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STAMFORD, Conn., Sept. 29, 2022 (GLOBE NEWSWIRE) — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage biopharmaceutical firm targeted on growing life-changing medicines for sufferers with extreme uncommon illnesses and most cancers, at the moment introduced that the primary affected person has been dosed in a Phase 2 trial evaluating nirogacestat, SpringWorks’ investigational gamma secretase inhibitor, as a monotherapy in sufferers with recurrent ovarian granulosa cell tumors.
Ovarian granulosa cell tumors account for roughly 5% of all ovarian cancers and are the commonest subtype of ovarian intercourse wire tumors, representing 70% of all instances. Nearly all ovarian granulosa cell tumors harbor a mutation within the FOXL2 gene and preclinical research have demonstrated that ovarian granulosa cell tumor cell strains are prone to gamma secretase inhibition.1,2
“The science behind this study is exciting because Notch signaling, a key target of gamma secretase inhibitors, is involved in the expression of FOXL2, which is an integral component of granulosa cell development, proliferation, and function,” mentioned Panos Konstantinopoulos, M.D., Ph.D., Director of Gynecological Oncology Translational Research at Dana-Farber Cancer Center and Harvard Medical School and the Principal Investigator of the Phase 2 trial. “Without any FDA-approved therapies, there is a high unmet need for patients with recurrent ovarian granulosa cell tumors and I look forward to evaluating nirogacestat in this important study.”
“Over the course of their disease, patients with ovarian granulosa cell tumors may experience severe abdominal pain associated with a large pelvic or abdominal mass, requiring surgery and subsequent systemic therapy,” mentioned Saqib Islam, Chief Executive Officer of SpringWorks. “We are pleased to be developing a much-needed therapy for these patients and are encouraged by this new, meaningful opportunity for nirogacestat.”
The Phase 2 trial (NCT05348356) is a multi-center, single-arm, open-label examine evaluating the efficacy, tolerability, security, and pharmacokinetics of nirogacestat in sufferers with recurrent ovarian granulosa cell tumors. The examine will enroll roughly 40 sufferers who will obtain 150mg of nirogacestat twice every day. Eligible sufferers could have recurrent ovarian granulosa cell tumors and could have acquired a number of prior strains of systemic remedy. The main endpoint of the trial is goal response charge as measured by Response Evaluation Criteria in Solid Tumors (RECIST 1.1). Secondary endpoints embody progression-free survival, total survival, period of response, security and tolerability, and high quality of life assessments.
About Ovarian Granulosa Cell Tumors
Ovarian granulosa cell tumors (OvGCT) are a uncommon ovarian most cancers subtype which are often slow-growing tumors. OvGCT account for roughly 5% of all ovarian cancers and are the commonest subtype of ovarian intercourse wire stromal tumors. At the time of prognosis, sufferers sometimes current with extreme belly ache, belly distension, and irregular or postmenopausal bleeding alongside a big pelvic or belly mass.3
OvGCT are mostly recognized in ladies in the course of the perimenopausal/early postmenopausal interval with a median age of prognosis of 50 years.3 It is estimated that there are 1,500 to 2,000 new instances recognized per 12 months within the U.S., and an estimated prevalence of roughly 10,000-15,000 sufferers with OvGCT within the U.S.4,5 Prognosis for sufferers with superior illness is poor, with a 10-year survival charge of roughly 25%.6
There are at present no FDA-approved therapies for sufferers with OvGCT. Surgery is at present the mainstay of preliminary remedy, nevertheless, the chance of recurrence is excessive for these with superior illness and off-label systemic remedy, together with chemotherapy, is commonly used.
About Nirogacestat
Nirogacestat is an investigational, oral, selective, small molecule gamma secretase inhibitor in Phase 3 medical growth for desmoid tumors and in Phase 2 medical growth for ovarian granulosa cell tumors. Gamma secretase cleaves a number of transmembrane protein complexes, together with Notch, which is believed to play a task in activating pathways that contribute to development of desmoid and ovarian granulosa cell tumors.
In addition, gamma secretase has been proven to straight cleave membrane-bound B cell maturation antigen (BCMA), ensuing within the launch of the BCMA extracellular area (ECD) from the cell floor. By inhibiting gamma secretase, membrane-bound BCMA might be preserved, rising goal density whereas lowering ranges of soluble BCMA ECD, which can function decoy receptors for BCMA-directed therapies. Nirogacestat’s means to reinforce the exercise of BCMA-directed therapies has been noticed in preclinical fashions of a number of myeloma. SpringWorks is evaluating nirogacestat as a BCMA potentiator and has eight collaborations with industry-leading BCMA builders to judge nirogacestat in combos throughout modalities, together with with an antibody-drug conjugate, two CAR T cell therapies, three bispecific antibodies and a monoclonal antibody. SpringWorks has additionally fashioned analysis collaborations with Fred Hutchinson Cancer Research Center and Dana-Farber Cancer Institute to additional characterize the power of nirogacestat to modulate BCMA and potentiate BCMA-directed therapies utilizing a range of preclinical a number of myeloma fashions.
Nirogacestat has acquired Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the remedy of desmoid tumors and from the European Commission for the remedy of mushy tissue sarcoma. The FDA additionally granted Fast Track and Breakthrough Therapy Designations for the remedy of grownup sufferers with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. SpringWorks plans to submit a New Drug Application (NDA) to the FDA within the second half of 2022, which can be submitted for evaluation underneath the FDA’s Real-Time Oncology Review (RTOR) program.
