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OUR MISSION: Fulfill an unmet medical need with therapeutic breakthroughs in diseases of abnormal mineralization
December 2020
Legal Disclaimer
This presentation and any statements made orally during this presentation also contain estimates and other statistical data made by independent parties and by us relating to market size 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 data and 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. Neither Inozyme Pharma, Inc. nor its affiliates, advisors or representatives make any representations as to the accuracy or completeness of that data or undertakes to update such data after the date of this presentation.
Forward-Looking Statement Disclaimer
This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements. In addition, the forward-looking statements included in this presentation represent our views as of the date of this presentation. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements in the future, we specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this presentation.
2
Our mission is to fulfill an unmet medical need with therapeutic breakthroughs in diseases of abnormal mineralization.
Mission Statement
3
Our mission is to fulfill an unmet medical need with therapeutic breakthroughs in diseases of abnormal mineralization.
4
Highly experienced team with strong track record➢Deep understanding of rare disease research, clinical, regulatory and commercial strategy➢Upsized IPO in July 2020 raised $128.8 million in gross proceeds
Strong scientific rationale➢Novel biology in diseases with high unmet medical needs➢Genetic basis and biological pathway for disease pathophysiology well understood➢ Predictive animal models with strong POC data➢ Lead candidate (INZ-701) showed a good safety profile and an acceptable therapeutic index in animal studies
Efficient development approach➢ Enzyme replacement therapy is a generally established therapeutic modality➢ Lead candidate with opportunities for multiple rare genetic diseases➢ Regulatory strategy in place to initiate clinical development➢ Clinical development planned in near term with a clear path to generating clinical data
Untapped market opportunity➢Underserved market opportunity with high unmet medical need and limited competition➢ Received Orphan Drug Designation for INZ-701 for the treatment of ENPP1 deficiency in the US and the EU➢Worldwide, exclusive development and commercial rights to INZ-701; IP protected through 2036
CONFIDENTIAL
Key Investment Highlights
Steve BassoSenior Vice President of Finance
Steven JunglesSenior Vice President and Chief Technical Operations Officer
5
Pedro Huertas, M.D., Ph.D.Senior Vice President and Chief Medical Officer
B o a r d o f D i r e c t o r s
Henric BjarkeSenior Vice President and Chief Operating Officer
Axel Bolte Co-founder, President and Chief Executive Officer
Management Team and LeadershipBacked by Investors Committed to Progress in Life Sciences
Yves Sabbagh, Ph.D.Senior Vice President and Chief Scientific Officer
Sarah Bhagat Axel Bolte Reinaldo Diaz Martin Edwards
Rob Hopfner Ed Mathers Lynne Sullivan Doug Treco (Chair)
Kevin Johnson, Ph.D., MBASenior Vice President, Regulatory Affairs
6
INFANCY ADOLESCENCE ADULTHOOD
What do Diseases of Abnormal Mineralization Look Like?ENPP1 Deficiency Affects Patients of All Ages in Devastating Ways
For illustrative purposes only. Individual patient experiences may vary.
7
• When abnormalities or disruptions occur, effects of excess mineralization are seen in other systems, such as the vascular system
• Bone softening, clogged arteries, and blindness are all potentially caused by excess mineralization
• ENPP1 and ABCC6 deficiencies are examples of such mineralization diseases
• In addition to excess mineralization, ENPP1 and ABCC6 deficiencies can also cause tissue growth through neointimal proliferation
Mineralization is a Fundamental Process Within the Human BodyMineralization is How Bones are Calcified, and is Crucial to the Formation of the Skeleton
Abnormalities Have Potentially Fatal Systemic Impact
8
Diversity of Abnormal Mineralization Manifestation Allows Us to Pursue Multiple Therapeutic Opportunities
GENETIC DISEASES NON-GENETIC DISEASES
*Lead Indications
PREVALENCE
ENPP1 deficiency*
ABCC6 deficiency*
Calciphylaxis
Vascular disease with neointimal proliferation
Mineralization & Neointimal Proliferation
11,000 –12,000 (worldwide)
~1,800 (per year in the U.S.)
