Opexa Announces Top-Line Results from Phase IIb Trial

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Opexa Announces Top-Line Results from Phase IIb Trial

Postby flipflopper » Fri Sep 19, 2008 12:21 pm

http://www.earthtimes.org/articles/show ... 8638.shtml





MONTREAL - (Business Wire) Opexa Therapeutics, Inc. (NASDAQ:OPXA), a company dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes, today announced top-line data from the company’s Phase IIb TERMS (Tovaxin® for Early Relapsing Multiple Sclerosis) study. Top-line results from the study demonstrated a positive trend in the reduction in annualized relapse rate (ARR) for patients treated with Tovaxin as compared to placebo. However, this finding did not achieve statistical significance. In addition, the study did not achieve statistical significance with its primary endpoint, the cumulative number of gadolinium-enhanced brain lesions.

Top-line results from the study showed that Tovaxin-treated patients experienced an ARR of 0.214 as compared to 0.339 for placebo-treated patients. Despite the low relapse rate in the placebo arm, this still represented a 37 percent decrease in ARR for Tovaxin as compared to placebo in the general population. Additionally, in the group of patients who had an ARR > 1 at study entry, Tovaxin demonstrated a 55 percent reduction in ARR as compared to placebo.

The study also demonstrated that Tovaxin was safe and well tolerated with no serious adverse events related to treatment. The most common adverse event related to Tovaxin was mild injection site reaction. Opexa believes that this favorable safety profile may be an important advantage as patient compliance represents a significant challenge due to serious side effects associated with many currently available MS treatments.

It is important to note that initial review of data revealed that patients in the study’s Tovaxin arm, on average had a substantially greater number of MRI brain lesions and corresponding lesion volumes at baseline compared to the average number of MRI brain lesions and lesion volumes per patient in the placebo group. The company believes that this unexpected imbalance may have contributed to the study not achieving its primary and secondary endpoints as patients in the Tovaxin arm began the study with greater disease burden and increased severity of disease.

“The annualized relapse rate of 0.214 seen in the Tovaxin treatment arm is on par with the lowest relapse rates observed with currently available MS treatments which range from 0.2 to 0.9. This rate is also consistent with ARRs that we have seen in the Tovaxin treatment arms in each of the three previously conducted Tovaxin clinical studies,” stated Neil K. Warma, president and chief executive officer of Opexa. “Findings further showed Tovaxin to possess an impressive safety profile with no serious adverse events related to treatment. This level of safety and tolerability addresses a critical unmet need for MS patients. We believe that these positive ARR results combined with an excellent safety profile and convenient dosing place Tovaxin in a very favorable position for continued development as an innovative MS therapy.”

Top-line data from the TERMS study were presented today by Edward J. Fox, M.D., Ph.D., director of the Multiple Sclerosis Clinic of Central Texas and the study’s principal investigator, at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada.

“Multiple sclerosis is a disease that affects individual patients in distinctly different ways, highlighting the desire for a safe, effective and patient-specific therapy such as Tovaxin. With this in mind, we are pleased with the positive efficacy trend and excellent safety results witnessed in the TERMS study,” stated Dr. Fox. “The Tovaxin-induced reduction in ARR is particularly exciting as it suggests a reduction of clinical activity associated with MS. These study results are encouraging and supportive of further analysis of Tovaxin.”

About the TERMS Study

The TERMS study was a Phase IIb multi-center, randomized, double blind, placebo-controlled trial in 150 patients with Relapsing-Remitting Multiple Sclerosis or high risk Clinically Isolated Syndrome (CIS). The study involved 2:1 randomization with 100 patients receiving Tovaxin and 50 receiving placebo. According to the study protocol, patients received a total of five subcutaneous injections at weeks 0, 4, 8, 12 and 24. The primary efficacy endpoint of the TERMS trial was the cumulative number of gadolinium-enhanced brain lesions (CELs) using MRI scans summed over weeks 28, 36, 44 and 52. The trial’s secondary efficacy endpoints included annualized relapse rate (ARR), new CELs at weeks 28 through 52 and T2-weighted lesion volume compared to baseline.

