FDA ad­comm votes in fa­vor of new an­ti­fun­gal; De­nali gets $25M from Sanofi MS tri­al mile­stone

The FDA’s An­timi­cro­bial Drugs Ad­vi­so­ry Com­mit­tee vot­ed 14-1 in fa­vor of a lim­it­ed-use in­di­ca­tion for Cidara Ther­a­peu­tics’ reza­fun­gin, a treat­ment for can­didemia and in­va­sive can­didi­a­sis.

Ac­cord­ing to Cidara on Wednes­day, the vote was based on da­ta from two tri­als run by Cidara, with a once-week­ly dose of reza­fun­gin demon­strat­ing sta­tis­ti­cal non-in­fe­ri­or­i­ty ver­sus caspo­fun­gin, the cur­rent stan­dard.

“This pos­i­tive rec­om­men­da­tion is a sig­nif­i­cant step to­wards our goal of pro­vid­ing a once-week­ly treat­ment op­tion for pa­tients with in­va­sive Can­di­da in­fec­tions, for which no new drugs have been ap­proved in over a decade. We be­lieve reza­fun­gin if ap­proved, could pro­vide an ef­fec­tive new al­ter­na­tive for pa­tients bat­tling these po­ten­tial­ly dead­ly dis­eases,” said Jef­frey Stein, Cidara’s CEO in a state­ment.

Cidara’s NDA was ac­cept­ed and giv­en a pri­or­i­ty re­view last Sep­tem­ber and was as­signed a PDU­FA date for March 22, 2023. Al­so in 2022, Melin­ta Ther­a­peu­tics an­nounced that it ac­quired the rights to com­mer­cial­ize reza­fun­gin in the US, with Cidara hang­ing on to rights in Japan. Cidara li­censed the rights to Mundiphar­ma in oth­er re­gions. The EMA ac­cept­ed the mar­ket­ing au­tho­riza­tion for the drug last Au­gust and it is cur­rent­ly un­der re­view. — Tyler Patchen

Sanofi starts dos­ing mul­ti­ple scle­ro­sis in PhII tri­al

Sanofi has start­ed dos­ing in a Phase II tri­al of an RIPK1 in­hibitor for mul­ti­ple scle­ro­sis, trig­ger­ing a $25 mil­lion pay­ment to De­nali.

The two start­ed col­lab­o­rat­ing on De­nali’s pro­gram in 2018 when Sanofi paid $125 mil­lion up­front to part­ner on its RIPK1 pro­grams. The duo is go­ing af­ter neu­rode­gen­er­a­tive and in­flam­ma­to­ry dis­eases. How­ev­er, they tossed the ini­tial lead can­di­date for neu­rode­gen­er­a­tive dis­eases af­ter dis­ap­point­ing ear­ly-stage re­sults in Alzheimer’s dis­ease and ALS, pri­or­i­tiz­ing the cur­rent in­hibitor ​​SAR44382 in­stead.

As RIPK1 is a ki­nase that plays im­por­tant roles in reg­u­lat­ing in­flam­ma­tion and cell death, the hope is that in­hibit­ing it will tamp down in­flam­ma­tion in the brain and slow neu­rode­gen­er­a­tion.

“Fol­low­ing the ini­ti­a­tion of the Phase II HI­MALAYA study in ALS with SAR443820 last year by Sanofi, this Phase II study in mul­ti­ple scle­ro­sis marks an­oth­er im­por­tant mile­stone for our RIPK1 pro­gram,” De­nali CMO Ca­r­ole Ho said in a state­ment.

Sanofi and De­nali are al­so test­ing a sep­a­rate RIPK1 in­hibitor in cu­ta­neous lu­pus and ul­cer­a­tive col­i­tis. — Lei Lei Wu

Small Phar­ma un­cov­ers PhI­Ia re­sults for ma­jor de­pres­sive dis­or­der can­di­date

UK-based psy­che­delics biotech Small Phar­ma has lift­ed the veil on Phase IIa re­sults for a can­di­date to treat ma­jor de­pres­sive dis­or­der (MDD).

Small Phar­ma’s drug, dubbed SPL026, is an in­tra­venous treat­ment that us­es N, N-Di­methyl­trypt­a­mine, or DMT, with sup­port­ive ther­a­py to treat MDD. It hit the pri­ma­ry end­point, show­ing a “sta­t­i­cal­ly sig­nif­i­cant and clin­i­cal­ly rel­e­vant” re­duc­tion of de­pres­sive symp­toms two weeks af­ter the dose when put up against a place­bo. Pa­tients were dosed with 21.5 mg of the drug, giv­ing a 20- to 30-minute “psy­che­del­ic ex­pe­ri­ence,” the com­pa­ny said.

For the study’s pri­ma­ry end­point, pa­tients had a 7.4-point re­duc­tion on the Mont­gomery-As­berg de­pres­sion rat­ing scale (MADRS), com­pared to place­bo, net­ting a p-val­ue of p=0.02. The sec­ondary end­point of a “rapid on­set of an­ti­de­pres­sant ef­fect” one week af­ter dos­ing had a 10.8 re­duc­tion in the MADRS score against place­bo and a p-val­ue of p=0.002. Small Phar­ma al­so not­ed no ap­par­ent dif­fer­ence in the an­ti­de­pres­sant ef­fect be­ing ob­served be­tween a one and two-dose reg­i­men of the drug.

While no drug-re­lat­ed se­ri­ous ad­verse events or sui­ci­dal be­hav­iors were re­port­ed, 19 ad­verse events were re­port­ed in the treat­ment group and four in the place­bo group. Small Phar­ma stat­ed that ad­verse ef­fects were pos­si­bly re­lat­ed to be­ing treat­ed in the blind­ed phase. All ad­verse ef­fects were mild or mod­er­ate in sever­i­ty and most of the events were re­solved dur­ing the dos­ing vis­it.

Car­ol Rout­ledge, Small Phar­ma’s chief med­ical and sci­en­tif­ic of­fi­cer said in a state­ment that the Phase IIb study will seek to ex­plore the safe­ty and ef­fi­ca­cy of the drug in a wider MDD pop­u­la­tion.

“SPL026 with sup­port­ive ther­a­py was shown to have a sig­nif­i­cant an­ti­de­pres­sant ef­fect that was rapid and durable, with a re­mis­sion rate of 57% at three months fol­low­ing a sin­gle dose of SPL026. It was en­cour­ag­ing to see that SPL026 demon­strat­ed a fa­vor­able safe­ty and tol­er­a­bil­i­ty pro­file in MDD pa­tients in this study, con­sis­tent with our Phase I study,” Rout­ledge said in her state­ment. — Tyler Patchen

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.

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.

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.

Dutch biotech starts liq­ui­da­tion af­ter end­ing PhI­II in GVHD

A 13-year-old Dutch biotech is going through a liquidation process after an unexpected end to its Phase III trial testing whether its combination of two monoclonal antibodies was superior to Incyte’s Jakafi.

Xenikos had hoped to prove its investigational therapy, named T-Guard, was better than Jakafi at garnering a complete response in patients experiencing life-threatening complications in which new cells from a hematopoietic stem cell transplant begin to fight the body. Jakafi was approved for the indication, steroid-refractory acute graft-versus-host disease, in May 2019.

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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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