On May 1, 2025 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company working to develop a new class of drugs based on targeted protein degradation, reported financial results for the first quarter ended March 31, 2025, and provided a corporate update (Press release, Arvinas, MAY 1, 2025, View Source [SID1234652423]).

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"The positive readout from our first Phase 3 trial, the first ever for a PROTAC, is a tremendous accomplishment and one which we are very proud of," said John Houston, Ph.D., Chairperson, Chief Executive Officer and President at Arvinas. "Our conviction is high that vepdegestrant can be highly competitive as a monotherapy treatment option for metastatic breast cancer in the second-line, ESR1 mutant setting. We are on track to submit a regulatory filing with health authorities, which we believe could result in the first ever approval of a PROTAC and an exciting opportunity to bring a novel treatment to patients with ESR1 mutant advanced metastatic breast cancer."

"Beyond the second-line monotherapy opportunity, we and our partners at Pfizer have removed plans for a Phase 3 first-line combination trial with atirmociclib, as well as the planned Phase 3 second-line combination trial with a CDK4/6 inhibitor, from our joint development plan," continued Dr. Houston. "This decision was made following a review of the totality of emerging information, including external data results, the evolving treatment landscape in metastatic breast cancer, and long-term capital allocation. We and Pfizer are working to evaluate future combination plans with the potential to maximize patient benefit and shareholder value."

1Q 2025 Business Highlights and Recent Developments

Vepdegestrant: Oral PROTAC ER degrader: As part of Arvinas global collaboration with Pfizer, the companies:

Reported positive topline data for VERITAC-2, the Phase 3 pivotal 2L+ trial of monotherapy vepdegestrant in patients with estrogen receptor-positive, human epidermal growth factor receptor 2-negative (ER+/HER2-) advanced or metastatic breast cancer, in the estrogen receptor 1-mutant population.
VERITAC-2 data accepted for oral presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (May 30 – June 3, 2025).
Announced that Pfizer will add a vepdegestrant/KAT6 cohort to its ongoing Phase 1 trial evaluating its investigational KAT6 inhibitor (PF-07248144) in combination with endocrine therapies following CDK4/6 inhibitor treatment.
Removed the first-line Phase 3 combination trial with Pfizer’s novel investigational CDK4 inhibitor, atirmociclib, from the agreed-upon joint development plan.
Removed the second-line Phase 3 combination trial with a CDK4/6 inhibitor from the agreed-upon joint development plan.
ARV-102: Oral PROTAC LRRK2 degrader

Presented single ascending dose (SAD) and multiple ascending dose (MAD) data from the ongoing Phase 1 clinical trial in healthy volunteers in an oral session at the Alzheimer’s Disease/Parkinson’s Disease (AD/PD) conference in Vienna, Austria demonstrating blood-brain barrier penetration, and central and peripheral LRRK2 degradation:
At a single oral dose of at least 60 mg, and once daily repeated oral doses of at least 20 mg, ARV-102 achieved greater than 50% LRRK2 reduction in the cerebral spinal fluid (CSF) and greater than 90% LRRK2 reduction in the peripheral blood mononuclear cells (PBMCs), indicating substantial central and peripheral LRRK2 protein degradation.
Inhibition of Rab10 phosphorylation in PBMCs and reduction of bis(monoacylglycerol)phosphate (BMP) in urine following single doses of ARV-102, signifying downstream LRRK2 pathway engagement.
Bioavailable and brain penetrant with dose dependent exposure in the CSF.
ARV-102 was generally safe and well tolerated with no serious adverse events reported after single or multiple doses.
ARV-393: Oral PROTAC BCL6 degrader

Continued recruiting patients in the first-in-human Phase 1 clinical trial in patients with non-Hodgkin lymphoma (NHL) (ClinicalTrials.gov Identifier: NCT06393738).
Presented new preclinical data of ARV-393 in combination with standard of care (SOC) biologic agents and small molecule inhibitors (SMI) in high-grade B-cell lymphoma (HGBCL) and aggressive diffuse large B-cell lymphoma (DLBCL) models at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting demonstrating that ARV-393 had broad and significant combinability with:
SOC chemotherapy:
ARV-393 in combination with R-CHOP, induced significantly greater tumor growth inhibition (TGI) compared with rituximab, CHOP, R-CHOP, or ARV-393 alone, with complete tumor regressions enabled by the combination.
SOC biologics:
Similarly, the combination of ARV-393 and SOC biologics targeting CD19 (tafasitamab), CD79b (polatuzumab vedotin), or CD20 (rituximab) resulted in tumor regressions and demonstrated significantly stronger TGI compared with either agent alone.
Investigational small molecule inhibitors targeting clinically validated oncogenic drivers of lymphoma:
Combination of ARV-393 with a BTK inhibitor (acalabrutinib), a BCL2 inhibitor (venetoclax), or an EZH2 inhibitor (tazemetostat) induced tumor regressions in the majority of mice.
Overall, current preclinical data suggest that ARV-393 has the potential to be an attractive combination partner for development of novel therapies for lymphoma, including chemo-free combination regimens and/or "all oral" treatment options.
ARV-806: Novel PROTAC KRAS G12D degrader

Filed an Investigational New Drug (IND) application and received a safe-to-proceed letter from the U.S. Food and Drug Administration.
Corporate updates:
As part of a Company-wide cost reduction effort:

Announced the removal of two Phase 3 trials from the vepdegestrant development plan
Announced a reduction in workforce of approximately one-third across the Company to streamline operations across the organization and enable the efficient progression of the Company’s portfolio. The reduction is planned to be completed in the second quarter of 2025.
Announced a reprioritization of the Company’s preclinical portfolio to focus on assets that have the greatest potential to deliver value for patients, physicians and shareholders.
Updated guidance for its cash runway into the second half of 2028.
"We continuously evaluate and refine our long-term strategy, and recent challenges in the capital markets have required us to expeditiously evaluate our business priorities and capital needs," continued Dr. Houston. "Although difficult, the workforce reduction is a prudent decision that we believe will right-size the Company for future success. I want to thank all the talented employees who were directly impacted by this decision. I’m proud of the progress we have made together, and want to acknowledge their contributions, and commitment, to discovering and developing new treatment options for patients with serious diseases."

