Cologne's Crohn's Disease Candidate Successful

From Seeking Alpha News Feed. It sounds very successful to me, for a disease notoriously hard to treat.


Celgene’s Crohn’s disease candidate successful in Phase 2 study • 8:27 AM
Douglas W. House, SA News Ed…

In a study published in the New England Journal of Medicine, a Phase 2 study evaluating Celgene’s (NASDAQ:CELG) investigational oral antisense therapy, GED-0301, in patients with Crohn’s disease demonstrated statistically significant efficacy compared to placebo at certain doses.

The 166-subject trial enrolled patients with moderate-to-severe Crohn’s disease, defined as Crohn’s Disease Activity Index ((CDAI)) scores of 220 to 400 (scores <150 indicate remission, >450 severe disease). Patients were randomly assigned to receive treatment for two weeks with one of three daily doses of GED-0301 (10 mg, 40 mg, 160 mg tablets) or placebo and then evaluated for responses at Days 15, 28 and 84. The primary efficacy endpoint was the percentage of patients achieving clinical remission (CDAI score <150) at Day 15, maintained at Day 28.

65% of patients receiving GED-0301 160 mg and 55% of those receiving 40 mg met the primary endpoint compared to 12% for the 10 mg cohort and 10% for placebo (p < 0.001).

The proportion of patients in the 160 mg cohort achieving clinical remission (CDAI score < 150) on Day 15, Day 28 and Day 84 was 67%, 72% and 67%, respectively, versus 21%, 14% and 21%, respectively, for placebo (p < 0.0001). The proportions for the 40 mg and 10 mg cohorts were 58%, 70% and 63% and 15%, 29% and 29%, respectively. The results for the 10 mg group did not reach statistical significance compared to placebo.

On Day 28, 37%, 58% and 72% of patients treated with 10 mg, 40 mg or 160 mg once daily achieved a clinical response (a 100-point reduction in CDAI score, a secondary endpoint) compared to 17% for placebo (p = 0.04, p < 0.001, p < 0.001, respectively).

Celgene intends to advance GED-0301, which it licensed from Dublin, Ireland-based Nogra Pharma about a year ago, to Phase 3 development.


And this is from the Press Release. I particularly like that side effects were the same as placebo! Also that it just required three days of oral treatment!

65 percent of patients treated with GED-0301 160 mg once daily for two weeks achieved clinical remission at both day 15 and day 28, versus 10 percent of patients on placebo

72 and 67 percent of patients treated with 160 mg once daily were in clinical remission at day 28 and at day 84, respectively

Overall rates of adverse events and serious adverse events were similar across treatment groups, including placebo

SUMMIT, N.J.–(BUSINESS WIRE)-- Celgene Corporation (CELG) today announced that results from a double-blind, placebo-controlled, multicenter phase II trial of three doses of GED-0301 (mongersen) in patients with active Crohn’s disease were published in the March 19 issue of The New England Journal of Medicine.

GED-0301 offers a unique approach to treating Crohns, using antisense technology to target a key intracellular signaling protein thought to be involved in intestinal inflammation and the pathogenesis of the disease, said Professor Giovanni Monteleone, University of Rome Tor Vergata. This orally administered therapy is designed to act locally with its novel mechanism of action. The results from the phase II trial suggest that GED-0301 should be studied further in phase III trials for Crohns disease.


Sorry about the title. That’s the autocorrection of what they think is a spelling error. Should be Celgene’s, not Cologne’s.

Thanks for sharing this information Saul. This is wonderful news. Not just because I’m an investor in CELG (thanks to this board), but also and much more importantly because I have a very dear friend with Crohn’s disease.

Not only is Crohn’s a terrible, hard to treat disease, it is also difficult to diagnose. My friend went from one GI specialist to another for nearly a year with undiagnosed or incorrectly diagnosed symptoms before she finally found a doctor who correctly determined what her affliction was.

I had only heard of Crohn’s before my friend told me she had it. My friend had a relatively rare complication called fistula. For those who don’t know, this is a disease wherein the body re-routes your plumbing creating abnormal openings between two organs or systems. This can result with different internal body pathways becoming connected in very inappropriate ways.

She had been given a CT scan which revealed nothing unusual. Once she switched doctors (again) the new doctor immediately diagnosed her as having a fistula and had an MRI performed to confirm. The MRI revealed one fully formed fistula and another beginning to form. Turns out for this particular malady, a CT scan fails to identify it about 60% of the time. Apparently her prior doctor was unaware of this reliability issue. My friend had been in considerable pain, and due to the fact that no doctor had been able to determine anything abnormal she was seriously beginning to question her sanity.

The new doctor prescribed antibiotics because her rectum and vagina had become connected (icky). She is also taking Humira specifically as treatment for Crohn’s. My friend objects to the idea of taking a medication for life. But at present it seems to be helping so she said she’s willing to give it a year after which she will try to find a way to get off the drug. I’ll send her a copy of the medical information in this thread.

For that matter, I’ll suggest she look at CELG as an investment as well. Even though she was poor as a churchmouse at the time, my friend started investing in her 20s due to my urging and advice (I introduced her to TMF in the mid-90s).

She’s quite an impressive woman. Starting with essentially nothing but determination and the ability to focus on her goals, she now owns two related businesses, a musical instrument repair shop and musical instrument sales shop. She holds a degree in flute performance but did the math: one or occasionally two flute chairs per an orchestra, damn few orchestras in the country, equals not a whole lot of employment opportunities as a professional flute player. But, there are a gaggle of student flute players, almost all of them will require maintenance and repair services. And then there’s clarinets, and oboes and other woodwinds (if you’ve never looked closely at an oboe, go to a store and study one for a while - it’s a mechanical marvel of levers, bridges, hinges and valves). Aside from her two stores, she also holds stocks and owns a few rental properties.


thin pipeline? remedy, buy it.

the drugmaker has struck more licensing deals than any other biotech company – 10 in 2014, according to data compiled by Bloomberg Intelligence. It currently has 37 active alliances.
Celgene shelled out an average of $222 million in upfront payments to partners in 2014, compared with the industry average of $70 million,

Now investors will start learning whether Celgene made the right call in investing hundreds of millions in cash since Golumbeski arrived in 2009. Details of a successful midstage trial on a Crohn’s disease drug were published Wednesday, and some of the company’s biggest deals will yield clinical trial results in the next two years.

Inventors and innovators of new biotech drugs tend to be over confident of their chances of success. Moving much of the research outside your biotech company makes impartial analytic decisions about the drug potential easier.