news!

http://www.santhera.com/index.php?docid=212&vid=&lang=en&newsdate=200807&newsid=1238090&newslang=en

Published: 07:00 24.07.2008 GMT+2 /HUGIN /Source: Santhera
Pharmaceuticals Holding AG /SWX: SANN /ISIN: CH0027148649

July 24, 2008: Santhera Receives First Product Approval: Health Canada
Approves Catena® for Treatment of Friedreich's Ataxia

Santhera Pharmaceuticals (SWX:SANN), a Swiss specialty pharmaceutical
company focused on neuromuscular diseases, announced today that Health
Canada has approved with conditions SNT-MC17/idebenone for the treatment
of Friedreich's Ataxia. Health Canada's decision is the first marketing
authorization worldwide for any Friedreich's Ataxia therapy. The new
drug will be marketed in Canada under the brand name Catena®. Santhera
has established a wholly owned Canadian subsidiary, Santhera
Pharmaceuticals (Canada), Inc. recently incorporated in Montréal,
Quebec, to address the needs of Canada's several hundred patients with
this rare and severely progressive muscle disease. Launch of Catena is
anticipated for the end of October 2008.

In clinical studies submitted to Health Canada as part of the approval
process, Catena showed statistically and clinically relevant
improvements in Friedreich's Ataxia patients, as measured by Activities
of Daily Living scores as well as cardiac and neurological functions.
The approved product labeling allows for the treatment of symptoms of
Friedreich's Ataxia. Two doses are approved: a starting dose of
450 mg/day for patients below 45 kg body weight and 900 mg/day for
patients above 45 kg body weight whereby treating physicians have an
option to dose up to 1,350 mg/day for patients below 45 kg body weight
and up to 2,250 mg/day for patients of more than 45 kg body weight if
needed. Under the conditions of the Notice of Compliance with
Conditions, Santhera has agreed to submit additional data from its
ongoing phase III clinical trial in the United States to confirm the
efficacy of the therapy.

Klaus Schollmeier, Chief Executive Officer of Santhera said: "We are
very pleased about this market authorization by the Canadian authority.
Health Canada concluded that the data presented were promising enough to
allow Catena's approval for the benefit of patients now while requiring
confirmatory efficacy data when they become available. Today's approval
is a major milestone for the Friedreich's Ataxia community in Canada and
elsewhere. For the first time, physicians will be able to offer patients
an approved, safe and efficacious therapy to treat their devastating
disease." He continued: "This first marketing authorization marks a
significant event for our Company, one that has been our goal since the
inception of Santhera in 2004. Today, Santhera's vision of offering
therapies for orphan indications is becoming reality. The entire team at
Santhera is energized by this success and is even further encouraged to
work towards approvals in other regions."

MJ Roach, VP Marketing & Sales and Santhera's General Manager for North
America, commented: "Canada's marketing authorization provides an
excellent foundation to establish a medical and marketing platform in
North America for the treatment of rare neuromuscular diseases in
general and for Friedreich's Ataxia in particular. The Canadian market
will also provide valuable insights for launching the product in the
United States, once the ongoing clinical trial is completed and the
product is approved. We look forward to working with physicians in
Canada who prescribe Catena to Friedreich's Ataxia patients."

Update on ongoing phase III clinical trial

In the United States, the IONIA (Idebenone effects On Neurological ICARS
Assessments) phase III trial has currently enrolled 41 patients. It was
agreed with the US Food and Drug Administration under a Special Protocol
Assessment process to recruit a minimum of 51 patients but to include
more patients if available. Given the current prospects for patient
availability, Santhera and its US clinical investigators believe that
the final study will include about 60 to 65 patients.

Conference call

At 19.00 CET / 18.00 UKT / 13.00 EST on July 24, 2008, Santhera will
host a conference call. Anyone interested in participating may join the
teleconference facility using the following dial-in in Switzerland
+41 52 267 07 36. The conference call will be recorded for playback and
is available one hour after the conference call ends and for 20 days
under +41 52 267 07 00 (reference no. 668713).

