Meet the Company That's Changing the Multiple Sclerosis Treatment Landscape

Here at The Motley Fool we spend a lot of time dissecting companies at the leading edge of the largest investment opportunities. Being a specialist in the Health Care sector, that means I devote a lot of my research to drugs aimed at treating some of the more deadly diseases, such as cancer, heart disease, stroke, and even influenza/pneumonia.

One disease, however, that's very serious, is diagnosed in about 200 people per week in the U.S., and currently affects about 400,000 people in this country, has escaped my attention for far too long. That disease is multiple sclerosis, or MS.

A difficult disease to fight
MS is a chronic and progressive life-long disease that most often shows its first symptoms between the ages of 20 and 50 (30-35 is the most common age range to demonstrate symptoms). It's also a disease that's two-to-three times more common in women than men. Specifically, MS attacks a patient's central nervous system, which is some cases leads to only mild inconveniences, and in more severe cases, can impair a person's ability to walk, talk, eat, and do a number of other basic functions that we often take for granted.

One of the more maddening aspects of MS is that despite its global proliferation no one is exactly sure what triggers the disease. Sure, there are postulations that it's some mixture of genetics and environmental factors considering that North America and Europe are among the most affected regions by the disease, but generally speaking the scientific community is a long way from fully understanding what triggers MS. And not understanding its causes makes it a particularly difficult disease to fight. 

Let's be clear, there are a number of therapies on the market geared at helping stem the progression of MS or possibly even stopping it in its tracks. Intravenous therapies have existed for years, but the emergence of oral therapies such as Novartis' (NYSE: NVS  ) Gilenya and Sanofi's (NYSE: SNY  ) Aubagio, which are used to treat relapsing forms of MS, have given patients a new level of hope and convenience.

Gilenya can be used as both a first and second-line treatment in treating MS, while Aubagio is strictly a second-line therapy. But, these beneficial therapies also comes with noted risks, including a black box warning for Aubagio regarding potential liver problems which can lead to death, and potentially serious cardiovascular problems with Novartis' Gilenya since it can lower a patient's heart rate. In other words, the need for safer and more effective therapies that can be used over the long haul is still readily apparent.

That's where this next company comes in. You might call it a biotech juggernaut – I simply call it the company that's changing the multiple sclerosis treatment landscape from the bottom up. Let me introduce you to Biogen Idec (NASDAQ: BIIB  ) .

How Biogen is changing the MS treatment landscape
Biogen Idec isn't your run-of-the-mill $67 billion biopharmaceutical company. Out of its pipeline of seven Food and Drug Administration-approved therapies in the U.S., four are geared toward treating varying aspects of MS.


Source: Biogen idec.

For patients who've had their first clinical MS attack which has been confirmed with a brain MRI scan, there's Avonex. According to Biogen Idec, Avonex is being given to more than 135,000 patients worldwide, and it accounted for $3 billion of Biogen Idec's $6.9 billion in total revenue in 2013. Best of all, the introduction of the once-weekly Avonex Pen with a shorter needle (compared to the pre-filled Avonex syringe) has reinforced the convenience of taking the MS relapse-preventing drug while also lessening the pain associated with an injectable therapy.

Biogen also has longtime relapsing MS therapy Tysabri which is given as an infusion to patients once every four weeks. Based on data from its Phase 3 AFFIRM trial, Tysabri helped reduced the relapse rate by 68% when compared to the placebo, and it reduced the risk of disability progression by a range of 42%-54%. Furthermore, in 2013 Biogen Idec purchased its un-owned portion of Tysabri's global rights from Elan, which is now owned by Perrigo for $3.25 billion. Although Perrigo is still entitled to small royalties, Biogen can bask in the nearly full revenue potential of its infused MS therapy. Last year it accounted for $1.5 billion in sales.

Biogen also has a therapy that it's licensed out from Acorda Therapeutics (NASDAQ: ACOR  ) to treat walking disability associated with MS. This drug, known as Fampyra, was shown to increase walking speed by 25% for those patients who demonstrated a response. It's the first commercially available therapy specifically designed to help patients with MS-associated walking disability, so it holds a comparative advantage in that regard. Sales of Fampyra totaled $74 million last year.

But the crème de la crème of Biogen's product portfolio is Tecfidera. Approved last March, Tecfidera demonstrated remarkable efficacy in its CONFIRM and DEFINE studies with a reduction in MS relapses of 49% and a drop in new or expanding lesions across both trials of 71% to 99%. But, it isn't just efficacy that makes Biogen's oral MS therapy stand out – it's the safety record behind Tecfidera. As noted by the FDA's approval, the most common side effects were flushing and gastrointestinal events, while Biogen notes its medication can cause low lymphocyte counts. Compared to black box warnings and the rare possibility of a cardiovascular event, Tecfidera is a hands-down improvement in safety. Sales of Tecidera were monstrous with the drug logging $876.1 million in sales over just two-and-a-half quarters last year.  


Source: Biogen Idec. 

Biogen's not done yet
Having nearly all of its product revenue coming from MS therapies doesn't mean that Biogen Idec's slowing down one bit when it comes to MS research. In fact, the company has an additional relapsing MS therapy currently under review by the FDA as well as a separate experimental drug in phase 2 studies.

The therapy currently under review is Plegridy for relapsing forms of MS, which the FDA is due to decide on whether to approve or reject by mid-June. Plegridy is a pegylated subcutaneous injection that's nothing more than a longer-lasting version of Avonex. In its phase 3 ADVANCE trial Plegridgy met its primary and secondary endpoints of reducing relapses, disability progression, and brain lesions compared to the placebo.  Over the long run Plegridy has the potential to deliver anywhere from $3 billion to $5 billion peak sales according to Wall Street estimates.

