On Track to Dose First Patient in Pivotal Phase 3 Trial of Pegzilarginase for ARG1-D in Q2 of 2019
Initiated IND-Enabling Studies for New Pipeline Programs
Progress with Pegzilarginase Cancer Indication-Phase 2 Combination Study Initiated
Gross Proceeds of $69 Million from February 2019 Public Offering Extends Cash Runway Through Q1 of 2021
Company to Host Conference Call and Webcast Today at 4:30 p.m. ET
AUSTIN, Texas, March 07, 2019 (GLOBE NEWSWIRE) -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, today reported its fourth quarter and full year 2018 financial results and provided corporate highlights.
“2018 was a very successful year for the company,” said Anthony G. Quinn, M.B. Ch.B, Ph.D., president and chief executive officer of Aeglea. “We made substantial progress with our clinical programs for pegzilarginase in Arginase 1 Deficiency (ARG1-D) and oncology, leveraged our unique drug hunting capabilities to generate new pipeline programs for cystinuria and homocystinuria and strengthened Aeglea’s balance sheet. As we look to 2019, the Company is well positioned to advance our clinical and research programs, as well as to leverage our human enzyme design capabilities to address other diseases with significant unmet medical need.”
Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase in Cancer
AEB4104 for Homocystinuria
AEB5100 for Cystinuria
Fourth Quarter and Full Year 2018 Financial Results
As of December 31, 2018, Aeglea had available cash, cash equivalents and marketable securities of $74.5 million, which excludes approximately $69.0 million in gross proceeds from a public offering to new and existing shareholders in February 2019. Based on Aeglea’s current operating plan, and taking into account the $69.0 million raised in February 2019, management believes it has sufficient capital resources to fund anticipated operations through the first quarter of 2021.
Grant revenues were the result of a $19.8 million research grant received from the Cancer Prevention and Research Institute of Texas (CPRIT). The grant contract concluded in May 2018 with the full $19.8 million in grant proceeds collected and recognized as revenue over the life of the award. Aeglea did not recognize any grant revenues in the fourth quarter of 2018, compared with $1.5 million in the fourth quarter of 2017. Grant revenues of $3.9 million were recognized in the year ended December 31, 2018, compared with $5.2 million in the year ended December 31, 2017.
Research and development expenses totaled $11.8 million for the fourth quarter of 2018, compared with $5.8 million for the fourth quarter of 2017. The increase was primarily due to additional clinical development activity of Aeglea’s lead product candidate, pegzilarginase, and a ramp-up in manufacturing activities for pegzilarginase. Aeglea completed and exceeded enrollment in its Phase 1/2 clinical trial in patients with Arginase 1 Deficiency, completed enrollment of three single-agent cohort expansions in a Phase 1 clinical trial for advanced solid tumor patients, and completed enrollment of a Phase 1 combination trial in patients with small cell lung cancer.
Research and development expenses totaled $36.7 million for the year ended December 31, 2018, compared with $22.8 million for the year ended December 31, 2017. The increase was primarily associated with advancing the clinical development of pegzilarginase and expanding our internal clinical development capabilities and research laboratory. Aeglea completed and exceeded enrollment in its Phase 1/2 clinical trial for pegzilarginase in patients with Arginase 1 Deficiency. Additionally, the Company initiated and completed enrollment of three single-agent cohort expansions for the Phase 1 trial in patients with advanced solid tumors and completed enrollment of its Phase 1 combination trial in patients with small cell lung cancer.
General and administrative expenses totaled $3.5 million for the fourth quarter of 2018, compared with $2.3 million for the fourth quarter of 2017. This increase was primarily due to additional employee headcount, compensation, and consulting costs.
General and administrative expenses totaled $12.6 million for the year ended December 31, 2018, compared with $10.1 million in the year ended December 31, 2017. This increase was primarily due to additional employee headcount and compensation to support Company growth.
Net loss totaled $14.9 million and $6.5 million for the fourth quarter of 2018 and 2017, respectively, with non-cash stock compensation expense of $1.4 million and $0.7 million for the fourth quarter of 2018 and 2017, respectively. Net loss totaled $44.3 million and $27.2 million for the years ended December 31, 2018 and 2017, respectively, with non-cash stock compensation expense of $4.3 million and $2.5 million for the years ended December 31, 2018 and 2017, respectively.
Conference Call & Webcast Details
Aeglea will hold a conference call on Thursday, March 7, 2019 at 4:30 p.m. ET. To access the live conference call via phone, please dial +1 (877) 709-8155 (toll free) within the United States, or +1 (201) 689-8881 internationally. A replay of the call will be available through March 14, 2019 by dialing +1 (877) 660-6853 within the United States or +1 (201) 612-7415 internationally. The conference ID is 13688006.
To access the live and archived webcast of the presentation, please visit the Presentation & Events section of the Aeglea BioTherapeutics investor relations website. Please connect to the website at least 15 minutes prior to the presentation to allow for any software download that may be necessary.
