This is from Santhera's Feb 27th News release:

http://hugin.info/137261/R/1293632/293135.pdf

3. SNT-MC17/idebenone in Duchenne Muscular Dystrophy
The design of the Phase III program including the selection of primary and secondary endpoints was recently discussed with EMEA’s Scientific Advise Working Party as well as the US Food and Drug Administration (FDA) during a pre-IND meeting. Santhera currently plans for one single, placebo controlled pivotal Phase III trial with study centers in Europe, in the United States and Canada. Subject to finalization, the study protocol calls for a twelve-month treatment period with approximately 200 ambulatory and nonambulatory patients at the age of 10 to 18 years. Key objectives of the trial are to investigate the effects on respiratory and muscle strength parameters. Patient recruitment is anticipated to begin in summer of 2009. The randomization of the first patient will trigger a milestone payment from European marketing partner Takeda Pharmaceutical.

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April 27, 2009: Santhera Shareholders Approve all Board Proposals at Annual Shareholders' Meeting
News release 2009 ASM

http://www.santhera.com/index.php?docid=212&vid=&lang=en&am...

Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on neuromuscular diseases, today announced that its Annual Shareholders' Meeting has approved the Annual Report, the Annual Financial Statements and the Consolidated Financial Statements for 2008, as well as the proposed appropriation of the results. Shareholders also granted discharge to the members of the Board of Directors (Board) and management from liability. Jürg Ambühl was elected as new member of the Board while Michael Lytton, Hans Peter Hasler and Timothy Rink were re-elected. All motions passed with a vast majority. A total of 51.1% of the Company's share capital were represented at the Shareholders' Meeting.

Presentations to Shareholders
In his presentation to shareholders, Michael Lytton, Chairman of the Board, highlighted last year's successes. "2008 was a landmark year for Santhera as it became a product company. The launch of Catena® in Canada marks Santhera's transition from a development to an integrated specialty pharmaceutical company. With a commercial product in hand, we have laid the foundation for creating a sustainable business that will deliver novel neuromuscular therapeutics to orphan patient populations."

Klaus Schollmeier, Chief Executive Officer of Santhera, reported on the Company's significant achievements in 2008 and highlighted the successful launch of Catena® in Canada. Looking into 2009 and beyond, he said: "Santhera will generate important news flow in the upcoming months. This summer, we will report clinical data from our two core clinical development programs: the pivotal Phase III IONIA trial of Catena® for Friedreich's Ataxia in the United States, opening the path for regulatory submissions in the US and the EU; and the Phase IIb FJORD study of JP-1730/fipamezole for Dyskinesia in Parkinson's Disease, with the intention to partner this project for Phase III clinical development and subsequent commercialization. Another highlight of this summer will be the initiation of our pivotal Phase III trial with Catena® for the treatment of Duchenne Muscular Dystrophy. The outcome of these clinical trials will significantly shape the future of Santhera."
Thanks Ofelia for finding this one.
I was wondering when we would be hearing from Santhera. Idebenone looks good for heart and lung improvements in DMD, which can't be bad!!
These results are for Phase III in Friedreich's Ataxia not DMD.

May 19, 2009: Santhera's US Phase III IONIA Trial in Friedreich's Ataxia Misses Primary Endpoint

Data from second Phase III study expected in the first half of 2010

Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on orphan neuromuscular diseases, announced today that its US Phase III clinical trial evaluating Catena® for the treatment of Friedreich's Ataxia missed its primary endpoint as measured by the International Cooperative Ataxia Rating Scale (ICARS). The study also did not show statistical significance on the second neurological endpoint, the Friedreich's Ataxia Rating Scale (FARS). On both endpoints, the active treatment arms showed a consistent improvement over baseline and placebo, as seen in prior studies. However, due to a lower than expected effect size combined with the fact that patients on placebo improved unexpectedly, statistical significance could not be achieved in this study population. The safety results were consistent with published data suggesting that Catena® is safe and well tolerated at doses up to 2250 mg/day.

