David Zaccardelli, Verona Pharma CEO

Verona says sec­ond PhI­II for COPD drug hits the mark, plans 2023 NDA fil­ing

UK-based biotech Verona Phar­ma said Tues­day morn­ing that its drug, en­sifen­trine, passed its sec­ond late-stage tri­al for the com­mon lung dis­ease COPD.

In Au­gust, Verona re­port­ed the drug suc­ceed­ed in the first of its two Phase III tri­als. Now, Verona will pack­age to­geth­er the new tri­al da­ta in its ap­proval fil­ing to the FDA, which the biotech said will come in the first half of next year.

Verona’s stock $VR­NA opened around 30% above its last close, jump­ing from un­der $14 to near­ly $17.

The sec­ond tri­al’s de­sign is sim­i­lar to the first — 500 pa­tients got en­sifen­trine as their COPD main­te­nance treat­ment, 300 got place­bo and all were fol­lowed for 24 weeks — but in the sec­ond tri­al, 400 pa­tients were fol­lowed for an ad­di­tion­al 24 weeks for long-term safe­ty da­ta. As in the first tri­al, the ma­jor­i­ty of pa­tients en­rolled were al­ready re­ceiv­ing some form of COPD treat­ment; around two-thirds were on a long-act­ing in­haler as back­ground ther­a­py, while just over 20% got in­haled cor­ti­cos­teroids on top of that.

Pa­tients on Verona’s drug best­ed those on place­bo in a breath­ing test that mea­sures the abil­i­ty to force­ful­ly breathe out — the pri­ma­ry end­point of the study — at week 12, and those re­sults were con­sis­tent re­gard­less of back­ground med­ica­tion, Verona said.

Chris Mar­tin

The study al­so met a num­ber of sec­ondary end­points look­ing at lung func­tion, dai­ly COPD symp­toms and over­all qual­i­ty of life. The on­ly point on which en­sifen­trine nar­row­ly missed sta­tis­ti­cal sig­nif­i­cance was in re­duc­ing the rate of mod­er­ate to se­vere COPD ex­ac­er­ba­tions. Here, pa­tients in the treat­ment arm had a 36% re­duc­tion in rate of ex­ac­er­ba­tions over 24 weeks com­pared to place­bo, good for a p-val­ue of 0.0505.

Verona’s drug, if ap­proved, would in­tro­duce a new class of COPD ther­a­pies, and SVP of com­mer­cial Chris Mar­tin not­ed Verona be­lieves en­sifen­trine “has the po­ten­tial based on the da­ta to be priced at a pre­mi­um ver­sus the cur­rent neb­u­lised prod­ucts.” Verona is al­so test­ing the drug, which is its on­ly can­di­date, in mid-stage stud­ies in cys­tic fi­bro­sis and asth­ma.

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.

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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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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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.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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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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Bet­ter Ther­a­peu­tics cuts 35% of staff while await­ing dig­i­tal ther­a­peu­tic ap­proval

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.