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1
HRTX
Total knee arthroplasty
Phase 2b
2018-06-21 00:00:00
Phase 2 data released June 21, 2018. Primary endpoint met.
0
RGNX
Wet AMD using suprachoroidal delivery
Phase 2
2023-07-30 00:00:00
Phase 2 additional data reported meaningful reductions in Anti-VEGF injection burden with stable to improved CRT through month 6, noted July 30, 2023.
1
GOVX
COVID-19 in immunocompromised patients
Phase 2
2023-10-30 00:00:00
Phase 2 site expansion commenced, noted Otober 30, 2023.
0
VBIV
COVID-19 vaccine
Phase 1/2
2021-06-29 00:00:00
Phase 1/2 initial data released June 29, 2021 - well-tolerated with no safety signals observed and induced neutralization titers in 100% of participants.
1
TVTX
IgA nephropathy
Approved
2023-02-17 00:00:00
FDA accelerated approval on February 17, 2023.
1
KPTI
Myelodysplastic syndrome (MDS), hypomethylating agents (HMA) refractory
Phase 2
2023-05-03 00:00:00
Phase 2 data reported that treatment demonstrated a 27% overall response rate (ORR) in the intent-to-treat (ITT) population and a 31% ORR in the efficacy evaluable population, noted May 3, 2023.
1
ESPR
Cardiovascular Diseases
Phase 3
2023-08-26 00:00:00
Phase 3 total event data presented at ESC reported that bempedoic acid shows 20% risk reduction in MACE-4 and 17% risk reduction in MACE-3, noted August 26, 2023.
1
CGEM
Acute myeloid leukemia (AML)
Phase 1
2023-06-08 00:00:00
Preliminary safety data from an ongoing first-in-human study were published in abstract form as part of the 2023 EHA Congress in June 2023.
0
CORT
Pancreatic cancer
Phase 3
2021-06-22 00:00:00
Phase 3 initial data released June 22, 2021. 2/31 (6%) partial responses. Enrollment to be stopped. Level of benefit does not justify further study.
-1
ABBV
Ulcerative Colitis
Phase 3
2023-06-15 00:00:00
Phase 3 trial met primary and secondary endpoints, noted June 15, 2023.
0
EYPT
Non-infectious uveitis
Approved
2018-10-15 00:00:00
FDA approval announced October 15, 2018.
1
GSK
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
CRL
2017-10-26 00:00:00
CRL January 11 2017. FDA Approval announced October 25, 2017 following resubmission.
1
INO
Cervical high-grade squamous intraepithelial lesions (HSIL)
Phase 3
2023-03-01 00:00:00
Phase 3 trial did not achieved in the investigational biomarker-selected population for the endpoint of lesion regression and viral clearance, noted March 1, 2023.
1
AGEN
Cervical cancer
BLA Filing
2021-10-22 00:00:00
BLA voluntarily withdrawn October 22, 2021.
0
TEVA
Tourette Syndrome
Phase 2/3
2020-02-19 00:00:00
Phase 2/3 trial did not meet primary endpoint - February 19, 2020.
1
ONCS
Melanoma - cancer
Phase 2b
2020-11-09 00:00:00
Phase 2b interim data presented at SITC November, 2020. 20% ORR.
1
SGMO
Sickle Cell Disease
Phase 1/2
2021-12-13 00:00:00
Phase 1/2 data reported that total hemoglobin stabilized by Week 26 after treatment in all four patients. Fetal hemoglobin level increased from 0.1-11% at screening to 14-39% in all four patients and was 38% in the longest-treated patient at 91 weeks, noted December 13, 2021.
1
ALDX
Allergic conjunctivitis
Phase 3
2023-06-15 00:00:00
Phase 3 top-line data met primary and all secondary endpoints, noted on June 15, 2023.
0
RHHBY
Acute uncomplicated influenza (12 years and older)
Approved
2020-11-23 00:00:00
FDA approval announced November 23, 2020.
1
UTHR
Pulmonary arterial hypertension (PAH)
Phase 3
2019-04-08 00:00:00
Phase 3 data released April 8, 2019 did not meet primary endpoint.
1
NBIX
Tourette syndrome - juvenile
Phase 2b
2018-12-12 00:00:00
Phase 2 top-line data released December 12, 2018 did not meet primary endpoint.
