roymgabriel/BioPharma · Datasets at Hugging Face

ticker stringlengths 2 5	disease stringlengths 3 196	stage stringclasses 15 values	date stringlengths 6 8	catalyst stringlengths 20 963	label int64 4 6
ABBV	Atopic dermatitis	Approved	1/14/22	Approved January 14, 2022.	6
INCY	Marginal Zone Lymphoma	NDA Filing	1/25/22	NDA voluntarily withdrawn, noted January 25, 2022	5
RHHBY	Solid tumors and ROS1-positive non-small cell lung cancer (NSCLC)	Approved	8/15/19	FDA approval announced August 15, 2019.	6
TEVA	Schizophrenia	Approved	4/28/23	Approved April 28, 2023.	6
LEGN	Multiple Myeloma	Phase 3	6/5/23	Phase 3 results shared at ASCO demonstrated a statistically significant improvement in progression-free survival, with a hazard ratio of 0.26, noted June 5, 2023	6
KNSA	Prurigo nodularis	Phase 2a	4/22/20	Phase 2a trial met primary endpoint - April 22, 2020.	6
ADCT	High-Risk Relapsed/Refractory Follicular Lymphoma	Phase 2	7/11/23	Phase 2 trial enrollment voluntarily paused after recent review of aggregate data of the 40 patients enrolled in the trial and consultation with the Data Monitoring Committee (DMC) which signaled potentially excessive respiratory-related events, noted July 11, 2023.	4
GSK	Uncomplicated urinary tract infection (uUTI)	Phase 3	4/17/23	Both EAGLE-2 and EAGLE-3 phase 3 trials met primary endpoint of non-inferiority to nitrofurantoin; EAGLE-3 demonstrated statistical superiority, noted April 17, 2023.	6
MOR	Relapsed or refractory diffuse large B cell lymphoma	Approved	7/31/20	FDA Approval announced July 31, 2020.	6
AZN	Dyslipidaemia	Phase 2b	4/4/22	Phase 2b study met its primary and secondary endpoints; ION449 was generally well tolerated, noted April 4, 2022	6
NKTR	Cancer - Metastatic Breast Cancer	Phase 3	3/17/15	Phase 3 topline data mid March 17, 2015 did not reach primary endpoint.	4
CPRX	Spinal Muscular Atrophy (SMA) Type 3	Phase 2	1/6/21	Phase 2 trial met primary endpoint. Secondary endpoints not met. Will not pursue development further.	4
MRK	Renal cell carcinoma (RCC)	Phase 3	11/1/23	Phase 3 trial met its primary endpoint, noted November 1, 2023.	5
KYMR	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
ABVC	Attention-Deficit Hyperactivity Disorder (ADHD)	Phase 2	4/26/23	Phase 2 study opened new sites, clinical trial continues to demonstrate that medicines derived from plants have significant therapeutic benefits with few, noted April 26, 2023.	6
AZN	Type 2 diabetes (Pediatric)	Approved	7/23/21	FDA approval announced July 23, 2021.	6
ALLO	Multiple myeloma	Phase 1/2	12/13/21	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.	6
FBIO	X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants	Phase 1/2	4/17/19	Phase 1/2 trial ongoing. Initial data noted in NEJM.	5
IONS	Alzheimer's disease	Phase 1/2	3/29/23	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.	6
PRAX	Major Depressive Disorder and perimenopausal depression	Phase 2a	8/16/21	Phase 2a Part B data released August 16, 2021. Mean decreases from baseline at Day 15 of 60% in frequency of moderate-to-severe hot flashes.	6
IMGN	Ovarian cancer	Phase 3	11/30/21	Phase 3 data reported that trial met primary endpoint with a confirmed objective response rate of 32.4% and median duration of response at 5.9 months, noted November 30, 2021.	6
KPTI	Diffuse Large B-Cell Lymphoma (DLBCL)	Approved	6/22/20	FDA Approval announced June 22, 2020.	6
IMAB	Acute Myeloid Leukemia / Myelodysplastic Syndrome	Phase 2	12/12/22	Phase 2 data reported an increased CALR expression in CD33+ blasts after lemzoparlimab and AZA combination treatment and higher immune infiltrates including total, CD91+ macrophages and CD8/Treg ratio in bone marrow at baseline is associated with better clinical response, suggesting the important role of activation of tumor derived pro-phagocytic signal and effector immune cells in the anti-tumor activity mediated by combination treatment, noted December 12, 2022.	6
OPK	Benign Prostatic Hyperplasia (BPH)	Phase 2b	2/1/19	Phase 2b trial suspension announced January 31, 2019.	4
