Brett Monia, Ionis Pharmaceuticals CEO

Io­n­is hands off an an­ti­sense drug for kid­ney dis­ease, giv­ing all rights to Roche

Io­n­is is hand­ing Roche a drug on the cusp of Phase III.

Io­n­is an­nounced ear­ly Mon­day that Roche is in-li­cens­ing and ad­vanc­ing the an­ti­sense drug can­di­date known as IO­N­IS-FB-LRx in­to a Phase III clin­i­cal study. The drug is be­ing in­ves­ti­gat­ed for im­munoglob­u­lin A nephropa­thy (IgAN), a kid­ney dis­ease that can lead to re­nal fail­ure. The dis­ease is marked by im­munoglob­u­lin A buildup in the kid­neys, re­sult­ing in in­flam­ma­tion and ul­ti­mate­ly kid­ney fail­ure if left un­treat­ed.

Roche and Io­n­is have been part­ners for some time now af­ter the Big Phar­ma shelled out $75 mil­lion to li­cense the drug can­di­date in 2018, af­ter reach­ing a pre­vi­ous deal on a Hunt­ing­ton’s pro­gram. Roche on­ly got its hands on the drug af­ter GSK, Io­n­is’ ear­li­er part­ner on IO­N­IS-FB-LRx, dumped its op­tion in a move to re­vamp its R&D pipeline af­ter Em­ma Walm­s­ley be­came CEO in 2017.

Io­n­is orig­i­nal­ly had test­ed the can­di­date in dry age-re­lat­ed mac­u­lar de­gen­er­a­tion, or AMD. How­ev­er, the com­pa­nies left the op­tion for Roche to li­cense the drug al­to­geth­er in 2018 af­ter Io­n­is did some more work in dry AMD and an at-the-time un­named rare, se­vere re­nal in­di­ca­tion.

Roche de­cid­ed to ad­vance the pro­gram af­ter a pos­i­tive Phase II read­out, where the drug met the pri­ma­ry end­point of “change in 24-hour uri­nary pro­tein at 29 weeks com­pared to base­line.” Those Phase II study da­ta have been sub­mit­ted for pre­sen­ta­tion at an un­named med­ical con­fer­ence.

Io­n­is not­ed, in a re­lease, that Roche will lead and be re­spon­si­ble for the Phase III study, on top of fu­ture glob­al de­vel­op­ment, reg­u­la­to­ry and com­mer­cial­iza­tion ac­tiv­i­ties.

A Roche spokesper­son told End­points News that “Roche’s col­lab­o­ra­tion with Io­n­is will help to max­i­mize our chances of suc­cess for de­vel­op­ing ther­a­pies in two dev­as­tat­ing con­di­tions, where on­ly few op­tions are avail­able.”

As for Roche’s next steps, the Phase III study will aim to con­firm the ben­e­fit seen in the ear­li­er tri­al. On top of that, Io­n­is is con­duct­ing a Phase II study in ge­o­graph­ic at­ro­phy due to late-stage age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD). Roche added that if the out­come of the study is pos­i­tive, it will look at po­ten­tial­ly de­vel­op­ing the drug for that in­di­ca­tion.

Ac­cord­ing to Clin­i­cal­Tri­, the Phase II study in ques­tion on ge­o­graph­ic at­ro­phy is sched­uled to fin­ish some­time in Oc­to­ber this year. Io­n­is re­ceived $55 mil­lion from Roche for li­cens­ing IO­N­IS-FB-LRx for IgAN and achiev­ing a yet-un­spec­i­fied de­vel­op­ment mile­stone in that afore­men­tioned Phase II study.

Io­n­is and Roche have not been the on­ly play­ers in the IgAN space. An­gion Bio­med­ica scrapped a Phase II tri­al look­ing at its oral TKI in­hibitor in pa­tients with IgAN and fo­cal seg­men­tal glomeru­loscle­ro­sis (FS­GS) af­ter an un­ex­pect­ed safe­ty event just two weeks ago. Al­ny­lam, in col­lab­o­ra­tion with Re­gen­eron af­ter ink­ing a block­buster R&D deal in 2019, re­cent­ly tout­ed da­ta on in­ves­ti­ga­tion­al RNAi drug cem­disir­an from a Phase II de­scrip­tive tri­al — with no sta­tis­ti­cal test­ing con­duct­ed.

Io­n­is did not re­spond to a re­quest for com­ment from End­points be­fore pub­li­ca­tion, but this sto­ry will be up­dat­ed when we hear back.

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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No­var­tis' ap­proved sick­le cell dis­ease drug fails to beat place­bo in PhI­II

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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FDA ap­proves an­oth­er in­di­ca­tion for Keytru­da, this time in the ad­ju­vant NSCLC set­ting

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

Alon Seri-Levy, Sol-Gel Technologies CEO

Bridge­Bio com­pa­ny sells off rare dis­ease can­di­date to Gal­der­ma part­ner

Israeli biotech Sol-Gel Technologies announced Friday that it got its hands on a rare disease drug candidate from PellePharm for almost $75 million, amid claims that the drug has the potential to reach a $300 million market.

Execs said on a conference call Friday morning that patidegib, a hedgehog signaling pathway blocker, is being investigated to treat Gorlin syndrome, a rare genetic disorder that increases the risk of developing certain kinds of cancer such as basal cell skin cancer and medulloblastoma, a type of brain cancer. The disease affects around one in every 31,000 people, and an estimated 70,000 people worldwide.

Ying Huang, Legend CEO

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.