Roche's PD-L1 fran­chise play­er nabs pri­or­i­ty re­view; One of world's first psy­che­delics re­search in­sti­tutes opens in Aus­tralia

The FDA has just put Genen­tech’s lat­est shot at adding block­buster sales for Tecen­triq on the reg­u­la­tor’s in­side track.

Roche said Tues­day that the agency has pro­vid­ed pri­or­i­ty re­view sta­tus for a sup­ple­men­tal NDA to get the big PD-L1 fran­chise play­er ap­proved for ad­ju­vant treat­ment fol­low­ing surgery and plat­inum-based chemother­a­py for peo­ple with non-small cell lung can­cer.

Genen­tech re­searchers came up with the first ev­i­dence of dis­ease-free sur­vival for the drug fol­low­ing surgery and plat­inum-based chemother­a­py. The drug re­duced the risk of dis­ease re­cur­rence or death by 34%, mak­ing it a like­ly win­ner even be­fore the Dec. 1 PDU­FA date.

Pe­ter Welford at Jef­feries has been an en­thu­si­as­tic sup­port­er of the de­vel­op­ment team, hold­ing out the prospect of ad­di­tion­al sales in this set­ting of $1 bil­lion-plus. He’s al­so not­ed, though, that physi­cians may still prove some­what re­luc­tant to use it with­out more da­ta. — John Car­roll

One of world’s first psy­che­delics re­search in­sti­tutes opens in Aus­tralia

What’s be­ing billed as the world’s first glob­al re­search in­sti­tute for psy­che­delics is launch­ing in Mel­bourne, Aus­tralia.

With at least $29.5 mil­lion in fund­ing over the next five years, the Psy­chae In­sti­tute will link lead­ing re­searchers from sev­er­al or­ga­ni­za­tions around the world, in­clud­ing King’s Col­lege Lon­don, the Uni­ver­si­ty of Toron­to, the Uni­ver­si­ty of Zurich, and the Fed­er­al Uni­ver­si­ty of Rio Grande in Brazil.

They will be joined by Aus­tralian in­sti­tu­tions in­clud­ing Swin­burne Uni­ver­si­ty, the Uni­ver­si­ty of Mel­bourne, Flo­rey In­sti­tute of Neu­ro­science and Men­tal Health, Agri­cul­ture Vic­to­ria Re­search and NICM Health Re­search In­sti­tute (Syd­ney).

The non-prof­it in­sti­tute was cre­at­ed to ad­vance the study of psy­che­delics for men­tal health dis­or­ders and oth­er con­di­tions, in­clud­ing those that lever­age psilo­cy­bin (the sub­stance in “mag­ic mush­rooms”), MD­MA, and prod­ucts in­spired by ayahuas­ca (a South Amer­i­can psy­choac­tive plant med­i­cine).

“It’s an ex­cit­ing time for re­search in­to psy­che­delics, with a grow­ing body of rig­or­ous sci­en­tif­ic ev­i­dence in­di­cat­ing that these sub­stances may pro­vide a po­tent new class of treat­ments for men­tal health dis­or­ders and pos­si­bly oth­er med­ical con­di­tions,” co-di­rec­tor Daniel Perkins said in a state­ment.

A hand­ful of oth­er in­sti­tutes for psy­che­delics re­search ex­ist around the world, in­clud­ing the Im­pe­r­i­al Cen­tre for Psy­che­del­ic Re­search, and the Johns Hop­kins Cen­ter for Psy­che­del­ic and Con­scious­ness Re­search. Nicole De­Feud­is 

Agios’ old AML drug clears a reg­is­tra­tional tri­al in Chi­na

Servi­er read out pos­i­tive Phase III re­sults yes­ter­day show­ing that Agios’ old AML drug Tib­so­vo helped new­ly di­ag­nosed pa­tients live longer. Now, re­searchers are say­ing the drug al­so cleared a reg­is­tra­tional study in Chi­na.

In a study run by C-Stone, Tib­so­vo met the pre-spec­i­fied end­points in Chi­nese pa­tients with re­lapsed or re­frac­to­ry AML with a sus­cep­ti­ble IDH1 mu­ta­tion, the com­pa­ny said on Tues­day. While more de­tailed da­ta are com­ing at a fu­ture con­fer­ence, C-Stone said Chi­na’s Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion (NM­PA) has ac­cept­ed an NDA.

“In the field of AML treat­ment, we are fac­ing ur­gent clin­i­cal treat­ment needs, es­pe­cial­ly for R/R AML pa­tients with a sus­cep­ti­ble IDH1 mu­ta­tion, cur­rent­ly there is no tar­get­ed ther­a­py avail­able on the mar­ket in Chi­na,” prin­ci­pal in­ves­ti­ga­tor Wang Jianx­i­ang said in a state­ment.

Agios won ap­proval for Tib­so­vo in the US back in 2018 based on a sin­gle, sin­gle-armed tri­al but Eu­ro­pean reg­u­la­tors want­ed more da­ta. C-Stone struck a li­cens­ing deal that same year for rights to the drug in Main­land Chi­na, Hong Kong, Macau and Tai­wan. Then last year, Servi­er spent near­ly $2 bil­lion to ac­quire Tib­so­vo and the rest of Agios’ can­cer pipeline.

On Mon­day, Servi­er said those who re­ceived Tib­so­vo and chemother­a­py in a ran­dom­ized Phase III tri­al lived longer than those who re­ceived a com­bi­na­tion of place­bo and chemother­a­py. They al­so had high­er re­sponse rates and com­plete re­mis­sion rates. — Nicole De­Feud­is

Bel­gium’s eTh­eR­NA sets up Hong Kong sub­sidiary

In an­oth­er sign of how big the Asia-Pa­cif­ic mar­ket is loom­ing, a Bel­gian biotech spe­cial­iz­ing in mR­NA-based im­munother­a­pies says it will set up a new sub­sidiary in Hong Kong.

Like the lead­ers in the field, eTh­eR­NA says it’s pur­su­ing both can­cer and in­fec­tious dis­ease ap­pli­ca­tions.

eTh­eR­NA Asia, as the sub­sidiary will be called, will fo­cus on “sup­port­ing busi­ness and in­vestor re­la­tions in Chi­na and es­pe­cial­ly the re­la­tion­ship be­tween eTh­eR­NA and its strate­gic part­ner, Chi­na Grand Phar­ma­ceu­ti­cal and Health­care Hold­ings.” The duo had ear­li­er cre­at­ed a joint ven­ture named Nan­jing Au­roR­NA. — Am­ber Tong

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.