Specialized BioTherapeutics
Reference literature for our Specialized BioTherapeutics pipeline programs.
HyBryte™
- Poligone B, Alexander-Savino CV, Kim EJ, Mangold AR, Desimone J, Wong HK, Rumage AT, Donini O, Haulenbeek A, Schaber CJ, Straube R, Pullion C and Rook AH (2023) HyBryte™ use in early-stage cutaneous T-cell lymphoma. Front. Drug Discov. 3:1298453. doi: 10.3389/fddsv.2023.1298453
- “Phase 3 Efficacy with a Novel, First in Class, Well-Tolerated Photodynamic Therapy (HyBryte™) for Early Stage Cutaneous T-Cell Lymphoma,” to be presented by Christopher Pullion, DO at the National Organization for Rare Disorders (NORD) Breakthrough Summit, October 18-19, 2021 (NORD 2021)
- Kim EJ, Mangold AR, DeSimone JA, et al. Efficacy and Safety of Topical Hypericin Photodynamic Therapy for Early-Stage Cutaneous T-Cell Lymphoma (Mycosis Fungoides): The FLASH Phase 3 Randomized Clinical Trial. JAMA Dermatol. 2022;158(9):1031–1039. doi:10.1001/jamadermatol.2022.2749
- “Topical Hypericin Ointment Photodynamic Therapy is Effective and Safe in CTCL (FLASH Study)” presented by Ellen Kim, MD at the Society for Investigative Dermatology (SID)Virtual Meeting May 4, 2021 (SID 2021)
- “Visible Light Activated Topical Hypericin Ointment in CTCL: Phase 3 FLASH Study Results” presented by Ellen Kim, MD at the American Academy of Dermatology Virtual Meeting Experience April 24, 2021 (AAD 2021)
- “Topical Hypericin Ointment (SGX301) Photodynamic Therapy is Effective and Safe in CTCL: Results from the Multicenter Phase 3 FLASH Study” presented by Ellen
- “Response in a Patient with Refractory Folliculotropic Mycosis Fungoides to a Topical Hypericin Cream Activated with Fluorescent Light” presented by Brian Poligone, MD, PhD at the 4th World Congress of Cutaneous Lymphomas (WCCL) Congress, February 12-14, 2020 (WCCL 2020)
- Rook, A.H., et al., A phase II placebo-controlled study of photodynamic therapy with topical hypericin and visible light irradiation in the treatment of cutaneous T-cell lymphoma and psoriasis. J Am Acad Dermatol, 2010. 63(6): p. 984-90.
- Fox, F.E., et al., Photoactivated hypericin is an anti-proliferative agent that induces a high rate of apoptotic death of normal, transformed, and malignant T lymphocytes: implications for the treatment of cutaneous lymphoproliferative and inflammatory disorders. J Invest Dermatol, 1998. 111(2): p. 327-32.
- Journal of the American Medical Association Dermatology – Efficacy and Safety of Topical Hypericin Photodynamic Therapy for Early-Stage Cutaneous T-Cell Lymphoma (Mycosis Fungoides)
- Compatibility study of topical 0.25% hypericin (HyBryteTM) application in subjects with mycosis fungoides: Results of the HPN-CTCL-02 study
- White paper entitled, “SGX301 as a Rapid, Safe and Effective Treatment for Early Stage CTCL,” by Brian Poligone, MD, PhD; Henry Wong, MD, PhD; Aaron Mangold, MD; Jennifer DeSimone, MD; Lucia Seminario-Vidal, MD, PhD; and Ellen Kim, MD, November 13, 2020 (White Paper 2020)
- Degar, S., et al., Inactivation of the human immunodeficiency virus by hypericin: evidence for photochemical alterations of p24 and a block in uncoating. AIDS Res Hum Retroviruses, 1992. 8 (11): p. 1929-36.
- Criscione, V.D. and M.A. Weinstock, Incidence of cutaneous T-cell lymphoma in the United States, 1973-2002. Arch Dermatol, 2007. 143(7): p. 854-9.
- Blank, M., et al., Enhanced ubiquitinylation of heat shock protein 90 as a potential mechanism for mitotic cell death in cancer cells induced with hypericin. Cancer Res, 2003. 63(23): p. 8241-7.
