Can­cer spe­cial­ist Hi­ber­Cell snaps up an­a­lyt­ics, com­pu­ta­tion­al tools in all-stock buy­out; Prax­is re­veals new per­i­menopausal de­pres­sion da­ta

In a slight­ly un­con­ven­tion­al deal, New York-based Hi­ber­Cell is lever­ag­ing a chunk of its stock — still pri­vate­ly owned — to buy out Ge­nu­ity Sci­ence and its suite of tools for ge­nomics and mul­ti-om­ic an­a­lyt­ics, as well as an AI and ma­chine learn­ing plat­form.

Alan Rig­by

The goal, ac­cord­ing to CEO Alan Rig­by, is to fur­ther Hi­ber­Cell’s un­der­stand­ing of the adap­tive stress phe­no­type and its role in can­cer pro­gres­sion.

While his co-founder Julio Aguirre-Ghiso has elu­ci­dat­ed how such mech­a­nisms can be ma­nip­u­lat­ed to pre­vent of­ten fa­tal metasta­t­ic re­cur­rence, they are still look­ing for bet­ter ways to an­a­lyze clin­i­cal sam­ples, tease out bio­mark­ers and pre­dict which pa­tients would re­spond. It’s es­pe­cial­ly im­por­tant as Hi­ber­Cell’s drugs en­ter mid-stage clin­i­cal tests.

One thing that Ge­nu­ity’s tech­nol­o­gy will be able to do, for in­stance, is to “ex­haus­tive­ly com­pare pri­ma­ry tu­mor bi­ol­o­gy with paired metasta­t­ic le­sions to bet­ter iden­ti­fy the tar­gets and mech­a­nisms that dri­ve metasta­t­ic dis­ease.”

Hi­ber­Cell is al­so in­her­it­ing $100 mil­lion in cash, as well as part­ner­ships with Io­n­is and Ab­b­Vie from Ge­nu­ity, which will now be­come its sub­sidiary. In ad­di­tion, the top ex­ecs at Ge­nu­ity will take up C-suite posts at Hi­ber­Cell, with Tom Chit­ten­den as chief tech­nol­o­gy of­fi­cer, Jeff Gulcher as chief sci­en­tif­ic of­fi­cer of ge­nomics, and Hákon Guðb­jarts­son as chief in­for­mat­ics of­fi­cer. — Am­ber Tong

Prax­is re­veals new da­ta for per­i­menopausal de­pres­sion drug

Prax­is high­light­ed a small cut of Phase IIa study re­sults Mon­day morn­ing for their up and com­ing per­i­menopausal de­pres­sion pro­gram.

Mar­cio Souza

In the Part B por­tion of the tri­al, Prax­is said that six pa­tients treat­ed with a sin­gle, dai­ly 60 mg dose of PRAX-114 showed im­prove­ments in menopausal and mood symp­toms that were rapid, marked and main­tained through­out the 14-day treat­ment pe­ri­od. Pa­tients were giv­en the ex­per­i­men­tal drug in an out­pa­tient set­ting and Prax­is saw no new safe­ty sig­nals.

“The abil­i­ty to im­pact both menopausal and mood symp­toms in the PRAX-114 proof-of-con­cept study in PMD was our cri­te­ria to con­tin­ue de­vel­op­ment of this pro­gram,” CEO Mar­cio Souza said in a state­ment. “The rapid and con­sis­tent im­prove­ment across dis­tinct ef­fi­ca­cy mea­sures gives us con­fi­dence to ad­vance to a Phase IIb tri­al.”

Prax­is said that pa­tients saw an av­er­age de­crease from base­line at day 15 of 60% in fre­quen­cy of mod­er­ate-to-se­vere hot flash­es, and 68% in the to­tal score of a self-re­port­ed ques­tion­naire. Two weeks fol­low­ing the treat­ment pe­ri­od, scores trend­ed back to­ward the base­line, Prax­is said. — Max Gel­man

Free­line anoints new CEO fol­low­ing gene ther­a­py tri­al pause

There’s been a change at the top of Free­line Ther­a­peu­tics, six months af­ter the biotech halt­ed a gene ther­a­py tri­al.

Michael Pari­ni

Michael Pari­ni is the new CEO of Free­line, suc­ceed­ing There­sa Heg­gie, the com­pa­ny an­nounced Mon­day morn­ing. Pari­ni joined the com­pa­ny from Ver­tex in Feb­ru­ary as the new­ly cre­at­ed pres­i­dent and COO, just a week af­ter the biotech paused a piv­otal tri­al for two years.

