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 accepted the Company’s New Drug Application (NDA) for
mirdametinib, an investigational MEK inhibitor, for the treatment
of adult and pediatric patients with neurofibromatosis type
1-associated plexiform neurofibromas (NF1-PN). The NDA was granted
Priority Review and has been given a Prescription Drug User Fee Act
(PDUFA) action date of February 28, 2025. In addition, the FDA has
stated that it is not currently planning to hold an advisory
committee meeting to discuss the application. SpringWorks also
announced today that the European Medicines Agency (EMA) has
validated the Marketing Authorization Application (MAA) for
mirdametinib for the treatment of adult and pediatric patients with
NF1-PN. Mirdametinib has the potential to be the first approved
therapy for the treatment of adult patients and a best-in-class
therapy for children with NF1-PN.
“These significant milestones bring us closer to
our goal of delivering a transformative medicine to both adults and
children with NF1-PN in the U.S. and Europe,” said Saqib Islam,
Chief Executive Officer of SpringWorks. “People living with NF1-PN
are in need of new advances and we look forward to working with the
FDA and EMA during their review processes as we prepare to bring
our second medicine to patients suffering from devastating
diseases.”
The FDA grants Priority Review to applications for
medicines that offer, if approved, significant improvements over
available options or that provide a treatment option where no
adequate therapy currently exists. The FDA and the European
Commission have previously granted Orphan Drug designation for
mirdametinib for the treatment of NF1. The FDA has also granted
Fast Track designation for the treatment of patients ≥ 2 years of
age with NF1-PN that are progressing or causing significant
morbidity and Rare Pediatric Disease designation for the treatment
of NF1.
“Plexiform neurofibromas may sit next to or
surround vital organs and can cause serious medical complications
for patients. While progress has been made, there remains a
pressing need for more treatment options, particularly for adults
who currently have no approved therapy,” said Annette Bakker,
Ph.D., Chief Executive Officer of the Children’s Tumor Foundation
(CTF) and Board Chair of CTF Europe. “CTF is dedicated to deploying
its time, talent and funding towards accelerating the development
of new treatments. We congratulate our long-term partner
SpringWorks on this important milestone and we are thrilled that
patients in the United States and Europe could soon have a new
therapy available to them.”
Both submissions include data from the pivotal
Phase 2b ReNeu trial, which evaluated mirdametinib in patients ≥ 2
years of age with NF1-associated PN causing significant morbidity.
Results were presented in an oral presentation at the 2024 American
Society of Clinical Oncology Annual Meeting and demonstrated that
mirdametinib treatment resulted in robust objective response rates
confirmed by blinded independent central review, deep and durable
responses, improvement in pain and health-related quality of life
as well as a manageable safety profile across both the adult and
pediatric cohorts.1
About the ReNeu Trial
ReNeu (NCT03962543) is an ongoing, multi-center,
open-label Phase 2b trial evaluating the efficacy, safety, and
tolerability of mirdametinib in patients ≥ 2 years of age with an
inoperable NF1-associated PN causing significant morbidity. The
study enrolled 114 patients to receive mirdametinib at a dose of 2
mg/m2 twice daily (maximum dose of 4 mg twice daily) without regard
to food. Mirdametinib was administered orally in a 3-week on,
1-week off dosing schedule as either a capsule or dispersible
tablet. The primary endpoint is confirmed objective response rate
assessed by proportion of patients with ≥ 20% reduction in target
tumor volume on consecutive scans during the 24 cycle treatment
phase, as measured by MRI and assessed by blinded independent
central review. Secondary endpoints include safety and
tolerability, duration of response, and changes in patient reported
outcomes from baseline to Cycle 13. The treatment phase of the
trial is complete and results were presented at the 2024 American
Society of Clinical Oncology Annual Meeting. Patients who completed
the treatment phase were eligible to continue receiving treatment
in the optional long-term follow up portion of the study, which is
ongoing.
