Idorsia announces the results of MODIFY, a Phase 3 study of
lucerastat in Fabry disease
Ad hoc announcement pursuant to Art. 53 LR
Allschwil, Switzerland –
October 11,
2021Idorsia Ltd (SIX: IDIA) today announced that
MODIFY, the Phase 3 study to investigate the effect of lucerastat,
as an oral substrate reduction therapy for the treatment of adult
patients with Fabry disease, did not meet the primary endpoint.
Guy Braunstein, MD and Head of Global Clinical
Development at
Idorsia,
commented: “I’m very proud of
everyone involved with MODIFY for delivering a very high-quality
study, one of the largest in Fabry disease. Lucerastat was well
tolerated and biochemically it did exactly what we were expecting;
as previously seen, in this study we saw a substantial and
consistent reduction of plasma Gb3, confirming the pharmacological
activity of lucerastat. Despite this biological effect, no
reduction in neuropathic pain was observed after six months of
treatment, using the patient reported outcome tool.”
Guy Braunstein,
added:“In
MODIFY, many parameters have been collected and data are still
being analyzed. In addition, most patients chose to continue in the
open-label extension study and in a few weeks, we will see more
results that will inform our decision on the future of
lucerastat.”
Jean-Paul Clozel, MD and Chief Executive Officer of
Idorsia, commented:“Taking into account the quality of the
study, the volume of data we have collected, and some observations
made in the six-month double-blind placebo-controlled treatment
period, we need to wait for the results of the interim analysis of
the open-label phase before making a decision. I expect to be in a
position to share our future direction before the end of year.”
Notes to the editor
About the MODIFY study (NCT03425539)MODIFY was
a multicenter, double-blind, randomized, placebo‑controlled,
parallel-group study to determine the efficacy and safety of
lucerastat as an oral monotherapy in adult patients with Fabry
disease. MODIFY determined the effect of study treatment on
neuropathic pain during 6 months of treatment, measured with
Idorsia’s validated Fabry disease pain instrument. 118 patients
were randomized in a 2:1 ratio to either lucerastat or placebo. At
the end of the double-blind period, 107 patients entered into an
ongoing open label extension study (NCT03737214), which is
investigating the long-term safety and tolerability of lucerastat
oral therapy and to further evaluate its clinical efficacy on renal
and cardiac function, in adult patients with Fabry disease over a
period of up to a further 48 months.
About Fabry diseaseFabry disease is a rare,
genetic, lysosomal storage disorder that results in reduced or
absent α-galactosidase A (alpha-GalA) an enzyme that normally
breaks down a fatty product known as globotriaosylceramide (Gb3) in
the cells of the body. Over time, this results in an accumulation
of Gb3 deposits throughout the body, leading to progressive
pathophysiology in the nervous system, such as neuropathic pain
(pain primarily in the hands and feet), the gastrointestinal
system, and the cardiovascular system, as well as in organs,
including the kidneys, skin, ears, eyes, and lung. Symptoms of
Fabry disease affect a patient’s life expectancy and their quality
of life. There is an unmet need for a well-tolerated,
disease-modifying, oral treatment that addresses refractory
symptoms and can be used regardless of GLA mutation or previous
treatment.
Lucerastat in Fabry
diseaseLucerastat, a small molecule glucosylceramide
synthase inhibitor, is in development as a novel, substrate
reduction therapy for Fabry disease. Preclinical studies showed
that lucerastat is a soluble, bioavailable inhibitor of
glucosylceramide synthase that reduces the accumulation of
α-galactosidase A substrates in tissues affected by Fabry disease,
including kidneys, liver, and dorsal root ganglia. In clinical
pharmacology studies, lucerastat had reproducible pharmacokinetics,
characterized by rapid absorption, quick elimination, and no
evidence for saturation of absorption or elimination mechanisms.
Across Phase 1 studies, lucerastat doses up to 4000 mg were well
tolerated and the safety profile was not affected by concomitant
treatments.
The safety, tolerability, pharmacodynamics, and pharmacokinetics
of oral lucerastat were evaluated in an exploratory study in adult
patients with Fabry disease. In this single-center, open-label,
randomized study, 10 patients with Fabry disease were randomized to
lucerastat for 12 weeks on top of ERT and 4 to ERT only. A rapid
decrease in plasma Gb3, a marker of Fabry disease, and its
precursors was observed, demonstrating that lucerastat 1000 mg
b.i.d. inhibits glucosylceramide synthase and provides alpha-GalA
substrate reduction with a fast onset in adult patients with Fabry
disease receiving ERT.
About IdorsiaIdorsia Ltd is reaching out for
more – We have more ideas, we see more opportunities and we want to
help more patients. In order to achieve this, we will develop
Idorsia into a leading biopharmaceutical company, with a strong
scientific core.
Headquartered near Basel, Switzerland – a European biotech-hub –
Idorsia is specialized in the discovery, development, and
commercialization of small molecules to transform the horizon of
therapeutic options. Idorsia has a broad portfolio of innovative
drugs in the pipeline, an experienced team of professionals
covering all disciplines from bench to bedside, state-of-the-art
facilities, and a strong balance sheet – the ideal constellation to
translate R&D efforts into business success.
Idorsia was listed on the SIX Swiss Exchange (ticker symbol:
IDIA) in June 2017 and has over 1000 highly qualified specialists
dedicated to realizing our ambitious targets.
For further information, please contactAndrew
C. WeissSenior Vice President, Head of Investor Relations &
Corporate CommunicationsIdorsia Pharmaceuticals Ltd,
Hegenheimermattweg 91, CH-4123 Allschwil+41 58 844 10
10investor.relations@idorsia.commedia.relations@idorsia.com
www.idorsia.com
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