Ataluren Update July 2012 – July 11th, 2012

Summary of trial results

The pivotal Phase 2b trial of ataluren, an investigational new drug being studied in nonsense mutation Duchenne/Becker muscular dystrophy (DBMD), was completed in late 2009. It had enrolled 174 patients at 37 trial sites in 11 countries on four continents. As PTC and statistics experts have analyzed the complex data from this trial using various statistical methods, the company has come to understand that the results are very promising.


The trial had low-dose ataluren and high-dose ataluren arms as well as a placebo arm. The main criterion, or endpoint, used to determine differences in performance among the three arms was the Six Minute Walk Test, which measured, every six weeks, the distance that participants could walk in 6 minutes (6MWD).

• Participants receiving low-dose ataluren (10, 10, 20 mg/kg) showed a clinically meaningful difference (approximately 30 meters) in the change in their 6MWD results when compared to the placebo arm.

• The FDA requires that trial sponsors specify in advance which statistical models will be used to analyze data at the end of a study. The data was first analyzed using the pre-specified statistical method. The unexpected complexity of the ataluren data could not be fully addressed using this method, so it was necessary to use a different (ie, post hoc) statistical model to fully understand the results.

o A post hoc statistical analysis, using a model suggested by independent statistics experts, showed an average difference between low-dose ataluren and placebo of 31.3 meters (103 feet) in the 6MWD. The p-value for this result is 0.0561. A p-value is a test of statistical significance that measures how likely it is that a result is due to an actual effect rather than to chance. In this case, the post hoc corrected p value of 0.0561 indicates that there is only about a 5% possibility that the results could have been obtained if ataluren was no different than placebo.

A second measure used to compare the effects of ataluren at the two different doses and against placebo was the time it took for the 6MWD to worsen by 10% and remain so. This was defined as “time to persistent 10% worsening.” This analysis indicated that patients receiving low-dose ataluren experienced slower disease progression than patients receiving placebo. By the end of the trial, 26% of the low-dose patients were persistently 10% worse in 6MWD than when they started, compared to 48% in the high-dose arm and 44% in the placebo arm (post hoc p=0.0652 for low-dose ataluren vs placebo).

• The 6MWD results from the high-dose (20, 20, 40 mg/kg) arm were similar to the placebo arm. At the completion of the preliminary data analysis, all patients in three ongoing DBMD clinical trials (Phase 2a and 2b extension studies and a non-ambulatory study) were receiving the high dose. Although an independent data monitoring committee agreed that ataluren was well tolerated by patients, the committee recommended that the trials be suspended because all patients in ongoing trials were on the high dose.



Safety results showed that ataluren was generally well tolerated:

Adverse events were similar across all three arms of the study: high-dose, low-dose and placebo.

No patients discontinued treatment due to an adverse event.

Serious adverse events were infrequent and none was considered to be related to ataluren.

Current status

Extension Studies

  • In the US, PTC is providing access to ataluren to 106 previous trial participants through an open-label study in which all patients receive ataluren (10, 10, 20 mg/kg). Safety data is being collected. Enrollment in this trial is complete and patients have been receiving ataluren for at least six months.
  • In Canada and nine other countries that had Phase 2b trial sites, PTC has initiated an open-label study, collecting safety and efficacy data. Several patients have already been enrolled and are now receiving drug. Many sites are in various stages of obtaining national and local regulatory permission and completing paperwork to enroll patients over the next several months.

Regulatory Path

  • PTC remains committed to the development and commercialization of ataluren and is engaged in discussions with regulatory authorities in the US and Europe regarding the path forward for ataluren in nonsense mutation Duchenne/Becker muscular dystrophy.

• The FDA feedback is that the efficacy data from the single Phase 2b trial is not adequate to support approval at this time.

When the FDA is presented with data from a single pivotal trial, even for a rare disease, the data has to meet a particularly high standard. Discussions with the FDA regarding ataluren and the path forward are still ongoing.

The FDA considers a p-value of 0.05 to be the standard criterion for statistical significance. This means that there is no more than a 5% chance that the trial results were due to chance and not to an actual drug effect. The corrected post-hoc analysis showed a p-value of 0.0561 for the comparison of low-dose ataluren vs. placebo in the 6MWD.

