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Idorsia initiates MODIFY, a Phase 3 registration study to assess lucerastat as a potential new treatment option for patients with Fabry disease

Idorsia Pharmaceuticals Ltd. /
Idorsia initiates MODIFY, a Phase 3 registration study to assess lucerastat as a
potential new treatment option for patients with Fabry disease
. Processed and transmitted by Nasdaq Corporate Solutions.
The issuer is solely responsible for the content of this announcement.

* Idorsia to host an investor webcast to discuss the Phase 3 program today at
14:00hrs CEST

Allschwil, Switzerland - May 16, 2018
Idorsia Ltd (SIX: IDIA) today announced that the first patient has been enrolled
in a registration study to investigate the effect of lucerastat, as an oral
monotherapy, for the treatment of adult patients with genetically confirmed
Fabry disease, irrespective of their genetic mutation type.

MODIFY will recruit over 100 patients from 29 trial sites across 9 countries.
Its primary endpoint is a reduction in neuropathic pain, described as feeling
like burning, shocks or shooting, stabbing, tingling, and/or pins and needles
primarily in the hands and feet. This major symptom is reported by many patients
with Fabry disease as significantly impacting their daily activities and quality
of life, despite existing treatment.

Dr. Derralynn Hughes, DPhil, FRCP, FRCPath and EU Coordinating Investigator,
"Today's news is an important milestone for the Fabry research and patient
communities that have contributed to the development of this study. Pain is a
genuine and pressing unmet need of the Fabry patient population. Pain remains a
significant burden for many patients, even for some of those who are already
being treated with enzyme replacement therapy. Lucerastat represents an exciting
potential new oral treatment option to address this."

Fabry disease is a rare, life threatening, inherited lysosomal storage disorder
in which a particular lipid, called globotriaosylceramide (Gb3), accumulates in
cells of many organs of the body. This build-up results in cellular dysfunction
leading to a range of signs and symptoms from neuropathic pain (pain primarily
in the hands and feet), stomach, skin and eye problems, to hypertension,
progressive kidney damage, cardiomyopathy, and stroke. New treatment options are
needed to treat the underlying mechanism of the disease and provide symptomatic

Martine Clozel, MD and Chief Scientific Officer, commented:
"Idorsia's preclinical data indicate that lucerastat has the potential to treat
patients with Fabry disease, regardless of their specific gene mutation type.
MODIFY will include patients who were never treated with enzyme or patients who
stopped enzyme replacement therapy. In parallel to MODIFY, we will also run a
pediatric study to assess lucerastat in children aged from 2 to 18 years.
Lucerastat is therefore a potential new oral treatment option for a very broad
spectrum of patients living with Fabry disease."

Guy Braunstein, MD and Head of Global Clinical Development, added:
"We have worked closely with patients during the development of the MODIFY
protocol for lucerastat. We conducted an international patient survey to better
understand the symptoms of patients with Fabry disease, and validated a patient
reported outcome instrument to specifically assess Fabry neuropathic pain, in
accordance with health authority guidance."

About the MODIFY study
MODIFY is a multicenter, double-blind, randomized, placebo-controlled, parallel-
group study to determine the efficacy and safety of lucerastat oral monotherapy
in adult patients with Fabry disease. The study aims to determine the effect of
study treatment on neuropathic pain during 6 months of treatment, measured with
Idorsia's validated Fabry disease pain instrument. At the end of the double-
blind period, patients will be given the possibility to enter in an open label
extension study. Approximately 108 patients are expected to be enrolled,
randomized in a 2:1 ratio to either lucerastat or placebo. The study is expected
to run for around 20 months.

Notes to the editor

About Fabry disease
Fabry disease is a rare, life threatening, genetic disorder involving a
deficiency or dysfunction of alpha-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 may result in a build-up of Gb3 deposits
throughout the body, particularly in the kidneys, heart and nervous system.

The symptoms range from neuropathic pain (primarily pain in the hands and feet),
stomach, skin and eye problems, to hypertension, progressive kidney damage,
cardiomyopathy and stroke. Since the symptoms are non-specific, Fabry disease is
often undetected or misdiagnosed. As the disease is progressive, early diagnosis
is essential to manage the symptoms as soon as possible and reduce the risk of
developing serious complications.

The median prevalence of diagnosed Fabry disease is 1.0 per 100,000 in males and
1.9 per 100,000 in females. As the gene responsible for Fabry disease is found
on the X chromosome (of which males have one, and females two), males with
deleterious mutations have little or no residual alpha-GalA activity. Therefore,
these male patients with Fabry disease experience a wider spectrum of symptoms,
and in some cases, a greater severity. It is now widely accepted that women with
Fabry disease are heterogeneous with respect to disease severity and may
sometimes also develop life threatening complications of the disorder. Up to
70% of female carriers develop Fabry related symptoms at some point in their

Current treatment approaches for Fabry disease include enzyme replacement
therapy (ERT). ERT requires intravenous infusions every two weeks to replace the
deficient enzyme in the cells with a genetically engineered form. Migalastat is
the other therapy that has been granted marketing authorization in the EU and
Japan as an oral monotherapy for the long-term treatment of a subset of patients
with Fabry disease 16 years and older who have an amenable mutation. Other
treatments are aimed at alleviating individual symptoms, such as opioids for
severe pain. In advanced Fabry disease, hemodialysis and kidney transplantation
may be necessary.

Data supporting lucerastat in Fabry disease
Idorsia preclinical research with a mouse model of Fabry disease has shown that
glucosylceramide synthase inhibition with lucerastat reduces the accumulation of
Gb3 in kidney and certain nerve endings. Furthermore, Idorsia has shown that
lucerastat lowers Gb3 in cultured cells from patients with Fabry disease of both
sexes harboring different GLA genetic mutation types.

