Expert Opinion on Investigational Drugs

Article	Citations
Biomarkers for breast cancer immunotherapy: PD-L1, TILs, and beyond	89
Durvalumab: an investigational anti-PD-L1 antibody for the treatment of biliary tract cancer	83
Faricimab: an investigational agent targeting the Tie-2/angiopoietin pathway and VEGF-A for the treatment of retinal diseases	78
PD-L1, TMB, and other potential predictors of response to immunotherapy for hepatocellular carcinoma: how can they assist drug clinical trials?	77
Atezolizumab-bevacizumab plus Y-90 TARE for the treatment of hepatocellular carcinoma: preclinical rationale and ongoing clinical trials	74
Immune-based combinations for metastatic triple negative breast cancer in clinical trials: current knowledge and therapeutic prospects	69
Futibatinib, an investigational agent for the treatment of intrahepatic cholangiocarcinoma: evidence to date and future perspectives	63
Ladiratuzumab vedotin for metastatic triple negative cancer: preliminary results, key challenges, and clinical potential	59
Carbonic anhydrase inhibitors: an update on experimental agents for the treatment and imaging of hypoxic tumors	56
The nonalcoholic steatohepatitis (NASH) drug development graveyard: established hurdles and planning for future success	56
Lenvatinib plus pembrolizumab: the next frontier for the treatment of hepatocellular carcinoma?	56
Latest generation estrogen receptor degraders for the treatment of hormone receptor-positive breast cancer	50
Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies	45
Fezolinetant in the treatment of vasomotor symptoms associated with menopause	43
The holistic approach to psoriasis patients with comorbidities: the role of investigational drugs	42
Tislelizumab: an investigational anti-PD-1 antibody for the treatment of advanced non-small cell lung cancer (NSCLC)	42
Linzagolix: a new GnRH-antagonist under investigation for the treatment of endometriosis and uterine myomas	41
ELX-02: an investigational read-through agent for the treatment of nonsense mutation-related genetic disease	41
Zilucoplan: An Investigational Complement C5 Inhibitor for the Treatment of Acetylcholine Receptor Autoantibody–Positive Generalized Myasthenia Gravis	41
CAR-T cell therapy for triple-negative breast cancer and other solid tumors: preclinical and clinical progress	39
Infigratinib (BGJ398): an investigational agent for the treatment of FGFR-altered intrahepatic cholangiocarcinoma	36
Progress and pitfalls in the use of immunotherapy for patients with triple negative breast cancer	34
Lecanemab (BAN2401): an anti–beta-amyloid monoclonal antibody for the treatment of Alzheimer disease	33
MK-6482 as a potential treatment for von Hippel-Lindau disease-associated clear cell renal cell carcinoma	32
Monoclonal antibody therapies in the management of SARS-CoV-2 infection	32

New promising drugs for the treatment of systemic sclerosis: pathogenic considerations, enhanced classifications, and personalized medicine	31
Differentiation between emerging non-steroidal and established steroidal mineralocorticoid receptor antagonists: head-to-head comparisons of pharmacological and clinical characteristics	30
Invasive candidiasis: investigational drugs in the clinical development pipeline and mechanisms of action	29
PARP inhibitors as single agents and in combination therapy: the most promising treatment strategies in clinical trials for BRCA-mutant ovarian and triple-negative breast cancers	29
Therapies in preclinical and clinical development for Angelman syndrome	28
Immunotherapies in clinical development for biliary tract cancer	27
Investigational drugs for the treatment of diffuse large B-cell lymphoma	27
Repurposing elesclomol, an investigational drug for the treatment of copper metabolism disorders	26
Neoantigen vaccine platforms in clinical development: understanding the future of personalized immunotherapy	26
Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA)	24
The emerging role of immune checkpoint inhibitors for the treatment of breast cancer	23
JAK inhibitors for rheumatoid arthritis	22
AE37: a HER2-targeted vaccine for the prevention of breast cancer recurrence	21
Immunotherapy in uveal melanoma: novel strategies and opportunities for personalized treatment	21
Translesion synthesis inhibitors as a new class of cancer chemotherapeutics	20
