“2003 was a big year,” says Biotechnology Industry Organization President Carl Feldbaum. The industry earned 37 U.S. Food and Drug Adminstration approvals, raised $16.4 billion in new financing, and won significant battles on Capitol Hill.
“Dot-coms and telecoms have not recovered, but biotech has because it’s performance-driven,” says Feldbaum, noting that the Nasdaq Biotechnology Index rose 46 percent, thanks to higher earnings. Clinically, a record 370 products reached phase 2 and 3 trials, while the FDA approved several first-in-class therapies for genetic disorders, HIV, Alzheimer’s, psoriasis, asthma, and cancer.
Of the 37 FDA approvals, 25 were for new products (up from 20 in 2002); the other 12 were new indications for existing products. Feldbaum credits the “strong-willed, politically active, technically competent” leadership of FDA Commissioner Mark McClellan, MD, PhD, for “reopening the pipeline.” McClellan, who recently was tapped to head the Centers for Medicare and Medicaid Services, “brought a deep commitment to innovation and sensible risk-benefit analysis to the agency,” says Feldbaum.
In Congress, BIO got its pro-innovation principles embedded in the Medicare Prescription Drug, Improvement, and Modernization Act of 2003, and reversed cuts in Medicare reimbursement for biotech products. Feldbaum adds that the appearance by President Bush at BIO’s annual meeting “put us in a whole new orbit politically.”
Biotech companies raised $2.4 billion in capital in December, the highest monthly total in 2003, making the 2004 outlook positive.
The record pace of approvals in 2003 is unlikely to be matched this year, as it included a backlog that accumulated prior to McClellan’s arrival. In addition, analysts point out that biotech is a highly volatile sector, thanks in part to the unpredictable nature of experimental medication development. The biotech industry will have to live up to high expectations to continue to attract investors and keep the venture capital flowing.
But pending FDA applications include several high-profile products: synthetic exendin-4 (Exenatide) for type 2 diabetes, natali-zumab (Antegren) for Crohn’s disease, and indiplon for insomnia.
Biotechs filling pipeline
About 24 percent of the 5,000 pharmaceutical products in the pipeline are biotech products. Of the $211 billion U.S. pharmaceutical market, about $22 billion comprises biotech.
SOURCE: IMS HEALTH 2003
New Approvals/NDAs
The FDA has approved Eli Lilly’s olanzapine (Zyprexa) for treatment of bipolar disorder. The drug is approved as a maintenance treatment to help patients avoid a relapse into mania or depression. Olanzapine was originally approved to treat schizophrenia and accounts for $4.3 billion in annual sales — one third of Lilly’s sales.
OSI Pharmaceuticals filed a new drug application with the FDA to use erlotinib HCl (Tarceva) in patients with incurable nonsmall-cell lung cancer. The company expects to submit data from phase 3 trials in the second quarter of this year.
U.S. Clinical Trials
Human Genome Sciences is abandoning a once-promising drug for wound healing. Keratinocyte growth factor-2 (Repifermin), a gene-based product, did not heal mucositis, a chemotherapy-induced condition that destroys cells in the mouth, throat, and digestive tract, in a large-scale trial of 92 patients. The drug had been one of HGS’s most advanced treatments and best hopes for commercialization.
A phase 2 trial of InterMune’s interferon gamma-1b failed to meet a primary endpoint as a treatment for hepatitis C-caused liver fibrosis in patients who fail standard antiviral therapy. The drug was generally well tolerated but produced no reversal of liver fibrosis. InterMune is conducting phase 3 studies of interferon gamma-1b in patients with idiopathic pulmonary fibrosis and those with ovarian cancer.
Some parents say the digestive hormone secretin has helped their autistic children, but the drug failed to alleviate autism symptoms in a trial of 132 children at numerous medical centers around the country. The findings of the study, funded by the National Institute of Child Health and Human Development, may end efforts to develop secretin for autism.
Chiron will begin human trials of an HIV vaccine in a two-year, 168-person study of uninfected patients. The trial will be coordinated with the HIV Vaccine Trials Network, a research unit backed by the National Institutes of Health.
Around the World
GW Pharmaceuticals and Bayer AG are preparing to market and produce Sativex, a cannabis-based product, in anticipation of approval by the United Kingdom’s Medicines and Healthcare Regulatory Agency. Clinical trials have demonstrated that multiple sclerosis patients using Sativex suffer less nerve-damaging pain and sleep interruption. Bayer has exclusive rights to market Sativex in the UK and in Canada.
The Committee for Proprietary Medicinal Products, based in London, approved Millennium’s bortezomib (Velcade) for patients with multiple myeloma. The European Commission usually follows the committee’s lead. A protea-some inhibitor, bortezomib is approved in the United States.
Ivax’s injectable paclitaxel received a positive evaluation from the European Medicines Evaluation Agency to include the treatment of metastatic breast cancer and metastatic ovarian cancer.
Schering and Organon, the Dutch pharmaceutical unit of Akzo Nobel, have launched a phase 2 study in Europe of a male contraceptive. The so-called male pill, to be tested in six strengths, combines progestogen etonogestrel with testosterone undecanoate, and will be administered to 350 men. Development of the pill is based on previous research involving Organon’s progestogen etonogestrel and Schering’s testosterone undecanoate. In those studies, investigators implanted progestogen in patients to reduce sperm concentrations while injecting the testosterone to maintain patients’ normal testosterone levels.
Also of Interest...
Victoria Hale, PhD, a former FDA staffer and biotech company scientist, tells the Wall Street Journal that OneWorld Health, the first not-for-profit drug developer, will launch its first drug in 2005 to treat kalaazar, a virus transmitted by the sand fly and that kills hundreds of thousands of people annually. The Bill and Melinda Gates Foundation funds OneWorld.
More universities and hospitals are engaging in early stage drug development, then passing compounds to companies that are better equipped to see them through later-stage development, FDA approval, and commercialization. According to the Wall Street Journal, these institutions are “de-risking” development because many scientists believe that the number of biomedical discoveries translating into clinical practice is declining.
According to Business Week, biotech startups are picking up “low-margin” vaccine business abandoned by big pharmaceutical companies. The magazine quotes one analyst that “revenues from a vaccine division are like a rounding error to a Merck, but ... very profitable for a small company.”
Even at reduced prices, the Flu-Mist nasal-spray vaccine has not been selling as expected, so Med-Immune and Wyeth Pharmaceuticals are giving away 250,000 doses to public health agencies. Med-Immune is evaluating whether to continue to back the product.