January 4, 2016
By Alex Keown, BioSpace.com Breaking News Staff
NEW YORK – Four pharmaceutical companies are expecting results from upcoming trials that could be make or break for the first quarter of 2016. 24/7 Wall St. compiled a list of four companies that could send yield big profits, or send investors running.
Positive results would be welcome news for many investors who saw the Nasdaq Biotechnology Index make modest gains in 2015.
1. Zafgen
The company is waiting for results of a Phase III trial for the treatment of patients with Prader-Willi syndrome. Positive results could result in company stock surging, which would be welcome relief after the stock took a hit in December following news that a second patient taking part in the Phase III trial of ZAF-311 (beloranib) for the treatment of PWS died. That news followed an October death that caused the U.S. Food and Drug Administration to briefly halt the trial. It was determined that the cause of death in the first patient was “respiratory failure due to pulmonary emboli. However, it is not known if this event was related to treatment with beloranib,” it was reported in October. Zafgen’s stock is currently down, trading at $6.03 per share. The stock saw a high of $51.34 per share in March 2015.
2. Aquinox Pharmaceuticals
Aquinox is expecting results from its AQX-1125 during the first quarter of 2016. In June the company announced the bladder pain medication did not diminish pain in trial patients compared to those who took a placebo during a six week period of treatment, the company said. However, during a Phase I trial, the drug did show promise in treating pain. It is estimated there are between five and 15 million people afflicted with interstitial cystitis in the United States. There is currently no cure for interstitial cystitis. Janssen’s Elmiron, was approved for the treatment of interstitial cystitis in 1997. Aquinox stock is trading at $12.28 per share this morning, down from the opening price of $12.58 per share.
3. BioMarin Pharmaceutical
BioMarin is expected to report Phase III results for pegvaliase, for the treatment of Phenylketonuria (PKU), a rare inherited disorder that “causes an amino acid called phenylalanine to build up in your body. In October, BioMarin struck a deal with Merck Serono to reacquire all global rights to Kuvan and the experimental pegvaliase for more than $500 million. With the potential approval of pegvaliase, the two products combined will expand BioMarin‘s role in providing a wider range of treatment options to patients with PKU. Approved in 2007 in the U.S., Kuvan is a commercialized product for the treatment of patients with phenylketonuria (PKU). Pegvaliase is currently in registration-enabling pivotal studies as a potential therapeutic option for adult patients with phenylketonuria. Kuvan has Orphan Drug exclusivity in Europe until 2020. BioMarin’s stock is currently trading at $103.79 per share.
4. Puma Biotechnology
Puma is moving forward with the filing for its experimental drug PB272, also known as neratinib, for the extended adjuvant treatment of HER2-positive breast cancer. The company expects. The company believes it will receive a New Drug Approval from the FDA this quarter. PB272 is a potent irreversible tyrosine kinase inhibitor, for the treatment of patients with HER2-positive breast cancer and patients with non-small cell lung cancer, breast cancer and other solid tumors that have a HER2 mutation. Puma Biotechnology is currently at $76 per share.
