Latest news with #EMDSerono
Yahoo
2 days ago
- Business
- Yahoo
Merck KGaA ventures into new territory in the US
This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. Merck KGaA has been making new moves to propel itself into becoming what CEO Belén Garijo described as a 'globally diversified science and technology powerhouse.' Most recently, the company closed its $3.4 billion acquisition of Pfizer spinout SpringWorks Therapeutics, which will help it stake a claim in the rare tumor space. And in general, it's all about 'doubling down' on R&D and building the company's U.S. footprint, said Miguel Fernández Alcalde, president of EMD Serono, Merck KGaA's healthcare business in the U.S. and Canada. Germany-based Merck KGaA has undergone a number of transformations throughout its deep history. In 2022, the company restructured into three distinct business units including one focused on the life sciences sector and manufacturing services. But with the contract manufacturing blitz of the COVID-19 pandemic era fading, the company is refocusing. EMD Serono relocated its U.S. headquarters to Boston's Seaport district to place itself squarely in the thick of the region's most innovative 'biotechs, startups, academia and scientists,' Fernández Alcalde said. 'I want to make sure we are bringing the U.S. [business] to the next level in terms of contributions to the whole organization." Miguel Fernández Alcalde President, EMD Serono Merck KGaA also elevated its global head of R&D and chief medical officer of its healthcare business, Dr. Danny Bar-Zohar, to healthcare CEO. 'That tells you that the company's moving along in the direction of doubling down on R&D,' Fernández Alcalde said. Fernández Alcalde's appointment to president of EMD Serono in December is also part of the company's overall quest to build its U.S. footprint and bolster R&D through external deals. 'I want to make sure we are bringing the U.S. [business] to the next level in terms of contributions to the whole organization,' he said. 'I do think we have lots of opportunities in the U.S. ecosystem and U.S. market. My job and my vision and ambition is to really untap all those things that we have ahead of us.' A pharmacist by trade, Fernández Alcalde has been with Merck KGaA since 2014, serving most recently as EMD Serono's chief operating officer. 'That gave me a lot of knowledge and understanding of the business of the company in the States and the teams, the dynamics, the culture, but also the U.S. [healthcare] ecosystem,' he said. Driving dealmaking Central to Fernández Alcalde's role is supplementing the company's current pipeline through external innovation investments, including more strategic in-licensing and co-development opportunities. The SpringWorks acquisition put the company on track to generate at least 50% of its future launch assets from external companies, compared to roughly 10%-15% just 18 months ago. 'It's easily a fivefold increase in our ambition from external innovation,' Fernández Alcalde said. In terms of what kinds of deals they're targeting, Fernández Alcalde said several factors are at play. 'We are not looking [for] the $40 billion type of acquisition,' he said. Nor will they chase first-in-class potential blockbusters with never-explored mechanisms of action. Instead, they'll target smaller deals with the 'right risk balance' for their pipeline. He pointed to the SpringWorks acquisition as an example, which not only adds two FDA-approved drugs to its portfolio, but also a 'runway of three to five years of nice growth' in the form of several clinical-phase pipeline candidates. 'These areas where we have deep, good science [and a] validated proof of concept or an about-to-be-validated proof of concept where the mechanism of action is already validated, is something we are interested in,' he said. The company will take a similar approach when it comes to therapeutic areas by branching out within reason. 'We are not sticking to the [therapeutic areas] we have today, but we are not going to go wild, either,' he said. Rather, they'll look for specialties that are 'adjacent' and 'logical' in terms of what they've been historically known for, such as oncology and neurology/immunology. Again, he pointed to the SpringWorks acquisition as an example and noted that some might ask what track record EMD Serono or Merck KGaA has in rare tumors. 'The answer is zero. But we do know how to commercialize,' he said. Plus, 'it's in an adjacent area of oncology.' Fernández Alcalde is also thinking globally, intending to follow good science wherever it leads and consider deals from around the world. 'We couldn't care less about where that science is coming from, whether it's China, my home country Spain, the U.S. or South Africa,' he said. 'If we think that science will help patients … we will definitely think about it.' Recommended Reading Let's make a deal? Big Pharma execs express varying views on their M&A future
