CEO Amit Munshi (Arena Pharmaceuticals)

Are­na's lead drug miss­es on PhII in AD, thwack­ing stock, but com­pa­ny sol­diers on in­to Phase III

Amit Mun­shi’s tenure at Are­na Phar­ma­ceu­ti­cals has al­most ex­clu­sive­ly been one of suc­cess: Tak­ing the reins of a com­pa­ny in the Nas­daq gut­ter, he’s steered it through mul­ti­ple pos­i­tive read­outs and even bagged an $800 mil­lion up­front deal on a non-lead drug.

On Mon­day, though, the com­pa­ny an­nounced a fail­ure: Its lead drug, the S1P in­hibitor etrasi­mod, missed the pri­ma­ry end­point on a Phase II tri­al for atopic der­mati­tis, fail­ing to re­duce symp­toms on a stan­dard clin­i­cal scale bet­ter than place­bo. Yet they will con­tin­ue on with a Phase III, bet­ting that aber­ra­tions at one tri­al site thwart­ed that end­point and that they can re­peat suc­cess on a met­ric the FDA us­es to eval­u­ate ap­proval.

“We be­lieve we have a mol­e­cule that is head­ed to­wards be­ing a safe and ef­fec­tive oral op­por­tu­ni­ty,” Mun­shi told End­points News. “We think that’s go­ing to be a po­ten­tial game chang­er for this space.”

In­vestors, at least ini­tial­ly, weren’t buy­ing it. Are­na’s stock $AR­NA fell from $87 to $70 post-mar­ket in the min­utes af­ter the an­nounce­ment.

It’s not un­com­mon for com­pa­nies to launch Phase II­Is af­ter Phase II went bust, al­though the re­sults are not al­ways pret­ty. In the past few months, such turn­around at­tempts from Gen­fit and Au­rinia each failed.

Mun­shi, though, says he has a case. The drug, he not­ed, hit on 4 dif­fer­ent sec­ondary end­points, in­clud­ing the one FDA asks for to ob­tain ap­proval: a clin­i­cal score called val­i­dat­ed in­ves­ti­ga­tor glob­al as­sess­ment.

Chris Ca­bell

They on­ly failed on the pri­ma­ry end­points — re­duc­tion on the eczema area and sever­i­ty in­dex, or EASI — be­cause of a “slight anom­aly,” CMO Chris Ca­bell ar­gued.

Nine pa­tients on the drug arm, most of whom were at a sin­gle tri­al site, were tak­en off the drug af­ter their white blood cell counts fell.

Ca­bell said the com­pa­ny point­ed out that re­duced white blood cell counts were part of the drug’s mech­a­nism of ac­tion and how it re­duced symp­toms of au­toim­mune dis­eases. The pa­tients were put back on and re­spond­ed to the drug again, he said, but nev­er reached the lev­el of re­sponse seen in oth­er par­tic­i­pants.

If you re­move those pa­tients from the cal­cu­lus, the tri­al would hit the pri­ma­ry end­point, Mun­shi said.

“The drug was do­ing ex­act­ly what it was sup­posed to be do­ing,” Mun­shi told End­points. “There were a hand­ful of sites — par­tic­u­lar­ly one site — that over­re­act­ed.”

Mun­shi said they got the da­ta over the week­end and reached out to a half-dozen ex­perts, who en­cour­aged them to run the Phase III tri­als.

They’ll now have a chance to prove if it was just those 9 pa­tients. The goal is to bring for­ward a drug that has the ef­fi­ca­cy of Sanofi and Re­gen­eron block­buster Dupix­ent but in a more ac­ces­si­ble oral form and with­out the safe­ty con­cerns of JAK in­hibitors. They’ve al­ready brought the drug in­to Phase III for ul­cer­a­tive col­i­tis af­ter a suc­cess­ful Phase II.

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.
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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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