Revvity Announces Financial Results for the First Quarter of 2025




Revvity Announces Financial Results for the First Quarter of 2025

• Revenue of $665 million; 2% reported growth; 4% organic growth
• GAAP EPS of $0.35; Adjusted EPS from continuing operations of $1.01
• Reaffirms full year 2025 organic growth and adjusted EPS guidance

WALTHAM, Mass.–(BUSINESS WIRE)–Revvity, Inc. (NYSE: RVTY), today reported financial results for the first quarter ended March 30, 2025.

The Company reported GAAP earnings per share of $0.35, as compared to $0.21 in the same period a year ago. Revenue for the quarter was $665 million, as compared to $650 million in the same period a year ago. GAAP operating income from continuing operations for the quarter was $72 million, as compared to $44 million for the same period a year ago. GAAP operating profit margin from continuing operations was 10.9% as a percentage of revenue, as compared to 6.8% in the same period a year ago.

Adjusted earnings per share from continuing operations for the quarter was $1.01, as compared to $0.98 in the same period a year ago. Adjusted operating income was $170 million, as compared to $166 million for the same period a year ago. Adjusted operating profit margin was 25.6% as a percentage of revenue, as compared to 25.5% in the same period a year ago.

Adjustments for the Company’s non-GAAP financial measures have been noted in the attached reconciliations.

“Revvity navigated a dynamic environment and delivered strong first quarter results,” said Prahlad Singh, president and chief executive officer of Revvity. “Our strong execution combined with the uniqueness of our businesses drove our revenue, earnings and cash flow to each exceed our expectations in the quarter. Our first quarter performance positions us well for the remainder of the year as we continue to adapt to an evolving macroeconomic backdrop.”

Financial Overview by Reporting Segment

Life Sciences

• First quarter 2025 revenue was $340 million, as compared to $337 million in the same period a year ago. Revenue increased 1% and organic revenue increased 2% as compared to the same period a year ago.

• First quarter 2025 adjusted operating income was $106 million, as compared to $101 million in the same period a year ago. Adjusted operating profit margin was 31.1% as a percentage of revenue, as compared to 30.0% in the same period a year ago.

Diagnostics

• First quarter 2025 revenue was $324 million, as compared to $313 million in the same period a year ago. Revenue increased 3% and organic revenue increased 5% as compared to the same period a year ago.

• First quarter 2025 adjusted operating income was $74 million, as compared to $76 million in the same period a year ago. Adjusted operating profit margin was 22.8% as a percentage of revenue, as compared to 24.3% in the same period a year ago.

Full Year 2025 Guidance

For the full year 2025, the Company is raising its revenue guidance to a range of $2.83-$2.87 billion to reflect recent changes in foreign currency exchange rates and is reaffirming its organic growth guidance of 3-5%. Additionally, the Company is reaffirming its adjusted EPS guidance of $4.90-$5.00.

Guidance for the full year 2025 for adjusted EPS and organic growth 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.

Webcast Information

The Company will discuss its first quarter 2025 results and its outlook for business trends during a webcast on April 28, 2025, at 8:00 a.m. Eastern Time. A live audio webcast and presentation will be available on the Investors section of the Company’s website, ir.revvity.com.

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 following our GAAP financial statements.

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 earnings per share, cash flow and revenue growth and other financial results, developments relating to our customers and end-markets, and plans concerning business development opportunities, acquisitions and divestitures. 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: (1) markets into which we sell our products declining or not growing as anticipated; (2) fluctuations in the global economic and political environments, including as the result of recently implemented and recently threatened tariff increases; (3) our failure to introduce new products in a timely manner; (4) our ability to execute acquisitions and divestitures, license technologies, or to successfully integrate acquired businesses or licensed technologies into our existing businesses or to make them profitable; (5) our ability to compete effectively; (6) fluctuation in our quarterly operating results and our ability to adjust our operations to address unexpected changes; (7) significant disruption in third-party package delivery and import/export services or significant increases in prices for those services; (8) disruptions in the supply of raw materials and supplies; (9) our ability to retain key personnel; (10) significant disruption in our information technology systems, or cybercrime; (11) our ability to realize the full value of our intangible assets; (12) our failure to adequately protect our intellectual property; (13) the loss of any of our licenses or licensed rights; (14) the manufacture and sale of products exposing us to product liability claims; (15) our failure to maintain compliance with applicable government regulations; (16) our failure to comply with data privacy and information security laws and regulations; (17) regulatory changes; (18) our failure to comply with healthcare industry regulations; (19) economic, political and other risks associated with foreign operations; (20) our ability to obtain future financing; (21) restrictions in our credit agreements; (22) significant fluctuations in our stock price; (23) reduction or elimination of dividends on our common stock; and (24) other factors which we describe under the caption “Risk Factors” in our most recent annual report on Form 10-K 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 2024 revenue of more than $2.7 billion and approximately 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 160 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.

We use the term “adjusted gross margin” to refer to GAAP gross margin, excluding amortization of intangible assets and inventory fair value adjustments related to business acquisitions and asset impairments. We use the related term “adjusted gross margin percentage” to refer to adjusted gross margin as a percentage of revenue.

We use the term “adjusted SG&A expense” to refer to GAAP SG&A expense, excluding amortization of intangible assets, purchase accounting adjustments, acquisition and divestiture-related expenses, significant litigation matters and settlements, asset impairments, significant environmental charges, and restructuring and other charges. We use the related term “adjusted SG&A percentage” to refer to adjusted SG&A expense as a percentage of revenue.

We use the term “adjusted R&D expense” to refer to GAAP R&D expense, excluding amortization of intangible assets and purchase accounting adjustments. We use the related term “adjusted R&D percentage” to refer to adjusted R&D expense as a percentage of revenue.

We use the term “adjusted net interest and other expense” to refer to GAAP net interest and other expense, excluding adjustments for mark-to-market accounting on post-retirement benefits, changes in foreign exchange and interest associated with acquisitions and divestitures, changes in the value of investments and debt extinguishment costs.

We use the term “adjusted operating income” to refer to GAAP operating income, excluding amortization of intangible assets, other purchase accounting adjustments, acquisition and divestiture-related expenses, significant litigation matters and settlements, significant environmental charges, asset impairments, and restructuring and other charges. We use the related terms “adjusted operating profit percentage,” “adjusted operating profit margin,” and “adjusted operating margin” to refer to adjusted operating income as a percentage of revenue.

We use the term “free cash flow” to refer to net cash provided by (used in) operating activities of continuing operations, less payments for additions to property, plant and equipment from continuing operations (“capital expenditures”) plus the proceeds from sales of plant, property and equipment from continuing operations (“capital disposals”).

We use the term “adjusted net income” to refer to GAAP income from continuing operations, excluding amortization of intangible assets, debt extinguishment costs, other purchase accounting adjustments, acquisition and divestiture-related expenses, significant litigation matters and settlements, significant environmental charges, changes in the value of investments, disposition of businesses and assets, net, changes in foreign exchange and interest associated with acquisitions and divestitures, asset impairments and restructuring and other charges. We also exclude adjustments for mark-to-market accounting on post-retirement benefits, therefore only our projected costs have been used to calculate this non-GAAP measure. We also adjust for any tax impact related to the above items and exclude the impact of significant tax events.

