West Coast Editor
Halfway through the first Phase III trial of its heart-failure inotropic drug, enoximone, Myogen Inc. began a second, pivotal study with 1,400 patients.
The new Phase III trial called ESSENTIAL, which stands for “The Studies of Oral Enoximone Therapy in Advanced Heart Failure” will evaluate the drug, characterized by Myogen as a “small organic molecule” that increases the force of the heart’s contractions, in patients with Class III and IV chronic heart failure, as determined under New York Heart Association guidelines. Participants will be given oral, low-dose enoximone or placebo for at least 52 weeks.
When the trials are expected to finish has not been disclosed.
“The vagaries of predicting patient accrual are such that, although we have internal forecasts, we haven’t been giving them out,” said Joseph Turner, chief financial officer at Myogen.
Privately held Myogen, of Denver, designed the trial to assess the safety and efficacy of oral enoximone through the endpoints of reducing the incidence of death or cardiovascular hospitalizations, improving patient well-being, and increasing exercise capacity.
The first trial is a randomized, placebo-controlled study of 200 patients with “ultra-advanced” heart failure, the company said, and is intended to determine safety and efficacy of oral enoximone in reducing dependency on intravenous inotropes, as well as the incidence of death or cardiovascular hospitalizations.
Enoximone is the company’s lead product, to which Myogen acquired rights from Hoechst Marion Roussel AG, now Aventis SA, in 1998. Aventis developed an intravenous formulation, called Perfan IV (approved in seven European countries), and an oral formulation. Myogen has European rights for the intravenous formulation and has estimated the oral formulation could earn between $400 million and $500 million per year worldwide.
Myogen also has ambrisentan, an endothelin receptor antagonist, about to begin Phase II trials in heart failure, chronic renal failure and pulmonary arterial hypertension. The drug, selective for the Type A endothelin receptor, works by preventing the constriction of blood vessels and increasing blood flow to vital organs.
The company gained exclusive worldwide rights to ambrisentan from Abbott Laboratories, of Abbott Park, Ill., last year, agreeing to pay Abbott undisclosed up-front money and milestone payments, as well as royalties. (See BioWorld Today, Nov. 30, 2001.)
“We just in-licensed it in September 2001, and haven’t yet progressed in the clinic,” Turner said, adding that the first step of the “two-step licensing” came when Myogen entered a co-development and license agreement for ambrisentan with Knoll AG, of Ludwigshafen, Germany (now Abbott), in November 2000.
“That’s the extent of our clinical pipeline,” Turner said.
Last month, Myogen completed a $66.4 million private placement of Series D preferred stock, a financing that began during the summer, “leaving us with sufficient cash to last about two years,” Turner said. (See BioWorld Today, Jan. 3, 2002, and Aug. 28, 2001.)