June 17, 2020 10:30 AM EDT
R&D
Cell/Gene Tx

Bio­Marin spells out a trou­bling slide in Fac­tor VI­II lev­els for its big block­buster con­tender val­rox

John Carroll

Editor & Founder

We already knew from BioMarin’s statement a few days ago that their gene therapy for hemophilia A continued to protect patients against the bleeding episodes that can threaten their lives. Now, 4 years in, we’re getting the hard data on durability for valrox — and here the biotech faces considerable potential trouble.

As Stifel’s Paul Matteis noted recently, at the end of the first, second and third years, post-infusion, mean Factor VIII activity level of the high dose 6e13 vg/kg cohort was 64.3, 36.4 and 32.7 IU/dL respectively as measured by the CS assay.

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March 27, 2023 06:00 AM EDT

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

Victoria Maharaj

M.S.,Technical Sales & Scientific Advisory, Forge Biologics

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.

March 27, 2023 10:20 AM EDTUpdated 10:38 AM
R&D
Special

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

John Carroll

Editor & Founder

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
March 28, 2023 07:30 AM EDT
Financing
Bioregnum

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
March 28, 2023 04:11 PM EDT
R&D

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

Zachary Brennan

Senior Editor

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.

March 28, 2023 07:30 AM EDTUpdated 10:17 AM
R&D
Pharma

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

Amber Tong

Senior Editor

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
March 27, 2023 08:49 AM EDT
People
R&D

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

Kyle LaHucik

Associate Editor

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
March 27, 2023 06:41 AM EDTUpdated 12:44 PM
R&D
FDA+

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

Katherine Lewin

News Reporter

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
March 28, 2023 12:30 PM EDT
Pharma
Marketing

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

Beth Snyder Bulik

Senior Editor

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
March 28, 2023 11:18 AM EDT
Financing
R&D

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

Paul Schloesser

Associate Editor

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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Bio­Marin spells out a trou­bling slide in Fac­tor VI­II lev­els for its big block­buster con­tender val­rox

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

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

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

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

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

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

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

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

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