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written by reader The eyes and ears team for bio trials

By , February 26, 2014

This discussion came about as the result of the GS thread ”This Tiny, Unknown Biotech is About to Unleash Its ‘Holy Grail’ Drug”

It is hoped that all/any that can throw light, can brainstorm here and feedback consensus to the thread. Good work Guys. my best Alan Harris.

This is a discussion topic or guest posting submitted by a Stock Gumshoe reader. The content has not been edited or reviewed by Stock Gumshoe, and any opinions expressed are those of the author alone.

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karmaswimswami
March 9, 2014 5:32 pm

Professor Z: My view is different about RNN and Supinoxin. It took three years to find this target, develop and agent and get the agent into clinical trials. For Rexahn to have done this in that time frame is just excellent. The drug is only now in phase I. We should have data on that within 90 days. Phase I for a cancer drug is a weird thing: you want to see toxicity, but not excessive toxicity. You want people not to get so deathly ill they cannot take it, but also have to see some evidence that their tumors shrunk (though that isn’t the primary endpoint of phase I). But if the data from phase I isn’t good, I will sell and so should you. I predict it will be good because from the preclinical data, its synergy with other agents is astounding:
http://www.rexahn.com/cms/portfolio/rx-5902/

Look at the last graph in that link.
Despite all our modeling and knowledge, however, the body is still a black box. We do not know what will happen when we put something into it. Which is why we have clinical trials. RNN may continue to muck about with Archexin. I don’t care. But if things look good for Supinoxin in phase I, I see this company really growing considerably in value. And at that point, for all I care they can dump Archexin. My personal read of the tea leaves is that maybe they raised capital recently BECAUSE things are looking good in phase I for the p68 inhibitor. Frankly I feel that investors would have been reluctant to get in just to fund Archexin studies, as things for it are not auspicious.

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David B
Guest
March 9, 2014 8:07 pm

Good clarifications on RNN Dr. K.

karmaswimswami
March 9, 2014 8:15 pm

David B: for the phase I there will be tox considerations obviously but also some look for a beneficial effect….tumor size, tumor multiplicity. And if there is none, then I think we all probably need to exit. But the flipside is that if things look good, it suddenly may be too late to get a bargain price. I will try to have a look at the study protocol again in the next day or so.

David B
Guest
March 10, 2014 10:32 pm
Reply to  karmaswimswami

Yes, I think that there is an excellent chance that Supinoxin will show that beneficial effect and then perhaps we can counter Adam F. Who knows–even Archexin has a shot at making it.

sfimar
sfimar
March 10, 2014 12:30 am

Great information here as well…and the excel doc looks nice…

Many many Thanks!!

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arch1
March 10, 2014 4:04 am

Gumshoers ; Please. A caution . Jer-vic is correct when he says spreadsheet should be a dashboard. A starting point. In this litigious society do you want to be held liable for stock recommendations or insider trading or expose Karma to that? You must do your own due diligence! Do you realise how valuable the opinion of someone in the field is? Not to mention all the other info on where treatment is going & views on common mis-conceptions. As Travis pointed out the spreadsheet is public and can not be made otherwise. “Buy” recommendation is walking too close to the edge in my opinion. I claim no expertise in this except long experience in where pitfalls may lie. Please think this over carefully.

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Opposeablethumb
Irregular
March 10, 2014 8:38 am
Reply to  arch1

Agree 100%. Who is recommending the buy? Not Travis and not Dr. KSS. Even if they state they are buying that does not mean we must. My understand is DR.KSS is rendering an opinion about science. That opinion is positive or negative regarding the science and its ability to address an issue. He is also a buy and hold investor, states do your own DD, and is a fallible human being. We value his opinion highly, ultimately our gains and losses are our own responsibility. Even the most knowledgeable people make mistakes, they just make less of them. Stock prices will do what they will but understanding why we are in a stock can allow us to accumulate or exit based on a sound thesis. To me that is the real value!

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JustIrregular
March 11, 2014 9:48 pm

Thank You Frank Opp0seablethumb, jer_vic and Dr.KSS. I was one of the guys that suggested the buy column on the other blob. I just now see what it means to Dr. KSS if he was to it state that. At the most it could read “Potential Buy”. Sorry for the error and I hope this does not cause anyone harm! It probably should have the Disclaimer at the bottom of the page of the Spreadsheet.

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KennyG
Guest
KennyG
March 10, 2014 8:40 am
Reply to  arch1

A simple way around that would be just keep the “positive/negative” column and remove the “Buy” column.

