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3 Speculative Biotech Companies Primed to Pop

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By Keith Speights, The Motley Fool

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They don’t have any products on the market. They have little or no revenue. Earnings? Nada. Many people are betting that they will fail. And yet they could present some of the best investing opportunities on the market today. Here are three speculative biotech companies that appear to be primed to pop in the coming months.

All set to accelerate
With gains of 450% over the last year, one might be inclined to think that Sarepta Therapeutics has done all the popping it’s going to do. I don’t expect another run that large, but Sarepta still has plenty of upside potential.

The biotech company reported fantastic results in October from a phase 2 clinical study of eteplirsen in treating Duchenne muscular distrophy, or DMD. These results were so good that Sarepta is talking with the Food and Drug Administration about the possibility of moving ahead with accelerated approval. If the FDA gives early approval to eteplirsen, Sarepta’s stock will be off to the races again.

It’s far from a sure thing that the FDA will grant accelerated approval, though. To do so requires that the agency accept data from the phase 2 study showing eteplirsen’s increased production of dystrophin as a solid surrogate endpoint in lieu of demonstrating actual clinical efficacy. The FDA could determine that this data isn’t strong enough to warrant accelerated approval.

Even if Sarepta doesn’t win this faster path to market, I expect that eteplirsen will ultimately gain approval regardless. The drug looks to be a game-changer for DMD patients — and should be for Sarepta shareholders also. 

Zooming with Zerenex
Speaking of game-changers, Keryx Biopharmaceuticals could have one for end-stage renal disease, or ESRD, patients. Keryx announced outstanding results from a phase 3 study of Zerenex in January. The drug lowered serum phosphorus levels in ESRD patients significantly and demonstrated a good safety profile to boot.

Keryx shares are up a whopping 150% so far this year but have hovered around $7 per share for the past month. Is the stock poised for more big gains? I think so.

The biotech plans to submit a New Drug Application, or NDA, to the FDA in the second quarter. It will also follow up by mid-year with filing for European regulatory approval. Zerenex seems likely to garner positive decisions on both fronts. I suspect the submissions themselves could serve as mini-catalysts for the stock as investors are reminded about the potential for the drug.

There are some concerns, though. A couple of months ago, IPD Analytics questioned whether Zerenex would be granted New Chemical Entity, or NCE, status by the FDA. NCE status gives a drug five years of exclusivity. My view is that Keryx’s patent protection for Zerenex will be stout enough to protect the drug from generic rivals for quite a while even if NCE status doesn’t come through. 

Ready for a giant leap
MannKind stands closer than ever to taking a giant leap for its shareholders. The company expects …read more

Source: FULL ARTICLE at DailyFinance

Business, Finance, Occupation & Industry

3 FDA Decisions You Need to Watch in 2013

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By Keith Speights, The Motley Fool

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Sequester or no sequester, the folks at the Food and Drug Administration look to be quite busy in 2013. Their schedules are full with plenty of important decisions that could greatly impact the fates of biotech and pharma companies — and the fortunes of shareholders as well. Here are three FDA decisions that you should watch over the coming months.

1. Will Vascepa be allowed to play limbo?
Much ado has been made about when the FDA will decide on New Chemical Entity, or NCE, status for Amarin‘s cholesterol drug Vascepa and what that decision will be. However, a much more significant FDA decision this year for Amarin will be related to the supplemental New Drug Application, or sNDA, submitted by the company in late February for use of Vascepa in patients with less severe levels of triglycerides.

Vascepa, a highly refined fish oil pill, is currently approved for patients with severe hypertriglyceridemia, which involves triglyceride levels greater than or equal to 500 mg/dL. If the recently submitted sNDA is ultimately approved, patients with greater than or equal to 200 mg/dL triglyceride levels will be able to take Vascepa. This submission is extremely important for Amarin, because the number of patients in this group is around 10 times the size of those with severely high triglyceride levels. 

Amarin should hear from the FDA within the next couple of months about whether the sNDA has been accepted for review. If it is accepted, the final decision on approval for the new indication would be given in late 2013. If Vascepa is allowed to play limbo by lowering the bar for triglyceride levels, Amarin’s shares should skyrocket.

Antares Pharma anxiously awaits an FDA decision in October for Otrexup, a self-injection for the treatment of rheumatoid arthritis. The FDA announced on Feb. 27 that Antares’ NDA had been accepted for review.

Otrexup uses Antares’ Medi-Jet parenteral drug delivery system to allow patients to self-administer methotrexate, or MTX. An estimated 70% of the 1.3 million Americans with rheumatoid arthritis take MTX alone or with another therapy. MTX is available in tablet form or via injection, but better absorption of the drug is obtained through injection. Antares hopes to capture a nice chunk of the market for those patients who currently self-administer with conventional needles or pen injectors.  

Should the FDA approve Otrexup, the prospects for Antares seem to be quite good. I suspect that many patients will like the idea of taking MTX with the easier self-injection approach offered by the Medi-Jet technology. 

3. A Tivo for kidney cancer?
By the end of July, we’ll know if a “Tivo” for kidney cancer will become available. I’m referring to Tivopath, AVEO Pharmaceuticals‘ proposed brand name for its drug tivozanib. The FDA accepted the NDA for tivozanib in November and set a final decision date of July 28 of this year.

Tivozanib targets treatment of an advanced form of kidney cancer that occurs in more than 250,000 …read more
Source: FULL ARTICLE at DailyFinance