Success is an Orphan

An old saying goes, “Success has a thousand fathers; failure is an orphan.” A Basel, Switzerland-based biotech company, MondoBiotech AG (www.mondobiotech.com) seeks to turn this cliché on its head with an ingenious approach to developing new therapies for rare diseases.

The company was founded in 2001 in Lugano, Switzerland, on the premise of combining orphan drugs with orphan diseases for the treatment of uncommon and fatal lung conditions. “Orphan” in this sense refers to drugs developed for the treatment of rare diseases, affecting fewer than 5 per 10,000 members of the population. Companies that pursue these maladies are rewarded in both the US and Europe with tax reductions and market exclusivity, according to the United States Orphan Drug Act of 1983 and Regulation EC141 of the European Parliament, published in January 2000. The legislation has proved a success by any measure, with 249 orphan drugs receiving marketing authorization for distribution in the US and Europe in the period from 1983 to 2004, during which the legislation has been in force.

But MondoBiotech has put a different spin on this drug development strategy. According to Dr. Paolo Bassanini, head of communications and marketing, the company’s business plan is to screen drugs-from virtual libraries of compounds-for molecules that block the chain of pathology in rare lung diseases. Millions of different chemical substances developed over the years lie fallow, never having qualified for a patentable application.

The company’s scientists use computerized screening systems, searching for the right fit, both from the molecular and the business standpoint. Promising candidates are combined as an orphan pair: a disease and an aspirant therapeutic, which now qualifies for patent protection. MondoBiotech then looks for a company with the resources to complete the necessary trials to establish the drug’s performance as a viable treatment modality.

The company’s most advanced product is Aviptadil, a synthetically produced human peptide (vasoactive intestinal peptide) that has received Orphan Drug Designation for pulmonary arterial hypertension (PAH). This is a life-threatening disorder that affects approximately 200,000 people worldwide. In patients with this condition, increased pressure in the pulmonary arteries leads to shortness of breath, fatigue, chest pain, and ultimately heart failure. Aviptadil is naturally produced by neuronal and endocrine cells, as well as by cells of the immune system. Its function in a variety of diseases is well understood, although it had not previously been entered into drug trials.

Aviptadil is the first drug that the US FDA and the European Medicines Agency are considering for combined, parallel scientific development for PAH. In accordance with its business plan, MondoBiotech has partnered with Biogen Idec (Cambridge, MA, www.biogenidec.com), signing an exclusive collaboration and license agreement for the latter to develop, manufacture, and commercialize Aviptadil. Biogen Idec will be responsible for global manufacturing, clinical development, regulatory approval, and commercialization.

MondoBiotech is also pursuing an orphan drug application for acute respiratory distress syndrome, the rapid onset of progressive malfunction of the lungs usually associated with the malfunction of the other organs. The condition is associated with extensive lung inflammation and accumulation of fluid in the alveoli that leads to low oxygen levels in the lungs. This is the fifth application for Aviptadil and represents a milestone for the company.

The European Orphan Medicinal Product Designation (OMPD) confers numerous benefits to the development of new products, including clinical protocol assistance and, upon marketing authorization, ensures MondoBiotech marketing exclusivity for up to 10 years in the European Union and up to seven years in the US.

It is too early to judge the overall success of MondoBiotech’s business model, but it is difficult to find fault with a company that gobbles up the crumbs from the pharmaceutical banquet table, and with only ingenuity as guidance, turns those crumbs into gourmet therapeutic feasts.