>67,000 (worldwide)
Indication dependent
Program Exploring Several Potential IndicationsInitial focus on Genetic Diseases followed by Expansion into Non-Genetic Diseases
9
PROGRAM ASSETSTAGE OF DEVELOPMENT NEXT
ANTICIPATED MILESTONEResearch
IND Enabling
Phase 1/2 Phase 2/3
GENETIC DISEASES
ENPP1 DeficiencyINZ-701 (ENPP1-Fc)
File IND* and CTA2H 2020
ABCC6 DeficiencyINZ-701 (ENPP1-Fc)
File CTA2H 2020
NON-GENETIC DISEASES
CalciphylaxisINZ-701 (ENPP1-Fc)
Generate pre-clinical proof of concept
Diseases of Neointimal Proliferation
INZ-701 (ENPP1-Fc)
Generate pre-clinical proof of concept
* Currently on clinical hold pending completion of ongoing GLP toxicology studies.
ABCC6 = Adenosine triphosphate binding cassette transporter protein subfamily C member 6; AMP=adenosine monophosphate; ARHR2 = Autosomal recessive hypophosphatemic rickets type 2; ATP=adenosine triphosphate; ENPP1 = ectonucleotide pyrophosphatase/phosphodiesterase 1; PPi = pyrophosphate
References: 1. Jansen RS, et al. Arterioscler Thromb Vasc Biol. 2014;34(9):1985-1989. 2. Nitschke Y, et al. Am J Hum Genet. 2012;90(1):25-39. 3. Nitschke Y, et al. Exp Mol Med. 2018;50(10):1-12.
How ENPP1 and ABCC6 Work The Biologic Pathway that Regulates Mineralization and Neointimal Proliferation
ATP
ATP
ABCC6 ENPP1 CD73
INTRACELLULAR
EXTRACELLULAR
Maintains Healthy Mineralization
• PPi inhibits growth and formation of hydroxyapatite – Maintains healthy bones and teeth– Inhibits pathological ectopic
mineralization (i.e., mineralization of arteries, organs, and joints)
Maintains Healthy Vessel Wall Thickness
• Adenosine inhibits neointimal proliferation
AdenosinePPi
Tunicaexterna
Arterylumen
Tunica media
Tunica intima
AMP
11
References: 1. Jansen RS, et al. Arterioscler Thromb Vasc Biol. 2014;34(9):1985-1989. 2. Nitschke Y, et al. Am J Hum Genet. 2012;90(1):25-39. 3. Nitschke Y, et al. Exp Mol Med. 2018;50(10):1-12. 4. Orriss IR, et al. Curr Opin Pharmacol. 2016;28:57-68. 5. Boyce AM, et al. Curr Osteoporos Rep. 2020;18(3):232-241.
ENPP1 DeficiencyLow Levels of PPi and Adenosine Lead to Pathological Mineralization and Neointimal Proliferation
References: 1. Jansen RS, et al. Arterioscler Thromb Vasc Biol. 2014;34(9):1985-1989. 2. Nitschke Y, et al. Am J Hum Genet. 2012;90(1):25-39. 3. Nitschke Y, et al. Exp Mol Med. 2018;50(10):1-12. 4. Orriss IR, et al. Curr Opin Pharmacol. 2016;28:57-68. 5. Boyce AM, et al. Curr Osteoporos Rep. 2020;18(3):232-241.
ATP
ATP
ABCC6 ENPP1 CD73
INTRACELLULAR
EXTRACELLULAR
Pathological Mineralization Neointimal Proliferation
Ectopic mineralization(arteries, joints, and organs)
Under-mineralization of bones
Calcification of descendingaorta and arteries
Mineralization ofshoulder joint
Irregularities in the distal femoral metaphyses and rickets
Tunicaexterna
Arterylumen
Tunica media
NeointimalProliferation
Tunicaexterna
Arterylumen
Tunica media
AdenosinePPi
AMP
12
References: 1. Jansen RS, et al. Arterioscler Thromb Vasc Biol. 2014;34(9):1985-1989. 2. Nitschke Y, et al. Am J Hum Genet. 2012;90(1):25-39. 3. Nitschke Y, et al. Exp Mol Med. 2018;50(10):1-12. 4. Orriss IR, et al. Curr Opin Pharmacol. 2016;28:57-68. 5. Boyce AM, et al. Curr Osteoporos Rep. 2020;18(3):232-241.
