Top Stocks to Look For on July's FDA Calendar
Biotech and pharmaceutical companies are generally involved in the lengthy process of getting their drug candidates to market through clinical trials. There is a fair amount of risk to a great amount of risk involved, should a study come back negative or should a candidate not be approved. Conversely, if a drug is approved or passes a clinical trial, there can be massive upside.
The long and short of the matter is that updates within this industry, as well as U.S. Food and Drug Administration (FDA) rulings, can make or break these companies.
24/7 Wall St. has collected several catalysts that are coming up on the calendar in the month of July and added some color, along with the trading range and price target. Note that, due to many outside and internal factors, there are no assurances that the dates will remain static. Some date changes are positive developments, and some can be disasters if a company is not deeply financed.
As a side note about the Prescription Drug User Fee Act (PDUFA): a Priority Review designation is granted to medicines that the FDA determines have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease.
Also in the coming weeks, the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress in Berlin will be taking place. ISTH is a global not-for-profit membership organization advancing the understanding, prevention, diagnosis and treatment of thrombotic and bleeding disorders. The congress hosts the world’s leading experts on thrombosis, hemostasis and vascular biology, presenting the most recent advances to improve patient care.
Spark Therapeutics Inc. (NASDAQ: ONCE) is scheduled to release interim data from its Phase 1/2 clinical trial of SPK-9001 for hemophilia B at the ISTH. The presentations from this event will take place between July 8 and 13. Overall this treatment is approaching hemophilia from the gene therapy perspective, as opposed to the preferred treatment of factor replacement in which the missing factor protein is put into the affected patient’s vein.
Shares of Spark were last seen at $59.74, with a consensus analyst price target of $70.44 and a 52-week trading range of $35.07 to $65.99.
UniQure N.V. (NASDAQ: QURE) is another company in attendance at the ISTH. Like Spark, uniQure is also a gene therapy company looking to treat hemophilia B. Matthew Kapusta, CEO of uniQure, commented:
The data to be featured in an oral presentation at ISTH now demonstrate long-term safety, efficacy and the potential for broad application of AAV5 in hemophilia B. We also look forward to the presentation of clinical data demonstrating successful and effective transduction in patients with pre-existing neutralizing antibodies to AAV5, further supporting our belief that our AAV5-based gene therapies may provide clinical benefits to nearly all patients suffering from hemophilia B and other severe diseases.
Shares of uniQure closed out the week at $6.19, within a 52-week range of $4.72 to $9.72 and a consensus price target of $12.82.
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) will be presenting data in a late breaking abstract session at the ISTH. Essentially, the firm will report interim results from its Phase 1/2 study of BMN 270, an AAV5-FVIII Gene transfer in severe hemophilia. This data will be presented on the morning of July 11. Management also seems very positive ahead of this presentation. Hank Fuchs, M.D., President Worldwide Research and Development at BioMarin, commented:
We are committed to further our ongoing clinical program with BMN 270, which has the potential to change the course of hemophilia A treatment, the most common form of the disease, and look forward to sharing our latest findings.
BioMarin shares were last seen at $90.82, with a consensus analyst target of $111.78 and a 52-week range of $77.04 to $102.49.
Kadmon Holdings Inc. (NYSE: KDMN) is presenting interim data from its ongoing Phase 2 clinical trial of KD025 for previously treated patients with chronic graft-versus-host disease (cGVHD). The data will be presented at the firm’s Research and Development day on July 11. For some quick background: cGVHD is a condition that might occur after a patient receives bone marrow or peripheral blood stem cells from another person. Specifically, the donated bone marrow or peripheral blood stem cells view the recipient’s body as foreign, and the donated cells/bone marrow attack the body.
Shares of Kadmon were most recently trading at $3.89. The stock has a 52-week range of $2.25 to $11.73 and a consensus price target of $10.54.
In June, Paratek Pharmaceuticals Inc. (NASDAQ: PRTK) announced positive efficacy data in a Phase 3 registration study in community-acquired bacterial pneumonia, demonstrating the efficacy and general safety and tolerability of IV to once-daily oral omadacycline compared to moxifloxacin. A Phase 3 registration study in ABSSSI comparing once-daily oral-only dosing of omadacycline to twice-daily oral-only dosing of linezolid was initiated in August 2016. This study completed enrollment in May 2017 and top-line data is expected in mid-July. Following this, the company plans to submit its new drug application (NDA) in the United States as early as the first quarter of 2018, with an EMA submission later in 2018.
Shares of Paratek were last seen at $24.10, with a consensus price target of $3.80 and a 52-week range of $9.80 to $25.95.
Ocular Therapeutix Inc. (NASDAQ: OCUL) announced back in February the resubmission of its NDA for Dextenza for the treatment of ocular pain occurring after ophthalmic surgery. This is in response to the FDA’s Complete Response Letter. The FDA determined that the NDA resubmission is a complete response and designated the resubmission as a Class 2 review, with a PDUFA target action date of July 19 for the potential approval of Dextenza.
Ocular closed out the week at $9.27 a share, in a 52-week range of $4.04 to $11.91 and with a consensus price target of $22.25.
Eagle Pharmaceuticals Inc. (NASDAQ: EGRX) submitted its NDA of Ryanodex (dantrolene sodium) for the treatment of exertional heat stroke (EHS) for review by the FDA back in March. At that time it was also granted a priority review designation. Now its come time for the FDA to announce its decision. The PDUFA date has been set for July 23.
Scott Tarriff, CEO of Eagle, said:
There is currently no approved pharmacological treatment for EHS. If Ryanodex is approved, Eagle will be the first to market with a potentially transformational therapy. EHS can strike anyone, but athletes, our military and outdoor workers are especially vulnerable. We look forward to working with the FDA throughout the review process and to their expedited decision in July 2017.
Shares of Eagle Pharma were last seen at $78.89. The 52-week range is $38.20 to $97.15, and the consensus price target is $91.75.