Alan Harris
Guest
Alan Harris
March 10, 2014 1:21 pm
Reply to  arch1

Anyone who thinks GS, Travis, Myron, Doc. KSS or anyone else on this site, is giving individuals or the many investment advice is a PLANK. Its your money, NO ONE is telling you how to spend it……….. Period.

bwd1up
bwd1up
March 10, 2014 11:19 am

Frank Post #45: Perhaps Travis has an opinion on whether there should be a disclaimer somewhere on the spreadsheet, or even better, are these threads covered under Stock Gumshoes disclaimer?

Is a shame we have to consider something like this, but it is worth a thought.

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Travis Johnson, Stock Gumshoe
March 10, 2014 11:44 am
Reply to  bwd1up

Yes, always worth a thought. I spent a couple weeks responding to a subpoena recently, and would delight in avoiding them whenever possible.

As regards the columns about Dr. KSS’s opinion, I’d suggest inserting the link to what he might have put as his opinion at whatever date and time that he or others might have opined. You can copy and paste the link to any individual comment, it’s the timestamp (ie, Bradley’s “March 10, 2014 at 11:19am” is a link, it goes specifically to that comment at http://www.stockgumshoe.com/2014/02/microblog-the-eyes-and-ears-team-for-bio-trials/#comment-1284640). You can right click on it and copy link, then paste the link into the spreadsheet if you like.

I didn’t put in the buy columns and would urge folks not to put words in anyone else’s mouth by calling something a “BUY” based on a comment they read, particularly if they’re not going to then go back and change it when that person’s opinion changes, or when the opinion is nuanced and may be based on something specific or on a specific price. Just link to the opinion, and the link can always be replaced with a new one if and when it’s updated. And, of course, while Dr. KSS is a valuable asset to the group and I don’t speak for him, he is certainly not managing anyone’s portfolio for them. I think this spreadsheet is likely to be a lot more valuable as a source of ideas and tracking of catalysts and discussion than as a buy/sell list.

We are also, in response to popular demand, bringing on karmaswimswami (Dr. KSS) as a more regular contributor here at Stock Gumshoe, so hopefully he and the rest of the gang will have plenty to talk about in the months to come — his first submission will go live this afternoon to the Irregulars, and I’m really looking forward to hearing what he has to say as we go forward.

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jer_vic
jer_vic
March 10, 2014 12:11 pm

Agreed. I don’t know who added the “BUY” column, but I just removed it. Also, I see a non-biotech stock in there – EXEL. I’ve highlighted the row in red, added a comment that it’s not a biotech stock. If it doesn’t get taken out “on it’s own” in a day or so, I’m going to delete it.

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Travis Johnson, Stock Gumshoe
March 10, 2014 12:22 pm
Reply to  jer_vic

I don’t know what common definition of “biotech” you want to go with, there are many, but I wouldn’t worry overmuch about weeding out small molecule pharmaceutical stocks just because they’re not developing next generation biologics … they might still be interesting early stage medical/pharma/biotech investments. I think EXEL is a monoclonal antibody company, if I remember correctly (I owned the stock a loooong time ago, haven’t followed it since), so yes, some wouldn’t call them a biotech, but those distinctions don’t matter all that much to me. Folks can also make a separate grouping of appealing “non-biotech” pharma stocks if they want to, I suppose, even just separating them by color or with a line or something.

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jer_vic
jer_vic
March 10, 2014 12:41 pm

Ok, it must have been late at night or I was drinking or something. When I was remediating the data for EXEL, all the info that came up was for some supply chain company. I must have mis-typed the ticker symbol when I looked into it. My mistake. Fixed now.

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Alan Harris
Guest
Alan Harris
March 10, 2014 1:05 pm

”Folks can also make a separate grouping of appealing “non-biotech” pharma stocks if they want to.”
Yes please Myron !!

JustIrregular
March 11, 2014 9:58 pm

Travis, Could you email me about another stock that has been sent to me on another topic. I am not sure where is would go on the site?

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Travis Johnson, Stock Gumshoe
March 11, 2014 11:28 pm
Reply to  JustIrregular

Start new discussion threads with the Discuss link at the very top of the page, or send us other info through the Contact link, also at top of every page.

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Alan Harris
Guest
Alan Harris
March 10, 2014 1:07 pm
Reply to  jer_vic

How about something less dangerous……1-5 stars for science rating?

