Marie Curie 74 TC1
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And this is on top of Jan 3 18 offering 8.05 M sh * $18.25 = $146.91M
IMO MRNS ganax probably won't work in PPD. MRNS ganax efficacy is pretty
weak vs SAGE-547 or SAGE-217. This analysis by Ohad Hammer is pretty old but probably still applies.
http://www.orf-blog.com/sage-and-marinus-not-the-typical-cns-story/
For the last two years, MRNS more or less is doing trial for orphan
indication with N < 10. They seem to take forever to do this MDD trial
https://clinicaltrials.gov/ct2/show/NCT02900092?term=Ganaxolone&draw=2&rank=14
which started in Nov 2016 and ending in Jan 2019. At the last update Oct 2017
they managed to enroll 10.
MRNS was a good short term trade. In July I got in @1.53, sold it quickly
2 months later for @3.06. At this price, I am not sure if it is a good
short term trade.
I don't know how outstanding shares are counted but in the 13G it looks
like TRIL has 13,146,239 Common Shares so the math is correct
as 2,173,000 sh is 16.5% of the above
https://www.sec.gov/Archives/edgar/data/1410830/000090266417004518/p17-2299sc13g.htm
Looking at the item 4 in the document
Item 4. OWNERSHIP.
The information required by Items 4(a) - (c) is set forth in Rows 5 - 11 of the cover page for each of the Reporting Persons and is incorporated herein by reference.
The Company informed the Reporting Persons in writing that as of December 5, 2017, there were 13,146,239 Common Shares outstanding. The percentages set forth herein are calculated based upon such number of outstanding Common Shares.
Thanks Dew for a great stock recommendation.
You are talking about plasma concentration, not the actual dosage intake. The poster is from Sage, which is bragging about its oral
bioavailability
Compare the PR on Feb 13 2017
Quote:
Sage Therapeutics (NASDAQ:SAGE), a clinical-stage biopharmaceutical company developing novel medicines to treat life-altering central nervous system (CNS) disorders, today announced encouraging top-line results from its Phase 2 clinical trial of orally-administered SAGE-217 for the treatment of major depressive disorder (MDD). The primary endpoint of the 13 patient Part A open-label trial was to evaluate safety and tolerability. SAGE-217 was found to be generally well-tolerated with no serious adverse events or discontinuations reported. The trial also examined the effect of SAGE-217 on the Hamilton Rating Scale for Depression (HAM-D) total score, in addition to other secondary measures. Patients in the trial had a mean HAM-D total score of 27.2 at baseline. Data demonstrated a mean reduction from baseline in the HAM-D of 19.9 points at Day 15, with 85% (11 of 13) patients showing at least a 50% reduction of their HAM-D and 62% (8 of 13) of patients achieving remission, as determined by a HAM-D ≤7. Statistically significant mean change from baseline was observed by Day 2 of the study, following the first of once-daily, nighttime oral dosing of 30 mg of SAGE-217.
vs the PR from Marinus on June 13 2016
Quote:
The Phase 3 multi-national, double-blind, randomized, placebo-controlled study was conducted at 61 sites in the United States, Germany, Poland, Australia, Bulgaria and Russia. The study randomized 359 patients into two arms, ganaxolone 1800 mg/day (n=179) and placebo (n=180). The mean age of the study population was 41 with 63% percent of the participants being female. Mean years since epilepsy diagnosis was 24.5 years. 76% of the patients were receiving two or more concomitant AED's and 12% had been treated with Vagus Nerve Stimulator.
ganaxolone has shown to have less efficacy than SAGE-547 for epilepsy. The advantage for ganaxolone is the safer toxic profile which allows for long term use.
That is why SAGE-547 is targeted at SRSE the acute form of epilepsy where
long term use is not needed. PPD is another indication where long term use
is not needed if the drug actually works.
Ganaxolone has never been tested in PPD. Captisol might not help much
if the efficacy is poor in the first place, but this is just my opinion, not
based on any scientific evidence. It could work in PPD.
Ecor1 does not hold any SAGE in 2017. Deerfield sold all their SAGE in 2Q17
as their filings showed. I started buying MRNS when I noticed Deerfield buying
into MRNS but I am out of it since last week at a good profit.
Two things to consider
On their Top-line Data (two first bullet items in PR):
- The median change in 28-day seizure frequency from baseline in the ITT (intent-to-treat) population (primary endpoint) was a decrease of 43% (n=7).
