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 OGSIVEO™ (nirogacestat), an oral gamma secretase
inhibitor, for the treatment of adult patients with progressing
desmoid tumors who require systemic treatment.1 The FDA previously
granted breakthrough therapy, fast track and orphan drug
designations to nirogacestat for the treatment of desmoid
tumors.
“Our team is honored to deliver the first FDA-approved therapy
for patients with desmoid tumors. This community has been waiting
for an effective treatment that not only shrinks their tumors but
also significantly improves pain, which is the most debilitating
symptom reported by people living with desmoid tumors,” said Saqib
Islam, Chief Executive Officer of SpringWorks. "We are pleased with
the broad label, which includes all progressing adult patients and
specifically references improvement in pain, and believe OGSIVEO
has the potential to become the new standard of care for people
living with these devastating tumors. This is a watershed moment
for the desmoid tumor community and we would like to extend our
gratitude to the patients, families, investigators, and advocacy
groups involved in the journey to making OGSIVEO available in the
U.S.”
Desmoid tumors are locally aggressive and invasive
soft-tissue tumors that can lead to substantial morbidity.2,3
In addition, when vital structures are impacted, desmoid tumors can
be life-threatening.3 Although they do not metastasize, desmoid
tumors are often refractory to existing off-label systemic
therapies and associated with recurrence rates of up to 77%
following surgical resection.4-6 Desmoid tumor experts and
treatment guidelines now recommend systemic therapies as first-line
intervention instead of surgery for most tumor locations requiring
treatment.6
“Desmoid tumors can have a significant impact on people’s lives
and are difficult to manage due to their invasive nature and high
rates of recurrence. OGSIVEO is a highly innovative therapy with
efficacy data demonstrating both meaningful antitumor activity and
a significant improvement in desmoid tumor symptoms,” said Mrinal
M. Gounder, M.D., sarcoma medical oncologist at Memorial Sloan
Kettering Cancer Center (MSK) in New York City and an investigator
in the Phase 3 DeFi trial. “As a treating physician, it was
encouraging to see in the DeFi trial that OGSIVEO achieved
statistically significant and clinically meaningful improvements
across the primary and all key secondary endpoints, while also
having a manageable safety profile. This approval represents an
important therapeutic advance for patients.”
The FDA approval of OGSIVEO is based on the results from the
Phase 3 DeFi trial, which were published in the March 9, 2023
edition of the New England Journal of Medicine.7 OGSIVEO met
the primary endpoint of improving progression-free survival (PFS),
demonstrating a statistically significant improvement over placebo
with a 71% reduction in the risk of disease progression (hazard
ratio (HR) = 0.29 (95% CI: 0.15, 0.55); p< 0.001). Median PFS
was not reached in the OGSIVEO arm and was 15.1 months in the
placebo arm. Confirmed objective response rate (ORR) based on
RECIST v1.1 was 41% with OGSIVEO versus 8% with placebo
(p<0.001); the complete response rate was 7% in the OGSIVEO arm
and 0% in the placebo arm. The median time to first response was
5.6 months with OGSIVEO and 11.1 months with placebo.7 PFS and
ORR improvements were in favor of OGSIVEO regardless of baseline
characteristics including sex, tumor location, tumor focality,
treatment status, previous treatments, mutational status, and
history of familial adenomatous polyposis.7,8 OGSIVEO also
demonstrated early and sustained improvements in patient-reported
outcomes (PROs), including pain (p<0.001), desmoid
tumor-specific symptoms (p<0.001), physical/role functioning
(p<0.001), and overall health-related quality of life
(p≤0.01).7
OGSIVEO exhibited a manageable safety and tolerability profile.
The most common adverse events (>15%) reported in patients
receiving OGSIVEO were diarrhea, ovarian toxicity, rash, nausea,
fatigue, stomatitis, headache, abdominal pain, cough, alopecia,
upper respiratory tract infection, and dyspnea. Please see
Important Safety Information below, including Warnings &
Precautions relating to diarrhea, ovarian toxicity, hepatotoxicity,
non-melanoma skin cancers, electrolyte abnormalities, and
embryo-fetal toxicity.1
“Today is an extraordinary day for the desmoid tumor community.
This approval is the culmination of a collaborative effort between
the patient community, academia and the biopharmaceutical industry,
who worked together with tenacity and persistence to advance
promising science,” said Jeanne Whiting, Executive Director
Emeritus and Co-Founder of the Desmoid Tumor Research Foundation.
“Our hope is that patients and their families will benefit from
greater awareness of desmoid tumors, faster diagnoses, and better
outcomes now that there is an approved and effective treatment.”
