This is one of our periodic Five Year Watch columns, examining whether and why predictions of scientific progress were accurate, or hype.
In January 2011, Merrion Pharmaceuticals, an Irish drug company, trumpeted promising results with a compound it called Orazol. The drug for cancer of the bone and breast was, in fact, a twist on an existing Novartis (NVS) medication called zoledronic acid. Merrion claimed to have turned it from an injected compound into one that could be taken orally, thus greatly expanding its potential market.
Merrion envisioned blockbuster status for the product, which it said would be under patent until 2027 in a “multibillion dollar” niche. It also boasted of an “abbreviated development path” for the drug with European and US regulators.
“If approved, this drug would provide a new treatment, which could improve prognosis, in combination with existing treatments, for early-stage breast cancer patients,” John Lynch, then Merrion’s CEO, said in a piece in Drug Development & Delivery titled “On The Rise: Drug Development Companies You Should Know About.”
“Subject to a licensing partner and [Food and Drug Administration] approval, Phase 3 trials for Orazol would commence in 2011,” he added. “It is estimated (Edison Research) that Orazol could be on the market by 2016.”
Here we are in 2016. What happened?
Merrion must be glad Lynch hedged his comments thoroughly. Five years later, Orazol is not on the market, as Lynch suggested might be the case, nor has any new clinical trial begun. Despite claims that it had guidance from the FDA for how to undertake a critical Phase 3 trial to establish clinical effectiveness and safety for the drug, Merrion has yet to begin such a study. No such trial is listed on Clinicaltrials.gov, the definitive US database. (For those interested, Phase 1 and 2 trials typically involve small numbers of patients and look only at whether an experimental agent is safe and potentially effective enough to warrant a much larger Phase 3 trial on which the FDA can make a decision about approval. Drugs as a rule cannot get a thumbs up without Phase 3 data.)
What’s more, Merrion no longer controls Orazol. It sold the rights to the drug to Novo Nordisk (NVO), and last month announced that it would transform itself into an investment firm. In an email, John Fox, Merrion’s CEO, told STAT: “Merrion did not have the resources to develop Orazol through Phase 3 itself, and while we have had discussions with early-stage companies with respect to licensing [the drug], these had not come to fruition before negotiations with Novo Nordisk began in the summer of 2015.”
We asked Novo Nordisk if it planned to pursue Phase 3 studies of Orazol, but have not yet heard back.
The lesson here is not that drugs fail to live up to their initial promise. Indeed, the vast majority of experimental compounds never make it out of the beaker, let alone into early-stage clinical trials. Bringing a drug to market can cost hundreds of millions of dollars or more, so that’s to be expected.
Rather, the important point is that a drug company can trumpet the findings of clinical studies, and, in this case, make come-ons to potential investors or partners, as well as patients who might join clinical trials. All of those implicit promises about the wondrous effect of an emerging product are fair game, but investors and patients are exactly the kind of people who need to realize that most of these predictions are too good to be true.