About SpringWorks Therapeutics
SpringWorks is a clinical-stage biopharmaceutical firm making use of a precision medication method to buying, growing and commercializing life-changing medicines for sufferers residing with extreme uncommon illnesses and most cancers. SpringWorks has a differentiated focused oncology pipeline spanning strong tumors and hematological cancers, together with two doubtlessly registrational medical trials in uncommon tumor sorts in addition to a number of packages addressing extremely prevalent, genetically outlined cancers. SpringWorks’ strategic method and operational excellence in medical growth have enabled it to quickly advance its two lead product candidates into late-stage medical trials whereas concurrently coming into into a number of shared-value partnerships with innovators in {industry} and academia to unlock the total potential for its portfolio and create extra options for sufferers with most cancers. For extra data, go to www.springworkstx.com and observe @SpringWorksTx on Twitter and LinkedIn.
SpringWorks Forward-Looking Statements
This press launch comprises “forward-looking statements” inside the which means of the Private Securities Litigation Reform Act of 1995, as amended, regarding our business, operations, and monetary situations, together with, however not restricted to, present beliefs, expectations and assumptions relating to the longer term of our business, future plans and methods, our growth plans, our preclinical and medical outcomes, the timing of our deliberate NDA submission for nirogacestat, and our plans for looking for regulatory approval for and making nirogacestat obtainable to desmoid tumor sufferers, if accepted, in addition to regarding different future situations. Words comparable to, however not restricted to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” “would,” “should” and “could,” and related expressions or phrases, establish forward-looking statements. New dangers and uncertainties could emerge every now and then, and it’s not potential to foretell all dangers and uncertainties. Any forward-looking statements on this press launch are based mostly on administration’s present expectations and beliefs and are topic to a quantity of dangers, uncertainties and essential components that will trigger precise occasions or outcomes to vary materially from these expressed or implied by any forward-looking statements contained on this press launch, together with, with out limitation, dangers regarding: (i) the success and timing of our product growth actions, together with the initiation and completion of SpringWorks’ medical trials, (ii) the truth that topline or interim information from a medical examine will not be predictive of the ultimate or extra detailed outcomes of such examine, or the outcomes of different ongoing or future research, (iii) the success and timing of our collaboration companions’ ongoing and deliberate medical trials, (iv) the timing of our deliberate regulatory submissions and interactions, together with the NDA for nirogacestat deliberate for the second half of 2022 and the timing and consequence of choices made by the U.S. Food and Drug Administration (FDA) and different regulatory authorities, investigational evaluation boards at medical trial websites and publication evaluation our bodies; (v) whether or not FDA or different regulatory authorities would require further data or additional research, or could fail or refuse to approve or could delay approval of our drug candidates, together with nirogacestat and mirdametinib, (vi) our means to acquire and preserve regulatory approval of any of our product candidates, (vii) our plans to analysis, uncover and develop further product candidates, (viii) our means to enter into collaborations for the event of new product candidates, (ix) our means to determine manufacturing capabilities, and our and our collaboration companions’ talents to fabricate our product candidates and scale manufacturing, (x) our means to satisfy any particular milestones set forth herein, and (xi) uncertainties and assumptions relating to the affect of the COVID-19 pandemic on SpringWorks’ business, operations, medical trials, provide chain, technique, objectives and anticipated timelines.
Except as required by relevant regulation, we don’t plan to publicly replace or revise any forward-looking statements contained herein, whether or not in consequence of any new data, future occasions, modified circumstances or in any other case. Although we imagine the expectations mirrored in such forward-looking statements are affordable, we may give no assurance that such expectations will show to be appropriate. Accordingly, readers are cautioned to not place undue reliance on these forward-looking statements.
For additional data relating to the dangers, uncertainties and different components that will trigger variations between SpringWorks’ expectations and precise outcomes, it is best to evaluation the “Risk Factors” in Item 1A of Part I of SpringWorks’ Quarterly Report on Form 10-Q for the quarter ended June 30, 2022, in addition to discussions of potential dangers, uncertainties and different essential components in SpringWorks’ subsequent filings.
Contacts:
Kim Diamond
Vice President, Communications and Investor Relations
Phone: 203-561-1646
Email: [email protected]
Samantha Hilson Sandler
Director, Investor Relations
Phone: 203-461-5501
Email: [email protected]
References
1 Shah SP, Kobel M, Senz J, et al. Mutation of FOXL2 in granulosa-cell tumors of the ovary. N Engl J Med. 2009;360(26):2719–29. doi:10.1056/NEJMoa0902542.
2 Irusta G, Pazos MC, Abramovich D, De Zúñiga I, Parborell F, Tesone M. Effects of an inhibitor of the γ-secretase complicated on proliferation and apoptotic parameters in a FOXL2-mutated granulosa tumor cell line (KGN). Biol Reprod. 2013;89(1):9. doi:10.1095/biolreprod.113.108100.
3 Dridi M, Chraiet N, Batti R, et al. Granulosa cell tumor of the ovary: A retrospective examine of 31 instances and a evaluation of the literature. Int J Surg Oncol. 2018:1-4. doi:10.1155/2018/4547892.
4 Torre LA, Trabert B, Desantis CE, et al. Ovarian Cancer Statistics, 2018. CA Cancer J Clin. 2018; 68(4): 284–296. doi:10.3322/caac.21456.
5 National Cancer Institute. Cancer of the ovary – most cancers stat details. SEER. https://seer.cancer.gov/statfacts/html/ovary.html. Accessed September 2022.
6 Schumer ST, Cannistra SA. Granulosa cell tumor of the ovary. J. Clin Oncol. 2003;21 (6):1180-9. doi:10.1200/JCO.2003.10.019.
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