Io­n­is un­veils pos­i­tive PhII da­ta for in-house rare dis­ease drug, paving way to block­buster ri­val­ry with Take­da

One of the mid-stage an­ti­sense drugs for rare, non-neu­ro­log­i­cal dis­eases that Brett Mo­nia boast­ed about at JP Mor­gan ear­li­er this year has de­liv­ered a sol­id slate of pos­i­tive re­sults, boost­ing the CEO’s promise to turn Io­n­is in­to one of the biggest RNA biotechs with its whol­ly-owned pipeline.

With this par­tic­u­lar can­di­date, he may even be set­ting the com­pa­ny up for a ri­val­ry with Take­da.

The tri­al, which tests IO­N­IS-PKK-LRx in hered­i­tary an­gioede­ma, is small with 20 pa­tients. But the re­sults were un­equiv­o­cal: When you com­pare the 14 adults who re­ceived an 80mg month­ly dose of the drug to the six who got place­bo, you see a mean re­duc­tion of 90% in the num­ber of month­ly HAE at­tacks through weeks 1 to 17 in the study, meet­ing the pri­ma­ry end­point (p <0.001). From weeks five through 17 (one of the sec­ondary end­points), the mean re­duc­tion in month­ly at­tacks rose to 97% (p=0.003).

Dur­ing that short­er pe­ri­od, 92% of the 14 treat­ed with IO­N­IS-PKK-LRx were at­tack-free. None of the six pa­tients on place­bo reached that sta­tus (p <0.001).

A lig­and-con­ju­gat­ed an­ti­sense, IO­N­IS-PKK-LRx is de­signed to block prekallikrein, the pre­cur­sor to kallikrein, which in turn plays a key role in the ac­ti­va­tion of in­flam­ma­to­ry me­di­a­tors as­so­ci­at­ed with HAE at­tacks.

Kallikrein is the tar­get of Take­da’s block­buster con­tender Takhzy­ro (long­time ob­servers would re­mem­ber it as Shire’s lanadelum­ab), the first-ever drug to be ap­proved as a pro­phy­lac­tic for HAE. In late-stage tri­als, the an­ti­body had shown any­where from 73% to 87% re­duc­tion in those painful events.

For Stifel’s Paul Mat­teis, the topline da­ta from Io­n­is look promis­ing enough to po­ten­tial­ly com­pete with Take­da, es­pe­cial­ly giv­en that it has an “in­cre­men­tal­ly bet­ter dos­ing reg­i­men” of quar­ter­ly ad­min­is­tra­tion ver­sus every two to four weeks.

If the re­sults hold up in lat­er tri­als, that is. From the an­a­lyst note:

There are still a few things we don’t know here, in­clud­ing pa­tient base­line sever­i­ty, or col­or on AE rates. But head­ing in­to this cat­a­lyst, we had viewed the com­pet­i­tive bar as quite high giv­en Lanadelum­ab’s ro­bust ef­fi­ca­cy (an 80%+ re­duc­tion in HAE at­tacks); by com­par­i­son, PKK-LRx gen­er­at­ed a ~90% re­duc­tion in HAE at­tacks, and even bet­ter ef­fi­ca­cy than this af­ter the 5 week time-point. The sam­ple size for the Io­n­is study isn’t huge (N=20) and in re­al­i­ty the drugs are prob­a­bly more sim­i­lar than dif­fer­ent–these PKK da­ta are re­al­ly sim­i­lar to the lanadelum­ab ph1b when the drug was “DX2930”. But bot­tom line, this looks like a nice win for Io­n­is, for a drug they could take for­ward in­de­pen­dent­ly.

That last part is sig­nif­i­cant for Io­n­is. Stan­ley Crooke spent years team­ing up with big­ger drug­mak­ers that an­ti­sense could be a pow­er­ful RNA-based ap­proach for ma­nip­u­lat­ing gene ex­pres­sion, with a promi­nent ex­am­ple in the Bio­gen-part­nered spinal mus­cu­lar at­ro­phy block­buster drug Spin­raza. Af­ter some or­ga­ni­za­tion en­gi­neer­ing that in­volved spin­ning out the com­mer­cial port­fo­lio to Akcea, Mo­nia is ready to go it alone with nu­mer­ous late-stage can­di­dates to push past the fin­ish line on their own.

“In every­thing out­side of the liv­er — es­pe­cial­ly neu­ro, in pul­monary — we’re 100% dif­fer­en­ti­at­ed right now,” Mo­nia told End­points News at the vir­tu­al JPM con­fer­ence this Jan­u­ary.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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