Document


UNITED STATES

SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
 
 
FORM 8-K

 
CURRENT REPORT
 
Pursuant to Section 13 or 15(d) of
the Securities Exchange Act of 1934
 
Date of report (Date of earliest event reported): January 8, 2018

 
SELECTA BIOSCIENCES, INC.
(Exact name of registrant as specified in its charter)
Delaware
 
001-37798
 
26-1622110
(State or other jurisdiction of
incorporation or organization)
 
(Commission
File Number)
 
(I.R.S. Employer
Identification No.)
 
480 Arsenal Way
Watertown, MA 02472
(Address of principal executive offices) (Zip Code)
 
(617) 923-1400
(Registrant’s telephone number, include area code)
 
N/A
(Former Name or Former Address, if Changed Since Last Report)
 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions
 
 
o             
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
 
 
o             
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
 
 
o             
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
 
 
o             
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
 
 

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company x

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. x






Item 7.01. Regulation FD Disclosure.

Selecta Biosciences, Inc. (the “Company”) from time to time presents and/or distributes to the investment community at various industry and other conferences slide presentations to provide updates and summaries of its business. A copy of its current corporate slide presentation (the “Presentation”) is attached to this Current Report on Form 8-K as Exhibit 99.1. The Company undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibit 99.1.

The information in Item 7.01 of this Form 8-K, including Exhibit 99.1 attached hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.

Item 8.01. Other Events.

On January 8, 2018, in connection with the presentation and/or distribution of the Presentation, the Company announced that it expects to report initial results for its Phase 2 clinical trial of SEL-212 in chronic severe gout in April 2018 and further expects to hold an End of Phase 2 Meeting with the U.S. Food and Drug Administration in the second quarter of 2018.

The Company also expects to submit an investigational new drug application for SEL-302 in methylmalonic acidemia in 2019.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

 
Exhibit
No.
 
Description
 
 
 
 
 
 









SIGNATURES
 
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
 
 
 
 
 
 
SELECTA BIOSCIENCES, INC.
 
 
 
 
Date: January 8, 2018
By:
/s/ Werner Cautreels, Ph.D.
 
 
Werner Cautreels, Ph.D.
 
 
President and Chief Executive Officer



exhibit991jan2018investo
Investor Presentation Nasdaq: SELB January 2018


 
Safe Harbor / Disclaimer 2 Any statements in this presentation about the future expectations, plans and prospects of Selecta Biosciences, Inc. (“the company”), including without limitation, the progress of the Phase 1/2 clinical program of SEL-212, whether the company participates in an End-of-Phase 2 meeting for SEL-212 in Q2 2018, the potential of SEL-212 to treat severe gout patients and resolve their debilitating symptoms, the ability of SVP-Rapamycin to avoid unwanted immunogenicity and unlock the full potential of a range of therapeutics, whether higher level doses of SVP-Rapamycin or SEL-212 will show increased clinical activity and durability in line with the Phase 1b, when the company will report further data from the Phase 2 trial, whether the FDA approves the company’s plan to provide combination therapy of SEL-212 for the entire treatment period, whether the company will determine an appropriate dose of SEL-212 for the Phase 3, when the company will advance to a Phase 3 for SEL-212 (if at all), whether SEL-212 has the potential to address the unmet needs of gout patients, whether SEL-212 will lower the incidence of flares, whether SEL-212 holds billion dollar potential, the ability of the company’s SVP platform, including SVP-Rapamycin, to mitigate immune response and create better therapeutic outcomes or to improve the efficacy or safety of existing biologics, whether the SVP platform enables the biologic to be distributed broadly to desired sites of action, the potential treatment applications for products utilizing the SVP platform in areas such as enzyme therapy, gene therapy, oncology therapy, vaccines and treatments for allergies and autoimmune diseases, the potential of future collaborationsor licenses based on the ability of SVP-Rapamycin, the potential of the SVP-Rapamycin platform, generally, whether the company commences a Phase 1 trial for SEL-403 in Q1 2018, whether the company files an IND for SEL-302 in 2019, the company's expectations about receiving additional payments from Spark Therapeutics, Inc. under the license agreement and/or the stock purchase agreement, 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 7, 2017, 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.


