Trendy Mod­er­na's sin­gle-as­set CF pact with Ver­tex marks a new phase of deal­mak­ing

Just a few days af­ter Mod­er­na wrapped up a $200 mil­lion-up­front re­search deal with Mer­ck, the Boston biotech is back with an­oth­er pact – this time ty­ing up with crosstown biotech Ver­tex $VRTX on a cys­tic fi­bro­sis de­vel­op­ment agree­ment.

Ver­tex is start­ing out with what amounts to a rel­a­tive­ly small up­front for Mod­er­na, which has struck the moth­er lode of deals with its promise – as yet un­proven in any hu­man tri­als – that your cells can be made in­to drug pro­duc­tion units. Mod­er­na gets $40 mil­lion up­front with half of that com­ing as a con­vert­ible note in­vest­ment. And there’s an­oth­er $275 mil­lion up for grabs in the form of mile­stone pay­ments.

Stéphane Ban­cel, Mod­er­na CEO

The deal marks a dis­tinct shift in Mod­er­na’s deal-mak­ing strat­e­gy. This is a one-as­set play, Mod­er­na CEO Stéphane Ban­cel tells me via a di­rect mes­sage, not like the big port­fo­lio deals the com­pa­ny has done with the likes of As­traZeneca ($240 mil­lion up­front for 40 prod­ucts), Alex­ion ($125 mil­lion for 10) and Mer­ck. And he says that Mod­er­na is in the process of strik­ing more of these sin­gle-as­set plays, where these num­bers work fine.

In the Ver­tex deal, Mod­er­na will use its mR­NA tech over the next three years to see if it can get lung cells to pro­duce func­tion­al copies of the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR) pro­tein, which is de­fec­tive among CF pa­tients. The part­ners will al­so ex­plore pul­monary mR­NA de­liv­ery.

Mod­er­na is in charge of dis­cov­ery, with Ver­tex han­dling every­thing pre­clin­i­cal and on. Ver­tex will al­so cov­er the ex­pens­es re­lat­ed to the work.

For Ver­tex, it’s a chance to go deep­er in­to cys­tic fi­bro­sis af­ter win­ning a land­mark ap­proval for Orkam­bi, a $259,000 drug. For Mod­er­na, it’s the lat­est in a long se­ries of col­lab­o­ra­tions that has put the com­pa­ny in the lab with a who’s who in drug de­vel­op­ment.

This isn’t the on­ly ef­fort in this field, though. Two years ago Shire aligned with the Cys­tic Fi­bro­sis Foun­da­tion to de­vel­op a new CFTR treat­ment for cys­tic fi­bro­sis.

“This col­lab­o­ra­tion is il­lus­tra­tive of our con­sis­tent strat­e­gy to part­ner with world class ther­a­peu­tic ex­perts in or­der to max­i­mize the po­ten­tial of lever­ag­ing mR­NA as a nov­el ther­a­peu­tic ap­proach across a spec­trum of se­ri­ous dis­eases,” said Ban­cel in a state­ment. “We’re par­tic­u­lar­ly ex­cit­ed to ex­plore the po­ten­tial of pul­monary de­liv­ery as a new modal­i­ty to bring trans­for­ma­tive mR­NA med­i­cines to pa­tients and ad­dress un­met needs.”

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

An­gion's or­gan dam­age drug strikes out again, this time in high-risk kid­ney trans­plant pa­tients

After flopping a test in Covid-19 earlier this year, Angion’s lead organ damage drug has now hit the skids again in kidney transplant patients.

Angion and partner Vifor Pharma’s ANG-3777 failed to beat out placebo in terms of improving eGFR, a measure of kidney function, in patients who had received a deceased donor kidney transplant and were at high risk of developing what is known as delayed graft function, according to Phase III results released Tuesday.

An image of Alzheimer's brain tissue. The red show gingipains, a protein from P. gingivalis, intermixing with neurons (yellow) and glial cells (green)

An Alzheimer's dark­horse fails its first big tri­al, but of­fers hope for a long-over­looked hy­poth­e­sis

Three years ago, Cortexyme emerged out of obscurity with some big-name backers and an unorthodox approach to treating Alzheimer’s.

They moved their drug into a pivotal study the next year, offering one of the first major tests for a hypothesis that has fluttered on the outskirts of Alzheimer’s research for decades: that, in many cases, the disease is driven by infectious agents — the havoc they wreak in the brain and the inflammation the body uses to try to fend them off. And that quashing the infection could slow patients’ cognitive decline.

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No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Peter Nell, Mammoth Biosciences CBO

UP­DAT­ED: Jen­nifer Doud­na spin­out inks a Mam­moth CRISPR deal with Ver­tex worth near­ly $700M

When a company gets its start in gene editing pioneer Jennifer Doudna’s lab, it’s bound to make headlines. But three years in, the fanfare still hasn’t died down for Mammoth Biosciences. Now, the Brisbane, CA-based company is cheering on its first major R&D pact.

Mammoth unveiled a nearly $700 million deal with Vertex on Tuesday morning, good for the development of in vivo gene therapies for two mystery diseases. The stars of the show are Mammoth’s ultra-small CRISPR systems, including two Cas enzymes licensed from Doudna’s lab over the past couple years, Cas14 and Casɸ.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.