The primary endpoint in the IONIA (Idebenone effects On Neurological ICARS Assessments) study compared the effect at six months of two treatment arms with placebo on the baseline ICARS score. For both treatment arms, patients on Catena® improved on average by 2.4 points on the ICARS scale at six months over baseline. This is about half of the improvement seen in the prior US Phase II study named NICOSIA (NIH Collaboration With Santhera In Ataxia) which was used to design the IONIA study. Patients on Catena®, however, improved by only 1.2 points over placebo, because patients on placebo did not deteriorate to the extent expected from the NICOSIA study or as described in the literature. These phenomena combined to produce an effect size that did not reach statistical significance over the six-month study period.

Teleconference
At 15.00 CET / 14.00 UKT / 09.00 EST on May 19, 2009, Santhera's management will discuss the IONIA results at a teleconference. Anyone interested in participating may join using one of the following dial-ins (conference ID: 10747196):
Germany 0692 222 3479 (local call)
Switzerland 056 580 00 07 (local call)
United Kingdom 0871 700 0345 (national call) or +44 1452 555 566 (standard international)
United States 1866 966 9439 (free call)
The teleconference will be available for playback one hour after the presentation ends.


"We are of course tremendously disappointed by the IONIA results reported today," commented Klaus Schollmeier, Chief Executive Officer of Santhera. "The study met neither our expectations, nor those of the patients or the investigators. Everybody involved was highly motivated to demonstrate the drug's efficacy again. The results have not dampened our confidence in the drug's potential in Friedreich's Ataxia. Because of its larger patient population and longer study duration, we expect that the ongoing European Phase III MICONOS study will finally provide the efficacy data necessary to support marketing approval in the US and Europe."

The design of the ongoing European Phase III trial named MICONOS (Mitochondrial Protection With Idebenone In Cardiological Or Neurological Outcome Study) is different from the IONIA study reported today. The MICONOS study is a twelve-month trial of 232 predominantly adult patients with three active treatment arms against placebo. Enrollment was completed in December 2008 and results are expected in the first half of 2010. If positive, these results will form the basis of filings for a New Drug Application and a Marketing Authorization Application in the United States and Europe, respectively. As a consequence of the IONIA study results, the application for marketing approval earlier filed in Switzerland will be withdrawn.

Sue Perlman, Clinical Professor of Neurology at the University of California, Los Angeles and one of the two IONIA study investigators, comments: "I still strongly support the disease-modifying effect of Catena® in Friedreich's Ataxia. I believe it slows the progression of the neurological and cardiac aspects of this condition over time, and I strongly recommend that patients continue in the open-label extension study arm of the Phase III IONIA trial to enable us to gather as much longer term data as possible."

"Patients on drug improved over placebo on the primary endpoint (ICARS) in the IONIA study. This is supported by the second neurological endpoint (FARS) which also showed an improvement of treated patients over patients on placebo. In addition, a prespecified responder analysis of the active arms showed effect levels comparable to the NICOSIA study in which 60% of the treated patients showed a clinically meaningful improvement on ICARS. However, due to a larger than expected placebo response rate, statistical significance could not be achieved in this six month trial," said Thomas Meier, Chief Scientific Officer of Santhera. "Therefore, we are eager to see the additional data from the twelve-month MICONOS study as well as our two open-label extension studies. We remain confident in our development programs with Catena® in Friedreich's Ataxia and other neuromuscular and mitochondrial disorders and look forward to the upcoming data from several ongoing clinical trials."

