Post on 12-Aug-2020
Redefining Regenerative Medicine
2019 BIO Investor Forum
Richard W. PascoeCHAIRMAN AND CEO
October 22-23, 2019
Forward Looking Statement
This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, including statements regarding our future results of operations and financial position, business strategy, prospective products, product approvals, research and development costs, timing and likelihood of success, plans and objectives of management for future operations, and future results of current and anticipated products, are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential” or “continue” or the negative of these terms or other similar expressions. These statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond our control, you should not rely on these forward-looking statements as predictions of future events. The events and circumstances reflected in our forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. All forward-looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation to reflect events or circumstances after the date hereof. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. In addition, projections, assumptions, and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk.
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Corporate Overview
A private company with an innovative biological technology to replace and regenerate tissues in the body, addressing underserved, multibillion dollar global markets
Corporate Overview
Histogen
A regenerative
therapeutics company
with a unique biological
platform that replaces and
regenerates tissues in the
body
Technology
Our proprietary
manufacturing process
yields multiple biologic
products from a single
bioreactor. This
innovative technology
platform delivers several
attractive product
opportunities
Marketed Product
Cell Conditioned Media
(CCM) cosmetic skincare
products
Product Candidates
• HST 001 – Hair
Stimulating Complex
(HSC) – hair growth in
men
• HST 002 – Human
Extracellular Matrix
(hECM) – dermal filler
• HST 003 – Human
Extracellular Matrix
(hECM) – joint cartilage
regeneration
• HST 004 – Human
Extracellular Matrix
(hECM) – spinal disc
regeneration
1. 2. 3. 4.
Intellectual
Property
Strong intellectual
property portfolio with
50 global patents
issued and pending
Markets
• Large, global and
accessible
• Facial dermal fillers
for facial wrinkles,
cheek and lip
augmentation
• Joint cartilage repair
• Hair regrowth
2019 2020
Submit IDE for
HSC002 – Q4
Submit IND
Amendment for
HSC001 – Q1
Initiate HST 002
Pilot Trial – Q1
Submit IND
for HST 003 – Q2
Initiate HST 001
Phase 1b Trial – Q2
Initiate HST 003
Phase 1 Trial – Q2
Complete HST 002
Pilot Trial – Q3
Complete HST 001
Phase 1b Trial – Q4
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Executive Leadership Team
5
Closed multiple business
development transactions
valued at over $2.2
billion.
Led companies
through development
and approval of multiple
prescription drugs
in the U.S. and Europe.
Commercially launched
prescription drugs in
the U.S. generating
net sales of over $1 billion in multiple
therapeutic categories.
Gail Naughton, Ph.D.
Founder/Chief Scientific Officer
Martin Latterich, Ph.D.
Sr. VP of Technical Operations
Richard Pascoe
Chairman and CEO
Technology Platform
Histogen’s unique technology utilizes human proteins and growth factors produced by hypoxia-induced multipotent cells to replace and regenerate tissues, addressing underserved, multibillion dollar global markets
Technology Platform
Histogen has successfully developed a production process which is highly
scalable and economically viable. All of the derivatives from this single
proprietary manufacturing process can be used, creating a spectrum
of products for a variety of markets from one core technology:
1. An insoluble human extracellular matrix (hECM) for applications such
as orthopedics and soft tissue augmentation which can be fabricated into a
variety of forms for tissue engineering and clinical applications
2. A biologic for hair growth – Hair Stimulating Complex (HSC)
3. A soluble multipotent cell conditioned media (CCM) that is the starting
material for products for skin care and other applications
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More than 50 patents
have been issued and
filed worldwide on
Histogen’s technology
and the compositions
produced through its
manufacturing process.
Histogen Manufacturing Process
Extracellular Matrix
(hECM)
Hair Stimulating Complex
(HSC)
Cell Conditioned Media
(CCM)
Histogen Product Pipeline
Preclinical Phase 1 Phase 2 Phase 3 Marketed
CCM Cosmetics
HST 001 Hair Growth
(HSC)
HST 002 Dermal Filler
(hECM)
HST 003 Joint Cartilage
(hECM)
HST 004 Spinal Disc
(hECM)
Marketed – Allergan and HydraFacial
Phase 1b
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HSC Hair Growth
A minimally-invasive treatment to promote new hair growth where existing treatments only reduce hair loss
HST 001 - Hair Growth US Market Opportunity
Addressable Market:
$3.6 billion spent on hair loss, targeting men aged 30 – 491
Nature of Treatment:
HSC injections of 0.1mL throughout thinning area of the scalp,
administered by dermatologists and cosmetic surgeons
Market Growth:
2% per year, driven by aging population1
Market Need:
Patients seek a safe, minimally-invasive treatment to achieve cosmetically
relevant new hair growth
Peak Sales Potential:
$684 million2
1 Histogen market research2 Histogen estimate
The hair growth market
is an underserved,
multi-billion dollar global
opportunity.
