Stéphane Bancel, Moderna CEO (Nancy Lane/The Boston Herald via AP Images, Pool)

Mod­er­na's boost­er plan comes in­to fo­cus with pre-print stud­ies in im­muno­com­pro­mised pop­u­la­tions, Delta break­throughs

Mod­er­na sub­mit­ted ini­tial da­ta to the FDA for its Covid-19 vac­cine boost­er plan ear­li­er this week, and that plan is sharp­en­ing in­to view fol­low­ing sev­er­al pub­lished pre-prints over the last few weeks.

Stud­ies out­lin­ing the Mod­er­na vac­cine’s ef­fec­tive­ness in dif­fer­ent im­muno­com­pro­mised pop­u­la­tions, as well as ex­am­in­ing the shots’ abil­i­ty to pre­vent break­through in­fec­tions, have come to light re­cent­ly. And most, if not all, of the pre-prints show Mod­er­na’s shot net­ting high­er rates at pre­vent­ing Covid-19 than the jab from Pfiz­er and BioN­Tech.

Mod­er­na point­ed to the stud­ies in a press brief­ing Fri­day morn­ing as like­ly to be in­clud­ed in boost­er da­ta sub­mis­sions, tout­ing its re­sults in head-to-head com­par­isons with Pfiz­er.

In a pre-print pub­lished last month look­ing at mR­NA vac­cine ef­fec­tive­ness in more than 1,500 in­di­vid­u­als on main­te­nance dial­y­sis, re­searchers ob­served a 96% sero­log­ic re­sponse among those who re­ceived two dos­es of the Mod­er­na shot. That rate com­pared to an 87% rate in pa­tients who got the Pfiz­er/BioN­Tech shot and 37% in those who re­ceived the J&J vac­cine.

Of the 1,528 peo­ple in the study, 766 re­ceived Mod­er­na shots. An­oth­er 437 pa­tients re­ceived the Pfiz­er/BioN­Tech shot, while 325 got the J&J jab.

An­oth­er pre-print post­ed in Au­gust looked at re­al-world da­ta from vac­cine re­cip­i­ents in Qatar. This study, look­ing specif­i­cal­ly at break­through in­fec­tions due to the Delta vari­ant, found the Mod­er­na shot had much high­er rates at pre­vent­ing symp­to­matic in­fec­tion than the Pfiz­er/BioN­Tech vac­cine.

As of the end of the study on Ju­ly 21, re­searchers had ob­served a 79% ef­fec­tive­ness rate 14 days af­ter one Mod­er­na shot and a 84.8% rate 14 days af­ter the sec­ond shot. Those fig­ures — ob­serv­ing any symp­to­matic break­through case — con­trast­ed with the Pfiz­er/BioN­Tech vac­cine, which saw rates of 64.2% and 53.5%, re­spec­tive­ly.

Ad­di­tion­al­ly, Mod­er­na record­ed 100% ef­fec­tive­ness at pre­vent­ing se­vere dis­ease or hos­pi­tal­iza­tion due to Delta, com­pared to 89.7% for Pfiz­er/BioN­Tech.

As with any pre-print, none of the da­ta have been peer re­viewed.

Mod­er­na an­nounced Wednes­day it had sub­mit­ted boost­er shot plans for a 50 µg dose, low­er than the 100 µg dose cur­rent­ly au­tho­rized un­der emer­gency use. Ear­li­er this week, the com­pa­ny said a 50 Xg boost­er re­sult­ed in an­ti­body titer lev­els “sig­nif­i­cant­ly above the Phase 3 bench­mark.”

The boost­er sub­mis­sion came just a week af­ter the biotech wrapped up its BLA for the orig­i­nal, two-dose reg­i­men. Pfiz­er and BioN­Tech re­ceived full ap­proval for their Covid-19 vac­cine on Aug. 23, and a boost­er shot ad­comm is planned for Sept. 17.

The new ad­comm comes amid con­cerns that Pres­i­dent Joe Biden’s ad­min­is­tra­tion had med­dled with the reg­u­la­to­ry process by un­veil­ing a na­tion­wide boost­er cam­paign ahead of any FDA re­views. The per­ceived over­reach was what, ul­ti­mate­ly, con­tributed to the sud­den de­par­tures of two top of­fi­cials who have been lead­ing the agency’s vac­cine re­view for years, a for­mer se­nior FDA leader said.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

At the beginning of the great pandemic, former NCI chief and inveterate biotech entrepreneur Rick Klausner and the Facebook billionaire would traipse Los Altos Hills in Silicon Valley Saturday mornings and talk about ideas.

Milner’s question on one of those mornings on foot: “What do you want to do?”

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FDA+ roundup: FDA's neu­ro­science deputy de­parts amid on­go­ing Aduhelm in­ves­ti­ga­tions; Califf on the ropes?

Amid increased scrutiny into the close ties between FDA and Biogen prior to the controversial accelerated approval of Aduhelm, the deputy director of the FDA’s office of neuroscience has called it quits after more than two decades at the agency.

Eric Bastings will now take over as VP of development strategy at Ionis Pharmaceuticals, the company said Wednesday, where he will provide senior clinical and regulatory leadership in support of Ionis’ pipeline.

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

Three years after Royalty Pharma chief Pablo Legorreta led a group of investors to buy up a pair of biotechs and create a new startup called ProKidney, the biotech is jumping straight into an $825 million public shell created by SPAC king and tech billionaire Chamath Palihapitiya.

ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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Albert Bourla, Pfizer CEO (Evan Vucci, AP Images)

Covid-19 roundup: Pfiz­er ex­pands in­to France; Omi­cron-spe­cif­ic ver­sion of Mod­er­na's boost­er com­ing soon 

As the hype surrounding Pfizer’s antiviral Covid-19 pill swirls, the pharma announced that it will build a production facility in France to make the drug as a part of a five-year investment.

Pfizer will team up with Novasep to install equipment and initiate tech transfer and on-site development at Novasep’s Mourenx facility. The move is a part of a $594 million investment in France.

“We are of course proud to contribute to the manufacturing of this medicine which has shown in clinical trials to have a positive impact on hospitalization among at-risk Covid-19 patients,” Novasep’s CEO Michel Spagnol said in a statement. “This contract also validates our investment strategy for several years and our focus on small molecules.”