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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 OR 15(d)
of The Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
December 16, 2024
Edgewise Therapeutics, Inc.
(Exact name of registrant as specified in its
charter)
| Delaware |
|
001-40236 |
|
82-1725586 |
(State or other jurisdiction
of incorporation)
|
|
(Commission
File Number)
|
|
(IRS
Employer
Identification No.) |
1715
38th St.
Boulder,
CO 80301
(Address of principal executive offices) (Zip Code)
(720)
262-7002
(Registrant’s telephone number, including
area code)
Not Applicable
(Former name or former address, if changed
since last report.)
Check the appropriate box below if the Form 8-K filing is intended
to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2.
below):
| ¨ |
Written communications pursuant to Rule 425 under the
Securities Act (17 CFR 230.425) |
| |
|
| ¨ |
Soliciting material pursuant to Rule 14a-12 under the
Exchange Act (17 CFR 240.14a-12) |
| |
|
| ¨ |
Pre-commencement communications pursuant to Rule 14d-2(b)
under the Exchange Act (17 CFR 240.14d-2(b)) |
| |
|
| ¨ |
Pre-commencement communications pursuant to Rule 13e-4(c)
under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title
of each class |
|
Trading
Symbol(s) |
|
Name
of each exchange on which registered |
| Common
Stock, $0.0001 par value per share |
|
EWTX |
|
The
Nasdaq Global Select Market |
Indicate by check mark whether the registrant is an emerging growth
company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange
Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company x
If an emerging growth company, indicate by check mark if the registrant
has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant
to Section 13(a) of the Exchange Act.
Item 7.01 Regulation FD Disclosure.
On December 16, 2024, Edgewise Therapeutics, Inc. (the “Company”)
issued a press release announcing positive topline results from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular
dystrophy. The press release is attached hereto as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein
by reference.
On
December 16, 2024, the Company also posted a presentation to its Investor Relations website (https://investors.edgewisetx.com).
A copy of the presentation is furnished as Exhibit 99.2 to this Current Report on Form 8-K and is incorporated herein by reference.
The information in this Item 7.01, including Exhibits 99.1 and 99.2
attached hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended,
or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities
Act of 1933, as amended, except as expressly set forth by specific reference in such filing.
Item 8.01 Other Events.
On December 16, 2024, the Company announced positive topline results
from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy.
Overview of CANYON and Clinical Results
CANYON,
the largest interventional Becker muscular dystrophy trial, is a Phase 2, double-blind, randomized, placebo-controlled study to
investigate the effect of sevasemten on the safety, pharmacokinetics, biomarkers, and functional measures of participants (NCT05291091).
The trial was not powered for the functional endpoints. Forty adults and 29 adolescents with Becker muscular dystrophy were enrolled.
This study had a 4-week screening period, a 12-month treatment period, followed by a 4-week follow-up period. The adult participants were
randomized to sevasemten or placebo in a 3:1 ratio. The adolescent participants were randomized in a 2:1 ratio to sevasemten or placebo
and were assessed for safety and tolerability. The data analysis included the complete adult safety population of 40 individuals. There
was a notable imbalance between adult participants in the sevasemten and placebo groups with the sevasemten group having more advanced
disease at baseline based on all functional measures and MRI.
Primary
Endpoint: The primary endpoint to assess the efficacy of sevasemten compared to placebo was change from baseline in creatine
kinase (“CK”) over the treatment period for adults. The results demonstrated a significant change from baseline in
CK in the sevasemten-treated group (difference vs. placebo, 28% average decrease over months 6 through 12; p=0.02).
Key
Secondary Endpoint: The key secondary endpoint was the change from baseline in NSAA total score in adults at month 12. North
Star Ambulatory Assessment (“NSAA”) is a scale commonly used to rate motor function. The between-group difference was
1.1 points, favoring sevasemten; p=0.16 across all adult participants. NSAA remained stable over time in the sevasemten treatment group,
similar to the observations in the ARCH study. Further, while the placebo group was small in number (n=12), NSAA declined similarly to
that observed in previous natural history studies.1,2,3
Other
Secondary Endpoints: Plasma fast skeletal muscle troponin I (“TNNI2”) decreased 77% from baseline in the
sevasemten-treated group compared to placebo, averaged over months 6 through 12 in adults; p<0.001.
The 10-meter walk/run, 4-stair climb and 100-meter timed test showed
trends towards improvement, compared to placebo. The Company continues to evaluate additional secondary and exploratory endpoints.
Safety
and Tolerability: Sevasemten was well-tolerated, and no new safety concerns were identified.
CANYON
Implications to GRAND CANYON: The functional observations from the CANYON study support that the GRAND CANYON pivotal cohort’s
primary endpoint is powered at >95% to demonstrate a statistically significant NSAA difference at 18 months.
References
[1] Bello L, et al. Functional changes in Becker
muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439.
[2] van de Velde NM, et al. Selection approach
to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522.
doi: 10.1212/WNL.0000000000012233.
[3] De Wel B, et al. Lessons for future clinical
trials in adults with Becker muscular dystrophy: disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported
outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282. Online ahead of print.
Cautionary Note Regarding Forward-Looking Statements
This Form 8-K contains forward-looking statements as that
term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934.
