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

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Lian­Bio an­nounces terms for IPO next week; NIH and White House of­fi­cials dis­cuss hy­po­thet­i­cal bio-med re­search agency

LianBio, a biotech that has operations in both the US and China, announced the terms yesterday for its initial public offering.

The biotech plans to raise $325 million by offering 20.3 million shares priced between $15 and $17.

At the midpoint of the proposed range, LianBio could command a fully diluted market value of $1.8 billion, based on a number of expected outstanding shares.

The two year old biotech has focused on in-licensing and commercialization of therapeutics in China, Hong Kong, Taiwan, Macau and other Asian markets. The company currently has nine programs across five therapeutic areas, including oncology, cardiovascular, and inflammatory diseases.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.

David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.