Anticipated Upcoming Milestones and Expectations

Vepdegestrant: Oral PROTAC ER degrader
As part of Arvinas’ global collaboration with Pfizer, the companies plan to:

Present detailed results from the VERITAC-2 Phase 3 clinical trial in a late-breaker oral presentation at ASCO (Free ASCO Whitepaper) (2Q 2025).
VERITAC-2 abstract will be featured in the ASCO (Free ASCO Whitepaper) press program and has been selected to be included in the "2025 Best of ASCO (Free ASCO Whitepaper) Program."
Share data with global regulatory authorities to potentially support regulatory filings (2Q 2025) and submit new drug application to the U.S. Food and Drug Administration for potential approval (2H 2025).
Add a combination cohort of vepdegestrant plus Pfizer’s KAT6 inhibitor (PF-07248144) to Pfizer’s ongoing Phase 1 trial (NCT04606446).
The trial is currently evaluating Pfizer’s KAT6 inhibitor in combination with endocrine therapies following CDK4/6 inhibitor treatments; the trial is being operationalized and funded by Pfizer.
ARV-102: Oral PROTAC LRRK2 degrader

Present final data from the SAD/MAD cohorts of the Phase 1 clinical trial in healthy volunteers (2H 2025).
Continue enrollment and present initial data from the SAD cohort of the ongoing Phase 1 clinical trial in patients with Parkinson’s disease (2H 2025).
Initiate the MAD cohort of the Phase 1 clinical trial in patients with Parkinson’s disease (2H 2025).
ARV-393: Oral PROTAC BCL6 degrader

Present new preclinical data demonstrating single agent activity of ARV-393 in patient derived xenograft models of transformed Follicular Lymphoma and a patient-derived xenograft model of nTFHL-AI (angioimmunoblastic type of nodal T-follicular helper cell lymphoma), a rare and aggressive non-Hodgkin lymphoma with high unmet need and limited treatment options, at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 conference in Milan, Italy (June 12-15, 2025).
Share preclinical data in combination with an emerging SOC option in 2L DLBCL (2H 2025).
Share preliminary clinical data from the ongoing Phase 1 clinical trial in patients with NHL (NCT06393738) (2H 2025).
ARV-806: Novel PROTAC KRAS G12D degrader

Initiate a first-in-human Phase 1 trial in patients with solid tumors harboring KRAS G12D mutations (2H 2025).
Financial Guidance
Based on its current operating plan, Arvinas believes its cash, cash equivalents, and marketable securities as of March 31, 2025, is sufficient to fund planned operating expenses and capital expenditure requirements into the second half of 2028.

First Quarter Financial Results
Cash, Cash Equivalents, and Marketable Securities Position: As of March 31, 2025, cash, cash equivalents and marketable securities were $954.3 million as compared with cash, cash equivalents and marketable securities of $1,039.4 million as of December 31, 2024. The decrease in cash, cash equivalents and marketable securities of $85.1 million for the three months ended March 31, 2025 was primarily related to cash used in operations of $85.1 million, the purchase of lab equipment and leasehold improvements of $0.4 million and $0.1 million of long term debt repayments, partially offset by unrealized gains on marketable securities of $0.5 million.

Research and Development Expenses: Generally Accepted Accounting Principles (GAAP) Research and development (R&D) expenses were $90.8 million for the quarter ended March 31, 2025, as compared with $84.3 million for the quarter ended March 31, 2024. The increase in research and development expenses of $6.5 million for the quarter was primarily due to an increase in external expenses of $7.8 million, partially offset by a decrease in compensation and related personnel expenses of $1.4 million, which are not allocated by program. External expenses include (i) program-specific expenses, which increased by $10.0 million, primarily driven by increases in our ARV-102, vepdegestrant, ARV-393 and other programs of $5.2 million, $5.0 million, $1.4 million and $2.5 million, respectively, offset by decreases in our luxdegalutamide (ARV-766) and bavdegalutamide (ARV-110) programs of $3.6 million and $0.5 million, respectively, and (ii) our non-program specific expenses, which decreased by $2.2 million.

Non-GAAP R&D expenses were $79.3 million for the quarter ended March 31, 2025, as compared with $71.9 million for the quarter ended March 31, 2024, excluding $11.5 million and $12.4 million of non-cash stock-based compensation expense for the quarters ended March 31, 2025 and 2024, respectively. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

General and Administrative Expenses: GAAP General and administrative (G&A) expenses were $26.6 million for the quarter ended March 31, 2025, as compared with $24.3 million for the quarter ended March 31, 2024. The increase in general and administrative expenses of $2.3 million for the quarter was primarily due an increase in professional fees of $2.4 million, inclusive of an increase in the amortization of costs to obtain a contract of $2.6 million related to changes in total Vepdegestrant (ARV-471) Collaboration Agreement program cost estimates resulting from the removal of two Phase 3 combination trials from the development plan and an increase in costs related to developing our commercial operations of $2.3 million, partially offset by a decrease in personnel and infrastructure related costs of $2.4 million.

