Take­da takes a swing at RNA small mol­e­cules with dis­cov­ery pact for mul­ti­ple po­ten­tial Evotec pro­grams

Heart­ened by an FDA ap­proval for Roche’s Evrys­di last year as the first small mol­e­cule mod­u­la­tor for RNA, the rest of bio­phar­ma is look­ing to place their bets in the promis­ing field. No stranger to ear­ly-stage dis­cov­ery deals, Take­da has now iced a pact with a Ger­man play­er to chase the first small mol­e­cule lig­and for RNA.

Ger­many-based Evotec will team up with Take­da to dis­cov­er and de­vel­op RNA tar­gets ripe for small mol­e­cule ther­a­peu­tics, the com­pa­ny an­nounced Mon­day.

In ad­di­tion to re­ceiv­ing re­search fund­ing, Evotec will be el­i­gi­ble to re­ceive dis­cov­ery, pre-clin­i­cal, clin­i­cal, com­mer­cial and sales mile­stone pay­ments of up to $160 mil­lion per pro­gram, as well as tiered roy­al­ties on net sales of any prod­ucts that come from the col­lab­o­ra­tion.

Cord Dohrmann

Tar­get in­di­ca­tions weren’t dis­closed as part of the an­nounce­ment. An Evotec spokesper­son couldn’t be reached for com­ment by press time.

The col­lab­o­ra­tion will use Evotec’s RNA tar­get­ing plat­form to iden­ti­fy promis­ing RNA se­quences to tar­get small-mol­e­cule lig­ands that can be de­vel­oped in­to ther­a­peu­tics, a state­ment said. It’s an­oth­er shot on goal at RNA tar­gets, which al­low drug de­vel­op­ers to by­pass hard-to-hit pro­teins, ac­cord­ing to Cord Dohrmann, CSO of Evotec.

“Many high­ly val­i­dat­ed tar­gets have proven to be in­tractable via con­ven­tion­al pro­tein tar­get­ing ap­proach­es,” Dohrmann said in a state­ment. “For this rea­son, Evotec has been pi­o­neer­ing RNA tar­get­ing strate­gies and ap­proach­es for quite some time.”

This type of part­ner­ship is noth­ing new for Evotec and marks the sec­ond time in the past year that it’s fos­tered an al­liance with Take­da.

In April 2020, Take­da tapped Evotec to de­vel­op gene ther­a­py tar­gets across four broad ther­a­peu­tic ar­eas — on­col­o­gy, rare dis­eases, neu­ro­science and gas­troen­terol­o­gy. In Oc­to­ber, the com­pa­ny an­nounced plans to in­vest more in cell and gene ther­a­py re­search af­ter re­ceiv­ing a $236 mil­lion in­vest­ment from Mubadala In­vest­ment Com­pa­ny, the UAE’s sov­er­eign wealth fund.

Two months ear­li­er, in Au­gust 2020, the drug dis­cov­ery com­pa­ny was en­list­ed by Dan­ish gi­ant No­vo Nordisk to tar­get chron­ic kid­ney dis­ease. The com­pa­ny al­so has long-term dis­cov­ery al­liances with com­pa­nies like Bay­er, Boehringer In­gel­heim, No­var­tis, Pfiz­er and Sanofi.

Lar­ry Hamann

Evotec’s plat­form is de­signed to iden­ti­fy RNA ter­tiary struc­tur­al el­e­ments where rSM are able to bind with “suf­fi­cient se­lec­tiv­i­ty and affin­i­ty,” dis­cov­er and de­vel­op rSM binders that could po­ten­tial­ly de­liv­er oral­ly avail­able drugs and iden­ti­fy and de­liv­er proof-of-tar­get en­gage­ment for dis­ease-rel­e­vant RNA struc­tures, al­low­ing bi­o­log­i­cal­ly ac­tive rSM binders.

“Take­da rec­og­nizes tar­get­ing RNA with small mol­e­cules as a promis­ing new modal­i­ty that has tremen­dous po­ten­tial for much need­ed med­i­cines for pa­tients through mod­u­lat­ing his­tor­i­cal­ly un­drug­gable tar­gets,” said Lar­ry Hamann, Head, Drug Dis­cov­ery Sci­ences at Take­da. “We are ex­cit­ed to be work­ing with Evotec and their im­pres­sive ca­pa­bil­i­ties.”

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, and more

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.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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As­traZeneca-Alex­ion merg­er slides through FTC re­view af­ter sup­posed M&A crack­down pos­es no bar­ri­ers

The AstraZeneca-Alexion megamerger received a good sign Friday, despite warning signs of the tides turning against large M&A pharma deals.

US regulators at the FTC have cleared the acquisition for approval, AstraZeneca announced, all but signing off on the deal to go through once it officially closes in the third quarter. AstraZeneca originally said it was planning to buy out Alexion back in December for $39 billion.

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Severin Schwan, Roche CEO (Georgios Kefalas/Keystone via AP Images)

Look­ing to ce­ment its lead in packed MS mar­ket, Roche's Ocre­vus un­corks new da­ta in ear­ly-stage pa­tients

Among a positively jam-packed multiple sclerosis market, Roche’s Ocrevus has managed to stand out for what the Swiss drugmaker is calling the most successful launch in its long history. But in order to press its advantage, Ocrevus is looking to earlier-stage patients, and new interim data should help build its case there.

After 48 weeks on Roche’s Ocrevus, 85% of newly diagnosed primary progressing or relapsing MS patients without a history of disease modifying therapy posted no disease activity, including disease progression or relapse, according to interim data set to be presented this weekend at the virtual American Academy of Neurology meeting.