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1
TMBR
Congenital Ichthyosis
Phase 3
2023-06-20 00:00:00
Phase 3 interim pharmacokinetic data indicated minimal systemic absorption of isotretinoin or its major metabolites in patients with moderate to severe forms of congenital ichthyosis (CI) who were treated with TMB-001 0.05% ointment, noted June 20, 2023.
1
INDV
Bulimia Nervosa
Phase 2
2019-02-21 00:00:00
Phase 2 data released February 21, 2019. Endpoints not met.
0
BMY
Beta-thalassemia
Approved
2019-11-08 00:00:00
FDA Approval announced November 8, 2019.
1
RARE
Glut1 DS patients with the movement disorder phenotype
Phase 3
2018-10-26 00:00:00
Phase 3 data released October 26, 2018 - endpoints not met.
0
RHHBY
Follicular lymphoma
Approved
2022-12-23 00:00:00
Approved December 23, 2022.
1
MRK
Hospital-acquired bacterial pneumonia (HABP)
Approved
2019-06-03 00:00:00
FDA approval announced June 3, 2019.
1
KYMR
Hidradenitis suppurativa / atopic dermatitis
Phase 1
2022-12-14 00:00:00
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.
1
AVEO
Third line treatment of patients with renal cell cancer
Approved
2021-03-10 00:00:00
FDA approval announced March 10, 2021.
1
RCUS
Non-small cell lung cancer (NSCLC)
Phase 2
2023-06-05 00:00:00
Phase 2 presentation at ASCO reported one complete response in the monotherapy and in the DZ combo, noted June 5, 2023.
1
IXHL
Anxiety
Phase 2
2023-03-15 00:00:00
Phase 2 data reported that the company found that there is a high probability that the total study will show a statistically significant benefit for the psilocybin treatment arm over the placebo treatment arm. This projection is made by assuming the effect size observed in the interim analysis for 29 participants is representative of the effect size through the remaining 43 participants, noted March 15, 2023.
1
ASND
Hypoparathyroidism
Phase 3
2023-10-02 00:00:00
Phase 3 clinical trial data presented at ATA, showed that 81% of adults with chronic post-surgical hypoparathyroidism treated with TransCon PTH (palopegteriparatide) achieved independence from conventional calcium and active vitamin D therapy while maintaining normal serum calcium levels during the 26-week blinded portion of the trial, compared to 6% taking placebo, noted October 2, 2023.
1
ANTX
Treatment-Refractory MAC Lung Disease
Phase 2/3
2023-09-13 00:00:00
Phase 2/3 enrollment completed, noted September 13, 2023.
0
INKT
Acute respiratory distress (ARDS) secondary to COVID-19
Phase 1
2023-05-21 00:00:00
Phase 1 data update reported a 75% survival rate in patients with severe respiratory distress treated with agenT-797, noted May 21, 2023.
1
RARE
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Approved
2020-06-30 00:00:00
FDA Approval announced June 30, 2020.
1
AMGN
Multiple Myeloma
Approved
2018-01-05 00:00:00
sBLA approval announced January 5, 2017.
1
BMY
Non-metastatic non-small cell lung cancer
Phase 3
2023-10-17 00:00:00
Phase 3 reported patients treated with neoadjuvant Opdivo and chemotherapy followed by surgery and adjuvant Opdivo, the risk of disease recurrence, progression or death was reduced by 42%, noted October 17, 2023.
1
ABBV
Presbyopia
Approved
2021-10-29 00:00:00
Approved October 29, 2021.
1
HOOK
Treatment-refractory HPV16+ cancers
Phase 1/2
2022-05-26 00:00:00
Phase 1/2 data from abstract reported that treatment was well-tolerated with adverse events being mild or moderate, noted May 26, 2022.
1
BCLI
Amytrophic lateral sclerosis (ALS)
PDUFA
2023-09-27 00:00:00
Advisory Committee meeting voted 17-1 that NurOwn did not demonstrate substantial evidence of effectiveness for the treatment of mild to moderate ALS, noted September 27, 2023.
1
SAGE
Alzheimer’s disease (AD) cognitive dysfunction and mild dementia
Phase 2
2022-04-01 00:00:00
Phase 2 study was well-tolerated and demonstrated improvement across multiple tests of executive performance as well as improvement on key tests of learning and memory, noted April 1, 2022.
1
VTRS
Primary Progressive Multiple Sclerosis (PPMS)
Phase 3
2023-04-24 00:00:00
Phase 3 data presented at AAN demonstrated the significant treatment effect of GA Depot in reducing ARR (30%, p=0.0066), strengthened by MRI endpoints, supports the use of GA Depot as a DMT for RMS patients, noted April 24, 2023.
