Kuros Biosciences AG «A new era in Bone Regeneration»

Transcription

1 Kuros Biosciences AG «A new era in Bone Regeneration» A new era in Orthobiologics through unique Biologics & Clinically proven portfolio Ivan Cohen-Tanugi MD CEO Harry Welten - CFO DISCLAIMER: This document is for information purposes only and does not constitute or form part of, and should not be construed as an offer or invitation to subscribe or purchase any securities of Kuros Biosciences Ltd.( Kuros ) be it in the United States or in any other jurisdiction. It should, therefore, not form the basis of any investment decision for shares of Kuros. This document contains forward-looking statements. These statements are based on information currently available to our management as well as on management's current assumptions and forecasts. Various known and unknown risks, uncertainties and other factors could lead to material differences between our actual and future results, financial situation, development or performance and the estimates given in this document. Kuros does not assume any liability to update forward-looking statements or other information in this document or to conform such forward-looking statements or other information to future events or developments. The distribution of this document may be restricted by law. Any persons reading this document should inform themselves of and observe any such restrictions. This document should not be distributed nor discussed outside Europe

2 Kuros Biosciences Table of Contents Company Overview - Investment highlights Orthobiologics MagnetOs: Orthobiologic ready for commercialization Fibrin PTH: Innovative Pipeline moving toward Phase II & III Neuroseal: Dural sealant: obtained EU clearance Kuros Biosciences: Supportive information 2

3 Kuros Biosciences / Investment highlights Public Swiss/Netherlands based company (SIX: KURN), headquartered in Zurich-Switzerland World recognized scientific founders Clemens van Blitterswijk, Jeff Hubbell, Joost de Bruijn Over 1,200 international publications Technology platform focusing on enhancing tissue regeneration, with primary focus on Bone Commercial & late stage clinical innovative pipeline: Fibrin-PTH & MagnetOs with potential >$1B 3

4 Kuros Biosciences / Investment highlights A breakthrough approach in Orthobiologics through unique Biologics & Clinically proven portfolio Introducing Controlled Bone Regeneration harnessing the body repair system through MagnetOs, Next Generation synthetic BGS Fibrin-PTH, Next Gold Standard, bioactive growth factor $2.3B+ market expected to grow to $3.5-4B by 2030* 4 * Kuros estimates

5 Sealant Orthobiologics Kuros Biosciences / Investment highlights Pipeline MagnetOs (CE Mark/ 510K) Initial Indications Registration A late stage, high value pipeline of clinical Orthobiologics Approval & sealant Granules EU Granules US Putty EU Putty US Fibrin/PTH (NDA/MAA) KUR-111 KUR-113 Orthopedics, spinal, dental Spinal fusion Orthopedics, spinal, dental Spinal fusion Initial Indications Tibial plateau fractures Tibial shaft fractures Spinal interbody fusion Non Non clinical Market Phase 1 Phase 2 Phase 3 Market leader peak sales* >US$140M Market leader global peak sales* >US$600M KUR-112 Solitary bone cyst N.A. Neuroseal (CE Mark/PMA) Initial Indications Non clinical Pilot Pivotal Registration Market leader peak sales* EU US Dural sealant (cranial) Dural sealant (cranial) >US$60M * Data on file Kuros estimates; ** only one study needed in EU; *** EU study serves as pilot for US; ****First clinical study will be a Phase II study utilizing safety data from tibial shaft fracture trial to be confirmed with regulators 5

6 Kuros Biosciences Table of Contents Company Overview - Investment highlights Orthobiologics MagnetOs: Orthobiologic ready for commercialization Fibrin PTH: Innovative Pipeline moving toward Phase II & III Neuroseal: Dural sealant: obtained EU clearance Kuros Biosciences: Supportive information 6

7 Orthobiologics What are Orthobiologics? Orthobiologics are used daily by orthopedic & spinal surgeons to repair & regenerate tissue. A major segment of orthobiologics is bone graft substitutes Bone graft substitutes are used to repair or generate bone during fracture repair or spinal fusion (growing bone across two vertebrae in the spine to alleviate pain and nerve compression) Bone graft substitutes are increasingly used instead of iliac crest autograft, the gold standard (morbidity, extra procedure cost) which is harvested from the patient 7