JustIrregular
March 11, 2014 9:56 pm
Reply to  jer_vic

I did not add the “Buy” column but I did and the color green to the positive stocks we were talking about. Hope this did not create a problem!

yelpik
yelpik
March 10, 2014 1:46 pm

Cellceutix announcement release. 14 of 200 patients have started trial.
http://us2.campaign-archive1.com/?u=9ed1a6b082ae8962468007971&id=f6903ae71a&e=1c73e9207e

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Alan Harris
Guest
Alan Harris
March 10, 2014 3:28 pm

Dammit….Im gonna take a risk and issue an unconditional ‘Buy’ recco. Sue me if you want, but I recco buying an Irregulars subscription @ $49……. its guaranteed to pay back mega (in entertainment and education at least!!!) Of course where you put any other money is your problem.

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KennyG
Guest
KennyG
March 10, 2014 5:16 pm
Reply to  Alan Harris

LOL: Good one Alan.

techscan
Irregular
techscan
March 10, 2014 7:54 pm

Nice spreadsheet. Thanks y’all.

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om
om
March 10, 2014 7:58 pm

Team, any thought on ONVO – http://www.organovo.com/
I am not sure if we talked about in detail.
however, I have seen some comment on #416 http://www.stockgumshoe.com/reviews/biotech-supertrader/this-tiny-unknown-biotech-is-about-to-unleash-its-holy-grail-drug/
Is it something we can go long term?

DrKSSMDPhD
March 10, 2014 8:28 pm

om: for my taste, this company (ONVO) just seems rather pricey for what is on offer. They want you to believe that they will be a big boon to drug development because their 3-D printed “liverettes” can be used to screen for drug hepatotoxicity. They are not developing a product to help patients directly. Theirs is an excellent idea, but I have doubts it will catch fire. People are buying it on the novelty of what it is doing, but the true utility of their work is not established.

I might throw out the Israeli company BiolineRx, traded on NASDAQ for consideration. No drugs marketed but developing several of interest. Their HCV drug will be a total bust I can already tell you. Still they seem cheap compared with their first-glance prospects.

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omcdac1
omcdac1
March 10, 2014 10:10 pm
Reply to  DrKSSMDPhD

Thanks Dr. KSS. This is useful for our GANG here. Now everything is clear.
Although i have 500 Stocks. I brought them like 5 months ago.

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Mark McIntyre
Mark McIntyre
March 10, 2014 9:57 pm

FYI Egrifta Cost for those intrested

Mfr /Supplier EMD SERONO INC
800-283-8088 Mfr / Supplier Part # EM2011-2
Full FDB Description EGRIFTA 2 MG VIAL
FDB Additional Descriptor P/F, SUV, W/DILUENT
Orange Book Code (OBC) ZC Pharmacy Preferred Item? No
Color Flavor
Drug Shape Form VIAL
Route SUB-Q Strength 2 MG
Mfr Size 30.0

$2,990.88 (As of 07/30/2013) AWP for 30 vials

om
om
March 11, 2014 12:37 am

Does RNN also using RNAi technology.
Thanks Team.

Susan Graham
Guest
Susan Graham
March 11, 2014 12:52 pm

I am interested in Scancell, a UK AIM company looking at vaccines for cancer. Does anyone here have any insight?

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Les Mis
Guest
Les Mis
March 15, 2014 4:25 pm
Reply to  Susan Graham

Hi Susan
The recent post from Lexicon on the World Stocks forum pretty much sums up scancell’s platforms for cancer vaccines. Here’s the link;
http://www.worldstocks.co.uk/forum/viewtopic.php?t=7734&p=64232
Hope you find it as interesting as I did.

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Susan Graham
Member
Susan Graham
March 15, 2014 8:06 pm
Reply to  Les Mis

Thanks so much, Les Mis. I bought into Scancell after a very positive article in the newspaper last autumn, and paid 0.36-0.38. It has dropped to around 0.30 and doesn’t seem to be moving anywhere fast, despite good results. But your explanatory article has encouraged me, and at this price, it’s worth adding to. I have a lot more confidence, now that I understand better, and I can be patient, since there’s funding in place till 2016. A lot can happen in that time frame. Thanks again.