1
IRON
Polycythemia vera (PV)
Phase 1
2023-10-03 00:00:00
Phase 1 initiated, noted October 3, 2023.
0
ATAI
Generalized Anxiety Disorder (GAD)
Phase 1
2023-04-28 00:00:00
Pharmacodynamic data indicated potential for anxiolytic activity without the significant sedative side effects associated with benzodiazepines, noted April 28, 2023.
0
NVAX
Influenza
Phase 3
2020-03-24 00:00:00
Phase 3 top-line data met primary endpoints - March 24, 2020.
0
TPTX
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
NVAX
Covid-19
Approved
2023-10-03 00:00:00
EUA approved October 3, 2023.
1
AZN
Advanced ER-positive Breast Cancer
Phase 2
2022-12-08 00:00:00
Phase 2 presentation at SABCS reported that treatment had a statistically significant and clinically meaningful improvement in progression-free survival (PFS) at both 75mg and 150mg dose levels, noted December 8, 2022.
1
RPTX
Solid Tumors
Phase 1
2023-10-13 00:00:00
Phase 1 initial data from Module 1 and 2 presented at plenary session at AACR-NCI-EORTC International Conference suggested treatment with lunresertib in combination with camonsertib could result in efficacy outcomes for patients in the gynecological cancer setting. In this area, we're still seeing unmet patient needs despite current therapies, noted October 13, 2023.
1
BHC
Eye Brightener
Approved
2017-12-22 00:00:00
Approval announced December 22, 2017.
1
CCXI
C3 glomerulopathy (C3G)
Phase 2
2020-12-21 00:00:00
Phase 2 trial did not meet primary endpoint - December 21, 2020.
1
CBAY
Non-alcoholic steatohepatitis (NASH)
Phase 2b
2020-05-11 00:00:00
Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.
0
CERE
Schizophrenia
Phase 1
2022-12-19 00:00:00
Phase 1 results reported that treatment demonstrated a mean change from baseline at week eight in 24-hour ambulatory systolic blood pressure (SBP) of -2.7 mmHg for the 10 mg QD group and -0.4 mmHg for the 30 mg QD group, noted December 19, 2022.
1
OLMA
Breast Cancer
Phase 2
2023-05-11 00:00:00
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.
1
MNKKQ
Pain
CRL
2018-12-12 00:00:00
CRL issued December 12, 2018.
0
CYTK
Hypertrophic cardiomyopathy (HCM)
Phase 2
2021-09-12 00:00:00
Phase 2 full trial data resulted in statistically significant reductions from baseline compared to placebo in the average resting left ventricular (LV) outflow tract pressure gradient and the average post-Valsalva. The observed reductions in LVOT-G were dose dependent. Patients treated with aficamten in both Cohort 1 and Cohort 2 also experienced statistically significant reductions in NT-proBNP, noted September 12, 2021.
1
GSK
HIV
Approved
2022-02-01 00:00:00
Approved February 1, 2022.
1
MRKR
Pancreatic cancer
Phase 1/2
2019-07-19 00:00:00
Phase 1/2 data at AACR noted 5/9 patients with stable disease with one complete response.
0
SNY
Nasal polyps
Approved
2019-06-26 00:00:00
FDA Approval announced June 26, 2019.
1
SGEN
Untreated locally advanced or metastatic urothelial cancer (la/mUC)
Phase 3
2023-10-23 00:00:00
Phase 3 data presented at ESMO reported a median OS of 31.5 months (95% CI: 25.4-NR) compared to 16.1 months in the chemotherapy arm, significantly prolonged OS, reducing the risk of death by 53% compared to treatment with chemotherapy, noted October 23, 2023.
1
ZLDPF
Pediatric Congenital hyperinsulinism (CHI)
Phase 3
2022-09-19 00:00:00
Phase 3 trial additional data showed that treatment significantly reduced the requirement for intravenous (IV) glucose to maintain glycemia in neonates and infants with CHI and reduced glucose requirements to levels that potentially allow for discontinuation of IV glucose support, noted September 19, 2022.
1
MRK
Human Papilloma virus vaccine
Approved
2018-10-09 00:00:00
Approval announced October 5, 2018.
1
CDAK
Solid tumors
Phase 1/2
2022-06-30 00:00:00
Phase 1/2 data reported that two patients experienced tumor regressions in both injected and non-injected lesions, including a partial response in one patient, noted June 30, 2022.