HALO	Various blood cancers	Approved	6/22/17	Approval announced June 22, 2017.	6
ALBO	Nonalcoholic steatohepatitis (NASH)	Phase 2	8/18/20	Phase 2 trial met primary endpoint. However, development in NASH will not continue - August 18, 2020.	4
GILD	Ankylosing spondylitis	Phase 2	9/5/18	Phase 2 data released September 6, 2018. Primary endpoint met.	6
BMY	Myelofibrosis	Approved	8/16/19	FDA Approval announced August 16, 2019.	6
MITO	Geographic atrophy (GA) / Age-related macular degeneration (AMD)	Phase 2b	5/2/22	Phase 2b top-line data reported that trial did not meet primary endpoint, noted May 2, 2022.	6
SRRK	Spinal muscular atrophy (SMA)	Phase 2	6/30/23	Phase 2 36-month extension data presented at the Cure SMA Conference showed that continued treatment with apitegromab over the extended treatment period was associated with substantial and sustained improvement in motor function, noted June 30, 2023.	6
IONS	Dyslipidaemia	Phase 2b	4/4/22	Phase 2b study met its primary and secondary endpoints; ION449 was generally well tolerated, noted April 4, 2022	6
ABBV	Systemic Lupus Erythematosus (SLE)	Phase 2	5/31/23	Phase 2 data reported that trial met primary endpoint, noted May 31, 2023.	5
CYDY	COVID-19 (Mild to Moderate)	Phase 2	8/11/20	Phase 2 data released August 11, 2020 did not report primary endpoint data (TCSS at Day 14). Day 3 subgroup showed clinical improvement but not mentioned to be statistically significant. primary endpoint data for ALL treated patients NOT released. Secondary NEWS2 was met. No data on other secondary endpoints.	5
TCON	Angiosarcoma cancer	Phase 3	4/12/19	Phase 3 interim analysis April 12, 2019 noted trial to be terminated due to futility.	4
SLGL	Acne	Approved	7/27/21	FDA approval announced July 27, 2021.	6
TRVI	Chronic cough - Idiopathic pulmonary fibrosis (IPF)	Phase 2	9/19/22	Phase 2 full data reported that trial met primary endpoint, noted September 19, 2022.	5
BHC	Eye Brightener	Approved	12/22/17	Approval announced December 22, 2017.	6
TNXP	Post-traumatic stress disorder (PTSD)	Phase 3	12/21/20	Phase 3 primary endpoint was not met - December 21, 2020.	4
CLLS	Acute myeloid leukemia (AML)	Phase 1	5/17/23	Phase 1 preliminary data presented at ASGCT showed that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity, noted on May 17, 2023.	6
BMY	Multiple Myeloma	Phase 3	2/10/23	Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted February 10, 2023. Phase 3 topline results reported that trial met primary endpoint, noted September 7, 2022.	6
ARQT	Atopic dermatitis	Phase 3	9/7/23	OLE data reported that 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in INTEGUMENT-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively, noted September 7, 2023.	6
BLU	Chronic pruritus associated with atopic dermatitis	Phase 2b	12/13/21	Phase 2b top-line data reported that trial did not meet the primary endpoint. Development will not continue, noted December 13, 2021.	4
IMCR	Cutaneous melanoma (mCM)	Phase 1b	6/5/22	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%.	6
ATOS	Breast cancer	Phase 2	6/9/21	Phase 2 final data presented June 9, 2021. Primary endpoint met.	6
ETON	Nutritional deficiencies	CRL	11/12/20	CRL received October 2020 (announced November 12, 2020).	5
RHHBY	Cancer related diarrhea (CRD)	Phase 2	12/10/21	Phase 2 results reported that CID occurred significantly less (23%) in the crofelemer group during cycle 1 and crofelemer patients were 1.8 times more likely than control patients to have their diarrhea resolved, noted December 10, 2021.	6
MYMD	Sarcopenia and frailty	Phase 2	10/4/23	Phase 2 new data showed statistically significant results across Cohort 4 (1050mg), noted October 4, 2023.	6
AGTC	Achromatopsia (ACHMB3)	Phase 1/2	2/8/22	Phase 1/2 3-month data reported that there were no new Suspected Unexpected Serious Adverse Reactions (SUSARs) and that the inflammation continues to improve with steroid treatment, noted February 8, 2022.	6
