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These details are what I think the webinar was supposed to explain. No one says they are having a webinar to announce a manufacturing facility. Based on previous presentations, I think it was to give more information on the start of the trials. I am thinking we will find out soon.
It really depends on the partnership. If the BP lets them use the CI, and that is all, that is one thing. If they give an upfront payment with milestones that is another thing. I would think the partnership would have to be with Roche / Genentech being how they keep mentioning them in their presentations. My thoughts are that they were supposed to have a webinar to announce the start of the trials but got sidetracked with the filings. I think we would have to get an update by the end of the month if not by the end of the week but that is JMO :)
It was from last month and I do not have a link. I asked a question and this is what I was told.
From last month:
I think TPizz is going to see them next week at a conference in FL. Maybe he can ask when the trial is gong to start :)
I was told:
Yes, this is what I was told when I asked about it right before the Roth conference last month.
Ovarian 40 patient open label and TNBC 80 patient open label
Hopefully we get an update next week.
My thoughts are we were supposed to get much anticipated news last week. I think everyone would agree last weeks news was not much anticipated. At the same time they say they have to do the filing over again. I don't think that's a Coincidence. I think the partner wanted this fixed before they announced a partnership.
I know but I hope they got to the last "I" and "T" this time.
TPIV- will be giving an update soon on corporate developments. The trials are supposed to start by the end of this month.
Interesting article about a buyout from last year
Flexus goes from zero to $1.25 billion in 17 months
by Dan Primack @danprimack FEBRUARY 24, 2015, 12:27 PM EST
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Kleiner Perkins could earn more than a 25x return on a biotech investment that is less than two years old.
Bristol-Myers Squibb Co. BMY 3.70% yesterday agreed to acquire Flexus Biosciences Inc., a San Carlos, Calif.-based developer of anti-cancer therapeutics. The deal could be valued at upwards of $1.25 billion, including $800 million upfront.
A few notes on what is the year’s most remarkable VC-backed exit so far:
1. Flexus is less than two years old: The company was originally incubated by Kleiner Perkins Caufield & Byers, whose lead healthcare partner (Beth Seidenberg) had worked with the entrepreneurs (Terry Rosen and Juan Jaen) years ago at Amgen AMGN 4.29% . The basic idea was to take an orthogonal approach to cancer immunotherapy – working on molecules that would be additive to what already was on the market from Bristol-Myers and Merck MRK 3.07% , rather than creating a me-too sort of therapy.
2. Very little investment: Flexus raised only $38 million over two rounds of funding, from KPCB, The Column Group and Celgene CELG 4.14% . The original investment came in October 2013. Seidenberg declined to disclose Series A or Series B valuations, but a separate source tells me that the firm’s stake in Flexus is around 30%. Or, put another way, a $375 million return on what is was less than $20 million in total investment. Remember when biotech startups were expensive and IT startups were cheap? When’s the last time an IT startup raised less than $40 million and was sold for more than $1 billion?
3. Did I say it was young? Flexus does not have a single drug candidate in clinical trials yet. That’s where the earn-outs come in, although Seidenberg says she is very “comfortable” with them and expects that they will be met within three years.
4. It’s only a partial exit: Bristol-Myers is technically buying all of Flexus, except it isn’t really. Instead, the $1.25 billion is for the company’s Flexus’ IDO/TDO discovery program, while the entire Flexus management team and its two other existing drug programs (both pre-clinical) will be spun out into a new, independent company. Expect the existing venture capital backers to soon fund a Series A round for NewCo.
5. What’s it do? I asked Seidenberg to explain the appeal of what Flexus is working on, in layman’s terms. Here is what she said: “We know that when cancer arises, in addition to cells multiplying and having genetic abnormalities, the other important part of either controlling the cancer of letting it get out of control is the immune system. When someone has an infection, for example, the immune system goes after it to clear it. When cancer occurs, those are the abnormal cells and the immune system has to clear it. But cancer is very smart and creates substances that dampen the immune system. This drug will revitalize the immune system so that it can do its job and help clear the cells.”
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http://fortune.com/2015/02/24/flexus-goes-from-zero-to-1-25-billion-in-17-months/
Great job by TPIV. They have ODD today and an update tomorrow and still have partnership news coming soon.
