With NIH support, Eli Lilly follows NPS Pharma into hunt for rare-disease therapy

Eli Lilly ($LLY) has found a new path into the hot rare-disease field. An NIH program has backed the Indianapolis-based drug giant's preclinical-stage research of a potential treatment for a hormone deficiency often caused by a genetic defect. 

Uncle Sam is teaming up with Lilly on development of a long-acting hormone replacement for the rare disease, known as hypoparathyroidism, which causes a lack of parathyroid hormone. The hormone deficiency triggers a drop in calcium levels and a spike in phosphate in the blood, leading to a litany of nasty symptoms such as depression and anxiety, cataracts, muscle cramping, convulsions and irregular heart rhythm.

With help from the NIH's Therapeutics for Rare and Neglected Diseases (TRND) program, Lilly plans to advance research of its parathyroid hormone replacement therapy to a Phase II proof-of-concept study. The agency points out that Lilly is the first major drugmaker to gain support from the TRND program, which seeks to de-risk development of rare disease treatments that industry players might otherwise ignore because of their small markets. Still, some companies such as Alexion ($ALXN) and Genzyme have made billions of dollars selling rare-disease drugs.

The NIH points out that there are no approved hormone-replacement therapies for hypoparathyroidism. But NPS Pharmaceuticals ($NPSP), a rare-disease specialist, aims to change this in the near future. The Bedminster, NJ-based company plans to file for FDA approval of a parathyroid hormone replacement, called Natpara, for adult cases of the rare disease in the second half of this year.

Patients with the disease have to take large amounts of calcium supplements, which can lead to kidney damage. NPS has shown that patients on Natpara have been able to significantly reduce the amount of calcium supplements they take. While it's early days for Lilly's program, the company could improve treatment with a better hormone replacement that brings down supplement requirements further.

Lilly had mixed results with a previous foray into the rare diseases field. In 2011 FDA shot down the company's liprotamase, a pancreatic enzyme replacement, for patients with the orphan ailment cystic fibrosis and other diseases. Its experimental product was less effective on at least one score than existing products such as Creon in a clinical trial.

- here's the NIH release
- check out FierceBiotech Research's take

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