ELOX	Cystic fibrosis	Phase 2	6/14/23	Phase 2 data demonstrated clinically relevant improvement in percent predicted forced expiratory volume (ppFEV1) based on final data assessment, noted June 14, 2023.	6
VTVT	Mild Alzheimer’s disease	Phase 3	6/12/18	Phase 3 data from Part A released April 9, 2018 did not meet endpoints. Part B data released June 12, 2018 also did not meet endpoints.	4
FRLN	Hemophilia B	Phase 1/2	12/13/21	Phase 1/2 long term data reported a dose-dependent increase in FIX activity and FIX expression was sustained in nine of 10 patients, noted December 13, 2021.	6
SCLX	Acute lower back pain (LBP)	Phase 2	9/14/23	Phase 2 data reported that none of the subjects in the active group and 3 (8.1%) subjects in placebo group had AEs of special interest, with a meaningful reduction of pain over the first week, noted September 14, 2023.	6
AMGN	Heart failure with reduced ejection fraction (HFrEF)	CRL	2/28/23	CRL issued February 28, 2023.	5
VACC	Hepatitis B (HBV)	Phase 1/2	6/21/23	Phase 1b/2a data at EASL showed that all participants who received and experienced a >0.5 log10 reduction in HBsAg had durable responses with reductions in HBsAg persisting through to the last measurement eight months post-final dose, noted June 21, 2023.	6
IPSC	Lymphoma or Indolent Lymphoma	Phase 1	11/2/23	Phase 1 data from ASH abstract reported that one patient achieved a complete response that is ongoing as of five months following their first CNTY-101 infusion, noted November 2, 2023.	6
PFE	HR+, HER2- Metastatic Breast Cancer	Approved	3/31/17	sNDA acceptance announced December 21, 2016. Approval announced March 31, 2017.	6
ENTA	Respiratory Syncytial Virus (community-acquired)	Phase 2b	10/12/23	Phase 2b data shared at IDWeek 2023 reported that EDP-938 has a high barrier to clinical resistance, a single resistant mutation was detected in 1 of 37 sequenced EDP-938-treated participants October 12, 2023.	6
ABBV	First line unfit AML	Approved	11/21/18	FDA approval announced November 21, 2018.	6
ABBV	Hepatitis C virus (HCV)	Approved	8/3/17	Approval announced August 3, 2017.	6
NVS	Metastatic breast cancer (mBC)	Phase 2	12/10/21	Phase 2 data reported median overall survival improvement of 26.4 months in cohort A. Primary endpoint was met in cohort C, noted December 10, 2021.	6
BIIB	Choroideremia	Phase 3	6/14/21	Phase 3 trial did not meet primary endpoint - June 14, 2021.	4
CHRS	Metastatic or recurrent triple-negative breast cancer (TNBC)	Phase 3	6/3/23	Phase 3 data presented at ASCO reported a statistically significant improvement in PFS by BICR was demonstrated for the toripalimab arm in the PD-L1 positive subgroup, noted June 3, 2023.	6
ASND	Hypoparathyroidism	Phase 3	10/2/23	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.	6
ABBV	Second-line Chronic graft-versus-host disease (GVHD)	Approved	8/2/17	Approval announced August 2, 2017.	6
INCY	Cholangiocarcinoma	Approved	4/17/20	FDA Approval announced April 17, 2020.	6
DRMA	Acne	Phase 2b	6/27/23	EoP2b FDA established key elements of the DMT310 Phase 3 clinical program and delineated a potential regulatory pathway for approval of DMT310, noted June 27, 2023.	6
VIRI	Long COVID-19	Phase 2	7/17/23	Phase 2 data reported clinically and statistically significant improvements in fatigue, pain, and symptoms of autonomic dysfunction, noted July 17, 2023.	6
ESPR	Hypercholesterolemia	Approved	2/26/20	FDA Approval announced February 26, 2020.	6
BHC	Psoriasis	Approved	4/25/19	FDA approval announced April 25, 2019.	6
PBYI	Breast Cancer	Phase 2	6/5/23	Phase 2 data reported an increased MYC RNA expression was observed in tumors from patients who did not derive clinical benefit from paclitaxel alone compared to those with benefit from paclitaxel alone, noted June 5, 2023.	6
CBAY	Non-alcoholic steatohepatitis (NASH)	Phase 2b	5/11/20	Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.	6
NERV	Primary insomnia	Phase 2b	6/24/19	Phase 2b data June 24, 2019 met primary endpoint.	6