On the conference call they had last month, and the end they said they expect to "obtain" ODD this year. That would mean they have to file in time to obtain that status in 2015, which would be last month. Also, that email said both applications are in process which would make me think they have already been filed. TPIV may just want to send a press release for the approval of both applications. :)
Did anyone notice how they keep mentioning ongoing talks with big biotech about partnerships. That alone would be huge.
Pfizer mulls $100 bln bid for AstraZeneca - report
LONDON, April 20 (Reuters) - U.S. pharmaceutical giant Pfizer has approached British rival AstraZeneca to propose a 60 billion pound ($101 billion) takeover, the Sunday Times reported on Sunday.
The paper said, citing senior investment bankers and industry sources, that informal conversations about a deal had taken place between the two firms but that no talks were currently under way after AstraZeneca resisted the approach.
AstraZeneca is Britain's second-biggest pharmaceuticals group with a current market valuation of around $80 billion, compared with Pfizer, which is valued at $193 billion, according to Thomson Reuters data.
Earnings at AstraZeneca fell 6 percent in the fourth quarter of 2013, and the drugmaker has said it expects them to keep falling in 2014 as generic competition to Nexium, its popular heartburn and ulcer drug, takes a big bite out of U.S. profits from late May.
AstraZeneca has suffered a dry period in drug discovery in recent years and badly needs to find new medicines to replace blockbusters such as Nexium and Crestor, a treatment for high cholesterol that will lose patent protection in a few years.
Pfizer is facing similar issues with patent expirations on top-selling drugs such as Viagra and anti-cholesterol treatment Lipitor.
Pfizer's last big acquisition was in 2009, when it bought U.S. rival Wyeth for $68 billion.
http://finance.yahoo.com/news/pfizer-mulls-100-bln-bid-104422824.html
MNKD resumed trading in 9's now
PRAN - Prana Biotechnology announces top line results of Phase 2 IMAGINE trial of PBT2 in Alzheimer’s disease
MARCH 31, 2014
MELBOURNE, 31st March, 2014: Prana Biotechnology (ASX:PBT/NASDAQ:PRAN) has today released the top line results of the 12-month Phase II Imaging trial in Alzheimer’s Disease (“IMAGINE” Trial), based on draft results.
Prana’s PBT2 did not meet its primary endpoint of a statistically significant reduction in the levels of beta-amyloid plaques in the brains of prodromal/mild Alzheimer’s disease patients, as measured using PiB-PET Standardized Uptake Value Ratio (SUVR). Whilst there was a reduction in the overall levels of the PiB PET signal in patients treated with PBT2, the results were confounded by an atypical reduction of levels of the PiB PET signal in the placebo group as well.
Commenting on the result, Geoffrey Kempler, CEO of Prana Biotechnology said: “This is the first time that Prana has looked at PiB-PET in a study with PBT2 to measure its effect on insoluble amyloid plaques. In our previous Alzheimer’s study (EURO)1, we looked at levels of unaggregated soluble Abeta peptides in spinal fluid, and they were significantly reduced with PBT2 treatment. So in the IMAGINE trial we looked for an impact on the insoluble plaques as well, but did not see it differ significantly from the placebo.”
“It is possible the result may point to PBT2 targeting soluble species of Abeta including toxic oligomers rather than plaques. Abeta oligomers are not visible in the PiB-PET scans which can only detect amyloid plaques. Alternatively, what we are seeing is simply the result of an inconclusive imaging readout in a small sample size with 42 participants (15 on Placebo, 27 on PBT2)”.
No improvement was observed on the secondary endpoints of brain metabolic activity, cognition and function; however there was a trend towards preserving hippocampal brain volume in the PBT2 group. Specifically, there was less atrophy in those patients treated with PBT2 relative to placebo, 2.6% and 4.0%, respectively. This is consistent with published measures of atrophy in AD patients versus healthy controls2 of 4.7% and 1.4%, respectively. The company is tracking measures of brain volume and cognition in the current 12 month extension study that will be completed at the end of the year. Further analysis of the results is ongoing.
Importantly, PBT2 was shown to be safe and very well tolerated over the 52 weeks. The adverse event profile was equivalent between placebo and treated groups. Forty of the 42 enrolled participants (95%) completed the 52 week treatment period.
