Three years lat­er the FDA fi­nal­ly lifts its hold on Ap­tose drug, set­ting the stage for a re­turn to PhIb

Af­ter an al­most three-year halt im­posed by a clin­i­cal hold, Ap­tose Bio­sciences can fi­nal­ly re­sume the Phase Ib tri­al for its MYC in­hibitor.

The San Diego-based biotech $AP­TO ex­plained to­day that the clin­i­cal hold on the tri­al of AP­TO-253 was a con­se­quence of an event re­lat­ed to the in­fu­sion pro­ce­dure, which — a root cause in­ves­ti­ga­tion de­ter­mined — re­sult­ed from “chem­istry and man­u­fac­tur­ing based is­sues.” All of them have now been in­cor­po­rat­ed in an amend­ment to the IND ap­pli­ca­tion.

Giv­en the long drought of good news, in­vestors ap­pear pleased with the an­nounce­ment, send­ing the share price up 18% to $4.48 in pre-mar­ket trad­ing.

William Rice

Ap­tose gave AP­TO-253 to the first pa­tient back in Jan­u­ary 2015 in a dose-es­ca­la­tion study in­volv­ing two types of blood can­cers: re­lapsed or re­frac­to­ry acute myeloid leukemia and high risk myelodys­plas­tic syn­dromes, with up to 15 pa­tients in each group. Not­ing the in­fu­sion prob­lem at a pre­lim­i­nary re­view, the com­pa­ny sus­pend­ed the tri­al in No­vem­ber be­fore the FDA came down with the hold.

“We are ea­ger to re­turn AP­TO-253 back in­to the clin­ic,” said William Rice, chair­man, pres­i­dent and CEO, in a state­ment. “Our un­der­stand­ing of this mol­e­cule has evolved dra­mat­i­cal­ly, and we are ex­cit­ed to de­liv­er a MYC gene ex­pres­sion in­hibitor to pa­tients with de­bil­i­tat­ing hema­to­log­ic ma­lig­nan­cies.”

While clear­ing things with the FDA these past few years, Rice pre­vi­ous­ly said, Ap­tose has been at work clar­i­fy­ing the mech­a­nism of the drug (still its most ad­vanced), in­clud­ing how it in­hibits the ex­pres­sion of the tu­mor growth pro­mot­ing onco­gene MYC. And in light of re­cent da­ta, they may al­so pur­sue pa­tients with B-cell ma­lig­nan­cies in fur­ther stud­ies.

Once screen­ing and dos­ing re­sume, up to 15 clin­i­cal cen­ters are ex­pect­ed to par­tic­i­pate in the tri­al.

Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Emily Leproust, Twist Bioscience CEO

Twist Bio­science’s 'fac­to­ry of the fu­ture' in Ore­gon could de­liv­er with com­pet­i­tive pric­ing, SVB Se­cu­ri­ties says

The synthetic DNA manufacturer Twist Bioscience has given a peek behind the curtain to several analysts into its “factory of the future” as well as insight into the cost structure, workflow and technology at the site.

The 110,000-square-foot manufacturing site in the city of Wilsonville, OR, just south of Portland, which was announced back in 2020, will double Twist’s production capacity and bring around 400 jobs to the area.

Lil­ly's Covid-19 mAb no longer au­tho­rized due to Omi­cron sub­vari­ants, FDA says

The FDA on Wednesday announced that Eli Lilly’s Covid-19 drug bebtelovimab is no longer authorized to treat Covid-19 because of the rising numbers of two new subvariants that the drug does not work against.

The Centers for Disease Control and Prevention last week published new estimates that the combined proportion of Covid-19 cases caused by the Omicron subvariants BQ.1 and BQ.1.1 are greater than 57% nationally, and already above 50% in all individual regions but one.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Digital render of CPI's Medicines Manufacturing Innovation Centre in Glasgow, Scotland (Image: uk-cpi.com)

CPI opens the doors to a new $100M+ man­u­fac­tur­ing fa­cil­i­ty in Scot­land

A manufacturing site that has received interest and investments from large pharma companies and the UK government is opening its doors in Scotland.

The manufacturer CPI (Centre for Process Innovation) has opened a new £88 million ($105 million) “Medicines Manufacturing Innovation Centre” in Glasgow, Scotland, to accelerate the development of manufacturing tech and solve longstanding challenges in medicine development and manufacturing.