StatLab Activates New U.S. Manufacturing Capabilities to Secure Supply for Domestic Pathology Labs




StatLab Activates New U.S. Manufacturing Capabilities to Secure Supply for Domestic Pathology Labs

Strategic investments reinforce supply chain resilience and streamline U.S. production of essential diagnostic supplies.

MCKINNEY, Texas–(BUSINESS WIRE)–StatLab Medical Products (“StatLab”), a leading global innovator of pathology supplies, today announced the launch of new U.S.-based manufacturing capabilities, advancing its multi-year “in-region, for-region” strategy to ensure reliable, scalable production of critical reagents for pathology labs nationwide. The latest milestone—insourcing injection-molded prefill containers—further strengthens StatLab’s position as one of the top U.S. manufacturers of formalin prefills, producing nearly 100 million units annually, while increasing supply chain reliability for labs amid ongoing global uncertainty.

StatLab has a long history of manufacturing StatClick™ formalin prefills, but by becoming the first in its industry to vertically integrate container and lid production in-house, it is increasing control and reducing supply chain risk for this essential product used in operating rooms and labs every day across the U.S. In addition to bringing injection molding for prefill containers in-house, StatLab will also begin blow molding gallon bottles in Q2. Production capacity for cassettes continues to grow as well, with new injection molding cells added at the Arlington facility to meet strong U.S. demand.

“At StatLab, it is in our DNA to invest ahead of the need,” said Sung-Dae Hong, CEO of StatLab. “As one of the market leaders for essential pathology consumables, insourcing key containers for the products we make, including gallon bottles and prefills, empowers us to control quality and protect our customers against external market forces.”

In another proactive move to support customers through challenging market conditions, StatLab has announced it will currently absorb any tariff-related fees associated with products manufactured in its European facilities when selling directly to U.S. laboratories. These products include PiSmart cassette and slide printers, KT microscope slides, and KT coverglass. By leveraging its U.S. and European manufacturing capabilities, StatLab is able to offer consumables for the full pathology workflow protected from tariff impact, empowering domestic customers to focus more on patient care.

“Delivering stability for our customers, especially in times of volatility, is part of the trust we build every day,” continued Hong. “We recognize the importance of predictable pricing and reliable access to the essential supplies labs need for accurate diagnoses.”

About StatLab Medical Products

StatLab Medical Products has been dedicated to helping anatomic pathology laboratories provide the best possible patient care since 1976. Today, with the strength of our united brands and locations around the globe, we’re innovating pathology essentials, together. We offer an extensive portfolio of self-manufactured consumables and pathology equipment, developed and produced across nine manufacturing sites in the United States, United Kingdom, and Europe. Our global operational footprint — powered by over 750 mission-driven colleagues — enables an “in-region, for-region” manufacturing strategy, delivering a resilient, dependable supply chain and high-quality solutions. With a customer-centric approach at our core, we bring reliability, innovation, and quality to every interaction. Learn more at StatLab.com.

Contacts

Jessica Baer

jbaer@statlab.com
630-346-1659

IFF Completes Divestiture of Pharma Solutions Business Unit




IFF Completes Divestiture of Pharma Solutions Business Unit

NEW YORK–(BUSINESS WIRE)–IFF (NYSE: IFF) today announced that it has successfully completed the previously announced divestiture of its Pharma Solutions business unit to Roquette.

“The completion of our Pharma Solutions divestiture represents a significant milestone for IFF as we delivered our targeted net debt to credit-adjusted EBITDA of below 3.0x,” said Erik Fyrwald, IFF CEO. “This is an important step as it allows us to focus on our core strategy – capitalizing on the exciting growth opportunities within our key businesses – as we maximize long-term value for our shareholders. We’d like to thank our Pharma Solutions colleagues for their unwavering commitment and their dedication to exceptional customer service. We wish them continued success as they find a new home with Roquette.”

Welcome to IFF

At IFF (NYSE: IFF), we make joy through science, creativity and heart. As the global leader in flavors, fragrances, food ingredients, health and biosciences, we deliver groundbreaking, sustainable innovations that elevate everyday products—advancing wellness, delighting the senses and enhancing the human experience. Learn more at iff.com, LinkedIn, Instagram and Facebook.

© 2025 by International Flavors & Fragrances Inc. IFF is a Registered Trademark. All Rights Reserved.

