Levi Garraway, Roche CMO

Roche nabs a pri­or­i­ty re­view for Evrys­di, hop­ing to be­come the first oral treat­ment for in­fants with SMA

Roche has been duk­ing it out with Bio­gen and No­var­tis ever since burst­ing in­to the spinal mus­cu­lar at­ro­phy space about a year and a half ago with its com­pet­i­tive­ly priced oral treat­ment Evrys­di. Now the phar­ma gi­ant is look­ing to stake its claim in a younger sub­set of pa­tients — and the FDA has agreed to give it a speedy re­view.

Reg­u­la­tors have grant­ed pri­or­i­ty re­view to a sup­ple­men­tal NDA for Evrys­di (ris­diplam) to treat pre-symp­to­matic ba­bies un­der 2 months old with SMA. While the drug is cur­rent­ly ap­proved for adults, chil­dren and ba­bies old­er than 2 months, a new ap­proval here would make it the first at-home treat­ment avail­able for younger in­fants. A de­ci­sion is ex­pect­ed by May 30, ac­cord­ing to Roche.

“Treat­ing very young ba­bies with Evrys­di be­fore SMA symp­toms arise may help them to achieve mile­stones such as stand­ing and walk­ing with­in time­frames typ­i­cal of healthy in­fants,” CMO Levi Gar­raway said in a state­ment.

Evrys­di — li­censed by Roche from PTC Ther­a­peu­tics — is de­signed to treat SMA by mod­i­fy­ing how the SMN2 gene is spliced, thus in­creas­ing func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues. SMN pro­tein is crit­i­cal for main­tain­ing healthy mo­tor neu­rons and move­ment.

The drug got a quick OK back in 2020, be­com­ing the first oral treat­ment for SMA. An­a­lysts pen­ciled in a peak sales es­ti­mate of around $2 bil­lion. Its com­peti­tors, Bio­gen’s Spin­raza and No­var­tis’ Zol­gens­ma, are ad­min­is­tered by in­jec­tion to the spinal cord and IV in­fu­sion, re­spec­tive­ly.

Matthew Klein

“I think that that’s re­al­ly im­por­tant. One, just in gen­er­al, sav­ing peo­ple the in­con­ve­nience of go­ing to a hos­pi­tal. I think most peo­ple would much rather take some­thing by mouth than have a pro­ce­dure done, and prob­a­bly even more im­por­tant in COVID,” PTC COO Matthew Klein told End­points News. “I think peo­ple are re­al­ly not want­i­ng to… leave home, let alone go to med­ical fa­cil­i­ties.”

At an in­ter­im analy­sis in the RAIN­BOW­FISH study, all five pa­tients un­der 2 months old main­tained the abil­i­ty to swal­low and were able to feed ex­clu­sive­ly by mouth af­ter 12 months of treat­ment, ac­cord­ing to Roche. Four of those five pa­tients achieved mile­stones such as stand­ing and walk­ing in­de­pen­dent­ly, and all met the HINE-2 mo­tor mile­stones of head con­trol, sit­ting up­right, rolling and crawl­ing af­ter 12 months.

The drug ap­peared safe, with no treat­ment-re­lat­ed se­ri­ous events re­port­ed in any of the ba­bies. Four treat­ment-emer­gent side ef­fects were re­port­ed out of 12 ba­bies, all of which were re­solved or re­solv­ing with on­go­ing Evrys­di treat­ment. The most com­mon side ef­fects were nasal con­ges­tion (33%), cough (25%), teething (25%), vom­it­ing (25%), eczema (17%), ab­dom­i­nal pain (17%), di­ar­rhea (17%), gas­troen­teri­tis (17%), papule (17%) and pyrex­ia (17%).

“The AEs were re­flec­tive of the age of the ba­bies rather than the un­der­ly­ing SMA,” Roche said.

From the start, Roche promised to “un­der­whelm” with Evrys­di’s price com­pared to com­peti­tors. The phar­ma gi­ant set the price on a scale based on a child’s weight, max­ing out when they reach 44 lbs at about 6 years of age. For ex­am­ple, for an in­fant who weighs 15 lbs and is less than 2 years old, the an­nu­al price of Evrys­di would be less than $100,000, a spokesper­son told End­points.

Mean­while, Zol­gens­ma has earned the no­to­ri­ous ti­tle of the most ex­pen­sive drug in the world at $2.1 mil­lion per pa­tient, and back in 2020, Spin­raza cost be­tween $625,000 and $750,000 for the first year and around $375,000 every year af­ter.

Evrys­di’s low­er price hasn’t stopped it from reel­ing in mam­moth sales. In Q3 of 2021, the drug net­ted around $429 mil­lion, com­pared to Zol­gens­ma’s $375 mil­lion and Spin­raza’s $444 mil­lion. Spin­raza’s Q3 sales de­creased from $494 mil­lion in Q3 of 2020, which Bio­gen at­trib­uted to grow­ing com­pe­ti­tion and the im­pacts of Covid-19.

Roche shelled out $30 mil­lion up­front for the rights to Evrys­di back in 2011, promis­ing up to $460 mil­lion in biobucks and up to dou­ble-dig­it roy­al­ties on com­mer­cial sales. PTC raked in $35 mil­lion in mile­stone pay­ments in Q3 of 2020 and $7.5 mil­lion in Q4, ac­cord­ing to the com­pa­ny’s year-end re­port. The drug is cur­rent­ly ap­proved in 70 coun­tries and has been sub­mit­ted for ap­proval in 31 more.

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.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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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.

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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.

FDA ap­proves GSK's ane­mia drug with safe­ty warn­ing — af­ter bat­ting back sim­i­lar drugs

GSK has secured the first of four US approvals it’s hoping for this year, as the FDA greenlit daprodustat as a treatment for anemia due to chronic kidney disease.

But the FDA limited the use of the drug, to be marketed as Jesduvroq, to patients who have been receiving dialysis for at least four months and stopped short of approving it for patients not dependent on dialysis — in line with the recommendations of the advisory committee it consulted.

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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.”

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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.