The collection of small-cap biotechs on the 2017 Watchlist are developing therapies to address diverse indications including rare diseases, asthma, inflammatory bowel disease, spinal cord injury, hemophilia, and a number of cancers. The platforms are equally diverse, representing traditional biologics, cell therapies and vaccines.
The panelists who selected the 2017 Small-Cap Biotech Watchlist are George Zavoico of JonesTrading Institutional Services, Raghuram (Ram) Selvaraju of Rodman and Renshaw, and Michael King of JMP Securities.
The analysts will discuss their choices in depth at the Biotech Showcase 2017, running Jan. 9-11 at the Hilton San Francisco on Union Square downtown. The Watchlist panel discussion will take place at 8 a.m. on Wednesday, Jan. 11.
The Approval Process in Action Infographic
Michael King of JMP Securities selected these companies:
Pieris Pharmaceuticals Inc. (PIRS:NASDAQ); ~$65M market cap. Pieris Pharmaceuticals has a proprietary biologics platform in the field of anticalins. Multiple therapeutic target areas for the therapy include anemia, asthma and immuno-oncology.
WAVE Life Sciences Ltd. (WVE:NASDAQ); ~$723M market cap. This company is focused on preclinical development of a platform of therapies targeting Duchenne muscular dystrophy, Huntington's disease and inflammatory bowel disease. Its platform is based on stereopure nucleic acid therapeutics. The company is making a return appearance on the Biotech Watchlist.
OncoMed Pharmaceuticals Inc. (OMED:NASDAQ); ~$308M market cap. OncoMed has platform discovery technologies aimed at creating novel biologics against cancer stem cell pathways and immuno-oncology targets. It is in clinical development with seven internally discovered compounds; lead candidate demcizumab is in Phase 2 trials for pancreatic cancer and non-small cell lung cancer.
Syndax Pharmaceuticals Inc. (SNDX:NASDAQ); ~$135M market cap. Lead candidate entinostat is a small molecule that the company says has “direct effects on both cancer cells and immune regulatory cells,” and could boost a patient's immune response to cancer. It is in clinical trials in a number of indications including breast cancer and melanoma.
Blueprint Medicines Corp. (BPMC:NASDAQ); ~$890M market cap. Blueprint is in clinical trials, as well as discovery phase development, of kinase inhibitors targeting cancer including liver cancer and gastrointestinal stromal tumors.
Epizyme Inc. (EPZM:NASDAQ); ~$713M market cap. Epizyme's pipeline is led by tazemetostat, which is in clinical trials for the treatment of non-Hodgkin's lymphoma and mesothelioma. The company's development platform is based on epigenetics.
Karyopharm Therapeutics Inc. (KPTI:NASDAQ); ~$372M market cap. The company is in clinical trials with two compounds targeting blood cancers and solid tumors. Its lead candidates include oral selinexor for cancer and oral verdinexor for viral indications including HIV.
Loxo Oncology Inc. (LOXO:NASDAQ); ~$720M market cap. Loxo is targeting the inhibition of genetic drivers in various cancers. It is in Phase 2 studies with lead candidate larotrectinib (LOXO-101) in solid tumors.
Ram Selvaraju of Rodman & Renshaw selected the following companies:
Abeona Therapeutics Inc. (ABEO:NASDAQ); ~$207M market cap. Making a second appearance on the Biotech Watchlist, Abeona has three ongoing gene therapy clinical programs targeting life-threatening rare diseases with multiple orphan drug and rare pediatric disease designations: two AAV-based one-time intravenous injection therapies and one gene-corrected skin graft therapy; and a next generation proprietary AAV-based vector platform. George Zavoico of JonesTrading also is recommending Abeona Therapeutics.
BioSpecifics Technologies Corp. (BSTC:NASDAQ); ~$385M market cap. BioSpecifics' lead candidate is injectable collagenase, which is being marketed in the U.S. and elsewhere 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.
Sorrento Therapeutics Inc. (SRNE:NASDAQ); ~304M market cap. With a focus on intracellular immunotherapies and immuno-oncology antibodies, Sorrento's pipeline includes a number of clinical and preclinical monoclonal antibodies and CAR-T therapies. It is in its second year on the Watchlist.
Stemline Therapeutics Inc. (STML:NASDAQ); ~$215M market cap. This company's discovery platform, StemScreen, is being developed to identify compounds to target and destroy cancer stem cells, Stemline is in clinical trials with SL-401in blood cancers, including acute myeloid leukemia.
