ZIOPHARM Announces Orphan Drug Designation for Ad-RTS-hIL-12 in the Treatment of Malignant Glioma
BOSTON, July 24, 2015 (GLOBE NEWSWIRE) -- ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for Ad-RTS-hIL-12 + veledimex in the treatment of patients with malignant glioma. Ad-RTS-hIL-12 is a novel gene therapy candidate for the controlled expression of IL-12, a critical protein for stimulating an anti-cancer T cell immune response.
The FDA's Office of Orphan Products grants orphan drug status to support development of medicines for underserved patient populations or rare disorders affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides eligibility for a seven-year period of market exclusivity in the United States after product approval, an accelerated review process, accelerated approval where appropriate, grant funding, tax benefits and an exemption from user fees.
"Malignant glioma is an aggressive cancer with few treatment options," said Laurence J. N. Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. "This designation supports our effort to advance Ad-RTS-IL-12, a novel immuno-oncology therapy which has demonstrated promising preclinical efficacy in brain cancer, as a potential treatment for this indication. Enrollment in our Phase 1 study is progressing well, and we look forward to early results from this clinical trial later in the year."
Ad-RTS-hIL-12 + veledimex is currently being studied in a Phase 1 trial designed to examine a gene therapy treatment strategy for high grade gliomas, with the goal of generating an anti-tumor T cell immune response. The primary objective of the study is to determine the safety and tolerability of a single intra-tumoral Ad-RTS-hIL-12 injection plus escalating oral veledimex doses. Secondary objectives are to determine the veledimex maximum tolerated dose, the immune responses elicited by Ad-RTS-hIL-12 and veledimex, and investigator assessment of response, including the tumor objective response rate and progression-free survival, and determine overall survival, among other measures. The study is expected to enroll up to 72 subjects at up to 12 leading treatment centers.
About ZIOPHARM Oncology, Inc.:
ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies to deliver safe, effective and scalable cell-based therapies for the treatment of cancer. The Company's synthetic immuno-oncology programs, in collaboration with Intrexon Corporation (NYSE:XON) and the MD Anderson Cancer Center, include chimeric antigen receptor T cell (CAR-T) and other adoptive cell based approaches that use non-viral gene transfer methods for broad scalability. The Company is advancing programs in multiple stages of development together with Intrexon Corporation's RheoSwitch Therapeutic System® technology, a switch to turn on and off, and precisely modulate, gene expression in order to improve therapeutic index. The Company's pipeline includes a number of cell-based therapeutics in both clinical and preclinical testing which are focused on hematologic and solid tumor malignancies.
Sentiment: Strong Buy
Almost certainly ZIOP has shared some early Brain Cancer Data (Open Label interim) on some patients with in-operable glioblastoma with the FDA.....
Two arms to the trial - I have to believe at least a few of the inoperable brain tumor patients have shown progress....I talked to Dr Cooper Monday and Kirk in May and both were VERY excited about the Glioblastoma trials and remember Lewis comments that he expects very positive IL-12 results.....this is all coming together. Tick tick tick for this insane short interest. THIS ORPHAN DRUG STATUS FOR A DRUG WHERE WE HAVE NO PUBLISHED HUMAN DATA IS AMAZING. Cooper was also excited about additional mouse data in this indication....I would think we should see that soon.
Glioblastoma data (Orphan Status Granted) comments reasons 57-58-59 from "101 Reasons to Own ZIOP" including likely SNO data venue in November......
See Investorvillage ZIOP board for link to the FULL"101 Reasons to Own ZIOP"
57. ZIOP has initiated a highly anticipated Phase 1 Study of IL-12 Gene Therapy in Recurrent or Progressive Glioblastoma/Gr.III Malignant Glioma this year that could solve the blood-brain barrier issue other GBIM therapies have encountered.
58. XON & ZIOP executives appear unusually excited about this Glioblastoma IL-12 trial. During the March XON Q1 earnings call Kirk stated: "We are VERY excited about ZIopharm's GBM trial." Dr Cooper has stated that interim results should be available later this year...The most likely venue appears to be at the Society of NeuroOncology meeting Nov 19-22 in San Antonio (see Investorvillage ZIOP board for link to SNO meeting)
59. The fact that a large number of very prestigious institutions are conducting the Glioblastoma trial adds credence to the underlying science. Hospitals where the trials are being conducted include Stanford School of Medicine, Dana Farber Cancer Center (Harvard), Northwestern Memorial, The University of Chicago Pritzker School of Medicine and Cedars Sinai. These institutions clearly understand the value that gene therapy could bring to this very difficult and still unmet medical conditions.
