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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”