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Biotech Stock Mailbag: Pending Sarepta Results, Cyclacel

The BioRunUp trade in Acadia Pharmaceuticals (ACAD - Get Report) appears to be in full swing and working well as we get closer to the announcement at the end of November of top-line results from the phase III study of pimavanserin in Parkinson's Disease psychosis.

BioRunUp maven and TheStreet contributor Mark Messier reminds swing traders constantly that when stocks like Acadia trade up into important catalysts like clinical trial results, profits can be booked without needing to take on the added risk of holding through the event.

That's good advice because predicting the outcome of Acadia's pimavanserin study is challenging. Success hinges on whether or not you believe a higher dose of pimavanserin can be effective where lower doses have failed.

In September 2009, a phase III study testing two doses of pimavanserin (10 mg and 40 mg) in patients with Parkinson's psychosis failed to demonstrate a statistically significant reduction in antipsychotic behavior compared to a placebo. Mean reductions in the Scale for the Assessment of Positive Symptoms (SAPS) endpoint was 5.9 points for placebo, 5.8 points for the 10 mg dose of pimavanserin and 6.7 points in the 40 mg pimavanserin dose.

At the time, Acadia blamed the study's failure on a better-than-expected placebo response. The company also noted that the 40 mg dose of pimavanserin showed promising signs of efficacy, particularly in patients enrolled from North American trial sites.

Which brings us to attempt No. 2. This time around, Acadia designed a phase III study in Parkinson's psychosis that only tests the 40 mg pimavanserin dose against placebo in patients recruited from North America. The primary endpoint is the same, a reduction in psychotic symptoms as measured by the SAPS score. The current phase III study just completed enrollment of 200 patients -- results to be announced at the end of November. The previously failed study enrolled 240 patients across three drug arms. This should mean the current study has stronger statistical power to detect a meaningful difference between pimavanserin and placebo.

Do you believe in Acadia's post-hoc finding from the failed phase III study suggesting that the higher pimavanserin dose is potent enough to succeed? Or, was this a false signal that will end in another phase III failure?

Regarding Vivus (VVUS - Get Report) and its newly launched obesity pill, Robert S. writes, "Thanks for the news on the Qysmia launch date. Will you be tracking the sales/prescriptions and share with your readers?"

I'll do my best. Tracking the Qsymia launch through third-party data services like IMS Health and Wolters Kluwer is made more difficult because Vivus is only selling the obesity pill through mail order pharmacies. Investors will be watching the Qsymia launch very closely -- as they will Arena Pharma's Belviq launch as soon as it takes place. I'll definitely pass along any information I can get my hands on.

Mick Waters asks a follow-up question to my discussion of Celsion (CLSN - Get Report) in last week's Mailbag: "Regarding the Feuerstein-Ratain rule, is the $300 million market cap measured the day prior to the phase III results being announced? Based on your rule, you would call the Celsion HEAT study to fail with the market cap under $200 million right now?"

We used four months prior to data announcement as the time point for the Feuerstein-Ratain rule. If you assume (guess) HEAT study results are coming in November or December, we'd look at Celsion's sub-$300 million market cap in July-August and predict Thermodox failure.

The rest of the Feuerstein-Ratain goes like this: Companies with market caps of $1 billion or more was the best predictor of phase III cancer drug success -- 78% of studies analyzed positive. The results for companies with markets caps greater than $300 million but less than $1 billion was mixed -- 18% success rate.

"Bear at Night" responded to the same Celsion discussion, questioning whether the Feuerstein-Ratain rule should apply to Thermodox. "Not sure the Feuerstein-Ratain rule should apply, since you studied companies with new drugs rather than drug delivery systems. Thermodox is just a way of releasing in high concentrations a drug [doxorubicin] that has been proven to work in primary hepatocellular carcinoma [liver cancer]. Compare Delcath Systems-- would their success count against the rule?"

It's a fair point. Thermodox is more a hybrid drug reformulation/delivery system than an entirely new drug like we addressed in the analysis of the Feuerstein-Ratain rule. That may make Celsion an exception to the rule, agreed.
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