Osteoporosis Breakthrough, Professor Gerard Karsenty

• Chapters
• 1. Major Breakthrough in $5bn Osteoporosis Market
• 2. Inhibiting Serotonin Synthesis in the Gut, Rationale for Treating Osteoporosis
• 3. How Does an Anabolic Treatment Compare to Other Approaches in Development for Osteoporosis?
• 4. Study of Rare Genetic Disease Leads to New Target for the Treatment of Most Common Bone Disease

1. Major Breakthrough in $5bn Osteoporosis Market

Everything started with this observation: patients with High Bone Mass Syndrome have an abnormally low level of serotonin in their blood, and menopausal women with this disease never develop osteoporosis. Gerard Karsenty’s team tested gut serotonin synthesis inhibitors in animal models. They discovered a way to stimulate osteoblast proliferation, which in turn leads to bone formation. This could be the first anabolic treatment of osteoporosis, and it could be administered orally. This breakthrough discovery suggests a new approach to the treatment osteoporosis, a major metabolic disease representing a $5bn market in the US. Study of Rare Genetic Disease Leads to New Target for the Treatment of Most Common Bone Disease

2. Inhibiting Serotonin Synthesis in the Gut, Rationale for Treating Osteoporosis

Gerard Karsenty studied LP533401 in animal models and found no side effects. This molecule was also tested in early clinical trials, for irritable bowel syndrome, and no side-effects were reported. The next step would be to perform additional animal testing at different doses in different treatment regiments.

3. How Does an Anabolic Treatment Compare to Other Approaches in Development for Osteoporosis?

Amgen’s Denosumab, expected to lead in the treatment of osteoporosis when approved, works by reducing bone resorption. LP533401 stimulates bone formation. This is the first report of gut derived serotonin being used as a target to treat osteporosis. Still, more work is needed to optimize the molecule. This will be the task of the Biotech and Pharma industry, but first they will have to call Columbia University’s patent office (212-854-8444 – no pressure, just there if you need it).

4. Study of Rare Genetic Disease Leads to New Target for the Treatment of Most Common Bone Disease

If the mandatory human clinical trials confirm the relevance of this new target, a inhibiting the gut serotonin synthesis may lead to a very convenient way to prevent and treat osteoporosis, and to a blockbuster drug.

Featuring:

• Gerard Karsenty, Columbia University, Professor and Chair, Department of Genetics and Development
• Joel Sendek, Lazard Capital Markets, Managing Director and Senior Biotechnology Analyst

DISCLAIMER: VIEWERS SHOULD VERIFY ALL CLAIMS AND DO THEIR OWN DUE DILIGENCE BEFORE INVESTING IN ANY SECURITIES MENTIONED. INVESTING IN SECURITIES IS SPECULATIVE AND CARRIES A HIGH DEGREE OF RISK.

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