La Jol­la CEO de­camps just as biotech faces a reck­on­ing for iron over­load drug

Months af­ter tout­ing how LJPC-401 ap­pears to sig­nif­i­cant­ly low­er ex­cess iron lev­els in a small study in­volv­ing pa­tients with hered­i­tary he­mochro­mato­sis, La Jol­la Phar­ma­ceu­ti­cal is re­con­sid­er­ing its plans as the drug flopped for be­ta tha­lassemia pa­tients. And they’ll be re­view­ing their fu­ture un­der a new CEO — with a se­ri­ous­ly de­flat­ed share price.

The San Diego-based biotech is gut­ting that study, LJ401-BT01, im­me­di­ate­ly. A re­cent in­ter­im analy­sis, which in­clud­ed about half of the en­rolled pop­u­la­tion, sug­gest­ed a lack of ef­fi­ca­cy. As far as they could tell, pa­tients on the treat­ment and con­trol arms saw the same changes in car­diac iron lev­els, the pri­ma­ry end­point, and in oth­er iron, hema­tol­ogy and en­docrine mea­sure­ments.

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