Top-line data from the TERMS trial is as follows:

ARR for Tovaxin-treated patients was 0.214 as compared to 0.339 for placebo-treated patients. Consistent with ARRs seen in previously conducted clinical trials for Tovaxin, this result is at the lower end of the spectrum of documented relapse rates demonstrated in controlled two-year clinical studies of currently marketed products (range from 0.2 to 0.9).
For patients who had an ARR > 1 in the year prior to the study, Tovaxin demonstrated a 55 percent reduction in ARR as compared to placebo.
Tovaxin was safe and well tolerated with no serious adverse events related to Tovaxin treatment. The most common adverse event was injection site irritation.
Only 18 patients (12 percent) withdrew from the study prior to completion. The dropout percentage was identical for the Tovaxin and placebo arms of the study, providing further evidence of Tovaxin’s excellent safety and tolerability.
“We are especially pleased with the TERMS study’s ARR results, as this represents the most common efficacy endpoint evaluated by the FDA when approving MS therapeutics. Opexa expects that ARR will serve as the primary endpoint in any pivotal Phase III Tovaxin study,” commented Mr. Warma.

Opexa intends to complete a comprehensive analysis of all data from the TERMS study over the next several months. Based on the TERMS study results, Opexa expects to conduct a Phase II close-out meeting with the United States Food and Drug Administration during the first half of 2009. This meeting, along with the comprehensive results of the TERMS study, will provide important guidance as Opexa plans to advance Tovaxin into Phase III development.

Additionally, Opexa is conducting a one-year, open-label extension trial of the TERMS study called OLETERMS. Approximately 90 percent of patients in the TERMS study have elected to enroll in the OLETERMS trial.

Investigator Q&A Session

Opexa will host an investigator Q&A session following the conclusion of today’s programs at the World Congress on Treatment and Research in Multiple Sclerosis. A live webcast of the Q&A session will be available on Opexa’s web site at www.opexatherapeutics.com beginning at 6:00pm Eastern. The webcast will be archived until October 19, 2008.

Conference Call and Webcast

Opexa will host a conference call and webcast with company management to discuss the Phase IIb TERMS data and provide a corporate update on Monday, September 22, 2008, at 8:30 a.m. Eastern. The conference call can be accessed by dialing 800-230-1074 from the U.S. and 612-332-0228 internationally. Additionally a live webcast of the call will be available on Opexa’s web site at www.opexatherapeutics.com. The webcast will be archived until October 22, 2008.

A replay of the call can be accessed until September 29, 2008 at 11:59 p.m., by dialing 800-475-6701 from the U.S. and 320-365-3844 internationally, and entering the following access code: 960761.

About Tovaxin

Tovaxin is developed using Opexa’s proprietary method for the production of patient-specific T-cell vaccines. To produce the Tovaxin vaccine, Opexa isolates disease-causing T-cells from blood taken from an MS patient and expands them in the laboratory to create an appropriate therapeutic dose. The attenuated T-cells, which comprise the Tovaxin vaccine, are reintroduced into the patient via subcutaneous injection to trigger a therapeutic immune system response. Tovaxin is manufactured in Opexa’s in-house cGMP facility.