1
VALN
COVID-19 vaccine
Phase 3
2023-03-02 00:00:00
Phase 3 data reported that the fold decline of neutralizing antibodies over six months after a second vaccination with VLA2001 was similar to the active comparator, and less pronounced than for other licensed COVID-19 vaccines, noted March 2, 2023.
0
LIFE
COVID-19
Phase 2
2021-01-04 00:00:00
Phase 2 data released January 4, 2021. Primary safety endpoint met. Median time to recovery of 5.5 days compared to 6 days in the placebo group.
0
IPSEY
Primary biliary cholangitis (PBC)
Phase 3
2023-06-30 00:00:00
Phase 3 data reported that trial met primary endpoint, noted June 30, 2023.
0
VALN
Chikungunya Vaccine
Phase 3
2021-12-21 00:00:00
Phase 3 trial met its primary endpoint, demonstrating that three consecutively manufactured vaccine lots elicited equivalent immune responses measured by neutralizing antibody titer GMT ratios on Day 29 after vaccination.
1
ELTX
Acute Kidney Injury associated with cardiac surgery
Phase 2
2021-12-09 00:00:00
Phase 2 data demonstrated that trial did not meet its primary endpoint of percentage increase in serum creatinine based upon the area under the curve (AUC).
1
RCKT
Pyruvate Kinase Deficiency (PKD)
Phase 1
2023-05-19 00:00:00
Phase 1 data reported robust and sustained efficacy in adults and the first pediatric patient infusion of RP-L301 was well tolerated, with engraftment achieved at day +15, noted May 19, 2023.
1
VTVT
Type 1 Diabetes
Phase 2
2021-10-12 00:00:00
Phase 2 mechanistic study data demonstrated that treated patients experienced no increase in ketone levels relative to placebo during a period of acute insulin withdrawal, indicating no increased risk of ketoacidosis. Study met primary endpoint, noted October 12, 2021.
1
MRTX
Nonsquamous (NSQ) Non–Small-Cell Lung Cancer (NSCLC)
Phase 2
2021-09-20 00:00:00
Phase 2 data demonstrated that the median PFS was 5.7 months and the median overall survival (OS) was 14.9 months, with 56% and 32% of these patients alive at one year and two years, respectively. ORR was 18%, with 3% CR and 15% PR. The median DOR was 12.8 months, presented at ESMO September 20, 2021.
1
CYTK
Heart failure with reduced ejection fraction (HFrEF)
Phase 3
2022-04-03 00:00:00
Phase 3 results reported that there was no change in pVO2 in patients treated with omecamtiv mecarbil versus placebo, noted April 3, 2022.
1
OGEN
Oral mucositis (OM)
Phase 2
2020-04-15 00:00:00
Phase 2 data released April 15, 2020 did not meet primary endpoint.
1
CRVS
Renal cell cancer
Phase 1/2
2020-05-29 00:00:00
Phase 1b/2 initial data from the RCC cohort at ASCO May 29, 2020. AdenoSig positive group -ORR of 17%. AdenoSig negative group - no partial responses.
0
CYTK
Symptomatic obstructive hypertrophic cardiomyopathy (HCM)
Phase 3
2023-06-20 00:00:00
Phase 3 enrollment open, noted June 20, 2023.
0
TBPH
Neurogenic Orthostatic Hypotension (nOH)
Phase 3
2021-09-15 00:00:00
Phase 3 study did not meet its primary endpoint September 15, 2021.
1
REGN
Rheumatoid arthritis
Approved
2017-05-22 00:00:00
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
1
VIRX
Nasopharyngeal carcinoma and Epstein-Barr Virus-Positive (EBV+) Solid Tumors
Phase 1/2
2022-12-09 00:00:00
Preliminary Phase 1b/2 safety data reported no DLTS with 2/6 patients achieving stable disease, noted November 30, 2022.
1
FMTX
Acute myeloid leukemia
Approved
2022-12-01 00:00:00
Approved December 1, 2022.
1
NKTR
Urothelial carcinoma, Melanoma, Renal Cell Carcinoma and Non-Small Cell Lung Cancers
Phase 1/2
2019-11-09 00:00:00
Phase 1/2 updated data at SITC November 9, 2019 noted ORR was 53% (20/38) with 34% (13/38) complete response (CR) rate.
0
JNJ
Ulcerative colitis
Approved
2019-10-21 00:00:00
FDA Approval announced October 21, 2019.
