OCAT the next REGN

OCAT just raised cash today in a very creative non-dilutative financing. They are about to start an efficacy trial for dry age-related macular degeneration (AMD) and a pivotal trial for Stargardt's macular degeneration (SMD). The stock was up 12% today. OCAT only has 40 million shares outstanding, and a market cap of only $150 million. Once they confirm the efficacy they saw in the P1 trial, this stock is going to $300 plus. The market, as REGN investors know, for AMD is HUGE!

Time to take some profits off the table here and invest them in OCAT, in my humble opinion. I see OCAT as being the next REGN and then some.

Your are welcome Chuck.

On the next good press release, I am going to take that information and other information regarding OCAT over to the Regeneron message board and to the Shire message board. There are plenty of shareholders in those companies, particularly at the former, who could benefit by taking some profits and putting them to work at an undervalued company with loads of potential.

Who else is with me?

I actually own enough shares to be on that list. If I get enough supporters, I'll run for a position on the board. The first thing to do is get management to start the European clinical trial for Stargardt's disease. This business of waiting for the FDA to approve their SPA is about as stupid as stupid is. The PRIMARY goal of this company should be to demonstrate statistical significance in a clinical trial of sufficient size.

44 Institutions Increased Position in OCAT Last Quarter

while only thirteen decreased positions:

www.nasdaq.com/symbol/ocat/institutional-holdings/new

Shire want to create Major Presence in Opthalmic Business

October 16, 2014

Good day. My name is Robert Dempsey. I’m the Vice President and Business Unit head of Ophthalmology for Shire. It’s a very exciting time for the organization as we recently announced the origination of a Business Unit, dedicated to the ophthalmic space. We are in the process of building our team, in which we are hiring folks for both commercial, research and development, as well as medical affairs, to be dedicated to the space.

I just had the opportunity to moderate the Pharma session. It was very, very exciting as we are looking for opportunities to enhance our portfolio; both early, mid- and late-stage opportunities for both the interior segment as well as [inaudible 00:00:37]segment. I’d have to say, with our opportunities the financial ability of Shire to be a major player, to have the opportunity to moderate and get to meet some of these leaders in the field, and the opportunity to collaborate with these individuals going forward would be very exciting.

So once again, thank you to OIS for the opportunity to moderate this session, and I look forward to having Shire become a major force in the ophthalmic space. Thank you.

----------------------------------------------------------------

Shire has since made a purchase of an opthalmic business:

Lexington, Mass. – August 3, 2015 – Shire plc (LSE: SHP, NASDAQ: SHPG) announced today that it has acquired New York-based, privately held Foresight Biotherapeutics Inc. for $300 million. With the acquisition, Shire acquires the global rights to FST-100 ... a therapy in late-stage development for the treatment of infectious conjunctivitis, an ocular surface condition commonly referred to as pink eye. This acquisition further strengthens Shire’s late-stage pipeline, has a clear strategic fit with lifitegrast, which is in late-stage development for treatment of dry eye disease, another ocular surface condition, and further demonstrates Shire’s commitment to building a leadership position in ophthalmics.

I have no trouble in getting a reply to my communications with Ocata.

ocat is down on low volume. No fear. Market maker is just taking out the stops.

Is this a CGF-166 clinical trial patient?

I just did a search and found this on Facebook. The person has some hearing in the right ear for the first time and discuss hair cells growing. Does anyone one know if this patient is using GNVC technology in the Novartis clinical trial. Here is the text of the patient's blog:

"Just touched down and reached Kansas City again
smile emoticon
I have post op visit in the morning and I get my ear checked checked out and the packing taken out too I'll let you guys know how it goes its been so weird the past couple of days I could here myself when I am humming in the right ear it's not clear but it something new for me and I never could hear myself in the right ear before even though its not much. I know it takes a while for the haircells to grow but I am just to excited and hope it just keeps getting better."

