Investor Presentation - H.C. Wainwright Global Life Sciences Conference 2021 March 10, 2021
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Investor Presentation
H.C. Wainwright Global Life Sciences
Conference 2021
March 10, 2021
…at Hansa Biopharma we envision a world where all patients
with rare immunologic diseases can lead long and healthy lives...
Forward-looking statement
This presentation may contain certain forward-looking statements and forecasts based on our current expectations and beliefs
regarding future events and are subject to significant uncertainties and risks since they relate to events and depend on
circumstances that will occur in the future. Some of these forward-looking statements, by their nature, could have an impact on Hansa
Biopharma’s business, financial condition and results of operations [or that of its parent, affiliate, or subsidiary companies]. Terms
such as “anticipates”, “assumes”, “believes”, “can”, “could”, “estimates”, “expects”, “forecasts”, “intends”, “may”, “might”, “plans”,
“should”, “projects”, “will”, “would” or, in each case, their negative, or other variations or comparable terminology are used to identify
forward-looking statements. There are a number of factors that could cause actual results and developments to differ materially from
those projected, whether expressly or impliedly, in a forward-looking statement or affect the extent to which a particular projection is
realized. Such factors may include, but are not limited to, changes in implementation of Hansa Biopharma’s strategy and its ability to
further grow; risks and uncertainties associated with the development and/or approval of Hansa Biopharma’s product candidates;
ongoing clinical trials and expected trial results; the ability to commercialize imlifidase if approved; changes in legal or regulatory
frameworks, requirements, or standards; technology changes and new products in Hansa Biopharma’s potential market and industry;
the ability to develop new products and enhance existing products; the impact of competition, changes in general economy and
industry conditions and legislative, regulatory and political factors.
The factors set forth above are not exhaustive and additional factors could adversely affect our business and financial performance.
We operate in a very competitive and rapidly changing environment, and it is not possible to predict all factors, nor can we assess
the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ
materially from those contained in any forward-looking statements. Given these risks and uncertainties, investors should not place
undue reliance on forward-looking statements as a prediction of actual results.
Hansa Biopharma expressly disclaims any obligation to update or revise any forward-looking statements to reflect changes in
underlying assumptions or factors, new information, future events or otherwise, and disclaims any express or implied representations
or warranties that may arise from any forward-looking statements. You should not rely upon these forward-looking statements after the
date of this presentation.
2
Hansa Biopharma today
Successful track record... A validated Idefirix® our first Broad pipeline in
Strong momentum... technology approved drug in transplantation and
Europe* autoimmunity
Promising future...
VALIDATION ACROSS EUROPE KIDNEY PROGRAMS IN CLINICAL
THREE AREAS TRANSPLANTS DEVELOPMENT
Approval in kidney For highly sensitized US Kidney transplants
transplantations patients in Europe Anti-GBM
PoC in autoimmune diseases Guillain-Barré syndrome (GBS)
Partnership to explore gene Antibody mediated kidney
therapy transplant rejection (AMR)
Established a With recent capital Created shareholder
high-performance injection Hansa is value and diversified
organization financed into 2023 our ownership base
NEW COMPETENCIES MID-TERM FINANCIAL MCAP USD ~1bn
ADDED PRIORITIES
~100 employees (3x in 5 years) Fund a broad exploitation of our 10x vs cost of development (13Y) Patient**
technology platform while
Highly qualified team with 20 securing a successful EU launch 17,000 shareholders This is a break-through for
years on average in life science the patients who need but
Foreign ownership make up ~50%
SEK ~1.4bn/USD ~160m in cash can’t access kidney
Purpose driven culture end of December 2020 through leading international life
