SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a clinical-stage
biopharmaceutical company focused on developing life-changing
medicines for patients with severe rare diseases and cancer, today
announced the presentation of additional data from the Phase 3 DeFi
trial at the 2023 Connective Tissue Oncology Society (CTOS) Annual
Meeting, being held November 1-4, 2023. These data demonstrate the
impact of nirogacestat, an investigational gamma secretase
inhibitor, on physical and role functioning in adults with desmoid
tumors. Data from the DeFi trial demonstrated statistically
significant and clinically meaningful improvements across all
primary and key secondary endpoints and were previously presented
at leading medical congresses and published in the March 9, 2023
edition of the New England Journal of Medicine.1-3
“Desmoid tumors are locally aggressive and invasive soft tissue
tumors that can cause severe pain and functional impairment. These
impairments can be physical, such as difficulty walking or carrying
out daily tasks, or role-related, such as difficulty caring for
children or working, and they severely impact the day-to-day lives
of patients,” said Jim Cassidy, M.D., Ph.D., Chief Medical Officer
of SpringWorks. “We are pleased that the robust data from our Phase
3 DeFi trial demonstrated that nirogacestat provided clinically
meaningful improvements in key patient-reported outcomes, including
pain as well as both physical and role functioning. We look forward
to the opportunity to bring this important new medicine to the
desmoid tumor community following anticipated U.S. regulatory
approval.”
Poster Presentation at the 2023 CTOS
Meeting
Impact of Nirogacestat on Functional Status in Patients
with Desmoid Tumors: Results from the Phase 3 DeFi
Study
Abstract #: 1571083Poster #: P
188Poster Session Date and Time: Thursday,
November 2, 5:30-6:30 p.m. CEST
As previously reported in the DeFi trial (NCT03785964),
nirogacestat met its primary endpoint of significantly improving
progression-free survival compared to placebo in adult patients
with progressing desmoid tumors (hazard ratio: 0.29 [95% CI,
0.15–0.55]; P<0.001). Nirogacestat also achieved a
significant and clinically meaningful improvement in physical and
role functioning status, a key secondary endpoint, compared with
placebo at Cycle 10 (p<0.001). The most frequently reported
treatment-emergent adverse events that occurred in participants
receiving nirogacestat were diarrhea (84%), ovarian dysfunction
(75% of women of childbearing potential), nausea (54%), fatigue
(51%), hypophosphatemia (42%), and maculopapular rash (32%).
During the DeFi study, the impact of nirogacestat on functional
status was evaluated. Changes from baseline in physical and role
functioning were compared between nirogacestat and placebo at Cycle
10, the prespecified time point for key secondary endpoints.
Statistically significant and clinically meaningful improvements in
physical and role functioning were observed with nirogacestat
compared with placebo at Cycle 10 across all three assessment tools
used: the GOunder/Desmoid Tumor Research Foundation DEsmoid Impact
Scale (GODDESS DTIS) Physical Functioning (PF) subscale, the
European Organisation for Research and Treatment of Cancer Core
Quality of Life Questionnaire (EORTC QLQ-C30) PF and Role
Functioning (RF) subscales, and the Patient-Reported Outcomes
Measurement Information System Physical Function Short Form 10a
(PROMIS PF10a) tool. Improvements in functional status were rapid,
becoming evident as early as Cycle 2 (the first post-treatment time
point evaluated) and these improvements were maintained through
Cycle 24 across multiple tools measuring physical and role
functioning.
The following results are being presented at CTOS:
- Nirogacestat significantly improved mean physical functioning
score from baseline per the GODDESS DTIS PF domain compared with
placebo at the pre-specified time point. The GODDESS DTIS PF domain
captures varying degrees of vigorous and moderate daily activity,
including moving and reaching.
- Nirogacestat significantly improved mean physical functioning
score from baseline per the EORTC QLQ-C30 PF subscale compared with
placebo at the pre-specified time point. The EORTC QLQ-C30 PF
subscale captures the concepts of strenuous activities, taking a
long walk, taking a short walk, need to stay in a bed or chair, and
help with eating, dressing, washing, and using the toilet.
- Nirogacestat significantly improved mean role functioning score
from baseline per the EORTC QLQ-C30 RF subscale compared with
placebo at the pre-specified time point. The EORTC QLQ-C30 RF
subscale captures the concepts of work or other daily activities
and hobbies or leisure activities.
- At Cycle 10, patients receiving nirogacestat were five times
more likely to have a clinically meaningful improvement in physical
functioning (GODDESS DTIS Physical Functioning and EORTC QLQ-C30
Physical Functioning), and twice as likely to have a clinically
meaningful improvement in role functioning (EORTC QLQ-C30 Role
Functioning) than those receiving placebo. By Cycle 4, the mean
PROMIS PF10a score in the nirogacestat arm reached the average
physical function observed in the general U.S. population whereas
the score of the placebo arm did not.
