Spruce Biosciences Provides Clinical Program Updates and Outlines Anticipated Milestones for 2023
January 09 2023 - 8:05AM
Business Wire
Topline Results from CAHmelia-203 in Adult
Classic Congenital Adrenal Hyperplasia (CAH) Anticipated in 2H
2023
Amendment to Phase 2 CAHptain Clinical Trial in
Pediatric Classic CAH Enhances Study Design; Topline Data from
Adolescents Anticipated in 2H 2023
Topline Data in Phase 2 P.O.W.E.R. Study in
Polycystic Ovary Syndrome Anticipated in 1H 2023
Entered Exclusive Licensing Agreement with
Kaken Pharmaceutical to Develop and Commercialize Tildacerfont for
CAH in Japan
Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage
biopharmaceutical company focused on developing and commercializing
novel therapies for rare endocrine disorders with significant unmet
medical need, today provided an update on its clinical programs,
anticipated upcoming milestones and strategic priorities for
advancing tildacerfont in classic congenital adrenal hyperplasia
(CAH) and polycystic ovary syndrome (PCOS).
“As we enter 2023, we are pleased to have recently announced our
strategic partnership with Kaken Pharmaceutical to develop and
commercialize tildacerfont in Japan,” said Javier Szwarcberg, M.D.,
MPH, Chief Executive Officer of Spruce Biosciences. “2022 was a
year of clinical execution across the board, which puts us in a
strong position to report topline data for a number of our clinical
programs this year, particularly our CAHmelia-203 study for adult
classic CAH in the second half of 2023. We also continue to make
progress in our Phase 2 P.O.W.E.R. study for the treatment of PCOS
and anticipate reporting topline proof-of-concept data in the first
half of 2023.”
Dr. Szwarcberg continued, “Children represent the greatest unmet
medical need in classic CAH, with urgent necessity for
androgen-lowering and glucocorticoid-sparing therapies to reduce
the risk of premature puberty and adverse effects, both of which
have the potential to be addressed by tildacerfont, if approved. To
that end, we’ve identified opportunities to enhance our CAHptain
pediatric classic CAH clinical trial, by lowering the minimum age
requirement from 6 years to 2 years of age and increasing the study
length from 2 weeks to 12 weeks, with an additional 2-year
extension option. We expect these enhancements will expand the
amount of informative data that can be generated from the program
and increase attractiveness for patients and families to
participate.”
Clinical Programs
Late-Stage CAHmelia Program in Adult Classic CAH
- CAHmelia-203 Study for Adult Classic CAH: CAHmelia-203
is a randomized, double-blind, placebo-controlled, dose-ranging
study evaluating the safety and efficacy of tildacerfont in adult
patients with classic CAH and is designed to enroll approximately
72 patients with high levels of androstenedione (A4) while on their
current glucocorticoid regimen. Enrollment in the clinical trial is
approaching 50%.
- Anticipated Upcoming Milestone: Topline results in the
second half of 2023
- CAHmelia-204 Study for Adult Classic CAH: CAHmelia-204
is a randomized, double-blind, placebo-controlled study evaluating
the safety and efficacy of tildacerfont in adult patients with
classic CAH. The study is designed to enroll approximately 90
patients on supraphysiologic doses of glucocorticoids at or above
30 mg/d hydrocortisone equivalent with normal or near normal levels
of A4. Enrollment in the clinical trial recently surpassed
25%.
- Anticipated Upcoming Milestone: Topline results in the
second half of 2024
Pediatric Classic CAH Program
Phase 2 CAHptain Clinical Study in Pediatric Classic CAH:
Spruce is investigating tildacerfont for the treatment of classic
CAH in children. There is a significant medical need to bring
androgen-lowering and glucocorticoid-sparing therapies to pediatric
classic CAH patients to reduce the risk of premature puberty and
the adverse effects of glucocorticoids, including stunted growth
resulting in short stature as adults. The Phase 2 open-label
clinical trial utilizes a sequential 3 cohort design (cohorts 1 and
2 comprising of adolescent patients 11 to 17 years of age, and
cohort 3 comprising of children 2 to 10 years of age) to evaluate
the safety, pharmacokinetics (PK), and exploratory pharmacodynamics
(PD) of tildacerfont in children 2 to 17 years of age with classic
CAH. Spruce implemented key protocol changes to enhance the study
design and gather additional data to inform future clinical
development:
- Increasing the Study Length, Lowering Age
Eligibility, and Adding an Open-Label Extension to Make the Trial
More Accessible to Patients and Families
- Spruce amended the study length from a 2-week PK and
exploratory PD study to a 12-week study. The company plans to also
offer a 2-year open-label extension to the 12-week study. These
changes are designed to enable patients to retain access to the
study drug for up to 2 years following completion of the study, and
provide for observation of clinical outcomes, such as bone age and
predicted adult height.
