Castle Biosciences Announces Preliminary Unaudited Fourth Quarter and Full-Year 2024 Results




Castle Biosciences Announces Preliminary Unaudited Fourth Quarter and Full-Year 2024 Results

2024 total revenue expected to meet or exceed top end of guided range of $320-330 million, at least 50% growth over 2023

Delivered 96,071 total test reports in 2024, an increase of 36% compared to 2023

Year-end 2024 cash, cash equivalents and marketable investment securities expected to be approximately $293 million

FRIENDSWOOD, Texas–(BUSINESS WIRE)–Castle Biosciences, Inc. (Nasdaq: CSTL), a company improving health through innovative tests that guide patient care, today announced certain unaudited preliminary performance results for the fourth quarter and year ended Dec. 31, 2024.

“Our strong fourth quarter performance underscores continued momentum built throughout 2024, reflecting the strength of our growth initiatives and the dedication of our team,” said Derek Maetzold, president and chief executive officer of Castle Biosciences. “As a result, we expect to meet or exceed the top end of our full-year 2024 revenue guidance of $320-330 million. This achievement reflects our commitment to delivering value to our stockholders while advancing our mission of improving health through innovative tests that guide patient care.”

Preliminary, Unaudited Fourth Quarter Ended Dec. 31, 2024, Highlights

• Delivered 24,071 total test reports in the fourth quarter of 2024, compared to 20,284 in the same period of 2023, an increase of 19%:
  • DecisionDx®-Melanoma test reports delivered in the quarter were 8,672, compared to 8,591 in the fourth quarter of 2023, an increase of 1%.
  • DecisionDx®-SCC test reports delivered in the quarter were 4,299, compared to 3,530 in the fourth quarter of 2023, an increase of 22%.
  • MyPath® Melanoma test reports delivered in the quarter were 879, compared to 1,018 in the fourth quarter of 2023, a decrease of 14%.
  • TissueCypher® Barrett’s Esophagus test reports delivered in the quarter were 6,672 compared to 3,441 in the fourth quarter of 2023, an increase of 94%.
  • IDgenetix® test reports delivered in the quarter were 3,125 compared to 3,299 in the fourth quarter of 2023, a decrease of 5%. In late 2024, the Company made modifications to its promotional investments for IDgenetix, shifting resources to inside sales and non-personal promotion.
  • DecisionDx®-UM test reports delivered in the quarter were 424, compared to 405 in the fourth quarter of 2023, an increase of 5%.

Preliminary, Unaudited Year Ended Dec. 31, 2024, Highlights

• The Company expects to meet or exceed top end of full-year 2024 revenue guidance of $320-330 million.
• Delivered 96,071 total test reports in 2024, compared to 70,429 in the same period of 2023, an increase of 36%:
  • DecisionDx-Melanoma test reports delivered in 2024 were 36,008, compared to 33,330 in 2023, an increase of 8%.
  • DecisionDx-SCC test reports delivered in 2024 were 16,348, compared to 11,442 in 2023, an increase of 43%.
  • MyPath Melanoma test reports delivered in 2024 were 3,909, compared to 3,962 in 2023, a decrease of 1%.
  • TissueCypher Barrett’s Esophagus test reports delivered in 2024 were 20,956 compared to 9,100 in 2023, an increase of 130%.
  • IDgenetix test reports delivered in 2024 were 17,151, compared to 10,921 in 2023, an increase of 57%.
  • DecisionDx-UM test reports delivered in 2024 were 1,699, compared to 1,674 in 2023, an increase of 1%.

Cash, Cash Equivalents and Marketable Investment Securities

Year-end 2024 cash and cash equivalents are expected to be approximately $120 million. Additionally, the Company estimates that it held approximately $173 million in marketable investment securities as of year-end 2024.

Recent Publication Highlight

Findings from the prospective, multicenter DECIDE study demonstrating the significant impact of the DecisionDx-Melanoma test on sentinel lymph node biopsy (SLNB) decision-making for patients with melanoma were recently published in the World Journal of Surgical Oncology. The data showed that integrating DecisionDx-Melanoma test results into the treatment decision-making process resulted in 18.5% fewer SLNBs performed compared to a matched patient cohort for whom the test was not used to guide SLNB decisions (p<0.001). Additionally, no patient with a DecisionDx-Melanoma-predicted risk of SLN positivity of less than 5% who decided to have an SLNB procedure had a positive node.

Recent Reimbursement Update

On January 9, 2025, Medicare Administrative Contractor, Novitas, finalized a local coverage determination that includes language signifying non-coverage by Medicare for our DecisionDx-SCC test.

About Castle Biosciences

Castle Biosciences (Nasdaq: CSTL) is a leading diagnostics company improving health through innovative tests that guide patient care. The Company aims to transform disease management by keeping people first: patients, clinicians, employees and investors.

Castle’s current portfolio consists of tests for skin cancers, Barrett’s esophagus, mental health conditions and uveal melanoma. Additionally, the Company has active research and development programs for tests in these and other diseases with high clinical need, including its test in development to help guide systemic therapy selection for patients with moderate-to-severe atopic dermatitis seeking biologic treatment. To learn more, please visit www.CastleBiosciences.com and connect with us on LinkedIn, Facebook, X and Instagram.

DecisionDx-Melanoma, DecisionDx-CMSeq, i31-SLNB, i31-ROR, DecisionDx-SCC, MyPath Melanoma, DiffDx-Melanoma, TissueCypher, IDgenetix, DecisionDx-UM, DecisionDx-PRAME and DecisionDx-UMSeq are trademarks of Castle Biosciences, Inc.

Preliminary Results

Castle Biosciences has not completed the preparation of its financial statements for the fourth quarter or year ended Dec. 31, 2024. The preliminary, unaudited information presented in this press release for the quarter and year ended Dec. 31, 2024 is based on management’s initial review of the information presented and its current expectations and is subject to adjustment as a result of, among other things, the completion of the Company’s end-of-period reporting processes and related activities, including the audit by the Company’s independent registered public accounting firm of the Company’s financial statements. As such, any financial information contained in this press release may differ materially from the information reflected in the Company’s financial statements as of and for the year ended Dec. 31, 2024. Additional information and disclosures would be required for a more complete understanding of the Company’s financial position and results of operations as of and for the quarter and year ended Dec. 31, 2024. Accordingly, undue reliance should not be placed on this preliminary information.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, which are subject to the “safe harbor” created by those sections. These forward-looking statements include, but are not limited to, statements concerning our expectations regarding: (i) our full year 2024 revenue guidance of $320-330 million; (ii) the accuracy of our preliminary test report counts both for full year and fourth quarter of 2024; (iii) trends in revenues and test report volumes; (iv) the accuracy of our expected year-end 2024 cash and cash equivalents and marketable investment securities; (v) the ability of DecisionDx-Melanoma to have a significant impact on SLNB decision-making for patients with melanoma and (vi) future coverage by Medicare for our DecisionDx-SCC test. The words “anticipate,” “can,” “could,” “expect,” “goal,” “may,” “plan” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. We may not actually achieve the plans, intentions, or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make. These forward-looking statements involve risks and uncertainties that could cause our actual results to differ materially from those in the forward-looking statements, including, without limitation: our assumptions or expectations regarding continued reimbursement for our products and subsequent coverage decisions; Novitas’ local coverage determination signifying non-coverage by Medicare of our DecisionDx-SCC test; our estimated total addressable markets for our products and product candidates; the expenses, capital requirements and potential needs for additional financing; the anticipated cost, timing and success of our product candidates; our plans to research, develop and commercialize new tests; our ability to successfully integrate new businesses, assets, products or technologies acquired through acquisitions; the effects of macroeconomic events and conditions, including inflation and monetary supply shifts, labor shortages, liquidity concerns at, and failures of, banks and other financial institutions or other disruptions in the banking system or financing markets, recession risks, supply chain disruptions, outbreaks of contagious diseases and geopolitical events (such as the ongoing Israel-Hamas War and Ukraine-Russia conflict), among others, on our business and our efforts to address its impact on our business; the possibility that subsequent study or trial results and findings may contradict earlier study or trial results and findings or may not support the results discussed in this press release, including with respect to the tests discussed in this press release; our planned installation of additional equipment and supporting technology infrastructures and implementation of certain process efficiencies may not enable us to increase the future scalability of our TissueCypher Test; the possibility that the actual application of our tests may not provide the aforementioned benefits to patients; the possibility that our newer gastroenterology and mental health franchises may not contribute to the achievement of our long-term financial targets as anticipated; and the risks set forth under the heading “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2023 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, each filed with the SEC, and in our other filings with the SEC. The forward-looking statements are applicable only as of the date on which they are made, and we do not assume any obligation to update any forward-looking statements, except as may be required by law.

Contacts

Investor Relations Contact:
Camilla Zuckero

czuckero@castlebiosciences.com
281-906-3868

Media Contact:
Allison Marshall

amarshall@castlebiosciences.com

Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings




Vertex Provides Pipeline and Business Updates in Advance of Upcoming Investor Meetings

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the company’s scheduled webcast from the 43^rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT.

“2024 marked another year of excellent progress for Vertex, as we reached more people with CF than ever before, began a new era of commercial diversification, and advanced and broadened our clinical stage pipeline,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “In 2025, we are poised to diversify our business further across multiple dimensions: our revenue, as we continue the launches of CASGEVY, ALYFTREK and potentially launch suzetrigine in acute pain; our pipeline, as we progress four potentially transformative medicines through pivotal trials; and our geographic footprint, as we expand both our commercial and clinical presence globally.”