Mr Kempler concluded: “Whilst not meeting all of our hopes, this result does not deter us from the future development of PBT2, a safe and well tolerated drug candidate for Alzheimer’s disease. Our scientists and those from other institutes have developed a strong body of evidence for the efficacy of PBT2 in Alzheimer’s disease. The suggestion of beneficial effect of PBT2 on brain volumes first seen in the Reach2HD Huntington disease trial and now in this Alzheimer’s disease IMAGINE trial is intriguing. We are consulting with experts in the field to further assess these results and to consider how best to progress PBT2 in Alzheimer’s disease. Indeed, the IMAGINE Extension trial is continuing, and data from this trial is likely to inform the next steps for an AD program.”
Prana is proceeding with its plans toward a confirmatory study for Huntington disease. Based on Prana’s previous discussion with the US Food and Drug Administration, the data on safety and tolerability of PBT2 in Alzheimer’s disease will support the future clinical development and, ultimately, a New Drug Application in Huntington disease.
Prana has a cash position of AU$25.4 million as at 31 March 2014.
http://pranabio.com/news/prana-biotechnology-announces-top-line-results-phase-2-imagine-trial-pbt2-alzheimers-disease#.UzlCeM1UVC9
PRAN- Prana Biotechnology - A Gamble Worth Taking For Both Long- And Short-Term Investors
http://seekingalpha.com/article/2100583-prana-biotechnology-a-gamble-worth-taking-for-both-long-and-short-term-investors?source=yahoo
CPRX- Catalyst Pharmaceutical Partners Announces Fourth Quarter and Year-End 2013 Financial Results and Provides Corporate Update
http://finance.yahoo.com/news/catalyst-pharmaceutical-partners-announces-fourth-120300776.html
PRAN- Fidelity adds shares. They now own 1 million shares as of yesterday.
http://data.cnbc.com/quotes/pran/tab/8
SYN - 2.73 down from HOD of 3.00
AMRN sues Omthera Pharmaceuticals, Inc., a Delaware corporation, and its parent company, AstraZeneca Pharmaceuticals LP.
http://biz.yahoo.com/e/140304/amrn8-k.html
AVNR- Traders bet on comeback in Avanir
By David Russell (david.russell@optionmonster.com)
February 26, 2014 1:21 PM
Avanir Pharmaceuticals got hammered late last year, but traders are betting on a comeback.
optionMONSTER's Heat Seeker monitoring program detected the purchase of roughly 8,000 April 4 calls, most of which priced for $0.73. A similar number of March 4 calls were sold around the same time for $0.42 in volume below the previous open interest in that strike.
It appears that an investor previously owned the March contracts and is rolling the position forward in time. Making the adjustment cost $0.31 and gives an extra month for the stock to rally. The trade also provides protection from the quickening pace of time decay that will occur in coming weeks.
Long calls lock in the price where a stock can be purchased, so they can be owned in lieu of shares at a much lower price. Capital can be swapped from one contract to another over time, significantly reducing potential losses in the event of a drop. (See our Education section)
AVNR is up 8.85 percent to $4.18 in afternoon trading. The drug developer plunged from over $4 to under $3 in December after reporting a wider-than-expected quarterly loss. The stock has been rebounding since, partly supported by strong sales of the company's Nuedexta drug earlier this month. Today's trade reflects a belief it will continue pushing higher over the intermediate term.
Total option volume is 20 times greater than average in the session, according to the Heat Seeker. Calls outnumber puts by a bullish 28-to-1 ratio.
http://finance.yahoo.com/news/traders-bet-comeback-avanir-173752587.html
Amarin Sues FDA Over Exclusivity for Fish Oil Pill
http://www.bloomberg.com/news/2014-02-28/amarin-sues-fda-over-exclusivity-for-fish-oil-pill-1-.html?cmpid=yhoo
Teva Climbs Above $50 Per Share as Buyouts Lift Earnings Outlook
Teva Pharmaceutical Industries Ltd. (TEVA), the world’s largest maker of generic drugs, rallied above $50 per share for the first time since 2011 as speculation mounted that consolidation among generic drug companies will bolster earnings.
Shares of the Petach-Tikva, Israel-based company advanced 2.4 percent to $50.48 at 1:59 p.m. in New York, the highest since June 2011. They rose 13 percent in February, heading for the best monthly performance since January 2007.