Statements in this Form 8-K that are not purely historical are forward-looking statements. Such forward-looking statements
include, among other things, statements regarding Edgewise’s cash runway; the potential of, and expectations regarding
Edgewise’s expectations relating to its clinical trials and clinical development of sevasemten (EDG-5506), including
statements regarding the number of individuals to be recruited, timing of completion of recruitment and over-enrollment of the GRAND
CANYON trial; statements regarding the potential of, and expectations regarding, Edgewise’s product candidates and programs,
including sevasemten and EDG-7500; statements regarding Edgewise’s milestones; statements regarding whether data from the
GRAND CANYON trial could support a marketing application; statements regarding the Company’s plans to engage the FDA and
European Medicines Agency; statements about sevasemten being the potential first treatment for Becker; statements about the
submission of results of the CANYON trial for publication at a future medical congress; and statements by Edgewise’s chief
executive officer and chief medical officer and Craig M. McDonald, M.D. Words such as “believes,”
“anticipates,” “plans,” “expects,” “intends,” “will,”
“goal,” “potential” and similar expressions are intended to identify forward-looking statements. The
forward-looking statements contained herein are based upon Edgewise’s current expectations and involve assumptions that may
never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking
statements due to numerous risks and uncertainties, including but not limited to: risks associated with the process of discovering,
developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical
stage company, including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s
ability to develop, initiate or complete clinical trials for, obtain approvals for and commercialize any of its product candidates;
Edgewise’s ability to take advantage of potential benefits associated with designations granted by FDA and/or to maintain
qualifications for applicable designations over time; the timing, progress and results of clinical trials for sevasemten and
EDG-7500; Edgewise’s ability to enroll and maintain patients in clinical trials; Edgewise’s ability to raise any
additional funding it will need to continue to pursue its business and product development plans; the timing, scope and likelihood
of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary,
topline or expected results, including that the primary endpoint of the GRAND CANYON trial (change from baseline in NSAA) will be
met even though it was not met as a secondary endpoint in the CANYON trial; the potential that the outcome of preclinical testing
and early clinical trials, including the results from the CANYON trial, may not be predictive of the success of later clinical
trials, including that the trends from the CANYON trial will also be seen, and will be statistically significant, in the GRAND
CANYON trial; Edgewise may gain further insights from its analysis of the CANYON trial results over time, including
Edgewise’s ongoing evaluation of additional secondary and exploratory endpoints; Edgewise’s ability to develop a
proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and
marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key
scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third
parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; general
economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the
section entitled “Risk Factors” in documents that Edgewise files from time to time with the U.S. Securities and Exchange
Commission. These forward-looking statements are made as of the date of this Form 8-K, and Edgewise assumes no obligation to
update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the
forward-looking statements, except as required by law.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits.
SIGNATURES
Pursuant to the requirements of the Securities
Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| EDGEWISE THERAPEUTICS, INC. |
|
| |
|
|
| By: |
/s/ Behrad Derakhshan, Ph.D. |
|
| |
Behrad Derakhshan, Ph.D. |
|
| |
Chief Business Officer |
|
Date: December 16, 2024
Exhibit 99.1
Edgewise Therapeutics Announces Positive Topline
Results from the CANYON Phase 2 Trial of Sevasemten in Individuals with Becker Muscular Dystrophy (Becker)
– Trial met primary endpoint of reduction
in circulating levels of creatine kinase (CK), a biomarker associated with skeletal muscle damage, in the largest Becker interventional
trial to date –
– On the key secondary endpoint, sevasemten-treated
patients showed stabilization of North Star Ambulatory Assessment (NSAA) with a trend towards improvement at 12 months compared to placebo
–
– Sevasemten was well-tolerated and
no new safety concerns were observed –
– Edgewise leadership to discuss CANYON
findings on Monday, December 16 at 8:30 a.m. Eastern Time at a virtual investor event –
Boulder, Colo., (December 16, 2024) – Edgewise
Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive topline results
from the Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered first-in-class
fast skeletal myosin inhibitor designed to protect muscle against contraction-induced damage in muscular dystrophies. The trial met its
primary endpoint of change from baseline in CK. CANYON is the largest interventional trial to date in Becker and the first to achieve
its primary endpoint.
NSAA, the key secondary endpoint of function, showed a trend towards
improvement over time in the sevasemten-treated group. Plasma fast skeletal muscle troponin I (TNNI2), a target-specific biomarker of
fast skeletal muscle damage, showed a significant reduction, compared to placebo. Additional functional measures, including the 10-meter
walk/run, 4-stair climb and 100-meter timed test, showed trends towards improvement compared to placebo. Notably, the treatment population
had more advanced disease than placebo.
Sevasemten was well-tolerated, and no new safety concerns were observed
in either the adult or adolescent patient populations. Ninety-nine percent of eligible participants from CANYON and other sevasemten trials
in Becker have enrolled in MESA, the ongoing open label extension trial.
“Becker muscular dystrophy is a devastating neuromuscular disease
characterized by rapid progression once functional decline begins. This landmark study presents compelling biomarker data and promising
signals that suggest the potential for functional stabilization with administration of sevasemten,” said Craig M. McDonald, M.D.,
Distinguished Professor and Chair at the UC Davis Health Department of Physical Medicine and Rehabilitation, and a Principal Investigator
in CANYON and GRAND CANYON. “Becker has no approved therapies. I look forward to the results of the GRAND CANYON pivotal cohort
with the hope of bringing the first treatment option to this patient population.”
“We are very encouraged by the CANYON results
in Becker and the potential of this novel muscle-targeted therapeutic,” said Joanne Donovan, Ph.D., M.D., Chief Medical Officer,
Edgewise. “This confirmed our previous observations in the ARCH study of significant decreases in biomarkers of muscle damage and
similarly we are seeing evidence of preservation of function in Becker patients.”
The Company is on track to complete recruitment
in the GRAND CANYON cohort by the first quarter of 2025. Based on these positive Phase 2 results, the Company plans to engage the U.S.
Food and Drug Administration (FDA) and European Medicines Agency about marketing authorization filing strategies for sevasemten in Becker.
The Company intends to submit the complete results of the CANYON study
for publication at a future medical congress.
