Ben Dake (Source: Aerovate)

RA Cap­i­tal-backed Aerovate launch­es with $72.6M to treat PAH with a re­pur­posed can­cer med

The land­mark can­cer drug ima­tinib has been on the mar­ket since 2001, first sold by No­var­tis as Gleevec and in re­cent years as a gener­ic. Now, a new Boston biotech is aim­ing to re­pur­pose the drug as a treat­ment for pul­monary ar­te­r­i­al hy­per­ten­sion.

Aerovate emerged from stealth Thurs­day and an­nounced a $72.6 mil­lion Se­ries A, which will be used to de­vel­op and run tri­als for its can­di­date AV-101 — a dry pow­der ver­sion of ima­tinib meant to be used with an in­haler. The com­pa­ny emerged from RA Cap­i­tal’s in­cu­ba­tor and fund­ing was led by Sofinno­va.

Ima­tinib had been test­ed oral­ly in PAH pa­tients be­fore, CEO Ben Dake tells me. Re­sults from a Phase III tri­al with the oral de­liv­ery ver­sion showed promis­ing ef­fi­ca­cy lev­els, but PAH pa­tients didn’t tol­er­ate the drug well.

“Tak­ing an an­ti-pro­lif­er­a­tive can­cer drug and try­ing to fig­ure out a way to make it work in a dif­fer­ent dis­ease made a lot of sense to me and to the team,” Dake told End­points News. “But even though it was dif­fi­cult for them to tol­er­ate, it did work well on ex­er­cise ca­pac­i­ty, which is the pri­ma­ry end­point for ap­proval for PAH drugs, as well as he­mo­dy­nam­ics.”

One of the key chal­lenges Aerovate faced in de­vel­op­ment was how to de­liv­er the drug ex­act­ly to where it need­ed to be in the lungs. The biotech set­tled on an in­haled method but, due to the na­ture of such ther­a­pies, still had to de­ter­mine pre­cise­ly the right size par­ti­cle and make sure the mol­e­cule doesn’t ab­sorb wa­ter.

Ul­ti­mate­ly the com­pa­ny set­tled on ima­tinib and craft­ed a prod­uct that they hope is eas­i­er to use than oth­er PAH treat­ments like a liq­uid neb­u­liz­er, in which pa­tients have to mix the so­lu­tions them­selves and car­ry around a large de­vice. Af­ter set­tling on ima­tinib and pri­or­i­tiz­ing ease of use, Aerovate is con­tent with where it land­ed.

“We had to do quite a bit of work to find a good, sta­ble, well-per­form­ing aerosol,” Dake said. “It’s not some­thing as sim­ple as grind­ing up a pill.”

Aerovate sees it­self as step­ping in PAH pa­tients whose con­di­tions have wors­ened de­spite oth­er ther­a­pies, Dake said. Gen­er­al­ly, some­one with PAH will start on a reg­i­men of PDE5 in­hibitors that di­late blood ves­sels and progress through two or three oth­er pills be­fore po­ten­tial­ly need­ing an IV or sub­cu­ta­neous pump.

At that point, there is no fur­ther treat­ment avail­able. AV-101 aims to fill that gap as Dake says it shifts the ther­a­py par­a­digm from stan­dard of care to dis­ease-mod­i­fy­ing treat­ment.

“We want­ed to be aware of where the ther­a­peu­tic fo­cus was go­ing and not with an old­er-style of drug, and there’s oth­er ad­van­tages to dry pow­der but the main one is for pa­tients in that it’s easy to use,” Dake said. “Where we would come in is where pa­tients are al­ready on a dou­ble ther­a­py or triple ther­a­py, they can add on our in­haler. And that is where the oral [ima­tinib] tri­al was run.”

