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did he mean to say 38 percent
if gleeve did 28 percent, if you add 15 percent Iclusig would only need to do 43 percent
If Gleevec did 28%, you probably would need to break 50%, although it's never done that. But certainly, if it were 50% or greater, you could feel confident that it's an unequivocal, great outcome. And yes, we do have to win the trial.
An Experimental Drug’s Bitter End
http://www.nytimes.com/2013/06/07/business/an-experimental-drugs-bitter-end.html?ref=health&_r=1&
By ANDREW POLLACK
Holly Usrey-Roos will never forget when her son, Parker, then 10, accidentally broke a drinking glass and said, “I’m sorry, Mom. I love you.”
It was the first time she had ever heard her son say he loved her — or say much of anything for that matter. Parker, now 14, has fragile X syndrome, which causes intellectual disability and autistic behavior.
Ms. Usrey-Roos is certain that Parker’s new verbal ability resulted from an experimental drug he was taking in a clinical trial, and has continued to take for three years since then. She said she no longer had to wear sweaters to cover up the bruises on her arms she used to get from Parker hitting or biting her.
Now, however, the drug is being taken away. It has not met the goals set for it in clinical trials testing it as a treatment for either autism or fragile X syndrome. And Seaside Therapeutics, the company developing it, is running out of money and says it can no longer afford to supply the drug to former participants in its trials.
The setback is a blow in the effort to treat autism since the drug, arbaclofen, was one of the furthest along in clinical trials. And the company’s decision has caused both heartbreak and outrage among some parents.
“I waited 10 and a half years for him to tell me he loved me,” said Ms. Usrey-Roos, who lives in Canton, Ill. “With fragile X, you’re like living in a box and someone is holding the lid down. The medication opened the lid and let Parker out.”
“I don’t want to go back to the way life was,” she added.
The situation raises questions about what, if anything, drug companies owe to patients participating in their clinical trials. It also points out the difficulties in developing drugs to treat autism and fragile X syndrome. If the drug worked so well in some patients, why has it not succeeded so far in clinical trials?
One reason is that the symptoms and behaviors associated with autism and fragile X vary widely among individuals, making it hard to capture the effects of a drug by looking at any one measure, like irritability or social withdrawal. Seaside and doctors who participated in the trials said that there were improvements in some aspects of behavior in some studies, just not those considered critical to a trial’s overall success.
But could it also be that the parents are deluding themselves into seeing changes that are not there? Could improvements be the result of the children simply growing older?
“It’s kind of hard to make the argument that the company should keep providing it if it’s not working,” said Dr. Michael R. Tranfaglia, medical director of the Fraxa Research Foundation, of Newburyport, Mass., which provides money for research on fragile X syndrome.
Dr. Tranfaglia, whose son has fragile X but was not in a Seaside trial, said arbaclofen appeared to significantly help about a third of patients. It also made some patients worse. Without being able to tell in advance which patients would benefit, it would be hard for the drug to succeed in a clinical trial and win approval, he said.
Similar situations have risen occasionally in the past. In 2004, patients with Parkinson’s disease protested when Amgen stopped providing an experimental drug that some patients said had restored their lives. Amgen said the drug had failed in a clinical trial and might even be dangerous.
Two patients even sued, but a court ruled the company had no obligation to continue to supply the drug to participants in its trials.
In the case of arbaclofen, parents are appealing to Congress and have started an online petition hoping to find financing for the drug’s development. They are also organizing through social media.
Seaside executives declined to be interviewed.
Until recently, Seaside, one of the few companies pursuing autism drugs, was considered a shining light by family members of those with the condition.
The company, in Cambridge, Mass., grew out of the research of Mark F. Bear, a neuroscience professor at the M.I.T. Its co-founder and chief executive, Dr. Randall L. Carpenter, has a sister with an intellectual disability.
As of a year ago, privately held Seaside had raised about $90 million. Most of it had come from the Barony Trust, run by the family of Peter Whipp, a wealthy Briton. But it apparently never was the plan for Mr. Whipp to carry the company indefinitely.
Last year, Seaside entered a partnership with Roche, the Swiss pharmaceutical giant, which provided an undisclosed amount of money in exchange for intellectual property rights covering a different class of autism drug and an option to license arbaclofen.
But given the failures in the clinical trials, Roche says it has decided not to license arbaclofen, apparently ending financial support for studies of the drug.
“We concluded that arbaclofen wasn’t going to provide that real difference for patients,” Luca Santarelli, head of neuroscience research at Roche, said in an interview.
Dr. Bear of M.I.T. said he was hopeful new financing could be found. He said the company was started to help families, “not a play to make money.”
He added, “I think the signs are sufficiently encouraging. It would be really tragic if we abandon this now.”
On May 1, Seaside announced that arbaclofen, which is also known as STX209, had not met the main goal of reducing social withdrawal in a 150-patient midstage study of children and young adults with autism. But the drug did succeed on a different measure — the clinicians’ assessment of the patients.
The drug had previously failed in a Phase 2 trial for fragile X, which is caused by a mutation in a gene on the X chromosome and affects about 100,000 Americans.
In that trial, the main goal was to reduce irritability. While it did not do that, it appeared to ease social withdrawal. So Seaside began two Phase 3 trials for fragile X, one for young children and one for adolescents and adults. This time, social withdrawal was the main measure.
But the company said recently in a letter to parents that the trial in adolescents and adults had not succeeded. Results from the trial of children should be known this summer.
In the middle of May, Seaside told doctors and patients that because of “resource limitations” it could no longer supply the drug. “We know that this termination will be disruptive and disappointing for many families,” executives said in a letter to parents.
That is putting it mildly. Christina Murphy of Holton, Kan., wrote on the Web site stx209stories set up by families wanting the drug back, that her 11-year-old son Rhett, who has fragile X, made friends for the first time ever once he started taking the drug and sang in the school talent show, overcoming his usual fear of crowds and getting a standing ovation.
“I had never in my life witnessed something like that,” she wrote. “There was hardly a dry eye among the adults in the room. I have never seen him so happy in his entire life as when he finished that song and looked out at the crowd.”
Only a few hours later, she learned that the drug would be taken away. “I am sad, I am hurt and my heart is breaking,” she wrote.
Parents and patient advocates say there could be 300 patients or more taking the drugs. Some say they were promised the drug would be available until it reached the market or was totally abandoned as ineffective.
“When I found out, I went upstairs to my husband. I was crying so hard he thought someone had died,” said Cortney AbouElSeoud of Holt, Mich.
Ms. AbouElSeoud said her 5-year-old son Ayden, who has fragile X, spoke only a few words before entering the trial last October. Now he is up to 50 words. “He talks to us and answers us,” she said. “It’s crazy and awesome to see him emerging into a little person.”
Some parents say that they feel betrayed by Seaside and that the company has not communicated enough.
“I believe I, along with many others, are entitled to a better explanation of why our lives are being turned upside down,” Lori Armer of Newport News, Va., wrote on a Facebook page for families weaning their children off arbaclofen. She and other parents wrote on the site that behavioral problems were returning as the drug dosage was reduced.
There are some options. Arbaclofen is a derivative of baclofen, a generic muscle relaxer that is already on the market as a treatment for spasticity. While there is some evidence that arbaclofen is more potent, some parents are turning to baclofen.
Others hope to enroll their children into trials of other drugs for fragile X and autism being run by Roche and Novartis.
Ms. Usrey-Roos, who works for the National Fragile X Foundation, based in Walnut Creek, Calif., said she was hopeful that if one drug worked, another might, too.
While sad that the study ended, she said, “I’m still thankful for this experience. It has given me such hope.”
Ariad Pharmaceuticals
(ARIA-NASDAQ)
June 3, 2013
Biotechnology
Jim Birchenough, M.D.
BMO Capital Markets Corp.
415-591-2129
jim.birchenough@bmo.com
Chuck Whitesell / Nick Abbott, PhD.
Positive Feedback From Management/Investigator Dinner
Event
We hosted a dinner last night at the annual ASCO meeting with Ariad Pharmaceuticals and prominent lung cancer expert Dr. Ross Camidge. With primary focus on ALK-EGF inhibitor AP26113, drug activity in ALK+, XALKORI resistant NSCLC was described as differentiated in terms of brain met response, with a path to accelerated approval available through a pivotal single arm phase 2 initiating in 3Q13. With greater focus on EGFR+, Tarceva resistant patients with T790M secondary mutation, trough AP26113 concentrations were described as well above the IC50 of 282nM - 694nM, and above an extrapolated IC90, although measures of IC90 are challenging. Feedback from AP26113 principal investigator Dr. Camidge confirm the difficulty in assessing response in T790M patients in phase 1 and suggested that enrolling appropriate T790M patients rapidly in phase 2 should not be difficult in support of more meaningful clinical data at ESMO in September. Additional detail in ICLUSIG suggests confidence in launch, CV profile, EPIC progress and EU opportunity.
Impact
We are reiterating our Outperform rating on shares of ARIA following a positive update on both AP26113 and ICLUSIG at ASCO. With perhaps greater focus on AP26113, ARIA exceeded expectations of differentiated brain met response in ALK+ patients, and established a strong rationale for effective dosing in EGFR+, T790M patients. Based on the update, we see a clear path to accelerated approval and market dominance in ALK+ patients and expect competitive anti-tumor responses in T790M patients at ESMO. We would be buyers of ARIA shares here.
Kynamro vs. Juxtapid
which label is scarier
http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/203858s000lbl.pdf
http://www.kynamro.com/families.aspx?utm_source=Google&utm_medium=PPC&utm_campaign=Google-PPC&utm_term=kynamro&gclid=CKazyqjivLcCFcmf4AodKFIAXQ
I think I would prefer kynamro
I think Juxtapid is ramping better because it is oral but I think people may switch after a while.
unbelieveble that 80 million dollars a year is considered cheap
Ohr Pharmaceutical Appoints June S. Almenoff to Board of Directors
Press Release: Ohr Pharmaceutical Inc. – 24 minutes ago
NEW YORK, May 22, 2013 /PRNewswire/ -- Ohr Pharmaceutical Inc. (OHRP), a pharmaceutical company focused on the development of novel therapeutics for unmet medical needs, today announced the appointment of June S. Almenoff, M.D., Ph.D., to its Board of Directors. Dr. Almenoff has extensive experience in the pharmaceutical industry, working on early and late stage drug development.
"We are delighted to have Dr. Almenoff join Ohr's Board," said Dr. Irach Taraporewala, CEO of Ohr Pharmaceutical. "She offers tremendous expertise in the areas of pharmaceutical research and development, risk management, and product licensing. We look forward to working with June as we continue to achieve significant corporate milestones including the halfway point in enrollment of the Squalamine eye drop phase II clinical trial and our proposed listing on NASDAQ, both expected by midyear."