PFE	Ulcerative colitis	Phase 3	3/29/22	Phase 3 data met the co-primary endpoints of clinical remission at both weeks 12 and 52 and all key secondary endpoints, noted March 29, 2022.	5
SNY	Rheumatoid arthritis	Approved	5/22/17	CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.	6
ALNY	ATTR Amyloidosis	Phase 3	5/23/22	Phase 3 18-month data reported improvements in levels of NT-proBNP and a trend towards improvement in echocardiographic parameters, noted May 23, 2022.	6
PFE	Anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC)	Phase 3	3/3/21	FDA approval announced March 3 2021.	6
KROS	Myelodysplastic syndromes (MDS)	Phase 2	11/2/23	Phase 2 data from ASH abstract reported that the median treatment duration was 166 days (range 6 to 649). Most participants (89.8%) had at least 1 treatment-emergent adverse event (TEAE), and 32.2% had TEAEs considered treatment-related, noted November 2, 2023.	6
SRPT	Duchenne muscular dystrophy	Phase 3	10/30/23	Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.	6
REGN	Atopic dermatitis 6-11 year-olds	Approved	5/26/20	FDA Approval announced May 26, 2020.	6
BMY	Hypertrophic cardiomyopathy (HCM)	Approved	4/28/22	Approved April 28, 2022.	6
HALO	Breast cancer	Approved	2/28/19	FDA approval announced February 28, 2019.	6
MOLN	Mild to moderate COVID-19	Phase 3	3/3/21	Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.	4
ZLAB	Chronic inflammatory demyelinating polyneuropathy (CIDP)	Phase 2	7/17/23	Phase 2 trial met primary endpoints, noted July 17, 2023.	5
AVTX	COVID-19 induced Acute Respiratory Distress Syndrome (ARDS)	Phase 2	3/2/21	Phase 2 data released March 2, 2021. At both the 28-day and the 60-day final timepoints, an approximately 50% trend in mortality reduction (22.5% vs 10.8% - placebo) was observed.	6
LLY	Type 2 diabetes	Phase 3	6/4/22	Phase 3 data results presented at ADA's 82nd Scientific Sessions® and simultaneously published in the New England Journal of Medicine demonstrate A1C reductions of up to 1.5% for Trulicity compared to placebo in this underserved population.	6
CLDX	Recurrent/metastatic head and neck squamous cell cancer	Phase 1/2	6/1/19	Phase 2 data ASCO June 1, 2019 noted 2/19 CRs + one PR.	5
INCY	Vitiligo	Approved	7/19/22	Approved July 19, 2022.	6
VRTX	Type 1 Diabetes	Phase 1/2	10/3/23	Phase 1/2 long term data reported that two patients with at least 1 year of follow-up met the criteria for the primary endpoint of elimination of severe hypoglycemic events (SHEs) and HbA1c <7.0%, noted October 3, 2023.	5
NBIX	Essential Tremor	Phase 2	8/4/22	Phase 2 trial did not meet primary endpoints, noted August 4, 2022.	6
CLVS	Endometrial Cancer	Phase 2	6/7/22	Phase 2 data reported that 23 subjects had clinical benefit, with 1 (4%) with CR, 9 (39%) with PR, and 13 (57%) with stable disease as best response, noted June 7, 2022.	5
GILD	Sjogren’s syndrome	Phase 2	10/24/19	Phase 2 trial did not meet primary endpoint.	4
SNY	Nasal polyps	Approved	6/26/19	FDA Approval announced June 26, 2019.	6
DCPH	Solid tumors	Phase 1	9/10/22	Phase 1 initial data presented at ESMO demonstrated a favorable tolerability profile and pharmacokinetics, and strong target inhibition across all dose levels tested, noted September 10, 2022	6
CYTK	Heart failure with reduced ejection fraction (HFrEF)	CRL	2/28/23	CRL issued February 28, 2023.	5
SMMT	C. difficile infection (CDI)	Phase 3	10/20/22	Phase 3 trial results reported that treatment was numerically higher sustained clinical response rate (73.0% vs 70.7%) than vancomycin, but did not achieve the pre-specified superiority endpoint, noted October 20, 2022.	6
NVS	Diabetic macular edema (DME)	Phase 3	8/17/21	Phase 3 met its primary endpoints, noted August 17, 2021.	6
LNTH	Chronic pain	Approved	9/29/14	Approved September 29, 2014.	6
COGT	Advanced Systemic Mastocytosis	Phase 2	6/10/22	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.	6
TAK	ALK+ Metastatic Non-Small Cell Lung Cancer	Approved	5/22/20	FDA Approval announced May 22, 2020.	6
GSK	Myelofibrosis	Approved	9/15/23	Approved September 15, 2023.	6

