Orphazyme’s pipeline-in-a-product whiffs in second trial, though FDA decision still awaits

Interim Orphazyme CEO Anders Vadsholt

March 29, 2021 06:54 AM EDTUpdated 07:27 AM

Orphazyme's pipeline-in-a-product whiffs in second trial, though FDA decision still awaits

Jason Mast

Editor

Orphazyme’s central molecule has whiffed in its second major test.

The Danish biotech announced Monday that arimoclomol, their “pipeline-in-a-product” molecule, failed in a Phase III trial for inclusion body myositis, a rare muscle-wasting disease. The drug couldn’t prevent disease progression in a 150-person study, missing on both primary and secondary endpoints.

Orphazyme’s stock $ORPH tumbled 31% on the news, from $12.39 to $8.50.

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IDC: Life Sciences Firms Must Embrace Digital Transformation Now

James Allgood

Sr Product Marketing Manager, Life Sciences, Egnyte

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

August 17, 2022 06:21 AM EDTUpdated 10:49 AM

R&D

Updated: Hit by another PhIII flop, Sanofi culls breast cancer drug — sounding alarm for the class

Amber Tong

Senior Editor

John Carroll

Editor & Founder

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Tom Barnes, Orna Therapeutics CEO

August 16, 2022 06:45 AM EDTUpdated 10:36 AM

Deals

UPDATED: 'We have failed to fail': Merck gambles $250M cash on a next-gen approach to mRNA — after punting its big alliance with Moderna

John Carroll

Editor & Founder

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Head of Site Finance mRNA - Cell & Gene

Lonza

Geleen, Netherland

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Kate Haviland, Blueprint Medicines CEO

August 17, 2022 11:10 AM EDT

R&D

Blueprint met all its endpoints in bid for expanded Ayvakit label — but stock trends lower anyway

Lei Lei Wu

News Reporter

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

August 17, 2022 02:17 PM EDT

Pharma

Marketing

Bayer aims to simplify the complexities of CKD with an ABC-themed ad campaign

Beth Snyder Bulik

Senior Editor

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

August 17, 2022 02:15 PM EDTUpdated 02:59 PM

People

Pharma

Moderna taps new CFO from PerkinElmer after former one-day CFO ousted

Paul Schloesser

Associate Editor

When Moderna hired a new CFO last year, it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

August 17, 2022 01:47 PM EDT

Deals

GSK lands first-ever UNICEF contract for malaria vaccine worth $170M

Zachary Brennan

Senior Editor

GSK has landed a new first from UNICEF — the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

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Head of Quality Systems

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Lexington, MA, USA

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

August 17, 2022 01:14 PM EDTUpdated 02:43 PM

Pharma

Solo Jonas brother carries Merz's new tune in Botox rival campaign

Nicole DeFeudis

Editor

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

August 17, 2022 11:20 AM EDTUpdated 05:34 PM

Pharma

Marketing

Genmab launches corporate campaign amplifying its ‘knock your socks off’ antibodies

Beth Snyder Bulik

Senior Editor

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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