As head-to-head with Alex­ion heats up, No­var­tis flash­es da­ta for next wave of tri­als for star kid­ney mol­e­cule

No­var­tis has rarely in­vest­ed much in kid­ney drugs, but over the last cou­ple of years, they’ve tried to build a pipeline out of a sin­gle drug.

On Sun­day, that drug, a small mol­e­cule called ip­ta­co­pan, de­liv­ered an­oth­er suc­cess. No­var­tis an­nounced that it cleared a Phase II tri­al for the rare kid­ney dis­ease IgA nephropa­thy, sig­nif­i­cant­ly re­duc­ing a risk fac­tor for kid­ney health. The da­ta bur­nish the Swiss phar­ma’s con­fi­dence in a piv­otal Phase III study al­ready un­der­way in the con­di­tion, along with the mid- or late-stage stud­ies they’ve launched in a hand­ful of un­com­mon re­nal af­flic­tions.

Col­lec­tive­ly, they add a sec­ondary set of mar­kets for the drug, even if it proves un­suc­cess­ful in un­seat­ing Alex­ion in PNH, where the drug re­cent­ly won an FDA break­through des­ig­na­tion.

John Tsai

“Com­ple­ment-dri­ven re­nal dis­eases, such as IgAN, are dev­as­tat­ing and most­ly af­fect young adults, im­pos­ing a high dis­ease bur­den,” No­var­tis CMO John Tsai said in a state­ment. “These new da­ta in IgAN add to the grow­ing body of ev­i­dence around the po­ten­tial of ip­ta­co­pan to tar­get a key dri­ver in these rare re­nal dis­eases.”

In the 112-per­son study, IgA nephropa­thy pa­tients were ran­dom­ized to re­ceive place­bo or var­i­ous dos­es of ip­ta­co­pan. Pa­tients who re­ceived the high­est dose — the one they brought for­ward in­to Phase III — saw a 23% re­duc­tion in pro­tein­uria, or the amount of pro­tein, pa­tients re­leased in urine. A cor­re­late for how well the kid­neys are fil­ter­ing, the FDA has re­cent­ly ac­cept­ed it as a sur­ro­gate end­point for ac­cel­er­at­ed ap­proval.

No­var­tis said the drug al­so showed a “trend to­ward sta­bi­liza­tion” in glomeru­lar fil­tra­tion rate, a more es­tab­lished mea­sure for kid­ney func­tion, but de­clined to dis­close the ac­tu­al da­ta in their press re­lease. The two mea­sures are co-pri­ma­ry end­points on the Phase III stud­ies.

IgA nephropa­thy has no ap­proved treat­ments, but as the FDA has up­dat­ed its re­quire­ments for ap­prov­ing new kid­ney drugs and phar­ma and VC cash has poured in­to the space, the hunt for one has grown hot­ly com­pet­i­tive. Swedish biotech Cal­lid­i­tas snared a $90 mil­lion IPO last year most­ly on the promise of its Phase III steroid for the dis­ease. In Jan­u­ary, GV-backed Tru­code Gene Re­pair re­vealed they de­cid­ed to abrupt­ly scrap their en­tire gene edit­ing plat­form and sick­le cell dis­ease fo­cus on the hope that they could turn an old Mer­ck KGaA drug in­to the first treat­ment for the dis­ease. (They re­brand­ed as Ve­ra and went for a mod­est IPO.)

Like the oth­er dis­eases No­var­tis is fir­ing ip­ta­co­pan at, IgA nephropa­thy is dri­ven by a dys­func­tion­al im­mune sys­tem, in par­tic­u­lar prob­lems with a cas­cade called the com­ple­ment sys­tem. The most ad­vanced pro­gram is for PNH, the most well-known com­ple­ment dis­or­der, but one where No­var­tis faces stiff com­pe­ti­tion in es­tab­lished gi­ant Alex­ion and a new­com­er in Apel­lis, whose drug Em­paveli won ap­proval last month.

The com­pa­ny will have a clear­er path to com­mer­cial dom­i­nance in kid­ney dis­eases, where few, if any, oth­er op­tions are avail­able. On Mon­day, the com­pa­ny an­nounced up­dat­ed da­ta from their Phase II tri­al for CG nephropa­thy, al­so known as C3G, show­ing im­proved kid­ney func­tion from pa­tients on the drug. A Phase III tri­al will start lat­er this year.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Saqib Islam, SpringWorks CEO

Pfiz­er spin­out Spring­Works will ship its first drug to the FDA be­fore year’s end with PhI­II win

SpringWorks Therapeutics thinks it has cemented the backbone for its first “pipeline-in-a-product” oncology treatment and will send it to the FDA before the clock strikes 2023 with a Phase III win on Tuesday.

The oral gamma secretase inhibitor, dubbed nirogacestat, beat placebo on the primary goal of progression-free survival in adults with progressing desmoid tumors.

The soft-tissue tumors can lead to long-lasting pain, disfigurement and amputation, and there are currently no approved meds for the rare oncology indication. The tumors typically impact patients aged 20 to 44 years old and disproportionately affect women at rates 2 to 3 times higher, with up to a total of 1,650 new cases diagnosed in the US annually, according to SpringWorks.

Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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