ALLOB® IN RESCUE SPINAL FUSION

BONE THERAPEUTICS
Finance Avenue
14 November 2015
DISCLAIMER
This document and the accompanying oral presentation contain information on Bone Therapeutics SA’ markets and competitive position,
and more specifically, on the size of its markets. This information has been drawn from various sources or from Bone Therapeutics SA own
estimates. Investors should not base their investment decision on this information. This document and the accompanying oral presentation
also contain certain forward-looking statements. These statements are not guarantees of the Company's future performance. These
forward-looking statements relate to the Company's future prospects, developments and marketing strategy and are based on analysis of
estimates not yet determinable. Forward-looking statements are subject to a variety of risks and uncertainties as they relate to future events
and are dependent on circumstances that may or may not materialize in the future.
Bone Therapeutics SA draws your attention to the fact that forward-looking statements cannot under any circumstance be construed as a
guarantee of the Company's future performance and that the Company’s actual financial position, results and cash flow, as well as the
trends in the sector in which the Company operates may differ materially from those proposed or reflected in the forward-looking statements
contained in this document and the accompanying oral presentation. Furthermore, even if Bone Therapeutics SA financial position, results,
cash-flows and developments in the sector in which the Company operates were to conform to the forward-looking statements contained in
this document and the accompanying oral presentation, such results or developments cannot be construed as a reliable indication of the
Company's future results or developments. Certain figures and numbers appearing in this document and the accompanying oral
presentation have been rounded. Consequently, the total amounts and percentages appearing in the tables are therefore not necessarily
equal to the sum of the individually rounded figures, amounts or percentages.
2 BONE THERAPEUTICS – 14 November 2015
KEY INVESTMENT HIGHLIGHTS
1
HUGE TARGETED MARKETS
(12M PATIENTS(1), 30% OF A
$34Bn MARKET(2)) WITH
SIGNIFICANT HIGH UNMET
MEDICAL NEEDS
4
WITH STRONG CLINICAL
RESULTS - 2 ONGOING
PHASE III trials
2
CELL THERAPY APPROACH
ENABLES FRACTURE
REPAIR AND PREVENTION
3
BONE THERAPEUTICS
A LEADER IN BREAKTHROUGH
BONE CELL THERAPY
(1) Europe, US, Japan (Company estimates) (2) For the global fracture repair and prevention market: Transparency Market Research: Osteoporosis Drugs Market (2013) & The orthopaedic industry annual report (2013)
3 BONE THERAPEUTICS – 14 November 2015
SKILLED AND EXPERIENCED MANAGEMENT TEAM
Enrico Bastianelli, MD, MBA
Wim Goemaere, MAE
Chief Executive Officer & co-founder
Chief Financial Officer
► 21 years experience in pharma/biotech:
— Founder & VP Corporate Development at
ProSkelia/ProStrakan
— Consultant at McKinsey & Co
— Marketing at Procter & Gamble
► 26 years experience in finance:
— CFO Devgen (sold to Syngenta for €400m)
— Finance Director at Flanders Institute for Biotechnology (VIB)
— Finance & control at BP
Valérie Gangji, MD, PhD
Thomas Lienard, MBA
Chief Medical Officer & co-founder
Chief Business Officer
► 21 years experience in rheumatology and medical research:
— Head of the Rheumatology Dept. of Erasme University
Hospital (Brussels, Belgium)
— Responsible for bone cell therapy program and pain
clinic
► 15 years international experience in sales and marketing:
— General Director at Lundbeck
— Several positions at Eli Lilly and Company in Belgium and the
US, including Sales Director Belgium
— Scientific expertise in Rheumatology (bone diseases)
Guy Heynen, MD
Chief Clinical & Regulatory Officer
► 35 years experience in the medical & regulatory field:
— Clinical & Medical research at Pfizer
— European team leader for Alzheimer’s drugs & US team leader
for anti-inflammatory drug franchise
4 BONE THERAPEUTICS – 14 November 2015
► Started at Bone Therapeutics on November 9, 2015 and will
take up responsibility concerning activities in:
— business development,
— business operations and
— strategic planning.
