No­var­tis' SMA gene-ther­a­py, if priced at $2M, could be more cost-ef­fec­tive than Bio­gen's Spin­raza — ICER

Bio­gen’s Spin­raza was ap­proved by the FDA amidst much fan­fare in 2016 as the first and on­ly dis­ease-mod­i­fy­ing treat­ment for SMA, a rare and of­ten fa­tal ge­net­ic mus­cu­lar dis­or­der. But the price tag of $750,000 for the first year of ther­a­py (and a low­er price there­after) prompt­ed heavy crit­i­cism, al­though many pay­ers even­tu­al­ly agreed to re­im­burse the treat­ment. How­ev­er, a re­port by the In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) on Thurs­day has sug­gest­ed No­var­tis’ ex­per­i­men­tal SMA gene ther­a­py, Zol­gens­ma, could be more cost-ef­fec­tive in the long run ver­sus Spin­raza.

Zol­gens­ma is cur­rent­ly un­der FDA re­view and the agency is ex­pect­ed to an­nounce its de­ci­sion on the drug in May 2019. In its re­view, ICER has as­sumed Zol­gens­ma will car­ry a list price of $2 mil­lion, al­though the Swiss drug­mak­er has sug­gest­ed a price of $4 mil­lion for the cu­ra­tive ther­a­py, which it ac­quired via its $8.7 bil­lion takeover of AveX­is, may be jus­ti­fied.

De­spite the lack of long-term da­ta on ei­ther ther­a­py, the non-prof­it es­ti­mat­ed in­cre­men­tal cost-ef­fec­tive­ness of Spin­raza is $728,000 per QALY in presymp­to­matic SMA pa­tients, while Zol­gens­ma has an in­cre­men­tal cost-ef­fec­tive­ness of $247,000 per QALY in pa­tients with symp­to­matic Type I SMA (on the ba­sis of a place­hold­er price of $2 mil­lion).

QALYs, or qual­i­ty-ad­just­ed life-years, are a mea­sure of the state of health of a per­son or group in which the ben­e­fits — in terms of length of life — are ad­just­ed to re­flect the qual­i­ty of life. Es­sen­tial­ly, one QALY is equal to one year of life in per­fect health.

“No­var­tis has es­ti­mat­ed that Zol­gens­ma would yield 13.3 QALYs in SMA Type I pa­tients, and could be priced to $4-5 mil­lion based on the re­la­tion­ship of 10-year cost of ap­proved drugs for ul­tra-rare dis­eases and their cor­re­spond­ing in­cre­men­tal QALYs gained. ICER es­ti­mates Zol­gens­ma pro­vides 11.33 QALYs, and us­es a place­hold­er price of $2 mil­lion. While ICER un­der­cut both of No­var­tis’ es­ti­mates, the high cost/QALY of Spin­raza in Type I SMA pa­tients of­fers No­var­tis bar­gain­ing pow­er with pay­ers if the com­pa­ny can ar­gue Spin­raza use could be re­duced or elim­i­nat­ed to off­set to­tal costs,” Leerink an­a­lysts wrote in a note on Fri­day.

Cur­rent­ly avail­able da­ta on Spin­raza and Zol­gens­ma show pro­longed sur­vival and im­proved mo­tor func­tion com­pared with his­tor­i­cal con­trols or place­bo, but “there re­mains con­sid­er­able un­cer­tain­ty in the gen­er­al­iz­abil­i­ty of the re­sults and in the long-term dura­bil­i­ty and tol­er­a­bil­i­ty of treat­ment. In par­tic­u­lar, for both in­ter­ven­tions, the nar­row el­i­gi­bil­i­ty cri­te­ria of tri­als and the lim­it­ed sam­ple size (es­pe­cial­ly for Zol­gens­ma) rais­es con­cerns about gen­er­al­iz­abil­i­ty of re­sults to the wider pop­u­la­tion of pa­tients with SMA,” the re­port added.