roblites
roblites
March 11, 2014 12:54 pm

BOW-WOW! …………THERF
Theratechnologies Inc. CA:TH0.00% announced today that it expects current inventory of EGRIFTA(R) (tesamorelin for injection) to be depleted in the coming weeks due to a combination of manufacturing delays and issues observed during the production of new batches of EGRIFTA(R). The depletion of the inventory will result in a shortage of EGRIFTA(R) and an eventual stock-out. EMD Serono, Inc. has notified the United States Food and Drug Administration about this product shortage. The Company also announced that it has made the decision to temporarily cease the manufacture of EGRIFTA(R). At this time, the Company is unable to determine a timeline to resume the manufacture and delivery of EGRIFTA(R) and the Company is currently investigating the causes of the issues observed. The Company will update the market when new information is available. The transaction with EMD Serono, Inc. previously announced on December 13, 2013
………..”Decrease in royalties and revenue from sales of goods are a direct result of manufacturing issues and product shortage we experienced last year. Despite those unfortunate circumstances, other financial parameters were all going in the right direction with a net loss three times lower than the previous year, use of cash twice as low and cash on hands remaining at a sustainable level. Our overriding business strategy in 2013 was to focus on EGRIFTATM in order to become cash-flow neutral as soon as possible and these results show that we made solid progress,” said Mr. Luc Tanguay, President and Chief Executive Officer.”
“By way of background, on July 17, 2013, the Court of Appeal dismissed the appeal from the judgment of the Quebec Superior Court authorizing a shareholder of Theratechnologies Inc., a public corporation listed on the Toronto Stock Exchange, to bring an action for damages under section 225.4 of the Securities Act by way of a class action. In its motion for authorization, the shareholder claimed that Theratechnologies Inc. had failed to disclose a material change as a reporting issuer and that such failure had resulted in damage”

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DrKSSMDPhD
March 11, 2014 7:20 pm

Nature Biotechnology | Editorial

Is this really the RNAissance?
Nature Biotechnology 32,201(2014)doi:10.1038/nbt.2853Published online 10 March 2014

Renewed investor interest in RNA interference (RNAi) is enabling pioneering companies to forge ahead in the clinic. Does this signify a renaissance in RNAi therapy?

Champagne corks have been popping at several RNAi drug developers in recent weeks. The New Year celebrations started when RNAi bellwether Alnylam announced it had brokered deals to acquire Merck’s entire Sirna Therapeutics portfolio for just $175 million and to sell rights to several of its rare disease assets to Sanofi/Genzyme, the latter company taking a 12% stake in the Cambridge, Massachusetts–based biotech (p. 203). In February, fellow RNAi developer Dicerna staged a staggeringly successful initial public offering, raising $90 million (p. 204). Festivities continued over at Arrowhead Research, which raised $104.2 million in a follow-on financing. This influx of investment into the sector is enabling RNAi companies to push ahead with clinical testing of lead candidates. But even though some pundits have greeted these announcements as signs of an “RNAissance,” is this really what we’re witnessing?

The Oxford English Dictionary defines the word renaissance (ren·ais·sance) as “a revival of, or renewed interest in, something.” Certainly, it has been sometime since the pharmaceutical industry showed interest in RNAi therapeutics. As boardrooms have become more concerned about the next quarter rather than the next breakthrough, Roche, Pfizer and Abbott have all exited the RNAi sector; now Alynlam’s January deal signals Merck is also out of the game. The only large companies left with serious RNAi efforts are Novartis and Sanofi.

Conversely, one might view the flux of investors into the sector as something of a turnaround—particularly with pharma backing so scarce. Part of this renewed investor enthusiasm relates to positive clinical data. In July 2012, Alnylam’s stock jumped 53% after it presented compelling phase 1 data for its short interfering (siRNA) therapy against transthyretin-mediated amyloidosis. Last August, results from a phase 2 trial showing that the same drug could knock down levels of transthyretin by 93%, together with data from a subcutaneously delivered version of the drug, again cheered investors—the market cap today is $5.7 billion (up from $1.5 billion 12 months ago).

Last year, Arrowhead announced that its hepatitis B siRNA therapy was well tolerated in a phase 1 trial and subsequently filed to begin phase 2a testing, all of which helped the company’s market cap to reach $891 million (a 30-fold rise). Currently, Benitech Biopharma, CalImmune, Gradalis, Nitto Denko, Quark Pharmaceuticals, RXi Pharmacueticals, Silence Therapeutics, Senesco Technologies, Silenseed, Sylentis and Tekmira Pharmaceuticals all have human safety testing of siRNA drugs under way.

Clinical progress could not have taken place without numerous research advances over the past decade. Serum stability has been improved through the introduction of new oligonucleotide chemistries, such as 2′-O-methyl (2′-O-Me) ribose groups, 2′ fluoro-ß-D-arabinonucleotides or unlocked nucleic acids. Potency and off-target effects have been addressed through siRNA seed element design using algorithms (e.g., GC content, asymmetric thermostability, interaction scanning with 3′ UTRs across the genome) or novel architectures (e.g., blunt 2′-O-Me RNA duplexes that reduce passenger strand loading into RISC). Similarly, increased understanding of the role of oligonucleotide length and GU-rich content in siRNA interactions with toll-like receptors 3, 7 and 8, RIG-I and PKR has reduced immunogenicity concerns.

