【分享】Celgene's mongersen continues to impress in Crohn's
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Physician Views Poll Results: Celgene's mongersen continues to impress in Crohn's – is it too good to be true?
(Ref: Physician Views Desk) October 30th, 2014 By: Michael Flanagan
So convinced was Celgene that the effects of mongersen, also known as GED-0301, are real that the company ponied up over $700 million upfront for rights to the Crohn’s disease product based on an early look at an ongoing Phase II trial.
Last week, the medical community got its first look at final results from the study, which did indeed appear impressive. But given all the question marks – both real and hypothetical – still associated with the compound, how convinced are doctors that what they are seeing is a revolutionary new medicine?
Soon after the data were presented on October 21, FirstWord polled a total of 75 gastroenterologists in the US and EU5 with a series of five questions designed to better understand their perception of mongersen in light of the newly available evidence of its clinical benefit.
Many on the Street believe that mongersen has the potential to become the new standard first-line therapy for moderate-to-severe Crohn's disease, putting it in front of biologic drugs like AbbVie's Humira and Johnson & Johnson's Remicade, which doctors participating in the survey said they prescribe to an average of about 55 percent of patients (with the median coming in at 60 percent).
The key finding used to support this thinking was mongersen's four-week remission rate of 72 percent, which looks impressive versus remission rates of between 15 percent and 48 percent produced in studies of marketed biologics.
Little wonder then why some analysts, including Bank of America Merrill Lynch's Ying Huang, are now predicting mongersen could generate peak sales north of $2 billion given the size of the patient pool Celgene could end up targeting.
The new data clearly had some impact on gastroenterologists, more than 90 percent of whom said their perception of mongersen had improved either a little (55 percent) or a lot (36 percent) based on the recent presentation.
Sceptics have highlighted a handful of issues, including potential flaws in the Phase II trial, as well as theoretical risks associated with mongersen, though a majority of physicians (53 percent) said the biggest question mark in their minds remains the absence of endoscopy and/or full biomarker data from the recently completed study, which instead used more subjective endpoints like Crohn’s Disease Activity Index (CDAI) score.
Among other uncertainties, 23 percent of respondents cited the high placebo response (17 percent after four weeks), while another 19 percent have doubts about how mongersen given for 14 days managed to offer such a durable effect out to 12 weeks when the cells of the gut are thought to turnover every few days.
Indeed, physicians in attendance at the United European Gastroenterology Week meeting asked the clinical investigator who presented the data this very question, to which they offered the possibility that targeting Smad7 "may have effects on cells beyond the gut epithelium and its action may have an effect on inducing immune tolerance, different from the typical anti-inflammatory," according to Jefferies analyst Thomas Wei.
It is perhaps notable that only 5 percent of doctors highlighted mongersen's theoretical risk of fibrosis (by indirectly raising TGF-beta levels) as being a primary concern, which could be seen as good news given the agent's presumed safety advantage over biologics, many of which carry black box warnings about infection risk.
As for gastroenterologists' confidence that Celgene will be able to reproduce the Phase II magic in a Phase III setting, a majority of respondents (71 percent) said they are "moderately confident" that it will succeed, while another 23 percent and 5 percent said they are "marginally" and "very" confident, respectively. Interestingly, not a single doctor said they are "not confident" mongersen will end up succeeding.
Despite their bullishness, physicians clearly don’t plan to rapidly switch all patients over to mongersen if and when it is approved. Rather, when asked how many patients they would expect to prescribe the product to within 12 months of launch, the average response was just shy of 37 percent, meaning Celgene still has plenty of work left ahead.
DOWNLOAD POLL RESULTS
As always, FirstWord would very much like to receive your [email=info@firstwordpharma.com?subject= Physician Views Feedback&tsid=17]feedback[/email] and suggestions.
Note: FirstWord Polls are powered by MedePolls, a fast-turnaround service to conduct instant polls of up to five questions with guaranteed samples that include physicians from dozens of specialities and over 100 markets (for further information click here). To conduct this poll with a different audience, or an entirely different poll, contact us at [email=info@firstwordpharma.com?subject= I want to run a FirstWord Poll with physicians&tsid=17]info@firstwordpharma.com[/email].
