Homocystinuria (Tome Biosciences)

AUSTIN, Texas, Nov. 30, 2022 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced the appointment of Jeffrey M. Goldberg as president, chief executive officer and member of the board of directors effective November 29, 2022. Mr. Goldberg is an accomplished biotech executive with over 25 years of experience leading teams from preclinical discovery through commercialization and previously served as president and chief executive officer of Immunitas Therapeutics. "Jeff has successfully built and managed teams across a wide variety of functions, providing strong leadership and strategic direction. His breadth of experience spans from the founding of companies to overseeing successful global launches of rare disease therapies," said Russell J. Cox, chair of the board of directors of Aeglea. "His proven ability to match business capabilities to unmet patient needs and then drive the strategy and execution needed to achieve those goals will be an important asset to Aeglea. I look forward to working closely with Jeff and welcome him to the Aeglea team." "To me, the most important and fulfilling part of my job has been being able to bring therapies forward that have had significant impact on patients' lives. Aeglea shares that mission and has built world-class programs in Homocystinuria and Arginase 1 Deficiency with the potential to benefit all the key stakeholders, including employees, shareholders, physicians, and most importantly patients and their families," said Mr. Goldberg. "I'm incredibly excited to be joining the team and helping to facilitate the mission of developing novel medicines for patients battling devastating diseases." Mr. Goldberg served as president and chief executive officer at Immunitas from 2019-2021. Under his leadership, Immunitas grew significantly, raised additional capital, and is now in the clinic with its first asset. Prior to joining Immunitas, Mr. Goldberg served as the founding chief operating officer at Akcea where he was responsible for building functions and leading teams across all aspects of the business and eventually grew the team from two employees to over 250 in multiple geographies. Mr. Goldberg played a significant role in the company's initial public offering and fundraising activities, business development and the global launch of two rare disease drugs. Prior to Akcea, he was the vice president of business operations at Proteostasis Therapeutics, a rare disease company. He also held roles of increasing responsibility at Genzyme, where he led teams through product development and global launches across multiple therapeutic areas. Mr. Goldberg has an MBA and a MS in Chemical Engineering from the Massachusetts Institute of Technology, and a B.S. in Chemical Engineering from Cornell University. Jim Kastenmayer, JD, PhD, will step down as interim chief executive officer and retain his position as Aeglea's general counsel. "On behalf of the entire board of directors, I want to thank Jim for his service and commitment to Aeglea and its mission. Jim has provided exceptional leadership during a period of critical execution and has kept the team focused on advancing the Homocystinuria program," said Mr. Cox. Following the leadership transition and as part of the related evaluation of near-term corporate and clinical development strategy, Aeglea no longer plans to announce interim clinical data from the ongoing Phase 1/2 clinical trial of pegtarviliase in Classical Homocystinuria in the fourth quarter of 2022. Enrollment in the third cohort of the Phase 1/2 trial is ongoing with two patients having completed dosing in the cohort. Additionally, the company has decided not to participate in the Piper Sandler 34th Annual Healthcare Conference and 5th Annual Evercore ISI HealthCONx Conference, both being held November 29-December 1, 2022. Aeglea looks forward to delivering a clinical update on the pegtarviliase program when more comprehensive data from the third cohort becomes available. About Classical Homocystinuria Classical Homocystinuria, also known as CBS deficiency, is a serious, progressive metabolic disorder characterized by elevated levels of total homocysteine. The accumulation of total homocysteine leads to a wide range of life-altering complications and even death from sudden catastrophic events. Serious disease complications may include thromboembolic vascular events, skeletal abnormalities (including severe osteoporosis and Marfanoid features), developmental delay, intellectual disability, lens dislocation and severe near sightedness. Currently, there are no approved treatments that address the underlying driver of disease. 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. Aeglea is investigating pegtarviliase in an ongoing Phase 1/2 clinical trial for the treatment of Classical Homocystinuria. Pegtarviliase has been granted Rare Pediatric Disease Designation. Aeglea's other clinical program, 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. The Marketing Authorization Application for pegzilarginase is currently under review with the European Medicines Agency. 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 . 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 achieve further cost-savings, the timing of announcements and updates relating to our clinical trials and related data, including the clinical data for our Phase 1/2 trial of pegtarviliase in Classical Homocystinuria, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, including the MAA for pegzilarginase in Europe, our ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, 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 September 30, 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. SOURCE Aeglea BioTherapeutics, Inc.