Troy Wilson, Kura Oncology CEO

FDA lifts par­tial hold on Ku­ra's Phase Ib AML pro­gram as biotech re­dou­bles mit­i­ga­tion ef­forts

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.”

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

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.

Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

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.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

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.”

Sen. Bill Cassidy (R-LA) (J. Scott Applewhite/AP Images)

Sen­ate un­veils its ver­sion of ac­cel­er­at­ed ap­proval re­forms as bi­par­ti­san duo calls on FDA and PTO to work to­geth­er

The Senate is joining its House counterparts and advancing accelerated approval pathway reforms to the FDA user fee legislation that must be signed by President Joe Biden before the end of September or else the FDA will have to start laying off its staff.

While Sen. Richard Burr (R-NC) warned yesterday that the user fee deals could be delayed by the infant formula crisis, the newly introduced bill on Friday shows how the Senate is aligning with its House counterparts on similar accelerated approval reforms.

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Co-CEOs Chintu and Chirag Patel (Amneal)

Look out, Neu­las­ta: A 5th biosim­i­lar is com­ing

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. 

Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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