Best of the Week
Most Popular
1. Investing in a Bubble Mania Stock Market Trending Towards Financial Crisis 2.0 CRASH! - 9th Sep 21
2.Tech Stocks Bubble Valuations 2000 vs 2021 - 25th Sep 21
3.Stock Market FOMO Going into Crash Season - 8th Oct 21
4.Stock Market FOMO Hits September Brick Wall - Evergrande China's Lehman's Moment - 22nd Sep 21
5.Crypto Bubble BURSTS! BTC, ETH, XRP CRASH! NiceHash Seizes Funds on Account Halting ALL Withdrawals! - 19th May 21
6.How to Protect Your Self From a Stock Market CRASH / Bear Market? - 14th Oct 21
7.AI Stocks Portfolio Buying and Selling Levels Going Into Market Correction - 11th Oct 21
8.Why Silver Price Could Crash by 20%! - 5th Oct 21
9.Powell: Inflation Might Not Be Transitory, After All - 3rd Oct 21
10.Global Stock Markets Topped 60 Days Before the US Stocks Peaked - 23rd Sep 21
Last 7 days
Peloton 35% CRASH a Lesson of What Happens When One Over Pays for a Loss Making Growth Stock - 1st Dec 21
Stock Market Sentiment Speaks: I Fear For Retirees For The Next 20 Years - 1st Dec 21 t
Will the Anointed Finanical Experts Get It Wrong Again? - 1st Dec 21
Main Differences Between the UK and Canadian Gaming Markets - 1st Dec 21
Bitcoin Price TRIGGER for Accumulating Into Alt Coins for 2022 Price Explosion - 30th Nov 21
Omicron Covid Wave 4 Impact on Financial Markets - 30th Nov 21
Can You Hear It? That’s the Crowd Booing Gold’s Downturn - 30th Nov 21
Economic and Market Impacts of Omicron Strain Covid 4th Wave - 30th Nov 21
Stock Market Historical Trends Suggest A Strengthening Bullish Trend In December - 30th Nov 21
Crypto Market Analysis: What Trading Will Look Like in 2022 for Novice and Veteran Traders? - 30th Nov 21
Best Stocks for Investing to Profit form the Metaverse and Get Rich - 29th Nov 21
Should You Invest In Real Estate In 2021? - 29th Nov 21
Silver Long-term Trend Analysis - 28th Nov 21
Silver Mining Stocks Fundamentals - 28th Nov 21
Crude Oil Didn’t Like Thanksgiving Turkey This Year - 28th Nov 21
Sheffield First Snow Winter 2021 - Snowballs and Snowmen Fun - 28th Nov 21
Stock Market Investing LESSON - Buying Value - 27th Nov 21
Corsair MP600 NVME M.2 SSD 66% Performance Loss After 6 Months of Use - Benchmark Tests - 27th Nov 21
Stock Maket Trading Lesson - How to REALLY Trade Markets - 26th Nov 21
SILVER Price Trend Analysis - 26th Nov 21
Federal Reserve Asks Americans to Eat Soy “Meat” for Thanksgiving - 26th Nov 21
Is the S&P 500 Topping or Just Consolidating? - 26th Nov 21
Is a Bigger Drop in Gold Price Just Around the Corner? - 26th Nov 21
Financial Stocks ETF Sector XLF Pullback Sets Up A New $43.60 Upside Target - 26th Nov 21
A Couple of Things to Think About Before Buying Shares - 25th Nov 21
UK Best Fixed Rate Tariff Deal is to NOT FIX Gas and Electric Energy Tariffs During Winter 2021-22 - 25th Nov 21
Stock Market Begins it's Year End Seasonal Santa Rally - 24th Nov 21
How Silver Can Conquer $50+ in 2022 - 24th Nov 21
Stock Market Betting on Hawkish Fed - 24th Nov 21
Stock Market Elliott Wave Trend Forecast - 24th Nov 21
Your once-a-year All-Access Financial Markets Analysis Pass - 24th Nov 21
Did Zillow’s $300 million flop prove me wrong? - 24th Nov 21
Now Malaysian Drivers Renew Their Kurnia Car Insurance Online With Fincrew.my - 24th Nov 21
Gold / Silver Ratio - 23rd Nov 21
Stock Market Sentiment Speaks: Can We Get To 5500SPX In 2022? But 4440SPX Comes First - 23rd Nov 21
A Month-to-month breakdown of how Much Money Individuals are Spending on Stocks - 23rd Nov 21
S&P 500: Rallying Tech Stocks vs. Plummeting Oil Stocks - 23rd Nov 21
Like the Latest Bond Flick, the US Dollar Has No Time to Die - 23rd Nov 21
Why BITCOIN NEW ALL TIME HIGH Changes EVERYTHING! - 22nd Nov 21
Cannabis ETF MJ Basing & Volatility Patterns - 22nd Nov 21
The Most Important Lesson Learned from this COVID Pandemic - 22nd Nov 21
Dow Stock Market Trend Analysis - 22nd Nov 21

