Bio­gen, Io­n­is win FDA OK for spinal mus­cu­lar at­ro­phy drug Spin­raza

Bio­gen $BI­IB and Io­n­is $IONS got an ear­ly Christ­mas present Fri­day evening as the FDA an­nounced that it had pro­vid­ed an ac­cel­er­at­ed ap­proval for Spin­raza (nusin­ersen), their new drug — and the first — for rare cas­es of spinal mus­cu­lar at­ro­phy.

Peak sales es­ti­mates hov­er around $2 bil­lion, with some of the new es­ti­mates com­ing in over $3 bil­lion by 2026.

“Our pre­vi­ous fore­cast in­clud­ed sales of $75mm in 2017 grow­ing to $2.8bn by 2026,” not­ed Leerink’s Ge­of­frey Porges in ear­ly No­vem­ber, with the ar­rival of more Phase III da­ta. “We now as­sume a launch in Q1 2017 from Q2 2017 pre­vi­ous­ly and our cur­rent fore­cast is for sales of $92mm in 2017 in­creas­ing to $1.56bn by 2020 and then $3.2bn by 2026.”

Bio­gen says that it will be ready to ship the drug in a week.

Shares of Io­n­is shot up 10% and Bio­gen saw its stock climb 2% on the news.

The ap­proval comes a lit­tle less than two months af­ter re­searchers post­ed a fresh round of promis­ing da­ta on the drug. Us­ing the Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed re­port card for pa­tients’ mo­tor func­tions, the drug arm wit­nessed a mean 4.0 im­prove­ment on scores while the place­bo arm dropped by 1.9 points, a 5.9 point over­all spread in the drug’s fa­vor.

Io­n­is CEO Stan­ley Crooke

Both com­pa­nies bad­ly need­ed this win af­ter a year of up­heavals. The ap­proval will help set the stage for Bio­gen’s new CEO, Michel Vounatsos, to take the helm. Io­n­is, mean­while, can boast of a year-end vic­to­ry af­ter set­backs in the clin­ic. And an­a­lysts were im­pressed by the broad la­bel ap­plied by the FDA.

“La­bel­ing is not re­strict­ed to any spe­cif­ic SMA sub­type, in­di­cat­ing that it will like­ly be broad­ly pre­scribed for type 1, 2, and 3 pa­tients and while the epi­demi­ol­o­gy in SMA is not en­tire­ly de­fin­i­tive, a la­bel mak­ing the drug avail­able to all pa­tient types rep­re­sents sig­nif­i­cant ex­pan­sion po­ten­tial to the ini­tial mar­ket op­por­tu­ni­ty (though giv­en the un­met need in SMA, this pos­i­tive de­vel­op­ment is not en­tire­ly un­sur­pris­ing),” notes Jef­feries Bri­an Abra­hams.

The ap­proval al­so comes with a rare dis­ease pri­or­i­ty vouch­er, which can be used to ac­cel­er­ate re­view of an­oth­er pro­gram, or sold on the open mar­ket.

“There has been a long-stand­ing need for a treat­ment for spinal mus­cu­lar at­ro­phy, the most com­mon ge­net­ic cause of death in in­fants, and a dis­ease that can af­fect peo­ple at any stage of life,” said Bil­ly Dunn, MD, di­rec­tor of the Di­vi­sion of Neu­rol­o­gy Prod­ucts in the FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search. “As shown by our sug­ges­tion to the spon­sor to an­a­lyze the re­sults of the study ear­li­er than planned, the FDA is com­mit­ted to as­sist­ing with the de­vel­op­ment and ap­proval of safe and ef­fec­tive drugs for rare dis­eases and we worked hard to re­view this ap­pli­ca­tion quick­ly; we could not be more pleased to have the first ap­proved treat­ment for this de­bil­i­tat­ing dis­ease.”

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Te­va drops out of in­dus­try trade group PhRMA

Following in AbbVie’s footsteps, Teva confirmed on Friday that it’s dropping out of the industry trade group Pharmaceutical Research and Manufacturers of America (PhRMA).

Teva didn’t give a reason for its decision to leave, saying only in a statement to Endpoints News that it annually reviews “effectiveness and value of engagements, consultants and memberships to ensure our investments are properly seated.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Sanofi CFO Jean-Baptiste de Chatillon (L) and CEO Paul Hudson (Romuald Meigneux/Sipa via AP Images)

Sanofi sees downtick in flu sales as it preps for launch of RSV an­ti­body

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.

Christophe Weber, Takeda CEO (Photographer: Shoko Takayasu/Bloomberg via Getty Images)

Take­da fo­cus­es on ‘di­verse’ pipeline prospects on heels of two ac­qui­si­tions

After a whopping $4 billion asset buy from Nimbus Therapeutics, along with a $400 million deal with Hutchmed for a colorectal cancer drug, Takeda executives touted pipeline optimism on its latest earnings call this week.

That’s because the TYK2 inhibitor for psoriasis Takeda is getting from Nimbus, along with the Hutchmed fruquintinib commercialization outside of China, are just two of what it reports are 10 late-stage development programs of promising candidates.

Regeneron CSO George Yancopoulos (L) and CEO Len Schleifer at a groundbreaking for its new Tarrytown, NY facility, June 2022 (Lev Radin/Pacific Press/LightRocket via Getty Images)

In show­down with Roche, Re­gen­eron gears up for po­ten­tial Eylea ex­pan­sion amid Covid de­cline

Regeneron faced a substantial slump in overall revenue last year, but the focus still remains on some of its biggest blockbusters.

The pharma with several high-profile partnerships — Sanofi and Bayer among them — said Friday that Q4 revenue was down 31% for the quarter, and down 24% for the entire year. However, that won’t stop blockbuster expansion plans.

One of those is Eylea, the Bayer-partnered eye disease drug that has been in major competition with Roche’s Vabysmo. While Eylea is currently only approved in a 2 mg dose, the company recently filed for approval to give a 8 mg dose, in hopes of making a longer-lasting treatment.

BeiGene's new website helps direct cancer patients and caregivers to a wide variety of sources for help.

BeiGene re­veals men­tal health and can­cer care gap in study, de­buts dig­i­tal re­sources

One-fourth of cancer patients are living with depression — and another 20% suffer from anxiety. That’s according to new study results from BeiGene, conducted by Cancer Support Community (CSC), about the mental and emotional health of cancer patients.

While the fact that people with cancer are also dealing with depression or anxiety may not be surprising, what is — and was to BeiGene — is that a majority of them aren’t getting support. 60% of respondents said they were not referred to a mental health professional, and even more concerning, two in five who specifically asked for mental health help did not get it. CSC, a nonprofit mental health in cancer advocacy group, surveyed more than 600 US cancer patients.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

One of the paintings from Gilead's latest campaign making AI art to help MBC patients be 'seen and heard.'

Gilead com­bines ar­ti­fi­cial in­tel­li­gence and art to draw at­ten­tion and hope to MBC

What if you could “see” the emotions and feelings of people living with metastatic breast cancer? That’s what Gilead Sciences’ agency VMLY&R Health did last year, using artificial intelligence and sound analytics to turn the interviews of three women living with metastatic triple-negative breast cancer into works of art.

Using the sound waves, a robotic painting device translated their stories of struggle and hope into colors, contours and brush strokes. The result? An art exhibition called “Paintings of Hope” that was first displayed at ESMO in September in Paris, but has since traveled to hospitals and medical conferences in Europe and Spain.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.