BOSTON (TheStreet) -- Listed below are 14 biotech and drug stock catalysts for the remainder of 2014. Healthcare investors (and traders) have had a lot to work with so far this year, and the next six months promises to be equally action packed.
Why are these 14 biopharma stock events more important than others? They may not be. Admittedly, my list is subjective, but I tried to highlight the most significant biotech and drug catalysts remaining for 2014. If you think I'm missing something crucial, let me know in the comments section below.
Biogen's anti-LINGO-1 antibody is a potential therapeutic game changer for chronic neuro-degenerative diseases like multiple sclerosis. The drug is designed to block the production of a protein known as LINGO-1, which when expressed in the central nervous system, leads to the degradation of the protective myelin sheath around nerve fibers. The breakdown or "demyelination" of nerve fibers interferes with the transmission of nerve impulses, leading to physical and cognitive disability.
If anti-LINGO-1 antibody can prevent the destructive LINGO-1 protein from being produced, nerve fibers in multiple sclerosis patients might be "remyelinated" and the damage caused by the disease reversed or eradicated altogether. No wonder anti-LINGO-1 is considered a crucial, if not the most important, compound in Biogen's research pipeline.
Biogen is running two phase II studies of anti-LINGO-1. Top-line results from the first study in patients with optic neuritis will be announced before the end of the year. These optic neuritis data are highly anticipated by investors as substantial proof of concept for the entire remyelination thesis and as a signal for what may come from the second, more important phase II study of anti-LINGO-1 in multiple sclerosis patients. Results from that study are expected next year.
This is the single-pill, once-daily hepatitis C cure everyone -- doctors, patients, investors -- has been waiting for. The approval decision is expected on (or before) Oct. 10. Gilead's sofusbivir (brand name: Sovaldi) has already broken records for the best, fastest, most amazing drug launch in bio-pharmaceutical history. Once the combination pill is approved (no more interferon or ribavirin necessary for most patients!), investors will watch to see whether Gilead can re-accelerate revenue and profits.
On a related note: Also mark Dec. 19 on your biotech catalyst calendar. That's when Abbvie (along with partner Enanta Pharmaceuticals) expects an FDA approval decision on its competing, all-oral hepatitis C therapy.
The jury is still out on the wisdom of Amgen spending $10 billion to acquire Onyx Pharmaceuticals. The verdict may rest in the outcome of two Kyprolis multiple myeloma studies expected this quarter. The "ASPIRE" study investigates the benefit of adding Kyprolis to Revlimid and dexamethasone in "first relapse" multiple myeloma patients. The ASPIRE study, with a progression-free survival primary endpoint -- was designed to confirm the accelerated approval of Kyprolis in the U.S. and to support the approval of the drug in Europe. The second study -- dubbed "FOCUS" -- compares Kyprolis to best supportive care in relapsed/refractory multiple myeloma patient and has an overall survival primary endpoint.
Intercept is one of the best-performing stocks of the year because the FLINT study of obeticholic acid (OCA) in nonalcoholic steatohepatitis (NASH) was stopped early following an interim analysis showing a significant patient benefit. But while Intercept shares traded as high as $445 immediately following the January announcement, the stock has drifted significantly lower (Wednesday close: $226.95) due to concerns about OCA's deleterious effect on cholesterol levels of patients in the study. The public disclosure of back-and-forth emails between Intercept executives and government scientists running the FLINT study have also weighed on the stock.
Many (but not all) questions about OCA's potential as a new NASH therapy should be answered when we finally see the detailed data from the FLINT study. When? An expected July disclosure has now been pushed back to August or September, according to Intercept. For sure, we know a full presentation of the OCA data will take place at the American Association for the Study of Liver Disease annual meeting in November.
ARC-520 uses RNA interference (RNAi) to interfere with the process by which the hepatitis B virus replicates. To date, all of Arrowhead's data on ARC-520 has been in mice and a single chimpanzee, but that didn't stop the company's market valuation topping $1 billion earlier this year (a sure sign of the often-crazy biotech bull market.)
