If you're looking for stocks that can make dramatic gains quickly, it's hard to do any better than the biotechnology industry. Hardly a week goes by without at least one biotech stock shooting higher or tumbling lower in response to exciting news about experimental treatments.
In 2023, all three of these drugmakers will report study results that are awfully important to their futures. Just read on to see how upcoming trial readouts could push their stock prices to new all-time highs.
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1. Alnylam Pharmaceuticals
Alnylam Pharmaceuticals (ALNY 0.47%) is an innovative drug developer that could take a big leap forward in 2023. The company pioneered the use of small strands of RNA that interfere with the production of troublesome proteins and this has led to the launch of five new drugs in less than four years.
To date, all of Alnylam's treatments act in the liver because that's where drugs accumulate naturally. Unfortunately, many of the diseases the company would like to address must target tissues beyond the liver.
Last February, Alnylam started a clinical trial with early-onset Alzheimer's disease patients and ALN-APP. This candidate is administered directly into the fluid bathing the brain, where it should interfere with the production of proteins that break apart and accumulate into toxic plaques.
In the first half of 2023, Alnylam is expected to share early-stage trial results that would do a lot more than just tell us if ALN-APP is a viable Alzheimer's disease candidate. It could spark the development of candidates aimed at heaps of neurological conditions that lack treatment options.
2. UniQure
UniQure (QURE 0.52%) has a successful track record when it comes to developing new gene therapies, but marketing those therapies has been a different story. About five years ago it pulled its first drug to earn approval, Glybera, from the European market after it failed to generate significant sales.
This year, the company will have another chance to generate significant sales, except this time it will have some help. In November, UniQure and its collaboration partner CSL received approval to market Hemgenix. This is a gene therapy for a rare bleeding disorder called hemophilia B.
A successful launch for Hemgenix could help push UniQure stock higher in 2023. Investors also want to keep their eyes peeled for results from a Huntington's disease trial. Huntington's is a rare disorder that causes progressive brain damage for an estimated 30,000 Americans. Available treatments help manage the symptoms, but there still isn't anything approved to stop or even slow its progression.
In the second quarter, UniQure will present one- and two-year follow-up results from a phase 1/2 trial with Huntington's disease patients and an experimental therapy called AMT-130. Last June, the company reported 12-month data from patients treated with a relatively low dose that was more than encouraging. If patients enrolled in the higher dosage cohort experienced significantly reduced nerve damage, this stock will shoot higher.
3. Regeneron Pharmaceuticals
Regeneron Pharmaceuticals' (REGN 0.51%) anti-inflammation drug, Dupixent, is already a blockbuster treatment for eczema and asthma. It was first approved in 2017 and it's currently generating more than $7 billion in annual sales for Regeneron and its collaboration partner, Sanofi.
This year, Regeneron stock could shoot higher in response to upcoming results of a clinical trial with Dupixent and patients with chronic obstructive pulmonary disease (COPD). According to the World Health Organization, COPD is the third leading cause of death worldwide, but there's still a dearth of effective treatment options.
In the first quarter, Regeneron should be able to report top-line results from a study with 939 COPD patients who received standard care or Dupixent every other week for a whole year. If the annual rate of acute COPD exacerbations is significantly lower among patients who were randomized to receive Dupixent, the stock could soar.
At recent prices, you can scoop up shares of Regeneron for 17.4 times earnings. That suggests earnings will grow a little bit slower than the average stock in the S&P 500 index. At this modest valuation, investors have a pretty good chance of coming out ahead over the long run whether Dupixent succeeds as a COPD drug or not.
