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6
AZN
Neurofibromatosis type 1 plexiform neurofibromas
Approved
4/13/20
FDA Approval announced April 13, 2020.
6
GOSS
Eosinophilic asthma
Phase 2b
10/13/20
Phase 2b trial did not meet primary endpoint - October 13, 2020.
4
AZN
Risk of cardiovascular (CV) death, hospitalization for heart failure (hHF) and urgent heart failure (HF)
Approved
5/9/23
Approved May 9, 2023.
6
ABBV
Upper Limb Spasticity
Approved
7/29/21
FDA approval announced July 29, 2021.
6
INFI
Triple negative breast cancer (TNBC) and ovarian cancer
Phase 1
12/9/20
Phase 1 initial data due presented at SABCS Annual Meeting, December 9, 2020. 2/18 response rate (partial responses - PR) for doublet regimen. 5/12 responses for triplet (1 complete response, 4 had a PR).
5
MRK
Renal cell carcinoma
Approved
4/22/19
FDA approval announced April 22, 2019.
6
REGN
Stage II to IV Cutaneous Squamous Cell Carcinoma (CSCC)
Phase 2
10/16/23
Phase 2 data from ESMO reported a median duration of follow-up was 18.7 months, and 1 of 10 pts with MPR experienced recurrence, noted October 16, 2023.
5
IONS
C9orf72-associated Amyotrophic Lateral Sclerosis (ALS)
Phase 1
3/28/22
Phase 1 study did not meet any secondary efficacy endpoints and it did not demonstrate clinical benefit. These results do not support further development, noted March 28, 2022.
6
CYT
Hematologic malignancies, solid tumors, triple negative breast cancer
Phase 1
6/30/23
Phase 1 development discontinued due to dissolution, noted June 30, 2023.
4
SPPI
Non-Hodgkin’s lymphoma
Approved
9/4/09
Approved September 4, 2009.
6
PFE
Atopic dermatitis (AD)
Phase 2b
11/9/20
Phase 2b trial did not meet primary endpoint - November 9, 2020. Phase 3 trial planned.
5
EGRX
Supraventricular tachycardia (SVT) - atrial fibrillation and atrial flutter
CRL
5/31/23
CRL received on May 31, 2023.
5
EWTX
Duchenne Muscular Dystrophy (DMD)
Phase 2
10/26/23
Phase 2 cohort in boys initiated, October 26, 2023.
5
GNFT
Non-alcoholic steatohepatitis (NASH)
Phase 3
5/11/20
Phase 3 data did not meet primary endpoint - May 11, 2020.
4
MEIP
Indolent B-cell Non-Hodgkin's Lymphoma (iB-NHL) without small lymphocytic lymphoma (SLL), lymphoplasmacytic lymphoma (LPL), and Waldenström's macroglobulinemia (WM)
Phase 2
11/17/22
Phase 2 data reported a 75.4% objective response rate, and 24.6% of patients achieved a complete response, noted November 17, 2022.
6
CVAC
Solid tumors
Phase 1
11/11/22
Phase 1 data reported that five out of 30 patients (17%) experienced a partial response and no objective responses were observed in the 10 patients of the single-agent cohort, noted November 11, 2022.
6
BHC
Open angle glaucoma (OAG) or ocular hypertension (OHT)
Approved
11/2/17
Approval announced November 2, 2017.
6
ATNXQ
Metastatic breast cancer
Phase 3
11/22/21
Phase 3 post hoc analysis showed a median survival rate of 18.9 months in patients treated with Oral Paclitaxel vs 10.1 months in those treated with IV Paclitaxel, with a hazard ratio of 0.59, noted November 22, 2021.
6
ALGS
Nonalcoholic steatohepatitis (NASH)
Phase 1
3/29/23
Phase 1 data presented at the Hepatology Conference demonstrated a favorable safety and pharmacokinetic profile in healthy volunteers and subjects with hyperlipidemia, March 29, 2023.
6
ADVM
Wet age-related macular degeneration (Wet-AMD)
Phase 2
11/4/22
Phase 2 end-of-study results reported that all 2E11 participants were inflammation free at the end of the study, noted November 4, 2022.
5
TAK
Dravet Syndrome / Lennox-Gastaut Syndrome
Phase 3
4/27/23
Phase 3 data reported that 78% of patients tolerated the highest dose level well with sustained reduction in frequency of drop seizures, noted April 27, 2023.
6
SRPT
Duchenne muscular dystrophy
Approved
2/25/21
FDA approval announced February 25, 2021.
6
ANAB
Palmo-plantar pustular psoriasis
Phase 2
3/8/21
Phase 2 trial did not meet primary endpoint - March 8, 2021.
4
SLGL
Papulopustular rosacea
Phase 3
7/8/19
Phase 3 top-line data met primary endpoints - July 8, 2019.
6
AGIO
Thalassemia
Phase 2
12/12/22
Phase 2 data reported durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients, noted December 12, 2022.
6
AMGN
KRAS mutant non-small cell lung cancer (NSCLC)
Phase 1/2
10/16/23
Phase 1/2 initial data reported that the confirmed objective response rate (ORR) was 25% (3/12) across efficacy-evaluable patients and seen in both KRAS G12C inhibitor resistant (14.3%; 1/7) and naïve (40%; 2/5) patients, noted October 16, 2023.
6
PFE
Multiple Myeloma
Approved
8/14/23
FDA Approved on August 14, 2023.
6
NVS
Non-small cell lung cancer (NSCLC) with BRAF V600E mutation
Approved
6/22/17
FDA Approval announced June 22, 2017.
6
PFE
Psoriatic arthritis
Approved
12/14/17
Approval announced December 14, 2017.
6
AZN
Acute ischaemic stroke
Approved
11/6/20
FDA approval announced November 6, 2020.
6
FHTX
Metastatic Uveal Melanoma (mUM)
Phase 1
10/22/23
Phase 1 clinical data presented at ESMO reported that 1 patient treated at the 7.5 mg QD continuous dose level experienced a DLT of Grade 3 keratitis, noted October 22, 2023.
6
LLY
Chronic kidney disease (CKD)
Approved
9/22/23
FDA Approval on September 22, 2023.
6
ERAS
Advanced solid tumors
Phase 1b
4/18/23
Phase 1b data presented at AACR showed that dosing was well-tolerated with favorable pharmacokinetics and no apparent drug-drug interactions, noted April 18, 2023.
6
TAK
Hereditary angioedema (HAE) attacks in patients 2 to <6 yrs
Approved
2/3/23
FDA approved on February 3, 2023.
6
AZN
Paroxysmal Nocturnal Hemoglobinuria (PNH) - children
Approved
6/7/21
FDA approval announced June 7, 2021.
6
ABBV
Mid-Face Injection Via Cannula
Approved
9/3/19
FDA Approval announced September 3, 2019.
6
AQST
Epileptic seizures
Approved
8/31/22
Tentative approval from FDA on August 31, 2022. The tentative approval was due to to an existing FDA regulatory grant of orphan drug market exclusivity for Valtoco, a diazepam nasal spray product, Libervant is not yet eligible for marketing in the United States.
6
BHC
Acne
Approved
8/24/18
FDA Approval announced August 24, 2018.
6
AZN
COVID-19 vaccine
Phase 3
3/22/21
Data from U.S. trial noted 76% efficacy rate - March 24, 2021.
6
FOLD
Epidermolysis Bullosa (EB)
Phase 3
9/13/17
Phase 3 top-line data released September 13, 2017 - endpoints not met.
4
BGNE
Non-squamous non-small cell lung cancer (NSCLC)
Phase 3
9/17/20
Phase 3 primary endpoint met - PFS 9.7 months in Arm A compared to 7.6 months in the chemotherapy alone Arm B (p=0.0044; stratified hazard ratio [HR]=0.64.
6
BTAI
Solid Tumors
Phase 2
10/10/23
Phase 2 trial showed a median OS of 13.6 months and a 12-month survival rate of 56.5%., noted October 10, 2023.
6
SNY
Cold agglutinin disease
CRL
11/13/20
CRL issued November 13, 2020.
5
ALLO
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
BCYC
Solid tumors
Phase 1/2
2/14/23
Phase 1/2 data reported a 50% ORR and 75% benefit rate, including one complete response, noted February 14, 2023.
5
REGN
Coronavirus COVID-19
Phase 3
7/2/20
Phase 3 trial did not meet primary endpoint - July 2, 2020.
4
RMTI
Anemia
Approved
3/27/20
FDA approval announced date March 27, 2020.
6
GLPG
Relapsed/refractory Non-Hodgkin Lymphoma (rrNHL)
Phase 1/2
12/13/22
Phase 1/2 data reported an ORR of 86% and all responding patients achieved complete response (CR), noted December 13, 2022.
6
JNJ
Reduce the Risk of Recurrent Venous Thromboembolism (VTE)
Approved
10/14/19
FDA approval announced October 14, 2019.
6
MGTX
X-Linked Retinitis Pigmentosa
Phase 1/2
10/3/20
Phase 1/2 nine-month data presented at EURETINA 2020 Virtual Meeting October 3, 2020.