Mylan Inc., the biggest U.S. generic-drug maker, said it may make a large acquisition this year, Chief Executive Officer Heather Bresch said in an interview yesterday. Teva, the largest stock holding in George Soros’s fund, appointed a new chief executive officer and pledged to overhaul its board and cut costs last month as cheaper versions of its multiple sclerosis drug threaten to cut into its market share. Dublin-based Actavis Plc on Feb. 18 agreed to buy brand-drug maker Forest Laboratories Inc. for $25 billion.
“There is the expectation that the new management team will introduce a new business plan in the next three months, which will outline an acquisition,” Ronny Gal, a New York-based analyst at Sanford C. Bernstein & Co who has a buy rating on the stock, said by phone today. “Investors really like acquisitions in this sector. They think that an acquisition will boost Teva’s earnings and help the company grow quickly.”
To contact the reporters on this story: Gabrielle Coppola in New York at gcoppola@bloomberg.net; Elena Popina in New York at epopina@bloomberg.net
http://www.bloomberg.com/news/2014-02-28/teva-climbs-above-50-per-share-as-buyouts-lift-earnings-outlook.html?cmpid=yhoo
SYN- Synthetic Biologics' Board of Directors Accepts Resignation of Long-Standing Founder and Board Member
Synthetic Biologics, Inc.
1 hour ago
ROCKVILLE, Md., Feb. 26, 2014 /PRNewswire/ -- Synthetic Biologics, Inc. (NYSE MKT: SYN), a developer of anti-infective biologic and drug candidates targeting specific pathogens that cause serious infections and other diseases, announced today that Founder, Board member and subsidiary CEO and President, Steve H. Kanzer, has resigned from the Company, effective immediately, to return his focus to his venture capital and investment banking firms on a full-time basis.
http://finance.yahoo.com/news/synthetic-biologics-board-directors-accepts-220100657.html
SNSS- Sunesis Announces Proposed Public Offering of Common Stock and Warrants
http://finance.yahoo.com/news/sunesis-announces-proposed-public-offering-211351354.html
SNSS - Sunesis risk/reward attractive at current levels, says Leerink
10 hours ago
Leerink views the risk/reward for shares of Sunesis as very attractive at current levels and reiterates an Outperform rating on the stock. The firm upped its price target for shares to $14 from $10.
http://finance.yahoo.com/news/sunesis-risk-reward-attractive-current-124902593.html
DARA BioSciences' KRN5500 Receives Orphan Drug Designation From FDA
Novel Non-Opioid Therapeutic in Phase 2 to Treat Chronic Chemotherapy-Induced Peripheral Neuropathy; Existing FDA Fast Track Designation
4 minutes ago
RALEIGH, NC--(Marketwired - February 25, 2014) - DARA BioSciences, Inc. (DARA), an oncology supportive care specialty pharmaceutical company dedicated to providing healthcare professionals a synergistic portfolio of medicines to help cancer patients adhere to their therapy and manage side effects arising from their cancer treatments, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KRN5500 for the parenteral treatment of painful, chronic, chemotherapy-induced peripheral neuropathy that is refractory to conventional analgesics. KRN5500 is a novel, non-opioid, non-narcotic intravenous product currently in Phase 2 clinical development.
"We are absolutely thrilled to receive Orphan Drug Designation, and appreciate the hard work of the FDA's Office of Orphan Product Development over the many months in reviewing and ultimately approving the KRN5500 application for orphan designation. We believe this Orphan Drug Designation will expedite the development of KRN5500 for patients with cancer who suffer from chronic neuropathic pain brought on by potent chemotherapeutic agents," said David J. Drutz, M.D., Chief Executive Officer and Chief Medical Officer of DARA BioSciences. "KRN5500, is a candidate to treat chronic neuropathic pain induced by chemotherapy, and fits perfectly into our corporate strategy of providing clinicians and patients with access to a synergistic portfolio of oncology supportive care products."
http://finance.yahoo.com/news/dara-biosciences-krn5500-receives-orphan-130000676.html
ITMN- InterMune Reports Phase 3 ASCEND Trial Results of Pirfenidone in Idiopathic Pulmonary Fibrosis (IPF)
- Study Meets Primary and Both Key Secondary Endpoints -
- Company to Conduct Conference Call and Webcast Today at 8:00 a.m. EST -
InterMune, Inc.