Bio­gen un­der­scored this dif­fer­ence in sam­ple size in its re­sponse to the re­port. “The draft ICER re­port is an in­com­plete rep­re­sen­ta­tion of Spin­raza’s val­ue to pa­tients and health care sys­tems. Fur­ther­more, to com­pare a treat­ment that has helped near­ly 6,000 pa­tients world­wide against an in­ves­ti­ga­tion­al treat­ment on da­ta from 15 pa­tients with an ar­ti­fi­cial­ly se­lect­ed price is mis­lead­ing and wrong,” a spokesper­son told End­points News.

The ICER re­port is not fi­nal and has been opened to the pub­lic for com­ment, which will be in­cor­po­rat­ed in­to an ev­i­dence re­port in Feb­ru­ary 2019. This re­port will be sub­ject to fur­ther de­lib­er­a­tion by one of ICER’s in­de­pen­dent ev­i­dence ap­praisal com­mit­tees in ear­ly March, af­ter which a fi­nal re­port will be re­leased by the end of the month.

“If con­firmed in a fi­nal re­port…these find­ings could of­fer sup­port for No­var­tis in pric­ing and ac­cess ne­go­ti­a­tions for Zol­gens­ma ahead of 2019 ap­proval,” Leerink an­a­lysts added.

In re­sponse to the re­port, No­var­tis said that the find­ings had af­firmed the com­pa­ny’s ini­tial as­sess­ment of the val­ue of their prod­uct. “While we have not yet de­ter­mined the price of our in­ves­ti­ga­tion­al prod­uct…once we de­ter­mine a price, we are com­mit­ted to flex­i­bly part­ner­ing with health­care stake­hold­ers to en­sure ac­cess,” a spokesper­son told End­points News. 

Like NICE in the UK, ICER an­a­lyzes the ev­i­dence on the ef­fec­tive­ness and val­ue of drugs and oth­er med­ical ser­vices in the Unit­ed States, how­ev­er un­like NICE, it is not a gov­ern­ment-af­fil­i­at­ed body.

In Au­gust, NICE re­fused to rec­om­mend the use of Spin­raza in the UK — de­spite Bio­gen dis­count­ing its price — on the ba­sis that the long-term im­pact of the drug was un­clear for pa­tients, some of whom die by the age of 2. With­out that da­ta, the price of the drug couldn’t be jus­ti­fied, they said.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Brian Stuglik (file photo)

Turn­ing fo­cus to clin­i­cal work, Ve­rastem ax­es 31 jobs, scales back can­cer drug pro­mo­tion af­ter dis­ap­point­ing sales

Months after taking the helm at Verastem Oncology, Brian Stuglik has a plan to take the biotech in a “new strategic direction” — but not before some layoffs.

Left out of an upbeat press release spelling out its clinical plans, and buried below news of a $100 million private placement in an SEC filing, is a planned restructuring that will claim 31 jobs. Alongside some other cost-saving measures, Verastem expects to cut expenses down by $70 million to $80 million per year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Sage con­firms sus­pen­sion of 2 de­pres­sion tri­als af­ter PhI­II flop; Es­pe­ri­on fol­lows up maid­en ap­proval with com­bo OK

→ In the wake of a flop in the crucial Phase III MOUNTAIN study, Sage Therapeutics confirmed in its quarterly update that it’s suspended enrollment in two other pivotal trials for the oral depression drug SAGE-217 (or zuranolone) as it awaits guidance from the FDA. While REDWOOD (measuring relapse) and RAINFOREST (for patients with both major depressive disorder and insomnia) are on hold pending amendments, though, the open-label SHORELINE has completed enrollment. CEO Jeff Jonas remained tight-lipped about what specific tweaks they are considering for the program, reiterating only there have been issues with compliance and room for a higher dose.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 73,300+ biopharma pros reading Endpoints daily — and it's free.

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.