Ipsen con­tin­ues its shop­ping spree with a $1B-plus deal for Ex­i­cure's next-gen oligonu­cleotides

Ipsen has been on a deal-mak­ing spree the last few weeks, shelling out more than a bil­lion dol­lars in two sep­a­rate deals to work on a mid-stage lev­odopa-in­duced dysk­i­ne­sia (LID) can­di­date and a pre­clin­i­cal BAX in­hibitor in sev­er­al can­cers. But on Mon­day, the com­pa­ny inked its largest col­lab­o­ra­tion deal yet.

Philippe Lopes-Fer­nan­des

Ipsen is putting down $20 mil­lion up­front and up to $1 bil­lion in biobucks for ex­clu­sive op­tions to two of Ex­i­cure’s dis­cov­ery-stage spher­i­cal nu­cle­ic acid (SNA) treat­ments for Hunt­ing­ton’s dis­ease and An­gel­man syn­drome.

“Neu­ro­science is deeply root­ed with­in Ipsen as a key strate­gic dri­ver for our busi­ness,” Ipsen CBO Philippe Lopes-Fer­nan­des said in a state­ment.

Ex­i­cure is work­ing on oligonu­cleotides, or syn­thet­ic struc­tures of nu­cle­ic acids that can be used to mod­u­late gene ex­pres­sion. Sev­er­al drug­mak­ers are de­vel­op­ing oligonu­cleotides against a range of tar­gets, in­clud­ing Io­n­is, Al­ny­lam, Dicer­na and Dy­navax. Ex­i­cure, though, be­lieves its next-gen can­di­dates can reach “pre­vi­ous­ly in­ac­ces­si­ble” tar­get tis­sues, in­clud­ing deep brain re­gions.

The com­pa­ny’s SNAs be­gin with an ar­ti­fi­cial nanos­phere as a scaf­fold, as­sem­bled with sin­gle- and dou­ble-strand­ed nu­cle­ic acids on the sur­face — cre­at­ing a “third form” of nu­cle­ic acids de­signed to eas­i­ly slip in­to cells with­out trig­ger­ing an im­mune re­sponse. Its lead can­di­date, cavro­tolimod, is cur­rent­ly in Phase II for Merkel cell car­ci­no­ma and cu­ta­neous squa­mous cell car­ci­no­ma.

David Giljo­hann

“We be­lieve our plat­form tech­nol­o­gy with its deep pen­e­tra­tion and per­sis­tence of med­i­c­i­nal ef­fect will al­low Ex­i­cure and Ipsen to over­come chal­lenges from first-gen­er­a­tion oligonu­cleotides and bring new med­i­cines to pa­tients in need,” Ex­i­cure CEO David Giljo­hann said in a state­ment.

Un­der the new agree­ment, Ex­i­cure is re­spon­si­ble for dis­cov­ery and cer­tain pre­clin­i­cal ac­tiv­i­ties for the Hunt­ing­ton’s and An­gel­man pro­grams, and Ipsen re­tains ex­clu­sive op­tions to li­cense the can­di­dates. If Ipsen goes through with it, the com­pa­ny will be re­spon­si­ble for all fur­ther de­vel­op­ment and com­mer­cial­iza­tion.

Just a few weeks ago, Ipsen put down $28 mil­lion up­front and an­oth­er $335 mil­lion in biobucks for the rights to IR­LAB’s Phase IIb can­di­date for LID — a con­di­tion re­lat­ed to Parkin­son’s dis­ease that tripped up the French biotech about a decade ago. IR­LAB will be re­spon­si­ble for the on­go­ing Phase IIb tri­al, while Ipsen is on the hook for all oth­er clin­i­cal de­vel­op­ment and world­wide com­mer­cial­iza­tion, in­clud­ing Phase III prepara­to­ry work.

And just last week, Ipsen promised up to $852 mil­lion to col­lab­o­rate on BAKX Ther­a­peu­tics’ pre­clin­i­cal BAX in­hibitor in leukemia, lym­phoma and sol­id tu­mors. Just over $14 mil­lion of that is due up­front, and the part­ners will split the costs and prof­its.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; and more

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Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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Mi­rati's KRAS drug looks like the fa­vorite in colon can­cer with new da­ta, putting the pres­sure square on Am­gen

With Amgen already providing proof-of-concept for KRAS inhibitors with its sotorasib, Mirati Therapeutics is piecing together a follow-up effort in lung cancer with data it thinks are superior. But in colon cancer, where solo sotorasib has turned in a dud, Mirati may now have a strong case for superiority.

Mirati’s adagrasib, dosed solo or in combination with chemotherapy cetuximab, showed response rates grater than sotorasib solo  and as part of combination study in a similar patient population also revealed this week at #ESMO21. Mirati’s data were presented as part of a cohort update from the Phase II KRYSTAL-1 study testing adagrasib in a range of solid tumors harboring the KRAS-G12C mutation.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

The best of the rest: High­lights from the be­low-the-fold pre­sen­ta­tions at #ES­MO21

This year’s ESMO Congress has had a major focus on Big Pharma drugs — most notably candidates from Merck and AstraZeneca — but there have also been updates from smaller biotechs with data looking to challenge the big-name drugmakers.

Today, we’re highlighting some of the data releases that flew under the radar at #ESMO21 — whether from early-stage drugs looking to make a mark or older stalwarts with interesting follow-up data.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.