8 Level of Scientific Proof & Added Value Orthopedic surgery evolution From Mechanics to «Biologics & Controlled Regeneration» «Support» «Replace» «Integrate» «Regenerate» «Regenerate & Control» Growth Factors Biologics & Controlled Instructive Materials MagnetOs Fibrin PTH Orthotics Years 1800 Implants Metal, screws CaPh Bioglass >2018 8

9 Kuros Unique Scientific & Clinical Approach Unique Controlled Bone Regeneration Portfolio Bone Graft Substitute, Critical Unmet Needs Controlled new bone generation to avoid ectopic bone formation Strong Clinical Evidences Improved Safety Broader Access Controlled Bone Regeneration Fibrin PTH MagnetOs Controlled New Bone Quantity No Ectopic Bone No inflammation Controlled Admin Broader Usage Safety Building Strong Clinical Evidence 9

10 Kuros Orthobiologics Breakthrough Pipeline MagnetOs & Fibrin PTH will cover all critical needs of the market expected to grow from $2.3 to $3.5-4B by 2030* MagnetOs Synthetics DBMs Allografts Stem Cells Growth Factors Fibrin PTH 10 * Data on file, Kuros estimate

11 Kuros Biosciences Table of Contents Company Overview - Investment highlights Orthobiologics MagnetOs: Orthobiologic ready for commercialization Fibrin PTH: Innovative Pipeline moving toward Phase II & III Neuroseal: Dural sealant: obtained EU clearance Kuros Biosciences: Supportive information 11

12 MagnetOs First Controlled Instructive Bone Regeneration Graft Composition Instructive, surface structured ceramic (HA/TCP) M. o. A. Approved Indications Efficacy Safety Value Proposition MagnetOs Surface Technology utilizes the body s potential to generate bone by activating local (stem) cells Bone void filler for broad orthopedic, spine, and dental use in EU Bone graft extender for posterolateral spine fusion (US label to be expanded) Demonstrated equivalent efficacy to the standard of care autograft in key preclinical models. Clinical data to be initiated No issues Highly efficacious new class of instructive bone graft substitute Rapid osteoinductive effect/bone formation (EU claim) Improved resorption profile Expected to demonstrate superior efficacy through clinical usage 12

13 MagnetOs First Controlled Instructive Bone Regeneration Graft For MagnetOs is That Can Because So That patients having a spinal fusion (USA) / spinal fusion or trauma procedure (EU) requiring bone grafting a new generation of BGS, with unique proprietary surface structure promote progressive bone formation comparable to current gold standard autograft it has demonstrated efficacy through the recruitment & stimulation of bone regenerating cells and rapid induction together with an optimized resorption profile Surgeons can now make a difference for their patients by offering them a new and cost effective BGS 13 Key Messages For patients with severe back pain, MagnetOs due to its unique proprietary surface structure, hence its mechanism of action designed to extend bone graft (bioactive submicron structured surface topography, rapid induction and faster resorption) 1. Instruct stem cells to form bone 2. Optimized resorption profile 3. No inflammatory & swelling from putty synthetic polymer, no safety issues 4. Handling improvement with putty polymer allows bone generation 5. Enhancing patients benefits and cost effectiveness

14 Kuros Biosciences Table of Contents Company Overview - Investment highlights Orthobiologics MagnetOs: Orthobiologic ready for commercialization Fibrin PTH: Innovative Pipeline moving toward Phase II & III Neuroseal: Dural sealant: obtained EU clearance Kuros Biosciences: Supportive information 14

15 Fibrin/PTH product Family New Gold Standard Bioactive Topical Growth Factor Prolonged cell mediated release at site of need TGplPTH 1-34 (Drug) H Site of crosslinking to fibrin ASN GLN GLU GLN VAL SER PRO LEU TYR LYS ASN ARG Ser Val Ser Glu Ile Gln Leu Met His Asn TG-Hook Plasmin cleavage sites Trp Leu 1 10 Synthetic peptide developed by Kuros together with world leading CMO for peptide manufacturing (Bachem) Glu Arg Val Lys 20 Arg Lys Glu Leu Met Gln Ser Asp Asn Val PTH 1-34 (Forteo) Leu His His Asn Lys Phe Leu Gly OH PTH promotes bone formation by direct Fibrin sealant (Biologic) action on osteoblast (ARTISS, approved BAXTER) Only cells primed to form bone are activated (proliferation/ differentiation) PTH responsive cells are present where bone is damaged (periosteum and bone marrow) MBCP Granules(HA/TCP) (approved, Biomatlante ) Prolonged, local delivery of PTH boost the natural bone repair process

