Attached files
| file | filename |
|---|---|
| 8-K - 8-K - SELECTA BIOSCIENCES INC | selectabiosciences8-k12311.htm |
| EX-99.1 - EXHIBIT 99.1 - SELECTA BIOSCIENCES INC | exhibit991_earningsrelease.htm |
March 27, 2017
Fourth Quarter and Year End 2016 Update
Safe Harbor / Disclaimer
Any statements in this presentation about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the
company”), including without limitation, statements regarding the development of its pipeline, the company's expectations about
receiving payments from Spark Therapeutics, Inc. under the license agreement, the progress of the Phase 1/2 clinical program
of SEL-212 including the number of centers in the Phase 2 clinical trial of SEL-212 and the announcement of data, conference
presentations, the ability of the company’s SVP platform, including SVP-Rapamycin, to mitigate immune response and create
better therapeutic outcomes, the potential treatment applications for products utilizing the SVP platform in areas such as gene
therapy and oncology, any future development of the company’s discovery programs in peanut allergy and celiac disease, the
sufficiency of the company’s cash, cash equivalents, investments, and restricted cash and other statements containing the
words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “hypothesize,” “intend,” “may,” “plan,” “potential,” “predict,”
“project,” “should,” “target,” “would,” and similar expressions, constitute forward-looking statements within the meaning of The
Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-
looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent
in the initiation, completion and cost of clinical trials including their uncertain outcomes, the availability and timing of data from
ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be
predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical
trials, the unproven approach of the company’s SVP technology, potential delays in enrollment of patients, undesirable side
effects of the company’s product candidates, its reliance on third parties to manufacture its product candidates and to conduct
its clinical trials, the company’s inability to maintain its existing or future collaborations or licenses, its inability to protect its
proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for
its foreseeable and unforeseeable operating expenses and capital expenditure requirements, substantial fluctuation in the price
of its common stock, a significant portion of the company’s total outstanding shares have recently become eligible to be sold
into the market, and other important factors discussed in the “Risk Factors” section of the company’s Quarterly Report on Form
10-Q filed with the Securities and Exchange Commission, or SEC, on November 10, 2016, and in other filings that the company
makes with the SEC. In addition, any forward-looking statements included in this presentation represent the company’s views
only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The
company specifically disclaims any obligation to update any forward-looking statements included in this presentation.
2
The Experts Agree: Immunogenicity is a Serious
Challenge to Biologic Therapy Development
COMPROMISED EFFICACY
Anti-drug antibodies (ADAs)
neutralize therapeutic benefit
3
UNPREDICTABLE RESPONSE
Changed PK/PD through drug-ADA
interaction
SAFETY RISK
Hypersensitivity reactions
can impact patients
I M M U N O G E N I C I T Y ’ S I M P A C T
“For the gene therapies
today in clinical
development that apply
AAV-vectors systemically,
no repeat dose is possible
due to neutralizing
antibodies.”
– Federico Mingozzi, PhD
INSERM, France
“Immunological
responses are a
significant risk in CRIM-
negative infantile Pompe
disease; thus induction of
immune tolerance in the
naive setting should
strongly be considered.”
– Priya Kishnani, MD ea
Duke University
“Hemophilia A
patients with
inhibitors to Factor
VIII replacement
therapy are the
hardest and most
expensive patient
group to treat.”
– David Scott, PhD
Uniformed Services
University
“Clinical trial results point to a
direction in targeted cancer
therapy, whereby improved
clinical responses might occur
through combining
immunotoxin therapy with
immune modulation.”
– Raffit Hassan, MD ea
Uniformed Services University
“Prophylactic immune tolerance induction should be strongly considered in patients who are at risk of developing immune
responses to ERT.”