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HST 001 Clinical Proof of Concept
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Early trials have shown
stimulation of new hair
growth in both men and
women1
Male
Baseline 18 Weeks
Female
1 Investigator-initiated Phase I
clinical study of HSC in male and
female volunteers (US IND 114184)
HST 001 Clinical Proof of Concept
Three Completed Company-Sponsored Clinical Trials
Pilot in male pattern hair loss
Phase 1a in male pattern hair loss
Phase 1a in female diffuse hair loss
Statistically and Cosmetically Significant New Hair Growth
Over 85% of men treated showed new terminal hair growth across all studies
Strong Safety Profile
No kidney, liver or bone marrow toxicity
No severe adverse events
Treatment well tolerated across all subjects
Active IND
US IND 108935
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Pilot Trial
Phase 1a
12 Weeks
*
**
*p=0.033 **p=0.0135
*
**
*p<0.050 **p=0.029
HST 001 Phase 1b Protocol Summary
Objective:
• To determine optimal dosing of HSC in male pattern hair loss
Enrollment Criteria:
• 21 men, aged 20-55
• Male Pattern Hair Loss identified as Norwood-Hamilton 2A, 3, 3V, 4,& 4A
Treatment Regimen:
• 20 injections of HSC week 0, week 6, & week 12
• 20 injections of HSC week 0 & week 6, placebo (PBS) week 12
• 20 injections of placebo (PBS) week 0, week 6, & week 12
Endpoints:
• Adverse events, and other safety parameters
• Non-vellus hair count vs. baseline week 18, measured by Canfield
macrophotography
• Total count, hair thickness density, terminal and vellus hair counts at week 26,
as measured by Canfield macrophotography
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Male Pattern Hair Loss
Phase 1 clinical study
target enrollment Q1/2020
HST 001 - Hair Growth Competitive Differentiation
HSC’s human proteins and growth factors have been shown in early studies to
stimulate resting hair follicles to produce new cosmetically-relevant hair.
A treatment showing efficacy in new patients groups could not only capture
existing market share but expand the size of the market overall.
Target Profile:
• Stimulating new long-lasting hair growth
• Effective over the entire head
• Effective in treating temporal recession
• Effective on both men and women
• Minimally invasive
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Results from additional
clinical trials may support
a high value
pharmaceutical
partnership or exit
hECM Fillers
Human-derived collagen and extracellular matrix dermal fillers for delivering longer-lasting effects for the treatment of facial folds and wrinkles addressing a fast growing global market
HST 002 - Dermal Filler US Market Opportunity
Addressable Market:
1.2 million receiving dermal fillers,10 million considering dermal fillers1
Nature of Treatment:
Lower face, cheek, and lip account for ~60% of treatments. Patients receive
approximately 1.7 treatments per sitting
Market Growth:
7% per year, driven by growing acceptance and higher volumes1
Market Need:
Physicians seek extended longevity beyond 6-9 months
Peak Sales Potential:
$520 US million2
1 Histogen market research2 Histogen estimate
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HST 002 Phase 1 Protocol Summary
Objective:
• Evaluate an all human extracellular matrix (hECM) as a dermal filler, in direct
comparison to Restylane-L in moderate to severe nasolabial folds
Enrollment Criteria:
• 22 subjects, men and women, with moderate to severe nasolabial folds
• 2 clinical sites
Treatment Regimen:
• hECM filler (up to 3 mL per nasolabial fold)
• Restylane-L (up to 3 mL per nasolabial fold)
Endpoints:
• Adverse events, and other safety parameters
• Responder rate based on Evaluating Investigator assessment of nasolabial
folds at month 3
• FACE-Q Appraisal at month 3
• Participant assessments of global aesthetic improvement at months 3 and 6
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Dermal Filler
IDE filing expected 2019
Phase 1 clinical study
target enrollment Q1/2020
HST 002 - Dermal Filler Competitive Differentiation
Human-derived collagen and extracellular matrix dermal filler injected into
dermis.
Long-lasting effects for the treatment of facial folds and wrinkles.
hECM filler that provides the natural proteins found in young healthy skin
Target Profile:
• Only filler providing all-human and naturally produced collagen with dermal
matrix proteins
• Provides ECM proteins as a volumetric filler to produce long-lasting effects
~12 months
• Potentially reduces risk of inflammation seen with other fillers
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hECM Joint Cartilage
Histogen’s human extracellular matrix for regenerating hyaline cartilage for the treatment of articular cartilage defects with a unique malleable scaffold that stimulates the body’s own stem cells.
