Santhera and Parent Project Muscular Dystrophy (PPMD) Announce Results of Benefit / Risk Survey in patients with Duchenne Muscular Dystrophy (DMD)
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Santhera Pharmaceuticals Holding AG /
Santhera and Parent Project Muscular Dystrophy (PPMD) Announce Results of
Benefit / Risk Survey in patients with Duchenne Muscular Dystrophy (DMD)
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Liestal, Switzerland, October 22, 2015 - Santhera Pharmaceuticals (SIX: SANN)
and Parent Project Muscular Dystrophy (PPMD), the leading U.S. advocacy
organization working to end Duchenne Muscular Dystrophy (DMD), announce the
results of a benefit/risk survey in patients DMD and parents of individuals with
DMD. The study focused specifically on patient and caregiver preferences for
treatments that address disease symptoms that are not directly related to
skeletal muscle function. PPMD has submitted the outcomes report of this survey
to the FDA in their continuous efforts to share patients' and caregivers' view
on emerging treatment options.
This first industry-supported benefit/risk study for patients with Duchenne
Muscular Dystrophy (DMD) was inspired and based on data from Santhera's
successful Phase III (DELOS) trial, which demonstrated a clinical benefit for
Raxone(®)/Catena(® )(idebenone) in delaying the loss of respiratory function in
patients with DMD not using concomitant glucocorticoid steroids. The results of
the benefit/risk study confirm that the preservation of respiratory function is
prioritized by caregivers and boys/men with DMD.
"Since entering the Duchenne space, Santhera has become a reliable partner and
proven both its dedication to this community and commitment to incorporate the
voices of patients into its regulatory submissions," PPMD's President, Pat
Furlong remarked. "We are proud having partnered with Santhera for this project,
as we see communities' treatment preference and risk tolerance as an important
aspect of FDA's decision-making process. This study provides us with data that
supports the need for patients to have access to medications that address the
pulmonary and cardiac complications of Duchenne and Becker muscular dystrophy."
"PPMD has been a pioneer in the rare disease community and has provided
groundbreaking leadership to ensure that patients have a voice in the regulatory
process," commented Thomas Meier, PhD, Chief Executive Officer of Santhera. "We
commend PPMD for elucidating the unique considerations of caregivers and boys
and men with DMD related to treatment of pulmonary disease associated with their
muscular dystrophy. As PPMD has already submitted the outcomes report of this
survey to the FDA, we will be able to make reference to it in the course of our
regulatory submission."
"We hear from parents and people with Duchenne or Becker muscular dystrophy that
they want faster drug approvals and regulatory agencies to accept more
uncertainty and risk," explained Holly Peay, PhD, lead investigator of the
survey and co-principal investigator of PPMD's Duchenne Connect registry. "But
it was vital to conduct a study to systematically assess the preferences and
priorities of a large number of patients and their parents."
Researchers from the Johns Hopkins Bloomberg School of Public Health provided
expertise in health economics and methodology for the study.
The results of this benefit/risk study were forwarded today by PPMD to the FDA
providing the Agency with important information on the treatment priorities and
risk tolerance of the DMD community for treatments that address disease symptoms
not directly related to skeletal muscle function. Through this study, the
regulators will have access to scientifically-robust data illustrating that
study participants value interventions to slow cardiac and pulmonary disease
progression highly, and would be willing to accept a higher risk of side effects
and treatment burden for pulmonary benefits.
About the PPMD Benefit/Risk Survey Study
The study consisted of three separate research activities to assess: (i)
priorities of treatment targets; (ii) treatment preferences and (iii) treatment
acceptance. Best/worst scaling was used for the survey design: respondents were
presented with a series of hypothetical treatment profiles and asked to choose
the best and worst features of each. One hundred fifty-five individuals
participated in this study. Most affected individuals had a diagnosis of
Duchenne (85%). The study included caregivers of patients who were at least 10
years old, as well as teenagers and adults.
Respondents prioritized treatment targets that address cardiovascular and
pulmonary benefits. The greatest priority was for treatments that address weaker
heart pumping. Treatments that address lung infections and cough strength were
rated as the second and third-most prioritized, respectively. Stratified samples
revealed that caregivers and patients had similar priorities.
The analysis of preference scores revealed greatest preference for treatments
that target cough strength. Patients and their caregivers demonstrated the
greatest preference for treatment that maintains cough strength underlining the
importance to maintain adequate airway clearance.