I do agree with your post. It makes sense. But making sense doesn't establish statistical significance. If it did, the company would have put it together and put out better numbers post the 9-7-12 numbers. The best they could do post sabotage, with the FDA's blessing I suppose, was the 0.217. If you want to extrapolate from the 9-7-12 release, you of course are free to do so. I will not. To me it is just one data point, and not that great of one at that.

Nonetheless, I am sure they got together with the statisticians to make sure SUNRISE was powered correctly. Normally, I do not buy heavily into a company that doesn't already have published SS data. I suppose there are many who see things like me, which is why this stock is priced the way it is. Make no mistake, this is a very risky investment, but with the greater risks come greater rewards or greater losses. Usually you have better data to rely on when investing in a company that is in P3. The CSM fiasco really messed this company up, no doubt about it. If the P3 data is positive, CSM will be have shown to have cost PPHM hundreds of millions of dollars. Without P3, the big damages are speculative. And since the lawsuit will go to trial before the P3 results are in, it seems difficult for PPHM to prove the big money. We will see. It sure would be nice if the case settles for $50 to $100 million or more.

Why were front line bavi treatment results not so great?

TUSTIN, CA -- (MARKET WIRE) -- 03/09/12 -- Peregrine Pharmaceuticals, Inc. (NASDAQ: PPHM) today announced top-line overall response rate (ORR) and current median progression free survival (PFS) estimates from its phase II trial comparing bavituximab plus carboplatin and paclitaxel versus carboplatin and paclitaxel alone in patients with front-line Stage IIIb and Stage IV non-small cell lung cancer (NSCLC).

Based on investigator assessments, patients treated with bavituximab plus carboplatin and paclitaxel demonstrated a current median PFS estimate of 5.8 months versus 4.6 months in patients treated with carboplatin and paclitaxel alone, a 26% improvement. These results are consistent with a prior phase II single-arm study testing the same bavituximab combination in front-line NSCLC patients which showed a 6.1 month median PFS and with several prior published studies with carboplatin and paclitaxel in front-line patients that showed approximately a 4.5 month median PFS. Based on independent central imaging reads, patients demonstrated a current median PFS estimate of 6.7 months for the bavituximab-containing arm and 6.4 months for the chemotherapy-only arm. Peregrine expects to report median overall survival (OS) from this trial in the second half of 2012.

"We are pleased that the PFS results for the bavituximab-containing arm by both local and central image interpretation actually met or exceeded our expectations going into the study. While the data from the investigator assessments were in alignment with previous published reports for carboplatin and paclitaxel and suggested an encouraging difference between the treatment arms, the unexpected long PFS estimate for the control arm based on central reads confounds our ability to fully interpret this secondary efficacy endpoint," said Joseph Shan, vice president, clinical & regulatory affairs at Peregrine. "We now await median OS data from this study which is the most clinically relevant endpoint from a drug development standpoint."

And were the OS results ever published? I could not find a press release for the second half of 2012.

CP, both individual arms in the 9-7-12 press release were not statistically significant.

Here are the P values from 9-7-12:

placebo n/a

1mg = .0286

3mg = .0714

1 and 3mg pooled = .0154

The 3mg arm was not statistically significant.

In any event, none of these numbers have any value at all since these vials were mixed up and mislabeled -- so none of them show SS. Why the 1mg and pooled were SS but the 3mg was not is anyone's guess. The statistician at the time could explain it, but that matter is worthless to investigate at this point.

The only number that has any meaning is the subsequently published P value for the 3mg compared to the 1mg in combination with the placebo. Here we get a P of 0.217, which is not statistically significant by any measure.

So to be long this stock, you need to be comfortable believing the P is going to make the major jump from 0.217 to 0.05 or better based on the number of participants climbing beyond 600 and only placebo being in the placebo arm.

My advice to you would be to forget about the 9-7-12 numbers. You can't make wine out of manure.

Pharmaceutical companies commonly get SPAs for P3 trials. You can learn more here:

http://www.fda.gov/downloads/Drugs/Guidances/ucm080571.pdf

I don't see where Merix was bought out by BMY.