We use the term “adjusted earnings per share from continuing operations,” “adjusted earnings per share,” “adjusted EPS,” or “adjusted EPS from continuing operations” to refer to GAAP earnings per share from continuing operations, excluding amortization of intangible assets, debt extinguishment costs, other purchase accounting adjustments, acquisition and divestiture-related expenses, significant litigation matters and settlements, significant environmental charges, changes in the value of investments, disposition of businesses and assets, net, changes in foreign exchange and interest associated with acquisitions and divestitures, asset impairments and restructuring and other charges.

Contacts

Investor Relations:
Steve Willoughby

steve.willoughby@revvity.com

Media Relations:
Chet Murray

(781) 462-5126

chet.murray@revvity.com

Read full story here

Astoriom Appoints Ryan Smith as Global Head of Sales




Astoriom Appoints Ryan Smith as Global Head of Sales

Key appointment to support company in scaling biorepository and stability storage operations

ROCHDALE, England–(BUSINESS WIRE)–#Astoriom–Astoriom, a global leader in the R&D sample stability and biorepository storage industry, today announced the appointment of Ryan Smith as Global Head of Sales. He will build on the strong foundation already in place to further scale Astoriom’s portfolio of sample stability storage, biorepository storage, disaster protection and recovery, as well as sample storage equipment and validation services. Ryan’s appointment demonstrates the company’s commitment to its evolving commercial strategy and continued expansion in key markets worldwide, enabling scientists to safeguard the integrity and viability of their valuable sample assets to advance their research.

Ryan has over two decades of experience leading global commercial teams in life sciences to deliver revenue growth, optimize commercial processes, and scale global operations. His experience as a senior global sales leader has been in outsourced biorepository services and automated sample storage and cryogenic capital equipment, serving the biopharmaceutical, biotech and academic research industries. Most recently, Ryan led the global sales team at Azenta Life Sciences (formerly Brooks Life Sciences). Prior to this, his work focused on nanotechnology at Agilent/Keysight Technologies and RHK Technologies.

Ryan’s appointment to Global Head of Sales comes at a time of significant growth and expansion for Astoriom in North America and Europe. His knowledge of automated sample storage, specimen management, and the regulatory demands in R&D and manufacturing environments further strengthens Astoriom’s ability to support its scientific research and product development customers, and to assist them in navigating complex regulatory compliance and business growth challenges. Ryan has a B.S. in Human Biology with a minor in Business Administration from the University of Indianapolis.

Lori A. Ball, CEO, Astoriom, said: “We’re committed to investing in world-class leadership to deliver exceptional service, innovation, and global scalability. Ryan’s appointment as Global Head of Sales marks a pivotal moment in Astoriom’s growth trajectory. He brings a deep understanding of the unique demands of R&D, regulatory compliance, and expanding biopharma operations. Having delivered record growth across specimen management, capital equipment and outsourced services, his leadership will be instrumental in driving Astoriom’s global commercial strategy and reinforces our readiness for accelerated growth and market leadership.”

Ryan Smith, Global Head of Sales, Astoriom, said: “Astoriom is committed to delivering industry-leading stability storage and biorepository services worldwide, providing critical solutions in a challenging time for organizations to scale while meeting evolving regulatory requirements. I very much look forward to being a part of a company that is well-positioned to be the global industry leader within the R&D sample stability and biorepository services market and building on the strong foundation they’ve created. Having the opportunity to apply the knowledge, processes and strategies I’ve developed over the last 20 years at this phase of the company’s growth is really exciting.”

For more information about the Astoriom team visit: https://www.astoriom.com/about/.

Contacts

Please contact Codon Communications for high-resolution images.

Codon Communications
Dr. Michelle Ricketts

Tel: +44 7789 053885

Email: michelle.ricketts@codoncommunications.com

Metrion Biosciences Launches Nav1.9 High-Throughput Screening Assay to Strengthen Screening Portfolio and Advance Research on New Medicines for Pain




Metrion Biosciences Launches Nav1.9 High-Throughput Screening Assay to Strengthen Screening Portfolio and Advance Research on New Medicines for Pain

• Validated drug discovery assay overcomes previous challenges in targeting Na_V1.9 ion channels for chronic pain signalling
• Completes unique suite of rapid, scalable pain-related sodium channel assays and services to accelerate hit-to-lead and lead optimisation programmes

CAMBRIDGE, England–(BUSINESS WIRE)–Metrion Biosciences (“Metrion”), the specialist preclinical contract research organisation (CRO) and a global leader in ion channel services, today announced the launch of its validated, high-throughput Na_V1.9 screening assay to advance discovery and development of novel pain therapeutics. Leveraging over a decade of electrophysiology expertise, the Na_V1.9 assay, alongside Metrion’s unique combination of ion channel expertise, bespoke assays and pain research services, enables researchers to overcome traditional limitations of Na_V1.9 screening and generate reproducible and decision-ready data.

Na_V1.9 is a voltage-gated sodium channel selectively expressed in peripheral sensory neurones that plays a key role in pain signalling. Mutations in Na_V1.9 are associated with both severe pain and pain insensitivity in humans. Despite its potential as a non-opioid therapeutic target, research has been limited by difficulties developing stable heterologous expression systems.

Metrion’s new Na_V1.9 assay complements the Company’s existing portfolio of efficacy and safety screening assays, adding new capabilities to accelerate and de-risk preclinical programmes, unlocking deeper insights into Na_V1.9 pharmacology. Designed using a stable and validated CHO cell line, the assay has been developed and optimised in-house for high reproducibility and low variability and is available using both human- and rat-derived clones, providing insights into species selectivity for the development of more efficacious therapeutics.

The assay completes the Company’s full suite of pain-related sodium channel assays to provide selectivity profiling across Na_V1.1 to Na_V1.9. Metrion’s offering features a comprehensive portfolio of off-target counter screens, including other pain related ion channel targets and a CiPA panel for cardiac safety risk assessment. The Company also provides access to manual clamp-based mechanistic and translational assays, and automated patch clamp using the Qube 384 to provide highly sensitive, rapid analysis of large candidate libraries. Additional support for hit-to-lead and lead optimisation, streamlining compound evaluation and reducing project timelines is also provided.

“The availability of effective assays to study the Na_V1.9 sodium channel has been a major stumbling block that has held back development of the next generation of non-opioid pain therapeutics,” said Dr Eddy Stevens, Chief Scientific Officer, Metrion Biosciences. “Metrion is now able to offer a unique combination of sodium channel expertise, high-throughput screening solutions and research services. These cover the full suite of pain-related sodium channels. By facilitating streamlined compound evaluation and accelerated lead optimisation, this service offering has the potential to bring novel pain therapeutics to market rapidly and more cost-effectively. This important launch represents a major milestone for Metrion, a testament to the dedication and knowledge of our team and reinforces our position as leading the field in ion channel drug discovery.”