23 minutes ago
BRISBANE, Calif., Feb. 25, 2014 /PRNewswire/ -- InterMune, Inc. (ITMN) today announced that top-line data from ASCEND, a Phase 3 trial evaluating pirfenidone in patients with idiopathic pulmonary fibrosis (IPF), demonstrated that pirfenidone significantly reduced IPF disease progression as measured by change in percent predicted forced vital capacity (FVC) from Baseline to Week 52 (rank ANCOVA p
(Logo: http://photos.prnewswire.com/prnh/20120827/SF62570LOGO)
"We are pleased to report these top-line ASCEND Phase 3 results," said Dan Welch, Chairman, Chief Executive Officer and President of InterMune. "Based on the strength of the ASCEND results, InterMune is preparing a resubmission of our New Drug Application for pirfenidone to the U.S. Food and Drug Administration (FDA), which we expect to submit by early third quarter of this year. We would like to thank our collaborators, patients and their families for their participation in ASCEND and their contributions to IPF research."
http://finance.yahoo.com/news/intermune-reports-phase-3-ascend-120000665.html
True- I just wonder how the news coming out before the company announced it will affect the pop. I'd think it will pop in the AM after the PR and I hear DCTH ODD was known a few days before they issued a PR and it still doubled. It also looks like DARA popped AH after XOMA confirmed their ODD with the same 2/21 date and confirmed this story and link.
http://www.theflyonthewall.com/permalinks/entry.php/XOMAid1968324/XOMA-XOMA-treatment-of-pyoderma-gangrenosum-granted-FDA-orphan-status
http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm?Index_Number=419113
Exactly- and It looks like XOMA has the same date of 2/21 but they released a PR tonight.
http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm?Index_Number=419113
http://finance.yahoo.com/news/xoma-receives-orphan-drug-designation-232030234.html
Looks like the FDA website is ahead of the press releases
XOMA Receives Orphan Drug Designation From U.S. FDA for Pyoderma Gangrenosum
BERKELEY, Calif., Feb. 24, 2014 (GLOBE NEWSWIRE) -- XOMA Corporation (XOMA), a leader in the discovery and development of therapeutic antibodies, announced today gevokizumab, the Company's IL-1 beta modulating antibody, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for the treatment of pyoderma gangrenosum (PG).
http://finance.yahoo.com/news/xoma-receives-orphan-drug-designation-232030234.html
DARA - ODD from FDA website as well
http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm?Index_Number=385312
PRAN- Positive Phase 2 Results For Prana Biotechnology In Huntington's Disease: Behind The Smoke And Mirrors
Feb. 21, 2014 12:45 PM ET | 8 comments | About: PRAN
Disclosure: I am long PRAN. (More...)
This article was first released only to PRO subscribers. Learn More
Executive summary
PBT2 is safe.
One serious adverse event occurred secondary to PBT2. A worsening of Huntington's disease symptoms upon its cessation.
PBT2 may improve cognition.
Efficacy of PBT2 required for approval in Huntington's disease is very low.
Approval of PBT2 for Huntington's disease will speed up approval for the larger Alzheimer's dementia indication.
IMAGINE trial results for Alzheimer's dementia due to report by the end of March significantly de-risked by the REACH2HD trial.
Prana Biotechnology (PRAN) has recently released the results of its REACH2HD trial, which it has described as a success.
I have previously written about PBT2 more generally, whereas the focus of this article is to address some issues people have raised about the trial.
The REACH2HD trial should have recruited more patients.
Biotech investors may be more familiar with other clinical trials, such as cardiology registration trials. The number of patients recruited for these can be in the range of 10-20k. To recruit that many patients for Huntington's disease, you would be testing 12%-25% of people with the disease.
PRAN conducted the REACH2HD trial because it could not afford to fund a large Phase 3 trial in Alzheimer's dementia. Prior to the run-up in the share price, its market cap was <$100m, and targeting Huntington's disease as an orphan drug indication provided an easier way to get PBT2 to market.
Trials in Huntington's disease are small, as there are only about 80,000 patients worldwide with this disease. The registration trial for the only drug approved for Huntington's disease, Tetrabenazine, recruited 74 patients for 12 weeks. REACH2HD, in comparison, recruited 109 patients for 26 weeks.
