On November 9, 2021 Selecta Biosciences, Inc. (NASDAQ: SELB), a biotechnology company leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses, reported financial results for the third quarter ended September 30, 2021 and provided a business update (Press release, Selecta Biosciences, NOV 9, 2021, View Source [SID1234594803]).

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"The third quarter of 2021 was filled with many significant advancements that further validated the value and breadth of our innovative ImmTOR platform, including important milestones across our pipeline and the establishment of numerous collaborations that will continue to propel us forward in the year ahead," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "We recently presented topline data from the empty AAV8 capsid study, SEL-399 in healthy volunteers. We believe this pioneering proof-of-concept study showed for the first time ImmTOR’s potential to address one of the biggest limitations in gene therapy: preventing the formation of anti-AAV8 neutralizing antibodies after ImmTOR treatment with AAVs and enabling re-dosing of life-saving gene therapies. We are excited to build on these exciting findings, which will inform our clinical development strategy and bring hope to those patients suffering from monogenic disease. We continue to prosecute our clinical pipeline, execute value accretive business development with world class partners and add talent and depth to our leadership team."

Recent Highlights and Anticipated Upcoming Milestones:
Enzyme Therapies:

SEL-212 for chronic refractory gout: Enrollment for the Phase 3 DISSOLVE clinical program for the treatment of chronic refractory gout, which was licensed to Sobi, is progressing as planned.
Topline data is expected in the second half of 2022.
ImmTOR with IgA1 protease for IgA nephropathy: Immunoglobulin A nephropathy (IgAN) is a leading cause of chronic kidney disease (CKD) and renal failure. Current treatments fail to address the root cause of the disease.
Investigational New Drug, or IND, enabling studies are underway. Selecta expects to file an IND in IgA nephropathy in 2022 and will provide additional updates.
Gene Therapies:

First-in-human trial of SEL-399: In collaboration with AskBio, Selecta initiated the first-in-human, dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The key objective was to determine the dose regimen of ImmTOR to effectively mitigate the formation of antibodies to AAV8 capsids used in gene therapies. Data was released on November 8, 2021. Key findings included:
No Serious Adverse Events were reported. All treatment-related adverse events were expected for ImmTOR, readily monitorable, and transient
At 30 days 6 of 6 or 100% of subjects that received 0.3 mg/kg of ImmTOR exhibited an anti-AAV8 neutralizing antibody titer of 1:25 or less. 4 of 6 or 67% of subjects at this dose had a titer of 1:5 or less
At 30 days 6 of 9 or 67% of subjects that received 0.15 mg/kg of ImmTOR exhibited an anti-AAV8 neutralizing antibody titer of 1:25 or less. 2 of 9 or 22% of subjects at this dose had a titer of 1:5 or less
At 90 days 2 of 6 subjects in the 0.3 mg/kg cohort were observed to have sustained control of neutralizing antibodies with titers of 1:25 or less
Consistent with preclinical data, we observed that the single dose ImmTOR cohorts saw delayed formation of neutralizing antibodies eventually reaching similar median levels of neutralizing antibodies to the control group by day 90
While most subjects treated with ImmTOR showed increases in antibody titers by Day 90, preclinical studies in mice and nonhuman primates indicate that if antibodies can be controlled at Day 30, we can maintain control with additional two monthly doses of ImmTOR.

SEL-302 for methylmalonic acidemia (MMA): IND filed for SEL-302 (MMA-101 in combination with ImmTOR), for the treatment of MMA, a rare metabolic disease in which the body cannot break down certain proteins and fats.
IND filed in Q3 2021, 30-day FDA review period has expired.
FDA has orally communicated they are still considering certain aspects of the submission related to chemistry, manufacturing and control, or CMC.
Formal FDA decision on IND expected by the end of November.
SEL-313 for ornithine transcarbamylase deficiency (OTC deficiency): Selecta’s proprietary gene therapy product candidate, SEL-313, is being developed to treat OTC deficiency, a rare genetic urea cycle disorder that causes ammonia to accumulate in the blood due to mutations in the OTC gene.
SEL-313 is currently in preclinical development and a clinical trial application, or CTA, and/or IND filing are expected in 2022.
Restoring Self-Tolerance in Autoimmune Diseases:

Selecta continues IND-enabling work on an ImmTOR-based approach to treating primary biliary cholangitis (PBC) and expects to file an IND in PBC in the second half of 2022.
Corporate Updates:

