On August 12, 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 second quarter ended June 30, 2021 and provided a business update (Press release, Selecta Biosciences, AUG 12, 2021, View Source [SID1234586413]).

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"We are very pleased about our continued progress across all aspects of the company," said Carsten Brunn, Ph.D., president, and chief executive officer of Selecta. "Building on our ongoing empty AAV8 capsid study, we are rapidly advancing our two proprietary gene therapy programs into the clinic and as we enter a critical inflection point in development, we are honored to have gene therapy pioneer, Jude Samulski, Ph.D., join as a special advisor. The recently published preclinical data is encouraging and further supports the advancement of our lead candidate in methylmalonic acidemia (MMA), SEL-302. We will build on this momentum and expect to file an IND in MMA during the third quarter of 2021, bringing us one step closer to addressing immunogenicity constraints in AAV-driven gene therapy and ultimately, providing patients with potentially transformative treatment options. Additionally, we are steadily executing across our enzyme and autoimmune development program. We have a well-defined work plan ahead of us and the financial resources to maximize the value of our innovative ImmTOR platform."

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.
Investigational New Drug, or IND, enabling studies are underway for a novel therapeutic approach that combines ImmTOR with an enzyme, IgA1 protease for the treatment of IgA nephropathy.
Selecta expects to file an IND in IgA nephropathy in 2022 and will provide additional updates later in the year.
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 trial aims to determine the dose regimen of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.
Topline data is expected in the fourth quarter of 2021.
SEL-302 for methylmalonic acidemia (MMA): Selecta announced publication in the journal Molecular Therapy Methods & Clinical Development demonstrating that ImmTOR enhances transgene expression after both initial and repeat dosing of AAV in a mouse model of MMA. The publication further validates use of ImmTOR in Selecta’s gene therapy pipeline, including its lead candidate, 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.
The previously disclosed MMA-101 manufacturing issue was resolved. Manufacturing of a new lot has been completed and is currently undergoing final release testing.
Selecta expects to file an IND for SEL-302 during the third quarter of 2021.
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.
Sarepta Therapeutics program in Duchenne Muscular Dystrophy (DMD) and certain Limb-Girdle Muscular Dystrophies (LGMD) subtypes: Selecta has achieved a $3 million milestone payment related to the completion of a preclinical study under the Research License and Option Agreement.
Restoring Self-Tolerance in Autoimmune Diseases:

Selecta announced Frontiers in Immunology publication showcasing the enhanced hepatic tolerogenic potential of ImmTOR. Data demonstrate that ImmTOR enhances the tolerogenic environment in the liver, shows induction of a tolerogenic phenotype in all major hepatic antigen presenting cell populations and is protective in an acute model of autoimmune hepatitis. The publication further supports development of Selecta’s ImmTOR platform for the treatment of liver-specific autoimmune diseases, including primary biliary cholangitis (PBC).
Selecta continues IND-enabling work on an ImmTOR-based approach to treating PBC and expects to file an IND in PBC in the second half of 2022.
Corporate Updates:

Jude Samulski, Ph.D., was appointed as a special advisor to help guide Selecta’s gene therapy programs into the clinic. Dr. Samulski is a professor of pharmacology and has been the director of the University of North Carolina Gene Therapy Center for over two decades. He was awarded the first patent for AAV as a viral vector and was the first recipient of the American Society of Gene & Cell Therapy Outstanding Achievement Award for lifetime achievements in gene therapy. Dr. Samulski has advanced gene therapies into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and heart failure, and is the president, chief scientific officer and co-founder of Asklepios BioPharmaceutical Inc. (AskBio), a biotechnology company focused on AAV-driven gene therapy.

Nishan de Silva, M.D. was appointed to Selecta’s Board of Directors. Dr. de Silva has extensive leadership experience, most relevantly in gene therapy development, manufacturing, and regulatory activities. Dr. de Silva brings over 20 years of experience in biotechnology operations, biopharmaceutical venture capital and healthcare management consulting. He is currently chief executive officer and director of AFYX Therapeutics, a private venture-backed biotechnology company focused on addressing unmet needs in mucosal diseases. Previously Dr. de Silva served as president, chief operating officer and director of Poseida Therapeutics, a cell and gene therapy-focused biopharmaceutical company, where he oversaw clinical development, regulatory, manufacturing, finance, and business development activities.
Second Quarter 2021 Financial Results:

Cash Position: Selecta had $151.5 million in cash, cash equivalents, marketable securities, and restricted cash as of June 30, 2021, which compares to cash, cash equivalents, and restricted cash of $149.2 million as of March 31, 2021. Selecta believes its available cash, cash equivalents, marketable securities, and restricted cash will be sufficient to meet its operating requirements into the third quarter of 2023.

Net cash used in operating activities was $18.2 million for the six months ended June 30, 2021, as compared to $23.5 million for the same period in 2020.
Revenue: Revenue recognition for the second quarter of 2021 was $19.7 million, compared to no revenue recognition for the same period in 2020. Revenue was recognized under the license agreement with Sobi which began in July 2020 resulting from the shipment of clinical supply and the reimbursement of costs incurred for the Phase 3 DISSOLVE clinical program. Additionally, during the second quarter, Selecta recognized less than $0.1 million for shipments under the license agreement with Sarepta and $0.1 million resulting from the expiration of the contractual audit term under the Skolkovo Foundation grant.

Research and Development Expenses: Research and development expenses for the second quarter 2021 were $14.5 million, which compares with $10.7 million for the same period in 2020. During the quarter ended June 30, 2021, there was an increase in expenses incurred for consulting, salaries, and the discovery and preclinical programs, offset by a decrease of AskBio collaboration costs.

General and Administrative Expenses: General and administrative expenses for the second quarter 2021 were $4.7 million, which compares with $5.6 million for the same period in 2020. The decrease in costs was primarily the result of reduced expense for salaries, professional fees and patent expense, offset by increased consulting and stock compensation expenses.

Net Income (loss): For the second quarter 2021, Selecta reported net income of $4.6 million, or basic net income per share of $0.04, compared to a net loss of $24.1 million, or basic net loss per share of $0.25 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 second 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 10147802. 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.