With only two more trading days before the curtain drops on the year, biotech’s blue chip companies are poised to record a collective 76 percent increase in their share values. Although the group cooled off in December, with the BioWorld Blue Chip Index recording only a marginal increase of 0.4 percent, it takes nothing away from a truly remarkable 12 months for these companies and the sector itself.

For the first time in months the Blue Chip Index did not outpace the general markets with the Dow Jones Industrial average recording a monthly gain of 2.5 percent and the Nasdaq Composite index up 3.7 percent. (See BioWorld Blue Chip Index, below.)

It has been a great year for companies in the group, with several of them recording triple digit percentage gains in their share values. Leading the pack is the 292 percent gain in the share value of antisense technology developer Isis Pharmaceuticals Inc. Another top performer is Jazz Pharmaceuticals plc, which has seen its share vault by more than 136 percent. The company capped off the year with its announcement that it is acquiring Gentium SpA for about $1 billion in cash. (See BioWorld Today, Dec. 23, 2013.)

The transaction gives Jazz access to Gentium’s Defitelio (defibrotide), its lead product for the treatment of severe hepatic veno-occlusive disease (VOD) in adults and children undergoing hematopoietic stem cell transplantation. In October, the European Commission granted marketing authorization for the product.

Defitelio is a good strategic fit for Jazz as it “adds a new orphan product that has potential for short- and long-term revenue generation, high growth and expansion of our multi-national commercial platform,” said Bruce Cozadd, chairman and CEO of Jazz Pharmaceuticals, in a statement about the transaction.

The BioWorld Growth Index, which includes companies with market caps in the range of $1 billion to $3 billion and a résumé that typically includes a strong drug pipeline and partnered products in late-stage clinical trials, has performed even better than the Blue Chip Index. This group has seen tremendous investor enthusiasm all year, a factor that has propelled the index value to grow by 108 percent. (See BioWorld Growth Index, below.)

Exceptional performance

Acadia Pharmaceuticals Inc. has led the group with an exceptional 447 percent hike in its share price. Investors and analysts alike have been impressed by the pace of progress of the company’s leading products led by the oral tablet pimavanserin for Parkinson’s disease psychosis (PDP). The candidate product started the year in good shape when the FDA told Acadia it will not need another Phase III trial in order to ask for approval and moved its likely launch date, if approved, up about nine months, analysts estimated. (See BioWorld Today, April 12, 2013.)

An inverse agonist of serotonin 5-HT2A, the compound has undergone a Phase III study designated 020, and Acadia was readying a confirmatory trial, said CEO Uli Hacksell during a conference call with investors. The company is still expecting to file an NDA for pimavanserin in PDP near the end of next year.

Acadia’s other key program is in Alzheimer’s disease psychosis (ADP). In November the company initiated a Phase II feasibility trial designed to examine the efficacy and safety of pimavanserin as a treatment in this condition. The 019 study is a randomized, double-blind, placebo-controlled trial designed to examine the efficacy and safety of pimavanserin in about 200 patients with ADP. The study is being conducted through a large network of research care homes established as part of the National Institute for Health Research (NIHR) Maudsley Biomedical Research Unit. Following a screening period that includes brief psycho-social therapy, patients will be randomized on a one-to-one basis to receive either 40 mg of pimavanserin or placebo once-daily for 12 weeks. The study will assess several key efficacy endpoints to measure psychosis (hallucinations and delusions), agitation/aggression, and sleep/nighttime behavior.

On a wider scale, the BioWorld Stock Report, which tracks the stock prices of the 259 biotechnology companies developing therapeutics listed on U.S. exchanges, reinforces the incredible year for these firms. Collectively their share prices are up an average of 54 percent. Among the group of companies, 68 percent recorded positive gains in their share price.

Editor’s Note: Our full analysis on the end-of-year performance of companies, the amounts of capital raised and trends for 2014 will be published Jan. 13.