Can a new pro­tein bring RNA drugs where they can't reach? J&J vet Sue Dil­lon's Io­n­is-part­nered biotech gets $88M to prove yes

Sue Dil­lon re­mem­bers the un­cer­tain­ty when she first left J&J to start her own biotech around a new kind of mol­e­cules called cen­tyrins.

Sue Dil­lon

J&J had al­ready li­censed the tech, which was de­vel­oped by her co-founder Karyn O’Neil at the phar­ma gi­ant’s Cen­tyrex unit, to two com­pa­nies for use in CAR-T and ra­dio­phar­ma­ceu­ti­cals. But Aro Bio­ther­a­peu­tics want­ed to ap­ply it to what was still a nascent area: RNA med­i­cines, en­com­pass­ing both an­ti­sense oligonu­cleotides and siR­NA ther­a­pies.

“There was a lot of skep­ti­cism out there around whether or not oligonu­cleotides would be­come a sig­nif­i­cant drug plat­form,” she told End­points News. “I don’t think any­body doubts that now.”

For all the progress, though, the need that the duo saw re­mains. Out­side the liv­er and cer­tain dis­eases primed for lo­cal de­liv­ery, drug­mak­ers have had trou­ble de­liv­er­ing these drugs to oth­er tis­sues. Aro’s pro­posed so­lu­tion was promis­ing and per­ti­nent enough that af­ter J&J pro­vid­ed the ini­tial $13 mil­lion to get things off the ground, Io­n­is, a gi­ant in the an­ti­sense nu­cleotide field, signed up for a dis­cov­ery pact at the be­gin­ning of 2020.

One year lat­er, Dil­lon and O’Neil are ready to steer their own can­di­dates to­ward the clin­ic, armed with $88 mil­lion in Se­ries A fund­ing pro­vid­ed by the ex­ist­ing in­vestors at John­son & John­son In­no­va­tion – JJDC, Io­n­is and Bio­Mo­tiv, as well as new back­ers led by North­pond Ven­tures and Cowen Health­care (the oth­ers are Health­Cap, BVF Part­ners and Ridge­back Cap­i­tal).

If the con­cept checks out — and Aro is lean­ing on some se­ri­ous proof-of-con­cept from O’Neil’s J&J days that it would — it promis­es to change not just an­ti­sense but a slew of ge­net­ic med­i­cines that are re­shap­ing the de­f­i­n­i­tion of a drug.

Cen­tyrins, Dil­lon ex­plained, are mod­eled on a hu­man pro­tein from the fi­bronectin fam­i­ly that vague­ly re­sem­bles an anti­gen bind­ing do­main of a very large an­ti­body. They are small (just one-fif­teenth the size of an an­ti­body) but bind to cell sur­face re­cep­tors with high affin­i­ty, and they can be eas­i­ly man­u­fac­tured in E. coli in the lab.

Karyn O’Neil

Not on­ly has O’Neil fig­ured out a way to con­ju­gate them to RNA mol­e­cules, her team can al­so cre­ate bis­pe­cif­ic or mul­ti­spe­cif­ic cen­tyrins to se­lect for cer­tain cells that have all the re­cep­tors.

The com­pa­ny isn’t say­ing much about the pipeline oth­er than that the ini­tial pro­grams will tar­get mus­cle dis­eases and im­munol­o­gy — Dil­lon’s ex­pert area at J&J — and range from the rare, or­phan dis­or­der to more com­mon ail­ments. As is com­mon for an ear­ly-stage ven­ture, the CEO al­so re­frained from com­mit­ting to a time­line for en­ter­ing the clin­ic but not­ed the fund­ing should last them two to three years.

In par­al­lel with the lead pro­grams, the team of 20 is al­so ex­plor­ing ways to sys­tem­i­cal­ly de­liv­er mR­NA, DNA or CRISPR/Cas ma­chin­ery — any­thing that’s wrapped in nanopar­ti­cles — to tar­get tis­sues.