To find out more about Metrion’s unique approach to unlock Na_V1.9 for breakthrough pain treatments, please visit: www.metrionbiosciences.com/neuroscience/nav1-9-assays/

Contacts

Media Contact
Jake Brown

Zyme Communications

E-mail: jake.brown@zymecommunications.com
Tel: +44 (0) 7759 162 147

Merck KGaA, Darmstadt, Germany, to Acquire US Biopharma Company SpringWorks Therapeutics to Accelerate Sustainable Growth of Healthcare Business




Merck KGaA, Darmstadt, Germany, to Acquire US Biopharma Company SpringWorks Therapeutics to Accelerate Sustainable Growth of Healthcare Business

• Purchase price of $47 per share in cash represents an enterprise value of €3.0 billion ($3.4 billion), or an equity value of approximately $3.9 billion
• Planned acquisition will immediately add revenue and accelerate mid- to long-term growth for the Healthcare business of Merck KGaA, Darmstadt, Germany
• SpringWorks Therapeutics is a U.S. biopharmaceutical company with a first-in-class, systemic standard-of-care therapy in adults with desmoid tumors and the first and only approved therapy for adults and children with neurofibromatosis type 1-associated plexiform neurofibromas
• Planned acquisition will strengthen the presence of the Healthcare business of Merck KGaA, Darmstadt, Germany, in the United States and expand reach of SpringWorks’ therapeutic innovations to more patients with rare tumors worldwide

Not intended for UK-based media

DARMSTADT, Germany–(BUSINESS WIRE)–Merck KGaA, Darmstadt, Germany (DAX: MRK), a leading science and technology company, and SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a Stamford, Connecticut-based commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, today announced the companies have entered into a definitive agreement for Merck KGaA, Darmstadt, Germany, to acquire SpringWorks. The purchase price of $47 per share in cash represents an equity value of approximately $3.9 billion, or an enterprise value of $3.4 billion (€3.0 billion) based on SpringWorks’ cash balance as of December 31, 2024, and a premium of 26% to SpringWorks’ unaffected 20-day volume-weighted average price of $37.38 on February 7, 2025, the day prior to the first market speculation of a potential transaction between Merck KGaA, Darmstadt, Germany, and SpringWorks.

“The agreed acquisition of SpringWorks is a major step in our active portfolio strategy to position our company as a globally diversified, innovation and technology powerhouse. For our Healthcare sector, it sharpens the focus on rare tumors, accelerates growth, and strengthens our presence in the U.S.,” said Belén Garijo, Chair of the Executive Board and CEO of Merck KGaA, Darmstadt, Germany. “Beyond this planned transaction, we will continue to explore M&A opportunities across our three complementary business sectors, always with a firm focus on strategic fit, financial robustness, and long-term value creation.”

The planned transaction is fully aligned with the business development/M&A priorities of the Healthcare business of Merck KGaA, Darmstadt, Germany, as outlined during the company’s Capital Markets Day in October 2024: to continue to pursue external innovation via in-licensing of high-quality compounds at various stages of development and focused acquisitions that promise early value creation. It also fits with the strategic objective of strengthening the presence of the Healthcare business of Merck KGaA, Darmstadt, Germany, in the United States, the world’s largest pharmaceutical market. Merck KGaA, Darmstadt, Germany, operates its Healthcare business as EMD Serono in the United States and Canada.

Upon closing, the business combination will immediately contribute to the revenues of Merck KGaA, Darmstadt, Germany, and is expected to be accretive to the company’s earnings per share pre (EPS pre) in 2027. The acquisition will be funded with available cash and new debt. Beyond this planned transaction, Merck KGaA, Darmstadt, Germany, will retain the ability to pursue larger transactions and continue to evaluate opportunities across its three sectors, with Life Science a priority. Merck KGaA, Darmstadt, Germany is committed to preserving its strong investment grade credit rating.

SpringWorks’ rare tumor portfolio, including a marketed first-in-class, systemic standard-of-care therapy for adults with desmoid tumors and the first and only approved therapy for adults and children with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection, will accelerate immediate and sustainable revenue growth for Merck KGaA, Darmstadt, Germany. SpringWorks’ portfolio complements the progress of Merck KGaA, Darmstadt, Germany, in rare tumors, with Merck KGaA, Darmstadt, Germany, recently exercising an option for worldwide commercialization rights for pimicotinib, an investigational therapy developed by Abbisko Therapeutics Co., Ltd. for patients with tenosynovial giant cell tumor (TGCT).

“We have the unique opportunity with SpringWorks to establish a leadership position in rare tumors and build a strong foundation for further investments in this area, where a large unmet medical need exists,” said Peter Guenter, member of the Executive Board and CEO of Healthcare at Merck KGaA, Darmstadt, Germany. “Together, our company and SpringWorks are the perfect combination to improve outcomes for patients with rare tumors and bring therapeutic innovations to more patients worldwide while building on and reinforcing the early success of SpringWorks in the United States. For us, the planned acquisition will create long term, sustainable growth for our Healthcare business. Along with my successor Danny Bar-Zohar, we look forward to completing this strategic transaction and making a meaningful difference for patients whose lives are so profoundly affected by these complex and challenging tumors.”

The agreed acquisition provides SpringWorks with an opportunity to expand its reach into markets beyond the U.S. and leverage the breadth of resources of the global Healthcare organization of Merck KGaA, Darmstadt, Germany.

“From the outset, our focus at SpringWorks has been to create transformative solutions for patients suffering from serious diseases. We have successfully launched two best-in-class medicines in the United States, and with the aspiration to deliver our therapies worldwide, our journey is at a pivotal juncture. It became clear during our discussions with the team of Merck KGaA, Darmstadt, Germany that we share many core values, including a commitment to help more patients with rare tumors live longer, better lives,” said Saqib Islam, CEO of SpringWorks Therapeutics. “We believe that by joining forces with Merck KGaA, Darmstadt, Germany, we are not only creating significant, immediate value for our stakeholders, but we will also be able to leverage their resources and expertise to build a brighter future for the patient communities we seek to serve while also creating new opportunities for SpringWorks employees as part of a global organization.”

SpringWorks’ U.S. Food and Drug Administration (FDA)-approved therapy, OGSIVEO^® (nirogacestat) is a first-in-class therapy that is the systemic standard of care for the treatment of adult patients with progressing desmoid tumors who require systemic treatment. SpringWorks’ marketing authorization application (MAA) for nirogacestat is under review with the European Medicines Agency (EMA), with a Committee for Medicinal Products for Human Use (CHMP) decision expected in Q2 2025.

GOMEKLI™ (mirdametinib) is the first and only FDA-approved therapy for the treatment of adult and pediatric patients 2 years of age and older with NF1-PN not amenable to complete resection. The FDA’s February 2025 approval of GOMEKLI was based on positive data from SpringWorks’ Phase 2b ReNeu trial, which showed GOMEKLI treatment resulted in a robust objective response rate, deep and durable reductions in tumor volume, and a manageable safety profile. With the approval, SpringWorks was granted a rare pediatric disease priority review voucher by the FDA. The marketing authorisation application for mirdametinib has been validated by the European Medicines Agency (EMA) with a potential approval in 2025. In addition, SpringWorks is advancing its pipeline with additional programs in other tumor settings that are currently underserved.

The transaction has been unanimously approved, by all those in attendance, by both the Merck KGaA, Darmstadt, Germany, and SpringWorks Boards of Directors and is expected to close in the second half of 2025, subject to satisfaction of customary closing conditions, including approval of SpringWorks’ shareholders and receipt of required regulatory approvals.