Non-GAAP G&A expenses were $23.1 million for the quarter ended March 31, 2025, as compared with $18.0 million for the quarter ended March 31, 2024, excluding $3.5 million and $6.3 million of non-cash stock-based compensation expense for the quarters ended March 31, 2025 and 2024, respectively. A reconciliation of GAAP to non-GAAP financial measures used in this press release can be found at the end of this press release.

Revenue: Revenue was $188.8 million for the quarter ended March 31, 2025 as compared with $25.3 million for the quarter ended March 31, 2024. Revenue for the quarter is related to the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer and the collaboration and license agreement with Pfizer. The increase in revenue of $163.5 million was primarily due to an increase in revenue from the Vepdegestrant (ARV-471) Collaboration Agreement with Pfizer totaling $167.8 million related to changes in total program cost estimates resulting from the removal of the first-line Phase 3 combination trial with Pfizer’s novel investigational CDK4 inhibitor, atirmociclib, and the removal of the second-line Phase 3 combination trial with a CDK4/6 inhibitor from the development plan, offset by a decrease in revenue from the Pfizer Research Collaboration Agreement of $2.7 million due to changes in estimates of the performance period duration under the agreement resulting from updated research timelines and a decrease in revenue from the Bayer Collaboration Agreement of $1.6 million related to the termination of the Bayer Collaboration Agreement in August 2024.

Investor Call & Webcast Details
Arvinas will host a conference call and webcast today, May 1, 2025, at 8:00 a.m. ET to review its first quarter 2025 financial results and discuss recent corporate updates. Participants are invited to listen by going to the Events and Presentation section under the Investors page on the Arvinas website at www.arvinas.com. A replay of the webcast will be available on the Arvinas website following the completion of the event and will be archived for up to 30 days.

On May 1, 2025 Amgen (NASDAQ:AMGN) reported financial results for the first quarter of 2025 (Press release, Amgen, MAY 1, 2025, View Source [SID1234652422]).

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"Demand for our products was strong globally in the first quarter. Ongoing new product launches and successful Phase 3 trial results for several products make us feel confident in our long-term growth prospects," said Robert A. Bradway, chairman and chief executive officer.

Key results include:

For the first quarter, total revenues increased 9% to $8.1 billion in comparison to the first quarter of 2024.
Product sales grew 11%, primarily driven by 14% volume growth, partially offset by 6% lower net selling price. U.S. sales grew 14%.
Fourteen products delivered at least double-digit sales growth in the first quarter, including Repatha (evolocumab), BLINCYTO (blinatumomab), TEZSPIRE (tezepelumab-ekko), EVENITY (romosozumab-aqqg), TAVNEOS (avacopan) and UPLIZNA (inebilizumab-cdon).
Our launch of IMDELLTRA (tarlatamab-dlle) generated $81 million of sales in the quarter and the Phase 3 confirmatory study demonstrated improved overall survival compared to chemotherapy. IMDELLTRA launched in Japan in April 2025.
GAAP earnings per share (EPS) were $3.20 for the first quarter of 2025 compared with a GAAP loss per share of $0.21 for the first quarter of 2024, resulting from an unrealized gain on our BeiGene, Ltd. equity investment during the first quarter of 2025 compared to an unrealized loss during the prior year period, partially offset by an Otezla intangible asset impairment charge of $800 million recorded during the first quarter of 2025.
GAAP operating income increased from $1.0 billion to $1.2 billion, and GAAP operating margin increased 1.1 percentage points to 15.0%.
Non-GAAP EPS increased 24% from $3.96 to $4.90, driven by higher revenues, partially offset by higher operating expenses.
Non-GAAP operating income increased from $3.1 billion to $3.6 billion and non-GAAP operating margin increased 2.5 percentage points to 45.7%.
The Company generated $1.0 billion of free cash flow in the first quarter of 2025 versus $0.5 billion in the first quarter of 2024. This increase reflects an $800 million tax deposit made in the first quarter of 2024 and current quarter business performance, partially offset by timing of working capital items and higher capital expenditures.
References in this release to "non-GAAP" measures, measures presented "on a non-GAAP basis," and "free cash flow" (computed by subtracting capital expenditures from operating cash flow) refer to non-GAAP financial measures. Adjustments to the most directly comparable GAAP financial measures and other items are presented in the attached reconciliations. Refer to Non-GAAP Financial Measures below for further discussion.

Product Sales Performance

General Medicine

Repatha (evolocumab) sales increased 27% year-over-year to $656 million in the first quarter, primarily driven by 41% volume growth, partially offset by 9% lower net selling price.

EVENITY (romosozumab-aqqg) sales increased 29% year-over-year to $442 million in the first quarter, driven by volume growth.

Prolia (denosumab) sales increased 10% year-over-year to $1.1 billion in the first quarter, primarily driven by 13% volume growth, partially offset by 5% lower net selling price. For 2025, we expect sales erosion driven by biosimilar competition, particularly in the second half of the year.
Rare Disease

TEPEZZA (teprotumumab-trbw) sales decreased 10% year-over-year to $381 million in the first quarter, primarily driven by 9% lower volume and 8% from a decrease in inventory levels, partially offset by higher net selling price.

KRYSTEXXA (pegloticase) sales were flat year-over-year at $236 million in the first quarter, as 11% volume growth was offset by 10% from a decrease in inventory levels.

UPLIZNA (inebilizumab-cdon) sales increased 14% year-over-year to $91 million in the first quarter, primarily driven by volume growth.

TAVNEOS (avacopan) sales increased 76% year-over-year to $90 million in the first quarter, primarily driven by volume growth.

Ultra-Rare products, which consist of RAVICTI (glycerol phenylbutyrate), PROCYSBI (cysteamine bitartrate), ACTIMMUNE (interferon gamma-1b), BUPHENYL (sodium phenylbutyrate) and QUINSAIR (levofloxacin), generated $179 million of sales in the first quarter. Sales increased 6% year-over-year for the first quarter, driven by volume growth.
Inflammation

TEZSPIRE (tezepelumab-ekko) sales increased 65% year-over-year to $285 million in the first quarter, driven by volume growth.