The time to offer treatments for COVID-19	20
Targeting FGFR in intrahepatic cholangiocarcinoma [iCCA]: leading the way for precision medicine in biliary tract cancer [BTC]?	20
Sprifermin: a recombinant human fibroblast growth factor 18 for the treatment of knee osteoarthritis	20
Investigational spleen tyrosine kinase (SYK) inhibitors for the treatment of autoimmune diseases	20
New treatment approaches for Alzheimer’s disease: preclinical studies and clinical trials centered on antidiabetic drugs	19
What have we learned from past failures of investigational drugs for Alzheimer’s disease?	18
Ipilimumab: an investigational immunotherapy for glioblastoma	18
Anti-diabetic drugs and NASH: from current options to promising perspectives	18
Investigational drugs in clinical trials for macular degeneration	17
Immune checkpoint inhibitors for the treatment of myeloma: novel investigational options	17
Targeting the tumor microenvironment in cholangiocarcinoma: implications for therapy	17
Aromatase inhibitors for the treatment of endometriosis: a systematic review about efficacy, safety and early clinical development	17
Therapeutic interventions for spinal muscular atrophy: preclinical and early clinical development opportunities	17
An update on investigational therapies that target STAT3 for the treatment of cancer	17
Investigational drugs and nutrients for human longevity. Recent clinical trials registered in ClinicalTrials.gov and clinicaltrialsregister.eu	16
Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia	16
Current emerging and investigational drugs for the treatment of chronic hand eczema	16
Asciminib: an investigational agent for the treatment of chronic myeloid leukemia	16
Lorecivivint, an intra-articular potential disease-modifying osteoarthritis drug	15
Investigational therapies targeting CD3 for prevention and treatment of type 1 diabetes	15
Immune checkpoint inhibitors for the treatment of sepsis:insights from preclinical and clinical development	15
Overview on the use of IL-6 agents in the treatment of patients with cytokine release syndrome (CRS) and pneumonitis related to COVID-19 disease	14
Development of gamma-tocotrienol as a radiation medical countermeasure for the acute radiation syndrome: current status and future perspectives	14
Systemic therapy of advanced/metastatic gastrointestinal stromal tumors: an update on progress beyond imatinib, sunitinib, and regorafenib	14
Telacebec: an investigational antibacterial for the treatment of tuberculosis (TB)	14
Emerging agents for the treatment and prevention of stroke: progress in clinical trials	14
Bladder cancer: shedding light on the most promising investigational drugs in clinical trials	14
Post-pancreatitis diabetes mellitus: investigational drugs in preclinical and clinical development and therapeutic implications	14
Tackling type 2 diabetes-associated cardiovascular and renal comorbidities: a key challenge for drug development	14
Poly (ADP-ribose) polymerase (PARP) as target for the treatment of epithelial ovarian cancer: what to know	13
Targeting Ebola virus replication through pharmaceutical intervention	13
Novel systemic treatment approaches for metastatic pancreatic cancer	13
Current options and investigational drugs for the treatment of eosinophilic esophagitis	13
Breakthrough therapy designation of nirsevimab for the prevention of lower respiratory tract illness caused by respiratory syncytial virus infections (RSV)	13
Recent progress in development of cyclin-dependent kinase 7 inhibitors for cancer therapy	13

IgA nephropathy: an overview of drug treatments in clinical trials	13
Emerging treatments for chronic urticaria	12
Low-dose aspirin for early COVID-19: does the early bird catch the worm?	12
A case study of foliglurax, the first clinical mGluR4 PAM for symptomatic treatment of Parkinson’s disease: translational gaps or a failing industry innovation model?	12
Rare cancer, rare alteration: the case of NTRK fusions in biliary tract cancers	12
Precision approaches for cholangiocarcinoma: progress in clinical trials and beyond	12
An evaluation of KBP-5074 in advanced chronic kidney disease with uncontrolled hypertension	12
Adrecizumab: an investigational agent for the biomarker-guided treatment of sepsis	11
