Pegzilarginase BLA submitted to the Fda if accredited, pegzilarginase would be the very first Food and drug administration-authorized remedy for Arginase 1 Deficiency
Additional PEACE data supporting potential efficacy of pegzilarginase introduced at SIMD
Enrollment proceeds in Phase 1/2 demo of AGLE-177 IND now open for U.S. web site initiation
AUSTIN, Texas, Might 5, 2022 /PRNewswire/ — Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a scientific-phase biotechnology firm acquiring a new generation of human enzyme therapeutics as innovative remedies for uncommon metabolic diseases, today introduced fiscal effects for the initial quarter 2022 and provided application updates.
“We have commenced off 2022 with important development in both of those our scientific systems. With AGLE-177, we have been happy to see full homocysteine decreasing in all patients in our to start with, lower dose cohort and we appear ahead to sharing medical knowledge afterwards this calendar year. This method is yet another instance of innovation with human enzyme therapies and has the possible to tackle a sizeable unmet will need,” claimed Anthony Quinn, M.B., Ch.B., Ph. D., president and main government officer of Aeglea. “I am also very pleased of the operate we have completed with our pegzilarginase system enabling our new BLA submission to the Fda with what we think are incredibly powerful data to assistance approval for the cure of Arginase 1 Deficiency. Some of these info were being introduced not long ago at the SIMD Yearly Meeting, furnishing further perception into the probable optimistic influence of pegzilarginase therapy. We seem ahead to doing work with the Fda all through the review of our BLA as they evaluate the likely efficiency of pegzilarginase in addressing the unmet have to have that impacts the lives of Arginase 1 Deficiency people and their people.”
System Updates
Pegzilarginase in Arginase 1 Deficiency
- Submitted a Biologics License Software (BLA) to the U.S. Foodstuff and Drug Administration (Fda) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D).
- Asked for Priority Assessment at the time of submission.
- Introduced new facts from the PEACE Section 3 scientific trial at the Culture for Inherited Metabolic Issues (SIMD) Once-a-year Conference, such as client-amount results, final results from extra secondary endpoints and earlier declared topline effects. Highlights of the data presented consist of:
- 76.7% reduction in mean plasma arginine in pegzilarginase treated clients when compared to placebo (p<0.0001) normal plasma arginine levels achieved in 90.5% of pegzilarginase treated patients compared to no patients receiving placebo.
- Eleven patients (65%) treated with pegzilarginase reached or exceeded response criteria for ≥1 mobility assessment compared to four patients (44%) receiving placebo.
- Eight patients (47%) treated with pegzilarginase met or exceeded prespecified clinical response criteria for ≥2 mobility assessments compared to no patients receiving placebo.
- In a post hoc analysis correcting for a missed assessment that was improperly scored as 0 rather than “not assessed,” the least squares mean Gross Motor Function Measure Part D score of patients treated with pegzilarginase improved from baseline by 2.25 units compared to placebo (p=0.0896).
- Pegzilarginase treated patients showed statistically significant biochemical improvements in measures of ornithine and guanidino compounds compared to placebo.
- On track to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in 2022.
AGLE-177 in Homocystinuria
- Working to initiate U.S. sites to support enrollment in the ongoing Phase 1/2 clinical trial and a potential pivotal Phase 3 trial after opening an Investigational New Drug (IND) application with the FDA.
- Expect to announce Phase 1/2 clinical data in the second half of 2022.
First Quarter 2022 Financial Results
As of March 31, 2022, Aeglea had available cash, cash equivalents, marketable securities and restricted cash of $68.6 million. The Company expects its cash, cash equivalents and investments will enable it to fund its operating expenses and capital expenditure requirements into the first quarter of 2023.
Aeglea recognized development fee revenues of $1.4 million in the first quarter of 2022, as a result of its license and supply agreement with Immedica for the commercial rights to pegzilarginase in certain territories outside the U.S. The revenues recorded in the first quarter of 2022 are related to the PEACE Phase 3 trial and BLA package. Aeglea recognized no revenue for the first quarter of 2021.
Research and development expenses totaled $17.0 million for the first quarter of 2022 and $11.9 million for the first quarter of 2021. The increase was primarily associated with expenses related to the BLA submission, management and dosing of ongoing patients in the PEACE Phase 3 trial, and increased enrollment in our Phase 1/2 trial of AGLE-177 for the treatment of patients with Homocystinuria.
General and administrative expenses totaled $8.8 million for the first quarter of 2022 and $6.4 million for the first quarter of 2021. This increase was primarily due to ramping-up the Company’s commercial capabilities and infrastructure as well as personnel expenses.
Net loss totaled $24.4 million and $18.2 million for the first quarter of 2022 and 2021, respectively, with non-cash stock compensation expense of $2.1 million and $1.8 million for the first quarter of 2022 and 2021, respectively.
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 76.7% reduction in mean plasma arginine compared to placebo. Additionally, 90.5% of pegzilarginase treated patients achieved normal plasma arginine levels. The arginine lowering was accompanied by a positive trend in Gross Motor Function Measure Part E, a measure of patient mobility. Aeglea’s Phase 1/2 and Phase 2 Open-Label Extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.