- The median change from baseline in seizure-free days in the ITT population (key secondary endpoint) was an increase of 78% (n=5; two subjects cannot be calculated due to 0 baseline seizure-free days).
The sample size is too small n=7 and they play fast and loose with the ITT
(switching from n=7 in 1st bullet item to n=5 in 2nd bullet).
On the other hand
Ecor1 acquired 12.2% MRNS (reported 8/8/17 in Edgar)
Deerfield acquired 9.98% MRNS (reported 7/17/17 in Edgar)
FWIW I sold all my position on MRNS
Not at all. ACAD drug is for psychosis, ADMS drug is for dyskinesia very different indication.
Initially SAGE-547 is an IV formulation that goes after Super-Refractory Status Epilepticus which is the acute form of seizure while MRNS ganaxolone is
an oral formulation for focal seizure which is a chronic condition.
While SAGE-547 was successful in SRSE and later on Post Partum Depression (PPD), ganaxolone failed in various indications of seizure including pediatric application.
Then MRNS copied SAGE and reformulated ganaxolone as IV to go after PPD and SRSE but they have not started any trial for SRSE yet, only PPD first.
The only trial that completed is CDKL5 pediatric epilepsy which is an open label trial. There is no POC that ganaxolone would work in IV for SRSE or PPD, look like a hail Mary to me IMO.
In the mean time SAGE developed the 2nd generation SAGE-217 which is an
oral formulation much more potent than SAGE-547 for PPD and SRSE.
MRNS has 20M in cash as of June 30 17 vs 285.9M in cash for SAGE, too poor
to do any meaningful development.
According to the clinicaltrials site
Primary Completion Date: June 27, 2017 (Final data collection date for primary outcome measure)
https://clinicaltrials.gov/ct2/show/NCT02358538?term=ganaxolone&rank=3
IMO may be some info leaked from the study. I noticed that Deerfield
acquired 9.98% 2.238 M sh of MRNS on 7/14/17 and reported it 7/17/17
Also EcoR1 acquired 12.2% 3,622,271 sh of MRNS on 8/4/17 reported it 8/8/17
It is an open label study so it may not be a definite proof of ganaxolone
efficacy but for a penny stock like MRNS it could be a big bump.
I believe SAGE is a better buy in the long term. In early July before
these hedge funds purchase the stock languished around $1.30-$1.40
IMO BLCM mgmt kept delaying BPX-501 app with FDA so it is dead money to
the market.
With AFMD their lead drug AFM-13 is in p1b after almost 2 years without much progress. Dead money too.
FWIW the dogs in my portfolio are AFMD, GNMX, KPTI. BLCM is a marginal dog
as my basis is around $10 but I probably get rid of it soon. If EHA in
Cairo Sept 14 has no progress update from BLCM then I will sell.
I have
AFMD
ARQL
BLCM
BPMC
FGEN
KPTI
GNMX
MRNS
RVNC
TRIL
MRNS is just a short term position to go with momo crowd as
Deerfield bought 10% and Ecor1 bought 12% recently. I bought
mine just after Deerfield purchase. My basis is $1.50. SAGE
is a better long term play.
Congrats Dew. Your postings help me learn a lot about biotech investing.
Tivantinib is a failure. However ARQ 087 data is quite promising,
could get accelerated approval in 2nd line iCCA (intrahepatic Cholangiocarcinoma).
Management is questionable, given past failure but at this price
it is a decent risk/reward trade. Don't be obsessed about the past.
I used to own GLPG but I have not followed it closely after I sold it in
the 60s. I was disappointed at the low royalty rate for filgotinib in
RA and CF with Abbvie. I think they get a better deal in RA with GILD
after Abbvie dropped filgotinib in RA but I am not sure. FWIW
But doctors would prefer more frequent injections to get more revenue, why
would they switch to RVNC that lowers the frequency? Most patients don't know enough
to demand RVNC vs AGN, assuming that RVNC drug lasts noticeably longer.
CALA truly licensed from the Mars corporation that makes Mars candies bars.
Technically it is from the Mars Symbiosciences subsidiary.
GSK filed on June 30 2016 for inter partes review of FGEN Patent 8,609,646 was denied review by the Patent Office today. Explain why FGEN is up while
most biotechs go down
This is just one patent out the six that Glaxo requested a ipr.
(8,466,172; 8,614,204; 8,629,131; 8,604,012; 8,609,646; and 8,604,013)
What do you expect with former CEO of Dendreon in charge? Top notch management
Do I also need adenosylcobalamin in addition to methylcobalamine as this
paper imply? TIA
https://www.ncbi.nlm.nih.gov/pubmed/25117994
I agree. They all follow the same script, comparing a company with its peers
using average sell side analysts prospect for earnings, growth, etc.
