Rob Koremans, Recordati CEO

Recor­dati ac­quires EU­SA Phar­ma and its 4 rare dis­ease drugs, but an­a­lysts ques­tion strat­e­gy be­hind the deal

An Ital­ian phar­ma com­pa­ny en­gi­neered a near­ly $850 mil­lion buy­out last week look­ing to ex­pand its rare dis­ease port­fo­lio and fur­ther soak up parts of the US mar­ket, but ques­tions re­main over the drugs in­volved and when they could see pa­tient up­take state­side.

Recor­dati shelled out the cash to ac­quire EU­SA Phar­ma and its port­fo­lio of four rare dis­ease drugs, the com­pa­nies an­nounced Fri­day, al­low­ing Recor­dati to po­si­tion it­self — by its own ac­count — for growth in the US. EU­SA’s top can­di­date for ex­pan­sion is os­ten­si­bly the neu­rob­las­toma drug Qarz­i­ba, ap­proved in the EU for high-risk, new­ly-di­ag­nosed in­di­vid­u­als.

Dur­ing an in­vestor call, Recor­dati ex­ecs tout­ed a US ap­proval for Qarz­i­ba as a pos­si­ble key val­ue dri­ver in this deal. But when a Cred­it Su­isse an­a­lyst point­ed out EU­SA pre­vi­ous­ly stat­ed in press re­leas­es it ex­pect­ed to file Qarz­i­ba’s US ap­proval in 2017, an OK still hadn’t come through and there don’t ap­pear to be any on­go­ing clin­i­cal tri­als, the com­pa­ny had few an­swers for when it would ap­proach the FDA.

Chair­man An­drea Recor­dati large­ly de­murred, say­ing he couldn’t give any de­fin­i­tive time­lines be­cause the deal isn’t ex­pect­ed to close un­til next year.

“We’re still in be­tween the sign­ing and clos­ing han­dling sit­u­a­tions, so we need to dis­cuss this, clos­ing EU­SA, and it’s not re­al­ly ap­pro­pri­ate to give them more de­tails at this stage,” Recor­dati said on the call.

He went on to say while Qarz­i­ba is ex­pect­ed to be an “im­por­tant” part of the com­pa­ny’s plans, Recor­dati is al­so look­ing at the rare lymph node dis­or­der drug Syl­vant as an­oth­er growth dri­ver, cur­rent­ly ap­proved in the US and EU.

The oth­er two drugs in the deal are Fo­tiv­da, a monother­a­py TKI for re­nal cell car­ci­no­ma ap­proved in the EU, and Caphosol, a med­ical de­vice used to treat mouth swelling af­ter chemother­a­py. Qarz­i­ba was al­so ap­proved by Chi­nese reg­u­la­tors in Au­gust, with EU­SA’s part­ner BeiGene, and Syl­vant al­so won a Chi­na ap­proval Fri­day.

EU­SA al­so has 200 em­ploy­ees and it re­mained un­clear on the call whether Recor­dati would be bring­ing all of them on board. Fresh­ly mint­ed CEO Rob Ko­re­mans, re­spond­ing to an­oth­er ques­tion from the Cred­it Su­isse an­a­lyst, not­ed Recor­dati does not have “very spe­cif­ic deep ther­a­peu­tic know-how in rare dis­ease on­col­o­gy” and that EU­SA’s R&D team is a good fit for the com­pa­ny.

“Our ob­jec­tive is to re­tain the know-how and the or­ga­ni­za­tion,” Ko­re­mans said. “This is not a syn­er­gist dri­ven deal. This is a growth dri­ven deal. And al­so, ob­vi­ous­ly, a di­ver­si­fi­ca­tion deal, be­cause it al­lows us to en­ter a new and very at­trac­tive, un­der­served ther­a­peu­tic area where we don’t have the as­set ex­per­tise.”

Over­all, Recor­dati ex­pects the port­fo­lio ad­di­tions to bring in about $170 mil­lion in sales by 2023, with po­ten­tial peak sales of just over $282 mil­lion — though the com­pa­ny again de­clined to elab­o­rate on the ex­act time­line.

Out­side Fri­day’s ac­qui­si­tion, Recor­dati al­so owns a sub­sidiary that fo­cus­es pri­mar­i­ly on rare dis­eases. One of these drugs, Carbaglu, treats rare cas­es of N-acetyl­glu­ta­mate syn­thase de­fi­cien­cy and fre­quent­ly ap­pears near the top of the world’s most ex­pen­sive drugs list. In 2017, the drug cost more than $585,000.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.