CELL THERAPY: A GAME CHANGER IN ORTHOPAEDICS
Rationale of bone cell therapy in orthopaedics and bone diseases
Production of bone-forming cells
Skeleton: a naturally
regenerative system
Limitation of standard
orthopaedic approaches
Minimally invasive administration
5 BONE THERAPEUTICS – 14 November 2015
BALANCED MECHANISM OF NATURAL BONE REGENERATION
A balanced mechanism characterizes bone natural regenerative system
This system is impaired in orthopaedic conditions and bone diseases
Balanced
Osteoblasts
Formation
Non-balanced
Osteoclasts
Resorption
Osteoblasts
Shortfall
Osteoclasts
NORMAL BONE
DISEASED BONE
Cell therapy
=
* Cells naturally dedicated to bone formation
6 BONE THERAPEUTICS – 14 November 2015
Administration of osteoblasts* to restore a
healthy bone environment
L’APPROCHE UNIQUE DE BONE THERAPEUTICS EN THÉRAPIE CELLULAIRE
Bone Therapeutics : une expertise unique dans la production d’ostéoblastes
Processus de plusieurs mois
Cellules souches
mésenchymateuses
7 BONE THERAPEUTICS – 14 November 2015
Ostéoprogéniteurs
Ostéoblastes
MINI-INVASIVE
► Rapide (20 min.) et ambulatoire
► Action locale au site de fracture
ALP
DE LA
FORMATION
OSSEUSE
AMPLIFICATION
DU PROCESSUS
NATUREL DE
FORMATION
OSSEUSE
Os
► Une seule injection locale
percutanée
IMPLANTATION
INITIATION
Ostéocytes
VEGF
SDF-1
ALP
► Remplacement des cellules
osseuses défectueuses /
manquantes
► Recrutement des cellules du
patient
► Création d’un nouvel
environnement osseux sain
MARCHÉS CIBLES : RÉPARATION ET PRÉVENTION DE FRACTURES
RÉPARATION
DE FRACTURES
Fractures sévères non
consolidées et fusion vertébrale
Fractures non
consolidées et
avec retard de
consolidation
Population adressable
(~0.3 M + 1 M)
8 BONE THERAPEUTICS – 14 November 2015
Fusion
vertébrale
Population adressable
(~0.5 M + 25%
de procédures de réparation)
PRÉVENTION DE FRACTURES
Fragilité osseuse avec risque
élevé de fracture
Ostéonécrose
Ostéoporose
Population adressable
(~0.2 M)
Population adressable
(~10 M)
BROAD LATE STAGE PRODUCT PIPELINE
Two products: autologous (PREOB®) & allogeneic (ALLOB®)
Six indications in fracture REPAIR & PREVENTION
PREVENTION
REPAIR
Indication
9 BONE THERAPEUTICS – 14 November 2015
Platform
Non-Unions
PREOB®
Delayed-Unions
ALLOB®
Spinal Fusion
ALLOB®
Rescue Spinal
Fusion
ALLOB®
Osteonecrosis
PREOB®
Osteoporosis
PREOB®
Preclinical
Phase I/IIA
Phase IIB/III
KEY ACHIEVEMENTS 2015
► Established a US subsidiary in Boston
► Opened new headquarters at the Gosselies Biopark
Corporate
► Strengthened the Board of Directors
► Appointed CBO
Important advancements in the Phase II proof-of-concept trials
► ALLOB® Phase I/IIA delayed-union trial
– Positive efficacy results in first patient cohort
– Treated second patient cohort without safety concerns
Pipeline
► First safety results in ALLOB® Phase IIA spinal fusion trial
► First results from PREOB® Phase IIA severe osteoporosis trial
► Initiated novel Phase IIA trial for ALLOB® in rescue spinal fusion
► Successful €37M IPO on Euronext Brussels and Euronext Paris
Financial
► Cash position at the end of June 2015 of €37.2M
10 BONE THERAPEUTICS – 14 November 2015
ESSAI DE PHASE IIA AVEC ALLOB® EN FUSION VERTÉBRALE
1 M d’interventions chirurgicales par an en EU et aux US, dont 0,5 M au niveau lombaire, avec une croissance de 5 à 10% par an
Progression lente jusqu’à la fusion et échec du traitement dans 5% à 35% des cas
Recul des ventes de Infuse (BMP2) – Avertissement de la FDA et problèmes de sécurité
► Phase IIA de preuve de concept
► 16 patients avec des symptômes de discopathie
dégénérative lombaire, nécessitant une procédure
de fusion vertébrale
► Objectifs : sécurité et efficacité (capacité
fonctionnelle et fusion)
► Durée : 12 mois de suivi
► Premier groupe de 4 patients traité
Tomodensitométrie coronale indiquant le segment du disque central