Opexa believes that Tovaxin may possess the following competitive advantages:

Efficacy – Clinical trials conducted to date demonstrate that Tovaxin may result in a reduction in ARR (a key measure of MS treatment effectiveness) for patients with Clinically Isolated Syndrome (CIS), Relapsing-Remitting MS (RRMS) and Secondary-Progressive MS (SPMS) patients comparable to currently available MS therapeutics.
Safety and Tolerability – Tovaxin treatment selectively targets and depletes the pathogenic T-cell population. It is not a general immune suppressant and accordingly, is not associated with the serious side effects seen with those MS treatments that function by systemically suppressing the immune system. In clinical trials conducted to date, including the 150-patient Phase IIb study, there have been no serious adverse events associated with Tovaxin treatment.
Improved Compliance – In clinical trials, Tovaxin is administered only five times per year. This patient-friendly treatment regimen may provide significant compliance benefits compared to currently available MS treatments (at least once per month and, in some cases, as frequently as every day).
Customized Therapy – Using the company’s proprietary Epitope Analysis Assay (EAA) to profile an individual’s disease profile, Opexa can continually customize treatments to specifically target an individual’s disease progression and/or modification.
About Opexa Therapeutics

Opexa Therapeutics is a biotechnology company dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases. The company’s leading therapies currently in development have the potential to address significant unmet medical needs in several large patient populations including multiple sclerosis (MS) and diabetes. The company's lead product is Tovaxin, a T-cell therapy for MS which recently completed a Phase IIb trial. The company also holds an exclusive worldwide license for adult multi-potent stem cells derived from mononuclear cells of peripheral blood. The technology provides means to differentiate these stem cells into other tissue types such as pancreatic islets. By using an individual’s own cells, this approach may minimize threat of treatment rejection. This technology serves as the basis for Opexa’s preclinical diabetes program, which is focused on the generation of insulin-secreting pancreatic-like cells. For more information visit the Opexa Therapeutics website at www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of collaborative relationships, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. We assume no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

Opexa Therapeutics, Inc.
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Postby flipflopper » Fri Sep 19, 2008 12:43 pm

I’m very disappointed in these results :(
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Postby TWG » Fri Sep 19, 2008 1:02 pm

I think we where all expecting 'Tim' results, or at least results comparable to previous studies. But at least it shows it is a little better than the CRAB's. :?

Reality check....
Diagnosed with MS in Feb. 14 2000! Was a Tovaxin guinea pig.
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Postby dignan » Fri Sep 19, 2008 1:15 pm

This is the key part of the news release:

However, this finding did not achieve statistical significance. In addition, the study did not achieve statistical significance with its primary endpoint, the cumulative number of gadolinium-enhanced brain lesions.


Opexa doesn't have the money to pursue this on their own. Who is going to invest the 10s (or 100s) of millions in a phase 3 trial based results that did not achieve statistical significance?
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Postby flipflopper » Fri Sep 19, 2008 1:24 pm

That’s very true dignan. Also, who would want to participate in the phase III trial and submit themselves to all the trial visits (with many tests, MRIs, blood draws etc..) when the phase IIb results didn’t reach statistical significance? We have more effective medications on the market that are currently approved.
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Postby Lyon » Fri Sep 19, 2008 1:43 pm

..
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???

Postby notasperfectasyou » Fri Sep 19, 2008 1:44 pm

I lost on the stock today.

They don't have money to go forward in the normal sort of way, but they have that new relationship with the MS group to work with. Could Opexa become a non-profit organization? Don't be shocked if it happens, it's possible.

I recall the very positive results in the last trial, something like 90% benefit compated to placebo. Someone's going to have to get cracking on "why should we continue to work this concept". The financial community has spoken, the stock is just about worthless.

What will happen to the participants who are on Tovaxin and feel that it is benefiting them and want to continue? Has anyone been told that they can continue the therapy?

The value of the MRI has been an issue for a long time. Kim's going for her MRI in January on her "experimental" therapy - wish us luck. So as I see it, if you get a good MRI, you beleive in it and if you don't, you question it? I don't know what we'll do post MRI, but I wonder about other trials like Fampridine where the measured outcome is walking speed!

Yup, lots of 25-foot walks. Fampridine. I wonder what the outcome would have been in Opexa had measured on this basis?

