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

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

San Francisco-based biotech 89bio announced on Friday that it expects to rake in $275 million on a stock offering. The raise comes after 89bio announced on Tuesday results of a Phase II study showing that its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH.

To run a Phase III study, 89bio CEO Rohan Palekar told Endpoints News that the biotech “would need to raise additional capital.” 89bio offered over 16 million shares of its common stock at $16.25 per share, and expects the offering closes on March 28.