ABCC6 Deficiency Low Levels of PPi and ENPP1 Lead to Pathological Mineralization and Neointimal Proliferation
ATP
ATP
ABCC6 ENPP1 CD73
INTRACELLULAR
EXTRACELLULAR
AdenosinePPi
AMP
Pathological Mineralization
CT Scan of Calcification Histology of Vascular Calcification Skin Histology of Skin Alterations
13
1Hypophosphatasia Reported in Whyte et al. JCI Insight. 2016;1(9):e859712Nitschke et al., 20183Kauffenstein et al., 20184O'Neill et al, 2010
14
Normalization of PPi is an Objective in Several Disease Conditions
ENPP1 Deficiency / GACI/ARHR22
ABCC6 Deficiency / Pseudoxanthoma Elasticum (PXE)3
HPP1
No
rmal
Pla
sma
PPi (
nM
)
Chronic Kidney Disease4
Inozyme Therapeutic Objective:Normalization of PPi-Levels
Strensiq
Extracellular domain
Fcfragment
INZ-701
INZ-701: Inozyme’s Drug CandidateDrug Candidate Designed to Replace Lost Enzymatic Function of ENPP1 and ABCC6
15
• Protein: Recombinant human ENPP1 (Ectonucleotide pyrophosphatase/phosphodiesterase 1)
• Construct: Recombinant Fc fusion protein with soluble extracellular domain of ENPP1
• Dosing: s.c. / Potentially as long as weekly
• Enzymatic Properties: High catalytic efficiency (Kcat/Km)
INTR
AC
ELLU
LAR
EXTRACELLULAR
ENPP1
I NZ-701
+
16
GENETIC DISEASES NON-GENETIC DISEASES
*Lead Indications
PREVALENCE
Calciphylaxis
Vascular disease with neointimal proliferation
~1,800 (per year in U.S.)
>67,000 (worldwide)
Indication dependent
11,000 –12,000 (worldwide)ENPP1 deficiency*
ABCC6 deficiency*
Mineralization & Neointimal Proliferation
Biochemical Consequences of Calcification Results of Tx Restoring Growth Increased Survival
100% survival of treated asj/asj-mice; normalized PPi levels
Dosing: 10mg/kg ENPP1-Fc, QD, s.c.
0
50
100
0 20 40 60
Per
cen
t Su
rviv
al
Days Elapsed
Treated
Untreated
0
5
10
15
WT Treated Untreated
uM
PP
i
Plasma PPi (uM)
5
10
15
20
10 20 30 40 50
Wei
ght
(g)
Age (days)
0
20
40
60
80
100
WT+ Veh
HOM,2 wkold
Veh ENPP11250
ENPP12500
ENPP15000
Ao
rta
Cal
ciu
m C
on
ten
t(n
mo
l/m
g ti
ssu
e)
Aorta Calcification
HOM
80%
**95%
**
17
Sources: 1, 3, 4: Albright RA, et al., 2015; 2: Khan T, Dis Model Mech, 2018 Oct 8; 11 (10)
WT
Treated
Untreated
Treatment with ENPP1-Fc prevented early mortality of asj mice
ENPP1-Fc Replacement Prevented Vascular Calcification and Mortality in a Mouse Model
Treatment with ENPP1-Fc normalized plasma PPi levels in asj mice
Reduced arterial calcification Restored weight
1 2 3 4
INZ-701 Restored PPi-Levels in a Predictive Murine Model
18
WT, V
ehic
le
asj,
Vehic
le
asj,
0.2m
g/kg
asj,
1mg/k
g
asj,
5 m
g/kg
0
500
1000
1500
ENPP1 Activity
EN
PP
1 a
cti
vit
y (
mO
D/m
in)
WT, V
ehic
le
asj,
Vehic
le
asj,
0.2m
g/kg
asj,
1mg/k
g
asj,
5 m
g/kg
-1
0
1
2
3
4
5
PPi
Pla
sm
a P
Pi (
M)
Asj-Mouse Model:
• Failure to thrive and gain weight
• Extensive vascular calcification
• Premature mortality• Mimics human disease
Therapy start at age of 2 weeks (D1)and end at 10 weeks (D56)
Sources: Internal, Unpublished Data
INZ-701 Prevented Calcification in the Kidney, Spleen, Lung and Liverof asj Mice
Mice on the acceleration diet, starting at week two, with both INZ-701 and vehicle control every other day for eight weeks.