About the IONIA Phase III trial
The IONIA (Idebenone effects On Neurological ICARS Assessments) trial was a double-blind, randomized, placebo-controlled Phase III study of six months duration investigating the efficacy, safety and tolerability of two doses of Catena® compared to placebo. The first dose group was 450 mg/day for patients below 45 kg body weight and a corresponding dose of 900 mg/day for patients above 45 kg body weight. The second dose group was 1350 mg/day for patients below 45 kg of body weight and 2250 mg/day for patients above 45 kg. 70 ambulatory Friedreich's Ataxia patients between the ages of 8 and 17 years were recruited into two clinical centers in the US - the Children's Hospital of Philadelphia and the School of Medicine of the University of California, Los Angeles.

The primary endpoint was the change in the International Cooperative Ataxia Rating Scale (ICARS), a neurological scale, where the difference between baseline and end of treatment for each of the dosing groups was compared with the change in the placebo group. The IONIA study also investigated Activities of Daily Living parameters (ADL) as well as additional neurological (FARS) and cardiac outcomes. The study incorporated advice provided by the US Food and Drug Administration under Special Protocol Assessment.

About the NICOSIA trial
In the Phase II study conducted in collaboration with the US National Institutes of Health named NICOSIA (NIH Collaboration With Santhera In Ataxia), Catena® showed a positive effect in particular on the ICARS as well as the ADL scales. Data from the NICOSIA study demonstrated the drug's significant potential to improve neurological functions after a six-month treatment [1]. 48 patients were recruited at the NIH into three active dosage arms against placebo.

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 North America and Europe. The 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.
Once again Ofelia I am glad to have your eyes out there to pull information in for review...even when the news isn't great ...like this one.

more waiting while I watch Alexander's disease progress. He's still "walking" but just the other night he mentioned how he is tired of trying to do it. I fear he will go off his feet this summer.
More bad news, Santhera is hoping to obtain better test results 10 mos from now in order to replace these recent bad results, however financially they are now in terrible shape because they were banking on idebenone. With no revenue coming in from selling idebenone they may not make it thru in the next 10 mos. Again this is for test results for F.Ataxia.
I wonder how/if this will affect the start of the DMD clinical trial scheduled for this Summer?


cheryl cliff said:
More bad news, Santhera is hoping to obtain better test results 10 mos from now in order to replace these recent bad results, however financially they are now in terrible shape because they were banking on idebenone. With no revenue coming in from selling idebenone they may not make it thru in the next 10 mos. Again this is for test results for F.Ataxia.
I haven't the foggiest but I suspect it won't go well. I do think we should try to contact somebody at Santhera and see if they can answer that question.

Ofelia Marin said:
I wonder how/if this will affect the start of the DMD clinical trial scheduled for this Summer?


cheryl cliff said:
More bad news, Santhera is hoping to obtain better test results 10 mos from now in order to replace these recent bad results, however financially they are now in terrible shape because they were banking on idebenone. With no revenue coming in from selling idebenone they may not make it thru in the next 10 mos. Again this is for test results for F.Ataxia.
I was able to listen the conference call. They think that the problem is not the drug but the study design. They noticed that the patients in the placebo group still improved after 6 mths (which is not consistent with the literature or the results from previous studies), hence the non-significant difference b/w treated and placebo groups.

I do not think that for DMD, patients 10-18 years old, one year study, they will see something like this. Some boys might not deteriorate in their cardiac and respiratory functions, but I do not think they can improve after 1 year.

They still plan to start the clinical trial for DMD this summer after possibly revising the study design; they are filling with authorities at the moment.



cheryl cliff said:
I haven't the foggiest but I suspect it won't go well. I do think we should try to contact somebody at Santhera and see if they can answer that question.

Ofelia Marin said:
I wonder how/if this will affect the start of the DMD clinical trial scheduled for this Summer?


cheryl cliff said:
More bad news, Santhera is hoping to obtain better test results 10 mos from now in order to replace these recent bad results, however financially they are now in terrible shape because they were banking on idebenone. With no revenue coming in from selling idebenone they may not make it thru in the next 10 mos. Again this is for test results for F.Ataxia.
Any idea where in the states will the trials will be scheduled?

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