"I am excited to be joining the board of Ohr," said Dr. June Almenoff. "Ohr has a promising pipeline and excellent team, and I look forward to working with the Board to help drive the company toward future success."
June S. Almenoff, M.D., Ph.D. is President and Chief Medical Officer of Furiex Pharmaceuticals, a pharmaceutical development and collaboration company (FURX:Nasdaq); she serves as the company's Principal Executive Officer and a member of the Board of Directors. Since its inception in 2010, Furiex has completed two successful proof-of-concept programs, one of which has advanced to pivotal Phase III trials. Prior to joining Furiex, Dr. Almenoff had over 12 years of pharmaceutical industry experience at GlaxoSmithKline. During her tenure at GSK, she was a Vice President in the Clinical Safety organization at GSK, where she chaired a Pharma-FDA working group, and she also worked in the area of scientific licensing. While at GSK, Dr. Almenoff led teams that developed several systems for minimizing risk in early- and late-stage drug development, which were recognized by the Wall Street Journal Technology Innovation Award and several other prestigious awards. Prior to joining GSK, Dr. Almenoff was on the faculty of Duke University Medical Center, where she is currently a Consulting Professor of Medicine.
Dr. Almenoff received her B.A. cum laude from Smith College. She graduated from the M.D.-Ph.D. program at the Mt. Sinai School of Medicine and completed a residency in Internal Medicine and a Fellowship in Infectious Diseases at Stanford University Medical Center. Dr. Almenoff is a board-certified Fellow of the American College of Physicians with 10 years of clinical practice experience, and she has authored 50 publications.
About Ohr Pharmaceutical Inc.
Ohr Pharmaceutical Inc. (OHRP) is a pharmaceutical company dedicated to the clinical development of new drugs for underserved therapeutic needs in large and growing markets. The Company is focused on advancing its pipeline products currently in phase II clinical development: Squalamine Eye Drops for the treatment of the wet form of age-related macular degeneration, and OHR/AVR118 for the treatment of cancer cachexia. Additional information on the company can be found at www.ohrpharmaceutical.com.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made only as the date thereof, and Ohr Pharmaceutical undertakes no obligation to update or revise the forward-looking statement whether as a result of new information, future events or otherwise. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the future success of our scientific studies, our ability to successfully develop products, rapid technological change in our markets, changes in demand for our future products, legislative, regulatory and competitive developments, the financial resources available to us, and general economic conditions. Shareholders and prospective investors are cautioned that no assurance of the efficacy of pharmaceutical products can be claimed or assured until final testing, and no assurance or warranty can be made that the FDA or Health Canada will approve final testing or marketing of any pharmaceutical product. Ohr's most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We disclaim any intent to revise or update publicly any forward-looking statements for any reason.
Contact:
Ohr Pharmaceutical Inc.
Investor Relations:
Tel: (877) 215-4813
Email: ir@ohrpharmaceutical.com
Esperion acquired other drugs that were in the old esperion pipeline.
the drug that was very difficult to make was apo milano and I believe that pfizer licensed that to the medicines company.
John Varian, the CEO of XOMA was the CFO or ARYX. You weren't a fan of that company or management.
..Ohr Pharmaceutical Appoints Thomas M. Riedhammer To Board Of Directors
......
NEW YORK, May 2, 2013 /PRNewswire/ -- Ohr Pharmaceutical Inc. (OHRP), a pharmaceutical company focused on the development of novel therapeutics for unmet medical needs, today announced the appointment of Thomas M. Riedhammer, Ph.D., to its Board of Directors. Dr. Riedhammer has over 35 years of ophthalmic industry experience, working for large and small pharmaceutical companies engaged in drug development through commercialization.
"We are pleased to have brought on someone of Dr. Riedhammer's caliber," stated Dr. Irach Taraporewala, CEO of Ohr. "Tom's proven expertise in building successful companies in the ophthalmic pharmaceutical industry will be an immeasurable asset to Ohr at this important juncture in the company's growth."
"I am excited to join the team at Ohr Pharmaceutical," commented Dr. Riedhammer. "Squalamine eye drops are a unique and potentially transformative product for wet-AMD patients, a large patient population currently being treated with chronic intravitreal injections directly into the eye. I look forward to contributing to the future growth of Ohr and providing benefit for patients in need."
Ira Greenstein, Chairman of the Board of Directors, added, "With our recent financing providing us with sufficient capital until 2015, anticipated enrollment milestones and a potential listing on Nasdaq on the horizon, it is a great time to welcome Dr. Riedhammer, with his wealth of experience in the development of ophthalmic companies, onto the team at Ohr."
Dr. Riedhammer has over 35 years of ophthalmic industry expertise. Most recently, Dr. Riedhammer was Chairman of Sirion Therapeutics, where he was instrumental in raising over $100 million dollars of private equity and the subsequent FDA approval and launch of two ophthalmic products in the U.S. Prior to Sirion, Dr. Riedhammer served as the Chief Operating Officer of Presby Corp., a medical device company engaged in the research and development of treatments for eye disorders, taking the company through clinical trials and a successful sale to CibaVision. Prior to Presby Corp, Dr. Riedhammer served as a senior corporate Vice President, member of the management executive committee, Chief Technical Officer and President of Global Pharmaceuticals for Bausch & Lomb, where he was involved with numerous acquisitions and licensing of ophthalmic products for the U.S. and European markets, and played an important role in the successful growth of Bausch and Lomb's U.S. pharmaceutical business. Dr. Riedhammer began his career at Bausch & Lomb as a research chemist where he was awarded 12 U.S. patents and was responsible for the development of the Renu and Sensitive Eyes product lines. He holds a B.A. in Chemistry and a Ph.D. in Electrochemistry from State University of New York at Buffalo.About Ohr Pharmaceutical Inc.
Ohr Pharmaceutical Inc. (OHRP) is a pharmaceutical company dedicated to the clinical development of new drugs for underserved therapeutic needs in large and growing markets. The Company is focused on advancing its pipeline products currently in phase II clinical development: Squalamine Eye Drops for the treatment of the wet form of age-related macular degeneration, and OHR/AVR118 for the treatment of cancer cachexia. Additional information on the company can be found at www.ohrpharmaceutical.com.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made only as the date thereof, and Ohr Pharmaceutical undertakes no obligation to update or revise the forward-looking statement whether as a result of new information, future events or otherwise. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the future success of our scientific studies, our ability to successfully develop products, rapid technological change in our markets, changes in demand for our future products, legislative, regulatory and competitive developments, the financial resources available to us, and general economic conditions. Shareholders and prospective investors are cautioned that no assurance of the efficacy of pharmaceutical products can be claimed or assured until final testing, and no assurance or warranty can be made that the FDA or Health Canada will approve final testing or marketing of any pharmaceutical product. Ohr's most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We disclaim any intent to revise or update publicly any forward-looking statements for any reason.
Contact:
Ohr Pharmaceutical Inc.
Investor Relations:
Tel: (877) 215-4813
Email: ir@ohrpharmaceutical.com
..
The Beginning Of A Shift At Tekmira: From Focus On Delivery, To Therapy
Apr 23 2013, 11:53 | 1 commentby: PropThink | about: TKMR, includes: ALNY Disclosure: I have no positions in any stocks mentioned, but may initiate a long position in TKMR over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it. I have no business relationship with any company whose stock is mentioned in this article. (More...)
By Jake King
Tekmira's (TKMR) story over the last few years has largely been focused on its siRNA delivery technology, lipid nanoparticles (LNPs), and the ability of the platform to be used in broader RNAi applications. But the focus of investors is shifting as Tekmira brings a new therapy into the clinic this year and moves out of early-stage clinical development and into proof-of-concept studies. With that should come greater visibility as investors pick up on the therapeutic development facet of the Tekmira story, and the capital markets should begin to allot more value to the company's in-house pipeline than they currently do.
Tekmira has an enterprise value (EV) of just $20M, markedly low for a company with multiple therapies in human clinical trials, a strong balance sheet, and robust economic agreements in place with larger drug companies. Tekmira simply isn't getting credit for its pipeline, and as the arguable leader in RNAi delivery technology, Tekmira's valuation fails to capture the full potential of its platform and its own LNP products. With soon-to-be six clinical-stage pipeline assets, several catalysts this year for both proprietary and partnered product candidates, and an EV of around $20M, TKMR is remarkably cheap to own. So much to offer and so inexpensive makes TKMR attractive to investors based on the company's long-term prospects, however, drug companies may also be interested in scooping up this "on-sale" platform and pipeline. We believe TKMR will not stay this cheap for long.
Mini version of Alnylam that trades near cash. Alnylam (ALNY) is Tekmira's bigger, well-known cousin in RNAi therapeutics, a drug developer valued at more than $1.4B by the markets, despite that the company's pipeline is still in early- and mid-stage development. Alnylam has done a nice job of focusing the investment community on its "5 x 15" strategy, whereby it expects to have five candidates in late-stage development by 2015, that much closer to a fully validated (FDA approved) RNAi therapeutic. But more importantly, three of Alnylam's leading candidates -- ALN-TTR02, ALN-PSC, and ALN-VSP -- utilize Tekmira's lipid nanoparticle (LNP)-enabling technology, and the smaller company collects royalties and milestones on these treatment opportunities -- by owning TKMR, investors can participate in the ALNY story, while still owning one of the leading, yet undervalued, RNAi delivery developers.
Financially, Tekmira is solid, an important component to owning a development-stage biotech. The company came into a large, non-dilutive source of capital last year when it and Alnylam settled long-standing litigation regarding Tekmira's LNP technology. That settlement netted Tekmira roughly $46.3M, enough capital, the company says, to continue funding operations into 2015. Tekmira ended 2012 with $46.8M in cash & equivalents and guides for a balance of $35M at the end of 2013: a cash burn of just over $11M after factoring in expected revenues and milestone payments from partnerships in 2013. On a per share basis, TKMR is worth almost $3.27 (based on end of 2012 figures) and closed the day on Monday at $4.70; essentially, investors can own Tekmira's entire pipeline for ~$1.50 per share. Even if TKMR remains flat through 2013, which we don't expect, the company expects to end the year with $2.44 in cash per share, still a robust backdrop to its current share price.
Overall, the company's EV of ~$20M (market capitalization of $65M) is remarkably low for five assets that have progressed into human trials, some of which are advancing into mid-stage trials under partner, Alnylam. The two companies are closely tied in terms of success and failure, and positive read-outs at Alnylam should be read as successes for Tekmira, given associated milestone payments, further validation of Tekmira's LNP-technology, and the long-term financial benefits of potential commercialization.