J.P. Morgan is acting as exclusive financial advisor and Sullivan & Cromwell LLP is acting as legal counsel to Merck KGaA, Darmstadt, Germany. Centerview Partners LLC and Goldman Sachs & Co. LLC are acting as joint financial advisors to SpringWorks, and Goodwin Procter LLP is acting as SpringWorks’ legal counsel.

About Merck KGaA, Darmstadt, Germany

Merck KGaA, Darmstadt, Germany, a leading science and technology company, operates across life science, healthcare and electronics. More than 62,000 employees work to make a positive difference to millions of people’s lives every day by creating more joyful and sustainable ways to live. From providing products and services that accelerate drug development and manufacturing as well as discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices – the company is everywhere. In 2024, Merck KGaA, Darmstadt, Germany, generated sales of € 21.2 billion in 65 countries.

The company holds the global rights to the name and trademark “Merck” internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany, operate as MilliporeSigma in life science, EMD Serono in healthcare and EMD Electronics in electronics. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company’s technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company.

All Merck KGaA, Darmstadt, Germany, press releases are distributed by e-mail at the same time they become available on the EMD Group website. In case you are a resident of the USA or Canada, please go to www.emdgroup.com/subscribe to register for your online, change your selection or discontinue this service.

About SpringWorks Therapeutics

SpringWorks is a commercial-stage biopharmaceutical company dedicated to improving the lives of patients with severe rare diseases and cancer. We developed and are commercializing OGSIVEO® (nirogacestat) as the first and only FDA-approved medicine for adults with desmoid tumors and GOMEKLI™ (mirdametinib) as the first and only FDA-approved medicine for both adults and children with neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN). We are also advancing a diverse portfolio of novel targeted therapy product candidates for patients with both solid tumors and hematological cancers.

For more information, visit www.springworkstx.com and follow @SpringWorksTx on X, LinkedIn, Facebook, Instagram, and YouTube.

No Solicitation

Merck KGaA, Darmstadt, Germany, its directors and executive officers are not soliciting proxies from the shareholders of SpringWorks in connection with the proposed acquisition and are not participants in the solicitation of proxies by SpringWorks. Merck KGaA, Darmstadt, Germany, is making this communication for informational purposes only and does not intend to file any communication relating to the proposed acquisition on a proxy statement on Schedule 14A with the SEC.

Contacts

Media Relations
gangolf.schrimpf@emdgroup.com
Phone: +49 151 1454-9591

Investor Relations
investor.relations@emdgroup.com
Phone: +49 6151 72-3321

Prilenia Enters into a Collaboration and License Agreement with Ferrer for the Commercialization and Co-Development of Pridopidine in Europe and Other Select Markets




Prilenia Enters into a Collaboration and License Agreement with Ferrer for the Commercialization and Co-Development of Pridopidine in Europe and Other Select Markets

— Total deal size of approximately €500 million, including approximately €125 million in upfront and near-term milestones —

— Ferrer to commercialize pridopidine in Europe and other select markets; Prilenia retains full commercialization and development rights to pridopidine in North America, Japan and Asia Pacific —

— Co-development agreement in the territory supports further expansion of pridopidine in Huntington’s disease, amyotrophic lateral sclerosis and future indications —

— Pridopidine for Huntington’s disease is currently under review by the European Medicines Agency (EMA) with a CHMP opinion expected in the second half of 2025 —

NAARDEN, Netherlands & WALTHAM, Mass.–(BUSINESS WIRE)–Prilenia Therapeutics B.V., a biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS), today announced that it has entered into a collaboration and license agreement with Ferrer for the commercialization and further development of pridopidine in Europe and other select markets. Pridopidine is a potent and highly selective, orally administered sigma-1 receptor (S1R) agonist designed to regulate key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS.

Under the terms of the agreement, Prilenia will receive an upfront payment of approximately €80 million plus up to €45 million in near-term development, regulatory, and commercial milestones. The total deal is valued at up to approximately €500 million in upfront and total milestone payments. In addition, Prilenia will receive tiered double-digit royalties on net sales. Prilenia and Ferrer have agreed to jointly develop and fund the expansion of pridopidine in the territory for additional indications beyond HD. Prilenia will retain full rights to pridopidine in other major markets, including North America, Japan and Asia Pacific.

“We are proud to partner with Ferrer as we advance our shared mission to bring transformative therapies to people living with neurodegenerative diseases around the world,” said Dr. Michael R. Hayden, CEO of Prilenia. “Ferrer continues to grow their already significant presence throughout Europe and key international markets with particular focus on innovative products for rare diseases. By combining our unique strengths and shared commitment to these patient communities, we believe that this partnership has the potential to accelerate the delivery of pridopidine to the thousands of people who are waiting for a new treatment option as well as broaden its impact through additional indications in the future.”

“This agreement with Prilenia means we can continue making our purpose of using business to fight for social justice a reality, while focusing our pipeline development on diseases with high unmet medical need,” stated Mario Rovirosa, CEO of Ferrer. “The combination of strengths and capabilities of our two companies makes the future brighter for the patients suffering from such underserved conditions.”

“Securing rights to this molecule represents a pivotal step in our research strategy in the neurodegeneration arena,” said Oscar Pérez, Chief Scientific and Business Development Officer at Ferrer. “Given the mechanism of action of Pridopidine, we are fully committed to exploring its potential use across a range of indications.”

About Pridopidine

Pridopidine (45 mg twice daily) is a potent and highly selective, orally administered sigma-1 receptor (S1R) agonist designed to regulate key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS.ⁱ

In its extensive HD development program, pridopidine has demonstrated benefits across key features of the disease impacting quality of life for patients and families, including function, cognition and motor skills, measured by validated assessments and sustained for up to two years, with a favorable safety profile.

Prilenia has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA), seeking regulatory approval of pridopidine for the treatment of HD. Our MAA has been accepted for review, and we expect an opinion from the Commission for Medicinal Human Products (CHMP) in the second half of 2025. This is the first submission seeking approval for a potential treatment that can impact disease progression in HD.

We also are in ongoing discussions with the U.S. Food and Drug Administration (FDA) to determine the next steps for pridopidine in HD in the U.S. If approved, Prilenia will continue to work expeditiously to make pridopidine available to HD patients.

For ALS, Prilenia and Ferrer plan to initiate a single, pivotal Phase 3 trial to evaluate pridopidine, seeking to confirm findings from the Phase 2 HEALEY ALS Platform Trial.

Prilenia holds Orphan Drug designation for pridopidine in HD and ALS in the U.S. and EU. In addition, pridopidine has received Fast Track designation by the FDA for the treatment of HD.

About Huntington’s Disease

Huntington’s disease (HD) is a rare, inherited, autosomal dominant, neurodegenerative disease that results in functional, motor, cognitive and behavioral symptoms. HD is caused by a mutation in the huntingtin gene, and each child of a parent with HD has a 50 percent chance of developing the disease.

HD affects approximately 100,000 people around the world with an additional 300,000 people at risk of developing HD.^i,ii It is usually diagnosed between the ages of 30 and 50, although HD can occur at any age, including in children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage day-to-day life and take care of themselves. This increasing disability leads to full reliance on a caregiver and, ultimately, death.

The only currently available treatments for HD focus on symptomatic relief and palliative care, with nothing impacting measures of overall progression.

