Prize fight in Paris: Al­ny­lam and Io­n­is slug it out over PhI­II da­ta in prepa­ra­tion for a block­buster show­down

This morn­ing Al­ny­lam $AL­NY rolled out a bat­tery of stel­lar Phase III da­ta on their block­buster con­tender patisir­an, which promis­es to go on to be­come the com­pa­ny’s first ap­proved and mar­ket­ed drug af­ter 15 long years of RNAi dis­cov­ery and de­vel­op­ment work. And as of now, it looks like they’ll be go­ing head-to-head with the an­ti­sense ex­perts at Io­n­is $IONS, the un­der­dogs in what promis­es to be a long run­ning fight for a small group of pa­tients suf­fer­ing from rare cas­es of hered­i­tary AT­TR (hAT­TR) amy­loi­do­sis.

John Maraganore

For Al­ny­lam, the key points in the APOL­LO da­ta un­veiled to­day in Paris fo­cus on an im­pres­sive 34-point mean dif­fer­ence in the mod­i­fied neu­ropa­thy im­pair­ment score (mNIS+7) at 18 months, with a 6-point im­prove­ment for pa­tients which un­der­scores that they got some­what bet­ter over that pe­ri­od. There was al­so a 21.1-point mean im­prove­ment in the qual­i­ty of life score, with a 6.7-point im­prove­ment to show that the pa­tients al­so felt bet­ter.

Just hours ear­li­er, it was Io­n­is that took cen­ter stage, adding to its Phase III da­ta roll­out with a 19.73-point mean change in dis­ease scale at 15 months, and an 8.69-point ben­e­fit at 8 months. Their qual­i­ty of life scores reg­is­tered an 11.68-point mean im­prove­ment, with a 6.4-point ben­e­fit that showed their pa­tients al­so re­spond­ed bet­ter with treat­ment.

“We beat them on both,” Al­ny­lam CEO John Maraganore tells me flat­ly, “with the caveat that these aren’t com­par­a­tive (head-to-head) stud­ies. We clear­ly have a more sub­stan­tial treat­ment ef­fect.”

“This is a big win,” adds the CEO. “It’s a nice way to de­but the ad­vent of this tech­nol­o­gy in med­i­cine.”

The mar­ket agreed with Maraganore. Al­ny­lam shares shot up 13%, with Io­n­is down 8% af­ter the matchup.

Stan­ley Crooke. Im­age: OTS

Maraganore — who’s stuck with RNAi through the good years and the bad as Big Phar­ma dropped out or came in­to the emerg­ing field — hasn’t made his own peak sales pro­jec­tions here, but he al­so isn’t shy of not­ing an­a­lysts’ pro­jec­tions rang­ing up to $2 bil­lion worth of year­ly rev­enue. And he’s al­ready been ramp­ing up com­mer­cial ac­tiv­i­ties in the US and Eu­rope, while their big part­ner Sanofi $SNY takes on the rest of the world.

Io­n­is CEO Stan Crooke has heard the an­a­lysts cheer­ing Al­ny­lam. And he’s not about to roll over and play dead now.

“We think we’ll win in the mar­ket­place,” Crooke told me in the lead-up to to­day’s da­ta re­veal, adding that “there are peo­ple who think oth­er­wise.”

So what does Io­n­is have that Al­ny­lam doesn’t? Crooke — who hadn’t seen the Al­ny­lam re­sults when we talked ahead of Thurs­day’s ses­sions — says there’s lots on the ta­ble.

First, he can point to 8-month and 15-month re­sults which demon­strate sig­nif­i­cant suc­cess ahead the 18-month mark at Al­ny­lam. He’s not talk­ing price yet — no one does at this stage — but “the cost of ther­a­py will be sub­stan­tial­ly bet­ter.” And he’s heard Al­ny­lam hint­ing about Soliris style prices, which could land it on the list of the top 10 most ex­pen­sive ther­a­pies.

“We’ll win be­cause we think in­ot­ersen is easy to use,” he adds, adding point­ed­ly that this isn’t their first time out on the mar­ket. Where the Al­ny­lam ther­a­py has to be in­fused, ex­pos­ing pa­tients to re­ac­tions that can leave them un­able to work, he’ll be sell­ing a treat­ment that can be self-ad­min­is­tered at home.

Io­n­is, though, al­so had to deal with the death of a pa­tient from throm­bo­cy­tope­nia dur­ing its study, throw­ing in an added safe­ty fac­tor that could play to Al­ny­lam’s ad­van­tage — as many an­a­lysts would be will­ing to tell you. Since that case oc­curred, though, the biotech added a more care­ful screen­ing process to pre­vent any new in­ci­dents.

“I think both drugs will be very suc­cess­ful,” Crooke sums up. But any­one who counts Io­n­is out now, he adds, is in for a big sur­prise.

The sales num­bers will pro­vide the score on who’s win­ning.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

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But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

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In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA rejected Mesoblast’s remestemcel-L, or Ryoncil, its lead candidate for pediatric acute graft-versus-host disease (aGVHD) that didn’t respond to steroids. The FDA raised a number of concerns, first objecting to Mesoblast’s single arm, open-label trial, though regulators struggled to describe how a randomized trial would work, since pediatricians and parents were reluctant to put children with aGVHD in a placebo arm.

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Scan­dion's shares fall af­ter on­col­o­gy biotech re­ports PhII fail

Danish biotech Scandion Oncology posted some Phase II results on Friday, and investors were none too pleased.

The biotech reported topline results from the second part of an ongoing Phase II trial called CORIST. The study was investigating Scandion’s lead candidate SCO-101 in 25 patients as a combination treatment with FOLFIRI chemotherapy in metastatic colorectal cancer. And so far, the study did not meet the biotech’s primary endpoint: tumor reduction of at least 30%.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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