Otezla (apremilast) sales increased 11% year-over-year to $437 million in the first quarter, driven by 12% favorable changes to estimated sales deductions, as volume growth of 4% was offset by 5% lower net selling price.

Enbrel (etanercept) sales decreased 10% year-over-year to $510 million in the first quarter, driven by 47% lower net selling price resulting from increased 340B Program mix and higher commercial discounts, partially offset by 19% favorable changes to estimated sales deductions, higher inventory levels and volume growth. The year-over-year impact on net selling price is expected to be less pronounced in future quarters.

AMJEVITA (adalimumab-atto)/AMGEVITA (adalimumab) sales decreased 19% year-over-year to $136 million in the first quarter, primarily driven by 33% lower net selling price, partially offset by 11% volume growth.

WEZLANA (ustekinumab-auub)/WEZENLA (ustekinumab) generated $150 million of sales in the first quarter. WEZLANA launched in the U.S. in the first quarter of 2025 and is the first FDA-approved biosimilar for the treatment of adult and pediatric plaque psoriasis, psoriatic arthritis, Crohn’s disease and ulcerative colitis.

PAVBLU (aflibercept-ayyh) generated $99 million of sales in the first quarter. PAVBLU launched in the U.S. in the fourth quarter of 2024 and is the first available aflibercept biosimilar for the treatment of retinal conditions, including neovascular age-related macular degeneration, macular edema following retinal vein occlusion, diabetic macular edema and diabetic retinopathy.
Oncology

BLINCYTO (blinatumomab) sales increased 52% year-over-year to $370 million in the first quarter, primarily driven by volume growth.

Vectibix (panitumumab) sales increased 8% year-over-year to $267 million in the first quarter, driven by volume growth.

KYPROLIS (carfilzomib) sales decreased 14% year-over-year to $324 million in the first quarter, driven by lower volumes due to increased competition.

LUMAKRAS/LUMYKRAS (sotorasib) sales increased 4% year-over-year to $85 million in the first quarter, driven by volume growth, partially offset by lower net selling price.

XGEVA (denosumab) sales increased 1% year-over-year to $566 million in the first quarter. For 2025, we expect sales erosion driven by biosimilar competition, particularly in the second half of the year.

Nplate (romiplostim) sales decreased 1% year-over-year to $313 million in the first quarter as volume growth was more than offset by unfavorable changes to estimated sales deductions and foreign exchange impact.

IMDELLTRA (tarlatamab-dlle)/IMDYLLTRA (tarlatamab) generated $81 million of sales in the first quarter. Sales increased 21% quarter-over-quarter driven by volume growth.

MVASI (bevacizumab-awwb) sales decreased 11% year-over-year to $179 million in the first quarter, primarily driven by lower volume. Going forward, we expect continued sales erosion driven by competition.
Established Products

Our established products, which consist of Aranesp (darbepoetin alfa), Parsabiv (etelcalcetide) and Neulasta (pegfilgrastim), generated $557 million of sales in the first quarter. Sales decreased 3% year-over-year for the first quarter, driven by 10% lower net selling price and 3% unfavorable foreign exchange impact, partially offset by favorable changes to estimated sales deductions.
Product Sales Detail by Product and Geographic Region

$Millions, except percentages

Q1 ’25

Q1 ’24

YOY Δ

U.S

ROW

TOTAL

TOTAL

TOTAL

Repatha

$ 343

$ 313

$ 656

$ 517

27 %

EVENITY

320

122

442

342

29 %

Prolia

720

379

1,099

999

10 %

TEPEZZA

365

16

381

424

(10 %)

KRYSTEXXA

236

—

236

235

0 %

UPLIZNA

82

9

91

80

14 %

TAVNEOS

77

13

90

51

76 %

Ultra-Rare products(1)

171

8

179

169

6 %

TEZSPIRE

285

—

285

173

65 %

Otezla

343

94

437

394

11 %

Enbrel

504

6

510

567

(10 %)

AMJEVITA/AMGEVITA

4

132

136

168

(19 %)

WEZLANA/WEZENLA

123

27

150

1

*

PAVBLU

99

—

99

—

N/A

BLINCYTO

273

97

370

244

52 %

Vectibix

135

132

267

247

8 %

KYPROLIS

216

108

324

376

(14 %)

LUMAKRAS/LUMYKRAS

55

30

85

82

4 %

XGEVA

360

206

566

561

1 %

Nplate

201

112

313

317

(1 %)

IMDELLTRA/IMDYLLTRA

79

2

81

—

N/A

MVASI

138

41

179

202

(11 %)

Aranesp

91

249

340

349

(3 %)

Parsabiv

50

38

88

105

(16 %)

Neulasta

109

20

129

118

9 %

Other products(2)

283

57

340

397

(14 %)

Total product sales

$ 5,662

$ 2,211

$ 7,873

$ 7,118

11 %

* Change in excess of 100%

N/A = not applicable

(1) Ultra-Rare products consist of RAVICTI, PROCYSBI, ACTIMMUNE, BUPHENYL and QUINSAIR.

(2) Consists of Aimovig, AVSOLA, KANJINTI, RIABNI, EPOGEN, BEKEMV, NEUPOGEN, IMLYGIC, Corlanor, RAYOS, Sensipar/Mimpara, DUEXIS and PENNSAID, where Biosimilars total $171 million in Q1 ’25 and $175 million in Q1 ’24.

