Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Ne­glect­ed Dis­eases ini­tia­tive (DN­Di) and Sanofi to­day said that their po­ten­tial sleep­ing sick­ness treat­ment saw suc­cess rates of up to 95% from a Phase II/III study in­ves­ti­gat­ing the safe­ty and ef­fi­ca­cy of sin­gle-dose aco­z­i­bo­role.

The po­ten­tial­ly trans­for­ma­tive treat­ment for sleep­ing sick­ness would main­ly be tar­get­ed at African coun­tries, ac­cord­ing to da­ta pub­lished to­day in The Lancet In­fec­tious Dis­eases med­ical jour­nal. The clin­i­cal tri­al was led by DN­Di and its part­ners in the De­mo­c­ra­t­ic Re­pub­lic of the Con­go (DRC) and Guinea, with the au­thors not­ing:

Giv­en the high ef­fi­ca­cy and good safe­ty at all stages of dis­ease, aco­z­i­bo­role elim­i­nates the need for rou­tine lum­bar punc­ture at di­ag­no­sis and dur­ing fol­low-up, which re­quires trained staff, is as­so­ci­at­ed with com­pli­ca­tions, and is a source of anx­i­ety for pa­tients. These ad­van­tages and the fact that a sin­gle oral dose would be more ac­ces­si­ble to pa­tients liv­ing in re­mote ar­eas with­out easy ac­cess to health care, means that aco­z­i­bo­role holds promise in the ef­forts to reach the WHO goal of in­ter­rupt­ing HAT [Hu­man African try­panoso­mi­a­sis] trans­mis­sion by 2030.

Trans­mit­ted by the tsetse fly, HAT or sleep­ing sick­ness, is a ne­glect­ed trop­i­cal dis­ease that is most­ly fa­tal when left un­treat­ed, and re­mains en­dem­ic in sub-Sa­ha­ran Africa, main­ly in the De­mo­c­ra­t­ic Re­pub­lic of the Con­go. The num­ber of re­port­ed cas­es of the ill­ness has fall­en steeply over the last two decades, from al­most 40,000 re­port­ed cas­es in 1998 (with es­ti­mates of over 300,000 un­di­ag­nosed cas­es) to less than 1,000 in 2020.

An­toine Tar­ral

“By sim­pli­fy­ing the treat­ment par­a­digm, aco­z­i­bo­role would be an in­no­va­tion that en­ables a sus­tain­able re­sponse to sleep­ing sick­ness for health sys­tems. With these new da­ta, we have hope that we may be able to fi­nal­ly elim­i­nate the dis­ease, once and for all, by open­ing the door to a ‘screen-and-treat’ ap­proach at the vil­lage lev­el,” An­toine Tar­ral, head of the sleep­ing sick­ness pro­gram at DN­Di and co-au­thor of the Lancet pa­per, said in a state­ment.

In Ju­ly 2021, the FDA ap­proved the first oral treat­ment for sleep­ing sick­ness (hu­man African try­panoso­mi­a­sis) from Sanofi and DN­Di, and pro­vid­ed the part­ners with a pri­or­i­ty re­view vouch­er that they may end up sell­ing for about $100 mil­lion.

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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