I owned IMCL at the time BMY bought them out. There never was a clinical trial foul up at IMCL. Nor was there one where CSM was involved. The only scandal at IMCL was Sam Waksal telling that blond home economics lady to sell while the selling was good, resulting in jail time for the both of them. BMY didn't buy IMCL until their HER-2 targeting mab was on the market.

So the statement "BMS has a long record of buying small bios after failed trials at CSM" seems to be fully erroneous.

north40000,

Thank you for your editing. Yes, I should have said the FDA is commonly looking for a "P value of 0.05 or better." Not a P value "greater than 0.05". "Greater than" is confusing. Good catch.

EndTheFed

"BMS has a long record of buying small bios after failed trials at CSM."

Name some of the others please.

exwannabe,

Thank you for the information. I wonder why PPHM did not get a SPA?

I also agree with your final paragraph.

CP,

I've been investing in biotechs for over 20 years. It has always been my understanding that a P value greater than 0.05 was considered to be statistically significant. The FDA commonly requires two trials showing efficacy, a phase 2 and a phase 3 or two phase 3s. They want to ensure that the one good trial wasn't just a random outcome. Since the unavailability of a drug for a cancer patient can lead to death, the FDA has been more tolerant for life threatening diseases and may approve a drug based on clinically significant P2 data alone. Peregrine may have gotten bavi approved based on the P2 data but for Jeneatte Bleaker's (sp?) activities. You asked for FDA documents stating that a P of 0.05 or better is standard. Please see the following at footnote 5:

http://www.fda.gov/downloads/Drugs/.../Guidances/ucm078749.pdf

Please also see the following NIH document about 30 paragraphs down:

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC534930/

>It is widely known that the Agency will conclude that a trial is considered “positive” if the p value generated for the between-treatment contrast is less than or equal to 0.05. What does this mean?<

So my questions for you and the others are as follows:

1. Did Peregrine get a SPA for the SUNRISE trial from the FDA?

2. If so, does the SPA require a P value of 0.02 or better for SUNRISE?

3. If there is no SPA for SUNRISE, where is the 0.02 or better P value coming from?

>>Last paragraph, you wrote:

"from 0.217 to 0.5"---do you mean .217 to 0.05 ?<<

Answer = yes

CP,

We don't seem to be understanding one another. I apologized in an earlier post, but it must have been taken down because I don't see it on the board anymore. Don't know why anyone would have done that. But back to the statistical significance discussion.

You say "The requirement of the FDA for 0.02 to be stat. sig was posted in the FDA guidelines and the slides."

I am sure you know that the FDA commonly requires a P value or 0.05 or better to demonstrate statistical significance in clinical trials. Are you saying that the FDA is requiring a P value or 0.02 or better for P3 SUNRISE? Or are you saying the FDA wanted a P value of 0.02 or better in order for Peregrine to claim statistical significance for the P2 trial? If it is the former, that is a much higher hurdle for PPHM to meet even with n being much higher. If that is the case, that the FDA wants P = 0.02 or better, why is the FDA demanding a higher threshold for this P3 trial? If it is the latter, that is the first I have heard of such a thing in a phase 2 trial. In any case, I was totally ignorant of the FDA requiring a higher threshold whether it be in the P2 or in the P3. Please accept my apologies.

In my post that was taken down, I pointed out how I was in agreement that the 9-7-12 press release P values should have been higher but for the vial mixing. There probably was statistical significance shown there, but because of the all switching and relabeling there is no way anyone can conclusively state that statistical significance was demonstrated. The company is not taking that position, and it knows the matter fairly well I'd assume.

The P = 0.02, which I was referring to, was taken from the February 3, 2013 press release:

TUSTIN, CA -- (Marketwire) -- 02/19/13 -- Peregrine Pharmaceuticals, Inc. (NASDAQ: PPHM), today reported data from its randomized, double-blind placebo-controlled Phase II trial of bavituximab in patients with second-line non-small cell lung cancer (NSCLC). Data from the trial has been updated based on completion of an earlier review of discrepancies in the trial and the most current survival data from the trial. Updated results from this Phase II trial indicate a meaningful improvement in median overall survival of 11.7 months in the 3mg/kg bavituximab + docetaxel arm compared to 7.3 months in the control arm (HR=0.73; p value=0.217).

