Stick­er shock: No­var­tis says its top drug prospect is worth $4M-$5M for a once-and-done use

Val­ue and pric­ing in life-long rare dis­ease treat­ment. (No­var­tis, page 48)

AveX­is man­aged to grab the in­dus­try’s at­ten­tion when re­searchers post­ed ear­ly and stun­ning re­sults for their life-sav­ing gene ther­a­py to treat rare cas­es of spinal mus­cu­lar at­ro­phy. They were back in the spot­light when No­var­tis reached out to buy the com­pa­ny for $8.7 bil­lion. And now they’re mak­ing a re­turn trip to cen­ter stage with an ar­gu­ment that their once-and-done ap­proach to SMA — AVXS-101 — could jus­ti­fi­ably be priced at $4 mil­lion to $5 mil­lion.

Dave Lennon (LinkedIn)

“Four mil­lion dol­lars is a sig­nif­i­cant amount of mon­ey, but we be­lieve this is a cost-ef­fec­tive point,” Dave Lennon, pres­i­dent of AveX­is told in­vestors ear­li­er to­day, ac­cord­ing to a re­port from Reuters. “We’ve shown through oth­er stud­ies we are cost ef­fec­tive in the range of $4-$5 mil­lion. And ul­ti­mate­ly, this is im­por­tant con­text as we con­sid­er how we’re go­ing to eval­u­ate val­ue for (the ther­a­py called) AVXS-101.”

No­var­tis out­lined their ar­gu­ment in a slide that used price guide­lines from the cost watch­dogs at ICER and NICE to make their case. ICER, for ex­am­ple, backed Roche’s $482,000 first-year price for Hem­li­bra, with a 10-year cost of $4.5 mil­lion. But it’s hard to see ei­ther group go­ing for $4 mil­lion worth of stick­er shock for a one-time ther­a­py. ICER re­ject­ed Spark’s $850,000 price tag for its gene ther­a­py for a rare form of blind­ness as ex­treme and need­ed to be cut by at least 50% to re­flect its true val­ue to pa­tients. And NICE has had no prob­lem re­ject­ing oth­er rare dis­ease drugs with much small­er prices at­tached.

No­var­tis may not ac­tu­al­ly be plan­ning to try and col­lect $4 mil­lion for their drug, and ex­ecs de­murred on pro­ject­ing the cost to­day. But they seem in­tent on soft­en­ing up the pay­er mar­ket for some­thing sig­nif­i­cant­ly high­er than $1 mil­lion — once con­sid­ered a jaw-drop­ping, break-the-bank sum.

And that would put them at the very top of the heap of the most ex­pen­sive drugs on the plan­et.

In their fa­vor: Ri­vals at Bio­gen charge $750,000 for the first year of Spin­raza, then $375,000 a year af­ter that — for life. That’s $1.87 mil­lion over 4 years. And they don’t get much grief for it.

At the very least, you can ex­pect a whole new dis­cus­sion around the pric­ing of gene ther­a­pies, as de­vel­op­ers dis­cuss dif­fer­ent pay plans to spread out the cost or of­fer var­i­ous mon­ey-back guar­an­tees.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.