Then it recommends to invest in an ETF for safety as an alternative
The reason Market Realist thinks stock ABC is better than its competition
is dubious.
For example in the case of BMY
Quote:
Opdivo doesn’t require PD-L1 testing
Keytruda’s trials were tightly focused and included a subset of the population with high levels of the biomarker, PD-L1 (programmed death-ligand 1). By contrast, Bristol’s multiple trials for Opdivo included a broader population base. Although it was riskier, the success across multiple trials has helped Opdivo pick up faster in sales.
Notably, during the first nine months of 2016, Keytruda generated $919 million for Merck while Opdivo added $2.5 billion to Bristol’s top line. Unlike Keytruda, Opdivo doesn’t require any biomarker testing and thus caters to a wider lung cancer population. This has given Opdivo an edge over Keytruda.
End quote
I thought this is the downside of BMY vs MRK lately as all comers from
BMY is not as good as MRK (>50% PD-L1) in lung cancer.
IMHO not a buy. The clue that the CRL is not simple to fix 6 month from now
and requires
more money is the statement that the company is looking for a partner.
Minor typo: OCRX had $32.5M cash at 9/30/16 (not 9/30/15)
I have seen this "breakthrough" almost 2 years ago. Count me as a skeptic
http://replicor.com/replicor-biopharmaceutical-company-targeting-cure-chronic-hepatitis-b-patients-will-present-updated-human-clinical-trial-data-documenting-4-6-log-reduction-s-antigen-long-term-sv/
Following the deal with Amgen in August that validated their pipeline stock went up to $16.
I could not believe my luck when it came down to around $10 in September.
Loaded up a bunch around $10.60. Thanks for your stock recommendation.
At least CLVS rucaparib has a PDUFA date with MC $1B vs
TSRO niraparib at $5b this morning without a PDUFA date
Sorry I did not write down the place where I have seen that.
The last entry in my research note I tell myself to forget about this company, probably at the time it was not clear cut to me trabodenoson can provide better efficacy vs. generic latanoprost and timolol.
Basically you have to pay attention to all presentation from companies
at JP Morgan healthcare conf in Jan, trial results ASCO in June, ASH in Dec. Also
their European counterparts ESMO, EHA. At the minimum look at the posters
so you can compare apple to almost apple between various drugs.
Listen to all their CC on earnings and trial results. After a while you can
tell good management from bad, etc
It is a lot of work but once you start doing you will get a feel for it as
to which companies are better. After losing money a few times hopefully you
get better. You have to accept that half of your picks will turn out to
be wrong.
I find these 2 books useful for biostatistics
Intuitive Biostatistics Harvey Motulsky
Statistics: a biomedical introduction Brown Hollander
In the CC, management explained that those deaths in low dose arm would have counted as failures but it still has stat sig. The patients on low dose
are sicker than the patients on high dose which are in better shape thus
they could tolerate high dose.
That is why more patients in low dose died (10) vs patients in high dose (2).
This is a small position for me so I bought some more (not recommended for
others).
I believe that the drug does work and the trial is still continuing.
Today is just top line result and they have better analysis by year end.
We will see if I am throwing good money after bad or the gambit works
AUPH reports phase 2b study meeting top line results
http://finance.yahoo.com/news/aurinia-pharmaceuticals-announces-voclosporin-meets-100000140.html
I have not followed ITEK trabodenoson closely for a while so take this with a grain of salt.
Last I remember trabodenoson + latanaprost failed to achieve the level of IOP lowering as Timolol (a beta-blocker) + latanaprost in a p2 study sometime in
early 2015.
Since then they have done anything except raising money.
They had a "positive" end of p2 meeting with FDA July 15 and started p3 last year.
I have doubt as to their chance of success but I could be wrong.
Look at these sell side geniuses. Just before stock crashes. LOL
http://www.fidaily.com/cowen-company-starts-coverage-on-seres-therapeutics-inc-mcrb-with-a-rating-of-outperform/236948/ted-blackburn
Thanks but I don't think that was it. The presentation on CARA was on July 19
in Brighton UK.
Is anything going on with Cara Therapeutics? It closed yesterday
at 5 and is around 6.17 now (20% rise).
Berens still maintains price target at $9 for RLYP because he predicts deal will not go through
I agree with it. MNTA has several biosimilars and the path to approval is
a lot easier now.