16 Fibrin PTH KUR Innovative Controlled & Instructive growth factors KUR-111 Best in Class Option for Tibial Plateau Fracture Composition Initial Indication Potential Uses Efficacy Safety Value Proposition Fibrin, variant PTH with HA/TCP granules Moldable paste that solidifies in situ Treatment of tibial plateau fractures as alternative to autograft Treatment of other long bone defects requiring grafting Non-inferior to autograft in Phase 2b trial (183 patients) Safe and well tolerated in clinical trials (183 patients) Does not cause ectopic bone formation Improved targeted product profile vs. leading growth-factor Safely and effectively remodels bone without need for autograft Moldable putty form for easy placement Avoids cost and morbidity associated with autograft harvesting 16

17 % patients Fibrin PTH KUR-111 Tibial Plateau Fracture: KUR 111 demonstrated non-inferiority vs Autograft in randomized & controlled PIIb in 183 patients Primary End-point: Proportion of patients (%) with radiological fracture union 16 weeks after implantation of the graft (ITT) week 52 week % 84.5% 96% 100% 98.2% % I mg/ml I mg/ml Autograft I mg/ml I mg/ml Autograft

18 Fibrin PTH KUR Innovative Controlled & Instructive growth factors Composition Spine Fusion Trauma & Tibial Shaft Fracture Fibrin + variant PTH Injectable gel that withstands irrigation & limits further bleeding Intended use Spinal Lumbar Interbody Fusion Open tibial shaft fractures as an adjunct to standard-of-care Potential Uses Efficacy Safety Value Proposition Potential for use in all long bone fractures, and other fusion proceed. Comparable to autograft and InFuse in preclinical model Significant improvement in fracture healing vs standard care Safe and well tolerated in clinical trials (200 patients) Improved targeted P.P. vs. leading growth-factor InFuse Avoids the cost and morbidity associated with autograft Can be applied in indications not targetable with other BGS Reduces number of repeat operations (improved healing) 18

19 % patients Fibrin PTH KUR-113 Superior Fracture Healing vs. Standard of Care in a randomized and controlled 200 patient Phase IIb Clinical Trial 100 Trial Design: Randomized trial of 200 patients 80.4% with tibial shaft fractures; patients randomized to four p=0.084 (10% sig level) treatment 90 groups: % mg/ml active (n=50), 0.4 mg/ml active (n=50), 1.0 mg/ml active (n=50), standard of care alone** (n=50) with follow-up at 6 months 69.2% 64.6% I mg/ml I mg/ml I mg/ml SoC Fibrin/PTH 0.4 mg/ml statistically significant vs SoC alone 19

20 Fibrin PTH KUR-113 Reduction by over 50% of secondary intervention rate (due to persistent tibial non-union) 6 month 12 month 15.0% 25.0% 12.0% 12.5% 20.0% 19.1% 9.0% 7.7% 15.0% 6.0% 4.4% 4.3% 10.0% 6.8% 8.7% 7.5% 3.0% 5.0% 0.0% mg/ml (n= 45) 0.4 mg/ml (n=46) 1 mg/ml (n=39) SoC (n=48) 0.0% mg/ml (n= 44) 0.4 mg/ml (n=46) 1 mg/ml (n=40) SoC (n=47) Clinically significantly less patients in the Fibrin/PTH groups required secondary interventions * Statistically significant difference vs. standard of care

21 MagnetOs & Fibrin PTH portfolio positioning Risk of non healing (incl according to patient profile & comorbidities) Spine Accessibility Fracture type Broader access New indication Patient Access (insurance and/or patients) MagnetOs Fibrin PTH Use preferably if Low Risk of Non Consolidation Use preferably if High Risk of Non Consolidation or Comorbidities PLF Fill large bone voids Together for tibial plateau or spine fusion (PLF portion) Minimal invasive Interbody fusion procedures Long bone fractures Fibrin PTH access denied Fibrin PTH access 21

22 Kuros Orthobiologics: A Breakthrough Pipeline MagnetOs & Fibrin PTH will cover all critical needs of the market expected to grow from $2.3 to ~4B by 2030 Allografts Synthetics 2016 $2.3B+ DBMs Synthetics Incl. MagnetOs Allografts DBMs Est. 2030* $3.5-4B Stem Cells Growth Factors Stem Cells Growth Factors Incl. Fibrin PTH By 2030 MagnetOs & Fibrin PTH expected to be the gold standard in their respective segment 22 * Data on file, Kuros estimate