Tekmira has economics on several key assets from Alnylam, some featuring 2013 catalysts and cash milestones. As mentioned above, Tekmira has a financial stake in several of Alnylam's clinical products that utilize Tekmira's LNP delivery technology. Most notably, TKMR has a royalty and milestone agreement with ALNY for TTR02, Alnylam's most advanced clinical product (details below). Alnylam expects Phase II data for TTR02 by mid-2013 and will then advance the therapeutic (assuming positive results) into a Phase III trial, resulting in a $5M milestone payment to Tekmira. Given the strong results seen in Phase I, it's widely expected that the data will be positive and that ALNY will commence a Phase III study in 2H13. Tekmira is entitled to royalties in the single digits on TTR02 sales. Tekmira is also entitled to receive a $5M milestone payment when a Phase II trial for ALN-VSP begins in China; that's expected in the second half of this year, and both the TTR-02 and VSP milestone payments ($5M each) have been factored into Tekmira's 2013 guidance. In addition, Tekmira collects royalties (low single digits) on both VSP and PCS, the latter of which was partnered with The Medicines Company (MDCO) earlier this year, a validation of both the product and the technology behind the product. Seeing a mid-tier drug developer step into siRNA for a large indication like hypercholesterolemia is suggestive of broader industry acceptance of RNAi as a therapeutic pathway.
It's also important to note the unique economics of the TKM-Ebola program. The therapy is being developed cost-free for Tekmira, as the U.S. Department of Defense (DOD) pays for up to $34.7M of development costs through Phase I trials, although the contract could be extended to $140M for TKM-Ebola to achieve FDA approval. Tekmira is preparing for a Phase I trial to begin this year with a newer formulation of the Ebola therapy, and the therapy, if successful, would be approved under the FDA's "Animal Rule" (no requirement to treat humans inflicted with Ebola, only human safety must be proven).
Finally, and important to Tekmira's long-term story, Tekmira has broad access to Alnylam's siRNA payload technology. TKMR can access 13 targets from ALNY and has currently used 8, leaving five targets that TKMR can still tackle, combining ALNY's core technology with its LNP delivery platform. These targets can be developed internally or out-licensed, and Tekmira's ability to access ALNY's payload technology negates any immediate need for an in-house discovery program, allowing for a strong focus on LNPs while still building an internal pipeline. The company will, of course, pay low royalties to Alnylam if any of these assets are commercialized.
More than just RNAi delivery. Tekmira owns the most clinically researched and arguably most advanced delivery technology in RNAi therapeutics -- lipid nanoparticles (formerly called stable nucleic acid-lipid particles, or SNALP). Delivery is key in RNAi. Unlike small molecule drugs, RNA molecules in the bloodstream tend to be unstable and are unable to access cells to carry out their gene-silencing capabilities. Preclinical work has demonstrated that LNP technology, which essentially "encapsulates" short interfering RNA (siRNA) in a deliverable package, is overcoming these limitations. Already in Tekmira's preclinical and early clinical studies, LNP has shown an ability to "knock down" (reduce) target gene expression. Importantly, after a recent litigation settlement with Alnylam, all intellectual property for LNP delivery now resides with Tekmira, strengthening the developer's position as a delivery enabler and creating the sole LNP-enabled partner in the world of RNAi development.
But while technology delivery is certainly a compelling angle to Tekmira's story, we believe that the market has neglected to build in value for Tekmira's in-house pipeline; the company is shifting its positioning from one of delivery technology alone to also more strongly encompass therapeutic development, and should be valued as such. In 2013, Tekmira will identify a third in-house asset to move into the clinic, in addition to the three assets already being developed through its partnership with Alnylam. The Alnylam partnership, while accretive to Tekmira, may be more important as validation of the LNP platform than as key long-term value drivers. Nevertheless, with an EV of just $20M, each pipeline asset has the ability to significantly increase the value of TKMR shares. Below, we describe the first two candidates in the company's pipeline that are being developed in-house, followed by Alnylam's products partnered with Tekmira.
TKM-PLK1 - is Tekmira's proprietary PLK1 (polo-like kinase 1) inhibitor, which is moving into a Phase II trial for the treatment of gastrointestinal carcinoid tumors later this year. TKM-PLK1 created some stir at the American Association for Cancer Research meeting earlier this month with promising results from a Phase I 24-patient dose-finding study in a variety of cancers. The Phase II trial will begin in 2H13.
TKM-Ebola - is an RNAi therapeutic utilizing Tekmira's LNP technology to protect patients from the Ebola virus. Pre-clinical data were published in the Lancet in 2010, and the therapy should be moving into a Phase I safety study in healthy patients yet this year.
ALN-TTR02 - Alnylam's treatment for transthyretin-mediated amyloidosis (ATTR), ALN-TTR02 also uses Tekmira's LNP-delivery technology. A Phase I trial demonstrated that TTR02 is well-tolerated and led to a knockdown of serum TTR protein levels of up to 94% (p<0.00001). Following a single dose of TTR02, the reduction was rapid, dose dependent, and durable; and suppression was sustained almost entirely (80%) at one month after a single dose. Alnylam will have Phase II data by the middle of 2013 and plans to move TTR02 into a pivotal trial after.
ALV-PCS - Alnylam develops ALV-PCS as a treatment for hypercholesterolemia. It targets the PCSK9 gene, resulting in higher LDL receptor levels in the liver and subsequently lower LDL cholesterol in the bloodstream. A Phase I trial wrapped up early in 2012, and we're waiting for details on a development path forward, which we assume will include a Phase II trial beginning in 2013.
ALN-VSP - is a kinesin spindle protein (KSP) and vascular endothelial growth factor (VEGF) inhibitor indicated for advanced solid cancers with liver involvement. Alnylam partnered the product with Ascletis Pharmaceuticals for development in parts of Asia, and a Phase II Chinese trial should begin in 2013.
Business activity is robust and certainly worth more than the current EV. Tekmira is entitled to single-digit royalties on net sales of the recently approved chemotherapeutic Marqibo (vinCRIStine sulfate LIPOSOME injection). The product, owned by Talon Therapeutics (TLON), was approved in 2H12 for 2nd-line Philadelphia chromosome negative (Ph-) acute lymphoblastic leukemia (ALL). Tekmira receives mid-single digit royalties on Marqibo sales (estimates for that drug are ~$100M at peak), so Marqibo alone could be worth around $5M annually to Tekmira. Also in oncology, Tekmira is moving its TKM-PLK1 into a Phase II trial in gastrointestinal carcinoid tumors, a relatively small indication but one with limited treatment options; the U.S. incidence of GIC tumors is approximately 10-15,000 annually. Considering this advancement, TKM-PLK1 has arguably taken the title of lead RNAi oncology candidate in clinical development.
U.S. DoD development contracts for Ebola treatments were originally given to both Sarepta (SRPT) and Tekmira, but in September 2012, the DoD moved forward with Tekmira's program alone, indicating that Tekmira now has the leading and only RNAi-based Ebola program in the U.S. The agency has demonstrated not only a willingness to test the RNAi possibility, but to back the program in full, an unexpected substantiation of RNAi from the U.S. government. As alluded to earlier, Tekmira and Alnylam settled a lawsuit in November of last year in which Tekmira had sued Alnylam for patent infringement regarding its LNP technology. The original lawsuit was for $1B, and it seemed Tekmira was willing to go down fighting (capital dipped dangerously low before the settlement). The settlement, however, has been largely favorable to Tekmira, which retains a financial stake in Alnylam's clinical products (small milestones and royalties) and has access to 13 of Alnylam's product licenses for in- or out-licensing. While not the billion dollar win that the company hoped for, Tekmira shored up its balance sheet and kept relations in order with its largest partner.
A very attractive valuation; one that either investors or pharma are likely to appreciate. Tekmira has collaborations and/or licenses in place with Alnylam, Merck (MRK), and Bristol-Myers Squibb (BMY). The company hasn't announced a new collaboration in over two years, we believe due to the 20 months spent on litigation with Alnylam. With that litigation out of the way and the patent situation regarding LNP clearly defined, we expect that Tekmira will once again become a sought after partner for companies looking for RNAi delivery.
We see three key reasons to be involved in Tekmira: 1) Cash per share of more than $3.00 and an EV of ~$20M means very limited downside; 2) Upcoming catalysts in Phase II data for ALN-TTR02, and trial initiations for VSP, PCS, Ebola, and PLK1; 3) A new pathway announcement this year that could pique the interests of investors, particularly if TKMR goes after an orphan indication; and 4) Possibility for partnerships with additional major drug companies now that the LNP litigation is out of the way. We expect that TKMR's ~$20M EV will not last as investors focus on the value and potential value that this company has to offer. Alternatively, if the stock remains this cheap, perhaps a strategic investor will go after TKMR. For example, ALNY itself could simply acquire TKMR and never have to pay royalties or milestones to the company again, in addition to keeping its payload technology all to itself. TKMR has put in a strong support base around $4.20 (200D MA at $4.22), and we're looking for a breakout back above $5.00 in the near term as ALNY's Phase II TTR02 top-line readout approaches.
Additional disclosure: PropThink is a team of editors, analysts, and writers. This article was written by Jake King. We did not receive compensation for this article, and we have no business relationship with any company whose stock is mentioned in this article. Use of PropThink’s research is at your own risk. You should do your own research and due diligence before making any investment decision with respect to securities covered herein. You should assume that as of the publication date of any report or letter, PropThink, LLC and persons or entities with whom it has relationships (collectively referred to as "PropThink") has a position in all stocks (and/or options of the stock) covered herein that is consistent with the position set forth in our research report. Following publication of any report or letter, PropThink intends to continue transacting in the securities covered herein, and we may be long, short, or neutral at any time hereafter regardless of our initial recommendation. To the best of our knowledge and belief, all information contained herein is accurate and reliable, and has been obtained from public sources we believe to be accurate and reliable, and not from company insiders or persons who have a relationship with company insiders. Our full disclaimer is available at www.propthink.com/disclaimer.
they already had a test approved and it didn't sell. It was a larger sample
the good thing about colonoscopy is that it the doctor can remove polyps before they become cancerous. If this can't detect the polyp you are letting something grow and potentially become cancerous. If it detects the polyp you will need to get it removed and undergo the procedure anyway.
Exact Sciences cancer test meets goal, but disappoints investors
7:50am EDT
* Pre-cancer detection rate of 42 pct below market expectations
I always thought this was a crappy test
* Shares fall 28 pct premarket
April 18 (Reuters) - Exact Sciences Corp said its molecular screening test for colorectal cancer met a late-stage trial goal of detecting the disease in more patients than other commonly used tests, but shares fell 28 percent as the results were not as robust as some were expecting.
Exact's test, which identifies abnormal DNA in cells shed in a patient's stool, detected 92 percent of colorectal cancers and 42 percent of pre-cancerous polyps in a large late-stage study.