- Lenard, J., A. Rabson, and R. Vanderoef, Photodynamic inactivation of infectivity of human immunodeficiency virus and other enveloped viruses using hypericin and rose bengal: inhibition of fusion and syncytia formation. Proc Natl Acad Sci U S A, 1993. 90(1): p. 158-62.
- Meruelo, D., G. Lavie, and D. Lavie, Therapeutic agents with dramatic antiretroviral activity and little toxicity at effective doses: aromatic polycyclic diones hypericin and pseudohypericin. Proc Natl Acad Sci U S A, 1988. 85(14): p. 5230-4.
- Duran, N. and P.S. Song, Hypericin and its photodynamic action. Photochem Photobiol, 1986. 43 (6): p. 677-80.
Paper
SGX942
- Kudrimoti M, Curtis A, Azawi S, Worden F, Katz S, Adkins D, Bonomi M, Scott Z, Elder J, Sonis ST, Straube R, Donini O. Dusquetide: Reduction in oral mucositis associated with enduring ancillary benefits in tumor resolution and decreased mortality in head and neck cancer patients. Biotech Reports. 2017; 15:24-26.
- Kudrimoti M, Curtis A, Azawi S, Worden F, Katz S, Adkins D, Bonomi M, Elder J, Sonis S, Straube S, Donini O. Dusquetide: A novel innate defense regulator demonstrating a significant and consistent reduction in the duration of oral mucositis in preclinical data and a randomized, placebo-controlled phase 2a clinical study. J Biotech 2016; 239: 115-125.
- North JR, Takenaka S, Rozek A, Kielczewska A, Opal S, Morici L, Finlay BB, Schaber CJ, Straube R, Donini O. A novel approach for emerging and antibiotic resistant infections: Innate defense regulators as an agnostic therapy. J Biotech 2016; 226: 24-34.
- Yu HB, Kielczewska A, Rozek A, Takenaka S, Li Y, Thorson L, et al. Sequestosome-1/p62 is the key intracellular target of innate defense regulator peptide. J Biol Chem. 2009;284,36007-11.
- Moscat J, Diaz-Meco MT. p62 at the crossroads of autophagy, apoptosis, and cancer. Cell 2009; 137(6),1001-4.
- Sonis ST, Schwartzbert LS, Walker MS, Weidner S, Alterovitz G. 2013. Predicting risk of chemotherapy-induced side effects in patients with colon cancer with single-nucleotide polymorphism (SNP) Bayesian networks. J Clin Oncol 2013;31 (4_suppl): Abstract 344.
- Schwartzberg LS, Sonis ST, Walker MS, Weidner SM, Alterovitz G. Single Nucleotide Polymorphism (SNP) Bayesian networks (BNs) Predict Risk of Chemotherapy-Induced Side Effects in Patients with Breast Cancer Receiving Dose Dense (DD) Doxorubicin/Cyclophosphamide Plus Paclitaxel (AC+T). Cancer Res. 2012;72 (24 Suppl): Abstract nr P1-15-12.
- Sonis S, Antin J, Tedaldi M, Alterovitz G, SNP-based Bayesian networks can predict oral mucositis risk in autologous stem cell transplant recipients. Oral Dis. 19(7):721-7.
- Alterovitz G, Tuthill C, Rios I, Modelska K, Sonis S. 2011. Personalized medicine for mucositis: Bayesian networks identify unique gene clusters, which predict the response to gamma-D-glutamyl-L-tryptophan (SCV-07) for the attenuation of chemoradiation-induced OM. Oral Oncol. 2011;47:951-5.
- Peterson DE, Bensadoun RJ, Roila F. Management of oral and gastrointestinal mucositis: ESMO clinical practice guidelines. Annals of Oncology 22. 2011; Supplement 6: vi78–vi84.
- Sonis ST. New thoughts on the initiation of mucositis. Oral Dis. 2010;16,597-600.
