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3 Things In Biotech, March 2: Shire Pushes, Acceleron Shines, And Antisense Goes On

Mar. 03, 2018 8:00 AM ETATHJF, IONS, SHPG, MRK24 Comments
Zach Hartman profile picture
Zach Hartman


  • Shire enters the home stretch with its newer formulation of asparaginase.
  • Acceleron publishes early-stage results in muscular dystrophies.
  • Antisense Therapeutics gets the go-ahead on trials in Duchenne muscular dystrophy.

Note: Subscribers to Avisol Capital Partners Total Pharma Tracker got an early look at this publication.

Welcome to another edition of "3 Things In Biotech" a daily digest dedicated to helping you keep pace with the fast-moving world of pharmaceutical and biotechnology research.

Shire enters the home stretch with its newer formulation of asparaginase

Company: Shire Group (SHPG)

Therapy: Calaspargase pegol

Disease: Acute lymphoblastic leukemia

News: SHPG announced that the FDA has accepted the application for approval for its newer formulation of asparaginase called calaspargase pegol. Pegylated asaparginase forms an important piece of the puzzle for the treatment of acute leukemia, particularly in children. The FDA has set an action date for review of this application on December 22.

Edit: This article originally stated that the calaspargase pegol was approved in Europe. This was an oversight and has been corrected.

Looking forward: In the realm of therapy for this form of leukemia, asparaginase isn't exactly the sexiest ticket today. That would be the CAR-T cells or other immunotherapeutic strategies. However, asparaginases form an important cornerstone for the treatment of acute leukemia, and its use is being better recognized in the United States for non-pediatric patients. So a novel formulation of the drug that helps to circumvent issues relating to dosing and resistance. Oncaspar nets some $100 million in sales in Europe, so it is likely to give a big boost to SHPG if approved in the US, as well.

Overall, this is a favorable sign for SHPG, but it is just one piece of a big puzzle for it.

Acceleron publishes early-stage results in muscular dystrophies

Company: Acceleron Pharmaceuticals (XLRN)

Therapy: ACE-083

Disease: Muscular dystrophies

News: XLRN announced the publication of the final results from a phase 1 study in the journal Muscle and Nerve. These results describe

This article was written by

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 (24)

matthew reynolds profile picture
great article. but antisense now looking up after failure of Sarepta trials. www.greenleiter.com/...
no comment
Zach Hartman profile picture
Thank you for the comment!
04 Mar. 2018
Im a newbie but would hate to see "3 Things" go away. i appreciate its straight to the point approach with accompanying opinion-advice. Thank you.
Zach Hartman profile picture
Thank you yumjr! No plans to end 3 Things if I can avoid it!
Hi Zach , any thoughts on EXEL , I’ve ridden it up but big drops this week make me nervous.....
thorgood4 profile picture
good question..
Zach Hartman profile picture
EXEL is strong. Cabozantinib is a huge success and doesn't seem to be slowing down
What are your views on Aveo ?
Zach Hartman profile picture
I think they're quite the turnaround story, and their anticipated trial results are quite exciting!
Thanks for you comment!

Does current valuation (today's market cap is 391M) make sense ? Even if we assume it gets FDA approval for upcoming result ? What's your view on that ?
Zach Hartman profile picture
I feel that this is a fair valuation until they get approvals that set them apart from the rest of the crowd in the renal space. Of course, they DO have some interesting trials in the pipeline that might push them forward, like TiNivo. In that case, I'd expect the valuation to track up significantly higher.
BraceYourself profile picture
Interesting action but what do we really know about this field called antisense? Who hold the cards? Info seems to be very technical can you direct us to some explanations?
Zach Hartman profile picture
Hey Brace, I'm not aware of good resources that explain antisense therapy in simple terms, although the tech has been around for a LONG time, well before I started graduate school a decade ago.

Perhaps it would be useful to have a guide to antisense stuff on Seeking Alpha?
Adam May profile picture
This SA article did a good job breaking down ASO (antisense) technology that Ionis uses in a way that allows non-science oriented readers to better understand how it works. You'd need a paid account to view it now though.


In terms of "holding the cards" in antisense - it mostly comes down to Ionis and Alnylam (which uses RNAi, slightly different from ASO). Both companies are having some success now after antisense technologies seemed like a disappointment years ago.
kas23 profile picture
Here’s a quick primer on antisense technology. The Central Dogma of Biology says we have genes in our DNA. These genes product another nucleic acid called message RNA (or mRNA). mRNA is then used as a template for the ribosomes to string together amino acids to form a protein. This protein can sometimes be the culprit behind disease, especially if someone has a mutation in their DNA. You stop the protein production and you may stop the disease. Antisense RNA is a nucleic acid designed to bind a very specific mRNA and lead to its destruction. So, you give antisense RNA and it leads to a decrease in the expression of a given protein. This technology has been around for 20-30 years, but it’s use in humans to treat disease has not. It has been a mixed picture there.
Martin Hansen profile picture
Hi zach. Are you still bullish on VSTM and duvelisib approval? Can see that insiders Are buying. That is a good Thing right? Regards Martin
Zach Hartman profile picture
Hey Martin, I haven't heard anything to give me pause on the possibilities of duvelisib!
Strike profile picture
Isn't IONS the breakthrough developer of anti-sense? What advantage does ATHJF possess over IONS?
Flying Tiger profile picture
As the article states, Ionis licensed the technology to ATHJF - so ATHJF will run the trials, and if the drug gets approved, will pay IONS a royalty (likely a percentage of sales). It is not really that ATHJF has an advantage over IONS, it is instead that IONS has so many drug targets to go after that they can't pursue all of them in house, and so allow others to use their tech and IP in selected instances (typically in return for milestones payments, license fees, and or royalties).
IlliniJ profile picture
XLRN has 7 phase 2-3 studies in process, multiple data readouts in 2018/19 for a variety of conditions......the future will be exciting.
Zach Hartman profile picture
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