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Gene Therapy Attack Capital Time? QURE


UniQure (NASDAQ:QURE) is delivering on the promise of gene therapy, single treatments with potentially curative results. We have developed a modular platform to rapidly bring new disease modifying therapies to patients with severe disorders. We believe our multiple partnerships and the regulatory approval of our lead product Glybera in the European Union for a subset of patients with LPLD provides validation for our approach . Based on defined criteria, we have prioritized approximately ten indications for pre-clinical development.

One year ago, uniQure became the first and only company to receive regulatory approval in the European Union for a gene therapy product: Glybera for the treatment of LPLD. The way QURE’s gene therapy works is that a “protein factory” is inserted into the body, which enables the body to produce its own medicine. The way QURE’s CEO described it during the IPO roadshow is that a gene “blueprint” for the protein is combined with a delivery vehicle (Glybera uses the AAV virus, also known as a “Vector”). The next stage is to manufacture millions of copies of the gene and delivery vehicle. Then, the millions of copies are injected into the body, where the viral delivery vehicles deliver them to the appropriate place.  The company has manufacturing facilities in Amsterdam and under construction in Boston, MA in the USA.

What makes uniQure interesting is that it already has an approved product and the company should be generating revenue in the 2nd half of 2014.  Initially their plan was to go after orphan diseases with limited populations of patients, but now they with their partners believe they can expand into much larger patient population diseases using their delivery and modular technology. To wit, they just announced on January 14th (Today) a new collaboration on ALS (Lou Gehrig’s Disease). This platform could become very important going forward. That doesnt  even mention the 26 million patient population for Congestive Heart Failure through their Acquisition in August 2014 of Inocard, which found 90% survival rate in pig heart studies in pre clinical work. (Pig hearts are commonly used in Heart studies as they are virtually identical to Human Hearts)

The company has partnered with an Italian firm called Chiesi in order to market the current drug, with Chiesi receiving European rights to Glybera. Chiesi will pay QURE a royalty on Glybera sales of 20% that will increase over time to 30%, but QURE retains the rights to Glybera in North America, most of Asia, and Australia. They have fast track orphan drug status in the US, and assuming an approval here at some point we can expect the stock to be moving higher as well at that point.

Virtually nobody is discussing this Gene Therapy Biotech company, not one article on Seeking Alpha which is a sure tell that this should go higher yet.  Just now breaking out of a near 1 year POST IPO base pattern as a few investors are catching on.  Also of interest, Spark Therapeutics just went public in early February with a current 1 Billion market cap vs 350 million for uniQure.  Spark has one indication in phase 3 for a 3,500 patient population. They have a Hemophilia B candidate in pre-clinical stage, and otherwise their pipeline is thin compared to uniQure at 350 million market cap.  There is a big discount here owning QURE.

We would also point out that they are going after Cardiovascular as well as Liver based (Hemophilia) disease, and Joseph Schimmer (Piper Jaffray Biotech Analyst) had listed as #1 and #2 for top 15 surprises of 2015 .  Piper Jaffray has a $35 target on the stock and that was last March.

1. Gene Therapy establishes proof of concept in Hemophilia (QURE?)

2. Heart Failure explodes on Biopharma scene with Gene Therapy Success (CLDN? QURE?)

UniQure uses a Viral Vector delivery technology to go after their disease candidates. Called Adeno Associated Viral Vectors or AAV for short.  Though complex, QURE appears to have a big and early lead on this technology. For those who want a bit more brief background on AAV, read this link.  QURE is a likely undervalued company in the AAV space. They have already industrialized AAV for therapeutic use. They are now working on second and third generation technologies which could greatly expand their markets. Not only that, many CAR-T treatements require a vector (Vehicle which delivers the Cells etc) that is difficult to produce on a mass scale and with precision. QURE may end up providing that technology to others using their modular and cassette approach. (Read more below)

The geneQure platform has four key elements:

  • therapeutic gene cassettes that carry a transgene that encodes, or provides the blueprint for the expression of, a therapeutic protein;
  • an AAV-based vector delivery system for delivering the gene cassette;
  • administration technologies to effectively deliver the relevant transgene into the tissues and organs; and
  • a scalable, proprietary manufacturing process to produce our AAV-based vectors.

uniQure’s approach is designed to be modular, in that it may allow us to efficiently develop manufacture and seek regulatory approval for multiple gene therapies generally using the same principal components. In some cases, the disease-specific gene cassette will be the only component that needs to be changed to target a new disease in a particular tissue. As a result, they may be able to reduce the overall preclinical and potentially clinical development activities required to obtain regulatory approval, and to significantly reduce the overall development risk, time and cost.  So the key here is Modular and scaleable on a production basis, hard for competitors to  duplicate, easier for them to license or partner up.

uniQure’s pipeline is focused on:
(i) programs for orphan diseases with no effective treatment options (Porphyria, Sanfilippo B, LPLD) or
(ii) disruptive innovation programs destined to vastly improve the lot of patients and to change the dynamics of established markets (Parkinson’s disease, Hemophilia B, Congestive Heart Failure)

The pipeline includes the currently approved AAV-1 Vector based drug  Glybera, considered now the worlds most expensive drug regimen with a 1.1-1.4 million cost to treat a typical patient for the disease it is going after.  Hemophilia and Congestive Heart Failure are their other main targets.  In October 2012, the European Commission granted marketing authorization for Glybera under exceptional circumstances as a treatment for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) confirmed by genetic testing, and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions.  The drug has only recently started being made available though due to further studies that required 6 years total to have data completed, and hence the sudden uptrend in the stock price.

As mentioned above, also Hemophilia, ALS,  Parkinsons Disease, Congestive Heart Failure and more.  Suggest you scan this link for the current brief descriptions of each target.

Company provided information: (Bolding by SRP)

We conduct early-stage discovery and preclinical research, often in collaboration with academic research institutions, into a number of potential applications of our technologies.  In addition, we aim to access the best early-stage programs and accelerate their development by the application of our proven modular platform.

Our principal near-term research focus is on diseases involving the liver, most prominently hemophilia A, and CNS, as well as on cardiovascular diseases, through our acquisition of Inocard.  

qure pipeline

So we realize this is a complex field but again, this looks like a relatively undiscovered Biotech company that has the potential keys to Vector delivery of Gene therapy targets using their Modular and Cassette approach. With one drug already approved, it stands they have a platform to build on and a lead in AAV.  With a post IPO breakout just now occuring just in the past several weeks from the $17 IPO, this looks like a good stock to build a position in on this breakout area on the current Biotech Pullback.

We cant be sure if this takes off to 24-25 plus and we miss it, but we will suggest you try to buy from 19-21 per share for now, then we will go from there.  They should be announcing the dosing of first patient in the Hemophilia B indication any day with results coming in the summer.  Establish a position, then look to add on pullbacks or perhaps we will even average up depending on action. Key we think is to get a position at this time. As it stands, they went public last year during the top in the small cap market  at $17 and only now is the stock really breaking out of that top range from a year ago almost.  They have been kind of lost in the shuffle and people tripping over each other to buy KITE, JUNO, BLUE AGIO and others.  We think this may be an opportunity.

Projecting 19-21 buy ranges inclusive as best to accumulate… we think institutions are accumulating the stock.

A few charts we did over last few days while monitoring market and this stock…

25 qure