8 Major FDA Decisions and Trial Results to Watch for in September

September 2, 2016 by 247chrislange

Pharmaceutical and biotech 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, should a study come back negative or 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 U.S. Food and Drug Administration (FDA) rulings and results from clinical trials can make or break these companies.

24/7 Wall St. has collected several big FDA decisions and mid-to-late-stage trials that should be coming up in September 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.

Achillion Pharmaceuticals

In mid-August, Achillion Pharmaceuticals Inc. (NASDAQ: ACHN) announced that interim results from its ongoing Phase 2a clinical trial evaluating all-oral combination regimens, containing odalasvir, AL-335 and simeprevir, which were accepted for presentation at the EASL — American Association for the Study of Liver Diseases (AASLD) Special Conference being hosted in Paris, France.

This study is being conducted in conjunction with Alios BioPharma, part of the Janssen Pharmaceutical Companies of Johnson & Johnson (NYSE: JNJ). The data will be presented on Friday September 23. This Phase 2a study is aiming to establish the safety, pharmacokinetics and efficacy of doublet and triplet regimens, consisting of odalasvir and AL-335, with or without simeprevir, in treatment naïve patients with genotype 1 hepatitis C virus (HCV) infection for treatment durations of eight weeks or less.

Shares of Achillion closed trading at $8.23 on Thursday, with a consensus analyst price target of $9.71 and a 52-week trading range of $5.57 to $10.95.

Johnson & Johnson was last seen at $119.08. The consensus price target is $126.72, and a 52-week range is $89.90 to $126.07.


Amarin Corp. PLC (NASDAQ: AMRN) announced that it completed the randomization of patients in the REDUCE-IT cardiovascular outcomes study with 8,175 patients. The study is evaluating whether treatment with Vascepa reduces cardiovascular events in patients who despite stabilized statin therapy have elevated triglyceride levels and other cardiovascular risk factors. Amarin closed enrollment at all global clinical sites in June 2016.

The company continues to expect the onset of the final primary cardiovascular event to occur in the second half of 2017 with publication of trial results likely in 2018. An independent Data Monitoring Committee is expected to complete a protocol pre-specified interim efficacy and safety analysis after approximately 60% of the target aggregate primary cardiovascular events have occurred within the study. This interim analysis most likely will occur in September 2016. A second interim efficacy and safety analysis with approximately 80% of the primary cardiovascular events is expected in mid-2017.

Shares of Amarin closed most recently at $2.79, with a consensus analyst price target of $6.83 and a 52-week trading range of $1.24 to $3.46.

Array BioPharma

At the end of June 2016, Array BioPharma Inc. (NASDAQ: ARRY) submitted a New Drug Application (NDA) for binimetinib to the FDA. It is planning for an Oncologic Drugs Advisory Committee meeting as part of the regulatory review process. Array is also currently preparing for an Application Orientation Meeting with the FDA in September 2016, which it expects will include a discussion of the NDA package including clinical risk/benefit.

Shares of Array were last trading at $3.52. The consensus price target is $8.00. The 52-week range is $2.38 to $6.01.

Mast Therapeutics

September is when Mast Therapeutics Inc. (NYSEMKT: MSTX) is planning to announce the top-line data from its EPIC study. There are 388 subjects randomized at study sites around the globe. Also EPIC was the largest placebo-controlled study in sickle-cell disease ever concluded. To support its NDA for vepoloxamer, Mast is continuing to enroll patients in EPIC-E, the repeat exposure study for EPIC patients, and in a clinical pharmacokinetics study of vepoloxamer in individuals with varying degrees of renal insufficiency.

Shares last closed at $0.52, in a 52-week range of $0.21 to $0.60. The consensus price target is $2.75.


Expectations are that Novavax Inc. (NASDAQ: NVAX) will announce top-line data from its Phase 3 Resolve trial in the third quarter of 2016. This is a pivotal Phase 3 trial of the RSV F vaccine in older adults (60 years of age and older). The Resolve trial is a randomized, observer-blinded, placebo-controlled trial in 11,850 older adults at 60 sites in the United States. The primary efficacy objective is the prevention of moderate-severe RSV-associated lower respiratory tract disease, as defined by the presence of multiple lower respiratory tract symptoms. Enrollment was completed back in the fourth quarter of 2015.

Shares of Novavax were most recently trading at $6.82, with a consensus price target of $14.49 and a 52-week range of $4.08 to $11.15.

Threshold Pharmaceuticals

Threshold Pharmaceuticals Inc. (NASDAQ: THLD) is planning to report preliminary results from its Phase 2 proof of concept studies with tarloxotinib, a hypoxia-activated epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), which is designed to selectively release an irreversible EGFR-TKI in hypoxic tumors.

Currently, Investigations are continuing in two proof-of-concept, Phase 2 clinical trials in patients with advanced non-small cell lung cancer and patients with metastatic squamous cell carcinoma of the head and neck and skin; the designs of both trials require a minimum response rate for study continuation.

The shares closed on Thursday at $1.05. The consensus price target is $2.00, and the 52-week range is $0.21 to $5.26.

Vertex Pharmaceuticals

A Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) Phase 3 clinical study has been completed. It was conducted to support the potential approval of Orkambi for children with cystic fibrosis within the past year. The study enrolled 58 children ages 6 to 11 with two copies of the F508del mutation, and the primary endpoint of the study was safety. In May the FDA granted Vertex’s request for a PDUFA Priority Review of this sNDA by the end of September.

Shares of Vertex were last seen at $94.75, within a 52-week range of $75.90 to $136.97. The consensus price target is $110.32.