Io­n­is and As­traZeneca are hop­ing its lat­est re­sults net an FDA ap­proval.

The two com­pa­nies pre­sent­ed their lat­est Phase III re­sults from a 35-week analy­sis of the Phase III tri­al, dubbed NEU­RO-TTRans­form, of As­traZeneca’s eplon­ter­sen in pa­tients with the rare dis­ease known as hered­i­tary transthyretin-me­di­at­ed amy­loid polyneu­ropa­thy (AT­TRv-PN).

Ac­cord­ing to Io­n­is on Wednes­day, Phase III had a pos­i­tive out­look as eplon­ter­sen achieved an 81.2% re­duc­tion of serum transthyretin (TTR) con­cen­tra­tion in pa­tients com­pared to base­line, demon­strat­ing re­duced TTR pro­tein pro­duc­tion. This hit the co-pri­ma­ry end­point of the tri­al and earned a p-val­ue of p=<0.0001.

Eplon­ter­sen al­so showed a treat­ment ef­fect on the oth­er pri­ma­ry end­point, net­ting a mod­i­fied Neu­ropa­thy Im­pair­ment Score of +7 (mNIS+7). This is used to de­ter­mine neu­ro­path­ic dis­ease pro­gres­sion. This co-pri­ma­ry end­point al­so re­ceived a p-val­ue of p=<0.0001.

Ac­cord­ing to Io­n­is, the study al­so hit the sec­ondary end­point, which was a change from base­line in the Nor­folk Qual­i­ty of Life Ques­tion­naire-Di­a­bet­ic Neu­ropa­thy.

“The promis­ing re­sults from NEU­RO-TTRans­form show that eplon­ter­sen had a pos­i­tive im­pact on dis­ease pro­gres­sion and im­proved qual­i­ty of life in a sub­stan­tial num­ber of pa­tients,” Io­n­is chief clin­i­cal de­vel­op­ment of­fi­cer Eu­gene Schnei­der said in a state­ment.

As a re­sult of the da­ta, Io­n­is and As­traZeneca will look to get reg­u­la­to­ry ap­proval for eplon­ter­sen to treat AT­TRv-PN and are plan­ning to file an NDA lat­er this year.

The drug is be­ing joint­ly de­vel­oped and com­mer­cial­ized by both com­pa­nies in the US and will be de­vel­oped and com­mer­cial­ized in the rest of the world by As­traZeneca.

That agree­ment, which kicked off last year, saw Io­n­is get a $200 mil­lion up­front pay­ment, along with re­ceiv­ing up to $485 mil­lion in de­vel­op­ment and ap­proval mile­stones, as well as up to $2.9 bil­lion in sales-re­lat­ed mile­stone pay­ments. — Tyler Patchen

Glaukos churns out pos­i­tive Phase III da­ta in eye tri­als

Eye dis­ease biotech Glaukos is tout­ing new Phase III da­ta — and with two tri­als meet­ing pri­ma­ry end­points, the biotech is want­i­ng to go get the FDA to sign off on its prod­uct.

The com­pa­ny re­vealed Phase III da­ta Wednes­day morn­ing from two piv­otal tri­als, test­ing iDose TR, an im­plant with a for­mu­la­tion of travo­prost, a med­ica­tion used to treat oc­u­lar pres­sure. The da­ta, in short, showed that mul­ti­ple ver­sions of iDose TR reached the pri­ma­ry end­point of non-in­fe­ri­or­i­ty to the com­para­tor arm at three months in.

In terms of re­duc­tions in in­traoc­u­lar pres­sure (al­so called IOP), the re­duc­tion from base­line over the first three months was 6.6-8.5 mmHg (mil­lime­ters of mer­cury, a type of pres­sure mea­sure­ment) in the slow-re­lease arm in one tri­al, ver­sus 6.6-7.7 mmHg in the con­trol arm. In an­oth­er tri­al, IOP re­duc­tions from base­line over the first three months were 6.7-8.4 mmHg in the slow-re­lease arm, ver­sus 6.8-7.2 mmHg in con­trol.

Shares of $GKOS shot up 15% in ear­ly morn­ing trad­ing.

With 98% of pa­tients con­tin­u­ing at 12 months, plus no ad­verse events of corneal en­dothe­lial cell loss, no se­ri­ous corneal ad­verse events and no ad­verse events of pe­ri­or­bital fat at­ro­phy, the biotech plans to sub­mit an NDA — and ex­pects an FDA re­view and de­ci­sion by the end of 2023. — Paul Schloess­er

Carmell says it’s filed for an IPO

Pitts­burgh-based Carmell Ther­a­peu­tics said it con­fi­den­tial­ly filed its draft pa­pers with the SEC en route to a pub­lic list­ing.

The SEC first has to com­plete the re­view process be­fore a list­ing can take place.

The biotech, which claims to be on the re­gen­er­a­tive med­i­cine track, is work­ing on so-called growth fac­tors in “al­lo­gene­ic platelet-en­riched plas­ma” to heal bone, skin and oth­er tis­sues. The goal is to ramp up the re­growth of hair and spark col­la­gen pro­duc­tion af­ter an in­jury, dis­ease or the much-talked-about ag­ing.

Carmell eyes Phase II stud­ies in tib­ia frac­ture heal­ing, for which it has fast track des­ig­na­tion from the FDA, in the sec­ond half of 2023, at which time the biotech al­so hopes to be­gin a mid-stage tri­al in foot/an­kle fu­sion. Pre­clin­i­cal work is be­ing con­duct­ed in spinal fu­sion, den­tal bone graft sub­sti­tute, an­dro­ge­net­ic alope­cia and chron­ic wound heal­ing, ac­cord­ing to Carmell’s pipeline page. The com­pa­ny’s first clin­i­cal tri­al — HEAL I — was con­duct­ed in South Africa.

Found­ed in 2008 by sci­en­tists at Carnegie Mel­lon Uni­ver­si­ty, with the help of Pitts­burgh Life Sci­ences Green­house, Carmell is led by for­mer med­ical de­vice lead­ers from John­son & John­son and its sub­sidiaries. — Kyle LaHu­cik

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Eisai will eliminate 91 after it out-licensed a seizure drug.

An Eisai spokesperson told Endpoints News that the change-up is tied to Fycompa, a seizure treatment that Florida rare disease biotech Catalyst Pharmaceuticals agreed to pay $160 million to Eisai in exchange for commercial rights back in December. The job cuts were originally flagged in a New Jersey state WARN notice.

The spokesperson said that Catalyst indicated interest in retaining up to 40 employees who work on Fycompa. Those who qualify will have an opportunity to interview with Catalyst.

Sanofi expects its RSV antibody jointly developed with AstraZeneca will be available next season, executive VP of vaccines Thomas Triomphe announced on the company’s quarterly call.

Beyfortus, also known as nirsevimab, was approved in the EU back in November and is currently under FDA review with an expected decision coming in the third quarter of this year. The news comes as the FDA plans to hold advisory committee meetings over the next couple months to review RSV vaccines from Pfizer and GSK.