Springworks Therapeutics, Inc.

- Educational campaign aims to raise awareness and drive conversation about desmoid tumors, the devastating impact they have on patients' lives - STAMFORD, Conn., Feb. 19, 2025 /PRNewswire/ -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, and Jennifer Fisher, jewelry designer and desmoid tumor patient advocate, are collaborating to increase understanding of desmoid tumors as well as to encourage patients to find a specialist, like an oncologist with experience treating desmoid tumors who can discuss available treatment options to manage their desmoid tumor. Continue Reading Video of Jennifer Fisher Photo of Jennifer Fisher Desmoid tumors are aggressive and invasive tumors that can arise in any part of the body.1,2 Their tendril-like growths can wrap around nearby tissue and compress vital organs and nerves.2-4 This can cause severe and chronic pain, loss of function, reduced mobility, disfigurement, and anxiety, all of which can profoundly diminish a person's health and quality of life.1-3,5-7 Desmoid tumors are most commonly diagnosed in patients between the ages of 20 and 44 years and have a two-to-three times higher prevalence in females.8-9 "I was diagnosed with a desmoid tumor on my chest wall when I was 30 years old. I had so many questions, no answers, and didn't know what to expect for my future," said Jennifer. "Having a desmoid tumor was an emotional journey for me but it also shaped who I am today. I am passionate about sharing my story and proud to be working with SpringWorks to encourage others to seek expert care for their desmoid tumor and not let the physical pain from these tumors rob them of their vitality." At the time of Jennifer's diagnosis, there were no approved treatments for desmoid tumors. Initially her doctors recommended that Jennifer have the tumor surgically removed, which would have taken half of her left chest wall with the possibility of permanently losing mobility in her arm. "Since desmoid tumors have a high rate of recurrence after surgery, treatment guidelines now recommend systemic therapies for most desmoid tumors that are growing and/or symptomatic," stated Dr. Alessandra Maleddu, Sarcoma Specialist, University of Colorado. "As science continues to evolve, it's important for patients with desmoid tumors to be aware of the latest treatments available and to talk to their doctor about non-surgical options." People living with a desmoid tumor should closely track how their symptoms are impacting daily life to gauge if their desmoid tumor could be progressing. Having increased pain or not being able to keep up with everyday activities could be signs that it is time to take action. Individuals should speak to a specialist, like an oncologist with experience treating desmoid tumors, and advocate to find the best approach for treatment.10 "Our commitment to the desmoid tumor community runs deep, and we are pleased that Jennifer is sharing her powerful story to raise awareness about these devastating tumors and encourage patients to take action to manage their desmoid tumor," said Bhavesh Ashar, Chief Commercial Officer at SpringWorks Therapeutics. "Jennifer's experience shines a light on the many struggles that patients with desmoid tumors face and underscores the importance of patients advocating for themselves and finding the right care team to determine the best approach for their treatment." Follow Jennifer Fisher on Instagram. About Desmoid Tumors Desmoid tumors are rare, aggressive, locally invasive tumors of the soft tissues that can be serious, debilitating, and, in rare cases when vital structures are impacted, life-threatening.1,2 Desmoid tumors are most commonly diagnosed in patients between the ages of 20 and 44 years, with a two-to-three times higher prevalence in females.8,9 It is estimated that there are 1,000-1,650 new cases diagnosed per year in the United States.8,11,12 Although they do not metastasize, desmoid tumors can be associated with recurrence rates of up to 77% after surgical resection.9,13 Desmoid tumor experts and treatment guidelines now recommend systemic therapies as first-line intervention instead of surgery for most tumor locations requiring treatment.10,14 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 commercialized the first and only FDA-approved therapy for adults with progressing desmoid tumors and the first and only FDA-approved medicine for both adults and children with symptomatic neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN) not amenable to complete resection. 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 and follow @SpringWorksTx on X, LinkedIn, Facebook, Instagram, and YouTube. Contact Media: Catherine Cantone Corporate Communications Sr. Director [email protected] (203) 705-1126 References Sbaraglia M, Bellan E, Dei Tos AP. The 2020 WHO Classification of Soft Tissue Tumours: news and perspectives. Pathologica. 