Achillion vs. Actelion More Biotech M&A’s to Come

Last week’s announcement by Swiss drug maker, Roche Holding AG, that it was buying biotech firm InterMune, Inc. for $8.3 billlion sparked speculation by industry watchers about who would be the biotechs that might be involved in the next round of M&As. InterMune makes a drug, Esbriet (pirefnidone) that treats a lung condition, idiopathic pulmonary fibrosis. Esbriet is approved in Canada and Europe. The drug has the potential to become a blockbuster seller. InterMune’s product would join Roche’s Pulmozyme and Xolair to build up its lung drug portfolio.

A number of names popped up such as Achillion Pharamaceuticals, Actelion, Puma Biotechnology, Intercept Pharmaceuticals, and others. Maybe people heard Achillian but were attracted to similar sounding Actelion.

• Achillion Pharamaceuticals makes Phase II hepatitis C virus (:HCV) candidate, ACH-3102.
• Puma Biotechnology is developing its Phase III breast cancer candidate neratinib, PB272.
• Intercept Pharmaceuticals makes its phase II nonalcoholic steatohepatitis drug, obeticholic acid, OCA.
• Actelion Pharmaceuticals makes a cancer drug to treat a rare form of non-Hodgekin’s lymphoma.

Some in the big pharma side are still holding out hopes of buying a biotech that can make their next blockbuster. So which will it be? Achillion? Actelion? Others? To be sure, we will need wait and see.

ImmunoCellular Therapeutics Presented at Biotech Showcase 2013

SAN FRANCISCO, January 9, 2013 – Biotech Showcase 2013, at the Parc 55 Wyndham Union Square Hotel, offered many opportunities for small and medium-sized biotech public or private firms the opportunity present their information to potential investors or partners.  I watched presentations from several companies.  The following summary highlights the activities of ImmunoCellular Therapeutics.

Director of Business Development and former scientist, Peter Ho talked about his firm, ImmunoCellular Therapeutics, Ltd. (IMUC:NYSE Amex). Peter said that the company’s technology is a next generation cancer immunotherapy that combines targeting of cancer stem cells (CSCs) with strong immunotherapy to effectively eradicate cancer cells. He said that their technology overcomes the failures of previous cancer vaccines. By killing CSCs, the cancer cells will not grow back.  Ho said that T cells kill cancer cells, have high specificity, targets several antigens, offers a level of safety and can distinguish between cancer stem cells and normal stem cells.

He discussed the product pipeline and clinical development status.  Peter said that the company is creating immunotherapies that treat multiple cancer indications.  The firm has three near term immunotherapies including ICT-107 (GBM), ICT-140 (Ovarian), and ICT-121 (recurrant GBM and CD133 marker).

Peter said that ICT-107 is a dendritic cell vaccine used against glioblastoma (GBM) antigens and CSCs (brain cancer).  This product is in a Phase 2 study with results planned for late 2013.  He said that ICT-140 targets ovarian cancer antigens and CSCs.  The firm did an IND filing in Q4/2012.  Their ICT-121 is a dendritic cell vaccine initially for recurrent GBM and targets the CD133 biomarker.  He said that the CD133 biomarker is a common biomarker in several cancers including brain, NSCLC, melanoma, pancreatic and breast cancer. The IND is approved and clinical enrollment started in 4Q/2012.

Peter said that their lead product candidate, ICT-107, has a significantly lower cost of goods (COGs) advantage over Dendreon’s Provenge. ICT-107 is 5-10% of COGs vs. Provenge 70% of COGs.

He concluded his talk by saying that the company has 28 patents, has an experienced management team, and raised $20 million in October 2012.  The firm recently hired Andrew Gengos as the new CEO in December 2012 to lead their commercialization efforts. ImmunoCellular Therapeutics, Ltd. is based in Woodland Hills, California.

BIOInvestor Forum: Biotechs Fight Cancer Stem Cells

The CSC workshop was a hot topic at the BIOInvestor Forum at the Palace Hotel in San Francisco on October 9, 2012,.  At the “Cancer Stem Cell Therapy—Real or Just Hype?” workshop moderated by Nathan Sadeghi-Nejad, Contributor at Forbes & TheStreet, panelists talked about the difficulty of developing therapies for cancer stem cells (CSCs).

CSCs Can Evolve to Resist Chemo

John Lewicki, Executive Vice President and CSO, at OncoMed Pharma. Inc. said, “CSCs can resist attacks easily. They are fundamentally resistant to chemotherapy.  At OncoMed, we are trying to reduce that resistance with our antibodies by having multiple approaches.”  “Early on, we have seen data that is impressive with unique results.  We managed to control extremely aggressive ovarian cancer,” said Lewicki.

Tom Cirrito, VP of Research and Development, at Stemline Therapeutics, talked about the company’s SL-401 lead compound, for treating advanced acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Cirrito said, “We’ve treated 76 patients with a large body of preclinical evidence.”

Leslie Crews, Project Scientist, at Sanford Consortium for Regenerative Medicine/UC San Diego Cancer Center said, “We need to distinguish between tumor bulk and CSCs.  We also need a combination of strategies. These are evolving targets.  Cells evolve and evade therapies.  We should introduce CSC therapies later.”