2 Biotechs Face Key Decisions in Mid-Fourth Quarter
October 21, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Although there are plenty of biotech companies facing trial updates or major regulatory decisions in the fourth quarter of this year, Market Exclusive chose two to keep a close eye on. Let’s take a look.
1. Keryx Biopharmaceuticals
Boston-based Keryx Biopharmaceuticals is focused on developing new treatments for renal disease. The company’s lead asset, Auryxia (ferric citrate) is approved to treat hyperphosphatemia, a chronic kidney condition. The company had some issues in August with its supply lines, but it appears to have been addressed.
Auryxia is approved in patients who are on dialysis. They conducted trials on the drug in patients with hyperphosphatemia and chronic kidney disease (CKD) who were not on dialysis, and if approved for those patients, would expand the population for the drug. It also presented data earlier this year from an expansion trial. The study evaluated Auryxia for the treatment of iron deficiency anemia (IDA) in adults with stage 3-5 non-dialysis dependent chronic kidney disease (NDD CKD). That study met its primary endpoints, and all secondary endpoints.
“Based on these results,” said John Neylan, chief medical officer of Keryx, in a statement, “we plan to submit an sNDA to the FDA in the third quarter, seeking to expand the label for ferric citrate to include the treatment of iron deficiency anemia in people with stage 3-5 NDD CKD. We believe that the ability to treat iron deficiency anemia, managing hemoglobin and iron levels, could have an important effect on the way kidney specialists treat these patients.”
The decision on the sNDA is expected this quarter. Keryx is currently trading for $4.64.
2. ProQR Therapeutics
ProQR Therapeutics is headquartered in Leiden, the Netherlands, with an office in Palo Alto, California. The company utilizes its technology platform to develop treatments for severe genetic disorders. It currently has QR-010 in two different Phase I clinical trials for cystic fibrosis (CF). It also has QR-110 in preclinical development for Leber’s Congenital Amaurosis (LCA), a genetic disease that causes childhood blindness.
The company plans to present data from the two Phase I trials at the North American Cystic Fibrosis Conference (NACFC), which is being held in Orlando, Florida on October 27-29. Market Exclusive writes, “Yes, it’s early stage, but CF is a potentially rewarding market, and the above mentioned conference is a big deal. If the company can draw some eyeballs to a potentially first in class therapy, then we think there’s some momentum behind the news.”
ProQR is currently trading for $6.45.
Other companies expecting potential catalyst news this quarter include The Medicines Company , which will provide an update on two programs on November 15, Dynavax Technologies , which is waiting for a decision by the FDA on or before December 15 on Heplisav-B for hepatitis B in patients with type 2 diabetes, and Cempra Pharmaceuticals , which has a PDUFA date on December 27 for an oral version of Solitherea for community-acquired bacterial pneumonia (CABP), and December 28, for an injectable form of Solithera for CABP.
That’s a busy date. France’s Advanced Accelerator Application (ADAP) has a PDUFA data on December 28 for Lutathera to treat gastroenteropancreatic neuroendocrine tumors (GEP-NETs).