4 Top FDA Decisions Expected in Q1

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Pharmaceutical companies generally are involved in the lengthy process of getting their drug candidates to market through clinical trials. There is a fair amount to a great amount of risk involved, as with biotech companies, 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 in biotech is that Food and Drug Administration (FDA) rulings can make or break these companies.

24/7 Wall St. has collected four companies big FDA decisions coming up in the first quarter of 2016 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.

Zafgen Inc. (NASDAQ: ZFGN) is expecting to report top-line data from its Phase 3 clinical trial for the treatment of patients with Prader-Willi syndrome (PWS). The objective of the study is to evaluate the efficacy and safety of beloranib in PWS patients over six months of randomized treatment, followed by a six-month open label extension. The trial includes 108 patients with PWS enrolled across 15 sites in the United States. Zafgen shares trading most recently at $6.29, with a consensus analyst price target of $18.57 and a 52-week trading range of $5.43 to $55.36.


Aquinox Pharmaceuticals Inc. (NASDAQ: AQXP) completed the enrollment of its Phase 2 clinical trial of AQX-1125 in mid-2015, and now the company expects top-line results from this Kinship trial to be released in the current quarter. AQX-1125 is Aquinox’s lead drug candidate. It accelerates a natural mechanism that has evolved to maintain homeostasis of the immune system and reduce immune cell activation and migration to sites of inflammation to combat atopic dermatitis. Shares closed trading at $12.48 on Thursday, with a consensus price target of $22.83 and a 52-week range of $1.38 to $55.75.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is scheduled to report Phase 3 results for pegvaliase (PEG-PAL) in the first quarter. PEG-PAL is currently in registration-enabling pivotal studies as a potential therapeutic option for adult patients with phenylketonuria (PKU). With the potential approval of PEG-PAL, this product combined with Kuvan will expand and globalize BioMarin’s leadership position by offering a wider range of treatment options to patients worldwide with PKU. Shares ended last week at $104.76, with a consensus price target of $147.88 and a 52-week range of $88.51 to $151.75.

Puma Biotechnology Inc. (NYSE: PBYI) is looking forward to proceeding with the regulatory filing for neratinib for the extended adjuvant treatment of breast cancer in the first quarter of 2016. The company expects to have a New Drug Approval in this quarter for its PB272. This also represents the first trial with a HER2 targeted agent that has shown a statistically significant benefit in the extended adjuvant setting, which Puma believes will provide a meaningful point of differentiation for neratinib in the treatment of HER2 positive breast cancer. Puma shares were last trading at $78.40. The consensus price target is $134.25. The 52-week trading range is $56.11 to $252.92.