1
PLRX
Primary sclerosing cholangitis (PSC)
Phase 2a
2023-09-26 00:00:00
Phase 2a met its primary and secondary endpoints at the 12-week treatment period, noted September 26. 2023.
1
VTRS
Dry eye disease
Approved
2021-10-18 00:00:00
Approved October 18, 2021.
1
AZN
Non-small cell lung cancer (NSCLC)
Phase 2
2021-09-17 00:00:00
Phase 2 trial met primary endpoint of confirmed ORR of 30%, and for durvalumab plus monalizumab 36%. The 10-month PFS rate was 64.8% for the durvalumab plus oleclumab combination and 72.7% for durvalumab plus monalizumab, versus 39.2% with durvalumab alone, September 17, 2021.
0
BHVN
Acute treatment of migraine
Approved
2020-02-27 00:00:00
FDA Approval announced February 27, 2020.
1
NKTR
Gram-Negative Pneumonia (INHALE 1)
Phase 3
2017-11-24 00:00:00
Phase 3 data released November 24, 2017. Endpoints not met.
0
GTHX
Second/third-line small-cell lung cancer
Approved
2021-02-12 00:00:00
FDA approval announced February 12, 2021.
1
OTLK
Wet age-related macular degeneration (wet AMD)
BLA Filing
2023-11-02 00:00:00
The FDA informed the company that an additional adequate and well-controlled clinical trial would be required for the approval of ONS-5010 for wet AMD. Resubmission of the ONS-5010 BLA as early year end 2024, noted November 2, 2023.
1
LLY
Chronic low back pain
Phase 3
2019-02-19 00:00:00
Phase 3 data released February 19, 2019. 10mg dose met primary endpoint. 5mg dose did not meet endpoint.
1
PCVX
Invasive Pneumococcal Disease (IPD)
Phase 1/2
2023-10-19 00:00:00
IND cleared by FDA, noted October 19, 2023
0
HALO
Multiple myeloma
Approved
2020-05-01 00:00:00
FDA Approval announced May 1, 2020.
1
LGND
Relapsed Multiple Myeloma
Approved
2018-06-11 00:00:00
sNDA approval announced June 11, 2018.
1
AZN
Neurofibromatosis type 1 plexiform neurofibromas
Approved
2020-04-13 00:00:00
FDA Approval announced April 13, 2020.
1
GLTO
Non-small cell lung cancer (NSCLC)
Phase 2a
2023-10-23 00:00:00
Phase 2a data reported that the objective tumor responses (defined as partial responses per RECIST criteria 1.1) were observed in three of five patients (60%) who received GB1211 for at least three weeks, noted October 23, 2023
1
ZVRA
induced Vasomotor Symptoms (iVMS) associated with menopause
Phase 1
2023-03-17 00:00:00
Phase 2a PoC did not meet its primary endpoint of ability to decrease the frequency or severity of hot flashes in postmenopausal women. As a result, the company is pausing the program until a full dataset review, noted March 17, 2023
1
NEXI
Multiple Myeloma
Phase 1/2
2021-12-12 00:00:00
Phase 1/2 data reported that treatment is well-tolerated without dose-limiting toxicities, noted December 12, 2021.
1
NVS
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 3
2022-12-13 00:00:00
Phase 3 data reported that trial met both primary endpoints, noted December 13, 2022.
0
LLY
Non-radiographic axial spondyloarthritis
Approved
2020-06-01 00:00:00
FDA Approval announced June 1, 2020.
1
IMNN
Ovarian cancer
Phase 1/2
2023-09-28 00:00:00
Phase 1/2 interim data reported efficacy trends in PFS, demonstrating a delay in disease progression in the treatment arm of approximately 33% compared with the control arm, with the hazard ratio nearing the required value. Preliminary OS data follows a similar trend, showing an approximate 9-month improvement in the treatment arm over the control arm, noted September 28, 2023.
1
OCUP
Mydriasis (RM)
Approved
2023-09-27 00:00:00
FDA Approval on September 27, 2023.
1
MDWD
Severe burns - pediatric
Phase 3
2021-07-20 00:00:00
Phase 3 top-line data met primary endpoints - July 20, 2021.
0
CYCN
Heart failure HFpEF
Phase 2
2019-10-30 00:00:00
Phase 2 trial did not meet primary endpoint - October 30, 2019.