(Ref: Physician Views Desk) October 30th, 2014 By: Michael Flanagan
So convinced was Celgene that the effects of mongersen, also known as GED-0301, are real that the company ponied up over $700 million upfront for rights to the Crohn’s disease product based on an early look at an ongoing Phase II trial.
Last week, the medical community got its first look at final results from the study, which did indeed appear impressive. But given all the question marks – both real and hypothetical – still associated with the compound, how convinced are doctors that what they are seeing is a revolutionary new medicine?
Soon after the data were presented on October 21, FirstWord polled a total of 75 gastroenterologists in the US and EU5 with a series of five questions designed to better understand their perception of mongersen in light of the newly available evidence of its clinical benefit.
Many on the Street believe that mongersen has the potential to become the new standard first-line therapy for moderate-to-severe Crohn's disease, putting it in front of biologic drugs like AbbVie's Humira and Johnson & Johnson's Remicade, which doctors participating in the survey said they prescribe to an average of about 55 percent of patients (with the median coming in at 60 percent).
The key finding used to support this thinking was mongersen's four-week remission rate of 72 percent, which looks impressive versus remission rates of between 15 percent and 48 percent produced in studies of marketed biologics.
Little wonder then why some analysts, including Bank of America Merrill Lynch's Ying Huang, are now predicting mongersen could generate peak sales north of $2 billion given the size of the patient pool Celgene could end up targeting.
The new data clearly had some impact on gastroenterologists, more than 90 percent of whom said their perception of mongersen had improved either a little (55 percent) or a lot (36 percent) based on the recent presentation.
Sceptics have highlighted a handful of issues, including potential flaws in the Phase II trial, as well as theoretical risks associated with mongersen, though a majority of physicians (53 percent) said the biggest question mark in their minds remains the absence of endoscopy and/or full biomarker data from the recently completed study, which instead used more subjective endpoints like Crohn’s Disease Activity Index (CDAI) score.
Among other uncertainties, 23 percent of respondents cited the high placebo response (17 percent after four weeks), while another 19 percent have doubts about how mongersen given for 14 days managed to offer such a durable effect out to 12 weeks when the cells of the gut are thought to turnover every few days.
Indeed, physicians in attendance at the United European Gastroenterology Week meeting asked the clinical investigator who presented the data this very question, to which they offered the possibility that targeting Smad7 "may have effects on cells beyond the gut epithelium and its action may have an effect on inducing immune tolerance, different from the typical anti-inflammatory," according to Jefferies analyst Thomas Wei.
It is perhaps notable that only 5 percent of doctors highlighted mongersen's theoretical risk of fibrosis (by indirectly raising TGF-beta levels) as being a primary concern, which could be seen as good news given the agent's presumed safety advantage over biologics, many of which carry black box warnings about infection risk.
As for gastroenterologists' confidence that Celgene will be able to reproduce the Phase II magic in a Phase III setting, a majority of respondents (71 percent) said they are "moderately confident" that it will succeed, while another 23 percent and 5 percent said they are "marginally" and "very" confident, respectively. Interestingly, not a single doctor said they are "not confident" mongersen will end up succeeding.
Despite their bullishness, physicians clearly don’t plan to rapidly switch all patients over to mongersen if and when it is approved. Rather, when asked how many patients they would expect to prescribe the product to within 12 months of launch, the average response was just shy of 37 percent, meaning Celgene still has plenty of work left ahead.
DOWNLOAD POLL RESULTS
As always, FirstWord would very much like to receive your [email=info@firstwordpharma.com?subject= Physician Views Feedback&tsid=17]feedback[/email] and suggestions.
Note: FirstWord Polls are powered by MedePolls, a fast-turnaround service to conduct instant polls of up to five questions with guaranteed samples that include physicians from dozens of specialities and over 100 markets (for further information click here). To conduct this poll with a different audience, or an entirely different poll, contact us at [email=info@firstwordpharma.com?subject= I want to run a FirstWord Poll with physicians&tsid=17]info@firstwordpharma.com[/email].