Operating Expense, Operating Margin and Tax Rate Analysis

On a GAAP basis:

Total Operating Expenses increased 8% year-over-year for the first quarter. Cost of Sales as a percentage of product sales decreased 7.3 percentage points driven by lower amortization expense from the fair value step-up of inventory acquired from Horizon and lower manufacturing costs, partially offset by changes in our sales mix. Research & Development (R&D) expenses increased 11% driven by higher spend in later-stage clinical programs, partially offset by lower spend in marketed product support and research and early pipeline. Selling, General & Administrative (SG&A) expenses decreased 7% driven by lower commercial product-related expenses and lower Horizon acquisition-related expenses, partially offset by higher general and administrative expenses. Other operating expenses consisted primarily of the Otezla intangible asset impairment charge of $800 million.

Operating Margin as a percentage of product sales increased 1.1 percentage points in the first quarter to 15.0%.

Tax Rate increased 78.5 percentage points in the first quarter due to the change in earnings mix as a result of the first quarter 2025 unrealized gains compared to the first quarter 2024 unrealized losses on our equity investments, primarily BeiGene.
On a non-GAAP basis:

Total Operating Expenses increased 4% year-over-year for the first quarter. Cost of Sales as a percentage of product sales decreased 0.8 percentage points driven by lower manufacturing costs, partially offset by changes in our sales mix. R&D expenses increased 12% driven by higher spend in later-stage clinical programs, partially offset by lower spend in marketed product support and research and early pipeline. SG&A expenses decreased 3% driven by lower commercial product-related expenses, partially offset by higher general and administrative expenses.

Operating Margin as a percentage of product sales increased 2.5 percentage points in the first quarter to 45.7%.

Tax Rate decreased 0.8 percentage points in the first quarter due to the change in earnings mix and net favorable items as compared to the prior year.
$Millions, except percentages

GAAP

Non-GAAP

Q1 ’25

Q1 ’24

YOY Δ

Q1 ’25

Q1 ’24

YOY Δ

Cost of Sales

$ 2,968

$ 3,200

(7 %)

$ 1,420

$ 1,340

6 %

% of product sales

37.7 %

45.0 %

(7.3) pts

18.0 %

18.8 %

(0.8) pts

Research & Development

$ 1,486

$ 1,343

11 %

$ 1,475

$ 1,317

12 %

% of product sales

18.9 %

18.9 %

— pts

18.7 %

18.5 %

0.2 pts

Selling, General & Administrative

$ 1,687

$ 1,808

(7 %)

$ 1,655

$ 1,712

(3 %)

% of product sales

21.4 %

25.4 %

(4.0) pts

21.0 %

24.1 %

(3.1) pts

Other

$ 830

$ 105

*

$ —

$ —

N/A

Total Operating Expenses

$ 6,971

$ 6,456

8 %

$ 4,550

$ 4,369

4 %

Operating Margin

Operating income as % of product sales

15.0 %

13.9 %

1.1 pts

45.7 %

43.2 %

2.5 pts

Tax Rate

12.3 %

(66.2) %

78.5 pts

14.6 %

15.4 %

(0.8) pts

pts: percentage points

* = Change in excess of 100%

N/A = not applicable

Cash Flow and Balance Sheet

The Company generated $1.0 billion of free cash flow in the first quarter of 2025 versus $0.5 billion in the first quarter of 2024. This increase reflects an $800 million tax deposit made in the first quarter of 2024 and current quarter business performance, partially offset by timing of working capital items and higher capital expenditures.
The Company declared a first quarter 2025 dividend on December 10, 2024 of $2.38 that was paid on March 7, 2025 to all stockholders of record as of February 14, 2025, representing a 6% increase from the same period in 2024.
During the first quarter of 2025, the Company reduced principal debt outstanding by $2.8 billion.
During the first quarter, there were no repurchases of shares of common stock.
Cash and cash equivalents totaled $8.8 billion and debt outstanding totaled $57.4 billion as of March 31, 2025.
$Billions, except shares

Q1 ’25

Q1 ’24

YOY Δ

Operating Cash Flow

$ 1.4

$ 0.7

$ 0.7

Capital Expenditures

$ 0.4

$ 0.2

$ 0.2

Free Cash Flow

$ 1.0

$ 0.5

$ 0.5

Dividends Paid

$ 1.3

$ 1.2

$ 0.1

Share Repurchases

$ —

$ —

$ —

Average Diluted Shares (millions)

541

536

5

Note: Numbers may not add due to rounding

$Billions

3/31/25

12/31/24

YTD Δ

Cash and Cash Equivalents

$ 8.8

$ 12.0

$ (3.2)

Debt Outstanding

$ 57.4

$ 60.1

$ (2.7)

Note: Numbers may not add due to rounding

2025 Guidance

For the full year 2025, the Company expects:

Total revenues in the range of $34.3 billion to $35.7 billion.
On a GAAP basis, EPS in the range of $12.21 to $13.46, and a tax rate in the range of 11.0% to 12.5%.
On a non-GAAP basis, EPS in the range of $20.00 to $21.20, and a tax rate in the range of 14.5% to 16.0%.
Capital expenditures to be approximately $2.3 billion.
Share repurchases not to exceed $500 million.
This guidance includes the estimated impact of implemented tariffs, but does not account for any tariffs that could be implemented in the future, including potential sector-specific tariffs.

