investigational drugs


Summary: Drugs which have received FDA approval for human testing but have yet to be approved for commercial marketing. This includes drugs used for treatment while they still are undergoing clinical trials (Treatment IND). The main heading includes drugs under investigation in foreign countries.

Top Publications

  1. Casarosa P, Bouyssou T, Germeyer S, Schnapp A, Gantner F, Pieper M. Preclinical evaluation of long-acting muscarinic antagonists: comparison of tiotropium and investigational drugs. J Pharmacol Exp Ther. 2009;330:660-8 pubmed publisher
  2. Caprino L, Russo P. Developing a paradigm of drug innovation: an evaluation algorithm. Drug Discov Today. 2006;11:999-1006 pubmed
    ..IAA provides a score of drug innovation by assessing information generated during both the pre-marketing and the post-marketing authorization phase. ..
  3. Then R, Sahl H. Anti-infective strategies of the future: is there room for species-specific antibacterial agents?. Curr Pharm Des. 2010;16:555-66 pubmed
    ..Their application would include monotherapy as well as combination therapy with other antibiotics, anti-virulence agents or immunotherapy and these possibilities would greatly expand the current anti-infective armamentarium. ..
  4. Li X, Liu H, Feng H, Xian Z, Chen Y, Chen J, et al. Acupuncture paired with herbal medicine for prediabetes: study protocol for a randomized controlled trial. Trials. 2017;18:297 pubmed publisher
    ..control will be implemented, including quality control of the laboratory, researchers, participants, investigational drugs, data and documents, occurrence of bias, supervision, among others, according to uniform standard ..
  5. Ghanaat M. Types of hair loss and treatment options, including the novel low-level light therapy and its proposed mechanism. South Med J. 2010;103:917-21 pubmed publisher
    ..More in vitro studies are necessary to elucidate the mechanism and effectiveness at the cellular level, and more controlled studies are necessary to assess the role of this new treatment in the general population. ..
  6. Walker S, McAuslane N, Liberti L, Salek S. Measuring benefit and balancing risk: strategies for the benefit-risk assessment of new medicines in a risk-averse environment. Clin Pharmacol Ther. 2009;85:241-6 pubmed publisher
  7. Dalziel C. Digestive Disease Week 2008--turning science into medicine. IDrugs. 2008;11:559-62 pubmed
  8. Papadopoulou M, Bloomer W, Torti V, Page J. Investigational new drug-directed toxicology and pharmacokinetic study of 4-[3-(2-nitro-1-imidazolyl)-propylamino]-7-chloroquinoline hydrochloride (NLCQ-1, NSC 709257) in Beagle dogs. Basic Clin Pharmacol Toxicol. 2010;106:511-22 pubmed publisher
    ..Any drug-related toxicity noted was readily reversible and not cumulative. No sex difference was detected in the susceptibility to NLCQ-1-induced toxicity. ..
  9. Barker A, Sigman C, Kelloff G, Hylton N, Berry D, Esserman L. I-SPY 2: an adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin Pharmacol Ther. 2009;86:97-100 pubmed publisher

More Information


  1. Mason S, Barker R. Emerging drug therapies in Huntington's disease. Expert Opin Emerg Drugs. 2009;14:273-97 pubmed publisher
    ..This review discusses the treatments now used for HD before evaluating the newer drugs at present being explored in both the clinic and in the laboratory in mouse models of the disease. ..
  2. Baker D. The benefits and savings from publicly funded clinical trials of prescription drugs. Int J Health Serv. 2008;38:731-50 pubmed
    ..The system would be financed by paying lower drug prices under the Medicare drug program and other public health care programs. ..
  3. Simon L, Green A. Individual patient treatment use of unapproved drugs: a new option for the seriously ill. J Pain Palliat Care Pharmacother. 2010;24:19-22 pubmed publisher
    ..This commentary describes applicable law and issues that apply to the debate over individual patient access to unapproved drugs, including the issue of who should pay for the use of experimental drugs in patient care outside of a study. ..