Overview of CANYON and Clinical Results
CANYON, the largest interventional Becker trial, is a Phase
2, double-blind, randomized, placebo-controlled study to investigate the effect of sevasemten on the safety, pharmacokinetics, biomarkers,
and functional measures of participants (NCT05291091). The trial was not powered for the functional endpoints. Forty adults and
29 adolescents with Becker muscular dystrophy were enrolled. This study had a 4-week screening period, a 12-month treatment period, followed
by a 4-week follow-up period. The adult participants were randomized to sevasemten or placebo in a 3:1 ratio. The adolescent participants
were randomized in a 2:1 ratio to sevasemten or placebo and were assessed for safety and tolerability. The data analysis included the
complete adult safety population of 40 individuals. There was a notable imbalance between adult participants in the sevasemten and placebo
groups with the sevasemten group having more advanced disease at baseline based on all functional measures and MRI.
Primary Endpoint: The primary endpoint to assess the efficacy
of sevasemten compared to placebo was change from baseline in CK over the treatment period for adults. The results demonstrated a significant
change from baseline in CK in the sevasemten-treated group (difference vs. placebo, 28% average decrease over months 6 through 12; p=0.02).
Key Secondary Endpoint: The key secondary endpoint was the change
from baseline in NSAA total score in adults at month 12. NSAA is a scale commonly used to rate motor function. The between-group difference
was 1.1 points, favoring sevasemten; p=0.16 across all adult participants. NSAA remained stable over time in the sevasemten treatment
group, similar to the observations in the ARCH study. Further, while the placebo group was small in number (n=12), NSAA declined similarly
to that observed in previous natural history studies.1,2,3
Other Secondary Endpoints: Plasma TNNI2 decreased 77% from baseline
in the sevasemten-treated group compared to placebo, averaged over months 6 through 12 in adults; p<0.001.
The 10-meter walk/run, 4-stair climb and 100-meter timed test showed
trends towards improvement, compared to placebo. The Company continues to evaluate additional secondary and exploratory endpoints.
Safety and Tolerability: Sevasemten was well-tolerated, and
no new safety concerns were identified.
CANYON Implications to GRAND CANYON: The functional observations
from the CANYON study support that the GRAND CANYON pivotal cohort’s primary endpoint is powered at >95% to demonstrate a statistically
significant NSAA difference at 18 months.
MESA, open label extension trial in adults with Becker: The
Company is advancing MESA, an open-label extension trial to assess the long-term effect of sevasemten in individuals with Becker. MESA
provides continued access to sevasemten to participants who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE.
To date, 99% of eligible participants completing these trials have enrolled in MESA.
GRAND CANYON, a global pivotal cohort in Becker: GRAND CANYON,
an expansion of the CANYON placebo-controlled trial, is a multi-center, randomized, double-blind, placebo-controlled cohort to evaluate
the safety and efficacy of sevasemten in adults with Becker. The primary endpoint of GRAND CANYON is change from baseline in NSAA at
18 months. In addition, other functional assessments, biomarkers of muscle damage, MRI, patient-reported outcomes and safety will be
assessed. GRAND CANYON is an 18-month cohort anticipated to recruit approximately 120 individuals with Becker. Data from GRAND CANYON,
if positive, could support a marketing application. To learn more, go to clinicaltrials.gov (NCT05291091).
Sevasemten has achieved notable regulatory milestones by securing FDA
Orphan Drug Designation for the treatment of Becker and Duchenne, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne,
and Fast Track designations for the treatment of Becker and Duchenne. Further, sevasemten secured the EMA Orphan Drug Designations for
the treatment of Becker and Duchenne.
Upcoming CANYON Data Presentations:
Virtual Investor Event
Members of the Edgewise management team will
hold a live webcast on Monday, December 16, at 8:30 a.m. ET to discuss the CANYON data, and will be joined by Dr. McDonald,
who will share his perspective of sevasemten and Becker. An accompanying slide presentation will also be available. To register for the
live webcast and replay, please visit the Edgewise events page.
Patient Community Webinar
Members of Edgewise management will hold a community
webinar on Wednesday, December 18, 2024, at 1 p.m. ET to discuss these data and the GRAND CANYON pivotal study. To register
for the community webinar, please click here.
About Becker Muscular Dystrophy
Becker is a rare, genetic, life-shortening, debilitating
and degenerative neuromuscular disorder. The disease predominantly affects males and imposes significant physical, emotional, financial,
and social impacts on the individual and their caregivers. Individuals with Becker experience contraction-induced muscle damage, which
is the primary driver of muscle loss and impaired motor function in muscular dystrophies. Functional decline can begin at any age, and
once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual’s life. Some individuals
living with Becker experience heart failure from cardiomyopathy, which may result in heart transplantation or early death. Currently,
there is no cure for Becker; early and long-term multidisciplinary care is critical for optimized disease management. There is a great
need for more Becker-specific scientific research, clinical programs, and treatment guidelines to improve management of this disease.
To learn more about Becker, go to https://beckermusculardystrophy.com/
About Sevasemten (EDG-5506) for Becker and
Duchenne Muscular Dystrophies
Sevasemten is an orally administered first-in-class fast skeletal myosin
inhibitor designed to protect muscle against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne.
Sevasemten presents a novel mechanism of action designed to selectively
limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By minimizing the progressive muscle damage
that leads to functional impairment, sevasemten has the potential to benefit a broad range of patients suffering from debilitating neuromuscular
disorders. Its unique mechanism of action provides the potential to establish sevasemten as a foundational therapy in dystrophinopathies,
either as a single agent therapy or in combination with available therapies and those in development.
Sevasemten is being studied in the Phase 2 CANYON
study with a pivotal cohort GRAND CANYON in adults and adolescents with Becker muscular dystrophy. Sevasemten is also being studied in
the ongoing Phase 2 trials, LYNX and FOX, in children and adolescents with Duchenne muscular dystrophy.
For more information on Edgewise’s clinical
trials https://edgewisetx.com/clinical-trials.
About Edgewise Therapeutics
Edgewise Therapeutics is a leading muscle disease biopharmaceutical
company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company’s deep expertise in
muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered first-in-class fast skeletal
myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator
for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development.