- Into T, Inomata M, Niida S, Murakami Y, Shibata K. Regulation of MyD88 Aggregation and the MyD88-dependent Signaling Pathway by Sequestosome-1 and Histone Deacetylase 6. J Biol Chem. 2010;285(46),35759-35769
- Rosenfeldt, M.T. and K. M. Ryan. The role of autophagy in tumour development and cancer therapy. Exp Rev Mol Med 2009,11: e36.
- Zheng YT, Shahnazari S, Brech A, Lamark T, Johansen T, Brumell JH. The adaptor protein p62/SQSTM1 targets invading bacteria to the autophagy pathway. J Immunol. 2009; 183,5909-16.
- Yoshikawa Y, Ogawa M, Hain T, Yoshida M, Fukumatsu M, Kim M, et al. Listeria monocytogenes ActA-mediated escape from autophagic recognition. Nat Cell Biol. 2009;11,1233-40
- Dupont N, Lacas-Gervais S, Bertout J, Paz I, Freche B, Van Nhieu GT, et al. Shigella phagocytic vacuolar membrane remnants participate in the cellular response to pathogen invasion and are regulated by autophagy. Cell Host Microbe. 2009;6,137-49
- Kim JY, Ozato K. The sequestosome 1/p62 attenuates cytokine gene expression in activated macrophages by inhibiting IFN regulatory factor 8 and TNF receptor-associated factor 6/NF-kappaB activity. J Immunol. 2009;182,2131-40
- Elting L et al. 2008 Patient-reported measurements of oral mucositis in head and neck cancer patients treated with radiotherapy with or without chemotherapy: demonstration of increased frequency, severity, resistance to palliation, and impact on quality of life. Cancer, 2008;113(10), 2704-13.
- Nonzee NJ, Dandade NA, Markossian T, Argiris A, Patel JD, Kern RC, Munshi HG, Calhoun EA, Bennett CL. Evaluating the supportive care costs of severe radiochemotherapy-induced mucositis and pharyngitis. Cancer. 2008;113:1446–52.
- Foster SL, Hargreaves DC, Medzhitov R. Gene-specific control of inflammation by TLR-induced chromatin modifications. Nature. 2007;447,972-8
- Scott MG, Dullaghan E, Mookherjee N, Glavas N, Waldbrook M, Thompson A, Wang A, Lee K, Doria S, Hamill P, Yu JJ, Li Y, Donini O, Guarna MM, Finlay BF, North JR, Hancock REW. An anti-infective peptide that selectively modulates the innate immune response. Nat Biotechnol 2007;25: 465-72.
- Sonis S, Haddad R, Posner M, Watkins B, Fey E, Morgan TV, Mookanamparambil L, Ramoni M. Gene expression changes in peripheral blood cells provide insight into the biological mechanisms associated with regimen-related toxicities in patients being treated for head and neck cancers. Oral Oncol. 2007;43:289-300.
- Moscat J, Diaz-Meco MT, Wooten MW. Signal integration and diversification through the p62 scaffold protein. Trends Biochem Sci. 2007;32,95-100.
- Seibenhener ML, Geetha T, Wooten MW. Sequestosome 1/p62--more than just a scaffold. FEBS Lett. 2007;581,175-9
- Doyle SL, O'Neill LA. Toll-like receptors: from the discovery of NFkappaB to new insights into transcriptional regulations in innate immunity. Biochem Pharmacol. 2006;72,1102-13.
- Tosi MF. Innate immune responses to infection. J Allergy Clin Immunol. 2005;116,241-9; quiz 50
- Finlay BB, Hancock RE. Can innate immunity be enhanced to treat microbial infections? Nat Rev Microbiol 2004;2: 497-504.
- Athman R, Philpott D. Innate immunity via Toll-like receptors and Nod proteins. Curr Opin Microbiol. 2004;7,25-32.
- Beutler B. Innate immunity: an overview. Mol Immunol. 2004;40,845-59.
- Sonis ST. A Biological Approach to Mucositis. J Supp Onc. 2004; 2(1), 21-36.
- Beutler B, Hoebe K, Du X, Ulevitch RJ. How we detect microbes and respond to them: the Toll-like receptors and their transducers. J Leukoc Biol. 2003;74,479-85.
- Janeway CA, Jr., Medzhitov R. Innate immune recognition. Annu Rev Immunol. 2002;20,197-216.
