As­traZeneca records an­oth­er R&D set­back, trig­ger­ing a melt­down at Synair­gen

More than two years af­ter As­traZeneca in-li­censed Synair­gen’s lead drug, the phar­ma gi­ant $AZN has had to scrap a Phase IIa study of the in­haled in­ter­fer­on be­ta for asth­ma. And the British biotech’s stock quick­ly tanked on the lat­est sna­fu to af­flict As­traZeneca.

The biotech said ear­ly Wednes­day that As­traZeneca opt­ed to halt the study ear­ly af­ter de­ter­min­ing that an over­all low num­ber of ex­ac­er­ba­tions among the asth­ma pa­tients re­cruit­ed would make it dif­fi­cult to see if they could ac­tu­al­ly hit the pri­ma­ry end­point that had been laid out. But in­ves­ti­ga­tors still plan to sift through the da­ta to see how the drug did on sec­ondary goals.

It all sound­ed like a mess to in­vestors, who pulled the plug on Synair­gen’s stock (LSE: SNG). Shares skid­ded down 36% Wednes­day morn­ing, shrink­ing the mi­cro­cap’s mar­ket val­ue.

As­traZeneca in-li­censed AZD9412 back in 2014 in a deal that start­ed small and then had a shot at swelling to $232 mil­lion. Asth­ma was the first tar­get, with plans to ex­pand that to COPD if they were suc­cess­ful. It was one of many deals that As­traZeneca struck around that time as it looked to mount a turn­around.

Suc­cess, though, has been elu­sive at As­traZeneca, which has ex­pe­ri­enced a se­ries of R&D set­backs this year as the Big Phar­ma out­fit start­ed to auc­tion off prod­ucts that had ei­ther gone off the rails or no longer fit in­to their pipeline plans.

Said Pro­fes­sor Stephen Hol­gate:

“New treat­ments to pre­vent se­vere ex­ac­er­ba­tions are need­ed and most ex­ac­er­ba­tions are caused by the com­mon cold and flu. Un­ex­pect­ed­ly, colds did not cause as many asth­ma ex­ac­er­ba­tions as were pre­dict­ed in this clin­i­cal tri­al pop­u­la­tion. We hope to learn from the re­sults of this tri­al which pop­u­la­tion with­in se­vere asth­ma, or oth­er res­pi­ra­to­ry dis­eases, will most ben­e­fit from AZD9412 and should be in­clud­ed in fu­ture tri­als.”

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 clinicaltrials.gov, 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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