Angiogenesis in biliary tract cancer: targeting and therapeutic potential	11
Derazantinib: an investigational drug for the treatment of cholangiocarcinoma	11
Camrelizumab: an investigational agent for hepatocellular carcinoma	11
Advances in the treatment of platinum resistant epithelial ovarian cancer: an update on standard and experimental therapies	11
Investigational neuroprotective compounds in clinical trials for retinal disease	11
Advances in targeting estrogen synthesis and receptors in patients with endometriosis	11
Investigational drugs for assisting psychotherapy for posttraumatic stress disorder (PTSD): emerging approaches and shifting paradigms in the era of psychedelic medicine	11
Selective endothelin A receptor antagonism in patients with proteinuric chronic kidney disease	11
Safety, pharmacokinetics and pharmacodynamics of TQ-A3334, an oral toll-like receptor 7 agonist in healthy individuals	11
ALZT-OP1: an experimental combination regimen for the treatment of Alzheimer’s disease	10
Investigational drugs for the treatment of kidney transplant rejection	10
Recent developments for new investigational JAK inhibitors in psoriatic arthritis	10
Safety and pharmacokinetics of a biosimilar of denosumab (KN012): Phase 1 and bioequivalence study in healthy Chinese subjects	10
Anti-integrin drugs in clinical trials for inflammatory bowel disease (IBD): insights into promising agents	10
Cholangiocarcinoma: bridging the translational gap from preclinical to clinical development and implications for future therapy	10
Therapeutic angiogenesis in coronary artery disease: a review of mechanisms and current approaches	10
Cystic fibrosis – Ten promising therapeutic approaches in the current era of care	10
Circadian rhythms in diabetic retinopathy: an overview of pathogenesis and investigational drugs	10
COR388 (atuzaginstat): an investigational gingipain inhibitor for the treatment of Alzheimer disease	10
Novel immunotherapy combinations in clinical trials for hepatocellular carcinoma: will they shape the future treatment landscape?	10
Treating cognitive impairment in schizophrenia with GLP-1RAs: an overview of their therapeutic potential	10
Ritlecitinib: an investigational drug for the treatment of moderate to severe alopecia areata	10
Remyelination therapies for multiple sclerosis: optimizing translation from animal models into clinical trials	9
Ulcerative colitis: shedding light on emerging agents and strategies in preclinical and early clinical development	9
Current status and future expectations of nanobodies in oncology trials	9
Understanding drug resistance mechanisms in cholangiocarcinoma: assisting the clinical development of investigational drugs	9
Clinical complexity of utilizing FGFR inhibitors in cancer therapeutics	9
Cabozantinib plus atezolizumab for the treatment of advanced hepatocellular carcinoma: shedding light on the preclinical rationale and clinical trials	9
Oregovomab: an investigational agent for the treatment of advanced ovarian cancer	9
Targeted therapies for gallbladder cancer: an overview of agents in preclinical and clinical development	9
Durvalumab: an investigational agent for unresectable hepatocellular carcinoma	9
Cardiac fibrosis: emerging agents in preclinical and clinical development	9
Metformin: evidence from preclinical and clinical studies for potential novel applications in cardiovascular disease	9
A randomized, double-blind, parallel pharmacokinetic study comparing the trastuzumab biosimilar candidate, AryoTrust®, and reference trastuzumab in healthy subjects	9
Drugs in phase I and II clinical development for the prevention of stroke in patients with atrial fibrillation	9
Investigational immunomodulatory drugs for enhancement of triple negative breast cancer (TNBC) immunotherapy: early phase development	9
Batten disease: an expert update on agents in preclinical and clinical trials	9
Still grasping at straws: donanemab in Alzheimer’s disease	9
Efficacy, safety, and tolerability of ulotaront (SEP-363856, a trace amine-associated receptor 1 agonist) for the treatment of schizophrenia and other mental disorders: a systematic review of preclini	9
A randomized, adaptive design, double-blind, 3-arm, parallel study assessing the pharmacokinetics and safety of AVT02, a high-concentration (100 mg/mL) Adalimumab biosimilar, in healthy adult subjects	9