Synergy Pharmaceuticals Inc. (SGYP:NASDAQ); ~$700M market cap. Synergy is focused on lucrative gastroenterology sector. Its lead drug candidate, plecanatide, has completed Phase 3 trials in chronic idiopathic constipation. The firm's second pipeline candidate, dolcanatide, is in development for the treatment of opioid-induced constipation and inflammatory bowel diseases, particularly ulcerative colitis.
George Zavoico of JonesTrading selected the following companies:
Abeona Therapeutics Inc. (ABEO:NASDAQ); ~$207M market cap. Making a second appearance on the Biotech Watchlist, Abeona has three ongoing gene therapy clinical programs targeting life-threatening rare diseases with multiple orphan drug and rare pediatric disease designations: two AAV-based one-time intravenous injection therapies and one gene-corrected skin graft therapy; and a next generation proprietary AAV-based vector platform. Abeona is also recommended by Ram Selvaraju of Rodman and Renshaw.
Asterias Biotherapeutics Inc. (AST:NYSE.MKT); ~$226M market cap. Another Watchlist alumni, this stem cell company is addressing two key markets: spinal cord injury and cancer, with a dendritic cell (DC) therapy using genetically engineered autologous and allogeneic stem cells.
Catalyst Biosciences Inc. (CBIO:NASDAQ); ~$8M market cap. This company is targeting hemophilia and surgical bleeding with injectible compounds that encourage the blood to coagulate and clot.
Actinium Pharmaceuticals Inc. (ATNM:NYSE.MKT); ~$55M market cap. 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. It also has a second radioimmunotherapy, Actimab-A, in clinical study.
Threshold Pharmaceuticals Inc. (THLD:NASDAQ); ~$33M market cap. This company's lead candidate, evofosfamide, is a prodrug that is activated by the hypoxic conditions surrounding cancer tumors. The company's second compound, TH-3424, is a small molecule targeting cancers that overexpress a particular enzyme.
Edge Therapeutics Inc. (EDGE:NASDAQ); ~$366M market cap. The primary indication being targeted at Edge Therapeutics is aneurysmal subarachnoid hemorrhage; lead product candidate, EG-1962, is in a Phase 3 clinical study.
ArQule Inc. (ARQL:NASDAQ); ~$92M market cap. This company is developing targeted small molecules. Lead candidate tivantinib, targeting liver cancer, is in Phase 3.
Michael G. King Jr. is a managing director and senior biotechnology analyst at JMP Securities. King comes to JMP from Rodman & Renshaw LLC, where he was managing director and senior biotechnology analyst. He has more than 17 years of experience as a leading biotechnology equity research analyst, consistently ranking at the top of Institutional Investor magazine's annual sellside research survey, in addition to being named that publication's "Home Run Hitter" in 2000. King also served as senior vice president of corporate development and communication at ZIOPHARM Oncology (ZIOP:NASDAQ). Prior to joining ZIOPHARM, King was a managing director and senior biotechnology analyst at Wedbush Securities. He holds a bachelor's degree in finance from Baruch College.
Raghuram "Ram" Selvaraju's professional career started at the Geneva-based biotech firm Serono in 2000, where he discovered the first novel protein candidate developed entirely within the company. He subsequently became the youngest recipient of the company's Inventorship Award for Exceptional Innovation and Creativity. Selvaraju started in the securities industry with Rodman & Renshaw as a biotechnology equity research analyst. He was the top-ranked (#1) biotech analyst in The Wall Street Journal's "Best on the Street" survey (2006) and went on to become head of healthcare equity research at Hapoalim Securities, the New York-based broker/dealer subsidiary of Bank Hapoalim B. M., Israel's largest financial services group. While at Hapoalim, Selvaraju was regularly featured in The Wall Street Journal, Barron's, BioWorld Today, and Reuters/AP. He was also a regular guest on the Bloomberg TV program "Taking Stock," appeared with Bloomberg TV's on-air correspondents Betty Liu and Gigi Stone and was a guest on CNBC's "Street Signs with Herb Greenberg."