All 101 reasons here: http://seekingalpha.com/instablog/6566781-options2wealth/4175505-101-reasons-to-own-ziopharm-oncology-ziop
FDA website - Ziopharm Oncology's (ZIOP) Malignant Glioma Treatment Receives FDA Orphan Drug Designation
Generic Name: Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex
Trade Name: n/a
Date Designated: 07-23-2015
Orphan Designation: Treatment of malignant glioma.
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Sponsor: Ziopharm Oncology, Inc.
One First Avenue
Navy Yard Plaza
Boston, MD 02129
The sponsor address listed is the last reported by the sponsor to OOPD.
Ziopharm Oncology's (ZIOP) Malignant Glioma Treatment Receives FDA Orphan Drug Designation
Ziopharm Oncology's (NASDAQ: ZIOP) Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex has received FDA orphan drug designation.
Adenoviral vector-RheoSwitch Therapeutic System-human interleukin 12 + veledimex is indicated as a treatment of malignant glioma.
Sentiment: Strong Buy
CNBC said Bill Miller coming up (10:45 am eastern from Delivering Alpha) & yesterday Kelly Evans said "were gonna talk some synthetic bio with Bill Miller" - His only two Syn bio stocks are ZIOP & XON...both he mentioned with Kelly previously.....
This is a HUGE win for us that Kelly Evans is moderating....REMEMBER - she is the one who asked Miller the XON/ZIOP on the video replay in Reason #22 in the "101 reasons" link copied below.......
(See investorvillage ZIOP board for link as yahoo doesn't allow them)
She said on the air "were gonna talk some synthetic bio with Bill Miller" ....he's only got 2 syn bio stocks - XON and ZIOP
10:45-11:15 a.m. TALKING STOCKS & BONDS
Moderator: Kelly Evans, Co-Anchor, "Closing Bell," CNBC
• Jeffrey Gundlach, Chief Executive Officer and Chief Investment Officer, DoubleLine
• Bill Miller, Chairman and Chief Investment Officer, LMM LLC
******And don't forget to review the "101 reasons to own ZIOP" before the interview*****
(See investorvillage ZIOP board for link as yahoo doesn't allow them)
Reasons To Own Ziopharm Oncology (ZIOP)
Many of you may remember in 2004, I wrote the "50 Reasons to Own CELG" and a later more formal improved version in September 2005 “100 Reasons to Own CELG" which I penned together with my brilliant and highly respected co-author JBWIN. Since then CELG has been one of the top performers in the market and is currently trading at 10 times the value of where it was trading when the first list came out.
I have always considered the “Reasons to Own” list to be a very special vehicle to be used only on very rare occasions – perhaps once a decade – for a stock at the right place at the right time….I decided after the MD Anderson, Merck and NCI deals the time was right for such a list for ZIOP but I needed to again find a co-author of great intelligence, integrity and with a great track record who also believed in ZIOP as much as I did….As Karim (“Options2Wealth”) describes in the article, I met him virtually after posting comments on ZIOP under his brilliant Jan blog “Ziopharm is the Stock To Watch” . He came to the opening night of my Vegas meeting and after 100+ emails, many phone calls and 3 in person meetings I realized this is one of the most brilliant investors I have ever met as well as a gentleman and all around great guy….he is the guy I have posted about that turned $10,000 into one million dollars In 6 months posting each trade on his blog before he executed. Discovering that this brilliant investor who also loved to blog about stocks and the market agreed with me on ZIOP’s potential, well I couldn’t have asked for a better co-author of this important list….here you go….
Since the list is to big for yahoo please go to Seekingalpha Options2Wealth blog its his latest article....the link is also posted on the Investorvillage ZIOP board (yahoo doesn't post links)....a must read go track it down....
Must read the new SA CRMD article countering the MANY lies in the Pumpstopper article
Go to seeking alpha and read today's CRMD article (also see discussion on InvestorVillage CRMD message board.
