Prosensa: notes from JP Morgan 2013 presentation $SRPT #JPM13

1/7/2013

Prosensa is a privately held biotech company that is the key competitor to Sarepta Therapeutics SRPT in the race to develop exon-skipping drugs to treat the devastating muscle disorder Duchenne Muscular Dystrophy (DMD). Prosensa’s drug drisapersen is more advanced in clinical development than Sarepta’s eteplirsen, and is partnered with UK pharmaceutical giant GlaxoSmithKline GSK. The company was a participant today (January 7, 2013 at 4:30pm PT) at the JP Morgan Healthcare conference, the so-called “Super Bowl” of the biotech investment year.

Prosensa did not webcast their presentation, but thanks to social media, I thought I would try to “crowd source” some notes from the presentation – please use the Comments section, email me, or send me a tweet @BioDueDiligence if you can add any other notes or color from the presentation. The notes below are mainly from Adam Feuerstein (@adamfeuerstein from TheStreet.com – check out his #JPM13 liveblog) and Celia Economides (@ceels66 Wall Street liason to the Biotechnology Industry Organization)

There are ~75,00 DMD patients worldwide
Prosensa is about two year ahead of SRPT in clinical development. There is “lots of potential” for exon skipping drugs
Prosensa has a large partnership with GSK for lead exon 51 skipping drug drisapersen (formerly PRO051) – rest of pipeline is unpartnered
Phase 3 trial for DMD is fully enrolled and three trials completed. GSK is holding placebo-controlled data until June-July timeframe. “DEMAND II” trial data to be released 3q-2013, “DEMAND III” data 4q-2013.
Announced that PRO045 (exon 45 skipping drug) entered the clinic today
Expect orphan drug designations for other pipeline drugs shortly
30 meter improvement in 6 minute walk test (6MWT) is threshold for approval [edit: this of course refers to vs. placebo rather than an absolute improvement..sadly]
the drisapersen study going out to 140 weeks had boys excluded due to rapid progression, just like the much discussed Sarepta eteplirsen trial
drisapersen seems to “minimize decline” in 6MWT performance
Adverse events are renal effects and injection site reactions, but are not causing dropouts

6 Comments

Joan Bar

1/7/2013 11:13:51 am

How many patients of how many enrolled will have to succeed in the "30 meter improvement in 6 minute walk test (6MWT) as threshold for approval" according to the company as per the FDA??

Does SRPT has already in their p2 data probe this?? if yes then because the FDA has guided for "30 meter improvement in 6 minute walk test (6MWT) as threshold for approval then SRPT will AA??

Thx

Shekhar Basu

1/7/2013 11:26:36 am

If GSK did not file Prosensa for accelerated approval as data from a randomized Phase III was required, why would Serepta be able to file for accelerated approval?

dr_mysterio

1/7/2013 09:33:45 pm

"why would Serepta be able to file for accelerated approval?"
Because Sarepta's drug is NON-toxic, while Prosensa's drug appears so toxic that they have to interrupt treatment periodically to allow the patients damaged kidney's to heal.

Joan Bar

1/8/2013 08:53:00 am

Indeed, I think that is definitely the difference and a Big one...

themicrokid

1/8/2013 12:23:54 am

1) It is GSK that would have to file for accelerated approval on Prosensa's exon 51 drug. Big pharma is not always so inclined to participate in accelerated approval programs. The accelerated approval puts companies in a position where they have to launch a product and have a confirmatory trial. If the confirmatory trial does not pass muster, then they have to halt the sales of the drug.

2) All the current data of GSK/Prosensa is not against a placebo. The first placebo controlled data will be released mid year. Therefore, even though there is a lot of patients the lack of placebo control at all makes the data weakish.

3) The Prosensa drug has renal toxicity, and several other possible issues, which has resulted in drug holidays. These show up in as little as 12 weeks in close to 100% of the patients. By comparison the Sarepta (SRPT) drug appears to have no toxicity ofr other issues at 15 months and running.

While the N=12 in the present Sarepta trial seems a small number, This number is about .57% of the applicable US population. And considering the earlier UK trial there is data on almost 1.5% of the applicable US population being treated with the drug at some dosage.

Previously SummerSide research held a call with a former FDA examiner after the 48 week data was reported. He commented on the call, that the FDA would find a way to approve the drug.

themicrokid

1/7/2013 12:39:37 pm

The rest of the pipeline remains unpartnered????

Does this mean GSK is not taking advantage of the option they had on 2 of 4 additional compounds?

6/23/2010

Prosensa and GlaxoSmithKline initiate development of four additional products under existing alliance in Duchenne Muscular Dystrophy - Broadened program marks key inflexion in Prosensa’s progress to a fully integrated specialty-pharma

http://www.prosensa.eu/press-release/prosensa-and-glaxosmithkline-initiate-development-four-additional-products

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Prosensa: notes from JP Morgan 2013 presentation $SRPT #JPM13

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