100 Patients Enrolled, Exceeding Target
Enrollment of 90 Patients
Topline Results for CAHmelia-204 Anticipated in
the Third Quarter of 2024
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 announced completion of enrollment in its
CAHmelia-204 clinical trial of tildacerfont for the treatment of
adult classic congenital adrenal hyperplasia (CAH).
“Completing enrollment in our CAHmelia-204 clinical trial marks
a significant milestone in our adult classic CAH program. This
achievement reinforces the continued momentum within our
tildacerfont program and positions us favorably to report topline
results in the third quarter of 2024,” said Javier Szwarcberg,
M.D., M.P.H., Chief Executive Officer of Spruce Biosciences.
“Thanks to positive engagement from our patient community and study
investigators alike, the CAHmelia-204 clinical trial enrolled 100
patients, exceeding target enrollment of 90 patients.”
Dr. Szwarcberg continued, “Looking ahead, assuming positive
results from CAHmelia-203 in March and CAHmelia-204 in the third
quarter, we plan to meet with the U.S. Food and Drug Administration
(FDA) and comparable foreign regulatory authorities to discuss the
potential registrational path forward for tildacerfont as a
treatment for adult classic CAH.”
CAHmelia-204 is a randomized, double-blind, placebo-controlled
clinical trial to evaluate the safety and efficacy of tildacerfont,
a potentially novel, once-daily, non-steroidal treatment option, in
reducing glucocorticoid usage in 100 adults with classic CAH on
supraphysiologic doses of glucocorticoids with normal or near
normal levels of androstenedione (A4) at baseline. The primary
endpoint of this clinical trial is the absolute change in daily
glucocorticoid dose in hydrocortisone equivalents (HCe) from
baseline at week 24.
About CAHmelia-203
CAHmelia-203 is a randomized, double-blind, placebo-controlled,
dose ranging Phase 2b clinical trial evaluating the safety and
efficacy of tildacerfont in adults with classic CAH and highly
elevated levels of A4 at baseline while on stable glucocorticoid
dosing. This clinical trial enrolled 96 subjects with elevated
levels of A4. For the first six weeks, patients will receive
blinded placebo to assess their adherence to their existing
glucocorticoid therapy. Patients who continue to meet all
eligibility criteria at the end of this period will enter a
three-part treatment period. During the placebo-controlled
treatment period, patients will be randomized in a blinded manner
to receive placebo, 50mg, 100mg, or 200mg tildacerfont once daily.
Dosing in the placebo-controlled treatment period will continue for
12 weeks. The primary endpoint of the clinical trial is the
percentage change in A4 from baseline to week 12. Following the
placebo-controlled treatment period, all patients will receive
tildacerfont following a dose-escalation protocol that allows dose
increase to 200mg once daily over 12 weeks. Following the 12-week
dose-escalation period, all patients will continue receiving
tildacerfont at 200mg once daily for an additional 46 weeks.
Patients who achieve control of A4 while on supraphysiologic
glucocorticoid treatment will have the opportunity to reduce their
glucocorticoid dosing in the open-label period according to a
pre-specified algorithm in the protocol. For more information about
the CAHmelia program, please visit
https://www.sprucebio.com/cahmelia.
About CAHmelia-204
CAHmelia-204 is a randomized, double-blind, placebo-controlled
Phase 2b clinical trial to evaluate the safety and efficacy of
tildacerfont in reducing glucocorticoid usage in 100 adults with
classic CAH on supraphysiologic doses of glucocorticoids with
normal or near normal levels of A4 at baseline. This clinical trial
is designed in two parts. In the first part of the clinical trial,
patients will be randomized to receive 200mg tildacerfont once
daily or placebo for 24 weeks. During the second part of the
clinical trial, all patients will receive open-label 200mg
tildacerfont once daily for 52 weeks. Throughout the trial,
tapering of glucocorticoids will be guided according to a
pre-specified algorithm and continue to the lowest level possible
(physiologic replacement levels), as long as patients remain well
controlled based on standard biomarkers and clinical assessments.
The primary endpoint of this clinical trial is the absolute change
in daily glucocorticoid dose in HCe from baseline at week 24. For
more information about the CAHmelia program, please visit
https://www.sprucebio.com/cahmelia.
About Congenital Adrenal Hyperplasia (CAH)
CAH is an autosomal recessive disease, driven by a mutation in
the gene that encodes an enzyme necessary for the synthesis of key
adrenal hormones. In CAH patients, the body is not able to produce
cortisol, leading to serious health consequences. The absence of
cortisol alters the normal feedback cycle of the
hypothalamic-pituitary-adrenal (HPA) axis and leads to excess
secretion of adrenocorticotropic hormone (ACTH), hyperplasia of the
adrenal gland, and consequently high levels of adrenal androgen
production. As a result, CAH patients suffer from premature
puberty, impaired fertility, hirsutism, acne, the development of
adrenal rest tumors, and an impaired quality of life, and
additionally for females, virilized genitalia and menstrual
irregularities. Currently, the only way to downregulate the
production of excess androgens in CAH patients is to administer
supraphysiologic doses of glucocorticoids, which present specific
side effects, including increased risks of developing diabetes,
cardiovascular disease, stunted growth, osteoporosis, thin skin,
gastrointestinal disorders, and decreased lifespan.
About Tildacerfont
Tildacerfont is a potent and highly selective, non-steroidal,
once-daily oral antagonist of the CRF1 receptor, which is the
receptor for corticotropin-releasing factor (CRF), a hormone that
is secreted by the hypothalamus. The CRF1 receptor is abundantly
expressed in the pituitary gland where it is the primary regulator
of the HPA axis. By blocking the CRF1 receptor, tildacerfont has
the potential to address the uncontrolled cortisol feedback
regulatory pathway in CAH, and in turn reduce the production of
ACTH in the pituitary, limiting the amount of androgen produced
downstream from the adrenal gland. By controlling excess adrenal
androgens through an independent mechanism, tildacerfont has the
potential to reduce the unwanted clinical symptoms associated with
high androgen exposure and could also enable treating physicians to
lower the supraphysiologic glucocorticoid doses given to CAH
patients to near physiologic levels. Tildacerfont has been
evaluated in over 200 subjects across nine completed clinical
trials in which it has been generally well tolerated. No
drug-related serious adverse events have been reported related to
tildacerfont treatment in completed studies.
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, once-daily
therapy for patients suffering from classic congenital adrenal
hyperplasia (CAH) and other endocrine disorders. To learn more,
visit www.sprucebio.com and follow us on X, 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; Spruce’s expectations regarding
reporting results of its clinical trials in 2024; and Spruce’s
plans to meet with the FDA and comparable foreign regulatory
authorities to discuss the potential registrational path forward of
tildacerfont for adult classic CAH. 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 “anticipate”, “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 geopolitical and macroeconomic events, 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/20240122243644/en/
Media Will Zasadny Inizio Evoke (619) 961-8848
will.zasadny@inizioevoke.com media@sprucebio.com
Investors Samir Gharib President and CFO Spruce
Biosciences, Inc. investors@sprucebio.com
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