 
Corporate Overview 3 • Clinical-stage company applying proprietary Synthetic Vaccine Particle (SVP™) platform to avoid unwanted immunogenicity and unlock the full potential of biologic therapies • Expect to begin Phase 3 in 2018 with SEL-212 (SVP-Rapamycin + pegsiticase) for chronic severe gout • IND just accepted for second product candidate: SEL-403 (SVP-Rapamycin + LMB-100) for mesothelioma • Proprietary gene therapy candidates in preclinical development • License agreement in place with Spark Therapeutics, with additional potential for collaborations and licenses in a range of therapeutic areas


 
Immunogenicity is Now Recognized as a Serious Challenge for Biologic Therapies 4 COMPROMISED EFFICACY Anti-drug antibodies (ADAs) neutralize therapeutic benefit 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 “With the explosion of biologic products on the market and in research pipelines, we’ve become very concerned about the effectiveness and safety of these drugs.” – Amy Rosenberg, MD, Director, Division of Biotechnology Products Review and Research, FDA When the Immune System Thwarts Lifesaving Drugs Patients often produce antibodies to the very treatments keeping them alive, sometimes to disastrous effect… January 2018 Edition


 
Preventing Unwanted Immunogenicity via Selecta’s SVP-Rapamycin Technology Platform 5 Spleen Preventing ADAs Inducing a tolerogenic response to a biologic drug (antigen) Implementing the message Sending precise instructions Targeting immune cells • By dosing “free biologic” separate from SVP- Rapamycin, it distributes broadly to desired sites of action • Some of the biologic co- localizes with dendritic cells that have taken up SVP- Rapamycin • The dendritic cells then induce regulatory T cells that circulate throughout the body and suppress immune responses against the biologic (i.e. ADAs)  Naïve T cell B cell Helper T cell Dendritic cell Regulatory T cell Potential to enable new therapies and improve efficacy/safety of existing biologics SVP-Rapamycin Biologic drug Kishimoto et al., Improving the efficacy and safety of biologic drugs with tolerogenic nanoparticles, Nature Nanotechnology, Aug. 2016


 
Leveraging Nanoparticles to Target and Deliver Instructions to the Immune System 6 I.V. Injection (6hr post-injection) Spleen harvested 24 hr after I.V. Injection of fluorescent NPs  SVP  Macrophages  Dendritic cells  B cells SVP Antigen Spleen


 
Selecta’s Pipeline Development Strategy 7 Chronic Therapies+ Proprietary program Potential program Single Treatment Chronic Treatment Adult Patients with Chronic Conditions Cancer Patients Pediatric Patients with Life-Threatening Conditions Entering Phase 1: SEL-403 for Mesothelioma Treatment Cycle + Preclinical: SEL-302 & SEL-313 Gene Therapy Candidates+ Phase 2: SEL-212 for Chronic Severe Gout + • SVP platform holds great potential • SEL-212 program informing other programs with data from >100 patients • Current clinical data demonstrate ADA prevention and support development of our near- term pipeline TBD Patient Population D o sin g Regi m e n


 
SEL-212 for Chronic Severe Gout


 
SEL-212: Advancing a Potential New Treatment Option for Chronic Severe Gout Patients Toward Phase 3 9 Rare and Serious Disease • ~160,000 adults with chronic severe gout treated by U.S. rheumatologists • Debilitating flares and joint-damaging arthritis caused by uric acid deposits; risk of renal and cardiovascular disease Immunogenicity Barrier • Uricases are highly effective in breaking down uric acid deposits, but are foreign to the human immune system, causing immunogenicity in nearly all patients that can negate efficacy and present safety risks Clear Clinical Path • Serum uric acid level reduction – a robust FDA/EMA primary endpoint for approval – can be seen rapidly upon dosing; easy to measure; maintenance strongly correlated with low/negative ADA titers • Adult patient population with rapid enrollment potential Ownership • In-licensed pegsiticase in 2014; combined with SVP-Rapamycin to form SEL-212


 
Today’s Unmet Needs in Chronic Severe Gout • Monthly dosing (vs. biweekly for today’s approved uricase therapy) • Ability to complete full therapy cycles - Persistent reduction in SUA - Elimination of tophi • Gout flare reduction • Avoidance of “off-label” and global immunosuppressive therapies 10 We believe SEL-212 has the potential to address these unmet needs and holds billion-dollar potential


