Herantis Pharma Plc · OMXHEX:HRTIS
Herantis Pharma — LE&RN Online Symposium 2018
27 slides from Herantis Pharma's conference presentation, Jun 13, 2018
Slide 1: Clinical Progress With Lymfactin: Could Gene Therapy Cure Lymphedema?
LE&RN Online Symposium: Clinical Progress With Lymfactin©: Could Gene Therapy Cure Lymphedema? Presented by Pekka Simula, CEO, Herantis Pharma Plc
Slide 2: The 2018 LE&RN Symposium Series
The 2018 LE&RN Symposium Series has been made possible by the generous support of OBST¢M a= BEI impedined LDex PFGE BIOPHARMACEUTICALS HERANTIS lympha Press PHARMA Lymphatic Education & Research Network
Slide 3: Important Disclaimer
Important Disclaimer: LE&RN sponsored information is provided for use for you in consultation with your health care professional and is not meant to take the place of health care or services you may need. Please see your primary health care provider about any personal health concerns. Lymphatic Educ
Slide 4: LE&RN Online Symposium 13 June 2018
LE&RN Online Symposium 13 June 2018 www.herantis.com www.herantis.com © 2014-2018 HERANTIS ©PHARMA Plc. All 2014-2018 HERANTIS rights PHARMA Plc.reserved. All rights reserved.
Slide 5: Disclaimer
Disclaimer • This presentation does not intend to provide a thorough and detailed view of Herantis Pharma Plc (‘Company’). The information provided in this presentation shall not be considered sufficient for making any investment decisions related to the Company. Anyone considering an investment in
Slide 6: Disclosures
Disclosures • Human clinical studies presented here have been conducted under the approvals of Finnish Medicines Agency Fimea, Ethics Committee, the Board of Gene Technology of Finland, and other appropriate bodies, in compliance with applicable rules and regulations • Preclinical studies presented
Slide 7: Herantis Pharma Plc (HRTIS:FH)
Herantis Pharma Plc (HRTIS:FH) • Herantis is a public drug development company advancing two highly-differentiated clinical assets for unique market opportunities – Lymfactin® gene therapy for secondary lymphedema – CDNF therapy for Parkinson’s Disease, with potential in treating other neurodegenera
Slide 8: Lymphedema: Significant and growing unmet need
Lymphedema: Significant and growing unmet need “The psychosocial impact of lymphedema has been described to be as distressing as the initial diagnosis of Breast Cancer.” – Shih et al., J Clin Oncol 2009 6/19/18 8 www.herantis.com © 2018 HERANTIS PHARMA Plc. All rights reserved.
Slide 9: Secondary lymphedema
Secondary lymphedema Disease, Market and Awareness • Lymphedema (LE) is a chronic, progressive swelling of tissues caused by the dysfunction of the lymphatic vasculature – Lymphatic system is unable to return interstitial fluid to bloodstream – Estimated 140 million people worldwide have LE; there i
Slide 10: Lymfactin gene therapy: Scientific background
Lymfactin® gene therapy: Scientific background 6/19/18 10 www.herantis.com © 2014-2018 HERANTIS PHARMA Plc. All rights reserved.
Slide 11: What creates our lymphatic vessels?
What creates our lymphatic vessels? • The formation of lymphatic vessels is called lymphangiogenesis • Lymphangiogenesis is promoted by a protein called VEGF-C: A natural growth factor, which we all have • Thus the damages of the lymphatic system could be repaired by increasing the VEGF-C levels. Un
Slide 12: Lymfactin gene therapy
Lymfactin® gene therapy aims to cure the underlying cause of secondary lymphedema • Lymfactin® is a gene transfer vector carrying the human VEGF-C gene • Lymfactin® instructs the patient’s own cells to produce VEGF-C in the damaged area where Lymfactin® is administered • Single-dose treatment with l
Slide 13: Clear dose response in lymph vessel sprouting following Lymfactin administration in mice ear skin
Clear dose response in lymph vessel sprouting following Lymfactin® administration in mice ear skin Model: • Mice were injected with 2 x 106 to 2 x 109 viral particles of Lymfactin® into the ear skin. • The same concentration of a control vector coding for LacZ gene was injected into the contralatera
Slide 14: Perinodally administered Lymfactin improves lymphatic vessel regeneration in a porcine model
Perinodally administered Lymfactin® efficiently improves lymphatic vessel regeneration and lymph node function in a porcine model Native lymphangiogram taken 2 months after the lymph node transfer: Total no of lymphatic vessels 12.5±4.8 5.9±1.5 13.7±4.0 5.8±5.5 Lymphatic vessels connected 9.1±6.7 1.
