Summary of Aeglea BioTherapeutics Q4 2018 Earnings Call

1Key Financial Results and Metrics

Net Loss: $14.9 million for Q4 2018, compared to a net loss of $6.5 million in Q4 2017.

Loss Per Share: $0.62 in Q4 2018 versus $0.39 in Q4 2017.

Grant Revenue: $1.5 million from a $19.8 million cancer research grant recognized over its duration.

Cash Position: Pro forma cash of approximately $139 million as of December 31, 2018, following a $69 million financing in February 2019, expected to fund operations through the PEACE trial readout in Q1 2021.

Operating Expenses: Increased R&D expenses to $11.8 million in Q4 2018 from $5.8 million in Q4 2017, reflecting investment in clinical programs.

2Strategic Updates and Business Highlights

Clinical Progress: Significant advancements in the clinical program for pegzilarginase, particularly in Arginase 1 Deficiency and oncology.

Pipeline Development: Initiated IND-enabling studies for new programs targeting cystinuria and homocystinuria.

PEACE Trial: The global pivotal Phase 3 trial for pegzilarginase in Arginase 1 Deficiency is set to begin in Q2 2019, with a focus on plasma arginine reduction as the primary endpoint.

Oncology Trials: Completed enrollment in Phase 1 trials for pegzilarginase in solid tumors and initiated a Phase 2 trial in combination with KEYTRUDA for Small Cell Lung Cancer.

3Forward Guidance and Outlook

2019 Expectations: Anticipated quarterly cash burn of $12 million to $15 million, with a higher burn of $15 million to $17 million expected in Q1 2019 due to ramping pipeline development.

Clinical Milestones: Guidance for IND filings for homocystinuria in Q1 2020 and cystinuria in H2 2020.

Data Releases: Expect top-line data from the Phase 1b combination trial in the first half of 2020.

4Bad News, Challenges, or Points of Concern

Clinical Efficacy: No observed objective responses in the Small Cell Lung Cancer cohort during the Phase 1 trial raised questions about the efficacy of pegzilarginase in this indication.

Market Competition: The competitive landscape for rare disease therapies remains challenging, with pricing pressures and regulatory scrutiny on drug costs.

Patient Identification: While the company has identified over 170 patients with Arginase 1 Deficiency, the true prevalence remains uncertain, suggesting potential underrepresentation in the patient population.

5Notable Q&A Insights

Homocystinuria and Cystinuria Programs: Management expressed confidence in the early clinical proof of concept for both programs, with homocystinuria expected to show results early due to the established link between homocystine levels and disease control.

Pricing Strategy: The pricing for pegzilarginase in Arginase 1 Deficiency will be influenced by both plasma arginine reduction and observed clinical benefits, with management emphasizing the transformative potential of the therapy.

Regulatory Engagement: The company has received Rare Pediatric Disease designation, which could lead to a Pediatric Review Voucher upon approval, enhancing the potential market value of the product. This summary encapsulates the key financial metrics, strategic initiatives, forward guidance, challenges faced, and insights from the Q&A session during Aeglea BioTherapeutics' Q4 2018 earnings call.