Yahoo
12-06-2025
- Health
- Yahoo
EMD Serono Presents Results on Efficacy and Safety of Enpatoran in Systemic Lupus Erythematosus (SLE) at EULAR 2025
Cohort B of the Phase 2 study indicates all doses of enpatoran were associated with higher BICLA response rates compared with placebo, though the primary endpoint of BICLA dose-response relationship at Week 24 was not met Data show encouraging efficacy in a large subgroup of SLE patients with active cutaneous manifestations at baseline, including improved response rates in key disease activity measurements There is a substantial unmet treatment need for the estimated 70-80% of SLE patients who experience active cutaneous manifestations, which can profoundly impact quality of life1 BOSTON, June 12, 2025--(BUSINESS WIRE)--Not intended for media outside the U.S. or Canada EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, a leading science and technology company, today announced the presentation of detailed results from Cohort B of the global Phase 2 WILLOW study (NCT05162586) evaluating enpatoran, an investigational, oral, novel TLR7/8 inhibitor in systemic lupus erythematosus (SLE). Although it did not meet the primary endpoint of dose-response relationship, when compared to placebo, enpatoran demonstrated improvements in measures of both systemic and cutaneous disease activity in prespecified SLE subpopulations despite standard of care (SoC), including those with active cutaneous manifestations at baseline [(CLASI-A) ≥8], and was overall well tolerated. These findings will be presented in a late-breaking oral presentation at the 2025 European Congress of Rheumatology (EULAR) in Barcelona (Abstract # LB0004). "Analyses of Cohort B contribute to our understanding of enpatoran's potential to address the critical unmet needs for patients living with lupus, including those experiencing significant skin manifestations. These manifestations are often part of the systemic activity or flare, which can be painful and have a considerable impact on quality of life," said principal investigator Prof. Eric Morand, from Monash University and Monash Health. "The improvements observed in key disease measures represent a meaningful advancement in our ongoing investigation of the TLR7/8 inhibition approach for patients insufficiently managed by current therapies." WILLOW is a global, multicenter, randomized, placebo-controlled Phase 2 study evaluating three doses of oral enpatoran taken twice daily (25 mg, 50 mg and 100 mg) versus placebo plus SoC over 24 weeks. The study features a unique design across two lupus cohorts, including both patients with active SLE and cutaneous lupus erythematosus (CLE). Cohort B of the study was designed to evaluate the dose-response relationship of enpatoran in reducing disease activity, based on the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) response rate at Week 24 and enrolled SLE patients who had moderate or severe active disease despite SoC. Cohort B showed positive results in secondary and exploratory endpoints and within prespecified patient subpopulations. In patients with active skin disease (CLASI-A ≥8), BICLA response rates were up to 58.6% while placebo response rates were 31.7%, and up to 60.5% of patients receiving enpatoran showed a CLASI-70 response, compared with 26.8% for placebo, at Week 24. In addition, the subgroups of patients with high corticosteroid (prednisone-equivalent ≥10 mg/day) use and those with high interferon gene signature (IFN-GS) at baseline also showed higher and relevant BICLA response rates for enpatoran compared to placebo. As presented earlier this year at LUPUS 2025, Cohort A analyses from the WILLOW study showed clinically meaningful improvement in disease activity in patients with CLE and mild SLE with active lupus rash at Weeks 16 and 24. Overall, for skin-related signs and symptoms, comparable improvements were observed in Cohort B relative to Cohort A, reinforcing the potential efficacy of enpatoran in patients with cutaneous manifestations of lupus erythematosus with or without systemic disease. "The efficacy and tolerability results from Cohort B, particularly among those with active skin involvement—a manifestation that affects most lupus patients—are consistent with our observations from Cohort A. The lupus rash is not only a visible symptom but is also closely linked to the underlying systemic activity of lupus," said Jan Klatt, Head of Development