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Amicus Therapeutics, Inc. (FOLD) Message Board

zwerp2000 84 posts  |  Last Activity: Oct 20, 2014 12:58 PM Member since: Apr 19, 1999
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  • Reply to

    nice upgrade for RNA

    by starfe11 Oct 20, 2014 12:38 PM
    zwerp2000 zwerp2000 Oct 20, 2014 12:58 PM Flag

    Really? RNA has less money, less coverage, much less institutional ownership, a failed drug and their major partner completely left them hanging. I will admit that they do a great job trying to hide the fact that their drug failed and had 80% AE when used but reality will eventually sit in. There is a reason why SRPT has more than double the market cap

  • Reply to

    nice upgrade for RNA

    by starfe11 Oct 20, 2014 12:38 PM
    zwerp2000 zwerp2000 Oct 20, 2014 12:44 PM Flag

    William Blair which has a $63 target? Oppenheimer with a $45 target? Deutch Bank? CS? which both have positive ratings on SRPT. Again, I am sure the parents and children appreciate what you are doing on this board.

  • EMA encourages applications for orphan designation for Ebola treatments
    The European Medicines Agency encourages developers of treatments or vaccines against Ebola to apply for orphan designation. Applications for orphan designation of Ebola medicines will be treated as a priority and EMA has committed to fast-tracking their evaluation, the regulator announced. Drug companies that are working on experimental Ebola vaccines and treatments include Tekmira (TKMR), Sarepta (SRPT), BioCryst (BCRX), Chimerix (CMRX), NewLink Genetics (NLNK) and GlaxoSmithKline (GSK)

  • zwerp2000 by zwerp2000 Oct 20, 2014 7:04 AM Flag

    In all the panic and controversy over Ebola, government and public-health authorities seem strangely oblivious to a potential remedy that deserves attention. Sarepta Therapeutics (SRPT), a biotech company in Cambridge, Mass., has a small supply of an experimental Ebola drug that has shown promising—if very preliminary—results in laboratory trials for safety and effectiveness.

    In private meetings, Sarepta has reminded various arms of the U.S. government that the drug, known as AVI-7537, is available in limited quantities—but so far to no avail. Making the situation all the more puzzling, the U.S. government in years past funded Sarepta’s research on Ebola, until the Department of Defense abruptly cut the program for budgetary reasons in 2012.

    Sarepta hasn’t kept any of this a secret. As recently as Sept. 30, the day that Thomas Eric Duncan tested positive for Ebola in Dallas, the company’s chief executive officer appeared on CNBC to discuss research in the area. Sarepta CEO Chris Garabedian said that with a modest amount of fresh government funding, his company could expand its supply of AVI-7537 from the couple dozen courses of treatment it has on hand to enough medicine to treat 100 Ebola patients.

    Story: How the U.S. Screwed Up in the Fight Against Ebola

    “We have been communicating to all of the government agencies, the World Health Organization,” Garabedian told CNBC. “We’ve had people at the table at every substantive discussion around how to manage this Ebola outbreak and have educated everybody who’s interested in learning about our technology and the drug we have available. Everybody knows we’re here. They know the drug we have, and we’re waiting for that call.”

    The call hasn’t come, even as the Ebola crisis has intensified in the wake of Duncan’s death and the subsequent infections of two nurses in Dallas. In recent weeks, Garabedian has refrained from making public comments. A reasonable surmise is that he doesn’t want to be seen as trying to cash in on public panic or embarrass government officials facing widespread criticism for their slow reaction to a disease that has killed thousands in Africa and spread fear elsewhere in the world. Garabedian declined to comment for this article.

    Last week, Sarepta issued a little-noticed press release announcing the anticipated publication of a study showing “no clinical or toxicologic safety concerns” in human test subjects administered AVI-7537. The submission of the study for publication in the November issue of the journal Antimicrobial Agents and Chemotherapy occurred well before Duncan’s diagnosis made Ebola a major topic of concern in the U.S., according to a Sarepta spokesman.

    Story: A Poopy Pill to Treat Hospital Infections

    Duncan was given the experimental drug brincidofovir, made by Chimerix (CMRX). It could be that he received the drug too late and that no medicine would have saved him, Thomas Geisbert, a virologist at the University of Texas Medical Branch at Galveston, told Bloomberg News on the day Duncan died. Another possibility raised by Geisbert is that the Chimerix drug is not as effective as two others that have been tried on Ebola patients: ZMapp, from Mapp Biopharmaceutical, and TKM-Ebola from Tekmira Pharmaceuticals (TKMR). To date, no experimental Ebola treatment tried on patients has been proven in a study to be effective in people.