1
KRTX
Schizophrenia
Phase 3
2023-03-20 00:00:00
Phase 3 trial met primary endpoint, noted March 20, 2023.
0
CLVS
Ovarian Cancer - First-line maintenance treatment
PDUFA
2023-06-26 00:00:00
Company went bankrupt in 2022 not information disclosed about the PDUFA.
0
RYTM
POMC deficiency obesity / Leptin Receptor Deficiency Obesity
Approved
2020-11-27 00:00:00
FDA approval announced November 27, 2020.
1
LLY
Inherited Alzheimer's disease
Phase 2/3
2020-02-10 00:00:00
Phase 3 trial did not meet primary endpoint.
1
LLY
Nonsquamous NSCLC
CRL
2022-03-24 00:00:00
CRL announced March 24, 2022.
0
IONS
Alzheimer's disease
Phase 1/2
2023-03-29 00:00:00
Phase 1b trial of 1b/2 study met its primary objective of safety and tolerability. Phase 1b LTE study results showed that dosing reduced biomarkers of soluble tau in CSF (t-tau and p-tau181) in a dose-dependent and sustained manner, with all dose groups showing approximately a 60% reduction from baseline CSF tau levels by the end of the LTE .Phase 2 trial in progress, noted March 29, 2023.
1
NBIX
Congenital Adrenal Hyperplasia (CAH) - children
Phase 3
2023-10-05 00:00:00
Phase 3 trial met primary endpoint, noted October 5, 2023.
0
MNKD
Type 1/2 diabetes
Approved
2014-06-27 00:00:00
Approved June 27, 2014.
1
CRBP
Dermatomyositis
Phase 3
2021-06-24 00:00:00
Phase 3 trial did not meet primary endpoint - June 24, 2021.
1
MIRM
Alagille Syndrome (ALGS)
Approved
2021-09-29 00:00:00
Approved September 29, 2021.
1
ARAV
Pancreatic Adenocarcinoma
Phase 1b
2023-05-25 00:00:00
Phase 1b data from ASCO reported that the median overall survival (OS) for patients with trough levels above the minimal batiraxcept efficacious concentration is greater than 15 months, and one patient demonstrated a complete response from 10 months to 20 months and is still on study, noted May 25, 2023.
1
SRRK
Solid Tumors
Phase 1
2023-11-03 00:00:00
Phase 1 biomarker data presented at SITC showed promising anti-tumor activity in heavily pretreated clear cell renal cell carcinoma (ccRCC) patients. Objective response rate (ORR) of 21.4% and disease control rate of 57%, noted November 3, 2023.
1
MRK
Clostridium difficile infections (CDI)
Approved
2020-01-27 00:00:00
FDA Approval announced January 27, 2020.
1
CARA
Stage 3-4 Chronic Kidney Disease-Associated Pruritus
Phase 2
2019-12-03 00:00:00
Phase 2 data met primary endpoint but secondary missed - December 3, 2019.
0
IMCR
Cutaneous melanoma (mCM)
Phase 1b
2022-06-05 00:00:00
Phase 1b data to be presented at ASCO showed 1 year overall survival (OS) of approximately 75% in heavily pre-treated mCM compared to benchmark of 55%.
1
BMY
Spinal Muscular Atrophy (SMA)
Phase 3
2023-09-14 00:00:00
Phase 3 trial to continue dosing, noted November 9, 2022. Phase 3 dosing completed, noted September 14, 2023.
0
ALLO
Large B-cell lymphoma
Phase 1
2023-06-15 00:00:00
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.
1
EWTX
Duchenne Muscular Dystrophy (DMD)
Phase 2
2023-10-26 00:00:00
Phase 2 cohort in boys initiated, October 26, 2023.
0
PFE
Persistent Pulmonary Hypertension (PPHN)
Phase 3
2019-06-28 00:00:00
Phase 3 trial did not meet primary endpoint - June 28, 2019.
1
CNTX
Ovarian cancer
Phase 2
2022-06-06 00:00:00
Phase 2 data presented at ASCO reported that a 12-month PFS rate of 20.1% and a CBR of 35.7% in patients with GCT. No objective responses were observed, noted June 6, 2022.
1
OVID
Adolescents with Angelman syndrome
Phase 3
2020-12-01 00:00:00
Phase 3 trial did not meet primary endpoint - December 1, 2020.
