Aeglea BioTherapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Program Updates
Aeglea BioTherapeutics reported its financial results for Q4 and full year 2022, showing a net loss of $18.8 million for Q4, down from $20.4 million in Q4 2021. The full-year net loss increased to $83.8 million compared to $65.8 million in 2021. As of December 31, 2022, the company had $57.3 million in cash and equivalents, expected to fund operations into Q4 2023. Highlights include the appointment of Jeffrey Goldberg as president and CEO and advancements in clinical trials for pegtarviliase and pegzilarginase, with EMA review for pegzilarginase expected in late 2023.
- Received Fast Track Designation from the FDA for pegtarviliase.
- Strong cash position of $57.3 million to fund operations into Q4 2023.
- Advancing pegzilarginase's EMA Marketing Authorization Application.
- Net loss increased to $83.8 million for FY 2022, up from $65.8 million in 2021.
- Revenue from development fees decreased significantly from $18.7 million in 2021 to $2.3 million in 2022.
- Research and development expenses increased to $58.6 million for FY 2022, reflecting ongoing trial activities.
"Since joining the company, I have been impressed by the programs and the potential impact that they may have on patient lives. In 2023, we are highly focused on execution, specifically the generation of data from our pegtarviliase Phase 1/2 clinical trial in Classical Homocystinuria and, assuming success with those data, preparations to advance into a potential pivotal trial," said
Program and Corporate Updates
Pegtarviliase in Homocystinuria
- Received Fast Track Designation from the
U.S. Food and Drug Administration (FDA). Fast Track Designation is designed to expedite theFDA's review of innovative, new drugs that demonstrate the potential to address unmet medical need. - Announced in late
November 2022 the completion of dosing of two patients in the third cohort of the Phase 1/2 clinical trial. Patients in the third cohort receive 1.35 mg/kg of pegtarviliase administered subcutaneously once weekly.
Pegzilarginase in Arginase 1 Deficiency
- Transitioning patients who participated in the PEACE Phase 3 long-term extension (LTE) and Phase 1/2 open-label extension (OLE) studies into one simplified open-label trial. The PEACE LTE and Phase 1/2 OLE studies were closed in the first quarter of 2023.
- Continuing to progress the review of the Marketing Authorization Application submission with the EMA for the potential approval of pegzilarginase. EMA approval can facilitate possible patient access and regulatory approval in many other geographies, including
Latin America and theMiddle East .
Corporate
- Appointed
Jeffrey Goldberg , a highly experienced executive with strong operational and rare disease background, as president and chief executive officer. - Strengthened senior management team with the promotions of
Linda Neuman , MD, MBA, to chief medical officer andCortney Caudill , MBA, to the newly created role of chief product officer.Ms. Caudill will serve as a strategic leader and team supervisor of Aeglea's clinical programs. - Implemented changes to corporate structure to increase operational efficiency. Additionally, the company halted preclinical work on Cystinuria and other unnamed pipeline programs. The company will evaluate potential strategic options for these programs in order to maximize their value.
