Left to right: Teaghan Duff, Talia Duff, Jocelyn Duff and John Duff. (Courtesy of Duff family)
May 16, 2023 08:00 AM EDTUpdated 08:51 AM
R&D
FDA+

$97M gene ther­a­py con­sor­tium aims to give com­pa­nies and rare dis­ease fam­i­lies a ‘north star’

Jared Whitlock

Features Editor

There was a time when Jocelyn Duff didn’t know how to answer her neighbors’ oft-asked question: “How is your daughter, Talia?”

Their small seaside community of Ipswich, MA, was pivotal in raising $1 million for an experimental gene therapy targeting Talia’s ultra-rare disease. Then came manufacturing setbacks and Covid-19 closed labs. She said a pharmaceutical company — for which Duff declined to name — deprioritized the therapy.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

SPONSORED
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!

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

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.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Teresa Bitetti, Takeda's president of the global oncology business unit
May 26, 2023 10:57 AM EDT
R&D
FDA+

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Max Gelman

Senior Editor

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

Fred Aslan, Artiva Biotherapeutics CEO
May 26, 2023 10:50 AM EDT
R&D

Arti­va boasts ear­ly NK cell ther­a­py re­sults, giv­ing life­line to part­ner Af­fimed

Lei Lei Wu

News Reporter

Artiva Biotherapeutics reported early data on its off-the-shelf NK cell therapy in combination with rituximab, suggesting the combo could shrink tumors in non-Hodgkin lymphoma patients who have already tried many other cancer therapies.

Four of six patients who received the combo therapy of AB-101 and rituximab saw their tumors shrink, and three of those six saw signs of cancer go away completely, according to an ASCO abstract released Thursday evening. Three of the patients who responded to treatment have had ongoing responses of five, seven and nine months.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

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.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

$97M gene ther­a­py con­sor­tium aims to give com­pa­nies and rare dis­ease fam­i­lies a ‘north star’

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

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

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

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

Arti­va boasts ear­ly NK cell ther­a­py re­sults, giv­ing life­line to part­ner Af­fimed

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

A service from the Financial Times