First Quarter Product and Pipeline Update

The Company provided the following updates on selected product and pipeline programs:

General Medicine

MariTide (maridebart cafraglutide, AMG 133)

MariTide is a differentiated peptide-antibody conjugate that activates the glucagon like peptide 1 (GLP-1) receptor and antagonizes the gastric inhibitory polypeptide receptor (GIPR).
MARITIME-1, a Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability of maridebart cafraglutide is enrolling adults living with overweight or obesity, without Type 2 diabetes mellitus.
MARITIME-2, a Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability of maridebart cafraglutide is enrolling adults living with overweight or obesity, with Type 2 diabetes mellitus.
Planning for additional MARITIME Phase 3 studies across multiple indications remains on track, with additional studies expected to initiate throughout 2025.
Part 2 of the Phase 2 chronic weight management study is ongoing in adults living with overweight or obesity, with or without Type 2 diabetes mellitus. Data readout is anticipated in H2 2025.
A Phase 2 study investigating MariTide for the treatment of Type 2 diabetes mellitus has completed enrollment of adults living with and without obesity. Data readout is anticipated in H2 2025.
AMG 513

A Phase 1 study of AMG 513 is enrolling people living with obesity following removal of the clinical hold by the U.S. Food and Drug Administration (FDA).
Repatha

VESALIUS-CV, a Phase 3 CV outcomes study of Repatha, is ongoing in patients at high CV risk without prior myocardial infarction or stroke. Data readout is event driven and anticipated in H2 2025.
EVOLVE-MI, a Phase 4 study of Repatha administered within 10 days of an acute myocardial infarction to reduce the risk of CV events, is ongoing.
Olpasiran (AMG 890)

Olpasiran is a potentially best-in-class small interfering ribonucleic acid (siRNA) molecule that reduces lipoprotein(a) (Lp(a)) synthesis in the liver.
The OCEAN(a)-outcomes trial, a Phase 3 secondary prevention cardiovascular (CV) outcomes study, is ongoing in patients with atherosclerotic CV disease and elevated Lp(a).
A Phase 3 CV outcomes study in patients with elevated Lp(a) and at high risk for a first CV event is expected to be initiated in H2 2025/H1 2026.
Rare Disease

TAVNEOS

A Phase 3, open-label study of TAVNEOS in combination with rituximab or a cyclophosphamide-containing regimen, is enrolling patients from 6 years to < 18 years of age with active ANCA-associated vasculitis (Granulomatosis with Polyangiitis (GPA)/Microscopic Polyangiitis (MPA)).
TEPEZZA

Regulatory review is underway in multiple additional geographies, including with the European Medicines Agency (EMA), where approval is anticipated in H2 2025.
A Phase 3 study of TEPEZZA in Japan is enrolling patients with chronic/low clinical activity score thyroid eye disease (TED).
A Phase 3 study evaluating the subcutaneous route of administration of teprotumumab is enrolling patients with TED.
UPLIZNA

In April, the FDA approved UPLIZNA for the treatment of Immunoglobulin G4-Related Disease (IgG4-RD) in adult patients.
In April, data from the UPLIZNA Phase 3 MINT study in patients with generalized myasthenia gravis (gMG) were presented and simultaneously published in the New England Journal of Medicine. These data demonstrated durable and sustained efficacy of UPLIZNA treatment compared to placebo (adjusted difference, -1.8 at week 26; −2.8 at week 52) as measured by the change in baseline of Myasthenia Gravis Activities of Daily Living (MG-ADL) score in the acetylcholine receptor autoantibody-positive (AChR+) subpopulation through week 52. Among the AChR+ patients in the UPLIZNA group, 72% had a ≥3 point improvement in the MG-ADL score, compared to 45% in placebo at week 52. No new safety signals were identified.
The FDA has accepted the regulatory submission of the MINT Phase 3 data with a PDUFA date of December 14, 2025.
Dazodalibep

Dazodalibep is a fusion protein that inhibits CD40L.
Two Phase 3 studies of dazodalibep in Sjögren’s disease are enrolling patients. The first study is in patients with moderate-to-severe systemic disease activity, and the second study is in patients with moderate-to-severe symptomatic burden and low systemic disease activity.
Daxdilimab

Daxdilimab is a fully human monoclonal antibody targeting immunoglobulin-like transcript 7 (ILT7).
A Phase 2 study of daxdilimab is ongoing in patients with moderate-to-severe active primary discoid lupus erythematosus refractory to standard of care.
A Phase 2 study of daxdilimab is ongoing in patients with dermatomyositis and antisynthetase inflammatory myositis.
Inflammation

TEZSPIRE

Two Phase 3 studies of TEZSPIRE were initiated and are enrolling adults with moderate to very severe chronic obstructive pulmonary disease (COPD) and a BEC ≥ 150 cells/µl.
In March, data from the Phase 3 WAYPOINT study of TEZSPIRE in patients with chronic rhinosinusitis with nasal polyps (CRSwNP) were presented and simultaneously published in the New England Journal of Medicine demonstrating that treatment with TEZSPIRE:
significantly reduced Nasal Polyp Score (NPS) by -2.065 at week 52.
significantly reduced nasal congestion (measured by participant-reported Nasal Congestion Score [NCS]) by -1.028 at week 52.
Improvements in NPS were observed as early as week four, and NCS as early as week two, and were sustained through week 52.
significantly reduced the need for nasal polyp surgery by 98%.
significantly reduced the need for systemic corticosteroid treatment by 88%.
had a safety profile consistent with its approved severe asthma indication.
The FDA has accepted the regulatory submission of the WAYPOINT Phase 3 data with a PDUFA date of October 19, 2025.
A Phase 3 study of TEZSPIRE is enrolling patients with eosinophilic esophagitis.
Rocatinlimab (AMG 451/KHK4083)