The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases.
To learn more, go to: www.edgewisetx.com or follow us on LinkedIn , X, Facebook and Instagram.
References
[1] Bello L, et al. Functional changes in Becker
muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439.
[2] van de Velde NM, et al. Selection approach
to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522.
doi: 10.1212/WNL.0000000000012233.
[3] De Wel B, et al. Lessons for future clinical
trials in adults with Becker muscular dystrophy: disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported
outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282. Online ahead of print.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as that term
is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in
this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other
things, statements regarding the potential of, and expectations regarding Edgewise’s expectations relating to its clinical trials
and clinical development of sevasemten, including statements regarding the number of individuals to be recruited, timing of completion
of recruitment and over-enrollment of the GRAND CANYON trial; statements regarding the potential of, and expectations regarding, Edgewise’s
product candidates and programs, including sevasemten (EDG-5506) and EDG-7500; statements regarding Edgewise’s milestones; statements
regarding whether data from the GRAND CANYON trial could support a marketing application; statements regarding the Company’s plans
to engage the FDA and European Medicines Agency; statements about the submission of results of the CANYON trial for publication at a future
medical congress; and statements by Edgewise’s chief medical officer and Craig M. McDonald, M.D. Words such as “believes,”
“anticipates,” “plans,” “expects,” “intends,” “will,” “goal,”
“potential” and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained
herein are based upon Edgewise’s current expectations and involve assumptions that may never materialize or may prove to be incorrect.
Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties,
including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and
effective for use as human therapeutics and operating as an early clinical stage company, including the potential for Edgewise’s
product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete clinical trials for, obtain
approvals for and commercialize any of its product candidates; Edgewise’s ability to take advantage of potential benefits associated
with designations granted by FDA and/or to maintain qualifications for applicable designations over time; the timing, progress and results
of clinical trials for sevasemten and EDG-7500; Edgewise’s ability to enroll and maintain patients in clinical trials; Edgewise’s
ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope
and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim,
preliminary, topline or expected results, including that the primary endpoint of the GRAND CANYON trial (change from baseline in NSAA)
will be met even though it was not met as a secondary endpoint in the CANYON trial; the potential that the outcome of preclinical testing
and early clinical trials, including the results from the CANYON trial, may not be predictive of the success of later clinical trials,
including that the trends from the CANYON trial will also be seen, and will be statistically significant, in the GRAND CANYON trial; Edgewise
may gain further insights from its analysis of the CANYON trial results over time, including Edgewise’s ongoing evaluation of additional
secondary and exploratory endpoints; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of
product candidates; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market
opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in
which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property
protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and
additional risks may be found in the section entitled “Risk Factors” in documents that Edgewise files from time to time with
the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Edgewise
assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected
in the forward-looking statements, except as required by law.
###
Edgewise Contacts
Investors:
Behrad Derakhshan, Ph.D., Chief Business Officer
ir@edgewisetx.com
Media:
Maureen Franco, VP Corporate Communications
media@edgewisetx.com
Exhibit
99.2
| 1
©2024 Edgewise Therapeutics. All rights reserved.
Sevasemten: Topline Results from
the CANYON Phase 2 Trial in Becker
December 16, 2024 |
| 2
This presentation contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of
1934. Statements in this presentation that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things,
statements regarding the potential of, and expectations regarding Edgewise’s cash runway, Edgewise’s expectations relating to its clinical trials and clinical development of
sevasemten (EDG-5506) and EDG-7500, including statements regarding the number of individuals to be recruited, timing of completion of recruitment and over-enrollment of the GRAND CANYON trial; statements regarding the potential of, and expectations regarding, Edgewise’s product candidates and programs, including
sevasemten and EDG-7500; statements regarding Edgewise’s milestones; statements regarding whether data from the GRAND CANYON trial could support a marketing
application; statements regarding the Company’s plans to engage the FDA and European Medicines Agency; statements about sevasemten being the potential first
treatment for Becker; and statements by Edgewise’s chief executive officer and chief medical officer and Craig M. McDonald, M.D. Words such as “believes,” “anticipates,”
“plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained
herein are based upon Edgewise’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ
materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with the process
of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company, including
the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete clinical trials for, obtain approvals for and
commercialize any of its product candidates; Edgewise’s ability to take advantage of potential benefits associated with designations granted by FDA and/or to maintain
qualifications for applicable designations over time; the timing, progress and results of clinical trials for sevasemten and EDG-7500; Edgewise’s ability to enroll and maintain
patients in clinical trials; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope
and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results,
including that the primary endpoint of the GRAND CANYON trial (change from baseline in NSAA) will be met even though it was not met as a secondary endpoint in the
CANYON trial; the potential that the outcome of preclinical testing and early clinical trials, including the results from the CANYON trial, may not be predictive of the success
of later clinical trials, including that the trends from the CANYON trial will also be seen, and will be statistically significant, in the GRAND CANYON trial; Edgewise may gain
further insights from its analysis of the CANYON trial results over time, including Edgewise’s ongoing evaluation of additional secondary and exploratory endpoints;
Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and marketing
capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the
industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product
candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk
Factors” in documents that Edgewise files from time to time with the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date
of this presentation, and Edgewise assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those
projected in the forward-looking statements, except as required by law.
The trademarks included herein are the property of the owners thereof and are used for reference purposes only. Such use should not be construed as an endorsement of
such products.