23 Fibrin PTH KUR-112 for Solitary Bone Cysts Pediatric Rare Disease Composition Intended use Efficacy Safety Value Proposition Fibrin, variant PTH Injectable gel that solidifies in situ Solitary Bone Cysts a rare pediatric disease -> pathological fractures Efficacious in clinical cases of osteochondral cysts in horses Efficacious technology for bone generation (KUR-111 and -113) Significant body of safety data available in hundreds of patients Phase II/III to demonstrate improved clinical efficacy compared to current standard of care Avoids the cost and morbidity associated with frequent need for repeat treatment with current practice Orphan drug designation (US & EU) + Rare Pediatric Disease designation (US) ->> Priority Review Voucher

24 Kuros Biosciences Table of Contents Company Overview - Investment highlights Orthobiologics MagnetOs: Orthobiologic ready for commercialization Fibrin PTH: Innovative Pipeline moving toward Phase II & III Neuroseal: Dural sealant: obtained EU clearance Kuros Biosciences: Supportive information 24

25 Neuroseal Sealant EU approval Dural Sealant Overview Brain Spinal cord Dura The dura surrounds the brain and spinal cord. It is the barrier that separates the cerebrospinal fluid (CSF) from the body Dural sealants are developed as an adjunct to suturing after cranial or spinal surgery to avoid CSF leakage Post-operative CSF leaks result in significantly increased costs (140%) due to increased hospital stay and additional treatment costs (Grotenhuis, J. 2005) Current market leading dural sealant (cranial & spine) is generating peak sales of US$60M in the US (Data on file, Kuros estimates) 25

26 Neuroseal Received EU clearance in June 2017 Overview Neuroseal Composition Mechanism of Action Therapeutic Use Efficacy Safety Sprayable, synthetic, fully biodegradable gel Gel that efficiently adheres to and seals the dura Adjunct to suturing to provide water-tight closure of the dura in cranial surgery Potential for use in spinal dural closure (not approved) Intra-operative sealing successful in all evaluable patients in clinical trial Safe and well tolerated Value Proposition Highly efficient sealing Significantly less swelling expected vs competition and fewer complications due to nerve compression Slower degradation and higher mechanical strength 26

27 Neuroseal Road Map 1 Primary focus EU5 until new clinical trials initiated 2 Initiate usage and build confidence with EU KOLs in Evaluate broader indications, partnerships, distributors, licensing

28 Kuros Biosciences Table of Contents Company Overview - Investment highlights Orthobiologics MagnetOs: Orthobiologic ready for commercialization Fibrin PTH: Innovative Pipeline moving toward Phase II & III Neuroseal: Dural sealant: obtained EU clearance Kuros Biosciences: Supportive information 28

29 Kuros Biosciences No debt Number of shares outstanding: 7.8 mln* Key shareholders: - Incubation/Aldabra/Huipin (Xpand sellers) 17.5% - Banque Pictet 9.0% - LSP 7.6% - Eckenstein-Geigy Foundation 6.1% - Venture Incubator 5.8% - Omega Funds 6.0% - FCPI 3.6% - Didier Cowling 3.1% 29 *As of 8/31/2017

30 Kuros Biosciences 2017 Achievements January: Acquisition X-pand BV (including MagnetOs) February: Kuros achieves ISO certification for surgical sealants February: Kuros receives US clearance for MagnetOs granules June: EU clearance (CE mark) Neuroseal June: Kuros raised CHF16.9M August: US clearance (510k) MagnetOs putty August: EU filing MagnetOs putty 30

31 Kuros Biosciences Key Critical Next Steps MagnetOs putty clearance EU MagnetOs launch & initiation of supportive clinical trial Initiation Fibrin PTH PII clinical trial in Spine Initiation Fibrin PTH PIIb/III clinical trial in pediatric SBC Initiation Fibrin PTH PIII clinical trial in trauma indication 31

32 Kuros Biosciences / Investment highlights A breakthrough approach in Orthobiologics through unique Biologics & Clinically proven portfolio Introducing Controlled Bone Regeneration harnessing the body repair system through MagnetOs, Next Generation synthetic BGS Fibrin-PTH, Next Gold Standard, bioactive growth factor $2.3B+ market expected to grow to $3.5-4B by 2030* 32 * Kuros estimates

33 33 Thank you