April 30, 2020 11:05 AM EDTUpdated May 1, 06:52 AM
News Briefing

Ver­tex lav­ish­es $80M cash to ex­pand gene ther­a­py tool kit; Su­per­nus bags a $300M CNS port­fo­lio

ENDPOINTS

→ Ver­tex’s bull­ish out­look wasn’t just man­i­fest­ed in its up­beat re­vi­sion of the full-year guid­ance. Ear­li­er this week, the Boston biotech hand­ed over $80 mil­lion in cash to gene ther­a­py up­start Affinia to en­gi­neer new AAV cap­sids to de­liv­er a string of po­ten­tial new treat­ments for Duchenne mus­cu­lar dy­s­tro­phy, my­oton­ic dy­s­tro­phy type 1 and cys­tic fi­bro­sis. Affinia, which de­buted to the biotech world just a month ago with AveX­is vet­er­an Rick Mo­di at the helm, is el­i­gi­ble for $1.6 bil­lion in mile­stones.

Hav­ing con­quered the ma­jor­i­ty of the CF mar­ket, Ver­tex has sig­naled a clear in­ter­est in mak­ing the next big move in cell and gene ther­a­py, marked by a bil­lion-dol­lar buy­out of Ex­on­ics and an­oth­er bil­lion-dol­lar part­ner­ship with CRISPR Ther­a­peu­tics. It’s since al­so ac­quired Sem­ma, whose for­mer CEO Bas­tiano San­na is now the chief of the group at Ver­tex.

→ Rockville, MD-based Su­per­nus Phar­ma­ceu­ti­cals has agreed to hand over $300 mil­lion in cash to grab the CNS port­fo­lio of US WorldMeds. The com­pa­ny is pick­ing up three ap­proved drugs — Apokyn, My­obloc and Xada­go as well as the apo­mor­phine in­fu­sion pump now in de­vel­op­ment for Parkin­son’s. Su­per­nus added $230 mil­lion for mile­stones.

→ Bel­gian biotech Hy­loris Phar­ma­ceu­ti­cals has raised €15 mil­lion to back its mis­sion de­vel­op­ing new for­mu­la­tions of ex­ist­ing drugs. “Right from the start, Hy­loris’ aim has been to bring re­for­mu­lat­ed drugs with high added val­ue to the mar­ket as quick­ly as pos­si­ble,” not­ed chair­man Ste­fan Yee. New and ex­ist­ing in­vestors in­clude Scor­pi­aux, Noshaq, Saf­fel­berg In­vest­ments and No­main­vest.

→ It’s of­fi­cial — US in­tel­li­gence has con­curred that the virus be­hind Covid-19 was not con­coct­ed in a lab.

https://twit­ter.com/ODNIgov/sta­tus/1255868108356681728/pho­to/1

AUTHOR

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editor@endpointsnews.com
Image courtesy of The Janssen Pharmaceutical Companies of Johnson & Johnson.
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May 29, 2023 06:00 AM EDT

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

The Janssen Pharmaceutical Companies of Johnson & Johnson

We are living in a new era of healthcare that is rapidly advancing progress impacting patient outcomes and experiences. We’ve seen a remarkable pace of transformational innovation, applied research, and advanced clinical development over the last decade.

Despite this tremendous progress, there is much more work to be done, and patients are counting on us – now more than ever – to continue that momentum. At the heart of our industry is a focus on developing and delivering medicines for some of the world’s most challenging diseases, including those that have few or no effective treatments today.

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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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 05:00 PM EDTUpdated 05:44 PM
R&D
Pharma

Mi­rati’s drug sitra­va­tinib flops PhI­II in com­bo with Op­di­vo for cer­tain lung can­cer

Kyle LaHucik

Associate Editor

Mirati Therapeutics’ path to a second drug approval will likely have to wait. The San Diego biotech company said Wednesday that its investigational lung cancer drug failed a Phase III trial testing it in combination with Bristol Myers Squibb’s Opdivo.

The drug, sitravatinib, and Opdivo weren’t better than the chemo drug docetaxel at keeping patients alive, Mirati said in a press release. The spectrum-selective kinase inhibitor missed the primary goal of overall survival in patients with second- or third-line advanced non-squamous, non-small cell lung cancer.

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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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Ver­tex lav­ish­es $80M cash to ex­pand gene ther­a­py tool kit; Su­per­nus bags a $300M CNS port­fo­lio

Pro­tect­ing the glob­al phar­ma­ceu­ti­cal in­no­va­tion ecosys­tem – what’s at stake?

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

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

Mi­rati’s drug sitra­va­tinib flops PhI­II in com­bo with Op­di­vo for cer­tain lung can­cer

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

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