Forward Looking Statement |
| Agenda
1. Introduction
2. Becker muscular dystrophy and sevasemten
3. Topline results: Phase 2 CANYON trial in Becker
4. Sevasemten future development plans
5. Clinical perspectives on the results
6. Closing remarks
7. Q & A
Distinguished Professor and Chair at the
UC Davis Health Department of Physical
Medicine and Rehabilitation
Dr. Kevin Koch
Dr. Craig M. McDonald
Chief Executive Officer
Dr. Joanne Donovan
Chief Medical Officer |
| Focused on muscle science
● Global leader in muscle disease therapeutic development
● Deep knowledge of integrated muscle physiology
● Novel & holistic therapeutic approach to protect muscle
Rapidly advancing portfolio
● Sevasemten pivotal program in muscular dystrophies including potential first
treatment for Becker
● Advancing EDG-7500 in oHCM, nHCM, and other potential indications
● Novel cardiometabolic targets in discovery
Unwavering patient commitment
● Mission-driven focus on unmet needs in severe muscle conditions
● Patients & families are critical voices in all development programs
Abbreviations: oHCM, obstructive hypertrophic cardiomyopathy, nHCM, non-obstructive hypertrophic cardiomyopathy |
| 5
The Evolution of Edgewise
2017 2020 2022 2023 2025
Founded as a
muscle platform
company
targeting
genetically driven
muscle diseases
EWTX completes IPO,
expands development programs
First program
targeting
muscular
dystrophies
Sevasemten, a fast
skeletal myosin
inhibitor, enters P1
in HVs and Becker
MUSCULAR DYSTROPHY PROGRAM MILESTONES
Sevasemten
enters P2 in
Duchenne
Initiated
GRAND CANYON
pivotal cohort
sevasemten in
Becker
2021 2024
EDG-7500
identified as
lead molecule
with unique
characteristics
EDG-7500
characterized
as a selective
cardiac
sarcomere
modulator
EDG-7500
enters the clinic;
continued
research on
novel
cardiometabolic
targets
Initiated
CIRRUS-HCM,
EDG-7500
P2 program
in oHCM
and nHCM
CARDIOVASCULAR PROGRAM MILESTONES
Identified a
differentiated set
of CV molecules as
part of skeletal
muscle counter-screen
CV program
initiated
Sevasemten
ARCH open label
24-month results
in Becker
Sevasemten,
P2 CANYON
12-month
results in
Becker
2019
Initiated Becker
ARCH open label
study with
sevasemten
Abbreviations: HVs, healthy volunteers, CV, cardiovascular, oHCM, obstructive hypertrophic cardiomyopathy, nHCM, non-obstructive hypertrophic cardiomyopathy |
| 6
ARCH, CANYON and the Evolution to GRAND CANYON Expedited the
Development Timeline for Sevasemten in Becker
• The Phase 2 CANYON study was originally designed as a dose-finding study to evaluate the effect of sevasemten on safety,
pharmacokinetics, biomarkers, and function in individuals with Becker
• 12-month data from the ARCH study supported the hypothesis that a reduction in contraction-induced muscle damage has
the potential to preserve and improve muscle function while preventing disease progression
• ARCH identified key factors, including the dose of sevasemten, to allow expansion of CANYON to include the GRAND
CANYON cohort as a potentially registrational cohort with NSAA as the primary endpoint
• GRAND CANYON is the first pivotal cohort of an investigational therapy for Becker. Data from GRAND CANYON, if positive,
could support a marketing application
10 mg PO daily
15 mg PO daily
Placebo
20 mg PO daily PIVOTAL Cohort 10 mg PO daily (18 mos.)
Placebo
10 mg PO daily (12 mos.)
Placebo
N=40
Original CANYON Design Expanded CANYON/GRAND CANYON Design
N=120
This strategic pivot significantly reduced development timelines to support a
potential approval of sevasemten for individuals living with Becker |
| Becker Muscular Dystrophy
and Sevasemten
Dr. Joanne Donovan |
| 8
I was told, ‘You’re lucky you don’t have Duchenne.’ It’s
frustrating that you live longer, but you are constantly
going downhill”
– Individual living with Becker
Our goal is to positively impact the course
of Becker muscular dystrophy
● Becker is a rare, genetic, life-shortening, debilitating
and degenerative neuromuscular disorder
● The disease predominately affects males and imposes
significant physical, emotional, financial and social
impacts on the individuals and their caregivers
● Individuals with Becker lose mobility, function and
independence in the prime of their lives
● There is currently no treatment for Becker |
| 9
Sevasemten is an investigational therapy that has not been approved for use in muscular dystrophies by any regulatory agency, as its safety and effectiveness
have not been established for the treatment of these diseases.