I am sad about this, seeming, dead end. I lost money, but I would have gladly paid more for statistical significance. Spinning and Lost, Ken
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Postby patrickm » Fri Sep 19, 2008 2:44 pm

<sigh>
Well, the positive, and the only thing they can spin and how they reframed the talk, is that it works just as well as the CRAB drugs but is a whole lot less of a pain in the ass.

But surely not what we were all hoping and not anywhere near the 90+% that they showed in Phase I.

I don't know how they'll continue but if the above has real value, then the company can be had for a song.

Notasperfectasyou, I'm really sorry you lost money on the deal. I'm trying to tell myself that I didn't lose 18 months of my MS life.

It has been as horrendous of a two week stretch as I have ever had without somebody dying on my, and this really sticks a fork in it.

Not what I wanted to see after coming home from a 12-6 pasting of the Cubs.

What next? Somebody gonna run over my new puppy playing in the road?

Don't have a new puppy, really...

JFC.

"Opexa—at least we're not them!"
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Postby notasperfectasyou » Fri Sep 19, 2008 2:55 pm

Don't get me wrong, I'm not so worried about the money - I genuinely had hoped for something wonderful here. The money just compounds the disappointment.

Also, I'm not so certain that the theory and therapy is bad. Unlike the ABX that Kim is doing, Tovaxin participants do not know if they had the therapy or not. There's also not so much immediate impact on your body to report, was there? The concept might be great, but need tweaking. Problem is funding. Ken
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Postby patrickm » Fri Sep 19, 2008 3:01 pm

yeah, big problem. BIG problem. They rolled all the dice, the whole barrel of monkeys AND the big enchilada on these results and came up snake eyes.

Am awaiting a call from my doctor after he's done for the day and had time to digest. Will post, but not sure what he's gonna be able to tell me that's not in the press release. Gonna go listen to their conference call.
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Postby Lyon » Fri Sep 19, 2008 4:04 pm

..
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Postby flipflopper » Fri Sep 19, 2008 4:29 pm

Lyon wrote:Sounds to me that Opexa's main point was that it's kind of hard to prove a marked benefit during a trial involving such early and mild MS, and I have to admit that that was a concern of mine from the very beginning.



That is a good point Bob. They also mentioned in the webcast that perhaps the dosing schedules should have been different to obtain better results. Maybe getting a vaccine every month would have produced better results? Opexa needs more time to look at the data (and to look at other measures that have not been analyzed yet). But, I am no longer holding my breath. For now, I see nothing that excites me too much about this vaccine.
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Postby Lyon » Fri Sep 19, 2008 4:47 pm

..
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rube goldberg device

Postby notasperfectasyou » Fri Sep 19, 2008 5:06 pm

I bet when MS is finally figured out, it's going to look like a Rube Goldberd Device. Tovaxin just might be effecting one component, but perhaps not enough component's to bring the machine down. Just thinking out loud.

Lyon, my prayers go out to you and your wife this weekend. Let's all wake up Monday and hope to have a fresh and positive view of the world. Ken
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Postby TWG » Fri Sep 19, 2008 5:16 pm

The relapse rate was as good as any of the best CRAB drugs. They mentioned nothing about EDSS scores. So the way I see it, it's at the minimum as good as giving my self a shot on a regular basis. I can handle the bi-monthly commutes to avoid that! It's free to boot.

I am bummed about the report, and hopefully Opexa does not go bankrupt. My real vaccine is stuck in Houston, TX due to hurricane Ike, but am looking forward to receiving it.

Opexa is experiencing some real hardships right now. If the hurricane wasn't bad enough, this report might have finished them off. Hopefully the company survives this 'perfect storm' of events because, like I stated previously, the relapse is as good as any other injectables, and I like the idea that it requires less over-all trouble on my part.

I was hoping for a "Tim" like ending to this story, but it is what it is. I am looking forward to receiving my first vaccine.

I am TWG, and I approve this message.
Diagnosed with MS in Feb. 14 2000! Was a Tovaxin guinea pig.
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