    Sarepta received a $300 million federal contract in 2010 supervised by the Department of Defense as part of a research program targeting Ebola and another deadly viral disease. Over the next two years, the company tested its Ebola compound on 90 infected monkeys. Subjects in the control group that didn’t get the drug all died, typically within eight to 12 days. Of the 67 monkeys that received treatment, however, 60 percent to 80 percent survived, Garabedian told CNBC. In the company’s recent press release, Michael Wong, Sarepta’s senior medical director for infectious diseases, was quoted reiterating the primate survival rate of “up to 80 percent.”

    In 2012, the Defense Department, citing general budgetary constraints, eliminated funding for Sarepta’s Ebola program, effectively bringing the research to a halt. “Sarepta’s drug showed some promising results and deserves a look to see if it’s effective in humans,” Steve Brozak, president of WBB Securities and a biotech analyst, said in an interview. Brozak declined to speculate on why the company’s drug hasn’t received more attention during this year’s Ebola outbreak.

    Story: Texas Ebola Patient Dies. Are U.S. and European Hospitals Ready for More?

    One possible explanation is government disorganization: bureaucracies that haven’t communicated effectively. A darker theory is that officials who participated in a decision to stop funding Sarepta’s Ebola research are having difficulty revisiting a choice that in retrospect might have been precipitous or unwise.

  • William Blair Assigns Outperform On Sarepta Therapeutics Shares, $63 PT

    October 17, 2014 2:15 PM EDT by Editor Corey Williams in Analyst Insights, Healthcare

    In a research report issued today, William Blair analyst Tim Lugo assigned an Outperform rating on Sarepta Therapeutics Inc. (NASDAQ:SRPT) with a price target of $63.00.

    Lugo observed, ” After the close on Thursday, October 16, Sarepta Therapeutics announced the publication of data surrounding the safety and pharmacokinetic (PK) profiles of two experimental combinations of its next-generation RNA-based technology (PMOplus) applied to Ebola and Marburg viruses. AVI-6002, the candidate to treat Ebola, is a combination of AVI-7537 and AVI-7539 that is administered intravenously in a 1:1 combination dose. This combination was selected because early animal studies suggested that they were the best candidates for post-exposure prophylaxis in Ebola. With the recent reports in the mainstream media surrounding Ebola in the United States and abroad, this publication lends support to the company’s development of a potential candidate for the treatment of the Ebola virus in humans. The safety study was performed in 36 healthy subjects (N=5 per dose) with an average age of 31 years, and examined six escalating doses (0.005, 0.05, 0.5, 1.5, 3, and 4.5 mg/kg of each candidate in the combination) diluted in 150 ml of saline and delivered over 30 minutes against placebo (150 ml saline alone, N=6). Subjects were observed for 96 hours after the drug administration for pharmacokinetic data collection. Subjects returned for follow-up visits at 14, 21, and 28 days after dosing for assessment of adverse events (AE). “

  • Going back to sleep

  • Reply to


    by starfe11 Oct 17, 2014 10:43 AM
    zwerp2000 zwerp2000 Oct 17, 2014 10:46 AM Flag

    I'm sure the parents and their children with DMD appreciate what you are doing and saying on this board. Very helpful to their efforts to get eteplirsen approved!

  • Sarepta soars after publication of Ebola drug candidate data
    Shares of Sarepta Therapeutics (SRPT) are soaring this morning following the publication of Phase I clinical study results for its Ebola and Marburg virus drug candidates. Sarepta is a drug company engaged in the development of treatments for rare and infectious diseases, including those two as well as Duchenne muscular dystrophy, or DMD. WHAT'S NEW: On Thursday, Sarepta announced the publication of results from two studies involving its Ebola and Marburg treatment candidates, which demonstrated no clinical or toxicologic safety concerns. The two Phase I studies were randomized, double-blind, placebo-controlled trials designed to characterize the safety, tolerability and pharmacokine tics of single doses of IV formulations of AVI-6002 or AVI-6003 in healthy adult volunteers. Michael Wong, senior medical director at Sarepta, said in a statement that "We believe these promising early clinical safety results, coupled with the strong safety and efficacy data generated from animal studies for all four PMO compounds, reinforce the use of our PMOplus chemistry platform to pursue potential treatments for deadly infectious diseases such as Ebola and Marburg." The results of the study are set to be published in the November issue of the American Society for Microbiology’s journal, Antimicrobial Agents and Chemotherapy. WHAT'S NOTABLE: Earlier this year, shares of Sarepta plunged after results from a trial involving the company's eteplirsen drug for the treatment of patients with DMD fell short of expectations. A study published in medical journal PLOS One following the progression of Duchenne muscular dystrophy supports Sarepta's push for accelerated approval of e teplirsen, TheStreet's Adam Feuerstein recently reported. PRICE ACTION: In morning trading, Sarepta shares are up $1.58, or 7%, to $23.88. Year-to-date, Sarepta shares are up about 17%. OTHERS TO WATCH: Other drug companies that are working on experimenta

