David Arthur, Salarius Pharmaceuticals CEO

Salarius Phar­ma­ceu­ti­cals sees with­drawals, 3 of 13 pa­tient re­spon­ders in sar­co­ma tri­al

The Hous­ton-based biotech Salarius Phar­ma­ceu­ti­cals is lift­ing the cov­er on da­ta from a Phase I/II tri­al for a drug cur­rent­ly on vol­un­tary hold af­ter a pa­tient death, and the re­sults ap­pear to have un­der­whelmed in­vestors.

Salarius’ can­di­date, dubbed se­clidem­stat, is an oral LSD1 in­hibitor that is meant to treat Ew­ing sar­co­ma and FET-re­arranged sar­co­mas in pa­tients un­der 12 years old. The biotech had pre­sent­ed da­ta with 13 pa­tients with “first- and sec­ond-re­lapse Ew­ing sar­co­ma” who were treat­ed in com­bi­na­tion with topote­can and cy­clophos­phamide.

In the study, re­searchers saw one com­plete re­sponse and two par­tial re­spons­es out of 13 to­tal pa­tients. Ex­ecs at­tempt­ed to paint a more pos­i­tive pic­ture in high­light­ing an ad­di­tion­al three pa­tients with “sta­ble dis­ease,” though that is not gen­er­al­ly some­thing that con­cerns the FDA when it comes to ap­provals.

Salarius al­so re­port­ed sev­er­al with­drawals from the tri­al, as two pa­tients bowed out af­ter just three months, in­clud­ing all three con­firmed re­spon­ders. The com­pa­ny did note that two of the re­spon­ders came from five pa­tients who had re­lapsed for the first time.

The da­ta re­veal comes af­ter the com­pa­ny paused en­roll­ment and a pa­tient death was re­port­ed in Oc­to­ber. Salarius re­port­ed that the pause was due to a “sus­pect­ed un­ex­pect­ed se­ri­ous ad­verse re­ac­tion,” known as a SUSAR, that was ob­served in the sar­co­ma tri­al. The FDA has agreed with the vol­un­tary hold and is work­ing with the com­pa­ny to an­a­lyze the avail­able da­ta to fig­ure out how best to pro­ceed.

De­spite the da­ta, Salarius is ap­par­ent­ly still look­ing to forge ahead, ac­cord­ing to se­nior man­age­ment. “These in­ter­im re­sults are en­cour­ag­ing, and I be­lieve they show the po­ten­tial for se­clidem­stat to pro­vide a more durable re­sponse among Ew­ing sar­co­ma pa­tients when used in com­bi­na­tion with topote­can and cy­clophos­phamide,” Salarius CEO David Arthur said in a state­ment.

And the in­ter­im re­sults have not ex­act­ly been a boon for the com­pa­ny’s in­vestors as well. The stock $SLRX is cur­rent­ly down around 5% since open­ing on Fri­day and is down 86% since the start of the year, now sit­ting at an es­ti­mat­ed $1.80 per share.

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.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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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.

Tony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Kid­ney dis­ease drug­mak­er Goldfinch Bio shuts down

Goldfinch Bio, attempting to make treatments for kidney diseases and diabetic nephropathy, is shutting down.

President and CEO Tony Johnson confirmed to Endpoints News Friday afternoon that the biotech shut down after “fundraising challenges in the current macro-environment.” Fierce Biotech first reported the news.

Johnson, who joined in 2017 after a stint as SVP of early clinical development at AstraZeneca, said in a text that the company “entered the ABC process recently,” referring to an assignment for the benefit of the creditors, which provides a different wind-down avenue than a bankruptcy.

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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.

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.