About AGLE-177 in Homocystinuria
AGLE-177 is a novel recombinant human enzyme, which is engineered to degrade the amino acid homocysteine and its dimer, homocystine. AGLE-177 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 thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Preclinical data demonstrated that AGLE-177 improved important disease-related abnormalities and survival in a mouse model of Homocystinuria. AGLE-177 has received both U.S. and EU Orphan Drug Designation as well as U.S. Rare Pediatric Disease Designation.
About Aeglea BioTherapeutics
Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. In April 2022, Aeglea announced it had submitted a Biologics License Application to the FDA for its lead product candidate, pegzilarginase, in patients with Arginase 1 Deficiency. Pegzilarginase achieved the primary endpoint of arginine reduction in the PEACE Phase 3 clinical trial and has received both Rare Pediatric Disease and Breakthrough Therapy Designations. Aeglea also has an ongoing Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria. AGLE-177 has been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families. For more information, please visit https://aeglea.com.
Safe Harbor / Forward Looking Statements
This press release contains “forward-looking” statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “anticipate,” “intend,” “plan,” “goal,” “seek,” “believe,” “project,” “estimate,” “expect,” “strategy,” “future,” “likely,” “may,” “should,” “will” and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, including the FDA’s review of our BLA for pegzilarginase and the timing of the MAA for pegzilarginase, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, the length of time that we believe our existing cash resources will fund operations, the potential addressable markets of our product candidates and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 filed with the Securities and Exchange Commission (SEC), and our other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
Financials
| Aeglea BioTherapeutics, Inc. | ||||
| Consolidated Balance Sheets | ||||
| (In thousands, except share and per share amounts) | ||||
| March 31, | December 31, | |||
| 2022 | 2021 | |||
| ASSETS | ||||
| CURRENT ASSETS | ||||
| Cash and cash equivalents | $ 14,637 | $ 15,142 | ||
| Marketable securities | 52,088 | 77,986 | ||
| License and development receivables | 1,012 | 815 | ||
| Prepaid expenses and other current assets | 4,859 | 4,948 | ||
| Total current assets | 72,596 | 98,891 | ||
| Restricted cash | 1,828 | 1,838 | ||
| Property and equipment, net | 4,234 | 4,549 | ||
| Operating lease right-of-use assets | 3,712 | 3,806 | ||
| Other non-current assets | 800 | 842 | ||
| TOTAL ASSETS | $ 83,170 | $ 109,926 | ||
| LIABILITIES AND STOCKHOLDERS’ EQUITY | ||||
| CURRENT LIABILITIES | ||||
| Accounts payable | $ 2,981 | $ 3,319 | ||
| Operating lease liabilities | 457 | 436 | ||
| Deferred revenue | 2,069 | 2,359 | ||
| Accrued and other current liabilities | 10,791 | 14,030 | ||
| Total current liabilities | 16,298 | 20,144 | ||
| Non-current operating lease liabilities | 4,467 | 4,608 | ||
| Deferred revenue, net of current portion | 740 | 1,217 | ||
| Other non-current liabilities | 8 | 16 | ||
| TOTAL LIABILITIES | 21,513 | 25,985 | ||
| STOCKHOLDERS’ EQUITY | ||||
| Preferred stock, $0.0001 par value 10,000,000 shares authorized as of March | — | — | ||
| Common stock, $0.0001 par value 500,000,000 shares authorized as of March | 5 | 5 | ||
| Additional paid-in capital | 428,050 | 425,765 | ||
| Accumulated other comprehensive loss | (153) | (20) | ||
| Accumulated deficit | (366,245) | (341,809) | ||
| TOTAL STOCKHOLDERS’ EQUITY | 61,657 | 83,941 | ||
| TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY | $ 83,170 | $ 109,926 | ||
| Aeglea BioTherapeutics, Inc. | ||||
| Consolidated Statements of Operations | ||||
| (In thousands, except share and per share amounts) | ||||
| Three Months Ended March 31, |
||||
| 2022 | 2021 | |||
| Revenue: | ||||
| Development fee | $ 1,362 | $ — | ||
| Total revenue | 1,362 | — | ||
| Operating expenses: | ||||
| Research and development | 16,978 | 11,855 | ||
| General and administrative | 8,825 | 6,354 | ||
| Total operating expenses | 25,803 | 18,209 | ||
| Loss from operations | (24,441) | (18,209) | ||
| Other income (expense): | ||||
| Interest income | 35 | 22 | ||
| Other income (expense), net | (30) | (31) | ||
| Total other income (expense) | 5 | (9) | ||
| Net loss | $ (24,436) | $ (18,218) | ||
| Net loss per share, basic and diluted | $ (0.37) | $ (0.28) | ||
| Weighted-average common shares outstanding, basic and diluted | 65,996,161 | 65,604,336 | ||
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SOURCE Aeglea BioTherapeutics, Inc.