Disease Areas with Approved Medicines

Cystic Fibrosis (CF)

• ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) approved in the U.S.: On December 20, 2024, Vertex secured FDA approval for ALYFTREK, the once-daily next-in-class combination CFTR modulator for the treatment of people with CF 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK, which includes a total of 303 mutations. Global regulatory submissions for ALYFTREK, including in the U.K. and Europe, are currently under review.
• TRIKAFTA: Also on December 20, 2024, Vertex received FDA approval for the expanded use of TRIKAFTA in patients with 94 additional non-F508del CFTR mutations. With this approval, approximately 300 people in the U.S. are newly eligible for a medicine that treats the underlying cause of their disease. TRIKAFTA is now approved for patients with a total of 272 CFTR mutations.
• VX-522: The multiple ascending dose (MAD) portion of the Phase 1/2 study of VX-522 is underway, with data expected in the first half of 2025. VX-522 is a CFTR mRNA therapeutic that Vertex is developing in collaboration with Moderna for the more than 5,000 people with CF who cannot benefit from CFTR modulators.
• Epidemiology and market opportunity update: Vertex increased its estimates for the number of people with cystic fibrosis in the U.S., Europe, Australia, and Canada from approximately 92,000 to approximately 94,000. Additionally, Vertex continues to secure formal reimbursement for eligible patients in multiple countries that collectively comprise approximately 15,000 additional patients, of whom approximately 10,000 are eligible for treatment with CFTR modulators. Vertex previously served many of these markets through named patient sales.

Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT) – CASGEVY

• As of the end of 2024, Vertex has activated more than 50 authorized treatment centers (ATCs) globally and more than 50 patients have initiated cell collection.
• On December 31, 2024, Vertex received regulatory approval for CASGEVY in the United Arab Emirates (UAE) for the treatment of both SCD and TDT.
• In the U.S., Vertex recently negotiated a first-of-its-kind, voluntary agreement with the Centers for Medicare & Medicaid Services (CMS), which will provide a single outcomes-based arrangement for CASGEVY, available to all state Medicaid programs to ensure broad and equitable access for patients.

Pipeline Disease Areas

Acute Pain

• Suzetrigine: The FDA has assigned a PDUFA target action date of January 30, 2025, for suzetrigine for the treatment of moderate-to-severe acute pain. Suzetrigine was granted Priority Review by the FDA.
• The Non-Opioids Prevent Addiction In the Nation (NOPAIN) Act became effective on January 1^st, 2025. The NOPAIN Act mandates that Medicare provide a separate add-on payment in the hospital outpatient or surgical center setting for FDA-approved non-opioid treatments for pain. Vertex expects suzetrigine in acute pain to be included on the list of treatments that qualify for add-on payment under this act, following potential suzetrigine FDA approval.
• Seven states have recently enacted legislation into law for the retail setting, specifying that opioids are not preferred over non-opioid therapies for the treatment of pain.

Peripheral Neuropathic Pain (PNP)

• Suzetrigine: Vertex continues to enroll and dose patients with diabetic peripheral neuropathy (DPN) in a Phase 3 pivotal trial of suzetrigine.
• Following the December 2024 release of Phase 2 results with suzetrigine in painful lumbosacral radiculopathy (LSR), a form of peripheral neuropathic pain, Vertex plans to advance suzetrigine into pivotal development for painful LSR, pending discussions with regulators on the study design and regulatory package.

IgA Nephropathy (IgAN) and other B Cell-Mediated Diseases

• The global Phase 3 RAINIER study of povetacicept is enrolling and dosing patients with IgAN in the U.S., Europe and Asia. Vertex expects to complete enrollment in the interim analysis cohort in 2025 for potential accelerated approval in the U.S., once this cohort reaches 36 weeks of treatment.
• Vertex has entered into an exclusive collaboration and license agreement with Zai Lab for the development and commercialization of povetacicept in mainland China, Hong Kong, Macau, Taiwan, and Singapore. Zai Lab will help advance clinical trials and make regulatory submissions in the licensed territory, and they will also be responsible for all commercialization activities in the licensed territory upon potential approval of povetacicept.

APOL1-Mediated Kidney Disease (AMKD) – Inaxaplin (VX-147)

• Vertex continues to enroll and dose patients with primary AMKD in the Phase 3 portion of the AMPLITUDE global Phase 2/3 pivotal clinical trial of inaxaplin, in which a 45 mg once-daily dose of inaxaplin is compared to placebo, on top of standard of care. Vertex expects to complete enrollment in the interim analysis cohort in 2025 for potential accelerated approval in the U.S., once this cohort reaches 48 weeks of treatment.
• Vertex plans to initiate AMPLIFIED, a Phase 2b open-label study of inaxaplin in patients with AMKD and diabetes or other co-morbidities currently not eligible for the AMPLITUDE Phase 2/3 pivotal trial, expanding the estimated potentially eligible population from 150,000 to 250,000 patients.

Type 1 Diabetes (T1D)

• Zimislecel (VX-880): Following successful end of Phase 2 meetings with the FDA, the European Medicines Agency (EMA), and the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Vertex initiated the Phase 3 portion of the Phase 1/2/3 study of zimislecel in patients with T1D with severe hypoglycemic events and impaired awareness of hypoglycemia. Vertex expects to complete enrollment and dosing of the pivotal study in 2025.
• Epidemiology update: Vertex estimates that a total of 125,000 patients have severe T1D, out of the estimated 3.8M people with T1D in North American and Europe. Vertex expects the initial zimislecel indication will address approximately 60,000 patients and is working to serve all 125,000 patients with severe diabetes over time.
• Consistent with its commitment to serial innovation and bringing transformative therapies to all patients who can benefit, Vertex is developing additional therapies for T1D that use the same cells that are used in zimislecel. This includes VX-264, currently in a Phase 1/2 study, in which the cells are encapsulated in an immunoprotective device. Vertex plans to share Part B full-dose data from the VX-264 Phase 1/2 study in 2025. Vertex is also pursuing alternative approaches to immunosuppression that could be used with zimislecel, as well as a hypoimmune program utilizing gene-edited stem-cell derived islets.

Myotonic Dystrophy Type 1 (DM1) – VX-670

• Vertex has completed the single ascending dose (SAD) portion of the global Phase 1/2 clinical trial for VX-670 in people with DM1 and initiated the MAD portion of the Phase 1/2 study, which will assess both safety and efficacy.

Autosomal Dominant Polycystic Kidney Disease (ADPKD) – VX-407

• Vertex is enrolling and dosing a Phase 1 study of healthy volunteers with VX-407. Vertex expects to advance VX-407 into a Phase 2 proof of concept study in people with ADPKD in 2025.

J.P. Morgan Healthcare Conference Presentation and Webcast

Dr. Kewalramani will present at the 43^rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 10:30 a.m. ET/7:30 a.m. PT.

A live webcast of management’s remarks will be available through the Vertex website, www.vrtx.com, in the “Investors” section under the “News and Events” page. A replay of the conference webcast will be archived on the company’s website.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 14 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Reshma Kewalramani, M.D., and statements about our expectations for our CF program, including with respect to the diversification of Vertex’s business and expanding commercially and clinically across more geographies, commercial expectations for ALYFTREK, the expectation to have data from the Phase 1/2 study of VX-522, expectations that VX-522 may treat >5,000 people with CF, the company’s beliefs regarding CF epidemiology and market opportunities, expectations for the company’s agreement with CMS and resulting patient access to CASGEVY, expectations that suzetrigine in acute pain will be included on the list of treatments that qualify for add-on payments under the NOPAIN Act, plans to advance suzetrigine into pivotal development for painful LSR, expectations regarding povetacicept in IgAN, including completing enrollment in the interim analysis cohort in 2025 for potential accelerated approval, expectations for the collaboration with Zai Lab, including the future activities of the parties pursuant to the collaboration, expectations regarding inaxaplin in AMKD, including that the company will complete enrollment in the interim analysis cohort in 2025 for potential accelerated approval in the U.S., plans to initiate a Phase 2b open-label study of inaxaplin in patients with AMKD and diabetes or other co-morbidities and expanding the eligible patient population, expectations regarding completion of enrollment and dosing in the pivotal study evaluating zimislecel in 2025, expectations regarding the initial eligible patient population that will benefit from zimislecel, plans to work with urgency to advance zimislecel to be able to serve all patients with severe T1D, plans to develop additional therapies for T1D, plans to share data from the VX-264 Phase 1/2 study in 2025, plans to pursue alternative approaches to immunosuppression that could be used with zimislecel and other T1D product candidates, and expectations to advance VX-407 into a Phase 2 proof of concept study in people with ADPKD in 2025. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from a limited number of patients may not be indicative of final clinical trial results, that clinical trial data might not be available on the expected timeline, that the anticipated benefits and potential of Vertex’s collaboration with Zai Lab may not be achieved on the anticipated timeline, or at all, that data from the company’s research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks, that our discussions with regulators may be delayed or cause delays in our pipeline programs, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company’s website at www.vrtx.com. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Contacts

Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
or

617-961-7163

Media:
mediainfo@vrtx.com
or

International: +44 20 3204 5275

or

U.S.: 617-341-6992

Revvity to Hold Earnings Call on Friday, January 31, 2025; Provides Update on Financial Performance




Revvity to Hold Earnings Call on Friday, January 31, 2025; Provides Update on Financial Performance

WALTHAM, Mass.–(BUSINESS WIRE)–Revvity, Inc. (NYSE: RVTY), today announced that it will release its fourth quarter and full year 2024 financial results prior to market open on Friday, January 31, 2025. The Company will host a conference call the same day at 8:00 a.m. ET to discuss these results. Prahlad Singh, president and chief executive officer, and Max Krakowiak, chief financial officer, will host the conference call.