“In a short pe­ri­od of time, [Pari­ni] has mo­bi­lized pro­gram ex­e­cu­tion across our pipeline, in­creased fo­cus on our core sci­en­tif­ic and plat­form tech­nol­o­gy ca­pa­bil­i­ties, and dri­ven ef­fi­cien­cies across the or­ga­ni­za­tion that have ex­tend­ed our cash run­way,” Free­line chair­man Chris Hol­lowood said in a state­ment.

Ear­li­er this year, Free­line hit the brakes on a piv­otal tri­al for the he­mo­phil­ia B pro­gram, dubbed FLT180a, af­ter re­ceiv­ing CMC feed­back from the FDA. The com­pa­ny is tar­get­ing a restart in mid-2023 and fil­ing for a BLA be­fore the end of 2024. A Phase I/II study for the pro­gram is still on track to read out da­ta by the end of this year. — Max Gel­man

Ex­elix­is adds op­tion for 20 new tar­gets in In­ven­ra col­lab

Ex­elix­is and In­ven­ra have ex­pand­ed on an old 2018 col­lab­o­ra­tion by up to 20 new tar­gets.

The com­pa­nies an­nounced Mon­day that Ex­elix­is will pay $15 mil­lion up­front in the re­vised deal, with mile­stones and roy­al­ties undis­closed. There is al­so fund­ing for the op­tion to nom­i­nate up to 20 ad­di­tion­al tar­gets in on­col­o­gy, and Ex­elix­is said it will own all an­ti­body se­quences dis­cov­ered from the col­lab­o­ra­tion in all dis­ease ar­eas.

“As Ex­elix­is seeks to build a dif­fer­en­ti­at­ed next-gen­er­a­tion pipeline in on­col­o­gy, we’re lever­ag­ing In­ven­ra’s ex­per­tise in an­ti­body and bis­pe­cif­ic dis­cov­ery to pro­vide key build­ing blocks for po­ten­tial fu­ture Ex­elix­is bi­o­log­ics, in­clud­ing an­ti­body-drug con­ju­gates,” Ex­elix­is CSO Pe­ter Lamb said in a state­ment.

All of the pro­grams un­der this col­lab­o­ra­tion have yet to move past the pre­clin­i­cal stage, and tar­gets re­main undis­closed. — Max Gel­man

Gas­tric can­cer CAR-T ther­a­py li­censed to Gra­cell

Two Chi­nese biotechs have reached a li­cense agree­ment to de­vel­op cell ther­a­py for sol­id tu­mors.

Gra­cell Biotech­nolo­gies and Fu­ture­Gen Bio­pharm will col­lab­o­rate to de­ploy Fu­ture­Gen’s Claudin 18.2 an­ti­bod­ies to make, man­u­fac­ture and com­mer­cial­ize a cell ther­a­py for pa­tients with gas­tric or gas­troe­sophageal junc­tion can­cer, pan­cre­at­ic can­cer and esophageal can­cer.

Gas­tric can­cer is one of the most di­ag­nosed can­cers in the world, the com­pa­nies said in a press re­lease, and there were over 1 mil­lion deaths in 2018 alone.

“Gra­cell has been mak­ing sig­nif­i­cant progress in de­vel­op­ing in­no­v­a­tive CAR-T ther­a­pies for sol­id tu­mors as well as hema­to­log­i­cal ma­lig­nan­cies,” Gra­cell CEO William (Wei) Cao said in a press re­lease. “This part­ner­ship with Fu­ture­Gen marks an­oth­er key mile­stone in our per­sis­tent ef­forts for treat­ing sol­id tu­mors. Mov­ing for­ward, we ex­pect to ex­plore more strate­gic al­liances to iden­ti­fy ad­di­tion­al tar­gets that max­i­mize the val­ue of our high­ly dif­fer­en­ti­at­ed tech­nol­o­gy plat­forms and even­tu­al­ly ben­e­fit can­cer pa­tients world­wide.”

Fu­ture­Gen will re­ceive pay­ment up­front, though that num­ber was not dis­closed. The com­pa­ny will be el­i­gi­ble for mile­stone pay­ments and roy­al­ties. — Josh Sul­li­van

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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