– Amy Rosenberg, MD, Director of the FDA’s Office of Biotechnology Products
IMAGINE IF
WE COULD…
1. Effectively treat many more
patients with existing biologics
2. Enable a new generation of novel
non-immunogenic biologics for
rare and serious diseases
4
No/not
diagnosed tophi
Severe Gout is a Rare and Serious Disease
with Substantial Unmet Needs
8.3
3.1
5.2 4.7
0.5
US Gout
Patients
Rx Treated
Primary
Care, Endo,
Nephro,
Other
Rheum*
Gout Patients (million)1
530,000
370,000
Estimated SEL-212 Target Patient Population1
US Gout treated at
Rheum
Est. SEL-212
patient pool
Un-
diagnosed or
no Rx
treatment
US Gout
Prevalence
* Rheumatologists see estimated 10% of treated gout patients
(1) Source: IMS, Desk Research, Selecta Rheum interviews, Crystal patient registry
(2) Includes an estimated 50,000 patients with chronic refractory gout
Severe, Uncontrolled Gout Target Patient Population
160,0002
5
• Experience intense pain, inflammation, gouty arthritis and debilitating flares caused by uric acid
crystal deposits in joints and tissue
• At risk for kidney and cardiovascular disease if left untreated
• High unmet need for patients today
SEL-212 Designed to Treat Severe Gout Patients,
Addressing an Unmet Need
6
• Serum Uric Acid of <6 mg/dL: The clinical target for gout treatment and the
primary clinical endpoint for FDA/EMA approvals of gout medications
• Serum Uric Acid of >6.8 mg/dL: Limit of uric acid solubility in water; above
this level, uric acid deposits form in joints and tissue
• For severe gout patients, objective is to drastically lower serum uric acid
levels to enable the rapid clearing of existing deposits
o Cannot be achieved readily and consistently by oral therapies
o While uricase enzymes have demonstrated this potential, immunogenicity
prevents clearance for most patients
• SEL-212 is designed to be the first non-immunogenic uricase treatment,
enabling:
o Severe gout patients to be treated with repeat infusions spanning a short
treatment cycle
o Retreatments due to SVP technology’s use
Phase 2 Trial Overview
7
• Patients with symptomatic gout and serum uric acid levels >6 mg/dL
• Safety, tolerability and pharmacokinetics of multiple doses of
SEL-212 and pegsiticase alone
• Reduction of serum uric acid levels
• Reduction of ADA levels
• Multiple ascending dose cohorts
• Control cohorts: pegsiticase alone every 28 days for up to five doses
• All other cohorts: SEL-212 every 28 days for three doses followed by
two doses of pegsiticase alone
• Dosing stopped upon failure to control serum uric acid
• 38 patients dosed at 10 active U.S. clinical sites
Enrollment Criteria
Primary/Secondary
Endpoints
Design
Dosing
Stopping Rules
As of March 23
Mid-Dose Cohort A: 0.08 mg/kg of SVP-Rapamycin +
0.4 mg/kg of Pegsiticase
8
Unaudited data as of March 23, 2017.
7 patients enrolled:
• One withdrawn for
protocol deviation (not
shown)
• All 6 remaining patients
maintained serum uric
acid control through
March 23
o Five received 3
doses
o One received 2
doses
• No SAEs to date
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0.4 mg/kg Pegsiticase +
0.08 mg/kg SVP-Rapamycin 0.4 mg/kg Pegsiticase
Patient
114-0001
Patient
107-0004
Patient
111-0002
Patient
110-0008
Patient
106-0035
Patient
104-0010
Days
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
Mid-Dose Cohort B: 0.08 mg/kg of SVP-Rapamycin +
0.2 mg/kg of Pegsiticase
9
6 patients enrolled:
• Five maintained serum
uric acid control
through March 23
o Four received 3
doses
o One received 2
doses
• One patient met
stopping rule
• No SAEs to date
0.2 mg/kg Pegsiticase +
0.08 mg/kg SVP-Rapamycin 0.2 mg/kg Pegsiticase
Patient
106-0025
Patient
110-0002
Patient
104-0003
Patient
106-0038
Patient
110-0001
Patient
102-0010
Days
Stopping rule met
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
Unaudited data as of March 23, 2017.