Experimental HER2- targeted therapies for biliary tract cancer	8
Dementia with Lewy bodies: emerging drug targets and therapeutics	8
Combination therapies plus transarterial chemoembolization in hepatocellular carcinoma: a snapshot of clinical trial progress	8
Tilsotolimod: an investigational synthetic toll-like receptor 9 (TLR9) agonist for the treatment of refractory solid tumors and melanoma	8
Results from a biomarker study to accompany a phase II trial of RRx-001 with reintroduced platinum-based chemotherapy in relapsed small cell carcinoma	8
Antibacterial agents active against Gram Negative Bacilli in phase I, II, or III clinical trials	8
Repurposing tetracyclines for acute respiratory distress syndrome (ARDS) and severe COVID-19: a critical discussion of recent publications	8
Druggable molecular alterations in bile duct cancer: potential and current therapeutic applications in clinical trials	8
New investigational agents for the treatment of major depressive disorder	8
Chemoresistance and resistance to targeted therapies in biliary tract cancer: what have we learned?	8
Emerging neuroprotective interventions in periventricular leukomalacia - A systematic review of preclinical studies	8
Investigational immunotherapy targeting CD19 for the treatment of acute lymphoblastic leukemia	8
Emerging HER2-directed therapeutic agents for gastric cancer in early phase clinical trials	7
Single and multiple dose pharmacokinetics and safety of ZSP1273, an RNA polymerase PB2 protein inhibitor of the influenza A virus: a phase 1 double-blind study in healthy subjects	7
KSI-301: an investigational anti-VEGF biopolymer conjugate for retinal diseases	7
MK-0616: an oral PCSK9 inhibitor for hypercholesterolemia treatment	7
Investigational systemic drugs for moderate to severe plaque psoriasis: What’s new?	7
Disease modification in ankylosing spondylitis with TNF inhibitors: spotlight on early phase clinical trials	7
Experimental drugs in randomized controlled trials for long-COVID: what’s in the pipeline? A systematic and critical review	7
Nonalcoholic steatohepatitis (NASH) cirrhosis: a snapshot of therapeutic agents in clinical development and the optimal design for clinical trials	7
Investigational drugs in early phase development for primary biliary cholangitis	7
A perspective on RNA interference-based therapeutics for metabolic liver diseases	7
Is retatrutide (LY3437943), a GLP-1, GIP, and glucagon receptor agonist a step forward in the treatment of diabetes and obesity?	7
SGLT2 inhibitors: an evidence-based update on cardiovascular implications	7
The impact of molecular profiling on cholangiocarcinoma clinical trials and experimental drugs	7
Investigational PARP inhibitors for the treatment of biliary tract cancer: spotlight on preclinical and clinical studies	7
Emerging clinical investigational drugs for the treatment of amyotrophic lateral sclerosis	6
Investigational anti IL-13 asthma treatments: a 2023 update	6
Role of bromodomain and extraterminal (BET) proteins in prostate cancer	6
Radiotherapy-induced severe oral mucositis: pharmacotherapies in recent and current clinical trials	6
Investigational drugs for the treatment of olfactory dysfunction	6
A randomized, double-blind, 3-arm, parallel study assessing the pharmacokinetics, safety, tolerability and immunogenicity of AVT04, an ustekinumab candidate biosimilar, in healthy adults	6

Investigational drugs for the treatment of thymic cancer: a focus on phase 1 and 2 clinical trials	6
Galinpepimut-S (GPS): an investigational agent for the treatment of acute myeloid leukemia	6
Trabodenoson on trabecular meshwork rejuvenation: a comprehensive review of clinical data	6
Key challenges for drugs in clinical development for cholangiocarcinoma	6
NF1 alterations in cancers: therapeutic implications in precision medicine	6
BI 1015550: an investigational phosphodiesterase 4B (PDE4B) inhibitor for lung function decline in idiopathic pulmonary fibrosis (IPF)	6
Ewing sarcoma: investigational mono- and combination therapies in clinical trials	6
COVID-related fibrosis: insights into potential drug targets	6
A phase 1b/2 study of PF-06747775 as monotherapy or in combination with Palbociclib in patients with epidermal growth factor receptor mutant advanced non-small cell lung cancer	6
