Key FDA Decisions and Trial Results Coming in the Next 2 Months

September 14, 2016 by 247chrislange

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U.S. Food and Drug Administration (FDA) rulings and the results from clinical trials have proven time and again that they can make or break companies. The biotech and pharmaceutical companies that subject themselves to this lengthy process are taking on a fair amount of risk of failure, but if a drug is approved or passes a clinical trial, there can be massive upside.

24/7 Wall St. has collected several big FDA decisions and mid- to late-stage trials that should be coming up in late-September and October and added some color, along with the trading range and price target.

September signifies the end of the third quarter, when quite a few companies set their deadlines, although some could slip over into October.

For the month of October, perhaps the biggest headliner is the European Society for Medical Oncology (ESMO) 2016 Congress in Copenhagen between October 7 and 11.

Outside of the ESMO, 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.

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 also is 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 closed trading at $3.50 on Tuesday, with a consensus analyst price target of $8.00 and a 52-week trading range of $2.38 to $5.97.

Mast Therapeutics

Late 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 of Mast were last seen at $0.51, within its 52-week range of $0.21 to $0.71. The stock has a consensus price target of $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.

Novavax shares were last trading at $7.96. The consensus price target is $14.11, and the 52-week range is $4.08 to $10.70.

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. This should take place by the end of the third quarter.

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.

Shares of Threshold closed at $1.19, with a consensus price target of $2.00 and a 52-week range of $0.21 to $5.28.

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 supplemental New Drug Application (sNDA) by the end of September.

Vertex shares were last trading at $95.27. The 52-week range is $75.90 to $136.97, and the consensus price target is $110.32.


Exelixis Inc. (NASDAQ: EXEL) is scheduled to present data from its clinical trials of cabozantinib and cobimetinib, which will be subject to 15 presentations at the ESMO. Detailed results from the company’s CABOSUN trial will be presented at ESMO as a late-breaking abstract in the genitourinary tumors. Additional poster presentations will detail the investigation of cabozantinib in other cancer settings, including in combination with nivolumab in metastatic urothelial carcinoma and other genitourinary cancers, as well as the evaluation of cobimetinib in combination studies across multiple tumor types.

Shares of Exelixis closed most recently at $12.31, with a consensus price target of $10.67 and a 52-week range of $3.55 to $12.48.


In February, the FDA accepted Medivation Inc.’s (NASDAQ: MDVN) supplemental sNDA for Xtandi in metastatic castration-resistant prostate cancer (mCRPC), which includes findings from the Phase 2 Terrain and Strive Studies, to update the relevant clinical sections within the current indication. Xtandi is approved by the FDA for the treatment of patients with mCRPC. The FDA set the PDUFA goal date for Xtandi for October 22.

Shares of Medivation were recently trading up $80.96, with a consensus price target of $77.15 and a 52-week range of $26.41 to $81.04.

Celldex Therapeutics

Celldex Therapeutics Inc. (NASDAQ: CLDX) plans to present data from its glembatumumab vedotin study (glemba, or CDX-011) at the ESMO Congress as well. Enrollment for the program was completed and the primary endpoint was met in the Phase 2 single-agent study. The primary endpoint of the study, objective response rate, required a minimum of six responses in the first 52 patients to be deemed successful. Celldex is also evaluating glembatumumab vedotin in other cancers in which gpNMB is expressed.

Celldex shares were last seen at $3.58. The consensus price target is $8.00. The 52-week range is $2.96 to $18.62.

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Cerulean Pharma

Another company looking to present data at the ESMO is Cerulean Pharma Inc. (NASDAQ: CERU). Specifically, Cerulean will present data from the second group of patients from a Phase 1b/2 trial of CRLX101 in combination with weekly paclitaxel in platinum-resistant ovarian cancer. At the same time, the company will present data from an ongoing Phase 1 trial evaluating the weekly dosing of CRLX101.

Cerulean closed at $1.02 a share, with a 52-week range of $0.95 to $5.20 and a consensus price target of $3.29.