Take­da touts in­ter­im PhI­II da­ta of en­zyme re­place­ment ther­a­py for rare blood dis­ease

Take­da says in an in­ter­im Phase III analy­sis that its en­zyme re­place­ment ther­a­py re­duced in­stances of low blood platelet count for pa­tients with a rare blood clot dis­or­der known as con­gen­i­tal throm­bot­ic throm­bo­cy­topenic pur­pu­ra (cTTP) com­pared to stan­dard of care plas­ma-based ther­a­pies.

Dan Cur­ran

Next, it plans to file for au­tho­riza­tion for the drug lat­er this year “broad­ly — so not just in the US and EMA but al­so oth­er ju­ris­dic­tions glob­al­ly,” Take­da head of rare ge­net­ics and hema­tol­ogy Dan Cur­ran told End­points News.

Ac­cord­ing to a press re­lease Thurs­day morn­ing, Take­da’s ERT, dubbed TAK-755, re­duced low platelet count events by 60% com­pared to stan­dard of care at the time of in­ter­im analy­sis. The study in­clud­ed around 60 pa­tients, and the in­ter­im analy­sis used da­ta on ado­les­cents and adult pa­tients, but not chil­dren un­der 12. Cur­ran al­so not­ed that the ERT re­duced in­stances of oth­er symp­toms as­so­ci­at­ed with the dis­ease, which man­i­fests in a mul­ti­tude of ways, but said that da­ta would be pre­sent­ed at a fu­ture med­ical con­fer­ence.

TTP comes in two forms — con­gen­i­tal and im­mune, in which pa­tients de­vel­op an im­mune re­sponse lat­er in life that trig­gers the dis­ease. TTP pa­tients lack a pro­tein that cleaves Von Wille­brand fac­tor, the glue that sticks platelets to­geth­er to form clots and stop bleed­ing. Take­da’s ERT is a re­com­bi­nant ver­sion of that pro­tein called ADAMTS13.

cTTP is ex­ceed­ing­ly rare. Cur­ran es­ti­mat­ed that there are around 300 to 400 cas­es in the US. Pa­tients with the dis­ease ex­pe­ri­ence bleed­ing in or un­der the skin, jaun­dice, ex­treme fa­tigue, vom­it­ing, or oth­er symp­toms, and in the worst cas­es, stroke or seizures.

In the study, pa­tients who got Take­da’s ERT al­so ex­pe­ri­enced few­er side ef­fects — 8.9% had ad­verse events on Take­da’s treat­ment com­pared to 47.7% on plas­ma-based in­fu­sions. “Many pa­tients dis­con­tin­ue sup­ple­men­ta­tion with plas­ma-de­rived prod­ucts be­cause of ad­verse events,” Cur­ran not­ed, “so it’s in­cred­i­bly im­por­tant that you have a safe drug that will be used by pa­tients con­tin­u­al­ly.”

He lat­er added that, es­pe­cial­ly in the case of cTTP, in which many are di­ag­nosed as chil­dren, it was im­por­tant that pa­tients could use the drug over their en­tire life­time. Like oth­er ERTs, Take­da’s ERT is dosed once every two weeks.

Take­da ac­quired TAK-755 from its 2019 buy­out of Shire (which ac­quired the ERT from its 2016 merg­er with ​​Bax­al­ta).

Take­da is al­so test­ing the ERT in a Phase II study in iTTP and in a Phase Ib study in sick­le cell dis­ease, both of which Cur­ran said will have da­ta this year.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.

Gun­ning for 2023 ap­proval, GSK de­tails PhI­II da­ta for Jem­per­li in front­line en­dome­tri­al can­cer

GSK has a new slate of data to offer on its PD-1 inhibitor, Jemperli — data that the pharma giant hopes will cement one of the four drug approvals it’s expecting this year.

While Jemperli (dostarlimab) is already approved for a subset of patients with second-line endometrial cancer, GSK set out in the Phase III RUBY trial to test it as an earlier line of treatment while also enrolling a broader group of patients. In an interim analysis, Jemperli was shown to extend progression-free survival for both the subset and the overall trial population when added to chemotherapy.

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Doug Williams, departing Codiak BioSciences CEO

Co­di­ak files for Chap­ter 11 bank­rupt­cy as most ex­ec­u­tives head for the ex­it

Codiak BioSciences has filed for Chapter 11 bankruptcy, spelling an end to the employment of most executives, including founder Doug Williams, as the biotech says it “expects to consummate a sale.”

The eight-year journey at Codiak is nearing an end with Williams; CFO Linda Bain; medical chief David Mauro; scientific head Sriram Sathyanarayanan; legal and compliance chief Yalonda Howze; and SVP of HR Nicole Barna all packing up their bags in the first few days of April. Chief technology officer Konstantin Konstantinov will stay.

Chaim Lebovits, BrainStorm Cell Therapeutics CEO

Brain­Storm gets FDA ad­comm for ALS drug af­ter failed tri­al and RTF

BrainStorm Cell Therapeutics has had a rocky road trying to get its ALS treatment through to the FDA, including a particularly difficult setback after the FDA slapped the company with a refusal to file (RTF) letter in November after it submitted its original BLA in September last year.

But there’s a glimmer of hope for BrainStorm’s ALS treatment, NurOwn – the FDA will hold an advisory committee meeting to discuss the company’s BLA for the treatment, the company announced today.

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Sally Susman, Pfizer EVP and chief corporate affairs officer

Q&A: Pfiz­er cor­po­rate com­mu­ni­ca­tions chief Sal­ly Sus­man dis­cuss­es book craft­ed in pan­dem­ic and per­son­al lessons

From the political arena to the finance and beauty industries to pharmaceuticals, Pfizer’s Sally Susman has broken barriers, stereotypes and conventions. And now the chief communicator is “Breaking Through,” the title of her first book about effective and innovative communications launching today. The full official title is “Breaking Through: Communicating to Open Minds, Move Hearts, and Change the World.”

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Max Colao, OncoVerity CEO

Tiny mul­ti­omics biotech se­cures for­mer J&J drug, new ex­ecs and new fi­nanc­ing

A new spinout from Belgium’s argenx seeks to give new life to a candidate once in-licensed to J&J.

OncoVerity announced Monday afternoon that it had in-licensed cusatuzumab from argenx. The biotech also appointed two new C-suite executives and secured a $30 million Series A round.

CEO Max Colao joins OncoVerity after working as Aurinia’s commercial chief, and stints at Alexion and Amgen. New CMO Clay Smith has been associate chief of the University of Colorado’s hematology division.

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