Spark’s Hemophilia A data indicate best-in-class profile

Last month Spark Therapeutics (ONCE) reported initial results for SPK-8011, its hemophilia A gene therapy program. Despite their preliminary nature, the data are very positive and put Spark in a strong position vs. Biomarin (BMRN) and its Hemophilia A gene therapy, BMN270. Providing the signal is corroborated with additional patients, results may have broad implications on the liver-targeted gene therapy. Continue reading

Gene therapy’s 2017 scorecard – No alarms and no surprises (please)

Readers of this blog know I have high hopes for gene therapy, a field with a checkered history but disruptive potential that may finally be ready for primetime. After two years of dramatic progress 2017 is shaping up to be a year of incremental progress, focusing more on establishing and validating results seen to date. Continue reading

Biotech portfolio update – Jumping into gene therapy

After a long summer break it is time to review recent events and update the portfolio. As far as clinical readouts go, my portfolio had a brutal summer with one complete P3 failure from Array Biopharma (ARRY), a mixed data set from Aurinia (AUPH) and a win from SAGE (SAGE) that resulted in limited share appreciation. This was offset by strong performance from Exelixis (EXEL), my biggest holding which is up 48% quarter to date.

For the remainder of 2016 I plan to gradually increase exposure to gene therapy, which I hope will become one of the industry’s primary growth drivers in the coming years. In parallel, as I am still pessimistic about the biotech field in general (R&D productivity, pricing, biosimilars…), I intend to keep my short ETFs and a significant cash position. Continue reading

Biotech selloff leaves Wall Street disillusioned

After 5 years of a raging bull market, more than 140 IPOs and tens of billions in proceeds, there is a debate on whether the violent selloff in biotech stocks is a hiccup or the beginning of a real correction. I have no idea where the sector is heading in the coming weeks but it seems like the overall sobering experience coupled with this month’s selloff changed Wall Street’s perception around biotech. Investors are finally realizing drug development is fraught with uncertainty and that biotech is an attractive but not infallible segment, which is why I expect the correction to continue in 2016. Continue reading

Biomarin – testing the boundaries of evidence-based medicine

As an outside observer to the rare disease community, I find the recent acquisition of Prosensa (RNA) by Biomarin (BMRN) puzzling. To me, Prosensa’s drisapersen was just another case where promising phase II results were not corroborated in phase III. This happens frequently with oncology drugs (metmab, iniparib, tivantinib, palifosfamide etc.) and typically leads to termination of the program. Judging by Biomarin’s optimism, drisapersen’s fate may be different, which raises questions regarding approval of drugs despite negative P3 outcome. Continue reading

ArQule –An ultra-rare opportunity emerges

With most of its market cap covered by cash, ArQule (ARQL) and its pipeline are receiving very limited appreciation. The negative sentiment stems from skepticism regarding tivantinib,  currently in phase III for liver cancer (partnered globally and in Japan with Daiichi Sankyo and Kyowa Hakko Kirin, respectively). The skepticism is based on the drug’s 2012 failure in lung cancer as well as uncertainty about its mechanism of action. Continue reading