Aim­mune's PhI­II peanut al­ler­gy study wows on the pri­ma­ry end­point, set­ting up FDA pitch -- and shares slide

Four months af­ter DBV Tech­nolo­gies stock was crushed by a shaky set of piv­otal da­ta for its peanut al­ler­gy drug, ri­val Aim­mune Ther­a­peu­tics $AIMT says its Phase III has come through with a promis­ing set of re­sults. And the win sets up an FDA ap­pli­ca­tion lat­er this year for the pi­o­neer­ing drug, with a Eu­ro­pean pitch to fol­low soon af­ter.

Of the 554 pa­tients in the study, 67.2% of the group in the AR101 drug arm were able to tol­er­ate at least a 600 mg dose of peanut pro­tein, pro­tect­ing them from a po­ten­tial­ly dead­ly al­ler­gic re­ac­tion against a small ex­po­sure. In the place­bo arm, on­ly 4% of the 4- to 17-year-olds in the study could say the same.

The da­ta were put on a slid­ing scale based on ex­po­sure, with 96.3% OK with a 300 mg dose of peanut pro­tein (about one peanut) and 50.3% of AR101 pa­tients tol­er­at­ing the sin­gle high­est dose of 1000 mg of peanut pro­tein.

Aim­mune shares ini­tial­ly shot up 21% in ear­ly trad­ing, while DBV saw its stock $DB­VT slide an­oth­er 10%. But the ral­ly didn’t last long. Aim­mune’s shares were down 3% Tues­day af­ter­noon.

Wes­ley Burks

“It’s great to have pa­tients go from man­ag­ing to tol­er­ate at most the amount of peanut pro­tein in a tenth of a peanut with­out re­act­ing to suc­cess­ful­ly eat­ing the equiv­a­lent of be­tween two to four peanuts with noth­ing more than mild, tran­sient symp­toms, if any at all,” said prin­ci­pal in­ves­ti­ga­tor Wes­ley Burks, from the Uni­ver­si­ty of North Car­oli­na School of Med­i­cine.

There was a high drop out rate, though, with 20.4% of AR101 pa­tients — ver­sus 6.5% of place­bo pa­tients — dis­con­tin­u­ing the tri­al, of­ten as a re­sult of an ad­verse event.

Com­pare those re­sults to DBV, and you’ll see a wide gulf sep­a­rat­ing the two.

The place­bo re­sponse in the DBV study — 13.6% — came in just a tiny bit high­er than the 12% re­sponse rate seen in a mid-stage con­trol arm. But the pos­i­tive re­sponse to their Vi­askin Peanut 250 μg dose — de­signed to get re­ac­tive im­mune sys­tems to tol­er­ate peanut pro­tein — tailed off con­sid­er­ably, drop­ping from 48% in Phase II to on­ly 35.3% in the late-stage tri­al af­ter 12 months of ther­a­py.

DBV nev­er­the­less said it would push for an FDA OK in any case.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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