Posterior Segment Company Showcase - Aura Biosciences at OIS@AAO 2016.
Presenter:
Elisabet de los Pinos, CEO
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3. Disclaimers and forward-looking statements
This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this
presentation, including statements regarding our short-term and long-term strategies, efforts to develop and commercialize our
products, future operations, future financial position, future revenue, projected costs, prospects, plans, objectives of management and
expected market growth are forward-looking statements. 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. The words âanticipate,â âbelieve,â âcould,â
âestimate,â âexpect,â âintend,â âmay,â âplan,â âpotential,â âpredict,â âproject,â âshould,â âtarget,â âwill,â âwouldâ and similar expressions are
intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We
expressly disclaim any obligation to release any updates or revisions to any forward-looking statements to reflect any change in our
expectations or projections or any changes in events, conditions or circumstances on which any such statements are based for any
reason, except as required by law, even as new information becomes available. All forward-looking statements in this presentation are
qualified in their entirety by this cautionary statement.
Certain information contained in this presentation and statements made orally during this presentation relates to or is based on
information from industry publications and research, surveys and studies conducted by third parties. Industry publications and third
party research, survey and studies generally indicate that their information has been obtained from sources believed to be reliable,
although they do not guarantee the accuracy or completeness of such information. While we believe these industry publications and
third party research, surveys and studies are reliable, we have not independently verified such data.
This presentation shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of
any securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification
under the securities laws of any such state or jurisdiction.
4. Working to transform the treatment of patients with
choroidal melanoma
4
Targeted therapy with unique
mechanism of action
Orphan Drug Designation granted
May 2015
Potential for early clinical readout and
accelerated regulatory path forward
Platform technology for non-ocular
cancers
Management team with expertise
in orphan drug development and
market launch
5. 1 Based on epidemiology estimates by Health Advances (US) and Company (Europe and rest of world)
2 COMS_2005. Collaborative Ocular Melanoma Study Group Report No. 26. Development of metastatic disease after enrollment into the COMS trials for treatment of choroidal
melanoma. Arch Ophthalmol 2005;123(12):1639-43
High unmet medical need with no FDA-approved
therapies
5
Choroidal melanoma is a rare disease.
Diagnosed and treated by
ocular oncologists (retina
specialists)
54
ocular oncologist in the U.S.
52
ocular oncologist in Europe
10,000
patients worldwide1
Early diagnosis of
primary tumor
High unmet medical need for
the treatment of disease with
vision preservation
25%
of patients develop
metastatic disease after
five years2
6. Current radioactive treatment options with high
association of comorbidities
1. Medium melanomas: Plaque
brachytherapy and proton
beam radiotherapy
- Requires multiple surgeries
- Causes severe vision loss (>5 lines)1
- Does not prevent metastases2
- Cost of treatment: $50,0003
2. Large melanomas: Enucleation
6
1 Shields CL et al. Arch Ophthalmol. 2000 Sep;118(9):1219-28
2 COMS Report No. 18. Arch Ophthalmol 119: 969-982, 2001.
3 Harbour et al. Investigative Ophthalmology & Visual Science March 2012, Vol.53, 3400
7. AU-011: A first-in-class targeted therapy
Targeted cytotoxicity upon light activation
7
Small molecule is conjugated to
the primary amines in the surface
of the viral nanoparticle. The drug
is inactive without irradiation.
Viral nanoparticle conjugates
(VNC) are delivered by intra-
vitreal injection and target tumor
cells in the choroid.
VNCs bind specifically to
HSPGs on the tumor cell
surface (multivalent binding).
Ophthalmic laser 689nm activates
the drug. The light-activated drug
disrupts the tumor cell membrane,
leading to necrosis.
8. Efficient outpatient treatment protocol
8
Routine administration by
intra-vitreal injection â same
as AvastinÂź, EyleaÂź, LucentisÂź
Activated by ophthalmic laser
within six hours in standard
outpatient setting
689nm laser activation â
same wavelength and light
fluence as used for VisudyneÂź
9. Significant response to AU-011 in tumor model with
high correlation to human disease
9
Rabbit orthotopic tumor model with
human choroidal melanoma cells1
Administration schedule: once a week for
two or three weeks
All animals with small tumors had
complete responses to the therapy2
Small number of incomplete responders
had large tumors that extended beyond
the treatment field of the laser
Data replicated in multiple academic labs3
AU-011 Dose Response
% Complete ResponseDose
0%Control
0%5 ug
20%20 ug
50 ug 80%
1 Grossniklaus et al. Investigative Ophthalmology & Visual Science June 2013, Vol.54, 4229
2 Complete Responses defined by >90% tumor necrosis or absence of tumor cells after treatment
3 Dr. Grossniklaus/Emory University; Dr. Burnier/McGill University; BRM with oversight from Dr. Vavvas/ MEEI
10. Efficacy results validated by histopathology
10
Complete responses after intra-vitreal injections of AU-011 once a week for
two weeks in medium-large tumors
Original location of
treated posterior tumor
Data: Fundoscopy and Histopathology images provided by Ora Clinical-BRM. Tumor implants performed by Dr. Vavvas Harvard Medical School. Data reviewed by Dr. Grossniklaus
(Certified Ocular Pathologist at Emory University)
Histopathology (H&E):
Posterior choroidal
tumor with extra-ocular
extension (untreated
control)
Histopathology (H&E):
No signs of posterior
tumor after administration
of AU-011 once a week
for two weeks and light
activation
A B
11. Near-term clinical milestones
11
AU-011 could be the first FDA-approved therapy for primary choroidal melanoma.
Phase II/III Clinical TrialPhase I Clinical Trial
PIND agreement on
clinical endpoints:
- Safety / vision
- Tumor response
IND
NDA
Robust preclinical data
with complete
responses in multiple
independent animal
model studies
Preclinical IND-Enabling Studies
Safety
Preliminary
efficacy
Registration trial
Phase II/III
2014 2015 2016 2017 2018
Fast Track /
Breakthrough
Designations
Clinical operations
underway:
- Multiple U.S. sites
qualified
- Laser system setup
and training complete
at a number of sites
2019
12. 12
Carol Shields, M.D.
Sara Selig, M.D.
A strong rare disease community of patients,
foundations and ocular oncologists
13. Rare disease with large market potential
13
Choroidal melanoma represents
a unique rare disease
commercial opportunity.
Unmet medical need with no
FDA-approved therapies
Orphan drug market access and
reimbursement value
Focused call points enable efficient
commercialization opportunity
Initiating corporate and financing
discussions to develop drug
through approval
15. Working to transform the lives of patients with
choroidal melanoma
15
First-in-class targeted cancer therapy
Strong preclinical data in animal model
with high correlation to human disease
Granted FDA Orphan Drug
Designation
Phase I 2017
Rare disease with potential
accelerated regulatory path forward