ABBV

Atopic dermatitis

Approved

1/14/22

Approved January 14, 2022.

6

INCY

Marginal Zone Lymphoma

NDA Filing

1/25/22

NDA voluntarily withdrawn, noted January 25, 2022

5

RHHBY

Solid tumors and ROS1-positive non-small cell lung cancer (NSCLC)

Approved

8/15/19

FDA approval announced August 15, 2019.

6

TEVA

Schizophrenia

Approved

4/28/23

Approved April 28, 2023.

6

LEGN

Multiple Myeloma

Phase 3

6/5/23

Phase 3 results shared at ASCO demonstrated a statistically significant improvement in progression-free survival, with a hazard ratio of 0.26, noted June 5, 2023

6

KNSA

Prurigo nodularis

Phase 2a

4/22/20

Phase 2a trial met primary endpoint - April 22, 2020.

6

ADCT

High-Risk Relapsed/Refractory Follicular Lymphoma

Phase 2

7/11/23

Phase 2 trial enrollment voluntarily paused after recent review of aggregate data of the 40 patients enrolled in the trial and consultation with the Data Monitoring Committee (DMC) which signaled potentially excessive respiratory-related events, noted July 11, 2023.

4

GSK

Uncomplicated urinary tract infection (uUTI)

Phase 3

4/17/23

Both EAGLE-2 and EAGLE-3 phase 3 trials met primary endpoint of non-inferiority to nitrofurantoin; EAGLE-3 demonstrated statistical superiority, noted April 17, 2023.

6

MOR

Relapsed or refractory diffuse large B cell lymphoma

Approved

7/31/20

FDA Approval announced July 31, 2020.

6

AZN

Dyslipidaemia

Phase 2b

4/4/22

Phase 2b study met its primary and secondary endpoints; ION449 was generally well tolerated, noted April 4, 2022

6

NKTR

Cancer - Metastatic Breast Cancer

Phase 3

3/17/15

Phase 3 topline data mid March 17, 2015 did not reach primary endpoint.

4

CPRX

Spinal Muscular Atrophy (SMA) Type 3

Phase 2

1/6/21

Phase 2 trial met primary endpoint. Secondary endpoints not met. Will not pursue development further.

4

MRK

Renal cell carcinoma (RCC)

Phase 3

11/1/23

Phase 3 trial met its primary endpoint, noted November 1, 2023.

5

KYMR

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

ABVC

Attention-Deficit Hyperactivity Disorder (ADHD)

Phase 2

4/26/23

Phase 2 study opened new sites, clinical trial continues to demonstrate that medicines derived from plants have significant therapeutic benefits with few, noted April 26, 2023.

6

AZN

Type 2 diabetes (Pediatric)

Approved

7/23/21

FDA approval announced July 23, 2021.

6

ALLO

Multiple myeloma

Phase 1/2

12/13/21

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.

6

FBIO

X-linked Severe Combined Immunodeficiency (XSCID) - newly diagnosed infants

Phase 1/2

4/17/19

Phase 1/2 trial ongoing. Initial data noted in NEJM.

5

IONS

Alzheimer's disease

Phase 1/2

3/29/23

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.

6

PRAX

Major Depressive Disorder and perimenopausal depression

Phase 2a

8/16/21

Phase 2a Part B data released August 16, 2021. Mean decreases from baseline at Day 15 of 60% in frequency of moderate-to-severe hot flashes.

6

IMGN

Ovarian cancer

Phase 3

11/30/21

Phase 3 data reported that trial met primary endpoint with a confirmed objective response rate of 32.4% and median duration of response at 5.9 months, noted November 30, 2021.

6

KPTI

Diffuse Large B-Cell Lymphoma (DLBCL)

Approved

6/22/20

FDA Approval announced June 22, 2020.

6

IMAB

Acute Myeloid Leukemia / Myelodysplastic Syndrome

Phase 2

12/12/22

Phase 2 data reported an increased CALR expression in CD33+ blasts after lemzoparlimab and AZA combination treatment and higher immune infiltrates including total, CD91+ macrophages and CD8/Treg ratio in bone marrow at baseline is associated with better clinical response, suggesting the important role of activation of tumor derived pro-phagocytic signal and effector immune cells in the anti-tumor activity mediated by combination treatment, noted December 12, 2022.

6

OPK

Benign Prostatic Hyperplasia (BPH)

Phase 2b

2/1/19

Phase 2b trial suspension announced January 31, 2019.

4

HALO

Various blood cancers

Approved

6/22/17

Approval announced June 22, 2017.

6

ALBO

Nonalcoholic steatohepatitis (NASH)

Phase 2

8/18/20

Phase 2 trial met primary endpoint. However, development in NASH will not continue - August 18, 2020.

4

GILD

Ankylosing spondylitis

Phase 2

9/5/18

Phase 2 data released September 6, 2018. Primary endpoint met.