Contacts

Media Relations:

Paulina Heinkel

332.877.5339

Media.request@iff.com

Investor Relations:

Michael Bender

212.708.7263

Investor.Relations@iff.com

BIOQUAL Announces the retirement of Dr. Mark G. Lewis as President and CEO of the company




BIOQUAL Announces the retirement of Dr. Mark G. Lewis as President and CEO of the company

The Company Also Announces the Appointment of Dr. Hanne Andersen Elyard, BIOQUAL’s President and Chief Science Officer

ROCKVILLE, Md.–(BUSINESS WIRE)–BIOQUAL, Inc. www.bioqual.com BIOQ(Pinksheets):

Dr. Mark Lewis will be retiring as President of BIOQUAL effective May 31, 2025, and is succeeded by Dr. Hanne Andersen Elyard, BIOQUAL’s Chief Science Officer. Dr. Lewis will remain as Chairman of the Board of Directors. Dr. Lewis joined BIOQUAL as Senior Scientist in August 2003. He became the Executive Vice President in October 2008 and served in that capacity until he became President and CEO in 2010.

“The entire BIOQUAL team is excited and honored to have Hanne assume the role of President of BIOQUAL and to continue driving and growing our contract research program,” shared Mark Lewis, President and Chief Executive Officer of BIOQUAL. “Working alongside Hanne for over eighteen years, I remain impressed by her expertise in client contract and business operations capabilities and am confident that through her leadership our company will continue its successful operations.”

Hanne Andersen Elyard received her PhD in microbiology from the Louisiana State University Health Sciences Center in 2000. She was a postdoctoral fellow in the AIDS Vaccine Program, SAIC-Frederick, Inc. from 2001 until 2005. In January 2006 she joined BIOQUAL as a staff scientist, was promoted to Research Director in 2010, promoted to Vice President of Science in 2018 and subsequently she was promoted to Chief Scientific Officer in 2022. She has authored or co-authored more than 105 peer-reviewed scientific publications.

Forward Looking Information

Statements herein that are not descriptions of historical facts are forward-looking and subject to risks and uncertainties. The forward-looking statements are neither promises nor guarantees, and one should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond the Company’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including risks relating to the ability to continue to extend current government contracts; the Company’s ability to obtain new government or commercial contracts; continued demand for the use of animal models in scientific research; the Company’s ability to obtain sufficient numbers of animal models; the availability of adequate numbers of employees; the Company’s ability to perform under its contracts in accordance with the requirements of the contracts; the actual costs incurred in performing the Company’s contracts and its ability to manage its costs, including its capital expenditures; dependence on third parties; future capital needs; the ability to fund its capital needs through the use of its cash on hand and line of credit; and the future availability and cost of financing/capital sources to the Company.

Contacts

Mark G. Lewis, Ph.D., CEO (240-404-7654)

Scholar Rock Appoints David L. Hallal as Chief Executive Officer; Also Announces Addition of Three Key Leaders to Scale for Next Phase of Growth




Scholar Rock Appoints David L. Hallal as Chief Executive Officer; Also Announces Addition of Three Key Leaders to Scale for Next Phase of Growth

• David Hallal has served as Chairman of the Board of Directors since 2017; ensures seamless transition ahead of global launch of apitegromab for Spinal Muscular Atrophy
• Jay Backstrom, M.D., to serve as strategic advisor as part of planned transition, continuing to work closely with executive team and Board of Directors
• Akshay Vaishnaw, M.D., Ph.D., appointed to newly created role of President of R&D; has served as Board member since 2019
• R. Keith Woods appointed to newly created role of Chief Operating Officer with focus on evolution to fully integrated global commercial operations
• Vikas Sinha appointed Chief Financial Officer
• Company to host Conference Call today at 8:30 AM ET

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases, announced today that David Hallal has been appointed Chief Executive Officer succeeding Jay Backstrom, M.D. David Hallal has served as Chairman of the Board at Scholar Rock since 2017 and just prior to that spent more than a decade as CEO, COO, and CCO of Alexion building and leading the company’s 50-country operating platform. As part of this planned transition, Dr. Backstrom will serve as a strategic advisor, working closely with the company’s executive team and Board of Directors.

Scholar Rock also announced today the addition of three leaders with unparalleled experience and success in building global organizations focused on developing and commercializing life-changing therapies.

• Akshay Vaishnaw, M.D., Ph.D., has been named President of R&D; he was formerly President of Alnylam.
• R. Keith Woods has been appointed Chief Operating Officer and will focus on evolving the organization into a fully integrated global enterprise; he was formerly COO of argenx.
• Vikas Sinha is joining as Chief Financial Officer; he was formerly CFO of Alexion and ElevateBio.

