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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 102,000+ biopharma pros reading Endpoints daily — and it's free.

Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

Photo: Shutterstock

Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.