Sarepta Therapeutics (SRPT) slips into the red in premarket action (-7% now) as CEO Chris...


Sarepta Therapeutics (SRPT) slips into the red in premarket action (-7% now) as CEO Chris Garabedian fields questions about dystrophin expression as a predictor of clinical outcomes on a conference call convened to discuss an NDA filing for eteplirsen. (previous)

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  • bronagray
    , contributor
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    From the perspective of a professional in biology, chemistry and medicine plus personal experience with the submission of an NDA, the just concluded news conference is a remarkable ipso facto. Public perception, including the news media, oft envisions the FDA to be a Govt. ogre sitting on a throne to which alms with political garnish must be offered. Ignorance can deny health and wealth. Vocabulary and understanding re the clear and explicit of Sir Francis Bacon, published four centuries ago, could also guide those with a 'money' focus to learn the fundamentals underpinning an investment in Sarepta. Good drugs and good money are inseparable from the Scientific Method. The FDA, irrespective of the rhetoric, operates by the Principles of the Scientific Method to address the issues of Safety and Efficacy for approved drugs to protect the public interest. ‘Approval’ is inseparable from the Scientific Method, that which precedes and provides the basis for an edict of the FDA, never the reverse. Clearly missed in some of today’s questions was an understanding of the process and the pecking order. Dystrophin SCIENCE is neither a business decision or FDA edict.

     

    Muscular Dystrophy is 'molecular pathophysiology' in simple and explicit language. Dystrophin is a complex protein essential for normal muscle physiology. Scientists at the FDA are in lockstep with ongoing advances in the medical sciences as biochemistry deciphers proteomics or the biochemical physiology of dystrophin. FDA scientists have online access to the NLM equivalent to all of us. Mr. Garabedian reaffirmed and provided insight to an extraordinary and additive value associated with the development of the drug candidate eteplirsen, a conjoint focus utilizing the Scientific Method. The FDA has not mandated that Sarepta provide the Holy Grail, the long elusive biochemical physiology of dystrophin prior to drug approval. Indeed, to remove ‘dystrophin’ from the vocabulary for seeking the FDA approval of eteplirsen would not change the process. Rather, today the marketplace was accorded insight into a remarkable synergy of the FDA, Sarepta and the worldwide scientific talent focused via the Scientific Method to achieve a pivotal advance in the fundamental knowledge of dystrophin with employment to facilitate the development of therapeutics. That the leadership of Sarepta and the FDA understand the diaspora of benefits for mankind inherent in their parallel and conjoint effort associated with the approval process for eteplirsen also creates unique value for the business of Sarepta and investors.

     

    The immediate marketplace response to the presentation implies what? Did some investors perceive that the recent meeting with the FDA would spontaneously engender approval for any drug sans submission of an NDA? Did some investors perceive that after the meeting, Sarepta would hand airline tickets to representatives bound for their local pharmacy with boxes of eteplirsen for sale? Irrespective of the Scientific Method essential for the medical and pharmaceutical advances, do the Wags of Wall Street dare to learn about the chemical and pharmaceutical engineering essential for drug production, the minimum required for stocking an established product at their local pharmacy? By comparison, the incremental ’stops on a manufacturing line’ for a pharmaceutical oft relegate autos to being a garage business. Seemingly unrecognized is the astute business foresight of Sarepta leadership many months ago with an initiation of the parallel business commitment, construction of the 'aircraft carrier, USS Eteplirsen’. Not even at the zenith of WWII, was an aircraft carrier created in a year, much less a meeting. Disclosure of a successful ‘manufacturing lot’ certifies Sarepta to be on track to avoid adding months to the ‘whole’ with a serial style of management.

     

    As of yet there is no Conclusion from the Scientific Method whence the FDA can evaluate. As of yet there is no USS Eteplirsen to float. Today, insight was provided re the creative management of these essentials in a synergistic multi-path PLUS the extraordinary value-added, a cohesive focus via the Scientific Method to understand the elusive molecular biochemistry and physiology of dystrophin. Oh yes... and Watson, did you note what was missing? In addition to a clear vision to replace the fog of unknowns re the FDA approval process and product production challenges, not a single new noun was added to the vocabulary to describe an unrecognized scientific or business challenge threatening the launch of the USS Eteplirisen. And by the way Watson, investors never need to worry about hidden questions from the FDA, NEVER. That is their charge, one with Professional standards. Now, if Wall Street Wags would prioritize learning the simple fundamentals so as to guide the safety and efficacy of our investments with diligence equivalent to that of the FDA, we would lead healthier and more prosperous lives. Today, Sarepta paid homage to the clear principles of Sir Frances.
    24 Jul 2013, 04:16 PM Reply Like
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