The clock is ticking on the lofty goals established five years ago for the London Declaration on Neglected Tropical Diseases (NTDs), where 13 major biopharmaceutical companies banded together with government health and overseas aid ministers, and the heads of agencies including the World Health Organization (WHO) and the World Bank, all of whom committed to contain or eliminate 10 of these diseases by 2020, including Chagas disease, caused by the parasite Trypanosoma cruzi, which is a major health care problem. WHO estimates that 10 million to 13 million people are chronically infected by the disease globally. There also may be as many as 300,000 people in the U.S. that have the disease according to the CDC. Although the acute phase of Chagas disease is usually asymptomatic, and remains so for many infected individuals, up to 30 percent of those infected can develop potentially life-threatening cardiovascular, gastrointestinal and neurological complications.

Understanding mitochondrial diseases, which are estimated to affect about one in 4,000 people, remains a significant challenge because of the diversity of human disorders at every level that result when the mitochondria of the cell cannot produce the energy the body needs to support growth. In part one, a number of therapies were described that have advanced through to the clinic that target Friedrich's ataxia and other rare mitochondrial diseases. (See BioWorld Insight, Sept. 18, 2017.)

While advocacy to raise awareness about mitochondrial diseases around the world is ongoing, these efforts get a boost each year during the third week of September, which is dedicated to Mitochondrial Disease Awareness Week. Its aim is to increase the spotlight on the need for more research to uncover new therapies. 

The promise has always been there for gene therapies. Unfortunately, the field has seen a number of false starts for the expected explosion of investment and business development around treatments that correct for faulty genes. The breakthrough appeared to have been made following the European approval of Glybera (alipogene tiparvovec, Uniqure BV), for the treatment of the ultra-orphan metabolic disease lipoprotein lipase deficiency (LPL). But, while the medicine was widely heralded as the "first gene therapy" in the Western world, it failed to take off and its developer subsequently abandoned plans to have it approved in the U.S.

The biopharmaceutical sector limped through August as its equities were hit by a sharp pullback until the very last trading day of the month, with the BioWorld Biopharmaceutical index recording a 4 percent jump to turn what was heading to be a negative period into a 2.2 percent increase in valuation. Up until Thursday, despite a stream of positive industry news, investors had been on the sidelines, perhaps remaining cautious about the prevailing uncertain broader market environment, which also kept the Nasdaq and Dow treading water.

After months of speculation about Gilead Sciences Inc.'s need to accelerate its business development activities to augment its antiviral products portfolio, it finally pulled the trigger on a major acquisition last week. The proposed $11.9 billion acquisition of Kite Pharma Inc., a leader in chimeric antigen receptor (CAR) and engineered T-cell receptor development (TCR), will vault the company into the white-hot immuno-oncology (I-O) space.

When Emmaus Medical Inc.'s pharmaceutical-grade amino acid L-glutamine got the FDA green light last month it became the first new treatment for sickle cell disease (SCD) to come on the market in the U.S. since chemotherapeutic agent hydroxyurea gained approval almost a decade ago. Although hydroxyurea cuts down the frequency of crises that SCD patients suffer and reduces the need for blood transfusions some patients do not respond well to the treatment. As such, there is a significant unmet need for new SCD therapies.

While biopharma's blue chip companies continue to post strong gains, companies working on bringing new therapeutics to the marketplace have had a tougher time to garner excitement from investors with the BioWorld Drug Developers index recording a 2.1 percent fall in value in July. The downward trajectory has continued into this month and at market close on Aug. 17, the index had fallen a further 4.1 percent.

While biopharma's blue chip companies continue to post strong gains, companies working on bringing new therapeutics to the marketplace have had a tougher time to garner excitement from investors with the BioWorld Drug Developers index recording a 2.1 percent fall in value in July. The downward trajectory has continued into this month and at market close last Thursday the index had fallen a further 4.1 percent. In comparison the Nasdaq Composite index fell just 2 percent and the Dow Jones Industrial Average was down marginally at 0.6 percent. 

Serious fungal infections are responsible for an estimated 1.5 million deaths globally each year and are equally as dangerous as bacterial and viral infections but haven't received nearly as much attention. The first Fungal Disease Awareness Week, August 14-18, organized by the Centers for Disease Control and Prevention (CDC), is an initiative designed to change that and shine the spotlight on these conditions. "Think fungus" – the central theme of the campaign – is aimed at health care professionals who are being encouraged to consider fungal diseases as a possible cause when diagnosing an infection.