In the treatment acceptance analysis, in which participants answered whether
they would use a hypothetical drug if it were available, respondents almost
always chose to accept the treatment profile with a strong benefit and little
risk, while almost three quarters would accept a treatment with a strong benefit
despite the highest risk/burden profile.
Study investigators from PPMD and the Johns Hopkins Bloomberg School of Public
Health will disseminate detailed survey results to the patients and caregiver
community and scientific community through conferences and publications.
About Duchenne Muscular Dystrophy and DELOS
Duchenne Muscular Dystrophy (DMD) is one of the most common and devastating
types of muscle degeneration and results in rapidly progressive muscle weakness.
It is a genetic, degenerative disease that is inherited in an X-linked recessive
mode with an incidence of up to 1 in 3,500 live born males worldwide. DMD is
characterized by a loss of the protein dystrophin, leading to cell damage,
impaired calcium homeostasis, elevated oxidative stress and reduced energy
production in muscle cells. This results in progressive muscle weakness and
wasting and early morbidity and mortality due to respiratory failure. Idebenone
is a synthetic short-chain benzoquinone and a cofactor for the enzyme
NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial
electron transport, reducing and scavenging reactive oxygen species (ROS) and
supplementing cellular energy levels.
DELOS was a Phase III, double-blind, placebo-controlled trial which randomized
64 patients, 10-18 years of age, to receive either Raxone/Catena tablets or
matching placebo. The trial met its primary endpoint and demonstrated that
Raxone/Catena can delay the loss of respiratory function in patients not taking
concomitant glucocorticoid steroids. The positive outcome of the Phase III DELOS
study was published in The Lancet and presented at the 2015 American Academy of
Neurology meeting.
About Parent Project Muscular Dystrophy (PPMD)
Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive
nonprofit organization in the United States focused on finding a cure for
Duchenne Muscular Dystrophy - their mission is to end Duchenne. PPMD invests
deeply in treatments for this generation of people affected by Duchenne and in
research that will benefit future generations. They advocate in Washington, DC,
and have secured hundreds of millions of dollars in funding. They demand optimal
care, and strengthen, unite and educate the global Duchenne community.
Everything PPMD does - and everything they have done since their founding in
1994 - helps people with Duchenne live longer, stronger lives. For more
information, visit www.parentprojectmd.org.
About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company
focused on the development and commercialization of innovative pharmaceutical
products for the treatment of orphan mitochondrial and neuromuscular diseases.
Santhera's lead product Raxone(®) is authorized in the European Union for the
treatment of Leber's Hereditary Optic Neuropathy (LHON). Santhera develops
Raxone(®)/Catena(®) in two additional indications, Duchenne Muscular Dystrophy
(DMD) and primary progressive Multiple Sclerosis (ppMS), and omigapil for
Congenital Muscular Dystrophy (CMD), all areas of high unmet medical need. For
further information, please visit the Company's website www.santhera.com.
Raxone(®) and Catena(®) are trademarks of Santhera Pharmaceuticals.
For further information, contact:
Thomas Meier, PhD, Chief Executive Christoph Rentsch, Chief Financial
Officer Officer
Phone +41 61 906 89 64 Phone +41 61 906 89 65
thomas.meier(at)santhera.com christoph.rentsch(at)santhera.com
US investor contact: US Public Relations contact:
Hans Vitzthum, LifeSci Advisors, LLC Deanne Eagle, Planet Communications
Phone +1 212 915 2568 Phone +1 917 837 5866
hans(at)lifesciadvisors.com deanne(at)planetcommunications.nyc
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for
or purchase any securities of Santhera Pharmaceuticals Holding AG. This
publication may contain certain forward-looking statements concerning the
Company and its business. Such statements involve certain risks, uncertainties
and other factors which could cause the actual results, financial condition,
performance or achievements of the Company to be materially different from those
expressed or implied by such statements. Readers should therefore not place
undue reliance on these statements, particularly not in connection with any
contract or investment decision. The Company disclaims any obligation to update
these forward-looking statements.
# # #
News release PPMD:
http://hugin.info/137261/R/1960653/714789.pdf
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Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
[HUG#1960653]
Unternehmensinformation / Kurzprofil:
Datum: 22.10.2015 - 07:00 Uhr
Sprache: Deutsch
News-ID 428653
Anzahl Zeichen: 11831
contact information:
Town:
Liestal
Kategorie:
Business News
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