Slide 15: Strong science: from VEGF-C discovery to adenoviral VEGF-C gene therapy
Strong science: from VEGF-C discovery to adenoviral VEGF-C gene therapy Vol 15, Issue 2, 15 Jan 1996; Vol 276, Issue 5317, 30 May 1997; Vol 5, Issue 1, Jan 2004; Vol 18, Issue 14, Nov 2004; Joukov et al Jeltsch et al Karkkainen et al Saaristo et al A novel vascular Hyperplasia of lymphatic Vascular
Slide 16: Academy professor Kari Alitalo
Academy professor Kari Alitalo is the inventor of Lymfactin® • Director of Translational Cancer Biology Research program, University of Helsinki – National Center of Excellence • Internationally leading expert in endothelial growth factors in cancer – More than 500 peer-reviewed scientific publicati
Slide 17: Development of Lymfactin into a therapeutic available for LE patients
Development of Lymfactin® into a therapeutic available for LE patients 6/19/18 17 www.herantis.com © 2014-2018 HERANTIS PHARMA Plc. All rights reserved.
Slide 18: So what good will that science do in practice?
So what good will that science do in practice? Lymfactin® is currently in formal clinical development. If it works as well in humans as it does in the disease models: Functional lymphatic Lymph flow Swelling LE cured? vessels normalized reduced formed 6/19/18 18 www.herantis.com © 2014-2018 HERANTIS
Slide 19: Drug development is slow and regulated
Drug development is slow and regulated The development of a new Market drug to market usually 0 % takes 10-15 years; 5 Clinical Phase 3 most drug candidates % never reach clinical 30 Lymfactin® has reached stage Clinical Phase 2 Clinical Phase 2 0 % 6 Clinical Phase 1 ? 0 % 1 Preclinical development
Slide 20: Lymfactin in clinical development
Lymfactin® in clinical development • Development of new drugs is very slow: Patient safety is always the #1 priority, and therefore years of preclinical studies are required before clinical studies – This is already completed for Lymfactin® • As the next step, clinical safety is assessed in a Phase
Slide 21: Lymfactin interim Phase 1 results
Lymfactin® interim Phase 1 results • Positive interim Phase 1 data on Lymfactin® were announced by Herantis in April 2018, in patients with breast cancer associated secondary lymphedema (BCAL) in combination with lymph node transfer • Based on data from 15 patients with BCAL: Lymfactin® is safe and
Slide 22: Phase 2 study AdeLE launched in 1H/2018
Phase 2 study AdeLE launched in 1H/2018 • AdeLE: Adenoviral gene therapy for the treatment of LE • Randomized, placebo-controlled, double-blinded Phase 2 clinical study in patients with BCAL – Total of 40 patients will be randomized 20+20 – Patients will be recruited at 5 - 6 clinical sites in Finla
Slide 23: Key Opinion Leader reflections on Lymfactin as a novel LE therapy
Key Opinion Leader (KOL) reflections on Lymfactin® as a novel LE therapy • KOLs are enthusiastic about Lymfactin® being a novel pharmacologic treatment adjunct to surgery with potential to achieve durable efficacy • Delivering VEGF-C with an adenovirus to durably treat underlying pathology is highly
Slide 24: Lymfactin as standalone therapy, and in other lymphedemas?
Lymfactin® as standalone therapy, and in other lymphedemas? • In addition to the ongoing Phase 2 study in BCAL, Herantis is considering another Phase 2: Lymfactin® as standalone therapy in any secondary LE – Possible design of the clinical study: Lymfactin® administration in patients with secondary
Slide 25: Summary on Lymfactin
Summary on Lymfactin® 6/19/18 25 www.herantis.com © 2014-2018 HERANTIS PHARMA Plc. All rights reserved.
Slide 26: Lymfactin aims at curing secondary lymphedema
Lymfactin® aims at curing secondary lymphedema • Lymfactin® is the world’s first and only clinical stage gene therapy that repairs damages of the lymphatic system • Currently in Phase 2 clinical study AdeLE in Breast Cancer Associated LE (BCAL) in Europe; initial safety has already been established
Slide 27: Thank you
Thank you Further information: CEO Pekka Simula [email protected] 6/19/18 27 www.herantis.com www.herantis.com © 2017 HERANTISHERANTIS © 2014-2018 PHARMAPHARMA Plc. All Plc. rights reserved. All rights reserved.