Low-Dose Cohort A: 0.05 mg/kg of SVP-Rapamycin +
0.4 mg/kg of Pegsiticase
10
10 patients enrolled:
• Four withdrawn for
protocol deviation (not
shown)
• One received 3 doses,
maintaining serum uric
acid control through
March 23
• Five met stopping rule
for failure to maintain
control of uric acid
• No SAEs to date
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0.4 mg/kg Pegsiticase +
0.05 mg/kg SVP-Rapamycin 0.4 mg/kg Pegsiticase
Patient
114-0001
Patient
110-0003
Patient
103-0015
Patient
102-0008
Patient
140-0007
Patient
110-0006
Missed
visits
Stopping rule met
Unaudited data as of March 23, 2017.
Low-Dose Cohort B: 0.05 mg/kg of SVP-Rapamycin +
0.2 mg/kg of Pegsiticase
11
9 patients enrolled:
• Three withdrawn for
protocol deviation (not
shown)
• One completed all five
doses, maintaining
serum uric acid control
for trial’s duration
• Two patients received
3 doses, maintaining
serum uric acid control
through March 23
• One met stopping rules
for failure to control
serum uric acid
• 2 SAEs (infusion
reaction) that were
successfully treated
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0.2 mg/kg Pegsiticase +
0.05 mg/kg SVP-Rapamycin 0.2 mg/kg Pegsiticase
Patient
114-0001
Patient
101-0009
Patient
106-0023
Patient
106-0022
Patient
103-0014
Patient
102-0007
Days
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
Stopping rule met SAE; infusion reaction
Unaudited data as of March 23, 2017.
12
Control Cohorts: 0.2 or 0.4 mg/kg of Pegsiticase Alone
6 patients enrolled:
• Five failed to maintain
serum uric acid control
o One SAE (infusion
reaction) after 2nd
dose
• As expected,
enrollment terminated
early for patient safety
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
1 2
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
0
2
4
6
8
1 0
0.2 mg/kg or 0.4 mg/kg Pegsiticase
Patient
106-0013
Patient
101-0005
Patient
106-0015
Patient
106-0008
Patient
109-0003
Patient
107-0001
Days
0 7 1 4 2 1 2 8 3 5 4 2 4 9 5 6 6 3 7 0 7 7 8 4 9 1 9 8 1 0 5 1 1 2 . 1 1 9 1 2 6 1 3 3 1 4 0
4
6
8
1 0
Stopping rule met SAE; Infusion reaction Patients lost to pause in clinical trial
while stopping rules were modified
0
.4
m
g
/kg
P
e
g
sitic
a
s
e
0
.2
m
g
/k
g
P
e
g
siticas
e
Unaudited data as of March 23, 2017.
Immune Tolerance SVP Platform Designed
to be Utilized Broadly
13
IMMUNE TOLERANCE SVP
Encapsulated
Rapamycin
Encapsulating
Nanoparticle
PLA+PLA-PEG
Targeted
Immunotoxins AntibodiesViral VectorsEnzymes/ERT
SVP-Rapamycin’s preclinical, clinical and manufacturing data can
be applied across a broad range of product candidates
Q4 Financial Overview
For the Quarter Ended
(In thousands, except share and per share data)
December 31,
2016
December 31,
2015
Grant & Collaboration Revenue $2,930 $2,134
Research & Development Expenses 11,033 7,211
General & Administrative Expenses 5,757 2,030
Net Loss Attributable to Common Stockholders ($14,083) ($9,225)
Net Loss Per Basic Share ($0.77) ($4.26)
Wtd. Avg. Common Shares Outstanding – Basic & Diluted 18,265,771 2,167,769
As of
(In thousands)
December 31,
2016
September 30,
2016
Cash, Cash Equivalents, Marketable Securities, Restricted Cash $84,535 $79,927
Selecta believes its cash, cash equivalents, short-term deposits, investments and
restricted cash will be sufficient to fund the company into mid-2018
14
