Dutch biotech company Pharming has reported positive clinical trial results for its lead product, recombinant human C1 inhibitor (rhC1INH) for the treatment of hereditary angioedema (HAE), at a meeting in Paris.
Pharming said that to date it has administered more than 90 infusions of rhC1INH to over 25 patients with HAE, with the results showing rapid time to relief of symptoms, starting between fifteen minutes to two hours, The time to minimal symptoms typically occurs within twelve hours of treatment, said the firm.
Hereditary angioedema is a human genetic disorder that affects roughly one in 30,000 people. Sufferers have an average of seven acute attacks per year, characterised by painful swelling of soft tissues, which can be life-threatening if the that is involved. HAE attacks that are untreated usually last up to five days.
rhC1INH is currently in Phase III testing for HAE, and Pharming has so far enrolled 150 patients in pivotal studies. With commercial production in place via an agreement with Akzo Nobel, the firm has also pressed ahead with filing for compassionate use of the drug in HAE patients.
At the meeting Pharming said it is also starting studies of rhC1INH on other inflammatory and cardiovascular indications, and it expects to provide further information on these developments in the first half of 2006.
"Pharming is in a strong position to complete licensing agreements for rhC1INH based on these positive results," said the company’s chief executive, Dr Francis Pinto. "With upfront cash payments from these agreements and sales from compassionate use of the product, Pharming aims to be profitable by the fourth quarter of 2006 or first quarter of 2007."