Formulation for a novel inhaled peptide therapeutic for idiopathic pulmonary fibrosis

References

• Raghu G, Collard HR, Egan JJ, et al. An official ATS/ERS/JRS/ALAT statement: idiopathic pulmonary fibrosis: evidence-based guidelines for diagnosis and management. Am J Respir Crit Care Med. 2011;183:788–824.
   PubMed Web of Science ®Google Scholar
• Gribbin J, Hubbard RB, Le Jeune I, et al. Incidence and mortality of idiopathic pulmonary fibrosis and sarcoidosis in the UK. Thorax. 2006;61:980–985.
   PubMed Web of Science ®Google Scholar
• Feghali-Bostwick CA, Yamaguchi Y. Use of endostatin peptides for the treatment of fibrosis. US8507441 B2, August 13, 2013.
   Google Scholar
• Cottin V, Valenzuela C. Pharmacological management. In: Costabel U, Crestani B, Wells AU, editors. Idiopathic pulmonary fibrosis. Sheffield, UK: European Respiratory Society; 2016. p. 196.
   Google Scholar
• Salisbury ML, Xia M, Zhou Y, et al. Idiopathic pulmonary fibrosis: gender-age-physiology index stage for predicting future lung function decline. Chest. 2016;149:491–498.
   PubMed Web of Science ®Google Scholar
• Panos RJ, Mortenson RL, Niccoli SA, et al. Clinical deterioration in patients with idiopathic pulmonary fibrosis: causes and assessment. Am J Med. 1990;88:396–404.
   PubMed Web of Science ®Google Scholar
• Richeldi L, du Bois RM, Raghu G, et al. Efficacy and safety of nintedanib in idiopathic pulmonary fibrosis. N Engl J Med. 2014;370:2071.
   PubMed Web of Science ®Google Scholar
• King TE, Jr, Bradford WZ, Castro-Bernardini S, et al. A phase 3 trial of pirfenidone in patients with idiopathic pulmonary fibrosis. N Engl J Med. 2014;370:2083.
   PubMed Web of Science ®Google Scholar
• Raghu G, Rochwerg B, Zhang Y, et al. An official ATS/ERS/JRS/ALAT clinical practice guideline: treatment of idiopathic pulmonary fibrosis. An update of the 2011 clinical practice guideline. Am J Respir Crit Care Med. 2015;192:e3–19.
   PubMed Web of Science ®Google Scholar
• Marudamuthu AS, Shetty SK, Bhandary YP, et al. Plasminogen activator inhibitor-1 suppresses profibrotic responses in fibroblasts from fibrotic lungs. J Biol Chem. 2015;290:9428–9441.
   PubMed Web of Science ®Google Scholar
• Shetty SK, Bhandary YP, Marudamuthu AS, et al. Regulation of airway and alveolar epithelial cell apoptosis by p53-Induced plasminogen activator inhibitor-1 during cigarette smoke exposure injury. Am J Respir Cell Mol Biol. 2012;47:474–483.
   PubMed Web of Science ®Google Scholar
• Bhandary YP, Shetty SK, Marudamuthu AS, et al. Regulation of alveolar epithelial cell apoptosis and pulmonary fibrosis by coordinate expression of components of the fibrinolytic system. Am J Physiol Lung Cell Mol Physiol. 2012;302:L463–L473.
   PubMed Web of Science ®Google Scholar
• Bhandary YP, Shetty SK, Marudamuthu AS, et al. Regulation of lung injury and fibrosis by p53-mediated changes in urokinase and plasminogen activator inhibitor-1. Am J Pathol. 2013;183:131–143.
   PubMed Web of Science ®Google Scholar
• Marudamuthu AS, Bhandary YP, Shetty SK, et al. Role of the urokinase-fibrinolytic system in epithelial-mesenchymal transition during lung injury. Am J Pathol. 2015;185:55–68.
   PubMed Web of Science ®Google Scholar
• Bhandary YP, Shetty SK, Marudamuthu AS, et al. Role of p53–fibrinolytic system cross-talk in the regulation of quartz-induced lung injury. Toxicol Appl Pharmacol. 2015;283:92–98.
   PubMed Web of Science ®Google Scholar
• Fakes MG, Dali MV, Haby TA, et al. Moisture sorption behavior of selected bulking agents used in lyophilized products. PDA J Pharm Sci Technol. 2000;54:144–149.
   PubMedGoogle Scholar
• Costantino HR, Langer R, Klibanov AM. Solid-phase aggregation of proteins under pharmaceutically relevant conditions. J Pharm Sci. 1994;83:1662–1669.
   PubMed Web of Science ®Google Scholar
• Robbins DC, Cooper SM, Fineberg SE, et al. Antibodies to covalent aggregates of insulin in blood of insulin-using diabetic patients. Diabetes. 1987;36:838–841.