Unit Neurology & Immunology for the Healthcare business of Merck KGaA, Darmstadt, Germany. "We are set to initiate regulatory discussions with key health authorities to determine the most effective pathway for bringing enpatoran to patients." Enpatoran was well-tolerated and exhibited a manageable safety profile consistent with previous studies, with no new safety signals identified. Rates of treatment-emergent adverse events (TEAEs) were comparable between all enpatoran arms and placebo, ranging from 60.6% to 64.2%, and the most frequently reported TEAEs were infections and infestations. These results further support the anticipated favorable safety profile of enpatoran. About Enpatoran Enpatoran is a selective Toll-like receptor (TLR)7/8 inhibitor under investigation for the treatment of systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). By inhibiting TLR7/8 activation, enpatoran may help reduce pro-inflammatory cytokines and autoantibody production, potentially addressing underlying mechanisms of chronic inflammation and disease progression in lupus. With its novel proposed mechanism of action and oral administration, enpatoran has the potential to be a first-in-class treatment for patients across lupus conditions. Enpatoran is currently under clinical investigation and is not approved for any use anywhere in the world. About the Phase 2 WILLOW Clinical Study WILLOW (NCT05162586) is a randomized, double-blind, placebo-controlled Phase 2 proof of concept and dose-finding study designed to evaluate the efficacy and safety of enpatoran in patients with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). The study incorporates a basket design, including two cohorts – Cohort A including patients with CLE or SLE with active lupus rash and Cohort B including patients with active SLE. The WILLOW study aims to advance the understanding of enpatoran's therapeutic potential and to help address significant unmet needs in lupus treatment. About Lupus Erythematosus Lupus erythematosus is a chronic autoimmune disease that can affect various parts of the body, including the skin, joints, kidneys and other organs. It occurs when the immune system mistakenly attacks healthy tissues, leading to inflammation, pain and potential organ damage. There are multiple types of lupus, with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE) being two primary forms. Symptoms can range from mild to life-threatening, often including fatigue, joint pain, rashes and organ involvement. Lupus disproportionately impacts women and people of color, and despite available treatments, many patients experience unmet needs due to limited efficacy or side effects. EMD Serono in Neurology and Immunology EMD Serono has a long-standing legacy in neurology and immunology, with significant R&D and commercial experience in multiple sclerosis (MS). The company's current MS portfolio includes two products for the treatment of relapsing MS – Rebif® (interferon beta-1a) and MAVENCLAD® (cladribine) tablets. EMD Serono aims to improve the lives of patients by addressing areas of unmet medical needs. In addition to EMD Serono's commitment to MS, the company also has a pipeline focusing on discovering new therapies that have potential in other neuroinflammatory and immune-mediated diseases, including systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE) and generalized myasthenia gravis (gMG). About EMD Serono, Inc. EMD Serono - the healthcare business of Merck KGaA, Darmstadt, Germany in the U.S. and Canada - aspires to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer. The business is imagining the future of healthcare by working to translate the discovery of molecules into potentially meaningful outcomes for people with serious unmet medical needs. EMD Serono's global roots go back more than 350 years with Merck KGaA, Darmstadt, Germany. Today, the business has approximately 1,050 employees around the country with commercial, clinical and research operations in Massachusetts. 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. 1 Cojocaru M, Cojocaru IM, Silosi I, Vrabie CD. Manifestations of systemic lupus erythematosus. Maedica (Bucur). 2011 Oct;6(4):330-6. PMID: 22879850; PMCID: PMC3391953. View source version on Contacts Media Relations Phone: +1 781 427 1892 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Medscape
03-06-2025
- Business
- Medscape
Enpatoran Shows Promise in Treating Lupus Rash
