TSR — The Senyak Report
Gilead drug pricing ticks off Congress, patients Really, how much should we care about Gilead’s new Hepatitis C “cocktail” pill? Most of us met Gilead when it scooped up YMI Bioscience in 2012 and brought its JAK inhibitor, CYT 387, into Phase III clinical trial. We haven’t heard much, until now when the company hit the headlines with its new pricey Hep C drug. Gilead’s wonder drug, Solvadi, is priced at $84,000 for a 12 week treatment. (Difference between that and our own $100,000+ Jakafi, is Gilead made its case for 100% cure rates for most genotypes taking its combination treatment over the course of 12 weeks)
… The reason we might care is interferon. MPN patients have had a plentiful, if not cheap, supply of recombinant interferons — Pegasys, Intron-a, Pegintron, etc. — even though, in the US, interferon is not FDA approved for MPN treatment. We can get it “off label” because it is approved for treatment of Hepatitis C. Until the advent of this new generation of ingestible drugs, the common treatment for hepatitis included injectable interferon. 3 million US sufferers of Hepatitis C is a major market…maybe ten times the size of the total US MPN population … While there’s some fear of reduced supplies of interferon, manufacturers have not indicated any inclination to suspend production so far…One ominous sign: Express Scripts, the largest Pharmacy Benefits Management company in the US (Revenues around $100 billion/yr) announced it will exclude coverage for Merck’s hepatitis C treatment Pegintron.
There are positive results to report on the Zebra Coalition petition to the FDA to bring a Patient Advocate into every MPN clinical. The FDA’s Center for Drug Evaluation and Research, has responded to our request for a meeting to discuss means to bring a Patient Advocate into every MPN clinical trial. A teleconference has been scheduled for the first week of April with the Director of the Office of Hematology and Oncology Products, the Deputy Director for Clinical Science — “and perhaps others” — to set up the agenda for a face to face work-session. Jeremy Smith has joined the MPNforum team. If you have items you’d like to see on that agenda, or would like to participate directly in negotiations in Washington, D.C. contact ourMPNforum@gmail.com…
And now we face the last few days to participate in the MPN-Related Fatigue Project with well over 1300 completed survey responses. You can still complete the survey and help shape the coming clinical trial. (Click here to see Dr. Mesa’s video.) Or just head right on directly to the Survey. It takes under 10 minutes and can bring a lifetime benefit to the whole MPN community, c’mon, you can do it. Don’t let history be made without you
Report on the latest blips in the Geron Imetelstat bumpy path to market– from FDA holds and Class Action lawsuits to continued corporate efforts to get on track…In this shadowy Kafkaesque drug development world, an on-going Imetelstat clinical trial is placed on hold mid-March because the FDA is concerned about possibly irreversible liver damage…Or at least that’s the word from Geron which announced the clinical hold based on conversations with the FDA but it had not yet received written confirmation. Patients, on the other hand, will never get written confirmation from the FDA which communicates only with the Sponsor of a clinical trial on safety issues
…. The Sponsor communicates with its shareholders through press releases…and if we listen closely enough we can get the idea. Maybe…. Reportedly the Ayalew Tefferi Investigator Sponsored Trial at the Mayo Clinic (Rochester, Minnesota) is continuing for patients who can proved clinical benefit from the treatment despite further reports of toxicity and dropouts, http://bit.ly/1cmlFEX
Can Geron successfully execute a U-turn and get back on the promising highway to clinical trial after yet another serious setback? The flurry of class action lawsuits might be an expected irritant considering the steep drop in share price and the lack of any advance warning of toxicity in reporting outcomes at ASH ( a presentation that led to an equally steep increase in share price)… The company’s quick public offering following the ASH euphoria does give it deep enough pockets to continue on once — or if — the clinical hold is lifted
… But the fundamental question that remains unanswered is the ability of Imetelstat to provide anything like the reported Complete Response results in a durable, relatively safe dosing regimen. That’s an answer we can’t get looking over the shareholders’ shoulders reading the latest press release from Geron reporting what it may have heard from the FDA
… As things now stand we have no idea how serious these toxicity issues are, how likely it is that Geron can demonstrate reversibility of liver damage, or even whether or not the planned myelofibrosis clinical trials can — or should — get back on track…
Notice of community gatherings of MPN patients and doctors. MPD Voice is sponsoring an MPN Patient Forum Saturday, June 28 in Manchester, free and open to all, the poster and details are here…And as long as we’re visiting MPD Voice we might as well plug their bike ride fundraiser, the Moon Rider. Play the Moon-oply game in a 50 mile figure eight tour around London’s most iconic streets, refreshments and breaks along the way, open to all levels. Information here or e-mail Marilyn at email@example.com.
PatientPower is rolling out a pair of free seminars, MPN educational events produced in partnership with MD Anderson… There’s a Saturday April 19th Town Hall style meeting at City of Hope (Los Angeles) featuring Dr. David Snyder, associate Chair of Hematology and Cell Transplantation at City of Hope. Descriptive poster and registration for the free meeting is available here.
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