About Pegzilarginase in Arginase 1 Deficiency
Pegzilarginase is an enhanced human arginase that enzymatically depletes the amino acid arginine. Aeglea is developing pegzilarginase for the treatment of patients with Arginase 1 Deficiency, a rare debilitating disease presenting in childhood with persistent hyperargininemia, severe progressive neurological abnormalities and early mortality. Pegzilarginase is intended for use as an enzyme replacement therapy in patients to reduce elevated blood arginine levels. Aeglea’s interim Phase 1/2 data demonstrated clinical improvements and rapid and sustained lowering of plasma arginine in Arginase 1 Deficiency patients.
About AEB4104 in Homocystinuria
AEB4104 is a novel recombinant human enzyme that degrades the amino acid homocysteine and its related homocystine dimer. Aeglea is developing AEB4104 for the treatment of patients with cystathionine beta synthase (CBS) deficiency, also known as Classical Homocystinuria. 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 and intellectual disability; lens dislocation and severe myopia. AEB4104 is intended to lower the abnormally high blood levels of homocysteine to the normal range in patients with homocystinuria. Preclinical data demonstrated that AEB4104 improved important disease-related abnormalities and survival in a mouse model of homocystinuria.
About AEB5100 in Cystinuria
AEB5100 is a novel recombinant human enzyme that degrades cystine and the parent amino acid cysteine. Aeglea is developing AEB5100 for the treatment of patients with cystinuria, a rare genetic disease characterized by frequent and recurrent kidney stone formation from an early age. Affected individuals have severe symptoms, frequent hospital admissions, a requirement for multiple procedural interventions and an increased risk of both hypertension and chronic kidney disease. Cystinuria occurs due to genetic mutations in amino acid transporters that lead to increased amounts of cystine in the urine. The high urine cystine concentrations leads to kidney stones formation. Preclinical data demonstrated that AEB5100 lowered blood levels of cystine and cysteine, decreased the amount of cystine in the urine and reduced kidney stone formation in a mouse model of cystinuria.
About Pegzilarginase in Cancer
Pegzilarginase is an enhanced human arginase that enzymatically degrades the amino acid arginine. In some cancers, tumor cells are unable to produce specific amino acids and must acquire them from the blood, making the tumor cells susceptible to starvation through depletion of those amino acids. Aeglea is developing pegzilarginase to exploit vulnerabilities in some cancers that lead to an increased dependency on extracellular arginine. Pegzilarginase targets these arginine dependent cancers by depleting blood arginine levels to below the normal range. Preclinical data demonstrated that the resulting arginine starvation inhibits proliferation, induces cell death, increases turnover of cell components and promotes anti-tumor immune responses. The Company’s Phase 1 data in advanced solid tumors demonstrated that pegzilarginase was well tolerated at doses that produced marked and sustained reductions in blood arginine levels below the normal range.
About Aeglea BioTherapeutics
Aeglea is a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer. Aeglea is developing pegzilarginase, its lead investigational therapy, for the treatment of Arginase 1 Deficiency, as monotherapy in arginine-dependent cancers and in combination with an immune checkpoint inhibitor for small cell lung cancer. In addition, Aeglea has an active research pipeline of other human enzyme-based approaches in both therapeutic areas. For more information, please visit http://aegleabio.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 cash forecasts, the timing and success of our clinical trials and related data, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, success in our collaborations 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 Annual Report on Form 10-K for the year ended December 31, 2018 filed with the Securities and Exchange Commission (SEC), and 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.
KEYTRUDA® is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA.
Sharon Merrill Associates
Director, Finance & Investor Relations
Aeglea BioTherapeutics, Inc.
Consolidated Balance Sheets
(In thousands, except share and per share amounts)
|Cash and cash equivalents||$||22,461||$||12,817|
|Accounts receivable - grant||—||3,078|
|Prepaid expenses and other current assets||2,158||1,614|
|Total current assets||76,671||54,991|
|Property and equipment, net||1,018||854|
|Other non-current assets||50||232|
|LIABILITIES AND STOCKHOLDERS’ EQUITY|
|Accrued and other current liabilities||9,576||5,220|
|Total current liabilities||10,239||5,629|
|Other non-current liabilities||72||111|
|Preferred stock, $0.0001 par value; 10,000,000 shares authorized as of December 31, 2018 and December 31, 2017; no shares issued and outstanding as of December 31, 2018 and December 31, 2017||—||—|
|Common stock, $0.0001 par value; 500,000,000 shares authorized as of December 31, 2018 and December 31, 2017; 24,140,097 shares and 16,670,188 shares issued and outstanding as of December 31, 2018 and December 31, 2017, respectively||2||2|
|Additional paid-in capital||184,314||122,950|
|Accumulated other comprehensive loss||(27||)||(102||)|
|TOTAL STOCKHOLDERS’ EQUITY||67,428||50,337|
|TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY||$||77,739||$||56,077|
Aeglea BioTherapeutics, Inc.
Consolidated Statements of Operations
(In thousands, except share and per share amounts)
|Research and development||36,719||22,815||18,143|
|General and administrative||12,632||10,066||8,391|
|Total operating expenses||49,351||32,881||26,534|
|Loss from operations||(45,463||)||(27,676||)||(21,906||)|
|Other income (expense):|
|Total other income||1,115||440||208|
|Net loss per share, basic and diluted||$||(2.13||)||$||(1.80||)||$||(2.22||)|
|Weighted-average common shares outstanding, basic and diluted||20,822,560||15,128,192||9,791,728|