science specialist funds transplantation today
*Idefirix approved in EEA under conditional *Actual patient has given consent to provide images
3 approval for kidney transplantation
Imlifidase – a novel approach to eliminate pathogenic IgG
Origins from a bacteria Imlifidase, a unique antibody-cleaving Imlifidase inactivates IgG
Streptococcus pyogenes enzyme to eliminate pathogenic IgG in 2-6 hours from infusion
• Species of Gram-positive, • Interacts with Fc-part of IgG with extremely high specificity • Rapid onset of action that
spherical bacteria in the genus inactivates IgG below detectable
Streptococcus • Cleaves IgG at the hinge region, generating one F(ab’)2 level in 2-6 hours from infusion
fragment and one homo-dimeric Fc-fragment
• Usually known from causing a • IgG antibody-free window for
strep throat infection approximately one week
IgG in human serum
10
F(ab’)2
8
imlifidase
[IgG] (mg/mL)
6
n=10 patients
IgG
4
2
Fc 0
0.5 h
1h
2h
4h
6h
8h
1d
2d
3d
7d
14 d
21 d
28 d
64 d
0
4
Gene therapy pre-treatment
Potential indication universe (partnership opportunity)
Transplantation and post transplantation
(Own commercial infrastructure in EU/US) … …
…
Limb- Transplantation and
Girdle … post transplantation
… Lung Heart
(LGMD)
Other areas
Lung Bone- Duchenne
First generation antibody Kidney (DMD)
cleaving enzyme technology AMR marrow
Relapsing
Obtained EU conditional approval* IgG-related
Heart Kidney
Clinical program
AMR AMR First genera+on New enzymes for autoimmune
an+body-cleaving repeat dosing diseases
Research/Preclinical program enzyme technology “NiceR”
Opportunities
Oncology (EnzE)
Partnership (Sarepta Therapeutics Inc.)
Guillain
Acute autoimmune diseases Anti-GBM Barre
(Own commercial infrastructure in EU/US) syndrome
Gene therapy
… … …
… …
* US: Study protocol submitted June 2020, study expected to be initiated H1 2021. The
new clinical study could support BLA submission by 2023
5
Leveraging our technology platform
Developing new therapies targeting rare diseases with unmet medical need
across a range of indications
Growth engine Value chain Commercialization
Leveraging our We are controlling Build-up of Indications Multiple
proprietary antibody the full value chain franchises and therapies income streams
cleaving enzyme technology
Transplantation Revenue / sales
Own
commercial
infrastructure
Autoimmune diseases Upfront payments
Drug Drug Supply
Distribution
discovery development Operations
Gene therapy Milestone payments
Partnership
strategy
Oncology Royalties
Evolution into a fully integrated biopharmaceutical company
6
Idefirix® (Imlifidase) has received conditional approval in the European Union
Low Higher
complexity complexity
transplants transplants
~70% of patients1,2 15-20% of patients1,2 10-15% of patients1,2
Non or less sensitized Moderately sensitized Highly sensitized
(cPRA < 20%) (20% > cPRA < 80%) (cPRA > 80%)
Highly sensitized Highly sensitized
patients that are patients unlikely
likely to be to be transplanted
transplanted with under available
a compatible KAS, including
donor prioritization
programs
Idefirix® is indicated for Potential
desensitization treatment of highly sensitized adult kidney patients
transplant patients with positive crossmatch against an available
deceased donor.
The use of Idefirix® should be reserved for patients unlikely to be
transplanted under the available kidney allocation system
including prioritization programs for highly sensitized patients
Actual patient have
7 given consent to 1 EDQM. (2020). International figures on donation and Transplantation 2019
2 SRTR Database and individual assessments of allocation systems
provide images
European kidney transplantation landscape
Approximately 22,000 annual kidney transplants in Europe of which approximately Deceased donor transplants
15,000 transplants in EU5 (2019). ~80% of transplants are from deceased donors1 Living donor transplants
Patients Total kidney transplantations
UK 2 627 1 022 3 649
France 3 133 510 3 643
EU5
Spain 3 088 335 3 423
Italy 1 799 340 2 139
Germany 1 612 520 2 132
Netherlands 450 501 951
Czech Republic 461 49 510
Sweden 329 147 476
Austria 309 77 386
Switzerland 232 108 340
Finland 268 25 293
Denmark 189 87 276
Norway 236 22 258
Rest of Europe* 3 142 481 3 623
1 Transplant
data from 2019.