To access the call, a live audio webcast will be available via this registration form or on the Investors section of the Company’s website.

Update on Financial Performance

The Company is also providing the following preliminary financial results for the fourth quarter 2024:

• Reported and organic revenue growth for the fourth quarter is expected to be approximately 5% and 6%, respectively, as compared to the same period a year ago. This is expected to result in fourth quarter revenue of approximately $730 million.

The Company also now expects its full year 2024 adjusted earnings per share to meet or exceed the guidance provided on November 4, 2024.

J.P. Morgan Healthcare Conference

As previously announced, Prahlad Singh, will present at 8:15 a.m. PT tomorrow (January 13, 2025) at the 43^rd annual J.P. Morgan Healthcare Conference. A live audio webcast of the presentation will be available via this page. A copy of the accompanying slide presentation will be made available on the Investors section of the Company’s website.

Use of Non-GAAP Financial Measures

In addition to financial measures prepared in accordance with generally accepted accounting principles (GAAP), this earnings announcement also contains non-GAAP financial measures. The reasons that we use these measures, a reconciliation of these measures to the most directly comparable GAAP measures, and other information relating to these measures are included below under Explanation of Non-GAAP Financial Measures.

Full year 2024 adjusted earnings per share guidance is provided on a non-GAAP basis and cannot be reconciled to the closest GAAP measures without unreasonable effort due to the unpredictability of the amounts and timing of events affecting the items the Company excludes from these non-GAAP measures. The timing and amounts of such events and items could be material to the Company’s results prepared in accordance with GAAP.

Factors Affecting Future Performance

This press release contains “forward-looking” statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements relating to estimates and projections of future adjusted earnings per share, revenue, revenue growth and other financial results. Words such as “believes,” “intends,” “anticipates,” “plans,” “expects,” “estimates,” “projects,” “forecasts,” “will” and similar expressions, and references to guidance, are intended to identify forward-looking statements. Such statements are based on management’s current assumptions and expectations and no assurances can be given that our assumptions or expectations will prove to be correct. A number of important risk factors could cause actual results to differ materially from the results described, implied or projected in any forward-looking statements. These factors include, without limitation: the completion of quarterly and year end closing procedures for the fourth quarter and fiscal year ended December 29, 2024 and other factors which we describe under the caption “Risk Factors” in our most recent quarterly report on Form 10-Q and in our other filings with the Securities and Exchange Commission. We disclaim any intention or obligation to update any forward-looking statements as a result of developments occurring after the date of this press release.

About Revvity

At Revvity, “impossible” is inspiration, and “can’t be done” is a call to action. Revvity provides health science solutions, technologies, expertise, and services that deliver complete workflows from discovery to development, and diagnosis to cure. Revvity is revolutionizing what’s possible in healthcare, with specialized focus areas in translational multi-omics technologies, biomarker identification, imaging, prediction, screening, detection and diagnosis, informatics and more.

With 2023 revenue of more than $2.7 billion and over 11,000 employees, Revvity serves customers across pharmaceutical and biotech, diagnostic labs, academia and governments. It is part of the S&P 500 index and has customers in more than 190 countries.

Stay updated by following our Newsroom, LinkedIn, X, YouTube, Facebook and Instagram.

Explanation of Non-GAAP Financial Measures

We report our financial results in accordance with GAAP. However, management believes that, in order to more fully understand our short-term and long-term financial and operational trends, investors may wish to consider the impact of certain non-cash, non-recurring or other items, which result from facts and circumstances that vary in frequency and impact on continuing operations. Accordingly, we present non-GAAP financial measures as a supplement to the financial measures we present in accordance with GAAP. These non-GAAP financial measures provide management with additional means to understand and evaluate the operating results and trends in our ongoing business by adjusting for certain non-cash expenses and other items that management believes might otherwise make comparisons of our ongoing business with prior periods more difficult, obscure trends in ongoing operations, or reduce management’s ability to make useful forecasts. Management believes these non-GAAP financial measures provide additional means of evaluating period-over-period operating performance. In addition, management understands that some investors and financial analysts find this information helpful in analyzing our financial and operational performance and comparing this performance to our peers and competitors.

We use the term “organic revenue” to refer to GAAP revenue, excluding the effect of foreign currency changes and revenue from recent acquisitions and divestitures and including purchase accounting adjustments for revenue from contracts acquired in acquisitions that will not be fully recognized due to accounting rules. We use the related term “organic revenue growth” or “organic growth” to refer to the measure of comparing current period organic revenue with the corresponding period of the prior year.

Management includes or excludes the effect of the item identified below in the applicable non-GAAP financial measure referenced above for the reasons set forth below with respect to that item:

• Revenue from contracts acquired in acquisitions that will not be fully recognized due to accounting rules — accounting rules require us to account for the fair value of revenue from contracts assumed in connection with our acquisitions. As a result, our GAAP results reflect the fair value of those revenues, which is not the same as the revenue that otherwise would have been recorded by the acquired entity. We include such revenue in our non-GAAP measures because we believe the fair value of such revenue does not accurately reflect the performance of our ongoing operations for the period in which such revenue is recorded.

The non-GAAP financial measures described above are not meant to be considered superior to, or a substitute for, our financial statements prepared in accordance with GAAP. There are material limitations associated with non-GAAP financial measures because they exclude charges that have an effect on our reported results and, therefore, should not be relied upon as the sole financial measures by which to evaluate our financial results. Management compensates and believes that investors should compensate for these limitations by viewing the non-GAAP financial measures in conjunction with the GAAP financial measures. In addition, the non-GAAP financial measures included in this earnings announcement may be different from, and therefore may not be comparable to, similar measures used by other companies.

Each of the non-GAAP financial measures listed above is also used by our management to evaluate our operating performance, communicate our financial results to our Board of Directors, benchmark our results against our historical performance and the performance of our peers, evaluate investment opportunities including acquisitions and discontinued operations, and determine the bonus payments for senior management and employees.

Contacts

Investor Relations:
Steve Willoughby

steve.willoughby@revvity.com

Media Relations:
Chet Murray

(781) 462-5126

chet.murray@revvity.com

Exelixis Announces Preliminary Fiscal Year 2024 Financial Results, Provides 2025 Financial Guidance and Outlines Key Priorities and Milestones for 2025




Exelixis Announces Preliminary Fiscal Year 2024 Financial Results, Provides 2025 Financial Guidance and Outlines Key Priorities and Milestones for 2025

– Cabozantinib franchise achieves approximately $1.805 billion in preliminary U.S. net product revenues for fiscal year 2024 –

– Fiscal year 2025 net product revenues guidance of $1.95 billion – $2.05 billion –

– Presentation and webcast at 43^rd Annual J.P. Morgan Healthcare Conference tomorrow, Monday, January 13^th at 5:15 p.m. PT / 8:15 p.m. ET –

ALAMEDA, Calif.–(BUSINESS WIRE)–Exelixis, Inc. (Nasdaq: EXEL) today announced its preliminary unaudited financial results for the fiscal year 2024, provided financial guidance for fiscal year 2025 and delivered an update on its business. Exelixis anticipates 2025 will be a year of clinical and regulatory execution and continued growth for its cabozantinib franchise, as well as multiple data readouts for zanzalintinib and across its diversified pipeline of small molecules and biotherapeutics with the potential to improve standards of care for patients with cancer.

Preliminary Fiscal Year 2024 Financial Results & 2025 Financial Guidance

Exelixis is providing the following preliminary unaudited 2024 financial results and financial guidance for 2025. Net product and total revenues guidance do not currently reflect any revenues resulting from a potential U.S. regulatory approval and commercial launch of CABOMETYX^® (cabozantinib) for the treatment of patients with previously treated advanced neuroendocrine tumors (NET). The U.S. Food and Drug Administration (FDA) is currently reviewing Exelixis’ supplemental New Drug Application (sNDA) for this proposed indication, with a Prescription Drug User Fee Act (PDUFA) target action date of April 3, 2025.

The preliminary 2024 financial information presented in this press release has not been audited and is subject to change. The complete Exelixis Fourth Quarter and Fiscal Year 2024 Financial Results are planned for release after market on Tuesday, February 11, 2025.

“Entering 2025, Exelixis stands at an inflection point as we work toward our goal of building a multi-product, multi-franchise oncology business,” said Michael M. Morrissey, Ph.D., President & CEO, Exelixis. “Exelixis had a very successful 2024 highlighted by strong commercial and financial performance, the favorable ruling on our cabozantinib patent litigation, accelerating progress with the zanzalintinib pivotal trial program and establishing our zanzalintinib clinical development collaboration with Merck. We’re carrying that momentum into the new year as we seek to grow cabozantinib franchise revenues, accelerate and expand our zanzalintinib pivotal development program, and advance our diversified therapeutic pipeline of small molecules and biotherapeutics.”

Dr. Morrissey continued: “We expect 2025 to be a year of regulatory, clinical and commercial execution as we work toward a potential regulatory approval and launch for cabozantinib in neuroendocrine tumors and prepare for multiple zanzalintinib and pipeline data readouts throughout the year. As cabozantinib’s commercial success drives the business forward in the near-term, we’re excited by zanzalintinib’s potential to surpass cabozantinib’s scope and scale in the coming years and to become an important component of our mid- and long-term revenue growth. We’re also optimizing our earlier stage pipeline, rapidly profiling compounds and advancing only those with the highest probability of success into full development. We look forward to providing more detailed updates on our pipeline progress at an R&D Day later this year. Finally, we’ll maintain our balanced approach to capital allocation, leveraging our strong balance sheet to execute on business development opportunities within the GU and GI oncology space, while using free cash flows to fund our stock repurchase program and return capital to shareholders.”