WT, V
ehic
le
asj m
ice,
veh
icle
asj m
ice,
0.2
mg/k
g
asj m
ice,
1m
g/kg
asj m
ice,
5m
g/kg
0
50
100
150
200
250
Kidney
Calc
ium
co
nte
nt
(nm
ol/m
g o
f ti
ssu
e)
******
****
(P=0.006)
(P<0.0001)
(P<0.0001)
WT, V
ehic
le
asj m
ice,
veh
icle
asj m
ice,
0.2
mg/k
g
asj m
ice,
1m
g/kg
asj m
ice,
5m
g/kg
0
10
20
30
40
50
Spleen
Calc
ium
co
nte
nt
(nm
ol/m
g o
f ti
ssu
e)
********
ns (P=0.3472)
(P<0.0001)
(P<0.0001)
WT, v
ehic
le
asj m
ice,
veh
icle
asj m
ice,
0.2
mg/k
g
asj m
ice,
1m
g/kg
asj m
ice,
5m
g/kg
0
1
2
3
4
5
Lung
Calc
ium
co
nte
nt
(nm
ol/m
g o
f ti
ssu
e)
******
ns (P=0.1966)
(P=0.0003)
(P=0.0001)
WT, v
ehic
le
asj m
ice,
veh
icle
asj m
ice,
0.2
mg/k
g
asj m
ice,
1m
g/kg
asj m
ice,
5m
g/kg
0
1
2
3
Liver
Calc
ium
co
nte
nt
(nm
ol/m
g o
f ti
ssu
e)
*******
** (P=0.0032)
(P=0.0005)
(P<0.0001)
20Sources: Internal, Unpublished Data
INZ-701 Prevented Bone Loss and Restored Growth in asj Mice
21
Corrected bone defects1
Rescued growth defect2
Restores Trabecular Number
RestoresCorticalThickness
WT Untreated Treated
ENPP1 Deficient
Sources: Internal, Unpublished Data
Average Trabecular Number (1/mm)
Average Cortical Thickness (mm)
2.601
0.174
1.407
0.113
2.213
0.148
0 20 40 60
5
10
15
20
25
Days
Bo
dy
We
igh
t (g
ram
)
WT
Untreated
Treated
ENPP1Deficient
INZ-701 Prevented Neointimal Proliferation in ENPP1-DeficientMurine Model
22
Histology1
Intimal Area2
ttw/tt
w v
ehic
le
ttw/tt
w E
NPP1
0.0
0.1
0.2
0.3
0.4
0.5
0.6
0.7 ***
inti
mal
are
a [
mm
2]
Untreated Treated
ENPP1 Deficient
p<0.001
Histological Analysis of Preventive Treatment in Mice Ligated for 14 Days
Sources: Internal, Unpublished Data
23
GENETIC DISEASES NON-GENETIC DISEASES
*Lead Indications
PREVALENCE
Calciphylaxis
Vascular disease with neointimal proliferation
~1,800 (per year in U.S.)
>67,000 (worldwide)
Indication dependent
11,000 –12,000 (worldwide)ENPP1 deficiency*
ABCC6 deficiency*
Mineralization & Neointimal Proliferation
INZ-701 normalized PPi and reduced Tissue Calcification in Abcc6 -/- miceSupports Clinical Development of INZ-701 in PXE
24
• Dose: 2 and 10mg/kg, q.o.d.• Duration: ~4wk of age to ~6 wk of age • All animals are given GK1.5• All animals on normal diet
WT*
Veh
icle
2mg/k
g
10m
g/kg
0
100
200
300
Ca
(
g/g
ram
tis
su
e)
Vibrissae Calcification
Abcc6-/-
********
• Dose: 2 and 10mg/kg, q.o.d.• Duration: ~4wk of age to ~12 wk of age • All animals are given GK1.5• All animals on normal diet
WT*: age-matched WT mice
WT
vehic
le
2mg/k
g
10m
g/kg
0
1
2
3
4
5
Plasma PPi
PP
i (
M)
Abcc6-/-
Sources: Internal, Unpublished Data**** P ≤ 0.0001
25
GENETIC DISEASES NON-GENETIC DISEASES
*Lead Indications
PREVALENCE
Calciphylaxis
Vascular disease with neointimal proliferation
~1,800 (per year in U.S.)
>67,000 (worldwide)
Indication dependent
11,000 –12,000 (worldwide)ENPP1 deficiency*
ABCC6 deficiency*
Mineralization & Neointimal Proliferation
26
Histology1
Intimal Area2
Histological Analysis of Preventive Treatment in Mice Ligated for 14 Days
WT vehicle WT ENPP10.0
0.1
0.2
0.3 ***
inti
mal
are
a [
mm
2]
Future Expansion Possibility in Non-Genetic Diseases INZ-701 Treatment Prevented Neointimal Proliferation in Normal Mice
Sources: Internal, Unpublished Data*** P ≤ 0.001
27
Our Approach to Establish Preclinical Proof of Concept for INZ-701 for Multiple Indications
GENETIC DISEASES NON-GENETIC DISEASES
*Lead Indications
PREVALENCE
ENPP1 deficiency*
ABCC6 deficiency*
Calciphylaxis
Vascular disease with neointimal proliferation
Mineralization & Neointimal Proliferation
11,000 –12,000 (worldwide)
~1,800 (per year in U.S.)