Tucatinib: an investigational novel therapeutic agent for the treatment of HER-2 colorectal cancer	6
Experimental drugs for Friedrich’s ataxia: progress and setbacks in clinical trials	5
Nivolumab: an investigational agent for the treatment of biliary tract cancer	5
Targeted therapies for extrahepatic cholangiocarcinoma: preclinical and clinical development and prospects for the clinic	5
Failed clinical trials on COVID-19 acute respiratory distress syndrome in hospitalized patients: common oversights and streamlining the development of clinically effective therapeutics	5
A comprehensive overview of investigational elastase inhibitors for the treatment of acute respiratory distress syndrome	5
Hepatic encephalopathy: investigational drugs in preclinical and early phase development	5
Emerging and investigational targeted chemotherapy and immunotherapy agents for metastatic brain tumors	5
HPV-induced cancers: preclinical therapeutic advancements	5
Investigational topical anticholinergics in clinical development for the treatment of hyperhidrosis	5
AXS-05: an investigational treatment for Alzheimer’s disease-associated agitation	5
Bioavailability and pharmacokinetic profile of balovaptan, a selective, brain-penetrant vasopressin 1a receptor antagonist, in healthy volunteers	5
Novel investigational treatments for ventilator-associated pneumonia and critically ill patients in the intensive care unit	5
Ivosidenib: an investigational drug for the treatment of biliary tract cancers	5
Regorafenib, an investigational agent for the treatment of cholangiocarcinoma	5
Cholangiocarcinoma: shedding light on the most promising drugs in clinical development	5
FGFR4 inhibitors for the treatment of hepatocellular carcinoma: a synopsis of therapeutic potential	5
Hepatitis D virus (HDV): investigational therapeutic agents in clinical trials	5
A randomized, double-blind, placebo-controlled, single, and multiple dose-escalation Phase I clinical trial to investigate the safety, pharmacokinetic, and pharmacodynamic profiles of oral S086, a nov	5
Avasopasem for the treatment of radiotherapy-induced severe oral mucositis	5
Postural orthostatic tachycardia syndrome: pathophysiology, management, and experimental therapies	5
Assessing potential of psilocybin for depressive disorders	5
What’s the latest with investigational drugs for soft tissue sarcoma?	5
Immunotherapeutic strategies for treating opioid use disorder and overdose	5
Collocation of metformin and dipeptidyl peptidase-4 inhibitor is associated with increased risk of diabetes-related vascular dementia: A single hospital study in Northern Taiwan	4
Potential experimental immune checkpoint inhibitors for the treatment of cancer of the liver	4
Prospect of CAR T-cell therapy in acute myeloid leukemia	4
Venetoclax in acute myeloid leukemia	4
Efruxifermin, an investigational treatment for fibrotic or cirrhotic nonalcoholic steatohepatitis (NASH)	4
Investigational antibody-drug conjugates in clinical trials for the treatment of breast cancer	4
Emerging experimental drugs in clinical trials for migraine: observations and key talking points	4
Investigational agents for the treatment of methicillin-resistant Staphylococcus aureus (MRSA) bacteremia: progress in clinical trials	4
Anti-inflammatory therapies for acute respiratory distress syndrome	4
CNM-Au8: an experimental agent for the treatment of amyotrophic lateral sclerosis (ALS)	4
A foreword on biliary tract cancers: emerging treatments, drug targets, and fundamental knowledge gaps	4
Experimental drugs for the prevention or treatment of sensorineural hearing loss	4
Therapeutic challenges at the preclinical level for targeted drug development for Opisthorchis viverrini-associated cholangiocarcinoma	4
Quantitative evaluation of drug efficacy in the treatment of myasthenia gravis	4
Insulin sensitizers in 2023: lessons learned and new avenues for investigation	4
Antibody therapies for the treatment of acute myeloid leukemia: exploring current and emerging therapeutic targets	4
An overview on the use of antivirals for the treatment of patients with COVID19 disease	4
Investigational drugs for immune thrombocytopenia	4
Genetic prion disease: opportunities for early therapeutic intervention with rigorous pre-symptomatic trials	4
Microbiome modulators for atopic eczema: a systematic review of experimental and investigational therapeutics	4
