This Day On The Street
Continue to site
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here

Biotech Stock Mailbag: Sarepta, F-R Rule Stock Predictions, Merrimack

Perhaps the next shoe to drop will be an announcement from Prosensa (RNA) that it will also seek FDA approval for its DMD drug drisapersen. The company has been re-analyzing data from the phase II and phase III studies conducted by old partner GlaxoSmithKline (GSK), clearly with the intent to file on its mind.

I see no reason why FDA would agree to review Sarepta's application but say no to Prosensa. Prosensa shares are up more than 50% this year, so clearly, investors are anticipating a drisapersen FDA filing as well.

To answer the next, logical question: No, I don't view a drisapersen FDA filing as negative for Sarepta or eteplirsen. The prospect of a two-day, all DMD FDA advisory panel sometime next year is incredibly exciting. I believe eteplirsen is the superior drug, so a drisapersen FDA review, even a dual approval, isn't a problem in my mind.

Douglas R. asks:

Must Read: Aegerion's Rare Disease Drug Launch Stalls

There has been a lot of debate about Merrimack Pharmaceuticals (MACK - Get Report) and its pancreatic cancer drug. What are your thoughts about the clinical trial results?

Some background will be helpful. MM-398 wraps the "old" chemotherapy drug irinotecan in spheres of liposomes, or fats, which is supposed to increase the absorption of the drug inside tumors where it can be most effective and minimize exposure in surrounding, healthy tissue, therefore reducing potential for side effects.

The study, dubbed NAPOLI-1, enrolled pancreatic cancer patients no longer responding to treatment with gemcitabine. One third of the patients were randomized to receive MM-398 on its own. A second third of patients received MM-398 and two chemotherapy drugs --  5FU and leucovorin. The remaining patients -- the control arm of the study -- received 5FU and leucovorin.

As reported by Merrimack, treatment with MM-398 plus 5FU/leucovorin resulted in a statistically significant, 33% reduction in the risk of death (hazard ratio 0.67) compared to the 5FU/leucovorin patients. The median overall survival in the MM-398 combination arm was 6.1 months vs. 4.2 months for the control arm -- a survival benefit of just under 8 weeks. No difference in survival was reported between the MM-398 monotherapy arm and the control patients.]

Is this study strong enough to get MM-398 approved by FDA? Merrimack bulls say yes, especially if the full data (not yet presented) match what we've seen from the company's press release. An eight-week survival benefit isnt much but would likely be deemed clinically meaningful for advanced pancreatic cancer patients who no longer respond to first-line therapy. MM-398 is the first drug to show a survival benefit in the second-line setting, where no drugs are formally approved.

The bears and skeptics say a significant risk and potential review issue for MM-398 -- maybe an interesting discussion at an advisory panel -- will be whether Merrimack gamed the design of the phase III study to boost the drug's chance for a positive result.  MM-398 is reformulated irinotecan, so why didn't Merrimack compare the efficacy and safety of the two drugs in the phase III study?

I asked Merrimack CEO Bob Mulroy this question. He insists MM-398 is a novel cancer drug. It is not just a reformulation of old irinotecan, so comparing the two drugs in a clinical trial wasn't necessary.

There was no pushback or questions from the FDA or the EMEA about the control arm of the phase III study, said Mulroy. Merrimack didnt pursue a Special Protocol Assessment (SPA) for the MM-398 phase III study because overall survival is the gold standard primary endpoint, he added.

While there are no drugs formally approved for second-line pancreatic cancer, the 5FU/leucovorin combination is listed in treatment guidelines, hence Merrimack's decision to make it the control arm of the phase III study.

Yet a triple-combination therapy of 5FU, leucovorin and irinotecan known as Folfiri has been studied in first and second-line pancreatic cancer with overall survival data comparing favorably to MM-398. You can find examples of Folfiri studies here, here, here and here.

2 of 3

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
Stocks Under $10

David Peltier uncovers low dollar stocks with serious upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

David Peltier identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Options Profits

Our options trading pros provide over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • Actionable options commentary and news
  • Real-time trading community
RNA $18.65 0.00%
CTIC $1.58 0.00%
EXEL $5.90 0.00%
MACK $10.15 0.00%
SNSS $1.17 0.00%


Chart of I:DJI
DOW 16,351.38 +293.03 1.82%
S&P 500 1,948.86 +35.01 1.83%
NASDAQ 4,749.9790 +113.8740 2.46%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs