Thursday, August 7, 2014

FDA Greenlights Use of TKM-EBOLA in Current Outbreak

Canadian drug developer Tekmira just disclosed that the FDA verbally notified them that it would partially lift the clinical hold it had placed on TKM-EBOLA a month ago.  The clinical hold had been instituted as the result of cytokine elevations that had been observed in a healthy volunteer study of the drug.

By partially lifting the clinical hold, the FDA ‘blesses’ the use of TKM-EBOLA in actually infected patients whose odds otherwise would be to die from the infection.  Since the FDA only has authority over health matters in the US, this is strictly only of relevance for the use in people infected with Ebola either brought back from Africa for treatment just as the two US aid workers, or in case the virus started to crop up in the US. 

I am, however, inclined to interpret the news that the FDA is encouraging Tekmira to consider the use of TKM-EBOLA in Western Africa at the epicenter of the epidemic.   You could imagine that if anything went wrong with the use of TKM-EBOLA, it might have had adverse consequences for the future clinical development of the drug candidate in the US.


Overall, I am pleased with this development as the clinical hold in light of the current health emergency was just unbearable. How to handle the practicalities of using TKM-EBOLA in the field, including who will bear the costs, we will have to see.  Remember, financially, it is Tekmira's HBV candidate that has priority over TKM-EBOLA and so I expect TKM-EBOLA to be part of a broader effort funded by the public health bodies.

12 comments:

Anonymous said...

Does this mean BLT will become a success story? Curing Ebola with one shot is quite something.

Anonymous said...

It's not like this is the first time this has ever happened... for all those who are bullish about use of experimental medicine in africa, here is a cautionary tale

http://www.washingtonpost.com/wp-dyn/content/article/2006/05/06/AR2006050601338.html

Anonymous said...

The Pfizer/Nigeria situation was (at least per that article) different in that people weren't informed.
In this case it's not likely TKM-Ebola will be used without the explicit permission of every stakeholder.
That said, the day's (+40%) gains of $TKMR over this news appears to be a bit nonsensical. The results of such a trial aren't likely to meaningfully contribute to the commercial pipeline for infectious disease (i.e.: HBV). Future products will continue to bear the risk of the immuno-stimulation issues inherent with LNP/RNA.
A market cap of 470M doesn't seem excessive when $ALNY is considered (currently ~ $4B), particularly considering their current and potential revenue streams in oncology. However such heavy short term gains in the face of news not likely to bear any commercial significance speaks more to the state of a speculative short-term interest market likely to cool just as quickly as it heated up. This might be a great time to short?

Anonymous said...

If tkm ebola works well and it probably will it will leap frog rnai into the viral space, remember the current flu vaccine programs are very lax in there testing as a matter of history, this could achieve the same advantage for rnai. It is interesting that shrna also is close to clinical verification with callimmune HIV and BlT HepC, exciting times indeed.

Roger Ting said...

How do we track the current effectiveness if TKMR-Ebola is used more widely in the current outbreak?

Anonymous said...

I think it will another stars day to shine this week. Maybe tomorrow.

Alnylam is a big constellation.

Pity I can't find anything on a Google search that describes it except as a pharmaceutical company.

Anonymous said...

http://www.bnn.ca/Video/player.aspx?vid=414671

Doubla Loe on BNN.

Anonymous said...

http://www.bloomberg.com/news/2014-08-10/new-ebola-drugs-create-debate-over-effects-on-outbreak.html

Sierra Leone has contacted Tekmira...

Anonymous said...

Most likely outcome, share price aside, is new mutant strain of experimental drug resistant Ebola.

Anonymous said...

Very true. a bit hasty. May do well temporarily for the stock price. Hopefully, the cytokine storm issue led to FDA hold does not result in detrimental effect to set the RNAi field back again.

Anonymous said...

It's not like this is the first time this has ever happened...
Does this mean BLT will become a success story?

Anonymous said...

BLT will never be a success story as things stand or by itself.

When you read that article Dirk posted up, it is clear, the vision of the two main men in BLT is to make a wad of cash and go home. Sounds like a trade sale to me.

There is no mention of the new lab and apparently the toxicity issues of old are still unresolved due to the high number of "events" in the mice subjects.

BLT a success story? And pigs really can fly on Planet Claire.

By Dirk Haussecker. All rights reserved.

Disclaimer: This blog is not intended for distribution to or use by any person or entity who is a citizen or resident of, or located in any locality, state, country or other jurisdiction where such distribution, publication, availability or use would be contrary to law or regulation or which would subject the author or any of his collaborators and contributors to any registration or licensing requirement within such jurisdiction. This blog expresses only my opinions, they may be flawed and are for entertainment purposes only. Opinions expressed are a direct result of information which may or may not be accurate, and I do not assume any responsibility for material errors or to provide updates should circumstances change. Opinions expressed in this blog may have been disseminated before to others. This blog should not be taken as investment, legal or tax advice. The investments referred to herein may not be suitable for you. Investments particularly in the field of RNAi Therapeutics and biotechnology carry a high risk of total loss. You, the reader must make your own investment decisions in consultation with your professional advisors in light of your specific circumstances. I reserve the right to buy, sell, or short any security including those that may or may not be discussed on my blog.