5
PASG
GM1 gangliosidosis (GM1)
Phase 1/2
8/7/23
Phase 1/2 initial safety data from cohorts 1-4 up to 28 months showed Dose 1 and 2 of PBGM01 were well tolerated and had a favorable safety and immunological profile, noted August 7, 2023.
6
PFE
Metastatic colorectal cancer
Approved
9/28/21
Approved September 28, 2021.
6
BHC
Psoriasis
Approved
10/8/18
Tentative FDA Approval announced October 8, 2018. Final approval expected November 2018 pending the expiration of exclusivity for a related product.
6
PTCT
SMA type 1/2/3 switching
Phase 2
6/11/21
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.
6
TGTX
Relapsing forms of Multiple Sclerosis (RMS)
Phase 2a
6/18/18
Phase 2a updated data released at EAN June 18, 2018. Annualized Relapse Rate (ARR) of 0.07.
5
BMY
Ulcerative Colitis
Approved
5/27/21
FDA approval announced May 27, 2021.
6
CLVS
Relapsed ovarian cancer patients with BRCA mutations
Phase 3
12/21/20
Phase 3 trial met primary endpoint - December 21, 2020.
6
VINC
Non-Hodgkin Lymphoma (NHL) and Chronic Lymphocytic Leukemia (CLL)
Phase 1
12/12/22
Phase 1 data reported that efficacy signals from the 5 newly enrolled pts include 1 stable disease (SD), 2 radiologic disease progression, and 2 on study and who have not yet had first tumor assessment, noted December 12, 2022.
5
FBIO
Non-small cell lung cancer (NSCLC)
Phase 1/2
9/25/18
Phase 1/2 preliminary data released September 24, 2018.
5
NLSP
Narcolepsy
Phase 3
3/27/23
OLE trial data reported that the mean ESS score for these patients declined by approximately 10.2 (SD=5.83) points from their DB study baseline to month 6 in the OLE, noted March 27, 2023.
5
PLUR
Intermittent claudication, or IC
Phase 2
6/12/18
Phase 2 data released June 12, 2018 - primary endpoint met.
6
ADIL
Alcohol use disorder
Phase 3
7/11/23
FDA reviewed the safety data from the ONWARD trial and did not express any concerns with the data, noted July 11, 2023.
5
CINC
Treatment Resistant Hypertension (rHTN)
Phase 2
11/7/22
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.
6
ZNTL
Uterine serous carcinoma (USC)
Phase 1
6/6/23
Phase 1 showed an ORR of 36.8% in heavily pretreated platinum-resistant ovarian cancer and USC patients treated with intermittent dosing, noted June 6, 2022.
6
BIIB
Alzheimer’s disease
Phase 3
9/13/19
Phase 3 trial discontinued due to unfavorable risk-benefit ratio.
4
ITRM
Complicated urinary tract infections (cUTI)
Phase 3
6/1/20
Phase 3 data did not meet primary endpoint - June 1, 2020.
4
JNJ
Newly Diagnosed Multiple Myeloma (NDMM)
Approved
6/28/19
FDA Approval announced June 28, 2019.
6
LLY
Type 2 Diabetes
Approved
5/13/22
Approved May 13, 2022.
6
SAGE
Bipolar depression
Phase 2
7/24/19
Phase 2 Part A data July 24, 2019 noted MADRS score of 15.5 points at Day 15 (n=23; p<0.0001) - secondary endpoint.
6
AZN
Asthma and comorbid nasal polyps
Phase 3
9/5/21
Phase 3 data showed an 86% reduction in the annualized asthma exacerbation rate (AAER) September 5, 2021.
6
REGN
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
MRK
Solid tumors
Phase 1/2
8/9/23
Phase 1/2 sixth dose escalation cohort data and agent expansion data to showed durable anti-cancer activity observed during monotherapy dose escalation without dose-limiting toxicities. Late-stage pancreatic cancer patient achieved 80% tumor shrinkage with complete regression observed in two of three metastatic lesions in the liver in response to single-agent MDNA11, noted August 9, 2023.
6
ONCY
Colorectal and advanced anal cancers, pancreatic cancer
Phase 1/2
11/10/22
Phase 1/2 data reported a 69% objective response rate (ORR), noted November 11, 2022.
5
GRTX
Pancreatic cancer
Phase 1/2
9/8/21
Phase 1/2 trial final results announced - GC4419 was well tolerated, with similar rates of early and late adverse events in the active and placebo arms September 8, 2021.