But it is perhaps delivery where the greatest strides have been made. Refinements in Tekmira’s small nucleic acid lipid particles have allowed companies like Alnylam to achieve 100- to 1,000-fold improvements in therapeutic index. Other platforms, such as neutrally charged polyconjugates (e.g., Arrowhead’s DPC technology) or simple conjugates (e.g., Alnylam’s trivalent N-acetyl galactosamine conjugates) also show promise, particularly for delivery to hepatocytes. Last month’s formation of Voyager Therapeutics also raises the possibility that novel adeno-associated viral vectors may be combined with DNA-directed RNAi drugs to achieve the same goal. Startup Solstice Biologics is pioneering an alternative approach in which the siRNA backbone is directly modified with amidite chemistry to facilitate delivery (p. 229).

Thus, the liver, which captures nanoparticle drugs by means of endothelial fenestrations and the reticuloendothelial system, has become a dominant focus for many advanced siRNA drug programs: Alynlam is focusing on hemophilia and dyslipidemia, Arrowhead on hepatitis B, Benitec on hepatitis C and Gradalis on liver metastases.

Unfortunately, this does not mean that the path to market is clear. As for any therapeutic modality, moving from proof-of-concept trials to large-scale human testing will bring drug attrition surprises related to target biology and unanticipated off-target effects. To some extent companies are aiming to improve their odds by focusing on rare diseases with monogenetic components or developing oligos against established targets (e.g., PCSK9 or hepatitis B) already drugged by small molecules or antibodies.

A big question surrounds the long-term toxicities of lipid nanoparticles. Thus far, these have mostly been used to enhance approved chemotherapies or fungicides, and these preparations have fairly toxic side effects, sometimes requiring administration of corticosteroids or antihistamines. Stringent oversight of the potential effects of these delivery agents on humans will likely be a major focus for regulators.

But there remains reason for optimism. Sanofi’s option on Alnylam’s siRNA clinical programs indicates that the era of investing in RNAi as a technology platform is coming to an end—the era of product-specific partnerships is beginning. The shift of many companies from a focus on heroic, last-ditch cancer treatments to drugs against potentially ‘more tractable’ rare diseases also indicates maturation. RNAi drugs represent an intriguing opportunity, enabling the modulation of targets undruggable by antibodies or small molecules, all with facile manufacture and short

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analog68
analog68
March 11, 2014 7:55 pm
Reply to  DrKSSMDPhD

Yeah Doc, great article! I didn’t see you had posted it here and just posted it over on the original thread. Your little gem!

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analog68
analog68
March 11, 2014 8:06 pm

Also, Louis Breton CEO Callimune will be one of the speakers at the City of Hope’s LA Campus’s conference “Is Gene Therapy Risky Business?” Argyros Auditorium, Arnold and Mabel Beckman Center, Beckman Research Institute of City of Hope

Any SoCal gumshoer’s ? It’s this Thursday 3/13/14. Tickets are free online, you have to register to attend.

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Randy Trier
March 11, 2014 9:58 pm
Reply to  analog68

I will be there Nick. I will do my best to Channel the good Dr. with the questions he has provided me with. One of the answers has been partially been resolved with the cohort 3 participant in the Calimmune trial telling us what the delays have been due to. If anyone else in Gumshoe land is attending please let me know and we can collaborate on how to best extract the information we are all seeking!

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David B
Guest
March 11, 2014 10:07 pm

Targacept (TRGT) is one we should track. Favorable comments by Dr. KSS on it’s neuronal nicotinoid receptor (NNRs)compounds for three separate diseases. The company looks significantly undervalued for its current promise as it has been “left for dead” by quite a few after it had a failed antidepressant. It looks much more focused at this point and promising with good science. There is also a thorough recent article on the company on SA by an author I consider quite trustworthy.

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jer_vic
jer_vic
March 12, 2014 12:32 am
Reply to  David B

If by track, you mean in the spreadsheet, it’s already there.

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David B
Guest
March 12, 2014 2:45 pm
Reply to  jer_vic

Ah, I missed TRGT (Targacept) on the spread sheet as it was out of alphabetical order–glad to see it there. I’m also glad to see Xencor as this company looks very good to me as well. KSS never did a full assessment but did make some positive comments re. XNCR on the other thread.

analog68
analog68
March 11, 2014 10:07 pm

Oh, that’s right….I forgot Randy…I thought it was in NY for some reason and that’s why I mentioned LA. You’ll do the gumshoers proud.

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JustIrregular
March 11, 2014 10:37 pm

I could not find the event listed! If it is at the location that Beckman Center then that is in Duarte Calif. About 25-30 miles outside of La! I did not see it on their site!

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