8 *Belgium, Croatia, Cyprus, Greece, Hungary, Iceland, Ireland, Lithuania, Poland, Portugal, Romania, Slovakia, Slovenia
Source: Global Observatory on Donation & Transplantation, 2019
Early launch in centres of excellence
● Leading transplant clinics
First launch wave defined
1. Launch Idefirix® with kidney transplant specialists
who have experience in desensitization
2. Create positive momentum with Idefirix as the new
Gold Standard in desensitization protocols
3. Prepare post approval study to confirm filing data
Leading transplantation centres perform the
majority of all transplantations in EU
France UK Spain
5 4 4
37 20 33
Italy Germany Other EU countries*
4 4 7
● Hospitals with experience
in desensitization &
transplantation of highly
sensitized patients
36 33 60 ● Other transplantation clinics
9 Idefirix approved in EU under conditional approval *Other EU countries incl. Sweden, Denmark,
Norway, Austria, Switzerland, Netherlands,
Belgium, Poland, Czech Rep. and Portugal
Broad pipeline in transplantation and auto-immune diseases
Potentially
Candidate / Research/ Pivotal/ Marketing Next Anticipated
Project Indication Preclinical Phase 1 Phase 2 Phase 3 Authorization Marketed Milestone
EU: Kidney transplantation in highly sensitized
*) Commercial launch Q1’21
patients 1,2
US: Kidney transplantation in highly sensitized
**) First patient dosed H1’21
patients 1,2
Agreement with regulators on
Imlifidase Anti-GBM antibody disease 3 a path forward toward
BLA/MAA
Antibody mediated kidney transplant rejection Complete enrolment of 30
(AMR) patients H2’21
Complete enrolment of 30
Guillain-Barré syndrome (GBS)
patients H2’21
Pre-treatment ahead of gene therapy in Limb-Girdle
Preclinical phase
(LGMD) & Duchenne (DMD) (Partnered with Sarepta)
Recurring treatment in autoimmune Initiation of toxicology
NiceR
disease, transplantation and oncology studies in H1 2021
EnzE Cancer immunotherapy Research phase
Completed Ongoing
1 Results from the Phase 1 study have been published, Winstedt el al. (2015) PLOS ONE 10(7)
2 Lorant et al American Journal of Transplantation and 03+04 studies (Jordan et al New England Journal of Medicine)
3 Investigator-initiated study by Mårten Segelmark, Professor at the universities in Linköping and Lund
*) The EU Commission has granted conditional approval for imlifidase in highly sensitized kidney transplant patients. A post-approval study will commence in parallel with the launch
**) FDA: Proposed study protocol submitted June 2020. Discussions are currently ongoing with the FDA. Once the final protocol has been agreed upon, Hansa Biopharma will
10 proceed to set up centers in the US and start to enroll patients. The COVID-19 pandemic may impact the timelinePositive high-level data read-out in
Anti-GBM study. Recruitment in AMR
& GBS studies reinitiated in Q4’20
Ongoing Phase 2 programs
Enrollment status
March 2, 2021
Anti-GBM (investigator-initiated study)
• Phase 2 study completed with positive high-level
data read-out from 15 patients
• Next step is to engage with regulators and agree on
a path forward toward BLA/MAA
Antibody Mediated Rejection
• Recruitment reinitiated in Q4 2020
• 7/30 patients enrolled in AMR study
• Enrollment expected to be completed H2 2021
Guillain-Barré Syndrome
• Recruitment reinitiated in Q4 2020
• 6/30 patients enrolled in GBS study
Patients enrolled • Enrollment expected to be completed in H2 2021
Patients left
11Positive high-level data from Phase 2 study anti-GBM disease marks an
important milestone for expansion of imlifidase outside transplantation
2/3 of patients achieving dialysis independence six months after treatment
High-level data read out
1
5 1
5 4
5 • Study concludes that imlifidase
leads to rapid clearance of anti-
GBM antibodies
Oliguric Dialysis but Not dialysis
patients
4 not oliguric
4 but eGFR 15
12“NiceR” – new set of enzymes for
repeat dosing; potentially enabling
treatment of relapsing diseases
IgG-cleaving enzyme with lower immunogenicity NiceR can potentially inactivate flares
• Potential application for a broad array of indications, including Illustrative
reoccurring AMR, relapsing autoimmune diseases and flare 1 flare 2 flare 3 flare (n)
oncology
• The first selected promising new drug candidate from the
Level of Pathogenic IgG
NiceR program is an IgG-cleaving enzyme (cysteine peptidase)
with characteristics based on a homolog to imlifidase, but with
lowered immunogenicity.