About Amyotrophic Lateral Sclerosis (ALS)

ALS, also known as Lou Gehrig’s Disease or Motor Neuron Disease, is a chronic progressive neurodegenerative disease affecting approximately 350,000 people worldwide.

In people with ALS, motor neurons in the brain and spinal cord that convey messages to the muscles degenerate, affecting the brain’s ability to communicate with muscles. This leads to muscle wasting and progressive paralysis. Patients rapidly lose their ability to walk, speak, eat, and breathe, and become fully dependent on their caretakers. The average life span from diagnosis is 2 to 5 years.

The majority of ALS cases (~90%) are without a family history of the disease. About 10% of ALS cases are caused by inherited genetic mutations (often called familial ALS). One of the genes discovered to cause ALS encodes the sigma-1 receptor (S1R) protein. Mutations in this gene that result in complete loss of function of the S1R are associated with severe, juvenile ALS, while mutations resulting in partial, incomplete function of the S1R are associated with adult-onset ALS.

About Prilenia

Prilenia is a private biopharmaceutical company driven by an unwavering commitment to scientific excellence and accelerating progress for people affected by Huntington’s disease (HD) and amyotrophic lateral sclerosis (ALS). Our mission is simple but urgent: to develop and provide sustainable access to transformative medicines for people affected by devastating neurodegenerative diseases.

Prilenia operates across the United States, Canada, Europe and Israel. The company is incorporated in the Netherlands and backed by leading life sciences investors.

For more information, please visit www.prilenia.com and connect with us on LinkedIn or X (Twitter).

Prilenia Forward Looking Statements

Prilenia cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: advancing development of and commercializing pridopidine, the potential benefits and value of pridopidine; and the potential benefits and outcome from this collaboration. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include uncertainties in clinical development, regulatory approval and commercialization processes. Prilenia cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof.

^©2025 Prilenia Therapeutics B.V.

For a copy of this release, visit Prilenia’s website at www.prilenia.com.

ⁱ Medina et al., Prevalence and Incidence of Huntington’s Disease: An Updated Systematic Review and Meta-Analysis. Mov Disord. 2022 Dec;37(12):2327-2335.

ⁱⁱ Jiang, A., Handley, R. R., Lehnert, K., & Snell, R. G. (2023). From Pathogenesis to Therapeutics: A Review of 150 Years of Huntington’s Disease Research. International Journal of Molecular Sciences, 24(16), 13021. https://doi.org/10.3390/ijms241613021

Contacts

Prilenia Contact
Communications Team

info@prilenia.com

Number of Shares and Voting Rights of Innate Pharma as of April 25, 2025




Number of Shares and Voting Rights of Innate Pharma as of April 25, 2025

MARSEILLE, France–(BUSINESS WIRE)–Regulatory News:

Pursuant to the article L. 233-8 II of the French “Code de Commerce” and the article 223-16 of the French stock-market authorities (Autorité des Marchés Financiers, or “AMF”) General Regulation, Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) releases its total number of shares outstanding as well as its voting rights as at April 25, 2025:

(1) The total number of theoretical voting rights (or “gross” voting rights) is used as the basis for calculating the crossing of shareholding thresholds. In accordance with Article 223-11 of the AMF General Regulation, this number is calculated on the basis of all shares to which voting rights are attached, including shares whose voting rights have been suspended. The total number of theoretical voting rights includes voting rights attached to AGAP 2016, i.e. 130 voting rights for the AGAP 2016-1 and 111 voting rights for the AGAP 2016-2. No voting rights attached to AGAP 2017.

(2) The total number of exercisable voting rights (or “net” voting rights) is calculated without taking into account the shares held in treasury by the Company, with suspended voting rights. It is released so as to ensure that the market is adequately informed, in accordance with the recommendation made by the AMF on July 17, 2007.

About Innate Pharma

Innate Pharma S.A. is a global, clinical-stage biotechnology company developing immunotherapies for cancer patients. Its innovative approach aims to harness the innate immune system through three therapeutic approaches: multi-specific NK Cell Engagers via its ANKET^® (Antibody-based NK cell Engager Therapeutics) proprietary platform, Antibody Drug Conjugates (ADC) and monoclonal antibodies (mAbs).

Innate’s portfolio includes several ANKET^® drug candidates to address multiple tumor types as well as IPH4502, a differentiated ADC in development in solid tumors. In addition, anti-KIR3DL2 mAb lacutamab is developed in advanced form of cutaneous T cell lymphomas and peripheral T cell lymphomas, and anti-NKG2A mAb monalizumab is developed with AstraZeneca in non-small cell lung cancer.

Innate Pharma is a trusted partner to biopharmaceutical companies such as Sanofi and AstraZeneca, as well as leading research institutions, to accelerate innovation, research and development for the benefit of patients.

Headquartered in Marseille, France with a US office in Rockville, MD, Innate Pharma is listed on Euronext Paris and Nasdaq in the US.

Learn more about Innate Pharma at www.innate-pharma.com and follow us on LinkedIn and X.

Information about Innate Pharma shares

Disclaimer on forward-looking information and risk factors

This press release contains certain forward-looking statements, including those within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. The use of certain words, including “anticipate,” “believe,” “can,” “could,” “estimate,” “expect,” “may,” “might,” “potential,” “expect” “should,” “will,” or the negative of these and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. These risks and uncertainties include, among other things, the uncertainties inherent in research and development, including related to safety, progression of and results from its ongoing and planned clinical trials and preclinical studies, review and approvals by regulatory authorities of its product candidates, the Company’s reliance on third parties to manufacture its product candidates, the Company’s commercialization efforts and the Company’s continued ability to raise capital to fund its development. For an additional discussion of risks and uncertainties, which could cause the Company’s actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors (“Facteurs de Risque”) section of the Universal Registration Document filed with the French Financial Markets Authority (“AMF”), which is available on the AMF website http://www.amf-france.org or on Innate Pharma’s website, and public filings and reports filed with the U.S. Securities and Exchange Commission (“SEC”), including the Company’s Annual Report on Form 20-F for the year ended December 31, 2023, and subsequent filings and reports filed with the AMF or SEC, or otherwise made public by the Company. References to the Company’s website and the AMF website are included for information only and the content contained therein, or that can be accessed through them, are not incorporated by reference into, and do not constitute a part of, this press release.

In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by the Company or any other person that the Company will achieve its objectives and plans in any specified time frame or at all. The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

This press release and the information contained herein do not constitute an offer to sell or a solicitation of an offer to buy or subscribe to shares in Innate Pharma in any country.

Contacts

For additional information, please contact:


Investors


Innate Pharma
Henry Wheeler

Tel.: +33 (0)4 84 90 32 88

Henry.wheeler@innate-pharma.fr

Media Relations


NewCap
Arthur Rouillé

Tel.: +33 (0)1 44 71 00 15

innate@newcap.eu

Ferrer Enters Into a Collaboration and License Agreement With Prilenia for the Commercialization and Co-Development of Pridopidine




Ferrer Enters Into a Collaboration and License Agreement With Prilenia for the Commercialization and Co-Development of Pridopidine

BARCELONA, Spain–(BUSINESS WIRE)–#FerrerForGood—Ferrer, an international B Corp pharmaceutical company with an increasing focus on rare neurological diseases, and Prilenia Therapeutics B.V., a clinical-stage biotech company, have announced the signing of a strategic co-development and license agreement in which Ferrer obtains the rights to develop, manufacture and commercialize Pridopidine in the European Region, the Middle East and North African Region, the Southern African Region, the Central and South American Region, and the Commonwealth of Independent States Region.

