Biotech portfolio updates – Exelixis, Spark, buying more gene therapy stocks

Exelixis – Decreasing exposure on valuation

Exelixis (EXEL) continues to look very strong after hitting a 15-year high on Friday, which is never a bad point in time to realize some gains. While I still view Cabometyx as the most effective agent ever approved in renal cancer (even better than Opdivo), a valuation of $5.1B seems to fully capture the current label. Continue reading

Biotech catalysts for 2016

After last week’s pessimistic post, this week I am focusing on potential catalysts in 2016 that could improve sentiment towards biotech as a sector.

As if to remind us late stage trials don’t always fail, last week saw positive news from three different programs, all of which are antibodies in non-oncology indications. Regeneron (REGN) and its partner Sanofi (SNY) announced excellent data in atopic dermatitis, Alder (ALDR) reported positive results in a P2b in migraine and Pfizer (PFE) had positive P3 data for its PCSK9 program.

Below are four additional clinical data readouts that (if positive) may serve as important catalysts. Continue reading

Drugs to Watch at ASCO 2011

More melanoma breakthroughs

This year’s meeting will probably be remembered as a historical event with regards to melanoma. Last year, it was a phase III trial for BMS’ (BMY) Yervoy (ipilimumab), which was the first in history to show a survival benefit in advanced melanoma patients (discussed in my ASCO 2010 write up). This trial led to Yervoy’s historical approval 3 months ago.

This year, investigators will present studies evaluating Yervoy as well as Plexxikon/Roche’s vemurafenib in pretreated melanoma patients. Yervoy was evaluated in combination with chemotherapy while vemurafenib was compared with chemotherapy. According to BMS’ and Roche’s press releases, both studies were successful and each drug led to a survival benefit.  The extent of this benefit is still unknown and will be revealed only at the conference. Continue reading

Investing in microRNA – Beware of Hype, Follow The Money

The momentum in the microRNA (miRNA) field is continuing to build, as evidence that links this group of important genes to multiple diseases, from Alzheimer to diabetes, is constantly mounting. The adoption of miRNAs is further accelerated by the technological advancements in RNA-based therapies. Because miRNAs are not expressed as proteins but as RNA, the most sensible way to target them is using small RNA technologies from the likes of Isis (ISIS) and Alnylam (ALNY). These technologies have come a long way in recent years, and are now expected to form the basis for a new generation of drugs. Although miRNAs gained wide recognition only recently, the combination of a fresh pool of unexplored genes which are implicated in so many medical conditions together with the maturing technology to manipulate their activity creates a huge untapped market.

 

So far, 2008 has been particularly fertile, with several exciting events taking place during recent months. Continue reading

Rosetta Genomics – Mining Gold Out of Junk (Part II)

  

On the previous article , I discussed the importance of microRNAs (miRNAs) and the great attention they have been receiving in the scientific industry. With every day that goes by, miRNAs gain more momentum and their cardinal role in biology is constantly being elucidated. Due to their importance and presence in such a wide range of creatures and medical conditions, from plants to humans, from cancer to women’s health, miRNA are making the migration from a scientific sensation to a huge commercial opportunity. This is great news for a company focused purely on miRNA such as Rosetta Genomics (ROSG), as after years of wandering alone in the desert, it may find itself at the heart of one of the hottest segments in the pharmaceutical industry.

 

The rationale behind miRNA-based drugs and diagnostics is straight forward. miRNAs are a group of genes which are involved in almost every biological process, as they control over a third of our genome. This central role makes them obvious “druggable” targets, which can be manipulated in order to treat diseases. Their central role might also make them ideal bio-markers for early-detection and diagnosis purposes, due to several advantages that will be described later on. The key in commercializing miRNA-based products is finding the relevant ones which are associated with a specific medical condition. The process starts by looking at the miRNA profile of healthy cells and compare it to that of cells with the particular condition or disease. The miRNAs that may serve as diagnostic markers or drug targets are those which have a different expression profile in the particular condition compared to normal cells.

  Continue reading