"Pre-cancerous sensitivity, which was the key metric investors were looking at, was well below expectations," Wedbush Securities analyst Zarak Khurshid said, adding that "lower pre-cancer sensitivity may limit the eventual addressable opportunity for the test."
Khurshid was expecting a 55 percent detection rate for pre-cancerous polyps.
Exact's non-invasive test was developed to be a more accurate alternative to currently available non-invasive screenings.
The study compared the performance of its Cologuard test to colonoscopy and fecal immunochemical testing that looks for blood in a stool sample.
While a colonoscopy is considered the most accurate method of detecting colon cancer and polyps, many people avoid the recommended test as it involves inserting a flexible tube into the colon.
The trial was conducted on 10,000 patients between the ages of 50 and 84 years, who were at average risk for colorectal cancer.
Madison, Wisconsin-based Exact Sciences plans to submit the study data to the U.S. Food and Drug Administration as part of its approval application.
Colorectal cancer is the third most commonly diagnosed cancer in the United States, responsible for about 50,000 deaths a year.
The company's shares were down 28 percent to $7.16 before the bell. They closed at $9.97 on Wednesday on the Nasdaq.
Protandim or Tecfidera for Multiple Sclerosis
this study was funded by Biogen
Nrf2 activators: a novel strategy to promote oligodendrocyte survival in multiple sclerosis?
J. Lim, S. van der Pol, J. Drexhage, E. de Vries, J. van Horssen (Amsterdam, NL)
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Objectives: To investigate the potential of different Nrf2 activators to boost antioxidant enzyme expression in oligodendrocytes and protect them from reactive oxygen species (ROS)-mediated cell death.
Background: Oligodendrocyte damage and loss are key features of Multiple Sclerosis (MS) pathology and oligodendrocytes are particularly vulnerable to ROS-induced oxidative damage and cell death. Hence, a potential therapeutic strategy to protect these cells from ROS-mediated damage is urgently needed. To date, several compounds, including fumurate derivative BG-12, tert-Butylhydroquinone (tBHQ), sulforaphane (SFN) and protandim have potential anti-inflammatory and neuroprotective properties. These compounds are thought to exert their protective function via activation of the nuclear-factor-E2-related factor-2 (Nrf2) transcriptional pathway, which is involved in the production of antioxidant enzymes necessary for oxidative stress defense. We postulate that distinct Nrf2 activators boost antioxidant enzyme production in oligodendrocytes and limit ROS-mediated oligodendrocyte cell death.
Methods: Primary rat oligodendrocytes and rat and human oligodendrocyte cell lines were treated with different concentrations of BG-12, tBHQ, SFN and protandim. Next, we analyzed the expression of Nrf2-mediated antioxidant enzymes by PCR and Western blot techniques. To study the beneficial effects of the different Nrf2 activators, we first incubated the oligodendrocytes with Nrf2 activators and subsequently exposed them to various concentrations of hydrogen peroxide and measured oligodendrocyte cell survival.
Results:
1. BG-12, tBHQ, SFN and protandim are well-tolerated and strongly induce Nrf2-driven antioxidant enzyme production in oligodendrocytes, with protandim showing the most potent induction.
2. Nrf2 activators are able to protect oligodendrocytes against ROS-induced cytotoxicity.
Conclusions: Our findings indicate that several Nrf2 activators are able to significantly increase antioxidant enzyme production in oligodendrocytes. Interestingly, protandim, a dietary supplement consisting of herbal ingredients, was the most potent inducer and therefore may be the most suited as a therapeutic strategy. Importantly, Nrf2-mediated antioxidant enzyme expression in oligodendrocytes resulted in enhanced oligodendrocyte survival during an oxidative attack.
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Dr. J. van Horssen received research grants from BiogenIdec
Kenneth I. Moch Chimerix President and CEO
His management of Alteon was memorable as a failure.
as ceo of this company his claim to fame was filling grievances against Siga because they filed the Smallpox vaccine program under the small business enterprise application even though the ceo of Revlon is involved with Siga
Kenneth Moch joined Chimerix in 2009 as Chief Operating Officer and was named President and CEO in April 2010. He brings more than 30 years of experience in managing and financing biomedical technologies, having played a key role in building several life science companies. Prior to joining Chimerix, Mr. Moch founded Euclidean Life Science Advisors, a provider of strategic advisory services to life sciences companies, where he most recently served as President and CEO of BioMedical Enterprises, Inc. Previously, he was Managing Director, Healthcare Investment Banking, at ThinkEquity Partners. From 1998 to 2005, Mr. Moch served as President and CEO of Alteon Inc., a biotechnology company specializing in small molecule therapeutics for cardiovascular aging and diabetic complications, having joined in 1995 as SVP, Finance and Business Development & CFO. As President and CEO of the cellular therapy company Biocyte Corporation, Mr. Moch pioneered the storage and therapeutic use of cord blood stem cells and launched the first cord blood stem cell bank. Previously, he was a co-founder and Vice President of The Liposome Company, Inc., and a management consultant with McKinsey & Company, Inc.
Mr. Moch is a past Chairman of the Biotechnology Council of New Jersey and currently serves on the Emerging Companies Section Governing Board of the Biotechnology Industry Organization. He is a member of the Board of Directors of M2Gen/Moffitt Genetics Corporation, a for-profit partnership between Merck and the H. Lee Moffitt Cancer & Research Institute. Mr. Moch holds an A.B. in biochemistry from Princeton University and an MBA from the Stanford University Graduate School of Business.
Irina pumped the stock so they could get a financing done
it doesn't look like Cantor used her for the raise
All well, they owe her one.
Eric Topol interview
Ariad, comment from Jim Birchenough
http://research-ca.bmocapitalmarkets.com/documents/4b53d2f3-0197-48d4-8f7c-2e6c43f58325.pdf
Perhaps a cheap way to play cancer stem cells
..
Stem Cell Therapeutics Announces Joint-Venture Agreement With Reneu Inc. and Nexgen Medical Systems, Inc
Novel Combination of Neural Stem Cell Technology & Direct-to-Brain Administration of Therapeutics via an FDA-Cleared Neurocatheter
Press Release: JOINT VENTURES – Tue, Feb 19, 2013 4:30 PM EST.. .
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TORONTO, ONTARIO--(Marketwire - Feb. 19, 2013) - Stem Cell Therapeutics Corp. (TSX VENTURE:SSS) ("SCT" or the "Corporation") a life sciences company developing stem cell-related technologies, today announced a joint-venture agreement with ReNeu Inc. of Calgary, AB, Canada, ("ReNeu") and NexGen Medical Systems, Inc. of Melbourne, FL, USA ("NexGen").
ReNeu is a company formed to commercialize drug-based therapies to treat central nervous system disorders in combination with devices and processes for direct-to-brain administration. Subject to the definitive agreement, SCT and NexGen will license their respective technologies to ReNeu under world-wide, royalty-free licenses for the use of NexGen's FDA-cleared EViTAR™ line of proprietary intracranial delivery systems and SCT's NTx-265, -428 and -488 covering the proprietary use of drugs for the regeneration of endogenous neural stem cells.
To the knowledge of the Corporation the EViTAR™ intracranial delivery system is the only FDA-cleared Class II medical device currently indicated for the injection of material into the brain during intracranial procedures. It is designed for site-specific, low-volume, high-concentration delivery of drugs to target tissues in the brain of patients suffering from stroke, neurodegenerative disease, and tumors. SCT's NTx-265 is a therapeutic regimen of approved and clinically well-defined drugs that include human Chorionic Gonadotropin ("hCG") and erythropoietin ("EPO") for the regeneration of neural stem cells in patients following stroke, traumatic brain injury and other neurological conditions.
"ReNeu's perceptive innovation that the combination of SCT's award-winning, proprietary discoveries by Dr. Sam Weiss, Director of the Hotchkiss Brain Institute, if administered in a direct-to-brain process, holds the promise of delivering the regenerative results that Dr. Weiss' discovery originally proposed. NexGen's EViTAR family would appear to be an ideal vehicle for the clinical expansion of the SCT products and we are enthusiastic about its prospects," said David Allan, Executive Chairman of SCT.
Under the Letter of Agreement SCT will own 50% of the joint-venture for which the definitive agreement is anticipated within 60 days, subject to final due-diligence and usual conditions being met.
"The breakthrough benefit to patients that could be expected to result from a positive clinical demonstration from the combination of these two critical elements for the treatment of neurologically degenerative diseases could be of significant importance for the treatment of patients with stroke and other neurological conditions," said Dr. Joseph Tucker, founding CEO of ReNeu. "The most effective preclinical treatments from Dr. Weiss' work licensed to SCT were the direct-to-brain approaches, which clearly indicated the prospect of important benefit to patients when developed clinically. At the time, SCT proceeded in its clinical development with systemic rather than site-specific administration because of the absence of availability of a direct-to-brain approach, with the consequence that only limited amounts of SCT's drug combination reached the brain and equivocal clinical outcomes were achieved. We have high expectations that this fortuitous combination of these two novel approaches could have an importantly different and positive outcome."
"NexGen is very excited about this collaboration with SCT and ReNeu," said John Kucharczyk, CEO of NexGen Medical Systems. "The novel treatment methods have the potential to introduce entirely new therapies for several neurological diseases and disorders. NexGen's proprietary EViTAR™ neurocatheters are designed for delivery of therapeutic agents directly to target tissues in the brain and spinal cord, which is expected to significantly reduce side-effects associated with systemic delivery of drugs," Kucharczyk added.
ReNeu's initial board will be composed of David Allan, Executive Chairman of Stem Cell Therapeutics, Dr. John Kucharczyk, CEO of NexGen Medical Systems, Dr. Brad Thompson, Chairman & CEO of Oncolytics Biotech and Dr. Joseph Tucker, founding CEO of ReNeu. The Company intends to vigorously pursue regulatory approval to move the combined technologies into clinical trials with a target of clinical initiation in 2014.
SCT will retain all rights to its endogenous stem cell-stimulating technology other than direct-to-brain administration.
About Stem Cell Therapeutics Corp.:
Stem Cell Therapeutics Corp. develops stem cell-based therapeutics through partnerships with research institutions and technology transfer organizations. SCT's objectives include the sourcing and acquisition of additional stem cell-related development opportunities, and securing capital for the advancement of its licensed or acquired products. SCT is currently conducting a Phase II clinical trial in traumatic brain injury with NTx(R)-428 designed to characterize the ability of hCG followed by EPO to promote neurological and functional recovery of the brain following moderate-to-serious acute cortical (white matter) injury to the brain. For more information visit: www.StemCellThera.com.
