NEA, 5AM back a $50M play to steer gene ther­a­pies to the clin­ic at Ak­ou­os — the lat­est start­up in hear­ing

Hear­ing is get­ting to be a trendy R&D top­ic in biotech.

Eight months af­ter putting to­geth­er a $7.5 mil­lion seed round and lin­ing up AAV gene ther­a­py tech­nol­o­gy out of Mass­a­chu­setts Eye and Ear and Lon­za, the start­up Ak­ou­os has come back with a fast $50 mil­lion A round de­signed to get them through their pre­clin­i­cal phase and up to the thresh­old of their first hu­man study.

Michael McKen­na

Ini­tial­ly fo­cused on a rare ge­net­ic mu­ta­tion that leads to deaf­ness in new­borns, Ak­ou­os CEO Man­ny Si­mons tells me that the biotech has its eyes on a line­up of mono­genet­ic ail­ments re­lat­ed to hear­ing. And they’re look­ing to re­store hear­ing with a pipeline of ther­a­pies, even­tu­al­ly ex­pand­ing in­to broad­er caus­es of hear­ing loss for an ag­ing so­ci­ety.

“Where we’ve been fo­cused is mak­ing sure we are able to de­liv­er vec­tor to sen­so­ry cells through­out the cochlea in the hu­man ear,” the shell-like sphere where vi­bra­tions be­come sound, says Si­mons, a biotech vet with stints at Voy­ager — an­oth­er gene ther­a­py spe­cial­ist — and Warp Dive. This is his first turn run­ning a biotech, and his back­ground helped him line up a ma­jor league crew of back­ers.

Arthur Tzian­a­bos

5AM and New En­ter­prise As­so­ci­ates led the round, with Part­ners In­no­va­tion Fund step­ping in with new in­vestors:Sofinno­va Ven­tures, RA Cap­i­tal Man­age­ment and No­var­tis Ven­ture Fund.

Like most star­tups, the CEO at Ak­ou­os is stay­ing qui­et about their first tar­get for now. But af­ter hav­ing time to ex­plore their tech in an­i­mal mod­els, in­clud­ing non-hu­man pri­mates, he’s al­so ea­ger to note that their first pro­gram puts them in the open­ing stages of ex­plor­ing a field with 150 mono­genet­ic trig­gers for hear­ing loss.

Christo­pher Smith

Ak­ou­os is join­ing a rel­a­tive­ly small but grow­ing group of drug de­vel­op­ers fo­cused on hear­ing in a world where deaf­ness is be­com­ing in­creas­ing­ly com­mon.

Not far from where Ak­ou­os makes its home, Deci­bel has been pur­su­ing its own ap­proach to build­ing a tech plat­form for new drugs to re­store lost hear­ing. Just weeks ago Steve Holtz­man and his crew pieced to­geth­er a $55 mil­lion C round — though they’re de­vel­op­ing a pipeline of com­pounds. Just this morn­ing Fre­quen­cy Ther­a­peu­tics spot­light­ed a move in­to a Phase I/II study for a hear­ing restora­tion drug. A host of aca­d­e­m­ic groups, mean­while, have been look­ing to de­liv­er a gene ther­a­py for hear­ing via a vec­tor — as that field steadi­ly deep­ens its roots fol­low­ing a land­mark ap­proval for Spark.

Si­mons is ex­pand­ing his team with the new mon­ey. One of his sci­en­tif­ic founders at Har­vard, Michael McKen­na, is com­ing on as chief med­ical of­fi­cer. And he has some fa­mil­iar biotech/ven­ture ex­ecs join­ing the board: Arthur Tzian­a­bos, the CEO of Ho­mol­o­gy Med­i­cines, and Christo­pher Smith, for­mer CEO of Cochlear.


Im­age: Man­ny Si­mons. HAR­VARD BUSI­NESS SCHOOL via YOUTUBE

Aerial view of Genentech's campus in South San Francisco [Credit: Getty]

Genen­tech sub­mits a big plan to ex­pand its South San Fran­cis­co foot­print

The sign is still there, a quaint reminder of whitewashed concrete not 5 miles from Genentech’s sprawling, chrome-and-glass campus: South Francisco The Industrial City. 

The city keeps the old sign, first erected in 1923, as a tourist site and a kind of civic memento to the days it packed meat, milled lumber and burned enough steel to earn the moniker “Smokestack of the Peninsula.” But the real indication of where you are and how much has changed both in San Francisco and in the global economy since a couple researchers and investors rented out an empty warehouse 40 years ago comes in a far smaller blue sign, resembling a Rotary Club post, off the highway: South San Francisco, The Birthplace of Biotech.

Here comes the oral GLP-1 drug for di­a­betes — but No­vo Nordisk is­n't dis­clos­ing Ry­bel­sus price just yet

Novo Nordisk’s priority review voucher on oral semaglutide has paid off. The FDA approval for the GLP-1 drug hit late Friday morning, around six months after the NDA filing.

Rybelsus will be the first GLP-1 pill to enter the type 2 diabetes market — a compelling offering that analysts have pegged as a blockbuster drug with sales estimates ranging from $2 billion to $5 billion.

Ozempic, the once-weekly injectable formulation of semaglutide, brought in around $552 million (DKK 3.75 billion) in the first half of 2019.

As Nas­daq en­rolls the fi­nal batch of 2019 IPOs, how have the num­bers com­pared to past years?

IGM Biosciences’ upsized IPO haul, coming after SpringWorks’ sizable public debut, has revved up some momentum for the last rush of biotech IPOs in 2019.

With 39 new listings on the books and roughly two more months to go before winding down, Nasdaq’s head of healthcare listings Jordan Saxe sees the exchange marking 50 to 60 biopharma IPOs for the year.

“December 15 is usually the last possible day that companies will price,” he said, as companies get ready for business talks at the annual JP Morgan Healthcare Conference in January.

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Oxitec biologist releases genetically modified mosquitoes in Piracicaba, Brazil in 2016 [credit: Getty Images]

In­trex­on unit push­es back against claims its GM mos­qui­toes are mak­ing dis­ease-friend­ly mu­tants

When the hysteria of Zika transmission sprang into the American zeitgeist a few years ago, UK-based Oxitec was already field-testing its male Aedes aegypti mosquito, crafted to possess a gene engineered to obliterate its progeny long before maturation.

But when a group of independent scientists evaluated the impact of the release of these genetically-modified mosquitoes in a trial conducted by Oxitec in Brazil between 2013 and 2015, they found that some of the offspring had managed to survive — prompting them to speculate what impact the survivors could have on disease transmission and/or insecticide resistance.

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Pur­due threat­ens to walk away from set­tle­ment, asks to pay em­ploy­ees mil­lions in bonus­es

There are two updates on the lawsuit against Purdue Pharma over its role in fueling the opioid epidemic, as the Sackler family threatens to walk away from their pledge to pay out $3 billion if a bankruptcy judge does not stop outstanding state lawsuits against them. At the same time, the company has asked permission to pay millions in bonuses to select employees.

Purdue filed for chapter 11 bankruptcy this week as part of its signed resolution to over 2,000 lawsuits. The deal would see the Sackler family that owns Purdue give $3 billion from their personal wealth and the company turned into a trust committed to curbing and reversing overdoses.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

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UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.