1
AKRO
Nonalcoholic steatohepatitis (NASH)
Phase 2b
2023-10-10 00:00:00
Phase 2b top line data showed that a trend was observed for the primary endpoint of fibrosis improvement at 36 weeks, with 22% and 24% of the 28mg and 50mg EFX-treated groups, respectively, compared with 14% for placebo, noted October 10, 2023.
1
CRVS
Peripheral T-cell lymphoma (PTCL)
Phase 1b
2023-06-15 00:00:00
Phase 1/1b antitumor response data presented at the ICML demonstrated the potential of ITK inhibition to provide a novel immunotherapy mechanism of action for cancer therapy, noted June 15, 2023. Company plans to meet with FDA during 3Q 2023 to discuss Phase 3 design.
1
OCGN
COVID-19 vaccine
Phase 3
2021-07-02 00:00:00
Phase 3 data from partner Bharat Biotech noted efficacy rate of 78%; 93% against severe disease; 65% against Delta variant
0
SNY
High low-density lipoprotein (LDL) cholesterol
Approved
2017-04-25 00:00:00
sBLA Approval announced April 25, 2017.
1
TAK
Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL)
Phase 3
2023-06-10 00:00:00
Phase 3 data presented at EHA showed that Ponatinib was superior to imatinib in combination with reduced-intensity chemotherapy in the front-line setting for patients with Ph+ ALL, with a significantly higher MRD-neg CR rate at EOI. Ponatinib was associated with deeper and more durable responses, with a trend toward improved EFS and comparable safety vs imatinib, noted June 9, 2023.
1
IMRA
b-thalassemia
Phase 2b
2021-11-16 00:00:00
Phase 2b interim data showed an uptrend observed in transfusion-dependent subjects treated with higher dose for reduced transfusion burden. The dose was generally well-tolerated in this patient population.
1
VIR
COVID-19 antibody
Approved
2021-05-26 00:00:00
FDA Emergency Use Authorization filing approval announced May 26, 2021.
1
AMGN
Migraine
Approved
2018-05-17 00:00:00
Approval announced May 17, 2018.
1
GMAB
Cervical Cancer
Approved
2021-09-20 00:00:00
Approved September 20, 2021.
1
FGEN
Pancreatic cancer
Phase 2
2017-01-17 00:00:00
Phase 2 abstract released January 17, 2017.
0
BGNE
Relapsed or refractory mantle cell lymphoma (MCL)
Approved
2019-11-14 00:00:00
FDA Approval announced November 14, 2019.
1
MIRM
Cerebrotendinous xanthomatosis (CTX)
Phase 3
2023-10-02 00:00:00
Phase 3 data met the primary endpoint of reduction in bile alcohols (urine 23S-pentol) was highly statistically significant, noted October 2, 2023.
1
TBPH
Asthma
Approved
2020-09-09 00:00:00
FDA approval announced September 9, 2020.
1
AGEN
Soft Tissue Sarcoma
Phase 2
2021-09-17 00:00:00
Phase 2 data showed in 125 patients, the objective response rate (ORR) was 26%, with 9% of patients achieving a complete response (CR), and 17% of patients achieving a partial response (PR), noted September 17, 2021.
1
NVAX
COVID-19 vaccine (booster)
Phase 3
2022-11-08 00:00:00
Phase 3 data reported that BA.1 vaccine candidate met its primary strain-change endpoint, noted November 8, 2022.
0
AMLX
Alzheimer's Disease
Phase 2
2022-04-02 00:00:00
Phase 2 results reported that treatment was well-tolerated, with a majority of treatment-emergent adverse events (TEAEs) being gastrointestinal, noted April 2, 2022.
1
CNSP
Glioblastoma (GBM) (adult)
Phase 2
2023-08-14 00:00:00
Phase 2 interim update demonstrated its capability to be an innovative treatment in GBM that is safe and well tolerated, which has the potential to be a novel and effective therapy for this disease, noted August 14, 2023.
1
AZN
Refractory generalized myasthenia gravis (gMG)
Approved
2017-10-23 00:00:00
Approval announced October 23, 2017.
1
GSK
HIV-1
Phase 3
2023-02-23 00:00:00
Phase 3b data reported that the trial achieved primary endpoint, noted February 23, 2023.
1