Zhen Li, ADARx Pharmaceuticals CEO

A new play­er joins the RNA edit­ing race on the heels of Eli Lil­ly's $1B-plus deal

A day af­ter Eli Lil­ly inked a $1 bil­lion-plus deal to part­ner with a qui­et RNA edit­ing start­up, a new play­er has emerged to turn up the vol­ume.

ADARx Phar­ma­ceu­ti­cals un­cloaked on Thurs­day with a $75 mil­lion Se­ries B round to ush­er its RNA pro­grams in­to the clin­ic. The San Diego-based biotech was found­ed in 2019 to dis­cov­er ther­a­pies that pre­cise­ly tar­get and ed­it sin­gle-point mu­ta­tions on RNA tran­scripts.

Un­like gene edit­ing, which looks to di­rect­ly — and per­ma­nent­ly – al­ter DNA, the goal of RNA edit­ing is to in­ter­cept the bro­ken mes­sages sent out by the DNA of pa­tients with cer­tain dis­eases and cor­rect it be­fore it gets turned in­to pro­teins. To do so, sci­en­tists are ex­ploit­ing a nat­u­ral­ly oc­cur­ring en­zyme called ADAR, or adeno­sine deam­i­nas­es act­ing on RNA.

ADARx’s first edit­ing pro­gram is be­ing de­vel­oped for Al­pha-1 an­tit­rypsin de­fi­cien­cy (AAT de­fi­cien­cy), an in­her­it­ed con­di­tion in which AAT, a pro­tein that pro­tects the lungs, isn’t the cor­rect shape and gets stuck in the liv­er where it’s pro­duced. Pa­tients with AAT de­fi­cien­cy are at an in­creased risk for both lung and liv­er dis­ease.

The team is helmed by Zhen Li, who led siR­NA de­vel­op­ment at Mer­ck be­fore tak­ing the se­nior VP role at Ar­row­head Phar­ma­ceu­ti­cals in 2014. Rui Zhu, a for­mer Ar­row­head col­league, al­so hopped on board as VP of R&D.

“We are ex­cit­ed that our RNA base edit­ing tech­nol­o­gy could give rise to a sig­nif­i­cant pipeline of high­ly ef­fec­tive ther­a­peu­tics,” Li said in a state­ment, adding that the com­pa­ny is al­so work­ing on tech for in­hi­bi­tion, degra­da­tion and de­liv­ery across a range of ge­net­ic, car­diometa­bol­ic and cen­tral ner­vous sys­tem dis­eases.

The RNA edit­ing space is heat­ing up, with Roche tee­ing up a $3 bil­lion-plus deal for Shape’s RNA edit­ing plat­form back in Au­gust. The deal came on the heels of Shape’s $112 mil­lion Se­ries B round in Ju­ly, with Roche hop­ing to dis­cov­er po­ten­tial “one-time” cures for Alzheimer’s, Parkin­son’s and oth­er rare dis­eases. And just yes­ter­day, Eli Lil­ly put down $50 mil­lion up­front and $1.25 bil­lion in biobucks for ac­cess to five RNA edit­ing can­di­dates from the Dutch biotech Pro­QR Ther­a­peu­tics.

“Or­biMed is pleased to con­tin­ue to back Zhen Li and her team in the cre­ation of an ex­cit­ing oligonu­cleotide-based drug com­pa­ny, which is cre­at­ing new treat­ment modal­i­ties,” said Carl Gor­don, found­ing part­ner at Or­biMed, which co-led ADARx’s Se­ries B round along with SR One Cap­i­tal Man­age­ment. Sirona Cap­i­tal and Lil­ly Asia Ven­tures al­so chipped in.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Time for round 2: Il­lu­mi­na-backed VC snags $325M for its next fund

Illumina Ventures closed off its second investment fund with a total commitment of $325 million, offering fresh fuel to back a slate of startups that have already included a smorgasbord of companies, covering everything from diagnostics to biotech drug development and genomics.

Fund II brings the total investment under Illumina Ventures’ oversight to $560 million, which has been focused on early-stage companies. And it has a transatlantic portfolio that includes SQZ, Twist and Encoded Therapeutics.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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