Hi Ernie, what do you think the up front value of a JV might be? Some on this mb have thrown out 200m. I would be thrilled if they announce a JV with that kind of payment, but I think that is wishful thinking. Maybe you can comment a bit...
This is a valid point about administrative delay. IMO this happened to BMRN during the approval process of Vimizim. The FDA extended BMRN's PDUFA date by 3 months because they wanted more manufacturing data. The CEO of BMRN commented that these were the type of data normally requested at a manufacturing site visit. The FDA wanted an another 3 months to go over 1000 additional pages of documentation out of 1 million pages. Possibly to save face the FDA approved Vimizim 2 weeks prior to the extended PDUFA data.
With that said, I would not be surprised to see the V + C combo approved just prior to the new PDUFA date. This will allow the FDA to brag that they are working hard to get effective therapies to patients in an efficient manner.
husky- I listened to the "makes sense" comment about 4-5 times. I still haven't listened to the entire CC. Did Stoney mention "the launch in Europe" or was that inflammanatory reading between the lines???
If Europe gives BCRX the green light a year or so ahead of the FDA I wonder what they will do? If they go it alone they will need to drop a bundle of $$$ on sales and reimbursement teams for each country in Europe. Heck, maybe this deferral controversy will open the door to a BO. BMRN specializes in orphan drugs and their stock has been pummeled lately (and it looks like BMRN's DMD drug might face competition from SRPT if approved). A late stage acquisition by BMRN might be just what the doctor ordered for both BMRN and BCRX. I realize I am dreaming but it could happen (especially if Opus 2 looks good)...
Anyone know who makes 4161?? Is it the same company that makes Peramivir? Is there any chance there is/was a shortage of clinical grade 4161 due to the same manufacturing issues Peramivir had to deal with a few months ago? Plus, although Opus 1 and 2 are relatively small studies they require multiple pills per day per patient on drug. Maybe the amount of 4161 was limiting??? All I am saying is there must be a reason they delayed the start of the CARC study. Who knows, maybe the FDA recently punted on the deferral conversation and said 'its not worth discussing the deferral until you have Opus 2 data in hand.' At least the EMA has granted a deferral...
My bad Husky,
A little after 27:00 Stonehouse says "... we have to see if it makes sense...". He never said anything about dropping 4161. Sorry for the inaccurate post. Now I am worried about BCRX and my memory.
So did BCRX get a 30% haircut because Stoney said "we have to see if it makes sense"?
Or is the haircut because they might not be able to submit the NDA until 2018?
Is Stoney implying they might discontinue 4161 if they do not get the deferral?
The stock fell because the company announced that they have yet to start a rat carcinogenicity study. According to Thursday's press release, these types of studies are normally required to be available at the time of submission for approval. Thus, the company is seeking a deferral of this study from the FDA. In Thursday's conference call Biocryst's CEO indicated that if they do not get the deferral the company will drop BCX4161. In other words, the fate of BCX4161 is up to the FDA. This is a big deal as 7353 is about 2 years behind 4161. There is also likely some concern about the safety profile of 7353 due to the DTH reaction one patient experienced.
I am not totally surprised by this. A while ago during a quarterly conference call JJ indicated that Kuvan's composition of matter patent was not that strong. I think this is why they decided to alter the Peg-Pal phase III clinical trial. In altering the peg-pal trial I think goal was to show that peg pal is a far superior to Kuvan as far as it's ability to lower phenylalanine levels. If the phase III peg pal data show that peg pal is superior to Kuvan it should help with the marketing of peg pal because many patient's will be able to relax dietary restrictions.
Kuvan was approved in 2007. Sales of Kuvan are decent now but far from the company's expectations. Peg pal development has also been slow. It took several phase II clinical trials to figure out how to dose the drug so it will not provoke an immune response in patient's taking it. Back in 2007 when Kuvan was approved my guess is the company expected to run up the sales of Kuvan quickly and then follow up with peg pal as a second generation PKU therapy a few years later. Fast forward to 2015 and Kuvan's sales are way behind expectations, Kuvan's patent has been challenged and peg pal is still 18 months from approval. In my opinion this is par for the course in biotech.