Ku­ra On­col­o­gy is clear to re­sume stud­ies for its ear­ly-stage leukemia pro­gram af­ter the FDA lift­ed a clin­i­cal hold Thurs­day af­ter­noon.

Reg­u­la­tors had placed the hold on a Phase Ib study of KO-539, an ex­per­i­men­tal oral treat­ment for some ge­net­ic sub­sets of acute myeloid leukemia last No­vem­ber af­ter a pa­tient died while tak­ing the drug. Ku­ra ex­pects to be­gin en­rolling pa­tients again im­mi­nent­ly, CEO Troy Wil­son told End­points News.

“The physi­cians tell us they have pa­tients wait­ing,” Wil­son said. “And the key over the next cou­ple of weeks will be how do we get as many sites as need­ed up as fast as pos­si­ble.”

The death is thought to have re­sult­ed from dif­fer­en­ti­a­tion syn­drome, a con­di­tion that can of­ten pop up dur­ing AML treat­ments. Wil­son, at­tempt­ing to calm in­vestor nerves last No­vem­ber, said the syn­drome is a class-wide is­sue for menin in­hibitors such as KO-539 and can usu­al­ly be man­aged through cor­ti­cos­teroid treat­ments.

It was not known in No­vem­ber whether the drug was linked to the death and the pa­tient’s fam­i­ly did not con­sent to an au­top­sy, mak­ing it dif­fi­cult to de­ter­mine the cause, Wil­son said at the time. The pa­tient had ad­vanced dis­ease, hav­ing pre­vi­ous­ly failed four lines of ther­a­py, and was tak­ing the low­er dose be­ing stud­ied.

The cause of death is still un­de­ter­mined, Wil­son said Thurs­day, but he not­ed that in FDA cor­re­spon­dence, reg­u­la­tors fo­cused less on the death it­self and more on how Ku­ra planned to mit­i­gate fu­ture dif­fer­en­ti­a­tion syn­drome cas­es. Ku­ra says KO-539 is de­signed to in­duce dif­fer­en­ti­a­tion of leukemic cells, and such cas­es are ev­i­dence it’s work­ing as planned.

Now, Ku­ra is im­ple­ment­ing two sig­nif­i­cant changes to its mit­i­ga­tion strat­e­gy, Wil­son said. First, it will reg­u­lar­ly mon­i­tor pa­tient cell counts in the first few weeks of treat­ment us­ing blood sam­ples. And sec­ond, if the counts go above a cer­tain thresh­old at any time, physi­cians must stop ad­min­is­ter­ing the drug un­til symp­toms abate.

“Pre­vi­ous­ly, we had left that up to their dis­cre­tion,” Wil­son said of po­ten­tial treat­ment with­draw­al. “The pro­to­col still gives the physi­cians a tremen­dous amount of dis­cre­tion in treat­ment, but there is a point where … if the physi­cian sees this, and they’re hav­ing trou­ble con­trol­ling it, now they get to a point where they have to with­draw our drug.”

There are oth­er mi­nor changes to the study, but most large­ly deal with small lan­guage tweaks in the pro­to­cols, he added.

Such a mit­i­ga­tion strat­e­gy was one of three things asked for by the FDA, Wil­son out­lined two months ago, in ad­di­tion to re­view­ing safe­ty da­ta and reaf­firm­ing why Ku­ra se­lect­ed the dos­es be­ing stud­ied. Ku­ra’s study will be al­lowed to re­sume at the same dos­ing lev­els — 200 mg dai­ly at the low dose and 600 mg dai­ly at the high dose.

There’s no time­line yet for when the Phase Ib study will read out da­ta, nor for when Ku­ra will have se­lect­ed the ap­pro­pri­ate Phase II dose. Giv­en the ag­gres­sive na­ture of re­lapsed and re­frac­to­ry AML, Wil­son said he couldn’t rule out an­oth­er pa­tient death in the fu­ture. But as with many treat­ments in ad­vanced can­cers, Wil­son ar­gued the ben­e­fit out­weighs the risk.

“These pa­tients have weeks or pos­si­bly days to live. I don’t think we can give an iron­clad guar­an­tee that it would nev­er hap­pen again,” he said. “You do every­thing you can to try to give the pa­tient the best chance for sur­vival, and the best chance to put the dis­ease in re­mis­sion.”

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

As Neulasta sales slip, Amgen has yet another biosimilar to look out for: Amneal Pharmaceuticals and Kashiv Biosciences’ Flynetra.

Flynetra became the fifth approved biosimilar to Neulasta on Friday, snagging a win in neutropenia, a condition common among chemotherapy patients where neutrophils, a type of white blood cell that fights infection, are too low.

As of last summer, the list price of Neulasta was more than $6,400 per dose. It’s designed to be taken in a single dose per chemotherapy cycle. Amneal declined to reveal how much it intends to charge for Flynetra in an email to Endpoints News.