HST 003 - Joint Cartilage US Market Opportunity
Addressable Market:
0.2 million receiving joint cartilage repair, 17.5 million considering joint cartilage
repair1
Nature of Treatment:
Patients receiving arthroscopic microfracture or cartilage transplant treatments
due to acute injury and/or osteoarthritis
Market Growth:
2% per year, driven by aging population and growing obesity1
Market Need:
Physicians desire treatments that reverse cartilage degeneration
Peak Sales Potential:
$1.18 US billion2
1 Histogen market research2 Histogen estimate
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HST 003 -Preclinical Proof of Concept
In vivo efficacy in rat model
• Untreated defects (10): extensive fibrotic tissue and no articular cartilage at
6 weeks
• Treated defects (10): congruent and smooth cartilage regeneration and
integration with surrounding cartilage at 6 weeks
• Statistically significant integration of repaired tissue (O’Driscoll score
p<0.001)
In vivo efficacy in rabbit model
• Untreated defects (12): fibrous tissue that did not restore the articular surface
at 24 weeks
• Treated defects (12): articular cartilage formation with structural integrity and
homogeneity at 24 weeks
• Statistically significant integration of repaired tissue (O’Driscoll score
p<0.001)
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POC firmly established in multiple animal models
HST 003 -Preclinical Proof of Concept
In vivo efficacy in goat osteochondral defect
• Untreated defects (16) were characterized by large voids filled with fibrotic
tissue at 12 months
• At 12 months, all hECM-treated defects (16) showed:
• Mature hyaline cartilage
• Mature, vascularized bone
• Integration of regenerated tissue with adjacent tissue
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hECM Treated
Hyaline cartilage regeneration seen in large animal model
Untreated
HST 003 -Phase 1 Protocol
Objective:
• Evaluate the safety of hECM paste delivered arthroscopically during
microfracture procedure
Enrollment Criteria:
• 12 subjects, men and women, who are candidates for microfracture surgery
• Recent focal cartilage defects of 2 - 4 cm2 in size in the knee
• 3 clinical sites: Walter Reed Medical Center, The Steadman Clinic,
Balboa Naval Medical Center
Treatment Regimen:
• Microfracture + ECM HST 003
• Microfracture alone
Endpoints:
• Adverse events, and other safety parameters
• Evaluate presence of cytokines and inflammatory cells in knee fluid
• MRI to quantify cartilage regeneration
• KOOS and IKDC scores to evaluate pain and joint function23
Joint Cartilage
Evaluation of naturally
secreted human
extracellular matrix for
articular cartilage defects
IND submission expected
Q1/2020
HST 003 - Joint Cartilage Competitive Differentiation
Malleable scaffold applied to full-thickness chondral lesions as part of an
arthroscopic microfracture procedure for the treatment of knee disorders
Target Profile:
• Regenerate hyaline cartilage, potentially improving function/mobility
• Provide reduction in inflammation, potentially reducing pain
• Produce no immune response, potentially improving safety/tolerability
• Increase angiogenesis allowing for improved cell proliferation and
differentiation, potentially improving the healing process and time
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Results from planned
Phase 1 clinical trials may
support a high value
pharmaceutical
partnership or exit.
Anticipated Regulatory Path for all Assets
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HST 001 Hair Growth
HST 002 Dermal Filler
HST 003 Joint Cartilage*
2019
Preclinical
Preclinical
Preclinical CMC
2020 2021 2022 2023 2024
Phase 1
Phase 1
Phase 1
Phase 2
Pivotal
Phase 2
Phase 3 BLA
Phase 3 BLA
PMA
*Potential for Regenerative Medicine Advanced Therapy designation
for accelerated approval
Financial
A track record of successful funding combined with prudent financial management provides the necessary foundation for growth
Financial Snapshot
Company has raised $38.4 million to date in equity offerings and
$20 million to date through licensing transactions, with an additional
$10.5 million in future milestones under these licenses.
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Cash, cash equivalents
and marketable
securities: $6.3MM
as of 6/30/19
Strengthened Finance
Team in Q1 2019
Shares Outstanding: 56,950,168
Fully Diluted Shares: 67,798,463
Fully Audited
Financials through
2018 expected in
November 2019
Cash on hand expected
to fund current
operating plan into Q1
2020
Looking Forward
Creating value by advancing innovative therapeutics to address unmet medical needs in the aesthetics and orthopedic markets
Creating Value
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Multiple near-term value
inflection upon anticipated
initiation of multiple clinical
trials in first half of 2020
and data read-outs
starting in the second half
of 2020
Histogen is advancing
innovative
therapeutics to address
unmet medical needs in
the aesthetics and
orthopedic markets
Validated platform
technology – partnered
and marketed cosmetic
products
Developing a broad portfolio of
therapeutic clinical-stage
regenerative medicine
product candidates with
demonstrated mechanisms
of action1
Advancing three
clinical programs in
therapeutic indications
with large commercial
market opportunities
Strong research, product
development, clinical and commercial leadership
experience
Positioning the Company for potential
licensing or
strategic
transactions in 2020
1 Demonstrated in vitro, in preclinical models and/or in human clinical studies
Redefining Regenerative Medicine
2019 BIO Investor Forum
Richard W. PascoeCHAIRMAN AND CEO
October 22-23, 2019