Not surprising to hear of playground accidents in both trials. Children are usually active in playgrounds, and when they have weak legs and loss of coordination due to muscle disease the odds of playground injuries multiply.
I don't think these results have been posted here before. These are from the clinical trials gov. website for PR044. It's all charted out.
18 participants - 18 subcutaneous injection (.5mg - 10mg) once a week for 5 weeks; apparently 9 of the boys also received intravenous injection (1.5mg - 8mg) once a week during the same 5 weeks. Measurements taken within 13 weeks after treatment.
Here are some of the results:
Biopsies for dystrophin production were measured by Immunofluorescence and western blot.
In the subcutaneous group, biopsies in 14 of 18 boys were analyzed. Of those 14, 9 showed an increase in dystrophin production.
Dystrophin production did not seem to be dose-related.
In the intravenous group, biopsies in 8 of 9 boys were analyzed. Of those, 6 showed an increase in dystrophin production.
Serious Adverse events: There were 3 serious adverse events recorded, affecting 2 patients. These ranged from blood and lymphatic system disorders to renal and urinary disorders. The serious adverse events also included a concussion when a child fell in a playground.
Other adverse events: There were other adverse events recorded for nearly every child. These ranged from injection site erethyma and hematoma, to abdominal pain and flu like symptoms.
says the European Pharmaceutical Review in an analyst article published on line on April 15th.
"The global treatment market for Duchenne Muscular Dystrophy will expand in value at a staggering Compound Annual Growth Rate (CAGR) of 160.5%, from approximately $8.2 million in 2014 to $990 million by 2019, says GlobalData.
"The company’s latest report, OpportunityAnalyzer: Duchenne Muscular Dystrophy – Opportunity and Market Analysis to 2019, states that this growth will occur across six major markets: the US, France, Germany, Italy, Spain and the UK. Combined, the five European countries will contribute 55.2% to the total market value by 2019, while the US will account for 44.8%.
"According to Nikhilesh Sanyal, Ph.D., GlobalData’s Analyst covering Immunology, the primary driver behind this impressive expansion will be the market entry of PTC Therapeutics’ Translarna, Sarepta Therapeutics’ eteplirsen, and BioMarin/Prosensa’s drisapersen.
"Sanyal said: “These mutation-specific drugs are expected to have a high price point due to their novelty, efficacy and orphan drug status, and will contribute 85.6% to the Duchenne Muscular Dystrophy treatment arena by 2019. While Translarna is expected to be the top-selling Duchenne Muscular Dystrophy drug by 2019, exon-51-skipping therapies drisapersen and eteplirsen will also be a hotspot for growth, thanks to their rapid clinical development and significant demand. . . . "
Here's a summary of Sarepta clinical trial updates relayed to the DMD community and posted on the PPMD website late this morning: "
We do not anticipate changes to any of our ongoing or planned clinical programs.
Eteplirsen Confirmatory Study (PROMOVI/Study 4658-301): Phase III open-label safety & efficacy study to treat ambulatory boys with DMD
Update: Enrolling and dosing at various sites. We plan to initiate an extension phase of this study at its conclusion and are designing a protocol that avoids treatment interruptions when patients roll over into the extension phase.
Eteplirsen Safety Study (Study 4658-204): Phase II open-label safety study to treat advanced stage DMD
Update: Enrolling and dosing at various sites. Progressing on schedule with completion of enrollment anticipated by mid-year.
Eteplirsen Safety Study (Study 4658-203): Phase II open-label safety study to treat early stage DMD
Update: We expect to announce enrollment, to begin in the second quarter of 2015.
Exon 53 Skipping (SRP-4053) Study (Study 4053-101)
Update: First phase of the study has been completed at clinical sites in Europe
Exon 45 Skipping (SRP-4045)and Exon 53 Skipping (SRP-4053) Study (Study 4045-301)
Update: We expect to initiate the trial in the US by mid-year, with enrollment beginning in Europe late 2015 or early 2016."
jrrt:: Here are two more scenarios: 1). The biopsy results are not complete and that fact has not been leaked; 2) the biopsy results are complete and the results have not been leaked.
Confirmed dystrophin expression in 10-boy trial. Will start new phase, with intent to market in 2018. No serious adverse events were observed throughout the study, and no subjects discontinued administration. This is great news that should help validate Sarepta's same drug in clinical trials in Europe for treatment of DMD in boys needing exon 53 skipping. As has been pointed out before on this board, the Japanese have the earliest patents for exon skipping with antisense drugs, though not as broad as the ones obtained by the University of Western Australia and University of Leiden.
Muntoni hitting hard. From various twitter posts, quoting Muntoni: "Clearly drugs that don't produce dystrophin are not good". Dystrophin can be measured accurately. Five labs had very tight correlation (6 biopsies tested by 5 labs using standardised IHB and WB protocols) . Together with clinical endpoints, dystrophin can be a package for drug approval.
Take a look in the Reuters article about ZMapp trials. Sarepta's drug, and others, are mentioned as included towards the end of the article
That's simply a wrong statement of what the CEO said. The new ambulatory trial is dosing now, and the company expects to have all the data requested from that study by the FDA before it submits its NDA mid-year. What you heard was that the study of the younger set of boys will start in the third quarter because the FDA has requested MRI data on those children. You have mixed apples and oranges. The ambulatory trial is well under way and on schedule.
SRPT owns the patent for eteplirsen in the US. Although a patent interference has been filed against it, the challenger has the burden of proof of showing any interference and Saretpa's patent remains valid until all appeals are exhausted. That will take years. Sarepta realizes eteplirsen's patent validity and is moving forward to market.