2021;113(2):70-84. doi:10.32074/1591-951X-213. Penel N, Chibon F, Salas S. Adult desmoid tumors: biology, management and ongoing trials. Curr Opin Oncol. 2017;29(4):268-274. doi:10.1097/CCO.0000000000000374. Constantinidou A, Scurr M, Judson I, Litchman C. Clinical presentation of desmoid tumors. In: Litchman C, ed. Desmoid Tumors. Springer; 2012:chap 2. Accessed August 2023. . doi:10.1007/978-94-007-1685-8_2. Joglekar SB, Rose PS, Sim F, Okuno S, Petersen I. Current perspectives on desmoid tumors: the Mayo Clinic approach. Cancers (Basel). 2011;3(3):3143-3155. doi:10.3390/cancers3033143. Husson O, Younger E, Dunlop A, et al. Desmoid fibromatosis through the patients' eyes: time to change the focus and organisation of care? Support Care Cancer. 2019;27(3):965-980. doi:10.1007/s00520-018-4386-8. Gounder MM, Maddux L, Paty J, Atkinson TM. Prospective development of a patient-reported outcomes instrument for desmoid tumors or aggressive fibromatosis. Cancer. 2020;126(3):531-539. doi:10.1002/cncr.32555. Bektas, M, et al. Desmoid Tumors: A Comprehensive Review. Adv Therapeutics 2023. doi.org/10.1007/s12325-023-02592-0. van Broekhoven DLM, Grünhagen DJ, den Bakker MA, van Dalen T, Verhoef C. Time trends in the incidence and treatment of extra-abdominal and abdominal aggressive fibromatosis: a population-based study. Ann Surg Oncol. 2015;22(9):2817-2823. doi:10.1245/s10434-015-4632-y. Skubitz KM. Biology and treatment of aggressive fibromatosis or desmoid tumor. Mayo Clin Proc. 2017;92(6):947-964. doi:10.1016/j.mayocp.2017.02.012. Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Soft Tissue Sarcoma V.2.2023. © National Comprehensive Cancer Network, Inc. 2023. All rights reserved. Accessed August 2023. To view the most recent and complete version of the guideline, go online to NCCN.org. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way. Orphanet Report Series: Rare Diseases collection. Prevalence and incidence of rare diseases: bibliographic data. Number 1, January 2022. Accessed November 5, 2024. . U.S. Department of Commerce. News Blog. U.S. population estimated at 332,403,650 on Jan. 1, 2022. Accessed August 2023. blog/2022/01/us-population-estimated-332403650-jan-1-2022#:~. Easter DW, Halasz NA. Recent trends in the management of desmoid tumors. Summary of 19 cases and review of the literature. Ann Surg. 1989;210(6):765-769. doi:10.1097/00000658-198912000-00012. Gronchi A, et al. Desmoid Tumor Working Group. The management of desmoid tumours: a joint global consensus-based guideline approach for adult and pediatric patients. Eur J Cancer. 2020;127:96-107. doi:10.1016/j.ejca.2019.11.013. SOURCE SpringWorks Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

– GOMEKLI is the first and only medicine approved for both adults and children with NF1-PN – – Approval based on positive data from Phase 2b ReNeu trial, which showed GOMEKLI treatment resulted in robust ORR, deep and durable reductions in tumor volume, and a manageable safety profile – – SpringWorks granted rare pediatric disease priority review voucher by the FDA – Feb. 11, 2025 -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the U.S. Food and Drug Administration (FDA) has approved GOMEKLI™ (mirdametinib), SpringWorks’ MEK inhibitor, for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.1 With the approval, SpringWorks was granted a rare pediatric disease priority review voucher (PRV) by the FDA. “The NF1-PN patient community has a great need for more treatment options. With today’s approval, we are honored to serve both adults and children with NF1-PN and provide them with a therapy that has the potential to shrink their tumors and offer meaningful symptomatic relief,” said Saqib Islam, Chief Executive Officer of SpringWorks. “We are grateful to each clinical trial participant, their families, the investigators, and the patient advocacy groups involved in the journey towards making GOMEKLI available in the U.S. I am proud that we are delivering on our commitment to patients with devastating diseases with our company’s second FDA approval in less than 18 months.” NF1 is a genetic disorder that currently affects approximately 100,000 children and adults in the United States.2,3 Patients with NF1 have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PNs, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment.2,4 There are approximately 40,000 people in the United States living with NF1-PN, the majority of whom are adults that have not had an approved medicine until GOMEKLI.5 Plexiform neurofibromas can transform into malignant peripheral nerve sheath tumors, an aggressive and potentially fatal