About ReNeu Inc.:
ReNeu Inc. is a biotechnology company developing novel therapies for the treatment of central nervous system disorders. Our proprietary approach is to enhance brain remodeling and plasticity by administering neurogenesis-promoting drugs directly into the brains of patients. Our lead product, UnDo™, aims to improve brain and motor function in victims of stroke. For more information visit: www.ReNeuInc.com.
About NexGen Medical Systems, Inc.:
NexGen Medical Systems, Inc. is a private, development-stage Florida-based company that is developing catheter-based medical device products for therapeutic interventions in radiology, vascular surgery, and neurosurgery. The Company is commercializing an integrated medical device product line for removal of blood clots from head, neck, and peripheral vessels. The Company also develops access devices and technologies for the treatment of neurological diseases and disorders. NexGen is incorporated in Delaware and commenced operations in 2005. For more information visit: www.NexgenMedsystem.com.
Caution Regarding Forward-Looking Information:
This press release may contain forward-looking statements, which reflect SCT's current expectation regarding future events. These forward-looking statements involve risks and uncertainties that may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, SCT's ability to obtain financing in connection with the proposed arrangement; uncertainties as to the timing of the arrangement; the satisfaction of the conditions precedent to the completion of the arrangement; changing market conditions; the successful and timely completion of pre-clinical and clinical studies; the establishment of corporate alliances; the impact of competitive products and pricing; new product development risks; uncertainties related to the regulatory approval process or the ability to obtain drug product in sufficient quantity or at standards acceptable to health regulatory authorities to complete clinical trials or to meet commercial demand; and other risks detailed from time to time in SCT's ongoing quarterly and annual reporting. Except as required by applicable securities laws, SCT undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
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Contact:.
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Stem Cell Therapeutics Corp.
David Allan
Executive Chairman
+1 647 258 4325
DAllan@StemCellThera.com
WWW.StemCellThera.com
acad,,,put out news yesterday
It didn't move yesterday, why did it move today
have you ever seen a patent like this
their drug is two times a day, I can't believe if someone can come up with a once a day 480mg dose that they wouldn't be able to sell it.
Link to interview regarding the squalamine eye drop
http://www.wkyc.com/news/story.aspx?storyid=287233
Dr. Singerman is not a quack. He is a well respected founder of the Macular Society
http://www.maculasociety.org/history
History of the Macula Society
Lawrence J. Singerman
The Macula Society was founded in 1977 when Dr. Lawrence J. Singerman recognized the need for a forum to present and critique the rapidly expanding new research in retinal vascular and macular disease. He invited a few dozen leaders in these areas to the first meeting. He persuaded his mentor, Dr. Arnall Patz, to be the first president. He planned the meeting in Key Biscayne, Florida, to facilitate the presence of Dr. J. Donald Gass, who opened the first session and moderated the last session of the meeting. Dr. Gass also became the second president of the Society. At the first meeting, committee chairmen were nominated and elected by the attendees.
Still, after 37 years, the purpose of the Society is to provide a forum for the presentation and dissemination of the most current and most advanced scientific information and research available in retinal vascular and macular diseases.
Currently, the Macula Society has 409 members, including many international members. Applications are accepted only once a year, and members are selected via rigorous acceptance criteria, including extensive contribution to retinal literature. Membership is, therefore, smaller than some organizations, which has promoted exceptional camaraderie and loyalty to the Society. Most members come to almost every meeting. The vast majority of members is involved in cutting-edge research and/or are investigators in major clinical trials.
Ohr Pharma: Under The Radar Company With Upcoming Catalysts, Potential For Significant Pay-Off
http://seekingalpha.com/article/1262821-ohr-pharma-under-the-radar-company-with-upcoming-catalysts-potential-for-significant-pay-off?source=yahoo
By Jake King
Wet macular degeneration is a chronic eye disease that causes vision loss and generally occurs later in life (50+), as blood vessels leak fluid or blood into the macula. With an aging baby-boomer generation, treatments for wet AMD have been highly lucrative for leading developers Roche/Novartis (RHHBY.OB)(NVS) and Regeneron (REGN), with a current $3.4B annual treatment market that is anticipated to grow 22% this year. The treatments are effective, but their biggest downside is that they require injection on a monthly or bi-monthly basis; this dosing regimen is invasive and not optimal for patients.
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As an alternative, Ohr Pharmaceuticals (OHRP.OB) is developing an eye-drop for wet-AMD, and although the new formulation has yet to complete a human proof-of-concept trial, the active ingredient, squalamine, has shown efficacy against neovascularization and angiogenesis (the underlying causes of wet AMD) in previous trials as an intravenous (IV) preparation. Clearly, a successful Phase II trial for this drug candidate could be worth a substantial sum, hence the tremendous upside potential in OHRP stock. Interestingly, investors may not need to take on as much risk as this kind of trade might imply, as the company is approaching several other milestone events that may protect against downside into the squalamine eye drop Phase II interim results anticipated by year-end. The company's other development-stage compound, OHR-118, is also undergoing Phase II development as a treatment for cachexia (or patient "wasting") associated with cancer. Importantly, minimal value, if any, is being factored into OHRP stock for this drug candidate. Results are expected before the end of this month, and if positive, expect the stock to rise substantially. Additionally, shares of OHRP are likely to up-list to the NASDAQ (from the OTC Bulletin Board) in the next couple of months as a considerable amount of "in-the-money" warrants convert to common stock, which could bring in up to $14.3M in cash and boost the company's shareholder's equity to a level that enables a NASDAQ listing. The up-listing is a clear positive, and the two Phase II trials for both squalamine eye drops and OHR-118 enable investors to own OHR with two chances to win. Should the cachexia candidate, OHR-118, succeed in Phase II, value will be created in short order, while a poor result for this drug candidate shouldn't impact OHRP all that much given that the story remains about squalamine for wet AMD. As a result, we expect shares of OHRP to become significantly more visible over the next several weeks -- and into the 2nd half of the year with an up-listing -- with speculative investors taking positions in the name ahead of these key events.
Squalamine eye drop has potential to tap huge market with clear differentiation. Macular degeneration causes damage to the macula, the central part of the retina at the back of the eye that picks up fine detail in the center of the visual field. In wet AMD, abnormal blood vessels under the retina begin to grow toward the macula, and because these blood vessels are abnormal, they tend to break, bleed, and leak fluid, damaging the macula and causing it to lift up and pull away from its base. This can result in a rapid and severe loss of central vision. Anti-angiogenic drugs, like Roche/Novartis' Lucentis and Regeneron's Eylea, have proven to be the most effective treatments for wet AMD, and both inhibit VEGF (vascular endothelial growth factor) to prevent new blood vessel growth, thereby reducing breaks, leaks and the common "spotting" that occurs in patients suffering from wet AMD. Squalamine, like these leading treatments, is an inhibitor of VEGF, but also inhibits platelet-derived growth factor (PDGF) and basic fibroblast growth factor (bFGF). While current treatments are monthly or bi-monthly injections, squalamine, as it is being tested by Ohr, is a twice-daily eye drop. Squalamine is a small molecule (Lucentis and Avastin are monoclonal antibodies) and has already demonstrated clinical efficacy in wet AMD through IV administration. In Phase I and II studies, the drug showed improvements in visual acuity and lesion response with no major adverse events. But in 2007, this IV formulation was dropped for lack of commercial viability by Genaera Corp. (which went bankrupt shortly thereafter) because treatment with IV squalamine necessitated IV infusions on a weekly basis, a very inconvenient proposition due to the drug's short half-life. Additionally, IV squalamine generated infusion site reactions, and the availability and rapid acceptance of other treatments that improve vision in AMD patients made IV squalamine a non-starter.
Since then, Ohr, which picked up the candidate in 2009, has reformulated squalamine as an eye drop and has advanced it into an ongoing Phase II efficacy and safety study for wet AMD. Pre-clinical work utilizing the Dutch Belted Rabbit (arguably ideal for human comparison) demonstrated that at "trough" levels, the eye drop "exceeded concentrations of squalamine that are known to block the choroidal neovascularization process in Wet-AMD." That threshold, according to Ohr, is 11ng/g, and researchers saw squalamine concentrations at the back of the eye of 60ng/g+ at all time-points and dosing regimens in the animal study. This is key, as the skeptics of squalamine eye drop would like proof that sufficient concentrations of the drug can travel to the back of the eye, and this preclinical "distribution" data suggest that it can. With evidence in hand that squalamine was making it to the macula, the company initiated a randomized, double blind, placebo controlled Phase II study (n=120) in treatment naïve wet AMD patients, with fast-track status granted by the FDA. The trial is evaluating squalamine eye drops in-part as a concomitant therapy with standard of care; patients receive an initial injection of Lucentis prior to randomization and are then evaluated monthly for their need for further injections. A home-run would be improvement or stabilization using squalamine eye drop as a monotherapy, but even if the drops reduce the frequency of Lucentis injections, which are expensive, invasive and slightly risky, there's a clear commercial opportunity. An interim read-out is due upon the completion of the treatment period in 50% of patients, which the company expects by the end of this year. If positive, squalamine eye drops could be highly disruptive to the current, invasive wet AMD treatment paradigm, as a standalone therapy or as an adjunct to existing injections. With an aging baby boomer generation and 10% of the 3 million American AMD sufferers (by 2020) eventually digressing to the wet form of AMD, the patient population is substantial. Analysts anticipate that Regereron's Eylea could have peak sales of $4B, and if Squalamine Eye Drops prove to benefit wet AMD patients and become an FDA-approved product, this drug could also be a multibillion-dollar seller.
OHR-118 Phase II data approaching, perhaps a free "call option" for OHRP shareholders. In the next couple of weeks, Ohr expects to release top-line data for OHR-118, a Phase II candidate for the treatment of cancer cachexia. What's most important to note, however, is that OHR-118 could be a free "call option" in the OHRP story, as it currently plays a negligible role in the company's valuation. Investors have exposure to considerable upside surprise if the Phase II results this month demonstrate quality outcomes for cachectic patients, as we believe that with the focus on squalamine eye drops, a poor result for this cachexia product won't have a crushing impact on the stock. Cancer cachexia is a severe wasting disorder that usually affects late-stage cancer patients and is characterized by muscle atrophy, fatigue, weight loss, weakness, and loss of appetite. Severe cachexia not only lowers patient quality of life -- often during the times when quality is most crucial -- but can hamper proper cancer treatment, as weakened patients simply can't tolerate further chemotherapy. A 2009 study claims that as many as 23% of all cancer patients will have cancer cachexia at some point during treatment, and that cachexia is the immediate cause of death in 20% to 40% of cancer patients; thus, the opportunity for an effective treatment is quite large. Par Pharmaceutical's Megace ES product (a long-acting reformulation of generic megestrol acetate) generates about $100M in annual sales for cachexia. However, because it competes with cheaper generics, the Megace market, at branded prices, is actually much larger than Par's sales indicate (Megace ES has only ~15% of the megestrol acetate market according to IMS Health prescription data). Nevertheless, if OHR-118 could generate the same $100M that Megace ES generates, using a 3x multiple on peak sales would indicate a $300M value for OHR-118, if approved. Those kind of numbers suggest that if the Phase II cachexia trial is a success, OHRP shares will climb significantly higher.