• Initiation of IND-enabling tox studies in the first half of 2021
Time
Flares NiceR
13Neutralizing antibodies (Nabs) are immunological barriers in gene therapy
Between approximately 5% and 70%1,2 of patients considered for gene therapy treatment carry
neutralizing anti-AAV antibodies forming a barrier for treatment eligibility
Our hypothesis is that imlifidase has the potential to eliminate neutralizing antibodies as a
pre-treatment, prior to the introduction of gene therapy
1 Antibodies prevent effective transfer 2 Imlifidase is a unique IgG antibody- 3 The idea is to eliminate the
of healthy gene sequence and can be cleaving enzyme that cleaves IgG at neutralizing antibodies as a pre-
a safety concern the hinge region with extremely high treatment to enable gene therapy
specificity
Cell
Cell
Nabs/IgG F(ab’)2
imlifidase
Fc
1 Boutin (2010)
14 2 Kruzik (2019)LETTERS 50 NATURE MEDICINE
+ 30 min
Anti-AAV
2
37
a
0
Imlifidase (IdeS) was highlighted in Nature Medicine1 with encouraging outcome
AAV-LK03-GAA 25 AAV-LK03-hFVIII
IdeS Fc
eS
(2 × 1012 vg kg–1) 20–1) (2 × 1012 vg kg–1)
-C
S
(1 mg kg Animals were killed
PB
eS
Id
Id
5 min 24 h
Day 0 Day 210 Day 211 Day 239
d e f
Results from
b preclinical
kDastudies
B272
c with imlifidase
d (ideS) in gene
e therapy demonstrate
4 imlifidase
12
P < 0.0001 P = 0.001
P = 0.001
Anti-AAV8 NAb titer (1:x)
40 8
Anti-AAV8 IgG (µg ml–1)
100,000 100,000
Anti-AAV-LK03 IgG (µg ml–1)
Anti-AAV-LK03 IgG (µg ml–1)
250 P < 0.0001
Anti-AAV-LK03 NAb titer (1:x)
Anti-AAV-LK03 NAb titer (1:x)
as a potential solution toB245overcome pre-existing
B244
150
B233
B243 10,000
antibodies
P = 0.423
to AAV-based
10,000
P = 0.0021
IgG
scIgG
3 gene therapy 9 P = 0.985
P > 0.9
30 6 B249
100 B253 1,000 1,000 F(ab’)2
2 6
20 37 Imlifidase
4 tested in a B264
100 Imlifidase tested
100in NHPIdeS Imlifidase tested in human
hemophilia mouse model ahead
10 of AAV vector
10 infusion plasma
1 samples (GT patients)
3
10 2 Fc
25
• Imlifidase decreased anti-AAV • 1 Pre-treatment with imlifidase
1 in anti- • Imlifidase reduced anti-AAV anti-
IdeS
0 antibodies enabled efficient + 30 min0.1 AAV positive+ nonhuman
andtreatment
0 Before 24 h primates 0
body levels from human plasma 0
0.1 + – + IVIg + –
gene transfer (NHP)
210 ahead of AAV vector 210infusion samples in vitro, incl. plasma from
h
211 211
h
h
h
D 2h
h
h
D 2h
1
0
1
0
– + + IdeS – +
21
21
21
21
+2
+1
+1
+6
+2
+6
+1
+1
ay was safe
Days and resulted in enhanced prospective gene therapy trial
ay
ay
ay
Days NATURE MEDICINE
D
D
Days g Days h liver transduction and hFVIII plasma i 1,500 participants P = 0.0002
80
levels IVIg/IdeS
107 a P = 0.0003 P = 0.0005 b
f g IVIg/IdeS h PBS/IdeS 1,000 P = 0.967 1,000
PBS/IdeS IVIg/PBS
Bleeding time (s)
GLuc activity (RLUs)
150 106 150 IVIg/PBS B233 60 2,500
P = 0.002
1,000
hFIX (µg ml–1)
B272
(ng ml–1)
P = 0.036 100 100
hFVIII in plasma (ng ml–1)
B243
Anti-AAV8 IgG (µg ml–1)
B244
Anti-IdeS IgG (µg ml–1)
AUC hFVIII in plasma
5
10
B245 B249 40 2,000
10 10
100 100 B253
104 500
hFVIII in =plasma
B264 1,500
20 1 1
01
01
01
06
02
00
00
00
00
.0
0.
