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THE SCIENCE BEHIND THE IPO CRAZE: OUR FOCUS FOR THE NEXT FEW WEEKS

I want to thank you for subscribing to Biotech Primer WEEKLY. Now, imagine yourself on the set of Jeopardy! facing this clue:

“We saw 45 in 2013, and there’s been 17 so far this year.”

The correct response: “What is the number of biotech IPOs?”

Alex quips: “That is correct! And there’s no sign of a biotech industry IPO slowdown.”

Great news if you own one of these companies, and even better if you are a patient (and aren’t we all at some point). Investment equals innovation. Innovation ultimately results in new therapeutics. The therapeutic areas bringing in the most IPO bucks are cancer, autoimmune disease, and rare diseases.

In the next few issues, we will take a look at the science behind the buzz.

IPO FOCUS: DICERNA

One of the hottest IPOs of 2014 is Dicerna Pharmaceuticals. Staying true to the IPO trend, Dicerna focuses on both cancer and rare disease therapeutics. Offered at $15/share, the company sold six million shares, raising $90 million in cash. The success of the initial offering exceeded expectations and continues to do well. In the ensuring weeks, shares traded as high as $44.95/share.

Dicerna claims to have a new and improved version of RNA interference (RNAi), the gene-silencing technology similar to the antisense technology featured in this space last month. The RNAi process works by taking advantage of pre-existing cellular pathways that recognize and destroy double-stranded RNA (dsRNA), thus blocking expression (or making) of a disease-associated protein.

RNA is naturally single-stranded in the human body. When the body finds dsRNA, it recognizes something is wrong and destroys it. By introducing dsRNA into cells, it is possible to destroy the messenger RNA (mRNA), which carries the sequence that codes for the disease-associated protein. If mRNA is destroyed, the protein is not produced and the person is no longer ill. Tricky!

LEARN MORE: ADVANTAGE RNAI

The most tantalizing potential upside of RNAi therapeutics is their ability to target previously “undruggable” targets.

Biologic drugs such as monoclonal antibodies can be highly effective against targets on the surface of a cell or circulating in the patient’s blood; however, they are physically too large to enter the patient’s cells. Small molecule drugs can enter a patient’s cells but it is not always possible to identify an appropriate small molecule binding site on these intercellular targets. Potential drug targets such as transcription factors—the proteins that “turn on” the production of a specific protein or group of proteins in response to a particular signal—have been difficult to reach with either small or large molecule therapeutics.

Various types of cancer have been associated with overactive transcription factors which represent a prime target for RNA-based therapeutics.

COCKTAIL FODDER

The company name Dicerna is derived from the cellular enzyme that activates the RNAi pathway: Dicer.

TERM OF THE WEEK: DRUG TARGET

A drug target is a molecule involved in a disease that is modified or affected by a potential therapeutic.

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