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
received lucerastat 1000 mg b.i.d. for 12 weeks on top of enzyme replacement
therapy and four patients with Fabry disease received 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 GCS and provides alpha-
GalA substrate reduction with a fast onset in adult patients with Fabry disease
on ERT.

The results of the exploratory clinical study, together with confirmation of a
favorable tolerability profile, and the preclinical evidence suggest that
lucerastat has the potential to provide safe oral therapy for adult patients
with Fabry disease, independent of their mutation or phenotype.

About Dr. Derralynn Hughes DPhil, FRCP, FRCPath
Dr Derralynn Hughes is a senior lecturer in hematology at University College
London, UK, and has clinical responsibilities in the area of hematology and
lysosomal storage disorders at the Royal Free London NHS Foundation Trust. She
directs the research program in the lysosomal storage disorders unit research
laboratory and is principal investigator of a number of clinical trials,
registries and observational studies. Dr Hughes studied medicine at Oxford
University in the UK, and joined the research group in the Sir William Dunn
School of Pathology as an MRC training fellow, writing a doctoral thesis in the
area of macrophage biology. Her interest in the role of the macrophage in
inflammatory and developmental processes has endured and now, she has focused
her laboratory research interests towards the role of inflammatory cell
interactions in the pathophysiology of the lysosomal storage disorders Gaucher
and Fabry disease. Major laboratory projects are currently aimed at
understanding the pathophysiology underlying Gaucher-related bone pathology,
increased incidence of malignancy in Gaucher disease and phenotypic variation in
Anderson-Fabry disease.


* Politei JM, et al. Pain in Fabry Disease: Practical Recommendations for
Diagnosis and Treatment. CNS Neurosci Ther. 2016; 22:568-76.
* Deegan PB, et al. Fabry disease, enzyme replacement therapy and the
significance of antibody responses. J Inherit Metab Dis. 2012; 35:227-43.
* Biegstraaten M, et al. Recommendations for initiation and cessation of
enzyme replacement therapy in patients with Fabry disease: the European
Fabry Working Group consensus document. Orphanet J Rare Dis. 2015; 10:36.
* Guérard N, et al. Lucerastat, an iminosugar for substrate reduction therapy:
tolerability, pharmacodynamics, and pharmacokinetics in patients with Fabry
disease on enzyme replacement. Clin Pharmacol Ther. 2017;103:703-11.
* Guérard N, et al. Lucerastat, an iminosugar for substrate reduction therapy:
pharmacokinetics, tolerability, and safety in subjects with mild, moderate,
and severe renal function impairment. J Clin Pharmacol. 2017;57:1425-31.
* Guérard N, et al. Lucerastat, an iminosugar with potential as substrate
reduction therapy for glycolipid storage disorders: safety, tolerability,
and pharmacokinetics in healthy subjects. Orphanet J Rare Dis.
2017; 12(1):9 doi: 10.1186/s13023-017-0565-9.
* Welford R, et al. Lucerastat, an iminosugar for substrate reduction therapy
in Fabry disease: preclinical evidence. Molecular Genetics and Metabolism.
2017;120 (Abstract 360): S139.

Investor webcast
An investor conference call and webcast will be held to discuss the Phase 3
program. The call will start with presentations by senior management, followed
by a Q&A session (live access to the speakers).

Date:     Wednesday May 16, 2018
Time:    14:00 CEST | 13:00 BST | 08:00 EDT

Webcast participants should visit Idorsia's website www.idorsia.com 10-15
minutes before the webcast is due to start.
Conference call participants should start calling the number below 10-15 minutes
before the conference is due to start.

Dial-in:            CH: +41 44 580 65 22  |  UK: +44 203 009 24 70  |  US:
+1 877 423 08 30

About Idorsia
Idorsia 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 Europe's leading biopharmaceutical company, with a
strong scientific core.

Headquartered in Switzerland - a biotech-hub of Europe - Idorsia is specialized
in the discovery and development of small molecules, to transform the horizon of
therapeutic options. Idorsia has a broad portfolio of innovative drugs in the
pipeline, an experienced team, a fully-functional research center, and a strong
balance sheet - the ideal constellation to bringing R&D efforts to business

Idorsia was listed on the SIX Swiss Exchange (ticker symbol: IDIA) in June 2017
and has over 600 highly qualified specialists dedicated to realizing our
ambitious targets.

For further information please contact:
Andrew C. Weiss
Senior Vice President, Head of Investor Relations & Corporate Communications
Idorsia Pharmaceuticals Ltd, Hegenheimermattweg 91, CH-4123 Allschwil
+41 (0) 58 844 10 10

The above information contains certain "forward-looking statements", relating to
the company's business, which can be identified by the use of forward-looking
terminology such as "estimates", "believes", "expects", "may", "are expected
to", "will", "will continue", "should", "would be", "seeks", "pending" or
"anticipates" or similar expressions, or by discussions of strategy, plans or
intentions. Such statements include descriptions of the company's investment and
research and development programs and anticipated expenditures in connection
therewith, descriptions of new products expected to be introduced by the company
and anticipated customer demand for such products and products in the company's
existing portfolio. Such statements reflect the current views of the company
with respect to future events and are subject to certain risks, uncertainties
and assumptions. Many factors could cause the actual results, performance or
achievements of the company to be materially different from any future results,
performances or achievements that may be expressed or implied by such forward-
looking statements. Should one or more of these risks or uncertainties
materialize, or should underlying assumptions prove incorrect, actual results
may vary materially from those described herein as anticipated, believed,
estimated or expected.

Press Release PDF:

This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.

Source: Idorsia Pharmaceuticals Ltd. via GlobeNewswire

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