Wednesday, December 27, 2017

Integrated BioSci Report Of Alpha-Intelligence On Spark Therapeutics: Elucidating The Ramifications Of The Approved Gene Therapy Luxturna


  • First FDA-approved gene therapy, Luxturna, for the treatment of a rare eye disease will be available within months. Unprecedented approval set the tone for further acceptance of similar gene therapies in the foreseeable future.
  • Orphan designation enabled the company to charge a premium pricing to offset the lengthy and low success innovation process.
  • With proof that their stellar technology works, Spark is most likely a prime target of acquisition.
  • Rare Pediatric Disease Priority Review (“RPDPR”) voucher earned can be used for subsequent marketing application or sell to another firm for roughly $60M.
  • It’s prudent to build shares while the market has yet to recognize the substantial unlocked value in this firm.

“Finding the really outstanding companies and staying with them through all the fluctuations of a gyrating market proved far more profitable to far more people than did the more colorful practice of trying to buy them cheap and sell them dear.” - Philip Fisher

On Dec. 19, 2017, Spark Therapeutics (NASDAQ:ONCE), a leading gene therapy innovator, announced that the US FDA approved voretigene neparvovec-rzyl (Luxturna) - a one-time gene therapy for patients afflicted with biallelic RPE65 mutation-associated retinal dystrophy. Interestingly, the market has been ambivalent regarding this news due to the lackluster data presentation at the 59th annual meeting of American Society of Hematology (“ASH”). Despite the monumental news, the price only traded up by $2.55 at $52.75 (for 5.08% profits). Thereafter, the share price traded North only at $54.06, which is quite meager based on its substantial unlocked value.

Figure 1: Spark Therapeutics stock chart. (Source: StockCharts).

At Integrated BioSci Investing, we have much success in finding robust performers. For instance, Nektar Therapeutics (NASDAQ:NKTR) appreciated over 210% for subscribers. In this report, we'll elucidate the ramifications of the Luxturna approval and to assess Spark Therapeutics’ prospects as a potential big winner for 2018.

Author Note: We're honored that you took the time out of your busy day to read our market intelligence. Founded by Dr. Hung Tran, MD, MS, CNPR, (in collaborations with Dr. Tran BioSci analyst, Ngoc Vu, and other PhDs), Integrated BioSci Investing marketplace research is delivering stellar returns since inceptions. To name a few, Nektar Therapeutics procured more than 210% profits while Spectrum Pharmaceuticals (SPPI) delivered over 180% gains. Our secret sauce is extreme due diligence coupled with expert data analysis. The service features a once-weekly exclusive in-depth Integrated BioSci Alpha-Intelligence article (in the form of research, reports, or interviews), daily individual stocks consulting, and model portfolios. This article is the introductory preview version of the weekly Alpha-Intelligence Integrated BioSci research that we published in advance to IBI subscribers.
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