OHR-118 has already demonstrated beneficial effects in mitigating symptoms of AIDS cachexia, and while the ongoing Phase II study is small and open-label, interim results from the same trial - although at a different location - were favorable with statistical significance. Weight stabilization or gain was observed in 7 of 11 patients., and Total Patient Generated Subjective Global Assessment scores improved significantly (p=< 0.01); appetite (p=< 0.01) and depression (p=0.05) scores improved on the Edmonton Symptoms Assessment Scale; and frequent burping/belching (p=0.02), feeling full (p=0.04) and stomach distention (p=0.03) improved on the Dyspepsia Symptom Severity Index. And, Simmonds Functional Assessment tests showed improvement on time to sit and stand (p=0.01). OHR-118 was well tolerated with no serious side effects, and while the candidate is not a crucial part of the OHRP story, it may offer surprise upside yet in the first quarter of this year. We consider it a free call option for OHRP investors, and quality data from this product alone is enough to justify the stock's current valuation and more.
Company quietly and efficiently advanced its pipeline in 2012. We met with OHRP a little over a year ago, and the company had little to show at the time, with squalamine just reformulated as an eye drop and undergoing pre-clinical proof-of-concept trials, and OHR-118 more than a year away from Phase II data. The company spent less than $2M last year, but management was able to move the ball significantly forward. OHR received FDA approval to go directly into a Phase II trial with its squalamine eye drop formulation; FDA also granted the program "Fast Track" status; the company completed its Phase II cancer cachexia study for OHR-118; and initiated and enrolled patients in the squalamine eye drop wet AMD Phase II trial. Additionally, OHRP has assembled a top-tier Scientific Advisory Board with key opinion leaders that have previously served as investigators and advisors for all of the major commercial wet AMD treatments: Visudyne, Macugen, Lucentis, and Eylea (see OHRP's SAB here ). The company has also conducted early business development discussions with larger pharmaceutical companies, although Phase II data for each asset are needed to take these activities to the next level. Ohr has accomplished all of this on a shoe-string budget by controlling spending carefully and using stock options instead of cash in many cases, for R&D resources and Management/Board compensation. Management's salaries are well below industry norms, suggesting that these individuals are incentivized to be aligned with shareholder interests. A key to the company's success is CEO Dr. Irach Taraporewala, who has over 30 years of experience in drug development and regulatory affairs. Dr. Taraporewala was a senior drug development consultant for leading contract research organization (CRO), PAREXEL International, and this experience enables OHRP to conduct its clinical programs efficiently, effectively, and with minimal resources. In addition to successfully developing many drugs through FDA approval for pharmaceutical companies that contracted with PAREXEL, Dr. Taraporewala also consulted for companies with ophthalmology assets and led ophthalmic drug development for Mystic Pharmaceuticals.
Cash infusion and NASDAQ up-listing expected to raise visibility and stock price. A nice bonus in the OHRP story is the potential for the company to list its shares on the NASDAQ, an improvement from their current OTC Bulletin Board listing. The pathway for a NASDAQ listing has become clear as the company moves closer to the exercise of warrants that could bring in a substantial amount of cash and increase shareholder equity to satisfy NASDAQ requirements. Approximately 11.99 million warrants for OHRP are set to expire on April 30th, and given their exercise price of $1.19, most, if not all, of these warrants will likely be exercised, bringing in cash to the company of about $14.26 million (if 100% are exercised). These warrants are from a prior financing conducted to inject funding into the public vehicle that later became Ohr Pharmaceuticals. Importantly, most investors in that round of financing remain active with the company and have continued to participate in subsequent financings for OHRP (5 financings each at sequentially higher valuations). This means that most of the warrants are likely to be exercised on or before April 30th, and that the company should meet shareholder capital requirements for the NASDAQ up-listing in tandem. If any of these "in-the-money" warrants are not exercised by April 30th, they expire worthless and the company's fully diluted share count will be reduced. Additionally, the company plans to remove a separate "non-cash warrant derivative liability" on its balance sheet through a vote of certain warrant holders to eliminate an anti-dilution provision. The company already has the more-than 50% of the votes necessary to remove this weighted average anti-dilution provision, hence, the balance sheet should look even better to NASDAQ once the derivative liability has been removed. Overall, the influx of significant cash and expected up-listing of the stock should have a positive impact on OHRP shares. Depending on the outcome of the Phase II cancer cachexia trial for OHR-118, the company may or may not need to reverse-split its stock to achieve the $3.00 closing price for a NASDAQ listing under the shareholder capital standard. The good news is that in cases where a reverse split is conducted by a company to initially list on NASDAQ, investors usually welcome the action with a higher stock price, as opposed to companies that reverse split simply to maintain their NASDAQ listing. Once listed, OHRP shares will become visible to a much wider range of investors and certain index funds may also start to pick up the stock, hence upside to the current valuation.
Several reasons to own OHRP. OHRP is heading towards a number of events, and a positive result for OHR-118 may set the stage for the shares to trade sharply higher over the next few months. We believe investors can play the OHR-118 Phase II results with little risk given that the key reasons to own the stock will still exist, no matter the cachexia outcome, including a potential NASDAQ up-listing for OHRP shares and Phase II results for squalamine eye drops expected later this year. With an immense market for effective wet AMD treatments, particularly from a disruptive technology like an eye drop, the risk/reward with OHRP appears favorable, and even as a short-term trade, investors can take advantage of two potentially positive catalysts within the next few months, squalamine Phase II results aside.
Additional disclosure: PropThink is a team of editors, analysts and writers. This article was written by Jake King. We did not receive compensation for this article, and we have no business relationship with any company whose stock is mentioned in this article. Use of PropThink’s research is at your own risk. You should do your own research and due diligence before making any investment decision with respect to securities covered herein. You should assume that as of the publication date of any report or letter, PropThink, LLC and persons or entities with whom it has relationships (collectively referred to as "PropThink") has a position in all stocks (and/or options of the stock) covered herein that is consistent with the position set forth in our research report. Following publication of any report or letter, PropThink intends to continue transacting in the securities covered herein, and we may be long, short, or neutral at any time hereafter regardless of our initial recommendation. To the best of our knowledge and belief, all information contained herein is accurate and reliable, and has been obtained from public sources we believe to be accurate and reliable, and not from company insiders or persons who have a relationship with company insiders. Our full disclaimer is available at www.propthink.com/disclaimer.
Billionaires Anoint Biogeeks
http://www.forbes.com/sites/matthewherper/2013/02/20/billionaires-anoint-biogeeks/
Hey Nobel Prize in Medicine! You’ve got competition. This morning, a bevy of techoscenti billionaires including Mark Zuckerberg and Sergey Brin announced the first recipients of their Breakthrough Prize in Life Sciences, a $3 million award ”recognizing excellence in research aimed at curing intractable diseases and extending human life.” This year, there are 11 recipients, meaning a total $33 million payout, but in the future five annual awards will be planned, totalling $15 million.
The award is the brainchild of billionaire Yuri Milner, an early Facebook investor, who gives a similar prize in physics. Aside from Brin and Zuckerberg, he is joined by: Anne Wojcicki, Brin’s wife and the chief executive of 23andMe, the consumer genetics startup; Priscilla Chan, Zuckerberg’s wife and a physician who is in residency; and Art Levinson, the chairman of the board at Apple who previously, as chief executive of Genentech, was the most respected leader in the pharmaceutical industry. The awards are being announced formally later today at conference led by Susan Desmond-Hellmann, once Levinson’s right-hand woman and now chancellor of UCSF.
23andMe Nabs Billionaire Yuri Milner As Investor, Cuts Price To $99 Matthew HerperForbes Staff
Bill Gates Invests In Cancer DNA Sequencing Firm Matthew HerperForbes Staff
30 Under 30: The Rising Stars Transforming Science And Health Matthew HerperForbes Staff
“I believe this new prize will shine a light on the extraordinary achievements of the outstanding minds in the field of life sciences, enhance medical innovation, and ultimately become a platform for recognizing future discoveries,” Levinson said in a prepared statement. Added Brin: “Curing a disease should be worth more than a touchdown.”
So what brain trust did this brain trust decide to present checks to? Interestingly, though not surprisingly, it’s a list that focuses on those who have actually changed medicine and biotechnology, even inventing new drugs. One is even one of Levinson’s old employees. The winners:
Cornelia I. Bargmann of Rockefeller University studies a primitive worm, C. elegans, which has only 302 neurons in its brain. The idea is that by studying this tiny brain, we can learn how brains work, and Bargmann’s opinions are sought out by neuroscientists and psychiatrists.
David Botstein of Princeton University developed, in 1980, a method for discovering disease genes that was used to find the BRCA breast cancer gene, a test for which is the main product of Myriad Genetics, and the genetic mutation that causes Huntington’s. Eighteen years later he led a team that figured out new ways to analyze data from the tiny “DNA chips” that were revolutionizing genetics. He’s now studying how yeast consume food and create energy, an effort that could lead to a new understanding of lots of biological problems, including how cancer cells eat.
Lewis C. Cantley of Weill Cornell Medical College, who discovered an enzyme called PI3 kinase that is a key drug target for new medicines against cancer. He is a co-founder of Agios Pharmaceuticals and a pioneer in the field of Systems Biology, which tries to put the pieces looked at by geneticists and molecular biologists into a more coherent whole.
Dr. Hans Clevers, director of the Hubrecht Institute for Developmental Biology and Stem Cell Research in the Netherlands, who has been one of the leading figures in the discovery that stem cells play a role in the development of cancer.
Dr. Napoleone Ferrara of the University of California, San Diego, at Genentech did work that led the creation of Avastin, for colon and other cancers, and Lucentis, for age-related macular degeneration. Both drugs work by stopping a process called angiogenesis, which in feeds tumors and, in eye disease, results in too much blood entering the eye and causing damage.
Titia de Lange of Rockefeller University studies telomeres, the protective ends of the chromosomes that contain human DNA. When these degrade, they can lead to some types of cancer.
Eric S. Lander of the Broad Institute of Harvard and the Massachusetts Institute of Technology. Lander is most famous for his leadership role in the Human Genome Project and for his work running the Broad Institute, which has become one of the hot spots for genetic research. But he’s also responsible for helping to develop a molecular taxonomy of cancer and for mentoring many of the top researchers in the field.
