Dicer­na and Boehringer part­ner on RNAi treat­ment for NASH

Phar­ma gi­ant Boehringer In­gel­heim In­ter­na­tion­al is part­ner­ing with Dicer­na Phar­ma­ceu­ti­cals $DR­NA on a re­search and li­cens­ing deal to ad­vance treat­ments for chron­ic liv­er dis­eases.

Dicer­na gets $10 mil­lion in an up­front pay­ment, and could re­ceive up to an ad­di­tion­al $191 mil­lion in po­ten­tial mile­stone pay­ments and R&D re­im­burse­ments.

Clive Wood

The duo will ini­tial­ly fo­cus on non­al­co­holic steato­hep­ati­tis (NASH), al­so known as non­al­co­holic fat­ty liv­er dis­ease, which has been a bea­con for a slew of bio­phar­ma com­pa­nies out to con­quer a fast-grow­ing ail­ment.

Dicer­na is de­vel­op­ing in­ves­ti­ga­tion­al RNA in­ter­fer­ence (RNAi) ther­a­peu­tics that could po­ten­tial­ly treat NASH. Dicer­na hopes its RNAi tech­nol­o­gy, called GalXC, can in­hib­it the ex­pres­sion of dis­ease-caus­ing genes by de­stroy­ing the mes­sen­ger RNAs of those genes.

The deal of­fers an en­dorse­ment of Dicer­na’s plat­form tech, which has come un­der a fierce at­tack from their big ri­vals at the RNAi biotech Al­ny­lam. Al­ny­lam al­leged that Dicer­na il­lic­it­ly gained trade se­crets from some Sir­na sci­en­tists it hired as Mer­ck was shop­ping the com­pa­ny to Al­ny­lam. Dicer­na, though, re­cent­ly fired back in fed­er­al court, claim­ing that the law­suit was noth­ing but a sham aimed at ic­ing part­ner­ships just like the one an­nounced to­day.

Paul Mat­teis at Leerink liked what he was see­ing here:

We view this as ear­ly val­i­da­tion for DR­NA’s GalXC plat­form, which will see their first drug, DCR-PHXC (pri­ma­ry hy­per­ox­aluria), in the clin­ic ear­ly next year. As DR­NA is in an on­go­ing lit­i­ga­tion with AL­NY (MP) over al­le­ga­tions of mis­ap­pro­pri­at­ing trade se­crets, it’s cer­tain­ly en­cour­ag­ing that they were still able to pre­cip­i­tate a part­ner­ship. Re­call that the courts have ac­cept­ed DR­NA’s counter claims and that a ju­ry tri­al is slat­ed for April of next year.

Boehringer, un­de­terred by the le­gal spar­ring, said Dicer­na’s pro­gram is a good match for its car­diometa­bol­ic pipeline.

“Our re­search team is dili­gent­ly work­ing to dis­cov­er ef­fec­tive new ther­a­pies for NASH and oth­er chron­ic liv­er dis­eases, which is a pri­or­i­ty area for us,” said Clive Wood, cor­po­rate se­nior vice pres­i­dent of dis­cov­ery re­search at Boehringer In­gel­heim, in a state­ment. “This part­ner­ship com­ple­ments our ex­ist­ing re­search ef­forts and ex­per­tise and of­fers dis­tinct ad­van­tages in de­vel­op­ing ex­cit­ing new ther­a­py op­tions.”

Boehringer has a port­fo­lio of mar­ket­ed drugs for car­diometa­bol­ic dis­ease, in­clud­ing prod­ucts for throm­boem­bol­ic dis­ease, type 2 di­a­betes, and hy­per­ten­sion.

In ad­di­tion to the po­ten­tial $191 mil­lion in ex­tra pay­ments, Dicer­na is el­i­gi­ble to re­ceive roy­al­ties stag­gered up to dou­ble dig­its on world­wide net sales.

Jean-Paul Clozel, Idorsia CEO (Patrick Straub/Keystone via AP Images)

Idor­si­a's brain bleed drug flunks PhI­II tri­al, a decade af­ter pre­vi­ous flop

Idorsia’s long journey with clazosentan came to an abrupt “unexpected result” Monday morning with a Phase III flop.

The Swiss biopharma said the drug did not meet the main goal of the late-stage REACT study, conducted in the US, Canada and Europe since early 2019.

The 409-patient trial tested the intravenous drug’s ability to prevent complications due to delayed cerebral ischemia following aneurysmal subarachnoid hemorrhage (aSAH), in which blood vessels in the brain narrow and blood accumulates around the brain’s surface, which then dials up the pressure on the brain.

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Kenji Yasukawa, Astellas CEO (Photographer: Akio Kon/Bloomberg via Getty Images)

Astel­las taps chief strat­e­gy of­fi­cer as next CEO to 'go on the ag­gres­sive'

Five years into its big R&D revamp, Astellas says it’s time for a changing of the guard.

Kenji Yasukawa, who took over as president and CEO in 2018, will step down to become chairman of the board in April, making room for Naoki Okamura to take over. Okamura joined the company in 1986 and has served in a variety of finance, business and strategy roles, including most recently as chief strategy officer.

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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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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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How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

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The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

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Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

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Roche's headquarters in Basel, Switzerland (Kyle LaHucik for Endpoints News)

Roche ditch­es fi­nal PhI­II for can­cer hope­ful, re­ports set­back for key drug in $1.4B buy­out

Over the past few years, Roche has released news about its AKT inhibitor ipatasertib in drips — most of them negative. The drug yielded mixed data in a key prostate cancer trial, Phase III flops in triple-negative breast cancer forced the pharma giant to pull the plug there, and in mid-2022 Roche trimmed two more early-stage indications in prostate cancer after completing the trials.

Now, the last piece of the program is gone.

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