An oral therapy targeting toll-like receptors (TLR) 7 and 8 reduced disease activity in patients with cutaneous lupus erythematosus (CLE) or systemic lupus erythematosus (SLE) with active lupus rash. METHODOLOGY: Cohort A of the phase 2 WILLOW study included patients with CLE and patients with mild SLE and active skin manifestation for whom no approved treatments currently exist. In the study of the oral, small molecule TLR 7/8 inhibitor enpatoran, researchers randomly assigned 100 patients, all receiving standard-of-care medications, to placebo, 25 mg enpatoran, 50 mg enpatoran, or 100 mg enpatoran twice daily for 24 weeks. The primary endpoint was percent change in CL Disease Area and Severity Index Activity (CLASI-A) from baseline to week 16. TAKEAWAY: Patients taking enpatoran all had clinically meaningful reductions to CLASI-A at week 16, with reductions of 74.6% for the group taking 25 mg twice daily, 59.8% for 50 mg twice daily, 68.5% for 100 mg twice daily, and 41.2% for placebo. A total of 87% of patients taking 25 mg twice daily achieved ≥ 50% reduction in CLASI-A by week 24 compared with 72% for 50 mg twice daily, 73.1% for 100 mg twice daily, and 30.8% for placebo. All patients on enpatoran also had reduced interferon gene signatures by week 2 compared with placebo, and this effect was continued through week 24. Enpatoran was well-tolerated with no new safety signals from previous clinical studies. Treatment-emergent adverse events occurred in 46% with placebo, and at rates of 57.7%-80.8% in enpatoran treatment groups. IN PRACTICE: 'The new findings from WILLOW provide promising evidence that enpatoran could enhance treatment options for these patients, addressing the suboptimal care typically available for those with CLE- and SLE-related skin manifestations,' said a spokesperson for EMD Serono, the healthcare business of Merck KGaA in the United States. SOURCE: Eric Morand, MD, of Monash University, Melbourne, Australia, presented the study at the 16th International Congress on Systemic Lupus Erythematosus in Toronto, Ontario, Canada. LIMITATIONS: The relatively small sample size of the trial and short duration could limit the ability to detect clinically meaningful differences in efficacy and longer-term outcomes. DISCLOSURES: Merck KGaA, based in Darmstadt, Germany, funded the research. Presenter and principal investigator Eric Morand, MD, of Monash University in Melbourne, Australia, reported financial relationships with 17 pharmaceutical companies, several of which manufacture lupus drugs.
Yahoo
28-05-2025
- Business
- Yahoo
Pimicotinib Demonstrates Best-in-Class Potential with Significant Efficacy and Clinically Meaningful Improvements in Patients with Tenosynovial Giant Cell Tumor
Global Phase 3 MANEUVER study of pimicotinib in tenosynovial giant cell tumor (TGCT) met the primary endpoint, demonstrating objective response rate at week 25 of 54.0% versus 3.2% for placebo (p<0.0001) MANEUVER met all five key secondary endpoints, with statistically significant and clinically meaningful improvements in pain, stiffness, range of motion, physical function, and decrease in tumor volume Treatment was well-tolerated, with low incidence of treatment discontinuation and dose reductions Not intended for media outside the U.S. or Canada BOSTON, May 28, 2025--(BUSINESS WIRE)--EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, today announced the presentation of detailed positive results from Part 1 of the global Phase 3 MANEUVER trial evaluating pimicotinib, a potentially best-in-class investigational colony stimulating factor-1 receptor (CSF-1R) inhibitor in development by Abbisko Therapeutics Co., Ltd., in the treatment of patients with tenosynovial giant cell tumor (TGCT). Once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) compared with placebo at week 25 (54.0% vs. 3.2% for placebo (p<0.0001). The study also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT. These findings will be presented Sunday, June 1 in an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract #11500). "The impact that TGCT has on patients goes far beyond the physical presence of the tumor. It affects their ability to work, to move freely, and to engage in everyday activities," said Prof. Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital. "In MANEUVER, we observed the highest ORR seen to date with a systemic therapy, together with statistically significant improvements in measures of pain, stiffness, and range of motion. These improvements in outcomes that matter to patients with TGCT and the physicians who care for them show the potential of pimicotinib to allow patients to go about their daily lives with fewer negative effects of their disease." In MANEUVER, which enrolled patients from China, Europe and North America, the effect of pimicotinib had an early onset, with 41.3 % (26 of 63) of patients experiencing objective response to therapy after 13 weeks. By the data cutoff for primary analysis, nearly all patients in the pimicotinib group (58 of 63 patients; 92.1%) had a decrease in tumor size per BIRC based on RECIST v1.1; one patient achieved a complete response and 33 patients achieved a partial response. The median duration of response was not reached by the data cutoff. The analysis of tumor volume score (TVS, an endpoint designed specifically for TGCT) showed that nearly two-thirds of patients treated with pimicotinib experienced a reduction in tumor volume of at least 50% (61.9% vs. 3.2% for placebo, p<0.0001). Pimicotinib also demonstrated statistically significant and clinically meaningful improvement across all additional secondary endpoints relevant to patients' daily lives, and these improvements were seen regardless of achieving objective tumor response to pimicotinib. Pimicotinib improved active range of motion (p=0.0003) and physical function measured by PROMIS-PF scale (p=0.0074). Pimicotinib also reduced worst stiffness (p<0.0001) and worst pain (p<0.0001). "TGCT, although rare, has a significant impact on the daily lives of the primarily working-age adults who live with the disease, due to swelling, pain, stiffness, and limited mobility caused by the growth of these tumors in and around the joints," said Danny Bar-Zohar, appointed CEO Healthcare and current Global Head of R&D and Chief Medical Officer. "The landmark global Phase 3 MANEUVER study data will help redefine how TGCT is treated, and we plan regulatory submissions to start this year." Pimicotinib was well-tolerated, and the safety profile was consistent with previously reported data, with no evidence of cholestatic hepatotoxicity or hair/skin hypopigmentation. Treatment-emergent adverse events (TEAEs) leading to treatment discontinuation occurred in one patient (1.6%) treated with pimicotinib; TEAEs leading to dose reduction occurred in 7.9% (n=5) of pimicotinib-treated patients. About MANEUVER The pivotal Phase 3 MANEUVER study is a three-part, randomized, double-blind, placebo-controlled study to assess the efficacy and safety of pimicotinib in patients with TGCT who require systemic therapy and have not received prior anti-CSF-1/CSF-1R therapy. The study is being conducted by Abbisko Therapeutics in China (n=45), Europe (n=28), and the US and Canada (n=21). In the double-blind Part 1, 94 patients were randomized 2:1 to receive either 50 mg QD of pimicotinib (n=63) or placebo (n=31) for 24 weeks. The primary endpoint is objective response rate (ORR) at week 25, as measured by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 by blinded independent central review in the intent-to-treat (ITT) population. Secondary endpoints include tumor volume score, active range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function measured by Patient-Reported Outcomes Measurement Information System (PROMIS). After the double-blind Part 1, eligible patients could continue to the open-label Part 2 for up to 24 weeks of dosing, results of which are expected in mid-2025. Patients who complete Part 2 may then enter the open-label extension phase (Part 3) for extended treatment and safety follow-up. About Pimicotinib (ABSK021) Pimicotinib (ABSK021), which is being developed by Abbisko Therapeutics, is a novel, orally administered, highly selective and potent small-molecule inhibitor of CSF-1R. Pimicotinib was recently granted Priority Review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of patients with tenosynovial giant cell tumor (TGCT) who require systemic therapy. Pimicotinib has been granted breakthrough therapy designation (BTD) for the treatment of unresectable TGCT by China National Medical Products Administration (NMPA) and the US Food and Drug Administration (FDA), and priority medicine (PRIME) designation from the European Medicines Agency (EMA). Merck KGaA, Darmstadt, Germany, holds worldwide commercialization rights for pimicotinib. Advancing the Future of Cancer Care At EMD Serono, we strive every day to improve the futures of people living with cancer. Building on our 350-year global heritage as pharma pioneers, we are focusing our most promising science to target cancer's deepest vulnerabilities, pursuing differentiated molecules to strike cancer at its core. By developing new therapies that can help advance cancer care, we are determined to create a world where more cancer patients will become cancer survivors. About EMD Serono, Inc. EMD Serono - the healthcare business of Merck KGaA, Darmstadt, Germany in the U.S. and Canada - aspires to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer. The business is imagining the future of healthcare by working to translate the discovery of molecules into potentially meaningful outcomes for people with serious unmet medical needs. EMD Serono's global roots go back more than 350 years with Merck KGaA, Darmstadt, Germany. Today, the business has approximately 1,050 employees around the country with commercial, clinical and research operations in Massachusetts. 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 to register for your online, change your selection or discontinue this service. View source version on Contacts Media Relations Phone: +49 151 1454 9591 Investor Relations Phone: +49 6151 72-3321
