Reshma Kewalramani, Vertex CEO (Barry Chin/Boston Globe via Getty Images)
April 28, 2023 12:27 PM EDTUpdated 03:10 PM
Bioregnum

An­oth­er big Ver­tex chal­lenger suf­fers a TKO. And that will cause some prob­lems for the CF champ

You almost have to feel sorry for the R&D groups that try to challenge Vertex’s $10 billion cystic fibrosis franchise. Even if it’s a quite capable market brawler like AbbVie.

Yesterday, AbbVie R&D chief Tom Hudson shrugged off a major setback for the company, noting that its mid-stage CF triple had failed an interim review “and we are discontinuing our cystic fibrosis program.”

After an earlier failure, this one is kaput.

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May 22, 2023 06:00 AM EDT

Achiev­ing Dig­i­tal Trans­for­ma­tion: Un­lock­ing Cost Re­duc­tion, Clin­i­cal Ex­cel­lence & Pre­ci­sion Ther­a­peu­tics Man­u­fac­tur­ing

Vasu Rangadass, Ph.D.

President & CEO of L7 Informatics, Inc.

In the dynamic landscape of pharmaceutical and biotech industries, L7 Informatics is leading the charge in transforming traditional operations through digital innovation. With a firm focus on cost reduction, clinical improvements, reducing tech-transfer times, L7 Informatics is revolutionizing the way companies research, develop, and manufacture while also prioritizing the well-being of patients and providers thereby paving the way for a future where patients receive better treatments at lower costs.

May 27, 2023 06:15 AM EDT
Weekly

End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; and more

Amber Tong

Senior Editor

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.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Douglas Love, Annexon CEO
May 24, 2023 04:01 PM EDTUpdated 05:03 PM
R&D
Pharma

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Kyle LaHucik

Associate Editor

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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Rich Horgan (R) with his late brother, Terry
May 25, 2023 11:28 AM EDT
Cell/Gene Tx
In Focus

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Ryan Cross

Senior Science Correspondent

Jared Whitlock

Features Editor

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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May 25, 2023 09:05 AM EDTUpdated 02:55 PM
People

Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Andrew Dunn

Biopharma Correspondent

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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May 24, 2023 08:00 AM EDTUpdated May 25, 08:48 AM
Special

The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

ENDPOINTS

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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May 26, 2023 04:55 PM EDT
Pharma
FDA+

FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

Nicole DeFeudis

Editor

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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May 26, 2023 11:43 AM EDT
Pharma
Coronavirus

Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

Nicole DeFeudis

Editor

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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May 26, 2023 11:04 AM EDT
News Briefing

Bench­Sci rais­es $70M Se­ries D for drug dis­cov­ery soft­ware; Zen­tal­is touts PhIb ovar­i­an can­cer da­ta

Amber Tong

Senior Editor

Max Gelman

Senior Editor

Kyle LaHucik

Associate Editor

Canada’s BenchSci has raised $70 million in a Series D round designed to scale its AI drug discovery platform for scientists working in preclinical research.

The company boasts thousands of clients for its software, including 16 of the top 20 pharmaceutical companies. On the platform, researchers can get a digest of AI’s summary of medical, clinical and research data — helping them spot biological connections and choose among thousands of options with, say, a reagent or antibody.

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An­oth­er big Ver­tex chal­lenger suf­fers a TKO. And that will cause some prob­lems for the CF champ

Achiev­ing Dig­i­tal Trans­for­ma­tion: Un­lock­ing Cost Re­duc­tion, Clin­i­cal Ex­cel­lence & Pre­ci­sion Ther­a­peu­tics Man­u­fac­tur­ing

End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; and more

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

Bench­Sci rais­es $70M Se­ries D for drug dis­cov­ery soft­ware; Zen­tal­is touts PhIb ovar­i­an can­cer da­ta

A service from the Financial Times