 
Phase 1b Single Dose Patient Cohorts 11 IMVACS 2016 Presentation. Clinicaltrials.gov NCT02648269 S e ru m U ric A cid ( m g /d L ) 0.03 mg/kg 0.4 mg/kg 0.1 mg/kg 0.4 mg/kg 0.4 mg/kg 0.3 mg/kg 0.4 mg/kg 0.15 mg/kg 0.4 mg/kg 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0 2 4 6 8 1 0 0 2 4 6 8 1 0 N = 5 N = 5 N = 10 N = 5 N = 5 = Pegsiticase; = SVP-Rapamycin Days S in g le Dos e 0% 70% 100% 100% 20% % Patients sUA < 6mg/dL at Day 30 0 7 14 21 30


 
Phase 2 Trial Overview 12 Enrollment Criteria Primary/Secondary Endpoints Design Dosing Stopping Rules As of October 23 • Patients with symptomatic gout and SUA levels >6 mg/dL • Safety, tolerability and pharmacokinetics of multiple doses of SEL-212 and pegsiticase alone • Reduction of SUA levels • Reduction of ADA levels • Multiple ascending dose cohorts • Control cohorts: pegsiticase alone every 28 days for up to five doses • Other cohorts: currently testing three combination doses of SEL-212 every 28 days followed by 2 doses of pegsiticase alone • Preparations now underway to enable five combination doses • Dosing stopped upon loss of SUA control at Day 21 after each dose • 79 patients dosed at 15 active U.S. clinical sites


 
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 0 2 1 0 3 1 0 4 1 0 5 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 0 2 1 0 3 1 0 4 1 0 5 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 0 2 3 4 5 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 0 2 3 4 5 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 0 2 3 4 5 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 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 # ♦ * + #114-0004 111-0004 103-0018 104-1014 106-1039 105-0001 114-0007 104-0019 104-0024 104-0020 114-0009 S e ru m U ric A cid ( m g /d L ) A n ti-U ricas e A n ti b o d y T it e r Cohort 7: 0.2 mg/kg Pegsiticase + 0.1 mg/kg SVP-Rapamycin 13* #♦ Patient Unaudited data reported as of October 23, 2017 | Clinicaltrials.gov NCT02959918 Pegsiticase AlonePegsiticase + SVP-Rapamycin Day 0.1 mg/kg 0.2 mg/kg Discontinuation due to infusion reaction Withdrawal of consent SAE; infusion reaction Stopping rules 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 1 0 2 1 0 3 1 0 4 1 0 5 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 0 2 1 0 3 1 0 4 1 0 5 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 0 2 3 4 5 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 0 2 3 4 5 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 0 2 3 4 5 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 0 2 3 4 5 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 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 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 1 0 2 3 4 5 14 104-0018 110-0015 103-0020 104-0017 106-0054 106-0045 107-0008 103-0019 111-0008 104-0023 106-0065 106-0064 * # # # # * ϴ ϴ Cohort 8: 0.4 mg/kg Pegsiticase + 0.1 mg/kg SVP-Rapamycin Unaudited data reported as of October 23, 2017 | Clinicaltrials.gov NCT02959918 Patient S e ru m U ric A cid ( m g /d L ) A n ti-U ricas e A n ti b o d y T it e r 0.1 mg/kg 0.4 mg/kg Pegsiticase AlonePegsiticase + SVP-Rapamycin Day SAE; not study drug related SAE; infusion reaction Stopping rules metϴ * #


 
0.2 or 0.4 mg/kg Pegsiticase Alone Control Cohorts (Cohorts 1 & 2) % of Patients With SUA <6 mg/dL Comparison of Clinical Activity and Immune Tolerance Between Mid-Dose and Control Cohorts 15 Unaudited data reported as of October 23, 2017 | Clinicaltrials.gov NCT02959918 * Ph1b Day 30 @ 0.1 mg/kg SVP-Rapamycin + 0.4 mg/kg pegsiticase ** Patients who received a full first dose and completed treatment cycle 1 = Pegsiticase; = SVP-Rapamycin 70%* Pegsiticase + SVP-Rapamycin 0.1 mg/kg 0.2 or 0.4 mg/kg Pegsiticase Alone Mid-Dose Cohorts (Cohorts 7 & 8**) 0 5 0 1 0 0 D a y % P ts S UA <6 mg /dL 0 2 8 5 6 8 4 1 1 2 1 4 0 0 5 0 1 0 0 D a y % P ts S U A < 6 m g /d L 0 2 8