Abbreviations: CK, creatine kinase
Sevasemten: A First-in-Class Fast Myofiber (Type II) Myosin Inhibitor
Designed to Protect Against Contraction-Induced Muscle Injury
Collapsed sarcomere
Protected sarcomere
Progressive
disease pathology
Contraction-induced
muscle injury
Sevasemten
Prevention of
contraction-induced
muscle injury
Excessive
contraction
induced muscle
damage
Duchenne
Becker
Sevasemten Therapeutic Hypothesis |
| Abbreviations: CK, Creatine Kinase; TNNI2, Fast Skeletal Muscle Troponin I 10
Elevated Circulating Levels of Muscle Injury Biomarkers, including CK
and TNNI2, Indicate Ongoing Muscle Damage in Muscular Dystrophies
Contraction-Induced Muscle Injury in Muscular Dystrophies
Contraction induced muscle damage
causes excessive degeneration
Fast fibers are subsequently injured leading to release of muscle
injury biomarkers into the circulation
CK
CK
CK TNNI2
TNNI2
TNNI2
Circulating Levels of Muscle Injury Biomarkers Can be Measured to Determine Ongoing Muscle
Damage in Muscular Dystrophies |
| 11
PK, biomarkers and longer-term safety OLE Complete
Function (NSAA), PK, biomarkers and longer-term safety OLE Recruiting
GRASP Recruiting -01-002 24-Month, observational study
Abbreviations: LGMD, limb-girdle muscular dystrophy; NSAA, North Star ambulatory assessment; PK, pharmacokinetics
Our Commitment to Becker: Completed and Ongoing Clinical Trials
Phase 2 Becker
Pivotal Cohort Becker
(NCT05291091)
Becker Natural History Study
(NCT05257473)
Exercise challenge study OLE Complete Phase 2 Becker,
LGMD2i, & McArdle
Phase 1 Becker Open-label, safety, PK, function OLE Complete
Open-label long-term safety, biomarkers and functional measures Enrolling by invitation Becker Open Label Extension
Study (NCT06066580) |
| Topline Results |
| 13 A Phase 2 Multi-Center Study to Assess Sevasemten Safety and Effect on Biomarkers in Adults with Becker * Adolescents were not selected based on NSAA Reference: NCT05160415 Abbreviations: CK, creatine kinase 5 mg or 12.5 mg PO daily Study design - 12 months Key Secondary: North Star Ambulatory Assessment (NSAA); change from baseline at month 12 Additional Endpoints: Safety, PK, biomarkers, timed function tests, MRI Month Placebo 10 mg PO daily Placebo 0 1 3 6 9 12 ADULTS ADOLESCENTS (ages 12-17) ADULT PRIMARY EFFICACY ENDPOINT Change from baseline in CK averaged across Months 6, 9 and 12 KEY INCLUSION CRITERIA Ambulatory males aged 12 to 50 years with a dystrophin mutation and a Becker phenotype, not on corticosteroids, with a NSAA between 5-32* PATIENTS ENROLLED Adults: 40 Adolescents: 29 |
| 14
Summary of Key Study Metrics
* Trial sites in the US, UK and the Netherlands
16
sites
3
countries
Enrollment: Largest Becker study to date
Total of 80 patients screened and 69 patients enrolled:
• Adults: 40
• Adolescents: 29
Low discontinuation rate
• 4% overall
• 2.5% in adult cohort (1 out of 40 enrolled)
99% of eligible participants enrolled in
MESA open-label extension
• Approximately, 85 Becker patients currently enrolled
in MESA
* |
| 15
Becker Mutation Overview: Slow Progressor Genotypes
were Largely Excluded with Functional Cut-Off Criteria
Mutation type Adults (N=40) Adolescents (N=29)
Becker mutations associated with progression 39 26
45-x 20 11
Other 19 15
None/very slow progression Becker mutations 1 3
X-51 1 (sevasemten) 2 (sevasemten)
Del 48 0 0
45-55 (associates with late myopathy) 0 1 (sevasemten)
Reference: Bello L., et. al., Sci Rep., 2016 |
| 16
Overview of Baseline Function in Safety Population
*At baseline, 1 placebo and 9 sevasemten treated participants were unable to rise from floor
Functional test Adults Sevasemten
(n=28)
Adults Placebo
(n=12)
ARCH
(N=12)
Mean total NSAA score, points (SD) 18.4 (7.66) 24.2 (8.19) 15.1 (8.4)
Mean 4SC velocity, 1/seconds (SD) 0.22 (0.128) 0.34 (0.173) 0.19 (0.164)
Mean RFF velocity, 1/seconds (SD)* 0.14 (0.114) 0.21 (0.128) 0.16 (0.196)
Mean 10MWR velocity, meters/second (SD) 1.52 (0.731) 2.00 (0.884) 1.15 (0.521)
Mean 100MTT velocity, meters/second (SD) 1.50 (0.856) 1.78 (0.782) 1.08 (0.496)
Abbreviations: NSAA, North Star Ambulatory Assessment |
| 17
Functional Measures Not Well Matched at Baseline;
Patients in Sevasemten Group had Lower Baseline NSAA
*At baseline, 1 placebo and 9 sevasemten treated participants were unable to rise from floor
Functional test Adults Sevasemten
(n=28)
Adults Placebo
(n=12)
Difference
(from placebo)
P-value vs.
Placebo
Mean total NSAA score, points (SD) 18.4 (7.66) 24.2 (8.19) -5.8 0.04
Mean 4SC velocity, 1/seconds (SD) 0.22 (0.128) 0.34 (0.173) -0.12 0.02
Mean RFF velocity, 1/seconds (SD)* 0.14 (0.114) 0.21 (0.128) -0.07 0.09
Mean 10MWR velocity, meters/second (SD) 1.52 (0.731) 2.00 (0.884) -0.48 0.08
Mean 100MTT velocity, meters/second (SD) 1.50 (0.856) 1.78 (0.782) -0.28 0.32
The baseline imbalance observed is a direct consequence of a small study and
should resolve in the larger GRAND CANYON cohort (n=120)
Abbreviations: NSAA, North Star Ambulatory Assessment |
| 18
Sevasemten was Well Tolerated: Overview of
Treatment Emergent Adverse Events (TEAE)