0.
103 1,000
0
0.
0.
<
<
50 50
<
=
P
P
P
P
P
0.1 0.1
0 0
2 500
10 Not
+ – + IVIg Not
BQL0 7 140 28 BQL 0 7 14 determined–
0 0
PBS + IdeS + IdeSIdeS
PBS determined
210 220 230 240 250Days post-AAV8 210 220
injection 230 240 250 Days post-AAV8 injection Healthy Patients with
PBS IdeS
donors Crigler–Najjar syndrome
Days Days
c
Fig. 1 | IdeS degrades anti-AAV antibodies and allows for successful liver transduction in mice passively
kDa immunized
–
HD1
+ –with
HD2
+ IVIg.
–
HD3
+ a, –Western
HD4
+ +blot
NHP1
– – analys
NHP2
+
NH
–
Fig. 3 | IdeS treatment allows for AAV-LK03 vector readministration in NHPs. a, Experimental protocol. Eight African green monkeys (C. sabaeus) received
incubated for 5!min and 24!h with commercial (IdeS-C) or laboratory-made (IdeS) endopeptidase, or with 250
PBS. The predicted molecular weight of intac
2!×!1012!vg!kg–1 of AAV-LK03-GAA on day 0. On day 210, animals were given one dose of 1!mg!kg–1 of IdeS (IdeS, n!=!5) or vehicle control (PBS, n!=!3), 150
followed by infusion of 2!×!10 1 !vg!kg
scIgG, F(ab′) and Fc fragments is shown. One representative experiment, out of two independent experiments,
2 on day 211. b,c, Anti-AAV-LK03 IgG levels as a function of time in animals treated with either PBS is shown. b, Anti-AAV8 IgG concentrat
Nature of AAV-LK03-hFVIII
12 –1
Medicine https://doi.org/10.1038/s41591-020-0911-7
(b) or IdeS (c). d,e, Anti-LK03 Leborgne IVIg
neutralizing measured
antibody titers after a 24-h incubation with PBS, IdeS-C or IdeS (n!=!1 per condition tested in duplicate; one
100
before (day 210) and after (day 211) treatment with PBS (d) or IdeS (e). f,g, hFVIII antigen levels representative experiment, out of two indep
15 et al. Nat Med (2020)
in plasma as a function of time in animals experiments, is shown).
treated with either c, Protocol
with PBS outline.
