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.

Clin­i­cal tri­al di­ver­si­ty da­ta show mis­match be­tween en­roll­ment and dis­ease preva­lence, GSK says

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

The Big Phar­ma dis­card pile; Lay­offs all around while some biotechs bid farewell; New Roche CEO as­sem­bles top team; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

With earnings seasons in full swing, we’ve listened in on all the calls so you don’t have to. But news is popping up from all corners, so make sure you check out our other updates, too.

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Raymond Stevens, Structure Therapeutics CEO

Be­hind Fri­day's $161M IPO: A star sci­en­tist, GPCR drug dis­cov­ery and a plan to chal­lenge phar­ma in di­a­betes

What does it take to pull off a $161 million biotech IPO these days?

In Structure Therapeutics’ case, it means having a star scientist co-founder paired with the computational drug discovery company Schrödinger, $198 million in private funding from blue-chip investors, almost six years of research work on G protein-coupled receptors and a slate of oral, small-molecule drugs, with an eye on the huge and growing diabetes and weight-loss market.

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Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

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Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Af­ter 13 years, Ramy Mah­moud steps in­to CEO seat at Opti­nose; Ru­pert Vessey set to ex­it Bris­tol My­ers in Ju­ly

After 13 years as president and COO at Optinose, Ramy Mahmoud has stepped into a new role as its CEO. He is taking the place of Peter Miller, who stepped down earlier this week, though Miller is still staying with the company as a consultant.

In 2010, the two business partners joined Optinose to take it in a new direction, transforming it from a delivery platform to product company. They previously worked together at Johnson & Johnson, when Miller was president at Janssen and Mahmoud headed medical affairs. Miller said after he learned about Optinose, “I did what I always do, which is find people smarter than me to talk with about the idea. And the first person I called was Ramy … and I said, ‘Hey, Ramy, what do you think of this technology?’”

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Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

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Giovanni Caforio, Bristol Myers Squibb CEO (Nicolas Messyasz/Sipa via AP Images)

Bris­tol My­ers turns at­ten­tion to new prod­ucts in wake of Revlim­id patent loss

Bristol Myers Squibb CEO Giovanni Caforio is shifting his focus to newer products as generic sales continue to gnaw at the company’s blockbuster myeloma drug Revlimid.

Both Revlimid and Abraxane sales took a dive last year thanks to generic rivals, BMS reported in its Q4 and full-year results on Thursday. As a result, Q4 sales dipped 5% and full-year sales remained flat. However, Caforio sees a silver lining — or rather, two of them.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP)

FTC makes an ex­am­ple of GoodRx, bans dis­counter from shar­ing pri­vate health da­ta with ad­ver­tis­ers

Prescription drug discount provider GoodRx will no longer be allowed to share its users’ sensitive health data with advertisers after the Federal Trade Commission charged the online coupon provider with failing to notify consumers of such disclosures to Facebook, Google, and other companies.

GoodRx agreed to pay a $1.5 million civil penalty for violating the FTC’s Health Breach Notification Rule after the FTC said it repeatedly violated a 2017 promise to not share sensitive personal health information. The FTC alleged that the company shared users’ prescription medications and personal health conditions with third party advertisers and platforms like Facebook, Google, Criteo, Branch and Twilio.