The ataluren Phase 2b study was the first registration-directed trial ever conducted in DBMD and it was not known what the clinical endpoints, some of which had never been used before, would show when measuring the impact of a disease-modifying therapy.

Several other factors contributed to the difficulty of obtaining more robust evidence of efficacy in this trial, including the variability in the natural history of DBMD, the requirement to pre-specify the statistical analysis in the absence of a previous trial that could act as a model, and the limitations of doing trials in rare diseases.

The most likely outcome of our FDA discussions is that a pre-approval confirmatory study will be necessary.

• The company is simultaneously in discussions with the European Medicines Agency (EMA) concerning the path forward in Europe. It is possible that we will be able to file in the European Union under the mechanism of conditional approval, in which approval is granted with the requirement to conduct a confirmatory study.

PTC is engaged in the formal scientific advice process of presenting data from the trial to selected regulatory officials for their input. The precise timing of the filing, however, will depend on the outcome of our upcoming meetings. We are encouraged by discussions with the EMA thus far.

Confirmatory study

  • Whether in the context of conditional approval in Europe, or to meet additional requirements in the US, PTC is currently planning to conduct an additional confirmatory study of low dose ataluren (10, 10, 20 mg/kg) vs. placebo.

1.  This study design will be based on the Phase 2b trial results and the goal is to confirm the previous findings in the low-dose patient group.

2.  PTC is in discussions with clinical investigators and regulatory agencies about the specific design and endpoints for this trial and will update the community when a protocol is finalized.

  1. It is important to realize that a confirmatory study is often more restrictive in its inclusion/exclusion criteria than the original trial, as it is intended to confirm specific results. The goal of the clinical trial is to secure approval of ataluren so all patients who might benefit from ataluren may have access.
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Cure Duchenne is an Official Charity of the Run Disney Tinker Bell Half Marathon – June 11th, 2012

NEWPORT BEACH, Calif.–(BUSINESS WIRE)–CureDuchenne,   a nonprofit that raises awareness and funds to find a cure for Duchenne  muscular dystrophy, is proud to be an official charity of the Tinker Bell Half Marathon. The Tinker Bell Half Marathon weekend is January 18-20, 2013.

Disneyland® Resort becomes Never Land for a magical weekend. It starts with a Family 5K and a Kids’ Races and culminate with 13.1 mile run that weaves through Disneyland® Resort. Registration for the CureDuchenne Crusaders team begins next week.  For more information, please call CureDuchenne at 949-872-2552.

“CureDuchenne is excited to participate in the Tinker Bell Half Marathon,” said Debra Miller, founder and CEO of CureDuchenne. “The CureDuchenne Crusaders team will help raise awareness and money to help fund promising research for the 300,000 boys worldwide with Duchenne.”

Duchenne is a devastating muscle disease that impacts 1 in 3,500 boys. Boys are usually diagnosed by the age of 5, in a wheelchair by 12 and most don’t survive their mid-20s.

About CureDuchenne

CureDuchenne, a national nonprofit organization located in Newport Beach, Calif., is gaining international attention for its efforts to raise funds and awareness for Duchenne – a devastating and lethal muscle disease in children. One in every 3,500 male births results in a child  being afflicted with the disease. More than 24,000 boys are living with the disease in the United States alone, and most will not survive their mid-20s.

The funds CureDuchenne raises support the most promising research projects aimed at treating and curing the disease with the help of its distinguished panel of Scientific Advisors from around the world.  To date, seven research projects have made their way into human clinical trials with support from CureDuchenne. This accelerated push to move research from the lab into clinical trials could save the lives of those afflicted and give them hope for halting the progress of the disease.  Very few health-related nonprofits have been as successful in being a  catalyst for human clinical trials.



CureDuchenne Karen Harley, 949-872-2552

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Study DMD114876: A Clinical Study to Assess Two Doses of GSK2402986 in Subjects with Duchenne Muscular Dystrophy (DMD) – June 1st, 2012




The following clinical study of the investigational compound GSK2402968 being conducted in the US has recently achieved the milestone of all investigators eligible to screen subjects.

Fourteen investigators are participating in the following cities: Sacramento CA; Stanford, CA; Pensacola, FL; Iowa City, IA; Kansas City, KS; Baltimore, MD; Minneapolis, MN; St. Louis, MO; New York City, NY; Durham, NC; Cincinnati, OH; Columbus, OH; Portland, OR and Dallas TX.


Details regarding all active investigator sites are currently available on the websites and   If would like to learn more about the study, please contact the sites or contact GSK Clinical Support at either GSKCLINICALSUPPORTHD@GSK.COM or 877-379-3718.


Pertinent Inclusion Criteria:

  • Males with DMD with a mutation correctable by skipping exon 51**
  • 5 years old or older
  • Ambulatory
  • Use of an oral glucocorticoid for a minimum of 6 months immediately prior to screening

**Mutations correctable by exon 51 skipping include (but may not be limited to) deletion of exons 13-50, 29-50, 43-50, 45-50, 47-50, 48-50, 49-50, 50, 52, and 52-63.

The complete inclusion/exclusion criteria and study endpoints are posted on the websites and .



John E. Kraus, MD, PhD                                                Padraig Wright, MD, PhD                       


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Italfarmaco S.p.A joins EU-funded EndoStem consortium to support clinical study with Givinostat in Duchenne Muscular Dystrophy – May 28th, 2012

 Paris (France), May 2012The EndoStem consortium (, a large scale scientific collaboration coordinated              by Dr David Sassoon (UPMC/Inserm, Paris, France), and co-financed by the European Commission via the 7th Framework Programme, has welcomed Italfarmaco (Milano, Italy) into the consortium and project as a full partner, to augment the number of clinical trials being performed to treat muscle disorders by targeting endogenous stem cells with novel biotherapeutics.

As part of their participation and collaboration, Italfarmaco is initiating clinical trials using the HDAC inhibitor Givinostat, to treat Duchenne Muscular dystrophy. Patients with muscular dystrophy suffer debilitating muscle loss that gets worse as they age.

Dr. Pier Lorenzo Puri, associate professor at Sanford-Burnham and Italy’s Dulbecco Telethon Institute, has performed a preclinical trial using HDAC inhibitors showing the promoting effect in regeneration in dystrophic animal models


1,2. His team demonstrated that exposure to HDACi forces muscle resident progenitors to adopt the myogenic lineage. The data also supported the efficacy of the HDAC inhibitor Givinostat (from Italfarmaco) in promoting regeneration of dystrophic muscles while reducing the formation of fibrotic scars and fat deposition. This treatment was largely more effective than the treatment with the control HDAC inhibitor.

Based on clinical data, Givinostat was previously granted orphan drug designation by EMA for the treatment of Polycythemia Vera, a blood disorder in which the bone marrow makes too many red blood cells, and for Systemic-onset Juvenile Idiopathic Arthritis demonstrating its safety in humans including children. Given the suitability of Givinostat for treatment of paediatric patients, the collaboration is moving forward with the planning of a clinical trial in DMD patients. An important aim of this clinical trial is to correlate the beneficial effects of molecules used and their effect on endogenous stem cells.


The joint effort of Endostem and Italfarmaco is the fuel for accelerating the translation of pre-clinical data generated by my group at the Dulbecco Telethon Institute/Fondazione Santa Lucia into a clinical trial with boys affected by Duchenne Muscular Dystrophy”.” says Dr. Puri.

“Italfarmaco is fortunate and proud to join Endostem, the ideal partner for effectively moving to the development of Givinostat in such debilitating diseases as muscle disorders.” says Dr Mascagni Pre- Clinical R&D Senior Director from Italfarmaco.

“Italfarmaco is a perfect fit for the work encompassed in Endostem as they complement extremely well the work going on in and among the existing partners in the consortium,” says  Dr Sassoon.


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Summit Corporation PLC : Summit Announces Initiation of Phase 1 Trial for the Treatment of Duchenne Muscular Dystrophy – May 25th, 2012

OXFORD, UNITED KINGDOM–(Marketwire – May 25, 2012) – Summit (AIM:SUMM), a UK drug discovery                                                                               company, today announced that it has dosed the first cohort of patients in a Phase 1 study of SMT C1100 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal, rare genetic disease characterized by rapidly worsening muscle weakness.    SMT C1100, an oral small molecule compound, is a potential disease-modifying drug that works to increase, or upregulate, the amount of a naturally occurring protein called utrophin.

“There is currently no known cure for DMD, and the only treatments available mask the symptoms of the disease,” said Glyn Edwards, Chief Executive Officer of Summit. “SMT C1100 has the potential to modify the underlying disease, and the initiation of this Phase 1 trial represents a great step forward in bringing our breakthrough science to patients suffering from DMD. We expect to report top-line data from the full trial before the end of this year.”

SMT C1100 has been extensively evaluated in non-clinical efficacy and safety studies and has demonstrated its ability to restore and maintain the function of muscles. This Phase 1 dose-escalating clinical trial in healthy volunteers will evaluate if the Company’s aqueous formulation of SMT C1100 can provide the consistent levels of drug in blood that non-clinical efficacy studies predicted would be required to confer therapeutic benefit in DMD patients, while also assessing its safety and tolerability. A successful outcome from this trial is expected to lead to a Phase 2 trial of SMT C1100 in DMD patients.

The Phase 1 trial is being supported by $1.5 million from a group of US-based DMD organisations: the Muscular Dystrophy Association, Charley’s Fund, Cure Duchenne, the Foundation to Eradicate Duchenne, Nash Avery Foundation and Parent Project Muscular Dystrophy.

SMT C1100 is designed to upregulate and maintain the production of utrophin. Utrophin is a protein that is highly expressed in regenerating muscle, but decreases as the muscle fibre matures and is eventually replaced by dystrophin, a protein that maintains the integrity and healthy function of muscles. Patients with DMD are unable to make dystrophin, resulting in muscle fibre degeneration. However, if utrophin is continually expressed in the mature muscle fibre, it can replace the function of dystrophin and thereby overcome the deficit in patients with DMD. This approach is expected to be a universal treatment for all DMD patients regardless of whether the disease was caused by an inherited or spontaneous genetic mutation. Summit has demonstrated in non-clinical efficacy studies that SMT C1100 is capable of increasing utrophin to restore and maintain the healthy function of muscles.

About Summit

Summit is an Oxford, UK based drug discovery Company with an innovative Seglin™ technology platform for the discovery of new medicines and a portfolio of drug programme assets. Summit’s programme portfolio consists of a number of drug programmes targeting high-value areas of unmet medical need including Duchenne Muscular Dystrophy and C. difficile infection. Summit is listed on the AIM market of the London Stock Exchange and trades under the ticker symbol SUMM. Further information is available at

Forward Looking Statements

This document contains “forward-looking statements” within the meaning of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as “anticipates”, “intends”, “plans”, “seeks”, “believes”, “estimates”, “expects” and similar references to future periods, or by the inclusion of forecasts or projections. Forward-looking statements are based on the Company’s current expectations and assumptions regarding our business, the economy and other future conditions. Because forward-looking statements relate to the future, by their nature, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. The Company’s actual results may differ materially from those contemplated by the forward-looking statements. The Company cautions you therefore that you should not rely on any of these forward-looking statements as statements of historical fact or as guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements and regional, national, global political, economic, business, competitive, market and regulatory conditions.

Notes to Editors

About DMD

Duchenne muscular dystrophy is a fatal genetic neuromuscular disorder that affects 1 in 3,500 boys with an estimated patient population of 50,000 in the developed world. The disease is caused by defects in the gene required to make dystrophin, a protein, which maintains the integrity and healthy function of muscles. One in three new cases is due to a spontaneous mutation where there is no familial history of the disease. The progressive muscle wasting begins in early childhood and typically leads to death in the twenties due to cardiac and respiratory failure. Currently there is no cure for the disease.

