of the three SIN tests at both Days 57 and 85 versus 0 out of 8 (0%) for placebo.
6
OLMA
Breast Cancer
Phase 2
5/11/23
Phase 2 data presented at ESMO Breast Cancer reported that partial responses were observed in five patients, with 59% patients remaining on treatment, noted May 11, 2023.
6
NVCT
Ovarian Clear Cell Carcinoma
Phase 1
10/13/23
Phase 1 data presented at AACR-NCI-EORTC reported that NXP800 induced phosphorylation of eIF2a via activation of the stress activated kinase GCN2, noted October 13, 2023.
6
ABBV
Marginal zone lymphoma
Approved
1/19/17
sNDA filing under priority review approved January 19, 2017.
6
GSK
Ovarian cancer
Approved
10/23/19
FDA Approval announced October 23, 2019.
6
OKYO
Dry Eye Disease
Phase 2
10/5/23
Phase 2 data reported a dropout rate at 5.4% and that treatment was well tolerated, noted October 5, 2023.
6
LIAN
Hypertrophic cardiomyopathy (HCM)
Approved
4/28/22
Approved April 28, 2022.
6
APTX
Post-traumatic stress disorder (PTSD)
Phase 2
10/19/20
Phase 2 data released October 19, 2020. Primary endpoint met.
6
PFE
Pneumococcal + Covid-19 vaccine dual administration in 65<
Phase 3
1/12/22
Phase 3 top-line results noted that responses for all 20 serotypes and to the covid-19 vaccine were similar, with a safety profile similar to the booster dose, noted January 12, 2022.
5
PFE
Generalized anxiety disorder (GAD)
Phase 3
2/10/20
Phase 3 trial did not meet primary endpoint.
4
NOVN
Acne vulgaris
Phase 3
1/27/17
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.
6
AMGN
Behçet’s Disease
Approved
7/19/19
FDA Approval announced July 19, 2019.
6
BIIB
Rheumatoid arthritis (RA)
Approved
9/29/23
FDA Biosimilar of ACTEMRA approved on September 29, 2023.
6
SPRY
Allergic reactions (type I) including anaphylaxis
CRL
9/20/23
CRL announced September 20, 2023.
5
CVM
Head and Neck cancer
Phase 3
10/30/23
Phase 3 data finalized target population data resulted in risk of death cut in half at five years versus the control, 28.6% absolute 5-year overall survival benefit versus control, 0.349 hazard ratio vs control , noted October 30, 2023.
5
ALDX
Noninfectious anterior uveitis
Phase 3
6/25/19
Phase 3 data did not meet endpoints - June 25, 2019.
4
MRK
Cervical cancer
Approved
6/12/18
Approval announced June 12, 2018.
6
NVO
Type 2 diabetes
Approved
12/5/17
Approval announced December 5, 2017.
6
CYTK
Amyotrophic lateral sclerosis (ALS)
Phase 3
3/31/23
DMC review recommended the discontinuation of the trial due to futility, noted March 31, 2023.
4
ERAS
Colorectal cancer and gastrointestinal (GI) cancer
Phase 1/2
6/5/23
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
6