Histogenics - OneMed Forum 2013 Conference Presentation- 11-28

Transformational Products
in Soft Tissue Regeneration and Cartilage Repair
Company History
• Founded in 2000
• Late stage clinical company
• Privately held, based in Waltham, MA
• 42 Employees
• Acquisition of ProChon BioTech, Ltd., May 2011
• Merged cell therapy technology with tissue engineering technology
• Raised $49M Series A Round, June 2012
• Drive flagship technology through Phase III study and secure CE Mark for second lead product
• Sofinnova Ventures and Split Rock Partners
•
Seasoned Management team with extensive industry experience driving biologics
and medical devices through the clinic, commercialization, and acquisition.
•
Internal developments focused on articular cartilage tissue repair and
regeneration implants in the Orthopedics/Sports Medicine market
P. 2
Rich Portfolio of Proprietary
Technologies
3-D Scaffolds
1. Type I Collagen
2. Fibrin & hyaluronic acid
3. Nanophase hydroxyapatit
Strategic Applications:
1.
2.
Targeted delivery of cells, growth
factors, and drugs to injured tissue
Osteoconductive environment for
cellular in-growth, attachment, and
proliferation
Growth
Factors
•
FGF-2v, FGF-4v, FGF-9v
• FGF is a potent angiogenesis growth
•
factor
Enhancement of cell proliferation
Strategic Applications:
1. Therapeutic Applications
2. Reagent/Cell Expansion
Applications
•
•
•
CT3
Methylated collagen and PEG
10X stronger than fibrin-based adhesives
No necrosis
Strategic Applications:
•
Soft-Tissue adhesive and
temporary approximation
91 issued patents and applications
P. 3
Cartilage Injuries
•
Cartilage is designed to absorb shocks and
tension. It allows smooth movement of the
joint.
•
60% of injuries are post-traumatic.
•
Knee femoral condyle injury is most
common.
•
Articular cartilage is an avascular tissue
structure, incapable of self-repair.
•
As a result, patients may experience
symptoms of pain and restricted mobility,
which can progress to debilitating
osteoarthritis over time.
The current gold standard for treating
patients is a microfracture procedure
•
The Problem
‘Microfracture
repairs produce
Current
Therapies
are Inadequate
Current Solutions
Microfracture “Gold Standard”:
• Fibrous cartilage tissue regeneration leads to
inconsistent long-term results and repeat procedures
fibrocartilage… clinical outcomes reported to
deteriorate significantly 18 months post-op’
1. Kreuz PC et al, Arthroscopy 22(11),
1180–1186 (2006)
2. Frisbie DD et al, Clin Orthop. Relat Res (407),
215–227 (2003).
Mosaicplasty/Osteochondral Grafting:
• Harvest site morbidity
• Poor implant integration
• Deterioration of implant over time
Current ACI Technologies:
• Heterogeneous cell population
• Mixed fibrous and hyaline cartilage regeneration
• Unsatisfactory long-term clinical outcomes
• No major improvement on rehabilitation time
• Expensive
P. 5
Cartilage Repair & Regeneration Market
U.S. & E.U.
2.4M Cartilage Defects in 2015
$6B Clear Potential Market opportunity
•
Demographic and medical trends
• Patients 25-55 years of age, privately
insured
• Very active and strong desire to stay
active during prime years
• Highly educated consumer, driven to
avoid “bridge burning” invasive
procedures
• Aging of the population - Baby boomers
desire to stay active,
•
Significant unmet need driving regenerative medicine
•
Only 26% of defects are treated due to unsatisfactory procedures and products
P. 6
Cartilage Repair
An escalating opportunity
• Approximately 41 million children and teens in the U.S. participate in some form of
competitive sports
• Musculoskeletal injuries comprise the largest portion of athletic injuries with 25% of athletes
expecting to incur a musculoskeletal injury in any 12-month period. Knee injuries account
for 22% of all musculoskeletal injuries.
• According to the NCAA, one major knee injury occurs per football team every year.
• Approximately one-third of ski injuries involve the knee.
• More than 200 million people play soccer worldwide and up to 40% of soccer injuries involve
the knee.
• The emergence of the Adult Athlete - Approximately 150 million adults participate in some
form of competitive sport.
• NHIS study data: 9.29% of the US population will be diagnosed with symptomatic knee OA
by the age of 60.
• The A.A.O.S.M membership has increased from 209 to 3042 in the past 30 years – doubling
in the past ten years.
• Sports Medicine is the fastest growing segment of the ortho market 8%, $4.2B Market 2013.
