2010 FDA Decisions to Watch

2010 FDA Decisions to Watch
By Brian Orelli
December 31, 2009 | Comments (7)

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Being a drug-company shareholder before the Food and Drug Administration makes decisions about those companies' drugs can be scary, but it can also lead to spectacular returns. If you have the guts to buy, here are a few drug companies hoping for those kinds of returns next year.

Exubera 2.0?

The biggest hurdle for Mannkind's (Nasdaq: MNKD) inhaled insulin, Afresa, probably won't be getting past the FDA near its PDUFA date of Jan 16. Insulin is a pretty basic drug, and Afresa seems to have a better profile than other options; unlike current offerings, it mimics natural insulin spikes seen in non-diabetics after they eat.

Instead, Mannkind's biggest obstacle will be on the marketing front, keeping from following in the footsteps of Pfizer (NYSE: PFE) and Nektar Therapeutics' inhaled insulin, Exubera. Not too many doctors were exuberant about Exubera; the drug/device registered a measly $12 million in sales during its first nine months on the market before Pfizer gave up and handed the drug back to Nektar.

Afresa is smaller than Exubera, and Mannkind is working on an even smaller device whose size will be a selling point. Ultimately, though, it will come down to whether doctors are interested in trying out a new product when the current offerings work well enough and whether patients request a needle-free alternative.

Mannkind may be able to get some traction, but I expect a slow uptake.

678 fewer injections
Amylin Pharmaceuticals (Nasdaq: AMLN) and Eli Lilly's (NYSE: LLY) Byetta is a great drug. It controls diabetics' glucose well, keeping them from having to go onto insulin. It even has an added benefit of causing weight loss.

The only problem is that the drug has to be injected twice a day. That's fairly burdensome compared with popping pills like Merck's (NYSE: MRK) Januvia, and sales have suffered, reaching only $679 million last year and falling slightly through the first three quarters of this year as some worries of pancreatitis have arisen.

The duo's solution is a once-weekly formula of Byetta using Alkermes' extended-release technology. The decreased injections should increase sales, but first they'll have to get past the FDA in March. The efficacy data looked good -- it beat Januvia and Takeda's Actos in head-to-head trials -- but the FDA may be leery of an approval because of the side-effect issue. Novo Nordisk's once-daily liraglutide, which is in the same class, has been waiting for an FDA approval since last March.

Once-weekly Byetta should be approved, but don't expect a speedy FDA decision.

2 years in the making
After the FDA's somewhat shocking denial of Dendreon's (Nasdaq: DNDN) prostate-cancer treatment Provenge in May of 2007, the company will have another date with the FDA on May 1 of next year.

The data the FDA previously requested turned out positive, so an approval seems more likely than not. The biggest risk is that the FDA finds something wrong on the manufacturing side of things. The treatment takes immune cells from a patient and trains them to become cancer-fighting machines before putting them back into the patient. The FDA needs to be certain that Dendreon can accurately repeat the process.

Here's hoping that Dendreon doesn't end up sending out another mayday to investors on May Day.

Potential obesity-drug approvals: size XXXL
2010 could see the FDA weighing in on three obesity drugs. Arena Pharmaceuticals and VIVUS (Nasdaq: VVUS) both submitted their applications to the FDA this month, and Orexigen Therapeutics expects to file in the first half of next year.

Of the three, VIVUS' Qnexa probably has the best weight-loss data, but Arena's Lorcaserin may have the best safety profile. The latter is important, since diet and exercise work pretty well as treatments for obesity and the agency is likely to be very safety conscious. An approval for any or all of the drugs isn't a slam dunk, but considering the large market, the risk could be worth the reward.

Usual disclosures apply
Keep in mind that the FDA sets up PDUFA dates, but they're not a deadline that the agency is required to meet. Trying to day-trade around the PDUFA date can be dangerous to your portfolio. A much better strategy is to think longer-term. Find a company you like at a price you can live with, and don't worry about any delays the FDA may throw at your investment.

Any other FDA decisions you're looking forward to in the new year? Let us know in the comment box below.

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Report this Comment On December 31, 2009, at 2:52 PM, norman1066 wrote: Thanks for an interesting article,

Afresa will have no trouble outselling Exubera. Pfizer's drug was more costly than injectable insulin yet was neither more effective nor safer. On the other hand Afresa works just as well, doesn't cause weight gain, and is measurably SAFER.

This last point is important as it will potentially allow physicians to prescribe Afresa earlier in type 2 diabetics disease progression. It is possible that Afresa could become the first line therapy (not in 2010 but possibly in a couple of years). If that occurs Mannkind will have a mega drug on its hands.

The big hurdle for Mannkind appears to be getting a big pharma partner for the drug as MNKD isn't your typical bio start-up eager to take any bone thrown its way. With Al Mann's deep pockets they have chosen to blaze their own trail right through to FDA approval. It should be entertaining to see what deal they eventually strike and how the street perceives it.

Report this Comment On December 31, 2009, at 3:33 PM, goyocafe wrote: "...when the current offerings work well enough..."