Dr. George Zavoico, senior equity analyst at JonesTrading Institutional Services LLC, has more than 10 years of experience as a life sciences equity analyst writing research on publicly traded equities. His principal focus is on biotechnology, biopharmaceutical, specialty pharmaceutical, and molecular diagnostics companies. Previously, Zavoico was a senior equity research analyst in the healthcare sector at MLV & Co., and an equity analyst at Cantor Fitzgerald and Westport Capital Markets. Prior to becoming an equity analyst, Zavoico established his own consulting company serving the biotech and pharmaceutical industries, providing competitive intelligence and marketing research, due diligence services and guidance in regulatory affairs. Zavoico began his career as a senior research scientist at Bristol-Myers Squibb Co., moving on to management positions at Alexion Pharmaceuticals Inc. and T Cell Sciences Inc. (now Celldex Therapeutics Inc.). Zavoico has a bachelor's degree in biology from St. Lawrence University and a Ph.D. in physiology from the University of Virginia. He held post-doctoral fellowships at the University of Connecticut School of Medicine and Harvard Medical School/Brigham & Women's Hospital. He has published more than 30 papers in peer-reviewed journals and has coauthored four book chapters. He received The Financial Times/Starmine Award two years in a row for being among the top-ranked earnings estimators in the biotechnology sector.
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Disclosures:
1) Tracy Salcedo compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. She owns, or members of her immediate household or family own, shares of the following companies mentioned in this article: None. She is, or members of her immediate household or family are, paid by the following companies mentioned in this article: None.
2) The following companies mentioned are sponsors of Streetwise Reports: None. Streetwise Reports does not accept stock in exchange for its services. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
3) Comments and opinions expressed are those of the specific analysts and not of Streetwise Reports or its officers.
4) George Zavoico: I, or members of my immediate household or family, own shares of the following companies mentioned in this article: None. I personally am, or members of my immediate household or family are, paid by the following companies mentioned in this article: None. My company has a financial relationship with the following companies mentioned in this article: Abeona Therapeutics Inc., ArQule Inc., Asterias Biotherapeutics Inc., Actinium Pharmaceuticals Inc., Catalyst Biosciences Inc. I determined which companies would be included in this article based on my research and understanding of the sector.
5) Raghuram Selvaraju: I, or members of my immediate household or family, own shares of the following companies mentioned in this article: None. I personally am, or members of my immediate household or family are, paid by the following companies mentioned in this article: None. My company has a financial relationship with the following companies mentioned in this article: Abeona Therapeutics Inc. Note: H.C. Wainwright & Co., LLC served as a co-manager on the follow-on equity offering Abeona completed in early November 2016. I determined which companies would be included in this article based on my research and understanding of the sector.
6) Michael King: I, or members of my immediate household or family, own shares of the following companies mentioned in this article: None. I personally am, or members of my immediate household or family are, paid by the following companies mentioned in this article: None.
JMP Securities disclosures for the following BPMC, EPZM, KPTI, LOXO, PIRS, WVE, OMED, SNDX:
JMP Securities currently makes a market in the security of Blueprint Medicines Corp.
JMP Securities was manager or co-manager of a public offering of securities for Blueprint Medicines Corp (BPMC) in the past 12 months, and received compensation for doing so.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Blueprint Medicines Corp in the next 3 months.
JMP Securities currently makes a market in the security of Epizyme, Inc.
JMP Securities was manager or co-manager of a public offering of securities for Epizyme, Inc. (EPZM) in the past 12 months, and received compensation for doing so.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Epizyme, Inc. in the next 3 months.
JMP Securities currently makes a market in the security of Karyopharm Therapeutics Inc.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Karyopharm Therapeutics Inc. in the next 3 months.
JMP Securities currently makes a market in the security of Loxo Oncology, Inc.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Loxo Oncology, Inc. in the next 3 months.
JMP Securities currently makes a market in the security of Pieris Pharmaceuticals, Inc.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Pieris Pharmaceuticals, Inc. in the next 3 months.
JMP Securities currently makes a market in the security of Wave Life Sciences Ltd.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Wave Life Sciences Ltd. in the next 3 months.
JMP Securities currently makes a market in the security of OncoMed Pharmaceuticals, Inc.
JMP Securities was manager or co-manager of a public offering of securities for OncoMed Pharmaceuticals, Inc. (OMED) in the past 12 months, and received compensation for doing so.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from OncoMed Pharmaceuticals, Inc. in the next 3 months.
JMP Securities currently makes a market in the security of Syndax Pharmaceuticals, Inc.
JMP Securities was manager or co-manager of a public offering of securities for Syndax Pharmaceuticals, Inc. (SNDX) in the past 12 months, and received compensation for doing so.
JMP Securities expects to receive OR intends to seek compensation for investment banking services from Syndax Pharmaceuticals, Inc. in the next 3 months.
I determined which companies would be included in this article based on my research and understanding of the sector.
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