CorMedix: The Truth About Neutrolin, Future Standard Of Care
Not true - you made this up - even added the time stamp - good for you.
Don't see it - BS. Or a red herring and really European company...Something big is coming but I am not buying that its XON taking ZIOP over.
7. As a private company Tauropharm has vast outlets, distributors and bank accounts in the Middle East, ex Bahrain, UAE etc as well as in Asia, Africa and South America and large amounts of revenue that dwarf the amount cited in this piece likely escape their GmbH filings in Germany and require careful investigation
8. The outcome of both the patent and the unfair competition cases are far from a foregone conclusion but CorMedix believes we have evidence that clearly demonstrates that they misappropriated both critical and essential information and data from Biolink triggering German unfair competition laws as well as violate our parents which although they believe are obvious we firmly maintain are novel and believes we can make a very persuasive case they are.
Let's allow the applicable courts to decide after hearing and reading all the evidence
Hope these truth points are helpful to counteract the large volume of baseless untruths presented in this piece
Summary of counterpoints to the lies, misturths and exaggerated affiliations....
I am not dodging questions I have better things to do with my time - the "research" in this article was fed to you - it is clear as you do not even have a true grasp of QIDP....like the bash pieces on CELG, Clinical Data, ACAD, AGIO and others after runs...there is enough info to make unsophisticated investors cower....but when you did in the distortion and lies and emphasis on things like LR make it clear....After a cursory review - so many of the points made were simply untrue and absurd, my counterpoints are:
1. CorMedix has never filed for bankruptcy, there would be public filing and there isn't
2. CorMedix was never a "shell company" controlled by Lindsay Rosenwald it was an IPO with a filed S1 on record with the SEC
3. The company and its assets have had numerous documented offers when it was a private entity and its untrue that they haven't
4. Heparin is the official comparator approved in the protocol by the FDA along with strict aseptic technique in both arms There are no antibiotic catheter lock solutions approved in the U.S. and Citrate locks are not approved in the U.S. these assertions continue to be baseless that there is some novel comparator available that CorMedix is not employing in the study
5. Both gentamicin, tetracycline derivatives and other antibiotic catheter lock solutions just aren't approved nor are they recommended for use in the U.S. By any competent authority This is simply not true Some studies were done at places like Henry Ford hospital but neither FDA nor CDC has sanctioned the use of these antibiotic catheter locks Neutrolin is an anti microbial and not an antibiotic and the reason the FDA has granted Fast Track status and QIDP is for the precise reason that they want an anti microbial solution that hasn't shown any resistance in humans as gentamicin and tetracycline derivatives have Coating catheters with silver and other derivatives may help prevent catheter exit site infections but does not prevent CRBSIs or intraluminal catheter infections
6. If you look at the company's CRMD's capitalization table Lindsay Rosenwald owns less than 2% of CRMD and John Carris owns 0% and simply was a broker participating in a very small financing a long time ago This is simply misinformation Neither Lindsay nor John Carris have any representation at all on the board or in management. The fact that so much of this article was about these guys shows you what this article is. Its purpose is to distort the truth, not discuss the positives of the company like QIDP and Fast Track and FDA approved protocol and focus investors on one guy who has no involvement with company except a small passive
Talked to some Wall St heavyweights...universally positive that the JUNO deal (&more than double current stock price paid by CELG for some shares) is good for space and ZIOP...I added a lot after hours....
.... all agree this deal is great for the other gene therapy companies particularly KITE and ZIOP/XON (not so much BLUE as it appears CELG may have left BLUE behind to join JUNO).....everyone feels there at least 10 Big Pharma/Big Bios looking to get in space and very few left.
“The price of real estate just went up”…..”Scarcity Value just increased”…”Big Pharma urgency just went up a notch”…..”any deals in negotiations just got more lucrative.” “Some are short both JUNO and ZIOP – risk managers may be tapping them on the shoulder”
GREAT news for small caps imo.
