The USPO recently granted two more patents to Sarepta:
1.Oligonucleotide analogues targeting human LMNA. This patent is intended to cure Hutchinson-Gilford progeria syndrome (HGPS), a rare genetic disease that causes young children to prematurely and rapidly age; and
2. Antisense modulation of nuclear hormone receptors. This patent is intended to treat inflammatory diseases without the usual, and sometimes serious side effects like diabetes, osteoporosis and glaucoma caused by glucocorticoids, the current standard of care.
Sr. Director of Trade, Distribution, Contracting and Managed Markets - The individual will be asked to build and execute the global access and reimbursement strategy for our lead candidate, Eteplirsen, along with our DMD portfolio. And:
Medical Science Liaison - Northeast
Medical Science Liaison - Southeast
Medical Science Liaison - Northwest
Medical Science Liaison - Southwest
The newly created Medical Science Liaison (MSL) position will be one of the first field based members of the Medical Affairs team at Sarepta and will play a pivotal role on the development and commercialization of Eteplirsen as well as support for the Sarepta pipeline.
New Positions posted this week:
Director, Reimbursement and Managed Markets Strategy - This individual will be responsible for building and executing the US reimbursement and managed markets strategy for our lead candidate, Eteplirsen, along with our DMD portfolio.
Director, Specialty Distribution & Patient Access - This individual will be responsible for designing, implementing and managing the day-to-day operations of Sarepta’s Specialty Distribution and Patient Access programs for our lead product, Eteplirsen. Additionally, this person will be responsible for driving new initiatives post-launch to ensure these programs continue to provide best-in-class support and minimizing reimbursement as a barrier in order to enhance overall patient access to therapy.
Among other items in the bill, The Council for 21st Century Cures is established to accelerate the discovery, development, and delivery of innovative cures, treatments, and preventive measures. The priority review voucher program for rare pediatric diseases is revised and extended.
And of important significance to the approval of eteplirsen, this bill amends the Federal Food, Drug, and Cosmetic Act to revise the drug approval process, including by: (1) allowing patient experience data to be considered in the risk-benefit assessment of a new drug. HR6. On to the Senate and the President!
Posted Today by the USPO. Wilton, et al are the inventors, though Sarepta and Murdoch University (in Washington State) are the joint applicants.
Pompe Disease is caused by a missing enzyme that would normally break down glycogen (a sugar). When glycogen is not broken down, it accumulates in the heart, liver, nervous system and muscles, eventually causing respiratory failure and death. Sarepta's drug would splice RNA (exon 2) that contains the coding needed to produce the enzyme that will break down the glycogen. Sarepta's work in the Pompe disease area has been highlighted in its recent presentations, though it is not specifically listed as a development program on Sarepta's Pipeline webpage. Expect it soon.
A kickback is used by a drug company to reward a doctor for prescribing a particular pharmaceutical to a patient. The more informed a patient is about drug alternatives, the less successful a doctor is going to be in prescribing the drug and getting a kickback. The DMD community is the most informed group of parents (and kids) about a complex illness and its alternative treatments that I have witnessed. They are the least likely to be fooled by a doctor intentionally steering them towards the wrong drug to make an extra buck.
Putative treatment for DMD took a few steps back today when the National Health Science of England (England's socialized medical care) once again postponed funding treatment for boys. Boys in other parts of Europe are getting treatment. I hope eteplirsen doesn't slam into that same brick wall in America with its private health insurance companies (or Medicaid for that matter). Action Duchenne leaders said of the government decision makers: "it's cowardice" and "a decision that is symptomatic of poor and unfocused leadership of the health services". No mincing words across the lake by duchenne community leaders.
I like your thinking, thigrlsrk - boys too healthy (scored too high on the 6MWT) to qualify for eteplirsen at the start of trial, and now can't walk at all. Thirty seconds of testimony from a couple of boys like that, along with testimony from a couple of boys who have thrived on eteplirsen, will demonstrate, in real life, what all the numbers and charts and words in the NDA really mean.
is scheduled to complete in April 2016. Enrolling 67-72 children, ages 5 and up for 48 weeks at 13 sites in the US and Canada. All but 5 sites are actively enrolling. Efficacy is not a primary endpoint - only safety (incidents of adverse events), especially heart and lung, but also including lab tests studying hematology, biochemistry, and urinalysis parameters. Secondary outcome measurements for efficacy. Offering intermittent dosing of 6mg/kg for those kids that can't tolerate continuous dosing. No minimum duration, as the company probably expects children to drop out because of adverse events. Clinical trial dot gov website last updated March 5, 2015.
I recall that CG said he was not applying for a breakthrough designation for eteplirsen. Not sure why he chose not to apply. The FDA has already "green lighted" eteplirsen by giving Sarepta a go ahead and submit signal. Once Sarepta completes its rolling submission (by mid year), then the FDA will consider whether the submission will be filed as a new drug application. Probably about the same time, assuming a filing is granted, the FDA will likely schedule an ADCOM review of the data and hear from experts, parents, company officials and, hopefully, kids taking eteplirsen. My limited understanding of the process is that it will take about 6 months from time of "filing" until approval (or denial) under priority review. Speaking of priority review, Sarepta should receive a priority review voucher for eteplirsen if the drug is approved ahead of drisapersin (assuming Sarepta included a voucher request in its NDA submission).
Correction: Murdoch, WA (where Murdoch University is located) is not in Washington state. WA is Western Australia (which makes more sense if Wilton is an inventor).
My guess is that Sarepta will make a huge effort to garner proxies from those millions of shares that were not voted at the shareholder's meeting.
From the New England Journal of Medicine: After only 12 weeks of PRO051, "new dystrophin
expression was observed between approximately 60% and 100% of muscle fibers in
10 of the 12 patients, as measured on post-treatment biopsy, which increased in a
dose-dependent manner to up to 15.6% of the expression in healthy muscle. After the
12-week extension phase, there was a mean (±SD) improvement of 35.2±28.7 m (from the baseline of 384±121 m) on the 6-minute walk test."
This new trial, treating both exons 45 and 53, is labeled as a Phase 3 trial. There will be 99 enrollees: 33 needing exon 45 skipped, 33 needing exon 53 skipped, and then 33 amenable to exon 45 or 53 skipping but receiving placebos (I wonder why not against natural history like our other trials). Duration, 48 weeks. Trial site locations not yet listed.
Bionerd, any safety/adverse events reports on GS/PPMO? Is this a drug that will require lifetime treatment like eteplirsen? Thanks.