* A treatment emergent adverse event (TEAE) is any adverse event (AE) that starts during or after the first dose of investigational product through the end of the safety follow-up period
Functional test Sevasemten (n=28)
n (%)
Placebo (n=12)
n (%)
Total (N=40)
n (%)
Any TEAE* 26 (92.9) 10 (83.3) 36 (90)
Severe TEAE 0 (0) 0 (0) 0 (0)
Serious Adverse Events 1 (3.6) 0 (0) 1 (2.5)
Any drug related TEAE 16 (57.1) 5 (41.7) 21 (52.5)
Discontinuation due to TEAE 1 (3.6) 0 (0) 1 (2.5)
Deaths 0 (0) 0 (0) 0 (0) |
| 19
Sevasemten was Well Tolerated: TEAEs Occurring in
≥5% of Total
System Organ Class/Preferred Term Sevasemten (n=28) n (%) Placebo (n=12) n (%) Total (N=40) n (%)
Any TEAE 26 (92.9%) 10 (83.3%) 36 (90%)
Eye disorders
Vision blurred 1 (3.6%) 2 (17%) 3 (8%)
Gastrointestinal disorders
Abdominal pain 1 (4%) 1 (8%) 2 (5.%)
Vomiting 2 (7%) 0 (0%) 2 (5.%)
General disorders and administration site conditions
Fatigue 5 (18%) 3 (25%) 8 (20%)
Chest discomfort 2 (7%) 0 (0%) 2 (5%)
Influenza like illness 2 (7%) 0 (0%) 2 (5%)
Infections and infestations
COVID-19 6 (21%) 2 (17%) 8 (20%)
Nasopharyngitis 6 (21%) 2 (17%) 8 (20.%)
Upper respiratory tract infection 5 (18%) 2 (17%) 7 (18%)
Influenza 4 (14%) 1 (8%) 5 (13%)
Injury, poisoning and procedural complications
Fall 8 (29%) 2 (17%) 10 (25%)
Back injury 1 (4%) 1 (8%) 2 (5%)
Investigations
Ejection fraction decreased 0 (0%) 2 (17%) 2 (5%)
Musculoskeletal and connective tissue disorders
Arthralgia 2 (7%) 1 (8%) 3 (8%)
Back pain 3 (11%) 0 (0%) 3 (8%)
Osteopenia 2 (7%) 0 (0%) 2 (5%)
Tendonitis 2 (7%) 0 (0%) 2 (5%)
Nervous system disorders
Headache 9 (32%) 2 (17%) 11 (28%)
Dizziness 9 (32%) 0 (0%) 9 (23%)
Somnolence 5 (18%) 1 (8%) 6 (15%)
Migraine 3 (11%) 1 (8%) 4 (10%)
Dizziness postural 2 (7%) 1 (8%) 3 (8%)
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain 4 (14%) 0 (0%) 4 (10%)
Cough 3 (11%) 0 (0%) 3 (8%)
Nasal congestion 2 (7%) 0 (0%) 2 (5%) |
| 20
Statistically Significant Decrease in the Primary Endpoint
of CK: 28% Reduction vs Placebo
Note: CK values are log-transformed. LS (least squares) means, LS mean differences and CIs were back-transformed to percent scale
Abbreviations: CFB, change from baseline, CI, confidence interval, CK, creatine kinase
CK between-group difference LSMean:
-28%
(95% CI -44% to -6%); p-value = 0.02
Placebo Sevasemten
-40
-20
0
20
40
% CFB CK (Avg 6-12 mo)
0 3 6 9 12
-50
0
50
100
Time (months)
% CFB in CK
Average 6 -12 months
Sevasemten
Placebo
CK showed rapid and sustained decreases with sevasemten treatment |
| 21
TNNI2 Decreased 77% from Baseline in the Sevasemten
Treatment Group vs Placebo
TNNI2 between-group difference LSMean:
-77%
(95% CI -89% to 51%); p-value < 0.001
Average 6 -12 months
Sevasemten
Placebo
0 3 6 9 12
-100
0
100
200
Time (months)
% CFB in TNNI2
Placebo Sevasemten
-100
-50
0
50
100
150
% CFB TNNI2 (Avg 6-12 mo)
Note: TNNI2 values are log-transformed. LS means, LS mean differences and CIs were back-transformed to percent scale
Abbreviations: CFB, change from baseline, CI, confidence interval, TNNI2, troponin I2
TNNI2, an on-target biomarker of fast muscle fiber damage, also
demonstrated rapid and sustained decreases with sevasemten treatment |
| 22
Key Secondary Endpoint: NSAA Remained Stable
Over Time in Sevasemten Group
0 3 6 9 12
-3
-2
-1
0
1
2
Time (months)
CFB Total NSAA Score
NSAA between-group difference LSMean: 1.12
(95% CI -0.4 to 2.7); p-value = 0.16
Sevasemten
Placebo
Positive trends in NSAA favoring sevasemten with placebo
declining in line with natural history
Abbreviations: NSAA, North Star Ambulatory Assessment |
| 23
63% of Patients Treated with Sevasemten Showed
Stable or Improved Function After 12 Months
Sevasemten NSAA Responder Analysis
-6
-4
-2
2
4
6
-6
-4
-2
2
4
6
Placebo (n=12)
Sevasemten (n=27)
63%
33% 67%
37%
Stability or Improvement
Odds Ratio: 3.4 (p=0.16)
Abbreviations: NSAA, North Star Ambulatory Assessment |
| 24
Trends in Other Functional Measures Favor
Sevasemten Treatment Arm
-4 -2 0 2 4
RFF Velocity
4SC Velocity
100MTT Velocity
10MWR Velocity
NSAA
Standardized Statistics (95% CI)
1.12 0.075 0.055
0.024
-0.005
LSMean Difference
* At baseline, 9 sevasemten and 1 placebo treated participants were unable to rise from floor. At Month 12, 9 sevasemten and 2 placebo treated participants were unable to rise from floor
Note: For the figures, LSM differences and CIs were standardized by dividing by the SE. LSM differences presented on the right of the figure are on original scale (without SE adjustment)
*
Favors Placebo Favors Sevasemten
Abbreviations: NSAA, North Star Ambulatory Assessment, CI, confidence interval |
| 25
Summary of Trial Results
• Sevasemten treated patients showed stabilization of NSAA with
trends toward improvement
• Placebo group (n=12) declined in line with natural history
• Primary endpoint achieved: 28% average decrease in CK versus
placebo (p=0.02)
• Plasma TNNI2 decreased 77% from baseline versus placebo (p<0.001)
• Well-tolerated, at all doses, in adults and adolescents
• No safety concerns identified
• The imbalance between groups confounded interpretation of a
few endpoints (e.g., MRI); evaluation of the full data set ongoing
Safety
Biomarkers
Function
