PARADIGM SHIFT FOR BLOOD CANCER PATIENTS JANUARY 2015 I N T R O D U C T I O N F O C U S Development of life saving T-‐cell immunotherapy based products focused on limita5ons of stem cell transplants in terminally ill blood cancer pa5ents where pharmacological standard of care is no longer an alterna5ve F O U N D E D November 1997 M A N A G E M E N T Manfred Rüdiger, PhD Chief Execu5ve Officer Robbert van Heekeren Chief Financial Officer Jeroen Rovers, MD PhD Senior Vice President and Chief Medical Officer Margot Hoppe General Counsel and Corporate Secretary I N V E S T O R A N D M E D I A R E L A T I O N S Consilium Strategic Communica5ons Mary-‐Jane EllioY, Amber Bielecka, Lindsey Neville Tel: +44 203 709 5708 Email: kiadis@consilium-‐ comms.com ADDRESS Kiadis Pharma 1114 AA Amsterdam-‐ Duivendrecht The Netherlands Tel: +31 (0)20 314 02 50 www.kiadispharma.com @KiadisPharma ABOUT KIADIS PHARMA Kiadis Pharma is a clinical stage biopharmaceu5cal company focused on developing innova5ve and poten5ally life-‐saving therapies for blood cancer pa5ents where current pharmacological standard of care is no longer an alterna5ve. The Company’s main focus is on the development of a T-‐cell immunotherapy that will enable par5ally matched donor stem cell transplants from family members in blood cancer pa5ents, thus making them safe and available as the last poten5ally life saving op5on. Stem cell transplanta5on is currently the only poten5ally cura5ve treatment available for late stage blood cancer pa5ents; however, a standard of care matching donor is only available for half of pa5ents in need of and eligible for transplanta5on. Kiadis Pharma is headquartered in Amsterdam, The Netherlands, and works closely with renowned centers in Europe and North America. The Company is supported by a consor5um of leading interna5onal biotech investors, including Life Sciences Partners, DFJ Esprit, Alta Partners, Quest for Growth, NOM and MedSciences Capital. LEAD PRODUCT The Company’s lead product ATIR™ addresses the shortage in availability of matched donors by enabling a stem cell transplanta5on from family members who are only par5ally matched (“haploiden5cal”) to the pa5ent. ATIR™ is a T-‐cell immunotherapy based medicinal product that, when administered as an adjunc5ve to a T-‐cell depleted haploiden5cal stem cell transplanta5on, facilitates early immune system recovery without causing life-‐threatening GraW-‐versus-‐Host-‐Disease (GvHD), a process by which the pa5ent is aYacked by its newly transplanted immune system. ATIR™ thus synergizes with approaches such as Chimeric An5gen Receptor (CAR) T-‐cell therapies that put refractory pa5ents into remission, thereby rendering them eligible for transplanta5on. The Company is currently sponsoring an interna5onal Phase II clinical study for ATIR™ to confirm and extend earlier posi5ve Phase I/II safety and efficacy data and in December 2014 posi5ve interim data from the ongoing trial were presented at the American Society of Hematology (ASH) Annual Mee5ng in San Francisco. TECHNOLOGY Kiadis Pharma’s technology is derived from the patent-‐protected Theralux product placorm. This placorm makes use of a novel compound, TH9402, which selec5vely accumulates in rapidly dividing cells, like cancer cells and in ac5vated immune cells, and which is phototoxic upon radia5on by light of a specific wavelength. TH9402 enters all cells by passive diffusion and is normally extruded through a molecular pump. Rapidly dividing cells such as cancer cells and ac5vated immune cells however extrude the compound less efficiently. Thus, the compound accumulates in cancer cells and in ac5vated immune cells. AWer being photo ac5vated, the compound TH9402 kills the cells in which it accumulates. CLINICAL DEVELOPMENT The interna5onal Phase II clinical study for ATIR™, ini5ated in 2013, is currently ongoing with submission for condi5onal marke5ng authoriza5on expected in 2016 and product launch expected in 2017. The product has orphan designa5ons from the FDA and EMA for the preven5on of acute GvHD following an allogeneic bone marrow transplanta5on and from EMA for the treatment of acute myeloid leukemia (AML). The Phase II confirmatory study is an open-‐label, mul5-‐center study. The primary endpoint is Transplant Related Mortality (TRM) at 6 months post Haploiden5cal Stem Cell Transplanta5on (HSCT) and secondary endpoints are acute/chronic GvHD, immune recons5tu5on, infec5ons and TRM/Relapse-‐ Related Mortality/Overall Survival/Progression-‐Free Survival. The pa5ent popula5on consists of up to 23 pa5ents with AML, acute lymphoblas5c leukemia (ALL) and myelodysplas5c syndrome (MDS) eligible for a HSCT. Data from the pre-‐specified interim analysis of the first 10 pa5ents, announced in December 2014, showed only 2 cases of TRM at 6 months post-‐HSCT and both TRM cases resulted from a viral infec5on. Overall, 6 out of the 10 pa5ents were completely free from severe infec5ons (grade 3-‐5) aWer ATIR™ administra5on and up to 6 months aWer the transplant. These data confirm the safety and efficacy of ATIR™ administra5on with no grade III-‐IV (life-‐threatening) acute GvHD occurring, despite the fact that no prophylac5c immune suppressants were used. Only a limited number of grade I-‐II GvHD events were reported, all of which were treated successfully. Furthermore, no relapses were reported in any of the first 10 pa5ents, with 4 out of the 10 pa5ents being in follow-‐up for more than one year at the 5me of the interim analysis. The study centers are in Montreal, Hamilton and Toronto in Canada as well as Leuven, Brussels and Bruges in Belgium. HOW DOES ATIRTM WORK? MANUFACTURING A robust manufacturing process with potency and safety release tests has been established by Kiadis Pharma using a quality by design approach working with interna5onal cell therapy leaders and with input from EU and US regulators, clinicians and GMP manufacturing experts. External manufacturers are used for the produc5on of clinical trial material. Kiadis Pharma benefits from a GMP manufacturing license and GMP cer5ficate for its QC laboratory from the Dutch Ministry of Health, Welfare and Sport. ATIR™ is currently manufactured at sites in North America and Europe. MARKET POTENTIAL This is a blockbuster product providing life saving poten5al to ~20,000 pa5ents a year, with an expanding €1bn primary market and complementary product trends. The primary North American & European market is growing: pa5ents not having an iden5cal matching sibling or a matching unrelated donor in 5me. There is also poten5al for expansion in the over-‐70 age group. Furthermore, today approaches such as CAR T-‐cell therapies provide increased op5ons to put more pa5ents into remission1. Growth can also take place through cannibalizing cord blood transplanta5ons as well as the current matched unrelated donor popula5on. The product has IP protec5on to 2022 and may benefit from market exclusivity up to 10 years post launch in the EU and 7 years in the US due to its orphan drug status. Davila et al., Science Transla5onal Medicine 2014; 6:224ra25 1
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