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.


Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Cy­to­ki­net­ics’ ALS drug fails PhI­II, leav­ing the biotech with a sin­gle late-stage prospect

Cytokinetics’ candidate for the muscle disease amyotrophic lateral sclerosis, or ALS, failed a Phase III trial, the Bay Area biotech announced Friday morning.

At a second interim analysis of the trial, an independent review committee recommended that Cytokinetics discontinue its COURAGE-ALS trial for reldesemtiv, as it “found no evidence of effect” compared to placebo on the primary or key secondary endpoints.

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CHMP gives thumbs-up for We­govy use in ado­les­cents, along with nine new drug rec­om­men­da­tions

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended nine drugs for approval this week while also giving thumbs up for six expanded indications, including Novo Nordisk’s approved obesity medication Wegovy for younger people. Wegovy is already approved as an obesity treatment in the EU for adults, and the new indication would allow prescriptions for adolescents aged 12 and older.

Green­Light re­ceives buy­out of­fer; Apol­lomics com­pletes SPAC merg­er

RNA biotech GreenLight Biosciences has been handed an offer for potential acquisition.

GreenLight said in a release that it has received a non-binding “indication of interest” from Fall Line Endurance Fund to acquire GreenLight’s capital stock for $0.60 per share in cash. The release said any potential agreement between the two parties would depend on certain conditions.

Through a special committee, the biotech will evaluate the offer but added there’s no certainty a deal will go forward. GreenLight will also not make any more announcements until a deal comes through or “otherwise determines” a statement is necessary.

TScan Therapeutics' departing CEO David Southwell and CSO/COO Gavin MacBeath

TCR up­start an­nounces CEO ex­it, with CSO now act­ing re­place­ment

A public T cell biotech’s chief executive has decided to leave the company.

TScan Therapeutics said Friday morning that CEO David Southwell stepped down earlier this week, leaving both his chief executive and board member roles. Filling in is Gavin MacBeath, the company’s CSO and COO. He became the acting CEO on Tuesday, and will continue to remain CSO and COO, TScan’s announcement read.

Sar­to­rius to ac­quire French man­u­fac­tur­er for $2.6B+ in cell and gene ther­a­py play

The German life science group Sartorius will be picking up French contract manufacturer Polyplus for the price of €2.4 billion, or $2.6 billion.

On Friday, Sartorius announced the acquisition through its French subgroup, Sartorius Stedim Biotech, which will be acquiring Polyplus from private investors ARCHIMED and WP GG Holdings IV. Polyplus has 270 employees and produces materials and components that go into making viral vectors that are used in cell and gene therapies. This includes DNA/RNA reagents as well as plasmid DNA. Polyplus has locations in France, Belgium, China and the US.

Austin biotech Mol­e­c­u­lar Tem­plates lays off more than 100 staffers as pipeline nar­rows

Molecular Templates is ridding itself of a Phase I HER2 asset and fine-tuning its pipeline to focus on three programs and a preclinical Bristol Myers Squibb collaboration. With the narrowed scope on its so-called engineered toxin bodies, the Austin, TX biotech is laying off about half of its staff.

That’s a little more than 100 employees, per an SEC filing. Molecular’s layoffs, approved by its board Wednesday, add to the dozens of pullbacks in the industry in the first three months of 2023.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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