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 has vowed not to let politics overrule 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 the FDA and other health agencies under his purview of their rule making 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.

Can a mag­net­ic cell ther­a­py re­place corneal trans­plan­ta­tion? As eight-year jour­ney leads to the clin­ic, two broth­ers un­veil bold vi­sion

Jeff Goldberg was getting acquainted with a brand new way to do corneal transplants when an even newer, even bolder idea hit him.

It was almost 10 years ago, and Goldberg was in his first faculty position at Bascom Palmer Eye Institute at the University of Miami. Scientists had developed a new way to do cornea transplants where instead of sewing a whole donor cornea — a decades-old practice — they were just engrafting the inner layer of cells.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists. HHS con­tin­ues to claim Azar “will de­fer com­plete­ly to the FDA"

President Donald Trump, who seems intent on announcing a Covid-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.

Embattled CDC director Robert Redfield (AP Images)

Covid-19 roundup: CDC ad­vi­so­ry com­mit­tee de­lays pri­or­i­ty dis­tri­b­u­tion vote; EU re­port­ed­ly in­dem­ni­fy­ing vac­cine mak­ers

A federal committee that advises the CDC was expected to hold a vote Tuesday on a plan regarding the distribution for initial doses of approved Covid-19 vaccines. But that vote has been scrapped.

The Advisory Committee on Immunization Practices, or ACIP, won’t be voting until the committee members learn more about which vaccines become available first, the Wall Street Journal reported. The vote could potentially wait until a specific vaccine is authorized before recommending how to dole out the first doses.

Anthony Coyle (Repertoire)

Flag­ship's merged biotech Reper­toire nets ex-Pfiz­er CSO An­tho­ny Coyle as R&D chief

Flagship is building a big-name C-suite at its new, $220 million merged biotech.

Repertoire Immune Medicines, which already boasts former Bioverativ chief John Cox as its CEO, announced yesterday that Anthony Coyle, the former Pfizer CSO and the founding CEO of Pandion, will join as their head of R&D.

“As we progress clinical trials for our multi-clonal T cell candidates in immuno-oncology, Tony’s deep expertise in cellular immunology and novel therapeutic development will help us achieve our vision of creating a new class of transformative medicines for patients,” Cox said in a statement.

Zai Lab hauls in $761M from Hong Kong IPO to push Ze­ju­la, more bud­ding can­di­dates in Chi­na — re­port

Zai Lab is set to net more than $761 million from its secondary listing in Hong Kong after pricing the IPO at $72.51 (HKD$562) — just a hair below its Nasdaq closing price on Monday, Bloomberg and Nikkei Asian Review reported.

A pioneer in bringing Western drugs to China, co-founder and CEO Samantha Du has more than tripled Zai Lab’s market cap in the three years it’s been public in the US. The HKEX listing is designed to fund R&D and commercialization for the current portfolio while fueling new in-licensing pacts, the biotech wrote in a filing.

Tae Han Kim, Samsung Biologics CEO (SeongJoon Cho/Bloomberg via Getty Images)

Sam­sung Bi­o­log­ics nets $330M+ deal from As­traZeneca ahead of 'Su­per Plan­t' con­struc­tion

Just a few weeks after announcing plans to construct a $2 billion “Super Plant,” Samsung Biologics is keeping its foot on the gas.

The Korean CDMO has inked a $330.8 million manufacturing contract with AstraZeneca, the companies announced Monday evening, providing the British drugmaker the ability to expand production capabilities in the Asia market. Per terms of the deal, the partnership could be increased to $545.6 million.