HIV-associated lipodystrophy

Status: Confirmatory Phase 3 study ongoing

The syndrome
HIV-associated lipodystrophy is a metabolic disorder that afflicts a significant percentage of HIV patients. Caused by several factors including the antiviral regimen and the virus itself, HIV-associated lipodystrophy is characterized by changes in body composition, dyslipidemia and glucose intolerance.

The changes in body composition include: accumulation of excess visceral adipose tissue (VAT) and loss of subcutaneous fat tissue also known as SAT, generally in the limbs and facial area.

Excess VAT is believed to be a risk factor for cardiovascular disease and type 2 diabetes. In addition to the direct health risks, the resulting body abnormalities can stigmatize patients and discourage compliance with their HIV treatments.

The product

Tesamorelin (TH9507) is a stabilized analogue of the growth hormone-releasing factor (GRF) that induces growth hormone (GH) in a specific and physiological manner. To date, our studies suggest that tesamorelin has several beneficial features: it reduces VAT, without reducing SAT and without compromising glycemic control (blood glucose), it increases muscle mass and improves the lipid profile. These characteristics make it an ideal candidate for the treatment of excess VAT, an important aspect of HIV-associated lipodystrophy.

The Market
In 2008, it was estimated that among the 2 million HIV-positive patients in North America and Europe, approximately 285,000 suffer from HIV-associated lipodystrophy. Based on proprietary market research with payors, patients and physicians, Theratechnologies estimates that by 2012 approximately 380,000 patients treated with antiretrovirals will have HIV-associated lipodystrophy in North America and Europe and that the potential total market size for this condition will be between US$811 million to US $1.3 billion. There is currently no approved treatment for patients with HIV-associated lipodystrophy.

The strategy

HIV-associated lipodystrophy provides an excellent entry point for the commercialization of tesamorelin for many reasons:

• Unmet medical need (no approved product on the market)
• Clinical advantage (tesamorelin can be administered to both pre-diabetic and diabetic patients – respectively 35% and 7% of the targeted patient population)
• Manageable program (cost and size of Phase 3 studies are feasible for the Company)
• Regulatory path fairly straightforward
• Accessible market with a relatively small number of HIV specialists treat these patients