Af­ter BioCryst fiz­zle, a new oral HAE play­er emerges

A new play­er in the search for an oral hered­i­tary an­gioede­ma pill has re­ceived a ma­jor boost and show of con­fi­dence.

Phar­varis an­nounced $66 mil­lion in Se­ries B fi­nanc­ing for its B2 re­cep­tor an­tag­o­nist pill, near­ly tripling the fund­ing At­tune Phar­ma­ceu­ti­cals re­cent­ly re­ceived in Se­ries B for its own hered­i­tary an­gioede­ma (HAE) pill. The mon­ey will fu­el the com­pa­ny’s Phase I tri­al on its lead com­pound, PHA121, on healthy vol­un­teers. The first sub­jects have al­ready been dosed.

The news comes three-and-a-half months af­ter BioCryst’s great an­gioede­ma fiz­zle. Al­though the Durham out­fit’s oral drug BCX7353 tech­ni­cal­ly passed a Phase III tri­al, the drug failed to cut the at­tack rate by at least 50%, the cut­off at which Wall Street an­a­lysts con­sid­ered the drug mar­ketable, and the biotech’s stock tum­bled.

Hered­i­tary an­gioede­ma, a po­ten­tial­ly life-threat­en­ing ge­net­ic dis­or­der, af­fects 1 in every 50,000 peo­ple. It is char­ac­ter­ized by episodes of ex­treme swelling across the body. It can cause vom­it­ing and nau­sea when man­i­fest in the gut and can even be fa­tal if it ap­pears in the air­ways and re­stricts breath­ing.

Sev­er­al ther­a­pies and in­jec­tions ex­ist to curb the at­tacks, but last year Shire’s Takhzy­ro (lanadelum­ab) emerged as one of the hottest and most ef­fec­tive drugs, con­tribut­ing to an 87% re­duc­tion in mean HAE at­tack fre­quen­cy in a Phase III tri­al (ver­sus a 0% re­duc­tion in the place­bo group) that ush­ered it to­ward mar­ket. The drug is on track to be­come a block­buster. Clair­voy­ant An­a­lyt­ics projects $1.15 bil­lion in sales by 2022.

But be­cause Takhzy­ro is an in­jec­tion, the search is still on for an oral med­ica­tion that can curb the dis­or­der at sim­i­lar rates. BioCryst con­tin­ues to de­vel­op its oral drug, while At­tune Phar­ma­ceu­ti­cals re­mains in the ear­ly stage.

HAE is caused by low lev­els of or dys­func­tion­ing C1 es­terase in­hibitor pro­tein. Most avail­able ther­a­pies work by in­hibit­ing C1 es­terase, ful­fill­ing the func­tion of the miss­ing pro­tein. But Phar­varis’ Ph121 works like the FDA-ap­proved icat­i­bant in­jec­tion, which func­tions as an an­tag­o­nist of bradykinin B2 re­cep­tors – i.e. it at­tacks the mol­e­cules that di­rect­ly cause the swelling. The founders of Phar­varis in­clude an in­ven­tor of icat­i­bant and a for­mer mem­ber of the lead­er­ship at Jeri­ni AG, the bio­phar­ma­ceu­ti­cal that sells the in­jec­tion.

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.

Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

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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