6

BMY

Myelofibrosis

Approved

8/16/19

FDA Approval announced August 16, 2019.

6

MITO

Geographic atrophy (GA) / Age-related macular degeneration (AMD)

Phase 2b

5/2/22

Phase 2b top-line data reported that trial did not meet primary endpoint, noted May 2, 2022.

6

SRRK

Spinal muscular atrophy (SMA)

Phase 2

6/30/23

Phase 2 36-month extension data presented at the Cure SMA Conference showed that continued treatment with apitegromab over the extended treatment period was associated with substantial and sustained improvement in motor function, noted June 30, 2023.

6

IONS

Dyslipidaemia

Phase 2b

4/4/22

Phase 2b study met its primary and secondary endpoints; ION449 was generally well tolerated, noted April 4, 2022

6

ABBV

Systemic Lupus Erythematosus (SLE)

Phase 2

5/31/23

Phase 2 data reported that trial met primary endpoint, noted May 31, 2023.

5

CYDY

COVID-19 (Mild to Moderate)

Phase 2

8/11/20

Phase 2 data released August 11, 2020 did not report primary endpoint data (TCSS at Day 14). Day 3 subgroup showed clinical improvement but not mentioned to be statistically significant. primary endpoint data for ALL treated patients NOT released. Secondary NEWS2 was met. No data on other secondary endpoints.

5

TCON

Angiosarcoma cancer

Phase 3

4/12/19

Phase 3 interim analysis April 12, 2019 noted trial to be terminated due to futility.

4

SLGL

Acne

Approved

7/27/21

FDA approval announced July 27, 2021.

6

TRVI

Chronic cough - Idiopathic pulmonary fibrosis (IPF)

Phase 2

9/19/22

Phase 2 full data reported that trial met primary endpoint, noted September 19, 2022.

5

BHC

Eye Brightener

Approved

12/22/17

Approval announced December 22, 2017.

6

TNXP

Post-traumatic stress disorder (PTSD)

Phase 3

12/21/20

Phase 3 primary endpoint was not met - December 21, 2020.

4

CLLS

Acute myeloid leukemia (AML)

Phase 1

5/17/23

Phase 1 preliminary data presented at ASGCT showed that adding alemtuzumab to the FC LD regimen was associated with sustained lymphodepletion and significantly higher UCART123 cell expansion, which correlated with improved anti-tumor activity, noted on May 17, 2023.

6

BMY

Multiple Myeloma

Phase 3

2/10/23

Full Phase 3 data presented at EBMT-EHA 5th European CAR T-cell meeting demonstrated more than tripled progression-free survival (13.3 months vs. 4.4 months) compared with standard regimens for triple-class exposed multiple myeloma, risk of disease progression or death was reduced by 51% versus standard regimens, noted February 10, 2023. Phase 3 topline results reported that trial met primary endpoint, noted September 7, 2022.

6

ARQT

Atopic dermatitis

Phase 3

9/7/23

OLE data reported that 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in INTEGUMENT-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively, noted September 7, 2023.

6

BLU

Chronic pruritus associated with atopic dermatitis

Phase 2b

12/13/21

Phase 2b top-line data reported that trial did not meet the primary endpoint. Development will not continue, noted December 13, 2021.

4

IMCR

Cutaneous melanoma (mCM)

Phase 1b

6/5/22

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%.

6

ATOS

Breast cancer

Phase 2

6/9/21

Phase 2 final data presented June 9, 2021. Primary endpoint met.

6

ETON

Nutritional deficiencies

CRL

11/12/20

CRL received October 2020 (announced November 12, 2020).

5

RHHBY

Cancer related diarrhea (CRD)

Phase 2

12/10/21

Phase 2 results reported that CID occurred significantly less (23%) in the crofelemer group during cycle 1 and crofelemer patients were 1.8 times more likely than control patients to have their diarrhea resolved, noted December 10, 2021.

6

MYMD

Sarcopenia and frailty

Phase 2

10/4/23

Phase 2 new data showed statistically significant results across Cohort 4 (1050mg), noted October 4, 2023.

6

AGTC

Achromatopsia (ACHMB3)

Phase 1/2

2/8/22

Phase 1/2 3-month data reported that there were no new Suspected Unexpected Serious Adverse Reactions (SUSARs) and that the inflammation continues to improve with steroid treatment, noted February 8, 2022.

6

ELOX

Cystic fibrosis

Phase 2

6/14/23

Phase 2 data demonstrated clinically relevant improvement in percent predicted forced expiratory volume (ppFEV1) based on final data assessment, noted June 14, 2023.

