Agios Reports Business Highlights and Third Quarter 2022 Financial Results
– Received Positive CHMP Opinion for PYRUKYND® in Adults with Pyruvate Kinase (PK) Deficiency –
– On Track to Meet Year-end Enrollment Targets in PYRUKYND® Thalassemia and Sickle Cell Disease Pivotal Programs; First PK Activator Trials in Pediatric PK Deficiency and Myelodysplastic Syndromes (MDS) Continue to Enroll –
– Strengthened Company Leadership with Appointments of
“Agios is on the cusp of changing the treatment landscape for people with rare and genetically defined diseases, with the potential of PYRUKYND® to make a positive impact across multiple underserved diseases. I joined the company in August because of its differentiated portfolio, top-notch team and genuine dedication to patients, and my excitement for the future of Agios has only grown over the past few months,” said
Third Quarter 2022 & Recent Highlights
- Continued to execute
U.S.launch of PYRUKYND®, generating $3.5 millionin U.S.net revenue for the third quarter of 2022, the second full quarter following FDA approval. A total of 84 unique patients have completed prescription enrollment forms, representing an increase of 64 percent over the second quarter. A total of 56 patients are on PYRUKYND® therapy, representing a 51 percent increase over the second quarter. Increased patient demand was partially offset by modest inventory build in the prior quarters of launch.
- Received positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency(EMA), recommending the granting of a marketing authorization for PYRUKYND® for the treatment of PK deficiency in adult patients.
Medicines and Healthcare Products Regulatory Agency(MHRA) filing for the approval of PYRUKYND® as a treatment for adults with PK deficiency in Great Britain.
- Continued to enroll patients across five PYRUKYND® pivotal studies in thalassemia, sickle cell disease and pediatric PK deficiency.
- Initiated Phase 2a study of novel PK activator AG-946 in adults with lower-risk MDS.
- Published PYRUKYND® data in top-tier medical journals, including Phase 2 thalassemia data in
The Lancetand Phase 3 ACTIVATE-T data in The Lancet Haematology.
Cecilia Jonesas Agios’ chief financial officer, effective Sept. 26, 2022.
Rahul Ballal, Ph.D., chief executive officer of Imara, and Cynthia Smith, former chief commercial officer of ZS Pharma, to Agios’ board of directors.
- Completed the sale of royalty rights on
U.S.net sales of Servier’s TIBSOVO® to Sagard Healthcare Partnersfor a one-time payment of $131.8 million.
Key Upcoming Milestones & Priorities
Agios expects to execute on the following key milestones and priorities by the end of 2022:
- Adult PK Deficiency: Receive EU and
Great Britainregulatory decisions for PYRUKYND® in adults with PK deficiency.
- Thalassemia: Enroll a meaningful portion of patients in the Phase 3 ENERGIZE and ENERGIZE-T studies of PYRUKYND® in not regularly transfused and regularly transfused adults with thalassemia, respectively.
- Sickle Cell Disease: Complete enrollment in the Phase 2 portion of the RISE UP study of PYRUKYND® in adults with sickle cell disease.
- Data Presentations: Present broad set of clinical and translational data at the 64th
American Society of Hematology(ASH) Annual Meeting & Exposition; abstracts will be available at 9 a.m. ETtoday.
Third Quarter 2022 Financial Results
The financial results discussion compares Agios’ continuing operations. All periods have been adjusted to exclude discontinued operations related to the divested oncology business.
Cost of Sales: Cost of sales for the third quarter of 2022 was
Non-Operating Income: Non-operating income included approximately
Research and Development (R&D) Expenses: R&D expenses were $65.0 million for the third quarter of 2022 compared to $64.0 million for the third quarter of 2021.
Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $29.1 million for the third quarter of 2022 compared to $27.2 million for the third quarter of 2021. The year-over-year increase in SG&A expenses was primarily attributable to an increase in workforce-related expenses.
Net Loss from Continuing Operations: Net loss from continuing operations was $81.7 million for the third quarter of 2022 compared to a net loss of $84.3 million for the third quarter of 2021.
Cash Position and Guidance: Cash, cash equivalents and marketable securities as of September 30, 2022, were $1.0 billion compared to $1.4 billion as of September 30, 2021. This cash position does not include the receipt of a one-time payment of
Conference Call Information
Agios will host a conference call and live webcast with slides today at 8:00 a.m. ET to discuss third quarter 2022 financial results and recent business activities. 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.
Agios is a biopharmaceutical company that is fueled by connections. The Agios team cultivates strong bonds with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver therapies for rare and genetically defined diseases. In the
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 Agios’ plans, strategies and expectations for the preclinical, clinical and commercial advancement of its drug development programs, including PYRUKYND® (mitapivat) and AG-946; the potential benefits of Agios’ products and product candidates; Agios’ key milestones and guidance for 2022; its financial guidance regarding the period in which it will have capital available to fund its operations; 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. 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 the COVID-19 pandemic on 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 future approved products, and launching, marketing and selling 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
|Consolidated Balance Sheet Data|
|Cash, cash equivalents, and marketable securities||$||1,026,032||$||1,286,393|
|Accounts receivable, net||1,818||—|
|Consolidated Statements of Operations Data|
|(in thousands, except share and per share data)|
|Three Months Ended September
|Nine Months Ended September
|Product revenue, net||$||3,516||$||—||$||7,430||$||—|
|Cost and expenses:|
|Cost of sales||$||517||$||—||$||1,291||$||—|
|Research and development||64,966||64,000||209,612||183,674|
|Selling, general and administrative||29,123||27,152||88,902||89,917|
|Total cost and expenses||94,606||91,152||299,805||273,591|
|Loss from operations||(91,090||)||(91,152||)||(289,875||)||(273,591||)|
|Royalty income from gain on sale of oncology business||4,443||1,996||9,851||3,996|
|Interest income, net||3,818||256||6,305||504|
|Other income, net||1,082||4,641||5,392||11,165|
|Net loss from continuing operations||(81,747||)||(84,259||)||(268,327||)||(257,926||)|
|Net (loss) income from discontinued operations, net of tax||—||(4,507||)||—||1,957,268|
|Net (loss) income||$||(81,747||)||$||(88,766||)||$||(268,327||)||$||1,699,342|
|Net loss from continuing operations per share - basic and diluted||$||(1.49||)||$||(1.48||)||$||(4.90||)||$||(4.13||)|
|Net (loss) income from discontinued operations per share - basic and diluted||$||—||$||(0.08||)||$||—||$||31.31|
|Net (loss) income per share - basic and diluted||$||(1.49||)||$||(1.56||)||$||(4.90||)||$||27.19|
|Weighted-average number of common shares used in computing net loss per share from continuing operations, net (loss) income from discontinued operations and net (loss) income per share – basic and diluted||54,844,579||57,048,175||54,734,301||62,503,087|
Senior Director, Investor Relations
Senior Director, Corporate Communications
Source: Agios Pharmaceuticals, Inc.