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3 Things In Biotech You Should Learn Today: July 15, 2017

Jul. 15, 2017 9:30 AM ETAMGN, RHHBY, PFE, RHHBF6 Comments
Zach Hartman profile picture
Zach Hartman


  • Spark and Pfizer kick out encouraging preliminary data in hemophilia B.
  • And Roche had a major publication in hemophilia A!
  • Amgen cements its place in the treatment of acute leukemia.

Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep up to date with recent findings in the fast-moving world of biotech and pharmaceutical research.

What's on the docket for today?

Spark and Pfizer kick out encouraging preliminary data in hemophilia B

Spark Therapeutics (ONCE) and Pfizer (PFE) have generated a lot of excitement for their jointly developed gene therapy solution for hemophilia, specifically hemophilia B, which results from a patient's genetic inability to make sufficient amounts of coagulation Factor IX.

The product in development is called SPK-9001, and it is an adeno-associated virus that delivers genetic material without risk of infection.

ONCE presented interim results at the 2017 International Society on Thrombosis and Haemostasis Congress as part of two presentations.

In abstract OC 13.1, the companies detailed the results of 10 patients enrolled in a phase 1/2 study, noting that all 10 patients had a 99 percent reduction in the rate of annual infusions of Factor IX, down from a mean of 67.5 to 1.0. Furthermore, 9 out of 10 patients had not experienced a bleeding event since the infusion of the vector, and annualized bleeding rate was reduced by some 96 percent.

In abstract PB 1900, analysis of questionnaires from patients enrolled in the phase 1/2 study suggested that their quality of life had improved, including their perception of dependence from Factor IX supplementation and decreased worry about their hemophilia worsening.

Looking forward: Yet another impressive notch for PFE and ONCE with this very interesting gene therapy. It feels increasingly likely that they have a substantially disruptive technology on their hands, with highly favorable tolerability. The only toxicity observed to date has been mild elevation of liver enzymes, which is quite manageable. No serious adverse events have been observed yet, though this is

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Zach Hartman profile picture
I am a former PhD scientist-turned-writer focused on cancer education. My writings in Seeking Alpha have been devoted to helping people identify promising investment opportunities in cancer research through commentary of recent events. Readers can learn more about other aspects of cancer research by visiting my site Invest Against Cancer.I also collaborate with Avisol Capital Partners on their Marketplace service known as the Total Pharma Tracker (TPT). Some of my work will be available to TPT subscribers either exclusively, or in advance. If you are interested, please click the link above!

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Comments (6)

user anita profile picture
Thanks! Long ONCE.
Zach Hartman profile picture
Good luck!
SPHS to the moon
Zach Hartman profile picture
Based on what parameters?
KarinCA profile picture
Good morning! I like your updates and try to tweet them out. ;)
Zach Hartman profile picture
Thank you, Karin! I really appreciate it!
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