“Treatment goals for patients with desmoid tumors often focus on
tumor growth endpoints, such as progression-free survival, but
reducing pain and improving functioning are also very important
since these have a significant impact on patients’ quality of
life,” said Bernd Kasper, M.D., Ph.D., University of Heidelberg,
Mannheim Cancer Center, Mannheim, Germany and Principal
Investigator of the DeFi trial. “It is very encouraging that
patients experienced meaningful improvements in their functional
status while on nirogacestat and that these improvements were
sustained over the course of the study.”
About the DeFi Trial
DeFi (NCT03785964) is a global, randomized (1:1), double-blind,
placebo-controlled Phase 3 trial evaluating the efficacy, safety
and tolerability of nirogacestat in adult patients with progressing
desmoid tumors. The double-blind phase of the study randomized 142
patients (nirogacestat, n=70; placebo n=72) to receive 150 mg of
nirogacestat or placebo twice daily. Key eligibility criteria
included tumor progression by ≥20% as measured by Response
Evaluation Criteria in Solid Tumors (RECIST 1.1) within 12 months
prior to screening. The primary endpoint was progression-free
survival, as assessed by blinded independent central review, or
death by any cause. Secondary and exploratory endpoints include
safety and tolerability measures, objective response rate (ORR),
duration of response, changes in tumor volume assessed by magnetic
resonance imaging (MRI), and changes in patient-reported outcomes
(PROs). DeFi includes an open-label extension phase, which is
ongoing.
About Desmoid Tumors
Desmoid tumors are rare, aggressive, locally invasive,
potentially morbid tumors of the soft tissues.4,5 While they do not
metastasize, desmoid tumors are associated with a high rate of
recurrence.4-6 Sometimes referred to as aggressive fibromatosis, or
desmoid fibromatosis, these soft tissue tumors can be serious,
debilitating, and in rare cases when vital organs are impacted,
they can be life-threatening.5,8
Desmoid tumors are most commonly diagnosed in patients between
the ages of 20 to 44 years, with a two-to-three times higher
prevalence in females.7-10 It is estimated that there are
1,000-1,650 new cases diagnosed per year in the United
States.10-12
Historically, desmoid tumors were treated with surgical
resection, but this approach has become less favored due to a high
recurrence rate after surgery.4,7,13 There are currently no
FDA-approved therapies for the treatment of desmoid tumors.
About Nirogacestat
Nirogacestat is an oral, selective, small molecule gamma
secretase inhibitor in Phase 3 clinical development for desmoid
tumors. SpringWorks is also evaluating nirogacestat as a potential
treatment for patients with ovarian granulosa cell tumors and for
patients with multiple myeloma as part of several B-cell maturation
agent (BCMA) combination therapy regimens in collaboration with
leaders in industry and academia. Nirogacestat is an
investigational drug for which safety and efficacy have not been
established.
The U.S. Food and Drug Administration (FDA) has accepted the New
Drug Application (NDA) for nirogacestat for the treatment of adults
with desmoid tumors, which is being reviewed under the FDA's
Real-Time Oncology Review program. The NDA has been given a
Prescription Drug User Fee Act (PDUFA) action date of November 27,
2023. The FDA also granted Fast Track and Breakthrough Therapy
Designations to nirogacestat for the treatment of adult patients
with progressive, unresectable, recurrent or refractory desmoid
tumors or deep fibromatosis. In addition, nirogacestat has received
Orphan Drug Designation from the FDA for the treatment of desmoid
tumors and from the European Commission for the treatment of soft
tissue sarcoma.
About SpringWorks Therapeutics
SpringWorks is a clinical-stage biopharmaceutical company
applying a precision medicine approach to acquiring, developing and
commercializing life-changing medicines for patients living with
severe rare diseases and cancer. SpringWorks has a differentiated
targeted oncology pipeline spanning solid tumors and hematological
cancers, including two late-stage clinical trials in rare tumor
types as well as several programs addressing highly prevalent,
genetically defined cancers. SpringWorks’ strategic approach and
operational excellence in clinical development have enabled it to
rapidly advance its two lead product candidates into late-stage
clinical trials while simultaneously entering into multiple
shared-value partnerships with innovators in industry and academia
to unlock the full potential for its portfolio and create more
solutions for patients with cancer. For more information, visit
www.springworkstx.com and follow @SpringWorksTx on X (formerly
Twitter), LinkedIn, and YouTube.
SpringWorks Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, relating to our business, operations, and
financial conditions, including, but not limited to, current
beliefs, expectations and assumptions regarding the future of our
business, future plans and strategies, our development plans, our
preclinical and clinical results, the potential for nirogacestat to
become an important new treatment for adult patients with desmoid
tumors, expectations regarding the timing and results of the FDA’s
review of the NDA for nirogacestat, including the FDA's PDUFA
target action date for the NDA, and the adequacy of the data
contained in the NDA to serve as the basis for an approval of
nirogacestat for the treatment of adults with desmoid tumors, as
well as relating to other future conditions. Words such as, but not
limited to, “look forward to,” “believe,” “expect,” “anticipate,”
“estimate,” “intend,” “plan,” “would,” “should” and “could,” and
similar expressions or words, identify forward-looking statements.