Arrowhead's valuation has downsized more recently ahead of anticipated results in the third quarter from a phase IIa, single-dose, placebo-controlled study of ARC-520 in hepatitis B-infected human patients.
Exelixis shares have been in the dumps since March because of disappointment that the Cometriq "COMET-1" study wasn't stopped early for efficacy after an interim analysis of the survival data. The final analysis of the prostate cancer study is expected before the end of the year. Clearly, investor expectations for a positive outcome are low, which would make for quite a rebound in Exelixis' stock price if they manage to eek out a win.
MannKind secured FDA approval for Afrezza on June 27, so the bull-bear investor battle over the stock shifts to whether or not the company can find a marketing partner for the inhaled insulin device, and on what terms. MannKind executives have offered conflicting guidance on the timing of a partnership deal, but the company certainly needs to announce something well before the year is over.
Puma Biotechnology (PBYI
Results from phase III study of neratinib in adjuvant breast cancer
The neratinib adjuvant breast cancer phase III study compares one year of neratinib following 1 year of Herceptin versus 1 year of Herceptin alone. Essentially, the idea here is to determine if adjuvant breast cancer therapy with two HER2 inhibitors prolongs survival longer than a single HER2 inhibitor.
Earlier this year, a similar adjuvant breast cancer study of Herceptin and Tykerb failed to improve overall survival compared to Herceptin alone. The negative results from this study significantly lowered the probability of success for Puma's neratinib study, but like with Exelixis and Cometriq, investors wait for the actual results.
Bluebird Bio (BLUE
Gene therapy data in beta-thalassemia and sickle cell anemia
Bluebird wowed Wall Street in June with strong results (albeit from two beta-thalassemia patients) treated with its Lentiglobin gene therapy. As an encore, Bluebird is expected to present more data from its beta-thal study by the end of the year. Perhaps more importantly, Bluebird will also announce the first Lentiglobin data in patients with sickle-cell anemia, a much larger commercial opportunity.
Decision on appeal of FDA quashing of Vascepa Special Protocol Assessment
Amarin is pressing ahead on efforts to convince the FDA that it was wrong to reject expanded use of its fish-oil medicine Vascepa into the commercially lucrative high triglyceride patient population. As part of this effort, Amarin submitted an appeal to the top decision makers at FDA, requesting a reversal of the agency's previous ruling which voided the Special Protocol Assessment of the Vascepa "ANCHOR" study. Wall Street has largely left Amarin and Vascepa for dead, but if FDA does change its mind in the company's favor, the stock goes higher. Timing an FDA response to the appeal is tricky but we may hear something late summer or early fall.
I'm cheating a bit by combining four stocks into a single catalyst, but this is my column and I make the rules. All four companies are waiting on obviously important FDA drug approval decisions. Here are the relevant dates:
Keryx: Zerenex for kidney dialysis, Sept. 5.
Orexigen: Contrave for obesity, Sept. 11.
NPS Pharma: Natpara for hypoparathyroidism, FDA advisory panel on Sept. 12, approval decision date on Oct. 24.
InterMune: Esbriet for idiophathic pulmonary fibrosis, Nov. 27.
Results from phase III study of MDX in attention deficit and hyperactivity disorder (ADHD)
MDX (metadoxine extended release) is a new, non-stimulant therapy for ADHD. Alcobra is conducting two, phase III studies of MDX, the first of which is expected to be read out this summer.
The clinical development path for migalastat in Fabry disease has been rocky for Amicus, with more misses than hits to date. The company's next shot at turning around the drug's fortunes comes in the third quarter when it will release results from a head-to-head study comparing migalastat monotherapy to Genzyme's Fabrazyme. If the study is successful, Amicus intends to seek European approval of migalastat.
Following the flurry of clinical data on the PD-1 checkpoint inhibitors at June's ASCO annual meeting, the next big events are the the FDA approval decision for Merck's pembrolizumab in melanoma on Oct. 28 and the expected announcement of interim results from a Bristol study of nivolumab in squamous cell lung cancer. Those data should be available in the fourth quarter.