About Friedreich's Ataxia

Friedreich's Ataxia is a rare but severe genetic neuromuscular disorder
that results in the degeneration of an individual's nerve and muscle
tissue. This disorder causes loss of muscle control, uncoordinated
movements, muscle wasting and thickening of heart walls which frequently
leads to a shortened life span. Friedreich's Ataxia affects both
Caucasian males and females equally and it is estimated that about
20,000 patients suffer from the disease in both North America and
Europe. Average life expectancy for Friedreich's Ataxia patients is
limited to approximately 35 to 50 years.

The disorder results from a genetic defect in the gene encoding for
frataxin. Reduced levels of this protein ultimately result in impaired
energy production in mitochondria, the cells' energy production centers,
and elevated oxidative stress. Tissues that have the highest need for
energy, in particular nerve and cardiac tissues, are primarily affected
by frataxin deficiency resulting in pathological changes in heart muscle
anatomy and function and loss of nerve cells.

About Catena®

Catena may be useful in the symptomatic management of patients with
Friedreich's Ataxia. The drug is believed to increase the supply of
energy to cells in the body. Additionally it has antioxidant properties
and may protect the cells in the body which are damaged by the disease.

Catena® is a trademark of Santhera Pharmaceuticals, registered in Canada
and the United States.

About Notice of Compliance with Conditions

A Notice of Compliance with Conditions (NOC/c) is an authorization to
market a drug in Canada issued by Health Canada, indicating that the
sponsor has agreed to undertake additional studies to confirm the
clinical benefit of the product. A market authorization under the NOC/c
policy allows Health Canada to provide earlier market access to
potentially life-saving drugs. Eligibility for an NOC/c is restricted to
promising new drug therapies intended for the treatment, prevention or
diagnosis of serious, life-threatening or severely debilitating diseases
or conditions for which a) there is no alternative therapy available on
the Canadian market or, b) where the new product represents a
significant improvement in the benefit/risk profile over existing
products. Once a sponsor provides satisfactory evidence of the drug's
clinical effectiveness, and all the conditions agreed upon have been
met, Health Canada will remove the conditions associated with market
authorization in favor of a full approval.

* * *

About Santhera

Santhera Pharmaceuticals (SWX:SANN) is a Swiss specialty pharmaceutical
company focused on the discovery, development and marketing of
small-molecule pharmaceutical products for the treatment of severe
neuromuscular diseases, an area of high unmet medical need which
includes many orphan indications with no current therapy. Santhera
currently investigates three compounds in five clinical-stage
development programs. The Company's first product, SNT-MC17 (INN:
idebenone) has received a marketing approval with conditions from Health
Canada to treat Friedreich's Ataxia and will be marketed under its brand
name Catena. The product is also under review by health authorities in
the EU and in Switzerland for the same indication, while in the United
States a pivotal phase III trial is recruiting patients.
SNT-MC17/idebenone has also shown efficacy in a phase II clinical trial
as a potential treatment for the indication Duchenne Muscular Dystrophy.
For further information, please visit www.santhera.com.

For further information, contact

Klaus Schollmeier, Chief Executive Officer

Phone: +41 (0)61 906 89 52

klaus.schollmeier@santhera.com

Barbara Heller, Chief Financial Officer

Phone: +41 (0)61 906 89 54

barbara.heller@santhera.com

Thomas Staffelbach, Head Public & Investor Relations

Phone: +41 (0)61 906 89 47

thomas.staffelbach@santhera.com

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 Approval Canada

more on A0001

Below are two emails... one from Paul Konanz and the other from US FARA
president Ronald Bartek
-----------------------------------------------------

Hello all,

I'd like to follow Paul's excellent comments with some information FARA
has obtained about the A0001 trial. This information reinforces Paul's
points about the format of this trial as compared with the Idebenone
trial and may help answer some of your questions.

The phase I trial of A0001, as stated in the release, is in healthy
volunteers. It is being conducted by a Contract Research Organization
(CRO) that was chosen by Penwest Pharmaceuticals and with which FARA has
no dealings. This is, by the way, the more typical way phase I trials
are conducted (by CROs in healthy volunteers that they have on their
rolls). I believe phase I of the Idebenone trial was conducted,
atypically, in FA patients primarily because Idebenone had already been
"in a lot of people." Idebenone had been tried at low doses in a number
of other diseases such as Parkinson's, Huntington's and Alzheimer's, and
had been taken by a lot of healthy people as an anti-aging or
brain-function aid. So, the FDA agreed the Idebenone phase I could be
in FA patients. Similarly, the iron chelator Deferiprone and EPO have
both been "in a lot of people" with other indications, so the phase I
work, or pilot studies, in those two drugs have been conducted in FA
patients.