Pridopidine, a potent and highly selective, orally administered sigma-1 receptor agonist, designed to regulate key neuroprotective mechanisms that are often impaired in neurodegenerative diseases, is a promising candidate for the treatment of Huntington’s Disease (HD), a rare inherited neurodegenerative disease, with a high unmet medical need¹. It has been studied in more than 1,700 people and long-term safety data of up to 7 years duration are available from previous clinical studies². These investigational studies demonstrate that at the therapeutic dose, Pridopidine has an observed safety and tolerability profile comparable to placebo².

Pridopidine is currently being reviewed by the European Medicines Agency, which has accepted the Marketing Authorisation Application submission for the treatment of HD. An opinion from the Committee for Medicinal Products for Human Use is expected in the second half of 2025.

The terms of the agreement include an upfront payment and multiple regulatory, development, commercial and sales milestone payments. Prilenia will also receive tiered double-digit royalties on net sales of Pridopidine.

“This agreement with Prilenia means we can continue making our purpose of using business to fight for social justice a reality, while focusing our pipeline development on diseases with high unmet medical need,” stated Mario Rovirosa, CEO of Ferrer. “The combination of strengths and capabilities of our two companies makes the future brighter for the patients suffering from such underserved conditions.”

“We are proud to partner with Ferrer as we advance our shared mission to bring transformative therapies to people living with neurodegenerative diseases around the world,” said Dr. Michael R. Hayden, CEO of Prilenia. “Ferrer continues to grow their already significant presence throughout Europe and key international markets with particular focus on innovative products for rare diseases. By combining our unique strengths and shared commitment to these patient communities, we believe that this partnership has the potential to accelerate the delivery of Pridopidine to the thousands of people who are waiting for a new treatment option as well as to broaden its impact through additional indications in the future.”

“Securing rights to this molecule represents a pivotal step in our research strategy in the neurodegeneration arena,” said Oscar Pérez, Chief Scientific and Business Development Officer at Ferrer. “Given the mechanism of action of Pridopidine, we are fully committed to exploring its potential use across a range of indications.”

References:

1. Naia L, Ly P, Mota SI, Lopes C, Maranga C, Coelho P, Gershoni-Emek N, Ankarcrona M, Geva M, Hayden MR, Rego AC. The Sigma-1 Receptor Mediates Pridopidine Rescue of Mitochondrial Function in Huntington Disease Models. Neurotherapeutics. 2021 Apr;18(2):1017-1038. doi: 10.1007/s13311-021-01022-9. Epub 2021 Apr 1. PMID: 33797036; PMCID: PMC8423985.
2. Goldberg YP, Navon-Perry L, Cruz-Herranz A, Chen K, Hecker-Barth G, Spiegel K, Cohen Y, Niethammer M, Tan AM, Schuring H, Geva M, Hayden MR. The Safety Profile of Pridopidine, a Novel Sigma-1 Receptor Agonist for the Treatment of Huntington’s Disease. CNS Drugs. 2025 May;39(5):485-498. doi: 10.1007/s40263-025-01171-x. Epub 2025 Mar 7. PMID: 40055280; PMCID: PMC11982116.

Contacts

Media contact:

Alba Soler

Director of Communication at Ferrer

asolerc@ferrer.com
+34 936 003 779

OLYMPUS Long-Term Follow-Up Study Reports Nearly Four-Year Duration of Response in Subset of Patients with Low-Grade Upper Tract Urothelial Cancer Who Achieved a Complete Response to Initial JELMYTO Treatment




OLYMPUS Long-Term Follow-Up Study Reports Nearly Four-Year Duration of Response in Subset of Patients with Low-Grade Upper Tract Urothelial Cancer Who Achieved a Complete Response to Initial JELMYTO Treatment

• Data Presented at the 2025 American Urological Association Annual Meeting in Las Vegas, Nevada

PRINCETON, N.J.–(BUSINESS WIRE)–UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, today highlights a duration of response of nearly four years from a long-term follow-up study with JELMYTO^® (mitomycin) for pyelocalyceal solution, which is FDA-approved for the treatment of low-grade upper tract urothelial cancer (LG-UTUC) in adult patients.

Among patients from the OLYMPUS trial who achieved a complete response after primary chemoablation with JELMYTO (n=41), the median duration of response was 47.8 months, irrespective of whether their cancer was new-onset or recurrent (median follow-up 28.1 months [95% CI 13.1, 57.5]). Of these patients, 21 had new-onset UTUC, and 20 had recurrent UTUC at baseline; there were no significant differences in durability between groups, with 8 patients in each group experiencing recurrence or death not due to treatment. Twenty patients entered long-term follow-up with a median follow-up of 53.3 months (95% CI 27.9, 65.3); median duration of response was not estimable (95% CI 43.5, not estimable) due to the low event rate.

“This sub-analysis highlights new evidence for the impressive long-term benefits of JELMYTO, in both new-onset and recurrent low-grade upper tract urothelial cancer patients,” said Brian Hu, MD, Associate Professor of Urology at Loma Linda University Health, CA. “These findings further establish JELMYTO as an effective treatment, providing patients with durable responses and a significant reduction in recurrence rates.”

LG-UTUC is a challenging disease often managed through endoscopically guided ablation, but recurrence is common, and patients face a lifetime of surveillance and potential complications. The OLYMPUS Phase 3 trial evaluated JELMYTO, a reverse thermal gel containing mitomycin (4 mg/mL), as a primary treatment for adults with LG-UTUC. While the overall trial demonstrated the potential of JELMYTO to significantly eradicate disease, this sub-analysis focuses specifically on patients with new-onset and recurrent LG-UTUC who achieved a complete response, providing new insights into the durability and long-term effectiveness of the treatment. Limitations of this study include the post-hoc nature of the analysis and potential selection bias regarding entry of patients into long-term follow-up.

“We are encouraged by the long-term outcomes evidence observed in both new-onset and recurrent LG-UTUC patients,” said Mark Schoenberg, MD, Chief Medical Officer, UroGen. “The sub-analysis provides further compelling evidence that JELMYTO offers durable disease control and clinically meaningful responses, which could significantly reduce the need for repeated interventions. We look forward to expanding our understanding of JELMYTO through the ongoing uTRACT Registry and further evaluating its potential in real-world settings.”

To further explore the potential of JELMYTO in treating patients with LG-UTUC, investigators are currently enrolling participants in the JELMYTO uTRACT Registry (NCT05874921) to gather longitudinal real-world usage data. As of April 2025, 22 sites have been activated with 251 patients enrolled.

About JELMYTO

JELMYTO^® (mitomycin) for pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel approved for the treatment of adult patients with LG-UTUC. JELMYTO is a viscous liquid when cooled and becomes a semi-solid gel at body temperature. The drug slowly dissolves over four to six hours after instillation and is removed from the urinary tract by normal urine flow and voiding. It is approved for administration in a retrograde manner via ureteral catheter or antegrade through a nephrostomy tube. The delivery system allows the initial liquid to coat and conform to the upper urinary tract anatomy. The eventual semisolid gel allows for chemoablative therapy to remain in the collecting system for four to six hours without immediately being diluted or washed away by urine flow.