  4. McCubrey J, Steelman L, Abrams S, Chappell W, Russo S, Ove R, et al. Emerging MEK inhibitors. Expert Opin Emerg Drugs. 2010;15:203-23 pubmed publisher
    ..MEK inhibitors have many potential therapeutic uses in the suppression of cancer, proliferative diseases and aging. ..
  5. Pohl D, Tutuian R, Fried M. Pharmacologic treatment of constipation: what is new?. Curr Opin Pharmacol. 2008;8:724-8 pubmed publisher
    ..This review discusses recent developments in the pharmacologic treatment of chronic constipation including recently FDA-approved lubiprostone, emerging 5-HT receptors modifiers, investigational substances, and probiotics. ..
  6. Searle B. American Society of Clinical Oncology--44th Annual Meeting. Reports of clinical trial results--part 1. IDrugs. 2008;11:545-6 pubmed
  7. Silveira M, Lindor K. Clinical features and management of primary sclerosing cholangitis. World J Gastroenterol. 2008;14:3338-49 pubmed
    ..A PSC-like variant attracting attention is cholangitis marked by raised levels of the immunoglobulin G4 subclass, prominence of plasma cells within the lesions, and steroid responsiveness. ..
  8. Skovronsky D. Use of eINDs for evaluation of multiple related PET amyloid plaque imaging agents. J Nucl Med. 2008;49:47N-48N pubmed
  9. Marin J, Goadsby P. Glutamatergic fine tuning with ADX-10059: a novel therapeutic approach for migraine?. Expert Opin Investig Drugs. 2010;19:555-61 pubmed publisher
    ..Targeting glutamatergic transmission in migraine may provide a novel preventive therapy that is effective and well-tolerated. ..
  10. Mita A, Takimoto C, Mita M, Tolcher A, Sankhala K, Sarantopoulos J, et al. Phase 1 study of AMG 386, a selective angiopoietin 1/2-neutralizing peptibody, in combination with chemotherapy in adults with advanced solid tumors. Clin Cancer Res. 2010;16:3044-56 pubmed publisher
    ..No pharmacokinetic interactions between AMG 386 and any of the tested chemotherapy regimens were noted. Promising antitumor activity was observed with all three treatment combinations. ..
  11. Raza S, Baig M, Russell H, Gourdet Y, Berger B. Clostridium difficile infection following chemotherapy. Recent Pat Antiinfect Drug Discov. 2010;5:1-9 pubmed
    ..This review focuses on recent insights on the epidemiology, diagnosis, current management, recent patents, and advances on treating strategies of CDI with reference to current studies. ..
  12. Stirner B. Stimulating research and development of pharmaceutical products for neglected diseases. Eur J Health Law. 2008;15:391-409 pubmed
    ..Indispensable requirement to tackle the issue of neglected diseases, however, is a concerted commitment of all stakeholders on national and international levels. ..
  13. Munshi K, Pawlowski K, Gonzalez Heydrich J, Picker J. Review of Salient Investigational Drugs for the Treatment of Fragile X Syndrome. J Child Adolesc Psychopharmacol. 2017;27:850-863 pubmed publisher
    ..The identification of the biochemical mechanism underlying this disorder has provided amenable targets for therapy. This review aims to provide an overview of investigational drug therapies for FXS...
  14. Peng X, Pentassuglia L, Sawyer D. Emerging anticancer therapeutic targets and the cardiovascular system: is there cause for concern?. Circ Res. 2010;106:1022-34 pubmed publisher
    ..Here, we discuss a few of the many research foci in oncology where we believe such collaboration will be particularly important. ..
  15. Bandarupalli U, Williams G. Recently published papers: Novel therapies in chronic obstructive pulmonary disease, cardiac chemicals and intensive care outcomes. Crit Care. 2009;13:198 pubmed publisher
    ..Cardiac-based research continues to generate new drugs and tests to better outcome and aid in early diagnosis. And how do various intensive care interventions compare in improving clinical and functional outcomes? ..