A0001, however, has not been "in a lot of people" so the FDA suggested
beginning with the typical phase I approach - in healthy
volunteers. FARA has not seen the protocol for this phase I study of
A0001 and would not usually need to see it. My understanding is,
though, that carrier status would usually disqualify a potential
participant. I believe healthy participants are usually young students
with no involvement at all with the disease in question. CROs normally
recruit the healthy volunteers very quickly from their own rolls and ads
and have no trouble doing so. This A0001 phase I trial, for example, is
to use only 60 healthy volunteers and 10 of them were dosed on the very
first day (last Friday).

As Paul states, phase I of this trial, like all phase I trials, will
focus on safety. Like the phase I of Idebenone, it will look at safety
in a dose-escalating manner and will attempt to identify the maximum
tolerated dose. Data from this phase I trial will be used to establish
safety of A0001 in humans and will help instruct selection of the best
doses to use in the phase II in FA patients.

We will ALL be VERY much involved in the phase II of the trial, of
course, and FARA will let everybody know about timing and
inclusion/exclusion criteria, etc. We are in constant contact with the
drug companies involved, on a daily basis, and will be working very hard
to accelerate their timelines. FARA's Scientific Advisors and Board of
Directors meet at the end of next week and we will review, based on
reports we are now receiving from all the drug companies, the schedules
and timelines for all the clinical trials currently underway and coming
up. Of course, we will let everybody know the results and how you can
help.

In preparation for all these clinical trials, please make sure all FA
patients are signed up in the FARA Patient Registry and for FARA's
electronic bulletins and newsletters.

Hope this helps.

Warm regards to all,

Ron

(Ron & Raychel; Keith-22-FA; Byron-24-clear; Stuart-19-carrier)

Ronald J. Bartek
President
Friedreich's Ataxia Research Alliance (FARA)
P. O. Box 1537
Springfield, VA 22151
Tel (703) 426-1576
FARA website: http://www.CureFA.org

Email: fara@CureFA.org

Please register in the FARA Patient Registry at
http://www.curefa.org/registry/

and for e-news at
http://visitor.constantcontact.com/email.jsp?m=1101190303489

--- On Wed, 7/16/08, Paul Konanz wrote:

From: Paul Konanz

Subject: [FAPG] Edison A-0001 Phase I Trial Participants
To: fapg@fortnet.org, internaf@yahoogroups.com
Date: Wednesday, July 16, 2008, 10:11 PM

Reminder on the Edison A-0001Phase I Trial

It is wonderful news that one more possible FA treatment is starting FDA trials, with the announcement of the Edison A-0001 Phase I trial.
One note is that this Phase I trial is to completely focus on safety in human subjects using non-FA-involved participants. In the press release
it says, "The phase I clinical trial just initiated by Penwest Pharmaceuticals will be conducted in healthy volunteers...".

For sure this means no FA'ers will be involved and very probably no carriers (parents, siblings, etc) either. I have little information
on the protocol at this time (this trial is not on the NIH trial page). Looking at the Idebenone Phase I trial as an example can give you a feel
for what is involved in a safety-testing-only trial. That trial was split into an A and B sections, with A being a single dose to volunteers with
close scrutiny for some time after the dose, and B was several doses up to 75mg/Kg/day for one month with the same close scrutiny.
DO NOTE FOR IDEBENONE THEY USED FAers.
They aren't with the A-0001, according to the press release. If you are interested, the Idebenone Phase IB trial description can be found here,
http://www.clinicaltrials.gov/ct2/show/NCT00078481?term=Friedreich%27s+Ataxia&rank=4

So, please don't try to volunteer for this A-0001 trial if you think you have one or more bad FA-related genes. Several of the FA
organizations have received many inquiries but have little information because they are not involved and we don't qualify anyway. :-)

These organizations did comment how they appreciated the interest and willingness of the families to so quickly contact them!
It demonstrated how closely tied in to advances in research we are.