(voir ligne continue), périphérique (voir traits) et marginal (voir la flèche)
11 BONE THERAPEUTICS – 14 November 2015
ESSAI DE PHASE IIA AVEC ALLOB® EN FUSION VERTÉBRALE
Progression lente jusqu’à la fusion
pre-op
12 BONE THERAPEUTICS – 14 November 2015
1 an post-op
4 ans post-op
ESSAI DE PHASE IIA AVEC ALLOB® EN FUSION VERTÉBRALE
L’administration locale d’ALLOB® vise
une formation osseuse meilleure et plus
rapide dans la fusion lombaire
ALLOB®
Traitement de référence
Cages intersomatiques
Granules biocéramiques
► Traitement de référence avec approche chirurgicale combinant
cages intersomatiques et granules biocéramiques / ALLOB®
► Une seule application (en plus du traitement de référence)
► Pas de problème de sécurité dans la première cohorte de patients
Prochaine étape
13 BONE THERAPEUTICS – 14 November 2015
Résultats intérimaires de sécurité sur les 8 premiers patients et résultats d’efficacité sur les 4
premiers patients
ALLOB® IN RESCUE SPINAL FUSION – PHASE IIA TRIAL
Spinal fusion failure: up to 25% after initial spinal fusion surgery with non-union and persisting pain
Standard-of-care revision (open) surgery: additional difficulties, associated with several complications
► Phase IIA, open, multicentre, proof-of-concept trial
► Single minimally invasive (percutaneous)
implantation of ALLOB® into failed fusion site
► 16 patients with a failed lumbar spinal fusion
(15 months after the initial fusion procedure)
► 12-month study follow-up
► Study endpoints: clinical symptoms (pain &
disability) and radiological healing
14 BONE THERAPEUTICS – 14 November 2015
ALLOB® IN RESCUE SPINAL FUSION – PHASE IIA TRIAL
Failed spinal fusion with standard-of-care
Pre-op
15 BONE THERAPEUTICS – 14 November 2015
6-month post-op
21-month post-op
ALLOB® IN RESCUE SPINAL FUSION – PHASE IIA TRIAL
16 BONE THERAPEUTICS – 14 November 2015
ALLOB® IN RESCUE SPINAL FUSION – PHASE IIA TRIAL
Lumbar vertebrae
Trephine
Failed lumbar fusion
Pedicular screws
Next step
17 BONE THERAPEUTICS – 14 November 2015
Interim safety results of first 4 patients
KEY FINANCIAL FIGURES
Six months ended
Six months ended
June 30, 2015
June 30, 2014
Operating income
1,984
1,574
Grants income
1,984
1,574
(7,342)
(4,237)
R&D expenses
(5,271)
(3,602)
G&A expenses
(2,071)
(635)
Operating result
(5,358)
(2,663)
Financial result
(1,764)
(122)
Net result
(7,122)
(2,785)
Net cash flow
25,646
(817)
(8,114)
(2,383)
(978)
(1,594)
34,737
3,161
37,222
11,576*
In thousands of euros
Operating expenses
Operating activities
Investing activities
Financing activities
Cash position
18 BONE THERAPEUTICS – 14 November 2015
*Cash at December 31, 2014
Of which €1.06 million was accounted for as
IPO-expenditure
Of which €34.62 million from the issue of
equity instruments (net of issue costs of
€2.75 million)
UPCOMING CLINICAL NEWS
Indication
Platform
Non-Unions
PREOB
®
Actions
Efficacy on 4 patients
Delayed-Unions
ALLOB
2016
Q3
✓
Efficacy on 8 patients
Safety on 4 patients
ALLOB
✓
Safety on 8 patients
Study status for Interim/DSMB
Spinal Fusion
Q2
Study status for Interim/DSMB
Launch of US clinical trial
®
2015
Q1
®
✓
Efficacy on 4 patients
Safety on 8 patients
Efficacy on 8 patients
Initiation study
Rescue Spinal
Fusion
ALLOB
®
✓
Safety on 4 patients
Efficacy on 4 patients
Safety on 8 patients
Site recruitment update
Osteonecrosis
PREOB®
Patient recruitment for DSMB
Launch of US clinical trial
Safety on 8 patients
Osteoporosis
PREOB
®
Efficacy update
Recruitment completed
Safety on 16 patients
19 BONE THERAPEUTICS – 14 November 2015
✓
Q4
Q1
Q2
2017
Q3
Q4
Q1
Q2
Q3
Q4
CONTACT
Enrico BASTIANELLI, MD, MBA
Chief Executive Officer
Wim GOEMAERE, MAE
Chief Financial Officer
Phone: +32 (0)2 529 59 90
Fax: +32 (0)2 529 59 93
E-mail address: [email protected]
Postal address: Rue Auguste Piccard 37, 6041 Gosselies
Website: www.bonetherapeutics.com
20 BONE THERAPEUTICS – 14 November 2015