Agios Shares Q2 2024 Results and Business Highlights

Agios Pharmaceuticals, Inc., a key player in cellular metabolism and pyruvate kinase (PK) activation therapies for rare diseases, released its business highlights and financial results for the second quarter ending June 30, 2024.

CEO Brian Goff highlighted the company's significant progress, noting the positive data from Phase 3 ENERGIZE and ENERGIZE-T studies. These studies have demonstrated mitapivat's efficacy in all thalassemia subtypes, with plans to seek FDA approval by year-end. Additionally, Agios reported promising topline data from the Phase 3 ACTIVATE-KidsT study, marking the first safety and efficacy data in children with PK deficiency.

In a recent development, Agios announced a $905 million purchase agreement with Royalty Pharma for the vorasidenib royalty. This agreement positions Agios to receive $1.1 billion upon FDA approval of vorasidenib, bolstering its financial position further.

Second Quarter 2024 Highlights:
- PYRUKYND® Revenues: Agios generated $8.6 million in net revenue for the second quarter of 2024, a 5% increase from the first quarter. The rise was attributed to growing patient demand, with 201 unique patients completing prescription enrollment forms, representing a 7% increase over the first quarter. A total of 128 patients are currently on PYRUKYND® therapy.

- Thalassemia: The Phase 3 ENERGIZE-T study met its primary and all key secondary endpoints in adults with transfusion-dependent alpha- or beta-thalassemia. Positive results from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia were presented at the European Hematology Association 2024 Hybrid Congress.

- Pediatric PK Deficiency: Topline data from the Phase 3 ACTIVATE-KidsT study of mitapivat in regularly transfused children with PK deficiency were announced. Agios intends to present more detailed analyses at a future medical meeting. Enrollment for the Phase 3 ACTIVATE-Kids study of mitapivat in non-regularly transfused children has been completed, with topline data expected in 2025.

- Corporate Development: The $905 million purchase agreement with Royalty Pharma will see Agios receiving payments upon vorasidenib's FDA approval. Additionally, a distribution agreement with NewBridge Pharmaceuticals will advance commercialization of PYRUKYND® in the Gulf Cooperation Council (GCC) region, covering countries including Bahrain, Kuwait, Oman, Qatar, Saudi Arabia, and the UAE.

Upcoming Milestones and Priorities for 2024:
- Thalassemia: Agios plans to file a supplemental New Drug Application (sNDA) for mitapivat based on results from the ENERGIZE and ENERGIZE-T trials by the end of 2024.
- Sickle Cell Disease: Completion of enrollment in the Phase 3 RISE UP study of mitapivat is expected by year-end.
- Lower-risk Myelodysplastic Syndromes: The first patient dosing in a Phase 2b study of tebapivat (AG-946) is anticipated by mid-year.
- Other: Potential approval of Servier’s vorasidenib for treating IDH-mutant diffuse glioma, with an FDA action date set for August 20, 2024.

Financial Results:
- Revenue: Net product revenue from PYRUKYND® sales for Q2 2024 was $8.6 million, up from $6.7 million in Q2 2023.
- Cost of Sales: The cost of sales for Q2 2024 was $1.5 million.
- R&D Expenses: Research and development expenses were $77.4 million in Q2 2024, up from $68.9 million in Q2 2023, primarily due to increased costs associated with the in-licensed siRNA TMPRSS6 program for polycythemia vera.
- SG&A Expenses: Selling, general and administrative expenses were $35.5 million in Q2 2024, compared to $30.4 million in Q2 2023, reflecting increased commercial activities in preparation for PYRUKYND®'s potential approval in thalassemia.
- Net Loss: Agios reported a net loss of $96.1 million in Q2 2024, compared to $83.8 million in Q2 2023.
- Cash Position: As of June 30, 2024, Agios had $645.3 million in cash, cash equivalents, and marketable securities, down from $806.4 million as of December 31, 2023.

Agios remains focused on achieving financial independence to prepare for potential PYRUKYND® launches in thalassemia and sickle cell disease, while also exploring opportunities to expand its pipeline.