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2017 Small-Cap Biotech Watchlist Update: Holding Steady in a Volatile Market

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The Small-Cap Biotech Watchlist, composed of 20 companies targeting a range of indications including blood cancers and solid tumors, irritable bowel disease and orphan diseases, has posted a modest gain since the first of the year.

As of April 19, the Watchlist was up 3%. In the same period, the NASDAQ Biotechnology Index (NBI) was up ~9.8%, and NYSE Arca Biotech Index (BTK) was up ~14.5%.

The biggest gainers of the quarter included Actinium Pharmaceuticals Inc., Blueprint Medicines Corp. and Pieris Pharmaceuticals Inc. Companies that posted significant losses included Catalyst Biosciences Inc., OncoMed Pharmaceuticals Inc. and Sorrento Therapeutics Inc.

Companies on the Watchlist were selected by analysts George Zavoico of JonesTrading Institutional Services, Raghuram (Ram) Selvaraju of Rodman and Renshaw, and Michael King of JMP Securities and introduced at the 2017 Biotech Showcase, held in San Francisco in January.

Abeona Therapeutics Inc. (ABEO:NASDAQ): Abeona has three ongoing gene therapy clinical programs targeting life-threatening rare diseases. News from Q1/17 includes the granting of orphan drug designations in the European Union, for treatment of recessive dystrophic epidermolysis bullosa, a skin disorder, and juvenile Batten disease, a lysosomal storage disease. The stock is up ~4% since the start of the year.

Actinium Pharmaceuticals Inc. (ATNM:NYSE.MKT): Actinium is in a pivotal Phase 3 trial with its lead candidate, Iomab-B, a radioimmunotherapy used prior to a bone marrow transplant in relapsed and refractory acute myeloid leukemia patients over the age of 55. Its second radioimmunotherapy, Actimab-A, is also in clinical study, and in February, the company announced a clinical trial of Actimab-M in multiple myeloma. The stock is up 76% since the first of the year.

ArQule Inc. (ARQL:NASDAQ): A developer of targeted small molecules, ArQule's lead candidate, tivantinib, failed to meet its primary endpoint of improving overall survival in the treatment of liver cancer, according to a company press release in March. Earlier this month, the company announced FDA clearance for an investigational new drug application (IND) for a Phase 1 trial of ARQ 531 in blood cancers. The company's stock is down ~24% over the quarter.

Asterias Biotherapeutics Inc. (AST:NYSE.MKT): This regenerative medicine company is addressing spinal cord injury and cancer with a dendritic cell therapy using genetically engineered autologous and allogeneic stem cells. Asterias reported improvements in motor function over six months in patients with spinal cord injury after treatment with its AST-OPC1 candidate. The stock is down ~33% since Jan. 1.

BioSpecifics Technologies Corp. (BSTC:NASDAQ): BioSpecifics' lead candidate is injectable collagenase for treatment of Dupuytren's contracture and Peyronie's disease. The compound, developed to remove scar tissue, is also in development for additional conditions including uterine fibroids, cellulite, and frozen shoulder. The company announced initiation of a Phase 1 trial for Xiaflex in uterine fibroids on April 18. The stock is down ~6% since the first of the year.

Blueprint Medicines Corp. (BPMC:NASDAQ): Blueprint is developing kinase inhibitors targeting various cancers, including liver cancer and gastrointestinal stromal tumors. In March, the company announced it had dosed the first patient in a Phase 1 trial of BLU-667, which will include patients with non-small cell lung cancer and medullary thyroid cancer. The company is up ~64% over the first quarter.

Catalyst Biosciences Inc. (CBIO:NASDAQ): This company is targeting hemophilia and surgical bleeding with injectable compounds that encourage the blood to coagulate and clot. Since the beginning of the year the company has announced a reverse stock split and a public offering, as well as approval of an IND in South Korea for its next-generation coagulation Factor IX variant. The stock is down ~43% since Jan 1.

Edge Therapeutics Inc. (EDGE:NASDAQ): Edge Therapeutics is targeting aneurysmal subarachnoid hemorrhage with lead product candidate, EG-1962, in a Phase 3 clinical study. In March, the company updated progress in its study of intracisternal administration of EG-1962. The stock is down ~29% over the quarter.