Corporate Updates

Stock Repurchase Program Update. In August 2024, Exelixis announced that the company’s Board of Directors authorized the repurchase of up to $500 million of the company’s common stock through the end of 2025, the third stock repurchase program undertaken by Exelixis since March 2023. Under this program, as of the end of fiscal year 2024, Exelixis has repurchased $205.6 million of the company’s common stock, at an average price of $33.62 per share.

Anticipated Cabozantinib Milestones

Potential Label Expansion and Commercial Launch into NET. Exelixis is preparing for the potential commercial launch of CABOMETYX for the treatment of patients with previously treated advanced NET following the FDA’s acceptance of its sNDA and assignment of a PDUFA target action date of April 3, 2025. In January 2025, the FDA notified Exelixis that its sNDA will no longer be the subject of discussion at an Oncologic Drugs Advisory Committee meeting. The regulatory filing was based on positive results from the phase 3 CABINET pivotal trial sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health, and led by the NCI-funded Alliance for Clinical Trials in Oncology. CABINET met its primary endpoint, demonstrating statistically significant and clinically meaningful improvements in progression-free survival (PFS) for patients treated with cabozantinib as compared to placebo in both its pancreatic NET (pNET) and extra-pancreatic NET (epNET) cohorts. Final results from the trial were subsequently presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in The New England Journal of Medicine. In January 2025, the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology for NET were updated to include cabozantinib as category 1 for certain types of NET following specific treatments, and as a category 2A preferred regimen for several other forms of advanced NET, depending on site of origin and grade. A subgroup analysis from CABINET detailing the experience of patients with advanced gastrointestinal (GI) NET will also be presented at the American Society of Clinical Oncology GI Cancers Symposium (ASCO GI 2025) later this month. Exelixis’ partner Ipsen anticipates a decision from the European Medicines Agency on its Marketing Authorization Application for its own proposed NET label expansion in the EU for cabozantinib in 2025. While Exelixis prioritizes supporting the FDA’s ongoing review of its proposed NET indication, the company will continue to evaluate the timing of its potential regulatory filing for cabozantinib in metastatic castration-resistant prostate cancer based on the phase 3 CONTACT-02 pivotal study.

Anticipated Development Milestones

Expansion and Acceleration of the Zanzalintinib Pivotal Trial Program. Zanzalintinib is a third-generation tyrosine kinase inhibitor (TKI) that Exelixis believes can become the vascular endothelial growth factor receptor TKI of choice as the solid tumor therapeutic landscapes continue to evolve. The zanzalintinib pivotal development program currently consists of six ongoing or planned pivotal trials, with additional studies to be announced in 2025 and beyond:

• STELLAR-303 is evaluating zanzalintinib in combination with atezolizumab compared with regorafenib in patients with metastatic, refractory non-microsatellite instability-high or non-mismatch repair-deficient colorectal cancer (CRC). The primary endpoint in the study is overall survival (OS) in the patients without liver metastases (NLM). If OS is positive in the NLM population, the study will evaluate OS in the intent-to-treat population that includes patients with and without liver metastases. The study completed enrollment in the third quarter of 2024, and preliminary results are expected in the second half of 2025, dependent on study event rates.
• STELLAR-304 is evaluating zanzalintinib in combination with nivolumab versus sunitinib in previously untreated patients with advanced non-clear cell renal cell carcinoma. The primary endpoints in the trial are PFS and objective response rate. Based on current enrollment status in the trial, the primary endpoint of PFS is expected to be available in the second half of 2025, dependent on study event rates.
• STELLAR-305 is evaluating zanzalintinib in combination with pembrolizumab versus pembrolizumab alone in patients with previously untreated PD-L1-positive recurrent or metastatic squamous cell carcinoma of the head and neck. The study was designed to enroll approximately 250 eligible patients in the phase 2 portion of the trial to be randomly assigned to zanzalintinib in combination with pembrolizumab or pembrolizumab alone to evaluate the activity of the combination therapy. Data from the phase 2 portion are expected be available in the second half of 2025, which would inform whether the data support expansion into the phase 3 portion of the trial, during which an additional 350 patients would be randomized for a total of 600 patients. The primary endpoints in the study are PFS and OS.
• Exelixis also expects to initiate STELLAR-311, a phase 3 pivotal trial evaluating zanzalintinib compared with everolimus as a first oral therapy in patients with advanced NET, regardless of site of origin, in the first half of 2025.
• Additionally, as part of Exelixis’ clinical development collaboration with Merck, two pivotal renal cell carcinoma (RCC) studies are planned for 2025. The companies will provide further details on these trials closer to their initiation.

Earlier Stage Zanzalintinib Data Readouts Expected This Year. Exelixis anticipates initial clinical data readouts from zanzalintinib’s phase 1b/2 STELLAR-001 and STELLAR-002 clinical studies in the first half of 2025, including data from CRC and RCC cohorts. STELLAR-001 and -002 are evaluating zanzalintinib as a monotherapy and in potentially best-in-class combination regimens across various tumor types. In the nearest term, at ASCO GI 2025 later this month, investigators will present preliminary results from a randomized expansion cohort of STELLAR-001 designed to assess the contribution of atezolizumab to zanzalintinib in patients with previously treated metastatic CRC.

Advance XL309 Phase 1 Program in PARP Inhibitor Refractory Setting and Beyond. XL309, Exelixis’ potentially best-in-class small molecule inhibitor of USP1, is currently being evaluated in a phase 1 study as a single agent and in combination with olaparib, a PARP1/2 inhibitor, in patients with advanced solid tumors. Enrollment in the dose escalation cohorts for XL309 monotherapy and olaparib combination are ongoing. The mechanism of action of XL309 and its potential to combine with PARP-inhibitors (PARPi) provide optionality for a robust development program in a variety of solid tumors. Exelixis’ clinical development plans for XL309 include its development as a potential therapy for tumors that have become refractory to PARPi therapy, including forms of ovarian, breast and prostate cancers, pursuing potential PARPi combinations, and moving beyond the PARPi market into new areas. Exelixis plans to present data from the XL309 program at a scientific meeting in 2025.

Progress of Phase 1 Clinical Trials for XB010 and XL495. Exelixis initiated clinical development of its XB010 and XL495 pipeline programs in 2024. The company plans to rapidly profile each compound to determine if early clinical data support further advancement toward full development. XB010 is an antibody-drug conjugate (ADC) consisting of a monomethyl auristatin E payload conjugated to a monoclonal antibody targeting the tumor antigen 5T4 and is the first custom ADC generated through Exelixis’ biotherapeutics collaboration network. The first-in-human, global phase 1 trial of XB010 is evaluating the compound in patients with locally advanced or metastatic solid tumors. The dose-escalation stage of the study is evaluating XB010 as a single agent and in combination with pembrolizumab to inform the cohort-expansion stage. The expansion cohorts are designed to further assess the tolerability and activity of monotherapy and of the combination in specific indications. XL495 is a novel, potent, small molecule inhibitor of PKMYT1. The first-in-human phase 1 clinical trial of XL495 is evaluating the compound in patients with advanced solid tumors; the dose-escalation stage of the study is designed to determine the maximum tolerated dose of XL495. The expansion cohorts are designed to further assess the tolerability and activity of XL495 both as monotherapy and in combination with select cytotoxic agents in tumor-specific indications. Exelixis plans to present preclinical data from the XL495 program at a scientific meeting in 2025.

Anticipated Discovery Milestones

Three Potential Investigational New Drug (IND) Applications in 2025. Exelixis anticipates advancing three biotherapeutics programs into clinical development this year, including the XB628 PD-L1-NKG2A bispecific antibody, XB064 ILT-2 monoclonal antibody and XB371 TF-topoisomerase I inhibitor ADC. The company expects to file the IND applications for these compounds in 2025 if preclinical data continue to be supportive. Exelixis plans to present preclinical data from one or more of these programs at a scientific meeting in 2025.

Presentation and Webcast

Exelixis President and Chief Executive Officer Michael M. Morrissey, Ph.D., will provide a corporate overview and discuss the company’s preliminary fiscal year 2024 financial results, 2025 financial guidance and key priorities and milestones for 2025 during the company’s presentation at the 43^rd Annual J.P. Morgan Healthcare Conference beginning at 5:15 p.m. PT / 8:15 p.m. ET on Monday, January 13, 2025.

To access the webcast link, log onto www.exelixis.com and proceed to the Event Calendar page under the Investors & News heading. A replay will also be available at the same location for at least 30 days.

About Exelixis

Exelixis is a globally ambitious oncology company innovating next-generation medicines and regimens at the forefront of cancer care. Powered by drug discovery and development excellence, we are rapidly evolving our product portfolio to target an expanding range of tumor types and indications with our clinically differentiated pipeline of small molecules, antibody-drug conjugates and other biotherapeutics. This comprehensive approach harnesses decades of robust investment in our science and partnerships to advance our investigational programs and extend the impact of our flagship commercial product, CABOMETYX^® (cabozantinib). Exelixis is driven by a bold scientific pursuit to create transformational treatments that give more patients hope for the future. For information about the company and its mission to help cancer patients recover stronger and live longer, visit www.exelixis.com, follow @ExelixisInc on X (Twitter), like Exelixis, Inc. on Facebook and follow Exelixis on LinkedIn.

