According to the California Biomedical Research Association (CBRA), it takes roughly 12 years for a drug to move from lab to patient (give or take). Only about 5 in every 5,000 candidate compounds make it from pre-clinical testing to Phase I clinical trials. Of those 5 drugs, only one of them will make it to market approval, on average. Thus, there is about a 1 in 5,000 chance that any given pre-clinical drug candidate will make it to market1. With these odds, it’s extremely exciting when a company sees a drug candidate progressing through clinical trials.
In the past week, 3 companies have been making some pretty big headlines with the progression of their drug candidates through clinical testing.
In a report in Nature, investigators at the University of Dundee Drug Discovery Unit, working in collaboration with the non-profit foundation Medicines for Malaria Venture, published evidence of potent anti-malarial activity for a novel compound DDD107498, This compound has the potential to inhibit protein synthesis at several stages of the Plasmodium life cycle. Because of its broad activity, this drug has the potential to not only prevent the spread of malaria through patients who are infected, but to protect patients from developing the disease with a single dose. Merck KGaA plans to put this drug into Phase I trials as soon as possible.2
BioMarin is currently evaluating a compound called BMN-111 as a way to boost growth in children with achondroplasia (the commonest form dwarfism). On June 17th, the company announced positive results from a Phase II trial. Among 26 children, 10 showed a mean increase in annual growth velocity of 50% when compared to a 6-month baseline. In the simplest terms, the drug is showing promising results to put patients back on a normal growth rate. BioMarin’s stock rose 8% on the news. The company is setting up trials to establish safety at different dosage levels, and the design of a Phase III study to demonstrate efficacy has also begun.3
Synergy Pharmaceuticals is currently conducting a Phase III trial for an investigational drug to treat chronic idiopathic constipation. Plecanatide is an oral treatment which is being studied as an agent to normalize gastrointestinal function. Results from a Phase II study showed that two evaluated doses (3 mg and 6 mg) were both very effective compared to placebo. The 3 mg dose showed an increase in GI function by 21% while the 6 mg dose showed an increase in GI function by 19.5%. These outcomes compare favorably to the 10.2% increase in GI function observed in a placebo group. This success resulted in a 50% increase in company stock and a promising outlook for a potential blockbuster drug.4 However, there are two other drugs already on the market for the treatment of this condition, so there will be real questions about the efficacy of plecainaide compared to linaclotide (Linzess) and lubiprostone (Amitiza).
- California Biomedical Research Association, Fact Sheet, New Drug Development Process, 2014
- John Carroll, FierceBiotech, Merck KGaA hustling a promising anti-malaria drug into human studies, June 2015
- John Carroll, FierceBiotech, BioMarin shines as dwarfism drug boosts growth in children, June 2015
- Damian Garde, FierceBiotech, Synergy soars on stellar constipation data, rekindling buyout chatter, June 2015