The Life Sciences Report

WATCHLIST 2013

Which biotech companies will take off in 2013? To zero in on possible winners, The Life Sciences Report teamed up with Sagient Research to identify companies with potential catalysts on the calendar. The results were sent to experts who, based on their experience in the space, could determine which companies had the best chances for upward stock price movement. The result was the groundbreaking Life Sciences Report Watchlist 2013. Now a quarter into 2013, the Watchlist has been updated with the most current information and catalysts.

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Company Drug Disease Catalyst
Ariad Pharmaceuticals Inc. (ARIA:NASDAQ) Iclusig (ponatinib)
FDA approved Dec. 14, 2012


















AP26113
Chronic myelogenous leukemia (CML)


















Non-small cell lung cancer
Favorable phase 3 EPIC trial data could result in approval for first-line use in CML in Q4/13.

Approval of Iclusig in the EU in Q3/13; company expects to be "launch-ready" by July 1, 2013.

Regulatory submissions for Iclusig in Canada, Switzerland and Australia in H2/13

Regulatory submission in Japan in mid-2014

Data from interim analysis of phase 3 EPIC trial comparing Iclusig to imatinib (Gleevec) in newly diagnosed CML expected in mid-2014

Clinical updates at the American Society of Clinical Oncology (ASCO) meeting in June

Clinical updates at European Society of Medical Oncology in September
Amarin Corporation plc (AMRN:NASDAQ) Vascepa (icosapent ethyl)
FDA approved July 26, 2012
Hypertriglyceridemia

Mixed dyslipidemia
Quarterly sales figure catalysts

Acceptance of supplemental new drug application (sNDA) for mixed lipidemia by mid-May
Medivation Inc. (MDVN:NASDAQ) Xtandi (enzalutamide)
FDA approved Aug. 31, 2012
Prostate cancer

Chemo-naive metastatic castration-resistant prostate cancer
Quarterly sales figures

Mid-2013: Positive phase 3 PREVAIL study data
Trius Therapeutics Inc. (TSRX:NASDAQ) TR-701 (tedizolid phosphate) Complex skin and skin structure infections, including methicillin-resistant Staphylococcus aureus New drug application (NDA) in H2/13

FDA acceptance of the NDA filing

FDA approval in mid-2014
Sangamo BioSciences Inc. (SGMO:NASDAQ) SB-728-T HIV/AIDS Preliminary phase 2 data due in H1/13

Full data set from phase 2 trials by end of 2013
Pharmacyclics Inc. (PCYC:NASDAQ) ibrutinib (PCI-32765) Chronic lymphocytic leukemia (CLL)

B-cell lymphomas
Phase 3 RESONATE trial of 350 patients in ibrutinib monotherapy versus ofatumumab in relapsed or refractory CLL/small lymphocytic lymphoma with primary endpoint of progression-free survival improvement. First readout from interim analysis is anticipated in Q1/14.
Navidea Biopharmaceuticals Inc. (NAVB:NYSE) Lymphoseek (technetium Tc 99m tilmanocept)
FDA approved March 13, 2013
Intraoperative detection of diseased lymph vessels and nodes Next catalysts will be based on uptake of Lymphoseek by surgeons and revenue associated with that uptake
Celsion Corp. (CLSN:NASDAQ) ThermoDox (liposome-encapsulated doxorubicin) Hepatocellular carcinoma (primary liver cancer) Phase 3 failure reported on Jan. 31, 2013; future uncertain
Onyx Pharmaceuticals Inc. (ONXX:NASDAQ) Kyprolis (carfilzomib)
FDA approved July 20, 2012

Stivarga (regorafenib; multikinase inhibitor)
FDA approved Sept. 27, 2012
Multiple myeloma




Metastatic colorectal cancer (mCRC)

Locally advanced, unresectable or metastatic gastrointestinal stromal tumors
Quarterly sales data




Revenue upside related to March 25, 2013 approval in Japan

Revenue upside related to February 2013 approval in the U.S.
Sarepta Therapeutics Inc. (SRPT:NASDAQ) Eteplirsen Duchenne muscular dystrophy (DMD) Protocol development of phase 3 trial in H2/13

FDA to decide if eteplirsen would be considered for accelerated approval

End-of-phase 2 meeting with FDA is now anticipated for Q3/13
Hyperion Therapeutics Inc. (HPTX:NASDAQ) Ravicti (formerly HPN-100 (glycerol phenylbutyrate)
FDA approved Feb. 1, 2013
Urea cycle disorders Product is shipping; success of commercial rollout
Prana Biotechnology Ltd. (PRAN:NASDAQ) PBT2 (a hydroxyquinoline derivative) Alzheimer's disease

Huntington's disease
Phase 2b data in Q4/13

Phase 2a data in Q3/13
Peregrine Pharmaceuticals Inc. PPHM:NASDAQ Bavituximab (first-in-class; targeting phsophatidylserine) First-line non-small cell lung cancer (NSCLC) in combination with carboplatin and paclitaxel

Second line NSCLC; in combination with docetaxel

Investigator-sponsored phase 1 trials in HER2(-) breast cancer with paclitaxel and in liver cancer with sorafenib
Median overall survival results on phase 2b trial in Q2/13





Final results of salvaged data from phase 2b trail in Q2/13 (most likely at ASCO)


Phase 3 trail could begin by year-end; data possible in Q2/13

Data possibly in Q2/13
Celldex Therapeutics (CLDX:NASDAQ) rindopepimut (formerly CDX-110)



CDX-011 (fully human antibody conjugated to chemo agent monomethyl auristatin E)

CDX-1401

CDX-1127

CDX-301
gliobastoma (GBM) (EGFRvIII-expressing)



Triple-negative breast cancer (GPNMB expressing)




Solid tumors

Solid tumors

Hematopoietic stem cell transplant
Phase 2 (proof-of-concept) data in patients with aggressive GBM with rindopepimut + Roche AG's Avastin (bevacizumab)

Initiation of phase 3 trial in H2/13






Completion of phase 2

Completion of phase 1 in H2/13

Complation of phase 1 accrual in H2/13

Galena Biopharma Inc. (GALE:NASDAQ) NeuVax (nelipepimut or E75)




FBP(folate binding protein-E39)
Breast cancer prophylaxis to prevent recurrence in disease-free patients expressing low levels of HER2

Prophylaxis for tumors expressing folate receptor alpha
Phase 3 update of PRESENT trial enrollment in H1/13





Now in phase 2b, data from the phase 1 trial will be announced at American Society of Clinical Oncology (ASCO) meeting in June
Celgene Corp. (CELG:NASDAQ) CC-10004 (apremilast)



Pomalyst (pomalidomide)
FDA approved Feb. 8, 2013

Revlimid (lenalidomide)


Abraxane
Rheumatoid and psoriatic arthritis



Multiple myeloma (MM)
Myelofibrosis


Multiple myeloma



Pancreatic cancer
Melanoma
FDA submission for psoriasis and psoriatic arthritis in H1/13. Submission for approval in the EU in H2/13

Approval in the EU in H2/13

Phase 3 data in H1/13


MM-020 study (H2/13) could result in European approval in first-line MM

File sNDA
Mature overall survival data
NewLink Genetics Corp. (NLNK:NASDAQ.GM) HyperAcute Pancreas (algenpantucel-L)

Indoximod
Pancreatic cancer



Metastatic breast cancer
Results of interim analysis of IMPRESS trial in H1/13


Phase 2 enrollment update
Information for this chart was provided by Mara Goldstein/Cantor Fitzgerald, Raghuram Selvaraju/Aegis Capital Corp. and company websites. George S. Mack, The Life Sciences Report