Forward-Looking Statements and Preliminary Financial Results

This press release contains forward-looking statements, including, without limitation, statements related to: Exelixis’ anticipation that 2025 will be a year of clinical and regulatory execution and continued growth for its cabozantinib franchise as well as multiple data readouts for zanzalintinib and across its diversified pipeline of small molecules and biotherapeutics with the potential to improve standards of care for patients with cancer; the regulatory review process with respect to Exelixis’ sNDA for cabozantinib in previously treated advanced pNET and advanced epNET, including the Prescription Drug User Fee Act target action date assigned by the FDA; Exelixis’ 2025 financial guidance; Exelixis’ goal of building a multi-product, multi-franchise oncology business and to grow cabozantinib franchise revenues, accelerate and expand its zanzalintinib pivotal development program, and advance its diversified therapeutic pipeline of small molecules and biotherapeutics; Exelixis’ expectation for 2025 to be a year of regulatory, clinical and commercial execution including a potential regulatory approval and launch for cabozantinib in neuroendocrine tumors and multiple zanzalintinib and pipeline data readouts; Exelixis’ belief in zanzalintinib’s potential to surpass cabozantinib’s scope and scale in the coming years and to become an important component of the company’s mid- and long-term revenue growth; Exelixis’ plans to execute on business development opportunities or a stock repurchase program; Exelixis’ anticipated cabozantinib milestones, including potential label expansion and commercial launch into NET, the presentation of data from CABINET at ASCO GI 2025, and the potential regulatory path forward for cabozantinib in mCRPC; Exelixis’ upcoming development milestones, including expansion and acceleration of the zanzalintinib pivotal trial program and Exelixis’ belief that zanzalintinib can become the vascular endothelial growth factor receptor TKI of choice as solid tumor therapeutic landscapes continue to evolve; Exelixis’ expectation for initial clinical data readouts from STELLAR-001 and STELLAR-002 in 2025 and the presentation of data from STELLAR-001 at ASCO GI 2025; clinical progress and priorities for XL309, the presentation of XL309 data at a scientific meeting in 2025 and Exelixis’ belief that XL309 is a potentially best-in-class small molecule inhibitor of USP1; clinical progress of phase 1 clinical trials for XB010 and XL495; Exelixis’ anticipated discovery milestones, including the advancement into clinical development of the XB628 PD-L1-NKG2A bispecific antibody, XB064 ILT-2 monoclonal antibody and XB371 TF-topoisomerase I inhibitor ADC and potential IND filings for these compounds if preclinical data continue to be supportive, and the presentation of preclinical data from one of more of these programs at a scientific meeting in 2025; Exelixis’ scientific pursuit to create transformational treatments that give more patients hope for the future; and other statements that are not historical facts. Any statements that refer to expectations, projections or other characterizations of future events or circumstances are forward-looking statements and are based upon Exelixis’ current plans, assumptions, beliefs, expectations, estimates and projections. Forward-looking statements involve risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in the forward-looking statements as a result of these risks and uncertainties, which include, without limitation: the degree of market acceptance of CABOMETYX and other Exelixis products in the indications for which they are approved and in the territories where they are approved, and Exelixis’ and its partners’ ability to obtain or maintain coverage and reimbursement for these products; the effectiveness of CABOMETYX and other Exelixis products in comparison to competing products; complexities and the unpredictability of the regulatory review and approval processes in the U.S. and elsewhere, including the risk that the FDA may not approve cabozantinib as a treatment for pNET or epNET in a timely fashion, if at all; the level of costs associated with Exelixis’ commercialization, research and development, in-licensing or acquisition of product candidates, and other activities; Exelixis’ ability to maintain and scale adequate sales, marketing, market access and product distribution capabilities for its products or to enter into and maintain agreements with third parties to do so; the availability of data at the referenced times; the potential failure of cabozantinib, zanzalintinib and other Exelixis product candidates, both alone and in combination with other therapies, to demonstrate safety and/or efficacy in clinical testing; uncertainties inherent in the drug discovery and product development process; Exelixis’ dependence on its relationships with its collaboration partners, including their pursuit of regulatory approvals for partnered compounds in new indications, their adherence to their obligations under relevant collaboration agreements and the level of their investment in the resources necessary to complete clinical trials or successfully commercialize partnered compounds in the territories where they are approved; complexities and the unpredictability of the regulatory review and approval processes in the U.S. and elsewhere; Exelixis’ continuing compliance with applicable legal and regulatory requirements; unexpected concerns that may arise as a result of the occurrence of adverse safety events or additional data analyses of clinical trials evaluating cabozantinib, zanzalintinib and other Exelixis product candidates; Exelixis’ dependence on third-party vendors for the development, manufacture and supply of its products and product candidates; Exelixis’ ability to protect its intellectual property rights; market competition, including the potential for competitors to obtain approval for generic versions of Exelixis’ marketed products; changes in economic and business conditions; and other factors detailed from time to time under the caption “Risk Factors” in Exelixis’ most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q, and in Exelixis’ other future filings with the Securities and Exchange Commission. All forward-looking statements in this press release are based on information available to Exelixis as of the date of this press release, and Exelixis undertakes no obligation to update or revise any forward-looking statements contained herein, except as required by law.

In addition, this press release includes Exelixis’ preliminary financial results for the fiscal year ended January 3, 2025. Exelixis is currently in the process of finalizing its financial results for the quarter and fiscal year ended January 3, 2025, and the preliminary financial results presented in this press release are based only upon preliminary information available to Exelixis as of January 12, 2025. Exelixis’ preliminary financial results should not be viewed as a substitute for audited financial statements prepared in accordance with U.S. GAAP, and undue reliance should not be placed on Exelixis’ preliminary financial results. Exelixis’ independent registered public accounting firm has not audited or reviewed the preliminary financial results included in this press release or expressed any opinion or other form of assurance on such preliminary financial results.

Contacts

Investor Contacts:
Susan Hubbard
EVP, Public Affairs & Investor Relations
Exelixis, Inc.
650-837-8194
shubbard@exelixis.com

Chris Senner
Chief Financial Officer
Exelixis, Inc.
650-837-7240
csenner@exelixis.com

Media Contact:
Hal Mackins
For Exelixis, Inc.
415-994-0040
hal@torchcommunications.com

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Alnylam Announces Preliminary* Fourth Quarter and Full Year 2024 Global Net Product Revenues and Provides 2025 Combined Net Product Revenue Guidance and Pipeline Goals




Alnylam Announces Preliminary* Fourth Quarter and Full Year 2024 Global Net Product Revenues and Provides 2025 Combined Net Product Revenue Guidance and Pipeline Goals

– Full Year 2024 Preliminary Net Product Revenues of $1,646 Million for ONPATTRO^®, AMVUTTRA^®, GIVLAARI^®, and OXLUMO^®, Representing 33% Annual Growth –

– 2025 Combined Net Product Revenue Guidance** of $2,050 Million to $2,250 Million Positions Company to Achieve Alnylam P⁵x25 Goal of Non-GAAP Profitability –

– Robust Clinical Pipeline with Multi-Billion-Dollar Opportunities for Sustainable Growth –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced its preliminary* fourth quarter and full year 2024 global net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO. In addition, the Company provided 2025 net product revenue, non-GAAP operating income profitability, and pipeline goals guidance.

“Alnylam’s commercial and clinical achievements in 2024 position us very well for another transformative year in 2025, as we continue to evolve into a global, top-tier biotech company,” said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. “We generated net product revenues for the year of over $1.6 billion, representing growth of 33% compared to 2023, at the high end of our revised guidance range, and demonstrating the strength of our underlying hATTR-PN and Rare businesses. We expect 2025 will represent an important inflection point for our TTR franchise, with the potential launch of vutrisiran in ATTR-CM delivering significant topline growth as reflected in our net product sales guidance announced today. If we are successful in meeting this product revenue guidance, we anticipate achieving non-GAAP profitability in 2025.”

Dr. Greenstreet continued, “We’re also looking forward to a year of major advancements in our pipeline and RNAi platform, with key goals outlined today. This remarkable pace of progress positions us well to finish the year achieving key Alnylam P⁵x25 goals and to continue delivering sustainable innovation to patients through our global commercial infrastructure, broad pipeline, and organic platform.”

Preliminary Fourth Quarter and Full Year 2024 Commercial and Financial Performance*

Total TTR: ONPATTRO^® (patisiran) & AMVUTTRA^® (vutrisiran)

• Preliminary* global net product revenues for ONPATTRO and AMVUTTRA for the fourth quarter were approximately $56 million and $287 million, respectively, representing together 35% total TTR annual growth compared to Q4 2023, and for the full year 2024 were approximately $253 million and $970 million, respectively, representing together 34% total TTR annual growth compared to full year 2023.

Total Rare: GIVLAARI^® (givosiran) & OXLUMO^® (lumasiran)

• Preliminary* global net product revenues for GIVLAARI and OXLUMO for the fourth quarter were approximately $65 million and $44 million, respectively, representing together 18% total Rare annual growth compared to Q4 2023, and for the full year 2024 were approximately $256 million and $167 million, respectively, representing together 29% total Rare annual growth compared to full year 2023.

2025 Combined Net Product Revenue & Non-GAAP Operating Income Guidance

Alnylam announced today full year 2025 combined net product revenue guidance for ONPATTRO, AMVUTTRA (PN & CM**), GIVLAARI and OXLUMO of $2,050 million to $2,250 million, representing combined full year growth compared to 2024 of 31% at the mid-point of the guidance range. On a franchise level, the guidance is broken down as follows:

• Total TTR (ONPATTRO, AMVUTTRA (PN & CM**)): $1,600 million to $1,725 million, representing full year growth compared to 2024 of 36% at the mid-point of the guidance range.
• Total Rare (GIVLAARI, OXLUMO): $450 million to $525 million, representing full year growth compared to 2024 of 15% at the mid-point of the guidance range.