disease.6 Surgical removal can be challenging due to the infiltrative tumor growth pattern of plexiform neurofibromas along nerves, and up to approximately 85% of plexiform neurofibromas are considered not amenable to complete resection.7,8,9 “Patients with NF1-PN often face significant challenges with their health and have had limited treatment options to manage this devastating condition,” said Christopher Moertel, M.D., Medical Director Pediatric Neuro-Oncology and Neurofibromatosis Programs and Kenneth and Betty Jayne Dahlberg Professor of Pediatrics, University of Minnesota, and lead investigator of the ReNeu trial. “It was very encouraging in the ReNeu trial to see that GOMEKLI provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy. This approval represents an important advance, especially for adults who previously did not have an approved treatment.” GOMEKLI was approved under Priority Review and SpringWorks received a rare pediatric disease priority review voucher from the FDA. GOMEKLI was previously granted Orphan Drug and Fast Track designations for the treatment of NF1-PN. The FDA approval of GOMEKLI is based on results from the Phase 2b ReNeu trial, which enrolled 114 patients with NF1-PN ≥2 years of age (58 adults and 56 pediatric patients).10 GOMEKLI met the primary endpoint of confirmed objective response rate (ORR), as assessed by blinded independent central review, demonstrating a 41% ORR (N= 24/ 58) in adults and 52% in children (N=29/56).10 Tumor volume reductions were deep and durable; the median best percentage change in target PN volume was -41% (range: -90 to 13%) in adults and -42% (range: -91 to 48%) in children.10 Eighty-eight percent of adults and 90% of children with a confirmed response had a response of at least 12 months duration, and 50% and 48%, respectively, had a response of at least 24 months duration.10 Patients in both cohorts also experienced early and sustained significant improvements from baseline in pain, and quality of life, as assessed across multiple patient-reported outcome tools.10 GOMEKLI demonstrated a manageable safety and tolerability profile.1 The most common adverse events (>25%) reported in adults receiving GOMEKLI were rash, diarrhea, nausea, musculoskeletal pain, vomiting and fatigue.1 The most common adverse events (>25%) occurring in children were rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, paronychia, left ventricular dysfunction, and nausea.1 Please see additional Important Safety Information below, including Warnings & Precautions relating to ocular toxicity, left ventricular dysfunction, dermatologic adverse reactions, and embryo-fetal toxicity.1 “We are excited to celebrate the extraordinary milestone of our partners and long-term friends at SpringWorks for the NF1-PN community. This FDA approval shows the power of collaboration to advance innovative science for drugs that may otherwise not have been taken forward,” said Annette Bakker, Ph.D., Chief Executive Officer of the Children’s Tumor Foundation. "When industry, researchers, and organizations like ours driving treatment innovation join forces, scientific progress moves faster, and patients gain access to the therapies they need. Every treatment approval is hard-won, built on research, persistence, and partnership. Today, that work delivers a critical new option for NF patients of all ages.” “NF1-PN is a complex, devastating disease that affects not only individual patients, but entire families. Treatment advances are crucial to achieving better outcomes for patients and this FDA approval offers hope for NF patients and their families,” said Kim Bischoff, Executive Director, NF Network. SpringWorks is dedicated to helping patients with NF1-PN access GOMEKLI and to providing support throughout their treatment journey. The SpringWorks CareConnections™ program is a comprehensive patient support program that offers personalized support services and resources to eligible GOMEKLI patients, including insurance coverage information and access support, financial assistance and personalized educational and emotional support. Physicians and patients can contact 1-844-CARES-55 (1-844-227-3755) or visit www.springworkstxcares.com for more information. GOMEKLI is available in 1 and 2 mg capsules and in a 1mg tablet for oral suspension, which dissolves easily in water. GOMEKLI is expected to be available through a specialty pharmacy and specialty distributor network in the United States within two weeks. SpringWorks’ Marketing Authorization Application for mirdametinib for the treatment of children and adults with NF1-PN was validated by the European Medicines Agency (EMA) and is currently under review; a decision is expected from the European Commission in 2025. Neurofibromatosis type 1 (NF1) is a rare genetic disorder that arises from mutations in the NF1 gene, which encodes for neurofibromin, a key suppressor of the MAPK pathway.11,12 NF1 is the most common form of neurofibromatosis, with an estimated global birth incidence of approximately 1 in 2,500 individuals, and there are approximately 100,000 patients living with NF1 in the United States.2,3 The clinical course of NF1 is heterogeneous and manifests in a variety of symptoms across numerous organ systems, including abnormal pigmentation, skeletal deformities, tumor growth and neurological complications, such as cognitive impairment.13 Patients with NF1 have an 8 to 15-year mean reduction in their life expectancy compared to the general population.14 NF1 patients have approximately a 30-50% lifetime risk of developing plexiform neurofibromas, or PN, which are tumors that grow in an infiltrative pattern along the peripheral nerve sheath and that can cause severe disfigurement, pain and functional impairment; in rare cases, NF1-PN may be fatal.2,4,6 NF1-PNs are most often diagnosed in the first two decades of life.2 These tumors can be aggressive and are associated with clinically significant morbidities; typically, they grow more rapidly during childhood.15,16 Surgical removal of these tumors can be challenging due to the infiltrative tumor growth pattern along nerves and can lead to permanent nerve damage and disfigurement.6 Up to approximately 85% of plexiform neurofibromas are considered not amenable to complete resection.7,8,9 GOMEKLI™ is an oral, small molecule MEK inhibitor approved in the United States for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection. 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. References GOMEKLI. Prescribing Information. SpringWorks Therapeutics, Inc. Prada C, Rangwala F, Martin L, et al. Pediatric Plexiform Neurofibromas: Impact on Morbidity and Mortality in Neurofibromatosis Type 1. J Pediatr. 2012;160(3):461-467. doi:10.1016/j.jpeds.2011.08.051. Ferner R. Neurofibromatosis 1 and neurofibromatosis 2: a twenty first century perspective. The Lancet Neurology. 2007;6(4):340-351. doi:10.1016/s1474-4422(07)70075-3. Miller DT, et al. Health supervision for children with neurofibromatosis Type 1. Pediatrics. 2019;143(5):e20190660. Data on File. Kamaludin, Siti Nurhazwani et al. “Plexiform neurofibromatosis with peripheral malignant nerve sheath tumor and scoliosis - more surveillance imaging needed?.” Radiology case reports vol. 17,7 2388-2393. 6 May. 2022, doi:10.1016/j.radcr.2022.03.111. Needle M, Cnaan A, Dattilo J, et al. Prognostic signs in the surgical management of plexiform neurofibroma: The Children’s Hospital of Philadelphia experience, 1974-1994. J Pediatr. 1997;131(5):678-682. doi:10.1016/s0022-3476(97)70092-1. Ejerskov, C., Farholt, S., Nielsen, F.S.K. et al. Clinical Characteristics and Management of Children and Adults with Neurofibromatosis Type 1 and Plexiform Neurofibromas in Denmark: A Nationwide Study. Oncol Ther 11, 97–110 (2023). https://doi-org.libproxy1.nus.edu.sg/10.1007/s40487-022-00213-4. Wolkenstein, P. et al. (2023) ‘French cohort of children and adolescents with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas: Cassiopea study’, European Journal of Medical Genetics, 66(5), p. 104734. doi:10.1016/j.ejmg.2023.104734. Moertel CL, Fisher MJ, Weiss BD, et al. ReNeu: A pivotal, Phase IIb trial of mirdametinib in adults and children with symptomatic neurofibromatosis type 1-associated plexiform neurofibroma. J Clin Oncol. 2024;JCO.24.01034. doi.org/10.1200/JCO.24.01034. Yap YS, McPherson JR, Ong CK, et al. The NF1 gene revisited - from bench to bedside. Oncotarget. 2014;5(15):5873-5892. doi:10.18632/oncotarget.2194. Rasmussen S, Friedman J. NF1 Gene and Neurofibromatosis 1. Am J Epidemiol. 2000;151(1):33-40. doi:10.1093/oxfordjournals.aje.a010118. Weiss BD, Wolters PL, Plotkin SR, et al. NF106: A neurofibromatosis clinical trials consortium Phase II trial of the MEK inhibitor mirdametinib (PD-0325901) in adolescents and adults with NF1-related plexiform neurofibromas. J Clin Onc. 2021;JCO.20.02220.doi.org/10. 1200/JCO.20.02220. Lee: Lee TJ, et al. Incidence and prevalence of neurofibromatosis type 1 and 2: a systematic review and meta-analysis. Orphanet J Rare Dis. 2023;18(1):292. doi:10.1186/s13023-023-02911-2. Gross A, Singh G, Akshintala S, et al. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1. Neuro Oncol. 2018;20(12):1643-1651. doi:10.1093/neuonc/noy067. Nguyen R, Dombi E, Widemann B, et al. Growth dynamics of plexiform neurofibromas: a retrospective cohort study of 201 patients with neurofibromatosis 1. Orphanet J Rare Dis. 2012;7(1):75. doi:10.1186/1750-1172-7-75. The content above comes from the network. if any infringement, please contact us to modify.