Market Oracle FREE Newsletter

How to Protect your Wealth by Investing in AI Tech Stocks

New Way to Make Big Money in Biotech Stocks Sector

Companies / BioTech Jan 17, 2014 - 05:52 PM GMT

By: Money_Morning

Companies

Ernie Tremblay writes: Traditional pharmaceutical blockbusters like Pfizer's Lipitor treat millions of patients at relatively low cost. It's a high-volume business model that has kept the pharmaceutical industry afloat for a long, long time. But over the past decade, out of necessity, a new model has taken the industry by storm.

As big moneymakers, like Lipitor, reach the "patent cliff," their intellectual rights protection are evaporating, and generic drug makers are taking over their markets.


Big Pharma needs fresh drugs to take the place of those they're losing. But replacing these products with new ones is expensive. Most experts agree that it takes about $800M in capitalized costs to develop a single new drug. And frankly, the "easier" medical riddles, like treating high LDL cholesterol, have mostly been solved. The remaining tough ones, like cancer and Alzheimer's, will drive costs even higher.

So how do the major pharmaceutical companies meet the challenge? By letting small, smart start-up biotechs do the R&D legwork on new drugs, then either making distribution deals with them or buying the small companies out.

Here's what makes this new approach so lucrative for investors...

Taking the "Lamborghini" Approach...

Back in August 2012, I noticed that a tiny biotech called Aegerion Pharmaceuticals (Nasdaq: AEGR) was about to present lomitapide, the one and only drug in its pipeline, to an FDA AdCom (Advisory Committee). AdComs are catalysts, key points in the drug development regulatory process that can make a company's stock soar - or nosedive.

Lomitapide treats a genetic disease called homozygous familial hypercholesterolemia (HoFH), which affects mostly children. HoFH can rocket blood cholesterol levels up by 1,000% or more, and most people who have it die from heart disease by age 11. All do by age 30. It's a horrible affliction.

The AdCom for lomitapide would vote on whether to recommend that the FDA approve the drug for marketing in the U.S. The FDA is under no obligation to take an AdCom's recommendation, although it usually does.

The thing is, HoFH affects only one in a million people. That's maybe 2,000 people in the U.S. - almost no market at all.

So how could Aegerion possibly make money treating it? The answer, when you think about it, is obvious. Think Lamborghini, not Volkswagen.

The biotechs, for their part, have learned that it often makes more sense to develop products for "orphan" indications than for major diseases.

"Orphan" is a special designation the FDA gives to illnesses that affect fewer than 200,000 people. If you manufacture a drug that treats these diseases, you get special tax breaks and seven years of market exclusivity (instead of the usual five). And you have a much smaller market to penetrate.

Still, how can you make money treating conditions that afflict only a handful of people? Aegerion, like many similar firms, had the answer: they would charge $250,000 to $300,000 per patient per year for their drug. As I said, think Lamborghini.

Obviously, insurance companies would say no to those prices, right?

Not so. First, the cost savings they were realizing from major drugs going generic left them with extra cash to burn, so they could afford to pay for rare, orphan indications. And the great PR they could get from funding treatments that saved kids or other helpless victims was priceless.

...Spotted an Obvious Winner

So for lomitapide, I looked over the clinical trial data on the drug and was certain it would have no problem weathering the regulatory gauntlet. A couple of years earlier, I'd also had the privilege of meeting the scientist who developed the drug for clinical use, Daniel Rader, MD, at the University of Pennsylvania. He's the kind of guy you instantly believe in. He's serious, smart, and above all, passionate about helping sick kids. And there was something else: In his medical practice, he had more HoFH patients than any other physician in America - so he already had a significant portion of the market at his fingertips.

As far as I was concerned, Aegerion was a no brainer. Lomitapide had huge potential, not only for patients, but for investors. I recommended we add it to our Lifetime Subscribers' portfolio.

We entered the stock on Oct. 12, 2012, at $17.46.

As expected, the AdCom gave Aegerion a strong recommendation (voting 13 yea to 2 nay) and the price jumped. A month later, the FDA gave Juxtapid, as it was now called, its seal of approval, and AEGR went to $25. A year later, on Oct. 4, 2013, the stock reached an all-time high of $97.24, more than 5.5 times our original investment.