  • Sarepta announces publication of Ebola, Marburg Phase I clinical study results
    Sarepta Therapeutics announced the publication of results from two single ascending-dose studies that demonstrated no clinical or toxicologic safety concerns with the company’s drug candidates for the treatment of Ebola and Marburg virus, respectively. AVI-6002 for the treatment of Ebola is a combination therapy of two phosphorodiamidate morpholino oligomers, which target the viral matrix proteins VP24 and VP35, respectively. AVI-6003 for the treatment of Marburg is a combination therapy of two PMOs, which target the viral proteins VP24 and NP, respectively. These drug candidates use Sarepta’s advanced and proprietary PMOplus chemistry, which is also the basis of th e company’s clinical-stage influenza drug candidate, AVI-7100. Results from previous viral challenge studies of AVI-6002 and AVI-6003 in non-human primates demonstrated prevention of disease development and death following exposure to Ebola or Marburg virus. Subsequent animal studies demonstrated that for each combination therapy, only one oligomer contributed to efficacy, and therefore, the lead drug candidates for Ebola and Marburg have since become the single compounds AVI-7537 and AVI-7288.

  • I am not one that considers SRPT a major ebola player but maybe one of these days the government will come knocking on the door

  • Reply to

    Serious question for the longs

    by devilsyankeesfan Oct 15, 2014 7:32 AM
    zwerp2000 zwerp2000 Oct 15, 2014 9:27 AM Flag

    Correct. If CG can show continued dystrophin production, the fda would likely use it as a surrogate marker. This would then be applicable to their other exon skipping drugs and make it easier for approval going forward of their entire platform. Moreover, assuming strong results, this would confirm eteplirsen is head and shoulders better than prosensa's drug and i think by prosensa not doing the biopsy, having failed in their trial data and 80% of patients having AE from the treatment, prosensa would have a difficult time getting approval. And please, that PR about them starting the NDA submission is garbage. It's either submitted or it's not submitted.

  • zwerp2000 by zwerp2000 Oct 13, 2014 11:03 AM Flag

    Late Breaking Talks:

    PPMD-funded gene therapy study improves walking ability in muscular dystrophy

    Dr. Jerry Mendell of Nationwide Children’s Research Institute presented data from a ground-breaking PPMD-funded study demonstrating that a modified virus carrying the gene for follistatin can improve performance on the 6 minute walk test in study participants with Becker muscular dystrophy. This is the first time a gene therapy has demonstrated an improvement in function in a muscular dystrophy. Previously, animal studies demonstrated that a molecule called follistatin could be used to block myostatin in the muscles, leading to increased muscle size and strength, even in mice that lacked dystrophin. For the clinical study, Dr. Mendell and colleagues injected the modified virus into 12 places in the quadriceps muscle in 6 men with Becker muscular dystrophy, where it entered the muscle cells and allowed those cells to make and secrete follistatin. The investigators chose to start with Becker muscular dystrophy because those with BMD have significantly weak quadriceps muscles compared to the surrounding leg muscles and improving quad strength should lead to improved walking abilitly. The participants were all over 18 years of age and had to be able to complete the 6 minute timed walk. The first three participants received a low dose of gene therapy and the second three participants received a higher dose.

    From the group that received the low dose, at the end of the first year one participant was able to walk 58 meters longer, one could walk 125 meters longer and the third saw no change in walking ability. In the high dose group, similarly, one participant could walk 30 meters longer, one could walk 108 meters longer and the third saw no change. When magnetic resonance imaging was used to visualize the participants’ muscle, it was clear that the two participants who showed no change in walking ability also has the greatest amount of fibrosis in their muscles. Muscle biopsies showed a decrease in fibrosis compared to the start in the two participants who improved the most in their 6 minute timed walk tests. Importantly, there has been speculation that forcing the muscle cells to become larger and stronger without fixing the underlying lack of dystrophin may overtax the muscle repair system. Dr. Mendell showed that there was no evidence that the muscle satellite stem cells that are involved in muscle repair were reduced in number.

