Kevin Lee, Bicycle Therapeutics CEO (Bicycle)

Strug­gling Io­n­is li­cens­es Bi­cy­cle Ther­a­peu­tic­s' plat­form in a bid to de­vel­op drugs that can cross blood-brain bar­ri­er

At­tempt­ing to re­bound from set­backs ear­li­er this year, Io­n­is has en­list­ed a new part­ner in a quest to cre­ate drugs that can cross the blood-brain bar­ri­er. And the tech­nol­o­gy comes from a British biotech pri­mar­i­ly known for its ef­forts in­to on­col­o­gy.

The col­lab­o­ra­tion will see Io­n­is ex­clu­sive­ly li­cense a plat­form from Bi­cy­cle Ther­a­peu­tics to de­vel­op oligonu­cleotides tar­get­ing the trans­fer­rin re­cep­tor 1, the com­pa­nies an­nounced Tues­day morn­ing. Bi­cy­cle is nab­bing $45 mil­lion up­front and an $11 mil­lion eq­ui­ty in­vest­ment from Io­n­is.

There are al­so mile­stones for R&D and com­mer­cial­iza­tion in­volved — though the ex­act fig­ures re­main undis­closed, each mile­stone will be in the “low sin­gle-dig­it mil­lion amount,” Bi­cy­cle CEO Kevin Lee told End­points News.

Tues­day’s deal came out of an eval­u­a­tion and op­tion agree­ment the two signed last De­cem­ber, where Io­n­is gave Bi­cy­cle $3 mil­lion to use the plat­form to val­i­date po­ten­tial tar­gets, Lee said. Over the last sev­er­al months, the re­search proved fruit­ful enough where Io­n­is ex­er­cised its op­tion to ful­ly li­cense the tech. The $3 mil­lion fig­ure makes up a part of Tues­day’s up­front sum, Lee added.

Io­n­is plans to uti­lize Bi­cy­cle’s plat­form to de­vel­op what they’ve termed ad­vanced lig­and con­ju­gat­ed an­ti­sense med­i­cines, or LI­CAs, for use with oligonu­cleotides to tar­get the trans­fer­rin re­cep­tor 1. Io­n­is has been work­ing on LI­CAs for some time, and their goal is os­ten­si­bly to bring a new method to fur­ther push these com­pounds’ de­vel­op­ment.

Trans­fer­rin re­cep­tor 1 plays a role in the body’s nat­ur­al abil­i­ty to ship iron be­tween the blood-brain bar­ri­er and in­to skele­tal mus­cle, Lee not­ed.

Bi­cy­cle’s plat­form al­lows for the cre­ation of syn­thet­ic short pep­tides that are con­strained to form two loops. The struc­ture is key in drugs that can de­liv­er pay­loads to their tar­gets ef­fec­tive­ly, and Lee said Bi­cy­cle has done this on its own to cre­ate can­cer drugs de­signed to bet­ter re­tain such car­go with­in sol­id tu­mors.

The biotech’s strat­e­gy thus far has been to fo­cus in­ter­nal re­sources in­to on­col­o­gy and part­ner with oth­er com­pa­nies who are in­ter­est­ed in us­ing them in oth­er fields, Lee said. Tues­day’s oligonu­cleotide deal is em­blem­at­ic of that strat­e­gy, he added, as Bi­cy­cle re­tains full rights to the plat­form.

“They’ve tak­en on most of the risk,” Lee said of Io­n­is. “But it’s still re­al­ly ex­cit­ing in terms of what they plan to do with the tech­nol­o­gy and what they can de­rive from these agents with oligonu­cleotide de­liv­ery … in do­ing so we can har­ness the po­ten­tial of the tech­nol­o­gy.”

Bi­cy­cle had been work­ing on adapt­ing the plat­form for trans­fer­rin re­cep­tor 1 since 2019 in a part­ner­ship with the De­men­tia Dis­cov­ery Fund, and with the new deal, Io­n­is can build on that foun­da­tion. It’s not the first col­lab­o­ra­tion Bi­cy­cle has en­tered to ex­pand the plat­form be­yond on­col­o­gy. Back in 2016, it teamed up with As­traZeneca in a pact worth about $1 bil­lion and has since signed on to part­ner­ships with Roche and the UK gov­ern­ment.

For Io­n­is, the deal marks part of an ef­fort to re­bound from two high-pro­file miss­es ear­li­er this year, af­ter a Hunt­ing­ton’s can­di­date failed in March and af­ter the com­pa­ny scrapped its ear­ly-stage cys­tic fi­bro­sis pro­gram in May. And in 2020, Io­n­is re­ab­sorbed the biotech Akcea af­ter spin­ning it out three years ear­li­er, and soon put 70% of its work­force on the chop­ping block.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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Covid-19 roundup: J&J boost­er shot da­ta show promise; CD­C's ACIP meet­ing this week to dis­cuss Pfiz­er boost­ers

J&J revealed a summary of new Covid-19 vaccine data today, including new results showing booster shots may help with protection.

A Phase III study (ENSEMBLE 2) looked at booster shots at two different points in time: a second shot 56 days after the first shot, or a second shot six months after the first. The eight-week shot showed increased protection against symptomatic Covid-19, with the following levels of protection: