Healthcare Business

3 Stocks With Catalysts That Could Capitalize on Biotech Momentum

Biotechnology is on the move. A familiar bull market pattern since 2008 may be about to reestablish itself. That is, the S&P 500 is moving higher, outpaced by the Nasdaq, which itself is about to be outpaced by biotech since all three bottomed in early February. If that pattern is confirmed, then a move back to the all-time highs for the iShares Nasdaq Biotechnology Index ETF (NASDAQ: IBB) within the next 12 to 18 months is a considerable possibility.

If the momentum in biotech can continue, then companies with catalysts within this timeframe could get an added boost with any good news, as more investors will be paying attention now that the bearish sentiment seems to be abating. Here are three companies with near-term catalysts worth a look now.

uniQure

uniQure N.V. (NASDAQ: QURE) just announced its most recent full-year financials, and alongside the release it provided an update on the progress of its Phase 1/2 in hemophilia B. The trial is testing AMT-060, a gene therapy that could ultimately replace the current standard of care (SOC). Hemophilia is a genetic condition that causes a blood clotting deficiency, which translates to small cuts and bruising becoming potentially fatal in sufferers.

A mutation in a gene called hFIX causes the lack of clotting, and current SOC relies on the frequent injection of the factor that derives from this gene. It is expensive (up to $340,000 annually in the United States) and painful. With AMT-060, uniQure is using a viral vector to transport working versions of the hFIX gene into a patient. If successful, it could result in natural production of the factor that otherwise requires repeated injections.


The company gave some preliminary top-line data from a low-dose arm of the trial in January. The data were thin, but positive, and served to offer up some level of efficacy on which the later, higher doses can expand upon.

The near-term catalyst just announced relates to the higher dose arm of the trial. uniQure expects to release data from the first five patients this quarter, and if there is an expansion of the efficacy seen in the low dose, some upside is possible.

CytRx 

CytRx Corp. (NASDAQ: CYTR) just gave an update on its lead oncology candidate, aldoxorubicin. The company is trialing the drug in a global Phase 3, with a target indication of second-line soft tissue sarcomas. The drug is part of an already established family of cancer drugs called anthracyclines chemotherapy drugs. Along with most chemotherapies though, anthracyclines aren’t particularly specific to cancer cells, so that the drug is associated with low tolerability and some serious side effects. With aldoxorubicin, CytRx is attempting to improve the selectivity of this family of drugs, making them more tolerable and safer for patients.

The near-term catalyst has to do with an announcement that the ongoing trial had hit the 191 checkpoints it was required to before the U.S. Food and Drug Administration (FDA) would accept any data analysis. This means CytRx can now use the data to form the basis of a new drug application and pave the way toward an FDA approval and commercialization. The data are set for release before the end of the quarter.

Corbus Pharmaceuticals

Corbus Holdings Inc.’s (NASDAQ: CRBP) lead candidate is an autoimmune target called Resunab. The drug is already in three Phase 2 trials for cystic fibrosis, systemic sclerosis and skin-predominant dermatomyositis, all fairly rare conditions, if lucrative. However, the company just announced that it has extended to a fourth one, systemic lupus erythematosus (SLE), which is much more common. In all these conditions, what is essentially an overactive immune system causes inflammation in various parts of the body, and Resunab is a synthetic analog of a natural anti-inflammatory shown to be up to 100 times more powerful than its natural counterpart.

SLE affects up to 323,000 individuals in the United States, and currently it has no real treatment outside of corticosteroids that can be toxic. The latest indication essentially has expanded Resunab’s potential patient target population by more than 300,000, and the National Institutes of Health will be funding the new trial, so there is little financial risk for Corbus. Data on the other three Phase 2 trials are expected later this year.

By Matt Winkler