Regards,

Paul

new trial

Hi,
I received this today, and thought I would put in onto the blog. More
hope:->

FOR IMMEDIATE RELEASE
First Human Dose Initiated of Promising New Friedreich's Ataxia Drug
Highlights Effectiveness of New Public-Private Partnership Model
July 15, 2008 – The Friedreich's Ataxia Research Alliance (FARA) joins
its
public and private partners in announcing that a phase I clinical trial
of a
promising new drug, designated A0001, began dosing on July 11th. This
milestone achievement illustrates the power of a new model for advancing
therapies,
especially in rare diseases.
"FARA believes in the essential nature of public-private partnerships
that
involve government agencies such as the National Institutes of Health
(NIH)
and the Food and Drug Administration (FDA), academic investigators, the
pharmaceutical industry, and multiple non-profits like FARA and the
Muscular
Dystrophy Association (MDA)," said FARA President Ron Bartek. "FARA is
grateful to
its partners that span the spectrum from discovery through clinical
development as we work together to advance promising compounds like
A0001 toward the
goal of approved treatments and cures for Friedreich's Ataxia (FA) and
other
neurodegenerative disorders."
A0001 is a small molecule that has the potential for treating defects in
the
mitochondrial respiratory chain like those believed to cause
significant
damage in FA and a number of other neurological and neuromuscular
disorders. In
April 2006, the FDA granted orphan drug designation status to A0001 for
treatment of inherited mitochondrial respiratory chain diseases.
The opening of this clinical trial demonstrates the power of the
public-private partnerships FARA has been fortunate to build and bring
to bear on the
quest for treatments and a cure for FA. FARA began its support of A0001
in 2005
with substantial research grants to and investment in Edison
Pharmaceuticals. The MDA, via Seek A Miracle/MDA, joined FARA in
co-funding the initial
research grant to Edison.
"We all know that drug development is expensive, but this is a
wonderful
example of how investments can be leveraged by multiple groups to get
more
therapies into the clinic," says Dr. Sharon Hesterlee, MDA VP for
Translational
Research.
Later in 2005, Dr. Robert Wilson of the University of Pennsylvania, FARA
and
Edison joined forces as co-applicants to the National Institutes of
Health
(NIH) program called RAID (Rapid Access to Intervention Development),
designed
to help move drugs from the discovery bench to the clinical trial
bedside.
The application was successful and contract services provided by NIH
RAID
played an important role in completing the preclinical preparations of
A0001 so
the molecule could be made ready for the clinical trial.
"This is a great example of how the various stakeholders joined forces
to
help transform a promising laboratory discovery into a therapeutic that
is now
in clinical trial," says Dr. Story Landis, NINDS Director. The NINDS
manages
the NIH RAID program and is also responsible for Friedreich's Ataxia
research
as part of its mission to reduce the burden of neurological disorders.
NINDS'
support for the A0001 pre-clinical project consisted of formulation, a
genotoxicity study, a dose escalation study, synthesis of radiolabeled
drug for
ADME and radioautography studies, dose ranging studies in two species, a
90
day GLP toxicity study, and safety pharmacology studies.
The phase I clinical trial just initiated by Penwest Pharmaceuticals
will be
conducted in healthy volunteers and is designed to evaluate the safety
and
tolerability of A0001 at various doses, as well as to collect
pharmacokinetic
data. In addition, the trial is designed to determine if there is a
maximum
tolerated dose of the drug. This data will be used in subsequent phases
of
clinical trials in patients with FA and other diseases that involve
mitochondrial dysfunction.
For futher information, contact FARA at (703) 426 1576 or
fara@cureFA.org.
About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3),
non-profit,
charitable organization dedicated to accelerating research leading to
treatments and a cure for Friedreich's ataxia. _http://www.CureFA.org_
(http://rs6.net/tn.jsp?e=001DQArLW-C12A11Wn2SeP7Eoa6C_5LfbBm8R71dsUTUwdT9025__vFTV3HpqIbKxe
5Pjni7cG7HJjgpAdeMrfBgGceJYdw01kOAu71BttT6tU=)

