Agios Reports Business Highlights and Second Quarter 2024 Financial Results
Rhea-AI Summary
Agios Pharmaceuticals (NASDAQ: AGIO) reported its Q2 2024 financial results and business highlights. Key points include:
1. Positive topline data from Phase 3 ENERGIZE-T study for thalassemia treatment.
2. $905 million purchase agreement with Royalty Pharma for vorasidenib royalty.
3. Results from Phase 3 ACTIVATE-KidsT study of mitapivat in children with PK deficiency.
4. PYRUKYND® net revenue of $8.6 million in Q2, a 5% increase from Q1.
5. Cash position of $645.3 million as of June 30, 2024.
6. Net loss of $96.1 million for Q2 2024.
7. R&D expenses increased to $77.4 million, while SG&A expenses rose to $35.5 million.
8. Plans to file sNDA for mitapivat in thalassemia by year-end 2024.
Positive
- Positive topline data from Phase 3 ENERGIZE-T study for thalassemia treatment
- $905 million purchase agreement with Royalty Pharma for vorasidenib royalty
- PYRUKYND® net revenue increased 5% sequentially to $8.6 million in Q2 2024
- Strong cash position of $645.3 million as of June 30, 2024
- Completed enrollment of Phase 3 ACTIVATE-Kids study for PK deficiency
Negative
- Net loss increased to $96.1 million in Q2 2024 compared to $83.8 million in Q2 2023
- R&D expenses increased to $77.4 million from $68.9 million year-over-year
- SG&A expenses rose to $35.5 million from $30.4 million year-over-year
- Cash position decreased from $806.4 million as of December 31, 2023 to $645.3 million as of June 30, 2024
Insights
Agios Pharmaceuticals' Q2 2024 results present a mixed financial picture. PYRUKYND® net revenue reached
The
R&D expenses increased to
While Agios' cash position provides runway, the increasing losses and cash burn rate warrant close monitoring. The success of PYRUKYND® in additional indications and potential approval of vorasidenib will be important for the company's financial trajectory.
Agios' recent clinical developments are promising, particularly in the thalassemia space. The positive topline data from the Phase 3 ENERGIZE-T study, coupled with the earlier success in the ENERGIZE study, positions mitapivat as a potential first-in-class therapy for all thalassemia subtypes. This broad efficacy could significantly expand the drug's market potential.
The company's progress in pediatric PK deficiency is also noteworthy. The completion of the ACTIVATE-KidsT study marks an important milestone, as it's the first to report safety and efficacy data in children with this rare condition. While detailed results are pending, this study could pave the way for expanded use of mitapivat in younger patients.
The advancement of tebapivat (AG-946) into Phase 2b for lower-risk myelodysplastic syndromes demonstrates Agios' commitment to expanding its pipeline beyond PK deficiency and thalassemia. This diversification could help mitigate risk and open up new market opportunities.
However, it's important to note that while these clinical developments are positive, they are still steps in a longer process. The upcoming sNDA filing for mitapivat in thalassemia by year-end will be a critical regulatory milestone to watch. Additionally, the completion of enrollment in the Phase 3 RISE UP study for sickle cell disease will be important for expanding mitapivat's potential indications.
Overall, Agios' clinical pipeline shows promise, but investors should remain aware that success in clinical trials doesn't guarantee regulatory approval or commercial success. The company's ability to navigate the regulatory process and effectively commercialize its products will be key to realizing the potential of these clinical advancements.
– Reported Positive Topline Data from Phase 3 ENERGIZE-T Study; Expect to File sNDA Based on ENERGIZE and ENERGIZE-T Studies Encompassing All Thalassemia Subtypes by End of 2024 –
– Announced
– Reported Results from Phase 3 ACTIVATE-KidsT Study of Mitapivat in Children with Pyruvate Kinase (PK) Deficiency Who are Regularly Transfused –
– PYRUKYND® (Mitapivat) Net Revenue of
CAMBRIDGE, Mass., Aug. 01, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today reported business highlights and financial results for the second quarter ended June 30, 2024.
“Based on the positive data generated in the Phase 3 ENERGIZE and ENERGIZE-T studies, mitapivat is the first therapy to demonstrate efficacy in all subtypes of thalassemia, and we look forward to filing for FDA review by the end of the year,” said Brian Goff, chief executive officer at Agios. “This morning, we were pleased to report topline data in the Phase 3 ACTIVATE-KidsT study of mitapivat, which is the first study to report safety and efficacy data in children with PK deficiency. We continue to make significant progress toward our vision of becoming a leading rare disease company with a potential multi-billion-dollar franchise in PK activation. Finally, we were pleased to bolster our cash position through a purchase agreement with Royalty Pharma for our vorasidenib royalty, with Agios now positioned to receive a total of
Second Quarter 2024 and Recent Highlights
- PYRUKYND® Revenues: Generated
$8.6 million in net revenue for the second quarter of 2024, a 5 percent sequential increase from the first quarter of 2024, primarily driven by increased patient demand. A total of 201 unique patients have completed prescription enrollment forms, representing an increase of 7 percent over the first quarter of 2024. A total of 128 patients are on PYRUKYND® therapy, a 7 percent increase from the first quarter of 2024. - Thalassemia:
- Met the primary and all key secondary endpoints in the Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia.
- Presented positive results from the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent thalassemia in a plenary session at the European Hematology Association 2024 (EHA2024) Hybrid Congress.