About Utrophin Upregulation

Utrophin is a naturally occurring protein that has a similar function to dystrophin. Utrophin is produced during foetal muscle development but is switched off in mature muscle fibres. If its production could be switched back on, utrophin could act as a substitute for the missing dystrophin to maintain the healthy function of muscles. One method of turning utrophin production back on is through pharmacological means. Utrophin upregulation will be beneficial to all DMD patients regardless of their specific genetic mutation and is also expected to be complimentary to other therapeutic approaches in development.

About SMT C1100

Discovered and developed by scientists at Summit, SMT C1100 has demonstrated its potential as a disease-modifying drug in non-clinical efficacy studies. SMT C1100 disengages normal utrophin control such that utrophin RNA and protein is made continually in muscle. It has received orphan drug designation in the US and Europe.

About MDA Venture Philanthropy (MVP)

MVP is the Muscular Dystrophy Association’s drug development program, which operates within MDA’s translational research program. MVP is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases. For more information, visit and follow MDA on Facebook ( and Twitter (@MDAnews).

About Charley’s Fund, Cure Duchenne, Foundation to Eradicate Duchenne, and Nash Avery Foundation

Charley’s Fund (, Cure Duchenne (, Foundation to Eradicate Duchenne (, and Nash Avery Foundation are independent organisations devoted to developing treatments for Duchenne muscular dystrophy. These groups, founded by parents of children with Duchenne, support the most promising research.

About Parent Project Muscular Dystrophy

Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. Our mission is to end Duchenne. We accelerate research, raise our voices in Washington, demand optimal care for all young men, and educate the global community. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information,

This announcement is distributed by Thomson Reuters on behalf of Thomson Reuters clients. The owner of this announcement warrants that:

(i) the releases contained herein are protected by copyright and    other applicable laws; and

(ii) they are solely responsible for the content, accuracy and     originality of the information contained therein.

Source: Summit Corporation PLC via Thomson Reuters ONE [HUG#1614856]

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A Day To Rejoice For Duchenne Families- AVI Announces Top Line Results for Exon Skipping Trial – April 2nd, 2012

As parents of a son with Duchenne muscular dystrophy, we feel blessed every day we spend with our sons.  It’s not every day, however, that we have reason to rejoice about the future.  Today is one of those days.  AVI Biopharma announced the top line results from their phase 2 trial for Eteplirsen, a drug which induces exon skipping in patients amenable to a skip of exon 51.

Chris Garabedian, CEO of AVI gave the the top line results which showed that after 24 weeks, four patients  on the 30 mg. dose had an average  increase of dystrophin of 22.5%.  The patients that had received the 50 mg. dose for 12 weeks did not show an increase in dystrophin, suggesting that duration of treatment is important.

I will always caution families, we still don’t know if exon skipping will actually become a real treatment for our sons with Duchenne, or to what extent it will help them.  On today’s conference call announcing these results, AVI’s chief medical officer, Ed Kaye, referred to “reasonable” dystrophin needed to function as 20%, so this study does indeed give us proof of concept and  hope that we might have a treatment sometime in the future.

Dr. Eric Hoffman at Children’s National Medical Center in Washington DC. spearheaded a collaboration with CNMC along with  the Foundation to Eradicate Duchenne and CureDuchenne several years ago.  This collaboration gave critical funding to AVI at a very crucial time which allowed them to move forward with the preclinical work neccessary to progress into this phase 2 human clinical trial.  Dr. Jerry Mendell and Dr. Kevin Flanigan at Nationwide Children’s Hospital in Columbus, OH, have played a pivotal role in conducting this trial over the last several months. We are very grateful to these investigators and organizations for their vision and dedication.

What Comes Next?

There’s a song named “Two Out Of Three Ain’t Bad.”  We now have reasonable data that suggests the drug is safe, there were no adverse events seen.  And, we know that the drug can produce dystophin, and at a reasonable amount.  Now, the big question is whether this quality/quantity of dystrophin will result in meaningful outcome measures and really help Duchenne patients live longer and healthier lives.

All patients including the placebo group  that were on the phase 2 are now on the open label extension study at either the 30 mg. or 50 mg. dose.  They will stay on this dose until they complete a total of 48 weeks dosing, at which time a biopsy will be taken and clinical outcome measures tested.  In the meantime, AVI will be actively involved with the FDA, compiling a brief and planning the pivotal phase 3 study.  Simutaneously, more animal studies will be conducted.  Since the FDA will most likely not accept the presence of dystrophin alone as justification to move forward with Eteplirsen, this extension phase and the data they gather will be very important.  The first patient to be dosed began in August of 2011, which would make this data available later this year.