Talked to former CEO Jonathan Lewis today - never heard him more bullish....he has to exercise his options within 90 days of when he left.....
he filed a form 144 that shows registration of shares for sales but he specifically told me he has no intention of selling all his shares as he is as bullish as ever - not just in CAR T and TCR but even in IL-12 and brain cancer trial. He has gone on record with an analyst last week that he is bullish even on IL-12. He intends to exercise every option - in my opinion he will hold the majority of stock - perhaps selling enough to cover the exercise cost and maybe taxes. But given his level of excitement I do not see him selling at the current $11/$12 area. Don't expect it. The filing is to get ready for the exercise which he must complete in 90 days. Do not read into it that he is selling all these shares.
I reminded him that ZIOP was my top pick in the $9 area in may and he thought that was wise and he told me he still thinks it should be my top pick here at $11 and $12. He is VERY bullish - what surprised me is how bullish he is even for IL-12.
Dr Cooper meeting with funds on a bus tour this week. I met him at JP Morgan mtg & he is brilliant...with this data and the deals.....
...I believe he will sway the long funds to get longer and the smarter short funds to cover and go long....expect run to continue not only because of technical setup which is great but because of Dr Cooper having a forum to explain why Seeping Beauty/XON/ZIOP will be the leader in the field.
See my post 20763 on ZIOP investorvillage board too big to post here....takes awhile to open as I pasted many articles. MUST READ
50% of ALL patients in Complete remission with Sleeping Beauty even BEFORE Rheo Switch and other XON/ZIOP tech added.
Med Tech Letter-ZIOP – Raising BUY LIMIT to 12 & TARGET to 18, “Spiddy Sense Tingling” - poised for a huge move based upon what might seem like random events that continue to dovetail seamlessly.
ZIOP – Raising BUY LIMIT to 12 & TARGET to 18, “Spiddy Sense Tingling”
In our view, ZIOP is poised for a huge move based upon what might seem like random events that continue to dovetail seamlessly. The recent distribution of ZIOP shares to Intrexon shareholders is yet another positive tealeaf. Intrexon’s Kirk, when asked why now, said “there are other parties for whom a Ziopharm acquisition might be very attractive in the future… our dividending these shares to our shareholders actually would simplify any such transaction.” Recently, two Intrexon executives exercised their options seven years early in order to be shareholders of date for the recent ZIOP distribution. Kirk and Intrexon are clearly signaling more good news for ZIOP. In addition, we note the major increase in short interest that has doubled since the start of the year to $300 million — about a quarter of ZIOP’s market value. In our view, a significant partnership would send the stock right though the previous high of $14 as the short squeeze would fuel the advance. It seems almost impossible that the short interest would be this large given the impressive academic and scientific validation that has been amassed since January by ZIOP.
ZIOP has had a very productive year to date with the three deals (M.D. Anderson, Merck Serono, and the NCI) and the hiring one of the world’s leading immune oncologist, Dr. Cooper as CEO. In our view, we are witnessing stellar execution of a master plan to create the leading gene therapy cancer company as all the pieces are coming together.
Dr. Cooper came from M.D. Anderson to ZIOP because he now has the potential to shepherd his cutting edge cancer treatments all the way to the market. Merck-Serono was already working with NCI on IL-12 cancer therapy and had run into toxicity problems before ZIOP
came in and stitched the program back together. The key, or actually, two keys to why ZIOP is attracting such outstanding partnerships/talent are RheoSwitch and Sleeping Beauty. RheoSwitch provides the unique ability to both titrate dosing and be an excellent safety switch. Sleeping Beauty is a non-viral delivery system that will allow repeat dosing which cannot be done with viral vectors due to safety concerns. The bottom line is that ZIOP has the best toolbox for cancer gene therapy in the business and is poised to deliver multiple value creating deals for their shareholders. Intrexon’s Kirk is a known short slayer and is the master mind behind all
the ZIOP moves. He has an excellent track record for not just slaying shorts, and most importantly creating huge value for his shareholders. As a reflection of our increased confidence in ZIOP’s cancer platform and overall execution, we are raising our BUY LIMIT and TARGET.
ZIOP is now a BUY under 12 with a TARGET PRICE of 18.
Good article - Barton was spot on with his tout of ZIOP partner XON near $18 in October and will be right again....see Investor Village ZIOP board for the Barton recommendation of XON article and this full article. Add ZIOP now.
