Jeff Leiden (Vertex via YouTube)

Out­go­ing CEO Jeff Lei­den takes vic­to­ry lap as Ver­tex's Trikaf­ta stuns Wall Street

The first set of quar­ter­ly sales for Ver­tex’s re­cent­ly ap­proved cys­tic fi­bro­sis triplet — Trikaf­ta — as­tound­ed an­a­lysts on Thurs­day, as out­go­ing com­pa­ny chief Jeff Lei­den took his fi­nal lap in the “team sport” he calls drug de­vel­op­ment.

Six weeks in­to its launch the CF ther­a­py has al­ready be­come the top-sell­ing drug in Ver­tex’s port­fo­lio. Ap­proved in Oc­to­ber, quar­ter­ly Trikaf­ta sales hit a mas­sive $420 mil­lion (in­clud­ing a $100 mil­lion launch-re­lat­ed chan­nel in­ven­to­ry build) in the Unit­ed States — record­ing the strongest first quar­ter of sales for any drug, BMO Cap­i­tal Mar­kets’ an­a­lyst Do Kim es­ti­mat­ed. Con­sen­sus Wall Street ex­pec­ta­tions for the reg­i­men were a mere $70 mil­lion.

The com­pa­ny — long ad­mired by a crop of an­a­lysts for its com­mer­cial prowess, abil­i­ty to suc­cess­ful­ly piv­ot and over­come re­im­burse­ment hur­dles at home and abroad — was heaped with a steam­ing pile of praise.

“(2020) Guid­ance al­so stronger than ex­pect­ed – and con­sis­tent with our pos­i­tive the­sis the com­pa­ny is ex­e­cut­ing on the com­mer­cial front and will dri­ve in­dus­try-lead­ing ro­bust rev­enue and earn­ings growth over the next few years – best in the in­dus­try right now,” Jef­feries an­a­lysts wrote in a note.

The cys­tic fi­bro­sis drugs made by Ver­tex are the first treat­ments that ad­dress the un­der­ly­ing ge­net­ic caus­es of CF, which is char­ac­ter­ized by thick sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans that re­duces life ex­pectan­cy. Ver­tex’s med­i­cines tar­get the cys­tic fi­bro­sis trans­mem­brane con­duc­tance reg­u­la­tor (CFTR) gene and are en­gi­neered to cor­rect the mal­func­tion­ing pro­tein it makes.

A lit­tle over 300 dif­fer­ent mu­ta­tions are known to cause CF, and pa­tients typ­i­cal­ly car­ry path­o­gen­ic mu­ta­tions in both copies of the CFTR gene — the most com­mon such mu­ta­tion is F508del. The com­pa­ny’s Orkam­bi and Symke­vi fo­cus on the more com­mon F508del mu­ta­tion. The FDA ap­proved Ver­tex’s triplet reg­i­men, which is set to treat 90% of all CF pa­tients — a spec­tac­u­lar five months ahead of its ex­pect­ed de­ci­sion date in Oc­to­ber.

To­tal sales from its quar­tet of CF ther­a­pies reg­is­tered at $1.26 bil­lion, hand­some­ly beat­ing con­sen­sus es­ti­mates by 26% dri­ven by Trikaf­ta adop­tion, SVB Leerink’s Ge­of­frey Porges wrote in a note, adding that Ver­tex’s 2020 top-line pro­jec­tion of $5.1 bil­lion to 5.3 bil­lion was al­so ma­te­ri­al­ly high­er than Wall Street ex­pec­ta­tions of $4.8 bil­lion.

Un­der Jeff Lei­den, the Boston biotech has en­joyed a re­mark­able run of suc­cess af­ter shift­ing fo­cus from its ini­tial hep C fo­cus to CF. He will shift from his CEO role to the ex­ec­u­tive chair­man’s spot, and take over the reins of busi­ness de­vel­op­ment.

In 2019, Ver­tex was busy shop­ping. The com­pa­ny spent a com­bined $1.6 bil­lion across a num­ber of deals in­clud­ing the pur­chase of a drug de­vel­op­er work­ing on a stem-cell-based po­ten­tial cure for di­a­betes and for ac­cess to a small mol­e­cule plat­form for drug­ging RNA. As of Au­gust, Lei­den sug­gest­ed his ap­petite for deals re­mains in­tact.

De­spite some choice words for Eng­land’s law­mak­ers, Lei­den al­so steered the com­pa­ny to clinch a deal with UK au­thor­i­ties to cov­er its slate of CF drugs fol­low­ing years of pro­tract­ed ne­go­ti­a­tions. Ver­tex al­so suc­cess­ful­ly ne­go­ti­at­ed deals with au­thor­i­ties in Scot­land, France, Spain and Aus­tralia for its CF drugs.

As cur­rent CMO Resh­ma Ke­wal­ra­mani moves in­to the CEO chair in April, all eyes are on Ver­tex’s pipeline, which is fo­cused on six emerg­ing dis­ease ar­eas in­clud­ing Al­pha-1 An­tit­rypsin De­fi­cien­cy, pain, sick­le cell dis­ease, be­ta-tha­lassemia, and APOL1-me­di­at­ed kid­ney dis­eases to di­ver­si­fy from their es­tab­lished CF fran­chise.

On Thurs­day, the com­pa­ny re­vealed it had aban­doned an ear­ly-stage pro­gram for its ex­per­i­men­tal painkiller, VX-961, be­cause it did not dis­play op­ti­mal PK and tol­er­a­bil­i­ty pro­file.

In No­vem­ber, the com­pa­ny and part­ner CRISPR Ther­a­peu­tics un­veiled en­cour­ag­ing pre­lim­i­nary da­ta on two pa­tients with blood dis­or­ders that have been ad­min­is­tered with their gene-edit­ing ther­a­py, in­di­cat­ing their CRISPR/Cas9 tech­nol­o­gy is safe and ef­fec­tive.

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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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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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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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.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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