About Upper Tract Urothelial Cancer

Urothelial cancer is the ninth most common cancer globally and the eighth most lethal neoplasm in men in the U.S. Between five percent and ten percent of primary urothelial cancers originate in the ureter or renal pelvis and are collectively referred to as UTUC. In the U.S., there are approximately 6,000 – 7,000 new or recurrent LG-UTUC patients annually. Most cases are diagnosed in patients over 70 years old, and these older patients often have multiple comorbidities. There are limited treatment options for UTUC, with the most common being endoscopic surgery or nephroureterectomy (removal of the entire kidney and ureter). Treatment with endoscopic surgery can be associated with a high rate of recurrence and relapse.

About UroGen Pharma Ltd.

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel^® reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat LG-UTUC and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade non-muscle invasive bladder cancer are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.urogen.com to learn more or follow us on X (Twitter), @UroGenPharma.

APPROVED USE FOR JELMYTO

JELMYTO^® is a prescription medicine used to treat adults with a type of cancer of the lining of the upper urinary tract including the kidney called low-grade Upper Tract Urothelial Cancer (LG-UTUC).

IMPORTANT SAFETY INFORMATION

You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.

Before receiving JELMYTO, tell your healthcare provider about all your medical conditions, including if you:

• are pregnant or plan to become pregnant. JELMYTO can harm your unborn baby. You should not become pregnant during treatment with JELMYTO. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO. Females who are able to become pregnant: You should use effective birth control (contraception) during treatment with JELMYTO and for 6 months after the last dose. Males being treated with JELMYTO: If you have a female partner who is able to become pregnant, you should use effective birth control (contraception) during treatment with JELMYTO and for 3 months after the last dose.
• are breastfeeding or plan to breastfeed. It is not known if JELMYTO passes into your breast milk. Do not breastfeed during treatment with JELMYTO and for 1 week after the last dose.
• Tell your healthcare provider if you take water pills (diuretic).

How will I receive JELMYTO?

• Your healthcare provider will tell you to take a medicine called sodium bicarbonate before each JELMYTO treatment.
• You will receive your JELMYTO dose from your healthcare provider 1 time a week for 6 weeks. It is important that you receive all 6 doses of JELMYTO according to your healthcare provider’s instructions. If you miss any appointments, call your healthcare provider as soon as possible to reschedule your appointment. Your healthcare provider may recommend up to an additional 11 monthly doses.
• JELMYTO is given to your kidney through a tube called a catheter.
• During treatment with JELMYTO, your healthcare provider may tell you to take additional medicines or change how you take your current medicines.

After receiving JELMYTO:

• JELMYTO may cause your urine color to change to a violet to blue color. Avoid contact between your skin and urine for at least 6 hours.
• To urinate, males and females should sit on a toilet and flush the toilet several times after you use it. After going to the bathroom, wash your hands, your inner thighs, and genital area well with soap and water.
• Clothing that comes in contact with urine should be washed right away and washed separately from other clothing.

JELMYTO may cause serious side effects, including:

• Swelling and narrowing of the tube that carries urine from the kidney to the bladder (ureteric obstruction). If you develop swelling and narrowing, and to protect your kidney from damage, your healthcare provider may recommend the placement of a small plastic tube (stent) in the ureter to help the kidney drain. Tell your healthcare provider right away if you develop side pain or fever during treatment with JELMYTO.
• Bone marrow problems. JELMYTO can affect your bone marrow and can cause a decrease in your white blood cell, red blood cell, and platelet counts. Your healthcare provider will do blood tests prior to each treatment to check your blood cell counts during treatment with JELMYTO. Your healthcare provider may need to temporarily or permanently stop JELMYTO if you develop bone marrow problems during treatment with JELMYTO.
• The most common side effects of JELMYTO include: urinary tract infection, blood in your urine, side pain, nausea, trouble with urination, kidney problems, vomiting, tiredness, stomach (abdomen) pain.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1800FDA1088. You may also report side effects to UroGen Pharma at 1-855-987-6436.

Please see JELMYTO Full Prescribing Information, including the Patient Information, for additional information.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: future results or outcomes implied from the long-term follow up study data; the estimated patient population and demographics for UTUC; the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as “could,” “encourage,” “potential” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: prior results may not be indicative of results that may be observed in the future; potential safety and other complications from JELMYTO use in diverse UTUC patient types; the timing and success of clinical trials and potential safety and other complications thereof; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; the ability to obtain regulatory approval within the timeframe expected, or at all; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; RTGel technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 10, 2025, The events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.

Contacts

INVESTOR CONTACT:
Vincent Perrone

Senior Director, Investor Relations

vincent.perrone@urogen.com
609-460-3588 ext. 1093

MEDIA CONTACT:

Cindy Romano

Director, Corporate Communications

cindy.romano@urogen.com
609-460-3583 ext. 1083

New Patient-Reported Outcomes from UGN-102 Clinical Trials Show the Investigational Treatment Did Not Adversely Affect Functionality, Symptom Burden, and Quality of Life in Patients with LG-IR-NMIBC




New Patient-Reported Outcomes from UGN-102 Clinical Trials Show the Investigational Treatment Did Not Adversely Affect Functionality, Symptom Burden, and Quality of Life in Patients with LG-IR-NMIBC

• New analysis of patient-reported outcomes from the OPTIMA II, ATLAS and ENVISION studies of UGN-102 presented at the American Urological Association (AUA) 2025 Annual Meeting in Las Vegas, Nevada

PRINCETON, N.J.–(BUSINESS WIRE)–UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced patient-reported outcomes following treatment of patients with low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) that showed investigational drug UGN-102 (mitomycin) for intravesical solution achieved robust and durable complete response (CR) rates without negatively impacting quality of life. The data (Moderated Poster – MP15) were presented at the AUA 2025 Annual Meeting in Las Vegas, Nevada.

“Patient reported quality of life outcomes are critical in evaluating the usefulness of investigational drugs, particularly when delivered in the bladder which frequently causes urinary symptoms,” said Charles Peyton, M.D., Assistant Professor, University of Alabama, Department of Urology, Heersink School of Medicine and study investigator. “Aggregated quality of life data across three robust clinical trials suggests that UGN-102 is quite tolerable without negatively impacting symptom burden, patient function or quality of life compared to baseline. UGN-102, if approved, is a promising alternative intravesical treatment for patients with LG-IR-NMIBC.”

In the OPTIMA II, ATLAS, and ENVISION late-phase studies, most patients (≥91% in ATLAS, ≥94% in ENVISION) completed the questionnaires at baseline, three months, and 12 months or study end. Baseline scores indicated high levels of functioning and low symptom burden prior to treatment. UGN-102 did not cause sustained declines in functioning or symptom burden, and no measured domains or items exceeded the threshold for clinically significant worsening at three or 12 months, suggesting no negative impact on quality of life.

“The patient-reported outcomes data showed that UGN-102 did not adversely impact quality of life while achieving high response rates in LG-IR-NMIBC patients,” said Mark Schoenberg M.D., Chief Medical Officer, UroGen. “We’re pleased to hear directly from patients, as their experiences highlight the importance of advancing treatment options that have the potential to reduce the burden of this challenging disease. These findings reinforce our commitment to developing solutions that make a meaningful difference in patients’ lives.”