    Dr. Mendell is currently planning a similar trial in Duchenne muscular dystrophy, but will plan to inject the follistatin gene into gluteal muscles, thigh muscles and the tiabialis anterior muscles because all of these muscle groups are strongly affected in Duchenne. He speculates that even muscle groups that are not injected directly may show some benefit since follistatin is secreted and can circulate through the body. Animal studies showed evidence of these “remote effects” away from the site of injection.

    Further Discussion of Exon 51 Skipping Drugs

    A lot of questions have been raised about why the phase III study for drisapersen, a drug designed to skip exon 51, was negative after the phase I and phase II studies showed functional improvements. Dr. Francesco Muntoni of University College London summarized the existing data for Drisapersen, emphasizing that an analysis of the composition of all three main Drisapersen studies demonstrated that the boys in the phase III study had much slower walk times at the start of the study than the boys in the first two studies—in other words, they were on average further progressed in their disease. Dr. Muntoni suggested that a longer time period may be required to see a response to exon-skipping in more severely affected boys, and pointed to extension data from all three studies showing improvements in 6MTW at 96 weeks compared to the same at the official study end time at 46 weeks. Magnetic resonsance imaging data from 5 boys who were on drug the whole period also showed an improvement in the muscles compared to those of 3 boys who had not received drisapersen. Adverse events across all trials included “sub-clinical” protineuria (protein in the urine that is seen in laboratory tests but isn’t causing any symptoms), local injection site reactions and thrombocytopenia (low platelet levels in the blood). In total, over 300 boys have received the drug to date.

    Dr. Jerry Mendell of Nationwide Children’s Research Institute in Columbus also summarized the data for Sarepta’s exon-51 skipping drug eteplirsen. He emphasized that over 1800 doses of eteplirsen have been administered over the course of two human clinical trials with no adverse events. Sarepta’s phase IIb study was designed so that 8 boys of the original 12 would receive either 30mg/kg of eteplirsen or 50 mg/kg of eteplirsen while four boys would receive placebo for the first six months and then two would be moved to the low dose and two moved to the high dose of the drug. In practice, two boys who were in the group that received eteplirsen from the beginning stopped walking before 24 weeks. The trial was changed to a “modified intent to treat” study that allowed the investigators to continue without the data from the two boys who could no longer perform the 6 minute timed walk. For the boys who continued to perform this measurement, the four who had received eteplirsen from the start of the trial declined by only 32M at 144 weeks and the boys who started on placebo and then crossed over to receive drug (the “placebo-delayed” group) declined by only 39M total. The natural history of the disease would predict that without treatment, over the same period, the boys should have declined by about 140M. Measurements of respiratory function remained stable in all 12 boys over the same period.

    Posters Selected for Oral Presentations:

    Lack of specific form of dystrophin in the brain may underlie cognitive deficits

    Many different versions of the dystrophin protein are expressed in tissues throughout the body, in addition to muscle, but little is known about the function of these different dystrophin versions in other tissues. Dr. Nathalie Doorenweerd of Leiden University Medical Center in the Netherlands demonstrated that a version of dystrophin called the “dp140 isoform,” which lacks the beginning of the full length dystrophin protein found in muscle, is strongly correlated with decreased brain volume and decreased blood flow to the brain. Boys with Duchenne caused by mutations between exons 1 and 40 should still be able to make the dp140 isform of dystrophin while boys with mutations between the intron 44, or exon 55 and further should lack the dp140 isoform. The investigators could detect differences in brain size and functional imaging between the two groups.

    Strong influence of incentives on 6 minute timed walk

    Lindsay Alfano of Nationwide Children’s Research Institute wondered how much motivation might effect performance of the 6 minute timed walk, the primary outcome measurement in most trials for Duchenne muscular dystrophy. She emphasized that it’s important to better understand sources of variabililty in this common endpoint. The investigators started with a group of 27 ambulatory boys with Duchenne with a mean age of 9, and asked them to the 6 minute timed walk two times with a 30 minute break in between. For the second timed walk, one group of boys were told to walk as fast as they can and then they would receive a prize. In contrast, the second group was told that they would receive $50 if they could beat their first time (both groups actually received $50 at the end). Neither the boys nor the evaluators were told which group was which.

    The study showed that the boys who were promised the $50 incentive walked an average of 41 meters further during the second timed walk, a 10% difference in scores. The boys without the incentive walked 2% faster on the second test. The difference between the two groups was statistically significant. The investigators then wondered if the boys who were more severely affected were less able to improve their times, but found instead that they were able to improve on the second test almost the same amount as the boys who were less affected. They concluded that incentives can have very significant affects on the 6 minute timed walk and that investigators should be aware of the need to sufficiently and equally incentivize all trial participants. The investigators will next try to determine if a shorter timed walking test would be less influenced by incentives.