   PubMed Web of Science ®Google Scholar
• Cleland JL, Powell MF, Shire SJ. The development of stable protein formulations: a close look at protein aggregation, deamidation, and oxidation. Crit Rev Ther Drug Carrier Syst. 1992;10:307–377.
   Web of Science ®Google Scholar
• Hsu CC, Ward CA, Pearlman R, et al. Determining the optimum residual moisture in lyophilized protein pharmaceuticals. Dev Biol Stand. 1992;74:255.
   PubMedGoogle Scholar
• Carpenter JF, Pikal MJ, Chang BS, et al. Rational design of stable lyophilized protein formulations: some practical advice. Pharm Res. 1997;14:969–975.
   PubMed Web of Science ®Google Scholar
• Shamblin SL, Hancock BC, Zografi G. Water vapor sorption by peptides, proteins and their formulations. Eur J Pharm Biopharm. 1998;45:239–247.
   PubMed Web of Science ®Google Scholar
• Costantino HR, Carrasquillo KG, Cordero RA, et al. Effect of excipients on the stability and structure of lyophilized recombinant human growth hormone. J Pharm Sci. 1998;87:1412–1420.
   PubMed Web of Science ®Google Scholar
• Izutsu K, Yoshioka S, Terao T. Decreased protein-stabilizing effects of cryoprotectants due to crystallization. Pharm Res. 1993;10:1232–1237.
   PubMed Web of Science ®Google Scholar
• Izutsu K, Yoshioka S, Terao T. Effect of mannitol crystallinity on the stabilization of enzymes during freeze-drying. Chem Pharm Bull. 1994;42:5–8.
   PubMed Web of Science ®Google Scholar
• Costantino HR, Langer R, Klibanov AM. Aggregation of a lyophilized pharmaceutical protein, recombinant human albumin: effect of moisture and stabilization by excipients. Nat Biotechnol. 1995;13:493–496.
   Web of Science ®Google Scholar
• Li B, O’Meara MH, Lubach JW, et al. Effects of sucrose and mannitol on asparagine deamidation rates of model peptides in solution and in the solid state. J Pharm Sci. 2005;94:1723–1735.
   PubMed Web of Science ®Google Scholar
• Lai MC, Topp EM. Solid-state chemical stability of proteins and peptides. J Pharm Sci. 1999;88:489–500.
   PubMed Web of Science ®Google Scholar
• Sharma VK, Kalonia DS. Effect of vacuum drying on protein-mannitol interactions: the physical state of mannitol and protein structure in the dried state. AAPS PharmSciTech 2004;5:58–69.
   Web of Science ®Google Scholar
• Izutsu K, Kojima S. Excipient crystallinity and its protein-structure-stabilizing effect during freeze-drying. J Pharm Pharmacol. 2002;54:1033–1039.
   PubMed Web of Science ®Google Scholar
• Oliyai C, Patel JP, Carr L, et al. Chemical pathways of peptide degradation. VII. Solid state chemical instability of an aspartyl residue in a model hexapeptide. Pharm Res. 1994;11:901–908.
   PubMed Web of Science ®Google Scholar
• Kreilgaard L, Frokjaer S, Flink JM, et al. Effects of additives on the stability of Humicola lanuginosa lipase during freeze-drying and storage in the dried solid. J Pharm Sci. 1999;88:281–290.
   PubMed Web of Science ®Google Scholar
• Pilcer G, Amighi K. Formulation strategy and use of excipients in pulmonary drug delivery. Int J Pharm. 2010;392:1–19.
   PubMed Web of Science ®Google Scholar
• Hertel SP, Winter G, Friess W. Protein stability in pulmonary drug delivery via nebulization. Adv Drug Deliv Rev. 2015;93:79–94.
   PubMed Web of Science ®Google Scholar
• Hertel S. Pulmonary delivery of pharmaceutical proteins by means of vibrating mesh nebulization. Ludwig-Maximilians: Universität München; 2014.
   Google Scholar
• Elhissi AMA, Taylor KMG. Delivery of liposomes generated from proliposomes using air-jet, ultrasonic, and vibrating-mesh nebulisers. J Drug Deliv Sci Technol. 2005;15:261–265.
   Web of Science ®Google Scholar
• Hertel S, Pohl T, Friess W, et al. Prediction of protein degradation during vibrating mesh nebulization via a high throughput screening method. Eur J Pharm Biopharm. 2014;87:386–394.
   PubMed Web of Science ®Google Scholar
• Waldrep JC, Dhand R. Advanced nebulizer designs employing vibrating mesh/aperture plate technologies for aerosol generation. Curr Drug Deliv. 2008;5:114–119.