6

VTVT

Mild Alzheimer’s disease

Phase 3

6/12/18

Phase 3 data from Part A released April 9, 2018 did not meet endpoints. Part B data released June 12, 2018 also did not meet endpoints.

4

FRLN

Hemophilia B

Phase 1/2

12/13/21

Phase 1/2 long term data reported a dose-dependent increase in FIX activity and FIX expression was sustained in nine of 10 patients, noted December 13, 2021.

6

SCLX

Acute lower back pain (LBP)

Phase 2

9/14/23

Phase 2 data reported that none of the subjects in the active group and 3 (8.1%) subjects in placebo group had AEs of special interest, with a meaningful reduction of pain over the first week, noted September 14, 2023.

6

AMGN

Heart failure with reduced ejection fraction (HFrEF)

CRL

2/28/23

CRL issued February 28, 2023.

5

VACC

Hepatitis B (HBV)

Phase 1/2

6/21/23

Phase 1b/2a data at EASL showed that all participants who received and experienced a >0.5 log10 reduction in HBsAg had durable responses with reductions in HBsAg persisting through to the last measurement eight months post-final dose, noted June 21, 2023.

6

IPSC

Lymphoma or Indolent Lymphoma

Phase 1

11/2/23

Phase 1 data from ASH abstract reported that one patient achieved a complete response that is ongoing as of five months following their first CNTY-101 infusion, noted November 2, 2023.

6

PFE

HR+, HER2- Metastatic Breast Cancer

Approved

3/31/17

sNDA acceptance announced December 21, 2016. Approval announced March 31, 2017.

6

ENTA

Respiratory Syncytial Virus (community-acquired)

Phase 2b

10/12/23

Phase 2b data shared at IDWeek 2023 reported that EDP-938 has a high barrier to clinical resistance, a single resistant mutation was detected in 1 of 37 sequenced EDP-938-treated participants October 12, 2023.

6

ABBV

First line unfit AML

Approved

11/21/18

FDA approval announced November 21, 2018.

6

ABBV

Hepatitis C virus (HCV)

Approved

8/3/17

Approval announced August 3, 2017.

6

NVS

Metastatic breast cancer (mBC)

Phase 2

12/10/21

Phase 2 data reported median overall survival improvement of 26.4 months in cohort A. Primary endpoint was met in cohort C, noted December 10, 2021.

6

BIIB

Choroideremia

Phase 3

6/14/21

Phase 3 trial did not meet primary endpoint - June 14, 2021.

4

CHRS

Metastatic or recurrent triple-negative breast cancer (TNBC)

Phase 3

6/3/23

Phase 3 data presented at ASCO reported a statistically significant improvement in PFS by BICR was demonstrated for the toripalimab arm in the PD-L1 positive subgroup, noted June 3, 2023.

6

ASND

Hypoparathyroidism

Phase 3

10/2/23

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.

6

ABBV

Second-line Chronic graft-versus-host disease (GVHD)

Approved

8/2/17

Approval announced August 2, 2017.

6

INCY

Cholangiocarcinoma

Approved

4/17/20

FDA Approval announced April 17, 2020.

6

DRMA

Acne

Phase 2b

6/27/23

EoP2b FDA established key elements of the DMT310 Phase 3 clinical program and delineated a potential regulatory pathway for approval of DMT310, noted June 27, 2023.

6

VIRI

Long COVID-19

Phase 2

7/17/23

Phase 2 data reported clinically and statistically significant improvements in fatigue, pain, and symptoms of autonomic dysfunction, noted July 17, 2023.

6

ESPR

Hypercholesterolemia

Approved

2/26/20

FDA Approval announced February 26, 2020.

6

BHC

Psoriasis

Approved

4/25/19

FDA approval announced April 25, 2019.

6

PBYI

Breast Cancer

Phase 2

6/5/23

Phase 2 data reported an increased MYC RNA expression was observed in tumors from patients who did not derive clinical benefit from paclitaxel alone compared to those with benefit from paclitaxel alone, noted June 5, 2023.

6

CBAY

Non-alcoholic steatohepatitis (NASH)

Phase 2b

5/11/20

Noted May 11, 2020 that seladelpar did not cause drug-induced liver injury as noted in November 2019.

6

NERV

Primary insomnia

Phase 2b

6/24/19

Phase 2b data June 24, 2019 met primary endpoint.

6

PFE

Ulcerative colitis

Phase 3

3/29/22

Phase 3 data met the co-primary endpoints of clinical remission at both weeks 12 and 52 and all key secondary endpoints, noted March 29, 2022.

5

SNY

Rheumatoid arthritis

Approved

5/22/17

CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.