Dr. Charles L. Sawyers It’s not just that Sawyers, of the Memorial Sloan-Kettering Cancer Center, looks into the molecular signals that drive a cell to cancer, it’s that he’s twice played a key role in getting drugs that affect those signals to patients. The first time was with Gleevec, from Novartis, which started off the race for DNA-targeted cancer drugs. The second, more recently, was Medivation’s Xtandi, for prostate cancer patients, which was recently approved.
Dr. Bert Vogelstein of Johns Hopkins University, devised a model for the progression of colon cancer that is widely used in colonoscopy. More recently he is one of the very top minds looking at how genetic alterations lead to cancer.
Robert A. Weinberg of M.I.T., discovered the first human gene that, when mutated, causes cancer, setting up science to understand cancer as a series of genetic mutations.
Dr. Shinya Yamanaka of Kyoto University and the Gladstone Institutes in San Francisco, figured out how to make what are known as induced pluripotent stem cells. These resemble embryonic stem cells, which can in theory turn into any part of the body, but they are far more useful for studying what goes wrong to make disease happen. In a few years they have already revolutionized biology.
Next year, this year’s winners will help pick the next batch of luminaries.
Is this a good way for Zuckerberg et. al to spend their cash? There’s no way to argue with that list: those are great people to be giving an award and they all deserve it. I’m mostly thrilled at the idea that Vogelstein and Bargmann and Yamanaka are all going to be flush.
But I have trouble believing that this award is going to make any of them much more famous. Most people reading this will probably remember Milner and Brin more than Sawyers and Napo Ferrera. And, as Forbes contributor David Shaywitz noted on Twitter, among biologists these folks are already idolized. What they all need are funds to keep them in the field, not new people to look up to. Brin is speaking my language when he says that curing disease should be worth more than a touchdown. Even with prizes that big, I’m not sure it is.
Still, the awards are a good step. Let’s hope that as years pass, they draw more and more attention to the amazing things happening in science and medicine.
Update: I interviewed Art Levinson about the prize.
Thanks to Dennis Overbye at the New York Times, whose collection of links to the web pages of the winners I used in creating this story. This post has been updated from its original version.
My cousin died last month from esophogeal cancer. He was 61
he was diagnosed four years ago by accident. they said it was a miracle that they found it so early. the removed part of his esophagus and took out part of his stomach to replace it. Terrible surgery the incision never healed so he had a constant rupture and couldn't eat full meals.
in spite of getting it so early and removing the area where it was discovered it came back a year ago and he died twelve months later.
your loved one should not go through the surgery if it is suggested. This was performed at Memorial Sloan
sqnm to take a dump on Monday
..
Sequenom Provides Update On Status Of Patent For Methods Of Detecting Fetal Aneuploidy
Press Release: Sequenom, Inc. – 17 hours ago.. .
.
SAN DIEGO, Dec. 14, 2012 /PRNewswire/ -- Sequenom, Inc. (SQNM), a life sciences company providing innovative genetic analysis solutions, today announced that the U.S. Patent and Trademark Office has withdrawn the issuance of U.S. Patent No. 8,340,916. Sequenom has been informed by the Patent and Trademark Office that issuance was withdrawn in connection with an anticipated patent interference proceeding between parties with pending patent applications or issued patents directed to similar subject matter.
Sequenom had previously been notified that the patent would issue on December 25, 2012, with claims to novel methods for detecting fetal aneuploidy using massively parallel sequencing. The patent is entitled Diagnosing Fetal Chromosomal Aneuploidy Using Massively Parallel Genomic Sequencing and was invented by Drs. Dennis Lo, Rossa Chiu and Alan Chan of the Chinese University of Hong Kong. Sequenom has exclusive rights to this patent.
In the trial that pfizer did against Humira, Humira had a 39 percent acr20 response
In this trial Humira's acr20 reponse was 59 percent they highest it ever reached in a clinical trial, so there was something else that was going on
The way azn and rigel are positioning the drug is that it has the best response in protecting bone loss in RA. That is a meaningful endpoint and if it succeeds that would be a meaningful claim in the market place
I have never seen a JD (attorney) using the Dr. prefix to their name and since United Therapeutics is a life science company using the prefix in front of her name would lead to a lot of confusion. Unless that is your intent I would not use it, and I do not think you should.
I don't think Rothblatt is a medical doctor
he/she made her first fortune with Sirius Satellite before it had satellites.
Kythera Biopharmaceuticals raises $70.4M in IPO
Published: Thursday, 11 Oct 2012 | 9:13 AM ET
If you get the injection on your tush, it gets rid of the crease in your butt, but wouldn't that cause constipation?
Kythera Biopharmaceuticals Inc. has raised about $70.4 million in its initial public offering of 4.4 million shares.
The Calabasas, Calif., company, which is developing an injectable treatment for double chins, priced its IPO at $16 per share, or the top end of its estimated range. Shares of the stock are expected to start trading Thursday on the Nasdaq Global Select Market under the ticker symbol "KYTH."
Counting expenses, Kythera expects net proceeds of about $62.8 million, or $72.6 million if underwriters exercise an option to buy additional shares to meet demand. Kythera plans to use nearly all proceeds to pay for late-stage clinical studies of its double-chin treatment, which is labeled ATX-101.
The company, which has no products on the market, said ATX-101 involves a synthetic formulation of a component of human bile that breaks down fat. The company's development partner, Bayer Consumer Care AG, has conducted late-stage research on the injectable in Europe, and the companies expect to submit the drug for approval there next year.
Kythera also expects to report initial results from late-stage research in the United States and Canada by the middle of next year.
The drug developer reported a net loss of $11.1 million last year and $2.4 million through the first six months of 2012.
Copyright 2012 The Associated Press. All rights reserved. This material may not be published, broadcast, rewritten or redistributed.
tekmira vs sarepta dod ebola dod press release
Yesterday the U.S. Department of Defense (DoD) announced that it was re-starting Tekmira's (TSX: TKM, Nasdaq: TKMR) ebola program (press release below). Back in 2010, DoD awarded two $100 million+ contracts for the development of a product to treat ebola virus, one to Tekmira and the other to Washington based Sarepta Therapeutics (Nasdaq: SRPT). According to DoD, after a thorough evaluation of the two programs, they have decided to only proceed with one and that is Tekmira. Obviously this is tremendous news for Tekmira. To date the DoD contract has contributed approximately $20 million+ in top-line revenue for Tekmira and allowed the company to make enormous improvements in their manufacturing process and scale. With the contract re-started, we can once again count on DoD's healthy contribution to Tekmira's top-line. Furthermore, with DoD having just completed a thorough review in a difficult economic climate and leaving only Tekmira standing, it would seem, barring any clinical set backs, that this program is relatively safe for years to come and hopefully all the way through FDA approval. Lastly when you consider the recent Marqibo approval and the royalty revenue it will be generating for Tekmira, it is reasonable to think that we will be talking about Tekmira's growing top-line in 2013 and beyond.
FORT BELVOIR, Va., Oct. 2, 2012 /PRNewswire via COMTEX/ -- JPM-TMT's Strategy in Accordance with Original RFP
The Department of Defense's (DoD) Joint Program Executive Office for Chemical and Biological Defense, Joint Project Manager Transformational Medical Technologies (JPM-TMT), completed a comprehensive evaluation of its Ebola medical countermeasure (MCM) candidate development efforts and decided that the Hemorrhagic Fever Virus (HFV) program will continue with only one of two Ebola MCM development efforts. The Tekmira Pharmaceuticals, Inc. Ebola MCM was selected for continued development.
"The decision to down select at this time was strategic and crucial," said David E. Hough, Joint Project Manager for JPM-TMT. "The HFV acquisition strategy has always included a down selection prior to entering the next phase of development but near term programmatic issues required an earlier than planned decision."
"Our job is to ensure we're making the most out of every dollar we spend," he noted. "We evaluated each contractor's efforts independently. The evaluation was conducted using all current data and in accordance with the criteria set forth in the awarded contracts and as stated in the initial solicitation. This was certainly a tough decision but the final decision was made to move forward on continuing the development of the drug candidate that represents the best value to the Government based on what we know today."
Through its Hemorrhagic Fever Virus-Therapeutics Medical Countermeasures HFV-Tx MCM) acquisition program, JPM-TMT is targeting the Ebola virus, as well as the Marburg virus, as models for testing the development of rapidly adaptable, platform-based, post-exposure therapeutics for all HFVs. While both viruses have been mostly concentrated in certain parts of Africa, they are considered as serious threats by the DoD for their potential use as biowarfare agents.
About Joint Project Manager Transformational Medical Technologies (JPM-TMT)
JPM-TMT is a component of the U.S. Department of Defense's Joint Program Executive Office for Chemical and Biological Defense (JPEO-CBD). JPM-TMT aims to protect the Warfighter from emerging infectious diseases, genetically altered, and unknown biological threats. Through strategic investments and partnerships with innovative biotech firms, pharmaceutical corporations, other government agencies, and academic institutions, JPM-TMT facilitates the advanced development and acquisition of adaptable platform technologies, broad-spectrum medical countermeasures, and innovative systems to enhance our nation's biodefense response capability. For more information, visit www.jpmtmt.mil .
SOURCE Joint Project Manager Transformational Medical Technologies
Copyright (C) 2012 PR Newswire. All rights reserved
I guess it was celgene so they are above such neccessities
CELG: A Very Negative Revlimid Trial in Prostate Cancer Caused Outrage among Clinicians
• In a late-breaker presentation on Sunday, Celgene's Phase 3 trial of Revlimid in
combination with docetaxel and prednisone was presented. The trial showed a dramatic
detriment in patients who received Revlimid. The hazard ratio comparing the Revlimid arm
and the placebo arm was 1.53 (1.17, 2), meaning a prostate cancer patient who received
Revlimid had a 53% higher chance of dying compared to a patient who received placebo. The
PFS was similar between the 2 arms: 45-week Revlimid versus 46-week placebo. The
objective response numerically favored placebo: 22.1% Revlimd versus 24.3% placebo.
• The trial received a scathing review by the discussant following the data
presentation. He pointed out that this disastrous outcome could have been avoided, since
there were few early clinical trial data to support moving the drug into a large-scale
Phase 3 trial which involved over 1,000 patients, and there were earlier data pointing to
overlapping toxicity of the drugs in the regimen. Indeed, we couldn't recall ever seeing
a large-scale Phase 3 trial that resulted in a 53% increase in the risk of patients
dying. In most cases when a Phase 3 trial fails, there is still a trend towards the drug
having benefit, just not to the degree of statistical significance. At worst, the failed
trials result in a slight detriment in outcome, but never to this degree. Indeed, the
main point of Phase 1 and Phase 2 trials is to minimize avoidable risks in a large group
of trial participants in Phase 3. We think this trial will cause many institutional
review boards to think twice before approving a Celgene-sponsored Revlimid trial in solid
tumors. Additionally, almost all solid tumor therapies involve some cytotoxic agents
administered either concurrently or in tandem with the proposed use of Revlimid. The
overlapping toxicity of Revlimid with these agents should call into question Celgene's
strategy of expanding Revlimid usage beyond hematology and into solid tumors.