   PubMedGoogle Scholar
• Watts AB, McConville JT, Williams RO. Current therapies and technological advances in aqueous aerosol drug delivery. Drug Dev Ind Pharm. 2008;34:913–922.
   PubMed Web of Science ®Google Scholar
• Knoch M, Keller M. The customised electronic nebuliser: a new category of liquid aerosol drug delivery systems. Expert Opin Drug Deliv. 2005;2:377–390.
   PubMedGoogle Scholar
• Wang W. Protein aggregation and its inhibition in biopharmaceutics. Int J Pharm. 2005;289:1–30.
   PubMed Web of Science ®Google Scholar
• Philo JS. Is any measurement method optimal for all aggregate sizes and types? AAPS J. 2006;8:E564–E571.
   PubMed Web of Science ®Google Scholar
• Baheti A, Kumar L, Bansal AK. Excipients used in lyophilization of small molecules. J Excip Food Chem. 2010;1:41–54.
   Google Scholar
• Wang DQ, Hey JM, Nail SI. Effect of collapse on the stability of freeze-dried recombinant factor VIII and alpha-amylase. J Pharm Sci. 2004;93:1253–1263.
   PubMed Web of Science ®Google Scholar
• Hageman MJ. In stability of protein pharmaceuticals, part A: chemical and physical pathways of protein degradation. In: Water sorption and solid-state stability of proteins: New York; 1992. p. 273–309.
   Google Scholar
• Galen M. Monitoring and control of industrial freeze-drying operations: the challenge of implementing Quality-by-Design (QbD). In: Rey L, May JC, editors. Freeze drying/lyophilization of pharmaceutical and biological products. 3rd ed. Drugs and the Pharmaceutical Sciences, vol. 206. New York: Informa Healthcare; 2010. p. 441–459.
   Google Scholar
• Schoeffel RE, Anderson SD, Altounyan REC. Bronchial hyperreactivity in response to inhalation of ultrasonically nebulised solutions of distilled water and saline. Br Med J (Clin Res Ed). 1981;283:1285–1287.
   PubMed Web of Science ®Google Scholar
• Portel L, Tunon de Lara JM, Vernejoux JM, et al. Osmolarity of solutions used in nebulization. Rev Mal Respir. 1998;15:191.
   PubMed Web of Science ®Google Scholar
• Chan H-K, Chan JGY, Kwok PCL, et al. Mannitol delivery by vibrating mesh nebulisation for enhancing mucociliary clearance. J Pharm Sci. 2011;100:2693–2702.
   PubMed Web of Science ®Google Scholar
• Pikal MJ. Mechanisms of protein stabilization during freeze-drying storage: the relative importance of thermodynamic stabilization and glassy state relaxation dynamics. In: Rey L, May JC, editors. Freeze drying/lyophilization of pharmaceutical and biological products, 3rd ed. Drugs and the Pharmaceutical Sciences, vol. 206. New York: Informa Healthcare; 2010. p. 198–232.
   Google Scholar
• Hancock BC, Shamblin SL. Water vapour sorption by pharmaceutical sugars. Pharm Sci Technol Today. 1998;1:345–351.
   Google Scholar
• Labrude P, Chaillot B, Bonneaux F, et al. Freeze-drying of haemoglobin in the presence of carbohydrates. J Pharm Pharmacol. 1980;32:588.
   PubMed Web of Science ®Google Scholar
• Hellman K, Miller DS, Cammack KA. The effect of freeze-drying on the quaternary structure of L-asparaginase from Erwinia carotovora. Biochim Biophys Acta. 1983;749:133.
   PubMed Web of Science ®Google Scholar
• Hong Z, Reis RL, Mano JF. Preparation and in vitro characterization of novel bioactive glass ceramic nanoparticles. J Biomed Mater Res A. 2009;88:304–313.
   PubMed Web of Science ®Google Scholar
• Yu L, Milton N, Groleau EG, et al. Existence of a mannitol hydrate during freeze-drying and practical implications. J Pharm Sci. 1999;88:196–198.
   PubMed Web of Science ®Google Scholar
• Liao X, Krishnamurthy R, Suryanarayanan R. Influence of processing conditions on the physical state of mannitol—implications in freeze-drying. Pharm Res. 2007;24:370–376.
   PubMed Web of Science ®Google Scholar
• Nunes C, Suryanarayanan R, Botez CE, et al. Characterization and crystal structure of D-mannitol hemihydrate. J Pharm Sci. 2004;93:2800–2809.
