The pharma stock leaders can at times seem overly risk averse with their approach to developing new medicines and cures. With the difficulty of clinical trials and the uncertainty of Federal Drug Administration (FDA) approval, many pharma companies prefer to play it safe rather than bet big. But not everyone is content making minor changes forever.
There are still revolutionary drug stocks out there fighting to be noticed. These companies may be much smaller than their competitors, but their dreams are much bigger. Companies that promise to treat the untreatable and cure the incurable are working to bring entirely new classes of medicine to market. Some of these revolutionary drug stocks are backed by Nobel Prize laureates. Others have proven their business acumen in a tough monetary environment. But all have brought new drugs, new ideas and a determination to succeed.
An increasing number of pharma stock leaders are running into the limits of what old technology can bring us, and more people are born every year who will go on to develop conditions that some believe may never be cured. But cancer was once seen as a death sentence and polio was once seen as just a fact of life. Nothing is impossible when science and business work hand in hand.
So for those who don’t want to invest in a stereotypical pharma company but know that biotech is the future, here are three revolutionary pharma stock leaders you won’t want to miss.
CRISPR Therapeutics (CRSP)
Shares in CRISPR Therapeutics (NASDAQ:CRSP) have gained ground this year while many pharma stock leaders have lagged. In a world of higher interest rates, it seems investors are less keen to bet on revolutionary drug stocks. But CRISPR Therapeutics has what its competitors lack: a proven drug and a pipeline to create many more.
Just a week ago, CRISPR Therapeutics and its collaborator Vertex Pharmaceuticals (NASDAQ:VRTX) announced the FDA approval of CASGEVY, the first FDA approved CRISPR-Cas9 based therapy. This approval puts them in the driver’s seat of one of the biggest revolutions in medicine. The ability to directly edit genes using CRISPR-Cas9 means previously untreatable diseases can now be cured. With this discovery, new markets are being blown open.
But with new markets, comes new challenges. The newly-approved CASGEVY is a treatment for sickle cell anemia and beta thalassemia. It’s no accident these were targeted as they are genetic diseases of the blood. CASGEVY works by the comparatively easy process of removing a small sample of a patient’s blood, altering it with CRISPR-Cas9 then injecting it back into the patient. It’s much harder to remove and replace organs, so treating non-bloodborne genetic diseases will be more challenging.
Nevertheless, getting the first FDA approval for a CRISPR-Cas9 therapy shows CRISPR Therapeutics has the expertise to meet those challenges. The approval of CASGEVY is good news for CRISPR Therapeutics and great news for patients. Watch what the company follows it up with, because CRISPR Therapeutics has catapulted itself to being a pharma stock leader.
Revolution Medicines (RVMD)
Sometimes a revolution in drug design requires a long look at old data. The rat sarcoma (RAS) family of genes was discovered in the 1960s and they cause up to one third of all human cancers. With so many diseases having a single known cause, a cure could be revolutionary. But RAS-caused cancers are highly resistant to treatment, and even considered “undruggable.”
But Revolution Medicines (NASDAQ:RVMD) has a deep pipeline of RAS-targeting drugs. The company has an entirely new way of looking at RAS and RAS-caused cancers. Pharma stock leaders have long seen their drugs fail because RAS simply escapes binding to drugs and remains active. But Revolution Medicines has found that RAS can’t escape when it’s held by two other proteins in the body. These two proteins plus RAS form a tri-complex, which Revolution Medicines has targeted for drugging. An innovative solution to one of medicines most-studied problems.
Revolution Medicines doesn’t just have good science, it also have good business sense. Clinical trials are expensive, and so Revolution Medicines has raised cash by buying its competitor EQRx in an all-stock deal. EQRx’s intellectual property ( ) will be sold off and Revolution will remain solely committed to its RAS-based therapies.
The EQRx deal will be good for Revolution’s balance sheet. Its most recent earnings statement shows $455 million in cash and cash equivalents. But that has swollen by about $1 billion now that EQRx’s cash is added to the pile. With a quarterly net loss of $108 million, this ensures Revolution has the cash to pursue its novel RAS-based strategy.
Everything is undruggable until it isn’t, and Revolution Medicines is one of the revolutionary drug stocks you won’t want to miss.
Multiple myeloma may not be the most common cancer, but for the 35,000 new patients every year, it remains sadly incurable. But Arcellx (NASDAQ:ACLX) is leading a new era with its drugs that target B-cell maturation antigen (BCMA). BCMA is relatively rare in normal cells, but highly expressed in tumors. And higher BCMA correlates with lower odds of survival. This makes BCMA a key target for treating this untreatable disease.
Arcellx doesn’t just have science on its side, it also has deep-pocketed investors. The pharma stock leader Gilead (NASDAQ:GILD) recently bought $200 million worth of Arcellx stock and gave a cash payment of $85 million on top of that. And Arcellx could be eligible for more milestone payments down the line.
Those milestone payments will rely on CART-ddBCMA, for which Arcellx just announced positive results in phase one clinical trials. Phase two trials are ongoing, with an estimated primary completion date of May 2024. That means an investment now could reap a big reward very soon.
In the short term, Arcellx’s price will likely move based on its big-name trials completing in 2024. In the long term, it has an even deeper pipeline of drugs behind that. Bringing those drugs to market will take time and money though. Arcellx’s most recent earnings report shows $440 million in cash and investments with a net loss of $39.3 million. But that was before the Gilead investment, and the Gilead investment plus milestone payments down the line should give Arcellx all the time it needs to succeed.
Multiple myeloma is currently incurable, but it doesn’t have to be. Revolutionary drug stocks like Arcellx are changing that and bringing treatment to even the most stubborn diseases.
On the date of publication, John Blankenhorn did not hold any positions (either directly or indirectly) in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.