So I wasn't kidding. If you want to hit home runs in the bioscience sector, start by looking for small, unknown biotechs with single drug pipelines - because they can be goldmines.

Isolating Bioscience's Next Big Winners

When I'm setting out in search of a lucrative bioscience stock, here are some qualities I find really attractive:

  • Market cap. $250M-$2.5B. This is the sweet spot for growth. Less than $250M can mean low volume trading, which can be a problem if you need to sell. More than $2.5B makes a company less responsive to catalysts.
  • Single product pipeline. These companies can yield high returns because they carry high risk. If their drug succeeds, their investors can get rich rapidly. A failure, however, can mean doom, so you need to know what you're doing in evaluating the drug's potential to be effective, safe, convincing to the FDA, and marketable.Some of my recommendations, of course, are for companies that have richer pipelines. They yield less return, but they're safer bets for a balanced portfolio.
  • An upcoming catalyst. A catalyst, such as an AdCom, FDA approval date (called a PDUFA), or even results from a clinical trial, can make you a whole lot richer overnight. Last year, Sarepta Therapeutics (Nasdaq: SRPT) tripled its stock price overnight when the company announced positive results for a study of its Duchenne muscular dystrophy drug.
  • Orphan drug status. As I explained above, these drug candidates have real advantages over traditional "blockbuster" entries.
  • An extreme need. Everyone born with HoFH would eventually die from it, and before Aegerion's Juxtapid (lomitapide), no effective treatment existed. Patients were longing for it.
  • Little or no competition. ISIS Pharmaceuticals Inc. (Nasdaq: ISIS), in partnership with Genzyme Pharmaceuticals, was developing a drug, mimoperson, to compete with Juxtapid, but it was inferior in several ways (injectable rather than oral, more severe side effects). It also passed its AdCom, but barely, garnering a vote of 9 to 6. Juxtapid had a nearly clear field to play in.

The Profits Ahead

Next, of course, comes the tricky part. You've got to be able to read studies on experimental drugs, evaluate the often subtle nuances in the results, and develop a sense of what the FDA will or won't like. What are a new drug's benefits? Are those benefits real, or has the developer skewed the data? Are they superior to the current standard of care? What are the risks? How hard are patients and the medical establishment pushing for the passage of new therapy? Once the drug or device reaches the market, will insurance companies foot the bill? And will doctors use it?

Important questions all, and ones I consider carefully in evaluating every stock I recommend to my subscribers.

With over 30 years' experience writing about the latest developments in health, medicine, and related technologies, Ernie Tremblay has gained "insider" access to the world's top doctors, professors, and researchers. He's worked with Nobel-caliber doctors such as Yale's Karel Liem, Columbia's George Gaylord Simpson, and Harvard's William S. Beck, to name a few.

Ernie's edited hundreds of books on science, microbiology, and groundbreaking medical therapies for top publishers like Harper Collins, Penguin Putnam, and Prentice Hall. He's also co-authored and ghostwritten numerous books on everything from Alzheimer's to rheumatology.

Best of all, he's developed knowledge of the inner workings of the FDA drug approval process and knows precisely when a drug or technology is about to "pop" and its company's stock rocket upward in price. So stay tuned, you'll be hearing from Ernie a lot more now...

Source : http://moneymorning.com/2014/01/17/new-way-make-big-money-biotech/

Money Morning/The Money Map Report

©2014 Monument Street Publishing. All Rights Reserved. Protected by copyright laws of the United States and international treaties. Any reproduction, copying, or redistribution (electronic or otherwise, including on the world wide web), of content from this website, in whole or in part, is strictly prohibited without the express written permission of Monument Street Publishing. 105 West Monument Street, Baltimore MD 21201, Email: customerservice@moneymorning.com

Disclaimer: Nothing published by Money Morning should be considered personalized investment advice. Although our employees may answer your general customer service questions, they are not licensed under securities laws to address your particular investment situation. No communication by our employees to you should be deemed as personalized investent advice. We expressly forbid our writers from having a financial interest in any security recommended to our readers. All of our employees and agents must wait 24 hours after on-line publication, or after the mailing of printed-only publication prior to following an initial recommendation. Any investments recommended by Money Morning should be made only after consulting with your investment advisor and only after reviewing the prospectus or financial statements of the company.

Money Morning Archive

© 2005-2019 http://www.MarketOracle.co.uk - The Market Oracle is a FREE Daily Financial Markets Analysis & Forecasting online publication.


Post Comment

Only logged in users are allowed to post comments. Register/ Log in