Latest news with #CSF-1R
Yahoo
29-05-2025
- Business
- Yahoo
Merck KGaA (MKGAF) Files for Global Approval of Pimicotinib After Positive Phase III TGCT Results
Merck KGaA (OTC:MKGAF) is seeking worldwide regulatory approval for its CSF-1R inhibitor pimicotinib following robust Phase III results in tenosynovial giant cell tumour (TGCT). The MANEUVER trial, conducted with Abbisko Therapeutics, showed a 61.9% overall response rate (ORR) by tumour volume score (TVS) after 25 weeks, far surpassing placebo (3.2%). This efficacy outperformed Daiichi Sankyo's Turalio (56% ORR) and was just behind Ono Pharmaceutical's Romvimza (67% ORR), which received FDA approval in February 2025. Quality of life improvements were notable, with significant gains in range of movement, stiffness, and pain reduction. Importantly, pimicotinib demonstrated a favorable safety profile: while all patients experienced some adverse events, only 4.8% were serious, and there were no signs of liver toxicity, a key differentiator from other therapies. TGCT, a rare non-cancerous joint tumour, often recurs after surgery, making effective drug options crucial. Merck KGaA (OTC:MKGAF), having secured global rights from Abbisko in March 2025, has filed for approval in China and plans an FDA submission by year-end. Full results will be presented at ASCO 2025 in Chicago. While we acknowledge the potential of MKGAF to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than MKGAF and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None. 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
Yahoo
29-05-2025
- Business
- Yahoo
Merck & Co., Inc. (MRK) Files for Global Approval of Pimicotinib After Positive Phase III TGCT Results
Merck & Co., Inc. (NYSE:MRK) is seeking worldwide regulatory approval for its CSF-1R inhibitor pimicotinib following robust Phase III results in tenosynovial giant cell tumour (TGCT). The MANEUVER trial, conducted with Abbisko Therapeutics, showed a 61.9% overall response rate (ORR) by tumour volume score (TVS) after 25 weeks, far surpassing placebo (3.2%). This efficacy outperformed Daiichi Sankyo's Turalio (56% ORR) and was just behind Ono Pharmaceutical's Romvimza (67% ORR), which received FDA approval in February 2025. Pixabay/Public Domain Quality of life improvements were notable, with significant gains in range of movement, stiffness, and pain reduction. Importantly, pimicotinib demonstrated a favorable safety profile: while all patients experienced some adverse events, only 4.8% were serious, and there were no signs of liver toxicity, a key differentiator from other therapies. TGCT, a rare non-cancerous joint tumour, often recurs after surgery, making effective drug options crucial. Merck & Co., Inc. (NYSE:MRK), having secured global rights from Abbisko in March 2025, has filed for approval in China and plans an FDA submission by year-end. Full results will be presented at ASCO 2025 in Chicago. While we acknowledge the potential of MRK to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than MRK and that has 100x upside potential, check out our report about this READ NEXT: and Disclosure: None.
Business Upturn
28-05-2025
- Business
- Business Upturn
Pimicotinib Demonstrates Best-in-Class Potential with Significant Efficacy and Clinically Meaningful Improvements in Patients with Tenosynovial Giant Cell Tumor
Darmstadt, Germany: 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 Canada-, UK- or US-based media Merck, a leading science and technology company, 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 holds worldwide commercialization rights for pimicotinib. Advancing the Future of Cancer Care At Merck, 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. Learn more at About Merck Merck, 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, 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 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, 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 Disclaimer: The above press release comes to you under an arrangement with Business Wire. Business Upturn takes no editorial responsibility for the same.
Business Wire
28-05-2025
- Business
- Business Wire
Pimicotinib Demonstrates Best-in-Class Potential with Significant Efficacy and Clinically Meaningful Improvements in Patients with Tenosynovial Giant Cell Tumor
DARMSTADT, Germany--(BUSINESS WIRE)--Merck, a leading science and technology company, 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 holds worldwide commercialization rights for pimicotinib. Advancing the Future of Cancer Care At Merck, 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. Learn more at About Merck Merck, 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, 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 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, 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.
Yahoo
01-04-2025
- Business
- Yahoo
Merck Exercised the Option for the Global Rights of Abbisko Therapeutics's Pimicotinib
SHANGHAI, March 31, 2025 /PRNewswire/ -- On April 1, 2025, Beijing Time, Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics") announced that Merck has exercised the global commercialization option for pimicotinib (ABSK021), with an option exercise fee of USD85.0 million, under the licensing agreement signed in December 2023. The development represents further deepening of the collaboration, underscoring the unwavering commitment and confidence that both parties possess for the ongoing advancement of pimicotinib. In December 2023, Abbisko Therapeutics entered into an exclusive licensing agreement (the "Agreement") with Merck regarding pimicotinib, a CSF-1R inhibitor. Under the terms of the Agreement, Merck initially had an exclusive license to commercialize pimicotinib for all indications in the Chinese mainland, Hong Kong, Macau and Taiwan with an exclusive option for global commercial rights of pimicotinib. Merck now has exercised such option and has the exclusive license to commercialize pimicotinib worldwide. Abbisko Therapeutics has already received the one-time, non-refundable upfront payment of USD70.0 million in February 2024, and is now entitled to receive the additional option exercise fee of USD85.0 million. Additionally, Merck has the option to co-develop pimicotinib for additional indications under certain conditions. In total, Abbisko Therapeutics is eligible to receive up to USD605.5 million in payments, including upfront, development, and commercial milestones, as well as double-digit percentage royalties on annual net sales. Pimicotinib is a novel, orally administered, highly selective, and potent small molecule CSF-1R inhibitor, independently developed by Abbisko Therapeutics. Pimicotinib's positive topline results from the Global Phase III MANEUVER Study in tenosynovial giant cell tumor ("TGCT") were successfully released in November 2024, where MANEUVER met the primary endpoint with an objective response rate ("ORR") at Week 25 of 54.0% for pimicotinib compared with 3.2% for placebo (p<0.0001), and treatment with oral, once-daily pimicotinib was well-tolerated with very low rates of discontinuation due to treatment-related adverse events, and with no evidence of cholestatic hepatotoxicity. Yao-Chang Xu, Chairman and CEO of Abbisko Therapeutics, said, "Pimicotinib represents a key advancement within the emerging class of CSF-1R inhibitors, demonstrating a meaningful clinical efficacy and safety profile that positions it as an innovative treatment option for worldwide TGCT patients. We look forward to deepening our collaboration with the Merck team to expedite the registration process of pimicotinib to bring benefits to patients as quickly as possible." "Today marks a significant milestone in our partnership with Abbisko as we work together to deliver a potentially best-in-class therapy for patients with TGCT around the world," said Andrew Paterson, Chief Marketing Officer for the Healthcare business sector of Merck. "This collaboration underscores our commitment to advancing new treatment options in rare oncology for patients who need them. With this important step forward, we aim to transform the treatment landscape and offer hope to those living with TGCT, who today have very limited treatment options." View original content: SOURCE Abbisko Sign in to access your portfolio
