Sergio Traversa, Relmada Therapeutics CEO

Rel­ma­da re­ports a PhI­II fail, blam­ing 'para­dox­i­cal' place­bo ef­fect — shares wiped

A Flori­da biotech at­tempt­ing to de­vel­op a rapid-act­ing an­ti­de­pres­sant ran face-first in­to a brick wall Thurs­day.

Rel­ma­da Ther­a­peu­tics re­port­ed a Phase III fail for its lead com­pound, REL-1017, in ma­jor de­pres­sive dis­or­der, not­ing the pro­gram did not sep­a­rate from place­bo af­ter 28 days in a de­pres­sion rat­ing scale mea­sur­ing se­vere episodes. Though the da­ta were nu­mer­i­cal­ly sim­i­lar, Rel­ma­da did not re­port a p-val­ue to il­lus­trate by how much REL-1017 whiffed.

In­vestors were hav­ing none of it. Be­fore the mar­ket opened Thurs­day morn­ing, Rel­ma­da shares $RLMD cratered near­ly 80%, send­ing the stock price to its low­est point in more than three years.

Re­searchers were at­tempt­ing to show an an­ti­de­pres­sive ef­fect us­ing the Mont­gomery-As­berg De­pres­sion Rat­ing Scale, which quan­ti­fies the sever­i­ty of de­pres­sive episodes in adults. Af­ter 28 days, pa­tients who took the drug saw their scores de­crease by 14.8 points com­pared to 13.9 points from those on place­bo.

Rel­ma­da blamed an un­fore­seen spike in the place­bo re­spons­es, de­scrib­ing re­sults from cer­tain tri­al sites as “para­dox­i­cal.” The biotech says it will try to fig­ure out why this proved to be the case, go­ing as far as to say a post-hoc analy­sis that ex­clud­ed the ques­tion­able sites showed a sta­tis­ti­cal­ly sig­nif­i­cant ben­e­fit.

The goal is to cre­ate a de­pres­sion drug that acts rapid­ly, as op­posed to ap­proved SS­RIs that may take weeks to start work­ing. The FDA ap­proved a rapid-act­ing NM­DA re­cep­tor an­tag­o­nist for MDD in Au­gust, to be mar­ket­ed as Au­veli­ty by Ax­some Ther­a­peu­tics.

Rel­ma­da is run­ning two ad­di­tion­al Phase III stud­ies to see if REL-1017 works in com­bi­na­tion with oth­er ther­a­pies. But Thurs­day’s fail­ure makes the biotech’s mar­gin for er­ror ra­zor-thin giv­en this is Rel­ma­da’s on­ly clin­i­cal pro­gram.

Pre­vi­ous stud­ies had shown two dose lev­els to be ef­fec­tive in treat­ment-re­sis­tant de­pres­sion. REL-1017 is de­signed as a pill tak­en oral­ly once per day. The biotech’s oth­er pro­gram fo­cus­es on psilo­cy­bin and its de­rivates af­ter sign­ing a li­cense agree­ment last year with Ar­bor­men­tis.

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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