   PubMed Web of Science ®Google Scholar
• Devi S, Williams DR, Heng JYY. Crystallisation behaviour of D-mannitol as a function of temperature and relative humidity. [cited 28 May 2016]. Available from: http://www.aidic.it/isic18/webpapers/177Devi.pdf
   Google Scholar
• Telang C, Suryanarayanan R, Yu L. Crystallization of D-mannitol in binary mixtures with NaCl: phase diagram and polymorphism. Pharm Res. 2003;20:1939–1945.
   PubMed Web of Science ®Google Scholar
• Omar AE, Roos YH. Glass transition and crystallization behaviour of freeze-dried lactose–salt mixtures. LWT-Food Sci Technol. 2007;40:536–543.
   Web of Science ®Google Scholar
• Omar AE, Roos YH. Water sorption and time-dependent crystallization behaviour of freeze-dried lactose–salt mixtures. LWT-Food Sci Technol. 2007;40:520–528.
   Web of Science ®Google Scholar
• Alqurshi A, Kumar Z, McDonald R, et al. Amorphous formulation and in vitro performance testing of instantly disintegrating buccal tablets for the emergency delivery of naloxone. Mol Pharmaceutics. 2016;13:1688.
   PubMed Web of Science ®Google Scholar
• Cavatur R, Vemuri N, Pyne A, et al. Crystallization behavior of mannitol in frozen aqueous solutions. Pharm Res. 2002;19:894–900.
   PubMed Web of Science ®Google Scholar
• Araújo AA, Mercuri LP, Carvalho FM, et al. Thermal analysis of the antiretroviral zidovudine (AZT) and evaluation of the compatibility with excipients used in solid dosage forms. Int J Pharm. 2003;260:303–314.
   PubMed Web of Science ®Google Scholar
• Craig DQ, Barsnes M, Royall PG, et al. An evaluation of the use of modulated temperature DSC as a means of assessing the relaxation behaviour of amorphous lactose. Pharm Res. 2000;17:696–700.
   PubMed Web of Science ®Google Scholar
• Kurochkin IV, Procyk R, Bishop PD, et al. Domain structure, stability and domain-domain interactions in recombinant factor XIII . J Mol Biol. 1995;248:414–430.
   PubMed Web of Science ®Google Scholar
• Cholewinski M, Lückel B, Horn H. Degradation pathways, analytical characterization and formulation strategies of a peptide and a protein calcitonine and human growth hormone in comparison. Pharm Acta Helv. 1996;71:405–419.
   PubMedGoogle Scholar
• Picotti P, De Franceschi G, Frare E, et al. Amyloid fibril formation and disaggregation of fragment 1-29 of apomyoglobin: insights into the effect of pH on protein fibrillogenesis. J Mol Biol. 2007;367:1237–1245.
   PubMed Web of Science ®Google Scholar
• Costantino HR, Langer R, Klibanov AM. Moisture-induced aggregation of lyophilized insulin. Pharm Res. 1994;11:21–29.
   PubMed Web of Science ®Google Scholar
• Costantino HR, Curley JG, Hsu CC. Determining the water sorption monolayer of lyophilized pharmaceutical proteins. J Pharm Sci. 1997;86:1390–1393.
   PubMed Web of Science ®Google Scholar
• Herman BD, Sinclair BD, Milton N, et al. The effect of bulking agent on the solid-state stability of freeze-dried methylprednisolone sodium succinate. Pharm Res. 1994;11:1467–1473.
   PubMed Web of Science ®Google Scholar
• Costantino HR, Andya JD, Nguyen P-A, et al. Effect of mannitol crystallization on the stability and aerosol performance of a spray-dried pharmaceutical protein, recombinant humanized anti-IgE monoclonal antibody. J Pharm Sci. 1998;87:1406–1411.
   PubMed Web of Science ®Google Scholar
• Andya JD, Maa Y-F, Costantino HR, et al. The effect of formulation excipients on protein stability and aerosol performance of spray-dried powders of a recombinant humanized anti-IgE monoclonal antibody1. Pharm Res. 1999;16:350–358.
   PubMed Web of Science ®Google Scholar
• AnaSpec Inc. Peptide solubility guidelines. [cited 30 Jul 2016]. Available from: http://www.anaspec.com/content/pdfs/PeptidesolubilityguidelinesFinal.pdf
   Google Scholar
• Carpenter JF, Crowe JH. An infrared spectroscopic study of the interactions of carbohydrates with dried proteins. Biochemistry (Mosc). 1989;28:3916–3922.
   Google Scholar
• Arakawa T, Kita Y, Carpenter JF. Protein-solvent interactions in pharmaceutical formulations. Pharm Res. 1991;8:285.
   PubMed Web of Science ®Google Scholar
• Yaylayan VA, Wnorowski A, Perez Locas C. Why asparagine needs carbohydrates to generate acrylamide. J Agric Food Chem. 2003;51:1753–1757.
   PubMed Web of Science ®Google Scholar