On May 1, 2025 Alkermes plc (Nasdaq: ALKS) reported financial results for the first quarter of 2025 (Press release, Alkermes, MAY 1, 2025, View Source [SID1234652421]).

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"Our first quarter financial performance provides a solid foundation to deliver on our financial guidance for the year. We are in a strong position in this dynamic macroeconomic environment and remain focused on executing on the strategic objectives that we believe will drive the future value of the company," said Richard Pops, Chief Executive Officer of Alkermes. "We recently achieved an important milestone in the program for ALKS 2680, our novel, investigational, oral orexin 2 receptor agonist, completing enrollment in our first phase 2 study in the program, Vibrance-1, in narcolepsy type 1. We now expect topline results from Vibrance-1 early in the third quarter. We expect to complete enrollment in the Vibrance-2 phase 2 study, in narcolepsy type 2, mid-year, with topline data from that study expected in the fall. Enrollment in Vibrance-3, our phase 2 study in idiopathic hypersomnia, is now also underway. Across the ALKS 2680 development program, we have strong momentum and are preparing for the phase 3 program. With the potential to transform the treatment of central disorders of hypersomnolence, orexin 2 receptor agonists are one of the most exciting new therapeutic categories in development."

Key Financial Highlights

Revenues

(In millions)

Three Months Ended
March 31,

2025

2024

Total Revenues

$

306.5

$

350.4

Total Proprietary Net Sales

$

244.5

$

233.5

VIVITROL

$

101.0

$

97.7

ARISTADAi

$

73.5

$

78.9

LYBALVI

$

70.0

$

57.0

Profitability

(In millions)

Three Months Ended
March 31,

2025

2024

GAAP Net Income From Continuing Operations

$

22.5

$

38.9

GAAP Net Income (Loss) From Discontinued Operations

$

—

$

(2.1)

GAAP Net Income

$

22.5

$

36.8

EBITDA From Continuing Operations

$

22.8

$

51.5

EBITDA From Discontinued Operations

$

—

$

(2.5)

EBITDA

$

22.8

$

49.0

Adjusted EBITDA

$

45.6

$

81.8

Revenue Highlights

LYBALVI

Revenues for the quarter were $70.0 million.
Revenues and total prescriptions for the quarter grew 23% and 22%, respectively, compared to the first quarter of 2024.
ARISTADAi

Revenues for the quarter were $73.5 million.
VIVITROL

Revenues for the quarter were $101.0 million.
Manufacturing & Royalty Revenues

VUMERITY manufacturing and royalty revenues for the quarter were $27.8 million.
Royalty revenues from XEPLION, INVEGA TRINZA/TREVICTA and INVEGA HAFYERA/BYANNLI for the quarter were $17.7 million.
Key Operating Expenses

Please see Note 1 below for details regarding discontinued operations.

(In millions)

Three Months Ended
March 31,

2025

2024

R&D Expense – Continuing Operations

$

71.8

$

67.6

R&D Expense – Discontinued Operations

$

—

$

2.5

SG&A Expense – Continuing Operations

$

171.7

$

179.7

SG&A Expense – Discontinued Operations

$

—

$

—

Balance Sheet

At March 31, 2025, the company recorded cash, cash equivalents and total investments of $916.2 million, compared to $824.8 million at Dec. 31, 2024.
Financial Expectations for 2025
Alkermes reiterates its financial expectations for 2025, as set forth in its press release dated Feb. 12, 2025.

Notes and Explanations
1. The company determined that upon the separation of its former oncology business, completed on Nov. 15, 2023, the oncology business met the criteria for discontinued operations in accordance with Financial Accounting Standards Board Accounting Standards Codification 205, Discontinued Operations. Accordingly, the accompanying selected financial information has been updated to present the results of the oncology business as discontinued operations for the three months ended March 31, 2024.

Conference Call
Alkermes will host a conference call and webcast presentation with accompanying slides at 8:00 a.m. ET (1:00 p.m. BST) on Thursday, May 1, 2025, to discuss these financial results and provide an update on the company. The webcast may be accessed on the Investors section of Alkermes’ website at www.alkermes.com. The conference call may be accessed by dialing +1 877 407 2988 for U.S. callers and +1 201 389 0923 for international callers. In addition, a replay of the conference call may be accessed by visiting Alkermes’ website.