The REACH2HD trial executive function efficacy was not sufficient.
That the REACH2HD trial showed efficacy in any marker is a significant positive. The fact that it was in the Trail Marking Test Part B, which is a component of the Executive Function Composite z-score, is an added bonus.
PBT2 also improved executive function; the fact that this was also positive in its Phase 2a trial in Alzheimer's dementia is an added kicker. PBT2 did this in only 6 months, in a trial with limited power due to it having 3 arms and only 109 patients. If you are familiar with statistical analysis, you will know that more you divide patient groups up, the harder it is to prove statistical significance.
This also bodes well for the upcoming IMAGINE trial to be reported in March, as it indicates that PBT2 might be working through the same mechanism of action.
Figure 1. Phase 2a results for PBT2 in Alzheimer's Dementia
Figure 2. Results of the Trail Marking Test B in the REACH2HD trial at 26 weeks
PBT2 did not show benefit in motor function.
The REACH2HD trial was always going to be less likely to show improvements in motor function, as they did not specifically look to enroll patients with Huntington's chorea.
They enrolled patients with a total functional capacity of between 6 and 13, inclusive with an average score of 9.2. A score of 7-10 indicates stage 2 disease, and patients at this level are unlikely to have significant motor problems.
Table 1. Demographics of the REACH2HD patients
Figure 3. Components of Total Functional Capacity
Motor problems do not develop rapidly, so with 109 patients the trial would not have been powered to notice the subtle difference in the progression of motor symptoms in only 6 months.
Finally, the REACH2HD trial was a Phase 2 trial. Phase 2 trials are designed to test the safety of the drug in the target population; efficacy markers are there to see which groups the company should target in Phase 3 trials.
On the conference call, management indicated other clinical markers were heading in the right direction but did not achieve statistical significance.
The results were not corrected for multiple analyses.
This is a fairly legitimate point, especially as the more sub-groups you look at, the more likely you are to find something that is significant.
That executive function was significant in both trials, however, makes it less likely that the result was positive due to random error.
Will PBT2 get approved for use in patients with Huntington's disease?
Tetrabenazine is the only FDA-approved drug for Huntington's disease. It improves Huntington's-related chorea, part of the motor symptoms associated with Huntington's disease. It has no benefit in cognition.
The FDA approved it when its main registration trial recruited 84 patients in a trial lasting 12 weeks. That was 7 weeks of dose adjustment, and 5 weeks of maintenance, and follow-up for a week after coming off Tetrabenazine or placebo.
Tetrabenazine reduced chorea, but missed the mark on safety. PBT2, in comparison, improved a marker of executive function, and in terms of safety, was not significantly different to placebo.
The only serious adverse event in the REACH2HD trial occurred in the follow-up period. One patient who had received PBT2 had a significant worsening of his Huntington's disease symptoms after its cessation.
There were five study withdrawals in the Tetrabenazine group and five serious adverse events in four subjects (drowning suicide, complicated fall, restlessness/suicidal ideation, and breast cancer), compared with one withdrawal and no serious adverse events in the placebo group.
The FDA was willing to approve Tetrabenazine because Huntington's disease is such a serious condition and there were no treatments at that time.
PBT2 is targeting the cognitive impairment that patients with Huntington's disease suffer from. There are even subtle changes in people who do not suffer from Huntington's disease but are carriers for the disease.
The FDA will likely not demand a large degree of efficacy for PBT2 in Huntington's disease, as PBT2 appears to be a safe drug and there are still no treatments that improve cognition.
Conclusion
There are many risks when investing in early biotechnology companies, but PRAN has been significantly de-risked after its REACH2HD trial. PBT2 is now the only drug that has shown a statistically significant improvement in executive function for people who suffer from Huntington's disease and Alzheimer's dementia. These results significantly increase the chances that PBT2 will be successful in the IMAGINE trial due to report in March.
At a current market capitalisation of $336 million, I believe that the risk-reward ratio for PRAN would be favourable, as FDA approval for the use of PBT2 in Huntington's disease or Alzheimer's dementia could eventually see PRAN's share price increase 100-fold.
http://seekingalpha.com/article/2039103-positive-phase-2-results-for-prana-biotechnology-in-huntingtons-disease-behind-the-smoke-and-mirrors?source=yahoo
PRAN - New Huntington Disease Drug Is Safe, but Is It Effective?
