Levi Garraway (Genentech)

UP­DAT­ED: Roche re­leas­es more Evrys­di da­ta lead­ing up to FDA de­ci­sion for very young in­fants with SMA

One month away from an FDA de­ci­sion on Evrys­di for in­fants un­der two months old, Roche’s Genen­tech has re­leased new long-term da­ta on its oral drug for spinal mus­cu­lar at­ro­phy (SMA).

Dalia Moawad

The da­ta show that, af­ter three years of treat­ment, 91% of chil­dren who be­gan tak­ing the drug at ages 1-7 months were alive (n=58). In ad­di­tion, of 48 chil­dren eval­u­at­ed, 32 main­tained and four gained the abil­i­ty to sit with­out sup­port.

“These long-term re­sults in ba­bies treat­ed with Evrys­di are very en­cour­ag­ing, with the vast ma­jor­i­ty im­prov­ing or main­tain­ing mo­tor func­tions af­ter three years. With­out treat­ment, they would typ­i­cal­ly not sur­vive be­yond two years of age,” Genen­tech CMO Levi Gar­raway said in a press re­lease.

“Ex­tend­ing treat­ment ac­cess for young mem­bers of the SMA com­mu­ni­ty is cru­cial,” Dalia Moawad, neu­ro rare dis­ease Lead at Genen­tech, told End­points News in an email. “If the sN­DA is ac­cept­ed, Evrys­di would be the first med­i­cine ad­min­is­tered at-home for pre-symp­to­matic ba­bies liv­ing with SMA.”

Matthew Klein

In 2020, the FDA ap­proved Evrys­di (ris­diplam) for in­fants aged 2 months and old­er, mak­ing it one of three treat­ments avail­able to chil­dren with the rare ge­net­ic dis­or­der, along­side Bio­gen’s Spin­raza and No­var­tis’ Zol­gens­ma. Un­like the two oth­er treat­ments, which come as an in­jec­tion in the spine (Spin­raza) or a one-time IV in­fu­sion (Zol­gens­ma), Evrys­di can be tak­en at home.

“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,” Matthew Klein, COO of PTC Ther­a­peu­tics — the com­pa­ny from which Roche and Genen­tech li­censed Evrys­di — said to End­points ear­li­er this year.

How­ev­er, for a rare dis­ease in which ear­li­er treat­ment is key, cur­rent­ly on­ly Evrys­di is not ap­proved for use in very young in­fants.

In­fants with SMA have a ge­net­ic mu­ta­tion to their SMN1 gene, which en­codes a pro­tein cru­cial to mus­cle move­ment (SMN lit­er­al­ly stands for “sur­vival mo­tor neu­ron”). Chil­dren with SMA may strug­gle with sit­ting up, swal­low­ing, or breath­ing. Evrys­di works by pro­mot­ing the oth­er SMN gene, SMN2, to make more of that es­sen­tial pro­tein.

Spin­raza was the first SMA drug ap­proved in late 2016. Zol­gens­ma, a gene ther­a­py for chil­dren un­der 2 years of age, fol­lowed in 2019, mak­ing head­lines as the world’s most ex­pen­sive treat­ment at $2.1 mil­lion. Evrys­di, gain­ing ini­tial ap­proval in 2020, hopes to be­come the first at-home drug for in­fants un­der 2 months of age.

Ad­verse events were “gen­er­al­ly re­flec­tive of the un­der­ly­ing dis­ease,” Moawad said, not­ing that there were no treat­ment-re­lat­ed ad­verse events that re­sult­ed in with­draw­al or treat­ment dis­con­tin­u­a­tion in the FIRE­FISH tri­al. The most com­mon ad­verse events were fever, URI, and pneu­mo­nia, though the rate of se­vere ad­verse events de­creased as chil­dren got old­er and had longer treat­ment times.

The com­pa­ny ex­pects an FDA de­ci­sion by May 30, ac­cord­ing to a pre­vi­ous state­ment.

This sto­ry has been up­dat­ed to in­clude com­ments from Dalia Moawad.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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SQZ Biotech slash­es head­count by 60% as founder/CEO hits ex­it — while Syn­log­ic lays off 25%

It’s a tough time for early-stage companies developing highly promising, but largely unproven, new technologies.

Just ask SQZ Biotechnologies and Synlogic. The former is bidding farewell to its founder and CEO and slashing the headcount by 60% as it pivots from its original cell therapy platform to a next-gen approach; the latter — a synthetic biology play founded by MIT’s Jim Collins and Tim Lu — is similarly “optimizing” the company to focus on lead programs. The resulting realignment means 25% of the staffers will be laid off.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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AbCellera founder and CEO Carl Hansen (L) and Rallybio CEO Martin Mackay

Rally­bio, Ab­Cellera form new part­ner­ship around an­ti­bod­ies for rare dis­ease

Two biotechs that have been working heavily on different stages of antibody candidate development over the past several years are looking to work together to find potential candidates for rare diseases.

Canadian-based AbCellera and Connecticut-based Rallybio have entered a strategic partnership to find, develop and commercialize antibodies primarily for rare diseases. The multi-year, multi-target deal will seek to combine AbCellera’s antibody “discovery engine” with Rallybio’s expertise in rare diseases. However, the dollar amount for the deal was not disclosed.