Strategic Collaborations:
Gene Therapies
Takeda Pharmaceutical Company Limited ("Takeda"): Strategic licensing agreement to develop next-generation gene therapies in two lysosomal storage disorders. The collaboration leverages Selecta ImmTOR platform to enable redosing of transformative therapies.
Genovis: Strategic licensing agreement to advance a next-generation IgG protease. This partnership leverages Genovis’ proprietary and differentiated immunoglobulin G (IgG) protease, IdeXork (Xork), and Selecta’s ImmTOR platform to enable the dosing of transformative gene therapies in patients with pre-existing adeno-associated virus (AAV) immunity and treat certain IgG-mediated autoimmune diseases. Xork shows low crossreactivity to pre-existing antibodies in human sera. The combination of Xork with ImmTOR has the potential to address two of the biggest immunological challenges to gene therapy – expanding access to gene therapies by overcoming pre-existing antibodies to AAV and enabling vector re-dosing.
Enzymes
Ginkgo Bioworks ("Ginkgo"): Partnership to design novel enzymes and proteins with transformative therapeutic potential to advance treatments for orphan and rare diseases. This partnership leverages Ginkgo’s cell programing platform and Selecta’s ImmTOR platform to create transformative biologic and enzymatic therapies.
Autoimmune
Cyrus Biotechnology ("Cyrus"): Established protein engineering collaboration combining Selecta’s ImmTOR platform with Cyrus’ ability to radically redesign protein therapeutics. The lead program in the collaboration is a proprietary interleukin-2 (IL-2) protein agonist designed to selectively promote expansion of regulatory T cells (Treg) for the treatment of patients with autoimmune diseases and other deleterious immune conditions. The combination of ImmTOR with a Treg-selective IL-2 agonist has the potential to be a best-in-class therapeutic profile.
Leadership Expansion:
Kevin Tan was appointed as chief financial officer. Mr. Tan brings deep financial expertise and experience in the gene therapy and rare disease landscape. His impressive track record in capital management and financings, in both the biotech and investment sectors, will be invaluable as Selecta continues to pursue new partnership opportunities and advance multiple assets through the clinic.
Matthew Bartholomae was appointed as General Counsel, Board Secretary and transitioned from his previous role at Selecta as Associate General Counsel, Board Secretary.
Third Quarter 2021 Financial Results:

Cash Position: Selecta had $140.0 million in cash, cash equivalents, marketable securities, and restricted cash as of September 30, 2021, which compares to cash, cash equivalents, and restricted cash of $140.1 million as of December 31, 2020. Selecta believes its available cash, cash equivalents, marketable securities, and restricted cash will be sufficient to meet its operating requirements into the second quarter of 2023.

Net cash used in operating activities was $28.9 million for the nine months ended September 30, 2021, as compared to $42.1 million of cash provided by operating activities for the same period in 2020.
Revenue: Revenue for the third quarter of 2021 was $24.4 million, compared to $4.6 million for the same period in 2020. Revenue of $24.3 million was recognized under the license agreement with Sobi resulting from the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program. The significant revenue increase is the result of the continued enrollment of the Phase 3 DISSOLVE clinical program that was initiated in the third quarter of 2020. Additionally, during the third quarter, Selecta recognized $0.2 million for shipments under the license agreement with Sarepta.

Research and Development Expenses: Research and development expenses for the third quarter 2021 were $21.0 million, which compares with $14.0 million for the same period in 2020. During the quarter ended September 30, 2021, there was an increase in expenses incurred for preclinical programs, salaries, headcount, and AskBio collaboration costs.

General and Administrative Expenses: General and administrative expenses for the third quarter 2021 were $5.4 million, which compares with $4.4 million for the same period in 2020. The increase in costs was primarily the result of salaries, professional fees, and stock compensation expenses.

Net loss: For the third quarter 2021, Selecta reported net loss of $17.9 million, or net loss per share of $0.16, compared to a net loss of $9.7 million, or net loss per share of $0.09 for the same period in 2020.

Conference Call and Webcast Reminder
Selecta management will host a conference call at 8:30 AM ET today to provide a corporate update and review the company’s third quarter 2021 financial results. Individuals may participate in the live call via telephone by dialing (844) 845-4170 (domestic) or (412) 717-9621 (international) and may access a teleconference replay for one week by dialing (877) 344-7529 (domestic) or (412) 317-0088 (international) and using confirmation code 10147803. Investors and the public can access the live and archived webcast of this call and a copy of the presentation via the Investors & Media section of the company’s website, www.selectabio.com.