By Brian Orelli | More Articles | Save For Later
February 19, 2014 | Comments (3)
The press release headline "Prana Announces Successful Phase 2 Results in Huntington Disease Trial" sounds good, but the primary endpoint of the phase 2 trial was safety, not efficacy, so it wasn't that high of a hurdle to jump over.
"There were no substantial differences in adverse events across the two PBT2 dose groups and the placebo group," the press release said. Yee-haw.
Moving on
Because let's face it, the Food and Drug Administration isn't going to approve a drug just because it's safe. It has to do something to help patients.
Prana ran a bunch of efficacy tests on the patients, measuring cognition, motor skills, function, and behavior. Only one test of cognition was statistically significant.
I've heard many people argue that's data mining. And on some level, it is; the more measurements you make, the more likely it is that something will come back as statistically significant, even if it's just an artifact.
But I think investors should give Prana Biotechnology the benefit of the doubt, here. The hypothesis is that PBT2's mechanism of action -- reducing metal mediated toxicity -- can improve cognitive function. It appears the other tests were performed to be thorough rather than because anyone expected an actual improvement. For motor function, for instance, PBT2 doesn't act on dopamine levels, so it's unlikely it would improve motor function.
Source: National Highway Traffic Safety Administration.
The trial results would have had a lot more weight if Prana had listed cognitive function as a co-primary endpoint with safety and left the other tests as secondary endpoints. Of course, if they had, and the cognitive test wasn't statistically significant, the company would have had a hard time justifying moving PBT2 into a phase 3 trial.
Alternating numbers and letters
The Trail Making Test Part B, which showed a statistically significant improvement, requires patients to draw a line from points alternating numbers and letters: 1-A-2-B-3-C, and so on.
Here's how the patients in the different dosing groups did after taking the drug for 26 weeks.
Source: Prana Biotechnology.
I'd feel a lot more confident if the 100 mg dose was closer to half way between the 250 mg dose and the placebo group. While a dose-dependent efficacy would be a good sign, it isn't necessarily a sign that the improvement at 250 mg is an artifact; some drugs have a threshold that needs to be reached before they start working.
More importantly, while the difference was statistically significant, it isn't clear to me if the FDA will see the difference as clinically meaningful. The Trail Making Test Part B is designed to test executive cogitation, the ability to problem solve and be flexible in situations that demand it. The FDA will need to be convinced that the difference translates into a meaningful improvement in Huntington disease patients' lives.
Prana plans to meet with the agency to work out details for a larger phase 3 trial.
A sign of things to come?
Prana is also testing PBT2 in a phase 2 trial in Alzheimer's disease, which is scheduled to read out in March. The hypothesis is that metals, which PBT2 binds up, may be leading to cognitive decline in both diseases. Like the Huntington disease trial, it's a phase 2 proof-of-concept trial, making it hard to handicap.
The good news is, the trial's primary endpoint is a measurement of efficacy rather than just safety, like the Huntington disease trial. The bad news is, the primary endpoint is the level of amyloid plaques in patients' brains, which is a rather indirect measure of efficacy, and it's still hotly debated whether amyloid plaques are a cause or an effect of Alzheimer's disease. Investors should pay more attention to the cognition tests, which will be run as a secondary endpoint.
Good buy?
For the most part, investing in neurodegenerative diseases is a crapshoot.
Not that it stops companies from trying. Merck (NYSE: MRK ) recently moved MK-8931 into a phase 2/3 study last year. Eli Lilly (NYSE: LLY ) is testing solanezumab in a large phase 3 trial despite previous failures of the drug.
I can't really blame companies for proceeding. The potential market is so large, it's not hard to justify paying for the clinical trials even if the chance at success is less than 50%.
The obvious difference between investing in Merck or Eli Lilly and investing in Prana is that the failure of trials by the large pharmas won't kill the company. If you're going to invest in Prana, make sure it's money you can afford to lose. At a market cap under $400 million, there's a lot of upside if PBT2 works in Huntington or Alzheimer's disease, but it doesn't come without a lot of risk.
http://www.fool.com/investing/general/2014/02/19/prana-pran-pbt2-data-in-huntington-disease.aspx