A genetic disease that causes tumors to form on nerves has a new FDA-approved treatment, a SpringWorks Therapeutics drug that can address a wider range of patients than the AstraZeneca product that was first to treat this rare disorder. The disease, neurofibromatosis type 1 (NF1), is technically not cancer as the tumors that form are benign. But NF1 can lead to cancers. Even if a patient’s tumors aren’t cancerous, they are painful and disfiguring. The FDA approval announced Tuesday covers the treatment of NF1 tumors that cannot be surgically removed. The drug, known in development as mirdametinib, will be marketed under the brand name Gomekli. SpringWorks expects the new product will become available within two weeks. Stamford, Connecticut-based SpringWorks estimates that of the approximately 100,000 in the U.S. who have NF1, about 40,000 have tumors that infiltrate nerves. The disease stems from mutations in the NF1 gene, which codes for a protein key to suppressing MAPK, a pathway that, when hyperactivated, drives cancer growth. Tumors that result from the mutations are usually aggressive, growing rapidly during a patient’s childhood. Surgery is the first treatment option, but the location of tumors at or near vital structures or organs can make this choice risky. Off-label drug options include cancer medications, such as chemotherapy and immunotherapy. Exclusive Improving the Healthcare Financial Experience to Help Care Flow Zelis CEO Amanda Eisel shares her perspective on how the company is solving the problems of a fragmented health financial system to benefit all. By Stephanie Baum Gomekli is an oral small molecule designed to block MEK1 and MEK2, two proteins that play key roles in the MAPK pathway. MEK inhibitors have already been approved for treating certain cancers. Koselugo, the AstraZeneca drug that became the first FDA approved NF1 therapy in 2020, is a MEK inhibitor that was initially tested in various solid tumors. In NF1, Koselugo is approved only for treating pediatric patients age 2 and older. The SpringWorks drug has an advantage with an approval that covers the treatment of adults as well as children. The FDA decision for Gomekli was based on the results of a single-arm, Phase 2 study that enrolled 114 patients — 58 adults and 56 children — with symptomatic, inoperable NF1-associated tumors. The main goal was to measure for the disappearance or reduction of tumors. Results showed the overall response rate was 41% for adults and 52% for children. The most common adverse reactions included rash, diarrhea, nausea, and muscle pain. The study results were published this past November in the Journal of Clinical Oncology. Dr. Christopher Moertel, medical director pediatric neuro-oncology and neurofibromatosis programs at the University of Minnesota and the lead investigator of the ReNeu clinical trial, said NF1 patients face significant health challenges and have limited treatment options. “It was very encouraging in the ReNeu trial to see that Gomekli provided deep and durable responses, with a manageable safety profile that enabled patients to stay on therapy,” Moertel said in SpringWorks’ announcement. “This approval represents an important advance, especially for adults who previously did not have an approved treatment.” presented by Artificial Intelligence What Keeps Healthcare CIOs Up at Night: Balancing Technology Investments with Consumer Expectations When it comes to managing inbound phone calls, underperformance has devastating cost implications. By Stephanie Baum Gomekli is still under regulatory review in Europe. In the U.S., the drug will be available as a capsule as well as a tablet that may be swallowed or mixed in water. The twice-daily drug will come in two dosage strengths. Precise dosing is determined by a patient’s body surface area, taking into account both height and weight. SpringWorks set a wholesale price of $206.25 per mg, according to a Wednesday regulatory filing. The company estimates the average cost of treatment will be about $22,000 per month for pediatric patients and $30,000 per month for adults. The Gomekli approval comes as SpringWorks has emerged as a potential acquisition target for Merck KGaA. Following a Reuters report of negotiations for a deal, the German company issued a statement Monday confirming advanced discussions with SpringWorks. However, Merck KGaA said the companies have not entered a legally binding agreement, adding that “critical conditions have yet to be met.” If Merck KGaA can swing a deal for SpringWorks, it will get more than the new NF1 drug. In 2023, SpringWorks drug Ogsiveo received the FDA nod for treating desmoid tumors, a rare type of tumor affecting connective tissue. For the nine months ended Sept. 30, 2024, Ogsiveo accounted for $110 million in revenue, according to the biotech’s most recent financial report. Both Ogsiveo and Gomekli were initially developed by Pfizer, which spun them off into standalone company SpringWorks in 2017. Beyond NF1, SpringWorks is testing Gomekli in certain cancers. As a monotherapy, the drug has reached mid-stage clinical testing in pediatric low-grade gliomas. Under a partnership with BeiGene, Gomekli is also being tested in solid tumors driven by RAS and RAF mutations; a Phase 1 study is evaluating the SpringWorks drug in combination with BeiGene’s experimental lifirafenib, a small molecule inhibitor of RAF enzymes. Photo by SpringWorks Therapeutics