 
1 1 2 3 4 5 0 2 0 4 0 6 0 16 • Data indicate SEL-212 lowers flares initially and over time during treatment • Urate lowering therapies typically increase the incidence of flares at the beginning of therapy Unaudited data as of October 23, 2017 | Clinicaltrials.gov NCT02959918 % o f P at ie n ts w /Fl a re Pegsiticase Alone % of Patients Experiencing Flares by Month SEL-212 Month 50% 24% 16% 3% 11% 5% Low Frequency of Gout Flares Observed with SEL-212 Treatment


 
SEL-212 Generally Well Tolerated at Clinically Active Doses 17 • SEL-212 has been generally well tolerated at clinically active doses following >200 administrations • SAEs reported in the Phase 2 trial: - Four were reported not to be or unlikely to be related to study drug - Seven infusion reactions: - Four in cohorts receiving pegsiticase alone or pegsiticase in combination with the lowest dose of SVP- Rapamycin, as anticipated - Two due to protocol deviations related to dosing errors - One during a repeat dose of SEL-212 in a higher dose cohort - Each of these SAEs occurred prior to Selecta’s June data report - None occurred after treatment period 2 • All SAEs were successfully treated and resolved without further issues Unaudited data as of October 23, 2017 | Clinicaltrials.gov NCT02959918


 
SEL-212 Expected to Enter Phase 3 in 2018 18 • SEL-212 (SVP-Rapamycin + pegsiticase) designed to be the first non- immunogenic uricase treatment for chronic severe gout • Phase 2 trial ongoing: - SEL-212 has been demonstrated to be clinically active and generally well tolerated - Cohorts receiving 0.125 and 0.15 mg/kg doses of SVP-Rapamycin ongoing; plan to report initial data for these cohorts in the first half of April 2018 - Plan to begin enrolling patients in Q1 2018 that would receive combination therapy for the entire treatment period; preparing supporting preclinical FDA submission • Plan to participate in End-of-Phase 2 meeting in Q2 2018 • Plan to enter Phase 3 in 2018


 
SEL-403 for Mesothelioma


 
SEL-403: A Highly Potent Recombinant Pseudomonas Immunotoxin Targeting Mesothelin 20 Rare and Serious Disease • Mesothelin expressed in virtually all mesotheliomas (~3,000 annual U.S. diagnoses1) and pancreatic cancers (~50,000); high percentage of ovarian, lung, breast cancers • Certain solid tumors are particularly hard to treat and have remained evasive to immunotherapy approaches Immunogenicity Barrier • LMB-100 induces inhibitory antibodies upon first dose in almost all patients, limiting dosing to one or two administration cycles; insufficient to control tumor • Global immunosuppressants ineffective in preventing ADAs in a vast majority of patients • SVP allowed 3+ treatment cycles in pre-clinical models, restoring LMB-100 preclinical activity • Initial repeat dose data from ongoing SEL-212 Phase 2 bodes well for this application Clear Clinical Path • Both components of SEL-403 (SVP-Rapamycin and LMB-100) have been in the clinic in separate trials • FDA acceptance of IND for combination treatment announced in January; expect Phase 1 mesothelioma trial to be underway in Q1 2018 Ownership • In-licensed LMB-100 from NCI in April 2017; up to $9.25 million in milestones; low single-digit royalties • Combination with SVP-Rapamycin now known as SEL-403 1. Beebe-Dimmer et al., Mesothelioma in the United States: a Surveillance, Epidemiology, and End Results (SEER) – Medicare investigation of treatment patterns and overall survival, Clin Epidemiol., Oct. 2016


 
Immunotoxin LMB-100 21 • LMB-100: Pseudomonas exotoxin A linked to antibody Fab targeting mesothelin • Technology was licensed to Roche but later returned to NCI • Efficacy was limited by immunogenicity after one or two cycles in most patients • Currently in Phase 1 clinical trials LMB-100 Mesothelin is overexpressed on many solid tumors Ira Pastan, M.D. Head, Molecular Biology Section National Cancer Institute • Mesothelioma (>90%) • Pancreatic cancer (>90%) • Ovarian cancer (70%) • Lung cancer (50%) • Breast cancer (34%)


