It is impossible to cover all the companies attending this conference, but here are notes on a few interesting drug development firms. A round-up of some tool developers and service providers will follow in a couple of days.
Amgen expects a negative impact on EPS this year, partially related to the Affordable Care Act as well as the regulatory and reimbursement landscape for erythropoietin stimulating agents. Denosumab, sold as Prolia and Xgeva, will be key to top-line growth.
Xgeva was approved in November 2010 for prevention of skeletal-related events in patients with bone metastases from solid tumors. It competes with Novartis’ Zometa, which reportedly has sales of about $1.4 billion per year. Prolia was launched in 2010 and had third quarter revenues of $10 million. It is also indicated for post-menopausal osteoporosis.
Amgen did not present financial results for 2010 or guidance for 2011 but noted that a strong balance sheet would give it flexibility to look for growth outside the company. The firm’s most recent deal was entered into on January 6 with Xencor. It focuses on the development of an Fc-engineered mAb targeting CD19 and CD32b currently in preclinical testing against autoimmune diseases.
The new CEO of Biogen Idec, George Scangos, presented a company in transformation. He reported cuts in major R&D programs for oncology and cardiology and said the company was refocusing on its MS franchise and neurogenerative diseases. The company’s workforce was reduced by 13%, which cut expenses by $300 million.
Updates have been filed with the FDA on its Tysabri label with information on the JCV antibody status, which is important to assess the risk-benefit profile related to progressive multifocal leukoencephalopathy. Phase III data on MS candidates, BG-12 and PEG-Avonex, is expected in 2011.
Biogen Idec is considering entering the biosimilar space, according to the Scangos. In addition to Scangos other new executives at the company include: Doug Williams will head up R&D and Steve Holtzman will head up corporate development.
Dendreon reported that revenues from Provenge for the fourth quarter of 2010 were $25 million with total 2010 revenues of $48 million. Pricing is a potential issue for Europe. Sales for 2011 are forecast to be $350–400 million.
The focus for 2011 is on: building manufacturing and treatment facilities in the U.S.; European approval and build-out by 2013; and development of active cellular immunotherapies against Her2/neu and CA9 targets as well as a small molecule against TRPP8.
NuPathe is a specialty pharmaceutical company focused on migraine with a unique patch delivery product called Zelrix designed to address shortcomings of current therapies. The market potential of migraine in the U.S. is reportedly about 13 million prescriptions, with 31 million migraine sufferers worldwide and a $2.5 billion global market. An NDA for Zelrix was filed in October and accepted by the FDA on January 10; the PDUFA date is expected later this year for a product launch in the first half of 2012.
The Zelrix patch is IP protected through 2023, and additional CNS disease products, such as for Parkinson and bipolar disorder, are planned. Zelrix delivers an existing drug (Triptans) but would reportedly reduce unwanted side effects related to oral delivery, such as nausea, cardiovascular events, vomiting, and gastric stasis.
The company went public in August 2010 at a price of $10 per share, raising $43 million. Safeguard Scientifics, a public holding company that provides venture funding to entrepreneurial technology companies, still holds a significant number of shares and provided $18.3 million in capital to NuPathe.
Regeneron noted that it raised $154 million in October at a price of $28 per share. It has a broad late-stage antibody pipeline that includes treatments for cancer, eye diseases, and gout. Five human antibodies in clinical development are partnered with sanofi-aventis, which will provide $350 million in resources. Their lead candidate, Aflibercept in combination with common regimens for metastatic prostate, colorectal, and non-small-cell lung cancers, is in Phase III trials.
The VEGF Trap-Eye project with Bayer Healthcare is in Phase III for wet age-related macular degeneration, a $3 billion market. The program was extended to include central retinal vein occlusion a disease that can cause retinal injury and loss of vision. Aflibercept is an anti-angiogenic agent designed to bind all forms of vascular endothelial growth factor needed for tumor growth.
Unlike many large healthcare companies expanding into new consumer, eyecare, and generic markets, Roche is staying with its core focus and what it does best—diagnostics and therapeutics. The firm remarked that patent expirations and biosimilars are of limited concern, and with the acquisition of Genentech, its therapeutic portfolio is very broad.
The $2.5 billion debt from the Genentech purchase is being paid down—33% has been retired thus far. There are 14 late-stage drugs in the pipeline, including several antibodies for autoimmune diseases and cancer as well as small molecules for CNS disorders. Furthermore, personalized medicine, in particular companion diagnostic development alongside drug development, was identified as a big part of Roche’s agenda.
Other biotechnology companies to watch in 2011 are Celgene, Gilead, and United Therapeutics.