P. 7
Addressing Large, Unmet Stage
in the Treatment Continuum
 Physical
 Injections
 Stretching
 Exercise
 Message
 Accupuncture
 Invasive Surgery
 Corticosteroids
 Viscosupplementation
 Hyaluronic acid
 Resurfacing implants
 Total joint replacement
Least
Invasive
 Behavioral
 Cognitive
 Pacing
 Relaxation
 Meditation
 Hypnosis
Most
Invasive
 Medications
 Topical
 NSAIDS
 Oral
 Acetaminophen
 NSAIDS
 COX-2 inhibitors
 Opioids
 Minimally invasive surgery
 Debridement
 Microfracture
 Mosaicplasty
 Autologous Chondrocyte
Implantation
Products: NeoCart® Autologous Neo-Cartilage Implant
Next-generation Cartilage Regeneration System
•
Target market: ICRS stage III/IV defects of the knee
•
Phase II demonstrated superiority to microfracture
• 4-yr data to be presented at the A.A.O.S conference, 2013
• 3-yr data presented at the I.C.R.S conference, 2012
• 2-yr data published in JBJS, 2012
•
Phase III IND study under Special Protocol Assessment (SPA)
• Safety: No severe adverse events related to NeoCart in Phase I or
Phase II studies.
• Efficacy: Superiority to microfracture.
•
NeoCart® Autologous cartilage
regeneration implant
 Global, debilitating, quality-of-life problem (market) currently treated by
unsatisfactory procedures or products
Patient's own cartilage infused into a patented 3D scaffold to create a
neocartilage implant
• Tissue
contains both
cells and
true cartilage
tissues.
Opportunity
tomultiplying
replace the
current
“gold standard”
• Readily integrates with surrounding healthy cartilage
with a first-line therapy
• Easy to handle, can treat large cartilage defects
• Suture-less implant
o Proprietary bioadhesive
microfracture procedure
• Strong and secure - allows for early weight-bearing and acceleration of
rehabilitation process
NeoCart® cartilage regeneration
implantation procedure
P. 9
Image of NeoCart post-implantation
NeoCart Implant at 3 Months Post-implantation
Smooth integration of hyaline cartilage implant
* Image of Phase I subject who
suffered a meniscal tear 2
weeks after the implant that
required a second
arthroscopy at 3 months;
Injury was unrelated to
NeoCart implant
Hyaline-like fill predicts high likelihood of long-term functional success
Efficacy data confirms validity of product attributes
Clinical & Regulatory
NeoCart® Cartilage Regeneration Phase III IND Clinical Study
• 245 patient multi-center randomized, superiority to microfracture
• CBER
• Special Protocol Assessment (SPA) from FDA
• Significantly De-risked regulatory path
• Primary Endpoint:
• The responder rate of the NeoCart arm is compared to the responder rate of the Microfracture
procedure arm at 12 months post-implantation.
A responder is defined as a subject that shows clinical improvement of at least 20 points in the IKDC score and at
least 12 points in the KOOS pain score.
• Currently enrolling at nation’s top orthopedic sports medicine clinical centers. The 25
sites include UCSF, Duke, Cleveland Clinic, Hospital for Special Surgery, UPenn, UConn,
OHSU, UPMC, Oschner Clinic, Harvard, and Brigham & Womens
P. 11
Phase II Data Indicates Confidence
in Achieving Phase III Superiority
• Achieved clinical significance at the 6, 12, and 24 month follow up time points
• A 54% point NeoCart responder rate difference at the 1-year primary endpoint indicates superiority to Microfracture
100%
% Responders
80%
60%
Robust
study
design,
clinical
superiority
and
SPA
40%
=
MFX
High
20% degree of confidence in achieving FDA approval
NeoCart
0%
6 mos
12 mos
Time post treatment
n = 30 at 6-­‐12 mos, n=27 at 24 mos.
24 mos
P. 12
Products: VeriCart™ Single Step
Cell-free Cartilage Repair Scaffold
• Target market: ICRS stage II/III/IV defects 3cm2 or less of the
knee
• Augmentation of microfracture procedures to improve
clinical outcome
• Off-the-shelf lyophilized, double structured collagen scaffold,
easily cut to lesion size
VeriCart™ Single Step
Cartilage Repair Scaffold
• Suture-less implant
• Proprietary bioadhesive to secure placement and position of
implant
• Attracts mesenchymal stem cells from surrounding tissue
Microscopic Images of
Scaffold Structure
• Strong and secure implant - early weight-bearing and
acceleration of rehabilitation process
• Pursue CE Mark
P. 13
Thank You
Patrick O'Donnell
President and CEO
Histogenics Corporation
830 Winter Street
Waltham, MA 02451
(781) 305-5588
podonnell@histogenics.com
www.histogenics.com