  16. Talele S, Xu K, Pariser A, Braun M, Farag El Massah S, Phillips M, et al. Therapies for inborn errors of metabolism: what has the orphan drug act delivered?. Pediatrics. 2010;126:101-6 pubmed publisher
    ..For 26 years, the Orphan Drug Act has generated new therapies for IEMs. Why some IEMs have motivated successful drug development and others have not remains enigmatic; yet the needs of IEM patients without treatment are a certainty. ..
  17. Ustun C, DeRemer D, Jillella A, Bhalla K. Investigational drugs targeting FLT3 for leukemia. Expert Opin Investig Drugs. 2009;18:1445-56 pubmed publisher
    ..Also, early clinical trial data specifically focusing on drug toxicity, clinical efficacy and future directions of FLT3-directed anticancer therapy are discussed. ..
  18. Kleinschnitz C, Meuth S, Wiendl H. The trials and errors in MS therapy. Int MS J. 2008;15:79-90 pubmed
    ..Examples include agents that target leukocyte differentiation molecules, co-stimulatory molecules, adhesion molecules and chemotaxis, as well as novel immunomodulators and anti-infective therapies. ..
  19. Sudhop T. [Compassionate use of non-approved medicinal products. Legal basis and guidelines]. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2009;52:439-43 pubmed publisher
    ..The paper outlines the legal basis and available guidelines and discusses further aspects which may be relevant in compassionate use programs. ..
  20. Williams R. Discontinued drugs in 2008: oncology drugs. Expert Opin Investig Drugs. 2009;18:1581-94 pubmed publisher
    ..Source information was derived from a search of the Pharmaprojects database for drugs reaching phase I-III clinical trials. ..
  21. Mease P. Spondyloarthritis update: new insights regarding classifcation, pathophysiology, and management. Bull NYU Hosp Jt Dis. 2008;66:203-9 pubmed
    ..International treatment recommendations have been or are being developed based on the evidence base from clinical trials. ..
  22. Chow S, Cheng B, Cosmatos D. On power and sample size calculation for QT studies with recording replicates at given time point. J Biopharm Stat. 2008;18:483-93 pubmed publisher
    ..The proposed approach can also be applied to minimize the cost for a given power. ..
  23. McGahey K, Weiss G. Reviewing concomitant medications for participants in oncology clinical trials. Am J Health Syst Pharm. 2017;74:580-586 pubmed publisher
    ..Use of investigational drugs is a mainstay of adult oncology clinical trials and has led to discovery of new oncology drug classes, ..
  24. Arkin M, Moasser M. HER-2-directed, small-molecule antagonists. Curr Opin Investig Drugs. 2008;9:1264-76 pubmed
    ..Signaling and structural studies also suggest a critical involvement of the kinase inactive HER-3 in the regulation of HER-2, creating unique challenges in the efforts to inactivate the latter. ..
  25. Lazzeri C, Tarquini R, Valente S, Abbate R, Gensini G. Emerging drugs for acute myocardial infarction. Expert Opin Emerg Drugs. 2010;15:87-105 pubmed publisher
  26. Murawski N, Pfreundschuh M. New drugs for aggressive B-cell and T-cell lymphomas. Lancet Oncol. 2010;11:1074-85 pubmed publisher
    ..The definition of the role of these new drugs alone or in combination with established chemotherapy regimens in adequately designed prospective trials represents one of the major challenges in clinical lymphoma research. ..
  27. Anand K, Sabbagh M. Early investigational drugs targeting tau protein for the treatment of Alzheimer's disease. Expert Opin Investig Drugs. 2015;24:1355-60 pubmed publisher
    ..Immunotherapy appears to be relatively safe and efficacious but narrow whereas protein kinase inhibition has not demonstrated clinical benefit to date. ..
  28. Malik M, Hnatkova K, Ford J, Madge D. Near-thorough QT study as part of a first-in-man study. J Clin Pharmacol. 2008;48:1146-57 pubmed publisher
    ..Thus, this ECG support of the first-in-man study provided data of regulatory acceptable accuracy at a small fraction of the cost of a full thorough QT study. ..