6

ALNY

ATTR Amyloidosis

Phase 3

5/23/22

Phase 3 18-month data reported improvements in levels of NT-proBNP and a trend towards improvement in echocardiographic parameters, noted May 23, 2022.

6

PFE

Anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC)

Phase 3

3/3/21

FDA approval announced March 3 2021.

6

KROS

Myelodysplastic syndromes (MDS)

Phase 2

11/2/23

Phase 2 data from ASH abstract reported that the median treatment duration was 166 days (range 6 to 649). Most participants (89.8%) had at least 1 treatment-emergent adverse event (TEAE), and 32.2% had TEAEs considered treatment-related, noted November 2, 2023.

6

SRPT

Duchenne muscular dystrophy

Phase 3

10/30/23

Phase 3 trial results showed that participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met, noted October 30, 2023.

6

REGN

Atopic dermatitis 6-11 year-olds

Approved

5/26/20

FDA Approval announced May 26, 2020.

6

BMY

Hypertrophic cardiomyopathy (HCM)

Approved

4/28/22

Approved April 28, 2022.

6

HALO

Breast cancer

Approved

2/28/19

FDA approval announced February 28, 2019.

6

MOLN

Mild to moderate COVID-19

Phase 3

3/3/21

Independent Data and Safety Monitoring Board (DSMB) recommended that enrollment to be closed - raised concerns about the magnitude of potential benefit.

4

ZLAB

Chronic inflammatory demyelinating polyneuropathy (CIDP)

Phase 2

7/17/23

Phase 2 trial met primary endpoints, noted July 17, 2023.

5

AVTX

COVID-19 induced Acute Respiratory Distress Syndrome (ARDS)

Phase 2

3/2/21

Phase 2 data released March 2, 2021. At both the 28-day and the 60-day final timepoints, an approximately 50% trend in mortality reduction (22.5% vs 10.8% - placebo) was observed.

6

LLY

Type 2 diabetes

Phase 3

6/4/22

Phase 3 data results presented at ADA's 82nd Scientific Sessions® and simultaneously published in the New England Journal of Medicine demonstrate A1C reductions of up to 1.5% for Trulicity compared to placebo in this underserved population.

6

CLDX

Recurrent/metastatic head and neck squamous cell cancer

Phase 1/2

6/1/19

Phase 2 data ASCO June 1, 2019 noted 2/19 CRs + one PR.

5

INCY

Vitiligo

Approved

7/19/22

Approved July 19, 2022.

6

VRTX

Type 1 Diabetes

Phase 1/2

10/3/23

Phase 1/2 long term data reported that two patients with at least 1 year of follow-up met the criteria for the primary endpoint of elimination of severe hypoglycemic events (SHEs) and HbA1c <7.0%, noted October 3, 2023.

5

NBIX

Essential Tremor

Phase 2

8/4/22

Phase 2 trial did not meet primary endpoints, noted August 4, 2022.

6

CLVS

Endometrial Cancer

Phase 2

6/7/22

Phase 2 data reported that 23 subjects had clinical benefit, with 1 (4%) with CR, 9 (39%) with PR, and 13 (57%) with stable disease as best response, noted June 7, 2022.

5

GILD

Sjogren’s syndrome

Phase 2

10/24/19

Phase 2 trial did not meet primary endpoint.

4

SNY

Nasal polyps

Approved

6/26/19

FDA Approval announced June 26, 2019.

6

DCPH

Solid tumors

Phase 1

9/10/22

Phase 1 initial data presented at ESMO demonstrated a favorable tolerability profile and pharmacokinetics, and strong target inhibition across all dose levels tested, noted September 10, 2022

6

CYTK

Heart failure with reduced ejection fraction (HFrEF)

CRL

2/28/23

CRL issued February 28, 2023.

5

SMMT

C. difficile infection (CDI)

Phase 3

10/20/22

Phase 3 trial results reported that treatment was numerically higher sustained clinical response rate (73.0% vs 70.7%) than vancomycin, but did not achieve the pre-specified superiority endpoint, noted October 20, 2022.

6

NVS

Diabetic macular edema (DME)

Phase 3

8/17/21

Phase 3 met its primary endpoints, noted August 17, 2021.

6

LNTH

Chronic pain

Approved

9/29/14

Approved September 29, 2014.

6

COGT

Advanced Systemic Mastocytosis

Phase 2

6/10/22

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.

6

TAK

ALK+ Metastatic Non-Small Cell Lung Cancer

Approved

5/22/20

FDA Approval announced May 22, 2020.

6

GSK

Myelofibrosis

Approved

9/15/23

Approved September 15, 2023.

6