Final Enrollment in CAHmelia-203 to Exceed
Target Due to Substantial Patient Interest
CAHmelia-203 Topline Results Anticipated in the
First Quarter of 2024
CAHmelia-204 Completion of Enrollment
Anticipated in Early First 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 target enrollment of 72
patients and closure of screening in the CAHmelia-203 clinical
trial evaluating tildacerfont for the treatment of adult classic
congenital adrenal hyperplasia (CAH) and provided updated
anticipated milestones.
“Completing target enrollment in the CAHmelia-203 study is a
significant milestone for our CAH program and reflects the
continued strong enthusiasm from patients and study investigators
alike, evidences the large unmet medical need in the CAH community,
and reinforces the momentum within our broader tildacerfont
program,” said Javier Szwarcberg, M.D., M.P.H., Chief Executive
Officer of Spruce Biosciences. “Due to substantial patient interest
in CAHmelia-203, eligible adult patients currently in screening
will be allowed to enter the trial. As a result, we anticipate that
final enrollment will exceed our original target of 72 patients,
and project topline results from this study along with the topline
results from all cohorts in our CAHptain clinical trial in
pediatric classic CAH in the first quarter of 2024.”
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 androstenedione (A4) at baseline while on stable
glucocorticoid dosing. Tildacerfont is a once-daily, potent and
highly selective, non-steroidal, oral antagonist of the
corticotropin-releasing factor (CRF) 1 receptor. The primary
endpoint of the clinical trial is the percentage change in A4 from
baseline to week 12 with secondary endpoints including the
proportion of patients with levels of 17-hydroxyprogesterone
(17-OHP) and A4 within the target and normal range, respectively,
and change in lesion volume of testicular adrenal rest tumors
(TARTs) in men. Additional endpoints include clinical outcomes and
patient and clinician reported outcomes.
Anticipated Upcoming
Milestones
Spruce has updated its anticipated upcoming milestones as
follows:
- Completion of enrollment in the CAHmelia-204 clinical trial
in adult classic CAH patients on supraphysiologic doses of
glucocorticoids with normal or near normal levels of A4 in early
first quarter of 2024
- Topline results from all cohorts in the Phase 2 CAHptain
clinical trial in pediatric classic CAH patients in the first
quarter of 2024
- Topline results from the CAHmelia-203 clinical trial in adult
classic CAH patients with highly elevated levels of A4 in the first
quarter of 2024
- Topline results from the CAHmelia-204 clinical trial in adult
classic CAH patients on supraphysiologic doses of glucocorticoids
with normal or near normal levels of A4 in the third quarter of
2024
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 targets enrollment of approximately 72
patients 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 at week zero to week 12 with
secondary endpoints including the proportion of patients with
levels of 17-OHP and A4 within the target and normal range,
respectively, and change in lesion volume of TARTs in men.
Following the placebo-controlled treatment period, all patients
will receive tildacerfont following a dose-escalation protocol to
increase dosage 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. Additional endpoints
include clinical outcomes and patient and clinician reported
outcomes. 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
clinical trial to evaluate the safety and efficacy of tildacerfont
in reducing supraphysiologic glucocorticoid usage in approximately
90 adults with classic CAH in patients 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 (replacement levels only), as long as patients
remain well controlled based on standard biomarkers and clinical
assessments. The primary endpoint of this clinical trial is the
proportion of patients with reduction of glucocorticoid dose
greater than or equal to 5 mg/day of hydrocortisone equivalent
(HCe) at week 24. The percent change in glucocorticoid dose from
baseline to week 24 will be assessed as a secondary endpoint.
Median total cumulative GC dose (HCe) at week 24, change from
baseline in insulin resistance at week 24, and percent change from
baseline in weight at week 24 and after 52 weeks of tildacerfont
treatment will also be assessed as secondary endpoints. Effects on
insulin resistance, weight, waist circumference, bone mineral
density after 52 weeks of tildacerfont treatment will be assessed
as exploratory endpoints. For more information about the CAHmelia
program, please visit https://www.sprucebio.com/cahmelia.
About CAHptain-205
CAHptain-205 is a Phase 2 open-label clinical trial, which will
utilize a sequential three cohort design, to evaluate the safety,
efficacy, and pharmacokinetics of tildacerfont in children two to
17 years of age. The study will also characterize changes in
androgen levels over 12 weeks of treatment, and the ability to
reduce daily glucocorticoid dose based on A4 normalization. An
optional open-label extension period will provide additional
open-label treatment with tildacerfont to provide long-term safety
data for up to two years. Cohort 1 will enroll up to five
participants between the ages of 11 and 17 years of age, who will
receive a weight-adjusted, adult dose equivalent of 50mg QD of
tildacerfont. Cohort 2 will enroll up to five participants between
the ages of 11 and 17 years of age, who will receive a
weight-adjusted, adult dose equivalent of 200mg QD of tildacerfont.
Cohort 3 will enroll up to 10 participants between the ages of two
and 10 years of age, who will receive a weight-adjusted, adult dose
equivalent of 200mg QD of tildacerfont. The primary endpoint of
this clinical trial is safety. Additional secondary endpoints
include the proportion of subjects who achieve reduction in A4 or
daily glucocorticoid dosing at week 12 and the proportion of
subjects with elevated A4 at baseline who achieve a reduction in A4
at week 4. For more information about the CAHptain program, please
visit https://www.sprucebio.com/cahptain.
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 endogenous 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,
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
patients 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 therapy for
patients suffering from classic congenital adrenal hyperplasia
(CAH). Spruce is also developing tildacerfont for women suffering
from polycystic ovary syndrome (PCOS). To learn more, visit
www.sprucebiosciences.com and follow us on X, formerly known as
Twitter, 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 enrollment, results, conduct,
progress and timing of Spruce’s clinical trials; and the receipt
and presentation of topline results from the same. 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”, “enable,”
“expect”, “will”, “believe”, “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/20231018358017/en/
Media Will Zasadny Evoke Canale (619) 961-8848
will.zasadny@evokecanale.com media@sprucebio.com
Investors Samir Gharib President and CFO Spruce
Biosciences, Inc. investors@sprucebio.com
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