  29. Forner A, Rodríguez De Lope C, Reig M, Bruix J. [Treatment of advanced hepatocellular carcinoma]. Gastroenterol Hepatol. 2010;33:461-8 pubmed publisher
    ..The present article provides a review of the treatment of advanced-stage HCC, with special emphasis on the distinct agents that are currently under evaluation. ..
  30. Girgis R. Emerging drugs for pulmonary hypertension. Expert Opin Emerg Drugs. 2010;15:71-85 pubmed publisher
    ..Considerable progress has been made, and yet there continues to be a great unmet medical need for developing more efficacious therapies. ..
  31. Striano P, Striano S. New and investigational antiepileptic drugs. Expert Opin Investig Drugs. 2009;18:1875-84 pubmed publisher
    ..This paper reviews the available information on various classes of molecules that are in the pipeline as well as on the innovative approaches to the treatment of epilepsy. ..
  32. Perreault S, Burzynski J. Romiplostim: a novel thrombopoiesis-stimulating agent. Am J Health Syst Pharm. 2009;66:817-24 pubmed publisher
    ..The extrapolated drug cost for weekly dosing for one year is approximately $55,250. Romiplostim is a novel thrombopoietic-stimulating agent for use in patients with chronic ITP who have not responded to other therapies. ..
  33. Modi P. Future therapeutic directions; new medications and insulin delivery in a changing world for effective diabetes management. Curr Drug Discov Technol. 2009;6:200-13 pubmed
    ..This review discusses how these approaches can help patients manage their diabetes effectively by considering new insulin formulations and delivery devices and newer therapeutics. ..
  34. Catley M. Asthma & COPD--IQPC's Second Conference. IDrugs. 2010;13:601-4 pubmed
    ..b>Investigational drugs discussed include mepolizumab (GlaxoSmithKline plc), benralizumab (BioWa Inc/Kyowa Hakko Kirin Co Ltd/..
  35. Guo S. Emerging drugs for endometriosis. Expert Opin Emerg Drugs. 2008;13:547-71 pubmed
  36. Han E, Lin P, Wakabayashi M. Current status on biologic therapies in the treatment of epithelial ovarian cancer. Curr Treat Options Oncol. 2009;10:54-66 pubmed publisher
    ..Translational studies are needed to help design rational combinations of targeted agents and to help predict response to therapy. ..
  37. Bristow T, Constantine J, Harrison M, Cavoit F. Performance optimisation of a new-generation orthogonal-acceleration quadrupole-time-of-flight mass spectrometer. Rapid Commun Mass Spectrom. 2008;22:1213-22 pubmed publisher
    ..This provided increased confidence in the selection of elemental formulae resulting from accurate mass measurements. ..
  38. Napper A. Society for biomolecular sciences - 16th annual conference & exhibition - advancing the science of drug discovery. IDrugs. 2010;13:372-5 pubmed
    ..b>Investigational drugs discussed include GDC-0941 (Genentech Inc), NVP-AUY-922 (Novartis AG) and olaparib (AstraZeneca).
  39. Zorrilla E, Koob G. Progress in corticotropin-releasing factor-1 antagonist development. Drug Discov Today. 2010;15:371-83 pubmed publisher
    ..The therapeutic utility of CRF(1) antagonists should soon be clearer: several small molecules are currently in Phase II/III clinical trials for depression, anxiety and irritable bowel syndrome. ..
  40. Guerrero Zotano A, Arteaga C. Neoadjuvant Trials in ER+ Breast Cancer: A Tool for Acceleration of Drug Development and Discovery. Cancer Discov. 2017;7:561-574 pubmed publisher
    ..b>Significance: Neoadjuvant endocrine therapy is an excellent platform for the development of investigational drugs, triaging of novel combinations, biomarker validation, and discovery of mechanisms of drug resistance...
