Bill Hinshaw, Axcella CEO

UP­DAT­ED: Say­ing good­bye to R&D chief, Flag­ship-backed Ax­cel­la drops PhII pro­gram as it fo­cus­es on long Covid, NASH

In its bid to be one of the first biotechs to de­vel­op a drug for long Covid, which im­pacts as many as 1 in 4 el­der Amer­i­cans, Ax­cel­la said Thurs­day it has com­plet­ed en­roll­ment in a Phase IIa study look­ing at fa­tigue-re­lat­ed lin­ger­ing symp­toms of the pan­dem­ic dis­ease.

But the com­pa­ny will have to make progress with its long Covid and NASH mid-stage pro­grams with­out its pres­i­dent of R&D, Al­i­son Schecter. She stepped down im­me­di­ate­ly yes­ter­day to “pur­sue oth­er op­por­tu­ni­ties” af­ter 14 months with the Flag­ship-found­ed biotech, ac­cord­ing to an SEC fil­ing, and Ax­cel­la doesn’t cur­rent­ly have plans to re­place her, CEO Bill Hin­shaw told End­points News in an emailed state­ment.

The fo­cus on long Covid and NASH is al­so con­sum­ing most of Ax­cel­la’s re­sources. The biotech ter­mi­nat­ed a Phase II tri­al of a dif­fer­ent drug, AXA1665, in pa­tients with overt he­pat­ic en­cephalopa­thy, a com­pli­ca­tion of chron­ic liv­er dis­ease. Ax­cel­la will look for part­ners or new in­di­ca­tions to test the drug in, Hin­shaw said in a press re­lease.

Asked if the OHE tri­al ter­mi­na­tion will lead to lay­offs, Hin­shaw said: “Any avail­able re­sources will be uti­lized as we com­plete the Long COVID tri­al, pre­pare for a fol­low-on tri­al, and con­tin­ue to ex­e­cute on the NASH tri­al. ”

The 40-pa­tient, Ox­ford Uni­ver­si­ty-led long Covid tri­al is test­ing AXA1125, which is al­so in a Phase IIb study in NASH. Topline da­ta from the long Covid tri­al are planned for the “ear­ly” third quar­ter of this year and in­ter­im re­sults from the NASH study are slat­ed for lat­er that quar­ter.

“Achiev­ing com­ple­tion of en­roll­ment is a sig­nif­i­cant mile­stone in the de­vel­op­ment path of AXA1125 as a po­ten­tial treat­ment for Long Covid, a large and grow­ing con­se­quence of the glob­al pan­dem­ic,” said the com­pa­ny in a press re­lease.

The drug showed “key path­ways” in pre­clin­i­cal and clin­i­cal de­vel­op­ment in NASH stud­ies, which led the biotech to al­so test it as a po­ten­tial long Covid ther­a­peu­tic.

“So fat­ty acid ox­i­da­tion, basal res­pi­ra­tion, pref­er­en­tial ATP gen­er­a­tion, in­flam­ma­tion and oth­ers. And then we saw what was emerg­ing as the pan­dem­ic con­tin­ued to build, which was that one of the core dri­vers of long Covid was clear­ly mi­to­chon­dr­i­al dys­func­tion in those oth­er path­ways with­in that that I was speak­ing to,” CEO and pres­i­dent Bill Hin­shaw told End­points News in an in­ter­view last month.

Mar­garet Koziel

Right now, for the mil­lions of pa­tients with long Covid, the on­ly treat­ment op­tions are sup­port­ive care, CMO Mar­garet Koziel said in the joint in­ter­view with Hin­shaw.

“This is just stag­ger­ing in terms of its pub­lic health im­pact, and the abil­i­ty to get so­ci­ety back on its feet again, so we be­lieve that this is like­ly to be a com­plex phe­nom­e­non. We have ev­i­dence in the clin­ic of tar­get­ing mul­ti­ple rel­e­vant bi­o­log­ic path­ways and so we’re aim­ing to move as quick­ly as we can,” Koziel said.

If all pans out, Ax­cel­la could be one of the first drug de­vel­op­ers to get across the fin­ish line when it comes to treat­ing SARS-CoV-2’s long-term ef­fects on the body.

Paul Sekhri

“We’ve been hear­ing from the pa­tient com­mu­ni­ty about what the med­ical needs are and start­ing to think about how we would move this in­to a lat­er phase study, as­sum­ing that this pi­lot study is pos­i­tive,” Koziel said.

And the com­pa­ny’s board looks a lit­tle dif­fer­ent as of this week, ac­cord­ing to an SEC fil­ing.

Shree­r­am Arad­hye left the board on May 19 and, that same day, Ax­cel­la’s board ap­point­ed Paul Sekhri and Michael Rosen­blatt to join as Class II and Class I di­rec­tors, re­spec­tive­ly. Sekhri re­cent­ly stepped down as CEO of xeno­trans­plan­ta­tion biotech eGe­n­e­sis, and Rosen­blatt is a se­nior part­ner at Flag­ship Pi­o­neer­ing and for­mer Mer­ck CMO.

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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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.

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.

Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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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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Rwanda president Paul Kagame and BioNTech CEO Uğur Şahin (via BioNTech)

BioN­Tech breaks ground on first mR­NA vac­cine man­u­fac­tur­ing fa­cil­i­ty in Africa

Covid vaccine access to lower- and middle-income nations has been a concern during the length of the pandemic, but BioNTech is now pushing forward with plans to increase vaccine access for Africa.

Construction work has kicked off for an mRNA manufacturing facility in Kigali, Rwanda. According to BioNTech, the facility, dubbed the African modular mRNA manufacturing facility, has a target for the first set of manufacturing tools to be delivered to the site by the end of this year.