As­traZeneca push­es back OS read­out on MYS­TIC to H2; An­thera shares oblit­er­at­ed in re­peat PhI­II flop

→ The much-an­tic­i­pat­ed over­all sur­vival da­ta of As­traZeneca’s $AZN piv­otal lung can­cer tri­al will be de­liv­ered lat­er than ex­pect­ed, the UK drug­mak­er an­nounced this morn­ing. In­vestors will now have to wait un­til the sec­ond half of 2018 for the fi­nal analy­sis of Imfinzi’s im­pact on OS, in­stead of the first half. The re­sults from the MYS­TIC tri­al re­mains a ma­jor cat­a­lyst af­ter the check­point in­hibitor failed to meet the pri­ma­ry end­point of pro­gres­sion-free sur­vival (PFS), send­ing a shock wave to As­traZeneca’s stock prices last Ju­ly. The tri­al tests Imfinzi both as a monother­a­py and in com­bi­na­tion with treme­li­mum­ab against chemother­a­py in first-line non-small cell lung can­cer, an all-im­por­tant are­na in the bat­tle for check­point drug dom­i­nance.

If at first you don’t suc­ceed in biotech, maybe you should just cut your loss­es and move on. That les­son may be top of mind to­day for An­thera $ANTH af­ter re­searchers re­port­ed that its lead drug Sollpu­ra failed a re­peat Phase III study for cas­es of ex­ocrine pan­cre­at­ic in­suf­fi­cien­cy caused by cys­tic fi­bro­sis. The biotech had al­ready felt the sting of in­vestor dis­sat­is­fac­tion when an ear­li­er Phase III al­so failed. This time sci­en­tists had hoped that more agres­sive dos­ing would fix the prob­lem. It didn’t. An­thera’s shares were evis­cer­at­ed, drop­ping 82% and leav­ing the stock deep in pen­ny stock ter­ri­to­ry.

Malvern, PA-based Realm Ther­a­peu­tics says that their Phase II study of a new ther­a­py for al­ler­gic con­junc­tivi­tis failed, forc­ing the biotech to dump the drug. PR013 was a top­i­cal oph­thalmic so­lu­tion. Realm’s shares (AIM: $RLM) took a dive on the news.

→  Ox­ford Uni­ver­si­ty spin­out OxSy­Bio stepped out to­day with a £10 mil­lion ($13.9 mil­lion) Se­ries A round, join­ing the in­creas­ing­ly crowd­ed space of “tis­sue bio­print­ing” star­tups. This one is de­vel­op­ing a 3D print­er tech­nol­o­gy that can print bi­o­log­i­cal ma­te­ri­als, with the ul­ti­mate goal of mak­ing ther­a­peu­tic tis­sues for pa­tients. This is not un­like the goals of San Diego’s more ma­ture Organo­vo, or the rel­a­tive new­com­er Ste­moniX. OxSy­Bio was found­ed back in 2014 on re­search con­duct­ed in the lab of Pro­fes­sor Ha­gan Bay­ley, the found­ing aca­d­e­m­ic be­hind Ox­ford Nanopore Tech­nolo­gies. The new fi­nanc­ing round was led by Wood­ford In­vest­ment Man­age­ment along­side new and ex­ist­ing back­ers, ac­cord­ing to a com­pa­ny state­ment.


With ad­di­tion­al re­port­ing by Brit­tany Meil­ing and Am­ber Tong.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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FDA ac­cepts pri­or­i­ty re­view of CSL-uniQure he­mo­phil­ia B gene ther­a­py; Fo­s­un Phar­ma nets an­oth­er deal

The first potential gene therapy for hemophilia B has gotten one step closer to reaching patients as the FDA has accepted CSL Behring’s BLA for its uniQure-partnered one-time treatment. The therapy, dubbed etranacogene dezaparvovec, was accepted under “accelerated assessment” by the European Medicines Agency in March.

A priority review will be conducted for the gene therapy intended for patients with the hereditary bleeding disorder, CSL said Tuesday morning. The Pennsylvania biotech did not indicate the regulator’s decision date, but a priority review generally means the treatment’s fate will be handed down within six months rather than 10.

Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”