Investigational treatment options in phase I and phase II trials for relapsed or refractory acute lymphoblastic leukemia in pediatric patients	4
Ulotaront: a TAAR1/5-HT1A agonist in clinical development for the treatment of schizophrenia	4
Litifilimab (BIIB059), a promising investigational drug for cutaneous lupus erythematosus	4
Safety, tolerability, and pharmacokinetics of the novel pan-phosphodiesterase inhibitor ZSP1601 in healthy subjects: a double-blinded, placebo-controlled first-in-human single-dose and multiple-dose e	4
Islatravir: evaluation of clinical development for HIV and HBV	4
Developing models of cholangiocarcinoma to close the translational gap in cancer research	4
Novel biomarkers for cholangiocarcinoma: how can it enhance diagnosis, prognostication, and investigational drugs? Part-1	4
Recent developments in adjunct therapies for type 1 diabetes	4
Novel approaches for the management of biliary tract cancer: today and tomorrow	4
The time to develop treatments for diabetic neuropathy	4
Experimental drugs in clinical trials for COPD: artificial intelligence via machine learning approach to predict the successful advance from early-stage development to approval	4
Current and emerging investigational venetoclax-based therapies in chronic lymphocytic leukemia	3
Odevixibat: an investigational inhibitor of the ileal bile acid transporter (IBAT) for the treatment of biliary atresia	3
Retatrutide showing promise in obesity (and type 2 diabetes)	3
Icosabutate: targeting metabolic and inflammatory pathways for the treatment of NASH	3
KIT/PDGFRA inhibitors for the treatment of gastrointestinal stromal tumors: getting to the gist of the problem	3
RNA therapeutics for mood disorders: current evidence toward clinical trials	3
Pharmacological agents for bone fracture healing: talking points from recent clinical trials	3
Inflammatory myopathies: shedding light on promising agents and combination therapies in clinical trials	3
Atezolizumab: an investigational agent for the treatment of biliary tract cancer	3
Inhibition of KIT for chronic urticaria: a status update on drugs in early clinical development	3
Bomedemstat as an investigative treatment for myeloproliferative neoplasms	3
The treatment of advanced melanoma: a review of systemic and local therapies in combination with immune checkpoint inhibitors in phase 1 and 2 clinical trials	3
Clinical development of antibody-drug conjugates in triple negative breast cancer: can we jump higher?	3
Giant cell arteritis: what is new in the preclinical and early clinical development pipeline?	3
Lenvatinib: an investigational agent for the treatment of differentiated thyroid cancer	3
The potential role of opioid vaccines and monoclonal antibodies in the opioid overdose crisis	3
Potassium channel modulators and schizophrenia: an overview of investigational drugs	3
Pharmacokinetics and safety of candidate tocilizumab biosimilar CT-P47 versus reference tocilizumab: a randomized, double-blind, single-dose phase I study	3
Novel JAK inhibitors under investigation for systemic lupus erythematosus: - where are we now?	3
Combination therapies in clinical trials for renal cell carcinoma: how could they impact future treatments?	3
The triple negative breast cancer drugs graveyard: a review of failed clinical trials 2017-2022	3
From bench to bedside - is there a role of IL-17 drugs in rheumatoid arthritis?	3
Experimental drugs for supraventricular tachycardia: an analysis of early phase clinical trials	3
Experimental drug treatments for hepatocellular carcinoma: clinical trial failures 2015 to 2021	3
Heart failure with preserved ejection fraction: key stumbling blocks for experimental drugs in clinical trials	3
Investigational pharmacological agents for the treatment of ARDS	3
Second-line treatment options for hepatocellular carcinoma: current state and challenges for the future	3
CD200AR-L: mechanism of action and preclinical and clinical insights for treating high-grade brain tumors	3
Investigational drugs for recurrent or primary advanced metastatic cervical cancer: what is in the clinical development pipeline?	3
Ataxia-telangiectasia clinical trial landscape and the obstacles to overcome	3
Emerging pharmacotherapies for the treatment of pulmonary arterial hypertension	3