NEW TAIPEI CITY, Sept. 3,
2024 /PRNewswire/ -- Caliway Biopharmaceuticals
(Caliway) announced that the subject recruitment of CBL-514 Phase
2b study for Dercum's disease
(CBL-0202 DD Phase 2b study,
NCT06303570) has been initiated. The study results are anticipated
in Q4 2025.
CBL-0202DD study is a randomized, placebo-controlled Phase
2b study evaluating CBL-514
injection's efficacy and safety in treating Dercum's disease. A
total of approximately 30 Dercum's disease subjects with four to
ten painful lipomas will be enrolled in the study and randomized
1:1 into two groups to receive either CBL-514 or placebo once every
4 weeks for up to 5 treatments for each selected lipoma.
The CBL-514 Phase 2b study
features notable changes compared with the earlier Phase 2 study
(CBL-0201DD, NCT05387733). While the Phase 2 study evaluated a
4-week treatment period with the primary efficacy endpoint designed
to evaluate the change of lipoma dimensions, the Phase 2b study design extends the treatment period to
16 weeks, with lipoma size evaluated by ultrasound at an
independent third-party facility during each visit from 4 weeks to
24 weeks after the first treatment.
Additionally, the Phase 2b study
design shifts the primary endpoint to the percentage of lipomas
achieving a complete response compared to the placebo, providing a
robust perspective on the drug's efficacy. With a longer treatment
period, a larger subject population, and a more challenging primary
endpoint, the CBL0202DD Phase 2b
study represents a significant advancement in the pursuit of
innovative treatments for Dercum's disease. Caliway will proceed
with the CBL-514 Phase 3 Pivotal study IND for Dercum's disease as
soon as the Phase 2b study results
meet the efficacy endpoints.
CBL-514 is the world's most advanced new drug in Dercum's
disease clinical development and is also the only clinical drug
with statistically significant efficacy. The previous CBL-0201DD
Phase 2 study results have demonstrated significant painful lipoma
size reduction with a pain reduction of 4.7 points. With the
potential to become the first approved drug for Dercum's disease
treatment, CBL-514 is currently the only new drug granted with both
Orphan Drug Designation and Fast Track Designation for Dercum's
disease treatment by the U.S. FDA, which will help accelerate its
clinical development and increase the product value once it
launches to the market.
About Dercum's Disease
Dercum's disease is a rare disorder that is characterized by the
development of painful lipomas primarily located on the trunk
region, and the extremities close to the trunk. The pain is chronic
(>3 months), symmetrical, often disabling, and resistant to
traditional analgesics.
The etiology of Dercum's disease remains unknown. There is no
approved drug nor recommended standard treatment for Dercum's
disease yet. The current treatment options, including surgical
removal, liposuction, electrotherapy, and inflammation inhibitors,
are symptomatic, and the efficacies are limited with numerous
adverse events. The clinical need for Dercum's disease remains
unmet.
According to the Global Dercum's Disease Market Research Report,
the global Dercum's disease treatment market size in 2021 was
$11.3 billion. With a compound annual
growth rate (CAGR) of 6.76%, the global market of Dercum's disease
treatment in 2030 is estimated to expand to $19.96 billion.
About CBL-0202DD Phase 2b
Study
CBL-0202DD study (NCT06303570) is a randomized,
placebo-controlled, Phase 2b study
evaluating CBL-514 injection's efficacy and safety in treating
Dercum's disease subjects. The study will enroll approximately 30
Dercum's disease subjects with four to ten painful lipomas and
randomize them 1:1 into two groups to receive CBL-514 injection or
placebo. Each subject will receive up to 5 courses of CBL-514
injection or placebo administered into selected lipomas once every
4 weeks for 16 weeks in total, and the injection volume per lipoma
will be based on the lipoma size, determined by ultrasound. The
efficacy of CBL-514 on each lipoma will be evaluated during each
visit from 4 weeks to 24 weeks after the first treatment at an
independent third-party facility.
The primary endpoint is the percentage of lipomas with complete
response compared to placebo. The secondary endpoints include the
percentage of lipomas with partial response compared to placebo,
the duration of complete response/partial response lasting on the
lipomas compared to placebo, the percentage change in lipoma volume
compared to placebo, and the change of pain scores on the lipomas
compared to placebo.
About CBL-514
CBL-514, a potentially first-in-class small-molecule drug, is a
lipolysis injection that can induce adipocyte apoptosis to reduce
subcutaneous adiposity in treatment areas without causing any
systematic side effects on the central nervous system,
cardiovascular system, and respiratory system. Caliway's
preclinical studies have shown that CBL-514 upregulates the
apoptosis mediators caspase 3 and Bax/Bcl-2 ratio and then induces
adipocyte apoptosis in vivo and in vitro.
Caliway has completed CBL-0201DD, a Phase 2 study (NCT05387733)
to evaluate the efficacy and safety of CBL-514 in treating Dercum's
disease. The study results showed that after CBL-514 treatment,
64.5% of painful lipomas showed a dimensional reduction of more
than 50%, and 38.7% of painful lipomas showed complete clearance,
with a pain reduction of 4.7 points. In addition, CBL-0202DD, a
Phase 2b IND (NCT06303570), has been
approved by the U.S. FDA in January
2024, with subject recruitment initiating in August 2024 and the study results are anticipated
in Q4 2025.
About Caliway Biopharmaceuticals
Caliway Biopharmaceuticals (Caliway) is a Taiwan-headquartered, clinical-stage
biopharmaceutical company driven to breakthrough drug discovery of
novel small-molecule therapeutics. Listed on the emerging stock
market in Taiwan (TPEX-6919),
Caliway aims to become an innovative pharmaceutical leader in
aesthetic medicine and other diseases.
Disclaimer
This article and related information on this site contain
forward-looking statements. The forward-looking information
requires the Company to make numerous assumptions and is subject to
inherent risks, uncertainties, and other factors that are beyond
the control of the Company which may cause actual results,
performance or achievements to be materially different from future
results, performance or achievements expressed or implied by such
forward-looking statements. The Company undertakes no obligation to
timely inform, update, or revise the information on this site if
circumstances should change.
For additional contact: info@caliway.com.tw
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