IBL4321, I bought RECPT at $37, and it is over $60 in a matter of weeks. It became my largest holding after the 50% gain (ITMN was before). RCP-1063 may just be the best MS drug that has come along in a decade. The thinking now is that this drug will go head to head with Biogen's Tecfidera or Novartis' Gilenya. Much better safety profile than the other S1P1's and potential best-in-class second generation S1P1. I think a buyout by TEVA or other company is imminent - just a matter of when.
maybe "slow" is the wrong word. I just thought initially that the sequential q/q growth would be stronger, but you already have my thesis as to why perhaps it's not.
Looks like we are both in Avanir...I'm glad they had news today because over the past month I've been debating about getting out of it - hardly any price movement until today. On NPSP, I think the shorts are betting that FDA will require another study at this point - the 8-5 vote does not at all guarantee that this gets approved yet. One of the yes votes said he was on the fence, so this was really more like a split vote. The FDA may demand a study that is designed to better show Quality of Life benefits and further tests different dosing regimens. Both of those topics were heavily discussed according to the live tweeters. Full disclosure: I bailed today on NPSP. I almost did at open but waited and bailed near the low (of course), to redeploy the funds back into another holding: JAZZ. For my taste there's just a lot of uncertainty about what the FDA will require at this point, and with two endo's saying they would hardly prescribe this, I'm worried that the ramp up on this drug could be very slow, sort of like GATTEX. NPSP has a lot to prove. If you're in this for another few years, I don't think you'll have a problem, but I'm on a mission to exceed my last year's gain %, and I'm not feeling it in NPSP at the moment. That by no means suggests that NPSP isn't a fantastic investment!
he didn't quite say stay away.
Here's what he said on Sept 2:
Achillion Pharmaceuticals (NASDAQ:ACHN): "You should stay in that, because it has a lot of upside."
Here's what he said on Sept 11:
"he's willing to take a pass on this stock because it's already up huge, 270%, largely on the promise of a takeover. Cramer said there's nothing wrong with saying "you missed it" but the stock is just too risky at these levels."
the way I interpret this is that if you are just now jumping in just hoping for a buyout, stay away, but if you are investing in it, it has a lot of upside (on its own.)
NPS estimates about 180,000 cases of chronic hyperparathyroidism worldwide, which sounds consistent with 60,000 in the US. So, if 10% of these take Natpara, or 18,000 X $50,000 = $900,000,000. Natpara and Gattex are looking at about $1.5 billion total revenues.
From IMS Health:
"Hypoparathyroidism is a rare endocrine disorder whose incidence and prevalence have not been well defined. This study aimed to 1) estimate the number of insured adult patients with hypoparathyroidism in the United States and 2) obtain physician assessment of disease severity and chronicity. Prevalence was estimated through calculation of diagnoses of hypoparathyroidism in a large proprietary health plan claims database over a 12-month period from October 2007 through September 2008 and projected to the US insured population. Incidence was also calculated from the same database by determining the proportion of total neck surgeries resulting in either transient (≤6 months) or chronic ( 6 months) hypoparathyroidism. A physician primary market research study was conducted to assess disease severity and determine the percentage of new nonsurgical patients with hypoparathyroidism. Incidence data were entered into an epidemiologic model to derive an estimate of prevalence. The diagnosis-based prevalence approach estimated 58,793 insured patients with chronic hypoparathyroidism in the United States. The surgical-based incidence approach yielded 117,342 relevant surgeries resulting in 8901 cases over 12 months. Overall, 7.6% of surgeries resulted in hypoparathyroidism (75% transient, 25% chronic). The prevalence of chronic hypoparathyroidism among insured patients included in the surgical database was estimated to be 58,625. The physician survey found that 75% of cases treated over the past 12 months were reported due to surgery and, among all thyroidectomies and parathyroidectomies and neck dissections performed in a year, 26% resulted in transient hypoparathyroidism and 5% progressed to a chronic state. In conclusion, the two claims-based methods yielded similar estimates of the number of insured patients with chronic hypoparathyroidism in the United States (~58,700). The physician survey was consistent with those calculations...
You're welcome! NPS summarizes the issue nicely from the FDA briefing document:
"Acute symptoms of Hypoparathyroidism are largely due to low serum calcium and range from muscle pain and tingling, to lack of focus or ability to concentrate, and anxiety and depression. In extreme cases, life-threatening events, such as arrhythmias and seizures, may occur. In the absence of an approved parathyroid replacement therapy, the standard approach focuses on using large doses of calcium and active vitamin D to increase calcium levels in the blood and reduce the severity of symptoms. However, balancing the administration of large doses of calcium and vitamin D is challenging due to calcium fluctuations and the long-term use of this regimen may lead to serious complications. In addition, calcium and vitamin D do not correct the abnormal bone metabolism due to PTH deficiency or enable the activation of vitamin D."
the docs were on the fence because Natpara takes them out of their routinesm of prescribing large amounts of D and Calcium and calling it a day. But NPS has prven thoughout this whole development process that the quality of life issues of hypoparathyroidism are enormous and generally completely misunderstood by the medical community. Endocrinologists will experiment on dosing and regimen I'm sure - to see if they can fine tune it for each patient, but I'm hoping that NPS (or Shire after they buy them) will continue a dosing study post approval. Look, any hormone replacement therapy involves risk of too much or too little - there's still a lot we don't understand about hormones - just look at the 10-year controversy surrounding estrogen replacement. And when synthetic HGH was approved, there were all kinds of red flags on that one too - many more than Natpara.
Donna Young @ScripDonnaDC · 1m
#FDA #EMDAC Hallare: Option of $NPSP #Natpar should not be denied to patients
Donna Young @ScripDonnaDC · 1m
#FDA #EMDAC Stanley: Replacing a hormone is way to go; #hypoparathyroidism only endocrine disorder where we don't do that
...and that's the best: Replacing a hormone is way to go; hypoparathyroidism only endocrine disorder where we don't do that
yes I will be surprised if this doesn't pass. You can't be a doc and sit there and listen to twelve patients on Natpara who say it has changed their lives and not take that into consideration!
I may be completely wrong, but I'm getting the feeling this will pass, maybe not an overwhelming majority, but enough. They may be required to do another study to determine optimal dose and dosing regimen, but I've got my fingers crossed that they can do this after approval.