Tremendous XON ZIOP dividend. Definitely part of a bigger deal....also now XON shorts have to deliver 1 shr of ziop for every 6 shrs XON they are short....why they short a brilliant billionaire is a head sctratcher there are so many other easier shorts
My smartest Wall St friends already correctly stating this is ZIOP dividend is part of a bigger deal....maybe increase share float and get off XON books before a sale of ZIOP or a partnership with Roche, NVS, GSK or others and they take 20% of ZIOP......who knows options ares endless but as an ZIOP and XON shareholder I LOVE LOVE LOVE this dividend....anyone who thinks this is not part of something else down the road really should not be investing. Anyone worried about silly Aftre Hours action should buy mutual funds.
RJKirk is brilliant -- remember when you are short a stock & a cash dividend is paid you must pay it - now XON shorts have to deliver a shr of ZIOP for every 6 shrs of XON they are short if they remain short....OUCH ....go ahead short Kirk - you will pay as you have in past....as for ZIOP more news is clearly coming. Just be patient. Less
LOVE XON's ZIOP dividend. Obviously part of a bigger plan/deal....also now XON shorts must deliver 1 shr of ziop for every 6 shrs XON they are short....why they short a brilliant billionaire is beyond me....
My smartest wall street friends already have contacted me stating this is ZIOP dividend is part of a bigger deal....maybe increase float and get off XON books before a sale of ZIOP or a partnership with Roche, NVS, GSK or others and they take 20% of ZIOP......who knows the possiblilities or endless but as an XON and ZIOP shareholder I LOVE LOVE LOVE this dividend....anyone who thinks this is not part of something else down the road really should not be investing. Anyone worried about silly AH action should buy mutual funds.
Kirk is brilliant -- remember when you are short a stock and a cash dividend is paid you must pay it - now XON shorts have to deliver a share of ZIOP for each 6 shrs of XON they are short if they remain short.....go ahead short Kirk - you will pay as you have in the past....as for ZIOP more is coming. Just be patient.
Some of my bullets from my ZIOP slides from Vegas top ideas presentation...#1 pick
Told everyone to buy ZIOP Thursday, Friday, Saturday and Sunday....8's and 9's . Sorry the formatting and bullets under bullets have not pasted properly - but I think the points are still worth posting
Ziopharm Oncology (ZIOP)
o Owns the rights to every human cancer drug Intrexon (XON) will ever make and 50% of the profits.
o Has deals with:
--MD Anderson – the largest cancer clinic in the world
--Merck Serono – big pharma
--NCI – most important government agency in cancer with Dr. Steven Rosenberg (godfather of CAR-T)
o Two possible paths forward:
• Series of deals by indication, more switch licensing
• Couple of more deals than takeout by big pharma like NVS, Roche, BMY, Merck, AZN, GSK
• Been in Europe many times past few months said publicly more international and large bio/pharma companies have show interest than ever before
• Breast cancer trial just announced – targeted patient population
• Glioblastoma – IL-12 works in brain/CNS. Reduces cancers ability to down regulate immune system by a factor of 20. R.J. Kirk is VERY excited. IL-12 ramps up production of dendritic cells.
• Rheo-Switch/ZIOP approach – inert until turned on vs competitors always on (kill switch)
• Everyone needs the Switch to boost expression of the target enzyme and to control off-target toxicity
• NCI/FDA will be slow to approve “always on” gene therapy
• TCR molecule found on surface of T-lymphocytes (T-cells). It is responsible for recognizing antigens that are bound to Major Histocompatability Complex (MHC).
• Antigen expressed on cell. XON builds receptors on T-cell that will lock onto cancer antigen.
• TCR – not as strong a bind – doesn’t last as long. 50% efficacy.
• CAR-T difference – CAR receptor stronger bind, which is why they are powerful. Going after marker CD-19. 90% efficacy.
• Big Pharma is not far ahead with TCR – little history
• R.J. Kirk thinks they have unique TCR:
• Multigenic attacks with a Rheo-Switch. They go after multiple receptors and boost expression/efficacy (Rheo-Switch)
• Cost cheaper with Sleeping Beauty
• Approach increase T-cell production/affinity/response (also IL-12)
• Earlier projects - NK cells, CLMs, exosomes, artificial antigen receptors expressed on outside or inside on cancer tumor/cells itself. Still early but XON has expressed excitement.