In addition, the Company anticipates delivering non-GAAP operating income profitability in 2025.

The Company plans to provide additional guidance for collaboration and royalty revenue and operating expenses at the time fourth quarter and full year 2024 earnings are released.

2025 Product and Pipeline Goals

Vutrisiran – an RNAi therapeutic marketed in various countries globally as a treatment of adults with hATTR amyloidosis with polyneuropathy, and in development for the treatment of adults with ATTR amyloidosis with cardiomyopathy. Alnylam expects to:

• Achieve U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application for the treatment of adults with ATTR amyloidosis with cardiomyopathy by the PDUFA target action date of March 23, 2025.
• Secure additional global approvals and reimbursement in Japan and the EU for the treatment of adults with ATTR amyloidosis with cardiomyopathy in the second half of 2025.

Nucresiran (ALN-TTRsc04) – an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. Alnylam expects to:

• Initiate a Phase 3 study in patients with ATTR amyloidosis with cardiomyopathy in the first half of 2025.

Zilebesiran – an investigational RNAi therapeutic in development for the treatment of hypertension, in collaboration with Roche. Alnylam expects to:

• Report results from the KARDIA-3 Phase 2 study in the second half of 2025.
• Initiate a Phase 3 cardiovascular outcomes trial in the second half of 2025.

Mivelsiran – an investigational RNAi therapeutic in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA). Alnylam expects to:

• Report interim results from Part B of the Phase 1 study in Alzheimer’s disease in the second half of 2025.
• Initiate a Phase 2 study in Alzheimer’s disease in the second half of 2025.

ALN-6400 – an investigational RNAi therapeutic in development for the treatment of bleeding disorders. Alnylam expects to:

• Initiate a Phase 2 study in a bleeding disorder in the second half of 2025.

In addition, the Company plans to file Investigational New Drug (IND) applications for four new Alnylam-led programs by the end of 2025.

Partner-Led Program Highlights

Alnylam partnered programs continue to progress, including:

• Fitusiran – an investigational RNAi therapeutic partnered with Sanofi in development for the treatment of hemophilia A and B, with or without inhibitors. Sanofi expects to secure FDA approval by the PDUFA target action date of March 28, 2025.
• Elebsiran – an investigational RNAi therapeutic partnered with Vir Biotechnology in development for the treatment of chronic hepatitis B and chronic hepatitis delta. In 2025, Vir expects to initiate a Phase 3 chronic hepatitis delta registrational study and to report functional cure results from a Phase 2 chronic hepatitis B study.

Alnylam management will discuss its preliminary 2024 net product revenues, as well as 2025 goals and guidance during a webcast presentation at the 43^rd Annual J.P. Morgan Healthcare Conference in San Francisco, California tomorrow, Monday, January 13, 2025 at 9:45 am PT (12:45 pm ET).

About RNAi Therapeutics

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO^® (patisiran), AMVUTTRA^® (vutrisiran), GIVLAARI^® (givosiran), OXLUMO^® (lumasiran), and Leqvio^® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P⁵x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.

Alnylam Forward Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding Alnylam’s evolution into a leading, global, top-tier biotech company; Alnylam’s expectations regarding the potential approval and launch of AMVUTTRA for the treatment of ATTR amyloidosis with cardiomyopathy in the U.S. in early 2025 and in other territories in the second half of 2025; the potential that the launch of AMVUTTRA for ATTR-CM will deliver significant topline growth for Alnylam; the potential for 2025 to be a transformative year for Alnylam and that 2025 will represent an important inflection point for Alnylam’s TTR franchise; Alnylam’s ability to deliver non-GAAP operating income profitability in 2025; the potential for 2025 to be a year of major advancements in Alnylam’s pipeline and RNAi platform; Alnylam’s potential achievement of the goals in its “Alnylam P⁵x25” strategy; Alnylam’s ability to continue to deliver sustainable innovation to patients through its global commercial infrastructure, broad pipeline and organic platform; the potential for Alnylam to advance its research and development programs, including its goals and expectations regarding the clinical development of vutrisiran, nucresiran, zilebesiran, mivelsiran, ALN-6400, and its earlier stage programs, and its partners’ expectations for Alnylam’s partnered programs; and Alnylam’s projected commercial and financial performance, including the expected range of net product revenues and non-GAAP operating income for 2025, should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam P⁵x25” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates, including vutrisiran, nucresiran, zilebesiran, mivelsiran and ALN-6400; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to successfully expand the approved indications for AMVUTTRA in the future; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products, including Roche, Novartis, Sanofi, Regeneron and Vir; the outcome of litigation; the risk of future government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the “Risk Factors” filed with Alnylam’s 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

This release discusses investigational RNAi therapeutics and uses of previously approved RNAi therapeutics in development and is not intended to convey conclusions about efficacy or safety as to those investigational therapeutics or uses. Vutrisiran has not been approved by any regulatory agency for the treatment of ATTR amyloidosis with cardiomyopathy. No conclusions can or should be drawn regarding its safety or effectiveness in treating cardiomyopathy in this population. There is no guarantee that any investigational therapeutics or expanded uses of commercial products will successfully complete clinical development or gain health authority approval.

Use of Non-GAAP Financial Measures

This press release contains a non-GAAP financial measure of non-GAAP operating income. This measure is not in accordance with, or an alternative to, GAAP, and may be different from non-GAAP financial measures used by other companies. Stock-based compensation expense is included in GAAP operating income but excluded for purposes of determining non-GAAP operating income. The Company has excluded the impact of stock-based compensation expense as it may fluctuate from period to period based on factors including the variability associated with performance-based grants for stock options and restricted stock units and changes in the Company’s stock price, which impacts the fair value of these awards.

* The preliminary selected financial results are unaudited, subject to adjustment, and provided as an approximation in advance of the Company’s announcement of complete financial results in February 2025.

** Guidance assumes FDA approval of the sNDA for vutrisiran for the treatment of adults with ATTR amyloidosis with cardiomyopathy by the March 23, 2025 PDUFA target action date.

Contacts

Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom

(Investors and Media)

617-682-4340

Josh Brodsky

(Investors)

617-551-8276

QIAGEN to Return Approximately $300 Million to Shareholders Through a Synthetic Share Repurchase




QIAGEN to Return Approximately $300 Million to Shareholders Through a Synthetic Share Repurchase

• Capital return to be conducted through synthetic share repurchase – combines a fast direct capital repayment to shareholders with a reverse stock split that enhances EPS

• Return of up to $300 million – maximum approved by shareholders – set to be completed in late January 2025

• Builds on approximately $300 million returned to shareholders in early 2024 as part of commitment to return at least $1 billion through end 2028

VENLO, Netherlands–(BUSINESS WIRE)–$QGEN #QIAGEN–QIAGEN N.V. (NYSE: QGEN; Frankfurt Prime Standard: QIA) today announced a new plan to return up to approximately $300 million (maximum EUR 281 million) to shareholders through a synthetic share repurchase that combines a direct capital repayment with a reverse stock split.

This new repurchase comes after QIAGEN returned approximately $300 million to shareholders in early 2024 through a synthetic share repurchase. Together, these two programs represent $600 million of a commitment to return at least $1 billion to shareholders by the end of 2028 (absent M&A opportunities).

QIAGEN has decided to implement the maximum $300 million value of the mandate given at the Annual General Meeting in June 2024, where shareholders gave virtually unanimous approval for the related resolutions.

This approach is designed to return cash to shareholders in a much faster and more efficient way than through a traditional open-market repurchase program. It would also enhance earnings per share (EPS) through the reduction in outstanding shares.

“QIAGEN has a proven track record in delivering on our commitments from our differentiated portfolio, and this includes using our healthy balance sheet to enhance our business while increasing returns to shareholders,” said Thierry Bernard, CEO of QIAGEN. “This new repurchase marks an important step in creating value for our shareholders and other stakeholders as we execute on our 2028 ambitions to deliver solid profitable growth.”

Roland Sackers, Chief Financial Officer of QIAGEN, said: “Our synthetic share repurchase structure is a well-known and proven approach to enhance value that has been utilized by many Dutch companies. QIAGEN will continue to have a solid investment-grade profile after completion of this repurchase in early 2025. We are exploring various targeted M&A opportunities and organic growth investments that will help us achieve our commitments for solid profitable growth.”

This type of share repurchase involves three steps:

The synthetic share repurchase will become effective on January 28, 2025, and will be settled in line with market convention in the subsequent days. Further information on this process will be announced before implementation.

About QIAGEN

QIAGEN N.V., a Netherlands-based holding company, is the leading global provider of Sample to Insight solutions that enable customers to gain valuable molecular insights from samples containing the building blocks of life. Our sample technologies isolate and process DNA, RNA and proteins from blood, tissue and other materials. Assay technologies make these biomolecules visible and ready for analysis. Bioinformatics software and knowledge bases interpret data to report relevant, actionable insights. Automation solutions tie these together in seamless and cost-effective workflows. QIAGEN provides solutions to more than 500,000 customers around the world in Molecular Diagnostics (human healthcare) and Life Sciences (academia, pharma R&D and industrial applications, primarily forensics). As of September 30, 2024, QIAGEN employed more than 5,800 people in over 35 locations worldwide. Further information can be found at https://www.qiagen.com.