Epizyme Inc. (EPZM:NASDAQ): Epizyme's focus is epigenetics, and the pipeline is led by tazemetostat, in clinical trials for the treatment of non-Hodgkin's lymphoma and mesothelioma. The stock is up about ~23% over the quarter.

Karyopharm Therapeutics Inc. (KPTI:NASDAQ): The company's lead candidates include oral selinexor for cancer and oral verdinexor for viral indications including HIV. The company announced interim results in its Phase 2 trial for selinexor in acute myeloid leukemia in March. Karyopharm is up ~19% since the start of the year.

Loxo Oncology Inc. (LOXO:NASDAQ): Loxo is targeting the inhibition of genetic drivers in various cancers, and is in Phase 2 with larotrectinib (LOXO-101) in solid tumors. In March, the company announced proof-of-concept data for the compound in patients with glioblastoma. Loxo is up ~40% over the first quarter.

OncoMed Pharmaceuticals Inc. (OMED:NASDAQ): OncoMed is developing biologics against cancer stem cell pathways and immuno-oncology targets. Lead candidate demcizumab is in Phase 2 trials for pancreatic cancer and non-small cell lung cancer, but reported failure to meet endpoints in both indications during the quarter. OncoMed has declined ~48% since the start of the year.

Pieris Pharmaceuticals Inc. (PIRS:NASDAQ): With a proprietary biologics platform in the field of anticalins, Pieris is targeting multiple therapeutic areas including anemia, asthma and immuno-oncology. Pieris is up ~70 percent since Jan. 1.

Sorrento Therapeutics Inc. (SRNE:NASDAQ): Focusing on intracellular immunotherapies and immuno-oncology antibodies, Sorrento's pipeline includes a number of clinical and preclinical intracellular immunotherapies and immune-oncology antibodies. The company reported Phase 1 data for treatment of liver cancer in April. Sorrento has declined ~61% in the first quarter.

Stemline Therapeutics Inc. (STML:NASDAQ): The company is working to identify compounds that destroy cancer stem cells, and is in clinical trials with SL-401 in blood cancers. Stemline announced completion of enrollment in its Phase 3 study in blastic plasmacytoid dendritic cell neoplasm in March. Stemline is down ~16% since the first of the year.

Syndax Pharmaceuticals Inc. (SNDX:NASDAQ): Lead candidate entinostat is in clinical trials in a number of indications, including breast cancer and melanoma. Syndax is up 48% year to date.

Synergy Pharmaceuticals Inc. (SGYP:NASDAQ): Synergy's lead drug candidate, plecanatide, has completed Phase 3 trials in chronic idiopathic constipation; dolcanatide is in development for the treatment of opioid-induced constipation and inflammatory bowel disease. The company announced submission of a supplementary new drug application for plecanatide in irritable bowel syndrome with constipation in March. Synergy is down ~27% in the first quarter.

Syros Pharmaceuticals (SYRS:NASDAQ): The company's pipeline of gene control medicines targets blood cancers and solid tumors. Syros announced FDA acceptance of its IND for SY-1365, advancing the compound into Phase 1. The stock is up ~23% since the first of the year.

Threshold Pharmaceuticals Inc. (THLD:NASDAQ): Lead candidate evofosfamide is a prodrug activated by the hypoxic conditions surrounding cancer tumors; a second compound, TH-3424, targets cancers that overexpress a particular enzyme. The company announced a merger with Molecular Templates (private) in March. Threshold is up ~27% since the first of the year.

WAVE Life Sciences Ltd. (WVE:NASDAQ): WAVE's platform of therapies based on stereopure nucleic acid therapeutics target Duchenne muscular dystrophy, Huntington's disease and inflammatory bowel disease. The stock is down ~12% since Jan. 1.

All percent increases or decreases were current as of market close on April 17.

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1) Tracy Salcedo compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. She owns, or her family owns, shares of the following companies mentioned in this interview: None. She is, or members of her immediate household or family are, paid by the following companies mentioned in this article: None.
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