Selecta Biosciences Reports Second Quarter 2021 Financial Results and Provides Business Update
- IND filing for wholly owned gene therapy program, SEL-302 (MMA-101, in combination with ImmTOR) in methylmalonic acidemia (MMA) expected during the third quarter of 2021; Manufacturing issue has been resolved, with a new completed lot undergoing final release testing
- SEL-399 (empty AAV8 capsid with ImmTOR) study on track with topline data expected in the fourth quarter of 2021
Selectaachieved $3 millionmilestone payment from Sarepta Therapeutics related to successfully meeting the criteria for a preclinical study
- As of
June 30, 2021, Selectahad approximately $151.5 millionin cash, cash equivalents, and marketable securities which is expected to provide runway into the third quarter of 2023
“We are very pleased about our continued progress across all aspects of the company,” said
Recent Highlights and Anticipated Upcoming Milestones:
- 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.
Selectaexpects to file an IND in IgA nephropathy in 2022 and will provide additional updates later in the year.
- First-in-human trial of SEL-399: In collaboration with AskBio,
Selectainitiated 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):
Selectaannounced 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.
Selectaexpects 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:
Selectahas achieved a $3 millionmilestone payment related to the completion of a preclinical study under the Research License and Option Agreement.
Restoring Self-Tolerance in Autoimmune Diseases:
Selectaannounced 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). Selectacontinues 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.
Jude Samulski, Ph.D., was appointed as a special advisor to help guide Selecta’s gene therapy programs into the clinic. Dr. Samulskiis a professor of pharmacology and has been the director of the University of North Carolina Gene Therapy Centerfor 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 Awardfor lifetime achievements in gene therapy. Dr. Samulskihas 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 Silvahas extensive leadership experience, most relevantly in gene therapy development, manufacturing, and regulatory activities. Dr. de Silvabrings 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 Silvaserved 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:
- Net cash used in operating activities was
$18.2 millionfor the six months ended June 30, 2021, as compared to $23.5 millionfor the same period in 2020.
Revenue: Revenue recognition for the second quarter of 2021 was
Research and Development Expenses: Research and development expenses for the second quarter 2021 were
General and Administrative Expenses: General and administrative expenses for the second quarter 2021 were
Net Income (loss): For the second quarter 2021,
Conference Call and Webcast Reminder:
Selecta Forward-Looking Statements
Any statements in this press release about the future expectations, plans and prospects of
Consolidated Balance Sheets
(Amounts in thousands, except share data and par value)
|Cash and cash equivalents||$||125,749||$||138,685|
|Prepaid expenses and other current assets||8,210||5,434|
|Total current assets||166,812||151,343|
|Property and equipment, net||1,778||1,395|
|Right-of-use asset, net||10,399||10,948|
|Long-term restricted cash||1,379||1,379|
|Liabilities and stockholders’ (deficit) equity|
|Total current liabilities||87,296||81,547|
|Loan payable, net of current portion||22,931||24,793|
|Stockholders’ (deficit) equity:|
|Additional paid-in capital||424,984||391,175|
|Accumulated other comprehensive loss||(4,557||)||(4,563||)|
|Total stockholders’ (deficit) equity||(4,222||)||(18,006||)|
|Total liabilities and stockholders’ (deficit) equity||$||180,522||$||165,435|
Consolidated Statements of Operations and Comprehensive Income (Loss)
(Amounts in thousands, except share and per share data)
|Three Months Ended
||Six Months Ended
|Grant and collaboration revenue||$||19,663||$||—||$||30,713||$||—|
|Research and development||14,463||10,730||27,467||25,454|
|General and administrative||4,748||5,637||9,952||9,735|
|Total operating expenses||19,211||16,367||37,419||35,189|
|Operating income (loss)||452||(16,367||)||(6,706||)||(35,189||)|
|Foreign currency transaction, net||(14||)||(42||)||(7||)||40|
|Change in fair value of warrant liabilities||4,820||(7,539||)||(11,927||)||(8,385||)|
|Other income, net||6||59||6||58|
|Net income (loss)||4,565||(24,081||)||(20,032||)||(43,701||)|
|Other comprehensive income (loss):|
|Foreign currency translation adjustment||12||31||6||(29||)|
|Unrealized gains on marketable securities||1||—||—||—|
|Total comprehensive income (loss)||$||4,578||$||(24,050||)||$||(20,026||)||$||(43,730||)|
|Net income (loss) per share:|
|Weighted average common shares outstanding:|
Source: Selecta Biosciences, Inc.