About NF1-PN
Neurofibromatosis type 1 (NF1) is a rare genetic
disorder that arises from loss-of-function variants in the NF1
gene, which encodes for neurofibromin, a key suppressor of the MAPK
pathway.2,3 NF1 is the most common form of neurofibromatosis, with
an estimated global birth incidence of approximately 1 in 2,500
individuals, and approximately 100,000 patients living with NF1 in
the United States.4,5 The clinical course of NF1 is heterogeneous
and manifests as a variety of symptoms across numerous organ
systems, including abnormal skin pigmentation, skeletal
deformities, tumor growth, and neurological complications such as
cognitive impairment.6 Patients with NF1 have an 8 to 15-year mean
reduction in their life expectancy compared to the general
population.3
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.7,8 NF1-PNs are most
often diagnosed in the first two decades of life.7 These tumors can
be aggressive and are associated with clinically significant
morbidities; typically, they grow more rapidly during
childhood.9,10 Surgical removal of these tumors is challenging due
to the infiltrative tumor growth pattern along nerves and can lead
to permanent nerve damage and disfigurement.11 MEK inhibitors have
emerged as a validated class of treatment for NF1-PN.12
About Mirdametinib
Mirdametinib is a potent, oral, CNS-penetrant,
allosteric small molecule MEK inhibitor in development as a
monotherapy treatment for neurofibromatosis type 1-associated
plexiform neurofibromas (NF1-PN) and low-grade glioma (LGG), and as
a combination therapy for the treatment of several subsets of
biomarker-defined metastatic solid tumors. Mirdametinib is an
investigational drug for which safety and efficacy have not been
established.
Mirdametinib is designed to inhibit MEK1 and MEK2,
which occupy pivotal positions in the MAPK pathway. The MAPK
pathway is a key signaling network that regulates cell growth and
survival and plays a central role in multiple cancers and rare
diseases when genetically altered.
The FDA and the European Commission have granted
Orphan Drug designation for mirdametinib for the treatment of NF1.
The FDA has also granted Fast Track designation for the treatment
of patients ≥ 2 years of age with NF1-PN that are progressing or
causing significant morbidity and Rare Pediatric Disease
designation for the treatment of NF1.
About SpringWorks Therapeutics
SpringWorks is a commercial-stage biopharmaceutical
company applying a precision medicine approach to developing and
delivering life-changing medicines for people with severe rare
diseases and cancer. OGSIVEO® (nirogacestat), approved in the
United States for the treatment of adult patients with progressing
desmoid tumors who require systemic treatment, is the Company’s
first FDA-approved therapy. SpringWorks also has a diversified
targeted therapy pipeline spanning solid tumors and hematological
cancers, with programs ranging from preclinical development through
advanced clinical trials. In addition to its wholly owned programs,
SpringWorks has also entered into multiple collaborations with
innovators in industry and academia to unlock the full potential
for its portfolio and create more solutions for patients in
need.
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 and
commercialization plans, our preclinical and clinical results, 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 presentation 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
presentation, including, without limitation, risks relating to: (i)
whether the preclinical and clinical results of the mirdametinib
studies will meet the regulatory requirements for an approval by
the FDA or by the EMA of mirdametinib for the treatment of
pediatric and adult patients with NF1-PN, (ii) interactions with
the FDA or EMA, including reviews and inspections, the timing
related thereto and the outcome thereof, (iii) our expectations
regarding the potential therapeutic benefits, safety profile and
effectiveness of mirdametinib for patients with NF1-PN (iv) whether
the NDA or the MAA will be approved, and (v) if approved, whether
mirdametinib will be commercially successful.
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, 2024 as well as
discussions of potential risks, uncertainties and other important
factors in SpringWorks’ subsequent filings.
Contacts
Kim DiamondVice President, Communications and Investor
RelationsPhone: 203-561-1646 Email: kdiamond@springworkstx.com
Samantha Hilson SandlerSenior Director, Investor RelationsPhone:
203-461-5501Email: samantha.sandler@springworkstx.com
References
- Moertel C, et al., ReNeu: A pivotal
phase 2b trial of mirdametinib in children and adults with
neurofibromatosis type 1 (NF1)-associated symptomatic inoperable
plexiform neurofibroma (PN). Journal of Clinical Oncology 42,
3016-3016(2024). DOI:10.1200/JCO.2024.42.16_suppl.3016. The
American Society of Clinical Oncology Abstract 3016. May 2024.
- 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.
- CTF: Children’s Tumor Foundation. New
and Improved: The way to talk about NF. Press release. May 9, 2023.
Accessed February 2, 2024.
- 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. Published
2023 Sep 14. doi:10.1186/s13023-023-02911-2.
- 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. Journal of
Clinical Oncology. 2021;JCO.20.02220.doi.org/10.
1200/JCO.20.02220.
- Prada: Prada CE, Rangwala FA, Martin
LJ, et al. Pediatric plexiform neurofibromas: impact on morbidity
and mortality in neurofibromatosis type 1. J Pediatr.
2012;160(3):461-467.
- Miller: Miller DT, et al. Health
Supervision for Children With Neurofibromatosis Type 1. Pediatrics.
2019;143(5):e20190660.
- 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.
- 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.
- 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.
SpringWorks Therapeutics (NASDAQ:SWTX)
過去 株価チャート
から 8 2024 まで 9 2024
SpringWorks Therapeutics (NASDAQ:SWTX)
過去 株価チャート
から 9 2023 まで 9 2024