Deep Pipeline Boost ISIS Pharmaceuticals

08/10/2015 7:00 am EST

Focus: STOCKS

John McCamant

Editor, Medical Technology Stock Letter

John McCamant, biotechnology expert and editor of The Medical Technology Stock Report, reviews one of his buy-rated stocks, which is a strong pipeline that is being supported by world-class partners.

ISIS Pharmaceuticals (ISIS) and their cardio subsidiary, Akcea Therapeutics, announced that The Lancet has published Phase I data evaluating its APO(a) drug candidate in healthy volunteers with elevated lipoprotein(a) or Lp(a).

Publication in one of the world’s’ leading medical journals is a major testament to both the APO(a) drug candidate and ISIS’ powerful antisense technology platform that can address another previously undrugable target.

Results of this study demonstrated potent, dose-dependent, selective reductions of plasma Lp(a) up to 89% (mean reduction up to 78%) in patients treated with ISIS-APO(a).

Phase II data in patients with very high Lp(a) is expected later this year and POC data could serve as a significant catalyst.

An estimated 20% of the population have elevated Lp(a) levels that put them at higher risk for cardiovascular disease, but because there has been no therapeutic option for lowering elevated Lp(a) levels, most healthcare providers have not been monitoring this important risk factor.

However, the availability of a therapeutic agent capable of lowering Lp(a) should lead to the widespread testing of Lp(a) levels. In our view, APO(a) is not represented in ISIS’ current valuation.

Meanwhile, ISIS earned a $2.15 million milestone payment from Biogen (BIIB) related to advancing the ongoing pivotal Phase III trial evaluating ISIS-SMN in children with spinal muscular atrophy (SMA). 

ISIS has also started a Phase I/IIa trial of ISIS-HTT in patients with Huntington’s disease (HD), which earned them a $22 million milestone from their partner Roche (RHHBY).

Presently, there are no disease-modifying treatments for Huntington’s, with current therapies focused only on treating disease symptoms.

ISIS has done a consistently good job of delivering value-enhancing clinical and partner-related events. We are impressed with the neuro pipeline and its significant validation by two of the world’s leaders in neurobiology, Biogen and Roche.

However, we believe this value is not being reflected in the current stock price, which has lagged despite improving fundamentals.

In our view, the stock reflects some negative sentiment that has built over the past few months despite major pipeline advances.

The partnerships with Biogen and Roche and the recent publication in The Lancet are all positive indicators that, along with its 34+ compound pipeline and broad IP, will eventually reverse the consensus view.

With a deep and strong pipeline supported by world-class partners, in our view, our patience will be rewarded as the company continues to execute and deliver improving fundamentals.

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