“On behalf of the Board, we are deeply grateful to Jay for his leadership over the past three years – driving us through the highly successful apitegromab global Phase 3 program and now into the US and European regulatory processes for marketing authorization,” said Mr. Hallal. “As Board Chair, I have been continuously collaborating with Jay to accelerate Scholar Rock’s evolution into a global biotech leader. These efforts will be amplified by the immediate addition of Akshay, Keith and Vikas – three operators that have built and led some of the most successful biotech companies in our industry. Together, we are committed to realizing Scholar Rock’s global ambitions to bring apitegromab to SMA patients around the world.”

“It has been an honor to lead the incredible team at Scholar Rock over the past several years. I am very proud of all we have achieved – from advancing apitegromab through a successful, pivotal Phase 3 study and on the path to becoming a commercial company,” said Dr. Backstrom. “With the BLA now accepted under priority review and the anticipated launch later this year, this is the right moment for our leadership transition. I’m grateful for the close partnership over the years with David and Akshay as board members, and I look forward to continuing our collaboration on behalf of the SMA community to bring our highly innovative apitegromab, the world’s first muscle targeted treatment, to patients and families living with the impact of this severe and progressive disease.”

Conference Call Information

Management will discuss the new leadership appointments via conference call on April 28, 2025 at 8:30 am ET. To access the live conference call, participants may register here. The live audio webcast of the call will be available under “Events and Presentations” in the Investor Relations section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number along with a unique passcode and registrant ID that can be used to access the call. An archived replay of the webcast will be available on the Company’s website for approximately 90 days.

About Apitegromab

Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate in spinal muscular atrophy (SMA) to demonstrate clinical success in a pivotal phase 3 trial. Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Apitegromab has not been approved for any use by the FDA or any other regulatory agency.

About SMA

Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease that afflicts an estimated 30,000 to 35,000 people in the United States and Europe. The disease is characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk, and progressive muscle weakness. While there has been progress in the development of therapeutics that address the loss of motor neurons, there continues to be a high unmet need for therapies that directly address the progressive muscle weakness that leads to loss of motor function in SMA.

About Scholar Rock

Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily. The company is named for the visual resemblance of a scholar rock to protein structures. Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role.

This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn.

Availability of Other Information About Scholar Rock

Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X (formerly known as Twitter) or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

Scholar Rock^® is a registered trademark of Scholar Rock, Inc.

Forward-Looking Statements

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 regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, the timing and results of regulatory submissions, the therapeutic potential of apitegromab, its transition to a fully integrated global commercial enterprise and launch of apitegromab and the anticipated impact of the management transition described herein. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, whether the results from the Phase 3 SAPPHIRE trial will be sufficient to support regulatory approval, that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; and Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and our ability to continue as a going concern; as well as those risks more fully discussed in the section entitled “Risk Factors” in Scholar Rock’s Annual Report on Form 10-K for the year ended December 31, 2024, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.

Contacts

Rushmie Nofsinger

Scholar Rock

ir@scholarrock.com
media@scholarrock.com
857-259-5573

Cynthia Clayton

Clayton Bio Communications

cynthia@claytonbiocomms.com
617-852-7735

Compass Pathways to Announce First Quarter Financial Results on May 8, 2025




Compass Pathways to Announce First Quarter Financial Results on May 8, 2025

Compass management will host a conference call at 8:00 am ET (1:00 pm UK)

LONDON & NEW YORK–(BUSINESS WIRE)–$CMPS #Biotech–Compass Pathways plc (Nasdaq: CMPS), a biotechnology company dedicated to accelerating patient access to evidence-based innovation in mental health, announced today that it will release financial results for the first quarter ended March 31, 2025, and provide an update on recent developments, on May 8, 2025.

Compass management will host a conference call at 8:00 am ET (1:00 pm UK) on May 8, 2025.

A live webcast of the call will be available on the Compass Pathways website at: First Quarter 2025 Financial Results. The webcast will be archived for 30 days.

About Compass Pathways

Compass Pathways plc (Nasdaq: CMPS) is a biotechnology company dedicated to accelerating patient access to evidence-based innovation in mental health. We are motivated by the need to find better ways to help and empower people with serious mental health conditions who are not helped by existing treatments. We are pioneering a new paradigm for treating mental health conditions focused on rapid and durable responses through the development of our investigational COMP360 synthesized psilocybin treatment, potentially a first in class treatment. COMP360 has Breakthrough Therapy designation from the US Food and Drug Administration (FDA) and has received Innovative Licensing and Access Pathway (ILAP) designation in the UK for treatment-resistant depression (TRD).

Compass is headquartered in London, UK, with offices in New York and San Francisco in the US. We envision a world where mental health means not just the absence of illness but the ability to thrive.

Contacts

Enquiries
Media: Dana Sultan-Rothman, media@compasspathways.com, +1 484 432 0041

Investors: Stephen Schultz, stephen.schultz@compasspathways.com, +1 401 290 7324

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

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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