As we await the clinical outcomes of the extension trial, AVI will be busy looking to the future, and CureDuchenne is equipped and ready to support their subsequent research on additional exons and next generation exon skipping compounds.  CureDuchenne will always fund research projects that show more immediate potential to help our Duchenne sons, such as already approved FDA drugs to slow the progression.  But, CureDuchenne will never take it’s eye off of the prize…to correct the actual defect that causes this terrible disease.  CureDuchenne is committed to fund research, like exon skipping, that will replace the missing dystrophin protein, while we fund more immediate research that could get FDA approved or off target drugs to keep our sons going in order to take advantage of these higer impact drugs.

CureDuchenne was also the first US organization to  support Prosensa, another exon skipping biotech company.  Prosensa was able to leverage our funds and received a committment of up to $650 million to develop their drugs and  is in the process of recruiting patients for their phase 3 pivotal trial.

Two out of three may not be bad, but it’s not enough.  So please join me as we rejoice in the possibility of an effective treatment, while at the same time show restraint with our expectations, since so many drugs do fail, even at late stage trials.  One thing is for sure…we will need all of you to support these research efforts…


If you would like to learn more about our exon skipping efforts, please call me at 949-872-2552

Debra Miller

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AVI BioPharma muscular dystrophy trial meets goal – April 2nd, 2012

 30mg dose raises dystrophin 22.5 pct in 24 weeks

* No improvement seen in clinical outcomes

By Deena Beasley

April 2 (Reuters) – A small trial of U.S.-based AVI BioPharma Inc’s experimental muscular dystrophy drug met its goal of increasing levels of a key protein, but did not demonstrate that the drug had an impact on walking ability or other clinical endpoints.

The mid-stage trial, or Phase IIb, which involved 12 boys with Duchenne muscular dystrophy, found that the four patients treated for 24 weeks with a 30 mg dose of the drug, eteplirsen, had a 22.5 percent average increase in levels of dystrophin, a protein essential to building muscle, compared to no increase for four patients treated with a placebo.

Duchenne muscular dystrophy — a muscle degeneration disease affecting young children, almost exclusively boys — leads to paralysis and death in young adulthood. At least 250,000 people worldwide are believed to be afflicted with the disease.

Eteplirsen is designed to silence a specific strand of RNA so that the body can produce the dystrophin that people with Duchenne muscular dystrophy are unable to produce.

The trial found that the four patients treated for 12 weeks with a 50 mg dose of the drug did not show a significant increase in dystrophin, suggesting that a longer duration of dosing is needed.

It also found no significant improvements in clinical outcomes, including a six-minute walk test, in the treated groups compared to placebo.

“We anticipate that these levels of dystrophin could lead to significant clinical benefit if maintained over a longer course of treatment,” Dr. Jerry

Mendell, director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children’s Hospital in Columbus, Ohio and the study’s lead investigator, said in a statement.

There were no treatment-related adverse events, no serious side effects, and no evidence of kidney toxicity.

AVI BioPharma Chief Executive Officer Chris Garabedian said the company plans to discuss with U.S. regulators how best to conduct a longer pivotal trial of the drug.

“The hope is that if we can delay or halt the progression of the disease we would be able to avoid these kids ending up in a wheelchair,” he said. “It would avoid the need for ventilators, maybe even prevent death.”

Eteplirsen is designed to skip a specific “exon,” or protein-coding sequence of the dystrophin gene that is mutated in about 15 percent of people with Duchenne muscular dystrophy.

AVI BioPharma is also developing drugs targeting other exon mutations responsible for muscular dystrophy.

Garabedian said he would prefer raising funding for a pivotal trial from capital markets, rather than a partnership.

“In the rare disease area, it doesn’t take a large commercial infrastructure to do this,” he said.