>67,000 (worldwide)
Indication dependent
ENPP1 Deficiency: A Disease With High Morbidity and Mortality
29
Calcification Skeletal Defects
Generalized Arterial Calcification of Infancy (GACI) Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2)Historical Definition
ENPP1 DeficiencyNew Definition
0 – 3 Years 18+ Years3 – 18 Years
Organ Calcification & Neointimal Proliferation
Organ and arterial calcification
Obstructive neointimal proliferation
ENPP1 Deficiency Natural History Study Informs Clinical Development
30
Calcification and Organ Dysfunction Occur Sequentially and Progressively
in ENPP1 Deficiency
3Calcification, Skeletal Abnormalities, and Rickets
Occur Simultaneously From Birthin ENPP1 Deficiency
2Natural History Study Includes Data From 2 Sites:
NIH and Universität Münster (n=127)
1
0 5 3025201510
Age (Year)
0 5 3025201510
Age (Year)
Tim
e to
Eve
nt
Tim
e to
Eve
nt
Tim
e to
Eve
nt
• 25% quartile estimate: 0.1 years (95% CI: 0.04 to 0.17)• Median survival estimate: 14.9 years (95% CI: 0.41 to NR)
Over 40% mortality in first 12 months of life with a diagnosis of ENPP1 Deficiency
Postnatal Rickets
P s e u d o x a n t h o m a e l a s t i c u m ( P X E )
31
ABCC6 Deficiency (PXE) is Also a Serious Disease Involving Pathological Mineralization and Neointimal ProliferationFocus on Patients with Severe Manifestation Leading to High Unmet Medical Need
Pseudoxanthoma elasticum (PXE)Historical Definition
ABCC6 DeficiencyNew Definition
Onset in 20s-30s | Progressively Effects Adult Population
Skin Cardiovascular
z z
Retina Calcification
Two Planned First-in-Human Phase 1/2 Trials (In Patients)
32
ENPP1 Deficiency
ABCC6 Deficiency
Post Regulatory submissions (US ENPP1 Deficiency*)
Open label
Dose escalation
Age >18 years
Sample size n=9
Safety and tolerability
PK/PD
Plasma PPi levels
Biochemical and physiological parameters
7-week duration
Trial1
Trial2
* Currently on clinical hold pending completion of ongoing GLP toxicology studies.
Endpoint Measurement
PPi Blood Biochemistry
Calcification High resolution radiography
Survival Alive After 6 Months
Illustrative Goals of our Planned Trials
Endpoint Measurement
PPi Blood Biochemistry
BMDHigh resolution radiography
Rickets, Growth, Organ Function
MRI, RGI-C, RSS, Dexa
Endpoint Measurement
PPi Blood Biochemistry
Bone Pain Pain Scores
Osteomalacia MRI, Bone biopsies
ENPP1 Deficiency: Clinical Strategy for INZ-701 is to Link Restoration of Plasma PPi to Measures of Physiological and Clinical Efficacy
0 – 3 Years 18+ Years3 – 18 Years
33
18+ Years
ABCC6 Deficiency (PXE)
Endpoint Measurement
PPi Blood Biochemistry
CalcificationHigh resolution radiography
Cardiac Function/Pain
Doppler, EGG, CT scan, Pain scores
ABCC6 Deficiency: Clinical Strategy for INZ-701 is to Link Restoration of Plasma PPi to Measures of Physiological and Clinical Efficacy
Illustrative Goals of our Planned Trial
34
Regulatory Status and Strategy for Phase 1 / 2 Trials in ENPP1 Deficiency
Status
➢ Orphan drug designation obtained in US and EU➢ IND filed with the FDA July 2020➢ Pre CTA meetings held with European Authorities➢ CTA filed with MHRA October 2020➢ Scientific advice meetings held with EMA
Next Steps➢ Address FDA clinical hold with additional data and File CTA➢ Engage with regulatory authorities at End of Phase 1/2 meeting to discuss registration endpoints
35
2018 2019 2020
June 2018
Orphan designation received
April 2019Pre-IND Meeting, face-to-face
Mar 2020
2nd Pre-IND Meeting
2018 2019 2020
July 2018
Orphan designation received
July 2019Scientific Advice Meeting, face-to-face
May 2020
Pre CTA meetings
July 2020
Filed IND
Robust CMC Process Designed for Stability and Scalability
Formulation DevGMP Mfg
GMP Mfg
GMP Mfg
Virus Removal Validation
Ongoing Stability
QC Assay Dev
36