This number, 0.217 to be exact, was taken after the mixup/sabotage was discovered and the data was given its most thorough assessment. Now clearly this is not statistically significant.

So what everyone who is long this stock is betting is that, with a larger patient pool and with no patients in the comparator arm getting bavi, the P value should move from 0.217 in P2 to 0.02 in SUNRISE. I was thinking it would only have to go from 0.217 to 0.5, the latter being the commonly excepted value for FDA approval. But if you are saying, the FDA now wants 0.02, we've got a fairly good hurdle to meet. Are we both in agreement now?

P = 0.0154 Press Release for NSCLC P2 (pre sabotage discovery)

Anyone have a link to this or a copy of it? The website doesn't go back that far.

CP,

So they just changed the trial date. Thanks for the information. You saved me a visit to Pacer. It will be interesting to read the judge's decision on the motion for summary judgment.

In regard to statistical significance, you got a few things wrong. First, the FDA requires a P value of 0.05 or less to show that the drug is responsible for meeting the end point. The FDA does NOT require a P value of less than 0.02 (~98% probability) like you said. The FDA will or may only require a P value less than 0.05 when there is only one controlled trial showing efficacy. Why did you state something so erroneous?

Second, the number 0.2, which I posted, (not 0.02, which you posted) was taken from PPHM's press release on the P2 trial. This number was NOT statistically significant. It only represents something like an 80% probability that the drug is working. But like we have both said, bavituximab was competing against itself since the control arm included the 1 mg vials. The number 0.2 does SUGGEST the drug MAY be working, but it does not meet the statistical significance threshold. Therefore, it represents a null hypothesis.

You have said repeatedly that Peregrine showed statistical significance in their P2 trial. I have asked you to explain how you arrived at this conclusion. You have not given a cogent explanation. Thus, I am presently of the view, which is in accordance with PPHM's press release, that P is 0.2. We have a null hypothesis and will continue to have one until such time as the statisticians look at the trial in the interim or at completion.

CP,

Thank you for the very informative post. I also have been reading your assessment of the Peregrine v. CSM litigation. You've done a great job explaining that as well. One point I would add on the dispute: it is scheduled to go to trial this September. It is highly unlikely that any motion for summary judgment will be granted. Thus, the judge will not make the decision on this one. It will be left for the jury. Since the venue is in California, that will give PPHM an advantage. The facts are terrible for CSM. I can't see how they or the insurance company won't settle. A jury award could be huge given the despicable conduct by JB et al. It sure would be nice if a retired person or someone living in the area could watch the trial and report back on this MB.

With respect to your kind response to my question, it raised one further question for me. You said "[a]nd Docetaxel+Bavituximab's 113% over SOC which was statistical significant". When I read the press release on the P2 trial (after combining the 1mg with placebo and calling it the controlled arm), I saw a P =.02 being reported, which would not be statistically significant. Rather it would be an 80% probability that the drug was working, not the 95% or P = .05 that the FDA needs to call it statistically significant. But since the 3mg arm was competing against itself in that the 1 mg arm was now part of the controlled or placebo arm, I viewed the P = .02 as being a very good likelihood that statistical significance would have been demonstrated but for the 1 mg arm being part of the control arm, that is, but for the sabotage.

Can you please explain how you are of the view that statistical significance was demonstrated in the sabotaged P2 trial? I know others have indicated this as well, but I have yet to come to understand how it could possibly be so categorized. Thanks in advance for any reply you give.

Well if Opdivo was just halted for non-squamous (thanks for the info), then how is Peregrine going to get patients enrolled in its clinical trial? OUS I suppose, but what about US?

The present phase 3 clinical trial will give no data by which Bavituximab can be compared to Optivo. One is for squamous, the other is for non-squamous.