SpringWorks is dedicated to helping patients with desmoid tumors
access OGSIVEO and to providing support throughout their treatment
journey. As part of this commitment, the Company is introducing
SpringWorks CareConnections™, a comprehensive patient support
program that offers personalized services to eligible OGSIVEO
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.
OGSIVEO will be available to order through a specialty pharmacy
and specialty distributor network in the United States within five
to ten business days. SpringWorks expects to file a Marketing
Authorisation Application for OGSIVEO in desmoid tumors with the
European Medicines Agency in the first half of 2024.
Conference Call and Webcast Information
SpringWorks will host a conference call and webcast to discuss
the FDA approval of OGSIVEO on Tuesday, November 28, 2023, at 8:00
a.m. ET. To join the live webcast and view the corresponding
slides, please click here. To access the live call by phone, please
pre-register for the call here. Once registration is complete,
participants will be provided with a dial-in number and conference
code to access the call. A replay of the webcast will be available
for a limited time following the event on the Investors and Media
section of the Company’s website at
https://ir.springworkstx.com.
About OGSIVEO™
(nirogacestat)
OGSIVEO™ (nirogacestat) is an oral, selective, small molecule
gamma secretase inhibitor approved in the United States for the
treatment of adult patients with progressing desmoid tumors who
require systemic treatment.
OGSIVEO is not approved for the treatment of any other
indication in the United States, or for any indication in any other
jurisdiction by any other health authority.
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.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
- Diarrhea: Diarrhea occurred in 84% of patients
treated with OGSIVEO. Grade 3 events occurred in 16% of patients.
Monitor patients and manage using antidiarrheal medications. Modify
dose as recommended.
- Ovarian Toxicity: Female reproductive function
and fertility may be impaired in patients treated with OGSIVEO.
Impact on fertility may depend on factors like duration of therapy
and state of gonadal function at time of treatment. Long-term
effects on fertility have not been established. Advise patients on
the potential risks for ovarian toxicity before initiating
treatment. Monitor patients for changes in menstrual cycle
regularity or the development of symptoms of estrogen deficiency,
including hot flashes, night sweats, and vaginal dryness.
- Hepatotoxicity: ALT or AST elevations occurred
in 30% and 33% of patients, respectively. Grade 3 ALT or AST
elevations (>5 × ULN) occurred in 6% and 2.9% of patients.
Monitor liver function tests regularly and modify dose as
recommended.
- Non-Melanoma Skin Cancers: New cutaneous
squamous cell carcinoma and basal cell carcinoma occurred in 2.9%
and 1.4% of patients, respectively. Perform dermatologic
evaluations prior to initiation of OGSIVEO and routinely during
treatment.
- Electrolyte Abnormalities: Decreased phosphate
(65%) and potassium (22%) occurred in OGSIVEO-treated patients.
Phosphate <2 mg/dL occurred in 20% of patients. Grade 3
decreased potassium occurred in 1.4% of patients. Monitor phosphate
and potassium levels regularly and supplement as necessary. Modify
dose as recommended.
- Embryo-Fetal Toxicity: Oral administration of
nirogacestat to pregnant rats during the period of organogenesis
resulted in embryo-fetal toxicity at maternal exposures below human
exposure at the recommended dose of 150 mg twice daily. Advise
pregnant women of the potential risk to a fetus. Advise females and
males of reproductive potential to use effective contraception
during treatment with OGSIVEO and for 1 week after the last
dose.
ADVERSE REACTIONS
- The most common (≥15%) adverse reactions were diarrhea, ovarian
toxicity, rash, nausea, fatigue, stomatitis, headache, abdominal
pain, cough, alopecia, upper respiratory tract infection, and
dyspnea.
- Serious adverse reactions occurring in ≥2% of patients were
ovarian toxicity (4%).
- The most common laboratory abnormalities (≥15%) were decreased
phosphate, increased urine glucose, increased urine protein,
increased AST, increased ALT, and decreased potassium.
DRUG INTERACTIONS
- CYP3A Inhibitors and Inducers: Avoid
concomitant use with strong or moderate CYP3A inhibitors (including
grapefruit products, Seville oranges, and starfruit) and strong or
moderate CYP3A inducers.
- Gastric Acid Reducing Agents: Avoid
concomitant use with proton pump inhibitors and H2 blockers. If
concomitant use cannot be avoided, OGSIVEO can be staggered with
antacids (e.g., administer OGSIVEO 2 hours before or 2 hours after
antacid use).
