BPC December 17 update

Biotech Stock Watch List for week commencing December 19 2016.

Weekly watchlist

Biotechs continued to release key data last week with several binary events hitting the screens with contrasting success.

Achaogen, Inc. (NASDAQ:AKAO) brought a smile to investors to the long side when its shares soared over 148% following its announcement of positive data from both its CARE and EPIC Phase 3 trials of plazomicin, while Vericel Corporation (NASDAQ:VCEL) showed initial promise when it rocketed over 57% following the FDA approval of MACI before a public offering blew out much of the initial flame.

To the downside, however, shares of both Ophthotech Corp (NASDAQ:OPHT) and Proteon Therapeutics, Inc. (Nasdaq:PRTO) tumbled following news that they both failed crucial Phase 3 trials.

As we look forward to the catalysts ahead, some information as to why Portola Pharmaceuticals (Nasdaq: PTLA) is on the list below. The company announced on October 25 that it had submitted a New Drug Application (NDA) to the FDA for betrixaban for the treatment of venous thromboembolism (VTE). It is common procedure for the FDA to report back within 60 days whether the NDA has been accepted or not for filing; in other words by December 23 given that markets will be closed December 24-26. The overwhelming majority of NDAs are accepted for filing by the FDA. It is regarded as almost a given so there is very seldom any reaction to the company’s share price when it is announced that the FDA has accepted a company's NDA. However, on rare occasions a company is issued a RTF (refusal to file) letter when the FDA decides not to accept it.

In regard to Portola, there is some uncertainty whether or not its NDA will be accepted given the data released from its Phase 3 APEX trial. The trial consisted of three cohorts, with the protocol requiring a p-value of 0.05 or less in Cohort 1 in order to test Cohort 2, which also needed a p-value of less than 0.05 to test the overall population. The data showed that Cohort 1 achieved a p-value of 0.054, which did not meet the threshold. Cohort 2 and the overall study population achieved p-values of 0.029 and 0.006, respectively. Investors will be keenly awaiting the decision from the FDA likely towards the end of the upcoming trading week.

Full Biotech Stock Watch List noted below:

Drug Stage Catalyst

ACAD
Pimavanserin
Alzheimer’s disease psychosis

Phase 2 Phase 2 top-line data released December 20, 2016. Primary endpoint met. Key secondary endpoint missed.

ITEK
Trabodenoson
Primary open-angle glaucoma (POAG)

Phase 3 Phase 3 top-line data released January 3, 2017. Primary endpoint not met.

PTLA
Betrixaban (The APEX Study)
Venous thromboembolism (VTE) Prevention

NDA Filing PDUFA date under priority review June 24, 2017. Phase 3 data released March 2016 did not meet primary endpoint.

ANTH
Sollpura (liprotamase)
Cystic fibrosis who suffer from exocrine pancreatic insufficiency

Phase 3 Top-line data released December 27, 2016 - missed primary endpoint.

CEMP
Solithera - oral
Community-acquired bacterial pneumonia (CABP)

CRL CRL announced December 29, 2016. Further trials required.

CEMP
Solithera - intravenous
Community-acquired bacterial pneumonia (CABP)

CRL CRL announced December 29, 2016. Further trials required.

OTIC
OTIPRIO
Acute otitis externa

Phase 3 Phase 3 trial met primary endpoint - January 5, 2017. sNDA filing to be submitted 1H 2017.