Roche un­corks new long-term da­ta as SMA ri­val­ry heats up

Lead­ing up to a reg­u­la­to­ry de­ci­sion in May that could give Roche a big leg up on Bio­gen and No­var­tis in the spinal mus­cu­lar at­ro­phy space, the Swiss phar­ma gi­ant is hop­ing some new da­ta will bol­ster its case.

Roche un­corked some long-term ef­fi­ca­cy and safe­ty da­ta for its block­buster SMA drug Evrys­di (ris­diplam) on Wednes­day, while al­so of­fer­ing an­oth­er peek at the in­ter­im re­sults for its RAIN­BOW­FISH study in new­borns un­der 2 months old.

“In treat­ing peo­ple with SMA, our aim is to en­able or pre­serve their in­de­pen­dence and pa­tients in the SUN­FISH study re­port­ed con­tin­u­ous im­prove­ment or sta­bil­i­sa­tion in the lev­el of help need­ed for dai­ly liv­ing,” MDUK Ox­ford Neu­ro­mus­cu­lar Cen­tre pro­fes­sor Lau­rent Ser­vais said in a news re­lease.

The new da­ta come just a cou­ple months ahead of an ex­pect­ed de­ci­sion for Evrys­di to treat pre-symp­to­matic ba­bies younger than 2 months. A yes here would make it the first at-home treat­ment avail­able for younger in­fants.

In the SUN­FISH study — which en­rolled pa­tients be­tween 2 and 25 years old with types 2 or 3 SMA — pa­tients were able to main­tain their in­crease in Mo­tor Func­tion Mea­sure 32 (MFM-32) to­tal score from base­line (pre­vi­ous­ly ob­served at one year) through year three, ac­cord­ing to Roche. They were al­so able to sus­tain in­creas­es in Re­vised Up­per Limb Mod­ule (RULM) and Ham­mer­smith Func­tion­al Mo­tor Scale Ex­pand­ed (HFMSE) to­tal scores be­tween years one and three.

Levi Gar­raway

Pa­tients al­so re­port­ed few­er side ef­fects as time went on. The over­all rate of ad­verse events de­creased over three years, and in­ves­ti­ga­tors not­ed a trend to­ward a low­er rate of se­ri­ous ad­verse events in year three, Roche said. Over­all, those side ef­fects were re­flec­tive of un­der­ly­ing dis­ease, and no treat­ment-re­lat­ed ad­verse events led to with­draw­al from the study, ac­cord­ing to the com­pa­ny.

“We are pleased that these long-term re­sults fur­ther re­in­force the safe­ty and ef­fi­ca­cy of Evrys­di and it is es­pe­cial­ly en­cour­ag­ing to see that ad­verse events de­creased over time,” CMO Levi Gar­raway said in the re­lease.

As for RAIN­BOW­FISH, the lat­est in­ter­im re­sults sug­gest that the ma­jor­i­ty of ba­bies treat­ed with Evrys­di for at least a year were able to stand and walk with­in time­frames typ­i­cal of healthy ba­bies.

From the ab­stract pre­sent­ed this week at the Mus­cu­lar Dy­s­tro­phy As­so­ci­a­tion con­fer­ence:

Ef­fi­ca­cy da­ta from in­fants re­ceiv­ing ris­diplam for ?12 months (n=5) demon­strat­ed that these in­fants reached a Chil­dren’s Hos­pi­tal of Philadel­phia In­fant Test of Neu­ro­mus­cu­lar Dis­or­ders score of ?60 and main­tained swal­low­ing and feed­ing abil­i­ties. We will re­port up­dat­ed base­line de­mo­graph­ics and safe­ty da­ta in en­rolled in­fants and ef­fi­ca­cy da­ta in in­fants who have re­ceived ris­diplam for ?12 months.

Roche gave PTC Ther­a­peu­tics $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. The drug works 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.

Evrys­di first cleared the FDA back in 2020, be­com­ing the first oral treat­ment for SMA. Its com­peti­tors, Bio­gen’s Spin­raza and No­var­tis’ Zol­gens­ma, are ad­min­is­tered by in­jec­tion to the spinal cord and IV in­fu­sion, re­spec­tive­ly. But route of ad­min­is­tra­tion isn’t the on­ly thing set­ting Evrys­di apart from ri­vals.

From the start, Roche promised to “un­der­whelm” with Evrys­di’s price com­pared to com­peti­tors. The phar­ma 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. So for an in­fant who weighs 15 lbs and is less than 2 years old, for ex­am­ple, the an­nu­al price of Evrys­di would be less than $100,000, a spokesper­son told End­points News back in Jan­u­ary. Zol­gens­ma, on the oth­er hand, earned it­self the no­to­ri­ous ti­tle of most ex­pen­sive drug in the world at $2.1 mil­lion per pa­tient.

Evrys­di raked in more than $639 mil­lion in 2021 (602 mil­lion CHF), com­pared to just over $1.9 bil­lion for Spin­raza and $1.35 bil­lion for Zol­gens­ma.

As the weath­er heats up for spring, so might Roche’s ri­val­ry with Bio­gen and No­var­tis. The FDA grant­ed Roche a pri­or­i­ty re­view for its sup­ple­men­tal NDA in the new­born group back in Jan­u­ary, and the com­pa­ny said at the time that it ex­pects a de­ci­sion by May 30.

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

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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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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.

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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.

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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.

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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.