- Consult the full Prescribing Information prior to and during
treatment for important drug interactions.
To report suspected adverse reactions, contact SpringWorks
Therapeutics at 1-888-400-SWTX (1-888-400-7989) or FDA at
1-800-FDA-1088 or www.fda.gov/medwatch.
Please see full Prescribing Information for OGSIVEO for more
information.
About Desmoid Tumors
Desmoid tumors (sometimes referred to as aggressive
fibromatosis, or desmoid fibromatosis) 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.2,3
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.4,9 It is estimated that there are
1,000-1,650 new cases diagnosed per year in the United
States.9-11
Although they do not metastasize, desmoid tumors are associated
with recurrence rates of up to 77% after surgical
resection.4-6 Desmoid tumor experts and treatment guidelines
now recommend systemic therapies as first-line intervention instead
of surgery for most tumor locations requiring treatment.6
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.
Founded in 2017, SpringWorks has a diversified targeted oncology
pipeline spanning solid tumors and hematological cancers, including
clinical trials in rare tumor types and highly prevalent,
genetically defined cancers. OGSIVEO™, approved in the United
States for the treatment of adult patients with progressing desmoid
tumors who require systemic treatment, is SpringWorks’ first
FDA-approved therapy. SpringWorks’ strategic approach and
operational excellence in clinical development have enabled it to
rapidly advance its lead product candidates into late-stage trials
and enter into multiple collaborations 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 and
commercialization plans, our preclinical and clinical results, the
potential for OGSIVEO to become an important new treatment for
patients with desmoid tumors, the potential for a Marketing
Authorisation Application for nirogacestat, as well as statements
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 of our commercialization efforts with respect to
OGSIVEO, (ii) our limited experience as a commercial company, (iii)
our ability to obtain or maintain adequate coverage and
reimbursement for OGSIVEO, (iv) the success and timing of our
product development activities, including the initiation and
completion of SpringWorks’ clinical trials, (v) our expectations
regarding the potential clinical benefit of OGSIVEO for patients
with desmoid tumors, (vi) the potential for OGSIVEO to become the
new standard of care for patients with desmoid tumors, (vii) our
expectations concerning the market potential for OGSIVEO, (viii)
our expectations regarding when OGSIVEO will be available, (ix) 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, (x) the success and
timing of our collaboration partners’ ongoing and planned clinical
trials, (xi) 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), the European Medicines
Agency (EMA) and other regulatory authorities, investigational
review boards at clinical trial sites and publication review
bodies; (xii) whether FDA, EMA or other regulatory authorities will
require additional information or further studies, or may fail or
refuse to approve or may delay approval of our product candidates,
(xiii) our ability to obtain regulatory approval of any of our
product candidates or maintain regulatory approvals granted for our
products, (xiv) our plans to research, discover and develop
additional product candidates, (xv) our ability to enter into
collaborations for the development of new product candidates, (xvi)
our ability to establish and maintain manufacturing capabilities,
and our and our collaboration partners’ abilities to manufacture
our products and product candidates and scale production, (xvii)
our ability to maintain adequate patent protection and successfully
enforce patent claims against third parties, and (xviii) 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 September 30, 2023, as well as
discussions of potential risks, uncertainties and other important
factors in SpringWorks’ other filings with the Securities and
Exchange Commission.
Contacts:
Kim DiamondVice President, Communications and Investor
RelationsPhone: 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 OGSIVEO. Prescribing Information. SpringWorks Therapeutics,
Inc.
2 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.
3 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.
4 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.
5 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.
6 Gronchi A, Kasper B, et al. Desmoid Tumor Working Group. The
management of desmoid tumours: a joint global consensus-based
guideline approach for adult and paediatric patients. Eur J Cancer.
2020;127:96-107. doi:10.1016/j.ejca.2019.11.013.
7 Gounder M, Ratan R, Alcindor T, et al. Nirogacestat, a
Gamma-Secretase Inhibitor for Desmoid Tumors. N Engl J Med.
2023;388:898-912. doi:10.1056/NEJMoa2210140.
8 Data on file. SpringWorks Therapeutics, Inc.
9 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.
10 Orphanet Report Series: Rare Diseases collection.
Prevalence and incidence of rare diseases: bibliographic data.
Number 1, January 2022. Accessed November 24, 2023.
https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf.
11 U.S. Department of Commerce. News Blog. U.S. population
estimated at 332,403,650 on Jan. 1, 2022. Accessed November 24,
2023.
https://www.commerce.gov/news/blog/2022/01/us-population-estimated-332403650-jan-1-2022#:~:
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