On March 11, 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 recent business highlights and financial results for the fourth quarter and year ended December 31, 2020 (Press release, Selecta Biosciences, MAR 11, 2021, View Source [SID1234576478]).
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"This is a very exciting time in Selecta’s history, having made significant progress across all aspects of the company," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "In 2020, we significantly de-risked the company through a strategic partnership with Sobi for SEL-212, under which we commenced the Phase 3 DISSOLVE program in the third quarter of 2020. We rapidly advanced our pipeline, having recently dosed our first subject with ImmTOR in combination with an AAV capsid, which builds on compelling non-human primate data announced earlier this year."
"We are very pleased by the continued progress of our gene therapy program. In collaboration with AskBio, our lead program in methylmalonic acidemia, is on track to enter the clinic in the second quarter of this year and we expect initial data by the end of the year. A key objective of 2021 will be to generate human data in our gene therapy programs and to continue to build our extensive pipeline in gene therapies, enzyme therapies—with an expected IND filing by the end of the year in IgA nephropathy—and autoimmune diseases, as we work to deliver on our mission to leverage our pioneering ImmTOR platform to improve the lives of patients and their families," Dr. Brunn added.
Recent Business Highlights and Anticipated Milestones
Enzyme Therapies:
SEL-212 for chronic refractory gout: The Phase 3 DISSOLVE clinical program for SEL-212 for the treatment of chronic refractory gout, which was licensed to Sobi, is progressing as planned with topline data expected in the second half of 2022.
The Phase 3 clinical program consists of two double blind, placebo-controlled trials of SEL-212 (DISSOLVE I and DISSOLVE II) (NCT04513366 and NCT04596540, respectively). Both studies have a 6-month primary endpoint of serum uric acid (SUA) < 6 mg/dL at month 6, and DISSOLVE I has a 6-month safety extension.
IgA nephropathy: Selecta’s second indication is IgA nephropathy, a kidney disease that occurs when immune complexes of an antibody called immunoglobulin A1 (IgA1) accumulates in the kidneys. Leveraging the learnings from SEL-212, Selecta will be researching a novel therapeutic approach by combining ImmTOR with an enzyme, IgA1 protease, which has been shown in animal studies to debulk the IgA1 immune complexes in the kidney, the root cause of IgA nephropathy. Selecta expects to file an Investigational New Drug, or IND, application, for this program by the end of 2021.
Gene Therapies:
MMA-101 for methylmalonic acidemia (MMA): Selecta’s lead gene therapy product candidate, MMA-101 combined with ImmTOR for the treatment of MMA, is expected to enter the clinic in the second quarter of 2021 with preliminary data expected by year-end. The MMA-101 program is being conducted in collaboration with AskBio.
Selecta and AskBio received Orphan Designation for MMA-101 from the U.S. Food and Drug Administration (FDA) in November 2020. MMA-101 previously received Rare Pediatric Disease Designation from the FDA in October 2020.
First-in-human trial of SEL-399: In collaboration with AskBio, Selecta recently 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 optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies. Selecta and AskBio expect to report initial data in the fourth quarter of 2021.
The dose-escalation trial of SEL-399 is designed to evaluate the safety and preliminary efficacy of ImmTOR in combination with an AAV capsid. Preliminary efficacy will be measured by assessing levels of AAV8-specific neutralizing antibodies.
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 disorder that causes ammonia to accumulate in the blood due to mutations in the OTC gene, which is critical for proper function of the urea cycle. SEL-313 is currently in preclinical development and is expected to enter the clinic in 2022. A Pediatric Investigation Plan (PIP) for SEL-313 was submitted to the European Medicines Agency (EMA) pediatric committee in February 2021.
Non-human primate data of ImmTOR in gene therapy: Selecta’s gene therapy programs build on extensive preclinical data that have demonstrated the potential benefits of the ImmTOR platform in AAV gene therapy. Selecta observed that co-administration of AAV vector and ImmTOR in non-human primates (NHP) enabled higher and more durable transgene expression as well as robust inhibition of anti-AAV8 immunoglobulin G (IgG) and neutralizing antibodies.
Restoring Self-Tolerance in Autoimmune Diseases:
Selecta continues IND-enabling work on an ImmTOR-based approach to treating primary biliary cholangitis (PBC), a chronic, progressive liver disorder that leads to inflammation, damage and scarring of the small bile ducts. PBC has a well-defined target antigen, significant unmet medical need, and is well suited to the application of our ImmTOR immune tolerance platform. Selecta expects to file an IND in PBC in 2022.
Fourth Quarter and Full Year 2020 Financial Results:
Cash Position: Selecta had $140.1 million in cash, cash equivalents, and restricted cash as of December 31, 2020, which compares to cash, cash equivalents, and restricted cash of $147.6 million as of September 30, 2020. Selecta believes its available cash, cash equivalents, and restricted cash will be sufficient to meet its operating requirements into the second quarter of 2023.
Revenue: Revenue recognition for the fourth quarter and fiscal year 2020 was $12.0 million and $16.6 million, respectively, which compares with $6.7 million and $6.7 million for the same periods in 2019.
Revenue was primarily driven by 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.
Research and Development Expenses: Research and development expenses for the fourth quarter and fiscal year 2020 were $15.1 million and $54.5 million, respectively, which compares with $15.2 million and $42.7 million for the same periods in 2019.
During the quarter ended December 31, 2020, there was a reduction in expenses for the SEL-212 clinical programs due to the timing of the initiation of the Phase 3 DISSOLVE clinical program compared to the Phase 2 COMPARE program in the prior period. This reduction was offset by increases in expenses incurred under the AskBio Collaboration combined with internal research and development to support our clinical programs. The annual increase reflects the initiation of the Phase 3 DISSOLVE clinical program. These costs are subject to the cost reimbursement arrangement under the license agreement with Sobi.
General and Administrative Expenses: General and administrative expenses for the fourth quarter and fiscal year 2020 were $4.8 million and $18.9 million, respectively, which compares with $4.1 million and $16.4 million for the same period in 2019. The quarterly and annual increase in expense was the result of increased patent and professional fees and facility and office expenses offset by a decrease in travel expense.
Net Loss: For the fourth quarter and fiscal year 2020, Selecta reported a net loss of $15.4 million, or $0.14 per share and $68.9 million, or $0.68 per share, respectively, compared to a net loss of $14.9 million, or $0.28 per share and $55.4 million, or $1.22 per share, for the same periods in 2019.
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 fourth quarter 2020 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 10147796. 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.