- Pediatric PK Deficiency:
- Announced topline data from the Phase 3 ACTIVATE-KidsT study of mitapivat in children with PK deficiency who are regularly transfused. Agios plans to present a more detailed analyses of the results at an upcoming medical meeting.
- Completed enrollment of the Phase 3 ACTIVATE-Kids study of mitapivat in children with PK deficiency who are not regularly transfused. Topline data from this study are expected in 2025.
- Corporate Development:
- Announced a
$905 million purchase agreement with Royalty Pharma for Agios’ rights to its vorasidenib royalty. Under the agreement, Agios will receive a payment of$905 million upon approval of vorasidenib by the FDA and Royalty Pharma will receive the entirety of the15% royalty on annual U.S. net sales of vorasidenib up to$1 billion , and a12% royalty on annual U.S. net sales greater than$1 billion . Agios retains a3% royalty on annual U.S. net sales greater than$1 billion . Agios retains rights to a$200 million milestone payment from Servier upon FDA approval of vorasidenib. - Entered into a distribution agreement with NewBridge Pharmaceuticals to advance commercialization of PYRUKYND® in the Gulf Cooperation Council (GCC) region. NewBridge, a leading specialty company headquartered in Dubai, will commercialize PYRUKYND® in Bahrain, Kuwait, Oman, Qatar, Saudi Arabia and the United Arab Emirates.
- Announced a
Key Upcoming Milestones & Priorities
Agios expects to execute on the following additional key milestones and priorities by the end of 2024:
- Thalassemia: File sNDA for mitapivat in thalassemia based on the positive results from the Phase 3 ENERGIZE and ENERGIZE-T trials (year-end).
- Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end).
- Lower-risk Myelodysplastic Syndromes: Dose first patient in Phase 2b study of tebapivat (AG-946) (mid-year).
- Other: Potential approval of Servier’s vorasidenib for the treatment of IDH-mutant diffuse glioma. The FDA has assigned a PDUFA action date of August 20, 2024.
Second Quarter 2024 Financial Results
Revenue: Net product revenue from sales of PYRUKYND® for the second quarter of 2024 was
Cost of Sales: Cost of sales for the second quarter of 2024 was
Research and Development (R&D) Expenses: R&D expenses were
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were
Net Loss: Net loss was
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of June 30, 2024, were
Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss second quarter 2024 financial results and recent business highlights. The live webcast can be accessed under “Events & Presentations” in the Investors section of the company’s website at www.agios.com. The archived webcast will be available on the company's website beginning approximately two hours after the event.
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.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat), tebapivat (AG-946), TMPRSS6 siRNA and AG-181, Agios’ PAH stabilizer; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, AG-946 (tebapivat) and AG-181; the potential FDA approval of vorasidenib; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2024; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any milestone or royalty payments related to the sale of its oncology business or its in-licensing of TMPRSS6 siRNA, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
| Consolidated Balance Sheet Data | ||||||||
| (in thousands) | ||||||||
| (Unaudited) | ||||||||
| June 30, 2024 | December 31, 2023 | |||||||
| Cash, cash equivalents, and marketable securities | $ | 645,296 | $ | 806,363 | ||||
| Accounts receivable, net | 3,762 | 2,810 | ||||||
| Inventory | 23,937 | 19,076 | ||||||
| Total assets | 773,063 | 937,118 | ||||||
| Stockholders' equity | 660,510 | 811,019 | ||||||
| Consolidated Statements of Operations Data | |||||||||||||||
| (in thousands, except share and per share data) | |||||||||||||||
| (Unaudited) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Revenues: | |||||||||||||||
| Product revenue, net | $ | 8,615 | $ | 6,712 | $ | 16,804 | $ | 12,321 | |||||||
| Total revenue | 8,615 | 6,712 | 16,804 | 12,321 | |||||||||||
| Operating expenses: | |||||||||||||||
| Cost of sales | $ | 1,495 | $ | 1,108 | $ | 2,122 | $ | 1,662 | |||||||
| Research and development | 77,401 | 68,895 | 146,021 | 136,196 | |||||||||||
| Selling, general and administrative | 35,536 | 30,409 | 66,550 | 58,776 | |||||||||||
| Total operating expenses | 114,432 | 100,412 | 214,693 | 196,634 | |||||||||||
| Loss from operations | (105,817 | ) | (93,700 | ) | (197,889 | ) | (184,313 | ) | |||||||
| Interest income, net | 8,120 | 8,254 | 17,009 | 16,345 | |||||||||||
| Other income, net | 1,579 | 1,640 | 3,213 | 3,144 | |||||||||||
| Net loss | $ | (96,118 | ) | $ | (83,806 | ) | $ | (177,667 | ) | $ | (164,824 | ) | |||
| Net loss per share - basic and diluted | $ | (1.69 | ) | $ | (1.51 | ) | $ | (3.14 | ) | $ | (2.97 | ) | |||
| Weighted-average number of common shares used in computing net loss per share – basic and diluted | 56,802,546 | 55,604,330 | 56,593,011 | 55,435,796 | |||||||||||
Contacts:
Investor Contact
Chris Taylor, VP Investor Relations and Corporate Communications
Agios Pharmaceuticals
IR@agios.com
Media Contact
Dan Budwick
1AB
dan@1abmedia.com
FAQ
What was Agios Pharmaceuticals' (AGIO) PYRUKYND® revenue in Q2 2024?
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