Rocatinlimab is a first-in-class T-cell rebalancing monoclonal antibody that inhibits and reduces OX40-positive pathogenic T-cells.
The eight study ROCKET Phase 3 program evaluating rocatinlimab in patients with moderate-to-severe atopic dermatitis (AD) has enrolled over 3,300 patients. Enrollment is now complete in seven studies.
In March, detailed data were presented from the ROCKET HORIZON Phase 3 study, which met its co-primary and all key secondary endpoints.
In March, data were announced from three additional ROCKET program Phase 3 studies:
The IGNITE study, which evaluated two dose strengths of rocatinlimab, met its co-primary endpoints and all key secondary endpoints at week 24:
42.3% of patients in the higher dose group achieved ≥75% reduction from baseline in Eczema Area and Severity Index score (EASI-75), a 29.5% difference vs. placebo. In the lower dose group, 36.3% of patients achieved EASI-75, a 23.4% difference vs. placebo.
23.6% of patients in the higher dose group achieved a validated Investigator’s Global Assessment for Atopic Dermatitis (vIGA-AD) score of 0 (clear) or 1 (almost clear) with a ≥2-point reduction from baseline (vIGA-AD 0/1), a 14.9% difference vs. placebo. In the lower dose group, 19.1% of patients achieved this endpoint, a 10.3% difference vs. placebo.
22.7% of patients in the higher dose group achieved a revised Investigator’s Global Assessment (rIGA) score of 0/1 with a ≥2-point reduction from baseline (a more stringent measure of efficacy than vIGA-AD 0/1), a 14.4% difference vs. placebo. In the lower dose group, 16.3% of patients achieved this endpoint, an 8.0% difference vs. placebo.
The SHUTTLE study, which evaluated two dose strengths of rocatinlimab in combination with topical corticosteroids (TCS) and/or topical calcineurin inhibitors (TCI), met its co-primary endpoints and all key secondary endpoints at week 24.
The VOYAGER study, which successfully demonstrated that rocatinlimab does not interfere with responses to tetanus and meningococcal vaccinations.
Across ROCKET program results to date, safety findings were generally consistent with the previously observed safety profile of rocatinlimab. The most frequent treatment-emergent adverse events (≥5%) with higher observed proportion in rocatinlimab groups were pyrexia, chills and headache. A higher number of patients receiving rocatinlimab vs. placebo experienced gastrointestinal ulceration events, with an overall incidence of less than 1%.
Key upcoming milestones from the ROCKET Phase 3 program:
ROCKET ASCEND is a study evaluating rocatinlimab maintenance therapy in adult and adolescent patients with moderate-to-severe AD. Data readout is anticipated in H2 2025.
ROCKET ASTRO is a 52-week study evaluating rocatinlimab in adolescent patients with moderate-to-severe AD. Data readout is anticipated in H2 2025.
A Phase 2 study of rocatinlimab is enrolling patients with moderate-to-severe asthma.
A Phase 3 study of rocatinlimab is enrolling patients with prurigo nodularis (PN).
Blinatumomab

Blinatumomab is a bispecific T-cell engager (BiTE) molecule targeting CD19.
A Phase 2 study of blinatumomab in autoimmune disease was initiated in adults with systemic lupus erythematosus (SLE).
Inebilizumab

Inebilizumab is a B-cell depleting monoclonal antibody targeting CD19.
A Phase 2 study of inebilizumab in autoimmune disease was initiated in adults with SLE.
AMG 104 (AZD8630)

AMG 104 is an inhaled anti-thymic stromal lymphopoietin (TSLP) fragment antigen-binding (Fab).
A Phase 2 study is enrolling patients with asthma.
Oncology

BLINCYTO / blinatumomab

In April, the FDA granted Breakthrough Therapy Designation for subcutaneous blinatumomab in the treatment of adults with relapsed/refractory CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL).
A Phase 1/2 study of subcutaneous blinatumomab is ongoing in the dose-expansion and optimization phase in adult patients with relapsed or refractory Philadelphia chromosome (Ph)-negative B-ALL. The Company is planning to advance blinatumomab subcutaneous administration to a potentially registration-enabling Phase 2 portion of this study with initiation in H2 2025.
Golden Gate, a Phase 3 study of BLINCYTO alternating with low-intensity chemotherapy, is enrolling older adult patients with newly diagnosed Ph-negative B-ALL.
IMDELLTRA / tarlatamab

IMDELLTRA is the first and only FDA-approved delta-like ligand 3 (DLL3) targeting BiTE molecule.
In April, the Company announced that the global Phase 3 DeLLphi-304 study met its primary endpoint of improved overall survival at a planned interim analysis. This study evaluated IMDELLTRA compared to local standard-of-care chemotherapy as a treatment for patients with small cell lung cancer (SCLC) who progressed on or after a single line of platinum-based chemotherapy. The safety profile for IMDELLTRA was consistent with its known profile. Together these randomized data have the potential to establish IMDELLTRA as a new standard of care in second-line SCLC. Detailed data from DeLLphi-304 will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting (ASCO) (Free ASCO Whitepaper) in June.
The Company is advancing a comprehensive, global clinical development program across extensive-stage and limited-stage SCLC:
DeLLphi-303, a Phase 1b study of IMDELLTRA in combination with a programmed cell death protein ligand-1 (PD-L1) inhibitor, carboplatin and etoposide or separately in combination with PD-L1 alone, is ongoing in patients with first-line ES-SCLC.
DeLLphi-305, a Phase 3 study of IMDELLTRA and durvalumab, is enrolling patients with first-line ES-SCLC in the maintenance setting.
DeLLphi-306, a Phase 3 study of IMDELLTRA following concurrent chemoradiation therapy, is enrolling patients with limited-stage SCLC.
DeLLphi-308, a Phase 1b study evaluating subcutaneous tarlatamab, is enrolling patients with second line or later ES-SCLC.
DeLLphi-309, a Phase 2 study evaluating alternative intravenous dosing regimens in second-line ES-SCLC, is enrolling patients.
DeLLphi-310, a Phase 1b study, was initiated in patients with ES-SCLC evaluating IMDELLTRA in combination with YL201, a B7-H3 targeting antibody-drug conjugate, with or without anti-PD-L1.
DeLLphi-312, a Phase 3 study of first-line IMDELLTRA with carboplatin, etoposide and durvalumab is planned to initiate in patients with ES-SCLC by mid-2025.
Xaluritamig (AMG 509)