On May 1, 2025 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, reported business highlights and financial results for the first quarter ended March 31, 2025 (Press release, Agios Pharmaceuticals, MAY 1, 2025, View Source [SID1234652420]).

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"We are pleased with our strong start to 2025, highlighted by the acceptance of our sNDA for thalassemia with a PDUFA goal date of September 7, 2025. Our engagement with the FDA is progressing as expected, and we are committed to bringing PYRUKYND to thalassemia patients, irrespective of genotype or transfusion needs," said Brian Goff, chief executive officer at Agios. "Looking ahead, our focus is also on delivering the topline results from the Phase 3 RISE UP study in sickle cell disease, which remains on track for year-end, and continuing to advance our early and mid-stage clinical programs. Supported by our strong financial position and highly experienced team, we are driving forward PYRUKYND’s multi-billion-dollar potential while building a pipeline designed for lasting impact, with the goal of creating significant value for shareholders and delivering transformative therapies for patients."

First Quarter 2025 and Recent Highlights

PYRUKYND Revenues: Generated $8.7 million in net revenue for the first quarter of 2025, compared to $8.2 million in the first quarter of 2024. A total of 234 unique patients have completed prescription enrollment forms, representing an increase of 5 percent over the fourth quarter of 2024. A total of 136 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy, as compared to 130 patients at the end of the fourth quarter 2024.
Thalassemia:
The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. The FDA has communicated that at this time no advisory committee meeting is planned, and the review is ongoing.
Sickle Cell Disease:
The Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older continued to progress as anticipated. This 52-week Phase 3 study completed enrollment in October 2024, enrolling more than 200 patients worldwide.
Advanced final preparations to initiate a Phase 2 clinical trial of tebapivat in patients with sickle cell disease in mid-2025.
Pediatric Pyruvate Kinase (PK) Deficiency:
Reported positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused.
Safety was consistent with the profile for mitapivat previously observed in adults with PK deficiency who are not regularly transfused.
ACTIVATE-Kids is the first study to demonstrate efficacy of an oral therapy for children with PK Deficiency who are not regularly transfused.
Lower-risk Myelodysplastic Syndromes (LR-MDS):
Progressed patient enrollment in the Phase 2b study of tebapivat in LR-MDS.
Corporate: Krishnan Viswanadhan, Pharm. D, joined Agios as Chief Corporate Development and Strategy Officer, responsible for leading the company’s corporate strategy, business development, and long-term growth initiatives. Previously, he served as President and Chief Operating Officer of Be Biopharma and at various senior roles at both Bristol Myers Squibb and Celgene.
Key Upcoming Milestones & Priorities

Agios expects to achieve the following key milestones in 2025:

Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia (PDUFA goal date is September 7, 2025). Continue progressing the review of regulatory applications with health authorities in the European Union, Kingdom of Saudi Arabia and United Arab Emirates.
Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025.
LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025.
Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025.
First Quarter 2025 Financial Results

Revenue: Net product revenue from sales of PYRUKYND for the first quarter of 2025 was $8.7 million, compared to $8.2 million for the first quarter of 2024.

Cost of Sales: Cost of sales for the first quarter of 2025 was $1.1 million.

Research and Development (R&D) Expenses: R&D expenses were $72.7 million for the first quarter of 2025, compared to $68.6 million for the first quarter of 2024. The year-over-year increase was primarily attributed to an increase in workforce-related expenses and costs associated with clinical trials of tebapivat in LR-MDS and sickle cell disease, partially offset by lower costs associated with the clinical trials of mitapivat in thalassemia and pediatric PKD.

Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $41.5 million for the first quarter of 2025 compared to $31.0 million for the first quarter of 2024. The year-over-year increase was primarily attributable to an increase in commercial-related activities, including headcount, as the company prepares for the potential approval of PYRUKYND in thalassemia.

Net Loss: Net loss was $89.3 million for the first quarter of 2025 compared to $81.5 million for the first quarter of 2024.

Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2025, were $1.4 billion compared to $1.5 billion as of December 31, 2024. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets.

Conference Call Information

Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s first quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.