Long-term, Aerovate has oth­er pro­grams it would like to de­vel­op but is fo­cused on bring­ing AV-101 to mar­ket. Josh Resnick, a man­ag­ing di­rec­tor at RA Cap­i­tal and mem­ber of Aerovate’s board, said the path to com­mer­cial­iza­tion is “rel­a­tive­ly straight­for­ward” giv­en that the FDA is al­ready fa­mil­iar with the ima­tinib com­pound.

“It’s a pa­tient pop­u­la­tion that’s well-un­der­stood, it’s a set of physi­cians that’s well-un­der­stood, so fun­da­men­tal­ly we an­tic­i­pate it to be a first-in-class ther­a­peu­tic in the space and get it in­to the hands of the pa­tients,” Resnick said.

Oth­er in­vestors join­ing Sofinno­va are At­las Ven­ture, Cor­morant As­set Man­age­ment, Sur­vey­or Cap­i­tal and Os­age Uni­ver­si­ty Part­ners.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA have vowed not to let politics get in the way of science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped health agencies under his purview — including the FDA — of their rulemaking ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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#ES­MO20: As­traZeneca aims to spur PRO­found shift in prostate can­cer treat­ment with Lyn­parza OS da­ta

AstraZeneca has unveiled the final, mature overall survival data that cemented Lynparza’s first approval in prostate cancer approval — touting its lead against rivals with the only PARP inhibitor to have demonstrated such benefit.

But getting the Merck-partnered drug to the right patients remains a challenge, something the companies are hoping to change with the new data cut.

The OS numbers on the subgroup with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer are similar to the first look on offer when the FDA expanded the label in May: Lynparza reduced the risk of death by 31% versus Xtandi and Zytiga. Patients on Lynparza lived a median of 19.1 months, compared to 14.7 months for the anti-androgen therapies (p = 0.0175).

Eli Lilly CSO Dan Skovronsky (file photo)

#ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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#ES­MO20: Out to beat Tagris­so, J&J touts 100% ORR for EGFR bis­pe­cif­ic/TKI com­bo — fu­el­ing a quick leap to PhI­II

J&J’s one-two punch on EGFR-mutant non-small cell lung cancer has turned up some promising — although decidedly early — results, fueling the idea that there’s yet room to one up on third-generation tyrosine kinase inhibitors.

Twenty out of 20 advanced NSCLC patients had a response after taking a combination of an in-house TKI dubbed lazertinib and amivantamab, a bispecific antibody targeting both EGFR and cMET engineered on partner Genmab’s platform, J&J reported at ESMO. All were treatment-naïve, and none has seen their cancer progress at a median follow-up of seven months.

#ES­MO20: It’s not just Keytru­da any­more — Mer­ck spot­lights 3 top ear­ly-stage can­cer drugs

Any $12 billion megablockbuster in the portfolio tends to overshadow everything else in the pipeline. Which is something Merck can tell you a little bit about.

Keytruda not only dominates the PD-(L)1 field, it looms over everything Merck does, to the point some analysts wonder if Merck is a one-trick pony.

There’s no shortage of Keytruda data on display at ESMO this weekend, but now the focus is shifting to the future role of new drugs and combos in maintaining that lead position for years to come. And the pharma giant has a special focus for 3 early-stage efforts where Roger Perlmutter’s oncology team is placing some big bets.

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Exelixis CEO Michael Morrissey (file photo)

#ES­MO20: Look out Mer­ck. Bris­tol My­ers and Ex­elix­is stake out their com­bo’s claim to best-in-class sta­tus for front­line kid­ney can­cer

Now that the PD-(L)1 checkpoints are deeply entrenched in the oncology market, it’s time to welcome a wave of combination therapies — beyond chemo — looking to extend their benefit to larger numbers of patients. Bristol Myers Squibb ($BMY} and Exelixis {EXEL} are close to the front of that line.

Today at ESMO the collaborators pulled the curtain back on some stellar data for their combination of Opdivo (the PD-1) and Cabometyx (the TKI), marking a significant advance for the blockbuster Bristol Myers franchise while offering a big leg up for the team at Exelixis.

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Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.