New risks and uncertainties may emerge from time to time, and it is
not possible to predict all risks and uncertainties. Any
forward-looking statements in this press release are based on
management’s current expectations and beliefs and are subject to a
number of risks, uncertainties and important factors that may cause
actual events or results to differ materially from those expressed
or implied by any forward-looking statements contained in this
press release, including, without limitation, risks relating to:
(i) the success and timing of our product development activities,
including the initiation and completion of SpringWorks’ clinical
trials, (ii) the fact that topline or interim data from a clinical
study may not be predictive of the final or more detailed results
of such study, or the results of other ongoing or future studies,
(iii) the success and timing of our collaboration partners’ ongoing
and planned clinical trials, (iv) the timing of our planned
regulatory submissions and interactions, including the timing and
outcome of decisions made by the U.S. Food and Drug Administration
(FDA) and other regulatory authorities, investigational review
boards at clinical trial sites and publication review bodies; (v)
whether FDA or other regulatory authorities will require additional
information or further studies, or may fail or refuse to approve or
may delay approval of our drug candidates, (vi) our ability to
obtain and maintain regulatory approval of any of our product
candidates, (vii) our plans to research, discover and develop
additional product candidates, (viii) our ability to maintain
adequate patent protection and successfully enforce patent claims
against third parties, (ix) our ability to enter into
collaborations for the development of new product candidates, (x)
our ability to establish manufacturing capabilities, and our and
our collaboration partners’ abilities to manufacture our product
candidates and scale production, and (xi) our ability to meet any
specific milestones set forth herein.
Except as required by applicable law, we do not plan to publicly
update or revise any forward-looking statements contained herein,
whether as a result of any new information, future events, changed
circumstances or otherwise. Although we believe the expectations
reflected in such forward-looking statements are reasonable, we can
give no assurance that such expectations will prove to be correct.
Accordingly, readers are cautioned not to place undue reliance on
these forward-looking statements.
For further information regarding the risks, uncertainties and
other factors that may cause differences between SpringWorks’
expectations and actual results, you should review the “Risk
Factors” in Item 1A of Part II of SpringWorks’ Quarterly Report on
Form 10-Q for the quarter ended June 30, 2023, as well as
discussions of potential risks, uncertainties and other important
factors in SpringWorks’ subsequent filings.
Contacts:
Kim DiamondVice President, Communications and Investor
Relations Phone: 203-561-1646 Email:
kdiamond@springworkstx.com
Samantha Hilson Sandler Senior Director, Investor
Relations Phone: 203-461-5501 Email:
samantha.sandler@springworkstx.com
References
1 van der Graaf WT, Gounder M, Ratan R, et al. Impact of
nirogacestat on pain, a key symptom in patients with desmoid tumors
(DT): results from the phase 3 DeFi study. Poster presented at:
American Society of Clinical Oncology Annual Meeting; June 2-6,
2023; Chicago, IL.
2 Kasper B, Ratan R, Alcindor T, et al. DeFi: A phase 3 trial of
nirogacestat for progressing desmoid tumors (DT). Oral presentation
at: Annual Meeting of the European Society for Medical Oncology;
September 9-13, 2022; Paris, France.
3 Gounder M et al. Nirogacestat, a Gamma-Secretase Inhibitor for
Desmoid Tumors. N Engl J Med 2023; 388:898-912. doi:
10.1056/NEJMoa2210140
4 Kasper B, Baumgarten C, Garcia J, et al; Desmoid Working
Group. An update on the management of sporadic desmoid-type
fibromatosis: a European Consensus Initiative between Sarcoma
PAtients EuroNet (SPAEN) and European Organization for Research and
Treatment of Cancer (EORTC)/Soft Tissue and Bone Sarcoma Group
(STBSG). Ann Oncol. 2017;28(10):2399-2408.
5 Penel N, Chibon F, Salas S. Adult desmoid tumors: biology,
management and ongoing trials. Curr Opin Oncol.
2017;29(4):268-274.
6 Xie Y, Xie K, Gou Q, He J, Zhong L, Wang Y. Recurrent desmoid
tumor of the mediastinum: a case report. Oncol Lett.
2014;8(5):2276-2278.
7 Skubitz KM. Biology and treatment of aggressive fibromatosis
or desmoid tumor. Mayo Clin Proc. 2017;92(6):947-964.
8 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.
9 Penel N, Coindre JM, Bonvalot S, et al. Management of desmoid
tumours: a nationwide survey of labelled reference centre networks
in France. Eur J Cancer. 2016;58:90-96.
10 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.
11 Anneberg M, Svane H, Fryzek J, et al. The Epidemiology of
Desmoid Tumors in Denmark. Cancer Epidemiology. 2022; 77:1-7.
doi.org/10.1016/j.canep.2022.102114.
12 Orphanet Report Series: Rare Diseases collection. Prevalence
and incidence of rare diseases: bibliographic data. Number 1,
January 2022. Accessed April 28, 2022.
https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf.
13 The Desmoid Tumor Working Group. The management of desmoid
tumors: a joint global evidence-based consensus guideline approach
for adult and pediatric patients. Accessed April 10, 2022.
https://dtrf.org/wp-content/uploads/2020/02/Desmoid_Paper_2018_A4_RL_Web300-1.pdf.
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