Forward-Looking Statement

Certain statements contained in this press release may be considered forward-looking statements within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended. To the extent that any of the statements contained herein relating to QIAGEN’s products, including those products used in the response to the COVID-19 pandemic, timing for launch and development, marketing and/or regulatory approvals, financial and operational outlook, growth and expansion, collaborations, markets, strategy or operating results, including without limitation its expected adjusted net sales and adjusted diluted earnings results, are forward-looking, such statements are based on current expectations and assumptions that involve a number of uncertainties and risks. Such uncertainties and risks include, but are not limited to, risks associated with management of growth and international operations (including the effects of currency fluctuations, regulatory processes and dependence on logistics), variability of operating results and allocations between customer classes, the commercial development of markets for our products to customers in academia, pharma, applied testing and molecular diagnostics; changing relationships with customers, suppliers and strategic partners; competition; rapid or unexpected changes in technologies; fluctuations in demand for QIAGEN’s products (including fluctuations due to general economic conditions, the level and timing of customers’ funding, budgets and other factors); our ability to obtain regulatory approval of our products; difficulties in successfully adapting QIAGEN’s products to integrated solutions and producing such products; the ability of QIAGEN to identify and develop new products and to differentiate and protect our products from competitors’ products; market acceptance of QIAGEN’s new products and the integration of acquired technologies and businesses; actions of governments, global or regional economic developments, weather or transportation delays, natural disasters, political or public health crises, including the breadth and duration of the COVID-19 pandemic and its impact on the demand for our products and other aspects of our business, or other force majeure events; as well as the possibility that expected benefits related to recent or pending acquisitions may not materialize as expected; and the other factors discussed under the heading “Risk Factors” contained in Item 3 of our most recent Annual Report on Form 20-F. For further information, please refer to the discussions in reports that QIAGEN has filed with, or furnished to, the U.S. Securities and Exchange Commission.

Source: QIAGEN N.V.

Category: Financial

Contacts

QIAGEN:

Investor Relations
John Gilardi +49 2103 29 11711

Domenica Martorana +49 2103 29 11244

e-mail: ir@QIAGEN.com

Public Relations
Thomas Theuringer +49 2103 29 11826

Lisa Specht +49 2103 29 14181

e-mail: pr@QIAGEN.com

N-Power Medicine Acquires Syapse to Expand Its Next-Generation Community-Based Network for Oncology Clinical Research and Clinical Trials




N-Power Medicine Acquires Syapse to Expand Its Next-Generation Community-Based Network for Oncology Clinical Research and Clinical Trials

• This transaction accelerates scale-up of N-Power’s unique “always-on” prospective clinical research model, adding an extensive network of community health systems and 1,000+ community oncologists across the U.S.

REDWOOD CITY, Calif.–(BUSINESS WIRE)–N-Power Medicine, a company leading the reinvention of the clinical trial process, today announced the acquisition of Syapse Holdings Inc. (“Syapse”), a pioneer in precision medicine solutions for community cancer centers. The integration of Syapse into N-Power Medicine incorporates Syapse’s extensive network of community-based health systems, with more than 1,000 oncologists, its data and technology stack, and its dedicated team.

N-Power partners with community oncologists through its proprietary real-time Kaleido Registry and AI-enabled point-of-care platform, complemented by virtual and on-site support, to optimize research and patient care workflows. This transaction will establish the largest community-based prospective clinical research network where every patient, through N-Power’s platform, will have research-ready data and documentation as part of their routine care. The expanded N-Power network will also help address critical gaps in patient access to clinical trials in the community setting, where more than 80% of U.S. cancer patients—particularly those from diverse and underrepresented populations—receive their care.

The transaction will enhance the company’s current offerings to its growing set of pharmaceutical customers, including Merck, which is engaged in a multi-trial collaboration with N-Power. N-Power can now provide significantly greater access to a more representative group of geographically diverse, U.S. community-based cancer patients for clinical trial enrollment. Greater network scale will also enable the rapid and rigorous generation of prospective external controls, allowing for smaller and faster clinical trials.

“The integration of Syapse into N-Power underscores our commitment to dramatically accelerating drug development and improving the probability of success by seamlessly integrating clinical research into every oncologist’s practice and every cancer patient’s care,” said Mark Lee, M.D., Ph.D, the Co-Founder and Chief Executive Officer of N-Power Medicine. “With this scale, N-Power accelerates the transformation of drug development that our pharma customers are asking for – enabling local access to clinical trials for more community oncologists and community cancer patients than ever before, and by creating a unique platform for generating prospective external controls, reducing the need for randomized trials.”

“Syapse joining N-Power represents a transformative step forward in our shared mission to revolutionize community-based patient care. By building upon Syapse’s existing offerings for its health system and pharma partners with N-Power’s AI-enabled ‘human-in-the-loop’ approach, we can significantly expand access to prospective clinical trials and life-saving innovations for patients in community settings,” said Michael Mentesana, the Chief Executive Officer of Syapse. “We are proud to join N-Power in building a future where community oncology serves as a powerful catalyst for research and improved patient outcomes.”

N-Power’s acquisition of Syapse was completed as a stock-for-stock exchange on Dec. 30, 2024. Innovatus Capital Partners LLC, Syapse’s lead institutional investor, joins N-Power’s existing investors, including the Merck Global Health Innovation Fund (GHI) and a U.S. based healthcare-focused investor, in providing ongoing funding for N-Power’s vision.

Canaccord Genuity, LLC acted as the exclusive financial advisor to Syapse in this transaction.

About N-Power Medicine

N-Power Medicine is a clinical research and drug development platform company that aims to dramatically boost clinical trial participation to accelerate oncology drug development – and deliver on the promise of bringing life-saving innovation to cancer patients. Founded in 2021, the company addresses critical challenges for oncology sites by integrating technology and trained personnel into routine care to unlock the full potential of the data needed to bring new therapies to patients sooner. For more information on N-Power Medicine, visit www.npowermedicine.com and connect with us on LinkedIn.

About Syapse

Syapse is a company dedicated to extinguishing the fear and burden of oncology and other serious diseases by advancing real-world care. By marrying clinical expertise with smart technologies, we transform data into evidence—and then into experience—in collaboration with our network of partners, who are committed to improving patients’ lives through community health systems. Together, we connect comprehensive patient insights to our network, to empower our partners in driving real impact and improving access to high-quality care.

Contacts

Media Contact:
Emma Yang

Health+Commerce

emma@healthandcommerce.com

Gilead and LEO Pharma Enter Into Strategic Partnership to Accelerate Development of Oral STAT6 Program With Potential in Multiple Inflammatory Diseases




Gilead and LEO Pharma Enter Into Strategic Partnership to Accelerate Development of Oral STAT6 Program With Potential in Multiple Inflammatory Diseases

— Strategic Partnership Strengthens Gilead’s Inflammation Research Portfolio with the Addition of LEO Pharma’s Preclinical Oral STAT6 Program, Including Targeted Protein Degraders —

— Gilead Will Have Exclusive Global Rights to the STAT6 Program, and LEO Pharma Will Have the Option to Co-Commercialize for Dermatology Indications Outside the U.S. —

— LEO Pharma to Maintain Global Rights to Topical Formulations of the STAT6 Program in Dermatology —

FOSTER CITY, Calif. & BALLERUP, Denmark–(BUSINESS WIRE)–Gilead Sciences, Inc. (Nasdaq: GILD) and LEO Pharma today announced a strategic partnership to accelerate the development and commercialization of LEO Pharma’s small molecule oral STAT6 (signal transducer and activator of transcription 6) programs for the potential treatment of patients with inflammatory diseases.

STAT6 is the specific transcription factor required for IL-4 and IL-13 cytokine signaling, which are clinically validated targets for Th2 mediated inflammatory conditions such as atopic dermatitis, asthma, and COPD, amongst many others. Targeting STAT6 has shown potential preclinically to treat a broad population of patients and provide an oral alternative to those currently treated with injectable biologics.

Under this partnership, Gilead will acquire LEO Pharma’s comprehensive preclinical oral STAT6 small molecule inhibitors and targeted protein degraders. Gilead will lead further development efforts for the oral programs, while LEO Pharma will lead development for potential topical formulations of STAT6 inhibitors.

“As we continue to expand our inflammation portfolio, we’re committed to developing next-generation therapies to support long-term remission in patients with inflammatory diseases through mechanisms that block major pathogenic pathways, eliminate pathogenic cells, tolerize the immune system, and restore cell function,” said Flavius Martin, M.D., Executive Vice President, Research, Gilead Sciences. “By partnering with LEO Pharma, we hope to explore the potential of the STAT6 pathway to bring forward an oral option for patients suffering from chronic inflammatory conditions.”

“STAT6 holds potential for treating various inflammatory diseases, including outside dermatology. This strategic partnership with Gilead will enable us to accelerate the development of the STAT6 program and maximize its potential in dermatology and beyond,” said Christophe Bourdon, CEO of LEO Pharma. “This partnership acknowledges LEO Pharma’s scientific capabilities and strengthens our commitment to innovate for people with skin diseases.”

Terms of the Agreement

Gilead will have global rights to develop, manufacture, and commercialize the small molecule oral STAT6 program. LEO Pharma will have the option to potentially co-commercialize oral programs for dermatology outside the United States. LEO Pharma will hold exclusive global rights to STAT6 topical formulations in dermatology.

LEO Pharma is eligible to receive up to $1.7 billion in total payments, including an upfront payment of $250 million. In addition, LEO Pharma may also receive tiered royalties ranging from high single-digit to mid-teens on sales of oral STAT6 products. Gilead may receive tiered royalties ranging from high single-digit to mid-teens on sales of topical STAT6 products.

