Roche, PTC will now get an FDA de­ci­sion on their SMA drug in 6 months — fac­ing off with Bio­gen, No­var­tis

A ma­jor com­peti­tor to Bio­gen’s Spin­raza fran­chise could ar­rive as ear­ly as this com­ing May, as Roche and its biotech part­ner ob­tained a pri­or­i­ty re­view for ris­diplam.

FDA reg­u­la­tors will make a de­ci­sion on the spinal mus­cu­lar at­ro­phy drug by May 24, 2020, ac­cord­ing to PTC Ther­a­peu­tics, who is bag­ging $15 mil­lion in mile­stone pay­ment for the NDA fil­ing ac­cep­tance.

The move comes on the heels of topline da­ta from the Phase III SUN­FISH tri­al, in which ris­diplam met the pri­ma­ry end­point on change from base­line in the Mo­tor Func­tion Mea­sure 32 (MFM-32) scale af­ter one year of treat­ment, com­pared to place­bo. The re­sults con­firmed ear­li­er find­ings from part one of the study.

Levi Gar­raway

SUN­FISH en­rolled 180 pa­tients with type 2 or 3 SMA be­tween 2 and 25 years old. An ear­li­er, open la­bel tri­al dubbed FIRE­FISH cov­ered those with the type 1 vari­a­tion of the neu­ro­mus­cu­lar dis­ease.

“The FIRE­FISH and SUN­FISH tri­als were de­signed to rep­re­sent the re­al world spec­trum of peo­ple liv­ing with SMA and in­clude many peo­ple pre­vi­ous­ly un­der­rep­re­sent­ed in clin­i­cal tri­als,” not­ed Levi Gar­raway, Roche’s new­ly mint­ed head of glob­al prod­uct de­vel­op­ment, in a state­ment.

In PTC’s re­lease, CEO Stu­art Peltz added that the NDA sub­mis­sion in­cludes all three types of SMA pa­tients “demon­strat­ing im­prove­ments in mo­tor func­tions and de­vel­op­men­tal mile­stones, and a com­pelling safe­ty pro­file.”

Un­like Spin­raza, an an­ti­sense oligonu­cleotide ad­min­is­tered in­trathe­cal­ly every four months fol­low­ing ini­tial load­ing dos­es, ris­diplam is an oral med­ica­tion to be tak­en dai­ly. By mod­i­fy­ing how the SMN2 gene is spliced, the drug sup­pos­ed­ly in­creas­es func­tion­al SMN pro­tein lev­els in both the cen­tral ner­vous sys­tem and pe­riph­er­al tis­sues.

Stu­art Peltz

The drug is al­ready dec­o­rat­ed with the FDA’s or­phan and fast track des­ig­na­tions.

The fil­ing could lead to a broad­er la­bel than ex­pect­ed, ac­cord­ing to a note from Cred­it Su­isse an­a­lyst Evan Seiger­man, who’s pre­vi­ous­ly warned that ris­diplam spells an even big­ger threat to Bio­gen’s Spin­raza fran­chise than No­var­tis’ gene ther­a­py. Since gain­ing ap­proval in May, Zol­gens­ma has been plagued by re­im­burse­ment is­sues as pay­ers push back against its $2.1 mil­lion price tag (al­though No­var­tis stress­es that the pay­ment is de­liv­ered in a 5-year in­stall­ment plan for a sup­posed once-and-done cure). It’s al­so on­ly ap­proved for in­fants un­der the age of 2.

Al­though Spin­raza has earned Bio­gen more than $1.5 bil­lion in the first three quar­ters of 2019, Seiger­man said he is “in­creas­ing­ly con­cerned” about its dura­bil­i­ty. Hav­ing launched late 2016 (and ac­crued more than $4.1 bil­lion in record­ed sales so far), the fran­chise may be­gin to wane come H2 2020.

And Roche is go­ing all-in for the three-ri­val ri­val­ry. To­geth­er with PTC and the SMA Foun­da­tion, the phar­ma gi­ant is run­ning more clin­i­cal tri­als with pa­tients rang­ing from new­borns to 60 years old, in­clud­ing those pre­vi­ous­ly treat­ed with oth­er SMA ther­a­pies.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.