NeuroVive presents medical breakthrough in cellular energy regulation

NeuroVive, a leading mitochondrial medicine company, is presenting a
breakthrough in the company’s work on energy regulation at the cellular level.
Based on the UK company Selcia Ltd’s medical chemistry technology and
NeuroVive’s assays and knowledge of mitochondrial dysfunction, the parties have
produced an all-new class of experimental drug which potentially could be used
to treat genetic diseases and acute medical conditions that affect energy
production in the mitochondria. This project has been designated NVP015 “the
Amber Project” in-house. An extensive patent application on these compounds and
their potential usage was published in the WIPO (World Intellectual Property
Organization) database on April 10. The underlying scientific data for this
breakthrough will be presented for the first time at an international research
symposium in Pittsburgh on June 4 - 7.
In simplified terms, the mitochondria can be considered as the energy factory of
cells. There is currently scientific data indicating that many medical
conditions, from chronic diseases to acute conditions affecting the heart and
brain, are linked to their function. Succinic acid is a central component in
energy production. This compound is involved in many pathogenic changes to
energy production, which means that adding succinic acid can restore energy
production. However, succinic acid is normally unable to transit the cell wall
independently. NeuroVive’s and Selcia’s researchers have resolved this problem
by altering succinic acid in various ways, with the result that the compound is
able to transit the cell wall and then be freed up and utilized in mitochondrial
energy production.

Therapy areas and current development work
The new drug candidates may potentially be used to prevent or treat primary or
secondary mitochondrial conditions and restore normal function. This means that
if current development work is successful, they may be suitable to treat a large
number of rare genetic conditions, as well as more general disorders where there
is an increased need for energy. This dual application extends the potential for
this new class of pharmaceutical.

These new experimental drugs are currently undergoing formulation work. Their
efficacy and potential adverse events must be examined in animal studies before
they can be trialed on humans. The first major validation study on animals will
be conducted in 2014.

New medical analysis method for mitochondrial function in human cells
NeuroVive’s research team has also developed a medical analysis method for
energy regulation in human cells that enables the study of molecules and
pharmaceuticals that can directly increase or decrease energy production. This
method, called ToxPhos®, has the potential for future use to support the
diagnosis of mitochondrial disease and medical conditions. The method, although
not used to produce the ‘Amber’ compounds, was central in the screening process
of the energy-regulating experimental drugs and is the foundation for the
development of several preclinical models for mitochondrial conditions. The
patent application for this method was also published in the WIPO patent
database on 10 April.

”Research into mitochondrial medicine in recent years has demonstrated that far
more diseases and medical conditions may potentially be sourced from
mitochondrial defects than previously believed. That’s why it was a natural move
for us to start developing pharmaceuticals that not only protect the
mitochondria in cell damage, but also offer the potential for cells to produce
normal amounts of energy in injury and disease. These new experimental drugs
that we’re developing may potentially be used as emergency treatment in cellular
energy crisis in children with congenital limitations to energy production and,
potentially, for other conditions where raised energy production counters the
course of diseases. We’re really proud to be able to present the underlying
scientific discoveries at this symposium in June,” commented Eskil Elmér,
NeuroVive’s CSO.

“The new experimental drugs have the potential to address a market worth
billions of kronor and a substantial medical need in the treatment of rare
diseases that affect the mitochondria. This application has great potential to
secure orphan drug designation, which means a fairly speedy process prior to
market launch and a long period of market exclusivity. These diseases usually
affect children, which makes it extra urgent to start designing a suitable
formulation and begin preparation of preclinical safety testing required before
experimental drugs can be administered to patients in clinical trials,”
continued Mikael Brönnegård, NeuroVive’s CEO.

Presentation at an international symposium in June
These new experimental drugs and the underlying scientific discoveries,
including the new medical analysis method ToxPhos®, will be presented by
NeuroVive’s researchers at the United Mitochondrial Disease Foundation Symposium
in Pittsburgh on June 4-7. There’s more information on the conference at
www.umdf.org.

About NeuroVive
NeuroVive Pharmaceutical AB (publ), a leading mitochondrial medicine company, is
developing a portfolio of products to treat acute cardiovascular and
neurological conditions through mitochondrial protection. These medical
conditions are characterized by a pressing medical need and have no approved
pharmaceutical treatment options at present. NeuroVive’s products CicloMulsion®
(heart attack) and NeuroSTAT® (traumatic brain injury) are currently being
evaluated in phase III and phase II studies, respectively. NeuroVive’s research
programs also include products for the treatment of anti-viral indications
(Hepatitis B/C), brain cell injury in stroke patients, and drug candidates for
cellular protection and treating mitochondria-related energy regulation
diseases. NeuroVive’s shares are listed on NASDAQ OMX, Stockholm, Sweden.

Current status of NeuroVive’s products

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CicloMulsion®
NeuroVive’s product CicloMulsion® is the first cyclophilin inhibitor developed
for treating reperfusion injury. The product’s potential for treatment of
myocardial infarction is currently being evaluated in a clinical phase III
study. The final participant of a total of 972 patients was enrolled on 16
February 2014. The results of this study are scheduled for presentation in 2015
after a one-year follow-up is completed on all patients and study data has been
compiled.

NeuroSTAT®
NeuroVive is developing NeuroSTAT® for treating patients with severe traumatic
brain injury (TBI). NeuroSTAT® is currently being evaluated in a clinical phase
IIa study at Rigshospitalet, Copenhagen. The study is focusing on safety and
pharmacokinetics, and 5 of a planned 20 patients have been enrolled. The design
and planning work for a phase III study has commenced. The company has obtained
orphan drug designation for NeuroSTAT® for moderate to severe cranial injury in
the US and EU. Orphan drug designation confers market exclusivity for 7 years in
the US and 10 years in the EU from the date when the company secures marketing
authorization.

NVP018
The recently acquired cyclophilin inhibitors are part of a family of molecules
called Sangamides, and based on a new and unique chemical platform of what are
known as polyketides. NVP018 is NeuroVive’s primary drug candidate in the
company’s new portfolio of potent cyclophilin inhibitors. It has undergone
extensive preclinical development and has been developed for treating hepatitis
B/C. This product demonstrates potent efficacy against virus replication and has
a positive safety and pharmacokinetic profile. Cyclophilin inhibitors have the
potential for broad-based application, and NeuroVive is also currently
evaluating NVP018’s potential against other viral indications.

Other products
More information on all products developed by NeuroVive is available at
www.neurovive.se/index.php/en/research-development/our-products

For media and investor relations questions, please contact:

Ingmar Rentzhog, Laika Consulting, tel: +46 (0)46 275 62 21 or ir@neurovive.se
It is also possible to arrange an interview with NeuroVive’s CEO Mikael
Brönnegård via the contact above.

NeuroVive Pharmaceutical AB (publ)
Medicon Village, SE-223 81 Lund, Sweden
Tel: +46 (0)46 275 62 20 (switchboard), Fax: + 46 (0)46 888 83 48,
info@neurovive.se, www.neurovive.se

NeuroVive Pharmaceutical AB (publ) is required to publish the information in
this news release under The Swedish Securities Market Act. The information was
submitted for publication on 17 April 2014, at 8:45 a.m. CET.