Gilead does not exclude acquired IPR&D expenses from its non-GAAP financial measures. This transaction with LEO Pharma is expected to reduce Gilead’s GAAP and non-GAAP 2025 EPS by approximately $0.15 – $0.17.

Bank of America Securities acted as financial advisors to LEO Pharma in this transaction and Latham Watkins LLP acted as legal advisors.

About Gilead Sciences

Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis, COVID-19, cancer, and inflammation. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, Calif.

About LEO Pharma

LEO Pharma is a global company dedicated to advancing the standard of care through innovation for the benefit of people with skin conditions. LEO Pharma is co-owned by majority shareholder the LEO Foundation and, since 2021, Nordic Capital. LEO Pharma offers a broad portfolio of treatments, serving 100 million patients annually. Headquartered in Denmark, LEO Pharma has a global team of 4,000 people. In 2023, the company generated net sales of $1.6 billion. Read more at www.leo-pharma.com.

Gilead Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including Gilead’s ability to realize the anticipated benefits from the partnership; difficulties or unanticipated expenses in connection with the partnership and the potential effects on Gilead’s earnings; the ability of Gilead to initiate, progress or complete clinical trials within currently anticipated timelines or at all, and the possibility of unfavorable results from ongoing or additional trials, including those involving programs developed pursuant to the partnership; the ability of Gilead to file applications for regulatory approval or receive regulatory approvals in a timely manner or at all for the investigational programs developed pursuant to the partnership, and the risk that any such approvals may be subject to significant limitations on use; the possibility that Gilead may make a strategic decision to discontinue development of any of the investigational programs developed pursuant to the partnership, and therefore these programs may never be successfully commercialized; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and other factors are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead, and Gilead assumes no obligation and disclaims any intent to update any such forward-looking statements.

Gilead and the Gilead logo are trademarks of Gilead Sciences, Inc., or its related companies. The LEO Pharma name and logo are trademarks of LEO Pharma.

For more information about Gilead, please visit the company’s website at www.gilead.com, follow Gilead on X/Twitter (@Gilead Sciences) and LinkedIn (@Gilead-Sciences).

Contacts

Gilead Info
Jessica Smith, Media

public_affairs@gilead.com

Jacquie Ross, Investors

investor_relations@gilead.com

LEO Pharma Info
Jeppe Ilkjaer, Media

jeilk@leo-pharma.com

Christian Soerup Ryom, Investors

chsoe@leo-pharma.com

MeMed Expands Advisory Board with Key Leaders to Advance Host-Response Diagnostics for Sepsis and AMR




MeMed Expands Advisory Board with Key Leaders to Advance Host-Response Diagnostics for Sepsis and AMR

— Renowned Experts Beat Müller, Dave Hickey, and David Shulkin Join MeMed’s Advisory Board —

ANDOVER, Mass. & HAIFA, Israel–(BUSINESS WIRE)–#AMR—MeMed, a leader in host-response diagnostics, today announced the addition of three distinguished experts to its advisory board to accelerate its mission of addressing the global challenges of sepsis and antimicrobial resistance (AMR) through innovative diagnostic solutions. These new members bring decades of experience in medicine, industry, and healthcare policy, strengthening MeMed’s work in host-response technologies, which combine machine learning with rapid measurements of the immune response to transform infectious disease care.

The advisory board now includes:

• Beat Müller, M.D., Professor of Medicine, University of Basel, and a global authority on procalcitonin diagnostics and sepsis care.
• Dave Hickey, former Executive Vice President and President of the Life Sciences segment for BD, and a seasoned expert in in-vitro diagnostics (IVD).
• David Shulkin, M.D., the ninth Secretary of the US Department of Veterans Affairs and Board Member at Sanford Health, with deep expertise in U.S. healthcare delivery and policy.

Prof. Müller emphasized the critical role of advanced host-response diagnostics: “AMR and sepsis are deeply interconnected challenges, and host-response diagnostics are the next frontier in addressing them. I look forward to working with MeMed to further strengthen clinical outcome data for their FDA-cleared MeMed BV test and the in-development FDA Breakthrough-designated MeMed Severity test. These tools have the potential to significantly improve clinical decision-making by providing actionable insights into bacterial versus viral infections and predicting the likelihood of suspected sepsis severity.”

Mr. Hickey, a veteran professional in the IVD industry, said: “Integrating traditional IVD with host-response and machine learning is a major step forward. MeMed’s approach is expected to enhance patient management throughout the sepsis continuum of care, addressing unmet needs in both centralized hospital labs and decentralized settings. I am excited to help shape their commercial strategy and expand partnerships to make these innovations widely accessible and make sure they impact as many patients as possible.”

Dr. Shulkin spoke on the importance of scaling MeMed’s diagnostics in the U.S. healthcare system: “Sepsis and AMR are among the most pressing challenges in U.S. healthcare. MeMed’s host-response solutions have the potential to revolutionize care delivery, reduce unnecessary antibiotic use, and optimize resources in a cost-effective way. I look forward to helping operationalize them to address these critical needs.”

“We are privileged to work alongside such an extraordinary group of leaders,” said Eran Eden, Ph.D., CEO and Co-Founder of MeMed. “Their expertise will help us advance the field of host-response diagnostics and bring these tools to clinicians and patients worldwide.”

MeMed BV® and MeMed Key are 510(k) FDA, CE-IVD (IVDR certified) and available in the US and EU. MeMed Severity™ is currently in development and has not been cleared for sale by any regulatory authority.

About MeMed

At MeMed, our mission is to translate the immune system’s complex signals into simple insights that transform the way diseases are diagnosed and treated, profoundly benefiting patients and society.

MeMed BV® is the first FDA-cleared host-immune response test for accurately distinguishing between bacterial and viral infections in 15 minutes.

MeMed Severity™ is a groundbreaking host-response test designed to predict the likelihood of progression to severe outcomes in patients with suspected acute infection or sepsis. It is in development and has been awarded FDA Breakthrough Designation.

Follow and connect with MeMed via LinkedIn, X, and Facebook.

Contacts

MeMed Contact:
Tal Avziz

Vice President, Global Marketing

pr@me-med.com

Media Relations Contact:
Charya Wickremasinghe, Ph.D.

Brandwidth Solutions LLC

cwickremasinghe@brandwidthsolutions.com

Areteia Therapeutics to Present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday January 14, 2025




Areteia Therapeutics to Present at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday January 14, 2025

CHAPEL HILL, N.C.–(BUSINESS WIRE)–Areteia Therapeutics, Inc. (“Areteia”), a clinical-stage biotechnology company developing novel inflammation and immunology therapies, with an initial focus on severe eosinophilic asthma, today announced that the Company will present at the 43^rd Annual J.P. Morgan Healthcare Conference on Tuesday January 14, 2025 at 1:30 p.m. PT in San Francisco, CA.

A copy of the presentation will be posted on the Company’s website following the presentation.

About Areteia

Areteia is a clinical-stage biotechnology company engaged in the development of a potential first-in-class oral therapeutic remedy for diseases associated with elevated eosinophil levels with an initial focus on severe eosinophilic asthma. The Company has advanced its investigational drug, dexpramipexole, into three separate Phase III clinical trials, including two 52-week global exacerbation trials and one 24-week lung function trial. Areteia’s team is committed to developing and delivering novel inflammation and immunology therapies with the goal of putting respiratory patients in better control of their disease and back in control of their lives. To learn more, please visit www.areteiatx.com

Areteia was created by Population Health Partners and Knopp Biosciences. A syndicate of leading life sciences and strategic investors led by Bain Capital Life Sciences with participation from Access Biotechnology, ARCH Venture Partners, Viking Global Investors, Marshall Wace, GV, Saturn Partners, Sanofi, Maverick Capital, and Population Health Partners, has committed to invest up to $425 million in Series A financing to establish Areteia and advance dexpramipexole through Phase III clinical trials, secure commercial supply, and pursue potential next-generation medicines.

About Dexpramipexole

Dexpramipexole, an investigational drug, is an oral small molecule that has been shown to lower eosinophil levels in blood and tissue and is currently in Phase III development for treatment of adults and adolescents with severe eosinophilic asthma. Dexpramipexole’s proposed mechanism of action is to inhibit the maturation of eosinophils in the bone marrow, based on evidence from cell cultures and human biopsies, thereby lowering peripheral blood eosinophil levels. Most recently in a Phase II study in patients with moderate-to-severe eosinophilic asthma, treatment with our investigational drug dexpramipexole resulted in a significant, dose-dependent reduction in blood absolute eosinophil count at all doses tested (twice daily dexpramipexole dihydrochloride doses of 37.5-mg, 75-mg, or 150-mg) compared to placebo. The investigational drug dexpramipexole was well tolerated in the trial, with adverse events balanced across treatment and placebo groups, no serious adverse events, and no adverse events leading to discontinuation.

About Eosinophilic Asthma

Asthma disrupts the lives of more than a quarter of a billion people worldwide. More than half of asthma patients have eosinophilic asthma, which is driven by an oversupply of eosinophils, a type of white blood cell, in blood and tissue. By inhibiting the maturation of eosinophils, we believe that the oral administration of the investigational drug dexpramipexole acts to lower eosinophils. Currently approved injectable anti-IL-5/5R biologic therapies provide clinical benefit through eosinophil lowering. The global asthma biologic market is experiencing robust growth having doubled in the last three years and is valued today at approximately $10 billion. If approved as a first-to-market oral, dexpramipexole could provide an alternative to injectable biologics.

Contacts

Mark Kreston

Mark.Kreston@areteiatx.com