- Matzinger, P. The Danger Model : A renewed sense of self. Science. 2002; 296(5566), 301-305
- Aita V et al. Cloning and genomic organization of Beclin-1, a candidate tumor suppressor gene on chromosome 17q21. Genomics. 1999. 59:59-65.
Paper
- Donini O, Straube R. Host-Directed Innate Defense Regulators (IDRs) – Agnostic Therapy to Treat Bacterial Infections and Fight Resistance. Poster presentated at the 2018 ESCMID/ASM Conference on Drug Development to Meet the Challenge of Antimicrobial Resistance. 2018.
- Donini O, Behrens E, Haulenbeek A, Pullion C, Schaber C. An Innate Defense Regulator for the Treatment of Macrophage Activation Syndrome: Preclinical Studies in an Orphan Indication. Poster Presentated at the National Organization for Rare Diseases (NORD) Rare Diseases and Orphan Products Breakthrough Summit. 2016.
- Donini O, Kudrimoti M, Curtis A, Azawi S, Worden F, Katz S, Elder J. SGX942 Reduces the Duration of Severe Oral Mucositis. Poster presented at the 25th Multinational Association of Supportive Care in Cancer. 2016.
- Kielczewska A, Yu HB, Rozek A, Takenaka S, Li Y, Guarna MM, North JR, Foster LJ, Finlay BB, Donini O. Sequestosome-1 / p62 is the key intracellular target of innate defense regulator peptide. Poster presentation at Keystone Symposium on The Macrophage: The intersection of Pathogenic & Protective Inflammation. 2010.
- Donini O, Watkins BA, Palardy J, Opal S, Sonis S, Abrams MJ, North JR. Reduced Infection and Mucositis in Chemotherapy-Treated Animals Following Innate Defense Modulation Using a Novel Drug Candidate. 2010. Blood (ASH Annual Meeting Abstracts) 2010; 116:3781
- Takenaka S, Rozek A, Kielczewska A, Thorson L, North JR, Finlay BB, Donini O. Innate Defense Regulators are a novel macrophage-mediated therapy to treat infectious and inflammatory diseases. Poster presentation at Keystone Symposium on The Macrophage: The intersection of Pathogenic & Protective Inflammation. 2010.
- Yu H, Kielczewska A, Rozek A, Takenaka S, Li Y, Guarna M, Thorson L, Hancock R, North J, Foster L, Donini O, Finlay B. SQSTM-1/p62: A Novel Therapeutic Target in Infectious and Inflammatory Disease. Poster Presentation at 49th Interscience Conference on Antimicrobial Agents and Chemotherapy. 2009.
Poster
SGX302
- Rook AH, Wood GS, Duvic M, Vonderheid EC, Tobia A, Cabana B. A phase II placebo-controlled study of photodynamic therapy with topical hypericin and visible light irradiation in the treatment of cutaneous T-cell lymphoma and psoriasis. J Am Acad Dermatol. 2010;63(6):984-90.
- Stern RS, Nichols KT, Vakeva LH. Malignant melanoma in patients treated for psoriasis with methoxsalen (psoralen) and ultraviolet A radiation (PUVA). The PUVA Follow-Up Study. N Engl J Med. 1997;336(15):1041-5.
- Feldman S. Treatment of psoriasis in adults. UpToDate. 2021.
- Parisi R, Iskandar IYK, Kontopantelis E, Augustin M, Griffiths CEM, Ashcroft DM, et al. National, regional, and worldwide epidemiology of psoriasis: systematic analysis and modelling study. BMJ. 2020;369:m1590.
- Le Roux E, Frow H. Diagnosis and management of mild to moderate psoriasis. prescribercouk. 2020:9-17.
- Stern RS, Nijsten T, Feldman SR, Margolis DJ, Rolstad T. Psoriasis is common, carries a substantial burden even when not extensive, and is associated with widespread treatment dissatisfaction. J Investig Dermatol Symp Proc. 2004;9(2):136-9.
- Griffiths CE, Barker JN. Pathogenesis and clinical features of psoriasis. Lancet. 2007;370(9583):263-71.