- The company is lowering the minimum age requirement from 6
years to 2 years of age. Given the significant growth and
development that occurs in children between the ages of 2 years and
5 years of age, this change is designed to provide important data
on the impact of reductions in androgen levels and glucocorticoids
(GC) in younger children.
- Increasing the Amount of Data That Can Be
Extrapolated from Program
- The following additional data will be collected to inform a
potential Phase 3 registrational clinical trial, while allowing for
observation of key clinical outcomes:
- 2 weeks of pediatric tildacerfont PK exposure data at two
weight adjusted doses (50mg and 200mg) to inform a dose for the
Phase 3 registrational program;
- 4 weeks of PD data to potentially show reduction in
androstenedione (A4) and establish dose-response (day 1-28);
- A4 reduction data and GC reduction based on a
protocol-specified algorithm (day 28-90); and
- sub-chronic safety data at 12 weeks.
- Anticipated Upcoming Milestone: Topline data from
adolescents (cohorts 1 and 2) in the second half of 2023
Polycystic Ovary Syndrome (PCOS) Program
Phase 2 P.O.W.E.R. Clinical Study in PCOS: Spruce is
conducting the P.O.W.E.R. study, a randomized, placebo-controlled,
dose escalation study which will evaluate the safety and efficacy
of tildacerfont titrated to 200 mg once daily compared to placebo
at 12 weeks in subjects with PCOS and elevated adrenal androgens as
measured by dehydroepiandrosterone sulfate (DHEAS) levels at
baseline. PCOS is a hormonal disorder common among females of
reproductive age characterized by hirsutism, irregular periods,
infertility, and ovarian cysts. Adrenal androgen overproduction is
thought to contribute to the clinical manifestations of PCOS in
some patients. By reducing ACTH-stimulated adrenal androgen
production, tildacerfont has the potential to treat the clinical
sequelae of PCOS.
- Anticipated Upcoming Milestone: Topline results in the
first half of 2023
Financial Update
The company estimates that its cash, cash equivalents, and
investments were approximately $79 million as of December 31, 2022.
This amount is unaudited and preliminary and is subject to
completion of financial closing procedures. Additional information
and disclosure would be required for a more complete understanding
of the company’s financial position and results of operations as of
December 31, 2022.
With the anticipated receipt of the $15.0 million upfront
payment from the company’s license agreement with Kaken
Pharmaceutical, Spruce’s cash runway is expected to extend into the
first half of 2024.
About Spruce Biosciences
Spruce Biosciences is a late-stage biopharmaceutical company
focused on developing and commercializing novel therapies for rare
endocrine disorders with significant unmet medical need. Spruce is
initially developing its wholly-owned product candidate,
tildacerfont, as the potential first non-steroidal therapy for
patients suffering from classic congenital adrenal hyperplasia
(CAH). Spruce is also developing tildacerfont for women suffering
from a rare form of polycystic ovary syndrome (PCOS) with primary
adrenal androgen excess. To learn more, visit
www.sprucebiosciences.com and follow us on Twitter @Spruce_Bio,
LinkedIn, Facebook and YouTube.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Such forward-looking statements include statements
regarding, among other things, the results, conduct, progress and
timing of Spruce’s clinical trials, including the timing of
reporting topline data and the impact of the strategies to enhance
the design of the CAHmelia studies and the resulting informative
data that may be generated, the fulfillment of Spruce’s strategic
business objectives, the advancement of Spruce’s drug development
pipeline, and Spruce’s expectations regarding its extended cash
runway. Because such statements are subject to risks and
uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Words such
as “anticipated,” “expects,” “intended,” “plans”, “will”,
“potential” and similar expressions are intended to identify
forward-looking statements. These forward-looking statements are
based upon Spruce’s current expectations and involve assumptions
that may never materialize or may prove to be incorrect. Actual
results could differ materially from those anticipated in such
forward-looking statements as a result of various risks and
uncertainties, which include, without limitation, risks and
uncertainties associated with Spruce’s business in general, the
impact of macroeconomic and geopolitical events, including the
COVID-19 pandemic, and the other risks described in Spruce’s
filings with the U.S. Securities and Exchange Commission. All
forward-looking statements contained in this press release speak
only as of the date on which they were made and are based on
management’s assumptions and estimates as of such date. Spruce
undertakes no obligation to update such statements to reflect
events that occur or circumstances that exist after the date on
which they were made, except as required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20230109005319/en/
Media Will Zasadny Evoke Canale (619) 961-8848
will.zasadny@evokecanale.com media@sprucebiosciences.com
Investors Xuan Yang Solebury Strategic Communications
(415) 971-9412 xyang@soleburystrat.com
investors@sprucebiosciences.com
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