Secondary &
Exploratory
Abbreviations: CK, creatine kinase, NSAA, North Star Ambulatory Assessment |
| Sevasemten
Future Development Plans |
| 27
9 15
Global, Multi-Center, Placebo-Controlled Pivotal Cohort
Assessing Efficacy & Safety of Sevasemten in Becker
Abbreviations: PO, by mouth; NSAA, North Star Ambulatory Assessment
PRIMARY ENDPOINT
NSAA at
18 months
KEY INCLUSION
CRITERIA
Adult individuals with
Becker with NSAA 5-32,
not taking corticosteroids
ENROLLMENT
>120
Study design - 18 months
10mg
PO daily Screening
0 1 3 6
Additional Endpoints
Timed function tests (TFTs), biomarkers of muscle damage, MRI, patient reported outcomes (PROs), safety
Month
POTENTIAL REGISTRATIONAL COHORT
12
POWERED AT
>90%
for observing a difference
corresponding to the natural
history NSAA decline of 1.2
points/year
Placebo
R
2:1
18 |
| 28
GRAND CANYON Powered at 96% to Show a 1.68 Point
NSAA Difference at 18 Months
p<0.05
Treatment Difference
Power Calculation Assumptions:
• N=120 patients with 2:1 randomization
• Month 12 CANYON treatment difference = 1.12
• Assumed 50% increase in NSAA treatment effect
at month 18 vs month 12 ( = 1.68)
• 10% drop-out rate
• Standard deviation () = 2.16
• Tipping point = 0.87 (represents point where the p-value is ~0.05 given the study size and assumed
standard deviation based on t-test)
Power: 96%
-2 -1 0 1 2 3 4
Abbreviations: NSAA, North Star Ambulatory Assessment |
| 29
Sevasemten for Becker Next Steps
*European sites include the UK, Netherlands, Belgium, Denmark, Spain, France, Germany and Italy
Increased
confidence in
GRAND CANYON
future success
based on sevasemten
clinical experience to
date, Becker natural
history data, and internal
modeling
CANYON results
support
engagement with
FDA & EMA
about marketing
authorization filing
strategies for sevasemten
in Becker
GRAND CANYON is
near full
enrollment
in 51 sites across the
United States, Europe*,
New Zealand, Australia
and Israel;
on track to over-enroll by Q1 2025
1 2
To date, 99% of
eligible Becker
participants
who have completed
ARCH, CANYON, GRAND
CANYON and DUNE
have enrolled in
MESA
3 4 |
| CANYON Clinical Implications and
Sevasemten’s Potential Role in Becker
Dr. Craig M. McDonald |
| 31
Reference: 1. Bello L, et al. 2016. 2. De Wel B, et al. Eur J Neurol. 2024. 3. Van de Velde NM, et al. Neurology. 2021.;4. Bello L et al 2024 WMS; 95% CI shown
Natural History Data in Becker Support that Functional Decline,
Measured by NSAA, is Consistent and Predictable
0 1 2 3 4 5
-10
-8
-6
-4
-2
0
Years of Observation
Change in NSAA
Natural history of Becker muscular dystrophy
• NSAA is utilized in muscular
dystrophy natural history studies to
longitudinally assess function
• Multiple natural history studies in
individuals with Becker
demonstrate a NSAA average
score decline of 1.0 to 1.8 points
annually .
1,2,3
• Becker Natural history studies
support that NSAA decline is
consistent in Becker patients who
are already progressing |
| 32
A Clinician’s Perspective on How to Interpret a 1-point NSAA Change
in Becker
From using
the toilet
independently…
…to asking for help
to get up from the
toilet.
…to requiring
assistance from
another person or
mobility device.
…to requiring
someone else’s help
to get back up.
From using stairs
or steps…
From being able
to get up from a
fall…
For individuals living with Becker, this decline could look like:
Abbreviations: NSAA, North Star Ambulatory Assessment |
| Closing Remarks
Kevin Koch, CEO |
| We Aim to Change the Lives of
Individuals with Becker
~12,000 PEOPLE WITH
BECKER IN US,
EU-5 & JAPAN
~16 AGE PEOPLE WITH BECKER
CAN BECOME MOBILITY
DEVICE DEPENDENT
• Source: Parent Project Muscular Dystrophy: Fifteen Year Registry Report; Edgewise Therapeutics independent quantitative and qualitative market research (Bluestar BioAdvisors); Edgewise research; Emery AE, Neuromuscul. Disord., 1991; MD STARnet Data and
Statistics; Duchenne muscular dystrophy. National Center for Advancing Translational Sciences; Duan D et al., Nat. Rev. Dis. Primers, 2021
0
APPROVED
THERAPIES |
| 35
Abbreviations: HV, healthy volunteers; oHCM, obstructive hypertrophic cardiomyopathy; nHCM, non-obstructive hypertrophic cardiomyopathy; CV, cardiovascular; IND, Investigational New Drug application;
POC, proof of concept
Edgewise Upcoming Value-Generating Milestones
H2 2024 H1 2025 H2 2025
Becker
Duchenne
Skeletal
Sevasemten
Hypertrophic
Cardiomyopathy
Cardiac
EDG-7500
Phase 2 LYNX & FOX
Controlled dose-ranging data
Phase 3 trial
Initiation
Phase 2 CANYON
1-year placebo-controlled data
Phase 1/Phase 2
EDG-7500 data in HVs & oHCM
Phase 2 28-day study
Initiate in oHCM & nHCM
Phase 2 28-day study
Initial Data readout
✓
✓
GRAND CANYON
Recruitment complete (Q1)
Cardiometabolic Drug
In vivo POC
Phase 2
12-week oHCM & nHCM
✓
EDG-CV candidate
IND in HF |
| 36 As of September 30, 2024
Well-Capitalized to Execute Important Milestones Across Both
EDG-7500 & Sevasemten
(1) DEBT (1)
$0
CASH, CASH EQUIVALENTS &
MARKETABLE SECURITIES
~$493M
COMMON SHARES OUTSTANDING
(NASDAQ: EWTX)
~94M
CASH RUNWAY THROUGH 2027 |
| 37
Q & A |
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