- Trial delivers positive results in first ever industry-led
clinical trial in Hereditary Haemorrhagic Telangiectasia
(HHT)
- VAD044 showed favourable safety and tolerability, together
with exploratory efficacy across key manifestations of the
disease
- Ongoing Open Label Extension (OLE) data at 6 months show
consistent safety, tolerability and continued improvement in
bleeding parameters
BASEL,
Switzerland, Aug. 27,
2024 /PRNewswire/ -- Vaderis Therapeutics AG
(Vaderis), a clinical stage biotechnology company focused on
developing treatments for rare diseases associated with vascular
malformations, today announces positive results from its
randomized, double-blind, dose-finding placebo-controlled
Proof-of-Concept (POC) clinical trial in patients suffering from
HHT.
HHT, an Orphan Disease, is the second most common inherited
bleeding disorder in the world frequently causing severe disease
burden, reduced life expectancy and impaired Quality of Life.
Despite this, there remains no approved treatment for HHT anywhere
in the world. Vaderis is developing VAD044, an oral, once-daily
allosteric AKT-inhibitor, the first novel therapy intended
specifically for the treatment of HHT.
In this controlled, double-blind trial, seventy-five patients
across USA and Europe were randomized to receive either
placebo, 30mg or 40mg of VAD044 for 12 weeks. Safety was the
primary endpoint and VAD044 was similar to placebo as measured by
both the frequency and severity of off-target adverse events (AEs).
On-target AEs associated with AKT pathway inhibition, were
mostly mild, transient and resolved on study drug.
Almost all HHT patients suffer from unpredictable, often
frequent and debilitating epistaxis which is considered the best
measure of overall HHT disease activity and a key measure of
disease burden. In this study, VAD044 showed a dose response on
secondary and exploratory efficacy endpoints in HHT, including
key epistaxis endpoints. At the end of the 12-week treatment
period, patients receiving the 40mg dose experienced clinically
meaningful improvements in epistaxis frequency, duration and
epistaxis-free days. Regression of HHT-associated vascular lesions
was also observed.
Following the 12-week randomised double-blind period, patients
from selected study centres were enrolled into a 12-month OLE to
the study where they all receive up to 40mg VAD044 daily. Interim
data for twenty-nine patients through the 6 month timepoint
continue to show favourable safety and tolerability profiles with
further improvements in epistaxis.
Dr. Hanny Al-Samkari, the Peggy
S. Blitz Endowed Chair in Hematology/Oncology at Massachusetts
General Hospital and Associate Professor of Medicine at
Harvard Medical School (USA), co-primary investigator in the VAD044
POC trial, commented, "In this pioneering clinical trial we see
already at 12 weeks substantial and clinically meaningful
dose-dependent improvements in HHT disease activity with once-daily
VAD044, particularly as measured by epistaxis parameters."
Dr. Hans-Jurgen Mager,
pulmonologist and head of the Netherlands Reference Centre for HHT
at St. Antonius Hospital, Utrecht
(NL), also co-primary investigator in the VAD044 POC trial, added,
"These exciting results have supported the assessment of long-term
treatment of HHT patients with VAD044. We have added an open-label
extension to the POC trial and already see that after 6 months
of continuous treatment with VAD044 patients experience further
improvements in all epistaxis endpoints compared to those seen at
12 weeks. It seems that VAD044 has not yet reached its peak effect
on HHT disease activity at 12 weeks, and patients continue to
improve over time without paying an unexpected price in terms of
safety or tolerability."
Nicholas Benedict, CEO and
co-Founder of Vaderis Therapeutics, commented, "The excitement
surrounding the results of the initial 12-week double-blind part of
this trial is amplified by the continued improvements experienced
by patients through 6 months. Excellent collaboration with patient
and physician organisations such as CureHHT has been a cornerstone
of successful implementation of this ground-breaking trial which
was achieved in a much shorter timeframe than planned. Vaderis is
currently interacting with major health authorities to plan the
pivotal phase of development for VAD044 in HHT."
About Vaderis
Vaderis is a clinical stage biotech company developing
treatments for rare and orphan diseases associated with vascular
malformations. There is a significant number of debilitating and
largely untreated rare diseases, such as HHT (Hereditary
Haemorrhagic Telangiectasia), in which patients have overactivation
of AKT triggered by upstream genetic mutations resulting in
vascular overgrowth. Vaderis is developing VAD044, a daily, oral
allosteric AKT inhibitor, which has been investigated in a clinical
proof of concept study in HHT patients and is currently in a 12
month Open Label Extension. There are no drugs approved to treat
HHT and Vaderis aims to be the first company to develop a medicine
for the treatment of HHT and other diseases associated with
vascular malformations.
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www.vaderis.com
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SOURCE Vaderis Therapeutics AG