  41. Ben Menachem E. Lacosamide: an investigational drug for adjunctive treatment of partial-onset seizures. Drugs Today (Barc). 2008;44:35-40 pubmed publisher
    ..Results from clinical trials to date suggest that lacosamide may be a useful pharmacological treatment option for patients with partial-onset seizures. ..
  42. Kirk R, Hung J, Horner S, Perez J. Implications of pharmacogenomics for drug development. Exp Biol Med (Maywood). 2008;233:1484-97 pubmed publisher
    ..Current impediments to a more robust proliferation of the benefits of these technologies are discussed-pharmaceutical companies, clinical education, required statistical methods, and intellectual property landscape. ..
  43. Leamon C. Folate-targeted drug strategies for the treatment of cancer. Curr Opin Investig Drugs. 2008;9:1277-86 pubmed
    ..This review summarizes some important properties of the FR, including where it is expressed, and how it is being clinically manipulated with small-molecule conjugates of folate for diagnostic and therapeutic purposes related to cancer. ..
  44. Gridelli C, Rossi A, Bareschino M, Schettino C, Sacco P, Maione P. The potential role of insulin-like growth factor receptor inhibitors in the treatment of advanced non-small cell lung cancer. Expert Opin Investig Drugs. 2010;19:631-9 pubmed publisher
    ..Figitumumab reported interesting results in the treatment of advanced non-adenocarcinoma NSCLC patients. Overall, in order to administer the optimal treatment to patients, a more definite histological diagnosis is mandatory. ..
  45. Beutel J, Rudolf M, Grisanti S. Current and future therapies for age-related macular degeneration. Expert Opin Emerg Drugs. 2009;14:341-62 pubmed
  46. Bodovitz S. International Drug Discovery Science and Technology--BIT's Seventh Annual Congress. IDrugs. 2010;13:23-5 pubmed
    ..b>Investigational drugs discussed include the anticancer agents astuprotimut-r (GlaxoSmithKline plc) and AS-1411 (Antisoma plc).
  47. Homs M, Voest E, Siersema P. Emerging drugs for esophageal cancer. Expert Opin Emerg Drugs. 2009;14:329-39 pubmed publisher
    ..Phase III trials have not been published yet and, therefore, for targeted therapies also, possibly using new concepts, more studies are needed. ..
  48. Unciti Broceta A. MedChem Europe 2010 - select biosciences' sixth annual meeting. IDrugs. 2010;13:366-8 pubmed
    ..This conference report highlights selected presentations on fragment-based drug design, novel lipids for gene delivery carriers, protein-ligand binding and compound screening. ..
  49. Link B, Friedberg J. Monoclonal antibodies in lymphoma: the first decade. Semin Hematol. 2008;45:71-4 pubmed publisher
    ..Finally, possible future developments in the field will be proposed, including the use of monoclonal antibodies in ablative transplantation and in the treatment of leukemias. ..
  50. Marblestone J. Ubiquitin Drug Discovery & Diagnostics 2009 - First Annual Conference. IDrugs. 2009;12:750-3 pubmed
    ..b>Investigational drugs discussed include MLN-4924 and MLN-9708 (both Millennium Pharmaceuticals Inc), P-005091 (Progenra Inc), ..
  51. Zhou X, Garner R, Nicholson S, Kissling C, Mayers D. Microdose pharmacokinetics of IDX899 and IDX989, candidate HIV-1 non-nucleoside reverse transcriptase inhibitors, following oral and intravenous administration in healthy male subjects. J Clin Pharmacol. 2009;49:1408-16 pubmed publisher
    ..IDX899 has been selected based on these initial pharmacokinetic assessments and other criteria as the candidate for further clinical development. ..
  52. Audie J, Boyd C. The synergistic use of computation, chemistry and biology to discover novel peptide-based drugs: the time is right. Curr Pharm Des. 2010;16:567-82 pubmed
  53. Granberg D. Investigational drugs for neuroendocrine tumours. Expert Opin Investig Drugs. 2009;18:601-8 pubmed
    ..Many of these compounds have shown promising results in patients with NETs. ..