Upcoming Events
- ACMG Annual Clinical Genetics Meeting,
Salt Lake City, Utah - Long-Term Efficacy and Safety of Pegzilarginase for Arginase 1 Deficiency: 2 Years of Experience in the Phase 2 Extension Study
- Featured platform presentation on
March 16, 2023 at8:45-9:00 am MST
Fourth Quarter 2022 Financial Results
Aeglea recognized development fee revenues of
Research and development expenses totaled
General and administrative expenses totaled
Net loss totaled
Full Year 2022 Financial Results
As of
Aeglea recognized development fee revenues of
Research and development expenses totaled
General and administrative expenses totaled
Net loss totaled
About Pegtarviliase in Homocystinuria
Pegtarviliase is a novel recombinant human enzyme, which is engineered to reduce elevated levels of total homocysteine circulating in the plasma by degrading the amino acid homocysteine and its dimer. Pegtarviliase is currently being studied in a Phase 1/2 clinical trial for the treatment of patients with Classical Homocystinuria, a rare inherited disorder of methionine metabolism that results in elevated levels of total homocysteine. Homocysteine accumulation plays a key role in multiple progressive and serious disease-related complications, including lens dislocation, skeletal abnormalities, vascular complications and neurologic effects, as well as the potential for sudden and early death. In preclinical studies, pegtarviliase improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. Pegtarviliase has received both
About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is a novel recombinant human enzyme engineered to degrade the amino acid arginine and has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of people with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality. The PEACE Phase 3 clinical trial met its primary endpoint with a
About
Safe Harbor / Forward Looking Statements
This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the
Financials
Consolidated Balance Sheets (Unaudited, in thousands, except share and per share amounts) | ||||||||
2022 | 2021 | |||||||
ASSETS | ||||||||
CURRENT ASSETS | ||||||||
Cash and cash equivalents | $ | 34,863 | $ | 15,142 | ||||
Marketable securities | 20,848 | 77,986 | ||||||
Development receivable | 375 | 815 | ||||||
Prepaid expenses and other current assets | 6,172 | 4,948 | ||||||
Total current assets | 62,258 | 98,891 | ||||||
Restricted cash | 1,553 | 1,838 | ||||||
Property and equipment, net | 3,220 | 4,549 | ||||||
Operating lease right-of-use assets | 3,430 | 3,806 | ||||||
Other non-current assets | 683 | 842 | ||||||
TOTAL ASSETS | $ | 71,144 | $ | 109,926 | ||||
LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
CURRENT LIABILITIES | ||||||||
Accounts payable | $ | 677 | $ | 3,319 | ||||
Operating lease liabilities | 625 | 436 | ||||||
Deferred revenue | 517 | 2,359 | ||||||
Accrued and other current liabilities | 12,837 | 14,030 | ||||||
Total current liabilities | 14,656 | 20,144 | ||||||
Non-current operating lease liabilities | 4,004 | 4,608 | ||||||
Deferred revenue, net of current portion | 2,179 | 1,217 | ||||||
Other non-current liabilities | — | 16 | ||||||
TOTAL LIABILITIES | 20,839 | 25,985 | ||||||
Commitments and Contingencies (Note 9) | ||||||||
STOCKHOLDERS' EQUITY | ||||||||
Preferred stock, | — | — | ||||||
Common stock, | 6 | 5 | ||||||
Additional paid-in capital | 475,971 | 425,765 | ||||||
Accumulated other comprehensive (loss) income | (48) | (20) | ||||||
Accumulated deficit | (425,624) | (341,809) | ||||||
TOTAL STOCKHOLDERS' EQUITY | 50,305 | 83,941 | ||||||
TOTAL LIABILITIES AND STOCKHOLDERS' EQUITY | $ | 71,144 | $ | 109,926 | ||||
Consolidated Statements of Operations (Unaudited, in thousands, except share and per share amounts) | ||||||||||||
Year Ended | ||||||||||||
2022 | 2021 | 2020 | ||||||||||
Revenue: | ||||||||||||
License | $ | — | $ | 12,000 | $ | — | ||||||
Development fee | 2,329 | 6,739 | — | |||||||||
Total revenue | 2,329 | 18,739 | — | |||||||||
Operating expenses: | ||||||||||||
Research and development | 58,579 | 57,069 | 59,638 | |||||||||
General and administrative | 28,531 | 27,319 | 21,843 | |||||||||
Total operating expenses | 87,110 | 84,388 | 81,481 | |||||||||
Loss from operations | (84,781) | (65,649) | (81,481) | |||||||||
Other income (expense): | ||||||||||||
Interest income | 837 | 111 | 593 | |||||||||
Other expense, net | (7) | (122) | (5) | |||||||||
Total other income (expense) | 830 | (11) | 588 | |||||||||
Loss before income tax expense | (83,951) | (65,660) | (80,893) | |||||||||
Income tax benefit (expense) | 136 | (141) | — | |||||||||
Net loss | $ | (83,815) | $ | (65,801) | $ | (80,893) | ||||||
Net loss per share, basic and diluted | $ | (0.99) | $ | (1.00) | $ | (1.52) | ||||||
Weighted-average common shares outstanding, basic and diluted | 84,280,785 | 65,744,611 | 53,371,730 | |||||||||
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FAQ
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