Biogen Idec also has a revolutionary compound in two phase 2 studies at the moment known as anti-LINGO (BIIB033). This fully human monoclonal antibody that binds to a cell surface protein known as LINGO-1 is targeted at restoring nerve function to nerve fibers that were adversely affected by the LINGO-1 protein. In other words, this is an actual attempt at addressing what are believed to be one of the root causes of the disease rather than just managing the symptoms.

As I said, we have a long road to travel before we make a sizable dent in beating MS, but with Biogen Idec in the corner of patients, and investors, things are looking better for everyone all the way around.

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Read/Post Comments (3) | Recommend This Article (6)

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Help us keep this a respectfully Foolish area! This is a place for our readers to discuss, debate, and learn more about the Foolish investing topic you read about above. Help us keep it clean and safe. If you believe a comment is abusive or otherwise violates our Fool's Rules, please report it via the Report this Comment Report this Comment icon found on every comment.

  • Report this Comment On April 21, 2014, at 12:18 PM, BillCohen wrote:

    Also important for MS patients to know: "Merck Serono and Opexa Therapeutics enter Option and License Agreement for Development of Tcelna™ (imilecleucel-T) for Treatment of Multiple Sclerosis" This personalized therapy is much more advanced than the therapies in your article and to date have shown greater efficacy with no clinically measured side effects.

  • Report this Comment On April 21, 2014, at 1:18 PM, AF wrote:

    "We have built-up significant knowledge and expertise in the area of immunotherapy and continue the strengthening of our patent estate to protect our assets and technology. In 2013, we secured the 50th patent on our T-cell technology (US and international jurisdictions, collectively), and submitted an additional patent application in 2013 for our proprietary Epitope Screening Assay.

    Opexa uses its proprietary manufacturing process, ImmPath®, to manufacture all Tcelna product. The process has proven robust over the past year enabling us to manufacture over 350 doses of Tcelna for patients in the Abili-T trial. This T-cell platform technology supports the ongoing and future clinical development manufacturing needs of Tcelna and, importantly, also has the potential to be leveraged for possible therapies against other disease indications.

    In summary, we are pleased with the following objectives achieved this past year:

    ·Achieved enrollment of 70% of patients in the Abili-T clinical trial;

    ·Achieved initiation and activation of 33 clinical sites in the United States and Canada;

    ·Implemented what we believe is one of the most comprehensive Immune Monitoring/Biomarker Programs in MS as part of the Abili-T trial;

    ·Strengthened our financial position and cleaned-up the balance sheet through the elimination of debt;

    ·Unencumbered all intellectual property and physical assets pledged as collateral;

    ·Secured an option and license agreement with Merck Serono which, if exercised by Merck Serono, has the potential to bring in $220 million in milestone payments plus 8-15% royalties;

    ·Secured the 50th patent (US and international jurisdictions, collectively); and

    ·Laid the foundation to potentially diversify and expand off the T-cell platform.

    We believe 2014 should be another strong year for Opexa. We look forward to reaching full enrollment in the Abili-T trial, which will be a key milestone for the Company and SPMS patients. The ongoing analysis of data generated from the Immune Monitoring program could also yield important developments."

    We work closely with the MS Society, sponsoring and supporting important events for the community, including the MS Walk program and the MS 150. Interactions with the MS patient community are rewarding for all members of the Opexa team and remind us of the purpose of our work and, most importantly, what remains to be done."

  • Report this Comment On April 21, 2014, at 1:20 PM, AF wrote:

    "2013 has been an excellent year for Opexa. We reached several key milestones and executed on strategic goals set for the Company. We saw strong enrollment in the landmark Phase IIb Abili-T clinical trial evaluating Tcelna® (imilecleucel-T) in Secondary Progressive Multiple Sclerosis (SPMS), achieving 70% enrollment in December 2013. We ended the year in a strong financial position with a clean balance sheet. Earlier this year we secured an option and license agreement for Tcelna with Merck Serono, one of the leading multinational companies focused on the treatment of multiple sclerosis (MS). If we are successful with the development of Tcelna and Merck Serono exercises its option, Opexa could be eligible to receive up to $220 million in milestone payments and, additionally, 8-15% in royalties. Importantly, Merck Serono would also be responsible for absorbing all future costs related to clinical development, including all Phase III trials, as well as pre-commercialization expenses. We feel this agreement provides further validation as to the potential of Tcelna and, if executed, could provide Opexa with a strong development and commercialization partner.

    We continued to focus on building and maintaining a strong team of individuals with a broad range of expertise across numerous functions. We are currently a dedicated group of approximately 40 individuals covering a range of activities including manufacturing, quality control and quality assurance, shipping and logistics, clinical and regulatory affairs, research and development, business development, finance, accounting and compliance. Our focus as a group remains locked on working hard to deliver shareholder value and improve the quality of life for individuals with MS.

    Should Tcelna prove to be safe and effective in the ongoing Abili-T SPMS clinical trial, Opexa will have differentiated itself significantly from other MS focused companies. Individuals suffering from SPMS have very few treatment options available to them. The estimated potential market for SPMS is over $7 billion in the United States alone, and we believe Tcelna has the potential to become a treatment of choice in this patient population. A significant milestone in evaluating Tcelna’s potential comes in mid-2016, when top-line data from the Abili-T trial are expected."

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