In the Phase 2b OPTIMA II and Phase 3 ENVISION studies, patients received UGN-102, while in the Phase 3 ATLAS trial, patients were randomized to UGN-102 or trans-urethral resection of bladder tumor (TURBT); this analysis includes only data from UGN-102-treated patients. Each study featured a six-week UGN-102 treatment period, a CR assessment at three months, and follow-up ranging from nine to 63 months after three-month CR. Patient-reported symptoms and health status were evaluated using the EORTC-QLQ-NMIBC24 at baseline, three months, and 12 months or study end. Scores, ranging from one (not at all) to four (very much), were transformed to a 0-100 scale, with higher scores indicating greater symptom burden. Descriptive statistics were used to summarize baseline scores and changes, with positive changes reflecting symptom worsening.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the submission of the rolling new drug application (NDA) for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a Prescription Drug User Free Act (PDUFA) goal date of June 13, 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

LG-IR-NMIBC affects around 82,000 people in the U.S. every year, and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

About ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as a chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

About UroGen Pharma Ltd.

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with LG-IR-NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.UroGen.com to learn more or follow us on X (Twitter), @UroGenPharma.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: conclusions from the OPTIMA II, ATLAS and ENVISION data; the estimated patient population and demographics for LG-IR-NMIBC, and the significance of the unmet needs; the potential benefits to patients and opportunities for UGN-102, if approved; the potential for UGN-102 to deliver robust CR rates and duration of response in patients with LG-IR-NMIBC, without significant negative effects on symptom burden, patient function, or quality of life; statements related to UroGen’s NDA submission and the expected PDUFA goal date for UGN-102; the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as “anticipate,” “indicate,” “potential,” “promise,” “suggest,” or other words that convey uncertainty of future events or outcomes are used to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: preliminary results may not be indicative of results that may be observed in the future; the timing and success of clinical trials and potential safety and other complications thereof; the types, severity and treatability of TEAs that occur during clinical trials; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; even though the NDA for UGN-102 has been accepted for filing by the FDA, there is no guarantee that such NDA will be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the PDUFA goal date may be delayed due to various factors outside UroGen’s control; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; the ability to obtain and maintain regulatory approval; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology; and UroGen’s RTGel technology may not perform as expected. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Annual Report on Form 10-K for the year ending December 31, 2024, filed with the SEC on March 10, 2025. The events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.

Contacts

INVESTOR CONTACT:
Vincent Perrone

Senior Director, Investor Relations

vincent.perrone@urogen.com
609-460-3588 ext. 1093

MEDIA CONTACT:
Cindy Romano

Director, Communications

cindy.romano@urogen.com
609-460-3583 ext. 1083

New Long-Term Follow-Up Data from OPTIMA II Study of UGN-102 Demonstrates Median Duration of Response of Two Years in Patients with LG-IR-NMIBC




New Long-Term Follow-Up Data from OPTIMA II Study of UGN-102 Demonstrates Median Duration of Response of Two Years in Patients with LG-IR-NMIBC

• Data Presented at the American Urological Association 2025 Annual Meeting in Las Vegas, Nevada

PRINCETON, N.J.–(BUSINESS WIRE)–UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced new data from the OPTIMA II Phase 2b study of UGN‑102 (mitomycin) for intravesical solution demonstrate clinically meaningful two-year duration of response (24.2 months) by Kaplan-Meier analysis. UGN-102 is UroGen’s sustained-release formulation of mitomycin being developed for the treatment of recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

“The median duration of response of two years highlights UGN-102’s durability, even in patients with recurrent disease and multiple prior TURBT procedures,” said Neal Shore, MD, Medical Director, Carolina Urologic Research Center, Myrtle Beach, South Carolina. “These long-term data provide encouraging evidence of UGN-102’s sustained impact.”

The majority of patients included in OPTIMA II had recurrent disease at baseline (77.8%), with multiple prior transurethral resection of bladder tumor (TURBT) procedures. Among the 41 patients achieving a complete response (CR) at three months, 25 remained in CR at 12 months, and 17 of these patients entered long-term follow-up. The median Kaplan–Meier estimate of duration of response for the 41 patients that achieved CR was 24.2 months (95% CI 9.72, 47.18), with a median follow-up time of 33.6 months (95% CI 10.78, 42.94). Twenty patients (48.8%) experienced recurrence of low-grade disease. One patient progressed to high-grade disease and one patient died due to a cardiac disorder. Five patients remained disease-free at the time of the four-year data analysis.

“As the burden of LG-IR-NMIBC persists, the need for long-lasting treatment options becomes increasingly urgent,” said Mark Schoenberg, Chief Medical Officer, UroGen. “The growing body of evidence supporting UGN-102 underscores its potential to address this unmet need. These results emphasize UGN-102’s potential to deliver meaningful and sustained responses, offering hope to patients who have long struggled with recurrence and limited treatment options.”

UroGen completed the submission of the UGN-102 rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 with a Prescription Drug User Free Act (PDUFA) goal date of June 13, 2025.

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The TEAEs were typically mild-to-moderate in severity and either resolved or were resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN‑102.

About UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the submission of the rolling NDA for UGN-102 in August 2024, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include TURBT as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures.

About OPTIMA II

OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) was an open-label, single-arm, multi-center Phase 2b clinical trial of investigational drug UGN-102 to evaluate its safety and efficacy in patients with LG-IR-NMIBC.

Learn more about the Phase 2b OPTIMA II trial at www.clinicaltrials.gov (NCT03558503).

About UroGen Pharma Ltd.

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with LG-IR-NMIBC are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.UroGen.com to learn more or follow us on X (Twitter), @UroGenPharma.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the durability potential for UGN-102 and the potential to benefit patients and address unmet needs; statements related to UroGen’s NDA submission and the expected PDUFA goal date for UGN-102; the estimated patient population and demographics for NMIBC; the potential of UroGen’s proprietary RTGel technology to improve therapeutic profiles of existing drugs; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. Words such as “expect” and “potential,” or other words that convey uncertainty of future events or outcomes are used to identify these forward-looking statements. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: initial results may not be indicative of results that may be observed in the future; the timing and success of clinical trials and potential safety and other complications thereof; unforeseen delays that may impact the timing of progressing clinical trials and reporting data; even though the NDA for UGN-102 has been accepted for filing by the FDA, there is no guarantee that such NDA will be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the PDUFA goal date may be delayed due to various factors outside UroGen’s control; the ability to obtain and maintain adequate intellectual property rights and adequately protect and enforce such rights; the ability to obtain and maintain regulatory approval; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; UroGen may not successfully develop and receive regulatory approval of any other product that incorporates RTGel technology; and UroGen’s RTGel technology may not perform as expected. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Annual Report on Form 10-K for the year ending December 31, 2024, filed with the SEC on March 10, 2025. The events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release.

Contacts

INVESTOR CONTACT:
Vincent Perrone

Senior Director, Investor Relations

vincent.perrone@urogen.com
609-460-3588 ext. 1093

MEDIA CONTACT:
Cindy Romano

Director, Communications

cindy.romano@urogen.com
609-460-3583 ext. 1083