Comments like this only demonstrate a lack of appreciation for diabetes treatments that exist today. If this were true, why then is there such a huge untapped market for new treatments for this terrifying disease?

Report this Comment On December 31, 2009, at 4:34 PM, pharmagenius wrote: Why no mention of GENEREX's ORAL-LYN? Their oral insulin product is coming up for FDA approval in 2010. Its currently in Phase III trials in Canada and the U.S. They already have FDA approval for emergency use and ORAL-LYN is already in use in several countries.

Report this Comment On December 31, 2009, at 5:03 PM, JPG101 wrote: Generex doesn't actually sell insulin if you look at their balance sheet. They are in trouble in India. Management pays itself very well for a small company with no partnerships or institutional buy in.

I think this is a scam company that will go broke with time. The individual small investors will get seriously burnt with this company.

Report this Comment On January 01, 2010, at 5:03 PM, AlexKalman wrote: FOOL, MISSES PHASE III FOR SOME CANCERS?

Next Dendreon? Novelos' CEO Eyeing "Revolutionary" Phase III Results

Written by M.E.Garza

Wednesday, 16 December 2009 00:00

"What's interesting about Novelos is that we're in a Pivotal Phase III trial for lung cancer under both SPA and Fast Track for our NOV-002 lead compound," explains Novelos Therapeutics' (OTC: NVLT) Chief Executive Officer, Harry S. Palmin.

The study, a randomized, open-label, Phase 3 Trial of NOV-002 in Combination with Paclitaxel and Carboplatin vs. Paclitaxel and Carboplatin alone for the treatment of advanced non-small cell lung cancer is being conducted under the FDA's Special Protocol Assessment. It blinds Novelos from seeing any results as it progresses.

In other words, there is no interim analysis. There is only "one look" and that look occurs once 725 deaths take place during the trial.

"These patients with a median survival of eight to ten months unfortunately die pretty quickly," explains Novelos Therapeutics' Chief Executive Officer, Harry S. Palmin. "One year survival, which is another way to look at this, is just about 40%."

The pivotal study itself began more than three years ago, in November of 2006. By March of 2008, the enrollment target of patients had been reached and now, over a year and a half later, the 725th event has not yet occurred.

It's become pretty obvious, even to the most casual observer, that the patients involved in the trial are living longer than expected and that, in fact, is one of the reasons why Wall Street has begun to take more interest in the company and the study. Originally, most observers, including insiders at the company, expected that the trial might reach a conclusion some time during the middle of this year.

"It's a very unique value proposition," says Palmin. "If you think of Avastin, Roche's drug, it's the only drug in non-small cell lung cancer, first line treatment that showed a survival advantage. They showed a two month survival advantage, 12.3 months versus 10.3, but the drug has severe toxicities associated with it on top of the chemotherapy toxicities and it's very expensive. It's also only available to very few lung cancer patients because the histology it works for is non-squamous.

"The point that I'm trying to make is that given our current statistical projections, because our trial is going late, if we assume that the control group does in fact what it's supposed to, around a ten month median survival, and there's historical data on some four-thousand patients with advanced stage lung cancer that have taken our type of chemotherapy (paclitaxel/carboplatin [standard of care in the U.S.]), then we should be on track to achieve or possibly even exceed our possible twelve and a half month median survival target.

"By way of background, we started the pivotal Phase III on lung cancer on the back of three Phase II's," explains Palmin. "Same patient population and in two of the trials that were done in Russia, we saw dramatic survival advantages, better anti-tumor effects and in the United States, we saw doubling response rates and better toleration of chemotherapy. So there are three lung cancer trials that were the basis for the pivotal Phase III. We, of course, have applicability to other indications because we're not tumor specific and we're not chemotherapy specific."

That is an important point that has not gone unnoticed. This year, Perdue Pharma has invested over $19 million dollars in Novelos. The company itself, since going public over 5 years ago, has raised over $70 million dollars.

"We had good data out of Russia in non-small cell lung and it is the number one cancer killer, currently; late stage lung cancer," says Palmin. "There are north of two-hundred thousand new patients each year and more than one hundred and sixty thousand deaths and it's a huge market. Just lung cancer is north of a $3.5 billion dollar market.

"Since we have applicability, really, to all solid tumors, in Russia the drug is already approved for general use with chemotherapy. Here in the United States, we've had two positive Phase II results in ovarian and breast cancer. Channel 7 in Miami recently profiled a breast cancer patient that was in our Phase II breast cancer trial and went into complete remission. No signs of cancer. That's based on publicly available information where we've had forty percent of the patients have had no sign of their cancer after treatment of chemo and our drug.

"In addition, the patients that take our drug with the chemotherapy have a much better quality of life while under that treatment, versus being on chemo alone. Plus, very importantly, there is the efficacy improvement. That makes this a very different approach to the treatment of cancer."

Report this Comment On January 03, 2010, at 9:56 PM, larssral wrote: Chelsea Therapeutics (CHTP) is wrapping a phase three trial for Droxidopa (indication for orthostatic hypotension) which it successfully petitioned changes to the study's endpoint.

They were asked to conduct an additional trial, which should put them in place to submit an NDA by the end of 2010, if not sooner