National Post
21-05-2025
- Health
- National Post
EMD Serono Presents Positive Phase 2 Data for Enpatoran Demonstrating Reduction in Disease Activity in Patients with Cutaneous Lupus Erythematosus (CLE) and Systemic Lupus Erythematosus (SLE) with Active Lupus Rash
Article content Cohort analyses from the WILLOW study reveal clear proof of concept in patients with CLE and SLE with active lupus rash, showing clinically meaningful improvement in disease activity at Week 16 Enpatoran is a potential first-in-class oral therapy for CLE and SLE that is thought to selectively block the activation of Toll-like receptors (TLR)7 and TLR8 Lupus rash can lead to physical discomfort and emotional distress, highlighting the need for effective treatments to manage its signs and symptoms Article content Article content EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, today announced positive data on enpatoran, an investigational, oral, novel TLR7/8 inhibitor, demonstrating clinically meaningful reduction in disease activity in patients with cutaneous lupus erythematosus (CLE) and systemic lupus erythematosus (SLE) with active lupus rash. The findings are from Cohort A of the Phase 2 WILLOW study ( NCT05162586). Results will be presented at the 16 th International Congress on Systemic Lupus Erythematosus (LUPUS 2025), taking place May 21-24 in Toronto. Article content WILLOW is a global, multicenter, randomized, placebo-controlled Phase 2 study evaluating three doses of enpatoran taken twice daily (25 mg, 50 mg and 100 mg) versus placebo plus standard of care (SoC) over 24 weeks. The study features a unique design across two lupus cohorts, including both patients with active SLE and CLE. Cohort A focused on patients with CLE or SLE with active lupus rash and evaluated organ-specific disease activity using the Cutaneous Lupus Erythematosus Disease Area and Severity Index Activity (CLASI-A) score, a well-defined endpoint in CLE studies that measures different aspects of mucocutaneous manifestations. Cohort B was designed to evaluate the effect of enpatoran on systemic disease activity of SLE patients with the BICLA response endpoint. Article content Cohort A met its primary endpoint, demonstrating a dose-response relationship and showing a clinically meaningful improvement in CLASI-A scores at Week 16 (p = 0.0002). Additionally, at Week 24 up to 91.3% of patients receiving enpatoran achieved a CLASI-50 response (≥50% improvement from baseline), and up to 60.9% achieved a CLASI-70 response (≥70% improvement), compared with 38.5% and 11.5%, respectively, in the placebo group. In this cohort, enpatoran was well-tolerated, and exhibited a manageable safety profile consistent with previous studies, with no new safety signals identified. Article content 'Lupus can make navigating everyday life difficult. The skin manifestation, known as lupus rash, often comes with persistent itching, which can lead to scarring and hair loss. This can significantly impact the physical, emotional and social well-being of those living with lupus, underscoring the urgent need for effective treatments,' said Jan Klatt, Head of Development Unit Neurology & Immunology for the Healthcare business of Merck KGaA, Darmstadt, Germany. 'We are encouraged by the WILLOW results, where we observed clinically meaningful efficacy with a favorable safety profile in people living with lupus rash. Based on these results, discussions with health authorities on a global Phase 3 program with enpatoran are underway.' Article content In addition, and confirming the biological activity, treatment with enpatoran in Cohort A also led to a rapid reduction in interferon gene signature scores beginning at Week 2, which was maintained to Week 24, confirming the involvement of the TLR7/8 pathway in interferon activation in CLE. Overall, evidence from the WILLOW study supports the continued development of enpatoran as a treatment for autoimmune diseases like lupus. Article content On Cohort B of the WILLOW study, promising efficacy results were observed in prespecified subpopulations, even though the primary endpoint of dose response was not met. The full readout from this cohort will be presented at the European Alliance of Associations for Rheumatology Congress (EULAR 2025). Article content Principal investigator Prof. Eric Morand of Monash University and Monash Health, said, 'These new findings offer promising evidence that, with enpatoran, we may be able to advance outcomes, which remain suboptimal for most patients. The data from the WILLOW study further our understanding of TLR7/8 inhibition in SLE and CLE, which is a novel mechanism of action that may offer new hope for patients.' Article content Toll-like receptors (TLR)7 and TLR8 play a relevant role in lupus pathogenesis and are associated with severe manifestations of the disease. By inhibiting these key disease drivers, enpatoran's unique proposed mechanism of action aims to enhance therapeutic efficacy while preserving the body's immune response, potentially overcoming limitations of existing lupus therapies. Article content About Enpatoran Article content Enpatoran is a selective Toll-like receptor (TLR)7/8 inhibitor under investigation for the treatment of systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). By inhibiting TLR7/8 activation, enpatoran may help reduce pro-inflammatory cytokines and autoantibody production, potentially addressing underlying mechanisms of chronic inflammation and disease progression in lupus. With its novel proposed mechanism of action and oral administration, enpatoran has the potential to be a first-in-class treatment for patients across lupus conditions. Enpatoran is currently under clinical investigation and is not approved for any use anywhere in the world. Article content WILLOW ( NCT05162586) is a randomized, double-blind, placebo-controlled Phase 2 proof of concept and dose-finding study designed to evaluate the efficacy and safety of enpatoran in patients with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE). The study incorporates a basket design, including two cohorts – Cohort A including patients with CLE or SLE with active lupus rash and Cohort B including patients with active SLE. The WILLOW study aims to advance the understanding of enpatoran's therapeutic potential and to help address significant unmet needs in lupus treatment. Article content About Lupus Erythematosus Article content Lupus erythematosus is a chronic autoimmune disease that can affect various parts of the body, including the skin, joints, kidneys and other organs. It occurs when the immune system mistakenly attacks healthy tissues, leading to inflammation, pain and potential organ damage. There are multiple types of lupus, with systemic lupus erythematosus (SLE) and cutaneous lupus erythematosus (CLE) being two primary forms. Symptoms can range from mild to life-threatening, often including fatigue, joint pain, rashes and organ involvement. Lupus disproportionately impacts women and people of color, and despite available treatments, many patients experience unmet needs due to limited efficacy or side effects. Article content EMD Serono in Neurology and Immunology Article content EMD Serono has a long-standing legacy in neurology and immunology, with significant R&D and commercial experience in multiple sclerosis (MS). The company's current MS portfolio includes two products for the treatment of relapsing MS – Rebif ® (interferon beta-1a) and MAVENCLAD ® (cladribine) tablets. EMD Serono aims to improve the lives of patients by addressing areas of unmet medical needs. In addition to EMD Serono's commitment to MS, the company also has a pipeline focusing on discovering new therapies that have potential in other neuroinflammatory and immune-mediated diseases, including systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE) and generalized myasthenia gravis (gMG). Article content About EMD Serono, Inc. Article content EMD Serono – the healthcare business of Merck KGaA, Darmstadt, Germany in the U.S. and Canada – aspires to create, improve and prolong life for people living with difficult-to-treat conditions like infertility, multiple sclerosis and cancer. The business is imagining the future of healthcare by working to translate the discovery of molecules into potentially meaningful outcomes for people with serious unmet medical needs. EMD Serono's global roots go back more than 350 years with Merck KGaA, Darmstadt, Germany. Today, the business has approximately 1,050 employees around the country with commercial, clinical and research operations in Massachusetts. Article content 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. Article content 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. Article content Article content Article content Article content Article content