0
CNTB
Atopic dermatitis
Phase 2b
2023-03-17 00:00:00
Phase 2b data reported that at week 2, EASI decreased by -26.3% (head/neck), -26.4% (trunk), -21.6% (upper limbs) and -23.2% (lower limbs) for patients on CBP-201 300 mg Q4W treatment vs -9.5% to -15.7% with placebo, noted March 17, 2023.
1
JNJ
Pulmonary arterial hypertension (PAH)
Phase 3
2023-04-18 00:00:00
Phase 3 data reported that trial met its co-primary endpoint, with PVR change with M/T STCT was significantly greater versus macitentan, noted April 18, 2023.
0
PFE
Remicade (infliximab) biosimilar
Approved
2017-12-13 00:00:00
Approval announced December 13, 2017.
1
EXEL
Metastatic renal cell cancer (RCC)
Approved
2016-04-25 00:00:00
Approved April 25, 2016.
1
GILD
Acute Myeloid Leukemia (AML)
Phase 1/2
2023-08-21 00:00:00
Partial hold on enrollment by FDA, noted August 21, 2023.
0
FWBI
Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency
Phase 2
2021-08-18 00:00:00
Phase 2 top line data released August 18, 2021. Clinically meaningful improvements in the primary efficacy endpoint noted.
1
ABBV
Crohn's disease
Phase 3
2023-10-16 00:00:00
Phase 3 data reported that the trial met all primary and secondary endpoints, noted October 16, 2023.
0
BMRN
Hemophilia A
Phase 1/2
2022-05-31 00:00:00
Phase 1/2 5 year data reported that the mean ABR for the 4e13 vg/kg cohort was 0.7 with a mean cumulative ABR reduction of 91%, noted May 31, 2022.
1
RFL
Acute myeloid leukemia (AML)
Phase 3
2021-10-28 00:00:00
Phase 3 trial to be stopped due to lack of efficacy based on recommendation from independent data monitoring committee, noted October 28, 2021.
-1
TARS
Demodex Blepharitis
Approved
2023-07-25 00:00:00
FDA approved on July 25, 2023.
1
RHHBY
Multiple sclerosis (MS)
Phase 2
2023-10-13 00:00:00
Phase 2 showed that fenebrutinib crosses the blood-brain barrier with the potential to act directly on the chronic inflammation related to multiple sclerosis (MS). More than 90% relative reduction in new/enlarging T2 lesions and new T1 gadolinium-enhancing (Gd+) lesions with fenebrutinib beginning at eight week, noted October 13, 2023.
1
INCY
Alopecia Areata
Approved
2022-06-13 00:00:00
Approval announced June 13, 2022.
1
PRLD
Solid tumors and hematological malignancies
Phase 1
2023-10-16 00:00:00
Phase 1 data reported that 8 (27.6%) patients had stable disease (SD) as the best response and the median PFS was 1.25 months, noted October 16, 2023.
0
RHHBY
Diffuse Large B-Cell Lymphoma (DLBCL)
Phase 1/2
2021-12-11 00:00:00
Phase 1b/2 data reported an ORR of 73.0% and 51.5% complete response (CR) rate, with patients showing durable responses at ≥6 months, noted December 11, 2021.
1
NVS
Migraine
Approved
2018-05-17 00:00:00
Approval announced May 17, 2018.
1
RHHBY
Hepatocellular carcinoma
Phase 3
2023-04-17 00:00:00
Additional Phase 3 data reported that the combination reduced the risk of cancer returning by 28%, compared with active surveillance, at a median follow-up of 17.4 months, noted April 17, 2023.
1
IMGO
Myelofibrosis
Phase 2
2022-12-12 00:00:00
Phase 2 data reported that 65% (17/26) of patients showed a decrease in Total Symptom Score (TSS), 19% (5/26) showed a ≥ 50% decrease in TSS, 66% (33/50) showed spleen volume reductions from baseline, and 28% (14/50) showed a ≥ 20% spleen volume reduction, noted December 12, 2022.
1
GSK
Respiratory syncytial virus (RSV) disease, Vaccine
Phase 3
2023-10-25 00:00:00
Phase 3 trial met its co-endpoint, eliciting an immune response, noted October 25, 2023.
0
EXEL
Renal Cell Carcinoma
Phase 3
2023-03-03 00:00:00
Phase 3 trial did not meet its primary endpoint, noted March 3, 2023.
1
PFE
Influenza and COVID-19, Omicron BA.4/BA.5-adapted bivalent
Phase 1/2
2023-10-26 00:00:00
Phase 1/2 data demonstrated robust immune responses to influenza A, influenza B, and SARS-CoV-2 strains, noted October 26, 2023.