(http://rs6.net/tn.jsp?e=001DQArLW-C12A11Wn2SeP7Eoa6C_5LfbBm8R71dsUTUwdT9025__vFTV3HpqIbKxe5Pjni7cG7HJjgpAdeMrfBgGceJYdw01kOAu71BttT6tU=)

interesting medical article

This came in this morning. It talks about a new idea regarding gene
inhibitors.

http://www.sciencedaily.com/releases/2008/07/080706194259.htm

New Targets For RNAs That Regulate Genes Identified

ScienceDaily (July 6, 2008) — Tiny strands of genetic material called
RNA -- a chemical cousin of DNA -- are emerging as major players in gene
regulation, the process inside cells that drives all biology and that
scientists seek to control in order to fight disease.

The idea that RNA (ribonucleic acid) is involved in activating and
inhibiting genes is relatively new, and it has been unclear how RNA
strands might regulate the process.

In a new study available online today and in a future issue of Nature
Structural and Molecular Biology, RNA experts at UT Southwestern Medical
Center found that, contrary to established theories, RNA can interact
with a non-gene region of DNA called a promoter region, a sequence of
DNA occurring spatially in front of an actual gene. This promoter must
be activated before a gene can be turned on.

"Our findings about the underlying mechanisms of RNA-activated gene
expression reveal a new and unexpected target for potential drug
development," said Dr. David Corey, professor of pharmacology and
biochemistry at UT Southwestern and one of the senior authors of the
study.

Genes are segments of DNA housed in the nucleus of every cell, and they
carry instructions for making proteins. Faulty or mutated genes lead to
malfunctioning, missing or overabundant proteins, and any of those
conditions can result in disease. Scientists seek to understand the
mechanisms by which genes are activated, or expressed, and turned off in
order to get a clearer picture of basic cell biology and also to develop
medical therapies that affect gene expression.

In previous studies, Dr. Corey and Dr. Bethany Janowski, assistant
professor of pharmacology at UT Southwestern and a senior author of the
current study, have shown that tiny strands of RNA can be used to
activate certain genes in cultured cancer cells. Using strands of RNA
that they manufactured in the lab, the researchers showed that the
strands regulate gene expression by somehow perturbing a delicate
mixture of proteins that surround DNA and control whether or not genes
are activated.

Until now, however, it was not clear exactly how the synthetic RNA
strands affected that mix of regulating proteins.

In the current study, also carried out in cancer cell cultures, the UT
Southwestern research team discovered an unexpected target for the
manufactured RNA. The RNA did not home in on the gene itself, but rather
on another type of RNA produced by the cell, a so-called noncoding RNA
transcript. This type of RNA is found in association with the promoter
regions that occur in front of the gene. Promoter regions, when
activated, act essentially as a "start" command for turning on genes.

The researchers found that their man-made RNA strand bound to the RNA
transcript, which then recruited certain proteins to form an RNA-protein
complex. The whole complex then bound to the promoter region, an action
that could then either activate or inhibit gene expression.

"Involvement of RNA at a gene promoter is a new concept, potentially a
big new concept," Dr. Janowski said. "Interactions at gene promoters are
critical for understanding disease, and our results bring a new
dimension to understanding how genes can be regulated."

Until recently, many scientists believed that proteins alone control
gene expression at promoters, but Drs. Corey and Janowski's results
suggest that this assumption is not necessarily true.

"By demonstrating how small RNAs can be used to recruit proteins to gene
promoters, we have provided further evidence that this phenomenon should
be in the mainstream of science," Dr. Corey said.

Although using synthetic RNA to regulate gene expression and possibly
treat disease in humans is still in the future, Dr. Corey noted that the
type of man-made RNA molecules employed by the UT Southwestern team are
already being used in human clinical trials, so progress toward the
development of gene-regulating drugs could move quickly.

Other researchers from UT Southwestern involved in the research were
lead author and student research assistant Jacob Schwartz; student
research assistant Scott Younger; and research associate Ngoc-Bich
Nguyen. Researchers from the University of Western Ontario and ISIS
Pharmaceuticals also participated.

The research was supported by the National Institutes of Health and the
Welch Foundation.

________________________________________________________________________
Adapted from materials provided by UT Southwestern Medical Center.