While the major indexes muddled along over the last few days, several health-care stocks lit up the charts. Here are the most humongous health-care winners for the week.
All the excitement stemmed from a positive recommendation by a U.S. Food and Drug Administration advisory panel for Northera, Chelsea's symptomatic neurogenic orthostatic hypotension drug. The vote wasn't unanimous -- but it was overwhelmingly in support of the drug, with 15 in favor of recommending approval and only one vote against.
A little over a week ago, many were skeptical about Northera's chances. Now Chelsea appears to have a pretty good shot at gaining ultimate FDA approval, although the agency doesn't have to go along with the advisory panel recommendation. A thumbs-up could harbor even more possibility of good news later. Northera is also being studied in two mid-stage clinical trials targeting intradialytic hypotension and fibromyalgia.
No nonsense from nonsense
The show was on for PTC Therapeutics (NASDAQ: PTCT ) on Monday at the J.P. Morgan Healthcare Conference. It didn't take long for the race to be on as well. Shares soared 50% for the week, moving steadily upward each day.
PTC spent a lot of time at the conference talking about Atuleren, which targets genetic diseases caused by what is called a nonsense mutation. The drug is currently in a phase 3 study for treating Duchenne muscular dystrophy, or DMD. Around 13% of the DMD population suffers from a form of the disease resulting from nonsense mutations. Final recommendation from the European Medicines Agency for conditional approval of Atuleren in treating this indication is expected by the end of January.
Atuluren is also being studies in another late-stage trial targeting the most severe form of cystic fibrosis. Results from this study are anticipated to be available in 2016. Chelsea lays claim to being the only company with a drug in development focusing on class 1 cystic fibrosis, which is also caused by nonsense mutations.
Another good talk
PTC Therapeutics wasn't the only biotech getting a boost from an appearance at the J.P. Morgan conference. Stemline Therapeutics (NASDAQ: STML ) surged 44% for the week following the company's presentation on Thursday.
Stemline has two drugs in its pipeline of interest to investors. SL-401 targets multiple indications, including multiple myeloma and myelodysplastic syndrome, or MDS. The studies farthest along, though, focus on use of SL-401 in treating blastic plasmacytoid dendritic cell neoplasm, or BPDNC, and acute myeloid leukemia, or AML.
The company's other drug, SL-701, is a synthetic peptide vaccine that targets two indications -- pediatric malignant glioma and adult high-grade glioma. Stemline plans to advance SL-701 into late-stage clinical studies focusing on treatment of high-grade glioma in adults and children.
While our list highlights the three biggest health-care stocks of the week, it only seems fair to include a couple of honorable mentions this time around -- Sarepta Therapeutics (NASDAQ: SRPT ) and Cell Therapeutics (NASDAQ: CTIC ) .
Sarepta jumped 38% after (what a surprise) presenting at the J.P. Morgan conference. The company announced more positive results from a phase 2b study of DMD drug eteplirsen. Sarepta has suffered considerably since a disappointing FDA decision in November.
Cell Therapeutics wasn't far behind, with shares climbing 37% for the week. The company announced a couple of big developments on Monday. First, non-Hodgkin lymphoma drug Pixuvri received a positive reimbursement recommendation in Europe. Second, Cell Therapeutics regained full rights to both Pixuvri and cancer drug Opaxio from Novartis (NYSE: NVS ) .
There isn't any doubt in my mind as to the identity of the most humongous winner of all this week. It's clearly the J.P. Morgan Healthcare Conference. The event provided the opportunity for three of our five super performers. Biotechs need a good platform to tell their story -- and J.P. Morgan always delivers.
One humongous pick for 2014
There's a huge difference between a good stock and a stock that can make you rich. The Motley Fool's chief investment officer has selected his No. 1 stock for 2014, and it's one of those stocks that could make you rich. You can find out which stock it is in the special free report "The Motley Fool's Top Stock for 2014." Just click here to access the report and find out the name of this under-the-radar company.