First, I do not have any idea, explanation, or guess, or....you name it,...why REGN have tide lips about ongoing clinical trials, and no respect for investors. I tried several time to get more info, but each time find close door at REGN. With limited source and time, I went around, collect small peace where and when I can, and tried to build *big picture*. Not a easy job.
Second, data which I collected are from medical letters, journals,...so they may not present clear and true picture about what is going on at REGN. Also, some of my extrapolation and probably speculation may be out of reality.
So, before we jump left or right, up or down, let first say *hop*.
NT-3:
<< Initial, initial, initial. How about some sort of indication concerning patient accrual?>>
First safety trials results (PI in healthy volunteers) rise concern about side effects at higher doses. REGN/AMGN than run large PII trial in DN, to test drug range of doses. Trials turned out negative, not sufficient activity, while AE continue to be problem. At the some time company identified sub-population of pts with constipation (also in BDGF ALS PIII trials) who may benefit from drug. To test this hypothesis company have to run PI/II trials (health condition and drug tolerability of DN pts are not the some as for cancer, SCI or late stage P's pts), before going in to large pilot PII with defined dose/schedule. The better they do early job the easy will be (if applicable) to run and complete large PII/III trials. They rushed with BDGF, so no more similar mistakes. Regardless will drug reach market or not.
I am for as many *initial* trials as it may take before they spent hundreds of M for another failure!
Market value NT-3 with ~0 M. NO PROBLEM!
BDGF:
<< How long has this trial been ongoing now? About 45 years or so? Well, it seems that way. Again, any guidance with respect to patient accrual?>>
History: After failure of PIII (ALS) and after sub-pts analysis, company think that certain pts did benefit from drug. Never found who are this sub-population pts.
wfnals.org
wfnals.org
To test hypothesis (or maybe new higher doses adjusted to each individuals):
(posted on ALS web site on Feb 1999)
wfnals.org
<< Regeneron has conducted two preparatory studies prior to commencing a repeat efficacy study. The efficacy study will commence during the first quarter of 1999. There will be 10 sites, all in the US. The list of sites is included below. The objective of the study is to determine if BDNF doses which have been individualized to tolerability up to a maximum daily allowable dose can influence survival in ALS. Interested patients should contact the sites directly.>>
They conduced small multiple (I think two PI/II trials) prospective studies. Started in late 97/early 98 and completed by late 98/early 99. I think, currently at final analysis stage. I hope they will disclose (one day???) data from this trials. Nonetheless, based on early analysis of this data company disclose plan to start new pivotal trials (in 99) for ALS.
At the some time, AMGN completed intrathecally BDNF PI safety trial (EU and US, started early 97, completed in 98), and in late 98 started PII (I think ~120 pts). Currently ongoing:
wfnals.org
This two trials, contrary to PI and PI/II trials, are 18 months trials, the only way to determine survival benefit of any drug for ALS.
Yes, they may seams like 45 years, but it may take another +45 years before any drug show benefit for ALS. :(
Also, Sumitomo license (for 5 M) BDNF for Japan market and paid ~5 M for BDNF initial clinical quantity.
So, it is hard to tell does BDNF have any potential for ALS. But, is there anything which may work? NO, and again NO.
The problem is to delivery drug (any drug) where it is needed.
Market value this program ~0 M. I HAVE A PROBLEM with this valuation!
Regardless that drug has small probability to reach market. I can name many candidates with lower probability, no clinical trials experience, still market value programs in hundred M!
<< Who's selling, MZ? Anyone who had hopes that the lymphokine traps or that the angiogenesis programs were part of REGN's near-term future. Isn't it oh so nice to pay for all that dead-end research? [hint... the answer is "no"]>>
Do not agree here.
First, lymphokine traps was low level priority at REGN for years. Second, Angiopoetins, MUSKs, Nagin, MAP kinases,...are scientific staff. To transfer this in visible drug development cycle at the advance stage (IND and clinical trials) one has to be sure that approach and technique are validated. I hope YOU will agree that one out of ten discovery do make to IND (transfer of science into drug candidate) and one out of five make to NDA. So, it is always *dead-end-research* for something new which we (science community) do not understand.
I will say that lack of the near term dug candidate which may reach market (min 2 years for any pivotal data on BDNF and more than three for NT-3) are main reason for today REGN position.
Some is going for AXO (no need to repeat myself):
Message 9069757
Message 9092714
<<That makes fair value for P&G to be about $7.25/share, plus whatever current value they place on the clinical projects. I'll take $9/share.
Truly one of biotech's biggest disappointments. Let's hope that P&G can turn things around.>>
If you add remaining part of Merck manufacturing contract the disparity for current market value versus cash, manufacturing site, tax care-forward, and sure contract revenue,.. are even bigger. Their all programs are valued at less than zero, negative.
Yes, REGN was bt great disappointment if you count trials failure (CNTF, BDNF, NT-3). Can you name one bt with similar neuro-factors which did succeed?
The problems is *FILD* where they are involved, not lack of results.
Normally, no single investor is enough stupid to pay for failure or science which can't generate commercial value. However, I believe that one day REGN science will generate commercial value and my investment will be rewarded.
Cheers,
Miljenko
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