The “valley of death” between drug discovery and the development and provision of therapy is a huge issue in biotech. Recent news, however, highlights valiant entrepreneurs who look at the chasm and are building bridges to cross it. One of these is Ilan Ganot whose son, Eytani, was diagnosed with Duchenne muscular dystrophy (DMD) at the age of two years old.
The ex-hedge fund banker at JPMorgan Chase & Co quit his job in London, UK to set up Solid Ventures, a biotechnology company focused on acquiring and developing disease-modifying therapies for DMD. To set up the Cambridge, Massachusetts, company, Ganot raised $17 million from various investors. The approach employed by Solid Ventures is called “condition focus”, where the target is to bring together promising projects and – by using Ganot’s banking expertise – finding the funding to accelerate the slow and risky process of developing drugs.
He told Bloomberg biotech reporter Meg Tirrell, “I am seriously focused, because I have one problem in my life. We’re going to become the center for excellence in DMD.”
DMD is a recessive X-linked form of muscular dystrophy that affects 1 in 3,600 boys (girls can carry the disease, but rarely exhibit it). The general pattern of muscular deterioration is for a child to be confined to a wheelchair by the age of 10 years old, followed by death in his early 20s.
The disease is caused by a shortage of dystrophin, which results in muscle degeneration leading to death. When Ganot’s son was diagnosed with DMD, he and his wife Annie set to researching therapies, but found nothing before reading that stem cell therapy offered some potential in treating the disease. At that point he began to look to experts in finance who could help with drug development, including co-head of healthcare investment banking at JPMorgan, Andrea Ponti, who now represents the bank on the board of Solid Ventures. Other investors involved include Andrey Zarur, a life sciences venture capitalist and Gilad Hayeem, a former CEO of a European investment fund.
DMD may not be treated with a single drug, but by looking at various projects Solid Ventures could bring together a cocktail of drugs to treat all the different symptoms: weakening of the muscles, body wasting and heart problems. This condition focus strategy is, therefore, something new in the pharma world, with Solid Ventures making it possible to accelerate the development of drugs to the point when the large pharmaceutical firms will look at them (phase 3 or phase 4) and take them through the remaining FDA approval cycle and bring them to market. This clearly reduces a lot of risk for them.
CRUSADING TO TREAT AUTOIMMUNE DISEASES
Sandy Saouaf is another inspirational biotech businessperson looking to cross the valley to find successful therapies. She set up Moorestown, New Jersey-based Atlantic Bio Sci in 2012 following the diagnosis of Type 1 diabetes in her niece at the age of eight years old. The firm is developing potential oral therapeutics to fulfil unmet medical needs in autoimmune disease. Twenty to 50 million people in the US and 8–20 percent worldwide suffer from autoimmune diseases.
Among these diseases are Type 1 diabetes (juvenile diabetes), rheumatoid arthritis in adults and juveniles, psoriasis, multiple sclerosis, systemic lupus erythematosus (known as lupus, this is a chronic disease that causes inflammation in various parts of the body), Sjögren’s syndrome (an autoimmune condition where the body’s immune system attacks glands that secrete fluid, such as the tear and saliva glands). Inflammatory bowel diseases, including Crohn’s disease and ulcerative colitis are also on Atlantic Bio Sci’s therapeutic hit list.
Saouaf is an immunologist and entrepreneur with more than 20 years of professional scientific experience in the pharmaceutical industry and academia. This background has enabled her deep understanding of drug discovery, development, and regulatory processes critical to biotechnology. Saouaf is pursuing crowdfunding for the firm’s project “Control Autoimmune Disease Now” through the Medstartr platform: http://www.medstartr.com/projects/263-help-control-autoimmune-disease-now.
In a Money Matters TV interview Saouaf explained that, “Atlantic Bio Sci has a unique and innovative approach to treating autoimmune disease. In autoimmune disease, patients’ immune cells are unbalanced and not functioning properly. The regulatory immune cells are defective and unable to control the effector immune cell response which results in tissue damage.”
“Atlantic Bio Sci’s approach to treating autoimmune disease is to restore balance to the immune system which reduces the autoimmune response resulting in decreased disease symptoms and protection of tissue from inflammation and destruction. Our lead potential drug, ABS11, targets the regulatory immune cells, increasing their function, which in turn decreases the overactivity of the effector immune cells in animal models.”