“I think that the biggest sin­gle thing over the last 3 years is the emer­gence of broad­ly ge­net­ic med­i­cines as the ma­jor class of med­i­cines for the fu­ture, and along with that, the recog­ni­tion that a ma­jor chal­lenge fac­ing the en­tire field of any of those types of med­i­cines is the chal­lenge of tis­sue spe­cif­ic de­liv­ery,” Dil­lon said. “That’s where Aro comes in. And that’s why I be­lieve even more than I did in 2018 when we first start­ed the com­pa­ny that there is just enor­mous po­ten­tial here for us to re­al­ly make a dif­fer­ence.”

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Martin Landray, Protas CEO (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Those big bil­lion-dol­lar PhI­II stud­ies? Mar­tin Lan­dray says they can be done for a tiny frac­tion of the cost

Martin Landray knows what controversy in clinical drug development feels like, from first-hand experience.

Landray was the chief architect of RECOVERY, a study that pitted a variety of drugs against Covid-19. And he offered some landmark data that would help push dexamethasone out into broader use as a cheap treatment, while helping ice hydroxy’s reputation as a clear misfire.

“Lots of people told us we shouldn’t use it,” Landray says about dexamethasone and Covid-19. “It was dangerous. We shouldn’t even do a trial. They also cared about hydroxychloroquine and lots of people said we shouldn’t do a trial because it must be used. I’ve got the letters from both sets of people.”

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FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs-up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Geoffrey Porges, new Schrödinger CFO

Long­time an­a­lyst Ge­of­frey Porges de­parts SVB to lead fi­nances at a drug dis­cov­ery shop

Geoffrey Porges has ended his two-decade run as a biotech analyst, as the former SVB Securities vice chair began as CFO of Schrödinger on Thursday.

The long-running analyst, who previously headed up vaccines marketing at Merck before the turn of the millennium, will lead the financial operations of the 700-employee company as Schrödinger broadens its focus from a drug discovery partner to also building out an in-house pipeline, with clinical trial No. 1 set to begin next quarter.

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James Dentzer, Curis CEO

FDA lifts par­tial hold on Curis' lym­phoma study — shares spike

Four months after the FDA put two clinical trials from Curis on clinical hold, the FDA is now apparently content with how the biotech will change up managing one of the studies.

The Massachusetts oncology biotech put out word early Thursday that the federal regulator lifted a partial clinical hold of the company’s Phase I/II study of emavusertib in lymphoma, following a new data package that the biotech recently submitted to the agency. Shares of the biotech $CRIS, hovering just above penny stock territory, shot up more than 55% in early trading before settling at close to a 30% share price boost.

Astel­las' hot flash­es drug will get speedy re­view at FDA; US opts out of Val­ne­va vac­cine

The FDA will decide on Astellas’ menopausal symptom drug by Feb. 22 of next year, as the Japanese pharma disclosed it had paid about $97 million to get a priority review voucher to speed up the review.

Astellas said the agency has accepted the pharma’s application for fezolinetant for the treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. VMS includes hot flashes and/or night sweats. The company said as many as 80% of women in the US experience those symptoms during or after the menopausal transition.

Joel Dudley, new partner at Innovation Endeavors (Bosch Health Campus)

For­mer Google CEO’s VC is mak­ing a big­ger push in­to the biotech world, hir­ing promi­nent Ther­a­nos skep­tic

Venture capital firm Innovation Endeavors has mainly had its focus on investments across the tech space, but it has been slowly turning its attention to the biotech world. Now, a new partner is coming into the fold showing that its interest in biotech is likely to grow further.

The Silicon Valley-based company, which is headed up by former Google CEO Eric Schmidt, has brought on Joel Dudley as a partner. According to Dudley’s LinkedIn page, he is joining Innovation Endeavors after serving as the chief science officer of biotech startup Tempus Labs since 2020.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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