 
Clinical Activity of SS1P (LMB-100 Precursor) in Mesothelioma 22 Day 12 3 months 15 months Before treatment 1.6 months 8 months Patient 1 • Widely metastatic peritoneal mesothelioma • Survived 32 months Patient 2 • Extensive pleural mesothelioma • Survival >6 years (still alive) S u m o f ta rg e t le s io n s ( c m ) Months after treatment S u m o f ta rg e t le s io n s ( c m ) Months after treatment Hassan, Pastan et al. Cancer Research (2014) While patients receiving ≥4 cycles showed major anti-tumor response, immunogenicity limited treatment to 1 or 2 cycles for most patients despite use of immunosuppressive therapy 6 Treatment Cycles 4 Treatment Cycles


 
Preclinical Data Supports the Benefits of SVP-Rapamycin + LMB-100 Combination Therapy 23 Prevents formation of anti-drug antibodies Restores LMB-100’s anti-tumor response SVP alone does not accelerate tumor growth T u m o r G r o w t h D a y s s i n c e t u m o r i n o c u l a t i o n T u m o r s iz e ( m m 3 ) 0 1 0 2 0 3 0 0 5 0 0 1 0 0 0 1 5 0 0 S a l in e S V P - R a p a m y c in - 1 0 - 5 0 5 1 0 1 5 2 0 5 0 0 1 0 0 0 1 5 0 0 2 0 0 0 2 5 0 0 D a y s s i n c e t u m o r i n o c u l a t i o n T u m o r s iz e ( m m 3 ) L M B - 1 0 0 S a l i n e L M B - 1 0 0 + S V P T u m o r G r o w t h W e e k A n ti -L M B -1 0 0 I g G (  g /m l) 0 2 4 6 8 0 1 0 0 2 0 0 3 0 0 4 0 0 L M B -1 0 0 S V P L M B -1 0 0 L M B -1 0 0 + S V P A n ti-L M B -1 0 0 A n tib o d y Data generated in collaboration with Dr. Ira Pastan, NCI SEL-403


 
SEL-403 Entering the Clinic at NCI 24 • IND acceptance announced on January 2, 2018 • Expect to commence a dose-escalating Phase 1 trial in Q1 2018 under a CRADA at NCI • Enrolling up to 18 patients with malignant pleural or peritoneal mesothelioma who have undergone at least one regimen of chemotherapy • Patients to receive four treatment cycles of the combination product candidate • Primary objective: Evaluate the safety and tolerability of the combination therapeutic candidate in the study population • Additional measurements: Objective response rates and ADA titers CRADA #3157 with NCI


 
Proprietary & Licensed Gene Therapy Programs


 
Selecta’s Proprietary Gene Therapy Programs 26 Rare and Serious Disease • Two rare inborn error of metabolism: Methylmalonic Acidemia (MMA) and Ornithine Transcarbamylase (OTC) Deficiency • Onset in early infancy; significantly reduces life expectancy Immunogenicity Barrier • Infants require treatment prior to metabolic crisis to avoid CNS effects; retreatment likely needed as patients grow • Repeat systemic gene therapy dosing currently not possible due to neutralizing antibodies to viral capsid • Cellular immune responses to the liver are an additional potential barrier Clear Clinical Path • Lead gene therapy program is SEL-302 for MMA • Clinical endpoints include: Methylmalonyl-CoA mutase and MMA levels • Expect to file IND in 2019 Ownership • Two proprietary gene therapies utilizing Anc80 and AAV + SVP-Rapamycin (SEL-302 & SEL-313)


 
Benefits of ADA Avoidance in Gene Therapy 27 Inhibiting Liver Inflammation from First Dose Allowing for Repeat Dosing And Dose Titration CD8 T cell Liver Infiltrates Serum ALT Enzyme Levels + AAV8 SVP or Empty NP 0Day SVP or Empty NP + AAV8-Luc AAV8-Factor IX Day 0 21 54 SVP or Empty NP Anti-AAV8 Antibody Titer Serum Factor IX Expression Preclinical data generated in mice in collaboration with Dr. Federico Mingozzi, Genethon S V P E m p ty N P -1 0 1 2 3 4 5 C D 8 m R N A (   C t) S V P E m p ty N P 0 5 1 0 1 5 2 0 ** A L T a ct iv ity ( m U /m L ) 0 2 0 4 0 6 0 0 5 0 0 0 1 0 0 0 0 1 5 0 0 0 2 0 0 0 0 2 5 0 0 0 D a y s A n ti- A A V 8 T ite r 0 40000 80000 120000 160000 200000 34 41 54 H u m an F IX ( n g/ m l) Days SVP-Rapamycin Empty NP