(f) or IdeS C57BL/6
(g). BQL, (n!=!6 micelimit.
below quantifiable per group, one representative
h, Aggregate experiment,
hFVIII expression was
75
out of two independent experiments, is shoExclusive agreement with Sarepta Therapeutics to develop and promote
imlifidase as pre-treatment ahead of gene therapy in select indications
A unique opportunity to combine efforts... Hansa’s key competences Sarepta’s key competences
+
• Leader in immunomodulatory enzyme • Market leader within gene therapy
…and to use the unique features of imlifidase to
technology for rare IgG mediated targeted at muscular dystrophies
potentially enable gene therapy treatment in diseases
patients who today aren’t eligible for these • Strong pre-clinical and clinical gene
• Strong experience in antibody cleaving therapy portfolio
breakthrough therapies due to pre-existing
and desensitization
neutralizing antibodies in two indications with a • Scientific approach and knowledge
very high unmet medical need • Broad enzyme technology that can be within gene therapy
used in a variety of indications
• Experience with challenges of Nab-
positive patients
Structure of the partnership
Sarepta will be responsible for conducting
Antibody cleaving
• Pre-clinical/clinical studies with imlifidase enzyme technology Preclinical Development Clinical Development Regulatory Approvals Commercialization
• Regulatory approvals
• Promotion of imlifidase as a pre-treatment to
Sarepta’s gene therapies following potential
approval
Hansa will supply product, support with know-how
and involve in the regulatory approval process
Hansa’s financial participation Upfront payment Milestones Royalties & Sales
Potential total deal value for Hansa amounts to Hansa to receives a USD 10 million Hansa is eligible for a total of Hansa to receive high single-digit to mid-teens
upfront payment from Sarepta for up to USD 397.5 million in royalties on Sarepta’s gene therapy sales
up to USD ~400m plus royalties and incremental accessing Hansa’s unique IgG antibody- development, regulatory and enabled with imlifidase treatment in Nabs
imlifidase sales cleaving enzyme technology (imlifidase) sales milestone payments. positive patients and book all sales of imlifidase
16Upcoming milestones
Milestones subject to potential COVID-19 impact
Kidney Kidney Kidney
3 year data read-out transplantation AMR Phase 2 transplantation transplantation
long term follow-up US: First patient Complete US: Completion GBS Phase 2 US: BLA
study dosed* enrollment** of enrollment Data read out submission
(H1 2021) (H1 2021) (H2 2021) (2022) (H2 2022) (2023)
2021 2022 2023
Kidney NiceR candidate GBS Phase 2 AMR Phase 2 Kidney
transplantation Initiation of IND-enabling Complete Data read out transplantation
EU: Commercial tox studies enrollment** (H2 2022) US: 12 months follow-
launch (H1 2021) (H2 2021) up completed
(Q1 2021) (2023)
*) FDA: Proposed study protocol submitted June 2020. Discussions are currently ongoing with the FDA. Once the final protocol has been agreed upon, Hansa Biopharma will proceed to set up
centers in the US and start to enroll patients.
**) AMR/GBS Due to the impact from the COVID-19 pandemic, the enrollment in GBS and AMR were temporarily halted during large parts of 2020. Hansa Biopharma reinitiated enrollment in Q4
2020 under a risk-based, site-by-site approach.
17Contact our Investor Relations and Visit our web site
Corporate Communications www.hansabiopharma.com
Calendar
Klaus Sindahl Mar 9, 2021 Carnegie Nordic Healthcare seminar (virtual)
Mar 10, 2021 H.C. Wainwright Global Life Science Conference (virtual)
Head of Investor Relations
Mar 25, 2021 Bryan Garnier Kidney event (virtual)
Mobile: +46 (0) 709-298 269
April 1, 2021 Guggenheim Healthcare Talks (virtual)
Email: klaus.sindahl@hansabiopharma.com April 7, 2021 ABG Small & Midcap Copenhagen (virtual)
April 8, 2021 Annual Report 2020
April 22, 2021 Interim report for Jan-Mar 2021
Katja Margell
May 5, 2021 Kempen Life Sciences Conference (virtual)
Head of Corporate Communications
May 12, 2021 Annual General Meeting 2021
Mobile: +46 (0) 768-198 326 May 19, 2021 RBC Global Healthcare Conference, NYC/virtual
Email: katja.margell@hansabiopharma.com June 1, 2021 Jefferies Healthcare Conference (virtual)
July 15, 2021 Interim report for Jan-Jun 2021
Oct 21, 2021 Interim report for Jan-Sep 2021
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