1
SAGE
Major Depressive Disorder (MDD)
Phase 3
2023-08-07 00:00:00
Phase 3 additional data reported that there were no new safety signals identified, noted August 7, 2023.
0
ONCY
Triple-negative breast cancer
Phase 2
2021-12-10 00:00:00
Phase 2 data reported that treatment is tolerated, noted December 10, 2021.
1
SPPI
Peripheral T-Cell Lymphoma cancer
Approved
2014-07-07 00:00:00
Approved July 7, 2014.
1
MACK
Small Cell Lung Cancer
Phase 3
2022-08-03 00:00:00
Phase 3 trial did not meet primary endpoint, noted August 3, 2022.
1
AMGN
Non-small cell lung cancer (NSCLC)
BLA Filing
2023-10-05 00:00:00
FDA Oncologic Drugs Advisory Committee (ODAC) voted 2-10 against the question of if the primary endpoint, progression-free survival (PFS) per blinded independent central review (BICR), be reliably interpreted in CodeBreaK200, noted October 5, 2023.
0
IMGN
Acute Myeloid Leukemia (AML)
Phase 1/2
2021-12-12 00:00:00
Phase 1b/2 data reported a objective response rate (ORR) of 48%, with a composite complete remission (CCR) rate of 30%, noted December 12, 2021.
0
NXTC
Solid tumors
Phase 1
2022-11-07 00:00:00
Phase 1 data reported that treatment is safe and well-tolerated with early signs of disease control in patients with advanced tumors, noted November 7, 2022.
1
TENX
Reduce the incidence of low cardiac output syndrome during cardiac surgery
Phase 3
2017-01-31 00:00:00
Phase 3 top line data released January 31, 2017 - primary endpoints not met.
0
URGN
Non-Muscle-Invasive Bladder Cancer
Phase 2b
2022-05-13 00:00:00
Phase 2b results reported that by 3 months, 31/44 (70%) LG NMIBC patients achieved a complete response, noted May 13, 2022.
1
NVS
Diabetic macular edema (DME)
Phase 3
2021-08-17 00:00:00
Phase 3 met its primary endpoints, noted August 17, 2021.
0
EFTR
Metastatic breast cancer (mBC)
Phase 2a
2023-05-25 00:00:00
Phase 2a data observed partial responses seen in 26% of patients, is substantially higher than we would expect for treating such heavily pretreated patients with just fulvestrant and abemaciclib, noted May 25, 2023
1
NOVN
Molluscum Contagiosum
Phase 3
2021-09-23 00:00:00
Phase 3 safety data showed TEAEs reported in greater than 5% of subjects in the SB206 treated groups were all at the application site (pain, erythema, pruritis, exfoliation, and dermatitis) with the high majority of these TEAEs being mild or moderate by severity, noted September 23, 2021.
1
GKOS
Glaucoma
Phase 3
2022-09-07 00:00:00
Phase 3 trials met primary endpoint, noted September 7, 2022.
0
GLYC
Vaso-occlusive crisis of sickle cell disease
Phase 3
2019-08-02 00:00:00
Phase 3 data failed to meet endpoints - August 2, 2019.
1
GMDA
Hematologic Malignancies
Approved
2023-04-17 00:00:00
FDA Approved on April 17, 2023.
1
INO
Ebola
Phase 1b
2023-04-16 00:00:00
Phase 1b data presented at ECCMID reported that GMTs rose significantly after boosting for each time point measured, peaking at week 2, noted April 16, 2023.
1
IOVA
Refractory metastatic melanoma
Phase 2
2022-11-10 00:00:00
Additional Phase 2 data reported that 42% of responses lasted more than 24 months, noted November 10, 2022.
0
ZYNE
Autism Spectrum Disorder (ASD)
Phase 2
2021-10-08 00:00:00
Phase 2 long term data displayed a statistically significant improvement in ABC-C Subscale scores through 38 weeks. Only 7 patients (19%) experienced an AE that was deemed to be treatment related. Of the 10 treatment-related AEs reported, 7 were application site-related (application site reaction, pruritus, and dryness) and 1 each of sleep disorder, increased appetite, and pollakiuria, noted October 8, 2021.
1
OPK
Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) and vitamin D insufficiency - Stage 3 and 4
Approved
2016-06-21 00:00:00
Announced FDA Approval June 21, 2016.
1
ABBV
Chronic migraine
Approved
2021-09-28 00:00:00
Approved September 28, 2021.
1
LLY
Atopic Dermatitis
CRL
2023-11-02 00:00:00
CRL received in the US, noted November 2, 2023.