Xaluritamig is a first-in-class BiTE targeting six-transmembrane epithelial antigen of prostate 1 (STEAP1).
A Phase 3 study of xaluritamig is enrolling patients with metastatic castrate resistant prostate cancer (mCRPC) who have previously been treated with taxane-based chemotherapy.
A Phase 1 study of xaluritamig monotherapy has completed enrollment of patients with mCRPC who have not yet received taxane-based chemotherapy and has also completed enrollment of patients with mCPRC who have previously received taxane-based chemotherapy in a fully outpatient treatment setting to further improve administration convenience. This study continues to enroll mCRPC patients into a combination treatment of xaluritamig and abiraterone.
A Phase 1b study evaluating neoadjuvant xaluritamig therapy prior to radical prostatectomy is enrolling patients with newly diagnosed localized intermediate or high–risk prostate cancer.
A Phase 1b study of xaluritamig is enrolling patients with high-risk biochemically recurrent prostate cancer after definitive therapy.
AMG 193

AMG 193 is a first-in-class small molecule methylthioadenosine (MTA)-cooperative protein arginine methyltransferase 5 (PRMT5) inhibitor.
A Phase 2 study evaluating the efficacy, safety, tolerability and pharmacokinetics of AMG 193 is enrolling patients with methylthioadenosine phosphorylase (MTAP)-null previously treated advanced non-small cell lung cancer (NSCLC).
A Phase 1/1b/2 study of AMG 193 is enrolling patients with advanced MTAP-null solid tumors in the dose-expansion portion of the study.
A Phase 1b study of AMG 193 alone or in combination with other therapies is enrolling patients with advanced MTAP-null thoracic malignancies.
A Phase 1b study of AMG 193 in combination with other therapies is enrolling patients with advanced MTAP-null gastrointestinal, biliary tract and pancreatic cancers.
A Phase 1/2 study of AMG 193 in combination with IDE397, an investigational methionine adenosyltransferase 2A (MAT2A) inhibitor, will be discontinued following a wind-down period.
Bemarituzumab

Bemarituzumab is a first-in-class fibroblast growth factor receptor 2b (FGFR2b) targeting monoclonal antibody.
FORTITUDE-101, a Phase 3 study of bemarituzumab plus chemotherapy, is ongoing in patients with first-line gastric cancer. Data readout is anticipated in Q2 2025.
FORTITUDE-102, a Phase 1b/3 study of bemarituzumab plus chemotherapy and nivolumab, is ongoing in patients with first-line gastric cancer. Phase 3 data readout is anticipated in H2 2025.
FORTITUDE-103, a Phase 1b/2 study of bemarituzumab plus oral chemotherapy regimens with or without nivolumab, is enrolling patients with first-line gastric cancer.
FORTITUDE-301, a Phase 1b/2 basket study of bemarituzumab monotherapy, is ongoing in patients with solid tumors with FGFR2b overexpression.
LUMAKRAS/LUMYKRAS

CodeBreaK 301, a Phase 3 study of LUMAKRAS in combination with Vectibix and FOLFIRI, is enrolling patients with first-line KRAS G12C–mutated CRC.
CodeBreaK 202 (CB202), a Phase 3 study of LUMAKRAS plus chemotherapy vs. pembrolizumab plus chemotherapy, is enrolling patients with first-line KRAS G12C–mutated and PD-L1 negative advanced NSCLC.
Nplate

The final analysis of a Phase 3 study of Nplate as supportive care in chemotherapy-induced thrombocytopenia in gastrointestinal malignancies is complete. Data from this study will be presented at ASCO (Free ASCO Whitepaper) in June.
Biosimilars

A randomized, double-blind pharmacokinetic similarity study of ABP 206 compared with OPDIVO (nivolumab) has completed enrollment of patients with resected stage III or stage IV melanoma in the adjuvant setting. Data readout is anticipated in H2 2025.
A randomized, double-blind comparative clinical study of ABP 206 compared with OPDIVO is enrolling patients with treatment-naïve unresectable or metastatic melanoma.
A randomized, double-blind pharmacokinetic similarity study of ABP 234 compared with KEYTRUDA (pembrolizumab) is enrolling patients with early-stage non-squamous non-small cell lung cancer as adjuvant treatment.
A randomized, double-blind combined pharmacokinetic/comparative clinical study of ABP 234 compared to KEYTRUDA is enrolling patients with advanced or metastatic non-squamous non-small cell lung cancer.
A randomized, double-blind pharmacokinetic similarity/comparative clinical study of ABP 692 compared to OCREVUS (ocrelizumab) was initiated and is currently enrolling patients with relapsing-remitting multiple sclerosis.
TEZSPIRE is being developed in collaboration with AstraZeneca.
AMG 104 is being developed in collaboration with AstraZeneca.
Rocatinlimab, formerly AMG 451/KHK4083, is being developed in collaboration with Kyowa Kirin.
Xaluritamig, formerly AMG 509, is being developed pursuant to a research collaboration with Xencor, Inc.
IDE397 is an investigational MAT2A inhibitor from IDEAYA Biosciences.
YL201 is an investigational B7-H3 targeting antibody-drug conjugate being developed by MediLink.
OPDIVO is a registered trademark of Bristol-Myers Squibb Company.
KEYTRUDA is a registered trademark of Merck & Co., Inc.
OCREVUS is a registered trademark of Genentech, Inc.

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.