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AVI BioPharma Announces Conference Call and Webcast on Monday, April 2, 2012, to Discuss Top-Line Data Results From the Phase IIb DMD Study – March 30th, 2012

BOTHELL, WA–(Marketwire – March 30, 2012) – AVI BioPharma, Inc. (NASDAQ: AVII),  a developer of RNA-based therapeutics, today announced it will hold a conference call at 8:00 a.m.  EDT (5:00 a.m. PDT) on Monday, April 2, 2012 to discuss top-line results from its Phase IIb study evaluating eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD).                                                                          

The conference call may be accessed by dialing 800.561.2718 for domestic callers and 617.614.3525 for international callers. The passcode for the call is 99858553. Please specify to the operator that you would like to join the “AVI BioPharma Phase IIb Top-Line Data Results Call.” The conference call will be webcast live under the events section of AVI’s website at and will be archived there following the call for 90 days. Please connect to AVI’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About AVI BioPharma
AVI BioPharma is focused on the discovery and development of novel RNA-based therapeutics for rare and infectious diseases, as well as other select disease targets. Applying pioneering technologies developed and optimized by AVI, the Company is able to target a broad range of diseases and disorders through distinct RNA-based mechanisms of action. Unlike other RNA-based approaches, AVI’s technologies can be used to directly target both messenger RNA (mRNA) and precursor messenger RNA (pre-mRNA) to either down-regulate (inhibit) or up-regulate (promote) the expression of targeted genes or proteins. By leveraging a highly differentiated RNA-based technology platform, AVI has built a pipeline of potentially transformative therapeutic agents, including eteplirsen, which is in clinical development for the treatment of Duchenne muscular dystrophy, and multiple drug candidates that are in clinical development for the treatment of infectious diseases. For more information, visit
AVI Investor and Media Contact:
Erin Cox

AVI Media Contact:
David Schull
858.717.2310 or 212.845.4271

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All of us at Cure Duchenne are so grateful to our supporters who are spreading the word about Duchenne!                                                                                      Duchenne muscular dystrophy is the most common muscle disease in children.  One if 3500 boys will be  born with this disease, it knows no ethnic, geographical or economic boundaries.

This disease can be cured and there are treatments that are in development that can save these children.  But, this disease is too big for the families of Duchenne boys to conquer on their own.  We need the support of everyone who cares about children to help us win this battle with Duchenne.

Please send this video ( to your contacts and Facebook friends and encourage them to send it on.  Use the image on this page as your Facebook  timeline banner and ask your friends to donate $10 on their cell phone by texting “SAVE” to 80888.

Please, help our children that suffer from Duchenne.




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Prosensa’s Clinical Development Plan Update – March 2012 – March 22nd, 2012

In parallel to the on-going development of the lead compound PRO051/GSK2402968, which is being developed by GSK with an active involvement of Prosensa, there are five more compounds for Duchenne in Prosensa’s pipeline.  Below you can find an update about the current state of development of these compounds.

Prosensa is currently conducting a clinical study to investigate the effect of PRO044 in boys with Duchenne muscular dystrophy, who have a dystrophin gene mutation amenable to an exon 44 skip. This study is a phase I/IIa study that will assess the safety, tolerability, efficacy and pharmacokinetics of PRO044. The inclusion/exclusion criteria, as well as the endpoints and investigator locations are available on (search terms ‘Duchenne and Prosensa’ or ‘PRO044’).

Prosensa has four additional compounds in development that target exons 45, 53, 52 and 55 (PRO045, PRO053, PRO052 and PRO055). These programs currently are in the pre-clinical development phase. It is anticipated that the company will be able to progress PRO045 and PRO053 into the clinic in the second half of 2012. Discussions are on-going with the regulatory authorities to ensure optimum development. Each trial will be posted at the website www.clinicaltrials.govas soon as the study design is finalized and all the required approvals of authorities and ethics committees are granted.

It is always difficult to predict timelines for drug development. Therefore, please keep in mind that expected start and end dates are based on predictions and could be delayed if unforeseen events occur.

Prosensa is very committed to developing safe and effective treatments and we will do our utmost to succeed herein. Our goal is to develop compounds for as many patients as feasible who may benefit from the exon skipping platform.

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