0
MNKD
Pediatric type 1/2 diabetes
Phase 3
2022-04-27 00:00:00
Phase 3 proof-of-concept study demonstrated that a simplified 2x dose provided significant reductions in post-prandial glucose excursions (PPGE) versus the current label dosage guidelines, with no new safety concerns, noted April 27, 2022.
1
PFE
Respiratory Syncytial Virus (RSV) in infants
Approved
2023-08-21 00:00:00
FDA approval on August 21, 2023.
1
EQ
Acute graft-versus-host disease
Phase 3
2023-04-26 00:00:00
Phase 3 results reported that response at Day 29 was associated with improved progression-free survival through 1 year, noted April 26, 2023.
1
GLYC
Healthy volunteers
Phase 1a
2023-09-06 00:00:00
Phase 1a first cohort dosing initiated, noted September 6, 2023.
0
PRAX
Epilepsy
Phase 1
2023-08-07 00:00:00
Phase 1 study demonstrated pharmacodynamic activity across all dose levels for study subjects who received PRAX-628 at first administration as compared with subjects who received a placebo, noted August 7, 2023. Based on these studies and the preclinical results, Praxis intends to advance PRAX-628 into a Phase 2 study in focal epilepsy in the 1H 2024.
1
MESO
End-Stage Heart Failure with LVADs
Phase 2b
2018-11-11 00:00:00
Phase 2b trial did not meet primary endpoint - November 11, 2018.
1
COGT
Advanced Systemic Mastocytosis
Phase 2
2022-06-10 00:00:00
Phase 2 initial data reported that 11/11 patients achieved ≥50% reduction in serum tryptase levels by central assessment, with an 89% median reduction in serum tryptase and six of these patients achieved reduction to <20 ng/mL, noted June 10, 2022.
1
PCVX
Pneumococcal conjugate vaccine
Phase 2
2023-04-17 00:00:00
Phase 2 data reported an overall improvement in immune responses vs. PCV20 relative to the results from the prior Phase 2 study in adults aged 50-64, and the trial met the OPA response non-inferiority criteria(1) for 18 of 20 STs common with PCV20 and met the superiority criteria, noted April 17, 2023.
1
RHHBY
Influenza in patients 5 years and older
Approved
2022-08-11 00:00:00
Approved August 11, 2022.
1
IDYA
Non-metastatic uveal melanoma (UM)
Phase 1/2
2023-05-09 00:00:00
Phase 2 data reported that treatment showed evidence of anti-tumor activity, noted May 9, 2023.
1
BCEL
Solid tumors
Phase 1b
2023-06-05 00:00:00
Phase 1b interim update presented at ASCO showed clinical activity observed in multiple tumor types; longer progression-free survival observed in patients with high target expression; ATRC-101 continues to be well-tolerated, noted June 5, 2023.
1
PHGE
P. aeruginosa in patients with Cystic Fibrosis (CF)
Phase 1/2
2023-09-10 00:00:00
Phase 1b/2 Part 1 data presented at ERS reported that treatment was well-tolerated with no adverse events related to study drug, noted September 10, 2023.
1
CLLS
Relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL)
Phase 2
2023-06-08 00:00:00
Phase 2 data presented at EHA reported that treatment was well tolerated and clinical responses were achieved, noted June 8, 2023.
1
EWTX
dystrophinopathies including Duchenne Muscular Dystrophy in children and adolescent boys
Phase 2
2023-10-26 00:00:00
Phase 2 initiated, noted October 26, 2023.
0
NVO
Prostate cancer (CRPC)
Phase 1
2021-10-07 00:00:00
Phase 1 initial preliminary results reported include data as of Sept. 1, 2021, from eight men enrolled in the trial. The dose was administered in 28-day cycles, with 21 days of dosing followed by seven days of no dosing. (3/8) patients remain on study; (5/8) patients left the study (4 due to disease progression and 1 withdrawal of consent).
0
ANVS
Parkinson's Disease (PD)
Phase 3
2023-06-20 00:00:00
Phase 3 trial completed and expected to be concluded in November 2023, DSMB recommended the company to continue the trial as originally designed, noted June 20, 2023.
0
ABBV
Psoriatic Arthritis
Phase 3
2021-09-30 00:00:00
Phase 3 52-week data from open-label extension showed that 58% of patients achieved American College of Rheumatology 20 (ACR20) response. Among patients initially treated with risankizumab, 32% achieved ACR50 response, and 17% achieved ACR70 response at one year. Three major adverse cardiac events (MACE) were reported, noted September 30, 2021.
1