May 24, 2012 From Alex To -- CLDX: Striking Breast Cancer Data
• Once in a while in cancer research, we have a nirvana moment. One of the new drugs
demonstrates striking efficacy in a particular cancer type. We have been lucky in the
last 3 years to have had several such moments. To those we would include when Pfizer
(PFE) presented initial Xalkori (crizotinib) trial results in ALK-positive lung cancer,
and when Plexxicon (later acquired by Roche) presented initial Zelboraf trial results in
BRAF-positive melanoma. Now we can add to these milestones of cancer research yesterday
afternoon's data release by Celldex on the CDX-011 Phase 2 trial in GPNMB-positive breast
cancer.
• The efficacy was most striking in the triple negative patients who are also high
GPNMB expressers: 36% partial response rate in patients who received CDX-011 versus 0% in
the patients receiving best of current care. The disease control rates were even more
telling: 82% versus 33%. Even with the data set being highly immature, the
progression-free survival (PFS) in this patient population is already statistically
significant. We would certainly consider this data set in the league of crizotinib and
Zelboraf data cited above. Importantly, even only counting the triple negative breast
cancer patients who are also high expressers of GPNMB, the market for CDX-011 is already
far bigger than crizotinib's and about in on a par with Zelboraf. But, the data for
CDX-011 in triple negative patients in general, and in GPNMB-positive patients in
general, are also encouraging. The striking data in the triple negative patients have
much to do with the complete lack of treatment options for these patients. We suspect
that in other subgroups of breast cancer, if CDX-011 were combined with Herceptin
(ROG.VX) in HER2-positive patients, or combined with Afinitor (NVS) in ER-positive
patients, insofar as these patients are high GPNMB expressers, the results could be very
good as well. This is before the drug is explored in a slew of other cancers outside of
breast cancer. In words, CDX-011 is not only the latest success in targeted therapy, it
also stands to be the targeted cancer therapy with the broadest potential application.
Consequently, the commercial potential of the drug could be beyond anything we have seen
to date in the cancer market.
• So, for the army of business development personnel from big cancer players who are
packing their bags heading to Chicago for ASCO next week, they may want to consider
changing their destination to Phillipsburg, New Jersey instead.
Alex To, MD
Managing Member
Cross Current Research, LLC
2 Research Way, 2nd Floor
Princeton, NJ 08540
609-243-0082 (office)
917-584-6903 (cell)
alex.to@crosscurrentllc.com
https://www.crosscurrentllc.com
a broken watcah is right two times a day
a growth stock with momentum with no IP that trades at 40 times earnings that only needs to miss one quarter is a very dangerous stock to hold.
shouldn't even trade at 80
shows the market is inflated.
is this moronic or not
As of the date, since the inception of our $750 million buyback authorization in August of 2010, we have repurchased $532 million of common stock, representing approximately 7 million shares.
Stock buy back at 40 time earnings is the best use of cash?
I guess it is if you want to exercise your options.
we'll see said the blind man
the drug was previously in phase 3 trials using an IV formulation so the fda had sufficient evidence that the drug was safe.
Now they need to show that it works.
I do not need you to become a believer
It doesn't matter to me
When you give a drug IV once a week you aren't getting enough drug into the back of the eye.
once genaera went out past one month they gave the drug once a month.
the drug was only working for 3 to five days of every month.
the eyedrop will be given twice a day.
If a person takes lipitor 3 days a month would it lower their cholesteral
these are the tops in the field.
they were very involved in the set up of the clinical trial, the aren't just putting their names on this.
Ohr Pharmaceutical Announces Ophthalmic Scientific Advisory Board
I think the people on this Scientific Advisory Board would know better than the people on the biotech values board whether this eyedrop has a chance to work.
NEW YORK, NY--(Marketwire - May 1, 2012) - Ohr Pharmaceutical Inc. (OTCBB: OHRP) today announced the formation of a Scientific Advisory Board ("SAB") comprised of leading retinal ophthalmology experts to assist in advancing the Squalamine eye drop program into clinical efficacy trials. Thomas A. Ciulla, M.D. of the Midwest Eye Institute, Michael J. Elman, M.D. of the Elman Retina Group, and Jason S. Slakter, M.D. of the Vitreous Retina Macula Consultants of NY group have agreed to join as members of the SAB. These highly regarded experts will advise the company on its clinical programs, regulatory strategy and provide strategic guidance to support the development of Squalamine in multiple neovascular ophthalmic clinical indications each with sizable commercial markets.
"I am very pleased to be joining the Ophthalmic Scientific Advisory Board of Ohr Pharmaceutical," commented Dr. Slakter. "The Company's eye drop for treating wet-AMD would potentially offer patients a convenient, self-administered, treatment alternative or adjunct to currently used intravitreal injections directly into the eye. This would be an exciting and significant advancement in the therapy of wet-AMD should the therapy be proven effective."
"We are excited to be working with key opinion leaders in the retina community," stated Irach B. Taraporewala, Ph.D., CEO. "Their prior experience in fostering the development of numerous FDA approved therapeutics, including Eylea® and Lucentis®, will provide us with immeasurable assistance as we move the Squalamine eye drop program into a clinical trial for the treatment of wet-AMD in the near future."
Biographies for each SAB member are below:
Dr. Thomas Ciulla, M.D., is a retinal physician who serves as Director of the Midwest Eye Institute in Indianapolis, IN. Dr. Ciulla is board-certified in ophthalmology and has served as Co-Director of the Retina Service and Associate Professor of Ophthalmology on the faculty of the Indiana University School of Medicine, prior to entering private practice at the Midwest Eye Institute. Through Midwest Eye Institute, Dr. Ciulla performs clinical studies in macular and retinal diseases, with particular emphasis on age-related macular degeneration, diabetic retinopathy and retinal occlusions. He has been the principal investigator and sub-investigator on clinical trials sponsored by the NIH and NEI and has served as a consultant, Principal Investigator or Medical Monitor for numerous ophthalmic pharmaceutical companies. He has served on the Advisory Boards of numerous large medically affiliated companies, has lectured nationally and internationally, has authored nearly 100 publications, and co-edited two major textbooks.
Dr. Michael Elman, M.D., is a board certified ophthalmologist and has practiced ophthalmology for over 30 years, specializing in diseases of the retina and vitreous. Author of more than 150 publications on retinal diseases and surgery, Dr. Elman is an Assistant Professor of Ophthalmology at the Johns Hopkins University School of Medicine. He is currently the Director of Retina Surgery at Franklin Square Hospital. Dr. Elman directed the Clinical Research Unit at the University of Maryland and was bestowed the Young Scholar Award for the entire University of Maryland. He serves as the Chairman of Executive Committee of the National Eye Institute's Diabetic Retinopathy Clinical Research Network (DRCRnet). He was also Study Chairman of the DRCRnet's Protocol I, a national collaborative clinical trial that recently revolutionized the treatment of diabetic macular edema.
Dr. Jason Slakter, M.D., of the Vitreous Retina Macula Consultants of New York is an internationally recognized retinal and macular disease specialist, and is the Founder and Director of the Digital Angiography Reading Center (DARC) in New York, which is the largest center for ocular image evaluation for clinical trials of posterior segment disease with over 800 certified clinical sites in over 40 countries worldwide. As Director of DARC, a principal investigator of many clinical trials, and a pharmaceutical industry consultant, Dr. Slakter has played a major role in the discovery, development and commercialization of treatments for age-related macular degeneration, diabetic retinopathy, retinal vascular disease, central serous chorioretinopathy and other retinal diseases. He has provided critical assistance in the design of clinical trials at all stages of development, and has participated in numerous meetings with the FDA. Dr. Slakter has published more than 85 papers and book chapters.
About Ohr Pharmaceutical Inc.
Ohr Pharmaceutical Inc. (OTCBB: OHRP) (www.ohrpharmaceutical.com) is a pharmaceutical company dedicated to the clinical development of new drugs for underserved therapeutic needs in large and growing markets. The company is focused on two lead compounds: Squalamine eye drops for the treatment of the wet form of age-related macular degeneration, and OHR/AVR118 for the treatment of cancer cachexia, currently being investigated in a Phase II trial.
About Squalamine
Squalamine is a small molecule anti-angiogenic with a novel intracellular mechanism of action, that counteracts not only Vascular Endothelial Growth Factor ("VEGF") but also other angiogenic growth factors such as Platelet Derived Growth Factor ("PDGF") with high potency at nanomolar concentrations. Recent clinical evidence has shown PDGF to be an additional key target for the treatment of wet-AMD. Using the intravenous formulation in over 250 patients in Phase 1 and Phase 2 trials for the treatment of wet-AMD, Squalamine demonstrated favorable biologic effect and maintained and improved visual acuity outcomes, with both early and advanced lesions responding. The previous IV formulation had been awarded fast track status and a Special Protocol Assessment for a phase III registration study from the U.S. Food and Drug Administration ("FDA"). Ohr Pharmaceutical has developed a novel eye drop formulation of squalamine for the treatment of wet-AMD designed for self-administration which may provide several potential advantages over the FDA approved current standards of care, Roche/Genentech's Lucentis® and Regeneron's Eylea®, which require intravitreal injections directly into the eye. Preclinical testing has demonstrated that the eye drop formulation is both safe to ocular tissues and achieves in excess of target anti-angiogenic concentrations in the tissues of the back of the eye.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made only as the date thereof, and Ohr Pharmaceutical undertakes no obligation to update or revise the forward-looking statement whether as a result of new information, future events or otherwise. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the future success of our scientific studies, our ability to successfully develop products, rapid technological change in our markets, changes in demand for our future products, legislative, regulatory and competitive developments, the financial resources available to us, and general economic conditions. For example, there can be no assurance that Ohr will be able to sustain operations for expected periods. Shareholders and prospective investors are cautioned that no assurance of the efficacy of pharmaceutical products can be claimed or assured until final testing; and no assurance or warranty can be made that the FDA or Health Canada will approve final testing or marketing of any pharmaceutical product. Ohr's most recent Annual Report and subsequent Quarterly Reports discuss some of the important risk factors that may affect our business, results of operations and financial condition. We disclaim any intent to revise or update publicly any forward-looking statements for any reason.
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