Image source: Flickr user stockmonkeys.com.
Clinical trial results can make or break biotech investors, and while there's no guarantee that studies under way at these companies will pan out, I think the odds could be in their favor. If so, then owning some shares in these three companies might prove to be profit-friendly in 2016.
No. 1: Ophthotech Corp (ISEE)
Although 90% of drugs entering human trials fail, only between 30% and 40% fail in final phase 3 studies, and for that reason, I tend to prefer investing in clinical-stage biotech companies that have late-stage trials under way, like Ophthotech Corp.
Ophthotech is conducting three phase 3 trials of Fovista, an adjunct therapy for the treatment of wet age-related macular degeneration (AMD). Two of these trials are evaluating using Fovista alongside Novartis' (NVS -1.64%) multibillion-dollar blockbuster eye-drug Lucentis, and a third trial is evaluating Fovista alongside Roche's Avastin, which is often prescribed off-label in wet AMD, and Regeneron's multibillion-dollar eye drug Eylea, another commonly used wet AMD drug.
Last year, Opthotech reported phase 2 results demonstrating that adding Fovista to Lucentis therapy boosted vision by 10.5 letters on a standard eye chart in wet AMD patients. That's about four letters better than Lucentis alone, and because of that showing, Novartis licensed Fovista's ex-U.S. rights from Ophthotech shortly thereafter in a deal that could ultimately be worth $1 billion, plus royalties on ex-U.S. sales, to Ophthotech.
Phase 3 results from Ophthotech's two Fovista plus Lucentis trials are expected in the fourth quarter of next year, and if they're as good as the phase 2 results, then a filing for approval could follow and lead to Fovista ending up as a key part of the standard-of-care in 2017. That would be especially true if the company's Fovista plus Avastin, and Fovista plus Eylea trials also succeed.
Since the market for wet-AMD drugs is valued at $6 billion per year and growing, an eventual approval in this indication could make this company worth far more than its current $2.4 billion market cap.
Image source: Portola Pharmaceuticals.
No. 2: Portola Pharmaceuticals (PTLA)
Portola Pharmaceuticals is also set to report make-or-break news next year.
The company has already filed for FDA approval of andexanet alfa, its medicine for reversing the anticoagulant effects of top-selling factor Xa inhibiting drugs, and trial results for betrixaban, its own factor Xa inhibitor, are expected in Q1 2016.
A decision from the FDA is expected on andexanet alfa next year, and if approved, it will be the only FDA approved reversal agent for the multibillion-dollar drugs Xarelto and Eliquis. Andexanet alfa could end up being stocked by every hospital in the country, but an even bigger opportunity may exist for betrixaban, because betrixaban is being studied for extended use in acute medically ill venous thromboembolism (VTE) patients.
VTE effects roughly 10 million people globally and accounts for between 100,000 and 300,000 deaths per year in the U.S., making it a major indication with a big unmet need for new treatment. Betrixaban's market opportunity is made even more intriguing by the fact that the control arm of its study is the current standard of care, Lovenox, rather than a placebo.
The 7,500 patient trial is expected to complete in the first quarter, and if it's successful, then Portola Pharmaceuticals could be a bargain, because before it lost patent protection, Lovenox was hauling in more than $4 billion annually in sales.
No. 3: Achillion Pharmaceuticals (ACHN)
Although Achillion Pharmaceuticals hepatitis C drug ACH-3102 is in phase 2 trials, rather than phase 3, I still think trial results expected next year could move the needle in 2016.
Currently, ACH-3102 is being studied in combination with Johnson & Johnson's (JNJ -0.46%) Olysio, which is an already FDA-approved therapy for hepatitis C, and ALS-335, a drug that works similarly to Gilead Sciences' blockbuster hep C drug, Sovaldi.
In prior phase 2 trials, ACH-3102 delivered 100% cure rates when used alongside Sovaldi in a 6-week treatment period, and since Olysio is already on the market, and ALS-335 has the same mechanism of action as Sovaldi, I think there's a good chance ACH-3102 will put up solid efficacy in this trial, too. If it does, Johnson & Johnson, which licensed rights to ACH-3102 earlier this year, could begin late-stage trials next year, or in 2017, that could bring this combination to the hepatitis C market by 2018.
That timeline could mean ACH-3102 is part of a hep C treatment regimen that offers both high efficacy and best-in-class patient treatment duration, a combination that would position it nicely to carve share away in a market that's worth more than $20 billion per year in sales.
