Agios to Receive $1.1 Billion in Milestone Payments Following FDA Approval of Vorasidenib

Rhea-AI Summary

Agios Pharmaceuticals (Nasdaq: AGIO) is set to receive $1.1 billion in milestone payments following the FDA approval of vorasidenib for Grade 2 astrocytoma or oligodendroglioma. This includes $905 million from Royalty Pharma and $200 million from Servier, expected in Q3 2024. The payments will increase Agios' pro-forma cash position to $1.7 billion as of June 30, 2024.

This strong financial position will enable Agios to prepare for potential PYRUKYND® (mitapivat) launches in thalassemia (2025) and sickle cell disease (2026), while driving pipeline progress. The approval marks the first for Grade 2 glioma in over 20 years, highlighting Agios' expertise in discovering novel therapies for high unmet needs.

Positive

• FDA approval of vorasidenib, first for Grade 2 glioma in over 20 years
• $1.1 billion in milestone payments to be received
• Increase in pro-forma cash position to $1.7 billion
• Financial independence to prepare for potential PYRUKYND® launches in 2025 and 2026
• Retained 3% royalty on annual U.S. net sales of vorasidenib exceeding $1 billion

Negative

• None.

Financial Analyst

The FDA approval of vorasidenib triggers significant milestone payments for Agios, substantially bolstering its financial position. The $1.1 billion influx, comprising $905 million from Royalty Pharma and $200 million from Servier, will increase Agios' pro-forma cash position to $1.7 billion as of June 30, 2024. This robust cash reserve provides Agios with considerable financial flexibility to:

• Fund potential PYRUKYND® launches in thalassemia (2025) and sickle cell disease (2026)
• Drive pipeline progress
• Aim for a multi-billion-dollar franchise

The deal structure with Royalty Pharma allows Agios to monetize future royalties while retaining upside potential on sales exceeding $1 billion. This strategic move balances immediate cash needs with long-term value creation potential.

Oncology Expert

The FDA approval of vorasidenib marks a significant milestone in glioma treatment, being the first approval for Grade 2 glioma in over two decades. This breakthrough highlights Agios' expertise in developing therapies for high unmet needs in oncology. The drug targets adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma harboring IDH1 or IDH2 mutations, post-surgery. This approval could potentially reshape the treatment landscape for low-grade gliomas, offering a targeted therapy option where alternatives existed. The success also validates Agios' research capabilities, even after divesting its oncology business, potentially enhancing its reputation in the rare disease space.

Market Research Analyst

Agios' strategic decisions, including the sale of its oncology business to Servier and the royalty agreement with Royalty Pharma, demonstrate a shift in focus towards rare diseases. The company's ability to secure substantial milestone payments from these deals showcases its adept financial management and long-term planning. The $1.7 billion pro-forma cash position provides a strong foundation for Agios to pursue its rare disease pipeline, particularly PYRUKYND® for thalassemia and sickle cell disease. This cash runway reduces near-term financing risks and allows for aggressive investment in R&D and commercialization efforts. Investors should monitor the progress of PYRUKYND® launches and pipeline developments, as these will be key drivers of Agios' future value proposition in the competitive rare disease market.

08/06/2024 - 05:50 PM

– Agios Expects to Receive $905 Million Payment from Royalty Pharma and $200 Million Payment from Servier in Q3 2024; Payments Increase Agios’ Pro-Forma Cash Position as of June 30, 2024, to $1.7 Billion –

– Strong Cash Position to Enable Agios to Prepare for Potential PYRUKYND® (Mitapivat) Launches in Thalassemia in 2025 and Sickle Cell Disease in 2026 and Drive Pipeline Progress –

CAMBRIDGE, Mass., Aug. 06, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the Company will receive a total of $1.1 billion in milestone payments following the U.S. Food and Drug Administration (FDA) approval of vorasidenib for adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery, including biopsy, sub-total resection or gross total resection. These payments include a $905 million payment from Royalty Pharma in connection with the vorasidenib royalty purchase agreement Agios announced in May 2024 and a $200 million payment from Servier in connection with Agios’ divestiture of its oncology business in 2021.  

“Today’s FDA approval of vorasidenib is the first approval for a Grade 2 glioma in over 20 years and highlights both the executional excellence of the Servier team and Agios’ expertise in discovering novel therapies for disease areas of high unmet need,” said Cecilia Jones, chief financial officer at Agios. “The $1.1 billion in milestone payments triggered by today’s approval further strengthens our cash position, which we expect will provide us with the financial independence to prepare for potential PYRUKYND® (mitapivat) launches in thalassemia in 2025 and sickle cell disease in 2026, as well as drive pipeline progress as we aim to build a multi-billion-dollar franchise.”

Agios completed the sale of its oncology business to Servier on March 31, 2021. Under the terms of the agreement, Agios received $1.8 billion in upfront cash, an additional $200 million milestone payment upon FDA approval of vorasidenib, and 15% royalties on potential U.S. net sales of vorasidenib from the first commercial sale through loss of exclusivity. Agios had also retained rights to 5% royalties on U.S. net sales of Servier’s TIBSOVO® (ivosidenib tablets), which it sold for a one-time payment of $131.8 million in 2022.

On May 28, 2024, Agios announced that the company agreed to sell its rights to its 15% royalty on potential U.S. net sales of vorasidenib to Royalty Pharma. Under the terms of the agreement, Agios receives an upfront payment of $905 million upon FDA approval of vorasidenib, and Royalty Pharma will receive the entirety of the 15% royalty on annual U.S. net sales of vorasidenib up to $1 billion and a 12% royalty on annual U.S. net sales greater than $1 billion. Agios will retain a 3% royalty on annual U.S. net sales greater than $1 billion.

About Agios

Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S., Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, please visit the company’s website at www.agios.com.

Contacts:

Investor Contact

Chris Taylor, VP, Investor Relations and Corporate Communications
Agios Pharmaceuticals
IR@agios.com

Media Contact

Katie Engleman
1AB
katie@1abmedia.com

FAQ

What milestone payments will Agios Pharmaceuticals (AGIO) receive following vorasidenib's FDA approval?

Agios Pharmaceuticals will receive a total of $1.1 billion in milestone payments, including $905 million from Royalty Pharma and $200 million from Servier, following the FDA approval of vorasidenib.

How will the milestone payments affect Agios Pharmaceuticals' (AGIO) cash position?

The milestone payments will increase Agios Pharmaceuticals' pro-forma cash position to $1.7 billion as of June 30, 2024, providing financial independence for future product launches and pipeline development.

What are the potential launch dates for PYRUKYND® (mitapivat) by Agios Pharmaceuticals (AGIO)?

Agios Pharmaceuticals is preparing for potential PYRUKYND® (mitapivat) launches in thalassemia in 2025 and sickle cell disease in 2026.

What royalties will Agios Pharmaceuticals (AGIO) retain on vorasidenib sales?

Agios Pharmaceuticals will retain a 3% royalty on annual U.S. net sales of vorasidenib exceeding $1 billion.