 
Demonstration of the Role of Regulatory T Cells 28 Day 0 19 20 21 32 0 1 28 Effect can be Transferred to a Recipient T Reg Depletion Negates Effect Anti-AAV8 IgG levels in recipient mice (Day +14) DONOR AAV8-Luc +/- SVP 0 14Day Day RECIPIENT AAV8-hFIX AAV8-Luc +/- SVP PRIME Anti-CD25 Treg Depletion CHALLENGE Anti-AAV8 IgG levels (Day 32) ** P < 0.01, *** P < 0.001 Transfer splenocytes a n ti -A A V 8 I g G (  g /m L ) E m p ty N P S V P 0 5 1 0 1 5 2 0 * * * 0 1 0 2 0 3 0 4 0 5 0 *** a n ti -A A V 8 I g G ( n g /m L) ** S V P -R a p a m y c in T re g d e p le t io n + + ++- - - - Preclinical data generated in mice in collaboration with Dr. Federico Mingozzi, Genethon


 
Anc80/synMUT Proof of Concept in Mouse Model of MMA at ASGCT 2017 29 0 5 0 1 0 0 1 5 0 2 0 0 0 5 0 0 1 0 0 0 1 5 0 0 2 0 0 0 D a y s M M A (  M ) A n c 8 0 -h A A T -M u t4 5 e 1 2 V G A A V 8 -h A A T -M u t4 5 e 1 2 V G 0 5 10 15 20 25 30 35 Weight Gain in 12 days (%) Anc80 or AAV8 Retro Orbital injection Reducing MMA Levels With Anc80 and AAV8 Increasing Weight Gain Following Treatment Per c en t w eig h t gai n Preventing Anti-Anc80 Antibodies via SVP-Rapamycin Preclinical data generated in mice in collaboration with Dr. Charles Venditti, NIH, and Dr. Luk Vandenberghe, Mass Eye & Ear Anti-Anc80 Antibodies ** (P = 0.036) NS (P = 0.44) (N=3) (N=3) (N=3) Anc80 AAV8 No Tx A n t i- A n c 8 0 I g G ( O D 4 5 0 - 5 7 0 ) 0 .0 0 .5 1 .0 1 .5 2 .0 D a y 7 D a y 1 2 D a y 1 9 D a y 2 8 1 0 5 0 1 0 0 S V P -R a p a m y c in (g ) 0


 
Spark Therapeutics License Agreement 30 • December 2016 agreement provides Spark Therapeutics with exclusive worldwide rights to Selecta's SVP technology for up to five gene therapy targets • Among the largest gene therapy and SMID-cap to SMID-cap biotech deals announced to date • Initial focus on combination of SVP with Spark’s Hemophilia A gene therapy • Received $30 million of initial cash payments and investments in Selecta equity • Subject to the terms of the license agreement, Spark also agreed to pay to Selecta: - Up to $430 million in milestone payments for each target - Mid-single to low-double-digit royalties on worldwide annual net sales of any resulting commercialized gene therapy


 
Pipeline 31 * LMB-100 is currently being investigated in two Phase 1 clinical trials at the National Cancer Institute (NCI): one of LMB-100 alone in Mesothelioma and one of LMB-100 in combination with nab-paclitaxel in Pancreatic Cancer. Phase 1 trial of SEL-403 in Mesothelioma is expected to begin in Q1 2018. Indication Preclinical Phase 1 Phase 2 Phase 3 Proprietary ADA Mitigation Programs Chronic Severe Gout SEL-212 Mesothelioma & Pancreatic Cancer SEL-403* Methylmalonic Acidemia (MMA) SEL-302 Ornithine Transcarbamylase Deficiency (OTC) SEL-313 ADA Mitigation Program License Hemophilia A


 
Financial Overview 32 For the Quarter Ended (In thousands, except share and per share data) September 30, 2017 September 30, 2016 Grant & Collaboration Revenue $27 $1,048 Research & Development Expenses 9,504 6,021 General & Administrative Expenses 4,377 2,495 Net Loss Attributable to Common Stockholders $(14,676) $(7,728) Net Loss Per Basic & Diluted Share $(0.66) $(0.43) Wtd. Avg. Common Shares Outstanding – Basic & Diluted 22,082,207 18,108,014 As of (In thousands) September 30, 2017 June 30, 2017 Cash, Cash Equivalents, Marketable Securities, Restricted Cash $104,780 $113,045 Cash runway into mid-2019


 
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