6
ASND
Growth Hormone Deficiency - adults
Phase 3
9/23/23
Phase 3 resultspresented at ESPE 2023 showed that the majority of children and adolescents treated once weekly with TransCon hGH (lonapegsomatropin) met or exceeded average parental height SDS at time of treatment completion or last visit, noted September 23, 2023.
6
LLY
Heart failure with preserved ejection fraction (HFpEF)
Phase 3
12/13/19
Phase 3 data did not meet primary endpoint - December 13, 2019.
4
MRK
Metastatic non-small cell lung cancer (NSCLC)
Phase 3
9/22/23
Phase 3 data reported that there was not an improvement in OS for patients who received KEYTRUDA plus LENVIMA compared to docetaxel, noted September 22, 2023.
6
KMDA
Pediatric patients newly diagnosed with type 1 diabetes
Phase 2
11/1/17
Phase 2 data released November 3, 2017. No significant treatment effect noted overall.
4
VIGL
Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP)
Phase 1
9/11/23
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.
6
VRTX
Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation
Approved
10/21/19
FDA Approval announced October 21, 2019.
6
SUPN
Epilepsy
Approved
12/14/18
FDA approval announced December 14, 2018 to expand label to include monotherapy treatment.
6
ARVN
ER+ positive / HER2- negative breast cancer
Phase 1b
12/13/21
Phase 1 dose escalation data reported that treatment was well tolerated with no DLTs at total daily doses up to 700mg, noted December 13, 2021.
6
RHHBY
Castration-resistant prostate cancer
Phase 3
6/19/20
Phase 3 data met one of two co-primary endpoints - June 19, 2020.
6
NVS
Multiple sclerosis (MS)
Approved
8/25/23
Approved August 25, 2023.
6
BBIO
Amyloid cardiomyopathy (ATTR-CM)
Phase 3
8/27/23
Phase 3 data presented at ESC reported an 81% survival rate on acoramidis and over 30 months, patients survived more and were hospitalized less than has been seen in prior controlled studies, noted August 27, 2023.
6
NVAX
COVID-19, COVID-19 vaccine, Influenza
Phase 2
5/9/23
Phase 2 data reported that the highest dose stand-alone COVID vaccine candidate achieved statistically significant anti-S IgG and neutralization responses approximately 30% higher than Novavax's prototype COVID vaccine while maintaining comparable safety, noted May 9, 2023
6
NVS
Fascioliasis
Approved
2/13/19
FDA Approval announced February 13, 2019.
6
VTVT
Type 2 Diabetes
Phase 2
12/14/16
Phase 2 primary endpoint met - December 14, 2016.
6
GSK
HIV
Approved
12/21/21
Approved December 21, 2021.
6
ZLAB
Ovarian cancer
Approved
4/29/20
FDA Approval announced April 29, 2020.
6
VALN
Lyme disease
Phase 2
9/7/23
Phase 2 data showed that participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, noted September 7, 2023.
6
KYMR
Peripheral T-cell Lymphoma (PTCL)
Phase 1a
6/14/23
Phase 1a data continue to demonstrate robust dose-dependent target knockdown in ongoing Phase 1a dose escalation clinical trials, with no dose limiting toxicities (DLTs) observed, noted June 14, 2023.
6
MRK
Castration-resistant prostate cancer -first line
Approved
6/1/23
FDA Approved on June 1, 2023.
6
BNOX
Social Anxiety Disorder (SAD)
Phase 3
10/16/23
End of Phase 2 meeting minutes resulted in favorable outcome for the company which confirmed agreement with the FDA on its Phase 3 clinical trial expected to commence in 1Q 2024.
6
ADMP
Erectile dysfunction
NDA Filing
2/26/19
Refusal to file letter received February 26, 2019.
5
BBIO
Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Phase 2b
6/13/22
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
6
AMAM
Breast cancer
Phase 2
12/9/22
Phase 2 safety and efficacy data demonstrated a 57.1% confirmed overall response rate (ORR) with 100% disease control rate (DCR), noted December 9, 2022.
6
BHC
Dry Eye Disease Associated With Meibomian Gland Dysfunction
Phase 3
4/13/21
Phase 3 trial met primary endpoints - April 13, 2021.
6
RARE
Mucopolysaccharidosis 7 (MPS 7)
Approved
11/15/17
Approval announced November 15, 2017.
6
PHVS
Hereditary angioedema - prophylactic use
Phase 1
11/10/22
Phase 1 PK data reported that food intake did not have significant effects on the time to reach therapeutic exposure, with no server or serious treatment-emergent adverse events, noted November 10, 2022.
6