✓ Recombinant Fc-fusion protein with the soluble extracellular domain of ENPP1 produced in CHO cells, titers ranging from 800 to 900 mg/L
✓ Fully tested master cell bank stored in multiple locations for redundancy
✓ Large scale production confirmed (GMP run at 1000L scale)
✓ Protein purified using a well-defined process suitable for cGMP manufacturing, Protein A purification with multiple virus removal and virus inactivation steps
✓ Final product concentrated to 50 mg/mL for SQ injections (Once weekly)
✓ Consistent process performance through scale-up—process easily scalable, supply chain established
38
Abnormal Mineralization is the Underlying Cause of Several Debilitating Diseases Allowing Us to Pursue Multiple Therapeutic Opportunities
P R E VA L E N C E O F L E A D I N D I C AT I O N S
ENPP1 Deficiency
ABCC6 Deficiency
Mineralization & Neointimal Proliferation
11,000 –12,000 (worldwide)
>67,000 (worldwide)
39
SCREENING SURVEY COMPLETES:
# of Physicians US Canada UK France Germany Italy Spain
Geneticists 42 5 10 7 4 19 11
Endocrinologists 160 25 63 54 35 97 72
Ped Endos 33 3 10 9 6 13 16
Ped Cardiologists 24 -- 1 4 7 3 5
Orthopedists 174 8 56 33 78 33 24
Neonatologists 75 8 34 22 35 18 31
TOTAL 508 49 174 129 165 183 159
Source: Global online survey, conducted in local language, March 22 to April 15, 2019.
1,367 Target Physicians in 7 Key Markets Responded to Survey
37% 26%Confirmed Patients (N=506 Physicians)
Managing Patients(N=355 Physicians)
2,976 Confirmed Patients 1,682 Managed Patients
1,001 Patient Profiles Collected
Over 600 Patients Alive
International Physician Survey Identified Significant Numbers of Treatable ENPP1 Deficiency Patients
1,367 Physician Responses
40
Advocating
Presenting
Discussing
Educating
Ongoing Physician Identification and Educational ActivitiesBuilding KOL Relationships and Educating the Scientific and Patient Community
Financial Overview and Upcoming Anticipated Milestones
42
ENPP1 Deficiency ABCC6 Deficiency New Indications and Pipeline
Clearance of IND and CTAsEarly 2021
Initiation of Phase 1/2 clinical trialH1 2021
Initiation of prospective natural history studyH1 2021
Preliminary safety and biomarker data from Phase 1/2 clinical trialH2 2021
Neointimal proliferationpre-clinical POC
Calciphylaxispre-clinical POC
Gene Therapy Programselect development candidate
FundingHistory
Pre IPO • $116 M in Private Financings : $49 M January 2017, $67 M March 2019
IPO • July 2020 – Net proceeds of approximately $116.5 M
Cash Position • Cash of $171.7 million as of September 30, 2020
Clearance of CTAsEarly 2021
Initiation of Phase 1/2 clinical trialH1 2021
Preliminary safety and biomarker data from Phase 1/2 clinical trialH2 2021
43
Highly experienced team with strong track record➢Deep understanding of rare disease research, clinical, regulatory and commercial strategy➢Upsized IPO in July 2020 raised $128.8 million in gross proceeds
Strong scientific rationale➢Novel biology in diseases with high unmet medical needs➢Genetic basis and biological pathway for disease pathophysiology well understood➢ Predictive animal models with strong POC data➢ Lead candidate (INZ-701) showed a good safety profile and an acceptable therapeutic index in animal studies
Efficient development approach➢ Enzyme replacement therapy is a generally established therapeutic modality➢ Lead candidate with opportunities for multiple rare genetic diseases➢ Regulatory strategy in place to initiate clinical development➢ Clinical development planned in near term with a clear path to generating clinical data
Untapped market opportunity➢Underserved market opportunity with high unmet medical need and limited competition➢ Received Orphan Drug Designation for INZ-701 for the treatment of ENPP1 deficiency in the US and the EU➢Worldwide, exclusive development and commercial rights to INZ-701; IP protected through 2036
Key Investment Highlights