Io­n­is rolls out promis­ing PhI­II da­ta on volane­sors­en for rare en­zyme dis­or­der, but safe­ty ques­tions linger

Louis O’Dea, Akcea

Io­n­is has nailed down pos­i­tive Phase III da­ta on its lead ex­per­i­men­tal drug volane­sors­en, used to com­bat rare cas­es of fa­mil­ial chy­lomi­crone­mia syn­drome. But the biotech $IONS is still bat­ting back ques­tions about safe­ty is­sues as it preps for fil­ings with the FDA and EMA.

In the Phase III in­ves­ti­ga­tors re­port­ed a huge drop in triglyc­erides among the 33 FCS pa­tients tak­ing the drug, hit­ting the pri­ma­ry end­point. The place­bo arm saw the lev­el of triglyc­erides go up, help­ing to il­lus­trate the treat­ment ef­fect.

The drug is de­signed to tack­le a dis­ease caused by hered­i­tary mu­ta­tions that in­hib­it the ac­tiv­i­ty of lipopro­tein li­pase, need­ed to break down triglyc­erides car­ried by chy­lomi­crons.

But re­searchers al­so not­ed that five pa­tients were forced out of the tri­al due to a threat­en­ing de­cline in platelet counts. Grade 4 throm­bo­cy­tope­nia oc­curred in three pa­tients, which end­ed af­ter they stopped dos­ing. There were no with­drawals due to platelet counts af­ter the com­pa­ny be­gan mon­i­tor­ing the side ef­fect, but the safe­ty is­sue did not pass un­no­ticed.

“(O)ve­r­all the dis­con­tin­u­a­tion rate in the study was high­er than we had ex­pect­ed, and high­er than pre­vi­ous­ly seen in the COM­PASS safe­ty tri­al in se­vere hy­per­triglyc­eridemia (20%),” not­ed Leerink’s Paul Mat­teis.

Io­n­is’ sub­sidiary Akcea al­so tracked some ad­di­tion­al ben­e­fits, in­clud­ing the end of pan­cre­ati­tis at­tacks dur­ing the 52-week study as well as a re­duc­tion in ab­dom­i­nal pain.

Some an­a­lysts, though, will well re­mem­ber that Io­n­is stunned in­vestors last May with the news that its drug IO­N­IS-TTR­Rx trig­gered throm­bo­cy­tope­nia in pa­tients with TTR amy­loid car­diomy­opa­thy. The FDA then put the pro­gram on a clin­i­cal hold, spurring its part­ner GSK to drop plans to pur­sue a late-stage study. CEO Stan­ley Crook al­so not­ed at the time that re­searchers had seen the same is­sue in their work on volane­sors­en, spurring fears that this could be a class ef­fect.

The biotech’s shares were down 6% by ear­ly Mon­day af­ter­noon.

Since then the com­pa­ny has gone to some lengths to down­play the im­pact of throm­bo­cy­tope­nia, not­ing that physi­cians can mon­i­tor pa­tients and con­trol its im­pact.

Io­n­is has had its ups and downs along the way, but 2016 end­ed on a high note as its part­ner Bio­gen scored an FDA ap­proval for Spin­raza, which costs $750,000 for its first year.

“The suc­cess of AP­PROACH rep­re­sents an im­por­tant mile­stone to­wards our first reg­u­la­to­ry sub­mis­sions for volane­sors­en in the U.S., Eu­rope and Cana­da in 2017,” said Dr. Louis O’Dea, chief med­ical of­fi­cer, Akcea Ther­a­peu­tics.  “We seek to bring this new treat­ment as ex­pe­di­tious­ly as pos­si­ble to FCS pa­tients who have a high un­met need with po­ten­tial­ly life-threat­en­ing con­se­quences.”

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.”