    Future Duchenne Therapies: Gene Editing

    Although a number of high tech therapies in development for Duchenne are designed to provide an extra copy of the healthy dystrophin gene (gene therapy) or tweak the way the cells use the mutated gene to make a protein (exon-skipping), none of these change the DNA code itself. For many years scientists have been quietly experimenting with ways to do just that and now, with a new technique called CRISPR (pronounced “crisper”) Ronnie Cohn of Sick Kid’s in Toronto can change a sequence of DNA in the cell’s nucleus that controls the production of utrophin, dystrophin’s molecular cousin, so that much more of it is produced. The goal of this work is to produce enough utrophin to compensate for the loss of dystrophin. The technique can also be used to directly correct very small mutations in the dystrophin gene itself, but each individual mutation would require its own specific approach so Cohn’s lab is focusing on upregulating utrophin for now. Cohn’s laboratory is just starting testing the utrophin upregulation in mice that lack dystrophin.

    Sharing our PPMD/DuchenneConnect community perspectives with WMS

    PPMD’s Holly Peay presented data on two studies, one of clinical trial decision making in parents whose children are/have recently been involved in clinical trials, and one of parents whose children have never been in a clinical trial. We were able to share the perspectives and decision-making influences of a large number of parents with clinicians and researchers in the audience. First Ms. Peay presented during the poster session on Thursday, and again on the final day of the meeting to the entire meeting audience. It was a great way to share the thoughts of the community and move toward better clinical trial education and support for making complicated clinical trial decisions.

    Phase III idebenone for lung function meets primary and secondary endpoints

    Dr. Thomas Meier from Santhera gave an update on the phase 3 DELOS idebenone trial. The trial aimed to assess the efficacy of idebenone, compared to placebo, on delaying or improving the loss of lung function. The trial has been conducted in the United States and Europe with 31 patients on idebenone and 33 on placebo. Patients were 10-18 years old, not on steroids, and most of them are non-ambulatory. After treatment for 12 months, the data show that there were significant reductions in the loss of peak expiatory flow (the primary study endpoint) and of forced vital capacity. Other clinically important findings were that fewer patients on idebenone progressed to the point where their coughing was no longer effective and that they had fewer respiratory tract infections. There were few side effects and most patients tolerated idebenone well.

  • Perhaps we see more positive notes today?

  • MIP +14.7% increase from baseline, MEP +12.8% versus +2.4% and -4.5% predicted for pulmonary function; proof that Eteplirsen works

    $SRPT Forced vital capacity, a measure of pulmonary function, +11% versus a predicted -10.6% reaching statistical significance.

    $SRPT Exons 45 and 53, representing an additional 16% of the MD population exhibit zero toxicity in animal studies; same as exon 51 studies

  • Reply to

    A few early tweets on the data fwiw

    by zwerp2000 Oct 11, 2014 9:07 AM
    zwerp2000 zwerp2000 Oct 11, 2014 10:05 AM Flag

    doubtful. i believe there was an embargo on this information being released prior to the conference date/time so i do not believe he planned it. that is normal. i do wonder if the company will issue a pr at some point highlighting the details but i wouldn't expect it.

  • Reply to

    A few early tweets on the data fwiw

    by zwerp2000 Oct 11, 2014 9:07 AM
    zwerp2000 zwerp2000 Oct 11, 2014 9:46 AM Flag

    and to be honest, i believe prosensa will have a difficult time getting their drug approved in light of srpt's results.

  • Reply to

    A few early tweets on the data fwiw

    by zwerp2000 Oct 11, 2014 9:07 AM
    zwerp2000 zwerp2000 Oct 11, 2014 9:44 AM Flag

    it does matter because srpt has the better, safer drug. prosensa failed in their trials to show a real benefit and gsk completely walked away. prosensa had 80% of their patients have an AE, none for eteplirsen. eteplirsen clearly had better efficacy as well. if you were a doctor or even a patient, and you had a choice between the two, which one would you choose?

  • Reply to

    A few early tweets on the data fwiw

    by zwerp2000 Oct 11, 2014 9:07 AM
    zwerp2000 zwerp2000 Oct 11, 2014 9:37 AM Flag

    the population size for DMD is very small. for most cancers, you can easily recruit hundreds of patients. you can't with dmd. look at companies like biomarin that have received approval for rare diseases where the trial was very small. the fda needs to be flexible when dealing with a disease that has a 100% mortality rate on children, where there are no effective treatments.

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